New MS experimental therapy misses main goals in clinical trial
Phase 2 study finds no efficacy signals for PIPE-307 despite good safety profile
- PIPE-307 failed to meet primary or secondary goals in a Phase 2 MS trial.
- The therapy was being tested for improvements in vision and other MS outcomes.
- Despite the lack of efficacy, PIPE-307 was generally safe and well tolerated.
A Phase 2 clinical trial testing PIPE-307, an experimental therapy designed to support myelin repair in people with multiple sclerosis (MS), has failed to meet its primary or secondary efficacy goals.
The U.S.-based VISTA trial (NCT06083753) enrolled more than 180 adults with relapsing-remitting MS (RRMS). Participants received one of two PIPE-307 doses or a placebo for about six months.
The trial’s main goals were to assess PIPE-307’s safety and to determine whether the oral therapy improved vision, as assessed with 2.5% low contrast letter acuity (LCLA). Secondary measures included walking ability, dexterity, cognitive function, and disease biomarkers.
PIPE-307 showed an “acceptable safety and tolerability at both doses,” but the trial “did not meet its prespecified primary or secondary efficacy [goals],” Contineum Therapeutics announced in a company press release.
How PIPE-307 is designed to support myelin repair
The company, which is developing PIPE-307 in partnership with Johnson & Johnson Innovative Medicine, didn’t share additional details, noting that analyses are ongoing and that full results will be presented at a future medical meeting and published in a journal.
“We’re disappointed by these results, but are grateful to the VISTA trial investigators, and especially to the patients and their families,” said Timothy Watkins, MD, chief medical officer and head of development at Contineum. “We intend to learn from these data and remain committed to pursuing novel therapies for patients with inflammatory and fibrotic diseases.”
MS is marked by inflammation that damages myelin in the brain and spinal cord. Myelin is a fatty, whitish substance that wraps around nerve fibers and helps them send electrical signals — similar to insulation wrapped around a wire.
In MS, damage to myelin disrupts nerve signaling, leading to disease symptoms. RRMS — the most common MS type — involves flares, where symptoms suddenly worsen, and remissions, where symptoms ease.
There are more than a dozen approved RRMS treatments, but they all work by reducing inflammation to slow myelin damage. To date, no MS therapy has been proven to promote myelin repair (a process called remyelination).
Finding a medication that can promote remyelination, potentially helping patients regain lost functionality, is often described as a “holy grail” in MS research.
PIPE-307 is designed to promote the maturation of oligodendrocytes, the brain cells responsible for making myelin. It works by blocking M1 muscarinic receptors, cell-surface proteins that have been shown to prompt immature oligodendrocytes to mature into full-grown myelin-making cells.
A previous Phase 1 trial (NCT04725175) found that PIPE-307 was well tolerated in healthy volunteers.
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