BioProcess Technology Consultants

CASE STUDIES

1 General CMC Consulting from Due Diligence through Commercialization

2 Evaluation of a Novel Discovery Technology

3 Market Assessment and Competitive Analysis of Contract Manufacturing Opportunity

4 Process and Product Development

5 Manufacturing Operations and Strategy

6 Manufacturing Asset Disposition

7 Evaluation of Product Opportunity

Case Study #1
General CMC Consulting from Due Diligence through Commercialization

A mid-sized biotechnology company engaged BioProcess Technology Consultants to assist in the evaluation of an in-licensing opportunity. At the time, the product candidate was approved for commercial sale in Europe but had not been registered in the US. As part of the due diligence team, BPTC performed a technical review and assessment of all CMC information available for the product, including:

Reviewing all documentation pertaining to production and testing of the biopharmaceutical product, including both manufacturing of bulk active (drug substance) and formulation and fill/finish of final drug product
Evaluation of the quality control laboratories and all data obtained to date regarding product stability and properties
Auditing the current manufacturing plant and associated facilities for compliance with US cGMP regulations and to determine the potential for expansion of the facility to accommodate expanded production
Critical review of on-going process development, optimization, and product characterization and development of recommendations for additional development and manufacturing activities required to support late stage clinical trials and commercialization of the product
Preparation of written and oral reports to Client senior management and manufacturing team detailing our findings, recommendations, and key action items.

Based on our technical and CMC regulatory review as well as other pertinent information, the client chose to in-license this product. BioProcess Technology Consultants was then retained to oversee the process development and manufacturing of the product and to assist in the commercialization of the product in the US. We participated in frequent on-site meetings at the manufacturing site, helped identify and solve many process-related problems, and enabled our client to successfully complete Phase 3 clinical trials and obtain product registration in the US without jeopardizing the existing European approval. This product currently has global sales in excess of $100 million.
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Case Study #2
Evaluation of a Novel Discovery Technology

A start-up biotechnology company had developed a novel and proprietary discovery technology to improve binding and specificity of existing therapeutic antibodies and to develop human antibody therapeutics directed toward new target molecules.

BioProcess Technology Consultants was asked to critically evaluate market opportunities for the most effective utilization of this discovery technology to rapidly create a product pipeline and visibility for the company and its technology. The Client intended to use their technology to develop follow-on antibody products with improved binding characteristics compared to antibodies that were already on the market. By analyzing currently marketed antibody products and antibody products in late stage clinical trials we were able to:

Develop options for efficient utilization of the Client’s discovery technology through self-funded drug discovery and development, corporate partnerships, or out-licensing strategies
Prepare a short list of suitable therapeutic molecular targets based on best options for technical success
Analyze market potential for therapeutic indications and provide recommendations based on technical review and market assessment
Provide input into shortest route to market and development plan and budget to achieve this goal

Using the valuable market and strategic information and analysis we provided, the Client was able to focus their limited internal resources on improving specific antibody therapeutic products for which there were a high likelihood of technical and market success. As a result, the Client has successfully engineered product candidates in two therapeutic areas and is currently in discussions with potential partners for further development and commercialization of these products.
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Case Study #3
Market Assessment and Competitive Analysis of Contract Manufacturing Opportunity

A medium-sized biopharmaceutical company with existing manufacturing capacity for products requiring BL-2 containment found that they had excess capacity due to a slow down in the development of their proprietary products. As a means of offsetting the fixed cost of maintaining the manufacturing facility, the company was considering using its spare capacity to offer contract manufacturing services to other companies requiring BL-2 containment for the production of their products.

BioProcess Technology Consultants was hired to assess the potential market opportunity for a contract manufacturer in cGMP manufacturing under BL-2 containment and to determine the requirements for entering and competing in this niche market. To help the client make this important strategic decision, we performed the following activities to assess the market drivers for BL-2 biopharmaceutical manufacturing:

Analyze current capacity supply and demand for products requiring BL-2 containment and for other products that might fit into the client’s facility
Assess customer satisfaction with current contract manufacturers (CMOs) offering manufacturing services in this area
Determine ideal CMO capabilities and qualifications for this product type
Recommend cost-effective, success-enabling strategy for entering CMO business

BioProcess Technology Consultants market research and analysis revealed that while there was sufficient existing capacity for BL-2 manufacturing services by contract organizations, there was significant customer dissatisfaction with the level of service and quality provided by these existing CMOs. We recommended that the Client consider entering the CMO market through a focused campaign to position themselves as a service-oriented and quality-based organization with a breadth and depth of expertise in the production of these specific BL-2 products. The Client has initiated an effort to develop this new business venture within their company and may offer contract services to the industry shortly.
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Case Study #4
Process and Product Development

A start-up biotechnology company was developing a biotherapeutic product and was manufacturing the product for early stage clinical development using an expression system developed in an academic laboratory. The process had been run at the 10 L scale but a significant scale and productivity increase was needed to support Phase 3 trials and commercialization. The company had identified a CMO to undertake the development of activities to a new, more efficient production strain and a scaleable, robust, and cost-effective manufacturing process and to manufacture the product at a larger scale for future clinical trials. BioProcess Technology Consultants was engaged to oversee the activities at the CMO and to provide technical and regulatory input on the new strain development program and the process development program to enable reliable, economic manufacture of the product. The Company chose us because of our expertise and strong experience in strain development, resolution of production issues for difficult-to-produce proteins, and their project management experience in overseeing and contributing valuable input into process development activities.

BioProcess Technology Consultants worked closely with the management team of this company and oversaw all aspects of the manufacture of the product. Specific activities performed by our team included:

Reviewing data from existing production system and manufacturing runs and assist company in identifying an appropriate strategy to develop and test new potential expression systems for production of clinical grade material in sufficient quantities for trials
Overseeing analytical development plan and support efforts to develop effective purification strategies
Working with a CMO to rapidly design and execute purification development plan through careful management and experimental design
Implementing a development plan that will lead to production of clinical grade material for Phase 3 within 12 months
Overseeing all product development activities at CMO through weekly teleconferences and quarterly face-to-face meetings with CMO production team
Providing QA support including GMP facility audit, batch record review and approval, man-in-plant during GMP production, review and resolution of deviations, and implementation of CAPA plans at the CMO

Through these efforts, BioProcess Technology Consultants was able to provide the Client with a cost-effective, robust manufacturing process scaled up to meet the product needs for Phase 3 human clinical trials which are ongoing. Further, we provided the Client with management oversight of all outsourcing activities, input into partnership discussions, and coordination of product supply for various experiments also performed at outside vendors.
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Case Study #5
Manufacturing Operations and Strategy

A Client with a small scale manufacturing facility for purification of a non-therapeutic protein required assistance in determining the suitability of the facility for purification of therapeutic proteins under cGMP. BioProcess Technology Consultants (BPTC) was engaged to perform this evaluation and gap analysis based on our knowledge of regulatory and operational requirements for such facilities. As a result of our review, BPTC determined that it would be possible to upgrade the existing facility at a reasonable cost. We assisted the Client achieve this by taking the following actions:

Review of manufacturing strategy for therapeutic protein
Audit of manufacturing facility for compliance issues and development of recommendations for achieving compliance
Complete budget for bringing existing facility into compliance and complete budget for outsourcing purification and fill/finish of the product
Oversight of implementation of facility modifications, including both physical and quality assurance changes

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Case Study #6
Manufacturing Asset Disposition

An existing BioProcess Technology Consultants Client had determined that one of their manufacturing facilities was no longer required to execute their overall product commercialization strategy. They desired a tenant to take over a multi-year lease on the fully equipped, FDA-inspected and approved facility as quickly as possible.

Bioprocess Technology Consultants used its broad industry knowledge and network of contacts to quickly identify a company to acquire this facility by doing the following:

Assemble complete, ranked list of companies developing biopharmaceuticals that would be candidates for using a commercially approved facility with mid-size bioreactors and process development suites
Work closely with the Client to develop contacts within target companies and explore interest levels
Manage database of all contacts, interactions, and follow-up items to achieve successful outcome of identifying suitable tenant
Prepare all sales materials, including web site, brochures, and other written and oral material describing facility and business terms
Attend trade conferences and distribute written material, make contacts, and provide further information to existing contacts

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Case Study #7
Evaluation of Product Opportunity

A Client needed help in prioritizing their product pipeline for continued development. Among the products being evaluated was a novel antibody with potentially broad applications in oncology.

BioProcess Technology Consultants conducted technical due diligence and market research on this product candidate to determine the potential value of such a product. This due diligence and analysis included:

Contacted and interviewed several physicians and other experts in discovery and development of antibody therapeutics
Determined market acceptance of therapeutic concept
Reviewed all available in vitro and in vivo data demonstrating efficacy of therapeutic concept to determine if data supported program continuation
Designed decision-enabling research program to address physician concerns about product and to provide sufficient information to determine whether continuation of program was warranted

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