[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]

While the technology sector – especially regarding artificial intelligence – may have captured the spotlight, investors ought to consider adding healthcare stocks to their portfolio. If I had to boil it down to one phrase, it would be permanent relevance. A bold claim but I believe there’s much validity to it.First and foremost, advanced medicine stocks help improve the human condition. Nobody in their last moments wished they had spent more time in the office. Instead, they regret that they didn’t spend en ...

Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) Q3 2023 Earnings Conference Call November 9, 2023 8:00 AM ET Company Participants Lindsey Allen - Head of Investor Relations & Communications Josh Cohen - Co-CEO & Co-Founder Justin Klee - Co-CEO & Co-Founder Margaret Olinger - Chief Commercial Officer James Frates - Chief Financial Officer Conference Call Participants Corinne Jenkins - Goldman Sachs Geoff Meacham - Bank of America Umer Raffat - Evercore ISI Marc Goodman - Leerink Partners Graig Suvannavejh - Mizuh ...

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 September 30, 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 F ...