Pervasis Case Study

Pervasis Receives Orphan Drug agnomen in Europe for Vascugel to oppose Arteriovenous Access Failure in Patients Undergoing Hemodialysis Cell-Based Therapy Aims to Regulate the tree trunks improve Response, Promoting Vascular Repair, Reducing the Need for Repeat functional Procedures and Improving Patient Outcomes Cambridge, Mass. , March 1, 2011 Pervasis Therapeutics, Inc. announced today that the European commission (EC) has given(p) Orphan Drug Designation for Vascugel for the sustainion of haemodialysis vascular approach path failure in patients with residue stage renal malady (ESRD).The designation follows a positive opinion from The Committee for Orphan medicative Products (COMP) within the European Medicines Agency (EMA) earlier this year. Vascugel is a novel endothelial cell-based therapy that aims to govern the bes healing response following surgical discourses to create vascular entry which are necessary for ESRD patients undergoing hemodialysis.By promotin g and enhancing vascular renovate, Vascugel reduces the invite for repeat surgical interventions and improves over on the whole patient outcomes. ESRD is an asseverated and irreversible rail treated mainly by hemodialysis or kidney transplantation. It is estimated that more than 250,000 ESRD patients in the EU receive hemodialysis, a affinity purification therapy designed to commute critical kidney functions such(prenominal) as filtering waste.Orphan Drug Designation by the EC provides regulatory and financial incentives for companies to break in and market therapies that treat a monstrous or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU). In increment to a 10year period of marketing exclusivity in the EU after produce approval, Orphan Drug Designation provides companies with scientific advice and regulatory assistance from the EMA during the production development phase, direct opening to centralized marketi ng authorization, as intimately as reductions in certain fees. We are committed to bringing Vascugel to patients with end stage renal disease who currently must(prenominal) endure dangerous complications and repeat surgical procedures so they faecal matter continue to receive life-saving hemodialysis treatment, Frederic Chereau, president and chief executive officer of Pervasis. Receiving Orphan Drug Designation in the EU is an important step, as it will help to advance the development process, and enhance our ability to deliver our novel cellbased therapeutic turn up to address this world-shaking unmet medical need. Vascugel has demonstrated proof of concept in two grade 2 clinical trials involving patients with ESRD who require a invariable arteriovenous (AV) access in order to undergo hemodialysis. In these trials, Vascugel exhibited an tenuous safety profile, and encouraging force trends were observed, including improved duration of patency (or unimpeded blood flow) an d a delay in time to first intervention as compared to placebo. In 2009, Pervasis received Orphan Drug Designation from the U.S. nutrient and Drug Administration (FDA) for Vascugel in patients with ESRD. Pervasis announced last month that the FDA had granted Fast Track review status for Vascugel. Last year, Pervasis reached an agreement with the FDA for a stagecoach 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 accounting entry seeking new drug approval.Failure of Hemodialysis Access Points Leads to Poor Outcomes During hemodialysis, blood is removed from the remains, 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 using a synthetic tube to join an artery and vein) are the two capital types of hemodialysis access.Due to an inflammatory cascade triggered by surgical intervention, the vascular access often has difficulty healing, and quickly become unusable or foul rapidly, prompting the need for additional, recurring surgeries to create a new access which can direct to multiple complications. Up to 60 percent of all arteriovenous (AV) grafts require re-intervention after one year. 1, 2 AV access failure is the most common reason for hospitalization among hemodialysis patients and can lead to anemia, infection, w eighter 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 bodys natural healing response. The endothelium is the thin layer of cells that lines the interior shape up of blood vass in the body. Endothelial cells are critical to tissue repair and health, and have a well-understood role in regulating 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 outside of the blood vessel at the AV access range during the surgical intervention to create the access. The endothelial formulation in Vascugel loose several factors that combat inflammation and promote proper vascular healing, decrease 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 allow meliorate outcomes in patients with skirting(prenomina l) artery disease (PAD) following 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 solid tumor growth, cancer recurrence and metastatic disease. About Pervasis Dixon et al.DAC reckon 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 trailing 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 ontogeny groundbreaking endothelial cellbased therapies designed to regulate the bodys natural healing and repair pro cesses in confused critical 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 funding from Flagship Ventures, Polaris Venture Partners, Highland swell Partners and the Richter Family Fund. For more information, please visit www. pervasistx. com. This news release contains certain innovative sta tements 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 query and clinical trials do not demonstrate safety or efficacy in clinical trials and the risk that the company will not stimulate approval to market its products. Company Contact Margaret OToole Pervasis Therapeutics, Inc. 617-871-1201 emailprotected com Media Contact Liz Falcone Feinstein Kean health care 617-256-6622 liz. emailprotected com