Savara, Inc. · NASDAQ:SVRA
Savara — Mast Therapeutics — Investor Presentation (April 2017)
24 slides from Savara's investor presentation, Apr 24, 2017
Slide 1: Corporate presentation April 2017
Corporate presentation APRIL 2017 Exhibit 99.1
Slide 2: Safe Harbor Statements
Safe Harbor Statements Forward Looking Statements. Savara cautions you that statements in this presentation that are not a description of historical fact are forward-looking statements which may be identified by the use of words such as “expect,” “intend,” “plan,” “anticipate,” “believe,” and “will,
Slide 3: Savara Overview
Savara Overview Clinical Stage Specialty Pharmaceutical Company… …Focused on Rare Respiratory Diseases Unique products with no approved alternatives Clinically proven treatment concepts Over $1B peak sales potential* Pre-Clinical Molgradex AeroVanc Aironite Phase 1 Phase 2 Phase 3 Cystic Fibrosis (C
Slide 4: MAST Merger
MAST Merger Merger announced in Jan 2017 Pending Mast shareholder consent Name change to Savara, Inc. Listing on NASDAQ: SVRA Combined Cash of $23m as of Q4 2016 Reverse Split Share Price* Shares Outstanding* Market Cap* 50-70 $6.5 to $9.1 16M to 23M $140M * Assuming a MSTX share price of $.13 and a
Slide 5: Molgradex: Inhaled GM-CSF for Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Molgradex Inhaled GM-CSF for Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Slide 6: PAP: excess of surfactant in the Lungs
PAP: excess of surfactant in the Lungs Anti-GM-CSF antibodies cause accumulation of surfactant in the alveoli Mechanism of disease well understood Courtesy of Dr. Bruce Trapnell
Slide 7: Inhaled GM-CSF Promising in academic studies
Inhaled GM-CSF Promising in academic studies aPAP patient before inhaled GM-CSF After 6 months of inhaled GM-CSF Improvement in alveolar to arterial oxygen gradient ((A-a)DO2) Published experience from treatment of more than 80 aPAP patients suggests potential for profound impact on oxygenation and
Slide 8: Molgradex: First Inhaled GM-CSF
Molgradex: First Inhaled GM-CSF Direct lung delivery to overcome functional GM-CSF deficiency Inhalation solution of rhGM-CSF PARI eFlow® Nebulizer Proprietary cell bank for GM-CSF manufacturing Considerable indication extension opportunity Orphan status (7-10 years exclusivity)
Slide 9: Molgradex: FDA Dialogue and Pivotal study for Europe
FDA Dialogue Ongoing Expand PII/III study with alternative endpoints or Confirmatory study Molgradex: PIVOTAL study for Europe Enrolling Pivotal EU & Japan (n=51 aPAP) Data read Q1/2018 Change in AaDO2 Time to WLL Phase I (Complete) Volunteers (n=42) SAD / MAD Molgradex well tolerated with a potent
Slide 10: Phase I – consistent WBC response to each dose
Phase I – consistent WBC response to each dose Safety in healthy volunteers comparable to placebo Potent dose-dependent pharmacodynamic effect
Slide 11: Design of Ongoing Molgradex Phase II/III Study
Design OF Ongoing Molgradex Phase II/III Study Period 1- Double-blind Period 2- Follow-up* Primary Endpoint Secondary Endpoints Requirement for/time to WLL Change from baseline in (A-a)DO 2 6 min walk distance Vital Capacity Quality of life
Slide 12: AeroVanc: Inhaled Vancomycin for MRSA in Cystic Fibrosis
AeroVanc Inhaled Vancomycin for MRSA in Cystic Fibrosis
Slide 13: High Unmet Need for Inhaled MRSA Treatment
HIGH UNMET NEED for INHALED MRSA Treatment Persistent lung infections managed with chronic inhaled antibiotics No approved inhaled MRSA antibiotic, emerging use of nebulized IV form of vancomycin MRSA infection associated with worse clinical outcomes CF Prevalence (US) 30,000 patients, 26% MRSA infe
Slide 14: AeroVanc: First Inhaled MRSA Antibiotic
AeroVanc: First Inhaled MRSA Antibiotic Grants from CFF & NIH Inhaled Dry Powder Vancomycin Drug Directly to Site of Infection Reduced Systemic Toxicity Manufacturing at Commercial Scale Orphan 7 yrs + QIDP 5 yrs (total 12 years exclusivity)
Slide 15: AeroVanc Phase III ready
Pivotal PC / DB (n=200) Enrollment anticipated to begin in Q3 2017 AeroVanc Phase III ready Proof of Concept PC / DB (n=87) Phase I (Complete) Volunteers (n=18) CF patients (n=7) Generally well tolerated & safe with favorable PK profile Inhalation tox & safety pharmacology 2010 - 11 2011 - 12 2013 -
Slide 16: Phase II: MRSA in Sputum - Primary Endpoint Met
Phase II: MRSA in Sputum - Primary Endpoint Met ITT population, n = 87 patients, 32mg and 64mg pooled Proof of concept established Microbiology not acceptable to FDA as Phase III primary endpoint
Slide 17: Dose Selected for Phase III Study
32 mg Dose Selected for Phase III Study Vancomycin trough concentrations in high excess over MIC at both doses ~ 50 % premature study drug discontinuations at 64 mg dose PK Population, AeroVanc treatments only, n=11
Slide 18: FEV1 Improvement Consistent with TOBI Data
FEV1 Improvement Consistent with TOBI Data PP population, 32 mg dose cohort, < 21 years of age, n = 16, post hoc analysis Best FEV1 improvement in young patients Similar FEV1 response profile in prior TOBI studies Absolute change in FEV1 selected as Phase III primary endpoint Phase III to be adequat
Slide 19: Improvement in Time to other antibiotics
Improvement in Time to other antibiotics ITT, 32 mg dose cohort, < 21 years of age, n = 20 , post hoc analysis Prolongation of time to use of other antibiotics In chronic use, a new treatment cycle would start at day 56, and events (use of other antibiotics) would not be expected at that time Time t
Slide 20: Phase III design agreed with FDA
Phase III design agreed with FDA Period 1- Double-blind Period 2- Open-label FEV 1 improvement (relative change, number of response cycles) FEV 1 improvement at Week 4 and Week 20 (absolute change analyzed sequentially) Respiratory Symptoms Diary Time to use of another antibiotic for pulmonary infec
Slide 21: Anticipated Corporate milestones
Anticipated Corporate milestones H2 ‘17 H1 ‘18 H2 ‘18 2019 H1 ‘17 2020 AeroVanc Phase III Enrollment to Start Molgradex Top Line Results from Phase II/III Study Molgradex US Study Design to be Announced Molgradex NDA / MAA Aironite Phase 2 Top Line Results AeroVanc NDA
Slide 22: Thank You!
THANK YOU!
Slide 23: Investment Highlights
Investment Highlights Pipeline Multiple late-stage assets addressing orphan diseases Previously approved active drug substances and drug delivery technologies High local drug concentrations reducing systemic toxicity Strong market protection Strategy Pursue AeroVanc and Molgradex indication expansio
Slide 24: Management & BOD
Management & BOD Executive Team Rob Neville, CEO Serial entrepreneur / Evity exit $100M / Series A lead investor Dr. Taneli Jouhikainen, President & COO Serial drug developer / 10+ license deals / public company and IPO experience / 20 yrs. in pharma mgmt. David Lowrance, CFO 15 years biotech / phar
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