Company announces positive results for drug to treat ALS

A company headed by a Rancho Santa Fe man is working on a drug that shows promise for the treatment of amyotrophic lateral sclerosis, or ALS, a deadly neurodegenerative disease for which there is currently no effective drug regimen.

David-Alexandre Gros, CEO of Eledon Pharmaceuticals

(Courtesy)

Irvine-based Eledon Pharmaceuticals in late May announced results from a Phase 2a study of tegoprubart, an investigational drug for the treatment of ALS and several other related conditions, said David-Alexandre Gros, CEO of Eledon.

The drug was initially developed by the ALS Therapy Development Institute, the world’s foremost lab dedicated to finding treatments for ALS, and brought forward into the Phase 2a trial in partnership with Eledon, which raised $110 million for further study, said Gros.

ALS, also known as Lou Gehrig’s disease, causes the immune system to attack and destroy nerves that connect the brain and muscles, said Gros. Patients lose the ability to walk, talk, breathe and swallow in a rapid progression that leads to death on average within three to five years of diagnosis.

About 30,000 adults currently live with ALS in the U.S., with about 5,000 new cases diagnosed each year, Gros said. There is no cure for ALS and no effective treatment to halt or reverse the progression of the disease.

“There’s a great unmet need to come up with new drugs that can slow this disease,” Gros said. “It’s a very hard disease.”

Scientists hope tegoprubart can slow down the body’s immune system, thus reducing attacks on nerves and ultimately slowing the progression of ALS, said Gros.

Eledon’s study involved following 54 patients with ALS who were administered tegoprubart, said Gros. The study was conducted overt a 12-week period at 13 treatment sites in the U.S. and Canada.

Study results revealed no serious side effects in the subjects, he said, and the drug did reach the intended target cells in the patients. The results also showed a decrease in inflammation of the target cells, he said.

Data obtained from the study, said Gros, indicate “that our drug is having the effect we believed it would have.”

The results gathered from trials so far support moving forward to Phase 3 trials for tegoprubart, said Gros. The next step, he said, will be to consult with the U.S. Food and Drug Administration, patient advocacy groups and academics to discuss how the trials might move forward.

Additional funding will be needed for Phase 3 trials, he said.

ALS patients and their families are closely monitoring developments regarding tegoprubart, said Gros. Among them is Augie Nieto, a fitness industry entrepreneur who was diagnosed with ALS in 2005. Nieto co-founded Augie’s Quest to Cure ALS, which raises money to fund ALS research. He’s chair for both Augie’s Quest and the ALS Therapy Development Institute.

In a video released following the announcement of results from the tegoprubart Phase 2a trial, Nieto, using a computerized voice program, said, “As chairman of the ALS Therapy Development Institute, and as someone who has lived with ALS for nearly two decades, it is gratifying to see the positive results of this research. I look forward to the next phase of development for tegoprubart. We will not stop until a cure for ALS is a reality.”

While the data on tegoprubaqrt is exciting, said Gros, more work is needed to effectively turn back and ultimately stop ALS.

“Multiple different drugs are going to be needed to address this disease,” he said.

Gros has served as CEO and member of the board of directors of Eledon since September 2020. Gros graduated from medical school at Johns Hopkins University and earned an MBA at Harvard University. He also served as an executive at several large biotech firms, including Sanofi.

As Eledon moves forward on studies of tegoprubart, Gros said it’s important to keep the focus on patients, the ultimate beneficiaries of the work.

“If we do right by patients everything else will fall into place,” he said.

Visit eledon.com for more information.