Many biopharma companies have promising molecules to develop, but they need to partner with outside companies to take their molecules through to manufacturing for clinical and commercial supplies. Choosing the right partner can have substantial implications far beyond the list of services the partner offers. The right partner can help to ensure continued funding, as well as fast and assured progress toward commercialization.
Funding is indeed a major concern for small biotech companies. In January 2019, to better understand the needs of these 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, finding expert help and having adequate funding.
Part one of this series focused on the first priority, exploring what small biotech companies might look at when assessing how fast a partner can get their drug to clinical trials. Part two looked at how finding the right expert support can move a company further along the drug development journey, enabling a seamless scale-up from Phase II trials to commercial supply.
In part three of this series, the experts at Lonza offer advice on funding issues that small biotech companies need to keep in mind as they look for partners. What qualities in a partner have the potential to enhance your molecule's attractiveness to future investors or development partners?
Three areas are of primary importance, says Abdelaziz Toumi, Head of commercial solutions, Ibex™ Design and Develop, and Charles Christy, Head of commercial solutions, Ibex™ Dedicate:
1. Choose an experienced company that offers platform processes using proven technologies.
Developing a drug is a complex process with many different interfaces and potential regulatory pathways. Choosing a partner that does not have the experience needed to see you through the steps with few or no bumps in the road can result in very costly delays and missed milestones, and can jeopardize future funding that depends on hitting those milestones.
"Leverage the experience of a trusted partner who has developed molecules before," advises Toumi. "Find a company that can bring experience and know-how and efficiency, because they have already developed many molecules and their platform is established."
Partnering with an experienced company will also look attractive to big pharma companies when you are looking for a development partner for your drug. "Big Pharma will do their due diligence, looking at your entire development process," says Toumi. "They’ll look at how you established the cell line, if you took any risks, is the process able to be commercialized and more. There are so many possible pitfalls along the way."
"If they find issues with the process, the product will lose value, you'll have to repeat studies and you'll delay your molecules' development by a year or more," adds Christy. In fact, forty-seven percent of Complete Response Letters (CRLs) from FDA name deficiencies in CMC requirements”, meaning a delayed market entry by an average of 14 months1. "So having an established process without any deficiencies is critical to the valuation and progress of the molecule." This further highlights the need for an experienced partner with proven processes and a strong track record of analytical and regulatory success including agency interactions and BLA submissions.
2. Choose a company that can do everything under one roof, from DNA to commercial supply.
A recent study by the Tufts Center for the Study of Drug Development2 found, "substantial financial benefits to sponsors from employing a single-source, as opposed to a multi-vendor, model of manufacturing outsourcing."
The study cited two sources of such savings: shorter development periods and earlier launches. The mean savings gained from faster development was $20.6 million, while the value of an earlier launch was on average $24.1 million per approved new drug, for a total of $44.7 million. Referring to the Tufts study, Toumi says: "the savings can be even higher if you have a blockbuster drug."
"This makes absolute sense, because with one vendor, you are reducing interfaces, avoiding complexities," says Toumi. "It simplifies everything. You have one point of contact, one program manager taking care of everything and they have end-to-end-responsibility. If you have an issue, you don't have to figure out who is accountable or who made the mistake. If you have one integrated partner, whatever the problem is, it's the partner's responsibility to figure it out."
Conversely, he notes, if you have many vendors, it can take months to figure out where a problem originates.
Working with a single vendor also saves you time and money in terms of audits, inspections, staffing and other steps. The partner, says Toumi, "is not just a provider of a drug, it's a provider for a whole solution."
3. Choose a company that offers exceptional predictability.
As a small biopharma company, you likely have limited funding until you hit your next milestone. You don't want to run into any surprises that could increase your costs or delay your timelines.
"Work with a trusted partner that is going to get you to your milestones at the right time, at the right speed," says Christy. Many companies will promise such results, but as Christy notes, with Ibex™ Design offering, Lonza commits to timelines, a minimum quantity of materials, and resupply to the clinic, as needed.
Lonza is able to make those commitments, he says, due to their highly flexible manufacturing assets. They can ramp up manufacturing quickly, if needed, to meet changing market demands. Most similar contract development and manufacturing organizations, explains Christy, are not as flexible and unable to meet the challenge of unpredictable clinical supply needs.
"Such predictability makes your financial forecasting easier," says Toumi. And working with a market leader that offers a well-designed, highly predictable process makes your product more valuable to any company that is considering in-licensing your product.
Think beyond your initial milestones. Make your partner decision based on quality, expertise and flexible manufacturing models, not price, advise Toumi and Christy. Choose one that has the experience you need, and a well-developed process, and your molecule will look much more valuable to potential investors. Alternatively, you could continue to commercialize yourselves with a process that will stand the test, together with a partner with a proven track record and game-changing ownership models. For example, if you opt for Ibex™ Dedicate, operations could begin with Lonza’s team running the facility, with ownership transferred to you later.
Welcome to 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 a 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.
- DiMasi JA, Smith Z, Getz KA. Assessing The Financial Benefits of Faster Development Times: The Case of Single-source Versus Multi-vendor Outsourced Biopharmaceutical Manufacturing. Clinical Therapeutics. 2018;40:963-972.