CAMBRIDGE, MA– Progentos Therapeutics, a Cambridge-based biotechnology company developing regenerative treatments for multiple sclerosis (MS) and other neurological disorders, has administered the first dose of its experimental drug PRO-1562 in a Phase 1/2a clinical trial aimed at evaluating its potential to repair damaged nerve insulation.
The investigational oral medication is designed to promote remyelination, a process that restores myelin, the protective coating surrounding nerve fibers in the central nervous system. Damage to myelin is a defining characteristic of multiple sclerosis and can disrupt communication between the brain and the rest of the body.
Unlike existing multiple sclerosis treatments that primarily target the immune system to reduce inflammation and prevent relapses, PRO-1562 is being developed to repair neurological damage by stimulating the body’s natural myelin-producing cells.
The clinical trial will evaluate the drug’s safety, tolerability, and ability to promote myelin regeneration in patients with multiple sclerosis.
The Phase 1 portion of the study is being conducted at a single clinical research site in Australia. It is a randomized, double-blind, placebo-controlled trial involving healthy adults who will receive increasing single doses of PRO-1562.
Researchers will examine the drug’s safety profile, how it is absorbed and processed by the body, and its ability to penetrate the central nervous system.
The Phase 2a portion will involve adults with relapsing multiple sclerosis and chronic optic neuropathy, a condition involving damage to the optic nerve.
That six-month study will evaluate different doses of PRO-1562 administered once monthly, comparing the treatment with a placebo.
Researchers will use magnetic resonance imaging (MRI), electrophysiological testing, and clinical assessments to determine whether the drug can promote remyelination and improve neurological function.
“This milestone is a significant step forward in translating promising remyelination science into a potential therapy for people living with MS,” said Paul Frohna, M.D., Ph.D., Chief Medical Officer of Progentos Therapeutics. “Disease-modifying immunomodulatory therapies have transformed the treatment of MS by reducing relapses and controlling inflammatory disease activity, yet many patients still accumulate neurological damage and disability over time. Today, there are no approved therapies to regenerate lost myelin. The preclinical data with PRO-1562 suggest that our novel remyelinating approach has the potential to fundamentally alter the treatment paradigm for patients with MS and improve their long-term prognosis.”
Multiple sclerosis is a chronic immune-mediated disease in which the immune system attacks myelin, causing inflammation and damage to nerve fibers in the brain and spinal cord.
As the disease progresses, the loss of myelin can interfere with nerve signaling and contribute to problems involving movement, sensation, vision, and cognitive function.
Although existing disease-modifying therapies can reduce relapses and inflammatory activity, they do not directly restore myelin that has already been lost.
Progentos is developing PRO-1562 to address that treatment gap by encouraging the body’s existing oligodendrocyte precursor cells to develop into mature oligodendrocytes, the specialized cells responsible for producing myelin.
In preclinical studies, the experimental compound stimulated oligodendrocyte development and promoted remyelination in models of chronic myelin damage.
The company also reported increased myelination in both white and gray matter, suggesting the treatment could potentially support sensory, motor, and cognitive functions.
Preclinical research demonstrated activity across multiple species, along with what the company described as an encouraging preliminary safety profile.
However, PRO-1562 remains in early-stage clinical development, and its safety and effectiveness in patients with multiple sclerosis have not yet been established.
The initiation of human testing represents a step in determining whether the regenerative effects observed in laboratory studies can be replicated in people living with the disease.
Progentos Therapeutics focuses on developing regenerative medicines designed to restore damaged myelin and potentially improve neurological function in patients with multiple sclerosis and other demyelinating conditions.
