LY3884961 / Eli Lilly - LARVOL DELTA

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) (clinicaltrials.gov) - P1/2 | N=15 | Recruiting | Sponsor: Prevail Therapeutics | Trial completion date: Oct 2030 ➔ Nov 2031 | Trial primary completion date: Oct 2030 ➔ Nov 2031

Trial completion date • Trial primary completion date • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Type 1 Gaucher Disease • GBA • GBA1

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) (clinicaltrials.gov) - P1/2 | N=32 | Recruiting | Sponsor: Prevail Therapeutics | N=20 ➔ 32

Enrollment change • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

Phase 1/2 dose-finding study to evaluate systemic administration of an AAV9-based gene therapy for peripheral manifestations of Gaucher disease: The Proceed Trial (ESGCT 2024) - P1/2 | "Initial clinical and biomarker data suggest LY3884961 to be safe, well-tolerated, and has the potential to be a one-time treatment for Gaucher disease Type 1. Enrolment in the PROCEED trial is ongoing; an update will be provided."

Gene therapy • P1/2 data • Anemia • CNS Disorders • Gaucher Disease • Gene Therapies • Genetic Disorders • Hematological Disorders • Metabolic Disorders • Movement Disorders • Parkinson's Disease • Thrombocytopenia • Type 1 Gaucher Disease • GBA • GBA1

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) (clinicaltrials.gov) - P1/2 | N=7 | Active, not recruiting | Sponsor: Prevail Therapeutics | N=15 ➔ 7

Enrollment change • Gaucher Disease • Genetic Disorders • Metabolic Disorders • GBA • GBA1

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) (clinicaltrials.gov) - P1/2 | N=20 | Recruiting | Sponsor: Prevail Therapeutics | Trial completion date: Jun 2029 ➔ Dec 2030 | Trial primary completion date: Jun 2029 ➔ Dec 2030

Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) (clinicaltrials.gov) - P1/2 | N=15 | Active, not recruiting | Sponsor: Prevail Therapeutics | Trial completion date: Dec 2028 ➔ May 2028 | Trial primary completion date: Dec 2028 ➔ May 2028

Trial completion date • Trial primary completion date • Gaucher Disease • Genetic Disorders • Metabolic Disorders • GBA

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) (clinicaltrials.gov) - P1/2 | N=15 | Active, not recruiting | Sponsor: Prevail Therapeutics | Recruiting ➔ Active, not recruiting | Trial completion date: Sep 2028 ➔ Dec 2028 | Trial primary completion date: Sep 2028 ➔ Dec 2028

Enrollment closed • Trial completion date • Trial primary completion date • Gaucher Disease • Genetic Disorders • Metabolic Disorders • GBA

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) (clinicaltrials.gov) - P1/2 | N=20 | Recruiting | Sponsor: Prevail Therapeutics | Trial completion date: Apr 2028 ➔ Jun 2029 | Trial primary completion date: Apr 2028 ➔ Jun 2029

Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) (clinicaltrials.gov) - P1/2 | N=20 | Recruiting | Sponsor: Prevail Therapeutics | Active, not recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) (clinicaltrials.gov) - P1/2 | N=15 | Recruiting | Sponsor: Prevail Therapeutics | Not yet recruiting ➔ Recruiting | Initiation date: Aug 2022 ➔ Dec 2022

Enrollment open • Trial initiation date • Gaucher Disease • Genetic Disorders • Metabolic Disorders • GBA

A baculovirus-produced gene therapy vector LY3884961 (formerly PR001A) demonstrates efficacy in mouse models of Gaucher Disease and tolerability in NHPs (ESGCT 2022) - No abstract available

Preclinical • Gaucher Disease • Gene Therapies • Genetic Disorders • Metabolic Disorders

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) (clinicaltrials.gov) - P1/2 | N=15 | Not yet recruiting | Sponsor: Prevail Therapeutics

New P1/2 trial • Gaucher Disease • Genetic Disorders • Metabolic Disorders • GBA

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) (clinicaltrials.gov) - P1/2 | N=24 | Active, not recruiting | Sponsor: Prevail Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA

PR001 Gene Therapy Increased GCase Activity and Improved Gaucher Disease Type 1 Phenotypes in Animal Models (ASGCT 2022) - "Long-term studies evaluating the persistence of PR001, and its therapeutic effects revealed durability out to at least one-year post-administration. Overall, PR001 demonstrated long-lasting efficacy in two independent mouse models of GD supporting further development of PR001 for patients with GD1."

Preclinical • Gaucher Disease • Gene Therapies • Genetic Disorders • Immunology • Inflammation • Metabolic Disorders • Type 1 Gaucher Disease

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: Prevail Therapeutics | N=12 ➔ 24 | Trial completion date: Jun 2027 ➔ Apr 2028 | Trial primary completion date: Jun 2027 ➔ Apr 2028

Enrollment change • Trial completion date • Trial primary completion date • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA

Gene Therapy for Parkinson's Disease Associated with GBA1 Mutations. (PubMed, J Parkinsons Dis) - "Studies in mouse models indicate that PR001, a rAAV9 vector-based gene therapy designed to deliver a functional GBA1 gene to the brain, suggest that this therapeutic approach may slow or stop disease progression. PR001 is currently being evaluated in clinical trials with Parkinson's disease patients carrying GBA1 mutations."

Journal • Review • Alzheimer's Disease • CNS Disorders • Dementia • Gene Therapies • Immunology • Inflammation • Movement Disorders • Parkinson's Disease • GBA

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) (clinicaltrials.gov) - P1/2; N=15; Recruiting; Sponsor: Prevail Therapeutics; Trial completion date: Apr 2028 ➔ Sep 2028; Trial primary completion date: Apr 2028 ➔ Sep 2028

Clinical • Trial completion date • Trial primary completion date • Gaucher Disease • Genetic Disorders • Metabolic Disorders • GBA

[VIRTUAL] PR001 Gene Therapy Increased GCase Activity and Improved Gaucher Disease Type 1 Phenotypes in Animal Models (ASGCT 2021) - "Long-term studies evaluating the persistence of PR001 and its therapeutic effects are ongoing. Overall, PR001 demonstrated efficacy in two independent mouse models of GD supporting further development of PR001 for patients with GD1."

Preclinical • Gaucher Disease • Gene Therapies • Genetic Disorders • Immunology • Inflammation • Metabolic Disorders • Type 1 Gaucher Disease

Eli Lilly Snaps Up Gene Therapy Company Prevail Therapeutics in $1.04 Billion Deal (BioSpace) - "Shares of Prevail Therapeutics have skyrocketed more than 84% in premarket trading after Eli Lilly announced it was acquiring the gene therapy company in a deal valued at approximately $1.04 billion. Since the deal was announced, Prevail’s stock has nearly doubled to $23 per share, higher than the tender offer of $22.50."

M&A • Stock price • CNS Disorders • Frontotemporal Lobar Degeneration • Parkinson's Disease • Tauopathies And Synucleinopathies

Lilly Announces Agreement to Acquire Prevail Therapeutics (Prevail Therapeutics Press Release) - "Eli Lilly and Company (NYSE: LLY) and Prevail Therapeutics Inc. (NASDAQ: PRVL) today announced a definitive agreement for Lilly to acquire Prevail...The CVR is payable (subject to certain terms and conditions) upon the first regulatory approval of a product from Prevail's pipeline....Prevail's lead gene therapies in clinical development are PR001 for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD) and PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN). Prevail's preclinical pipeline includes PR004 for patients with specific synucleinopathies, as well as potential gene therapies for Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and other neurodegenerative disorders."

M&A • CNS Disorders • Frontotemporal Lobar Degeneration • Parkinson's Disease • Tauopathies And Synucleinopathies

".@LillyPad firmly steps into #GeneTherapy vs #neurodegenerative diseases, acquiring @PrevailTx for $1B Leads incl PR001 vs #GBA1-mutant #Parkinson’s & neuronopathic #Gaucher; PR006 vs #GRN-mutant #frontotemporal #dementia https://t.co/YiEJVqjf9J #neuroscience #FTD @TenBridge" (@DDNewsOnline)

Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Gene Therapies • Movement Disorders • Parkinson's Disease

Prevail Therapeutics Granted Composition of Matter Patent for Experimental Gene Therapy Program PR001 (GlobeNewswire) - "Prevail Therapeutics...today announced that the United States Patent and Trademark Office (USPTO) on November 17, 2020 issued a composition of matter patent, U.S. Patent No. 10,837,028, with claims directed to the AAV vector used in PR001, Prevail’s experimental gene therapy program for the treatment of Parkinson’s disease...The base patent term extends until October 3, 2038, excluding patent term extensions or coverage in additional related patent filings."

Patent • CNS Disorders • Parkinson's Disease

Prevail Therapeutics Reports Third Quarter 2020 Financial Results and Business Highlights (GlobeNewswire) - "Patient Dosing Continues in the Phase 1/2 PROPEL Trial of PR001 for Parkinson’s Disease with GBA1 Mutations...The Company expects to provide the next biomarker and safety analysis on a subset of patients in the PROPEL trial by mid-2021....Phase 1/2 PROCLAIM Trial Expected to Initiate Enrollment in Fourth Quarter of 2020: Initiation of patient enrollment remains on track for the fourth quarter of 2020 for the Phase 1/2 PROCLAIM clinical trial of PR006 for frontotemporal dementia with GRN mutations (FTD-GRN)....The Company currently anticipates it will provide a biomarker and safety analysis on a subset of patients in the PROCLAIM trial in 2021."

Biomarker • Enrollment status • P1/2 data • Trial status • CNS Disorders • Frontotemporal Lobar Degeneration • Parkinson's Disease

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) (clinicaltrials.gov) - P1/2; N=15; Recruiting; Sponsor: Prevail Therapeutics; Not yet recruiting ➔ Recruiting; Trial completion date: Dec 2027 ➔ Apr 2028; Initiation date: Jun 2020 ➔ Sep 2020; Trial primary completion date: Dec 2027 ➔ Apr 2028

Clinical • Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date • Gaucher Disease • Genetic Disorders • Metabolic Disorders • GBA

Prevail Therapeutics Reports Second Quarter 2020 Financial Results and Business Highlights (GlobeNewswire) - "...The company has received initial data from a Type 2 Gaucher disease patient treated with PR001....The Type 2 Gaucher disease patient demonstrated an increase in CSF GCase enzyme activity from an undetectable level at baseline to 1.0 μmol/L/d at month 1 and 4.7 μmol/L/d at month 4 following PR001 administration (adult normal range: 1.1 – 8.1 μmol/L/d)....Study startup activities are continuing for the PROVIDE Phase 1/2 clinical trial of PR001 for Type 2 Gaucher disease patients, and the Company expects to initiate enrollment in the second half of 2020....In addition, the initiation of the PROGRESS Phase 1/2 clinical trial of PR001 for Type 3 Gaucher disease will be postponed until additional clinical data from the PROPEL and PROVIDE trials is available to inform the clinical development strategy for this indication."

Clinical data • Enrollment status • Trial status • Gaucher Disease • Genetic Disorders