imatinib dry powder (AV-101) / Jade Biosciences - LARVOL DELTA

IMPAHCT: A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH) (clinicaltrials.gov) - P2/3 | N=202 | Terminated | Sponsor: Aerovate Therapeutics | N=462 ➔ 202 | Trial completion date: Jan 2025 ➔ Aug 2024 | Active, not recruiting ➔ Terminated | Trial primary completion date: Dec 2024 ➔ Aug 2024; The IMPAHCT study demonstrated that while inhaled imatinib was well tolerated, it did not prove to be efficacious at any of the doses of AV-101 evaluated in the study.

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Inhaled Imatinib Pulmonary Arterial Hypertension Clinical Trial - Follow Up Long Term Extension (IMPAHCT-FUL) (clinicaltrials.gov) - P3 | N=186 | Terminated | Sponsor: Aerovate Therapeutics | N=462 ➔ 186 | Trial completion date: Dec 2025 ➔ Aug 2024 | Active, not recruiting ➔ Terminated | Trial primary completion date: Dec 2025 ➔ Aug 2024; Results from the parent study (IMPAHCT) demonstrated that while inhaled imatinib was well tolerated, it did not prove to be efficacious at any of the doses of AV-101 evaluated in the study.

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Inhaled Imatinib Pulmonary Arterial Hypertension Clinical Trial - Follow Up Long Term Extension (IMPAHCT-FUL) (clinicaltrials.gov) - P3 | N=462 | Active, not recruiting | Sponsor: Aerovate Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

IMPAHCT: A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH) (clinicaltrials.gov) - P2/3 | N=462 | Active, not recruiting | Sponsor: Aerovate Therapeutics | Recruiting ➔ Active, not recruiting

Enrollment closed • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Baseline Characteristics From the IMPAHCT Trial of AV-101, Inhaled Imatinib, in Subjects With Pulmonary Arterial Hypertension (ATS 2024) - "This typical characteristic of study populations underscores the limitations of current therapies and the persistent unmet need for treating PAH. The IMPAHCT trial, which is currently enrolling into phase 3, will provide robust dose response information including clinical data on the safety and efficacy of AV-101 (imatinib) in PAH patients."

Clinical • Cardiovascular • Heart Failure • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

IMPAHCT: A randomized phase 2b/3 study of inhaled imatinib for pulmonary arterial hypertension. (PubMed, Pulm Circ) - P2/3 | "IMPAHCT has the potential to advance therapies for patients with pulmonary arterial hypertension by assessing the efficacy and safety of a novel investigational drug-device combination (AV-101) using an improved study design that has the potential to save 6-12 months of development time. ClinicalTrials.gov Identifier: NCT05036135."

Clinical • Journal • P2/3 data • P2b data • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Aerovate Therapeutics Announces Full-Year 2023 Financial Results and Business Highlights (GlobeNewswire) - "...'In 2024, we look forward to presenting baseline characteristics of IMPAHCT at the ATS international conference in May, as well as announcing Phase 2b topline data in June with the full Phase 2b clinical trial results to be presented at an upcoming scientific meeting'."

P2b data • Pulmonary Arterial Hypertension

IMPAHCT: A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH) (clinicaltrials.gov) - P2/3 | N=462 | Recruiting | Sponsor: Aerovate Therapeutics

Trial completion date • Trial primary completion date • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

IMPAHCT: A Study of AV-101 (Dry Powder Inhaled Imatinib) in Patients With Pulmonary Arterial Hypertension (PAH) (clinicaltrials.gov) - P2/3 | N=462 | Recruiting | Sponsor: Aerovate Therapeutics | Phase classification: P2b ➔ P2/3

Phase classification • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Designing a Successful Path for Imatinib in PAH (ATS 2023) - "Aerovate has developed AV-101, a powder imatinib for inhalation. Development of AV-101 has focused on i) an inhaled formation of imatinib that maximizes local versus systemic exposure, ii) delivering imatinib as an easy and convenient product to administer, iii) dose ranging – dose response investigation to identify an optimal AV-101 dose that is considerably lower than oral imatinib approved dosing for oncology indications, and iv) an efficient and adaptive phase 2b/3 clinical program."

Oncology

The Influence of Route and Formulation on the Pulmonary Pharmacokinetics of Imatinib in Nonclinical Species (ATS 2023) - "There was no evidence of significant lung contribution to metabolism.ConclusionsThe observations in nonclinical species demonstrate that pulmonary delivery of imatinib can increase lung exposure compared to systemic delivery and therefore potentially enhance the response to the widespread pulmonary vasculopathy observed in PAH. Furthermore, inhaled delivery of dry powder imatinib may achieve a significant reduction in human dose, and thereby systemic exposure, and may result in an improved therapeutic profile compared to oral delivery."

PK/PD data • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

AV-101, a novel inhaled dry-powder formulation of imatinib, in healthy adult participants: a phase 1 single and multiple ascending dose study. (PubMed, ERJ Open Res) - P2b | "AV-101 was well tolerated in healthy adults, and targeted doses of AV-101 significantly reduced the systemic exposure of imatinib compared with oral imatinib. An ongoing phase 2b/phase 3 study (IMPAHCT; clinicaltrials.gov identifier NCT05036135) will evaluate the safety/tolerability and clinical benefit of AV-101 for PAH."

Journal • P1 data • Cardiovascular • Cough • Hypertension • Pain • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial - Follow Up Long Term Extension (IMPAHCT-FUL) (clinicaltrials.gov) - P3 | N=462 | Recruiting | Sponsor: Aerovate Therapeutics | Phase classification: P2/3 ➔ P3

Phase classification • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial - Follow Up Long Term Extension (IMPAHCT-FUL) (clinicaltrials.gov) - P2/3 | N=462 | Recruiting | Sponsor: Aerovate Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Inhaled iMatinib Pulmonary Arterial Hypertension Clinical Trial - Follow Up Long Term Extension (IMPAHCT-FUL) (clinicaltrials.gov) - P2/3 | N=462 | Not yet recruiting | Sponsor: Aerovate Therapeutics

New P2/3 trial • Cardiovascular • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

A Phase 1 Single and Multiple Ascending Dose (SAD/MAD) Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of AV-101, a Novel Inhaled Dry Powder Formulation of Imatinib in Healthy Subjects (ATS 2022) - "The most common AEs following administration of AV-101 were cough in subjects (26.9%) and headache in 4 subjects (15.4%), mainly at the highest dose of 90 mg. There were no abnormalities in any other safety tests.ConclusionInhaled AV-101 was well tolerated and displayed low systemic exposure compared to oral imatinib."

Clinical • P1 data • PK/PD data • Cough • Pain • Respiratory Diseases