Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Zafgen — Merger or Acquisition (January 2020)
19 slides from Larimar Therapeutics's merger presentation, Jan 13, 2020
Slide 1: Zafgen and Chondrial Therapeutics Planned Definitive Merger
Zafgen and Chondrial Therapeutics Planned Definitive Merger January 2020 Exhibit 99.1
Slide 2: Forward Looking Statements: Additional Information about the Proposed Merger
Forward Looking Statements Additional Information about the Proposed Merger and Where to Find It This communication relates to the proposed merger transaction involving Zafgen, Inc. (“Zafgen”) and Chondrial Therapeutics, Inc. (“Chondrial”) and may be deemed to be solicitation material in respect of
Slide 3: Forward Looking Statements
Forward Looking Statements Zafgen Forward-Looking Information is Subject to Risks and Uncertainty This communication contains forward-looking statements based upon Zafgen’s and Chondrial’s current expectations. Forward-looking statements involve risks and uncertainties, and include, but are not limi
Slide 4: Definitive merger agreement announced December 2019
Definitive merger agreement announced December 2019 Criteria included: Near-term milestones within 6 – 9 months post-transaction Well funded beyond value inflection milestone Either early stage (IND or Phase 1) or late (pivotal trial) Innovative scientific approach in an attractive field – both mark
Slide 5: About the proposed merger
About the proposed merger Post-transaction ownership ~60% Chondrial / ~40% Zafgen stockholders Anticipated 1H 2020 closing New company will operate under new name, Larimar Therapeutics Blended Board of Directors (four from Chondrial / three from Zafgen) President and Chief Executive Officer will be
Slide 6: Compelling value-creation opportunity
Compelling value-creation opportunity Publicly traded, clinical-stage biopharmaceutical company focused on rare disease Strong scientific approach and platform opportunity Targeting significant market opportunities Upcoming near-term value creating events expected Experienced leadership team Potenti
Slide 7: Chondrial Therapeutics Overview
Chondrial Therapeutics Overview
Slide 8: Introduction to Chondrial Therapeutics
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 potential treatment of Friedreich’s ataxia (FA) Only frataxin (FXN) protein replacement therapy in clinical d
Slide 9: Proprietary protein replacement platform
Peptide technology with proven ability to design molecules using cell-penetrating peptides (CPPs) that enable intracellular delivery of bioactive cargos Highly relevant and applicable to a range of cargos and rare disease indications Proprietary protein replacement platform
Slide 10: Friedreich's ataxia: rare disease caused by low frataxin levels
Rare disease caused by a genetic defect resulting in abnormally low levels of frataxin (FXN), affects ~5,000 patients in U.S. and ~10,000 patients in EU-5 Progressive, irreversible, systemic disease that affects multiple body systems, particularly the brain and heart Onset: Age of onset correlated w
Slide 11: CTI-1601 – Delivering frataxin to the mitochondria
NH3 CPP MTS Human FXN Mature Human FXN COOH CTI-1601: recombinant fusion protein intended to deliver human frataxin, the protein deficient in FA Site of MPP cleavage STEP 1: CTI-1601 crosses cell membrane into cytoplasm and then crosses mitochondrial membrane STEP 2: Mitochondrial Processing Peptida
Slide 12: CTI-1601: Proof of concept achieved through multiple nonclinical studies
Nonclinical efficacy and PD data provides proof of concept in support of continued development to potentially replace FXN in patients with FA Extended survival in a well-characterized nonclinical model of FA Prevents ataxic gait in another nonclinical model of FA Demonstrated capability of deliverin
Slide 13: CTI-1601 extends survival in FXN-deficient KO mice
TAT-FXN was administered 10 mg/kg SC every other day 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) CTI-1601 extended survival in a well characterized cardiac mouse model of FRDA Confirms
Slide 14: CTI-1601 prevents neurological phenotype and ataxic gait in KO mice
Pvalb-Cre FXN-KO mouse CTI-1601 given intraperitoneally (IP) three times per week Single dose level: 10 mg/kg CTI-1601 or Vehicle Dosing Groups: Wild Type, Pvalb-CRE (Vehicle), Pvalb-Cre (CTI-1601) Outcome Measures Prevents behavioral phenotype in mice that lack endogenous FXN in the brain/DRG Gait
Slide 15: 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 acti
Slide 16: Human frataxin distributed into all tissues tested
Observed hFXN across all tissue and cell types tested: Tissues Examined, By Study Study Study Vehicle Human Frataxin Distribution TOX-1601-01 Rats Brain, Heart, Liver PHARM-1601-02 Neuro KO Mice Brain, Dorsal Root Ganglia, Spinal Cord PHARM-1601-03 Cardiac KO Mice Mitochondria of Skeletal Muscle and
Slide 17: CTI-1601: Safe and well tolerated in multiple animal models
No systemic clinical or pathological observations related to CTI-1601 in GLP studies 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 t
Slide 18: CTI-1601: Initiated Phase 1 clinical program in FA patients
Double, placebo-controlled SAD/MAD clinical trials Assessing safety, tolerability, PK/PD of subcutaneous CTI-1601 vs. placebo in adult patients with Friedreich's ataxia Changes from baseline in frataxin levels and gene expression will also be evaluated Patient dosing began December 2019 Sufficient d
Slide 19: - @eeChondrial
- @eeChondrial Zafgen Therapeutics, Inc.