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:
Full Year 2019 Financial Results
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.
Prevail Therapeutics Inc.
Statements of Operations
(in thousands, except share and per share data)
Year ended December 31, 2019 | Year ended December 31, 2018 | |||||||
Operating Expenses: | ||||||||
Research and development | $ | 48,798 | $ | 14,127 | ||||
General and administrative | 17,005 | 4,682 | ||||||
Operating loss | (65,803 | ) | (18,809 | ) | ||||
Change in fair value of derivative liabilities | — | (781 | ) | |||||
Other income | — | 87 | ||||||
Interest income | 2,615 | 887 | ||||||
Interest expense | — | (471 | ) | |||||
Total other income (expense), net | 2,615 | (278 | ) | |||||
Net loss | $ | (63,188 | ) | $ | (19,087 | ) | ||
Net loss per share | ||||||||
Basic and diluted | $ | (2.22 | ) | $ | (3.71 | ) | ||
Weighted average shares outstanding: | ||||||||
Basic and diluted | 28,494,950 | 5,145,469 |
Prevail Therapeutics Inc.
Balance Sheets
(in thousands, except share and per share data)
December 31, 2019 | December 31, 2018 | |||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 168,051 | $ | 63,014 | ||||
Prepaid expenses and other current assets | 6,410 | 563 | ||||||
Total current assets | 174,461 | 63,577 | ||||||
Property and equipment, net | 2,549 | 678 | ||||||
Operating lease right-of-use assets | 10,001 | 8,534 | ||||||
Restricted cash | 91 | 91 | ||||||
Total assets | $ | 187,102 | $ | 72,880 | ||||
Liabilities, redeemable convertible preferred stock, and stockholders' equity (deficit) | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 5,162 | $ | 1,241 | ||||
Accrued expenses and other current liabilities | 5,330 | 1,477 | ||||||
Operating lease liabilities | 1,341 | 917 | ||||||
Total current liabilities | 11,833 | 3,635 | ||||||
Long-term operating lease liabilities | 9,927 | 7,952 | ||||||
Total liabilities | 21,760 | 11,587 | ||||||
Commitments and contingencies (see Note 15) redeemable convertible preferred stock | ||||||||
Series Seed Preferred Stock - $0.0001 par value, 0 and 6,480,000 shares authorized, issued and outstanding as of December 31, 2019 and 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 December 31, 2019 and 2018, respectively | — | 76,186 | ||||||
Stockholders' equity (deficit) | ||||||||
Common stock - $0.0001 par value, 200,000,000 and 28,398,600 shares authorized as of December 31, 2019 and 2018, respectively, 34,098,819 and 7,209,000 shares issued and outstanding as of December 31, 2019 and 2018, respectively | 3 | 1 | ||||||
Additional paid-in capital | 249,441 | 2,496 | ||||||
Accumulated deficit | (84,102 | ) | (20,914 | ) | ||||
Total stockholders’ equity (deficit) | 165,342 | (18,417 | ) | |||||
Total liabilities, redeemable convertible preferred stock, and stockholders' equity (deficit) | 187,102 | $ | 72,880 |
Media Contact:
Mary Carmichael
Ten Bridge Communications
mary@tenbridgecommunications.com
Investor Contact:
investors@prevailtherapeutics.com