GSK & PTC Updates

GSK

First off, a qualifier. If you are reading this for inside information, such as the start date of the trial, the protocol, the willingness (or not) to consider compassionate use, etc., stop reading right now.

We (Sharon Hesterlee, Kimberly Galberaith, and I) met the GSK team and while all of those thoughts were on our mind, we knew that those were not the questions to ask and that we were not meeting for the purpose of asking for confidential information in an effort to leak it to the greater community. Rather we were meeting to think about how we might work together to support and accelerate trials (and hopefully treatments) for our sons.

The GSK folks we met with consisted of the people involved in scientific communications, global commercial strategy, alliance development, and public policy. We went into this meeting without an agenda. We felt a synergy the moment we met and the conversation flowed from there, covering a range of topics to include urgency, high unmet need, anticipation, and expectations. We discussed the GSK relationship with Prosensa and the capability and capacity they bring to the table. It was obvious that GSK is committed to Duchenne. It felt good to see their interest in Duchenne, their openness to new opportunities, and here’s the thing...having GSK (better known as ‘big pharma’) onboard is quite a coup to be honest. These folks know the ropes, they have experience in taking drugs through the clinical process and to market.

We spent some time talking about the individuals and families who do not fit into the current antisense compounds (rare deletions, duplications), how the community feels fragmented based on mutations, as if, for some boys, there is less hope than for others. We talked about the fragility of the community, the increased frustration and desperation as strength decreases and walking is threatened. They understood, perhaps not to the degree of parents/families watching minute by minute, but the message was heard loud and clear. We spent a good bit of time discussing the need for a regular communications plan, a website where all foundations/organizations would direct interested individuals: the one-stop shopping for accurate information and consistent messaging, so that information does not arrive in sound bites or tidbits of information from anyone, rather information we all can count on to be accurate. We learned that while this sounds easy on some level, there are loads of hoops before information is released and SEC and regulatory concerns that need to be considered, such as communication that might suggest results or promise, or anything that can be perceived as persuasive in terms of trial recruitment.

We discussed clinical sites and whether more with fewer patients is better or less with more patients might produce better data with less variability. We discussed using clinical sites with expertise and experience (and trust by the community) in Duchenne AND with clinical trial experience.

We discussed their commitment. And while it is true, none of the people we met with have a child with Duchenne, they all have a story about their interest in medicine, the soundtrack of their lives where the twists and turns of their careers brought them to this place, this world of Duchenne. And they seemed happy to be here, dedicated to making a difference and joined the ‘church’ of believers, that Duchenne can and will have treatments.

As you might imagine, we left with that same laundry list of questions in our head, but respected the fact that data would become public during the AAN meeting. We discussed protocols, asked if they would consider expanded access or compassion and the ever present “WHEN” question. No answers at the moment and all possibilities are in discussion.

GSK/Prosensa will release data from the European trial during the upcoming AAN meeting, April 12-17 i in Toronto. Exact date is April 15. Stay tuned.

PTC

Same qualifier as above. If you are looking for sub-analysis data and next steps, stop reading.

Later the same day, we met with Stuart Peltz, Langdon Miller, Theresa Nalatacchio, and Diane Goetz. Fatigue was obvious as the analysis continues. You all already know, I think the PTC folks are what my mother calls “good people.” I have known Stu Peltz for 7 years. He is committed to Duchenne. He is a fighter. With his team he is sorting the data, sifting through to see the path forward. They believe there is a path and I think this coincides with the community’s belief. It isn’t easy and the path is not crystal clear at this moment as they weight the arguments and plans for moving forward.

We discussed the fact that the community feels like the rug has been pulled out from under, that the messaging from the clinical sites varying and frustrating, that parents are worried about what happens physically and psychologically as the drug/compound is stopped. And that frustration has turned to anger and there is no place to put the anger. Stu and his colleagues understand.

I guess that’s hard to imagine from where you sit, but it was quite obvious on their faces. This was not the plan. PTC has been the pioneer, the oldest child (in a manner of speaking) trying to carve a path forward in the best way possible, with a community that has not participated in registration trials, a community that does not have rigorous natural history data, a community with high unmet medical needs, and a sense of urgency that is hard to actually put into words. An urgency that makes a parents’ blood flow feel like lava when their child falls, or stops walking, or worse.

I believe in PTC and believe that they are working very hard to develop a plan for success. We talked about access programs, about compassion, and talked about Genzyme’s program. I was surprised to learn that Genzyme’s program for expanded access involved less than 5 people. PTC has 200 +/- boys who have participated in trials to consider as they plan next steps. They are listening. But at the same time, they need to succeed, otherwise even expanded use or compassionate use would come to a stop.

Ataluren needs a path forward, so that all boys who could likely benefit, have access to that opportunity.