MedPath

Avalyn's Inhaled Nintedanib Shows Promising Safety Profile in Phase 1 Trials for IPF Treatment

• Avalyn Pharma has successfully completed Phase 1 clinical trials of AP02, an inhaled formulation of nintedanib, demonstrating safety and favorable tolerability at all tested dose levels in healthy volunteers and IPF patients.

• The novel inhaled delivery approach aims to maximize therapeutic impact while minimizing systemic exposure, potentially addressing key limitations of current IPF treatments that are associated with significant toxicities.

• Based on these positive results, Avalyn plans to advance AP02 into Phase 2 clinical trials for idiopathic pulmonary fibrosis, with full data presentation scheduled for the American Thoracic Society 2025 International Conference in May.

Avalyn Pharma Inc. announced today that its novel inhaled formulation of nintedanib (AP02) demonstrated safety and favorable tolerability in Phase 1 clinical trials, positioning the therapy to advance into Phase 2 development for idiopathic pulmonary fibrosis (IPF).
The Cambridge, Massachusetts-based clinical-stage biopharmaceutical company reported positive topline data from both single-ascending dose (SAD) and multiple-ascending dose (MAD) studies conducted in healthy adult volunteers and patients with IPF.
"We are excited to complete this innovative set of clinical trials for AP02, where we assessed lung exposure in both studies of the inhaled compared to oral nintedanib," said Lyn Baranowski, Chief Executive Officer of Avalyn. "Through its enhanced drug delivery directly to the lungs, our approach allows us to address the underlying pathophysiology of the disease and positions AP02 as a potential future standard-of-care for patients with IPF."

Trial Design and Results

The most recently completed Phase 1 trial enrolled 60 healthy adult volunteers and was structured in three parts:
  • A single-ascending dose (SAD) portion evaluating AP02 at 2.0 mg, 4.0 mg, and 8.0 mg once-daily (QD)
  • A multi-ascending dose (MAD) portion assessing twice-daily (BID) dosing at the same dose levels over seven days
  • A bronchoalveolar lavage (BAL) component measuring lung exposure following a single 4.0 mg AP02 dose across three timepoints
The company reports that AP02 was safe and well-tolerated at all dose levels tested. Complete data on safety, tolerability, and pharmacokinetics from the two Phase 1 studies will be presented at the American Thoracic Society 2025 International Conference in May 2025.

Addressing Unmet Needs in IPF Treatment

IPF represents a significant unmet medical need, characterized by progressive scarring of lung tissue, declining lung function, reduced exercise capacity, and diminished quality of life. The condition is largely untreatable and associated with high mortality rates.
While current approved therapies can slow disease progression, they come with significant toxicities that often limit their use and dosing. Avalyn's approach with AP02 aims to overcome these limitations by delivering nintedanib directly to the lungs, potentially maximizing therapeutic impact while minimizing systemic exposure.
"There is a clear need to bring treatment options to patients that maximize therapeutic impact while minimizing systemic exposure and transform the disease from a fatal diagnosis into a manageable condition," noted the company in its announcement.

Pipeline and Development Strategy

AP02 represents Avalyn's second clinical program. The company's lead candidate, AP01, is an optimized inhaled formulation of pirfenidone currently being evaluated in the ongoing MIST Phase 2b study for progressive pulmonary fibrosis (PPF).
According to the company, AP01 has been assessed in over 150 individuals with various forms of pulmonary fibrosis and has demonstrated clinical proof-of-concept with improved efficacy and safety compared to historical data with existing therapies.
Based on the positive Phase 1 results for AP02, Avalyn plans to advance the inhaled nintedanib formulation into a Phase 2 clinical program specifically for IPF patients. This development represents a significant step forward in the company's mission to reimagine pulmonary fibrosis treatment through novel inhaled formulations of approved medicines.

The Potential Impact

If successful in later-stage trials, AP02 could potentially transform the treatment landscape for IPF by addressing the disease directly at its source while minimizing the systemic side effects that often limit patient adherence to current therapies.
The inhaled approach tackles the underlying pathophysiology of pulmonary fibrosis at its source and is designed to reduce systemic exposure while delivering medication directly to the site of disease. This targeted delivery system could potentially allow for more effective dosing regimens and improved patient outcomes.
As Avalyn prepares to present the full dataset at the upcoming American Thoracic Society conference, the pulmonary fibrosis community will be watching closely to see if this novel approach could indeed represent a significant advancement in the management of this challenging condition.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

Pharming Group Initiates Phase II Trial of Leniolisib for CVID with Immune Dysregulation

Pharming Group has dosed the first patient in a Phase II clinical trial evaluating leniolisib for common variable immunodeficiency (CVID) with immune dysregulation, representing a significant expansion beyond its approved APDS indication.
The multi-center trial will enroll approximately 20 patients aged 12 and older across sites in the US, UK, and EU, targeting a condition with an 11-fold higher mortality rate compared to CVID patients with infectious manifestations alone.

COSCIENS Biopharma Advances Oat-Derived Anti-Inflammatory Compound to Phase 2a Following Successful Safety Trial

COSCIENS Biopharma has successfully completed Phase 1 trials of its avenanthramide product with no significant adverse events reported in 72 healthy subjects receiving doses up to 960 mg daily.
The Phase 2a portion of the AvenActive study will enroll 20 patients with mild to moderate inflammation at the Montreal Heart Institute, with initial dosing expected on March 14, 2025.

AceLink's Oral GCS Inhibitor Shows Promising Phase 2 Results in Fabry Disease Treatment

AceLink Therapeutics' oral GCS inhibitor AL01211 demonstrated 50% reduction in GL3 levels at 30mg daily dose, with higher reductions observed at 60mg in Phase 2 trial for classic male Fabry disease patients.
Interim data revealed AL01211 is well-tolerated and shows positive trends in stabilizing disease symptoms, including eGFR and proteinuria levels, while improving pain and quality of life measures.

VYNE Therapeutics Advances VYN201 and VYN202 in Vitiligo and Psoriasis Trials

VYNE Therapeutics completes enrollment for Phase 2b trial of VYN201 gel (repibresib) in nonsegmental vitiligo, with top-line data expected mid-2025.
A Phase 1b trial of oral VYN202 for moderate-to-severe plaque psoriasis has begun, with results anticipated by the end of 2025.

Boehringer Ingelheim's Nerandomilast Shows Promise in Phase III Trials for Pulmonary Fibrosis

Boehringer Ingelheim's nerandomilast met its primary endpoint in the Phase III FIBRONEER-ILD trial, improving lung function in patients with progressive pulmonary fibrosis.
The company plans to submit a New Drug Application (NDA) to the FDA and other health authorities for nerandomilast in progressive pulmonary fibrosis.

AI-Discovered Drug INS018_055 Enters Phase 2 Trials for Idiopathic Pulmonary Fibrosis

INS018_055, an AI-discovered drug developed by Insilico Medicine, has advanced to Phase 2 clinical trials for Idiopathic Pulmonary Fibrosis (IPF).
The drug targets TNIK, an antifibrotic protein identified using Insilico's PandaOmics and Chemistry42 AI platforms, demonstrating safety and tolerability in Phase 1 trials.

Mirdametinib Shows Promise in Neurofibromatosis Type 1-Associated Plexiform Neurofibromas

Mirdametinib demonstrates significant objective response rates and a manageable safety profile in both adult and pediatric patients with NF1-PN.
The ReNeu trial highlights deep and durable tumor volume reductions, alongside early and sustained improvements in pain and quality of life.

Altimmune Achieves FDA Agreement on Phase 3 Program for Pemvidutide in Obesity Treatment

Altimmune has reached an agreement with the FDA on the design of its Phase 3 program for pemvidutide in treating obesity, marking a significant regulatory milestone.
The Phase 3 program will consist of four randomized, double-blind, placebo-controlled trials, involving approximately 5,000 subjects over a 60-week treatment period.

Infigratinib Receives FDA Breakthrough Therapy Designation for Achondroplasia

The FDA granted Breakthrough Therapy Designation to BridgeBio's infigratinib for treating children with achondroplasia, a genetic bone growth disorder.
Phase 2 PROPEL 2 trial data showed infigratinib led to a statistically significant increase in annualized height velocity (AHV) in children.

Alentis Therapeutics Reports Positive Topline Results from Phase 1 Multiple Ascending Dose Cohorts Study

Alentis Therapeutics announced positive results from the multiple-ascending dose part of its first-in-human Phase 1 clinical study of ALE.F02, targeting Claudin-1. The study confirmed ALE.F02's safety and tolerability across multiple dose levels, with no serious adverse events reported. The findings support the progression to further clinical trials in liver and renal studies.

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy