Amylyx Pharmaceuticals, Inc.

Previous wording

We are a clinical-stage pharmaceutical company with a mission to develop and advance 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 for which we believe these therapies can make the greatest impact, based on well-defined mechanistic rationales, clear clinical outcomes and biomarkers, and rigorous preclinical data, agnostic of modality. We are currently developing four investigational therapies for potential impact across several diseases: avexitide in PBH, AMX0035 in Wolfram syndrome, AMX0114 in ALS, and AMX0318 in PBH and other rare diseases.

Current wording

We are a pre-commercial pharmaceutical company with a mission to develop and advance novel therapies for communities with high unmet medical needs. We have nonclinical 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 for which we believe these therapies can make the greatest impact, based on well-defined mechanistic rationales, clear clinical outcomes and biomarkers, and rigorous nonclinical data, agnostic of modality. We are currently developing four investigational therapies for potential impact across several diseases: avexitide in PBH, AMX0318 in PBH and other rare diseases, AMX0035 in Wolfram syndrome, and AMX0114 in ALS.

Previous wording

LUCIDITY (NCT06747468) is a 78-participant, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in participants with PBH following RYGB surgery. The Phase 3 trial is being conducted at 21 sites in the U.S. Participants were randomized 3:2 to receive either 90 mg of avexitide subcutaneously once daily or placebo. The trial includes an up to six-week screening period, including a three-week run-in period, a 16-week double-blind treatment period, and an open-label extension (OLE) period with a duration of 32 weeks. The last participant completed the final trial visit in the 16-week double-blind period of the LUCIDITY trial of avexitide. The primary efficacy objective of LUCIDITY is to evaluate the FDA-agreed-upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Safety and tolerability will also be evaluated. We continue to expect to announce topline data in the third quarter of 2026, and, if approved, a commercial launch in 2027.

Current wording

LUCIDITY (NCT06747468) was a 78-participant, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in participants with PBH following RYGB surgery. The primary objective of LUCIDITY was to evaluate the prespecified primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Safety and tolerability were also evaluated.

Previous wording

LUCIDITY was informed by data from five clinical trials of avexitide in people with PBH showing consistent, dose-dependent effects across studies. The five clinical trials include a Phase 1 trial, a single ascending dose trial, a multiple ascending dose trial, and two Phase 2 trials:

Current wording

LUCIDITY was informed by data from five clinical trials of avexitide in people with PBH showing consistent, statistically significant, dose-dependent effects across studies.

Previous wording

AMX0318 is a novel GLP-1 receptor antagonist for long-acting administration selected as a development candidate for PBH and other rare diseases in January 2026. AMX0318 was selected as a development candidate after demonstrating robust preclinical and chemical properties, including a favorable pharmacokinetic profile that may support long-acting administration, a robust chemical stability profile, strongin vitro potency, evidence ofin vivo activity and tolerability, and high solubility. AMX0318 was identified through a research collaboration with Gubra, a company specializing in peptide-based drug discovery and preclinical contract research services. IND-enabling studies for AMX0318 are underway with an IND submission targeted for 2027.

Current wording

AMX0318 is a novel GLP-1 receptor antagonist for long-acting administration selected as a development candidate for PBH and other rare diseases in January 2026. AMX0318 was selected as a development candidate after demonstrating robust nonclinical and chemical properties, including a favorable pharmacokinetic profile that may support long-acting administration, a robust chemical stability profile, strongin vitro potency, evidence ofin vivo activity and tolerability, and high solubility. AMX0318 was identified through a research collaboration with Gubra, a company specializing in peptide-based drug discovery and nonclinical contract research services. IND-enabling studies for AMX0318 are underway with an IND submission targeted for 2027.

Previous wording

In addition to avexitide, we are advancing AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol in Wolfram syndrome, AMX0114 in ALS, and AMX0318 in PBH and other rare diseases.

Current wording

In addition, we are advancing AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol in Wolfram syndrome and AMX0114 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 Wolfram syndrome, a neurodegenerative disease where ER stress and mitochondrial dysfunction are implicated.

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 Wolfram syndrome, a neurodegenerative disease where ER stress and mitochondrial dysfunction are implicated. The FDA and European Medicines Agency, or EMA, previously granted AMX0035 Orphan Drug Designation for Wolfram Syndrome.

Previous wording

Wolfram syndrome is a rare, monogenic, progressive neurodegenerative disorder that progressively impacts multiple organs and systems. Wolfram syndrome is characterized by childhood-onset diabetes mellitus, optic nerve atrophy, and neurodegeneration. Common manifestations of Wolfram syndrome include diabetes mellitus and diabetes insipidus, gradual vision loss leading to blindness, hearing loss, neurogenic bladder, difficulties with balance and coordination, and difficulty breathing that can lead to respiratory failure and premature mortality. Wolfram syndrome is most commonly caused by pathogenic variants in Wolfram syndrome type 1 gene, or WFS1. Because of the clear link between WFS1 mutations and ER stress, Wolfram syndrome is considered a prototypical ER stress disorder. Wolfram syndrome affects approximately 3,000 people living in the U.S., and there are currently no FDA-approved treatment options.

Current wording

Wolfram syndrome is a rare, monogenic, progressive neurodegenerative disorder that progressively impacts multiple organs and systems and is characterized by childhood-onset diabetes mellitus, optic nerve atrophy, and neurodegeneration. Wolfram syndrome is most commonly caused by pathogenic variants in Wolfram syndrome type 1 gene, or WFS1. Because of the clear link between WFS1 mutations and ER stress, Wolfram syndrome is considered a prototypical ER stress disorder. Wolfram syndrome affects approximately 3,000 people living in the U.S., and there are currently no FDA-approved treatment options.

Previous wording

In May 2025, we announced positive data from HELIOS at Week 48. Consistent with the HELIOS trial’s previously presented primary efficacy outcome of improvement in pancreatic function (as described above), treatment with AMX0035 through Week 48 demonstrated continued and sustained improvement in pancreatic beta cell function. Treatment with AMX0035 from Week 24 to Week 48 also showed sustained improvements or stabilization in glycemic control, as measured by hemoglobin A1c, or 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. In June 2026, we presented longer-term Week 96 (n=9) data from HELIOS. At Week 96, measures of pancreatic function and glycemic control were stable or improved relative to baseline in most participants. Visual acuity and patient- and clinician-reported outcomes showed patterns consistent with disease stabilization with interpretation limited by the open-label, single-arm design and small sample size.

Current wording

In October 2024, we announced positive topline data from the Phase 2 open-label HELIOS (NCT05676034) clinical trial of AMX0035 in 12 adults living with Wolfram syndrome. HELIOS is a single-site, single-arm, open-label, proof of biology, Phase 2 trial designed to study the effect of AMX0035 on safety and tolerability, and various measures of endocrinological, neurological, and ophthalmologic function in adult participants living with Wolfram syndrome. At Week 24, treatment with AMX0035 resulted in improvement in pancreatic beta cell function, as measured by C-peptide response, the study’s primary efficacy endpoint,. with improvements or stabilization observed across all secondary endpoints, including hemoglobin A1c (HbA1c), time in target glucose range assessed by continuous glucose monitoring, and visual acuity. Longer term data through Week 48 demonstrated continued improvement in pancreatic beta cell function and sustained improvements or stabilization in glycemic control, and 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. In June 2026, we presented longer-term Week 96 (n=9) data from HELIOS. At Week 96, measures of pancreatic function and glycemic control were stable or improved relative to baseline in most participants. Visual acuity and patient- and clinician-reported outcomes showed patterns consistent with disease stabilization with interpretation limited by the open-label, single-arm design and small sample size.

Previous wording

AMX0114 is an investigational antisense oligonucleotide, or ASO, targeting calpain-2, or CAPN2, with FDA Fast Track Designation for the potential treatment of ALS. Decades of scientific literature and published data demonstrate that CAPN2, a protein involved in neurofilament biology, plays an essential role in axonal degeneration, which is a critical effector in the progression of various neurodegenerative diseases including ALS. ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. One of the ways ALS progresses is through axonal degeneration, which disrupts neural connectivity and contributes significantly to disease pathology. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and, eventually, death. ALS is defined as a rare disease, but it affects as many as 30,000 adults in the U.S. and 3,000 in Canada. The most common form of the disease is sporadic ALS, with more than 90% of people with ALS showing no clear family history.

Current wording

ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. One of the ways ALS progresses is through axonal degeneration, which disrupts neural connectivity and contributes significantly to disease pathology. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and, eventually, death. ALS is defined as a rare disease, but it affects as many as 30,000 adults in the U.S. and 3,000 in Canada. The most common form of the disease is sporadic ALS, with more than 90% of people with ALS showing no clear family history.

Previous wording

In preclinical studies, treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in CAPN2 mRNA and calpain-2 protein levels in disease-relevant cell models of axonal degeneration. This translated to improved neuronal survival and reductions in extracellular neurofilament light chain, or NfL levels, a broadly researched biomarker for axonal degeneration in ALS, across multiple disease models and paradigms of neuronal injury. AMX0114 was generally well-tolerated in in vivo preclinical safety studies.

Current wording

In nonclinical studies, treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in CAPN2 mRNA and calpain-2 protein levels in disease-relevant cell models of axonal degeneration. This translated to improved neuronal survival and reductions in extracellular neurofilament light chain, or NfL levels, a broadly researched biomarker for axonal degeneration in ALS, across multiple disease models and paradigms of neuronal injury. AMX0114 was generally well-tolerated in in vivo nonclinical safety studies.

Previous wording

In June 2026, we presented Cohort 1 biomarker data at the Annual Meeting of the European Network to Cure ALS. AMX0114 showed no drug-related serious adverse events, or SAEs, and no serious nervous system disorder adverse events, or AEs, in Cohort 1 (lowest dose level 12.5 mg). These safety findings support continued evaluation of AMX0114 at higher dose levels in this multiple ascending dose trial. Biomarker levels, including spectrin breakdown product 145, or SBDP-145, neurofilament light, or NfL, and phosphorylated neurofilament heavy, or pNFH, remained near baseline at Day 145. Cohorts 1 and Cohort 2 (25 mg) are fully enrolled, and Cohort 3 (50 mg) is currently enrolling.

Current wording

In June 2026, we presented Cohort 1 biomarker data at the Annual Meeting of the European Network to Cure ALS. AMX0114 showed no drug-related serious adverse events, or SAEs, and no serious nervous system disorder adverse events, or AEs, in Cohort 1 (lowest dose level 12.5 mg). These safety findings support continued evaluation of AMX0114 at higher dose levels in this multiple ascending dose trial. Biomarker levels, including spectrin breakdown product 145, or SBDP-145, neurofilament light, or NfL, and phosphorylated neurofilament heavy, or pNFH, remained near baseline at Day 145. Cohort 1, Cohort 2 (25 mg) and Cohort 3 (50 mg) are fully enrolled and Cohort 4 (100 mg) recently began enrolling.

Previous wording

As of June 30, 2026, we had cash, cash equivalents and marketable securities of $250.8 million and an accumulated deficit of $836.1 million. We believe our existing cash, cash equivalents and marketable securities as of June 30, 2026 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. See “Liquidity and Capital Resources” below.

Current wording

As of September 30, 2026, we had cash, cash equivalents and marketable securities of $768.1 million and an accumulated deficit of $881.6 million. We believe our existing cash, cash equivalents and marketable securities as of September 30, 2026 provide us with the financial resources to support our planned operations, including the potential commercialization of avexitide, if approved, and advance our pipeline priorities. 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. See “Liquidity and Capital Resources” below.

Previous wording

expenses incurred under agreements with CROs, CMOs, as well as investigative sites and consultants that conduct our clinical trials, preclinical studies and other scientific development services;

Current wording

expenses incurred under agreements with contract research organizations, or CROs, contract manufacturing organizations, or CMOs, as well as investigative sites and consultants that conduct our clinical trials, nonclinical studies and other scientific development services;

Previous wording

manufacturing scale-up expenses and the cost of acquiring and manufacturing drug product for our preclinical studies and clinical trials, including manufacturing registration and validation batches, as well as pre-commercial manufacturing activities;

Current wording

manufacturing scale-up expenses and the cost of acquiring and manufacturing drug product for our nonclinical studies and clinical trials, including manufacturing registration and validation batches, as well as pre-commercial manufacturing activities;

Previous wording

Certain of our indirect research and development expenses are not tracked on an indication-by-indication basis. We do not allocate employee costs and facilities, including depreciation or other indirect costs, to specific indications because these costs are deployed across multiple indications and, as such, are not separately classified. We use internal resources to oversee the research and discovery as well as to manage our preclinical development, process development, manufacturing and clinical development activities. These employees work across multiple indications and, therefore, we do not track their costs by indication.

Current wording

Certain of our indirect research and development expenses are not tracked on an indication-by-indication basis. We do not allocate employee costs and facilities, including depreciation or other indirect costs, to specific indications because these costs are deployed across multiple indications and, as such, are not separately classified. We use internal resources to oversee the research and discovery as well as to manage our nonclinical development, process development, manufacturing and clinical development activities. These employees work across multiple indications and, therefore, we do not track their costs by indication.

Previous wording

the efficacy and safety profile from clinical trials and preclinical studies of our current or future product candidates; and

Current wording

the efficacy and safety profile from clinical trials and nonclinical studies of our current or future product candidates; and

Previous wording

the timing and progress of preclinical and clinical development activities;

Current wording

the timing and progress of nonclinical and clinical development activities;

Previous wording

the number and scope of preclinical and clinical trials for separate indications we decide to pursue;

Current wording

the number and scope of nonclinical and clinical trials for separate indications we decide to pursue;

Previous wording

Research and development expenses were $23.8 million for the three months ended June 30, 2026, compared to $27.2 million for the three months ended June 30, 2025. The decrease was primarily due to a $7.1 million decrease in expenses related to AMX0035 for the treatment of PSP after its discontinuation. The decrease was partially offset by a $2.3 million increase in expenses related to the pivotal Phase 3 LUCIDITY clinical trial in PBH and other costs related to avexitide.

Current wording

Research and development expenses were $28.4 million for the three months ended September 30, 2026, compared to $19.9 million for the three months ended September 30, 2025. The increase was primarily due to a $5.8 million increase in expenses related to the pivotal Phase 3 LUCIDITY clinical trial in PBH and other costs related to avexitide, and a $3.3 million increase in expenses related to other programs. The increase was partially offset by a $3.4 million decrease in expenses related to AMX0035 for the treatment of PSP after its discontinuation.

Previous wording

Selling, general and administrative expenses were $21.9 million for the three months ended June 30, 2026 compared to $15.6 million for the three months ended June 30, 2025. The increase was primarily due to increases of $5.6 million in consulting and professional services and $1.0 million in payroll and personnel-related costs, including stock-based compensation, partially offset by a decrease of $0.3 million in other expenses. The increase in consulting and professional services was primarily due to increased spend for legal expenses related to the settlement of the February 2024 Shih Complaint, as well as an increase in commercial and marketing activity as we prepare for a potential commercial launch of avexitide, if approved. The increase in payroll and personnel-related costs was primarily due to hiring additional personnel to support our pre-commercialization activities.

Current wording

Selling, general and administrative expenses were $21.3 million for the three months ended September 30, 2026 compared to $16.2 million for the three months ended September 30, 2025. The increase was primarily due to increases of $3.2 million in payroll and personnel-related costs, including stock-based compensation and $1.9 million in consulting and professional services. The increase in payroll and personnel-related costs was primarily due to hiring additional personnel to support our pre-commercialization activities and the vesting of performance-based restricted stock units, or PSUs, upon the achievement of certain performance metrics during the period. The increase in consulting and professional services was primarily due to increased spend for commercial and marketing activity as we prepare for a potential commercial launch of avexitide, if approved.

Previous wording

The following table summarizes our research and development expenses for thesix months ended June 30, 2026 and 2025(in thousands):

Current wording

The following table summarizes our research and development expenses for the nine months ended September 30, 2026 and 2025 (in thousands):

Previous wording

Research and development expenses were $51.4 million for the six months ended June 30, 2026, compared to $49.3 million for the six months ended June 30, 2025. The increase was primarily due to a $6.8 million increase in expenses related to the pivotal Phase 3 LUCIDITY clinical trial in PBH and other costs related to avexitide, and a $4.1 million increase in expenses related to other programs, including milestone payments totaling $4.0 million made to Gubra following the selection and handover of AMX0318 as a development candidate for PBH and other rare diseases during the six months ended June 30, 2026. The increase was partially offset by a $11.0 million decrease in expenses related to AMX0035 for the treatment of PSP after its discontinuation.

Current wording

Research and development expenses were $79.7 million for the nine months ended September 30, 2026, compared to $69.2 million for the nine months ended September 30, 2025. The increase was primarily due to a $12.6 million increase in expenses related to the pivotal Phase 3 LUCIDITY clinical trial in PBH and other costs related to avexitide, and a $7.4 million increase in expenses related to other programs, including milestone payments totaling $4.0 million made to Gubra following the selection and handover of AMX0318 as a development candidate for PBH and other rare diseases during the nine months ended September 30, 2026. The increase was partially offset by a $14.4 million decrease in expenses related to AMX0035 for the treatment of PSP after its discontinuation.

Previous wording

Selling, general and administrative expenses were $38.1 million for the six months ended June 30, 2026 compared to $31.3 million for the six months ended June 30, 2025. The increase was primarily due to increases of $6.3 million in consulting and professional services and $1.0 million in payroll and personnel-related costs, including stock-based compensation, partially offset by a decrease of $0.5 million in other expenses. The increase in consulting and professional services was primarily due to increased spend for legal expenses related to the settlement of the February 2024 Shih Complaint, as well as an increase in commercial and marketing activity as we prepare for a potential commercial launch of avexitide, if approved. The increase in payroll and personnel-related costs was primarily due to hiring additional personnel to support our pre-commercialization activities.

Current wording

Selling, general and administrative expenses were $59.4 million for the nine months ended September 30, 2026 compared to $47.5 million for the nine months ended September 30, 2025. The increase was primarily due to increases of $8.2 million in consulting and professional services and $4.2 million in payroll and personnel-related costs, including stock-based compensation, partially offset by a decrease of $0.5 million in other expenses. The increase in consulting and professional services was primarily due to increased spend for legal expenses related to the settlement of the February 2024 Shih Complaint, as well as an increase in commercial and marketing activity as we prepare for a potential commercial launch of avexitide, if approved. The increase in payroll and personnel-related costs was primarily due to hiring additional personnel to support our pre-commercialization activities and the vesting of PSU awards upon the achievement of certain performance metrics during the period.

Previous wording

As of June 30, 2026, we had cash, cash equivalents and marketable securities of $250.8 million and an accumulated deficit of $836.1 million. We believe our existing cash, cash equivalents and marketable securities as of June 30, 2026 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.

Current wording

As of September 30, 2026, we had cash, cash equivalents and marketable securities of $768.1 million and an accumulated deficit of $881.6 million. We believe our existing cash, cash equivalents and marketable securities as of September 30, 2026 provide us with the financial resources to support our planned operations, including the potential commercialization of avexitide, if approved, and advance our pipeline priorities for the foreseeable future. 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

Since inception, we have devoted substantially all of our efforts to research and development, pre-commercialization and commercialization activities, including recruiting management and technical staff, raising capital, producing materials for preclinical studies and clinical trials, and building infrastructure to support such activities. As of June 30, 2026, we have funded our operations primarily through public offerings of our common stock, private sales of preferred stock, convertible notes, and through revenue from sales of RELYVRIO and ALBRIOZA in the U.S. and Canada, respectively, between July 2022 and April 2024.

Current wording

Since inception, we have devoted substantially all of our efforts to research and development, pre-commercialization and commercialization activities, including recruiting management and technical staff, raising capital, producing materials for nonclinical studies and clinical trials, and building infrastructure to support such activities. As of September 30, 2026, we have funded our operations primarily through public offerings of our common stock, private sales of preferred stock, convertible notes, and through revenue from sales of RELYVRIO and ALBRIOZA in the U.S. and Canada, respectively, between July 2022 and April 2024.

Previous wording

We expect our expenses to increase in connection with our ongoing activities, particularly as we advance the preclinical activities, manufacturing and clinical trials of avexitide, AMX0035, AMX0114, AMX0318 and any other current or future product candidates or acquire or in-license additional product candidates or products. We may also incur expenses related to business development activities, such as in-licensing or acquisition of product candidates. In addition, we expect to incur additional costs associated with operating as a public company, including significant legal, accounting, investor relations and other expenses. We expect to incur significant expenses as we:

Current wording

We expect our expenses to increase in connection with our ongoing activities, particularly as we advance the nonclinical activities, manufacturing and clinical trials of avexitide, AMX0035, AMX0114, AMX0318 and any other current or future product candidates or acquire or in-license additional product candidates or products, and as we build a commercial enterprise as part of our commercial readiness efforts for avexitide in PBH, if approved. We may also incur expenses related to business development activities, such as in-licensing or acquisition of product candidates. In addition, we expect to incur costs associated with operating as a public company, including significant legal, accounting, investor relations and other expenses. We expect to incur significant expenses as we:

Previous wording

conduct preclinical studies and clinical trials for AMX0035 for additional indications and for potential future product candidates;

Current wording

conduct nonclinical studies and clinical trials for AMX0035 for additional indications and for potential future product candidates;

Previous wording

continue our preclinical efforts of AMX0318, including advancing IND-enabling studies;

Current wording

continue our nonclinical efforts of AMX0318, including advancing IND-enabling studies;

Previous wording

hire and retain additional personnel, such as preclinical, clinical, quality assurance, regulatory affairs, manufacturing, distribution, legal, compliance, finance, general and administrative, commercial and scientific personnel; and

Current wording

hire and retain additional personnel, such as nonclinical, clinical, quality assurance, regulatory affairs, manufacturing, distribution, legal, compliance, finance, general and administrative, commercial and scientific personnel; and

Previous wording

the scope, progress, results and costs of drug discovery, laboratory testing, preclinical and clinical development for avexitide, AMX0035, AMX0114, AMX0318 and any future product candidates;

Current wording

the scope, progress, results and costs of drug discovery, laboratory testing, nonclinical and clinical development for avexitide, AMX0035, AMX0114, AMX0318 and any future product candidates;

Previous wording

timing delays with respect to preclinical and clinical development of avexitide, AMX0035, AMX0114, AMX0318 and any future product candidates, including as result of any future outbreak of any highly infectious or contagious diseases;

Current wording

timing delays with respect to nonclinical and clinical development of avexitide, AMX0035, AMX0114, AMX0318 and any future product candidates, including as result of any future outbreak of any highly infectious or contagious diseases;

Previous wording

During the six months ended June 30, 2026, operating activities used $68.3 million of cash, primarily resulting from our net loss of $84.7 million, $1.8 million net accretion of discounts on investments, and $0.4 million of net cash used by changes in our operating assets and liabilities, offset by $14.4 million of non-cash stock-based compensation expense and a $4.0 million charge for IPR&D milestones related to the Gubra collaboration.

Current wording

During the nine months ended September 30, 2026, operating activities used $102.0 million of cash, primarily resulting from our net loss of $130.2 million and $2.9 million net accretion of discounts on investments, offset by $24.8 million of non-cash stock-based compensation expense, a $4.0 million charge for IPR&D milestones related to the Gubra collaboration, and $2.1 million of net cash provided by changes in our operating assets and liabilities.

Previous wording

Net cash used by changes in our operating assets and liabilities primarily consisted of a $3.5 million decrease in accrued expenses and a $0.7 million decrease in operating lease liabilities, offset by a $1.1 million decrease in prepaid expenses and other current assets and a $2.0 million increase in accounts payable.

Current wording

Net cash provided by changes in our operating assets and liabilities primarily consisted of a $4.9 million increase in accrued expenses, a $1.3 million increase in accounts payable, and a $0.9 million decrease in operating right-of-use, or ROU, assets, offset by a $4.0 million increase in prepaid expenses and other current assets and a $1.0 million decrease in operating lease liabilities.

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, 2026, net cash used in investing activities was $113.5 million, resulting primarily from $200.5 million of purchases of marketable securities and $4.0 million in IPR&D milestone payments related to the Gubra collaboration, offset by $91.0 million of investments that matured.

Current wording

During the nine months ended September 30, 2026, net cash used in investing activities was $439.2 million, resulting primarily from $604.0 million of purchases of marketable securities and $4.0 million in IPR&D milestone payments related to the Gubra collaboration, offset by $168.8 million of investments that matured.

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, 2026, net cash provided by financing activities was $4.7 million. This amount consisted of $4.7 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, 2026, net cash provided by financing activities was $554.9 million. This amount consisted primarily of $544.6 million in proceeds from the Offering, net of underwriting discounts and commissions and offering costs, and $10.2 million of proceeds from exercises of stock options and vesting of stock awards, net of withholding taxes paid on stock-based awards.

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

We enter into agreements in the normal course of business with CMOs for raw material purchases and manufacturing services. As of June 30, 2026, our remaining contractual payment obligations under these agreements totaled approximately $35.0 million and are expected to be paid through 2028. Of this amount, approximately $13.6 million is expected to be paid within the next 12 months, and approximately $21.5 million is expected to be paid thereafter through 2028.

Current wording

$30.0 million and are expected to be paid through 2028. Of this amount, approximately $18.8 million is expected to be paid within the next 12 months, and approximately $11.3 million is expected to be paid thereafter through 2028.