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Our actual results could differ materially from those anticipated in these forward-looking statements as a result of various factors, including those set forth in the section titled “Risk Factors” under Part II, Item 1A below.
−Removed: Overview and Pipeline
We are a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases.
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We are currently studying PBFT02 in FTD- GRN , for which there are currently no approved disease-modifying therapies.
−Removed: In addition to the continued clinical development of PBFT02 to treat FTD- GRN , we intend to pursue PBFT02 in additional adult neurodegenerative diseases where we believe increasing PGRN levels could provide benefit.
+Added: In light of the FDA’s recent guidance that a randomized controlled registrational study is required for PBFT02 in FTD- GRN and the associated ethical, logistical, and financial challenges, we are evaluating potential next steps in the clinical development of PBFT02 in FTD- GRN and FTD- C9orf72 .
Third-party preclinical studies have shown that increased PGRN levels reduce the pathologic accumulation of TAR DNA binding protein 43, or TDP-43.
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We have received positive regulatory feedback on the clinical pathway to treating FTD- C9orf72 patients and ALS patients with PBFT02.
−Removed: We are proceeding with clinical development of PBFT02 in FTD- C9orf72 patients, and have opened enrollment in the upliFT-D study for this population.
−Removed: We are party to a series of sublicense agreements, as amended, with Gemma Biotherapeutics, Inc., or Gemma, a newly formed genetic medicines company co-founded by Dr.
−Removed: James Wilson in connection with the outlicensing of PBGM01 for the treatment of GM1 gangliosidosis, or GM1, PBKR03 for the treatment of Krabbe disease, and PBML04 for the treatment of metachromatic leukodystrophy, or MLD, collectively the Outlicensed Programs, and such agreements, the Amended Gemma Sublicenses.
−Removed: In addition, we have entered into a Transition Services Agreement, as amended, and a research, collaboration and license agreement, or the Gemma Collaboration Agreement, with Gemma.
+Added: We have initiated clinical development of PBFT02 in FTD- C9orf72 patients in the upliFT-D trial for this population.
+Added: We have a preclinical research program to develop a genetic medicine to treat Huntington’s disease through the Gemma Collaboration Agreement.
+Added: Huntington’s disease, or HD, is an adult-onset, progressive neurodegenerative disease characterized by motor, cognitive, and behavioral deterioration, ultimately leading to death within approximately 15 to 20 years after symptom onset.
+Added: There are currently no disease-modifying therapies approved for the treatment of HD, and we estimate the prevalence of HD in the United States and Europe is approximately 70,000, based on available literature.
+Added: We are also party to a series of sublicense agreements, as amended, with Gemma in connection with the outlicensing of three pediatric programs we had previously advanced to clinical stage development, collectively the Outlicensed Programs, and such agreements, the Amended Gemma Sublicenses.
+Added: In addition, we entered into a Transition Services Agreement, as amended, with Gemma.
We refer to the Amended Gemma Sublicenses, the Transition Services Agreement, and the Gemma Collaboration Agreement, collectively, as the Outlicense Transaction Agreements.
−Removed: Prior to the execution of the Outlicense Transaction Agreements, we progressed four product candidates from preclinical to clinical stage development and had one active preclinical program in Huntington’s disease through our research collaboration with the Trustees of the University of Pennsylvania’s, or Penn’s, Gene Therapy Program, or GTP.
+Added: Prior to the execution of the Outlicense Transaction Agreements, we advanced our preclinical programs through our research collaboration with the Trustees of the University of Pennsylvania’s, or Penn’s, Gene Therapy Program, or GTP.
This collaboration provided access to differentiated scientific expertise for the conduct of rigorous preclinical studies to generate promising product candidates.
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† US/EU prevalence per third-party sources
+Added: In addition to the indications above, we believe ALS and AD represent future potential pipeline expansion opportunities for PBFT02.
+Added: However, as described above, we are currently evaluating potential next steps in the clinical development of PBFT02.
PBFT02 for the Treatment of FTD-GRN
−Removed: We are currently developing PBFT02, a gene replacement therapy which utilizes an AAV1 capsid to deliver a functional copy of GRN encoding for PGRN, for the treatment of FTD- GRN .
+Added: PBFT02 is a gene replacement therapy which utilizes an AAV1 capsid to deliver a functional copy of GRN encoding for PGRN, for the treatment of FTD- GRN .
FTD- GRN is an inheritable form of FTD caused by reductions in PGRN production due to mutations in the GRN gene.
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ICM administration of AAV1 to NHPs resulted in elevated CSF levels of human PGRN when compared with CSF levels in healthy human subjects, and in excess of levels achieved in NHPs with AAVhu68 or AAV5.
−Removed: We have an active IND application from the U.S.
+Added: We have an active Investigational New Drug, or IND, application from the U.S.
Food and Drug Administration, or the FDA, and approved clinical trial authorizations, or CTAs, in multiple countries for PBFT02.
We are conducting our upliFT-D trial, an international, multi-center, open-label, single-arm Phase 1/2 clinical trial of PBFT02 in patients with a diagnosis of symptomatic FTD- GRN .
−Removed: In June 2025, we reported biomarker data from patients in our upliFT-D trial.
−Removed: Dose 1 of PBFT02 (3.3e10 genome copies/g estimated brain weight, or 4.5e13 total genome copies) resulted in robust and durable increases in CSF PGRN levels, with concentrations increasing from below 3.0 ng/mL at baseline to a mean of 12.4 ng/mL at one month (n=7), 19.4 ng/mL at six months (n=6), 25.9 ng/mL at 12 months (n=4), and 23.8 ng/mL at 18 months (n=2).
+Added: In April 2026, we reported updated interim biomarker data from patients in our upliFT-D trial.
+Added: ● CSF and Plasma PGRN:
+Added: Dose 1 of PBFT02 (3.3e10 genome copies/g estimated brain weight, or 4.5e13 total genome copies) resulted in robust and durable increases in CSF PGRN levels, with concentrations increasing
+Added: from below 3.0 ng/mL at baseline to a mean of 12.4 ng/mL at one month (n=7), 19.4 ng/mL at six months (n=7), 22.8 ng/mL at 12 months (n=6), and 24.2 ng/mL at 18 months (n=3).
These levels of CSF PGRN are higher than the range found in healthy adult controls of 3.3 to 8.2 ng/mL (mean=4.8 ng/mL;
−Removed: CSF PGRN levels for the first patient treated with Dose 2 of PBFT02 (1.6e10 genome copies/g estimated brain weight, or 2.2e13 total genome copies) increased substantially from 1.5 ng/mL at baseline to 7.6 ng/mL at one month, approaching the upper limit of the range found in healthy adult controls.
+Added: Dose 2 of PBFT02 (1.6e10 genome copies/g estimated brain weight, or 2.2e13 total genome copies) increased substantially from 1.5 ng/mL at baseline to 8.6 ng/mL at one month (n=2), above the upper limit of the range found in healthy adult controls, and increased to 22.6 ng/mL at six months (n=1), achieving comparable CSF PGRN levels as Dose 1 at the same time point.
In contrast, following PBFT02 administration, plasma PGRN levels were unaltered, remaining similar to baseline concentrations and below mean levels found in healthy adult controls.
−Removed: Dose 1 of PBFT02 resulted in an average 4% increase in plasma neurofilament light chain, or NfL, levels, a biomarker associated with disease progression, compared to baseline at 12 months post-treatment (n=4).
−Removed: This change in plasma NfL after PBFT02 administration contrasts with an expected increase in plasma NfL levels of approximately 28% and 29% per year among untreated, symptomatic FTD- GRN patients, based on analysis of the ALLFTD natural history data and published natural history data (Saracino 2021), respectively.
−Removed: As of June 2025, interim safety highlights from PBFT02 in FTD- GRN patients (n=8) included:
−Removed: ● In five of eight patients, all treatment emergent adverse events were mild to moderate in severity.
−Removed: ● Three of eight patients experienced a total of four serious adverse events, or SAEs.
−Removed: Patient 1 experienced the asymptomatic SAEs of venous sinus thrombosis, or VST, and hepatotoxicity.
−Removed: Patient 7 also experienced the SAE of VST, which was asymptomatic and completely resolved prior to day 30 following treatment with anticoagulants.
−Removed: The first Dose 2 patient (Patient 8) experienced the SAE of pulmonary embolism in the setting of a concurrent systemic infection six weeks after receiving PBFT02.
−Removed: The patient responded to treatment with anticoagulants, and the SAE was assessed as possibly related to treatment.
−Removed: ● No evidence of dorsal root ganglion toxicity, as measured by nerve conduction studies, and no complications during ICM administration were observed across any of the eight treated patients.
−Removed: As of November 2025, we have completed dosing of Cohorts 1 and 2 in the upliFT-D study.
+Added: ● Whole Brain Atrophy:
+Added: PBFT02-treated patients with a global Clinical Dementia Rating, or CDR, score of 1 at baseline experienced a 64% reduction in whole brain atrophy at 12 months (n=2), on average, as compared to vMRI analysis of untreated global CDR 1 patients from the ALLFTD natural history data.
+Added: PBFT02-treated global CDR 1 patients experienced 3.1% atrophy at 12 months (n=2), on average, compared to 8.7% atrophy at 12 months (n=7) in ALLFTD sample of global CDR 1 individuals.
+Added: ● Frontotemporal Cortex Atrophy:
+Added: PBFT02-treated patients with global CDR score of 1 at baseline experienced a 54% reduction in frontotemporal cortex atrophy at 12 months (n=2), on average, as compared to vMRI analysis of untreated global CDR 1 patients from the ALLFTD natural history data.
+Added: PBFT02-treated global CDR 1 patients experienced 4.6% atrophy at 12 months (n=2), on average, compared to 9.9% atrophy at 12 months (n=7) in ALLFTD sample of global CDR 1 individuals
+Added: ● Plasma Neurofilament Light Chain, or NfL:
+Added: PBFT02-treated patients showed an average reduction of 1.0 pg/mL in plasma NfL levels at 12-months (n=6) compared to baseline.
+Added: In contrast, analysis of untreated symptomatic FTD- GRN patients from the ALLFTD natural history data showed an average increase of 13.5 pg/mL at 12 months (n=7) compared to baseline.
+Added: As of our March 2026 data disclosure, interim safety highlights from PBFT02 (n=10 FTD- GRN patients and n=1 FTD- C9orf72 patient) included:
+Added: ● Eight patients experienced a collective total of 32 treatment emergent adverse events, or TEAEs, considered related to PBFT02.
+Added: ● Two patients experienced a total of three serious TEAE considered related to PBFT02.
+Added: These included venous sinus thrombosis (2 patients) and hepatoxicity (1 patient).
+Added: These serious TEAE all occurred at Dose 1, were asymptomatic and responded to treatment.
+Added: ● One patient experienced one serious TEAE of pulmonary embolism in the setting of a concurrent systemic infection six weeks after receiving PBFT02 considered unrelated to PBFT02.
+Added: ● No evidence of thrombotic angiopathy, dorsal root ganglion toxicity, and no complications during ICM administration were observed across any of the eleven treated patients.
+Added: We have completed the dosing of Cohorts 1 and 2 in the upliFT-D trial in July 2025.
Cohort 1 consists of 5 patients who received Dose 1 of PBFT02, and Cohort 2 consists of 4 patients, split equally between Dose 1 and Dose 2 of PBFT02.
−Removed: We have amended the upliFT-D clinical trial protocol to introduce a short course of low dose prophylactic anticoagulation, a decision supported by study investigators and the Independent Data Monitoring Committee, or IDMC.
−Removed: We have implemented the amended protocol at initial trial sites and are currently enrolling Cohort 3, which is expected to consist of five to ten FTD-GRN patients receiving Dose 2 of PBFT02.
−Removed: In September 2025, we completed a Type D Chemistry, Manufacturing, and Controls meeting with the FDA and aligned on key elements of the analytical plan to establish comparability of our high-productivity, suspension-based PBFT02 manufacturing process.
−Removed: We expect to deliver on the following related to our upliFT-D trial for PBFT02 for the treatment of FTD- GRN :
−Removed: ● Report updated interim safety and biomarker data from Dose 2 in the first half of 2026;
−Removed: ● Seek regulatory feedback on registrational trial design in FTD-GRN in the first half of 2026.
+Added: Cohorts 1 and 2 included participants with a global Clinical Dementia Rating, or CDR, plus National Alzheimer’s Coordinating Center with Frontotemporal Lobar Degeneration, or NACC FTLD, score of 1 or 2 at baseline.
+Added: The global CDR rating is scored from 0 (normal/asymptomatic) to 3 (severe).
+Added: In advance of enrolling Cohort 3, which we expected to consist of 10 FTD- GRN patients receiving Dose 2 of PBFT02, we amended the upliFT-D clinical trial protocol to introduce a short course of low dose prophylactic anticoagulation.
+Added: We also amended the protocol to exclude patients with a global CDR score of 2 (moderate) at baseline and include only patients with global CDR scores of 0.5 (prodromal) or 1 (mild) at baseline.
+Added: We have initiated enrollment and dosing patients in Cohort 3 across our global trial sites.
+Added: In September 2025, we completed a Type D Chemistry, Manufacturing, and Controls meeting with the FDA and aligned on key elements of the analytical plan to establish comparability of product manufactured with our high-productivity, suspension-based PBFT02 manufacturing process to the current product being used in our ongoing clinical trial.
+Added: In April 2026, we disclosed feedback from a Type C meeting with the FDA regarding key elements of a future registrational trial design of PBFT02 for FTD- GRN in which FDA indicated that a randomized controlled registrational study design is required for PBFT02 in this indication.
+Added: In light of the ethical, logistical, and financial challenges posed
+Added: by a randomized controlled registrational trial, we are evaluating potential next steps in the clinical development of
+Added: PBFT02 in FTD- GRN and FTD- C9orf72 in the upliFT-D trial.
PBFT02 for the Treatment of FTD-C9orf72 and ALS
−Removed: We are also evaluating PBFT02 for the treatment of additional adult neurodegenerative diseases where we believe elevated PGRN levels could provide benefits.
+Added: We have initiated an evaluation of PBFT02 for the treatment of additional adult neurodegenerative diseases where we believe elevated PGRN levels could provide benefits.
This approach stems from PGRN’s pleiotropic cellular effects including the regulation of microglial activation and lysosomal function, and in particular its potential to ameliorate TDP-43 pathology.
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We anticipate that elevating neuronal PGRN levels in diseases with TDP-43 pathology may provide significant benefits to patients.
−Removed: We received positive regulatory feedback on the clinical pathway to treating FTD- C9orf72 with PBFT02 in the ongoing upliFT-D trial and revised the study to include two cohorts.
−Removed: Cohorts 4 and 5 will consist of three to five symptomatic FTD patients with C9orf72 gene mutations who will initially receive Dose 2 PBFT02.
−Removed: There are no disease modifying therapies approved for the treatment of FTD- C9orf72 .
+Added: We have initiated preclinical studies to extend these initial observations.
Based on available literature, we estimate the prevalence of FTD- C9orf72 in the United States and Europe is approximately 21,000.
−Removed: We have implemented the amended protocol at initial trial sites and are currently enrolling Cohort 4.
−Removed: Similarly, we received positive regulatory feedback on the clinical pathway to treating ALS with PBFT02.
+Added: There are no disease modifying therapies approved for the treatment of FTD- C9orf72 .
+Added: We received positive regulatory feedback on the clinical pathway to treating FTD- C9orf72 with PBFT02 in the ongoing upliFT-D trial, and we have planned for Cohorts 4 and 5 of upliFT-D to consist of three to five symptomatic FTD patients with C9orf72 gene mutations who will initially receive Dose 2 PBFT02.
+Added: We have initiated enrollment and dosing patients in Cohort 4 across our global trial sites.
+Added: Similarly, we received positive regulatory feedback on the clinical pathway to treating ALS with PBFT02 which we believe may represent a future pipeline opportunity.
PBFT02 for the Treatment of AD
−Removed: We believe that elevating PGRN levels has the potential to improve the course of AD in patients who carry the GRN rs5848 single nucleotide polymorphism, or GRN SNP.
+Added: We also believe that elevating PGRN levels has the potential to improve the course of AD in patients who carry the GRN rs5848 single nucleotide polymorphism, or GRN SNP.
The GRN SNP has an allele frequency of approximately 30% and is associated with reduced PGRN levels.
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Third party preclinical studies in animal models have demonstrated that low levels of PGRN may exacerbate AD pathology and, conversely, high levels of PGRN may reduce AD pathology.
+Added: This may represent a future pipeline opportunity for PBFT02, however, we are currently evaluating potential next steps in the clinical development of PBFT02 for the treatment of AD.
Clinical Supply
−Removed: Through our partners, we have manufactured the PBFT02 clinical supply to support completion of the ongoing Phase 1/2 clinical trial in FTD- GRN and FTD- C9orf72 , and initiation of a registrational study in FTD- GRN .
+Added: Through our partners, we have manufactured the PBFT02 clinical supply to support completion of the ongoing Phase 1/2 clinical trial in FTD- GRN and FTD- C9orf72 .
+Added: We are currently evaluating potential next steps in the clinical development of PBFT02 in FTD- GRN .
Active Research Programs
−Removed: We have one unnamed preclinical research program through the Gemma Collaboration Agreement and are exploring multiple potential treatment targets for Huntington’s disease.
−Removed: Beyond this program, as a result of the Gemma Collaboration Agreement, we also have the option to license programs for four additional new indications in CNS diseases from Gemma.
−Removed: Paused Research Programs
−Removed: We also have a research program through the Gemma Collaboration Agreement for Temporal Lobe Epilepsy, or TLE, which was previously conducted under the research, collaboration and licensing agreement with Penn, as amended, previously the Penn Agreement and now referred to as the Penn License Agreement.
−Removed: In order to reduce operating expenses, we have paused development of this program.
−Removed: Reverse Stock Split
−Removed: On May 28, 2025, our stockholders provided authorization for our Board of Directors to effect a reverse stock split to regain compliance with Nasdaq’s listing requirements.
−Removed: On July 14, 2025, we effected a 1-for-20 reverse stock split of our common stock, or the Reverse Stock Split.
−Removed: No fractional shares were issued in connection with the Reverse Stock Split.
−Removed: Stockholders who were otherwise entitled to receive fractional shares received the number of shares of Common Stock as rounded up to the nearest whole share.
−Removed: All share and per share amounts in the accompanying financial statements and notes thereto, including the stock options, restricted stock units, and employee stock purchase plan activity, have been adjusted retroactively to reflect the Reverse Stock Split for all periods presented.
+Added: We have a preclinical research program through the Gemma Collaboration Agreement to develop a genetic medicine to treat HD.
+Added: HD is an autosomal dominant disorder caused by a mutation in the huntingtin gene, or HTT , in which a CAG trinucleotide repeat tract in the DNA is expanded.
+Added: This leads to the expression of mutant huntingtin protein.
+Added: HTT CAG repeat tracts are unstable and can continue to elongate over time, termed somatic instability.
+Added: In neurons, CAG expansion occurs at different rates in different cells, and CAG expansion to above a certain threshold leads to neuronal dysfunction and death.
+Added: DNA repair proteins such as MSH3 play a key role in driving somatic instability in HD, by erroneously incorporating extra CAG repeats into HTT DNA in certain circumstances.
+Added: Published literature has shown that reducing somatic instability by decreasing MSH3 expression reduced disease pathology in HD mice.
+Added: Further, published human genetic studies have shown that certain genetic MSH3 variants which reduce somatic instability are associated with delayed disease onset and slowed progression in HD patients.
+Added: Our approach is to reduce somatic instability and thereby slow neurodegeneration in HD by suppressing MSH3 expression in the brain, via AAV-mediated delivery of a miRNA gene.
+Added: Beyond this program, through the Gemma Collaboration Agreement, we also have the option to license programs for four additional new indications in CNS diseases .
Business Overview
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Historically, we have funded our operations through the sale of convertible preferred stock and public offerings of common stock.
−Removed: Our net losses were $7.7 million and $19.3 million for the three months ended September 30, 2025 and 2024, respectively, and $32.5 million and $52.0 million for the nine months ended September 30, 2025 and 2024, respectively.
−Removed: As of September 30, 2025, we had an accumulated deficit of $691.8 million.
−Removed: Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures, and to a lesser extent, general and administrative expenditures.
+Added: Our net losses were $7.6 million and $15.4 million for the three months ended March 31, 2026 and 2025, respectively.
+Added: As of March 31, 2026, we had an accumulated deficit of $712.3 million.
+Added: Our primary use of cash is to fund operating expenses, which consist primarily of research and development expenditures and general and administrative expenditures.
Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of one or more of our current or future product candidates.
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Our net losses may fluctuate significantly from quarter to quarter and year to year, depending on the timing of our clinical trials and our expenditures on other research and development activities.
+Added: As of March 31, 2026, we had cash and cash equivalents of $33.3 million, which we do not expect to be sufficient to meet our capital requirements over the next 12 months.
We will need to raise substantial additional capital to support our continuing operations and pursue our growth strategy.
Until such time as we can generate significant revenue from product sales, if ever, we plan to finance our operations through the sale of equity, debt financings or other capital sources, which may include collaborations with other companies or other strategic transactions.
−Removed: There are no assurances that we will be successful in obtaining an adequate level of financing as and when needed to finance our operations on terms acceptable to us or at all.
+Added: There are no assurances that we will be successful in obtaining an adequate
+Added: level of financing as and when needed to finance our operations on terms acceptable to us or at all.
Any failure to raise capital as and when needed could have a negative impact on our financial condition and on our ability to pursue our business plans and strategies.
If we are unable to secure adequate additional funding, we may have to significantly delay, scale back or discontinue the development and commercialization of one or more product candidates or delay our pursuit of potential in-licenses or acquisitions.
−Removed: As of September 30, 2025, we had cash and cash equivalents of $52.8 million.
−Removed: We expect our existing cash and cash equivalents will enable us to fund our operating expenses and capital expenditure requirements into the first quarter of 2027.
+Added: As a result of these factors, there is substantial doubt about our ability to continue as a going concern within one year after the date the interim financial statements included in this Quarterly Report on Form 10-Q are issued.
+Added: On April 20, 2026, we announced that we have initiated a review of strategic alternatives to maximize shareholder value.
+Added: These strategic alternatives may include, but are not limited to, merger or acquisition transactions, a reverse merger, a sale of assets of the Company, strategic partnerships, licensing opportunities, or other potential paths.
+Added: We do not intend to provide updates on the strategic review until our board of directors approves a specific action or otherwise determines that disclosure is appropriate or required.
+Added: There can be no assurance that the process will result in any such transaction.
+Added: On April 28, 2026, in connection with our review of strategic alternatives, we announced a restructuring of our workforce, or the Restructuring Plan, to decrease operating expenses by reducing the workforce by approximately 75%.
+Added: The implementation of the Restructuring Plan should be substantially complete in the second and third quarters of 2026.
+Added: We estimate the aggregate severance and related costs for the Restructuring Plan will be approximately $3.3 million, which will be recorded primarily in the second quarter of 2026.
+Added: These estimates are subject to a number of assumptions, and actual results may differ materially.
+Added: We may also incur additional costs not currently contemplated due to events that may occur as a result of, or that are associated with, the Restructuring Plan as well as our review of strategic alternatives.
Financial Operations Overview
−Removed: License Agreement
+Added: License Agreements
University of Pennsylvania
−Removed: As a result of the Outlicense Transaction Agreements, we restructured our research, collaboration and licensing agreement with Penn, as amended, previously the Penn Agreement and now referred to as the Penn License Agreement.
+Added: As a result of the Outlicense Transaction Agreements, as discussed below, we restructured our research, collaboration and licensing agreement with Penn, as amended, previously the Penn Agreement and now referred to as the Penn License Agreement.
Pursuant to the Penn License Agreement, as of July 31, 2024, we (i) terminated the funding of discovery research programs;
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(iv) terminated the transaction fee payable to Penn in the event of certain corporate transactions;
−Removed: and (v) retained our current exclusive and non-exclusive licenses to our programs in FTD, GM1, Krabbe and MLD and certain platform technologies resulting from the discovery programs that we funded.
+Added: and (v) retained our current exclusive and non-exclusive licenses to our programs in FTD, GM1, Krabbe, MLD and certain platform technologies resulting from the discovery programs that we funded.
For our licensed programs in FTD, GM1, Krabbe and MLD, the Penn License Agreement requires that we make payments of up to $16.5 million per product candidate.
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In addition, on a product-by-product basis, we are obligated to make up to $55.0 million in sales milestone payments on each licensed product based on annual worldwide net sales of the licensed product in excess of defined thresholds.
−Removed: Pursuant to the Amended Gemma Sublicenses, Gemma is responsible for the payments to Penn related to the Outlicensed Programs.
+Added: Pursuant to the Amended Gemma Sublicenses, as discussed below, Gemma is responsible for the payments to Penn related to GM1, Krabbe and MLD, collectively the Outlicensed Programs.
Upon successful commercialization of a product using the licensed technology, we are obligated to pay to Penn, on a licensed product-by-licensed product and country-by-country basis, tiered royalties (subject to customary reductions) in the mid-single digits percentage on annual worldwide net sales of such licensed product.
In addition, other than the Amended Gemma Sublicenses, we are obligated to pay to Penn a percentage of sublicensing income, ranging from the mid-single digits to low double digits, for sublicenses under the Penn License Agreement.
−Removed: The agreement will expire on a licensed product-by-licensed product and country-by-country basis upon the later of (i) the expiration of the last valid claim of the licensed patent rights that covers the exploitation of such licensed product in such country, and (ii) the expiration of the royalty period.
+Added: The agreement will expire on a licensed product-by-licensed product and country-by-country basis upon the later of (i) the expiration of the last valid claim of the licensed patent rights that covers the exploitation of such licensed product in such country, and (ii) the
+Added: expiration of the royalty period.
Pursuant to the Amended Gemma Sublicenses, Gemma is responsible for the payments to Penn related to the Outlicensed Programs.
Gemma - Research, Collaboration and License Agreement
−Removed: In connection with the transfer of the Outlicensed Programs, on July 31, 2024, we entered into a research, collaboration and license agreement with Gemma, or the Gemma Collaboration Agreement.
−Removed: Pursuant to the Gemma Collaboration Agreement, (i) Gemma will conduct certain preclinical and IND-enabling work for our active research program in Huntington’s disease and a currently paused research program in TLE, which were previously being conducted by Penn under the Penn Agreement and (ii) Gemma will grant us options to conduct mutually agreed research programs in four new CNS indications.
+Added: In connection with the transfer of the Outlicensed Programs, on July 31, 2024, we entered into the Gemma Collaboration Agreement.
+Added: Pursuant to the Gemma Collaboration Agreement, (i) Gemma will conduct certain preclinical and IND application enabling work for our active research program in Huntington’s disease and a currently paused research program in TLE, which were previously being conducted by Penn under the Penn Agreement and (ii) Gemma will grant us options to conduct mutually agreed research programs in four new CNS indications.
The Gemma Collaboration Agreement requires that we make payments of up to (i) $16.5 million per product candidate in the aggregate for Huntington’s disease and any future CNS indications available to us under our four options and (ii) $39.0 million per product candidate in the aggregate arising from the research program for TLE.
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Gemma - Sublicense Agreements and Transition Services Agreement
−Removed: In connection with the transfer of the Outlicensed Programs to Gemma, on July 2024, we entered into the Gemma Sublicenses.
+Added: In connection with the transfer of the Outlicensed Programs to Gemma, in July 2024, we entered into the Gemma Sublicenses.
On May 7, 2025, we agreed to amend each of the Gemma Sublicenses to revise certain financial terms related to the Outlicensed Programs, or the Amended Gemma Sublicenses.
−Removed: Pursuant to the Amended Gemma Sublicenses, we are entitled to receive (i) an aggregate total of $15.0 million in initial payments for licenses and clinical product supply, of which $5.0 million was previously received and $5.0 million of which was due in May 2025;
+Added: Pursuant to the Amended Gemma Sublicenses, we are entitled to receive (i) an aggregate total of $15.0 million in initial payments for licenses and clinical product supply, of which $7.5 million was previously received, $2.5 million of which was due in May 2025, and $5.0 million of which was due in March 2026;
(ii) an additional $5.0 million contingent on Gemma completing certain business milestones;
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In addition, we entered into the Transition Services Agreement, as amended by the First Amendment to the Transition Services Agreement, dated January 31, 2025, pursuant to which, we provided transitional services at cost to Gemma through May 31, 2025, and are entitled to reimbursement for transitional services performed retroactively from March 1, 2024, related to the transfer of the Outlicensed Programs.
−Removed: As of September 30, 2025, we have collected $7.5 million in initial payments and $4.8 million in transition services payments under these agreements.
+Added: As of March 31, 2026, we have collected $7.5 million in initial payments and $4.8 million in transition services payments under these agreements.
In addition, we have applied $1.5 million in amounts owed to Gemma for the Huntington’s disease program against amounts due to us for transition services.
+Added: We refer to the Amended Gemma Sublicenses, the Transition Services Agreement, and the Gemma Collaboration Agreement, collectively, as the Outlicense Transaction Agreements.
Collaboration and Manufacturing and Supply Agreements
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The limited exclusive relationship under the Amended Catalent Agreements converts to a non-exclusive relationship (i) in the event Catalent fails to meet certain performance standards and (ii) following certain conditional events related to the divestiture by us of either FTD or GM1, in which case, if such events occur, we would pay Catalent certain fees.
−Removed: The outlicense of GM1 to Gemma under the Outlicense Transaction Agreements, and subsequent business decisions implemented by Gemma in their sole discretion, could be considered an event related to the divesture of GM1 under the Amended Catalent Agreements and require us to make payment of certain fees to Catalent, which fees are immaterial.
+Added: In the event of certain transactions, we may terminate the Amended Catalent Agreements for convenience with respect to such products, in which case, we would pay Catalent a certain termination fee.
+Added: The outlicensed and completed transition of GM1 to Gemma under the Outlicense Transaction Agreements is deemed by Catalent to be a divestiture under the Amended Catalent Agreements.
+Added: As such, we are required to make payment of $0.9 million to Catalent which is accrued as of March 31, 2026.
Components of Results of Operations
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● personnel expenses, including salaries, benefits and share-based compensation expense for employees engaged in research and development functions;
−Removed: ● expenses incurred at and for our lab facilities, including rent, utilities, depreciation and amortization;
−Removed: ● expenses incurred to conduct the necessary preclinical studies and clinical trials required to obtain regulatory approval, including payments to clinical research organizations, or CROs, and payments to Gemma for preclinical research and development;
+Added: ● expenses incurred at and for our lab facilities, including rent, utilities, depreciation, amortization and maintenance;
+Added: ● expenses incurred to conduct the necessary preclinical studies and clinical trials required to obtain regulatory approval, including payments to clinical research organizations, or CROs, and payments to Gemma and Penn for preclinical research and development;
● expenses and fees paid to consultants who assist with research and development activities;
−Removed: expenses incurred under agreements with contract development and manufacturing organizations, or CDMOs, including the cost of acquiring and manufacturing preclinical study and clinical trial materials.
+Added: expenses incurred under agreements with contract development and manufacturing organizations, or CDMOs, including the cost of acquiring and manufacturing preclinical trial and clinical trial materials.
We track outsourced development expenses and other external research and development expenses to specific product candidates on a program-by-program basis, such as fees paid to CROs, CDMOs and research laboratories in connection with our preclinical development, process development, manufacturing and clinical development activities, expenses incurred under our prior collaboration with Penn, and expenses incurred under the Gemma Collaboration Agreement.
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Product candidates in later stages of clinical development generally have higher development expenses than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials.
−Removed: We expect that the reduction of expenses related to the Outlicensed Programs pursuant to the Outlicense Transaction Agreements will offset the increased expenses of advancing our remaining product candidates.
−Removed: As such, we expect our research and development expenses to remain consistent in the near future.
−Removed: If our product candidate portfolio progresses into later-stage clinical trials, we expect that our research and development expenses will increase in the future to support continued research and development activities and production of clinical supply.
+Added: In April 2026, we announced we are evaluating potential next steps in the clinical development of PBFT02, and in connection with our review of strategic alternatives, we announced a restructuring of our workforce to decrease operating expenses by approximately 75%.
+Added: As a result, we expect our research and development expenses to decrease in the near future.
General and Administrative Expenses
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General and administrative expenses also include professional and consulting services, headquarters facility costs, including rent, utilities, depreciation, amortization and maintenance, legal expenses related to intellectual property, litigation and corporate matters, insurance expense, expenses related to contract modifications or terminations, software expenses, expenses incurred to engage with patient advocacy organizations, and recruitment related expenses.
−Removed: We expect our general and administrative expenses to remain consistent in the near future.
−Removed: If our product candidate portfolio progresses into later-stage clinical trials, we expect that our general and administrative expenses will increase in the future to support our continued research and development activities and potential commercialization efforts.
−Removed: These increases will likely include increased expenses related to the hiring of additional personnel in general and administrative functions, and expenses related to pre-commercialization efforts.
−Removed: If any of our current or future product candidates obtain regulatory approval, we expect that we would incur significantly increased expenses associated with building a commercial sales and marketing team.
+Added: In connection with our review of strategic alternatives, we announced a restructuring of our workforce to decrease operating expenses by approximately 75%.
+Added: As a result, we expect our general and administrative expenses to decrease in the near future.
Impairment of Long-Lived Assets
Impairment of long-lived assets consists of non-cash impairment charges recorded to our assets.
−Removed: We review long-lived assets, such as the right of use assets, or ROU assets, or property and equipment, for impairments when events or changes in circumstances indicate the carrying amount of the assets may not be recoverable.
−Removed: During the nine months ended September 30, 2025, we recognized impairment expenses related to property and equipment and certain other assets in connection with the announcement to reduce our workforce by 55% and cease our lab operations in Hopewell, New Jersey.
−Removed: We reassessed asset groups at the lab in Hopewell, New Jersey, and evaluated such asset groups for impairment.
+Added: We review long-lived assets, such as the right of use assets, or ROU assets, and property and equipment, for impairments when events or changes in circumstances indicate the carrying amount of the assets may not be recoverable.
+Added: During the three months ended March 31, 2026, we did not recognize impairment expenses.
+Added: During the three months ended March 31, 2025, we recognized impairment expenses as a result of the announcement in January 2025 to reduce our workforce by 55% and cease our lab operations in Hopewell, New Jersey.
+Added: We reassessed asset groups and evaluated such asset groups for impairment.
We determined the laboratory equipment was a separate asset group based on management’s implemented plans to sell the laboratory equipment and estimated the fair value of the laboratory equipment based on the estimated future cash flows from the sale of such equipment.
+Added: Net Gain on Lease Termination
+Added: The net gain on lease termination was a result of the Hopewell Lease Termination Agreement, for the laboratory lease agreement related to our laboratory facility in Hopewell, New Jersey.
+Added: As a result of the Hopewell Lease Termination Agreement, we recognized a net gain on the lease termination comprised of a gain on the write-off of assets and liabilities for operating leases and a loss on the disposal of property and equipment.
Other Income (Expense), Net
−Removed: Other income (expense), net consists of interest earned on our cash equivalents and marketable securities, amortization of premium and discount on our marketable securities, and income from subleases.
+Added: Other income (expense), net consists of interest earned on our cash equivalents and marketable securities, amortization of premium and discount on our marketable securities, income from subleases, and the sale of certain tax credits.
Results of Operations
−Removed: Comparison of the three months ended September 30, 2025 and 2024
−Removed: The following table sets forth our results of operations for the three months ended September 30, 2025 and 2024:
+Added: Comparison of the three months ended March 31, 2026 and 2025
+Added: The following table sets forth our results of operations for the three months ended March 31, 2026 and 2025:
Three months ended
−Removed: September 30,
(in thousands)
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Impairment of long-lived assets
+Added: Net gain on lease termination
Loss from operations
1 unchanged sentence
Research and Development Expenses
−Removed: Research and development expenses decreased by $4.4 million to $4.3 million for the three months ended September 30, 2025 from $8.7 million for the three months ended September 30, 2024.
+Added: Research and development expenses decreased by $3.6 million to $4.1 million for the three months ended March 31, 2026 from $7.7 million for the three months ended March 31, 2025.
The decrease was primarily due to the following:
● a decrease of $2.1 million in wages and benefits due to a lower headcount following our restructuring in January 2025;
−Removed: ● a decrease of $0.8 million in facility and other expenses related to decreased depreciation expenses in connection with the disposal of our laboratory equipment;
+Added: ● a decrease of $1.0 million in facility and other expenses related to decreased rent expenses in connection with the Hopewell Lease Termination Agreement;
● a decrease of $0.6 million in clinical operations expenses due to decreased activity in the GM1 program partially offset by increased activity supporting the FTD program;
● a decrease of $0.1 million in share-based compensation expense related to reductions in headcount.
−Removed: ● a decrease of $0.4 million in professional fees;
−Removed: ● a decrease of $0.3 million in chemistry, manufacturing and control expenses primarily related to reduced costs in connection with the restructuring and ceased operations of the lab in Hopewell, New Jersey;
−Removed: ● a decrease of $0.3 million in preclinical research expenses primarily related to reduced Huntington’s disease program expenses.
−Removed: General and Administrative Expenses
−Removed: General and administrative expenses decreased by $3.0 million to $4.3 million for the three months ended September 30, 2025 from $7.3 million for the three months ended September 30, 2024.
−Removed: The decrease was primarily due to the following:
−Removed: ● a decrease of $1.3 million in facility and other expenses primarily due to accruals for litigation matters in the three months ended September 30, 2024 which were subsequently reversed;
−Removed: ● a decrease of $1.0 million and $0.4 million in wages and benefits and share-based compensation expense, respectively, related to reductions in headcount;
−Removed: ● a decrease of $0.3 million in professional fees.
−Removed: Impairment of Long-Lived Assets
−Removed: During the three months ended September 30, 2025, we did not record any impairment expense.
−Removed: During the three months ended September 30, 2024, we recorded $4.8 million of impairment expense related to the Hopewell Laboratory Space.
−Removed: The impairment charges consisted of $2.5 million and $2.3 million recorded to the ROU assets and property and equipment, net, respectively.
−Removed: Other Income (Expense), Net
−Removed: Other income (expense), net decreased by $0.5 million to $0.9 million for the three months ended September 30, 2025 from $1.4 million for the three months ended September 30, 2024.
−Removed: The decrease was primarily due to the following:
−Removed: ● a decrease of $0.6 million in the amortization of premium and discount on our marketable securities.
−Removed: The decrease was partially offset by:
−Removed: ● an increase of $0.1 million attributable to income from subleases.
−Removed: Comparison of the nine months ended September 30, 2025 and 2024
−Removed: The following table sets forth our results of operations for the nine months ended September 30, 2025 and 2024:
−Removed: Nine months ended
−Removed: September 30,
−Removed: (in thousands)
−Removed: Operating expenses:
−Removed: Research and development
−Removed: General and administrative
−Removed: Impairment of long-lived assets
−Removed: Loss from operations
−Removed: Other income (expense), net
−Removed: Research and Development Expenses
−Removed: Research and development expenses decreased by $12.7 million to $17.9 million for the nine months ended September 30, 2025 from $30.6 million for the nine months ended September 30, 2024.
−Removed: The decrease was primarily due to the following:
−Removed: ● a decrease of $3.7 million in preclinical research expenses primarily related to the termination of our discovery research obligation under the Penn Agreement and reduced Huntington’s disease program expenses;
−Removed: ● a decrease of $3.3 million in wages and benefits due to a lower headcount from our restructuring in January 2025;
−Removed: ● a decrease of $1.9 million in facility and other expenses related to decreased depreciation expenses in connection with the disposal of our laboratory equipment;
−Removed: ● a decrease of $1.4 million in share-based compensation expense related to reductions in headcount;
−Removed: ● a decrease of $1.2 million in chemistry, manufacturing and control expenses primarily related to reduced costs in connection with the restructuring and ceased operations of the lab in Hopewell, New Jersey;
−Removed: ● a decrease of $1.1 million in professional fees;
−Removed: ● a decrease of $0.1 million in clinical operations expenses due to decreased activity in the GM1 program partially offset by increased activity supporting the FTD program.
+Added: These decreases were partially offset by:
+Added: ● an increase of $0.2 million in preclinical research expenses related to Huntington’s disease program expenses.
General and Administrative Expenses
−Removed: General and administrative expenses decreased by $5.3 million to $15.0 million for the nine months ended September 30, 2025 from $20.3 million for the nine months ended September 30, 2024.
+Added: General and administrative expenses decreased by $1.3 million to $4.8 million for the three months ended March 31, 2026 from $6.1 million for the three months ended March 31, 2025.
The decrease was primarily due to the following:
−Removed: ● a decrease of $2.1 million in professional fees;
−Removed: ● a decrease of $1.5 million in facility and other expenses primarily due to accruals for litigation matters in the nine months ended September 30, 2024 which were subsequently reversed;
● a decrease of $0.9 million and $0.2 million in wages and benefits and share-based compensation expense, respectively, related to reductions in headcount;
+Added: ● a decrease of $0.3 million in facility and other expenses.
+Added: These decreases were partially offset by:
+Added: ● an increase of $0.1 million in professional fees.
Impairment of Long-Lived Assets
−Removed: During the nine months ended September 30, 2025, we recorded $2.6 million of impairment expense related to laboratory equipment and certain other assets which were revalued and subsequently sold from the Hopewell Laboratory Space.
−Removed: During the nine months ended September 30, 2024, we recorded $5.2 million of impairment expense primarily consisting of $2.5 million and $2.3 million recorded to the ROU assets and property and equipment, net, respectively, related to the Hopewell Laboratory Space.
−Removed: In addition, we recorded $0.4 million of impairment expenses related to property and equipment for a construction in progress asset we no longer planned to deploy.
+Added: During the three months ended March 31, 2026, we did not record any impairment expense.
+Added: During the three months ended March 31, 2025, we recorded $2.6 million of impairment expense related to laboratory equipment and certain other assets which were revalued and subsequently sold from the Hopewell laboratory space.
+Added: Net Gain on Lease Termination
+Added: During the three months ended March 31, 2026, we recorded a $0.6 million net gain on the termination of the Hopewell Laboratory Lease Agreement.
+Added: The net gain was comprised of a $3.8 million gain on the write-off of assets and liabilities for operating leases offset by a $3.2 million net loss on disposal of property and equipment.
+Added: During the three months ended March 31, 2025, we did not record any lease termination gain or loss.
Other Income (Expense), Net
−Removed: Other income (expense), net decreased by $1.2 million to $2.9 million for the nine months ended September 30, 2025 from $4.1 million for the nine months ended September 30, 2024.
−Removed: The decrease was primarily due to the following:
−Removed: ● a decrease of $1.7 million in the amortization of premium and discount on our marketable securities.
−Removed: The decrease was partially offset by:
−Removed: ● an increase of $0.5 million attributable to income from subleases.
+Added: Other income (expense), net decreased by $0.4 million to $0.7 million for the three months ended March 31, 2026 from $1.1 million for the three months ended March 31, 2025.
+Added: The decrease was due to a $0.4 million decrease in the amortization of premium and discount on our marketable securities.
Liquidity and Capital Resources
−Removed: As of September 30, 2025, we had $52.8 million in cash and cash equivalents and had an accumulated deficit of $691.8 million.
−Removed: We expect our existing cash and cash equivalents will enable us to fund our operating expenses and capital expenditure requirements into the first quarter of 2027.
+Added: As of March 31, 2026, we had $33.3 million in cash and cash equivalents and had an accumulated deficit of $712.3 million.
Funding Requirements
−Removed: Our primary use of cash is to fund operating expenses, most significantly research and development expenditures.
+Added: Our primary use of cash is to fund operating expenses.
Cash used to fund operating expenses is impacted by the timing of when we pay these expenses, as reflected in the change in our outstanding accounts payable, accrued expenses and prepaid expenses.
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Because of the numerous risks and uncertainties associated with the development and commercialization of our product candidates, we are unable to estimate the amounts of increased capital outlays and operating expenditures associated with our current and anticipated clinical studies.
−Removed: Until such time, if ever, as we can generate substantial product revenue, we expect to finance our operations through a combination of equity offerings, debt financings, collaborations, strategic alliances and marketing, distribution or licensing arrangements.
+Added: Until such time, if ever, as we can generate substantial product revenue, we expect to finance our operations through a combination of equity offerings, debt financings, collaborations, strategic alliances, reverse merger or other business combination transactions, and marketing, distribution or licensing arrangements.
To the extent that we raise additional capital through the sale of equity or convertible debt securities, existing stockholders’ ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect existing stockholders’ rights as common stockholders.
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If we raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or drug candidates, or grant licenses on terms that may not be favorable to us.
+Added: If we pursue a reverse merger or other business combination transaction, we may be subject to significant transaction costs, our stockholders may experience substantial dilution, our management team may change, and we may not achieve the anticipated benefits of such a transaction.
If we are unable to raise additional funds through equity or debt financings or other arrangements when needed, we may be required to delay, limit, further reduce or terminate our research, product development or future commercialization efforts, or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
On March 5, 2021, we entered into a Sales Agreement, or the Sales Agreement, with Cowen and Company, LLC, or Cowen, relating to the applicable terms of at-the-market equity offerings, or the ATM Facility, pursuant to which we may, but are not obligated to, offer and sell, from time to time, shares of our common stock with an aggregate offering price up to $125.0 million through Cowen, as sales agent in the ATM Facility.
−Removed: We issued 6,000,000 shares (300,000 shares adjusted for the Reverse Stock Split) of common stock under the ATM Facility, resulting in net proceeds of $8.7 million, after deducting offering costs of $0.3 million in March 2024.
−Removed: We are limited in our capacity to offer and sell shares of our common stock under this sales agreement pursuant to the prospectus supplement to our shelf registration statement on Form S-3, filed on March 5, 2025.
−Removed: As of September 30, 2025, $15.8 million of capacity remains available to be sold under the ATM Facility.
+Added: We issued 300,000 shares of common stock under the ATM Facility, resulting in net proceeds of $8.7 million, after deducting offering costs of $0.3 million in March 2024.
+Added: As a result of our public float as of January 9, 2026, we are currently limited to $21.1 million in our capacity to offer and sell shares of our common stock under the Sales Agreement pursuant to our shelf registration statement on Form S-3, filed on March 4, 2024.
+Added: As of March 31, 2026, $15.8 million of capacity remains available to be sold under the ATM Facility.
The following table shows a summary of our cash flows for the periods indicated:
−Removed: Nine months ended
−Removed: September 30,
+Added: Three months ended
(in thousands)
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Net Cash Provided by (Used in) Operating Activities
−Removed: During the nine months ended September 30, 2025, we used $25.0 million of net cash in operating activities.
−Removed: Cash used in operating activities reflected a net loss of $32.5 million and a decrease in our operating assets of $1.8 million and non-cash charges of $5.7 million related to depreciation, amortization, share-based compensation, amortization of premium and discount, net, impairment of long-lived assets, and other non-cash items.
−Removed: The primary use of cash was to fund our operations related to the development of our product candidates.
−Removed: During the nine months ended September 30, 2024, we used $39.5 million of net cash in operating activities.
−Removed: Cash used in operating activities reflected a net loss of $52.0 million, partially offset by a net decrease in our operating assets of $1.5 million, and net non-cash charges of $11.0 million related to depreciation, amortization, share-based compensation, amortization of premium and discount, net, and impairment of long-lived assets.
+Added: During the three months ended March 31, 2026, we used $13.0 million of net cash in operating activities.
+Added: Cash used in operating activities reflected a net loss of $7.6 million and a decrease in our operating assets of $5.5 million offset by non-cash charges of $0.1 million related to depreciation, amortization, share-based compensation, loss recognized on disposal of long-lived assets, and other non-cash items.
+Added: The primary uses of cash were to fund our operations related to the development of our product candidates and the payment of the lease termination fee in connection with the Hopewell Lease Termination Agreement.
+Added: During the three months ended March 31, 2025, we used $13.8 million of net cash in operating activities.
+Added: Cash used in operating activities reflected a net loss of $15.4 million and a decrease in our operating assets of $2.2 million, partially offset by non-cash charges of $3.8 million related to depreciation, amortization, share-based compensation, amortization of premium and discount, net, and impairment of long-lived assets.
The primary use of cash was to fund our operations related to the development of our product candidates.
Net Cash Provided by (Used in) Investing Activities
−Removed: During the nine months ended September 30, 2025 , we had sales and maturities of $39.0 million in marketable securities and received cash proceeds of $1.2 million related to the sale of property and equipment and certain other assets.
−Removed: We did not make any purchases of property and equipment for the nine months ended September 30, 2025.
−Removed: During the nine months ended September 30, 2024, we purchased $72.6 million in marketable securities, and had sales and maturities of $113.9 million in marketable securities.
−Removed: Purchases of property and equipment were de minimis for the nine months ended September 30, 2024.
+Added: During the three months ended March 31, 2026 , we received de minimis cash proceeds related to the sale of property and equipment.
+Added: During the three months ended March 31, 2025, we had sales and maturities of $39.0 million in marketable securities and received cash proceeds of $0.6 million related to the sale of property and equipment and certain other assets.
Net Cash Provided by (Used in) Financing Activities
−Removed: During the nine months ended September 30, 2025, we received de minimis proceeds from the issuance of common stock under the ESPP.
−Removed: During the nine months ended September 30, 2024, we received $8.7 million in net proceeds from the issuance of common stock under the ATM Facility.
−Removed: We received gross proceeds of $9.0 million, net of offering costs of $0.3 million.
−Removed: We received $0.1 million in proceeds from the issuance of common stock under the ESPP and exercises of employee stock options.
+Added: During the three months ended March 31, 2026 and 2025, we had no gross receipts or outflows of cash related to financing activities .
Contractual Obligations and Other Commitments
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Our sublease agreements do not relieve us from our primary obligations under the 2005 Market Street Lease Agreement, however, we do expect cash inflows from the agreements to partially offset our future obligations for the duration of the sublease agreements.
−Removed: We lease approximately 62,000 square feet of laboratory space in Hopewell, New Jersey, or the Laboratory Lease Agreement.
−Removed: The lease will expire in March 2036.
−Removed: Our sublease agreement does not relieve us from our primary obligations under the Laboratory Lease Agreement, however, we do expect cash inflows from the agreement to partially offset our future obligations for the duration of the sublease agreement.
−Removed: The aggregate estimated rent payments due over the remaining terms of our leases and sublease are $38.2 million.
+Added: The aggregate estimated rent payments due over the remaining terms of our leases are $7.4 million.
Under the exclusive relationship under the Amended Catalent Agreements, following certain conditional events related to the divestiture by us of either FTD or GM1, we would pay Catalent certain fees.
−Removed: The outlicense of GM1 to Gemma under the Outlicense Transaction Agreements, and subsequent business decisions implemented by Gemma in their sole discretion, could be considered an event related to the divesture of GM1 under the Amended Catalent Agreements and require us to make payment of certain fees to Catalent, which fees are immaterial.
+Added: In the event of certain transactions,
+Added: we may terminate the Amended Catalent Agreements for convenience with respect to such products, in which case, we would pay Catalent a certain termination fee.
+Added: The outlicense and completed transition of GM1 to Gemma under the Outlicense Transaction Agreements, is deemed by Catalent to be a divestiture under the Amended Catalent Agreements.
+Added: As such, we are required to make payment of $0.9 million to Catalent which has been accrued as of March 31, 2026.
These contractual obligations and commitments are associated with contracts that are enforceable and legally binding and that specify all significant terms, including fixed or minimum services to be used, fixed, minimum or variable price provisions, and the approximate timing of the actions under the contracts.
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Critical Accounting Policies and Estimates
−Removed: During the nine months ended September 30, 2025, there were no material changes to our critical accounting policies and estimates from those described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Policies and Estimates” in our 2024 Annual Report filed on Form 10-K.
−Removed: JOBS Act Accounting Election
−Removed: We are an “emerging growth company” as defined in the Jumpstart Our Business Startups Act of 2012, or the JOBS Act.
−Removed: Under the JOBS Act, emerging growth companies can delay adopting new or revised accounting standards issued subsequent to the enactment of the JOBS Act until such time as those standards apply to private companies.
−Removed: We have elected to use this extended transition period for complying with new or revised accounting standards that have different effective dates for public and private companies until the earlier of the date we (i) are no longer an emerging growth company or (ii) affirmatively and irrevocably opt out of the extended transition period provided in the JOBS Act.
−Removed: As a result, our financial statements may not be comparable to companies that comply with new or revised accounting pronouncements as of public company effective dates.
−Removed: We will remain an emerging growth company until December 31, 2025.
−Removed: We are also a “smaller reporting company,” meaning that the market value of our stock held by non-affiliates is less than $700.0 million and our annual revenue is less than $100.0 million during the most recently completed fiscal year.
−Removed: We may continue to be a smaller reporting company if either (i) the market value of our stock held by non-affiliates is less than $250.0 million or (ii) our annual revenue is less than $100.0 million during the most recently completed fiscal year and the market value of our stock held by non-affiliates is less than $700.0 million.
−Removed: If we are a smaller reporting company at the time we cease to be an emerging growth company, we may continue to rely on exemptions from certain disclosure requirements that are available to smaller reporting companies.
−Removed: Specifically, as a smaller reporting company we may choose to present only the two most recent fiscal years of audited financial statements in our Annual Report on Form 10-K and, similar to emerging growth companies, smaller reporting companies have reduced disclosure obligations regarding executive compensation.
+Added: During the three months ended March 31, 2026, there were no material changes to our critical accounting policies and estimates from those described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Critical Accounting Policies and Estimates” in our 2025 Annual Report filed on Form 10-K.
Recent Accounting Pronouncements
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Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.