napazimone (KL1333) / Abliva, KT&G Corp - LARVOL DELTA

Falcon-OLE: Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease (clinicaltrials.gov) - P2 | N=140 | Recruiting | Sponsor: Pharming Technologies B.V. | Not yet recruiting ➔ Recruiting

Enrollment open • Metabolic Disorders

Mitochondrial DNA A3243G variant-associated MELAS: Recent advances in clinical trials, therapeutic interventions, and disease-modifying strategies. (PubMed, Intractable Rare Dis Res) - P3 | "Key advances include: i) regulatory approval of high-dose taurine supplementation (9-12 g/day), which achieved complete prevention of stroke-like episodes in 60% of participants in a phase III trial; ii) phase IIb data (company-reported) indicating that sonlicromanol (KH176) showed signals of improvement in cognition, mood, and fatigue, supporting progression to a phase III registrational trial (KHENERFIN, NCT06451757); iii) ongoing trials of zagociguat and TTI-0102 targeting vascular dysfunction and oxidative stress; iv) KL1333, a novel NAD+ modulator, in phase II evaluation (FALCON trial); and v) promising preclinical gene-based approaches-including mitochondria-targeted TALENs, DdCBE base editors, and mitoARCUS nucleases-demonstrating heteroplasmy shifting in patient-derived cells and animal models, though direct clinical applicability to m.3243A>G MELAS requires further investigation. The therapeutic landscape for MELAS is evolving from symptomatic care..."

Journal • Review • Cardiovascular • CNS Disorders • Fatigue • Gene Therapies • Metabolic Disorders

KL1333 2025-104B: An open-label, single-arm extension study to evaluate the long-term safety, tolerability, and efficacy of KL1333 (napazimone) in patients with primary mitochondrial disease (clinicaltrialsregister.eu) - P1/2 | N=28 | Not yet recruiting | Sponsor: Pharming Technologies B.V.

New P1/2 trial • Metabolic Disorders

NQO1-AMPK axis regulates energy balance and metabolic health in diet-induced obesity (ECO 2026) - "In conclusion, KL1333, a novel activator of NQO1 represents a promising therapeutic drug as an anti- obesity drug to prevent obesity and related metabolic disorders"

Genetic Disorders • Inflammation • Obesity • AMPK • CD36 • NQO1 • PPARA • SCARB1 • SIRT1

Falcon-OLE: Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease (clinicaltrials.gov) - P2 | N=140 | Not yet recruiting | Sponsor: Pharming Technologies B.V.

New P2 trial • Metabolic Disorders

FALCON: Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease (clinicaltrials.gov) - P2 | N=180 | Recruiting | Sponsor: Abliva AB | Active, not recruiting ➔ Recruiting | Trial completion date: Dec 2025 ➔ Nov 2027 | Trial primary completion date: Oct 2025 ➔ Oct 2027

Enrollment open • Trial completion date • Trial primary completion date • Metabolic Disorders

Optimizing rare disorder trials: a phase 1a/1b randomized study of KL1333 in adults with mitochondrial disease. (PubMed, Brain) - "Importantly, the data from the trial support efficacy of KL1333 based on improvements in fatigue and functional strength and endurance. Furthermore, the study highlights the value in using phase 1 studies to capture data that helps optimize later phase efficacy trial design."

Journal • P1 data • Fatigue • Gastrointestinal Disorder • Metabolic Disorders • Rare Diseases

FALCON: Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease (clinicaltrials.gov) - P2 | N=180 | Active, not recruiting | Sponsor: Abliva AB | Recruiting ➔ Active, not recruiting

Enrollment closed • Metabolic Disorders

FALCON: Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease (clinicaltrials.gov) - P2 | N=180 | Recruiting | Sponsor: Abliva AB | Not yet recruiting ➔ Recruiting

Enrollment open • Metabolic Disorders

FALCON: Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease (clinicaltrials.gov) - P2 | N=180 | Not yet recruiting | Sponsor: Abliva AB

New P2 trial • Metabolic Disorders

Efficacy of KL1333 in adult patients with primary mitochondrial disease (clinicaltrialsregister.eu) - P3 | N=180 | Ongoing | Sponsor: Abliva AB

New P3 trial • Fatigue • Metabolic Disorders • Myositis

"BioStock: Celebrating IND approval Abliva eyes next step with KL1333 https://t.co/l5Bn2eCL5o" (,@CisionNews)