Amylyx(AMLX) - 2025 Q2 - Quarterly Results

Q2 2025 Business and Financial Overview Amylyx Pharmaceuticals highlights clinical pipeline advancements and stable financial performance in Q2 2025 Management Commentary 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] - 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] Second Quarter and Recent Updates 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] - 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] - 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] - Received FDA Fast Track designation for AMX0114, an investigational treatment for amyotrophic lateral sclerosis (ALS), in June 2025[7] Upcoming Expected Milestones 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] - 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] - 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] - AMX0114 (LUMINA Trial for ALS): Early cohort data from the Phase 1 trial, evaluating safety and biological activity, is expected in 2025[10] Q2 2025 Financial Performance The company significantly reduced its net loss in Q2 2025, driven by lower operating expenses and a strong cash position Financial Results Summary 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] Research and Development (R&D) Expenses 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] Selling, General, and Administrative (SG&A) Expenses 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] Net Loss 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 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] Clinical Pipeline and Programs Amylyx is advancing multiple clinical programs, including avexitide for PBH, AMX0035 for Wolfram syndrome and PSP, and AMX0114 for ALS Avexitide Program (for PBH) Avexitide is an investigational GLP-1 receptor antagonist for post-bariatric hypoglycemia (PBH), currently in a pivotal Phase 3 trial About Avexitide 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] - The FDA has granted avexitide Breakthrough Therapy Designation for both post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI)[15] About Post-Bariatric Hypoglycemia (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] - The condition can cause debilitating neuroglycopenic symptoms like impaired cognition, loss of consciousness, and seizures. There are no approved therapies for PBH[16] About the LUCIDITY Trial 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][18] - 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] Other Key Programs Amylyx is advancing AMX0035 for Wolfram syndrome and PSP, and AMX0114 for ALS, with several upcoming data readouts AMX0035 (for Wolfram Syndrome & PSP) 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][10] - 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] AMX0114 (for ALS) 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] - The Phase 1 LUMINA trial is a multiple ascending dose study in approximately 48 participants, with early cohort data expected in 2025[10] Financial Statements Detailed financial statements for Q2 2025 show balance sheet and income statement performance Condensed Consolidated Balance Sheets 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 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) |