Prevail Therapeutics (PRVL) Up Over 75% In 3 Months, What's Ahead?

(RTTNews) - Shares of Prevail Therapeutics Inc. (PRVL) have gained more than 75% over the last three months, thanks to the progress on its pipeline of AAV-based gene therapies for patients with neurodegenerative disorders.

The Company's lead program is PR001 for the treatment of Parkinson's disease patients with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease.

-- A phase I/IIa clinical trial of PR001 for the treatment of PD-GBA patients, dubbed PROPEL, was started last October. This trial is designed to enroll up to 16 patients.

It is estimated that about 7 million people worldwide have Parkinson's disease, and in the U.S, about 1 million people are affected by this disease. About 7% to 10% of Parkinson's disease patients worldwide have GBA1 mutation.

-- A phase I/II trial of PR001 for neuronopathic Gaucher disease is expected to be initiated during the first half of 2020.

PR001 uses a modified, harmless version of an adeno-associated virus (AAV9) to deliver the functional GBA1 gene, which encodes glucocerebrosidase (GCase). Mutations in the GBA1 gene can increase the risk of Parkinson's and such mutations can also cause Gaucher disease.

Next up in the pipeline are:

-- PR006, a gene therapy candidate for patients with frontotemporal dementia with a GRN mutation (FTD-GRN). The Company expects to initiate a phase I/II clinical trial of PR006 for FTD-GRN during the first half of 2020.

GRN-related frontotemporal dementia is a progressive brain disorder that can affect behavior, language, and movement. The symptoms of this condition become evident in a person's fifties or sixties, and affected people usually live 6 to 7 years after the appearance of symptoms. (Source: Genetics Home Reference, NIH).

About 50,000 people in the U.S. and 80,000 to 110,000 people in the EU have FTD-GRN, with 5-10% of those patients having GRN mutation. There are no approved therapies for FTD-GRN.

-- PR004, a gene therapy, in preclinical development for patients with certain synucleinopathies.

Synucleinopathies are a group of rare diseases associated with abnormal accumulation of the protein a-synuclein in the nervous system and worsening neurological deficits. Patients with synucleinopathies and signs of CNS-deficits are frequently diagnosed with Parkinson's disease (PD), dementia with Lewy bodies (DLB) or multiple system atrophy (MSA).(Source: ClinicalTrials.gov)

Partnership:

In October 2019, Prevail and Lonza Group, a Swiss multinational, chemicals and biotechnology company, entered into a strategic collaboration. This collaboration focuses on baculovirus/Sf9 expression system for Prevail's pipeline of novel AAV9-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.

Balance Sheet:

The Company's 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. No revenues from product sales have been generated to date.

Cash and cash equivalents as of September 30, 2019, totaled $183.1 million.

Prevail Therapeutics went public on the Nasdaq Global Market on June 20, 2019, offering its shares at a price of $17 each.

The stock has thus far hit a low of $7.41 and a high of $18.28. The stock closed yesterday's (Jan.7, 2020) trading at $17.88, up 14.91%.