EATG » FDA granted Breakthrough Therapy designation for lonafarnib

FDA granted Breakthrough Therapy designation for lonafarnib

Eiger announces Breakthrough Therapy designation granted by FDA for Lonafarnib for treatment of hepatitis delta virus (HDV) infection

– Phase 3 HDV “D-LIVR” international study initiating

PALO ALTO, Calif., December 17, 2018 — Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for rare and ultra-rare diseases, today announced that the Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for lonafarnib for the treatment of hepatitis delta virus (HDV) infection.  FDA Breakthrough Therapy designation involves a Fast Track development and FDA review process with guidance designed to expedite the development and review of medicines intended to treat serious or life-threatening diseases. HDV is the most severe form of human viral hepatitis and has no approved treatment. Lonafarnib is a first-in-class prenylation inhibitor for the treatment of HDV infection.

“There is an urgent medical need to treat HDV, the most serious form of viral hepatitis,” said David Apelian, MD, PhD, MBA, Chief Operating Officer and Executive Medical Officer.  “We look forward to collaborating with the FDA as we accelerate development of lonafarnib for HDV-infection.  The Phase 3 D-LIVR Study is the first-ever, global registration trial in HDV, with the potential to bring two separate, lonafarnib-based treatment regimens to HDV patients.”

This Breakthrough Therapy designation is supported by data from Phase 2 clinical studies of lonafarnib-based treatment regimens in HDV-infected patients, achieving combined primary endpoints of ≥ 2 log10 decline in HDV RNA and normalization of alanine aminotransferase (ALT) which reflect an improvement in liver condition and virologic response rarely observed in untreated HDV patients.

About Lonafarnib

Lonafarnib is a well-characterized, late-stage, orally active inhibitor of farnesyl transferase, an enzyme involved in modification of proteins through a process called prenylation.  HDV uses this host cell process inside liver cells to complete a key step in its life cycle.  Lonafarnib inhibits the prenylation step of HDV replication inside liver cells and blocks the virus life cycle at the stage of assembly.  Lonafarnib has been dosed in over 120 HDV-infected patients across international academic centers and is in Phase 3 development for HDV with a single, pivotal trial.  Lonafarnib has been granted Orphan Drug designation by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA), and Fast Track and now Breakthrough designation by U.S. FDA.  Lonafarnib is not approved for any indication, and is licensed from Merck Sharp & Dohme Corp. (known as MSD outside of the United States and Canada).

About Hepatitis Delta Virus (HDV)

Hepatitis Delta is caused by infection with HDV and is considered to be one of the most severe forms of viral hepatitis in humans.  Hepatitis delta occurs only as a co-infection in individuals harboring Hepatitis B Virus (HBV).  Hepatitis delta leads to more severe liver disease than HBV alone and is associated with accelerated liver fibrosis, liver cancer, and liver failure.  Hepatitis delta is a disease with a significant impact on global health, which may affect up to approximately 15-20 million people worldwide.  The prevalence of HDV varies among different parts of the world.  Globally, HDV infection is reported to be present in approximately 4.3% to 5.7% of chronic Hepatitis B carriers.  The prevalence of HDV in patients infected with chronic HBV is even higher in certain regions, including certain parts of Mongolia, China, Russia, Central Asia, Pakistan, Turkey, Africa, Middle East and South America, with an HDV prevalence as high as 60% being reported in HBV-infected patients in Mongolia and Pakistan.

About D-LIVR Study

D-LIVR (Delta Liver Improvement and Virologic Response in HDV) is an international, multi-center, Phase 3 study of approximately 300 lonafarnib (LNF)-treated patients (total N=400 patients including controls) to evaluate an all-oral arm of LNF boosted with ritonavir (RTV) and a combination arm of LNF boosted with RTV combined with pegylated interferon-alfa (PEG IFN-alfa), with each arm to be compared to a placebo arm (background HBV nucleos(t)ide only), in HDV-infected patients.  A PEG IFN-alfa alone arm will be dosed to demonstrate contribution of effect only.  The LNF containing arms will not be required to demonstrate superiority over PEG IFN-alfa alone.  A combined primary endpoint of ≥ 2 log10 decline in HDV RNA and ALT normalization at end of 48 weeks of treatment will be used to assess activity of LNF-based regimens versus placebo in the D-LIVR study.

About Eiger

Eiger is a late-stage biopharmaceutical company focused on the accelerated development and commercialization of a pipeline of targeted, first-in-class therapies for rare and ultra-rare diseases.  The company’s lead program is in Phase 3, developing lonafarnib, a first-in-class prenylation inhibitor for the treatment of Hepatitis Delta Virus (HDV) infection.  The company is also preparing an NDA with plans to file in 2019 for lonafarnib in the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Progeroid Laminopathies.  For additional information about Eiger, please visit www.eigerbio.com.