Rare Disease Report

CMO of Catalyst Biosciences Explains the Current State of Hemophilia

DECEMBER 11, 2017
Howard Levy, MBBCh, PhD, MMM
Howard Levy, MBBCh, PhD, MMM, and Chief Medical Officer at Catalyst Biosciences, sat down with Rare Disease Report at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition.

With all of the exciting breakthroughs in the hemophilia space lately, most of which pertains to therapies that reduce annual bleeding rates (ABR), Levy says it's important to keep one common goal in mind: a complete elimination bleeding. In this video, you'll hear his stance on gene therapy, why it's so important to work toward goals for this patient population, and his company's plans as potential therapies for the condition move through the pipeline.

Rare Disease Report (RDR): How excited are you about the potential of gene therapy as a treatment for hemophilia?

Levy: There’s a lot of exciting stuff happening in hemophilia and promises for the future. Gene therapy gets a lot of press, but good effective therapies, I believe, are still the way to go, because we know how to use them so well.

RDR: What has Catalyst Biosciences been working on pertaining to hemophilia?

Levy: Hemophilia B has become a very neglected disease with the recent approval of emicizumab. Hemophilia has a patient population devastated by disease; people need knee replacements and have fatal bleeds. It’s an ever present worry for families and children, and those who need to treat these patients. Catalyst is developing a subcutaneous way of administering factor IX and factor VIIa for patients with inhibitors. It’s not just the convenience of a subcutaneous injection; it’s the fact that we use the pharmacokinetics of subcutaneous delivery, which prolongs the half-life to achieve equivalent half-lives to the extended half-life intravenous agents.

RDR: What are Catalyst’s goals for 2018? What goals do you have personally?

Levy: My vision is to deliver normal levels greater than 50%. This is not something that has been possible before in hemophilia B. We typically talk about 3% up to 5%, and a lot of the time with extended half-lives, the patients are at risk of bleeding, especially before the next injection. We plan to have minimal levels in the normal range all the time. They will only increase a little bit over the 50% level, and this will provide the ultimate protection—not just the bleeding from joints but also mucosal bleeds. We know that bleeding occurs in the brain and, even without microscopic bleeding, in joints. Normal levels in about 50% will prevent these attributes. Our vision for hemophilia is no more bleeding. In the past, we’ve counted analyzed bleed rates and talked about the bleed rates, but our vision has to be better.

RDR: Is “better” obtainable?

Levy: We can definitely deliver more in the future for hemophilia B. Patients with inhibitors have always been the more difficult population. Emicizumab cannot treat hemophilia A with inhibitors, so hemophilia B with inhibitors is a rare disease population, but now we are among the few people working in the area to give them therapy that will normalize their lives.


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