Altus Falls Sharply on Cystic Fibrosis Trial Results (ALTU)
Altus Pharmaceuticals Inc. (ALTU) is trading lower after mixed results in a cystic fibrosis Phase III trial. Unfortunately, the results weren’t quite what the investment community was hoping for.
The study met some of the endpoints, but there were hopes for better results that had been indicated and some results varied geographically between those in the U.S. and those outside the U.S. This was its Phase III efficacy trial for cystic fibrosis patients suffering from pancreatic inefficiency.
So far this morning we have also seen a downgrade as Cowen & Co. lowered its rating to NEUTRAL from Outperform.
Shares are down over 30% pre-market at $3.36 as of 8:40 AM EST.
JON OGG
August 12, 2008
Genzyme Targeting Genetic Disorders in New Pact (GENZ)
Genzyme Corporation (Nasdaq: GENZ) has formed a collaboration and development pact to go after genetic disorders. It has partnered with PTC Therapeutics, Inc. in an exclusive global collaboration to develop and commercialize PTC124. This is PTC’s novel oral therapy in late-stage development for the treatment of many genetic disorders due to nonsense mutations.
PTC will commercialize PTC124 in the United States and Canada, while Genzyme will commercialize the treatment in all other countries. Genzyme is making an up-front payment of $100 million to PTC and has committed to potential milestone and royalty payments to PTC as well in an amount that could be up to $337 million. PTC will be financially responsible for one ongoing and three additional clinical trials of PTC124.
The release notes that this is potentially applicable to hundreds of genetic diseases. It is currently being evaluated in a phase IIb trial for Duchenne muscular dystrophy (DMD), and a phase IIb trial in cystic fibrosis (CF) is expected to begin by the end of this year.
Jon C. Ogg
July 17, 2008
Inspire Scores on Cystic Fibrosis Phase III (ISPH)
Inspire Pharmaceuticals Inc. (NASDAQ: ISPH) is seeing shares surge in pre-market trading this Friday morning. The company announced that it reached its primary endpoint in Phase III trial of Denufosol for Cystic Fibrosis.
This is regarding its top-line results from a 352-patient, double-blind, 24-week placebo-controlled portion of its Phase III trial with Denufosol tetrasodium inhalation solution for the treatment of cystic fibrosis. Denufosol demonstrated statistical significance for the change in FEV1 from baseline at the trial endpoint, its primary endpoint. There was a statistically significant improvement in FEV1 compared to the placebo.
Patients on denufosol improved relative to baseline whereas patients on placebo remained essentially unchanged; while the treatment effect of denufosol increased over the 24-week placebo controlled period. Preliminary data from 210 patients who have completed the ongoing open-label safety extension suggest that FEV1 continued to improve during the extension period from weeks 24 to 48.
Shares closed at $3.85 yesterday, and shares are now up at $6.40 in pre-market trading with just under an hour to go. Before the pop, this was a $217 million market cap.
Jon C. Ogg
June 6, 2008