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BVS857

Phase 2

Spinal and Bulbar Muscular Atrophy | Small molecule | Neurology |Novartis AG|Last Updated: Jan 5, 2021

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment37

FDA Designations

No designations recorded

Clinical trial landscape

BVS857 · 2 trials · 2 indications

Phase 2 1Phase 1 1
NCT02024932Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular AtrophySpinal and Bulbar Muscular Atrophy
COMPLETED37 Analytics
PHASE2COMPLETED
Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy
Spinal and Bulbar Muscular AtrophyUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Patients With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths as a Measure of Safety and Tolerability
After 78 days in Part A and after 85 days in Part B.

Safety was monitored throughout the study.

Number of Mild, Moderate and Severe Adverse Events as a Measure of Safety and Tolerability
After 78 days in Part A and after 85 days in Part B.

Safety was monitored throughout the study.

Mean Percent Change From Baseline in Thigh Muscle Volume in Part B, Cohort 5
Baseline, Day 85

Thigh muscle volume was assessed by magnetic resonance imaging (MRI). Change from baseline was calculated from the ratio of the post-baseline mean value to the baseline mean value: \[(Day 85/baseline) - 1)\] x 100. A positive change from baseline indicates improvement.

Blood glucose
Continuously up to 48 hours

Secondary Endpoints

Mean Change From Baseline in Score on the Adult Myopathy Assessment Tool (AMAT) in Part B, Cohort 5
Baseline, Day 85
Mean Change From Baseline in Total Lean Body Mass (LBM) in Part B, Cohort 5
Baseline, Day 85
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part A, Cohort 1
Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
BVS857 Part A Open label (Cohort 1)EXPERIMENTALParticipants received single doses of 0.01 mg/kg BVS857 intravenously (i.v.) on day 1, 0.01 mg/kg BVS857 subcutaneously (s.c.) on day 15, 0.03 mg/kg BVS857 s.c. on day 29, 0.06 mg/kg BVS857 s.c. on day 43 and 0.10 mg/kg BVS857 s.c. on day 57.
BVS857 Part A double blind (Cohort 2)EXPERIMENTALParticipants received single doses of 0.03 mg/kg BVS857 i.v on day 1, 0.03 mg/kg BVS857 s.c. on day 15, 0.06 mg/kg BVS857 s.c. on day 29, 0.10 mg/kg BVS857 s.c. on day 43 and 0.10 mg/kg BVS857 s.c. on day 57. (BVS857 concentrations differed on days 43 and 57.)
Placebo Part A double blind (Cohort 2)PLACEBO_COMPARATORParticipants received single doses of matching placebo i.v. on day 1 and matching placebo s.c. on days 15, 29, 43 and 57.
BVS857 Part B open-label (Cohort 4)EXPERIMENTALParticipants received 0.1 mg/kg BVS857 i.v. weekly for 12 weeks.
BVS857 Part B double blind (Cohort 5)EXPERIMENTALParticipants received 0.06 mg/kg (maximum 6 mg) BVS857 i.v. weekly for 12 weeks.
Placebo Part B double blind (Cohort 5)PLACEBO_COMPARATORParticipants received matching placebo i.v. to BVS857 weekly for 12 weeks.
BVS857EXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -

Interventions

NameTypeDescription
BVS857DRUGBVS857 lyophilisate in vial; the lyophilisate was reconstituted with sterile water for injection, diluted as appropriate, and administered either i.v. or s.c..
PlaceboDRUGPlacebo lyophilisate in vial; the lyophilisate was reconstituted with sterile water for injection, diluted as appropriate, and administered either i.v. or s.c..
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Eligibility Criteria

Age Range18 Years to N/A
SexMALE
Healthy VolunteersNo
Study Sites6

Key Inclusion Criteria: * Genetic diagnosis of SBMA with symptomatic muscle weakness * Able to complete 2 minute timed walk * Serum IGF-1 level less than or equal to 170 ng/mL Key Exclusion Criteria: * Medically treated diabetes mellitus or known history of hypoglycemia * History of Bell's palsy ...

Countries:United StatesDenmarkGermanyItaly
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Frequently asked questions about BVS857

What is BVS857 used for?

BVS857 is an investigational small molecule being studied for insulin resistance and spinal and bulbar muscular atrophy (SBMA). It has been evaluated in clinical trials for both conditions, including a Phase 1 study in insulin resistance and a Phase 2 study in SBMA.

Who makes BVS857?

BVS857 is being developed by Novartis AG, a multinational pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS. Novartis has sponsored clinical trials of BVS857 in the United States and other countries.

What phase is BVS857 in?

BVS857 has completed a Phase 1 trial in insulin resistance and a Phase 2 trial in spinal and bulbar muscular atrophy. Both trials are completed, and BVS857 remains an investigational drug that has not been approved by regulatory authorities.

What clinical trials is BVS857 in?

BVS857 has been studied in two completed clinical trials. NCT01435330 was a Phase 1, randomized, double-blind, placebo-controlled study of 43 participants with insulin resistance in the United States. NCT02024932 was a Phase 2 study of 37 male participants with spinal and bulbar muscular atrophy in the United States, Denmark, Germany, and Italy.

Is BVS857 the same as any other drug?

No alternative names for BVS857 have been reported. It is identified by its code name BVS857 in clinical trial registries and is not known to be marketed under any other brand name.