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BeOne Medicines Establishes Standard for Long-Term Disease Control in CLL with BRUKINSA 78-Month Data at ASCO 2026

On May 29, 2026 BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported the treatment paradigm in chronic lymphocytic leukemia (CLL) at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. With extensive long-term follow-up, the SEQUOIA study of BRUKINSA (zanubrutinib) reinforces its role as the foundational BTK inhibitor, showing sustained disease control over years of therapy. These findings are further supported by real-world evidence across three large analyses encompassing more than 250,000 patients, underscoring consistent effectiveness and safety in clinical practice. Additionally, BEQALZI (sonrotoclax), which was recently approved by the U.S. Food and Drug Administration, and its development in combination with BRUKINSA (ZS) highlight the potential for next-generation, time-limited treatment approaches in CLL.

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Amit Agarwal, M.D., Ph.D., Chief Medical Officer, Hematology, BeOne Medicines, said:
"CLL is a disease patients live with for years, and the real measure of a therapy is how it performs over the long arc of treatment. Our data at ASCO (Free ASCO Whitepaper) show that BRUKINSA continues to deliver sustained disease control, which can give physicians and patients confidence to stay the course. Additionally, robust, real-world analyses reinforce its role as a best-in-class BTK inhibitor, with data favoring BRUKINSA over other BTK inhibitors across several efficacy and safety endpoints. With BRUKINSA as the foundation, we are building a broad, differentiated hematology franchise designed to push the field further, including our ZS combination, which achieved deep responses and unprecedented rates of uMRD, and emerging approaches like our BTK degrader, tacabrutideg. Together, these foundational therapies reflect our commitment to redefining what patients should expect from therapy both today and in the future."

78-month SEQUOIA data highlight the long-term impact of first-line treatment choice in CLL (Poster Presentation: 544; June 1, 2026, 9:00 AM-12:00 PM CDT)
SEQUOIA now provides the longest reported follow-up for a next-generation BTK inhibitor in first-line CLL, enabling a deeper understanding of how treatment outcomes evolve over time. After a median follow-up of 84.01 months (range, 0.0-101.5), BRUKINSA continued to show benefit over bendamustine-rituximab (BR) in patients with treatment-naive CLL/SLL, with progression-free survival (PFS) outcomes that are unprecedented among BTK inhibitors. Key highlights include:

78-month PFS: 71.8% (95% CI, 65.3-77.3) for BRUKINSA vs. 31.0% (95% CI, 24.3-37.9) for BR
78-month COVID-adjusted PFS: 74.6% (95% CI, 68.1-79.9) for BRUKINSA vs. 31.4% (95% CI, 24.7-38.4) for BR
PFS for patients with unmutated IGHV: 70.4% (95% CI, 61.0-77.9) for BRUKINSA vs. 17.4% (95% CI, 9.6-27.1) for BR
PFS for patients with mutated IGHV: 81.8% (95% CI: 72.2-88.4) for BRUKINSA and 45.1% (95% CI: 34.4-55.2) for BR
78-month PFS2: 81.3% (95% CI, 75.6-85.8) for BRUKINSA vs. 74.4% (95% CI, 67.8-79.8) for BR
78-month COVID-adjusted PFS2: 84.7% (95% CI, 79.2-88.8) for BRUKINSA and 76.4% (95% CI, 69.9-81.7) for BR
Of the BRUKINSA-treated patients who progressed (26/241), half received subsequent therapy with BCL2 inhibitor-based salvage therapy and 69.2% had not progressed after more than 3 years of follow-up.
Time to next treatment (TTNT) favored BRUKINSA over BR (HR, 0.24; 95% CI, 0.16-0.35; P<.0001)
Safety: consistent with the results of prior BRUKINSA studies with no new safety signals.
PFS2 captures outcomes beyond first disease progression, measuring time to disease progression on subsequent therapy or death. In CLL, this endpoint provides important insight into how first-line treatment impacts long-term disease control across multiple lines of therapy.

Constantine Tam, M.B.B.S., M.D., Head of Lymphoma Service at Alfred Health and Professor of Haematology at Monash University, said:
"In an indolent disease like CLL, many patients value maintaining disease control over the course of their life, not just in the first year or two of treatment. The continued long-term follow-up from SEQUOIA shows that zanubrutinib can deliver sustained disease control. This is the kind of evidence that allows clinicians and patients to make first-line decisions with real confidence about what lies ahead."

Real-world efficacy and safety data consistently underscore foundational BRUKINSA as the best-in-class BTKi for TN CLL (Poster Presentations: 545, 543 and 540; June 1, 2026, 9:00 AM-12:00 PM CDT)
In addition to the update from SEQUOIA, BeOne will present data from new analyses of large and robust datasets, which demonstrate consistent and significant real-world benefits of using BRUKINSA over other BTK inhibitors. Key highlights include:

In a real-world analysis of 10,523 Medicare patients, who were diagnosed with CLL/SLL and received frontline treatment with a BTK inhibitor, patients treated with BRUKINSA had a statistically significantly lower risk of death, advancing to next line, or discontinuing treatment, than those on ibrutinib or acalabrutinib. Similar results were observed across age subgroups. (Poster Presentation: 545)
In a separate real-world analysis of Komodo database claims from 16,788 patients with treatment-naïve CLL, BRUKINSA had a longer TTNT (unadjusted HR, 0.88; 95% CI, 0.79-0.97; P=.009) and overall survival (OS; HR, 0.72; 95% CI, 0.62-0.82; P<.001). (Poster Presentation: 543)
A retrospective analysis of 233,362 newly diagnosed CLL patients who initiated treatment with a BTK inhibitor, the atrial fibrillation rate within 1 year was lowest for BRUKINSA at 11% and 13% for acalabrutinib and 16% for ibrutinib (overall P<.0001). (Poster Presentation: 540)
Deep, rapid responses with BRUKINSA plus sonrotoclax (ZS) point to the future of time-limited treatment in CLL, including high-risk disease (Poster Presentation: 541; June 1, 2026, 9:00 AM-12:00 PM CDT)
In the Phase 1/1b study in patients with treatment-naïve CLL/SLL (median follow-up of ~34 months), the all-oral combination of BRUKINSA and next-generation BCL2 inhibitor sonrotoclax (ZS) demonstrated unprecedented rates and kinetics of undetectable minimal residual disease (uMRD), including in patients with high-risk cytogenetics. Key highlights include:

Overall response rate (ORR): 100%, with complete responses in 59.5% of patients
Best uMRD4 rate 98.8%
No patient that achieved uMRD4 reverted to uMRD positivity.
Best uMRD in patients with TP53 mutation/del(17p): 92.9% across 2 dose levels
Median time from combination start to uMRD4: 4.5 months
No disease progression events observed at the recommended Phase 2 dose of 320mg, including patients who electively discontinued therapy
Safety: consistent with previously reported BRUKINSA and sonrotoclax combination studies.
These data will also be presented as encore presentations at the 2026 European Hematology Association (EHA) (Free EHA Whitepaper) Congress (June 11–14, Stockholm) along with more than 30 other data sets from BeOne.

About BRUKINSA (zanubrutinib)
BRUKINSA is an orally available, small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared with other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues.

With the broadest label globally, BRUKINSA is the foundational BTK inhibitor and is the only BTK inhibitor to demonstrate superiority to another BTK inhibitor in a Phase 3 study. It is also the only BTK inhibitor to provide the flexibility of once or twice daily dosing.

The global BRUKINSA clinical development program includes more than 8,000 patients enrolled in over 30 countries and regions across more than 45 trials. BRUKINSA is approved in 80 markets in at least one indication, and more than 290,000 patients have been treated globally.

About BEQALZI (sonrotoclax)
BEQALZI (sonrotoclax) is a foundational, next-generation and potentially best-in-class B-cell lymphoma 2 (BCL2) inhibitor with a unique pharmacokinetic and pharmacodynamic profile. Preclinical and clinical studies in early drug development have shown that sonrotoclax is a highly potent and specific BCL2 inhibitor with a short half-life and no drug accumulation. Sonrotoclax has shown promising clinical activity across a range of B-cell malignancies, including chronic lymphocytic leukemia (CLL), and is in development as a monotherapy and in combination with other therapeutics, including zanubrutinib. To date, more than 2,500 patients have been enrolled across the broad sonrotoclax global development program.

BEQALZI is approved by the U.S. Food and Drug Administration (FDA) and China’s National Medical Products Administration for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL), after at least two lines of systemic therapy, including a BTK inhibitor. It is also approved in China for adult patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) who have previously received at least one systemic therapy, including a BTK inhibitor.

About Tacabrutideg (BGB-16673)
Tacabrutideg is a foundational and potential first-in-class and best-in-class orally available Bruton’s tyrosine kinase (BTK) degrader. With 1,200+ patients dosed to date in an extensive global clinical development program, tacabrutideg is the most advanced BTK degrader in the clinic. This program includes three randomized Phase 3 trials in R/R CLL, including the head-to-head Phase 3 trial versus pirtobrutinib, which began enrolling in Q4 2025. Originating from BeOne’s chimeric degradation activation compound (CDAC) platform, tacabrutideg is designed to promote the degradation, or breakdown, of both wildtype and mutant forms of BTK, including those that commonly result in resistance to BTK inhibitors in patients who experience progressive disease.

The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to tacabrutideg for the treatment of adult patients with relapsed or refractory (R/R) chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL), and adult patients with R/R mantle cell lymphoma (MCL). Additionally, the European Medicines Agency (EMA) granted tacabrutideg PRIority MEdicines (PRIME) designation for the treatment of patients with Waldenstrom’s macroglobulinemia (WM) previously treated with a BTK inhibitor.

Select Important Safety Information for BRUKINSA
Serious adverse reactions, including fatal events, have occurred with BRUKINSA, including hemorrhage, infections, cytopenias, second primary malignancies, cardiac arrhythmias, and hepatotoxicity (including drug-induced liver injury).

In the pooled safety population (N=1729), the most common adverse reactions (≥30%), including laboratory abnormalities, in patients who received BRUKINSA were neutrophil count decreased (51%), platelet count decreased (41%), upper respiratory tract infection (38%), hemorrhage (32%), and musculoskeletal pain (31%).

Please see full U.S. Prescribing Information including U.S. Patient Information.

Select Important Safety Information for BEQALZI (sonrotoclax)
Serious and sometimes fatal adverse reactions have occurred with BEQALZI, including tumor lysis syndrome (TLS), serious infections, neutropenia, and embryo-fetal toxicity. BEQALZI is contraindicated with strong CYP3A inhibitors at initiation and during the ramp-up phase due to the potential for an increased risk of tumor lysis syndrome.

In the safety population (N=115), tumor lysis syndrome occurred in 7% of patients who followed the recommended dose ramp-up. Serious infections occurred in 14% of patients, and Grade 3 or 4 infections occurred in 17% (fatal: 2.6%), with pneumonia (10%) being the most common Grade 3 or greater infection. Grade 3 or 4 decreases in neutrophils occurred in 18% of patients (Grade 4: 6%), and febrile neutropenia occurred in 1.7% of all patients. The most common adverse reactions (≥15%) were pneumonia (16%) and fatigue (16%). The most common Grade 3–4 laboratory abnormalities (≥15%) were decreases in lymphocytes (29%) and neutrophils (18%).

Please see full Prescribing Information.

The information provided in this press release is intended for a global audience. Product indications vary by region.

(Press release, BeOne Medicines, MAY 29, 2026, View Source [SID1234666234])

Natera Announces Collaboration with Diakonos Oncology for Signatera™ in Refractory Melanoma

On May 29, 2026 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and precision medicine, reported a new collaboration with Diakonos Oncology Corp., a clinical-stage biotechnology company developing immunotherapies to treat challenging and aggressive cancers. As part of the collaboration, Signatera will be used to longitudinally assess molecular response in patients with refractory melanoma enrolled in Diakonos’ DOC-RM Phase I/II investigational immunotherapy trial.

The DOC-RM trial, which began enrollment in May, is evaluating DOC1021 (dubodencel), a first-in-class, personalized dendritic cell investigational therapy that recently received Fast Track designation by the U.S. Food and Drug Administration (FDA) in unresectable or metastatic cutaneous melanoma. With Signatera, Natera will conduct analyses of circulating tumor DNA (ctDNA) at multiple timepoints during and following treatment.

Refractory melanoma is an area of significant unmet need. Although immunotherapy has transformed the treatment landscape for advanced melanoma, many patients either do not respond or eventually develop resistance, underscoring the need for novel therapeutic approaches. Because radiographic response assessment can be challenging in immunotherapy-treated patients, serial ctDNA monitoring may provide earlier insight into molecular response and disease dynamics during treatment.1

"With an FDA Fast Track designation in hand, DOC1021’s path forward in refractory melanoma will benefit from early, high-quality evidence of activity," said Jay Hartenbach, president and COO of Diakonos Oncology. "Natera’s Signatera test is the most trusted tumor-informed MRD platform in oncology, making them a natural partner to help evaluate molecular response in a population where imaging often lags the biology."

"Signatera is uniquely positioned to help biopharma partners evaluate molecular response throughout the course of therapy, and we are thrilled to partner with Diakonos on this exciting program," said Eric Matthews, general manager, BioPharma, Natera. "By assessing MRD status across multiple timepoints, this collaboration has the potential to provide deep insight into treatment response dynamics and support future development efforts for patients with difficult-to-treat cancers."

(Press release, Diakonos Oncology, MAY 29, 2026, View Source [SID1234666233])

PanTher Completes Dose Escalation Phase in Non-Metastatic Pancreatic Cancer Phase 1b Trial and Appoints Tim Clackson to the Board of Directors

On May 29, 2026 PanTher Therapeutics ("PanTher" or the "Company"), a clinical-stage company redefining early cancer treatment with therapeutic implants administered directly at the tumor site, reported that it has concluded the dose escalation portion of its Phase 1b clinical trial of PTM-101 in pancreatic ductal adenocarcinoma (PDAC). PanTher has identified the PTM-101 400mg paclitaxel-containing implant as the recommended Phase 2 dose (RP2D) in combination with neoadjuvant standard of care chemotherapy. In addition, PanTher announced the appointment of senior life sciences executive Dr. Tim Clackson to its Board of Directors. This news reflects a pivotal stage in the company’s development to achieve the promise of the PTM-101 technology.

The ongoing Phase 1b study builds on PanTher’s first-in-human trial that demonstrated the safety of PTM-101 at 100 mg and reported promising tumor shrinkage, with reduction in overall tumor volume of up to 70%. The current trial is an open-label, multicenter, single-arm study designed to evaluate the safety, tolerability, and pharmacokinetics (PK) of PTM-101 in patients with newly diagnosed, treatment naïve, non-metastatic pancreatic cancer. In the dose-finding phase, subjects were treated at higher doses of paclitaxel (200 mg and 400 mg) with no systemic paclitaxel detected, no evidence of significant implant-related toxicities, and no additive toxicities with standard of care chemotherapy. Plasma pharmacokinetic data show no evidence of systemic exposure to paclitaxel with levels below quantifiable limits. Based on these data from a total of 12 patients, the Study Safety Committee endorsed the 400mg dose as the RP2D for further evaluation. This dose represents 4x the Maximum Tolerable Dose (MTD) of paclitaxel when delivered systemically, underscoring the potential benefit of localized therapy.

Approximately 50% of PDAC patients present with non-metastatic disease at diagnosis, when timely anti-tumor treatment and surgery still offer the potential for cure. Yet multi-agent systemic chemotherapy is often limited by toxicity. PTM-101 is a proprietary paclitaxel implant administered at the tumor surface during a standard tumor staging laparoscopy, immediately after diagnosis, providing ~6 weeks of sustained high-dose therapy directly to the tumor, with no systemic exposure. As an add-on therapy, PTM-101 integrates seamlessly with neoadjuvant standard of care FOLFIRINOX by adding a fourth chemotherapy agent while creating a new window for early intervention and improved outcomes.

The ongoing Phase 1b study (NCT06673017) is assessing safety, tolerability, and anti-tumor activity of PTM-101 when combined with standard of care neoadjuvant chemotherapy (FOLFIRINOX) in subjects with borderline resectable or locally advanced PDAC. PanTher is now enrolling patients in the dose expansion phase, which will enroll approximately 15 additional patients at sites across the United States to further characterize the safety and efficacy of PTM-101. For more information on the trial, please visit View Source

PanTher also announced today the appointment of Dr. Tim Clackson to its Board of Directors. Dr. Clackson brings more than three decades of experience building oncology companies to PanTher’s board, and his appointment reflects PanTher’s commitment to assembling a world-class team to support the advancement of PTM-101 and the Company’s broader pipeline.

"We are thrilled to welcome Tim to PanTher’s Board of Directors," said Laura Indolfi, Ph.D., Chief Executive Officer and Co-founder of PanTher Therapeutics. "His experience in developing and commercializing oncology therapies will be invaluable as we continue to build on the momentum of our Phase 1b clinical program and work to bring PTM-101 to patients in need."

Laura continued, "For patients with localized pancreatic cancer, every treatment decision made at diagnosis can impact the future of that patient and the development of their diagnosis. PTM-101 offers the potential to integrate directly into the neoadjuvant standard treatment paradigm from the outset, delivering additional anti-tumor activity without compounding systemic side effects. It is our aim with PTM-101 to meaningfully improve clinical outcomes for patients, alongside the current standard of care, to drive deeper and longer treatment responses before a tumor has the opportunity to become metastatic."

"PanTher is taking a highly differentiated and promising approach to tackling one of the most challenging cancers. By intervening right after diagnosis, with localized, high dose and sustained therapy, PTM-101 has the potential to transform PDAC outcomes before metastasis," said Tim Clackson, Ph.D. "As a simple add-on therapy that integrates seamlessly with existing workflows and therapies, it has the potential for wide adoption. I look forward to working with Laura and the PanTher team to help bring PTM-101, and a broader pipeline of localized medicines, to patients in need."

PTM-101 is the lead product candidate within PanTher’s pipeline of implantable medicines designed to directly address hard-to-treat solid tumors. PanTher is additionally developing polymeric drug formulations for the treatment of a range of other solid tumor types.

The company expects to share topline data from the Phase 1b study of PTM-101 in the first half of 2027.

About PTM-101

PanTher’s most advanced investigational product candidate, PTM-101, is an absorbable thin film formulation of paclitaxel for non-metastatic pancreatic cancer. PTM-101 is designed to deliver continuous, long-lasting, high-dose chemotherapy to the tumor with little to no systemic exposure. The product, laparoscopically implanted at the tumor site, easily integrates with common minimally invasive procedures used in staging pancreatic cancer. PTM-101 is currently being evaluated in a Phase 1b clinical trial (NCT06673017) with support from the Cancer Prevention & Research Institute of Texas (CPRIT) DP220066.

(Press release, PanTher Therapeutics, MAY 29, 2026, View Source [SID1234666232])

Seven-Year Analysis from Pfizer’s LORBRENA CROWN Trial Shows Longest Progression-Free Survival Reported to Date in Advanced Non-Small Cell Lung Cancer

On May 29, 2026 Pfizer Inc. (NYSE: PFE) reported unprecedented seven-year follow-up results from the Phase 3 CROWN trial evaluating LORBRENA (lorlatinib, a third-generation ALK inhibitor, available in Europe under the brand name LORVIQUA) versus XALKORI (crizotinib) in people with previously untreated, anaplastic lymphoma kinase (ALK)-positive advanced or metastatic non-small cell lung cancer (NSCLC).

At seven years, patients treated with LORBRENA had a 55% likelihood of remaining alive without disease progression (95% Confidence Interval [CI], 46-63) compared to 3% (95% CI, 1-8) in the XALKORI treatment arm. Further, an updated analysis at seven years of median follow-up showed that investigator-assessed median progression-free survival (PFS) had not been reached with LORBRENA, with an estimated Hazard Ratio (HR) of 0.19 (95% CI, 0.13-0.26), representing an 81% reduction in the risk of disease progression or death compared to XALKORI. Full results from the analysis will be presented today in an oral presentation at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Abstract #8502) and simultaneously published in Annals of Oncology.

"The updated results from the CROWN trial show unprecedented long-term clinical benefit, with estimates indicating the majority of patients treated with LORBRENA remained alive and progression-free at seven years. While definitive conclusions cannot be drawn across studies, this appears to represent the longest observed progression-free survival reported to date in metastatic or advanced lung cancer," said Jeff Legos, Chief Oncology Officer, Pfizer. "These findings further showcase Pfizer’s world-class discovery expertise and our commitment to developing breakthroughs that help improve care for people with advanced NSCLC."

Lung cancer is the leading cause of cancer-related deaths worldwide,1 and nearly 230,000 new cases are expected in the U.S. in 2026.2 NSCLC accounts for approximately 75-80% of lung cancers,2,3 with ALK-positive tumors occurring in about 3-5% of NSCLC cases.4 Approximately 25-40% of people with ALK-positive advanced NSCLC may develop brain metastases within two years from initial diagnosis, which are associated with poorer survival and can profoundly affect cognitive function and quality of life.5

LORBRENA was specifically designed and developed by Pfizer to inhibit tumor mutations that drive resistance to other ALK inhibitors and to penetrate the blood-brain barrier. Results from this seven-year follow-up showed that LORBRENA prevented and controlled brain metastases, with a 94% reduction in the risk of developing intracranial (IC) progression (HR, 0.06; 95% CI, 0.03-0.12) and no new IC progression events occurring after the first 30 months. The median time to IC progression was not reached (95% CI, NR-NR) with LORBRENA and was 16.4 months (12.7-21.9) with XALKORI. At the time of analysis, 44% of patients in the CROWN trial were still receiving LORBRENA compared to 3% of patients receiving XALKORI.

"These seven-year outcomes from the CROWN study are remarkable not only for their durability of tumor response but for what they represent—a fundamental shift in what clinicians and patients might reasonably expect from treatment for advanced-stage NSCLC," said Tony Shu-Kam Mok, BBS, Endowed Professor, Li Shu Fan Medical Foundation, Chairman of the Dept. of Clinical Oncology, Chinese University of Hong Kong, and Principal Investigator of the CROWN trial. "Observing this level of long-term benefit with a once-daily oral therapy, both in terms of sustained progression-free survival and prevention of brain metastases, would have been difficult to imagine when we first developed ALK-specific targeted therapy a decade ago and underscores the significance of these results for the lung cancer community."

"Behind every clinical trial is a person continuing to live their life—raising children, pursuing careers, making memories—without their cancer progressing," said Kenneth Culver, M.D., Director of Research and Clinical Affairs at the non-profit organization ALK Positive. "These seven-year results provide compelling evidence that long-term disease control is possible, and we applaud Pfizer’s dedication to advancing treatments that are changing what it means to live with ALK-positive lung cancer."

The safety profiles of LORBRENA and XALKORI were consistent with previous findings, with no new safety signals observed. In this analysis, the most frequent (≥20%) adverse events (AEs) of interest reported in patients treated with LORBRENA included edema, weight gain, peripheral neuropathy, cognitive effects, mood effects, diarrhea, dyspnea, arthralgia, hypertension, headache, cough, pyrexia, hypercholesterolemia, and hypertriglyceridemia. All-cause grade 3/4 AEs occurred in 77% of patients with LORBRENA and in 57% of patients with XALKORI. Treatment-related AEs led to permanent treatment discontinuation in 5% and 6% of patients in the LORBRENA and XALKORI arms, respectively. No new permanent discontinuations due to treatment-related AEs occurred after the first 26 months with LORBRENA.

Pfizer is continuing its commitment to help non-scientists understand the latest findings with the development of abstract plain language summaries (APLS) for company-sponsored research being presented at ASCO (Free ASCO Whitepaper), which are written in non-technical language. Those interested in learning more can visit www.Pfizer.com/apls to access the summaries.

About the CROWN Trial

CROWN is a Phase 3, randomized, open-label, parallel two-arm trial in which 296 people with previously untreated ALK-positive advanced NSCLC were randomized 1:1 to receive LORBRENA monotherapy (n=149) or XALKORI monotherapy (n=147). The primary endpoint of the CROWN trial was PFS based on Blinded Independent Central Review (BICR) with a key secondary endpoint of overall survival (OS) for which follow-up is ongoing and results will be reported in the future. Additional secondary endpoints include PFS based on investigator’s assessment, objective response rate (ORR), intracranial objective response rate (IC-ORR), and safety. Given that median PFS was not reached after three years of follow-up, and then later after five years of follow-up, unplanned post-hoc analyses were executed with the intent to further quantify long-term outcomes based on investigator tumor assessment from this trial. The present analysis was performed at a clinically meaningful landmark follow-up of seven years.

About LORBRENA (lorlatinib)

LORBRENA is approved in the U.S. for the treatment of adults with metastatic NSCLC whose tumors are ALK-positive as detected by an FDA-approved test. In addition to the U.S., LORBRENA has received approval in more than 80 countries, including Australia, Canada, China, European Union, Japan, and South Korea.

Please see Full Prescribing Information for LORBRENA (lorlatinib) or visit View Source

IMPORTANT LORBRENA (lorlatinib) SAFETY INFORMATION FROM THE U.S. PRESCRIBING INFORMATION

Contraindications: LORBRENA is contraindicated in patients taking strong CYP3A inducers, due to the potential for serious hepatotoxicity.

Risk of Serious Hepatotoxicity with Concomitant Use of Strong CYP3A Inducers: Severe hepatotoxicity occurred in 10 of 12 healthy subjects receiving a single dose of LORBRENA with multiple daily doses of rifampin, a strong CYP3A inducer. Grade 4 ALT or AST elevations occurred in 50% of subjects, Grade 3 in 33% of subjects, and Grade 2 in 8% of subjects. ALT or AST elevations occurred within 3 days and returned to within normal limits after a median of 15 days (7 to 34 days); median time to recovery in subjects with Grade 3 or 4 or Grade 2 ALT or AST elevations was 18 days and 7 days, respectively. LORBRENA is contraindicated in patients taking strong CYP3A inducers. Discontinue strong CYP3A inducers for 3 plasma half-lives of the strong CYP3A inducer prior to initiating LORBRENA.

Central Nervous System (CNS) Effects: A broad spectrum of CNS effects can occur; overall, CNS effects occurred in 52% of the 476 patients receiving LORBRENA. These included seizures (1.9%, sometimes in conjunction with other neurologic findings), psychotic effects (7%; 0.6% severe [Grade 3 or 4]), and changes in cognitive function (28%; 2.9% severe), mood (including suicidal ideation) (21%; 1.7% severe), speech (11%; 0.6% severe), mental status (1.3%; 1.1% severe), and sleep (12%). Median time to first onset of any CNS effect was 1.4 months (1 day to 3.4 years). Overall, 2.1% and 10% of patients required permanent or temporary discontinuation of LORBRENA, respectively, for a CNS effect; 8% required dose reduction. Withhold and resume at same or reduced dose or permanently discontinue based on severity.

Hyperlipidemia: Increases in serum cholesterol and triglycerides can occur. Grade 3 or 4 elevations in total cholesterol occurred in 18% and Grade 3 or 4 elevations in triglycerides occurred in 19% of the 476 patients who received LORBRENA. Median time to onset was 15 days for both hypercholesterolemia and hypertriglyceridemia. Approximately 4% and 7% of patients required temporary discontinuation and 1% and 3% of patients required dose reduction of LORBRENA for elevations in cholesterol and in triglycerides in Study B7461001 and Study B7461006, respectively. Eighty-three percent of patients required initiation of lipid-lowering medications, with a median time to onset of start of such medications of 17 days. Initiate or increase the dose of lipid-lowering agents in patients with hyperlipidemia. Monitor serum cholesterol and triglycerides before initiating LORBRENA, 1 and 2 months after initiating LORBRENA, and periodically thereafter. Withhold and resume at same dose for the first occurrence; resume at same or reduced dose of LORBRENA for recurrence based on severity.

Atrioventricular (AV) Block: PR interval prolongation and AV block can occur. In 476 patients who received LORBRENA at a dose of 100 mg orally once daily and who had a baseline electrocardiography (ECG), 1.9% experienced AV block and 0.2% experienced Grade 3 AV block and underwent pacemaker placement. Monitor ECG prior to initiating LORBRENA and periodically thereafter. Withhold and resume at reduced or same dose in patients who undergo pacemaker placement. Permanently discontinue for recurrence in patients without a pacemaker.

Interstitial Lung Disease (ILD)/Pneumonitis: Severe or life-threatening pulmonary adverse reactions consistent with ILD/pneumonitis can occur. ILD/pneumonitis occurred in 1.9% of patients, including Grade 3 or 4 ILD/pneumonitis in 0.6% of patients. Four patients (0.8%) discontinued LORBRENA for ILD/pneumonitis. Promptly investigate for ILD/pneumonitis in any patient who presents with worsening of respiratory symptoms indicative of ILD/pneumonitis (e.g., dyspnea, cough, and fever). Immediately withhold LORBRENA in patients with suspected ILD/pneumonitis. Permanently discontinue LORBRENA for treatment related ILD/pneumonitis of any severity.

Hypertension: Hypertension can occur. Hypertension occurred in 13% of patients, including Grade 3 or 4 in 6% of patients. Median time to onset of hypertension was 6.4 months (1 day to 2.8 years), and 2.3% of patients temporarily discontinued LORBRENA for hypertension. Control blood pressure prior to initiating LORBRENA. Monitor blood pressure after 2 weeks and at least monthly thereafter. Withhold and resume at reduced dose or permanently discontinue based on severity.

Hyperglycemia: Hyperglycemia can occur. Hyperglycemia occurred in 9% of patients, including Grade 3 or 4 in 3.2% of patients. Median time to onset of hyperglycemia was 4.8 months (1 day to 2.9 years), and 0.8% of patients temporarily discontinued LORBRENA for hyperglycemia. Assess fasting serum glucose prior to initiating LORBRENA and monitor periodically thereafter. Withhold and resume at reduced dose or permanently discontinue based on severity.

Embryo-fetal Toxicity: LORBRENA can cause fetal harm. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use an effective non-hormonal method of contraception, since LORBRENA can render hormonal contraceptives ineffective, during treatment with LORBRENA and for at least 6 months after the final dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with LORBRENA and for 3 months after the final dose.

Adverse Reactions: In the pooled safety population of 476 patients who received 100 mg LORBRENA once daily, the most frequent (≥ 20%) adverse reactions were edema (56%), peripheral neuropathy (44%), weight gain (31%), cognitive effects (28%), fatigue (27%), dyspnea (27%), arthralgia (24%), diarrhea (23%), mood effects (21%), and cough (21%). The most frequent (≥ 20%) Grade 3-4 laboratory abnormalities in patients receiving LORBRENA were hypercholesterolemia (21%) and hypertriglyceridemia (21%).

In previously untreated patients, serious adverse reactions occurred in 34% of the 149 patients treated with LORBRENA; the most frequently reported serious adverse reactions were pneumonia (4.7%), dyspnea (2.7%), respiratory failure (2.7%), cognitive effects (2.0%), and pyrexia (2.0%). Fatal adverse reactions occurred in 3.4% of patients and included pneumonia (0.7%), respiratory failure (0.7%), cardiac failure acute (0.7%), pulmonary embolism (0.7%), and sudden death (0.7%). In the Phase 1/2 study, serious adverse reactions occurred in 32% of the 295 patients; the most frequently reported serious adverse reactions were pneumonia (3.4%), dyspnea (2.7%), pyrexia (2%), mental status changes (1.4%), and respiratory failure (1.4%). Fatal adverse reactions occurred in 2.7% of patients and included pneumonia (0.7%), myocardial infarction (0.7%), acute pulmonary edema (0.3%), embolism (0.3%), peripheral artery occlusion (0.3%), and respiratory distress (0.3%).

Drug Interactions: LORBRENA is contraindicated in patients taking strong CYP3A inducers. Avoid concomitant use with moderate CYP3A inducers, strong CYP3A inhibitors, and fluconazole. If concomitant use of moderate CYP3A inducers cannot be avoided, increase the LORBRENA dose as recommended. If concomitant use with a strong CYP3A inhibitor or fluconazole cannot be avoided, reduce the LORBRENA dose as recommended. Avoid concomitant use of LORBRENA with CYP3A substrates and P-gp substrates, which may reduce the efficacy of these substrates.

Lactation: Because of the potential for serious adverse reactions in breastfed infants, instruct women not to breastfeed during treatment with LORBRENA and for 7 days after the final dose.

Hepatic Impairment: No dose adjustment is recommended for patients with mild (total bilirubin ≤ ULN with AST > ULN or total bilirubin >1 to 1.5 × ULN with any AST) to moderate (Child-Pugh B) hepatic impairment. In patients with severe hepatic impairment (Child-Pugh C), the recommended dosage of LORBRENA is 50 mg orally once daily.

Renal Impairment: In patients with CLcr 15 to <30mL/min (estimated by Cockcroft-Gault), the recommended dosage of LORBRENA is 75 mg orally once daily. No dose adjustment is recommended for patients with CLcr 30 to 89 mL/min (estimated by Cockcroft-Gault).

INDICATION

LORBRENA (lorlatinib) is indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA-approved test.

About XALKORI (crizotinib)

XALKORI is a tyrosine kinase inhibitor (TKI) indicated for the treatment of patients with metastatic NSCLC whose tumors are ALK- or ROS1-positive as detected by an FDA-approved test. XALKORI has received approval for patients with ALK-positive NSCLC in more than 90 countries including Australia, Canada, China, Japan, South Korea and the European Union. XALKORI is also approved for ROS1-positive NSCLC in more than 60 countries.

The full prescribing information for XALKORI can be found here.

IMPORTANT XALKORI (crizotinib) SAFETY INFORMATION FROM THE U.S. PRESCRIBING INFORMATION

Hepatotoxicity: Drug-induced hepatotoxicity with fatal outcome occurred in 0.1% of patients treated with XALKORI across clinical trials (n=1719). Increased transaminases generally occurred within the first 2 months. Monitor liver function tests, including ALT, AST, and total bilirubin, every 2 weeks during the first 2 months of treatment, then once a month, and as clinically indicated, with more frequent repeat testing for increased liver transaminases, alkaline phosphatase, or total bilirubin in patients who develop increased transaminases. Permanently discontinue for ALT/AST elevation >3 times ULN with concurrent total bilirubin elevation >1.5 times ULN (in the absence of cholestasis or hemolysis); otherwise, temporarily suspend and dose-reduce XALKORI as indicated.

Interstitial Lung Disease/Pneumonitis: Severe, life-threatening, or fatal interstitial lung disease (ILD)/pneumonitis can occur. Across clinical trials (n=1719), 2.9% of XALKORI-treated patients had any grade ILD, 1.0% had Grade 3/4, and 0.5% had fatal ILD. ILD generally occurred within 3 months after initiation of treatment. Monitor for pulmonary symptoms indicative of ILD/pneumonitis. Exclude other potential causes and permanently discontinue XALKORI in patients with drug-related ILD/pneumonitis.

QT Interval Prolongation: QTc prolongation can occur. Across clinical trials (n=1616), 2.1% of patients had QTcF (corrected QT by the Fridericia method) ≥500 ms and 5% of 1582 patients had an increase from baseline QTcF ≥60 ms by automated machine-read evaluation of ECGs. Avoid use in patients with congenital long QT syndrome. Monitor ECGs and electrolytes in patients with congestive heart failure, bradyarrhythmias, electrolyte abnormalities, or who are taking medications that prolong the QT interval. Permanently discontinue XALKORI in patients who develop QTc >500 ms or ≥60 ms change from baseline with Torsade de pointes, polymorphic ventricular tachycardia, or signs/symptoms of serious arrhythmia. Withhold XALKORI in patients who develop QTc >500 ms on at least 2 separate ECGs until recovery to a QTc ≤480 ms, then resume at next lower dosage.

Bradycardia: Symptomatic bradycardia can occur. Across clinical trials, bradycardia occurred in 13% of patients treated with XALKORI (n=1719). Avoid use in combination with other medications known to cause bradycardia. Monitor heart rate and blood pressure regularly. If bradycardia occurs, re-evaluate for the use of concomitant medications known to cause bradycardia. Permanently discontinue for life-threatening bradycardia due to XALKORI; however, if associated with concomitant medications known to cause bradycardia or hypotension, hold XALKORI until recovery to asymptomatic bradycardia or to a heart rate of ≥60 bpm. If concomitant medications can be adjusted or discontinued, restart XALKORI at 250 mg once daily with frequent monitoring.

Severe Visual Loss: Across clinical trials, the incidence of Grade 4 visual field defect with vision loss was 0.2% of 1719 patients. Discontinue XALKORI in patients with new onset of severe visual loss (best corrected vision less than 20/200 in one or both eyes). Perform an ophthalmological evaluation. There is insufficient information to characterize the risks of resumption of XALKORI in patients with a severe visual loss; a decision to resume should consider the potential benefits to the patient.

Vision Disorders: Most commonly visual impairment, photopsia, blurred vision or vitreous floaters, occurred in 63% of 1719 patients. The majority (95%) of these patients had Grade 1 visual adverse reactions. 0.8% of patients had Grade 3 and 0.2% had Grade 4 visual impairment. The majority of patients on the XALKORI arms in Studies 1 and 2 (>50%) reported visual disturbances which occurred at a frequency of 4-7 days each week, lasted up to 1 minute, and had mild or no impact on daily activities.

Embryo-Fetal Toxicity: XALKORI can cause fetal harm when administered to a pregnant woman. Advise of the potential risk to the fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment and for at least 45 days (females) or 90 days (males) respectively, following the final dose of XALKORI.

ROS1-positive Metastatic NSCLC: Safety was evaluated in 50 patients with ROS1-positive metastatic NSCLC from a single-arm study and was generally consistent with the safety profile of XALKORI evaluated in patients with ALK-positive metastatic NSCLC. Vision disorders occurred in 92% of patients in the ROS1 study; 90% of patients had Grade 1 vision disorders and 2% had Grade 2.

Adverse Reactions: Safety was evaluated in a phase 3 study in previously untreated patients with ALK-positive metastatic NSCLC randomized to XALKORI (n=171) or chemotherapy (n=169). Serious adverse events were reported in 34% of patients treated with XALKORI, the most frequent were dyspnea (4.1%) and pulmonary embolism (2.9%). Fatal adverse events in XALKORI-treated patients occurred in 2.3% of patients, consisting of septic shock, acute respiratory failure, and diabetic ketoacidosis. Common adverse reactions (all grades) occurring in ≥25% and more commonly (≥5%) in patients treated with XALKORI vs chemotherapy were vision disorder (71% vs 10%), diarrhea (61% vs 13%), edema (49% vs 12%), vomiting (46% vs 36%), constipation (43% vs 30%), upper respiratory infection (32% vs 12%), dysgeusia (26% vs 5%), and abdominal pain (26% vs 12%). Grade 3/4 reactions occurring at a ≥2% higher incidence with XALKORI vs chemotherapy were QT prolongation (2% vs 0%), esophagitis (2% vs 0%), and constipation (2% vs 0%). In patients treated with XALKORI vs chemotherapy, the following occurred: elevation of ALT (any grade [79% vs 33%] or Grade 3/4 [15% vs 2%]); elevation of AST (any grade [66% vs 28%] or Grade 3/4 [8% vs 1%]); neutropenia (any grade [52% vs 59%] or Grade 3/4 [11% vs 16%]); lymphopenia (any grade [48% vs 53%] or Grade 3/4 [7% vs 13%]); hypophosphatemia (any grade [32% vs 21%] or Grade 3/4 [10% vs 6%]). In patients treated with XALKORI vs chemotherapy, renal cysts occurred (5% vs 1%). Nausea (56%), decreased appetite (30%), fatigue (29%), and neuropathy (21%) also occurred in patients taking XALKORI.

Drug Interactions: Use caution with concomitant use of moderate CYP3A inhibitors. Avoid grapefruit or grapefruit juice which may increase plasma concentrations of crizotinib. Avoid concomitant use of strong CYP3A inducers and inhibitors. Avoid concomitant use of CYP3A substrates where minimal concentration changes may lead to serious adverse reactions. If concomitant use of XALKORI is unavoidable, decrease the CYP3A substrate dosage in accordance with approved product labeling.

Lactation: Because of the potential for adverse reactions in breastfed children, advise women not to breastfeed during treatment with XALKORI and for 45 days after the final dose.

Hepatic Impairment: Crizotinib concentrations increased in patients with pre-existing moderate (any AST and total bilirubin >1.5x ULN and ≤3x ULN) or severe (any AST and total bilirubin >3x ULN) hepatic impairment. Reduce XALKORI dosage in patients with moderate or severe hepatic impairment. The recommended dose of XALKORI in patients with pre-existing moderate hepatic impairment is 200 mg orally twice daily or with pre-existing severe hepatic impairment is 250 mg orally once daily.

Renal Impairment: Decreases in estimated glomerular filtration rate occurred in patients treated with XALKORI. Administer XALKORI at a starting dose of 250 mg taken orally once daily in patients with severe renal impairment (CLcr <30 mL/min) not requiring dialysis.

(Press release, Pfizer, MAY 29, 2026, View Source [SID1234666231])

Tempus Receives FDA Approval for Tumor Only xT CDx, Enabling Migration of its Entire DNA Solid Tumor Portfolio

On May 29, 2026 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine, reported that the U.S. Food and Drug Administration (FDA) has granted approval for a tumor-only indication for its xT CDx next-generation sequencing platform. With this expanded label, Tempus is the first laboratory to hold FDA companion diagnostic (CDx) approval for both tumor-only and tumor-normal comprehensive genomic profiling.

Tempus xT CDx is a 648-gene tissue-based assay intended for molecular profiling of all solid tumor malignancies. It also serves as a companion diagnostic to identify colorectal cancer patients who may benefit from targeted therapies, specifically Erbitux (cetuximab) and Vectibix (panitumumab).

While xT CDx previously required a patient’s matched normal sample, this regulatory milestone allows the test to run as a tumor-only assay when a matched normal specimen (blood or saliva) is not viable or available. This approval paves the way for Tempus to migrate its entire DNA solid tumor portfolio to FDA-approved assays priced under its current ADLT (Advanced Diagnostic Laboratory Test) pricing.

"This approval marks a milestone in both our regulatory and reimbursement strategy, as this allows the migration of our entire solid tumor DNA portfolio to be under unified ADLT pricing," said Jim Rogers, Chief Financial Officer at Tempus. "As we have previously highlighted, we expect an estimated $200 ASP benefit beginning in 2027 as a result of this approval."

"Our goal is to support clinicians with advanced genomic profiling options," said Kate Sasser, PhD, Chief Scientific Officer at Tempus. "With FDA approval for both tumor-only and tumor-normal comprehensive genomic profiling, Tempus xT CDx provides flexibility for a range of clinical scenarios. While tumor-normal matched sequencing remains an important approach, we recognize that a matched sample is not always available, and now, patients can still benefit from an FDA-approved test that can help inform treatment decisions."

xT CDx is a qualitative Next Generation Sequencing (NGS)-based in vitro diagnostic device intended for use in the detection of substitutions (single nucleotide variants (SNVs) and multi-nucleotide variants (MNVs)) and insertion and deletion alterations (INDELs) in 648 genes in patients with previously diagnosed solid malignant neoplasms. The assay uses DNA isolated from Formalin-Fixed Paraffin Embedded (FFPE) tumor tissue specimens and, when available, patient-matched blood or saliva specimens. Additionally, the device detects microsatellite instability (MSI) status based on a genomic signature from the tumor specimen only. The test is intended as a companion diagnostic (CDx) to identify patients who may benefit from treatment with the targeted therapies listed in the Companion Diagnostic Indications table in accordance with the approved therapeutic product labeling. Additionally, xT CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with previously diagnosed solid malignant neoplasms. Genomic findings other than those listed in the Companion Diagnostic Indications table are not prescriptive or conclusive for labeled use of any specific therapeutic product.

Click to view the complete xT CDx label, including companion diagnostic indications and important risk information.

(Press release, Tempus, MAY 29, 2026, View Source [SID1234666230])