The globally recognised medicine company, Pfizer (PFE, Financial), announced the agreement with privately held U.S. company, Spark Therapeutics to find a drug to treat haemophilia by adopting the gene therapy platform. Pfizer has been focusing on introducing drugs for treatment of rare diseases for more than a decade now and it has a global portfolio of 22 medicines approved worldwide that treats rare diseases in the areas of hematology, neuroscience, inherited metabolic disorders, pulmonology and oncology. This candid move will mark Pfizer’s expansion into the field of gene therapy in the latest sign that the technology for fixing faulty genes may finally be ready for prime time, following earlier setbacks. Let’s dive in and find out what benefits Pfizer would get from the association and what it wants to achieve in this new domain of interest.
The collaboration with Spark
On December 8, the U.S. drug maker made public that it is establishing a gene therapy platform to study potential treatments, led by a top UK expert, and has struck a deal with privately owned U.S. biotech firm Spark Therapeutics to develop a treatment for haemophilia. The Spark program is expected to enter early stage clinical trials for haemophilia B in the first half of the coming year. The biotech firm would be responsible for the Phase I/II tests, with Pfizer taking over the late-stage studies, any regulatory approvals and potential commercialisation. Spark would be entitled to receive double-digit royalties based on global product sales.
Spark would be given $20 million upfront and would be eligible for additional payments based on product success worth up to $260 million. Through this global collaboration development of SPK-FIX would be tried out, which could lead to potential treatment of haemophilia B. CEO of Spark, Jeffrey D. Marrazzo, shared, “We are excited to announce our collaboration with Pfizer, as we believe it marks an important step towards bringing a potentially life-altering therapeutic to patients with hemophilia B. … The collaboration also marks another milestone for Spark, following our recent clinical and regulatory progress and key leadership hires."
Why the drug is vital for treatment
Haemophilia B is a rare genetic blood disorder that affects approximately 4,000 males in the U.S. and about 26,000 males worldwide. Current treatment is painful requiring recurrent intravenous infusions of either plasma-derived or recombinant Factor IX to control bleeding episodes. Spark’s proprietary, bio-engineered vectors are designed to deliver a high-activity Factor IX gene to patients, allowing endogenous production of Factor IX, with the potential to remain effective for a number of years.
This program leverages a long track record of haemophilia B gene therapy research and clinical development conducted by Spark’s scientific team over the past two decades. Spark’s scientists are of this opinion when they commented on the association with Pfizer: “Pfizer's longtime experience in hemophilia, including strong relationships with physicians, patients and payors, as well as clinical, regulatory and commercial capabilities, will complement our team's deep knowledge of AAV-mediated gene transfer for the disease. We look forward to working with Pfizer with the goal of making gene therapy for hemophilia B a reality for patients.”
What Pfizer has to say
“By establishing our gene therapy capabilities, we hope to gain a deeper understanding of the mechanisms that could potentially bring true disease modification for those suffering from devastating hematologic and neuromuscular diseases”, stated Pfizer’s R&D Chief Mikael Dolsten.
Pfizer’s move is the latest affirmation of gene therapy’s renewed lustre among the world’s largest drug makers. Pfizer is indeed in the face of competition in this life science domain as Biogen Idec (BIIB, Financial) has already made a splash in this sector by unveiling plans to build an internal gene therapy division and other drug developers such as Bayer AG (BAYN:GR, Financial), GlaxoSmithKline (GSK, Financial) and Baxter (BAX, Financial) are working on in-house treatments in the field.
Final word
To date, Pfizer has been on an exclusive growth trajectory, and this expansion plan of the well-acclaimed drug maker might help it to see shining days in the near future if the Spark program sees the light of success. Let’s stay tuned and watch how Pfizer and Spark association ultimately creates a difference in the domain of life sciences.
