Frequency Therapeutics — Hearing Loss Regeneration
- Thread starter RB2014
- Start date
That's the hope yes, but it depends on a lot of factors. I don't think 2021 is very likely but 2022 is possible.
No I don't think 2021/2022 is very likely, unless you're in a trial. They are still enrolling patients for Phase 2a and following them for 210 days. That puts us in January 2021 at the earliest before they finish. Then they have to analyze the data, talk with the FDA etc. so now it's probably mid 2021 before they release Phase 2a data. And that's conservative. Keep in mind enrollment for Phase 1/2 finished in October 2018 with data release in April 2019 and they followed patients for only 90 days. Phase 2b will likely take us all the way through 2022 and Phase 3 probably takes even longer considering the larger amount of patients. I think 2025/2026 is more realistic. Carl LeBel himself said that he thinks a hearing less drug will be released "this decade".
Member- Mar 14, 2020
- 180
- Tinnitus Since
- 12/19
- Cause of Tinnitus
- Noise Exposure + Viral Infection
- Feb 14, 2020
- 1,630
- Tinnitus Since
- 1-2019
- Cause of Tinnitus
- 20+ Years of Live Music, Motorcycles, and Power Tools
I recall a few months back reading how much the Fast Track and Breakthrough Therapy designations affect the successful release of drugs to the market vs. those that do not gain any status. It was geared towards investors seeking to make gains investing in small single-drug research firms like FREQ. The study looked at the overall differences in timeline from the start of the first clinical trial.
If I recall correctly. The Fast Track, on average, shortened the timeline to 7 years. Breakthrough, on average, shortened the timeline to 5 years.
So, we know that the first clinical trial for FX-322 started 2017.
Based on that research, Fast Track designation puts the market availability at likely Mid 2024, Early 2025. Breakthrough may bump it up to 2023. (Since it may be released concurrently with the Phase 3).
Also @FGG shared an article a few comments back about the FDA possibly helping "speed things along" in response (pressure) due to COVID-19 putting a pause on research/development.
In my humble opinion, 2024 seems like a reasonable median year.
Side note: 2024 won't seem so far away if in the Phase 2A, we learn that it reduces tinnitus symptoms.
- May 2, 2020
- 202
- Tinnitus Since
- 01/2019 T, 04/2020 H
- Cause of Tinnitus
- Headphones/Car Alarm/Immune Response?
I agree. Hopefully they ramp up production and open up compassionate use after phase 2a. If not then perhaps after phase 2b.
- Sep 21, 2016
- 1,051
- Tinnitus Since
- 2011 - T, 2016- H, relapsed 2019
- Cause of Tinnitus
- noise-induced
Frequency will be participating in a virtual "fireside chat" tomorrow at the Goldman Sachs Annual Healthcare Conference - webcast will be available on their site. I suspect it will be much the same as their latest conference but might be good to tune in.
I really do hope it's earlier. If they do end up skipping Phase 2b that would be really good for us.
If compassionate use is available after Phase 2a I'm definitely signing up for it. My life is on pause right now.
- Mar 14, 2020
- 180
- Tinnitus Since
- 12/19
- Cause of Tinnitus
- Noise Exposure + Viral Infection
Jesus Christ, these numbers are insane. 7 years of waiting for a potentially life changing drug, after it's already been fast tracked, is asinine.
FDA reform needs to happen today, not tomorrow.
I agree. The article about the FDA wanting to speed up the process should be a good thing for us. Let's hope that we can get this drug out of the market ASAP. No one deserves to suffer from this.
- May 2, 2020
- 202
- Tinnitus Since
- 01/2019 T, 04/2020 H
- Cause of Tinnitus
- Headphones/Car Alarm/Immune Response?
They have a financial incentive in the form of a milestone payment from Astellas to not skip phase 2b. That being said, I would be surprised if they do not open up compassionate use by 2022.
I agree Breakthrough Status would be a game-changer. In that case 2025/2026 would be a bit bearish.
Why not participate in a trial then? You don't have to wait for the drug to be released. Nothing speeds up FDA approval more than patients willing to participate in trials.
Okay, then this seems to be common practice to publish trial data not directly after the study finishes and we shouldn't be so hard with Audion.
However maybe Frequency is more communicative and we can ask them whether they open up for compassionate use when the phase 2a study has some positive results. Hopefully they will at least publish some partial results of this study already this year. Even for compassionate use demand will be quite high if this treats noise induced tinnitus. Probably pricing will be some multiple of stem cell treatment. Anyway I would sell my apartment if this has a high chance of being effective.
So you would apply for off-label compassionate use? I don't want to be an asshole, but I don't see how the FDA is going to go for that. Should FX-322 also get a tinnitus indication things might be different, but then you're better off in a trial anyway. I mean, let's not forget that, apart from any FDA hurdle, Frequency has an interest in people participating in their trials. Compassionate use doesn't help them and if they are too lenient with that, why would people participate in trials where they have a chance of getting a placebo?
Watching the Fireside, I have hearing issues so hopefully someone can confirm:
--sounds like they want their next trial after this to be the "pivotal" one. Meaning whether 2b it 3, they want it to be the last one before approval. So it looks like there will 1 more trial after this one, not 2.
--measuring phase 1 people after a year, the four they were able to follow up with, all maintained improvements. 1/4 didn't maintain the same degree of improvement but still improved. This is something they will try to get wider data on in phase 2. This means these weren't "more fragile" hair cells as some people worried about.
--sounds like they want their next trial after this to be the "pivotal" one. Meaning whether 2b it 3, they want it to be the last one before approval. So it looks like there will 1 more trial after this one, not 2.
--measuring phase 1 people after a year, the four they were able to follow up with, all maintained improvements. 1/4 didn't maintain the same degree of improvement but still improved. This is something they will try to get wider data on in phase 2. This means these weren't "more fragile" hair cells as some people worried about.
- Feb 14, 2020
- 1,630
- Tinnitus Since
- 1-2019
- Cause of Tinnitus
- 20+ Years of Live Music, Motorcycles, and Power Tools
Questions:
COVID Challenges
Phase 1 Data. What are they looking for in Phase 2?
COVID Challenges
- All clinics determined to be essential. All remained open.
- Continued to support subjects on the trial.
- Able to continue to recruit.
- Lightening Restrictions = more activity in clinical
- Look at Pre-Clinical Data. They did their work before the trials to validate the science
- Dosing extracted cochlea tissue with FX-322 and able to show cells dividing
- Study design = convincing data (statistically significant, clinically meaningful)
- P2 Objectives:
- Replicate Phase 1 Study
- Impact of multiple doses
- All subjects have a certain threshold that allows them to improve a certain amount
- Longer period of time. Determine durability.
- Replicate Phase 1 Study
- 12 - 21 Months later.
- 4 Subjects that were statistically significant;
- 3 maintained hearing levels
- 4th maintained benefit; fell out of significance
- Hair cells likely durable. Frequency says very encouraging.
- Dodged a "yes/no" question.
- Preliminary evidence for long-term durability.
- New hair cells were created; they are durable.
- Replicating same process as what happened in womb. So, should be for decades.
- Does it get there? And how much? Does it have a signal?
- 7 subjects received FX-322
- Simulation model
- Both drugs get into cochlea
- Achieve improvements within the range predicted
- Bottom Line:
- We know in the lab what levels need to make human hair cells grow
- Now we know at the clinical level is needed to make human hair cells grow
- Data:
- Replicate what had been seen in in Phase 1 study
- Add specificity
- Hearing Signal Improvement (Stat sig, clin meaningful)
- Additional Injections = Improved Signal
- Validate : Extended high frequency improvement (Word clarity)
- Hypothesis (Lebel):
- Increase responses with more individuals (more people see similar improvements)
- Increase range of improvement (some people see wider range of improvement .. khz?)
- Or both
- 6-Months duration:
- Need to find the plateau. Still saw improvements at end of Phase 1.
- Know that hearing will STAY; permanent improvement.
- Overwhelmingly positive feedback - ENT/Audiologists
- FDA views hearing loss as clearly life-altering
- Growing awareness of hearing loss affecting depression/anxiety/other maladies
- Understanding of the importance of restoring hearing
- Phase 1 interaction was positive, continues to be engaged
- Fast Track Status
- Allows more interactions
- Looking forward to Phase 2A Readout
- Identify dose schedule
- Reinforce end-points
- Further characterize patient
- Identify dose schedule
- Hoping to go to Pivotal in next phase (after reviewing Phase 2A with FDA)
- Go to Market Strategy
- Clarity of sound is the unmet clinical need
- Expand the population of people that ENT/Audiologists can help
- Durability is compelling
- Advocacy Groups
- People are ready to engage/improve hearing
- Clarity of sound is the unmet clinical need
- Size of market
- US: 10s of millions people, prevalence is considerable.
- World: Same ratio as the US
- US: 10s of millions people, prevalence is considerable.
- International Opportunity
- Regular meetings with Astellas.
- Astellas is working on regulatory process world-wide
- Astellas is working with key opinion leaders globally. Overwhelming positive feedback, response.
- 2A readout is extremely important.
- Regular meetings with Astellas.
- Mar 14, 2020
- 180
- Tinnitus Since
- 12/19
- Cause of Tinnitus
- Noise Exposure + Viral Infection
Also it's been mentioned in this thread that Frequency is measuring tinnitus levels as a part of their phase 2a study. Most agree that tinnitus is associated with hearing loss in the majority of individuals who have it...
I really don't think getting FX-322 via expanded use for debilitating tinnitus is much of a stretch at all. Even if it were technically considered off-label, there's a profound relation between hearing loss & tinnitus - I think the powers in charge of approval would see likely see that connection.
- Mar 14, 2020
- 180
- Tinnitus Since
- 12/19
- Cause of Tinnitus
- Noise Exposure + Viral Infection
Super informative & reassuring! Now the only questions I have left for Frequency are:
1.) Expanded Use, yes or no?
2.) What's the plan for reaching lower frequencies? Since they've implied that is their goal.
3.) How long will this "pivotal" trial go on for?
Of course I suspect all of these will be answered in time, I just wanted to list what I currently see as the most important questions...
In the Fireside, Frequency has said the FDA considers hearing loss to be a very serious disease that dramatically affects quality of life and for which there is an unmet need. Since hearing loss is the cause of cochlear origin tinnitus, it's not only not a stretch, it's the way more likely scenario imo.
FDA says "if participation in a trial is not possible". That's not the same as getting it for a different disease than that the company is aiming the drug for.
I think my point still holds. If Phase 2a has some positive results for tinnitus, Frequency would be fools to not include it in a next trial (try to get in!), if it doesn't do anything/barely anything for tinnitus, why would the FDA give it to you even before it's approved? I can imagine, however, an ENT willing to give it a shot for tinnitus after it's approved for hearing loss only. That's off label use like dexamethasone that's being used for tinnitus already.
Great stuff. I wonder if "pivotal" actually means it's the last trial or if it's the final trial to determine patient population (with a Phase 3 after that to confirm). Wikipedia says final trial https://en.wikipedia.org/wiki/Pivotal_trial.
Edit: presentation is online. https://kvgo.com/GSHC/frequency-therapeutics-june-2020
Aye, they might be too cheap to finance it themselves, but it obviously helps their soldiers.
- Feb 14, 2020
- 1,630
- Tinnitus Since
- 1-2019
- Cause of Tinnitus
- 20+ Years of Live Music, Motorcycles, and Power Tools
The US Military and the VA would certainly be two very reliable consumers of this product.
In the Fireside chat did they actually specifically say that after Phase 2a there will be one more pivotal phase before approval?
Does that mean it's most likely going to be Phase 3?
Does that mean it's most likely going to be Phase 3?
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