The Antidote: Inside the World of New Pharma (26 page)

Bob Kauffman pressed hard to convince the FDA that in placebo-controlled midstage studies, Vertex could truncate the standard treatment time while also improving cure rates. Long-term safety concerns arose. Telaprevir was highly potent. The longer you took it, the higher the risk to your body, but if you went off it too soon, it could unleash viral resistance. Kauffman partly won over regulators with virology data and modeling. “In the end, the FDA agreed to a multiprong study that allowed us to study shorter duration with some patients,” he recalls. “We were really obsessed about twelve weeks at the time, and the FDA was very concerned. We believed in our modeling. But they did let us do a small number of patients with twelve weeks, and in Europe they allowed us to do more.”

The race to market was decidedly on, not just with Schering-Plough and Boehringer, but with dozens of other companies, including several
that, like Gilead, had started trials with nucs and other polymerase inhibitors that in preclinical assays looked as potent as telaprevir. “We were pushing very hard for an aggressive development program,” Kauffman says. “We knew we were in a competitive area, there were other people working in the field, and it was starting to heat up. We really wanted to both advance our program quickly but also really give patients something that was good at the end—not just higher SVR rates, but higher SVR rates with shorter-duration treatment. All of our advisors kept telling us that would be fabulous.

“We had a real impetus to stay in the lead. It was frustrating sometimes when it took a few months to negotiate a protocol with the regulatory agencies. We were pushing to get stuff done. But you have to see this from the regulators’ point of view. This was a new area, new field. Nobody had ever done this before. Nobody had even shown that you could get an SVR with a direct-acting antiviral until we did it. So they were somewhat skeptical, and concerned. They wanted things tied up well.”

Within two months of starting Phase II, while Kieffer prepared her slides for AASLD and Smith captivated Wall Street with tantalizing visions of a monotherapy for the leading cause of liver cancer and transplantation, a few patients at different study sites began developing severe rashes. Investigators reported them to Vertex and the FDA. Kauffman, keenly aware that dozens of drugs cause two forms of life-threatening skin toxicities in rare cases, and that a clear correlation can halt a drug from being approved, grew increasingly worried.

“Things had been swimming along: fabulous swoosh, great data, start Phase II. Then this,” he says. “There was one case, another one, and another one, and we all started looking at each other. You know, the first one, you just go, ‘What was that?’ After three of them, you go, ‘Oh. This could be telaprevir.’ After five of them, you’re pretty sure it really is the drug.”

During World War II, George Merck made sure that his drug company was central to the war effort. Pharmaceuticals were in low regard—deservedly, since very few of them did anything useful—and profiting from medical research was anathema; an idea whose time had not yet
come. Merck, approaching age fifty, chaired the government’s biological weapons program. He also helped drive—and was a prime beneficiary of—Washington’s efforts to marshal the nation’s research labs in defeating the fascist powers.

President Franklin Roosevelt’s chief science advisor, Vannevar Bush, chose Merck’s top consultant and scientific architect, Dr. Alfred Newton Richards, to chair the Committee on Medical Research—the biomedical equivalent of the Manhattan Project. On August 7, 1941, four months before Pearl Harbor, the CMR heard intelligence reports that the Germans had isolated the active substance from the cortex of the adrenal gland—cortisone—and were feeding it to their pilots to help them withstand the rigors of aerial combat. The committee made cortisone production its highest priority. That evening, after returning by train to Philadelphia, Richards received two visiting researchers from England, Howard Florey and Ernst Chain, who had experimented with a highly scarce mold extract—penicillin—to treat burns and infections, harvesting it in desperation from patients’ urine and reinjecting it until they ran out. By the end of the war, under Richards’s direction, Merck led other drugmakers and federal labs in making enough of the antibiotic to supply the nation.

Driven to expand his business and assimilate himself and his company at ever higher levels of American life, Merck built deftly on his wartime public service. Bush, forward-seeing architect of the nation’s postwar scientific order, joined the company’s board, soon becoming chairman. In the labs, chemists working under the fiery, protean head of research Max Tishler—Boger’s mentor—labored another decade to develop a synthesis to make cortisone commercially available. George Merck became a spokesman for the industry’s idealistic nature, his company exalted for its scientific leadership and social progressivism. In August 1952 he appeared on the cover of
Time
over the caption “Medicine is for people, not for profits.”

Boger had a similar trajectory in mind for himself and Vertex, but the War on Terror hadn’t produced comparable opportunities for drugmakers to pitch in. The only Axis was the name-only “axis of evil,” and the wars in Afghanistan and Iraq offered few opportunities for ambitious, public-spirited company builders to assume national leadership roles
against a major threat. Boger seized on an alternative route. Envisioning a leading position for Vertex locally, within the global health industry and with government regulators and payers, he set about building a highly visible web of outside responsibilities, connections, and interests. “Externalities,” he called them. To keep his employees up on what he was doing, he started to blog about his activities internally, for Vertex-only consumption.

Boger became the first biotech executive in nearly a decade to chair the Massachusetts High Technology Council, and he joined the board of the Biotechnology Industry Organization (BIO), the national biotech trade organization. He sat on the board of fellows at Harvard Medical School and became a major fund-raiser for the Greater Boston Food Bank’s capital campaign. Thinking globally but acting locally, he became a close advisor of Governor Deval Patrick, representing the state’s broad scientific, academic, and business interests, and advising Patrick on how to position Massachusetts for the future. “Unlike many of his peers—who feel that since they represent the future, politicians should come to them—he sees the value of involving himself deeply in Beacon Hill affairs,”
Boston
magazine noted, ranking him among the city’s power elite.

He also heartily adopted a few of the trappings of the role, the VIP/inside-the-ropes attention, access, and other entitlements that major CEOs enjoy. In January, a week after returning from the Morgan conference where he outlined Vertex’s plans for the trials of telaprevir and VX-770, he and his wife, Amy, traveled with the New England Patriots to the AFC championship football game against Indianapolis, sitting three rows behind the team’s cerebral head coach, Bill Belichick, during the flight. In March they flew to Mountain View, California, for the XPrize Foundation’s Radical Benefit for Humanity at Google’s sprawling headquarters, special guests of one of the foundation’s board members. With no structure taller than a few floors and “curiously conservative landscaping,” he observed in a ten-page, single-spaced blog post, the campus looked “like an unnaturally clean summer camp for geeks.”

He was seated at a table with Charles Lindbergh’s grandson Erik, who four years earlier had piloted a replica of his grandfather’s
Spirit of St. Louis
on the seventy-fifth anniversary of the first solo flight across the
Atlantic—a grueling physical and mental feat. Erik Lindbergh had been diagnosed in his early thirties with rapidly progressing rheumatoid arthritis and had been forced to stop flying before he started to take Enbrel, which halted the disease and restored his life, and for which he was so grateful that he’d become a celebrity spokesperson for the product. “And so, I casually asked him,” Boger wrote, “ ‘How important to you would be a once-a-day pill that did about the same thing?’ Unhesitatingly, he jumped forward. ‘That would be great. I hate Enbrel. I’d be curled up in a home without it, but I hate it. The injections: They burn. The new less-frequent injections burn a lot worse than the older ones. The infections. I hate it.’ We talked about Vertex’s decade-plus-long commitment to arthritis. Vertex has a new fan.”

The
Boger Blog
recounted in colorful detail the glittering events of the evening: the A-list sightings (Larry Page, Sergey Brin, Richard Branson, Jerry Brown, Arianna Huffington, and Robin Williams, who performed after dinner); the extravagant table setups and posh, all-organic cuisine by Google’s in-house chefs. It showcased Boger’s far-flung interests, erudition, cultural commentary, and own geek “cred”—unscrolling in a rush of asides. About a lackluster finale of “Hallelujah” by singer Rufus Wainwright, for instance, he wrote, “Get the Leonard Cohen version, on his
Various Positions
album. It’s chillingly superb recitative, a mature creation to Wainwright’s empty simulacrum. (The pop Jeff Buckley version, on the album
Grace,
is second derivative and even more self-indulgent than Wainwright’s.)”

During the silent auction, Boger hoped to win a zero-gravity flight with Stephen Hawking. A lifelong space travel enthusiast who views Hawking as the greatest physicist of our time, he was enthralled by the idea of flying weightless alongside Hawking in NASA’s “Vomit Comet”—a specially modified Boeing 707 with a padded cabin where, for twenty to thirty seconds, as the plane flew sweeping parabolas in an invisible, protected corridor above the Atlantic, they would float free. Hawking is as famous for his motor neuron disease, paralysis, electronically modified voice, specially equipped wheelchair, and expansive wit as he is for his theories. Boger confessed to feeling bereft when he at first learned that he’d lost out to another attendee who outbid him, a mix-up
that wasn’t resolved until the end of the night. He blogged, parenthetically: “
Star Trek
fans have already seen Dr. Hawking in space, playing himself (the only person ever to play himself in any
Star Trek
episode) in
Star Trek: The Next Generation
episode #252, ‘Descent—Part I,’ first aired 21 June 1993 (Stardate 46982.1). As every fan knows, Hawking, as himself, plays poker with the android Data, in a holodeck simulation including Albert Einstein and Isaac Newton. Actors played the latter two characters. Best episode ever.”

It would be hard to overstate Boger’s joy and fulfillment on a Tuesday in April, when he flew to Florida to meet up with two dozen other passengers to take the trip on “G-Force One.” Earlier in the month, McHutchison had given a late-breaker presentation in Barcelona, Spain, at the European Liver Meeting on the first shortened-duration telaprevir trial. The results suggested that twelve weeks of telaprevir-based therapy with peg-riba enabled some patients to clear the virus. The company also now had a corrector for CFTR—VX-809—on track for clinical development, the key ingredient in the drug cocktail that could be the elixir of life for the great majority of those with cystic fibrosis. Boger was soaring. “Jet Blue to Orlando,” he wrote. “This was no Mickey Mouse tour. I didn’t go to Tomorrow Land. Felt a little Goofy, but that was understandable. I was headed for weightlessness. Weightless. With Steven Hawking. Weightless.”

Hawking was accompanied by four handlers and two physicians who laid him on the padded floor among the other fliers, guided and monitored him through eight plunges, and concluded that he was in “tremendous condition”—heart rate, blood pressure, oxygen levels all normal and perfect. “Space, here I come,” Hawking pronounced ecstatically after the flight. Boger felt much the same way.

“Zero gravity is unlike anything you have experienced,” he blogged. “It’s not at all like cresting a hill in a fast car. It’s not at all like dropping rapidly in an elevator shaft. It’s not at all like jumping out of a plane. It’s not at all like scuba diving in blue open water. It is not at all like any of these. Imagine a dimmer switch for a room light. You glide it slowly down, and the light slowly dims. Raise it back, and the light comes back on, slowly, evenly. There are no jumps, no bumps, no stomach shifting,
no wind, no resistance. The light just goes down, and then it comes back up. That’s what zero gravity feels like.”

It was in this frame of mind that Boger consolidated his external positioning at the May BIO convention in Boston. At the annual membership meeting, he was elected chairman of the board—in effect, the industry’s face in Washington. At the same time, his efforts on Beacon Hill bore fruit. Governor Patrick—with Boger beaming at his side, rolling his right thumb and forefinger together like a safecracker—touted to thousands of conventioneers recent gains in the Massachusetts economy that helped make the state a world leader in biomedicine. With one in seven jobs in the health care sector, and with more than $2 billion annually flooding into its research hospitals and university labs from the NIH, the state was proof of the power of government-subsidized science and assembled intelligence as a platform for prosperity.