Amylyx(AMLX) - 2025 Q1 - Quarterly Results

Company Overview and First Quarter Highlights The company reports Q1 2025 results, highlighting clinical pipeline progress and an extended cash runway through 2026 First Quarter 2025 Business and Financial Highlights Amylyx Pharmaceuticals reported Q1 2025 results, highlighting significant clinical pipeline progress and a strengthened financial position - Pivotal Phase 3 LUCIDITY trial of avexitide for post-bariatric hypoglycemia (PBH) is underway; completion of recruitment expected in 2025, with topline data anticipated in the first half of 2026[6] - Week 48 data from the ongoing Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome to be presented from May 10-13[6] - Phase 1 LUMINA trial of AMX0114 for amyotrophic lateral sclerosis (ALS) is underway; early cohort data expected in 2025[6] - Cash runway expected through the end of 2026[6] Cash Position (as of March 31, 2025) | Metric | Amount (Millions) | | :----- | :---------------- | | Cash, cash equivalents, and marketable securities | $204.1 | Co-CEOs' Strategic Outlook The Co-CEOs expressed confidence in pipeline progress and achieving goals with the extended cash runway through 2026 - Continued progress across the pipeline, including strong clinical execution of the pivotal Phase 3 LUCIDITY trial of avexitide in post-bariatric hypoglycemia[4] - Completion of enrollment for LUCIDITY expected in 2025 and topline data in the first half of 2026[4] - First patient dosed in Phase 1 LUMINA trial of AMX0114 in ALS, with early cohort data expected later this year[4] - Anticipated cash runway through the end of 2026, supporting confidence in achieving current goals and upcoming milestones[4] Pipeline and Clinical Development Updates The company details advancements in its key clinical programs for avexitide, AMX0114, and AMX0035 across various indications Avexitide Program (Post-Bariatric Hypoglycemia - PBH) The avexitide program for PBH is advancing with the pivotal Phase 3 LUCIDITY trial, which has begun dosing participants - Avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist[16] - FDA has granted avexitide Breakthrough Therapy Designation for both PBH and congenital hyperinsulinism (HI), Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for hyperinsulinemic hypoglycemia[16] - Post-bariatric hypoglycemia (PBH) affects approximately 8% of people in the U.S. (around 160,000 people) who underwent bariatric surgery, with no approved therapies[17] LUCIDITY Trial (Phase 3) - Pivotal Phase 3 LUCIDITY clinical trial of avexitide for PBH has begun dosing, with the first participant dosed in April 2025[6][7] - The trial is an approximately 75-participant, randomized, double-blind, placebo-controlled study evaluating efficacy and safety of avexitide in participants with PBH following Roux-en-Y gastric bypass (RYGB) surgery at approximately 20 sites in the U.S[7][18] - Primary efficacy objective: reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16, as agreed upon with the FDA[12][19] - Completion of enrollment expected in 2025, with topline data anticipated in the first half of 2026, and potential commercial launch in 2027 if approved[6][12] Other Avexitide Data Presentations - Amylyx plans to present additional data from the Phase 2 PREVENT and Phase 2b trials of avexitide in PBH at the Endocrine Society's annual meeting (ENDO) 2025 on July 12-15 in San Francisco, CA[12] AMX0114 Program (Amyotrophic Lateral Sclerosis - ALS) Amylyx has initiated the Phase 1 LUMINA trial for AMX0114, an investigational antisense oligonucleotide targeting calpain-2 for people living with ALS LUMINA Trial (Phase 1) - Phase 1 LUMINA clinical trial of AMX0114, an investigational antisense oligonucleotide (ASO) targeting knockdown of calpain-2, for people living with ALS has begun dosing, with the first participant dosed in April 2025[6][7] - The multinational, randomized, double-blind, placebo-controlled, multiple ascending dose trial is designed to evaluate the safety and biological activity of AMX0114, and will assess ALS biomarkers, including changes in neurofilament light (NfL) levels[7] - Early cohort data from the LUMINA trial are expected in 2025[6][12] AMX0035 Program (Wolfram Syndrome & Progressive Supranuclear Palsy - PSP) Amylyx continues to advance AMX0035 for Wolfram syndrome and PSP, with key data readouts expected in 2025 HELIOS Trial (Wolfram Syndrome - Phase 2) - Data through Week 48 from the ongoing Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome to be presented at the Joint Congress of the European Society for Pediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) from May 10-13[6][12] - Data from participants at Week 48 and ongoing discussions with the FDA will inform the design of a Phase 3 trial of AMX0035 in Wolfram syndrome[12] ORION Trial (Progressive Supranuclear Palsy - Phase 2b/3) - Unblinded analysis of the Phase 2b portion of the Phase 2b/3 ORION trial evaluating AMX0035 for progressive supranuclear palsy (PSP) expected in the third quarter of 2025[12] - The Phase 2b portion was fully enrolled in January 2025 with a total of 139 participants randomized[12] - Efficacy and safety Phase 2b data from an unblinded analysis will be used to inform a go/no-go decision on the Phase 3 portion of the trial[12] Financial Performance and Outlook The company reports a reduced net loss for Q1 2025, a strengthened cash position, and an extended operational runway First Quarter 2025 Financial Results Amylyx reported a significantly reduced net loss for Q1 2025, driven by lower operating expenses following the discontinuation of RELYVRIO®/ALBRIOZA™ Key Financial Highlights (Three Months Ended March 31) | Metric | 2025 (in thousands) | 2024 (in thousands) | Change (YoY) | | :--------------------------------------- | :------------------ | :------------------ | :----------- | | Product revenue, net | $— | $88,643 | -100% | | Total operating expenses | $37,803 | $210,773 | -82.1% | | Loss from operations | $(37,803) | $(122,130) | -70.0% | Research and Development (R&D) Expenses - Decrease primarily due to a decrease in spending on AMX0035 for the treatment of ALS, payroll and personnel-related costs, and a decrease in preclinical development activities[10] R&D Expenses (Three Months Ended March 31) | Period | Amount (in thousands) | Change (YoY) | | :----- | :-------------------- | :----------- | | Q1 2025 | $22,119 | -39.6% | | Q1 2024 | $36,608 | | Stock-Based Compensation Expense (R&D) | Period | Amount (in millions) | | :----- | :------------------- | | Q1 2025 | $1.8 | | Q1 2024 | $2.7 | Selling, General and Administrative (SG&A) Expenses - Decrease primarily due to a decrease in payroll and personnel-related costs and a decrease in consulting, professional services, and other expenses[11] SG&A Expenses (Three Months Ended March 31) | Period | Amount (in thousands) | Change (YoY) | | :----- | :-------------------- | :----------- | | Q1 2025 | $15,684 | -72.8% | | Q1 2024 | $57,759 | | Stock-Based Compensation Expense (SG&A) | Period | Amount (in millions) | | :----- | :------------------- | | Q1 2025 | $5.0 | | Q1 2024 | $7.2 | Net Loss Net Loss (Three Months Ended March 31) | Period | Net Loss (in thousands) | Net Loss Per Share | | :----- | :---------------------- | :----------------- | | Q1 2025 | $(35,907) | $(0.42) | | Q1 2024 | $(118,793) | $(1.75) | Cash Position and Runway Amylyx's cash position increased to $204.1 million, which is expected to fund operations through the end of 2026 - Based on current operating plans, Amylyx expects its cash runway to be through the end of 2026[6][13] - The cash position reflects payments of $6.0 million in Q1 2025 related to product rebates and settlement of purchase commitments for AMX0035, established prior to the voluntary discontinuation of RELYVRIO®/ALBRIOZA™ sales in April 2024[13] - Residual cash obligations related to the discontinuation of RELYVRIO/ALBRIOZA are $3.1 million, expected to be paid through 2025[13] Cash, Cash Equivalents, and Marketable Securities | Date | Amount (in thousands) | | :--- | :-------------------- | | March 31, 2025 | $204,068 | | December 31, 2024 | $176,501 | Public Offering and Cash Runway Extension A January 2025 public offering generated net proceeds of approximately $65.5 million, extending the company's cash runway - Amylyx closed its underwritten public offering of 19.7 million shares of its common stock in January 2025[8] - The offering extended the company's expected cash runway through the end of 2026[8] Public Offering Details | Metric | Value | | :----- | :---- | | Net proceeds to Amylyx | ~$65.5 million | Additional Company Information This section provides details on investor communications, the company's mission, and important forward-looking statements Investor Conference Call and Available Information Amylyx hosted a conference call on May 8, 2025, and directs investors to its corporate websites for periodic disclosures - Amylyx' management team hosted a conference call on May 8, 2025, at 8:00 a.m. ET to discuss financial results and provide a business update[14] - A live audio webcast of the call is available under 'Events and Presentations' on the Investor section of the Company's website, with an archived replay available for 90 days[14] - The company periodically provides information for investors on its corporate and investor relations websites, including press releases, financial performance, corporate governance, and SEC filings, and intends to use its website for disclosing material non-public information[15] About Amylyx Pharmaceuticals Amylyx Pharmaceuticals is dedicated to treating diseases with high unmet needs, focusing on three investigational therapies - Amylyx's mission is to usher in a new era of treating diseases with high unmet needs[20] - The company is currently focused on three investigational therapies across several neurodegenerative and endocrine diseases[20] Forward-Looking Statements The report contains forward-looking statements regarding clinical programs, financial performance, and strategy, which are subject to risks and uncertainties - Statements in the press release regarding matters that are not historical facts are 'forward-looking statements' subject to risks and uncertainties[21] - Risks include the success, cost, and timing of program development activities; ability to execute regulatory development plans; ability to fund operations; and the impact of global macroeconomic uncertainty, geopolitical instability, and public health events[21] - Amylyx undertakes no obligation to update such statements to reflect events or circumstances after the date they were made, except as required by law[21] Condensed Consolidated Financial Statements This section presents the unaudited condensed consolidated balance sheets and statements of operations as of March 31, 2025 Condensed Consolidated Balance Sheets The balance sheet shows increased total assets and stockholders' equity, driven primarily by a higher cash position as of March 31, 2025 Condensed Consolidated Balance Sheets (Unaudited, in thousands) | Item | March 31, 2025 | December 31, 2024 | | :-------------------------------- | :------------- | :---------------- | | Assets | | | | Cash, cash equivalents and marketable securities | $204,068 | $176,501 | | Accounts receivable, net | $300 | $447 | | Prepaid expenses and other current assets | $11,776 | $12,484 | | Other assets | $3,532 | $4,202 | | Total assets | $219,676 | $193,634 | | Liabilities and Stockholders' Equity | | | | Accounts payable and accrued expenses | $16,877 | $26,888 | | Other liabilities | $1,382 | $1,981 | | Total liabilities | $18,259 | $28,869 | | Stockholders' equity | $201,417 | $164,765 | | Total liabilities and stockholders' equity | $219,676 | $193,634 | Condensed Consolidated Statements of Operations The statement of operations reflects zero product revenue and a substantially reduced net loss for Q1 2025 compared to the prior year Condensed Consolidated Statements of Operations (Unaudited, in thousands, except share and per share data) | Item | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--------------------------------------- | :-------------------------------- | :-------------------------------- | | Product revenue, net | $— | $88,643 | | Operating expenses: | | | | Cost of sales | $— | $5,945 | | Cost of sales - inventory impairment and loss on firm purchase commitments | $— | $110,461 | | Research and development | $22,119 | $36,608 | | Selling, general and administrative | $15,684 | $57,759 | | Total operating expenses | $37,803 | $210,773 | | Loss from operations | $(37,803) | $(122,130) | | Other income, net | $1,896 | $3,579 | | Loss before income taxes | $(35,907) | $(118,551) | | Provision for income taxes | $— | $242 | | Net loss | $(35,907) | $(118,793) | | Net loss per share — basic and diluted | $(0.42) | $(1.75) | | Weighted-average shares used in computing net loss per share — basic and diluted | 85,697,108 | 67,854,356 |