Sangamo BioSciences (NASDAQ: SGMO) recently reported third quarter financial results and updates on clinical programs, primarily providing guidance on the future of the SB-728-T program towards a functional cure for HIV/AIDS. The data presented at ICAAC and CROI earlier this year on SB-728-T are helping to form a clinical path for the experimental therapy, and the company plans to present data in 2012 from the ongoing trial in treatment-naïve patients as well as initiating two new trials in patients already on HAART. In other development news, Sangamo unfortunately discontinued further investigation of SB-509 in diabetic neuropathy due to failure to meet key endpoints in Phase 2b study. Furthermore, the company continues to explore ZFP technology in monogeneic diseases and plans to deliver “data-driven guidance” in the coming quarters as preclinical results become available.
Management provided the most guidance for SB-728-T as this is now Sangamo’s most advanced program. Phase 1 data from ICAAC demonstrated that CCR5-modified T-cells are safe and engraft, traffic, and persist as desired. During HAART treatment interruption after SB-728-T treatment, some patients saw a decrease in viral load, and one patient who was already heterozygous for CCR5 mutation experienced a return to undetectable levels of HIV. These data demonstrated that the decrease in viral load during treatment interruption is statistically significantly correlated with the number of biallelically modified cells infused, informing the design of subsequent trials. Sangamo has begun patient screening for a trial in CCR5 mutation heterozygotes who will undergo a 16-week treatment interruption. A second trial in wild-type CCR5 patients will examine a method for increasing growth and engraftment of biallelically modified T-cells using a technique called leukopenic preconditioning. In this protocol, patients are treated with an immunosuppressive agent that removes patient T-cells to “make room for new ones” and provide conditions in which SB-728-modified CD4+ T-cells should be able to multiply to a much greater degree than in previous studies. This will be a dose escalation study of an agent that has been used previously in autoimmune disorders and adoptive T-cell work.
On the financial front, Sangamo reported a net loss for the quarter of $9.6MM compared to $8.7MM for 3Q2010. The company had cash, equivalents, and marketable securities of $85MM on hand at quarter’s end, and Sangamo expects to have this same amount by year’s end as revenues for the 4th quarter should offset expenses. Total operating expenses for 2011 are still expected to be relatively flat compared to 2010 in the range of $43MM to $47MM.
Disclosure: Long SGMO stock, Short SGMO Calls and Puts.
