Enrollment complete in Phase 2 trial of remyelination therapy for RRMS
Recruiting finished early for ongoing VISTA study of Contineum's PIPE-307
Patient enrollment has been completed ahead of schedule in a Phase 2 clinical trial evaluating Contineum Therapeutics‘ PIPE-307, an oral therapy aiming to restore myelin — a process known as remyelination — in people with relapsing-remitting multiple sclerosis (RRMS), the company announced.
The VISTA trial (NCT06083753), launched in late 2023, has now recruited its target of 168 participants across 21 sites in the U.S., according to a Contineum press release. The study is investigating two daily doses of PIPE-307 against a placebo in people who are already receiving a standard MS disease-modifying therapy.
Treatment will be given for 26 weeks, or about 6.5 months. The last patient is expected to complete the trial in the third quarter of this year, or by the end of September, per the release.
“We are pleased to reach this significant milestone for the PIPE-307 VISTA trial ahead of schedule,” said Stephen Huhn, MD, Contineum’s chief medical officer. “We believe that PIPE-307 could represent the next evolution in the treatment paradigm for RRMS.”
Main goals of Phase 2 trial are safety, changes in visual acuity
Multiple sclerosis (MS) is caused by a misguided inflammatory response within the body that causes damage to the myelin sheath, a protective coating around nerve fibers that helps with nerve signal transmission.
In healthy people, this damage can be repaired by oligodendrocytes, which are specialized cells in the brain and spinal cord responsible for making new myelin. However, due to the ongoing inflammation and the abnormal maturation of oligodendrocytes in people with MS, this process is often faulty.
Finding therapies that can improve myelin repair has long been an important field of research in MS. While many treatments are available to ease inflammation and prevent or slow myelin damage, current therapies cannot reverse damage that has already taken place.
PIPE-307 is designed to boost remyelination by promoting the transition of oligodendrocyte precursor cells, or OPCs, into mature oligodendrocytes that can produce new myelin. It works by blocking M1 muscarinic receptors, which are found particularly in OPCs close to MS lesions with ongoing inflammation.
The trial is designed to show evidence of remyelination as a potentially first-in-class and differentiated therapy for RRMS patients.
In a mouse model of MS, the therapy increased myelin repair as designed and reduced physical disability relative to untreated animals. Moreover, it was found to be generally safe and well tolerated at multiple doses in Phase 1 trials involving healthy volunteers.
The VISTA study is now testing two doses of PIPE-307 in adults, ages 18 to 50, with RRMS. This is the most common form of MS and is characterized by relapses, or periods when symptoms suddenly worsen, followed by periods of remission where symptoms ease or disappear entirely.
The main goals are to test PIPE-307’s safety and changes in visual acuity, indicative of better myelination in the nerve that sends messages from the eyes to the brain, in a subgroup of patients. Other vision measures, as well as changes in walking function, dexterity, cognition, and disease biomarkers, will also be assessed.
“The trial is designed to show evidence of remyelination as a potentially first-in-class and differentiated therapy for RRMS patients,“ Huhn said. “We are grateful for the VISTA trial investigators, patients and their families, and we look forward to sharing topline data from this trial.”
PIPE-307 is being developed under the terms of an agreement between Contineum and Janssen Pharmaceutica, a Johnson & Johnson company.
About the Author
