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U.S. FDA Approves Tablet Formulation of BeOne’s BRUKINSA® for All Approved Indications

On June 11, 2025 BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reported that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of BRUKINSA (zanubrutinib) for all five approved indications (Press release, BeiGene, JUN 11, 2025, View Source [SID1234653826]). BRUKINSA remains the leader in new chronic lymphocytic leukemia (CLL) patient starts across all lines of therapy in the U.S., and for the first time, has become the overall BTK inhibitor market share leader.1

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BRUKINSA tablets have the same efficacy and safety as BRUKINSA capsules based on the results of two single-dose, open-label, randomized Phase 1 crossover studies of healthy adults designed to establish bioequivalence. BRUKINSA is the only BTK inhibitor to offer the flexibility of once or twice daily dosing, with the ability to tailor the schedule to patient needs. It also continues to be the only BTK inhibitor with recommended dosing for severe hepatic impairment.

"BRUKINSA’s leadership in the U.S. underscores the trust physicians and patients have placed in its differentiated clinical profile," said Matt Shaulis, General Manager of North America, BeOne. "With this new tablet formulation, we are making treatment simpler and more convenient—an important step forward for patients facing certain B-cell cancers."

The recommended dose of BRUKINSA remains at 320 mg daily. The new BRUKINSA tablets are 160 mg each, allowing patients to take two tablets daily rather than four of the current 80 mg capsules. Additionally, BRUKINSA tablets are smaller than the capsules and have a film coating, making them easier to swallow. The BRUKINSA tablets will replace capsules starting in October 2025.

The European Medicines Agency is currently reviewing a Type II variation marketing authorization application (MAA) for the new tablet formulation of BRUKINSA in all currently approved indications, with approval expected later this year.

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. Additionally, 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 in at least one indication, 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.

Hoth Therapeutics Launches Expanded Access Initiative for HT-001, Offering Critical Relief to Cancer Patients Facing Severe Skin Toxicities

On June 11, 2025 Hoth Therapeutics, Inc. (NASDAQ: HOTH), a patient-focused biopharmaceutical company advancing breakthrough therapies for underserved conditions, reported a major milestone in the development of its lead asset, HT-001, with the engagement of Premier Research to support submission of an Expanded Access Program (EAP) application (Press release, Hoth Therapeutics, JUN 11, 2025, View Source [SID1234653825]). This move positions Hoth to offer compassionate access to HT-001 for cancer patients suffering from painful and debilitating skin toxicities caused by epidermal growth factor receptor (EGFR) inhibitor therapies—an area of high unmet medical need.

HT-001 has shown promising signs of clinical benefit in early evaluations, and now Hoth is preparing to make the investigational drug available to qualified patients outside of ongoing clinical trials. Expanded Access—often referred to as "compassionate use"—represents an important regulatory pathway that allows patients ineligible for traditional trials to gain access to experimental treatments when no approved alternatives exist.

"We’re seeing encouraging signals from our open-label Phase 2a cohort and are taking bold steps to accelerate availability for patients in need," said Robb Knie, Chief Executive Officer of Hoth Therapeutics. "This Expanded Access initiative not only reflects our dedication to patient care but also enhances HT-001’s clinical visibility and commercial potential."

The strategic move expands HT-001’s reach and reinforces Hoth’s long-term vision to be a leader in cancer supportive care. With a clearly defined regulatory pathway, a fast-growing addressable market, and no currently approved targeted treatment for EGFR-inhibitor-induced skin toxicities, HT-001 is uniquely positioned to capture attention from both the medical community and the investor marketplace.

Why It Matters for Investors

High Unmet Need: Over 100,000 cancer patients annually receive EGFR inhibitors, with the majority experiencing skin toxicities that currently lack targeted treatments.

Strong Market Potential: HT-001 targets a rapidly growing segment in oncology supportive care.

Regulatory Momentum: Expanded Access status creates a faster route to commercialization and increases real-world data generation.
Investors and partners are encouraged to monitor updates closely as Hoth Therapeutics continues to advance HT-001 toward its next key inflection points.

For more information about HT-001, clinical trials, or Expanded Access eligibility, visit www.hoththerapeutics.com.

Kelun-Biotech’s Novel CLDN18.2 ADC SKB315 IND in Combination with Tagolizumab Receives NMPA Approval for the First-Line Treatment of Gastric/Gastro-oesophageal Junction Cancer

On June 11, 2025 Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd. (the "Company") reported that the Company has received a clinical trial notice approving the investigational new drug application for the its CLDN18.2-directed antibody-drug conjugate (ADC) SKB315 in combination of the anti-programmed death ligand 1 (PD-L1) monoclonal antibody (mAb) tagitanlimab for the first-line treatment of gastric cancer / gastro-oesophageal junction cancer (GC/GEJC) from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) (Press release, Kelun, JUN 11, 2025, View Source [SID1234653824]).

SKB315 is a novel CLDN18.2-directed ADC for advanced solid tumors, configured with a proprietary, in-house developed humanized CLDN18.2 mAb and a differentiated payload-linker design. Building upon ongoing monotherapy studies in CLDN-expressing tumors such as GC/GEJC and pancreatic ductal adenocarcinoma, the Company is also initiating clinical studies of its combination therapy with immunotherapy for the first-line treatment of CLDN-positive GC/GEJC.

Tagitanlimab is the first PD-L1 mAb to receive authorization for the first-line treatment of nasopharyngeal carcinoma (NPC). It has been approved by the NMPA for marketing in China as a combination therapy with cisplatin and gemcitabine for the first-line treatment of patients with recurrent or metastatic (R/M) NPC, as well as monotherapy for the treatment of patients with R/M NPC who have failed after prior 2L+ chemotherapy.

Previously, tagitanlimab in combination with TROP2-directed antibody-drug conjugate (ADC) sacituzumab tirumotecan (sac-TMT) granted Breakthrough Therapy Designation by the CDE of the NMPA of China for the first-line treatment of locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) without actionable genomic alterations.

Encouraging clinical development progress has been made in recent years with combination strategies based on PD-(L)1 antibodies. The innovative ADC SKB315 combined with the PD-L1 monoclonal antibody tagitanlimab is expected to generate synergistic effects through their distinct mechanisms of action, overcoming tumor heterogeneity among different patients and delivering greater survival benefits.

Kazia Therapeutics Announces Transformative Preclinical Data Demonstrating Paxalisib’s Potential to Overcome Immunotherapy Resistance in Triple-Negative Breast Cancer (TNBC)

On June 11, 2025 Kazia Therapeutics Limited (NASDAQ: KZIA), a clinical-stage biotechnology company pioneering next-generation oncology therapeutics, reported the publication of transformative preclinical research in the journal Molecular Cancer Therapeutics, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) that underscores the powerful potential of its lead asset, paxalisib, in reshaping the treatment landscape for triple-negative breast cancer (TNBC), one of the most aggressive and treatment-resistant cancer subtypes (Press release, Kazia Therapeutics, JUN 11, 2025, View Source [SID1234653823]).

The preclinical program was conducted by Professor Sudha Rao at the renowned QIMR Berghofer Medical Research Institute, revealing how paxalisib can reprogram the tumor microenvironment and enhance immune response, showing substantial synergy with immune checkpoint inhibitors. The data provide strong scientific and translational rationale for continued development of paxalisib as part of immunotherapy-based regimens.

Key Findings

Dual targeting of PI3K and mTOR but not PI3K alone inhibits cancer cell proliferation and migration in vitro.
Paxalisib remodels the TNBC tumor microenvironment, increasing CD4+ and CD8+ T cell infiltration and activation
The combination of paxalisib with KEYTRUDA (pembrolizumab) demonstrated synergistic antitumor activity in advanced breast cancer, resulting in robust tumor regression and prolonged survival in preclinical models
The data validate a mechanistic rationale for ongoing clinical exploration of paxalisib in combination with checkpoint inhibitors and PARP inhibitors
"This landmark study offers a mechanistic and translational foundation for our newly launched Phase 1b clinical trial of paxalisib in advanced breast cancer," said Dr. John Friend, CEO of Kazia. "It not only extends the therapeutic potential of paxalisib beyond brain cancers but also positions it at the forefront of innovative immunotherapy combinations in solid tumors."

The publication, "Combination of the PI3K/mTOR inhibitor paxalisib with immune checkpoint inhibitors enhances antitumor activity in preclinical models of triple-negative breast cancer," is available online at View Source

Clinical Milestone
Kazia recently announced that the first patient has been dosed in the Phase 1b trial evaluating paxalisib in combination with checkpoint inhibitors and chemotherapy in advanced breast cancer, marking a critical step toward clinical translation of these findings.

For investor and media, please contact Alex Star, Managing Director LifeSci Advisors LLC

Intensity Therapeutics, Inc. Announces Patients Receiving INT230-6 Prior to the Start of Standard-of-Care May Achieve High Levels of Tumor Necrosis in the Ongoing Randomized Controlled Phase 2 INVINCIBLE-4 Study

On June 11, 2025 Intensity Therapeutics, Inc. (Nasdaq: INTS) ("Intensity" or the "Company"), a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, reported first few patients receiving INT230-6 achieved high levels of necrosis after 8 days in the Phase 2, INVINCIBLE-4 study, before they initiated the standard-of-care regimen (Press release, Intensity Therapeutics, JUN 11, 2025, View Source [SID1234653822]).

The INVINCIBLE-4 Study is a randomized, open-label, multicenter study to determine the clinical activity, safety, and tolerability of INT230-6 in patients with early-stage, operable triple-negative breast cancer ("TNBC") who undergo standard of care neoadjuvant immunochemotherapy ("SOC") treatment and SOC alone. The primary endpoint is pathological complete response ("pCR"), i.e., the absence of live cancer in the primary tumor and affected lymph nodes. Patients are being randomized one-to-one to receive either a regimen of two doses of INT230-6 followed by SOC, which consists of pembrolizumab, anthracyclines, carboplatin, cyclophosphamide, and paclitaxel (i.e., the Keynote-522 regimen), or SOC alone. The study is recruiting patients in Switzerland and France and is expected to enroll 54 patients.

"We are encouraged to see high levels of tumor necrosis from the MRI scans and evidence of tumor inflammation after two INT230-6 injections and before initiation of the SOC in our first patients," said Ursina Zürrer, M.D. Chief Physician for Genetic Counseling, Department of Medical Oncology and Hematology Cantonal Hospital Winterthur, Switzerland, and the Coordinating Investigator for the INVINCIBLE-4 Study. "We are encouraged that these patients achieved such a good response to INT230-6 prior to their beginning the Standard immune-chemo regimen and look forward to continuing enrollment in the study."

"We are excited to see that INT230-6 is achieving meaningful levels of necrosis in patients with evidence of immune activation. TNBC Patients who have no live cancer in their tumor or nodes at the time of surgery have a significantly improved event-free survival advantage compared to those who do not have a pathological complete response. TNBC patients risk their lives to achieve a pCR, and about forty percent fail to achieve the desired result. We look forward to seeing the pathological complete response data being generated by our partners at SAKK and Unicancer," said Lewis H. Bender, President and CEO of Intensity.

About INT230-6

INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug is comprised of two proven, potent anti-cancer agents, cisplatin and vinblastine sulfate, and a diffusion and cell penetration enhancer molecule ("SHAO") that helps disperse potent cytotoxic drugs throughout tumors for diffusion into cancer cells. These agents remain in the tumor, resulting in a favorable safety profile. In addition to local disease control and direct tumor killing, INT230-6 causes a release of a bolus of neoantigens specific to the malignancy, leading to immune system engagement and systemic anti-tumor effects. Importantly, these effects are mediated without immunosuppression, which often occurs with systemic chemotherapy.

About Triple Negative Breast Cancer in the Presurgical Setting

Women with aggressive forms of breast cancer, such as TNBC, are often counseled to undergo pre-surgical (neoadjuvant) systemic therapy in advance to reduce the risk of the disease returning. Having a pathological complete response, meaning the absence of live cancer at the time of surgery, has been shown to result in a lower risk of recurrence. Approximately 11-17% of breast cancers test negative for estrogen receptors (ER), progesterone receptors (PR), and overexpression of human epidermal growth factor receptor 2 (HER2) protein, qualifying them as triple negative. There are approximately 56,000 new cases of TNBC in the US and 420,000 Worldwide diagnosed each year, the majority of which are local to the breast. TNBC is considered to be more aggressive and has a poorer prognosis than other types of breast cancer, because there are fewer available targeted medicines. Most patients with local TNBC typically receive immune/chemotherapy before surgery. Since the publication of Keynote-522, the standard neoadjuvant treatment for TNBC includes systemic chemotherapy (anthracyclines, cyclophosphamide, paclitaxel, carboplatin) and the anti-PD-1 monoclonal antibody pembrolizumab. pCR rates are 65%, with rates generally lower in the larger-sized tumors or with lymph node metastasis. The toxicity of the Keynote-522 regimen is high, with 80% of patients experiencing grade 3 or higher treatment-related AEs, including treatment-related adverse events that lead to death in 0.5% of patients.