Amylyx Pharmaceuticals has fulfilled its promise of regulatory review of its amyotrophic lateral sclerosis drugs.The company said on Tuesday that it submit A new drug application submitted to the FDA based on the results of an interim clinical trial, even if the biotech company continues its larger phase 3 research program.
The experimental Amylyx treatment has been subject to regulatory review in Canada, and plans to be submitted in Europe are still in progress. The drug AMX0035 submitted by the FDA almost never happened. The drug released positive Phase 2 data, and the agency has a precedent that can accelerate the approval of drugs for diseases that are urgently needed and few treatments are available, such as ALS. But in April, Amylyx said the FDA asked to see more data from another placebo-controlled study. This biotechnology company based in Cambridge, Massachusetts developed a plan for the clinical trial and raised funds to fund it- $135 million in Series C financing.
The prospects for earlier submission of regulatory documents improved in the summer. Amylyx has had several meetings with the FDA, including a meeting in July dedicated to discussing potential regulatory submissions. In September, the company stated that based on these discussions, it expects to officially Seek FDA approval For the “coming months” drugs.
The Amylyx drug is a pairing of two main drug ingredients, taurorsodiol and sodium phenylbutyrate. The drug combination aims to block the cell death pathway in the mitochondria, which is the part of the cell that produces energy, and the cell death pathway in the endoplasmic reticulum, which is the cell transport system for proteins.
In a placebo-controlled phase 2 study of 137 people, the drug achieved its main goal of showing a statistically significant improvement in the reduction of functional decline after six months. Study participants were evaluated under the scoring scale used to assess the physical function of ALS patients.
Participants in clinical trials can choose to continue the open-label study for up to three years. In this extended study, Amylyx reported that those who started using Amylyx in the placebo-controlled portion of the Phase 2 study had a 44% lower risk of death compared with those who started taking placebo. The average life expectancy of patients who started using Amylyx drugs has also been extended by approximately 6.5 months.The result of this research is Publish Last year in the New England Journal of Medicine.
Amylyx stated that the planned Phase 3 AMX0035 test will recruit approximately 600 patients in the United States and Europe and will be based on the safety and efficacy data generated in the Phase 2 test. The company’s goal is to begin the third phase of the study later this quarter.
Clene Nanomedicine ALS drug failed its phase 2 test
Clene Nanomedicine is developing nano-therapies designed to increase the energy production of central nervous system cells and promote the protection and repair of these cells. Main drug candidate CNM-Au8 Failure Achieve the primary and secondary objectives of the second phase of the ALS study. However, executives at Clene, based in Salt Lake City, pointed out that their results for some patients indicated that the drug showed encouraging signs. Shared restraint of ALS drug developers have Failed in the clinic.
CNM-Au8 is an oral suspension of gold nanocrystals. Clene said these crystals can cross the blood-brain barrier without causing the toxic effects associated with synthetic gold compounds or nanoparticles made in other ways. The main goal of the Phase 2 ALS study is to show improvement based on the measurement of functional neurons in the muscle. The secondary goal is to measure lung function. Although neither of these goals were achieved, Clene said that after 12 weeks, a “significant effect” was observed in ALS patients with limb attacks. The company added that the group showed an improvement trend in week 36. Clene pointed out that limb-onset ALS accounts for about 70% of ALS patients.
Through daily oral administration for 36 weeks, the patient tolerated the Clene drug well. No serious adverse events were reported. Clene stated that it expects to publish data from the study at the ALS/MND International Symposium scheduled for December 8-10.in a Investor introductionClene said that the Phase 2 results support the Phase 3 development of its drug.
The Clene drug has entered Phase 3 testing. In 2019, the drug was one of the selected drugs Key research Conducted by the Sean M. Healey & AMG ALS Center of Massachusetts General Hospital. The study is a platform trial in which multiple treatments are evaluated at the same time and other treatments are added when available. This method, which has been used in cancer, aims to accelerate the clinical development of ALS therapies.To date, the other two ALS treatments selected for testing in this platform study are from UCB and Bioport Pharmaceutical.
In addition to ALS, Clene is also testing its lead drug in patients with chronic optic neuropathy with stable relapsing multiple sclerosis. The plan is in phase 2.
Photos of Flickr users Gemetlau Through knowledge sharing license
