Shares in Swiss pharma company Santhera Pharmaceuticals took a 35% nosedive on the news that its lead compound, Catena®, failed to meet its primary and secondary endpoints in the Phase III European trial against Friedreich ataxia (FA). The firm admits the results represent a major setback but says it will wait to analyze longer-term data from two ongoing extension studies before deciding on how to proceed with development of the drug for FA.
Catena has already been granted marketing clearance for the FA indication in Canada, pending additional data from an ongoing study to verify its clinical benefit. The drug is separately in Phase II/III development against Duchenne muscular dystrophy, Leber hereditary optic neuropathy, MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes), and primary progressive multiple sclerosis.
The 12-month placebo-controlled Miconos FA study evaluated the safety and efficacy of three doses of Catena in 232 primarily adult patients. The results showed treatment with Catena did not meet the primary endpoint of mean change in the International Cooperative Ataxia Rating Scale (ICARS) score from baseline. Secondary endpoints including the proportion of patients improving on ICARS score and change in the Friedreich ataxia rating scale were also not met.
A meta-analysis of Santhera’s three Phase II and III trials showed ‘“trends for improvement” following treatment with Catena, the firm points out. “We are surprised and disappointed by the Miconos results,” remarks Thomas Meier, Ph.D., CSO. “However, we now have results from three clinical studies, which, when combined, show a trend for superiority of Catena compared to placebo as assessed by the ICARS. We are convinced that individual patients benefit from Catena, although we were not able to demonstrate this conclusively in this clinical trial of still limited size and duration.”
Santhera is anticipating interim data from the Phase III Catena trial in Duchenne muscular dystrophy during the second half of 2010. Results from a Phase II study in Leber hereditary optic neuropathy are due within weeks, the company states.
The firm currently has two other drugs in clinical development. Fipamezole has successfully completed a Phase IIb trial in the treatment of dyskinesia in Parkinson disease. The North American development and commercialization program for fipamezole is partnered with Biovail. Omigapil, a candidate licensed from Novartis, is currently being prepared for a Phase II/III development program in patients with congenital muscular dystrophy. Santhera also has a preclinical-stage melanocortin-4 receptor antagonist (MC-4R), which it expects to start in clinical development during 2010.