Abstract ▼

IV. Research Results
Milestone 1: Finishing phase 1 clinical trials and IND approval for phase 2 clinical trials A dose-block randomized, placebo controlled(double blind), active controlled(open-label), dose escalation study to investigate the tolerability, and PK/PD of HL2351 after a single subc

IV. Research Results
Milestone 1: Finishing phase 1 clinical trials and IND approval for phase 2 clinical trials A dose-block randomized, placebo controlled(double blind), active controlled(open-label), dose escalation study to investigate the tolerability, and PK/PD of HL2351 after a single subcutaneous administration in healthy male subjects
- Phase 1 trial was complete： Phase 1 trial was done and analyzed by SNU Clinical Trials Center .
- In vivo ADME: Obtained the data on PK profile in Juvenile monkey - Nonclinical trial [Completed Long-term(6 months) toxicity study on monkey]: Obtained safety data on monkey to perform Phase 2 trial
- CDP consulting
- Manufacturing drug substance and its stability test for Phase 2 trial
- IND application and approval for phase 2 clinical trials

Milestone 2: Phase 2 trial was initiated but terminated
- Manufacturing drug substance for phase 2 clinical trial: Not applicable as it was completed in Milestone 1
- Phase 2 trial was terminated:
• Multiple dose regimen of 4mg/kg, twice-weekly was determined as per PK/PD results obtained during the Single Dose PK/PD period.
• Five subjects were enrolled but withdrawn in Multiple dose HL2351 treatment phase.
The main reason of withdrawal was that the maximum dose deliverable via SC is not sufficient for CAPS patients.
• PK method validation was completed.
- Manufacturing drug substance and its stability test : Not applicable due to Study termination
- Nonclinicai trial (Reproductive Toxicity Study) : Not applicable due to Study termination
- Formulation development： Not applicable due to Study termination
- NDA: Not applicable due to Study termination
- Licensing out activity： Due diligence by Swedish Orphan Biovitrum AB(publ) and Kiniksa Pharmaceuticals

(출처 : SUMMARY 6p)

목차 Contents

• 표지 ... 1
• 제출문 ... 2
• 보고서 요약서 ... 3
• 요약문 ... 4
• SUMMARY ... 6
• CONTENTS ... 8
• 목차 ... 9
• 제1장 연구개발과제의 개요 ... 10
• 제2장 국내외 기술개발 현황 ... 15
• 가. HL2351 관련기술 ... 15
• 나. 지속형제제 개발 기술 ... 15
• 나. HL2351의 독창성 및 경쟁력 분석 ... 17
• 제3장 연구개발수행 내용 및 결과 ... 19
• 1. 1차 마일스톤 ... 19
• 2. 2차 마일스톤 ... 38
• 제4장 목표달성도 및 관련분야에의 기여도 ... 48
• 1. 1차 마일스톤 ... 48
• 2. 2차 마일스톤 ... 49
• 제5장 연구개발결과의 활용계획 ... 51
• 제6장 연구개발과정에서 수집한 해외과학기술정보 ... 52
• 제7장 연구시설·장비 현황 ... 53
• 제8장 참고문헌 ... 58
• 끝페이지 ... 59