Inhaled AP01 slows lung function decline in IPF, progressive PF: Trial

Results of 'disease stabilization' consistent with ATLAS study

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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One-year of high-dose AP01, Avalyn Pharma’s inhaled pirfenidone solution, slowed lung function decline in people with idiopathic pulmonary fibrosis (IPF).

That’s according to top-line data from the ongoing open-label extension AP01-005 study (2020-005103-39) that’s testing a 100 mg high dose of AP01, administered twice daily.

The results align with a “trend toward disease stabilization consistent with what was observed in the ATLAS study at the same time point,” Avalyn stated in a press release.

The trial recruited 18 patients with progressive pulmonary fibrosis (PPF), a group with poor prognosis that lacks effective therapeutics.

After 48 weeks (nearly a year) of treatment, predicted forced vital capacity (FVC), a measure of lung capacity, declined by 0.9% in the PPF group and 2.2% in a newly enrolled IPF group compared to the start of the AP01-005 study.

AP01 was well tolerated, with the most frequent treatment-related side effects being cough, fatigue, and pain in the middle part of the throat.

Avalyn plans to present the findings from this study at the American Thoracic Society (ATS) annual meeting, in Washington, D.C., May 19-24.

“We are encouraged to see a consistency in AP01’s tolerability and efficacy profile in participants with IPF and PPF and look forward to opening future clinical studies for participants with both diagnoses,” said Lyn Baranowski, Avalyn’s CEO.

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Inhaled nintedanib formulation AP02 found safe in IPF patients

Stable lung function with AP01

Esbriet (pirfenidone) is an oral medication approved for IPF. While it helps slow the progression of lung fibrosis (scarring), it often requires larger doses since it’s taken by mouth. Real-world data suggest half of IPF patients are intolerant to the most effective dose and almost 30% are intolerant at any dose, according to Avalyn.

“Oral pirfenidone is not approved for patients with PPF,” Baranowski said. However, since its progression is similar to IPF, Avalyn expected “AP01 to show a treatment effect in both patient groups.”

AP01 is delivered with a nebulizer, letting more of it reach the lungs. This enables the delivery of a lower dose as a soft mist, which may reduce the risk of whole-body toxicity.

The Phase 1b ATLAS trial (ACTRN12618001838202), also known as AP01-002, tested the safety and effectiveness of AP01 at a low dose of 50 mg once daily, or a high dose of 100 mg twice daily in adults with IPF who were intolerant to or ineligible for Esbriet or Ofev (nintedanib).

From an initial 91 patients, 77 (85%) completed 24 weeks of treatment and all were recommended to switch to the higher dose for up to 72 weeks, after a review of the data by the trial’s s data safety monitoring board.

The results showed most patients treated with high-dose AP01 had relatively stable lung function at weeks 24 and 48, roughly six months and one year.

Of the 55 patients who reached week 72, 41 joined AP01-005 and continue to receive high-dose AP01. Another 59 adults with either IPF or PPF also were recruited. Eight of the 100 participants enrolled to date have been treated more than three years.

“Our ultimate goal in developing a pipeline of inhaled medications is to enable patients with life-threatening lung diseases to remain healthy longer, which can only be accomplished with long-term treatment. The tolerability profile of AP01 may allow patients with IPF and PPF to remain on medication longer than they can tolerate the oral systemic compound,” Baranowski said.