Boehringer Ingelheim’s Faldaprevir-Based Treatment Regimen Achieved Viral Cure in up to 80 Percent of Patients with Hepatitis C in Phase 3 Pivotal Trial

Ridgefield, CT, April 23, 2013 – Boehringer Ingelheim Pharmaceuticals, Inc. today announced results from the pivotal Phase 3 STARTVerso^™1 trial of faldaprevir (BI 201335) in combination with pegylated interferon and ribavirin (PegIFN/RBV). In previously untreated patients with genotype-1 hepatitis C virus (HCV) who received once-daily faldaprevir plus PegIFN/RBV, 79% and 80% in the 120mg and 240mg arms, respectively, achieved viral cure when measured 12 weeks after treatment was completed (SVR12). This is compared with 52% of patients receiving PegIFN/RBV plus placebo (p<0.0001).

In addition, protocol-defined early treatment success (ETS)* was achieved by 87% and 89% of patients treated with the faldaprevir-based regimen (120mg or 240mg, respectively), meaning they were eligible for an overall shorter treatment duration of 12 weeks faldaprevir/24 weeks PegIFN/RBV. Of those patients who completed treatment early, 86% and 89% (120mg, or 240mg, respectively) went on to achieve viral cure (SVR12). This demonstrates that an overall treatment regimen of 24 weeks was sufficient to achieve viral cure in most patients, cutting treatment duration in half when compared to treatment with PegIFN/RBV alone (48 weeks). A goal in HCV treatment innovation is to reduce the amount of time patients are exposed to treatment with interferon.

STARTVerso^™1 included a diverse range of genotype-1a and 1b patients, including patients with compensated cirrhosis (a condition where the liver is heavily scarred but still able to function), who are challenging to treat and cure. The results will be presented tomorrow at EASL’s International Liver Conference^™ (ILC) as part of the official press conference and by Professor Peter Ferenci as a late-breaking oral presentation on April 27 (abstract #3281).

“In the STARTVerso^™1 study, faldaprevir has shown efficacy in a range of genotype-1a and 1b HCV patients with and without cirrhosis,” said Principal Investigator Professor Peter Ferenci of the Medical University Vienna. “The viral cure rates and potential for shorter treatment duration seen in STARTVerso^™ 1 are very encouraging for the many patients currently facing a year of interferon-based treatment.”

Serious adverse events were experienced by 6% (n=8) of placebo patients, 7% (n=17) of patients receiving 120mg faldaprevir and 7% (n=17) of patients receiving 240mg faldaprevir. Anemia (11%, 13%, 12%), rash (6%, 8%, 9%) and gastrointestinal issues (3%, 7%, 12%) were the most common Grade 2-4 adverse events in placebo, faldaprevir 120mg and faldaprevir 240mg arms, respectively. No rash events were life-threatening; 1% of patients discontinued treatment in each study arm due to rash. Adverse events led to discontinuation of study medications in 4% (n=5), 4% (n=10) and 5% (n=14) in the placebo, faldaprevir 120mg and faldaprevir 240mg arms, respectively.

“With the results of the first of our pivotal faldaprevir trials, STARTVerso^™1, now available, Boehringer Ingelheim is making strides toward our short-term goal of providing a treatment option with a shorter duration for HCV patients eligible for interferon-based regimens,” said Peter Piliero, MD, vice president, Clinical Development and Medical Affairs, Boehringer Ingelheim Pharmaceuticals, Inc. “We are developing faldaprevir as a potential foundation for both interferon-based and interferon-free treatment regimens. We are optimistic that ongoing studies with our pipeline compounds will lead to faldaprevir-based interferon-free regimens for patients with HCV.”

In separate abstracts at EASL’s ILC meeting, sub-analyses from Boehringer Ingelheim’s interferon-free Phase 2b SOUND-C2 study are also being presented. These include data regarding viral response rates by level of fibrosis (abstract #1227), predictors of anemia (abstract #1186) and pharmacokinetic modeling of the relationship between virologic response and the level of faldaprevir or BI 207127 found in the blood (abstract #1212). The SOUND-C2 trial evaluated the interferon-free combination of faldaprevir and BI 207127, an investigational non-nucleoside NS5B polymerase inhibitor, plus ribavirin.

Presentation abstracts can be accessed through the congress website.

About STARTVerso^™1
STARTVerso^™1 is a double-blind, placebo-controlled Phase 3 trial of faldaprevir in combination with PegIFN/RBV. The study enrolled and treated 652 treatment-naïve patients from Europe and Japan who were infected with chronic genotype-1 HCV. Patients were randomized to receive PegIFN/RBV in combination with a once-daily dose of 120mg faldaprevir, 240mg faldaprevir or placebo. Treatment duration depended on whether patients met criteria for ETS, protocol-defined early treatment success (week 4 below limit of quantification [BLQ] and week 8 below limit of detection [BLD]).

• Group 1: All patients received 24 weeks of placebo with PegIFN/RBV followed by 24 weeks of PegIFN/RBV (48 weeks total treatment).
• Group 2: All patients received 12 weeks of 120mg faldaprevir with PegIFN/RBV. Patients who met ETS criteria received an additional 12 weeks of PegIFN/RBV (24 weeks total treatment). Patients who did not meet ETS criteria received an additional 12 weeks of 120mg faldaprevir with PegIFN/RBV followed by 24 weeks of PegIFN/RBV (48 weeks total treatment).
• Group 3: All patients received 12 weeks 240mg faldaprevir with PegIFN/RBV followed by 12 weeks of PegIFN/RBV. Patients who met ETS criteria stopped treatment after 24 weeks total treatment. Patients who did not meet ETS criteria received an additional 24 weeks of PegIFN/RBV (48 weeks total treatment).

About Boehringer Ingelheim in Hepatitis C Virus (HCV)
In partnership with the scientific community, our clinical trial program is rigorously designed to find answers to the challenges that HCV patients face, including those who are the most difficult to treat. Our pivotal HCV clinical trials for faldaprevir and BI 207127 are comprised of two multi-trial programs, STARTVerso^™ and HCVerso^™.

Faldaprevir, also known as BI 201335, is an investigational, oral protease inhibitor that is specifically designed to target viral replication in the liver. Boehringer Ingelheim is developing faldaprevir as a core component of both interferon-based and interferon-free hepatitis C treatment regimens. STARTVerso^™1 is the first of an ongoing multi-study Phase 3 trial program that is evaluating faldaprevir combined with PegIFN/RBV. Three additional trials in treatment-naïve, treatment-experienced and HIV co-infected patients with chronic genotype-1 HCV are near clinical completion. BI 207127 is an investigational NS5B non-nucleoside polymerase inhibitor that has shown the potential to eliminate interferon from HCV treatment when combined in a regimen with faldaprevir and RBV. Phase 2 trials of this interferon-free regimen have been completed and Phase 3 HCVersoTM trials investigating this regimen are now underway. As part of our long-term commitment to HCV, the company is exploring other combinations of investigational HCV compounds that work in complementary ways. The recent collaboration of Boehringer Ingelheim with Presidio Pharmaceuticals, Inc. for a Phase 2 clinical study investigating an interferon-free, all-oral combination is part of the company’s continued commitment to discover and develop innovative options for the treatment of HCV.

Faldaprevir and BI 207127 are investigational compounds and not approved by the FDA. Their safety and efficacy have not been established.

Hepatitis C is a blood-born infectious disease and a leading cause of chronic liver disease, transplant and failure that affects as many as 150 million people globally. In the United States, an estimated 4.1 million Americans have been infected with HCV, of which approximately 3.2 million have chronic HCV infection. Since 1999 there has been a significant increase in deaths due to chronic HCV, which accounts for 10,000 - 12,000 deaths in the United States per year.

About Boehringer Ingelheim Pharmaceuticals, Inc.
Boehringer Ingelheim Pharmaceuticals, Inc., based in Ridgefield, CT, is the largest U.S. subsidiary of Boehringer Ingelheim Corporation (Ridgefield, CT) and a member of the Boehringer Ingelheim group of companies.

The Boehringer Ingelheim group is one of the world’s 20 leading pharmaceutical companies. Headquartered in Ingelheim, Germany, it operates globally with 145 affiliates and more than 42,000 employees. Since it was founded in 1885, the family-owned company has been committed to researching, developing, manufacturing and marketing novel products of high therapeutic value for human and veterinary medicine.

As a central element of its culture, Boehringer Ingelheim pledges to act socially responsible. Involvement in social projects, caring for employees and their families, and providing equal opportunities for all employees form the foundation of the global operations. Mutual cooperation and respect as well as environmental protection and sustainability are intrinsic factors in all of Boehringer Ingelheim’s endeavors.

For more information, please visit http://us.boehringer-ingelheim.com and follow us on Twitter at http://twitter.com/boehringerus.

*ETS = protocol-defined early treatment success (week 4 below limit of quantification [BLQ] and week 8 below limit of detection [BLD])

Related Material

• EASL Press Release