Welcome to conference week. That’s not an official moniker, but is there any busier time on the biopharma calendar? As the American Society of Clinical Oncology meeting wrapped up in Chicago, the annual BIO conference kicked off in Philadelphia, and a deep dive into diabetes begins today in San Francisco.
With half the biopharma world, it seems, either on an airplane or roaming the halls of a conference center, it’s possible you missed other headlines, such as Europe’s milestone approval of a gene therapy to treat the rare blood disorder beta thalassemia. It’s not the first gene therapy nod in the EU, but the first two never gained traction; both the promise and the price of potential cures are central right now in a crucial debate on this side of the Atlantic.
We’ll get to that that story and more, including comments from the new FDA commissioner, new IPO hopefuls, and a Washington Post story that poked a stick in the biopharma beehive. What’s that buzz? It’s our weekly roundup.
The Chinese scientist who modified the DNA of the so-called CRISPR babies to give them immunity to HIV infection might have put them at risk of a shorter lifespan, according to a new study. University of California, Berkeley researchers analyzed data from more than 400,000 people in the UK and told Xconomy they want to repeat the study in other populations.
—Another year, another ASCO. Long-term results for immunotherapy, a glimmer of hope in pancreatic cancer, and first data from what’s long been considered an “undruggable” target: We rounded up some of the headlines here.
—Two studies released at ASCO showed that the Affordable Care Act has helped narrow racial disparities in cancer care, and that patients with ovarian cancer were receiving earlier diagnoses and treatment.
—Acting FDA commissioner Ned Sharpless kicked off a different conference, the international BIO shindig in Philadelphia, with an onstage chat. He encouraged more use of real-world evidence to evaluate drugs and called the media “cynical” for fixating on the $2 million price tag of newly-approved gene therapy Zolgensma.
—Another medical conference is upon us: the American Diabetes Association gathering in San Francisco this weekend. There are pharmaceutical trends and results to watch for, but there’s a broader context to the meeting this year: US diabetes cases appear to be dropping, even while obesity rates keep expanding.
Politics & Policy
—The Trump administration is halting National Institutes of Health research using human fetal tissue acquired via elective abortions, which received $31 million last year. NIH funding of academic projects, which received about $84 million last year, will continue but only under lengthy case-by-case review. NIH is also cancelling three major projects, including one at the University of California, San Francisco.
—For the second straight year ACA rate hikes seem modest, Politico reports.
—The FDA announced Project Facilitate, a call center to help doctors apply to use experimental therapies for cancer patients outside the bounds of clinical trials.
Deals & Dollars
—Third Rock Ventures closed a $770 million fund, the life sciences venture firm’s fifth.
—Deerfield Management and Columbia University formed Hudson Heights Innovations to invest up to $130 million over 10 years to develop Columbia research into new therapies. Hudson Heights will have options to license drugs from the partnership.
—The biotech IPO train picked up two more passengers. Boston neurology drug developer Karuna Health and Inhibrix, a La Jolla, CA-based cancer drug developer, each set preliminary $75 million targets for their IPOs.
—EpimAb Biotherapeutics of Shanghai, China, closed a $74 million Series B round to advance its pipeline of experimental cancer drugs.
—Lung Therapeutics raised $36M in Series C funding. The Austin, TX, biotech is preparing to start clinical trials with its idiopathic pulmonary fibrosis drug and advance its therapy for loculated pleural effusions, a pneumonia complication, to Phase 2 testing.
Data & Decisions
—More expansion: The FDA said ceftolozane and tazobactam (Zerbaxa), a Merck (NYSE: MRK) combination antibiotic initially approved for abdominal and urinary tract infections, could also treat pneumonia acquired in a hospital or from a ventilator.
—Takeda Pharmaceutical (NYSE: TAK) halted a Phase 3 trial in light-chain amyloidosis after its drug failed to achieve one of the study’s two goals. The drug, ixazomib (Ninlaro), is already approved to treat multiple myeloma.
—The Washington Post raised eyebrows with a report that Pfizer (NYSE:PFE) quashed a pursuit of blockbuster autoimmune drug etanercept (Enbrel) as an Alzheimer’s treatment. Chemist and commentator Derek Lowe posted a critical take on the Post story.
—The clock has started on the FDA’s review of ozanimod, one of the five drugs in the Celgene (NASDAQ: CELG) pipeline that Bristol-Myers Squibb (NYSE: BMY) has touted to justify its $74 billion acquisition of the New Jersey biotech. The deadline for an approval decision is next March 25.
People on the Move
The Bristol-Myers Squibb-Celgene megadeal is leading to management shakeup. Bristol CEO Giovanni Caforio will remain at the top but chief scientific officer Tom Lynch is leaving. The company has hired Samit Hirawat away from Novartis (NYSE: NVS) to become chief medical officer.
—Patricia Hurter is leaving Vertex Pharmacecuticals (NASDAQ: VRTX) to become CEO of Lyndra Therapeutics… Pfizer has hired Jeff Settleman away from Calico to run its cancer R&D in San Diego… Immunovant hired Eli Lilly’s Pete Salzmann as CEO… and LabCorp (NYSE: LH) CEO David King is retiring.
Frank Vinluan contributed to this report.