Shares of Sangamo BioSciences, Inc. (SGMO - Free Report) jumped more than 17% to $22.96 per share on Mar 6 following positive data on its HIV candidate SB-728-T. The company presented encouraging early stage data from the SB-728-T program at the conference on retroviruses and opportunistic infections (CROI).
SB-728-T makes use of the company’s zinc finger DNA-binding protein nucleases (ZFN) genome- editing technology. SB-728-T is an autologous CD4+ T-cell candidate where the gene for CCR5, a co-receptor for the dreaded virus to enter, is modified using ZFN-mediated genome editing to disrupt the CCR5 protein. The disruption of the CCR5 protein makes the relevant T-cells resistant to infection by the most common HIV strain.
Data from a phase I /II open-label, dose escalation, multi-center study (SB-728-1101) revealed that HIV patients treated with Bristol-Myers Squibb Company's (BMY - Free Report) chemotherapy drug Cytoxan (cyclophosphamide) responded better to the subsequent SB-728-T infusion. The patients did not take antiretroviral drugs. The candidate was found to be safe and well tolerated.
However, the positive results were witnessed for a very small population. The highest dose of Cytoxan, 1.0 g/m2, prior to SB-728-T infusion led to the greatest decrease in viral load. Data revealed that the virus stabilized (but was detectable) in 2 of the 3 patients treated with the dose. Sangamo said that the study was extended to include six more HIV patients to determine the optimal dose of the chemotherapy drug. The company intends to evaluate 12 more HIV patients at the optimal dose.
At the same conference, Sangamo also provided an update on another ongoing study (SB-728-902, cohort 5) on SB-728-T. Sangamo said that data from the phase II study revealed that SB-728-T treatment caused HIV levels to drop significantly in a patient who was without treatment for 31 weeks.
Sangamo also said that it has developed a new method to use messenger RNA (mRNA) delivery of the ZFNs used in CCR5 genome modification of the T-cells. This will be used in the next study on SB-728-T, expected to commence by Jun 30, 2014. We believe investor focus will remain on updates on SB-728-T going forward.
We remind investors that Sangamo was also in the news earlier this year when it inked a deal with Biogen Idec (BIIB - Free Report) on the development of therapeutics for hemoglobinopathies resulting from the abnormal structure or underproduction of hemoglobin.
Sangamo currently carries a Zacks Rank #3 (Hold). Better-ranked stocks include Alexion Pharmaceuticals (ALXN - Free Report) and Biogen. While Alexion carries a Zacks Rank #1 (Strong Buy), Biogen holds a Zacks Rank #2 (Buy).