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ISIS 388626

Phase 1

Type 2 Diabetes Mellitus | Small molecule | Metabolic |Ionis Pharmaceuticals, Inc.|Last Updated: Feb 3, 2012

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment103
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00836225Safety, Tolerability and Activity Study of Multiple Doses of ISIS-SGLT2Rx in Healthy VolunteersPHASE1 COMPLETED 103Jan 1, 2009Jan 1, 2012Feb 3, 20121 Netherlands
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Study Endpoints
Primary Endpoints
To evaluate the safety and tolerability of single and multiple doses of ISIS-SGLT2Rx administered subcutaneously
30 days for single dose, 11 weeks for 6-week dosing, 18 weeks for 13-week dosing
Secondary Endpoints
Pharmacokinetic profile all doses and Pharmacodynamics for multi-dose groups
3 days for single dose, 11 weeks for 6-week dosing, 18 weeks for 13-week dosing
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AEXPERIMENTAL50 mg ISIS 388626 vs Placebo, s.c. injection
BEXPERIMENTAL100 mg ISIS 388626 vs Placebo, s.c. injection
CEXPERIMENTAL200 mg ISIS 388626 vs Placebo, s.c. injection
DEXPERIMENTAL400 mg ISIS 388626 vs Placebo, s.c. injection
AAEXPERIMENTAL50 mg ISIS 388626, 3x over 1 week s.c. injection, weekly s.c. injection for 5 weeks vs Placebo
BBEXPERIMENTAL100 mg ISIS 388626, 3x over 1 week s.c. injection, weekly s.c. injection for 5 weeks vs Placebo
AAAEXPERIMENTAL50 mg ISIS 388626, weekly s.c. injection for 13 weeks vs Placebo
BBBEXPERIMENTAL100 mg ISIS 388626, weekly s.c. injection for 13 weeks vs Placebo
CCCEXPERIMENTAL200 mg ISIS 388626, weekly s.c. injection for 13 weeks vs Placebo
FFFEXPERIMENTAL50 mg ISIS 388626, weekly s.c. injection for 13 weeks vs Placebo
Interventions
NameTypeDescription
ISIS 388626DRUG4 arms of single dose s.c. injections: A 50 mg, B 100 mg, C 200 mg, D 400 mg, 2 arms of multi-dose: 3 s.c. injections over 1 week followed by 5 weekly s.c. injections: AA 50 mg, BB 100 mg 3 arms of multi-dose 13 weekly s.c. injections: AAA 50 mg, BBB 100 mg, CCC 200 mg, FFF 50 mg Placebo: 0.9% sterile saline
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Age 18 to 65 years * Male or female gender although females must be post-menopausal or surgically sterile (hysterectomy, oophorectomy or tubal ligation) * Give written informed consent to participate in the study and availability for all study requirements * Fasting plasma glu...

Countries:Netherlands
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