Category: Press Releases


Prevail Therapeutics Announces Investigational New Drug Application Active for PR006 for the Treatment of Frontotemporal Dementia with GRN Mutation

By michelle@orrbitt.com,

NEW YORK, March 02, 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 accepted the Company’s Investigational New Drug (IND) application for its experimental gene therapy program, PR006, for the treatment of frontotemporal dementia patients with GRN mutation (FTD-GRN), and that the Company may proceed with the initiation of its Phase 1/2 clinical trial.

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.

“FTD-GRN is a serious and progressive neurodegenerative disease for which there are no approved treatments,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “Now that the PR006 IND is active, we look forward to advancing clinical development of PR006, which has the potential to positively impact the lives of patients by slowing or stopping the progression of this devastating disease.”

The PROCLAIM Phase 1/2 clinical trial will investigate the safety and tolerability of PR006, and will also measure key biomarkers and exploratory efficacy endpoints in patients with FTD-GRN. The Company plans to begin dosing for PROCLAIM this year. We believe this makes PR006 the first gene therapy for FTD-GRN to enter clinical trials.

The FDA has granted Orphan Drug Designation for PR006 for the treatment of patients with FTD.

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. 50,000 to 60,000 people in the U.S. and 80,000 to 110,000 individuals in the European Union are affected by FTD. Frontotemporal dementia with a GRN mutation (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 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 ability of PR006 to increase progranulin levels in the brains of FTD-GRN patients, and timing of enrollment and dosing of patients in the PROCLAIM 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

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Source: Prevail Therapeutics

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Prevail Therapeutics’ PR001 Receives Orphan Drug Designation and Rare Pediatric Disease Designation from FDA

By michelle@orrbitt.com,

Orphan Drug Designation Granted for Treatment of Gaucher Disease 

Rare Pediatric Disease Designation Granted for Treatment of
Neuronopathic Gaucher Disease

NEW YORK, Feb. 12, 2020 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (Nasdaq: PRVL) (“Prevail” or the “Company”), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the Company’s investigational gene therapy, PR001, for the treatment of patients with Gaucher disease. The Company also announced that the FDA has granted Rare Pediatric Disease Designation for PR001 for the treatment of neuronopathic Gaucher disease (nGD), the most severe form of the condition.

“We are pleased to receive these important designations from the FDA, which underscore the critical nature of our work,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “These designations support our conviction that new gene therapies for Gaucher disease are urgently needed — especially for the severe, neuronopathic form of the disease, for which there are no FDA-approved therapies.”

PR001 is also being developed as a potentially disease-modifying, single-dose gene therapy for Parkinson’s disease with GBA1 mutation (PD-GBA).

Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.

Rare Pediatric Disease Designation is granted by the FDA in the case of serious or life-threatening diseases affecting fewer than 200,000 people in the United States, primarily those 18 years of age and younger. The sponsor of a drug with Rare Pediatric Disease Designation may, upon marketing approval, qualify for receipt of a priority review voucher applicable to a subsequent marketing application, which voucher is fully transferable.

Prevail announced in December 2019 that its Investigational New Drug (IND) application for PR001 for the treatment of nGD is active. Prevail is proceeding with its Phase 1/2 clinical trial for Type 2 Gaucher disease patients and expects to initiate patient dosing during the first half of 2020. The Company also plans to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients in the second half of 2020 under the same IND.

About Neuronopathic Gaucher Disease

Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology. Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease. Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as seizures and gaze and motor abnormalities. There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher 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 timing and progress of clinical trials, and the potential advantages of Orphan Drug Designation and Rare Pediatric Disease Designation by the FDA. 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; despite receiving Rare Pediatric Disease designation, the Company may not be successful in obtaining a priority review voucher; 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

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Source: Prevail Therapeutics

 

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Prevail Announces Presentations at the 16th Annual WORLDSymposium™ 2020

By michelle@orrbitt.com,

NEW YORK, Feb. 06, 2020 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (Nasdaq: PRVL) (“Prevail” or the “Company”), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced the upcoming presentation of three abstracts at the 16th Annual WORLDSymposium™, taking place February 10-13, 2020, in Orlando, FL.  The abstracts include preclinical data from Prevail’s programs PR001 for neuronopathic Gaucher disease and PR006 for frontotemporal dementia with GRN mutation.

Two abstracts will be presented during the poster session on Monday, February 10th from 4:30 p.m. to 6:30 p.m. ET.  Poster information is as follows:

Abstract Title: Gene therapy PR006 increased progranulin levels and improved lysosomal related phenotypes in model systems (poster #52)
Presenter: Laura Heckman, Ph.D.

Abstract Title: Gene therapy PR001 increased GCase activity and improved neuronopathic Gaucher disease phenotypes in mouse models (poster #159)
Presenter: Laura Heckman, Ph.D.

One abstract will be presented during the Contemporary Forum session on Thursday, February 13th at 1:30 p.m. ET. Oral presentation information is as follows:

Abstract Title: Gene therapy PR006 increased progranulin levels and improved lysosomal related phenotypes in model systems
Presenter: Asa Abeliovich, M.D., Ph.D.

In addition to its presentations at WORLDSymposium, on Wednesday, February 12th at 8:30 p.m. ET, management will be hosting a presentation by Professor Ari Zimran, M.D., a key opinion leader in the field of neuronopathic Gaucher disease, on “Unmet Needs and Emerging Therapeutic Options for Neuronopathic Gaucher Disease”, with a roundtable discussion to follow.  A recording of this event and accompanying slides will be made available immediately following its conclusion and can be found under “Events and Presentations” in the Investors & Media section of the company’s website, www.prevailtherapeutics.com. The webcasts will be archived for 90 days following the presentation.

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. 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:

Katie Engleman
1AB
katie@1ABmedia.com

Investor Contact:

investors@prevailtherapeutics.com

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Source: Prevail Therapeutics

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Prevail Therapeutics Provides PR001 Program Update

By michelle@orrbitt.com,

PR001 Administered to International Type 2 Gaucher Disease Patient Via Compassionate Use Pathway

Company Advancing its Phase 1/2 Clinical Trial for the Treatment of Neuronopathic Gaucher Disease and Expects to Initiate Patient Dosing in First Half of 2020

NEW YORK, Jan. 09, 2020 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (Nasdaq: PRVL) (“Prevail” or the “Company”), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today provided an update on its investigational program, PR001, an AAV9-based gene therapy delivering the GBA1 gene, for the neuronopathic Gaucher disease (nGD) indication.

Prevail has granted a compassionate use request for the administration of PR001 to a single patient with Type 2 Gaucher disease via a compassionate use pathway, following approval by an international regulatory authority, and the patient was recently dosed. Type 2 Gaucher disease is the more severe form of nGD, which presents in infancy and involves rapidly progressing neurodegeneration leading to death in infancy or early childhood.

Separately, as announced on December 26, 2019, Prevail’s Investigational New Drug (IND) application for PR001 for the treatment of nGD is now active. The Company is proceeding with its Phase 1/2 clinical trial for Type 2 Gaucher disease patients and expects to initiate patient dosing during the first half of 2020. The Company also plans to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients in the second half of 2020 under the same IND. Prevail intends for the Company’s clinical studies to serve as the primary option for patients to receive access to any of Prevail’s investigational drugs.

“Our decision to grant a compassionate use request for this patient and our continued progress toward initiating clinical trials underscore our deep dedication to our mission of treating all eligible patients with neuronopathic Gaucher disease, the most severe form of Gaucher disease,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “We continue to believe that PR001 has the potential to be a disease-modifying therapy for the treatment of Parkinson’s disease with GBA1 mutation and neuronopathic Gaucher disease, which share the same underlying genetic mechanism.”

Prevail is also developing PR001 for Parkinson’s disease patients with a GBA1 mutation (PD-GBA). The Company has an active IND for PR001 for the treatment of PD-GBA and the PROPEL Phase 1/2 clinical trial for PD-GBA patients is now recruiting.

About Neuronopathic Gaucher Disease
Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology.  Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease.  Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as gaze and motor abnormalities and seizures.  There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher 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 and the timing and progress of clinical trials. 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:

Katie Engleman
1AB
katie@1ABmedia.com

Investor Contact:

investors@prevailtherapeutics.com

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Source: Prevail Therapeutics

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Prevail Therapeutics Announces IND Active for PR001 for Treatment of Neuronopathic Gaucher Disease

By michelle@orrbitt.com,

NEW YORK, Dec. 26, 2019 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (Nasdaq: PRVL) (“Prevail” or the “Company”), 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 notified Prevail that the Company’s Investigational New Drug (IND) application for PR001 for the treatment of neuronopathic Gaucher disease (nGD) patients is now active and that Prevail may proceed with initiating its proposed clinical trial. As previously reported, Prevail’s IND for PR001 for the treatment of nGD had been put on clinical hold by the FDA, and this clinical hold has now been removed.

The Company’s planned Phase 1/2 clinical trial for nGD patients will commence at a dose higher than originally proposed. Prevail submitted nonclinical data in which no PR001-related safety events or adverse findings were observed, supporting the initiation of the Phase 1/2 clinical trial at this higher dose.

Prevail is activating a Phase 1/2 clinical trial for Type 2 Gaucher disease patients and expects to initiate patient dosing during the first half of 2020. Type 2 Gaucher disease is the more severe form of nGD, which presents in infancy and involves rapidly progressing neurodegeneration leading to death in infancy or early childhood. The Company also plans to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients in the second half of 2020, under the same nGD IND.  Type 3 Gaucher disease is a form of nGD that typically presents in childhood and involves multiple neurological manifestations.

“We are pleased to now have an active IND for PR001 for the nGD indication and look forward to initiating a Phase 1/2 clinical trial in the first half of 2020,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “Patients with nGD have the most severe form of Gaucher disease and a significant unmet need for therapies to treat their neurological manifestations. We believe PR001 has tremendous potential to help patients suffering from this devastating disease.”

Prevail is also developing PR001 for Parkinson’s disease patients with a GBA1 mutation (PD-GBA). The Company has an active IND for PR001 for the treatment of PD-GBA and the PROPEL Phase 1/2 clinical trial for PD-GBA patients is now recruiting.

About Neuronopathic Gaucher Disease

Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology.  Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease.  Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as gaze and motor abnormalities and seizures.  There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher 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 timing of initiation of clinical trials for the treatment of nGD, the potential for PR001 to slow or stop disease progression in these patients and 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; 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:

Katie Engleman
1AB
katie@1ABmedia.com

Investor Contact:

investors@prevailtherapeutics.com

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Source: Prevail Therapeutics

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Prevail Therapeutics Announces FDA Orphan Drug Designation Granted to PR006 for the Treatment of Patients with Frontotemporal Dementia with a GRN Mutation

By michelle@orrbitt.com,

NEW YORK, Dec. 16, 2019 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) for the company’s gene therapy candidate, PR006, for the treatment of patients with frontotemporal dementia (FTD) with a GRN mutation (FTD-GRN). PR006 is designed to increase progranulin levels in FTD-GRN patients by delivering a healthy GRN gene using an AAV9 vector.

Orphan Drug designation is granted by the U.S. Food and Drug Administration to drugs or biologics intended to treat a rare disease or condition, which is a disease or condition that affects fewer than 200,000 individuals in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.

“FTD with a GRN mutation is characterized by progressive difficulties in decision-making, behavior and language,” said Asa Abeliovich, M.D., Ph.D., founder and chief executive officer of Prevail. “With no approved treatments for FTD, there is an urgent unmet need for therapies that slow or stop this disease. Orphan drug designation is an important milestone as we prepare to bring PR006 into the clinic in the first half of 2020.”

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. 50,000 to 60,000 people in the U.S. and 80,000 to 110,000 individuals in the European Union are affected by FTD. Frontotemporal dementia with a GRN mutation (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 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 and the ability to derive benefits from Orphan Drug designation. 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:

Katie Engleman
1AB
katie@1ABmedia.com

Investor Contact:

investors@prevailtherapeutics.com

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Source: Prevail Therapeutics

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Prevail Therapeutics Reports Third Quarter 2019 Financial Results and Recent Business Highlights

By michelle@orrbitt.com,

Opened Enrollment of PROPEL Phase 1/2 Trial of PR001 to Treat Parkinson’s Disease Patients with GBA1 Mutations

PR001 for Neuronopathic Gaucher Disease and PR006 for Frontotemporal Dementia with GRN Mutations Progressing Towards Clinic

Ongoing Collaboration with Lonza to Support the Manufacturing of our Pipeline of Novel AAV9-Based Gene Therapy Programs

NEW YORK, Nov. 12, 2019 (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 recent business highlights and third quarter 2019 financial results.

“Prevail made important progress throughout the third quarter in the advancement of our gene therapy programs for the treatment of neurodegenerative diseases for patients with urgent unmet medical needs. We activated the first sites for our PROPEL Phase 1/2 clinical trial of PR001 for patients with Parkinson’s disease with GBA1 mutations, and we expect to begin dosing patients in the fourth quarter. This is an exciting step forward for patients suffering from this devastating disease,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “In addition, we are working diligently to finalize the IND submissions for PR001 for patients with neuronopathic Gaucher disease and PR006 for frontotemporal dementia patients with GRN mutations. We expect to initiate those Phase 1/2 trials in the first half of next year. Finally, our collaboration with Lonza is advancing our process development and manufacturing capabilities as we prepare to supply our late-stage clinical trials.”

Recent Business Highlights

  • Actively Recruiting for PROPEL Phase 1/2 Trial of PR001 for PD-GBA: Prevail previously announced that the U.S. Food and Drug Administration (FDA) accepted the company’s Investigational New Drug (IND) application for its lead program, PR001 for the treatment of Parkinson’s disease patients with GBA1 mutations (PD-GBA). The PROPEL trial is now open for enrollment. The trial will enroll up to 16 patients to investigate the safety and tolerability of PR001 and will also measure key biomarkers and exploratory efficacy endpoints. The company expects to initiate patient dosing this year.
  • Planning to Initiate the Phase 1/2 Trial of PR001 for Neuronopathic Gaucher Disease Patients in 1H 2020: Prevail is also developing PR001 for the potential treatment of neuronopathic Gaucher disease (nGD). In September, the company announced that, based on feedback from the FDA and findings from its preclinical studies, it is modifying the design of the Phase 1/2 clinical trial in nGD to commence at a dose higher than originally proposed. Based on this feedback, the IND was placed on clinical hold pending FDA review of an amendment to the nGD IND, which will detail this modification. To support the higher dose, Prevail recently completed a non-human primate (NHP) safety study in which no PR001-related safety events or adverse findings were observed. Prevail expects to initiate a Phase 1/2 clinical trial for patients with nGD during the first half of 2020.
  • Phase 1/2 Trial of PR006 for Frontotemporal Dementia Patients with GRN Mutations (FTD-GRN) Expected to Initiate in 1H 2020: Prevail has completed the necessary preclinical efficacy and safety studies and is targeting submission of an IND for PR006 at the end of the year. In a mouse model of FTD-GRN, PR006 increased progranulin expression, reduced markers of neuroinflammation, and reduced measures of lysosomal pathology and no PR006-related safety events or adverse findings were observed. In a GLP NHP safety study, PR006 treatment increased progranulin levels in the brain in a dose-dependent manner. An extremely minor degree of nerve fiber degeneration in spinal cord and glial cellularity in dorsal root sensory ganglia was observed and was not considered adverse. The company expects to initiate its Phase 1/2 clinical trial for FTD-GRN during the first half of 2020. Prevail believes PR006 has the potential to be a first-in-class, disease-modifying treatment for patients with FTD-GRN.
  • Established Collaboration with Lonza to Enable Large Scale Gene Therapy Production: In October 2019, Prevail and Lonzaannounced the establishment of a collaboration focused on GMP manufacturing utilizing the baculovirus/Sf9 expression system for Prevail’s pipeline of novel AAV-based gene therapy programs for patients with neurodegenerative diseases. Under the terms of the agreement, Lonza will manufacture PR001 and PR006 at its gene therapy center of excellence in Houston, Texas. The collaboration also has the potential to extend to Prevail’s future pipeline of AAV-based gene therapy programs.
  • Continued Expansion of Internal Expertise and Capabilities: Prevail continued to build out its team of over 50 employees and moved into a new office space in the Alexandria Center for Life Science in New York City. The new location includes approximately 12,000 square feet of lab space to support early-stage research and in-house process and analytical development utilizing both HEK293 and baculovirus/Sf9 AAV expression systems.

Third Quarter 2019 Financial Results

  • Cash Position: Cash and cash equivalents were $183.1 million as of September 30, 2019, as compared to $202.1 million as of June 30, 2019.
  • R&D Expenses: R&D expenses were $16.8 million for the third quarter of 2019, compared to $4.6 million for the third quarter of 2018. The increase was primarily related to our development programs, as a result of increased manufacturing-related spend, clinical and preclinical activities, and headcount.
  • G&A Expenses: G&A expenses were $4.4 million for the third quarter of 2019, compared to $0.9 million for the third quarter of 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 $20.3 million, or $0.62 loss per share, for the third quarter of 2019, compared to $5.2 million, or $0.99 loss per share, for the third quarter of 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 genetically defined sub-populations of patients with neurodegenerative diseases. 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 of Prevail’s gene therapies to modify 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; Prevail’s ability to work with Lonza to supply Prevail’s late stage trials and commercial production; and the applicability of the collaboration to Prevail’s future pipeline of AAV-based gene therapy programs. 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 June 30, 2019, filed with the SEC on August 14, 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.

Prevail Therapeutics Inc.
Statements of Operations
(Unaudited)
(in thousands, except share and per share data)

Three Months Ended September 30,     Nine Months Ended September 30,  
    2019     2018     2019     2018  
Operating Expenses:
Research and development $ 16,836 $ 4,599 $ 37,202 $ 9,110
General and administrative 4,452 920 10,050 2,520
Total operating loss (21,288 ) (5,519 ) (47,252 ) (11,630 )
Change in fair value of derivative liabilities (781 )
Interest income 989 320 1,905 543
Interest expense (471 )
Total other income (expense), net 989 320 1,905 (709 )
Net loss $ (20,299 ) $ (5,199 ) $ (45,347 ) $ (12,339 )
Net loss per share, basic and diluted $ (0.62 ) $ (0.99 ) $ (1.68 ) $ (2.47 )
Weighted average shares outstanding, basic and diluted 32,864,156 5,244,585 26,950,854 4,989,604

Prevail Therapeutics Inc.
Balance Sheets
(Unaudited)
(in thousands, except share and per share data)

September 30,
2019
December 31,
2018
ASSETS
CURRENT ASSETS:
Cash and cash equivalents $ 183,074 $ 63,014
Prepaid expenses and other current assets 7,425 563
Total current assets 190,499 63,577
Property and equipment, net 2,527 678
Operating lease right-of-use assets 10,312 8,534
Restricted cash 91 91
TOTAL ASSETS $ 203,429 $ 72,880
LIABILITIES, REDEEMABLE CONVERTIBLE PREFERRED
  STOCK, AND STOCKHOLDERS’ EQUITY (DEFICIT)
CURRENT LIABILITIES:
Accounts payable $ 4,952 $ 1,241
Accrued expenses and other current liabilities 5,089 1,477
Operating lease liabilities 1,134 917
Total current liabilities 11,175 3,635
Long-term operating lease liabilities 10,226 7,952
TOTAL LIABILITIES 21,401 11,587
COMMITMENTS AND CONTINGENCIES (Note 13) REDEEMABLE
CONVERTIBLE PREFERRED STOCK
Series Seed preferred stock – $0.0001 par value, 0 and 6,480,000 shares
authorized, issued and outstanding as of September 30, 2019 and
December 31, 2018, respectively
3,524
Series A preferred stock – $0.0001 par value, 0 and 9,072,000 shares
authorized, 0 and 8,997,085 shares issued and outstanding as of
September 30, 2019 and December 31, 2018, respectively
76,186
STOCKHOLDERS’ EQUITY (DEFICIT)
Common stock – $0.0001 par value, 200,000,000 and 28,398,600
shares authorized as of September 30, 2019 and December 31, 2018,
respectively, 34,098,819 and 7,209,000 shares issued and outstanding
as of September 30, 2019 and December 31, 2018, respectively
3 1
Additional paid-in capital 248,286 2,496
Accumulated deficit (66,261 ) (20,914 )
Total stockholders’ equity (deficit) 182,028 (18,417 )
TOTAL LIABILITIES, REDEEMABLE CONVERTIBLE
PREFERRED STOCK, AND STOCKHOLDERS’ EQUITY
(DEFICIT)
$ 203,429 $ 72,880

Media Contact:

Katie Engleman
1AB
katie@1ABmedia.com

Investor Contact:

investors@prevailtherapeutics.com

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Source: Prevail Therapeutics

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Prevail Therapeutics and Lonza Establish Strategic Collaboration to Develop and Manufacture Prevail’s Novel AAV9 Gene Therapy Programs for Patients with Neurodegenerative Diseases

By michelle@orrbitt.com,

Strategic collaboration focuses on baculovirus/Sf9 expression system for Prevail’s pipeline of novel AAV9-based gene therapy programs for patients with neurodegenerative diseases

Companies to partner on process development, analytical development, and large-scale manufacturing for late-stage clinical and commercial supply

Process development and manufacturing to be located in Lonza’s cGMP facility in Houston, Texas

BASEL, Switzerland and NEW YORK, Oct. 08, 2019 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, and Lonza (SWX: LONN) today announced that the companies have entered into a strategic collaboration.

Lonza and Prevail have been working together since 2018, with an initial focus on process development, towards the GMP manufacturing of Prevail’s two lead programs, PR001 and PR006. Under this collaboration, focused on the baculovirus/Sf9 production system for gene therapies, Lonza will manufacture Prevail’s pipeline of novel AAV-based gene therapy programs for patients with neurodegenerative diseases at its gene therapy center of excellence in Houston, TX.

“Prevail is a brilliant trailblazer and it is a privilege to partner with them,” said Alberto Santagostino, SVP Head of Cell & Gene Technologies, Lonza Pharma & Biotech. “This strategic collaboration reflects both companies’ commitment to fueling the innovation of novel medicines. The partnership combines Prevail’s frontier science with Lonza’s operational expertise to support the development and commercialization of potentially life-changing treatments. Our cell and gene therapy center of excellence in Houston, TX, will support Prevail in their journey as they develop and potentially bring to market AAV-based gene therapies for patients suffering from debilitating neurodegenerative diseases.”

Prevail’s pipeline includes PR001, an AAV9-based gene therapy delivering GBA1, in development for Parkinson’s disease patients with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease patients (nGD). Prevail plans to initiate a Phase 1/2 clinical trial of PR001 in PD-GBA in 2019. The company is also developing PR006, an AAV9-based gene therapy delivering GRN, for frontotemporal dementia patients with a GRN mutation (FTD-GRN). Prevail anticipates PR006 will enter the clinic in 2020. The collaboration also has the potential to extend to Prevail’s future pipeline of AAV-based gene therapy programs.

“We are pleased to partner with Lonza, a leader in the manufacturing of AAV gene therapy vectors, to enable large-scale production of our novel gene therapies that we believe hold potential to halt the course of neurodegenerative diseases for patient populations with urgent unmet needs,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer, Prevail Therapeutics. “Lonza and Prevail will work together closely on process development and scaling up production of PR001, our gene therapy for Parkinson’s disease with GBA1 mutations and neuronopathic Gaucher disease, to supply late-stage clinical trials and for commercial production. In addition, we look forward to a collaborative relationship to support our PR006 gene therapy program for patients with frontotemporal dementia with GRN mutations, and for future gene therapy programs in our pipeline.”

Prevail has built in-house process and analytical development capabilities utilizing both HEK293 and baculovirus/Sf9 AAV expression systems. Under this collaboration, Prevail and Lonza will work closely together on process development, analytical development, and large-scale production using the baculovirus/Sf9 process for late-stage clinical and commercial supply at Lonza’s GMP facility in Houston.

About Lonza

Lonza is an integrated solutions provider that creates value along the Healthcare Continuum®. Through our Pharma Biotech & Nutrition segment and our Specialty Ingredients segment businesses, we harness science and technology to serve markets along this continuum. We focus on creating a healthy environment, promoting a healthier lifestyle and preventing illness through consumers’ preventive healthcare, as well as improving patient healthcare by supporting our customers to deliver innovative medicines that help treat or even cure severe diseases.

Patients and consumers benefit from our ability to transfer our pharma know-how to the healthcare, hygiene and fast-moving consumer goods environment and to the preservation and protection of the world where we live.

Founded in 1897 in the Swiss Alps, Lonza today is a well-respected global company with more than 100 sites and offices and approximately 15,500 full-time employees worldwide at the end of 2018. The company generated sales of CHF 5.5 billion in 2018 with a CORE EBITDA of CHF 1.5 billion. Further information can be found at www.lonza.com.

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. 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 of Prevail’s gene therapies to halt the course of neurodegenerative diseases; the anticipated timing of Prevail’s Phase 1/2 clinical trial of PR001 in PD-GBA and Prevail’s clinical trial of PR006; Prevail’s ability to work with Lonza to supply Prevail’s late stage trials and commercial production; and the applicability of the collaboration to Prevail’s future pipeline of AAV-based gene therapy programs. 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 June 30, 2019, filed with the SEC on August 14, 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.

Additional Information and Disclaimer

Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited (“SGX-ST”). Lonza Group Ltd is not subject to the SGX-ST’s continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

Media Contact:

Katie Engleman
1AB
katie@1ABmedia.com

Investor Contact:

investors@prevailtherapeutics.com

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Source: Prevail Therapeutics

  Category: Press Releases
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