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."
During the American Association for Cancer Research (AACR) meeting in Atlanta recently, the FDA's Oncology Center of Excellence director, Richard Pazdur, called for more clinical teamwork among the makers of PD-1/PD-L1 inhibitors, and he's getting his wish – not from them but from Odonate Therapeutics Inc., developer of the oral taxane tesetaxel.
With the recent granting of the IND for trials to begin with CG-806 in the hands of Aptose Biosciences Inc., the rich and busy field of Bruton's tyrosine kinase (BTK) inhibitors may be set to further evolve.
Less than a week after starting the phase I trial with its lead NLRP3 antagonist, IFM-2427, Boston-based IFM Therapeutics LLC chalked up a contract potentially worth more than $1.5 billion for anti-inflammatory therapies with Novartis AG, which brings aboard the whole portfolio in the class via the buyout of subsidiary IFM Tre.
The FDA ended last week and started this one with drug approvals, clearing Darmstadt, Germany-based Merck KGaA's Mavenclad (cladribine) tablets for relapsing multiple sclerosis (MS) in adults – with a label that includes relapsing-remitting disease as well as active secondary progressive MS (SPMS) – and followed with marketing clearance for Duaklir, a fixed-dose inhaled combination from Circassia Pharmaceuticals Inc., of Oxford, U.K., that pairs long-acting muscarinic antagonist (LAMA) aclidinium bromide with long-acting beta agonist (LABA) formoterol fumarate for chronic obstructive pulmonary disease (COPD) maintenance.
"We got it wrong," mourned Maxim analyst Jason McCarthy after Waltham, Mass.-based Proteon Therapeutics Inc. disclosed results from the phase III trial called Patency-2, a 603-patient experiment with vonapanitase, made up of recombinant human elastase, in patients with chronic kidney disease (CKD) undergoing creation of radiocephalic (wrist) fistulas for vascular access in hemodialysis.
