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

Bombardier Will Fully Cooperate with the AMF Review16.11.2018 00:06Pressemelding

MONTRÉAL, Nov. 15, 2018 (GLOBE NEWSWIRE) -- Bombardier acknowledges the announcement by the Autorité des marchés financiers (AMF) that it is reviewing the operations surrounding the establishment by Bombardier of an Automatic Securities Disposition Plan (ASDP) in August 2018 and various subsequent announcements. The ASDP had been reviewed by the AMF prior to its establishment on August 15, 2018. Bombardier intends to fully cooperate with the AMF in its review. The Company has taken the necessary measures to suspend all sales of securities pursuant to the ASDP until further notice. About Bombardier With over 69,500 employees across four business segments, Bombardier is a global leader in the transportation industry, creating innovative and game-changing planes and trains. Our products and services provide world-class transportation experiences that set new standards in passenger comfort, energy efficiency, reliability and safety. Headquartered in Montreal, Canada, Bombardier has product

ERYTECH Partners with New York Blood Center for Red Blood Cell Supply and Research15.11.2018 22:05Pressemelding

LYON, France and CAMBRIDGE, Mass., Nov. 15, 2018 (GLOBE NEWSWIRE) -- ERYTECH Pharma (Euronext: ERYP - Nasdaq: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating drug substances inside red blood cells, today announced a strategic partnership with New York Blood Center (NYBC) for red blood cell supply and research. The partnership encompasses NYBC becoming a long-term supplier of donor red blood cells (RBC) to ERYTECH, enabling ERYTECH to diversify and broaden its supply of RBC source materials for the production of eryaspase and future product candidates derived from its proprietary ERYCAPS® platform as the company ramps up clinical development. Specifically for the Phase 3 trial in second-line metastatic pancreatic cancer that recently started enrolling patients in Europe, and the Phase 2 trial in first-line triple negative breast cancer (TNBC), which are both expected to begin enrollment in the United States early in 2019. The partnershi

Zageno Deepens Engagement with Scientists and Vendors through Its Meaningful and Unbiased Rating System for Scientific Products — Scientific Score gets Algorithm Update15.11.2018 15:37Pressemelding

Call for Contributions: Zageno’s rating system for scientific products — the Scientific Score — adds a crucial dimension to help research scientists purchase the right material for their experiment. Scientists: share your experimental data and professional experience about products; Life Science Vendors: provide additional product-specific resources and data points to make your products reach the right scientist CAMBRIDGE, Mass. USA and BERLIN, Germany, Nov. 15, 2018 (GLOBE NEWSWIRE) -- Zageno Inc., the largest and fastest-growing online marketplace for research materials, today issues an open call for scientists and vendors alike to contribute to the continuous improvement of the Scientific Score. Designed as an unbiased indicator to help purchasing scientists navigate the enormous amount of product information, the Scientific Score is the result of proprietary algorithms calculating and weighing different data points across multiple information formats. Among others, factors like rec

Motiv Ring Expands International Footprint and Delivers Industry-First Sizing Process15.11.2018 15:00Pressemelding

The wearable ring lands in 20 new countries and eight new stores, and introduces frictionless sizing method SAN FRANCISCO, Nov. 15, 2018 (GLOBE NEWSWIRE) -- Motiv Inc., creators of breakthrough wearable technology, today announced an increase of its product availability through new international availability and a new digital sizing set alternative. Motiv Ring’s international expansion will be through its website MyMotiv.com. Featured countries include: North America (Canada), Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Hungary, Italy, Lithuania, Latvia, Netherlands, Poland, Portugal, Spain, Sweden, United Kingdom, Norway and Switzerland), as well as Japan, Taiwan, Turkey, Australia and New Zealand. Good fit is imperative to great functionality. To date, Motiv Ring users select their sizes after receiving a physical sizing kit in the mail. Releasing in Open Beta , Motiv Ring users will now have the additional option of determining their Motiv size via a

SignalFx Delivers Next Generation of Application Performance Monitoring15.11.2018 15:00Pressemelding

The first to enable directed troubleshooting for applications and microservices SAN MATEO, Calif., Nov. 15, 2018 (GLOBE NEWSWIRE) -- SignalFx, the leader in real-time cloud monitoring for infrastructure, microservices, and applications, today unveiled SignalFx Microservices APM™, the industry’s first real-time application performance monitoring solution designed to accelerate troubleshooting for DevOps teams through advanced real-time analytics. Powered by NoSample™ distributed tracing architecture, SignalFx Microservices APM observes every single transaction – not just a small random sample – and reports on every anomaly. With the new Outlier Analyzer™, the most challenging issues can now be pinpointed and resolved with a single click. SignalFx Microservices APM is built on top of SignalFx’s advanced streaming analytics platform for metrics which applies unsurpassed data science in real-time to identify the root cause of critical application problems. Integration with advanced automat

Minerva Neurosciences to Host Roluperidone Update and Key Opinion Leader Discussion of Negative Symptoms in CNS/Psychiatric Diseases on November 20, 201815.11.2018 14:30Pressemelding

Minerva management to provide update on Phase 3 clinical trial with roluperidone and single-dose escalation study findings Additional topics to include review of recent BDNF data with roluperidone and KOL discussion of trans-diagnostic approach to treating negative symptoms in CNS/psychiatry diseases WALTHAM, Mass., Nov. 15, 2018 (GLOBE NEWSWIRE) -- Minerva Neurosciences, Inc. (NASDAQ: NERV), a clinical-stage biopharmaceutical company focused on the development of therapies to treat central nervous system (CNS) disorders, today announced that it will host a roluperidone update and key opinion leader event on Tuesday, November 20, 2018 beginning at 8:00 a.m. Eastern Time in New York. Members of Minerva’s senior management will be joined by an expert key opinion leader. Discussions will feature the following speakers and topics: Gregory P. Strauss, Ph.D., Assistant Professor; Director: Clinical Affective Neuroscience Laboratory; Director: Georgia Psychiatric Risk Evaluation Program; Depa

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

Besøk vårt presserom