RIBOMIC Inc., a Tokyo-based leader in aptamer therapeutics, has reported significant progress in its early phase II study of umedaptanib pegol. This breakthrough involves treating pediatric patients with Achondroplasia, a rare genetic disorder characterized by short stature and limb shortening.

Early Phase II Study Progresses

The company has successfully enrolled 13 children aged 5 to 14 years in an observational study of umedaptanib pegol, an anti-FGF2 aptamer. This study is a critical step in assessing the drug’s efficacy and safety. The low-dose subcutaneous study, referred to as Cohort 1, is underway with six patients who have completed the observational phase. Results from this cohort are expected by November 2024.

RIBOMIC Inc. has also initiated dosing for the high-dose subcutaneous study, known as Cohort 2, with the first patient having already received treatment. No safety issues have been reported in either the early phase II study or the extension study, underscoring the promise of umedaptanib pegol in treating Achondroplasia.

Umedaptanib Pegol’s Potential

Umedaptanib pegol, formerly designated as RBM-007, is an innovative oligonucleotide-based aptamer that targets fibroblast growth factor 2 (FGF2). This drug aims to address the root cause of Achondroplasia by inhibiting the overactive FGF2 activity. Umedaptanib pegol has also shown clinical proof of concept in treating exudative age-related macular degeneration, further validating its therapeutic potential.

Achondroplasia arises from a mutation in the fibroblast growth factor receptor type 3 (FGFR3), which leads to impaired cartilage growth. This genetic disorder, affecting approximately 1 in 25,000 newborns, significantly impacts patients’ height and limb length, highlighting the urgent need for effective treatments.

RIBOMIC’s Commitment to Innovation

RIBOMIC Inc. is dedicated to advancing aptamer therapeutics through its RiboART system, a cutting-edge platform for discovering novel aptamer drugs. The company’s ongoing research and development efforts aim to address a broad range of unmet medical needs, including rare childhood diseases and ocular conditions.

RIBOMIC Inc.’s advancements with umedaptanib pegol. He emphasizes the drug’s potential to transform Achondroplasia treatment by directly targeting FGF2 activity, offering hope for better management of this challenging condition.

What is umedaptanib pegol and what are its potential applications?

Umedaptanib pegol, developed by RIBOMIC Inc., is a novel oligonucleotide-based aptamer targeting fibroblast growth factor 2 (FGF2). Initially researched for conditions like wet age-related macular degeneration (wAMD), it has now expanded into trials for treating Achondroplasia, a genetic disorder causing short stature​.

What are the recent developments in the clinical trials of umedaptanib pegol for Achondroplasia?

RIBOMIC Inc. has successfully progressed to an early phase II study of umedaptanib pegol for Achondroplasia, with the trial currently evaluating the drug’s efficacy and safety. The company has completed enrolling pediatric patients and has initiated dosing in both low-dose and high-dose cohorts.

What makes umedaptanib pegol a significant development in treating Achondroplasia?

The drug aims to inhibit the overactive FGF2 activity linked to Achondroplasia, potentially addressing the root cause of the disorder rather than just managing symptoms. Its development is particularly significant due to the limited treatment options currently available for this condition​.

How does RIBOMIC Inc. contribute to the field of aptamer therapeutics?

RIBOMIC is at the forefront of aptamer therapeutic development, utilizing its RiboART system to discover novel aptamer drugs. The company focuses on addressing a wide range of medical needs, including rare diseases and ocular conditions, showcasing its commitment to innovative treatment solutions​.

What are the future prospects for umedaptanib pegol?

With ongoing phase II trials and a positive outlook based on early data, umedaptanib pegol shows promise for broader applications beyond Achondroplasia, including potential uses in treating eye diseases and other fibroblast growth factor-related disorders. The outcomes of these trials will be crucial in determining its path towards regulatory approval and clinical use​.

  Achondroplasia treatment, aptamer therapeutics, fibroblast growth factor 2, RBM-007, RiboART system, RIBOMIC Inc., Tokyo, umedaptanib pegol