Another bout of rain grayed skies over J.P. Morgan Healthcare Conference in San Francisco this year. Unlike last year, however, there was more excitement and a bit of buzz as the industry expects deals to pick up now that a tax overhaul is complete. Early signs point to some activity on that front, led by Celgene Corp.'s acquisition of Impact Biomedicines Sunday night.
The conference boasts 450 presenting companies, as well as more than 10,000 attendees.
Heard 'round the halls
Around the halls of the Westin St. Francis and the Hilton Union Square (where the simultaneous Biotech Showcase is being held), conference goers heard much talk over gene therapy and CAR-T. Panelists on a state-of-the industry panel on gene therapy acknowledged that reimbursement might just be the trickiest part of these innovative therapies.
Elsewhere, a session on the microbiome noted there is more to the space than fecal transplants and 'bugs as drugs.' In fact, it might surprise some to hear a number of therapeutic areas are now being investigated as potential spaces where the microbiome could play a role. Traditionally, microbiome research has focused on the gastrointestinal tract and infectious skin disorders, but panelists suggested oncology actually could be a hot space as well. One participant even suggested the microbiome in cancer could outgrow immuno-oncology, and is only about five years behind. Guess we'll have to wait and see.
Celgene: We're fine ... really
Celgene kicked off the conference news early, announcing Sunday night a potential $7 billion deal to acquire Impact Biomedicines and its NDA-ready blood disease drug fedratinib.
On Monday, the big biotech opened the meeting's first official day with a presentation aimed at reassuring investors worried about long-term growth. CEO Mark Alles spoke excitedly about the prospects of new drugs like Otezla (apremilast) and Idhifa (enasidenib), while highlighting a line-up of ten experimental candidates he thinks can be billion-dollar earners by 2022.
In the nearer term, the company expects double-digit revenue growth in 2018, and pre-announced Q4 earnings per share that beat market expectations.
But the sunny outlook didn't appear to totally convince investors shaken by last year's surprise cut to revenue guidance. Celgene shares dropped by nearly 4% Monday morning, before gaining back some of those losses.
Analyst reaction to the Impact deal was positive, but with only modest enthusiasm for fedratinib's potential contribution.
"This deal alone is unlikely be a major needle-mover for the company," wrote Mizhuo Securities analyst Salim Syed in a Jan. 8 note, while acknowledging the deal's structure helped mitigate risk.
Alnylam taps brake on near-term patisiran sales forecasts
Sometime around the middle of this year, Alnylam Pharmaceuticals Inc. is expected win the first-ever approval of a RNAi therapeutic. The drug, patisiran, showed dramatic efficacy in a Phase 3 study of patients with a rare disease known as hereditary ATTR amyloidosis.
Shares in Alnylam have risen by 70% since the biotech announced the drug's clinical success, pushed up by hopes of the drug's commercial potential.
On Monday, CEO John Maraganore sought to temper some of those expectations of an overnight fortune in the making.
"Clearly this is an area where medical education will need to improve. Diagnosis rates will need to improve. And because of that, we believe that some of the analyst models today are a little bit over-aggressive on the near term for revenue growth for patisiran but, frankly, are too conservative in the mid-to longer-term," he said.
One area of uncertainty is whether the Food and Drug Administration will agree with the company's request for a broad label in the disease. A more limited indication could close off some patients — although Alnylam contends the drug delivers a clear benefit for anyone with hATTR amyloidosis.
J&J still bullish on cardio/metabolic
Cardiovascular and diabetes were once the hot blockbuster spaces every big pharma wanted to join, but the patent cliff and pushback from payers spurred most companies to leave the space. But not Johnson & Johnson.
The mega multinational only has two noteworthy drugs on the market in the space — the anticoagulant Xarelto (rivaroxaban) and the SGLT-2 inhibitor Invokana (canagliflozin), which respectively brought in about $1.8 billion and $844 million over the first nine months of 2017.
But Jim List, Janssen's head of cardio and metabolic, told BioPharma Dive the research currently being conducted on just these two drugs "potentially will change the way that we treat these very common diseases."
J&J divides the cardio/metabolic space into three 'buckets': diabetes/obesity, NASH/chronic kidney disease and thrombosis. Either Invokana or Xarelto is under study in each of these areas, but the company also has a slew of drugs in earlier stages of its pipeline.
"We've matured the current view of innovation, but there's plenty of room for further innovation," said List, who argued for innovation that isn't just incremental, but monumental. And he said that J&J believes in setting the bar higher.
List admits he's "never satisfied with our pipeline," and that the company is agnostic about whether innovation comes from internal or external sources. But J&J isn't looking to add to its late-stage assets necessarily; the company likes to add value through expertise into its deals, which usually happens in earlier stage deals, List says.
"We're looking for innovative deals, not financial deals," he added.
Axovant calls it quits on intepirdine
When Axovant Sciences revealed its closely watched Alzheimer's disease candidate intepirdine failed to beat out placebo, company CEO David Hung had sounded an optimistic note on the drug's potential in dementia.
Now that hope has evaporated, too. Axovant said Monday it would discontinue the intepirdine program after the drug failed to meet its primary efficacy objective in the Phase 2b HEADWAY study.
Shares in the biotech promptly halved in value and now trade at 90% of the stock price before Axovant announced its setback in Alzheimer's last September.
Looks like GlaxoSmithKline plc, the drug's original developer, had it right when it sold the drug for $5 million in 2014.