The ALS Therapy Development Institute (ALS TDI) is excited to share a significant step in the advancement of AT-1501, a drug invented at ALS TDI, as a potential treatment for people with amyotrophic lateral sclerosis (ALS). Novus Therapeutics, Inc., a clinical stage biopharmaceutical company, announced today that the first subject has been enrolled in the Phase 2a clinical trial evaluating AT-1501 in adults.

In early September, Anelixis Therapeutics, the for-profit clinical-stage development company that advanced the drug AT-1501 to a Phase 1 clinical trial, announced that it had been acquired by Novus Therapeutics, which is now committed to further clinical studies of AT-1501 in people living with ALS.

This recent news regarding Phase 2a clinical trial enrollment is evidence of this commitment.

“We are excited to see that Novus Therapeutics has remained committed to advancing AT-1501 through Phase 2a clinical trials for ALS. We are hopeful that AT-1501, this drug invented at ALS TDI, will demonstrate requisite safety and evidence of biomarker modulation to inform next steps of clinical development. The objective of our science program is to advance more promising treatments like AT-1501 into ALS clinical development as quickly as possible.” said Fernando G. Vieira, M.D., ALS TDI’s Chief Scientific Officer.

As a research institute/non-profit biotech, a key element of ALS TDI’s strategy to end ALS has always been to conduct preclinical research to identify potential treatments. AT-1501 was invented by ALS TDI, and in 2019, Anelixis Therapeutics successfully completed phase 1 clinical trials for AT-1501 as a treatment for ALS.

Charitable support that ALS TDI has received over the years through donations and fundraisers has been an important source of funding for the research behind the early discoveries and invention of AT-1501. Vital funding has also come from organizations such as Augie's Quest, ALS Association, ALS Finding a Cure, ALS ONE and the Department of Defense CDMRP, among others.

ALS TDI did not receive any financial benefits from the Novus acquisition of Anelixis and continues to rely on funding from the community to advance more potential treatments for ALS. The advancement of AT-1501 provides hope that, with support from the community, more potential treatments can be advanced from ALS TDI’s lab and into people with ALS.

In a press release today, Novus announced that the AT-1501 Phase 2a trial in ALS is a 12-week, open label, dose escalating, safety and biomarker study. The endpoints of the study are safety and tolerability, and changes in pro-inflammatory biomarkers as well as neurofilament light chain. Exploratory clinical endpoints will also be assessed.

“As this recent announcement has shown, Novus is committed to providing people living with ALS and their families with a therapeutic solution to treat this progressive and devastating disease as expeditiously as possible,” said Steven Perrin, Ph.D., Chief Executive Officer of ALS TDI.

AT-1501 is the first potential treatment to be invented at ALS TDI and successfully advanced through a Phase 2 clinical study. Scientists at ALS TDI are continuing their efforts to discover additional potential treatments for people with ALS.

As a nonprofit biotech, ALS TDI is pleased to see that partnerships have made it possible to bring drugs like 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.

About the ALS Therapy Development Institute
The ALS Therapy Development Institute (ALS TDI) and its researchers discover and develop potential treatments for ALS. It is the world’s first and largest nonprofit biotech focused 100 percent on ALS research. Led by drug development experts and people with ALS, ALS TDI understands the urgent need to slow and stop this disease.

Based in Cambridge, MA, ALS TDI has more than 30 full-time, industry trained, drug development experts on staff. ALS TDI is internationally recognized as a leader in preclinical and translational ALS research, and partners with pharmaceutical companies and biotech organization1s all around the world. Rated a four-star nonprofit on Charity Navigator, ALS TDI spends 87 cents of every dollar raised on finding effective treatments and cures for ALS. Visit for more information.

About AT-1501
AT-1501 is a humanized IgG1 anti-CD40L antibody with high affinity for CD40L, a well-validated target with broad therapeutic potential. The CD40/CD40L pathway plays a central role in generating pro-inflammatory responses in autoimmune disease, allograft transplant rejection, and neuroinflammation. In a Phase 1 safety study of healthy volunteers and patients with ALS, AT-1501 was well tolerated at all doses tested.