One year ago, Takeda wrapped up its acquisition of Shire plc, which created one of the largest drug developers focused on rare diseases. Over the past year, the company strived to transform itself into becoming a frontrunner in multiple areas, including cell and gene therapies.
During the company’s 2019 R&D Investor Day, Takeda highlighted the importance of gene and cell therapies as it moves forward in its continued transformation from the Shire deal, as well as other collaborations the company has forged since. However, in what might be considered a refreshing moment of real honesty, Andy Plump, Takeda’s head of R&D, recently noted that sometimes being on the cutting edge of treatments does not mean your treatment will be the top-selling preference. In an interview with the Boston Business Journal, Plump revealed that he did not believe the company’s hemophilia A gene therapy treatment would be a market leader. In fact, he went so far as to say the treatment would probably not even be a contender.
“I think the likelihood is that our hemophilia A gene therapy is not going to be a competitor in the market… But I don’t think you need to be a frontrunner in a field to really be effective. In fact, I think, sometimes in the world we live in, being the frontrunner can have disadvantages,” he told the Journal.
In its gene therapy pipeline, Takeda is developing TAK-754 for hemophilia A. The asset is currently in a Phase I study. The company also has TAK-748 in preclinical studies for treatment of hemophilia B. As the Journal reported, the company sees a strong future for its gene therapy programs and considers them a future driver of business. While the Journal’s article did not go into specifics as to why Plump feels that the company’s gene therapy won’t be a market driver in hemophilia, it’s likely due to the fact that it is still in the early stages of development. Spark Therapeutics, a division of Roche, is moving its gene therapy treatment into late-stages of study following highly impressive data that showed a one-time treatment yielded a 97% response ratein reduced bleeding events in hemophilia A patients. When Roche made its bid for Spark, the Swiss pharma giant saw that company’s gene therapies for hemophilia as a complement to its own products for the bleeding disorder, including hemophilia treatment Hemlibra.
While the hemophilia treatment may not be a competitor in the marketplace, Plump told the Journal that Takeda is looking at bringing nearly a dozen treatments to market by 2024. Those treatments are aimed at conditions such as “cytomegalovirus infections, a rare disease called Hunter Syndrome and complications of premature birth.” Takeda believes these therapies can drive a combined $10 billion in peak sales, the Journal said.
In September, Takeda published the results of a Phase II study of TAK-620 (maribavir) for cytomegalovirus (CMV). The drug is designed to target a specific CMV protein, which may lead to inhibition of CMV DNA replication and encapsidation.
Additionally, Takeda has remained busy inking collaborations with multiple companies to advance its drug development programs. Earlier this month, the company struck a multi-year drug discovery deal with Charles River Laboratories. The companies will develop potential drug candidates across Takeda’s four core therapeutic areas—oncology, gastroenterology, neuroscience and rare disease. Only weeks prior to the deal with Charles River, Takeda forged a collaboration worth up to $1 billion with Turnstone Biologics to tackle a number of cancer indications using that company’s vaccinia virus platform. The company and Cerevance teamed up to tackle diseases of the gastrointestinal tract that have their roots in the central nervous system.
