Eiger BioPharmaceuticals Completes Submission of New Drug Application to FDA for Lonafarnib for Treatment of Progeria and Progeroid Laminopathies

- Progeria MAA Submitted to EMA and Granted Accelerated Assessment

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Mar 23, 2020, 08:00 ET

PALO ALTO, Calif., March 23, 2020 /PRNewswire/ -- Eiger BioPharmaceuticals, Inc. (Nasdaq: EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that it has completed submission to the U.S. Food and Drug Administration (FDA) of a New Drug Application (NDA) for lonafarnib for the treatment of Progeria and Progeroid Laminopathies. The FDA previously granted Breakthrough Therapy Designation and Rare Pediatric Disease Designation to lonafarnib, which enables eligibility for Priority Review, if relevant criteria are met. Eiger expects to hear from FDA regarding submission acceptance and Priority Review within 60 days.

Lonafarnib is a first-in-class, oral farnesyltransferase inhibitor which has demonstrated extended survival in children and young adults with Progeria, an ultra-rare and fatal disease that causes premature aging in children. Without treatment, children with Progeria die of heart disease at an average age of 14.5 years. Many patients with Progeria or Progeroid Laminopathies have received continuous lonafarnib treatment for greater than 10 years in clinical trials and through the Eiger Managed Access Program.

"We are committed to the Progeria community, including The Progeria Research Foundation and Progeria Family Circle, to bring the first approved treatment to children and young adults with Progeria and Progeroid Laminopathies," said David Cory, President and CEO of Eiger. "The submission of this NDA, as well as the MAA earlier this month, are important steps to achieving this goal. We would like to thank all the children and young adults with Progeria and their families who have made this possible."

About Progeroid Laminopathies
Progeroid Laminopathies are genetic conditions of accelerated aging caused by a constellation of mutations in the lamin A and/or Zmpste24 genes yielding farnesylated proteins that are distinct from progerin. While non‐progerin producing, these genetic mutations result in disease manifestations with phenotypes that have overlap with, but are distinct from, Progeria. Collectively, worldwide prevalence of Progeroid Laminopathies is similar to Progeria.

About Lonafarnib
Lonafarnib is a well‐characterized, late‐stage, orally active inhibitor of farnesyltransferase, an enzyme involved in modification of proteins through a process called prenylation. Progerin is a farnesylated, aberrant protein that researchers believe cannot be cleaved, resulting in tight association with the nuclear envelope, which leads to changes in nuclear envelope morphology and subsequent cellular damage.

Lonafarnib blocks the farnesylation of progerin and has been dosed in over 90 children with Progeria at Boston Children's Hospital in Phase 1/2 and Phase 2 studies. In patients with HGPS, lonafarnib monotherapy was associated with a lower mortality rate after 2.2 years of follow-up compared with no treatment (3.7% vs 33.3%, respectively) with a hazard ratio of 0.12 or a reduction in risk of mortality of 88%.

Lonafarnib has been granted Orphan Drug Designation for Progeria by the FDA and EMA and Breakthrough Therapy Designation and Rare Pediatric Disease Designation by the FDA. Lonafarnib is not approved for any indication, and is licensed by Eiger from Merck Sharp & Dohme Corp.

About The Progeria Research Foundation
The Progeria Research Foundation was established in 1999 by the family of Sam Berns, a child with Progeria. Within four years of its founding, the PRF Genetics Consortium, led by Francis Collins, MD, PhD, discovered the Progeria gene. PRF has also been the driving force behind studies to evaluate lonafarnib as a potential treatment for Progeria and supports scientists who conduct Progeria research. Today, PRF is the only non-profit organization in the world solely dedicated to finding treatments and the cure for Progeria and its age-related conditions, including heart disease. For more information, please visit www.progeriaresearch.org.

About Progeria Family Circle
The Progeria Family Circle (PFC) is a European patient network, founded in 1997, supporting children and young adults with Progeria and their families on a European level. The PFC consists of a network of parents and experts in Europe, visiting medical congresses to keep informed about research developments in Progeria. The PFC goal is to support better and faster recognition of symptoms of Progeria, raise awareness for a better acceptance of Progeria children and young adults in public communities, provide opportunities for families to meet each other through many annual reunions throughout Europe, and offer other necessary and permanent support for families with children with Progeria. For more information, please visit www.progeriafamilycircle.blogspot.com.

About Eiger
Eiger is a late-stage biopharmaceutical company focused on the development and commercialization of first-in-class, well-characterized drugs for serious rare and ultra-rare diseases for patients with high unmet medical needs, for which no approved therapies exist.

Eiger has completed an NDA and MAA submission for lonafarnib for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Progeroid Laminopathies. Eiger has also established a global Managed Access Program, expected to span greater than 40 countries, to ensure all children and young adults with Progeria and Progeroid Laminopathies have access to treatment.

The company's lead program is in Phase 3, developing lonafarnib, a first-in-class oral prenylation inhibitor for the treatment of Hepatitis Delta Virus (HDV) infection. The company is also advancing peginterferon lambda, a first-in-class interferon, toward registration for the treatment of HDV. For additional information about Eiger and its clinical programs, please visit www.eigerbio.com.

Note Regarding Forward-Looking Statements
This press release contains "forward-looking" statements that involve substantial risks and uncertainties. All statements other than statements of historical facts, including statements regarding our future financial condition, timing for and outcomes of clinical results, business strategy and plans and objectives for future operations, are forward-looking statements. These forward-looking statements include terminology such as "believe," "will," "may," "estimate," "continue," "anticipate," "contemplate," "intend," "target," "project," "should," "plan," "expect," "predict," "could," "potentially" or the negative of these terms. Forward-looking statements are our current statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things, our anticipating significant milestones in 2020, the timing of our ongoing and planned clinical development, including the potential for approval of our lonafarnib product candidate in the US and EU for Progeria and Progeroid Laminopathies; our progression and enrollment of our Phase 3 D-LIVR study in HDV; our announcement of data from the trial of peginterferon lambda and lonafarnib boosted with ritonavir for HDV (LIFT); our plans to advance peginterferon lambda in HDV in the US and EU; our plans for continued advancement of avexitide in registration trials; our ability to transition into a commercial stage biopharmaceutical company; our ability to finance the continued advancement of our development pipeline products; that the company's expectations regarding the effects of COVID-19 on the Company's trials and development may be incorrect, and the potential for success of any of our product candidates.

These statements concern product candidates that have not yet been approved for marketing by the U.S. Food and Drug Administration (FDA). No representation is made as to their safety or effectiveness for the purposes for which they are being investigated.

Various important factors could cause actual results or events to differ materially from the forward-looking statements that Eiger makes, including the risks described in the "Risk Factors" sections in the Annual Report on Form 10-K for the year ended December 31, 2019 and Eiger's subsequent filings with the SEC. Eiger does not assume any obligation to update any forward-looking statements, except as required by law.

Investors and Media:
Ingrid Choong, PhD
Sr VP, Clinical Development
(650) 619-6115
[email protected]

Sri Ryali
CFO
(650) 272-6138
[email protected]

SOURCE Eiger BioPharmaceuticals, Inc.