Trial to Validate a Second Generation Viral Vector for Potential Use in Future Clinical Studies in Rare and Orphan Diseases
WAYNE, PA, USA and MISGAV, Israel I June 5, 2014 I Medgenics, Inc. (NYSE MKT:MDGN), the developer of a novel platform technology for the sustained production and delivery of therapeutic proteins in patients using ex-vivo gene therapy and their own tissue for the treatment of rare and orphan diseases, today announced that the first patient has been enrolled in its Phase 1/2 clinical trial of MDGN-201 (EPODURETM).
The aim of the trial is to validate the potential of the Company’s BioPump(TM) platform using a second generation vector, which was developed to enhance durability of the proposed therapeutic effect. The study will evaluate the potential of the updated platform to offer sustained production and delivery of erythropoietin (EPO) to treat anemia in dialysis patients with end-stage renal disease (ESRD) or chronic kidney disease (CKD).
“The initiation of this trial is a testament to the capabilities and dedication of the Medgenics Research and Development Group. Our goal in conducting this clinical trial is to validate the new BioPump platform using an updated protocol and second generation viral vector. While we expect to see the same advantages over other forms of protein replacement that we saw with the first generation vector, we also hope to see substantially sustained levels of protein production,” commented Dr. Garry Neil, Global Head, Research and Development of Medgenics. “Treatment alternatives for EPO-dependent anemia patients are still needed, so we hope to demonstrate the BioPump’s capability to produce physiologically relevant levels of autologous EPO in patients with CKD and ESRD. We also hope to validate the new platform as a delivery mechanism for multiple rare and orphan diseases. As part of this strategy, we expect to initiate several in vivo proof-of-concept studies in established mouse models in rare and orphan disease indications later this year.”
About the Trial (MG-EP-RF-02)
The trial will enroll up to 18 patients with either CKD or ESRD who require erythropoietin treatment for anemia. Each patient will receive one or more BioPumps and will be followed for at least one year. The trial endpoints include plasma erythropoietin levels, blood counts and safety assessment. Preliminary results are expected by the end of 2014. Additional details may be found on www.clinicaltrials.gov using identifier NCT02117427.
About Chronic Kidney Disease and End-stage Renal Disease
CKD is a progressive loss of kidney function over months to years. The three most common causes of CKD are diabetes mellitus, hypertension and glomerulonephritis. ESRD is also known as Stage 5 CKD. ESRD requires kidney replacement therapy, either dialysis or kidney transplantation.
About Medgenics, Inc.
Medgenics is developing and commercializing BioPump(TM), a proprietary platform for the sustained production and delivery of therapeutic proteins using ex-vivo gene therapy and the patient’s own tissue for the treatment of orphan and rare diseases. For more information, visit the Company’s website at www.medgenics.com.
SOURCE: Medgenics
Post Views: 92
Trial to Validate a Second Generation Viral Vector for Potential Use in Future Clinical Studies in Rare and Orphan Diseases
WAYNE, PA, USA and MISGAV, Israel I June 5, 2014 I Medgenics, Inc. (NYSE MKT:MDGN), the developer of a novel platform technology for the sustained production and delivery of therapeutic proteins in patients using ex-vivo gene therapy and their own tissue for the treatment of rare and orphan diseases, today announced that the first patient has been enrolled in its Phase 1/2 clinical trial of MDGN-201 (EPODURETM).
The aim of the trial is to validate the potential of the Company’s BioPump(TM) platform using a second generation vector, which was developed to enhance durability of the proposed therapeutic effect. The study will evaluate the potential of the updated platform to offer sustained production and delivery of erythropoietin (EPO) to treat anemia in dialysis patients with end-stage renal disease (ESRD) or chronic kidney disease (CKD).
“The initiation of this trial is a testament to the capabilities and dedication of the Medgenics Research and Development Group. Our goal in conducting this clinical trial is to validate the new BioPump platform using an updated protocol and second generation viral vector. While we expect to see the same advantages over other forms of protein replacement that we saw with the first generation vector, we also hope to see substantially sustained levels of protein production,” commented Dr. Garry Neil, Global Head, Research and Development of Medgenics. “Treatment alternatives for EPO-dependent anemia patients are still needed, so we hope to demonstrate the BioPump’s capability to produce physiologically relevant levels of autologous EPO in patients with CKD and ESRD. We also hope to validate the new platform as a delivery mechanism for multiple rare and orphan diseases. As part of this strategy, we expect to initiate several in vivo proof-of-concept studies in established mouse models in rare and orphan disease indications later this year.”
About the Trial (MG-EP-RF-02)
The trial will enroll up to 18 patients with either CKD or ESRD who require erythropoietin treatment for anemia. Each patient will receive one or more BioPumps and will be followed for at least one year. The trial endpoints include plasma erythropoietin levels, blood counts and safety assessment. Preliminary results are expected by the end of 2014. Additional details may be found on www.clinicaltrials.gov using identifier NCT02117427.
About Chronic Kidney Disease and End-stage Renal Disease
CKD is a progressive loss of kidney function over months to years. The three most common causes of CKD are diabetes mellitus, hypertension and glomerulonephritis. ESRD is also known as Stage 5 CKD. ESRD requires kidney replacement therapy, either dialysis or kidney transplantation.
About Medgenics, Inc.
Medgenics is developing and commercializing BioPump(TM), a proprietary platform for the sustained production and delivery of therapeutic proteins using ex-vivo gene therapy and the patient’s own tissue for the treatment of orphan and rare diseases. For more information, visit the Company’s website at www.medgenics.com.
SOURCE: Medgenics
Post Views: 92
