Zafgen evaluating strategic options
Metabolic company Zafgen Inc. (NASDAQ:ZFGN) has retained advisory firm MTS Health Partners to explore strategic alternatives after receiving new preclinical data that the company believes will be insufficient for FDA to lift a clinical hold on its second-generation methionine aminopeptidase 2 (MetAP2) inhibitor. FDA placed the hold in November on an IND for ZGN-1061 to treat Type II diabetes. Zafgen, which slipped 14% to $0.70on Thursday and has lost 98% of its value since October 2015, said it will seek to maximize shareholder value, potentially via a M&A transaction or licensing deal (see "Setback for Zafgen").
BridgeBio launches subsidiary for neuromuscular disease
BridgeBio Pharma Inc. (NASDAQ:BBIO) launched subsidiary ML Bio Solutions to develop BBP-418, a substrate supplementation therapy for limb-girdle muscular dystrophy type 2I. The compound originated from the McColl-Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health, formerly Carolinas HealthCare System. ML Bio Solutions aims to begin a natural history study this half. BridgeBio in-licenses preclinical and clinical assets from academia and pharma and houses them in individual subsidiaries.
Priority Review for Global Blood's first NDA
FDA granted Priority Review to an NDA from Global Blood Therapeutics Inc. (NASDAQ:GBT) seeking accelerated approval of voxelotor to treat sickle cell disease. Its PDUFA date is Feb. 26, 2020. The allosteric modifier of hemoglobin oxygen affinity has breakthrough therapy, Fast Track, Orphan Drug and rare pediatric disease designations in the U.S. FDA has notified Global Blood that it does not expect to hold an advisory committee meeting, the company said. Global Blood rose $5.17 (11%) to $52.66 on Thursday (see "GBT Heading for NDA").
Akouos licenses hearing loss gene therapy from Case Western
Akouos Inc. (Boston, Mass) exclusively licensed development and commercialization rights from Case Western Reserve University for a gene therapy to treat hearing loss associated with hereditary deaf-blind disorder Usher syndrome type 3A. Akouos is developing adeno-associated virus (AAV)-based gene therapies to treat sensorineural hearing loss resulting from mutations in individual genes. It raised $50 million in a series A round last year (see "Sound Story").
Mallinckrodt sinks after report suggests bankruptcy is an option
Mallinckrodt plc (NASDAQ:MNK) did not respond to inquiries Thursday after a Bloomberg report said the company has retained restructuring advisers, including turnaround firm AlixPartners, as it mulls options ahead of a federal trial concerning its sale and marketing of opioids. The report, which cited undisclosed sources, said Mallinckrodt could file for bankruptcy to limit its liabilities. The company's shares sank 39% to $1.59 on Thursday, leaving its shares off 95% from their 52-week high.
uBiome files for bankruptcy
Microbiome company uBiome Inc. (San Francisco, Calif.) filed for Chapter 11 bankruptcy Wednesday. Shareholders with a 10% or greater stake are 8VC Fund and Andreessen Horowitz. uBiome raised $83 million in a series C round last year (see "A Bigger 'Biome").