Janssen acquires rights to PIPE-307, potential myelin restoring therapy

Pipeline Therapeutics is collaborating with a branch of Janssen Pharmaceuticals to advance PIPE-307, a potential oral treatment of nervous system conditions that is expected to enter a clinical trial in people with relapsing-remitting multiple sclerosis (RRMS).

The compound previously was found to be safe and tolerable in healthy volunteers, and the U.S. Food and Drug Administration has cleared PIPE-307, aiming to restore myelin, for testing in MS patients.

Under the terms of their agreement, Janssen Pharmaceutica NV — the company is part of Johnson & Johnson — gains exclusive global rights to develop and commercialize PIPE-307 for all indications, including multiple sclerosis and mood disorders. Pipeline will continue to advance the compound for RRMS patients though a Phase 2 clinical trial.

“We are excited to announce this agreement, which is aligned with our vision to maximize the reach of PIPE-307 through clinical evaluation in RRMS and other neurological disorders,” Carmine Stengone, president and CEO at Pipeline, said in a company press release.

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PIPE-307 aims to restore myelin sheath by promoting myelin-generating cells

MS is marked by an exacerbated inflammatory response that damages the myelin sheath, a fatty substance surrounding nerve fibers that helps them send electrical signals.

Damaged myelin usually can be repaired by oligodendrocytes, myelin-producing cells of the brain and spinal cord.

However, this process tends to be faulty in MS patients. Ongoing inflammation impairs the generation of mature oligodendrocytes from their precursor cells, affecting the production of myelin by these specialized cells.

PIPE-307 aims to boost remyelination by promoting the transition of oligodendrocyte precursor cells into mature oligodendrocytes. It works by selectively inhibiting the action of M1 muscarinic receptors, which regulate a number of brain functions.

Pipeline has completed various Phase 1 trials of PIPE-307 in healthy volunteers, which generally show the medication to be safe and well tolerated at multiple doses. It also has shown an ability to reach the brain at doses deemed effective in animal models.

Notably, no cognitive effects related to the therapy were evident in Phase 1 trials, as assessed by tests of psychomotor and executive functions, learning, and memory, Pipeline reported.

The potential Phase 2 trial is expected to enroll about 45 RRMS patients at various U.S. sites, who will receive one of two PIPE-307 doses or a placebo, the company reported last year. Pipeline did not disclose information regarding the trial in its recent release, and it’s not clear if modifications were made to its protocol.

Upon the collaboration agreement being finalized, Pipeline will receive a $50 million upfront payment from Janssen, and up to $25 million in equity investment from Johnson & Johnson Innovation, plus up to another $25 million in equity investments from existing investors.

Pipeline also will be eligible for about $1 billion if certain clinical, regulatory, and commercial milestones are met, as well as potential royalty payments.

“We will strengthen our financial position with the upfront payment from this agreement … to continue advancing our portfolio in small molecule neuroregeneration,” Stengone said.