Sirna Science

Sirna Therapeutics has reported the final results from its Phase I trial for Sirna-027, a therapy for age-related macular degeneration. Single ascending doses of Sirna-027 were safe and well-tolerated, and all 26 patients showed visual acuity stabilization eight weeks after a single injection. In addition, at the same time point, five of 26 patients experienced clinically significant improvement in visual acuity, indicated by an increase of at least three lines on an eye chart.

Translation: Five of 26 patients read the first three lines on an eye chart. None read 4. Single ascending doses is over the Cargo Cult Scientists head. I'm not an investor.

Patients received a single intra-vitreal injection of the drug in doses ranging from 100 to 1,600 micrograms. Three months after a single injection, 24 of 26 patients showed visual acuity stabilization, with four of 26 patients experiencing clinically significant improvement in visual acuity. Only two of 26 patients experienced a reduction in visual acuity of three lines or more. A decrease in foveal thickness was observed in some patient groups, which is an indication of biological activity of Sirna-027.

Translation: 26 people received siRNA through a needle injection direction into the eyeball. The range is 0.2 to 1.6 (!!!) mgs but how that range was divided up among the patients is not revealed. RNAi effects, which generally last less than 72 hours, are given the credit for improved visual acuity three months later. Dose dependence of the improvements is not mentioned. There is no control group of 26 who received scrambled RNA. Scrambled RNA is a sequence not known to have homology with any mRNA. There is no control!!!

Sirna is now into a Phase II trial with Sirna-027. Merck has offered 1.1 billion for the company.