Core Insights - Amylyx Pharmaceuticals announced topline results from the PHOENIX trial, which did not meet its primary endpoint of statistical significance in ALSFRS-R total score change at Week 48 (p=0.667) [1][5] - The company plans to engage with regulatory authorities and the ALS community to discuss the trial results and may consider withdrawing RELYVRIO/ALBRIOZA from the market [2][3] - AMX0035 was well-tolerated in the trial, with no new safety signals reported, reinforcing its favorable safety profile [5] PHOENIX Trial Results - The PHOENIX study enrolled 664 adults with ALS, randomized to receive either AMX0035 or placebo, alongside standard-of-care treatments [4][9] - No significant differences were observed in secondary endpoints, and the treatment did not show efficacy in participants who met CENTAUR trial criteria [5] - European participants completing the trial have the option to enroll in an ongoing open-label extension phase [5][9] Future Plans and Community Engagement - The company will share the PHOENIX results with the ALS community and regulatory authorities over the next eight weeks [2][3] - Amylyx remains committed to ALS research and is exploring AMX0035's potential in other neurodegenerative diseases, including Wolfram syndrome and progressive supranuclear palsy [3][6] Ongoing Studies - The ORION trial of AMX0035 in progressive supranuclear palsy is ongoing, with topline results expected in 2025 or 2026 [7] - Preliminary data from the HELIOS trial in Wolfram syndrome is anticipated in the second quarter of 2024 [7] About AMX0035 - AMX0035 is a fixed-dose combination of sodium phenylbutyrate and taurursodiol, approved as RELYVRIO in the U.S. and ALBRIOZA in Canada for ALS treatment [19][20] - The drug targets neurodegenerative pathways by mitigating endoplasmic reticulum stress and mitochondrial dysfunction [6][19]

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced that it has granted stock options and restricted stock units (“RSUs”) to Linda Arsenault, the Company’s newly appointed Chief Human Resources Officer. The Compensation Committee of Amylyx’ Board of Directors approved the grant, effective February 20, 2024, to Ms. Arsenault of non-qualified stock option awards to purchase up to 101,400 shares of the Company’s common stock and 67,600 RSUs ...

Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) Q4 2023 Earnings Call Transcript February 22, 2024 8:00 AM ET Company Participants Lindsey Allen - Head, IR & Communications Justin Klee - Co-CEO Jim Frates - CFO Camille Bedrosian - CMO Josh Cohen - Co-CEO Conference Call Participants Charlie Young - Bank of America Neena Bitritto-Garg - Deutsche Bank Canada Corinne Jenkins - Goldman Sachs Michael DiFiore - Evercore ISI Graig Suvannavejh - Mizuho Madhu Yennawar - Leerink Partners Ananda Ghosh - HC Wainwright Opera ...

Amylyx Pharmaceuticals, Inc. (AMLX) came out with quarterly earnings of $0.07 per share, missing the Zacks Consensus Estimate of $0.20 per share. This compares to loss of $0.65 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -65%. A quarter ago, it was expected that this company would post earnings of $0.42 per share when it actually produced earnings of $0.30, delivering a surprise of -28.57%.Over the last four quarters, the ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Securities registered pursuant to Section 12(b) of the Act: FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 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 ...

[Executive Summary & Financial Highlights](index=1&type=section&id=Executive%20Summary%20%26%20Financial%20Highlights) Amylyx reported **$380.8 million** full-year 2023 revenue and **$49.3 million** net income, ending with **$371.4 million** cash Q4 & Full Year 2023 Key Financials | Metric | Full Year 2023 | Fourth Quarter 2023 | | :--- | :--- | :--- | | Net Product Revenue | $380.8 million | $108.4 million | | Net Income | $49.3 million | $4.7 million | | Cash, Cash Equivalents and Short-term Investments | $371.4 million | $371.4 million | [Management Commentary](index=1&type=section&id=Management%20Commentary) Management highlighted successful 2023 RELYVRIO/ALBRIOZA delivery, focusing on international expansion and advancing key pipeline trials - In 2023, the company successfully delivered RELYVRIO/ALBRIOZA to thousands of people with ALS in North America and plans to continue expanding its U.S. and international presence in 2024[2](index=2&type=chunk) - Topline data from the Phase 3 PHOENIX trial, which will provide additional efficacy and safety data for AMX0035 in a larger ALS population, is expected during or before the second quarter of 2024[2](index=2&type=chunk) - Pipeline advancements include the ongoing Phase 3 ORION trial in Progressive Supranuclear Palsy (PSP), expected data from the Phase 2 HELIOS trial in Wolfram syndrome in 2024, and the planned clinical entry of antisense oligonucleotide AMX0114[2](index=2&type=chunk) [Business Highlights](index=1&type=section&id=Business%20Highlights) The company achieved continued commercial progress with RELYVRIO®/ALBRIOZA™, advanced key clinical trials, and strengthened its leadership team [Commercial Progress](index=1&type=section&id=Commercial%20Progress) Q4 2023 net product revenue reached **$108.4 million**, with growth driven by Canada and international named patient sales Quarterly Net Product Revenue Growth | Period | Net Product Revenue | | :--- | :--- | | Q4 2023 | $108.4 million | | Q3 2023 | $102.7 million | - Revenue growth in Q4 2023 relative to Q3 2023 was importantly sourced from Canada and international named patient sales programs[3](index=3&type=chunk) [Clinical Pipeline and R&D](index=1&type=section&id=Clinical%20Pipeline%20and%20R%26D) Amylyx advanced its clinical pipeline, initiating Phase 3 ORION for PSP, completing Phase 2 HELIOS enrollment, and progressing AMX0114 - The first patient was dosed in the Global Phase 3 ORION Study of AMX0035 in Progressive Supranuclear Palsy (PSP) in December 2023, with topline results anticipated in 2025 or 2026[3](index=3&type=chunk) - Enrollment was completed in the Phase 2 HELIOS study of AMX0035 for Wolfram syndrome, with data expected in 2024[3](index=3&type=chunk) - The investigational antisense oligonucleotide AMX0114, targeting calpain-2, is advancing through IND-enabling studies with the goal of entering the clinic during 2024[5](index=5&type=chunk) - Published post-hoc analyses of the CENTAUR trial demonstrated a 10.4-month longer median overall survival for the AMX0035 group compared to a historical control group and showed a significant reduction in key plasma neuroinflammatory biomarkers[5](index=5&type=chunk) [Corporate Developments](index=1&type=section&id=Corporate%20Developments) The company strengthened its leadership team with key appointments including Chief Medical Officer and Head of U.S. Commercial Markets - **Chief Medical Officer:** Camille L. Bedrosian, MD, was appointed in November 2023[3](index=3&type=chunk)[5](index=5&type=chunk) - **Chief Human Resources Officer:** Linda Arsenault joined in 2024[3](index=3&type=chunk)[5](index=5&type=chunk) - **General Manager and Head of U.S. Commercial Markets:** Dan Monahan joined in 2024[3](index=3&type=chunk)[5](index=5&type=chunk) [Detailed Financial Results (Q4 & Full Year 2023)](index=2&type=section&id=Detailed%20Financial%20Results%20%28Q4%20%26%20Full%20Year%202023%29) Amylyx achieved full-year net product revenue of **$380.8 million** and **$49.3 million** net income in 2023, a significant turnaround [Net Product Revenue](index=2&type=section&id=Net%20Product%20Revenue) Net product revenue significantly increased to **$108.4 million** in Q4 and **$380.8 million** for the full year, driven by U.S. RELYVRIO sales Net Product Revenue Comparison (in millions) | Period | 2023 | 2022 | | :--- | :--- | :--- | | **Fourth Quarter** | $108.4 | $21.9 | | **Full Year** | $380.8 | $22.2 | - The increase in revenue was primarily driven by units of RELYVRIO sold in the U.S. following its regulatory approval in late September 2022[6](index=6&type=chunk) [Operating Expenses](index=2&type=section&id=Operating%20Expenses) Full-year 2023 operating expenses significantly increased across all categories, driven by commercialization, personnel, and the ORION trial Operating Expenses Comparison (in millions) | Expense Category | Full Year 2023 | Full Year 2022 | Q4 2023 | Q4 2022 | | :--- | :--- | :--- | :--- | :--- | | Cost of Sales | $25.4 | $3.0 | $9.4 | $2.8 | | R&D Expenses | $128.2 | $93.5 | $44.9 | $22.8 | | SG&A Expenses | $188.4 | $127.1 | $52.2 | $40.8 | - The increase in R&D expenses was primarily driven by higher personnel-related costs, spending on the Phase 3 ORION trial in PSP, and preclinical development activities[7](index=7&type=chunk) - The increase in SG&A expenses was primarily driven by higher personnel-related costs to support the commercial launch and operations as a public company[8](index=8&type=chunk) [Profitability and Cash Position](index=2&type=section&id=Profitability%20and%20Cash%20Position) The company achieved full-year net income of **$49.3 million** in 2023, a significant turnaround, with a strong cash position of **$371.4 million** Net Income (Loss) and EPS Comparison | Metric | Full Year 2023 | Full Year 2022 | Q4 2023 | Q4 2022 | | :--- | :--- | :--- | :--- | :--- | | **Net Income (Loss)** | $49.3 M | ($198.4 M) | $4.7 M | ($42.7 M) | | **Diluted EPS** | $0.70 | ($3.39) | $0.07 | ($0.65) | - Cash, cash equivalents, and short-term investments were **$371.4 million** at December 31, 2023[9](index=9&type=chunk) [Financial Statements](index=7&type=section&id=Financial%20Statements) This section presents unaudited Condensed Consolidated Balance Sheets and Statements of Operations for Q4 and full years 2023 and 2022 [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of December 31, 2023, total assets grew to **$517.5 million**, driven by cash, receivables, and inventories, with increased liabilities and equity Condensed Consolidated Balance Sheets (in thousands) | Account | Dec 31, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash, cash equivalents and short-term investments | $371,362 | $346,945 | | Accounts receivable, net | $40,050 | $15,306 | | Inventories | $83,280 | $9,769 | | **Total assets** | **$517,454** | **$391,453** | | **Liabilities and Stockholders' Equity** | | | | Accounts payable and accrued expenses | $79,785 | $44,569 | | **Total liabilities** | **$84,022** | **$50,846** | | **Stockholders' equity** | **$433,432** | **$340,607** | | **Total liabilities and stockholders' equity** | **$517,454** | **$391,453** | [Condensed Consolidated Statements of Operations](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) The 2023 statement of operations shows net product revenue of **$380.8 million** and total operating expenses of **$342.0 million**, yielding **$49.3 million** net income Condensed Consolidated Statements of Operations (in thousands) | Account | Full Year 2023 | Full Year 2022 | Q4 2023 | Q4 2022 | | :--- | :--- | :--- | :--- | :--- | | Product revenue, net | $380,786 | $22,230 | $108,449 | $21,885 | | Total operating expenses | $341,984 | $223,571 | $106,515 | $66,478 | | Income (loss) from operations | $38,802 | ($201,341) | $1,934 | ($44,593) | | **Net income (loss)** | **$49,271** | **($198,375)** | **$4,731** | **($42,704)** | | Diluted net income (loss) per share | $0.70 | ($3.39) | $0.07 | ($0.65) | [Product Information: RELYVRIO®/ALBRIOZA™](index=4&type=section&id=Product%20Information%3A%20RELYVRIO%C2%AE%2FALBRIOZA%E2%84%A2) RELYVRIO®/ALBRIOZA™ is an oral, fixed-dose combination for ALS in the U.S. and Canada, with warnings for specific patient disorders, high sodium, and common GI adverse reactions - RELYVRIO® is an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol approved for ALS in the U.S. and Canada (as ALBRIOZA™)[13](index=13&type=chunk) - Warnings include risks for patients with disorders of enterohepatic circulation, pancreatic, or intestinal issues due to the taurursodiol (a bile acid) component[14](index=14&type=chunk) - The product has a high salt content (928 mg of sodium in a daily maintenance dose), requiring caution in patients with heart failure, hypertension, or renal impairment[15](index=15&type=chunk) - The most common adverse reactions (at least 15% and 5% greater than placebo) were diarrhea, abdominal pain, nausea, and upper respiratory tract infection[16](index=16&type=chunk)

Core Viewpoint - A lawsuit has been filed against Amylyx Pharmaceuticals for allegedly providing false or misleading statements to investors regarding its financial performance and prescription data for its drug RELYVRIO [1][2][4]. Group 1: Lawsuit Details - The lawsuit is on behalf of investors who purchased shares between November 11, 2022, and November 8, 2023 [1]. - The lawsuit claims that Amylyx overstated the commercial prospects and prescription rates of RELYVRIO and attempted to conceal negative trends by blocking analysts from accessing prescription data [4]. Group 2: Financial Performance - On November 9, 2023, Amylyx Pharmaceuticals reported disappointing Q3 financial results, indicating a slowdown in new prescriptions due to increased discontinuations [3]. - Company management stated that 60% of patients remained on RELYVRIO therapy six months after initiation in the U.S. [3]. Group 3: Market Reaction - Following the announcement of the financial results, Amylyx Pharmaceuticals' shares fell over 31% on November 9, 2023, significantly impacting investors [4].

Core Viewpoint - The biotechnology sector is crucial for financial markets and human health, providing innovative solutions for complex diseases. The article highlights three promising biotech stocks for investment. Group 1: MiMedx Group (MDXG) - MiMedx specializes in advanced wound care solutions, particularly for diabetic foot ulcers and venous leg ulcers [2] - The company recently launched EPIEFFECT, enhancing its product range for deep or tunnel wounds [2] - In its last quarterly report, MiMedx reported a 20.7% increase in net sales and a net income of $8.5 million [2] - The company has improved its capital structure by refinancing existing debt and securing new lines of credit [2][3] - MiMedx secured credit agreements for approximately $95 million, indicating confidence in future growth [3] Group 2: Amylyx Pharmaceuticals (AMLX) - Amylyx focuses on innovative treatments for severe neurodegenerative diseases and received regulatory approval for its flagship product, RELYVRIO [4] - The company experienced significant growth in net product revenues, reflecting increasing demand for its products [5] - Amylyx is committed to future growth through ongoing investments in research and development, including projects like AMX0035 for progressive supranuclear palsy [5] - The company completed recruitment for the HELIOS Phase 2 trial, targeting Wolfram syndrome, a rare genetic disorder [5] Group 3: BeiGene (BGNE) - BeiGene is making strides in cancer treatment, particularly for chronic lymphocytic leukemia and other solid tumors [6] - The company’s product BRUKINSA received approval from European and British regulatory authorities and saw a 130% increase in global sales [6] - BeiGene reported a remarkable 101.6% growth in total revenues in its latest quarterly report, showcasing its strong market position [6] - The company acquired an exclusive global license for an oral CDK2 inhibitor from Ensem Therapeutics, emphasizing its commitment to innovative solutions [7]

Core Insights - Amylyx Pharmaceuticals has completed enrollment for its Phase 2 HELIOS trial of AMX0035 for treating Wolfram syndrome, a rare genetic disease with no known cure, involving 12 adult participants, with preliminary results expected in the second half of 2024 [1][2] Group 1: Clinical Trial Details - The HELIOS trial is an open-label proof of biology study aimed at assessing the safety, tolerability, and effects of AMX0035 on various functions in adults with Wolfram syndrome [2] - Preliminary findings from the HELIOS trial are anticipated later this year, which will inform future trials for individuals with Wolfram syndrome [2][4] Group 2: Background on Wolfram Syndrome - Wolfram syndrome is characterized by childhood-onset diabetes, optic nerve atrophy, and neurodegeneration, leading to a poor prognosis and premature death in many cases [5] - The disease is difficult to diagnose and currently has limited treatment options focused mainly on symptom management [2][5] Group 3: AMX0035 Overview - AMX0035 is a fixed-dose combination of sodium phenylbutyrate and taurursodiol, currently approved for treating amyotrophic lateral sclerosis (ALS) under the brand name RELYVRIO in the U.S. and ALBRIOZA in Canada [6] - The drug is being explored for potential treatment in other neurodegenerative diseases, including Wolfram syndrome [6] Group 4: Regulatory Status - The FDA granted orphan drug designation to AMX0035 for Wolfram syndrome in November 2020, which provides certain benefits, including financial incentives and potential market exclusivity upon regulatory approval [4]