Skip to main content
Premium Trial:

Request an Annual Quote

Sirna Files IND for RNAi-based AMD Drug


Sirna Therapeutics announced this week that it has filed an investigational new drug application to begin clinically testing an RNAi-based treatment for age-related macular degeneration, making it the second company to have done so.

“We’re very happy with this IND,” Nassim Usman, Sirna’s COO, told RNAi News. “This is actually approximately three months earlier than we had scheduled it for.” Sirna’s filing also comes about a month after Acuity Pharmaceuticals reported that it had filed an IND with the US Food and Drug Administration for its own AMD therapy, Cand5 (see RNAi News, 8/13/2004).

If approved by US regulators, Sirna’s IND will allow the company to begin human trials of Sirna-027, an siRNA targeting vascular endothelial growth factor receptor-1. Sirna said it expects to begin enrollment in its phase I trial before the end of the year, and have top-line data available by the third quarter of 2005.

According to Usman, the dose-escalating phase I study is set to enroll about 25 AMD patients, who would receive a single intravitreal injection of Sirna-027. Patients will be recruited at multiple sites across the country, he said, including the Massachusetts Eye and Ear Infirmary, a Harvard Medical School teaching hospital; the Cleveland Clinic Foundation’s Cole Eye Institute; the Bascom Palmer Eye Institute at the University of Miami School of Medicine; and the Wilmer Eye Institute at Johns Hopkins University.

The primary endpoints of the clinical trial are safety and tolerability of Sirna-027, Usman said. “We will also be looking at surrogate markers of biological activity, such as … the extent of choroidal neovascularization, [blood] vessel proliferation and leakage, macular edema, and retinal thickening,” he said.

If the phase I data are positive, Sirna intends to advance Sirna-027 into phase II, Usman said, but whether it will do so on its own has not yet been established.

“We’ve publicly stated that we are interested in partnering with a major pharmaceutical or ophthalmic company,” he said, adding that a deal could occur “at any time” within the drug development and commercialization timeline.

Usman declined to comment specifically on whether discussions with potential partners are ongoing, but said that “there’s significant interest in the program.”

— DM


The Scan

Fertility Fraud Found

Consumer genetic testing has uncovered cases of fertility fraud that are leading to lawsuits, according to USA Today.

Ties Between Vigorous Exercise, ALS in Genetically At-Risk People

Regular strenuous exercise could contribute to motor neuron disease development among those already at genetic risk, Sky News reports.

Test Warning

The Guardian writes that the US regulators have warned against using a rapid COVID-19 test that is a key part of mass testing in the UK.

Science Papers Examine Feedback Mechanism Affecting Xist, Continuous Health Monitoring for Precision Medicine

In Science this week: analysis of cis confinement of the X-inactive specific transcript, and more.