Nasdaq GlobeNewswire

Abeona Therapeutics Announces Authorization to Commence Phase 1/2 Gene Therapy Clinical Study for Patients with MPS IIIB in Spain

Del
  • Clinical trial for ABO-101 (AAV-NAGLU) marks the 2nd MPS III program in Europe
  • Company plans to initiate additional clinical sites in European countries including France, Germany and the United Kingdom

NEW YORK and CLEVELAND, Sept. 12, 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, today announced authorization to move forward with a Phase 1/2 clinical trial in Spain for the Company’s gene therapy product ABO-101 (AAV-NAGLU) for patients with MPS IIIB (Sanfilippo syndrome type B). The clinical study was approved by the Agencia Espanola de Medicamentos y Productos Sanitarios and is being conducted at Hospital Clinico Universitario of Santiago de Compostela, Spain. This will be the Company’s second clinical trial conducted in Europe, alongside the ongoing Phase 1/2 clinical trial for patients with MPS IIIA (Sanfilippo syndrome type A). Abeona first initiated this trial in the United States. Abeona plans to add clinical sites for the trial in three European countries including France, Germany and the United Kingdom.

“The authorization of our ABO-101 trial in Spain is a significant milestone for children suffering from MPS IIIB, a devastating and deadly disease with no approved treatment options,” stated Carsten Thiel, PhD, CEO. “We are encouraged by the preliminary results observed in our US trial to date, both in clinically relevant biomarkers and in the ongoing safety profile and are excited to bring this therapy to patients in Europe.”

Subjects in the Phase 1/2 trial receive a single, intravenous infusion of ABO-101, which uses an AAV vector to introduce the functional NAGLU gene to treat patients with MPS IIIB disease. Subjects will be evaluated at multiple time points post-injection for safety assessments and efficacy parameters.  The clinical program is supported by a Natural History Study which included potential efficacy assessments consisting of neurocognitive evaluations, biochemical assays and MRI data generated over one year of follow-up assessments.

ABO-101 has been granted Rare Pediatric Disease Designation in the U.S., and Orphan Product Designation in both the U.S. and the European Union.

About ABO-101 (AAV-NAGLU): ABO-101 is Abeona’s first-in-human, adeno-associated viral (AAV)-based gene therapy for MPS III (Sanfilippo syndrome). Treatment involves a one-time intravenous delivery of a functioning copy of the N-acetyl-α-D-glucosaminidase (NAGLU) gene to cells of the central nervous system (CNS) and peripheral organs, with the aim of correcting the effects that result from the genetic aberrations that are the root cause of the disease. Following administration of a single dose in Sanfilippo preclinical animal models, ABO-101 induced cells in the Central Nervous System and peripheral organs to produce the missing NAGLU enzyme, which then restored underlying sugar (glycosaminoglycan or GAG) storage pathology to normal levels in cells. In preclinical in vivo efficacy studies in Sanfilippo syndrome animal model, ABO-101 demonstrated functional benefits that continue for months after treatment. A single dose of ABO-101 significantly restored normal cell and organ function, corrected cognitive defects, increased neuromuscular function and normalized the lifespan of animals with MPS IIIB after treatment compared to untreated control animals. These results are consistent with studies from several laboratories suggesting AAV treatment could potentially benefit patients with Sanfilippo syndrome. Safety and efficacy studies of AAV gene therapy treatments for Sanfilippo syndrome have recently been published in several peer-reviewed scientific journals.

About MPS IIIB: (also known as Sanfilippo syndrome type B) is a genetic, progressive, and devastating rare lysosomal storage disease. In patients with MPS IIIB, genetic mutations result in a marked decrease in NAGLU enzyme activity, which leads to accumulation of heparan sulfate (HS) in the brain and other organs as well as progressive brain atrophy with cortical gray matter volume loss. The accumulation of abnormal HS results in neurocognitive decline, behavioral disturbances, speech loss, increasing loss of mobility, and premature death. MPS IIIB typically presents in children during the first few years of life, and 70% of patients do not reach 18 years of age. There are no approved treatments for MPS IIIB.

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, ABO-202 (AAV-CLN1) for treatment of infantile batten disease, 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™, for next generation product candidates. For more information, visit www.abeonatherapeutics.com.

Investor Contact:
Christine Silverstein
SVP, Finance & Investor Relations
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 Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. Such statements include that we are encouraged by the results of the US trial, and that we plan to add clinical sites for the trial in three European countries.  Actual results  may  differ  materially from those indicated by such forward-looking statements as a result of various important factors, 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 secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain  necessary  regulatory  approvals, the impact of changes in the financial markets and global economic conditions; risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q 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.

Om Nasdaq GlobeNewswire

Nasdaq GlobeNewswire
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

Service Providers Speed Deployment, Reduce Costs with Patton Cloud Edge Orchestration Service20.9.2018 22:16Pressemelding

Now, Telecom and UCaaS service providers can easily provision, manage, monitor, secure, alert, troubleshoot, analyze, and optimize services on the customer premise GAITHERSBURG, Md., Sept. 20, 2018 (GLOBE NEWSWIRE) -- Patton Electronics—US-manufacturer of UC, cloud, and IoT-enabling solutions for carrier, enterprise, and industrial networks—has launched the new Patton Cloud service for carriers and service providers that deliver All-IP and hybrid voice-and-data services to business subscribers. Patton Cloud delivers a low-cost, easy-to-use, web-based edge-orchestration service that delivers a superior user experience. It offers remote touchless provisioning, monitoring, and troubleshooting—as well as security and alerting tools that reduce service turn-up costs, trouble tickets, and subscriber attrition. The Patton Cloud service offers quality-control mechanisms including pro-active monitoring and troubleshooting—combined with feature-license distribution pools for managing value-added

Inspirata Promotes Top Executives to Expanded Roles in Digital Pathology20.9.2018 21:12Pressemelding

Inspirata Founder and Executive Vice President, Mark Lloyd, is taking on an expanded role overseeing product management, and Inspirata Senior Vice President, Andrew Chomos, has been appointed to General Manager, Digital Pathology, overseeing the Center of Excellence in Pittsburgh along with all North America sales and customer support efforts. Tampa, FL, Sept. 20, 2018 (GLOBE NEWSWIRE) -- Cancer informatics and digital pathology workflow solution provider Inspirata®, Inc. announced today that two of its executives have taken on expanded roles in the company. The two are Inspirata Executive Vice President and Founder, Mark Lloyd, and Senior Vice President and General Manager, Digital Pathology, Andrew Chomos. Dr. Lloyd has accepted the expansion of his role to include product management for Digital Pathology. He is former cancer researcher at both Lombardi Cancer and Moffitt Cancer Center and is Inspirata’s foremost subject matter expert and thought leader on digital pathology and image

Tetraphase Pharmaceuticals Announces Adoption of Commission Decision Granting Marketing Authorisation Approval in the European Union for XERAVA™ (eravacycline) for the Treatment of Complicated Intra-Abdominal Infections20.9.2018 19:08Pressemelding

– Broad Product Label for Treatment of Complicated Intra-Abdominal Infections – – Phased Commercial Launch Expected in the First Half of 2019 – WATERTOWN, Mass., Sept. 20, 2018 (GLOBE NEWSWIRE) -- Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on developing and commercializing novel antibiotics to treat life-threatening multidrug-resistant (MDR) infections, today announced that the European Commission (EC) has adopted the Decision granting marketing authorisation for XERAVA (eravacycline) for injection for the treatment of complicated intra-abdominal infections (cIAI) in adults in the European Union. In clinical trials, XERAVA was well-tolerated and achieved high clinical cure rates in patients with cIAI, demonstrating statistical non-inferiority to two widely used comparators – ertapenem and meropenem. “The European approval of XERAVA, right after our recently announced FDA approval, marks our second significant regulatory approval within one month

UPDATE— Taconic Biosciences Becomes the First Animal Model Provider on General Services Administration Contract Schedule20.9.2018 18:07Pressemelding

RENSSELAER, N.Y., Sept. 20, 2018 (GLOBE NEWSWIRE) -- Taconic Biosciences, a global leader in providing genetically engineered rodent model solutions, announces a new relationship with Government Scientific Source (GSS). GSS is a leading dedicated distributor of laboratory equipment to federal, state, and local governments on contract with the GSA (General Services Administration). By having Taconic’s products available via GSS, they can now be accessed by all federal agencies, including but not limited to the National Institutes of Health and the Department of Defense. Taconic is the only animal model provider affiliated with GSS and the only animal model provider available on GSA Contract. “An important component of providing customers with the best animal model solutions is access to key resources. Through this exciting new relationship with GSS, Taconic can now easily connect with scientists doing some of the most valuable research in the world and provide them with high quality sol

Digital Transformation is Not a One-Off — It’s About the Digital Journey20.9.2018 17:44Pressemelding

SoftServe study confirms four digital journey states with 71 percent of enterprises reporting early stages of adoption AUSTIN, Texas, Sept. 20, 2018 (GLOBE NEWSWIRE) -- SoftServe, a leading digital authority and consulting company, today released the results of its market research study that examines how enterprises are responding to the digital economy. The research found that 71 percent of the respondents in the United States and the European Union are in the early stages—Reveal and Transform—of four unique states across the Enterprise Digital Journey. For an in-depth analysis and the full results, read The Enterprise Digital Journey white paper. With fierce competition in today’s global landscape, leveraging cutting-edge technology and deploying new business models is not a one-off pursuit, but rather a continuous process of iteration and refinement. Companies should stop referring to Digital Transformation and instead plan for a continuous Digital Journey. “The term Digital Transfo

Anomali Announces Major Expansion of Threat Platform Partner Ecosystem20.9.2018 16:00Pressemelding

New Developer SDKs enable partners to integrate solutions with Anomali Threat Platform NATIONAL HARBOR, Md., Sept. 20, 2018 (GLOBE NEWSWIRE) -- Anomali today announced at the Detect Conference 2018 the expansion of the partner ecosystem with the launch of a suite of partner developer resources. The new tools enable easier and faster integration with the Anomali Threat Platform and APP Store, allowing clients immediate access to partner content, integrations and analytics. The partner program expansion includes the launch of a suite of software development kits (SDKs). The SDKs address three critical integration areas: Threat Intelligence Feeds SDK: integrate proprietary threat intelligence feeds and make these accessible via the APP Store Integration SDK: integrate threat intelligence from the Anomali Threat Platform into third party products and solutions Enrichment SDK: integrate 3rd party threat analysis and data enrichments into the Anomali Threat Platform “Organizations need a com

I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.

Besøk vårt presserom