I realise this and my original post actually mentioned how various drugs have very different statistical outcomes, but I deleted it because I sounded like I was rambling on. I know quite a bit about how drugs make their way to market, so I apologise for simplifying it to make my post brief. I just don't have the energy anymore after COVID-19. My brain is frazzled.
We've beaten this to death on this thread, but a big part of the efficacy results will rest on successful recruiting of people like the Phase 1 responders. As I showed, the people who respond tend to respond big time. Our expectations for below 8 kHz ranges should be low (although some did reach 6 kHz), but above that, it's quite promising, considering there is currently nothing.
Just to note... the hyping on Twitter or elsewhere doesn't affect whether it fails or not from an FDA standpoint. For the most part the FDA cares about significant primary outcomes + safety.
Should the Phase 2A outcomes (primary and experimental) look even remotely favorable, I anticipate that Frequency Therapeutics will expand the breadth of hearing loss topics / media / communications to include those new endpoints.
Hmm... I ran my own Xmas themed analysis and I came up with these percentages for heading to the next phase at least:
If I stare at the pie with my left eye on the red half and my right eye on the green half, the right side of my brain is perceiving red, which is aggressive, and the left side of my brain is perceiving green, which is bright.
Agreed! I think there is much left on the table to analyze!
Excellent effort and very inspiring to see we will proceed regardless of outcome!
Terrific analysis and review, it is heartwarming to see the support and that the science is behind us on this one!
Nobody knows for sure, yet. Frequency Therapeutics appears to be actively working on improving delivery — which many think means getting FX-322 deeper into the cochlea. So, it may not in the first product, but it's likely the next version will go deeper.
I think if Phase 2a succeeds, eventually they can figure out how to penetrate deeper. All we need is confirmation the drug works in a meaningful way.
If your lowest dip in standard audiogram is only 5 dB then probably this won't be the cause. Have you checked your higher frequencies (above 8 kHz?). You might have there much more hearing loss without even knowing.
We will have the penetrance data by the end of this month, likely by the end of next week.
I'm glad you brought up Carl LeBel and his Otonomy connection. Regardless of anything else, Otonomy knows intratympanic formulations probably better than anyone else...
Highly likely. Carl LeBel and Chris Loose have also said that the goal for recruiting in the Phase 2A was to create a "homogeneous" group of participants that "closely matched the conditions of those responders from the Phase 1/2"... LeBel in January also reiterated that outside of the Phase 2A inclusion criteria, Frequency Therapeutics had a "second filter" that they were keeping secret to again, ensure patients met those responder conditions. I stated a few posts back what I believed they were based on the available data from the patent/Phase 1b study and comments from the firm.
Frequency Therapeutics has referenced the terms, "Dead, Missing, and Damaged" as it relates to FX-322 regenerating inner and outer hair cells. The extent of the damage is unclear at the cellular level.
Yep I saw that, I agree FWIW with your WR deficit range, based on Raffin table for statistical significance ranges + clinically meaningful I expect WR deficit range minimum is likely between 70-85% minimum, WR deficit needed to be eligible. If they did do long term onset hearing loss criteria in the trial, I'll be excited since that does appear to reflect the responder population much more as well as durability too (potentially reflecting their damage is at a steady state vs patients who are earlier onset and may suffer ongoing lower frequency damage despite restoration of EHF ranges).
FYI, the drug action (PCA) is activated for damaged hair cells as well. As @FGG pointed out to me, Carl LeBel said on the Tinnitus Talk Podcast that he didn't see the non-responding damaged cells as being a major issue. We'll see.
I won't get into the weeds, but the placebo data was nothing at all. They set a 90% ceiling threshold before the study. Of the 4 eligible, 2 improved slightly and 2 worsened slightly by day 90.
He could use a talk with Toby.
The fact that they are calling this a "stem cell treatment" shows their level of knowledge about the drug...
I recall an article a while back confusing FX-322 as a "stem cell" treatment. I think the author mis-understood Frequency Therapeutics' description of progenitors being "stem cell like." I imagine it's happened more than once, since it's pretty difficult to get someone's head wrapped around progenitors in less than 2 minutes. So, now every once in a while FX-322 is now erroneously associated by the uninitiated as a stem cell treatment.
It's funny how that works. I spent a High School year in England and took A level history and one of the only things I remember is that millions of "witches" didn't die in England witch hunts (60,000 tops) but one history book got it wrong and everyone just quoted that one (and quoted others down the line) for years until someone realized the mistake.
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