June 29, 2017
By Mark Terry, BioSpace.com Breaking News Staff
A decision by the U.S. Food and Drug Administration (FDA) can be a big boost—or a big negative—for biotech stocks. Keith Speights, writing for The Motley Fool, analyzes five biotech companies facing FDA decisions in July.
Headquartered in Redwood City, Calif., AcelRx Pharmaceuticals focuses on developing and commercializing sublingual therapies for moderate-to-severe acute pain in medically supervised settings. The FDA is expected to make a decision on its drug/device combo to deliver sufentanil for acute pain by the end of July. It has a bit of a history. In 2014, the FDA rejected it. AcelRx expected to address the agency’s concerns and resubmit by the end of 2014, but instead it had to perform another clinical trial. It started in September 2016 and will finish at the end of July. If positive, the company will probably resubmit Zalviso later this year.
In addition, in March, AcelRx announced that the European Medicines Agency (EMA)’s Marketing Authorization Application (MAA) for ARX-04 (sufentanil sublingual tablet, 30 mcg) had passed validation and the scientific review was underway. Called DSUVIA in the U.S., the company expects an opinion on it in the first half of 2018.
AcelRx is currently trading for $2.10.
Based in Cambridge, Mass., Alnylam Pharmaceuticals focuses on RNA interference (RNAi) to develop therapeutics. In late April, the company announced final 24-month results from a Phase II open-label extension trial of patisiran, an RNAi therapeutic that targets transthyretin (TTR) to treat hATTR amyloidosis. The study showed a decrease in the modified neuropathy impairment score and a mean improvement in mNIS+7 across the full range of neuropathy severity.
With its partner Sanofi , Alnylam expects to complete final data collection of this trial in July. Speights writes, “Results from this study should be enormously important for Alnylam, with analysts estimating the drug could generate peak annual sales of over $750 million if approved.”
Alnylam is currently trading for $79.97.
Headquartered in Berkeley, Calif., Dynavax Technologies uses Toll-like receptor (TLR) biology to develop immuno-oncology therapies and vaccines. The company has failed to gain FDA approval for Heplisav-B, a vaccine for hepatitis B, twice so far. Speights writes, “Whether that optimism pays off or not might be known soon after July 28. That’s when the Vaccines and Related Biological Products Advisory Committee (VRBPAC) will review Heplisav-B. If this committee gives a thumbs-up, it will be a very good sign for Dynavax’s chances for the third time being the charm when the FDA announces its approval decision on August 10.”
Dynavax is currently trading for $10.15.
UK-based GlaxoSmithKline received approval by the FDA in 2011 for intravenous Benlysta to treat lupus. In 2016, it brought in $300 million, an increase of 33 percent from the previous year. GSK has created a subcutaneous formulation and is expecting an FDA decision by July 23.
The regulatory submission was announced on September 23, 2016 and included submissions to the European Medicines Agency. Systemic lupus erythematosis (SLE) is the most common type of lupus and affects about 70 percent of the 5 million people with lupus worldwide.
GSK is currently trading for $43.70.
Located in Los Angeles, Puma Biotechnology received a positive vote of 12 to 4 from the FDA’s Oncologic Drugs Advisory Committee (ODAC) on May 24 for neratinib as adjuvant treatment of HER2-positive early stage breast cancer. The FDA it scheduled to decide on the drug by July 21.
In addition, on June 3, Puma presented positive data from an ongoing Phase II trial of neratinib to treat HER2-positive metastatic breast cancer that has metastasized to the brain.
Puma is currently trading for $88.85.
