DF-003 / Drug Farm - LARVOL DELTA

Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) | Trial completion date: May 2026 ➔ Nov 2026 | Trial primary completion date: Feb 2026 ➔ Jul 2026

Trial completion date • Trial primary completion date

Evaluating the Safety, Tolerability, and Pharmacokinetics of DF-003 in Healthy Subjects (clinicaltrials.gov) - P1 | N=96 | Active, not recruiting | Sponsor: Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) | Trial completion date: Jan 2025 ➔ Oct 2026 | Trial primary completion date: Mar 2024 ➔ Oct 2026

First-in-human • Trial completion date • Trial primary completion date

Preliminary immunologic and clinical Phase 1b study outcomes from patients with ROSAH syndrome treated with the first-in-class ALPK1 inhibitor DF-003 (IMMUNOLOGY 2026) - P1 | "Inhibition of ALPK1 with DF-003 demonstrated early evidence of clinical and pharmacodynamic improvements without safety concerns. These results warrant evaluation of DF-003 in a Phase 2 trial."

Clinical • P1 data • Inflammation • Musculoskeletal Pain • Ocular Inflammation • CCL2 • CRP • CXCL10 • CXCL8 • IL1B • IL6 • TNFA

Drug Farm Announces U.S. FDA Orphan Drug Designation for DF-003 for the Treatment of ROSAH Syndrome (Businesswire) - "DF-003 is a first-in-class ALPK1 inhibitor designed to correct the genetic root cause of ROSAH syndrome by inhibiting disease-causing ALPK1 mutations. Preclinical data and early clinical observations support its potential to reduce systemic inflammation associated with ROSAH syndrome."

Orphan drug • Inflammation • Inherited Retinal Dystrophy • Rare Diseases

Drug Farm Announces FDA Fast Track Designation for First-in-Class ALPK1 Inhibitor DF-003 for the Treatment of ROSAH Syndrome (Businesswire) - "DF-003 is the first ALPK1 inhibitor to successfully complete a first-in-human Phase 1 study and has now entered a Phase 1b clinical trial in patients with ROSAH syndrome (NCT06395285), where it is being evaluated for safety, pharmacokinetics, pharmacodynamics and efficacy. Clinical data from the trial will be reported at meetings in 2026."

Fast track • P1 data • Inflammation • Retinal Disorders

Discovery of a selective alpha-kinase 1 inhibitor for the rare genetic disease ROSAH syndrome. (PubMed, Nat Commun) - "In cell-based assays, DF-003 suppresses inflammatory cytokine signaling mediated both by wild-type ALPK1 and the disease-causing ALPK1[T237M] mutant. Using mice heterozygous for wild-type human ALPK1 and ALPK1T237M established to model ROSAH syndrome that exhibit retinal microglial infiltration, astrocyte activation, and inflammatory cytokine upregulation in the retina, optic nerve, and cortex, we show that orally administered DF-003 is sufficient to inhibit these inflammatory phenotypes."

Journal • Genetic Disorders • Inherited Retinal Dystrophy • Pain • TRAF6

Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) | Trial completion date: Sep 2026 ➔ May 2026 | Trial primary completion date: Jun 2026 ➔ Feb 2026

Trial completion date • Trial primary completion date

Evaluate DF-003 in ex Vivo Assays Using Peripheral Blood Mononuclear Cell From Subjects With ROSAH Syndrome (clinicaltrials.gov) - P=N/A | N=4 | Recruiting | Sponsor: Hospices Civils de Lyon | Trial completion date: Jan 2025 ➔ May 2028 | Trial primary completion date: Jan 2025 ➔ May 2028

Trial completion date • Trial primary completion date • Inherited Retinal Dystrophy • Pain

Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) | Not yet recruiting ➔ Recruiting | Trial completion date: Nov 2025 ➔ Sep 2026 | Initiation date: Sep 2024 ➔ Apr 2025 | Trial primary completion date: Aug 2025 ➔ Jun 2026

Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date

Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) | Initiation date: May 2024 ➔ Sep 2024

Trial initiation date • CCL2 • CRP • CXCL10 • CXCL9 • IL10 • IL18 • IL2RA • IL6 • TNFA

Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) | N=36 ➔ 12

Enrollment change • CCL2 • IFNB1 • IL17A • IL18 • IL1B • IL6

Evaluating the Safety and Tolerability of Multiple Doses of Orally Administered DF-003 in ROSAH Syndrome Patients (clinicaltrials.gov) - P1 | N=36 | Not yet recruiting | Sponsor: Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)

New P1 trial • CCL2 • IFNB1 • IL17A • IL18 • IL1B • IL6

Evaluating the Safety, Tolerability, and Pharmacokinetics of DF-003 in Healthy Subjects (clinicaltrials.gov) - P1 | N=96 | Active, not recruiting | Sponsor: Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm) | Recruiting ➔ Active, not recruiting

Enrollment closed

Evaluate DF-003 in ex Vivo Assays Using Peripheral Blood Mononuclear Cell From Subjects With ROSAH Syndrome (clinicaltrials.gov) - P=N/A | N=4 | Recruiting | Sponsor: Hospices Civils de Lyon | Trial completion date: Jan 2024 ➔ Jan 2025 | Trial primary completion date: Jan 2024 ➔ Jan 2025

Trial completion date • Trial primary completion date • Inherited Retinal Dystrophy • Pain • TNFA

Evaluating the Safety, Tolerability, and Pharmacokinetics of DF-003 in Healthy Subjects (clinicaltrials.gov) - P1 | N=96 | Recruiting | Sponsor: Zhejiang Yao Yuan Biotechnology Ltd. (also known as Drug Farm)

New P1 trial