Coya Raises $10M to Advance T-cell Therapy Now in Phase 2 ALS Trial

Coya Therapeutics has raised $10 million in funding to advance the development of ALS001, a potential regulatory T-cell (Treg) therapy aiming to halt amyotrophic lateral sclerosis (ALS) progression, now in a Phase 2 study in patients.

Proceeds raised in this series A funding round will also be used to introduce into testing similar treatments for other neurodegenerative disorders.

“Patients with neurodegenerative diseases are in desperate need of transformative therapeutic options; harnessing the neuro-protective effects of Treg cell therapy shows great potential in unlocking a new treatment paradigm and may enable us to revolutionize care for patients with devastating neurodegenerative diseases,” Stanley H. Appel, MD, co-director of Houston Methodist Neurological Institute, said in a press release.

Tregs are negative regulators of the immune system, meaning they work to shut down excessive inflammatory responses, such as those involved in nerve cell degeneration.

Previous work by Appel showed a link between ALS progression and low Treg levels and other problems with these immune cells in people with the disease, making them less effective at dampening inflammation, Coya stated in its release. He and his research team also discovered that isolating the cells could allow their repair.

The company, building on Appel’s research, developed a way to isolate and remove troubled Tregs from a patient, convert them to “highly functional and neuroprotective” cells, and then expend them in the lab into “billions” of Tregs to be returned to the patient via intravenous infusion, it reported on its website.

Using its cryopreservation for Tregs (CTreg) platform, the company also developed a way to freeze the expanded cells without losing effectiveness. This means, Coya stated, that it can generate enough Tregs for a year of treatment in a single manufacturing process, freeze them, and then thaw the cells as needed for regular, even monthly, infusion — essentially creating an “off-the-shelf” product tailored to each patient.

“The ability to manufacture and cryopreserve a 12-month patient’s supply of cells from one manufacturing run overcomes prior limitations and revolutionizes supply chain management, allowing for maintenance monthly infusions,” said Howard Berman, PhD, the company’s CEO.

A Phase 1 pilot trial (NCT03241784), sponsored by Appel, found ALS001 to be safe and well tolerated, and that treatment “slowed progression rates during early and later stages of disease” in its three enrolled ALS patients. Trial results were published in the journal Neurology in 2018, and the study was supported by a $2.5 million grant from the ALS Association, ALS Finding a Cure, and the Muscular Dystrophy Association (MDA).

An ongoing Phase 2a trial (NCT04055623) is now investigating the Treg therapy, given via monthly infusions for six months, against a placebo in 12 ALS patients. After this period, all those who finish this part will be invited to continue or begin receiving monthly ALS001 infusions in this study’s six-month, open-label extension.

The trial’s main goal is to determine changes over time in Treg function. Additional measures include Treg numbers, overall disease progression, and lung health. Data is expected in the next few months. The study, taking place at sites in Houston and Boston, is expected to conclude in September 2024.

In addition to ALS, Coya is working on similar treatment approaches for other neurodegenerative and autoimmune diseases in which defective Tregs are thought to also play a role. These include Parkinson’s disease, Alzheimer’s disease, frontotemporal dementia, and multiple sclerosis.

“Through our sponsored research program in conjunction with Dr. Appel, we look forward to continuing advancement of this promising work and translating this work into a meaningful therapy for patients,” Berman added.