Atossa Therapeutics, Inc. · NASDAQ:ATOS
Atossa Therapeutics — Atossa Genetics — Corporate Update (September 2017)
34 slides from Atossa Therapeutics's corporate update, Sep 14, 2017
Slide 1: Preliminary Topical Endoxifen Phase 1 Results All Objectives Successfully Met
Preliminary Topical Endoxifen Phase 1 Results All Objectives Successfully Met September 14, 2017
Slide 2: Forward-Looking Statements
Forward - Looking Statements Some of the information presented herein may contain projections or other forward - looking statements regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on managemen
Slide 3: Discussion Topics
DISCUSSION TOPICS • Atossa Genetics Overview • Endoxifen • Phase 1 Study • Safety Summary • Tolerability Summary • Pharmacokinetic Summary • Upcoming Milestones 107 Spring St. Seattle WA 98104 3
Slide 4: Atossa Genetics Overview
Atossa Genetics Overview Atossa Genetics, Inc. 1616 Eastlake Ave, Seattle, WA 98102 4
Slide 5: Atossa Genetics
Atossa Genetics: • Clinical - stage • Novel drugs & delivery methods • Breast Cancer & other breast conditions • Headquartered in Seattle, WA 107 Spring St. Seattle WA 98104 5
Slide 6: Endoxifen
Endoxifen 107 Spring St. Seattle WA 98104 6
Slide 7: Endoxifen
Endoxifen • Most active metabolite of tamoxifen • Tamoxifen has been widely studied • Tamoxifen was first approved in 1977 107 Spring St. Seattle WA 98104 7 Tamoxifen Metabolites, including Endoxifen
Slide 8: Current Paradigm for Tamoxifen Use in US
Current Paradigm for Tamoxifen Use in US 107 Spring St. Seattle WA 98104 8 1 million breast cancer patients take oral tamoxifen for at least five years 10 million patients with a high risk of breast cancer are indicated for chemoprevention with oral tamoxifen Source: Freedman AN, Graubard BI, Rao SR
Slide 9: Unmet Medical Needs Are >$1B Markets
Unmet Medical Needs Are >$1B Markets 107 Spring St. Seattle WA 98104 9 500,000 breast cancer patients don’t benefit from tamoxifen because of metabolism problems 9.9 million high risk patients won’t take oral tamoxifen because of actual or the risk of systemic side effects Source: Freedman AN, Graub
Slide 10: Emerging Unmet Medical Need
Emerging Unmet Medical Need • Breast Density – Independent risk for breast cancer – Patients required to be notified in 30 states – No approved treatment • Tamoxifen reduces breast density 107 Spring St. Seattle WA 98104 10
Slide 11: Mandatory Patient Notification of Breast Density
Mandatory Patient Notification of BD 107 Spring St. Seattle WA 98104 11 PINK : Enacted Law — RED: Introduced Bill — BLUE: Working on Bill — WHITE: No Action — BLACK : Insurance Coverage Law From: AreYouDense.org
Slide 12: Why Endoxifen?
Why Endoxifen? • Address unmet medical needs for: – Providing a topical alternative to oral tamoxifen for reducing breast density – Enh ancing the chemoprevention properties of tamoxifen in those patients who are refractory • Extensive characterization of tamoxifen, the parent drug 107 Spring St. Se
Slide 13: Phase 1 Study
107 Spring St. Seattle WA 98104 13 Phase 1 Study
Slide 14: Preliminary Phase 1 Topical Results
Preliminary Phase 1 Topical Results • Study objectives achieved • Demonstrated: - Safety - Tolerability - Verification of transdermal delivery Supports continued development 107 Spring St. Seattle WA 98104 14
Slide 15: Development Timeline 2016-2017
107 Spring St. Seattle WA 98104 15 Q2 2106 • Contracted to develop endoxifen API Q3 2016 • First batch of API for clinical use Q4 2016 • Topical formulation development in US • Retained CRO in Australia Q1 2017 • Retained CMO in Australia (for topical and oral presentations) Q2 2017 • Approval to st
Slide 16: Preliminary Study Conclusions
Preliminary Study Conclusions • All study objectives successfully achieved – Safety: There were no clinically significant safety signals and no clinically significant adverse events in participants receiving topical Endoxifen. – Tolerability: Topical Endoxifen was well tolerated at each dose level a
Slide 17: Phase 1 Study Design
Phase 1 Study Design PART A (Topical). Cohorts, dose levels and number of participants. Cohort Dose Level Number of Participants (mg per breast) (Total mg) (Z) - Endoxifen Placebo 1 1 2 6 2 2 3 6 6 2 3 5 10 6 2 PART B (Oral). Cohorts, dose levels and number of participants. Dose Level (mg/day) (Z) -
Slide 18: Study Design Summary
Study Design Summary 107 Spring St. Seattle, WA 98104 18 • Two - part double - blinded, placebo controlled, dose escalation trial investigating the safety and pharmacokinetics of (Z) - Endoxifen in healthy female volunteers – Part A: Topical – liquid applied to the breasts – Part B: Capsule – taken
Slide 19: Topical Design Summary
Topical Design Summary Cohort Dose Number of Participants mg per Breast Total mg (Z) - Endoxifen Placebo 1 1 2 6 2 2 3 6 6 2 3 5 10 6 2 107 Spring St. Seattle WA 98104 19 x Healthy females, 18 to 65 years of age x Body Mass Index of 18 to 30 x No chronic or acute disease x Daily administration diari
Slide 20: Safety Summary
Safety Summary 107 Spring St. Seattle WA 98104 20
Slide 21: Safety Summary
Safety Summary • No safety signals observed in weekly assessments of/in: – Blood chemistry – Coagulation parameters – Hematology parameters – Urinalysis – Vital Signs – Heart – Physical Examinations 107 Spring St. Seattle WA 98104 21
Slide 22: Adverse Events
Adverse Events • There were no Serious Adverse Events • Two subjects (one placebo and one active) experienced a Treatment Emergent Adverse Event of moderate severity and possibly drug - related – Headache – Nausea 107 Spring St. Seattle WA 98104 22
Slide 23: Tolerability Summary
Tolerability Summary 107 Spring St. Seattle WA 98104 23
Slide 24: Tolerability Data – by Participant
Tolerability Data – by Participant 107 Spring St. Seattle WA 98104 24 Parameter Participant Identification Dose Group Itching Low Intermediate High Placebo Mild #11 #27 #37 #29 Moderate Pain #33, #31 Mild #14 #37 #28 Moderate #11 #36 Redness Mild #27 #32 Moderate Burning Mild #33, #37 Moderate Irrit
Slide 25: Local Tolerability By Instances
Local Tolerability By Instances • A daily self - assessment of local tolerance was performed – 24 subjects for 28 days for a total of 672 daily assessments • Redness, Burning, Pain, Itching and Irritation were assessed each day • Scoring was None, Mild, Moderate or Severe for all five parameters 107
Slide 26: Overall Tolerability Scoring Based on Instance
Overall Tolerability Scoring Based on Instance 107 Spring St. Seattle WA 98104 26 None Mild Moderate Severe Not Done None 97.3% Mild 1.8% Moderate 0.2% Severe 0.0% Not Done 0.6%
Slide 27: Parameter Summary by Instance
Parameter Summary by Instance • One subject had mild irritation 17/28 days and moderate irritation on the End of Study assessment 107 Spring St. Seattle , WA 98104 27 Parameter None Redness 99% Burning 99% Pain 98% Itching 97% Irritation 97%
Slide 28: In-person Interview
In - person Interview • Each participant was interviewed every seven days for side - effect information • The participant at the highest dose who was bothered by side - effects was the same who reported topical irritation 107 Spring St. Seattle WA 98104 28 Result Cohort Not at All A little Bit Some
Slide 29: Pharmacokinetic Summary
107 Spring St. Seattle WA 98104 29 Pharmacokinetic Summary
Slide 30: Pharmacokinetic Objective
Pharmacokinetic Objective 107 Spring St. Seattle WA 98104 30 Formulation placed on skin surface Endoxifen absorbed into breast tissue to bind to ER and retard growth Breast blood vessels connect to systemic circulation taking “excess” endoxifen into systemic circulation Goal was to prove absorption
Slide 31: Endoxifen was detected in blood
Endoxifen was detected in blood 107 Spring St. Seattle WA 98104 31 11 7 2 0 0 2 4 6 8 10 12 10 mg 6 mg 2 mg Placebo Blood Level > 2.0 ng/mL N umber of Samples
Slide 32: Preliminary Study Conclusions
Preliminary Study Conclusions • All primary and secondary endpoints successfully met – Safety: There were no clinically significant safety signals and no clinically significant adverse events in participants receiving topical Endoxifen . – Tolerability: Topical Endoxifen was well tolerated at each d
Slide 33: Upcoming Milestones
Up Coming Milestones • 2017 – Complete analysis of oral endoxifen Phase 1 data – Finalize Phase 2 Study design for topical endoxifen – Finalize Phase 2 Study design for oral endoxifen – Final report of combined topical and oral Phase 1 by CRO – IRB and government submission for Phase 2 – Advance bot
Slide 34: NASDAQ: ATOS
NASDAQ: ATOS