AT-1501 | ALS Therapy Development Institute

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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.

AT-1501 (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. AT-1501 successfully completed a Phase 1 clinical trial in 2019 and is now heading into a Phase 2 study.

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About AT-1501

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 AT-1501 is the first treatment that was actually invented by ALS TDI scientists and moved into trials.

AT-1501 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, AT-1501 was advanced through clinical trials by Anelixis Therapeutics, a for-profit clinical-stage development company. In 2019, Anelixis successfully completed phase 1 trials of AT-1501. In 2020, Anelixis was acquired by Novus Therapeutics, a publicly traded company. Through this acquisition, AT-1501 is expected to advance to the next stage of clinical development, a phase 2 trial.

ALS TDI is extremely hopeful that AT-1501 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 AT-1501

Many years of research and learnings contributed to the development of AT-1501. The timeline below demonstrates what ALS TDI scientists did to advance AT-1501 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 AT-1501 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 AT-1501

Bringing a drug from the lab and into clinical trials is extremely risky and expensive. For this reason, when ALS TDI discovers or invents a promising treatment, it then needs to be handed off to an external company that has the funding to take it through trials.

In the case of AT-1501, Anelixis Therapeutics was established to further develop AT-1501. The following milestones show how AT-1501 progressed after leaving ALS TDI's lab.

As the first nonprofit biotech in any disease to attempt to move a drug invented in their lab into clinical trials, ALS TDI realized that they would need to find or establish a for-profit clinical stage development company to secure funding for trials.
In order to access capital needed to advance AT-1501 (antiCD40L) through clinical trials, ALS TDI established Anelixis Therapeutics as a for-profit clinical stage development company.
Anelixis completed a phase I trial in healthy volunteers and people with ALS in 2019.
In 2020, Anelixis was acquired by Novus Therapeutics. A press released announced that Novus is committed to further clinical studies of AT-1501 in people living with ALS.

AT-1501 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 AT-1501.

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 AT-1501? Read our FAQ

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“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 AT-1501 Now?

The first in-human clinical trials of AT-1501 were completed in 2019, sponsored by Anelixis Therapeutics. In 2020, Anelixis Therapeutics was acquired by Novus Therapeutics. Through Novus Therapeutics, AT-1501 is being developed as a potential treatment in ALS, renal transplantation, islet cell transplantation, autoimmune nephritis. ALS will be the first of these indications to begin enrolling for Phase 2 trials, and enrollment is expected to begin by the end of 2020.

Additional information about AT-1501 can be found at www.novustherapeutics.com.

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