Disease Landscape/

Trial Update Monitoring

Facilitator: Rajni Sharma

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What it is and why we do it?
Components of Disease Landscape

➢ Summary
➢ Disease Overview
➢ Epidemiology
➢ Diagnosis and Treatment
➢ Patient Journey and Healthcare Burden
➢ Marketed Therapies
➢ Unmet Needs
➢ Pipeline Overview
➢ Late / Early-Stage pipeline profiles
➢ Abbreviations
➢ Appendix
Components of Disease Landscape

➢ Disease Overview
➢ Epidemiology
➢ Diagnosis and Treatment
➢ Patient Journey and Healthcare Burden
➢ Marketed Therapies
➢ Unmet Needs
➢ Pipeline Overview
➢ Late / Early-Stage pipeline profiles
➢ Abbreviations
➢ Appendix
➢ Summary
 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

Disease Overview

➢ Disease Definition
➢ Etiology
➢ Pathophysiology
➢ Disease Categorization
o Subtypes
o Severity

➢ Clinical Features
➢ Prognosis
➢ Disease Complications, Comorbidities & Patient Care
 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

Epidemiology

➢ Incidence
➢ Prevalence:
o Diagnosis prevalence
o Treatment prevalence

➢ Focused markets
➢ Epidemiology Trends:
o Co-morbidities
o Mortality
 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

Diagnosis and Treatment

Diagnosis Treatment

➢ Diagnosis Guidelines ➢ Treatment Guidelines

➢ Diagnosis Work-up ➢ Treatment Algorithm focused
➢ Diagnostic Tools and Techniques markets: US, EU etc.

➢ Referral Pattern ➢ Treatment Guidelines -

Comparative View
➢ Diagnosis Algorithm focused
markets: US, EU etc. ➢ Care Team for Disease

➢ Barriers to Diagnosis
 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

Patient Journey and Healthcare Burden

➢ Patient Journey
➢ Disease Burden
➢ Economical burden including
cost of therapy
➢ Impact on QoL
 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

Marketed Therapy
 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

Unmet needs

➢ Unmet needs and Treatment gaps

➢ Different perspectives:
o Patient perspective
o HCP perspective
 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

Pipeline Overview

➢ Competitive landscape
➢ Clinical Endpoint Analysis
➢ Disease Activity and QoL scores
➢ Late-Stage Pipeline Profiles (Ph3):
o Product Profile
o Clinical Trial Profile
➢ Early-Stage Pipeline Profiles (Ph2)
➢ Early-Stage Candidates (Ph1 &
preclinical)
➢ NDR / Discontinued Candidates
 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

Late / Early-Stage pipeline profiles

 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

Early-Stage Candidates (Ph1 & Preclinical)

Product Target
Phase MOA Comments Link
Company patient/indication

Secukinumab • NCT04274166: Withdrawn (Contracting

/Cosentyx never completed, closed the IRB)
interleukin-17A PG with non-healing
(Universities Phase 1/2 • NCT02733094: Single-arm Study to [Link]
antagonist ulcer
collaboration Assess a Potential Effect of Anti-IL-17.
with Novartis) PCD: September 10, 2019

• Confirmed severe
• Study initiated of local application of the
form of any type of
Monoclonal Antibody (Mab) sB24M in
pyoderma with
Patients With PG on May 24, 2021
lesions of the skin [Link]
sB24M (SWISS CD47 / TNF-α • Monoclonal antibody (Mab) sB24
and muscle tissue Trial
BIOPHARMA Early Phase 0/1 Monoclonal negatively regulates immune-inflammatory
• Secondary Bulletin
MED GmbH)2 antibody processes through CD47 / TNF-α Axis
treatment failure up .com
promotes epithelialization of damaged
to one previous
tissue3
TNFα antagonist
• PCD: August 25, 2022
and corticosteroid

• ATI 450 is being developed by Aclaris

ATI-450 Investor
Therapeutics as an oral, selective, small
(Aclaris R&D day
Preclinical MK2 inhibitor Not specified molecule inhibitor of the mitogen-activated
Therapeutics ) ppt
protein (MAP) kinase-activated protein
Aclaris
kinase-2 (MK2) pathway4
 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

NDR / Discontinued Candidates

Phas
Asset Sponsor Design Patients Population N End Points Challenges
e

Janssen • >18 years old with • Primary endpoint:

• No
Bermekima Research & • Open Label Single history of PG with or • Change in Physician's Wound
II 10 Development
b (Xilonix)1 Developmen Group Assignment without other Assessment & Patient's Global
Reported
t systemic disease Assessment (28days)

• Primary endpoint:
• % subjects at Day 126 with
complete closure of the PG
• Randomized, • Classic PG patients 16:(NCT0 target ulcer confirmed 2 weeks
Gevokizum Double-blind, with active 2315417) later (at Day 140) and without
III XOMA(US) • Terminated
ab2,3 Placebo Controlled, pyoderma 9:(NCT02 the need for rescue treatment
Parallel Assignment gangrenosum ulcer 326740) • Secondary endpoints:
• % subjects at Day 126 with
reduction in the target ulcer area
of ≥ 75% or ≥ 90% from baseline

• Primary endpoint: at week 12

• Change in Physician and patient
Global Assessments for Active
Skin Manifestations vs baseline • Terminated
APD334 • Open-label, Proof of • Change in DLQI and C-reactive (Sponsor
Arena • PG with active, non-
(etrasimod) II Concept, Single 2 Protein Levels vs baseline decision to
4 Pharma healing ulcer
Group Assignment • Assessments of Target Lesions terminate the
& Punch Biopsies study)
• Secondary endpoints:
• Number of patients with AE,
severity of AE
 Diagnosis and Patient Journey and Late / Early-Stage
Disease Overview Epidemiology Marketed Therapies Unmet Needs Pipeline Overview Abbreviations
Treatment Healthcare Burden pipeline profiles

Abbreviations
Summary

➢ Executive Summary
➢ Key findings – Disease Overview
➢ Key findings – Epidemiology
➢ Key findings – Comorbidities & Patient care
➢ Key findings – Treatment
➢ Key findings – Unmet needs
➢ Key findings – Disease Burden
➢ Marketed Drug Summary
➢ Key finding – Phase III/II Candidates
➢ Key finding – Phase II Candidate
➢ Endpoints Comparison – Phase III & Phase II Trials
➢ Key Findings - Launch Timelines
Trial Update Monitoring
Facilitator: Rajni Sharma
Clinical Trial and Its Terminology

• What is a Clinical trial: • Terminology :

o [Link]
important-terms-related-clinical-trials
Secondary Sources – Clinical trial/pipeline information
Websites/databases
Clinical trial/ Pipeline information Key points
Public databases
[Link] is a database of privately and publicly funded clinical studies
[Link] conducted around the world. Purpose is to provide information about clinical studies
to the public, researchers, and HCPs
EudraCT is the European Clinical Trials Database of all clinical trials of
EudraCT
investigational medicinal products with at least one site in the European Union
The Clinical Trials Search Portal provides access to a central database containing the
trial registration data sets provided by the registries listed in Australia, China,
International Clinical Trials Registry Platform
[Link], EU clinical trials, ICRTN, Netherland, Brazil, India, Germany,
Cuban, Korea, Iran, Japan, Pan-Africa, Sri Lanka, Thai and Peruvian trials.
Private/commercial databases
A database for drug development and research, disease treatment and decision
Adis R&D Insight
making
Citeline's Pharma projects has been covering pharma R&D across global markets for
35+ years. With 68,000+ drug profiles including 15,000 drugs in active development,
Pharmaprojects
it’s the go-to resource for preclinical, clinical, and pipeline coverage, and lifecycle
management tracking.
Cortellis Clinical Trials Intelligence is a powerful resource to accelerate clinical
Cortellis Clinical Trials Intelligence development decisions, inform portfolio strategy, select trial sites and provide key
competitive intelligence.
Comprehensive, accurate and up-to-date source of pharmaceutical clinical trials data,
TrialTrove
supported by experienced industry analysts

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Secondary Sources – Clinical trial/pipeline information
[Link] (Extracting pipeline, RSS feed, and checking the details of the clinical trial)

Strong search queries/

good understanding of what we are looking for

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Secondary Sources – Clinical trial/pipeline information
How to read the trial???
• The key is Glossary on [Link] (all the answers are in the glossary)
• Once you are familiar with the terms it is easy to explain and understand the trial

Important aspects of any clinical trial Changes in the trial – the trials are updated as and when
✓ Trial title required
✓ Trial name (Acronym) • Monitoring the changes in the trial is very important for the
✓ Recruitment status of the trial important assets in the pipeline of your therapy area
✓ Sponsor How to do that???
✓ Estimated enrolment • RSS feed (Clinical [Link])
✓ Study start date • Checking the trial site once/twice everyday
✓ Primary completion date Process
✓ Primary and Secondary endpoint (Outcome measure) • Check the last updated date
✓ Inclusion and Exclusion criteria for patients/ volunteers • Change history of the trial (gives you comparison of what
✓ Trial sites (along with investigator and locations in a country) has changed)

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Secondary Sources – Clinical trial/pipeline information
EudraCT

How to search??

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Clinical trial timeline and mapping

• Study Start Date

• Primary Completion Date: when primary endpoint follow-up is complete for all study participants
• Study Completion Date: when all endpoint follow-up for all participants completes

• Objective of timeline monitoring:

o To anticipate the trial completion and data readout
o To estimate regulatory filing and launch

• Mapping/ assumptions for drug launch:

o Expected topline data release: Study start date + recruitment period + primary endpoint follow-up time + 3 months
o Expected regulatory filing: Expected topline data release time for submission enabling Ph3 studies
o Expected launch: Expected regulatory filing + 10-12 months based on regulatory authority (FDA/ EMA) and application type (NDA/
sNDA/ BLA/ sBLA)

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Thank you for
attention!