After three days drawing thousands of biopharma executives and investors to the Westin St. Francis, the J.P. Morgan Healthcare Conference came to a close Thursday with the last 60 biotechs presenting to a noticeably thinner crowd.
One could be forgiven for thinking this year's meeting was light on major news.
Investors looking for a flurry of deals to kick off the year, for example, could have headed home Sunday night. Outside of Celgene Corp.'s deal for Impact Biomedicines and Novo Nordisk A/S's spurned $3 billion bid for Ablynx NV, little else was publicly announced at a meeting pegged as a tone-setting event for biotech.
To be sure, deals in the offing were likely discussed in the hotels dotted around Union Square and the Westin St. Francis. With the tax overhaul slashing the corporate rate and giving a discount to bring offshore cash home in law, confidence appears high that M&A will continue to emerge throughout 2018.
This year's meeting also avoided 2017's worries of political action from President Donald Trump. A year after famously saying pharma was "getting away with murder," the commander-in-chief stayed silent on the industry.
Winners and losers
#JPM18 Winners: China Biotech, Tax Reform, IL-2, Sanofi Castoffs, Cell/Gene Tx, Llamas, Late night karaoke.#JPM18 Losers: Glaxo Castoffs, Press Release Fact Checkers, Channel Stuffers, Clueless $CELG leadership, pessimists, umbrellas.— Brad Loncar (@bradloncar) January 11, 2018
Brad Loncar, a biotech investor and founder of a cancer immmunotherapy ETF, summed up the conference in one short tweet. For those of our readers who are visual thinkers, BioPharma Dive mapped out some of the largest stock moves for biotech companies over the past four days.
Brighter days ahead for neuroscience
News on neuroscience this week may obscure a larger trend of optimism.
Pfizer Inc. titled its presentation to investors "A New Era of R&D Productivity." That optimism apparently didn't apply to neuroscience drug development, as the pharma giant announced in the run up to the conference it would halt efforts to find new drugs for Alzheimer's and Parkinson's.
While Pfizer has not been well-known for its presence in the space, the decision speaks to the many challenges the industry faces in finding new treatments for neurological disease.
Elsewhere at the J.P. Morgan meeting, Axovant Sciences Ltd. — fresh off a high-profile failure for its lead treatment in Alzheimer's — announced the drug didn't work in dementia, either.
Such setbacks add to a long list of failure, particularly in Alzheimer's. Yet such gloom might disguise some green shoots. New treatments of spinal muscular atrophy, multiple sclerosis and several rare diseases point toward progress.
Kristina Burow, a managing partner at ARCH Ventures, contends neuroscience remains an area of opportunity for the industry, due in part to an improved scientific understanding of the brain.
Borrowing a page from oncology could help, too, by no longer viewing neurological diseases as a single monolith, Burow said. "We're getting better at breaking down these definitions and making these more biologically driven, or symptomatically driven."
The dividends from this knowledge could take years to emerge, but over time Burow predicts a resurgence of interest in neuro-behavioral health.
Some companies, such as Biogen Inc., have already doubled down.
"The opportunity is significant. The space is complex — high risk but also high reward for those who succeed," said Biogen CEO Michel Vounatsos on Monday. "The objective of the organization moving forward [is] to be the leader in neuroscience."
Alkermes swats away allegations
One biotech is trying to clear its name after what it calls "vicious attacks" in major media last summer.
The criticism blasted Alkermes as "a bad company and a bad medicine and that nobody should use [Vivitrol],” CEO Richard Pops told BioPharma Dive at the J.P. Morgan Healthcare Conference in San Francisco this week.
“We’ve just been swatting that down,” he added.
Pops said that several articles last year led to questions from Washington.
Specifically, Senator Kamala Harris launched an investigation into the company, suggesting Alkermes is trying to artificially boost sales of Vivitrol. The drug has been on the market for years, first as an alcohol dependence treatment and later as a treatment for opioid dependence. It struggled to produce any sales for years, but the recent opioid epidemic has changed that. It is now expected to bring in $265-275 million in 2017.
According to the California Democrat, Alkermes has been aggressively marketing the opioid dependence treatment to “people in the criminal justice system, often convincing judges and corrections officials to offer Vivitrol to inmates and parolees rather than treatment drugs with more proven efficacy.”
Pops insists the claims are false and that there are studies comparing Vivitrol to opioid replacement therapies like suboxone that show the Alkermes drug works just as well.
“It is impeding the integrity of us and the FDA and all the systems around the country that have been using Vivitrol around the country.”
Satisfying both regulators and payers
Increasingly, it's becoming important for biotechs and pharmas to go beyond what they need to do to satisfy regulatory requirements, but also consider what payers are looking for when designing clinical trials.
Getting a drug approved is not the finish line; Indeed, gaining reimbursement and access to patients can often be harder.
"More and more today we have to go beyond just regulatory approval," said Nawal Ouzren, CEO of Sensorion, at a panel hosted by BioPharma Dive during the J.P. Morgan Healthcare Conference. "Is the product going to be reimbursed and that's at least a price you consider acceptable," are major challenges.
At the helm of a Paris-based biotech, Ouzren is no stranger to dealing with regulations making such demands, especially the fragmented payer system in the EU. Although she noted that it's not just Europe that pricing is a problem anymore.
"Even in the U.S. I think it's becoming a hot topic to really be able to demonstrate you can bring value from the patients and justify the price," she added.
Orchard Therapeutics CEO Mark Rothera concurred, adding, "I think we're seeing more and more there's a kind of connection between the regulators and the HTA [health technology assessment] bodies that allows you to even have joint discussions about what is required, not just for the approval but in fact to get reimbursement."
Samumed's CEO Osman Kibar noted that the discussion often revolves around risk to the stakeholder.
"Between the regulators and the payers, they both try to minimize risk but they do define risk differently. In the case of the FDA, when they talk about risk, we're talking about medical risk, safety, side effects, patient, risk to the patients," he said.
"Whereas in the case of payers, they have their tables, they have to follow all the financials, obviously. So their risk is more about minimizing uncertainty."
So whether the drug is going to work in what percent of the population of the patients and whether you can predict that with biomarkers or otherwise, that's the kind of products that they love. Because that helps them minimize their risk in terms of future reimbursement and whether they are getting what they are paying for in the form of drugs," Kibar said.