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 AT-1501 originated in our lab at ALS TDI. Our scientists performed preclinical research to discover the CD40L protein as a therapeutic target for ALS and to invent the molecule that is now called AT-1501 to target CD40L.

How does ALS TDI bring treatments it discovers, such as AT-1501, to clinical development?

ALS TDI is the first and largest nonprofit biotech completely focused on ALS research. A key element of our strategy to end ALS has always been to fill existing ALS research gaps by conducting our own preclinical research to find potential treatments. Moving a drug through human clinical trials, regulatory approval and manufacturing phases, however, is prohibitively expensive for a nonprofit and requires for-profit capital. Transactions like those associated with AT-1501, where ALS TDI discoveries and inventions can be licensed with independent drug development partners, ensures that there is a path for new drug development and hopefully regulatory approval.


How did ALS TDI go about developing AT-1501?

ALS TDI was the first nonprofit biotech in ALS to attempt to move a drug invented in its own lab into 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 including 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 created Anelixis as a for-profit subsidiary to secure funding needed to advance lab discoveries through the early stages of drug development. Funds invested to advance AT-1501 through manufacturing, safety studies, and Phase 1 clinical trials, were raised privately by Anelixis and were not contributed by ALS TDI.

Why was Anelixis acquired by Eledon Pharmaceuticals in September 2020?

Developing a potential drug is prohibitively expensive for a nonprofit. In order to move research discoveries and inventions, like AT 1501, beyond the preclinical stage, large-scale for-profit investment is necessary to pay for the manufacturing, safety studies, and the many phases of clinical trials.

After successfully completing Phase 1 trials of AT-1501, Anelixis agreed to be acquired by Eledon in a transaction which in parallel raised over $108 million from new investors primarily for the future development of the drug. The transaction was approved by ALS TDI as an Anelixis shareholder. The licensing agreement with ALS TDI provides for milestone payments and royalties if the drug is successfully developed. These funds would help to further ALS TDI’s nonprofit mission to find treatments for everyone living with ALS.

Dr. Steve Perrin and Anelixis Chairman of the Board Walter Ogier were named to Eledon’s Board of Directors. Through their participation, Eledon’s decisions related to future drug development will continue to be informed by ALS TDI’s history and mission.


Did ALS TDI profit from the Eledon acquisition of Anelixis?

All Anelixis shareholders, including ALS TDI, received Eledon stock in exchange for their Anelixis holdings, and we continue to be invested in Eledon as a minority shareholder. ALS TDI did not receive a cash payment as a result of this transaction, and the Eledon shares held by ALS TDI constitute restricted securities. This means that ALS TDI is subject to restrictions relating to the frequency that it may sell its Eledon shares and the volume of Eledon shares that it may sell on the stock market. ALS TDI also controls certain patent rights to AT-1501 which are licensed to Anelixis for the purpose of drug development and commercialization that is needed to make it available to the public. ALS TDI may receive license fees and royalties under the License Agreement at various stages in the drug development process.

As a 501(c)(3), ALS TDI invests all funds to fulfill our charitable mission. Funds from future AT-1501 milestones and royalties, as well as from any sale of Eledon shares, would be re-invested in ALS research and further ALS TDI’s nonprofit mission to find treatments for everyone living with ALS.


What happened to the money from investors in the financing associated with the Eledon acquisition, and how will it be used going forward?

Eledon has stated that money raised from investors to support the Anelixis transaction will be used to fund the company operations, including the AT-1501 phase 2 clinical trials of which ALS is the first.
What is ALS TDI’s mission now following the acquisition of Anelixis, and who’s in charge?

Our vision and mission remain unwavering – to end ALS – and our leadership structure reflects this commitment. The ALS Therapy Development Institute (ALS TDI) Board of Directors appointed Fernando G. Vieira, M.D., President, Chief Executive Officer, and Chief Scientific Officer of ALS TDI during its April 7, 2021 meeting.


As Chief Scientific Officer since 2018, Dr. Vieira has overseen ALS TDI’s research team of 30 full-time professionals in their efforts to discover targets, drugs, and biomarkers to slow, halt, and end ALS. He received his medical degree from Harvard Medical School and a Bachelor’s degree in Biological Engineering from the University of Florida.


In his new role as CEO, Dr. Vieira will assume strategic leadership of the organization while also maintaining the dual role of CSO. He will be responsible for ensuring that ALS TDI continues to advance its mission to discover and develop effective therapeutics for everyone impacted by ALS. He leading a group of talented and highly motivated scientists, and Bashar Alnakhala continues to lead our operations team, serving as Chief Operations Officer.


Dr. Steve Perrin continues to serve as ALS TDI Vice Chair and a Director. He remains passionately committed to furthering our mission.

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

Besides AT-1501, what else has ALS TDI been working on?
  • In addition to the advancement of AT-1501, our work this past 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?

Ending ALS will require many treatments to meet the needs of every individual living with this disease. 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. To get involved or make a donation to ALS research, visit als.net.