CEO Maurits Geerlings told BioWorld Today that Formula Pharmaceuticals Inc. may have found a way around the drawbacks of chimeric antigen receptor (CAR) methods that deploy autologous T cells, thanks to the worldwide exclusive license the firm has gained to an allogeneic, nonviral CAR platform that leverages instead cytokine-induced killer (CIK) cells as immune effectors.

Briefing documents for Tuesday's meeting of the FDA's Pulmonary-Allergy Drugs Advisory Committee (PADAC) showed reviewers are unconvinced about Vertex Pharmaceuticals Inc.'s Orkambi, which consists of the firm's already approved Kalydeco (ivacaftor) and lumacaftor combined for homozygous F508del cystic fibrosis (CF) patients.

Spyryx Biosciences Inc. is "quickly going from virtual to nonvirtual" with $18 million of series A money in hand, CEO John Taylor told BioWorld Today, and aims to move a therapeutic respiratory compound into the clinic as soon as possible.

In rare conditions, "it's very difficult to see an opportunity when there's such a lack of awareness for the disease," CEO David Hallal said, but Alexion Pharmaceutical Inc.'s established expertise lets the firm "evaluate all the medical literature and connect the dots" in order to find a market worth growing.

The Medicines Co. may have prospectively eased the pain of generic competition that looms for flagship product Angiomax (bivalirudin) – and may have helped stanch the money hemorrhage that would result – by winning FDA approval of two drug-device combo therapies: one for patients who need opioid analgesia in the hospital and another to control bleeding.

"Casting a wide net" with its phase I trial testing MRX34, Mirna Therapeutics Inc. raked in $41.8 million by way of a series D financing that will enable a phase II experiment in the first half of next year, in a cancer indication yet to be decided upon, CEO Paul Lammers told BioWorld Today.

Resverlogix Corp. CEO Donald McCaffrey told BioWorld Asia he is "very confident" about collecting the potential $400 million-plus from a licensing deal in China, Hong Kong, Taiwan and Macau with Shenzhen Hepalink Pharmaceutical Co. Ltd., centered on RVX-208, a late-stage, first-in-class, small-molecule BET bromodomain inhibitor. "We downplayed the number," he said.

Having banked $38 million in a series A round late last year, cancer immunotherapy firm Potenza Therapeutics Inc. is "in good shape in terms of financing," thanks also to an undisclosed amount of new money from a deal with Astellas Pharma Inc., CEO Daniel Hicklin told BioWorld Asia.