On May 31, 2022, Eledon Pharmaceuticals announced topline results from their Phase 2a trial of tegoprubart (formerly AT1501), a drug invented and developed at the ALS Therapy Development Institute (ALS TDI).

The Phase 2a trial in ALS was a 12-week, open label, dose escalating, safety, and biomarker study. The endponts of the study were safety and tolerability, and changes in pro-inflammatory biomarkers. Tegoprubart was well-tolerated, with no drug-related serious adverse events. The trial demonstrated tegoprubart dose-dependent target engagement and pro-inflammatory biomarkers were dose-dependently reduced. Pro-inflammatory biomarker reduction was associated with a non-significant trend toward slowing disease progression as measured by ALSFRS-R.

“We are excited to learn that tegoprubart has been demonstrated to be safe and well-tolerated in people with ALS and to learn that the drug is hitting its target pathways in humans. We look forward to Eledon’s next steps in the development of this drug for people with ALS,” said Fernando G. Vieira, M.D., ALS TDI CEO and CSO.

Tegoprubart is an antibody therapeutic targeting, CD40L, a well-validated biologic taget with broad therapeutic potential. It is the culmination of years of research and experimentation at ALS TDI ¬– made possible by the generous support of the ALS community.

After being invented at ALS TDI, a nonprofit biotech, tegoprubart was licensed to Anelixis Therapeutics, a for-profit biotech company. In 2019, Anelixis oversaw the completion of a Phase 1 trials tegoprubart. In 2020, Anelixis was acquired by Eledon Pharmaceuticals, a for-profit clinical-stage drug development company. Tegoprubart has been the cornerstone of Eledon’s efforts to develop the next generation of antiCD40 ligand antibodies for patients in need of potentially life-saving treatment.

Ending ALS will require many treatments to meet the needs of every individual living with this disease. As the Drug Discovery Engine for ALS, ALS TDI is solely focused on inventing and discovering treatments until everyone with ALS has access to effective treatments.

“This is an important milestone. We are proud of this progress, but our work is far from finished. We hope tegoprubart proves to be the first of multiple effective treatments for ALS invented at ALS TDI. ALS TDI will continue our work to invent and discover more promising drugs until we have met the treatment needs of all ALS patients. Together we will end ALS.” said, Augie Nieto, ALS TDI Chairman.

Charitable support that ALS TDI has received over the years through donations and fundraisers has been an important source of funding for the research behind the early discoveries and invention of tegoprubart. Vital funding has also come from organizations such as Augie's Quest, ALS Association, ALS Finding a Cure, ALS ONE, the Muscular Dystrophy Association, and the Department of Defense CDMRP, among others.

For more information about ALS TDI’s invention of AT-1501, click here. To learn more about how you can support ALS TDI’s continued ALS research, click here.