CAMBRIDGE, Mass.–(BUSINESS WIRE)–Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) today reported financial results for the fourth quarter and full year ended December 31, 2023.
“In 2023, we made significant strides towards transforming the way ALS is treated. We delivered RELYVRIO, also known as ALBRIOZA and AMX0035, the first and only ALS therapy that has been shown to slow disease progression, help maintain functional independence, and extend overall survival in the same clinical trial, to thousands of people living with ALS in North America,” said Joshua Cohen, co-CEO of Amylyx. “As we reflect on the many stories that we have heard from people living with ALS who are taking RELYVRIO, we are working with urgency to continue expanding our U.S. and international presence in 2024 and bringing RELYVRIO to more people living with ALS.”
“We look forward to sharing topline data from our Phase 3 PHOENIX trial during or before the second quarter of 2024. PHOENIX will provide additional efficacy and safety data in a larger population of people living with ALS, building on the robust and positive results observed in the CENTAUR study,” said Justin Klee, co-CEO of Amylyx. “We also remain focused on advancing our pipeline, which includes studying AMX0035 in Progressive Supranuclear Palsy in the Phase 3 ORION trial. We look forward to several expected upcoming milestones for our pipeline in 2024, including data from our Phase 2 HELIOS trial in Wolfram syndrome and our antisense oligonucleotide, AMX0114, entering the clinic.”
Full Year 2023 and Recent Business Highlights:
- Commercial launches of RELYVRIO® in the U.S. and ALBRIOZA™ in Canada, also known as AMX0035, continued to progress, and eligible people living with ALS in multiple countries are also accessing RELYVRIO through early access pathways. Net product revenue for the three months ended December 31, 2023 was $108.4 million, compared to net product revenue of $102.7 million for the three months ended September 30, 2023. The vast majority of Amylyx’ net product revenue is generated in the U.S.; revenue in Canada and from named patient sales programs in international markets represented an important source of revenue growth in three months ended December 31, 2023 relative to the Company’s financial results for the three months ended September 30, 2023.
- Amylyx announced the first patient dosed in the Global Phase 3 ORION Study of AMX0035 in Progressive Supranuclear Palsy (PSP). ORION is a global, randomized, double-blind, placebo-controlled Phase 3 clinical trial designed to assess the efficacy, safety, and tolerability of AMX0035 compared to placebo. Approximately 600 participants will be enrolled across North America, Europe, and Japan. The first participant was dosed in December 2023, and topline results are anticipated in 2025 or 2026.
- Amylyx continues to progress its R&D programs. The Company announced completion of enrollment in the HELIOS study, a Phase 2 clinical trial of AMX0035 for the treatment of Wolfram syndrome and continues to expect data from the study in 2024. The Company is also working on a novel composite diagnostic test to assist in diagnosing ALS earlier. Additionally, Amylyx is working on a new taste-masked formulation of RELYVRIO that may allow for new intellectual property.
- Amylyx added new members to its executive leadership team and U.S. commercial leadership team. In November 2023, the Company announced the appointment of Camille L. Bedrosian, MD, as Chief Medical Officer. Dr. Bedrosian has nearly 30 years of experience addressing unmet medical needs for people with rare and serious diseases through successful clinical and translational research programs. In addition, during 2024, Linda Arsenault joined as the Company’s new Chief Human Resources Officer and Dan Monahan joined as General Manager and Head of U.S. Commercial Markets. Ms. Arsenault has over 30 years of human resources leadership experience, including in the biopharmaceutical sector, most recently from Sunovion Pharmaceuticals where she served as Chief Human Resources Officer. Mr. Monahan has over 20 years of experience in sales, marketing, and market access, including his most recent position as VP of CNS Marketing and Portfolio Strategy at Otsuka Pharmaceutical Companies.
- A post hoc survival analysis comparing the CENTAUR clinical trial to historical clinical trial control was published in the Annals of Clinical and Translational Neurology. The results of this post hoc analysis demonstrated that the median overall survival was 10.4 months longer in the CENTAUR AMX0035 group than in the historical clinical trial control group.
- Post hoc analyses on CENTAUR trial participants were published in Journal of Neurology, Neurosurgery and Psychiatry. The results of the post hoc analyses demonstrated a significant reduction in plasma concentrations of YKL-40 (also known as chitinase-3-like protein 1) and the systemic inflammatory biomarker C-reactive protein (CRP), two plasma neuroinflammatory biomarkers in ALS, over 24 weeks, with reductions observed as early as Week 12 in participants from the CENTAUR trial.
- Data on RELYVRIO and AMX0114, the Company’s investigational antisense oligonucleotide targeting calpain-2, were presented at the 34th International Symposium on ALS/MND. An update on kinetic profiling experiments of AMX0114 was presented as well as results from a collaboration with Dr. Sami Barmada and his team at the University of Michigan School of Medicine in which the impact of AMX0114 on survival was evaluated in human iPSC-derived motor neurons with an ALS associated mutation in TDP43. The Company is advancing AMX0114 through investigational new drug (IND) enabling studies and the goal is to enter the clinic during 2024. Additional information, including copies of each of the posters presented at the event, are available in the in the “Publications and Presentations” section of the Amylyx website.
Financial Results for the Fourth Quarter and Year Ended December 31, 2023
Net product revenue: Net product revenue was $108.4 million for the three months ended December 31, 2023, compared to net product revenue of $21.9 million for the same period in 2022. Net product revenue for the year ended December 31, 2023 was $380.8 million, compared to net product revenue of $22.2 million for the same period in 2022. The increase was primarily driven by units of RELYVRIO sold in the U.S. following regulatory approval in late September 2022.
Cost of Sales: Cost of sales were $9.4 million in the three months ended December 31, 2023, compared to cost of sales of $2.8 million for the same period in 2022. For the year ended December 31, 2023, cost of sales were $25.4 million, compared to cost of sales of $3.0 million for the year ended December 31, 2022.
R&D Expenses: Research and development expenses for the fourth quarter of 2023 were $44.9 million, compared to $22.8 million for the same period in 2022, and $128.2 million for the year ended December 31, 2023 compared to $93.5 million for the year ended December 31, 2022. The increase was primarily driven by an increase in personnel-related expenses due to added headcount to support research and development efforts, an increase in spending related to the initiation of the Phase 3 ORION trial of AMX0035 in PSP, and an increase in preclinical development activities.
SG&A Expenses: Selling, general, and administrative expenses for the fourth quarter of 2023 were $52.2 million, compared to $40.8 million for the same period in 2022 and $188.4 million for the year ended December 31, 2023, compared to $127.1 million for the year ended December 31, 2022. The increase was primarily driven by higher personnel-related expenses due to added headcount to support the launch, commercialization initiatives, and operations as a public company.
Net Income: Net income for the three months ended December 31, 2023 was $4.7 million, or $0.07 on a fully diluted per share basis, compared to a net loss of $42.7 million, or $0.65 per share for the same period in 2022. Net income for the year ended December 31, 2023 was $49.3 million, or $0.70 on a fully diluted per share basis, compared with a net loss of $198.4 million, or $3.39 per share for the year ended December 31, 2022.
Cash Position: Cash, cash equivalents, and short-term investments were $371.4 million at December 31, 2023, compared to $355.0 million at September 30, 2023.
About RELYVRIO®/ALBRIOZA™/AMX0035
RELYVRIO® (also known as AMX0035), an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol (known as ursodoxicoltaurine outside of the U.S.), is approved to treat amyotrophic lateral sclerosis (ALS) in adults in the U.S. and approved with conditions as ALBRIOZA™ for the treatment of ALS in Canada. AMX0035 is being studied as an investigational drug in several other regions for the potential treatment of ALS and other neurodegenerative diseases. The formulation of RELYVRIO, ALBRIOZA, and AMX0035 are identical.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative community through the discovery and development of innovative new treatments. Amylyx is headquartered in Cambridge, Massachusetts and has operations in Canada, EMEA, and Japan. For more information, visit amylyx.com and follow us on LinkedIn and X, formerly known as Twitter. For investors, please visit investors.amylyx.com.
