Getting to the heart of gene therapy

Bristol-Myers Squibb and uniQure to explore gene therapy for cardiovascular diseases

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NEW YORK & AMSTERDAM—In what uniQure CEO Jörn Aldag says is a deal “consistent with our strategy of expanding gene therapy beyond orphan indications and into common diseases,” the Amsterdam-based company and New York-based pharma giant Bristol-Myers Squibb Co. (BMS) in early April announced an agreement that provides BMS with exclusive access to uniQure N.V.’s gene therapy technology platform for multiple targets in cardiovascular disease.
“We believe this is one of the largest and most comprehensive collaborations in the gene therapy sector to date,” noted Aldag in a webcast about the deal. “We chose to collaborate with BMS because of their excellent and longstanding track record of success in discovering and developing treatments for cardiovascular diseases. They are also the perfect partner for this mission because of their passion for embracing advanced technologies for the treatment of human disease, as witnessed by their position in immuno-oncology. Our part in this mission is to continue employing our expertise and platform to develop and make these exciting new gene therapy products.”
As for why BMS chose uniQure, Aldag sees it as being in large part due to his company’s experience in bringing a gene therapy product all the way to market, “and that we have the most comprehensive platform of manufacturing technologies and vectors to do it again. We also bring to the collaboration our world-class manufacturing facility that is built to provide the capacity of producing quantities of gene therapy product to even support a launch of a product in a disease with a population as large as the one of congestive heart failure.”
The collaboration includes uniQure’s proprietary gene therapy program for congestive heart failure, which aims to restore the heart’s ability to synthesize S100A1, a calcium sensor and master regulator of heart function, and thereby improve clinical outcomes for patients with reduced ejection fraction. Beyond cardiovascular diseases, the agreement also includes the potential for target-exclusive collaboration in other disease areas. Ultimately, the companies could collaborate on as many as 10 targets, including S100A1.
How much money this means for uniQure is pretty open-ended, but it could mean something in the neighborhood of $1 billion or more for BMS in terms of its investment if all goes well in terms of research, development, milestones and the like. Admittedly, though, the short-term outlay is more modest. Under the terms of the agreement, BMS will make near-term payments of approximately $100 million, including an upfront payment of $50 million to be made at the closing of the transaction, a $15-million payment for the selection of three collaboration targets, in addition to S100A1, to be made within three months of the closing and an initial equity investment in uniQure for a number of shares that will equal 4.9 percent of the total number of shares outstanding following such issuance, at a purchase price of $33.84 per share, or at least $32 million in total. Bristol-Myers-Squibb will acquire an additional 5 percent of ownership before Dec. 31, 2015, at a 10-percent premium, and will be granted two warrants to acquire up to an additional 10 percent in terms of equity interest, at a premium, based on additional targets being introduced into the collaboration. The parties have also agreed to enter into a supply contract, under which uniQure will undertake manufacturing of all gene therapy products under the collaboration.
In addition, uniQure will be eligible to receive research, development and regulatory milestone payments, including up to $254 million for the lead S100A1 therapeutic and up to $217 million for each other gene therapy product potentially developed under the collaboration. Moreover, uniQure is eligible to receive net sales-based milestone payments and tiered single- to double-digit royalties on product sales.
The deal calls for uniQure to lead discovery efforts and be responsible for manufacturing of clinical and commercial supplies using its vector technologies and its industrial, proprietary insect cell-based manufacturing platform. Bristol-Myers Squibb will lead development and regulatory activities across all programs and be responsible for all research and development costs, and BMS also will be solely responsible for commercialization of all products from the collaboration.
“Bristol-Myers Squibb has an excellent and longstanding track record of success in discovering and developing treatments for cardiovascular diseases and in embracing advancing technologies for the treatment of human diseases,” said Dr. Carl Decicco, head of discovery and R&D at BMS. “Collaborating with uniQure, a clear leader in the field with an innovative and validated gene therapy platform, further strengthens our capability to bring forward transformational new therapeutics for difficult-to-treat diseases, including cardiovascular diseases such as heart failure.”
“Bristol-Myers Squibb’s strength in the cardiovascular area and its commitment to gene therapy will allow them to leverage the full breadth and capacity of our platform for cardiovascular diseases,” said uniQure’s Aldag. “This collaboration will accelerate the application of gene therapy for large patient populations suffering from heart diseases and will complement the further development of uniQure’s internal pipeline in two focus areas: liver diseases, including hemophilia, and CNS, including lysosomal storage diseases.”
After the announcement, Piper Jaffray analyst Joshua Schimmer lifted his target price for uniQure stock from $35 to $40 and wrote, “The importance of this deal is multi-fold, in that it: 1) helps validate [uniQure’s] gene therapy platform and baculovirus manufacturing capabilities and highlights the company's leadership in this field; 2) locks in value for the S100A1 program ahead of [Celadon’s] P2b data in April; 3) helps takes the pressure off Glybera EU launch and sets up the stock for hemophilia B data in 2H15; and 4) includes an equity deal at $33.84/share well ahead of latest close of $22.86/share.”

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