IND-enabling studies include all the experiments that are required by the FDA before applying for permission to start human clinical trials. These studies include:
The timeline to complete IND-enabling studies is typically 18 to 24 months with an average cost of $10 to $15 million per lead compound. If the FDA approves the IND it’s off to the races with clinical trials in humans.
The AveXis/Novartis product is currently at this phase of development. The basic science and lead compound development work was funded by RSRT through our Gene Therapy Consortium 1.0. We purposefully recruited Brian Kaspar into the Consortium with the hope that the data generated would be sufficiently encouraging that his company, AveXis, would want to pursue Rett as an indication. A million things could have gone wrong but fortunately the pieces fell into place. AveXis funded the IND-enabling experiments and filed the IND application with the FDA. We are currently awaiting word regarding when the clinical trial will start.
When the human trials are completed and the company is confident of safety and efficacy of the therapy a New Drug Application, or NDA, is submitted to the FDA for review. If the treatment has demonstrated sufficient safety and efficacy up to FDA standards, drug approval and commercialization follow.
So now you know the process and at which stage our programs fall. What you may not know is how difficult every step of this is. Each step comes with its own set of challenges not to mention expense. And none of it happens on its own. Academics are great at making discoveries but they are not the ones who develop drugs. The great divide between an academic discovery and lead compound development is aptly called the “Valley of Death”. One of the key responsibilities of RSRT’s research team is to not only ensure that discoveries are made but to then ensure that they don’t languish. Each program needs to be nurtured and the right partners brought in to move it along. Fortunately our research team collectively has decades of drug development experience as well as business development experience. We are well suited to take on this massive responsibility.
We are thrilled that gene therapy trials in Rett will begin soon. If that proves to be a cure, our work will expand to include ensuring that gene therapy is widely accessible for all with Rett. In the meantime we continue to move our rich pipeline of potential products forward. Our goal is to bring a gene therapy 2.0 product to the clinic by 2021 followed by four other non-gene therapy products. I and my team look forward to keeping you updated.