September 12, 2016
By Mark Terry, BioSpace.com Breaking News Staff
At least seven biotech companies are facing big decisions this month, including Achillion Pharmaceuticals , Amarin Corporation and Array BioPharma . But as we enter into the last quarter of the year, TheStreet takes a look at 10 biotech companies that are also having big events before the end of the year.
Eli Lilly , based in Indianapolis, has a lot riding on solanezumab to treat Alzheimer’s disease. The drug, which was designed to eliminate beta amyloid plaques in the brain, has already disappointed in two Phase III trials. Its latest study, Expedition-3, expects topline results in December. The original study design had co-primary endpoints of cognition and function. The change made it a single primary endpoint of cognition using the Alzheimer’s Disease Assessment Scale-Cognitive subscale (ADAS-Cog14). Functional outcomes will be viewed as key secondary endpoints.
Adam Feuerstein, writing for TheStreet, says, “Whether or not the drug will actually benefit Alzheimer’s patients in a meaningful way is going to be a hotly debated topic.”
Cambridge, Mass.-based Biogen is also looking for a readout on its aducanumab for Alzheimer’s disease. It also is focused on clearing beta amyloid plaques. It’s shown promise in cutting amyloid-beta plaques in previous trials, but it’s phase III where most Alzheimer’s drugs go to die, and there’s always that possibility here as well. On the other hand, even if it’s a modest success and is approved, it’ll be worth a bundle.
Kite Pharma is headquartered in Santa Monica, California, and is a leader in developing CAR-T immune-oncology therapies. It has four pipeline products in Phase II trials, four in Phase I and 10 in pre-clinical studies. It’s expected to announce interim results for its Zuma-1 trial of KTE-C19 in patients with advanced diffuse large B-cell lymphoma (DLBCL) before the end of this month. Feuerstein writes, “Kite aims to be the first company to secure approval of a CAR-T cancer therapy, beating competitors Novartis and Juno Therapeutics to the market. But only if the KTE-C19 data are positive.”
Bluebird bio is something of a moonshot company focused on gene therapy. Although it has a number of products in its pipeline, only two are in Phase II/III trials. They are Lenti-D for cerebral ALD and LentiGlobin for transfusion-dependent beta-thalassemia.
In December, the company plans to provide clinical updates on these studies at the American Society of Hematology (ASH) annual meeting.
There should be some sort of award for Most Dramatic Clinical Trials given to Sarepta Therapeutics . The company’s drug for Duchene Muscular Dystrophy (DMD), eteplirsen, has had weather-related delays, congressional letters, lengthy public meetings, experts (and non-experts) weighing in, and the U.S. Food and Drug Administration (FDA) delaying its decision on the drug, although in early August there was some hint that the FDA and Sarepta were recruiting more patients for its Phase III ESSENCE trial.
At the moment, who knows? Feuerstein writes: “Dear FDA—What Is taking you so long to render an approval decision on the Duchenne muscular dystrophy drug eteplirsen? Any day now? Heads will explode if we don’t hear news by the end of the year.”
Ophthotech , based in New York and Princeton, New Jersey, focuses on therapies to treat diseases on the back of the idea, specifically age-related macular degeneration (AMD). Its lead product, Fovista, is in a Phase III trial in combination with anti-VEGF monotherapy to treat newly diagnosed wet AMD patients. Results are expected in the fourth quarter.
Feuerstein writes, “The looming Fovista study results are a big-time binary event for Ophthotech and its stock price.”
Bristol-Myers Squibb ’s Opdivo is an amazing drug, even though in August the company had to admit that the drug failed to delay tumor progression or death in newly diagnosed lung cancer patients. One of the leaders in the PD1/PDL-1 class of immune-oncology checkpoint inhibitors, the setback made the market a bit more of a bouncing ball with other companies standing a chance of getting in on the competition. The company will give more data in October on Opdivo as a first-line lung cancer treatment at the European Society of Medical Oncology (ESMO) meeting.
Acadia Pharmaceuticals plans to report results of a Phase II study of Nuplazid for Alzheimer’s disease psychosis by the end of the year. Feuerstein writes, “Acadia is one of those biotech companies often mentioned as takeout bait because approved products to treat neuro-psychiatric illnesses are a relatively scarce commodity. The company has a chance to tart itself up even more for a possible sale when it reports results from a Phase II study of Nuplazid.”
Celgene is expected to release data of GED-0301 for the treatment of Crohn’s disease by the end of this month, with a formal presentation later in the fall. GED-0301 is one of three drugs that make up most of the company’s immunology and inflammation franchise.
And Feuerstein brings up Gilead Sciences as number 10, not because of a clinical trial announcement, but that most investors and analysts think some sort of merger or acquisition deal is going to go down. Feuerstein writes, “Gilead believes its internal research pipeline holds potentially valuable products and is under-appreciated by investors, but its executives also acknowledge the need to buy some stuff. The Gilead checkbook is out of the drawer and on the desk.”
