NEW YORK, July 24 /PRNewswire-FirstCall/ -- MacroChem Corporation today announced results of a Phase 2 clinical trial involving 37 patients who completed a 48-week, U.S. multi-center, open-label efficacy and safety study of EcoNail, a topical antifungal lacquer for the treatment of onychomycosis (nail fungus). The composite primary efficacy endpoint of the trial was “complete cure” defined as negative mycology (negative fungal culture and negative KOH) and clear nail (as determined by expert panel review of clinical photographs). EcoNail is the company’s patented, topically-applied lacquer which contains the antifungal econazole and MacroChem’s enhancer SEPA(R).
A protocol-mandated, external expert panel assessed clinical photographs of 37 onychomycosis patients who completed 48 weeks of EcoNail treatment. The panel observed that 24 patients (65%) showed evidence of clinical improvement, defined in the protocol as an increase in uninvolved (clear) nail area. While none of the 37 patients reached all criteria of the composite primary endpoint, the consensus judgment of the panel was that 15 of 37 patients (41%) demonstrated significant (greater than or equal to 25%) clinical improvement. All patients had fungal culture-proven nail infections at entry, but after 48 weeks of once-daily treatment with EcoNail, 100% of patients had cultures that were negative for dermatophyte growth. Eight of the 37 patients (22%) achieved the secondary endpoint of negative mycology (negative fugal culture plus negative KOH evaluation) at 48 weeks. The panel observed no signs of local irritation related to the once-daily EcoNail treatment. During the trial, no patient required interruption of dosing due to local intolerability. Through 56 weeks of observation, no cutaneous adverse events were attributed by the investigators to EcoNail.
The laboratory of Boni Elewski, MD, Professor of Dermatology at the University of Alabama-Birmingham, performed mycological cultures for this trial. According to Dr. Elewski, “The proportion of patients with negative mycology, that is both negative culture and negative KOH, together with the fact that all the patients had negative cultures at 48 weeks, indicates that EcoNail has clinically relevant antifungal activity. Consistent with results from the study’s interim analysis, the final results showed that a meaningful proportion of patients demonstrated progressive clinical improvement and reduction of fungal disease burden. Based on the practical definition of ‘cure’ published in Scher et al in 2007 in the Journal of the American Academy of Dermatology, it is my opinion upon review of the clinical photographs that 4 of the 37 (11%) patients achieved a cure. Minimal onycholysis and no subungual debris in the clinical setting of negative mycology is for practical purposes cured.” Dr. Elewski added, “If we exclude 6 patients whose baseline nail involvement in my opinion was too severe to warrant entry into this trial, then 4 of 31 (13%) would be considered cured.” The clinical and mycologic findings to date suggest reason for optimism and further study to fully characterize the benefits of this new topical treatment in a very common and difficult to cure condition.”
Michael Goldfarb, MD, Attending Physician, Department of Dermatology at the University of Michigan, who assisted MacroChem in the development of the protocol, noted, “As a practicing dermatologist who has limited treatment options to offer patients with onychomycosis, whether oral or topical, I was particularly impressed with the lack of skin irritation and the progressive clinical improvement.” Dr. Goldfarb added, “There are at least 4 patients in this trial who, if under my care, I would have considered to have shown resolution of their disease. These factors suggest that this new topical agent could be an acceptable and valuable treatment for this typically chronic condition.”
Robert J. DeLuccia, Chairman of MacroChem, stated, “We initiated this non-registration trial in late 2006 primarily to determine the anti-fungal efficacy and safety of EcoNail for the treatment of onychomycosis. In designing our trial protocol, we adopted the clinical endpoints for defining clinical trial success in this patient population which were traditionally accepted at that time. Presently, experts in onychomycosis have developed a new consensus for clinical trial endpoints which, among other things, more clearly define resolved fungal nail infection and normal-appearing nail in clinical trials evaluating therapeutic intervention. There appears to be a greater appreciation today that onychomycotic nails may have a persistent abnormal appearance even in patients where antifungal treatment has been completely effective. Furthermore, long-standing concepts of nail growth have evolved to recognize that some nails may take up to 78 weeks to fully grow.”
He added, “In view of this new consensus among experts in the medical community to better define resolution of onychomycosis, the negative mycology achieved together with the progressively greater clinical improvement and the number of patients achieving a practical cure together with the complete lack of local skin irritation seen in our trial, we will now continue our discussions with potential licensees who have expressed interest in further development of this promising new topical treatment for onychomycosis.”
About the EcoNail Phase 2 trial and Onychomycosis
Patients participating in the EcoNail study, which is was conducted under MacroChem’s U.S. Investigational New Drug application filed with the FDA, received 48 weeks of treatment at three U.S. sites and included efficacy assessments using standard criteria of nail appearance and mycology. This trial was specifically designed, with the assistance of well-known onychomycosis experts, to address three important objectives: to assess early signs of efficacy, to maintain robust clinical endpoints in the full study, and, if successful, to facilitate advancement to Phase 3 as soon as possible.
Onychomycosis affects 20-30 million people in the United States alone, including nearly half of those over age 70. Untreated, the disease causes nails to thicken, resulting in localized pressure-related pain. Topical treatments currently marketed in the U.S. reportedly succeed in fewer than 12 percent of patients, and existing systemic treatments, which are effective in less than half of all cases, have known toxicity. Despite the shortcomings of these existing drugs, sales of treatments for onychomycosis top approximately $1.5 billion annually worldwide.
About MacroChem
MacroChem is a specialty pharmaceutical company that develops and seeks to commercialize pharmaceutical products. Product candidates in our clinical development portfolio are: pexiganan, EcoNail(R) and SR9025 which was recently acquired. Pexiganan, a novel topical anti-infective for the treatment of diabetic foot infection, was recently in-licensed and has already completed two Phase 3 trials. EcoNail is a topically applied SEPA-based econazole lacquer for the treatment of onychomycosis, a condition commonly known as nail fungus. SR9025 is a new generation nucleoside analog which has demonstrated both pre-clinical and clinical activity in certain cancers.
On April 23, 2008, MacroChem announced it acquired Virium Pharmaceuticals Inc. Virium was a non-public, development stage company whose business is developing and commercializing novel therapeutics with a focus in oncology. By acquiring Virium, the Company has in-licensed a group of product candidates targeted for development and commercialization in several oncology or oncology-related indications. These opportunities involve compounds that Virium believes show promising late-stage, pre-clinical or early clinical data.
On July 10, 2008, Access Pharmaceuticals, Inc. announced it had signed an agreement and plan of merger with MacroChem pursuant to which MacroChem is expected to be merged with and into a wholly-owned subsidiary of Access. The transaction is expected to close in H2 2008.
Forward Looking Statements
With the exception of historical information contained in this press release, the matters described herein are forward-looking statements that involve risks and uncertainties. MacroChem’s actual results may differ significantly from the results discussed in the forward-looking statements. Factors that might cause such a difference include, but are not limited to, those discussed or referred to in the section entitled “Risk Factors” in MacroChem’s Annual Report on Form 10-K, as well as those discussed elsewhere therein, and include, without limitation, risks regarding product development, the timing and results of clinical trials, the regulatory approval process, capital requirements, financial condition, patent protection and dependence on third parties for development and licensing arrangements. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. MacroChem undertakes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. For more information visit our website, www.macrochem.com.
CONTACT: David P. Luci, President & Chief Business Officer of MacroChem
Corporation, +1-212-514-8094
Web site: http://www.macrochem.com/
