1st Abata Candidate Will Be T-cell Therapy ABA-101 for Progressive MS
Now in early studies, MS treatment expected to enter trials in 2024
The therapy is now in early studies that aim to support an investigational new drug application or IND — a formal request to U.S. regulatory authorities asking for permission to start testing the therapy in humans. ABA-101 is on track to enter clinical trials in 2024, according to Abata.
The company’s platform is focused on regulatory T-cells, or Tregs — a specialized group of anti-inflammatory immune cells, which normally help to “put the brakes” on excess inflammation in the body.
“Since our inception, we’ve been acutely focused on advancing transformative therapies for people with autoimmune diseases,” Samantha Singer, president and CEO of Abata, said in a company press release.
“We believe that our Treg product engine has yielded a candidate in ABA-101 that is truly differentiated and can have significant benefit for people living with progressive MS,” Singer said.
A new treatment for progressive MS
Like other T-cells, Tregs are equipped with a specialized protein receptor that recognizes a specific molecular target with very high specificity. In most T-cells, the receptor binding to its target triggers the cell to launch an inflammatory attack. But when a Treg’s receptor binds to its target, it triggers the cell to secrete anti-inflammatory signaling molecules to reduce inflammation.
“Our approach of leveraging the natural role of Tregs in the immune system enables us to resolve complex inflammatory mechanisms, offering new hope for patients with no treatment options,” said Richard M. Ransohoff, MD, co-founder and chief medical officer at Abata, and a venture partner at Third Rock Ventures.
In MS, inflammation causes damage to the myelin sheath, a fatty covering around nerve fibers that helps them send electrical signals. ABA-101 basically involves harvesting Tregs from a patient, then modifying the cells so they have a receptor that will bind to myelin fragments. The cells would then be infused back into the patient.
The idea is that the modified Tregs will be activated by damaged myelin, cuing an anti-inflammatory Treg response that can reduce the inflammation driving MS.
“ABA-101 utilizes a [T-cell receptor] to target our product specifically to the site of ongoing pathology and to offer the potential for a robust, highly durable effect,” Singer said.
The candidate will be developed as a treatment for people with progressive, non-relapsing forms of MS. There are far fewer approved therapies for these patients than are available for individuals with relapsing forms of the disease.
It is specific for patients who have a DRB1*15:01 haplotype — a genetic variation that affects immune function and has been linked with an increased risk of developing MS. According to Abata, this covers about 45,000 patients in the U.S.
“The progressive pathology of MS has not been effectively treated because existing therapies don’t address the [central nervous system]-compartmentalized inflammation that drives that aspect of the disease,” Ransohoff said.
Abata has collaborated with ElevateBio BaseCamp to develop a manufacturing process that engineers and grows Tregs for therapeutic use.
“Working with our partners at ElevateBio BaseCamp, we’ve rapidly established a foundational manufacturing process in less than one year, showing we can successfully engineer and expand Tregs for use as therapies.”
Singer said the work “gives us great confidence … in our ABA-101 program.”
Michael Paglia, MS, chief operating officer at ElevateBio BaseCamp, said his company is looking forward to its “continued partnership” with Abata.
“It’s been a pleasure working with the Abata team creating and refining a manufacturing process that enables them to expand Tregs engineered with an antigen-specific [T-cell receptor] at yields that will support clinical development of ABA-101 and serves as a template for their future programs,” Paglia said.