nomlabofusp (CTI-1601) / Larimar Therap - LARVOL DELTA

FORWARD-FA: A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P3 | N=150 | Recruiting | Sponsor: Larimar Therapeutics, Inc.

New P3 trial • Ataxia • Friedreich ataxia • Movement Disorders

Nomlabofusp Treatment Produces Frataxin Levels That Correlate Across Peripheral Tissues: Preclinical and Clinical Support for Surrogate Tissue Sampling. (PubMed, Clin Transl Sci) - "Correlation patterns were maintained across species, supporting the robustness and translational relevance of the observed relationships. Collectively, these data support the use of peripheral tissues-particularly skin and buccal cells-for cross-sectional and longitudinal monitoring of hFXN supplementation in FRDA."

Journal • Preclinical • Ataxia • Friedreich ataxia • Movement Disorders

CLIN-1601-301: A Study to Determine if Nomlabofusp is Effective and Safe in Adults and Children with Friedreichs Ataxia (clinicaltrialsregister.eu) - P2/3 | N=18 | Not yet recruiting | Sponsor: Larimar Therapeutics Inc.

New P2/3 trial • Ataxia • Friedreich ataxia • Movement Disorders

Jive: An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P2 | N=85 | Recruiting | Sponsor: Larimar Therapeutics, Inc. | Enrolling by invitation ➔ Recruiting

Enrollment status • Ataxia • Friedreich ataxia • Movement Disorders

Larimar Therapeutics Announces FDA Breakthrough Therapy Designation for Nomlabofusp in FA (GlobeNewswire) - "The Breakthrough Therapy Designation request for nomlabofusp included preliminary clinical data from the OL study demonstrating increases in skin FXN to levels expected in asymptomatic carriers and consistent directional improvement across four key clinical outcomes including modified Friedreich Ataxia Rating Scale (mFARS) score, FARS-Activities of Daily Living (ADL), 9 Hole Peg Test (9-HPT), and Modified Fatigue Impact Scale (MFIS) after 1-year on treatment...We continue to plan for a June 2026 BLA submission seeking accelerated approval and are excited to initiate our confirmatory Phase 3 study in the U.S., E.U., U.K., Canada and Australia."

Breakthrough therapy • FDA filing • New P3 trial • Friedreich ataxia

A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia (clinicaltrials.gov) - P1 | N=18 | Terminated | Sponsor: Larimar Therapeutics, Inc. | N=30 ➔ 18 | Trial completion date: Aug 2025 ➔ Apr 2025 | Recruiting ➔ Terminated | Trial primary completion date: Aug 2025 ➔ Apr 2025; Review of Cohort 1 data facilitated direct enrollment into open label study and eliminate pause in study drug administration between PK study and open label study.

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Ataxia • Friedreich ataxia • Movement Disorders

Pharmacokinetics and Pharmacodynamics of Nomlabofusp in Non-clinical Studies of Friedreich's Ataxia. (PubMed, AAPS J) - "In the knockout mice, we show that the pharmacokinetics and processing of nomlabofusp were comparable with wild type animals and that treatment with nomlabofusp halts the progression of cardiac dysfunction and significantly increased survival. Together, the findings from these non-clinical studies demonstrate that nomlabofusp exposure increases human frataxin in Friedreich's ataxia-relevant tissues and provide evidence of pharmacologic effects."

Journal • PK/PD data • Ataxia • Friedreich ataxia • Movement Disorders

Larimar Therapeutics Announces FDA Recommendations on Safety Database, and Other Details of Nomlabofusp BLA Submission for Friedreich’s Ataxia Program (GlobeNewswire) - "Written FDA recommendations for safety database include a total of at least 30 participants with continuous exposure for 6 months including a subset of at least 10 with 1-year; large majority of the exposure should be on the 50 mg dose BLA submission seeking accelerated approval planned in the second quarter of 2026 to allow for inclusion of the recommended safety data for adults and children...OLE data expected in September 2025 from 30-40 participants who received at least one dose of nomlabofusp; data will include participants on the 50 mg dose Adolescent PK run-in data expected in September 2025 from 14 participants (some on placebo); participants currently screening and enrolling into OLE"

FDA event • FDA filing • Trial status • Friedreich ataxia

Nomlabofusp, a Fusion Protein of Human Frataxin and a Cell Penetrant Peptide, Delivers Mature and Functional Frataxin into Mitochondria. (PubMed, AAPS J) - "Finally, using buccal swab tissue samples taken from study subjects in a Phase 1 clinical trial who received nomlabofusp, we show increases in mature frataxin levels along with marked changes in gene expression post-administration suggesting intracellular pharmacodynamic activity. Together, these results demonstrate that nomlabofusp enters the cell and localizes to the mitochondria, releasing mature frataxin that appears to be biologically active and support the use of nomlabofusp as a potential treatment for patients with Friedreich's ataxia."

Journal • Ataxia • Friedreich ataxia • Genetic Disorders • Movement Disorders

Larimar Therapeutics Announces Dosing of Adolescents in Nomlabofusp Pediatric Pharmacokinetic Run-In Study for Patients with Friedreich’s Ataxia (GlobeNewswire) - "Larimar Therapeutics...announced that dosing of adolescents 12-17 years old has started in the Company’s pediatric PK run-in study for patients with Friedreich’s ataxia (FA)....Adolescents receive a weight-based dose equivalent to the 50 mg adult dose. Adolescents 12-17 years old who complete participation in the pharmacokinetic (PK) run-in study will be eligible to screen in the ongoing open label extension (OLE) study. Larimar is continuing to enroll adolescent patients and plans to initiate a cohort of children 2-11 years old in 1H 2025. Long-term 50 mg data from adults in OLE study and available adolescent PK run-in data expected mid-2025."

P1 data • P2 data • Trial status • Friedreich ataxia

A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia (clinicaltrials.gov) - P1 | N=30 | Recruiting | Sponsor: Larimar Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Ataxia • Friedreich ataxia • Movement Disorders

A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia (clinicaltrials.gov) - P1 | N=30 | Not yet recruiting | Sponsor: Larimar Therapeutics, Inc.

New P1 trial • Ataxia • Friedreich ataxia • Movement Disorders

Jive: An Open Label Extension Study of CTI-1601 in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P2 | N=75 | Enrolling by invitation | Sponsor: Larimar Therapeutics, Inc.

New P2 trial • Ataxia • Friedreich ataxia • Movement Disorders

A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P2 | N=28 | Completed | Sponsor: Larimar Therapeutics, Inc. | Recruiting ➔ Completed

Trial completion • Ataxia • Friedreich ataxia • Movement Disorders

Safety, pharmacokinetics, and pharmacodynamics of nomlabofusp (CTI-1601) in Friedreich's ataxia. (PubMed, Ann Clin Transl Neurol) - P1 | "Results from this study support a favorable safety profile for nomlabofusp. Subcutaneous nomlabofusp injections were quickly absorbed; higher doses and daily administration resulted in increased tissue frataxin concentrations. Future studies will evaluate longer-term safety and possible efficacy of nomlabofusp."

Journal • PK/PD data • Ataxia • CNS Disorders • Friedreich ataxia • Movement Disorders

A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P2 | N=30 | Recruiting | Sponsor: Larimar Therapeutics, Inc. | N=15 ➔ 30

Enrollment change • Ataxia • Friedreich ataxia • Movement Disorders

A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P2 | N=15 | Recruiting | Sponsor: Larimar Therapeutics, Inc.

New P2 trial • Ataxia • Friedreich ataxia • Movement Disorders

Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P1; N=27; Completed; Sponsor: Larimar Therapeutics, Inc.; Active, not recruiting ➔ Completed; Trial completion date: Jun 2021 ➔ Mar 2021; Trial primary completion date: Jun 2021 ➔ Mar 2021

Clinical • Trial completion • Trial completion date • Trial primary completion date • Ataxia • Friedreich ataxia • Movement Disorders

Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P1; N=27; Active, not recruiting; Sponsor: Larimar Therapeutics, Inc.; Recruiting ➔ Active, not recruiting

Enrollment closed • Ataxia • Friedreich ataxia • Movement Disorders

Mitochondria-Targeted Drug Development Digital Summit (pharmaphorum) - "The Mitochondria-Targeted Drug Development Digital Summit (April 27-29) is the only industry-led meeting focused on end-to-end mitochondria-targeting....Speakers set to share their ground-breaking research developments, straight to your home or office, this is your chance to learn from the industry’s very best, including: Larimar Therapeutics examining CTI-1601 as a potential protein replacement therapy under development for Friedreich’s Ataxia."

Review • Friedreich ataxia

Larimar Therapeutics Announces Completion of Dosing of the Single Ascending Dose Clinical Trial in Friedreich’s Ataxia Patients and Provides Program Update (GlobeNewswire) - P1, N=28; NCT04176991; Sponsor: Larimar Therapeutics, Inc.; "Preliminary data suggest that single subcutaneous injections of CTI-1601 were well tolerated at doses up to 100 mg; Company remains on track to report topline data...in Q2 2021; A Safety Review Committee reviewed...and recommended continuation...Analysis of clinical trial results remains ongoing....Eligible participants from the SAD trial may enroll in an ongoing double-blind, placebo-controlled, MAD clinical trial. To date, dosing of the first two MAD cohorts has been completed, with dosing of the third cohort expected to begin in the first quarter of 2021....Patients completing the SAD and/or MAD clinical trials are eligible to screen for an open-label extension clinical trial, which is expected to initiate in the second half of 2021. Larimar also expects to initiate a MAD clinical trial in patients under 18 years of age in the second half of 2021."

DSMB • New trial • P1 data • Trial completion • Trial status • Friedreich ataxia

Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P1; N=28; Completed; Sponsor: Larimar Therapeutics, Inc.; Recruiting ➔ Completed

Clinical • Trial completion • Ataxia • Friedreich ataxia • Movement Disorders

Larimar Therapeutics Reports Third Quarter 2020 Operating and Financial Results (GlobeNewswire) - "Phase 1 trials evaluating CTI-1601 as a treatment for Friedreich’s ataxia on track for topline data in 1H 2021; Received orphan drug designation for CTI-1601 from the European Commission....Research and development expenses for the third quarter of 2020 were $6.9 million compared to $8.0 million for the third quarter of 2019."

Commercial • Orphan drug • Friedreich ataxia

Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P1; N=30; Recruiting; Sponsor: Larimar Therapeutics, Inc.

Clinical • New P1 trial • Ataxia • Friedreich ataxia • Movement Disorders

Larimar Therapeutics Reports Second Quarter 2020 Operating and Financial Results (GlobeNewswire) - "Research and development expenses for the second quarter of 2020 were $8.9 million compared to $3.1 million for the second quarter of 2019. The increase in research and development expenses compared to the prior year period was primarily due to an increase in external development costs for CTI-1601, an increase in personnel related costs due to headcount additions in our research and development functions and an increase in stock-based compensation."

Clinical • Friedreich ataxia