Last week at the Goldman Sachs Healthcare Conference in Rancho Palos Verdes, Calif., Sarepta Therapeutics Inc. CEO Douglas Ingram noted that "more than a kid a day dies" from Duchenne muscular dystrophy (DMD) in the U.S., adding that officials at his Cambridge, Mass.-based firm "feel an enormous obligation to move heaven and earth to get this [gene] therapy to the community if it's possible" and quickly.

Pfizer Inc. wasn't shy about talking specifics with regard to synergy in its merger with Array Biopharma Inc., for which the pharma giant has agreed to pay $48 per share in cash – a 62% premium to Friday's closing price of $29.59 – in a deal with an enterprise value of about $11.4 billion. The boards of both companies have approved the merger, expected to finish in the second half of this year.

Kura Oncology Inc. CEO Troy Wilson told BioWorld that, based on powerful interim data with tipifarnib (often called "tipi" by researchers) in relapsed or refractory peripheral T-cell lymphoma (PTCL), to amend the ongoing phase II study – which has been rejiggered once already – into a registrational experiment is "a possibility. I think that would not be our preference, but you could do that."

Prelude Therapeutics Inc. founder and CEO Kris Vaddi said the new $60 million in series B money – which brings to $95 million the amount raised since the firm's inception in 2016 – should pull the current phase I research through proof-of-concept stage, targeting protein arginine methyltransferase 5 (PRMT5) in cancer.

Cymabay Therapeutics Inc.'s chief scientific officer, Charles McWherter, told BioWorld that the concept of liver enzyme scores as a powerful indicator of efficacy vs. liver fat reductions in nonalcoholic steatohepatitis (NASH) "is not an entirely made-up idea, just because we got this result" in the phase IIb study of peroxisome proliferator-activated receptor (PPAR) delta agonist seladelpar. "If you had to choose between reducing fat, which can be harmful or can be inert, or showing evidence of improving the underlying lesions and injury" as a way of showing a drug's worth in NASH, he said, researchers might choose the latter as "more proximal to the injury," McWherter said. As Newark, Calif.-based Cymabay's chief medical officer, Pol Boudes, put it, "the fat story is not the end of the story" in NASH.

Tilos Therapeutics Inc. CEO Barbara Fox told BioWorld that the relationship with Merck & Co. Inc. "went pretty deep pretty fast," culminating in the pharma giant's decision to take over her firm for as much as $773 million. "We never intended to do this," she said. "We always assumed we were going to raise a series A, take these [candidates] forward independently, get some clinical proof of concept and look for partnerships later."

Xeris Pharmaceuticals Inc. CEO Paul Edick said a fourth-quarter launch of Gvoke (ready-to-use glucagon injection) for severe hypoglycemia is "still doable. Is it now a tad more of a stretch? Yes." His remarks came as investors learned that the PDUFA date for the injector pen has been pushed back by three months, putting the new decision time for the severe hypoglycemia treatment at Sept. 10, 2019.

CHICAGO – Though it's not optimal in some other cancer types (such as pancreatic and cervical), the minimally invasive method of laparoscopic surgery in colorectal cancer (CRC) with liver metastases vs. the more dramatic "open" surgery leads to the same rate of survival. That's according to findings unwrapped at the American Society of Clinical Oncology (ASCO) annual meeting, where attendees heard results from the randomized study called Oslo-Comet determining that patients lived more than 6.5 years after surgery, regardless of whether it was laparoscopic or open.