sports,
Not sure how to frame my response but will give it a shot. Going back a couple years ACT repeatedly made the case that starting with the SMD trials "was the only practical choice" and "Highest likelihood of seeing signal in phase I" made a ton of sense to me. I saw it as the road map to the AMD trials and was hopeful the SMD patient treatment would be well underway with some positive indications in place prior to EU trials and even the AMD trial in the States. I perceived this as important, logical and agreed with ACT this was the prudent pathway. As it stands now, we could have 3 or possibly even 4 trials underway simultaneously. Wonderful if everything goes as planned, not so if it doesn't. Some might say it's because they "know" it will work. I would respond by saying 85% of all clinical trials do NOT get past Phase 3 and receive NDA approval and I am sure most of the 85% did not spend 10's of millions of dollars without being just as confident. With that said, I await info on clinicaltrials.gov to get the ball rolling here in the US to treat a patient...jmo
1)Orphan Drug Designation
• Stargardt’s Disease is the only practical indication with which to begin clinical trial
• Highest likelihood of seeing signal in phase I.
2)ACT has obtained Orphan Drug Designation in United States
Significance of Market Exclusivity: Barrier to Entry
The only practical choice for first clinical trial involving
RPE cells for macular degeneration.
AI
