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What is AT-1501?

AT-1501 is a novel antibody which acts in a highly targeted, disease-specific way to tamp down the immune system. Invented at the ALS Therapy Development Institute (ALS TDI), this potential therapeutic has been screened in more preclinical efficacy experiments than any other compound proposed in the history of the battle to end ALS.

AT-1501 was invented by the ALS Therapy Development Institute, and in 2019, Anelixis Therapeutics successfully completed phase 1 clinical trials for AT-1501 as a treatment for ALS. The study, involving both healthy volunteers and participants with ALS, showed that AT-1501 was well tolerated at all doses tested and that the pharmacokinetic properties of AT-1501 were typical for an IgG1 antibody therapeutic.

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 Anelixis Therapeutics.

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Steve Perrin, Ph.D., CEO at ALS TDI
“AT-1501 is the most effective treatment tested at the ALS Therapy Development Institute.”
- Steve Perrin, Ph.D., CEO at ALS TDI

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 development 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.
Andrea Peet, diagnosed with ALS in 2014
“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
Timeline Timeline
AT-1501 Timeline
• First in-human clinical trials began following a successful submission of Investigational New Drug application to the FDA.
• Significant progress made in the lyophilization process for the antibody. Lyophilization involves turning drug from liquid form to powder, as powder is more stable.
• Non-human primate studies are conducted and confirm safety of AT-1501.
• Pre-Investigational New Drug application submitted to the FDA.
• A clinical advisory board is formed. Walter Ogier joins Anelixis as Executive Chairman.
• A large scale manufacturing of AT-1501 results in a sufficient yield for clinical trials. A scale up and long-term stability test confirms readiness of material. Early steps are taken to achieve a liquid formulation strategy for the drug.
• Anelixis, ALS TDI and Lonza work together to develop and select a master cell line that will produce AT-1501 for human clinical trials. A successful cell line is found to be sucessful producing high quality quantities of antibody. Master cell line is banked at Lonza.
• Lonza Biologics is named as the manufacturer of AT-1501.
• Settled on final human antibody. Lead cell line selection and safety experiments at Lonza conducted.
• Preclinical studies continue.
• Anelixis Therapeutics, LLC established by ALS TDI to access capital needed to advance AT-1501 (antiCD40L) through clinical trials.
• Preclinical studies continue at ALS TDI. This work required hundreds of additional experiments to understand what AT-1501 treatment does and how best to optimize the drug as a potential treatment for ALS.
• Preclinical mechanism of action and dose ranging studies begin at ALS TDI.
• ALS TDI publishes From transcriptome analysis to therapeutics anti-CD40L treatment in the SOD1 model of amyotrophic lateral sclerosis in Nature Genetics. Work shows preclinical antiCD40L treatment slows down disease progression, improves body weight retention over time and increases overall survival in mouse model.
• ALS TDI begins rigorously testing compounds to regulate the activity of the CD40L pathway.
• Mouse antibodies brought in-house for testing in the SOD1 mouse model.
• ALS TDI discovers a key innate immune system pathway (CD40L) to be over-active and increasing in activity overtime across multiple tissues (spinal cord, muscle and sciatic nerve) related to ALS disease progression.
• ALS TDI conducts world's largest gene expression analysis of the SOD1 mouse model.

Where is AT-1501 Now?

Thanks in large part to the generosity of ALS TDI donors, first in-human clinical trials of AT-1501 started in 2018, sponsored by Anelixis Therapeutics. Anelixis Therapeutics was created by ALS TDI to raise the funding needed to bring AT-1501 through clinical trials and to explore other revenue generating opportunities specifically for the advancement of AT-1501. At this time, ALS TDI remains the majority shareholder of Anelixis Therapeutics.