NurOwn stem cell phase 3 study

[lgelb]

Jethro, it is routine to file patents well before effectiveness has been demonstrated. There is a long lead time (years) before they are granted. In fact, you have to, because if someone else files first, they beat you out and you cannot file later. So don't read anything into that.

 

[Par220]

I have really searched for any new updates of people in the trial. I'm not able to find anything. Is it possible they asked participants to keep quiet?

 

[Nikki J]

That is usual yes. Participants are generally told not to speak of perceived results or lack thereof while a trial is in progress. I was surprised at the public comments made ( I think by a CALS) about apparent improvement in a PALS in this trial a while ago.

 

[Par220]

I wanted to post this interim safety analysis for the trial from this morning. I was really hoping they would have some preliminary efficacy data so there would be some small chance there would be accelerated approval if it appears to be working. Considering the diagnosis it seems like they should look sooner so they can present to FDA if there is a positive trend.

Does anyone know if they will look at efficacy at any point during the trial or will that only be at the end?

BrainStorm Cell Therapeutics Announces Successful Interim Safety Analysis in Phase 3 Trial of NurOwn® Therapy for ALS Nasdaq:BCLI

 

[lgelb]

The interim safety eval is standard, esp. for a biological. But given the design and staggered enrollment/treatment intervals, I see no possibility that an interim efficacy analysis could drive an earlier BLA filing than planned.

 

[oya99]

@par220
This review is standard procedure and does not speak to the efficacy of the procedure, only confirms that best practices are followed by an outside body (DSMB) as expected by the FDA.

To be clear the following is my conjecture:
What is new in this article is the information about the timeline. The clinical trial documents gave a timeline suggesting that the trial results will be available mid 2019; however, this article is mentioning that the clinical trial will not complete enrolling patients until mid 2019. The initial goal was to enroll 200 patients, only 110 will be enrolled by January 2019. Following the last patient’s enrollment, first there is a several month treatment period, then comes the observation period of another 6 months. This puts the completion of the study towards mid 2020 and possibly the results being prepared and published at the end of 2020 or later. Any possible approval of the procedure by FDA (only if the results are favorable) for usage and subsequent approval by the insurance companies for payment with this timeline is pushed further into late 2021 at the earliest.
In my view, that is a significant delay compared to what the CEO has been saying up until recently i.e. an early 2020 availability.

 

[Par220]

I agree. The timetable has been pushed back which is hard to hear, but unfortunately so common with these trials. I wish somehow they could keep pushing for RTT as so many will be gone by then without a chance.

 

[oya99]

@par220
I have talked to someone at the Goldwater Institute (one of the major forces behind RTT) around the passing of the law, and they said that it is completely compulsory for both ends i.e. both the patient and the pharmaceutical company. Furthermore, the patient needs a recommending doctor who is willing to prescribe a treatment that is not approved by FDA.
Unfortunately, pushing or pressuring is not possible under RTT’s current design, it will take a true “miracle” to get any of these companies to comply.

 

[lgelb]

Ova, I think you meant "optional" instead of "compulsory."

Best,
Laurie

 

[oya99]

Should have typed “not” compulsory...

 

[Suesuetoo]

I have had no progression for a year and a half just by following the ALS society's program
Al

My daughter in law diagnosed April this year.
Can you tell me what is the ALS society program?

 

[Firefighter58]

Hi Suesuetoo I was put on the program in Canada but is easy to follow. Look under supplements my whole program is there. I'am still doing well.
Al

 

[Par220]

Hi everyone,

I just wanted to put this out there for anyone who agrees and is interested. I still write regular emails to Brainstorm urging them not to abandon offering pathways for access for those of us not eligible for the trial (right to try, hospital exemption). They were mentioned specifically by the president and the CEO really seemed to be behind it at first. I just want them to hear from those of us desperately wanting a chance to try. Here is the email address/info for anyone else interested.

[email protected]
Mary Kay Turner
Vice President
Patient Advocacy and Government Affairs
Brainstorm Cell Therapeutics Ltd.
1745 Broadway, 17th Floor
New York, NY 10019
Phone: 201-488-0460 Ext. 105

 

[lgelb]

A few notes from the Q3 earnings call (not sure this link will work for all but you can sign up to get these):

Enrollment should complete in first half next year. It's said to be going well, but they didn't reveal exact numbers (will be 200 total).

Top-line efficacy data should be available first half 2020.

They are filing another unspecified IND (application to research a different use) this year for NurOwn (confirming their commitment to the platform).

They cannot address any possible compassionate use to continue treatment for those who were/will be treated during the trial until the trial is unblinded (revealing who was treated and who wasn't). This is standard stuff, but since an analyst asked the question, obviously not everyone is aware.

All in all, a positive call.