Frequency Therapeutics — Hearing Loss Regeneration

It was live but they may archive it later like they did with the JP Morgan one for a while.

 

If you go to ‘Presentations’ on Frequency's website and then to the webinar, it says that they will upload it later (the conference organization, not Frequency itself).

 

I am desperate for tinnitus relief and I'm sure other people are too! Do you think there's any way we could organize a group of individuals to convince Frequency Therapeutics/FDA to allow us to take FX-322 under "compassionate use" before it is released to the market?

 

It's available here: https://investors.frequencytx.com/

 

It's possible but we should at least wait for the results of phase 2a (unfortunately).

 

Compassionate use is not up to the FDA. It's solely up to the company. On their website they said they are waiting until they can produce enough to meet demand and implied they are also waiting for further testing to show what dosage they would need.

Personally, if you can convince a philanthropy organization to fund their production ramp up, that might be the only thing that would further it along quicker.

 

Is anybody here friends with Bill Gates?

 

Maybe we should contact the Gates Foundation. One of its goals is to enhance health care.

 

In Europe at least the national authority needs to approve compassionate use of the drug. Companies can’t go out giving people unapproved drugs without government permission.

 

Sorry to hear this, like you say it sounds awful. Not sure how I'd cope in that situation. Masking is the only way for me to escape it. Guess in that sense I feel lucky. It does seem like some form of hyperacusis as you said. Hope it gets better and/or you find a way to deal with it. I mainly posted because once I found effective masking techniques my life became so much better.

 

In the US, they can for conditions that greatly affect quality of life for which there are no good treatment options. Your doctor can apply for it for you if you fit the bill but the company has to approve it.

 

Yes, but the FDA has to approve it as well. Companies can’t go handing out unapproved drugs to patients without the FDA approving it.

https://www.fda.gov/news-events/expanded-access/expanded-access-information-patients#fda-consider

 

From what I understand, all diseases with high morbidity are approved. The FDA is not the road block here. High morbidity and no available treatment is the reason it got Breakthrough status in the first place.

 

https://theconferenceforum.org/wp-content/uploads/2020/05/CMO_360_newsletter.pdf

 

Or Bill Clinton. He has hearing loss from guns so you'd think he'd be all over this.

 

We’ll have to wait and see how the FDA will look at this since there aren’t any approved hearing loss drugs on the market. My understanding is that authorities like the FDA are careful when considering compassionate use. And for hearing loss at least there’s the, albeit unattractive, alternatives of hearing aids or cochlear implants that the FDA will consider when making a decision.

 

That's true but I also think from the way that Frequency's worded things they are very much framing their drug as meeting an unmet need, e.g. the emphasis on clarity when hearing aids are amplification devices. Cochlear implant surgery is also not really a viable alternative if you don't have severe loss IMO, it's very expensive and of course irreparably damages the cochlea and in no way restores natural hearing function.

 

With regard to cochlear implants that’s the catch-22 isn’t it? If your hearing loss is severe/profound, which might be an illness for which compassionate use is meant, you can get a CI. If you have mild hearing loss it’s probably not considered for compassionate use in the first place. And you could still get hearing aids for that.

All in all I think everyone’s best bet is to get into a trial. If you’re on this forum, you’re probably among the best informed patients. Use the information to get into a trial. If you’re denied because of not enough hearing loss, I don’t see why the FDA would allow you to get the drug with compassionate use.

 

Presentation is online.

http://wsw.com/webcast/jeff126/freq/

 

Hearing aids are not the same as a Therapeutic Treatment. Hearing aides are a prosthetic device. A Breakthrough Therapy Designation is often given to Therapeutic Treatments for diseases where none currently exists.

There is no therapeutic treatment for Sensorineural Hearing Loss. End of story.

Only from a commercial standpoint would Hearing Aides or CIs be considered a marketing alternative/competitor for FX-322. But, I would argue not from a strictly medical standpoint; which I hope is the lens that FDA would view the treatment from.

 

Breakthrough Status means the FDA feels this is a serious condition with an unmet need. Expanded Use is specifically for those conditions. The only reason they would deny it is safety concerns but the FDA has a history of allowing even unsafe last ditch cancer drugs to be used under the law.

There is a reason Frequency worded their Expanded Use policy on their website the way they did. They know it's up to them.

 

FX-322 hasn’t gotten Breakthrough Status. Frequency probably has their policy listed because they also have to approve compassionate use. It does not mean the FDA will just write a blanc cheque should Frequency open up access to their drug. You also can’t compare cancer drugs to hearing loss drugs.

 

It doesn't have to be a cancer drug. It does not even have to be life threatening, it just has to be a drug with high quality of life implications for which people don't qualify for a clinical trial. The only reason it would have been declined is safety issues of which there weren't any.

That being said, cancer drugs are certainly easier because your doctor can just say "they have cancer" where with this, your doctor has to describe in detail the poor quality of life you have and why you need the drug. I think this will be easier for tinnitus after phase 2a results support its use for that.

 

Agreed. Here's some info from the FDA website...

Requirements to qualify for expanded use:

• Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.

• There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.

• Patient enrollment in a clinical trial is not possible.

• Potential patient benefit justifies the potential risks of treatment.

• Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

What the process actually looks like:

1. Patient
   Consults with licensed physician to explore and decide about alternative options.
2. Licensed Physician
   Agrees to oversee the patient's treatment and works with industry (e.g., medical product developer), files paperwork with FDA and IRB (for many expanded access request types), and is responsible for patient care and reporting.
3. Industry (Company)
   Willing to provide the investigational medical product and either submits the expanded access request to FDA, allows the FDA to cross-reference their IND (for drugs and biologics) or IDE (medical devices) on behalf of the expanded access sponsor-investigator through the use of a letter of authorization, or provides the necessary investigational medical product information for the sponsor-investigator to submit to support an expanded access request.
4. Investigational Review Board (IRB)
   Reviews expanded access protocol and consent to ensure that the patient is informed about the nature of the treatment.
5. FDA
   Reviews the expanded access request and determines if the treatment may proceed. (I read on another page that this takes 30 days after the FDA receives the request).

There is no special policy for cancer as far as I can tell.

The process can be accelerated (immediate FDA approval via phonecall) if the situaton is an emergency, however. See the link below for additonal info regarding emergency requests and more.

https://www.fda.gov/drugs/investiga...gle-patient-expanded-access-compassionate-use

Really it'd be nice if we could confirm with Frequency that they plan on ramping up production & opening expanded use if phase 2a is promising. Maybe we really could help somehow.

Otherwise I guess I'm hoping that phase 3 will have way more lax requirements to be accepted as a patient...

 

If that happens, it's probably a good idea to gather some evidence that you've tried other treatments to no avail, or you might be excluded.

I'm not going to argue tinnitus can be life-threatening, but the thing is, how does one prove it to the outside world and thus he or she should be entitled to compassionate use?

 

This is the thing I am most concerned about - would you have had to exhausted all other treatment options to be permitted compassionate use? Say if we discover that FX-322 helps hyperacusis patients, for example, would they have had to have tried out sound therapy (which some people may be reluctant about understandably in case of worsening)?

 

@Bartoli

If in the Phase 2A, they are able to draw a significant correlation between the lessening of a participant’s TFI scores and improvement in thresholds at the treated frequencies, that should be enough.

A physician would need to indicate that a patient complains of debilitating tinnitus and has poor hearing thresholds that FX-322 is known to treat.

 

And this good news, even though COVID-19 has delayed things, in the long run it could accelerate the FDA process:

https://www.fiercebiotech.com/biote...lerating-covid-19-measures-a-top-fda-priority

 

I was thinking about this too. I assume your doctor could say along the lines that you have tried sound therapy through the use of speakers but unfortunately it made the patient worse because it’s similar to hearing devices. If you can’t cope with speakers then you can’t cope with hearing devices.

 

I listened to their presentation. Nothing new as far as I can tell. They are still recruiting for Phase IIa so that means, as has been pointed out, they’re not going to release results in the second half of 2020 since they are following patients for 9 months. Mid 2021 is more likely.

They also stated their financial position is looking good with funding up until at least 2022 and mentioned that there are payment milestones with Astellas if they conduct trials in Europe or Asia. If Phase IIa goes well I think we will see a Phase IIb trial in Europe.

Listening to their talk it’s clear to me again they are settling for high frequency hearing loss (for now at least) which is understandable considering Phase 1/2 results. Sucks for people with Meniere’s and people with hearing loss across the board though.