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JP Morgan Healthcare Conference Day 1: Bristol Myers Squibb, Vertex, Sarepta, Novartis, and More 

JPM Day 1 V2

NEW YORK – On the first day of the 42nd annual JP Morgan Healthcare Conference in San Francisco, Bristol Myers Squibb discussed using acquisitions to diversify its portfolio beyond immunotherapies; Vertex talked about improving access to its sickle cell gene therapy Casgevy, and Novartis said it is putting Pluvicto's supply challenges in the rear view. 

Bristol Myers Squibb 

Bristol Myers Squibb is aiming to expand its oncology portfolio beyond immunotherapy in the next several years by adding antibody-drug conjugates, targeted therapies, and radiopharmaceuticals through acquisitions. Ahead of the loss of market exclusivity for several products toward the end of the decade, including its blockbuster immunotherapy Opdivo (nivolumab), BMS has gone on an acquisition spree to diversify its oncology assets. 

For example, BMS acquired Turning Point Therapeutics in 2022 and Mirati Therapeutics in 2023 to add new targeted treatments to its portfolio. Those acquisitions have already brought in commercialized precision medicine products for BMS in advanced non-small cell lung cancer, including Mirati's KRAS G12C inhibitor Krazati (adagrasib) and Turning Point's ROS1 inhibitor Augtyro (repotrectinib). Through the Mirati acquisition, BMS also gained multiple investigational RAS inhibitors, a SOS1-targeted program, and a PRMT5/MTA inhibitor, while with the Turning Point purchase, BMS brought the MET inhibitor elzovantinib, the RET inhibitor TPX-0046; and the ALK inhibitor TPX-0131 under its aegis. 

Most recently, BMS acquired RayzeBio for its pipeline of actinium-based radiopharmaceutical therapeutics for the treatment of solid tumors, including gastroenteropancreatic neuroendocrine tumors, small cell lung cancer, hepatocellular carcinoma, and other cancers. Beyond acquisitions, BMS CEO Chris Boerner said the firm is also focusing on internally developing targeted protein degraders and cell therapies. 

In 2024, BMS is expecting readouts from several trials of its new assets, including a study of Krazati with Merck's checkpoint inhibitor Keytruda (pembrolizumab) in first-line KRAS-mutant NSCLC with a PD-L1 tumor proportion score greater than 50 percent and a study of single-agent Krazati in second-line KRAS-mutant NSCLC. BMS is also looking ahead to data on Mirati's PRMT5/MTA inhibitor in MTAP-deleted cancers this year. 

In coming years, BMS will continue to use business development to enhance its pipeline, Boerner said. "We've got a great set of assets internally. We've got a great R&D organization with a strong pipeline, but we don't have a monopoly on great ideas," he said. 

Vertex Pharmaceuticals 

Vertex Pharmaceuticals is aiming to have 50 US sites and 25 European sites within its network of treatment centers authorized to administer its newly approved sickle cell gene-editing therapy, Casgevy (exagamglogene autotemcel). 

Vertex, which received permission to begin marketing Casgevy as a treatment for sickle cell disease in the US and as a treatment for sickle cell and transfusion-dependent beta thalassemia in the UK and Bahrain last year, is setting up a network of hospitals with clinicians trained to administer the first-ever approved CRISPR gene-editing treatment. 

Casgevy involves editing the BCL11A gene in patients' own CD34-positive hematopoietic stem and progenitor cells ex vivo with CRISPR-Cas9 to increase production of fetal hemoglobin, the adult version of which is dysfunctional in patients with sickle cell and transfusion-dependent beta thalassemia. 

So far, Vertex has authorized nine treatment centers in the US and three in Europe. 

Casgevy represents multibillion-dollar market potential, according to Vertex CEO and President Reshma Kewalramani, and marks the start of Vertex's move into new diseases. 

Previously, Vertex's approved therapies had all been in cystic fibrosis. 

"We will begin to diversify our revenues in 2024 with the launch of Casgevy," she said. 

Vertex, which priced Casgevy at a whopping $2.2 million, has signed an agreement with Synergie Medication Collective, a medication contracting organization founded by a group of Blue Cross and Blue Shield-affiliated companies covering about 100 million lives, to provide access to the treatment. The company is working on setting up contracts with other payor organizations. 

Casgevy is currently under regulatory review in Switzerland and the Kingdom of Saudi Arabia, and Vertex plans to submit for approval in Canada in the first half of this year. 

Kewalramani also said Vertex expects to select a dose and begin the Phase III portion of an ongoing Phase II/III pivotal trial of inaxaplin, a therapy for kidney disease associated with certain mutations in the APOL1 gene, in the first quarter of 2024. Inaxaplin aims to inhibit the function of APOL1 proteins, which can cause damage to kidney cells. 

Sarepta Therapeutics 

Sarepta Therapeutics estimates that its 2023 net product revenue from its gene therapy Elevidys (delandistrogene moxeparvovec-rokl), which received accelerated approval last year, was $200.4 million, based on preliminary and unaudited financial results. 

In the fourth quarter of 2023, specifically, Sarepta estimates that Elevidys brought in $131.3 million in net product revenue. 

Elevidys, the first gene therapy to garner approval as a treatment for Duchenne muscular dystrophy (DMD) in the US, delivers a copy of a gene that encodes for microdystrophin, an engineered protein developed by Sarepta. The firm says microdystrophin can carry out the normal functions of dystrophin, the protein deficient in patients with DMD. 

Sarepta's expected total net product revenue for the year is $1.15 billion, compared to $843.8 million in 2022. 

Sarepta CEO and President Doug Ingram expressed optimism about the company's finances in 2024 but said he could not provide financial guidance yet since it will depend on whether the US Food and Drug Administration expands the label for Elevidys, as the company requested late last year

"Nevertheless, it is quite clear that 2024 is an enormously important year for us," he said. 

The FDA in June last year granted Sarepta accelerated approval for Elevidys. However, it limited the indication to patients between 4 and 5 years old. Sarepta has submitted additional data to the agency as it seeks to expand the indication to all ages and convert the accelerated approval to a traditional approval. 

Ingram said he expects the FDA to make its decision on the expanded indication by August, since he hopes that the agency will grant the company priority review for the request. 

But "we are very hopeful that we can move more expeditiously than that," he added. 

He said he does not expect the FDA to request another meeting of its Cellular, Tissue, and Gene Therapies Advisory Committee to weigh in on the decision. 

Outside of DMD, the company is also advancing multiple gene therapy candidates for limb-girdle muscular dystrophy and will begin screening patients for a pivotal trial of one of the candidates, dubbed SRP-9003, this year.

At the end of 2023, Sarepta had preliminary cash, cash equivalents, restricted cash, and investments of about $1.7 billion. 

Sarepta said it will report complete fourth quarter and full-year 2023 financial results in February. 


Novartis said that its chronic myeloid leukemia drug Scemblix (asciminib) met the primary and secondary endpoints in the Phase III ASC4FIRST clinical trial, and the firm will share the update with regulatory authorities. 

In ASC4FIRST, patients with newly diagnosed Philadelphia chromosome-positive chronic-phase CML achieved higher rates of major molecular responses when treated with Scemblix versus standard-of-care tyrosine kinase inhibitors, including Novartis' Gleevec (imatinib) or Tasigna (nilotinib), Bristol Myers Squibb's Sprycel (dasatinib), or Pfizer's Bosulif (bosutinib). Scemblix also had a better safety and tolerability profile than standard treatments, and fewer patients discontinued Scemblix than they did other TKIs. 

Novartis said that it will report the specific data at an upcoming medical conference and submit the data to regulatory authorities in 2024. 

"From our perspective, as the company that brought Gleevec to the world in 2001 and redefined the opportunity to treat patients with targeted therapies in cancer … to once again raise the standard of care in the frontline setting with an oral medicine … is one of the triumphs of Novartis scientists and Novartis chemistry," Novartis CEO Vas Narasimhan said. 

Finally, Novartis appears to be putting its supply challenges for Pluvicto (lutetium vipivotide tetraxetan) in the rearview mirror now that its largest radioligand therapy manufacturing facility has netted US Food and Drug Administration approval for commercial use. The 70,000-square-foot facility, based in Indianapolis, will be used to manufacture both Pluvicto and the neuroendocrine cancer radiopharmaceutical Lutathera (177Lu-dotatate). 

"For radioligand therapy, we are now fully supply-unconstrained both for Lutathera and Pluvicto and our pipeline," Narasimhan said. 

Although Narasimhan acknowledged that Novartis is one of the only major pharma companies to not enter an antibody-drug conjugate (ADC)-focused deal this year, he said the firm's focus is on radioligand acquisitions instead. Novartis is focused on trying to advance HER2- and HER3-targeting radioligands into the clinic, for instance, which are both frequently targets for ADC development. 


Biogen said it will file an application in the first quarter seeking the US Food and Drug Administration's approval for Leqembi (lecanemab), an anti-amyloid monoclonal antibody, as a maintenance treatment for Alzheimer's disease. 

Leqembi, developed by Biogen and Eisai, targets beta-amyloid, proteins known to form plaques that are a hallmark of Alzheimer's disease. The FDA granted Leqembi full approval in July 2023, converting the agent's accelerated approval earlier in the year, based on data showing that it slowed Alzheimer's progression. The drug is indicated for adult Alzheimer's patients with mild cognitive impairment or mild dementia, and the drug's label instructs doctors to confirm the presence of elevated beta-amyloid before starting the treatment. 

However, there's been uncertainty around how long patients need to receive the treatment. The drug is an intravenous infusion administered every two weeks, but it's unclear if patients could stop treatment once they reach amyloid clearance or if they will need to take it for longer. Additionally, there is ongoing research looking at whether Leqembi can be administered earlier to patients to delay or prevent cognitive decline in patients who have elevated levels of beta-amyloid without Alzheimer's symptoms. Taking all this into account, patients might be on the treatment for "quite a long time," Biogen President and CEO Christopher Viehbacher said. 

Viehbacher cited data that Biogen presented at the Clinical Trials on Alzheimer's Disease conference in October from an open-label extension study of Leqembi, in which patients continued to demonstrate cognitive and functional benefits compared to placebo groups after 24 months of treatment. 

Biogen is also working on a subcutaneous formulation of Leqembi, on which the company also expects to file a biologics license application this quarter. "That's going to be extremely important," Viehbacher said, especially if patients may be on the therapy for a longer period of time. 

Leqembi is Biogen and Eisai's second anti-amyloid Alzheimer's drug, following the controversial approval of Aduhelm (aducanumab) in 2021 despite mixed evidence and concerns raised by an advisory committee for the FDA. Biogen has previously said there is no longer a commercial effort behind Aduhelm, and Viehbacher acknowledged that figuring out a path forward for Aduhelm was "one of the hardest business problems" he's had. 

While he maintained that the product is effective against Alzheimer's, the investment to complete confirmatory studies may not be worth it. "We are actually in discussion with outside parties and outside financial interests, and we're coming to the conclusion of that," he said, noting that the company will provide an update on these discussions in Q4 2024. 

Gilead Sciences 

Gilead Sciences is on track to reach its goal of having its oncology portfolio comprise one-third of all sales by 2030, according to CEO Dan O'Day. The drugmaker is planning to provide updates on more than 10 oncology trials in 2024, including a Phase III trial assessing the antibody-drug conjugate Trodelvy (sacituzumab govitecan) in first-line metastatic triple-negative breast cancer. 

Gilead also said it anticipates upcoming improvements to its cell therapy manufacturing time. Currently, it takes a total of 16 days to manufacture the autologous B-cell lymphoma cell therapies, Yescarta (axicabtagene ciloleucel) and Tecartus (brexucabtagene autoleucel), but O'Day said he expects the firm to shorten this time to fewer than 16 days during 2024. 

Gilead is also targeting 2026 for the launch of another autologous cell therapy, anitocabtagene autoleucel, which it is developing with Arcellx as a treatment for B-cell maturation antigen (BCMA)-targeting multiple myeloma. The firm will share preliminary data from the Phase II iMMagine-1 trial during the second half of 2024. 

In late November 2023, the US Food and Drug Administration warned that patients receiving autologous CAR T-cell therapies, including those targeting CD19 such as Yescarta and Tecartus, may be at higher risk of developing new T-cell malignancies. 

Gilead Chief Commercial Officer Johanna Mercier said that across 13,000 patients treated with Yescarta and Tecartus, the firm has not seen a causal relationship between these autologous cell therapies and secondary T-cell cancers. "This [risk] is something we have known from the start," Mercier said. "It's a theoretical concern. It has been embedded within our protocol and within our informed consents and our label since 2017. The FDA is sharing that concern [but] we are not seeing causality as an impact of secondary T-cell malignancies [with Yescarta] and the same for Tecartus." 

Gilead will announce its 2023 fourth quarter and full-year financial earnings on Feb. 6. 

Exact Sciences 

Exact Sciences CEO Kevin Conroy touted the potential growth of Cologuard over the next few years, saying that the firm eventually plans to get to about 14 million Cologuard tests performed each year. That figure assumes Cologuard reaches 40 percent market share. 

Conroy also mentioned the next-generation version of Cologuard, which is intended to reduce the false-positive rate of the current test. In the presentation of the firm's Blue-C study in October, the next-generation test had a specificity of 91 percent and increased sensitivity for both cancer and advanced precancer, to 94 percent and 43 percent, respectively. That increase in specificity implies up to 30 percent fewer false positives, Conroy said in the firm's third-quarter earnings call in November. 

In December, Exact Sciences completed its submission to the US Food and Drug Administration for the next-generation test and expects approval by early 2025, Conroy said on Monday. 

Meanwhile, Exact Sciences CFO Jeff Elliott, who announced on Sunday that he was stepping down this year due to personal reasons, said that Cologuard revenues grew 31 percent year over year. He noted that the company has longer-term growth ambitions of more than $7 billion in revenue from Cologuard alone. Conroy added that the firm also intends to grow revenues for the Oncotype DX tests from more than $600 million to more than $1 billion. 

Also on Sunday, the company announced preliminary financial results, saying it expects fourth quarter revenue to grow 17 percent year over year to between $645.5 million and $647.5 million, beating analysts' average estimate of $631.5 million. 

Everett Cunningham, the company's chief commercial officer, said that the Cologuard growth levers are clear. They include the rescreening of patients, which constitutes 20 percent of Cologuard's volumes, as well as patients aged 45 years old to 49 years old. Growth also would come from greater screening of African American patients and making it easier to order the test by electronically interfacing with customers. 

The firm's 2024 focus areas include greater adoption of Cologuard as a first-line screening test and expanding the impact of its Oncotype DX test for breast cancer globally, Conroy said. The company also intends to deepen its relationships with health systems and to launch future tests. 

Exact is also opening new markets for Oncotype DX, with Japan a key area of focus and presenting the "largest growth opportunity for Exact Sciences on the oncology side outside of the US," Elliott said. 

Lastly, the company is working on its "rich pipeline," Conroy said, which includes the next-generation version of Cologuard, the OncoDetect minimal residual disease test, the Cancerguard multi-cancer screening test, OncoExTra for therapy selection using tissue, and OncoLiquid for therapy selection using blood. 


NeoGenomics Laboratories CEO Chris Smith said the company plans to push ahead with its MolDx submissions for its Radar minimal residual disease (MRD) assay despite a preliminary injunction issued against the company restricting its sales of the test. 

The US District Court for the Middle District of North Carolina issued the order at the end of December as part of Natera's ongoing patent infringement lawsuit against NeoGenomics. It bars the company from "making, using, selling, or offering for sale in the US" as well as from promoting or advertising the test, though NeoGenomics may continue offering the assay for existing patients and for clinical trials, research studies, and projects already in process. 

NeoGenomics said it plans to appeal the ruling, a position Smith reiterated Monday during the company's presentation, noting that "we feel really good about our legal position," adding that it plans to continue to invest in the assay and take it through the MolDx process. 

During Q3 2023, NeoGenomics completed three submissions to MolDx, one for breast cancer, one for lung cancer, and one for head and neck cancer. During Q2 2023, the company received Medicare coverage for use of the Radar test in patients with HR-positive, HER2-negative breast cancer. 

Smith said NeoGenomics believes MRD testing is an area of significant opportunity for the company, putting it at a roughly $20 billion market that is only around 5 percent penetrated. 

"I think it's important to be there with a sensitive, informed assay," he said, adding that the company currently has 28 studies using the Radar product underway. 

Smith highlighted several areas of focus for NeoGenomics in 2024, including increasing test volumes, particularly in the community setting; improving the efficiency of its sales force; and continuing to improve revenue cycle management. 

Smith also tackled the question of how the US Food and Drug Administration's plans to regulate laboratory-developed tests might impact NeoGenomics, noting that while the outcome of the FDA's effort remains uncertain, "our view is that [FDA regulation of LDTs] probably ultimately becomes a reality." 

In particular, Smith said the company expected that MRD and next-generation-sequencing tests would have to go through the FDA given the complexity of those technologies and said that NeoGenomics is putting such tests under design control. 

He added that he saw potential upside to FDA oversight of LDTs "for companies that are in front of this." 

He noted, for instance, that one challenge NeoGenomics currently faces is hospitals wanting to internalize NGS testing. He suggested that smaller hospitals in particular might be discouraged from doing this if the FDA begins regulating such tests. 

"The companies that move quickly and are prepared are, I think, the ones that are going to come out ahead," he said. "But it is going to be a long time before we see how this shakes out." 


Veracyte CEO Marc Stapley further explained the impetus behind the company's purchase of C2i Genomics for up to $95 million, announced on Monday, noting that it enables Veracyte to move into the minimal residual disease (MRD) testing market and to "expand our role across the cancer care continuum." 

C2i has developed a platform that uses whole-genome tumor sequencing for MRD testing. 

In looking at potential options to enter the MRD market, Veracyte sought a company with a clear path to commercialization in its existing specialty channels, he said. Veracyte has "been thinking about expanding the markets that we're addressing for a number of years," he said, and MRD was a particular area of interest because "we want to be able to take care of the patient during and after treatment." For this deal, "the timing was right," and integrating the acquisition will be a key focus going forward, he added. 

Whole-genome sequencing was the best fit for Veracyte's platform, he added, and there are a "number of benefits" that make C2i's technology the best solution, including its small sample requirement and the rapid generation of patient-specific signatures. Veracyte believes the whole-genome approach offers a "path to move from tumor-informed to tumor-naïve [testing] if desired," he added. The firm will begin by focusing on markets where it has a "strong commercial channel and relationships" and build out from there. Although the company intends to develop tests for multiple indications, including urologic and lung cancers, its first indication will be in muscle-invasive bladder cancer, with the first test expected to launch in the first half of 2026. 

"With our established urology channel and relationships, we can partner from diagnosis on and benefit from a well-defined path to reimbursement," he said. 

Stapley also commented briefly on his thoughts about the US Food and Drug Administration's proposal to regulate laboratory-developed tests, saying that the firm is "poised, prepared, and ready to respond" to any guidelines the FDA enacts. "Given the level of evidence around our tests, I clearly have confidence our test would be able to go through whatever the IVD bar is in the US," he said. Because the firm already abides by CLIA and New York state laboratory requirements, it is ready to meet more stringent requirements, he added. 

"We're already held to very high standards in clinical testing, and so I don't really see having to go through the FDA process, whatever it ends up being, as a major milestone for us to have to climb over," he said. 

The company also announced preliminary Q4 and full-year 2023 financial results on Monday. Stapley addressed a decline in forecast biopharmaceutical revenues, which are expected to be about 50 percent of 2023 biopharma revenues, noting that the biopharma segment makes up only about 5 percent of total revenues. Veracyte hopes that the difficult macroeconomic environment around biopharma is nearing its end and is continuing to build out its portfolio for that business, he said. 

Guardant Health 

Guardant Health co-CEO Helmy Eltoukhy emphasized the importance of the Guardant360 test to the company, saying that it is the primary driver of volume growth, with 173,000 clinical tests performed in 2023. The company expects the test's average sales price (ASP) to increase by about $200 dollars in Q1 2024 compared to Q3 2023, due to changes in reimbursement from Medicare. The firm has also started to see "faster and better payments" from private payors, such as United Healthcare and Anthem, which may lead to additional upside on the test's ASP, CFO Michael Bell said. 

Eltoukhy noted that the firm is on track this year to launch an updated version of Guardant360 using its "smart liquid biopsy" platform, which incorporates genomic and epigenomic biomarkers. 

The upgraded version of the test "will help identify more patients for existing therapies that are undetectable by current [comprehensive genomic profiling] tests, identify novel targets for new therapies for biopharma partners, and provide detailed phenotypic information about the tumor, such as histology, subtype, and more," he said. 

It also allows for new features to be continually added to the test as they are clinically validated. The company also plans to launch an upgraded and expanded version of its Guardant360 TissueNext test this year, he said. 

Eltoukhy noted the importance of the National Comprehensive Cancer Network's updated non-small cell lung cancer testing guidelines released in December. The guidelines now include the use of concurrent testing of tissue and plasma to improve time to test results. Lung cancer "tends to be the lead indication when you think about solid tumors," he said, and the addition of concurrent testing "bodes well for other cancer types." The guidelines are "taking what we think is the right stance in terms of maximizing sensitivity for patients — essentially they recognize that liquid and tissue are both valid means for getting this information," he added. 

Guardant Reveal, the company's minimal residual disease test, has already been upgraded to the smart platform and is available for colorectal, breast, and lung cancer testing, with expansions to other tumor types planned for the future. 

Beyond its therapy selection assays, co-CEO AmirAli Talasaz provided updates on the company's screening test, Guardant Shield. The firm has an advisory committee panel discussion with the US Food and Drug Administration in March as the next step in the review of its submission for premarket approval for colorectal cancer, he said. After approval, which the company expects this year, Guardant will launch the test, seek Medicare coverage, and hopefully get guideline inclusions in 2025 and 2026. 

The firm also plans to expand Shield's indications, working first on lung cancer. It is "continuing to make good progress" on enrollment in its lung cancer screening study and is also developing a multi-cancer early detection indication expansion for the Shield test, he said. 

In contrast to many other firms that have been impacted by downturns in the biopharma market, Eltoukhy said that biopharma has been "a really nice area of growth and strength" for Guardant. He also noted that potential collaborations with biopharma companies may expand beyond oncology to other areas of precision medicine. 


Pfizer CEO Albert Bourla conceded that 2023 was "not a very good year" for the company, largely due to high expectations for the performance of its COVID-19 products. For 2024, however, Pfizer has reduced expectations for its COVID-19 products and has refocused on its oncology business and new product portfolio, he said at the conference. 

With Pfizer's $43 billion acquisition of Seagen, which closed in December, the firm's oncology pipeline has doubled its number of candidates, and the firm gained new cancer therapies already on the market. Pfizer has further created a separate structure for its oncology business in the wake of the acquisition, one that better brings together early- and late-stage research and commercial activities, Bourla said. 

"The first thing that we discussed with all the Seagen leaders was: 'What is your secret of success?'" he said. "I realized that the handoff in oncology between early and late [research] is way faster [than other therapeutic areas], where there are very distinct phases, where in oncology it's all blended. It was extremely important to bring early and late together." 

He also learned from Seagen that bringing the commercial division into the mix earlier for oncology was important. "Oncology is very technical," he said. "You are not selling consumer conviction. You're selling on data and your ability to explain those data to the people. So, it was decided that [R&D and commercial] would become one." 

The new Pfizer Oncology division is led by Chris Boshoff as chief oncology officer. Bourla also noted that the leadership of this unit is split evenly between Pfizer and Seagen leaders, as is the overall employee mix in the division. 

"We have seen multiple times that big companies like us buying smaller companies like Seagen and killing the innovation in the first year because they bring them into their own bureaucracy," Bourla said. 

This year, Bourla is excited about several oncology products entering Phase III studies, including the multiple myeloma treatment elranatamab, the investigational CDK4 inhibitor in breast cancer, and a Seagen antibody-drug conjugate asset, SGN-B6A, in non-small cell lung cancer.