Short Bowel Syndrome and Complex Crohn’s Perianal Fistulas

from the beginning

Short Bowel Syndrome and Complex Crohn’s Perianal Fistulas

IMPORTANT NOTICE

1. Takeda’s Initiatives in Gastroenterology (GI) Therapeutic Area

• We aspire to be the leading GI company

• Our GI strategy has evolved to focus on leadership in critical unmet needs in GI and liver diseases

2. Short Bowel Syndrome

• 2. Short Bowel Syndrome

• Definition:What is Short Bowel Syndrome (SBS)?

• Definition:Absorptive function of the gastrointestinal tract by parts

• Definition: Main Causes of Short Bowel Syndrome (SBS)

• Epidemiology: Prevalence estimates of SBS

• Symptoms and Burden on Daily Life: Clinical symptoms and the mechanisms

• Symptoms and Burden on Daily Life: Burden on patient daily life

• Symptoms and Burden on Daily Li fe: SBS Patient’s Voice

• Symptoms and Burden on Daily Li fe: Survival rate of adult patients with SBS

• Recommended treatment strategy/methods:SBS management/treatment strategy flow chart by ESPEN

• Recommended treatment strategy/methods:Therapeutic approaches and Goals for SBS patients

• Recommended treatment strategy/methods:How to enhance intestinal adaptation by growth factors?

• GATTEX/REVESTIVE is the first and only approved GLP-2 analog for the treatment of SBS

• Clinical Trials:GATTEX/REVESTIVE Phase 3 study with adult SBS patients (STEPS study)

• Clinical Trials: STEPS Results - Primary Endpoint Responder Rate

• Clinical Trials:STEPS Results - Secondary Endpoint Absolute Reduction PS Volume

• Clinical Trials: STEPS Results - Adverse Events -1

• Clinical Trials: STEPS Results - Adverse Events -2

• Clinical Trials:GATTEX/REVESTIVE Phase 3 study with pediatric SBS patients (TED-C14-006 study)

• Clinical Trials: TED-C14-006 Results - Primary endpoint

• Clinical Trials: TED-C14-006 Results - Other efficacy

• Clinical Trials: TED-C14-006 Results - Most common AEs occurring in patients treated with GATTEX/REVESTIVE

• Take-home Messages

3. Complex Crohn’s Perianal Fistulas

• 3. Complex Crohn’s Perianal Fistulas

• Disease Background: What is Crohn’s Disease

• Disease Background: What are Crohn’s Perianal Fistulas (CPF)?

• Current standard of care: Despite medical and surgical advancements, Crohn’s Perianal Fistulas remain challenging to treat

• Symptoms and Burden on Daily Life:What it looks like, if you have CPF and have setons?

• Symptoms and Burden on Daily Life:CPF Patient’s Voice

• Characteristics:What are s tem c ells?

• Characteristics:What are mesenchymal stem cells (MSCs)?

• Characteristics:Therapeutic areas in wh ich clinical trials of MSCs are being conducted

• Characteristics:Advantages of a dipose d erived s tem c ells (ASCs)

• Product characteristics of ALOFISEL

• Characteristics:Proposed immunomodulatory mechanism of action of ALOFISEL

• Clinical trials:Development of ALOFISEL in Europe

• Clinical trials:Development strategy in Japan (clinical data package)

• Clinical trials:Development of ALOFISEL in Japan

• Clinical trials:Combined remission at Week 24 and 52

• Clinical trials:Relapse at W52

• Clinical trials:Overview of Adverse Events

• Clinical trials: Ongoing Clinical studies

• Manufacturing and Supply of ALOFISEL

• Efforts in Logistics

• Take-home Messages

4. Q&A Session

• Q&A Panelists

• Q&A 1

• Q&A 2

• Q&A 3

• Q&A 4

• Q&A 5