STAINES-UPON-THAMES, United Kingdom and ABINGDON, United Kingdom and BUFFALO, N.Y., Dec. 21, 2018 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, and NPXe Limited (NPXe) today confirmed enrollment of the first patient in the pivotal Phase 3 trial of Xenon Gas for Post Cardiac Arrest Syndrome (PCAS). Xenon gas for inhalation is an investigational drug, the safety and effectiveness of which have not yet been established. Mallinckrodt will pay NPXe $5 million as recognition of this milestone achievement per the terms of the License and Commercialization Agreement between the parties.
The clinical study is targeting approximately 1,400 patients suffering an out-of-hospital cardiac arrest who have been successfully resuscitated, and will compare 24 hours of xenon gas treatment, in combination with targeted temperature management (TTM), with the current standard of care, which is TTM alone. The study is expected to include about 70 study centers across the U.S. and Europe, and will look for an improvement in functional outcomes and reduced mortality rates. The companies expect an interim analysis of the study to occur in the fourth quarter of 2019 and for the study to complete in 2020.
PCAS is a range of complications, principally to the brain and the heart which follow the sudden loss of blood flow (ischemia) after a cardiac arrest and the subsequent reflow of blood (reperfusion) following successful resuscitation. There is no approved pharmacotherapy for neuroprotection for PCAS. There are approximately 140,000 potential cardiac arrest patients in the U.S. and 145,000 in Europe each year who may be eligible for xenon gas treatment, if approved.
The U.S. Food and Drug Administration (FDA) approved a Special Protocol Assessment for the trial and the drug has been granted its Fast Track designation. The companies expect the regulatory submission to be a drug and delivery device combination.
Matt Napoletano, COO of NPXe, commented, "Our goal is to have approximately 70 sites eligible to enroll patients in the XePOHCAS trial in North America and Europe. Many sites have demonstrated substantial interest in participating in the trial due to the unmet medical need."
"The enrollment of the first patient in this study is an important step in addressing this critical unmet medical need," said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. "If the Phase 3 trial is successful, we believe xenon gas for inhalation, if approved, has the potential to change treatment paradigms, adding a therapeutic option in a population of resuscitated cardiac arrest patients."
Learn more about the U.S. trial on clinicaltrials.gov here.
This announcement contains inside information for the purposes of Article 7 of Regulation (EU) 596/2014.
About Xenon Gas for Inhalation in Post-Cardiac Arrest Syndrome (PCAS)
Xenon is a noble gas that has been used as an inhaled therapy in several studies to date. In PCAS, more N-methyl-D-aspartate receptors (NMDAR, a calcium channel found in neurons) are overactivated, causing extreme ion imbalances, neuronal damage and cell death. Studies have shown that xenon may help to inhibit the NMDAR through a unique inhibition at the glycine-binding site and can help moderate the flow of damaging ions through the calcium channel. By mitigating neuronal damage and cell death, xenon may be able to improve functional outcomes and reduce mortality rates in survivors. The FDA has not yet established the safety and effectiveness of xenon gas for inhalation for PCAS.
About NPXe Limited
NPXe Limited is developing xenon gas for inhalation for the treatment of Post-Cardiac Arrest Syndrome. The Company has a Special Protocol Agreement with the FDA and A Scientific Working Party Agreement with the European Medicines Agency (EMA). Both the FDA and EMA have granted the sought PCAS indication with Orphan Drug status. Orphan status prolongs the period of market exclusivity post approval. The FDA has also granted the drug Fast Track designation. Mallinckrodt has licensed the rights to market xenon gas for inhalation in North America, Australia and Japan upon approval in each country. The rights to market xenon gas for inhalation in the EU, United Kingdom, Norway, Switzerland and Iceland are licensed to a third party.
Mallinckrodt is a global business that develops, manufactures, markets and distributes specialty pharmaceutical products and therapies. Areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, nephrology, pulmonology and ophthalmology; immunotherapy and neonatal respiratory critical care therapies; analgesics and gastrointestinal products. To learn more about Mallinckrodt, visit www.mallinckrodt.com.
Mallinckrodt uses its website as a channel of distribution of important company information, such as press releases, investor presentations and other financial information. It also uses its website to expedite public access to time-critical information regarding the company in advance of or in lieu of distributing a press release or a filing with the U.S. Securities and Exchange Commission (SEC) disclosing the same information. Therefore, investors should look to the Investor Relations page of the website for important and time-critical information. Visitors to the website can also register to receive automatic e-mail and other notifications alerting them when new information is made available on the Investor Relations page of the website.
CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING STATEMENTS
This release includes forward-looking statements concerning xenon gas for inhalation including expectations with regard to clinical trials, future regulatory actions and potential impact on patients. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; issues with product quality, manufacturing or supply, or patient safety issues; future commercialization efforts; and other risks identified and described in more detail in the "Risk Factors" section of Mallinckrodt's most recent Annual Report on Form 10-K and other filings with the SEC, all of which are available on its website. The forward-looking statements made herein speak only as of the date hereof and Mallinckrodt does not assume any obligation to update or revise any forward-looking statement, whether as a result of new information, future events and developments or otherwise, except as required by law.
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© 2018 Document #US-1800377
 The Special Protocol Assessment allows for an interim analysis at 50% of trial completion of the primary endpoint follow up, and if the primary and secondary endpoints are positive at that review the trial would be halted for success. If the primary or secondary endpoints are futile at that point, the trial will be stopped for futility
 The Fast Track designation is provided to drug candidates that "treat a serious condition and fill an unmet medical need." The key benefits to recipients of a Fast Track designation include more frequent contact with the FDA on the development program and the option of Rolling Review, which allows a company to submit completed sections of the New Drug Application (NDA) for individual review by the Agency. This compares with the normal process where the entire NDA must be completed before submission. The frequency of communication under the Rolling Review better assures that questions and issues are resolved quickly, potentially leading to earlier drug approval and patient access.
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