Less than a week after Cell Therapeutics Inc. (CTI) snagged a handsome deal with Baxter International Inc. for its Janus kinase (JAK) inhibitor pacritinib – adding another industry vote for the approach – Sanofi SA disclosed safety problems with its JAK inhibitor fedratinib and ended the Phase III effort, which sparked a new look at the class.
Likely breathing easier ahead of Tuesday’s FDA advisory committee meeting is Biomarin Pharmaceutical Inc., with Vimizim (elosulfase alfa) slated to undergo scrutiny as an enzyme replacement therapy (ERT) for mucopolysaccharidosis Type IVA, also called Morquio A syndrome.
Epizyme Inc.’s Phase I study, designed mainly to determine the safety of EPZ-5676, an inhibitor of the DOT1L histone methyltransferase (HMT) for the treatment of acute leukemia with rearrangements in the mixed lineage leukemia gene (MLL-r), ended up showing treatment effects in four of eight patients.
Synta Pharmaceuticals Corp.’s new money from a public offering – about $52.5 million in gross proceeds – will fuel an expanded Phase III push with the heat-shock protein 90 inhibitor ganetespib in non-small-cell lung cancer (NSCLC).
The FDA altered its view of Sarepta Therapeutics Inc.’s data with exon-skipping therapy eteplirsen for Duchenne muscular dystrophy (DMD) after Prosensa Holding NV’s failure with exon-skipper drisapersen for the condition, but Sarepta CEO Chris Garabedian hopes the agency can still be brought around.
Something of a mystery surrounds the Phase II dashed hopes with Zalicus Inc.’s oral calcium channel blocker Z160, which missed its primary endpoints in two pain indications, testing patients with lumbosacral radiculopathy and postherpetic neuralgia.
Salix Pharmaceuticals Ltd.’s $2.6 billion buyout of Santarus Inc. for $32 per share in cash provides entry into the primary care physician space, while bringing aboard more products – albeit a couple that are facing patent expirations fairly soon.
The Johnson & Johnson (J&J) Innovation Center, through J&J unit Janssen Pharmaceuticals Inc., is pledging up to $10 million up front per program, followed by as much as $145 million more for each, in a deal with Evotec AG that aims to find and target the true originators of Alzheimer’s disease (AD).
The FDA’s partial clinical hold on CUDC-427 because of a cancer patient’s death from liver failure overshadowed third-quarter earnings for Curis Inc., which is analyzing possible causes.
Keryx Biopharmaceuticals Inc.’s Zerenex (ferric citrate) may be poised to become the first oral iron booster that also provides phosphorous control in chronic kidney disease (CKD) patients, if new top-line data from a Phase II trial hold up.
