Contineum Therapeutics, a San Diego-based biotechnology company, disclosed Thursday that its most advanced experimental drug has failed as a multiple sclerosis treatment in a mid-stage clinical trial.
The study compared two different doses of Contineum’s drug, code-named PIPE-307, to a placebo against the most common, “relapsing-remitting” form of MS. Though both doses showed “acceptable” safety and tolerability, they had no significant impact on the trial’s main measure: a vision test that tasks participants to read faint letters. Blurry or impaired eyesight is often one of the first noticeable symptoms of MS.
PIPE-307 also missed the trial’s so-called secondary endpoints. In a statement, Contineum said it will continue to analyze data from “exploratory” study ojectives, and plans to present the full results at a medical meeting as well as publish them in a peer-reviewed journal. The company remains “committed” to finding new therapies for inflammatory and fibrotic diseases, according to Chief Medical Officer and head of development Timothy Watkins.
“While we are disappointed by the result, we had always viewed the study as risky,” wrote Myles Minter, an analyst at the investment firm William Blair, in a note to clients.
Contineum shares fell more than 12% in after-hours trading Thursday, to hover around $12.20 apiece.
The biotech formed in 2017 under the name Pipeline Therapeutics. It secured $30 million in late 2019 from a Series B fundraising round that saw participation from a handful of backers, including its founding investor, Versant Ventures. Less than two years later, Contineum closed a $80 million Series C round. The company then brought in $110 million by going public in 2024.
Continuem came to the Nasdaq in the midst of a historical downturn in biotech stocks. For the past year, company shares traded below their $16 debut price.
The company has two drugs in human testing. One, PIPE-791, is in early-stage trials for chronic pain, progressive MS and a lung disease known as IPF. To Minter, PIPE-791 is responsible for the “lion’s share” of Contineum’s value, and the way it works — by blocking “LPAR1,” a multifunctional receptor protein — is an “exciting” approach to treating IPF. A few other companies, including Bristol Myers Squibb, are pursuing similar programs.
PIPE-307, meanwhile, is designed to inhibit “M1” — one member of a protein family that regulates the nervous system and, research suggests, might also help control parts of the immune system. In MS, the immune system mistakenly attacks the protective coatings that surround nerve cells, leading to a variety of sensory, mobility and cognitive issues. A study published this year in the journal Molecular Pharmacology describes how M1 may impede the body from repairing these coatings.
Notably, Cobenfy, a Bristol Myers drug that was recently approved to treat schizophrenia, amplifies both M1 and a related protein, “M4.”
Researchers are also investigating whether PIPE-307 can be useful treating major depression. Minter wrote that he sees “little read-through” from the failed MS trial to an ongoing depression study, though the latter is still risky. The William Blair team estimates a 35% probability of success.
PIPE-307 has attracted interest from one of the world’s largest pharmaceutical firms, Johnson & Johnson, which in 2023 paid $50 million for an exclusive, worldwide license to study, develop and commercialize it.
