Pfizer Fails in C.diff, BMS Announces Priority Review for Opdivo in NSCLC
Phase III clinical trials are significant milestones for any biopharmaceutical company. This week has already brought three major announcements regarding separate Phase III clinical trials being run by Pfizer, Bristol Myers Squibb and Saol Therapeutics.
Pharma giant Pfizer suffered stock losses after announcing the results of its Phase III study for its Clostridioides difficile infection vaccine (sometimes abbreviated C. diff. or CDI). CDI, a harmful bacteria that causes severe colon inflammation and diarrhea, can be fatal. About half a million patients in the United States are infected with the bacteria each year, reported the U.S. Centers for Disease Control and Prevention (CDC).
The Phase III trial failed to meet its primary endpoint. However, the vaccine did show promise in several of the secondary endpoints. The vaccine was tested in approximately 17,500 adults aged 50 and older, where it achieved 100% efficacy when compared to a placebo. Additionally, the vaccine protected patients with a 75-80% reduction in disease episodes, so infected patients experienced far milder symptoms.
However, because the vaccine failed to meet its primary endpoint, Pfizer said it is currently re-evaluating its vaccine program for CDI.
This news from Pfizer comes after several other competitors entered the C. diff. treatment space. Summit Therapeutics also ran a Phase III clinical trial in December 2021 for an experimental CDI treatment that failed to meet its primary endpoint. However, in January 2022, French biotech company DEINOVE got a favorable opinion from the Data Safety Monitoring Board following a Phase II clinical trial for its CDI Drug.
In further significant news, Bristol Myers Squibb announced that the FDA has accepted Opdivo for priority status review as a neoadjuvant treatment in resectable NSCLC. The company has a Prescription Drug User Fee Act goal date of July 13, 2022.
Opdivo's success in the CheckMate-816 study was described by BMS as "demonstrat[ing] a statistically significant and clinically meaningful improvement in pathologic complete response (pCR) and event-free survival (EFS) versus chemotherapy alone when given before surgery."
BMS submitted the supplemental biologics license application based on the success of this study. Opdivo is currently approved for the treatment of several types of cancer, including metastatic NSCLC.
Saol Therapeutics also revealed clinical trial news. The privately-owned Roswell, Georgia-based company is researching treatments for rare diseases. Saol announced that it is partnering with GeneDx, Inc., a pioneer in genomic analysis, to recruit patients for a Phase III trial on Saol's investigational drug, dichloroacetate (DCA).
DCA is intended for young children who have pyruvate dehydrogenase complex deficiency (PDCD), a usually fatal metabolic disease. The condition affects less than 300 children in the U.S. each year, which helped the FDA grant Orphan Product designation to Saol's DCA therapy.
GeneDx has a screening technology to help identify the genetic code that causes PDCD. Saol Therapeutics hopes that GeneDx can help find patients for the Phase III clinical trial and help develop an understanding of how DCA works through this partnership.
"There are currently no FDA-approved treatments for patients with PDCD. Despite this, finding and recruiting children appropriate for participation in clinical trials is not easy. With the help of GeneDx, we hope to complete trial recruitment this year," said Dr. Peter Stacpoole, principal investigator of the DCA trial and professor of medicine at the University of Florida.