Good news today. Clear presentation of progress. The biggest factor for today's mediocre market response is the uncertainty of whether or not the FDA will have any last questions ahead of IND effective.
The really good news is that they expect that a presentation and abstract will be ready by this time next year. That means P1 preliminary reports should be ready long before then, and all data will be ready and interpreted for a major presentation of results. So, although the primary endpoints are safety and tolerance, the secondary endpoints will include efficacy in select terminal patients. There is no reason to believe from animal studies that positive results for all endpoints can't be achieved.
But, first the IND needs to be effective and the DF protocol started.
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