The J.P. Morgan event is known as the event for biotech and the investor community and, although typically aimed at generalists, bio journalists are always on the lookout for deals, critical updates, and using the conference as a litmus test for what the year ahead may bring.
We got a taste of that with a series of deals, many from Japan (and in fact Takeda taking much of the weight in that regard) with U.S. biotechs, but the one big deal that many lined up to see—one from Gilead—never materialized.
After the success of its Spinraza approval, FierceBiotech spoke to Ionis, one-half of the drug’s team with Biogen, about its trials and tribulations last year and its NDA hopes going forward, and we spoke with Bioverativ’s chief about the biotech’s unusual start to life.
Sarepta saw a 21% bump in its share price on Tuesday, Jan. 19, after it announced a new, early-stage research deal that could help treat all Duchenne muscular dystrophy (DMD) patients, and told investors in San Francisco that its new and controversial med for the disease, Exondys 51 (eteplirsen), had already made $5.4 million after its fall approval—above many analysts’ estimates.
Everyone wanted to listen to Gilead at the J.P. Morgan Healthcare Conference, but it did not come up with what many investors most wanted to hear: a major deal. Market watchers have been screaming for Gilead to deploy its massive cash pile, built up from its wildly successful hep C franchise, but M&A was not on the agenda.
CureVac had some disappointing news to report to investors at JPM after its lead drug failed a phase 2b trial in prostate cancer. The German specialist in mRNA-based drugs said CV9014 was unable to meet its primary objective of improving survival in the trial, which involved patients with asymptomatic or minimally symptomatic metastatic castration-resistant prostate cancer.
Moderna, often accused of being secretive after failing to reveal, publicly at least, its pipeline of meds, finally did so at the start of the JPM week on Monday, Jan. 9, with its candidates targeting a host of infectious diseases, I-O and CV diseases. In one of the more anticipated sessions of the conference, the private company, believed to be worth up to $5 billion, said it now has 12 mRNA development candidates, with five now in the clinic.
Most biotechs start life with little money, one or several early-stage assets, a handful of employees and big dreams but face an uphill struggle to stay alive, let alone get to market with a new med. Biogen’s new spinoff Bioverativ, however, will be trading on its own on the Nasdaq next from the start of next month with two products, $325 million in the bank, hundreds of staffers and a new preclinical pipeline of meds for noncancer blood disorders and cash to boost its R&D efforts. We spoke to its new CEO.
Ionis told FierceBiotech that it is seeking to get its heart drug volanesorsen in front of the FDA as soon as possible, with its CEO Dr. Stanley Crooke saying the biotech is “gearing up to file the NDA in a very short time after we get our latest phase 3 results.” We also spoke about why the biotech quietly dropped another R&D project with Biogen at the start of the month.
And finally, when everything seemed to be OK, the Nasdaq biotech index and the S&P biotech both took a nosedive when, during a rare media press conference toward the end of JPM (but not of course connected to the event), PEOTUS Donald Trump raised the spectre of drug pricing again with a vague but forceful promise to introduce a bidding process into pricing.