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Acuity Says Primate Data on RNAi Drug Positive, Schedules Meeting with FDA

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Acuity Pharmaceuticals said this week that data from a recently completed primate study of Cand5, its RNAi treatment for age-related macular degeneration, indicate that the drug is both safe and effective.

The company has also scheduled a meeting with US regulators to discuss the details of filing an investigational new drug application for Cand5, RNAi News has learned.

The primate study was designed to evaluate the effect of Cand5 on the proliferation of corneal neovascularization and on blood vessel leakage, Dale Pfost, president and CEO of Acuity, told RNAi News.

“The primate data were done in a clinical trial-like setting — they were double-masked and [placebo] controlled,” Pfost said. “The results showed essentially complete elimination of proliferation and leakage, and they did so in a dose-dependent fashion.”

Pfost added that the company also included a variety of safety measures in the preclinical trial. “All of them were baseline,” he said. “There was nothing distinguishable between the treated and untreated animals. There was no deviation from the normal.”

Pfost declined to provide any additional details about the primate study, noting that the data have been submitted to a peer-reviewed journal. Although not yet formally accepted by the periodical, the results, Pfost said, are expected to be published in the “early part of next year.”

With the positive data in hand, Pfost said that Acuity’s next step is to sit down with the US Food and Drug Administration at a meeting slated for December and discuss the details of filing an IND on Cand5. He added that an actual IND filing is expected in the second half of 2004 — probably in the latter half of the third quarter or early in the fourth quarter — with the initiation of a phase I trial remaining on schedule for before year-end.

In preparation for that clinical trial, Acuity has selected clinical trial centers, and is in the process of selecting a manufacturer of Cand5 for the phase I trial.

“We are well up on all the formal preparations, all the IND-enabling activities,” Pfost said, declining to comment further.

The pre-IND meeting, along with the anticipated granting of a patent on the company’s core RNAi intellectual property (patent application number 20020173478, Post-Transcriptional Gene Silencing by RNAi in Mammalian Cells) early next year and the publication of the Cand5 primate data, comprise a three-tiered plan to boost the company’s profile and attract investment, Pfost said.

But while Pfost had told RNAi News in mid-September that Acuity was in the process of raising between $7 million and $9 million in a Series A round of financing, the proceeds of which would be used to complete phase I testing of Cand5, that plan has been amended.

Instead, Acuity is in the process of raising approximately $2.5 million in seed financing from new and existing investors, which will be used to carry the company through the achievement of the aforementioned milestones, he said.

“All the current shareholders are very interested in capturing an investment opportunity,” he said. “The founders are investing in the com- pany at this point and are very excited about the [primate data].”

Currently, the University of Pennsylvania, which licensed to Acuity its core technology, holds a roughly 25 percent stake in the company. The company’s founders, UPenn researchers Michael Tolentino and Samuel Reich, along with Pfost and certain friends and family members, hold the remainder.

Later on, after the company has met with the FDA, published the primate data, and received its patent, a Series B financing will commence, Pfost noted.

“Those three events … will catalyze quite a significant financing in, probably, the end of Q1 or early Q2 next year,” he said. “That financing … will be sufficient to take us through both phase I and phase II clinical trials, so it may be … in excess of $10 million.”

Cand5 is an siRNA drug that suppresses vascular endothelial growth factor (VEGF), a common target for AMD treatments, and is administered via intravitreal injection. The company has not disclosed the structure of the drug, but Acuity’s director of business development, Todd Wallach, said that the siRNA is “not significantly modified.”

—DM

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