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Long-Term "GameChangers" in Biotech

When I’m hunting for unicorns, I start with biotech. They won’t all be winners. But in a well-constructed portfolio, enough of them will hit their commercial targets with sufficient force to make their shareholders very happy, explains Hilary Kramer, editor of GameChangers.

Start at the earliest stage of development with Kaleido BioSciences (KLDO), which just went public on February 28. It is not much to look at today.

The company has a dream of harnessing the body’s internal microorganisms to clean harmful chemicals out of the blood more efficiently. Currently, it is using $75 million in cash to build on that vision. We aren’t even looking for Phase 2 trial data before 2020, so investors need a lot of patience.

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But as far as I’m concerned, the technology is worth at least $500 million now and should become more valuable every day that it moves towards real commercial applications. Maybe in five years, KLDO's early shareholders will be extremely happy.

Or look at Kiniksa Pharmaceuticals Ltd. (KNSA), which hit the market nearly a year ago and is still a long way from a real commercial profile.

As far as I’m concerned, KNSA won’t even be at a meaningful revenue stage before 2022, when its next-generation dermatology drugs will be cleared to prescribe. While a lot can happen in the intervening time, if everything plays out well, this company can become a profoundly lucrative enterprise.

Sometimes the market gives us a chance to grab a more advanced company at a start-up valuation. CytomX Therapeutics (CTMX) was a $1 billion stock on Feb. 25, only to lose half that value on what people called "an earnings miss".

Earnings aren’t even a factor here for the foreseeable future. This company is all about the dream of new broad-spectrum cancer therapies that will one day be ready for the regulators to review.

In the meantime, there’s $430 million in cash to back up that vision. Burning through that capital to create something real is what good executives do. Biotech companies are no exception.

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But if you don’t have years to wait, the biotech world is full of established franchises that are making money right now and are also priced at a fraction of what the ride-sharing unicorns currently command.

Take BioMarin Pharmaceutical (BMRN) as an example. It is one of the biggest biotech stocks on Wall Street with even bigger prospects in curing hemophilia and other blockbuster diseases. In my view, BMRN is on the verge of sustainable profitability at its current scale.

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