- Sanofi has ended a long-running alliance with Sangamo Therapeutics to develop genetic medicines for inherited blood disorders, among them an experimental sickle cell disease therapy that is in early clinical testing.
- In a regulatory filing, Sangamo disclosed the French pharma will end the deal on June 28 as part of a "change in strategic direction." The two have been developing complex, personalized treatments, led by a sickle cell drug known as SAR445136. But Sanofi is now more interested in off-the-shelf approaches, which are meant to be more convenient.
- The decision concludes a partnership Sangamo originally formed with Biogen in 2014 and Sanofi inherited when it acquired Biogen's blood disease spinout, Bioverativ, four years later. But Sangamo has received less than $14 million of the roughly $276 million in conditional payments that were spelled out in the deal, which Sanofi ended one month after Sangamo delivered the first clinical results for its sickle cell drug.
Through a spate of dealmaking over the past few years, Sanofi has made its intentions in the gene and cell therapy field clear. The company seems uninterested in hard-to-manufacture treatments that consist of individual patient cells genetically modified outside the body, instead favoring ones meant to be used "off the shelf."
In 2020, for instance, the French drugmaker bought Kiadis Pharma, which is working on more convenient cell treatments for cancer. Sanofi acquired another off-the-shelf cell therapy developer, Tidal Therapeutics, the following year, and envisions its recent investments in messenger RNA technology as aiding in the effort.
In doing so, Sanofi isn't just bypassing the first generation of so-called CAR-T therapies, which are powerful tools against certain blood cancers but are limited by their complexity. It's also casting aside outside-the-body treatments for genetic diseases, a class that includes drugs from CRISPR Therapeutics and Bluebird bio that have shown considerable promise treating blood diseases like beta thalassemia and sickle cell.
The approach comes with some risks. While off-the-shelf methods could help broaden the reach of their more complex counterparts, they haven't yet proven to be as potent or long-lasting. Safety questions have also emerged following the Food and Drug Administration's decision to pause all of the ongoing clinical tests from Allogene Therapeutics, one of the field's leaders.
Sanofi jettisoned Sangamo's program just as clinical testing had begun. Initial study results Sangamo reported in December showed its treatment — which relies on a form of gene editing known as zinc finger technology — promises to reduce the risk of the painful episodes that characterize sickle cell disease. But Sangamo is far behind the leading gene editing therapy from CRISPR, which could be submitted to U.S. regulators this year. The study findings disclosed so far also appeared "marginally worse" than CRISPR's treatment, according to a note from Stifel analyst Benjamin Burnett.
"We speculate that even while the data convey some benefit, [Sanofi] may have viewed the commercial setup as challenging, motivating the decision to walk away," Burnett wrote.
In a statement, Sanofi research chief John Reed noted the move is "not a reflection on the potential" of Sangamo's drug. Sanofi is focusing on "universal genomic medicine approaches," he said, and will explore "other possible collaboration opportunities" while the program is transitioned back to Sangamo.
Nonetheless, the move is a setback for Sangamo's work in blood diseases. In October, Sanofi and Sangamo ended work on a potential beta thalassemia program, while the FDA suspended a Phase 3 study of an experimental hemophilia treatment a month later.
Sanofi will continue to pay the costs of the ongoing sickle cell trial until the deal ends in June, and still expects to treat the study's final patients in the third quarter. But the company said it will evaluate other options for the drug in the meantime, including seeking a new development partner.
Sangamo shares fell about 9% Thursday morning, before regaining some of those losses later on.