Wave Life Sciences Announces Initiation of DYSTANCE 51, a Phase 2/3 Clinical Trial of Suvodirsen
Global study designed to assess the efficacy and safety of suvodirsen in DMD patients amenable to exon 51 skipping
Results intended to support regulatory approvals globally
CAMBRIDGE, Mass., June 27, 2019 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced the initiation of DYSTANCE 51, the Phase 2/3 efficacy and safety clinical trial of suvodirsen, an investigational stereopure oligonucleotide intended for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
“The initiation of the DYSTANCE 51 Phase 2/3 study represents a significant milestone for Wave and our DMD clinical program, as well as an important development for the families affected by Duchenne,” said Michael Panzara, MD, MPH, Chief Medical Officer of Wave Life Sciences. “We are grateful to be working in collaboration with the U.S. Food and Drug Administration, global regulators and our partners from the Duchenne community on this innovative and exciting trial. We are proud to have worked with patient groups to incorporate their feedback into the trial design to better address the needs of boys and families living with DMD.”
"We are pleased that Wave Life Sciences has incorporated important input from the Duchenne community in designing their Phase 2/3 clinical trial of suvodirsen. This global study is designed around the needs of families while measuring a robust set of biomarker and functional endpoints. PPMD looks forward to learning more about this stereopure exon-skipping program when Wave presents at our 25th Annual Conference later this week," said Pat Furlong, founding President and CEO of Parent Project Muscular Dystrophy (PPMD).
DYSTANCE 51 is a global study and is expected to include clinical trial sites in the United States, Europe, Australia, Canada and Japan. Results from this trial are intended to support global regulatory filings for suvodirsen.
DYSTANCE 51 Phase 2/3 Clinical Trial Design
DYSTANCE 51 is a global Phase 2/3, multicenter, randomized, double-blind, placebo-controlled clinical trial that will evaluate the efficacy and safety of suvodirsen in ambulatory boys who are between 5 and 12 years of age (inclusive) with a genetically confirmed diagnosis of DMD amenable to exon 51 skipping. The trial will test two dose levels of suvodirsen and is expected to enroll approximately 150 patients. The primary efficacy endpoints of the clinical trial will measure change in dystrophin protein level and change in the North Star Ambulatory Assessment (NSAA) score. In addition to the NSAA, the trial will include other functional outcome measures as secondary efficacy endpoints.
The DYSTANCE 51 clinical trial is the first study ever selected by the U.S. Food and Drug Administration (FDA) for its pilot program for complex innovative trial designs. As a participant in the pilot program, the company has held multiple meetings with FDA staff to discuss the design elements of the trial, including the potential use of Bayesian methods to adapt the trial with the aim of maximizing efficiency while ensuring robust clinical results.
Suvodirsen is an investigational stereopure oligonucleotide currently being evaluated in an ongoing open-label extension (OLE) study for the treatment of boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Data from the company’s completed Phase 1 clinical trial of suvodirsen demonstrated a favorable safety and tolerability profile after single doses, supporting its continued clinical development, including the launch of DYSTANCE 51 and continuation of the OLE study. Wave remains on track to deliver an interim analysis of dystrophin expression from muscle biopsies in boys receiving suvodirsen in the OLE study in the second half of 2019.
Approximately 13% of DMD patients have genetic mutations that are amenable to treatment with an exon 51 skipping therapy. Exon-skipping technology has the potential to induce cellular machinery to ‘skip over’ a targeted exon and restore the reading frame, resulting in the production of internally truncated, but functional dystrophin protein.
Suvodirsen has been granted orphan drug designation for the treatment of DMD by the FDA and the European Commission, as well as rare pediatric disease designation by the FDA. Pending positive clinical dystrophin expression data, the company expects to file for an accelerated approval of suvodirsen in the United States in the second half of 2020.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic neuromuscular disorder caused predominantly by out-of-frame deletions in the dystrophin gene, resulting in absent or defective dystrophin protein. Dystrophin protein is needed for normal muscle maintenance and operation. Because of the genetic mutations in DMD, the body cannot produce functional dystrophin, which results in progressive and irreversible loss of muscle function, including the heart and lungs. Worldwide, DMD affects approximately one in 5,000 newborn boys.
About Wave Life Sciences
Wave Life Sciences (NASDAQ: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop best-in-class medicines across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future. To find out more, please visit www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the countries in which the Phase 2/3 clinical trial for suvodirsen is expected to be conducted, the expected timing and plans to report interim data from the ongoing OLE, the belief that the safety and tolerability data from the Phase 1 clinical trial support initiation of the Phase 2/3 clinical trial, the anticipated benefits of participating in FDA’s complex innovative trial designs pilot program, the intention to use the results of the OLE and Phase 2/3 trials to seek various regulatory approvals globally and the anticipated timing of such regulatory filing in the U.S. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release. These risks and uncertainties include but are not limited to the following: Wave’s current and planned clinical trials, other studies for suvodirsen and Wave’s other product candidates may not be successful or may take longer and be more costly than anticipated; product candidates that appeared promising in earlier research and clinical trials may not demonstrate safety and/or efficacy in later-stage or larger-scale clinical trials; and the other risk factors discussed under the heading “Risk Factors” contained in Wave’s Annual Report on Form 10-K for the year ended December 31, 2018 filed with the Securities and Exchange Commission (SEC), as well as in other filings Wave makes with the SEC from time to time. All statements contained in this press release are made only as of the date of this press release, and Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances.
Media and Patient Contact:
One Liberty Plaza - 165 Broadway
NY 10006 New York
GlobeNewswire is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.
Subscribe to releases from GlobeNewswire
Subscribe to all the latest releases from GlobeNewswire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from GlobeNewswire
XBT Provider AB (Publ) Publishes Half Year Interim Report22.7.2019 23:44:00 CEST | Press release
Stockholm, July 22, 2019 -- XBT Provider AB (Publ) ("XBT" or the "Company") is pleased to announce that the Interim Report for the period ended 30 June 2019 has been posted on the Company's website. The Guarantor, CoinShares (Jersey) Limited, has also provided an unaudited set, of their financial statements for the period ending on 30 June 2019, which are also posted on the Company's website. Interested persons may access an electronic copy of these documents at www.xbtprovider.com ### About XBT Provider XBT Provider AB (Publ) (XBT Provider), a CoinShares company, is the Swedish-domiciled issuer of the Bitcoin Tracker One (SE0007126024), Bitcoin Tracker Euro (SE0007525332), Ether Tracker One (SE0010296574) and Ether Tracker Euro (SE0010296582), Litecoin Tracker One (SE0011414465), Litecoin Tracker Euro (SE0011414457), XRP Tracker One (SE0011414481), XRP Tracker Euro (SE0011414473), series of certificates (collectively, the “Certificates”) which are designed to synthetically track the p
PRESS RELEASE: BIGBEN INTERACTIVE: 1ST QUARTER SALES OF 58.6 M€ UP 20.3 %22.7.2019 18:00:00 CEST | Press release
Press Release Lesquin, 22 July 2019 18:00 1st quarter sales of 58.6 M€ up 20.3 % Strong rise of Gaming (+27.3%) Increase of Current Operating Profit rate for FY 2019/20 now set in excess of 10% IFRS – M€ Sales Non audited data 2019/2020 2018/2019 Change 1st Quarter Of which Gaming Of which Mobile Of which Audio 58.6 29.9 23.1 5.5 48.7 23.5 21.1 4.1 + 20.3% + 27.3% + 9.7% + 34.5% Strong growth in Gaming in Q1 2019/20 In the first quarter of FY 2019/20 (April to June 2019), Bigben achieved sales of 58.6 M€, a 20.3% increase. GAMING The Gaming business recorded a 27.3% growth in Q1 2019/20 with 29.9 M€ sales. Publishing: The shift from the business model towards more digital sales accelerated in Q1 2019/20 with an increase exceeding 250% in this distribution channel. This channel now accounts for more than two-thirds of revenues. The releases of Warhammer®: Chaosbane, The Sinking City, Tennis World Tour® Roland Garros Edition, Pro Cycling Manager 2019 and Tour de France® 2019 also contrib
EVS Broadcast Equipment reports update of share buyback program22.7.2019 17:45:00 CEST | Press release
Publication on July 22, 2019, 5.45pm CET Regulated information – reporting share buyback EVS Broadcast Equipment S.A.: Euronext Brussels (EVS.BR), Bloomberg (EVS BB), Reuters (EVSB.BR) EVS reports update of share buyback program EVS Broadcast Equipment reports that the following transactions, conducted within the framework of the share buyback program announced on October 24, 2018, took place between July 15, 2019 and July 17, 2019. Date Number of shares acquired Average price (EUR) Total (EUR) 15/07/2019 1,856 20.3713 37,809 16/07/2019 1,760 20.0932 35,364 17/07/2019 378 20.9000 7,900 As of July 22, 2019, and since the start of the buyback program, EVS has bought 255,028 shares at an average price of EUR 20.8174, representing in total EUR 5,309,024. After aforementioned transactions the total number of own shares amounts now to 333,676 shares as of July 22, 2019 (including 93,144 shares already held by the company before the start of the share buyback program and taking into account t
Change in Amer Sports Corporation’s financial reporting schedule22.7.2019 17:15:00 CEST | Press release
Amer Sports Corporation STOCK EXCHANGE RELEASE July 22, 2019 at 6:15 p.m. Change in Amer Sports Corporation’s financial reporting schedule Amer Sports changes the publication date of its half-year financial report 2019. The updated schedule for the half-year report is 30 September, 2019 at approximately 1:00 p.m. Finnish time. The previously announced publication date was July 25, 2019. The change is related to the recent ownership change in the company. ANTA Sports Products Limited consolidates Amer Sports as an associated company in its half-year financial report which will be published on 26 August, 2019. On the same day, Amer Sports will publish a stock exchange release disclosing its financial information included in ANTA Sports’ half-year financial report. On March 28, 2019, Mascot Bidco Oy completed a voluntary recommended public cash tender offer for all the issued and outstanding shares in Amer Sports Corporation and on April 1, 2019 announced that it will exercise its redempt
Changes in Huhtamaki’s Global Executive team22.7.2019 17:00:00 CEST | Press release
HUHTAMÄKI OYJ STOCK EXCHANGE RELEASE 22.7.2019 AT 18:00 Changes in Huhtamaki’s Global Executive team Michael Orye, Executive Vice President, Fiber Packaging and a member of Huhtamaki Global Executive Team has decided to leave Huhtamaki to pursue other career opportunities. Eric Le Lay, Executive Vice President Foodservice EAO will assume the leadership and take full responsibility of Fiber Packaging in addition to his current role. This change is effective as of 22.7.2019. “I want to thank Michael for his contribution to Huhtamaki and wish him all success in his future endeavors” says Charles Héaulmé, President and CEO of Huhtamaki. Following the aforementioned change, the members of the Global Executive Team are: Charles Héaulmé (chairman), President and CEO Thomas Geust, Chief Financial Officer; Leena Lie, Senior Vice President, Marketing and Communications; Sami Pauni, Senior Vice President, Corporate Affairs and Legal, Group General Counsel; Teija Sarajärvi, Senior Vice President,
Banks net position in the Riksbank22.7.2019 16:42:00 CEST | Press release
JUL 22, 2019 SEK MILLION LENDING BORROWING 281