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AP01

Phase 2

Progressive Pulmonary Fibrosis | Small molecule | Respiratory |Avalyn Pharma Inc.|Last Updated: Aug 28, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment340

FDA Designations

No designations recorded

Clinical trial landscape

AP01 · 2 trials · 11 indications

Phase 2 2
NCT06951217An Extension Study of Subjects Who Received an Avalyn Inhaled Antifibrotic Agent (SAIL)Progressive Pulmonary Fibrosis
RECRUITING340 Analytics
NCT06329401A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With Progressive Pulmonary FibrosisPulmonary Fibrosis
ACTIVE NOT_RECRUITING375 Analytics
PHASE2RECRUITING
An Extension Study of Subjects Who Received an Avalyn Inhaled Antifibrotic Agent (SAIL)
Progressive Pulmonary FibrosisUnlock trial analytics
PHASE2ACTIVE NOT_RECRUITING
A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With Progressive Pulmonary Fibrosis
Pulmonary FibrosisUnlock trial analytics

Study Endpoints

Primary Endpoints

To evaluate the long-term safety and tolerability outcomes of subjects receiving Avalyn nebulized antifibrotic medications (AP01).
Through end of study; average of 6 years

The incidence of adverse events will be evaluated from the time of signing of the informed consent until approximately 2 weeks after the last dose of study drug. Examples of potential adverse events include: respiratory symptoms, liver toxicity, and skin conditions. Study participants will be monitored for adverse events via the following: vital signs (blood pressure assessed using a sphygmomanometer, temperature assessed by thermometer, pulse and respiratory rate assessed by pulse oximeter), oxygen saturation, spirometry, physical exams, blood chemistry, and quality of life assessments.

To evaluate the effect of AP01 high dose twice a day (BID) or AP01 low dose twice a day (BID) compared to placebo twice a day (BID)
Week 52

Change from baseline in forced vital capacity (FVC) (mL)

Secondary Endpoints

To evaluate the long-term effect of Avalyn nebulized antifibrotic medications on lung function the change from baseline in forced vital capacity (FVC) will be evaluated.
Through end of study; average of 6 years
To evaluate the long-term effect of Avalyn nebulized antifibrotic medications on stabilization of disease.
Through end of study; average of 6 years
To evaluate the long-term impact of Avalyn nebulized antifibrotic medications on subject-reported quality of life (QoL).
Through end of study; average of 6 years
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Open-label AP01 treatment armEXPERIMENTALAll trial participants will enroll from a Sponsor-led parent study of AP01 and receive open-label AP01 for continued evaluation of long-term safety and tolerability.
AP01 High Dose BIDEXPERIMENTALPirfenidone Solution for Inhalation
AP01 Low Dose BIDEXPERIMENTALPirfenidone Solution for Inhalation
Placebo BIDPLACEBO_COMPARATORPlacebo solution for inhalation

Interventions

NameTypeDescription
AP01COMBINATION_PRODUCTAP01 will be administered open-label via the eFlow Nebulizer System
PlaceboOTHERPlacebo oral inhalation solution
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites72

Inclusion Criteria: * Previously participated in an Avalyn-sponsored inhaled antifibrotic clinical study for subjects with either idiopathic pulmonary fibrosis (IPF) (IPF subjects are excluded from the US and Canada) or progressive pulmonary fibrosis (PPF) and with the approval of the Study Physici...

Countries:United StatesArgentinaAustraliaCanadaCzechiaFranceGermanyItalyNetherlandsNew ZealandPolandSpainUnited KingdomBelgiumTurkey (Türkiye)
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Recent Changes (Last 90 Days)

LOWAug 28, 2026NCT06951217lastUpdatePostDate: changed
LOWAug 28, 2026NCT06951217lastUpdatePostDate: changed
LOWAug 7, 2026NCT06951217lastUpdatePostDate: changed
LOWAug 7, 2026NCT06951217lastUpdatePostDate: changed
LOWJul 2, 2026NCT06951217lastUpdatePostDate: changed
LOWJul 2, 2026NCT06329401lastUpdatePostDate: changed
LOWJul 2, 2026NCT06951217lastUpdatePostDate: changed
LOWJul 2, 2026NCT06329401lastUpdatePostDate: changed
LOWJul 2, 2026NCT06951217lastUpdatePostDate: changed
LOWJul 2, 2026NCT06329401lastUpdatePostDate: changed
MEDIUMJun 18, 2026NCT06329401Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMJun 18, 2026NCT06329401Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMJun 18, 2026NCT06329401Status: RECRUITING → ACTIVE_NOT_RECRUITING

Frequently asked questions about AP01

What is AP01 used for in progressive pulmonary fibrosis?

AP01 is an investigational small molecule being developed for progressive pulmonary fibrosis and other forms of pulmonary fibrosis. It is administered by inhalation. AP01 is currently in Phase 2 clinical development and has not been approved by the FDA.

Who makes AP01?

AP01 is being developed by Avalyn Pharma Inc., a biopharmaceutical company. Avalyn Pharma is publicly traded under the ticker symbol AVLN.

What phase is AP01 in?

AP01 is currently in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Avalyn Pharma is evaluating the safety and efficacy of inhaled AP01 in participants with progressive pulmonary fibrosis.

What clinical trials is AP01 in?

AP01 is being studied in two Phase 2 clinical trials. The first trial, NCT06329401, is evaluating the safety and efficacy of inhaled AP01 in participants with progressive pulmonary fibrosis and is active but not recruiting. The second trial, NCT06951217, is an extension study for subjects who received an Avalyn inhaled antifibrotic agent and is currently recruiting.

Is AP01 the same as SAIL?

AP01 is not the same as SAIL. SAIL is the name of an extension study, NCT06951217, that enrolls subjects who previously received an Avalyn inhaled antifibrotic agent such as AP01. AP01 is the investigational drug being evaluated in that study.