ADX-324 / ADARx - LARVOL DELTA

Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients (clinicaltrials.gov) - P1/2 | N=53 | Recruiting | Sponsor: ADARx Pharmaceuticals, Inc. | Phase classification: P1 ➔ P1/2

Phase classification • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients (clinicaltrials.gov) - P1 | N=53 | Recruiting | Sponsor: ADARx Pharmaceuticals, Inc. | Trial primary completion date: Jan 2025 ➔ Jul 2025

Trial primary completion date • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients (clinicaltrials.gov) - P1 | N=53 | Recruiting | Sponsor: ADARx Pharmaceuticals, Inc. | Trial completion date: Nov 2024 ➔ Dec 2025 | Trial primary completion date: Jan 2024 ➔ Jan 2025

Trial completion date • Trial primary completion date • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema. (PubMed, J Allergy Clin Immunol Pract) - "Donidalorsen is an investigational GalNAc-conjugated antisense oligonucleotide that binds to prekallikrein mRNA in the liver and reduces the expression of prekallikrein...ADX-324 is a GalNAc-conjugated siRNA being investigated in HAE...NTLA-2002 is an investigational in vivo clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9-based therapy designed to knock out the prekallikrein-coding KLKB1 gene in hepatocytes; a phase 1/2 study is ongoing. Findings from these and other ongoing studies are highly anticipated with the expectation of expanding the array of treatment options in HAE."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Gene Therapies • Hereditary Angioedema

HAE Prophylaxis by Targeting Prekallikrein With siRNA- Phase 1 Interim Safety Outcomes and Prekallikrein Levels (ACAAI 2023) - P1 | "Conclusion The interim outcomes provide preliminary evidence that ADX-324 is safe and significantly decreases PKK levels for at least 3 months. This should translate to effective prevention of HAE attacks with convenient long intervals between doses."

Clinical • P1 data • Pain

Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients (clinicaltrials.gov) - P1 | N=53 | Recruiting | Sponsor: ADARx Pharmaceuticals, Inc.

New P1 trial • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema