Lilly Acquires Prevail Therapeutics-AAV9 Based Gene Therapy Company

   Lilly to acquire Prevail for $22.50 per share in cash (or an aggregate of approximately $880 million) payable at closing plus one non-tradable contingent value right ("CVR") worth up to $4.00 per share in cash (or an aggregate of approximately $160 million), for a total consideration of up to $26.50 per share in cash (or an aggregate of approximately $1.040 billion). The CVR is payable (subject to certain terms and conditions) upon the first regulatory approval of a product from Prevail's pipeline as set forth.

Highlights of Prevail Therapeutics Inc. 

- Biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases
- Developing therapies for Parkinson’s disease with GBA1 mutations (PD-GBA), neuronopathic Gaucher disease (nGD);  Frontotemporal dementia with GRN mutations (FTD-GRN), & certain synucleinopathies.
- PR001 Receives U.S. FDA Fast Track Designation for Neuronopathic Gaucher Disease
- Cash, cash equivalents, and investments of $114.3 million 
- Net loss of  $18.6 million

Stock Symbol: PRVL

Prevail Therapeutics is developing a pipeline of gene therapies to slow or stop the underlying disease process for patients with Parkinson’s disease and other neurodegenerative disorders. The company utilizes a precision medicine approach targeting patient populations with urgent unmet needs, where there are currently no available therapies.

Platform: Adeno-associated virus (AAV 9)-based gene therapies for the treatment of neurodegenerative diseases. 

Prevail Therapeutics Portfolio

Parkinson's Disease  with GBA1 mutations (PD-GBA)
Neuronopathic Gaucher Disease (nGD)
Frontotemporal dementia (FTD)


Parkinson's Disease  with GBA1 mutations (PD-GBA)
PR001 — a potentially disease-modifying, single-dose gene therapy designed to correct the GCase deficiency — for the treatment of PD-GBA. GBA1 encodes the lysosomal enzyme beta-glucocerebrosidase (GCase), which is needed for the disposal and recycling of glycolipids — a type of cellular lipid component that is known to accumulate with aging. 
Patient Dosing Continues in Phase 1/2 PROPEL Trial of PR001 for Parkinson’s disease with GBA1 mutations (PD-GBA).  The Company expects to provide the next biomarker and safety analysis on a subset of patients in the PROPEL trial by mid-2021.

Neuronopathic Gaucher Disease (nGD)
Gaucher disease is a lysosomal storage disorder caused by mutations in both copies of the GBA1 gene, which can have a wide range of effects on organs throughout the body. PR001 is targeted for the treatment of Type 2 and Type 3 Gaucher disease with GBA1 mutations.
Phase 1/2 PROVIDE Trial Expected to Initiate Enrollment in the Fourth Quarter of 2020. The Company currently anticipates it will provide the next update on PR001 biomarker and safety data for neuronopathic Gaucher disease (nGD) in 2021.

Frontotemporal Dementia with GRN Mutations (FTD-GRN)
Frontotemporal dementia (FTD) is the second most common cause of dementia in people under the age of 65 (after Alzheimer’s disease), affecting 50,000 to 60,000 patients in the U.S. and 80,000 to 110,000 in the European Union. PR006 is targeted for the treatment of Frontotemporal Dementia with GRN Mutations (FTD-GRN)
Phase 1/2 PROCLAIM Trial Expected to Initiate Enrollment in Fourth Quarter of 2020. The Company currently anticipates it will provide a biomarker and safety analysis on a subset of patients in the PROCLAIM trial in 2021.

Financial Highlights
  • Cash Position:  Cash, cash equivalents, and investments were $114.3 million as of September 30, 2020
  • Net Loss: Net loss was $18.6 million, or $0.55 loss per share, for the third quarter of 2020.

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