There's a great future in biotech: Scenes from BIO 2015

PHILADELPHIA – Kicking off the last panel of this year's BIO International Convention, radio and television host David Brancaccio referenced the scene from The Graduate, when a family friend tells Dustin Hoffman's character, "There's a great future in plastics." Brancaccio asked a group of panelists during the Scientific American Worldview session what word or phrase signifies to each of them a great future in today's world.

"Wellness is the next plastics," answered Leroy Hood, president of the Institute for Systems Biology, who earlier in the day described his plans for P4 medicine and presented data from a pilot study involving 107 volunteers. "On the one hand, it's about optimizing human potential," he said of wellness. "On the other hand, we have to start with wellness to understand disease."

For Karen Nelson, president of the J. Craig Venter Institute, it's gut microbiology. "There's still an untapped world out there to be looked at," she said. Her work was part of the first paper published on the microbiome nearly a decade ago. Since then, "a whole field has taken off," including the formation of several companies. "For me, it's been fascinating."

It came as no surprise that Martin Naley, founder and CEO of a company called Cure Forward, which describes itself as a "patient activation" company, offered that phrase as the next "plastics." Patient activation, Naley explained, involves the "notion of getting activated by your own data," and allowing patients to gain information that can lead to insights that shape options for individual care.

It's an interesting idea, particularly against the backdrop of the Precision Medicine Initiative and other patient-centric and genomics-based approaches, and one that Apple Tree Partners certainly liked. The investment firm has backed Cure Forward in a $15 million series A announced just this week.

ROYALTY REIGNS

Triangle Insights Group partner Ben Bonifant, described as a guru on royalty rates, shared his observations in which pre-investigational new drug application royalty rates averaged around 5.3 percent for academic and small biotech deals. Those rates rose to double digits for deals done past proof-of-concept, and infectious disease assets tended to have the highest royalty levels at the early stage.

"If you are expecting great value in your asset, you want to negotiate toward the tiered royalty," he said during "Trends in Early Therapeutic Research and Funding – Project Initiation, Patent Filing, Licensing and Venture Capital."

GALAPAGOS: A SUCCESS STORY

Fee-for-service strategies and alliances helped to fund the early years of Mechelen, Belgium-based Galapagos NV, said its senior vice president of corporate development, Andre Hoekema, at Thursday's "How to Grow a Sustainable Biotech Company? Three Success Stories."

Galapagos used equity to build a preclinical pipeline and acquired chemistry screening libraries. A partnership with Glaxosmithkline plc also played a role in the company's advancement, as did a successful $275 million U.S. IPO and private placement this year, timed with the release of positive phase II JAK1 inhibitor filgotinib data in rheumatoid arthritis. (See BioWorld Today, April 16, 2015, and April 29, 2015.)

It hasn't all been smooth sailing, though. Hoekema described some headaches: pharma partners losing interest in therapeutic areas; interested executives who later leave the company; and partners deciding not to pay milestones despite a contractual obligation. "It is so frustrating because as a biotech there's nothing you can do," Hoekema said, later adding, "Make sure you have a mechanism to get that program you worked so hard on back if your partner chooses to put it on the shelf."

GERMLINES AND AN ETHICAL DILEMMA

One of the more troubling comments emerged from a precision medicine panel Wednesday. Participants reported that about 7 percent of patients who have somatic genetic alterations examined also show germline alterations in genes with clinical implications. And researchers are faced with an ethical dilemma of whether to report the findings to the patients.

"We're really struggling with this right now," said Jeff Boyd, senior vice president of molecular medicine at the Fox Chase Cancer Center in Philadelphia. "If we are going to report these findings to patients that requires an entirely new infrastructure that does not currently exist."

DOING IT FRAMINGHAM-STYLE

During a spirited panel session Tuesday debating the link between microbiota and human health, speakers agreed that finding ways of figuring out that link is both critical and exceptionally daunting. It would take, suggested Peter DiLaura, president and CEO of Second Genome Inc., the ability to look at how humans, who are "essentially sterile in utero," get colonized from birth, particularly in those early years of development.

"I suspect that it's a very evolutionarily attuned process, the way it all comes together," he said. "I don't think the field has a good handle on it now."

When asked what tool he would put on the top of his wish list for investigating the microbiome, DiLaura responded without hesitation: "An exceptionally large cohort of newborns." To be able to "follow them longitudinally – when they got an infection, when they got antibiotics, when they took a vacation" – would help provide a better picture, he added.

So, basically a microbiota version of the Framingham project, said John Rex, head of infection and global medicines development at Astrazeneca plc, referring to the long-term cardiovascular study on residents in Framingham, Mass., that began in 1948. Rex acknowledged that a "Framingham microbiota project" would be an interesting idea, though he wondered aloud who would apply for the grant for such a project.

At that, an audience member quipped, "Somebody very young!"

A LITTLE PRICING PERSPECTIVE

Mention the drug pricing debate and Gilead Sciences Inc.'s hepatitis C virus (HCV) pill Sovaldi (sofosbuvir) – and its $1,000-per-pill headlines – usually come to mind. Indeed, Sovaldi, which costs about $84,000 per treatment period, and HCV drug Harvoni (sofosbuvir/ledipasvir), which runs about $94,500 for a 12-week course, have become the go-to examples for those criticizing the rising cost of drugs.

But focusing on a single therapy "denies the impact these medications can have," noted Christi Shaw, president of Novartis Pharmaceuticals Corp., who moderated a Tuesday panel on patient access to new medicines.

She urged attendees to keep a little perspective, pointing to data showing that prescription medications have "consistently accounted for 10 cents on every health care dollar spent" for more than 50 years; that percentage is expected to stay constant.

Most of the costs are still coming from hospital care and physician/clinical services, and those are expenses that effective drugs can help curtail. In HCV, for example, shorter and more effective course of treatment can cut down on hospital visits. But, as Alkermes plc's CEO Richard Pops noted, improving patient compliance also could help slash those costs.

Dublin-based Alkermes is working on versions of antipsychotic drugs that could be more convenient for schizophrenia patients, reducing relapses, which often result from noncompliance. "People don't think of [schizophrenia] as a progressive neurodegenerative disease but it is," Pops said, with noncompliant relapses frequently resulting in hospitalization.

NO PRIMACY FOR PRIVACY?

That same session also raised issues of paying for efficacy, an outcome that could have greater assurance with the accumulation of patient data. Building up databases with patients' disease and genomic information is the latest push in precision medicine – the UK's 100,000 Genomes Project and the 100K Wellness Project are two examples – and the industry continues to move full speed ahead into the age of digital medicine, where health information is recorded, tracked and, sometimes, shared.

So HIPAA and patient privacy laws might be on their way to becoming passé.

In some ways, said bioethicist Arthur Caplan, of New York University Langone Medical Center, they already are. "You talk to young people, and they say, 'What privacy?'"

"We're spending a fair amount of time and money establishing rules for privacy that I'm not sure people care about," he said. To make an evidence-based payment system work would require maintenance and surveillance, suggesting "we've got to back off privacy."

And giving up privacy would have a positive tradeoff, Caplan suggested. "That could be an incentive, too," he said. "Let us watch you more closely" and patients get access to drugs more readily.

"I think it's something worth looking at," he said.

UPDATES FROM THE NIH

The NIH has lost about 23 percent of its purchasing power over the last 12 years, said NIH director Francis Collins during Wednesday's keynote luncheon. "We're leaving about half the good science on the table, which is very frustrating."

He discussed the potential of incorporating a liquid biopsy in precision medicine, in which a test to determine whether a fetus has normal chromosomes can actual reveal early cancer in the mother. "This is a very attractive and powerful way to think about how we might do surveillance of people who appear to be healthy."

AGONY OF THE FEET

If you were suffereing foot fatigue by the third day of BIO, there was a reason for that. Since BIO was last held in Philadelphia, the capacity of the Pennsylvania Convention Center was doubled, according to Mayor Michael Nutter, who spoke at the Tuesday Keynote luncheon. The growth, he said, was in part to accommodate the growing BIO meeting.

Nutter also made what he called a "shameless" plea for BIO to return to his city for its 2019 convention.

ALL THAT JAZZ

Grammy-award winning trumpeter Wynton Marsalis performed and was interviewed at Wednesday evening's keynote, before a multitude of people working in life sciences, saying: "In music, the two closest things together spatially are the furthest apart harmonically," and, "If you can't play blues, you can't play jazz." The evening ended with a two-hour reception at Reading Terminal Market, adjacent to the convention center, and the 2015 meeting concluded Thursday.

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