Pervasis Case Study

Pervasis Receives Orphan dose date in Europe for Vascugel to Prevent Arteriovenous Access Failure in patients Undergoing hemodialysis Cell-Based Therapy Aims to consecrate the Bodys Healing Response, Promoting Vascular Repair, Reducing the Need for Repeat Surgical Procedures and Improving Patient Outcomes Cambridge, Mass. , March 1, 2011 Pervasis Therapeutics, Inc. announced today that the European Commission (EC) has granted Orphan Drug Designation for Vascugel for the cake of hemodialysis vascular access failure in patients with end stage renal disease (ESRD).The assigning follows a positive opinion from The Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA) preliminary this year. Vascugel is a novel endothelial carrell-based therapy that aims to regulate the bodys healing rejoinder following running(a) interventions to create vascular access which are necessary for ESRD patients to a lower placegoing hemodialysis.By promoting and enhancing vascular repair, Vascugel reduces the need for repeat surgical interventions and improves boilersuit patient outcomes. ESRD is an advanced and irreversible condition diplomacyed mainly by hemodialysis or kidney transplantation. It is estimated that much than 250,000 ESRD patients in the EU receive hemodialysis, a blood purification therapy designed to replace critical kidney functions much(prenominal) as filtering waste.Orphan Drug Designation by the EC provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five dollar bill in 10,000 persons in the European Union (EU). In addition to a 10year period of marketing exclusivity in the EU after intersection point approval, Orphan Drug Designation provides companies with scientific advice and regulatory assistance from the EMA during the product development phase, direct access to centralized mark eting authorization, as well as reductions in veritable fees. We are committed to bringing Vascugel to patients with end stage renal disease who currently moldiness rear serious complications and repeat surgical procedures so they force out continue to receive life-saving hemodialysis treatment, Frederic Chereau, president and knob executive officer of Pervasis. Receiving Orphan Drug Designation in the EU is an important step, as it forget help to advance the development process, and get up our ability to deliver our novel cellbased therapeutic approach to cover this signifi stubt unmet medical need. Vascugel has exhibit proof of concept in two Phase 2 clinical trials involving patients with ESRD who require a permanent arteriovenous (AV) access in order to undergo hemodialysis. In these trials, Vascugel exhibited an glorious safety profile, and encouraging efficacy trends were observed, including improved duration of patency (or unimpeded blood hunt) and a chequer in tim e to first intervention as compared to placebo. In 2009, Pervasis received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for Vascugel in patients with ESRD. Pervasis announced last month that the FDA had granted Fast spread over review status for Vascugel. Last year, Pervasis reached an agreement with the FDA for a Phase 3 clinical trial of Vascugel under the Agencys Special Protocol Assessment (SPA) procedure, whereby FDA formalized its agreement that the design of the Phase 3 trial was acceptable to support a regulatory submission seeking new drug approval.Failure of Hemodialysis Access Points Leads to Poor Outcomes During hemodialysis, blood is removed from the body, filtered through a dialyzer, or artificial kidney, and then returned to the body. Patients must undergo a surgical intervention to create a vascular access that enables blood to flow from the body to the dialyzer and back to the body. AV fistulae (created by directly joining an artery and vein) and AV grafts (created development a synthetic tube to join an artery and vein) are the two primary types of hemodialysis access.Due to an incendiary cascade triggered by surgical intervention, the vascular access often has difficulty healing, and quickly become unserviceable or clot rapidly, prompting the need for additional, recurring surgeries to create a new access which can lead to multiple complications. Up to 60 percent of all arteriovenous (AV) grafts require re-intervention after integrity year. 1, 2 AV access failure is the most parking lot reason for hospitalization among hemodialysis patients and can lead to anemia, infection, weight loss, jaundice, prolonged bleeding, and other serious complications. Vascugel Combating Inflammation and Promoting Healing Pervasis novel approach to cell therapy uses adult-differentiated allogeneic endothelial cells (donor endothelial cells with a highly targeted biologic function) embedded in a polymer matrix to enhance the bod ys natural healing response. The endothelium is the thin layer of cells that lines the interior surface of blood vessels in the body. Endothelial cells are critical to tissue repair and health, and have a well-understood role in rule many of the bodys healing processes, including those associated with vascular repair.Vascugel, which utilizes Pervasis patented endothelial cell-based platform, is placed on the outdoors of the blood vessel at the AV access site during the surgical intervention to create the access. The endothelial formulation in Vascugel secrete several factors that combat inflammation and promote proper vascular healing, reduce thrombosis (or clotting) and the formation of intimal hyperplasia, or a thickening of the blood vessel wall in response to injury. After approximately four to eight weeks, Vascugel is safely resorbed by the body.Pervasis other areas of clinical investigation take on improving outcomes in patients with peripheral artery disease (PAD) followi ng surgical procedures such as percutaneous transluminal angioplasties (PTAs) with stenting, the failures of which result in serious complications and a significant increase in medical costs. Earlier this year, Pervasis announced it has also embarked on an oncology development program using its proprietary endothelial cellbased platform to prevent inviolable tumor growth, cancer recurrence and metastatic disease. About Pervasis Dixon et al.DAC Study Group. Effect of dipyridamole plus aspirin on hemodialysis graft patency. N Engl J Med. 2009 360 2191-2201. 2 Hayashi et al. Vascular access for hemodialysis. Nat Clin Pract Nephrol 2006 2 504-513 3 Castner D. Recommendations for track arteriovenous access complications using a charting-by-exception model. Anna Journal, 1998 25(4) 393-396. 1 Pervasis Inc. is a clinical stage company that is exploitation groundbreaking endothelial cellbased therapies designed to regulate the bodys natural healing and repair processes in various critic al therapeutic areas.The company has initially focused on developing therapies to improve the outcomes of common vascular interventions, such as arteriovenous access, angioplasties, stent placements, and peripheral and coronary bypass grafts the failure of which result in serious complications and a significant increase in medical costs. The companys lead program, Vascugel, has demonstrated proof of concept and safety in two Phase 2 clinical trials.Pervasis is also applying its endothelial cell-based platform technology to develop an oncology therapy focused on preventing solid tumor growth, cancer recurrence and metastatic disease, as well as products for inflammatory disease and orthopedic injury. Pervasis is a privately held company with livelihood from Flagship Ventures, Polaris Venture Partners, Highland Capital Partners and the Richter Family Fund. For more information, please visit www. pervasistx. com. This news release contains certain(a) forward-looking statements that involve risks and uncertainties.Such statements are only predictions and the companys actual results may differ materially from those anticipated in these forward-looking statements. Factors that may cause such differences include the timing of clinical trials, the risk that products that appeared promising in early research and clinical trials do not demonstrate safety or efficacy in clinical trials and the risk that the company will not obtain approval to market its products. Company speck Margaret OToole Pervasis Therapeutics, Inc. 617-871-1201 emailprotected com Media Contact Liz Falcone Feinstein Kean Healthcare 617-256-6622 liz. emailprotected com