Neurons die. This is a sign of amyotrophic lateral sclerosis and other neurodegenerative diseases, but drug developers are mostly hindered in their attempts to help these nerve cells survive. Amylyx Pharmaceuticals designed its experimental therapies to address the pathways that lead to neuronal cell death. The company Announce On Wednesday, the FDA accepted its new drug application. The agency set the target date for the decision as June 29, 2022.
The acceptance of drug applications brings a busy first half of 2022 to Amylyx, headquartered in Cambridge, Massachusetts. The FDA placed Amylyx’s drug application under priority review, and the name is reserved for drugs that can significantly improve the treatment of serious diseases. Before the FDA makes any decision, the company stated that the regulator plans to hold a meeting of independent experts to discuss the safety and effectiveness of its drug AMX0035. The date of this advisory committee meeting has not yet been determined. By then, Amylyx may already be a public company.Earlier this month, biotech companies Submit IPO documents With securities regulators.
Amylyx stated in its IPO document that although most cells in the body die and are replaced regularly, mature neurons usually resist cell death, but they usually cannot regenerate. Their death is triggered by the activation of a variety of stress factors, many of which stem from the dysfunction of the endoplasmic reticulum, which is a cell network that plays a role in the production, modification and transport of proteins. Amylyx aims to improve the survival rate of neurons by solving the problems of endoplasmic reticulum and mitochondria, which are the energy-producing components of cells.
Amylyx stated in the submission that when mitochondria detect enough cell damage, they initiate a cell death cascade. This process involves recruiting certain proteins. Amylyx’s ALS drug consists of two small molecules, namely tauroursodeoxycholic acid and sodium phenylbutyrate. Amylyx said that by using these two molecules to block key cell death proteins, its drugs may increase the threshold of cell death and extend the survival time of neurons. In laboratory tests and preclinical models of ALS, Alzheimer’s disease and multiple sclerosis, Amylyx stated that its drugs can keep neurons alive under various conditions and stresses. Tauroursodeoxycholic acid and sodium phenylbutyrate are not patents of Amylyx, but the company’s intellectual property includes fixed-dose combinations of molecules.
“We believe that the proprietary combination of these two mechanisms of action will allow us to target abnormal cell death and prevent neurodegeneration better than treatment with either mechanism alone,” Amylyx said in the document.
The AMX0035 submitted by Amylyx is based on data from a placebo-controlled Phase 2 clinical trial that enrolled 137 patients. Based on a scale that measures physical function in patients with ALS, this 24-week study achieved the main goal of slowing the progression of the disease. After the trial is over, the study participants continue the open-label extension study. Amylyx reported that compared with placebo participants who started treatment during the expansion study, participants who started using Amylyx during the blinded study and continued treatment in the expansion study had a 44% lower risk of death. In addition, those who initially started taking the study drug lived an average of 6.5 months longer than those who started taking the placebo.The complete research result is Publish Published in the New England Journal of Medicine in September 2020.
Earlier this year, the FDA tells Amylyx that it needs to conduct Phase 3 studies before seeking regulatory approvalBut throughout the summer, several discussions with the agency opened the door for the company to submit a drug application based on the Phase 2 results. Amylyx submits application in November.
AMX0035 is the main drug candidate of Amylyx, and so far, it is the only drug candidate of the biotech company. The company aims to test its methods to improve neuron survival rates as a way to treat various neurodegenerative diseases. In addition to ALS, the company has also reached a mid-term clinical trial of the drug to treat Alzheimer’s disease.The next disease target is Wolfram syndrome, A rare hereditary neurodegenerative disease According to the IPO document, Amylyx is expected to submit a research drug application for Wolfram Syndrome in the first half of 2022. Drug applications for the other two diseases are also planned for next year. These diseases are not specified, but the company does say that the Phase 2 test for Alzheimer’s disease “can help us prioritize other indications for AMX0035.”
At the same time, the Phase 3 clinical trial of AMX0035 is underway in ALS. Although the study is not currently part of the FDA application, Amylyx stated that the study aims to provide additional safety and efficacy data for AMX0035 in ALS, which will support the company’s efforts to seek regulatory approval in other countries. The drug is currently under review in Canada. Amylyx plans to submit marketing applications in Europe in the first quarter of 2022. The company said it is preparing an expanded access plan, which is expected to be launched in the next few months for ALS patients who do not qualify for the Phase 3 study.
Amylyx’s IPO document contains a $100 million fundraising goal. The company has applied to be listed on NASDAQ under the stock code “AMLX”. According to the document, Amylyx plans to use the IPO proceeds to fund regulatory and pre-commercial work, and if the drug is approved, it will also carry out activities. Part of the funds will be used for the ongoing Phase 3 testing of AMX0035 in AMX and the development of the drug for other neurodegenerative diseases.
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