At Avalyn Pharma Inc., safety and tolerability of AP01 (inhaled pirfenidone), an experimental inhaled drug candidate in development for the treatment of pulmonary fibrosis, was presented at an oral presentation from the ongoing AP01-005 open-label extension (OLE) trial.
With the long-term efficacy and tolerability profile of AP01 therapy from the phase 1b ATLAS study, some of whom have been receiving this therapy for up to 3 years, a durable and consistent response was evident. This is great news as we look forward to the potential future treatment of pulmonary fibrosis with AP01.
At ATS 2023, findings reported from the OLE’s roll-over, naïve IPF and naïve PPF cohorts showed that AP01 had fewer reported adverse events than what was seen with oral pirfenidone – the most commonly reported AEs (nausea, rash, diarrhea) were experienced at a much lower frequency of less than 10% among the AP01-005 patients.
This was impressive given that the ATLAS participants were significantly older than those enrolled in the oral pirfenidone studies with few clinically meaningful differences seen in tolerability between the PPF & IPF participants. It comes as no surprise that AP01 is proving to be a safe and effective option for those with IPF and PPF who are intolerant of or ineligible for oral antifibrotic medication and have no other treatment options.
Felix Woodhead, MD PhD, Senior Medical Director at Avalyn, presented the results of an unique OLE study that allowed for PPF patients – who would not otherwise qualify for anti-fibrotic treatment – to benefit from the targeted inhaled delivery of AP01.
The results of the study showed the patienst could tolerate the treatment with a variety of background therapies. Moreover, the favorable safety and efficacy findings among the PPF patients and IPF participants further solidified the intention to continue the OLE study indefinitely for patients to maintain their treatment regimen.
At ATS 2023, Lyn Baranowski, CEO of Avalyn, participated in a dynamic company showcase during the Respiratory Innovation Summit (RIS). She presented Avalyn’s cutting-edge strategies for tackling life-threatening pulmonary diseases – much to the delight and anticipation of the audience.
Developing AP01 as an inhaled therapeutic provides pulmonary fibrosis patients with a potential disease-modifying medication with a long-term tolerability profile. This innovative formulation of pirfenidone may allow patients to live healthier, longer lives.
The long-term safety and efficacy data suggest that AP01 may offer a more durable safety and efficacy profile compared to oral medication. Tolerability of oral pirfenidone is suboptimal, with up to 50% discontinuation within one year due to associated side effects such as nausea, diarrhea, and vomiting. We remain optimistic that AP01 will provide a more bearable solution for those suffering from this often debilitating disease.
On May 22nd, Avalyn presented at the American Thoracic Society’s Mini Symposium to explore the emerging data on disease and symptom-based therapeutics for IPF patients. Along with their oral presentation, a unique e-poster entitled “Long-term Safety Data of Inhaled Pirfenidone (AP01) in Idiopathic Pulmonary Fibrosis (IPF) and Non-IPF Interstitial Lung Disease (ILD) Patients” was also showcased. After the end of the ATS conference, the e-poster will be made available on the Avalyn website.
About AP01 (Pirfenidone Solution for Inhalation)
Avalyn is committed to developing AP01, an inhaled aerosol formulation of pirfenidone, to maximize the benefit and minimize the toxicity associated with traditional oral delivery. Delivered using an optimized eFlow® Technology Nebulizer, a high efficiency vibrating membrane nebulizer similar in design and operation to PARI’s 510(k) cleared/FDA-approved eFlow-based nebulizers, AP01 has been shown to reduce fibroblast differentiation and ECM production. Avalyn plans to begin its next phase of clinical testing in 2023 with AP01 administered at 100 mg BID.
About Avalyn Pharma
At Avalyn, we are on a mission to create targeted therapeutics to treat rare respiratory diseases, including idiopathic pulmonary fibrosis and other interstitial lung diseases. These illnesses are characterized by scarring, decline in lung function, and a reduction in quality of life, which can lead to increased mortality. Approved therapeutic options exist, but unfortunately they have limitations when it comes to both dosage and potential side effects.
That’s why we are working hard to develop our inhaled therapeutic pipeline. AP01, our lead candidate, is an inhaled formulation of pirfenidone – and based on clinical trials with 91 people suffering from IPF, it holds the promise of improved efficacy and safety over existing treatments. To learn more, visit us at www.avalynpharma.com.