onasemnogene abeparvovec (Zolgensma), the gene therapy now owned by Novartis (NYSE: [[ticker:NVS]]) for spinal muscular atrophy. The agency is giving the treatment a “priority” review, quicker than normal, and should make a decision by May.
—As several companies race to develop the first treatment for NASH, the FDA published much-anticipated guidelines for clinical trial design.
—Germany’s Bayer was forced to stop testing the drug vilaprisan, an experimental treatment for uterine fibroids, after signals of toxic side effects in long-term rodent studies.
—The FDA approved an Ocular Therapeutix (NASDAQ: [[ticker:OCUL]]) treatment for pain following eye surgery. The approval of Dextenza follows the FDA’s 2017 rejection of the Ocular product due to manufacturing issues.
—Alnylam Pharmaceuticals (NASDAQ: [[ticker:ALNY]]) filed for approval of its RNA interference drug for transthyretin amyloidosis, patisiran (Onpattro), in Canada and Switzerland, and began submitting paperwork to the FDA for its second RNAi drug, givosiran, for acute hepatic porphyrias.
—An experimental drug from Vanda Pharmaceuticals (NASDAQ: [[ticker:VNDA]]), tradipitant, succeeded in a Phase 2 study in diabetic gastroparesis. Shares climbed 18 percent.
—Roche said its drug-antibody combination Kadcyla bested its antibody predecessor trastuzumab (Herceptin) in a phase 3 study for patients who have undergone surgery for Her2-positive breast cancer.
Cash Grabs & New Starts
—Johnson & Johnson paid Dutch firm Argenx $300 million up front and made a $200 million equity investment in the Dutch firm for rights to cusatuzumab, a drug being tested in patients with myelodysplastic syndrome and acute myeloid leukemia.
—Synthorx (NASDAQ: [[ticker:THOR]]), of San Diego, CA, raised $131 million in an IPO to develop drugs for cancer and autoimmune diseases.
—China-focused Brii Biosciences added a hepatitis B drug to its pipeline through a deal with VBI Vaccines, and the startup also announced plans to launch an R&D site in Beijing.
—Following a string of setbacks to its cancer drugs, OncoMed Pharmaceuticals (NASDAQ: [[ticker:OMED]]) of Redwood City, CA, agreed to combine with Mereo BioPharma in a reverse merger. In addition to gaining a U.S. stock listing, the deal gives London-based Mereo OncoMed’s three early-stage cancer drugs.
—Biogen (NASDAQ: [[ticker:BIIB]]) grabbed rights to an RNA-based drug for amyotrophic lateral sclerosis from Ionis Pharmaceuticals (NASDAQ: [[ticker:IONS]]) following encouraging Phase 1 results. Biogen had an option to the drug as part of the sprawling $1 billion partnership between the two companies two develop RNA drugs for neurological diseases.
—New York City-based Kallyope added $21 million to its Series B round, an investment led by Bill Gates that brings the total financing to $87 million. Kallyope is developing small molecule drugs that target the gut brain axis, a signaling network between cells in the gut and the central nervous system.
—Buffalo, NY-based Tactiva Therapeutics raised a $35 million Series A led by Panacea Ventures to develop T cell therapies for multiple myeloma and solid tumors.
—Nestle Health Science opened its new Bridgewater, NJ-based global research hub, the Nestle Product Technology Center, which will staff more than 170 employees, and develop consumer and nutrition products.
Burrill Sentenced
—G. Steven Burrill, a once noted biotech investment fund manager, was sentenced to 30 months in federal prison for defrauding investors and evading taxes.
Frank Vinluan and Alex Lash contributed to this report.
