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KDDF-201904-10 Non-Clinical Study and Preparation for Phase 2 Clinical Trial of a Novel Biotherapeutics for Duchenne Muscular Dystrophy(Metabolic Disorders, Protein) [2019-10-25]

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Ongoing Project
Section Lead
Generation
Lead
Optimization
Preclinical Phase I Phase II Phase III

Development and Market Objectives

To complete an IND application of PF1801 for global phase 2 clinical trial in patients with Duchenne muscular dystrophy from US FDA, after contract manufacturing of clinical samples of PF1801

Unmet Medical Need & Target Patients

- Target patient group: Patient group diagnosed with Duchenne muscular dystrophy

- Unmet Medical Needs:  

  • Approved steroid (Emflaza) incurs serious adverse effects from long-term, high-dose treatment, and a gene therapy products (Exondys51, Translarna) are efficacious to only a portion of the patients (approx.. 26%) with limited efficacy but the cost is very high (approx.. 300,000 USD per year).

  • Very strong needs for a highly efficacious, versatile, economical, and safe drug

Status

- Non-clinical trial is on-going

- Global contract manufacturing of clinical sample is under preparation

- Package preparation for US FDA pre-IND meeting is underway

Intellectual Property

- Korean patents registered

- International patents filed and under review: PCT, USA, EU, Japan, China

Competitive Advantages

- ‘Dual blocker’ of skeleton muscle breakdown proteins in myokine pathway

- MoA for not only muscle mass increase but also muscle strength improvement

- Its safety had been already recognized by US FDA (from Phase 2 clinical trials for other indication)

- Superior PK for once-a-week self-administration

- Economical drug cost

Contact & Company Overview

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