Bio Roundup: Rare Disease Showdowns, Prices on TV, Dealmania & More

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the two, including five other Limb-Girdle therapies Sarepta acquired when it bought Nationwide spinout Myonexus Therapeutics. Xconomy recently reported on Nationwide’s rise to gene therapy prominence and its relationship with Sarepta.

—Boston Scientific (NYSE: BSX) reached a deal to acquire privately held Vertiflex for $465 million in cash. Vertiflex sells a minimally invasive device for patients who have a narrowing of the spinal canal called lumbar spinal stenosis.

—Germany-based cancer drug developer BioNTech acquired antibodies and infrastructure from San Diego-based MabVax Therapeutics for an undisclosed sum.

—Protagonist Therapeutics (NASDAQ: PTGX) and Janssen Biotech are expanding their partnership to develop peptide drug PTG-200. In addition to Crohn’s disease and ulcerative colitis indications, the firms will go after inflammatory bowel disease and more. Janssen will pay a $25 million milestone to Newark, CA-based Protagonist.

—Bluebird Bio (NASDAQ: BLUE) will work with the Seattle Children’s Research Institute to develop cell therapies for acute myeloid leukemia.

—Gilead Sciences (NASDAQ: GILD) committed $109 million to Goldfinch Bio in a multi-year collaboration to discover and develop new drugs to treat diabetic kidney disease and rare kidney disorders.

—Skyhawk Therapeutics cut a deal with Takeda to develop small molecule drugs for neurodegenerative diseases, adding to a similar pact with Celgene. Terms weren’t disclosed.

—Pfizer paid $340 million to buy privately-held Swiss biotech Therachon Holding, which is developing drugs for the rare diseases short bowel syndrome and achondroplasia, the most common form of short-limbed dwarfism.

—Antibody discovery shop Adimab expanded partnerships with Regeneron Pharmaceuticals and Novartis and inked a tech transfer deal with Takeda. Such tech transfer deals—including previous agreements with GlaxoSmithKline and Biogen—have long been part of the strategy of Adimab, which remains privately held and profitable.


—Longtime New York Times healthcare reporter Robert Pear died at the age of 69. The rival Washington Post tips its hat to Pear and reflects on his coverage of President Clinton’s attempt to pass healthcare reform.


—Cortexyme (NASDAQ:CRTX), Axcella Health (NASDAQ: AXLA), and Milestone Pharmaceuticals (NASDAQ: MIST) were among the clinical-stage biotechs that went public this week, the busiest for life science IPOs in recent memory.

—Verve Therapeutics emerged with $58.5 million in funding and a remit to develop gene-editing medicine to prevent heart attacks. Prominent cardiologist and geneticist Sekar Kathiresan will run the company.

Stat reported that Boston-based biotech investors Third Rock Ventures are working on a liquid biopsy startup called Thrive Sciences. The company includes several people once affiliated with Foundation Medicine, which is also in the cancer diagnosis business.

—Belgium’s Promethera BioSciences closed $44 million in Series D funding to push clinical tests of its stem cell treatment for liver disorders.

—The Multiple Myeloma Research Foundation’s $50 million investment fund made an equity investment in NexImmune to support development of its experimental treatment NEXI-002, which is approaching clinical trials.


—FibroGen (NASDAQ: FGEN) disclosed an analysis meant to show its anemia pill roxadustat is “non-inferior” to a placebo or standard-of-care erythropoiesis-stimulating agents in close to 9,000 tested patients with failing kidneys who are or aren’t on dialysis. FibroGen said the results were positive and plans to file for FDA approval of roxadustat later this year. But the data weren’t totally clear, which led to “confusion among investors about the communication,” wrote Leerink analyst Geoffrey Porges, and shares fell 18 percent.

—Cytokinetics (NASDAQ: CTYK) said its drug reldesemtiv failed to improve patients’ lung function in a 485-patient Phase 2 trial, FORTITUDE-ALS. But the company will press ahead with a larger Phase 3 study because it saw positive signs outside the trial design. Stat has more.

—A study published in the Annals of Internal Medicine examined instances of a rare, potentially fatal genital infection called Fournier gangrene occurring in patients taking the Type 2 diabetes drugs known as SGLT2 inhibitors.

—The AstraZeneca blood cancer drug acalbrutinib (Calquence) succeeded in the first of two Phase 3 studies in chronic lymphocytic leukemia. The drug, acquired when AstraZeneca took a majority stake in Acerta Pharma in 2015, is already approved for mantle cell lymphoma.

—The Bristol-Myers Squibb immunotherapy nivolumab (Opdivo) failed another Phase 3 study in brain cancer. Nivolumab failed to best bevacizumab (Avastin) in a brain cancer study two years ago.

—The first cannabis-derived medicine approved in the US, Epidiolex, hit its marks in a late-stage trial for a rare form of childhood epilepsy, its owner GW Pharmaceuticals (NASDAQ: GWPH) announced. The drug is already marketed for other types of epilepsy and exceeded early commercial expectations.

—In its quarterly earnings report, Moderna (NASDAQ: MRNA) said that partner Merck scrapped development of an experimental vaccine for shingles, mRNA-1278. Moderna and Merck also halted development of mRNA-1777, a vaccine for respiratory syncytial virus, in favor of a potentially more potent version.

—Partners AstraZeneca Daiichi Sankyo said they will ask this year for approval of breast cancer drug DS-8201 based on positive Phase 2 data. The antibody-drug conjugate is aimed at patients who have Her2-positive cancer and have already tried Roche’s trastuzumab emtansine (Kadcyla).


—Former Juno Therapeutics executive Howard Stern joined FogPharma as chief scientific officer.

Alex Lash and Frank Vinluan contributed to this report.

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