Urology 'Gold Journal' Publishes Successful Phase 2 Study of Innovative Topical Drug For Erectile Dysfunction

02-21-2001: MacroChem Corporation (Nasdaq: MCHM) announced today that the February issue of UROLOGY, a respected peer-reviewed specialty journal, carries a paper detailing a successful Phase 2 study demonstrating the safety and efficacy of Topiglan(R), the company's topical drug for erectile dysfunction (ED). The company has filed the results of this study with the U.S. Food & Drug Administration, and is now in a phase 3 pivotal trial to support an application for marketing approval.

Instead of a tablet or capsule like all other erectile-dysfunction drugs now in phase 3, Topiglan is a gel. Men will apply Topiglan to the glans ("head") of their penises at least 15 minutes before attempting intercourse. Topiglan's active ingredient, alprostadil, affects only the penis. Alprostadil should pose no risk to men prescribed nitrates or other drugs for a heart condition. MacroChem hopes Topiglan will become first-line therapy for all ED patients, including those who can't take Viagra(R) or Viagra(R) class ("PDE-5 inhibitor") drugs. Lead author is world-renowned Boston ED expert.

Irwin Goldstein, MD, a recognized authority on male and female sexual dysfunction, performed the randomized, placebo-controlled study at Boston University School of Medicine, and co-authored the UROLOGY paper. The patient base for the study was a group of 60 men with well-documented moderate-to- severe erectile dysfunction - typical of the difficult patients referred to the University's clinic. Many of the patients in the Topiglan study had failed to respond to Viagra(R) and other treatments for impotence.

"To simplify recruitment, we accepted all patients referred to us into this trial, including many who could obtain erections only with direct injection of vasoactive drugs into their penises, and even some who failed to respond to that therapy," Dr. Goldstein said. "You don't include such individuals when you're trying to optimize a trial to show efficacy."

"We were looking for more than evidence of potential efficacy - we were looking for potential local and systemic side effects, too" Dr. Goldstein explained. "We got valuable information documenting absence of serious local and systemic effects from men who didn't respond to Topiglan, as well as from men who did."

"In this tough group of patients, 38.9% of those who received Topiglan obtained an erection deemed capable of initiating and completing vaginal intercourse, versus 6.8% receiving placebo gel," Dr. Goldstein reported.

"The statistical difference between response to Topiglan and placebo was highly significant: p<0.005 - meaning that the likelihood of pure chance producing such results is just 5 in 1000. The low (6.8%) placebo response may demonstrate the severity of the erectile dysfunction."

"These results demonstrate that MacroChem had good reason to proceed to a Phase 3 home trial, because Topiglan clearly showed potential for both good efficacy and safety in our very difficult tertiary-referral patient base," Dr. Goldstein said.

Topiglan is a topical gel combining prostaglandin E1 (alprostadil) and MacroChem's patented through-the-skin absorption-enhancing excipient known as SEPA(R). Prior to Topiglan, alprostadil could be used to treat erectile dysfunction only if delivered in a highly invasive manner - either by injection into the penis with a hypodermic needle (Caverject(R)), or insertion into the urethra as a small suppository, with a syringe-like device (Muse(R)). SEPA is added to encourage sufficient absorption of alprostadil through intact skin to induce erection.