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]

Key PointsAmylyx may get its flagship treatment for ALS approved by the European Medicines Agency (EMA), opening another avenue for revenue growth.  Puma Biotechnology will begin a mid-stage trial for its lead cancer drug.  Cassava Sciences forecasts top-line results for a Phase 3 study on its Alzheimer's disease drug to be available by the end of the calendar year.  5 stocks we like better than Amylyx PharmaceuticalsThere's a reason that many biotech investors prefer to stick with large-cap stocks. Investi ...

Core Insights - The article compares two companies in the Medical - Drugs sector: Amylyx Pharmaceuticals, Inc. (AMLX) and Neurocrine Biosciences (NBIX), focusing on which stock may be undervalued for investors [1][3] Valuation Metrics - AMLX has a forward P/E ratio of 10.06, significantly lower than NBIX's forward P/E of 29.42, indicating that AMLX may be undervalued [2] - The PEG ratio for AMLX is 0.15, while NBIX has a PEG ratio of 0.75, suggesting that AMLX has a better growth outlook relative to its valuation [2] - AMLX's P/B ratio is 2.52 compared to NBIX's P/B of 6.97, further indicating that AMLX is more attractively valued [2] Investment Ratings - AMLX currently holds a Zacks Rank of 2 (Buy), while NBIX has a Zacks Rank of 3 (Hold), reflecting a stronger earnings outlook for AMLX [1] - Based on the analysis of valuation metrics and estimate revisions, AMLX is deemed the superior option for value investors at this time [3]

Group 1: Biotech Sector Overview - The biotech sector has massive potential due to the importance of health and the high prices commanded by firms producing therapeutics for human diseases [1] - Wall Street analysts closely monitor the sector, influencing investment decisions with their ratings [1] Group 2: Amylyx Pharmaceuticals (AMLX) - Amylyx Pharmaceuticals focuses on therapeutics for neurodegenerative diseases, particularly ALS, and is highly rated by analysts with seven 'Buy' ratings and one 'Overweight' rating [2] - The company's lead drug, Relyvrio, received FDA approval in 2022 and generated sales of $102.7 million in Q3 [3] - Positive Phase 3 trial results expected in March are crucial for further growth and potential European market approval [3] Group 3: Bio-Techne (TECH) - Bio-Techne produces diagnostic products and services rather than therapeutics, holding a strong 'Buy' rating from Wall Street [5] - The diagnostics market is projected to grow at a compound annual growth rate of 6.7% from 2024 to 2032, indicating steady growth [5] - The company has seen earnings growth of over 16% annually in the past five years, with expectations of more than 11% annual growth in the next five years [6] Group 4: Exelixis (EXEL) - Exelixis holds an 'Overweight' rating and has recently undergone a corporate restructuring to focus on its clinical programs [7] - The company laid off 175 employees, representing 13% of its workforce, to reduce costs and prioritize label expansions for its cancer drug Cabometyx [8] - The restructuring is expected to enhance overall company performance and is positively viewed by Wall Street analysts [8]