“We are grateful to Cytokinetics for their partnership and transparency, enabling the global research community to leverage these data in their ongoing analyses and research activities,” said Neil Thakur, Ph.D., Chief Mission Officer at The ALS Association.
When the FDA recently accepted Biogen’s new drug application for tofersen, it signaled hope for thousands of people with SOD1 mutations that cause ALS. The FDA has granted priority review to this new drug application and is expected to decide on whether or not to approve tofersen by January 25, 2023.
The researchers focus on an earlier phase of ALS development, which is commonly understood to be a “silent” phase – before symptoms of the disease begin to manifest. Those minor motor impairments in the earlier phase are currently insufficient for a confirmed diagnosis.
The ALS Association filed formal objections with the Institute for Clinical and Economic Review, commonly known as ICER, over their flawed draft report on the cost-effectiveness of AMX0035 and oral edaravone.
People with ALS, caregivers and people at risk of an ALS diagnosis have the opportunity to help the National Institute of Neurological Disorders and Stroke (NINDS) review applications for funding to support research into expanded access.
The Food and Drug Administration has extended the timeline of its review of AMX0035 citing additional data further showing that it is effective at reducing harms associated with the disease.
Follow-up analyses from the Phase 3 Valor study and the open-label extension study of tofersen, an investigational antisense drug, showed that the drug was effective in slowing down progression of ALS in people with SOD1 mutations.
New long-term analysis published in the Journal of Neurology, Neurosurgery & Psychiatry showed AMX0035 was effective at reducing a variety of harms associated with ALS during the previously published Phase 2 Centaur trial.
The ALS Association, in collaboration with ALS Finding a Cure, recently awarded $400,000 to support research into the role SARM1 gene mutations play in the development and progression of ALS, with the ultimate goal of using an improved understanding of SARM1 to develop new treatments.
The ALS Association has launched a new funding opportunity to support exploratory research that has the potential for a significant impact on the fight against ALS.
The ALS Association, thanks to support from the Greater New York Chapter, has awarded $525,000 to four projects through its Milton Safenowitz Postdoctoral Fellowship program, which supports the development of new scientists in the field of ALS research.
Biogen and Ionis Pharmaceuticals have decided to discontinue research testing the investigational drug BIIB078 after a phase 1 clinical trial did not meet any secondary endpoints on efficacy and did not demonstrate clinical benefit.
“The Lawrence and Isabel Barnett Drug Development Program has fostered critical relationships between academia and industry and helped move the science forward. We are proud of the fact that projects supported by this program have leveraged that support to attract 6 more dollars for every $1 we initially invested,” said Dr. Kuldip Dave, vice president of research at The ALS Association.
Today we filed public comments with the FDA advisory committee considering Amylyx Pharmaceutical’s new drug application for AMX0035, urging the agency to approve the application.
The ALS Association announced today that it has awarded a $620,000 grant to the Packard Center for ALS Research at Johns Hopkins to support the Answer ALS Data Portal. Launched in January this year, the Data Portal provides open access to the world’s most comprehensive collection of ALS data.