
SAN FRANCISCO – The 43rd annual JP Morgan Healthcare Conference took off here on Monday, bringing together companies developing precision medicines for cancer, rare conditions, and a variety of other diseases, diagnostic firms with tests for informing diagnosis and treatment, investors, and other industry players.
Below are short summaries of presentations from select companies that provided updates on their precision medicine and diagnostic pipelines on Monday.
Vertex Pharmaceuticals
Vertex Pharmaceuticals is considering pursuing accelerated approval for a kidney disease drug that is currently undergoing a pivotal trial.
Vertex has six commercialized products. That includes Casgevy (exagamglogene autotemcel), a therapy for sickle cell disease and transfusion-dependent beta thalassemia, which it codeveloped with CRISPR Therapeutics and which holds the distinction as the first-ever CRISPR drug to reach the market. The company also sells five CFTR modulators for cystic fibrosis.
As it expands into new disease settings, Vertex is testing multiple candidates within its growing renal franchise, including inaxaplin in APOL1-mediated kidney disease (AMKD). The daily therapy is designed to inhibit the function of APOL1 proteins, and the company is testing its activity in the Phase III portion of the Phase II/III AMPLITUDE trial. Vertex expects to complete enrollment of the interim analysis cohort this year, paving the way for a regulatory submission in the US.
That "positions us to file for potential accelerated approval in the US once this cohort completes 48 weeks of treatment," said Vertex CEO and President Reshma Kewalramani. The firm is also preparing to initiate an open-label Phase IIb trial dubbed AMPLIFIED to study inaxaplin in patients with AMKD and other comorbidities who weren't eligible for the AMPLITUDE trial.
If that second study demonstrates positive findings, it would increase the addressable market for inaxaplin from 150,000 to 250,000 patients, Kewalramani estimated.
Vertex is also in the midst of launching Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a daily oral medication for cystic fibrosis and its fifth CFTR modular to reach the US market following approval by the US Food and Drug Administration at the end of 2024.
Kewalramani said Vertex initially plans to focus on treating the roughly 6,000 patients who previously discontinued a CFTR modulator, as well as the estimated 250 patients with rare CFTR mutations who aren't eligible for other commercialized products. In addition, Vertex expects to transition some patients who are currently taking another one of its CFTR modulators, Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), to Alyftrek over time, since the newly approved drug offers a more convenient dosing schedule and may improve patients' outcomes.
Bristol Myers Squibb
Bristol Myers Squibb highlighted advances in its immuno-oncology portfolio in 2024 and also discussed forthcoming data from its oncology pipeline in 2025.
BMS CEO Chris Boerner reported double-digit sales growth in 2024 for the firm's portfolio, which includes several precision oncology products, such as the immunotherapies Opdivo (nivolumab) and Opdualag (nivolumab and relatlimab), the CAR T-cell therapies Breyanzi (lisocabtagene maraleucel) and Abecma (idecabtagene vicleucel), and the targeted therapies Augtyro (repotrectinib) and Krazati (adagrasib).
In 2025, Boerner expects sales of medicines in BMS's growth portfolio to comprise more than 50 percent of the firm's overall revenues. He identified five products that were key to this growth, including Breyanzi and Opdualag alongside its treatments for psychiatric disorders and cardiovascular disease.
As BMS worked to stabilize its manufacturing capacity for Breyanzi in 2024, the firm has been able to ramp up sales of the drug to meet demand. The greater manufacturing capacity helped the company scale up as Breyanzi netted two new approvals in 2024 in mantle cell lymphoma and follicular lymphoma and set up the drug to continue growing in the near term, according to Boerner.
"We've got the broadest indications set within lymphomas for a cell therapy like this, and we had good growth in 2024 with [Breyanzi]," Boerner said at the conference. "That's a function of that best-in-class profile, and we are no longer constrained in terms of product supply. We anticipate [that] this product continues to grow as we get into this year and beyond."
Opdualag, meanwhile, which is approved in the US in the first-line setting in an all-comer melanoma population and a biomarker-defined population in Europe, is expected to be "a key building block for [BMS's] immuno-oncology business into the next decade," Boerner said. The company is currently studying the drug as an adjuvant treatment for melanoma.
The firm's recent US approval of subcutaneous Opdivo in December, allowing for easier administration of the drug, is also part of its plan to "extend [the] immuno-oncology business into the next decade," he added.
"The injection is a fraction of the time associated with the infusion of IV nivolumab, and we think there's real benefit for patients who don't necessarily have to come in and see a physician every time," Boerner said, adding that a permanent billing code for this product, expected by mid-2025, will likely be an important inflection point, affecting uptake among 30 percent to 40 percent of the Opdivo business.
New precision oncology data expected in 2025 includes several readouts from early-stage trials in the first-line setting. BMS is looking ahead to results this year from the Phase II trial of Krazati in first-line KRAS G12C-mutant non-small cell lung cancer and from the Phase I study of its radiopharmaceutical therapy RYZ101 in first-line SSTR-expressing extensive-stage small cell lung cancer.
Gilead Sciences
Gilead said that it is eyeing 2026 for a potential launch of its autologous cell therapy, anitocabtagene autoleucel (anito-cel) for patients with relapsed or refractory multiple myeloma. The firm presented anito-cel data during the 2024 American Society of Hematology annual meeting in December, and in the month since, Gilead CEO Daniel O'Day said those data have been "very well received."
"The profile of this medicine is what many physicians are looking for, with … the type of efficacy that we're seeing and the type of safety profile, which is extremely important with this population of patients," he said.
Gilead is working on equipping more treatment centers with the ability to deliver its existing autologous CAR T-cell therapies and anito-cel to patients closer to home.
The drugmaker expects to end 2025 with around $10 billion in cash and cash equivalents, which it says will allow it to pursue M&A and partnerships to continue to supplement what it calls its most diverse product pipeline and portfolio.
Sarepta Therapeutics
Sarepta Therapeutics reported an estimated $638.2 million in net product revenue in the fourth quarter of 2024 and $1.79 billion during full-year 2024. This included $384.2 million in revenue from its Duchenne muscular dystrophy gene therapy Elevidys (delandistrogene moxeparvovec) in Q4 2024 and $821.0 million over the full year, per unaudited financial results.
The company's annual net product revenue exceeded its full-year 2024 guidance by more than $100 million and was a 56 percent increase compared to the previous year, said Sarepta President and CEO Doug Ingram. Sarepta reported $1.5 billion in cash, cash equivalents, restricted cash, and investments at the end of 2024.
In 2025, the company expects net product revenue in the range of $2.9 billion to $3.1 billion.
Elevidys, which the US Food and Drug Administration initially approved in 2023, is Sarepta's fourth commercialized product. Ingram touted Elevidys as "undoubtedly the most successful gene therapy launch in all of history."
He highlighted that Elevidys netted $3.1 billion in revenue in the US in the first 30 months of its launch, surpassing sales of previous gene therapy products. The gene therapy with the second most lucrative launch in the same period, according to Sarepta, was Novartis' Zolgensma (onasemnogene abeparvovec), which was approved in 2019 for spinal muscular atrophy.
Ingram expects Elevidys' uptake to continue to increase. "We have barely scratched the surface of the opportunity in front of us," Ingram said, estimating that Sarepta has reached less than 5 percent of the on-label addressable patient population for this product.
Elevidys became the first gene therapy for Duchenne to reach the market in the US after the FDA granted it accelerated approval for ambulatory patients between 4 and 5 years of age. Last year, the FDA converted that accelerated approval to a traditional approval, and expanded the indication to cover older patients, despite concerns from some agency reviewers. In the second half of 2025, Sarepta expects to submit a BLA supplement for Elevidys to cover patients younger than 4 years of age.
Ingram cited other opportunities for Elevidys including netting royalties from ex-US sales, expanding its use to patients with preexisting anti-adeno-associated virus antibodies who currently aren't eligible to receive the gene therapy, and reducing its manufacturing costs.
By 2030, Sarepta aims to have 10 approved treatments on the market, including a gene therapy for limb-girdle muscular dystrophy type 2E, for which Sarepta expects to submit a biologics license application by year-end, and small interfering RNA therapeutics that it will acquire when it completes a deal with Arrowhead Pharmaceuticals.
Arrowhead's therapeutic candidates will diversify Sarepta's pipeline to include treatments for chronic diseases and central nervous system, cardiomyopathy, and pulmonary disorders. Ingram projected that Sarepta could bring its investigational therapies for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1 to market by 2028 and 2029, respectively. "With those first two programs alone, we'll have multibillion-dollar opportunities before the end of this decade if we're successful," he said.
Recursion
Recursion provided a pipeline update following its business combination with Exscientia, completed in November 2024. The combined company now has more than 10 clinical and preclinical programs underway including the RBM39 degrader REC-1245 and REC-4881.
Recursion is evaluating REC-1245 in patients with unresectable, locally advanced, or metastatic solid tumors or relapsed or refractory lymphoma in the Phase I/II DAHLIA trial using an undisclosed set of biomarkers to study the safety, tolerability, pharmacokinetics, and preliminary activity of the drug. Salt Lake City-based Recursion has developed REC-1245 and other pipeline drugs using its artificial intelligence-based platform. REC-1245 is a molecular glue that degrades RBM39 leading to downregulation of CDK12 and other DNA damage response networks.
In the DAHLIA trial, investigators are enrolling about 85 patients with 55 allocated to the Phase I portion and between 10 patients and 30 patients to Phase II. In the Phase Ia dose escalation portion of the trial, patients will receive ascending oral doses of REC-1245. In Phase Ib, Recursion researchers will test two doses in separate arms. Researchers will track measures of safety and efficacy including adverse events, objective response rate, disease control rate, progression-free survival, and overall survival.
Recursion Cofounder and CEO Chris Gibson said the first patient in the trial was dosed in the fourth quarter of 2024. The company expects to have another update in the first half of 2026 following Phase I dose escalation.
Recursion is also testing the MEK1/2 inhibitor REC-4881 in a Phase Ib/II trial in patients with familial adenomatous polyposis (FAP), an inherited rare disease caused by a mutation in the APC gene that leads to the development of colon cancer. Patients with familial adenomatosis may begin developing colon polyps early in life which typically progresses to colon cancer before the age of 40. Gibson said patients must usually have their colons removed in their late teens or early 20s or else, "with 100 percent probability," they will progress to cancer.
In the Phase I/II TUPELO trial, Recursion scientists are studying REC-4881 in about 73 patients with FAP. To be eligible for the trial, patients must have undergone a colectomy and, for the dose finding portion of the trial, test positive for an APC mutation. Researchers are tracking pharmacokinetics, adverse events, and percent change from baseline in polyp burden, and they are determining a recommended Phase II dose. Gibson said for the first time Recursion is able to report that the initial dose used in the trial is pharmacologically active and well tolerated. Gibson said the company will share Phase Ib/II safety and early efficacy data in the first half of 2025.
Ultragenyx Pharmaceutical
Ultragenyx Pharmaceutical is preparing to launch a gene therapy for Sanfilippo syndrome type A (MPS IIIA), a regulatory application for which is currently being reviewed by the US Food and Drug Administration.
Ultragenyx is optimistic that the agency will approve a biologics license application (BLA) for UX111 it submitted in December seeking its approval as a treatment for the rare lysosomal storage disorder. If approved, the company expects to launch the product in the second half of this year, and as such, it is building a commercial supply of the gene therapy, said Ultragenyx President and CEO Emil Kakkis.
"This is an urgent indication," he said, referring to the severity of the disease. If approved, UX111 would be the company's first commercial gene therapy.
Ultragenyx acquired global rights to UX111 in 2022 from Abeona Therapeutics, which was planning to discontinue developing the drug, then called ABO-102, according to Kakkis. Under the terms of the deal, Abeona is eligible to receive tiered royalties of up to 10 percent on net sales and potential commercial milestone payments.
Ultragenyx expects to file a BLA seeking approval of another gene therapy, DTX401, in mid-2025, as a therapy for glycogen storage disease type 1a. If approved, the company anticipates launching this drug on the market as early as 2026.
Ultragenyx also provided an update on GTX-102, an antisense oligonucleotide (ASO) it is developing for Angelman syndrome. The company expects to complete enrollment in the pivotal Phase III Aspire trial, in which it is evaluating the drug in pediatric patients with a full maternal UBE3A gene deletion, in the second half of 2025. It is planning to launch another study, the Phase II/III Aurora trial, later this year to test the ASO in patients with other genotypes and in other age groups.
Kakkis said the company has multiple investigational new drug (IND) applications prepared that it hasn't submitted for approval yet as it looks to manage its expenses.
Ultragenyx estimates that it earned between $555 million and $560 million in revenue in 2024, exceeding its guidance of $530 million to $550 million and representing an increase of 29 percent year over year, according to preliminary unaudited results. That's primarily from the company's four commercial products, which are available in multiple countries.
Ultragenyx reported $745 million in estimated cash, cash equivalents, and marketable debt securities as of the end of 2024.
This year, Ultragenyx is expecting full-year revenue in the range of $640 million and $670 million. Ultragenyx has projected it will reach full-year profitability in 2027.
Beam Therapeutics
Beam Therapeutics is expanding clinical testing for BEAM-302, an investigational treatment for lung and liver disease related to alpha-1 antitrypsin deficiency (AATD), to sites outside of the US.
BEAM-302 is the company's lead genetic medicine program. It is designed to correct a mutation in the SERPINA1 gene using in vivo base editing, a type of gene editing in which a genetic mutation can be precisely corrected at the level of a single nucleotide. In BEAM-302, the base editors are delivered with lipid nanoparticles.
Beam has already begun testing BEAM-302 within a Phase I/II trial in the US. The dose-exploration and dose-expansion Phase I/II trial includes a Part A, focused on AATD-associated lung disease, and a Part B, which includes patients with AATD-associated lung and liver disease. The company will test up to four dose levels in both parts of the trial, identify a dose for a pivotal study, and assess early safety and efficacy.
More recently, regulators in the UK, New Zealand, Australia, and the Netherlands approved the company's clinical trial applications to test BEAM-302, said Beam CEO John Evans.
Beam expects to treat the first patient with BEAM-301, another liver-targeted therapeutic candidate, within a Phase I/II trial early this year. The base-editing treatment for glycogen storage disease type 1a is designed to correct a mutation in the G6PC gene. The company intends to initially focus BEAM-301's development in the US.
Beam estimates it has $850.7 million in cash, cash equivalents, and marketable securities as of the end of 2024, which it expects will fund operations into 2027, including expected commercial readiness activities for its lead sickle cell disease candidate BEAM-101. Beam expects to present updated data on BEAM-101 from the Phase I/II BEACON trial by mid-year.
Revolution Medicines
Revolution Medicines is preparing for the potential approval and commercialization of its lead drug candidate daraxonrasib, an inhibitor of the active, GTP-bound form of RAS, also called a RAS(ON) inhibitor.
CEO Mark Goldsmith said at the conference that the company is building out its commercial and medical affairs capabilities to prepare for the drug's potential approval. Daraxonrasib is currently being studied in a registrational Phase III trial in metastatic pancreatic cancer patients harboring a mutation in any KRAS G12 variant or another RAS mutation such as NRAS or HRAS.
The firm did not indicate when it expects to report Phase III data or when it may submit an application seeking approval for daraxonrasib, previously called RMC-6236. Goldsmith noted that the firm is also seeking commercial partnership opportunities to expand daraxonrasib development outside the US.
The firm's other clinical candidates, the KRAS G12C inhibitor elironrasib and the KRAS G12D inhibitor zoldonrasib, are advancing in early-stage trials as monotherapies and in combination with daraxonrasib and other treatments.
"We're now focused on executing registrational trials for daraxonrasib in previously treated pancreatic and lung cancer and on advancing this compound into a first pivotal trial in first-line pancreatic cancer," Goldsmith said. "Committing to a first pivotal trial with elironrasib or zoldonrasib is also a strategic priority."
Alongside the registrational trial of daraxonrasib in pancreatic cancer, Revolution is also studying the drug as a monotherapy in RAS-mutant advanced non-small cell lung cancer and expects to begin a registrational trial in NSCLC in the first quarter of 2025, Goldsmith said. There are also ongoing trials of daraxonrasib in other RAS-mutant solid tumors.
Also this year, Revolution expects to prioritize which combination regimens it will evaluate for all three of its clinical candidates in earlier disease settings.
Revolution is further studying the combination of daraxonrasib with elironrasib or zoldonrasib as a potential means of addressing resistance mutations in KRAS-mutant tumors. There are also ongoing combination studies of these treatments with immunotherapies, chemotherapy, and other targeted therapies in several tumor types.
"We are moving aggressively to develop RAS(ON) inhibitors in earlier lines of therapy including in first-line locally advanced or metastatic [tumors] and in adjuvant treatment settings," Goldsmith said. "A key element of the earlier disease strategy is developing optimal biologically rational drug combinations."
Finally, Revolution said it plans to move its fourth candidate into the clinic this year: RMC-5127, which targets KRAS G12V.
Pfizer
Pfizer's most important priority for 2025 is improving R&D productivity and efficiency and advancing its pipeline, according to CEO Albert Bourla. With the recent appointment of Chris Boshoff, previously the chief oncology officer at Pfizer, as CSO and president of research and development, the firm is affirming its commitment to its oncology pipeline.
"[In 2024], we integrated Seagen and doubled the size of our indications in oncology," Bourla said at the conference. "That made us the number three oncology company in terms of sales in the US."
He highlighted several oncology candidates that the firm expects to advance in the coming year, including two candidates for biomarker-defined cancers: its CDK4 inhibitor atirmociclib and the PD-L1-targeted antibody-drug conjugate (ADC) SGN-PDL1V (PF-08046054).
The CDK4 inhibitor is currently in a Phase III trial with fulvestrant in hormone receptor (HR)-positive, HER2-negative advanced or metastatic breast cancer patients who progressed on a prior CDK4/6 inhibitor.
Bourla said that PF-07220060, also called atirmociclib, has potential for "significant benefits compared to the current standard of care" treatment with CDK4/6 inhibitors, which have more toxicity.
"We have fully committed to developing [PF-07220060] in the first line," Bourla said. "CDK4/6 [inhibitors] changed the paradigm, but CDK4 is what brings the efficacy and CDK6 is what brings most of the toxicity. With the CDK4 [alone], you minimize the issues with toxicity so … the profile really is ideal for first line because it's very benign."
The advancement of atirmociclib comes as Pfizer has reported drops in quarterly sales of its CDK4/6 inhibitor Ibrance (palbociclib) in the first three quarters of 2024 (see here, here, and here).
Another candidate Bourla featured for 2025 is an ADC that Pfizer gained in its acquisition of Seagen, PF-08046054. The drug is currently being studied in a Phase I trial in PD-L1-expressing tumors, including non-small cell lung, head and neck, esophageal, ovarian, melanoma, and triple-negative breast cancer.
"PD-L1 is expressed in a lot of cancers," Bourla said. "We have seen good data even with low PD-L1 expression, but clearly, the no-brainer is to go with high PD-L1 [expressing] cancers and probably combinations with immunotherapy [for this drug]."
Kyverna Therapeutics
Kyverna Therapeutics has selected the cell therapy candidate for which it intends to file its first biologic license application (BLA) with the US Food and Drug Administration and which could be the first such therapy indicated for an autoimmune condition.
Kyverna has been advancing KYV-101, an autologous anti-CD19 CAR T-cell therapy, as its lead candidate. However, it's been under development for multiple rheumatological and neurological autoimmune conditions, including lupus nephritis, systemic sclerosis, myasthenia gravis, and stiff-person syndrome (SPS).
Kyverna is planning to submit a BLA for KYV-101 in SPS next year, said Warner Biddle, who took the helm as the company's CEO late last year. If approved, KYV-101 in SPS would be Kyverna's first commercial product, as well as the first CAR T-cell therapy to reach the market in the autoimmune setting.
"This is going to be a pivotal year for us," Biddle said, adding that Kyverna will be transitioning from an early-stage company to one focused on late-stage development and commercialization.
Kyverna is evaluating KYV-101 in an ongoing Phase II trial of SPS patients, which the company intends to use as a pivotal study. The Phase II trial, which launched last year, is currently 40 percent enrolled, and Kyverna expects to complete enrollment by mid-year and report top-line data from the clinical study in the first half of 2026.
"This really allows us to rapidly establish a footprint in the autoimmune space," Biddle said of the decision to prioritize seeking regulatory approval in SPS. "We can then pivot from there and actually build on that with two larger fast-follow indications."
Once KYV-101 is approved in SPS, Kyverna plans to submit another BLA for the cell therapy in myasthenia gravis, for which it intends to meet with regulators this year to confirm a registrational path. Then, it hopes to submit an application in lupus nephritis, which would be the company's first rheumatologic indication. From there, future indications for KYV-101 could include a range of autoimmune diseases.
Kyverna expects to report Phase II data on myasthenia gravis patients and Phase I data on lupus nephritis patients in the second half of 2025.
The company also is working on a "next-generation" cell therapy product, KYV-102, Biddle said. KYV-102, which is in preclinical development, could be manufactured more quickly and would use whole blood, so it would not require a patient to undergo apheresis. Kyverna anticipates filing an investigational new drug (IND) application for KYV-102 in the second half of the year.
Kyverna had roughly $321.6 million in cash, cash equivalents, and marketable securities as of Sept. 30. It expects its cash runway to fund the company's operations into 2027.
Merck
Merck is gearing up for the regulatory approval and launch of a subcutaneous version of its blockbuster checkpoint inhibitor Keytruda (pembrolizumab) during 2025. The drugmaker anticipates that approximately 30 percent to 40 percent of all Keytruda treatment will be subcutaneous injections within 12 to 18 months of when the new version launches. The company expects the subcutaneous formula will attract first-time recipients and patients currently receiving intravenous infusions of the drug.
The accelerated timeline for the launch of this new, more accessible version of Keytruda comes as Bristol Myers Squibb received FDA approval for the subcutaneous injection Opdivo Qvantig (nivolumab and hyaluronidase) at the end of last year. Merck is also trying to launch subcutaneous Keytruda ahead of the blockbuster's upcoming loss of exclusivity in 2028, when several key patents are set to expire.
Offering a subcutaneous version of Keytruda, which Merck plans to sell at what it calls a "competitive price point," is intended to help keep patients opting for Keytruda over generic versions of the intravenous PD-1 inhibitor. "Our goal is to maintain share [of the PD-1 inhibitor market], and we think we can do that at a value that's going to be meaningful to protect the revenue that we think we need to try to have post-[loss of exclusivity]," Merck CEO Rob Davis said.
Davis also said that by the mid-2030s, even as Keytruda loses exclusivity, Merck's oncology portfolio is expected to be worth $25 billion in revenue opportunities due to the company's portfolio diversification efforts. For instance, within the last few years, the firm acquired Harpoon Therapeutics to develop a pipeline of T-cell engagers and partnered with Daiichi Sankyo to develop a series of antibody-drug conjugates.
"It's not just about Keytruda," said Dean Li, the president of Merck Research Laboratories, highlighting that the firm is continuing to combine the blockbuster immunotherapy with other new agents and moving it into early treatment settings. "All of those are exciting to us."
Exact Sciences
Exact Sciences CEO Kevin Conroy said in his presentation that the company is prepared to launch three new long-awaited cancer tests in 2025 in its "most productive year" yet.
The next-generation version of its Cologuard test Cologuard Plus is expected to launch early in the second quarter of 2025, while its molecular residual disease test Oncodetect is forecast for launch sometime in the second quarter. Its multi-cancer screening test Cancerguard will likely launch in the second half of 2025. Conroy added that the firm expects to announce new data for its blood-based colon cancer test around the midpoint of 2025, as well.
Conroy noted that Cologuard Plus received pricing from the US Centers for Medicare and Medicaid Services last year and has already nabbed coverage from some large commercial payors. He added that the test will launch into the Medicare population first and then become available to privately insured patients. It will take approximately 18 months to transition customers from Cologuard to Cologuard Plus, Conroy said, while CFO Aaron Bloomer said the launch will "lead to growth acceleration for the next several years, as well as margin expansion."
Oncodetect is expected to launch with CMS coverage for colorectal cancer, and the firm plans to expand to other types of cancers from there, Conroy said. The test currently looks at between 50 and 200 mutations but in the future will look for 2,000 to 5,000 mutations, he added. That ability to look for thousands of mutations confers "an advantage that over time will allow us to get more people tested with Oncodetect," he said. Conroy also cited the existing relationships Exact Sciences has with most health systems in the US, thanks to its Oncotype DX assay, as a way to potentially increase patient access to Oncodetect.
Cancerguard, meantime, will launch "at a reasonable price point" for patients to pay out of pocket, he said. Exact Sciences believes that the price, which Conroy did not disclose, will be accessible to patients and is one that "payors will be willing to pay because of the health economics." He said that the firm does not expect commercial payor coverage for Cancerguard in 2025, while Bloomer added that Exact does not expect material revenue contribution from the test in 2025.
Regarding the Q4 and full-year 2024 preliminary financial results released on Sunday, Bloomer said that growth in the quarter was driven by Cologuard. He noted that orders impacted by the hurricanes in Florida in Q3 came back "a bit better than anticipated" at the end of Q4.
Bloomer added that 2024 was a "record year of profitability" as well as for free cash flow for the company. The priority for capital allocation will continue to be reinvesting in the business and the firm will maintain its disciplined approach to M&A, he said.
Guardant Health
Guardant Health co-CEO Helmy Eltoukhy said that the firm's 2024 year-over-year growth in clinical test volumes was largely driven by its Guardant360 test. The company saw continuing improvements in Guardant360's average selling price, reaching its long-term goal of an ASP of $3,000 per test roughly four years ahead of schedule, thanks to an increase in the Medicare payment rate to $5,000.
Guardant CFO Michael Bell added that there is "still room to grow" on the ASP for Guardant360 and noted that some coverage gaps remain among commercial payors. Some insurers cover only the companion diagnostic version of the test while others only cover limited cancer types, so the firm aims to expand that coverage.
Eltoukhy noted that the test was transitioned to Guardant's Smart Liquid Biopsy platform in July, which expanded the number of genes included and improved the test's sensitivity. The firm plans to "continually add new applications to the test," he said. As it continues to test more patients and collect more data, it can "generate an almost unlimited number of differentiated applications" for the platform, he added.
The company has also seen "excellent uptake" of the firm's Guardant360 TissueNext test thus far and has plans to launch the Guardant360 Tissue comprehensive genomic profiling test using the Smart platform technology this year.
Regarding its minimal residual disease business, Eltoukhy said the company has submitted data to CMS's MolDx program for reimbursement of Guardant Reveal in the colorectal cancer surveillance MRD setting and hopes for reimbursement in early 2025. It has also submitted data for publication supporting potential Medicare reimbursement for the test's use in breast cancer and therapy monitoring and has ongoing clinical validity studies for additional cancers.
Guardant co-CEO AmirAli Talasaz noted that the firm is ramping up its commercial infrastructure for the Guardant Shield test, which received FDA clearance for colorectal cancer screening last year. The company performed approximately 6,400 Shield tests in Q4 2024, driven largely by Medicare beneficiaries, he said. The test is currently priced at $920 per test but the company is seeking ADLT designation that will raise the price to $1,495 per test.
The firm is also expecting data from the second version of Shield in 2025 and could have version 2 on the market this year. The second version is more analytically sensitive and has an optimized algorithm, but the actual assay is the same, Talasaz said. Guardant doesn't know yet if the improved analytical sensitivity translates to better clinical performance, but it expects sensitivity for stage I colorectal cancer detection to increase.
Talasaz added that the firm's therapy selection business is free cash flow positive, the MRD business is expected to reach breakeven by 2026, and the company expects to reach free cash flow breakeven in 2028.
Guardant expects oncology clinical volume growth of more than 20 percent across all products this year, Talasaz said.