Well - I would hope for a bump to $3-4.00 range after mtd and safety established. A little more with documentation of decreased specific biomarkers and p53 activation evidence.
So - over the past decade - the model for PhII has totally changed. It used to be all about the single arm phII - using small # patients and historical control data (which is useless to novel agent study - no applicable null hypothesis for truly novel agent). For novel molecules - there are 3 main ways the company can go.
A randomized selection design has no control, randomly enrolls patients into 1 or more arms, testing different dosages and dosing schedules. The arms aren't compared with each other - researchers just choose the one that looks best (remember - this is about structuring the best phIII trial) and the false positive rate is 10-20%. This design is my favorite because researchers can run multiple arms at the same time (even 10 years ago people ran them sequentially), hugely saving time on the path to a phIII.
Second novel agent design is randomized comparison design - this uses formal statistical design to compare experimental groups with a control(standard of care treatment) or placebo. This design looks a bit like a phIII - but with the small numbers of participants, the false positive rate is much higher at 10-20%, and the small patient pool means toxicity and intolerance signals are often missed and then picked up in phIII, which is a really expensive mistake...
Third design is randomized discontinuation design. All patients receive treatment in the beginning of the trial. Nonresponders and patients that didn't tolerate the drug well are removed from the study. Patients who experience stable disease (no better but no worse) are then randomized into placebo and active treatment groups. Patients who showed good response (tumor shrinkage) from trial initiation continue treatment uninterrupted.
So - all of these designs help the company select a target population for a phase III trial. That is biggest goal - to provide findings that will be confirmed in phIII with the populations most susceptible to the drugs activity. They establish efficacy data - but with a much larger margin of error that a phIII. Tumor growth inhibition can be acceptable response criteria for a novel agent - as they have been seen to prolong life with minimal to no tumor reduction....They continue providing safety/tolerance/PK/PD info - and liver stress profile much more meaningful here. They further define biomarker correlates. Dosages are refined.
Phase II studies can take anywhere from 6 months to 2 years.
If I were running ctix - I would do a muti-arm phII and have about 6 small arms running concurrently - with the cancers shown to be the most sensitive in this phase one...
