Promedior Receives Orphan Designation in Europe for Recombinant Human Pentraxin-2 for the Treatment of Idiopathic Pulmonary Fibrosis
MALVERN, PA, August 28, 2012 —Promedior
, Inc., a clinical stage
biotechnology company developing novel biologic therapeutics for the treatment of fibrosis, today announced that the European Commission granted Orphan Drug Designation to Promedior’s lead drug candidate, recombinant human Pentraxin-2 (rhPTX-2), also called PRM-151, for the treatment of idiopathic pulmonary fibrosis (IPF). IPF is a progressive, debilitating fatal disease that affects approximately 76,000 people in Europe.
Developed by Promedior for multiple systemic fibrotic diseases, PRM-151 is currently being tested in a Phase 1b clinical study in IPF patients. PRM-151 is a recombinant form of human Pentraxin-2, an endogenous human protein that is specifically active at the site of tissue damage and works as an agonist, preventing and potentially reversing fibrosis. In a Phase 1a clinical study, PRM-151 was shown to be generally safe and well tolerated in healthy volunteers and IPF patients. In the Public Summary of Opinion granting Orphan Drug Designation1
, the European Medicines Agency (EMA) noted that “the sponsor has provided sufficient information to show that recombinant human pentraxin-2 might be of significant benefit for patients with idiopathic pulmonary fibrosis because early studies in experimental models show that it works in a different way to existing treatments and might improve the outcome of patients with this condition.” PRM-151’s benefits to IPF patients will need to be confirmed at the time of marketing authorization.
“Orphan Designation is a significant step forward in the development of PRM-151, and we see tremendous opportunity to better meet patient needs in IPF,” said Suzanne L. Bruhn, Ph.D., President and Chief Executive Officer of Promedior. “Promedior’s initial strategy is focused on applying our Pentraxin-2 therapeutic platform to rare diseases involving fibrosis, while we also explore future opportunities for a wide range of fibrotic diseases.”
Orphan designation of PRM-151 for IPF in Europe qualifies Promedior for certain development and commercial incentives, including protocol assistance, access to centralized authorization procedures, reduced fees for regulatory activities, and ten years of market exclusivity after authorization. In March 2012, Promedior announced that the United States Food and Drug Administration (FDA) Office of Orphan Products Development granted an Orphan Drug designation to PRM-151 for the treatment of idiopathic pulmonary fibrosis (IPF).
IPF is a serious, life-limiting lung disease characterized by fibrosis and scarring of lung tissue. Replacement of normal lung tissue by fibrosis results in restriction in the ability to fill the lungs with air and decreased transfer of oxygen from inhaled air into the bloodstream. This decreased oxygen transfer results in lower oxygen delivery to the brain and other organs, and produces symptoms of shortness of breath, particularly with exertion; chronic, dry, hacking cough; fatigue and weakness, chest discomfort, loss of appetite and rapid weight loss. While estimates vary, it is believed that IPF could affect approximately 130,000 patients in the US and approximately 76,000 patients in Europe. There is no disease-modifying therapy for IPF, and the only treatment that results in significant improvement is lung transplant.
Promedior is a clinical-stage biotechnology company pioneering the development of targeted therapeutics to treat diseases involving fibrosis. Fibrosis is a harmful process that occurs in many diseases, when normal healthy tissue is replaced with scar tissue, compromising function and ultimately leading to organ failure. Promedior’s proprietary platform is based upon Pentraxin-2, a naturally-occurring human protein that is specifically active at the site of tissue damage and works as an agonist, preventing and potentially reversing fibrosis.
By acting as a master regulator upstream in the fibrosis cascade, pentraxin-2 therapeutics harness the innate healing power of the immune system and open up new potential to treat a wide range of systemic fibrotic diseases for which there are no approved therapies. Promedior has successfully advanced lead therapeutic candidates in human clinical trials, and is initially focused on rare fibrotic diseases, including idiopathic pulmonary fibrosis (IPF), myelofibrosis, and fibrovascular retinal diseases such as Age Related Macular Degeneration (AMD). Promedior is backed by leading global healthcare venture investors, has a significant intellectual property estate relating to the discoveries and applications of pentraxin-2 therapeutics and is led by an experienced management team.
For additional information about Promedior, please visit www.promedior.com
European Medicines Association's Committee for Orphan Medicinal Products "Public summary of opinion on orphan designation. Recombinant human pentraxin-2 for the treatment of idiopathic pulmonary fibrosis." EMA/COMP/441505/2012. 28 August 2012.
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