On March 25, 2025 WuXi Biologics (Cayman) Inc. ("WuXi Biologics" or "the Group", stock code: 2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO) service company offering end-to-end solutions for biologics discovery, development and manufacturing, reported its audited annual results for the year ended December 31, 2024 ("Reporting Period") (Press release, WuXi Biologics, MAR 25, 2025, View Source [SID1234651428]).

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Financial Highlights

Revenue: For the year ended December 31, 2024, the Group’s revenue grew 9.6% YoY to RMB18,675.4 million, with non-COVID revenue increasing 13.1% YoY. The growth was primarily driven by: (i) the successful execution of the Group’s "Follow and Win the Molecule" strategies, coupled with the leading technology platform, best-in-industry timeline and excellent execution track record; (ii) enlarged spectrum of services offered to the biologics industry, fast growing technology platforms including ADCs and bispecific antibodies; (iii) growth of research services revenue generated from the Group’s various cutting-edge technologies; and (iv) the utilization of existing and newly expanded capacities, including ramp-up of the manufacturing sites in Europe.

Gross Profit and Gross Profit Margin: IFRS gross profit increased 12.1% YoY to RMB7,650.8 million, while adjusted gross profit rose 9.8% YoY to RMB8,479.5 million. IFRS gross profit margin reached 41.0%, with an adjusted gross profit margin of 45.4%. The increase in gross profit and margin was primarily due to the efficiency improvements driven from WBS and digitalization, and also the improvements of the ramp-up impacts from the recently commissioned facilities in Europe and the U.S. as expected.

EBITDA and EBITDA Margin: For the year ended December 31, 2024, EBITDA grew 16.7% to RMB6,547.8 million, while adjusted EBITDA increased 14.4% YoY to RMB7,999.3 million. EBITDA margin reached 35.1%, with an adjusted EBITDA margin of 42.8%.

Net Profit and Net Profit Attributable to Owners of the Company: IFRS net profit rose 10.5% YoY to RMB3,945.4 million. Net profit attributable to owners of the Company was RMB3,356.1 million, reflecting a slight YoY decline of 1.3%.

Adjusted Net Profit: Adjusted Net Profit for the period increased 9.0% YoY to RMB5,396.9 million. Adjusted net profit margin was 28.9%.

Basic Earnings Per Share (EPS): The Group’s basic earnings per share (EPS) remained flat at RMB 0.82 for the years ended December 31, 2024 and 2023. Diluted EPS slightly increased by 1.3% from RMB 0.77 for the year ended December 31, 2023 to RMB 0.78 for the year ended December 31, 2024.

Business Highlights

Integrated Project Wins
The Group added 151 integrated projects in 2024, bringing the total to 817. Notably, over half of the new projects originated from U.S. clients, reflecting strong client trusts and the Group’s resilience amid a dynamic environment. The Group supported 66 Phase III projects and 21 non-COVID commercial projects, strengthening its manufacturing pipeline.

During the Reporting Period, the Group continued to execute its "Win-the-Molecule" strategy, securing 20 post-IND projects, including 13 in late-phase and commercial stage, bringing total wins to 89 since 2018.

Research
By leveraging industry-leading technology platforms, the Group’s Research business has reached an inflection point following years of strategic cultivation. The Group has developed a portfolio of leading Immune Cell Engager (ICE) technologies, extending beyond traditional T-cell engagers (TCEs). Our diverse immune cell-targeting modalities—including CD3 Bispecific T Cell Engagers, Non-cytotoxic T Cell Engagers, γδ T Cell Engagers, NK Engagers, and Macrophage Engagers—are designed to unlock novel therapeutic potential in oncology and autoimmune diseases.

The Group is advancing these innovations in close collaboration with its clients and partners, driving the development of next-generation anti-tumor and autoimmune therapies. In 2024, the Group enabled seven global programs for molecule discovery and is eligible to receive approximately $140 million in near-term payments, with total potential payments exceeding $2.3 billion. To date, the Group’s Research Services has enabled 50+ programs that are potentially eligible for future milestone payments and sales royalties, establishing a consistent revenue and profit stream.

Development
The Group added 148 new development projects in 2024, further strengthening one of the industry’s largest portfolios of complex biologics. This portfolio includes 151 bispecifics & multispecifics, 194 ADCs, 80 fusion proteins and 24 vaccines. The Group continues to advance cutting-edge technology platforms that accelerate biologics development and manufacturing. Key platforms include WuXia (cell line development), WuXiDARx (drug-to-antibody ratio technology), WuXiHigh (high concentration and high-throughput DP Development), WuXiUI (ultra-intensified fed-batch platform) and WuXiUP (ultra-high productivity continuous bioprocessing platform).

Highlighting the unique advantages of its CRDMO platform and industry-leading technologies, the Group’s client, Curon Biopharmaceutical’s investigational B-cell depletion therapy, CN201, was acquired by Merck & Co., Inc. in 2024. This asset was discovered through the Group’s proprietary TCE and WuXiBody platforms, while WuXiUP effectively addresses complex CMC challenges.

The Group has shortened the development timeline for monoclonal antibody projects from DNA to IND to just nine months and successfully supported over 600 IND applications by the end of the Reporting Period. These advancements help clients accelerate development cycles and achieve cost-effective solutions across the biologics value chain.

Manufacturing
In 2024, the Group supported 66 Phase III projects and 21 non-COVID commercial manufacturing projects, completed 16 process performance qualification (PPQ) projects, with 24 scheduled for 2025. The Group also achieved a DS and DP PPQ success rate exceeding 98%, establishing a solid foundation for commercial manufacturing operations.

To further support its growing commercial pipeline and meet client needs, the Group continues to execute its "Global Dual Sourcing" strategy, providing comprehensive manufacturing services through its global network.
Ireland: All three facilities have secured GMP certification from the Irish Health Products Regulatory Authority (HPRA) and completed multiple 16,000-liter PPQ runs. The site also initiated commercial production in 2024.
Singapore: The lifting of fabricated modules for XDC’s Production Facility has been completed, with critical utilities in the final phase of design and construction. Additionally, significant progress has been made in the design of Biologics’ Production Assets.
U.S: The Group continues the expansion of MFG11 at Worcester, MA, one of the largest single-use-technology facilities in the U.S., featuring six 6,000L upstream tanks connected to a single downstream line, with high-throughput processing and extensive automation. Upon completion, MFG11 will be integrated with MFG18 (Cranbury, NJ), and Boston Research Service Center, enabling the Group to offer end-to-end capabilities in the U.S., from research, development, clinical manufacturing, to both small- and large-scale commercial manufacturing.

Asset optimization
In January 2025, the Group and MSD International GmbH entered into an agreement for the asset transfer of WuXi Vaccine’s Dundalk, Ireland facility. This transaction enables MSD International GmbH to better integrate vaccine production within its global network while enhancing the Group’s operational flexibility, asset efficiency and margins. The Group will focus on its vaccines CDMO services from WuXi Vaccines’ Suzhou, China site.

Backlog
As of December 31, 2024, total backlog stood at US$18.5 billion, comprising US$10.5 billion in service backlog and US$8.0 billion in potential milestones. Backlog within 3 years was US$3.7 billion. Following the announced asset transaction with MSD International GmbH, approximately US$3 billion in services backlog was removed. Adjusting for this divestiture, backlog grew by approximately US$0.9 billion YoY.

Quality
The Group remains committed to the highest quality standards, safeguarding the interests of clients and patients. Backed by a world-class quality system, the Group has successfully completed 42 regulatory inspections by various national regulatory agencies since 2017, including 22 by EU EMA and U.S. FDA, with no critical issues and zero data integrity findings. In Q4 2024, the Group successfully passed the HPRA inspection in Ireland with no critical observations. These regulatory milestones further validate the Group’s premier quality system, which adheres to the highest global quality standards.

Talents
People are WuXi Biologics’ greatest assets. As of December 31, 2024, the Group’s total employee count reached 12,575, including 4,383 scientists, with a key talent retention rate of 95.8%. The Group’s successful global recruitment efforts strengthened its worldwide operations, enabling it to efficiently deliver project commitments and drive continuous innovation.

WBS (WuXi Biologics Business System)
The Group has been continuously focusing on its WBS initiatives, driving operational excellence and efficiency. In 2024, the Group executed over 260 WBS Kaizen events, achieving 1-point improvement in gross profit margin through cost-savings, enhanced labor productivities, and inventory reductions. Additionally, ESG-focused Kaizen projects reduced carbon emissions, water usage, waste generation, and material consumption. The Group remains committed to establishing WBS as a lean management system, fostering continuous improvement, talent development, and further enhancing value creation for its clients.

Sustainability
The Group has integrated Sustainability as a core pillar of its business growth strategy, earning widespread recognition from leading ESG rating agencies and institutional investors. Notable achievements encompass the inclusion in the Dow Jones Sustainability Indices, an AAA rating from MSCI ESG Ratings, a Platinum Medal from EcoVadis, and recognition as an ESG Industry Top-Rated and APAC Regional Top-Rated Company by Sustainalytics.

During the Reporting Period, the Group was also selected in the S&P Global Sustainability Yearbook 2024, MSCI ESG Leaders Indexes 2024, FTSE4Good Index Series, and Hang Seng ESG 50 Index.
Management Comment

Dr. Chris Chen, CEO of WuXi Biologics, stated, "In 2024, we navigated many crosscurrents in the macro environment, remained resilient and delivered a solid 9.6% YoY growth in the Group revenue. Empowered by our unique CRDMO business model and well-established ‘Follow and Win the Molecule’ strategies, we are confident in our ability to provide unparalleled support to our clients, ensuring the delivery of sustainable outcomes in 2025 and beyond."

Dr. Chen added, "Looking ahead, we anticipate accelerated and profitable growth in 2025 and beyond, driven by strong performances across all Research, Development, and Manufacturing platforms. With enhanced operational efficiencies, increasing adoption of next-generation technology platforms, and disciplined execution of our strategic initiatives, we are well-positioned to capture new opportunities and drive innovation."

Dr. Ge Li, Chairman of WuXi Biologics, concluded, "In 2024, we remained steadfast in our mission and vision – to empower anyone and any company to discover, develop and manufacture biologics from concept to commercial manufacturing. As we look ahead, we are committed to delivering exceptional value to our partners, reinforcing our position as a premier, one-stop service provider for the biologics industry, and advancing our vision that ‘every biologic can be made’."

Key Financial Ratios

(For the Twelve Months Ended Dec. 31)

Key Financial Ratio

2024

2023

Change

Revenue (In RMB million)

18,675.4

17,034.3

9.6 %

Gross Profit (In RMB million)

7,650.8

6,827.9

12.1 %

Margin (%)

41.0 %

40.1 %

Net Profit (In RMB million)

3,945.4

3,570.6

10.5 %

Margin (%)

21.1 %

21.0 %

Net Profit Attributable to Owners of
the Company (In RMB million)

3,356.1

3,399.7

(1.3 %)

Margin (%)

18.0 %

20.0 %

Adjusted Net Profit (In RMB million)

5,396.9

4,950.4

9.0 %

Margin (%)

28.9 %

29.1 %

EBITDA (In RMB million)

6,547.8

5,613.2

16.7 %

Margin (%)

35.1 %

33.0 %

Adjusted EBITDA (In RMB million)

7,999.3

6,993.0

14.4 %

Margin (%)

42.8 %

41.1 %

Adjusted Basic EPS (In RMB)

1.17

1.13

3.5 %

On March 25, 2025 2seventy bio, Inc. (Nasdaq: TSVT), reported financial results and recent highlights for the fourth quarter and full year ended December 31, 2024 (Press release, 2seventy bio, MAR 25, 2025, View Source [SID1234651444]).

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"2024 was a pivotal year for 2seventy as we made significant changes to our business to streamline cost structure and focus solely on Abecma," said Chip Baird, chief executive officer, 2seventy bio. "This week marks four years since Abecma received FDA approval as the first anti-BCMA CAR T cell therapy approved for relapsed or refractory multiple myeloma. Together with BMS, we remain committed to expanding the reach of this important therapy. We launched 2seventy with the goal of providing more time to patients, and we believe with BMS’ experience and resources, we can continue to improve outcomes for people living with multiple myeloma."

On March 10, 2seventy bio announced a definitive merger agreement to be acquired by Bristol Myers Squibb (BMS). Under the terms of the agreement, BMS will commence a tender offer to acquire all outstanding shares of 2seventy bio at a price of $5.00 per share in an all-cash transaction. 2seventy bio’s Board of Directors unanimously recommends that 2seventy bio stockholders tender their shares in the tender offer.

ABECMA COMMERCIAL AND REGULATORY HIGHLIGHTS

Full year Abecma (idecabtagene vicleucel; ide-cel) U.S. sales, as reported by Bristol Myers Squibb (BMS), were $242 million.
2seventy bio and BMS continue to focus on competitively differentiating Abecma’s safety and efficacy profile supported by the strength of the KarMMa-3 and real-world data.
The Company and BMS share equally in all profits and losses related to development, manufacturing, and commercialization of Abecma in the U.S. 2seventy bio reported share of collaboration loss of approximately $3.3 million related to the collaboration with BMS for the three months ended December 31, 2024.
SELECT FOURTH QUARTER AND FULL YEAR 2024 FINANCIAL RESULTS

Total revenues were $2.9 million for the three months ended December 31, 2024, compared to $10.7 million for the three months ended December 31, 2023. Total revenues were $37.9 million for the twelve months ended December 31, 2024, compared to $100.4 million for the twelve months ended December 31, 2023.
Research and development expenses were $8.7 million for the three months ended December 31, 2024, compared to $51.2 million for the three months ended December 31, 2023. Research and development expenses were $76.9 million for the twelve months ended December 31, 2024, compared to $230.8 million for the twelve months ended December 31, 2023.
Selling, general and administrative expenses were $8.5 million for the three months ended December 31, 2024, compared to $16.2 million for the three months ended December 31, 2023. Selling, general and administrative expenses were $43.9 million for the twelve months ended December 31, 2024, compared to $69.4 million for the twelve months ended December 31, 2023.
Net loss was $19.5 million for the three months ended December 31, 2024, compared to $56.8 million for the three months ended December 31, 2023. Net loss was $57.2 million for the twelve months ended December 31, 2024, compared to $217.6 million for the twelve months ended December 31, 2023.
Cash, cash equivalents, and marketable securities totaled $183.6 million as of December 31, 2024.
Merger Agreement Details and Path to Completion
The closing of the transaction with BMS is expected to occur in the second quarter of 2025 and is subject to customary closing conditions, including the tender of a majority of the outstanding shares of 2seventy bio’s common stock and the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976. Following the successful closing of the tender offer, BMS will acquire all remaining shares of 2seventy bio common stock that are not tendered in the tender offer through a second-step merger at the same price in the tender offer of $5.00 per share.

Following the completion of this transaction, 2seventy bio’s common stock will no longer be listed for trading on Nasdaq.

In connection with the execution of the merger agreement, certain stockholders of 2seventy bio owning approximately 5.3% of the outstanding shares of 2seventy bio’s common stock have entered into tender and support agreements pursuant to which they have agreed to tender all of their owned shares in the offer.

In light of the announced transaction, 2seventy will not be hosting an earnings conference call or providing financial guidance for 2025.

ABECMA U.S. INDICATION
ABECMA is a B-cell maturation antigen (BCMA)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma after two or more prior lines of therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.

U.S. Important Safety Information

BOXED WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES, HLH/MAS, PROLONGED CYTOPENIA and SECONDARY HEMATOLOGICAL MALIGNANCIES

Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients following treatment with ABECMA. Do not administer ABECMA to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab or tocilizumab and corticosteroids.
Neurologic Toxicities, which may be severe or life-threatening, occurred following treatment with ABECMA, including concurrently with CRS, after CRS resolution, or in the absence of CRS. Monitor for neurologic events after treatment with ABECMA. Provide supportive care and/or corticosteroids as needed.
Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome (HLH/MAS) including fatal and life-threatening reactions, occurred in patients following treatment with ABECMA. HLH/MAS can occur with CRS or neurologic toxicities.
Prolonged Cytopenia with bleeding and infection, including fatal outcomes following stem cell transplantation for hematopoietic recovery, occurred following treatment with ABECMA.
T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including ABECMA
ABECMA is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the ABECMA REMS.
Warnings and Precautions:

Early Death: In KarMMa-3, a randomized (2:1), controlled trial, a higher proportion of patients experienced death within 9 months after randomization in the ABECMA arm (45/254; 18%) compared to the standard regimens arm (15/132; 11%). Early deaths occurred in 8% (20/254) and 0% prior to ABECMA infusion and standard regimen administration, respectively, and 10% (25/254) and 11% (15/132) after ABECMA infusion and standard regimen administration, respectively. Out of the 20 deaths that occurred prior to ABECMA infusion, 15 occurred from disease progression, 3 occurred from adverse events and 2 occurred from unknown causes. Out of the 25 deaths that occurred after ABECMA infusion, 10 occurred from disease progression, 11 occurred from adverse events, and 4 occurred from unknown causes.

Cytokine Release Syndrome (CRS): CRS, including fatal or life-threatening reactions, occurred following treatment with ABECMA. Among patients receiving ABECMA for relapsed refractory multiple myeloma in the KarMMa and KarMMa-3 studies (N=349), CRS occurred in 89% (310/349), including ≥ Grade 3 CRS (Lee grading system) in 7% (23/349) of patients and Grade 5 CRS in 0.9% (3/349) of patients. The median time-to-onset of CRS, any grade, was 1 day (range: 1 to 27 days), and the median duration of CRS was 5 days (range: 1 to 63 days). In the pooled studies, the rate of ≥Grade 3 CRS was 10% (7/71) for patients treated in dose range of 460 to 510 x 106 CAR-positive T cells and 5.4% (13/241) for patients treated in dose range of 300 to 460 x 106 CAR-positive T cells.

The most common manifestations of CRS (greater than or equal to 10%) included pyrexia (87%), hypotension (30%), tachycardia (26%), chills (19%), hypoxia (16%). Grade 3 or higher events that may be associated with CRS include hypotension, hypoxia, hyperbilirubinemia, hypofibrinogenemia, ARDS, atrial fibrillation, hepatocellular injury, metabolic acidosis, pulmonary edema, coagulopathy, renal failure, multiple organ dysfunction syndrome and HLH/MAS.

Identify CRS based on clinical presentation. Evaluate for and treat other causes of fever, hypoxia, and hypotension. CRS has been reported to be associated with findings of HLH/MAS, and the physiology of the syndromes may overlap. HLH/MAS is a potentially life-threatening condition. In patients with progressive symptoms of CRS or refractory CRS despite treatment, evaluate for evidence of HLH/MAS.

Of the 349 patients who received ABECMA in clinical trials, 226 (65%) patients received tocilizumab; 39% (135/349) received a single dose, while 26% (91/349) received more than 1 dose of tocilizumab. Overall, 24% (82/349) of patients received at least 1 dose of corticosteroids for treatment of CRS. Almost all patients who received corticosteroids for CRS also received tocilizumab. For patients treated in dose range of 460 to 510 x 106 CAR-positive T cells, 76% (54/71) of patients received tocilizumab and 35% (25/71) received at least 1 dose of corticosteroids for treatment of CRS. For patients treated in dose range of 300 to 460 x 106 CAR-positive T cells, 63% (152/241) of patients received tocilizumab and 20% (49/241) received at least 1 dose of corticosteroid for treatment of CRS.

Monitor patients at least daily for 7 days following ABECMA infusion at the REMS-certified healthcare facility for signs or symptoms of CRS and monitor patients for signs or symptoms of CRS for at least 4 weeks after ABECMA infusion. At the first sign of CRS, institute treatment with supportive care, tocilizumab and/or corticosteroids as indicated. Ensure that a minimum of 2 doses of tocilizumab are available prior to infusion of ABECMA. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time.

Neurologic Toxicities: Neurologic toxicities, including immune-effector cell-associated neurotoxicity (ICANS), which may be severe or life- threatening, occurred concurrently with CRS, after CRS resolution, or in the absence of CRS following treatment with ABECMA.

In patients receiving ABECMA in the KarMMa and KarMMa-3 studies, CAR T cell-associated neurotoxicity occurred in 40% (139/349), including Grade 3 in 4% (14/349) and Grade 4 in 0.6% (2/349) of patients. The median time to onset of neurotoxicity was 2 days (range: 1 to 148 days). The median duration of CAR T cell-associated neurotoxicity was 8 days (range: 1 to 720 days) in all patients including those with ongoing neurologic events at the time of death or data cut off. CAR T cell-associated neurotoxicity resolved in 123 of 139 (88%) patients and median time to resolution was 5 days (range: 1 to 245 days). One-hundred and thirty four out of 349 (38%) patients with neurotoxicity had CRS. The onset of neurotoxicity during CRS was observed in 93 patients, before the onset of CRS in 12 patients, and after the CRS event in 29 patients. The rate of Grade 3 or 4 CAR T cell-associated neurotoxicity was 5.6% (4/71) and 3.7% (9/241) for patients treated in dose range of 460 to 510 x 106 CAR-positive T cells and 300 to 460 x 106 CAR-positive T cells, respectively. The most frequent (greater than or equal to 5%) manifestations of CAR T cell-associated neurotoxicity include encephalopathy (21%), headache (15%), dizziness (8%), delirium (6%), and tremor (6%).

At the safety update for KarMMa-3 study, one patient developed fatal neurotoxicity 43 days after ABECMA. In KarMMa, one patient had ongoing Grade 2 neurotoxicity at the time of death. Two patients had ongoing Grade 1 tremor at the time of data cutoff.

Cerebral edema has been associated with ABECMA in a patient in another study in multiple myeloma. Grade 3 myelitis and Grade 3 parkinsonism have occurred after treatment with ABECMA in another study in multiple myeloma.

Monitor patients at least daily for 7 days following ABECMA infusion at the REMS-certified healthcare facility for signs or symptoms of neurologic toxicities and monitor patients for signs or symptoms of neurologic toxicities for at least 4 weeks after ABECMA infusion and treat promptly. Rule out other causes of neurologic symptoms. Neurologic toxicity should be managed with supportive care and/or corticosteroids as needed. Counsel patients to seek immediate medical attention should signs or symptoms occur at any time.

Hemophagocytic Lymphohistiocytosis (HLH)/Macrophage Activation Syndrome (MAS): In patients receiving ABECMA in the KarMMa and KarMMa-3 studies, HLH/MAS occurred in 2.9% (10/349) of patients. All events of HLH/MAS had onset within 10 days of receiving ABECMA, with a median onset of 6.5 days (range: 4 to 10 days) and occurred in the setting of ongoing or worsening CRS. Five patients with HLH/MAS had overlapping neurotoxicity. The manifestations of HLH/MAS include hypotension, hypoxia, multiple organ dysfunction, renal dysfunction and cytopenia.

In KarMMa-3, one patient had Grade 5, two patients had Grade 4 and two patients had Grade 3 HLH/MAS. The patient with Grade 5 HLH/MAS also had Grade 5 candida sepsis and Grade 5 CRS. In another patient who died due to stroke, the Grade 4 HLH/MAS had resolved prior to death. Two cases of Grade 3 and one case of Grade 4 HLH/MAS had resolved.

In KarMMa, one patient treated in the 300 x 106 CAR-positive T cells dose cohort developed fatal multi-organ HLH/MAS with CRS. In another patient with fatal bronchopulmonary aspergillosis, HLH/MAS was contributory to the fatal outcome. Three cases of Grade 2 HLH/MAS resolved.

HLH/MAS is a potentially life-threatening condition with a high mortality rate if not recognized early and treated. Treatment of HLH/MAS should be administered per institutional guidelines.

ABECMA REMS: Due to the risk of CRS and neurologic toxicities, ABECMA is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the ABECMA REMS. Further information is available at www.AbecmaREMS.com or contact Bristol-Myers Squibb at 1-866-340-7332.

Hypersensitivity Reactions: Allergic reactions may occur with the infusion of ABECMA. Serious hypersensitivity reactions, including anaphylaxis, may be due to dimethyl sulfoxide (DMSO) in ABECMA.

Infections: ABECMA should not be administered to patients with active infections or inflammatory disorders. Severe, life-threatening, or fatal infections occurred in patients after ABECMA infusion.

In all patients receiving ABECMA in the KarMMa and KarMMa-3 studies, infections (all grades) occurred in 61% of patients. Grade 3 or 4 infections occurred in 21% of patients. Grade 3 or 4 infections with an unspecified pathogen occurred in 12%, viral infections in 7%, bacterial infections in 4.3%, and fungal infections in 1.4% of patients. Overall, 15 patients had Grade 5 infections (4.3%); 8 patients (2.3%) with infections of pathogen unspecified, 3 patients (0.9%) with fungal infections, 3 patients (0.9%) with viral infections, and 1 patient (0.3%) with bacterial infection.

Monitor patients for signs and symptoms of infection before and after ABECMA infusion and treat appropriately. Administer prophylactic, pre-emptive, and/or therapeutic antimicrobials according to standard institutional guidelines.

Febrile neutropenia was observed in 38% (133/349) of patients after ABECMA infusion and may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated.

Viral Reactivation: Cytomegalovirus (CMV) infection resulting in pneumonia and death has occurred following ABECMA administration. Monitor and treat for CMV reactivation in accordance with clinical guidelines. Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against plasma cells. Perform screening for CMV, HBV, hepatitis C virus (HCV), and human immunodeficiency virus (HIV) in accordance with clinical guidelines before collection of cells for manufacturing. Consider antiviral therapy to prevent viral reactivation per local institutional guidelines/clinical practice.

Prolonged Cytopenias: In patients receiving ABECMA in the KarMMa and KarMMa-3 studies, 40% of patients (139/349) experienced prolonged Grade 3 or 4 neutropenia and 42% (145/349) experienced prolonged Grade 3 or 4 thrombocytopenia that had not resolved by Month 1 following ABECMA infusion. In 89% (123/139) of patients who recovered from Grade 3 or 4 neutropenia after Month 1, the median time to recovery from ABECMA infusion was 1.9 months. In 76% (110/145) of patients who recovered from Grade 3 or 4 thrombocytopenia, the median time to recovery was 1.9 months. Five patients underwent stem cell therapy for hematopoietic reconstitution due to prolonged cytopenia. The rate of Grade 3 or 4 thrombocytopenia was 62% (44/71) and 56% (135/241) for patients treated in dose range of 460 to 510 x 106 CAR-positive T cells and 300 to 460 x 106 CAR-positive T cells, respectively.

Monitor blood counts prior to and after ABECMA infusion. Manage cytopenia with myeloid growth factor and blood product transfusion support according to local institutional guidelines.

Hypogammaglobulinemia: In all patients receiving ABECMA in the KarMMa and KarMMa-3 studies, hypogammaglobulinemia was reported as an adverse event in 13% (46/349) of patients; laboratory IgG levels fell below 500 mg/dL after infusion in 37% (130/349) of patients treated with ABECMA.

Hypogammaglobulinemia either as an adverse reaction or laboratory IgG level below 500 mg/dL after infusion occurred in 45% (158/349) of patients treated with ABECMA. Forty-one percent of patients received intravenous immunoglobulin (IVIG) post-ABECMA for serum IgG <400 mg/dL.

Monitor immunoglobulin levels after treatment with ABECMA and administer IVIG for IgG <400 mg/dl. Manage appropriately per local institutional guidelines, including infection precautions and antibiotic or antiviral prophylaxis.

Use of Live Vaccines: The safety of immunization with live viral vaccines during or after ABECMA treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during ABECMA treatment, and until immune recovery following treatment with ABECMA.

Secondary Malignancies: Patients treated with ABECMA may develop secondary malignancies. In KarMMa-3, myeloid neoplasms (four cases of myelodysplastic syndrome and one case of acute myeloid leukemia) occurred in 2.2% (5/222) of patients following treatment with ABECMA compared to none in the standard regimens arm at the time of the safety update. The median time to onset of myeloid neoplasm from ide-cel infusion was 338 days (Range: 277 to 794 days). Three of these five patients have died following the development of myeloid neoplasm. One out of the five cases of myeloid neoplasm occurred after initiation of subsequent antimyeloma therapy.

T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including ABECMA. Mature T cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusion, and may include fatal outcomes.

Monitor life-long for secondary malignancies. In the event that a secondary malignancy occurs, contact Bristol-Myers Squibb at 1‑888‑805‑4555 for reporting and to obtain instructions on collection of patient samples for testing of secondary malignancy.

Effects on Ability to Drive and Operate Machinery: Due to the potential for neurologic events, including altered mental status or seizures, patients receiving ABECMA are at risk for altered or decreased consciousness or coordination in the 8 weeks following ABECMA infusion. Advise patients to refrain from driving and engaging in hazardous occupations or activities, such as operating heavy or potentially dangerous machinery, during this initial period.

Adverse Reactions: The most common nonlaboratory adverse reactions (incidence greater than or equal to 20%) include pyrexia, CRS, hypogammaglobulinemia, infections – pathogen unspecified, musculoskeletal pain, fatigue, febrile neutropenia, hypotension, tachycardia, diarrhea, nausea, headache, chills, upper respiratory tract infection, encephalopathy, edema, dyspnea and viral infections.

Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide.

On March 25, 2025 Aprea Therapeutics, Inc. (Nasdaq: APRE) ("Aprea", or the "Company"), a clinical-stage biopharmaceutical company developing innovative treatments that exploit specific cancer cell vulnerabilities while minimizing damage to healthy cells, reported financial results for the fourth quarter and full year ended December 31, 2024, and provided a business update (Press release, Aprea, MAR 25, 2025, View Source [SID1234651393]).

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"We made excellent progress across our pipeline in 2024, laying a strong foundation for the year ahead," said Oren Gilad, Ph.D., President and Chief Executive Officer of Aprea. "We continue to enroll patients in the ACESOT-1051 trial evaluating our WEE1 kinase inhibitor, APR-1051, which we believe has best in class potential. The compound appears safe and well tolerated to date with no hematologic toxicity. We look forward to reporting open label data from ACESOT-1051 in the second half of the year. We are also advancing ATRN-119, our highly selective first-in-class macrocyclic ATR inhibitor. The ongoing ABOYA-119 trial is now evaluating ATRN-119 as continuous once daily and twice daily monotherapy in order to maximize therapeutic benefit. Our ultimate goal is to transform the treatment paradigm for difficult to treat cancers by unlocking the full potential of DDR-based therapies."

Key Business Updates and Potential Upcoming Key Milestones

ACESOT-1051: A Biomarkers Focused, Phase 1 Trial of Oral WEE1 inhibitor, APR-1051

● APR-1051 is a potent and selective small molecule that has been designed to potentially solve tolerability challenges of the WEE1 class and may achieve greater clinical activity than other programs currently in development. Aprea is advancing APR-1051 as monotherapy in cancers with Cyclin E over-expression, as well as other biomarkers that may predict sensitivity to WEE1 inhibition. Cancers over-expressing Cyclin E represent a high unmet medical need. Patients with Cyclin E over-expression have poor prognosis and, currently, have no effective therapies available.
● Patients are now being enrolled in Cohort 5 (70 mg dose) of the ACESOT-1051 (A Multi-Center Evaluation of WEE1 Inhibitor in Patients with Advanced Solid Tumors, APR-1051) trial. This Phase 1 clinical trial is evaluating single-agent APR-1051 in advanced solid tumors harboring cancer-associated gene alterations. No hematological toxicities have been observed to date. The primary objectives of the Phase 1 study are to measure safety, dose-limiting toxicities (DLTs), maximum tolerated dose or maximum administered dose (MTD/MAD), and recommended Phase 2 dose (RP2D); secondary objectives are to evaluate pharmacokinetics and preliminary efficacy according to RECIST or PCWG3 criteria; pharmacodynamic parameters are exploratory objectives.

● In October 2024, preliminary findings from the ACESOT-1051 trial were reported in a poster at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics, in Barcelona, Spain. The poster can be viewed on Aprea’s corporate website here.
● Preliminary efficacy data from ACESOT-1051 are expected in the second half of 2025. For more information, refer to ClinicalTrials.gov NCT06260514.

ABOYA-119: Ongoing Clinical Trial Evaluating ATR inhibitor, ATRN-119

● ATRN-119 is a potent and highly selective first-in-class macrocyclic ATR inhibitor, designed to be used in patients with mutations in DDR-related genes. Cancers with mutations in DDR-related genes represent a high unmet medical need. Patients with DDR-related gene mutations have a poor prognosis and, currently, there are no effective therapies available for them.
● ATRN-119 is being evaluated in the open-label Phase 1/2a clinical trial of ABOYA-119 as monotherapy in patients with advanced solid tumors having at least one mutation in a defined panel of DDR-related genes. Patients are currently being enrolled at Dose Level 7, with both 1100 mg once daily and 550 mg twice daily doses being evaluated independently and in parallel. The addition of twice daily dosing was implemented to potentially optimize ATRN-119’s activity across a 24-hour cycle thereby providing better target coverage and maximal clinical benefit. This is expected to increase the likelihood of achieving superior clinical outcomes and may potentially accelerate the path to regulatory approval and commercialization.
● An update from the ABOYA-119 trial was provided in a poster at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics in October, 2024. A copy of the poster can be viewed here.
● For more information on ABOYA-119, please refer to clinicaltrials.gov NCT04905914.

Corporate

● Aprea engaged Philippe Pultar, MD in October 2024 as senior medical advisor to support the development and advancement of APR-1051. Dr. Pultar is a seasoned pharmaceutical executive with extensive experience in oncology. He was most recently employed at Zentalis Pharmaceuticals where he played a key role in the strategy and execution of the global clinical development of azenosertib, a WEE1 inhibitor.

Select Financial Results for the Fourth Quarter ended December 31, 2024

● For the quarter ended December 31, 2024, the Company reported an operating loss of $3.2 million, compared to an operating loss of $3.7 million in the fourth quarter of 2023.
● Research and Development (R&D) expenses were $2.4 million for the quarter ended December 31, 2024, compared to $2.0 million for the fourth quarter of 2023. The increase in R&D expense was primarily related to an increase in personnel costs primarily related to new hires and severance.
● General and Administrative (G&A) expenses were $1.1 million for the quarter ended December 31, 2024, compared to $1.6 million for the comparable period in 2023.
● The Company reported a net loss of $2.9 million ($0.49 per basic share) on approximately 6.0 million weighted-average common shares outstanding for the quarter ended December 31, 2024, compared to a net loss of $3.4 million ($0.92 per basic share) on approximately 3.7 million weighted average common shares outstanding for the comparable period in 2023.

Select Financial Results for the Year ended December 31, 2024

● As of December 31, 2024, the Company reported cash and cash equivalents of $22.8 million compared to $21.6 million as of December 31, 2023. The Company believes its cash and cash equivalents as of December 31, 2024 will be sufficient to meet its currently projected operating expenses and capital expenditure requirements into the first quarter of 2026.
● For the year ended December 31, 2024, the Company reported an operating loss of $14.3 million, compared to an operating loss of $15.5 million for the year ended December 31, 2023.
● R&D expenses were $9.4 million for the year ended December 31, 2024, compared to $7.6 million for the year ended December 31, 2023. The increase in R&D expense was primarily related to the ABOYA-119 clinical trial to evaluate ATRN-119, the initiation of the ACESOT-1051 clinical trial to evaluate APR-1051 and an increase in personnel costs primarily related to new hires and severance.
● G&A expenses were $6.5 million for the year ended December 31, 2024, compared to $8.4 million for the year ended December 31, 2023. The decrease in G&A expenses was primarily due to a decrease in personnel costs primarily related to severance expense for former executives and insurance premiums.
● The Company reported a net loss of $13.0 million ($2.35 per basic share) on approximately 5.5 million weighted-average common shares outstanding for the year ended December 31, 2024, compared to a net loss of $14.3 million ($3.95 per basic share) on approximately 3.6 million weighted average common shares outstanding for the comparable period in 2023.

On March 25, 2025 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company developing novel small molecule therapeutics to treat unmet needs in immune-mediated diseases, reported positive topline results from the PORTOLA Phase 2a clinical trial evaluating zetomipzomib, a novel, first-in-class selective immunoproteasome inhibitor, in patients with autoimmune hepatitis (AIH) and reported fourth quarter and year end 2024 financial results (Press release, Kezar Life Sciences, MAR 25, 2025, View Source [SID1234651404]).
"We are pleased to announce these exciting results from the PORTOLA trial, the first successful randomized study in treatment-refractory AIH," said Chris Kirk, CEO and co-founder of Kezar. "We are encouraged by the safety and efficacy data in this difficult-to-treat patient population, specifically durable and steroid-sparing remissions experienced by patients treated with zetomipzomib. We are eager to work with the FDA Division of Hepatology and Nutrition to remove the partial clinical hold and align on an appropriate trial design to demonstrate the clinical benefit of zetomipzomib in AIH. I’d like to thank the team at Kezar, the physicians and staff at our clinical trial sites, and most importantly, the patients and their caregivers, for their participation, sacrifice and hard work."

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"I am impressed by the totality of efficacy and safety data from the PORTOLA study," said Gideon Hirschfield, Chair of Autoimmune Liver Disease Research and Director of the Francis Family Liver Clinic, at the Toronto General Hospital. "Zetomipzomib represents a potent and targeted therapy for patients, and I believe these results will positively contribute to the design of a registrational trial of zetomipzomib in AIH, where patients are in need of better treatment options."
PORTOLA is a placebo-controlled, randomized, double-blind Phase 2a clinical trial evaluating the efficacy and safety of zetomipzomib in patients with AIH that are insufficiently responding to standard of care or

have relapsed from a previous CR. The trial enrolled 24 patients, randomized (2:1) to receive 60 mg of zetomipzomib or placebo in addition to background therapy for 24 weeks, with a protocol-suggested steroid taper. All patients were required to receive a starting daily steroid dose of 20-40 mg/day of prednisone (or budesonide equivalent) and physicians were encouraged to taper daily steroid usage to 5 mg/day consistent with AIH treatment guidelines established by the American Association for the Study of Liver Diseases (AASLD).
The primary efficacy endpoint of PORTOLA, which was not powered for efficacy, evaluated the proportion of patients who achieved a CR by Week 24, measured as normalization of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and Immunoglobulin G (IgG) values (if elevated at baseline), with steroid dose levels not higher than baseline. A key secondary endpoint was the proportion of patients who achieved a CR by Week 24 with a steroid dose of 5 mg/day or less. The PORTOLA trial included a pre-specified subgroup analysis of patients who were on steroid-based therapy at the time of screening, which represents a patient population with unmet medical needs for a potential future registrational study. Additional exploratory endpoints included changes in histologic assessment of AIH as measured in biopsies taken within six months of screening and repeated at Week 24 of the trial.
Summary of Topline Results
PORTOLA enrolled 24 patients as the ITT population. Consistent with the AASLD treatment goals, zetomipzomib treatment resulted in higher rates of complete biochemical responses (CR) combined with reduction of steroid dosage to 5 mg/day or less, compared to placebo. In the ITT population:
•Without regard to steroid taper, 50.0% (8 of 16) of zetomipzomib patients achieved a CR, compared to 37.5% (3 of 8) of placebo patients.
•31.3% (5 of 16) of zetomipzomib patients achieved both a CR and steroid taper to 5 mg/day or less, compared to 12.5% (1 of 8) of placebo patients.
•18.8% (3 of 16) of zetomipzomib patients achieved both a CR and complete steroid withdrawal to zero mg/day, compared to 0% (0 of 8) of placebo patients.
•Median duration of response in zetomipzomib patients achieving a CR was 27.6 weeks (including the ongoing open-label extension), and no disease flares were reported in any zetomipzomib-treated patient achieving CR. Disease flares were defined as a sustained increase in ALT values to 25% above the CR value or 1.25-fold higher than the upper limit of normal for more than one week and requiring a restart or increase in steroid dose.
In the pre-specified subgroup analysis, 21 of the 24 patients entered the study on a steroid-based therapy at the time of screening. One patient in the placebo arm and two patients in the zetomipzomib arm were not receiving steroids at screening. All patients on study were required to initiate treatment with an initial prednisone dose of 20–40 mg/day. Of the 21 patients in this subgroup analysis:
•Median steroid use at screening was 20 mg/day for patients enrolled in the zetomipzomib arm, compared to 10 mg/day in the placebo arm, indicating that the zetomipzomib-treated arm was more refractory than the placebo arm.
•Without regard to steroid taper, 57.1% (8 of 14) of zetomipzomib patients achieved a CR, compared to 28.6% (2 of 7) of placebo patients.

•35.7% (5 of 14) of patients on the zetomipzomib arm achieved a CR and steroid taper to 5 mg/day or less, compared to 0% (0 of 7) of placebo patients.
•21.4% (3 of 14) of patients on the zetomipzomib arm achieved a CR and complete steroid withdrawal to zero mg/day, compared to 0% (0 of 7) of placebo patients.
Safety
Treatment-emergent adverse events (TEAEs) were seen in all patients, with injection site reactions (ISRs) being the most commonly reported TEAE in both arms. Systemic injection reactions (SIRs), with onset occurring 8 to 24 hours post-dose and usually resolving within 48 hours, were all Grade 1 and Grade 2. SIRs are a protocol-defined set of specific adverse events (AEs) consisting of one or more of the following signs/symptoms: hypotension, tachycardia, nausea, vomiting, dizziness, headache, pyrexia, rigors and/or chills.
Three patients experienced treatment-emergent serious adverse events (SAEs): one in the placebo arm, a Grade 3 variceal bleeding with hematemesis and atrial fibrillation; and two in the zetomipzomib arm, a Grade 3 fever occurring after the Week 24 liver biopsy, and a Grade 3 influenza infection that fully resolved during study. All SAEs were considered unrelated to study treatment, and all three patients completed the double-blind treatment period. Infectious AEs were reported in 85.7% (6 of 7) of patients in the placebo arm and 56.3% (9 of 16) of patients in the zetomipzomib arm. One patient discontinued from the placebo arm for a UTI requiring antibiotic treatment prior to receiving a dose on study, and three patients discontinued on the zetomipzomib arm for a Grade 1 fatigue, Grade 2 hives and a Grade 2 AIH disease flare. The safety population (n=23) includes all patients who received at least one dose of study treatment:

Placebo Zetomipzomib
n=7 n=16
Adverse Events in Double-blind Treatment Period n (%) n (%)
Participants with at least 1 Treatment Emergent Adverse Event (TEAE) 7 (100.0) 16 (100.0)
Most common TEAE:
Injection Site Reaction (ISR) 4 (57.1) 15 (93.8)
Systemic Injection Reaction (SIR) 1 (14.3) 12 (75.0)
TEAE leading to study drug discontinuation 0 (0) 3 (18.8)
Grade 3 TEAE (no Grade 4 or 5 TEAEs reported) 1 (14.3)
3 (18.8)
Serious TEAE 1 (14.3) 2 (12.5)
Infectious TEAE 6 (85.7) 9 (56.3)
Grade ≥3 Infectious TEAE 0 (0) 1 (6.3)
Opportunistic Infections 0 (0) 0 (0)
Death 0 (0) 0 (0)

Financial Results
•Cash, cash equivalents and marketable securities totaled $132.2 million as of December 31, 2024, compared to $201.4 million as of December 31, 2023. The decrease was primarily attributable to cash used in operations to advance clinical-stage programs.
•Revenue decreased by $7.0 million in 2024 compared to 2023 due to the October 2023 upfront payment received under the collaboration and license agreement with Everest Medicines.

•Research and development (R&D) expenses for the fourth quarter of 2024 decreased by $6.6 million to $16.0 million, compared to $22.6 million in the fourth quarter of 2023. Full year R&D expenses decreased by $20.0 million to $65.7 million in 2024, compared to $85.7 million in 2023. This decrease was primarily due to the Company’s strategic restructuring in October 2023 to prioritize its clinical-stage programs, reducing personnel-related costs and spending in its early-stage research activities, and a $5.0 million milestone payment made in 2023 under Kezar’s exclusive license agreement with Onyx Therapeutics. The decrease was partially offset by the increased clinical trial costs related to the PALIZADE and PORTOLA trials.
•General and administrative (G&A) expenses for the fourth quarter of 2024 decreased by $0.3 million to $5.5 million compared to $5.8 million in the fourth quarter of 2023. Full year G&A expenses decreased by $3.1 million to $23.4 million in 2024, compared to $26.5 million in 2023. The decrease was primarily due to a decrease in legal and professional service expenses and non-cash stock-based compensation.
•Restructuring and impairment charges decreased by $4.7 million to $1.5 million in 2024, compared to $6.2 million in 2023. The decrease was primarily related to one-time severance-related costs made in 2023 and higher impairment costs recognized in 2023 than 2024 on the right-of-use asset for the vacated floor in the leased office facility and certain equipment no longer utilized.
•Net loss for the fourth quarter of 2024 was $20.2 million, or $2.77 per basic and diluted common share, compared to a net loss of $32.3 million, or $4.44 per basic and diluted common share, for the fourth quarter of 2023. Net loss for 2024 was $83.7 million, or $11.49 per basic and diluted common share, compared to a net loss of $101.9 million, or $14.04 per basic and diluted common share in 2023. The weighted-average shares used to compute net loss per basic and diluted common share have been retroactively adjusted to reflect the one-for-ten reverse stock split completed on October 29, 2024.
•Total shares of common stock outstanding was 7.3 million shares as of December 31, 2024, after taking into effect the one-for-ten reverse stock split completed on October 29, 2024.
Conference Call and Webcast
Kezar Life Sciences will host a webcast and conference call today, March 25, 2025, at 8:00 a.m. ET to discuss topline results from the PORTOLA Phase 2a clinical trial. To access the live audio webcast with slides and dial-in information as needed, please register here: View Source
A live webcast of the event can also be found on the Kezar website at View Source A replay of the event will be available for 90 days following the presentation.

On March 25, 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 a radionuclide-drug conjugate (RDC) drug SKB107 (formerly TBM-001) from the Center for Drug Evaluation of the National Medical Products Administration (Press release, Kelun, MAR 25, 2025, View Source [SID1234651429]). SKB107 is the first company RDC drug clinical project.

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SKB107 is jointly developed by the Company and Professor Chen Yue’s team of the Affiliated Hospital of Southwest Medical University (the "Affiliated Hospital of SMU"). It utilizes a small molecule as the targeting ligand, combined with a suitable conjugation technology, chelator, and therapeutic radionuclide, and is intended to be used for treatment of bone metastases in solid tumors. Compared with traditional external radiation therapy, the RDC drug SKB107 can benefit patients with systemic multiple bone metastases and is highly targeted, which can reduce the damage to normal tissues, and is expected to show good safety; and compared with traditional bone-modifying drugs, it can effectively kill tumor cells with bone metastases, and is expected to have potential for the treatment of bone metastases efficacy. The Company entered into an exclusive license agreement with the Affiliated Hospital of SMU for a RDC drug SKB107 on Sept. 14, 2023.

About Bone Metastasis of Malignant Tumors

Bone is the most common site of metastasis in advanced malignant tumors, and about 70%~80% of patients with advanced malignant tumors will eventually develop bone metastasis. Among common tumors, prostate cancer, breast cancer, thyroid cancer, lung cancer and kidney cancer have a higher incidence of bone metastasis, accounting for more than 80% of all bone metastatic tumors[1]. Bone metastasis can lead to serious complications such as severe bone pain and bone-related events, such as pathologic fracture and spinal cord compression, which can seriously reduce patients’ quality of life and increase the risk of death. Currently, treatments for bone metastases of malignant tumors mainly include comprehensive treatments such as analgesic therapy, radiation therapy, bone-modifying drug therapy, and surgery. However, these treatments are still limited in improving patients’ quality of life, delaying or avoiding the occurrence of SREs, and prolonging patients’ survival, and the development of new mechanisms of action and safer and more effective drugs is imminent.