Moderna's defense, biosimilar battles and other updates
Since those headlines, in the scores of company presentations at San Francisco's Westin St. Francis, drugmakers vied for investor attention with forecasts and updates for the year ahead.
Here's a snapshot of highlights from Monday and Tuesday.
Like artists, unicorns can be misunderstood, too.
Moderna, the Cambridge, Massachusetts-based "unicorn" that holds the distinction of biotech's largest-ever IPO, arrived in San Francisco on the heels of a stock slide that erased roughly $2 billion of the company's lofty valuation.
The biotech's promise rests on its mRNA technology, which it claims can be used to spur the body to produce its own therapeutic proteins. If all goes to plan, Moderna contends mRNA affords it the opportunity for cheaper drug development with higher probabilities of success.
Those investors who remain skeptical, its executives suggest, might just be looking at the company the wrong way.
"We're asking investors to undertake a different source of pattern recognition as it relates to developing a platform technology across a wide array of applications," said Lorence Kim, Moderna's chief financial officer, in a breakout session at the conference Tuesday.
One concern stems from Moderna targeting vaccines first, an area typically seen as less commercially compelling.
Stéphane Bancel, Moderna's CEO, described the company's approach as designed to mitigate biological risk. Working on projects likely to succeed could validate mRNA technology for riskier, but more lucrative targets.
"This has never been about one drug getting to market, but about a lot of medicines getting to patients," Bancel told investors Tuesday.
Moderna now has 21 candidates, with 11 in early clinical testing. A pipeline update Tuesday detailing plans for several immuno-oncology and rare disease programs appeared to buoy investor enthusiasm, sending Moderna shares higher by 4%.
At JPM, Bancel also made the case that Moderna would aim high even as it manages risk.
"We will not be scared to run high-bar trials, like pharma does most of the time," the CEO said. One example? A Phase 2 study with Merck & Co. that will pit Moderna's personalized cancer vaccine plus Merck's Keytruda (pembrolizumab) against Keytruda alone.
If the vaccine doesn't improve complete responses and survival versus Keytruda, Bancel says, "we don't have a product."
While 16 biosimilars are approved in the U.S., only seven have launched as patent roadblocks and litigation have held up the rest.
The latest of the copycat biologics to reach patients is Coherus BioSciences' Udenyca (pegfilgrastim-cbqv), which launched on Jan. 3 with a list price 33% less than that of the biologic drug it mimics, Neulasta (pegfilgrastim).
With Neulasta biosimilars from Mylan and now Coherus available, Amgen could find its second best-selling drug under pressure this year.
Coherus on Tuesday told investors it has sufficient inventory and supply of Udenyca for a broad launch, making the threat more immediate for Amgen.
Murdo Gordon, the biotech's new commercial head, noted in a Tuesday session that Amgen has modeled a wide range of outcomes for its Neulasta business given encroaching biosimilar competition. Onpro, a delivery device that's more convenient than the pre-filled syringe offerings from Mylan and Coherus, could help Amgen defend its business.
Amgen, of course, is playing on the biosimilar side of the market as well, with its own pipeline of copies of other companies' blockbusters.
"This time next year I think we will be talking about biosimilars, and the fact that biosimilars will be a source of revenue growth and earnings growth for Amgen," said the biotech's CEO Robert Bradway in a Tuesday presentation to investors.
Novartis, another pharma invested in biosimilar development, appears even more bullish.
"Our strategy is to pivot hard to biosimilars and hard-to-make molecules," said Novartis CEO Vas Narasimhan in a Monday presentation, referring to Novartis' Sandoz business in particular.
Investors hadn't given Alnylam Pharmaceuticals much time to celebrate last year's approval of its RNA-targeting therapy Onpattro (patisiran), pushing shares in the biotech down in November after early sales of the drug fell short of expectations.
Fourth quarter numbers, pre-announced Monday, have gone over much better. Alnylam's stock has risen by 13% this week over last Friday's close, bringing the price per share back to about the level it traded at before release of third quarter results.
Alnylam estimates sales of its therapy, which carries a typical annual list price of $450,000 per patient, totaled between $11 million and $12 million in the three months ending Dec. 31.
The biotech also noted value-based agreements with Humana, Harvard Pilgrim Health Care and a third, unspecified insurer. Another 15 such contracts are under negotiation with other commercial payers.
When Bristol-Myers Squibb bought Celgene last week, the pharma highlighted six experimental drugs it hopes to launch within the next two years.
One, a treatment for myelofibrosis called fedratinib, had been slated for a submission to U.S. regulators by the end of 2018.
No mention of a filed New Drug Application during a conference call discussing the buyout, however, raised the possibility Celgene got caught on the wrong side of a partial government shutdown that's affected the FDA's ability to review new applications.
Celgene CEO Mark Alles cleared up the confusion Monday, indicating the biotech was able to successfully file fedratinib with the agency. The companies expect to secure an approval by year's end.
Celgene still has two other filings to check off before the deal is expected to close, though. Submissions are scheduled over the next six months for ozanimod in multiple sclerosis and luspatercept in myelodysplastic syndromes and beta-thalassemia.
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