Sentynl Therapeutics Announces U.S. FDA Acceptance and Priority Review of New Drug Application for CUTX-101 (Copper Histidinate) Product Candidate for Treatment of Menkes Disease

Rhea-AI Summary

Sentynl Therapeutics has announced that the FDA has accepted and granted Priority Review to its New Drug Application (NDA) for CUTX-101, intended for the treatment of Menkes disease, a rare and fatal pediatric disorder.

The FDA has set a six-month review period with a target action date of June 30, 2025. If approved, CUTX-101 would be the first FDA-approved treatment for Menkes disease, which is caused by mutations in the ATP7A gene affecting copper transport in the body. Current estimates suggest a prevalence of 1 in 34,810 to 1 in 8,664 live male births.

Topline clinical efficacy results show that early treatment with CUTX-101 significantly improves overall survival in Menkes disease patients, reducing the risk of death by nearly 80% compared to an untreated historical control cohort. Median overall survival was 177.1 months for the early treatment cohort versus 16.1 months for the historical control.

Sentynl has taken over the development and commercialization of CUTX-101 from Cyprium Therapeutics, a subsidiary of Fortress Biotech. The drug has received multiple designations from the FDA, including Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designations, as well as Orphan Designation from the EMA.

Positive

• FDA acceptance and Priority Review of NDA for CUTX-101.
• Potential first FDA-approved treatment for Menkes disease.
• Six-month Priority Review with a target action date of June 30, 2025.
• Statistically significant improvement in overall survival for early treatment cohort.
• Nearly 80% reduction in risk of death compared to untreated cohort.
• Multiple FDA designations including Breakthrough Therapy and Fast Track.

Negative

• None.

Insights

Medical Research Analyst

The FDA's acceptance and priority review of CUTX-101 represents a pivotal development in rare disease therapeutics. The drug's clinical data showing 80% reduction in mortality risk and extension of median survival from 16.1 to 177.1 months is remarkably impressive for Menkes disease. This rare X-linked condition affects between 1 in 34,810 to 1 in 8,664 male births, with current patients facing a devastating prognosis of death typically within 2-3 years. The multiple FDA designations (Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug) underscore the critical unmet need and therapeutic potential. For FBIO shareholders, the June 30, 2025 PDUFA date could mark a significant catalyst, as rare disease treatments often command premium pricing due to patient populations and high unmet need.

Financial Analyst

This regulatory milestone positions FBIO for potential value creation through its subsidiary Cyprium Therapeutics. While Sentynl now leads commercialization, FBIO stands to benefit from milestone payments and potential royalties if CUTX-101 receives approval. The orphan drug market is particularly lucrative, with treatments often priced at premium levels due to small patient populations and significant development costs. With an estimated prevalence of up to 1 in 8,664 male births and no current FDA-approved treatments, CUTX-101 could capture a valuable market position. The priority review status, reducing the standard 10-month review to 6 months, accelerates the potential time to market and revenue generation. For a company with a market cap of $58.2M, successful commercialization of CUTX-101 could significantly impact FBIO's valuation.

Six-month priority review granted for CUTX-101 copper histidinate with PDUFA target action date set for June 30, 2025

CUTX-101 has potential to be the first FDA-approved treatment for Menkes disease, a rare and fatal pediatric disease

SOLANA BEACH, Calif. and AHMEDABAD, India and MIAMI, Jan. 6, 2025 /PRNewswire/ -- Sentynl Therapeutics, Inc. ("Sentynl"), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. ("Zydus Group"), and Fortress Biotech, Inc. (Nasdaq: FBIO) ("Fortress") announced that the U.S. Food and Drug Administration (FDA) has accepted for filing and Priority review Sentynl's New Drug Application (NDA) for CUTX-101, the product candidate for the treatment of Menkes disease. Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. Recent estimates suggest a prevalence of 1 in 34,810 to as high as 1 in 8,664 live male births.

Sentynl's NDA is supported by positive topline clinical efficacy results for CUTX-101, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment with CUTX-101, with a nearly 80% reduction in the risk of death compared to an untreated historical control cohort. Median overall survival was 177.1 months for CUTX-101 early treatment cohort compared to 16.1 months for the untreated historical control cohort.

"The NDA acceptance of CUTX-101 marks an important step towards our vision to transform lives and meaningfully impact patients, caregivers, and the rare disease community at large," said Punit Patel, President and CEO, Zydus Americas. "We remain committed to providing access to path-breaking discoveries that can address unmet needs globally, as evident in the potential impact of CUTX-101 on Menkes disease patients and their families."

"Menkes disease presents a difficult journey for patients and their caregivers, as ATP7A mutations impact the transport of copper to a range of organs and systems, such as the lungs, brain and heart. With no known cure or current FDA-approved treatments, death typically occurs between 2 to 3 years of age," said Matt Heck, President & Chief Executive Officer of Sentynl. "We are eager for the FDA to review our application for CUTX-101, which has the potential to be the first FDA-approved therapy for this devastating condition."

CUTX-101 was granted Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug Designations by the FDA and Orphan Designation by the European Medicines Agency (EMA). Under the Prescription Drug User Fee Act (PDUFA), the FDA set a six-month period with a target action date of June 30, 2025.

In December 2023, Sentynl assumed full responsibility for the development and commercialization of CUTX-101 from Cyprium Therapeutics ("Cyprium"), a Fortress subsidiary company focused on the development of novel therapies for the treatment of Menkes disease and related copper metabolism disorders.

"We congratulate Sentynl on the NDA acceptance for filing and review of CUTX-101, a program that Cyprium advanced and proudly supports," said Lindsay A. Rosenwald, M.D., Chairman, President and Chief Executive Officer of Fortress and Chairman of Cyprium. "The drug has a demonstrated safety and efficacy profile for the treatment of Menkes disease and, if approved, CUTX-101 will fill a significant unmet need for children suffering from this rare, fatal pediatric disease."

About CUTX-101 (Copper Histidinate)
CUTX-101 is an investigational drug currently under NDA review with the U.S. FDA to treat patients with Menkes disease. CUTX-101 is a subcutaneous injectable formulation of copper histidinate manufactured under current good manufacturing practice ("cGMP") that is intended to improve tolerability due to its physiological pH. In a Phase 1/2 clinical trial conducted by Stephen G. Kaler, M.D., M.P.H., at the National Institutes of Health ("NIH"), early treatment of patients with Menkes disease with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. Cyprium previously reported positive topline clinical efficacy results for CUTX-101, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment (ET) with CUTX-101, compared to an untreated historical control cohort, with a nearly 80% reduction in the risk of death. Median overall survival (OS) was 177.1 months for CUTX-101 ET cohort compared to 16.1 months for the untreated historical control cohort. CUTX-101 has been granted FDA Breakthrough Therapy, Fast Track, Rare Pediatric Disease and FDA Orphan Drug Designations. Additionally, the European Medicines Agency granted Orphan Designation for CUTX-101. An expanded access protocol for patients with Menkes disease is ongoing at multiple U.S. medical centers.

About Menkes Disease
Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. The minimum birth prevalence for Menkes disease is believed to be 1 in 34,810 live male births, and potentially as high as 1 in 8,664 live male births, based on recent genome-based ascertainment (Kaler SG, Ferreira CR, Yam LS. Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome Aggregation Database (gnomAD). Molecular Genetics and Metabolism Reports 2020 June 5;24:100602). The condition is characterized by distinctive clinical features, including sparse and depigmented hair ("kinky hair"), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, failure to thrive, and neurodevelopmental delays. Mortality is high in untreated Menkes disease, with many patients dying between 2-3 years of age. Milder versions of ATP7A mutations are associated with other conditions, including Occipital Horn Syndrome and ATP7A-related Distal Motor Neuropathy. Currently, there is no FDA-approved treatment for Menkes disease and its variants.

About Sentynl Therapeutics
Sentynl Therapeutics, Inc. ("Sentynl") is a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients suffering from rare diseases. The company was acquired by the Zydus Group in 2017. Sentynl's experienced management team has previously built multiple successful pharmaceutical companies. With a focus on commercialization, Sentynl looks to source effective and well-differentiated products across a broad spectrum of therapeutic areas to address unmet needs. Sentynl is committed to the highest ethical standards and compliance with all applicable laws, regulations and industry guidelines. For more information, visit https://sentynl.com.

About Zydus Group
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. The group employs over 27,000 people worldwide, including 1,400 scientists engaged in R & D, and is driven by its mission to unlock new possibilities in lifesciences through quality healthcare solutions that impact lives. The group aspires to transform lives through path-breaking discoveries. For more information, visit https://www.zyduslife.com/zyduslife.

About Fortress Biotech
Fortress Biotech, Inc. ("Fortress") is an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue. The company has seven marketed prescription pharmaceutical products and over 20 programs in development at Fortress, at its majority-owned and majority-controlled partners and subsidiaries and at partners and subsidiaries it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is focused on leveraging its significant biopharmaceutical industry expertise and network to further expand the company's portfolio of product opportunities. Fortress has established partnerships with some of the world's leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including AstraZeneca, City of Hope, Fred Hutchinson Cancer Center, Nationwide Children's Hospital and Sentynl. For more information, visit www.fortressbiotech.com.

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SOURCE Sentynl Therapeutics

FAQ

What does the FDA Priority Review of CUTX-101 mean for FBIO shareholders?

The FDA Priority Review of CUTX-101 indicates a faster review process, potentially leading to quicker market approval and increased shareholder value for FBIO.

When is the FDA target action date for CUTX-101?

The FDA target action date for CUTX-101 is set for June 30, 2025.

What is the significance of CUTX-101 for Menkes disease treatment?

CUTX-101 could become the first FDA-approved treatment for Menkes disease, significantly improving survival rates for patients.

How effective is CUTX-101 in treating Menkes disease?

Clinical trials showed that early treatment with CUTX-101 reduced the risk of death by nearly 80% and increased median overall survival to 177.1 months.

What designations has CUTX-101 received from the FDA?

CUTX-101 has received Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug Designations from the FDA.

Who is responsible for the development of CUTX-101?

Sentynl Therapeutics, a subsidiary of Zydus Lifesciences, has taken over the development and commercialization of CUTX-101 from Cyprium Therapeutics, a Fortress Biotech subsidiary.