Essay

January 26 — February 1, 2015

Briefs: Tax refunds at Walmart; the JCPenney catalog is back 25

Facebook flies above the censors 22

YouTube’s wardrobe- malfunction-proof halftime show 21

Charlie Rose talks to fashion designer Stella McCartney 23

Target’s new boss calls it quits in Canada 24

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▶▶ Social media raises the stakes in the race for drug approvals

▶▶ If Josh dies, “how will Moch live w/himself?”

In early 2014, 7-year-old Josh Hardy was dying. He’d been battling kidney cancer since he was a baby and sur- vived 10 intensive chemotherapy treat- ments, which severely weakened his immune system. Soon after under- going a bone marrow stem cell trans- plant last January, he developed a life-threatening respiratory virus.

Chimerix, a small biotechnology company in Durham, N.C., had an experimental, unapproved drug in development that might help, Josh’s doctor at St. Jude Children’s Research Hospital told the boy’s family. They twice asked Chimerix to provide Josh the drug and were refused both times. “Please help us save our son,” his mother, Aimee, wrote on her Facebook page on March 6. “If anyone with influence can help us convince the Chimerix Inc to release the drug for compassionate care for our son, we

would be forever grateful.” Within several days, a

#SaveJosh hashtag was trending on Twitter. A Facebook page set up for Josh’s

cause registered 27,000 “likes” in one week. Social media posts supporting the family’s effort to get the drug were directed at Chimerix and its chief executive officer, Kenneth Moch, who also received thousands of e-mails and phone calls. If Josh dies, asked one tweet, “how will Moch live w/himself ?” Another said that Moch’s decision to withhold the drug carried “a HEAVY Karmic price!” Yet another called Chimerix executives “cowards …  Ken Moch, I can only hope this doesn’t happen to your child.” And Moch was the target of death threats that the FBI deemed credible, so he and his wife moved out of their home for several days.

Under intense pressure, the company worked out a plan with the Food and Drug Administration to add a clinical trial for the drug, brincidofovir, and to enroll Josh as its first patient. “It was relatively an easier campaign because we had a very tangible goal,” says William Burns, Josh’s uncle, who first used the hashtag and set up the “Save Josh” Facebook page. “We had an immediate need, not like, ‘Give more money to cure cancer 10 years from now.’ ”

The CEO Who

Saved a Life and

Lost His Job Former Chimerix CEO Moch

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32,478

Last Resort Requests granted by the FDA for compassionate use of a drug, in the 12 months ended Sept. 30

While the question of whether to give Josh the drug seemed like a no- brainer to his family and support- ers, it presented Chimerix with tricky issues to sort out. Compassionate use— formally known as expanded access— allows experimental drugs that haven’t been approved by the FDA to be given to patients in l ife-or-death situations who have no other treatment options. While the FDA is required to sign off on compassionate-use cases—it usually approves about 1,000 a year—the deci- sion about whether to give out an unap- proved drug rests with drugmakers. A patient’s physician typically requests a drug from the company developing it. The company weighs several factors, including the most potentially tragic— that the drug might hasten or cause the death of a patient. Such a death can complicate a company’s efforts to secure FDA approval and to ultimately make the drug widely available.

For a small biotech like Chimerix, which had no product on the market and no source of revenue, handing out experimental drugs, usually for free, can be especially costly. Compassionate use also can delay the FDA approval process, because drugs and other resources are diverted to patients who aren’t enrolled in formal trials, which collect data neces- sary for gaining regulatory approval.

“Everyone asks this question,” Moch says. “ ‘If it were my child, would I do what the Hardys did?’ Absolutely, yes. ” But that wasn’t his role, he says. “As the CEO, I have to think not just about the individual, but the many.”

On April 9, three weeks after Josh got into the brincidofovir trial, Moch stepped down as CEO. The company says he resigned. Moch says he was replaced and can’t comment in detail about his departure other than to say he would have “loved to continue to be CEO, but the board felt otherwise.” Chimerix declined to comment beyond its press release announcing Moch’s departure and his replacement. “We are now entering a critical stage of the clin- ical development of brincidofovir,” the release said. “Our goal is to complete the

requirements for regulatory approval of brincidofovir as rapidly as possible.”

Moch, 60, joined Chimerix as CEO in 2010. He studied biochemistry as an undergraduate at Princeton and had worked in the life sciences industry since getting his MBA from Stanford in 1980. In 1982 he co-founded Liposome, a devel- oper of treatments for cancer, including breast cancer; drugmaker Elan acquired Liposome in 2000. Moch held various executive-level positions with medical companies before joining Chimerix.

Last year, when Chimerix was approached for help by the Hardy family, the company had 55 employees, no approved drugs, and was relying on funds from investors. It was in the final phase of testing brincidofovir for FDA approval to market the drug to prevent cytomegalovirus in bone marrow stem cell transplant patients. Chimerix also had tested brincidofovir as a preven- tive therapy for adenovirus, the virus afflicting Josh, in an earlier stage of the trial. At the end of March 2014, the company had an accumulated deficit of $173 million since its founding in 2000, according to regulatory filings.

Chimerix had previously given brincidofovir to 430 patients for com- passionate use in a program funded in part by the government. When funding ran out in 2012, Chimerix closed the program to focus on getting the drug through the FDA, Moch says. Allocating any available doses to Josh could have slowed that process, he says. “It’s horri- bly conflicting,” he says of the dilemma he faced in March. “You go into this business to saves lives.”

Some patient advocates insist that companies have a moral obligation to make their drugs available to desper- ately ill patients. “Here’s a kid where it’s life or death, hanging on to Ken Moch’s

decision,” says Nancy Goodman, execu- tive director of Kids v Cancer, a nonprofit advocacy group for children with cancer which helped publicize the Hardy fami- ly’s campaign. “It’s profoundly unethical for him not to give the drug.”

Kevin Donovan, director of the Center for Clinical Bioethics at the Georgetown University Medical Center, says the conflict between companies and patients lies in a clash of respon- sibilities. “The company’s obligation is the greatest good for the greatest number,” he says. “If it’s your loved one, if it’s you, if you’re a patient, your moral obligation is to the welfare of that individual.” Both approaches are legitimate, he says.

These complexities get lost in a social media maelstrom, says Darshak Sanghavi, an associate professor at the University of Massachusetts Medical School who’s conducted research on compassionate-use issues. “I think that whenever you have 140 characters to describe a complicated medical deci- sion, it’s going to be oversimplified— ‘there’s a dying child, why won’t the drug company give the drug?’ ” Sanghavi says. “It’s so easily amplified, and any nuance, even if it was present early on, rapidly gets rubbed out,” he says.

James Greenwood, CEO of the Biotechnology Industry Organization (BIO), says social media’s role in the Hardy case unnerved the indus- try. “When you add social media and start tweeting all over the place, then reason flies out of the window,” says Greenwood. “Social media creates opinion storms, we all know that.”

Moch stresses that in the end, Josh’s brincidofovir treatment was not compassionate use. As part of a new clinical trial, his response to the drug was included in data Chimerix was gathering in its push for FDA approval. The trial tested brincidofovir as a treatment for adenovirus, not just a preventive therapy. Barring any delays, the FDA could approve the drug by the end of 2016.

Moch’s point is one that matters deeply to small biotech companies trying to get their first drug to market. One example, says BIO’s Greenwood, is CytRx, a biotech whose trials for its experimental cancer drug aldoxorubi- cin were temporarily placed on hold on Nov. 18 after the death of a patient who received the medication under compassionate use. The potential for the FDA to stop a trial because of a

tweets worldwide using #savejosh from Jan. 16, 2014, to Jan. 16, 2015

2k

1k

0

*12 MONTHS ENDED OCT. 12 DATA: FOOD AND DRUG ADMINISTRATION

57% Urgent requests on

behalf of patients who could die within hours

2010* 2011* 2012 2013 2014

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Kravitz. Yet that star power isn’t deter- ring YouTube from offering its own halftime show online, hosted by Harley Morenstein, the ringleader of food stunt group Epic Meal Time. In 2010, Morenstein and his pals made history when they wowed Web viewers with a 5,210 calorie pizza made of fast-food items (boasting 286 grams of fat). In addition to music, he prom- ises fake Super Bowl ads and stunts such as online celebrities jumping into a vat of cheese.

The show, featuring some of YouTube’s biggest stars, is part of a broader initiative to promote ads on the Google- owned site—as well as to exploit

The Other Super Bowl Contest YouTube’s quirky, alternative halftime show will challenge NBC’s pop star-studded extravaganza

Kravitz, a four-time Grammy awardee, has also appeared

in two Hunger Games films

Wong’s main channel on

YouTube has more than 7.4 million

subscribers

Turner’s offbeat antics have helped

draw 3.2 billion video views to his

three channels

Entertainers Katy Perry Lenny Kravitz

Typical Ad Price $4.5m 30-second spot

Demographic Anyone breathing

Viewers of 2014 halftime show 115.3m

Viewers who tuned in to 2014 halftime show but skipped the game 3.8m

Entertainers Harley Morenstein Freddie Wong Toby Turner Kurt Hugo Schneider Smosh Games FouseyTUBE Matt Santoro MysteryGuitarMan

Typical ad price $9.11 per 1,000 views

Demographic 18-34 year olds

Subscribers to the acts in YouTube’s show 60m

patient death or some adverse event outside the scope of a trial, for which patients are carefully screened, could further deter biotechs from giving out their drugs, Greenwood says.

BIO is working with its member companies, particularly small ones, to prepare for campaigns like the one the Hardy family waged against Chimerix and Moch. The group sug- gests that members make it clear how a compassionate-use program will operate and develop a crisis management plan.

About a week after receiving brincidofovir, Josh’s mother reported on her blog that his condition had improved dramatically; he was dis- charged from the hospital in early April. Moch says he’s in active discus- sions to take a new CEO job. He’s spent the past nine months meeting regu- larly with chief executives of other small companies about compassion- ate use issues and consulting for a working group of ethicists at New York University’s Langone Medical Center that’s studying the challenges of com- passionate use.

“For the biotech community, what happened to Moch was a shock,” says Greenwood. “Within the BIO board and in our community, Ken Moch is a very loved person. People were hurt to see what happened to him, and they worried that this could be me.” �Caroline Chen The bottom line Biotechs have watched closely the experience of Chimerix, the target of a social media campaign.

NBC

YouTube

Media

YouTube Wants to Be a Super Bowl Winner, Too

▶ The site’s alternative halftime show is a bid to win advertisers’ attention

▶ Figuring “what was Freddie Wong’s draw compared to Katy Perry”

The Super Bowl halftime show has become a showcase for some of music’s biggest stars in recent years, from Paul McCartney and the Rolling Stones to Beyoncé, Prince, and Madonna. True to form, this year’s broadcast, on Feb. 1 on Comcast’s NBC, will feature singer Katy Perry, one of pop music’s biggest acts, with an assist by rocker Lenny

“YouTube is the place where people go to consume advertisements willingly. It’s some capitalist dystopian nightmare.” ——Freddie Wong

America’s cult of loving and hating the expensive ads that air during the Super Bowl, the year’s most-watched television event. Last year’s game, broadcast on Fox, was seen by more than 111.5 million viewers, a record. The YouTube specta- cle will stream live on its website during the game. YouTube has long competed

for video advertising dollars with Facebook and Twitter, and it will use the halftime show and the ads around it to differenti- ate itself and demonstrate its value to top brands.

YouTube says the growing profile of its stars like Freddie Wong, whose main channel has more than 7.4 million

Cooking stunt auteur Morenstein

will host YouTube’s

halftime show

Perry was No. 1 on Billboard’s Hot 100

list of artists in 2014 21

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