Akari Therapeutics plc's interim CEO and chief operating officer, Clive Richardson, told BioWorld that the company plans to enroll more seriously afflicted bullous pemphigoid (BP) patients in the phase II trial that has yielded positive results in three subjects treated with the firm's once-daily subcutaneous therapy, nomacopan (previously known as coversin), based on a protein originally discovered in the saliva of the Ornithodoros moubata tick. "Hopefully on the back of that expanded trial we would go to the FDA early next year and agree with them [on] what would be the pathway to a pivotal study," he said.
A rivalry is shaping up between heavyweight Amgen Inc. and smaller contender Mirati Therapeutics Inc. in the targeting of KRAS G12C, a protein that researchers once believed altogether intractable because it is lacking in hydrophobic pockets and binding grooves.
Word hardly shook the world in early April when Cambridge, Mass.-based Acceleron Pharma Inc. dropped ACE-2494, a systemic muscle agent in phase I tests in healthy volunteers, because anti-drug antibodies (ADAs) developed. Although the compound had shown signs of early target engagement and ADAs had not been associated with any adverse event, Acceleron said the candidate wasn't worth going ahead with.
Although analysts urged a more moderate stance, investors sheared Wave Life Sciences Ltd.'s stock, apparently displeased with final results from the phase I trial with suvodirsen, or WVE-210201, in Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
Arrowhead Pharmaceuticals Inc.'s plan to start an adaptive phase II/III trial in the second quarter of this year with ARO-AAT brought more attention to the busy therapeutic space around alpha-1 antitrypsin deficiency (AATD).
Johnson & Johnson (J&J) unit Janssen Pharmaceutical's accelerated approval Friday from the FDA for Balversa (erdafitinib) to treat adults with locally advanced or metastatic urothelial carcinoma (mUC) with the susceptible genetic alteration fibroblast growth factor (FGFR)3 or FGFR2 brought new attention to FGFR drug candidates, hardly in short supply. Once-daily, oral Balversa is the first FGFR kinase inhibitor to gain U.S. regulators' blessing.
In the second week of April, Plymouth Meeting, Pa.-based Inovio Pharmaceuticals Inc. nailed a third indication milestone in its deal with Astrazeneca plc, of London, as a result of dosing the first patient in a phase II combination trial testing MEDI-0457 (formerly called INO-3112) in combination with durvalumab, an anti-PD-L1 immune checkpoint inhibitor, to target cervical, anal, penile and vulvar cancers associated with human papillomavirus (HPV).
Zogenix Inc. CEO Stephen Farr said the refusal to file (RTF) letter from left field for Fintepla (ZX-008, fenfluramine hydrochloride) for seizures associated with Dravet syndrome (DS) could set the compound back "12 to 15 months, would be our best guess" if the FDA insists that the Emeryville, Calif.-based firm do further nonclinical toxicology studies. "We had constructive interactions with the FDA on our program, which started in 2015," Fuller said during a conference call with investors. "This requirement is definitely unexpected for us."
The scientific teams of Regeneron Pharmaceuticals Inc. and Alnylam Pharmaceuticals Inc. have "enough work to occupy us for the next 10 years comfortably," thanks to known genetically validated targets, according to the latter's R&D chief, Akshay Vaishnaw. "Having said that, new targets will continue to emerge."
