Abeona Therapeutics Receives Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten Disease
NEW YORK and CLEVELAND, April 20, 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 European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation (EMA/OD/013/18) for Abeona's gene therapy program ABO-202 for the treatment of subjects with neuronal ceroid lipofuscinosis, also known as Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children. ABO-202 is an adeno-associated virus developed to introduce a functional copy of the CLN1 gene into cells in order to restore the enzyme activity that is needed to break down certain lipopigment proteins that are deficient in patients with CLN1 disease.
"This designation is an important step in our CLN1 program as it encourages us to proceed towards clinical development," stated Carsten Thiel, Ph.D, CEO of Abeona Therapeutics Inc. "Batten disease, particularly CLN1 disease, is a severely debilitating disorder where patients do not currently have adequate therapeutic options. Compelling pre-clinical data underscore the viability of ABO-202 as a potential treatment for CLN1 disease in humans. We remain committed to our vision of developing transformational gene therapies for patients suffering from devastating rare diseases."
ABO-202, developed with Steven J. Gray, Ph.D, Associate Professor, Department of Pediatrics, UT Southwestern and the support of Garrett the Grand Batten Fighter, The Saoirse Foundation, Taylor's Tale, Hayden's Batten Disease Foundation, and the Batten Disease Support and Research Association, is anticipated to enter clinical trials in 2018.
"ABO-202 is an AAV gene therapy that has shown promising preclinical efficacy utilizing multiple administration routes to ameliorate CNS burden in the CLN1 animal model of disease. ABO-202 improved survival, muscle function and behavior after intrathecal, intravenous and importantly, combination dosing with both routes of administration in CLN1 animals," stated Steven J. Gray, Ph.D.
ABO-202 has been granted Orphan Drug and Rare Pediatric Disease Designations from the US Food and Drug Administration (FDA). Preclinical data from the program were recently presented at the WORLDSymposium held in San Diego and an update will be presented at the American Society for Gene and Cell Therapy (ASGCT) later this year. Key findings reported included:
- The data demonstrate that a single intravenous, single intrathecal (IT) or combination therapy utilizing both administration routes of a self-complementary adeno-associated virus encoding the human CLN1 gene to CLN1 mice significantly increased their survival, improved behavior and reduced motor deficits.
- Higher doses further improved these observations, suggesting that methods increasing total CNS exposure may be beneficial and provided some survival and behavioral benefit to symptomatic mice.
- A combination approach delivering ABO-202 by both intravenous and intrathecal routes of administration further increased survival efficacy and improved potential treatment options for older animals with advanced disease manifestations.
About CLN1 Disease (infantile and late infantile onset): Infantile and late infantile neuronal ceroid lipofuscinosis is a severe lysosomal disease caused by mutations in the CLN1 gene, which encodes the soluble lysosomal enzyme Palmitoyl-Protein-Thioesterase-1 (PPT1) and result in osmiophilic granules accumulating in lysosomes and leading to neuroinflammation, neurodegeneration and death. CLN1 disease is neurodegenerative, manifests shortly after birth, and is fatal in its classic form by 6 to 12 years of age. In the classic form, aggressive clinical features appear, including rapid speech and motor deterioration, refractory epilepsy, ataxia, myoclonus, and visual failure between the ages of 6 and 24 months. By 5 years of age, CLN1 disease patients with the classic infantile form are typically poorly responsive and are no longer communicative. These patients subsequently die in the following few years.
About European Union (EU) Orphan Drug Designation: The European Commission grants orphan drug designation status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the European Union. The orphan drug designation provides Abeona with incentives and benefits in the EU, including protocol assistance, reduced fees and protection from market competition once ABO-202 is approved for patients with CLN1 disease.
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 CLN3 disease, ABO-202 (AAV-CLN1) for treatment of CLN1 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(TM), for next generation product candidates. For more information, visit www.abeonatherapeutics.com.
SVP, Finance & Investor Relations
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 are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, EMA orphan drug designation for ABO-202 provides Abeona certain benefits and incentives, including marketing exclusivity, that are strategically important from a regulatory and commercial perspective, our preclinical work for ABO-202 and the recently published data supporting its clinical translation for patients with CLN1 disease demonstrated the importance of selecting the right vector and delivery route to target tissues in the CNS and treat the symptoms associated with the underlying disease pathology, we look forward to advancing the ABO-202 program, 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; 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
Om 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
Must Read Whitepaper, ‘Making Money With Network Slicing’, Hits The Shelves23.10.2018 10:00 | Pressemelding
Network Slicing Leader Cloudstreet Showcases the Vast Opportunities of this Promising New Mobile Business Model Espoo, Finland & Berlin, Germany, Oct. 23, 2018 (GLOBE NEWSWIRE) -- Cloudstreet, ‘The Network Slicing Company’, is pleased to announce the publication of its most recent industry whitepaper, “Making Money with Network Slicing”. A business-forward take on the practice, the paper puts slicing at the very center of the mobile industry’s transition now underway. With a focus on practical, market-ready models, the authors unravel the misconceptions about Network Slicing, exploring its potential to disrupt the cultural and business logjams that have hampered growth. Less about technology than a new way of doing business, Network Slicing has quickly risen from a mere buzzword to a budget line item, as mobile operators retool for the opportunities to come “Inherently disruptive, it will create new business ecosystems to support unique and evolving demands that touch quite literally e
Skybox Security Receives Common Criteria Certification23.10.2018 09:00 | Pressemelding
Receiving the internationally recognized certification will help organizations and particularly federal agencies assess the security and assurance of Skybox solutions SAN JOSE, Calif., Oct. 23, 2018 (GLOBE NEWSWIRE) -- Skybox® Security, a global leader in cybersecurity management, has received Common Criteria for Information Technology Security Evaluation (Common Criteria or CC) certification for Skybox Security Suite. Skybox is honored to receive the high-standard certification, demonstrating our commitment to top-tier products and delivering premium solutions to our customers. “The Common Criteria certification is an important milestone for the Skybox product,” said Skybox Security Vice President of R&D and CTO Ron Davidson. “It also enables us to provide cybersecurity solutions in the federal space, which not only requires the CC stamp of approval, but also has an acute need for the attack surface visibility and situational intelligence Skybox delivers.” The Skybox Security Suite en
UCB and Biogen Announce Topline Results from a Phase 2b Study of Dapirolizumab Pegol in Systemic Lupus Erythematosus23.10.2018 07:00 | Pressemelding
BRUSSELS, Belgium and CAMBRIDGE, Mass., Oct. 23, 2018 (GLOBE NEWSWIRE) -- Today UCB (Euronext Brussels: UCB) and Biogen Inc. (Nasdaq: BIIB) announced top-line results from a Phase 2b study evaluating the safety and efficacy of dapirolizumab pegol (DZP), an anti-CD40L pegylated Fab, in adults with moderately-to-severely active systemic lupus erythematosus (SLE) despite receiving standard-of-care treatment such as corticosteroids, anti-malarials and non-biological immunosuppressants. The primary endpoint of the study to demonstrate a dose response at 24 weeks on the British Isles Lupus Assessment Group (BILAG)-based Composite Lupus Assessment (BICLA) was not met (p=0.06). The study did demonstrate consistent and potentially meaningful improvements for the majority of clinical endpoints in patients treated with DZP compared with placebo. In addition, biomarker data demonstrated evidence of proof of biology. DZP was well tolerated and demonstrated an acceptable safety profile. Biogen and U
Constellation Brands Prices Offering of Senior Notes22.10.2018 23:52 | Pressemelding
VICTOR, N.Y., Oct. 22, 2018 (GLOBE NEWSWIRE) -- Constellation Brands, Inc. (NYSE: STZ and STZ.B), a leading beverage alcohol company, announced today that it priced the public offering of $2.15 billion aggregate principal amount of Senior Notes, consisting of (i) $650.0 million of Senior Floating Rate Notes due 2021 (the “senior floating rate notes”); (ii) $500.0 million of 4.400% Senior Notes due 2025 (the “2025 notes”) for a public offering price of 99.812% of the principal amount of the 2025 notes; (iii) $500.0 million of 4.650% Senior Notes due 2028 (the “2028 notes”) for a public offering price of 99.720% of the principal amount of the 2028 notes; and (iv) $500.0 million of 5.250% Senior Notes due 2048 (the “2048 notes”) for a public offering price of 99.592% of the principal amount of the 2048 notes (collectively, the “notes”). The notes will be senior obligations that rank equally with all of Constellation’s other senior unsecured indebtedness, and will be guaranteed by the subs
Forterra Announces Third Quarter 2018 Earnings Schedule22.10.2018 22:30 | Pressemelding
IRVING, Texas, Oct. 22, 2018 (GLOBE NEWSWIRE) -- Forterra, Inc. (“Forterra”) (Nasdaq: FRTA) plans to release third quarter 2018 financial results before the market opens on Tuesday, November 6, 2018. A conference call to review financial results will also be held on Tuesday, November 6, 2018 at 10:00 a.m. Eastern time (9:00 a.m. Central). Hosting the call will be Jeff Bradley, Chief Executive Officer, and Charlie Brown, Executive Vice President and Chief Financial Officer. CONFERENCE CALL AND WEBCAST DETAILS Webcast Information Event: Q3 2018 Forterra, Inc. Earnings Call Date: Tuesday, November 6, 2018 Time: 10:00 a.m. Eastern Time (9:00 a.m. Central) https://edge.media-server.com/m6/p/6dinjr8x Conference Call Information U.S.: 1-574-990-1396 Toll-Free: 1-844-498-0572 Participant Passcode: 2467118 Replay Information A replay of the conference call will be available within 24 hours after the call on the “Events & Presentations” page under the Investor section of the Company’s website at
Ondot Announces Investment from Citi Ventures to Power Enriched Transactions22.10.2018 17:00 | Pressemelding
The investment will fuel Ondot’s international growth and give cardholders accessing info via Ondot’s platform increased control over and visibility into each transaction Santa Clara, Ca, Oct. 22, 2018 (GLOBE NEWSWIRE) -- Ondot Systems, the global leader in mobile payments services, today announced that it has secured a strategic funding investment from Citi Ventures. Ondot works directly with financial institutions to give consumers increased control over and visibility into their personal payment transactions. With Ondot, consumers can manage functions such as fraud alerts, transaction limits and purchase location restrictions, all from the convenience of an app on their smartphone or desktop. This investment will further support Ondot’s efforts to grow its international presence and product offerings. Today’s digitally connected consumers are looking for more ways to access detailed information about their digital lives — including personal payment transactions. Ondot’s platform add
I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.Besøk vårt presserom