• 06 May 2024
• Admin
• News Article

Sentynl Acquires Zokinvy to Treat Hutchinson-Gilford Progeria Syndrome

Sentynl, a Zydus Group company acquires Zokinvy to treat Hutchinson-Gilford progeria syndrome from Eiger BioPharma

Overview

Sentynl Therapeutics, Inc. (Sentynl), a US-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (Zydus Group), and Eiger BioPharmaceuticals, Inc., a commercial-stage biopharmaceutical company focused on the development of innovative therapies for rare metabolic diseases, announced the closing of the sale of Eiger’s Zokinvy (lonafarnib) programme to Sentynl.

Zokinvy: Sole Treatment for Progeria

• Zokinvy is the first and only treatment approved by the US Food and Drug Administration (FDA) to target the cause and symptoms of progeria, also known as Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies (PDPL), in young people 12 months of age and older. 
• Collectively known as progeria, HGPS and PDPL are ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients. 
• Following its US approval in 2020, Zokinvy secured approval in the European Union and Great Britain (2022) and in Japan (January 2024).

Words from Zydus Lifesciences

Speaking on the acquisition, Dr Sharvil Patel, managing director of Zydus Lifesciences, said, “This acquisition marks an important milestone in growing our portfolio of medicines for rare and orphan diseases, which can have devastating consequences if left untreated. We are focused on supporting patients to live healthier and more fulfilled lives. The acquisition of Zokinvy directly furthers this mission, as it has a demonstrated meaningful impact on young patients and their families.”

Words from CEO: Sentynl

• “It is an honour to add Zokinvy to our portfolio of products that have a tangible impact on the lives of rare disease patients, whose needs are too often unmet or overlooked,” said Matt Heck, president & chief executive officer of Sentynl. 
• “We are firmly committed to provide best-in-class global access to Zokinvy and are eager to serve the patients and their families affected by progeria. We are grateful to Eiger and The Progeria Research Foundation for their dedicated effort to develop and secure availability of this life-changing product.”

Words from CEO: Eiger

• “We are pleased to complete this agreement with Sentynl, given our shared commitment to supporting patients of life-threatening, rare conditions with high unmet medical needs,” said David Apelian, MD, PhD, MBA, CEO of Eiger. 
• “We thank The Progeria Research Foundation for their continued support of Zokinvy.”

PRF on Importance of Zokinvy

• Audrey Gordon, president and executive director of The Progeria Research Foundation (PRF), added, “Without Zokinvy therapy, children with progeria die of the same heart disease that affects millions of normally aging adults, but by an average age of 14.5 years old. Zokinvy gives these beautiful children longer, healthier lives.”
• “Since we first launched PRF in 1999, we have achieved tremendous progress in global awareness, breakthrough research, and treatment of progeria. We are thankful for our successful partnership with Eiger, and are excited to now join forces with Sentynl in our journey to continue advancing the research and treatment of this syndrome, with the ultimate goal to find the cure.”

Legal processes Related to Sale of Eiger

• As previously disclosed by Eiger, on April 1, 2024, Eiger and its direct subsidiaries filed voluntary petitions for relief under chapter 11 of Title 11 of the United States Code (Chapter 11 Cases) in the United States Bankruptcy Court for the Northern District of Texas (Bankruptcy Court). 
• On April 17, 2024, following the completion of the auction held as part of the Eiger’s court-supervised sale process, Sentynl was designated the winning bidder with a final bid during the auction of a base price in the amount of $46.1 million less a credit in the amount of $0.9 million for the termination fee resulting in a net base price in the amount of $45.2 million, subject to certain purchase price adjustments, including a reduction of $100,000 per diem if the sale closed after April 24, 2024. 
• At a hearing held on April 23, 2024, the Bankruptcy Court approved the sale to Sentynl, with the sale closing on May 3, 2024. Under the terms of the acquisition, Sentynl acquired global rights to Zokinvy and will be responsible for its manufacture and commercialization.

Genetic Premature Aging Diseases

Collectively known as progeria, Hutchinson-Gilford progeria syndrome and progeroid laminopathies are ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients.

HGPS

• HGPS is caused by a point mutation in the LMNA gene, yielding the farnesylated aberrant protein, progerin. 
• Progeroid laminopathies are genetic conditions of accelerated aging caused by a constellation of mutations in the LMNA and/or ZMPSTE24 genes yielding farnesylated proteins that are distinct from progerin.

Absence of Zokinvy Therapy 

• Without Zokinvy therapy, children with HGPS commonly die of the same heart disease that affects millions of normally aging adults (arteriosclerosis), by an average age of 14.5 years. 
• Disease manifestations include severe failure to thrive, scleroderma–like skin, global lipodystrophy, alopecia, joint contractures, skeletal dysplasia, global accelerated atherosclerosis with cardiovascular decline, and debilitating strokes.

About Zokinvy

• Zokinvy is a first-in-class disease-modifying agent that blocks the accumulation of defective progerin and progerin-like proteins which leads to cellular instability and premature aging in children and young adults with progeria. 
• Zokinvy has demonstrated a statistically significant survival benefit in children and young adults with HGPS.

Adverse Reactions

• The most commonly reported adverse reactions were gastrointestinal (vomiting, diarrhoea, nausea), and most were mild or moderate (Grade 1 or 2) in severity. 
• Many progeria patients have received continuous Zokinvy therapy for more than 10 years.

FDA Approval

Zokinvy is FDA approved for the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation.

Rights to Lonafarnib: Eiger

Eiger licensed exclusive worldwide rights to lonafarnib for the treatment of H-G progeria from MSD, the tradename of Merck & Co., Inc, Rahway, N.J., USA. MSD provided lonafarnib free of charge for clinical studies supported by the PRF and waived royalty and milestone obligations on lonafarnib from Sentynl for people living with the condition.

Exclusive Distribution Agreement

• Eiger and AnGes entered into an exclusive distribution agreement for the treatment of HGPS and PDPL indications, Zokinvy (Lonafarnib), in Japan on May 10, 2022. 
• In March 2023, the Ministry of Health, Labour and Welfare designated Zokinvy as an orphan drug.

Sentynl Therapeutics

• Sentynl Therapeutics is a US-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases. 
• The company was acquired by the Zydus Group in 2017. Sentynl’s experienced management team has previously built multiple successful pharmaceutical companies.

Zydus Lifesciences

Zydus Lifesciences Ltd. with an overarching purpose of empowering people with freedom to live healthier and more fulfilled lives, is an innovative, global lifesciences company that discovers, develops, manufactures, and markets a broad range of healthcare therapies.

About Eiger

Eiger is a commercial-stage biopharmaceutical company focused on the development of innovative therapies for rare metabolic diseases.

Progeria Research Foundation

• The Progeria Research Foundation (PRF) was established in 1999 by the family of Sam Berns, a child with Progeria. 
• Within four years of its founding, the PRF Genetics Consortium discovered the Progeria gene, a collaboration led by Dr. Francis Collins, Acting Science Advisor to the President of the United States and former Director of the National Institutes of Health (NIH).