On the day Francois Nader joined NPS Pharma in 2006, the company had $260 million in cash, 435 employees, and a big fat no from the FDA.

NPS was burning through about $140 million a year and its business model, built entirely around bringing an osteoporosis drug named Preos to market, was dead in the water. The FDA gave the company a chance to reintroduce Preos, which would have cost NPS $50 million it didn’t have. Besides, by the time the company figured everything out and (hopefully) got its approval, the osteoporosis market would probably be flooded with competitors and the whole exercise would have been moot.

To make matters worse, the price per share of NPS stock had dropped by 80 percent.

To put it mildly, the Bedminster-based NPS was hemorrhaging. “This is when I put on my physician’s hat,” Nader say. “The first thing to do was stop the bleeding.”

The treatment was rather blunt and unpleasant, actually. The commercial and medical departments at NPS needed to go immediately, which meant the cutting of 200 jobs. Before the wound closed fully the staff would drop to 17 and the company would shift direction so sharply that its original headquarters in Salt Lake City would close and the very reason it existed would give way to a brave new world on the fringe.

Eighteen months later, NPS would turn into a company that would eventually inspire an Irish biotech to pay handsomely for the right to buy its rare-disease drugs and turn a fifth of the company’s shareholders into millionaires before the end of a workday.

Nader will be the keynote speaker at the New Jersey Technology Council’s annual CFO Breakfast Awards event on Thursday, June 11 at 7:30 a.m. at Forsgate in Monroe. Melissa Orsen, CEO of the NJ Economic Development Authority, will also speak at the event. Cost: $110. Visit www.NJTC.org.

Born in Lebanon, where his father was a CFO, Nader grew up wanting to help humanity be healthy. He figured that being a doctor was the way to go, and he received his M.D. from St. Joseph’s University in Beirut. “But being a physician meant treating only one patient at a time,” he says. “The reason I got into the [pharma] industry was because I could help thousands of people at a time.”

Nader’s first role in public health was in vaccines, where he got to witness millions of people get help. He traveled the world, living in France for 10 years before he landed a position with Marion Merrell Dow in Canada. The company sent him to Kansas City 22 years ago to develop a U.S. medical affairs group and a global economics group and he has been in the United States since.

In 2006 a mutual friend introduced Nader to Tony Coles, then-CEO of NPS. Coles was looking for a chief medical officer, a position Nader was profoundly disinterested in. Coles came back with “an offer I couldn’t refuse” and a unique title — chief medical and commercial officer, Nader says.

“If you know anything about our industry, you know that those two terms should not exist in the same sentence,” he says. But the title only lasted six months until Nader became COO. Nader and Coles then looked at their two options.

“The easiest would have been to sell our assets and transform the company into a royalty shell,” Nader says, referring to collecting royalties on products NPS had licensed to Amgen. “I came up with a different idea that created what NPS is today.”

Or, at least, what it was until Dublin-based Shire Pharmaceuticals bought NPS for $5.3 billion in February. Having looked over the growing competitive osteoporosis market, Nader found that repurposing two osteoporosis drugs to fight rare diseases was the better way. Preos turned out to be a promising drug for hypoparathyroidism and Gattex (teduglutide) became a drug against a condition known as short bowel syndrome.

Nader took over as CEO in 2008, inheriting a company in transition from a huge biotech to a streamlined project management firm. Nader shut down R&D and outsourced everything that wasn’t project management. More importantly, he built a new culture, starting with those 17 employees — who now assumed the role of “thinkers and doers,” not just one or the other — and turning it into a 35-person operation. “Rather than fixing the old NPS, we created a new company from a blank slate,” he says.

The blank slate started with $145 million in capital, a stock price of $4 per share, and a handful of stalwart shareholders who believed in NPS’s new direction. Though “cash poor,” Nader says the company managed to raise $550 million from various funding methods that helped get Gattex FDA-approved in 2013. (It also bought back two drugs from a Japanese company for $50 million.) And Preos, which nearly shut the company down as an osteoporosis drug in 2006, got approved this year.

Actually, the Preos approval was the reason the deal with Shire happened so quickly. From first phone call to signing the papers was 90 days. Shire, Nader says, wanted to close before the FDA approved Preos.

The funny thing is, Nader had no designs on selling the company. He figured the new direction would build NPS into a powerhouse over the next decade or so. NPS, in fact, already had “feet on the ground” in 18 countries when Shire came calling, Nader says. But the Shire offer, which began great and honed into a $46-per-share deal, he says, was the right thing to do for the firm and the stockholders who had stuck with a company that was all but waiting for the priest to administer last rights just a few years ago.

It was also right for the patients, Nader says. Ten years ago, people who lived with rare diseases — like his nephew, who lives with an uncommon illness — would have to hear “You’ll just have to live with it for the rest of your life.” Now research into rare diseases is a huge percentage of the pharma market. More than a third of FDA approvals last year, in fact, were for rare-disease drugs. And with 6,000 rare diseases to fight, there’s a lot of room in the market.

The fact that rare diseases, by nature, affect few people (NPS was targeting as few as 5,000 at a time) has historically kept biotechs from the fringe. There were simply not enough people in need to warrant expensive research that had to jump through a ridiculous number of hoops in regulation worldwide.

But things are getting better. By 2020, rare disease research is projected to be worth $176 billion, and an easing of international regulatory hurdles is opening the global market for research and development.

On the downside, Nader says, is pricing and reimbursement. Europe, for example, still has country-by-country pricing. In some federal states, like Germany and Spain, there are state-by-state rules to follow. So where there is one open door, there is still a familiar locked window. And this is where the global biotech industry needs to focus its energies, Nader says, because these pricing fiefdoms are only keeping valuable drugs out of the hands of people who need them.

Having shown the value of drugs that few will need has been central to Nader’s most fulfilling professional episode, at the helm of NPS. “When you have such a small number of patients, you get to know them,” he says. “And what I mean by that is, you get to know them personally.”

These small enclaves of patients suffered without hope, he says. His own nephew keeps asking if anyone can help him, and Nader answers “not yet.” Which may sound bad, but actually is a long-jump better than “no, sorry,” like he used to say.

Giving people this hope, seeing the results of lives changed for the better, from where there once was no hope has brought more than one tear to Nader’s eye. And it has rewarded him for his decision to leave behind the life of a GP.

“As a physician, that’s been my greatest success,” he says. “It’s been a very fulfilling journey.”

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