Center for Gene Therapy

Porto Alegre, Brazil

Center for Gene Therapy

Porto Alegre, Brazil
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News Article | May 11, 2017
Site: globenewswire.com

CHICAGO, May 11, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported financial results for the first quarter ended March 31, 2017, recent corporate highlights and upcoming milestones. “We are very pleased with our progress during the first quarter and recent weeks, including the recently reported encouraging results from the closeout of the Phase 1 trial of AVXS-101 in SMA Type 1,” said Sean Nolan, President and Chief Executive Officer of AveXis. “Our team is focused on executing our plan to bring AVXS-101 to patients suffering from SMA Type 1 as quickly and safely as possible.” Results from the Phase 1 Trial of AVXS-101 in SMA Type 1: The Phase 1, open-label, dose-escalating study was designed to evaluate the safety and tolerability of AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1. The key measures of efficacy were the time from birth to an “event,” which was defined as either death or at least 16 hours per day of required ventilation support for breathing for 14 consecutive days in the absence of acute reversible illness or perioperatively, and video confirmed achievement of ability to sit unassisted. Additionally, several exploratory objective measures were assessed, including a standard motor milestone development survey and Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND). Presented AVXS-101 research at the Annual Meeting of the American Academy of Neurology: Jerry Mendell, M.D., director of the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, presented results from the Phase 1 study of AVXS-101 in SMA Type 1 during a plenary session, including video evidence of children achieving motor milestones. Completed Type B chemistry manufacturing and controls (CMC) meeting with the U.S. Food and Drug Administration (FDA): On May 1, 2017, AveXis participated in a Type B CMC meeting with the FDA. The purpose of the meeting was to present to the agency AveXis’ proposed process for producing the intended commercial scale GMP-derived gene therapy product, to gain alignment with the agency on the proposed assay qualification plan, and to gain alignment on the proposed protocol for demonstrating comparability of the intended commercial scale GMP-derived product with the material administered to patients in the Phase 1 trial of AVXS-101 in SMA Type 1. The company expects to provide an update on its further plans and development timelines following receipt of the minutes of the CMC Type B meeting, currently anticipated in early June 2017. Joao Siffert Appointed to Board of Directors: On April 19, 2017, AveXis announced the appointment of Joao Siffert to its Board of Directors, effective upon the completion of the annual meeting of stockholders. Dr. Siffert brings important knowledge to the Board based on his experience in central nervous system drug development and regulatory expertise in both the U.S. and Europe, as well as his experience working with global health care companies. Conference Call Information The AveXis conference call and webcast of April 25, 2017 was conducted in lieu of a first quarter 2017 financial and operating results conference call. AveXis will not host a conference call and webcast related to its first quarter 2017 financial and operating results. The Company expects to host its next conference call and webcast following receipt of the minutes from the CMC meeting with the FDA, expected approximately 30 days following the meeting, which took place May 1, 2017. About SMA SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. SMA is caused by a genetic defect in the SMN1 gene that codes SMN, a protein necessary for survival of motor neurons. The incidence of SMA is approximately one in 10,000 live births. The most severe form of SMA is Type 1, a lethal genetic disorder characterized by motor neuron loss and associated muscle deterioration, which results in mortality or the need for permanent ventilation support before the age of two for greater than 90 percent of patients. SMA Type 1 is the leading genetic cause of infant mortality. About AVXS-101 AVXS-101 is a proprietary gene therapy candidate of a one-time treatment for SMA Type 1 and is designed to address the monogenic root cause of SMA and prevent further muscle degeneration by addressing the defective and/or loss of the primary SMN gene. AVXS-101 also targets motor neurons providing rapid onset of effect, and crosses the blood brain barrier allowing an IV dosing route and effective targeting of both central and systemic features. About AveXis, Inc. AveXis is a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases. The company’s initial proprietary gene therapy candidate, AVXS-101, is in an ongoing Phase 1 clinical trial for the treatment of SMA Type 1. For additional information, please visit www.avexis.com. Forward-Looking Statements This press release contains "forward-looking statements," within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things, AveXis’ research, development and regulatory plans for AVXS-101, including the potential of AVXS-101 to positively impact quality of life and alter the course of disease in children with SMA Type 1 and statements about the effects of SMA Type 1 on developmental milestones and timing of regulatory feedback. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual results to differ materially from those projected in its forward-looking statements. Meaningful factors which could cause actual results to differ include, but are not limited to, the scope, progress, expansion, and costs of developing and commercializing AveXis’ product candidates; regulatory developments in the U.S. and EU, as well as other factors discussed in the "Risk Factors" and the "Management's Discussion and Analysis of Financial Condition and Results of Operations" section of AveXis’ Annual Report on Form 10-K for the year ended December 31, 2016, filed with the SEC on March 16, 2017. In addition to the risks described above and in the Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the SEC, other unknown or unpredictable factors also could affect AveXis’ results. There can be no assurance that the actual results or developments anticipated by AveXis will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, AveXis. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein. AveXis cautions investors not to rely too heavily on the forward-looking statements AveXis makes or that are made on its behalf. These forward-looking statements speak only as of the date of this press release (unless another date is indicated). AveXis undertakes no obligation, and specifically declines any obligation, to publicly update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Non-GAAP Financial Measure In addition to disclosing financial results that are determined in accordance with GAAP, in order to understand and evaluate our operating performance, and provide a more complete understanding of factors and trends affecting our business, we also measure the increase in research and development expenses and general and administrative expenses excluding non-cash stock-based compensation expense.  We believe that excluding this expense better reflects the increase in research and development and general and administrative expenses during the period, as compared to the prior period.  This non-GAAP financial metric should be considered supplemental to and not a substitute for financial information prepared in accordance with GAAP. Because non-GAAP financial metrics exclude the effect of items that will increase or decrease the company’s reported results of operations, we strongly encourage investors to review our consolidated financial statements and periodic reports in their entirety.


--Positive dose response in central nervous system with 60.7% +/- 8.8% reduction of disease-causing heparan sulfate GAG observed in Cohort 2 --Reduction of disease manifestation observed in decreased liver volume of 14.81% (+/- 1.2%) --ABO-102 well-tolerated in six subjects through 1100 days follow up with no Serious Adverse Events --Cohort 1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at six months --Conference call today at 10:00am EDT; 877-269-7756 for domestic callers and 201-689-7817 for international callers NEW YORK and CLEVELAND, May 12, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. 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 Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. “Abeona continues to advance gene therapy for MPS IIIA patients and we are excited about the positive dose response in the CNS seen in Cohort 2. The observation of a dose response supports our clinical approach, and we are encouraged to observe further reductions in central nervous system (CNS) heparan sulfate, reduction in liver volume, and preliminary evidence of slowed neurocognitive decline, are very encouraging. We look forward to accelerating enrollment with the recently initiated global sites (Spain and Australia) and reporting additional clinical data in the ABO-102 global MPS IIIA trial later this year,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. 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. Highlights reported data on five (n=3 Cohort 1, n=2 Cohort 2) out of the six patients treated to date in the gene therapy trial included: Biopotency: positive dose response observed in Cohort 2. --At 30 days post-injection, two patients in the Cohort 2 demonstrated 60.7%(+/- 8.8%) reduction in cerebral spinal fluid (CSF) heparan sulfate (HS). Hepatosplenomegaly: consistent reduction in liver volume observed. --At 30 days post-injection, Cohort 2 subjects demonstrated reductions in liver volumes of 14.81% (+/- 1.2%). --The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver volumes averaging 116% at baseline that did not change over a year of follow-up. Cognitive Assessments: evidence of cognitive stabilization at six months in Cohort 1. --Cognitive assessments, taken at baseline, at the six-month timepoint for the Cohort 1 (n=3), subjects showed evidence of stabilization or improvement in the Leiter-R non-verbal IQ and Vineland (adaptive behavior) scales. --Cognitive assessments are taken at six-month and twelve-month follow-up visits. --Leiter Nonverbal IQ assessments in Cohort 1 subjects demonstrated stabilized or improved scores at six-months post-injection. Notably, one subject improved +10 (+/-6) points, while age-matched controls in the Natural History study would have predicted a decrease of -11.1 (+/-2.7) points over 6 months. --Vineland assessments in Cohort 1 at six months post-injection suggest stabilization in adaptive behavior scores. Safety: well-tolerated in all subjects through 1100 days cumulative post-injection. --No serious adverse events (SAEs) reported in subjects in either cohort receiving ABO-102 (Cohort 1: 5E12 vg/kg and Cohort 2: 1E13 vg/kg). “We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 and in the initial two subjects of Cohort 2,” stated Kevin M. Flanigan, M.D., principal investigator, Director of the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “We are pleased to see decreases in CSF HS compared to the Cohort 1 at 30 days post-injection, and we look forward to enrolling additional high-dose patients.” The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the FDA, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA.  The global clinical study is supported by a 25-subject MPS III 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. Company Conference Call Details: Abeona will host a live conference call briefing today at 10:00am EDT. Analysts and investors can participate in the conference call by dialing 877-269-7756 for domestic callers and 201-689-7817 for international callers. 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), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), 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 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 www.abeonatherapeutics.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 include without limitation the statement that the addition of two additional global clinical site will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA.  Such 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 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; 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 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 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.


--Positive dose response in central nervous system with 60.7% +/- 8.8% reduction of disease-causing heparan sulfate GAG observed in Cohort 2 --Reduction of disease manifestation observed in decreased liver volume of 14.81% (+/- 1.2%) --ABO-102 well-tolerated in six subjects through 1100 days follow up with no Serious Adverse Events --Cohort 1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at six months --Conference call today at 10:00am EDT; 877-269-7756 for domestic callers and 201-689-7817 for international callers NEW YORK and CLEVELAND, May 12, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. 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 Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. “Abeona continues to advance gene therapy for MPS IIIA patients and we are excited about the positive dose response in the CNS seen in Cohort 2. The observation of a dose response supports our clinical approach, and we are encouraged to observe further reductions in central nervous system (CNS) heparan sulfate, reduction in liver volume, and preliminary evidence of slowed neurocognitive decline, are very encouraging. We look forward to accelerating enrollment with the recently initiated global sites (Spain and Australia) and reporting additional clinical data in the ABO-102 global MPS IIIA trial later this year,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. 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. Highlights reported data on five (n=3 Cohort 1, n=2 Cohort 2) out of the six patients treated to date in the gene therapy trial included: Biopotency: positive dose response observed in Cohort 2. --At 30 days post-injection, two patients in the Cohort 2 demonstrated 60.7%(+/- 8.8%) reduction in cerebral spinal fluid (CSF) heparan sulfate (HS). Hepatosplenomegaly: consistent reduction in liver volume observed. --At 30 days post-injection, Cohort 2 subjects demonstrated reductions in liver volumes of 14.81% (+/- 1.2%). --The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver volumes averaging 116% at baseline that did not change over a year of follow-up. Cognitive Assessments: evidence of cognitive stabilization at six months in Cohort 1. --Cognitive assessments, taken at baseline, at the six-month timepoint for the Cohort 1 (n=3), subjects showed evidence of stabilization or improvement in the Leiter-R non-verbal IQ and Vineland (adaptive behavior) scales. --Cognitive assessments are taken at six-month and twelve-month follow-up visits. --Leiter Nonverbal IQ assessments in Cohort 1 subjects demonstrated stabilized or improved scores at six-months post-injection. Notably, one subject improved +10 (+/-6) points, while age-matched controls in the Natural History study would have predicted a decrease of -11.1 (+/-2.7) points over 6 months. --Vineland assessments in Cohort 1 at six months post-injection suggest stabilization in adaptive behavior scores. Safety: well-tolerated in all subjects through 1100 days cumulative post-injection. --No serious adverse events (SAEs) reported in subjects in either cohort receiving ABO-102 (Cohort 1: 5E12 vg/kg and Cohort 2: 1E13 vg/kg). “We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 and in the initial two subjects of Cohort 2,” stated Kevin M. Flanigan, M.D., principal investigator, Director of the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “We are pleased to see decreases in CSF HS compared to the Cohort 1 at 30 days post-injection, and we look forward to enrolling additional high-dose patients.” The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the FDA, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA.  The global clinical study is supported by a 25-subject MPS III 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. Company Conference Call Details: Abeona will host a live conference call briefing today at 10:00am EDT. Analysts and investors can participate in the conference call by dialing 877-269-7756 for domestic callers and 201-689-7817 for international callers. 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), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), 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 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 www.abeonatherapeutics.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 include without limitation the statement that the addition of two additional global clinical site will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA.  Such 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 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; 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 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 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.


--Positive dose response in central nervous system with 60.7% +/- 8.8% reduction of disease-causing heparan sulfate GAG observed in Cohort 2 --Reduction of disease manifestation observed in decreased liver volume of 14.81% (+/- 1.2%) --ABO-102 well-tolerated in six subjects through 1100 days follow up with no Serious Adverse Events --Cohort 1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at six months --Conference call today at 10:00am EDT; 877-269-7756 for domestic callers and 201-689-7817 for international callers NEW YORK and CLEVELAND, May 12, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. 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 Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. “Abeona continues to advance gene therapy for MPS IIIA patients and we are excited about the positive dose response in the CNS seen in Cohort 2. The observation of a dose response supports our clinical approach, and we are encouraged to observe further reductions in central nervous system (CNS) heparan sulfate, reduction in liver volume, and preliminary evidence of slowed neurocognitive decline, are very encouraging. We look forward to accelerating enrollment with the recently initiated global sites (Spain and Australia) and reporting additional clinical data in the ABO-102 global MPS IIIA trial later this year,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. 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. Highlights reported data on five (n=3 Cohort 1, n=2 Cohort 2) out of the six patients treated to date in the gene therapy trial included: Biopotency: positive dose response observed in Cohort 2. --At 30 days post-injection, two patients in the Cohort 2 demonstrated 60.7%(+/- 8.8%) reduction in cerebral spinal fluid (CSF) heparan sulfate (HS). Hepatosplenomegaly: consistent reduction in liver volume observed. --At 30 days post-injection, Cohort 2 subjects demonstrated reductions in liver volumes of 14.81% (+/- 1.2%). --The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver volumes averaging 116% at baseline that did not change over a year of follow-up. Cognitive Assessments: evidence of cognitive stabilization at six months in Cohort 1. --Cognitive assessments, taken at baseline, at the six-month timepoint for the Cohort 1 (n=3), subjects showed evidence of stabilization or improvement in the Leiter-R non-verbal IQ and Vineland (adaptive behavior) scales. --Cognitive assessments are taken at six-month and twelve-month follow-up visits. --Leiter Nonverbal IQ assessments in Cohort 1 subjects demonstrated stabilized or improved scores at six-months post-injection. Notably, one subject improved +10 (+/-6) points, while age-matched controls in the Natural History study would have predicted a decrease of -11.1 (+/-2.7) points over 6 months. --Vineland assessments in Cohort 1 at six months post-injection suggest stabilization in adaptive behavior scores. Safety: well-tolerated in all subjects through 1100 days cumulative post-injection. --No serious adverse events (SAEs) reported in subjects in either cohort receiving ABO-102 (Cohort 1: 5E12 vg/kg and Cohort 2: 1E13 vg/kg). “We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 and in the initial two subjects of Cohort 2,” stated Kevin M. Flanigan, M.D., principal investigator, Director of the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “We are pleased to see decreases in CSF HS compared to the Cohort 1 at 30 days post-injection, and we look forward to enrolling additional high-dose patients.” The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the FDA, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA.  The global clinical study is supported by a 25-subject MPS III 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. Company Conference Call Details: Abeona will host a live conference call briefing today at 10:00am EDT. Analysts and investors can participate in the conference call by dialing 877-269-7756 for domestic callers and 201-689-7817 for international callers. 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), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), 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 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 www.abeonatherapeutics.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 include without limitation the statement that the addition of two additional global clinical site will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA.  Such 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 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; 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 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 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.


--Positive dose response in central nervous system with 60.7% +/- 8.8% reduction of disease-causing heparan sulfate GAG observed in Cohort 2 --Reduction of disease manifestation observed in decreased liver volume of 14.81% (+/- 1.2%) --ABO-102 well-tolerated in six subjects through 1100 days follow up with no Serious Adverse Events --Cohort 1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at six months --Conference call today at 10:00am EDT; 877-269-7756 for domestic callers and 201-689-7817 for international callers NEW YORK and CLEVELAND, May 12, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. 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 Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. “Abeona continues to advance gene therapy for MPS IIIA patients and we are excited about the positive dose response in the CNS seen in Cohort 2. The observation of a dose response supports our clinical approach, and we are encouraged to observe further reductions in central nervous system (CNS) heparan sulfate, reduction in liver volume, and preliminary evidence of slowed neurocognitive decline, are very encouraging. We look forward to accelerating enrollment with the recently initiated global sites (Spain and Australia) and reporting additional clinical data in the ABO-102 global MPS IIIA trial later this year,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. 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. Highlights reported data on five (n=3 Cohort 1, n=2 Cohort 2) out of the six patients treated to date in the gene therapy trial included: Biopotency: positive dose response observed in Cohort 2. --At 30 days post-injection, two patients in the Cohort 2 demonstrated 60.7%(+/- 8.8%) reduction in cerebral spinal fluid (CSF) heparan sulfate (HS). Hepatosplenomegaly: consistent reduction in liver volume observed. --At 30 days post-injection, Cohort 2 subjects demonstrated reductions in liver volumes of 14.81% (+/- 1.2%). --The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver volumes averaging 116% at baseline that did not change over a year of follow-up. Cognitive Assessments: evidence of cognitive stabilization at six months in Cohort 1. --Cognitive assessments, taken at baseline, at the six-month timepoint for the Cohort 1 (n=3), subjects showed evidence of stabilization or improvement in the Leiter-R non-verbal IQ and Vineland (adaptive behavior) scales. --Cognitive assessments are taken at six-month and twelve-month follow-up visits. --Leiter Nonverbal IQ assessments in Cohort 1 subjects demonstrated stabilized or improved scores at six-months post-injection. Notably, one subject improved +10 (+/-6) points, while age-matched controls in the Natural History study would have predicted a decrease of -11.1 (+/-2.7) points over 6 months. --Vineland assessments in Cohort 1 at six months post-injection suggest stabilization in adaptive behavior scores. Safety: well-tolerated in all subjects through 1100 days cumulative post-injection. --No serious adverse events (SAEs) reported in subjects in either cohort receiving ABO-102 (Cohort 1: 5E12 vg/kg and Cohort 2: 1E13 vg/kg). “We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 and in the initial two subjects of Cohort 2,” stated Kevin M. Flanigan, M.D., principal investigator, Director of the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “We are pleased to see decreases in CSF HS compared to the Cohort 1 at 30 days post-injection, and we look forward to enrolling additional high-dose patients.” The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the FDA, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA.  The global clinical study is supported by a 25-subject MPS III 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. Company Conference Call Details: Abeona will host a live conference call briefing today at 10:00am EDT. Analysts and investors can participate in the conference call by dialing 877-269-7756 for domestic callers and 201-689-7817 for international callers. 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), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), 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 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 www.abeonatherapeutics.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 include without limitation the statement that the addition of two additional global clinical site will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA.  Such 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 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; 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 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 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.


--Positive dose response in central nervous system with 60.7% +/- 8.8% reduction of disease-causing heparan sulfate GAG observed in Cohort 2 --Reduction of disease manifestation observed in decreased liver volume of 14.81% (+/- 1.2%) --ABO-102 well-tolerated in six subjects through 1100 days follow up with no Serious Adverse Events --Cohort 1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at six months --Conference call today at 10:00am EDT; 877-269-7756 for domestic callers and 201-689-7817 for international callers NEW YORK and CLEVELAND, May 12, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. 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 Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. “Abeona continues to advance gene therapy for MPS IIIA patients and we are excited about the positive dose response in the CNS seen in Cohort 2. The observation of a dose response supports our clinical approach, and we are encouraged to observe further reductions in central nervous system (CNS) heparan sulfate, reduction in liver volume, and preliminary evidence of slowed neurocognitive decline, are very encouraging. We look forward to accelerating enrollment with the recently initiated global sites (Spain and Australia) and reporting additional clinical data in the ABO-102 global MPS IIIA trial later this year,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. 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. Highlights reported data on five (n=3 Cohort 1, n=2 Cohort 2) out of the six patients treated to date in the gene therapy trial included: Biopotency: positive dose response observed in Cohort 2. --At 30 days post-injection, two patients in the Cohort 2 demonstrated 60.7%(+/- 8.8%) reduction in cerebral spinal fluid (CSF) heparan sulfate (HS). Hepatosplenomegaly: consistent reduction in liver volume observed. --At 30 days post-injection, Cohort 2 subjects demonstrated reductions in liver volumes of 14.81% (+/- 1.2%). --The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver volumes averaging 116% at baseline that did not change over a year of follow-up. Cognitive Assessments: evidence of cognitive stabilization at six months in Cohort 1. --Cognitive assessments, taken at baseline, at the six-month timepoint for the Cohort 1 (n=3), subjects showed evidence of stabilization or improvement in the Leiter-R non-verbal IQ and Vineland (adaptive behavior) scales. --Cognitive assessments are taken at six-month and twelve-month follow-up visits. --Leiter Nonverbal IQ assessments in Cohort 1 subjects demonstrated stabilized or improved scores at six-months post-injection. Notably, one subject improved +10 (+/-6) points, while age-matched controls in the Natural History study would have predicted a decrease of -11.1 (+/-2.7) points over 6 months. --Vineland assessments in Cohort 1 at six months post-injection suggest stabilization in adaptive behavior scores. Safety: well-tolerated in all subjects through 1100 days cumulative post-injection. --No serious adverse events (SAEs) reported in subjects in either cohort receiving ABO-102 (Cohort 1: 5E12 vg/kg and Cohort 2: 1E13 vg/kg). “We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 and in the initial two subjects of Cohort 2,” stated Kevin M. Flanigan, M.D., principal investigator, Director of the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “We are pleased to see decreases in CSF HS compared to the Cohort 1 at 30 days post-injection, and we look forward to enrolling additional high-dose patients.” The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the FDA, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA.  The global clinical study is supported by a 25-subject MPS III 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. Company Conference Call Details: Abeona will host a live conference call briefing today at 10:00am EDT. Analysts and investors can participate in the conference call by dialing 877-269-7756 for domestic callers and 201-689-7817 for international callers. 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), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), 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 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 www.abeonatherapeutics.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 include without limitation the statement that the addition of two additional global clinical site will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA.  Such 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 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; 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 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 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.


--Positive dose response in central nervous system with 60.7% +/- 8.8% reduction of disease-causing heparan sulfate GAG observed in Cohort 2 --Reduction of disease manifestation observed in decreased liver volume of 14.81% (+/- 1.2%) --ABO-102 well-tolerated in six subjects through 1100 days follow up with no Serious Adverse Events --Cohort 1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at six months --Conference call today at 10:00am EDT; 877-269-7756 for domestic callers and 201-689-7817 for international callers NEW YORK and CLEVELAND, May 12, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. 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 Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. “Abeona continues to advance gene therapy for MPS IIIA patients and we are excited about the positive dose response in the CNS seen in Cohort 2. The observation of a dose response supports our clinical approach, and we are encouraged to observe further reductions in central nervous system (CNS) heparan sulfate, reduction in liver volume, and preliminary evidence of slowed neurocognitive decline, are very encouraging. We look forward to accelerating enrollment with the recently initiated global sites (Spain and Australia) and reporting additional clinical data in the ABO-102 global MPS IIIA trial later this year,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. 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. Highlights reported data on five (n=3 Cohort 1, n=2 Cohort 2) out of the six patients treated to date in the gene therapy trial included: Biopotency: positive dose response observed in Cohort 2. --At 30 days post-injection, two patients in the Cohort 2 demonstrated 60.7%(+/- 8.8%) reduction in cerebral spinal fluid (CSF) heparan sulfate (HS). Hepatosplenomegaly: consistent reduction in liver volume observed. --At 30 days post-injection, Cohort 2 subjects demonstrated reductions in liver volumes of 14.81% (+/- 1.2%). --The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver volumes averaging 116% at baseline that did not change over a year of follow-up. Cognitive Assessments: evidence of cognitive stabilization at six months in Cohort 1. --Cognitive assessments, taken at baseline, at the six-month timepoint for the Cohort 1 (n=3), subjects showed evidence of stabilization or improvement in the Leiter-R non-verbal IQ and Vineland (adaptive behavior) scales. --Cognitive assessments are taken at six-month and twelve-month follow-up visits. --Leiter Nonverbal IQ assessments in Cohort 1 subjects demonstrated stabilized or improved scores at six-months post-injection. Notably, one subject improved +10 (+/-6) points, while age-matched controls in the Natural History study would have predicted a decrease of -11.1 (+/-2.7) points over 6 months. --Vineland assessments in Cohort 1 at six months post-injection suggest stabilization in adaptive behavior scores. Safety: well-tolerated in all subjects through 1100 days cumulative post-injection. --No serious adverse events (SAEs) reported in subjects in either cohort receiving ABO-102 (Cohort 1: 5E12 vg/kg and Cohort 2: 1E13 vg/kg). “We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 and in the initial two subjects of Cohort 2,” stated Kevin M. Flanigan, M.D., principal investigator, Director of the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “We are pleased to see decreases in CSF HS compared to the Cohort 1 at 30 days post-injection, and we look forward to enrolling additional high-dose patients.” The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the FDA, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA.  The global clinical study is supported by a 25-subject MPS III 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. Company Conference Call Details: Abeona will host a live conference call briefing today at 10:00am EDT. Analysts and investors can participate in the conference call by dialing 877-269-7756 for domestic callers and 201-689-7817 for international callers. 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), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), 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 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 www.abeonatherapeutics.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 include without limitation the statement that the addition of two additional global clinical site will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA.  Such 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 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; 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 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 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.


PubMed | Center for Gene Therapy, Beckman Research Institute and Clinical Trial Office.
Type: Journal Article | Journal: Blood | Year: 2016

Attenuated poxvirus modified vaccinia Ankara (MVA) is a useful viral-based vaccine for clinical investigation, because of its excellent safety profile and property of inducing potent immune responses against recombinant (r) antigens. We developed Triplex by constructing an rMVA encoding 3 immunodominant cytomegalovirus (CMV) antigens, which stimulates a host antiviral response: UL83 (pp65), UL123 (IE1-exon4), and UL122 (IE2-exon5). We completed the first clinical evaluation of the Triplex vaccine in 24 healthy adults, with or without immunity to CMV and vaccinia virus (previous DryVax smallpox vaccination). Three escalating dose levels (DL) were administered IM in 8 subjects/DL, with an identical booster injection 28 days later and 1-year follow-up. Vaccinations at all DL were safe with no dose-limiting toxicities. No vaccine-related serious adverse events were documented. Local and systemic reactogenicity was transient and self-limiting. Robust, functional, and durable Triplex-driven expansions of CMV-specific T cells were detected by measuring T-cell surface levels of 4-1BB (CD137), binding to CMV-specific HLA multimers, and interferon- production. Marked and durable CMV-specific T-cell responses were also detected in Triplex-vaccinated CMV-seronegatives, and in DryVax-vaccinated subjects. Long-lived memory effector phenotype, associated with viral control during CMV primary infection, was predominantly found on the membrane of CMV-specific and functional T cells, whereas off-target vaccine responses activating memory T cells from the related herpesvirus Epstein-Barr virus remained undetectable. Combined safety and immunogenicity results of MVA in allogeneic hematopoietic stem cell transplant (HCT) recipients and Triplex in healthy adults motivated the initiation of a placebo-controlled multicenter trial of Triplex in HCT patients. This trial was registered at www.clinicaltrials.gov as #NCT02506933.

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