Recent content by Sunshine007

The responder definitions are not the same, Frequency Therapeutics uses the Thornton Raffin scale to classify a responder which is a pretty high bar. If you have a reliable history of word recognition scores and end up being a responder in that scale, that's a very significant response.

One issue with the X+Y argument is that there might be a ceiling effect to X that dwarfs Y and you don't quite have the candidate profile to observe Y. Let us assume people with true scores of 45 whose baseline was established at 25, we are not going to see a 10 word increase that might have...

I agree it's bizarre they did no validation of baseline data.They reported only the responder rate. I wish they had put out some more metrics on the extent of improvement as well. Maybe nothing worthy of reporting there, else they might have put it out. On Table 2 there seems to be an improving...

If it does lead to hair cell loss, shouldn't FX-322 be able to do its magic? Do we summarize that no vascular build up is a necessary condition for FX-322 to work? Another issue is people who have both NIHL and ARHL, should we not expect them to see any benefit?

What does the failure of age-related hearing loss mean in terms of how FX-322 may be working? Can we make any new hypotheses on the mechanism of action? Earlier we thought FX-322 produced hair cells, just that multiple injections interfered in seeing results. Is age-related hearing loss in some...

My understanding of this is there will be a couple of tests before a baseline is accepted. I think they will want to see stable word recognition scores (within a tight range) during the lead in period before receiving treatment.

Good one :) At the end of the day we let the data take us where we want to go or where we have to go :)

I meant in a Phase 2 trial we are looking for a signal and trying to find the right dose. I do not think a Phase 2 is powered to observe statistically significant effect vs placebo. Usually there is an arm in the Phase 2 trial that does exactly what was observed in the Phase 1 trial but with a...

Based on my understanding Frequency Therapeutics is saying all arms, all doses behaved the same. All treatment arms were impacted by the lawn theory. There is just a lot riding on lawn theory here because ordinarily we would expect to see the 1 dose cohort perform the same as past trials. They...

Ultimately the proof that the drug works and is safe is confirmed in a pivotal trial.We need to know the dose to do that. It's not clear to me that a Phase 2 trial needs to demonstrate a level of efficacy to advance to pivotal. While Phase 1 was designed to explore safety it demonstrated...

On the following hypothetical: If Phase 1 showed safety and no effect but Phase 2 did show efficacy, Phase 2 would have shown a multiple dose schedule was the optimal way forward. My point is we are finding the therapeutic dose, it is a question of finding what works best. Based on all...

If we ignore the phase monikers, essentially the reported Phase 2 trials and Phase 1 trials differ in n and probably the randomization criteria. If we are talking about the same dose, I am totally with you on the above, however here we have a situation where the higher n was the wrong...

I think it really comes down to the upcoming readouts. If the readouts are similar to the Phase 1, it is quite possible in my opinion. Whether they will is of course the million dollar question :) It is is a different population age wise and hearing loss wise. If the readouts are good, they...

Based on my understanding, Phase 1 is very low n to show drug does not have major safety issues. Phase 2 trials are meant to enlarge population and study optimal dose and also fine tune the target population. Phase 3 is the pivotal trial where you show with statistical significance what the...

Essentially what has been stated by Frequency Therapeutics is an artificially low baseline was established. The faker intent being speculated here may not really apply to the entry but people might have faked their baseline thinking they are helping the trial outcome without really understanding...