After handing over the development of the gene therapy candidate SB-525 to Pfizer, Sangamo Therapeutics has now concluded its collaboration agreement with Biogen to advance and market gene therapies for Alzheimer’s and Parkinson’s diseases, and other neuromuscular and other neurological disorders.
Among other goals, the funding will support a potential application seeking approval of SB-525 for people with hemophilia A in the U.S., Sangamo said.
Under the terms of the agreement, Sangamo was paid $225 million, and will receive an additional upfront license fee of $125 million from Biogen.
In addition, Sangamo is eligible to receive up to $2.37 billion upon the completion of certain developmental, regulatory, and commercial milestones. These include up to $925 million in pre-approval milestone payments and up to $1.445 billion in commercial sales. The company also is eligible for royalties on net sales of all products arising from the partnership with Biogen.
“The Biogen transaction exemplifies our strategy to partner assets when we believe that a collaborator’s financial resources and clinical and therapeutic area expertise will enable more rapid development and availability of new treatments to patients,” Sandy Macrae, CEO of Sangamo, said in a press release.
“Importantly, with the addition of the upfront consideration from this agreement, we believe we have the balance sheet strength to execute on our wholly owned and partnered development programs through multiple important milestones, including the potential filing of the BLA [biologics license application] for SB-525 for hemophilia A,” Macrae said.
SB-525 is a one-time gene therapy intended to restore the production of factor VIII (FVIII) in the liver to remove, or at least minimize, the need for replacement therapies. FVIII is the blood clotting protein missing in people with hemophilia A.
The therapy previously received orphan drug, fast track, and regenerative medicine advanced therapy designations from the U.S. Food and Drug Administration (FDA), as well as orphan medicinal product designation from the European Medicines Agency.
Although it is leading a Phase 2 trial (NCT03061201) to test the safety and tolerability of SB-525, Sangamo transferred the therapy’s investigational new drug application to Pfizer. This filing is a necessary step to receive clearance to conduct clinical trials by the FDA.
Pfizer is sponsoring a six-month, lead-in Phase 3 trial (NCT03587116) expected to result in a future registrational Phase 3 study of SB-525. A registrational study is designed to support regulatory approval of a therapy.
The current trial is evaluating the effectiveness and safety of preventive replacement therapies used in the usual care setting to establish a baseline for the future registrational study. It also will support the advancement of a Phase 3 trial of fidanacogene elaparvovec (SPK-9001), a gene therapy currently being investigated to treat patients with hemophilia B.
Recruitment for both the Phase 2 and Phase 3 studies may be ongoing, although many trials are on temporary hold during the current COVID-19 pandemic. For more information about contacts and locations, click on trial’s identifiers (NCT links).
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