If you’re keeping track, FDA drug approvals have been on a tear in the past month with several decisions coming well ahead of their targeted dates. And then there’s Sarepta Therapeutics.
Late Thursday, the FDA announced approval of the Cambridge, MA, biotech’s drug for Duchenne muscular dystrophy in patients who have a specific mutation. The drug, golodirsen (Vyondys 53), is the same one the agency rejected four months ago due to safety concerns. Responding to a rejection and addressing FDA concerns can take up to one year but Sarepta (NASDAQ: [[ticker:SRPT]]) says the issues were “rapidly evaluated and resolved.” If things are moving more quickly at the FDA, it will be interesting to see whether the new FDA commissioner, who was confirmed this week, continues that pace.
In other news this week, new venture funds raised cash, big pharma announced big immunotherapy deals, and drug price legislation advanced in Congress. Let’s get to those stories and more in this week’s roundup.
LET’S MAKE A DEAL
—Merck (NYSE: [[ticker:MRK]]) maneuvered to expand its cancer immunotherapy portfolio with a $2.7 billion deal to acquire ArQule (NASDAQ: [[ticker:ARQL]]), whose lead drug is in early-stage testing in blood cancers.
—France’s Sanofi (NYSE: [[ticker:SMY]]) also moved to bolster its immunotherapy unit, agreeing to acquire San Diego’s Synthorx (NASDAQ: [[ticker:THOR]]) and its pipeline of cancer drugs that leverage an “expanded” genetic alphabet to reduce toxicities for $2.5 billion.
— AbbVie (NYSE: [[ticker:ABBV]]) is doubling down on a research collaboration with The Scripps Research Institute, expanding a partnership established last year around drug discovery and development in oncology to other areas, including immunology, neurology, and fibrosis.
—Gatehouse Bio has teamed up with AstraZeneca (NYSE: AZN) to identify potential drug targets in respiratory and cardiovascular diseases.
—3M (NYSE: [[ticker:MMM]]) is selling its drug delivery division to investment firm Altaris Capital Partners for $650 million. Excluded from the deal is 3M’s transdermal drug delivery components business.
NEW CASH, FRESH STARTS
—Perceptive Advisors launched a $210 million venture capital fund focused on investing in early-stage life science companies.
—Omega Funds closed its sixth fund with $438 million in capital commitments that the investment firm plans to use to invest in “the most urgent medical needs.”
—Zentalis Pharmaceuticals emerged from stealth this week, revealing a total of $147 million raised and big plans for small molecule cancer drugs.
—Flagship Pioneering spun out another new biotech, Cellarity, which aims to develop drugs by deploying machine-learning guided know-how of “network biology,” or the behavior of how cells act and interact.
PIPELINE PROGRESS
—Sarepta Therapeutics (NASDAQ: [[ticker:SRPT]]) won FDA approval for golodirsen (Vyondys 53), a treatment for Duchenne muscular dystrophy patients who have a specific mutation found in an estimated 8 percent of patients who have the inherited disease.
—Aurinia Pharmaceuticals (NASDAQ: [[ticker:AUPH]]) plans an FDA submission after its experimental lupus nephritis drug met the goals of a Phase 3 study.
—Colorado biotech miRagen Therapeutics (NASDAQ: [[ticker:MGEN]]) is shifting resources to a preclinical compound for a form of fibrosis affecting the lungs, part of a restructuring that will cut 18 jobs.
—BlackThorn Therapeutics plans to advance its lead drug to Phase 2 testing after reporting data showing that the compound engages receptors in the brain at levels thought to be effective in treating depression and other central nervous system disorders.
—Digital therapeutics developer Akili Interactive reported that its experimental depression treatment, AKL-T03, showed a statistically significant improvement in sustaining a patient’s attention, which is an FDA cleared endpoint.
—Paul Aisen, head of the University of Southern California’s Alzheimer’s Therapeutic Research Institute, argued at the Clinical Trials on Alzheimer’s Disease conference that the once-bagged, now-revived Biogen (NASDAQ: [[ticker:BIIB]]) drug aducanumab has irrevocably changed Alzheimer’s disease drug discovery and development.
—The FDA lifted a clinical hold on tests of an experimental Abeona Therapeutics (NASDAQ: [[ticker:ABEO]]) treatment for a form of epidermolysis bullosa, an inherited connective tissue disorder affecting the skin. New York-based Abeona plans to start the Phase 3 study in the first quarter of 2020.
TRIALS AND TRIBULATIONS
—Safety concerns led the FDA to place partial holds on clinical trials for palovarotene, a drug in development to treat rare connective tissue disorders. Ipsen acquired the compound through its $1 billion acquisition of Clementia Pharmaceuticals earlier this year.
—Iterum Therapeutics (NASDAQ: [[ticker:ITRM]]) reported that its experimental antibiotic, sulopenem, fell just shy of the main goal of a Phase 3 study in intra-abdominal infections. The biotech hopes for better results from an ongoing late-stage study in urinary tract infections, but investors still drove shares down more than 40 percent.
—An experimental drug that KalVista Pharmaceuticals (NASDAQ: [[ticker:KALV]]) is developing in partnership with Merck failed a mid-stage study for diabetic macular edema. The drug is also being tested in Phase 2 for the rare disorder hereditary angioedema.
—This week, 23andMe was slapped with a $100 million lawsuit by Celmatix, a fertility research startup and its former research partner. Celmatix claims 23andMe breached a contract and failed to perform its obligations. It also claims the genetic testing company blocked Celmatix’s ability to raise capital.
—The FDA slapped Alkermes (NASDAQ: [[ticker:ALKS]]) with a warning letter for its extended-release opioid dependence drug naltrexone (Vivitrol), saying a print ad omits information, including the risk of a fatal overdose.
—Gosuranemab, an experimental Biogen drug for the brain disorder progressive suprnuclear palsy, failed a Phase 2 study. Biogen licensed the compound from Bristol-Myers Squibb (NYSE: [[ticker:BMY]]) in 2017 for $300 million up front.
FROM THE BELTWAY
—The FDA now has a new commissioner. The Senate confirmed Stephen Hahn by a 72-18 vote.
—The USMCA trade agreement signed this week dropped a provision granting 10 years of marketing exclusivity for biological drugs. Pharmaceutical industry lobbyists argued that the protections are necessary to allow companies to recoup their investments so they can keep developing innovative new medicines.
—The US House of Representatives passed a wide-ranging prescription drugs bill that would allow the federal government to negotiate with pharmaceutical companies, a part of the legislation intended to reduce drug prices. Industry groups claim the legislation will stifle pharmaceutical innovation.
—One day after an FDA advisory panel voted 11-2 against recommending approval of a Correvio Pharma (NASDAQ: [[ticker:CORV]]) atrial fibrillation drug, the Vancouver, BC-based company said it would explore a possible sale of the business.
PEOPLE ON THE MOVE
—BridgeBio Pharma (NASDAQ: [[ticker:BBIO]]) appointed Eli Wallace its chief scientific officer in residence for oncology… Serafim Batazoglu joined Insitro as chief data officer… Flexion Therapeutics (NASDAQ: [[ticker:FLXN]]) co-founder and Chief Scientific Officer Neil Bodick will retire in January… Jean Franchi joined Replimune Group (NASDAQ: [[ticker:REPL]]) as chief financial officer… Prometheus Bio appointed Thierry Dervieux as its chief development officer of diagnostics and medical laboratory director… and former Casebia Therapeutics executive Jim Burns was appointed CEO of Locana.
Sarah de Crescenzo and Melissa Fassbender contributed to this report.
Photo by Flickr user daveynin via a Creative Commons license
