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.
Rgds

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So, is the BioMarin BMN-125 the same drug as what Summit calls SMTC1100, or are there two utrophin upregulators being worked on?

There's a nice chart of drug development status in the latest Quest magazine.

http://www.mda.org/publications/Quest/q161InFocus.html
Yes. It is the same drug. Summit & BioMarin signed a deal last year to develop this. Of course, the original plan was for Summit to complete the preclinical work by the end of 2008 and BioMarin to start clinical in 2009. Now plans changed as stated in AD's message.


Keith said:
So, is the BioMarin BMN-125 the same drug as what Summit calls SMTC1100, or are there two utrophin upregulators being worked on?

There's a nice chart of drug development status in the latest Quest magazine.

http://www.mda.org/publications/Quest/q161InFocus.html
Thanks for the info. You're really on top of this stuff!
This is from BioMarin's yesterday conference call. Unfortunately, it looks like the reformulation involves more preclinical work and toxicology studies. It doesn't look like something that can be resolved fast.

http://seekingalpha.com/article/121337-biomarin-pharmaceutical-inc-...


In terms of our other preclinical programs, as announced in July of 2008, BioMarin and Summit signed a development agreement for the clinical development of SMT 1100, a small molecule inducer of utrophin, a protein that can potentially replace the defective dystrophin protein in patients with Duchenne Muscular Dystrophy.

Based on some early preclinical work, some reformulation work was needed as well as additional toxicology studies, which has pushed out the start of the clinical program. 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.
Thanks for the update. Do you know where this work is physically taking place?
My understanding is that both BioMarin and Summit are doing preclinical work (so both in CA and UK). The original agreement was that Summit will finalize the preclinical work by the end of '08, but since the reformulation BioMarin seems to help with that too.

Keith said:
Thanks for the update. Do you know where this work is physically taking place?
Is this BioMarin/Summit project the only one you're aware of that's anywhere near a clinical trial ?
For utrophin upregulation yes. PTC is doing some work and there is also Justin Fallon's biglycan, but I do not think they are close to entering clinics, definitely not this year. Pat should have more info about those timelines.


Keith said:
Is this BioMarin/Summit project the only one you're aware of that's anywhere near a clinical trial ?
http://phx.corporate-ir.net/phoenix.zhtml?c=106657&p=RssLanding...

...

In the first quarter of 2009, BioMarin recorded impairment loss on investments of $5.9 million, comprised of $1.4 million related to its equity investment in Summit Corporation plc and $4.5 million related to its equity investment in La Jolla Pharmaceutical Company. The remaining investments of $0.2 million in Summit Corporation and $1.8 million in La Jolla Pharmaceutical are reflected on the balance sheet as of March 31, 2009. Since both of these companies have announced that they do not have sufficient resources to fund operations for the next twelve months, the guidance below reflects the assumption that the remaining investments in both companies will be written down to zero by the end of the year.

...

BMN-195 - Utrophin upregulator for Duchenne Muscular Dystrophy: BioMarin is completing reformulation work and toxicology studies and expects to initiate a Phase I trial by the first quarter of 2010. BMN-195 is an orally available small molecule which may upregulate utrophin, a potential replacement for the missing dystrophin protein in DMD patients.

...

Anticipated Upcoming Milestones


2Q09: Results from PEG-PAL Phase I trial
2Q09: Initiation of PEG-PAL Phase II trial
3Q09: Decision on strategy for BMN-103 for Pompe Disease
3Q09: Decision on 6R-BH4 cardiovascular program
1Q10: Initiation of Phase I trial for BMN-195 for DMD
1H10: Results from Phase I/II trial for GALNS for MPS IVA
Mid-2010: Results from PEG-PAL Phase II trial
2H10: Initiation of pivotal Phase III trial for GALNS for MPS IVA
2H10: Availability of blood Phe monitor
Thanks for the information. Michelle
I contacted a researcher at BioM a few weeks back. Can't remember his name but he said they were a little behind their previous schedule but still planned to go forward. Not sure what the latest financial statement will do...hope it still goes to trial. First Q10 isn't that far away.
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.

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