You’re a leader at a small, emerging biotech company, and you’ve got a great molecule in hand. Early preclinical studies have been promising, and now it’s time to take it to the next level. You’re ready to start the journey toward filing an Investigational New Drug (IND) application and launching clinical trials. But now what? Your molecule could be the next blockbuster drug, but you’re a small company with limited resources. What’s most important to consider? Where do you go from here?
In January 2019, to better understand the needs of small biotech companies, BioPharma Dive’s Brand Studio surveyed small-biopharma leaders on their plans and concerns for developing their molecules. The results (see survey summary below) showed that three issues were of highest importance: getting their drugs to the clinic quickly (while not adding risk to their program), finding expert help and having adequate funding.
In a three-part article series, experts in drug development and commercialization share their insights into each of those areas, outlining the major issues to think about before choosing a partner company. First up, Shiva Khalafpour, vice president and head of commercial development, mammalian and microbial development and manufacturing at Lonza, addresses strategic choices that will help get your drug to the clinic quickly.
1. Think ahead about expression system
As you begin your drug development journey, one of the first decisions you have to make is choosing the right expression technology that is supported by a robust and scalable manufacturing process for your molecule. This can speed up your timeline greatly.
Select an expression system that has a strong track record in reliably delivering manufacture of products and has repeatedly demonstrated success, Khalafpour says. Choosing a proven system that your company or others have already submitted to regulatory authorities in earlier INDs can minimize the likelihood of getting difficult questions from regulators.
Also, make sure the selected host cell line for your product expression is easily traceable and well documented. Be sure you can demonstrate where the host cell line came from, provide genetic information that is known about it, especially in relation to any engineering that has been performed, and provide information on materials used to create it. This will allow you to present to regulators a well-documented package of information about the heritage of your cell line.
The preferred option is an expression technology with a robust manufacturing process that the U.S. Food and Drug Administration or European Medicines Agency regulators are familiar with and has proven its robustness to them. With such a choice, not only approval of your drug will likely be more straightforward avoiding significant delays, but it will also add value to your product for any future partnering discussions.
2. Avoid overcomplicated development and manufacturing plans
As a small biotech, you likely have limited in-house expertise and will need outside experts to move forward in your drug development journey. It may be tempting to try to incorporate the assistance of several different companies, but that can just make your journey more complicated.
Most small biopharma companies don’t have the bandwidth to work with several partners on the many aspects of developing their drugs. Consolidating everything under one roof is easier to negotiate up front, easier to manage over time and provides a faster way to get you to your next clinical milestone project timeline, according to Khalafpour. "A partner with reliable integrated drug substance and drug product development capabilities and a track record that can support you holistically and together with you create a development strategy with the endpoint in mind."
Compared to outsourcing work to several companies, working with just one is likely to shave several months off your timeline, as well as save on costs.
3. Take steps now to ensure manufacturing capacity later
At the early stages of any drug development journey, product demand is largely unknown. This makes it difficult to predict product requirements.
The key consideration here for small biotech is to look for a partner company with flexibility, one that has a business model that allows them to easily and quickly meet changing manufacturing needs and one that has the global manufacturing network to manage their products’ life cycle needs.
“Small biopharma companies need assurance that they have access to capacity as their products progress through clinical stages,” says Khalafpour. “If they suddenly have good clinical results, but they don’t have access to manufacturing capacity, they’ll be sitting on a good molecule and won’t be able to progress as fast as they want."
To help companies navigate through this uncertainty, de-risk and accelerate their clinical development, Lonza has created Ibex™ Solutions. Khalafpour noted that this innovative CDMO service offering anticipates clients’ capacity needs. “We set aside capacity for them, and we have designed our facility in a way that we can add more capacity very quickly,” says Khalafpour.
Taking your promising molecule through the drug development journey is an exciting process. It’s natural to want to speed up your timeline and get it into the clinic as fast as possible. But cutting corners could eventually compromise the quality of your product as well as your regulatory success.
For the best possible chances of a successful product launch, select a partner company that has the expertise and the experience to reliably deliver a quality product fast without compromise and guide you through a successful regulatory filing, and is able to quickly respond to your future product needs and fluctuating demand.
Learn more about Ibex™ Solutions
Ibex™ Solutions consist of three CDMO offerings: Ibex™ Design, Ibex™ Develop and Ibex™ Dedicate that span the complete biopharmaceutical lifecycle - from preclinical to commercial stages, from drug substance to drug product, all in one location. The variety of solutions offerings provide the flexibility of a complete program - from gene to drug product, or the option to drop in at a later stage depending on where you are in your journey.
- Ibex™ Design is a pioneering gene to vial package delivering drug product for your clinical trials within 12 months. Also included is a manufacturing slot reserved for your clinical resupply needs.
- Ibex™ Develop accelerates market readiness and can help you achieve BLA submission in 22 months. It offers tailored capacity to flexibly reach your clinical and commercial expansion requirements.
- Ibex™ Dedicate is technology agnostic supply solution that helps you save up to 30 months-time to market and control investment risk with tailored business and ownership models.
Learn more at www.ibex.lonza.com
BioPharma Dive’s Brand Studio surveyed industry leaders on their plans for developing new drugs. Close to 200 people responded to the survey. The majority of them (61%) were from small/start-up or mid-sized biotech companies, while 25% were from large pharma companies. Most (60%) are developing biologics/large molecules, and plan to develop their drugs by themselves or with a contract development and manufacturing organization (CDMO) partner, rather than selling or licensing their drugs to another company.
When asked about their “biggest concern about taking a molecule through early development,” speed was clearly the top issue for many. Thirty-seven percent of respondents ranked “We are worried about moving quickly enough, without adding risk to our program,” as their top concern. The second highest-ranked issue was “having enough funding to take our project to the next phase.”
Speed was also a primary concern in selecting a CDMO partner. Of survey respondents, 37% ranked “Speed: We need a CDMO that can provide accelerated timelines without adding risk to the project and delivers on stated timelines,” as a top issue, second only to expertise (41%).
Likewise, “Being able to move forward quickly,” was ranked the top concern by 46% of respondents, when asked their thoughts on what was important when considering applying for an accelerated approval pathway