JPM Day 1: Highlights

San Francisco Bay

Follow updates from the JPM Annual Healthcare Conference - BioSpace will be tracking key updates here all day. All times are Eastern Standard. 

5:15 p.m.: AstraZeneca didn’t waste time waiting for its Monday afternoon presentation at JPM to make waves. The British pharma made a big move to augment its cardiovascular and kidney business with the $1.8 billion acquisition of CinCor Pharma, announced early in the day.

The deal’s crown jewel is baxdrostat, which has completed Phase II trials as a treatment to lower blood pressure in patients with treatment-resistant hypertension. While the drug failed to meet the primary endpoint, CinCor emphasized its plans to move forward with pivotal trials in the first half of 2023. This will now be AstraZeneca’s call.

The experimental therapy is also in Phase II clinical trials for aldosteronism and chronic kidney disease.

Astra also hopes to pair baxdrostat with its blockbuster diabetes drug Farxiga (dapagliflozin), which also possesses strong cardiorenal therapeutic activity.

Astra’s heart, kidney and diabetes portfolio combined generated about $6.9 billion of the $33 billion-plus the company raked in during the first three quarters of 2022. The CinCor deal is a clear statement that Astra intends to increase that percentage.  

5:15 p.m.: Along with touting an unaudited $18.4 billion in sales for its COVID-19 vaccine in 2022 and projecting an additional $5 billion in 2023, Moderna CEO Stéphane Bancel highlighted the company’s late-stage infectious disease programs.  

Bancel referred to “exciting near-term catalysts” for Phase III RSV and flu data. The pivotal Phase III study of RSV candidate mRNA-1345 in adults 60-plus has accrued enough cases for Moderna to complete a first interim efficacy analysis, according to the business update at JPM.   

Other highlights of Bancel’s presentation included: 

  • Recently announced positive data from the Phase IIb KEYNOTE-942 trial assessing Moderna’s investigational mRNA cancer vaccine in combination with Merck’s Keytruda for stage III/IV melanoma. The combo reduced the risk of recurrence or death by 44% compared to Keytruda alone. 

  • A Phase III study of this asset in melanoma is slated to begin in 2023, with “rapid expansion to additional tumor types.” 

  • Seasonal flu vaccine update: Moderna reported that the mRNA-1010 Southern Hemisphere immunogenicity study in adults 18-plus is fully enrolled with a data readout expected in the first quarter of 2023.   

1:30 p.m.: In November, the FDA accepted Sarepta Therapeutics’ Biologics License Application for the accelerated approval of SRP-9001 for Duchenne muscular dystrophy.

If approved, SRP-9001, which treats the underlying cause of the disease by delivering a functional shortened dystrophin to muscle, would be the first gene therapy on the market for Duchenne.

Understandably, Sarepta President and CEO Doug Ingram used his allotted time at JPM Monday to outline the data behind the BLA submission and the path to the BLA.

“2023 will be a bellwether, not merely for Sarepta, but for families with Duchenne muscular dystrophy and I think also, in a very real sense, for the promise of gene therapy,” Ingram said.

He went on to describe the design of the gene cassette and discussed an evidence set that included preclinical animal models, functional data and “a wealth” of patient biomarkers.

The FDA has set a regulatory action date of May 29, 2023.

12:51 p.m.: BioMarin Chairman and CEO J.J. Bienaimé touted the company’s “strongest launch” to date with the expected commercial launch of Roctavian, a therapy to treat severe hemophilia A.

Hemophilia A is an inherited bleeding disorder caused by the lack of a clotting protein known as factor VIII.

Coupled with Voxzogo, a therapy used to increase linear growth in children with achondroplasia (aged 5 years and older), Bienaimé predicted $4-5 billion in growth for BioMarin by mid-decade.

What’s more, when forecasting the use of Roctavian to treat hemophilia A worldwide, the company cited a $14 billion potential gain in market share.

Bienaimé said he expected more than 75% of U.S. prescribers to adopt Roctavian within one year of market availability to treat severe adult hemophilia A patients.

12:45 p.m.: Alnylam Pharmaceuticals CEO Yvonne Greenstreet kicked off her presentation by reviewing the highlights of her first year on the job. Greenstreet cited three in particular:

1.) “Excellent progress” in the company’s commercial performance.

2.) “Landmark results” from the Phase III APOLLO-B trial of patisiran in transthyretin-mediated amyloidosis. 

3.) The approval of Amvuttra (vutrisiran), an RNAi therapeutic developed for adults with hereditary (hATTR) amyloidosis.

Greenstreet said Alnylam will continue this momentum into 2023, as the company anticipates clinical data from ALN-APP, the first-ever RNAi therapeutic targeting CNS diseases (Alzheimer’s and cerebral amyloid angiopathy).

She also noted that pending regulatory review, Alnylam expects the expansion of Onpattro to treat cardiomyopathy of ATTR Amyloidosis, which would mark an “important inflection point” in the build-out of the company’s ATTR franchise.

In terms of this recent sNDA submission, Greenstreet said Alnylam expects a standard review from the FDA. 

12:45 p.m.: Johnson & Johnson CEO Joaquin Duato outlined the healthcare behemoth’s strategy to downsize from a three-segment company to a more specialized two-unit business focused solely on medtech and pharmaceuticals. The company’s CEO noted that J&J remains on track to generate $60 billion in pharmaceutical sales by 2025.  

Duato said the bulk of this growth will come from the company’s existing portfolio of largely de-risked assets, such as the multiple myeloma medicine Darzalex and the plaque psoriasis treatment Tremfya. Duato noted that Wall Street seems to be underestimating the commercial potential of several of the company’s newer pharma products such as the novel depression treatment Spravato, its pulmonary arterial hypertension assets and its burgeoning multiple myeloma franchise.  

On the business development front, Duato said the company is well-equipped to identify and onboard external development pipeline opportunities. However, he didn’t share specific details regarding the company’s M&A priorities. He did deny J&J’s reported interest in Horizon Pharma, a company that was eventually purchased by Amgen.  

12:30 p.m.: Arrowhead Pharma CEO Christopher Anzalone, Ph.D., discussed Phase II data released just a few hours prior on fazirsiran, an investigational RNA therapeutic, in liver disease associated with alpha-1 antitrypsin deficiency (AATD-LD).

The therapy, developed jointly with Takeda, showed that 50% of patients treated with fazirsiran achieved an improvement in fibrosis of at least one point. This was compared to 38% of patients treated with a placebo, which was unusually high, causing some to doubt the efficacy of the therapeutic. 

During the presentation, Anzalone explained Arrowhead's motivations behind moving the therapeutic into Phase III despite doubts from investors. Arrowhead's stock plummeted over 20% following the data announcement Monday morning. The company expects to enter Phase III later this month.

11:15 a.m.: During a Monday morning presentation, Vas Narasimhan, CEO of Novartis, stated the company’s 2023 strategy is focused on five therapeutic areas: cardiovascular, immunology, neuroscience, solid tumors and hematology. 

One way the company plans to advance in these areas, Narasimhan said, is through its gene therapy portfolio, in which Narasimhan stated Novartis has “well over 15 projects in-house that are continuing to advance.” 

In terms of immunology, Narasimhan said Novartis will focus much of its efforts on cell and gene therapy using its T-Charge platform.

Specifically, Novartis is “rapidly trying to progress” into late-stage systemic lupus erythematosus (SLE) patients as well as other severe immunological diseases to create an “immune system reset” using CAR-T. 

Narasimhan stressed Novartis’ midterm target of 4% growth several times throughout the presentation, stating that though the company is the “number one player” in Europe, one of its primary goals this year is to improve its position in the U.S. market by implementing a “U.S. first” approach.

10:30 a.m.: Bristol-Myers Squibb revealed its plans to move beyond key patent expires later this decade. In the company’s presentation Monday morning, Bristol’s Executive Vice President and Chief Commercial Officer Chris Boerner, Ph.D. said the pharma titan expects annual sales to rise by low- to mid-single digits over the course of 2020 to 2025.  

In-line brands such as the Pfizer-partnered blood thinner, Eliquis, ought to contribute $8 billion to $10 billion in sales growth over this five-year period. Meanwhile, newer product launches ought to deliver between $10 billion to $13 billion in additional sales during the same time frame.

Looking forward, Boerner said the company’s late-stage pipeline has the potential to deliver upwards of $10 billion in risk-adjusted sales in the second half of the decade. BMS's vice president highlighted six late-stage product candidates as key growth drivers for the company in the back half of the decade: (milvexian, iberdomide, mezigdomide, BMS-986278, repotrectinib and cendakimab).

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