Patients, doctors, family members, and drug-price watchdogs continued to wait for the expected approval of Zolgensma, a Novartis (NYSE: NVS) gene therapy for the rare inherited disease spinal muscular atrophy. Originally developed at a children’s hospital in Ohio, Zolgensma will be a litmus test for the nascent field. If approved this week or next, it would be just the second gene therapy to come to market in the US.
Novartis CEO Vas Narasimhan made sure Zolgensma stayed in the headlines. In an op-ed posted on CNBC this week, Narasimhan wrote that the U.S. healthcare system must adapt to pay for one-time treatments, like gene and cell therapies, that are meant to last a lifetime. He then told reporters at an event in Boston that, while Zolgensma would be cost-effective in the $5 million range, Novartis expects to price it “far lower than that.” Drug-value watchdog ICER said last month that Zolgensma could bear a fair value of no more than $900,000.
A big sticking point: there are no long-term data to bear out the assumption of a cure. It remains a hope, in particular for patients and their families, but part of the Zolgensma reality check will be insurers’ willingness to pay huge sums upfront without assurance of a cure. Or will Zolgensma’s arrival, due by month’s end, force healthcare players to devise creative ways to share the substantial financial risk? While you ponder that question, check out the rest of the week’s news. It’s roundup time.
POLICY & REGULATION
—The US Supreme Court ordered a lower court to reconsider a decision to allow lawsuits to proceed against Merck (NYSE: MRK). The suits allege Merck failed to warn patients about bone fracture risks associated with its osteoporosis drug alendronate (Fosamax). Merck argued that it wanted to add the warning to the drug’s label, but the FDA denied the request.
—Former FDA commissioner Scott Gottlieb returned to the venture firm New Enterprise Associates, where he had spent a decade before his two-year tenure in the Trump administration.
—Health data and community site PatientsLikeMe was forced into a fire sale last month because a Chinese digital health company had a majority ownership stake. CEO Jamie Heywood spoke publicly for the first time about the situation and the federal government’s scrutiny of foreign investments.
DATA UPS AND DOWNS
—With the American Society for Clinical Oncology meeting coming up, BioPharma Dive looked at five ways drug companies are advancing beyond the first wave of cancer immunotherapy drugs, known as checkpoint inhibitors.
—The most successful checkpoint inhibitor, Merck’s pembrolizumab (Keytruda), failed to best chemotherapy in a Phase 3 study with triple-negative breast cancer patients who have failed one or two therapies. Merck continues to test immunotherapy regimens in other breast cancer trials.
—It was a bad week for brain cancer patients. Last Friday, an antibody-drug conjugate from AbbVie (NYSE: ABBV) known as Depatux-M failed a Phase 3 study in newly diagnosed patients. This week, San Diego’s Tocagen said it would press ahead after an interim look at Phase 3 data for its unusual gene therapy. Investors took the news as a bad sign that the data weren’t positive enough to end the trial early.
—The Medicines Co. (NASDAQ: MDCO) reported that patients in an open-label extension study of its drug inclisiran saw their “bad” cholesterol levels fall by an average of 51 percent with no significant side effects. Some patients have been treated up to three years. Phase 3 data for inclisiran, an RNA interference drug administered twice a year, are expected in the third quarter.
—Four drug makers said they would use data tools and techniques developed by Verily’s Project Baseline in future clinical trials. Here’s more about Baseline.
—OTX-TP, an implantable device from Ocular Therapeutix (NASDAQ: OCUL) that dispenses the glaucoma drug travoprost into the eye, failed a Phase 3 trial. Ocular says it will meet with the FDA to discuss the drug’s future.
DEALS AND DOLLARS
—Merck agreed to pay $1 billion for cancer drug developer Peloton Therapeutics of Dallas. The deal was announced the day before Peloton was expected to price its IPO.
—The London nonprofit LifeArc sold its royalty interest in pembrolizumab to the Canada Pension Plan Investment Board for $1.3 billion. LifeArc will invest the proceeds in new experimental treatments. The Financial Times has more.
—Houston-based AlloVir closed $120 million in Series B funding to back a Phase 3 study of its cell therapy for viral infections. The startup also joined ElevateBio, a new company building a manufacturing facility for cell and gene therapies in Waltham, MA.
—The New York Stock Exchange is lowering fees for pre-revenue companies to make itself more competitive with the Nasdaq, which has been the preferred exchange for biotech companies preparing IPOs. The Wall Street Journal has more.
—Nektar Therapeutics (NASDAQ: NKTR) launched a subsidiary, Inheris Biopharma, which will take over an opioid painkiller submitted for FDA approval and several preclinical programs for central nervous system disorders.
—Cala Health raised $50 million in Series C financing to support commercialization of a wrist-worn medical device that uses electric stimulation to control hand tremors.
—Amgen (NASDAQ: AMGN) offered $167 million to buy Danish firm Nuevolution, which has impressed Amgen with its drug discovery capabilities.
—Schrödinger, a maker of computational software used in drug discovery, added new investors to its most recent financing round, which now totals $110 million.
—The New York Genome Center received $125 million from two foundations, earmarked for the center’s work on neurological disease and cancer.
—German firm Evotec paid $60 million, with $30 million more in potential milestones, for Seattle’s Just Biotherapeutics, a developer of biologic medicines. Just received funding from the Bill and Melinda Gates Foundation to support its effort to make biologics affordable in developing countries.
—K2 Healthcare Ventures launched a new $400 fund mainly to provide debt financing to healthcare companies. K2 said it will focus on “underserved and underinvested areas” of healthcare but did not get more specific.
PEOPLE ON THE MOVE
Voyager Therapeutics (NASDAQ: VYGR) chief scientific officer Dinah Sah is retiring in June… former Arsanis (NASDAQ: ASNS) executive René Russo was named CEO of Akrevia Therapeutics… and Greg Ryslik joined Celsius Therapeutics as chief data officer.
Ben Fidler and Frank Vinluan contributed to this report.