Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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...
Top 11 of 15 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Bristol-Myers Squibb Company | BMY | 13 | PHASE3 | Mavacamten |
| Cytokinetics, Incorporated | CYTK | 3 | PHASE3 | Aficamten |
| Lexicon Pharmaceuticals, Inc. | LXRX | 1 | PHASE3 | Sotagliflozin |
| Lexeo Therapeutics, Inc. | LXEO | 6 | PHASE2 | LX2006, LX2020 |
| BridgeBio Pharma, Inc. | BBIO | 2 | PHASE3 | Acoramidis |
| Edgewise Therapeutics, Inc. | EWTX | 1 | PHASE2 | EDG-7500 |
| AstraZeneca PLC | AZN | 4 | PHASE1 | AZD4063, ALXN2350 |
| Tenaya Therapeutics, Inc. | TNYA | 4 | PHASE1 | TN-401, TN-201 |
| Bayer | BAYRY | 1 | PHASE3 | BAY3723113 |
| Rocket Pharmaceuticals, Inc. | RCKT | 4 | PHASE1 | RP-A601, RP-A701 |
| Atrium Therapeutics, Inc. | RNA | 1 | PHASE1 | ATR 1072 |
Fx-1006A is an investigational small molecule being developed for transthyretin-associated amyloidosis, including familial amyloid polyneuropathy, transthyretin-associated amyloidosis with polyneuropathy, and cardiomyopathy. It has been studied in patients with these conditions, as well as in those with mild hepatic dysfunction.
Fx-1006A is being developed by Pfizer, Inc., a biopharmaceutical company listed on the New York Stock Exchange under the ticker symbol PFE. The drug is currently in Phase 2 clinical development for transthyretin amyloidosis indications.
Fx-1006A is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Clinical trials have been completed, but the drug remains under investigation for the treatment of transthyretin-associated amyloidosis.
Fx-1006A has been studied in several completed clinical trials, including NCT00630864 in patients with non-V30M transthyretin amyloidosis with polyneuropathy, NCT00694161 in patients with V122I or wild-type TTR amyloid cardiomyopathy, NCT00791492 as an extension study in transthyretin amyloid polyneuropathy, and NCT01358565 in subjects with hepatic dysfunction.
Fx-1006A targets transthyretin, a protein that can misfold and accumulate as amyloid deposits in tissues, leading to polyneuropathy and cardiomyopathy. The drug is designed to stabilize transthyretin, preventing the formation of amyloid fibrils that cause disease.
Fx-1006A is also known as tafamidis, a name that may be used in clinical literature and regulatory filings. Pfizer has studied this compound under both names, and it is the same investigational small molecule for transthyretin amyloidosis.