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its own antibiotics. At some point, most micro-innovators face a similar choice — sell the research, sell the platform, or bet the farm on
product development. All of them, by definition, choose development.
ACT's Rabin makes it clear why the company went beyond the
platform-business model. "We see ourselves as a regenerative medicine company focused on the development of treatments, and as
such, are oriented as a product-development company," he says.
"Because of our size, we tend to use common pluripotent cell platforms as our starting materials for our various product development
efforts, such as embryonic stem (ES) cells and induced pluripotent
stem (iPS) cells. However, in the end our focus is much more on the
therapeutic product opportunity than on the underlying cell source
we use as a starting material for manufacture."
Once committed to the product-development path, companies
typically begin to refine each product according to their understanding of how it will be used in practice — a step I have taken to
calling "market modeling."
For example, Melinta selected specific formulation and dosage/
delivery forms in consideration of how patients with serious infections receive treatment. Its lead clinical candidate, delafloxacin,
has both IV and oral formulations, allowing patients who begin
IV treatment in a hospital to transition to oral dosing for homebased care. "We believe this IV-to-oral switch has the potential to
increase patient convenience, lower the overall cost of treatment,
and reduce the length of hospital stays," says Szela. Delafloxacin
covers a broad spectrum of pathogens, and Melinta expects it to
be a preferred treatment in hospitals because it avoids the need for
doctors to specifically identify the pathogen and change medications in mid-treatment, adding cost and complexity.
WHERE THE RUBBER MEETS THE ROAD
We pause here, because I thought a normal article length was insufficient to share all the valuable experiences and lessons gathered
from our three micro-innovator companies and their CEOs. When we
return with Part Two, we'll look at how the companies have embarked
on the often rough road of drug development, paying for translational
research and clinical trials, planning the development pathway and
designing the needed studies, and conducting the trials aimed at
regulatory approval. Please stay tuned for the next installment of "The
Micro-Innovators."
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December 2013
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