Acusphere Announces Submission of Marketing Authorisation Application for Imagify™ in Europe and Start of Procedural Review
Acusphere, Inc. (ACUSD.PK) today announced that it has submitted the Marketing Authorisation Application (MAA) for its lead product candidate, Imagify™ (Perflubutane Polymer Microspheres) lyophilisate for dispersion for injection with the European Medicines Agency (EMA) to support the regulatory review of Imagify in the European Union (EU). The MAA is being submitted via the centralized procedure that would enable simultaneous approval in all EU countries. The validation process for the MAA has been successfully completed, and the substantive review process is now underway by the Committee for Medicinal Products for Human Use (CHMP). The CHMP opinion is expected in Q4 2013.
The MAA submission is supported by a clinical program of 11 scientific studies in humans, including two Phase 3 pivotal studies that were conducted in the targeted patient population - stable chest pain patients with suspected or known coronary artery disease (CAD). The pivotal studies compared the performance of Imagify with stress ultrasound to nuclear stress testing, which is recommended for this patient population by clinical guidelines in Europe and the U.S. The objective of the primary efficacy analysis was to demonstrate that Imagify with stress ultrasound had non-inferior accuracy to nuclear stress testing. The primary endpoint, accuracy, was met in both trials. The results of these trials, called Real Time Assessment of Myocardial Perfusion (RAMP) 1 and RAMP 2, have been published in the European Journal of Echocardiography in a 2009 paper which concluded that Imagify with stress ultrasound was well tolerated and had comparable performance to nuclear stress testing.
“Nuclear stress testing, the current standard of care for assessing myocardial perfusion, is expensive and exposes millions of patients each year to harmful radiation,” said Sherri C. Oberg, President and CEO of the company. “Imagify enables the assessment of myocardial perfusion, the most sensitive, non-invasive marker of CAD, with stress ultrasound, a radiation-free, lower cost test, important benefits in today's cost conscious health care environment. Imagify with stress ultrasound is the first and only test that has demonstrated comparable efficacy to nuclear stress testing in an international, multi-center registration trial. We are very pleased with our continued progress on Imagify's regulatory submission in Europe, since this development is so important for Acusphere as well as for patients who want to avoid radiation exposure, and for a health care system looking for better quality at a lower cost.”
Acusphere owns global rights to Imagify and is in discussions with potential partners with the capabilities and resources required to commercialize Imagify in Europe upon approval and to fund the additional clinical trial required for U.S. approval. Regulatory guidelines in the U.S., which differ from European guidelines, now require placebo trials. Acusphere has reached agreement with the Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) on the design of a placebo trial to demonstrate that stress ultrasound with Imagify has superior efficacy to stress ultrasound without Imagify. Placebo trials are not required under European regulatory guidelines and Acusphere confirmed in its pre-MAA submission meetings with European regulators that this trial would not be required for European approval.
Coronary artery disease (CAD) is the leading cause of death in Europe and throughout the western world, including the U.S. Imagify remains the leading development candidate for myocardial perfusion assessment with ultrasound. Acusphere believes that Imagify continues to show promise as a lower cost radiation-free test for perfusion assessment. The current standard of care, nuclear stress testing, is expensive and each episode of nuclear stress testing delivers a radiation dose equivalent to between 140 and 800 chest x-rays depending upon the radiopharmaceutical used. Based upon an addressable market of at least 3 million stress imaging procedures annually in Europe, the market opportunity for Imagify is $600 million. Acusphere believes this market could grow significantly since the European population is similar to the U.S. population and the prevalence of heart disease is similar as well. In the U.S., the addressable market is approximately 10 million stress imaging procedures with an Imagify market opportunity of $2 billion.
About Acusphere, Inc.
Acusphere, Inc. (ACUSD.PK) is a specialty pharmaceutical company focused on the development and regulatory approval of our lead product candidate, Imagify™ (Perflubutane Polymer Microspheres) lyophilisate for dispersion for injection. Imagify is a cardiovascular drug for the detection of coronary artery disease, the leading cause of death in Europe and the United States. Imagify was created using proprietary technology that enables Acusphere to control the porosity and size of nanoparticles and microparticles, which were customized to control the delivery of gas needed for ultrasound myocardial perfusion assessment. For more information about Acusphere visit the Company's web site (www.acusphere.com).
The above press release contains forward-looking statements, including statements regarding the MAA submission for Imagify and likelihood of regulatory approval and the commercial opportunity for Imagify. There can be no assurance that Imagify will be approved for the indication the Company is seeking, or at all. The Company's actual results may differ materially from those anticipated in these forward-looking statements based upon a number of factors, including uncertainties associated with research, development, testing and related regulatory approvals, uncertainties regarding results of the trial agreed to under the Company's proposed Special Protocol Assessment with FDA and proposed Paediatric Investigational Plan with EMA, and uncertainties regarding the potential effects of not achieving clinical endpoints, capital needs and uncertainty of additional financing, uncertainties regarding the cost, timing and ultimate success of the qualification of the Company's commercial manufacturing facility in accordance with applicable regulatory requirements, complex manufacturing, high quality requirements, lack of commercial manufacturing experience, dependence on third-party manufacturers, suppliers and collaborators, uncertainties associated with intellectual property, competition, loss of key personnel, uncertainties associated with market acceptance and adequacy of reimbursement, technological change and government regulation. The Company notes that effective as of March 3, 2009, pursuant to a Form 15 filing made with the SEC, it is not currently required to file periodic reports with the SEC, including annual reports on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K. Readers are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this press release. The Company undertakes no obligation to publicly release the results of any revisions to these forward-looking statements that may be made to reflect events or circumstances that occur after the date of this press release or to reflect the occurrence of unanticipated events.
Kelley Wharff, 617-925-3444