Biopharma is a complex, rapidly evolving industry that is highly regulated and closely watched — and that means there is constant news. Here's a closer look at the clinical trials, M&A, cool science and regulations that are driving the industry this week.
In case you missed it
- Valeant's problems continue with another warning letter
- Kite Pharma moving full steam ahead with CAR-T plans
- Public and private incubators are bridging the innovation gap
Mergers & analysis
The week was overshadowed by an unexpected turn of events in the U.S. Presidential election as Donald Trump claimed victory over Hillary Clinton. While little is known about Trump's plans for policy, biotech and pharma may be a few of the only industries that consider the outcome a win.
Stocks in both sectors have been decidedly in the green since the market opened on Wednesday and sighs of relief reverberated throughout pharma as concerns about a drug pricing crackdown eased a bit. Meanwhile, the industry hummed with anticipation about the prospect of tax reform that could be a repatriation of overseas cash.
Dealmaking has been a steady trickle over the last few months, with small deals dripping out every few weeks. This week was no different; Bristol-Myers Squibb agreed to pay $100 million upfront for the worldwide rights to Nitto Denko's lead candidate for its Non-alcoholic Steatohepatitis (NASH) candidate.
And as further evidence of its re-aligning, Bristol-Myers shed a preclinical chronic nerve pain drug to Lexicon Pharmaceuticals.
Several clinical trials got a red light from the U.S. Food and Drug Administration after safety concerns cropped up. Arrowhead tanked on news that its hepatitis B drug was put on full clinical hold due to a toxicity issue in animal studies. Swedish biotech BioInvent fell on similar news as its multiple myeloma trial was stopped due to adverse cardiopulmonary events in the trial. Both holds were only a verbal notice from the agency and written word, as well as more details, are expected to follow.
Biogen, on the other hand, and its partner Ionis got some positive news for their SMA drug when they announced Monday that the Phase 3 candidate showed statistically significant results in improving motor function for the rare disease patients. Biogen was quick to talk launch plans for the drug for both the U.S. and Europe.
Monthly decisions from the EU came in this week, with the CHMP giving positive recommendations to three biosimilars and 9 other medicines. The regulatory body also decided to extend PTC Therapeutics' conditional approval for its DMD drug Translarna, sending the stock up significantly. Meanwhile, Ultragenyx pulled back its application for EU approval of its GNE Myopathy treatment, driving shares down.
On this side of the Atlantic, the FDA hosted a meeting about the promotion of off-label use of medications. The meeting raised five issues that concern the FDA, including how off-label promotion could discourage sponsors from conducting clinical trials in that indication, how off-label promotion could make it harder to discern the real medical evidence and when would this be brought up with payers.
Off the bench
Amongst the other deals that Bristol-Myers signed this week was a five-year collaboration with John Hopkins University to study the development of resistance in patients taking checkpoint inhibitors like its PD-1 inhibitor Opdivo (nivolumab).
Researchers at Japan's Juntendo University demonstrated differentiation from stem cells into specialized cells that can be used to treat hereditary deafness. “Human cochlear cells are not readily accessible for biopsy or direct drug administration because of anatomical limitations,” said the researchers in a report. “Therefore, ES/iPS [embryo stem/induced pluripotent stem] cells are an important tool for studying the molecular mechanisms underlying inner-ear pathology as well as for generating cells for replacement therapies.”
Connecticut's NanoViricides has linked-up with SUNY Upstate Medical Center University to test the company's treatment for the shingles virus. The goals of the research will be to pick a lead drug candidate that can be tested in human trials for safety and efficacy.