Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Investor Presentation (June 2020)
21 slides from Larimar Therapeutics's investor presentation, Jun 3, 2020
Slide 1: Investor Presentation June 2020
Investor Presentation June 2020 Exhibit 99.1
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements that are based on the Company’s beliefs and assumptions and on information currently available to management. All statements contained in this presentation other than statements of historical fact are forward-looking st
Slide 3: Larimar Therapeutics Introduction
Created by merger of Zafgen and Chondrial Therapeutics Traded under ticker “LRMR” Merger accompanied by $80 million private placement – World class life science investor shareholder base Financing led by Cowen Healthcare Investments with Acuta, Janus, Logos, OrbiMed, RA Capital and Vivo Continued sh
Slide 4: Clinical-stage biotech with novel protein replacement therapy platform
Clinical-stage biotech with a novel protein replacement therapy platform to address untreated, serious and complex rare diseases Lead candidate, CTI-1601, in Phase 1 clinical development for treatment of Friedreich’s ataxia (FA) We believe frataxin (FXN) protein replacement therapy is the only prote
Slide 5: Friedreich's ataxia (FA): Rare and Progressive Disease
Rare disease caused by genetic defect resulting in abnormally low levels of frataxin (FXN) Affects ~5,000 patients in U.S. and ~20,000 patients in EU Disease affects multiple body systems, particularly the brain and heart Onset: Age of onset correlated with severity and speed of progression (earlier
Slide 6: CTI-1601 – Designed to Deliver Frataxin (FXN)
CTI-1601 – Designed to Deliver Frataxin (FXN) CTI-1601 similar to frataxin except it has a CPP attached on end to allow it to move into cell and mitochondria
Slide 7: CTI-1601 – Delivering Frataxin to the Mitochondria
CTI-1601 – Delivering Frataxin to the Mitochondria 1 CTI-1601 crosses the cell membrane into cytoplasm 2 CTI-1601 crosses the mitochondrial membrane 3 Mitochondrial Processing Peptide (MPP) cleaves CTI-1601. MTS and CPP will leave cell mitochondria 4 Mature human frataxin remains within the mitochon
Slide 8: CTI-1601 – POC Achieved Through Multiple Non-Clinical Studies
Extended survival in a well-characterized nonclinical model of FA Prevented ataxic gait in another nonclinical model of FA Demonstrated capability of delivering sufficient amounts of FXN to mitochondria Prevented left ventricle dilation and maintained function Safe and well tolerated in multiple spe
Slide 9: CTI-1601 Extends Survival in FXN-deficient KO Mice
TAT-FXN was administered 10 mg/kg SC every other day CTI-1601 extended survival in a well-characterized cardiac mouse model of FRDA Median Survival of MCK-Cre FXN-KO Mice 166 days (CTI-1601) vs 98.0 days (Vehicle) Survival beyond Vehicle mean (107.5 days) 87.5% (CTI-1601) vs. 33% (Vehicle) Confirms
Slide 10: CTI-1601 Prevents Neurological Phenotype and Ataxic Gait in KO mice
Pvalb-Cre FXN-KO mouse Single dose level: 10 mg/kg CTI-1601 or Vehicle given intraperitoneally (IP) three times per week hFXN replacement with CTI-1601 prevents development of ataxic gait Treated mice survive longer than untreated mice Human frataxin present in brain, dorsal root ganglia and spinal
Slide 11: CTI-1601 Effectively Traffics to Mitochondria; Delivers hFXN
hFXN concentration within mitochondria increases in a dose-dependent manner Given subcutaneously, CTI-1601 functionally replaces hFXN in mitochondria of KO mice SDH* activity increases in a dose-dependent manner after administration of CTI-1601; activity plateaus at 30 mg/kg and is equivalent to act
Slide 12: CTI-1601 Prevents Left Ventricle (LV) Dilation
CTI-1601 Prevents Left Ventricle (LV) Dilation Left ventricular volume increases in systole in untreated mice by 8 weeks (after 4 weeks of dosing with vehicle), but remains similar to wildtype when treated with CTI-1601 CTI-1601-treated mice similar to controls; echocardiogram shows significant diff
Slide 13: CTI-1601 Preserves Left Ventricle (LV) Function
CTI-1601 Preserves Left Ventricle (LV) Function LV function drops significantly in untreated mice by week 8 CTI-1601-treated mice similar to controls; echocardiogram again shows significant differences between KO:Vehicle and KO:CTI-1601 treated mice LV Fractional Shortening LV Ejection Fraction KO:
Slide 14: Human Frataxin Distributed Into All Tissues Tested
Observed hFXN across all tissue and cell types tested: Tissues Examined, By Study Study Vehicle Human Frataxin Distribution Rats Brain, Heart, Liver Neuro KO Mice Brain, Dorsal Root Ganglia, Spinal Cord Cardiac KO Mice Mitochondria of Skeletal Muscle and Cardiomyocytes Cynomolgus Monkey CSF, Skin, B
Slide 15: PK/PD Study in Healthy Cynomolgus Monkeys
Study Design PK/PD Study in Healthy Cynomolgus Monkeys CTI-1601 is bioavailable when given subcutaneously Sustained levels of processed hFXN are found after 14 days in the cerebrospinal fluid of monkeys, suggesting CNS penetration Sustained levels of hFXN are found in blood cells (platelets) and per
Slide 16: CTI-1601: Safe and Well Tolerated in Multiple Animal Models
Injection Site Observations Some injection sites raised and firm; increased injection site pathology at higher doses most likely due to local irritation Systemic Toxicity Analysis No other clinical observations or treatment-related changes in food consumption, body weight or organ weight No systemic
Slide 17: CTI-1601: Ongoing Phase 1 Clinical Program in FA Patients
Dosing regimen: Single Ascending Doses given SC (SAD); Multiple Ascending Doses given SC (MAD) Number of subjects: SAD: approximately 32-34 subjects (currently 4 cohorts planned) MAD: 24-30 subjects (currently 3 cohorts planned) Outcome measures: Primary: Safety and tolerability Secondary: PK; PD (h
Slide 18: Marketing Exclusivity and Accelerated Programs
FDA has granted CTI-1601: Orphan Drug Status Granted July 2017 Eligible for 7 years market exclusivity upon approval Fast Track Designation Granted November 2019 Actions to expedite development and review Rare Pediatric Disease Designation Granted December 2019 Eligible for voucher upon BLA approval
Slide 19: Strong Relationship with FARA
Company has strong relationship with Friedreich’s Ataxia Research Alliance (FARA) National, non-profit organization dedicated to the pursuit of scientific research leading to treatments and a cure for FA FARA provides industry with several key items Assists with patient recruitment and education Acc
Slide 20: Summary
Clinical-stage biotech with a novel protein replacement therapy platform CTI-1601 in Phase 1 clinical development for treatment of Friedreich’s ataxia (FA) We believe frataxin (FXN) protein replacement therapy is the only protein replacement therapy currently in clinical development Nonclinical stud
Slide 21: Investor Relations
Investor Relations investors@larimartx.com
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