Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Investor Presentation (August 2020)
23 slides from Larimar Therapeutics's investor presentation, Aug 6, 2020
Slide 1: Corporate Presentation August 2020
Corporate Presentation August 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
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
Slide 4: Investment Highlights
Investment Highlights Clinical-stage biotech with novel protein replacement therapy platform to address untreated, serious and complex rare diseases Lead candidate, CTI-1601, in ongoing Phase 1 clinical development for treatment of Friedreich’s ataxia (FA) To our knowledge, frataxin (FXN) protein re
Slide 5: Frataxin (FXN): Crucial for Mitochondrial Function
Frataxin (FXN): Crucial for Mitochondrial Function Mitochondria is essential energy source for nearly all cells in body Frataxin (FXN ) needed for synthesis of several key enzymes in mitochondria Low levels of FXN leads to deficiency of these enzymes reducing mitochondrial function Disease affects m
Slide 6: Friedreich's ataxia (FA): Rare and Progressive Disease
Friedreich’s ataxia (FA): Rare and Progressive Disease Rare disease caused by genetic defect resulting in abnormally low levels of FXN Affects ~5,000 patients in U.S. and ~20,000 patients in EU Onset: Age of onset correlated with severity and speed of progression >70% of patients present before age
Slide 7: 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 8: 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 9: CTI-1601 – POC Achieved Through Multiple Non-Clinical Studies
CTI-1601 – POC Achieved Through Multiple Non-Clinical Studies Nonclinical efficacy and PD data support of continued development to potentially replace FXN in patients with FA Extended survival in a well-characterized nonclinical model of FA Prevented ataxic gait in another nonclinical model of FA De
Slide 10: CTI-1601 Extends Survival in FXN-deficient KO Mice
CTI-1601 Extends Survival in FXN-deficient KO Mice Initial Proof of Concept for FXN Replacement Therapy in FA 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)
Slide 11: CTI-1601 Prevents Neurological Phenotype and Ataxic Gait in KO mice
CTI-1601 Prevents Neurological Phenotype and Ataxic Gait in KO mice In-Vivo Efficacy Data in Neurologic KO Mouse Model 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 ataxi
Slide 12: CTI-1601 Effectively Traffics to Mitochondria; Delivers hFXN
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
Slide 13: 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 14: 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 KO: CTI-1601 KO: Vehicle Wildtype: Vehicle
Slide 15: Human Frataxin Distributed Into All Tissues Tested
Human Frataxin Distributed Into All Tissues 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, Bucca
Slide 16: PK/PD Study in Healthy Cynomolgus Monkeys
PK/PD Study in Healthy Cynomolgus Monkeys Study Design CTI-1601 is bioavailable when given subcutaneously Sustained levels of hFXN are found in blood cells (platelets) and peripheral tissues (buccal cells, skin) as early as the 7th day and still present after 14 days Sustained levels of processed hF
Slide 17: CTI-1601: Safe and Well Tolerated in Multiple Animal Models
CTI-1601: Safe and Well Tolerated in Multiple Animal Models Cynomolgus Monkey Sprague Dawley Rat 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 obser
Slide 18: CTI-1601: Ongoing Phase 1 Clinical Program in FA Patients
CTI-1601: Ongoing Phase 1 Clinical Program in FA Patients Dosing regimen: Single Ascending Doses given SC (SAD); Multiple Ascending Doses given SC (MAD) Patient dosing began December 2019 Three cohorts dosed-SAD Phase 1 Number of subjects: SAD: approximately 32-34 subjects (currently 4 cohorts plann
Slide 19: Marketing Exclusivity and Accelerated Programs
Marketing Exclusivity and Accelerated Programs EMA Committee for Orphan Medicinal Products (COMP) recently issued positive opinion on application for orphan drug designation for CTI-1601 Expect European Commission will formally grant the designation this year FDA has granted CTI-1601: Orphan Drug St
Slide 20: Strong IP Position with Expected Market Exclusivity
Strong IP Position with Expected Market Exclusivity TAT- FXN composition of matter and methods of use patents expected to provide protection to 2024 and 2025, with provisional patents specific to CTI-1601 potentially extending protection to 2040 Exclusive licenses from Wake Forest and Indiana Univer
Slide 21: Strong Relationship with FARA
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
Slide 22: Summary
Summary Novel protein replacement therapy platform CTI-1601 in Phase 1 clinical development for treatment of Friedreich’s ataxia (FA) Topline Phase 1 data expected in 1H 2021 Experienced leadership team Extensive IP Strong balance sheet of ~ $116M in cash from merger and financing* *as of 6/1/2020,
Slide 23: Larimar
Larimar
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