Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Investor Presentation (November 2020)
28 slides from Larimar Therapeutics's investor presentation, Nov 10, 2020
Slide 1: Larimar Therapeutics Corporate Presentation November 2020
Exhibit 99.2 Larimar Therapeutics Corporate Presentation November 2020 1Exhibit 99.2 Larimar Therapeutics Corporate Presentation November 2020 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
Investment Highlights Novel protein replacement therapy platform designed to address complex rare diseases Lead candidate CTI-1601 is a recombinant fusion protein being developed to CTI-1601 deliver human frataxin to the mitochondria for the treatment of Friedreich’s ataxia (FA) Placebo-controlled P
Slide 4: Scientific Advisory Board
Scientific Advisory Board Giovanni Manfredi, Marshall Summar, Marni J. Falk, Russell Clayton, Mark Payne, MD MD, PhD DO (Chairman) MD MD Finbar and Marianne Kenny Co-founder of Chondrial Chief of the Division of Executive Director of the Former Chief Medical Officer Professor in Clinical and Therape
Slide 5: Friedreich's Ataxia (FA) Rare and Progressive Disease
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 1 depending on the tissue considered >70% of patients present before age 14 • Initial symptoms may include unsteady pos
Slide 6: Market Opportunity
Market Opportunity Regulatory Prevalence Dosing Benefits Replacement therapy may be 5,000 patients in US and Well known by FDA; “Voice of needed throughout life to 20,000 patients in EU the Patient” report for FA was Dosing Dosing Dosing maintain frataxin (FXN) levels released in 2017 Additional aff
Slide 7: CTI-1601 is Being Developed to Deliver Frataxin (FXN)
CTI-1601 is Being Developed to Deliver Frataxin (FXN) CTI-1601 Maintains the Cleavage Site Between the MTS and Mature Human FXN STRUCTURE OF CTI-1601 STRUCTURE OF ENDOGENOUS FXN Mitochondrial Targeting Mature Mitochondrial Targeting Mature Sequence (MTS) Human FXN Sequence (MTS) Human FXN Cleavage b
Slide 8: CTI-1601 – Delivering Frataxin to the Mitochondria
CTI-1601 – Delivering Frataxin to the Mitochondria CPP allows CTI-1601 to traverse the 01 cell membrane into the cytoplasm CPP allows CTI-1601 to traverse 02 the mitochondrial membrane MPP cleaves CTI-1601. MTS and CPP 03 leave cell mitochondria Mature human frataxin remains within 04 the mitochondr
Slide 9: 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 • Assistance with
Slide 10: CTI-1601: Phase 1 Clinical Program in FA
CTI-1601: Phase 1 Clinical Program in FA Phase 1 Development Plan • Two Double-blind, Placebo Controlled Dosing Studies • Patient dosing began December 2019 • Safety Review Committee assesses all blinded data between each cohort to ensure patient safety • Topline results expected in 1H 2021 Number o
Slide 11: Upcoming Clinical Milestones
Upcoming Clinical Milestones 1H 2021 Topline Phase 1 Data Future Planned Studies Include: Open-label extension (OLE) study Pediatric MAD Study Phase 2/3 Double-Blind for patients who participated in (followed by an OLE) Placebo-Controlled Study SAD or MAD studies 11Upcoming Clinical Milestones 1H 20
Slide 12: CTI-1601 Open Label Extension Trial
CTI-1601 Open Label Extension Trial Patients from SAD and MAD trials are eligible to enter an open label extension (OLE) Multicenter Open Label Extension Trial Dose Level: To be determined based on PK/PD from SAD and MAD trials SAD Trial Patients Patients eligible to enroll Treatment Duration: Plann
Slide 13: CTI-1601: Positive Non-Clinical Data Support Development
CTI-1601: Positive Non-Clinical Data Support Development Studies demonstrate the ability of CTI-1601 to deliver Proof-of-Concept Achieved Through Multiple sufficient amounts of FXN to mitochondria in rodent Non-Clinical Studies: and non-human primate non-clinical models CTI-1601 extended survival in
Slide 14: 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 15: 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 Cardiac Mouse Model of FA 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 P=0.0001 Survival beyond vehi
Slide 16: CTI-1601 Prevents Ataxic Gait in Pvalb-Cre FXN-KO Mouse
CTI-1601 Pvalb-Cre FXN-KO mouse Prevents The Single dose level: 10 mg/kg CTI-1601 or vehicle given intraperitoneally three times per week Development of hFXN replacement with CTI-1601 prevents the development of ataxic gait Ataxic Gait in CTI-1601-treated mice survive longer than untreated mice KO m
Slide 17: 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 funct
Slide 18: 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
Slide 19: 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-16
Slide 20: Favorable PK/PD Profile in Healthy Cynomolgus Monkeys
Favorable PK/PD Profile in Healthy Cynomolgus Monkeys Study Design • CTI-1601 is bioavailable when given subcutaneously 6 healthy cynomolgus monkeys (3M / 3F) • Sustained levels of hFXN are found in blood cells (platelets) and peripheral Pre-dosed for 2 days with Vehicle tissues (buccal cells, skin)
Slide 21: Biodistribution in Healthy Cynomolgus Monkey
Biodistribution in Healthy Cynomolgus Monkey Buccal Swabs Skin Biopsies Platelets (Pre-treatment) (Pre-treatment) (Pre-treatment) CTI-1601 Human FXN Human + Monkey FXN • Treatment of monkeys with CTI-1601 results in sustained levels of hFXN in peripheral tissues that are accessible in the clinic • F
Slide 22: CTI-1601: Safe and Well Tolerated in Early Tox Studies
CTI-1601: Safe and Well Tolerated in Early Tox Studies Cynomolgus Monkey Sprague Dawley Rat Injection Site Observations Injection Site Observations • Some injection sites raised and firm; • Some injection sites showed irritation, increased injection site pathology at higher firmness, inflammation at
Slide 23: CTI-1601 is Protected by a Strong IP Portfolio
CTI-1601 is Protected by a Strong IP Portfolio Pending CTI-1601 patent application extends into 2040 2000 2005 2010 2015 2020 2025 2030 2035 2040 2045 Expiration TAT-MTS-FXN Composition of Matter (broad coverage of CTI-1601) US 9,045,552 (Exclusive license from Wake Forest) October, 2024 Methods of
Slide 24: Investment Highlights
Investment Highlights Novel protein replacement therapy platform designed to address complex rare diseases Lead candidate CTI-1601 is a recombinant fusion protein being developed to CTI-1601 deliver human frataxin to the mitochondria for the treatment of Friedreich’s ataxia (FA) Placebo-controlled P
Slide 25: Thank You
Larimar Therapeutics Corporate Presentation THANK YOU 25Larimar Therapeutics Corporate Presentation THANK YOU 25
Slide 26: Leadership Team
Leadership Team Michael Celano Carole Ben-Maimon, MD Jennifer Johansson, JD Chief Financial Officer Chief Executive Officer VP Regulatory Affairs & Counsel David Bettoun, PhD Nancy M. Ruiz, MD, FACP, FIDSA Keith E. Lynch, Jr. VP Discovery & Non-clinical R&D VP, Manufacturing and Supply Chain Chief M
Slide 27: 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. • Pri
Slide 28: Friedreich's Ataxia (FA) Symptoms & Natural History
Friedreich’s Ataxia (FA) Symptoms & Natural History 70% of patients present before age 14 Age 10 – 30 years: Progression of disease Symptoms continue to worsen and may include Significant asymptomatic period of disease development of advanced limb ataxia often requiring patient 01 03 Age of onset co
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