Investors have found a flaw in Morphic’s glittering EMERALD-1 trial. Months after raising $240 million on the back of top-line results that sent its stock soaring, the biotech has shared a fuller look at the data on its oral ulcerative colitis candidate and wiped out its earlier gains.
The results come from an open-label, single-arm phase 2a trial that gave MORF-057, an oral inhibitor of α4β7, to 35 adults with moderately to severely active ulcerative colitis. In April, Morphic revealed that 25.7% of participants were in clinical remission after 12 weeks of twice-daily dosing, suggesting that the candidate may be similarly effective as Takeda’s injectable anti-α4β7 monoclonal antibody Entyvio.
Now, Morphic has shared more results that have raised doubts about how MORF-057 compares to Entyvio and other ulcerative colitis treatments. The expanded data set, which Morphic shared at United European Gastroenterology Week Copenhagen, includes the rate of endoscopic improvement.
At Week 12, endoscopic improvement was achieved in 25.7% of patients. The rate is lower than expected based on other ulcerative colitis trials, which typically find the rate of endoscopic improvement is higher than the rate of clinical remission.
The rates of clinical remission and endoscopic improvement in Takeda’s phase 3 were 31.3% and 39.7%, respectively, at Week 52. That trial linked AbbVie’s Humira to a higher rate of endoscopic improvement than clinical remission, too. Similarly, a study of Protagonist Therapeutics' oral ɑ4β7 antagonist, which had its own issues, linked (PDF) one dose to 27.5% clinical remission and 43.1% endoscopic improvement at Week 12.
Comparing data across the clinical trials could give a misleading impression of efficacy, particularly when viewing results from Morphic’s small, single-arm study and Takeda’s large, controlled trial. But the track record of earlier programs suggested Morphic would see a higher rate of endoscopic improvement than 25.7%.
In the hours leading up to the publication of the data on Friday afternoon, investors sent the biotech’s share price down 29% to $36.68. The stock drop effectively erased the gains Morphic had made on the back of the top-line data release in April. Having bounced between $34 and $38 in the run-up to the data drop, the stock had climbed above $60 in the weeks after the April statement.
A randomized, double-blind, placebo-controlled phase 2b trial of MORF-057 is already underway. That trial, which is scheduled to complete its primary endpoint analysis in the first half of 2025, could give a clearer picture of whether MORF-057 is a genuine challenger for the ulcerative colitis market.