The closing weeks of summer could heat up for a few biotechs with experimental new therapies getting close to the finish line. TG Therapeutics, Inc. (TGTX 1.06%) and Sangamo Therapeutics, Inc. (SGMO 2.12%) will reveal some hotly anticipated clinical trial results in the weeks ahead. Akcea Therapeutics, Inc. (AKCA) is a lot closer to commercializing its first product in the U.S., but it's still relying on two approval decisions in its near future.
Here's what investors need to know about the catalysts that could move these biotech stocks in the weeks ahead.
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1. Akcea Therapeutics, Inc.: Homecoming parade?
This Ionis Pharmaceuticals (IONS 1.88%) spinoff earned approval to market a rare disease drug called Tegsedi in the European Union earlier this year, but the U.S. Food and Drug Administration (FDA) won't make a decision about the drug we also know as inotersen until October. In the meantime, the agency is expected to deliver an approval decision on or before August 30 for Waylivra, formerly volanesorsen, and the outcome is far from certain.
Waylivra helped people with a rare lipid disorder lower their triglyceride count dramatically, but it also led to disturbing losses of blood platelets that caused some people to exit the trial. Many patients with FCS suffer from excruciating bouts of pancreatitis, and in midstage studies, it looked like Waylivra effectively lowered the risk of pancreatitis attacks along with triglyceride levels. Akcea hasn't shown a measurable survival benefit yet, which is a reason independent physicians voted 12 to eight in favor of approving Waylivra to treat familial chylomicronemia syndrome (FCS) earlier this year.
Given the advisory panel vote, there's a small chance the FDA will delay Waylivra's launch by asking Akcea and Ionis to generate more data, but a restrictive prescribing label seems more likely. If the agency signals confidence Waylivra's benefits outweigh its risks with a less restrictive approval, though, the stock could jump.
2. TG Therapeutics, Inc.: A big presentation
Imbruvica's a tablet that's becoming popular among new patients with the most commonly diagnosed form of leukemia. It's generating billions in annual sales for AbbVie and Johnson & Johnson, but it doesn't work for everybody. TG Therapeutics is developing umbralisib as a different sort of kinase inhibitor for people who can't use Imbruvica. Umbralisib produced encouraging responses from patients with several forms of cancer during a mid-stage study, and this biotech quickly ushered it into a large pivotal trial called Unity.
The Unity study is expected to produce data by the end of the summer and it needs to impress. The company noticed an encouraging rate of tumor responses in mid-stage studies, but TG Therapeutics needs more time to show the sort of long-term survival benefit that could drive this drug into 10-figure annual sales territory.
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Investigators won't have survival data ready for a while yet, but they do intend to present response rate figures by the end of the summer. If umbralisib can shore up its efficacy profile with strong numbers this stock could get a nice boost.
3. Sangamo Therapeutics, Inc.: Chance to come back
This biotech stock soared earlier this year due to excitement over a gene therapy candidate that allowed patients with a rare bleeding disorder to produce a vital clotting factor on their own after a single dose. Pfizer signed on to the hemophilia candidate and Gilead inked a discovery deal for cancer candidates that sent Sangamo stock to a peak that it has steadily slid from as competition in these spaces heats up.
Sangamo has a gene therapy candidate for people with a rare disease called mucopolysaccharidosis 2 (MPS2), or Hunter syndrome that could help the stock bounce back. If all goes as intended, a single administration of Sangamo's wholly owned candidate, SB-913 will allow people born with Hunter syndrome to produce a lifelong supply of an important digestive enzyme.
Sangamo's going to spill the beans on the first five patients treated with SB-913 at a medical conference in early September. Success with SB-913 would be a great sign for an MPS1 treatment that didn't begin human studies until this July, and send the stock on climbing again.
A good time to be a spectator
Investors are expecting some impressive results from TG Therapeutics and Sangamo, and we still don't know if they'll be able to deliver. With Akcea, investors are anticipating an approval, albeit a restrictive one.
Better-than-predicted outcomes could help these stocks climb a bit in the weeks ahead, but I think there's a much better chance one of them could disappoint in a way that leads to a swift fall. It's probably best to watch this show unfold from the sidelines.
