– Based on positive results from Phase 3 AFFINE trial, which met primary and secondary endpoints, Sangamo plans to explore all options to commercialize the asset, including seeking a potential new collaboration partner
RICHMOND, CA, USA I December 30, 2024 I Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced it will regain development and commercialization rights to giroctocogene fitelparvovec, an investigational gene therapy product candidate for the treatment of adults with moderately severe to severe hemophilia A that it has co-developed with, and licensed to Pfizer Inc., following a decision by Pfizer to terminate the global collaboration and license agreement between the parties. Sangamo intends to explore all options to advance the program, including seeking a potential new collaboration partner. Pfizer has indicated to Sangamo that this termination reflects its decision not to proceed with the Biologics License Application (BLA) and Marketing Authorisation Application (MAA) submissions for, or to pursue commercialization of, giroctocogene fitelparvovec.
In July 2024, Pfizer announced positive topline results from the Phase 3 AFFINE trial evaluating giroctocogene fitelparvovec that demonstrated the trial met primary and key secondary objectives of superiority compared to prophylaxis. On December 9, 2024, Pfizer presented detailed AFFINE data in an oral presentation at the 66th ASH Annual Meeting and Exposition, with the presentation selected as part of the “Best of ASH” program, which highlights the most cutting-edge science presented during the Annual Meeting. In November 2024, Pfizer indicated to Sangamo that Pfizer was discussing these data with regulatory authorities. Pfizer previously indicated to Sangamo that anticipated BLA and MAA submissions were expected to occur in early 2025.
“Giroctocogene fitelparvovec has demonstrated the potential to be a life changing gene therapy treatment for hemophilia A patients, and following positive results from the Phase 3 AFFINE trial, we believe it is well positioned for regulatory submissions and potential commercialization,” said Sandy Macrae, Chief Executive Officer of Sangamo Therapeutics. “While we were surprised and extremely disappointed by Pfizer’s decision to end our collaboration so close to the anticipated BLA and MAA submissions, especially given the compelling pivotal clinical trial data, we appreciate their collaboration in leading a robust and successful clinical development program and for advancing the asset to this important stage. We are committed to exploring the optimal path forward for this important treatment, including seeking the right partner with the focus and understanding of the genomic medicine commercial environment to bring this medicine to patients. In parallel, we remain focused on advancing our wholly owned neurology genomic medicine pipeline and progressing our Fabry gene therapy program towards a potential BLA submission in the second half of 2025.”
The collaboration and license agreement with Pfizer will terminate effective April 21, 2025, at which time Pfizer will be required to transition the giroctocogene fitelparvovec program back to Sangamo. All trial participants will continue to be monitored as planned during the transition period.
Sangamo believes that its recently announced partnerships with Genentech and Astellas, and advanced business development discussions for its Fabry gene therapy program, will allow it to chart a path forward for its neurology genomic medicine pipeline as it prepares to initiate expected enrollment of patients in the Phase 1/2 study of ST-503 for idiopathic small fiber neuropathy in mid-2025, and file an anticipated Clinical Trial Authorisation (CTA) submission for the prion disease program in Q4 2025, each subject to securing adequate additional funding.
About the AFFINE Trial
The Phase 3 AFFINE (NCT04370054) trial is an open-label, multicenter, single-arm trial to evaluate the efficacy and safety of a single infusion of giroctocogene fitelparvovec in adult male participants (n=75 dosed participants) with moderately severe to severe hemophilia A. Trial participants included in the assessments of the key endpoints of the primary efficacy analysis (n=50) completed a minimum six months of routine FVIII replacement prophylaxis therapy during the lead-in study (NCT03587116) providing data to compare with post giroctocogene fitelparvovec treatment.
In July 2024, Pfizer announced that the AFFINE trial achieved its primary objective of non-inferiority, as well as superiority, of total annualized bleeding rate (ABR) from Week 12 through at least 15 months of follow up post-infusion compared with routine Factor VIII (FVIII) replacement prophylaxis treatment. Following a single 3e13 vg/kg dose, giroctocogene fitelparvovec demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period (1.24 vs 4.73; one-sided p-value=0.0040). Key secondary endpoints as defined by the trial protocol were met and also demonstrated superiority compared to prophylaxis. In the AFFINE trial, giroctocogene fitelparvovec was generally well tolerated.
Giroctocogene fitelparvovec has been developed as part of a collaboration agreement for the global development and commercialization of gene therapies for hemophilia A between Sangamo Therapeutics and Pfizer. In late 2019, Sangamo transferred the manufacturing technology and the Investigational New Drug application to Pfizer. Under the agreement, Pfizer assumed responsibility for pivotal studies, any regulatory activities, and potential global commercialization of giroctocogene fitelparvovec.
About Hemophilia A
Hemophilia is an inherited, rare bleeding disorder that causes people to bleed for longer than normal due to a deficiency of a protein required for normal blood clotting, known as clotting Factor VIII (FVIII) in hemophilia A. The severity of hemophilia is determined by the amount of the factor in the blood. The lower the amount of the factor, the more likely it is that bleeding will occur, which can lead to serious health problems.
Hemophilia A occurs in approximately 25 in every 100,000 male births worldwide. Approximately 55-75% of males with hemophilia A have a moderate to severe form of the disease. For people who live with hemophilia A, there is an increased risk of spontaneous bleeding as well as bleeding following injuries or surgery. It is a lifelong disease that requires constant monitoring and therapy.
SOURCE: Hemophilia
Post Views: 349
– Based on positive results from Phase 3 AFFINE trial, which met primary and secondary endpoints, Sangamo plans to explore all options to commercialize the asset, including seeking a potential new collaboration partner
RICHMOND, CA, USA I December 30, 2024 I Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced it will regain development and commercialization rights to giroctocogene fitelparvovec, an investigational gene therapy product candidate for the treatment of adults with moderately severe to severe hemophilia A that it has co-developed with, and licensed to Pfizer Inc., following a decision by Pfizer to terminate the global collaboration and license agreement between the parties. Sangamo intends to explore all options to advance the program, including seeking a potential new collaboration partner. Pfizer has indicated to Sangamo that this termination reflects its decision not to proceed with the Biologics License Application (BLA) and Marketing Authorisation Application (MAA) submissions for, or to pursue commercialization of, giroctocogene fitelparvovec.
In July 2024, Pfizer announced positive topline results from the Phase 3 AFFINE trial evaluating giroctocogene fitelparvovec that demonstrated the trial met primary and key secondary objectives of superiority compared to prophylaxis. On December 9, 2024, Pfizer presented detailed AFFINE data in an oral presentation at the 66th ASH Annual Meeting and Exposition, with the presentation selected as part of the “Best of ASH” program, which highlights the most cutting-edge science presented during the Annual Meeting. In November 2024, Pfizer indicated to Sangamo that Pfizer was discussing these data with regulatory authorities. Pfizer previously indicated to Sangamo that anticipated BLA and MAA submissions were expected to occur in early 2025.
“Giroctocogene fitelparvovec has demonstrated the potential to be a life changing gene therapy treatment for hemophilia A patients, and following positive results from the Phase 3 AFFINE trial, we believe it is well positioned for regulatory submissions and potential commercialization,” said Sandy Macrae, Chief Executive Officer of Sangamo Therapeutics. “While we were surprised and extremely disappointed by Pfizer’s decision to end our collaboration so close to the anticipated BLA and MAA submissions, especially given the compelling pivotal clinical trial data, we appreciate their collaboration in leading a robust and successful clinical development program and for advancing the asset to this important stage. We are committed to exploring the optimal path forward for this important treatment, including seeking the right partner with the focus and understanding of the genomic medicine commercial environment to bring this medicine to patients. In parallel, we remain focused on advancing our wholly owned neurology genomic medicine pipeline and progressing our Fabry gene therapy program towards a potential BLA submission in the second half of 2025.”
The collaboration and license agreement with Pfizer will terminate effective April 21, 2025, at which time Pfizer will be required to transition the giroctocogene fitelparvovec program back to Sangamo. All trial participants will continue to be monitored as planned during the transition period.
Sangamo believes that its recently announced partnerships with Genentech and Astellas, and advanced business development discussions for its Fabry gene therapy program, will allow it to chart a path forward for its neurology genomic medicine pipeline as it prepares to initiate expected enrollment of patients in the Phase 1/2 study of ST-503 for idiopathic small fiber neuropathy in mid-2025, and file an anticipated Clinical Trial Authorisation (CTA) submission for the prion disease program in Q4 2025, each subject to securing adequate additional funding.
About the AFFINE Trial
The Phase 3 AFFINE (NCT04370054) trial is an open-label, multicenter, single-arm trial to evaluate the efficacy and safety of a single infusion of giroctocogene fitelparvovec in adult male participants (n=75 dosed participants) with moderately severe to severe hemophilia A. Trial participants included in the assessments of the key endpoints of the primary efficacy analysis (n=50) completed a minimum six months of routine FVIII replacement prophylaxis therapy during the lead-in study (NCT03587116) providing data to compare with post giroctocogene fitelparvovec treatment.
In July 2024, Pfizer announced that the AFFINE trial achieved its primary objective of non-inferiority, as well as superiority, of total annualized bleeding rate (ABR) from Week 12 through at least 15 months of follow up post-infusion compared with routine Factor VIII (FVIII) replacement prophylaxis treatment. Following a single 3e13 vg/kg dose, giroctocogene fitelparvovec demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period (1.24 vs 4.73; one-sided p-value=0.0040). Key secondary endpoints as defined by the trial protocol were met and also demonstrated superiority compared to prophylaxis. In the AFFINE trial, giroctocogene fitelparvovec was generally well tolerated.
Giroctocogene fitelparvovec has been developed as part of a collaboration agreement for the global development and commercialization of gene therapies for hemophilia A between Sangamo Therapeutics and Pfizer. In late 2019, Sangamo transferred the manufacturing technology and the Investigational New Drug application to Pfizer. Under the agreement, Pfizer assumed responsibility for pivotal studies, any regulatory activities, and potential global commercialization of giroctocogene fitelparvovec.
About Hemophilia A
Hemophilia is an inherited, rare bleeding disorder that causes people to bleed for longer than normal due to a deficiency of a protein required for normal blood clotting, known as clotting Factor VIII (FVIII) in hemophilia A. The severity of hemophilia is determined by the amount of the factor in the blood. The lower the amount of the factor, the more likely it is that bleeding will occur, which can lead to serious health problems.
Hemophilia A occurs in approximately 25 in every 100,000 male births worldwide. Approximately 55-75% of males with hemophilia A have a moderate to severe form of the disease. For people who live with hemophilia A, there is an increased risk of spontaneous bleeding as well as bleeding following injuries or surgery. It is a lifelong disease that requires constant monitoring and therapy.
SOURCE: Hemophilia
Post Views: 349
