An intriguing "excellent outcome" in certain patients plus Multistem's clean safety profile may let Athersys Inc. and Japan partner Chugai Pharmaceutical Co. Ltd. refine the targeted patient population for better results in future trials, but for now the cell therapy has failed overall in top-line data from an exploratory phase II study in ischemic stroke.

On the heels of last summer's phase IIb failure of TC-5214 in overactive bladder – a disappointment that pushed shares to a then-52-week low of $2.76 – and a string of other duds with neuronal nicotinic receptors (NNRs), Targacept Inc. said top-line results from a phase I/II exploratory study of TC-6499 as a treatment for diabetic gastroparesis also missed its primary endpoint and the program will be discontinued.

It might not add much to Pfizer Inc.'s bottom line, since oral Ibrance (palbociclib) is approved already as a first-line therapy, but the first randomized phase III data with the compound made a strong showing in the trial called PALOMA-3, stopped early because of solid efficacy.

The Medicines Co.'s intravenous antiplatelet agent cangrelor is taking center stage Wednesday because of an FDA advisory panel, but backers of the Parsippany, N.J.-based firm increasingly are looking at the rest of the pipeline, including the reformulated version of the intravenous Minocin (minocycline), RPX-602, due for a decision on approval from U.S. regulators this month.

As an advisory panel for the FDA prepared to take up the matter Tuesday of safety issues with Astrazeneca plc’s marketed dipeptidyl peptidase-4 (DPP-4) inhibitor for diabetes, Viking Therapeutics Inc. lowered the target for its IPO in a move that could help the firm advance the licensing deal with Ligand Pharmaceuticals Inc. – also with a strong diabetes component, though otherwise unrelated and involving drugs with a different mechanism of action).

The prospect of establishing a specially focused orphan drug megafund, through which investors put money down and collect on the spoils as drugs and intellectual property rights are sold to venture capitalists or pharma firms, is one due to be explored during a panel talk at the Allicense 2015 conference by Andrew Lo, Sloan professor of finance at the Massachusetts Institute of Technology (MIT).

Green-lighted by the FDA, Halozyme Therapeutics Inc. plans to start by the end of 2016's first quarter the phase III push with PEGPH20, a pegylated form of the firm's recombinant human hyaluronidase, in patients with metastatic pancreatic cancer – and the trial design, which could allow for approval based on either progression-free survival (PFS) or overall survival (OS) endpoints, has analysts enthused.

With breakthrough therapy designation (BTD) for rucaparib as a monotherapy in advanced ovarian cancer (OC), Clovis Oncology Inc. joins a limited few in the sector with more than one BTD win, having earlier achieved the same status for rociletinib, an epidermal growth factor receptor inhibitor for non-small-cell lung cancer.

The latest chapter in the long saga of Repros Therapeutics Inc.'s quest to get an estrogen blocker approved for secondary hypogonadism in overweight men gave investors cause for both optimism and concern, as the FDA's acceptance for filing of the new drug application (NDA) brought questions about the timing of an advisory committee meeting – and what U.S. gatekeepers might want next.