Celtic Pharma Announces Successful Completion of Phase I/II study of Xerecept® in Pediatric Patients with Cerebral Tumors

 May 22, 2012 08:00 AM Eastern Daylight Time  NEW YORK, LONDON & HAMILTON, Bermuda--(BUSINESS WIRE)--Celtic Pharmaceutical Holdings L.P. (“Celtic Pharma”), a global private equity firm focused on late stage pharmaceutical assets, announces that it has completed a successful Phase I/II study of the investigational new drug Xerecept® (corticorelin acetate) in pediatric patients who are dependent on the steroid Decadron (dexamethasone) to treat peritumoral brain edema associated with cerebral tumors. Initial indications are that Xerecept® can reduce or eliminate the need for Decadron use and provide significant quality of life improvements. “It is exciting to observe the ability of Xerecept® to be associated with dexamethasone dosing reduction in pediatric patients. In this study, all of the key stakeholders including patients, parents and investigators were impressed with the study outcome and supported further development of Xerecept® for a pediatric indication.” . The full data from this pediatric study of Xerecept® will be presented at the forthcoming International Symposium for Pediatric Neuro-Oncology in Toronto June 24-27, 2012.The study, involving 15 children, was conducted by Stewart Goldman M.D. at Children's Memorial Hospital in Chicago, IL and Mark Kieran M.D. at Dana-Farber Cancer Institute in Boston, MA. All of the children enrolled on the study had severe steroid-induced side effects and had been unable to decrease dexamethasone dosing in spite of two attempts by the attending physicians to reduce the daily steroid dose levels.Consistent with responses seen in the adult clinical trials of Xerecept®, nearly all of the pediatric patients were able to substantially reduce their dose of dexamethasone from baseline. Many of these patients were able to eliminate steroid use altogether for significant periods. The daily maximum tolerated dose of Xerecept® in this study was 60μg per kilogram given in divided doses subcutaneously morning and evening.The benefit of Xerecept® was also seen with clinically important improvements in patient functioning being recorded utilizing the PedsQL, a quality of life instrument which has been validated for use in pediatric patients with cerebral tumors. No significant drug-related side effects were observed in any of these patients.Dr. O'Connor, head of clinical development for Celtic Pharma, stated: “It is exciting to observe the ability of Xerecept® to be associated with dexamethasone dosing reduction in pediatric patients. In this study, all of the key stakeholders including patients, parents and investigators were impressed with the study outcome and supported further development of Xerecept® for a pediatric indication.”“For those of us who treat children with brain tumors in our daily practice, the prospect of a safe and effective alternative to corticosteroids to control edema in these patients is indeed exciting”, said Dr Stewart Goldman, Children’s Memorial in Chicago.Dr. Mark Kieran, Dana-Faber/Children’s Hospital Cancer Center in Boston, also commented, “The destructive effects of chronic high-dose steroids on these children are painful to watch, so Xerecept® holds the promise of substantive medical and quality-of-life benefits for them.”Subsequent to completion of the study, Celtic Pharma has held an end of Phase II meeting with the FDA’s Oncology Division, to discuss the pathway for registration of Xerecept® in this pediatric indication and the appropriate primary end-point for a follow on pivotal study. The FDA has provided good guidance on these points, and it was agreed that Celtic Pharma would submit a detailed protocol for a pivotal study in pediatric patients for review by the Agency in the near future.

About Xerecept®  Xerecept® is a synthetic version of the natural peptide hormone ‘corticotrophin releasing factor’. Xerecept® is in Phase III development for the treatment of cerebral edema associated with primary and metastatic brain tumors in adults, and has been demonstrated in prior trials to be an effective alternative to the use of chronic, high-dose corticosteroids in this large patient population, while offering a benign side-effect profile even over several years of chronic administration. Recent preclinical studies from leading cancer centers have indicated that Xerecept® may also have direct anti-tumor effects, and may be synergistic with Avastin in certain solid tumor indications.

About Celtic Pharmaceutical Holdings L.P.Celtic Pharmaceutical Holdings L.P. (Celtic Pharma) is a global private equity investment firm focused on the biotechnology and pharmaceutical industries. Celtic Pharma was founded by Stephen Evans-Freke and John Mayo, CBE and is based in Bermuda, with offices in New York and London. Celtic Pharma has acquired and invested in late stage pharmaceutical programs and manages these programs through their development for ultimate sale to established pharmaceutical companies. Celtic Pharma is fully invested at this time. Celtic Pharma’s aim has been to bridge the gap between the established pharmaceutical companies’ new product pipeline crisis and the biotech industry’s capital drought. For further information, please visit Celtic Pharma’s website at www.celticpharma.com.