The 21st Century Cures Act is now law. New York wants to play in the biotech big leagues. The FDA under Trump will have license to approve drugs faster and could have a former top official back on board as commissioner. An eye drug failed, an Alzheimer’s drug got scrutinized, and a new antibiotic passed two key tests. Let’s get to the roundup…
—In what is likely the last major legislative action of his eight-year presidency, President Barack Obama signed into law the 21st Century Cures Act to boost NIH funding for projects his administration kickstarted. The bill also pledges $1 billion to state governments to combat the opioid crisis, which has created a heartbreaking litany of family upheaval, and reforms mental health policy.
—Many parts of the Cures Act look to change the FDA’s product review process, bringing kudos from some patient advocates, among others, but warnings of lax standards from pharma watchdogs and consumer groups. Adding to speculation about the FDA’s role in the upcoming Trump administration, a new potential candidate for commissioner emerged: Scott Gottlieb, a doctor with ties to drug companies and the investment world. Gottlieb was a top FDA official in the George W. Bush administration.
—In the span of 24 hours, the state of New York and New York City committed a combined $1.15 billion to life sciences economic development in the Empire State. Both the state and city plans are long-term efforts that would use tax incentives, paid internships, money for startups, and perhaps most important, make available millions of square feet of laboratory space.
—Jefferies analyst Gena Wang asked payers and doctors their opinion of Duchenne muscular dystrophy drug eteplirsen (Exondys 51), which Sarepta Therapeutics (NASDAQ: SRPT) priced at $300,000 per patient after a controversial FDA approval this year. Based on the survey, Wang expects “stronger payer pushback” and a slower launch of the drug, which runs counter to analysts and patient advocates whom Xconomy spoke with in October. Sarepta’s shares have been cut in half since eteplirsen was approved in September.
—Gilead Sciences (NASDAQ: GILD), already slumping due to slowing sales, was dealt a blow late Thursday when a federal jury ruled that the Foster, CA, company’s two hepatitis C drugs, sofosbuvir (Sovaldi) and sofosbuvir-ledipasvir (Harvoni) infringed on patents held by Merck (NYSE: MRK) through its buyout of Idenix Pharmaceuticals. Gilead was ordered to pay Merck $2.54 billion, though as Bloomberg reports here, Gilead plans to appeal.
—Alexion Pharmaceuticals (NASDAQ: ALXN) said Monday its CEO and CFO were resigning, effective immediately and with little explanation, and the new CEO said it was time to “move on.” The company revealed last month it was conducting an internal investigation of sales fraud allegations and would delay its quarterly financial report. Alexion’s main product is a drug for a rare disease that costs hundreds of thousands of dollars a year.
—Bristol-Myers Squibb (NYSE: BMY) announced plans to close two R&D facilities in Hopewell, NJ, and Seattle, open a new site at its Lawrenceville, NJ, campus, and expand two other existing hubs in New Brunswick, NJ, and Devens, MA. Bristol has already said it is closing a plant in Wallingford, CT, that employs about 800.
CLINICAL UPS AND DOWNS
—Ophthotech’s (NASDAQ: OPHT) pegpleranib (Fovista), a highly anticipated drug for a common form of vision loss called age-related macular degeneration, failed two Phase 3 trials, sending shares down more than 80 percent. The drug had a chance to change the standard of care for the “wet” form of AMD, but its future is now unclear.
—Researchers dug through data in the latest failed Phase 3 trial of Eli Lilly’s Alzheimer’s drug solanezumab. The result, according to experts queried by Xconomy: solanezumab isn’t quite dead yet, and neither is the “amyloid hypothesis” that many believe explains the cause of Alzheimer’s. Meanwhile, positive early signals from another amyloid-clearing Alzheimer’s drug, aducanumab from Biogen (NASDAQ: BIIB), should be viewed with very cautious optimism.
—Safety problems caused Cambridge, MA-based Agios Pharmaceuticals (NASDAQ: AGIO) to scrap one of the two drugs it has been developing for a rare type of anemia called pyruvate kinase deficiency. Work will continue on the other anemia drug, AG-348, currently in Phase 2 trials.
—Achaogen (NASDAQ: AKAO) reported that its lead antibiotic product produced positive data in two trials, one to combat a drug-resistant “nightmare bacteria,” the other to fight urinary tract and kidney infections. The firm plans to ask U.S. and European regulators for approval.
—Proteon Therapeutics’ (NASDAQ: PRTO) lead drug for chronic kidney disease patients failed one of two Phase 3 tests, sending the Waltham, MA-based company’s shares down more than 70 percent. Results from the second study are expected in 2018.
—Cambridge, MA-based Goldfinch Bio raised $55 million from Third Rock Ventures to build a patient database and develop drugs that address the genetic causes of chronic kidney disease.
VENTURE IN, VENTURE OUT
—Cambridge, MA-based Flagship Ventures unveiled a new name—Flagship Pioneering—and a $285 million follow-on fund that will invest in portfolio companies that need capital to rapidly grow.
—Summit, NJ-based Celgene (NASDAQ: CELG) paid Evotec $45 million up front in a deal to discover drugs for neurodegenerative diseases like Alzheimer’s and Parkinson’s.
—Former Google Ventures chief Bill Maris told Recode that his first post-Google project won’t be a $230 million healthcare fund, after all.
—Versant Ventures and Bayer pledged $225 million to a U.S.-Canadian startup, BlueRock Therapeutics, to develop stem cell therapies to repair heart damage and neurodegeneration.
—TCR² Therapeutics raised $44.5 million in a Series A round to develop technology that the company says could make the body’s immune cells even better cancer fighters.
—Apogen Biotech emerged from startup creator Accelerator with $7 million in funding to develop cancer therapies that are meant to overcome drug resistance. ApoGen is the third startup Accelerator has funded this year, following Petra Pharma and Lodo Therapeutics in New York.
Ben Fidler contributed to this report.