Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
belcesiran · 1 trial · 1 indication
The incidence of adverse events (AE), serious adverse events (SAE), DLT, and AE leading to study drug discontinuation
The incidence of clinically significant physical examination (PE) findings
Absolute QTc \> 500 msec and/or QTc change of \> 60 msec from baseline will be evaluated
| Arm | Type | Description |
|---|---|---|
| belcesiran | EXPERIMENTAL | Healthy volunteers will be administered a single dose of belcesiran. |
| Placebo | PLACEBO_COMPARATOR | Healthy volunteers will be administered a single dose of matching placebo. |
| Name | Type | Description |
|---|---|---|
| belcesiran | DRUG | belcesiran will be administered subcutaneously (SC) at dose levels planned. |
| Placebo | DRUG | Sterile normal saline (0.9% NaCL) matching volume of belcesiran doses will be administered subcutaneously (SC). |
Inclusion Criteria: * Male or Female aged 18 to 55 years, inclusive. Female participants must be either surgically sterile or postmenopausal. No women of childbearing potential are eligible for enrollment. * Overtly Healthy, as determined by the investigator. * Serum A1AT protein concentration \>10...
Belcesiran is an investigational RNA therapy being developed by Novo Nordisk A/S for alpha 1-antitrypsin deficiency, a rare genetic disease. It is a small interfering RNA (siRNA) therapy and has completed a Phase 1 clinical trial. It is not yet approved for any indication.
Belcesiran is being developed as a potential treatment for alpha 1-antitrypsin deficiency. It is an investigational siRNA therapy designed to reduce production of the abnormal protein that causes the disease. It has not been approved for this or any other use.
Belcesiran is a small interfering RNA (siRNA) therapy. siRNA molecules work by targeting and degrading specific messenger RNA, which reduces production of the corresponding protein. In this case, belcesiran is designed to reduce alpha 1-antitrypsin protein production, addressing the underlying cause of alpha 1-antitrypsin deficiency.
Belcesiran is being developed by Novo Nordisk A/S, a pharmaceutical company traded on the New York Stock Exchange under the ticker NVO. Novo Nordisk is responsible for the clinical development program for belcesiran in alpha 1-antitrypsin deficiency.
Belcesiran is in Phase 1 clinical development. It has completed one Phase 1 trial and is not currently in any active trials. It is an investigational therapy and has not received FDA approval or approval from any other regulatory agency.
Belcesiran has been studied in one completed Phase 1 trial, NCT04174118, titled Study of DCR-A1AT in Healthy Adult Volunteers. The trial enrolled 30 healthy adult volunteers in New Zealand and Sweden and was randomized, double-blind, and placebo-controlled. No active trials are currently listed.