Targeted failure of the week. Post No 141-142. Cabozantinib and amikacin

Exelixis Inc. (NASDAQ:EXEL) fell $2.54 (39%) to $3.90 on Wednesday after an independent DMC said the Phase III COMET-1 trial evaluating Cometriq cabozantinib for metastatic castration-resistant prostate cancer (CRPC) did not meet criteria to support early trial unblinding. The DMC recommended continuing the trial based on a planned interim analysis of the primary overall survival (OS) endpoint. A final analysis is scheduled when there are 578 events, with data expected this year. The trial enrolled 960 patients and is comparing Cometriq vs. prednisone in patients who have progressed following treatment with docetaxel, Zytiga abiraterone from Johnson & Johnson (NYSE:JNJ) or Xtandi enzalutamide from Medivation Inc. (NASDAQ:MDVN) and partner Astellas Pharma Inc. (Tokyo:4503).

Insmed Inc. (NASDAQ:INSM) fell $2.39 (13%) to $15.90 on Wednesday after reporting that once-daily Arikayce amikacin plus standard of care (SOC) missed the primary endpoint vs. placebo plus SOC in 89 evaluable patients in the Phase II TARGET NTM (TR02-112) trial to treat treatment-resistant non-tuberculosis mycobacterial (NTM) lung infection. The primary endpoint was a reduction in mycobacterial density as measured by a seven-point scale from baseline to day 84. Arikayce plus SOC did meet the secondary endpoint of a greater proportion of patients with culture conversion to negative by day 84 vs. placebo plus SOC (25% vs. 7%, p=0.01). Additional data are slated to be presented at the American Thoracic Society meeting in May.
Insmed plans to meet with FDA and EMA to determine next steps for Arikayce in the indication, for which the product has Qualified Infectious Disease Product (QIDP) designation in the U.S. The company said it also plans to apply for breakthrough therapy designation in the U.S. based on the culture conversion data in TARGET NTM.
This half, Insmed plans to submit regulatory applications to EMA and Health Canada for Arikayce to treat Pseudomonas aeruginosa infection in cystic fibrosis (CF) patients. Insmed gained the inhaled liposomal amikacin through its 2010 acquisition of Transave Inc. Arikayce is administered with the eFlow Nebulizer System from Pari GmbH (Starnberg, Germany).