At this pace...
I'd wager:
2021: Breakthrough Designation
2022: Pivotal Phase
2023: FDA Approval
I think that this is quite feasible, however I wouldn't be surprised if Frequency Therapeutics got the treatment released under compassionate use early if it is successful --- even if it is not as successful compared to what they can get out there post the pivotal trial. How FDA is seemingly increasing its approvals for medicine to get fast tracked and also with their willingness to allow compassionate use more, I don't see the FDA being hesitant to release successful hearing medicines. Particularly when this is an area with no treatment options currently available and also that it will provide great benefit.
I don't see the FDA withholding a release if it is the case that FX-322 is effective and also actually provides benefit. At this point, I am also of the opinion that Frequency Therapeutics will also want to release FX-322 as quickly as possible so they can start attaining revenue from the sales.
Supplements are different. As long as the claims are vague the FDA allows this and does not regulate (unfortunately in the case of scams though overregulating supplements would have drawbacks).
Not to mention that nowadays a high proportion of those magazines are really just infomercials in print form and as a result the scientific validity of the articles they publish is questionable.
So FX-322 will be out in 2023 and not earlier?
Not at all. At this stage the questions which need to be answered are:
1. Whether FX-322's phase 2 results comply with the FDA requirements to obtain compassionate use. If they do, then Frequency Therapeutics can apply to the FDA to allow people to use the medicine under the right to try provision.
2. The next question is whether Frequency Therapeutics would allow people to obtain FX-322 for compassionate use. Frequency Therapeutics has stated that they would consider revisiting compassionate use access when this became an appropriate and/or relevant option to consider. Consequently if compassionate use was granted then treatment could start post phase 2.
Therefore there tends to be a reasonable probability that FX-322 could be released prior to the trial process being completed. Currently I do not think that there would be any desire for Frequency Therapeutics to flat out refuse to allow compassionate access as they wouldn't be willing to consider this otherwise. Therefore I think that there could be a real possibility that FX-322 may get released earlier/prior to phase 3.
Until there's more data or widespread use, what they put out will be the most accurate. I would take any testimonial you find with a grain of salt. That was a big mistake in the discussion around OTO-313. A user came on here claiming they must have had the real thing and said it was poison, but then it turned out they had the placebo and the drug in fact worked.
A lot of these testimonials that turn up in these magazines are actually also designed to simply advertise and/or promote a particular product. Pretty sure that we can just look at the Lenire article as a good example of how you can try and convince people a treatment works under the guise of supposed scientific research. Frequency Therapeutics is not looking to do that and actually has seemingly neither be interested in nor worried whether stuff about FX-322 gets published from how I perceive things to be.
I really think that this tells us a lot about Frequency Therapeutics too because they are only interested in information which is seemingly objective and verified. Same can be said with what we have seen from Otonomy. Thus I tend to have a lot more confidence in what these organisations are doing and that their treatment goals are focused on delivering people the best outcomes foremost.
We don't know anything about the Phase 2a, except the design of the trial. I assume the 90-day release will be a top-line readout. Which means they will show the baseline and 90-day aggregate result for each endpoint and each injection cohort compared to placebo.
EXAMPLE: (all at 90-days):
All would show injection vs placebo:
Word Score - Quiet: - 1-injection - 50%, 2-injection - 60%, 4 injection 80% (p-value 0.010)
Word Score - Noise: - 1-injection - 30%, 2-injection - 50%, 4 injection 70% (p-value 0.0x0)
Pure Tone Average: - 1-injection 10 dB improvement, 2-injection 20 dB, etc etc
TFI: 1-injection 10% reduction, 2-injection 20% reduction, etc etc
You get the idea... I would be delighted to see similar detail as the Phase 1/2 results; with hopefully some insight into audiogram improvements and breakouts into cohort improvements. However, I expect it to be a top-line summary of sorts; otherwise I don't see how it could be done without ublinding the trial.
I don't think that they necessarily need to unblind the trial to get a reasonable understanding of what the trial outcomes were. Unblinding would make data interpretation much easier because you'd know exactly who got what and also actually what their benefit was.
I think if they provided the data about various participants like how you described above then there tends to be a reasonable ability for someone to assume and/or predict what might have happened. For example if you got a bigger improvement, it is reasonable to assume at the moment that you got more doses of FX-322. If the unblinding gave a different result (such as less doses produced a better result) then I think that Frequency Therapeutics would reassess why this is the case and also actually reevaluate their dosing process probably. Pretty confident that the treatment with FX-322 will work better with more doses based off of the research.
Overall I think that the thing most will want to see is that FX-322 is providing benefit and that it is hopefully providing more benefit with more injections. If it is the case that more FX-322 is providing more benefit, then it may also further give us insight into if more doses are required/will yield a bigger benefit. This is because if we are seeing that more doses is working more substantially and also reach deeper into the cochlea it will not only confirm Frequency Therapeutics' position that more intratympanic injections will lead to more deeper penetrance and also that it is entirely possible that users will benefit from even more than four shots as well.
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