Nasdaq GlobeNewswire

Abeona Therapeutics Receives Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten Disease

Del

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

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 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.




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 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

GridGain Systems Named to Inc. 500 List of America’s Fastest-Growing Private Companies for Second Consecutive Year16.8.2018 09:00Pressemelding

GridGain Ranks 17th among Software Companies, 13th in San Francisco Metro Area FOSTER CITY, Calif., Aug. 16, 2018 (GLOBE NEWSWIRE) -- GridGain Systems, provider of enterprise-grade in-memory computing solutions based on Apache® Ignite™, today announced it has been named to the 37th annual Inc. 500 list, a highly regarded ranking of the nation’s fastest-growing private companies. This is the second year in a row GridGain has been named to the list. GridGain’s rank of 158 on this year’s Inc. 500 list is up from number 187 last year. The company is ranked 17th among software companies, 13th in the San Francisco metro area, and 37th in the state of California. The Inc. 500 represents a unique look at the most successful companies within the American economy’s most dynamic segment – its independent small and midsized businesses. GridGain provides an in-memory computing platform that delivers speed, scale and high availability to data-intensive applications. Built on a memory-centric archite

Perceptyx Named One of America’s Fastest Growing Companies for the 4th Consecutive Year16.8.2018 05:37Pressemelding

INC 5000 award recognizes Perceptyx’s continued innovation and partnership with many of the world’s best companies SAN DIEGO, Aug. 15, 2018 (GLOBE NEWSWIRE) -- For the 4th consecutive year, Perceptyx has made the prestigious INC 5000 list of America’s fastest growing private companies. A recognized leader in people analytics, Perceptyx specializes in facilitating organizational change through the strategic use of employee surveys and management consulting. Longtime partners with many of the world's largest organizations, Perceptyx is uniquely capable of delivering insights across heavily distributed organizations with complex hierarchies in a way that’s as unique as an organization’s culture and brand. “We’re proud to be recognized among America’s fastest growing companies again,” says John Borland, Co-founder and CEO of Perceptyx. “We believe that our clients are the real heroes of their organizations. It has been our honor to help so many of the world’s best companies realize their g

Bombardier Establishes Automatic Securities Disposition Plan15.8.2018 22:48Pressemelding

Not for distribution to U.S. news wire services or public dissemination in the United States Plan permits sales of vested shares earned by certain senior executives in accordance with predetermined instructions Plan reinforces the incentive effect of performance-based compensation by allowing for the sale of shares on the open market over a period of up to two years at prevailing market prices, regardless of any subsequent material non-public information participants may receive Plan participants must satisfy Bombardier’s minimum stock ownership guidelines for senior executives MONTRÉAL, Aug. 15, 2018 (GLOBE NEWSWIRE) -- Bombardier announced today that it has established an automatic securities disposition plan (“ASDP”) in accordance with applicable Canadian provincial securities legislation. The ASDP allows for the exercise and sale of vested securities earned by certain senior executives of Bombardier as part of their overall performance-based compensation. Under Canadian securities

Cisco Reports Fourth Quarter and Fiscal Year 2018 Earnings15.8.2018 22:05Pressemelding

• Q4 Results: Revenue: $12.8 billion ▪ Increase of 6% year over year ▪ Recurring revenue was 32% of total revenue, up 1 point year over year Earnings per Share: GAAP: $0.81; Non-GAAP: $0.70 ▪ Non-GAAP EPS increased 15% year over year • FY 2018 Results: Revenue: $49.3 billion; increase of 3% year over year Earnings per Share: GAAP: $0.02; Non-GAAP: $2.60 ▪ Non-GAAP EPS increased 9% year over year ▪ GAAP results include a $10.4 billion charge related to the enactment of the Tax Cuts and Jobs Acts • Q1 FY 2019 Guidance: Revenue: 5% to 7% growth year over year Earnings per Share: GAAP: $0.69 to $0.74; Non-GAAP: $0.70 to $0.72 SAN JOSE, Calif., Aug. 15, 2018 (GLOBE NEWSWIRE) -- Cisco today reported fourth quarter and fiscal year results for the period ended July 28, 2018. Cisco reported fourth quarter revenue of $12.8 billion, net income on a generally accepted accounting principles (GAAP) basis of $3.8 billion or $0.81 per share, and non-GAAP net income of $3.3 billion or $0.70 per share.

Williams Scotsman Completes ModSpace Acquisition15.8.2018 22:05Pressemelding

BALTIMORE, Aug. 15, 2018 (GLOBE NEWSWIRE) -- WillScot Corporation (Nasdaq: WSC) (“Williams Scotsman”), the leading specialty rental services provider of innovative modular space and portable storage solutions across North America, today announced that it has completed its acquisition of Modular Space Holdings, Inc. (“ModSpace”) for a total purchase price of approximately $1.2 billion. With the addition of ModSpace, Williams Scotsman now manages over 160,000 modular space and portable storage units serving an even broader customer base from over 120 locations across the United States, Canada and Mexico. The acquisition also expands the breadth and depth of its Ready to Work solutions to existing and incremental customers and markets. Brad Soultz, President and Chief Executive Officer of Williams Scotsman, commented, “We are pleased to confirm the completion of this transformational acquisition and would like to thank our collective customers, employees, and stakeholders for their suppor

Algeco Announces Second Quarter 2018 Financial Results Conference Call and Change to Reporting in Euro15.8.2018 18:00Pressemelding

BALTIMORE, Aug. 15, 2018 (GLOBE NEWSWIRE) -- Algeco Investments B.V. (together with its subsidiaries, “Algeco”), the leading global business services provider of modular space, secure portable storage solutions and remote workforce accommodations, today announced that it will hold its second quarter 2018 financial results conference call on Wednesday, August 29, 2018 at 10:00 a.m., Eastern Time. To access the call, please dial (847) 585-4422 or (888) 424-8151 (US toll free) and enter participant PIN code 6638830# approximately ten minutes prior to the start of the call. You will be placed on hold until the event begins. The conference call will also be broadcast over the internet with an accompanying slide presentation. To join the web conference, go to http://web.meetme.net/r.aspx?p=2&a=UTiLPVrenccJZd. Please enter your name, email address and company to join the call. The customer service team can be reached at any time by pressing *0 on your telephone keypad. Prior to the call, the

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

Besøk vårt presserom