The FDA cleared oral pretomanid, developed by the non-profit TB Alliance, under the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD) as part of a three-drug, six-month, all-oral regimen for the treatment of tuberculosis (TB) in people with extensively drug-resistant disease (XDR-TB) or multidrug-resistant TB (MDR-TB) who are treatment-intolerant or non-responsive. Read More
Phase III results for evinacumab from Regeneron Pharmaceuticals Inc. released Wednesday show a 49% LDL cholesterol reduction in patients with homozygous familial hypercholesterolemia (HoFH) when compared to lipid-reducing therapies alone. Read More
During its recent conference call on earnings, Fate Therapeutics Inc. gave out what one analyst called "a wake-up call for the entire industry" with regard to cost of goods, just as more are starting to awaken regarding the company's potential therapeutically. Read More
A little over a month after its debut as a public company, Seattle-based Adaptive Biotechnologies Inc. reported second-quarter results. The company saw revenues of $22.1 million, which topped William Blair estimates, as well as the consensus of $19.4 million. Read More
Nearly a decade in the making, Philadelphia-based Renovacor Inc. set sail with its first institutional raise. The company closed an $11 million series A co-led by Novartis Venture Fund, Broadview Ventures and Bioadvance and joined by New Leaf Venture Partners and Italy-based Innogest Capital. Read More
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Eli Lilly and Co., of Indianapolis, and Sanford Burham Prebys Medical Discovery Institute in La Jolla, Calif., disclosed a three-year extension to their existing collaboration to discover and develop immunological therapies. The goals of the agreement are to advance ongoing projects, including a phase I trial of an immune therapy that arose from the research collaboration, and to expand the previous scope to include omics-based target identification studies. Researchers will use technologies, including CRISPR, to discover novel drug targets to treat autoimmune disorders. Read More
Strongbridge Biopharma plc, of Trevose, Pa., appointed Richard S. Kollender chief operating officer, Robert Lutz chief financial officer and David Gill to its board, all effective Sept. 3. Read More
The FDA finalized its 2016 guidance discussing the nonclinical evaluation of drugs intended to treat osteoporosis. The guidance provides recommendations on the design of bone quality studies in appropriate animal models that are needed to support the approval of osteoporosis drugs and biologics. It also discusses bone-specific pharmacologic and toxicologic endpoints to evaluate the effects of a drug on bone quality in the nonclinical testing. The final guidance modifies the recommended number of dose levels to be evaluated in the bone quality studies and adds a recommendation to use a multifactorial approach to determine the treatment duration in the studies, according to a notice to be published in Thursday's Federal Register. Read More
Portola Pharmaceuticals Inc., of South San Francisco, priced its underwritten public offering of about 8 million shares of common stock at a price to the public of $28 per share. Underwriters of the offering have been granted a 30-day option to purchase up to about 1.2 million more shares. The deal is expected to close Friday. Goldman Sachs & Co. LLC, Citigroup, Cowen, and William Blair are acting as joint book-running managers. Oppenheimer & Co. Inc. is serving as lead manager. Read More
Rare disease specialist Ultragenyx Pharmaceutical Inc. has agreed to pay $20 million for an exclusive option to acquire Genetx Biotherapeutics LLC, the developer of a University of Texas-sourced antisense oligonucleotide for the potential treatment of Angelman syndrome, GTX-102. An IND for the asset, now in late preclinical development, is expected to be filed with the FDA in the first half of 2020. Read More
