Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Investor Presentation (December 2020)
31 slides from Larimar Therapeutics's investor presentation, Dec 8, 2020
Slide 1: Larimar Therapeutics Corporate Presentation
December 2020 Larimar Therapeutics Corporate Presentation 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: Investment Highlights
Lead candidate CTI-1601 is a recombinant fusion protein being developed to deliver human frataxin to the mitochondria for the treatment of Friedreich’s ataxia (FA) Investment Highlights Regulatory benefits Strong balance sheet Placebo-controlled Phase 1 clinical trials in Friedreich’s ataxia patient
Slide 4: Scientific Advisory Board
Scientific Advisory Board Giovanni Manfredi, MD, PhD Finbar and Marianne Kenny Professor in Clinical and Research Neurology at Weill Cornell Medicine. Professor of Neuroscience at Weill Cornell Medicine. Russell Clayton, DO (Chairman) Former Chief Medical Officer at Alcresta Therapeutics, a medical
Slide 5: Friedreich's Ataxia (FA): Rare and Progressive Disease
Caused by genetic defect resulting in low levels of frataxin Patients with FA only produce ~20-40% of normal frataxin levels depending on the tissue considered1 >70% of patients present before age 14 Initial symptoms may include unsteady posture, frequent falling and progressive difficulty in walkin
Slide 6: Market Opportunity
Market Opportunity Dosing Dosing Replacement therapy may be needed throughout life to maintain frataxin (FXN) levels Disease is progressive and irreversible; initiating therapy early and continuing replacement therapy throughout life may be a necessity Dosing Prevalence 5,000 patients in US and 20,0
Slide 7: CTI-1601 is Being Developed to Deliver Frataxin (FXN)
CTI-1601 is Being Developed to Deliver Frataxin (FXN) The CPP allows CTI-1601 to traverse the cell and mitochondrial membranes where the CPP and MTS are removed by mitochondrial processing peptidase to produce mature human FXN STRUCTURE OF ENDOGENOUS FXN STRUCTURE OF CTI-1601 Cleavage by mitochondri
Slide 8: CTI-1601 – Delivering Frataxin to the Mitochondria
CTI-1601 – Delivering Frataxin to the Mitochondria 01 02 03 04 Mature human frataxin remains within the mitochondria to function MPP cleaves CTI-1601. MTS and CPP leave cell mitochondria CPP allows CTI-1601 to traverse the mitochondrial membrane CPP allows CTI-1601 to traverse the cell membrane into
Slide 9: 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 Assistance with patient recruitment and education
Slide 10: Summary of Clinical Friedreich's Ataxia Program
Double-blind, placebo-controlled, single ascending dose (SAD) trial completed follow-up in Q4 2020. Based on preliminary blinded data, single subcutaneous injections of CTI-1601 of up to 100 mg were thought to be well tolerated. Summary of Clinical Friedreich’s Ataxia Program Open Label Extension Tr
Slide 11: CTI-1601: Ongoing Phase 1 Clinical Program in FA
CTI-1601: Ongoing Phase 1 Clinical Program in FA Phase 1 Development Plan Two Double-blind, Placebo Controlled Dosing Trials Patient dosing began December 2019 Safety Review Committee assesses all blinded data between each cohort to ensure patient safety Topline results expected in Q2 2021 Number of
Slide 12: Preliminary SAD Data Suggest Favorable Safety and Tolerability
Preliminary summary of blinded data: Single dose (25 mg, 50 mg, 75 mg and 100 mg) of CTI-1601 or placebo given subcutaneously. 28 patients were dosed in the trial. Injection site adverse events were mild and transient No serious adverse events Preliminary SAD Data Suggest CTI-1601’s Favorable Safety
Slide 13: Upcoming Clinical Milestones
Upcoming Clinical Milestones Open label extension (OLE) trial for eligible patients who participated in SAD or MAD trials (expected initiation 2H 2021) MAD trial in patients under 18 years of age, followed by OLE (expected initiation 2H 2021) Global Phase 2/3 Double-Blind Placebo-Controlled Trial Fu
Slide 14: CTI-1601 Open Label Extension Trial
Dose Level: To be determined based on PK/PD from SAD and MAD trials Treatment Duration: Planned for 24 months with any necessary extensions Number of Subjects: Up to 50 Dose Regimen: To be determined based on PK/PD from SAD and MAD trials External Control Arm: Derived from Critical Path Institute Da
Slide 15: CTI-1601: Positive Non-Clinical Data Support Development
CTI-1601: Positive Non-Clinical Data Support Development CTI-1601 extended survival in a well-characterized non-clinical mouse model of FA CTI-1601 prevented left ventricle dilation and maintained function in non-clinical mouse models CTI-1601 prevented ataxic gait in another non-clinical mouse mode
Slide 16: Human Frataxin Distributed Into All Tissues Tested
Human Frataxin Distributed Into All Tissues Tested Tissues Examined 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 Cerebrospinal fluid, Skin,
Slide 17: CTI-1601 Extends Survival in FXN-deficient KO Mice
CTI-1601 Extends Survival in FXN-deficient KO Mice Median Survival of MCK-Cre FXN-KO Mice 166 days (CTI-1601) vs. 98 days (Vehicle) CTI-1601 was administered 10 mg/kg SC every other day Survival beyond vehicle mean (107.5 days) 87.5% (CTI-1601) vs. 33% (Vehicle) Demonstrates that CTI-1601 is capable
Slide 18: 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 three times per week hFXN replacement with CTI-1601 prevents the development of ataxic gait CTI-1601-treated mice survive longer than untreated mice Human frataxin present in brain, dorsal root ganglia and
Slide 19: CTI-1601 Delivers hFXN to Mitochondria in KO Mice
CTI-1601 Delivers hFXN to Mitochondria in KO Mice hFXN concentration within mitochondria increases in a dose-dependent manner Given subcutaneously, CTI-1601 functionally replaces hFXN in mitochondria of KO mice *Succinate dehydrogenase (SDH) activity, which is indicative of mitochondrial function, i
Slide 20: CTI-1601 Prevents Left Ventricle Dilation in KO Mice
CTI-1601 Prevents Left Ventricle Dilation in KO Mice Left ventricular (LV) 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 (10 mg/kg every other day) CTI-1601-treated mice have similar LV volu
Slide 21: CTI-1601 Preserves Left Ventricle Function in KO Mice
CTI-1601 Preserves Left Ventricle Function in KO Mice Left ventricular (LV) function drops significantly in vehicle treated mice by week 8 CTI-1601-treated (10 mg/kg every other day) mice have similar LV as healthy controls; echocardiogram shows significant differences between vehicle and CTI-1601 t
Slide 22: Favorable PK/PD Profile in Healthy Cynomolgus Monkeys
Favorable PK/PD Profile in Healthy Cynomolgus Monkeys 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 hFXN are foun
Slide 23: Biodistribution in Healthy Cynomolgus Monkey
Biodistribution in Healthy Cynomolgus Monkey Treatment of monkeys with CTI-1601 results in sustained levels of hFXN in peripheral tissues that are accessible in the clinic FXN levels increase ~4X or more following CTI-1601 administration For comparison, FA patients show FXN levels that range from ~2
Slide 24: CTI-1601 is Protected by a Strong IP Portfolio
CTI-1601 is Protected by a Strong IP Portfolio 2005 2010 2020 2025 2030 2035 2040 2045 2000 Pending Granted TAT-MTS-FXN Composition of Matter (broad coverage of CTI-1601) US 9,045,552 (Exclusive license from Wake Forest) Expiration October 2024 CTI-1601 Composition of Matter and Methods of Treatment
Slide 25: Investment Highlights
Lead candidate CTI-1601 is a recombinant fusion protein being developed to deliver human frataxin to the mitochondria for the treatment of Friedreich’s ataxia (FA) Investment Highlights Regulatory benefits Strong balance sheet Placebo-controlled Phase 1 clinical trials in Friedreich’s ataxia patient
Slide 26: Thank You
THANK YOU Larimar Therapeutics Corporate Presentation
Slide 27: Leadership Team
Leadership Team Carole Ben-Maimon, MD Chief Executive Officer Nancy M. Ruiz, MD, FACP, FIDSA Chief Medical Officer John Berman, CPA VP Finance & Operations Jennifer Johansson, JD VP Regulatory Affairs & Counsel David Bettoun, PhD VP Discovery & Non-clinical R&D Noreen Scherer VP, Clinical Operations
Slide 28: Scientific Advisory Board
Scientific Advisory Board Russell (Rusty) Clayton, DO, Scientific Advisory Board Chair Nearly two decades of executive experience in pharmaceutical, biologics and medical device development and commercialization as a consultant in clinical development, medical affairs and regulatory affairs. Prior t
Slide 29: Friedreich's Ataxia (FA) Symptoms & Natural History
Friedreich’s Ataxia (FA) Symptoms & Natural History 70% of patients present before age 14 Significant asymptomatic period of disease Age of onset correlated with severity and speed of progression (earlier onset correlated with more drastic progression) Age 10 – 30 years: Progression of disease Sympt
Slide 30: SAD Patient Demographics and Disease Characteristics
SAD Patient Demographics and Disease Characteristics Demographics Parameter Statistic Overall (n=28) Sex Male n (%) 17 (60.7) Female n (%) 11 (39.3) Age (years) Mean 31.7 Std. Deviation 12.95 Median 27.0 Min, Max 19, 69 Race White n (%) 28 (100.0) Ethnicity Hispanic/Latino n (%) 2 (7.1) Not Hispanic
Slide 31: CTI-1601: Safe and Well Tolerated in Toxicity Studies
CTI-1601: Safe and Well Tolerated in Toxicity Studies Toxicology findings in 28-day and 13-week studies support initiation of extended dosing of patients Sprague Dawley Rat (28-day and 13-week studies) Cynomolgus Monkey (28-day and 13-week studies) Injection Site Observations Some injection sites sh
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