Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ALN-TTR01 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| ALN-TTR01 | ACTIVE_COMPARATOR | - |
| Sterile Normal Saline (0.9% NaCl) | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| ALN-TTR01 | DRUG | Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion |
| Sterile Normal Saline (0.9% NaCl) | DRUG | Calculated volume to match active comparator |
Inclusion Criteria: * Diagnosis of TTR amyloidosis * Adequate blood counts, liver and renal function * Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control * Males agree to use appropriate contraception * Wi...
ALN-TTR01 is an investigational drug being developed for transthyretin mediated amyloidosis (ATTR), a rare disease. It is intended to treat TTR-mediated amyloidosis, a condition where abnormal transthyretin protein deposits cause organ damage. The drug is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
ALN-TTR01 is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company. Alnylam's stock is listed under the ticker symbol ALNY. The company is conducting clinical trials to evaluate the safety and tolerability of ALN-TTR01 in patients with transthyretin mediated amyloidosis.
ALN-TTR01 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The drug is being studied in early-stage clinical trials to assess its safety, tolerability, and pharmacokinetics in humans.
ALN-TTR01 has been studied in two completed Phase 1 clinical trials. The first trial, NCT01148953, evaluated safety and tolerability in 32 patients with transthyretin mediated amyloidosis across France, Portugal, Sweden, and the United Kingdom. The second trial, NCT01559077, assessed safety, tolerability, and pharmacokinetics in 17 healthy volunteers in the United Kingdom.
No, ALN-TTR01 and ALN-TTR02 are different investigational drugs. Both are being developed by Alnylam Pharmaceuticals for transthyretin mediated amyloidosis, but they are distinct compounds. ALN-TTR01 was studied in patients with ATTR, while ALN-TTR02 was evaluated in healthy volunteers in a separate Phase 1 trial.