BMS dives whole-heartedly into gene therapy with uniQure
This article was originally published in Scrip
Bristol-Myers Squibb is ready to invest more than $2bn in gene therapy, according to a deal the big pharma inked on 06 April with uniQure for its novel cardiovascular treatments and potential work in other disease areas. The news sent the biotech's shares soaring and signals further resurgence of the space.
In exchange for use of uniQure's gene therapy technology platform, which utilizes adeno-associated viruses (AAV) to move corrective genes to their intended targets and depends on an insect-cell-based manufacturing technology to create scale, BMS has agreed to pay $50m upfront as well as $45m in near-term payments for three collaboration targets.
In total, the team may work on ten gene therapy targets together, including the heart function regulator S100A1. The biotech will lead discovery efforts and be responsible for manufacturing the commercial supply.
"We believe this is one of the largest and most comprehensive collaborations in the gene therapy space," said uniQure CEO Jorn Aldag on a 06 April call with investors. "We believe that BMS chose uniQure because we have the ability to bring a gene therapy all the way to market."
The tie-up is one of many that has occurred recently as gene therapy has become a focus again after more than a decade of being out of favor with big pharma. Gene therapy is now seen as the best way for companies to develop potential game-changing treatments for diseases like hemophilia. Companies believe that current long-acting treatments for the bleeding disorder could be replaced by gene therapies that would allow patients to go months or even years without taking an injection. Both Pfizer and Novartis have teamed up with biotech's in recent years to develop gene therapies for hemophilia.
The financial details
Beyond the $95m the big pharma has agreed to pay in the near-term, BMS also opted to buy into the Dutch biotech at a price of $33.84 per share (about $32m) – approximately a 50% premium to the stock's closing price on 03 April, the final trading day before the deal became public. BMS is buying a 4.9% stake in uniQure now, as well as another 5% before the end of the year at a 10% premium. The big pharma also has the option to buy another 10% stake in the company should other targets be added to the collaboration. The deal with the big pharma sent uniQure's shares surging, adding more than 45% to trade above $33 a share.
The Dutch biotech could cash in further – it's eligible to receive $254m in development and regulatory milestones related to the lead target, S100A1, as well as $217m for milestones related to other targets. uniQure also has the potential to be paid sales milestones and tiered double-digit royalties and on any products that result.
"The financial terms of this collaboration have been structured to incentivize both companies to succeed in making important advances in the treatment of cardiovascular disease," said Aldag, who noted the cash from the deal will allow uniQure to move its own internal pipeline forward.
The biotech has a focus on liver diseases and is currently preparing for an early-stage trial in hemophilia B.