A patient-funded ALS drug offers hope where few treatments exist

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When fitness mogul Augustine “Augie” Nieto II received the devastating diagnosis of amyotrophic lateral sclerosis (ALS), known as Lou Gehrig’s disease, in 2005, his response was to spring into action.

Eledon Pharmaceuticals chief scientific officer Steve Perrin

Augie Nieto

Permission granted by Augie’s Quest/Augie Nieto

“Rather than just getting that horrible diagnosis and hearing that you’ve possibly got three to five years to live, and going about your business and enjoying life with your family, Augie started seeking out advice on how he could help leverage his networks to find effective treatments for ALS,” says Steve Perrin, president and chief scientific officer of the Irvine, Calif.-based, clinical stage biotech, Eledon Pharmaceuticals

Nieto, the founder of Lifecycle Inc., which later became the exercise equipment company Life Fitness, set his sights on a cure, an effort that has raised some $160 million for ALS research. The initiative recently bore fruit when its first drug discovery — tegoprubart — entered clinical trials. The drug was developed by the ALS Therapy Development Institute, which partnered with Nieto’s organization Augie’s Quest to Cure ALS. Nieto now serves as chairman of both.

Eledon chief scientific officer Steve Perrin

Permission granted by Eledon/Steve Perrin

An early trial of this new drug candidate offered up a glimmer of hope that tegoprubart may prove to be an effective option for treating ALS, filling the void that currently exists. “There’s no effective treatment [for ALS],” Perrin says. “Even though there are approved drugs, they really don’t slow down disease or improve quality of life.”

While there is much work to be done, the progress made on tegoprubart is encouraging.

“All of that work is funded by patients and families,” Perrin says. “Augie and I went out and we raised the money in the nonprofit with traditional grassroots fundraising, by going out and telling people what we’re trying to build, knowing that they’ve been diagnosed with the same horrible disease that Augie had been. ”

The drug would not be a cure, but it could slow the progression of the disease — buying time for people who have very little.

“People want a drug that’s going to allow them to see their kids grow up, to see their kids graduate from college and get married and have grandkids,” Perrin says. “They know that a cure might not be there, but how about something that I can take the time to slow the disease down.”

‘Exciting’ study results

Tegoprubart is an anti-CD40L drug, acquired for development by Eledon. The drug targets a protein linked to the body’s immune response to reduce inflammation in the brain, spinal cord and muscle that is thought to drive ALS, which causes deterioration of those organs leading to weakness and paralysis.

Eledon, which focuses on organ and cell transplantation, autoimmune conditions and neurodegenerative disease, acquired the rights to develop tegoprubart and recently reported topline results from a small phase 2a trial that included 54 people with ALS, using each patient’s baseline data as a control.

It found that the drug not only appears to be safe — no serious drug-related adverse events occurred — it also successfully reduced 23 of 32 elevated protein biomarkers associated with ALS in some patients, demonstrating a dose-dependent response. In addition to reducing ALS biomarkers, researchers also found a trend toward slowing in disease progression, an outcome that surprised researchers. The trial was not designed to measure a clinical response to the drug, so the finding was both unexpected and exciting, according to Perrin.

Eledon CEO David-Alexandre Gros

Eledon CEO David-Alexandre Gros

Permission granted by Eledon/David-Alexandre Gros

“It was a fairly short study as far as duration — it was only 12 weeks,” Perrin says. “And typically to monitor disease progression in an ALS clinical trial it’s usually six to 12 months or longer.”

When researchers noticed that the drug effectively blocked the signaling and also appeared to correlate with disease progression even in a short trial, the result was unexpected.

“It was quite exciting that in a short study, you could see something like that,” Perrin says.

Having specific targets and being able to measure the response gave the findings some additional validity, according to Dr. David-Alexandre Gros, CEO of Eledon.

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