Two Pharma Companies Begin February with Hits and Misses

Belgium-based UCB hit the mark in its Phase III clinical trial assessing zilucoplan as a potential treatment for adults with generalized myasthenia gravis, a rare autoimmune disease. The study hit its primary and secondary endpoints and set up potential regulatory approval.

UCB‘s zilucoplan is a self-administered, subcutaneous peptide inhibitor of complement component 5, a driver of the pathology of generalized myasthenia gravis (gMG). Data from the Phase III RAISE study showed that zilucoplan demonstrated a clinically significant and statistically substantial improvement from baseline in Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) after 12 weeks compared to placebo. Detailed data from the study will be presented at a future medical conference.

In addition to the primary endpoint, UCB said zilucoplan met all key secondary endpoints, including statistically significant improvements from baseline in Quantitative Myasthenia Gravis (QMG) score, Myasthenia Gravis Composite (MGC) score and MG-QoL15r after 12 weeks.

James F. Howard, chief of Neuromuscular Disorders Section at the University of North Carolina School of Medicine and lead investigator in the RAISE trial, said gMG patients could experience varying and debilitating symptoms. The study shows that zilucoplan can offer a significant step forward in addressing the unmet needs of people living with gMG.

UCB Chief Medical Officer Iris Loew-Friedrich said the data from the Phase III RAISE study builds on previous data from the late-stage MycarinG study in gMG. That study, which assessed rozanolixizumab, an SC-infused monoclonal antibody targeting the neonatal Fc receptor, also met primary and secondary endpoints. UCB is the only company investigating two potential treatments with different mechanisms of action in gMG.

“This is the latest in a series of positive Phase 3 data announcements across UCB’s product pipeline validating our patient value strategy and laying foundations for future sustainable growth. Today’s results represent another significant milestone in UCB’s efforts to bring transformational outcomes to those living with myasthenia gravis,” Loew-Friedrich said in a statement. “Positive results for zilucoplan and rozanolixizumab – each with a different mechanism of action – bring us one step closer to achieving our ambition of delivering choice and flexibility for a broad range of patients and physicians.”

Based on data from the RAISE study, UCB intends to seek regulatory approval in the United States, Europe and Japan. Filings are expected later this year.

While UCB celebrated its Phase III data, Vanda Pharmaceuticals was bemoaning the failure of its late-stage study of tradipitant as a treatment for the symptoms of gastroparesis, a digestive disorder. The Phase III VP-VLY-686-3303 study failed to meet its primary endpoint, which was the change of the severity of nausea compared to placebo at 12 weeks.

While the trial failed to hit the mark, Vanda said both treatment arms saw significant improvements with nausea as well as the other core symptoms of gastroparesis.

Mihael H. Polymeropoulos, president, chief executive officer and chairman of the board of directors at Vanda Pharmaceuticals, expressed disappointment in the trial failure but noted that the company is encouraged by some positive signals the trial data is showing. Some initial exploratory analysis have identified “potential confounders that may have masked the beneficial effect of the drug previously observed in the Phase II study of tradipitant,” the company said.

The confounders include what the company said was a baseline imbalance of rescue medication use between the two treatment arms as well as observed poor compliance with study drug for some patients in the study. When the data is restricted to those patients who did not use rescue medications at baseline and adjusts for poor compliance, the company believes there is strong evidence of tradipitant’s efficacy.

“We are encouraged by the evidence that is emerging from further analysis that confirms observations made in the prior clinical study. We are committed to completing our analysis and working to bring tradipitant to patients with gastroparesis to fill a significant unmet medical need,” Polymeropoulos said in a statement.

Vanda Pharmaceuticals intends to continue the data analysis for review in a journal and submission to regulatory agencies.

Shares of Vanda Pharmaceuticals have plunged more than 26% on the news of the trial failure. The stock is trading at $11.71 in premarket trading, down from Thursday’s close of $14.65 per share.