Category: Press Releases

NEW YORK, July 27, 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 announced that the United States Patent and Trademark Office (USPTO) on June 23, 2020 issued a composition of matter patent, U.S. Patent No. 10,689,625, with claims directed to the AAV vector used in PR006, Prevail’s experimental gene therapy program for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN). The base patent term extends until October 2038, excluding patent term extensions or coverage in additional related patent filings.

“At Prevail, we are working continuously to bring innovative treatments to patients with neurodegenerative diseases,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “FTD-GRN is a devastating condition, with no disease-modifying therapeutic options available. We are excited about the possibility of making a significant impact with PR006.”

In March of this year, Prevail announced an active IND for the Phase 1/2 clinical trial of PR006 for the treatment of FTD-GRN. The U.S. Food and Drug Administration (FDA) has granted the therapy Fast Track Designation.

PR006 is being developed as a potential one-time gene therapy for FTD-GRN, a progressive neurodegenerative disease caused by mutations in the GRN gene that reduce production of progranulin, a protein critical for lysosomal function, neuronal survival and normal microglial activities. The progranulin deficiency leads to lysosomal dysfunction, ineffective protein degradation and recycling, neuroinflammation, and ultimately neurodegeneration and death, typically within three to ten years of diagnosis.

PR006 is designed to increase progranulin levels in the brains of FTD-GRN patients by delivering a healthy GRN gene using an AAV9 vector.

About Frontotemporal Dementia with GRN Mutations

Frontotemporal dementia (FTD) is the second most common cause of dementia in people under the age of 65, after Alzheimer’s disease. FTD affects 50,000 to 60,000 people in the U.S. and 80,000 to 110,000 individuals in the European Union. FTD-GRN represents 5-10% of all patients with FTD. FTD results from the progressive degeneration of the frontal and temporal lobes of the brain, which control decision-making, behavior, emotion and language. In FTD-GRN patients, reduced levels of progranulin lead to age-dependent lysosomal dysfunction, neuroinflammation, and neurodegeneration. There are no approved treatments for FTD or FTD-GRN.

About Prevail Therapeutics

Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. In addition to PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN), the company is developing PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease, and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson’s with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to Prevail

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail’s ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail’s novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail’s gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail’s filings with the Securities and Exchange Commission (SEC), including the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on May 14, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Preclinical Data Demonstrate Potential of Prevail’s AAV Gene Therapy Approach to Slow or Halt Progression in Multiple Neurodegenerative Diseases

Company Provides Overview of Planned Phase 1/2 PR006 PROCLAIM Clinical Trial for FTD-GRN Patients

Company to Host a Panel Discussion and Q&A Session on FTD-GRN
with Jonathan Rohrer, Ph.D., MRCP, an Expert in Frontotemporal Dementia (FTD) and its Genetic Causes

NEW YORK, July 22, 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 announced three upcoming poster presentations at the Alzheimer’s Association International Conference (AAIC) 2020. These data underscore the robust preclinical evidence in support of Prevail’s AAV-based gene therapy approach, and highlight the Company’s strategy to validate these data in the planned PROCLAIM clinical trial evaluating PR006 for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN). The conference will be held virtually July 27-31, 2020.

“Our novel gene therapy candidates have the potential to transform the treatment of patients with FTD-GRN and other devastating neurodegenerative diseases,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “An increased understanding of genetically defined forms of neurodegenerative diseases, including the role that GRN mutations can play in frontotemporal dementia, has opened exciting possibilities to stop or slow disease progression using a gene therapy approach.”

PR001

Prevail will highlight preclinical data demonstrating the effect of PR001 treatment on key disease biomarkers and functional parameters. These data provide the basis for the Company’s clinical trials for Type 2 neuronopathic Gaucher disease (nGD) patients and Parkinson’s disease with GBA1 mutations (PD-GBA) patients.

Poster title: PR001 Gene Therapy Improved Phenotypes in Models of Parkinson’s Disease with GBA1 Mutation
Session date and time: Monday, July 27, 12:00 a.m. – 11:59 p.m. CDT

PR006

Prevail will present the design of the PROCLAIM Phase 1/2 clinical trial for the treatment of FTD-GRN patients and preclinical data demonstrating the effect of PR006 treatment on progranulin expression, lysosomal dysfunction and inflammation in the CNS.

Poster title: Preclinical Development of PR006, a Gene Therapy for the Treatment of Frontotemporal Dementia with Progranulin Mutations
Session date and time: Monday, July 27, 12:00 a.m. – 11:59 p.m. CDT

Poster title: Design of a Phase 1/2 Study of an AAV9-Based Gene Therapy for Fronto-Temporal Dementia Patients with Pathogenic GRN Mutations (PROCLAIM Trial)
Session date and time: Wednesday, July 29, 12:00 a.m. – 11:59 p.m. CDT

FTD-GRN Panel Discussion and Q&A Session

In addition to its presentations at AAIC, on Wednesday, July 29 at 2:30 p.m. EDT, management will be hosting a panel discussion and Q&A session on FTD-GRN by Jonathan Rohrer, Ph.D., MRCP, an expert in frontotemporal dementia (FTD) and its genetic causes. A live webcast of the event and replay following its conclusion will be available on the Events and Presentations section of the Company’s website at https://ir.prevailtherapeutics.com/events.

About Prevail Therapeutics

Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson’s with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to Prevail

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevail’s gene therapy candidates to transform the treatment of patients with, and slow or halt the progression of, FTD-GRN and other neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail’s novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail’s gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail’s filings with the Securities and Exchange Commission (SEC), including the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on May 14, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:

Mary Carmichael
Ten Bridge Communications
mary@tenbridgecommunications.com
617-413-3543

Investor Contact:

investors@prevailtherapeutics.com

Source: Prevail Therapeutics

Phase 1/2 Trial of PR001 for Parkinson’s Disease with GBA1 Mutations Ongoing; Study Startup Activities
Progressing for Phase 1/2 Trials of PR001 for Type 2 Neuronopathic Gaucher Disease and PR006 for
Frontotemporal Dementia with GRN Mutations

Data Presentations Highlight Potential of AAV Gene Therapy Approach to
Slow or Stop Neurodegenerative Disease Progression in Preclinical Models

NEW YORK, May 14, 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 business highlights and reported financial results for the first quarter ended March 31, 2020.

“We are excited to continue the clinical development of PR001 and are on track to report interim data for a subset of patients from our Phase 1/2 clinical trial of PR001 for Parkinson’s disease with GBA1 mutations (PD-GBA) later this year. In addition, we are advancing our AAV gene therapy-based pipeline, with the planned mid-year initiation of Phase 1/2 clinical trials of PR001 for Type 2 neuronopathic Gaucher disease (nGD) and PR006 for frontotemporal dementia with GRN mutations (FTD-GRN),” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “In addition, at ASGCT and AAT-AD/PD, we presented or will present data that validate the potential of these products for neurodegenerative disease patients with urgent unmet needs, and detailed our ongoing and planned clinical trials.”

Recent Business Highlights and Updates:

• Data and Clinical Trial Designs Presented or to be Presented for PR001 in nGD and PR006 in FTD-GRN at the 2020 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting: In separate presentations, Prevail described the design of two planned clinical trials: the PROVIDE Phase 1/2 clinical trial of PR001 for the treatment of patients with Type 2 nGD, and the PROCLAIM Phase 1/2 clinical trial of PR006 for the treatment of FTD-GRN. Preclinical data serving as the basis for the company’s clinical programs, including PR001 for nGD and PD-GBA and PR006 for FTD-GRN, will also be presented.

• Data and Clinical Trial Design Presented for PR001 in PD-GBA and PR006 in FTD-GRN at the AAT-AD/PD Focus Meeting 2020: Advances in Alzheimer’s and Parkinson’s Therapies: Presentations at the AAT-AD/PD Focus Meeting included details on the design of Prevail’s PROPEL Phase 1/2 clinical trial to evaluate the safety and efficacy of PR001 in patients with PD-GBA, and preclinical data related to PR001 for PD-GBA and PR006 for FTD-GRN.

• Clinical Development of PR001: As announced in March 2020, Prevail initiated dosing for the PROPEL Phase 1/2 trial of PR001 for patients with PD-GBA. As previously reported, in response to the COVID-19 pandemic, trial sites had temporarily suspended patient screening and enrollment activity. Prevail now expects patient screening to resume by the end of the second quarter of 2020 at certain clinical sites. The Company remains on track to report interim data on a subset of patients in the second half of 2020.In addition, study startup activities are continuing for the PROVIDE Phase 1/2 clinical trial of PR001 for Type 2 nGD, and the Company intends to initiate dosing in mid-2020. Prevail also continues to expect to initiate the PROGRESS Phase 1/2 clinical trial of PR001 for Type 3 nGD in the second half of 2020. The timelines for PR001 are subject to any delays related to the COVID-19 pandemic.Clinical Development of PR006: Study startup activities are also underway for the PROCLAIM Phase 1/2 clinical trial of PR006 for FTD-GRN patients, which is planned to initiate in mid-2020, subject to any delays related to the COVID-19 pandemic.

First Quarter 2020 Financial Results

• Cash Position: Cash and cash equivalents were $149.6 million as of March 31, 2020, as compared to $168.1 million as of December 31, 2019. As previously guided, the Company expects its current cash position to be sufficient to fund operating expenses into the first half of 2022.

• R&D Expenses: R&D expenses were $11.4 million for the three months ended March 31, 2020, compared to $8.4 million for the three months ended March 31, 2019. The increase was primarily related to the Company’s development programs, specifically as a result of increased clinical trial activities, and headcount.

• G&A Expenses: G&A expenses were $7.9 million for the three months ended March 31, 2020, compared to $1.9 million for the three months ended March 31, 2019. The increase was primarily due to an increase in professional services fees associated with the ongoing arbitration matter, personnel costs resulting from increased headcount, and other corporate and public company-related expenses.

• Net Loss: Net loss was $18.6 million, or $0.56 loss per share, for the three months ended March 31, 2020, compared to $9.9 million, or $1.73 loss per share, for the three months ended March 31, 2019.

About Prevail Therapeutics

Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson’s with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to Prevail

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the potential impact of COVID-19 on Prevail’s ongoing and planned clinical trials, business and operations; the potential of Prevail’s gene therapies to modify the course of neurodegenerative diseases; the anticipated timing of Prevail’s clinical trials of PR001 in PD-GBA and in nGD and Prevail’s clinical trial of PR006, including resuming of delayed trials and initiation of new trials; the expected timing of reporting of interim data for a subset of patients from Prevail’s Phase 1/2 clinical trial of PR001; and expectations regarding Prevail’s cash runway. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail’s novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail’s gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises.

These and other risks are described more fully in Prevail’s filings with the Securities and Exchange Commission (SEC), including the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on or about May 14, 2020, the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2019, filed with the SEC on March 26, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Prevail Therapeutics Inc.
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Balance Sheets
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Media Contact:

Mary Carmichael
Ten Bridge Communications
mary@tenbridgecommunications.com
617-413-3543

Investor Contact:

investors@prevailtherapeutics.com

Source: Prevail Therapeutics

Preclinical Data Demonstrate Potential of Prevail’s AAV Gene Therapy Approach to Slow or Halt Progression in Multiple Neurodegenerative Diseases

Company Provides Design Overview for Planned Phase 1/2 PR001 PROVIDE and PR006 PROCLAIM Clinical Trials

NEW YORK, April 29, 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 announced two upcoming oral presentations and two poster presentations at the virtual 2020 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. These data underscore the robust preclinical evidence in support of Prevail’s AAV-based gene therapy approach, and highlight the company’s strategy to validate these data in multiple ongoing or planned clinical trials.

“Patients living with neurodegenerative diseases are in urgent need of novel therapies with the potential to slow or stop the course of their disease,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “Our gene therapy candidates have the potential to improve outcomes for patients with genetically defined forms of Parkinson’s disease, frontotemporal dementia and neuronopathic Gaucher disease.”

PR001

Prevail will highlight the design of the planned PROVIDE Phase 1/2 trial for the treatment of Type 2 neuronopathic Gaucher disease (nGD) patients and preclinical data demonstrating the impact of PR001 treatment on key disease biomarkers and functional parameters. These data provide the basis for the company’s clinical programs in nGD and Parkinson’s disease with GBA1 mutations (PD-GBA).

Poster title: Design of a Phase 1/2 Study to Evaluate the Safety and Efficacy of an AAV9-Based Gene Therapy in Infants with Type 2 Gaucher Disease (PROVIDE Trial)
Session title: AAV Vectors – Clinical Studies
Session date and time: Wednesday, May 13^th, 5:30 p.m.-6:30 p.m. ET

Presentation title: PR001 Gene Therapy Increased GCase Activity and Ameliorated GBA1-Associated Disease Phenotypes
Session title: AAV Vectors Preclinical and Proof-of-Concept Studies in Systemic Diseases
Presentation date and time: Thursday, May 14^th3:45 p.m.-4:00 p.m. ET
Presenter: Patty Sheehan, Ph.D., Senior Scientist, Prevail Therapeutics

PR006

Prevail will present the design of the PROCLAIM Phase 1/2 clinical trial for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN) and preclinical data demonstrating the impact of PR006 treatment on progranulin expression, lysosomal dysfunction and inflammation in the CNS.

Poster title: Proposed Patient Population and Outcome Measures for a First in Human Study of PR006, an AAV9-Based Gene Therapy, for Fronto-Temporal Dementia Patients with Pathogenic GRN Mutations
Session title: AAV Vectors – Clinical Studies
Session date and time: Wednesday, May 13^th, 5:30 p.m.-6:30 p.m. ET

Presentation title: PR006, an AAV Gene Therapy Vector Expressing Progranulin, Improved FTD-GRN Phenotypes In Vitro and In Vivo
Session title: AAV Vectors Preclinical and Proof-of-Concept Studies in CNS Disorders
Presentation date and time: Friday, May 15^th10:15 a.m.-10:30 a.m. ET
Presenter: Laura D. Heckman, Ph.D., Director, Preclinical Development, Prevail Therapeutics

About Prevail Therapeutics

Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson’s with GBA and OrbiMed, and is headquartered in New York, NY.

Media Contact:

Mary Carmichael
Ten Bridge Communications
mary@tenbridgecommunications.com
617-413-3543

Investor Contact:

investors@prevailtherapeutics.com

Source: Prevail Therapeutics

Design of PROPEL Phase 1/2 Clinical Trial for Parkinson’s Disease Patients with GBA1 Mutations to Be Presented

Preclinical Data of PR001 for Parkinson’s Disease with GBA1 Mutations and PR006 for Frontotemporal Dementia with GRN Mutations to Be Presented

NEW YORK, March 31, 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 announced three upcoming presentations at the AAT-AD/PD Focus Meeting 2020: Advances in Alzheimer’s and Parkinson’s Therapies, taking place virtually April 2-5, 2020. All presentations will be available on-demand for registered participants in the AAT-AD/PD™ Virtual Meeting platform. The presentations include information about Prevail’s PR001 program for Parkinson’s disease patients with GBA1 mutations (PD-GBA), and Prevail’s PR006 program for frontotemporal dementia patients with GRN mutations (FTD-GRN).

Preclinical data related to PR001 for PD-GBA and the design of PROPEL, Prevail’s Phase 1/2 clinical trial to evaluate the safety and efficacy of PR001 in patients with PD-GBA, will be presented on Saturday, April 4, 2020:

Presentation title: PR001 Gene Therapy Improves PD-GBA Phenotypes in Mouse Models by Increasing GCase Activity
Symposium title: Emerging Therapies for PD Related to Glucocerebroside Mutations: From Bench to First in Human Trial
Date: Saturday, April 4, 2020
Presenter: Asa Abeliovich, M.D., Ph.D., Founder and CEO, Prevail Therapeutics

Presentation title: Design of the First-in-Human Trial of Novel AAV9-based Gene Therapy for Parkinson’s Disease with Pathogenic GBA1 Mutations
Symposium title: Emerging Therapies for PD Related to Glucocerebroside Mutations: From Bench to First in Human Trial
Date: Saturday, April 4, 2020
Presenter: Olga Uspenskaya, M.D., Ph.D., Senior Medical Director, Prevail Therapeutics

Preclinical data related to PR006 for FTD-GRN will also be presented on Saturday, April 4, 2020:

Presentation title: PR006 Gene Therapy Improves FTD-GRN Phenotypes in Model Systems by Increasing Progranulin Levels
Symposium title: ALS, FTD and PSP: Diagnosis and Treatment
Date: Saturday, April 4, 2020
Presenter: Laura D. Heckman, Ph.D., Director, Prevail Therapeutics

About Prevail Therapeutics

Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson’s with GBA and OrbiMed, and is headquartered in New York, NY.

Media Contact:

Mary Carmichael
Ten Bridge Communications
mary@tenbridgecommunications.com
617-413-3543

Investor Contact:

investors@prevailtherapeutics.com

Source: Prevail Therapeutics

Dosing Initiated in PROPEL Phase 1/2 Trial of PR001 for Treatment of Parkinson’s Disease with GBA1 Mutations

Preparations Underway to Initiate PROVIDE Phase 1/2 Trial of PR001 for Treatment of Type 2 Neuronopathic Gaucher Disease in Mid-2020

IND for PR006 for Treatment of Patients with Frontotemporal Dementia with GRN Mutation Active; Initiation of PROCLAIM Phase 1/2 Trial Planned for Mid-2020

Cash Runway into First Half of 2022

NEW YORK, March 26, 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 reported financial results for the full year ended December 31, 2019, and reviewed recent business highlights.

“2019 was an important year for Prevail as we continued to make important clinical and regulatory progress across our portfolio of novel AAV9-based gene therapy candidates for neurodegenerative diseases and brought the company public,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “Dosing has begun in our PROPEL clinical trial of PR001 for patients with Parkinson’s disease with GBA1 mutations, or PD-GBA. Additionally, we are working to initiate three more Phase 1/2 clinical trials this year: the PROVIDE and PROGRESS trials of PR001 for the treatment of Type 2 and Type 3 neuronopathic Gaucher disease, or nGD, respectively, and the PROCLAIM trial of PR006 for the treatment of frontotemporal dementia patients with GRN mutation, or FTD-GRN — potentially the first gene therapy for this condition to enter clinical trials. Our ability to advance multiple candidates towards the clinic over the last year reflects the dedication of our team, the potential reach of our platform, and the urgent need for novel potentially disease-modifying therapies for patients with these genetically-defined neurodegenerative diseases.”

Recent Business Highlights and Updates:

• PROPEL trial for PD-GBA underway: Prevail has initiated dosing in its PROPEL Phase 1/2 trial of PR001 for patients with PD-GBA. In response to the COVID-19 pandemic, trial sites have temporarily suspended patient screening and enrollment activity. The Company still intends to report interim data on a subset of patients in the second half of 2020, however, this may be impacted if there is a prolonged suspension of enrollment. This randomized, double-blind trial is enrolling up to 16 moderate-to-severe PD-GBA patients to evaluate the safety and tolerability of two escalating doses of PR001, and will measure key biomarkers and exploratory efficacy endpoints. Currently, there are no treatments available to slow or stop disease progression for PD-GBA patients.

• Clinical Development of PR001 for nGD Advancing: Prevail announced in December 2019 that its Investigational New Drug (IND) application for PR001 for the treatment of nGD has been accepted by the U.S. Food and Drug Administration (FDA) and is now active. Study startup activities are ongoing for the PROVIDE Phase 1/2 clinical trial for Type 2 nGD. The Company intends to initiate dosing in the PROVIDE trial in mid-2020 and in the PROGRESS Phase 1/2 clinical trial for Type 3 nGD in the second half of 2020, in each case, subject to any delays related to the COVID-19 pandemic. There are no effective treatments available to address the neurological manifestations associated with either type of neuronopathic Gaucher disease.

• PR001 Granted Orphan Drug and Rare Pediatric Disease Designations: The FDA has granted Orphan Drug Designation for PR001 for the treatment of patients with Gaucher disease, and Rare Pediatric Disease Designation for the treatment of nGD, the most severe form of the condition. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity. Programs with Rare Pediatric Disease status may, upon marketing approval, qualify for a fully transferable priority review voucher applicable to a subsequent marketing application.

• IND Active for PR006 for the Treatment of Patients with FTD-GRN: Prevail announced in March 2020 that the FDA accepted the Company’s IND application for PR006 for the treatment of FTD-GRN. FTD-GRN is a fatal, rapidly progressive, early-onset form of dementia for which there are no FDA approved treatments. The Company believes that PR006, which is being developed as a potential one-time gene therapy, has the ability to slow or stop progression of FTD-GRN. Prevail plans to initiate the PROCLAIM Phase 1/2 clinical trial for FTD-GRN patients in mid-2020, subject to any delays related to the COVID-19 pandemic.

• PR006 Granted Orphan Drug and Fast Track Designations: The FDA has granted Orphan Drug Designation for PR006 for the treatment of FTD and Fast Track Designation for FTD-GRN. Programs with Fast Track status receive accelerated approval or rolling review of a company’s Biologics License Application (BLA). In addition, such a product candidate could be eligible for Priority Review if supported by clinical data at the time of BLA submission.

• Preclinical Data Presented for PR001 in nGD and PR006 in FTD-GRN at WORLDSymposium™: Prevail presented preclinical data at the 16th Annual WORLDSymposium in February 2020 demonstrating proof of concept in both nGD and FTD-GRN. Prevail also hosted a presentation and roundtable discussion during the WORLDSymposium featuring Professor Ari Zimran, M.D., a key opinion leader in the field of nGD. A recording of this event, including Dr. Zimran’s presentation “Unmet Needs and Emerging Therapeutic Options for Neuronopathic Gaucher Disease,” can be found under “Events and Presentations” in the Investors & Media section of the company’s website, www.prevailtherapeutics.com.

• Strengthened Leadership with Board Appointment: Prevail appointed Morgan Sheng, Ph.D., to its Board of Directors. Dr. Sheng, who brings a wealth of expertise in neurodegenerative disease drug development, is a Core Institute Member and Co-Director of the Stanley Center for Psychiatric Research at the Broad Institute of MIT and Harvard, and a Professor in the Department of Brain and Cognitive Science at MIT. Previously, he was Vice President of Neuroscience at Genentech, where he was head of neuroscience research and drug discovery.

Full Year 2019 Financial Results

• Cash Position: Cash and cash equivalents were $168.1 million as of December 31, 2019, as compared to $63.0 million as of December 31, 2018. Following a strategic review and prioritization of programs, the Company expects its current cash position to be sufficient to fund operating expenses into the first half of 2022, versus mid-2021 as previously guided.

• R&D Expenses: R&D expenses were $48.8 million for the year ended December 31, 2019, compared to $14.1 million for the year ended December 31, 2018. The increase was primarily related to the Company’s development programs, as a result of increased manufacturing-related spend, clinical and preclinical activities, and headcount.

• G&A Expenses: G&A expenses were $17.0 million for the year ended December 31, 2019, compared to $4.7 million for the year ended December 31, 2018. The increase was primarily due to an increase in personnel costs resulting from increased headcount, professional services fees, and other corporate-related expenses.

• Net Loss: Net loss was $63.2 million, or $2.22 loss per share, for the year ended December 31, 2019, compared to $19.1 million, or $3.71 loss per share, for the year December 31, 2018.

About Prevail Therapeutics
Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson’s disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with a GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson’s with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to Prevail
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the potential impact of COVID-19 on Prevail’s ongoing and planned clinical trials, business and operations; the potential of Prevail’s gene therapies to modify the course of neurodegenerative diseases; the anticipated timing of Prevail’s Phase 1/2 clinical trials of PR001 in PD-GBA and in nGD and Prevail’s clinical trial of PR006; and the potential impacts of Orphan Drug and Rare Pediatric Disease Designations. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail’s novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail’s gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises.

These and other risks are described more fully in Prevail’s filings with the Securities and Exchange Commission (SEC), including the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2019, to be filed with the SEC on or about March 26, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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NEW YORK, March 24, 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 announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the Company’s experimental gene therapy program, PR006, to slow the progression of frontotemporal dementia with a GRN mutation (FTD-GRN). Prevail announced an active IND for the Phase 1/2 clinical trial of PR006 for the treatment of FTD-GRN earlier this month.

“The FDA’s decision to grant Fast Track Designation for PR006 is an important step forward in our mission to deliver a potentially disease-modifying gene therapy to FTD-GRN patients as quickly as possible,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “FTD-GRN progresses rapidly and there are currently no therapeutic options available. We believe PR006 has the potential to fill this unmet medical need and make a significant impact for patients.”

About Fast Track Designation

The FDA’s Fast Track designation is a process designed to facilitate the development and expedite the review of product candidates to treat serious conditions and fill an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. It allows for accelerated approval or rolling review of a company’s Biologics License Application (BLA). In addition, such a product candidate could be eligible for Priority Review if supported by clinical data at the time of BLA submission.

About Frontotemporal Dementia with a GRN Mutation

Frontotemporal dementia (FTD) is the second most common cause of dementia in people under the age of 65, after Alzheimer’s disease. FTD affects 50,000 to 60,000 people in the U.S. and 80,000 to 110,000 individuals in the European Union. FTD-GRN represents 5-10% of all patients with FTD. FTD results from the progressive degeneration of the frontal and temporal lobes of the brain, which control decision-making, behavior, emotion and language. In FTD-GRN patients, reduced levels of progranulin lead to age-dependent lysosomal dysfunction, neuroinflammation, and neurodegeneration. There are no approved treatments for FTD or FTD-GRN.

About Prevail Therapeutics

Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR006 for patients with frontotemporal dementia with a GRN mutation (FTD-GRN); PR001 for patients with Parkinson’s disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson’s with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to Prevail

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail’s ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases and slow the progression of FTD-GRN. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail’s novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail’s gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevail’s filings with the Securities and Exchange Commission (SEC), including the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:

Mary Carmichael
Ten Bridge Communications
mary@tenbridgecommunications.com

Investor Contact:

investors@prevailtherapeutics.com

Source: Prevail Therapeutics

NEW YORK, March 03, 2020 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, provided an update today on the clinical advancement of its gene therapy program PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA). Enrollment in the PR001 Phase 1/2 PROPEL clinical trial is progressing, patient dosing continues, and the Company is on track to report interim data on a subset of patients in the second half of 2020.

The Company will present on its clinical progress at the Cowen & Co. Annual Healthcare Conference in Boston today.

“We believe the PROPEL trial makes PR001 the first potentially disease-modifying gene therapy for PD-GBA patients to enter clinical trials. Its ongoing progress brings us a step closer to new treatment options for patients living with PD-GBA,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “We are excited about the potential of PR001 to slow or stop disease progression for PD-GBA patients.”

The PROPEL trial is a randomized, double-blind Phase 1/2 clinical trial evaluating the safety and tolerability of two escalating dose levels of PR001 in up to 16 patients with moderate-to-severe PD-GBA. The trial also evaluates the effect of PR001 on biomarkers of disease activity and on Parkinson’s disease clinical efficacy measures. Full trial details are available at clinicaltrials.gov.

PR001 utilizes an AAV9 viral vector to deliver the GBA1 gene to a patient’s cells, correcting the lysosomal enzyme deficiency caused by PD-GBA patients’ GBA1 mutations. GBA1 encodes the lysosomal enzyme, beta-glucocerebrosidase, or GCase, which is required for the disposal and recycling of glycolipids. PD-GBA patients have a mutation in at least one chromosomal copy of GBA1.

In addition to the PROPEL clinical trial for patients with PD-GBA, PR001 is also being developed for neuronopathic Gaucher disease, a devastating disorder that shares the same underlying genetic mechanism. In December 2019, the Company announced that its IND for PR001 for the treatment of neuronopathic Gaucher disease is active.

About PD-GBA

Parkinson’s disease is a chronic, progressive neurodegenerative disorder that affects up to one million people in the United States and more than seven million people worldwide. PD-GBA affects 7% to 10% of the total Parkinson’s disease population worldwide and an estimated 90,000 individuals in the United States alone. GBA1 encodes the lysosomal enzyme, beta-glucocerebrosidase, or GCase. Mutations in the GBA1 gene lead to a deficiency of GCase, resulting in lysosomal dysfunction in CNS cells, which we believe leads to the inflammation and neurodegeneration present in PD-GBA. GBA1 mutations impact the risk of developing Parkinson’s disease as well as many other aspects of the disease course, including the severity, age of onset and rate of progression of disease and the likelihood of dementia. There are no treatments available that modify the progressive course or the underlying disease process of Parkinson’s disease.

About Prevail Therapeutics

Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinson’s disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinson’s with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to Prevail

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning Prevail’s ability to develop meaningful therapeutic advances for patients with neurodegenerative diseases, the continued progression of our PROPEL Phase 1/2 clinical trial, the continued dosing of patients and the expected timing for the delivery of interim data from a subset of patients from our PROPEL Phase 1/2 clinical trial. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevail’s novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevail’s gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; and the fact that gene therapies are novel, complex and difficult to manufacture. These and other risks are described more fully in Prevail’s filings with the Securities and Exchange Commission (SEC), including the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the period ended September 30, 2019, filed with the SEC on November 12, 2019, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:

Mary Carmichael
Ten Bridge Communications
mary@tenbridgecommunications.com

Investor Contact:

investors@prevailtherapeutics.com

Source: Prevail Therapeutics