What are the major issues or problems for which companies approach you for help?
Mr. Ball: First and foremost, prospective clients come to us with the problem of having a great idea, a concept that seems to have great therapeutic benefit, but no infrastructure to scaleup the process. Nearly as often, prospective clients come to us because they have a process that is not optimized and they need a partner with the experience to move up in scale.
Often times these are virtual or near virtual companies that may have a great relationship with a contract research organization, but that CRO doesn’t have the horsepower needed to perform the technical aspects of scale-up for analytics, validation, or support for chemistry, manufacturing, and control.
Mr. Geffroy: It very much depends on the type of client. Startups and small companies are looking for more than manufacturing, they expect a full-service offering that includes biomarkers, API, analytical support, preformulation and polymorph expertise, and drug product experience. They are also looking for advice and recommendation to take their clinical candidate to their key milestones in line with their funding strategy.
Clients come to us at various stages in the biomarker development pipeline, and we are able to assist them in progressing the biomarker into a clinically useful test. We then subsequently support our clients by running these tests in our CLIA laboratory for clinical trial enrichment strategies.
Mid-size and big pharma are looking for capacity, flexibility, and technologies. We have a strong reputation in biocatalysis, and our enzymes can create a lot of value for our clients. Recently, an eight-step API process was reduced to three steps using one of Almac’s Keto-reductase enzymes. This is especially attractive when the clinical candidate is in Phase IIb onward and cost of goods is becoming more important.
Finally, the most typical problem that all of our clients are facing—virtual, biotech, mid-size, and big pharma—are challenges with the physical form of their API or drug product. We have solved so many problems in this field, by screening and searching for all polymorphs and ensuring that the most stable form is taken forward to clinical development. It is also a nice opportunity to enhance IP for our client.
Mr. Head: In addition to its cGMP manufacturing services, Cytovance Biologics offers process development, cGMP cell banking, and support services for a number of clients. Underfunding and unrealistic timelines rank among the highest issues among companies seeking the services of a CMO.
Companies ask for help stretching their funds to move projects along, so they can reach the next round of funding. This can result in tight timelines. High pressure from investors getting through milestones to receive more funding can cause a rush through the development process. Cytovance understands the importance of setting realistic timeframes and expectations up front.
Mr. McGrath: One of the major issues customers face is the unpredictable behavior of launching a product. Risk mitigation becomes a key factor in outsourcing decisions. Customers from the large pharmaceutical arena are generally looking to mitigate their capital risk by allowing them to delay or avoid potential in-house investments/expansions.
Speed and time to market are at the forefront of our customers’ minds. Our process development and scaleup expertise allows customers to feel confident in their process as they progress through the pipeline to market. Smaller organizations are looking for a flexible and custom-tailored development program that will allow them to meet their early milestones and reach their IND filing as quickly as possible.
Dr. Nachtmann: From our perspective, clients are demanding process development, particularly for processes that are commercially not viable. Sandoz offers process development at all stages. Customers are also looking for flexibility in scale since predicting commercial requirements is difficult at early development stages.
Sandoz operates various scales in microbial (1,300 to 40,000 L) and mammalian (3,000 to 2x13,000 L) GMP manufacturing to fulfill these needs. Finally, customers are looking for regulatory assistance during the approval process, which Sandoz can provide based on the track record of various approvals in EU, U.S., Japan, and other countries.
Dr. Payne: Companies approach a CMO for breadth of integrated expert solutions to support customers in the transition from discovery to development, through the clinic, to commercialization and lifecycle management.
Specific expertise includes protein expression in mammalian cell lines through our GPEX expression system; preformulation (biophysical and physicochemical characterization); formulation and drug delivery technologies including oral, sterile, and inhalation; clinical and commercial packaging; and analytical support for API, components, and products.
Dr. Pohlmeyer: Next to experience in process/product development and manufacturing, customers request that CMOs provide regulatory support, qualified person support, preparation of INDs, IMPDs, and registration dossiers as well as participation in meetings with regulatory authorities. This is especially true for small biotech companies.
As a result of the reluctant investment climate, mainly driven by the lack of VC money, customers are asking for new business models for external manufacturing, for example co-development approaches or risk-sharing models. In any case, even in situations with a strict fee-for-service approach, the highest flexibility is requested to share the financial risks of biotechnology development projects.
Dr. Voss: Our customers approach us for our broad capability set covering the entire process chain from DNA to drug product (one-stop-shop), which is especially valued by small biotech customers. Furthermore we have high credibility as a strategic partner for large pharma/biotech companies, with an excellent track record for development, manufacturing, quality, and regulatory tasks. Recently we have further strengthened our global presence by the acquisition of Amgen’s Fremont, California, facility to even better serve our customers on a global base.
Mr. Wessels: We are being looked to more and more for our manufacturing expertise and technological solutions. For example, DSM offers technology solutions to significantly drive down the costs of biologics manufacturing including operating costs, capital requirements, and scaleup risks. To work with the shifting pharma model we offer our technologies through our CMO services and through licensing directly to biologics developers.
This is the same technology that DSM will install in the biomanufacturing facility currently under construction in Brisbane, Australia. The Federal Government of Australia and the Queensland Government recognized the benefits of this new “biologics plant of the future” concept and support the construction with grants. The facility will be operational by mid 2013.