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Is the TPP for your IND MIA?

Tuesday Nov 11, 2014

By: Al Doig  (adoigatbptcdotcom)   and Taylor Burtis  (tburtisatbptcdotcom)  

You are a small to mid-size biotech company with a molecule you believe has efficacy for the treatment of one or more human disease indications. Your pre-clinical studies appear to have gone well and a suitable, but non-optimized, production process is nearly in place that should generate sufficient drug material to support Phase I dosing studies. Your investors are pushing you to get your molecule into the clinic by filing an IND with the U.S. FDA and/or a Clinical Trial Application (CTA), which contains an Investigational Medicinal Product Dossier (IMPD), for European Medicines Agency (EMA) regulators or both if your management is thinking globally. The multimillion dollar bet at this point is; are you really ready to start assembling the CTD sections of a Phase I clinical trial application?

If you ask us, the answer will be a resounding “no” if you haven’t completed a comprehensive, stage appropriate, target product profile (TPP) review upon which to base your pre-IND regulatory strategy. Many claim there are too many unknowns at this point to possibly generate a useful TPP. From experience, we argue that if you haven’t spent the time to develop a robust TPP as a development management tool, you will surely be overlooking, missing, or underappreciating an issue or issues that will cause program delays and drain available cash as you move forward. Think of the TPP as a dynamic document that provides a clear focus on product development objectives over time. Remember, the landscape is littered with biotechs with good molecules that crashed and burned for lack of a well-thought through regulatory strategy.

At a minimum, you need to perform some competitive intelligence scouting as part of the TPP exercise. A TPP should include a search for information on clinical trials of drugs targeting the same indication. You need to know as much as you can glean from this site in order to evaluate your strategy. For example, if you think your drug will be administered by injection, and someone is already in clinical trials with an orally administered drug, you need to consider the implications related to mode of administration now, not later.

We also recommend you review the relevant FDA Advisory Committee documents to garner information about how the Agency is evaluating drugs targeting your indication(s) of choice or drugs having related modes of action. These committees provide independent expert advice to the Agency. The information provided by industry and the FDA to the various committee’s is available to the public.

If you are thinking about global markets, we recommend you include a review of the European Public Assessment Reports, (EPARs). EPARs are published by the EMA for every medicine granted a central marketing authorization by the European Commission following an assessment by the EMA’s Committee for Medicinal Products for Human Use (CHMP).

Beg, borrow or steal the time and resources to put a solid TPP in place before preparing your IND or IMPD. The effort will pay dividends over the life cycle of your product. The first payback will be a clear, well written IND or IMPD that will be accepted by the regulators and launch your clinical trials.

In a future post, we will present our thoughts on how to create project and document management tools for generating your IND or IMPD application. Be sure to subscribe to our RSS feed so you don’t miss it.


Inaugural BioProcessing Asia (BPA)

Saturday Oct 18, 2014

BPTC is proud to be a sponsor of the  inaugural Bioprocessing Asia (BPA) conference in Langkawi, Malaysia. Tom Ransohoff  (transohoffatbptcdotcom)  , Vice President, will be attending on BPTC’s behalf and presenting a paper entitled “The Potential Impact of Biosimilars on the Biopharmaceutical Capacity Landscape​,” which will provide an analysis of the topic using data from BPTC’s proprietary bioTRAK database. We hope to see you in beautiful Langkawi!


Integrating Critical Early Development Activities

Wednesday Sep 3, 2014

By Susan Dana Jones  (sjonesatbptcdotcom)  

Integrating Critical Early Development Activities to progress From Clone to Clinic® cost-effectively is the theme of IBC’s newly launched track on Success Factors for Early Stage Biologics and the topic of what is sure to be a lively discussion panel hosted by BPTC. This track, part of the BioProcess International Conference & Exhibition held in Boston this fall, will take place on Wednesday October 22 starting at 10:30 AM. The content of this panel will range from funding strategies, manufacturing decisions, and “doing more with less,” all of which is designed specifically to provide management and staff of biotech start-up companies with a road-map of the “must-have”, “nice-to-have”, and “can be postponed without major risk” CMC development activities.

Many start-up biotech companies don’t have their own development capabilities and therefore seek the services of a qualified CRO or CMO to perform the different activities. However, it is the sponsor, not the CRO or CMO, who is responsible for the proper conduct of all development activities, the risk assessment of proceeding with a sub-optimal process, and the eventual quality of the product produced for human clinical evaluation. Managing the sponsor-CMO or CRO relationship is therefore a critical activity in developing new biopharmaceuticals. For biosimilar development, the sponsor company must be even more vigilant to insure that the CRO/CMO expectations and activities are fully aligned with the goal of designing a process to produce a product that is comparable in all the required ways to the reference, innovator product.

Cell line development is the first, and critical path, activity for any development program and this is a potentially lengthy activity for products that must be expressed in a mammalian system. With a multitude of options available through service providers and enabling technologies available through purchase or license, the decision of how and where to initiate this activity sets the strategy and risk profile for the entire development program. The history of the production cell line will be carefully reviewed by regulatory authorities worldwide and therefore any risks taken at this early stage could impact the eventual request to initiate trials in human subjects. Does this mean that cell line development must be lengthy and expensive? Not necessarily. We don’t believe that it needs to take more than 4-5 months to obtain a clonal, genetically stable, high expressing mammalian cell line for products that don’t require novel enzymatic processing, co-factors to assist in folding or secretion, or other unusual elements. The bigger question in early development is what else can be done while the cell lines are under construction?

The IBC track is focused on integration of all activities, minimization of upfront cash flow while mitigating the most likely risks in early development, and achieving a product that performs as desired in animals and patients. BPTC’s panel discussion at 4:30 PM will include seasoned industry veterans from both BPTC’s team of expert consultants and from companies developing novel, first-in-class or biobetter products. By drawing together experts in all aspects of early development and accessing the breadth of knowledge across this group, BPTC and IBC hope to implant the importance of well-managed, well-thought out development strategies starting with lead candidate selection through to delivery of the drug to patients in need of new therapies. If you still haven’t registered for the conference, you can register using BPTC’s discount code, BP14SKRE3, for 20% off the standard rates.


Cell Culture World Congress USA 2014

Monday Sep 1, 2014

Join BPTC at Terrapinn’s Cell Culture World Congress USA conference, co-located with the HPAPI Congress, in Boston, MA from September 15-16.  We look forward to mingling with fellow cell culture experts to discuss process enhancements, innovative technologies, and bottle-neck reduction opportunities. Please come visit us at booth 13 to learn how we can help improve your upstream process and accelerate your development programs.


2014 BioProcess International Conference & Exhibition

Friday Aug 29, 2014

Join BPTC at IBC’s 11th Annual BioProcess International Conference & Exhibition in Boston, MA from October 20-23, 2014. In addition to exhibiting at the conference (booth #810), BPTC consultants will be actively participating in a number of sessions.  Susan Dana Jones, Vice President and Principal Consultant, will be leading a panel discussion on “Identifying and Integrating Critical Early Development Activities to Enable Successful Progression from Discovery to First-in-Human Clinical Trials” on October 22 at 4:30PM. Susan will also be leading a course titled, “Introduction to BioPharmaceutical Development and Manufacturing” with Sheila Magil, Senior Consultant.  This course will provide an overview of the technical considerations and activities required to progress a biopharmaceutical from discovery through early process development and manufacturing. If you still haven’t registered, take advantage of our discount code, BP14SKRE3, for 20% off the standard rates. See you there! For more information, visit: