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Fx-1006A · 5 trials · 6 indications
TTR tetramer was assessed using a validated immunoturbidimetric assay. The Fraction of Initial (FOI) is the ratio of the measured TTR tetramer concentration after denaturation to the measured TTR tetramer concentration before denaturation. TTR tetramer stabilization is based on the difference between the on-treatment FOI and the baseline FOI expressed as a percentage of the baseline FOI.
Response to treatment indicated by either improvement(decrease from baseline) or stabilization(change from baseline of 0 to less than\[\<\] 2) in NIS-LL score,based on mean of 2 scores in 1 week period.NIS-LL assessed muscle weakness,reflexes,sensation.Each item scored separately for left,right limbs.Components of muscle weakness:0(normal)-4(paralysis),higher score=more weakness;reflexes,sensation:0=normal,1=decreased,or 2=absent.Total NIS-LL score range 0-88,higher score=more impairment. For tafamidis-tafamidis group, NIS-LL baseline value of previous study FX-005(NCT00409175) used as reference.
Response to treatment indicated by either improvement(decrease from baseline) or stabilization(change from baseline of 0 to less than\[\<\] 2) in NIS-LL score,based on mean of 2 scores in 1 week period.NIS-LL assessed muscle weakness,reflexes,sensation.Each item scored separately for left,right limbs.Components of muscle weakness:0(normal)-4(paralysis),higher score=more weakness;reflexes,sensation:0=normal,1=decreased,or 2=absent.Total NIS-LL score range 0-88,higher score=more impairment. For tafamidis-tafamidis group, NIS-LL baseline value of previous study FX-005(NCT00409175) used as reference.
Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptoms was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life.
Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptoms was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life.
TTR tetramer was assessed using a validated immunoturbidimetric assay. The Fraction of Initial (FOI) is the ratio of the measured TTR tetramer concentration after denaturation to the measured TTR tetramer concentration before denaturation. TTR tetramer stabilization is based on the difference between the on-treatment FOI and the baseline FOI expressed as a percentage of the baseline FOI.
Response to treatment was indicated by either improvement (decrease from baseline) or stabilization (change from baseline of 0 to less than\[\<\] 2) in NIS-LL score, based on mean of 2 scores in 1 week period. NIS-LL: assessed muscle weakness, reflexes, sensation. Each item scored separately for left, right limbs. Components of muscle weakness scored on 0(normal) to 4(paralysis) scale, higher score=greater weakness. Components of reflexes, sensation scored 0=normal, 1=decreased, or 2=absent. Total NIS-LL score range 0-88, higher score=greater impairment.
Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of diabetic neuropathy on the quality of life of participants with diabetic neuropathy; Item 1 to 7: related to symptoms and presence of symptom was assessed as 1 and absence was assessed as 0. Item 8-35: related to activities of daily living and scored on a 5-point Likert scale, where 0= no problem and 4= severe problem (except item 32, where -2= much better, 0=about the same, 2=much worse). TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life.
| Arm | Type | Description |
|---|---|---|
| Fx-1006A | EXPERIMENTAL | - |
| 1 | EXPERIMENTAL | Fx-1006A 20mg soft gelatin capsules once daily for 12 months |
| 1. | EXPERIMENTAL | Fx-1006A |
| 2. | PLACEBO_COMPARATOR | Placebo |
| Mild and Moderate Hepatic Dysfunction | ACTIVE_COMPARATOR | Patients with mild and moderate hepatic dysfunction |
| Healthy Volunteers | ACTIVE_COMPARATOR | Healthy volunteers |
| Name | Type | Description |
|---|---|---|
| Fx-1006A | DRUG | Fx-1006A 20mg soft gelatin capsules once daily (at the same time each day) for 12 months |
| Placebo | DRUG | Fx-1006A 20mg or matched placebo once daily (at the same time each day) for a period of 18 Months |
Inclusion Criteria: 1. Patient is \> 40 years-old. 2. Patient participated in FoldRx Study Fx-001 (TRACS) OR Patient has documented TTR amyloid cardiomyopathy and NYHA Classification of I or II. TTR amyloid cardiomyopathy is defined as: 1. Variant TTR amyloid cardiomyopathy as defined as: V1...
Fx-1006A is an investigational small molecule being developed for transthyretin-associated amyloidosis with polyneuropathy, familial amyloid polyneuropathy, cardiomyopathy, and mild hepatic dysfunction. It has been studied in Phase 2 clinical trials for these conditions, though it remains investigational and is not approved.
Fx-1006A targets transthyretin, a protein involved in amyloidosis. It is designed to stabilize transthyretin, potentially preventing the formation of amyloid deposits that cause disease. This mechanism is being evaluated in patients with transthyretin amyloidosis affecting the nerves and heart.
Fx-1006A is being developed by Pfizer, Inc., a biopharmaceutical company listed on the New York Stock Exchange under the ticker symbol PFE. Pfizer has sponsored clinical trials evaluating the drug's safety and efficacy in transthyretin amyloidosis.
Fx-1006A has completed Phase 2 clinical trials for transthyretin amyloidosis with polyneuropathy and cardiomyopathy. It has also completed a Phase 1 trial in hepatic dysfunction. The drug is investigational and has not received regulatory approval.
Fx-1006A has been studied in completed trials including NCT00630864 for non-V30M transthyretin amyloidosis with polyneuropathy, NCT00694161 for V122I or wild-type TTR amyloid cardiomyopathy, NCT00791492 as an extension study in familial amyloid polyneuropathy, and NCT01358565 for hepatic dysfunction.
Fx-1006A is also known as tafamidis, a transthyretin stabilizer developed by Pfizer. Clinical trials have evaluated it under the Fx-1006A name for amyloidosis, and it is the same compound marketed under the brand name Vyndaqel or Vyndamax.