Corporate Presentation March 2019 - Zosano Pharma

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Corporate Presentation March 2019

Transcript of Corporate Presentation March 2019 - Zosano Pharma

Corporate Presentation March 2019

This presentation contains forward-looking statements regarding Zosano’s technology and product candidates, including ADAM and Qtrypta, and other future events and expectations. Readers are urged to consider statements that include the words "may," "will," "would," "could," "should," "might," "believes," "estimates," "projects," "potential," "expects," "plans," "anticipates," "intends," "continues," "forecast," "designed," "goal," "approximately" or the negative of those words or other comparable words to be uncertain and forward-looking. These statements are subject to risks and uncertainties that are difficult to predict, and actual outcomes may differ materially. These include, without limitation, risks and uncertainties associated with the process of discovering, developing and commercializing products that are safe and effective for use as human therapeutics, risks inherent in the effort to build a business around such products and other risks and uncertainties described under the heading "Risk Factors" in the Company's most recent quarterly report on Form 10-Q and on it’s most recent annual report on Form 10-K. Although Zosano believes that the expectations reflected in these forward-looking statements are reasonable, we cannot in any way guarantee that the future results, level of activity, performance or events and circumstances reflected in forward-looking statements will be achieved or occur. All forward-looking statements are based on information currently available to Zosano and Zosano assumes no obligation to update any such forward-looking statements.

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FORWARD-LOOKING STATEMENTS

▪ Proprietary intracutaneous drug delivery platform▪ Lead Program: QtryptaTM (M207)

– Acute migraine therapy– Strong clinical data – NDA filing expected in Q4 2019

▪ Potential portfolio expansion in 2019– QtryptaTM for Cluster headache– Anti-Emetic (Nausea)

▪ Innovative application for delivery of Large Molecules– Proteins– Vaccines – Monoclonal Antibodies

▪ Partnering focus– QtryptaTM

– Large Molecules

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INVESTMENT HIGHLIGHTS

TRANSFORMING HOW DRUGS GET DELIVERED

Stratum corneum

Cellular epidermis

▪ Novel INTRACUTANEOUS patch containing 340μm sized microneedles

▪ Microneedles can be coated with both large and small molecules

▪ Allows for rapid and consistent dissolution of drugs into capillary bed

▪ Shallow depth of penetration of proprietary microneedles into superficial skin layers designed to minimize stimulation of nerve endings

▪ Quarter size patch designed to be convenient and discreet

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INTRACUTANEOUS DELIVERY SYSTEM

Adhesive Patch

Array

Inner Ring

Outer Ring

Reusable Applicator

LEAD PROGRAM: QTRYPTA™• ACUTE MIGRAINE THERAPY

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39 MILLION PEOPLE IN THE U.S. SUFFER FROM MIGRAINES

▪ 3rd Most Prevalent Disorder in the World▪ Impacts ~25% of U.S. Households ▪ Occurs in 12% of U.S. Population▪ 85% of Chronic Migraine patients are women

▪ $36B in Lost Productivity▪ $5.4B in Treatment Costs

Source: Migraine Research Foundation

PREVALENT

DEBILITATING

COSTLY

▪ 90% Unable to Function Normally▪ 1.2 MM ER Visits per Year▪ 25% of women experience 4+ Migraines/month▪ Attacks Last 4-72 Hours

▪ Never developed as an injectable— Zolmitriptan delivered by nasal spray, orally,

and ODT is well-tolerated

— Well-tolerated orally from 1 to 50 mg

▪ Potent triptan— Ki = 2.51nM / 5-HT1D

— 6.31nM / 5-HT1B

▪ Fast on rate with target receptors

▪ Longer dwell time

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CHOOSING ZOLMITRIPTAN FOR CLINICAL DEVELOPMENT

0

2

4

6

8

10

12

14

0 0.2 0.4 0.6 0.8 1 1.2 1.4 1.6 1.8 2

Zolm

itri

pta

n C

on

cen

trat

ion

, ng

/ml

Time, h

ADAM zolmitriptan 2 X 1.9 mg zolmitriptan tablet 2.5 mg

▪ Mean plasma zolmitriptan concentration following Qtrypta or 2.5mg oral tablet1

Source Kellerman DJ, Ameri M, Tepper SJ. Rapid systemic delivery of zolmitriptan using an adhesive dermally applied microarray. Pain Management doi: 10.2217/pmt-2017-0036 (2017).

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Qtrypta™

QTRYPTA: ENHANCED DELIVERY – PHASE 1

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QTRYPTA: PIVOTAL PHASE 2/3 EFFICACY STUDY DESIGN

Migraine Patients:

360

Population: Migraine frequency of 2-8 episodes per month

Design:Each migraine sufferer will take a single dose to treat a qualifying migraine

Sites: 35 centers in U.S.

Dosing: M207 1, 1.9, and 3.8 mg vs placebo

Primary Endpoints:

Proportion of subjects with pain freedom and most bothersome symptom freedom at 2 hours post-dose

Secondary Endpoints:

• Pain relief at 15 and 30 minutes and 2 hours • Time to sustained pain freedom for 24 hours• Photophobia free at 2 hours• Phonophobia free at 2 hours• Nausea free at 2 hours

41.5%

68.3%

14.3%

42.9%

0%

20%

40%

60%

Pain Freedom MBS

% S

ub

ject

s A

chie

vin

g P

ain

Fre

ed

om

or

Fre

ed

om

fro

m M

BS

at 2

H

ou

rs

Qtrypta Placebo

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STATISTICALLY SIGNIFICANT IMPACT ON PAIN FREEDOM AND MBS

ACHIEVEMENT OF CO-PRIMARY ENDPOINTS

p=<0.0001TG* = 27.2

p=<0.0009TG* = 25.4

* TG = Therapeutic Gain

Spierings ELH et al. Randomized, double-blind, placebo-controlled, parallel-group, multi-center study of the safety and efficacy of ADAM zolmitriptan for the acute treatment of migraine. Cephalalgia 2018 Feb;38(2):215-224.

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~80% OF PATIENTS HAD PAIN RELIEF THROUGH 48 HOURS

Spierings ELH et al. Randomized, double-blind, placebo-controlled, parallel-group, multi-center study of the safety and efficacy of ADAM zolmitriptan for the acute treatment of migraine. Cephalalgia 2018 Feb;38(2):215-224.*p=<0.05 **p=0.01

23.2%

46.3%

56.1%

68.3%*

80.5%** 81.7%**82.9%**

80.5%**78.0%** 78.0%**

0%

20%

40%

60%

80%

15 minutes 30 minutes 45 minutes 1 hour 2 hour 3 hour 4 hour 12 hours 24 hours 48 hours

% S

ub

ject

s A

chie

vin

g P

ain

Re

lief

Pain Relief 15 Minutes to 48 Hours

Qtrypta Placebo

44.4% 44.1%

15.90%13.70%

0%

20%

40%

% S

ub

ject

s A

chie

vin

g Pa

in F

reed

om

at 2

Ho

urs

Qtrypta Placebo13

STRONG PERFORMANCE IN DIFFICULT SUBTYPES OF MIGRAINE

Tepper SJ, Dodick DW, et al. Efficacy of ADAM Zolmitriptan for the Acute Treatment of Difficult-to-Treat Migraine Headaches. Headache 2019 in Press.

p = 0.0005p=0.0056

N=36

N=44

N=59

N=51

PAIN FREEDOM

Patients with NauseaMorning Migraine

Primary Objective▪ Long-term safety of Qtrypta for the acute treatment of

migraine

Study Design

▪ Adults who suffer from 2-8 migraine attacks per month on average (with or without aura)

▪ 250 subjects eligible for treatment phase— At least 150 must complete six months— At least 50 must complete one year

Status – Completed final milestone▪ Initiated November 2017▪ Fully enrolled May 2018

— 344 subjects ▪ 6 month data announced October 2018▪ 12 month data announced in February 2019

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PHASE 3 OPEN-LABEL LONG TERM SAFETY STUDY

31 sites in the US

44%

81%

68%

0%

30%

60%

90%

Pain Freedom Pain Relief MBS

% S

ub

ject

s A

chie

vin

g P

F, P

R a

nd

Fre

edo

m f

rom

MB

S at

2 H

ou

rs

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LONG TERM SAFETY RESULTS CONSISTENT WITH PIVOTAL STUDY

Efficacy Safety

*

* Most site reactions resolve within 24 – 48 hours

Investigator reported AE’s after 5,800+ treatments by category5,800+ migraines treated

297

161

373

831

Other

Triptan-associated AE's

Application SiteReactions

▪ QTRYPTA expected to be filed as a 505(b)(2) NDA

▪ Collaborative relationship with the FDA’s Neurology & Dermal Divisions

– Two positive End of Phase 2 meetings held

– Two pre-NDA meetings anticipated in 2019

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QTRYPTA HAS A WELL DEFINED REGULATORY PATH

On Track for NDA Submission currently expected at the End of 2019

79%of Migraineurs Would Be Willing to Try Another Acute Medication….

87% 86%

83% 79%

COMPLETE PAIN RELIEF NO RECURRENCE

RAPID ONSET NO SIDE EFFECTS

Lipton RB, Stewart WF. Headache. 1999;39(suppl 2):S20-S26

MANY MIGRAINE PATIENTS DISSATISFIED WITH CURRENT TREATMENTS

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81% Pain Relief

in 2 Hours

POTENTFAST

Peak Plasma Concentration in 15 Mins

COMPLETE SUSTAINED

41.5% Pain

Freedom in 2 Hours 63% Sustained Pain

Relief From 2-48 Hours

Kellerman DJ, Ameri M, Tepper SJ. Rapid systemic delivery of zolmitriptan using an adhesive dermally applied microarray. Pain Management doi: 10.2217/pmt-2017-0036 (2017). Spierings ELH et al. Randomized, double-blind, placebo-controlled, parallel-group, multi-center study of the safety and efficacy of ADAM zolmitriptan for the acute treatment of migraine. Cephalalgia 2018 Feb;38(2):215-224.

QTRYPTA IS DIFFERENTIATED AND ADDRESSES UNMET NEEDS

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QTRYPTA IS WELL POSITIONED AGAINST CURRENT TRIPTANS

0%

20%

40%

60%

80%

100%

0 0.5 1 1.5 2

% o

f Su

bje

cts

Time, h

Zomig 5mg Oral Zomig 5mg Nasal Imitrex 6mg Inj Imitrex 100mg Tab M207

PAIN RELIEF OVER TIME1 (REFERENCE COMPOUNDS)

1. Comparisons are not based on data resulting from head-to-head trials and are not direct comparisons of safety or efficacy. Different protocol designs, trial designs, patient selection and population number of patients, trial endpoints, trial objectives and other parameters that are not the same between the relevant trials may cause any comparisons of results from different trials to be unreliable.

Qtrypta™ (M207)

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POSITIONING AGAINST NEW ENTRANTS

41.5%

32.2%

38.8%

19.2% 19.6% 20.7% 21.8% 21.2%

34.3%

51.0%

68.3%

40.7%

48.7%

36.6% 37.6%

34.1%

38.9% 37.7%

64.1%

0%

20%

40%

60%

Qtrypta Lasmiditan 200mgSamurai study

Lasmiditan 200mgSpartan study

Rimegepant 75 mg301 study

Rimegepant 75 mg302 study

Ubrogepant 25mg Ubrogepant 50mg Ubrogepant 100mg Onzetra Xsail - nasal Zembrace 3 mg -injectable

% o

f Su

bje

cts

ach

ievi

ng

Pai

n F

ree

do

m a

nd

MB

S Fr

ee

do

m a

t 2

Ho

urs

Comparison of Pain Freedom and MBS Freedom at 2 Hours1

Pain Freedom MBS

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1. Comparisons are not based on data resulting from head-to-head trials and are not direct comparisons of safety or efficacy. Different protocol designs, trial designs, patient selection and population number of patients, trial endpoints, trial objectives and other parameters that are not the same between the relevant trials may cause any comparisons of results from different trials to be unreliable.

2. Onzetra Xsail was not tested for MBS Freedom

▪ Differentiated product

▪ Broad potential applicability

— Morning migraine

— Delayed treatment

— Nausea

▪ Efficacy data well positioned vs. current competitive offerings

▪ Clear Regulatory path

▪ Manufacturing in place

▪ NDA submission expected in Q4 2019

▪ Peak Sales potential of approximately $400 Million+

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QTRYPTA™ PARTNERSHIP OPPORTUNITY

▪ Cluster Headache

— Qtrypta

▪ Anti – Emetic (Nausea)

▪ Small molecule— 5HT3 inhibitor

▪ Large Molecules

— Biosimilar

— Monoclonal antibodies

— Vaccines

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PLANS FOR EXPANDING THE PORTFOLIO – 2019

SIGNIFICANT UNMET NEED IN CLUSTER HEADACHE

▪ Highly disabling chronic neurological condition1

➢ Recognized as most severe pain known to humans

➢ 15%-22% have suicidal ideations2

▪ Short-lasting headache attacks characterized by3:➢ Severe unilateral temporal/orbital pain

➢ Ipsilateral autonomic symptoms

➢ Lasting 15–180 minutes (when untreated)

➢ Sense of restlessness or agitation

▪ Prevalence of cluster headache➢ Approximately 1 in 1000 (0.12%) suffer from the condition

➢ Prevalence similar to Parkinson's and Multiple Sclerosis4,5

➢ Over 300,000 people suffer from cluster headache in the US

1. Jürgens T, Gaul C, et al. Impairment in episodic and chronic cluster headache. Cephalalgia 2010; 31:671–682.2. Rozen TD, Fishman RS. Cluster headache in the United States of America: demographics, clinical characteristics, triggers, suicidality, and personal burden. Headache 2012;52:99-113. 3. Headache Classification Committee of the International Headache Society (IHS). The International Classification of Headache Disorders, 3rd edition. Cephalalgia 2018: 38, 1–211. 4. Fischera M, Marziniak M, Gralow I, Evers S. The incidence and prevalence of cluster headache: a meta-analysis of population-based studies. Cephalalgia 2008;28:614-8. 5. Ford HL, Gerry Em Johnson M et al. A prospective study of the incidence, prevalence and mortality of multiple sclerosis in Leeds. J Neurol 2002; 249:260-5. 6. Klapper, et all, (2011) Cluster Headache. Headache, Face, Neck Pain Science, Evaluation and Management. Retrieved from https” books.Google.com. 23

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• 2.5mg Oral Zolmitriptan• 3.8mg M207• 6.0mg SC Sumatriptan

QTRYPTA’S PK PROFILE COMPARE TO SUMATRIPTAN SC SUGGESTS IT SHOULD WORK

CP-2015-007 MEAN PLASMA CONCENTRATIONS VERSUS TIME 0-2 HRS (N=20)

QTRYPTA’S PK PROFILE COMPARED TO SUMATRIPTAN SC

Qtrypta™

▪ 6.5% Annual growth rate

▪ Approximately $6 Billion Market in 2023*

▪ 5-HT3 Receptor antagonists 46% of overall market

▪ Indications

– Chemo therapy

– Gastroenteritis

– General anesthetics

– Dizziness

– Pregnancy

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ANTI – EMETIC MARKET

*BCC Research

▪ Original focus of Intracutaneous drug delivery ▪ 2017 Sales of $221 Billion▪ Demonstrated delivery– Proteins

➢ Biosimilar market – Vaccines

➢ Therapeutic Immunotherapy– Monoclonals

➢ Patent cliffs

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LARGE MOLECULES

✓ Life cycle management✓ Differentiation in Market

Preclinical Phase 1 Phase 2 Phase 3Target

Indication

Migraine

Small Molecule

Qtrypta™

Antiemetic

Cluster

Headache

FDA Filing

BiosimilarBiologics

TARGET YEAR END 2019 PRODUCT PIPELINE

Qtrypta™

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TARGET 2019 MILESTONES

Goal Timing

Publication of difficult to treat migraine data in Headache Q1

Completion of long term safety study – Qtrypta™ Q1

Initiate Phase II in Cluster Headache – Qtrypta™ Q3

Initiate Pre-clinical development with Biosimilar 2H

Partner Qtrypta™ YE

Complete Phase I with 5HT-3 Anti – Emetic Q4

File NDA - Qtrypta™ Q4

Name Title Experience

John Walker Chief Executive Officer

Greg Kitchener Chief Financial Officer

Hayley Lewis SVP, Operations

Donald Kellerman, PharmDVP, Clinical Development &

Medical Affairs

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EXPERIENCED MANAGEMENT