ADX-324 / ADARx - LARVOL DELTA

ADARx Pharmaceuticals Announces First Patient Dosed in Phase 3 STOP-HAE Clinical Trial and Orphan Drug Designation for ADX-324, a Long-Acting siRNA in Development for the Prophylactic Treatment of Hereditary Angioedema (HAE) (The Manila Times) - "The Phase 3 STOP-HAE clinical trial of ADX-324 is a randomized, double-blind, placebo-controlled study designed to evaluate the efficacy of ADX-324 in preventing HAE attacks in adults with Type I and Type II HAE.....Enrollment for this Phase 3 program follows positive Phase 1/2 data demonstrating that ADX-324 achieved significant and durable suppression of prekallikrein (PKK) protein levels with a favorable safety profile."

Orphan drug • Trial status • Hereditary Angioedema

STOP-HAE: A Phase 3 Study of ADX-324 in HAE (clinicaltrials.gov) - P3 | N=90 | Recruiting | Sponsor: ADARx Pharmaceuticals, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

AbbVie expands siRNA push with $335m ADARx deal (MSN News) - "AbbVie has signed a collaboration and licensing agreement with US-based ADARx Pharmaceuticals, committing $335m upfront to access its small interfering RNA (siRNA) platform...Under the deal, ADARx will apply its proprietary siRNA discovery technology to identify and develop new molecular candidates. In addition, AbbVie will contribute expertise in antibody engineering, antibody-drug conjugates (ADCs), and targeted tissue delivery – capabilities intended to enhance the therapeutic potential and precision of the siRNA candidates...ADARx’s internal pipeline includes ADX-324, a treatment for hereditary angioedema, which is nearing Phase III development."

Licensing / partnership • Hereditary Angioedema

STOP-HAE: Efficacy, Safety, PK, PD and HRQoL of 2 Dose Levels and Regimens of ADX-324 in Preventing HAE Attacks Compared With Placebo in Participants With Type 1 and Type II HAE (clinicaltrials.gov) - P3 | N=90 | Not yet recruiting | Sponsor: ADARx Pharmaceuticals, Inc.

New P3 trial • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

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