Larimar Therapeutics, Inc. · NASDAQ:LRMR
Larimar Therapeutics — Zafgen — Merger or Acquisition (December 2019)
16 slides from Larimar Therapeutics's merger presentation, Dec 18, 2019
Slide 1: Company Update: Zafgen and Chondrial Therapeutics Definitive Merger Agreement
Company Update: Zafgen and Chondrial Therapeutics Definitive Merger Agreement December 18, 2019 Exhibit 99.2
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: Identifying the highest-potential value creation opportunity
Identifying the highest-potential value creation opportunity 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 market opportu
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: Introduction to Chondrial Therapeutics
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) pr
Slide 7: Proprietary protein replacement platform
Proprietary protein replacement platform Peptide technology with proven ability to design differentiated cell-penetrating peptides (CPPs) that enable intracellular delivery of bioactive cargos Highly relevant and applicable to a range of cargos and rare disease indications
Slide 8: Friedreich's ataxia (FA): significant medical need
Friedreich’s ataxia (FA): significant medical need 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 t
Slide 9: CTI-1601 – Delivering frataxin to the mitochondria
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 mitochondria
Slide 10: CTI-1601: proof of concept achieved through multiple nonclinical studies
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 Demonstrated capabi
Slide 11: CTI-1601 extends survival in FXN-deficient KO Mice
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
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: Human frataxin distributed into all tissues tested following CTI-1601 dosing
Human frataxin found to be distributed into all tissues tested following CTI-1601 dosing 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 Ro
Slide 14: CTI-1601: safe and well tolerated in multiple animal models
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 Sy
Slide 15: CTI-1601: Initiated Phase 1 clinical program in FA
CTI-1601: Initiated Phase 1 clinical program evaluating safety and efficacy in FA Double, placebo-controlled SAD/MAD clinical trial Assessing safety, tolerability, PK/PD of subcutaneous CTI-1601 vs. placebo in adult patients with Friedreich's ataxia Patient dosing began recently Sufficient drug supp
Slide 16: 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