Win Some, Lose Some: Clinical Trial Updates

After last week’s avalanche of clinical trial news associated with the American Society of Hematology (ASH) Annual Meeting, there are far fewer announcements, but some companies are getting their news out ahead of the holidays. Here’s a look at some of the wins and losses today.

Lilly’s Lebirkizumab Hits Mark in Atopic Dermatitis

Eli Lilly announced that its Phase III trial of lebrikizumab hit the primary and key secondary endpoints in moderate-to-severe atopic dermatitis (AD). The drug is an IL-13 inhibitor. In patients with atopic dermatitis, the IL-13 protein is overexpressed. The Adhere Phase III trial lebrikizumab combined with topical corticosteroids (TCS) hit the Investigator Global Assessment (GA) score of clear (0) or almost clear (1) skin with a decrease of at least two points from baseline and at least a 75% change from baseline in the Eczema Area and Severity Index (EASI) score, both at Week 16.

Dr. Eric Simpson, Professor of Dermatology and Director of Clinical Research at Oregon Health & Science University in Portland and principal investigator of the study, said, “I’m encouraged by the aggregate efficacy and safety data which have demonstrated the potential for lebrikizumab as both monotherapy and combination therapy to address unmet needs and improve care for people living with persistent itch and inflamed skin caused by AD.”

Pfizer Publishes Successful 5-Year Data on ATTR-CM

Pfizer published results from a post-hoc, interim analysis of five-year follow-up data of Vyndaqel (tafamidis meglumine)/Vyndamax (tafamidis), showing the drugs provided a clinically significant survival benefit at five years for patients with transthyretin amyloid cardiomyopathy (ATTR-CM). They published the data from the Phase III Transthyretin Amyloid Cardiomyopathy Clinical Trial (ATTR-ACT) as well as the long-term extension study in Circulation: Heart Failure.

“The results from this analysis build upon the positive primary results from the pivotal trial, ATTR-ACT, and reinforce that Vyndaqel and Vyndamax have the potential to significantly extend survival for patients with AATR-CM,” said Dr. Brenda Cooperstone, Chief Development Officer, Rare Disease, Pfizer Global Product Development.

Aldeyra’s Dry Eye Disease Drug Has Mixed Results in Phase III

Aldeyra Therapeutics announced top-line data from the Phase III TRANQUILITY trial of reploxalap for dry eye disease. The drug is a 0.25% reproxalap ophthalmic solution. The study missed the primary endpoint of ocular redness but did hit statistical significance for dry eye disease sign of Schirmer test, a secondary endpoint.

“Following the achievement of statistical significance in ocular redness in our recent Phase II clinical trial, the achievement of statistical significance of Schirmer test in TRANQUILITY may provide an additional option to satisfy the remaining objective sign requirement for dry eye disease NDA submission,” said Dr. Todd C. Brady, President and Chief Executive Officer of Aldeyra. “Subject to agreement with the FDA, we believe that the TRANQUILITY results allow for the possibility that, pending the outcome of TRANQUILITY-2, the NDA submission for reproxalap could represent the first time a dry eye disease drug will have qualified for the demonstration of activity for two objective signs.”

Summit’s C. Diff Drug Missed Superiority in Phase III Trial

Summit Therapeutics announced that its Phase III Ri-CoDIFy trial of ridinilazole for C. difficile infection failed to hit the primary endpoint of superiority over vancomycin. Ridinilazols is a precision antibiotic. The drug resulted in a higher observed Sustained Clinical Response (SCR) for patients with C. diff infection but wasn’t superior to vancomycin.

“We believe this study was indicative of worthwhile work, as the knowledge that we have acquired as a team over the past 18 years is priceless,” said Robert W. Duggan, Chairman and Chief Executive Officer of Summit. “The differences between episodes of recurrence experienced by patients in the two arms of this study may be indicative as to the significance of precision medicinal therapies that spare the microbiome from damage.”