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Menlo Therapeutics Inc.June 2019
Developing Serlopitant, a Once-Daily Oral NK1 Receptor Antagonist for Pruritus
Special Note Regarding Forward-Looking StatementsThis presentation contains forward-looking statements, including statements about our plans to develop and commercialize our product candidates, our planned clinical trials for serlopitant, the timing of the availability of data from our clinical trials, the timing of our planned regulatory filings, the timing of and our ability to obtain and maintain regulatory approvals for serlopitant and the clinical utility of serlopitant, alone and as compared to other treatment options, the duration of patent protection, and other statements regarding strategy, future operations and plans, as well as assumptions underlying such statements. These statements involve substantial known and unknown risks, uncertainties and other factors, some of which are outside of our control, that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements, including risks related to the clinical drug development process, the regulatory approval process, our reliance on third parties, and our ability to defend our intellectual property. We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements we make. Additional information that could lead to material changes in our performance is contained in our filings with the U.S. Securities and Exchange Commission. The forward-looking statements in this presentation represent our views as of the date of this presentation. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we have no current intention of doing so except to the extent required by applicable law. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this presentation. Additional InformationThis presentation concerns a product that is under clinical investigation and which has not yet been approved for marketing by the U.S. Food and Drug Administration. It is currently limited by Federal law to investigational use, and no representation is made as to its safety or effectiveness for the purposes for which it is being investigated.
Safe Harbor Statements
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Menlo Investment Highlights
Serlopitant Phase 3 Development in 2 Pruritus Indications + Pipeline• Phase 3 prurigo nodularis
• Granted Breakthrough Therapy Designation by the FDA• Phase 3 psoriasis • Phase 2 chronic pruritus of unknown origin
Success in Large, Placebo-controlled Phase 2 Pruritus Trials• Hit primary endpoint in 3 of 4 large placebo-controlled Phase 2 trials• In all 4 trials, at every time point, showed improvement in pruritus vs. placebo
Large Market Opportunity in Pruritus• PN: 300-600K patients in the U.S. with no approved therapy• Psoriasis: 7.5M patients diagnosed in the U.S.; >90% experience pruritus
Cash Runway through the end of 2020• ~$122M cash as of 3/31/19
Solid IP• U.S. composition of matter to 2030, incl. extensions; methods of use to 2033
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Indication Phase 1
Phase 2
Phase3 2019 2020
Serlopitant Pipeline and Upcoming MilestonesSerlopitant is a highly selective small molecule inhibitor of NK1 Receptor, given once daily, as an oral tablet
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Recent and Anticipated Milestones
Psoriasis
Chronic Pruritus of Unknown Origin
EOP2 Mtg
File NDA
Prurigo Nodularis Complete enrollment
2 Phase 3 trials ongoing
Targeting to initiate Phase 3 program in 2019
Phase 2 initiated in January 2019
Trial Results
Complete enrollment
Targeted initiation
-2.5
-2
-1.5
-1
-0.5
0
0 2 4 6
Atopic Dermatitis – MTI-103
Serlopitant Met Primary Endpoints in 3 of 4 Phase 2 Pruritus Trials and Demonstrated Improvement Over Placebo at Every Assessed Time Point
VAS
% C
hang
e fr
om B
asel
ine
WI-N
RS C
hang
e fr
om B
asel
ine
-2.01-2.25-2.32 p=0.11
p=0.17
Chronic Pruritus – TCP-101
Treatment Week
Treatment Week
-9mm-15mm -19mm-18mm
-25mm
-36mm
-40
-30
-20
-10
0
0 2 4 6 8
Avg
Itch
VAS
Cha
nge
from
Bas
elin
e
p=0.001
Treatment Week
Prurigo Nodularis – TCP-102
Psoriasis – MTI-109
0%5%
10%15%20%25%30%35%
0 Day 7 2 3 4 5 6 7 8
WI-N
RS 4
-poi
nt R
espo
nder
Rat
e 33%
21%
-50
-40
-30
-20
-10
0
0 1 2 3 4 5 6
N=127
*
*Primary endpoint
N=257
*
Serlopitant 0.25 mg Serlopitant 5 mgPlacebo Serlopitant 1 mg
-42.5%p=0.013
-34.1%-41.4%
-28.3%
N=203N=484
*
5
p=0.028*
Treatment Week
26%
47%
Phase 2 Pruritus Studies – Serlopitant 5mg Responder Rate
WI-NRS 4-Point Responder Analysis (Baseline to Week 8)Prurigo Nodularis – TCP-102Chronic Pruritus – TCP-101
17%21%
Atopic Dermatitis – MTI-103
21%
33%
WI-NRS 4-Point Responder Analysis (Baseline to Week 8)WI-NRS 4-Point Responder Analysis (Baseline to Week 6)Psoriasis – MTI-109
p=0.17 p=0.028*
Placebo Serlopitant 5mg
23%
46%NRS 4-Point Responder Analysis (Baseline to Week 6)
N=53 N=52 N=39 N=43
N=101 N=102N=158 N=160
p=0.011 p=0.05
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*Primary endpoint.
23.1% 22.2%
46.2%
30.8%35.7%
29.6%
52.9%
33.3%Placebo
.25 mg
1 mg
5 mg
Post Hoc Analyses of Completed Phase 2 Studies with Serlopitant
Observed patterns in three completed Phase 2 studies in pruritus suggest the following types of patients respond better to serlopitant:• patients without inflammatory skin disease• older patients• patients with longer duration of pruritus
(1) Includes patients with a self-reported history of cutaneous lupus erythematosus, dermatitis, atopic dermatitis, contact dermatitis, dry skin, dyshidrotic eczema, eczema, asteatotic eczema, nummular eczema, hand dermatitis, keratosis pilaris, lichen planus, mechanical urticaria, neurodermatitis, photosensitivity reaction, psoriasis, rash, erythematous rash, pruritic rash, seborrheic dermatitis, stasis dermatitis, and urticaria.
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TCP-101 Post-hoc Analysis Results
N=27 N=26 N=27 N=18 N=26 N=26 N=28 N=34
Patients with a Self-reported History of Inflammatory Skin Disease (1)
Patients Without a Self-reported History ofInflammatory Skin Disease
NRS
4-p
oint
Res
pond
er R
ate
at W
eek
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Consolidated Safety Summary of Serlopitant
Evaluated in ~1,600 patients and shown to be well-tolerated, including in patients who received treatment up to one year
Most commonly reported treatment emergent adverse events across completed Phase 2 trials were:
N=670 N=1261TEAE Placebo (%) Serlopitant (%)
Urinary Tract Infection 2.5% 4.8%Nasopharyngitis 3.7% 4.8%
Diarrhea 3.4% 4.7%Headache 6.3% 4.4%
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Serlopitant for Pruritus Associated with Prurigo Nodularis
Granted Breakthrough Therapy Designation by the FDAPhase 3 Program OngoingTargeting 2020 NDA Filing
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Prurigo Nodularis
Prurigo Nodularis (PN)
A chronic intensely pruritic skin condition
Scratching leads to nodules which lead to more itch
No approved therapies in US or EU No effective treatment options
Image courtesy of Prof. Sonja Stander, University Munster
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Itch is the Primary Complaint Among 100% of PN Patients (1)
(1) Company survey of 30 physicians who treat PN patients.
0% 20% 40% 60% 80% 100%Itch
0% 20% 40% 60% 80% 100%
Sleep Pain / Burning / Bleeding Lesion / Appearance Other
Secondary Complaint
Primary Complaint
Limited Treatment Options Frustrate Patients and Physicians
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ManagementTreatmentDiagnosisLiving with PN
• Constant itch is extremely bothersome and alters lifestyle
• Majority of patients diagnosed by dermatologists
• Treatments are not always effective and may have limitations such as side effects
• “Trial and error” - topical steroids- antihistamines- intralesional injections- phototherapy - cryotherapy
• Patients resign themselves to live with it
• Severe patients may visit physician regularly (every 3-6 months)
“Yes, I had some type of relief. But, then, it started again. Every time I'd have a little bit of relief, and then it seems like another trigger…”
—PN Patient
“It can be frustrating...very limited options to treat patients with. And even the options that we have, sometimes don't help or help just a little bit. So, these patients you see on an ongoing basis.”
—Dermatologist
Attractive Commercial Opportunity in PN
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300K – 600K patients in US (1)
~185K PN patients visit physicians each year (1)
• 75% of PN patients are diagnosed by a dermatologist
On therapy 2-6 months per year (1)
$900 – $2,400 per month(2)
(1) Internal company estimates (2) Estimates based on company payer research and symptom relief analogs
• ~5K derms treat majority of PN patients
• Estimated 50 person field force could reach high-prescribing derms
• If approved, serlopitant would be 1st to market in PN
26%
47%
Placebo (N=63) VPD-737 5mg (N=64)
25%
54%
Placebo (N=63) VPD-737 5mg (N=64)
VAS – Primary Endpoint
WI-NRS - 4 Point Responder Analysis (Baseline to Week 8)Worst Itch NRS
NRS
VAS - 40mm Responder Analysis (Baseline to Week 8) Average Itch VAS
PN Phase 2 Study: Data Consistent Across Multiple Endpoints
PlaceboPlacebo Serlopitant Serlopitant
-9mm-15mm
-19mm
*
-18mm *-25mm * -36mm
-50
-40
-30
-20
-10
0
0 2 4 6 8 10
Aver
age
Itch
VAS
Chan
ge
from
Bas
elin
e (m
m)
Treatment Week
*p ≤ 0.05bars show standard error
Placebo5 mg -1.5
-1.9
-2.4
*-2.4*
-3.0* -3.7
-5
-4
-3
-2
-1
0
0 2 4 6 8 10
Aver
age
Itch
NRS
Cha
nge
from
Bas
elin
e (p
oint
s)
Treatment Week
*p ≤ 0.05bars show standard error
Placebo5 mg
13
p=0.002 p=0.05
serlopitant serlopitant
Ongoing NULARIS Phase 3 Studies in Prurigo Nodularis
Placebo
Primary endpoint
WI-NRS 4-point responder rate at
Week 10
Serlopitant 5 mg
MTI-105 (US) & MTI-106 (EU)
Over 160 patients enrolled in each trial as of 5/2/19Data expected H1 2020
N~280 per trialPN of ≥6 weeks Trial 1: ~50 sites in US Trial 2: ~50 sites in
Europe•Screening pruritus ≥7 on WI-NRS
•Excludes active pruritic skin disease
2-4 week screening 10-week treatment 3-5 week follow-up
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Serlopitant for Pruritus Associated with Psoriasis
Phase 3 Targeted for 2019
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Psoriasis
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Itch is the Most FrequentPatient Complaint(2)
Psoriasis is a chronic inflammatory disease of multiple systems causing areas of thickened skin, itch and pain No cure; treatments aim to improve symptoms ~7.5M diagnosed in the U.S.(1)
25% Mod / Severe Psoriasis(3)
75% Mild Psoriasis
(1) Armstrong, Dermatol Ther 2016 (2) Quality of Life & Work Productivity Impairment among Psoriasis Patients: Findings from National Psoriasis Foundation Survey 2003–2011 (3) National Psoriasis Foundation (4) Armstrong, JAMA Derm 2013
Moderate-Severe Psoriasis(1)
• 59% not treated• 9% biologics• 13% oral systemic• 17% topicals
Mild Psoriasis(4)
• 49% not treated• 42% topicals only
Current Standard of Care Does not Target Itch for Majority of Patients
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Large Market Opportunity for Pruritus with Psoriasis
~7.5M diagnosed patients in the US (1)
74% with moderate to severe itch (2) (~5.5M)
~75% not well controlled with current therapy(2) (~4M)
On therapy 4-8 months per year (3)
$900 – $1,200 per month(3)
~100-150 person field force could target high-prescribing derms and PCPs
(1) Armstrong, Dermatol Ther 2016 (2) Company market research (3) Internal company estimates
PSORIXA Phase 2 Study in Psoriasis – Data Announced Dec. 2018
Primary endpoint
WI-NRS 4-point responder rate at Week 8
MTI-109
N=204 Plaque-type PsO for
≥6 months, ≤10% BSA Pruritus ≥ 4 weeks WI-NRS ≥ 7 w/in 24
hours of initial screening
Serlopitant 5 mg
2-4-week screening 8-week treatment 2-week follow-up
Placebo
PsO = PsoriasisClinicalTrials.gov ID: NCT03343639
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21%
33%
Psoriasis Phase 2 Study: Data Consistent Across Multiple Endpoints
19
WI-N
RS C
hang
e fr
om B
asel
ine
WI-NRS (Change From Baseline)
Primary Endpoint - WI-NRS 4-Point Responder Analysis (Baseline to Week 8)
p=0.028
11%
21%
Secondary Endpoint: WI-NRS 4-Point Responder Analysis (Baseline to Week 4)
p=0.039
-3-2.5
-2-1.5
-1-0.5
0
-2.02
-2.77
*
*Follow-up visit at Week 10. Treatment through Week 8 only. Placebo Serlopitant 5mg
0%5%
10%15%20%25%30%35%
WI-N
RS 4
-poi
nt R
espo
nder
Rat
e 33%
21% p=0.028
WI-NRS 4-Point Responder Analysis (Change from Baseline)
*
Serlopitant for Chronic Pruritus of Unknown Origin (CPUO)
Phase 2 Initiated in Q1 2019
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Chronic Pruritus of Unknown Origin – A Large and Underserved Market
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CPUO Defined as pruritus lasting 6 weeks or longer, with no identified underlying disease
• Also referred to as idiopathic pruritus or pruritus of undetermined origin • Overlaps with pruritus of the elderly / senile pruritus Large market
• 8-15%(1) of ~80M chronic pruritus patients(2)
No approved therapies in US or EU
(1) Weisshaar, 2012 (2) Lifetime prevalence. Matterne, Acta Der Venereol, 2013
In patients with severe pruritus that cannot be eliminated by identifying and treating an underlying cause…, topical therapy and lifestyle changes are unlikely to be sufficient, and systemic
therapy should be considered. Given a paucity of data from randomized trials to evaluate various therapies, therapeutic
choices are largely based on clinical experience and anecdotal reports. Sedating antihistamines are commonly used as first-line
therapy but often have only modest efficacy….
Yosipovitch. NEJM. 2013
Phase 2 Study in CPUO
Primary endpoint
WI-NRS 4-point responder rate at
Week 10
MTI-117
Data Expected mid-2020
N~200
Chronic pruritus of unknown origin ≥6 months
WI-NRS ≥7 prior to screening
Serlopitant 5 mg
2- or 4-week screening
10-week treatmentPrimary endpoint at week 10
3-week follow-up
Placebo
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Financial Information and Conclusion
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Key Financial Highlights
Quarter Ended March 31, 2019
Cash & investments ~$122M
Operating expenses Including $1M non-cash stock-based compensation
$20M
Common shares outstanding 23.7M
Forward-Looking Information
2019 Estimated operating expenses*Including ~$3M-$6M non-cash stock-based compensation
$78M-$88M
Estimated cash runway Through 2020
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*Provided 2/27/19