Contineum Therapeutics’ M1 receptor antagonist has failed to move the needle in a phase 2 vision test for patients with a common form of multiple sclerosis.
The Johnson & Johnson-partnered asset, coded PIPE-307, failed to trigger a significant change in binocular 2.5% low contrast letter acuity across treatment arms, the San Diego biotech reported after market close Nov. 20. The lack of significant change caused the trial to miss its primary efficacy goal, and the study failed to hit secondary efficacy endpoints as well.
The midstage trial enrolled 182 patients with relapsing-remitting multiple sclerosis, a condition that is commonly associated with vision problems. The double-blind, placebo-controlled study looked at two different doses of PIPE-307.
The trial did demonstrate an “acceptable safety and tolerability at both doses,“ according to Contineum. The biotech said it is continuing to “interrogate the trial data related to its exploratory endpoints” and plans on sharing a full data set at a future medical conference and in a peer-reviewed medical journal.
While William Blair is “disappointed by the result,“ the analyst said it had “always viewed the study as risky,“ citing mixed data from the H1 histamine antagonist clemastine on the same vision acuity metric in MS populations.
“We intend to learn from these data and remain committed to pursuing novel therapies for patients with inflammatory and fibrotic diseases,” Contineum Chief Medical Officer and Head of Development Timothy Watkins, M.D., said in a Thursday press release.
Contineum’s stock has dropped 14% in post-market trading, slipping from $12.22 per share at market close to $10.50 at 5 p.m. ET.
Contineum is developing PIPE-307 in partnership with J&J. In 2023, the pharma’s Janssen unit penned a $50 million upfront licensing deal for the oral MS drug, tacking on the possibility of $1 billion in biobucks for the biotech.
The Big Pharma is currently assessing PIPE-307 in a mid-stage trial for major depressive disorder (MDD).
“We see little read-through from the RRMS indication to MDD, but we acknowledge that study also remains risky, evidenced by our 35% probability of success for PIPE-307 in MDD,” William Blair wrote in a Nov. 20 note. The likelihood considers “the risky nature of placebo-controlled studies in MDD, where placebo responses have eroded effect sizes and patient heterogeneity can lead to volatility in patient response rates on active drug.”
Topline data from the J&J study are expected in mid-2026.
As for Contineum, William Blair said it continues to “view the lion’s share of value for Contineum in the development of the company’s LPAR1 antagonist PIPE-791 in idiopathic pulmonary fibrosis (IPF).”
In August, the biotech pumped the brakes on two projects, halting plans for PIPE-791 in progressive multiple sclerosis and for an LPAR1 antagonist called CTX-343 in peripheral fibrotic disease so it could focus on the IPF indication. Contineum is carrying PIPE-791 into a phase 2 trial for the lung disease, where it hopes to eventually challenge Bristol Myers Squibb.
Editor's note: This article was updated at 9:45 a.m. ET on Nov. 21 to include analyst insight.