By Erin Harris, Editor-In-Chief, Cell & Gene
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During The ASCGT 24th Annual Meeting, BridgeBio announced an update on two of its gene therapy programs that are on track to enter clinical studies this year. The company presented data from IND-enabling studies for BBP-631 for congenital adrenal hyperplasia (one of its core four programs) and BBP-812 for Canavan disease. In addition, BBP-631 has been granted Fast Track Designation and Rare Pediatric Disease Designation by the FDA and Orphan Drug Designation by the FDA and European Medicines Agency (EMA). After ASGCT, I caught up with Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy to learn more about the company’s long-term strategy as well as manufacturing expectations for BBP-631. Here’s what he had to say.
What is BridgeBio’s broad, long-term strategy for treatment development for both Canavan disease and congenital adrenal hyperplasia? What can the sector expect and when?
David: Our overall strategy for the gene therapy programs is the same as that for BridgeBio overall, which is to get our therapies to patients as fast and as safely as possible. We expect to explore not only Fast Track (already granted for congenital adrenal hyperplasia) but other designations, and plan to work collaboratively with the FDA and potentially regulators in other countries to move as aggressively as we can be given the huge unmet need for both congenital adrenal hyperplasia (CAH) and Canavan disease patients and families. The IND application for our CAH program has been cleared by the FDA and site activation for initiation of a first-in-human Phase 1/2 study is ongoing, with initial data anticipated in late 2021 or early 2022. Our Canavan trial is expected to begin in the coming months.
BBP-631 has been granted Fast Track Designation and Rare Pediatric Designation by the FDA. What can patients expect should this be granted approval?
David: The IND application has been cleared by the FDA and site activation for initiation of a first-in-human Phase 1/2 study is ongoing, with initial data anticipated in late 2021 or early 2022. As always, we intend to work with the FDA and potentially regulators in other countries to move the program forward as quickly as we can. I would point you to my answer to question #1 above.
What manufacturing challenges and wins are expected if BBP-631 is approved?
David: While we do not currently expect any manufacturing challenges, all gene therapy companies acknowledge that manufacturing for gene therapy can entail unanticipated risks. As we have mentioned in the past, BridgeBio Gene Therapy has an experienced CMC team in our labs in Raleigh, NC that works on manufacturing strategy, process development and optimization, and analytical development (including potency assay development). They also work closely with our manufacturing partners at Catalent where we have a dedicated suite in one of Catalent’s GMP facilities. To date, our clinical manufacturing campaigns for both the CAH and Canavan programs have gone according to plan. We continue to look at ways of further optimizing our clinical-stage vectors, with the goal of reducing cost and improving safety beyond our already exacting standards.
What is on the horizon for BridgeBio?
David: In addition to our clinical-stage gene therapy programs for CAH and Canavan disease, we also have a robust and growing pipeline of several early-stage programs, one of which we have announced publicly: an AAV program for TMC1 hearing loss. We are unusual among gene therapy companies in being agnostic to tissue or disease area — we work to select programs based on where the unmet need for patients is greatest and where we think that unmet need could be best addressed by a gene therapy option. One of the wonderful things about BridgeBio is that the company itself is agnostic to modality: We focus on addressing the greatest unmet needs in rare diseases and targeted oncology, and if we can meet those needs with a small molecule drug or a conventional biologic, we will pursue those approaches. However, some diseases are best addressed with gene replacement therapy, and those are the ones my team focuses on.