Nasdaq GlobeNewswire

Abeona Therapeutics Provides Update from ABO-102 Phase 1/2 MPS IIIA Clinical Trial at the 13th Annual WORLDSymposium™ 2017


NEW YORK and CLEVELAND, 2017-02-17 16:04 CET (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq:ABEO):

  • ABO-102 gene therapy well-tolerated in 4 subjects (N=3 low dose, N=1 high dose) through 650 days follow up with no Serious Adverse Events
  • 63% +/- 0.5% central nervous system reduction of heparan sulfate GAG 6 months post-injection (N=2)
  • Continued evidence of biopotency including reduced liver and spleen volumes and decreased urinary GAGs
  • Two subjects assessed at the 6-month timepoint showed evidence for stabilization or improvement (average 60% over 2 subjects) in several Mullen subdomains
  • Adaptive behavior ratings on the Vineland stabilized
  • Subjects showed improved ability to complete individual items on the Leiter-R non-verbal IQ assessment resulting in improved raw scores

Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, announced updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA), at the 13th Annual WORLDSymposium™ 2017 lysosomal storage disorders conference in San Diego, CA. The ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH) is a first-in-man clinical trial utilizing a single intravenous injection of AAV gene therapy for subjects with MPS IIIA, a rare autosomal recessive disease affecting every cell and organ in the body, which results in neurocognitive decline, speech loss, loss of mobility, and premature death in children.

“We remain encouraged by continued signs of tolerability and biopotency in the low-dose cohort, and enrollment of the high-dose cohort is underway,” stated Kevin M. Flanigan, M.D., principal investigator with the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “Additionally, we are pleased to see further decreases in CSF GAG measurements, as well as preliminary evidence for stabilization or improvement of some cognitive functions, at six months post-dosing.”

Per the design of the clinical trial, subjects received a single, intravenous injection of ABO-102 to deliver the AAV viral vector systematically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease. Subjects are evaluated at multiple time points post-injection for safety assessments and initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system. Observations reported at the WORLDSymposium™ conference included:

  • Safety:  ABO-102 is well-tolerated in subjects injected with the low dose of 5E13 vg/kg ABO-102, with no treatment related adverse events or serious adverse events (SAEs) through over 650 days cumulative post-injection. Enrollment in the high dose cohort has commenced with no Serious Adverse Events (SAEs) reported to date.
  • Biopotency:  As reflected in published natural history studies evaluating MPS III subjects, cerebral spinal fluid (CSF) and urine GAG (heparan sulfate or “HS”) are significantly elevated in the subject population as a symptom of disease pathology. As announced previously, all subjects in the low-dose cohort experienced reductions from baseline in CSF HS of 25.6% +/- 0.8%, suggesting ABO-102 crossed the blood brain barrier after intravenous administration.  At the six-month follow-up (n=2), CSF HS continued to decrease to 63.1% +/- 0.5% of baseline values, suggesting further improvement in the elimination of the storage pathology. Data presented showed reduction in urinary heparan sulfate and urinary total GAG fragments.
  • Hepatosplenomegaly:  The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver and spleen volumes averaging 116% and 88%, respectively at baseline that did not change over a year of follow-up. All three subjects demonstrated significant reductions in liver volumes at 30-days post injection (17.1% +/- 1.9%). At the six-month follow-up in low dose subjects (n=2), this effect was sustained, with a liver volume further decreased by 29.7 – 30.3% and spleen volume by 2.2 – 12.9% from baseline.
  • Cognitive Assessments:  The clinical trial utilizes three validated neurocognitive and behavioral assessment tools, including the Leiter International Performance Scale Third Edition (Leiter-3), the Vineland Adaptive Behavior Scale, Second Edition (Vineland-II) and the Mullen Scale of Early Learning. Cognitive assessments are taken at baseline, and have been taken at the six-month (n=2) and will be taken at the twelve-month follow-up visits. These assessments provide the opportunity to measure several sub-domains, such as fine motor, visual acuity, expressive language, receptive language, among others. Assessments at six-month for the first two lose-dose patients provided early evidence of cognitive stabilization. The two subjects assessed at the 6-month timepoint showed evidence for stabilization or improvement of scores (average of 60% across 2 subjects) in several Mullen subdomains. Adaptive behavior ratings on the Vineland also stabilized.  Both subjects showed improved ability to complete individual items on the Leiter-R non-verbal IQ assessment resulting in improved raw scores.

“The data demonstrate an early and robust systemic delivery of ABO-102, and the increased reductions in CNS HS GAG support our approach for intravenous ABO-102 delivery for subjects with Sanfilippo syndromes,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. “We are excited about continued biomarker signals in this trial, as well as early positive signals in the neurocognitive assessments. While we are still very early in the trial, we are extremely encouraged by these early results and look forward to expanding enrollment in this clinical trial with enrollments accelerating at two additional international clinical sites.”

Abeona’s MPS IIIA program, ABO-102, has been granted Orphan Product Designation in the USA and in the European Union, has received the Rare Pediatric Disease Designation in the US, and recently received Fast Track designation by the US FDA.

Sanfilippo syndromes (or mucopolysaccharidosis (MPS) type III): a group of four inherited genetic diseases each caused by a single gene defect, described as type A, B, C or D, which cause enzyme deficiencies that result in the abnormal accumulation of glycosaminoglycans (GAGs, or sugars) in body tissues. MPS III is a lysosomal storage disease, a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. The incidence of MPS III (all four types combined) is estimated to be 1 in 70,000 births. Mucopolysaccharides are long chains of sugar molecule used in the building of connective tissues in the body. There is a continuous process in the body of replacing used materials and breaking them down for disposal. Children with MPS III are missing an enzyme which is essential in breaking down the used mucopolysaccharides called heparan sulfate. The partially broken down mucopolysaccharides remain stored in cells in the body causing progressive damage. In MPS III, the predominant symptoms occur due to accumulation within the central nervous system (CNS), including the brain and spinal cord, resulting in cognitive decline, motor dysfunction, and eventual death. Importantly, there is no cure for MPS III and treatments are largely supportive care.

About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome (MPS IIIA and IIIB, respectively). Abeona is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), EB-201 for epidermolysis bullosa (EB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) gene therapy for treatment of infantile Batten disease (INCL), and 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 has a plasma-based protein therapy pipeline, including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit

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 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 impact of changes in the financial markets and global economic conditions; our belief that initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system; our belief that the data demonstrate an early and robust systemic delivery of ABO-102, and the increased reductions in CNS GAG support our approach for intravenous delivery for subjects with Sanfilippo syndromes, 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.

         Investor Contact:
         Christine Silverstein
         Vice President, Investor Relations
         Abeona Therapeutics Inc.
         +1 (212)-786-6212
         Media Contact:
         Andre'a Lucca
         Vice President, Communications & Operations
         Abeona Therapeutics Inc.
         +1 (212)-786-6208

Om Nasdaq GlobeNewswire

Nasdaq GlobeNewswire
Nasdaq GlobeNewswire
One Liberty Plaza - 165 Broadway
NY 10006 New York

+1 212 401 8700

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

Abeona Therapeutics Reports Fourth Quarter 2017 Financial Results and Business Highlights16.3.2018 22:32Pressemelding

Investor Conference Call on Tuesday, March 27th at 10:00 am ET NEW YORK and CLEVELAND, March 16, 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 financial results for the fourth quarter. The Company will host a call to update investors on recent clinical developments and year-end financial results on Tuesday, March 27th at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-407-9210 (toll free domestic) or 201-689-8049 (International). "The past year was marked by several defining events in the company's history, having advanced our two lead clinical programs, EB-101 in Epidermolysis Bullosa and ABO-102 in MPS IIIA, and initiated our third clinical program, ABO-101 in MPS IIIB. The strong safety and biopotency data observed in our three active clinical trials and the s

Mandela Golden Hands Collection Sells for US$10 Million at PDAC 2018 Conference16.3.2018 15:40Pressemelding

Arbitrade, a new coin and cryptocurrency exchange, to purchase the unique gold collection with Bitcoin NEW YORK, March 16, 2018 (GLOBE NEWSWIRE) -- The Board of Arbitrade, a new coin and cryptocurrency exchange, has announced it will purchase the Nelson Mandela Golden Hands Collection. Made from 20 lbs, 99.999 of pure gold, it includes 3 life size impressions of Mandela's hands and two others of his palm and fist. It was cast in 2002 by South Africa's Harmony Gold mining group, one of the world's leading gold producers, 12 years after Nelson Mandela was released from prison. The seller, Malcolm Duncan, a South African businessman now living in Calgary, Canada, knew Mandela. He said that Harmony's intention was to make one full set of gold artefacts consisting of a fist, a full hand and a palm impression of his right hand for each of the 27 years Mandela had spent behind bars. Duncan had purchased the sets dedicated to 1964 and 1990, marking the year Mandela was incarcerated and the yea

RSK Chooses Decentral's Jaxx Blockchain Platform, Paving Way For Smart Contracts On Bitcoin16.3.2018 14:44Pressemelding

BUENOS AIRES, Argentina, March 16, 2018 (GLOBE NEWSWIRE) -- RSK, the smart contract platform powered by the Bitcoin network, today announced it has launched on Decentral's Jaxx cryptocurrency wallet and multi-token digital platform. This integration paves the way for RSK's open source platform to implement Ethereum-style smart contracts over the Bitcoin network. RSK combines the flexibility of smart contracts with the Bitcoin infrastructure, bringing endless possibilities to build a more flexible and inclusive financial system that will improve the life of billions of people. RSK successfully released its MainNet network, built as a side-chain to the Bitcoin mainnet, in January 2018. It is now presenting the first wallet that will help dApp developers manage their fuel while creating their solutions powered by RSK and the Bitcoin Network. RSK's CEO, Diego Gutierrez Zaldivar said: "We're very happy to partner with Jaxx, a highly-secure and a very easy-to-use wallet that will be the firs

Up to $200 Billion in Illegal Cybercrime Profits Is Laundered Each Year, Comprehensive Research Study Reveals16.3.2018 13:00Pressemelding

Cybercriminals turning to virtual currencies, video game currency and digital payment systems like PayPal to convert illegal revenue into clean cash CUPERTINO, Calif., March 16, 2018 (GLOBE NEWSWIRE) -- Bromium®, Inc., the pioneer and leader in application isolation using virtualization-based security, today announced the findings of an independent, academic study into the macro economics of cybercrime and how cybercriminals launder and 'cash out' the profits of criminal endeavours. The findings are part of a larger nine-month study titled Into the Web of Profit, sponsored by Bromium. The full findings will be presented at the RSA Conference in April by Dr. Mike McGuire, Senior Lecturer in Criminology at Surrey University, England. According to the report, cybercriminal proceeds make up an estimated 8-10 percent of total illegal profits laundered globally; amounting to an estimated $80-$200 billion each year 1. Other key findings include: Virtual currencies have become the primary tool

Technology empowering an increasingly connected SEA for financially inclusive communities16.3.2018 11:41Pressemelding

Global speakers IFC, MAS shared their views at Ant Financial Technology Exploration Conference in Singapore SINGAPORE, March 16, 2018 (GLOBE NEWSWIRE) -- Ant Financial Services Group ("Ant Financial" or "Ant") today wrapped up its participation at Money20/20 Asia in Singapore with the Ant Technology Exploration Conference (ATEC), with guests Giri Jadeja, Global Head of Financial Innovation at International Finance Corporation (IFC) and Sopnendu Mohanty, Chief Fintech Officer at Monetary Authority of Singapore (MAS) sharing their views on financial inclusion alongside Cheng Li, Chief Technology Officer and Chief Operations Officer for global business group at Ant Financial. Giri Jadeja shared his vision as global head of financial innovation at IFC - to reach out to the 2 billion unbanked and underserved population in the world, the majority of whom reside in Asia. The region has in recent years seen an exponential growth of new fintech companies. Giri is optimistic that technological c

Z-Wave Alliance Dominates Building Automation Space at Light + Building 201816.3.2018 11:00Pressemelding

Exhibitors introduce new products and demonstrate interoperability and collaboration in international Z-Wave smart building and IoT ecosystem FRANKFURT, Germany, March 16, 2018 (GLOBE NEWSWIRE) -- Light + Building 2018 - Hall 9.1, Booth E46 - The Z-Wave Alliance, a global membership organization dedicated to advancing the popular Z-Wave wireless smart home protocol, will host the Z-Wave Pavilion at Light + Building from March 18 - 23, 2018. The Alliance is also demonstrating their award-winning installation toolkit to make installation and testing of a Z-Wave smart home mesh network even easier for the pro installer community. The Z-Wave Pavilion will be on display at the show featuring the latest in European home and building automation and connected lighting products. Z-Wave Alliance members will demonstrate smart home lighting, thermostats, switches, air quality monitors and more. Support for the Z-Wave protocol has never been stronger - the Z-Wave Alliance now boasts over 700-membe

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

Besøk vårt presserom