On May 14, 2025 Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa" or the "Company"), a clinical-stage biopharmaceutical company developing innovative medicines for breast cancer, reported full results from the Phase 2 Endocrine Optimization Pilot (EOP) sub‑study within the I‑SPY 2 TRIAL evaluating low‑dose oral (Z)‑endoxifen (10 mg daily) as a neoadjuvant treatment in 20 women with stage II/III estrogen‑receptor–positive (ER+), HER2‑negative breast cancer (Press release, Atossa Therapeutics, MAY 14, 2025, View Source [SID1234653098]).

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Key Findings:

Primary feasibility goal surpassed – 95 percent of participants (19/20) completed at least 75 percent of planned dosing, far exceeding the predefined 75 percent threshold.
Early anti‑proliferative activity – Median Ki‑67 fell from 10.5 percent at baseline to 5 percent by Week 3 (prior to ovarian suppression); 65 percent of patients achieved Ki‑67 ≤ 10 percent at that time point, and suppression was maintained at surgery.
Robust imaging response – Median functional tumor volume (FTV) measurement (performed at baseline, week 3, week 12, and at surgery) decreased 77.7 percent (range 9.0 cc → 1.2 cc) from baseline to surgery, and the longest tumor diameter from baseline to preoperative MRI was reduced by 36.8 percent.
Well‑tolerated safety profile – Adverse events were predominantly Grade 1; vasomotor symptoms (hot flushes) and fatigue were most common. Only three Grade 3 events (two skin infections, one post‑procedural infection) occurred in a single patient and were deemed unrelated to study drug; no Grade 4 or Grade 5 events were reported.
Pathologic Findings:
No participants achieved a pathologic complete response (pCR), and residual cancer burden (RCB) classes skewed toward RCB‑II/III, indicating moderate to extensive residual disease. This result was anticipated based on earlier window‑of‑opportunity (neoadjuvant) studies that showed significant pathologic clearance typically requires higher systemic exposures to (Z)-endoxifen (> 500 ng/mL) to enable inhibition of PKCβ1 in addition to ERα. The 10 mg dose in this pilot was intentionally selected to establish tolerability and early biologic activity in the endocrine‑naïve setting; therefore, limited pCR rates at this dose are consistent with prior dose–response observations.

"These results show that even at a low capsule strength (Z)‑endoxifen is bioactive, producing rapid Ki‑67 suppression and meaningful tumor shrinkage, while remaining highly tolerable. The study strengthens our scientific hypothesis that dual targeting of ERα and PKCβ1 is key to inducing a pathological response," said Steven Quay, M.D., Ph.D., President and Chief Executive Officer of Atossa. "Importantly, it paves the way for our ongoing I‑SPY 2 cohorts evaluating higher, potentially PKCβ1‑engaging doses of (Z)‑endoxifen alone and in combination with the CDK4/6 inhibitor abemaciclib, where we aim to translate early biomarker gains into deeper pathologic responses."

Atossa is actively enrolling participants into an additional I‑SPY 2 cohort testing (Z)‑endoxifen at a 40 mg daily dose and in combination with abemaciclib, with and without ovarian function suppression. Top‑line data from these arms are expected beginning in 2026.

About (Z)-Endoxifen
(Z)-endoxifen is a highly potent Selective Estrogen Receptor Modulator (SERM) with demonstrated ability to inhibit—and potentially degrade—estrogen receptors. It has shown activity even in tumors that have developed resistance to other endocrine therapies. Beyond its anti-estrogenic properties, (Z)-endoxifen also targets protein kinase C beta 1 (PKCβ1), an oncogenic signaling protein, at clinically achievable blood levels. Importantly, (Z)-endoxifen seems to deliver comparable or superior bone-protective effects relative to tamoxifen, while exhibiting minimal or no endometrial proliferative activity—addressing key limitations of current standard-of-care therapies.

Atossa is developing a proprietary oral formulation of (Z)-endoxifen that is enteric-coated to bypass stomach acid, which would otherwise convert the active (Z)-isomer to its inactive (E)-form. This innovation ensures optimal bioavailability and therapeutic integrity. Clinical studies have shown Atossa’s (Z)-endoxifen to be well tolerated in both healthy women and those with breast cancer.

Atossa is prioritizing the development of (Z)-endoxifen for the treatment of metastatic breast cancer, where novel therapeutic options are urgently needed. The compound is currently being evaluated in three Phase 2 trials: one in women with ductal carcinoma in situ (DCIS) and two in women with ER+/HER2- breast cancer, including the EVANGELINE trial and the combination I-SPY study. Atossa’s (Z)-endoxifen program is supported by a growing global intellectual property portfolio, including three recently issued U.S. patents and numerous pending applications worldwide.

On May 14, 2025 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines, reported its first quarter financial results and provided a business update (Press release, Cyclacel, MAY 14, 2025, View Source [SID1234653165]).

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"As part of the Company’s efforts to reduce operating costs it has determined to focus on the development of the plogosertib ("plogo") clinical program only. Accordingly, on March 10, 2025, the Company repurchased certain assets related to plogo from Cyclacel Limited for approximately $0.3 million in cash, to allow us to continue our efforts on developing an alternative salt, oral formulation of plogosertib with improved bioavailability," said Datuk Dr. Doris Wong, Chief Executive Officer. "Cyclacel Limited’s other drug development program, fadraciclib, is being marketed for sale by that entity’s liquidator in the U.K. pursuant to creditors voluntary liquidation of Cyclacel Limited announced in the London Gazette on January 31, 2025."

"Upon the commencement of the liquidation of Cyclacel Limited, the Company lost operational and strategic control over Cyclacel Limited and thus its financial results have been deconsolidated from the Company effective January 31, 2025; as a result, the Company anticipates a significant decrease to research and development expenses for the year ended December 31, 2025 as we focus on plogo and have no further expenditures related to fadraciclib," said Kiu Cu Seng, Chief Financial Officer. "The deconsolidated of our former subsidiary, Cyclacel Limited, resulted in a gain on deconsolidation, and thus an increase in stockholders’ equity of approximately $5.0 million, which we have reported in the Company’s Form 10-Q for the three months ended March 31, 2025."

Due to the current difficult economic environment and our lack of funding to implement our business plan, we have begun to analyze strategic alternatives available to the Company to continue as a going concern. Such alternatives include raising additional debt or equity financing or consummating a merger or acquisition with a partner that may involve a change in our business plan. As a result, the Company entered into an Exchange Agreement with FITTERS Diversified Berhad on April 6, 2205, to exchange all of the ordinary shares owned by FITTERS in exchange for approximately 19.99% of the Company’s common stock to acquire FITTERS’ wholly-owned subsidiary, Fitters Sdn. Bhd., a Malaysia-based private limited company specializing in supplying, and trading various protective and fire safety equipment.

Financial Highlights

As of March 31, 2025, cash and cash equivalents totaled $3.5 million, compared to $3.2 million as of December 31, 2024.

Net cash used in operating activities was $3.3 million for the three months ended March 31, 2025,. The Company estimates that its current cash resources will fund planned programs into the second quarter of 2025.

Research and development expenses were $0.8 million for the three months ended March 31, 2025, as compared to $2.8 million for the same period in 2024. Expenditure for the transcriptional regulation program ceased as a result of the Company’s UK subsidiary, Cyclacel Limited, being liquidated on January 24, 2025. Research and development expenses relating to plogosertib decreased by $0.6 million relative to the respective comparative period whilst we continue to explore and develop an alternative salt, oral formulation with improved bioavailability.

General and administrative expenses increased by approximately $2.6 million from $1.6 million for the three months ended March 31, 2024 to $4.2 million for the three months ended March 31, 2025, due to several one-time costs associated with the change of control of the Company; primarily stock compensation expense of $1.4 million, D&O insurance costs of $0.7 million, compensation expense of $0.3 million and legal costs of $0.1 million.

Total other (expense) income, net, for the three months and year ended March 31, 2025, were $5.0 million, compared to $0.1 million for the same period of the previous year, primarily due to a $5.0 million gain on deconsolidation of the UK subsidiary.

United Kingdom research & development tax credits for the three months ended March 31, 2024, were $1.4 million. There were no research and development tax credits for the three months ended March 31, 2025, following the liquidation of the UK subsidiary and the subsequent loss of eligibility for recoverable tax credits as a result thereof.

Net loss for the three months ended March 31, 2025, was $0.1 million (including stock-based compensation expense of $1.6 million), compared to $2.9 million (including stock-based compensation expense of $0.2 million) for the same period in 2024.

On May 14, 2025 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta (γδ) T cell therapies for cancer and autoimmune diseases, reported new preclinical data from its INB-619 program at the 2025 American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting (Press release, In8bio, MAY 14, 2025, View Source [SID1234653060]). The data shows that INB-619, a CD-19 targeted γδ TCE, successfully eliminated disease-causing B cells in blood samples from patients with active Systemic Lupus Erythematosus (SLE, or Lupus).

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These findings highlight the potential of INB-619 as a novel treatment option for autoimmune diseases, where harmful B cells secrete antibodies that drive inflammation and tissue damage. Unlike traditional TCEs that target CD3 and can often trigger severe side effects, INB-619 harnesses the unique properties of γδ T cells to selectively clear B cells while minimizing inflammatory responses.

William Ho, Chief Executive Officer of IN8bio, commented, "Our findings show that INB-619 not only eliminates pathogenic B cells, but also expands γδ T cell levels, which are often reduced in patients with chronic disease. This represents a fundamentally different approach to immune modulation – one that could potentially lead to safer, more effective treatments for autoimmune diseases and beyond."

Highlights from the Preclinical Data:

INB-619 demonstrated complete, targeted depletion of B cells in lupus patient-derived samples.
IN8bio’s γδ TCE successfully expands both Vδ1+ and Vδ2+ subtypes of γδ T cells, which are critical for targeting both circulating and tissue-resident B cells to potentially achieve deeper B cell depletion.
Unlike conventional CD3-based TCEs, INB-619 did not trigger significant release of inflammatory cytokines such as IL-6, which are commonly associated with serious toxicities, including CRS.
The study reinforces the potential of γδ T cell engagers as a safer, more precise immunotherapy platform for autoimmune diseases.
With these encouraging results, IN8bio continues to explore potential partnerships and additional autoimmune and cancer indications where γδ T cell biology and B cell targeting intersect.

On May 14, 2025 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported that multiple abstracts showcasing clinical data for Revuforj (revumenib) and Niktimvo (axatilimab-csfr) were accepted for presentation at the 30th European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress Meeting being held June 12-15, 2025, in Milan, Italy (Press release, Syndax, MAY 14, 2025, View Source [SID1234653080]).

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"The data being presented at EHA (Free EHA Whitepaper) showcase the potential for revumenib and axatilimab to transform the treatment paradigm for patients with acute leukemia and chronic GVHD, respectively," said Nick Botwood, MBBS, Head of Research & Development and Chief Medical Officer at Syndax. "We and our clinical collaborators are particularly pleased to share updated data from the BEAT AML frontline trial of revumenib in combination with venetoclax and azacitidine in mNPM1 and KMT2Ar AML. We anticipate that the oral presentation of these encouraging results will drive even greater physician interest in the ongoing EVOLVE-2 frontline global registration trial of this same combination."

Key data being presented at EHA (Free EHA Whitepaper) 2025 include:

An oral presentation reporting updated results from the Phase 1 BEAT AML trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed mutant NPM1 (mNPM1) or KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML) patients aged 60 years or older.
A poster presentation reporting data from the 77 patients with relapsed or refractory (R/R) mNPM1 AML who met the efficacy evaluable criteria in the Phase 2 cohort of the AUGMENT-101 trial of revumenib. The poster will expand upon the topline data that was reported from this population in December 2024.
A poster presentation reporting outcomes in patients with R/R NUP98r acute leukemias in the Phase 1 portion of the AUGMENT-101 trial.
A poster presentation reporting the dynamics of overall and organ-specific responses in the AGAVE-201 trial of axatilimab in chronic graft-versus-host disease (GVHD).
The accepted abstracts listed below are now available online at the EHA (Free EHA Whitepaper) conference website. Copies of the oral and poster presentations will be made available in the ‘Publications & Meetings Presentations’ section of the Syndax website after the presentations occur.

Presentations During EHA (Free EHA Whitepaper) 2025 (all times in CEST):

Revumenib

Abstract Titles Presentation Details
Azacitidine, Venetoclax, and Revumenib for Newly Diagnosed Older Adults with Acute Myeloid Leukemia (AML) and NPM1 mutation or KMT2A rearrangement: Updated Results from the Beat AML Consortium Oral presentation
Abstract #: S138
Thursday, June 12
Session 5:00-6:15 pm
Patients With Relapsed or Refractory (R/R) Nucleophosmin 1–Mutated (NPM1m) Acute Myeloid Leukemia (AML): Updated Results From the Phase 2 AUGMENT-101 Study Poster presentation (Session 2)
Abstract #: PS1467
Saturday, June 14 at 6:30-7:30 pm
Updated Results and Longer Follow-Up From the AUGMENT-101 Phase 2 Study of Revumenib in All Patients With Relapsed or Refractory (R/R) KMT2Ar Acute Leukemia Poster presentation (Session 2)
Abstract #: PS1473
Saturday, June 14 at 6:30-7:30 pm
Encore presentation
Revumenib Activity in Patients with NUP98r Acute Leukemia: Results from the AUGMENT-101 Phase 1 Study Poster presentation (Session 2)
Abstract #: PS1501
Saturday, June 14 at 6:30-7:30 pm
Trial in Progress: A Phase 1 Study of Revumenib + Intensive Chemotherapy in Patients With Newly Diagnosed Acute Myeloid Leukemia Harboring Genetic Alterations in KMT2A, NPM1, or NUP98 (SNDX-5613-0708) Publication only*
Abstract: PB2576
Treatment Patterns in Patients with Relapsed or Refractory NPM1 Mutated (NPM1m) Acute Myeloid Leukemia (AML) in the United States (US): Real-world Data Analysis Publication only*
Abstract: PB2513
* Abstracts will be published in the online Abstract Book, a supplement of HemaSphere (EHA’s official journal), EHA (Free EHA Whitepaper) Library and Congress platform.

Axatilimab

Abstract Titles Presentation Details
The Effects of Prior Lines of Therapy on Clinical Outcomes for Patients With Chronic Graft-Versus-Host Disease Receiving Axatilimab: A Post Hoc Analysis of AGAVE-201 Poster presentation (Session 2)
Abstract #: PS2029
Saturday, June 14 at 6:30-7:30 pm
Encore presentation
Dynamics of Overall and Organ-Specific Responses to Axatilimab in Chronic Graft-Versus-Host Disease: Analysis From the AGAVE-201 Study Poster presentation (Session 1)
Abstract #: PF1035
Friday, June 13 at 6:30-7:30 pm
Encore presentation
Trial in Progress: A Randomized, Open-Label, Phase 3 Study of Axatilimab Versus Best Available Therapy in Patients With Chronic Graft-Versus-Host Disease After ≥2 Prior Lines of Systemic Therapy Poster presentation (Session 1)
Abstract #: PF1090
Friday, June 13 at 6:30-7:30 pm
Correlations of Clinician-Reported Responses With Other Response Measures in Patients With Chronic Graft-Versus-Host Disease: An Analysis From the AGAVE-201 Trial Poster presentation (Session 1)
Abstract #: PF1041
Friday, June 13 at 6:30-7:30 pm
Encore presentation

About Revuforj (revumenib)

Revuforj (revumenib) is an oral, first-in-class, selective menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older.

Revumenib is in development for the treatment of R/R acute myeloid leukemia (AML) with a nucleophosmin 1 mutation (mNPM1). Positive pivotal data from the AUGMENT-101 trial in this population with revumenib as a monotherapy were recently published and the Company submitted a supplemental NDA for revumenib in R/R mNPM1 AML in April 2025. Additionally, multiple trials of revumenib in combination with standard-of-care agents in mNPM1 AML or KMT2A-rearranged acute leukemia are ongoing or planned across the treatment landscape, including in newly diagnosed patients.

Revumenib was previously granted Orphan Drug Designation for the treatment of AML, ALL and acute leukemias of ambiguous lineage (ALAL) by the U.S. FDA and for the treatment of AML by the European Commission. The U.S. FDA also granted Fast Track designation to revumenib for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation and Breakthrough Therapy Designation for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.

About Niktimvo (axatilimab-csfr)

Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).

In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications.

Axatilimab is being studied in frontline combination trials in chronic GVHD – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774) are underway. Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

Niktimvo is a trademark of Incyte.

All other trademarks are the property of their respective owners.

Revuforj (revumenib)

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation syndrome: Revuforj can cause fatal or life-threatening differentiation syndrome (DS). Symptoms of DS, including those seen in patients treated with Revuforj, include fever, dyspnea, hypoxia, peripheral edema, pleuropericardial effusion, acute renal failure, and/or hypotension. In clinical trials, DS occurred in 39 (29%) of 135 patients treated with Revuforj. DS was Grade 3 or 4 in 13% of patients and fatal in one. The median time to onset was 10 days (range 3-41 days). Some patients experienced more than 1 DS event. Treatment interruption was required for 7% of patients, and treatment was withdrawn for 1%.

Reduce the white blood cell count to less than 25 Gi/L prior to starting Revuforj. If DS is suspected, immediately initiate treatment with systemic corticosteroids (e.g., dexamethasone 10-mg IV every 12 hours in adults or dexamethasone 0.25-mg/kg/dose IV every 12 hours in pediatric patients weighing less than 40 kg) for a minimum of 3 days and until resolution of signs and symptoms. Institute supportive measures and hemodynamic monitoring until improvement. Interrupt Revuforj if severe signs and/or symptoms persist for more than 48 hours after initiation of systemic corticosteroids, or earlier if life-threatening symptoms occur such as pulmonary symptoms requiring ventilator support. Restart steroids promptly if DS recurs after tapering corticosteroids.

QTc interval prolongation: In the clinical trials, QTc interval prolongation was reported as an adverse reaction in 39 (29%) of 135 patients treated with Revuforj. QTc interval prolongation was Grade 3 in 12% of patients. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 8%, and the increase from baseline QTcF was greater than 60 msec in 18%. Revuforj dose reduction was required for 5% of patients due to QTc interval prolongation. QTc prolongation occurred in 16% of the 31 patients less than 17 years old, 33% of the 88 patients 17 years to less than 65 years old, and in 50% of the 16 patients 65 years or older.

Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to treatment with Revuforj. Perform an electrocardiogram (ECG) prior to initiation of Revuforj, and do not initiate Revuforj in patients with QTcF >450 msec. Perform an ECG at least once weekly for the first 4 weeks and at least monthly thereafter. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation.

Interrupt Revuforj if QTcF increases >480 msec and <500 msec, and restart Revuforj at the same dose twice daily after the QTcF interval returns to ≤480 msec
Interrupt Revuforj if QTcF increases >500 msec or by >60 msec from baseline, and restart Revuforj twice daily at the lower-dose level after the QTcF interval returns to ≤480 msec
Permanently discontinue Revuforj in patients with ventricular arrhythmias and in those who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia.
Embryo-fetal toxicity: Revuforj can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with Revuforj and for 4 months after the last dose of Revuforj.

ADVERSE REACTIONS

Fatal adverse reactions occurred in 4 (3%) patients who received Revuforj, including 2 with differentiation syndrome, 1 with hemorrhage, and 1 with sudden death.

Serious adverse reactions were reported in 99 (73%) patients. The most frequent serious adverse reactions (≥5%) were infection (24%), febrile neutropenia (19%), bacterial infection (17%), differentiation syndrome (12%), hemorrhage (9%), and thrombosis (5%).

The most common adverse reactions (≥20%) including laboratory abnormalities, were hemorrhage (53%), nausea (51%), phosphate increased (50%), musculoskeletal pain (42%), infection (41%), aspartate aminotransferase increased (37%), febrile neutropenia (35%), alanine aminotransferase increased (33%), parathyroid hormone intact increased (33%), bacterial infection (31%), diarrhea (30%), differentiation syndrome (29%), electrocardiogram QT prolonged (29%), phosphate decreased (25%), triglycerides increased (25%), potassium decreased (24%), decreased appetite (24%), constipation (23%), edema (23%), viral infection (23%), fatigue (22%), and alkaline phosphatase increased (21%).

DRUG INTERACTIONS

Drug interactions can occur when Revuforj is concomitantly used with:

Strong CYP3A4 inhibitors: reduce Revuforj dose
Strong or moderate CYP3A4 inducers: avoid concomitant use with Revuforj
QTc-prolonging drugs: avoid concomitant use with Revuforj. If concomitant use is unavoidable, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold Revuforj if the QTc interval is >480 msec. Restart Revuforj after the QTc interval returns to ≤480 msec.
SPECIFIC POPULATIONS

Lactation: advise lactating women not to breastfeed during treatment with Revuforj and for 1 week after the last dose.

Pregnancy and testing: Revuforj can cause fetal harm when administered to a pregnant woman. Verify pregnancy status in females of reproductive potential within 7 days prior to initiating Revuforj.

Pediatric: monitor bone growth and development in pediatric patients.

Geriatric: compared to younger patients, the incidences of QTc prolongation and edema were higher in patients 65 years and older.

Infertility: based on findings in animals, Revuforj may impair fertility. The effects on fertility were reversible.

To report SUSPECTED ADVERSE REACTIONS, contact Syndax Pharmaceuticals at 1-888-539-3REV or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Full Prescribing Information, including BOXED WARNING.

Niktimvo (axatilimab-csfr)

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS
Infusion-Related Reactions
Niktimvo (axatilimab-csfr) can cause infusion-related reactions. Infusion-related reactions, including hypersensitivity reactions, occurred in 18% of patients who received Niktimvo in the clinical trial (AGAVE-201), with Grade 3 or 4 reactions in 1.3%.

Premedicate with an antihistamine and an antipyretic for patients who have previously experienced an infusion-related reaction to Niktimvo. Monitor patients for signs and symptoms of infusion-related reactions, including fever, chills, rash, flushing, dyspnea, and hypertension. Interrupt or slow the rate of infusion or permanently discontinue Niktimvo based on severity of the reaction.

Embryo-Fetal Toxicity
Based on its mechanism of action, Niktimvo may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose.

ADVERSE REACTIONS
Serious adverse reactions occurred in 44% of patients who received Niktimvo (N=79). Serious adverse reactions in >2 patients included infection (pathogen unspecified) (14%), viral infection (14%) and respiratory failure (5.1%). Permanent discontinuation of Niktimvo due to an adverse reaction occurred in 10% of patients and dose reduction due to adverse reaction occurred in 8% of patients. Dose interruptions due to an adverse reaction occurred in 44% of patients. The adverse reactions leading to dose interruption in >2 patients were viral infection, infection (pathogen unspecified), bacterial infection, musculoskeletal pain, and pyrexia.

The most common (≥15%) adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea.

Clinically relevant adverse reactions in <10% of patients who received Niktimvo included:

Eye disorders: periorbital edema
Skin and subcutaneous skin disorders: pruritus
Vascular disorders: hypertension
Immunogenicity: Anti-Drug Antibody–Associated Adverse Reactions
Across treatment arms in patients with cGVHD who received Niktimvo in clinical trials, among the patients who developed anti-drug antibodies (ADAs), hypersensitivity reactions occurred in 26% (13/50) of patients with neutralizing antibodies (NAb) and in 4% (2/45) of those without NAb.

USE IN SPECIFIC POPULATIONS
Lactation
Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment and for 30 days after the last dose of Niktimvo.

Females and Males of Reproductive Potential
Pregnancy Testing
Verify pregnancy status in females of reproductive potential prior to initiating Niktimvo.

Contraception
Females
Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose of Niktimvo.

DOSAGE AND ADMINISTRATION
Dosage Modifications for Adverse Reactions
Monitor aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP), creatine phosphokinase (CPK), amylase, and lipase prior to the start of Niktimvo therapy, every 2 weeks for the first month, and every 1 to 2 months thereafter until abnormalities are resolved. See Table 1 in the Prescribing Information for more recommendations.

Please see the full Prescribing Information for Niktimvo.

On May 14, 2025 BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that will change its name to BeOne Medicines Ltd., reported it will share data across a range of hematologic malignancies at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress in Milan, Italy, June 12–15 (Press release, BeiGene, MAY 14, 2025, View Source [SID1234653099]). BeiGene has 31 abstracts accepted at EHA (Free EHA Whitepaper) 2025, with four selected for oral presentations, featuring data from its best-in-class Bruton’s tyrosine kinase (BTK) inhibitor BRUKINSA (zanubrutinib) and its investigational pipeline assets – a next-generation BCL2 inhibitor, sonrotoclax, and BTK protein degrader, BGB-16673. These data reflect BeiGene’s vision to redefine standards of care in hematology through next-generation science and patient-focused innovation.

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"With three cornerstone hematology assets – BRUKINSA, sonrotoclax and BGB-16673 – we are advancing a potentially best-in-class portfolio in B-cell malignancies," said Lai Wang, Ph.D. Global Head of R&D. "At EHA (Free EHA Whitepaper) 2025, we’ll share 31 accepted abstracts that highlight the progress of our hematology clinical development program and our commitment to targeted therapies that raise the standard of care. As BRUKINSA continues to expand its impact and our next-generation assets advance, we hope to transform the future of treatment for patients facing B-cell malignancies."

BeiGene’s next-generation pipeline assets, including BGB-16673 and sonrotoclax, continue to demonstrate promising clinical activity and generally well-tolerated safety profiles across multiple B-cell malignancies. Together, these programs have enrolled more than 2,500 patients globally (1,900+ patients for sonrotoclax and 600+ patients for BGB-16673) and are positioned to potentially play a significant role in treatment strategies for chronic lymphocytic leukemia (CLL), Waldenström’s macroglobulinemia (WM) and mantle cell lymphoma (MCL). Additionally, presentations further reinforce BRUKINSA’s durable efficacy and consistent safety profile, as well as its position as a foundational therapeutic option in frontline CLL. Key highlights include:

Two oral presentations featuring updated results from the ongoing Phase 1 CaDAnCe-101 study of BGB-16673 in patients with relapsed or refractory (R/R) CLL/SLL and patients with R/R WM, showing continued and promising early efficacy and a tolerable safety profile.
Two oral presentations showcasing updated Phase 1 results of sonrotoclax in combination with BRUKINSA in R/R CLL/SLL and R/R MCL, which demonstrate deep and durable responses. This combination is now under evaluation in the ongoing registrational Phase 3 fixed-duration CELESTIAL-TNCLL study (NCT06073821) in patients with treatment-naïve CLL/SLL, which completed enrollment earlier this year, and CELESTIAL-RRMCL study (NCT06742996) in patients with relapsed / refractory MCL, which is currently enrolling.
Results from Arm C and D of the SEQUOIA study, which evaluated BRUKINSA in patients with TN CLL/SLL and del(17p) (Arm C) and BRUKINSA plus venetoclax in patients with TN CLL/SLL with del(17p) and/or TP53 mutation or without either mutation (Arm D).
Robust analyses across clinical trials and real-world evidence that deepen understanding of treatment patterns, safety, and outcomes in CLL/SLL, MCL and diffuse large B-cell lymphoma (DLBCL).
BeiGene Presentations and Publications at EHA (Free EHA Whitepaper)2025

Abstract Title

Presentation Details (CEST)

Lead Author

BGB-16673 (BTK CDAC)

Updated efficacy and safety of the Bruton tyrosine kinase degrader BGB-16673 in patients with relapsed or refractory CLL/SLL: results from the ongoing phase 1 CaDAnCe-101 study

Oral Presentation: S158

Session Title: Chronic lymphocytic leukemia and related disorders – Clinical

Session Date/Time: Sunday, June 15, 11:00 – 12:15

L. Scarfò

Updated efficacy and safety of the Bruton tyrosine kinase degrader BGB-16673 in patients with relapsed/refractory Waldenström macroglobulinemia: ongoing phase 1 CaDAnCe-101 study results

Oral Presentation: S231

Session Title: Indolent and mantle-cell non-Hodgkin lymphoma – Clinical

Session Date/Time: Saturday, June 14, 17:00 – 18:15

A.M. Frustaci

Sonrotoclax (BCL2 Inhibitor)

Updated results from the phase 1 study of sonrotoclax (BGB-11417), a novel BCL2 inhibitor, in combination with zanubrutinib for relapsed/refractory CLL/SLL show deep and durable responses

Oral Presentation: S159

Session Title: Chronic lymphocytic leukemia and related disorders – Clinical

Session Date/Time: Sunday, June 15, 11:00 – 12:15

C.Y. Cheah

Combination treatment with novel BCL2 inhibitor sonrotoclax (BGB-11417) and zanubrutinib induces high rate of complete remission for patients with relapsed/refractory mantle cell lymphoma

Oral Presentation: S234

Session Title: Indolent and mantle-cell non-Hodgkin lymphoma – Clinical

Session Date/Time: Saturday, June 14, 17:00 – 18:15

C.S. Tam

Sonrotoclax monotherapy for treatment of patients with relapsed/refractory CLL: data from an ongoing phase 1/1b study (BGB-11417-101)

Poster #: PF580

Poster Presentation

Session Title: Poster Session 1

Session Date/Time: Friday, June 13, 18:30 – 19:30

S.S. Opat

Updated safety and efficacy results of a phase 1 study of the novel BCL2 inhibitor sonrotoclax (BGB-11417) for relapsed/refractory Waldenström’s macroglobulinemia

Poster #: PF887

Poster Presentation

Session Title: Poster Session 1

Session Date/Time: Friday, June 13, 18:30 – 19:30

C.Y. Cheah

Primary analysis results of novel BCL2 inhibitor sonrotoclax (BGB-11417) monotherapy in patients with relapsed/refractory b-cell malignancies

Poster #: PF574

Poster Presentation

Session Title: Poster Session 1

Session Date/Time: Friday, June 13, 18:30 – 19:30

C. Li

Updated safety and antileukemic activity data for sonrotoclax (BGB-11417), a potent and selective BCL2 inhibitor, in patients with relapsed/refractory acute myeloid leukemia

Poster #: PF491

Poster Presentation

Session Title: Poster Session 1

Session Date/Time: Friday, June 13, 18:30 – 19:30

P. Montesinos

Updated safety and antileukemic activity data of sonrotoclax (BGB-11417), a potent and selective BCL2 inhibitor, in treatment-naive patients with acute myeloid leukemia unfit for intensive chemotherapy

Poster #: PF477

Poster Presentation

Session Title: Poster Session 1

Session Date/Time: Friday, June 13, 18:30 – 19:30

J. Shortt

Updated interim results of sonrotoclax plus dexamethasone in patients with t(11;14)-positive relapsed/refractory multiple myeloma: an all-oral treatment

Poster #: PF721

Poster Presentation

Session Title: Poster Session 1

Session Date/Time: Friday, June 13, 18:30 – 19:30

B. Dhakal

BGB-11417-302, a phase 3, randomized, double-blind study of sonrotoclax (BGB-11417) plus zanubrutinib versus placebo plus zanubrutinib in patients with relapsed or refractory mantle cell lymphoma

Publication Only

M. Hughes

BRUKINSA

SEQUOIA 5-year follow-up in arm C: frontline zanubrutinib monotherapy in patients with del(17p) and treatment-naive chronic lymphocytic leukemia/small lymphocytic lymphoma

Poster #: PS1565

Poster Presentation

Session Title: Poster Session 2

Session Date/Time: Saturday, June 14, 18:30 – 19:30

C.S. Tam

Combination of zanubrutinib plus venetoclax for treatment-naive CLL/SLL: results in SEQUOIA arm D

Poster #: PS1566

Poster Presentation

Session Title: Poster Session 2

Session Date/Time: Saturday, June 14, 18:30 – 19:30

M. Shadman

Final analysis of a phase 1 study of zanubrutinib plus lenalidomide in patients with relapsed/refractory diffuse large b-cell lymphoma

Poster #: PS1918

Poster Presentation

Session Title: Poster Session 2

Session Date/Time: Saturday, June 14, 18:30 – 19:30

Z. Song

Additional Abstracts

Integrative Evidence Generation and Health Economics Related to Zanubrutinib

Final independent review data supports sustained benefit of zanubrutinib over ibrutinib in patients with R/R CLL/SLL in ALPINE

Publication Only

J. Brown

Preference Survey and Patient Experience Study

Preferences for treatment in first-line chronic lymphocytic leukemia: A multi-criteria decision analysis in Italy

Publication Only

P. Sportoletti

A qualitative study to explore the patient experience of continuous covalent Bruton tyrosine kinase inhibitors in chronic lymphocytic leukemia: study methodology

Publication Only

L. Scarfò

Real-World Evidence

Real-world burden of disease, treatment patterns and outcomes in patients with mantle cell lymphoma

Poster #: PF899

Poster Presentation

Session Title: Poster Session 1

Session Date/Time: Friday, June 13, 18:30 – 19:30

A. Alencar

Real-world comparative effectiveness of first-line Bruton tyrosine kinase inhibitors in patients with chronic lymphocytic leukemia

Poster #: PS1578

Poster Presentation

Session Title: Poster Session 2

Session Date/Time: Saturday, June 14, 18:30 – 19:30

R. Jacobs

Real-world treatment utilization patterns, discontinuation and healthcare resource utilization of first-line Bruton tyrosine kinase inhibitors in chronic lymphocytic leukemia: age-related disparity

Poster #: PF585

Poster Presentation

Session Title: Poster Session 1

Session Date/Time: Friday, June 13, 18:30 – 19:30

K. Yang

Risk of hypertension in patients newly diagnosed with chronic lymphocytic

leukemia/small lymphocytic lymphoma (CLL/SLL) and treated with covalent Bruton tyrosine kinase inhibitors: a real-world study

Poster #: PF588

Poster Presentation

Session Title: Poster Session 1

Session Date/Time: Friday, June 13, 18:30 – 19:30

A. Ali

Real-world Bruton tyrosine kinase inhibitor use and clinical outcomes among patients with chronic lymphocytic leukemia/small lymphocytic lymphoma

Publication Only

J. Hou

Real-world zanubrutinib treatment patterns in chronic lymphocytic leukemia/small lymphocytic lymphoma among US community oncology patients with prior acalabrutinib therapy

Publication Only

J. Hou

Real-world zanubrutinib treatment patterns in mantle cell lymphoma among US community oncology patients with prior Bruton tyrosine kinase inhibitor therapy

Poster #: PF914

Poster Presentation

Session Title: Poster Session 1

Session Date/Time: Friday, June 13, 18:30 – 19:30

R. Choksi

Evaluating uptake of targeted agents by race/ethnicity in patients receiving first-line treatment for chronic lymphocytic leukemia

Publication Only

A.S. Kittai

Serious infections in patients with CLL/SLL treated with combination venetoclax and obinutuzumab compared with those treated with zanubrutinib: a real-world study

Poster #: PS1571

Poster Presentation

Session Title: Poster Session 2

Session Date/Time: Saturday, June 14, 18:30 – 19:30

N. Lamanna

Matching-Adjusted Indirect Comparison

Comparative efficacy of zanubrutinib versus fixed-duration acalabrutinib plus venetoclax for first-line treatment of chronic lymphocytic leukemia: a matching-adjusted indirect comparison

Poster #: PS1581

Poster Presentation

Session Title: Poster Session 2

Session Date/Time: Saturday, June 14, 18:30 – 19:30

T. Munir

Efficacy of continuous zanubrutinib vs fixed-duration venetoclax in combination with obinutuzumab in treatment-naive chronic lymphocytic leukemia: a matching-adjusted indirect comparison

Publication only

T. Munir

Adverse events of interest with zanubrutinib vs fixed-duration combination of venetoclax plus obinutuzumab in treatment-naive chronic lymphocytic leukemia

Publication only

N. Lamanna

Network Meta-Analysis

A network meta-analysis of efficacy of zanubrutinib versus fixed-duration acalabrutinib plus venetoclax in treatment-naïve chronic lymphocytic leukemia

Publication Only

M. Shadman

Ociperlimab (BGB-A1217)

Advantig-101: a phase 1b/2 study of ociperlimab plus tislelizumab or rituximab in relapsed/refractory diffuse large b-cell lymphoma

Poster #: PS1995

Poster Presentation

Session Title: Poster Session 2

Session Date/Time: Saturday, June 14, 18:30 – 19:30

Q. Cai

About Sonrotoclax (BGB-11417)
Sonrotoclax is designed to block the B-cell lymphoma 2 (BCL2) protein, which is one of several proteins that help cancer cells survive. It is part of a group of drugs called BH3 mimetics, which mimic natural cell death signals. Studies in the lab and during early drug development have shown that sonrotoclax is a potent and specific inhibitor of BCL2 with a short half-life and no accumulation. Sonrotoclax has shown promising clinical activity across a range of B-cell malignancies, and more than 1,900 patients have been enrolled to date across the global development program. The U.S. Food and Drug Administration (FDA) granted sonrotoclax Fast Track Designation for the treatment of adult patients with mantle cell lymphoma (MCL) and Waldenström macroglobulinemia (WM).

About BGB‑16673
BGB-16673 is an orally available Bruton’s tyrosine kinase (BTK) targeting protein degrader from BeiGene’s chimeric degradation activation compound (CDAC) platform. BGB-16673 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. BGB-16673 is the most advanced BTK protein degrader in the clinic, with an extensive global clinical development program. The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to BGB-16673 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).

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.

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

The global BRUKINSA clinical development program includes about 7,100 patients enrolled in 30 countries and regions across more than 35 trials. BRUKINSA is approved in more than 75 markets, and more than 200,000 patients have been treated globally.

U.S. Indications and Important Safety Information for BRUKINSA (zanubrutinib)

INDICATIONS

BRUKINSA is a kinase inhibitor indicated for the treatment of adult patients with:

Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
Waldenström’s macroglobulinemia (WM).
Mantle cell lymphoma (MCL) who have received at least one prior therapy.
Relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen.
Relapsed or refractory follicular lymphoma (FL), in combination with obinutuzumab, after two or more lines of systemic therapy.
The MCL, MZL and FL indications are approved under accelerated approval based on overall response rate and durability of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Hemorrhage

Fatal and serious hemorrhage has occurred in patients with hematological malignancies treated with BRUKINSA. Grade 3 or higher hemorrhage including intracranial and gastrointestinal hemorrhage, hematuria, and hemothorax was reported in 3.8% of patients treated with BRUKINSA in clinical trials, with fatalities occurring in 0.2% of patients. Bleeding of any grade, excluding purpura and petechiae, occurred in 32% of patients.

Bleeding has occurred in patients with and without concomitant antiplatelet or anticoagulation therapy. Coadministration of BRUKINSA with antiplatelet or anticoagulant medications may further increase the risk of hemorrhage.

Monitor for signs and symptoms of bleeding. Discontinue BRUKINSA if intracranial hemorrhage of any grade occurs. Consider the benefit-risk of withholding BRUKINSA for 3-7 days before and after surgery depending upon the type of surgery and the risk of bleeding.

Infections

Fatal and serious infections (including bacterial, viral, or fungal infections) and opportunistic infections have occurred in patients with hematological malignancies treated with BRUKINSA. Grade 3 or higher infections occurred in 26% of patients, most commonly pneumonia (7.9%), with fatal infections occurring in 3.2% of patients. Infections due to hepatitis B virus (HBV) reactivation have occurred.

Consider prophylaxis for herpes simplex virus, pneumocystis jirovecii pneumonia, and other infections according to standard of care in patients who are at increased risk for infections. Monitor and evaluate patients for fever or other signs and symptoms of infection and treat appropriately.

Cytopenias

Grade 3 or 4 cytopenias, including neutropenia (21%), thrombocytopenia (8%) and anemia (8%) based on laboratory measurements, developed in patients treated with BRUKINSA. Grade 4 neutropenia occurred in 10% of patients, and Grade 4 thrombocytopenia occurred in 2.5% of patients.

Monitor complete blood counts regularly during treatment and interrupt treatment, reduce the dose, or discontinue treatment as warranted. Treat using growth factor or transfusions, as needed.

Second Primary Malignancies

Second primary malignancies, including non-skin carcinoma, have occurred in 14% of patients treated with BRUKINSA. The most frequent second primary malignancy was non-melanoma skin cancers (8%), followed by other solid tumors in 7% of the patients (including melanoma in 1% of patients) and hematologic malignancies (0.7%). Advise patients to use sun protection and monitor patients for the development of second primary malignancies.

Cardiac Arrhythmias

Serious cardiac arrhythmias have occurred in patients treated with BRUKINSA. Atrial fibrillation and atrial flutter were reported in 4.4% patients treated with BRUKINSA, including Grade 3 or higher cases in 1.9% of patients. Patients with cardiac risk factors, hypertension, and acute infections may be at increased risk. Grade 3 or higher ventricular arrhythmias were reported in 0.3% of patients.

Monitor for signs and symptoms of cardiac arrhythmias (e.g., palpitations, dizziness, syncope, dyspnea, chest discomfort), manage appropriately, and consider the risks and benefits of continued BRUKINSA treatment.

Hepatotoxicity, Including Drug-Induced Liver Injury

Hepatotoxicity, including severe, life-threatening, and potentially fatal cases of drug-induced liver injury (DILI), has occurred in patients treated with Bruton tyrosine kinase inhibitors, including BRUKINSA.

Evaluate bilirubin and transaminases at baseline and throughout treatment with BRUKINSA. For patients who develop abnormal liver tests after BRUKINSA, monitor more frequently for liver test abnormalities and clinical signs and symptoms of hepatic toxicity. If DILI is suspected, withhold BRUKINSA. Upon confirmation of DILI, discontinue BRUKINSA.

Embryo-Fetal Toxicity

Based on findings in animals, BRUKINSA can cause fetal harm when administered to a pregnant woman. Administration of zanubrutinib to pregnant rats during the period of organogenesis caused embryo-fetal toxicity, including malformations at exposures that were 5 times higher than those reported in patients at the recommended dose of 160 mg twice daily. Advise women to avoid becoming pregnant while taking BRUKINSA and for 1 week after the last dose. Advise men to avoid fathering a child during treatment and for 1 week after the last dose. If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus.

Adverse Reactions

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

Drug Interactions

CYP3A Inhibitors: When BRUKINSA is co-administered with a strong CYP3A inhibitor, reduce BRUKINSA dose to 80 mg once daily. For coadministration with a moderate CYP3A inhibitor, reduce BRUKINSA dose to 80 mg twice daily.

CYP3A Inducers: Avoid coadministration with strong or moderate CYP3A inducers. Dose adjustment may be recommended with moderate CYP3A inducers.

Specific Populations

Hepatic Impairment: The recommended dose of BRUKINSA for patients with severe hepatic impairment is 80 mg orally twice daily.

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

This information is intended for a global audience. Product indications vary by region.