ARCT-032 / Arcturus Therap - LARVOL DELTA

Arcturus Therapeutics Announces First Quarter 2025 Financial Update and Pipeline Progress (Businesswire) - "Recent Corporate Highlights: Arcturus is advancing enrollment of adult CF participants in the open label Phase 2 multiple ascending dose CF study (NCT06747858) with daily inhaled treatments of ARCT-032 over a period of 28 days and expects to complete enrollment by year end. The Company expects to provide Phase 2 interim data from the first two cohorts in mid-2025."

Enrollment status • P2 data • Cystic Fibrosis

Arcturus Therapeutics Announces Initiation of Dosing in Phase 2 Multiple Ascending Dose Studies for Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency (Businesswire) - "Arcturus Therapeutics Holdings...announced that the first CF and OTC deficiency participants initiated dosing in December 2024, in the Company’s Phase 2 multiple ascending dose studies. Each participant in the Phase 2 CF study (NCT06747858) is expected to receive daily treatments of ARCT-032 over a period of 28 days. The first OTC deficient participant receiving 0.5 mg/kg ARCT-810 initiated dosing in December 2024 in the United States. Each participant is expected to receive five intravenous infusions administered over two months....Phase 2 interim data for both mRNA therapeutic programs on track for first half of 2025."

P2 data • Trial status • Cystic Fibrosis • Genetic Disorders

LunairCF: Safety, Tolerability and Efficacy Study of ARCT-032 in People with Cystic Fibrosis (clinicaltrials.gov) - P2 | N=12 | Recruiting | Sponsor: Arcturus Therapeutics, Inc.

New P2 trial • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Arcturus Therapeutics Announces Third Quarter 2024 Financial Update and Pipeline Progress (Businesswire) - "The Company remains on track to share ARCT-032 Phase 2 proof-of-concept (POC) interim data in 1H25."

P2 data • Cystic Fibrosis

ARCT-032-01: Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects and Adults with Cystic Fibrosis. (clinicaltrials.gov) - P1 | N=39 | Completed | Sponsor: Arcturus Therapeutics, Inc. | Recruiting ➔ Completed

Trial completion • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Safety and tolerability of inhaled LUNAR-CFTR mRNA (ARCT-032) demonstrated in a Phase 1 study (NACFC 2024) - "Single doses of ARCT-032 were generally safe and well tolerated in healthy adults. Further results from Parts 1 and 2 will be presented at the conference. Data from this study will support advancement of ARCT-032 to a multiple-dose study in PwCF."

Clinical • P1 data • Cough • Pain • Pulmonary Disease • Respiratory Diseases

Arcturus Therapeutics Receives Clearance of an Investigational New Drug Application to U.S. Food and Drug Administration for ARCT-032, an Investigational Inhaled mRNA Therapeutic to Treat Cystic Fibrosis (Businesswire) - "Arcturus Therapeutics Holdings...announced that the U.S. Food and Drug Administration (FDA) has issued a “Study May Proceed” notification for the Company’s Investigational New Drug (IND) application, ARCT-032, to treat cystic fibrosis (CF). FDA clearance of the ARCT-032 IND application enables the Company to initiate a Phase 2 multiple ascending dose study to evaluate the safety, tolerability and efficacy of ARCT-032 in people with Cystic Fibrosis."

IND • New P2 trial • Cystic Fibrosis

Arcturus Therapeutics Provides Updates for Ornithine Transcarbamylase (OTC) Deficiency and Cystic Fibrosis (CF) Programs (Businesswire) - "ARCT-032 (CF) Update: The Company plans to submit an IND application in the next 60 days for an ARCT-032 Phase 2 multiple ascending dose study designed to identify a safe and effective dose in Class I (null) and other CF participants who do not benefit from CFTR modulators. This study is supported by safety and tolerability data collected in healthy volunteers (N = 32) and the ongoing two-administration Phase 1b study....The Phase 1b study is ongoing with the seventh and final participant scheduled to be dosed soon. Of the seven total CF participants in Phase 1b, six are receiving CFTR modulator treatment while one subject has Class I mutations and therefore does not benefit from modulator therapy."

IND • Trial status • Cystic Fibrosis

Arcturus Therapeutics Receives Orphan Medicinal Product Designation from the European Commission (EC), for ARCT-032, for the Treatment of Cystic Fibrosis (Businesswire) - "Arcturus Therapeutics Holdings...announced that the European Commission (EC), based on a positive opinion issued by the European Medicines Agency (EMA), has granted orphan medicinal product designation for the Company’s product candidate ARCT-032 to treat Cystic Fibrosis (CF)....After successful completion of the single-ascending-dose portion of the study in healthy adults, the first participant with CF in the Phase 1b study part enrolled and completed two administrations of ARCT-032. Arcturus remains on track to share interim Phase 1b data in H1 2024."

Orphan drug • P1 data • Trial status • Cystic Fibrosis

Arcturus Therapeutics Presents New Clinical Data at 47th Annual European Cystic Fibrosis Conference (Businesswire) - P1 | N=38 | NCT05712538 | Sponsor: Arcturus Therapeutics, Inc. | "Arcturus Therapeutics Holdings...today presented Phase 1 results in healthy volunteers and Phase 1b interim data in people with CF for ARCT-032, an inhaled investigational mRNA therapeutic, at the 47th European Cystic Fibrosis Conference in Glasgow, Scotland....The Phase 1b trial showed improvements in FEV1 (Forced Expiratory Volume in 1 second) in the four adults with CF after two inhaled administrations. The absolute change in percent predicted FEV1 averaged +4.0% on Day 8 (5 days after 2nd dose). The relative change in FEV1 averaged +5.8% on Day 8."

P1 data • Cystic Fibrosis

Inhaled LUNAR ® -CFTR mRNA (ARCT-032) is safe and well-tolerated: A Phase 1 Study (ECFS 2024) - "In vitro and animal studies demonstrated that LUNAR-CFTR effectively transfects airway epithelial cells and restores functional CFTR activity... Single doses of ARCT-032 were generally safe and well-tolerated in healthy adults. Unblinded safety data will be presented at the conference, as well as preliminary data from Part 2 CF participants."

P1 data • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Arcturus Therapeutics Announces Positive Development for Cystic Fibrosis Program (Businesswire) - 'Arcturus Therapeutics...today announced Arcturus will be presenting Phase 1 results in healthy volunteers and new Phase 1b interim data in CF patients for ARCT-032, an inhaled investigational mRNA therapeutic to treat CF, at the 47th European Cystic Fibrosis Conference on June 7, 2024. 'We were pleased to observe ARCT-032 treatments for the first four patients were generally safe and well tolerated with no serious adverse events,'...."

P1 data • Cystic Fibrosis

Arcturus Therapeutics Announces First Quarter 2024 Financial Update and Pipeline Progress (Businesswire) - "Arcturus is advancing ARCT-032, an inhaled mRNA therapeutic for cystic fibrosis. The Company remains on track to share Phase 1b interim data on July 1, 2024. Each CF patient in this trial receives two inhaled administrations of ARCT-032."

P1 data • Cystic Fibrosis

Arcturus Therapeutics Announces Fourth Quarter and Fiscal Year 2023 Financial Update and Pipeline Progress (Businesswire) - "ARCT-032 remains on track for Phase 1b interim data in Q2 2024."

P1 data • Cystic Fibrosis

Arcturus Therapeutics Receives Orphan Drug Designation from the U.S. FDA for ARCT-032, for the Treatment of Cystic Fibrosis (Businesswire) - "Arcturus Therapeutics Holdings Inc...announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the Company’s product candidate ARCT-032 to treat cystic fibrosis (CF)....The first CF patient in our Phase 1b study successfully completed two administrations of ARCT-032. We remain on track to share interim Phase 1b data in H1 2024."

Orphan drug • P1 data • Cystic Fibrosis • Genetic Disorders

Arcturus Therapeutics Announces Third Quarter 2023 Financial Update and Pipeline Progress (Businesswire) - "Phase 1b enrollment initiated October 2023, with dosing of first participant scheduled this month. The study is designed to enroll up to 8 adults with cystic fibrosis, with each participant receiving two administrations of ARCT-032....ARCT-032 received Rare Pediatric Disease Designation from the FDA. As such, if ARCT-032 achieves FDA approval for a pediatric indication, Arcturus is eligible to receive a priority review voucher of a subsequent marketing application for a different product."

FDA event • Trial status • Cystic Fibrosis

LUNAR®-CFTR mRNA Improves Mucociliary Clearance in CF Ferrets (NACFC 2023) - "The CF animals displayed lung disease resembling early CF after cessation of VX-770 treatment for longer than 1 month. This study demonstrated that the LUNAR platform is a robust vehicle for therapeutic mRNA delivery into airway epithelial cells in vivo and that LUNAR-CFTR can correct the CF-associated MCC defect in ferrets. Furthermore, CF ferrets with the ROSA26mT/mG transgenic background are a robust tool to evaluate the efficiency of gene transfer and gene editing in mucus-laden airways."

Gene Therapies • Genetic Disorders • Pulmonary Disease • Respiratory Diseases • CFTR

LUNAR®-CFTR mRNA replacement therapy restores CFTR expression and function in human bronchial epithelial cells (NACFC 2023) - "The stimulated Isc measured in the absence of a Cl gradient was approximately 4 times as great in LUNAR-CFTR-treated cells as in LUNAR-TdT-treated cells and comparable with elexacaftor-tezacaftor-ivacaftor (ETI)-corrected Isc, which remained at approximately 50% of wt hBECs, although in the presence of Cl gradient, the mean Isc driven by LUNAR-CFTR was comparable with that in wt cells under similar conditions. LUNAR-CFTR restored CFTR expression and CFTR-dependent Cl transport in differentiated primary F508del CF hBECs in the presence or absence of a Cl gradient. Absolute current magnitude after LUNAR-CFTR treatment was comparable with that in wt controls in the presence of a Cl gradient and equivalent to ETI in its absence. Immunohistochemistry characterization is in progress to evaluate CFTR-transfected cell popula-tions contributing to Cl transport in CF hBECs."

Pulmonary Disease • Respiratory Diseases • CFTR

Arcturus Therapeutics and Cystic Fibrosis Foundation Extend Agreement to Advance ARCT-032, an Investigational Messenger RNA (mRNA) Therapeutic to Treat Cystic Fibrosis (Businesswire) - "Arcturus Therapeutics...announced today that Arcturus and the Cystic Fibrosis Foundation (CF Foundation) have extended their ongoing agreement. The CF Foundation agreed to increase its financial commitment to ~$25 million to advance ARCT-032, a novel messenger RNA (mRNA) therapeutic candidate formulated with Arcturus’ LUNAR® delivery technology....'The resources and valuable expertise provided by the CF Foundation will support the continued clinical development of ARCT-032, including the completion of a Phase 1b study that we plan to initiate imminently in adults living with cystic fibrosis.'"

Licensing / partnership • Cystic Fibrosis • Genetic Disorders

Arcturus Therapeutics Announces Second Quarter 2023 Financial Update and Pipeline Progress (Businesswire) - "ARCT-032, the Company’s inhaled mRNA therapeutic for cystic fibrosis, has completed dosing in a Phase 1 study in New Zealand, including 32 subjects across four (4) ascending single-dose cohorts. The Company received regulatory approval of a protocol amendment to allow the transition to a Phase 1b clinical study of ARCT-032 in up to 8 adult cystic fibrosis patients."

Clinical protocol • Trial status • Cystic Fibrosis • Genetic Disorders

Arcturus Therapeutics Announces First Quarter 2023 Financial Update and Pipeline Progress (Businesswire) - "ARCT-032...has achieved the recruitment target and completed administration in a Phase 1 single ascending dosing study with 32 healthy participants (8 subjects per cohort)....Arcturus is amending the protocol to allow the dosing of patients with CF and expects to initiate the enrollment in Q3 2023."

Enrollment status • Cystic Fibrosis • Genetic Disorders

ARCT-032: Data from P1 LUNAR-CF trial (NCT05712538) for cystic fibrosis in 2023 (Arcturus Therap) - Corporate Presentation

P1 data • Cystic Fibrosis

Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects (clinicaltrials.gov) - P1 | N=32 | Recruiting | Sponsor: Arcturus Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Arcturus Therapeutics Announces Fourth Quarter 2022 Financial Update and Pipeline Progress (Businesswire) - "ARCT-032, the Company’s inhaled mRNA therapeutic for cystic fibrosis, has advanced to a Phase 1 study, presently enrolling up to 32 participants in New Zealand. The primary objective of this study is to assess safety and tolerability at four (4) dose levels. The first two cohorts have been dosed successfully with no significant adverse events reported to date. The Company expects to complete the full 32 subject study enrollment in Q2 2023."

Enrollment status • Cystic Fibrosis • Genetic Disorders

Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects (clinicaltrials.gov) - P1 | N=32 | Not yet recruiting | Sponsor: Arcturus Therapeutics, Inc.

New P1 trial • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases