CSL Behring Bolsters Hemophilia Lineup With Deal for uniQure’s Gene Therapy

Xconomy Philadelphia — 

CSL Behring is joining the chase for a genetic treatment for hemophilia by acquiring a uniQure gene therapy that’s already in late-stage testing.

King of Prussia, PA-based CSL Behring is paying $450 million up front to acquire the uniQure (NASDAQ: QURE) asset, etranacogene dezaparvovec (“EtranaDez” for short), the companies announced late Wednesday. Netherlands-based uniQure will complete the Phase 3 test of the hemophila B gene therapy and will also be responsible for submitting an application for FDA review, expenses that CSL Behring has agreed to reimburse. If the bleeding disorder therapy reaches the market, the agreement calls for the company to pay uniQure up to $1.6 billion for regulatory and commercialization milestones, plus royalties from sales.

CSL Behring, a subsidiary of Australia-based CSL Limited, has extensive experience in bleeding disorders, including hemophilia B, which is caused by a mutation to the gene that codes for a clotting protein called factor IX (FIX). Standard treatment for the condition includes regular infusions of the FIX protein from products such as those currently marketed by CSL Behring. A gene therapy offers the prospect of a potential one-time fix for the inherited disorder.

The uniQure gene therapy is comprised of a patent-protected version of the FIX gene delivered using an engineered adeno-associated virus. During the annual meeting of the American Society of Hematology last year December, uniQure reported mid-stage results showing that one year after receiving the gene therapy, the average FIX activity in the three patients studied was 41 percent of what a person without the condition would produce. Furthermore, none of the patients had reports of bleeding or required preventative measures to guard against such episodes.

The acquisition of EntranaDez puts CSL Behring in the mix of companies vying to commercialize gene therapies for hemophilia. Valoctocagene roxaparvovec, a BioMarin Pharmaceutical (NASDAQ: BMRN) gene therapy developed to treat the more common hemophilia A, is expected to receive an FDA decision by late August. Hemophilia A is caused by a mutation to the gene that codes for factor VIII, another clotting protein. Meanwhile, Pfizer (NYSE: PFE) and partner Sangamo Therapeutics (NASDAQ: SGMO) continue to make progress with their hemophilia A gene therapy, giroctocogene fitelparvovec. Last week, the companies reported Phase 1/2 results showing that all five patients who received a dose of the investigational treatment showed sustained activity of the factor VIII protein. No patients experienced bleeding or required infusions of the protein, the companies said.

In a research note to investors, SVB Leerink analysts said that while they expected EntranaDez to post successful results later this year, CSL Behring’s long track record in bleeding disorders makes the company a natural fit for commercializing the gene therapy. Furthermore, the licensing agreement is “an important strategic move for [uniQure] which enables acceleration of other —and potentially more attractive—pipeline assets,” they wrote.

UniQure said in an investor presentation that its deal with CSL Behring puts EntranaDez in the hands of a company with extensive commercial experience in selling hemophilia products and the global reach to make the gene therapy quickly available to as many patients as possible. With the gene therapy set to change hands, uniQure said it will “deprioritize” AMT-180, its research program for hemophilia A. But the company says it can now focus on the rest of its pipeline, including Huntington’s disease gene therapy candidate AMT-130. UniQure says the first patients in a Phase 1/2 study were dosed this month. The company expects to report safety data later this year and preliminary efficacy data in 2021.

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