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

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

Larimar Therapeutics Announces Positive Opinion on Orphan Drug Designation Received from the European Medicines Agency for CTI-1601 for the Treatment of Friedreich’s Ataxia (GlobeNewswire) - "Larimar Therapeutics, Inc....today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the company’s application for orphan drug designation for CTI-1601, a potential treatment for Friedreich’s ataxia (FA)....Larimar expects that the European Commission, based on this positive opinion of the COMP, will formally grant the orphan drug designation for the European Union (EU) this year....'We look forward to working closely with EMA and continuing our U.S. Phase 1 trial of CTI-1601....We remain on track to report topline data in the first half of 2021.'"

European regulatory • P1 data • Friedreich ataxia

Larimar Therapeutics Announces Dosing of Patients in Third Cohort of Phase 1 SAD Trial of CTI-1601 for Treatment of Friedreich’s Ataxia (GlobeNewswire) - "Larimar Therapeutics, Inc....today announced that patients have been dosed in the third cohort of a Phase 1 clinical trial to evaluate the safety and tolerability of single ascending doses (SAD) of CTI-1601 for the treatment of Friedreich’s ataxia (FA). The trial was previously delayed due to the impact of the COVID-19 pandemic....To date, two cohorts of patients have completed the Phase 1 clinical trial. Topline results are planned for the first half of 2021."

P1 data • Trial status • Friedreich ataxia

Form S-3 Larimar Therapeutics (Streetinsider.com) - "We are currently evaluating CTI-1601 in a single ascending dose....The first two cohorts of patients have completed the SAD clinical trial....We have delayed initiation of the next cohort in the SAD clinical trial. We are conducting the clinical trial at one clinical trial site in New Jersey....We cannot estimate when the next cohort of patients will begin the clinical trial. While top line results from the SAD and the planned multiple ascending dose, or MAD, clinical trial...being expected in first half of 2021....We are also evaluating CTI-1601 in good laboratory practices...including 90-day GLP toxicity studies in rats and non-human primates, or NHPs. These studies are ongoing and the results of these studies are intended to be used to support the initiation of clinical trials..."

P1 data • Preclinical • Trial status • Friedreich ataxia

Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia (clinicaltrials.gov) - P1; N=32; Recruiting; Sponsor: Larimar Therapeutics, Inc.; Trial completion date: Mar 2020 ➔ Sep 2020; Trial primary completion date: Mar 2020 ➔ Sep 2020

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

Chondrial Therapeutics and Zafgen complete merger and begin operating as Larimar Therapeutics (BioSpace) - "Chondrial Therapeutics, Inc....today announced the completion of its reverse merger with Zafgen, Inc....The combined, publicly traded clinical-stage biotechnology company will operate under the name Larimar Therapeutics, Inc....The financing is being led by Cowen Healthcare Investments....'We believe our lead product candidate, CTI-1601, has the potential to become the first frataxin replacement therapy for patients with Friedreich’s ataxia.'"

Financing • M&A • Friedreich ataxia

Zafgen and Chondrial Therapeutics announce definitive merger agreement (GlobeNewswire, Zafgen, Inc.) - “Zafgen, Inc…today announced they have entered into a definitive merger agreement under which Chondrial will become a wholly-owned subsidiary of Zafgen and the stockholders of Chondrial Therapeutics will become the majority owners of Zafgen’s outstanding common stock upon the close of the merger. The proposed merger will result in a combined publicly traded, clinical-stage biopharmaceutical company operating under a new name, Larimar Therapeutics, Inc.”

M&A