December 20, 2021 / Clinical Trials

Update on Pfizer’s Phase 1B Open-Label Mini-Dystrophin Gene Therapy Trial for Duchenne

Today we learned that there has been a devastating loss of a young man that was participating in Pfizer’s Phase 1B Open-Label study. This study is evaluating PF- 06939926 which is Pfizer’s AAV9 Gene Therapy candidate for Duchenne.

According to the company, “At this time, we do not yet have complete information and are actively working with the trial site investigator to understand what happened. Screening and dosing in our Phase 1b clinical trial have been paused and we are working closely with the independent External Data Monitoring Committee to review the data. Regulators have been notified and the U.S. FDA has placed the Investigational New Drug (IND) Application on clinical hold. The safety and well-being of the patients in our clinical trial remains our top priority, and we are committed to sharing more information with the medical and patient community as soon as we can.”

Pfizer issued this community letter informing us of the tragic news.

We are heartbroken for this family and know that there is much to learn around what has happened. As a community, we will come together to get through today, and tomorrow.

A note from PPMD’s Pat Furlong

Talking to the community, whether through writing or Zooms or in person at meetings and conferences or in the chilly hours before a Disney World sunrise breaks over our runners, remains a source of joy for me. And that’s because it’s connection–the purest, most powerful gift we have to give one another. Today I call upon this fellowship in our Duchenne family to address a recent, devastating loss of a beautiful young man with Duchenne that has caused us all to suffer.

Shock. Sadness. Anger. Confusion. Heartbreak.

All emotions are valid and deserve to be felt, experienced, expressed. The words to describe the unjust nature of a tragedy like this lay meaningless on the page, they are swallowed up in our throats along with our grief. No force of expression comes close for this family, for our Duchenne family.

Our hearts go out to this family, grateful for their bravery, accepting unknown risks and uncertain terrain.

And our hearts go out to all of you, heart in hand, as we hold this family in our prayers and hold onto hope.

Science is mapping on the grandest and sometimes most fantastical scale. Centuries ago we could only see the outlines of a continent here, the peaks of distant mountains there. The pioneers’ work is never done. Just when we think we’ve reached the limits of a quadrant, the most distant shore, someone spots a new passage, another valley, a whole other land mass of undiscovered riches and unknown challenges. So it is with gene therapy.

Together we fan out as one community moving over this new terrain. Our imaginations are captured and energized by the word “therapy,” a term we have waited to hear for so long for all of the promise and possibilities it holds. Then, as all know who have ever pushed into strange frontiers, there are the realities that come with world building. In this case, I am referring to what must also be considered along with the, now familiar, phrase “gene therapy,” which are the words around it: Investigational Gene Therapy Study (Trial).

These bookends carry immense weight. They signal there is more to understand in this landscape than just what we want to see or are eager to find. Investigational means under investigation; its synonyms gesture to discovery– examine, study, explore, interrogate, probe –uncertainty and unknowns in both risks and benefits. Study (Trial) is the path, the way, the steps we take to comprehend what has happened, what needs to happen, and what may cause us to revise and refine and even rebuild the cobblestones we are piecing together as we move forward into largely uncharted lands.

Our hearts are heavy. The journey and the work feels too hard to undertake. It’s okay to rest, to take time; it’s necessary to reflect deeply and ask questions and check in with each other along the way. That, too, is part of the precious connection we share. To be able to lift one another while we climb so that we may continue onward together with resilience, courage, and hope.

And we will move forward resolutely, thoughtfully, judiciously. We will be vigilant. We will seek answers. We will not ever give up our hope.

We must continue to invest in genetic therapies because of the science and possibilities, but we must do with blinders removed from our hearts and minds… we lift one another while we climb, even if we do so with heavy hearts and weary hands… we keep going forward…

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