While expressing disappointment, William Blair analysts were unsurprised by the Phase II failure, having assigned the VISTA study a high level of risk given the “mixed” performance of a similar drug in a prior multiple sclerosis study.
Johnson & Johnson and Contineum Therapeutics’ PIPE-307, an investigational M1 muscarinic receptor agonist, failed to significantly improve vision outcomes in patients with a specific type of multiple sclerosis.
Without providing specific data, the partners revealed in a statement on Thursday that at two tested doses, the drug candidate “did not meet its prespecified primary or secondary efficacy endpoints.”
In particular, PIPE-307 was unable to significantly improve binocular 2.5% low contrast letter acuity, a test that measures visual acuity at low contrast, in patients with relapsing-remitting multiple sclerosis (RRMS).
J&J and Contineum continue “to interrogate the trial data” and the study’s exploratory endpoints and intend to present the full analysis at a future scientific congress.
Contineum stock dropped about 20% in after-hours trading, hitting a low of $9.78 per share versus its previous $12.22 closing price on Thursday.
Reacting to the announcement, William Blair told investors that while its analysts are “disappointed” by the readout, “we had always viewed the study as risky, given clemastine’s mixed data in prior studies on the low contrast letter acuity metric.”
Like PIPE-307, clemastine is an M1 muscarinic receptor agonist. In March last year, a study arm of the Phase I/II TRAP-MS study, led by the National Institute of Allergy and Infectious Diseases, was halted after investigators detected signals of accelerating accumulation of disability in treated patients.
As for safety, J&J and Contineum on Thursday said that PIPE-307 had an “acceptable” profile at both tested doses.
J&J and Contineum partnered in April 2023 (when Contineum was still called Pipeline Therapeutics), with the pharma fronting $50 million to collaboratively develop PIPE-307 in nervous system disorders. If all milestones are met, Contineum could receive around an additional $1 billion, plus royalties if the drug reaches the market.
The partnership is ongoing despite Thursday’s failure. J&J continues to study PIPE-307, also dubbed JNJ-89495120, for major depressive disorder (MDD). Through its Janssen subsidiary, the pharma is running the Phase II Moonlight-1 study in this indication, which is currently enrolling patients and has a primary completion date in June 2026.
“We see little read-through from the RRMS indication to MDD, but we acknowledge that study also remains risky,” William Blair wrote on Thursday, noting that “placebo responses [in MDD studies] have eroded effect sizes.” Patient heterogeneity, the analysts added, “can lead to volatility in patient response rates.” Topline data from Moonlight 1 are expected mid-2026.
