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Strong Safety Profile, Maintenance of Lung Function Reported in Lomucin Trial
The results are being presented by Professor Gerry McElvaney, M.D., department chair and chief of medicine at the Royal College of Surgeons, Ireland, based at Beaumont Hospital, Dublin. The Phase II clinical trial, supported by a grant from CFFT, the nonprofit drug discovery and development affiliate of the CF Foundation, was a randomized, double-blind study conducted in Ireland in 55 individuals with CF. Patients were treated with the study dose, 740 milligrams of LOMUCIN (talniflumate) three times daily for four weeks, or matching placebo. In the trial, 36 individuals were randomized to LOMUCIN and 19 to placebo. When compared with placebo, individuals receiving LOMUCIN demonstrated the following clinical and laboratory effects:
* Change in FEV1 (forced expiratory volume in one second) from baseline at four weeks measured in liters (primary endpoint): LOMUCIN improvement of 0.04 liters vs. placebo worsening of -0.08 liters;
* Change in FEV1 from baseline at four weeks measured as relative change in percent predicted FEV1: LOMUCIN improvement of 1.07 percent, placebo worsening of -3.12 percent;
* Change in FVC (forced vital capacity) from baseline at four weeks measure as relative change in percent predicted FVC: LOMUCIN improvement of 0.47 percent, placebo worsening of -3.67 percent;
* There was no improvement in residual volume measures of pulmonary function;
* There was no improvement in pulmonary symptoms as measured by the Acute Respiratory Illness Checklist;
* Four patients were withdrawn from therapy: two on LOMUCIN (diarrhea; abdominal pain), and two on placebo (bronchospasm; lost to follow up); and;
* No serious adverse events related to study medications; there was one unrelated serious adverse event (respiratory tract infection).
"CFFT will work closely with Genaera to evaluate the Phase II trial results and to plan further clinical evaluation of LOMUCIN in CF. The potential therapeutic benefit of a mucoregulator approach in CF could be very significant for our patients and therefore deserves an intense and dedicated follow-up effort to determine next steps," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation and CFFT. "As always, our focus remains on pursuing the most promising therapies to improve and extend the lives of those with CF."
"LOMUCIN was well tolerated by people with CF, and we believe that larger and longer term trials might be considered for demonstrating potentially beneficial effects on pulmonary function. We plan to complete our detailed evaluation of these results and to work with CFFT to determine the most appropriate path forward for LOMUCIN in CF," said Roy C. Levitt, M.D., Genaera's president and CEO. "We believe we have a strong intellectual property position developing for the target hCLCA1. We are also eager to explore all strategies for modulating hCLCA1 function, including antibody approaches, based on program specific funding for these opportunities."
Background on LOMUCIN(TM) and the Mucoregulator Program
Clinical development of LOMUCIN(TM) for CF is supported by an initial award of up to $1.7 million from CFFT. Extensive consultation and intellectual support was provided by CFFT and the CFFT-supported Therapeutics Development Network.
The mucoregulator program is Genaera's second product development program based on its genomics discoveries. Based on the role of the hCLCA1 chloride channel in respiratory diseases, such as CF, the Company has developed LOMUCIN. LOMUCIN is intended to block the hCLCA1-dependent mucus overproduction present in respiratory and sinus disorders, and thereby provide a new strategy for maintaining airway diameter and easing breathing in patients with these diseases.
LOMUCIN is a known compound, talniflumate, which was discovered, developed and marketed as an anti-inflammatory drug by Laboratorios Bago of Buenos Aires, Argentina, the leading independent pharmaceutical company in South America. Talniflumate has been approved and marketed for almost 20 years in Argentina, and selected other countries excluding the United States, Europe, and Japan. The effects of talniflumate in blocking hCLCA1 and mucus overproduction were discovered by Genaera scientists who have submitted patent applications protecting the novel uses of talniflumate as a mucoregulator. Genaera has an exclusive agreement with Laboratorios Bago to develop and commercialize LOMUCIN as a new chemical entity and mucoregulator drug in all major pharmaceutical markets including the United States, Europe, and Japan.
There is an extensive unmet medical need for a therapy that can prevent abnormal mucus production. Chronic sinusitis is one of the most common reasons for physician visits in the United States, with about 35 million cases per year. It is thought that many of the symptoms of chronic sinusitis result from excess mucus production. According to the National Institute of Allergies and Infectious Disease (NIAID) and the American Lung Association, there are more than 65 million patients suffering from diseases where mucus overproduction may be hCLCA1 mediated, including 15 million patients with chronic bronchitis and other forms of chronic obstructive pulmonary disease (COPD), 17 million asthmatics, and 35 million respiratory allergy sufferers. Mucus overproduction and small airway plugging is one of the hallmarks of asthma and excess mucus production is also associated with COPD, chronic bronchitis, and CF.
Source: Genaera Corporation, Cystic Fibrosis Foundation Therapeutics