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In addition to conservation of neurocognitive advancement with ABO-102 in MPS IIIA, brand-new scientific outcomes of ABO-102 in MPS IIIA and ABO-101 in MPS IIIB continue to reveal dose-dependent and continual decreases in disease-specific biomarkers, representing clear biologic impacts
In addition, ABO-102 and ABO-101 continue to reveal beneficial security profile in continuous research studies
Abeona to host financier webinar on Tuesday, February 16, 2021 at 1:00 p.m. EST
New York City and CLEVELAND, Feb. 12, 2021 (WORLD NEWSWIRE)– Abeona Therapies Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell treatment, today revealed brand-new favorable information from 2 continuous Stage 1/2 scientific trials of the business’s investigational AAV-based gene treatments ABO-102 and ABO-101 in MPS IIIA and MPS IIIB, respectively. The interim information existed in late-breaking platform oral discussions at the 17 th Yearly WORLD Seminar ™. The discussion slides are readily available on the business’s site at www.abeonatherapeutics.com.
Michael Amoroso, Principal Executive and Chief Operating Officer of Abeona, mentioned, “We are delighted to share upgraded favorable effectiveness and security results that continue to recommend ABO-102 has the prospective to be a life-altering treatment alternative for kids with MPS IIIA, an uncommon, incapacitating condition without any accepted treatment that results in progressive neurodevelopmental and physical decrease, and frequently leads to death early in life. We have actually asked for a conference with the FDA later on this quarter to talk about the ABO-102 information and the prospective course towards a Biologics License Application declare ABO-102. In addition, the brand-new arise from the Transpher B research study continue to support ABO-101’s biologic activity in clients with MPS IIIB.”
The upgraded arise from the Transpher A research study assessing ABO-102 in Sanfilippo syndrome type A (MPS IIIA) showed that neurocognitive advancement was maintained within typical variety of a non-afflicted kid for 2.5 years to 3 years (the most recent time point determined) after treatment with ABO-102 (3×10 13 vg/kg) in 3 young clients in the high-dose friend 3. The 3 young clients were treated with ABO-102 at ages 27 months, 19 months, and 12 months and are now at ages varying from 3.5 years to 5+ years, the timepoint at which clients with MPS IIIA have actually currently begun to experience neurocognitive decrease based upon the nature of illness development. Dose-dependent and statistically substantial decreases in cerebrospinal fluid heparan sulfate, representing enzyme activity in the main nerve system (CNS), and liver volume were sustained for 2 years after treatment. ABO-102 has actually been well-tolerated with long-lasting security staying beneficial 24-55 months following treatment. There have actually been no treatment-related extreme negative occasions and no scientifically substantial negative occasions reported.
Kevin Flanigan, M.D., Director, Center for Gene Treatment at AWRI at Nationwide Kid’s and Transpher A research study principal detective, stated, “The outcomes provided today reveal a single intravenous dosage of ABO-102 can assist maintain neurocognitive advancement for approximately 3 years following treatment in young MPS IIIA clients throughout early phases of their illness. The information reveals ABO-102’s capability to provide a practical copy of the disease-causing SGSH gene to cells of the CNS and peripheral organs, as evidenced by the scientific advantages in neurocognition and biophysical steps and enhancements in disease-specific biomarkers.”
Arise From the Transpher B research study assessing ABO-101 in Sanfilippo syndrome type B (MPS IIIB) revealed that treatment with ABO-101 is connected with a dose-dependent and continual enhancement in main nerve system and systemic biomarkers, suggesting the powerful biologic result of ABO-101 in clients with MPS IIIB. ABO-101 has actually been well-tolerated without any infusion associated or early intense responses and no scientifically substantial negative occasions or lab irregularities.
Maria Jose de Castro, M.D., Healthcare Facility Clínico Universitario Santiago de Compostela and Transpher B research study detective, stated, “The arise from the Transpher B research study supply proof of ABO-101’s effect on illness biomarkers and prospective to break down the build-up of glycosaminoglycans that underlie MPS IIIB pathology. We anticipate continued follow-up to evaluate ABO-101’s capacity to maintain neurocognitive advancement in clients with MPS IIIB.”
Financier Webinar on MPS III Gene Treatment Programs
Abeona management together with Dr. Flanigan and Dr. de Castro will host a financier webinar on February 16, 2021 at 1:00 p.m. EST. The live webinar, consisting of audio and discussion slides, will be available at https://investors.abeonatherapeutics.com/events at the time of the conference. To sign up ahead of time for the live webinar, click on this link.
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An archived replay of the webinar will be readily available after the conclusion of the live occasion at https://investors.abeonatherapeutics.com/events.
About the Yearly WORLD Seminar ™
The WORLD Seminar ™ is created for fundamental, translational and scientific scientists, client advocacy groups, clinicians, and all others who have an interest in finding out more about the most recent discoveries connected to lysosomal illness and the scientific examination of these advances. For extra info on the 17 th Yearly WORLD Seminar ™, please go to https://worldsymposia.org/.
About the Transpher A Research Study
The Transpher A Research Study (NCT02716246) is a continuous, two-year, open-label, dose-escalation, Stage 1/2 international scientific trial examining ABO-102 for the treatment of clients with Sanfilippo syndrome type A (MPS IIIA). The research study, likewise called ABT-001, is planned for clients 6 months to 2 years of age, or clients older than 2 years with a cognitive developmental ratio of 60% or above. ABO-102 gene treatment is provided utilizing AAV9 innovation by means of a single-dose intravenous infusion. The research study main endpoints are neurodevelopment and security, with secondary endpoints consisting of habits assessments, lifestyle, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.
About the Transpher B Research Study
The Transpher B Research Study (NCT03315182) is a continuous, two-year, open-label, dose-escalation, Stage 1/2 international scientific trial examining ABO-101 for the treatment of clients with Sanfilippo syndrome type B (MPS IIIB). The research study, likewise called ABT-002, is planned for clients 6 months to 2 years of age, or clients older than 2 years with a cognitive developmental ratio of 60% or above. ABO-101 gene treatment is provided utilizing AAV9 innovation by means of a single-dose intravenous infusion. The research study main endpoints are neurodevelopment and security, with secondary endpoints consisting of habits assessments, lifestyle, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.
About ABO-102
ABO-102 is an unique gene treatment in Stage 1/2 advancement for Sanfilippo syndrome type A (MPS IIIA), an uncommon lysosomal storage illness without any accepted treatment that mainly impacts the main nerve system (CNS). ABO-102 is dosed in a one-time intravenous infusion utilizing a self-complementary AAV9 vector to provide a practical copy of the SGSH gene to cells of the CNS and peripheral organs. The treatment is created to attend to the underlying SGSH enzyme shortage accountable for unusual build-up of glycosaminoglycans in the brain and throughout the body that leads to progressive cell damage and neurodevelopmental and physical decrease. In the U.S., Abeona holds Regenerative Medication Advanced Treatment, Fast Lane, Rare Pediatric Illness, and Orphan Drug classifications for the ABO-102 scientific program. In the EU, the Business holds PRIME and Orphan medical item classifications.
About ABO-101
ABO-101 is an unique gene treatment in Stage 1/2 advancement for Sanfilippo syndrome type B (MPS IIIB), an uncommon lysosomal storage illness without any authorized treatment that mainly impacts the main nerve system (CNS). ABO-101 is dosed in a one-time intravenous infusion utilizing a self-complementary AAV9 vector to provide a practical copy of the NAGLU gene to cells of the CNS and peripheral tissues. The treatment is created to attend to the underlying NAGLU enzyme shortage accountable for unusual build-up of glycosaminoglycans in the brain and throughout the body that leads to progressive cell damage and neurodevelopmental and physical decrease. In the U.S., Abeona hangs on Track and Rare Pediatric Illness classifications for ABO-101 and Orphan Drug classification in both the U.S. and EU.
About Sanfilippo Syndrome Type A (MPS IIIA)
Sanfilippo syndrome type A (MPS IIIA) is an uncommon, deadly lysosomal storage illness without any accepted treatment that mainly impacts the CNS and is identified by fast neurodevelopmental and physical decrease. Kids with MPS IIIA present with progressive language and cognitive decrease and behavioral irregularities. Other signs consist of sleep issues and regular ear infections. In addition, unique facial functions with thick eyebrows or a unibrow, complete lips and extreme body hair for one’s age, and liver/spleen enhancement are likewise present in early youth. MPS IIIA is brought on by hereditary anomalies that result in a shortage in the SGSH enzyme accountable for breaking down glycosaminoglycans, which build up in cells throughout the body leading to fast health decrease connected with the condition.
About Sanfilippo syndrome type B (MPS IIIB)
Sanfilippo syndrome type B (MPS IIIB) is an uncommon and deadly lysosomal storage illness without any authorized treatment that mainly impacts the main nerve system and is identified by fast neurodevelopmental and physical decrease. Kids with MPS IIIB present with progressive language and cognitive decrease and behavioral irregularities. Other signs consist of sleep issues and regular ear infections. In addition, unique indications such as facial functions with thick eyebrows or a unibrow, complete lips and extreme body hair for one’s age and liver/spleen enhancement are likewise present. The underlying reason for MPS IIIB is a shortage in the NAGLU enzyme accountable for breaking down glycosaminoglycans, which build up throughout the body leading to fast decrease connected with the condition.
About Abeona Therapies
Abeona Therapies Inc. is a clinical-stage biopharmaceutical business establishing gene and cell treatments for major illness. Abeona’s scientific programs consist of EB-101, its autologous, gene-corrected cell treatment for recessive dystrophic epidermolysis bullosa in Stage 3 advancement, along with ABO-102 and ABO-101, unique AAV-based gene treatments for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Stage 1/2 advancement. The Business’s portfolio likewise includes AAV-based gene treatments for ophthalmic illness with high unmet medical requirements. Abeona’s unique, next-generation GOAL ™ capsids have actually revealed prospective to enhance tropism profiles for a range of destructive illness. Abeona’s totally practical, gene and cell treatment GMP production center produces EB-101 for the essential Stage 3 VIITAL ™ research study and can scientific and industrial production of AAV-based gene treatments. To find out more, go to www.abeonatherapeutics.com.
Positive Declarations
This news release includes particular declarations that are positive within the significance of Area 27A of the Securities Act of 1933, as changed, and Area 21E of the Securities Exchange Act of 1934, as changed, which include dangers and unpredictabilities. These declarations consist of declarations about the Business checking out all tactical alternatives, consisting of the sale of some or all of its properties or sale of the Business. We have actually tried to recognize positive declarations by such terms as “may,” “will,” “think,” “quote,” “anticipate,” and comparable expressions (along with other words or expressions referencing future occasions, conditions or situations), which make up and are planned to recognize positive declarations. Real outcomes might vary materially from those shown by such positive declarations as an outcome of numerous crucial elements, various dangers and unpredictabilities, consisting of however not restricted to the prospective effects of the COVID-19 pandemic on our company, operations, and monetary condition, the result of the tactical evaluation, continued interest in our unusual illness portfolio, our capability to register clients in scientific trials, the result of any future conferences with the U.S. Fda or other regulative firms, the effect of competitors, the capability to protect licenses for any innovation that might be essential to advertise our items, the capability to attain or get essential regulative approvals, the effect of modifications in the monetary markets and international financial conditions, dangers connected with information analysis and reporting, and other dangers revealed in the Business’s newest Yearly Report on Kind 10-K and subsequent quarterly reports on Kind 10-Q and other regular reports submitted with the Securities and Exchange Commission. The Business carries out no commitment to modify the positive declarations or to upgrade them to show occasions or situations taking place after the date of this news release, whether as an outcome of brand-new info, future advancements or otherwise, other than as needed by the federal securities laws.
Financier and Media Contact: . Greg Gin . VP, Financier Relations and Corporate Communications . Abeona Therapies . +1 (646) 813-4709 . ggin@abeonatherapeutics.com
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