FDA gave FX-322 Fast Track status with data on 15 dosed patients in 2019. FDA cleared FX-322 for three concurrent trials in 2021 with multi-location support. And they're having an end-of-Phase 2 meeting in Q4 of 2021. Hardly seems like foot dragging to me...
Frequency Therapeutics seem to be excited to see that 90-day data, and confident. The "group" level data will be split into dose cohorts. They consider a success to be a measurable improvement over baseline that is clear between each cohort, improved QoL measures.
The JP discussion disagrees with this theory, unfortunately. They spoke about a future trial after those in 2021 complete. So, it's likely that there will be a 2022 Pivotal trial with the product probably available in 2023.
What is the likelihood that the mention of a pivotal trial was a conservative estimate? They may not need to do a pivotal trial if the multiple trials show positive effects.
I'm just going off other info from this thread. Hopefully by adjusting the dosing they can have more than 10 decibel gains @ 8 kHz.
Is this something that is a concern for human trials? I thought they were doing multi-dose trials on humans right now.
We'll know more at the end of the Phase 2A in probably June if there are specific audiogram improvements.
Yes they are doing multiple dosing on humans. I do not think this is a concern. It only explains why they didn't maybe do multiple dosing on animals before humans? Either way, a tiny mouse ear is likely more physically fragile than a human ear, so maybe they determined that. In my opinion, it's a non issue.
Animal models apparently could not withstand multidosing (presume anatomical differences) but human subjects can. Good news if multiple injections yields proportionately better results.
Well hopefully they will figure it out. It would be amazing to move on with life.
It would be certainly great if they got Breakthrough Therapy designation and could skip the pivotal trial. The more data they can compile that FX-322 is safe for multiple hearing loss designations, and if multiple dosing shows to be effective, obviously the more convincing it will be to skip the pivotal trial.
I tend toward predicting a product launch late 2022 if endpoint results are met in the the three currently running trials with no adverse effects. That does put some faith in the FDA to do their job in a timely fashion and award Breakthrough Therapy designation. If tinnitus results are also statistically promising and the VA gets involved (Veterans' #1 disability) then maybe some external pressure would accelerate the process. The media blitz following Phase 2a could prove helpful. Really the difference between @Diesel's opinion and mine on the timeline is but months.
No. But we will learn a lot that we don't know, and have burned hundreds of pages on this forum discussing:
We will know more about the answers to those questions as soon as March and then we can stop speculating
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It's not a concern. Other drugs in humans like Dexamethasone are given in multi doses without issue.
Number 4 as well though, no? I'm excited about the March results but more so for the other trial for severe hearing loss patients as that's where my damaged ear is at. (Moderately Severe to Severe).We will know more about the answers to those questions as soon as March and then we can stop speculating
The answer to number 3 will be a little more complicated because of bilateral tinnitus and the contribution of synaptopathy but there should be at least a significant difference between control and treated cohorts. This difference would be much more magnified if those factors were controlled for but we should still see a difference between the two groups.
Interesting enough, LeBel did mention on the J.P. Morgan presentation that they had internal filtering criteria to ensure a specific "benchmark" for all Phase 2A participants. I suppose it stands to reason that all 95 reporting having SNHL-related tinnitus. I wonder if they could get as specific as to recruit unilateral cases only?We will know more about the answers to those questions as soon as March and then we can stop speculating
The answer to number 3 will be a little more complicated because of bilateral tinnitus and the contribution of synaptopathy but there should be at least a significant difference between control and treated cohorts. This difference would be much more magnified if those factors were controlled for but we should still see a difference between the two groups.
Yes this is true but if the Phase 2a show positive results following on from the the 1 injection in the initial trial, then I think it's reasonable to assume any improvements, even if very minor (for the severe hearing loss population), in Phase 1b is something to be optimistic about for when they do their pivotal trial.
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