Boehringer Ingelheim Expands Investigation of Interferon-free Hepatitis C Treatment Regimens to Reach More Patient Types through Presidio Pharmaceuticals Clinical Collaboration

Ridgefield, CT, September 10, 2013 – Boehringer Ingelheim Pharmaceuticals, Inc. today announced the completion of patient enrollment for a Phase 2a clinical trial (NCT01859962) investigating a new interferon-free, all-oral, direct-acting antiviral (DAA) combination treatment for patients with genotype-1a chronic hepatitis C virus (HCV) infection. This trial is conducted in collaboration with Presidio Pharmaceuticals, Inc. and evaluates Boehringer Ingelheim’s investigational compounds, the protease inhibitor faldaprevir (BI 201335) and non-nucleoside NS5B polymerase inhibitor, deleobuvir (BI 207127), in combination with Presidio’s investigational pan-genotypic NS5A inhibitor, PPI-668, with and without ribavirin.

The trial includes 36 treatment-naïve genotype-1a infected patients treated for 12 weeks with this all-oral DAA regimen, with 24 weeks of post-treatment follow-up. The primary endpoint of the trial is sustained virologic response 12 weeks after treatment is completed (SVR12). This Phase 2a trial of a novel combination therapy is a part of Boehringer Ingelheim’s commitment to develop new, tailored interferon and ribavirin-free HCV regimens for a broad range of HCV patients.

In March 2013, the two companies entered a non-exclusive collaboration to evaluate the three DAAs in combination regimens. Both companies will retain all rights to their respective compounds. Presidio has operational responsibility for this collaborative trial, with oversight by an intercompany project team. Final results are expected in Q2 2014.

“We are proud to announce that we have completed enrollment in this collaborative trial designed to investigate an all-oral combination of compounds to treat patients with HCV. This further compliments our ongoing Phase 3 HCVerso^® trial program, which aims to provide interferon-free treatment to genotype-1b patients,” said Peter Piliero, M.D., Vice President, Clinical Development and Medical Affairs, Boehringer Ingelheim Pharmaceuticals, Inc. “Through the development of our own pipeline compounds and through new collaborations like this we aim to provide interferon- and ribavirin-free regimens for a wide range of patients  and hepatitis C genotypes, including the most challenging types to cure. In prior clinical trials, PPI-668 has shown pan-genotypic properties that bear the potential to explore the efficacy of this regimen in a wider range of HCV genotypes.”

Eliminating injectable interferon from treatment regimens is a critical goal in hepatitis C management. Clinical studies have shown that up to 50 percent of patients may not be eligible for treatment with interferon due to contraindications. Patients may also find interferon challenging due to long treatment duration and side-effects. These side-effects commonly include fatigue, anxiety, depression, gastrointestinal and flu-like symptoms. More serious side-effects may include heart failure, sepsis, leukopenia and vision loss.

“Clinical trials evaluating multiple DAAs are exciting because they can help bring us closer to our goal of developing more effective and tolerable interferon-free and potentially ribavirin-free therapies,” said Jacob Lalezari, M.D., Director of Quest Clinical Research in San Francisco, CA. “By treating patients with multiple compounds that attack the hepatitis C virus in different ways, we hope to be able to cure more patients in less time with fewer of the side-effects associated with existing treatment options.”

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

Boehringer Ingelheim has a comprehensive interferon-free clinical trial program, HCVerso^®, which includes three pivotal Phase 3 studies. The HCVerso^® studies aim to enroll approximately 1,100 treatment-naïve and experienced HCV genotype-1b (GT-1b) patients, including those who are interferon eligible or ineligible, and those with compensated liver cirrhosis.

For more information regarding the trial, please visit www.clinicaltrials.gov.

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 deleobuvir 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. Deleobuvir, also known as 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 HCVerso^® 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 2a clinical study investigating an interferon- and ribavirin-free, all-oral combination is part of the company’s continued commitment to discover and develop innovative options for the treatment of HCV.

STARTVerso^® and HCVerso^® are registered service marks of Boehringer Ingelheim International GmbH.

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
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 140 affiliates and more than 46,000 employees. Since it was founded in 1885, the family-owned company has been committed to researching, developing, manufacturing and marketing novel medications of high therapeutic value for human and veterinary medicine.

Social responsibility is a central element of Boehringer Ingelheim’s culture. 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 www.us.boehringer-ingelheim.com

About Presidio in Hepatitis C Virus (HCV)
Presidio Pharmaceuticals, Inc. is a San Francisco-based clinical stage specialty pharmaceutical company focused on the discovery and development of oral pan-genotypic therapeutics for HCV patients.  Efforts are currently focused on novel inhibitors of both the HCV NS5A and NS5B genes. PPI-668 is an investigational, pan-genotypic, once daily, NS5A inhibitor. In earlier clinical studies in healthy volunteers and HCV-infected patients, PPI-668 has been well-tolerated to date with no serious or severe adverse events and no apparent pattern of treatment-related clinical side effects or laboratory abnormalities. PPI-668 achieves plasma concentrations high enough to inhibit most pre-existing resistant variants and achieves steady-state levels after a single dose. In a clinical study of PPI-668 monotherapy in GT1 HCV-infected patients, viral load reductions of 3.5 to 3.7 log10 HCV were achieved in 1-2 days. Activity was also noted in GT3a HCV-infected patients.

Presidio’s NS5B inhibitor, PPI-383, is a novel pan-genotypic non-nucleoside inhibitor with potential to inhibit all of the major HCV genotypes. This compound is currently in Phase 1 studies in healthy subjects.  For more information, please visit our website at: www.presidiopharma.com.

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