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Myobloc

Phase 3

Sialorrhea | Small molecule | Neurology |Supernus Pharmaceuticals, Inc.|Last Updated: Mar 7, 2023

Target and mechanism

ModalitySmall molecule

Also known as Phase 2; Low Dose MYOBLOC, Phase 3; MYOBLOC

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment374

FDA Designations

No designations recorded

Clinical trial landscape

Myobloc · 3 trials · 7 indications

Phase 3 2Phase 2 1
NCT02610868Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in AdultsSialorrhea
COMPLETED187 Analytics
NCT01994109Efficacy and Safety Study of MYOBLOC® Followed by Open-Label Multiple-Treatment With MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adult SubjectsSialorrhea
COMPLETED187 Analytics
PHASE3COMPLETED
Open-Label and Single-Arm Study of MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adults
SialorrheaUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety Study of MYOBLOC® Followed by Open-Label Multiple-Treatment With MYOBLOC® in the Treatment of Troublesome Sialorrhea in Adult Subjects
SialorrheaUnlock trial analytics

Study Endpoints

Primary Endpoints

Occurrence, Seriousness, Severity, and Causality Assessment of Treatment Emergent Adverse Events (TEAE)
Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1

TEAEs Related to Study Medication include TEAEs classified as Possibly, Probably and Definitely Related. Treatment Session (TS)

Occurrence of Adverse Events of Special Interest (AESI)
Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1

Treatment Session (TS) AESI includes: Aspiration, Aspirational pneumonia, Choking and Dysphagia.

Columbia Suicide Rating Scale (C-SSRS): Suicidal Ideation, Behavior Scores and Intensity Scores
Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13

Participants Analyzed does not match the Participant Flow because one participant discontinued prior to the first post-injection visit. Per protocol participants were only required to complete two treatment sessions with a maximum of 4 treatment sessions allowed. All subjects received 3500 U doses except for 13 subjects who had decreased doses at Treatment Session 2 and/or 3 (2500 U to 3000 U).

Occurrence of Dental Adverse Events
Visits at Weeks 4 and 13 combined for each TS; including Visit at Week 8 for TS1

Treatment Session (TS)

Unstimulated Salivary Flow Rate (USFR) at Week 4 Post-injection Visit (Part A)
4 Weeks

Change weight of expectorated saliva at a Week 4 post-injection visit.

Clinical Global Impression Change (CGI-C) at Week 4 Post-injection (Part A)
4 weeks

CGI-C was assessed on a 7-point scale ranging from "very much improved" to "very much worse" with 1 assigned to "very much improved" and 7 assigned to "very much worse"; ranging from a minimum score of 1 and a maximum score of 7.

Effect of MYOBLOC on the Modified Ashworth Scale (MAS) for tone of the Primary Target Muscle Group (PTMG) [Phase 2 and Phase 3]
Baseline and Week 4

The first co-primary endpoint is the change from baseline in Modified Ashworth Scale (MAS) score for tone of the Primary Target Muscle Group (PTMG) selected for treatment at Week 4 post-injection. The MAS is an internationally accepted and validated instrument used to measure resistance during passive soft-tissue stretching. Resistance will be measured and recorded using a 6-point scale ranging from 0 (no increase in muscle tone) to 4 (affected part\[s\] rigid in flexion or extension). A lower change from baseline MAS score (\<0) represents a better outcome.

Effect of MYOBLOC on Clinical Global Impression of Change (CGI-C) Scale [Phase 2 and Phase 3]
Week 4

The second co-primary endpoint is the Clinical Global Impression of Change (CGI-C) score in functional ability at Week 4 post-injection. The CGI-C scale is a single item clinician assessment of how much the patient's functional ability has improved, worsened or has not changed relative to the patient's baseline state prior to treatment (injection). The CGI-C is rated on a 7-point Likert scale from 1 to 7, where 1 = "Very much improved", 2 = "Much improved", 3 = "Minimally improved", 4 = "No change", 5 = "Minimally worse", 6 = "Much worse", and 7 = "Very much worse". Successful therapy is indicated by a lower score (\<4) in subsequent testing.

Secondary Endpoints

Change From Baseline in Unstimulated Salivary Flow Rate (USFR): Treatment Sessions 1-4
Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Change From Baseline in Clinical Global Impression of Severity (CGI-S)
Baseline (Day 1) of each TS to the following: TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
Clinical Global Impression of Change (CGI-C)
TS1 Visits at Weeks 4, 8, 13; TS2-4 Visits at Weeks 4, 13
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
MYOBLOC InjectionEXPERIMENTALAfter a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections over the course of 1 year.
MYOBLOC 2500 UACTIVE_COMPARATORSubjects will receive specified dose of MYOBLOC
MYOBLOC 3500 UACTIVE_COMPARATORSubjects will receive specified dose of MYOBLOC
PlaceboPLACEBO_COMPARATORSubjects will receive volume matched Placebo
Phase 2; Low Dose MYOBLOCEXPERIMENTALLow Dose MYOBLOC is a single treatment and will be compared to volume-matched placebo
Phase 2; High Dose MYOBLOCEXPERIMENTALHigh Dose MYOBLOC is a single treatment and will be compared to volume-matched placebo
Phase 2; PlaceboPLACEBO_COMPARATORVolume-matched placebo is a single treatment
Phase 3; MYOBLOCEXPERIMENTALMYOBLOC is a single treatment and will be compared to volume-matched placebo
Phase 3; PlaceboPLACEBO_COMPARATORVolume-matched placebo is a single treatment

Interventions

NameTypeDescription
MYOBLOCDRUGAfter a screening period (up to 21 days), subjects who satisfy all eligibility criteria may receive single dose injections of 3,500 Units of MYOBLOC every 13 weeks (±2 weeks) for a total of 4 treatments via injections into the submandibular and parotid glands. Subjects will undergo safety and effectiveness assessments at Weeks 4, 8, 13, 17, 26, 30, 39, 43, and 52, and telephone follow up will occur at Weeks 21, 34, and 47
PLACEBOOTHER -
Phase 2; Low Dose MYOBLOCDRUGIntramuscular injections on Day 1
Phase 2; High Dose MYOBLOCDRUGIntramuscular injections on Day 1
Phase 2; PlaceboDRUGIntramuscular injections on Day 1
Phase 3; MYOBLOCDRUGIntramuscular injections on Day 1
Phase 3; PlaceboDRUGIntramuscular injections on Day 1
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Eligibility Criteria

Age Range18 Years to 85 Years
SexALL
Healthy VolunteersNo
Study Sites35

Inclusion Criteria: * Seeking treatment for troublesome sialorrhea for at least 3 months that is occurring secondary to any disorder or related to any cause, including, but not limited to, Parkinson's Disease (PD), adult cerebral palsy, amyotrophic lateral sclerosis (ALS), stroke, traumatic brain i...

Countries:United StatesBelarusUkraineRussiaCzechia
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Frequently asked questions about Myobloc

What is MYOBLOC used for?

MYOBLOC is used for the treatment of troublesome sialorrhea, which is excessive drooling, in adult subjects. It is being developed by Supernus Pharmaceuticals, Inc. and is currently in Phase 3 clinical development for this indication.

Who makes MYOBLOC?

MYOBLOC is being developed by Supernus Pharmaceuticals, Inc., a company traded on the stock exchange under the ticker symbol SUPN. The drug is currently in Phase 3 clinical trials for the treatment of sialorrhea in adults.

What phase is MYOBLOC in?

MYOBLOC is in Phase 3 clinical development. Two Phase 3 trials have been completed for the treatment of troublesome sialorrhea in adult subjects. The drug is investigational and has not been approved by regulatory authorities.

What clinical trials is MYOBLOC in?

MYOBLOC has been studied in two completed Phase 3 clinical trials. The first trial, NCT01994109, enrolled 187 adults with sialorrhea and was conducted in the United States, Russia, and Ukraine. The second trial, NCT02610868, also enrolled 187 adults and took place in the United States, Belarus, and Ukraine.

Is MYOBLOC the same as other sialorrhea treatments?

MYOBLOC is a distinct drug being developed specifically for troublesome sialorrhea in adults. It is a small molecule administered in a placebo-controlled, double-blind, randomized trial setting. No alternative names for MYOBLOC have been reported.