{"id":380665,"date":"2020-11-13T07:03:29","date_gmt":"2020-11-13T12:03:29","guid":{"rendered":"http:\/\/www.marketnewsdesk.com\/?p=380665"},"modified":"2020-11-13T07:03:29","modified_gmt":"2020-11-13T12:03:29","slug":"prevail-therapeutics-reports-third-quarter-2020-financial-results-and-business-highlights","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/prevail-therapeutics-reports-third-quarter-2020-financial-results-and-business-highlights\/","title":{"rendered":"Prevail Therapeutics Reports Third Quarter 2020 Financial Results and Business Highlights"},"content":{"rendered":"
\n

\n Patient Dosing Continues in the Phase 1\/2 PROPEL Trial of PR001 for Parkinson\u2019s Disease with GBA1 Mutations<\/em>
\n \n <\/p>\n

\n Phase 1\/2 PROVIDE Trial of PR001 for Type 2 Gaucher Disease and Phase 1\/2 PROCLAIM Trial of PR006 for Frontotemporal Dementia with GRN Mutations Expected to Initiate Enrollment in Fourth Quarter of 2020<\/em>\n <\/p>\n

\n PR001 Receives U.S. FDA Fast Track Designation for Neuronopathic Gaucher Disease <\/em>\n <\/p>\n

NEW YORK, Nov. 13, 2020 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today reviewed recent clinical and business updates and reported financial results for the third quarter ended September 30, 2020.<\/p>\n

\u201cWe\u2019re pleased to be making significant progress across our pipeline as we seek to develop urgently needed disease-modifying gene therapy treatments for patients with neurodegenerative diseases,\u201d said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. \u201cWe are encouraged by the continuation of patient dosing in our Phase 1\/2 PROPEL trial of PR001 for Parkinson\u2019s disease with GBA1<\/em> mutations, and we are excited to advance our PROVIDE and PROCLAIM clinical trials for Type 2 Gaucher disease and frontotemporal dementia with GRN<\/em> mutations, respectively, this year.\u201d<\/p>\n

\n Recent Business <\/strong>
\n Updates<\/strong>\n <\/p>\n

\n Patient Dosing Continues in Phase 1\/2 PROPEL Trial of PR001 for P<\/strong>
\n arkinson\u2019s disease with <\/strong>
\n
\n GBA1<\/em>
\n <\/strong>
\n mutations (PD-GBA)<\/strong>
\n : <\/strong>Enrollment in the Phase 1\/2 PROPEL clinical trial for PD-GBA has resumed following implementation of modifications to the clinical protocol. As previously announced, Prevail elected to modify the immunosuppression regimen in the clinical protocol for PROPEL and has adapted the trial design to be open-label. The Company expects to provide the next biomarker and safety analysis on a subset of patients in the PROPEL trial by mid-2021.<\/p>\n

\n Phase 1\/2 PROVIDE Trial Expected to Initiate Enrollment in Fourth Quarter of 2020: <\/strong>Initiation of patient enrollment remains on track for the fourth quarter of 2020 for the Phase 1\/2 PROVIDE clinical trial of PR001 for Type 2 Gaucher disease. The optimized immunosuppression regimen used in the amended PROPEL trial will also be implemented in the PROVIDE trial. The Company currently anticipates it will provide the next update on PR001 biomarker and safety data for neuronopathic Gaucher disease (nGD) in 2021.<\/p>\n

\n Phase 1\/2 PROCLAIM Trial Expected to Initiate Enrollment in Fourth Quarter of 2020: <\/strong>Initiation of patient enrollment remains on track for the fourth quarter of 2020 for the Phase 1\/2 PROCLAIM clinical trial of PR006 for frontotemporal dementia with GRN<\/em> mutations (FTD-GRN). The optimized immunosuppression regimen used in the amended PROPEL trial will also be implemented in the PROCLAIM trial. The Company currently anticipates it will provide a biomarker and safety analysis on a subset of patients in the PROCLAIM trial in 2021.<\/p>\n

\n PR001 Granted U.S. FDA Fast Track Designation for <\/strong>
\n nGD<\/strong>
\n : <\/strong>The U.S. Food and Drug Administration (FDA) granted Fast Track designation for PR001 for the treatment of nGD. The FDA previously granted PR001 Rare Pediatric Disease designation for the treatment of nGD, and Orphan Drug designation for the treatment of patients with Gaucher disease. In addition, the FDA has granted Fast Track designation for PR001 for the treatment of PD-GBA.<\/p>\n

\n Strengthened Leadership with Board Appointment:<\/strong> Prevail has appointed William H. Carson, M.D., to its Board of Directors. Dr. Carson was most recently the President and CEO of\u00a0Otsuka Pharmaceutical Development & Commercialization, Inc.\u00a0(OPDC), leading the development and regulatory approvals of Otsuka\u2019s global compounds. Before joining Otsuka, he held several roles in the\u00a0CNS Research\u00a0and Development department at Bristol Myers Squibb. Dr. Carson\u00a0currently serves as Chairman of the Board of Directors of OPDC and is also the Chairman of the Board of the\u00a0Sozosei Foundation, a newly established Otsuka charitable organization with a main focus on decriminalization of mental illness. He is a Board Member of Excision Biotherapeutics and Trustee of the non-profit Internet2. He is a Distinguished Fellow of the\u00a0American Psychiatric Association, the\u00a0National Medical Association\u00a0and the\u00a0Executive Leadership Council.\u00a0Prior to joining the pharmaceutical industry,\u00a0Dr. Carson, a board-certified psychiatrist, was an Associate Professor in the\u00a0Department of Psychiatry and Behavioral Sciences\u00a0at the\u00a0Medical University of South Carolina.<\/p>\n

\n Favorable Decision\u00a0Received\u00a0in Alector Arbitration:\u00a0<\/strong>Prevail announced a favorable decision in the arbitration proceeding brought in 2019 by Alector Inc. against Prevail\u2019s Founder and Chief Executive Officer, Asa Abeliovich, M.D., Ph.D. The arbitrator rejected Alector\u2019s claims against Dr. Abeliovich that Alector confidential information was used in connection with his work on behalf of Prevail and that Alector had rights to Prevail\u2019s patents and patent applications. The arbitrator found that Dr. Abeliovich did not breach his confidentiality obligations to Alector under his consulting agreement. Prevail was not a party to this arbitration.<\/p>\n

\n Third Quarter 2020 Financial Results<\/strong>\n <\/p>\n