What changed in this filing

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Item 2. Management’s Discussion and Analysis

2025-08-14 compared with 2025-05-12 · 112 added, 53 removed, 166 unchanged (50% of the section changed)

16 unchanged sentences

• Guillain-Barré Syndrome, or GBS:

We are advancing our lead candidate, tanruprubart (formerly ANX005), an investigational, full-length monoclonal antibody, or mAb, formulated for intravenous administration as the potential first-targeted treatment for patients with GBS.

Tanruprubart has consistently demonstrated rapid and sustained functional improvements in multiple placebo-controlled trials.

GBS is a rare antibody-mediated autoimmune disease that is the most common cause of acute neuromuscular paralysis, with no therapies in the United States approved by the U.S.

Food and Drug Administration, or FDA.

The rare and acute nature of GBS has shaped our clinical development program conducted primarily in Southeast Asia to generate a comprehensive data package that includes successful placebo-controlled proof-of-concept and Phase 3 data, Real-World Evidence indirect comparison data of tanruprubart’s treatment effect versus current standard of care, and drug-drug interaction safety data with tanruprubart with current standard of care.

We are also preparing to initiate in the second quarter of 2025 the open-label tanruprubart FORWARD study, measuring pharmacokinetics, pharmacodynamics, early efficacy in week 1, and safety in up to 30 subjects in the United States, Canada, and Europe, which is designed to broaden Western patient, physician and healthcare community experience.

We anticipate this comprehensive data package to support our Biologics License Application, or BLA, and are targeting a meeting with the Center for Drug Evaluation and Research of the FDA in the second quarter of 2025, ahead of our planned BLA submission.

We are advancing tanruprubart (formerly ANX005), an investigational targeted immunotherapy delivered in a single infusion to rapidly halt aggressive neuroinflammation and damage in GBS, an acute, rare, neuromuscular emergency that affects ~150,000 people worldwide each year.

There are currently no therapies approved by the U.S.

Food and Drug Administration, or FDA, for GBS and no substantial evidence of effectiveness from the current standard of care.

In the placebo-controlled Phase 3 trial, approximately 90% of GBS patients treated with tanruprubart improved by week 1 and more than twice as many treated patients achieved a normal state of health at week 26.

Tanruprubart has consistently demonstrated rapid and sustained functional improvements across a comprehensive data package that includes successful placebo-controlled proof-of-concept, or POC, and Phase 3 data, Real-World Evidence, or RWE, indirect comparison data of tanruprubart’s treatment effect versus current standard of care, and drug-drug interaction safety data with tanruprubart with current standard of care.

Completed placebo-controlled studies were conducted in Southeast Asia given GBS’ sudden and debilitating disease course and lack of approved comparator in the U.S.

The open-label FORWARD study in the U.S.

and Europe is ongoing and currently designed to broaden Western experience with tanruprubart by measuring pharmacokinetics, pharmacodynamics, early efficacy in week 1, and safety in up to 30 subjects including pediatric patients.

We continue to engage with applicable EU and U.S.

regulators to advance tanruprubart towards registration worldwide.

Our Marketing Authorization Application, or MAA, submission for registration in Europe is expected in the first quarter of 2026.

In parallel, we are in ongoing dialogue with the FDA to gain clarity on the generalizability package to support a BLA submission.

Tanruprubart has been granted Fast Track and orphan drug designation for the treatment of GBS from the FDA.

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• Geographic Atrophy, or GA:

We are advancing ANX007, an antigen-binding fragment, or Fab, formulated for intravitreal administration, as the first potential program with a global registration path to approval in Europe and the United States for the treatment of dry AMD with GA.

Dry AMD with GA is a leading cause of vision loss in the elderly, that affects more than an estimated eight million people globally, and there are no approved therapies targeting the preservation of vision in

this disease.

ANX007 is designed to block C1q locally in the eye, to provide more complete protection against excess classical complement activity and the loss of photoreceptor neurons.

It is the only investigational therapy in GA to show significant vision preservation on assessments of best corrected visual acuity, or BCVA, and low luminance visual acuity, or LLVA, demonstrating significant protection from vision loss in both normal and low light conditions, as well as significant preservation of central retinal photoreceptors necessary for visual acuity as demonstrated in the Phase 2 ARCHER trial.

In 2024, we initiated our Phase 3 ARCHER II trial, a global, sham-controlled, double-masked trial expected to enroll ~630 patients who have dry AMD with GA.

The primary endpoint of ARCHER II is prevention of ≥15-letter loss of BCVA, and a secondary objective structural measure is prevention of ellipsoid zone (EZ) loss.

The single-study program will be analyzed as two sub-studies for the U.S.

in accordance with the FDA’s two-trial recommendation.

Accordingly, we no longer plan to conduct a second injection-controlled head-to-head Phase 3 trial.

We expect to complete enrollment of ARCHER II in the third quarter of 2025 and report topline data in the second half of 2026.

ANX007 is the first and only therapeutic candidate for the treatment of GA to receive Priority Medicine, or PRIME, designation by the EMA, which provides early and proactive support to developers of promising medicines that may offer a major therapeutic advantage over existing treatments or benefit to patients without treatment options.

We are advancing vonaprument (formerly ANX007), an investigational neuroprotective inhibitor of C1q and the classical complement cascade delivered intravitreally for dry AMD with GA, a leading cause of blindness affecting more than eight million people worldwide.

There are no approved therapies for GA targeting the preservation of vision.

Vonaprument is the only investigational therapy in GA to show significant vision preservation on assessments of best corrected visual acuity, or BCVA, and low luminance visual acuity, or LLVA, demonstrating significant protection from vision loss in both normal and low light conditions, as well as significant preservation of central retinal photoreceptors necessary for visual acuity as demonstrated in the Phase 2 ARCHER trial.

In July 2025, we completed enrollment of 659 patients in ARCHER II, a global, sham-controlled, double-masked Phase 3 trial.

The primary endpoint of ARCHER II is prevention of ≥15-letter loss of BCVA, and a secondary objective structural measure is prevention of ellipsoid zone, or EZ, loss.

We have established a global registration path with the FDA and EMA which supports the potential of vonaprument to be the first treatment approved in both Europe and the U.S.

for the protection of vision in patients who have dry AMD with GA, assuming positive Phase 3 results.

We plan to report topline data in the second half of 2026.

Vonaprument is the first and only therapeutic candidate for the treatment of GA to receive Priority Medicine, or PRIME, designation by the EMA, which provides early and proactive support to developers of promising medicines that may offer a major therapeutic advantage over existing treatments or benefit to patients without treatment options.

Vonaprument was also selected by EMA for Product Development Coordinator (PDC) Pilot launched in July 2025 to help PRIME designation holders efficiently navigate regulatory interactions including expedited scientific advice, MAA submission readiness activities, and ad-hoc queries throughout the development program.

• ANX1502 for Autoimmune Indications:

We are advancing ANX1502, a novel oral small molecule inhibitor of classical complement which we believe is first-in-kind.

We are advancing ANX1502, a novel oral small molecule inhibiting the activated form of C1s, an enzyme carried by C1q to initiate the classical cascade, which we believe is first-in-kind and has the potential to offer the advantages of selective upstream classical complement inhibition with the convenience and flexibility of oral administration.

In a Phase 1 single-ascending dose, or SAD, and multiple-ascending dose, or MAD, clinical trial in healthy volunteers designed to evaluate the safety, tolerability, pharmacokinetics, or PK, and pharmacodynamics, or PD, ANX1502 was generally well tolerated across cohorts with no serious adverse events, achieved target levels of active drug and showed supportive impact on a PD biomarker of complement activity.

We are evaluating a tablet formulation of ANX1502 in an ongoing proof-of-concept study in patients with cold agglutinin disease, or CAD, to assess PK, PD and clinical efficacy endpoints (e.g., hemolysis as measured by reduction of elevated bilirubin) and data are expected in mid-2025.

Following the successful completion of the proof-of-concept study, we intend to evaluate ANX1502 in a host of diseases, including serious complement-mediated autoimmune diseases, with the aim of providing enhanced efficacy and offering convenient dosing administration for long-term treatment of chronic conditions.

We are evaluating a tablet formulation of ANX1502 in an ongoing POC study in patients with cold agglutinin disease, or CAD.

Exposure exceeded target concentrations in fasted patients treated to date in the POC study, and evaluation of PK/PD in relation to food intake, and reduction in complement and bilirubin markers as a measure of hemolysis, are ongoing.

We anticipate that PK/PD learnings from CAD patients will inform application of ANX1502 in a broad array of other autoimmune indications, and we plan to provide an update by the end of 2025.

We were incorporated in March 2011 and commenced operations later that year.

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We have incurred net losses each year since our inception.

Our net losses were $54.4 million and $25.2 million for the three months ended March 31, 2025 and 2024, respectively.

As of March 31, 2025, we had an accumulated deficit of $765.1 million and cash and cash equivalents and short-term investments of $263.7 million.

Our net losses were $49.2 million and $29.6 million for the three months ended June 30, 2025 and 2024, respectively, and $103.5 million and $54.8 million for the six months ended June 30, 2025 and 2024, respectively.

As of June 30, 2025, we had an accumulated deficit of $814.2 million and cash and cash equivalents and short-term investments of $227.0 million.

Components of Operating Results

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Results of Operations

Comparison of the Three Months Ended March 31, 2025 and 2024

Comparison of the Three Months Ended June 30, 2025 and 2024

The following tables summarize our results of operations for the periods presented:

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Total research and development expenses

__________________

*Greater than 200% change for the periods presented.

Research and development expenses increased by $27.2 million, or 130%, for the three months ended March 31, 2025 compared to the same period in 2024.

The change was primarily attributable to a $14.1 million increase in contract manufacturing expenses related to the timing of activities for tanruprubart (ANX005) supporting the preparation of the BLA submission, as well as the initiation of the manufacturing technology transfer of ANX007 to a commercial-ready facility.

Research and development expenses increased by $19.1 million, or 76%, for the three months ended June 30, 2025 compared to the same period in 2024.

The change was attributable to $8.6 million in additional direct clinical and nonclinical outside services costs associated with the Phase 3 ARCHER II clinical trial and the initiation of the tanruprubart FORWARD study in GBS.

Contract manufacturing expenses increased $3.9 million due to activities for tanruprubart (ANX005) supporting the preparation of the BLA submission, as well as the manufacturing technology transfer of vonaprument (ANX007) to a commercial-ready facility.

In addition, compensation and personnel-related expenses increased by $3.5 million, driven by higher headcount.

Consulting and professional services expenses increased by $3.1 million, primarily to support the preparation of our BLA submission for tanruprubart in GBS and to provide ongoing support for the ARCHER II clinical trial.

We also incurred $3.9 million in additional direct clinical and nonclinical outside services costs associated with continued advancement of the ARCHER II clinical trial.

General and Administrative Expenses

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Total general and administrative expenses

General and administrative expenses increased by $1.6 million, or 21%, for the three months ended March 31, 2025 compared to the same period in 2024.

The increase was primarily due to an increase in compensation and personnel-related expenses of $0.8 million due to an increase in headcount, and an increase of $0.6 million in consulting and professional services and recruitment costs.

General and administrative expenses decreased by $1.0 million, or 12%, for the three months ended June 30, 2025 compared to the same period in 2024.

The decrease was primarily driven by a $0.7 million reduction in consulting and professional services costs.

Compensation and personnel-related expenses also declined by $0.5 million, mainly due to the full amortization of previously granted high fair value stock options, partially offset by higher headcount.

Other expenses increased by $0.3 million due to cloud-based infrastructure costs.

Interest and other income, net

Interest and other income, net, decreased by $0.3 million, or 10%, for the three months ended March 31, 2025 compared to the same period in 2024.

Interest and other income, net, decreased by $1.4 million, or 35%, for the three months ended June 30, 2025 compared to the same period in 2024.

The decrease was primarily due to lower average cash and investment balances.

Comparison of the Six Months Ended June 30, 2025 and 2024

The following tables summarize our results of operations for the periods presented:

Six Months Ended

(in thousands)

Operating expenses:

Research and development

General and administrative

Total operating expenses

Loss from operations

Interest and other income, net

Research and Development Expenses

Six Months Ended

(in thousands)

Direct costs:

Clinical and nonclinical outside services

Contract manufacturing

Consulting and professional services

Laboratory supplies and materials

Indirect costs:

Compensation and personnel-related

(including stock-based compensation)

Facilities and depreciation

Total research and development expenses

__________________

*Greater than 200% change for the periods presented.

Research and development expenses increased by $46.4 million, or 101%, for the six months ended June 30, 2025 compared to the same period in 2024.

The change was attributable to a $18.0 million increase in contract manufacturing expenses related to the activities for tanruprubart (ANX005) supporting the preparation of the BLA submission, as well as the manufacturing technology transfer of vonaprument (ANX007) to a commercial-ready facility.

There was also a $12.5 million increase in direct clinical and nonclinical outside services costs associated with the Phase 3 ARCHER II clinical trial and the initiation of the tanruprubart FORWARD study in GBS.

Compensation and personnel-related expenses increased by $8.1 million, driven by higher headcount.

In addition, consulting and professional services expenses increased by $7.3 million, primarily to support the preparation of our BLA submission for tanruprubart in GBS and to provide ongoing support for the ARCHER II clinical trial.

General and Administrative Expenses

Six Months Ended

(in thousands)

Compensation and personnel-related

(including stock-based compensation)

Consulting and professional services

Facilities and depreciation

Total general and administrative expenses

General and administrative expenses increased by $0.6 million, or 4%, for the six months ended June 30, 2025 compared to the same period in 2024.

The change was primarily driven by a $0.3 million increase in compensation and personnel-related expenses, mainly due a higher headcount, partially offset by the full amortization of previously granted high fair value stock options.

Other expenses increased by $0.4 million due to cloud-based infrastructure costs.

Interest and other income, net

Interest and other income, net, decreased by $1.7 million, or 24%, for the six months ended June 30, 2025 compared to the same period in 2024.

The decrease was primarily due to lower average cash and investment balances.

Liquidity and Capital Resources

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We have funded our operations primarily through the sale of equity securities.

As of March 31, 2025, we had available cash and cash equivalents and short-term investments of $263.7 million and an accumulated deficit of $765.1 million.

As of June 30, 2025, we had available cash and cash equivalents and short-term investments of $227.0 million and an accumulated deficit of $814.2 million.

Historical Cash Flows

Three Months Ended

Six Months Ended

(in thousands)

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Cash Flows from Operating Activities

Cash used in operating activities for the three months ended March 31 , 2025 was $50.1 million, which consisted of a net loss of $54.4 million, partially offset by $4.1 million in non-cash charges and a net change of $0.2 million in our operating assets and liabilities.

Cash used in operating activities for the six months ended June 30, 2025 was $88.1 million, which consisted of a net loss of $103.5 million, partially offset by $8.0 million in non-cash charges and a net change of $7.4 million in our operating assets and liabilities.

The non-cash charges consisted of stock-based compensation of $9.3 million, depreciation and amortization of $1.1 million and a reduction in the carrying amount of right-of-use assets of $0.7 million, partially offset by accretion of discount on available-for-sale securities of $3.1 million.

Cash used in operating activities for the three months ended March 31, 2024 was $28.3 million, which consisted of a net loss of $25.2 million and a net change of $7.5 million in our operating assets and liabilities, partially offset by $4.4 million in non-cash charges.

Cash used in operating activities for the six months ended June 30, 2024 was $50.0 million, which consisted of a net loss of $54.8 million and a net change of $3.6 million in our operating assets and liabilities, partially offset by $8.4 million in non-cash charges.

The non-cash charges consisted of stock-based compensation of $9.6 million, depreciation and amortization of $1.1 million and a reduction in the carrying amount of right-of-use assets of $0.6 million, partially offset by accretion of discount on available-for-sale securities of $2.9 million.

Cash Flows from Investing Activities

Cash provided by investing activities for the three months ended March 31, 2025 was $97.6 million, which consisted of $159.5 million of proceeds from maturities of available-for-sale securities, partially offset by $61.8 million of purchases of available-for-sale securities and $0.1 million of purchases of property and equipment.

Cash used in investing activities for the three months ended March 31, 2024 was $77.3 million, which consisted of $100.3 million of purchases of available-for-sale securities, partially offset by $23.0 million of proceeds from maturities of available-for-sale securities.

Cash provided by investing activities for the six months ended June 30, 2025 was $170.7 million, which consisted of $291.3 million of proceeds from maturities of available-for-sale securities, partially offset by $120.5 million of purchases of available-for-sale securities and $0.1 million of purchases of property and equipment.

Cash used in investing activities for the six months ended June 30, 2024 was $173.9 million, which consisted of $266.2 million of purchases of available-for-sale securities, partially offset by $92.3 million of proceeds from maturities of available-for-sale securities.

Cash Flows from Financing Activities

Cash provided by financing activities for the three months ended March 31, 2025 was $0.1 million, which consisted of $0.1 million of proceeds from the exercise of common stock options.

Cash provided by financing activities for the three months ended March 31, 2024 was $32.4 million, which consisted of $32.2 million of net proceeds from the issuance of common stock under our 2021 ATM program and $0.2 million of proceeds from the exercise of common stock options.

Cash provided by financing activities for the six months ended June 30, 2025 was $0.2 million, which consisted of proceeds from the exercise of common stock options and employee stock purchase plans.

Cash provided by financing activities for the six months ended June 30, 2024 was $156.1 million, which consisted of $116.8 million of net proceeds from the issuance of common stock and pre-funded warrants under our 2024 financing, $38.4 million of net proceeds from the issuance of common stock under our 2021 ATM program and $0.6 million of proceeds from the exercise of common stock options and employee stock purchase plans.

Funding Requirements

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• the timing, receipt and amount of sales of any future approved products.

Based upon our current operating plan, we believe that our existing cash and cash equivalents and short-term investments will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2026.

We will require substantial additional financing to achieve our goals, and a failure to obtain this necessary capital when needed on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our product development programs, commercialization efforts or other operations.

We will require substantial

additional financing to achieve our goals, and a failure to obtain this necessary capital when needed on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our product development programs, commercialization efforts or other operations.

We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect.

Until such time, if ever, as we can generate substantial product revenue, we will be required to seek additional funding in the future and currently intend to do so through public or private equity offerings or debt financings, credit or loan facilities, collaborations or a combination of one or more of these funding sources.

We will be required to seek additional funding in the future until such time, if ever, as we can generate substantial product revenue, and currently intend to do so through public or private equity offerings or debt financings, credit or loan facilities, collaborations or a combination of one or more of these funding sources.

We may also need to seek additional funds sooner than planned as result of changes in our development plans and regulatory requirements to support registration of our product candidates.

Additional funds may not be available to us on acceptable terms or at all.

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The pre-funded warrants remain exercisable until exercised in full.

The common warrants have an exercise price of $5.806875 per share and expire on June 30, 2025.

Both the pre-funded and common warrants are immediately exercisable, subject to beneficial ownership limitations.

The common warrants had an exercise price of $5.806875 per share and expired on June 30, 2025.

Both the pre-funded and common warrants were immediately exercisable, subject to beneficial ownership limitations.

We issued an aggregate of 2,582,557 shares of common stock upon the cashless exercise of the pre-funded warrants in March 2023.

We issued an aggregate of 19,901 and 2,739,096 shares of common stock upon the cashless exercise of the common warrants and pre-funded warrants in June 2024 and November 2024, respectively.

We issued an aggregate of 19,901 shares of common stock upon the cashless exercise of the 322,893 common warrants in June 2024.

In June 2025, we and holders of common warrants to purchase 6,877,622 shares of our common stock entered into amendments to the common warrants held by such holders.

The amendments extended the term of the common warrants by one year until June 30, 2026, and removed the cashless exercise option.

If all such warrants are exercised in full for cash (without regard to any applicable ownership limitations), we would receive aggregate gross proceeds of approximately $39.9 million.

The remaining common warrants to purchase 1,226,993 shares of our common stock not subject to these amendments expired unexercised on June 30, 2025.

We concluded that the modified common warrants retain equity classification.

The excess of fair value of the modified warrants over the fair value of the warrants immediately before the modification of $1.9 million was recognized as a deemed dividend.

This modification included a related party transaction involving 613,497 common warrants held by a member of our board of directors, which accounted for $0.2 million of the deemed dividend recognized.

As we had an accumulated deficit as of June 30, 2025, the deemed dividend was recognized in additional paid-in capital with zero net impact on stockholders' equity.

Pre-Funded and Common Warrants

The following summarizes warrant activity during the three months ended March 31, 2025 and 2024:

The following summarizes warrant activity during the six months ended June 30, 2025 and 2024:

Number of Common Warrants

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Balances as of December 31, 2024

Balances as of March 31, 2025

Balances as of June 30, 2025

Number of Common Warrants

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Balances as of December 31, 2023

Balances as of March 31, 2024

Balances as of June 30, 2024

2024 At-the-Market (ATM) Program

1 unchanged sentence

TD Cowen is entitled to compensation up to 3% of the aggregate gross proceeds for the common stock sold through the 2024 ATM program.

As of March 31, 2025, approximately $95.4 million remained available for the offer and sale of shares of common stock under the 2024 ATM program.

No sales were made under the 2024 ATM program during the six months ended June 30, 2024 and 2025.

As of June 30, 2025, approximately $95.4 million remained available for the offer and sale of shares of common stock under the 2024 ATM program.

2021 ATM Program

1 unchanged sentence

TD Cowen is entitled to compensation up to 3% of the aggregate gross proceeds for the common stock sold through the 2021 ATM program.

During the three months ended March 31, 2024, we sold 6,639,348 shares of common stock under the 2021 ATM program for net proceeds of approximately $32.2 million, after deducting commissions paid to TD Cowen and other financing costs.

During the six months ended June 30, 2024, we sold 7,576,067 shares of common stock under the 2021 ATM program for net proceeds of approximately $38.4 million, after deducting commissions paid to TD Cowen and other financing costs.

The Form S-3 registration statement, which registered the 2021 ATM Program, expired on August 15, 2024.

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Actual results may differ from these estimates under different assumptions or conditions and any such differences may be material.

During the quarter ended March 31, 2025, there were no material changes to our critical accounting policies or in the methodology used for estimates from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report on Form 10-K for the year ended December 31, 2024.

During the quarter ended June 30, 2025, there were no material changes to our critical accounting policies or in the methodology used for estimates from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report on Form 10-K for the year ended December 31, 2024.

Recent Accounting Pronouncements Not Yet Adopted

3 unchanged sentences

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