Pfizer commits new resource to gene therapy
Pfizer Inc is prepared to commit up to $280 million to an experimental gene therapy for haemophilia which is under development by Spark Therapeutics in the US, while at the same time reinforcing its gene therapy research capacity in the UK.
Pfizer Inc is prepared to commit up to $280 million to an experimental gene therapy for haemophilia which is under development by Spark Therapeutics in the US, while at the same time reinforcing its gene therapy research capacity in the UK.
The US multinational announced the two initiatives on 8 December. The decisions effectively give gene therapy a new strategic importance by positioning the programmes within Pfizer’s rare disease business unit. Pfizer is one the few Big Pharma companies with a dedicated rare disease unit. There are an estimated 7,000 rare disease in the world today of which about 80% are understood to be caused by gene mutations. This would make them theoretically amenable to treatment, if not cure, by gene therapy.
The agreement with Spark focuses on an existing programme at the Philadelphia-based company under which bio-engineered adeno-associated virus vectors are being investigated for their potential treatment of haemophilia B. The vectors have been designed to deliver Factor IX gene to patients with the goal of enabling the endogenous production of the factor. The treatment would be expected to last for a number of years.
Spark will receive an upfront payment of $20 million from Pfizer, and will also be eligible for development and commercialisation milestones of up to $260 million. The potential milestones relate to multiple haemophilia B product candidates that may be developed under the collaboration.
Spark will be responsible for all Phase 1/2 studies while Pfizer will assume responsibility for pre-registration studies, regulatory submissions and the potential global commercialisation of a product. Spark is also eligible for double-digit royalties on any sales.
In a separate decision, Pfizer has expanded its gene therapy presence in the UK with the appointment of Michael Linden as head of gene therapy research within the company’s rare disease research unit. Mr Linden is a professor at King’s College, London and director of the University College London Gene Therapy Consortium. Prof Linden joined Pfizer on 1 December on a two-year secondment from academia.
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