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
- MedCo gains antibiotic approval ahead of potential sale
- Lilly readies a refiling for baricitinib
- Celgene pays out to extend deal with Forma
The Food and Drug Administration made multiple historic (and some odd) moves this week, demonstrating that we really are in a new era under Commissioner Scott Gottlieb. Arguably one of the biggest events in the industry this year was the approval of Novartis AG's CAR-T therapy Kymriah (tisagenlecleucel). The FDA dubbed it the first U.S. gene therapy approval, causing many to raise questions about what exactly the moniker 'gene therapy' means (many people consider CAR-T to be cell therapy.) For the big pharma, this is a huge win, but don't break out the champagne yet. As Dendreon (and gene therapies in Europe) taught us some years ago, even highly innovative therapies can fail to gain commercial traction.
Beyond the Novartis approval, which was more than a month ahead of its user fee goal date, the FDA approved several other therapies this week, including a drug that could treat the side effects of CAR-T therapy. It also issued two refuse-to-file letters, handing back New Drug Applications before even starting a review to both Acorda and SteadyMed. This is a move that the old regime FDA rarely made.
Another unusual move by the agency was the alert sent out yesterday regarding Merck & Co.'s blockbuster PD-1 inhibitor, Keytruda (pembrolizumab). It was revealed earlier this summer that there were a disproportionate number of deaths in two clinical trials testing Keytruda in combination with other drugs in patients with multiple myeloma. Shortly after, the FDA put clinical holds on three of the big pharma's clinical trials. The alert this week didn't add much new information, but did reveal that the agency is looking closely at other drugs in the class, which could spell problems for a number of companies.
Mergers & analysis
In what appeared to be a prophetic turn of events, Gilead Sciences Inc. finally (after years of investor whining) dipped into its pile of cash to start the week off with a $11.9 billion acquisition of Novartis CAR-T competitor KITE Pharma Inc. The biotech is also waiting on an approval from the FDA for its CAR-T therapy (albeit in a different patient population).
This acquisition — and Novartis' subsequent approval — will set up the two companies for an intriguing pricing war. Novartis set the price of its drug at $475,000, substantially lower than the market was expecting, but still incredibly expensive. While the Kite drug is looking to serve a larger patient population, Gilead has a reputation for setting sky-high prices. It will be interesting to see if Gilead will choose to go with the higher price tag anyway and if the Kite drug can be commercially competitive.
Going under the radar after the Gilead deal, CSL Behring decided to get into the gene therapy arena as well this week, shelling out $91 million upfront to acquire Calimmune and its stem cell gene therapy.
Other companies pulled out of deals this week. Daiichi Sankyo Co. Ltd. is continuing its restructuring plan in an effort to become an oncology powerhouse. The Japanese pharma gave back the rights to a group of hydrochodone product to Charleston Laboratories after the lead compound got an FDA rejection earlier this year. The company also handed off a preclinical asset to Boston Pharmaceuticals. Five Prime Therapeutics also ended a collaboration with La Jolla biotech Inhibrx.
Merck finally revealed this week the results from its hotly anticipated REVEAL cardiovascular outcomes study. While the big pharma noted earlier in the summer that the results for its CETP inhibitor were positive, the cholesterol drug only reduced the risk of major coronary events by 9%. The only mildly positive level of efficacy means that the drug is probably not a commercial success. And Merck knows it — the company is still weighing whether or not it's worth filing for approval. Analysts say it's not worth it.
Elsewhere, Otonomy Inc. watched its stock plummet after announcing a Phase 3 failure of its vertigo treatment. The company opted to discontinue development of the drug and is still looking into why the compound failed.