Sentynl and Fortress ‘COMEBACK’ with its Menkes disease therapy

Introduction

Sentynl Therapeutics, a U.S. biopharmaceutical company with its exclusive Zydus Lifesciences, centralising its aim at first place for establishing and introducing specialised, effective and innovative therapies for mainly rare diseases. Zydus Lifesciences is the parent company of Sentynl.

Fortress Biotech, Inc. is a leading biopharmaceutical company that acquires, establishes, and markets promising commercialising medicines and also supports the development of drug candidates at the early-stage. The entire delivery and operation process is carried out through the company’s own parent and partner companies. With their unique and promising portfolio, the in-licensing of powerful assets gets space to create a value-based foundation.

Announcement

After the rejection faced by Fortress Biotech and Sentynl Therapeutics for Zycubo for Menkes disease in October 2024, the companies upped their efforts to introduce the new replacement therapy. This modification not only fixed the potential value of the companies but also helped them receive approval for their brilliant copper replacement therapy for Menkes disease. This therapy will be marketed as ‘Zycubo’. Making weakness your biggest strength spells the dedication level of both companies.

The therapy is much needed, as around 100,000 to 250,000 infants are born with Menkes disease. This rare genetic disorder causes stress to babies and their parents. This disorder is erupted from the mutations to the ATP7A gene that can be treated by the protein that brings back the balance in copper levels and restores overall health. This milestone is a reward for the long-serving excellence and contribution of the companies to the vast healthcare sector.

Symptoms and new therapy

The symptoms for this condition are damage to the nervous system, slow development, seizures and intellectual disabilities. Menkes disease is a life-threatening condition as it takes the life of the individual within three years. Following these stressful symptoms, Zycubo will be the relief, a hope for the babies and parents.

Zycubo is administered through hypodermic injection and actively performs by replacing copper volume. In the previous Phase 2 readouts, around 80% mitigation was captured in the patient’s death rate if the drug was dosed within four weeks of birth, and the other dosed to the undiagnosed historical controls.

Though the efficacy rate was not satisfying, the drug was said to be better than the unattended historical controls. This new approval for Zycubo was registered after the FDA approved Waskyra for Wiskott-Aldrich syndrome, which became the first ever gene therapy for the same indication (for rare diseases).