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GHR-LRX

Phase 2

Acromegaly | Small molecule | Endocrine |Ionis Pharmaceuticals, Inc.|Last Updated: Oct 3, 2024

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
RandomizedCONTROLLEDBiomarker
Total Trials1
Total Enrollment34
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04522180A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With AcromegalyPHASE2 COMPLETED 34Jan 4, 2021May 4, 2023Oct 3, 202422 United States, Estonia +8
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Study Endpoints
Primary Endpoints
Percent Change in Insulin-like Growth Factor I (IGF-1) From Baseline to Week 27
Baseline to Week 27

IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. Baseline of IGF-1 is defined as the average value of Screening and Day 1. A negative percent change from Baseline indicated improvement.

Secondary Endpoints
Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.2 Times Gender and Age Limits at Day 183 (Week 27)
At Week 27
Percentage of Participants Who Achieve Normalized IGF-1 Levels to Within 1.0 Times Gender and Age Limits at Day 183 (Week 27)
At Week 27
Change From Baseline in Serum IGF-1 Over Time
Up to approximately 80 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
GHR-LRX 120 mgEXPERIMENTALParticipants received GHR-LRX 120 mg subcutaneous (SC) injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3).
GHR-LRX 160 mgEXPERIMENTALParticipants received GHR-LRX 160 mg SC injection once every month for 73 weeks with a booster dose administered on Day 15 (Week 3).
Interventions
NameTypeDescription
GHR-LRXDRUGGHR-LRX was administered by SC injection.
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites22

Inclusion Criteria: 1. Males or females with a documented diagnosis of Acromegaly\* who are 18 to 75 years old (inclusive) at the time of informed consent. 2. Have had pituitary surgery (e.g. transsphenoidal) unless there was a contraindication to surgery and are either acromegaly medical treatment...

Countries:United StatesEstoniaHungaryItalyLatviaLithuaniaPolandRomaniaRussiaSerbia
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