Several ALS organizations announced on Wednesday that the first participant was successfully given a dose of AT-1501 in the first in-human trial of the potential treatment for ALS. AT-1501 was developed by the ALS Therapy Development Institute (ALS TDI), with support from The ALS Association, ALS ONE, and ALS Finding a Cure® , and is being brought into trials by Anelixis Therapeutics, Inc., a for-profit subsidiary of ALS TDI.
The phase 1 safety trial of AT-1501 is enrolling healthy volunteers and people living with ALS, following the acceptance of an Investigational New Drug (IND) application by the Food & Drug Administration (FDA). A total of 8 people with ALS will receive AT-1501 in this study, with the aim of determining safety and tolerability of the experimental antibody. The trial will also determine the pharmacokinetics of AT-1501, i.e. how the drug moves within the body.
AT-1501 is thought to block a specific immune system target expressed on lymphocytes (white blood cells), called CD40LG, which is known to drive neurodegeneration. Published research by ALS TDI has implicated a disease mechanism in the disease progression of patients with ALS, which may be blocked by the immune modulatory effects of anti-CD40 ligand monoclonal antibody therapy.
"We are excited to work closely with Anelixis to accelerate bringing AT-1501 to people with ALS," said Dr. Merit Cudkowicz, Director of the Healey Center for ALS at Mass General Hospital, and Chief Medical Officer for ALS Finding a Cure®. "The ALS ONE-supported trial design team members at the Healey Center are already working together with Anelixis to design the next trial to assess longer term safety and efficacy. It is critical to move faster from exciting lab-based scientific discovery to clinical trials in our patients."
On Wednesday, The ALS Association announced it had awarded an additional $1 million to Anelixis to help fund the trial. "We are proud to partner with Anelixis and other ALS organizations to help advance this promising compound into clinical trials," said Calaneet Balas, President and CEO of The ALS Association. "Through this new funding, we are continuing our support of novel, early stage potential treatments into human clinical trials."
"The support from other ALS organizations for this trial has been crucial to its successful launch," said Steve Perrin, Ph.D. President & CEO of Anelixis Therapeutics. Perrin also serves as the President & CEO of the ALS Therapy Development Institute. "Today's dosing of the first person with ALS is a significant milestone in determining the potential utility of this approach in battling back this horrific disease."
If safety is achieved, a next step forward could be a phase IIA safety study to further assess the safety, tolerability and pharmacokinetic profile of AT-1501, along with exploratory biomarker studies for example. However, the company has made no announcements regarding next steps, rather, staying focused on completing enrollment and conducting analysis on data collected through this initial phase 1 study.
About The ALS Association
The ALS Association is the only national nonprofit organization fighting Lou Gehrig's Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The ALS Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.alsa.org.
About ALS Finding a Cure®
ALS Finding a Cure® is dedicated to being a game changer in discovering a cure for this fatal disease. Founded in 2014 by noted philanthropist and Conair Founder and Chairman, Leandro P. Rizzuto, as a tribute to Christie Rizzuto, his daughter-in-law who was diagnosed with ALS in 2009 at the age of 41, and under the leadership of Denis Rizzuto, Peter N. Foss, and a team of renowned ALS researchers, ALS Finding a Cure® is focused on identifying the gaps in the scientific understanding of ALS that are preventing the development of a cure. The organization, a program of The Leandro P. Rizzuto Foundation, collabora...
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