Slide1: The Challenge: Create a uniform approach for valuing specific compounds and R&D portfolios for market opportunity What is it worth? Using a Patient-based Approach to Market Valuation


Benefits of Understanding Your Market: Benefits of Understanding Your Market Market Assessment-understanding the dynamics and forces Technology and Product Evaluation-what are the opportunities Licensing negotiation-what is it worth under what scenario(s) Product selection-creating a strategic framework for R&D choicemaking Portfolio analysis-Given our strategic direction, what kind of pipeline do we want to construct? Marketing strategies


Critical Business Decisions: Critical Business Decisions Is there a licensing opportunity in this emerging therapy; and, what is the product’s potential market value when seeking a development/marketing partner? How should we focus our R&D portfolio?


Some General Approaches: Some General Approaches Comparables Incremental value Top-down Market Based models NPV Real options Bottom up or Patient Based Forecasting


Some Key Questions: Some Key Questions What is the size of the target market Patient population, i.e., epidemiology Segmentation What are the unmet needs in the target market? Clinical Gold Standard Market What are the regulatory hurdles? What is the competition within the target market? What is the reimbursement environment?


Slide6: Critical Information: The Market —Base Year Number of Treated Patients Compliance Rate Price = Sales Days of Therapy per Year X X X Reconciled with prescription volume and top-line sales.


Slide7: Critical Information: Patient Population *May apply to the incident or prevalent population, depending on the indication. Definitions Number of newly occurring cases in a given year Total number of persons with a particular disease Number of persons diagnosed by a physician with a particular disease in a given year* Incident population Prevalent population Diagnosed population Drug-treated population Number of diagnosed patients who are under the care of a physician and who have received a prescription drug regimen*


Slide8: Unmet Clinical Need Criteria Mortality Morbidity Pain Non-Pain Symptoms Disability Hospitalization Efficacy Compound Attributes Effect on Non-Pain Symptoms Overall Response Complete Response Effect on Mortality Effect on Pain Overall Response Effect on Potential for Complications Effect on Hospitalization Effect on Disability Disease Severity Unmet Product Need How serious is the disease? How effective is the gold-standard therapy? Complete Response Compound Attributes Side Effects Drug Interactions/ Contraindications Dosage Form Dosing Frequency Onset of Action Minor Significant Safety Convenience


Patient-based Commercial Assessment : Patient-based Commercial Assessment ASSUMPTION # 1 ASSUMPTION #2 ASSUMPTION #3


Patient-based Commercial Assessment : Patient-based Commercial Assessment Diagnosis Rate Prevalence of Molecular Target


Patient-based Commercial Assessment : Patient-based Commercial Assessment Diagnosis Rate Prevalence of Molecular Target Clinical Unmet Need & Product Unmet Need Assessment of Product Compared to Current Gold Standard Primary research/Conjoint Analysis Competitive landscape Analog Models Sales and prescription audit data Market- and region-specific factors


Patient-based Commercial Assessment : Patient-based Commercial Assessment Diagnosis Rate Prevalence of Molecular Target Clinical Unmet Need & Product Unmet Need Assessment of Product Compared to Current Gold Standard Competitive landscape Primary research/Conjoint Analysis Analogue Models Sales and prescription audit data Market- and region-specific factors Degree of Clinical Advance (Improved efficacy, product attributes) Price/Demand Elasticity Regional Considerations Pricing Analysis will be further refined in Phase III/pre-launch


Patient-based Commercial Assessment : Patient-based Commercial Assessment Diagnosis Rate Prevalence of Molecular Target Clinical Unmet Need & Product Unmet Need Assessment of Product Compared to Current Gold Standard Competitive landscape Primary research/Conjoint Analysis Analogue Models Sales and prescription audit data Market- and region-specific factors Degree of Clinical Advance (Improved efficacy, product attributes) Price/Demand Elasticity Regional Considerations Pricing Analysis will be further refined in Phase III/pre-launch 5. Market Value = 2. Candidate Population x 3. Market Uptake x 4. Price


Slide14: Case Study-Licensing “CelGen Industries, Inc.” Medium-sized biotech firm Focus on cancer and inflammatory disease Potential in-licensing opportunity: “AntiMab’s” BT-504 “AntiMab” is a small biotech involved in the development of antibody therapeutics targeting cancer epitopes BT-504 is currently in Phase II development, to date has completed 2 Phase I trials 1 Phase II trial


Product Overview: BT-504: Target Oncology Indications: Non-Hodgkins lymphoma (NHL) Chronic Lymphocytic Leukemia (CLL) Acute Lymphocytic Leukemia (ALL) Clinical Data Favorable pharmacokinetics Phase I/II data shows good safety profile Preliminary activity in Phase I/II promising Internal R&D evaluation of BT-504 is positive Product Overview: BT-504


Tasks to Accomplish: Tasks to Accomplish Your tasks are to: 1). Assess the market potential of BT-504 2.) Prioritize the value of three oncology indications (NHL, ALL, CLL) and advise on clinical development strategy. 3.) Provide a recommendation on in-licensing BT-504. (Based on current portfolio and assuming CelGen has the resources for a maximum of 4 products)


CelGen’s Pipeline: CelGen’s Pipeline


Key Questions in the Market Assessment: Key Questions in the Market Assessment 1. What is the size of the product’s target patient population? 2. How will the product be positioned within the market (first-line therapy, sub-segment of the population, etc.)—function of product profile compared with current gold standard therapy? 3. What peak-year uptake (%) can be expected? 4. What is a reasonable pricing scheme? 5. What is the value ($) of this market?


Prevalence: Prevalence


Sales: Sales


Go to Spreadsheet: Go to Spreadsheet


Impact of Market Research on R&D: Impact of Market Research on R&D Synergies with current R&D program Is the product aligned with CelGen’s internal capabilities/expertise? R&D Strategy Focus on oncology franchise? Risk/cost associated with developing internal R&D capabilities vs. Market Value of BT-504 Reallocation of resources to support in-licensed program Impact on R&D staff expertise, interest, and morale


Strategic Determinations: Strategic Determinations 1. What is the market value of BT-504? 2. Should BT-504 be in-licensed? 3. Which indication(s) should be prioritized? 4. What is the impact of in-licensing BT-504 on CelGen’s R&D portfolio?


Case Outcome: Case Outcome Product In-licensing agreement $9 million (1995 US$) upfront payments in equity and investment fees $17.5 million equity funding prior to approval $30.5 million in milestone and option payments Total potential value of the collaboration $57 million. Co-promotion in the U.S., CelGen has complete commercialization rights ex-US. Developed for NHL Approval for second-line Rx of CD20+ NHL in US 1997, Europe 1998, extensive off-label use 1st line. Clinical studies ongoing for first-line NHL, CLL, aggressive NHL. G7 market value 2000 $400M+


The Value of a Patient-Based Approach to Market Strategy: The Value of a Patient-Based Approach to Market Strategy Understanding your markets can help craft strategy Licensing opportunities R&D activity prioritization and portfolio construction Foundation for NPV and other valuation approaches Better long-term strategic decision-making