If there's a small hitch in the phase II/III data rolled out by Orphazyme A/S with arimoclomol against the lysosomal storage disease Niemann-Pick type C (NPC), it's that the trial missed a co-primary endpoint chosen by the FDA, and the firm plans to take the results with the heat-shock protein (Hsp) amplifier to regulators in the U.S. and EU in the first half of next year.
Toronto-based Triphase Accelerator Corp. and its majority shareholder, Fight Against Cancer Innovation Trust (FACIT), disclosed a new epigenetics deal with Celgene Corp. for what's described as a first-in-class preclinical therapeutic targeting the WDR5 protein for the treatment of blood cancers, including leukemia.
Investors and others in the field of Parkinson's disease (PD) are weighing the odds of two major players in treating "off" episodes: Sunovion Pharmaceuticals Inc. with apomorphine sublingual film (APL-130277), for which the FDA set Tuesday as the PDUFA date; and Acorda Therapeutics Inc., of Ardsley, N.Y., preparing to launch Inbrija (levodopa inhalation powder) this quarter. Off periods are times when PD symptoms return because of low dopamine levels between doses of oral carbidopa/levodopa.
In December, when Puma Biotechnology Inc. disclosed data with the breast cancer drug Nerlynx (neratinib), a kinase inhibitor that irreversibly binds to EGFR, HER2 and HER4, questions that were already on the burner bubbled harder regarding the drug's future, especially given the shifting treatment setup in HER2-positive disease.
CEO Andrew McDonald told BioWorld that Attune Pharmaceuticals Inc. made 1,200 compounds – "a tour de force, from a medicinal chemistry perspective" – in order to find two that worked the way the company wanted as oral plasma kallikrein inhibitors in the prophylactic treatment of hereditary angioedema (HAE).
Company watchers remained upbeat as analysts during a conference call Friday pressed Immunomedics Inc. for details about the complete response letter (CRL) regarding the BLA related to antibody-drug conjugate (ADC) sacituzumab govitecan for metastatic triple-negative breast cancer (TNBC).
Investors awaiting word from Immunogen Inc. in the first half of this year regarding the phase III trial called Forward-1 in platinum-resistant ovarian cancer continue to debate the odds of success with antibody-drug conjugate (ADC) mirvetuximab soravtansine. Previously known as IMGN-853, mirvetuximab soravtansine targets folate receptor alpha (FRa).
When he took the position about a year ago, CEO Joe Turgeon of Spectrum Pharmaceuticals Inc. saw "right off the bat, when I met with my senior staff" that the company needed to change, he told BioWorld. "We had these important products, but they were small niche products, and we were classified as a specialty pharma company."
What Oppenheimer analyst Leland Gershell a few months ago called "the shift toward non-opioid/locally delivered treatment strategies [in pain], driven by increasing regulatory/payer/institutional hurdles" has brought a handful of players to the fore.
Norwood, Mass.-based Corbus Pharmaceuticals Inc.'s deal earlier this month with Kaken Pharmaceutical Co. Ltd., of Tokyo, to develop and commercialize lenabasum for systemic sclerosis (SS) and dermatomyositis (DM) in Japan shone more light on drug developers' prospects in the former indication, but interest already ran high in some quarters.
