Processa Pharmaceuticals, Inc. · NASDAQ:PCSA
Processa Pharmaceuticals — 20th Orphan Drugs & Rare Diseases 2023 Americas
17 slides from Processa Pharmaceuticals's conference presentation, Nov 16, 2023
Slide 1: Benefit-Risk Analysis for FDA Approval Requires More Than a Pivotal Trial
@ Processa Pharmaceuticals Benefit-Risk Analysis for FDA Approval Requires More Than a Pivotal Trial 20* ORPHAN DRUGS & RARE DISEASES 2023 AMERICAS November 16, 2023 David Young, PharmD, PhD President of R&D Processa Pharmaceuticals, Inc (NASDAQ: PCSA)
Slide 2: Disclaimer: Forward Looking Statements
Disclaimer: Forward Looking Statements The following summary is provided for informational purposes only and does not constitute an offer or solicitation to acquire interests in the investment or any related or associated company The information contained here is general in nature and is not intende
Slide 3: How Do You Approach Drug Development?
How Do You Approach Drug Development? CMC Studies Pre-Clinical Studies CLINICAL STUDIES Patient Commercial Benefit-Risk Benefit = -" - Benefit-Risk Analysis for FDA Approval "(7 Proce Pharmavestcale 20° Orphan Drugs & Rare Diseases 2028 Amerie
Slide 4: Benefit-Risk Assessment: Foundation for FDA's Regulatory Review/Approval
Benefit-Risk Assessment: Foundation for FDA’s Regulatory Review/Approval FDA States “For a drug to be approved for marketing, FDA must determine that the drug is effective and that its expected benefits outweigh its potential risks to patients. This assessment is informed by an extensive body of evi
Slide 5: Benefit-Risk Integrated Assessment
Benefit-Risk Integrated Assessment Added Importance for Orphan Disease Dimension Disease and Drug Analysis of Condition |+ Identify patients and the seriousness of their condition while recognizing and measuring what matters most to patients Current Treatment |+ Define standard of care and therapeut
Slide 6: Examples from Processa Founders and Processa Pipeline
Examples from Processa Founders and Processa Pipeline > Evaluate and Minimize Risk ¢ Acthar FDA approval - Risk is more than the safety of a drug (Questcor sNDA approval in 2010) > Next Generation Cancer Drugs with Proven Cancer-Killing Mechanisms and Superior Benefit-Risk Profile * Next Generation
Slide 7: Example 1: Acthar Approval for Infantile Spasms
@ Processa Pharmaceuticals Example 1: Acthar Approval for Infantile Spasms (Questcor sNDA approval in 2010) Risk = More Than Drug Safety in a Clinical Trial
Slide 8: Example 1: Risk is More than Safety of Drug
Example 1: Risk is More than Safety of Drug > Typically occurs in children less than 2 years old; occurs in 2,000 - 2,500 children per year > Premature death rate of 5-31% and > 60% of untreated/inadequately treated patients have serious mental/physical disabilities with other types of seizures deve
Slide 9: Example 1: Acthar Gel Used Off-Label for IS
Example 1: Acthar Gel Used Off-Label for IS Acthar, a purified preparation of adrenocorticotropic hormone in a gel, was used off-label for treatment of IS for over 30 years with IS sales in 2006-2007 representing almost 100% of sales Acthar was approved in 1952 and became a DESI (Drug Efficacy Study
Slide 10: Benefit Outweighed Safety Risk & Risks Associated with Off-Label Use
Benefit Outweighed Safety Risk & Risks Associated with Off-Label Use >» Negotiated with FDA what was to be included in the sNDA given conducting a trial when patients are treated off-label for IS was not ethical > BENEFIT + Emphasized that approval would provide consistent guidance on Acthar IS trea
Slide 11: Acthar FDA Approval
Acthar FDA Approval > Negotiated new, updated Acthar label with FDA > Questcor desired to eliminate many of the over 50 indications and obtained a modernized label with 19 indications by providing the data supporting each of the 19 indications > FDA appropriately evaluated more than the efficacy and
Slide 12: Example 1: Benefit-Risk Integrated Assessment of IS
Ce Example 1: Benefit-Risk Integrated Assessment of IS Dimension Disease and Drug Analysis of Condition + Identify patients and the seriousness of their condition while recognizing and measuring what matters most to patients Very serious orphan condition resulting in death or severe mental disabilit
Slide 13: Example 2: Next Generation Cancer Drugs
@ Processa Pharmaceuticals Example 2: Next Generation Cancer Drugs with Proven Cancer-Killing Mechanisms and Superior Benefit-Risk Profile Benefit = More Than Demonstrating Efficacy, More Patients Treated Next Generation Chemotherapy — Capecitabine (NGC-Cap) Next Generation Chemotherapy — Gemcitabin
Slide 14: Example 2: NCE Next Generation Chemotherapy (Not New Formulations)
Example 2: NCE Next Generation Chemotherapy (Not New Formulations) >» NCE Next Generation Chemotherapies (NGCs) are designed to improve the efficacy and safety profile for more cancer patients than their currently FDA-approved counterparts >» The cancer-killing metabolites of the NGCs are either the
Slide 15: Example 2: Oncology Opportunity
Example 2: Oncology Opportunity > The FDA-approved counterpart of each NGC is used to treat multiple types of cancers with more than 200,0000 new patients diagnosed in the US with these cancers >» Orphan-designated cancers presently treated (e.g., pancreatic, ovarian, gastric, biliary, small cell lu
Slide 16: Benefit-Risk Integrated Assessment
Ce Benefit-Risk Integrated Assessment Dimension Disease and Drug Analysis of Condition + Identify patients and the seriousness of their condition while recognizing and measuring what matters most to patients Serious condition with 35%-85% of treated patients not receiving ODR, poor survival Current
Slide 17: Benefit-Risk Assessment: Foundation for FDA's Regulatory Review/Approval
Benefit-Risk Assessment: Foundation for FDA’s Regulatory Review/Approval FDA States “For a drug to be approved for marketing, FDA must determine that the drug is effective and that its expected benefits outweigh its potential risks to patients. This assessment is informed by an extensive body of evi
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