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CTI-1601

Phase 2

Friedreich Ataxia | Monoclonal antibody | Neurology |Larimar Therapeutics, Inc.|Last Updated: May 13, 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials4
Total Enrollment168
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06447025An Open-Label Study of CTI-1601 in Subjects With Friedreich's AtaxiaPHASE2 RECRUITING 85Jan 25, 2024Jan 1, 2027May 13, 20268 United States
NCT05579691A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's AtaxiaPHASE2 COMPLETED 28Sep 21, 2022Dec 4, 2023Nov 29, 20241 United States
NCT04519567Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's AtaxiaPHASE1 COMPLETED 27Jul 31, 2020Mar 16, 2021Jun 30, 20211 United States
NCT04176991Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's AtaxiaPHASE1 COMPLETED 28Dec 11, 2019Oct 31, 2020Nov 12, 20201 United States
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Study Endpoints
Primary Endpoints
Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum Severity
Up to 24 months

Number of subjects

Change from baseline in electrocardiogram (ECG) parameters including, but not limited to, HR, RR interval, PR interval, QRS duration, QT interval, and QTcF interval
Up to 24 months

Number change in ECG parameters

Change from baseline in left ventricular ejection fraction (LVEF)
Up to 24 months

LVEF indicates the percentage of change in LV volume from diastole to systole that measures how well the left ventricle of the heart pumps blood.

Change from baseline in left ventricular end-diastolic volume (LVEDV)
Up to 24 months

LVEDV is the amount of blood, measured in milliliters (mL), in the heart's left ventricle just before the heart contracts.

Number of subjects with any suicidal ideation or behavior (Categories 1-10) of the Columbia Suicide Severity Rating Scale (C-SSRS)
Up to 24 months

The Columbia Suicide Severity Rating Scale (C-SSRS) is a tool used to assess the occurrence, severity, and frequency of suicidal thoughts and behaviors. A higher score on the C-SSRS generally indicate a worse outcome, as they signify a higher level of suicidal ideation or behavior.

Change from baseline at each collection timepoint in tissue frataxin concentrations normalized to total protein observed in buccal cells collected from cheek swabs and skin cells collected from skin punch biopsies
Up to 24 months
Change from baseline in motor function as assessed by 9-hole peg test (9-HPT)
Up to 24 months
Change from baseline in motor function as assessed by the timed 25-foot walk test (T25-FW)
Up to 24 months
Change from baseline in neurologic function as assessed by the modified Friedreich's Ataxia Rating Scale (mFARS) total score
Up to 24 months

The Modified Friedreich's Ataxia Rating Scale (mFARS) is a modified neurologic scale involving direct subject participation and targets specific areas impacted by Friedreich's ataxia (bulbar, upper limb, lower limb, and upright stability), with scores ranging from 0-67 points, with higher scores indicating a greater level of disability.

Change from baseline in neurologic function as assessed by the upright stability subscale examination of the mFARS
Through study completion, up to 24 months

The Upright Stability Subscale is an assessment of an individual's ability to maintain balance and stability while standing upright. It has a minimum value of 0 and a maximum value of 36. A higher score indicates a better outcome, reflecting greater stability and balance abilities while standing upright.

Change in activities of daily living (ADLs) as assessed by the Friedreich's Ataxia Rating Scale Activities of Daily Living (FARS_ADL)
Up to 24 months

The FARS\_ADL, scored 0 to 36, is a subscale of FARS assessing a subject's ability to complete activities of daily living. A higher score indicates a greater level of disability. The FARS\_ADL questionnaire will be performed at the timepoints indicated in protocol.

Change from baseline in total fatigue score and all the subscale scores as assessed by the Fatigue Impact Scale (MFIS)
Up to 24 months

The Modified Fatigue Impact Scale (MFIS) is a revised form of the Fatigue Impact Scale based on items derived from interviews with MS patients concerning how fatigue impacts their lives. This instrument provides an assessment of the effects of fatigue in terms of physical, cognitive, and psychosocial functioning. Participants rate on a 5-point scale, with 0 = 'Never' to 4 = 'Almost always' their agreement with 21 statements. Total score (0-84) and subscales for physical (0-36), cognitive (0-40) and psychosocial functioning (0-8). The 5-item version is scored (0-20). Higher numbers indicate greater fatigue. The MFIS will be performed at the timepoints indicated in protocol.

Change from baseline in the assessment of disease as assessed by the Functional Staging for Ataxia
Up to 24 months
Overall impression of change as assessed by the patient using the Patient Global Impression of Change (PGI-C) Scale
Up to 24 months

The Patient Global Impression of Change (PGI-C) reflects a patient's assessment about the efficacy of treatment. PGIC is a 7 point scale depicting a patient's rating of overall improvement. Patients rate their change as "very much improved," "much improved," "minimally improved," "no change," "minimally worse," "much worse," or "very much worse." The PGI-C will be performed at the timepoints indicated in protocol.

Overall impression of change assessed by a clinician using the Clinical Global Impression of Change (CGI-C)
Up to 24 months

The Clinical Global Impression of Change (CGI-C) is an assessment to measure change in clinical status (symptoms and functional ability) of the subject's condition from baseline with study drug. CGI-C scores range from 1 (very much improved) through to 7 (very much worse). The CGI-C will be performed at the timepoints indicated in protocol.

Area under the concentration-time curve for the dosing interval (AUC0-tau)
Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months
Area under the concentration-time curve from time 0 to the time of last quantifiable concentration (AUC0-t)
Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months
Mean maximum observed concentration (Cmax)
Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months
Mean time of maximum observed concentration (Tmax)
Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months
Concentration reached immediately before the next dose is administered (Ctrough)
Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months
Number of Participants with Treatment Emergent Adverse Events
Through study completion, an average of 93 days

Overall summary of Participants with Treatment Emergent Adverse Events

Number of Participants with Treatment Emergent Adverse Events by System Organ Classification and Preferred Term
Through study completion, an average of 75 days

Overall summary of Participants with Treatment Emergent Adverse Events by System Organ Classification (MedDRA version 23.0)

Number of Participants with Treatment-Emergent Adverse Events
Through study completion, an average of 70 days

Overall summary of the Participants with Treatment Emergent Adverse Events

Number of Treatment Emergent Adverse Events by System Organ Classification and Preferred Term
Through study completion, an average of 70 days

Overall summary of Participants with Treatment Emergent Adverse Events by System Organ Classification (MedDRA version 22.0)

Secondary Endpoints
Maximum observed plasma concentration (Cmax) of CTI-1601 after multiple doses
At baseline and up to 29 days
Area under the concentration time curve (AUC) of CTI-1601 from time 0 through the last measurable time point
At baseline and up to 29 days
Time to maximum observed plasma concentration (tmax) of CTI-1601 after multiple doses
At baseline and up to 29 days
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CTI-1601EXPERIMENTALOnce daily subcutaneous injection of 50 mg CTI-1601 in subjects ≥ 18 years of age or a weight-based dose of 0.8 mg/kg up to a maximum of 50 mg in subjects ≥ 2 to 17 years of age.
CTI-160lEXPERIMENTALCTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
PlaceboPLACEBO_COMPARATORPlacebo Comparator
Interventions
NameTypeDescription
CTI-1601DRUGCTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in patients with Friedreich's ataxia
PlaceboOTHERPlacebo Comparator
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Eligibility Criteria
Age Range2 Years — 60 Years
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * Subjects with FRDA who have or have not previously completed participation in a study of CTI-1601 are eligible to participate in this study unless the subject experienced one or more of the following in a previous CTI-1601 study: a) serious adverse event (SAE) related to study...

Countries:United States
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT06447025Status: ENROLLING_BY_INVITATION → RECRUITING
LOWMay 24, 2026NCT06447025studyFirstPostDate: changed