Sometimes disruption comes in waves. Since the 1990s, hemophilia patients have had to inject themselves with drugs a few times a week to prevent dangerous internal bleeding. That could soon change. One drug already approved, from Roche, may only need to be taken once a month by hemophilia A patients. Another experimental therapy from Alnylam Pharmaceuticals might offer similar benefits for both hemophilia A and B patients. And then there’s gene therapy, a single treatment that might keep bleeding at bay for years and obviate the need for other drugs. Pivotal testing should start next year.
Many of these treatments were the topic of discussion at a big hemophilia meeting this week, and to be sure, they each have important questions to answer. But it’s not a stretch to say that for many born with the disease, hemophilia could be a lot easier to manage five or ten years from now. That story and the rest of the week’s headlines below.
HEMOPHILIA HAPPENINGS
—Swiss drugmaker Roche revealed detailed data from two studies, HAVEN-3 and HAVEN-4, that could make its antibody drug emicizumab (Hemlibra) the choice option for more people with hemophilia A. Safety concerns and emerging competition, however, will challenge both the drug’s rise and its staying power.
—Some of the competition in hot pursuit of emicizumab is coming from gene therapy, which could offer a long-lasting effect with a single infusion. The companies developing hemophilia gene therapies—BioMArin Pharmaceutical (NASDAQ: [[ticker:ONCE]]), Spark Therapeutics (NASDAQ: [[ticker:ONCE]]), and UniQure (NASDAQ: [[ticker:QURE]])—each updated their experimental programs. Here’s more on those updates from BioPharma Dive.
ON CAPITOL HILL
—Congress passed a “right to try” bill that gives terminally ill patients easier access to experimental drugs without enrolling in a clinical trial. President Trump is expected to sign it, a major victory for a once-obscure movement led by a libertarian think tank. Opponents say the FDA’s current “compassionate use” program already works well, and that the new law will add more risks than benefits for patients. FDA commissioner Scott Gottlieb tweeted his support last week.
—At a speech at the meeting before the Alliance for Regenerative Medicine, Gottlieb outlined steps the agency is taking to update its policy and approval pathway for gene and cell therapies.
—AstraZeneca (NYSE: [[ticker:AZN]]) won FDA approval of sodium zirconium cyclosilicate (Lokelma), a drug that treats hyperkalemia, a condition in which high potassium levels are found in the blood. AstraZeneca bought the drug when it acquired ZS Pharma for $2.7 billion in 2015.
—The agency also approved BioMarin’s pegvaliase, a drug for the rare genetic disease phenylketoneuria. The drug has an yearly list price of $267,000 and an average annual net price of $192,000, according to reports.
—“All of Us,” the National Institutes of Health’s longterm study of the health of as many as 1 million volunteers, will award contracts to two genome centers to analyze participants’ genomic data. Applications are due in July.
BRII RISES & NEW STARTS
—Led by former GlaxoSmithKline (NYSE: [[ticker:GSK]]) infectious disease executive Zhi Hong, startup Brii Biosciences unveiled $260 million in financing and a plan to acquire and develop new drugs to bring to the market in China.
—Xconomy posted a slideshow and look back at “What’s Hot in Boston Biotech,” which examined the implications of the recent launches of gene therapy and CAR-T, among other issues.
—Norbert Bischofberger, the longtime CSO at Gilead Sciences (NASDAQ: [[ticker:GILD]]) is now president and CEO of Kronos Bio, a Cambridge, MA, startup. Bischofberger stepped down from Gilead last month. Former Kite Pharma CEO Arie Belldegrun is chairman and a main backer of Kronos, which is developing cancer drugs.
—Endpoints News reported that George Golumbeski, a longtime top dealmaker at Celgene (NASDAQ: [[ticker:CELG]]), left the company on April 16 and has become an independent consultant. Former Celgene dealmaking colleague Thomas Daniel took a similar path two years ago.
—Fremont, CA-based Rain Therapeutics closed an $18 million Series A to fund a Phase 2 trial for a targeted lung cancer drug, tarloxotinib.
—Dutch startup Lava Therapeutics raised €16 million to pursue bispecific cancer drugs that simultaneously target tumor cells and stimulate a specific type of a patient’s immune cells.
DATA DUMPS
—A combination of chemotherapy and the Merck (NYSE: [[ticker:MRK]]) immunotherapy pembrolizumab (Keytruda) again succeeded in a lung cancer trial, this time in a study of patients just diagnosed with squamous non-small cell lung cancer. Merck will reveal more specifics from the study, Keynote-407, at the American Society of Clinical Oncology’s meeting next month.
—AstraZeneca posted more positive data from its PACIFIC trial, which led to FDA approval of its immunotherapy durvalumab (Imfinzi) for unresectable Stage 3 lung cancer patients whose disease hasn’t spread after chemotherapy.
—Esperion Therapeutics (NASDAQ: [[ticker:ESPR]]) of Ann Arbor, MI, released positive results from the third of five pivotal trials for its cholesterol-lowering drug bempedoic acid. If eventually approved, bempedoic acid, a pill, will be offered as a lower-cost version of the expensive injectable drugs known as PCSK9 inhibitors.
ON THE IPO TRAIL
—Two Boston-area biotechs, Scholar Rock (NASDAQ: [[ticker:SRRK]]) and Kiniksa Pharmaceuticals (NASDAQ: [[ticker:KNSA]]), raised $227 million combined in their respective IPOs, continuing a prolonged, strong run for biotech offerings.
—Neurological drug developer Aptinyx of Evanston, IL, signaled its intent to go public on the Nasdaq under the symbol APTX. The firm is working on drugs that target the NMDA receptor on neurons.
—Magenta Therapeutics also outlined plans to go public. The Cambridge company is trying to develop drugs that can help improve bone marrow transplants.
DEALS, FUNDINGS & RESTRUCTURINGS
—Grail, a developer of a blood test to detect early cancer, raised another $300 million, bringing its total fundraising past the $1.5 billion mark.
—Celgene paid Evotec $65 million to co-discover and develop cancer drugs. It also announced $3 billion in share buybacks.
—San Francisco’s Verge Genomics is partnering with two academic centers to study gene activity in the brain cells of Parkinson’s disease patients. Xconomy profiled Verge CEO Alice Zhang in 2016.
—Sublimity Therapeutics raised $64 million in financing that the Dublin, Ireland company will use to continue mid-stage clinical testing of an ulcerative colitis drug.
—Pfizer and Merck led a $26 million Series B round for startup Strata Oncology, which aims to get cancer patients better access to tumor sequencing tests.
—Astellas Pharma is the latest big pharma company to cut jobs as part of a corporate restructuring. Fierce Biotech reports that the Tokyo-based company is offering an early retirement program hoped to trim 600 jobs in R&D, as well as sales and marketing.
Frank Vinluan and Alex Lash contributed to this report.
Photo by Flickr user Julien Mattei via a Creative Commons license
