After three and a half years of incubation at Third Rock Ventures, Abata Therapeutics is ready to unveil itself with $95 million in series A funds to bring cell therapies into the clinic for autoimmune and inflammatory diseases.
First up, a lead program using regulatory T cells (Tregs) for treatment of progressive multiple sclerosis (MS), which will have early safety and biomarker data in 2024, said Samantha Singer, Abata CEO and president, in an interview. By the end of 2025, Abata will have an MS program in the clinic, as well as programs for Type 1 diabetes and inclusion body myositis, the CEO said.
Third Rock is joined by ElevateBio, Lightspeed Venture Partners, Invus, Samsara BioCapital and the JDRF T1D Fund in the financing.
Tregs are the “ideal therapy for autoimmune disease” because they are locally activated, address various types of inflammation, release soluble factors that change the surrounding environment and have the potential to repair.
“This is a moment in time when the engineered cell therapy is now ready for primetime, and this is the right place to use it,” said Richard Ransohoff, M.D., chief medical officer and co-founder of Abata, in a joint interview with Singer.
Not to mention, Tregs last a long time, which is critical for chronic diseases like MS, according to Singer. Abata’s Tregs are derived from a patient’s own cells, which reduces the risk of rejection when the engineered cells are given back to the patient.
MS does not shorten life but typically hits patients in their 20’s and 30’s, which means the disease can impact a person for decades. During that time, the disease itself changes, as well as the person’s immune system. MS symptoms range from involuntary muscle spasms to vision problems to fatigue and numbness, among others that vary person to person. The disease damages nerve cells in the central nervous system, which impedes communication between the brain and body.
Over time, the immune reaction of a person with MS “tends to die away” and “little nests of immune cells” get compartmentalized into the meninges, a system of membranes within the central nervous system where spinal fluid circulates, Ransohoff said.
Current treatments are “incredibly effective for relapsing MS” but for progressive MS are unable to get into the meninges area or the treatments simply do not work there, said Ransohoff, who is also a Third Rock venture partner.
Existing treatments for MS range from oral drugs like Merck KGaA’s Mavenclad and Biogen’s Tecfidera and Vumerity; injections like Merck KGaA’s Rebif and Biogen’s Avonex; and infusions like Sanofi’s Lemtrada and Biogen’s Tysabri. Last month, the FDA rejected a subcutaneous formulation of Tysabri after Biogen had received approval for the drug in the EU the month prior.
Abata is confident that its lead candidate, once infused into the patient, will get to the meninges. The T-cell receptor (TCR) engineered by the Abata team will be used to recognize that “‘Yes, now I’m home, I’m going to take up residence here,'” Ransohoff said.
“The properties of the T-reg, which are it travels through the body looking for where it is supposed to settle down and the ability to give it a TCR that will settle it exactly where it needs to be for MS, and the characteristics of this progressive pathology, where you have lymphoid aggregates in this very special compartment of the body, within the meninges, that’s a perfect fit,” Ransohoff said.
Ransohoff is among a founding team that includes Diane Mathis, Ph.D., a Harvard Medical School professor who leads a T cell differentiation lab; Michael Birnbaum, Ph.D., an MIT assistant professor whose lab examines TCRs; Roland Martin, M.D., a University Hospital Zurich professor who leads an MS center focused on developing novel treatments for the disease; and Daniel Reich, M.D., Ph.D., a senior investigator within the National Institutes of Health’s neurological disorders division and a Johns Hopkins professor whose lab focuses on using MRI techniques to understand MS.
As part of ElevateBio’s partnership in the series A, Abata will collaborate with the company’s R&D and manufacturing center on process development and end-to-end manufacturing.
“Every kind of step in the process of creating a TCR Treg has been done in some way, shape or form, either with different types of cells in cancer therapies or in academic settings, so the trick is being able to do it end to end and do in a way that is tailored specifically to MS,” Singer said.
Tregs play a role in an immunotherapeutic protein candidate from HCW Biologics that is meant to treat autoimmune disorders. Tregs also contribute to an early-stage cancer immunotherapy asset from Agenus that has garnered the small biotech $200 million upfront and up to $1.36 billion in biobucks from Bristol Myers Squibb.
Senda Biosciences and Novartis have also made waves in MS recently. Novartis recently signed a collaboration pact, which could lead to a buyout, with Cellerys, a Swiss biotech conducting a phase 2 trial for an MS drug. Senda, which has an initial focus on metabolic diseases but additional programs around MS and Parkinson’s, closed a $98 million series B earlier this month.