NASDAQ OMX

Abeona Receives FDA Regenerative Medicine Advanced Therapy Designation for EB-101 Gene Therapy in Epidermolysis Bullosa

Dela
  • 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:

https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ucm537670.htm

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.

Investor Contact:
Christine Silverstein
SVP, Investor Relations & Finance
Abeona Therapeutics Inc.
+1 (646) 813-4707
csilverstein@abeonatherapeutics.com

Media Contact:
Lynn Granito
Berry & Company Public Relations
+1 (212) 253-8881
lgranito@berrypr.com

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.




This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
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 OMX
NASDAQ OMX



Följ NASDAQ OMX

Abonnera på våra pressmeddelanden.

Senaste pressmeddelandena från NASDAQ OMX

Skybox Security Expands Footprint in APAC with New Channel Partners and Leadership in Australia20.6.2018 00:00Pressmeddelande

SAN JOSE, Calif., June 19, 2018 (GLOBE NEWSWIRE) -- Skybox(TM) Security, a global leader in cybersecurity management, today announced that two Australia-based firms, NEXTGEN and Insentra have joined its Australia channel partner program. In addition, Skybox has filled a key leadership role in Australia and New Zealand, continuing the company's expansion in the Asia Pacific region (APAC), which is among the company's fastest-growing global markets. The Skybox APAC channel program enables managed security service providers (MSSPs), value-added resellers (VARs), distributors and system integrators to meet the needs of the market by deploying and offering services that leverage the Skybox(TM) Security Suite. By adding the Suite to their offerings, local partners can continue to serve as trusted cybersecurity advisors to Australian businesses. "Skybox is working with many of the world's largest, most complex networks, providing security teams with the visibility and context needed to addres

Platinum Equity to Sell Worldwide Flight Services for €1.2 Billion19.6.2018 15:00Pressmeddelande

Successful investment adds to Platinum Equity's continued momentum in Europe LOS ANGELES, June 19, 2018 (GLOBE NEWSWIRE) -- Platinum Equity today announced it has signed a definitive agreement to sell Worldwide Flight Services (WFS) to affiliates of Cerberus Capital Management, L.P. in a transaction valued at approximately €1.2 billion. The sale is expected to close during Q4 2018, subject to regulatory approval and certain closing conditions. Since Platinum Equity acquired the company in 2015, WFS has grown significantly, both organically and through a transformative add-on acquisition, and is now the world's largest air cargo handler and one of the world's leading providers of ground handling and technical services, with annual revenues of more than €1.2 billion. "Through a combination of growth and operational initiatives, WFS has evolved and expanded significantly over the past three years and today enjoys record top-line and earnings performance," said Platinum Equity Partner Loui

Quantenna's New QSR10GU-AX Plus Raises the Bar for Wi-Fi Performance and Functionality19.6.2018 14:00Pressmeddelande

SAN JOSE, Calif., June 19, 2018 (GLOBE NEWSWIRE) -- Quantenna Communications, Inc. (NASDAQ:QTNA), the innovator and global leader of high performance Wi-Fi solutions, today announced that the QSR10GU-AX Plus, a new enhanced solution targeting gateways and access points based on the draft IEEE 802.11ax standard, is now sampling to select customers. Building upon Quantenna's pioneering QSR10G-AX Wi-Fi platform, QSR10GU-AX Plus offers unique capabilities, including Adaptive 5 GHz, SuperTx and ESP, providing significant performance gains across a variety of home configurations. The QSR10GU-AX Plus can operate concurrently in both 8x8 MIMO in the 5 GHz band and 4x4 MIMO in the 2.4 GHz band supporting a total of 12 streams. Unique features of QSR10GU-AX Plus include: Dynamic switching between 8x8 MIMO and dual 4x4 MIMO in the 5 GHz band by applying adaptive algorithms, enabled by Quantenna's unique baseband and RF architecture design. This provides the end users with the best possible MIMO c

CrownBio Enters Partnership with Pierre Fabre to Accelerate Discovery and Development of Immuno-Oncology Agents19.6.2018 14:00Pressmeddelande

SAN DIEGO, June 19, 2018 (GLOBE NEWSWIRE) -- Crown Bioscience, a global drug discovery and development services company providing translational platforms to advance oncology, inflammation, cardiovascular and metabolic disease research, announces that it has been selected by Pierre Fabre Research Institute as one of their partners to advance their immuno-oncology drug discovery pipeline. CrownBio boasts the world's largest collection of patient-derived xenograft (PDX) models and is a leading provider of humanized solutions for immuno-oncology drug development. Leveraging these exclusive capabilities, CrownBio will engraft PDX models selected by Pierre Fabre into CD34+ humanized mouse models. These customized, humanized models will be used to accelerate Pierre Fabre's innovative immuno-oncology drug discovery pipeline. "Pierre Fabre recognizes the value of CrownBio's extensive PDX collection and their expertise in implementing innovative humanization strategies," said Nathalie Corvaia, P

Glatfelter to Acquire Georgia-Pacific's European Nonwovens Business for $185 Million19.6.2018 13:44Pressmeddelande

YORK, Pa., June 19, 2018 (GLOBE NEWSWIRE) -- June 19, 2018 - Glatfelter (NYSE:GLT), a global supplier of specialty papers and engineered materials, today announced it has signed a definitive agreement to purchase Georgia-Pacific's European nonwovens business (the "Business") for $185 million, subject to customary purchase price adjustments. The proposed transaction includes Georgia-Pacific's operations located in Steinfurt, Germany, along with sales offices located in France and Italy. The Steinfurt facility produces high-quality airlaid products for the table-top, wipes, hygiene, food pad, and other nonwoven materials markets, competing in the marketplace with nonwoven technologies and substrates, as well as other materials focused primarily on consumer based end-use applications. The Steinfurt facility is a state-of-the-art, 32,000-metric-ton-capacity manufacturing facility that employs approximately 220 people. "Glatfelter's agreement to acquire the European nonwovens business demon

Industry stakeholders partner on blockchain based margin and collateral solution19.6.2018 13:00Pressmeddelande

AMSTERDAM, the Netherlands, BRUSSELS, Belgium, LONDON and STOCKHOLM, Sweden, June 19, 2018 (GLOBE NEWSWIRE) -- ABN AMRO Clearing, EuroCCP, Euroclear and Nasdaq have completed a joint proof of concept to make the use of securities more efficient when used to cover margin calls, including after business hours, using blockchain, or distributed ledger technology (DLT). This solution addresses significant business challenges and inefficiencies related to the current provision of collateral to Central Counterparties (CCPs) and has demonstrated that a shared, resilient network can be built between collateral givers, collateral takers and intermediaries. The inefficiencies of collateral processing have been heightened as a result of recent market changes such as extended trading hours by stock exchanges and the requirement to centrally clear derivatives traded bilaterally (OTC) under the European Market Infrastructure Regulation (EMIR). Today, a CCP margin call typically needs to be covered by

I vårt pressrum kan du läsa de senaste pressmeddelandena, få tillgång till pressmaterial och hitta kontaktinformation.

Besök vårt pressrum