Ultragenyx Pharmaceutical Inc.’s top-line win in January with DTX-301 gene therapy in ornithine transcarbamylase (OTC) deficiency seemed to presage even better things to come later this year, and analysts more recently hailed fourth-quarter earnings that showed satisfying progress with Crysvita (burosumab).
Revolution Medicines Inc. (NASDAQ:RVMD) shares closed at $28.90, a 70% jump above the $17 price in its upsized IPO of 14 million shares, which raised $238 million, showing further confidence in the Redwood City, Calif.-based company’s bid to blast cancer targets once deemed “undruggable.”
Alx Oncology Inc. pulled down a $105 million series C equity financing to support the expansion into phase II trials with ALX-148, described as a next-generation CD47 myeloid checkpoint inhibitor, paired with other cancer therapeutics. ALX-148 uses a “dead” Fc domain that does not bind to macrophages, thus reducing cytopenia and other toxicities associated with the class.
A half-day open meeting intended to examine “how the public perceives and values pharmaceutical quality,” convened by the Robert J. Margolis Center for Health Policy at Duke University in cooperation with the FDA, included a rundown of the agency’s oversight program, results of surveys to measure viewpoints of patients and providers – and tart commentary from a two-member “reactant panel.”
With the Simpliciti-T1 phase II trial testing new-mechanism TTP-399 as adjunct therapy in type 1 diabetes (T1D), High Point, N.C.-based Vtv Therapeutics Inc. nailed the HbA1c endpoint with none of the safety concerns foiling developers of type 2 diabetes (T2D) drugs who sought to broaden their labels.
The less-frequent dosing regimen of Basel, Switzerland-based Novartis AG’s cholesterol therapy, inclisiran, under development in the hands of subsidiary The Medicines Co., positions the small interfering RNA (siRNA) drug to take on marketed proprotein convertase subtilisin/kexin type 9 (PCSK9)-targeting antibodies as well as statins in the busy therapeutic space. Statins are the gold standard now, but about 80% of patients don’t reach their lipid goals.
Emeryville, Calif.-based Zogenix Inc.’s positive top-line data from the phase III study with Fintepla (fenfluramine oral solution) in Lennox-Gastaut syndrome (LGS) failed to charm Wall Street, which by day’s end trimmed the shares (NASDAQ:ZGNX) by $20.50, or 39%, putting the final price at $32.12.
Lineage Cell Therapeutics Inc. CEO Brian Culley told BioWorld that concerns about the pace of the company’s dry age-related macular degeneration (AMD) phase I/IIa trial have been alleviated after an independent data safety monitoring board (DSMB) decreed that the study’s protocol-mandated treatment stagger can be removed.
A half-day open meeting intended to examine “how the public perceives and values pharmaceutical quality,” convened by the Robert J. Margolis Center for Health Policy at Duke University in cooperation with the FDA, included a rundown of the agency’s oversight program, results of surveys to measure viewpoints of patients and providers – and tart commentary from a two-member “reactant panel.”
Shares of Bridgewater, N.J.-based Insmed Inc. (NASDAQ:INSM) closed at $28.88, up $8.34, or 40.6%, on positive top-line results from the global, randomized, double-blind placebo-controlled phase II study called Willow, testing INS-1007 once daily in adults with non-cystic fibrosis bronchiectasis (NCFBE).
