The Therapeutic Goods Administration is the regulatory body for therapeutic goods in Australia. It is a Division of the Australian Department of Health established under the Therapeutic Goods Act 1989 . The TGA is responsible for conducting assessment and monitoring activities to ensure that therapeutic goods available in Australia are of an acceptable standard and that access to therapeutic advances is in a timely manner. Wikipedia.


Time filter

Source Type

NEW YORK and CLEVELAND, May 09, 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, today announced Australian regulatory approval to initiate a Phase 1/2 for the ABO-102 gene therapy program for patients with Sanfilippo syndrome type A (MPS IIIA).  The clinical study was approved by the Australian Government Department of Health Therapeutic Goods Administration and the Company is conducting the Phase 1/2 clinical study at Adelaide Women’s and Children’s Hospital, Australia. "Abeona has enrolled more MPS IIIA patients in a gene therapy trial than any other group in the world, and the addition of a third clinical site will accelerate our ability to evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA. We remain encouraged by the recently reported clinical data and look forward to enrolling patients in Australia and Spain shortly. Our work in Australia would not have been possible without The Sanfilippo Children’s Foundation and families for helping advance these potentially life-changing therapies into global clinical trials," stated Timothy J. Miller, Ph.D., President & CEO. “MPS IIIA is a profound degenerative disorder of childhood, which manifests progressive neuromotor and cognitive decline with associated systemic complications and premature death, typically before adulthood. The reported clinical data has proved most encouraging with a reduction in central nervous system (CNS) heparan sulphate, a reduction in liver volume, and preliminary evidence of slowed neurocognitive decline,” indicated Nicholas Smith, M.D., Ph.D., Principal Investigator and Dept. Head, Neurology and Pediatric Neurologist at Women’s and Children’s Hospital. “With no approved treatments for this relentless disease, this investigational gene therapy approach, delivered as a single intravenous injection to treat the whole body, holds great potential for patients and their families.” 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.  Per the design of the trial, subjects receive 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, particularly the CNS. Subjects are evaluated at multiple time points over the initial six-months post-injection for safety assessments and initial signals of biopotency. 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. “Abeona continues to demonstrate global leadership in advancing clinical and commercial development of promising gene therapies for children with Sanfilippo syndrome, and the additional clinical site in Australia will help advance global access to this potential treatment for families suffering from this devastating disease,” said Megan Donnell, Executive Director of The Sanfilippo Children’s Foundation. 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 a third global clinical site will accelerate our ability to 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.


NEW YORK and CLEVELAND, May 09, 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, today announced Australian regulatory approval to initiate a Phase 1/2 for the ABO-102 gene therapy program for patients with Sanfilippo syndrome type A (MPS IIIA).  The clinical study was approved by the Australian Government Department of Health Therapeutic Goods Administration and the Company is conducting the Phase 1/2 clinical study at Adelaide Women’s and Children’s Hospital, Australia. "Abeona has enrolled more MPS IIIA patients in a gene therapy trial than any other group in the world, and the addition of a third clinical site will accelerate our ability to evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA. We remain encouraged by the recently reported clinical data and look forward to enrolling patients in Australia and Spain shortly. Our work in Australia would not have been possible without The Sanfilippo Children’s Foundation and families for helping advance these potentially life-changing therapies into global clinical trials," stated Timothy J. Miller, Ph.D., President & CEO. “MPS IIIA is a profound degenerative disorder of childhood, which manifests progressive neuromotor and cognitive decline with associated systemic complications and premature death, typically before adulthood. The reported clinical data has proved most encouraging with a reduction in central nervous system (CNS) heparan sulphate, a reduction in liver volume, and preliminary evidence of slowed neurocognitive decline,” indicated Nicholas Smith, M.D., Ph.D., Principal Investigator and Dept. Head, Neurology and Pediatric Neurologist at Women’s and Children’s Hospital. “With no approved treatments for this relentless disease, this investigational gene therapy approach, delivered as a single intravenous injection to treat the whole body, holds great potential for patients and their families.” 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.  Per the design of the trial, subjects receive 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, particularly the CNS. Subjects are evaluated at multiple time points over the initial six-months post-injection for safety assessments and initial signals of biopotency. 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. “Abeona continues to demonstrate global leadership in advancing clinical and commercial development of promising gene therapies for children with Sanfilippo syndrome, and the additional clinical site in Australia will help advance global access to this potential treatment for families suffering from this devastating disease,” said Megan Donnell, Executive Director of The Sanfilippo Children’s Foundation. 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 a third global clinical site will accelerate our ability to 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.


NEW YORK and CLEVELAND, May 09, 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, today announced Australian regulatory approval to initiate a Phase 1/2 for the ABO-102 gene therapy program for patients with Sanfilippo syndrome type A (MPS IIIA).  The clinical study was approved by the Australian Government Department of Health Therapeutic Goods Administration and the Company is conducting the Phase 1/2 clinical study at Adelaide Women’s and Children’s Hospital, Australia. "Abeona has enrolled more MPS IIIA patients in a gene therapy trial than any other group in the world, and the addition of a third clinical site will accelerate our ability to evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA. We remain encouraged by the recently reported clinical data and look forward to enrolling patients in Australia and Spain shortly. Our work in Australia would not have been possible without The Sanfilippo Children’s Foundation and families for helping advance these potentially life-changing therapies into global clinical trials," stated Timothy J. Miller, Ph.D., President & CEO. “MPS IIIA is a profound degenerative disorder of childhood, which manifests progressive neuromotor and cognitive decline with associated systemic complications and premature death, typically before adulthood. The reported clinical data has proved most encouraging with a reduction in central nervous system (CNS) heparan sulphate, a reduction in liver volume, and preliminary evidence of slowed neurocognitive decline,” indicated Nicholas Smith, M.D., Ph.D., Principal Investigator and Dept. Head, Neurology and Pediatric Neurologist at Women’s and Children’s Hospital. “With no approved treatments for this relentless disease, this investigational gene therapy approach, delivered as a single intravenous injection to treat the whole body, holds great potential for patients and their families.” 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.  Per the design of the trial, subjects receive 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, particularly the CNS. Subjects are evaluated at multiple time points over the initial six-months post-injection for safety assessments and initial signals of biopotency. 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. “Abeona continues to demonstrate global leadership in advancing clinical and commercial development of promising gene therapies for children with Sanfilippo syndrome, and the additional clinical site in Australia will help advance global access to this potential treatment for families suffering from this devastating disease,” said Megan Donnell, Executive Director of The Sanfilippo Children’s Foundation. 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 a third global clinical site will accelerate our ability to 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.


NEW YORK and CLEVELAND, May 09, 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, today announced Australian regulatory approval to initiate a Phase 1/2 for the ABO-102 gene therapy program for patients with Sanfilippo syndrome type A (MPS IIIA).  The clinical study was approved by the Australian Government Department of Health Therapeutic Goods Administration and the Company is conducting the Phase 1/2 clinical study at Adelaide Women’s and Children’s Hospital, Australia. "Abeona has enrolled more MPS IIIA patients in a gene therapy trial than any other group in the world, and the addition of a third clinical site will accelerate our ability to evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA. We remain encouraged by the recently reported clinical data and look forward to enrolling patients in Australia and Spain shortly. Our work in Australia would not have been possible without The Sanfilippo Children’s Foundation and families for helping advance these potentially life-changing therapies into global clinical trials," stated Timothy J. Miller, Ph.D., President & CEO. “MPS IIIA is a profound degenerative disorder of childhood, which manifests progressive neuromotor and cognitive decline with associated systemic complications and premature death, typically before adulthood. The reported clinical data has proved most encouraging with a reduction in central nervous system (CNS) heparan sulphate, a reduction in liver volume, and preliminary evidence of slowed neurocognitive decline,” indicated Nicholas Smith, M.D., Ph.D., Principal Investigator and Dept. Head, Neurology and Pediatric Neurologist at Women’s and Children’s Hospital. “With no approved treatments for this relentless disease, this investigational gene therapy approach, delivered as a single intravenous injection to treat the whole body, holds great potential for patients and their families.” 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.  Per the design of the trial, subjects receive 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, particularly the CNS. Subjects are evaluated at multiple time points over the initial six-months post-injection for safety assessments and initial signals of biopotency. 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. “Abeona continues to demonstrate global leadership in advancing clinical and commercial development of promising gene therapies for children with Sanfilippo syndrome, and the additional clinical site in Australia will help advance global access to this potential treatment for families suffering from this devastating disease,” said Megan Donnell, Executive Director of The Sanfilippo Children’s Foundation. 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 a third global clinical site will accelerate our ability to 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.


NEW YORK and CLEVELAND, May 09, 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, today announced Australian regulatory approval to initiate a Phase 1/2 for the ABO-102 gene therapy program for patients with Sanfilippo syndrome type A (MPS IIIA).  The clinical study was approved by the Australian Government Department of Health Therapeutic Goods Administration and the Company is conducting the Phase 1/2 clinical study at Adelaide Women’s and Children’s Hospital, Australia. "Abeona has enrolled more MPS IIIA patients in a gene therapy trial than any other group in the world, and the addition of a third clinical site will accelerate our ability to evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA. We remain encouraged by the recently reported clinical data and look forward to enrolling patients in Australia and Spain shortly. Our work in Australia would not have been possible without The Sanfilippo Children’s Foundation and families for helping advance these potentially life-changing therapies into global clinical trials," stated Timothy J. Miller, Ph.D., President & CEO. “MPS IIIA is a profound degenerative disorder of childhood, which manifests progressive neuromotor and cognitive decline with associated systemic complications and premature death, typically before adulthood. The reported clinical data has proved most encouraging with a reduction in central nervous system (CNS) heparan sulphate, a reduction in liver volume, and preliminary evidence of slowed neurocognitive decline,” indicated Nicholas Smith, M.D., Ph.D., Principal Investigator and Dept. Head, Neurology and Pediatric Neurologist at Women’s and Children’s Hospital. “With no approved treatments for this relentless disease, this investigational gene therapy approach, delivered as a single intravenous injection to treat the whole body, holds great potential for patients and their families.” 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.  Per the design of the trial, subjects receive 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, particularly the CNS. Subjects are evaluated at multiple time points over the initial six-months post-injection for safety assessments and initial signals of biopotency. 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. “Abeona continues to demonstrate global leadership in advancing clinical and commercial development of promising gene therapies for children with Sanfilippo syndrome, and the additional clinical site in Australia will help advance global access to this potential treatment for families suffering from this devastating disease,” said Megan Donnell, Executive Director of The Sanfilippo Children’s Foundation. 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 a third global clinical site will accelerate our ability to 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.


News Article | May 18, 2017
Site: www.prweb.com

Dr. Ralph Mobbs of the Neuro Spine Clinic completed the procedure on April 28, 2017 at the Prince Of Wales Private Hospital. The procedure was performed on a 46-year-old male patient suffering from a degenerative cervical disc at level C6-C7. The patient failed conservative treatments prior to undergoing surgery. The AxioMed viscoelastic disc is a next-generation disc replacement that restores natural disc height, lordosis, stability and motion in the human spine. AxioMed was approved in 2016 to market and sell their viscoelastic cervical and lumbar Freedom total disc replacements in Australia by the Therapeutic Goods Administration. Dr. Ralph Mobbs spoke to the advantages of the AxioMed cervical disc after the operation, stating, "The Freedom Cervical Disc is a new and innovative treatment that I've been eager to use. I found the prosthesis matched the patient's anatomy well, restored disc height and fit very well within the intervertebral space. The disc was easily placed with AxioMed’s simple and effective instrumentation. I believe in this viscoelastic technology and its benefits and I look forward to using the Freedom Cervical Disc to treat my patients in the future." AxioMed CEO Dr. Kingsley R. Chin explained how an experience like Dr. Mobbs’ aligns with AxioMed’s vision. “AxioMed believes it can replicate the success of total joint restoration in the spine with our innovative and advanced viscoelastic total disc replacements with a high degree of patient satisfaction," he said. Dr. Chin added, “With the addition of the lateral lumbar technique, we expect AxioMed to be the worldwide leader in disc replacement surgery.” Dr. Mobbs currently practices neurosurgery with a special interest in minimally invasive and complex spine surgery. About AxioMed Founded in 2001, AxioMed began its journey of exhaustively proving the Freedom® Disc through clinical studies in the USA and Europe, research, development and testing. In 2014, KICVentures recognized the disc’s enormous potential and acquired the company into their healthcare portfolio. AxioMed owns an exclusive viscoelastic material license on its proprietary Freedom Disc technology.


DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of the "Australia Market Report for Female Urinary Incontinence Slings 2017 - MedCore" report to their offering. The female urinary incontinence sling market includes both synthetic slings and non-synthetic slings. Autologous tissue and allogenic grafts are also available options for slings; however, these treatments are not the focus of this report. The volatility in the market is attributed to ongoing scrutiny by healthcare regulatory agencies such as the FDA in the United States and adverse reactions documented by the Australian Government Department of Health TGA (Therapeutic Goods Administration). The controversy surrounding urinary incontinence slings predominantly concerns synthetic slings. Due to the negative side effects publicized about slings, however, procedures using both synthetic and biological slings have decreased. The adverse side-effects have resulted in patients and, to a lesser degree, physicians, becoming more cautious about sling procedures. While the repercussions of this controversy will still extend throughout the forecast period, as a result surgeons are giving greater attention to stronger patient consent protocols including full disclaimers of the risks involved. The negative publicity regarding slings has had the greatest impact in countries without public healthcare. In Australia, there has been a faster response to treat patients with adverse effects with no additional costs for the patient. This has led to fewer lawsuits and complaints regarding the procedure helping to keep the market stable. Despite the contraction of the urinary incontinence sling market, the number of procedures is still substantial. The number of patients suffering from female urinary incontinence is increasing. The number of patients is also expected to continue increasing throughout the reporting period, as urinary incontinence affects older demographics. For more information about this report visit http://www.researchandmarkets.com/research/97vmbp/australia_market


News Article | February 15, 2017
Site: www.newscientist.com

Australia has become the latest country to ban over-the-counter sales of medications containing the opioid painkiller codeine. The new regulation, which will come into effect on 1 February 2018, is hoped to halt the rise in codeine-related deaths, which have more than doubled in Australia since 2000. Codeine is used to treat pain and suppress coughing, and can currently be purchased in Australia and the UK without a prescription; low doses are found in some painkillers, cough syrups, and cold and flu tablets. However, even in small doses, codeine has the potential to become addictive. Inside the body, it is partially metabolised to morphine, which can produce feelings of relaxation and euphoria. As a result, some people increase their use of codeine-containing medications. The US, Germany and Japan have already banned over-the-counter sales of the drug. Since banning such sales, these countries have seen a fall in codeine-related deaths, says Michael Vagg, a pain specialist in Geelong, Australia. “But that’s not even the most compelling argument for doing this,” he says. “It’s also a pretty rubbish drug that doesn’t actually help people as much as they think it does.” In its decision to make codeine prescription-only, Australia’s Therapeutic Goods Administration noted that codeine doesn’t provide effective pain relief at the low doses used in over-the-counter medications. Long-term codeine use has even been shown to have a pain-sensitising effect in people who take it regularly for headaches, ultimately making their pain worse. When people start taking high doses, codeine can cause severe breathing difficulties. But there is another harmful effect for people who become addicted. Over-the-counter drugs that contain codeine also often contain other drugs like paracetamol (acetaminophen) or ibuprofen, and as a person increases their use of these, they can experience liver, gut or kidney damage. A recent study found that paracetamol or ibuprofen was involved in 55 per cent of the 1200 codeine-related deaths recorded in Australia between 2000 and 2013. Every hospital in Australia will tell you stories of addiction to such combined drugs, says Vagg. “You often see it in young women. Some have to be tube-fed because their guts are so damaged,” he says. “Others have to go on dialysis because their kidneys are wrecked.” However, George Tambassis, president of the Pharmacy Guild of Australia, says that making codeine prescription-only will unfairly affect people who genuinely need it and use it safely. The issue of misuse would be better managed by a real-time monitoring system that identified people at risk of misusing codeine at the point of sale, he says. In April 2015, the UK’s Medicines and Healthcare products Regulatory Agency recommended that children under 12 shouldn’t be given over-the-counter codeine. However, no plans have been announced yet to make codeine prescription-only in the UK.


- Toujeo® provides a flatter profile than insulin degludec with less within-day fluctuations - Sanofi presented today the full results of a pharmacokinetic / pharmacodynamic (PK/PD) study with Toujeo® (insulin glargine 300 Units/mL) and insulin degludec 100 Units/mL (Deg-100) at the 16th Annual Diabetes Technology Meeting, Bethesda, Maryland, U.S.[1] In patients with type 1 diabetes, the Toujeo® PK/PD profile at a clinically relevant dose (0.4 U/kg/day, the average dose used in worldwide clinical practice)[2] was flatter and more evenly distributed over 24 hours compared with the profile of insulin degludec. The within-day fluctuation of metabolic activity was 20% lower (p=0.047) for Toujeo® than insulin degludec. An overall flat PK/PD profile and an evenly distributed exposure over 24 hours were observed for Toujeo® at both dose levels studied (0.4 and 0.6 U/kg/day). "These new findings may have clinical implications for people with type 1 diabetes on basal insulin therapy," said Dr. Timothy Bailey, Clinical Associate Professor, University of California, San Diego. "Toujeo® has already shown an improved PK/PD profile when compared to insulin glargine 100 Units/mL (Gla-100), with a flatter profile. Also in a previous CGM study[3] in adults with type 1 diabetes versus Gla-100, Toujeo showed less between-day variability and less within-day fluctuations, associated with a lower risk of nocturnal confirmed ( 54 mg/dL) or severe hypoglycemia in the first 8 weeks.[3] The results observed in the current PK/PD study comparing Toujeo® versus Deg-100 confirm that Toujeo has an interesting profile with the potential to help the people with diabetes to reach their targets with less risk of hypoglycemia." "In this PK/PD study, we observed a more favorable profile for Toujeo® compared to insulin degludec," said Riccardo Perfetti, Head of Global Diabetes Medical Team, Sanofi. "The clinical implications of these findings are currently being investigated." Although basal insulin injections cannot perfectly replicate endogenous insulin production, the 'ideal' basal insulin for diabetes therapy would have stable pharmacodynamic (PD) and pharmacokinetic (PK) profiles. Smaller and less variable glucose excursions may result in more predictable 24hour profiles and less hypoglycemia, and could translate into an improved experience for people on basal insulin and better adherence to therapy. This was a randomized, double-blind, 2x2, crossover, euglycemic-clamp study in two parallel cohorts of people with type 1 diabetes (48 people in total) to compare the pharmacodynamic and pharmacokinetic properties of 0.4 and 0.6 U/kg/day insulin glargine 300 Units/mL (Toujeo®, Gla-300) with the same dose levels of insulin degludec 100 Units/mL (Deg-100) in steady state after 8 days multiple dosing regimen. Within-day fluctuation of the smoothed glucose infusion rate (GIR-smFL0-24) was significantly lower with Gla-300 versus Deg-100 at the 0.4 U/kg dose level. At the 0.4 U/kg dose level, relative 6-hour fractions of GIR-AUC0-24 were more evenly distributed with Gla-300 versus Deg-100. GIR-smFL0-24 and 6-hour fractions of GIR-AUC0-24 were similar between insulins at the supra-therapeutic 0.6 U/kg dose level. At both dose levels, GIR-AUC0-24 and GIRmax were lower for Gla-300 versus Deg-100, while T50%-GIR-AUC0-24 and duration of BG control were similar for each insulin. Both dose levels of Gla-300 provided plateau-like insulin profiles up to 16 hours post-dose, followed by a subsequent slow decline. For Deg-100, insulin increased at both dose levels from the time of injection until ~10 hours after dosing, with subsequent slow decline. Exposure of both insulins was measurable until clamp end (30 hours). At both dose levels the 6-hour fractions of INS-AUC0-24 were more evenly distributed with Gla-300 versus Deg-100. Continuous glucose monitoring (CGM), is a valuable way to confirm whether the differences observed in the PK and PD properties of insulins under experimental condition translate to clinically relevant differences in their 24-hour glucose profiles and other parameters of glycemic control, including hypoglycemia. This was a multicenter, 16-week, open-label, Phase II, parallel-group, two-period crossover study including 59 people with type 1 diabetes (PDY12777; NCT01658579). The primary endpoint was the mean percentage of time within the glucose range of 4.4-7.8 mmol/L (80-140 mg/dL) during the last 2 weeks of treatment in each treatment period (Weeks 7-8 and Weeks 15-16). The main secondary endpoints were the average 24-hour glucose profiles based on CGM (mmol/L) for the last 2 weeks of treatment in each treatment period and the incidence of participants experiencing at least one hypoglycemic event (defined by American Diabetes Association criteria) in either study period. The percentage of time within the glucose range of 4.4-7.8 mmol/L (primary endpoint) was similar for participants receiving Gla-300 vs Gla-100 (LS mean difference 0.75%, p=0.73). Average 24-hour glucose profiles showed a more constant glucose level with Gla-300 vs. Gla-100. Toujeo® is a once-daily basal insulin based on a broadly-used molecule (insulin glargine). Toujeo has been approved by the U.S. Food and Drug Administration (FDA), the European Commission, Health Canada, the Therapeutic Goods Administration in Australia, and the MHLW in Japan (where its approved brand name is Lantus® XR), and is under review by other regulatory authorities around the world. Sanofi, a global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi is organized into five global business units: Diabetes and Cardiovascular, General Medicines and Emerging Markets, Sanofi Genzyme, Sanofi Pasteur and Merial.


News Article | October 28, 2016
Site: www.prweb.com

Catalent Pharma Solutions, the leading global provider of advanced delivery technologies and development solutions for drugs, biologics and consumer health products, today announced that it is to provide fill-finish production services for the commercial supply of SB4, a biosimilar referencing Enbrel® [1] (etanercept), on behalf of the Korea-based biopharmaceutical company Samsung Bioepis Co., Ltd. The services will be provided at Catalent’s flagship sterile pre-filled syringe facility in Brussels, Belgium. Used in the treatment of adults with moderate to severe rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis and plaque psoriasis, SB4 is the first etanercept biosimilar to receive regulatory approval by the European Commission (EC), as Benepali®. [2] SB4 has also received regulatory approvals from Korea’s Ministry of Food and Drug Safety (MFDS), Australia’s Therapeutic Goods Administration (TGA), and Canada’s Health Canada, as BRENZYS™. [3,4] “We are proud to have partnered with Samsung Bioepis on the launch of this biosimilar therapy, which was the first etanercept biosimilar to receive European Commission approval, and look forward to a continued partnership as we continue to commercial supply,” commented Jonathan Arnold, Vice President & General Manager, Drug Delivery Solutions at Catalent. Catalent’s 265,000 square foot Brussels facility offers the latest in innovative pre-filled syringe fill-finish processing and associated packaging. With experts in technology transfer, scale-up and life-cycle management, its annual syringe filling capacity is more than 200 million units. 1 Enbrel is a registered trademark of Immunex Corp. 2 Benepali® is a registered European trademark of Biogen Idec International Holding Ltd. 3 BRENZYS™ has not been approved for the treatment of patients with psoriatic arthritis and plaque psoriasis in Canada 4 BRENZYS™ is a trademark of Merck Sharp & Dohme Corp. About Samsung Bioepis Established in 2012, Samsung Bioepis is a biopharmaceutical company committed to realizing healthcare that is accessible to everyone. Through innovations in product development and a firm commitment to quality, Samsung Bioepis aims to become the world's leading biopharmaceutical company. Samsung Bioepis continues to advance a broad pipeline of 13 biosimilar candidates that include six first-wave candidates that cover the therapeutic areas of immunology, oncology and diabetes. Samsung Bioepis is a joint venture between Samsung BioLogics and Biogen. For more information, please visit: http://www.samsungbioepis.com About Catalent Catalent is the leading global provider of advanced delivery technologies and development solutions for drugs, biologics and consumer health products. With over 80 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable clinical and commercial product supply. Catalent employs approximately 9,200 people, including over 1,400 scientists, at more than 30 facilities across five continents, and in fiscal 2016 generated $1.85 billion in annual revenue. Catalent is headquartered in Somerset, New Jersey. For more information, visit http://www.catalent.com

Loading Therapeutic Goods Administration collaborators
Loading Therapeutic Goods Administration collaborators