UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________________ to ___________________ Commission File Number: 001-41199 Amylyx Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) ...

[Q2 2025 Business and Financial Overview](index=1&type=section&id=Amylyx%20Pharmaceuticals%20Reports%20Second%20Quarter%202025%20Financial%20Results) Amylyx Pharmaceuticals highlights clinical pipeline advancements and stable financial performance in Q2 2025 [Management Commentary](index=1&type=section&id=Management%20Commentary) Management expressed encouragement regarding pipeline strength and progress toward key clinical milestones - The company is focused on disciplined execution across its clinical programs, with several key data readouts and updates expected through **H1 2026**[3](index=3&type=chunk) - Key upcoming milestones include: - **Avexitide (LUCIDITY Trial):** Complete recruitment in **2025**, with topline data in **H1 2026** - **AMX0035 (ORION Trial):** Unblinded Phase 2b analysis in **Q3 2025** to inform Phase 3 progression - **AMX0035 (Wolfram Syndrome):** Program update expected later in **2025** - **AMX0114 (LUMINA Trial):** Received FDA Fast Track designation; early cohort data expected later in **2025**[3](index=3&type=chunk) [Second Quarter and Recent Updates](index=1&type=section&id=Second%20Quarter%20and%20Recent%20Updates) Amylyx highlighted significant progress in its clinical trials and maintained a strong cash position through 2026 - Cash, cash equivalents, and marketable securities stood at **$180.8 million** as of June 30, 2025, with a projected cash runway through the end of **2026**[5](index=5&type=chunk) - Presented new data for avexitide (for PBH) showing the **90 mg once-daily dose** led to a **64% reduction (p=0.0031)** in hypoglycemic events versus baseline in a Phase 2b trial[6](index=6&type=chunk) - Presented long-term Week 48 data from the Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome, demonstrating continued and sustained improvement in pancreatic beta cell function and stabilization in other disease progression outcomes[6](index=6&type=chunk) - Received FDA Fast Track designation for AMX0114, an investigational treatment for amyotrophic lateral sclerosis (ALS), in June 2025[7](index=7&type=chunk) [Upcoming Expected Milestones](index=2&type=section&id=Upcoming%20Expected%20Milestones) The company outlined several key catalysts expected in the near term across its clinical pipeline - **Avexitide (LUCIDITY Trial):** Expect to complete recruitment in **2025**, with data readout in **H1 2026** and a potential commercial launch in **2027** if approved[10](index=10&type=chunk) - **AMX0035 (ORION Trial for PSP):** An unblinded analysis of the Phase 2b portion is expected in **Q3 2025**, which will determine the progression to Phase 3[10](index=10&type=chunk) - **AMX0035 (Wolfram Syndrome):** An update on the program, including the design of a Phase 3 trial informed by positive Phase 2 data and FDA discussions, is expected in **2025**[10](index=10&type=chunk) - **AMX0114 (LUMINA Trial for ALS):** Early cohort data from the Phase 1 trial, evaluating safety and biological activity, is expected in **2025**[10](index=10&type=chunk) [Q2 2025 Financial Performance](index=2&type=section&id=Financial%20Results%20for%20the%20Second%20Quarter%20Ended%20June%2030%2C%202025) The company significantly reduced its net loss in Q2 2025, driven by lower operating expenses and a strong cash position [Financial Results Summary](index=2&type=section&id=Financial%20Results%20Summary) In Q2 2025, Amylyx reported a significantly reduced net loss driven by lower operating expenses, while maintaining a solid cash position | Financial Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | R&D Expenses | $27.2 million | $23.3 million | | SG&A Expenses | $15.6 million | $21.6 million | | Net Loss | $41.4 million | $72.7 million | | Net Loss Per Share | $0.46 | $1.07 | - The company's cash, cash equivalents, and marketable securities were **$180.8 million** at the end of Q2 2025, with a projected cash runway through the end of **2026**[12](index=12&type=chunk) [Research and Development (R&D) Expenses](index=2&type=section&id=R%26D%20Expenses) R&D expenses increased in Q2 2025 due to advancing clinical programs for avexitide and AMX0035 for PSP | Expense Category | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | R&D Expenses | $27.2 million | $23.3 million | | Stock-based Compensation (in R&D) | $2.0 million | $2.4 million | - The increase in R&D spending was mainly due to advancing the avexitide and PSP programs[9](index=9&type=chunk) [Selling, General, and Administrative (SG&A) Expenses](index=3&type=section&id=SG%26A%20Expenses) SG&A expenses decreased in Q2 2025, primarily due to lower payroll and professional services costs | Expense Category | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | SG&A Expenses | $15.6 million | $21.6 million | | Stock-based Compensation (in SG&A) | $5.4 million | $7.1 million | - The decrease in SG&A was driven by reduced personnel costs and lower use of consulting and professional services[11](index=11&type=chunk) [Net Loss](index=3&type=section&id=Net%20Loss) The company reported a significantly reduced net loss in Q2 2025 compared to the prior year, reflecting improved cost management | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | Net Loss | $(41.4) million | $(72.7) million | | Net Loss Per Share | $(0.46) | $(1.07) | [Cash Position and Runway](index=3&type=section&id=Cash%20Position) Amylyx maintained a strong cash position of $180.8 million as of June 30, 2025, projected to fund operations through 2026 | Date | Cash, Cash Equivalents, and Marketable Securities | | :--- | :--- | | June 30, 2025 | $180.8 million | | March 31, 2025 | $204.1 million | - Based on current operating plans, the company's cash runway is expected to last through the end of **2026**[12](index=12&type=chunk) [Clinical Pipeline and Programs](index=3&type=section&id=Clinical%20Pipeline%20and%20Programs) Amylyx is advancing multiple clinical programs, including avexitide for PBH, AMX0035 for Wolfram syndrome and PSP, and AMX0114 for ALS [Avexitide Program (for PBH)](index=3&type=section&id=Avexitide%20Program%20(for%20PBH)) Avexitide is an investigational GLP-1 receptor antagonist for post-bariatric hypoglycemia (PBH), currently in a pivotal Phase 3 trial [About Avexitide](index=3&type=section&id=About%20Avexitide) Avexitide is a first-in-class GLP-1 receptor antagonist with FDA Breakthrough Therapy Designation for PBH and HI - Avexitide is an investigational GLP-1 receptor antagonist designed to inhibit the effect of GLP-1, thereby decreasing insulin secretion and stabilizing blood glucose[15](index=15&type=chunk) - The FDA has granted avexitide Breakthrough Therapy Designation for both post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI)[15](index=15&type=chunk) [About Post-Bariatric Hypoglycemia (PBH)](index=3&type=section&id=About%20Post-Bariatric%20Hypoglycemia%20(PBH)) PBH is a debilitating condition affecting approximately 160,000 people in the U.S. with no approved therapies - PBH is estimated to affect approximately **8%** of people in the U.S. who have undergone sleeve gastrectomy or Roux-en-Y gastric bypass surgery (approx. **160,000 people**)[16](index=16&type=chunk) - The condition can cause debilitating neuroglycopenic symptoms like impaired cognition, loss of consciousness, and seizures. There are no approved therapies for PBH[16](index=16&type=chunk) [About the LUCIDITY Trial](index=4&type=section&id=About%20the%20LUCIDITY%20Trial) The LUCIDITY trial is a pivotal Phase 3 study evaluating avexitide's efficacy and safety in PBH, with reduction in hypoglycemic events as the primary endpoint - LUCIDITY is a Phase 3, randomized, double-blind, placebo-controlled trial enrolling approximately **75 participants** at **20 sites** in the U.S[17](index=17&type=chunk)[18](index=18&type=chunk) - The trial is evaluating a **90 mg once-daily dose** of avexitide, with the primary outcome being the reduction in Level 2 and Level 3 hypoglycemic events through Week 16[18](index=18&type=chunk) [Other Key Programs](index=1&type=section&id=Other%20Key%20Programs) Amylyx is advancing AMX0035 for Wolfram syndrome and PSP, and AMX0114 for ALS, with several upcoming data readouts [AMX0035 (for Wolfram Syndrome & PSP)](index=1&type=section&id=AMX0035%20(for%20Wolfram%20Syndrome%20%26%20PSP)) AMX0035 is being evaluated for Wolfram syndrome and progressive supranuclear palsy (PSP), with key data readouts expected soon - **Wolfram Syndrome:** Long-term Week 48 data from the Phase 2 HELIOS trial demonstrated continued sustained improvement or stabilization across key measures, including pancreatic function and vision[6](index=6&type=chunk)[10](index=10&type=chunk) - **PSP:** The Phase 2b portion of the ORION trial is fully enrolled, and an unblinded analysis of Week 24 data in **Q3 2025** will inform a go/no-go decision for the Phase 3 portion[10](index=10&type=chunk) [AMX0114 (for ALS)](index=2&type=section&id=AMX0114%20(for%20ALS)) AMX0114, an investigational antisense oligonucleotide for ALS, received FDA Fast Track designation, with early Phase 1 data expected in 2025 - AMX0114 received FDA Fast Track designation in June 2025, allowing for more frequent communication with the agency[7](index=7&type=chunk) - The Phase 1 LUMINA trial is a multiple ascending dose study in approximately **48 participants**, with early cohort data expected in **2025**[10](index=10&type=chunk) [Financial Statements](index=5&type=section&id=Financial%20Statements) Detailed financial statements for Q2 2025 show balance sheet and income statement performance [Condensed Consolidated Balance Sheets](index=5&type=section&id=CONDENSED%20CONSOLIDATED%20BALANCE%20SHEETS) As of June 30, 2025, Amylyx reported total assets of $194.6 million and a strong cash position of $180.8 million | (in thousands) | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash, cash equivalents and marketable securities | $180,826 | $176,501 | | Total assets | $194,598 | $193,634 | | **Liabilities and Stockholders' Equity** | | | | Total liabilities | $26,721 | $28,869 | | Stockholders' equity | $167,877 | $164,765 | | Total liabilities and stockholders' equity | $194,598 | $193,634 | [Condensed Consolidated Statements of Operations](index=5&type=section&id=CONDENSED%20CONSOLIDATED%20STATEMENTS%20OF%20OPERATIONS) For Q2 2025, Amylyx reported no product revenue and a net loss of $41.4 million, a significant improvement from Q2 2024 | (in thousands, except per share data) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Product revenue, net | $— | $(1,023) | | Total operating expenses | $42,857 | $75,263 | | Loss from operations | $(42,857) | $(76,286) | | Net loss | $(41,443) | $(72,700) | | Net loss per share — basic and diluted | $(0.46) | $(1.07) |

Core Insights - Amylyx Pharmaceuticals presented new exploratory analyses of avexitide at the Endocrine Society's annual meeting, focusing on its potential to treat post-bariatric hypoglycemia (PBH) [1] Group 1: Clinical Trials and Efficacy - The Phase 2b trial of avexitide showed a 64% reduction in moderate to severe hypoglycemic events with a 90 mg once-daily dose, and over half of participants experienced no such events during treatment [2] - The ongoing Phase 3 LUCIDITY trial is evaluating avexitide's safety and efficacy in approximately 75 patients with PBH following Roux-en-Y gastric bypass surgery, with a primary endpoint of reducing Level 2 and 3 hypoglycemic events [3] - Previous trials demonstrated consistent, dose-dependent reductions in hypoglycemic events, with the 90 mg dose showing sustained 24-hour GLP-1 receptor inhibition [4] Group 2: Safety Profile and Future Prospects - Across five clinical trials, avexitide has shown statistically and clinically meaningful efficacy with a favorable safety profile [5] - LUCIDITY is expected to complete enrollment in 2025, with top-line results anticipated in the first half of 2026 and a potential commercial launch in 2027 [5] Group 3: Market Impact - Following the presentation of these findings, AMLX stock increased by 7.55%, reaching $8.26 [5]

Summary of Amylyx Pharmaceuticals (AMLX) Update / Briefing July 13, 2025 Company Overview - **Company**: Amylyx Pharmaceuticals (AMLX) - **Focus**: Development of Avexatide, a potential GLP-1 receptor antagonist for treating post-bariatric hypoglycemia (PBH) with Breakthrough Therapy Designation from the FDA Industry Context - **Condition**: Post-bariatric hypoglycemia (PBH) is a serious metabolic condition that occurs in patients after bariatric surgery, characterized by severe hypoglycemic episodes - **Prevalence**: Approximately 400,000 individuals in the U.S. experience clinically important hypoglycemia after bariatric surgery, with around 166,000 requiring medical management [42][43] Key Points and Arguments 1. **Patient Experience**: A patient shared her struggles with PBH, highlighting the debilitating nature of the condition, which severely limits daily activities and quality of life [8][9][15][16] 2. **Medical Need**: There is a significant unmet medical need for effective treatments for PBH, as current dietary modifications and off-label medications often fail to provide relief [39][40] 3. **Mechanism of PBH**: PBH is caused by an exaggerated GLP-1 response post-surgery, leading to hyperinsulinism and subsequent hypoglycemia. This condition can develop 2-3 years after surgery, but may occur anytime from 2 to 20 years post-operation [19][41] 4. **Current Treatments**: Existing treatments are limited and often ineffective, with no FDA-approved medications specifically for PBH. Dietary modifications and off-label medications have high failure rates and significant side effects [22][39] 5. **Avexatide's Mechanism**: Avexatide targets the GLP-1 receptor to normalize insulin secretion and stabilize glucose levels, addressing the underlying pathophysiology of PBH [49][50] 6. **Clinical Trials**: The Phase II and Phase IIb studies showed that Avexatide significantly reduced the frequency of Level II and Level III hypoglycemic events by over 50% [57][72] 7. **FDA Breakthrough Therapy Designation**: Avexatide is the only drug in development for PBH to receive this designation, indicating its potential to address a serious condition with high unmet medical need [57] 8. **LUCIDITY Phase III Trial**: The ongoing Phase III trial will evaluate the efficacy of Avexatide at a dose of 90 mg once daily, focusing on reducing hypoglycemic events [78][81] Additional Important Content - **Patient Education**: There is a critical need for increased awareness and education about PBH among healthcare providers and patients, as many patients are misdiagnosed or experience delays in receiving appropriate care [38][41] - **Quality of Life Impact**: The unpredictable nature of hypoglycemic events leads to social isolation and significant emotional distress for patients, emphasizing the need for effective treatment options [66][68] - **Future Outlook**: The company is actively recruiting for the LUCIDITY trial, with data expected in the first half of 2026, aiming to bring a much-needed therapy to market for PBH patients [85][86]

Core Viewpoint - Bragar Eagel & Squire, P.C. is investigating potential claims against Amylyx Pharmaceuticals, Inc. due to a class action complaint alleging breaches of fiduciary duties by the company's board of directors during the specified class period [1][2]. Summary by Relevant Sections Allegations of Misconduct - The complaint asserts that the Individual Defendants made false and misleading statements regarding the Relyvrio commercial launch, claiming significant demand that was not sustainable [2]. - It is alleged that the initial surge in demand for Relyvrio was temporary and had already subsided within months of its launch, undermining growth potential among newly diagnosed ALS patients [2]. - The complaint highlights that there were high, undisclosed discontinuation rates for Relyvrio, which negatively impacted the drug's commercial viability and inflated the perceived potential for acquiring new patients [2]. Legal Representation - Bragar Eagel & Squire, P.C. is representing long-term stockholders of Amylyx and is open to inquiries regarding the claims and rights of investors [3]. Firm Background - Bragar Eagel & Squire, P.C. is a recognized law firm specializing in representing individual and institutional investors in various complex litigations across state and federal courts [4].

Core Viewpoint - Guggenheim Partners has initiated coverage on Amylyx Pharmaceuticals, highlighting the potential of post-bariatric hypoglycemia (PBH) as a significant market opportunity for the company [1][2]. Company Overview - Amylyx Pharmaceuticals is positioned as a first-to-market player in the PBH space, which is estimated to be a $2 billion+ market opportunity [2]. - The company's lead drug, avexitide, is advancing towards pivotal Phase 3 results in the LUCIDITY study, expected to read out in the first half of 2026 [3]. Market Potential - Guggenheim estimates that there are 50,000 to 100,000 individuals in the U.S. suffering from moderate to severe uncontrolled symptoms of PBH [4]. - The global market for GLP-1 antagonists to treat PBH could exceed $2 billion by 2035, with projections of $1.8 billion in the U.S. and $410 million internationally, assuming a 60% peak market share among patients with severe PBH [4]. Competitive Landscape - The main competitor in the PBH market is MBX Biosciences, which is developing a once-weekly GLP-1 antagonist called MBX 1416 [5]. - Analyst estimates suggest that a once-weekly competitor could launch by 2031, while Amylyx's life cycle management strategy may enhance its market position [5]. Stock Performance - As of the last check, Amylyx Pharmaceuticals' stock (AMLX) has increased by 24.5%, reaching $6.25 [5].

Study Overview - The HELIOS trial evaluated the safety, tolerability, and efficacy of PB&TURSO in Wolfram Syndrome patients [1, 15] - The study was an open-label Phase 2 trial [1, 16] - The trial included 12 enrolled participants, with 11 in the Per Protocol (PP) population who had genetically confirmed Wolfram Syndrome [16, 26] Efficacy Results - At Week 24, the Per Protocol group (N=11) showed improvement in C-peptide response to Mixed Meal Tolerance Test (MMTT) compared to screening [30, 31] - At Week 24, C-Peptide AUC increased by 16.8 min*ng/mL in ITT population and 20.2 min*ng/mL in Per Protocol population [34] - 10 out of 11 participants in the Per Protocol group maintained or decreased time to peak C-peptide at Week 24 compared to screening [37, 38] - HbA1c levels showed a mean change from baseline of -0.09% in the ITT group and -0.16% in the Per Protocol group at Week 24 [40, 45] - 9 of 11 Per Protocol participants demonstrated reduced or unchanged HbA1c from Screening to the latest available time point [42] - 8 of 11 participants in the Per Protocol group demonstrated improved or stable visual acuity in their best eye from Screening to the latest available timepoint [51] Safety and Tolerability - PB&TURSO was generally well-tolerated [53] - 91.7% of participants (11 out of 12) experienced at least one Treatment Emergent Adverse Event (TEAE) [53] - Diarrhea was the most common TEAE, affecting 50% of participants, but all cases were of mild severity [55]

Summary of Amylyx Pharmaceuticals (AMLX) FY Conference June 10, 2025 Company Overview - Amylyx Pharmaceuticals has undergone significant changes in the past year, focusing on three different assets across four ongoing clinical trials [1][2] Key Assets and Clinical Trials 1. **Avexatide** - A GLP-1 receptor antagonist aimed at treating post-bariatric hypoglycemia (PBH) - Currently in a pivotal study with enrollment completion targeted by the end of 2025 and data expected in the first half of 2026 [3][6] - Previous studies supported FDA Breakthrough Therapy Designation, with five prior studies demonstrating its efficacy [3][19] 2. **AMX-35** - A small molecule targeting endoplasmic reticulum (ER) stress and mitochondrial dysfunction - Trials ongoing for Wolfram's syndrome and progressive supranuclear palsy (PSP) - Positive data from the HELIOS trial for Wolfram's syndrome was recently announced, showing increased C-peptide levels, indicating improved insulin production [4][5] 3. **AMX-114** - An antisense oligonucleotide targeting calpain-2 for ALS and other neurodegenerative diseases - A dose-escalating placebo-controlled study has commenced, with safety and early biomarker data expected by the end of 2025 [5][6] Market Insights and Patient Population - The estimated addressable patient population for PBH is around 160,000, based on studies indicating that approximately 8% of bariatric surgery patients develop PBH [10][11] - Symptoms of PBH can severely disrupt patients' lives, leading to dizziness, loss of consciousness, and confusion, making effective treatment critical [9][10] - Adult endocrinologists primarily manage PBH symptoms, with many clinics reporting a significant number of patients under care [13][14] Clinical Data and Efficacy - Phase II trials of Avexatide showed a 53% reduction in level II hypoglycemic events and a 66% reduction in level III events, both statistically significant [23][24] - The Phase III trial is designed to maintain consistency with Phase II, focusing on patients experiencing at least one hypoglycemic event per week [25][28] Regulatory and Commercial Considerations - The FDA's Breakthrough Therapy Designation facilitates more frequent interactions and support for the pivotal study [35] - Pricing strategies will be informed by market research and comparisons with other rare endocrine launches, with a focus on demonstrating the unmet need and efficacy to payers [37][39] Intellectual Property and Collaborations - Avexatide has a base case patent extending to 2037, with additional method of use and formulation patents [41] - A collaboration with Gubra aims to develop a long-acting GLP-1 receptor antagonist, with potential applications in other hypoglycemia-related conditions [43][44] Future Directions - The company is exploring additional indications for Avexatide, including congenital hyperinsulinism and hypoglycemia resulting from other surgeries [50][51] - Ongoing discussions with regulators will focus on the design of the Phase III trial for Wolfram's syndrome, aiming to establish key endpoints and study duration [61][64] Financial Position - Amylyx ended the last quarter with $204 million in cash, providing a runway through the end of 2026, covering pivotal studies and ongoing programs [74][75]

Summary of Amylyx Pharmaceuticals (AMLX) Conference Call Company Overview - **Company**: Amylyx Pharmaceuticals (AMLX) - **Focus**: Developing therapies for rare diseases, particularly post bariatric hypoglycemia (PBH) and other neurodegenerative conditions [5][6] Key Points on Lead Asset: Avexatide - **Indication**: Avexatide is a competitive inhibitor of GLP-1, targeting PBH, a rare condition following bariatric surgery characterized by severe hypoglycemic events [5][6] - **Clinical Trials**: - Five past trials demonstrated significant benefits, including a 53% reduction in level two hypoglycemic events and a 66% reduction in level three events [6][21] - Phase III study recruitment expected to complete by the end of 2025, with data anticipated in the first half of 2026 [7][49] - **Market Opportunity**: - PBH affects an estimated 160,000 people in the U.S., with the number expected to rise due to increasing bariatric surgeries (approximately 270,000 annually) [12][13] - Current treatment options are limited, primarily involving medical nutrition therapy [14][15] Regulatory Designation and Pricing - **Breakthrough Therapy Designation**: Received based on prior trial data, facilitating closer collaboration with the FDA [7][20] - **Pricing Strategy**: Pricing has not been finalized; however, the company is considering the orphan drug designation and recent market entries in rare endocrine disorders as benchmarks [17][18] Other Assets and Trials - **AMX35**: - Studied in Wolfram syndrome and progressive supranuclear palsy (PSP) [7][58] - Recent data from Wolfram syndrome trials showed stabilization and improvement in various outcomes over 48 weeks [60][61] - **AMX-114**: An antisense oligonucleotide targeting calpain two, currently in a clinical trial for ALS, with data expected by the end of the year [9] Competitive Landscape - **Current Competition**: No known drugs have shown efficacy in treating PBH, positioning Avexatide uniquely in the market [54] - **GLP-1 Agonists**: While there are concerns about competition from GLP-1 agonists, the company believes that the target population for bariatric surgery is distinct and will continue to grow [55][56] Future Milestones - **Phase III Trial**: Expected to provide pivotal data for FDA approval, with a focus on maintaining consistency with Phase II trial parameters [36][62] - **Market Access**: Preparations for market access strategies are underway, emphasizing the importance of ensuring patient access post-approval [52] Conclusion - Amylyx Pharmaceuticals is advancing its lead asset Avexatide through critical clinical trials aimed at addressing significant unmet needs in rare diseases, particularly PBH. The company is optimistic about its market potential and regulatory pathway, with ongoing efforts to ensure successful trial outcomes and market access strategies.

Financial Data and Key Metrics Changes - The company is actively recruiting for a pivotal study of Avexatide, with top-line results expected in the first half of next year, potentially supporting commercialization in 2027 [3][18] - The company has cash reserves expected to last through the end of 2026, allowing it to fund ongoing clinical trials and development programs [36] Business Line Data and Key Metrics Changes - Avexatide, the lead asset, has shown significant reductions in level two and level three hypoglycemic events, with a 53% reduction in level two events and a 66% reduction in level three events in previous trials [12] - AMX-35 has demonstrated stabilization or improvement in diabetic outcomes in patients with Wolfram syndrome, with continued improvements observed at the 48-week mark [24][25] Market Data and Key Metrics Changes - There are an estimated 160,000 people in the United States suffering from post-bariatric hypoglycemia (PBH), a condition with no FDA-approved treatments currently available [10][18] - Wolfram syndrome affects approximately 3,000 individuals in the U.S., while progressive supranuclear palsy (PSP) is estimated to impact around 23,000 people [21][29] Company Strategy and Development Direction - The company focuses on diseases with substantial unmet needs, aiming to develop therapies that can effectively address these conditions [38] - The strategic direction includes advancing multiple clinical trials, with a strong emphasis on obtaining positive data to support further investment in promising programs [31][37] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential of Avexatide to meet a significant unmet need in PBH, highlighting the growing population affected by this condition [17][18] - The company is preparing for a Phase III program for AMX-35, with plans to share the design before the end of the year [37] Other Important Information - The company has received FDA breakthrough therapy designation for Avexatide, which underscores the potential significance of this treatment in the market [3] - The company is also working on AMX-114, an antisense oligonucleotide targeting CALPIN-two for ALS, with early cohort data expected by the end of the year [36] Q&A Session Summary Question: What are the upcoming milestones for Avexatide? - The company expects to complete recruitment for the pivotal study by the end of the year and have data in the first half of next year, which could support filing for approval in PBH [37] Question: How is the company addressing the needs in Wolfram syndrome? - The company has reported positive 48-week data for AMX-35 in Wolfram syndrome and is working on finalizing the Phase III design [37] Question: What is the status of the PSP program? - Recruitment for the Phase 2b study in PSP has been completed, with data expected in Q3 of this year [30][37]