Sangamo and Pfizer announced their focus on translating genomic medicines using gene therapy for severe hemophilia A has shown promising results. Shares in $SGMO and $PFE reacted positively to the news.
Sangamo and Pfizer announced their focus on translating genomic medicines using gene therapy for severe hemophilia A has shown promising results. Shares in $SGMO and $PFE reacted positively to the news.
Pfizer and Sangamo have an exclusive, global collaboration and license agreement to develop and commercialize gene therapies for hemophilia. Sangamo and Pfizer are also collaborating for the development and commercialization of therapeutics using our zinc finger protein transcription factors (ZFP-TFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene.
“Collaborations have led to recent advances including neuro-stimulators, stent technologies, biomarkers, robotic assistance, and implantable electronic devices. Since innovation fuels the medical device sector’s ongoing quest for better ways to treat and diagnose medical conditions, when coupled with patient life expectancy increasing and aging populations globally, the medical device sector should continue growing at a positive rate in the future.”
Sangamo Therapeutics and Pfizer interim data from Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A.
Data indicate that SB-525 was generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII (FVIII) levels across the four dosage cohorts. Eight patients total were dosed. Based on these results, the Safety Monitoring Committee (SMC) recommended cohort expansion at the 3e13 vg/kg dose.
“The interim data from the first eight patients with hemophilia A treated with SB-525 gene therapy in the Alta study are encouraging and demonstrate a dose-dependent relationship, evidence of sustained factor levels, and low variability, both within each patient and within each cohort,” said Edward Conner, MD, Chief Medical Officer of Sangamo
“These interim results suggest that SB-525 may be well-tolerated and may prove to have the predictability and sustained treatment effect that can bring clinical benefit in patients with hemophilia A. 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.”
The Phase 1/2 interim data include eight patients treated across four ascending dosage cohorts (9e11 vg/kg, 2e12 vg/kg, 1e13 vg/kg and 3e13 vg/kg, with two patients per cohort).
Patients demonstrated a dose-dependent increase in FVIII levels, achieving clinically relevant increases in FVIII activity in the higher dosage cohorts and normal FVIII levels in the 3e13 vg/kg dose cohort (normal range:50-150%). At week 6 post infusion, the two fourth dose cohort patients reached 140% and 94% of normal (as measured by one-stage clotting assay) and 93% and 65% (as measured by chromogenic assay).
A dose-dependent reduction in the use of Factor VIII replacement therapy was also observed, with patients in the highest dose cohort not requiring factor replacement therapy after initial use of prophylactic factor and experiencing no bleeding events to date. SB-525 was generally well-tolerated, with one patient (treated with the 3e13 vg/kg dose) reporting a treatment-related serious adverse event of hypotension and fever, which occurred following vector infusion and resolved with treatment within 24 hours of completion of vector infusion.
Recent active Healthcare companies in the medical device industry include:
- Soliton, Inc. SOLY
- T2 Biosystems, Inc. TTOO
- Sangamo Therapeutics, Inc. SGMO
- Pfizer, Inc. PFE
- Melinta Therapeutics, Inc. MLNT
About Sangamo Therapeutics
Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic medicines with the potential to transform patients’ lives using gene therapy, ex vivo gene-edited cell therapy, in vivo genome editing, and gene regulation. For more information about Sangamo, visit www.sangamo.com.
About Pfizer Inc.
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world’s best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time.
Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.pfizer.com. In addition, to learn more, please visit us on www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
From The TradersCommunity Research Desk