Companies in the biotech and pharmaceutical industries are subject to a sizable amount of risk and scrutiny with regards to their drug candidates passing clinical trials and gaining regulatory approval. Yet, with the risk of failure, which can spell disaster for a stock, comes great reward as well. Some companies can see massive upside just from winning a single trial or even achieving as much as a Fast Track designation from the U.S. Food and Drug Administration (FDA).
In simpler terms, these updates within the industry have the potential to make or break biopharma companies.
This year was wildly positive for the health care sector. In fact, the iShares Nasdaq Biotechnology ETF (NASDAQ: IBB) hit a multiyear high in this time. Loads of positive trials and approvals fueled these gains, and with any luck, companies within this sector will continue on this course.
Here, 24/7 Wall St. has included a calendar of a few of the biggest clinical trial and FDA updates to watch for going into 2020.
Note that these dates may be subject to change due to various outside and internal factors, or some of these dates may have changed already. Some of these date changes are positive developments, while some can be disasters if a company is deeply financed.
As a side note about the Prescription Drug User Fee Act (PDUFA): a Priority Review designation is granted to medicines that the FDA determines have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease. The FDA grants Priority Review to medicines that may offer significant advances in treatment or may provide a treatment where no adequate therapy exists.
Merck & Co. Inc. (NYSE: MRK) announced on December 17 that the Oncologic Drugs Advisory Committee of the U.S. FDA voted nine to four in favor of recommending Keytruda for the treatment of certain patients with high-risk, non-muscle invasive bladder cancer. The committee provides the FDA with independent advice and recommendations on marketed and investigational medicines for use in the treatment of cancer. Merck has indicated that a PDUFA target action date would be in January 2020, based on priority review. That said, investors at least have to consider that the FDA is not bound by any of its committees’ guidance or recommendations, even if it does take their advice into consideration to approve a drug.
Pfizer Inc. (NYSE: PFE) today announced that the FDA has accepted and granted priority review to the company’s supplemental New Drug Application for Braftovi in combination with Erbitux based on results from the Phase 3 Beacon trial. That trial evaluated the efficacy and safety of Braftovi in combination with Erbitux with or without Mektovi in patients with advanced metastatic colorectal cancer following one or two lines of therapy. The PDUFA goal date for a decision by the FDA is in April 2020.
Intercept Pharmaceuticals Inc. (NASDAQ: ICPT) announced on December 13 that it had submitted its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for obeticholic acid for the treatment of fibrosis due to nonalcoholic steatohepatitis. The company has noted positive interim analysis results from the pivotal Phase 3 Regenerate study in patients with liver fibrosis due to nonalcoholic steatohepatitis. The FDA has tentatively scheduled the Advisory Committee meeting for April 22, 2020, but Intercept also anticipates that the FDA accordingly will extend the recently announced March 26, 2020, PDUFA target action date for its application. Intercept previously announced the FDA’s acceptance of the NDA and granting of priority review.
VBI Vaccines Inc. (NASDAQ: VBIV) completed its pivotal Phase 3 Constant study back in September, but top-line data are expected in early January 2020. Successful completion of the second pivotal Phase 3 Constant study is required for the Biologics License Application (BLA) to the FDA, the MAAs to the EMA, and the New Drug Submission to Health Canada. If approved, this vaccine could play an important role in the prevention of hepatitis B, addressing a significant unmet medical need in the adult population.
Aimmune Therapeutics Inc. (NASDAQ: AIMT) announced earlier this quarter that the Allergenic Products Advisory Committee convened by the FDA voted to support the use of AR101 in children and teens with peanut allergy. The committee voted seven to two that the efficacy data and eight to one that the safety data, in conjunction with additional safeguards, are adequate to support the use of AR101. Aimmune’s BLA seeking approval for AR101 for the treatment of children and adolescents with peanut allergy currently is under review by the FDA, with a review action due date of late January 2020.
Global Blood Therapeutics Inc. (NASDAQ: GBT) received accelerated approval for voxelotor for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older back in November. SCD affects an estimated 100,000 people in the United States and millions of people throughout the world. The FDA’s PDUFA target date for voxelotor in SCD patients is set for February 26, 2020.
Durect Corp. (NASDAQ: DRRX) announced in October that the FDA will be discussing its Class 2 NDA resubmission for Posimir will be discussed at a meeting of the Anesthetic and Analgesic Drug Products Advisory Committee scheduled in January 2020. Posimir is the company’s investigational post-operative pain relief depot product. It has previously demonstrated a significant decrease in pain and opioids consumed over the zero to 72-hour period following surgery. The FDA previously assigned a user fee goal date of December 27, 2019, but a new user fee goal date is expected to come from this meeting.