Project Catalyst : what is the present status of their utrophin upregulation drug candidate

Initiated by PPMD in 2003 Project catalyst was born out of a belief that present generation of young boys deserves a decisive and positive response to the challenges they face.

                     We are in the end of q1 2012, but there are not a single clinical trial by them. I emailed them about utrophin upregulation drug and they replied me on 07/11 that they expect utrophin compound in discovery to be in preliminary trials in the next 18 to 24 months.

                             My son ( my only child , 6y old) is not eligible for either " exon skipping " or "stop codon". After his diagnosis  I am listening about exon skipping trial news. My sincere wishes with them. But the rest of 85% boys including my son also deserve a positive treatment other than steroid.So, my only hope is Utrophin upregulation drug. ( considering the fact, that ace 031 is on hold)At this point I can see only SUMMIT's motivation that they are starting ph 1 of their reformulated drug. Other utrophin drug like TAT Utrophin and DR. Khurana's compound are still in preclinical stage( as per my knowledge).

             Pat , you are our strength. Could you please influence project catalyst's utrophin drug to be in clinical trial in 2012. The field is ripe, the horizon is nearer ( with all your effort) ,so what for we are waiting?

I am all ears to listen something from you or Sharon.

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Hi Rupjani:


The Project Catalyst utrophin upregulator probably won't go to trial until 2013.  I know this has been a really long road--I think we first gave them funding to do the original drug screen in 2004.  We did a Webinar a while back with PTC Therapeutics to go over progress on these projects and Ellen Welch, the lead scientist, explained in extremely great detail what work remained to be completed before they can go to trial but unfortunately we weren't allowed to record it.  Basically they want  to go into the study with the best compound they can create given that this is something they can control and there are so many other variables they can't.  You could probably get into the clinic faster with a drug that was doomed to failure due to forseeable problems and they want to avoid that scenario.

I know it's frustrating, but there are at least three utrophin upregulators in the works, plus Dr. Kurana's candidates.  And there may be different drug trials planned that would not be mutation specific that we will hear about soon--can't say more than that unfortunately.



Hi Sharon

Thanks for your reply.I just wonder the trial you mentioned is the healthy volunteer study or DMD boys study.

Best Regards


Not sure yet, actually.




I hate to be the downer, but get used to this glacial pace of work when it comes to traditional biotech. There are families so frustrated with this mess of an industry that they are literally buying the intellectual property rights from biotech companies because the KNOW they can do it faster.

So if you have a few million bucks laying around you might have a chance to speed things up. Otherwise, welcome to the "All you can do is wait and donate" club.

 David.  Not to be difficult, but aren't the Secklers raising money for this project through donations?  I know they bought the IP, but after that it becomes another industry drug development project--Halo Therapeutics is a for-profit company.  Halofuginone will face the same hurdles as any other drug in development except that it has had some exposure in humans before and was previously in use by the poultry industry.  The Project Catalyst drugs were developed "from scratch" by screening thousands of compounds to develop candidate drugs -- these weren't compounds that had been previously developed for any other use.  So it will take longer to get these drugs into clinical testing than some others, but Project Catalyst is only a small part of the PPMD portfolio.  We are also supporting drugs at Tivorsan, Summit and AVI that are in clinical testing or are closer to clinical testing and our business plan for research is specifically focused on increasing the efficiency of drug development (I can send it to you if you are interested).  All of that being said, I happen to like the data for Halofuginone and think it's a very interesting prospect. 


I can feel your frustration and am happy to discuss this at greater length.



I'm not trying to compare the actual drugs or specific programs. I'm talking about the level of frustration with the accepted pace of work that IMHO is endemic in this field regardless of whether for-profit or non-profit organizations are involved.

This isn't even specific to Project Catalyst, although the lack of viewable progress there makes it a great example.

What I'm saying is that I don't accept that the pace of change in this industry is because of factors beyond everyone's control, which seems to be the response that is always provided when the questions like Rupjani's are asked. These projects set their own dates, years out, and then fail to meet them on a consistent basis. They put treatments on the shelf for months or years with no substantive information on why or when it may start again.

And then when the dates slip past and questions are asked, they typically respond as you did: "other variables they can't control." This does not fill me with confidence to say the least. Isn;t that was science is all about?

So, I applaud any effort that also refuses to accept the status quo in that respect. The Secklers themselves said it best:

"It opened our eyes that we could not control the pace until we had operational control" of a drug's development, he says.


Hi David

Neither I have few million bucks nor I want to join " wait and watch " club. But you, me and other " non exon skipping" parents can do one thing that we keep on posting focussing only small molecule drugs like utrophin ,miostatin inhibitor etc so that we can give things a nudge. At least we can expext same speed and seriousness like exon skipping.After all, this utrophin drug could be beneficial for exon skipping boys also. Enough is enough !!! This is the time we must have something in our hand to give our boys other than steroid. after that we will think about gene and stem cell therapy like big things.



David, I absolutely agree that on the whole there is too much acceptance of status quo for timelines and regulatory requirements etc.  PPMD has focused its research efforts on activities specifically designed to speed up the pace of drug development and decrease the costs:  This is the filter through which every funding decision is made.  No one can afford to "donate and wait."  I guess my point is that we are not doing that either but our feeling is that some of the bottlenecks in the system have to be managed and changed at a level that is higher than that of a single project (hence our focus on FDA regulatory changes in Washington).   But on a project basis, most of our larger projects are milestone-driven--the investigators don't get paid until they get the work accomplished and we can cancel projects and get funds back with interest if they fail to meet milestones.  We are also focusing on prioritizing approved drugs for clinical testing (and funding trials in approved drugs in parallel--not waiting).

Honestly, I just don't want you to feel like no one is thinking about this--we have different answers to the problem, but we haven't accepted the status quo either. 



I am the grandmother of two boys with DMD who are also  among the "other" 85%.  They are ages 7 and 5 (I used to say ages 6 and 4, and ages 5 and 3 and ages 4 and 2 and ages 3 and 1 and age 2 when the first one was diagnosed.)  Will I be saying ages 22 and 20 and still waiting?  I agree with Rupjani and David. 


What could be other variables mentioned by you. One variable that is in my mind is the magic figure. A drug development company surely will focus on  the revenue they will earn from a specific drug. At present total number of DMD boys globally is 60,000 ( I am not sure but that is my best knowledge).Isn't it the number that could motivate a drug company. And these small molucle drug will be beneficial for BMD boys as well as exon skipping boys also.I think 100% is greater than 15%.But my point is that project catalyst is not a typical drug company, isn't it? Unlike a hybernation with ACE 031 they can share some information with us like what are the obstacle they are facing ? 

I think a good example of what David mentioned above is ACE-031. It has been a year since the trial was suspended and we have not heard anything. I am sorry to say that this is way beyond frustrating at this point. More than one year on hold when these boys are rapidly declining is not acceptable and I do not believe that they are moving as fast as they can on this one I am sorry to say. Whatever happens at Shire or Acceleron with this drug would be very interesting to know, is this sitting on a shelf now, are they working on it still?


Rupjani, my son is one of the ones that might be treated by exon skipping and I do not feel any better. That drug can fail at any time and the results so far regarding improvement as reported by parents in those trial and the companies are variable and not stelar. So I think that we need to focus on moving forward several of these strategies to have a chance to some combination significantly modifying this disease. Unfortunately our boys are getting older and their chance for benefiting from these is getting smaller. 


And don't even get me started about a replacement to steroids so the boys do not suffer debilitating side effects. That's not moving any faster either. I heard about Eric Hoffman's steroid replacement starting trial for the past 2 years and with every passing year the timeline stays the same or expands. 


That is my point also.I think we must concentrate only on utrophin drug.Because

1. there will not be any immunological issue.

2. will be beneficial for all boys

3. much simple process

we need any positive drug immediately so that we can buy some time for our boys and then focus on other treatment.We don't want to expand "frustrating parents " club.

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