Key Commercialization Activities, Resources and Partnerships Ofer Reizes, Ph.D Director, Skills Development January 17, 2019 1 Research vs. Innovation Research: Transforming knowledge into information Innovation: Transforming knowledge into products to create value for the consumer • Medicinal Product Innovation: Chemistry (structure/drug class), Method of Synthesis (process), Formulation, PK/PD, Pharmacogenetic, Device design, … • The Consumer: Patient, Physician, Third Party Payer (health-care systems, government) 2 Innovation-Novelty Novelty and “Newness” are distinct concepts • “Newness” implies a new creation (i.e. a new chemical entity) • Novelty can be a NCE or it can be a new use for a known compound, or a new method, formulation, etc. • From a patent standpoint, it must be something that is “non-obvious” to someone skilled in the Art • Novelty/Newness is a key component of Innovation 3 Innovation-Usefulness Unless an invention is useful, it is not innovative 4 Discussion Points • Commercialization Activities • What does it mean? • How do you prioritize? • Resources • What are they? • How do you identify them? • Partnerships • What is the purpose of partnerships? • How to do you seek them? 5 Key Activities, Resources and Partners Value Customer Proposition Segments Key Customer Activities Relationships Key Partners Key Channels Resources Costs Revenue Streams 6 How Do You Create Value? • Understand to whom are you creating value? • Patient, Hospital, Physician • Investors • Regulators, etc… • Designing experiment or choosing the commercial activities that provide the highest-possible evidence supporting your value proposition? • Describe your value proposition and one commercial activity that would create value. 7 Commercialization Activities Core Question: What key development and validation activities are required to deliver your value propositions? Value Customer Proposition Segments Key Customer Activities Relationships Key Partners Key Channels Resources Costs Revenue Streams 6 Key Activities • Activities are related to de-risking your program to achieve a successful market adoption. • What is “de-risking”? • Examples of commercialization activities: • Filing strong IP to protect your asset • Generate data: • Bench, pre-clinical, clinical • Define a robust regulatory path • Understand reimbursement • Keep in mind the value proposition (improve clinical outcome or reduce cost) 9 Navigating the Pathway of Translational Science Understanding your ecosystem is critical for your activities Medical Device Product Development In Vitro Diagnostic Product Development Needs Invention & Pre- Product Basic Preclinical First in FDA Filing/ Clinical Regulatory Identification Prototyping Clinical Launch Research Development Man Approval NCAI* NCAI* Biomarker Clinical Prototype Sterilization Regulatory Acute Human Selection Performance Iteration Non-clinical Clinical Review PMA/510(k)/ Testing Testing Mechanical Feasibility Regulatory de novo Bench Testing Studies Testing Review (IDE) Chronic Simulated Non-clinical Prototype Analytical Clinical Use Testing Testing Assay Performance Validation 1 Tissue Testing Biocompatibility Analytical Testing Validation Therapeutic Produc*Nt CDAIeve Projeclopmentt Activity Areas *NCAI Project Activity Areas Target Lead Pre- Phase 1 Lead ID … ID Validation Optimization Clinical Clinical Studies NCAI* Medicinal ADME Two Species Chemistry Efficacy Studies Efficacy Pharmaco- Studies kinetics Large In vitro Animal Toxicology Toxicology In vivo Toxicology Pre-IND 1 *NCAI Project Activity Areas 9 Pre-clinical Models – Can you show… • Disease modifying mechanism of action • Benchmark against competing programs • You are competing against everything including generics and pre- clinical programs • Generate and evaluate • Highest quality therapeutics or optimal design of your device • Metrics need to be understood from day one and constantly reviewed • Your expertise must be the best in the world 10 Clinical Trials are a Critical Activity 11 Minimum Viable Product • Generate prototype to demonstrate problem-solution relationship. • Articulate a problem definition based on your understanding/experience • Provide High-Level Engineering Specifications • Understand the minimum feature list • Prototype Customer Segment Value Proposition Prototype’ • Bench testing to demonstrate solution • Simulation to aid engineering specifications (e.g. stent design, fatigue testing,…) • Pre-Clinical model for safety and efficacy (acute & chronic models) • Generate data to substantiate your claim and test hypothesis • What are you trying to prove? • To whom are you trying to prove it? (customer, VC, FDA or regulatory body) • Think feasibility ($) versus statistical significance ($$$) 13 The Critical Three Elements Compelling Intellectual Clinical Property Need Compelling Intellectual Clinical Property Need Technological Solution MVP Technological Technological Solution 12 Goal of MVP Prototype • Show-n-Tell” to your stakeholder and help envision product features and discuss product-market fit. • Articulate the main idea and could point-out to design risks. This is important for Reg. activity of dFMEA for instance. • Helps you in choosing your product features and iterate prototype to determine the minimum features needed and not dilute your value proposition. • Question = What should your M.V.P prototype look like? 15 Commercialization Resources Core Question: What key intellectual, physical, human, and financial assets are required for your value propositions? Value Customer Proposition Segments Key Customer Activities Relationships Key Partners Key Channels Resources Costs Revenue Streams 6 Key Resources Resources Are Put to Work within a Business Model • What does it mean to have a Business Model? • A business model describes the rationale of how an organization creates, delivers, and captures value, in economic, social, cultural or other context [Medical]. • So, what are the Most Important Resources required to make the Business Model work? 17 Questions • How much will your activities/milestones cost? • Resources should match your critical activities • NIH, Foundation, DoD, philanthropic, SRA, etc. • Do you need to acquire or partner with others to obtain funding? • What commitments must be made to obtain support? • What human resources will you need? • What equipment resources will you need? • For Clinical Trials – will you need to identify a physician to collaborate with? Do you have Clinical Trial Design training? 16 Early Determination of Regulatory Pathway is Critical • 75% of all money raised will be spent on clinical studies and regulatory approvals • Major factor in getting financed • Major factor in Product Market Fit and strategy • Customer Discovery and Interviews can help • Identify the data set necessary to show that your invention does or does not deliver the hypothetical value in humans • Identify clinical trial design options • Regulatory approval often involves the opposite of IP goals similarity to existing approaches versus novelty • Identify path of least resistance in getting regulatory approval 17 Financial Resources • Venture Capital • Corporate Funding • Venture Debt • Bank Financing • Lease-lines • Venture Capital • Factoring • High Net Worth • Vendor Financing • Angels • SBIR/STTR • Self-Funding • SBA. • Friends • Family • Crowdfunding • Angels • Grants Lower Magnitude High Magnitude 20 Technologies Need to Generate Scientific Evidence (Data) • The system for ranking the quality of clinical evidence is known as “Evidence-based Medicine” • Demonstrating Safety and Efficacy of therapeutic requires hard data with clear endpoints • The highest level of evidence (and most expensive) is a prospective, randomized, placebo-controlled, double blind trial (RCT) published in a peer review journal • The value of your company and technology will be bracketed by the strength and clarity of your data • Major risk to getting funding: Equivocal Data 19 Typical Development Program Nonclinical • Animal data usually surrounding safety but efficacy might be added in POC Clinical • Phase I Usually safety, SAD, MAD, PK/PD, very short term • Phase II Efficacy signal studied, dose ranging, may include PK/PD Longer than in phase I, some safety data accumulated • Phase III More robust, larger numbers, and longer duration, would serve as pivotal trials for approval NDA/BLA • * POC proof of concept; SAD single ascending dose; MAD multiple ascending doses; PK pharmacokinetic; PD pharmacodynamic; NDA New Drug Application; BLA= Biologic Licensing Application 20 So will anyone pay for your technology? 21 Commercialization Partnerships Core Question: What strategic engagements are required to deliver the complete value proposition? Value Customer Proposition Segments Key Customer Activities Relationships Key Partners Key Channels Resources Costs Revenue Streams 6 Why Should You Seek Partners? • Building a business is not a silo-process. • There are dependencies to access Key Resources and Key Activities, that might not be readily available to your current business. • Goal is to: • Improve business competitive advantage, and/or • Reduce cost when acquiring commodities or service. • It needs to make sense to both parties: • Economic (e.g. supplier-type relationship) • Strategic (e.g. access to customers) • Know-how (e.g. unique technology) • “1+1 = 3” (e.g. joint venture, co-development, etc…) 25 Why Have Partners? ● Faster time to market ● Broader product offering ● More efficient use of capital ● Unique customer knowledge