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.
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Mergers & analysis
First quarter earnings continued this week with a bit of a whimper as biopharma majors like Pfizer and Gilead reported weaker-than-expected performance over the first three months of the year. Despite the lack of any significant dealmaking — the perennially ambitious Pfizer said a mega-merger is off the table — a handful of smaller tie-ups peppered the headlines this week.
United Therapeutics kicked off the week with a $216 million pickup of rival pulmonary arterial hypertension drugmaker SteadyMed. The smaller biotech has been a thorn in the side of the larger company while its drug-patch combo Trevyent has inched close enough to approval to make United Therapeutics nervous. The small buy will save it from having to deal with a competitive threat down the road.
Johnson & Johnson may be late to the party, but the multi-national added privately held BeneVir Biopharm to its portfolio in order to beef up its presence in immuno-oncology. The deal, which could eventually be worth as much as $1 billion, gives J&J an oncolytic virus platform to target solid tumors. BeneVir's candidates, however, are currently preclinical.
Obesity drugmaker Vivus, meanwhile, aims to avoid the fate of struggling competitor Orexigen Therapeutics, buying the North American rights to digestive compound Pancreaze (pancrelipase) from J&J. Vivus hopes the deal will begin a turnaround process for the company. With shares trading under $1, however, investors don't seem too enthused.
Cambridge-based biotech Ironwood Pharmaceuticals stirred up interest this week when it announced its plans to split into two publicly traded companies — one that maintains the gastrointestinal franchise and already marketed products, and another that will house the other R&D efforts. The announcement was followed later in the week with a letter to shareholders urging them to pick Ironwood's existing slate of board members over the activist investor Alex Denner. We will have to wait until an annual meeting on May 31 to better gauge shareholder sentiment.
Elsewhere, Allergan CEO Brent Saunders said during a first-quarter call with analysts that the pharma would keep all options on the table as it reviews how to respond to continued share price pressure.
Clinically relevant
Investors and analysts are questioning whether closely watched Esperion Therapeutics can eke past a clinical setback it announced this week. The company touted positive results from a long-term safety study of its oral cholesterol drug bempedoic acid, but shares fell sharply on the revelation that more patients died in the drug arm of the study compared to the placebo group.
Esperion insists the deaths are not due to the drug and are in high-risk patients with pre-existing conditions. But the seed of doubt has been sowed in investors' minds.
The IDO1 inhibitor class took a big hit this week too. After the hotly watched combination study of Incyte's epacadostat and Merck's Keytruda (pembrolizumab) failed, a number of companies in the immuno-oncology space have decided to cut their losses and discontinue some development of IDO blockers. Combination strategies in immuno-oncology are here to stay, but it doesn't appear the magic combination is going to be so easy to find.
On the other end of the oncology spectrum, Karyopharm took investors by STORM, reporting Phase 2b results for its drug selinexor in multiple myeloma that not only showed better-than-expected response rates than those typically needed for approval, but were well ahead of the rates seen at the interim analysis. The biotech now expects to submit an application for approval in the second half of the year.
Highly regulated
Novartis quietly gained a second indication for its historic CAR-T therapy Kymriah (tisagenlecleucel) in diffuse large B cell lymphoma (DLBCL), setting it up to compete directly with Gilead's similar CAR-T treatment Yescarta (axicabtagene ciloleucel). So far, sales of both drugs remain immaterial to either company, although a slow ramp-up was generally expected. Yescarta was a rare bright spot during Gilead's first quarter earnings call this week. The big biotech missed expectations in just about every area, but the CAR-T therapy came in more than double what analysts were expecting — posting $40 million in sales for the quarter.
Elsewhere, a speech by FDA Commissioner Scott Gottlieb appeared to set the stage for the Trump administration's forthcoming drug pricing announcement. Speaking to a group of lawyers at the 2018 Food and Drug Law Institute Annual Conference on May 3, the FDA head hinted the government could reexamine whether drugmaker rebates to insurers should be protected from anti-kickback statutes.