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Exclusive Life Science Feature viral agents; the product won FDA and EU approvals in 2012. Gilead will commercialize the product, relying on Aptalis to manufacture and supply the oral powder. The product uses the Aptalis Microcaps tastemasking technology in an oral powder form which is easier on the small patients, who may begin therapy as young as age 2. Aptalis has also continued to expand its market range for Pylera, an oral-capsule combination antibacterial of bismuth subcitrate potassium, metronidazole, and tetracycline. In combination with omeprazole, Pylera is indicated for the eradication of Helicobacter pylori and prevention of relapse of peptic ulcers in patients with active or prior H. pylori-associated ulcers. Pylera gained FDA approval in 2006, and the company has followed the mutual recognition path in Europe and is now launching the product in the first 10 European markets. The elementary method of combining the three most-prescribed drugs for H. Pylori into a patented capsule-in-capsule therapy may simplify treatment for patients. THREE Ms IN ONE: CENTERED ON THE PATIENT Whether it is mindset, mission, or model that best describes how Aptalis approaches specialty pharma, according to Verwiel, the uniting principle of development is matching delivery modes to patient needs. "Whenever we talk about development projects, we want to make sure that we understand what we can do to provide the patients with better care." He describes how the company recently restructured its sales force to create CF account managers, who visit CF centers to deliver supportive information and product offerings for improving patient care. It has also created an award-winning CF patient support program called Live2Thrive, employing an interactive website where members, caregivers, and physicians can get help managing the disease with educational resources, along with access to Aptalis products such as vitamins and nutritional shakes — the patients are often struggling to avoid malnourishment. But one of the most important patient-centric activities occurs long before products reach the market. Although drug optimization early in drug development is always a good idea, it is especially critical with SP products. Poor formulation — improper concentration, density, aggregation — can defeat the best delivery method. Conversely, the right formulation can help achieve the best, most tolerable, and effective treatment experience. "All of our three main technologies enable product profiles that can be tailored early to optimize drug performance," says Verwiel. Two engines drive the selection process for SP in-licensing and development: the reliable old standard, "unmet medical need" for new products, and complementary presence in the targeted therapeutic areas for existing products. "We know the GI and CF spaces very well, so we can identify opportunities and have dialogues with the parties that hold the assets. And quite often the dialogue can be long term. For example, we began speaking with ProStrakan Group years before we in-licensed Rectiv," says Verwiel. The company also maintains a dedicated pipeline group, which he describes as "an experienced team of medical and pharmaceuti- cal scientists whose mission in life is to look at medical need with formulation science and invent, design, and test new concepts that we could develop. These can be new technologies, new applications for existing drugs, new applications for our proprietary technologies, and combinations of all three. The outcomes of this invention feed into our development portfolio and are in the clinic." BEYOND THE Ms: SPECIALTY PHARMA HEADS ON These days, in the dearth of new primary-care blockbusters, large pharma companies have ventured into new territories such as biotech, personalized medicine, and premium orphan drugs in the quest for profitable new products. Will they now gobble up specialty pharma as well? More than an observer, Verwiel still sees the large pharma companies with a somewhat distanced eye. "In specialty pharma, there's still a large unmet medical need, and you can still make significant advances in the medical services you offer. So it's very logical that Big Pharma is entering the space. But specialty pharma demands a different organizational DNA than large pharmas can maintain. Their only recourse may be to buy a successful specialty drug company and let it operate on an independent basis to preserve the special DNA, the special culture needed to be successful in specialty pharma." It's true that only people can formulate a mission, and mindset, and a model to ensure the continued prosperity of a sector that effectively created itself. Specialty pharma may be one space Big Pharma cannot completely overtake, and its future role in innovation may grow larger than anyone imagines — or could have imagined. SPECIALTY PHARMA LESSONS LEARNED President and CEO Frank Verwiel of Aptalis shares some key lessons from his company's experience that may be useful to others in the specialty pharma business. • It's important to be extremely focused. "We made a very deliberate choice to be in GI and CF and focus on those areas." • It's important to know what you do — and even more important to know what you don't do. "For an organization of our size, we have a very good understanding of what's in our market. That means that the probability of success is much, much higher." • You need to have a specific kind of DNA in the organization that recognizes opportunities. "In R&D;, the commercial area, or a larger organization active in multiple areas, seeing new opportunities is much more difficult to do." • Do the basics first, but explore new knowledge. "This is true for everybody but maybe more so for specialty pharma: We were originally only in the GI component of CF, but as our patient-centric SP approach taught us more about the disease and what a patient goes through, we were able to move beyond GI into the pulmonary area of CF." July 2013 LifeScienceLeader.com 27