$31.45 +2.14 (+7.30%) As of 04:00 PM Eastern
We are a clinical-stage biopharmaceutical company pioneering inhaled therapies to transform the treatment paradigm of serious, rare respiratory diseases with significant unmet needs. Our approach is designed to address the limitations of current oral therapies by delivering optimized inhaled formulations of approved oral medicines directly to the lungs to enhance efficacy and minimize systemic exposure that contributes to side effects and treatment discontinuation. Our current pipeline is focused on treating pulmonary fibrosis, a life-threatening disease with a median survival of three to five years, which is a significantly shorter prognosis than that observed for many forms of cancer. Pulmonary fibrosis is characterized by scarring of the lungs, which can lead to a decline in lung function, progressive shortness of breath, reduced quality of life, and increased mortality risk. There are currently three approved oral antifibrotic therapies for pulmonary fibrosis: pirfenidone, or ESBRIET®; nintedanib, or OFEV®; and nerandomilast, or JASCAYD®. While these oral antifibrotics slow fibrosis progression, none stop progression entirely. In addition, their use is frequently limited by tolerability challenges associated with their systemic delivery. Due to their side effects, around 50% of patients who start treatment with available therapies discontinue within one year. Despite limited use, ESBRIET® and OFEV® generated more than $4.0 billion in gross global sales in 2024. Our most clinically advanced candidates, AP01 and AP02, are inhaled formulations of pirfenidone and nintedanib for the treatment of progressive pulmonary fibrosis, or PPF, and idiopathic pulmonary fibrosis, or IPF, respectively. We have completed ATLAS, a Phase 1b clinical trial of AP01 in patients with IPF. Patients from ATLAS transitioned into an ongoing open-label extension, or OLE, trial, along with compassionate-use cohorts of patients with IPF and PPF. We currently have over four and a half years of data demonstrating a favorable tolerability profile and preliminary data on clinical activity, including near-stabilization of lung function. AP01 is currently being evaluated in MIST, a global Phase 2b clinical trial for the treatment of PPF. We have also completed Phase 1 trials of AP02 in healthy adult volunteers and patients with IPF. AP02 is currently being evaluated in AURA, a global Phase 2 clinical trial for the treatment of IPF. We are also advancing AP03 into a Phase 1 clinical trial. AP03 is a fixed-dose combination of inhaled pirfenidone and nintedanib designed to deliver dual antifibrotic mechanisms with the potential for additive or synergistic benefit. We believe our inhaled therapies, if successfully developed and approved, may offer improved tolerability and efficacy for long-term treatment. These programs are intentionally designed, leveraging our deep expertise in rare respiratory diseases, advanced inhaled drug formulation, and our exclusive license to customize the delivery device. Our Approach to Transforming the Treatment Paradigm for Pulmonary Fibrosis Interstitial lung diseases, or ILDs, are a heterogeneous group of rare pulmonary disorders, comprising over 200 distinct conditions, many of which lack approved or effective treatments. ILDs are characterized by varying degrees of inflammation, fibrosis, or both. These conditions impair gas exchange and patients’ quality of life, or QoL, by causing chronic breathlessness, fatigue, coughing, and reduced ability to perform daily activities. Pulmonary fibrosis is a pathologic phenotype observed across a large subset of ILDs characterized by chronic injury, aberrant repair, progressive, typically irreversible, fibrosis, and stiffening of lung tissue. Although different ILDs have distinct underlying etiologies, pathophysiology, and clinical courses, many forms of ILD can ultimately result in pulmonary fibrosis. Once pulmonary fibrosis develops, disease progression may continue even if the underlying cause of ILD is treated or removed. ILDs represent a significant global health burden, affecting over 650,000 in the U.S. alone, of which approximately 300,000 have pulmonary fibrosis. The median survival of pulmonary fibrosis is three to five years, which is a significantly shorter prognosis than that observed for many forms of cancer. There are two primary clinical forms of pulmonary fibrosis: IPF and PPF. IPF is an ILD of unknown etiology, affecting approximately 120,000 people in the U.S. PPF, by contrast, refers to ILDs with a known etiological factor, such as autoimmune disease or exposure to allergens, where a subset of patients develop a progressive pulmonary fibrotic phenotype. PPF represents a more prevalent form of pulmonary fibrosis, affecting approximately 180,000 people in the U.S. --- Current standard of care oral antifibrotic therapies slow, but do not halt nor reverse, disease progression. As a result, current oral antifibrotic therapies expose patients to chronic systemic drug exposure without directly targeting the site of disease, contributing to significant limitations including safety concerns and tolerability issues. Although oral pirfenidone and nintedanib are approved for the treatment of pulmonary fibrosis, both agents are associated with high rates of gastrointestinal, or GI, and other treatment-limiting toxicities that frequently require dose reductions, treatment interruptions, or permanent discontinuations. As a result, only approximately 30% of patients with IPF are actively treated with antifibrotics and 50% of patients discontinue within one year of treatment, largely due to systemic adverse events, or AEs. We believe we may be able to address these limitations through the development of optimized inhaled formulations of these established oral antifibrotic agents. By delivering drugs directly to the lungs, our approach is designed to fundamentally improve the benefit-risk profile of antifibrotic therapy by achieving higher drug concentration at the site of disease while substantially reducing systemic exposure. We believe this lung-targeted delivery strategy enables sustained long-term treatment, addressing key limitations of existing systemic therapies and supporting better treatment adherence and long-term disease management for patients with pulmonary fibrosis. The change we aim to make in the treatment paradigm of pulmonary fibrosis and other ILDs parallels the decades-long evolution seen in the treatment of asthma and COPD. In those diseases, treatments advanced through successive innovations, moving from broad, systemic oral therapies to targeted inhaled treatments, and ultimately to combination inhalers. We see a similar opportunity in pulmonary fibrosis, where the field still relies on oral antifibrotics today. Our programs are designed to drive a similar evolution, first by shifting treatment toward inhaled, lung-targeted formulations of existing antifibrotics that aim to improve safety and efficacy. We aspire to deliver inhaled therapies that combine complementary mechanisms into a single device for even greater therapeutic impact. Importantly, most investigational therapies for pulmonary fibrosis, including those targeting novel biological pathways, are in development as potential add-on systemic treatments to existing oral antifibrotics. Given we are developing inhaled formulations of these background standard-of-care antifibrotics, we believe our product candidates could be used as standalone therapies or in combination with other approved medicines. The commercial performance of current oral treatments is limited by their suboptimal benefit-risk profile, highlighting a significant unmet medical need. This gap creates a substantial commercial opportunity for therapies like our product candidates, which aim to preserve lung function while offering a more favorable tolerability profile. Our principal executive offices are located in Boston, Massachusetts.
