Shares of Mirati Therapeutics Inc. (NASDAQ:MRTX) doubled at their opening Friday morning and spiked to a 15-month high of $12.40 on word that the combination of the company's tyrosine kinase inhibitor (TKI), sitravatinib (MGCD-516), with Opdivo (nivolumab, Bristol-Myers Squibb Co.) showed three confirmed partial responses (PRs) in the first 11 evaluable patients with non-small-cell lung cancer (NSCLC) who had progressed following checkpoint therapy.

Amicus Therapeutics Inc. minced no words on the failure of its phase III study of SD-101 in epidermolysis bullosa (EB), the object of a potential $847 million take-out of Scioderm Inc., but John Crowley, Amicus chairman and CEO, was equally quick to defend the merits of the acquisition on a conference call Wednesday.

Sage Therapeutics Inc. sought to emphasize the lessons learned and increased understanding from the top-line readout of its first phase III trial, which assessed brexanolone (SAGE-547) in patients with super-refractory status epilepticus, or SRSE. The highly anticipated trial missed its primary endpoint of comparing the candidate's success in weaning of third-line agents and resolution of potentially life-threatening status epilepticus vs. placebo (43.9 percent vs. 42.4 percent; p=0.8775) when added to standard of care.

Alnylam Pharmaceuticals Inc. suspended dosing in ongoing studies of fitusiran following the report of a fatal thrombotic event in a patient with hemophilia A without inhibitors that occurred during the therapy's phase II open-label extension (OLE) study. In July, the antithrombin-targeting RNAi therapy developed by Alnylam with partner Sanofi Genzyme, entered phase III territory with the launch of the three-part ATLAS program in hemophilia A and B. (See BioWorld, July 10, 2017.)

Chatter about a potential new drug application (NDA) filing for golodirsen (SRP-4053) started as soon as Sarepta Therapeutics Inc. reported that the phase I/II study (4053-101) of the exon 53 skipping candidate achieved statistical significance on its primary and secondary biological endpoints in boys with confirmed deletions of the gene, one of several associated with Duchenne muscular dystrophy (DMD).

Deciphera Pharmaceuticals LLC and Nucana Ltd. became the latest entrants into the 2017 IPO queue, filing with the SEC to raise up to $100 million and $115 million, respectively.

In the wake of Kymriah's approval, the ability to ensure a sustainable manufacturing and supply chain emerged as a major differentiator among CAR T contenders. With deep pockets, Novartis AG leveraged its global R&D and manufacturing expertise "to take a cutting-edge, complicated individualized process from the first three patients treated at Penn and expand it to a clinical trial program across 25 centers and four continents, forming the basis for the regulatory filing in the U.S. and, soon, Europe," explained Bill Hinshaw, general manager of U.S. Oncology for the Basel, Switzerland-based company.

As the dust settled following the FDA’s precedent-setting approval of Kymriah (tisagenlecleucel, previously CTL-019), the chimeric antigen receptor T-cell (CAR T) immunotherapy developed by Novartis AG, analysts and observers began to parse the long-term impact to the players and broader implications for the field.