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CPK850

Phase 1

Retinitis Pigmentosa | Monoclonal antibody | Ophthalmology |Novartis AG|Last Updated: Jan 15, 2026

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
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment12
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03374657A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis PigmentosaPHASE1 ACTIVE NOT_RECRUITING 12Aug 22, 2018May 11, 2026Jan 15, 20261 Sweden
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Study Endpoints
Primary Endpoints
Number of participants with adverse events (AEs), serious adverse events (SAEs) and deaths
Up to year 5

Safety events

Number of responders in dark adaptation
Screening/baseline up to year 1

A patient is considered a responder if sensitivity recovery values at 1 hour post-bleach are observed to be outside of the patient's prediction interval at ≥2 consecutive post-treatment visits within one year after treatment.

Secondary Endpoints
Number of patients with recovery of the cone system
Screening/baseline up to year 1
Number of patients with improvement in rod function in the treated eye vs the untreated eye
Screening/baseline up to year 1
Change from screening/baseline in Visual field perimetry mean deviation
Screening/baseline up to year 1
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingSINGLE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CPK Dose 1 (lowest dose)EXPERIMENTALCPK850, one subretinal injection to the study eye
CPK Dose 2 (next lowest dose)EXPERIMENTALCPK850, one subretinal injection to the study eye
CPK Dose 3 (third lowest dose)EXPERIMENTALCPK850, one subretinal injection to the study eye
CPK Dose 4 (highest dose)EXPERIMENTALCPK850, one subretinal injection to the study eye
Interventions
NameTypeDescription
CPK850BIOLOGICALIn one of 4 dose levels administered via subretinal injection under anesthesia
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Eligibility Criteria
Age Range18 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Male and female patients aged 18 to 70 years inclusive. * The visual acuity in the study eye at the screening 1 visit should be no better than 60 ETDRS letters. * Clinical diagnosis of Bothnia dystrophy, Newfoundland rod-cone dystrophy or other progressive retinitis pigmentosa...

Countries:Sweden
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT03374657primaryCompletionDate: changed
LOWMay 24, 2026NCT03374657studyFirstPostDate: changed