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.

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 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage global leader and pioneer in the field of antibody drug conjugates (ADCs), reported data presentations from the LOTIS-7 Phase 1b clinical trial evaluating ZYNLONTA (loncastuximab tesirine-lpyl) in combination with glofitamab in patients with relapsed/refractory (r/r) diffuse large B-cell lymphoma (DLBCL) (Press release, ADC Therapeutics, MAY 14, 2025, View Source [SID1234653097]). Updated results will be shared at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress (EHA2025) taking place in Milan, Italy with an oral encore presentation at the 18th International Conference on Malignant Lymphoma (ICML) in Lugano, Switzerland. Updated LOTIS-5 safety run-in data evaluating the combination of ZYNLONTA plus rituximab (Lonca-R) will also be featured at EHA (Free EHA Whitepaper)2025.

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"We are excited to present the latest LOTIS-7 data from a larger subset of patients with longer follow-up at EHA (Free EHA Whitepaper) and ICML," said Mohamed Zaki, MD, PhD, Chief Medical Officer of ADC Therapeutics. "The robust efficacy and manageable safety seen to date with the combination of ZYNLONTA and glofitamab, two potent anti-cancer agents with different mechanisms of action, reinforce the potential for this regimen to change the treatment paradigm for patients with aggressive lymphoma."

The LOTIS-7 abstract provides data as of the January 17, 2025, cutoff, in which 31 patients received ≥1 ZYNLONTA dose and were safety evaluable, with 22 patients efficacy evaluable. Four of these patients (2 each at 120µg/kg and 150 µg/kg) converted to complete response (CR) within 3 weeks after the data cutoff and are included as CRs. Updated data will be presented during EHA (Free EHA Whitepaper)2025.

Key highlights in the LOTIS-7 abstract are as follows:

In the efficacy evaluable population, overall response rate (ORR) was 95.5% (21/22), complete response (CR) rate was 90.9% (20/22), and median duration of response (DOR) was not reached.
Among 31 patients treated, the combination demonstrated a manageable safety profile.
Adverse events were consistent with known profiles of the individual agents, with neutropenia (32.3%) being the most common Grade ≥3 treatment-emergent adverse event (TEAEs). Grades 3/4 TEAEs of interest included generalized edema, pericardial effusion, photosensitivity reaction, rash, sepsis and pneumonia (each 3.2%). Grade 1/2 AE of CRS (29.0%/9.7%) and ICANS (0%/6.5%) were observed with no Grade ≥3 at the time of data cut off.
Details of the EHA (Free EHA Whitepaper)2025 poster presentations are as follows:
Title: Initial Results From LOTIS-7: A Phase 1b Study of Loncastuximab Tesirine Plus
Glofitamab in Patients With Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL)
Session: Poster Session 2
Session Date and Time: Saturday, June 14; 12:30-1:30 p.m. ET / 18:30 -19:30 CEST
Location: Poster Hall, Allianz MiCo, Milano Convention Centre
Presenting Author: Juan Pablo Alderuccio, MD, Associate Professor of Medicine and Hematologist at Sylvester Comprehensive Cancer Center, University of Miami
Abstract: PS1911

Title: Updated Safety Run-In Results From LOTIS-5: A Phase 3, Randomized Trial of Loncastuximab Tesirine With Rituximab Versus Immunochemotherapy in Patients With R/R DLBCL/HGBL
Session: Poster Session 2
Session Date and Time: Saturday, June 14; 12:30-1:30 p.m. ET / 18:30 -19:30 CEST
Location: Poster Hall, Allianz MiCo, Milano Convention Centre
Presenting Author: Carmelo Carlo-Stella, MD, PhD, section chief of Lymphoid Malignancies and Cancer Therapeutics at Humanitas Clinical and Research Center (IRCCS)
Abstract: PS1957

Details of the ICML oral encore presentation are as follows:
Title: Initial Results From LOTIS-7: A Phase 1b Study of Loncastuximab Tesirine Plus Glofitamab in Patients With Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL)
Session: 13 – Aggressive B-Cell Lymphomas
Session Date and Time: Friday, June 20; 8:00 a.m.-9:30 a.m. ET / 14:00-15:30 CEST
Location: Room A, broadcast in Cinema Corso, Lugano Convention Centre, Palazzo dei Congressi
Presenting Author: Juan Pablo Alderuccio, MD, Associate Professor of Medicine and Hematologist at Sylvester Comprehensive Cancer Center, University of Miami
Abstract: 078

Additionally, a poster entitled, "Updated analysis of a phase 2 multicenter study of the loncastuximab in relapsed/refractory marginal zone lymphoma demonstrates high rate of complete responses" will be presented at ICML. This single-arm, open-label investigator-initiated study is being conducted at the Sylvester Comprehensive Cancer Center at University of Miami and City of Hope, and led by Izidore Lossos, MD, Professor, Director, Lymphoma Program at the Sylvester Comprehensive Cancer Center, University of Miami.

About LOTIS-7
LOTIS-7 is a Phase 1b global multicenter, multi-arm study in patients with relapsed or refractory B-cell non-Hodgkin lymphoma (B-NHL) including Part 1 (dose escalation) and Part 2 (dose expansion). The three dosing arms include ZYNLONTA plus polatuzumab vedotin, ZYNLONTA plus glofitamab, and ZYNLONTA plus mosunetuzumab T-cell-engaging bispecific monoclonal antibodies (BsAbs). Enrollment in LOTIS-7 includes Part 1 of the study with a 3+3 dose escalation in 3L/3L+ heavily pre-treated patients with ZYNLONTA doses starting at 90 µg/kg and then proceeding to 120 µg/kg and 150 µg/kg. Part 2 includes dose expansion in 2L/2L+ large B-cell lymphoma in the ZYNLONTA plus glofitamab arm at dose levels determined from Part 1 (120 µg/kg and 150 µg/kg of ZYNLONTA plus the approved dosing of glofitamab). Primary endpoints of the study include safety and tolerability. Secondary efficacy endpoints include ORR, DOR, CRR, PFS, RFS, and OS as well as pharmacokinetics and immunogenicity.

For more information about the LOTIS-7 trial, visit clinicaltrials.gov (NCT04970901).

About ZYNLONTA
ZYNLONTA is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death.

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval and in the European Union under conditional approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Please see full prescribing information including important safety information about ZYNLONTA at www.ZYNLONTA.com.

ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.

On May 14, 2025 Inhibrx Biosciences, Inc. (Nasdaq: INBX) ("Inhibrx" or the "Company") reported financial results for the first quarter of 2025. Following the completion of the sale of INBRX-101 by Inhibrx, Inc. (the "Former Parent") to Sanofi S.A. and the Former Parent’s concurrent spin-off of the Inhibrx business in May 2024, the biopharmaceutical company now has two programs in ongoing clinical trials, with data readouts for each expected within the current year (Press release, Inhibrx, MAY 14, 2025, View Source [SID1234653096]). Because the spin-off was accounted for as a reverse spin-off, for periods prior to the spin-off, the Company’s financial statements are the historical financial statements of the Former Parent.

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Upcoming Milestones

INBRX-109
Data from the registration-enabling Phase 2 trial in unresectable or metastatic conventional chondrosarcoma are expected during the third quarter of 2025; and
Initial data on the colorectal cancer expansion cohort are anticipated in the third quarter of 2025 with interim data from the Ewing sarcoma expansion cohort expected in the second half of 2025.
INBRX-106
Initial Phase 2 data from the randomized Phase 2/3 trial in head and neck squamous cell carcinoma in combination with KEYTRUDA (pembrolizumab) are expected during the fourth quarter of 2025; and
Interim data from the Phase 1/2 checkpoint inhibitor refractory or relapsed non-small cell lung cancer are expected during the fourth quarter of 2025.
Financial Results

Cash and Cash Equivalents. As of March 31, 2025, Inhibrx had cash and cash equivalents of $216.5 million, compared to $152.6 million as of December 31, 2024. The Company’s cash balance increased following the receipt of $100.0 million in gross principal in January 2025 under a loan and security agreement (the "2025 Loan Agreement") entered into with Oxford Finance LLC ("Oxford").
R&D Expense. Research and development expenses were $36.9 million during the first quarter of 2025, compared to $63.9 million during the first quarter of 2024. The decrease in research and development expenses was primarily due to a decrease in clinical trial expenses and contract manufacturing expenses following the spin-off of our INBRX-101 program in the second quarter of 2024.
G&A Expense. General and administrative expenses were $6.0 million during the first quarter of 2025, compared to $10.0 million during the first quarter of 2024. The decrease in general and administrative expenses was primarily due to:
a decrease in non-cash stock option expense as a result of fewer stock options outstanding in the current period under the Company’s new 2024 Omnibus Incentive Plan following the termination of its prior plan in connection with the spin-off;
a decrease in professional service expenses in the current period as a result of higher legal and accounting services in the prior period to support the spin-off transaction and the conclusion of legal proceedings.
Other Income (Expense). Other expense was $0.4 million during the first quarter of 2025, compared to other expense of $4.9 million during the first quarter of 2024. Other expense consists of interest expense incurred on the Company’s outstanding third-party debt during each period, offset in part by interest income earned on the Company’s sweep and money market account balances. The Company incurred less interest expense during the first quarter of 2025 as compared to the first quarter of 2024 due to a lower principal balance under its 2025 Loan Agreement as compared to its prior borrowings with Oxford which were extinguished as a result of the spin-off transaction.
Net Loss. Net loss was $43.3 million during the first quarter of 2025, or $2.80 per share, basic and diluted, compared to a net loss of $78.7 million during the first quarter of 2024, or $5.77 per share, basic and diluted.

On May 14, 2025 AbbVie (NYSE: ABBV) and ADARx Pharmaceuticals, a late clinical-stage biotechnology company developing next-generation RNA therapeutics, reported a collaboration and license option agreement to develop small interfering RNA (siRNA) therapeutics across multiple disease areas, including neuroscience, immunology and oncology (Press release, AbbVie, MAY 14, 2025, View Source [SID1234653095]).

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siRNA represents a class of molecules capable of regulating gene expression and protein production. Unlike traditional modalities such as antibodies and small molecules, siRNA regulates the expression of genes. These molecules are designed to prevent the production of disease-causing proteins by targeting the messenger RNA (mRNA) that encodes for such proteins.

The strategic collaboration will leverage ADARx’s RNA discovery expertise and proprietary siRNA technology, which has the potential to enable sustained and precise mRNA silencing. AbbVie will contribute its expertise in antibody engineering, antibody drug conjugates (ADCs) and tissue delivery approaches as appropriate, to augment ADARx’s discovery efforts.

"siRNA is a promising genetic medicine approach for silencing disease-causing genes, but challenges still remain in targeting and delivering siRNA effectively," said Jonathon Sedgwick, Ph.D., senior vice president and global head, discovery research, AbbVie. "We are very pleased to collaborate with ADARx, leveraging their proprietary RNA technology alongside our antibody, ADC, and therapeutic area research and development expertise to bring siRNA forward as a potential novel therapeutic modality alongside our other established approaches. Together, we’re committed to developing innovative solutions for difficult-to-treat diseases across neuroscience, immunology and oncology."

"This collaboration with AbbVie further validates the differentiated RNA technology that we have developed at ADARx and has the potential to unlock tremendous clinical and commercial potential across multiple disease areas. AbbVie’s research and development expertise combined with its global commercial reach make them the ideal strategic collaborator to accelerate these programs for the potential benefit of patients worldwide," said Zhen Li, Ph.D., co-founder, president and chief executive officer of ADARx. "In addition to this strategic collaboration with AbbVie, ADARx continues to advance a deep pipeline of wholly-owned clinical-stage programs that span complement-mediated, cardiovascular and thrombotic diseases and various preclinical discovery programs including in obesity and neurodegeneration."

Under the terms of the agreement, ADARx will receive a $335 million upfront payment and will be eligible to receive several billion dollars in additional contingent payments including option-related fees and milestone payments, as well as tiered royalties.