JPM24, Day 1: Even after M&A deals, BMS CEO focuses on pipeline; Amgen spotlights obesity prospects

As the J.P. Morgan Healthcare Conference kicked off in San Francisco, EY released its annual report on the industry's dealmaking firepower. Plus, Regeneron, Moderna, bluebird and many other companies updated investors on their businesses heading into 2024. 

We'll update this page throughout the day; be sure to check back regularly.

UPDATED: Monday, Jan. 8 at 7:20 p.m. ET

Regeneron is happy with the launch of its high-dose version of Eylea. In Q4, its first full quarter on the market, sales reached $123 million, the company revealed in an SEC filing on Monday. Later in the day, at the J.P. Morgan Healthcare Conference, CEO Len Schleifer called the figure “quite a good achievement by all measures compared to other recent launches in the space.” Schleifer, of course, was referring to Roche’s Vabysmo, which by comparison pulled in $112 million in its first full quarter on the market, Q2 of 2022, and was assured of blockbuster sales in 2023. Vabysmo’s success is attributed to its longer duration of effectiveness, allowing patients to need injections every three to four months versus Eylea, which is dosed every two months. The HD version of Eylea is Regeneron and Bayer’s answer to Vabysmo, showing durability in trials that is superior to that of Vabysmo for some patients. The successful launch of Eylea HD came despite some adversity. “There’s still some amount of prescriber hesitancy related to the fact that we don’t have a permanent J-code,” Schleifer said, referring to the identifying system that allows pharmacies, hospitals and physicians to bill for medications. Once Eylea HD receives its J-code, it will facilitate better access to the drug, said Regeneron’s commercial chief Marion McCourt, who added that patient feedback has been “very positive.”

UPDATED: Monday, Jan. 8 at 4:18 p.m. ET

After last year’s $7.3 billion buyout of Reata, Biogen CEO Chris Viehbacher touted a “tremendous” launch of Friedreich's ataxia therapy Skyclarys. The drug is “outperforming” other rare-disease launch analogues, including Biogen’s own spinal muscular atrophy launch Spinraza, the CEO said. Because of a prior manufacturing-related delay for the drug, there had been a buildup of patients eager to start on therapy, Viehbacher explained. Now with approvals in the U.S. and Europe, Biogen is working to make the most of a medicine it inherited with the Reata buyout. He noted that the company still needs to conduct a pediatric study of Skyclarys because many patients are diagnosed with Friedreich's ataxia before the age of 16. 

As Johnson & Johnson moves forward without its consumer healthcare group, the company is leaning on several new launches to drive growth. In multiple myeloma, J&J figures Tecvayli, Talvey and Carvkyti can handily beat analyst expectations by 2027, CEO Joaquin Duato said Monday at the J.P. Morgan Healthcare Conference. Between new launches and the blockbuster Darzalex, J&J’s multiple myeloma franchise is heading toward potential annual sales of $25 billion, according to the CEO. In addition, depression nasal spray Spravato is poised to beat out analyst estimates, according to the company’s calculations. All this comes as J&J steams toward $57 billion in pharmaceutical sales by 2025. Story 

UPDATED: Monday, Jan. 8 at 3:53 p.m. ET

For Gilead Sciences, 2024 is set to be a “very catalyst-rich” year, according to CEO Daniel O’Day. With up to two dozen clinical trial readouts and no patent cliffs on the horizon, the company is moving into a “new era," he explained. Four years ago, the drugmaker pulled in some $500 million from its oncology offerings. Now armed with antibody-drug conjugate Trodelvy and cell therapies Yescarta and Tecartus, the company’s oncology portfolio is generating around $3 billion annually. As O’Day sees it, that performance falls in line with Gilead’s previously communicated goal of pulling a third of its overall revenues from the oncology field by 2030. The work with Trodelvy isn't done yet, though, as the company looks to advance the med into earlier lines of therapy, plus expand into different tumor types. Then there’s Arcellex-partnered CAR-T anito-cel, which is “well-positioned” for a 2026 launch in multiple myeloma. While Gilead remains busy with its oncology ambitions, the company hasn’t forgotten about its bread-and-butter HIV franchise. Gilead is looking at five potential new launches by 2030 in both treatment and pre-exposure prophylaxis (PrEP), many of which use the company’s long-acting HIV treatment lenacapivir (currently marketed as Sunlenca).

UPDATED: Monday, Jan. 8 at 1:35 p.m. ET

2024 looks to be a “busy year” for Amgen, CEO Robert Bradway said during his company's presentation on Monday. After integrating Horizon Therapeutics and building out its biosimilar business, the company is putting a particular focus on its obesity program, led by phase 2 asset MariTide (maridebart cafraglutide). Despite intense competition in the obesity space, the company says that its contender has a differentiated profile. Amgen plans to review results from a clinical study of the prospect later this year. Aside from MariTide, Amgen is working on an oral small molecule that’s currently in phase 1, as well as “half a dozen or so” preclinical programs, Bradway said. In oncology, Amgen's tarlatamab is up for an FDA decision by June 12 in certain patients with advanced small cell lung cancer. Beyond that indication, the company looks to study the med in earlier disease stages. As for biosimilars, Amgen's next three launches will target Johnson & Johnson’s Stelara, Regeneron’s Eylea and Alexion’s Soliris. Behind those three meds is a biosimilar candidate targeting Bristol Myers Squibb’s Opdivo and two other undisclosed medicines.

UPDATED: Monday, Jan. 8 at 12:50 p.m. ET

It took Bristol Myers Squibb’s new CEO Chris Boerner 16 minutes into his 24-minute presentation to address the big news the company made last month with its holiday buyouts of Karuna Therapeutics, for $14 billion, and RayzeBio, for $4.1 billion. What Boerner most wanted to highlight was the company’s pipeline, which could deliver more than 16 new products through 2030. “These products are overwhelmingly first or best in class,” Boerner said. “And it is the pipeline momentum that supports the growth opportunities that we see in the back half of the decade.” That period will be crunch time for BMS as it bears the brunt of patent expirations and Inflation Reduction Act effects for its trio of blockbusters drugs Revlimid, Eliquis and Opdivo, which accounted for 65% of the company’s revenue in 2022. Story

UPDATED: Monday, Jan. 8 at 12:44 p.m. ET

Blueprint Medicines has “decided to discontinue global development and marketing of Gavreto in territories excluding the U.S. and Greater China,” the company said in a Monday update. Before this disclosure, Blueprint’s former partner on the targeted cancer medicine, Roche, opted to end their collaboration. Blueprint cited a “lack of global infrastructure in lung and thyroid cancer” as its reason for discontinuing certain efforts on the medicine. Besides the Gavreto disclosure, Blueprint said it’s ending further investment in pipeline meds BLU-945 and BLU-451. Meanwhile, Blueprint expects Ayvakit’s launch in indolent systemic mastocytosis to “drive strong revenue growth in 2024,” the company said. Release 

Even after a late-stage trial failure for sparsentan in focal segmental glomerulosclerosis (FSGS), Travere Therapeutics said it’s conducting additional reviews and “will engage with regulators to evaluate potential regulatory pathways.” The development comes after the company last month said it was cutting 20% of its workforce to reduce operating costs in the wake of the trial failure. Travere’s sparsentan is already approved with the brand name Filspari to treat primary immunoglobulin A nephropathy. Since the launch in February 2023, the drug has generated $30 million, the company said on Monday. Release 

As Apellis progresses with the launch of two key brands, the company said it pulled down roughly $366 million during 2023. Syfovre, Apellis’ treatment for geographic atrophy, generated around $275 million, while paroxysmal nocturnal hemoglobinuria medicine Empaveli was responsible for $91 million of the company's haul. Both drugs use the same active ingredient, pegcetacoplan, which Apellis is also testing in several other rare diseases. In a statement, Apellis’ CEO Cedric Francois, M.D., Ph.D., said the company’s recent commercial progress “has positioned us for an even stronger 2024.” Release 

UPDATED: Monday, Jan. 8 at 11:21 a.m. ET

Regeneron hopes its longer-lasting Eylea formulation, Eylea HD, can build on the success of the company's original blockbuster product. With $123 million in sales during Eylea HD's first quarter on the market, the eye med is showing early indicators of broad initial uptake, CEO Leonard Schleifer, M.D., Ph.D., said in a corporate update. But overall Q4 Eylea sales, at $1.46 billion, missed analyst expectations of $1.49 billion to $1.56 billion, according to Piper Sandler analyst Christopher Raymond. Meanwhile, Sanofi-partnered immunology superstar Dupixent posted solid sales growth in 2023 continues to build on its “pipeline in a product” potential, according to Regeneron. The drugmaker highlighted the biologic's potential in chronic obstructive pulmonary disease, an indication where Dupixent has picked up an FDA breakthrough therapy designation. Elsewhere, the company looks to gain ground in oncology with its Libtayo offering. The med is slated to exceed $1 billion in global net product sales in 2024, Regeneron said. Release

UPDATED: Monday, Jan. 8 at 10:35 a.m. ET

Exelixis has preliminarily disclosed 2023 U.S. Cabometyx revenues of $1.63 billion—compared with $1.4 billion in 2022—and it projects 2024 product sales will land between $1.65 billion and $1.75 billion. Cabometyx, used in combination with Roche’s Tecentriq, recently beat a change of hormonal therapy on progression-free survival in metastatic castration-resistant prostate cancer patients who had tried a hormonal therapy. As the study continues to evaluate its other primary endpoint of overall survival, Exelixis said it will continue to talk to the FDA to find a potential regulatory path forward. Another investigator-initiated trial also showed Cabometyx’s benefit in advanced neuroendocrine tumors, and Exelixis is working on a potential FDA filing in that indication this year. Amid uncertainties related to Cabometyx’s label expansion, Exelixis will launch a restructuring that will cut about 13% of its workforce, or about 175 employees, to focus on its pipeline. The company’s board also authorized a $450 million stock buyback for 2024. Release

UPDATED: Monday, Jan. 8 at 10:10 a.m. ET

For all of 2023, PTC Therapeutics brought in $946 million in unaudited revenues, good for a 35% increase from the prior year. Besides product revenues of $661 million, PTC’s collaboration and royalty revenue associated with Roche’s commercialization of spinal muscular atrophy therapy Evrysdi came to $278 million, the company said in a release. PTC is kicking off JPM with a 10:30 a.m. ET presentation today, where it’ll share updates about its ongoing launches and pipeline advancement efforts. In 2024, the company expects to finalize FDA and European Medicines Agency filings for sepiapterin for phenylketonuria. The drugmaker also expects to submit an FDA filing for Upstaza for the treatment of AADC deficiency during the first quarter of the year, among other planned R&D developments. Release 

After a “transformative year,” Mirum Pharmaceuticals CEO Chris Peetz said the company is looking toward more milestones in 2024. Specifically, the company projects continued revenue growth and a potential label expansion for Livmarli in progressive familial intrahepatic cholestasis by March 13. Further, the company is preparing a regulatory filing for Chenodal in cerebrotendinous xanthomatosis after a phase 3 trial win. As for 2023, the company said it generated $186 million to $188 million, but that the figure is unaudited. The company’s Alagille syndrome therapy Livmarli pulled down $141 million to $143 million last year, good for around 89% growth from the prior year. Mirum’s full JPM presentation will be on Wednesday at 1:30 p.m. ET. Release

UPDATED: Monday, Jan. 8 at 9:10 a.m. ET

With nine late-stage pipeline programs in development and 45 overall, Moderna expects to have an “exciting year," CEO Stéphane Bancel said in a business update. The mRNA specialist pulled down $6.7 billion in 2023 revenues, including $6.1 billion in COVID-19 vaccine sales. Moderna expects its Spikevax to remain profitable and projects some $4 billion in 2024 product sales, which includes contributions from an anticipated RSV vaccine launch. Other than its RSV prospect, the company looks to file its seasonal flu vaccine with regulators this year and is expecting data from several assets, including its next-generation COVID vaccine and its cytomegalovirus vaccine. Moderna’s full presentation at the conference will take place at 6:45 p.m. ET today. Release

EY's Firepower report, which publishes annually on the opening day of the J.P. Morgan Healthcare Conference, assesses the capacity of the industry’s top 25 companies to execute M&A deals based on the strength of their balance sheets. EY tabulated the available firepower of the companies at $1.37 trillion entering 2024, which is the second-highest figure in the 10 years of the analysis. Another factor that bodes well for M&A in 2024 is the sudden surge in high-dollar deals at the end of 2023. Several other elements are in play—including patent expirations, government regulations and the recent failure of several late-stage assets—which signal urgency by large pharma companies to make deals for late-stage and commercial assets, EY analysts say. Story

Busy with three gene therapy launches, bluebird bio on Monday said it has cash on hand to last until the first quarter of 2025. On the heels of a recent FDA approval for bluebird’s sickle cell disease gene therapy Lyfgenia, 35 treatment centers are ready to receive referrals for the medicine, bluebird said in a release. Plus, after recently striking two outcomes-based deals on Lyfgenia, the company says talks with other payers are in “advanced discussions.” Besides Lyfgenia, bluebird is also progressing with the launches of beta thalassemia medicine Zynteglo and cerebral adrenoleukodystrophy medicine Skysona. Bluebird’s full presentation at the J.P. Morgan Healthcare Conference is on Tuesday at 1:30 p.m. ET. Release