What changed in this filing

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Item 2. Management’s Discussion and Analysis

2026-05-14 compared with 2025-11-05 · 67 added, 120 removed, 130 unchanged (59% of the section changed)

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We undertake no obligation to publicly update these forward-looking statements, whether as a result of new information, future events or otherwise.

You should read the “Risk Factors” section included in our 2024 Annual Report, in addition to the (“Risk Factors”) and “Cautionary Note Regarding Forward-Looking Statements” sections of this Quarterly Report for a discussion of important factors that could cause actual results to differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.

You should read the “Risk Factors” section included in our 2025 Annual Report, in addition to the“Cautionary Note Regarding Forward-Looking Statements” sections of this Quarterly Report for a discussion of important factors that could cause actual results to differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.

We are a clinical-stage biotechnology company focused on developing treatments for patients suffering from complex rare diseases using our novel cell penetrating peptide (“CPP”) technology platform.

Our lead product candidate, nomlabofusp (nomlabofusp is the International Nonproprietary Name and the United States Adopted Name for CTI-1601), is a subcutaneously administered, recombinant fusion protein intended to deliver tissue frataxin (“FXN”), an essential protein, to the mitochondria of patients with Friedreich's ataxia (“FA”).

Our lead product candidate, nomlabofusp, is a subcutaneously administered, recombinant fusion protein intended to deliver frataxin (“FXN”), an essential protein, to the mitochondria of patients with Friedreich's ataxia (“FA”).

FA is a rare, progressive, and fatal disease in which patients are unable to produce sufficient FXN due to a genetic abnormality.

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Nomlabofusp represents the first potential therapy designed to systemically increase FXN levels in patients with FA.

We believe that our CPP platform, which enables a therapeutic molecule to cross a cell membrane in order to reach intracellular targets, has the potential to enable the treatment of other rare and orphan diseases.

We believe that our CPP platform, which enables a therapeutic molecule to cross a cell membrane in order to reach intracellular targets, also has the potential to enable the treatment of other rare and orphan diseases.

We intend to use our proprietary platform to target additional orphan indications characterized by deficiencies in or alterations of intracellular content or activity.

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Nomlabofusp Program Update

We initiated human clinical studies of nomlabofusp in 2019.

Since then, we have completed four clinical studies:

(i) two Phase 1 clinical studies, (ii) a Phase 2 dose exploration study and (iii) a PK run-in study in adolescents (12-17 years old).

In 2024, we initiated our open label extension study in patients with FA.

The open label extension study's eligibility criteria only allowed patients who previously participated in our prior nomlabofusp Phase 1 or Phase 2 studies.

In 2025, we changed the name of the open label extension study to the open label study, due to the inclusion of patients who had not participated in prior nomlabofusp clinical studies.

We have engaged in multiple discussions and interactions with the U.S.

Food and Drug Administration (“FDA”) in connection with our clinical development of nomlabofusp.

Communications remain ongoing.

We currently participate in the FDA’s Center for Drug Evaluation and Research (“CDER”) Support for Clinical Trials Advancing Rare Disease Therapeutics (“START”) pilot program.

The objective of this program is to accelerate the development of drugs for rare diseases that lead to significant disability or death by facilitating frequent advice and regular communication with the FDA staff to expedite the review process.

We have also had numerous interactions with the European Medicines Agency (“EMA”) and the United Kingdom’s Medicines and Healthcare Regulatory Agency (“MHRA”) regarding the clinical development of nomlabofusp.

We have received Orphan Drug Designation and Fast Track Designation from the FDA.

We have been granted orphan drug designation in the European Union (the “EU”) and access to the EU’s Priority Medicines

Program (“PRIME”) Scheme.

We have also received access to the EMA’s Innovative Licensing and Access Pathway (“ILAP”).

All these programs are designed to facilitate drug development of therapeutics for rare and/or orphan indications.

We have Orphan Drug Designation, Fast Track Designation, Pediatric Rare Disease Designation and Breakthrough Therapy Designation from the FDA and have been granted orphan drug designation and access to the European Medicines Agency’s (“EMA’s”) Priority Medicines Program (“PRIME”) scheme in the European Union (the “EU”).

We have also received access in the United Kingdom (the “UK”) to the Medicines and Healthcare Regulatory Agency’s (“MHRA”) Innovative Licensing and Access Pathway (“ILAP”).

These programs are designed to facilitate development of certain therapeutics such as those for rare and serious diseases, and those that have the potential to meet an unmet medical need.

The FDA’s Center for Drug Evaluation and Research selected nomlabofusp as one of a few drug development programs for participation in the Support for Clinical Trials Advancing Rare Disease Therapeutics (“START”) Pilot Program.

The objective of the program is to accelerate the development of drugs for rare diseases that lead to significant disability or death by facilitating frequent advice and regular communication with the FDA staff to expedite the review process of biologics and drugs.

We have engaged in multiple discussions and interactions with the FDA in connection with our clinical development of nomlabofusp and communications remain ongoing.

We have also had numerous interactions with the EMA, the MHRA, and Canada’s Health Canada regarding the clinical development of nomlabofusp.

We have completed four clinical studies:

(i) two Phase 1 clinical studies in adults, (ii) a Phase 2 dose exploration study in adults and (iii) a Phase 1 pharmacokinetic (“PK”) run-in study in adolescents (12-17 years old).

We currently have an ongoing open label (“OL”) study (previously referred to as the Open Label Extension, (“OLE”) study) in adults and adolescents with FA.

Recent significant developments are as follows:

• In December 2024, we reported positive initial data from our ongoing open label study.

This data included safety, FXN levels, clinical and pharmacokinetic data.

Also in December 2024, we announced that we were increasing the dose in the open label study to 50 mg of nomlabofusp daily for then currently enrolled participants.

All newly enrolled participants will start dosing at 50 mg of nomlabofusp daily.

In March 2025, we announced that our Safety Monitoring Team had identified anaphylaxis as an adverse drug reaction likely associated with nomlabofusp.

• In January 2025, we initiated dosing of adolescents (12-17 years old) in our PK run-in study for patients with FA.

Study participants were randomized 2:1 to receive either nomlabofusp at a weight-base dose expected to match the PK of adults receiving the 50 mg dose, or placebo, daily for seven days.

We completed dosing of 14 adolescents in March 2025.

• In February 2025, the FDA accepted the data supporting the comparability of the lyophilized drug product to the frozen solution and agreed with our plans to introduce the lyophilized product into our clinical development program.

The frozen solution is the dosage form currently being used in the open label study.

In July 2025, we began to introduce the lyophilized product formulation, which is intended for commercialization, into the open label study with new study participants.

• In March 2025, we announced that FDA stated as part of a START pilot program that it is open to considering the use of FXN concentration as a reasonably likely surrogate endpoint (“RLSE”) and the acceptability of FXN concentration as an RLSE to support accelerated approval will be a matter of review in a future marketing application.

The FDA recommended focusing on assessments of skin FXN concentrations rather than buccal FXN concentrations due to more consistent sampling and less variability.

FDA acknowledged that data we recently submitted appear to support a relationship between increased FXN concentrations in skin cells and relevant tissues such as the heart, dorsal root ganglion and skeletal muscle.

FDA also acknowledged that the nonclinical studies we submitted were performed at relevant human doses.

FDA also suggested that we consider exploring the relationship between increases in FXN in skin and changes in pharmacodynamic (“PD”) markers such as lipid profiles and/or clinical measures to provide additional support for the use of FXN as an RLSE.

We continue to interact with the FDA under the START pilot program.

• In June 2025, the FDA provided safety database recommendations for a Biologics License Application, (“BLA”) seeking accelerated approval, which included evaluating safety in at least 30 participants with continuous study drug exposure for six months and a subset of at least 10 of those participants with continuous study drug exposure for one year, with the large majority of data coming from participants receiving the 50 mg dose.

• In July 2025, we announced the publication of nonclinical data evaluating the mechanism of action, pharmacodynamics and pharmacology of nomlabofusp as a novel FXN protein replacement therapy designed to address the underlying cause of FA in two peer-reviewed articles.

These data were included in the briefing package reviewed by the U.S.

FDA in support of using skin FXN concentrations as a RLSE for our registrational program seeking accelerated approval for nomlabofusp.

• In September 2025, we announced positive data from our ongoing long-term open label study:

o In four completed studies and the ongoing open label study, 65 participants received at least one dose of nomlabofusp, including 39 in the open label study, with 14 on treatment for at least six months and eight for over one year in the open label study.

o Consistent directional improvements across four key clinical outcomes observed in the open label study relative to a Friedreich’s Ataxia Clinical Outcomes Measure Study (FACOMS) reference population and the observed increase in skin FXN levels.

We believe these new data, as well as the improvement in abnormal lipid profiles observed in prior completed studies, provide support that nomlabofusp increases FXN in patients with FA and that the strategy of FXN replacement has the potential to result in a clinical benefit.

o Changes observed in skin FXN levels, and clinical outcomes after nomlabofusp administration across diverse participants with FA, including individuals with advanced disease are all directionally consistent and suggest a potential treatment effect.

o Of the 39 participants in the open label study (and of 65 total participants who received at least 1 dose in all nomlabofusp studies), 7 experienced anaphylaxis in the first 6 weeks of dosing and returned to their usual state of health after standard treatment.

Anaphylaxis is more common upon re-exposure to a drug after a gap in dosing;

in the open label study, of the 10 participants who had not had prior exposure to nomlabofusp only 1 experienced anaphylaxis (this reaction was one of the 7 events discussed above).

Excluding these events, long term dosing of nomlabofusp was generally well tolerated including 14 on treatment for at least 6 months and 8 on treatment for over 1 year.

Our nomlabofusp plans and milestones include the following:

• We amended the open label study protocol to include adolescent and adult patients who have not previously participated in a prior nomlabofusp study.

After the anaphylaxis events discussed above, we introduced a new dosing regimen that the FDA accepted.

Participants will initially receive a 5 mg test dose followed by a 25 mg dose one hour later under observation.

Nomlabofusp 25 mg will then be administered once daily through Day 30 and then the dose will be increased to 50 mg once daily.

• We plan to provide an update on regulatory discussions and an open label study update in the first quarter of 2026.

• We plan to submit a BLA seeking accelerated approval in the second quarter of 2026.

• The modified starting dose regimen is also being incorporated into the global phase 3 protocol.

We continue to qualify sites globally and prepare for study initiation and patient enrollment.

• In February 2026, the FDA granted Breakthrough Therapy Designation (“BTD”) ”) to nomlabofusp for the treatment of adults and children with FA.

The designation was based on the FDA’s review of available clinical data from our ongoing OL study evaluating nomlabofusp in adult and pediatric patients with FA.

BTD is intended to expedite the development and regulatory review of a drug intended to treat a serious condition.

A drug is eligible for BTD if preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available treatments in one or more clinically significant endpoints.

• Additionally, in February 2026, as a result of a START pilot program meeting with the FDA, we announced continued alignment with the FDA to consider the use of skin FXN as a novel surrogate endpoint reasonably likely to predict clinical benefit to support a planned Biologics License Application (“BLA”) submission seeking accelerated approval and that the use of FXN as a novel surrogate endpoint to support accelerated approval.

There was also agreement on the type of analyses required to support the exposure response relationships for the nomlabofusp program.

The FDA also stated that the adequacy of the safety database will be a matter of review at the time of BLA submission.

We have scheduled a Type B meeting with the FDA later in the second quarter of 2026.

We plan to provide topline study data from our OL study in the second quarter of 2026 and we plan to initiate a rolling BLA seeking accelerated approval with the submission of nonclinical and clinical modules in June 2026.

We are continuing to focus on the completeness of our chemistry, manufacturing and controls ("CMC") module, and plan to submit the final modules, including the CMC module, in the second half of 2026.

If approved, we are targeting a U.S.

launch in the first half of 2027.

For our global confirmatory Phase 3 study, we are planning sites in the U.S., E.U., U.K., Canada and Australia.

We have obtained feedback from both the FDA and the EMA on the study protocol.

We plan to initiate dosing of the first patient in mid-2026.

Financing Activities, including Recent Material Financings

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(“Zafgen”) and, prior to the 2020 merger with Zafgen, capital contributions from Chondrial Holdings, LLC.

In February 2024, we completed an underwritten public offering in which we issued and sold 19,736,842 shares of our common stock at a public offering price of $8.74 per share.

We received net proceeds of approximately $161.8 million after deducting underwriting discounts, commissions and other offering expenses.

In May 2024, we entered into a Sales Agreement (“2024 ATM Agreement”) with Guggenheim Securities, LLC in connection with the establishment of an “at-the-market” offering program providing for the sale of up to an aggregate of $100 million of shares of our common stock from time to time.

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We received net proceeds of approximately $65.0 million, after deducting underwriting discounts, commissions and other offering expenses.

In February 2026, we completed an underwritten public offering in which we issued and sold 23,000,000 shares of our common stock, including the exercise in full of the underwriters' option to purchase additional shares, at a public offering price of $5.00 per share.

We received net proceeds of approximately $107.6 million, after deducting underwriting discounts, commissions, and other offering expenses.

Critical Accounting Policies and Significant Judgments and Estimates

Our condensed, consolidated financial statements are prepared in accordance with Generally Accepted Accounting Principles (“GAAP”).

The preparation of our consolidated financial statements and related disclosures requires us to make estimates and assumptions that affect the reported amount of assets, liabilities, costs and expenses, and related disclosures.

The preparation of our consolidated financial statements and related disclosures requires us to make estimates and assumptions that affect the reported amount of assets, liabilities, costs and

expenses, and related disclosures.

We believe that the estimates and assumptions involved in the accounting policies described below may have the greatest potential impact on our consolidated financial statements and, therefore, consider these to be our critical accounting policies.

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We expect to increase our investment in research and development in order to advance nomlabofusp through additional clinical trials.

As a result, we expect that our research and development expenses will continue to increase in the foreseeable future as we pursue clinical development of nomlabofusp and/or any other product candidates we develop.

As a result, we expect that our research and development expenses will continue to increase in the foreseeable future as we pursue clinical development of nomlabofusp, including manufacturing activities, and/or any other product candidates we develop.

Stock Compensation Expense

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The Board established specified performance criteria and a corresponding performance period over which such performance criteria must be achieved, the satisfaction of which are conditions to earning the January 2025 PSU Awards, and vesting of the underlying RSUs.

As of September 30, 2025, the underlying performance criteria of the January 2025 PSU Awards were determined to be not probable of achievement for accounting purposes and no stock-based compensation expense was recognized for the three months ended September 30, 2025.

As of March 31, 2026, the underlying performance criteria of the January 2025 PSU Awards were determined to be not probable of achievement for accounting purposes and no stock-based compensation expense was recognized for the three months ended March 31, 2026.

We classify stock-based compensation expense in our consolidated statements of operations and comprehensive loss in the same manner in which the award recipient’s payroll costs are classified or in which the award recipient’s service payments are classified.

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A change in the outcome of one or more of these variables with respect to the development of a product candidate could significantly change the costs, timing, and viability associated with the development of that product candidate.

For example, if the FDA or another regulatory authority were to require us to conduct additional non-clinical or clinical trials beyond those that we currently anticipate will be required for the completion of clinical development of a product candidate, or if we experience significant delays in enrollment in any of our clinical trials,

we could be required to expend significant additional financial resources and time on the completion of clinical development.

General and Administrative Expenses

General and administrative expenses consist primarily of personnel costs, consisting of salaries, related benefits and stock-based compensation, costs related to our executive, finance, commercial, information technology, and costs related to other administrative functions.

General and administrative expenses also include insurance expenses and professional fees for auditing, commercial readiness costs, tax, and legal services, including legal expenses to pursue patent protection for our intellectual property as well as reimbursability and related costs.

We expect that our general and administrative expenses will increase in the foreseeable future as we hire additional employees to implement, improve, and scale our operational, financial, commercial and management systems.

Results of Operations

Comparison of three months ended September 30, 2025 and 2024

The following table summarizes our results of operations for the three months ended September 30, 2025 and 2024:

Three Months Ended September 30,

(in thousands)

Statement of Operations Data:

Operating expenses:

Research and development

General and administrative

Total operating expenses

Loss from operations

Other income (expense), net

Research and Development Expenses

Research and development expenses for the three months ended September 30, 2025 increased $31.0 million compared to the three months ended September 30, 2024.

The increase in research and development expenses was primarily attributable to an increase of $25.8 million in nomlabofusp manufacturing costs, an increase of $1.8 million in professional and consulting fees associated with ongoing clinical studies and increasing worldwide regulatory activities, an increase of $1.5 million in clinical costs primarily associated with the start of an anticipated confirmatory study that are required as part of an accelerated approval of our planned BLA submission expected in the second quarter of 2026, an increase of $1.0 million in personnel costs associated with increased headcount, and an increase of $0.9 million in non-clinical costs related to assay development.

General and Administrative expenses

General and administrative expenses for the three months ended September 30, 2025 increased $0.2 million compared to the three months ended September 30, 2024.

The increase in general and administrative expenses was primarily due to an increase of $0.3 million of personnel costs associated with increased headcount and an increase of $0.2 million in professional fees primarily related to legal services performed.

The increase was largely offset by a decrease of $0.2 million in stock compensation costs related to the full vesting of previously issued stock options granted at higher value stock options.

Other Income (expense), net

Other income (expense), net was $1.8 million income in the three months ended September 30, 2025 compared to $2.8 million income in the three months ended September 30, 2024.

The decrease was primarily driven by lower interest and accretion income due to lower interest yields and lower average investable cash, cash equivalents, and marketable securities balances.

Comparison of nine months ended September 30, 2025 and 2024

The following table summarizes our results of operations for the nine months ended September 30, 2025 and 2024:

Nine Months Ended September 30,

Comparison of three months ended March 31, 2026 and 2025

The following table summarizes our results of operations for the three months ended March 31, 2026 and 2025:

Three Months Ended March 31,

(in thousands)

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Research and Development Expenses

Research and development expenses for the nine months ended September 30, 2025 increased $48.3 million compared to the nine months ended September 30, 2024.

The increase in research and development expenses was primarily attributable to an increase of $32.9 million in nomlabofusp manufacturing costs, an increase of $5.4 million in professional consulting fees associated with ongoing clinical studies and worldwide regulatory activities, an increase of $4.8 million in clinical costs primarily associated with initiating the anticipated confirmatory study required as part of an accelerated approval of our planned BLA submission expected in the second quarter of 2026, an increase of $3.9 million in personnel costs associated with increased quality and regulatory headcount and an increase of $1.7 million in nonclinical costs related to assay development, and partially offset by a decrease of $0.8 million in costs incurred under the TRACK-FA program.

Research and development expenses for the three months ended March 31, 2026 decreased $1.5 million compared to the three months ended March 31, 2025.

The decrease in research and development expenses was primarily driven by a decrease of $3.1 million in nomlabofusp manufacturing related costs and a decrease of $0.5 million in clinical trial costs primarily related to the completion of the Company’s adolescent run-in study in the first half of 2025, partially offset by an increase of $1.6 million in professional consulting fees predominantly related to BLA preparation and inspection readiness, and an increase of $0.2 million in personnel expenses due to increased headcount.

General and Administrative expenses

General and administrative expenses for the nine months ended September 30, 2025 increased $0.6 million compared to the nine months ended September 30, 2024.

The increase in general and administrative expenses was primarily due to an increase of $1.1 million of personnel costs associated with increased headcount (primarily commercial) and an increase of $0.5 million in professional service expenses primarily related to commercial start-up activities, partially offset by a decrease of $0.8 million in stock compensation costs related to the full vesting of previously issued stock options granted at higher value stock options.

General and administrative expenses for the three months ended March 31, 2026 increased $1.5 million compared to the three months ended March 31, 2025.

The increase in general and administrative expenses was primarily due to an increase of $1.1 million in professional fees related to commercial consulting services performed and an increase of $0.3 million of personnel costs associated with increased headcount.

Other Income (expense), net

Other income (expense), net was $5.3 million income in the nine months ended September 30, 2025 compared to $7.8 million income in the nine months ended September 30, 2024.

Other income (expense), net was $1.5 million income in the three months ended March 31, 2026 compared to $1.9 million income in the three months ended March 31, 2025.

The decrease was primarily driven by lower interest and accretion income due to lower interest yields and lower average investable cash, cash equivalents, and marketable securities balances.

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The following table summarizes our sources and uses of cash for each of the periods presented below:

Nine Months Ended September 30,

Three Months Ended March 31,

(in thousands)

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Net cash used in operating activities

During the nine months ended September 30, 2025, operating activities used $74.7 million of cash, resulting from our net loss of $103.2 million, adjusted for noncash expenses of $4.0 million and changes in our operating assets and liabilities resulting in a source of cash of $24.5 million.

During the three months ended March 31, 2026, operating activities used $44.6 million of cash, resulting from our net loss of $29.6 million, adjusted for noncash expenses of $1.9 million and changes in our operating assets and liabilities resulting in a use of cash of $16.9 million.

Our net loss was primarily attributed to research and development activities related to our nomlabofusp program and our general and administrative expenses as described above.

Noncash expenses primarily relate to stock-based compensation expenses.

The change in operating assets and liabilities was primarily due to a increases in prepaid expenses, accounts payable, and accrued expenses.

During the nine months ended September 30, 2024, operating activities used $48.9 million of cash, resulting from our net loss of $51.8 million, adjusted for noncash expenses of $2.4 million and changes in our operating assets and liabilities resulting in a source of cash of $0.4 million.

The change in operating assets and liabilities was primarily due to an decrease in accrued expenses.

During the three months ended March 31, 2025, operating activities used $26.5 million of cash, resulting from our net loss of $29.3 million, adjusted for noncash expenses of $1.1 million and changes in our operating assets and liabilities resulting in a source of cash of $1.6 million.

Our net loss was primarily attributed to research and development activities related to our nomlabofusp program and our general and administrative expenses as described above.

Noncash expenses are primarily stock-based compensation expenses.

The change in operating assets and liabilities was primarily due to an increase in accrued expenses and accounts payable, partially offset by a decrease in prepaid expense.

The change in operating assets and liabilities was primarily due to a decrease in prepaid expenses, partially offset by a decrease in accrued expense and accounts payable.

Net cash provided by (used in) investing activities

During the nine months ended September 30, 2025, investing activities provided $66.3 million.

This source of cash resulted from the maturities of $150.0 million of marketable securities partially offset by purchases of $83.6 million of marketable securities.

During the nine months ended September 30, 2024, investing activities used $104.6 million.

This net use of cash was due to the purchase of $189.2 million of marketable securities, partially offset by $85.0 million of cash provided by maturities of marketable securities.

During the three months ended March 31, 2026, investing activities provided $29.0 million of cash from the maturities of marketable securities.

During the three months ended March 31, 2025, investing activities provided $14.4 million of cash from the maturities of $67.0 million of marketable securities, partially offset by the purchase of $52.5 million in marketable securities.

Net cash provided by financing activities

During the nine months ended September 30, 2025, financing activities provided $65.3 million of cash flows from an offering of common stock.

During the nine months ended September 30, 2024, financing activities provided $161.8 million of cash flows primarily from an offering of common stock.

During the three months ended March 31, 2026, financing activities provided $108.1 million of cash flows from an offering of common stock.

During the three months ended March 31, 2025, there were no financing activities.

Operating Capital Requirements

We have not yet commercialized any products and do not expect to generate revenue from the commercial sale of any products for several years, if at all.

We have incurred net losses of approximately $103.2 million and $51.8 million for the nine months ended September 30, 2025 and 2024, respectively.

As of September 30, 2025, we had an accumulated deficit of $372.3 million and cash, cash equivalents, and marketable securities of $175.4 million, excluding restricted cash of $0.6 million.

We have incurred net losses of approximately $29.6 million and $29.3 million for the three months ended March 31, 2026 and 2025, respectively.

As of March 31, 2026, we had an accumulated deficit of $464.4 million and cash, cash equivalents, and marketable securities of $200.4 million, excluding restricted cash of $0.6 million.

Losses have resulted principally from costs incurred in connection with research and development activities, and general and administrative costs associated with the development of nomlabofusp and our operations.

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• expand our operational, financial, commercial and management systems and personnel, including personnel to support our clinical development and future commercialization efforts and our operations as a public company.

We anticipate that current cash, cash equivalents, and marketable securities as of September 30, 2025 will fund operations into the fourth quarter of 2026.

In July 2025, we sold 21,562,500 shares of our common stock, after deducting underwriters' discounts, commissions and other offering expenses and received net proceeds of approximately $65.0 million, after deducting underwriting discounts, commissions, and other offering expenses.

We received net proceeds of approximately $107.6 million, after deducting underwriting discounts, commissions, and other offering expenses.

We anticipate that our current cash, cash equivalents, and marketable securities as of March 31, 2026 will fund operations into the second quarter of 2027.

If we encounter unexpected delays in our clinical trials or if there are other unanticipated changes to our operating plan from our current assumptions that negatively impact our operations, we may reduce expenditures in order to further extend our existing cash resources.

Until we can generate substantial revenue, if ever, we expect to seek additional funding through a combination of public or private equity offerings, debt/royalty financings, collaborations, strategic alliances, licensing arrangements or other sources.

Until we can generate substantial revenue, if ever, we could seek additional funding through a combination of public or private equity offerings, debt/royalty financings, collaborations, strategic alliances and licensing arrangements or other sources.

The incurrence of indebtedness would result in increased fixed payment obligations and we may be required to agree to certain restrictive covenants, such as limitations on our ability to incur additional debt, minimum cash balances, limitations on our ability to acquire, sell or license intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our business.

Any additional fundraising efforts may divert our management from their day-to-day activities, which may adversely affect our ability to develop and commercialize our product candidates.

There can be no assurance that we will be able to raise sufficient additional capital on acceptable terms, if at all.

There can be no assurance that, if required, we would be able to raise sufficient additional capital on acceptable terms, if at all.

If such additional financing is not available on satisfactory terms, or is not available in sufficient amounts, or we do not have sufficient authorized shares, we may be required to delay, limit, or eliminate the development of business opportunities and our ability to achieve our business objectives, our competitiveness, and our business, financial condition, and results of operations will be materially adversely affected.

We could also be required to seek funds through arrangements with collaborative partners, strategic alliances or otherwise at an earlier stage than otherwise would be desirable and we may be required to relinquish rights to some of our technologies or product candidates or otherwise agree to terms unfavorable to us, any of which may have a material adverse effect on our business, operating results and prospects.

In addition, geopolitical tensions including regional conflicts around the world, adverse macroeconomic events, including those due to inflationary pressures, rising interest rates, banking instability, monetary policy changes, changes in trade policies, including the current U.S.

Government shutdown, tariffs (including tariffs and trade protection measures that have been or may in the future be imposed by the U.S.

or other countries), new laws and regulations, including, but not limited to the recently enacted Public Law 119-21, economic slowdowns or recessions, health epidemics, unforeseen emergencies and other outbreaks of communicable diseases could disrupt our operations, the operations of third parties on which we rely, or the operations of regulatory agencies we interact with in the development of nomlabofusp and any other product candidates that we may develop which may reduce our ability to access capital, which could negatively affect our liquidity and ability to continue as a going concern.

If we are unable to obtain sufficient funding when needed and/or on acceptable terms, we may be required to significantly curtail, delay or discontinue one or more of our research and development programs, the manufacture of clinical and commercial supplies, product portfolio expansion and/or commercialization efforts, which could adversely affect our business prospects, or we may be unable to continue operations.

Certain restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell, or license intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our

Any additional fundraising efforts may divert our management from their day-to-day activities, which may adversely affect our capability to develop and commercialize our product candidates.

Off-Balance Sheet Arrangements

During the periods presented, and through the date of this filing, we do not have any off-balance sheet arrangements, as defined under applicable SEC rules, such as relationships with unconsolidated entities or financial partnerships, which are often referred to as structured finance or special purpose entities established for the purpose of facilitating financing transactions that are not required to be reflected on our balance sheets.

In addition, geopolitical tensions, volatility of capital markets, and other adverse macroeconomic events, including those due to inflationary pressures, rising interest rates, bank instability and the ability of the U.S.

government to manage federal debt limits, as well as the potential impact of health crises on the global financial markets may reduce our ability to access capital, which could negatively affect our liquidity and ability to continue as a going concern.

If we are required to obtain sufficient funding, but are unable to do so, when needed and/or on acceptable terms, we may be required to significantly curtail, delay or discontinue one or more of our research and development programs, the manufacture of clinical and commercial supplies, product portfolio expansion and/or pre commercialization efforts, which could adversely affect our business prospects, or we may be unable to continue operations.

Certain restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell or license intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our business.

Any additional fundraising efforts may divert our management from their day-to-day activities, which may adversely affect our ability to develop and commercialize our product candidates.

During the periods presented we did not have, and we currently do not have, any off-balance sheet arrangements, as defined under applicable SEC rules, such as relationships with unconsolidated entities or financial partnerships, which are often referred to as structured finance or special purpose entities, established for the purpose of facilitating financing transactions that are not required to be reflected on our balance sheets.

Recently Issued 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.

What changed 10-Q

Item 2. Management’s Discussion and Analysis