ASTX had a hard time at the Ad Comm meeting this morning. The discussion was quite interesting. A committee member said it was the most difficult decision he had made since he began serving on these committees.
The question presented to the committee was whether ASTX had shown a favourable benefit/risk profile for Dacogen in the elderly AML setting. While it was acknowledged that the drug had a well understood and manageable safety profile (the risk side of the equation), the company had failed to prove benefit. This ultimately led to the final vote of 10 nos to 3 yeses.
Based on the discussion I thought there might be more in the yes camp. The clinicians seemed to be coming down in favor of the drug due to the potential for benefit, the safety profile, and the lack of available treatments for the disease.
It would be very surprising if a CRL were not forthcoming based on this meeting. The FDA commented that they also had had difficulties with many of the same questions discussed and thanked the panel for its guidance. Trading was halted until 2pm. Currently ASTX is at ~$2.
This is still a company that has a product on the market. I would guess, based on the discussion, that there will be significant off-label use of Dacogen in the AML population. In addition, it makes money and has $1.35 per share in cash. So worth keeping an eye on this one.