Bio Roundup: Spark v. ICER, A Solid Revelation, T Cell Deals & More

Xconomy National — 

The United States has a new Health and Human Services chief who is expected to weigh in on the country’s drug-pricing debate. But how Alex Azar, a former pharma executive at Eli Lilly (NYSE: LLY), plans to throw his weight around remains to be seen.

One of the latest exhibits in the debate is an $850,000 gene therapy to treat a rare, inherited form of vision loss. Xconomy examined its owners’ justification for the price, and why a drug-value watchdog pushed back hard. We’ve got more gene therapy coverage, plus news about big M&A—perhaps the first big splashes from incoming waves unleashed by the Republican tax cut.

Sure, Chinese scientists have produced the world’s first cloned monkeys, but we’ve got something that can never be duplicated. Let’s get to the roundup.


—Spark Therapeutics (NASDAQ: ONCE) priced its blindness treatment voretigene neparvovec (Luxturna), the first gene therapy approved in the U.S., at $850,000. The nonprofit Institute for Clinical and Economic Review believes it should cost much less. Xconomy delved into the difference of opinion, and why it illustrates a drug valuation problem only likely to become more common.

—Meanwhile, Novartis (NYSE: NVS) likes Luxturna’s value. It paid Spark $105 million upfront for rights outside of the U.S. Spark is still responsible for securing European approval.

—Two late disclosures from the $125 million IPO of Solid Biosciences (NASDAQ: SLDB) have highlighted questions about the potential treatment of Duchenne muscular dystrophy with gene therapy.


—Sanofi (NASDAQ: SNY) agreed to pay $11.6 billion for Bioverativ (NASDAQ: BIVV), in a gamble that the Biogen spinoff’s hemophilia drugs will continue to generate sales in the face of emerging, cutting-edge competition.

—Celgene (NASDAQ: CELG) turned its 10 percent stake in CAR-T developer Juno Therapeutics (NASDAQ: JUNO) into full ownership, a $9 billion buyout that pins hopes on approval of Juno’s JCAR017 lymphoma treatment in 2019. The deal means the first wave of CAR-T products, requiring complicated manufacturing and logistics, are all in the hands of rich, powerful biopharma firms.

—Tmunity Therapeutics, spun out of the University of Pennsylvania more than a year ago, raised $100 million in a Series A funding to develop its pipeline of next-generation T cell cancer immunotherapies.

—The New York City Economic Development Corp. has put up $100 million and a call for ideas to create a sprawling New York campus called the Applied Life Sciences Hub. Xconomy spoke with the NYCEDC and others in the New York biotech scene about the ingredients required to make an impact.

—Boston Scientific (NYSE: BSX) invested $90 million in Santa Rosa, CA, heart device developer Millipede. The deal includes an option to acquire the startup.

—Redwood City, CA-based Menlo Therapeutics (NASDAQ: MNLO) raised $119 million in its IPO to finance mid-stage studies of serlopitant, a treatment for itching associated with skin conditions.

—Boston-based ResTORbio, an affiliate of PureTech Health, bagged $85 million an IPO to fund testing of a drug being developed for immunosenescence, an age-related decline in immune function.

—Eyenovia (NASDAQ: EYEN) raised $27 million with its IPO, which the New York startup will use to develop micro-dose versions of approved eye drugs.

—Research Triangle Park, NC-based BioCryst Pharmaceuticals (NASDAQ: BCRX) will merge with Idera Pharmaceuticals in a stock deal that brings together two rare disease drug developers.

—Tech entrepreneur Bill Haney has seeded his second biotech startup, Cambridge, MA-based Skyhawk Therapeutics, with the same private family offices (including the Disney family) he used to fund Dragonfly Therapeutics.

—Biogen (NASDAQ: BIIB) paid Karyopharm Therapeutics (NASDAQ: KPTI) $10 million upfront for rights to an experimental neurological disease drug, KPT-350, in preclinical testing.


—The Senate confirmed Alex Azar as the new HHS secretary, giving the former drug executive oversight of the nation’s federal insurance programs, public health and biomedical research, and much more. Azar has spoken publicly against the Affordable Care Act, has contributed to anti-abortion politicians, and clerked for the late Supreme Court Justice Antonin Scalia.

—Included in the temporary funding measure that ended a government shutdown was a six-year extension of the Children’s Health Insurance Program, which covers families who don’t qualify for Medicaid but can’t afford private insurance.

—The National Institutes of Health launched a $190 million, six-year funding program to improve gene editing in humans.

—Apple (NASDAQ: AAPL) unveiled new features to let iPhone users gain access to their health information. A dozen major health centers, including Johns Hopkins Medicine and UC San Diego Health, are the first to open their systems to their customers.

—Xconomy San Diego editor Bruce Bigelow reported on Luna DNA, a startup run by Illumina (NASDAQ: ILMN) alumni that is developing blockchain technology and “Luna coins” to encourage people to take control of their own genomic information.

— The FDA issued a warning to companies it says are selling opioid addiction treatments that are either unapproved or are making unsupported claims.


—Amgen (NASDAQ: AMGN) reported strong Phase 3b results for its migraine drug erenumab (Aimovig) to which it shares rights with Novartis. The FDA has a May 17 deadline to consider the drug’s approval.

—Puma Biotechnology (NASDAQ: PBYI) shares lost about a quarter of their value Wednesday after European regulators gave Puma’s drug neratinib a negative review for breast cancer. Neratinib was approved in the U.S. last year despite a marginal benefit over placebo and side effects of severe diarrhea.

—The FDA rejected Braeburn Pharmaceuticals’ buprenorphine injection for treating opioid addiction. Princeton, NJ-based Braeburn did not explain why the FDA rejected its drug, but said no new clinical trials are needed.

Ben Fidler and Frank Vinluan contributed to this report.

Image: Joe Flintham via a Creative Commons license.