Frequency Therapeutics — Hearing Loss Regeneration

[tommyd87]

I thought it meant 1 shot a week for 4 weeks.

It does. It doesn't mean that they all got a shot of FX-322 per week though.

 

[tommyd87]

That's correct. I hope that by the end of March we will get good news that additional doses of FX-322 help with restoring hair cells.

I'm hoping we will get positive results too.

Something seems strange about why they want to release results right after 90 days though. This makes me mostly cautiously optimistic that the trial outcomes are positive.

I'm pretty sure Frequency Therapeutics wouldn't be doing this if they didn't see benefit.

 

[Lucifer]

I'm hoping we will get positive results too.

Something seems strange about why they want to release results right after 90 days though. This makes me mostly cautiously optimistic that the trial outcomes are positive.

I'm pretty sure Frequency Therapeutics wouldn't be doing this if they didn't see benefit.

My thoughts exactly. Why would they release results up to Day 90 earlier than wait till end of May to release results up to Day 210.

To me it seems it must be good news if they want to release results earlier. But I hope I'm not wrong though.

 

[frpp]

I'm praying this thing works. If FX-322 cures my tinnitus, relieves my hyperacusis, and gives me back my hearing it'll be that happiest day of my life. I will literally run the whole length of Broadway Manhattan buck naked on that day and the arrest ticket will be well worth it.

 

[Lucifer]

I'm praying this thing works. If FX-322 cures my tinnitus, relieves my hyperacusis, and gives me back my hearing it'll be that happiest day of my life. I will literally run the whole length of Broadway Manhattan buck naked on that day and the arrest ticket will be well worth it.

Me too. None of us deserve to have these conditions. I hope my hyperacusis and tinnitus goes away once I get FX-322. I hope the wait will be worth it in the end. We will have to wait and see.

 

[weab00]

I'm praying this thing works. If FX-322 cures my tinnitus, relieves my hyperacusis, and gives me back my hearing it'll be that happiest day of my life. I will literally run the whole length of Broadway Manhattan buck naked on that day and the arrest ticket will be well worth it.

I'm 100% with you, hyperacusis and tinnitus have torn all of our lives apart. But I would guess that the effects would be gradual.

 

[Billy_Shears]

I'm praying this thing works. If FX-322 cures my tinnitus, relieves my hyperacusis, and gives me back my hearing it'll be that happiest day of my life. I will literally run the whole length of Broadway Manhattan buck naked on that day and the arrest ticket will be well worth it.

It's A Wonderful Life... as George Bailey was just as happy at the end of this Christmas Classic movie. It is all in the hands of those brilliant minds in Woburn, MA. I hope so much your prayers and those of others come true!

 

[Philip83]

I know the discussion might have happened in this thread before, but I can't find it at the moment.

As soon as FX-322 is offered to the public in the US, can foreigners book appointments at US clinics to get treated? I guess if it's private doctors, it should be possible?

 

[ajc]

As soon as FX-322 is offered to the public in the US, can foreigners book appointments at US clinics to get treated? I guess if it's private doctors, it should be possible?

@FGG has already spoken about this. Yes yes yes, it is possible to see a doctor privately in the US and have the treatment done once it's available. Make sure to save enough cash, could be veeeeeeeryyyyyyy expensive!

 

[Philip83]

@FGG has already spoken about this. Yes yes yes, it is possible to see a doctor privately in the US and have the treatment done once it's available. Make sure to save enough cash, could be veeeeeeeryyyyyyy expensive!

Any guesstimates? If the results are more or less guaranteed to be good, I'd take out a loan if I have to.

Another thing is that the Swedish government actually will sometimes sponsor you to go to another country to get treated if a specific treatment can't be provided in Sweden. Hopefully this would be covered!

 

[L34]

And what about if there is going to be opportunity for compassionate use/right to try, will us foreigners also be allowed? I am from Europe...

 

[tommyd87]

I know the discussion might have happened in this thread before, but I can't find it at the moment.

As soon as FX-322 is offered to the public in the US, can foreigners book appointments at US clinics to get treated? I guess if it's private doctors, it should be possible?

You mightn't even have to do that. The thing is if FX-322 is successful in the current trials then I cannot see Frequency Therapeutics trying to delay its progression at all and so it might be released internationally by the time you can get it in the US.

My thoughts exactly. Why would they release results up to Day 90 earlier than wait till end of May to release results up to Day 210.

To me it seems it must be good news if they want to release results earlier. But I hope I'm not wrong though.

I think Frequency Therapeutics would be very brave to release any actually adverse results early when we wholly consider where they are at as an organisation.

 

[Christiaan]

And what about if there is going to be opportunity for compassionate use/right to try, will us foreigners also be allowed? I am from Europe...

I don't know if it's helpful to mention this, but you could also wait for treatment in Europe. The process works twofold:

The first condition concerns EMA (European's FDA): According to their guidelines, compassionate use is allowed if the treatment is in clinical test phase + that the said treatment has a chance to help a patient who has a debilitating or life threatening illness. So that may not be a big problem, as FX-322 will be tested in Europe in the nearby future.

The second condition is Astellas (who has bought the licence for future commercialisation of FX-322 in Asia & Europe): are they willing to offer compassionate use to those who are in dire need of a fix? So basically, it's really up to them to fill in a request at the EMA for compassionate use if they want to help us ASAP.

 

[ajc]

As @Diesel already posted before, compassionate use is not going to happen. Too many drawbacks to it from a commercial point of view. Too many people would like to get it.

Frequency Therapeutics will rather focus their efforts on full-blown commercialization than offering it via compassionate use. Mark my words, people.

 

[L34]

You mightn't even have to do that. The thing is if FX-322 is successful in the current trials then I cannot see Frequency Therapeutics trying to delay its progression at all and so it might be released internationally by the time you can get it in the US.

My concerns are just...

It might not be Frequency Therapeutics that will delay the opportunities for us in Europe. It might be the European Union that wants to make an agreement in all the European countries before launching FX-322. It is a bit difficult for me to describe it exactly in English, but other members in here from the EU will know what I mean. It can take YEARS to get anything through the EU. And that is a big concern of mine... fingers crossed...

 

[FGG]

As @Diesel already posted before, compassionate use is not going to happen. Too many drawbacks to it from a commercial point of view. Too many people would like to get it.

Frequency Therapeutics will rather focus their efforts on full-blown commercialization than offering it via compassionate use. Mark my words, people.

That's not really what he said if I recall correctly. He said that (as I have also speculated), that they might wait until recruitment for the next trial is finished first to ensure full enrollment.

There aren't commercial drawbacks or delays at that point.

It's all speculation though.

 

[L34]

I don't know if it's helpful to mention this, but you could also wait for treatment in Europe. The process works twofold:

The first condition concerns EMA (European's FDA): According to their guidelines, compassionate use is allowed if the treatment is in clinical test phase + that the said treatment has a chance to help a patient who has a debilitating or life threatening illness. So that may not be a big problem, as FX-322 will be tested in Europe in the nearby future.

The second condition is Astellas (who has bought the licence for future commercialisation of FX-322 in Asia & Europe): are they willing to offer compassionate use to those who are in dire need of a fix? So basically, it's really up to them to fill in a request at the EMA for compassionate use if they want to help us ASAP.

Oh, our posts just crossed each other

Thanks for your answer

 

[Diesel]

That's not really what he said if I recall correctly. He said that (as I have also speculated), that they might wait until recruitment for the next trial is finished first to ensure full enrollment.

There aren't commercial drawbacks or delays at that point.

It's all speculation though.

Agree. My position has been that if they have finite resources to commit to compassionate use or a pivotal trial, it's going to be better for business to get that final trial enrolled. At the speed they're planning to go in 2021, I see them going pivotal in 2022.

If the pivotal trial isn't some multi-year trial, since benefits of the drug appear after 90 days, why would they invest in a short 1-2 year period for compassionate use if they can just start making the product for commercial use?

 

[tommyd87]

My concerns are just...

It might not be Frequency Therapeutics that will delay the opportunities for us in Europe. It might be the European Union that wants to make an agreement in all the European countries before launching FX-322. It is a bit difficult for me to describe it exactly in English, but other members in here from the EU will know what I mean. It can take YEARS to get anything through the EU. And that is a big concern of mine... fingers crossed...

I think that this is the reason why Frequency Therapeutics might have planned out their final trials to take place in Europe because essentially they can get the trials needed to complete the FDA process done there, and getting FX-322 recognised in the EU (as the trials will have been conducted there), while also actually having the option to allow compassionate use in the US if they choose to do so.

One promising thing for Europe is that there is now an agreement between the EU regulators and FDA to recognise trials that have been conducted in each other's jurisdictions. This surely shall assist the progression and release of FX-322.

 

[Street Novelist]

I was under the impression the next trial, Phase 3, would be next year, presumably in the fall. Would we really have to wait until 2022?

 

[tommyd87]

Agree. My position has been that if they have finite resources to commit to compassionate use or a pivotal trial, it's going to be better for business to get that final trial enrolled. At the speed they're planning to go in 2021, I see them going pivotal in 2022.

If the pivotal trial isn't some multi-year trial, since benefits of the drug appear after 90 days, why would they invest in a short 1-2 year period for compassionate use if they can just start making the product for commercial use?

Frequency Therapeutics might offer compassionate use themselves because they own the rights to FX-322 in the US. Consequently Frequency Therapeutics might decide to start selling FX-322 while the trials are ongoing to benefit both those wishing to get FX-322 and the organisation.

Obviously I am not sure how complex the process of offering compassionate use in the US is, however if this can be largely handled and instigated by a single staffer then it will be easy and cost effective to get this done. This is especially the case when you weigh the costs of compassionate use against the funds attained from the FX-322 sales and I think the revenue obtained will be far greater.

 

[Born To Slay]

Any guesstimates? If the results are more or less guaranteed to be good, I'd take out a loan if I have to.

Another thing is that the Swedish government actually will sometimes sponsor you to go to another country to get treated if a specific treatment can't be provided in Sweden. Hopefully this would be covered!

It'd definitely be in the thousands. An ENT appointment without insurance alone is about $300 and you'd need multiple appointments, so I'd plan on spending at least a grand for 3-4 appointments. and if each shot cost a thousand dollars and you end up needing 4 in each ear, that could be 8 grand. So I'd say at least 9 thousand dollars without travel expenses.

That's really cool that Sweden does that, I bet they'd cover this. It is essential.

 

[Christiaan]

My concerns are just...

It might not be Frequency Therapeutics that will delay the opportunities for us in Europe. It might be the European Union that wants to make an agreement in all the European countries before launching FX-322. It is a bit difficult for me to describe it exactly in English, but other members in here from the EU will know what I mean. It can take YEARS to get anything through the EU. And that is a big concern of mine... fingers crossed...

Yeah, there are two ways of getting authorisation for a medicine in EU:

1. Centralised procedure; via EMA, which results in single marketing authorisation valid throughout the EU;

2. National authorisation procedure; each individual EU member state authorise medicine for use in their own territory

I think it's more likely that Astellas will opt for centralised procedure via EMA for obvious reasons. One big reason is that they can provide a treatment to almost an entire continent (= making more bucks) after approval if they play by EMA's book. If they go through the national authorisation procedure, the treatment might easily get approved in Belgium (for example) due to more favourable or lower standards of clinical testing, but that doesn't mean that it automatically gets approved in other countries.

Unfortunately, we do not know yet where Astellas will open their clinics for the upcoming FX-322 trial. I assume that they will open a couple of sites in different countries in order to seek approval via centralised procedure + to get as many people as they possible can for their research of this treatment. It may seem likely that they will open in countries that have favourable clinical testing standards. Generally, Belgium and Germany are one of those countries that have the best ''climate'' for clinical trials, from what I gather.

 

[Philip83]

as FX-322 will be tested in Europe in the nearby future.

Is this official?

 

[Diesel]

Frequency Therapeutics might offer compassionate use themselves because they own the rights to FX-322 in the US. Consequently Frequency Therapeutics might decide to start selling FX-322 while the trials are ongoing to benefit both those wishing to get FX-322 and the organisation.

Obviously I am not sure how complex the process of offering compassionate use in the US is, however if this can be largely handled and instigated by a single staffer then it will be easy and cost effective to get this done. This is especially the case when you weigh the costs of compassionate use against the funds attained from the FX-322 sales and I think the revenue obtained will be far greater.

Setting up contract drug manufacturing is COMPLEX. It won't be a single staffer, it will be a coordinated operation, even for compassionate use.

They'd still need setup contracts, supply chain, quality assurance, administrative, distribution, communication processes to essentially provide a short-run of the drug for a limited number of patients for a short period of time while they wait for the pivotal phase and FDA approval to go commercial. This is a HUGE investment. So, why not just invest all those same people, resources, processes in ensuring the unknown US drug maker and Astellas can pump out massive quantities of the drug worldwide as soon as the pivotal phase ends?

This doesn't even include dealing with the demand from millions of patients and doctors that will overwhelm Frequency Therapeutics with requests for compassionate use.

So, after all that; even if it does happen, the cost to the patient to finally get the drug might be substantially higher than getting the mass-produced product; let alone the wait time to get through the administrative process.

 

[Philip83]

It'd definitely be in the thousands. An ENT appointment without insurance alone is about $300 and you'd need multiple appointments, so I'd plan on spending at least a grand for 3-4 appointments. and if each shot cost a thousand dollars and you end up needing 4 in each ear, that could be 8 grand. So I'd say at least 9 thousand dollars without travel expenses.

That's really cool that Sweden does that, I bet they'd cover this. It is essential.

Cool, thanks for the estimate.

 

[serendipity1996]

Yeah, there are two ways of getting authorisation for a medicine in EU:

1. Centralised procedure; via EMA, which results in single marketing authorisation valid throughout the EU;

2. National authorisation procedure; each individual EU member state authorise medicine for use in their own territory

I think it's more likely that Astellas will opt for centralised procedure for obvious reasons. One big reason is that they can provide a treatment to almost an entire continent (= making more bucks) after approval if they play by EMA's book. If they go through the national authorisation procedure, the treatment might easily get approved in Belgium (for example) due to more favourable or lower standards of clinical testing, but that doesn't mean that it automatically get approved in other countries.

Unfortunately, we do not know yet where Astellas will open their clinics for the upcoming FX-322 trial. I assume that they will open a couple of sites in different countries in order to seek approval via centralised procedure + to get as many people as they possible can for their research of this treatment. It may seem likely that they will open in countries that have favourable clinical testing standards. Generally, Belgium and Germany are one of those countries that have the best ''climate'' for clinical trials, from what I gather.

I wonder how and when FX-322 would be available in the UK on the NHS, given that we're heading for a no-deal Brexit...

 

[Christiaan]

Is this official?

This is from Frequency Therapeutics' business report from a few months ago:

 

[Christiaan]

I wonder how and when FX-322 would be available in the UK on the NHS, given that we're heading for a no-deal Brexit...

Oomph, that's one tough cookie, especially when it concerns Scotland's desire to stay in the EU (given SNP's + Scottish Greens' current polls). I really do hope that Astellas will also put a lot of effort in expanding their clinical trials to non-EU countries like the UK, although I understand that it seems like a lot of hassle for the company from a judicial & financial point of view.

 

[Philip83]

This is from Frequency Therapeutics' business report from a few months ago:

View attachment 41584

Nice, thanks!