MS patient enrollment complete in trial testing anti-inflammatory drug
Phase 1b study to use PET imaging to assess impact of PIPE-791 therapy
Two of three groups — one composed of people with progressive forms of multiple sclerosis (MS), and the other comprising healthy volunteers — have been fully enrolled in a Phase 1b clinical trial that’s testing a new anti-inflammatory drug being developed by Contineum Therapeutics.
The Phase 1b study (NCT06683612), launched late last year, is evaluating a therapy called PIPE-791, which is designed to reduce inflammation and tissue damage contributing to disease progression in both MS and a lung condition called idiopathic pulmonary fibrosis (IPF). IPF is marked by lung scarring from unknown causes that leads to breathing difficulties among patients.
While the same trial is studying the experimental treatment in people with both diseases, enrollment among IPF patients is still ongoing, according to a Contineum press release.
The trial’s goal is to assess, using positron emission tomography (PET) imaging, how much of the anti-inflammatory drug gets into the brain and lungs of participants. Researchers also will monitor both the MS and IPF patients for side effects, and intend to study the pharmacological properties of PIPE-791 once inside the body.
A total of 12 healthy adults and four people with progressive MS have now been enrolled, with the last two MS participants added in early June, according to Contineum. The inclusion of these two patients has led to a slight delay in the study timeline, per the company, with Contineum now expecting to report topline data from these two groups in the third quarter of 2025, or by the end of September.
“In early June, consistent with our protocol, we enrolled two additional [progressive MS] patients to assess PET imaging of the brain,” said Timothy Watkins, MD, chief medical officer and head of development at Contineum. The single-center study is being conducted in the U.K.
“While adding these additional patients to the trial has led to a modest delay in reporting our topline data, we believe the enhanced clinical understanding of PIPE-791 in patients is important to the program,” Watkins said. “We now anticipate reporting topline data from these fully enrolled [groups] in the third quarter of 2025.”
Trial results will guide dose selection for further drug development
In MS, the immune system mistakenly attacks the myelin sheath, a protective fatty layer around nerve fibers that helps them efficiently send electrical signals. Although the body can normally repair myelin through a process called remyelination, this process is often impaired in people with MS, resulting in permanent damage.
PIPE-791 is a small molecule designed to enter the brain and selectively block the lysophosphatidic acid 1 (LPA1) receptor, which is thought to contribute to faulty myelin repair.
This receptor is found at the surface of oligodendrocytes, the cells responsible for producing myelin in the brain and spinal cord, and microglia, the brain’s resident immune cells. It binds to LPA, a proinflammatory molecule that’s found at high levels in people with MS, and which can trigger inflammation and reduce oligodendrocyte survival and maturation.
Preclinical studies have shown that PIPE-791 eases inflammation and promotes remyelination as intended. Further, a Phase 1 study (NCT05983939) completed in 2024 also showed that the drug was generally safe and well tolerated in healthy volunteers, when given at single and multiple ascending doses.
The ongoing PET study is now testing how much of LPA1 receptors in the brain and lungs are occupied by PIPE-791 when given as a single oral dose. The findings will help determine the optimal dose for future development in Phase 2a trials.
This trial builds on our compelling preclinical data and improves our understanding of receptor occupancy in healthy volunteers and patients, helping guide our dose selection in the next stages of clinical development [of PIPE-791].
Participants will receive a radiolabeled version of the drug called 18F-PIPE-497, which can be seen in PET scans. Those with progressive MS — either secondary progressive or primary progressive multiple sclerosis — and a group of healthy volunteers will only receive brain scans, while the rest will have lung scans.
The researchers also will assess the treatment’s safety and whether there’s a relationship between LPA1 occupancy and the levels of the drug in the blood.
“This trial builds on our compelling preclinical data and improves our understanding of receptor occupancy in healthy volunteers and patients, helping guide our dose selection in the next stages of clinical development,” Watkins said.
Contineum is also developing another candidate for relapsing-remitting MS in collaboration with Janssen, a Johnson & Johnson company. Called PIPE-307, the therapy is designed to help restore myelin by blocking M1 muscarinic receptors. It’s being tested as an add-on to a standard MS disease-modifying therapy in a Phase 2 trial (NCT06083753).
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