September 1, 2016
By Mark Terry, BioSpace.com Breaking News Staff
In the biotech industry, positive clinical trial data can send stock soaring, and negative readouts can kill a company. Todd Campbell, writing for The Motley Fool, looks at three biotech companies with key trial readouts coming out soon.
Novavax , based in Rockville, Maryland, uses recombinant nanoparticles and its Matrix-M adjuvant technology to develop vaccines. It has several products in its pipeline for respiratory syncytial virus (RSV), the flu, as well as for Ebola. The study that is furthest ahead is a Phase III trial for RSV. RSV is a common respiratory disease in infants and the elderly, and causes about 180,000 hospital admissions in the U.S. every year.
Campbell writes, “If RSV-F is eventually approved, then Novavax believes its addressable market in the United States could exceed 100 million people in 2020. That has management thinking that peak annual sales for RSV-F could be north of $6 billion.”
Although investors seem optimistic, not all analysts are. Cory Renauer, writing for The Motley Fool on August 29, said, “The big source of disagreement about Novavax’s future stems from the RSV-F Vaccine’s performance in a Phase II trial with about 1,600 patients age 60 and over. During the year following dosing, 23 patients receiving the vaccine showed RSV symptoms compared to 39 injected with a placebo. To the uninitiated, those results might not seem so hot, but they were just good enough to be considered statistically significant.”
Novavax is currently trading for $6.81.
Agile Therapeutics , based in Princeton, New Jersey, is focused on developing new prescription contraception products. The lead product, Twirla (AG200-15) is a once-weekly prescription contraceptive patch. The company also has other transdermal contraceptive products in its pipeline. Data from Twirla’s Phase III trial are expected by the end of this year.
The company argues that the patch would have fewer side effects than oral contraceptives, and that the market is worth about $5 billion annually. A previous new drug application (NDA) was rejected by the U.S. Food and Drug Administration (FDA), apparently over the trial design and concerns about patient tracking and compliance.
However, if the drug was approved, Campbell writes, “Then this company could be significantly undervalued given that this could be a nine-figure drug. The company’s market cap is currently only $211 million.”
Agile Therapeutics is currently trading for $7.43.
Newton, Massachusetts-based Karyopharm Therapeutics focuses on regulating intracellular communication to develop drugs for various cancers. Its lead drug, KPT-330 (selinexor), is an XP01 inhibitor being evaluated in several late-stage trials in patients with relapsed and/or refractory hematological and solid tumor cancers.
Karyopharm is expecting to report data from a Phase IIb trial of selinexor before the end of this month. Campbell writes, “Selinexor may provide newfound hope to these heavily pre-treated patients. In the past, response rates for patients who have already been treated with four different therapies have been in the 20 percent to 25 percent range. If combining selinexor with chemotherapy results in higher response rates than that, the FDA may consider accelerated approval.”
So if approved, the drug has the potential to be a blockbuster—$1 billion or more a year.
The stock jumped recently after the company announced it was doubling the size of its STORM Phase IIb trial of selinexor in multiple myeloma patients. The company was founded by Sharon Sacham and led by her husband, Michael Kauffman. In 2013, the company raised $104 million from its IPO, although it’s been up and down since, including losing about two-thirds of its value since early August until a recent jump.
Karyopharm is currently trading for $9.85.
