- The Companies are collaborating on an orderly transition through first half of 2022 while Sangamo explores options to advance the program, including seeking a new partner
- Phase 1/2 PRECIZN-1 study of investigational SAR445136 expected to be completed as planned; final patients in study expected to be dosed in third quarter of 2022
- Preliminary proof-of-concept data presented at ASH 2021 showed no treatment-related AEs and treated patients did not require blood transfusions post engraftment
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced 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. 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.
“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 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.
About Sangamo Therapeutics
Sangamo Therapeutics is a clinical-stage biopharmaceutical company with a robust genomic medicines pipeline. Using ground-breaking science, including our proprietary zinc finger genome engineering technology and manufacturing expertise, Sangamo aims to create new genomic medicines for patients suffering from diseases for which existing treatment options are inadequate or currently don’t exist. For more information about Sangamo, visit www.sangamo.com.
This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements regarding the therapeutic potential of SAR445136, including its potential to relieve people living with sickle cell disease of some of their most challenging symptoms, the anticipated completion of the PRECIZN-1 study and dosing of the final patients in the PRECIZN-1 study and the anticipated timing thereof, Sanofi’s continued funding of the PRECIZN-1 study, plans and timing for the transition of the SAR445136 program from Sanofi to Sangamo, the ability of Sangamo to identify and secure options to bring the SAR445136 program forward, including a potential new collaboration partner for the SAR445136 program, potential future collaborations between Sanofi and Sangamo, the potential for conducting future clinical trials of SAR445136 and related discussions with regulatory authorities, and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Sangamo’s actual results may differ materially and adversely from those expressed in these forward-looking statements. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to: the evolving COVID-19 pandemic and its impact on the global business environment, healthcare systems and the business and operations of Sangamo and Sanofi, including the operation of clinical trials; the research and development process; the uncertain timing and unpredictable nature of clinical trial results, including the risks that therapeutic effects observed in the preliminary proof-of-concept results from the Phase 1/2 PRECIZN-1 study will not be durable in patients and that final clinical trial data will not validate the safety and efficacy of SAR445136; reliance on results of early clinical trials, which results are not necessarily predictive of future clinical trial results; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; the manufacturing of products and product candidates; the commercialization of approved products; the potential for technological developments that obviate technologies used by Sangamo in SAR445136; the potential for Sanofi to breach its Collaboration Agreement or any related transition agreements with Sangamo; the potential for Sanofi and Sangamo to fail to come to agreement on appropriate transition agreements or to execute an orderly transition under the Collaboration Agreement; the potential that Sangamo will not be able to identify and secure options or new collaborators for the SAR445136 program; the potential for Sangamo to cease development of the SAR445136 program, whether due to its inability to secure options to bring the program forward or otherwise; Sangamo’s lack of resources to fully develop, obtain regulatory approval for and commercialize its product candidates; and the potential that Sanofi and Sangamo fail to reach agreement on and enter into potential future collaborations. These risks and uncertainties are described more fully in Sangamo's filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K for the year ended December 31, 2020 and the most recent Quarterly Report on Form 10-Q for the quarter ended September 30, 2021. The information contained in this release is as of January 6, 2022, and Sangamo undertakes no duty to update forward-looking statements contained in this release except as required by applicable laws.
Sangamo Investor Relations & Media Contact