Tegoprubart (formerly known as AT-1501) is the first drug to be invented by ALS TDI scientists and 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 tegoprubart.

Where is tegoprubart now?

The first in-human clinical trials of tegoprubart 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 2 trials in ALS in 2020.

In May 2022, Eledon Pharmaceuticals announced that Tegoprubart had completed phase 2a trials. The press announcement reported data showing 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.

How did ALS TDI discover tegoprubart in its drive to find treatments for ALS? 

The science behind tegoprubart 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 tegoprubart to target CD40L.

How does ALS TDI bring treatments it discovers, such as tegoprubart, 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 tegoprubart, 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 tegoprubart?

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 tegoprubart. 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 tegoprubart?

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 tegoprubart 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 tegoprubart, 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 tegoprubart, 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 tegoprubart 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 would be used to fund the company operations, including the tegoprubart phase 2 clinical trials of which ALS is the first. In May 2022, Eledon announced that tegoprubart successfully completed a phase 2a trial. Read more here.

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. 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 tegoprubart, what else has ALS TDI been working on?

In addition to the advancement of tegoprubart, 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.
  • Publishing a paper with Google on the development of new digital biomarkers of ALS.

Read details about our recent accomplishments and goals here.

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.