Frequency Therapeutics — Hearing Loss Regeneration

What I'm curious about regarding the below response is:

1.) Were phase 1 and 1/2 that are completed considered 2 of 3 phases?
2.) Phase 2a is starting this year. Does that mean there will be multiple studies in Phase 2? Like 2b, 2c, etc.
3.) If Phase 2 and all of its incarnations are successful, will they need to do an official phase 3? Some people in this thread say you are required to have an official phase 3 by the FDA. I wonder if it has more to do with number of test subjects rather than the actual phase denotation. Would a 2a or 2b be considered a third phase?

 

I believe 2a is to ensure that phase 2 is properly differentiated from phase 1/2. I think we have good reason to hope that there is only phase 2a within phase 2; this is not an industry where you can retry a drug till it works! I would expect there to be a phase 3, most likely in part at least to reduce the stringency regarding trial participants and thus gather data on how well the drug works vs more mild hearing loss. I would expect varying dosages to be trialled in phase 2a.

This follows an engineers view of test procedures that ramp up complexity progressively and manage trial risk.

My expectations only.

 

I think it's just split in two parts for phase 2 for the simple reason that phase 1 was done on 1 person or a cochlear outside of the human body.

Phase 2 (a) was one dose on a small sample of people approx 18 or whatever it was.

Phase 2 (b) will be multiple doses to a small sample of people.

Phase 3 will require lots of funding and investment and will push this puppy into a mass trial. When Phase 3 happens it's going to take something catastrophic to go wrong for it to not hit the market really. People will lose lots of money if this happens so they need to make sure everything is going to be successful for a phase 3 trial.

They need as much data as possible in the meantime without taking stupid risks.

 

Untrue. 40% of phase 3 trials fail.

 

True, if I've read this correctly. But it depends on the kind of medicine.

 

Thank you. Mine is viral. I have viral sudden sensorineural hearing loss. It's severe and I cannot use hearing aids because all sounds are distorted. It would be good if I could use hearing aids, I do not expect full recovery.

 

That’s true.

I think that’s the reason Frequency Therapeutics is making phase 2 as exhaustive as possible.

Surprises in phase 2 allow you to readjust and try different things.

Surprises in an expensive phase 3 can bury a cure.

I love phase 2. Let them tinker, try stuff, take number of consecutive injections to the max... let them do it right... so that phase 3 can be a breeze... a formality without nasty surprises.

A victory lap.

My two cents...

 

Hey Piney,
It's not a long shot, I'm convinced that's how it's going to go down. You need that recommendation, get the VA audiologist on it, I think that's the protocol...

Good luck...

 

A fight that the tinnitus community may have to face is the price. If insurance companies don't deem your tinnitus to be clinically significant, or if you have hidden hearing loss and a normal audiogram, they are likely going to fight to pay for this. Lucchino did also mention something about "commanding a premium".

 

What does commanding a premium mean. A cost that will not change? Or a cost of a product where you can reap full benefits?

 

Sad but maybe true!

Depending on how much the FrequencyTx treatment will cost, insurance companies will fight.

However if you bring up a psychological attest declaring that "you suffer" they probably won't be able to refuse payment but will cancel your insurance at next possible date.

Would be great if tinnitus assesment and - even better - diagnostic tools improved in the next years.

For example:

You hear a high pitch ringing of 10 kHz.

Audiometry shows you start hearing 10 kHz sounds at a level of 60 dB and your tinnitus matches exactly at 10 kHz at 70 dB.

Your audiologist explains that your tinnitus is only as loud as 10 dB. Rain and waving leafs are far louder. A psychologist then explains to your insurance company: "hey guys, if you don't pay, I d like you all to wear earbuds with a 10 kHz sound at 70 dB for a year, your brains are going to habituate, no big deal, life is wonderful, a little bit of tinnitus doesn't kill anyone"

Besides: If hair cell regeneration works out good/perfect and it stops tinnitus or brings it on a level where you must concentrate to hear it, they for sure want to make money with it!

It won't be as cheap as nosedrops, but similar to standard eye/laser operations.

Let's hope the race for the cure goes on, and that there are also other players (that we don't know about) out there on the field.

 

When he says commands a premium, he may be referring to valuation of the company, not necessarily premium of treatment. My guess is though yes if successful they will get a premium for the treatment. Two times Hearing Aids might be the floor. If the treatment is too expensive and insurance does not cover, they will not get the patient participation. 500 Million hard of hearing times say $10,000 USD is a huge revenue number.

 

Well, not 100% accurate or relevant for this study.

The statistic quoted here considers 38 studies, of which 18 (almost 50%) are oncology studies where efficiency is not met. Most cancers have at least a type of cure and most of these studies prove they are not as efficient as the one(s) that currently exist. For other diseases as well (cardio etc) there are some kind of cures, more or less effective.

In the breakthrough category (for which no cure exists), like this one, normally if there are no (big) side effects, the cure should be approvable.

Sure, I can understand they are trying to lower our expectations with theses type of statistics, but just putting some numbers without proper context is just wrong.

My 2 cents.

 

Why the hell does someone put money into insurance companies, the whole industry is literally a fraud.

Premium is when a brand can charge higher price than competitors for the same product.

 

The survival rate for all cancers is 66%. 16% of all cancers are metastatic (Source CEBP / Lois B. Travis). Mortality rates for metastatic cancer is almost 100%. (My 2 cents.)

 

I am going to assume he meant 'type of treatment'. Either way, that modification stands up to better scrutiny and the base point still holds.

 

Any theories on if this technology is likely to help in hearing loss caused by Meniere's disease? Hair cell loss happens but I'm wondering how much, if any, hydrops could be a factor in how well the drug can interact with progenitor cells.

 

It’s a complex disease. There are people with no hearing disorders and some are almost deaf. Some have severe vertigo issues and some have almost no issues.

Hopefully this can help at least some people if not everyone. I’m one of them and are keeping my fingers crossed. Hydrops will probably also vary in patients.

 

If everything goes well, can this drug be available to the market by 2020/2021?

 

I think that because this is such a new realm of medicine, and they haven't figured out the dosing schedule AND each FDA trial is so slow, that it could, despite my earlier beliefs, take several more years.

 

It can only help hearing loss via ear drum injections because the cochlea has a permeable membrane called the round window. Will McLean stated somewhere that the drug actually does a better job regrowing vestibular haircells in vitro but how would you administer it to the vestibule?

 

I believe even if everything goes "sooper dooper" that not before 2023.

 

Audion's treatment could be available by then. European system will allow for a much faster arrival of the drug on the market than the American one.

 

Not just Audion. If Frequency Therapeutics decided to launch from Europe, this could be out a year earlier than in the US. Unfortunately, they won't. Europe's drug approval process seems to be a little more streamlined than the FDA for some reason.

 

Which is why it's so important to get into the next trial. That way, you won't have to wait that long.

 

How do you do that?

 

You more than likely have to be an American, or at least be able to stay during the trial duration.

 

But why the heck did they not do that then?? Aren't they run as a business or what?? They should be very aware that whichever company reaches the market first will grab the most profits and command premium as it is the one that's made the most people hear. So the time is everything with regards to their profitability so why didn't they go to Europe or China? Their fault.

 

Anyone know when the next trial starts for Frequency Therapeutics?

 

I was referring to the hearing loss, although would love to get my balance back, too (lost fully on one side, now starting to affect the other). Would this drug navigate the round window properly and, if so, would you suppose hydrops would interfere with effectiveness?

I guess the only option for the vestibule would be surgical application. That obviously seems much trickier but would be worth it if effective.