Prevail Therapeutics Reports Third Quarter 2019 Financial Results and Recent Business Highlights
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
Ongoing Collaboration with
“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
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
FDAand 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 FDAreview 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
Lonzato 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, Lonzawill 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 Sciencein 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 millionas of September 30, 2019, as compared to $202.1 millionas of June 30, 2019.
- R&D Expenses: R&D expenses were
$16.8 millionfor the third quarter of 2019, compared to $4.6 millionfor 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 millionfor the third quarter of 2019, compared to $0.9 millionfor 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 $0.62loss per share, for the third quarter of 2019, compared to $5.2 million, or $0.99loss per share, for the third quarter of 2018.
Prevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for genetically defined sub-populations of patients with neurodegenerative diseases. Prevail was founded by Dr.
Forward-Looking Statements Related to Prevail
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning: the potential of Prevail’s gene therapies to modify course of neurodegenerative diseases; the anticipated timing of Prevail’s Phase 1/2 clinical trials of PR001 in PD-GBA and in nGD and Prevail’s clinical trial of PR006; Prevail’s ability to work with
Statements of Operations
(in thousands, except share and per share data)
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Research and development||$||16,836||$||4,599||$||37,202||$||9,110|
|General and administrative||4,452||920||10,050||2,520|
|Total operating loss||(21,288||)||(5,519||)||(47,252||)||(11,630||)|
|Change in fair value of derivative liabilities||—||—||—||(781||)|
|Total other income (expense), net||989||320||1,905||(709||)|
|Net loss per share, basic and diluted||$||(0.62||)||$||(0.99||)||$||(1.68||)||$||(2.47||)|
|Weighted average shares outstanding, basic and diluted||32,864,156||5,244,585||26,950,854||4,989,604|
(in thousands, except share and per share data)
|Cash and cash equivalents||$||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|
|LIABILITIES, REDEEMABLE CONVERTIBLE PREFERRED
STOCK, AND STOCKHOLDERS’ EQUITY (DEFICIT)
|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|
|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
|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
|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
|Additional paid-in capital||248,286||2,496|
|Total stockholders’ equity (deficit)||182,028||(18,417||)|
|TOTAL LIABILITIES, REDEEMABLE CONVERTIBLE
PREFERRED STOCK, AND STOCKHOLDERS’ EQUITY
Source: Prevail Therapeutics