Tegoprubart (AT-1501) | ALS Therapy Development Institute

Tegoprubart (formerly known as AT-1501) is the first potential ALS treatment to be invented by a nonprofit biotech, the ALS Therapy Development Institute (ALS TDI), and make it to human clinical trial.

Tegoprubart (antiCD40L) is an antibody therapeutic with comprehensive and promising preclinical data. It blocks specific immune cell activation and protects nerves against the progression of ALS and Alzheimer's disease. Tegoprubart successfully completed a Phase 2a clinical trial in May of 2022.

Learn More

Tegoprubart

(AT-1501)

Learn More

About Tegoprubart

As a nonprofit biotech, the ALS TDI’s sole mission is to develop treatments for ALS. Our lab focuses on all areas of preclinical drug development so that we can identify potential treatments and hand them off to be moved into clinical development.

ALS TDI has validated several drugs to help them get moved into trials, but tegoprubart is the first treatment that was actually invented by ALS TDI scientists and moved into trials.

Tegoprubart is a novel antibody which acts in a highly targeted, disease-specific way to tamp down the immune system.

After being invented at ALS TDI, tegoprubart was advanced through clinical trials by Anelixis Therapeutics, a for-profit clinical-stage development company. In 2019, Anelixis successfully completed phase 1 trials of tegoprubart. In 2020, Anelixis was acquired by Novus Therapeutics (now Eledon Pharmaceuticals), a publicly traded company. Through this acquisition, tegoprubart is expected to advance to the next stage of clinical development, a phase 2a trial.

ALS TDI is extremely hopeful that tegoprubart will continue to be advanced through trials for ALS, and that it will eventually be made available as a treatment for people living with ALS today.

Invention of tegoprubart

Many years of research and learnings contributed to the development of tegoprubart. The timeline below demonstrates what ALS TDI scientists did to advance tegoprubart through preclinical drug development. All of this work was funded by donations from the community.

The following milestones shows the steps that went in to the invention of tegoprubart by ALS TDI scientists.

After ALS TDI conducted the world's largest gene expression analysis of the SOD1 mouse model, they discovered a key innate immune system pathway (CD40L) to be over-active and increasing in activity across multiple tissues related to ALS disease progression.
Following these findings, ALS TDI began rigorously testing compounds to see if they could regulate the activity of the CD40L pathway.
In 2010, ALS TDI published, From transcriptome analysis to therapeutics anti-CD40L treatment in the SOD1 model of amyotrophic lateral sclerosis, in Nature Genetics. Their work showed that the compound they invented, antiCD40L, was able to slow down disease progression, improve body weight retention over time and increase overall survival in mouse model.
In 2011, ALS TDI began to study preclinical mechanism of action and dose ranging for antiCD40L.
ALS TDI performed hundreds of additional experiments to understand how blocking CD40L worked, and how best to optimize the drug as a potential treatment for ALS.

Development of tegoprubart

Bringing a drug from discovery through clinical trials is a long, expensive process that often requires resources beyond those available to nonprofit research organizations. While ALS TDI specializes in inventing and advancing potential therapies, later-stage development typically requires significant investment in manufacturing, regulatory activities, and large clinical studies.

To continue advancing tegoprubart, ALS TDI helped establish Anelixis Therapeutics, a clinical-stage biotechnology company focused on developing the program beyond the laboratory. This approach enabled tegoprubart to attract the capital and infrastructure needed to move into human clinical trials while allowing ALS TDI to remain focused on its core mission of discovering and developing new treatments for ALS.

The following milestones highlight tegoprubart's development journey:

2019: Anelixis completed a Phase 1 clinical trial in healthy volunteers and people living with ALS.
2020: Anelixis was acquired by Novus Therapeutics (now Eledon Pharmaceuticals), which announced its intention to continue the clinical development of tegoprubart, including studies in ALS.
November 2020: Novus announced the initiation of enrollment for a Phase 2a clinical trial in ALS.
January 2021: Novus changed its name to Eledon Pharmaceuticals and reaffirmed its commitment to the ongoing Phase 2a ALS study.
May 2022: Eledon announced results from the Phase 2a study, reporting that tegoprubart was safe and well tolerated in people living with ALS and demonstrated evidence of target engagement in humans.

ALS TDI remains proud of its role in inventing and advancing tegoprubart. The program represents an important milestone in ALS drug discovery and demonstrates how nonprofit research can help move promising therapeutic ideas from the laboratory toward clinical development. Consistent with ALS TDI's mission, any financial returns generated through its research programs are reinvested in efforts to discover and develop treatments for ALS.

Tegoprubart Funding and Support

Funded by the Community

The charitable support that ALS TDI received from tens of thousands of individuals over the years has been the primary source of funding for the research behind the early discoveries and invention of tegoprubart.

Thanks to our community members for holding fundraisers, throwing beanbags, toasting Ales for ALS, riding in the Tri-State Trek, donating over and over again, and continually asking friends and family to help.

Additional funding has come from Augie's Quest, ALS Association, ALS Finding a Cure, ALS ONE and the Department of Defense CDMRP, among others.

Let's keep going.
We know that treating, and ultimately curing, ALS will require multiple therapies. The research at ALS TDI is moving faster, but we can't do anything without your help.

Have additional questions about the invention and development of tegoprubart? Read our FAQ

Help Fund Treatments

“I will continue supporting ALS TDI's expanding pipeline of potential treatments knowing now that with their scientists and our support, dreams can become reality.”

- Andrea Peet, diagnosed with ALS in 2014

Where is tegoprubart Now?

The first in-human clinical trials of tegoprubart (formerly AT-1501) were completed in 2019, sponsored by Anelixis Therapeutics. In 2020, Anelixis Therapeutics was acquired by Novus Therapeutics (now Eledon Pharmaceuticals). Through Eledon Pharmaceuticals, tegoprubart is being developed as a potential treatment in ALS, renal transplantation, islet cell transplantation, autoimmune nephritis. Eledon began enrollment for Phase 2a trials in ALS in 2020.

In May 2022, Eledon Pharmaceuticals announced that tegoprubart had completed phase 2a trials. The press announcement reported that the drug was demonstrated to be safe and well-tolerated in people with ALS and that the drug is hitting its target pathways in humans.

Additional information about tegoprubart can be found at www.eledon.com.

What Do I

Do Next?