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Last update 21 Feb 2025

Bortezomib

Last update 21 Feb 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

[(1R)-3-methyl-1-({(2S)-3-phenyl-2-[(pyrazin-2-ylcarbonyl)amino]propanoyl}amino)butyl]boronic acid, Bortezomib (JAN/USAN/INN), N-[(1R)-1-(DIHYDROXYBORYL)-3-methylbutyl]-N-(pyrazin-2-ylcarbonyl)-L-phenylalaninamide

+ [16]

Target

Proteasome

Mechanism

Proteasome inhibitors

Therapeutic Areas

NeoplasmsImmune System DiseasesCardiovascular Diseases+ [4]

Active Indication

Immunoglobulin Light-Chain AmyloidosisWaldenstrom MacroglobulinemiaMantle-Cell Lymphoma+ [15]

Inactive Indication

Acute Graft Versus Host DiseaseAcute Lymphoblastic LeukemiaAcute myeloid leukaemia with 11q23 abnormality+ [103]

Originator Organization

Millennium Pharmaceuticals, Inc.

Active Organization

Sun Pharmaceutical Industries (Europe) BV

Celgene Corp.

Janssen Pharmaceutica NV

+ [21]

Inactive Organization

Johnson & Johnson Pharmaceutical Research & Development LLC

Spectrum Pharmaceuticals, Inc.

Genentech, Inc.

+ [33]

Drug Highest PhaseApproved

First Approval Date

US (13 May 2003),

Multiple Myeloma

RegulationAccelerated Approval (US), Orphan Drug (US), Orphan Drug (EU), Orphan Drug (JP)

Structure/Sequence

Molecular FormulaC19H25BN4O4

InChIKeyGXJABQQUPOEUTA-RDJZCZTQSA-N

CAS Registry179324-69-7

1,082

Clinical Trials associated with Bortezomib

NCT06015750

/ Not yet recruitingPhase 4

An Interventional, Prospective Open-Label Study of Immunosuppressive Therapies to Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia (RESTORE)

The primary objective of this study is to evaluate the effect of immunosuppressive therapy (IST) in participants treated with asfotase alfa who demonstrate immune-mediated loss of effectiveness (LoE).

Start Date02 Jul 2025

Sponsor / Collaborator

Alexion Pharmaceuticals, Inc.

NCT06348147

/ Not yet recruitingPhase 2IIT

An Attenuated Schedule Dara-RVd Induction for Patients With Newly Diagnosed Multiple Myeloma Who Are Eligible for Autologous Stem Cell Transplantation

This Phase II hybrid decentralized trial will examine the effect of daratumumab-based quadruplet induction therapy administered at an attenuated schedule in subjects with newly diagnosed multiple myeloma (NDMM) who are eligible for standard-of-care autologous stem cell transplantation (ASCT). Daratumumab, lenalidomide, bortezomib, and dexamethasone (Dara-RVd) have recently become a standard induction regimen for patients with NDMM who are eligible for ASCT in the United States. As implemented in clinical trials, Dara-RVd involves twice weekly bortezomib administration, which is inconvenient for patients and may result in increased rates of limiting toxicity, such as peripheral neuropathy. Adoption of alternate schedules involving once-weekly bortezomib is common in real-world practice, however a paucity of prospective data supporting this practice exists.
This study examines the efficacy of an attenuated Dara-RVd schedule involving once-weekly bortezomib dosing.

Start Date01 Mar 2025

Sponsor / Collaborator

UNC Lineberger Comprehensive Cancer Center

NCT06390319

/ RecruitingPhase 2IIT

SJALL23T: Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)

This is a clinical trial testing whether the addition of one of two chemotherapy agents, dasatinib or venetoclax, can improve outcomes for children and young adults with newly diagnosed T-cell acute lymphoblastic leukemia and lymphoma or mixed phenotype acute leukemia.
Primary Objective
* To evaluate if the end of induction MRD-negative rate is higher in patients with T-ALL treated with dasatinib compared to similar patients treated with 4-drug induction on AALL1231.
* To evaluate if the end of induction MRD-negative rate is higher in patients with ETP or near-ETP ALL treated with venetoclax compared to similar patients treated with 4-drug induction on AALL1231.
Secondary Objectives
* To assess the event free and overall survival of patients treated with this therapy.
* To compare grade 4 toxicities, event-free survival (EFS) and overall survival (OS) of patients treated with this therapy in induction and reinduction to toxicities of similar patients treated on TOT17.

Start Date27 Dec 2024

Sponsor / Collaborator

St. Jude Children's Research Hospital, Inc.

[+1]

100 Clinical Results associated with Bortezomib

100 Translational Medicine associated with Bortezomib

100 Patents (Medical) associated with Bortezomib

15,174

Literatures (Medical) associated with Bortezomib

01 Dec 2025·Blood Research

Efficacy of bortezomib combined with Hyper-CVAD in adults with relapsed acute lymphoblastic leukemia or positive measurable residual disease; effect of bortezomib in leukemia

Article

Author: Pérez Sámano, Daniela ; Gallardo Rodríguez, Adán Germán ; Martínez Tovar, Adolfo ; Cabrera García, Álvaro ; Olarte Carrillo, Irma ; Barranco Lampón, Gilberto ; Ramos Peñafiel, Christian Omar ; Terreros Palacio, Camila ; Martínez Murillo, Carlos

Abstract:

Purpose:

Despite advances in the treatment of adult acute lymphoblastic leukemia (ALL), relapse remains the most significant challenge in improving prognosis. Measurable residual disease (MRD) assessment can predict bone marrow relapse based on MRD positivity. As access to innovative therapies remains limited because of the high cost, chemotherapy is the widely utilized treatment option. The efficacy of a combination of bortezomib and Hyper-CVAD has been reported in patients with multiple myeloma; however, its efficacy has not yet been confirmed in patients with ALL.

Methods:

This prospective cohort study involved patients with ALL who presented with MRD-positive results or relapse and received treatment with a combination of bortezomib and Hyper-CVAD at two reference centers in Mexico City.

Results:

Of the 20 patients with positive MRD included in this study, 60% (n = 12) exhibited MRD negative results after combination treatment, 30% (n = 6) persisted positive MRD results, and 10% (n = 2) passed away. Of the 23 patients with bone marrow relapse, 43.5% (n = 10) achieved a second complete remission (2CR), 34.8% (n = 6) exhibited refractory status, and 21.7% (n = 5) passed away. To achieve a 2CR, 20% (n = 2) patients required less than four cycles of treatment, 50% (n = 5) required four cycles (two A and B cycles each), and 30% (n = 3) required six cycles.

Conclusion:

The combination of bortezomib and Hyper-CVAD treatment exhibited better results in achieving MRD negative results, indicating its potential as a promising first-line treatment strategy for ALL.

01 Dec 2025·CANCER CHEMOTHERAPY AND PHARMACOLOGY

Lipidomic profiling of plasma from patients with multiple myeloma receiving bortezomib: an exploratory biomarker study of JCOG1105 (JCOG1105A1)

Article

Author: Suzuki, Yasuhiro ; Tabayashi, Takayuki ; Maruta, Masaki ; Kuroda, Junya ; Saito, Yoshiro ; Harada, Yasuhiko ; Katsuya, Hiroo ; Yoshida, Shinichiro ; Takayama, Nobuyuki ; Iida, Shinsuke ; Mizuno, Ishikazu ; Norifumi, Tsukamoto ; Negoro, Eiju ; Yoshida, Isao ; Minami, Yosuke ; Miyazaki, Kana ; Maruyama, Dai ; Asano, Arisa ; Takamatsu, Yasushi ; Ohtsuka, Eiichi ; Munakata, Wataru ; Yoshimitsu, Makoto ; Tsujimura, Hideki ; Nagai, Hirokazu ; Fukuhara, Suguru ; Kanato, Keisuke ; Fukuhara, Noriko ; Ri, Masaki ; Ota, Shuichi ; Saito, Kosuke ; Saito, Toko

PURPOSE:

A comprehensive analysis of metabolites (metabolomics) has been proposed as a new strategy for analyzing liquid biopsies and has been applied to identify biomarkers predicting clinical responses or adverse events associated with specific treatments. Here, we aimed to identify metabolites associated with bortezomib (Btz)-related toxicities and response to treatment in newly diagnosed multiple myeloma (MM).

METHODS:

Fifty-four plasma samples from transplant-ineligible MM patients enrolled in a randomized phase II study comparing two less-intensive regimens of melphalan, prednisolone and Btz (MPB) were subjected to the lipidomic profiling analysis. The amount of each lipid metabolite in plasma obtained prior to MPB therapy was compared to toxicity grades and responses to MPB therapy.

RESULTS:

High levels of 7 phospholipids (4 lysophosphatidylcholines and 3 phosphatidylcholines) were observed in cases with Btz-induced ≥ grade 2 peripheral neuropathy (BiPN) (n = 11). In addition, low levels of 3 fatty acids (FAs)-FA (18:2), FA (18:1), and FA (22:6)-were observed in patients who developed severe skin disorders ≥ grade 2 (n = 10). No metabolite significantly associated with treatment response was identified.

CONCLUSION:

We conclude that levels of specific plasma lipid metabolites are associated with the severity of BiPN and skin disorders in patients with MM. These metabolites may serve as candidate biomarkers to predict Btz-induced toxicity in patients with MM before initiating Btz-containing therapy.

01 Apr 2025·COMPUTATIONAL BIOLOGY AND CHEMISTRY

Machine learning and molecular subtyping reveal the impact of diverse patterns of cell death on the prognosis and treatment of hepatocellular carcinoma

Article

Author: Yan, Xinyue ; Ji, Lurao ; Li, Xiaoqin ; Wang, Meng ; Gao, Bin

Programmed cell death (PCD) is a significant factor in the progression of hepatocellular carcinoma (HCC) and might serve as a crucial marker for predicting HCC prognosis and therapy response. However, the classification of HCC based on diverse PCD patterns requires further investigation. This study identified a novel molecular classification named PCD subtype (C1, C2, and C3) based on the genes associated with 19 PCD patterns, distinguished by clinical, biological functional pathways, mutations, immune characteristics, and drug sensitivity. Validated in 4 independent datasets, diverse cell death pathways were enriched in the C3 subtype, including apoptosis, pyroptosis, and autophagy, it also exhibited a highly infiltrative immunosuppressive microenvironment and demonstrated higher sensitivity to compounds such as Paclitaxel, Bortezomib, and YK-4-279, while C1 subtype was significantly enriched in cuproptosis and metabolism-related pathways, suggesting that it may be more suitable for cuproptosis-inducing agent therapy. Subsequently, utilizing the machine learning algorithms, we constructed a cell death-related index (CDRI) with 22 gene features and constructed prognostic nomograms with high predictive performance by combining CDRI with clinical features. Notably, we found that CDRI effectively predicted the response of HCC patients to therapeutic strategies, where patients with high CDRI were more suitable for sorafenib drug therapy and patients with low CDRI were more ideal for transarterial chemoembolization (TACE). In conclusion, the PCD subtype and CDRI demonstrate significant efficacy in predicting the prognosis and therapeutic outcomes for patients with HCC. These findings offer valuable insights for the development of precise, individualized treatment strategies.

376

News (Medical) associated with Bortezomib

19 Feb 2025

·www.prnewswire.com

Karyopharm Reports Fourth Quarter and Full Year 2024 Financial Results, Announces Update to Phase 3 XPORT-EC-042 Trial and Highlights Recent Company Progress

– Total Revenue of $145 Million and U.S. XPOVIO® (selinexor) Net Product Revenue of $113 Million for Full Year 2024 – – Top-Line Data Readout from Phase 3 SENTRY Trial Evaluating Selinexor in Combination with Ruxolitinib in Patients with JAKi-Naïve Myelofibrosis Anticipated in 2H 2025; Company on Track to Complete Enrollment in 1H 2025 – – Company Announces Update to Phase 3 XPORT-EC-042 Trial of Selinexor as Maintenance Therapy in Advanced or Recurrent TP53 Wild-Type Endometrial Cancer. Following Dialogue with the FDA Regarding the Evolving Treatment Landscape, Trial to Focus Enrollment on Patients with Either pMMR Tumors or Patients with dMMR Tumors that are Medically Ineligible for Checkpoint Inhibitors. Increasing Sample Size to 276; Top-Line Data Now Expected in Mid-2026 – – Company Provides Full-Year 2025 Total Revenue Guidance of $140 Million to $155 Million, Including U.S. XPOVIO Net Product Revenue Guidance of $115 Million to $130 Million – – Conference Call Scheduled for Today at 8:00 a.m. ET – NEWTON, Mass., Feb. 19, 2025 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today reported financial results for the fourth quarter and full year ended December 31, 2024. In addition, Karyopharm highlighted select corporate milestones and provided an overview of its key clinical development programs. "In 2025, our teams remain focused on the transformative opportunity to redefine the standard of care in myelofibrosis, with top-line data from our Phase 3 SENTRY trial evaluating selinexor in combination with ruxolitinib on-track for the second half of 2025. We look forward to completing enrollment of our SENTRY trial in the first half of this year and leveraging our demonstrated commercialization capabilities in multiple myeloma to support a rapid launch, subject to approval," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "In endometrial cancer, today we announced our updated plans for our Phase 3 trial and intend to primarily focus on the TP53 wild-type pMMR population, which represents approximately 50% of all women with endometrial cancer, and expect to share top-line data in mid-2026." Fourth Quarter 2024 and Recent Highlights XPOVIO Commercial Performance Achieved U.S. net product revenue for the year ended December 31, 2024 of $113 million, compared to $112 million for the year ended December 31, 2023. U.S. net product revenue for the fourth quarter of 2024 was $29 million, compared to $25 million for the fourth quarter of 2023. Demand for XPOVIO was consistent in 2024 versus 2023, with demand growth in the second half of 2024 in both the community setting, which represents approximately 60% of XPOVIO net product revenue, and the academic setting, offsetting a decline in demand in the first half of the year due to an intensified competitive landscape. XPOVIO net product revenue was impacted year-over-year by higher gross-to-net adjustments in 2024, driven primarily by increased 340B discounts and Medicare rebates. Expanded global patient access for selinexor in 2024 with favorable reimbursement decisions in the United Kingdom, France, Italy, China and South Korea and additional regulatory approvals in UAE, Kuwait, China, Malaysia, Turkey, Thailand, and South Korea in various indications, increasing the number of countries where selinexor is now approved in more than 45 countries. Research and Development (R&D) Highlights Myelofibrosis Updated the co-primary endpoint in the Phase 3 SENTRY trial (XPORT-MF-034; NCT04562389) to absolute mean change in total symptom score (Abs-TSS) following alignment with the U.S. Food and Drug Administration (FDA) and proactively increased the total sample size to approximately 350 patients to further increase the statistical powering. Abs-TSS measures the average improvement in symptom scores over 24 weeks relative to the baseline symptom score. Abs-TSS is viewed by many key opinion leaders (KOLs) and patient advocacy organizations as a more accurate assessment of symptom improvement in head-to-head clinical trials, such as SENTRY which is evaluating selinexor in combination with ruxolitinib in patients with JAK inhibitor (JAKi) naïve myelofibrosis versus ruxolitinib alone. Spleen volume reduction ≥35% (SVR35) at week 24 remains the other co-primary endpoint. These two co-primary endpoints will be tested sequentially starting with SVR35 followed by Abs-TSS. Hosted an investor event with leading KOLs in October 2024 to discuss the change in the co-primary endpoint in the Phase 3 SENTRY trial to Abs-TSS and highlight the strength of the data from the Company's Phase 1 trial in myelofibrosis. Data from the Company's Phase 1 trial, evaluating the combination of selinexor 60 mg plus ruxolitinib in JAKi naïve myelofibrosis patients, demonstrated that 79% of patients in the intent to treat population (n=14) achieved SVR35 and an average Abs-TSS improvement of 18.5 points in the efficacy evaluable population (n=9), at week 24 relative to baseline. Acknowledging the small sample size, these data suggest that the combination is favorable compared to historical ruxolitinib monotherapy data which indicates that less than half of patients achieve SVR35 and an Abs-TSS improvement of 11 to 14 points1. As of the most recent data cut off, the safety profile remained consistent and no new safety signals were identified. The Company continues to enroll patients into the 60 mg cohort of the Phase 2 SENTRY-2 trial of selinexor monotherapy in JAKi naïve patients with moderate thrombocytopenia (XPORT-MF-044; NCT05980806). 1Phase 3 MANIFEST trial. Rampal R, et al. ASH 2023. Oral 628; Phase 3 TRANSFORM-1 trial Pemmaraju N, et al. ASH 2023 abstract 620. Endometrial Cancer The Company is modifying the design of its Phase 3 XPORT-EC-042 trial evaluating selinexor as a maintenance-only therapy following systemic therapy versus placebo in patients with TP53 wild-type advanced or recurrent endometrial cancer. In late 2024 and early 2025, the Company engaged in communications with the FDA regarding the design adequacy of XPORT-EC-042 given the changing standard of care in endometrial cancer, particularly the approval of checkpoint inhibitors for patients with advanced or recurrent endometrial cancer regardless of mismatch repair status. In light of the FDA's acknowledgement that the magnitude of benefit achieved from checkpoint inhibitors is less for patients with pMMR tumors than patients with dMMR tumors, consistent with the biology and mechanism of action of checkpoint inhibitors, the Company's modifications include defining two patient populations for which the primary endpoint of progression free survival (PFS), tested sequentially, and key secondary endpoint of overall survival (OS) will be evaluated: a modified intent to treat population (mITT) that will include patients with: TP53 wild-type tumors with proficient mismatch repair status (pMMR); or, TP53 wild-type tumors with deficient mismatch repair status (dMMR), who are medically ineligible to receive checkpoint inhibitors. the trial's original intent to treat (ITT) population, which will include all patients enrolled in the trial whose tumors are TP53 wild-type, regardless of MMR status. The Company is increasing the trial sample size from 220 patients to approximately 276 patients, to ensure that the mITT population includes approximately 220 patients who are either: a) TP53 wild-type pMMR or b) TP53 wild-type dMMR and medically ineligible to receive a checkpoint inhibitor. The increase in sample size maintains sufficient power for the primary endpoint of PFS in the mITT population. To date, approximately 80% of patients enrolled meet the new eligibility definition for the mITT population. The proposed modifications are intended to address certain of the FDA's feedback regarding the evolving treatment landscape, including the approval of multiple checkpoint inhibitors for advanced/recurrent endometrial cancer patients with pMMR and/or dMMR tumors in 2023 and 2024. Enrollment continues in the XPORT-EC-042 trial and, depending on the strength of the data, the Company intends to pursue regulatory approval. As a result of the proposed modifications, the Company now expects topline data in mid-2026. Multiple Myeloma Completed enrollment of the Phase 3 XPORT-MM-031 trial (EMN29; NCT05028348) of approximately 120 patients, leveraging the data published on selinexor 40 mg, pomalidomide and dexamethasone (SPd40) in 2024. The Phase 3 XPORT-MM-031 trial is being conducted in collaboration with the European Myeloma Network and is evaluating the all-oral combination SPd40 in patients with previously treated multiple myeloma who received an anti-CD38 in their immediate prior line of therapy. Pending ongoing engagement with regulatory agencies on the updated protocol and statistical plan, the Company intends to provide an update on this trial. Anticipated Catalysts and Operational Objectives in 2025 Myelofibrosis Announce completion of enrollment of the Phase 3 SENTRY trial evaluating selinexor in combination with ruxolitinib in JAKi naive myelofibrosis patients in 1H 2025. Report preliminary data on a subset of participants in the 60 mg cohort from the Phase 2 SENTRY-2 trial evaluating selinexor as a monotherapy in patients with JAKi naïve myelofibrosis with moderate thrombocytopenia in 1H 2025. Report top-line results from the Phase 3 SENTRY trial in 2H 2025. Multiple Myeloma Maintain the Company's commercial foundation in the competitive multiple myeloma marketplace and drive increased XPOVIO revenues. Continue global launches and reimbursement approvals for selinexor by partners in ex-U.S. territories. Continue to follow patients that are enrolled in the Phase 3 XPORT-MM-031 (EMN29) trial. Pending ongoing engagement with regulatory agencies on the updated protocol and statistical plan, the Company intends to provide an update on this trial. Endometrial Cancer Continue to enroll patients into the Phase 3 XPORT-EC-042 trial of selinexor as a maintenance monotherapy for patients with TP53 wild-type advanced or recurrent endometrial cancer. 2025 Financial Outlook Based on its current operating plans, Karyopharm expects the following for full year 2025: Total revenue to be in the range of $140 million to $155 million. Total revenue consists of U.S. XPOVIO net product revenue and license, royalty and milestone revenue earned from partners. U.S. XPOVIO net product revenue to be in the range of $115 million to $130 million. R&D and selling, general and administrative (SG&A) expenses to be in the range of $240 million to $255 million, which includes approximately $20 million of estimated non-cash stock-based compensation expense. The Company expects that its existing cash, cash equivalents and investments, and the revenue it expects to generate from XPOVIO net product sales, as well as revenue generated from its license agreements, will be sufficient to fund its planned operations into the first quarter of 2026.2 2Excluding re-payment of $24.5 million aggregate principal amount of the Company's remaining senior convertible notes due October 2025 (the 2025 Notes) and $25.0 million minimum liquidity covenant under the Company's senior secured term loan due 2028. Taking into account the repayment of the 2025 Notes and the minimum liquidity covenant, Karyopharm expects its cash, cash equivalents and investments will be sufficient to fund its operations into the fourth quarter of 2025. Full Year and Fourth Quarter 2024 Financial Results Total revenue: Total revenue for the fourth quarter of 2024 was $30.5 million, compared to $33.7 million for the fourth quarter of 2023. Total revenue for the year ended December 31, 2024 was $145.2 million, compared to $146.0 million for the year ended December 31, 2023. Net product revenue: Net product revenue for the fourth quarter of 2024 was $29.3 million, compared to $25.1 million for the fourth quarter of 2023. Net product revenue for the year ended December 31, 2024 was $112.8 million, compared to $112.0 million for the year ended December 31, 2023. License and other revenue: License and other revenue for the fourth quarter of 2024 was $1.3 million, compared to $8.7 million for the fourth quarter of 2023. License and other revenue for the year ended December 31, 2024 was $32.4 million, compared to $34.0 million for the year ended December 31, 2023. Cost of sales: Cost of sales for the fourth quarter of 2024 was $1.3 million, compared to $1.5 million for the fourth quarter of 2023. Cost of sales for the year ended December 31, 2024 was $6.0 million, compared to $4.9 million for the year ended December 31, 2023. Cost of sales reflects the costs of XPOVIO units sold and third-party royalties on net product revenue. R&D expenses: R&D expenses for the fourth quarter of 2024 were $33.3 million, compared to $39.4 million for the fourth quarter of 2023. R&D expenses for the year ended December 31, 2024 were $143.2 million, compared to $138.8 million for the year ended December 31, 2023. The increase in both periods was primarily due to increased clinical trial activity, partially offset by a reduction in headcount and contractors. SG&A expenses: SG&A expenses for the fourth quarter of 2024 were $27.2 million, compared to $30.7 million for the fourth quarter of 2023. SG&A expenses for the year ended December 31, 2024 were $115.4 million, compared to $131.9 million for the year ended December 31, 2023. The decrease in both periods was primarily due to a reduction in headcount and contractors as well as lower commercial-related activities in connection with cost optimization efforts. Interest income: Interest income for the fourth quarter of 2024 was $1.5 million, compared to $2.5 million for the fourth quarter of 2023. Interest income for the year ended December 31, 2024 was $7.4 million compared to $10.9 million for the year ended December 31, 2023, due to lower investment balances in 2024 as compared to 2023. Interest expense: Interest expense for the fourth quarter of 2024 was $11.2 million, compared to $6.2 million for the fourth quarter of 2023. Interest expense for the year ended December 31, 2024 was $37.4 million, compared to $23.8 million for the year ended December 31, 2023. The increase in both periods was primarily due to the term loan and convertible debt that were issued in 2024. Gain on extinguishment of debt and other income: Other income for the fourth quarter of 2024 was $10.1 million due to non-cash fair value remeasurements. The Company had immaterial other expense in the fourth quarter of 2023. Gain on extinguishment of debt and other income for the year ended December 31, 2024 was $73.1 million primarily due to the recognition of a $44.7 million non-cash gain on extinguishment of debt and $28.7 million non-cash fair value remeasurements, both of which related to the refinancing transactions that were completed in mid-2024. The Company had immaterial other expense for the year ended December 31, 2023. Net loss: Karyopharm reported a net loss of $30.8 million, or $0.24 per basic and diluted share, for the fourth quarter of 2024, compared to a net loss of $41.8 million, or $0.36 per basic and diluted share, for the fourth quarter of 2023. Net loss included non-cash stock-based compensation expense of $3.9 million and $5.2 million for the fourth quarters of 2024 and 2023, respectively. Karyopharm reported a net loss of $76.4 million, or $0.63 per basic share and $0.93 per diluted share, for the year ended December 31, 2024, compared to a net loss of $143.1 million, or $1.25 per basic and diluted share, for the year ended December 31, 2023. Net loss included non-cash stock-based compensation expense of $18.4 million and $21.7 million for the years ended December 31, 2024 and 2023, respectively. Cash position: Cash, cash equivalents, restricted cash and investments as of December 31, 2024 totaled $109.1 million, compared to $192.4 million as of December 31, 2023. Conference Call Information Karyopharm will host a conference call today, February 19, 2025, at 8:00 a.m. Eastern Time, to discuss the fourth quarter and full year 2024 financial results, the financial outlook for 2025 and to provide other business updates. To access the conference call, please dial (800) 836-8184 (local) or (646) 357-8785 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. A live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company's website. An archived webcast will be available on the Company's website approximately two hours after the event. About XPOVIO® (selinexor) XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm's Selective Inhibitor of Nuclear Export (SINE) compounds for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with VELCADE® (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) under accelerated approval in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO® (also known as NEXPOVIO® in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, Mainland China, Taiwan, Hong Kong, Australia, South Korea, Singapore, Israel, and Canada. XPOVIO®/NEXPOVIO® is marketed in these respective ex-U.S. territories by Karyopharm's partners: Antengene, Menarini, Neopharm, and FORUS. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in endometrial cancer and myelofibrosis. For more information about Karyopharm's products or clinical trials, please contact the Medical Information department at: Tel: +1 (888) 209-9326; Email: [email protected] XPOVIO® (selinexor) is a prescription medicine approved: In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd). In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti‐CD38 monoclonal antibody (Xd). For the treatment of adult patients with relapsed or refractory diffuse large B‐cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). SELECT IMPORTANT SAFETY INFORMATION Warnings and Precautions Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care. Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony‐stimulating factors. Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care. Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care. Serious Infection: Monitor for infection and treat promptly. Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes. Embryo‐Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception. Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract. Adverse Reactions The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3‐4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%. The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%. The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3‐4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients. Use In Specific Populations Lactation: Advise not to breastfeed. For additional product information, including full prescribing information, please visit . To report SUSPECTED ADVERSE REACTIONS, contact Karyopharm Therapeutics Inc. at 1‐888‐209‐9326 or FDA at 1‐800‐FDA‐1088 or . About Karyopharm Therapeutics Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company whose dedication to pioneering novel cancer therapies is fueled by a belief in the extraordinary strength and courage of patients with cancer. Since its founding, Karyopharm has been an industry leader in oral compounds that address nuclear export dysregulation, a fundamental mechanism of oncogenesis. Karyopharm's lead compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is approved in the U.S. and marketed by the Company in three oncology indications. It has also received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including Europe and the United Kingdom (as NEXPOVIO®) and China. Karyopharm has a focused pipeline targeting indications in multiple high unmet need cancers, including in multiple myeloma, endometrial cancer, myelofibrosis, and diffuse large B-cell lymphoma (DLBCL). For more information about our people, science and pipeline, please visit , and follow us on LinkedIn and on X at @Karyopharm. Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding Karyopharm's guidance on its 2025 total revenue, 2025 U.S. net product revenue and 2025 R&D and SG&A expenses; expected cash runway; expectations with respect to commercialization efforts; the ability of selinexor to treat patients with multiple myeloma, endometrial cancer, myelofibrosis, diffuse large B-cell lymphoma and other diseases; and expectations with respect to the clinical development plans and potential regulatory submissions of selinexor. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond Karyopharm's control, that may cause actual events or results to differ materially from Karyopharm's current expectations. For example, there can be no guarantee that Karyopharm will successfully commercialize XPOVIO or that any of Karyopharm's drug candidates, including selinexor, will successfully complete necessary clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in the development or commercialization of Karyopharm's drug candidate portfolio will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: the adoption of XPOVIO in the commercial marketplace, the timing and costs involved in commercializing XPOVIO or any of Karyopharm's drug candidates that receive regulatory approval; the ability to obtain and retain regulatory approval of XPOVIO or any of Karyopharm's drug candidates that receive regulatory approval; Karyopharm's results of clinical trials and preclinical trials, including subsequent analysis of existing data and new data received from ongoing and future trials; the content and timing of decisions made by the U.S. Food and Drug Administration and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, including with respect to the need for additional clinical trials; the ability of Karyopharm or its third party collaborators or successors in interest to fully perform their respective obligations under the applicable agreement and the potential future financial implications of such agreement; Karyopharm's ability to enroll patients in its clinical trials; unplanned cash requirements and expenditures; substantial doubt exists regarding Karyopharm's ability to continue as a going concern; development or regulatory approval of drug candidates by Karyopharm's competitors for products or product candidates in which Karyopharm is currently commercializing or developing; the direct or indirect impact of the COVID-19 pandemic or any future pandemic on Karyopharm's business, results of operations and financial condition; and Karyopharm's ability to obtain, maintain and enforce patent and other intellectual property protection for any of its products or product candidates. These and other risks are described under the caption "Risk Factors" in Karyopharm's Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, which was filed with the Securities and Exchange Commission (SEC) on November 5, 2024, and in other filings that Karyopharm may make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and, except as required by law, Karyopharm expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. XPOVIO® and NEXPOVIO® are registered trademarks of Karyopharm Therapeutics Inc. SOURCE Karyopharm Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Drug ApprovalClinical ResultPhase 1Financial Statement

18 Feb 2025

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HALOZYME REPORTS FULL YEAR 2024 RECORD REVENUE OF $1.015 BILLION AND EXCEEDS ITS FINANCIAL GUIDANCE FOR ROYALTY REVENUE, ADJUSTED EBITDA AND NON-GAAP DILUTED EPS

Fourth Quarter Total Revenue Increased 30% YOY to $298 million and Royalty Revenue Increased 40% YOY to $170 million Fourth Quarter Net Income Increased 60% YOY to $137 million; Adjusted EBITDA Increased 61% YOY to $196 million; GAAP EPS Increased 63% YOY to $1.06; non-GAAP EPS Increased 54% YOY to $1.261 Record Full Year 2024 Total Revenue Increased 22% YOY to $1,015 million and Record Royalty Revenue Exceeded Guidance Increasing 27% YOY to $571 million Full Year 2024 Net Income Increased 58% YOY to $444 million; GAAP EPS Increased 63% YOY to $3.43; Adjusted EBITDA Increased 48% YOY to $632 million and non-GAAP EPS Increased 53% YOY to $4.231, Both Exceeding Guidance Reiterating 2025 Financial Guidance Ranges for Total Revenue of $1,150 - 1,225 million, Representing YOY Growth of 13% - 21%, Adjusted EBITDA of $755 - $805 million, Representing YOY Growth of 19% - 27% and non-GAAP Diluted EPS of $4.95 - $5.35, Representing YOY Growth of 17% - 26%1 SAN DIEGO, Feb. 18, 2025 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme" or the "Company") today reported its financial and operating results for the fourth quarter and full year ended December 31, 2024, provided an update on its recent corporate activities and reiterated its 2025 financial guidance. "I am excited to announce that the significant growth we achieved throughout the year culminated in two important milestones for the Company: achievement of more than $1 billion in total revenue and reaching a cumulative one million patients with our ENHANZE drug delivery technology. Our 2024 royalty revenue exceeded guidance driven by continued strong growth of DARZALEX SC and Phesgo, with modest initial contribution from VYVGART Hytrulo resulting from growing adoption and use primarily from its first indication for generalized myasthenia gravis. These three products will continue to drive our 2025 royalty revenues, with VYVGART Hytrulo becoming the largest dollar growth contributor in 2025," said Dr. Helen Torley, president and chief executive officer of Halozyme. "I am also pleased that we achieved four additional, significant ENHANZE regulatory product and indication approvals in the U.S. and EU in 2024 for VYVGART Hytrulo for CIDP, Tecentriq Hybreza, Ocrevus Zunovo and Opdivo Qvantiq, positioning Halozyme for strong continued growth, post 2025, once coverage reimbursement and access are fully established," said Dr. Torley. "Our leadership position in rapid subcutaneous drug delivery and long-term growth was further fortified with five new ENHANZE nominations from argenx and ViiV and an extension of our ENHANZE patent in Europe out to 2029, with a similar patent submitted in the U.S. Our 2025 guidance reflects our confidence in continuing robust total revenue, royalty revenue, adjusted EBITDA and non-GAAP EPS growth," Dr. Torley concluded. Fourth Quarter and Recent Corporate Highlights: Reiterating 2025 financial guidance previously announced on January 8, 2025 including total revenue of $1,150 million to $1,225 million, representing year-over-year growth of 13% to 21%, adjusted EBITDA of $755 million to $805 million, representing year-over-year growth of 19% to 27% and non-GAAP diluted earnings per share of $4.95 to $5.35, representing year-over-year growth of 17% to 26%. In December 2024, Halozyme entered into an Accelerated Share Repurchase agreement to repurchase $250.0 million of its common stock under the $750 million approved program from February 2024. Fourth Quarter and Recent Partner Highlights: In February 2025, Janssen-Cilag International NV, a Johnson & Johnson company, received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency recommending an extension of marketing authorization for a subcutaneous ("SC") formulation of RYBREVANT® (amivantamab) with ENHANZE® in combination with LAZCLUZE® (lazertinib) for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations, and as a monotherapy for the treatment of adult patients with advanced NSCLC with activating EGFR exon 20 insertion mutations after failure of platinum-based therapy. In December 2024, Bristol Myers Squibb announced the U.S Food and Drug Administration ("FDA") approved Opdivo® Qvantig (nivolumab and hyaluronidase-nvhy) with ENHANZE® for SC use in most previously approved adult, solid tumor IV Opdivo® (nivolumab) indications resulting in the recognition of a $20.0 million milestone payment, and in January 2025, Opdivo® Qvantig was made available to patients. In December 2024, argenx announced the Ministry of Health, Labour and Welfare ("MHLW") in Japan approved VYVDURA® for the treatment of patients with chronic inflammatory demyelinating polyneuropathy ("CIDP"). In December 2024, Takeda announced the MHLW in Japan approved HYQVIA® with ENHANZE for patients with agammaglobulinemia or hypogammaglobulinemia disorders characterized by very low or absent levels of antibodies and an increased risk of serious recurring infection caused by primary immunodeficiency or secondary immunodeficiency. In November 2024, Zai Lab Limited (argenx commercial partner for China) announced the National Medical Products Administration approved VYVGART® Hytrulo for the treatment of patients with CIDP. In November 2024, Janssen announced the submission of regulatory applications to the FDA and European Medicines Agency ("EMA") seeking approval of a new indication for DARZALEX FASPRO® in the U.S. and DARZALEX® SC in the EU as a monotherapy for the treatment of adult patients with high-risk smoldering multiple myeloma. In October 2024, argenx initiated two studies evaluating VYVGART® Hytrulo with ENHANZE®, a Phase 3 study for adult patients with ocular myasthenia gravis and a Phase 2 study for kidney transplant recipients with antibody mediated rejection. In October 2024, Janssen announced the European Commission approved DARZALEX® SC for the treatment of patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant in combination with bortezomib, lenalidomide and dexamethasone. Fourth Quarter and Full Year 2024 Financial Highlights: Revenue was $298.0 million, compared to $230.0 million in the fourth quarter of 2023. The 30% year-over-year increase was primarily driven by royalty revenue growth and higher revenues under collaborative agreements mainly due to the timing of milestones achieved. Revenue for the quarter included $170.4 million in royalties, an increase of 40% compared to $122.1 million in the fourth quarter of 2023, primarily attributable to increases in revenue of DARZALEX® SC, VYVGART® Hytrulo and Phesgo®. Total revenue for the full year was $1,015.3 million, compared to $829.3 million in 2023, representing 22% year-over-year growth. The increase was primarily driven by royalty revenue growth, higher revenues under collaborative agreements mainly due to the timing of milestones achieved and higher sales of our proprietary products. Cost of sales was $42.1 million, compared to $52.3 million in the fourth quarter of 2023. The decrease was primarily due to lower bulk rHuPH20 sales. Cost of sales for the full year was $159.4 million, compared to $192.4 million in 2023. The decrease was primarily due to lower bulk rHuPH20 and device sales, partially offset by higher proprietary product sales. Amortization of intangibles expense was $17.8 million, compared to $17.8 million in the fourth quarter of 2023. Amortization of intangibles expense for the full year was $71.0 million, compared to $73.8 million in 2023. The decrease was primarily due to an impairment charge of $2.5 million recognized in the prior year to fully impair the TLANDO® product rights intangible asset. Research and development expense was $20.4 million, compared to $21.3 million in the fourth quarter of 2023. Research and development expense for the full year was $79.0 million, compared to $76.4 million in 2023. The increase was primarily due to planned investments in ENHANZE® related to the development of our new high-yield rHuPH20 manufacturing processes. Selling, general and administrative expense was $42.2 million, compared to $37.6 million in the fourth quarter of 2023. The increase was primarily due to increased compensation expense and consulting and professional service fees. Selling, general and administrative expense for the full year was $154.3 million, compared to $149.2 million in 2023. The increase was primarily due to increased compensation expense and consulting and professional service fees, partially offset by planned reductions in commercial marketing expense. Operating income was $175.5 million, compared to $101.0 million in the fourth quarter of 2023. Operating income for the full year was $551.5 million, compared to $337.6 million in 2023. Net income was $137.0 million, compared to $85.4 million in the fourth quarter of 2023. Net income for the full year was $444.1 million, compared to $281.6 million in 2023. EBITDA was $195.8 million, compared to $121.7 million in the fourth quarter of 2023. Adjusted EBITDA was $195.8 million, compared to $121.7 million in the fourth quarter of 2023.1 EBITDA for the full year was $632.2 million, compared to EBITDA of $435.6 million in 2023. Adjusted EBITDA for the full year was $632.2 million, compared to $426.2 million in 2023.1 GAAP diluted earnings per share was $1.06, compared to $0.65 in the fourth quarter of 2023. Non-GAAP diluted earnings per share was $1.26, compared to $0.82 in the fourth quarter of 2023.1 GAAP diluted earnings per share for the full year was $3.43, compared to $2.10 in 2023. Non-GAAP diluted earnings per share for the full year was $4.23, compared to $2.77 in 2023.1 Cash, cash equivalents and marketable securities were $596.1 million on December 31, 2024, compared to $336.0 million on December 31, 2023. The increase was primarily a result of cash generated from operations. Financial Outlook for 2025 The Company is reiterating its financial guidance for 2025, which was initially provided on January 8, 2025. For the full year 2025, the Company expects: Total revenue of $1,150 million to $1,225 million, representing growth of 13% to 21% over 2024 total revenue, primarily driven by increases in royalty revenue and product sales from XYOSTED®. Revenue from royalties of $725 million to $750 million, representing growth of 27% to 31% over 2024. Adjusted EBITDA of $755 million to $805 million, representing growth of 19% to 27% over 2024. Non-GAAP diluted earnings per share of $4.95 to $5.35, representing growth of 17% to 26% over 2024. The Company's earnings per share guidance does not consider the impact of potential future share repurchases. Table 1. 2025 Financial Guidance Webcast and Conference Call Halozyme will host its Quarterly Update Conference Call for the fourth quarter and full year ended December 31, 2024 today, Tuesday, February 18, 2025 at 1:30 p.m. PT/4:30 p.m. ET. The conference call may be accessed live with pre-registration via link: . The call will also be webcast live through the "Investors" section of Halozyme's corporate website and a recording will be made available following the close of the call. To access the webcast and additional documents related to the call, please visit Halozyme.com. About Halozyme Halozyme is a biopharmaceutical company advancing disruptive solutions to improve patient experiences and outcomes for emerging and established therapies. As the innovators of ENHANZE® drug delivery technology with the proprietary enzyme rHuPH20, Halozyme's commercially-validated solution is used to facilitate the subcutaneous delivery of injected drugs and fluids, with the goal of improving the patient experience with rapid subcutaneous delivery and reduced treatment burden. Having touched one million patient lives in post-marketing use in nine commercialized products across more than 100 global markets, Halozyme has licensed its ENHANZE® technology to leading pharmaceutical and biotechnology companies including Roche, Takeda, Pfizer, Janssen, AbbVie, Eli Lilly, Bristol-Myers Squibb, argenx, ViiV Healthcare, Chugai Pharmaceutical and Acumen Pharmaceuticals. Halozyme also develops, manufactures and commercializes, for itself or with partners, drug-device combination products using its advanced auto-injector technologies that are designed to provide commercial or functional advantages such as improved convenience, reliability and tolerability, and enhanced patient comfort and adherence. The Company has two commercial proprietary products, Hylenex® and XYOSTED®, partnered commercial products and ongoing product development programs with Teva Pharmaceuticals and Idorsia Pharmaceuticals. Halozyme is headquartered in San Diego, CA and has offices in Ewing, NJ and Minnetonka, MN. Minnetonka is also the site of its operations facility. For more information visit and connect with us on LinkedIn and Twitter. Note Regarding Use of Non-GAAP Financial Measures In addition to disclosing financial measures prepared in accordance with U.S. generally accepted accounting principles ("GAAP"), this press release and the accompanying tables contain certain non-GAAP financial measures. The Company reports earnings before interest, taxes, depreciation, and amortization ("EBITDA"), adjusted EBITDA, Non-GAAP diluted earnings per share, Non-GAAP diluted shares, and guidance with respect to those measures, in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company calculates non-GAAP diluted earnings per share excluding share-based compensation expense, amortization of debt discounts, intangible asset amortization, one-time changes, if any, such as changes in contingent liabilities, inventory adjustments, impairment charges, and certain adjustments to income tax expense. The Company calculates non-GAAP diluted shares excluding the dilutive impact of convertible notes which is used in calculating non-GAAP diluted earnings. The Company calculates EBITDA excluding interest, taxes, depreciation and amortization. The Company calculates adjusted EBITDA excluding one-time items, if any, such as changes in contingent liabilities, inventory adjustments, impairment charges and transaction costs for business combinations. Reconciliations between GAAP and Non-GAAP financial measures are included at the end of this press release. The Company does not provide reconciliations of forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in share-based compensation expense and the effects of any discrete income tax items. For the same reasons, the Company is unable to address the probable significance of the unavailable information. The Company provides non-GAAP financial measures that it believes will be achieved, however it cannot accurately predict all of the components of the adjusted calculations and the U.S. GAAP measures may be materially different than the non-GAAP measures. The Company evaluates other items of income and expense on an individual basis for potential inclusion in the calculation of Non-GAAP financial measures and considers both the quantitative and qualitative aspects of the item, including (i) its size and nature, (ii) whether or not it relates to the Company's ongoing business operations and (iii) whether or not the Company expects it to occur as part of the Company's normal business on a regular basis. Non-GAAP financial measures do not have any standardized meaning and are therefore unlikely to be comparable to similarly titled measures presented by other companies. These non-GAAP financial measures are not meant to be considered in isolation and should be read in conjunction with the Company's consolidated financial statements prepared in accordance with GAAP, and are not prepared under any comprehensive set of accounting rules or principles. In addition, from time to time in the future there may be other items that the Company may exclude for purposes of its non-GAAP financial measures, and the Company may in the future cease to exclude items that it has historically excluded for purposes of its non-GAAP financial measures. The Company considers these non-GAAP financial measures to be important because they provide useful measures of the operating performance of the Company, exclusive of factors that do not directly affect what the Company considers to be its core operating performance, as well as unusual events. The non-GAAP measures also allow investors and analysts to make additional comparisons of the operating activities of the Company's core business over time and with respect to other companies, as well as assessing trends and future expectations. The Company uses non-GAAP financial information in assessing what it believes is a meaningful and comparable set of financial performance measures to evaluate operating trends, as well as in establishing portions of our performance-based incentive compensation programs. Safe Harbor Statement In addition to historical information, the statements set forth in this press release include forward-looking statements including, without limitation, statements concerning the Company's financial performance (including the Company's expected financial outlook for 2025) and expectations for future growth, profitability, total revenue, royalty revenue, EBITDA, Adjusted EBITDA, and non-GAAP diluted earnings-per-share and potential share repurchases under its share repurchase program. Forward-looking statements regarding the Company's ENHANZE® drug delivery technology may include the possible benefits and attributes of ENHANZE®, its potential application to aid in the dispersion and absorption of other injected therapeutic drugs and facilitating more rapid delivery and administration of higher volumes of injectable medications through subcutaneous delivery and the expected expiration date of our ENHANZE® patent in Europe. Forward-looking statements regarding the Company's business may include potential growth and receipt of royalty and milestone payments driven by our partners' development and commercialization efforts, potential new clinical trial study starts and clinical data, regulatory submissions and product launches, the size and growth prospects of our partners' drug franchises, potential new or expanded collaborations and collaborative targets and regulatory review, and potential approvals of new partnered or proprietary products, and the potential timing of these events. These forward-looking statements are typically, but not always, identified through use of the words "expect," "believe," "enable," "may," "will," "could," "intends," "estimate," "anticipate," "plan," "predict," "probable," "potential," "possible," "should," "continue," and other words of similar meaning and involve risk and uncertainties that could cause actual results to differ materially from those in the forward-looking statements. Actual results could differ materially from the expectations contained in these forward-looking statements as a result of several factors, including unexpected levels of revenues, expenditures and costs, unexpected delays in the execution of the Company's share repurchase program, unexpected results or delays in the growth of the Company's business, or in the development, regulatory review or commercialization of the Company's partnered or proprietary products, regulatory approval requirements, unexpected termination of the European ENHANZE® patent prior to the date of expiration, unexpected adverse events or patient outcomes and competitive conditions. These and other factors that may result in differences are discussed in greater detail in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. Except as required by law, the Company undertakes no duty to update forward-looking statements to reflect events after the date of this release. Contacts: Tram Bui VP, Investor Relations and Corporate Communications 609-333-7668 [email protected] Samantha Gaspar Teneo 212-886-9356 [email protected] Footnotes: 1. Reconciliations between GAAP reported and non-GAAP financial information for actual results are provided at the end. SOURCE Halozyme Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug ApprovalFinancial StatementImmunotherapyPhase 3Phase 2

13 Feb 2025

·www.prnewswire.com

Antengene Announces XPOVIO® Approved for Public Health Insurance Coverage in Taiwan Market, Benefiting More Patients with R/R MM in the Region

XPOVIO® is the first XPO1 inhibitor approved in Taiwan for the treatment of adult patients with relapsed/refractory multiple myeloma (R/R MM). After the mainland of China, South Korea, Australia and Singapore, Taiwan market is the fifth APAC market in which XPOVIO® has been approved for public health insurance coverage. XPOVIO® is expected to extend public health insurance coverage across APAC markets. SHANGHAI and HONG KONG, Feb. 13, 2025 /PRNewswire/ -- Antengene Corporation Limited (" Antengene", SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class medicines for cancer, today announced that XPOVIO® (selinexor) in combination with bortezomib and dexamethasone (XVd) for the treatment of adult patients with relapsed/refractory multiple myeloma (R/R MM) who have received at least two prior therapies, has been approved for reimbursement in Taiwan. Starting from March 1, 2025, XPOVIO® will be officially included in the NHI drug reimbursement scheme. With a novel mechanism of action, XPOVIO® is the world's first approved orally-available, selective XPO1 inhibitor, which has already been approved in nine countries and regions in APAC, and included in the public insurance schemes in five of those markets (the mainland of China, Taiwan market, Australia, Singapore and South Korea). Moving forward, XPOVIO® is expected to extend public health insurance coverage across APAC markets. Multiple myeloma (MM) is a malignancy caused by the dysregulated proliferation of plasma cells. According to epidemiology data, MM is the second most prevalent hematologic malignancy in Taiwan market, accounting for approximately 700 to 800 newly diagnosed cases and around 400 relevant deaths each year.[1] Most patients with MM have to face a range of challenges in treatment, including high propensity to relapse, short period of survival, and limited treatment options. The inclusion of XPOVIO® for reimbursement coverage in Taiwan market, will further reduce the financial burden on many patients, benefiting more patients and their families. While bringing XPOVIO® to more APAC markets, Antengene is also striving to expand the indications of XPOVIO®. Leveraging the drug's novel mechanism of action, XPOVIO® is currently being developed with multiple combination regimens for the treatment of various additional indications including myelofibrosis (MF) and endometrial cancer. About XPOVIO® (selinexor) XPOVIO® is the world's first approved orally-available, selective inhibitor of the nuclear export protein XPO1. It offers a novel mechanism of action, synergistic effects in combination regimens, fast onset of action, and durable responses. By blocking the nuclear export protein XPO1, XPOVIO® can promote the intranuclear accumulation and activation of tumor suppressor proteins and growth regulating proteins, and down-regulate the levels of multiple oncogenic proteins. XPOVIO® delivers its antitumor effects through three mechanistic pathways: 1) exerting antitumor effects by inducing the intranuclear accumulation of tumor suppressor proteins; 2) reducing the level of oncogenic proteins in the cytoplasm by inducing the intranuclear accumulation of oncogenic mRNAs; 3) restoring hormone sensitivity by activating the glucocorticoid receptors (GR) pathway. To utilize its unique mechanism of actions, XPOVIO® is being evaluated for use in multiple combination regimens in a range of indications. At present, Antengene is conducting multiple clinical studies of XPOVIO® in the mainland of China for the treatment of relapsed/refractory hematologic malignancies and solid tumors (3 of these studies are being jointly conducted by Antengene and Karyopharm Therapeutics Inc. [Nasdaq:KPTI]). About Antengene Antengene Corporation Limited ( "Antengene", SEHK: 6996.HK) is a leading commercial-stage R&D-driven global biopharmaceutical company focused on the discovery, development, manufacturing and commercialization of innovative first-in-class/best-in-class therapeutics for the treatment of hematologic malignancies and solid tumors, in realizing its vision of "Treating Patients Beyond Borders". Since 2017, Antengene has built a pipeline of 9 oncology assets at various stages going from clinical to commercial, including 6 with global rights, and 3 with rights for the APAC region. To date, Antengene has obtained 31 investigational new drug (IND) approvals in the U.S. and Asia, and submitted 10 new drug applications (NDAs) in multiple Asia Pacific markets, with the NDA for XPOVIO® (selinexor) already approved in Mainland of China, Taiwan China, Hong Kong China, Macau China, South Korea, Singapore, Malaysia, Thailand and Australia. Forward-looking statements The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development. For a further discussion of these and other factors that could cause future results to differ materially from any forward-looking statement, please see the other risks and uncertainties described in the Company's Annual Report for the year ended December 31, 2023, and the documents subsequently submitted to the Hong Kong Stock Exchange. Reference 2019 Cancer Registry Annual Report, Health Promotion Administration of Taiwan Ministry of Health and Welfare For more information, please contact: Investor Contacts: Donald Lung E-mail: [email protected] Mobile: +86 18420672158 PR Contacts: Peter Qian E-mail: [email protected] Mobile: +86 13062747000 SOURCE Antengene Corporation Limited WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Drug Approval

100 Deals associated with Bortezomib

External Link

KEGG	Wiki	ATC	Drug Bank
D03150	Bortezomib	L01XX32	DB00188

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Immunoglobulin Light-Chain Amyloidosis	JP	Janssen Pharmaceutical KK	20 Oct 2006
Waldenstrom Macroglobulinemia	JP	Janssen Pharmaceutical KK	20 Oct 2006
Mantle-Cell Lymphoma	EU	Janssen-Cilag International NV	26 Apr 2004
Mantle-Cell Lymphoma	IS	Janssen-Cilag International NV	26 Apr 2004
Mantle-Cell Lymphoma	LI	Janssen-Cilag International NV	26 Apr 2004
Mantle-Cell Lymphoma	NO	Janssen-Cilag International NV	26 Apr 2004
Multiple Myeloma	US	Takeda Pharmaceuticals U.S.A., Inc.	13 May 2003

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Diffuse large B-cell lymphoma recurrent	Phase 3	IT	Janssen-Cilag Ltd.	04 Feb 2013
Diffuse large B-cell lymphoma recurrent	Phase 3	IT	Janssen Pharmaceutica NV	04 Feb 2013
Diffuse large B-cell lymphoma refractory	Phase 3	IT	Janssen Pharmaceutica NV	04 Feb 2013
Diffuse large B-cell lymphoma refractory	Phase 3	IT	Janssen-Cilag Ltd.	04 Feb 2013
Plasma Cell Leukemia	Phase 3	CN	Janssen Scientific Affairs LLC	13 Jan 2012
Plasma Cell Leukemia	Phase 3	AU	Janssen Scientific Affairs LLC	13 Jan 2012
Plasma Cell Leukemia	Phase 3	KR	Janssen Scientific Affairs LLC	13 Jan 2012
Diffuse Large B-Cell Lymphoma	Phase 3	CH	Janssen-Cilag Ltd.	01 Apr 2011
Diffuse Large B-Cell Lymphoma	Phase 3	GB	Janssen-Cilag Ltd.	01 Apr 2011
B-Cell Lymphoma	Phase 3	US	Millennium Pharmaceuticals, Inc.	01 Mar 2006

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05564052 (VEGA, CTgov)	Phase 2	Mantle-Cell Lymphoma	36	Ibrutinib+Rituximab (Arm A1: Ibrutinib 560 mg + Rituximab 375 mg/m^2)	stvbivuizs(jhohllysqj) = jugglauvrh ioxlltxenm (ljrrldjhct, rpqsxjnffy - kudeqkbssi) View more	-	13 Jan 2025
				Ibrutinib+Rituximab (Arm A2: Ibrutinib 420 mg + Rituximab 375 mg/m^2)	stvbivuizs(jhohllysqj) = ciwnzuvzfr ioxlltxenm (ljrrldjhct, fillsntupx - dkbosqmflh) View more
NCT04246047 (DREAMM-7, ASH2024) Manual	Phase 3	Multiple Myeloma	494	Belantamab mafodotin, bortezomib, and dexamethasone (BVd)	zlmnguhwoe(xtaxlqxhxx) = szksyuvbne kqjrxmgndt (wznsngwzid, 28.4 - NR) Met View more	Positive	09 Dec 2024
				Daratumumab, bortezomib, and dexamethasone (DVd)	zlmnguhwoe(xtaxlqxhxx) = aczlhedknd kqjrxmgndt (wznsngwzid, 11.1 - 17.5) Met View more
NCT03620903 (ECWM-2, ASH2024) Manual	Phase 2	Waldenstrom Macroglobulinemia First line	53	Bortezomib+Ibrutinib+Rituximab	jqzyyasijg(jrqnwkihzj) = tytycbnzmy ljdxtebliw (cweftjlkas ) View more	Positive	09 Dec 2024
NCT03319667 (IMROZ, ASH2024) Manual	Phase 3	Multiple Myeloma First line	446	Isatuximab, Bortezomib, Lenalidomide, and Dexamethasone (Isa-VRd)	ulnxwljlpt(hkjonrbiix) = ihaqfzplwn ejhvyimabb (gdrjdsappa ) View more	Positive	09 Dec 2024
				Bortezomib, Lenalidomide, and Dexamethasone (VRd)	ulnxwljlpt(hkjonrbiix) = pamrvgdcct ejhvyimabb (gdrjdsappa ) View more
ASH2024	Not Applicable	Multiple Myeloma	-	Pomalidomide, Bortezomib, Dexamethasone	zxeiemuyot(bgjgqqfzsx) = giqorwsjhc uzfdguovke (unpxrmavvt ) View more	-	09 Dec 2024
ASH2024	Not Applicable	Multiple Myeloma	-	Ixazomib-lenalidomide-dexamethasone (IRd)	yakufprnip(fkeowxknjd) = Any grade TEAEs reported in ≥25% of pts were diarrhea (26%) in the 1−3 cycles group; PN not elsewhere classified (NEC; 34%), fatigue (34%), diarrhea (34%), and edema NEC (26%) in the 4−9 cycles group; and diarrhea (68%), PN NEC (53%), fatigue (42%), edema NEC (38%), arthralgia (35%), nausea (33%), and back pain (29%) in the >9 cycles group. duaspavsfs (lqrjaehwhq ) View more	-	09 Dec 2024
NCT03617731 (GMMG-HD7, ASH2024)	Phase 3	Multiple Myeloma CD38	662	Isatuximab, Lenalidomide, Bortezomib, Dexamethasone	ujouhuqfpj(eogrtzkbgh) = itbuhkqcif rbtjbdrcbx (ccstdprzyj, 95% CI 0.52 - 0.94) View more	Positive	09 Dec 2024
				Lenalidomide, Bortezomib, Dexamethasone	ujouhuqfpj(eogrtzkbgh) = opjdhxrupj rbtjbdrcbx (ccstdprzyj, 95% CI 46 - 48) View more
CASTOR (ASH2024)	Phase 3	Multiple Myeloma	-	Daratumumab/bortezomib/dexamethasone (DVd)	ziwizwsgyd(onzskdpsdj) = epeuztsnts nixzazqcsd (txquyvfqyk, 48.5 - 56.2) View more	Positive	08 Dec 2024
ASH2024	Not Applicable	Multiple Myeloma	-	Daratumumab, Bortezomib, Lenalidomide, and Dexamethasone (DVRd)	npjqudjplv(ewafgunarw) = awrxtbween komndqabxw (zuaxfmtusw ) View more	-	08 Dec 2024
				DVRd-DR	npjqudjplv(ewafgunarw) = gtaeyjmncm komndqabxw (zuaxfmtusw ) View more
ASH2024	Not Applicable	Multiple Myeloma	-	Daratumumab, Lenalidomide, Bortezomib, Dexamethasone (D-RVd)	keksqgkrbw(qjjsyqhvyo) = 82% iduucnrece (szzhhyzkvq ) View more	-	08 Dec 2024

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