Abeona Receives FDA Regenerative Medicine Advanced Therapy Designation for EB-101 Gene Therapy in Epidermolysis Bullosa
- First gene therapy RMAT designation for Epidermolysis Bullosa
- Enables accelerated approval path and real world data usage
NEW YORK and CLEVELAND, Jan. 29, 2018 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, announced today that the US Food and Drug Administration (FDA) has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to EB-101, the Company's gene-corrected autologous cell therapy product for patients with recessive dystrophic epidermolysis bullosa (RDEB).
"EB-101 is an autologous gene-corrected cell therapy approach that utilizes a patient's own cells and genetically re-engineers them to produce the missing collagen protein, which helps hold skin on to the body. This reduces the number of painful blisters caused by injury and has demonstrated improved wound healing in our Phase 1/2 clinical trial for over 2 years," said Timothy J. Miller, Ph.D., President and CEO of Abeona. "The receipt of the RMAT and Breakthrough designations, both over the last six months, reaffirms the significance of the EB-101 clinical trial results and the need to advance promising therapies in areas of considerable unmet medical need. We are pleased that the FDA granted the RMAT designation, which will help accelerate the development of EB-101, and look forward to continuing our collaborative discussions in defining the pathway forward for the Phase 3 trial set to begin later this year."
Established under the 21st Century Cures Act, the RMAT designation is an expedited program for the advancement and approval of regenerative medicine products where preliminary clinical evidence indicates the potential to address unmet medical needs for life-threatening diseases or conditions. Similar to Breakthrough Therapy designation, the RMAT allows companies developing regenerative medicine therapies to work more closely and frequently with the FDA, and RMAT-designated products may be eligible for priority review and accelerated approval. In November 2017, the FDA expanded the RMAT designation for gene therapies. The sponsor of a RMAT therapy that is granted accelerated approval and is subject to post-approval requirements may, as appropriate, fulfill such requirements through submission of clinical evidence, clinical studies, patient registries, or other sources of real world data. For information on RMAT designation, visit the FDA website:
The Company continues to engage the FDA on its pivotal Phase 3 clinical trial design, and will provide an update on the program in the coming months. Abeona's EB-101 product is an autologous, ex-vivo gene-corrected cell therapy in which the COL7A1 gene is inserted into a patient's own skin cells (keratinocytes) for the treatment of the underlying disease in Recessive Dystrophic Epidermolysis Bullosa. The EB-101 program has been granted Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease Designations from the US Food and Drug Administration (FDA) and Orphan Drug Designation from the European Medicines Agency (EMA).
About EB-101: In the completed Phase 1/2 clinical trial, EB-101 was administered to non-healing chronic wounds on each subject and assessed for wound healing at predefined time points. The trial met the primary endpoints for safety and efficacy, where wound healing after EB-101 administration was compared to control untreated wounds from a supporting natural history study that evaluated 128 patients and approximately 1500 chronic and recurring RDEB wounds. Secondary endpoints included expression of collagen C7 and restoration of anchoring fibrils at three and six-months post-administration. Clinical data demonstrated that EB-101 treated wounds were significantly healed >50% for more than two years post-administration. The data included:
Wound healing, defined as >50% closure after EB-101 administration, was observed in:
--- 100% (42/42 treated wounds, n=7 subjects) at 3 months;
--- 90% (38/42 treated wounds, n=7 subjects) at 6 months;
--- 83% (20/24 treated wounds, n=4 subjects) at 12 months;
--- 88% (21/24 treated wounds, n=4 subjects) at 24 months;
--- 100% (6/6 treated wounds, n=1 subject) at 36 months post-administration.
Collagen VII (C7) expression: C7 and morphologically normal NC2 reactive anchoring fibrils were observed as early as 1 month in EB-101 treated wounds and have remained for at least two years post-administration. Importantly, data from a supportive natural history study of approximately 1,500 wounds from 128 patients with RDEB, established by Stanford and EBCare Registry, were also presented to the FDA. Notably, 13 RDEB patients with a total of 15 chronic wounds were treated with an allograft product, including Apligraf® and Dermagraft®. Of these wounds treated with allografts, only 7% (1/15 treated wounds) remained healed after 12 weeks, and 0% (0/15 treated wounds) remained healed after 24 weeks. This is a meaningful finding of the natural history study, as there are no approved therapies for RDEB patients that demonstrate significant wound closure after two months post-application.
About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and ABO-101 (AAV-NAGLU), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type B (MPS IIIB). Abeona is also developing ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona is developing a proprietary vector platform, AIM(TM), for next generation product candidates. For more information, visit www.abeonatherapeutics.com.
SVP, Investor Relations & Finance
Abeona Therapeutics Inc.
+1 (646) 813-4707
Berry & Company Public Relations
+1 (212) 253-8881
This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include, without limitation, our plans for continued development and internationalization of our clinical programs, that patients will continue to be identified, enrolled, treated and monitored in the EB-101 clinical trial, and that studies will continue to indicate that EB-101 is well-tolerated and may offer significant improvements in wound healing. These statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the ability to secure licenses for any technology that may be necessary to commercialize our products; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Abeona Therapeutics Inc via Globenewswire
Om Nasdaq GlobeNewswire
One Liberty Plaza - 165 Broadway
NY 10006 New York
+1 212 401 8700http://www.nasdaqomx.com
NASDAQ (NASDAQ: NDAQ) is a leading provider of trading, exchange technology, information and public company services across six continents.
Følg saker fra Nasdaq GlobeNewswire
Registrer deg med din epostadresse under for å få de nyeste sakene fra Nasdaq GlobeNewswire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra Nasdaq GlobeNewswire
INTA Selects Singapore for 142nd Annual Meeting Slated for April 202021.5.2018 02:10 | Pressemelding
SEATTLE, May 20, 2018 (GLOBE NEWSWIRE) -- The International Trademark Association (INTA) today announced that it will hold its 142nd Annual Meeting-the world's largest trademark event-in Singapore from April 25 to 29, 2020, at the Marina Bay Sands. This marks the second time INTA is bringing its Annual Meeting to Asia, and the first time that the organization will host the event in Southeast Asia. INTA CEO Etienne Sanz de Acedo made the highly anticipated announcement at the opening ceremonies of the Association's 140th Annual Meeting, taking place in Seattle, Washington, now through May 23. Nearly 11,000 trademark practitioners and other intellectual property (IP) professionals from 150+ countries are gathering for this event. The number of registrants has been increasing each year. In 2014, INTA held its first-ever Annual Meeting in Asia-the 136th Annual Meeting in Hong Kong SAR, China. After that successful event, INTA's Board of Directors approved a rotation strategy to host the An
GlobeNewswire Test Release19.5.2018 07:00 | Pressemelding
LOS ANGELES, May 18, 2018 (GLOBE NEWSWIRE) -- This is a test from GlobeNewswire. Readers are advised to disregard. TESTING -- TESTING -- TESTING -- TESTING -- TESTING -- TESTING -- TESTING -- TESTING – -END- CONTACT: Karen Yu Director, GlobeNewswire Product Management Direct: +1 310 258 6917 Mobile: +1 626 389 7427 or Magali Rolandez Senior Manager, Media Relations/Content Distribution Europe Direct: +44 (0) 203 753 2215 Mobile: +44 (0) 7785 619 539
BK Ultrasound, Powered by Analogic, Launches bkSpecto Ultrasound System for Urology at the 2018 American Urological Association Annual Meeting18.5.2018 23:23 | Pressemelding
PEABODY, Mass., May 18, 2018 (GLOBE NEWSWIRE) -- Analogic Corporation (Nasdaq:ALOG), enabling the world's medical imaging and aviation security technology, today announced that it will launch the bkSpecto(TM) ultrasound system at the 113th AUA (American Urological Association) Annual Meeting, May 18-21, in San Francisco. bkSpecto-taken from the Latin word Specto, meaning 'I see'-is BK Ultrasound's all-new ultrasound platform designed for clinical practices. bkSpecto combines BK's advanced auto-optimization algorithms with faster processing to render exceptional anatomical imaging detail with minimal manual adjustment by the user. The system has a unique glass touch-based interface using BK's Tru-Sense(TM) technology to provide 'sightless navigation.' The interface enables tactile feedback with or without gloves, providing an intuitive, self-guided user experience. The interface is also customizable, enabling personalized and faster user workflows. The small footprint of bkSpecto makes
Brookfield Asset Management Announces Renewal of Normal Course Issuer Bid18.5.2018 12:55 | Pressemelding
TORONTO, May 18, 2018 (GLOBE NEWSWIRE) -- Brookfield Asset Management Inc. (NYSE:BAM) (TSX:BAM.A) (EURONEXT:BAMA) ("Brookfield") today announced it has received approval from the Toronto Stock Exchange ("TSX") for the renewal of its normal course issuer bid to purchase up to 82,315,909 Class A Limited Voting Shares ("Class A Shares"), representing 10% of the public float of Brookfield's outstanding Class A Shares. Purchases under the bid will be made through the facilities of the TSX, the New York Stock Exchange ("NYSE") and any alternative Canadian trading system. The period of the normal course issuer bid will extend from May 24, 2018 to May 23, 2019, or an earlier date should Brookfield complete its purchases. Brookfield will pay the market price at the time of acquisition for any Class A Shares purchased. As at May 1, 2018, the number of Class A Shares issued and outstanding totalled 991,857,711, of which 823,159,095 shares represented the public float. In accordance with the rules
Skybox Security and Exclusive Networks Announce New Partnership for Middle East Distribution of the Skybox Security Suite17.5.2018 18:41 | Pressemelding
The cybersecurity management platform ensures networks remain secure and compliant as organisations migrate to cloud and merge networks SAN JOSE, Calif., May 17, 2018 (GLOBE NEWSWIRE) -- Skybox(TM) Security, a global leader in cybersecurity management, announced today a new partnership with Exclusive Networks for distribution of Skybox solutions in the Middle East, including the Gulf Cooperation Council (GCC) region and Egypt. Exclusive Networks will deploy and service the Skybox(TM) Security Suite, a broad security management platform that provides comprehensive attack surface visibility and enables unified vulnerability management and firewall/security policy management across an organization's entire network, including in physical, multi-cloud and OT environments. "Driven by government outlays (with oil, gas and defence industries leading the charge), Middle East organisations are looking for trusted security advisors who can navigate digital transformations and reduce risks - espec
Dermatological Symposium Tackles the Effects of Pollution on Skin17.5.2018 16:03 | Pressemelding
Researchers Present Compelling Evidence Showing the Effects of Pollution on Skin Health at Symposium Sponsored by Mary Kay Inc. DALLAS, May 17, 2018 (GLOBE NEWSWIRE) -- Mary Kay Inc. announced four keynote speakers for its symposium to be held during the prestigious International Investigative Dermatology (IID) Meeting on May 16 to 19, 2018 in Orlando, Florida. The IID meeting occurs once every 5 years, and brings together scientists from across the globe, highlighting the latest in cutting edge science in dermatology. The Mary Kay sponsored Symposium features four global experts including Dr. Liang Liu, Assistant Professor of Dermatology, Columbia University; Dr. Max Costa, Professor and Chairman of the department of Environmental Medicine, New York University School of Medicine; Dr. Jean Krutmann, Chair of Environmental Medicine at the Medical Faculty of Heinrich Heine University Düsseldorf and Director of the Leibniz Research Institute of Environmental Medicine in Dusseldorf, German
I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.Besøk vårt presserom