What changed in this filing

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Item 1. Business

2026-03-16 compared with 2025-03-06 · 234 added, 237 removed, 600 unchanged (44% of the section changed)

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Our compounds target families of oncogenic mutations in clinically validated pathways.

Our lead clincal-stage program, BDTX-1535, a brain-penetrant, fourth-generation epidermal growth factor receptor (EGFR) MasterKey inhibitor, is currently being studied in a Phase 2 clinical trial in patients with epidermal growth factor receptor mutant (EGFRm) non-small cell lung cancer (NSCLC) and in an investigator-sponsored trial in patients with glioblastoma (GBM) with EGFR alterations.

We are actively evaluating partnership opportunities for a second clinical-stage program, BDTX-4933, a brain-penetrant, RAF MasterKey inhibitor targeting KRAS, NRAS and BRAF alterations in solid tumors.

Our lead clinical-stage program, silevertinib (formerly BDTX-1535), a brain-penetrant, fourth-generation epidermal growth factor receptor (EGFR) MasterKey inhibitor, is currently being studied in a Phase 2 clinical trial in patients with epidermal growth factor receptor mutant (EGFRm) non-small cell lung cancer (NSCLC).

We plan to initiate a randomized Phase 2 trial of silevertinib in newly diagnosed patients with EGFR altered glioblastoma (GBM) in the second quarter of 2026.

The Black Diamond Therapeutics approach

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We have a pipeline of orally available, potent and selective small molecule MasterKey inhibitors that target families of driver mutations in individual oncogenes for the treatment of cancer and genetic diseases.

Our pipeline includes two clinical-stage product candidates and one development-stage product candidate.

In addition to our lead program, silevertinib, our pipeline also includes an outlicensed clinical-stage product candidate and one development-stage product candidate.

An overview of our pipeline is shown in the table below.

a brain-penetrant, irreversible EGFR MasterKey inhibitor with broad mutation coverage

We believe that BDTX-1535 has the potential to treat newly diagnosed patients with EGFRm NSCLC as well as those with recurrent disease, based upon BDTX-1535’s ability to address greater than 50 classical and non-classical oncogenic driver mutations with greater potency than other EGFR tyrosine kinase inhibitors (TKI’s), as well as uniquely target the C797S resistance mutation which can be acquired after treatment with osimertinib.

Historically, EGFR inhibitors have demonstrated significant clinical benefit in patients with classical EGFR activating mutations and are the current first-line standard of care.

Silevertinib:

a brain-penetrant, irreversible EGFR MasterKey inhibitor with potential to treat both EGFRm NSCLC and EGFR altered GBM

We believe that silevertinib has the potential to treat newly diagnosed patients with EGFRm NSCLC as well as those with recurrent disease, based upon silevertinib’s ability to address greater than 50 classical and non-classical oncogenic driver mutations with greater potency than other EGFR tyrosine kinase inhibitors (TKI’s), as well as uniquely target the C797S resistance mutation which can be acquired after treatment with osimertinib.

Historically, EGFR inhibitors have demonstrated significant clinical benefit in patients with classical EGFR activating mutations and are the current frontline standard of care.

However, over time, almost all patients acquire resistance and relapse.

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The majority of first and second-generation EGFR inhibitors do not adequately penetrate the CNS.

While the third-generation EGFR inhibitor osimertinib has been shown to exhibit some CNS penetration and delay the progression of CNS metastases, there are few options remaining to patients when resistance presents due to the acquired resistance mutation C797S or to “P-loop αC-helix compressing” mutations (PACC, a subset of non-classical mutations) that are insensitive to osimertinib.

BDTX-1535 has been observed to be highly brain-penetrant in preclinical tumor models and in the clinical setting.

There are presently no therapies approved for the treatment of patients with EGFRm NSCLC harboring the C797S mutation.

BDTX-1535 received Fast Track Designation from the U.S.

While the third-generation EGFR inhibitor osimertinib has been shown to exhibit some CNS penetrance and delay the progression of CNS metastases, there are few options remaining to patients with “P-loop αC-helix compressing” mutations (PACC, a subset of non-classical mutations) that are insensitive to osimertinib.

Moreover, there are no therapies approved for the treatment of patients when resistance to osimertinib presents due to the acquired resistance mutation C797S.

Silevertinib has been observed to be highly brain-penetrant in the clinical setting.

Silevertinib has also received Fast Track Designation from the U.S.

Food and Drug Administration (FDA) for the treatment of patients with EGFR mutant C797S-positive NSCLC whose disease has progressed on/after a third-generation EGFR TKI.

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Time to treatment discontinuation for patients with non-classical mutations was < 8 months, which is much shorter than the > 14 months observed for patients with a classical mutation who were receiving osimertinib.

Our preclinical data for BDTX-1535 showed greater potency against these non-classical mutations, including “P-loop αC-helix compressing” mutations (PACC), than currently available EGFR TKI’s that are used either off label or for a narrowly defined set of mutations in the first-line setting.

In the first quarter of 2024, we initiated a Phase 2 trial in newly-diagnosed patients with non-classical mutations.

Initial results from this trial in patients with newly-diagnosed EGFRm NSCLC are anticipated in the second quarter of 2025.

We have recently completed enrollment of BDTX-1535 in a Phase 2 clinical trial of 83 patients with EGFRm NSCLC in the second- and third-line settings.

In the third quarter of 2024, we announced initial data from this trial demonstrating encouraging clinical responses and durability of BDTX-1535 in patients with recurrent EGFRm NSCLC.

Based on pharmacokinetics, safety and tolerability data from the Phase 2 trial, the 200 mg daily dose of BDTX-1535 was selected for pivotal development, showing robust EGFRm target coverage and a favorable tolerability profile with no new safety signals observed.

The majority of adverse events were mild or moderate, and no new safety signals were observed.

The most common on-target treatment-related adverse events were rash (70%) and diarrhea (35%).

There were two cases of grade 3 rash, and no reported cases of grade 4 rash or grade 3/4 diarrhea.

Based on an August 2024 data cutoff, a preliminary overall response rate (ORR) of 42% was seen in 19 patients with known osimertinib resistance EGFR mutations (PACC “P-loop αC-helix compressing” and C797S mutations).

Acquisition of C797S was frequently observed in patients who progressed following treatment with osimertinib.

PACC mutations represent a structure-function group of non-classical oncogenic driver mutations which may accumulate or be acquired following treatment with osimertinib.

Encouraging durability was noted with a duration of response (DOR) of approximately eight months or more in the first three patients who achieved a partial response (PR), while 14 of the 19 patients remained on treatment.

We expect to present updated results from this trial in the second half of 2025.

In the second quarter of 2024, at the American Society of Clinical Oncology (ASCO) Annual Meeting, we presented preliminary results from the Phase 1 trial of BDTX-1535 in patients with relapsed/recurrent GBM, demonstrating encouraging duration of treatment and clinical activity, and a tolerability profile consistent with the initial safety data from the dose escalation portion of the Phase 1 trial.

Our collaborators at the Ivy Brain Tumor Center also presented initial intratumoral pharmacokinetic data from a “window of opportunity” study in patients with recurrent high-grade glioma (HGG) with EGFR alterations and/or fusions at initial diagnosis.

This study, also known as a Phase 0/1 “Trigger” trial, is sponsored by the Ivy Brain Tumor Center in Phoenix, Arizona.

Initial results from this investigator-sponsored trial demonstrated that BDTX-1535 exceeded the pre-specified threshold for drug concentration in the brain tumor tissue and was generally well tolerated.

In the fourth quarter of 2024, at the European Association of Neuro-Oncology (EANO) Annual Meeting and the Society of Neuro-Oncology (SNO) Annual Meeting, the Ivy Brain Tumor Center presented additional promising data from the trial which demonstrated that BDTX-1535 penetrates rarely accessible regions of glioblastoma and suppresses EGFR signaling in patient tumors.

The study, sponsored by the Ivy Brain Tumor Center, is expected to expand into a “Phase 0/2” trial in newly diagnosed glioblastoma patients with EGFR alterations in the first quarter of 2025.

a highly selective, brain-penetrant RAF MasterKey inhibitor

BDTX-4933 is designed to be a potent and selective, reversible oral inhibitor that targets broad families of oncogenic BRAF, KRAS and NRAS alterations.

Our preclinical data for silevertinib showed greater potency against these non-classical mutations, including “P-loop αC-helix compressing” mutations (PACC), than currently available EGFR TKI’s that are used either off label or for a narrowly defined set of mutations in the frontline setting.

Silevertinib is currently being studied in a Phase 2 clinical trial in patients with EGFRm NSCLC in both the frontline and recurrent settings.

In the fourth quarter of 2025, we announced initial data from our Phase 2 trial of 43 frontline NSCLC patients harboring a broad spectrum of 35 distinct non-classical EGFR mutations, including 16 patients with brain metastases (7 of whom had measurable CNS target lesions).

All patients were enrolled at a 200 mg oral daily dose of silevertinib.

Efficacy and safety were assessed with a November 3, 2025 data cutoff and median follow-up time as of this date was 7.2 months.

The study is fully enrolled and remains ongoing.

The initial data from this trial was encouraging with 25 confirmed partial responses and 1 confirmed complete response equating to a 60% Objective Response Rate (ORR by RECIST 1.1).

CNS ORR (by RANO-BM) was 86% and the disease control rate (DCR) was 91%.

No new safety signals were observed.

Adverse events (AEs) experienced by a majority of patients include rash, stomatitis, diarrhea and paronychia and were managed with standard supportive care and dose interruptions or reductions without compromising response depth or durability.

As of the November 3, 2025 data cutoff, 29 patients remained on therapy (5 of 29 after progression) with one patient having been on therapy for more than 19 months.

We plan to present updated results from the Phase 2 NSCLC trial, including preliminary duration of response (DOR) and progression-free survival (PFS) data in the frontline setting (43 patients) as well as updated clinical results in the recurrent setting (83 patients), at a medical meeting in the second quarter of 2026.

Silevertinib has demonstrated encouraging CNS activity in multiple trials across NSCLC and GBM.

In our Phase 2 clinical trial in frontline patients with EGFRm NSCLC 86% of patients achieved a confirmed CNS response.

The results from our Phase 1 trial of silevertinib in patients with relapsed/recurrent GBM (presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in 2024) showed encouraging duration of treatment and clinical activity, and a tolerability profile consistent with the initial safety data seen in patients with recurrent NSCLC.

A Phase 0/1 “window of opportunity” study sponsored by the Ivy Brain Tumor Center in Phoenix, Arizona in patients with recurrent high-grade glioma (HGG) with EGFR alterations and/or fusions at initial diagnosis demonstrated that silevertinib exceeded the pre-specified threshold for drug concentration in the brain tumor tissue and was generally well tolerated.

It also showed that silevertinib penetrates non-contrast enhancing regions of glioblastoma and suppresses EGFR signaling in patient tumors.

Based on these data, together with the robust CNS ORR demonstrated by silevertinib to date in our Phase 2 trial in frontline EGFRm NSCLC patents, we believe that silevertinib is uniquely positioned as a potential treatment for patients with newly diagnosed EGFR-altered GBM.

Following feedback on the study design received from the FDA in January 2026, we are preparing to initiate a randomized Phase 2 trial in this patient population in the second quarter of 2026.

After a combination safety lead-in, the trial is expected to enroll approximately 150 newly diagnosed patients, randomized to receive temozolomide (TMZ) (as the control arm) or silevertinib plus TMZ (as the experimental arm).

The eligible patient population will be on EGFRvIII-positive patients (approximately 30% of GBM patients) who are O-6-methylguanine-DNA methyltransferase (MGMT) -negative (unmethylated).

Randomization and treatment will begin after patients have had surgical resection and radiation and are eligible for maintenance TMZ.

The primary endpoint of the trial will be PFS (RANO by blinded independent committee review, or BICR), with a planned futility analysis, and an interim PFS analysis anticipated in the first half of 2028.

The secondary endpoint of the trial will be overall survival (OS).

The trial will be governed by an independent data monitoring committee (IDMC).

We are also continuing to explore potential partnership opportunities to advance silevertinib into pivotal development.

a highly selective, brain-penetrant RAF MasterKey inhibitor – global rights licensed to Servier

BDTX-4933 (also known as S241656) was designed to be a potent and selective, reversible oral inhibitor that targets broad families of oncogenic BRAF, KRAS and NRAS alterations.

BDTX-4933 selectively targets constitutively active RAF dimers resulting from either BRAF mutations or other upstream oncogenic MAPK pathway alterations, such as KRAS and NRAS alterations.

In preclinical tumor models, we observed that BDTX-4933 demonstrated brain-penetrant activity and achieved regression of tumors carrying a broad spectrum of KRAS mutations, NRAS alterations, as well as BRAF Class I, II, and III mutations.

We initiated a Phase 1 clinical trial for BDTX-4933 in the second quarter of 2023 in patients with BRAF and select KRAS and NRAS mutation-positive cancers, with an emphasis on patients with non-G12C KRAS mutant NSCLC.

In the fourth quarter of 2024, we announced we are deprioritizing BDTX-4933 and actively seeking partnerships for this asset, as we focus our resources on our lead program, BDTX-1535.

Early-stage programs

The program was in a Phase 1 clinical trial in patients with BRAF and select KRAS and NRAS mutation-positive cancers, with an emphasis on patients with non-G12C KRAS mutant NSCLC when it was outlicensed to Servier Pharmaceuticals LLC (Servier) in the first quarter of 2025.

Pursuant to the license agreement, we granted to Servier a global license to develop and commercialize BDTX-4933.

Under the terms of the license agreement, Servier will lead the development activities and the global commercialization of BDTX-4933 across multiple indications, including NSCLC, with potential applications in other solid tumors.

In consideration for the license granted to Servier, we received an upfront payment of $70.0 million in March 2025 and will be eligible to receive up to $710.0 million in development and commercial sales milestone payments, along with tiered royalties based on global net sales.

We are exploring partnership opportunities for our FGFR2/3 selective development candidate BDTX-4876.

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The critical components of our strategy include:

• Generate Phase 2 clinical trial results for BDTX-1535 in patients with EGFRm NSCLC to enable a potential pivotal trial.

We believe that the ability of BDTX-1535 to target approximately 50 oncogenic mutations that are not sufficiently addressed by existing therapies, as well as its oral administration and tolerability profile, offers significant potential to treat patients with EGFRm NSCLC.

Based on initial clinical data from newly diagnosed patients anticipated in the second quarter of 2025, we plan to seek regulatory feedback on a potential registrational path for BDTX-1535 in first-line EGFRm NSCLC patients with non-classical mutations in the second half of 2025.

We also expect to complete the evaluation of BDTX-1535 in patients with recurrent EGFRm NSCLC and explore potential combination opportunities to maximize the therapeutic potential of BDTX-1535 in the recurrent setting.

• Generate clinical data for BDTX-1535 in first-line patients with EGFR altered GBM to support a potential pivotal study.

Based on results from the Phase 0/2 “window of opportunity” trial for BDTX-1535 in newly diagnosed patients with GBM with EGFR alterations anticipated to start in the first quarter of 2025, we anticipate planning for a potential pivotal trial of BDTX-1535 as a first-line treatment in that setting.

• Evaluate potential strategic partnerships to maximize the value of our pipeline and to provide non-dilutive funding for the further development of BDTX-1535.

We are actively evaluating potential partnership opportunities for BDTX-4933, our Phase 1 clinical program targeting KRAS, NRAS and BRAF alterations.

We are also exploring potential partnership opportunities and strategic alternatives for our FGFR program, BDTX-4876.

• Generate Phase 2 clinical trial results for silevertinib in patients with EGFRm NSCLC to enable a potential pivotal trial.

We believe that the ability of silevertinib to target approximately 50 oncogenic mutations that are not sufficiently addressed by existing therapies, as well as its oral administration, tolerability profile, CNS penetrance, and clinical data generated to date, offers significant potential to treat patients with EGFRm NSCLC.

We expect to seek guidance from the FDA on a potential pivotal trial design in the frontline setting, based on preliminary DOR and PFS data anticipated in the second quarter of 2026.

• Conduct a Phase 2 clinical trial for silevertinib in newly diagnosed patients with EGFR altered GBM to support a potential pivotal study.

Silevertinib has demonstrated encouraging CNS activity in multiple trials across NSCLC and GBM, and we believe that it is uniquely positioned as a potential treatment for patients with newly diagnosed EGFR-altered GBM, a disease with significant unmet need.

We plan to initiate a randomized Phase 2 trial in newly diagnosed GBM patients in the second quarter of 2026, with interim efficacy data expected in the first half of 2028.

• Evaluate potential strategic partnerships for the pivotal development of silevertinib and to maximize the value of our pipeline.

We are exploring potential partnerships for the further development of silevertinib, including a potential pivotal trial in frontline EGFRm NSCLC.

We also continue to review potential partnership opportunities and strategic alternatives for our FGFR program, BDTX-4876, as well as other transactions that may provide non-dilutive funding to the company.

Our history and team

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We have a pipeline of orally available, potent and selective small molecule MasterKey therapies that target genetic drivers in several cancers.

We own worldwide commercial rights to all of our product candidates.

We own worldwide commercial rights to silevertinib and our FGFR2/3 selective development candidate, BDTX-4876.

Silevertinib:

a brain-penetrant, mutant selective, irreversible EGFR MasterKey inhibitor targeting EGFR classical, non-classical, and acquired resistance mutations in NSCLC and EGFR mutations expressed in GBM

Background and limitations of current EGFR inhibitors

In NSCLC, EGFR inhibitors have demonstrated significant clinical benefit in patients with classical EGFR activating mutations (Exon19del, L858R) and are the current first-line standard of care.

In NSCLC, EGFR inhibitors have demonstrated significant clinical benefit in patients with classical EGFR activating mutations (Exon19del, L858R) and are the current frontline standard of care.

However, over time, almost all patients acquire resistance and relapse.

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Our solution:

BDTX-1535 is a potent, selective, irreversible, oral and brain-penetrant small molecule inhibitor designed to address the critical unmet need in NSCLC and GBM driven by EGFR alterations.

The pharmacological activity of BDTX-1535 was optimized to inhibit a wide spectrum of EGFR mutations that drive resistance, and to target CNS tumors through its potential for high brain exposure.

Silevertinib is a potent, selective, irreversible, oral and brain-penetrant small molecule inhibitor designed to address the critical unmet need in NSCLC and GBM driven by EGFR alterations.

The pharmacological activity of silevertinib was optimized to inhibit a wide spectrum of EGFR mutations that drive resistance, and to target CNS tumors through its potential for high brain exposure.

EGFR is a potent oncogene commonly altered in many cancers, including NSCLC and GBM.

EGFR mutations in NSCLC commonly impact the kinase domain, while in GBM they occur primarily in the extracellular domain.

In cell-based assays, BDTX-1535 achieved potent inhibition of families of oncogenic EGFR mutations and selectivity versus normally expressed EGFR WT as compared to osimertinib.

In cell-based assays, silevertinib achieved potent inhibition of families of oncogenic EGFR mutations and selectivity versus normally expressed EGFR WT as compared to osimertinib.

This includes classical, non-classical, acquired resistance, and complex mutations expressed in NSCLC.

BDTX-1535 is not a potent inhibitor of Exon20 insertions or the T790M resistance mutation.

In cell-based assays, BDTX-1535 achieved potent MasterKey inhibition of a family of oncogenic EGFR variants expressed in GBM and EGFR amplification with selectivity versus normally expressed EGFR WT.

Silevertinib is not a potent inhibitor of Exon20 insertions or the T790M resistance mutation.

In cell-based assays, silevertinib achieved potent MasterKey inhibition of a family of oncogenic EGFR variants expressed in GBM and EGFR amplification with selectivity versus normally expressed EGFR WT.

Clinical development

The IND application for BDTX-1535 was cleared by the FDA in the first quarter of 2022, and the first-in-human clinical trial (BDTX1535-101) was also initiated in the first quarter of 2022.

The IND application for silevertinib was cleared by the FDA in the first quarter of 2022, and the first-in-human clinical trial (BDTX1535-101) was also initiated in the first quarter of 2022.

The Phase 1 dose escalation part of this trial enrolled patients harboring EGFR oncogenic alterations both in NSCLC and GBM and was completed in 2023.

Clinical data from the dose escalation portion of the trial demonstrated promising clinical activity and a favorable tolerability profile for BDTX-1535 in the recurrent setting.

In the third quarter of 2023, we began the BDTX-1535 Phase 2 NSCLC clinical trial to assess ORR by RECIST 1.1 and DOR in patients with NSCLC in two initial cohorts:

Clinical data from the dose escalation portion of the trial demonstrated promising clinical activity and a favorable tolerability profile for silevertinib in the recurrent setting.

In the third quarter of 2023, we began the silevertinib Phase 2 NSCLC clinical trial to assess ORR by RECIST 1.1 and DOR in patients with NSCLC in two initial cohorts:

one cohort with the EGFR acquired resistance C797S mutation after progression on a third-generation EGFR TKI, and the other cohort in non-classical driver mutations (NCMs) after progression on an EGFR TKI, in each case with and without brain metastases.

In September 2024, we announced initial data from the Phase 2 trial demonstrating encouraging clinical responses and durability of BDTX-1535 in the second- and third-line settings.

The majority of adverse events were mild or moderate, and no new safety signals were observed.

The most common on-target treatment-related adverse events were rash (70%) and diarrhea (35%).

There were two cases of grade 3 rash, and no reported cases of grade 4 rash or grade 3/4 diarrhea.

In the third quarter of 2024, we announced initial data from the Phase 2 trial demonstrating encouraging clinical responses and durability of silevertinib in the second- and third-line settings.

Based on an August 2024 data cutoff, a preliminary ORR of 42% was seen in 19 patients with known osimertinib resistance EGFR mutations (PACC “P-loop αC-helix compressing” and C797S mutations).

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Encouraging durability was noted with a DOR of approximately eight months or more in the first three patients who achieved a PR, while 14 of the 19 patients remained on treatment as of the cut-off date.

Data from September 23, 2024 disclosure;

*Retrospective liquid and tissue biopsy NGS testing;

Pt 2118 withdrew consent prior to first scan (see patient in swimmer plot);

O-osimertinib;

C- carboplatin, Cis – cisplatin, Pem- pemetrexed;

Pac- paclitaxel;

B- bevacizumab;

HER3-Dxd- patritumab deruxtecan;

Data from September 23, 2024 disclosure;

N=19 in swimmer plot, including Pt 2118 who withdrew consent (WC) prior to first scan *Patient discontinued (pneumonitis)

We have recently completed enrollment of BDTX-1535 in 83 patients with recurrent EGFRm NSCLC, and expect to present updated results in the second half of 2025.

We are also exploring potential combination opportunities for BDTX-1535 in the recurrent setting.

Following feedback from the FDA, in the first quarter of 2024, we also initiated a Phase 2 cohort in first-line patients with NSCLC harboring non-classical mutations.

The cohort can enroll up to 40 patients at a 200 mg dose.

Initial results from the first-line cohort are anticipated in the second quarter of 2025.

We intend to meet with FDA as results become available from the Phase 2 first-line cohort in patients with NSCLC to discuss a potential registrational path.

The majority of adverse events were mild or moderate, and no new safety signals were observed.

The most common on-target treatment-related adverse events were rash (70%) and diarrhea (35%).

There were two cases of grade 3 rash, and no reported cases of grade 4 rash or grade 3/4 diarrhea.

We completed enrollment of silevertinib in 83 patients with recurrent EGFRm NSCLC, and expect to report final data at a medical meeting in the second quarter of 2026.

Following feedback from the FDA, in the first quarter of 2024, we also initiated a Phase 2 cohort in frontline patients with NSCLC harboring non-classical mutations.

Enrollment in this cohort was completed in July 2025 and in December 2025, we announced initial clinical data.

43 frontline NSCLC patients were enrolled harboring a broad spectrum of 35 distinct non-classical EGFR mutations, including 16 patients with brain metastases (7 of whom had measurable CNS target lesions).

All patients were enrolled at a 200mg oral daily dose of silevertinib.

Efficacy and safety were assessed with a November 3, 2025 data cutoff and median follow-up time as of the November 2025 data cutoff was 7.2 months.

ORR by RECIST 1.1 was 60% with 25 confirmed partial responses and 1 confirmed complete response.

CNS ORR (by RANO-BM) was 86% and the DCR was 91%.

No new safety signals were observed.

AEs experienced by a majority of patients include rash, stomatitis, diarrhea and paronychia and were managed with standard supportive care and dose interruptions or reductions without compromising response depth or durability.

As of the November 2025 data cutoff, 29 patients remained on therapy (5 of 29 after progression) with one patient having been on therapy for more than 19 months.

Data from November 3, 2025 cut.

(1) Patient awaiting confirmatory scan;

if confirmed, CNS ORR is 100%.

3 patients not on waterfall plot are not evaluable (NE).

(2) Patients with compound mutations are classified as “PACC” if at least one PACC mutation is present.

Data from November 3, 2025 cut.

(1) Patients with compound mutations are classified as “PACC” if at least one PACC mutation is present.

We plan to present updated results from the Phase 2 NSCLC trial, including DOR and PFS data in the frontline setting (43 patients), at a medical meeting in the second quarter of 2026.

GBM is a difficult-to-treat, aggressive malignancy of the central nervous system.

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In preclinical models, we have shown that the mechanism of activation for these EGFR oncogenic alterations involves the formation of a constitutive dimer that exhibits a conformation leading to ligand-independent signaling shared by a family of extracellular domain EGFR alterations expressed in GBM.

BDTX-1535 was designed to overcome these challenges and address the significant unmet need for new treatments for patients with GBM.

Silevertinib was designed to overcome these challenges and address the significant unmet need for new treatments for patients with GBM.

In the fourth quarter of 2023, we announced dose escalation results in 22 patients with GBM demonstrating clinical activity in heavily pre-treated patients, including 1 confirmed partial response and 8 patients with stable disease among 19 patients with measurable disease by RANO criteria.

Of 22 patients evaluable for efficacy, 3 patients were shown to be on therapy longer than 10 months, 1 patient longer than 6 months, and 5 patients longer than 4 months.

Importantly, historical progression free survival (PFS) in this population is expected to last approximate 2-4 months.

BDTX-1535 was shown to be generally well tolerated, with no new safety signals observed.

In the second quarter of 2024, at the ASCO Annual Meeting, we presented preliminary data from the Phase 1 trial of BDTX-1535 in patients with relapsed/recurrent GBM, demonstrating encouraging duration of treatment and clinical activity, and a tolerability profile consistent with the initial safety data from the dose escalation portion of the Phase 1 trial presented in 2023.

Also, in the second quarter of 2024, initial results were presented at the ASCO Annual Meeting from an investigator (Ivy Brain Tumor Center) sponsored “window of opportunity” trial of BDTX-1535 in patients with recurrent high-grade glioma with EGFR alterations and/or fusions at initial diagnosis and who underwent surgical intracranial tumor resection.

The data presented demonstrated that BDTX-1535 exceeded the pre-specified threshold for drug concentration in the brain tumor tissue and was generally well tolerated with expected EGFR-mediated side effects.

Additional promising results from this trial were presented by the investigator at the EANO and SNO meetings in the fourth quarter of 2024.

The data demonstrated that BDTX-1535 penetrates rarely accessible regions of glioblastoma and suppresses EGFR signaling in patient tumors.

In the first quarter of 2025, the program is expected to expand into a Phase 0/2 “window of opportunity” trial in newly diagnosed glioblastoma patients with EGFR aberrations.

a highly selective, brain-penetrant RAF MasterKey inhibitor targeting oncogenic mutations in KRAS, NRAS, and BRAF Class I, II, III

Background and limitations of RAF inhibitors

BRAF mutations are among the most common mutations found in tumors.

Oncogenic alterations affecting BRAF include the V600E mutation (Class I) active site mutation together with families of non-canonical BRAF mutations (Class II and Class III) that are active as dimers.

While the V600E Class I mutation has been successfully targeted in melanoma and other solid tumors, there are currently no approved therapies that target the full spectrum of Class II and Class III mutations that are expressed in melanoma and a range of other solid tumors.

Additionally, expression of all classes of BRAF mutations commonly occurs in patients with CNS tumors or with brain metastasis, which currently remain unaddressed by approved drugs due to their poor brain penetration.

Approved BRAF inhibitors can lead to unwanted paradoxical activation, which may lead to poor efficacy and secondary malignancies.

Our solution:

BDTX-4933 is designed as a brain-penetrant, oral small molecule MasterKey inhibitor of oncogenic KRAS, NRAS and BRAF Class I, II and III mutations, while avoiding paradoxical activation.

We believe that BDTX-4933 could offer an improved approach for treating solid tumors expressing activating MAPK pathway alterations, including those with brain metastases.

In cell-based assays, BDTX-4933 demonstrated potent inhibition of a wide spectrum of BRAF alterations including fusions and exhibited dose-dependent inhibition of cell proliferation.

In preclinical tumor models expressing KRAS, NRAS, and BRAF Class I, II and III mutations, daily dosing of BDTX-4933 demonstrated dose-dependent tumor growth inhibition, tumor regression and survival advantage consistent with potent on-target and on-pathway inhibition.

BDTX-4933 also demonstrated robust brain penetration properties and activity in intracranial mouse tumor models expressing the Class I V600E mutation.

In the fourth quarter of 2023, we presented mechanism of action data for BDTX-4933 at the European Organization for Research and Treatment of Cancer-National Cancer Institute-American Association for Cancer Research (EORTC-NCI-AACR) Symposium on Molecular Targeted and Cancer Therapeutics demonstrating that BDTX-4933 forms a “RAS/RAF clamp” by blocking activating RAS mutations upstream and locking it in an inactivated form.

We believe this is a differentiated mechanism of action within the RAF and RAS landscape.

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Clinical development

The IND for BDTX-4933 was cleared by the FDA in the first quarter of 2023 and, in the second quarter of 2023, we initiated a Phase 1 clinical trial for BDTX-4933 to evaluate the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of BDTX-4933 in RAF/RAS-mutant solid tumors.

In the fourth quarter of 2024, we announced that we are deprioritizing BDTX-4933 and would seek partnerships to focus resources on our lead program, BDTX-1535.

Importantly, historical PFS in this population is expected to last approximate 2-4 months.

Silevertinib was shown to be generally well tolerated, with no new safety signals observed.

In the second quarter of 2024, at the ASCO Annual Meeting, we presented preliminary data from the Phase 1 trial of silevertinib in patients with relapsed/recurrent GBM, demonstrating encouraging duration of treatment and clinical activity, and a tolerability profile consistent with the initial safety data released in 2023.

Also, in the second quarter of 2024, initial results were presented at the ASCO Annual Meeting from an investigator (Ivy Brain Tumor Center) sponsored Phase 0/1 “window of opportunity” trial of silevertinib in patients with recurrent high-grade glioma with EGFR alterations and/or fusions at initial diagnosis and who underwent surgical intracranial tumor resection.

The data presented demonstrated that silevertinib exceeded the pre-specified threshold for drug concentration in the brain tumor tissue and was generally well tolerated with expected EGFR-mediated side effects.

Additional promising results from this trial were presented by the Ivy Brain Tumor Center at the European Association of Neuro-Oncology (EANO) annual meeting in the fourth quarter of 2024, and at the EANO, the Society of Neuro-Oncology (SNO), and the American Association for Cancer Research (AACR) annual meetings in 2025.

These data demonstrated that silevertinib penetrates non-contrast enhancing regions of glioblastoma and suppresses EGFR signaling in patient tumors.

In March 2025, the Phase 0/1 trial was modified by the Ivy Brain Tumor Center to include newly diagnosed GBM patients with EGFR alterations.

Based on these data and together with the robust CNS ORR demonstrated by silevertinib to date in our Phase 2 trial in frontline EGFRm NSCLC patents, we believe that silevertinib is uniquely positioned as a potential treatment for patients with newly diagnosed EGFR-altered GBM.

Following feedback on the study design received from the FDA in January 2026, we are preparing to initiate a randomized Phase 2 trial in this patient population in the second quarter of 2026.

After a combination safety lead-in of silevertinib plus temozolomide (TMZ), the trial is expected to enroll approximately 150 newly diagnosed patients, randomized to receive TMZ (as the control arm) or silevertinib plus TMZ (as the experimental arm).

The eligible patient population will be on EGFRvIII-positive patients (approximately 30% of GBM patients) who are O-6-methylguanine-DNA methyltransferase (MGMT) -negative (unmethylated).

Randomization and treatment will begin after patients have had surgical resection and radiation and are eligible for maintenance TMZ.

The primary endpoint of the trial will be PFS (RANO by BICR), with a planned futility analysis, and an interim PFS analysis anticipated in the first half of 2028.

The secondary endpoint of the trial will be OS.

The trial will be governed by an IDMC.

Early-stage programs

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We face competition with respect to our current product candidate and will face competition with respect to our future product candidates, from segments of the pharmaceutical, biotechnology and other related markets that pursue targeted therapies for patients with genetically defined cancers.

There are currently compounds approved and in development which target the EGFR pathway and against which we expect BDTX-1535 to compete, including:

There are currently compounds approved and in development which target the EGFR pathway and against which we expect silevertinib to compete, including:

• In patients with EGFR acquired resistance:

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patritumab deruxtecan, which is under development by Merck and Daiichi Sankyo Co.;

datopotamab deruxtecan, which is developed by AstraZeneca and Daiichi Sankyo Co.;

datopotamab deruxtecan, which is developed by AstraZeneca plc and Daiichi Sankyo Co.;

ivonescimab, which is under development by Summit Therapeutics and Akeso;

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• In patients with EGFR non-classical mutations:

afatinib, or Gilotrif, which is marketed by Boehringer Ingelheim and approved as first-line treatment of NSCLC patients with S768I, L861Q, and/or G719X mutations;

afatinib, or Gilotrif, which is marketed by Boehringer Ingelheim and approved as frontline treatment of NSCLC patients with S768I, L861Q, and/or G719X mutations;

osimertinib, or Tagrisso, which is marketed by AstraZeneca plc and is being prescribed off-label for NSCLC patients with exon 18 mutations;

Furmonertinb, which is under development by Arrivent Biopharma and Shanghai Allist Pharmaceuticals;

and ORIC-114, which is under development by ORIC Pharmaceuticals.

firmonertinib, which is under development by ArriVent Biopharma, Inc.

and Shanghai Allist Pharmaceuticals Co.

ORIC-114, which is under development by ORIC Pharmaceuticals, Inc.;

zipalertinib, which is under development by Taiho Oncology, Inc.

and Cullinan Therapeutics, Inc.;

and BH-30643, which is under development by BlossomHill Therapeutics, Inc.

• In patients with EGFR alterations present in GBM:

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We expect to continue to develop product candidates that can be produced cost-effectively at third-party contract manufacturing organizations (CMOs).

We generally expect to rely on one or more potential partners for the manufacture of companion diagnostics for our products, which are assays or tests to identify an appropriate patient population.

Commercialization

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Patent term may be inadequate to protect our competitive position on our products for an adequate amount of time.

As of February 28, 2025, we own ten U.S.

provisional patent applications, six U.S.

patent applications, and one issued U.S.

We also own seven Patent Cooperation Treaty (PCT) patent applications, 71 foreign patent applications, and two issued foreign patent.

or foreign patent issuing from these patent applications would be scheduled to expire in 2039 to 2045, excluding any additional term for patent term adjustment or patent term extension, and assuming that conversions are timely made based upon U.S.

As of March 11, 2026, we own four U.S.

provisional patent applications, eight U.S.

patent applications, and two issued U.S.

We also own nine Patent Cooperation Treaty (PCT) patent applications, 127 foreign patent applications, and 18 issued foreign patents.

or foreign patent issuing from these patent applications would be scheduled to expire from 2039 to 2046, excluding any additional term for patent term adjustment or patent term extension, and assuming that conversions are timely made based upon U.S.

provisional patent applications, that national phase entries are timely made based upon the pending PCT applications, and the payment of all applicable maintenance or annuity fees.

Below is an overview of patent applications covering BDTX-1535, BDTX-4933, BDTX-4876, and our MAP drug discovery engine.

As of February 28, 2025, we own seven U.S.

provisional patent applications, three U.S.

patent applications, five PCT patent applications, 45 foreign patent applications, and two issued foreign patents that cover our non-small cell lung cancer and glioblastoma program, including the composition of matter for BDTX-1535, metabolites of BDTX-1535, formulation of BDTX-1535, polymorphs of BDTX-1535, as well as methods of using and making BDTX-1535.

Below is an overview of patent applications covering silevertinib (formerly BDTX-1535), BDTX-4933, BDTX-4876, and our MAP drug discovery engine.

As of March 11, 2026, we own four U.S.

provisional patent applications, four U.S.

patent applications, one issued U.S.

patent, six PCT patent applications, 51 foreign patent applications, and 17 issued foreign patents that cover our non-small cell lung cancer and glioblastoma program, including the composition of matter for silevertinib, metabolites of silevertinib, formulation of silevertinib, polymorphs of silevertinib, as well as methods of using and making silevertinib.

or foreign patent issued from these pending applications would be scheduled to expire between 2040 and 2046, assuming that conversions are timely made based upon U.S.

provisional patent applications, and that national phase entries are timely made based upon the pending PCT applications, excluding any additional term for patent term adjustment or patent term extension.

As of February 28, 2025, we own six U.S.

provisional patent applications, one U.S.

patent application, one PCT patent application, and 16 foreign patent applications that cover our BRAF program, including the composition of matter for BDTX-4933, polymorphs of BDTX-4933, formulations of BDTX-4933, as well as methods of using and making BDTX-4933.

As of March 11, 2026, we own two U.S.

patent applications, two PCT patent applications, and 66 foreign patent applications that cover the BRAF program licensed to Servier pursuant to the license agreement entered in the first quarter of 2025, including the composition of matter for BDTX-4933, polymorphs of BDTX-4933, formulations of BDTX-4933, as well as methods of using and making BDTX-4933.

or foreign patent issued from these pending applications would be scheduled to expire between 2042 and 2045, assuming that conversions are timely made based upon U.S.

As of February 28, 2025, we own one U.S.

patent application, one PCT patent application, and ten foreign patent applications that cover our FGFR program, including the composition of matter for BDTX-4876, polymorphs of BDTX-4876, as well as methods of using and making BDTX-4876.

As of March 11, 2026, we own one U.S.

patent application, one PCT patent application, ten foreign patent applications, and one foreign patent that cover our FGFR program, including the composition of matter for BDTX-4876, polymorphs of BDTX-4876, as well as methods of using and making BDTX-4876.

or foreign patent issued from these pending applications would be scheduled to expire between 2042 and 2044, assuming that conversions are timely made based upon U.S.

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MAP drug discovery engine

As of February 28, 2025, we own one U.S.

As of March 11, 2026, we own one U.S.

patent application that covers our MAP drug discovery engine and the use thereof in developing and applying therapeutics.

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If we do not timely file non-provisional patent applications, we may lose our priority date with respect to our provisional patent applications and any patent protection on the inventions disclosed in our provisional patent applications.

While we intend to timely file non-provisional and national stage patent applications relating to our provisional and PCT patent applications, we cannot predict whether any of our future patent applications for BDTX-1535, BDTX-4933, BDTX-4876, or any of our other product candidates or technology will result in the issuance of patents that effectively protect BDTX-1535, BDTX-4933, BDTX-4876, or our other product candidates or technology.

If we do not successfully obtain patent protection, or, even if we do obtain patent protection, if the scope of the patent protection we or our potential licensors obtain with respect to BDTX-1535, BDTX-4933, BDTX-4876, or our other product candidates or technology is not sufficiently broad, we will be unable to prevent others from using our technology or from developing or commercializing technology and products similar or identical to ours or other competing products and technologies.

While we intend to timely file non-provisional and national stage patent applications relating to our provisional and PCT patent applications, we cannot predict whether any of our future patent applications for silevertinib, BDTX-4933, BDTX-4876, or any of our other product candidates or technology will result in the issuance of patents that effectively protect silevertinib, BDTX-4933, BDTX-4876, or our other product candidates or technology.

If we do not successfully obtain patent protection, or, even if we do obtain patent protection, if the scope of the patent protection we or our potential licensors obtain with respect to silevertinib, BDTX-4933, BDTX-4876, or our other product candidates or technology is not sufficiently broad, we will be unable to prevent others from using our technology or from developing or commercializing technology and products similar or identical to ours or other competing products and technologies.

For more information regarding the risks related to our intellectual property, please see “Risk Factors—Risks Related to our Intellectual Property.”

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Orphan designation does not convey any advantage in or shorten the duration of the regulatory review and approval process, though companies developing orphan products are eligible for certain incentives, including tax credits for qualified clinical testing and waiver of application fees.

If a product that has orphan designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to a seven-year period of marketing exclusivity during which the FDA may not approve any other applications to market the same therapeutic agent for the same indication, except in limited circumstances, such as a subsequent product’s showing of clinical superiority over the product with orphan exclusivity or where the original applicant cannot produce sufficient quantities of product.

If a product that has orphan designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to a seven-year period of marketing exclusivity during which the FDA may not approve any other applications to market the same therapeutic agent for the same approved use or indication, except in limited circumstances, such as a subsequent product’s showing of clinical superiority over the product with orphan exclusivity or where the original applicant cannot produce sufficient quantities of product.

Competitors, however, may receive approval of different therapeutic agents for the indication for which the orphan product has exclusivity or obtain approval for the same therapeutic agent for a different indication than that for which the orphan product has exclusivity.

Orphan product exclusivity could block the approval of one of our products for seven years if a competitor obtains approval for the same therapeutic agent for the same indication before we do, unless we are able to demonstrate that our product is clinically superior.

Orphan product exclusivity could block the approval of one of our products for seven years if a competitor obtains approval for the same therapeutic agent for the same approved use or indication before we do, unless we are able to demonstrate that our product is clinically superior.

If an orphan designated product receives marketing approval for an indication broader than what is designated, it may not be entitled to orphan exclusivity.

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If a PRV is received, it may be sold or transferred an unlimited number of times.

The FDA’s rare pediatric disease priority voucher program began to sunset on December 20, 2024, on failure to pass a continuing resolution package that included its reauthorization.

Under the amended statutory sunset provisions, after December 20, 2024, the FDA may award a priority review voucher for an approved rare pediatric disease product application only if the sponsor has rare pediatric disease designation for the drug and if that designation was granted by December 20, 2024.

After September 30, 2026, the FDA may not award any rare pediatric disease priority review vouchers.

Congress may vote to reauthorize this program, but its future remains unknown at this time.

Under current law, after September 30, 2029, the FDA may not award any rare pediatric disease priority vouchers, although the FDA’s authority to do so could be extended by Congress in the future.

Expedited development and review programs for drugs

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or mandated modification of promotional materials and labeling and issuance of corrective information.

Regulation of companion diagnostics

We believe that the success of certain of our product candidates may depend, in part, on the development and commercialization of a companion diagnostic.

Companion diagnostics identify patients who are most likely to benefit from a particular therapeutic product;

identify patients likely to be at increased risk for serious side effects as a result of treatment with a particular therapeutic product;

or monitor response to treatment with a particular therapeutic product for the purpose of adjusting treatment to achieve improved safety or effectiveness.

Companion diagnostics are regulated as medical devices by the FDA.

In the United States, the FD&C Act and its implementing regulations, and other federal and state statutes and regulations govern, among other things, medical device design and development, preclinical and clinical testing, premarket clearance or approval, registration and listing, manufacturing, labeling, storage, advertising and promotion, sales and distribution, export and import, and post-market surveillance.

Unless an exemption or FDA exercise of enforcement discretion applies, diagnostic tests generally require marketing clearance or approval from the FDA prior to commercialization.

The primary types of FDA marketing authorization applicable to a medical device are clearance of a premarket notification, or 510(k), submission, approval of a premarket approval, or PMA, application, or grant of a de novo request for classification.

To obtain 510(k) clearance for a medical device, or for certain modifications to devices that have received 510(k) clearance, a manufacturer must submit a premarket notification demonstrating that the proposed device is substantially equivalent to a previously cleared 510(k) device or to a preamendment device that was in commercial distribution before May 28, 1976, or a predicate device, for which the FDA has not yet called for the submission of a PMA.

In making a determination that the device is substantially equivalent to a predicate device, the FDA compares the proposed device to the predicate device or predicate devices and assesses whether the subject device is comparable to the predicate device or predicate devices with respect to intended use, technology, design and other features which could affect safety and effectiveness.

If the FDA determines that the subject device is substantially equivalent to the predicate device or predicate devices, the subject device may be cleared for marketing.

The 510(k) premarket notification pathway generally takes from three to twelve months from the date the application is completed, but can take significantly longer.

PMA applications must be supported by valid scientific evidence, which typically requires extensive data, including technical, preclinical, clinical and manufacturing data, to demonstrate to the FDA’s satisfaction the safety and effectiveness of the device.

For diagnostic tests, a PMA application typically includes data regarding analytical and clinical validation studies.

As part of its review of the PMA, the FDA will conduct a pre-approval inspection of the manufacturing facility or facilities to ensure compliance with the Quality System Regulation, or QSR, which requires manufacturers to follow design, testing, control, documentation and other quality assurance procedures.

The FDA’s review of an initial PMA application is required by statute to take between six to ten months, although the process typically takes longer, and may require several years to complete.

If the FDA evaluations of both the PMA application and the manufacturing facilities are favorable, the FDA will either issue an approval letter or an approvable letter, which usually contains a number of conditions that must be met in order to secure the final approval of the PMA.

If the FDA’s evaluation of the PMA or manufacturing facilities is not favorable, the FDA will deny the approval of the PMA or issue a not approvable letter.

A not approvable letter will outline the deficiencies in the application and, where practical, will identify what is necessary to make the PMA approvable.

Once granted, PMA approval may be withdrawn by the FDA if compliance with post-approval requirements, conditions of approval or other regulatory standards is not maintained or problems are identified following initial marketing.

On July 31, 2014, the FDA issued a final guidance document addressing the development and approval process for “In Vitro Companion Diagnostic Devices.” According to the guidance document, for novel therapeutic products that depend on the use of a diagnostic test and where the diagnostic device could be essential for the safe and effective use of the corresponding therapeutic product, the premarket application for the companion diagnostic device should be developed and approved or cleared contemporaneously with the therapeutic, although the FDA recognizes that there may be cases when contemporaneous development may not be possible.

However, in cases where a drug cannot be used safely or effectively without the companion diagnostic, the FDA’s guidance indicates it will generally not approve the drug without the approval or clearance of the diagnostic device.

The FDA also issued a draft guidance in July 2016 setting forth the principles for co-development of an in vitro companion diagnostic device with a therapeutic product.

The draft guidance describes principles to guide the development and contemporaneous marketing authorization for the therapeutic product and its corresponding in vitro companion diagnostic.

To date, the FDA has required premarket approval for nearly all companion diagnostics for cancer therapies.

In January 2024, FDA announced its intention to initiate the reclassification process for most in vitro diagnostics, including companion diagnostics.

Further, FDA indicated that in addition to the reclassification process, FDA will continue taking a risk-based approach in the initial classification of individual in vitro diagnostics to determine whether a new test may be classified into class II through the de novo classification process.

In so doing, FDA indicated that it may regulate most future companion diagnostics as class II devices.

Once cleared or approved, the companion diagnostic device must adhere to post-marketing requirements including the requirements of the FDA’s quality system regulation, adverse event reporting, recalls and corrections along with product marketing requirements and limitations.

Like drug makers, companion diagnostic makers are subject to unannounced FDA inspections at any time during which the FDA will conduct an audit of the product(s) and the company’s facilities for compliance with its authorities.

Other regulatory matters

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In the United States, these laws include, without limitation, state and federal anti-kickback, false claims, physician transparency, and patient data privacy and security laws and regulations, including but not limited to those described below.

• The federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration (including any kickback, bribe, or rebate), directly or indirectly in cash or in kind, to induce or reward either the referral of an individual for, or the purchase, lease, order, arrangement or recommendation of, any good, facility, item or service, for which payment may be made under federal and state healthcare programs such as Medicare and Medicaid.

• The federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving, paying or providing remuneration (including any kickback, bribe, or rebate), directly or indirectly in cash or in kind, to induce or reward either the referral of an individual for, or the purchase, lease, order, arrangement or recommendation of, any good, facility, item or service, for which payment may be made, in whole or in part, under federal and state healthcare programs such as Medicare and Medicaid.

A person or entity does not need to have actual knowledge of the federal Anti-Kickback Statute (AKS) or specific intent to violate it to have committed a violation.

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In addition, the government may assert that a claim including items or services resulting from a violation of the AKS constitutes a false or fraudulent claim for purposes of the federal False Claims Act (FCA) or federal civil money penalties.

There are a number of statutory exceptions and regulatory safe harbors protecting some common activities from prosecution.

• The federal civil and criminal false claims laws, including the FCA, and civil monetary penalty laws which can be enforced through civil whistleblower or qui tam actions, which imposes civil and criminal penalties against individuals or entities for knowingly presenting or causing to be presented, to the federal government, claims for payment or approval from Medicare, Medicaid or other government payors that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay to the federal government, with potential liability including mandatory treble damages and significant per-claim penalties.

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The FCA also permits a private individual acting as a “whistleblower” to bring actions on behalf of the federal government alleging violations of the FCA and to share in any monetary recovery.

When an entity is determined to have violated the federal civil False Claims Act, the government may impose civil fines and penalties for each false claim, plus treble damages, and exclude the entity from participation in Medicare, Medicaid and other federal healthcare programs.

• The federal Health Insurance Portability and Accountability Act of 1996 (HIPAA), which prohibits, among other things, knowingly and willfully executing or attempting to execute, a scheme or artifice to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations, or promises, any of the money or property owned by, or under the custody or control of, any healthcare benefit program, regardless of the payor (e.g., public or private), and knowingly and willfully falsifying, concealing or covering up by any trick or device a material fact or making any materially false, fictitious or fraudulent statements in connection with the delivery of or payment for, healthcare benefits, items or services relating to healthcare benefits, items or services relating to healthcare matters.

Similar to the AKS, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation.

• HIPAA, as further amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (HITECH) and their respective implementing regulations, including the Final Omnibus Rule published in January 2013, which impose certain requirements, including mandatory contractual terms, on covered entities subject to the rule, such as health plans, healthcare clearinghouses and certain healthcare providers, as well as their respective business associates and their subcontractors that perform services for them that involve the creation, maintenance, receipt, use, or disclosure of, individually identifiable health information, relating to the privacy, security, and transmission of such individually identifiable health information relating to the privacy, security and transmission of individually identifiable health information.

HITECH also created new tiers of civil monetary penalties, amended HIPAA to make civil and criminal penalties directly applicable to business associates, and gave state attorneys general new authority to file civil actions for damages or injunctions in federal courts to enforce the federal HIPAA laws and seek attorneys’ fees and costs associated with pursuing federal civil actions.

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Several states also impose other marketing restrictions or require pharmaceutical companies to make marketing or price disclosures to the state and require the registration of pharmaceutical sales representatives.

State and foreign laws, including for example the EU General Data Protection Regulation (GDPR), which became effective May 2018 also govern the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.

There are ambiguities as to what is required to comply with these state requirements and if we fail to comply with an applicable state law requirement we could be subject to penalties.

Finally, there are state and foreign laws governing the privacy and security of health information, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.

State and foreign laws, including the EU’s General Data Protection Regulation (GDPR), and dozens of US state and federal regulations also govern the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.

There are ambiguities as to what is required to comply with these myriad requirements and if we fail to comply with an applicable state law requirement, federal regulation, or foreign privacy regime we could be subject to penalties.

We have adopted a code of business conduct and ethics, but it is not always possible to identify and deter employee misconduct, and the precautions we take to detect and prevent inappropriate conduct may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to be in compliance with such laws or regulations.

Payments made to physicians in certain EU Member States must be publicly disclosed.

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and require companies to pay rebates to Medicare for certain drug prices that increase faster than inflation, also including small molecule drugs.

Further, under the IRA, orphan drugs were previously exempted from the Medicare drug price negotiation program;

however, this exemption was restricted to drugs with only one orphan designation and for which the only approved indication is for that disease or condition.

If a product received multiple orphan designations or had multiple approved indications, it would not qualify for the orphan drug exemption.

Under the One Big Beautiful Bill Act of 2025, or the OBBB Act, this restriction was eliminated;

and effective for the 2028 initial price applicability year, all orphan drugs, regardless of the number of orphan designations or indications, are exempt from the Medicare drug price negotiation program.

The effects of the IRA on our business and the healthcare industry in general is not yet known.

Additionally, there has been increasing legislative and enforcement interest in the United States with respect to drug pricing practices.

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A number of these and other proposed measures may require authorization through additional legislation to become effective.

The Trump administration also previously released a “Blueprint” to lower drug prices and reduce out of pocket costs of drugs that contains additional proposals to increase manufacturer competition, increase the negotiating power of certain federal healthcare programs, incentivize manufacturers to lower the list price of their products and reduce the out of pocket costs of drug products paid by consumers.

HHS solicited feedback on some of these measures and has in the past implemented others under its existing authority.

For example, in May 2019, CMS issued a final rule to allow Medicare Advantage Plans the option of using step therapy, a type of prior authorization, for Part B drugs effective January 1, 2020.

Congress and the Trump administration have indicated that they will continue to seek new legislative measures to control drug costs.

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The IRA further extended the delay in implementing this rule under 2032.

Additionally, the FDA published a final rule, effective November 30, 2020, that allows for the importation of certain prescription drugs from Canada.

Under the final rule, states and Indian Tribes, and in certain future circumstances pharmacists and wholesalers, may submit importation program proposals to the FDA for review and authorization.

On September 25, 2020, CMS stated drugs imported by States under this rule will not be eligible for federal rebates under Section 1927 of the Social Security Act and manufacturers would not report these drugs for “best price” or Average Manufacturer Price purposes.

Since these drugs are not considered covered outpatient drugs, CMS further stated it will not publish a National Average Drug Acquisition Cost for these drugs.

Separately, the FDA also issued a final guidance document outlining a potential pathway for manufacturers to obtain an additional National Drug Code, or NDC, for an FDA-approved drug that was originally intended to be marketed in a foreign country and that was authorized for sale in that foreign country.

On April 15, 2025, the Trump Administration published Executive Order 14273, “Lowering Drug Prices by Once Again Putting Americans First,” which generally directs the federal government to take measures to reduce drug prices, including eliminating the so-called “pill penalty” under the Inflation Reduction Act that creates a distinction between small molecule and large molecule products for purposes of determining when a drug may be eligible for drug price negotiation.

On May 12, 2025, the Trump Administration published Executive Order 14297, “Delivering Most-Favored-Nation Prescription Drug Pricing to American Patients” which generally, among other things, directs the federal government to establish and communicate most-favored-nation (MFN) price targets to pharmaceutical manufacturers to bring prices for American patients in line with comparably developed nations.

Further, the Executive Order directs the federal government to support regulatory paths to allow direct-to-patient sales for companies that meet these targets.

It also states that the Administration will take additional aggressive action (for example, examining whether marketing approvals should be modified or rescinded or opening the door for individual drug importation waivers) should manufacturers fail to offer American consumers the MFN lowest price.

It also directs the Secretary of Commerce and the U.S.

Trade Representative to “take all necessary and appropriate action to ensure foreign countries are not engaged in any act, policy, or practice that may be unreasonable or discriminatory or that may impair United States national security .

including by suppressing the price of pharmaceutical products below fair market value in foreign countries.” Notably, a similar “Most Favored Nation” pricing rule enacted under the first Trump Administration was subject to an injunction resulting from judicial challenges to the rule, which was formally rescinded by the former Biden Administration in August 2021.

On December 19, 2025, CMS released two proposed rules that would incorporate MFN pricing principles into federal reimbursement for prescription drugs.

The first proposal, the Global Benchmark for Efficient Drug Pricing Model (GLOBE) for Medicare Part B, would require manufacturers of specified single source drugs and sole source biologics to pay incremental rebates based on international benchmark prices, with participation triggered for products meeting CMS’s spending and eligibility criteria.

The second proposal, the Guarding U.S.

Medicare Against Rising Drug Costs (GUARD) model for Medicare Part D, would similarly mandate manufacturer rebates for qualifying sole source drugs where the Medicare net price exceeds an MFN benchmark derived from international reference pricing methodologies.

As proposed, GLOBE would begin a five year performance period on October 1, 2026 and GUARD would begin its performance period in 2027.

These proposals will likely be subject to legal challenges that could delay their implementation or modify their impact on manufacturer pricing and revenue.

Additionally, in November 2025, CMS introduced the GENErating cost Reductions fOr U.S.

Medicaid (GENEROUS) Model, a voluntary MFN framework for manufacturers participating in the Medicaid Drug Rebate Program.

Although it is voluntary, the GENEROUS Model could also impact the drug pricing landscape for manufacturers.

At the state level, individual states are increasingly aggressive in passing legislation and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.

Certain states are also pursuing cost containment efforts through Prescription Drug Affordability Boards (PDABs) and similar entities.

While many PDABs have been granted authority to promote drug price transparency and reporting, some states have granted PDABs more expansive authority, including to set Upper Payment Limits (UPLs) on select, high price drugs.

The adoption and implementation of UPLs may put downward pressure on drug prices and impact our company’s future revenues.

In addition, regional health care authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug and other health care programs.

Some of these proposed measures, including drug importation and pharmacy benefit manager rebate rule changes, face legal challenges from industry groups and participants.

These measures could reduce the ultimate demand for our products, once approved, or put pressure on our product pricing.

Outside the United States, ensuring coverage and adequate payment for a product also involves challenges.

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Some countries provide that products may be marketed only after a reimbursement price has been agreed.

Some countries may require the completion of additional studies that compare the cost-effectiveness of a particular product candidate to currently available therapies or so-called health technology assessments, in order to obtain reimbursement or pricing approval.

For example, the EU provides options for its Member States to restrict the range of products for which their national health insurance systems provide reimbursement and to control the prices of medicinal products for human use.

EU Member States may approve a specific price for a product or it may instead adopt a system of direct or indirect controls on the profitability of the company placing the product on the market.

Some countries may require the completion of additional studies or health technology assessments that compare the cost-effectiveness of a particular product candidate to currently available therapies in order to obtain reimbursement or pricing approval.

For example, in the EU Member States may restrict the range of products for which their national health insurance systems provide reimbursement or impose price controls on medicines.

EU Member States may approve a specific price for a product or may instead adopt a system of direct or indirect controls on the profitability of the company placing the product on the market.

Other EU Member States allow companies to fix their own prices for products, but monitor and control prescription volumes and issue guidance to physicians to limit prescriptions.

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Political, economic and regulatory developments may further complicate pricing negotiations, and pricing negotiations may continue after reimbursement has been obtained.

Reference pricing used by various EU Member States, and parallel trade, i.e., arbitrage between low-priced and high-priced member states, can further reduce prices.

There can be no assurance that any country that has price controls or reimbursement limitations for pharmaceutical products will allow favorable reimbursement and pricing arrangements for any products, if approved in those countries.

Reference pricing used by various EU Member States, and parallel trade, i.e., arbitrage between low-priced and high-priced EU Member States, can further reduce prices.

There can be no assurance that any country that has price controls or reimbursement limitations for pharmaceutical products will grant favorable reimbursement and pricing arrangements for any products, if approved in those countries.

Compliance with other federal and state laws or requirements;

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• Centralized procedure —If pursuing marketing authorization of a product candidate for a therapeutic indication under the centralized procedure, following the opinion of the European Medicines Agency’s, (EMA), Committee for Medicinal Products for Human Use (CHMP), the European Commission issues a single marketing authorization valid across the EU, and in the additional countries of the European Economic Area (Iceland, Liechtenstein and Norway).

The centralized procedure is compulsory for human medicines derived from biotechnology processes or advanced therapy medicinal products (such as gene therapy, somatic cell therapy and tissue engineered products), products that contain a new active substance indicated for the treatment of certain diseases, such as HIV, AIDS, cancer, neurodegenerative disorders, diabetes, autoimmune diseases and other immune dysfunctions, viral diseases, and designated orphan medicines.

The centralized procedure is compulsory for human medicines derived from biotechnology processes or advanced therapy medicinal products (i.e.

gene therapy, somatic cell therapy and tissue engineered products), products that contain a new active substance indicated for the treatment of certain diseases, such as HIV, AIDS, cancer, neurodegenerative disorders, diabetes, autoimmune diseases and other immune dysfunctions, viral diseases, and designated orphan medicines.

For medicines that do not fall within these categories, an applicant has the option of submitting an application for a centralized marketing authorization to the EMA, as long as the medicine concerned contains a new active substance not yet authorized in the EU, is a significant therapeutic, scientific or technical innovation, or if its authorization would be in the interest of public health in the EU.

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Clock stops may extend the timeframe of evaluation of an MAA considerably beyond 210 days.

Where the CHMP gives a positive opinion, it provides the opinion together with supporting documentation to the European Commission, who makes the final decision to grant a marketing authorization, which is issued within 67 days of receipt of the EMA’s recommendation.

Accelerated assessment might be granted by the CHMP in exceptional cases, when a medicinal product is expected to be of a major public health interest, particularly from the point of view of therapeutic innovation.

Where the CHMP gives a positive opinion, it provides the opinion together with supporting documentation to the European Commission, which makes the final decision to grant the marketing authorization, which is issued within 67 days of receipt of the EMA’s recommendation.

Accelerated assessment may be granted by the CHMP in exceptional cases, for medicinal products of major public health interest, particularly from the perspective of therapeutic innovation.

If the CHMP accepts such request, the time limit of 210 days will be reduced to 150 days, excluding clock stops, but it is possible that the CHMP may revert to the standard time limit for the centralized procedure if it determines that the application is no longer appropriate to conduct an accelerated assessment.

• National authorization procedures —There are also two other possible routes to authorize products for therapeutic indications in several countries, which are available for products that fall outside the scope of the centralized procedure:

• National authorization procedures —There are also two other possible routes to authorize products for therapeutic indications in several countries in the EU, which are available for products that fall outside the scope of the centralized procedure:

◦ Decentralized procedure —Using the decentralized procedure, an applicant may apply for simultaneous authorization in more than one EU country of medicinal products that have not yet been authorized in any EU country and that do not fall within the mandatory scope of the centralized procedure.

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Following this, additional marketing authorizations can be sought from other EU countries in a procedure whereby the countries concerned recognize the validity of the original, national marketing authorization.

Under the above described procedures, before granting the marketing authorization, the EMA or the competent authorities of the Member States of the EU make an assessment of the risk-benefit balance of the product on the basis of scientific criteria concerning its quality, safety and efficacy.

Under the above described procedures, before granting a marketing authorization, the EMA (for centralized procedures) or the competent authorities of the Member States (for national, decentralized or mutual recognition procedures) assess the risk-benefit balance of the product on the basis of scientific criteria concerning its quality, safety and efficacy.

In the EU, new products for therapeutic indications that are authorized for marketing (i.e., reference products) qualify for eight years of data exclusivity and an additional two years of market exclusivity upon marketing authorization.

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An orphan product can also obtain an additional two years of market exclusivity in the EU for pediatric studies.

The ten-year market exclusivity may be reduced to six years if, at the end of the fifth year, it is established that the product no longer meets the criteria for orphan designation, for example, if the product is sufficiently profitable not to justify maintenance of market exclusivity.

The ten-year market exclusivity may be reduced to six years if, at the end of the fifth year, it is established that the product no longer meets the criteria for orphan designation, including if the product is sufficiently profitable not to justify maintenance of market exclusivity.

Otherwise, orphan medicine marketing exclusivity may be revoked only in very select cases, such as if (i) it is established that a similar medicinal product is safer, more effective or otherwise clinically superior to the authorized product;

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or (iii) the marketing authorization holder of the authorized orphan product cannot supply enough orphan medicinal product.

Orphan designation must be requested before submitting an application for marketing approval.

Orphan designation must be requested before submission of the for marketing authorization application.

Orphan designation does not convey any advantage in, or shorten the duration of, the regulatory review and approval process.

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Devices that comply with the requirements of the IVDR will be entitled to bear the CE conformity marking, indicating that the device conforms to the general safety and performance requirements of the IVDR, and, accordingly, can be commercially distributed throughout the EU (in-vitro diagnostic medical devices cannot be marketed in the EU without a CE mark).

The method of assessing conformity varies depending on the class of the product, but normally involves a third-party assessment by a “Notified Body”.

The method of assessing conformity varies depending on the class of the product, but normally involves a third-party assessment by an independent “Notified Body”.

This third-party assessment may consist of an audit of the manufacturer’s quality system and specific testing of the manufacturer’s product.

Similar to the United States, the various phases of non-clinical and clinical research in the EU are subject to significant regulatory controls.

In April 2014, new Clinical Trials Regulation, (EU) No 536/2014 (Clinical Trials Regulation) was adopted, which replaced the Clinical Trials Directive 2001/20/EC on January 31, 2022.

The Clinical Trials Regulation is directly applicable in all the EU Member States, meaning no national implementing legislation is required.

In April 2014, Regulation (EU) No 536/2014 on clinical trials (Clinical Trials Regulation) was adopted, which replaced the Clinical Trials Directive 2001/20/EC on January 31, 2022.

The Clinical Trials Regulation is directly applicable in all EU Member States, meaning no national implementing legislation is required.

The Clinical Trials Regulation aims to simplify and streamline the approval of clinical trials in the EU.

The main characteristics of the regulation include:

a streamlined application procedure via a single-entry point, the Clinical Trials Information Systems, or “CTIS”;

a streamlined application procedure via a single-entry point, the Clinical Trials Information System (CTIS);

a single set of documents to be prepared and submitted for the application as well as simplified reporting procedures for clinical trial sponsors;

and a harmonized procedure for the assessment of applications for clinical trials, which is divided in two parts.

Part I is assessed by coordinated assessment of the competent authorities of all EU Member States in which an application for authorization of a clinical trial has been submitted (Member States concerned), of the review of a Reference Member State.

Part I is assessed in a coordinated procedure led by a Reporting Member State, with the involvement of the Member States concerned.

Part II is assessed separately by each Member State concerned.

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All of the aforementioned EU rules are generally applicable in the European Economic Area (EEA), which consists of the EU Member States, plus Norway, Liechtenstein and Iceland.

The collection and use of personal data in the EEA is governed by the General Data Protection Regulation (GDPR), which became effective on May 25, 2018.

The European Commission introduced legislative proposals in April 2023 that, if implemented, will replace the current regulatory framework in the EU for all medicines (including those for rare diseases and for children).

In April 2024, the European Parliament adopted its position on the legislative proposals and, in June 2025, the Council of the European Union adopted its position.

A common position on the text has been agreed upon on December 11, 2025, in the context of subsequent inter-institutional trilogue negotiations.

The proposed revisions remain to be adopted, and are not expected to become applicable before 2028.

The collection and use of personal data in the EEA is governed by the General Data Protection Regulation (GDPR).

The GDPR has extraterritorial application and applies to organizations based outside the EEA that provide goods or services to residents in the EU.

This expansion would incorporate any clinical trial activities in EU Members States.

The GDPR imposes strict requirements on controllers and processors of personal data, including special protections for “sensitive information” which includes health and genetic information of data subjects residing in the EEA.

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Further, the GDPR enables individuals to claim damages for violations and introduces the right for non-profit organizations to bring claims on behalf of data subjects.

Further, the GDPR imposes strict rules on the transfer of personal data out of the EEA to the United States or other regions that have not been deemed to offer “adequate” privacy protections.

The GDPR imposes strict rules on the transfer of personal data out of the EEA to the United States or other regions that have not been deemed to offer “adequate” privacy protections.

Failure to comply with the requirements of the GDPR and the related national data protection laws of the EEA Member States, which may deviate slightly from the GDPR, may result in fines of up to 4% of global revenues, or €20 million, whichever is greater.

As a result of the implementation of the GDPR, we may be required to put in place additional mechanisms ensuring compliance with the new data protection rules.

In addition, further to the United Kingdom (UK)’s exit from the EU on January 31, 2020, the GDPR ceased to apply in the UK at the end of the transition period on December 31, 2020.

However, as of January 1, 2021, the UK’s EU (Withdrawal) Act 2018 incorporated the GDPR (as it existed on December 31, 2020 but subject to certain UK specific amendments) into UK law, referred to as the UK GDPR.

The GDPR introduced additional complexity and requirements for additional mechanisms ensuring compliance with the new data protection rules.

In addition, further to the United Kingdom (UK)’s exit from the EU, the UK’s EU (Withdrawal) Act 2018 incorporated the GDPR (as it existed on December 31, 2020 but subject to certain UK specific amendments) into UK law, referred to as the UK GDPR.

The UK GDPR and the UK Data Protection Act 2018 set out the UK’s data protection regime, which is independent from but aligned to the EU’s data protection regime.

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The UK government has confirmed that personal data transfers from the UK to the EEA remain free flowing.

There is uncertainty related to the manner in which data protection authorities will seek to enforce compliance with GDPR.

For example, it is not clear if the authorities will conduct random audits of companies doing business in the EEA, or if the authorities will wait for complaints to be filed by individuals who claim their rights have been violated.

In 2025, the Data (Use and Access) Bill (DUA Act) passed both Houses of UK Parliament and received Royal Assent.

The DUA Act alters requirements for international data transfers and marks a shift in the UK’s approach to data protection.

Enforcement uncertainty and the costs associated with ensuring GDPR compliance are onerous and may adversely affect our business, financial condition, results of operations and prospects.

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Brexit and the Regulatory Framework in the United Kingdom

The UK formally left the EU on January 31, 2020.

As a result of the Northern Ireland protocol, following Brexit, the EMA remained responsible for approving novel medicines for supply in Northern Ireland under the EU centralized procedure, and a separate authorization was required to supply the same medicine in Great Britain (England, Wales and Scotland).

On February 27, 2023, the UK government and the EC announced a political agreement in principle to replace the Northern Ireland Protocol with a new set of arrangements, known as the “Windsor Framework”.

The Windsor Framework was approved by the EU-UK Joint Committee on March 24, 2023, and the medicines aspects of the Windsor Framework have applied since January 1, 2025.

This new framework fundamentally changes the previous system under the Northern Ireland Protocol, including with respect to the regulation of medicinal products in the UK.

In particular, the MHRA is now responsible for approving all medicinal products destined for the UK market (i.e., Great Britain and Northern Ireland), and the EMA no longer has any role in approving medicinal products destined for Northern Ireland under the EU centralized procedure.

A single UK-wide MA will be granted by the MHRA for all novel medicinal products to be sold in the UK, enabling products to be sold in a single pack and under a single authorization throughout the UK.

In addition, the new arrangements require all medicines placed on the UK market to be labelled “UK only”, indicating they are not for sale in the EU.

On January 1, 2024, the MHRA put in place a new international recognition framework which means that the MHRA may have regard to decisions on the approval of MA’s made by the EMA and certain other regulators when determining an application for a new UK MA.

There is now no pre-MA orphan designation in the UK.

Instead, the MHRA reviews applications for orphan designation in parallel to the corresponding MAA.

The criteria are essentially the same, but have been tailored for the UK market, i.e., the prevalence of the condition in the UK (rather than the EU) must not be more than five in 10,000.

Should an orphan designation be granted, the period of market exclusivity will be set from the date of first approval of the product in the UK.

Following the end of the Brexit transition period on January 1, 2021 and the implementation of the Windsor Framework on January 1, 2025, the United Kingdom (UK) is not generally subject to EU laws in respect of medicines.

The EU laws that have been transposed into UK law through secondary legislation remain applicable in the UK however, new legislation such as the EU Clinical Trials Regulation, is not applicable in the UK.

As of January 1, 2021, the Medicines and Healthcare products Regulatory Agency (MHRA) is the UK's standalone medicines and medical devices regulator.

As a result of the Northern Ireland Protocol, different rules applied in Northern Ireland than in England, Wales, and Scotland (together, "Great Britain", or GB), with Northern Ireland continuing to follow the EU regulatory regime for a period after Brexit.

However, on January 1, 2025 a new arrangement called the "Windsor Framework" came into effect and reintegrated Northern Ireland under the regulatory authority of the MHRA with respect to medicinal products.

The Windsor Framework removes EU licensing processes and EU labeling and serialization requirements in relation to Northern Ireland and introduces a UK-wide licensing process for medicines.

In particular, the MHRA is now responsible for approving medicinal products placed on the UK market (i.e., Great Britain and Northern Ireland), and the EMA no longer has a role in UK marketing authorizations.

A single UK-wide marketing authorizations will be granted by the MHRA for medicinal products to be sold in the UK, enabling products to be sold in a single pack and under a single authorization throughout the UK.

In addition, the new arrangements require, for packs placed on the UK market on or after January 1, 2025, a "UK Only" label is required, indicating they are not for sale in the EU.

However, although separate authorization is now required to market medicinal products in the UK, since January 1, 2024, the MHRA may rely on the International Recognition Procedure, or IRP, when reviewing certain types of MAAs.

Pursuant to the IRP, the MHRA will take into account the expertise and decision-making of trusted regulatory partners (e.g.

the medicines regulatory authorities in Australia, Canada, Switzerland, Singapore, Japan, the U.S.A.

and the EMA in the EU).

There is no pre-marketing authorization orphan designation in the UK.

The MHRA reviews applications for orphan designation with the corresponding MAA.

The criteria for orphan designation in the UK are similar to those in the EU, but are applied to the UK population not more than five in 10,000 in the UK).

If granted, orphan market exclusivity is set from the date of first approval in the UK.

Human Capital Resources

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To facilitate talent attraction and retention, we strive to make Black Diamond a safe and rewarding workplace, with opportunities for our employees to grow and develop in their careers, supported by strong compensation, benefits and health and wellness programs, and by programs that build connections between our employees.

As of February 28, 2025, we had 24 full-time employees.

As of March 11, 2026, we had 21 full-time employees.

Three of our employees have Ph.D.

degrees, three have Pharm.D.

degrees, two have Pharm.D.

degrees, and three have M.D.

The following table shows the number of full-time employees as of February 28, 2025 engaged in either research and development or administrative functions.

The following table shows the number of full-time employees as of March 11, 2026 engaged in either research and development or administrative functions.

All of our employees are located in the United States.

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We consider our relationship with our employees to be good.

As of February 28, 2025, approximately 58% of the Company's workforce, and 57% of our employees in managerial roles, identified as female.

As of February 28, 2025, approximately 13% of our workforce, and 13% of our employees in managerial roles, identified as racially or ethnically diverse.

The success of our business is fundamentally connected to the well-being of our employees.

A highly skilled and engaged workforce is critical to achieving our business objectives and bringing value to patients, shareholders, and all stakeholders.

We foster an inclusive culture that attracts, retains, engages, and develops our people.

Additionally, the success of our business is fundamentally connected to the well-being of our employees.

Accordingly, we are committed to their health, safety and wellness.

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We provide robust compensation and benefits programs to help meet the needs of our employees.

In addition to salaries, these programs include potential annual discretionary bonuses, stock awards, a 401(k) Plan, healthcare and insurance benefits, health savings and flexible spending accounts, paid time off, family leave and flexible work schedules, among others.

In addition to salaries, these programs include potential annual discretionary bonuses, stock awards, a 401(k) Plan, healthcare and insurance benefits, flexible spending accounts, paid time off, family leave and flexible work schedules, among others.

In addition to our broad-based equity award programs, we have used targeted equity-based grants with vesting conditions to facilitate retention of personnel.

We lease a facility containing approximately 25,578 square feet of office space for our principal office, which is located at One Main Street, Cambridge, MA 02142.

We lease office space for our principal office on a month-to-month basis, which is located at 245 First Street, 18th Floor, Cambridge, MA 02142.

We lease a facility containing approximately 25,578 square feet of office space, which is located at One Main Street, Cambridge, MA 02142.

The lease expires on August 31, 2028, subject to an option to extend the lease for five additional years.

In December 2022, we entered into a sublease for one floor, approximately 14,439 square feet, of our Cambridge, MA office space, which also terminates on August 31, 2028.

In December 2025, we entered into a sublease for the other floor, approximately 11,139 square feet, of our Cambridge, MA office space, which also terminates on August 31, 2028.

We also lease approximately 18,120 square feet of office and laboratory space at 430 East 29th Street, New York, New York 10016.

The lease expires on June 30, 2032, subject to an option to extend the lease for five additional years.

The lease expires on August 30, 2032, subject to an option to extend the lease for five additional years.

In June 2024, we entered into a sublease for our office and laboratory space in New York, NY.

The sublease terminates on June 30, 2026, with the option to extend to June 30, 2027.

The sublease terminates on June 30, 2026.

We believe that our current facilities are adequate for our current needs and that suitable additional or substitute space at commercially reasonable terms will be available as needed to accommodate any future expansion of our operations.

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On January 2, 2018, we changed our name to Black Diamond Therapeutics, Inc.

Our principal executive offices are located at One Main Street, Cambridge, MA 02142, and our telephone number is 617-252-0848.

Our principal executive offices are located at 245 First Street, 18th Floor, Cambridge, MA 02142, and our telephone number is 617-252-0848.

As of December 31, 2025, we have one subsidiary, Black Diamond Therapeutics Security Corporation, which was incorporated in 2019.

Our second subsidiary, Black Diamond Therapeutics (Canada) Inc., which was incorporated in 2018, was dissolved in October 2023.

We are an “emerging growth company” as defined in the Jumpstart Our Business Startups Act of 2012.

We will remain an emerging growth company until the earlier of:

(i) the last day of the fiscal year (a) following the fifth anniversary of the completion of the initial public offering (IPO), (b) in which we have total annual gross revenue of at least $1.235 billion, or (c) in which we are deemed to be a large accelerated filer, which means the market value of our common stock that is held by non-affiliates exceeds $700.0 million as of the prior June 30th, and (ii) the date on which we have issued more than $1.0 billion in non-convertible debt during the prior three-year period.

Effective as of December 31, 2025, the fifth anniversary of the closing of our IPO, we will no longer qualify as an “emerging growth company.”

Financial Information and Segments

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What changed 10-K

Item 1. Business