Pipeline Raises $80M to Pursue Potential Myelin Restoring Therapy

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by Forest Ray PhD |

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funding for remyelination therapy

Pipeline Therapeutics announced that it has raised $80 million in investor financing to develop neuroregenerative therapies for neurological disorders, including those like multiple sclerosis (MS) that are marked by the loss of myelin.

The money will support the research and development of several candidate therapies, with three aimed at promoting and restoring neuronal health.

“We are proud to have this significant support for our mission of developing first- and best-in-class neuroregenerative product candidates for unserved and underserved neurological indications,” Carmine Stengone, president and CEO of Pipeline, said in a press release.

The lead treatment candidate for MS is called PIPE-307, and is designed to help regrow myelin (remyelination), the protective sheath that insulates nerve cell axons against signal loss, much like the coating found around electrical wires.

Pipeline Therapeutics recently launched a Phase 1 clinical trial (NCT04725175) to assess the safety and tolerability of PIPE-307 in up to 72 healthy volunteers, ages 18 to 55, in Australia. Contact information for this trial, which is not yet recruiting, can be found here.

The study is separated into three parts. Part 1 consists of a single ascending dose (SAD) study, involving about 48 volunteers over six weeks. Part 2 consists of a multiple ascending dose (MAD) study in approximately 24 people over seven weeks.

In part 3, roughly eight individuals from the SAD study will take PIPE-307 with a meal to evaluate how food might affect the medication’s bioavailability. Part 3 will run for six weeks.

Although remyelination occurs spontaneously in MS lesions, disease progression increasingly wears on this process to the point that it eventually fails. As myelin is lost, nerve signals traveling from the brain to distant parts of the body are disrupted, and nerve damage and chronic disability occur.

No available therapy can restore myelin, making this one of the greatest unmet needs for people with MS.

PIPE-307 is a muscarinic receptor type 1 (M1) antagonist, meaning that it inactivates the M1 receptor. Blocking muscarinic receptors has the effect of stimulating oligodendrocyte progenitor cells to grow into mature, myelin-producing oligodendrocyte cells.

Other muscarinic receptors have been evaluated as potential therapeutic targets for MS. To date, a lack of knowledge concerning exactly which receptor subtypes are most involved in MS has slowed their path to clinical use.

Other candidate therapies for neurological disorders in Pipeline’s portfolio include compounds targeting remyelination and neuroinflammation, axonal repair, and sensorineural hearing loss.

Pipeline’s Series C financing round was “oversubscribed,” the company reported in its release.

“We are delighted to welcome the new investors in our Series C round, and are grateful to our existing shareholders for their continued support,” Stengone said. “We are proud to have this significant support for our mission of developing first- and best-in-class neuroregenerative product candidates for unserved and underserved neurological indications.”

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