Amylyx Pharmaceuticals, Inc.

Previous wording

We are a clinical-stage pharmaceutical company with a mission to develop novel therapies for communities with high unmet medical needs. We have preclinical and clinical development programs underway in neurodegenerative diseases and endocrine conditions. We are advancing a pipeline in which we have matched investigational therapies with diseases where we believe they can make the greatest impact, based on well-defined mechanistic rationale, clear clinical outcomes and biomarkers, and rigorous preclinical data, agnostic of modality. We are currently developing three investigational therapies for potential impact across several diseases: avexitide in PBH, AMX0035 in Wolfram syndrome and PSP, and AMX0114 in ALS.

Current wording

We are a clinical-stage pharmaceutical company with a mission to develop novel therapies for communities with high unmet medical needs. We have preclinical and clinical development programs underway in endocrine conditions and neurodegenerative diseases. We are advancing a pipeline in which we have matched investigational therapies with diseases where we believe they can make the greatest impact, based on well-defined mechanistic rationale, clear clinical outcomes and biomarkers, and rigorous preclinical data, agnostic of modality. We are currently developing three investigational therapies for potential impact across several diseases: avexitide in PBH, AMX0035 in Wolfram syndrome and AMX0114 in ALS. In addition, we are collaborating with Gubra A/S, an industry leader in peptide-based drug discovery, to develop a novel long-acting GLP-1 receptor antagonist. We expect to make a decision on a potential development candidate in the next few months, and pending a candidate nomination, expect to initiate Investigational New Drug (IND)-enabling studies.

Previous wording

Our lead investigational asset is avexitide, a first-in-class glucagon-like peptide-1, or GLP-1 receptor antagonist. Avexitide has been evaluated as a treatment for PBH and congenital hyperinsulinism, or HI, two indications characterized by hyperinsulinemic hypoglycemia. The U.S. Food and Drug Administration, or the FDA, has granted avexitide Breakthrough Therapy Designation for both PBH and congenital HI, Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI).

Current wording

Our lead investigational asset is avexitide, a first-in-class inhibitor of glucagon-like peptide-1, or GLP-1, receptor activity. Avexitide has been evaluated as a treatment for PBH and congenital hyperinsulinism, or HI, two indications characterized by hyperinsulinemic hypoglycemia. The U.S. Food and Drug Administration, or the FDA, has granted avexitide Breakthrough Therapy Designation for both PBH and congenital HI, Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI).

Previous wording

PBH is a condition that affects about 160,000 people in the U.S., or approximately 8% of those in the U.S. who have undergone the two most common types of bariatric surgery: sleeve gastrectomy and Roux-en-Y gastric bypass, or RYGB surgery. Currently, there are no FDA-approved therapies for PBH. PBH is thought to be caused by an excessive GLP-1 response, leading to hypoglycemia and impaired quality of life. Clinical manifestations can include impaired cognition, loss of consciousness, and seizures. Avexitide is designed to bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the effects of excessive GLP-1 in PBH, mitigating hypoglycemia by decreasing insulin secretion and stabilizing blood glucose levels.

Current wording

PBH is a condition that is estimated to affect about 160,000 people in the U.S., or approximately 8% of people in the U.S. who have undergone the two most common types of bariatric surgery: sleeve gastrectomy and Roux-en-Y gastric bypass (or RYGB surgery). Currently, there are no FDA-approved therapies for PBH. PBH is thought to be caused by an excessive GLP-1 response, leading to hypoglycemia and impaired quality of life. Clinical manifestations can include impaired cognition, loss of consciousness, and seizures. Avexitide is designed to bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the effects of excessive GLP-1 in PBH, mitigating hypoglycemia by decreasing insulin secretion and stabilizing blood glucose levels.

Previous wording

In April 2025, we dosed the first participant for the pivotal Phase 3 LUCIDITY clinical trial for avexitide in PBH following RYGB surgery. LUCIDITY (NCT06747468) is a multicenter, randomized, double-blind, placebo-controlled, 16-week clinical trial evaluating the efficacy and safety of avexitide in participants with PBH following RYGB surgery. The Phase 3 trial is being conducted at approximately 20 sites in the U.S. Approximately 75 participants will be randomized 3:2 to receive either 90 mg of avexitide subcutaneously once daily or placebo. Participants who complete the 16-week double-blind period of the ongoing study will be eligible to enter an open-label extension, or OLE, period with a duration of 32 weeks. The primary efficacy objective of LUCIDITY is to evaluate the FDA-agreed primary endpoint of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Safety and tolerability will also be evaluated. Completion of recruitment is expected in 2025, with topline data anticipated in the first half of 2026 and, if approved, commercial launch is anticipated in 2027.

Current wording

In April 2025, we dosed the first participant for the pivotal Phase 3 LUCIDITY clinical trial for avexitide in PBH following RYGB surgery. LUCIDITY (NCT06747468) is a multicenter, randomized, double-blind, placebo-controlled, 16-week clinical trial evaluating the efficacy and safety of avexitide in participants with PBH following RYGB surgery. The Phase 3 trial is being conducted at approximately 20 sites in the U.S. Approximately 75 participants will be randomized 3:2 to receive either 90 mg of avexitide subcutaneously once daily or placebo. Participants who complete the 16-week double-blind period of the ongoing study will be eligible to enter an open-label extension, or OLE, period with a duration of 32 weeks. The primary efficacy objective of LUCIDITY is to evaluate the FDA-agreed primary endpoint of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Safety and tolerability will also be evaluated. We expect to complete recruitment in the first quarter of 2026, with topline data expected in the third quarter of 2026. We continue to expect to commercially launch avexitide in 2027, if approved.

Previous wording

RYGB surgery and other upper-gastrointestinal surgeries. Participants in the Phase 2b trial receiving 90 mg once daily of avexitide, the dose Amylyx is evaluating in LUCIDITY, saw a statistically significant 53% reduction in Level 2 hypoglycemic events (p=0.004) and a statistically significant 66% reduction in Level 3 hypoglycemic events (p=0.0003). There were no reported serious AEs, and AEs were mostly mild to moderate and resolved without medical treatment. The most common AEs included diarrhea, headache, bloating, and injection site reaction/bruising. No participant withdrew due to AEs. In the Phase 2b trial, 90 mg once daily of avexitide has also demonstrated a favorable pharmacokinetic, or PK, profile maintaining exposure in the therapeutic range through 24 hours, supporting once daily dosing.

Current wording

In the Phase 2b, 28-day, open-label, investigator-initiated, crossover trial (N=16), 90 mg once daily and 45 mg twice daily of avexitide met its primary endpoint and significantly reduced rates of hypoglycemic events in participants following RYGB surgery and other upper-gastrointestinal surgeries. Participants in the Phase 2b trial receiving 90 mg once daily of avexitide, the dose Amylyx is evaluating in LUCIDITY, saw a statistically significant 53% reduction in Level 2 hypoglycemic events (p=0.004) and a statistically significant 66% reduction in Level 3 hypoglycemic events (p=0.0003). There were no reported serious AEs, and AEs were mostly mild to moderate and resolved without medical treatment. The most common AEs included diarrhea, headache, bloating, and injection site reaction/bruising. No participant withdrew due to AEs. In the Phase 2b trial, 90 mg once daily of avexitide has also demonstrated a favorable pharmacokinetic, or PK, profile maintaining exposure in the therapeutic range through 24 hours, supporting once daily dosing.

Previous wording

In December 2024, we announced a collaboration with Gubra A/S for the development of a potential novel long-acting GLP-1 receptor antagonist. As part of this collaboration, we anticipate identifying a lead development candidate to enter Investigational New Drug, or IND-enabling studies.

Current wording

In December 2024, we announced a collaboration with Gubra A/S for the development of a potential novel long-acting inhibitor of GLP-1 receptor activity. Our research collaboration continues to show proof of concept, with new molecules demonstrating strong potency values bothin vitro andin vivo, along with extended half-lives. We expect to make a decision on a potential development candidate in the next few months, and pending a candidate nomination, expect to initiate IND-enabling studies.

Previous wording

In addition to avexitide, we are investigating AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol in Wolfram syndrome and PSP, and AMX0114, an antisense oligonucleotide targeting calpain-2 in ALS.

Current wording

In addition to avexitide, we are investigating AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol in Wolfram syndrome and AMX0114, an antisense oligonucleotide targeting calpain-2 in ALS.

Previous wording

AMX0035 is designed to slow or mitigate neurodegeneration by targeting endoplasmic reticulum, or ER stress, and mitochondrial dysfunction, two connected central pathways that lead to cell death and neurodegeneration. We are investigating AMX0035 in neurodegenerative diseases and endocrine conditions where ER stress and mitochondrial dysfunction are implicated, including Wolfram syndrome and PSP.

Current wording

AMX0035 is designed to slow or mitigate neurodegeneration by targeting endoplasmic reticulum, or ER stress, and mitochondrial dysfunction, two connected central pathways that lead to cell death and neurodegeneration. We are investigating AMX0035 in neurodegenerative diseases and endocrine conditions where ER stress and mitochondrial dysfunction are implicated, including Wolfram syndrome.

Previous wording

Treatment with AMX0035 at Week 24 and at Week 48 also showed sustained improvements or stabilization in glycemic control, as measured by hemoglobin A1c (HbA1c) and time in target glucose range assessed by continuous glucose monitoring, as well as visual acuity. All participants with available measurements met the responder criteria, defined as either improvement or no change, on both the Patient Global Impression of Change and Clinician Global Impression of Change at Weeks 24 and 48, indicating stability or improvement in their Wolfram syndrome-related symptoms. Results from qualitative on-study interviews further supported the potential positive impact of AMX0035 on symptom burden. Safety data were consistent with safety data from prior studies of AMX0035. All AEs were mild or moderate, and there were no serious AEs related to AMX0035 treatment. The analysis performed at Week 24 included 11 participants in the Per Protocol population with genetically confirmed Wolfram syndrome (one participant was excluded from the Per Protocol population due to not meeting the study inclusion criteria of genetically confirmed Wolfram syndrome) and 12 participants in the Intent-to-Treat population. Long-term data at Week 48 included 10 participants in the Per Protocol population with genetically confirmed Wolfram syndrome and 11 participants in the Intent-to-Treat population. One participant discontinued between Week 24 and Week 48 for reasons unrelated to safety. These data and discussions with the FDA will inform the design of a Phase 3 trial of AMX0035 in Wolfram syndrome.

Current wording

Treatment with AMX0035 at Week 24 and at Week 48 also showed sustained improvements or stabilization in glycemic control, as measured by hemoglobin A1c (HbA1c) and time in target glucose range assessed by continuous glucose monitoring, as well as visual acuity. All participants with available measurements met the responder criteria, defined as either improvement or no change, on both the Patient Global Impression of Change and Clinician Global Impression of Change at Weeks 24 and 48, indicating stability or improvement in their Wolfram syndrome-related symptoms. Results from qualitative on-study interviews further supported the potential positive impact of AMX0035 on symptom burden. Safety data were consistent with safety data from prior studies of AMX0035. All AEs were mild or moderate, and there were no serious AEs related to AMX0035 treatment. The analysis performed at Week 24 included 11 participants in the Per Protocol population with genetically confirmed Wolfram syndrome (one participant was excluded from the Per Protocol population due to not meeting the study inclusion criteria of genetically confirmed Wolfram syndrome) and 12 participants in the Intent-to-Treat population. Long-term data at Week 48 included 10 participants in the Per Protocol population with genetically confirmed Wolfram syndrome and 11 participants in the Intent-to-Treat population. One participant discontinued between Week 24 and Week 48 for reasons unrelated to safety.

Previous wording

In April 2025, we dosed the first participant in Canada for the Phase 1 LUMINA clinical trial (NCT06665165), a multicenter, randomized, double-blind, placebo-controlled, multiple ascending dose trial designed to evaluate the safety and biological activity of AMX0114 in people living with ALS. LUMINA will also assess ALS biomarkers, including change from baseline in NfL levels. In addition to the activated sites in Canada, we are continuing to open U.S. sites for screening, enrollment, and dosing. Approximately 48 participants will be randomized 3:1 to receive AMX0114 or placebo by intrathecal administration once every four weeks, for up to four doses. Early cohort data from LUMINA are expected in 2025.

Current wording

In April 2025, we dosed the first participant in Canada for the Phase 1 LUMINA clinical trial (NCT06665165), a multicenter, randomized, double-blind, placebo-controlled, multiple ascending dose trial designed to evaluate the safety and biological activity of AMX0114 in people living with ALS. LUMINA will also assess ALS biomarkers, including change from baseline in NfL levels. Approximately 48 participants will be randomized 3:1 to receive AMX0114 or placebo by intrathecal administration once every four weeks, for up to four doses. In September 2025, we fully enrolled cohort 1 (n=12) and expect to present early cohort data at the 36th International Symposium on ALS/MND held from December 5-7. Based on biomarker collection and analysis timelines, we anticipate biomarker data will be available in the coming months and expect to present these at a medical meeting in the first half of 2026.

Previous wording

Research and development activities are central to our business model. Product candidates such as avexitide and AMX0035 in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, such as AMX0114, primarily due to the increased size and duration of later-stage clinical trials and related product manufacturing expenses. Despite a decline in research and development expenses in 2024 and in the first quarter of 2025 compared to prior respective periods, research and development expenses increased in the second quarter of 2025 compared to the prior year period and we expect that our research and development expenses will continue to increase in connection with our planned clinical development activities in the near term and in the future. At this time, we cannot accurately estimate or know the nature, timing and costs of the efforts that will be necessary to complete the clinical development of avexitide, AMX0035, AMX0114 and any future product candidates. Our clinical development costs may vary significantly based on factors such as:

Current wording

Research and development activities are central to our business model. Product candidates such as avexitide and AMX0035 in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, such as AMX0114, primarily due to the increased size and duration of later-stage clinical trials and related product manufacturing expenses. Despite a decline in research and development expenses in 2024 and in the third quarter of 2025 compared to prior respective periods, we expect that our research and development expenses will increase in connection with our planned clinical development activities in the near term and in the future. At this time, we cannot accurately estimate or know the nature, timing and costs of the efforts that will be necessary to complete the clinical development of avexitide, AMX0035, AMX0114 and any future product candidates. Our clinical development costs may vary significantly based on factors such as:

Previous wording

The successful development and commercialization of avexitide, AMX0035 and any other current or future product candidates is highly uncertain, due to the numerous risks and uncertainties associated with product development and commercialization, including the following:

Current wording

The successful development and commercialization of avexitide, AMX0035, AMX0114 and any other current or future product candidates is highly uncertain, due to the numerous risks and uncertainties associated with product development and commercialization, including the following:

Previous wording

Selling, general and administrative expenses consist primarily of salaries and related costs for personnel in executive, finance, sales, marketing, as well as administrative functions. Selling, general and administrative expenses also include legal fees relating to patent and corporate matters; professional fees for accounting, auditing, tax and administrative consulting services; corporate insurance costs; administrative travel expenses; sales and marketing expenses; information technology; charitable donations to independent charitable foundations; facility-related and other operating costs. In April 2024, we announced a restructuring plan, or the Restructuring Plan, designed to focus our resources on key clinical and preclinical programs. The restructuring included a reduction in force which reduced our workforce by approximately 70% and a decrease in external financial commitments outside our priority areas. As a result, our selling, general and administrative expenses decreased during the three and six months ended June 30, 2025 as compared to the three and six months ended June 30, 2024. However, we expect that general and administrative expenses will increase in future periods as we advance our clinical pipeline.

Current wording

Selling, general and administrative expenses consist primarily of salaries and related costs for personnel in executive, finance, sales, marketing, as well as administrative functions. Selling, general and administrative expenses also include legal fees relating to patent and corporate matters; professional fees for accounting, auditing, tax and administrative consulting services; corporate insurance costs; administrative travel expenses; sales and marketing expenses; information technology; charitable donations to independent charitable foundations; facility-related and other operating costs. In April 2024, we announced a restructuring plan, or the Restructuring Plan, designed to focus our resources on key clinical and preclinical programs. The restructuring included a reduction in force which reduced our workforce by approximately 70% and a decrease in external financial commitments outside our priority areas. As a result, our selling, general and administrative expenses decreased during the three and nine months ended September 30, 2025 as compared to the three and nine months ended September 30, 2024. However, we expect that selling, general and administrative expenses will increase in future periods as we advance our clinical pipeline.

Previous wording

Research and development expenses were $27.2 million for the three months ended June 30, 2025, compared to $23.3 million for the three months ended June 30, 2024. The increase was primarily due to a $7.1 million increase in expenses related to the pivotal Phase 3 LUCIDITY clinical trial for avexitide in PBH, a $3.6 million increase in expenses related to AMX0035 for the treatment of PSP and a $1.7 million increase in expenses related to other research and development activities. The increase was partially offset by a $8.6 million decrease in spending on AMX0035 for the treatment of ALS following the topline data from the PHOENIX trial.

Current wording

Research and development expenses were $19.9 million for the three months ended September 30, 2025, compared to $21.2 million for the three months ended September 30, 2024. The decrease was primarily due to a $1.9 million decrease in expenses related to AMX0035 for the treatment of PSP after its discontinuation and a $4.1 million decrease in spending on AMX0035 for the treatment of ALS following the topline data from the PHOENIX trial. The decrease was offset by a $4.8 million increase in expenses related to the pivotal Phase 3 LUCIDITY clinical trial in PBH and other costs related to avexitide.

Previous wording

Selling, general and administrative expenses were $15.6 million for the three months ended June 30, 2025 compared to $21.6 million for the three months ended June 30, 2024. The decrease was primarily due to a decrease of $0.8 million in payroll and personnel-related costs, a decrease of $2.0 million in consulting and professional services and a decrease of $3.2 million in other expenses. The decrease in payroll and personnel-related costs was primarily related to a decrease in the number of employees as a result of the Restructuring Plan. The decrease in consulting and professional services is primarily due to a decrease in commercial sales and marketing activity as a result of the RELYVRIO®/ALBRIOZA™Discontinuation. The decrease in other expenses is primarily due to a decrease in facilities and IT-related expenses.

Current wording

Selling, general and administrative expenses were $16.2 million for the three months ended September 30, 2025 compared to $17.8 million for the three months ended September 30, 2024. The decrease was primarily due to a decrease of $2.1 million in other expenses and a decrease of $0.8 million in consulting and professional services. The decrease was partially offset by an increase of $1.2 million in payroll and personnel-related costs. The decrease in other expenses is primarily due to lower facilities and IT-related expenses. The decrease in consulting and professional services is primarily due to a decrease in commercial sales and marketing activity as a result of the RELYVRIO®/ALBRIOZA™Discontinuation. The increase in payroll and personnel-related costs was primarily driven by an increase in stock-based compensation.

Previous wording

As a result of the RELYVRIO®/ALBRIOZA™ Discontinuation, we did not generate revenue from product sales for the six months ended June 30, 2025. For the six months ended June 30, 2024, product revenue, net was primarily related to units of RELYVRIO and ALBRIOZA previously sold in the U.S. and Canada.

Current wording

As a result of the RELYVRIO®/ALBRIOZA™ Discontinuation, we did not generate revenue from product sales for the nine months ended September 30, 2025. For the nine months ended September 30, 2024, product revenue, net was primarily related to units of RELYVRIO and ALBRIOZA previously sold in the U.S. and Canada.

Previous wording

As a result of the RELYVRIO®/ALBRIOZA™ Discontinuation, we did not generate cost of sales for the three months ended June 30, 2025. Cost of sales for the three months ended June 30, 2024 consisted of costs to procure, manufacture and distribute our marketed products, RELYVRIO and ALBRIOZA. As a result of the RELYVRIO®/ALBRIOZA™ Discontinuation, we recorded approximately $7.4 million of charges associated with the write-down of inventory and losses on firm purchase commitments for the three months ended June 30, 2024.

Current wording

As a result of the RELYVRIO®/ALBRIOZA™ Discontinuation, we did not generate cost of sales for the nine months ended September 30, 2025. Cost of sales for the nine months ended September 30, 2024 consisted of costs to procure, manufacture and distribute our marketed products, RELYVRIO and ALBRIOZA. As a result of the RELYVRIO®/ALBRIOZA™ Discontinuation, we recorded approximately $118.7 million of charges associated with the write-down of inventory and losses on firm purchase commitments for the nine months ended September 30, 2024.

Previous wording

The following table summarizes our research and development expenses for the six months ended June 30, 2025 and 2024 (in thousands):

Current wording

The following table summarizes our research and development expenses for the nine months ended September 30, 2025 and 2024 (in thousands):

Previous wording

Research and development expenses were $49.3 million for the six months ended June 30, 2025, compared to $60.0 million for the six months ended June 30, 2024. The decrease was primarily due to a $22.6 million decrease in spending on AMX0035 for the treatment of ALS following the topline data from the PHOENIX trial, and a $7.1 million decrease in payroll and personnel-related costs primarily related to a decrease in the number of employees as a result of the Restructuring Plan. The decrease was partially offset by a $12.5 million increase in expenses related to the pivotal Phase 3 LUCIDITY clinical trial for avexitide in PBH, a $4.0 million increase in expenses related to AMX0035 for the treatment of PSP and a $2.5 million increase in expenses related to other research and development activities.

Current wording

Research and development expenses were $69.2 million for the nine months ended September 30, 2025, compared to $81.2 million for the nine months ended September 30, 2024. The decrease was primarily due to a $26.6 million decrease in spending on AMX0035 for the treatment of ALS following the topline data from the PHOENIX trial, and a $6.8 million decrease in payroll and personnel-related costs primarily related to a decrease in the number of employees as a result of the Restructuring Plan. The decrease was partially offset by a $17.3 million increase in expenses related to the pivotal Phase 3 LUCIDITY clinical trial in PBH and other costs related to avexitide, a $2.0 million increase in expenses related to AMX0035 for the treatment of PSP, and a $2.1 million increase in expenses related to other research and development activities.

Previous wording

Selling, general and administrative expenses were $31.3 million for the six months ended June 30, 2025 compared to $79.4 million for the six months ended June 30, 2024. The decrease was primarily due to a decrease of $16.8 million in payroll and personnel-related costs, a decrease of $14.8 million in consulting and professional services and a decrease of $16.5 million in other expenses. The decrease in payroll and personnel-related costs was primarily related to a decrease in the number of employees as a result of the Restructuring Plan. The decrease in consulting and professional services is primarily due to a decrease in commercial sales and marketing activity as a result of the RELYVRIO®/ALBRIOZA™Discontinuation. The decrease in other expenses is primarily due to a decrease in charitable contributions and activity to wind down commercial operations.

Current wording

Selling, general and administrative expenses were $47.5 million for the nine months ended September 30, 2025 compared to $97.2 million for the nine months ended September 30, 2024. The decrease was primarily due to a decrease of $15.6 million in payroll and personnel-related costs, a decrease of $15.6 million in consulting and professional services and a decrease of $18.5 million in other expenses. The decrease in payroll and personnel-related costs was primarily related to a decrease in the number of employees as a result of the Restructuring Plan. The decrease in consulting and professional services is primarily due to a decrease in commercial sales and marketing activity as a result of the RELYVRIO®/ALBRIOZA™Discontinuation. The decrease in other expenses is primarily due to a decrease in charitable contributions, lower facilities and IT-related expenses, and a decrease in activity to wind down commercial operations.

Previous wording

We did not recognize restructuring expenses for the three months ended June 30, 2025. During the three months ended June 30, 2024, restructuring expenses were approximately $22.9 million which includes employee severance and termination benefits of approximately $21.8 million, contract termination costs, impairment of long-lived assets and other costs of $1.0 million. We substantially completed the Restructuring Plan in the second quarter of 2024.

Current wording

We did not recognize restructuring expenses for the nine months ended September 30, 2025. During the nine months ended September 30, 2024, restructuring expenses were approximately $22.9 million, which includes employee severance and termination benefits of approximately $21.8 million, contract termination costs, impairment of long-lived assets and other costs of $1.0 million. We substantially completed the Restructuring Plan in the second quarter of 2024.

Previous wording

As of June 30, 2025, we had cash, cash equivalents and marketable securities of $180.8 million and an accumulated deficit of $684.0 million. We believe our existing cash, cash equivalents and marketable securities as of June 30, 2025 will be sufficient to meet our anticipated operating and capital expenditure requirements through 2026. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.

Current wording

As of September 30, 2025, we had cash, cash equivalents and marketable securities of $344.0 million and an accumulated deficit of $718.4 million. We believe our existing cash, cash equivalents and marketable securities as of September 30, 2025 will be sufficient to fund our operations into 2028. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect.

Previous wording

continue our research and development efforts of AMX0035, including our ongoing Phase 2b/3 trial of AMX0035 in PSP and our ongoing Phase 2 trial of AMX0035 for the treatment of Wolfram syndrome;

Current wording

continue our research and development efforts of AMX0035, including our ongoing Phase 2 trial of AMX0035 for the treatment of Wolfram syndrome and winding down of the Phase 2b/3 trial of AMX0035 in PSP;

Previous wording

During the six months ended June 30, 2025, operating activities used $65.1 million of cash, primarily resulting from our net loss of $77.4 million and $3.2 million net accretion of discounts on investments, offset by $14.2 million of non-cash stock-based compensation expense and $0.8 million of net cash provided by changes in our operating assets and liabilities.

Current wording

During the nine months ended September 30, 2025, operating activities used $95.4 million of cash, primarily resulting from our net loss of $111.7 million, $4.5 million net accretion of discounts on investments, and $1.2 million of net cash used by changes in our operating assets and liabilities, offset by $21.3 million of non-cash stock-based compensation expense.

Previous wording

Net cash provided by changes in our operating assets and liabilities primarily consisted of a $6.5 million decrease in prepaid expenses and other current assets, and a $1.1 million decrease in operating ROU assets, offset by a $6.5 million decrease in accrued expenses and a $1.2 million decrease in operating lease liabilities.

Current wording

Net cash used by changes in our operating assets and liabilities primarily consisted of a $5.4 million decrease in accrued expenses and a $1.2 million decrease in operating lease liabilities, offset by a $3.3 million decrease in prepaid expenses and other current assets and a $1.4 million decrease in operating ROU assets.

Previous wording

During the six months ended June 30, 2024, operating activities used $67.1 million of cash, primarily resulting from our net loss of $191.5 million, $5.8 million net accretion of discounts on investments and $8.5 million of net cash used by changes in our operating assets and liabilities, offset by $117.9 million of inventory impairment and loss on firm purchase commitments and $19.5 million of non-cash stock-based compensation expense.

Current wording

During the nine months ended September 30, 2024, operating activities used $108.6 million of cash, primarily resulting from our net loss of $264.2 million, $8.1 million net accretion of discounts on investments and $19.1 million of net cash used by changes in our operating assets and liabilities, offset by $118.7 million of inventory impairment and loss on firm purchase commitments and $26.3 million of non-cash stock-based compensation expense. Our net loss non-cash adjustments also include $36.2 million of acquired IPR&D assets, which are classified as investing activities.

Previous wording

Net cash used by changes in our operating assets and liabilities primarily consisted of a $36.0 million decrease in accounts receivable, net, offset by a $9.3 million increase in inventory capitalized during the period, net of inventory written-off, a decrease of $20.3 million in accrued expenses and a $17.1 million decrease in accounts payable.

Current wording

Net cash used by changes in our operating assets and liabilities primarily consisted of a $38.3 million decrease in accounts receivable, net, offset by a $9.3 million increase in inventory capitalized during the period, net of inventory written-off, a decrease of $33.4 million in accrued expenses and a $20.6 million decrease in accounts payable.

Previous wording

During the six months ended June 30, 2025, net cash used in investing activities was $20.7 million, resulting primarily from $142.6 million of purchases of marketable securities, offset by $122.0 million of investments that matured.

Current wording

During the nine months ended September 30, 2025, net cash used in investing activities was $15.4 million, resulting primarily from $201.4 million of purchases of marketable securities, offset by $186.0 million of investments that matured.

Previous wording

During the six months ended June 30, 2024, net cash used in investing activities was $28.1 million, resulting primarily from $232.0 million of purchases of short-term investments during the period, offset by $204.0 million of investments that matured.

Current wording

During the nine months ended September 30, 2024, net cash provided by investing activities was $10.7 million, resulting primarily from $279.0 million of investments that matured, offset by $232.0 million of purchases of marketable securities and a $36.2 million cash outflow to acquire IPR&D assets related to the avexitide asset acquisition.

Previous wording

During the six months ended June 30, 2025, net cash provided by financing activities was $65.6 million. This amount consisted primarily of $65.6 million in proceeds from the January 2025 Offering, net of offering costs.

Current wording

During the nine months ended September 30, 2025, net cash provided by financing activities was $257.2 million. This amount consisted primarily of $65.5 million in proceeds from the January 2025 Offering, net of offering costs paid, and $191.1 million in proceeds from the September 2025 Offering, net of offering costs paid.

Previous wording

During the six months ended June 30, 2024, net cash provided by financing activities was $0.2 million. This amount consisted primarily of $0.2 million of proceeds from exercises of stock options and vesting of stock awards, net of withholding taxes paid on stock-based awards.

Current wording

During the nine months ended September 30, 2024, net cash provided by financing activities was $0.2 million. This amount consisted primarily of $0.2 million of proceeds from exercises of stock options and vesting of stock awards, net of withholding taxes paid on stock-based awards.