Pfizer Completes Over $600 Million Acquisition of Bamboo Therapeutics to Advance Gene Therapy Pipeline
Recently, Pfizer announced the acquisition of the biotechnology company Bamboo Therapeutics. In the first quarter of 2016, Pfizer paid approximately $43 million to acquire about 22% of Bamboo’s fully diluted equity. Under the terms of this transaction, Pfizer has acquired all remaining equity in Bamboo, with an initial payment of $150 million. Subject to the progressive milestones achieved in the development, regulatory approval, and commercialization of key assets, the selling shareholders of Bamboo will be eligible to receive up to $495 million in contingent milestone payments. With the completion of the acquisition, Bamboo has become a wholly owned subsidiary of Pfizer.
Bamboo Therapeutics is a privately held biotechnology company based in Chapel Hill, North Carolina, USA, focused on the research and development of potential gene therapies for the treatment of certain neuromuscular and rare diseases affecting the central nervous system. This acquisition significantly expands Pfizer’s expertise and capabilities in the field of gene therapy, bringing to Pfizer a clinical-stage asset and several preclinical assets that complement its rare disease portfolio, advanced recombinant adeno-associated virus (rAAV) vector design and product manufacturing technologies, as well as the full-scale I/II clinical-stage functional gene therapy manufacturing facilities that Bamboo acquired from the University of North Carolina earlier this year.
The company’s product portfolio includes best-in-class potential gene therapies based on recombinant adeno-associated virus (rAAV). This therapy will complement Pfizer’s portfolio in rare diseases and gene therapy in two priority areas: first, neuromuscular diseases, where it has a preclinical asset for Duchenne Muscular Dystrophy (DMD); and second, central nervous system disorders, where it has preclinical assets for Friedreich’s ataxia and Canavan disease, as well as a Phase I asset for giant axonal neuropathy.
The Company’s currently operational production facility occupies approximately 11,000 square feet and is fully staffed, with proven expertise in producing Phase I/II materials using high-quality suspension systems. This suspension system is a cell-based production platform that enhances process scalability, efficiency, and formulation purity. The platform supports therapeutic programs requiring large quantities of recombinant adeno-associated virus (rAAV), such as those for the treatment of Duchenne muscular dystrophy and other indications. Previously known as the University of North Carolina Vector Core Facility, this production site has served as a qualified supplier of rAAV vectors to several healthcare companies and academic institutions.
“We believe that Bamboo’s industry-leading capabilities in the design and manufacturing of recombinant adeno-associated virus (rAAV) vectors can complement Pfizer’s rare disease therapeutic strategy, helping to advance Pfizer’s mission to deliver life-changing innovative therapies to patients who need them most,” said Gregory LaRosa, Chief Scientific Officer of Pfizer’s Rare Disease Research and Development unit. “Combining the deep scientific understanding of rAAV biology and complex biomanufacturing technologies held by colleagues at both Pfizer and Bamboo will help us achieve success in this field. We are delighted to welcome our new colleagues from Bamboo to Pfizer and look forward to collaborating closely to bring innovative gene therapies to patients in areas with high unmet medical needs.”
Jude Samulski, Chief Scientific Officer and CEO of Bamboo Therapeutics, will also join Pfizer. Dr. Samulski is a leading expert in recombinant adeno-associated virus (rAAV) vectors, with over 25 years of experience in the field. Dr. Samulski and the Bamboo team will play a pivotal role in building and rapidly advancing Pfizer’s gene therapy research capabilities.
“We are delighted to begin working with Pfizer, marking a significant step forward in our journey to bring Bamboo’s product portfolio into the clinic and ultimately deliver potential new medicines to patients,” said Dr. Samulski.
Gene therapy is an emerging field of medical research focused on addressing the root causes of diseases caused by genetic mutations through highly specialized, one-time, and revolutionary treatments. Gene therapy is a promising clinical investigational technology, particularly suitable for patients with rare diseases often caused by single-gene mutations. This technique involves introducing genetic material into the body, delivering a modified copy of a gene to patient cells to compensate for genetic defects. There are various methods for delivering genetic material into cells, with viral vectors such as recombinant adeno-associated viruses being the most commonly used. To date, the United States has not approved any gene therapy products.
“In recent years, the field of gene therapy research has made tremendous progress. We are delighted to further solidify our leadership in this area through our transaction with Bamboo,” said Mikael Dolsten, President of Worldwide Research, Development and Medical at Pfizer. “We believe that gene therapy has the potential to be transformative for patients with devastating diseases. We hope to turn this promise into reality in the coming years through Pfizer’s efforts and potential collaboration opportunities.”
Pfizer is committed to becoming an industry leader in the field of gene therapy. Gene therapy holds great promise for delivering highly specific, one-time innovative therapeutics to areas with significant unmet medical needs, particularly for patients with rare monogenic diseases characterized by loss of function. To support this endeavor, Pfizer has been investing in gene therapy over the past few years, striving to integrate partners’ critical expertise and know-how in recombinant adeno-associated virus (rAAV) vector design and development with its internal disease biology knowledge, manufacturing capabilities, and analytical capacities.
In 2014, Pfizer established the Genetics Medicine Institute (GMI) within its Rare Disease Research Unit in London, UK. The institute is a specialized entity dedicated to gene therapy research under the leadership of Dr. Michael Linden, a leading scientist in the field. Dr. Linden and the GMI are responsible for identifying potential gene therapy programs and supporting them through scientific discovery, process development, and translational development.
In addition to its investment in the London-based Institute of Genetic Medicine, Pfizer has established a collaboration with Philadelphia-based Spark Therapeutics on the SPK-9001 clinical program. SPK-9001 is being investigated as a one-time treatment for hemophilia B, utilizing an engineered recombinant adeno-associated virus (AAV) vector. Preliminary data from the ongoing Phase I/II trial have shown promising early results, and the SPK-9001 clinical program has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA).
Pfizer has entered into research agreements with several leading academic institutions, including an agreement with King’s College London for the development of a series of recombinant adeno-associated virus (rAAV) gene therapy vectors, and an agreement with the University of Iowa Research Foundation to leverage the University of Iowa’s laboratories for the development of potential gene therapies for cystic fibrosis.
Pfizer has also entered into a collaboration and license agreement with 4D Molecular Therapeutics (4DMT), based in Emeryville, California, to discover and develop next-generation recombinant adeno-associated virus vectors for cardiovascular diseases. Additionally, Pfizer made an equity investment in the company in October 2015.