The president and chief executive officer of The ALS Association provided insight on how the organization is advancing the therapeutic pipeline of ALS agents for the foreseeable future. [WATCH TIME: 3 minutes]
WATCH TIME: 3 minutes
"We’ve got several [agents] in phase 2 and phase 1. Again, what I see is that when we start to invest in some of those early on, that’s where we see them actually get to clinics. That’s some of the work that’s happening already."
Part of The ALS Association’s initiative to make amyotrophic lateral sclerosis (ALS) a livable disease by 2030 includes an emphasis on finding new treatments and cures, with more clinical trials and participation with research and more focused research as well. They have stood the test thus far, providing funding for 155 active projects in 12 countries around the world, representing a current commitment of $52 million in 2021. Since 2014, they have committed more than $116 million in expanding research for new treatments.
Although there are only 2 FDA-approved medications—riluzole (Rilutek) and edaravone (Radicava)—there are several different investigational agents that are drawing the eye of experts. One of the most notable, AMX0035 (Amylyx), has shown promise through phase 3 settings as a slower of functional decline in patients with the disease. From an action meeting that brought the voice of the ALS community directly to the FDA, to a recent call for the agency to conduct a priority review of AMX0035, The ALS Association has been a strong supporter of bringing this promising new treatment to the market as quickly as possible.
Amylyx is expected to hear back from the FDA regarding AMX0035’s new drug application in June. Others, including Calaneet Balas, MSc, MBA, president and chief executive officer of The ALS Association, have been hopeful of its potential as well. Balas sat down with NeurologyLive®to discuss the organization’s focus on building a strong pipeline of ALS therapeutics, along with some of the exciting agents the community should key in on.