Updates about Summit & BioMarin's Utrophin Upregulator

Not so good news coming from Summit & BioMarin. The original plan was for a clinical trial to start "early" this year.

BioMarin is now helping with the preclinical work. http://www.bmrn.com/pipeline/utrophin-inducer.php

The following message is from ActionDuchenne who initiated contact with both Summit and BioMarin to inquire about the apparent delay. (Thanks Action Duchenne!)

Hi there,

We have received a speedy response from Biomarin (which I suspect has prompted the update on their website) which states that although Summit has the responsibility for all preclinical activities, as was contemplated in the original agreement, 'BioMarin is providing substantial assistance with this work'

They advise that during the third quarter of last year, it became apparent that 'the proposed formulation of the drug did not have suitable bio-availability and therefore Summit and BioMarin are currently working to modify the formulation.

Biomarin state that if the reformulation and related pre-clinical research are successful, they intend to commence trials as soon as feasible.

Naturally, we will continue to push for progress on this with both Summit and Biomarin.

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I remember something about Summit going BK. I didn't follow up on them but instead called BioM directly and asked the status. I guess the exact status of Summit could be found on their web page with latest financials. I specifically asked the researcher if Summit going BK was going to halt the whole thing and he said no, we are just a little behind our original timeframe but still plan to go forward. But, now that BioM has stated their financials aren't that great...anything could happen. Sometimes the researchers don't know what the accounting dept does.

You could call BioM directly, it does take a few times to actually reach somebody but the guy I spoke to did return my call.

Keith said:
So, based on their earlier statement of:

"We have not yet set a time for the clinical study, but once the formulation and preclinical plan are resolved, we will be providing a more precise timeline for moving ahead to a clinical program."

...and that they're now setting a timeline, can we assume they have resolved the bio-availability issue they were concerned about earlier?

If so, that's awesome.

Any idea what "initiation" of trial means? Is that when they start planning it, or start doing it? Any talk of where it would happen? Their corporate HQ is in CA - is that where this particular project is being worked?

Is Summit out of business now? Were they working on anything else MD related?

Thanks for posting these updates, Ofelia.
This was posted on Action Duchenne about BioMarin's Utrophin upregulator.


Good evening all, been chasing up BioMarion in the USA to get some firm answers on the Summit Utrophin project

We asked if they need any financial support to speed things things up and if they require any other assistance got the following 2 responses

Dear Mr. Levene,

Thank you for your recent telephone inquiry regarding the status of funds for the preclinical program for Duchenne Muscular Dystrophy. Mr. Bienaime forwarded your message to me and asked that I respond.

We are pleased to inform you that the need to raise outside funds to support this ongoing research program is not necessary as BioMarin Pharmaceutical has purchased all development and licensing rights for the program from Summit PLC. BioMarin is now funding and managing the entire program independently. Currently, preclinical studies are ongoing and we hope to initiate clinical studies in the first quarter of 2010.

Best Regards,

Susan Berg
Senior Manager, Corporate Communications
BioMarin Pharmaceutical Inc.
105 Digital Drive
Novato, CA 94949 USA

At this point I asked them to confirm no research is being conducted in the u.k. and everything is well funded and now been moved to the USA got the following reply.....

Dear Mr. Levene,

You are correct. BioMarin is now independently conducting preclinical research with Utrophin in our laboratories in the United States and we hope to initiate clinical studies in the first quarter of 2010.

We very much appreciate your ongoing interest in this program and we are happy to continue an open dialogue with you as the program progresses.

Best Regards,

Susan Berg
Hey, this is good news.
Thanks Ofelia for making my day a bit brighter - I live for bits of good news just like this one!
Well, the BMN 195 clinical trial has started in healthy volunteers. Let's hope that everything goes as planned and phase 2 will be following up shortly. Has anyone heard anything since it has started?
I haven't heard anything Kristi. Maybe someone else has.

Kristi Koop said:
Well, the BMN 195 clinical trial has started in healthy volunteers. Let's hope that everything goes as planned and phase 2 will be following up shortly. Has anyone heard anything since it has started?
Thanks a lot Ofelia, Kristi and everybody else sharing their information with the PPMD community. After the still not resolved but increasingly bleak picture coming out of PTC, we really need some good news around here!
I believe results for phase 1 are due this quarter. Biomarin is presenting a company update on 05/19.

FYI, Biomarin will be presenting at the Duchenne Connect conference this Summer in the joint session with families and scientists. An excellent chance to ask questions!


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