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
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.
Følg saker fra GlobeNewswire
Registrer deg med din epostadresse under for å få de nyeste sakene fra GlobeNewswire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra GlobeNewswire
New YPO Global Leadership Survey Finds Young Business Leaders Overwhelmingly Believe Business is a Force For Good23.1.2019 06:01 | Pressemelding
Ninety-three percent surveyed say business should have a positive impact on society DAVOS, Switzerland, Jan. 23, 2019 (GLOBE NEWSWIRE) -- YPO, the premier global leadership organization for more than 27,000 chief executives, conducted a survey of its members in over 130 countries to get their thoughts on if the purpose of business is to have an impact on society beyond profits and wealth. YPO’s 2019 Global Leadership Survey , conducted 23 December 2018 through 4 January 2019 with more than 2,200 CEO respondents, found that a massive cultural shift about the role of business leadership is in progress as indicated by the following findings: • A large majority of respondents (74 percent) say their perspective on their role as a business leader has changed in the past five years. Employees (43 percent), colleagues (40 percent) and their children (37 percent) were cited as some of the top influencers behind this new belief. • Business leaders shared that they are much more inclined to make
Cloudian Provides Seamless Hybrid and Multi-cloud Storage Integration for Veeam Environments22.1.2019 20:05 | Pressemelding
Object Storage Platform Delivers Highly Cost-effective, Exabyte-scalable Backup, Archive and Disaster Recovery Under Single Data Management Architecture SAN MATEO, Calif., Jan. 22, 2019 (GLOBE NEWSWIRE) -- Cloudian today announced that its HyperStore object storage platform integrates seamlessly with Veeam’s newest data protection software release, Veeam Availability Suite 9.5 Update 4. This integration provides new and existing Veeam customers with highly cost-effective, limitlessly scalable storage that works across public and private cloud environments. With data playing an increasingly central role in an organization’s success, keeping it safe and readily available is more critical than ever. However, doing so has become more challenging in the face of continued rapid data growth. Veeam software is designed to provide both data protection and availability for all workloads—physical, virtual and cloud—and one of the key features in the new release is support of S3-compatible object
Brookfield Asset Management Notice of 2018 Year End Results Conference Call and Webcast22.1.2019 15:37 | Pressemelding
Date: Thursday, February 14, 2019 Time: 11:00 a.m. (Eastern Time) BROOKFIELD, NEWS, Jan. 22, 2019 (GLOBE NEWSWIRE) -- Brookfield Asset Management (NYSE:BAM)(TSX:BAM.A)(EURONEXT AMSTERDAM:BAMA): You are invited to participate in Brookfield Asset Management’s 2018 Year End Results Conference Call & Webcast on Thursday, February 14, 2019 at 11:00 a.m. (Eastern Time) to discuss with members of senior management our results and current business initiatives. These results will be released on February 14th before 7:00 a.m. (Eastern Time) and will be available following the release on our website at www.brookfield.com “News – Press Releases.” The Conference Call will also be Webcast live on our website, where it will be archived for future reference. To participate in the Conference Call, please dial 1-866-688-9425 toll free in North America, or for overseas calls please dial 1-409-216-0815 (Conference ID: 7174879) at approximately 10:50 a.m. The Conference Call will also be Webcast live at ht
K2 Acquires Distributor K2NE, Strengthening Its Digital Process Automation Capabilities Across Europe22.1.2019 15:00 | Pressemelding
Acquisition expands the digital process automation company’s presence into new markets Bellevue, Wash., Jan. 22, 2019 (GLOBE NEWSWIRE) -- SourceCode Technology Holdings, Inc. (“K2”), the leader in low-code digital process automation, today announced the acquisition of one of its distributors, K2NE. With this acquisition, K2 establishes a direct presence in mainland Europe, increasing the direct customer footprint to 14 European countries, further investing and committing to growth across the continent. K2 customers will now see increased opportunities and offerings centered around strengthening their digital transformation. K2NE has a demonstrated history of strong business growth and success gaining market shares in Spain, Switzerland and Germany. “We are thrilled to welcome the K2NE team to the K2 family,” said Evan Ellis, K2 CEO. “As two organizations built around customer-first approaches to digital process automation, we can together leverage our common vision, expand our geograph
Altus Group Report Reveals Rapid Acceleration of PropTech Adoption and Rise of Disintermediation as Major Trends Impacting the Commercial Real Estate Industry in 201922.1.2019 15:00 | Pressemelding
Automation, use of AI and disintermediating business models having major impact on CRE industry; growing number of CRE firms are directly investing in PropTech companies TORONTO, Jan. 22, 2019 (GLOBE NEWSWIRE) -- Altus Group Limited (“Altus Group”) (TSX: AIF), a leading provider of software, data solutions and independent advisory services to the global commercial real estate (“CRE”) industry, today released the latest Altus Group CRE Innovation Report. The report provides an outlook on technology trends impacting the global CRE industry and highlights the significant acceleration in adoption of property technology (“PropTech”), the effect of disintermediating technologies, as well as a growing trend of CRE firms investing directly in PropTech companies and startups themselves. According to the report, which is based on a global survey of 400 CRE executives at firms with assets under management (“AUM”) of at least US $250 million representing a total AUM of over US $2 trillion, driving
ATTO Technology is Now Certified DataCore Ready™ and will Showcase at the DataCore Partner Conference in Frankfurt, Germany22.1.2019 14:47 | Pressemelding
AMHERST, N.Y., Jan. 22, 2019 (GLOBE NEWSWIRE) -- ATTO Technology, Inc., a global leader of storage, network connectivity and infrastructure solutions for data-intensive computing environments for over 30 years, is proud to announce ATTO Celerity™ Gen 6 32Gb and 16Gb Fibre Channel host bus adapters (HBAs) are certified DataCore Ready™. In addition, as a member of the DataCore Ready Partner Program, ATTO will highlight its certified connectivity options for software-defined storage and data management solutions at the DataCore Partner Conference January 22-23 in Frankfurt, Germany. “We are excited to certify the ATTO Celerity HBAs as DataCore Ready as they will help IT departments achieve the flexibility and freedom that software-defined storage solutions offer, while providing the network performance and reliability that mission-critical applications need,” said Gerardo Dada, CMO of DataCore Software. “We look forward to seeing the products in action at our annual partner conference.” A