Sangamo Therapeutics, Inc. (SGMO - Free Report) and its partner Pfizer (PFE - Free Report) announced encouraging interim data from the phase I/II Alta study evaluating their gene therapy candidate, SB-525, in patients with severe hemophilia A. Data showed that the gene therapy candidate holds potential.
Shares of Sangamo rallied almost 28% on Apr 2 following the data readout. However, shares of the company have increased 7% so far this year compared with the industry’s rise of 11.7%.
The phase I/II study evaluated four doses of SB-525 – 9e11 vg/kg, 2e12 vg/kg, 1e13 vg/kg and 3e13 vg/kg – with two patients per cohort. Data from the study showed that patients treated with the highest dosage (3e13 vg/kg) achieved normal factor VIII levels. Dose dependent increase in factor VIII levels were observed in the eight patients treated with SB-525 gene therapy across the four dosage cohorts. Factor VIII boosts blood clotting, which is a major symptom of haemophilia. Moreover, the candidate was well tolerated in every dosage cohort.
The company believes that SB-525 may achieve predictability and sustained treatment effect, which can lead to clinical benefit in haemophilia A patients.
Based on the data from the study, the Safety Monitoring Committee ("SMC") recommended expansion of the 3e13 vg/kg cohort. Total patients in the cohort will increase to five. Longer-term follow up data will be presented at a future scientific meeting.
Although interim data from the study seems promising, the clinical development of SB-525 is in early stage with commercialization prospects years away.
We note that there are other treatments available for treating hemophilia A and several others are in development including gene therapies. In February 2019, Novo Nordisk’s (NVO - Free Report) Esperoct was approved for the indication. Companies developing gene therapy candidates for treating hemophilia A include Spark Therapeutics and uniQure. The most advanced gene therapy for hemophilia A treatment in clinical studies is BioMarin’s valoctocogene roxaparvovec (BMN-270). Late-stage studies are evaluating 6e13 vg/kg and 4e13 vg/kg doses of the candidate.
Apart from SB-525, Sangamo is also developing gene therapies in collaboration with Pfizer for treating amyotrophic lateral sclerosis and frontotemporal lobar degeneration.
Zacks Rank
Sangamo currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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Sangamo Therapeutics, Inc. (SGMO - Free Report) and its partner Pfizer (PFE - Free Report) announced encouraging interim data from the phase I/II Alta study evaluating their gene therapy candidate, SB-525, in patients with severe hemophilia A. Data showed that the gene therapy candidate holds potential.
Shares of Sangamo rallied almost 28% on Apr 2 following the data readout. However, shares of the company have increased 7% so far this year compared with the industry’s rise of 11.7%.
The phase I/II study evaluated four doses of SB-525 – 9e11 vg/kg, 2e12 vg/kg, 1e13 vg/kg and 3e13 vg/kg – with two patients per cohort. Data from the study showed that patients treated with the highest dosage (3e13 vg/kg) achieved normal factor VIII levels. Dose dependent increase in factor VIII levels were observed in the eight patients treated with SB-525 gene therapy across the four dosage cohorts. Factor VIII boosts blood clotting, which is a major symptom of haemophilia. Moreover, the candidate was well tolerated in every dosage cohort.
The company believes that SB-525 may achieve predictability and sustained treatment effect, which can lead to clinical benefit in haemophilia A patients.
Based on the data from the study, the Safety Monitoring Committee ("SMC") recommended expansion of the 3e13 vg/kg cohort. Total patients in the cohort will increase to five. Longer-term follow up data will be presented at a future scientific meeting.
Although interim data from the study seems promising, the clinical development of SB-525 is in early stage with commercialization prospects years away.
We note that there are other treatments available for treating hemophilia A and several others are in development including gene therapies. In February 2019, Novo Nordisk’s (NVO - Free Report) Esperoct was approved for the indication. Companies developing gene therapy candidates for treating hemophilia A include Spark Therapeutics and uniQure. The most advanced gene therapy for hemophilia A treatment in clinical studies is BioMarin’s valoctocogene roxaparvovec (BMN-270). Late-stage studies are evaluating 6e13 vg/kg and 4e13 vg/kg doses of the candidate.
Apart from SB-525, Sangamo is also developing gene therapies in collaboration with Pfizer for treating amyotrophic lateral sclerosis and frontotemporal lobar degeneration.
Zacks Rank
Sangamo currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Is Your Investment Advisor Fumbling Your Financial Future?
See how you can more effectively safeguard your retirement with a new Special Report, “4 Warning Signs Your Investment Advisor Might Be Sabotaging Your Financial Future.”
Click to get it free >>
Zacks Names "Single Best Pick to Double"
From thousands of stocks, 5 Zacks experts each have chosen their favorite to skyrocket +100% or more in months to come. From those 5, Director of Research Sheraz Mian hand-picks one to have the most explosive upside of all.
It’s a little-known chemical company that’s up 65% over last year, yet still dirt cheap. With unrelenting demand, soaring 2022 earnings estimates, and $1.5 billion for repurchasing shares, retail investors could jump in at any time.
This company could rival or surpass other recent Zacks’ Stocks Set to Double like Boston Beer Company which shot up +143.0% in little more than 9 months and NVIDIA which boomed +175.9% in one year.
Free: See Our Top Stock and 4 Runners Up >>
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