On January 6, 2022 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, reported that Sanofi will be transitioning its rights and obligations related to SAR445136, a zinc finger nuclease gene-edited cell therapy candidate in development by Sangamo and Sanofi for the treatment of sickle cell disease (SCD), back to Sangamo over the first half of 2022 (Press release, Sangamo Therapeutics, JAN 6, 2022, View Source [SID1234607774]). The Companies are collaborating on an orderly transition, while Sangamo explores options to advance the program, including seeking a new collaboration partner. This transition follows Sanofi’s termination for convenience of the Global Research, Development and Commercialization Collaboration and License Agreement (Collaboration Agreement) between the Companies to develop genomic medicines for hemoglobinopathies. Sanofi has elected to transition the SCD program to Sangamo following a recent change in Sanofi’s cell therapy strategy.
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"Although the preliminary Phase 1/2 clinical data for the autologous sickle cell treatment are encouraging, Sanofi has made the decision to terminate the collaboration on the SAR445136 program, which is consistent with our strategy to focus on universal genomic medicine approaches," said John Reed, M.D. Ph.D., Global Head of Research and Development at Sanofi. "Sangamo has been a good partner and this decision is not a reflection on the potential of the SAR445136 program. We continue to view them as a pioneer in the area of genomic medicines and will explore other possible collaboration opportunities as we work together to transition the autologous sickle cell program back to Sangamo."
"We remain committed to progressing this program and believe SAR445136 has the potential to relieve people living with sickle cell disease of some of their most challenging symptoms. We appreciate Sanofi’s collaboration in advancing the SAR445136 program and presenting promising preliminary proof-of-concept clinical data. We expect the Phase 1/2 study to be completed as planned, for final patients in the study to be dosed in the third quarter of 2022, and for discussions regarding potential future clinical trials to continue with health authorities," said Sandy Macrae, CEO of Sangamo. "We will vigorously investigate alternative options to bring this genomic medicine forward to patients."
Sanofi notified Sangamo of its termination for convenience on December 30, 2021. Sangamo expects the Phase 1/2 PRECIZN-1 study of SAR445136 to be completed as planned. Sangamo expects that Sanofi will continue to pay the costs of the Phase 1/2 PRECIZN-1 study until the termination date of June 28, 2022, as contemplated by the Collaboration Agreement.
About the PRECIZN-1 Study
PRECIZN-1 is an ongoing first-in-human, open label, single arm, multi-site Phase 1/2 study in up to eight patients with SCD evaluating the safety and tolerability of cell therapy candidate SAR445136. The therapeutic product is manufactured by ex vivo gene editing of a patient’s own (autologous) hematopoietic stem cells using non-viral delivery of zinc finger nuclease technology targeting the BCL11a gene erythroid-specific enhancer (ESE) to increase endogenous fetal hemoglobin (HbF) production. SAR445136 has received Fast Track Designation from the FDA and Orphan Medicinal Product from the EMA.
Preliminary proof-of-concept results presented at ASH (Free ASH Whitepaper) 2021 as of the cutoff date September 22, 2021 showed that no adverse events (AEs) related to investigational SAR445136 were reported through 91 weeks for the longest-treated patient and through 26 weeks for the most recently treated patient. All four treated patients experienced increases in total hemoglobin, fetal hemoglobin and percent F cells and none required blood transfusions post engraftment. Total hemoglobin stabilized by Week 26 after treatment with SAR445136 in all four patients. Most AEs reported in the screening, mobilization, apheresis and conditioning periods were SCD-related events. One serious adverse event of sickle cell anemia with crisis (vaso-occlusive crisis or VOC) was reported approximately nine months after treatment with SAR445136 in one patient, and no other SCD-related events were reported in the four patients post-infusion. The poster presentation is available on Sangamo’s website in the Investors and Media section under Events and Presentations.