Category: Press Releases


Prevail Therapeutics Provides Program Update on PR001 in Parkinson’s Disease with GBA1 Mutations and Neuronopathic Gaucher Disease

By michelle@orrbitt.com,

NEW YORK, Sept. 10, 2019 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (NASDAQ: PRVL), a biotechnology company developing AAV-based gene therapies for patients with neurodegenerative disorders, today provided an update on the Company’s clinical development of PR001, a potentially disease-modifying, single-dose gene therapy in development for Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD).

PR001 for Treatment of PD-GBA

Prevail has an open Investigational New Drug Application (IND) for PR001 for the treatment of PD-GBA, the first indication the Company is pursuing. The Company is in the process of activating clinical sites for its Phase 1/2 clinical trial in PD-GBA and is on track to initiate patient dosing this year. Approximately 7%-10% of Parkinson’s disease patients harbor an underlying mutation in the GBA1 gene, and it is estimated that there are 90,000 PD-GBA patients in the U.S. alone. No therapies currently available have shown the ability to slow or stop the disease progression of PD-GBA. This randomized, double-blind, sham procedure-controlled, ascending dose Phase 1/2 clinical trial will enroll up to sixteen PD-GBA patients. Two escalating dose cohorts are planned. In each cohort, six patients will receive PR001, administered as a single injection, and two patients will receive a sham procedure as control. The trial will investigate the safety and tolerability of PR001 and will also measure key biomarkers and exploratory efficacy endpoints.

PR001 for Treatment of Pediatric nGD

Prevail is also developing PR001 for pediatric nGD, a second indication with urgent unmet need.  The Company submitted an IND to the U.S. Food and Drug Administration (FDA) for PR001 for the treatment of pediatric patients with nGD. Following discussions with the FDA, and based on preclinical studies that demonstrated increased efficacy at a higher dose, Prevail is modifying the design of the Phase 1/2 clinical trial in nGD to commence at a dose higher than originally proposed. Prevail’s IND for PR001 for the treatment of pediatric nGD has been placed on clinical hold pending FDA review of an amendment to the nGD IND, which will detail this modification. No safety or adverse events have been observed in any of Prevail’s studies of PR001. The start of the Phase 1/2 trial in nGD is anticipated to be delayed approximately one quarter and to begin enrollment in the first half of 2020. The modification to the nGD Phase 1/2 trial design is not anticipated to delay the overall timeline to trial completion.

“Prevail is developing PR001 for two indications, both with urgent unmet needs,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “We are excited to begin dosing patients in our Phase 1/2 clinical trial for PD-GBA this year and are dedicated to developing PR001 for pediatric nGD, the most progressive form of Gaucher disease, which involves neurological manifestations that cause severe morbidity and mortality. We believe PR001 has tremendous potential to slow or stop disease progression in patients with PD-GBA or nGD, who currently have no disease-modifying therapeutic options.”

About Prevail Therapeutics

Prevail Therapeutics 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. 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

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 likelihood of its interactions with the FDA to support Prevail’s clinical development plans; the anticipated timing of Prevail’s Phase 1/2 clinical trials of PR001 in each of its two indications; and the ability of PR001 to slow or stop disease progression in patients with PD-GBA or neuronopathic Gaucher disease. 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; PR001 or Prevail’s other gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; and the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change. 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.

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 Second Quarter 2019 Financial Results and Recent Business Highlights

By michelle@orrbitt.com,

ND Active for Phase 1/2 Trial of PR001 to Treat Parkinson’s Disease Patients with GBA1 Mutations

Successful $125.0 Million Initial Public Offering Completed to Support Program Advancement and Expansion

NEW YORK, Aug. 14, 2019 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (NASDAQ: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, today reported financial results for the second quarter ended June 30, 2019 and provided an update on recent business highlights.

“Since Prevail’s inception in 2017, we have made rapid progress toward our goal of bringing urgently needed therapies to patients with Parkinson’s disease and other neurodegenerative disorders. With the first IND active for our lead gene therapy program, PR001, in Parkinson’s disease with a GBA1 mutation, we are one step closer to our goal, and remain focused on initiating a Phase 1/2 clinical trial of PR001 before the end of this year,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “Our strong cash balance of $202 million following our $125 million IPO in June enables us to continue advancing PR001 and to initiate additional clinical trials, including for our second gene therapy candidate, PR006, for the treatment of frontotemporal dementia patients with a progranulin mutation.”

Recent Business Highlights

  • IND Active for PR001: In May 2019, Prevail’s Investigational New Drug Application (IND) for its lead gene therapy candidate, PR001, for the treatment of Parkinson’s disease patients with a GBA1 mutation (PD-GBA) was accepted by the U.S. Food and Drug Administration (FDA) and is now active. Clinical site activation is ongoing, with patient dosing on track for the fourth quarter of 2019.
  • Fast Track Designation Received for PR001: In July 2019, Prevail announced that the FDA granted Fast Track Designation for PR001 for the treatment of PD-GBA.
  • Preclinical Development of PR006 for Frontotemporal Dementia Patients with a GRN Mutation (FTD-GRN) advanced: Based on pre-IND regulatory interaction with the FDA in June 2019, Prevail intends to initiate its Phase 1/2 clinical trial in FTD-GRN patients in 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.
  • Leadership Team Strengthened and Board of Directors Expanded to Support Growth: In May 2019, Prevail announced the promotion of Yong Dai, Ph.D., to Chief Technology Officer. Dr. Dai will lead the Company’s process and analytical development and establish advanced manufacturing. In addition, the Company appointed Tim Adams, Chief Financial Officer of ObsEva, to its Board of Directors and as Audit Committee Chair, and expanded the role of Board member Francois Nader, M.D. to Non-Executive Chairman.
  • Successful Completion of Initial Public Offering: In July 2019, Prevail announced the closing of its initial public offering at a price to the public of $17.00 per share. The Company raised $125.0 million in aggregate gross proceeds to support the advancement of its three initial gene therapy programs and business operations.

Year to Date 2019 Financial Results

  • Cash Position: Cash and cash equivalents were $202.1 million as of June 30, 2019, as compared to $63.0 million as of December 31, 2018.
  • R&D Expenses: Research and development (R&D) expenses were $20.4 million for the six months ended June 30, 2019, compared to $4.5 million for the six months ended June 30, 2018. The increase was primarily due to a $11.2 million increase in external research and development expenses, a $3.2 million increase in personnel costs resulting from increased headcount, and a $1.5 million increase in REGENXBIO license fees.
  • G&A Expenses: General and administrative (G&A) expenses were $5.6 million for the six months ended June 30, 2019, compared to $1.6 million for the six months ended June 30, 2018. The increase was primarily due to a $1.8 million increase in personnel costs resulting from increased headcount, a $1.7 million increase in consulting and professional service fees and other expenses, and a $0.5 million increase in facility rent expense.
  • Net Loss: Net loss was $25.0 million, or $1.05 loss per share, for the six months ended June 30, 2019, compared to $7.1 million, or $0.42 loss per share, for the six months ended June 30, 2018.

Second Quarter 2019 Financial Results

  • Cash Position: Cash and cash equivalents were $202.1 million as of June 30, 2019, as compared to $100.3 million as of March 31, 2019.
  • R&D Expenses: R&D expenses were $12.0 million for the second quarter of 2019, compared to $3.2 million for the second quarter of 2018. The increase is primarily due to a $6.3 million increase in external research and development expenses, a $1.3 million increase in personnel costs resulting from increased headcount, and a $1.2 million increase in REGENXBIO license fee.
  • G&A Expenses: G&A expenses were $3.7 million for the second quarter of 2019, compared to $1.0 million for the second quarter of 2018. The increase is primarily due to a $1.4 million increase in personnel costs resulting from increased headcount, a $0.9 million increase in consulting and professional services fees and other expenses, and a $0.4 million increase in facility rent expense.
  • Net Loss: Net loss was $15.1 million, or $0.58 loss per share, for the second quarter of 2019, compared to $3.9 million, or $0.19 loss per share, for the second quarter of 2018.

About Prevail Therapeutics

Prevail Therapeutics 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. 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

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 ability of Prevail’s balance sheet to enable continued development of its product candidates; the anticipated use of proceeds from Prevail’s initial public offering; the likelihood of its interactions with the FDA to support Prevail’s clinical development plans; and the timing of initiation of Prevail’s Phase 1/2 clinical trials of PR001 and PR006. 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; PR001 or Prevail’s other gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; and the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change. 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 final prospectus for its initial public offering, filed with the SEC on June 20, 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
June 30,
    Six Months Ended
June 30,
 
2019     2018     2019     2018  
Operating Expenses:
Research and development $ 11,955 $ 3,207 $ 20,366 $ 4,511
General and administrative 3,713 959 5,598 1,600
Total operating loss (15,668 ) (4,166 ) (25,964 ) (6,111 )
Change in fair value of derivative liabilities (781 )
Interest income 565 222 916 222
Interest expense (471 )
Total other income (expense), net 565 222 916 (1,030 )
Net loss $ (15,103 ) $ (3,944 ) $ (25,048 ) $ (7,141 )
Net loss per share, basic and diluted $ (0.58 ) $ (0.19 ) $ (1.05 ) $ (0.42 )
Weighted average shares outstanding, basic and
diluted
26,212,356 20,326,716 23,945,198 16,970,543
 Prevail Therapeutics Inc.
Balance Sheets
(Unaudited)
(in thousands, except share and per share data)
June 30,
2019
December 31,
2018
ASSETS
CURRENT ASSETS:
Cash and cash equivalents $ 202,095 $ 63,014
Prepaid expenses and other current assets 4,253 563
Total current assets 206,348 63,577
Property and equipment, net 2,123 678
Operating lease right-of-use assets 7,516 8,534
Restricted cash 91 91
TOTAL ASSETS $ 216,078 $ 72,880
LIABILITIES, REDEEMABLE CONVERTIBLE PREFERRED
  STOCK, AND STOCKHOLDERS’ EQUITY (DEFICIT)
CURRENT LIABILITIES:
Accounts payable $ 2,003 $ 1,241
Accrued expenses and other current liabilities 4,555 1,477
Operating lease liabilities 1,101 917
Total current liabilities 7,659 3,635
Long-term operating lease liabilities 7,380 7,952
TOTAL LIABILITIES 15,039 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 June 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 June 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 June 30, 2019 and December 31, 2018,
respectively, 34,021,194 and 7,209,000 shares issued and outstanding as of
June 30, 2019 and December 31, 2018
3 1
Additional paid-in capital 246,998 2,496
Accumulated deficit (45,962 ) (20,914 )
Total stockholders’ equity (deficit) 201,039 (18,417 )
TOTAL LIABILITIES, REDEEMABLE CONVERTIBLE
  PREFERRED STOCK, AND STOCKHOLDERS’ EQUITY (DEFICIT)
$ 216,078 $ 72,880

Media Contact:

Katie Engleman
1AB
katie@1ABmedia.com

Investor Contact:

investors@prevailtherapeutics.com

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Prevail Therapeutics Receives U.S. FDA Fast Track Designation for PR001 for the Treatment of Parkinson’s Disease Patients with a GBA1 Mutation

By michelle@orrbitt.com,

Company Plans to Initiate Phase 1/2 Clinical Trial in the Second Half of 2019

NEW YORK, July 08, 2019 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. (Nasdaq: PRVL) (Prevail), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the Company’s lead gene therapy program, PR001, for the treatment of Parkinson’s disease patients with a GBA1 mutation (PD-GBA). PR001 is a potentially disease-modifying, single-dose, AAV9-based gene therapy being developed for the treatment of PD-GBA and neuronopathic Gaucher disease.

“We are pleased that the FDA has granted Fast Track Designation for PR001, which underscores the unmet need of patients with Parkinson’s disease with a GBA1 mutation, a chronic and progressive neurodegenerative disorder that comprises 7% to 10% of the total Parkinson’s disease population worldwide,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “With no treatments available that modify the progressive course or the underlying disease process of Parkinson’s disease, a potential disease-modifying therapy like PR001 could significantly transform the lives of patients with this disease.”

Following the FDA’s acceptance of Prevail’s Investigational New Drug (IND) application in June, the Company is on track to begin dosing patients in a Phase 1/2 clinical trial in the second half of 2019. The trial will investigate the safety and tolerability of PR001, and will also measure key biomarkers and exploratory efficacy endpoints, in patients with PD-GBA.

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 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. 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

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 PR001 to transform the lives of patients with Parkinson’s disease; the timing of initiation of Prevail’s Phase 1/2 clinical trial of PR001; and the potential advantages of FDA’s Fast track 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:  our novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; PR001 may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; and the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change. These and other risks are described more fully in Prevail’s filings with the Securities and Exchange Commission. 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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Prevail Therapeutics Announces IND Active for Phase 1/2 Trial of its Gene Therapy PR001 to Treat Parkinson’s Disease Patients with GBA1 Mutations

By Matt Dennis,

NEW YORK–(BUSINESS WIRE)–Prevail Therapeutics Inc. (Prevail), a gene therapy company developing AAV-based gene therapies for patients with neurodegenerative diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug (IND) application for its lead gene therapy program, PR001. PR001 is a potentially disease-modifying, single-dose, AAV9-based gene therapy being developed for the treatment of Parkinson’s disease patients with a GBA1 mutation (PD-GBA).

“We are pleased that the FDA has accepted the IND for our first program, which we believe has the potential to transform the lives of patients with Parkinson’s disease with a GBA1 mutation,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “At Prevail, our goal is to halt the progression of serious neurodegenerative diseases by applying precision medicine to the development of gene therapies. Our active IND brings us a step closer to achieving that goal, and we look forward to entering this new phase as a clinical-stage company.”

The active IND allows Prevail to initiate a Phase 1/2 clinical trial that will investigate the safety and tolerability of PR001, and will also measure key biomarkers and exploratory efficacy endpoints, in patients with PD-GBA. The Company plans to begin dosing patients in the trial this year.

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 Therapeutics 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. 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.

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The Silverstein Foundation, REGENXBIO, and OrbiMed Launch Prevail Therapeutics

By Matt Dennis,

NEW YORK–(BUSINESS WIRE)–The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO Inc. (“REGENXBIO”), and OrbiMed today announced the formation and launch of Prevail Therapeutics, Inc., (“Prevail”), a new company focused on discovering and developing novel biologic therapies for Parkinson’s Disease and other neurodegenerative diseases.

Parkinson’s Disease is a progressive neurodegenerative disorder that affects more than 10 million people worldwide1. Gene mapping in recent years has led to the identification of new genes and risk factors for Parkinson’s, including the glucocerebrosidase (GBA1) gene mutation. First observed in 2004, GBA1 is the most common of currently known Parkinson’s mutations, and it is estimated that up to 10 percent of people with Parkinson’s in the United States carry this mutation2. Recent discoveries around the link between the GBA1 gene, glucocerebrosidase (GCase) protein production, and accumulation of alpha-synuclein in the brain may have implications for the broader Parkinson’s population2, as well as in other neurodegenerative diseases. Prevail Therapeutics has assembled a world-class team to discover and develop novel disease-modifying treatments for patients.

In conjunction with its launch, Prevail has entered into an exclusive worldwide license agreement with REGENXBIO to develop and commercialize gene therapy products using REGENXBIO’s NAV AAV9 vector for the treatment of Parkinson’s Disease and other related neurodegenerative diseases.

“As a leader in gene therapy, we are pleased to collaborate with Prevail in the development of life-changing therapies for patients suffering from serious neurodegenerative diseases for which better treatment options are needed,” said Ken Mills, president and CEO of REGENXBIO. “We view the formation of Prevail as an important part of our mission to enable the use of the our NAV Technology Platform to develop successful new therapeutics for patients suffering from severe diseases with significant unmet need.”

The Company was co-founded by Asa Abeliovich, M.D., Ph.D., faculty at Columbia University and co-founder of Alector, who will lead Prevail as Chief Executive Officer. “Current treatments for Parkinson’s Disease are focused on alleviating symptoms,” Dr. Abeliovich said. “Prevail is focused on developing disease-modifying therapies that slow, stop, and reverse the neurodegenerative process.”

Jonathan Silverstein, partner and co-head of Private Equity at OrbiMed, added, “We are proud to partner with REGENXBIO and Dr. Abeliovich in founding Prevail. We believe REGENXBIO’s NAV Technology Platform provides us with a promising opportunity to develop potential life-changing therapies for patients suffering from Parkinson’s Disease and other neurodegenerative diseases. Dr. Abeliovich’s leadership and deep expertise in the molecular mechanisms of neurodegeneration uniquely position Prevail to make a significant mark in developing innovative biologic therapies for Parkinson’s Disease and other central nervous system diseases with huge unmet medical needs.”

About Prevail Therapeutics

Prevail Therapeutics is a biotechnology company focused on developing novel biologic therapies for Parkinson’s Disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

About The Silverstein Foundation for Parkinson’s with GBA

The Silverstein Foundation is a 501(c)(3) non-profit organization founded in 2017 focused on investing in cutting edge research with the goal of discovering new therapies for the treatment of Parkinson’s Disease in glucocerebrosidase (GBA1) mutation carriers. For more information, please visit www.silversteinfoundation.org.

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

About OrbiMed

OrbiMed is a leading investment firm dedicated exclusively to the healthcare sector, with over $14 billion in assets under management. OrbiMed invests globally across the spectrum of healthcare companies, from venture capital start-ups to large multinational companies utilizing a range of private equity funds, public equity funds, royalty/debt funds and other investment vehicles. OrbiMed maintains its headquarters in New York City, with additional offices in San Francisco, Shanghai, Mumbai and Herzliya. OrbiMed seeks to be a capital provider of choice, with the flexibility to provide equity and debt capital solutions that are tailored to the unique needs of our portfolio companies. The firm’s global team of over 80 professionals brings the resources and experience required to be an exceptional long-term partner in building world-class healthcare companies. www.OrbiMed.com.

1 “Statistics on Parkinson’s.” Parkinson’s Disease Foundation. Retrieved from http://www.pdf.org/parkinson_statistics

2 “GBA1 and Parkinson’s Disease.” The Michael J. Fox Foundation for Parkinson’s Research. Retrieved from https://www.michaeljfox.org/understanding-parkinsons/living-with-pd/topic.php?gba1-parkinsons

 

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