The future of ASN-100, the lead and only clinical candidate at Arsanis Inc., is in doubt after the Waltham, Mass.-based company halted the phase II test of the hemolysin alpha inhibitor for the prevention of Staphylococcus aureus (S. aureus) pneumonia in hospitalized, mechanically ventilated patients. A planned interim analysis of unblinded data by an independent data review committee (DRC) concluded the study was unlikely to meet its primary efficacy endpoint of preventing the virulent infection for up to 21 days in heavily colonized patients following a single intravenous administration, dosed at 20 mg.
The tone was solemn on Summit Therapeutics plc's conference call early Wednesday as CEO Glyn Edwards briefly restated the Oxford, U.K., firm's terse press release acknowledging failure of ezutromid to meet primary or secondary endpoints following 48 weeks of treatment in the phase II PhaseOut DMD study in individuals with Duchenne muscular dystrophy (DMD). The findings were an unexpected turnabout from interim data suggesting the small-molecule utrophin modulator seemed to help reduce muscle fiber damage and to increase levels of the protein utrophin, linked to the restoration and maintenance of healthy muscle function.
Agios Pharmaceuticals Inc. had the near-commercial asset and Cstone Pharmaceuticals Co. Ltd. had the market intel and rapidly maturing R&D engine. Together, the companies represent a formidable team to advance ivosidenib (Tibsovo, AG-120) in China, including Hong Kong, Macau and Taiwan, as monotherapy or in combination efforts. The oral isocitrate dehydrogenase 1 mutant, or IDH1m, inhibitor is under priority review by the FDA with an Aug. 21 PDUFA date for the treatment of individuals with relapsed or refractory acute myeloid leukemia (r/r AML).
Merrimack Pharmaceuticals Inc. is moving forward without MM-141 (istiratumab) after top-line data from the phase II CARRIE study in front-line metastatic pancreatic cancer showed that adding the bispecific, tetravalent monoclonal antibody (MAb) to standard-of-care (SOC) nab-paclitaxel/gemcitabine failed to beat the SOC regimen plus placebo in the primary efficacy endpoint of progression-free survival (PFS). The study also missed secondary efficacy endpoints: objective response rate, disease control rate, duration of response and overall survival.
Privately held Impel Neuropharma Inc. is looking to thrust its lead asset, INP-104, into the highly competitive migraine therapeutics mix as it embarks on the phase III trial of the drug-device candidate. A successful effort could enable an NDA filing by year-end using the 505(b)(2) pathway.
For the first time since BioWorld began tracking the biotech industry's public financings in 1999, five U.S. companies priced their IPOs within a 24-hour period and made their U.S. market debuts on a single day. Ironically, they picked June 21, the Northern Hemisphere's summer solstice. Whether the date was coincidental or a metaphor for long workdays ahead remains to be seen, but all five companies – Aptinyx Inc., Avrobio Inc., Kezar Life Sciences Inc., Magenta Therapeutics Inc. and Xeris Pharmaceuticals Inc. – started in the plus column. Eidos Therapeutics Inc., which priced a day earlier, began that trend when its shares (NASDAQ:EIDX) gained $6.12, or 36 percent, Wednesday to close its first trading day at $23.12.
Fresh off presentations this month at the American Society of Clinical Oncology in Chicago and the European Hematology Association in Stockholm that confirmed continued progress in its late-stage pipeline, TG Therapeutics Inc. struck a deal to collaborate on the development and commercialization of a preclinical anti-CD47/anti-CD19 bispecific antibody discovered by Novimmune SA. The companies will jointly advance the fully human IgG1, known as TG-1801 (previously NI-1701), on a global basis with a focus on hematologic B-cell malignancies.
The impetus to match individual patients with personalized therapies gained major momentum with the decision by Roche Holding AG to acquire the outstanding shares of Foundation Medicine Inc. (FMI) in a cash deal valued at $2.4 billion. The Basel, Switzerland-based pharma took a majority stake in FMI in a $1.05 billion deal disclosed on the opening day of the 2015 J.P. Morgan Healthcare Conference in San Francisco.
Results were preliminary and the database included just three children, but Sarepta Therapeutics Inc. scored a solid win with investors and analysts by exceeding expectations in its first assessment of AAVrh74.MHCK7.micro-dystrophin in individuals with Duchenne muscular dystrophy (DMD).
