Amylyx Pharmaceuticals has shown in a clinical trial that its amyotrophic lateral sclerosis (ALS) drug slows the progression of muscle wasting disease. The drug is now preparing for a larger, pivotal study aimed at replicating these results, and the company has raised $135 million in new funding to support this research.
Amylyx, headquartered in Cambridge, Massachusetts, initially hoped that the positive data from Phase 2 would be enough to seek accelerated FDA approval. This approach can deliver drugs to patients more quickly based on less evidence than is required for standard drug reviews, but still requires the company to pass additional post-marketing tests to confirm patient benefits.The Series C financing announced on Tuesday was announced three months later in Cambridge, Massachusetts Amylyx says the FDA will need data from larger placebo-controlled studies Before it can consider the company’s application.
Amylyx’s drug AMX0035 is designed to interfere with the cellular pathways that cause nerve cell death. The drug is a proprietary combination of tauroursdiol and sodium phenylbutyrate. It is formulated as a powder and must be dissolved in water and taken orally twice a day.
The placebo-controlled phase 2 study enrolled 137 ALS patients.in Preliminary results Released at the end of 2019, 24 weeks of treatment with the drug resulted in a statistically significant slowdown in ALS progression, which is the main research goal. Patients are evaluated on a scale that measures how people with ALS do things like walking, dressing, eating, talking, and breathing.
Based on the complete Phase 2 clinical trial results published in September last year New England Journal of MedicineCompared with the placebo group, patients receiving Amylyx drug treatment scored an average of 2.32 points higher on the evaluation scale. In the secondary clinical trial goals that assess muscle strength, breathing, and hospitalization, patients treated with AMX0035 have improved values compared with patients receiving placebo.
Based on the results of Phase 2, Amylyx Archive Obtained regulatory approval for its drug in Canada in June. The company said it plans to submit the drug to the European Medicines Agency by the end of this year. It is planned to begin supporting the Phase 3 study submitted by the FDA later this quarter. The clinical trial will be conducted in the United States and Europe.
Amylyx said that the new round of funding will be used to support the clinical development and potential launch of its ALS drugs. The cash will also support the continued development of AMX0035 in other diseases. The Alzheimer’s disease project is in phase 2 testing. Procedures to evaluate the drug in the rare Wolfram syndrome are in preclinical development.
Viking Global Investors led Amylyx’s Series C financing. Other investors in this round of financing include Bain Capital Life Sciences, Perceptive Advisors, Rock Springs Capital, Woodline Partners, Marshall Wace, Tybourne Capital Management, Verition Fund Management, aMoon Fund and Falcon Edge. Earlier investors Morningside Ventures, 683 Capital Management, Belinda Termeer and Polaris Founders Capital also participated in the financing.
“We have a clear mission at Amylyx: to develop new drugs for ALS and other progressive neurodegenerative diseases,” Amylyx’s Chief Financial Officer James Frates said in a prepared statement. “We are eager to cooperate with Viking and our experienced biotech investor consortium because we execute our plan and hope to bring AMX0035 to people with ALS as soon as possible.”
Photo: Matthew Howwood, Getty Images
