Skip to main content

5/31/22:  Eledon Pharmaceuticals announced positive topline results today from their Phase 2 trial of tegoprubart (formerly AT-1501) demonstrating safety, target engagement, and biomarker response in patients with ALS.  Eledon’s release stated: 

  • Tegoprubart was well-tolerated, with no drug-related serious adverse events
  • Dose dependent target engagement was demonstrated, and ALS associated pro-inflammatory biomarkers were both observed and significantly reduced in a dose dependent manner
  • Target engagement and level of pro-inflammatory biomarker reduction were associated with a trend in the slowing of disease progression as measured by ALSFRS slope when compared to a cohort from the ALS PRO-ACT database
  • Biomarkers significantly reduced included biomarkers also associated with IgA nephropathy and kidney allograft transplant rejection 

“I’m proud to be the Chairman at Augie’s Quest to Cure ALS and ALS TDI, and excited to see the next phase of development for tegoprubart – as our work is far from finished.  This milestone showcases ALS TDI’s ability to create quality therapeutics in their lab, and Augie’s Quest is proud to fund this critical science.  Together, we will find a cure for ALS.”  said Augie Nieto.  Augie’s Quest has been the lead funder of this critical science granting over $72 million to ALS TDI under Augie’s leadership. 

Tegoprubart is an antibody therapeutic targeting CD40L, a well-validated biologic target with a broad therapeutic potential.  The Phase 2a trial was a 12-week, open label, dose escalating, safety, and biomarker study.  The endpoints of the study were safety and tolerability, and changes in pro-inflammatory biomarkers.  

“Neuroinflammation is a driving force in the pathogenesis and progression of ALS. The ability to suppress inflammatory responses may translate into clinical benefit,” said Stanley H. Appel, MD, Co-Director of the Houston Methodist Neurological Institute and Chair of the Stanley H. Appel Department of Neurology at Houston Methodist. “These results reinforce the exciting potential of tegoprubart as a promising therapy for patients with ALS.”

After being invented at ALS TDI with funding from Augie’s Quest to Cure ALS, tegoprubart (previously called AT-1501) was licensed to Anelixis Therapeutics, a for-profit biotech company that oversaw the completion of a Phase 1 safety trial in 2019 (Augie Nieto was chairman of Anelixis). In 2020, Anelixis was acquired by Eledon Pharmaceuticals, a for-profit clinical-stage drug development company.  Eledon is developing precision therapies that target the CD40 Ligand pathway for use in organ and cellular transplantation, and for the treatment of autoimmune and neurodegenerative disease (including ALS).

“We are excited by the results from the phase 2 trial and by tegoprubart’s emerging clinical profile. The positive data represent a significant step in the development of tegoprubart as a potential treatment for people living with ALS,” said Steve Perrin, President and Chief Scientific Officer at Eledon Pharmaceuticals. “We thank all of the patients and families, as well as the investigators, who have supported us over the years for their commitment to finding a novel therapeutic option for ALS.”

Ending ALS will require many treatments to meet the needs of every individual living with this disease, and Augie’s Quest will continue to raise the funds and awareness urgently needed to advance cutting-edge research, fast-track effective treatments and ultimately, find a cure for ALS. 

For more information, please see Eledon’s full press release here:

Leave a Reply

ALS won’t stop and neither will Augie’s Quest

Augie’s Quest to Cure ALS
PO Box #9886
Denver, CO 80209

T: 949-506-1007

Copyright © 2022 Augie's Quest. All Rights Reserved.