Amylyx(AMLX) - 2023 Q4 - Annual Results

Executive Summary & Financial Highlights 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 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] - 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] - 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] Business Highlights The company achieved continued commercial progress with RELYVRIO®/ALBRIOZA™, advanced key clinical trials, and strengthened its leadership team Commercial Progress 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] Clinical Pipeline and R&D 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] - Enrollment was completed in the Phase 2 HELIOS study of AMX0035 for Wolfram syndrome, with data expected in 2024[3] - The investigational antisense oligonucleotide AMX0114, targeting calpain-2, is advancing through IND-enabling studies with the goal of entering the clinic during 2024[5] - 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] Corporate Developments 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][5] - Chief Human Resources Officer: Linda Arsenault joined in 2024[3][5] - General Manager and Head of U.S. Commercial Markets: Dan Monahan joined in 2024[3][5] Detailed Financial Results (Q4 & Full Year 2023) 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 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] Operating Expenses 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] - 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] Profitability and Cash Position 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] Financial Statements This section presents unaudited Condensed Consolidated Balance Sheets and Statements of Operations for Q4 and full years 2023 and 2022 Condensed Consolidated Balance Sheets 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 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™ 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] - Warnings include risks for patients with disorders of enterohepatic circulation, pancreatic, or intestinal issues due to the taurursodiol (a bile acid) component[14] - 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] - The most common adverse reactions (at least 15% and 5% greater than placebo) were diarrhea, abdominal pain, nausea, and upper respiratory tract infection[16]