InterMune Initiates Phase 3 ASCEND Study of Pirfenidone in IPF

BRISBANE, Calif., July 7, 2011 /PRNewswire/ -- InterMune (NASDAQ: ITMN) today announced that patient enrollment has begun in ASCEND, a new Phase 3 study of pirfenidone for patients who suffer from idiopathic pulmonary fibrosis (IPF).  ASCEND is a multinational, randomized, double-blind, placebo controlled Phase 3 trial designed to evaluate the safety and efficacy of Esbriet® (pirfenidone) in IPF patients with mild to moderate impairment in lung function.  The primary endpoint is lung function, as measured by change in forced vital capacity (FVC) from baseline to Week 52.  The trial will enroll a total of approximately 500 patients who will be randomly assigned 1 to 1 to receive oral pirfenidone (2403 mg/day) or placebo.  Patients will be enrolled at centers in the United States, Mexico, South America, Australia and New Zealand.  

Steve Porter, M.D., Ph.D., Chief Medical Officer of InterMune, said, "We are very pleased to report enrollment of the first patient in ASCEND.  This study represents our continued commitment to making Esbriet available for the more than 100,000 IPF patients in the United States who are afflicted with IPF but currently have no FDA-approved therapies available to them."  

ASCEND Phase 3 Trial

InterMune's new Phase 3 study of pirfenidone is named ASCEND (Assessment of Pirfenidone to Confirm Efficacy and Safety in IPF).  ASCEND will add to pirfenidone's body of clinical evidence and support a future submission for FDA approval.

InterMune expects the trial to be fully enrolled by the first half of 2012, and that results from the study will be available in mid-2013.  InterMune anticipates a New Drug Application (NDA) resubmission for pirfenidone in the second half of 2013 and an FDA action in the first half of 2014.  

Relative to InterMune's previous studies of pirfenidone in IPF, the entry criteria for ASCEND have been refined to enrich the study population for patients who are more likely to experience decline in lung function and disease progression during the study.  InterMune believes that enrolling patients whose disease is more likely to progress will provide greater opportunity to demonstrate a treatment effect in a one-year study.  While the entry criteria have been refined, the study is conservatively powered based on the intent-to-treat analyses from the previous CAPACITY Phase 3 program.  

Patient eligibility criteria for ASCEND include the following:

• Centrally confirmed diagnosis by High Resolution Computed Tomography (HRCT) (+/- surgical lung biopsy)
• HRCT extent of fibrosis greater than emphysema
• %FVC  50% - 90%
• %DLco  30% - 90%
• FEV1/FVC ratio greater than 0.80
• Time since IPF diagnosis greater than six months and less than four years

The primary endpoint in the ASCEND study is change in percent predicted FVC, with the primary outcome analysis a Rank ANCOVA at Week 52.  The magnitude of effect will be presented on a categorical basis as the proportion of patients with decrements of less than 0% or greater than 10% at pre-specified study time points.

Key secondary endpoints include change in six-minute walk test (6MWT) distance and progression-free survival, which will be based on the earliest of time to death, FVC decrement of 10% or greater, or decrement in 6MWT distance of 50 meters or more.

Other secondary endpoints include all-cause mortality, evaluated both independently as well as pooled with the previous CAPACITY data, and on-treatment IPF-related deaths, which also will be evaluated independently and pooled with the CAPACITY data, and dyspnea.

About Pirfenidone

Pirfenidone is an orally active, small molecule drug that inhibits the synthesis of TGF-beta, a chemical mediator that controls many cell functions including proliferation and differentiation, and plays a key role in fibrosis.  It also inhibits the synthesis of TNF-alpha, a cytokine that is known to have an active role in inflammation.

On February 28, 2011, the European Commission (EC) granted marketing authorization for Esbriet® (pirfenidone) for the treatment of adults with mild to moderate IPF.  The approval authorized marketing of Esbriet in all 27 EU member states.  Esbriet has since been approved for marketing in Norway and Iceland.

Since 2008, pirfenidone has been marketed in Japan as Pirespa® by Shionogi & Co. Ltd. Pirfenidone is still under investigation for the treatment of IPF in the United States and has not been approved by the U.S. Food and Drug Administration for this use.  

About IPF

Idiopathic pulmonary fibrosis (IPF) is a progressive, debilitating and ultimately fatal disease that affects approximately 235,000 people in Europe and the United States combined.  IPF is characterized by inflammation and scarring (fibrosis) in the lungs, hindering the ability to process oxygen and is a progressive disease, meaning that over time, lung scarring and symptoms increase in severity.  The median survival time from diagnosis is two to five years, with a five-year survival rate of approximately 20 percent.  Patients diagnosed with IPF are usually between the ages of 40 and 70, with a median age of 63 years and the disease tends to affect slightly more men than women.

About InterMune

InterMune is a biotechnology company focused on the research, development and commercialization of innovative therapies in pulmonology and fibrotic diseases. In pulmonology, we are focused on therapies for the treatment of idiopathic pulmonary fibrosis (IPF), a progressive and fatal lung disease. Pirfenidone, the only medicine approved worldwide for IPF, is approved for marketing by InterMune in the EU as Esbriet® and is currently in a Phase 3 clinical trial in the United States. Pirfenidone is also approved for the treatment of IPF in Japan, where it is marketed by Shionogi & Co. Ltd. under the trade name Pirespa®. InterMune's research programs are focused on the discovery of targeted, small-molecule therapeutics and biomarkers to treat and monitor serious pulmonary and fibrotic diseases. For additional information about InterMune and its R&D pipeline, please visit www.intermune.com.

Forward-Looking Statements

This news release contains forward-looking statements within the meaning of section 21E of the Securities Exchange Act of 1934, as amended, that reflect InterMune's judgment and involve risks and uncertainties as of the date of this release, including without limitation the statements related to anticipated timing of ASCEND trial enrollment and NDA resubmission for FDA approval.  All forward-looking statements and other information included in this press release are based on information available to InterMune as of the date hereof, and InterMune assumes no obligation to update any such forward-looking statements or information.  InterMune's actual results could differ materially from those described in InterMune's forward-looking statements.  

Other factors that could cause or contribute to such differences include, but are not limited to, those discussed in detail under the heading "Risk Factors" in InterMune's most recent annual report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 9, 2011 (the "Form 10-K"), and other periodic reports filed with the SEC, including but not limited to the following: (i) risks related to the uncertain, lengthy and expensive clinical development process for the company's product candidates, including having no unexpected safety, toxicology, clinical or other issues and having no unexpected clinical trial results such as unexpected new clinical data and unexpected additional analysis of existing clinical data; (ii) risks related to the regulatory process for the company's product candidates, including the possibility that the results of the new 52-week Phase 3 clinical trial (ASCEND) having an FVC endpoint may not be satisfactory to the FDA for InterMune to receive regulatory approval for pirfenidone in the United States; (iii) risks related to unexpected regulatory actions or delays or government regulation generally; (iv) risks related to the company's manufacturing strategy, which relies on third-party manufacturers and which exposes InterMune to additional risks where it may lose potential revenue; (v) government, industry and general public pricing pressures; and (vi) InterMune's ability to obtain or maintain patent or other proprietary intellectual property protections.  The risks and other factors discussed above should be considered only in connection with the fully discussed risks and other factors discussed in detail in the Form 10-K and InterMune's other periodic reports filed with the SEC, all of which are available via InterMune's web site at www.intermune.com.    

Esbriet® is a registered trademark of InterMune, Inc.

SOURCE InterMune, Inc.