MedPath

Pirfenidone

Generic Name
Pirfenidone
Brand Names
Esbriet, Pirfenidone axunio (previously Pirfenidone AET), Pirfenidone Viatris
Drug Type
Small Molecule
Chemical Formula
C12H11NO
CAS Number
53179-13-8
Unique Ingredient Identifier
D7NLD2JX7U
Background

Pirfenidone is a synthetic pyridone drug. It is an antifibrotic agent with anti-inflammatory and antioxidant properties that is used to treat idiopathic pulmonary fibrosis (IPF), which is a chronic, progressive form of interstitial pneumonia. While its mechanism of action is not yet fully understood, pirfenidone is proposed to primarily regulate tumor necrosis factor (TNF) pathways and modulate cellular oxidation. The FDA first approved pirfenidone alongside nintedanib as one of the first drugs to treat IPF.

Indication

Pirfenidone is indicated for the treatment of idiopathic pulmonary fibrosis (IPF). In Canada and Europe, it is approved in adults only.

Associated Conditions
Idiopathic Pulmonary Fibrosis (IPF), Mild Idiopathic Pulmonary Fibrosis, Moderate Idiopathic Pulmonary Fibrosis

Leap Therapeutics Halves Workforce and Refocuses Cancer Drug Development Amid Market Challenges

• Leap Therapeutics has announced a significant restructuring, reducing its workforce by approximately 50% and narrowing the development focus of its lead cancer drug candidate in response to challenging market conditions. • The strategic pivot aims to extend the company's cash runway while concentrating resources on the most promising clinical applications of its lead oncology asset, potentially improving its chances for regulatory success. • This move follows similar restructuring trends across the biotech sector, with companies like Arcturus, NGM Bio, and Erasca all recently announcing staff reductions and pipeline reprioritizations to navigate the difficult funding environment.

NMPA Approves Gyre Therapeutics' Clinical Trial for Pirfenidone in Oncology-Related Lung Complications

• China's NMPA has approved Gyre Therapeutics' clinical trial application to evaluate pirfenidone for treating radiation-induced lung injury and immune-related pneumonitis in cancer patients. • The adaptive Phase 2/3 trial represents a significant expansion of pirfenidone beyond its established use in idiopathic pulmonary fibrosis into oncology supportive care. • Currently, no targeted therapies exist for lung injuries caused by radiation or immunotherapy, with 5-25% of radiation therapy patients and 13-19% of immunotherapy patients experiencing pulmonary complications.

Pliant Therapeutics Halts BEACON-IPF Trial Following Safety Board Review

• Pliant Therapeutics has temporarily suspended enrollment and dosing in its Phase IIb/III BEACON-IPF trial of Bexotegrast following recommendations from the independent Data Safety Monitoring Board. • The clinical trial pause impacts the study's original timeline, which aimed to complete Phase IIb enrollment of 360 idiopathic pulmonary fibrosis patients by Q1 2025. • The company's stock experienced significant decline, dropping 34.81% in regular trading and an additional 58% in after-hours trading to $3.27.

Gyre Therapeutics Anticipates Key Data Readouts and Product Launches in 2025

• Gyre Therapeutics expects topline results from its Phase 3 trial of F351 for Chronic Hepatitis B-associated liver fibrosis in China during Q1 2025. • The company plans to initiate a Phase 2 trial of F351 in MASH-associated liver fibrosis in the U.S. in 2025, contingent on positive Phase 3 results. • Gyre is also preparing to launch Avatrombopag maleate tablets for thrombocytopenia and Nintedanib for idiopathic pulmonary fibrosis in China in 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. • Nerandomilast, an oral PDE4B inhibitor, has also shown positive results in a separate Phase III trial for idiopathic pulmonary fibrosis (IPF), with an NDA already submitted. • The FIBRONEER-ILD trial included 1,178 patients across 40 countries, evaluating two doses of nerandomilast against placebo over 52 weeks.

Gyre Therapeutics Anticipates Key Data and Product Launches in 2025

• Gyre Therapeutics reported Q3 2024 revenues of $25.5 million, a decrease from $32.0 million in Q3 2023, alongside a net income of $2.9 million. • The company completed its Phase 3 trial for F351 in CHB-associated liver fibrosis, with topline data expected in the first quarter of 2025. • Commercial launches of avatrombopag and nintedanib in China are planned for 2025, expanding Gyre's product offerings. • A U.S. Phase 2 trial of F351 in MASH-associated liver fibrosis is slated to begin in 2025, pending Phase 3 results.

Novel Therapies Show Promise in Idiopathic Pulmonary Fibrosis Treatment

• Bridge Biotherapeutics' BBT-877, a first-in-class autotaxin inhibitor, is undergoing Phase 2 trials with data expected in April, showing promising safety and efficacy. • Daewoong Pharmaceutical's Versiporocin (DWN12088), targeting PRS protein action, is in Phase 2 trials and has received FDA and EMA rare drug designations, aiming for completion in 2025. • Ildong Pharmaceutical is developing IL1512, a CXCR7-selective agent, set to enter GLP toxicity testing, while Hanmi Pharmaceutical's HM15211 shows promise in preclinical studies for IPF. • The IPF treatment market is projected to grow from $4.6 billion to $7 billion by 2030, driven by unmet needs and the pursuit of first-in-class and best-in-class therapies.

Insilico Medicine's AI-Designed Drug Shows Promise in Phase IIa IPF Trial

• Insilico Medicine's ISM001-055 met its primary safety endpoint and secondary efficacy endpoints in a Phase IIa trial for idiopathic pulmonary fibrosis (IPF). • The AI-designed drug demonstrated a dose-dependent improvement in forced vital capacity (FVC), a key measure of lung function, in IPF patients. • The company plans to engage with regulatory authorities to design a Phase IIb study, exploring extended treatment durations and larger patient cohorts. • ISM001-055 targets TNIK, offering a novel approach to treating IPF by addressing both fibrosis and inflammation, potentially modifying the disease.

Nerandomilast Shows Promise in Phase 3 Trial for Idiopathic Pulmonary Fibrosis

• Boehringer Ingelheim's nerandomilast met its primary endpoint in the Phase 3 FIBRONEER-IPF trial, demonstrating improved lung function in IPF patients. • The trial is the first in a decade to meet its primary endpoint, offering hope for a new treatment option for this progressive disease. • Nerandomilast, a phosphodiesterase 4B inhibitor, aims to reduce inflammation and fibrosis in the lungs, addressing a high unmet need. • Full data will be presented in the first half of 2025, with plans for regulatory submissions to the FDA and other agencies.

Boehringer Ingelheim Seeks Approval for Nerandomilast After Positive Phase III IPF Trial

• Boehringer Ingelheim is seeking regulatory approval for nerandomilast after its Phase III FIBRONEER-IPF trial met the primary endpoint of improving forced vital capacity in patients with idiopathic pulmonary fibrosis (IPF). • The FIBRONEER-IPF trial, involving 1,177 patients, demonstrated that nerandomilast significantly improved lung function compared to placebo over 52 weeks; full data will be presented in H1 2025. • Nerandomilast, a preferential phosphodiesterase 4B (PDE4B) inhibitor, received breakthrough therapy designation from the FDA in 2022 and is also being investigated for progressive fibrosing interstitial lung diseases. • With limited FDA-approved treatments for IPF, nerandomilast represents a potential new option, as the trial is the first successful late-stage IPF trial in a decade.
© Copyright 2025. All Rights Reserved by MedPath