Abbvie Inc. trumped Allergan plc in the effort to treat women with abnormal bleeding due to uterine fibroids. Abbvie, of North Chicago, reported top-line data from the phase III ELARIS UF-EXTEND extension study, which showed that, at 12 months, elagolix (300 mg twice daily), in combination with low-dose hormone (add-back) therapy (estradiol 1.0 mg/norethindrone acetate 0.5 mg), reduced heavy menstrual bleeding, with 87.9 percent of women with uterine fibroids achieving clinical response.
Two-year-old Drugcendr Inc. became the latest biopharma to take a stab at pancreatic cancer by advancing lead compound, CEND-1, into a phase I trial in individuals with metastatic pancreatic ductal adenocarcinoma (PDAC) undergoing combination therapy with Abraxane (nab-paclitaxel, Celgene Corp.) and gemcitabine.
Hours apart, two biotechs with big pharma connections – Principia Biopharma Inc. and Entasis Therapeutics Holdings Inc. – each filed for IPOs of up to $86.25 million, including overallotments, and listings on the Nasdaq Global Market as PRNB and ETTX, respectively.
Fasinumab, the nerve growth factor (NGF) receptor antagonist that Regeneron Pharmaceuticals Inc. is advancing with partner Teva Pharmaceutical Industries Ltd., passed its efficacy test for the treatment of chronic pain from osteoarthritis (OA) of the knee and hip, based on findings from a substudy of a larger, long-term phase III study. However, two higher-dose regimens – 3 mg every four weeks and 6 mg every eight weeks – were discontinued earlier this year at the behest of an independent data monitoring committee. And the waiting continues for top-line safety data.
Ethan Perlstein acknowledged up front that Perlara, "as the first biotech PBC," is "a different type" of C corporation but not – as some have surmised – a not-for-profit entity. That said, Perlstein – who founded and serves as CEO of Perlara, previously Perlstein Lab, following his postdoctoral fellowship at Princeton University's Lewis-Sigler Institute – wanted the 2014 startup to be "180 degrees different in every regard" from the typical biopharma, from its scientific approach to its branding and business model. The PBC, or public benefit corporation, provided a structure to define social and environmental missions that were aligned with venture philanthropy and social entrepreneurship and to create a footprint for drug discovery that regarded patients as true partners rather than volunteers for clinical trials.
Realm Therapeutics plc led its second-quarter earnings with a dud: Top-line data from its randomized, double-blind phase II study in atopic dermatitis showed that PR-022 performed no different from placebo, compared to baseline, on the primary endpoint of percent change in Eczema Area Severity Index, or EASI, at day 29. The company did not disclose details. CEO Alex Martin commented only that a full review of the data was underway "to determine whether there is a path forward for our proprietary technology in atopic dermatitis and to evaluate the implications for our acne and psoriasis programs."
The team responsible for leveraging a preclinical nonalcoholic steatohepatitis (NASH) asset into the acquisition of developer Akarna Therapeutics Ltd. by Allergan plc now is spearheading a psoriasis program at Escalier Biosciences BV, where lead program ESR-114, a topical gel, has moved into a phase I/IIa study. ESR-114 targets the retinoid acid receptor gamma t (RORyt), which Escalier co-founder and CEO Raju Mohan described as the master regulator of Th17 cell differentiation and the production of pro-inflammatory cytokines, including IL-17A and IL-17F, which are linked to the pathophysiology of psoriasis.
FDA approval of patisiran, branded Onpattro, to treat hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) represents several firsts for Alnylam Pharmaceuticals Inc., not least of which is the distinction of bringing the initial RNAI interference (RNAi) drug to market. Although the Cambridge, Mass.-based company also beat competitor Akcea Therapeutics Inc., which is developing Tegsedi (inotersen), to first approval in the hATTR indication, Alnylam now faces the challenge of wooing physicians and patients to a drug in a class without precedent.
Details were scant, and Edward Conner, senior vice president and chief medical officer at Sangamo Therapeutics Inc., cautioned that "it's obviously still early days," but the Richmond, Calif.-based company clearly is encouraged by early data from the phase I/II ALTA trial evaluating SB-525, its cDNA gene therapy candidate to treat hemophilia A.