AT-1501 is the first drug to be invested by ALS TDI scientists and be moved out of the lab and into clinical trials. Below, ALS TDI has provided answers to some frequently asked questions about the invention and development of AT-1501.

How did ALS TDI discover AT-1501 in its drive to find treatments for ALS?

The science behind the invention of what is now called AT-1501 originated in our lab at ALS TDI. Our scientists performed preclinical research to discover CD40L as a therapeutic target for ALS, then to invent the molecule that is now AT-1501, and followed up with exhaustive testing to optimize preclinical efficacy of this potential treatment.


What is the standard approach taken by ALS TDI to scale up the treatments it discovers, such as AT-1501?

As a research institute/non-profit biotech, a key element of our strategy to end ALS has always been to conduct the preclinical research to find potential treatments. In order to advance treatments that we discover (like the molecule for AT-1501) out of our lab, we can then license them to a for-profit company, which can take on the responsibility of setting up a manufacturing process to make the drug in significant quantity, completing safety studies in animal models that are required by the FDA, and then advancing these treatments through clinical trials. This is a necessary step, as bringing a drug from the lab and into clinical trials is extremely expensive. 


How did ALS TDI go about developing AT-1501?

ALS TDI was the first nonprofit biotech in any disease state to attempt to move a drug invented in its own lab and into clinical trials. Early on we realized that we would need to find or establish a for-profit clinical stage development company to secure funding for the drug manufacturing, safety studies, and the many phases of clinical trials.

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. Funding for research has also come from organizations like Augie's Quest, ALS Association, ALS Finding a Cure, ALS ONE and the Department of Defense CDMRP, among others.


Why was Anelixis created and how did Anelixis further develop AT-1501?

ALS TDI started Anelixis as a for-profit subsidiary in order to advance AT-1501 through the necessary steps to make it available for people with ALS. ALS TDI licensed AT-1501 to Anelixis so that Anelixis could take the various steps needed to develop the drug and move it forward to clinical trials. After its inception, Anelixis then took on the responsibility of finding investors to move the molecule forward. The funds that were invested to advance AT-1501 through manufacturing, safety studies, and Phase 1 clinical trials, were secured by Anelixis and did not come from ALS TDI.
This was the first time ALS TDI went through the process of moving a discovery out of their lab and working to see that it successfully made it into pharmaceutical development.


Does Anelixis still exist?

Anelixis’ goal from its inception was to seek the financial support needed to move AT-1501 through clinical trials and to make it available as a treatment for people with ALS. In 2019, Anelixis moved AT-1501 through Phase 1 trials and had promising results. Additional funding was needed to move AT-1501 to phase 2 trials. In 2020 Anelixis was acquired by Novus Therapeutics, a publicly traded company. Novus has stated that the proceeds from the acquisition will be used to advance Phase 2 clinical trials of AT-1501. Anelixis now exists as a subsidiary of Novus Therapeutics.


Did ALS TDI gain financially from the Novus acquisition of Anelixis?

ALS TDI received no funds from the acquisition. ALS TDI may have potential to benefit financially if and when AT-1501 goes to market, however, this may not be for many years.  If so, these funds would be used to help move additional research forward.


What happened to the money from the acquisition, and how will it be used going forward?

Novus has stated that they money raised in the Anelixis/Novus transaction will be used to fund the company operations and the AT-1501 phase 2 clinical trials.


What is ALS TDI’s mission now following the acquisition of Anelixis, and who’s in charge?

Our vision and mission remain unwavering – to help find a cure for ALS – and our leadership structure remains intact. Steve Perrin has been performing the dual role of ALS TDI chief executive officer and Anelixis CEO for the past 7 years. We anticipate that his role at ALS TDI will remain, and he is passionate about solving a cure for ALS. Meanwhile, Fernando Vieira serves as our Chief Scientific Officer, leading a group of talented and highly motivated scientists, and Bashar Alnakhala continues to lead our operations team, serving as Chief Operations Officer.

As a nonprofit biotech, ALS TDI knows that the processes and transactions that took place were needed to get AT-1501 closer to being available for people with ALS. Our goal is to continue to advance more potential treatments, as our mission is to help find treatments for everyone living with ALS as quickly as possible.


Besides AT-1501, what else has ALS TDI been working on?

In addition to the advancement of AT-1501, our work this year has produced additional promising results, with highlights including:

  • Identify and optimize lead drug candidates by leveraging our existing world-class ALS preclinical pharmacology program.
  • Work to reveal potential ALS treatments by applying induced pluripotent cell-based assay systems for screening compound libraries.
  • Identify drug candidates to treat C9orf72 and potentially other subtypes of ALS by advancing research of Type I Protein Arginine Methyltransferases (PRMTs), a new therapeutic target discovered by ALS TDI scientists.
  • Accelerate the clinical drug development process by leveraging learnings from the Precision Medicine Program to learn about ALS from people with ALS.


What are the biggest priorities for ALS TDI in the near and long-term future?

We continue to be laser focused on research and development for innovative and ground-breaking ALS treatments. We value our ALS community of supporters, partners and advocates as much now as ever before. With continued funding, we know we’ll have more discoveries, inventions and success stories to share with the ALS community. s to get involved or make a donation to fund ALS research.