Back to the subject of controlled clinical trials, Craig P. writes, "Do you know if Ariad Pharmaceuticals ( ARIA) used uncontrolled studies in their Phase II trials of deforolimus?" The phase II study of deforolimus in sarcoma patients did not contain a control arm. This study of 212 sarcoma patients resulted in a 29% response rate and median progression-free survival of 15 weeks. Again, there was no control arm for comparison, but historical median PFS rate for these patients is around seven weeks, according to a recent research note from Susquehanna Financial Group.
Moving on, Fred B. writes: "What do you think of Alexza Pharmaceuticals ( ALXA)? The company expects to report top-line results in a second phase 3 study of AZ-004 by the end of this year for acute agitation in patients with schizophrenia or bipolar disorder. An earlier phase 3 study met the primary endpoint. They also have had clinical success with AZ-104 (Staccato loxapine, in a low-dose form) for migraine. The stock is currently trading with a market cap of $100 million at $3 a share, which matches its cash level." Alexza is developing a small portable device dubbed Staccato that vaporizes drugs into a mist that can be easily inhaled into the lungs. The appeal of the Staccato system is that inhaled delivery of a drug into the lungs (and therefore into the bloodstream or brain) accelerates the drug's onset of action much more than conventional means of delivery. Pills, for instance, take a relatively long time to work. Injectable drugs work fast but most require trained personnel to deliver them. Staccato makes the most sense for patients with diseases characterized by acute attacks. Think migraines or anxiety attacks.