- The stock price of uniQure (QURE) increased by over 6% intraday today. This is why.
The stock price of uniQure (QURE) increased by over 6% intraday today.
Why: uniQure announced that its partner, global biotechnology leader CSL (CSL), has received approval from the U.S. Food and Drug Administration (FDA) for HEMGENIX (etranacogene dezaparvovec-drlb), a one-time gene therapy for the treatment of adults 18 years of age and older living with hemophilia B.
The product was approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. And CSL licensed the exclusive global rights to HEMGENIX from uniQure in May 2021 and is now solely responsible for the further development, registration, and commercialization of the therapy.
Hemophilia B is a rare and lifelong bleeding disorder caused by a single gene defect, resulting in insufficient production of factor IX, a protein primarily produced by the liver that helps blood clots form. And the treatments for moderate to severe hemophilia B include prophylactic infusions of factor IX replacement therapy to temporarily replace or supplement low levels of blood-clotting factor and, while these therapies are effective, those with hemophilia B must adhere to strict, lifelong infusion schedules. They may also still experience spontaneous bleeding episodes as well as limited mobility, joint damage or severe pain as a result of the disease. For appropriate patients, HEMGENIX allows people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding.
In the ongoing clinical trial, HEMGENIX reduced the rate of annual bleeds and 94% of patients discontinued factor IX prophylaxis and remained prophylaxis-free.
uniQure had conducted the research and clinical development for the product, which included three clinical trials across 34 global sites and involving 67 adults with hemophilia B. And in May 2021, uniQure and CSL completed a licensing transaction providing CSL Behring with exclusive rights to commercialize and continue clinical development of HEMGENIX globally. uniQure is responsible for the global manufacturing of the product at its licensed Lexington, MA facility.
Under the terms of the deal, uniQure has received payments from CSL totaling approximately $500 million and is eligible to receive up to an additional $1.5 billion in commercial milestone payments and tiered, double-digit royalties in a range up to a low-twenties percentage of net product sales arising from the collaboration.
The FDA approval is supported by results from the pivotal HOPE-B trial, the largest gene therapy trial in hemophilia B to date. And the results from the study demonstrated that HEMGENIX allowed patients to produce mean factor IX activity of 39% at 6 months and 36.7% at 24 months post infusion. And 7 to 18 months post-infusion, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by 54% compared to the six-month lead-in period on factor IX prophylactic replacement therapy (4.1 to 1.9). In addition, 94% (51 out of 54) of patients treated with HEMGENIX discontinued use of prophylaxis and remained free of previous continuous routine prophylaxis therapy. The most common side effects (incidence ≥5%) were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea and feeling unwell.
“Today’s landmark approval represents a major milestone in the field of genomic medicine and ushers in a new treatment paradigm for patients living with hemophilia B. This is uniQure’s second internally-developed gene therapy to achieve approval and the world’s first gene therapy for hemophilia B, an historic achievement based on more than a decade of research and clinical development, as well as the tireless dedication of our employees, clinicians, patients and their families. For nearly 25 years, uniQure has been driven by a singular mission: to transform peoples’ lives by harnessing the power of gene therapy. This groundbreaking approval delivers on this promise by providing people with hemophilia B the possibility of being liberated from burdensome infusions and an ability to unlock the potential in their daily lives. We offer our sincere gratitude to the hemophilia community, without whose years of dedication and invaluable support this medical breakthrough could not have been achieved.”
— Matt Kapusta, chief executive officer of uniQure
“CSL is proud to have been at the forefront of providing life-changing medicines for rare diseases for over a century. HEMGENIX, originally developed by our uniQure colleagues, is just such a medicine. Today’s historic approval builds on CSL’s promise to put patients first in all that we do to discover, develop and deliver biotherapeutics and vaccines that meet their needs. With HEMGENIX, CSL now offers people living with Hemophilia B another remarkable option for better and more durable control over their disease.”
— Dr. Bill Mezzanotte, head of research & development and chief medical officer of CSL
“We are thrilled to witness this milestone in hemophilia B treatment. Over the years we have seen a variety of advancements for the hemophilia community, but gene therapy is the first treatment option to offer those living with hemophilia B and caregivers the possibility of freedom from the need for regular, ongoing infusions.”
— Kim Phelan, chief operating officer of the Coalition for Hemophilia B
“HEMGENIX is unique in its approach to increase mean factor IX activity and hemostatic protection in those with hemophilia B, and today’s approval could fundamentally transform the treatment paradigm for this life-long condition. As a clinician, I look forward to being able to provide a new treatment option that may help patients treated with HEMGENIX become free from the regular infusion schedule that many people living with hemophilia B rely on to protect them from the debilitating effects of the condition.”
— Dr. Steven Pipe, professor and the Laurence A. Boxer Research professor of pediatrics and professor of pathology at the University of Michigan and a lead investigator in the HOPE-B study