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ORX750

Phase 2

Narcolepsy Type 1 | Small molecule | Neurology |Centessa Pharmaceuticals plc|Last Updated: Apr 27, 2026

Target and mechanism

Molecular targetOX2R
Target classReceptor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials2
Total Enrollment338

FDA Designations

No designations recorded

Clinical trial landscape

ORX750 · 2 trials · 3 indications

Phase 2 2
NCT07096674A Long-term Extension Study of ORX750 in Participants With Narcolepsy and Idiopathic HypersomniaNarcolepsy Type 1
RECRUITING90 Analytics
NCT06752668A Study of ORX750 in Participants With Narcolepsy and Idiopathic HypersomniaNarcolepsy Type 1
RECRUITING248 Analytics
PHASE2RECRUITING
A Long-term Extension Study of ORX750 in Participants With Narcolepsy and Idiopathic Hypersomnia
Narcolepsy Type 1Unlock trial analytics
PHASE2RECRUITING
A Study of ORX750 in Participants With Narcolepsy and Idiopathic Hypersomnia
Narcolepsy Type 1Unlock trial analytics

Study Endpoints

Primary Endpoints

Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
Up to day 70

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs

Abnormal Laboratory Tests
Up to day 70

Number of Participants With Abnormal Changes From Baseline in Laboratory Tests

Abnormal Vital Signs
Up to day 70

Number of Participants With Abnormal Change From Baseline in Vital Signs

Abnormal ECG
Up to day 70

Number of Participants With Abnormal Change From Baseline in Electrocardiograms (ECG)

Suicidal Ideation or Behavior
Up to day 70

Number of Participants With Suicidal Ideation or Suicidal Behavior as Measured Using Columbia-Suicide Severity Rating Scale (C-SSRS)

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs
Up to Day 35
Number of Participants With Abnormal Changes From Baseline in Laboratory Tests
Up to Day 35
Number of Participants With Abnormal Change From Baseline in Vital Signs
Up to Day 35
Number of Participants With Abnormal Change From Baseline in Electrocardiograms (ECG)
Up to Day 35
Number of Participants With Suicidal Ideation or Suicidal Behavior as Measured Using Columbia-Suicide Severity Rating Scale (C-SSRS)
Up to Day 35

Secondary Endpoints

Cmax
Up to day 63
Tmax
Up to day 63
AUClast
Up to day 63
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Narcolepsy Type 1EXPERIMENTALNarcolepsy Type 1 (Open Label)
Narcolepsy Type 2EXPERIMENTALNarcolepsy Type 2 (Open Label)
Idiopathic HypersomniaEXPERIMENTALIdiopathic Hypersomnia (Open Label)
Narcolepsy Type 1: ORX750 and PlaceboEXPERIMENTAL -
Narcolepsy Type 2: ORX750 and PlaceboEXPERIMENTAL -
Idiopathic Hypersomnia: ORX750 and PlaceboEXPERIMENTAL -

Interventions

NameTypeDescription
ORX750DRUGOral ORX750
PlaceboDRUGORX750 matching placebo capsule.
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites24

Inclusion Criteria: * Diagnosis of narcolepsy (NT1 or NT2) or IH who was eligible for and completed the full treatment period in the eligible parent study ORX750-0201 (CRYSTAL-1) * Is willing and able to adhere to additional protocol requirements Exclusion Criteria: * Development of any new disea...

Countries:United StatesCanadaItalySpainFrance
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Frequently asked questions about ORX750

What is ORX750 used for in Narcolepsy Type 1?

ORX750 is an investigational small molecule being developed for the treatment of Narcolepsy Type 1. It is currently in Phase 2 clinical trials, which also include participants with Narcolepsy Type 2 and Idiopathic Hypersomnia. The drug is being studied as a potential therapy to address symptoms associated with these sleep disorders.

What does ORX750 target?

ORX750 targets the OX2R receptor, which is the orexin 2 receptor. This receptor plays a role in regulating wakefulness and sleep. By targeting OX2R, ORX750 is designed to potentially modulate the orexin system, which is implicated in narcolepsy and related hypersomnia conditions.

Who makes ORX750?

ORX750 is being developed by Centessa Pharmaceuticals plc, a biopharmaceutical company. The company's stock is traded under the ticker symbol CNTA. Centessa is advancing ORX750 through clinical development for sleep disorders, including Narcolepsy Type 1.

What phase is ORX750 in?

ORX750 is currently in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. Two Phase 2 trials are actively recruiting participants to evaluate the drug's safety and efficacy in narcolepsy and idiopathic hypersomnia.

What clinical trials is ORX750 in?

ORX750 is being studied in two Phase 2 clinical trials. The first trial, NCT06752668, is a study in participants with narcolepsy and idiopathic hypersomnia, with an enrollment of 248. The second trial, NCT07096674, is a long-term extension study with an enrollment of 90. Both trials are currently recruiting.

Is ORX750 the same as any other drug?

ORX750 is a distinct investigational drug and is not known to be the same as any other marketed medication. It is being developed specifically for narcolepsy and idiopathic hypersomnia. No alternative names for ORX750 have been identified in the available clinical trial information.