CNM-Au8 update

[Nikki J]

The company is going for accelerated approval and the fda is looking for nfl and survival data as well as more data on target engagement. All very reasonable. In the meantime Clene is proceeding with a phase 3 trial to start in the new year. It would be a confirmatory trial if they did get early approval.

FDA Provides Roadmap for Accelerated Approval Pathway Through Submission of Additional CNM-Au8® Biomarker Data in ALS | Clene Inc.

FDA recommends that Clene leverage additional Neurofilament Light (NfL) data from its three Expanded Access Protocols (EAPs) and the HEALEY ALS Platform Trial to support earlier presented findings FDA recommends a follow-up meeting to discuss in more detail the analyses needed to support the

invest.clene.com

 

[JAM]

Ty Nikki! Also with the Clene drug was does confirmatory approval mean/entail?

 

[Nikki J]

IF they get early approval it will likely be provisional pending results of the phase 3. Somewhat like relyvrio though thecapprpval wasn’t provisional. However the company did promise orally to withdraw the drug if it failed phase 3 and to their credit they withdrew it quickly without fda prompting.

Another example is qalsody. They had a phase 3 which frankly was not long enough and did not meet endpoints. They got a conditional approval and their confirmatory trial is ATLAS which is looking at asymptomatic carriers and following nfl. When it rises they go into a placebo controlled trial to see if onset is delayed or prevented. If someone gets ALS during any phase of the trial they get qalsody. Of course the prevention type of trial is only possible for carriers at this point

 

[santimo]

This seems moderately promising!

 

[Nikki J]

We have no idea what data they will find if/ when they get it. The fda did refuse their two previous requests

I am happy the fda is requiring nfl data as I think biomarker data is important to get a clearer picture of what works and what doesn’t

 

[JAM]

Do you know why FDA denied in past? Just curious as to what would be different now?

 

[Nikki J]

Presumably because the phase 2 results were not strong enough to warrant accelerated approval and whatever they added to their second request was not either. Approval after phase 2 is very uncommon. Of course companies want it so they can start selling even if they have to do a phase 3 anyway. Trials - especially large phase 3s- are VERY expensive.

 

[JAM]

Welp, I guess if a company really believes in its product the expense of phase 3 would not be too much of an issue, bc they would have confidence that it would be successful in Ph3 and in return make gobs of money.. This one isn’t passing the smell test rn

 

[Nikki J]

That is probably a little harsh. They are a small (85 total employees last year) new company with no current drug on the market. They are consistently losing money and operating with grants and investor money while running trials and doing research into neurodegenerative diseases. This is not to say they deserve early approval- that depends on data but wishing for it is not a sign they are only in it for money. You could even argue they believe on ot and want current pals to benefit now instead of pals in several years

 

[JAM]

I hope you are right and that their persistence is bc they feel there product is that good. We would all welcome that and something is so needed

 

[Megatron]

Hi; I don't understand your seemingly pejorative comments regarding qalsody. Those with familial ALS are widely reported to be significantly improving after being treated with qalsody/tofersen. It is surely being prescribed to everyone with familial als who is aware of it and wants it. It appears that all comments are from this last week. Moreover, within the last few days it's been approved for coverage by Medicare. Can you please clarify?

 

[Nikki J]

Qalsody is a fabulous drug. It isn’t a cure but has been a game changer for sod1. My comment was solely that had the trial been longer they would have had the data for a straight out approval. The phase three did not meet end points. That is fact. My comment that the trial needed to be longer has been made by doctors who were involved in the trial. It really took a year for the clinical benefit to show in the ole data. The actual trial was six months.

As I said in the other thread the new cms rule is for Medicare Advantage plans. Original medicare already covered it.

And again it is only for SOD1 ALS Those of us with other forms of FALS would not benefit from it.