Opportunity Mapping Expertise
Companies and institutions may want an early-stage analysis of feasibility and potential for their R&D programs or technology platforms to help raise or make an investment, or to attract BD partners. It may be to validate the selection of drug discovery targets, or for allocation of internal resources or R&D budget. Such early stage assessments can help a management team to explore scenarios for company growth, value creation, risk and financing needs.
We have rich experience mapping opportunities for individual programs, for multiple programs in a portfolio, for drug discovery platforms, or for enabling technology platforms. Opportunity mapping involves quantifying the realistic commercial potential, mapping out the go-to-market path, and describing the key risks and sensitivities. The process may also involve primary market research to garner additional expert opinion and to validate assumptions. Such assessments often involve making trade-offs between multiple potential lead indications, and across several geographic territories.
Target Product Profile Development
Of fundamental importance to the integrity of this kind of assessment is consistency between the assumptions driving commercial potential and those describing what it will take to get there. For drug products, the anchor point for this alignment is a realistic description of the launch product, captured in a Target Product Profile (TPP) which describes an achievable and competitive set of product performance parameters, against which all other assumptions should be grounded. Any significant mismatch between the assumed time, cost, risk and return for a program or platform can be extremely damaging for a business.
Commercial Opportunity Characterization
To characterize commercial opportunities, we typically start by listing all possible indications for a product or all applications for a technology and undertake a high-level prioritization. This may be followed by a deeper assessment of a lead indication. These assessments typically include a clear description and sizing of the target patient population, current market size and dynamics, identification of key medical and payer constituents, current medical practice and treatments, disease burden and unmet medical need, current and future drug and non-drug competition, pricing potential and value arguments, launch and patent expiry dates. In addition to the parameters above which feed into a peak year sales estimate, we often provide budget estimates to launch and sell the drug (i.e. marketing, medial affairs, commercial data infrastructure, supply chain, patient services and sales).
Path to Market
For path to market, we create a high-level map of the required discovery and development steps required for the lead indication. This often includes the key steps in the CMC process of delivering compliant clinical and commercial supply of drug product. For each major step we estimate the duration and cost. Key program risks or challenges are also described. Benchmarking analysis of drugs previously developed for an indication (or one similar to it) can yield information on the possible wording of the approved indication, the nature and number of trials required, the patient numbers needed to treat, the primary and secondary endpoints, and the duration of late stage trials. For nascent indications, we rely more heavily on our subject matter experts in all aspects of the discovery and development process, and on industry or KOL contacts, to derive assumptions.
The below list of recent projects and case studies can help illustrate the type of expertise we typically provide in this area.
- Commercial development plan for a mesenchymal stem cell therapy in acute GvHD: Our client was a leading UK Research Institute requiring assistance in assigning internal commercialization funding to the most promising research groups within the university. Alacrita was asked to assess the viability and market attractiveness of selected projects and to develop individual Commercial Development Plans (CDP) that could be reviewed by the funding committee. One such group requiring a CDP was developing a new mesenchymal stem cell (MSC) therapeutic for the treatment of acute graft versus host disease.
- Opportunity mapping and prioritization of new R&D programs: A clinical stage pharmaceutical company was interested in leveraging their existing antivirals drug discovery and development capabilities to expand the company’s future product pipeline. To that end, they requested a comprehensive and methodical strategic assessment of the viral disease landscape and the prioritization and profiling of commercial opportunities therein.
- Indication selection assessment for clinical stage oncology small molecule: A venture capital firm was interested in an independent indication selection strategic assessment of a clinical stage small molecule oncology asset they had invested in, based on the preliminary results of a phase 1a/1b basket clinical study.
- Assessment of TLR immunomodulators in oncology and HIV: A European publicly-listed biotech was facing a Phase III milestone for its lead immunomodulator and had a next generation technology in IND enabling studies. The company was operating in a field which had suffered notable setbacks over the years, partially due to deficiencies in clinical development strategy, and was concerned that its Phase III trial would not show the expected benefits. In this context, the client hired Alacrita to undertake a comprehensive review of all its clinical and preclinical data to assess the best options for developing a sustainably differentiated position.
- Opportunity mapping for hemojuvelin antibody: A US-based biotech company developing a novel biologic which impacts iron metabolism, wanted help deciding which disease and patient groups would make the best lead indication for development and commercialization. The company had already narrowed its shortlist of diseases down to three, based upon strength of scientific rationale. Alacrita was engaged to prioritize these based on commercial opportunity and development feasibility.
- Indication triaging for gene therapies: A biopharmaceutical company developing novel immunomodulatory enzymes was exploring the potential clinical use of its lead candidate in combination with AAV based gene therapies to treat rare diseases. Alacrita was commissioned to provide an overview of the opportunity in minimizing host-rejection of AAV-based gene therapies in development for 13 rare diseases.
Case Study: Mapping an oncology project opportunity to support fundraising
Challenge: The technology transfer function of a leading cancer research institute needed a development plan for a promising small molecule lead series against a novel anti-invasion/anti-metastasis target for solid tumors. The plan was required to support fundraising from venture capital investors with the complication of demonstrating the prospective differentiation of inhibitors for this proprietary target versus recent new entrants.
Solution: Our consultants reviewed the scientific literature and the lead academic’s proprietary results to determine therapeutic areas where the known activity provided the most compelling rationale. We then assessed the competitive landscape, both for marketed drugs and for those in clinical development, to determine the most likely options for positioning.
Next we created a credible target therapeutic profile and positioning for a successful agent, developing an outline screening cascade, combining the academic’s proprietary in vitro and in vivo assays with others relevant to the mechanism of action and a cascade of standard ADME, PK and toxicology screens. This was to create an optimization and a preclinical development plan for the lead product. Indicative costs for process development and primary and secondary production of clinical trial materials were then produced.
We assessed strategic options for early-stage clinical development and produced an integrated project plan and timetable. Finally, we costed the overall plan, with a range of contingencies, to provide a funding requirement for the project. The client then successfully used the plan to obtain a term sheet from a venture capital investor.
Case Study: R&D pipeline planning for gene therapy company
Challenge: A biotechnology company developing proprietary synthetic biology technology in gene therapy expression for several clients was looking to evolve its business model. The company engaged Alacrita to find a way to build their own internal cell and gene therapy (including gene editing) pipeline and to put together a plan in order to establish medical indications the technology could be applied to. The company was mainly interested in developing the technology where its technology would deem the product as differentiating, ensuring the development of stand-out products.
Solution: Alacrita first reviewed hundreds of relevant gene therapy indications, and triaged potential clinical opportunities for which the synthetic technology was best suited based on pre-determined criteria. We then conducted a preliminary assessment of each opportunity, profiling the top seven clinical opportunities by scoring each disease and scrutinizing the market opportunity.
In our final report, we outlined the feasibility of the development of this technology in each profiled indication and analyzed how well the technology fitted, described the market opportunity and the scope for product differentiation, and pointed out the potential challenges and risks facing the client.