Well it depends on when the next trial starts and how long of a follow up they intend to do. The end of this year will provide much better time frame estimates.
Frequency Therapeutics — Hearing Loss Regeneration
- Thread starter RB2014
- Start date
MemberWe have the rest of Phase 2a which will seemingly take until early to mid 2021 to conclude. Then, the follow-up and results will need to be analyzed and published. Recruitment for either Phase 2b or 3 will take some time. This phase will be the largest in terms of trial population.
It's possible that FX-322 could be granted Breakthrough Therapy designation by the FDA following Phase 2a, but it doesn't seem like Frequency is expecting that considering their recent presentations. They anticipate Phase 2b or 3 being the "pivotal" phase prior to approval. Of course, that phase will also take a good amount of time as the study design has to well-constructed.
In my opinion, the first formulation of FX-322 is 3-5 years away from reaching the market. Our best shot for receiving it earlier would be through Compassionate Access/Expanded Use. Frequency is not ready for this quite yet as they are testing efficacy and safety of increased dosage in Phase 2a and production lines will have to be significant to meet the substantial demand. I feel that provided everything goes well, after Phase 2a, Frequency could begin to grant Expanded Use cases. Phase 2a results will be very telling.
My prediction: they might allow compassionate use after they are done recruiting for the pivotal phase as not to slow down recruitment for people who would rather not risk getting the placebo.
This would put that end of 2021 at the earliest, possibly early 2022 if they want every trial participant dosed at least once already.
- Feb 14, 2020
- 1,630
- Tinnitus Since
- 1-2019
- Cause of Tinnitus
- 20+ Years of Live Music, Motorcycles, and Power Tools
I think the only reason they haven't been bringing up the Breakthrough Designation talk, like they were back in November of 2019, is that they can't hype it as a publicly traded company. Over the past 6-months, we've seen how they've changed their tone now that they're under the scrutiny of the SEC and institutional stockholders.
Having said that, as I learn more about the Breakthrough Therapy Status; if they are able to meet the endpoints in the P2A referenced by Carl LeBel in the Goldman fireside chat, I have a hard time seeing how they wouldn't get it.
Directly from the Goldman discussion, Phase 2A is intended to:
- Recruit a patient population with similar Moderate to Moderately-Severe SNHL (Greater room for % improvement)
- Replicate the same statistically significant & clinically meaningful results with a single dose as the Phase 1/2
- Show outcomes of the additional 2 or 4 doses that are significant.
(Either greater participant response in the same hearing range, or an extended range of hearing improvement deeper into the cochlea)
These three points alone, in my opinion, pretty much meet the Breakthrough Designation guidelines on the FDA website; and they already have meaningful data from the Phase 1/2. Phase 2A is literally enriching the data.
https://www.fda.gov/patients/fast-t...approval-priority-review/breakthrough-therapy
BONUS: Add to that the more recent findings from the Perilymph study and the durability findings from the Phase 1/2 participants that they re-tested after 12-21 months.
With all that known, I suspect Phase 2A will probably be concluded at the end of 2020 due to the COVID-19 delay.
I think the Phase 2A findings will likely make FX-322 eligible for Breakthrough Status.
Based on the chart below as a reference.
I am going to speculate that Frequency conducts the next phase as its Pivotal phase. That Phase (2b/3) is a 12-month study, includes 300-500 participants and is modeled similarly to the Phase 2A. It starts mid-2021 and ends mid-2022. After the study ends, the 6-month NDA process takes place. It is approved for market use at the start of 2023. Patients may be able to gain access to dosing at some point early-mid 2023.
NOTE: There are no hard rules indicating that drug makers have to wait to start the manufacturing process until AFTER the NDA is approved by the FDA. They may very well be able to start the manufacturing process on or before the NDA process to meet expected demand in 2023. Astellas may also start manufacturing drugs for use in countries in EU/Asia that may not be as strict as the US. Also, Frequency has claimed a few times that FX-322 is a small molecule drug, and therefore should be simple to manufacture.
Having said that, as I learn more about the Breakthrough Therapy Status; if they are able to meet the endpoints in the P2A referenced by Carl LeBel in the Goldman fireside chat, I have a hard time seeing how they wouldn't get it.
Directly from the Goldman discussion, Phase 2A is intended to:
- Recruit a patient population with similar Moderate to Moderately-Severe SNHL (Greater room for % improvement)
- Replicate the same statistically significant & clinically meaningful results with a single dose as the Phase 1/2
- Show outcomes of the additional 2 or 4 doses that are significant.
(Either greater participant response in the same hearing range, or an extended range of hearing improvement deeper into the cochlea)
These three points alone, in my opinion, pretty much meet the Breakthrough Designation guidelines on the FDA website; and they already have meaningful data from the Phase 1/2. Phase 2A is literally enriching the data.
https://www.fda.gov/patients/fast-t...approval-priority-review/breakthrough-therapy
BONUS: Add to that the more recent findings from the Perilymph study and the durability findings from the Phase 1/2 participants that they re-tested after 12-21 months.
With all that known, I suspect Phase 2A will probably be concluded at the end of 2020 due to the COVID-19 delay.
I think the Phase 2A findings will likely make FX-322 eligible for Breakthrough Status.
Based on the chart below as a reference.
I am going to speculate that Frequency conducts the next phase as its Pivotal phase. That Phase (2b/3) is a 12-month study, includes 300-500 participants and is modeled similarly to the Phase 2A. It starts mid-2021 and ends mid-2022. After the study ends, the 6-month NDA process takes place. It is approved for market use at the start of 2023. Patients may be able to gain access to dosing at some point early-mid 2023.
NOTE: There are no hard rules indicating that drug makers have to wait to start the manufacturing process until AFTER the NDA is approved by the FDA. They may very well be able to start the manufacturing process on or before the NDA process to meet expected demand in 2023. Astellas may also start manufacturing drugs for use in countries in EU/Asia that may not be as strict as the US. Also, Frequency has claimed a few times that FX-322 is a small molecule drug, and therefore should be simple to manufacture.
- Mar 14, 2020
- 180
- Tinnitus Since
- 12/19
- Cause of Tinnitus
- Noise Exposure + Viral Infection
All things considered, we'd be lucky to see it hit the market in 2023. Here's to hoping for expanded use before that point... It's not only our soonest chance for relief, but is also the only part of this whole thing that doesn't seem insane.
The thing is Frequency's main focus is going to be FDA approval, so even if they can technically open up expanded use, most of their resources are going to be pointed elsewhere.
@FGG has even pointed out that compassionate use could be slowed down as a result of the upcoming trial.
6 months to process new a drug approval (NDA)? After years have already been spent in FDA trials?
I never fail to be astounded with how inefficient the FDA is... meanwhile we are losing individuals to suicide, dementia, debilitating anxiety and other disorders. Though, this process is supposed to protect those same individuals, right?
Sometimes the 'solution' is worse than the problem... sigh.
I'm going to write the FDA and petition my support for the reform that has been mentioned by them before, and emphasize how urgent it is for so many of us. May as well strike while the iron is hot.
- Feb 14, 2020
- 1,630
- Tinnitus Since
- 1-2019
- Cause of Tinnitus
- 20+ Years of Live Music, Motorcycles, and Power Tools
I acknowledge your frustration, but you can also find cases where drugs move through the process quicker than average. Typically it has to do with safety profile. Frequency reiterates two talking points continuously, "favorable safety profile" and "clinically meaningful data."
It seems to me that when the benefit of a drug has to be weighed against some pretty nasty side effects, that is when the process needs much more consideration.
Take for example some of the more recent cancer and MS drugs that have been given breakthrough status. Sure, they slow down or eliminate the progression of the disease, but they interact with a ton of drugs and do things like destroy the patient's immunity for months after treatment. So the FDA requires a ton more data, more trials, a larger more diverse patient population.
FX-322 so far has seen consistent positive outcomes without any major side effects in the short term or long term; otherwise it would have been mentioned in the trial. This is incredibly promising and will make the decision-making process more efficient.
I think after Phase 2a trials we should get a better idea if the 4 doses of FX-322 is safe and efficient. If it meets both safety and efficiency this would give FX-322 a better chance at getting Breakthrough Therapy status but allow the Frequency Therapeutics ease of mind when allowing compassionate use to sufferers like us as they would have more trust in the drug that does not cause serious side effects.
Phase 1 was concluded about 1,5 years ago, no? If so, that's too short of a time frame to analyse long term adverse effects.
- Jan 5, 2020
- 90
- Tinnitus Since
- 2011, Spike 2019, Spike 2020
- Cause of Tinnitus
- 1e Loud Music, 2e earwax irrigation, 3e padel tennis
For me it's simple. At this very moment in time only FX-322 (not mentioning other things because they still take 5-10 years) seem to have a possibility of lowering tinnitus. So why wait 3 years for maybe compassionate use? It's not like anything else is going to solve anything for us in the near future. An alternative medicine may be available in 10 years. I would like to take my chances now, instead of this useless waiting game. It makes no sense, I would like to roll the dice.
- Mar 14, 2020
- 180
- Tinnitus Since
- 12/19
- Cause of Tinnitus
- Noise Exposure + Viral Infection
Yeah, I know it could definitely be worse and genuinely appreciate your reply.
This drug, and other upcoming drugs are extremely promising. They could not have existed at all or been delayed that much further. I don't want to come off as ungrateful for the possibilities here.
It simply angers me that we have to wait years to have chance to try those possibilities. You've been far more than objective than I (I'm pretty much kicking and screaming over here). I'll just vent below.
At a certain point, for many individuals, it makes no difference if there is a risk of side effects or negative reactions to other drugs/conditions. Many have and will commit suicide before FX-322 sees the light of day. At the very least, they (us) will lose years of quality from their lives.
Despite this, the system is set up to incentivize conditions where no one can access these drugs until half a decade (at the least) and tens of millions of dollars are poured into trials. This is deemed to be in the public's best interest, but what about the public that may be gone tomorrow?
Though expanded use does exist, it doesn't seem like it's the priority - getting the FDA's approval to go onto the market is.
From the way things are set up, this is understandable from the drug company's point of view: why open expanded use immediately when you can prioritize finishing trials and make more profit in a few years?
However we have the internet and we also have competent healthcare providers. We could leverage this in better ways. It should be simpler for the healthcare providers & patients to make a judgement call when they are desperate and buy drugs directly.
There is no legal remedy here; even expanded use is riddled with red tape.
Any company that isn't drug related does it's own internal testing for a product and, in most cases, releases new products annually.
They are kept in check with fraud laws, misadvertising laws, malpractice laws, malicious intent laws, etc.
I fail to see why drug companies should be different. Honestly it seems like fear mongering ("without the FDA everyone would be given medicine that gives them cancer!") is the only reason we allow this to happen.
What's to keep a toy company from using lead paint? Or food from the local farmers market (or even at Walmart) being poisoned? Even approved FDA drugs still ruin many people's lives. You cannot account for every variable.
When shit does happen, the nation is alerted quickly thanks to modern communication, and (the vast majority of the time) the product is pulled.
I'm sick of hearing that lawmakers know what's better for someone's life than they do. Vet the drugs, but in the meantime let the drug go onto the market labeled as non-approved. Let healthcare providers buy them for the patients who may not last until vetting is done.
Anyways I'll stop here. I'll draft that letter sometime soon (the tone will be less vicious towards the system and more friendly) and maybe post it on this forum for review & anyone who wants to add their name.
- Mar 14, 2020
- 180
- Tinnitus Since
- 12/19
- Cause of Tinnitus
- Noise Exposure + Viral Infection
You can't fully analyze long term effects unless you watch trial patients until they die.
That's sort of my point, we're always going to be taking a certain amount of risk regardless of how policies are set up.
Koz
Member
- Dec 15, 2013
- 153
- Tinnitus Since
- 2009
- Cause of Tinnitus
- Unilateral sensorineural hearing loss
With all due respect and I know this isn't a competition as we're all on the same boat in shit-creek together, despite some of us handling it better than others, its a very not ideal situation either way but...
2023 is not a long way away when you consider how long some of us that have had this (if not worse than yours) for longer. Let's hope it comes sooner either way and 2023 is relatively quick compared to 5+ years.
In the meantime protect your ear(s) as best you could and focus on your life as much as you are able to one day at a time. I wish you all the best.
Me too. Why wait if there is something for us to take to allow us to get back to our daily lives.
I'm tired of playing the waiting game. The longer I suffer the chances of being able to build a career with my University degree diminishes.
The people at the FDA want to be sure they thoroughly exhaust the certification process before granting. Their primary concern is covering themselves. So, paperwork, red tape, and the years go by.. Same thing with NASA. However, SpaceX found a way around it. What we need is a SpaceX approach to the FDA, if the government would allow it.
In my version of an ideal world everyone would do their own research, determine their own risk, and be able to decide for themselves what drugs they want to take. The problem is people are dumb, they want the government to control their lives for them, and the government will gladly step in to do it.
At least that's my take on it all. Your opinion may differ, and that's OK with me.
In my version of an ideal world everyone would do their own research, determine their own risk, and be able to decide for themselves what drugs they want to take. The problem is people are dumb, they want the government to control their lives for them, and the government will gladly step in to do it.
At least that's my take on it all. Your opinion may differ, and that's OK with me.
I think none have been reported so far. On the other hand FX-322 was given in phase 1 to only 15 persons. So we can't really know what side effects it has. But since its applied locally to the ear I would guess that risk of systemic side effects are much more reduced. And if this is safer than stem cell therapy and actually works, I don't really understand why it does not has breakthrough status. If I had a few million over I would give it to them to see how FX-322 works for 100 tinnitus & hyperacusis sufferers.
I fully agree. Hearing restoration is one of the holy grails in medicine. We might see the first step in just a few years. People before us unfortunately couldn't say the same thing.
Having said that, I'm preparing for only a moderate effect on my hearing loss from this drug. "Disease modification" is what Frequency calls it now. I have a 70 dB PTA on 250-8000 Hz. If i can get 30+ dB back on some of the higher frequencies and up my word recognition, I'll be happy camper and will just wait for the next generation of treatments. Ultimately I don't think my ear will ever be the same.
Money!!!
If Trump does cut down on FDA regulations which results in the drug getting to market sooner, my dislike for him will be significantly diluted.
- Mar 14, 2020
- 180
- Tinnitus Since
- 12/19
- Cause of Tinnitus
- Noise Exposure + Viral Infection
I agree 1000%. We could potentially all have had restored hearing by now if not for the behemoth time & resources that go into getting drugs approved.
However it's hard for me to imagine a 'Space X' approach to the FDA. Any actual ideas on what this would look like?
It's very illegal for two consenting parties to have a drug transaction unless the FDA approves it. Personally I think the FDA should be looked at as a guideline, not a draconian-style lockdown on what adults can & can't do...
- Mar 14, 2020
- 180
- Tinnitus Since
- 12/19
- Cause of Tinnitus
- Noise Exposure + Viral Infection
If there were some unforeseen side effect for the COVID-19 vaccine, millions in the country could suffer from it. However the Government more or less decides that we should have the freedom to take that risk because COVID-19 is detrimental and makes the vaccine available asap.
They'll also pour lots of resources into that year to get as much testing done as possible... but let's be realistic, it's 1 year as opposed to the 5-10 most drugs see. They are bending rules due to urgency.
Less than %10 of the population has tinnitus, and a fraction of that would get FX-322 right away. However it is extremely detrimental (even fatal) to many of our lives. Being in the minority however, there is no such expediency.
This is why I value individual rights over majority rules. If you are not in the majority, you often get screwed.
- Feb 14, 2020
- 1,630
- Tinnitus Since
- 1-2019
- Cause of Tinnitus
- 20+ Years of Live Music, Motorcycles, and Power Tools
Now that you mention this, and considering Frequency has now revealed they they have done two "extra curricular" studies outside of the Phase 2A; meaning the perilymph and the "durability" study on Phase 1/2 patients. It would not surprise me if they don't have other things in the works to coincide with the Phase 2A results.
- Jan 5, 2020
- 90
- Tinnitus Since
- 2011, Spike 2019, Spike 2020
- Cause of Tinnitus
- 1e Loud Music, 2e earwax irrigation, 3e padel tennis
It's up to Frequency to allow expanded access. Unfortunately, they said they are not ready to do so at this time.
- Feb 14, 2020
- 1,630
- Tinnitus Since
- 1-2019
- Cause of Tinnitus
- 20+ Years of Live Music, Motorcycles, and Power Tools
I would say that Frequency is doing a good job so far generating interest/awareness and increasing speculative demand. Outside of regularly meeting with the FDA as part of the Fast Track program, they're continuing to be visible at relevant investor conferences, medical conferences, and on social media. So, it's safe to say they're making the case that there is a business need from several different perspectives. In this month alone, the will have done 3 conferences already, and it seems their Linked-In and Twitter followings are growing a steady rate as well.
On the Goldman fireside chat, one of the execs at Frequency reminded the interviewer that the FDA is well aware of the enthusiasm amongst the clinical community for FX-322 as well as advocacy groups. Also, as mentioned a few months back, Carl LeBel from Frequency is expected to attend an interview on the Tinnitus Talk Podcast. So, all of these activities will continue to show cause to move things along for the FDA.
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