Axonyx Inc. this week intends to begin enrolling its second Phase III international trial with Phenserine, a dual-action Alzheimer's candidate that is being evaluated as a disease modifier.
The New York-based company expects to take a year to enroll the 450-patient, randomized, placebo-controlled, double-blind trial. Meanwhile, Axonyx is in the midst of running two other key trials.
Of the two, the largest is a 375-patient Phase III European trial designed to evaluate Phenserine at two doses. Data are expected toward the end of December. (See BioWorld Today, June 27, 2003.)
The other trial, a 75-patient Phase IIb, is designed to determine whether Phenserine is a disease modifier as opposed to a symptomatic therapy, Colin Neill, Axonyx's chief financial officer, told BioWorld Today. Specifically, the Phase IIb will evaluate Phenserine's ability to lower the levels of the beta-amyloid precursor protein and beta-amyloid in the plasma and cerebrospinal fluid of mild to moderate Alzheimer's disease (AD) patients. The presence of toxic beta-amyloid in the brains of AD patients is considered to be involved in the causation, as well as the progression, of AD.
Phenserine is a dual-action acetylcholinesterase and beta-amyloid precursor protein inhibitor licensed from the National Institute of Aging of the National Institutes of Health in Bethesda, Md.
The Phase III study will evaluate the safety and efficacy of two different doses of Phenserine in mild to moderate AD patients following a six-month treatment period. The company is taking advantage of the regulatory approvals already granted in Europe for its ongoing clinical development program, and the second Phase III trial will be conducted at multiple clinical sites throughout Europe and Israel. The company intends to include clinical trial sites in the U.S. on FDA approval.
The second Phase III trial will recruit patients to randomly receive Phenserine 10 mg twice daily, Phenserine 15 mg twice daily or placebo. Patients in both Phase III trials will undergo testing with the standard memory and cognition exams, which are the efficacy endpoints required by the FDA and European regulatory authorities for potential marketing approval, the company said.
Since September, Axonyx has raised $100 million, Neill said. (See BioWorld Today, Sept. 15, 2003; Jan. 9, 2004; and May 5, 2004.)
Axonyx has just less than $100 million in cash, and its burn rate in the past has been about $6 million to $7 million a year. However, Neill said he expects to burn about $25 million this year.
The company has five employees and 52 million outstanding shares.
Marketing clearance of Phenserine would prompt the company to contract with a partner.
Axonyx's stock (NASDAQ:AXYX) Wednesday was up 33 cents to close at $5.50.