France's Transparency Commission (CT) Issues Favorable Opinion for Reimbursement of Esbriet® (pirfenidone)
-- InterMune expresses confidence in ASCEND Phase 3 study outcome; updates timeline --
-- Company to conduct conference call and webcast at 4:30 p.m. EDT today --
BRISBANE, Calif., April 5, 2012 /PRNewswire/ -- InterMune, Inc. (NASDAQ: ITMN) today reported that the Transparency Commission (CT: Commission de Transparence) of the French National Health Authority (HAS: Haute Autorite de Sante) has issued a favorable opinion for the reimbursement of Esbriet® (pirfenidone) by French National Health Insurance.
The CT noted that no other treatment provided evidence of a clinical benefit in IPF and considering all available information, Esbriet was granted an Amelioration du Service Medical Rendu (ASMR) rating of level IV. ASMR is a rating of added clinical value in comparison with existing therapies. The CT focused on the risk/benefit ratio for assessing the actual medical benefit (Service Medical Rendu – SMR), and rated it as "Low." Patients suffering with severe, chronic diseases in France are typically reimbursed 100% for all services and medicines associated with the management of their condition, regardless of SMR level other than "Insufficient," (under a specific program for such diseases (Affection de Longue Duree – ALD). This program covers more than 8.3 million French citizens.
The CT has recommended that Esbriet be reimbursed as a "medicament d'exception", which means that it will be reimbursed only for the labeled indication, further defined by the CT as IPF patients with forced vital capacity (FVC) > 50% and DLco > 35%. The CT also recommended that InterMune collect information in the form of a patient registry regarding the use of Esbriet in clinical practice. InterMune will now enter the next step in the process, which is to determine the price and any reimbursement conditions of Esbriet in France with the CEPS (Comite Economique des Produits de Sante).
Dan Welch, Chairman, Chief Executive Officer and President of InterMune, said, "We are pleased to receive the CT's favorable opinion for reimbursement of Esbriet. In the recently more challenging market access environment in France, our rating of ASMR IV is a positive outcome for Esbriet. The SMR and ASMR ratings we secured for Esbriet allow us to advance to the next step in the French process and we are optimistic about successfully concluding negotiations with the CEPS and making Esbriet available to patients in France as soon as possible and practical thereafter."
Based on information published on the HAS website, since early 2011 the CT did not recommend reimbursement in 32% of all its new product appraisals, compared with an historic average of 4%.
ASCEND Phase 3 Trial
InterMune provided an update on patient enrollment in the company's Phase 3 "ASCEND" study, which is designed to confirm the efficacy and safety of pirfenidone and to support an NDA re-submission for FDA approval of Esbriet (pirfenidone) in the United States. The company now expects ASCEND to be fully enrolled around the end of 2012. InterMune's previous guidance was the second quarter of 2012.
Steve Porter, M.D., Ph.D., Chief Medical Officer for InterMune, said, "Our primary mission for ASCEND is to deliver a high-quality result, which we aim to achieve by concentrating on a select number of high-quality sites. While a number of factors have contributed to the extended timeline, we are firmly convinced that the trade-off between data quality and time is a very good one. We believe the trial design and refined entry criteria of ASCEND significantly increase our probability of a successful outcome because they provide that patients enrolled in the study have the characteristics that we know from our many studies in IPF predict disease progression. We are extremely pleased with the continued enthusiasm of IPF physicians for ASCEND and look forward to sharing the results at the end of the study."
InterMune noted that it will discuss the status and projected timing for completion of the ASCEND Phase 3 clinical trial of Esbriet during today's conference call and webcast.
About Esbriet® (pirfenidone) in France
Esbriet is the first-ever drug approved by the EU regulatory authorities in adults for the treatment of mild-to-moderate idiopathic pulmonary fibrosis (IPF). In its assessment, the CT cited 7,700 mild-to-moderate IPF patients in France. The CT is the agency in France responsible for assessing medicinal products and advising the health authorities on whether those products provide sufficient benefit to be covered by French National Health Insurance.
About SMR and ASMR
Under the French system, a new medicine may be granted one of four SMR levels. Of these, three are considered "sufficient" for the CT to recommend favorable inclusion on the reimbursed drug list. The three sufficient SMR ratings are: Important, Moderate and Low. Each rating is associated with different levels of reimbursement.
The fourth SMR level, "Insufficient," is a recommendation by the CT that is unfavorable to inclusion on the reimbursed drug list and if assigned to an application, no ASMR rating is given by the CT. In this case, the manufacturer does not proceed to price negotiations with the Pricing Committee (CEPS).
There are five levels of ASMR rating:
ASMR I: Major therapeutic advance
ASMR II: Important improvement as far as effectiveness and/or reduction of side effects are concerned
ASMR III: Moderate improvement as far as effectiveness and/or reduction of side effects are concerned
ASMR IV: Minor improvement as far as effectiveness and/or clinical or pharmacokinetics usefulness are concerned
ASMR V: Absence of improvement but favorable opinion for registration on the reimbursed drugs list
Since the beginning of 2011, with its significantly more challenging reimbursement process, benefit ratings of new medicines have trended significantly lower. Based on information published on the HAS website, since early 2011 the CT has granted an ASMR rating of IV in 23% of applications. A rating of V (absence of improvement) was granted in 33%, no ASMR rating was given (due to an SMR of "Insufficient") in about 32% of applications, and only 11% of applications were granted an ASMR rating higher than IV (I, II or III).
In France, diseases requiring a long-term, expensive treatment are defined as ALD (Affection de Longue Duree – Long Term Diseases) and patients are fully reimbursed by the National Social Security for all the costs related to this disease (hospitalizations, lab tests, medicines, etc.). More than 8 million French citizens are reimbursed under this scheme. There are 30 broad disease categories – including cancer, several cardiovascular diseases and others – as well as smaller diseases grouped in a separate category. Most orphan diseases fall into this scheme.
Conference Call and Webcast Details
InterMune will host a live webcast of a conference call today at 4:30 p.m. EDT to discuss the CT's assessment of Esbriet in France, and the ASCEND Phase 3 clinical trial of Esbriet in IPF patients in the United States and other territories. Interested investors and others may participate in the conference call by dialing 800-891-8257 (U.S.) or +1-212-271-4651 (international), conference ID# 21587875. A replay of the webcast and teleconference will be available approximately three hours after the call.
To access the webcast, please log on to the company's website at www.intermune.com at least 15 minutes prior to the start of the call to ensure adequate time for any software downloads that may be required.
A telephonic replay will be available for 10 business days following the call and can be accessed by dialing 800-633-8284 (U.S.) or +1 402-977-9140 (international), and entering the conference ID# 21587875.
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 are randomly assigned 1:1 to receive oral pirfenidone (2403 mg/day) or placebo.
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.
Patient eligibility criteria for ASCEND include the following:
- Centrally confirmed diagnosis by High Resolution Computed Tomography (HRCT) (+/- surgical lung biopsy)
- %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
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 Esbriet® (pirfenidone)
Esbriet is an orally active 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 in adults for the treatment of mild to moderate IPF. The approval authorizes marketing of Esbriet in all 27 EU member states. Esbriet has since been approved for marketing in Norway and Iceland. Esbriet is commercially available in Germany, Austria, Norway, Denmark and Luxembourg.
Since 2008, pirfenidone has been marketed in Japan as Pirespa® by Shionogi & Co. Ltd.
InterMune is conducting a Phase 3 study, ASCEND, to pursue the registration of Esbriet for the treatment of IPF in the United States.
Idiopathic pulmonary fibrosis (IPF) is a progressive, debilitating and ultimately fatal disease characterized predominantly by fibrosis (scarring) in the lungs, hindering the ability for gas exchange in the lungs. IPF 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 20-40 percent, which makes IPF more rapidly lethal than many cancers, including breast, ovarian and colorectal. Published epidemiology studies suggest there is a range of between 85,000 and 141,000 IPF patients in Europe, with approximately 113,000 being the median estimate. Patients diagnosed with IPF are primarily between the ages of 40 and 80, with a median age of 63 years. The disease tends to affect slightly more men than women.
InterMune is a biotechnology company focused on the research, development and commercialization of innovative therapies in pulmonology and fibrotic diseases. In pulmonology, InterMune is focused on therapies for the treatment of idiopathic pulmonary fibrosis (IPF), a progressive and fatal lung disease. Pirfenidone, the only medicine approved for IPF anywhere in the world, is approved for marketing by InterMune in the EU as Esbriet® and is currently in a Phase 3 clinical trial in the United States (ASCEND). 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 orphan fibrotic diseases. For additional information about InterMune and its R&D pipeline, please visit www.intermune.com.
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 our expectation that IPF patients in France will be reimbursed at a 100% level for drugs with an SMR rating other than "Insufficient", statements related to the anticipated timing of commercial launch for Esbriet® (pirfenidone) in France, statements related to anticipated pricing and reimbursement of Esbriet in France and statements related to our ASCEND trial, including projected timing for completion of enrollment of the trial and expectations for improved probability of the results of such trial based in part on refined enrollment criteria. 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.
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 February 29, 2012 (the "Form 10-K"), and other periodic reports filed with the SEC, including but not limited to the following: (i) risks related to unexpected regulatory actions or delays or government regulation generally; (ii) 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; (iii) risks related to government, industry and general public pricing pressures; (iv) risks related to our ability to successfully launch and commercialize Esbriet in the EU, including successfully establishing a commercial operation in the EU and receiving favorable governmental pricing and reimbursement approvals in each EU country; (v) risks related to the timing of enrollment of our ASCEND clinical trial, the results of such trial and the prospects for FDA approval of Esbriet in the United States; 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.