Genomic medicine company Sangamo Therapeutics Inc SGMO had a trio of news releases Tuesday: data readout from a clinical trial, an update on broader clinical development and an agreement to secure access to a large-scale adeno-associated virus manufacturing from a contract development and manufacturing organization.
Incidentally, Sangamo shares came under pressure Feb. 7 following the release of interim results from two genome editing studies presented at the WORLDSymposium 2019 in Orlando, Florida.
Midstage Hemophilia A Asset Found Effective
Interim data from the Phase 1/2 Alta study evaluating Sangamo's investigational SB-525 gene therapy for severe hemophilia A showed that it was well-tolerated and demonstrated a dose-dependent increase in Factor VIII levels across the four dosage cohorts.
The company is partnering with Pfizer Inc. PFE on the therapy.
Based on the results from the eight dosed patients, the Safety Monitoring Committee, or SMC, recommended cohort expansion at the 3e13 vg/kg dose, Sangamo said.
"We need to continue observing how the data mature and how additional patients in the expansion cohort respond to SB-525. We look forward to working with Pfizer to potentially advance SB-525 into a registrational study," Edward Conner, Sangamo's chief medical officer, said in a statement.
Sangamo said longer-term follow-up data is likely to be presented at an upcoming scientific meeting. Based on the SMC recommendation, the company said the fourth cohort will be expanded by up to five patients.
Rip-Roaring Pipeline
Separately, Sangamo gave an update on ST-400, its gene-edited cell therapy for beta thalassemia, which is an autologous cell therapy that involves gene editing of a patient's own hematopoietic stem cells using non-viral delivery of zinc finger nuclease, or ZFN, technology. Sangamo is partnering with Sanofi SA SNY on ST-400.
Sangamo noted that following the ST-400 infusion, the patient demonstrated neutrophil and platelet recovery within two and four weeks of infusion, respectively. Changes have been detected in circulating white blood cells, indicating successful editing of the BCL11A gene, the company said.
"While these data are very early and will require confirmation in additional patients as well as longer follow-up to draw any clinical conclusion, they are promising. The detection of indels in peripheral blood with increasing fetal hemoglobin at seven weeks is suggestive of successful gene editing in this transfusion-dependent beta thalassemia patient," Angela Smith, a principal investigator of the study at the University of Minnesota, said in a statement.
The company also said the Phase 1/2 clinical trials evaluating its SB-913 in the CHAMPIONS study for treating MPS II, SB-318 for MP 1 and SB-FIX for hemophilia are ongoing. Further updates on these studies are due later this year, it said.
Sangamo also plans to initiate a new clinical trial to evaluate its second-generation ZFNs to treat MPS II in the second half of 2019. Using data from this study, the company expects to make a Phase 3 decision for SB-913 program in 2020, and also define the next steps for the SB-318 and SB-FIX programs.
Expanding Manufacturing Footprint
Even as Sangamo progresses with its clinical programs, the company announced an option agreement with Brammer Bio, a CDMO, for obtaining access to large-scale AAV manufacturing.
The company also said its new facilities in Brisbane, California — where the construction of a Phase 1/2 cGMP manufacturing facility is underway — will become operational in 2020.
Sangamo shares were up 27.23 percent at $12.12 at the time of publication Tuesday.
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