On May 7, 2025 2seventy bio, Inc. (Nasdaq: TSVT), reported financial results and recent highlights for the first quarter ended March 31, 2025 (Press release, Bristol-Myers Squibb, MAY 7, 2025, View Source [SID1234652636]).

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"We started 2seventy with a singular focus on harnessing the power of cell therapy to deliver more time for people living with cancer," said Chip Baird, chief executive officer, 2seventy bio. "Over the past four years we have delivered on that mission, treating thousands of multiple myeloma patients with Abecma and developing an R&D pipeline of innovative treatments for liquid and solid tumors that we transitioned to Regeneron and Novo Nordisk. With the anticipated closing of the acquisition of 2seventy by Bristol Myers Squibb (BMS) this quarter, we will continue to deliver Abecma (idecabtagene vicleucel; ide-cel) as part of BMS. I would like to express my deep gratitude to our current and former team members and more broadly the dedicated community of patients, scientists, providers, partners, and investors who worked tirelessly to deliver more time for patients."

On March 10, 2025, 2seventy bio announced that it had entered into a definitive merger agreement to be acquired by BMS. Under the terms of the agreement, BMS commenced a tender offer on April 14, 2025 to acquire all outstanding shares of common stock of 2seventy bio at a price of $5.00 per share in an all-cash transaction. 2seventy bio’s Board of Directors unanimously recommended that 2seventy bio stockholders tender their shares in the tender offer. The waiting period applicable to the tender offer under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act) expired at 11:59 p.m. Eastern Time on May 2, 2025. The transaction remains subject to the tender of a majority of the outstanding shares of 2seventy bio’s common stock, as well as other customary closing conditions. Unless the tender offer is extended, the offer and withdrawal rights will expire at midnight (New York City time), one minute after 11:59 p.m. New York City time, on May 12, 2025.

ABECMA COMMERCIAL HIGHLIGHTS

First quarter Abecma U.S. sales, as reported by BMS, were $58.6 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.
2seventy bio and BMS share equally in all profits and losses related to development, manufacturing, and commercialization of Abecma in the United States. 2seventy bio reported collaboration revenue of approximately $19.1 million related to the collaboration with BMS for the three months ended March 31, 2025.
SELECT FIRST QUARTER FINANCIAL RESULTS

Total revenues were $22.9 million for the three months ended March 31, 2025, compared to $12.4 million for the three months ended March 31, 2024.
Research and development expenses were $5.4 million for the three months ended March 31, 2025, compared to $43.9 million for the three months ended March 31, 2024.
Selling, general and administrative expenses were $14.9 million for the three months ended March 31, 2025, compared to $12.7 million for the three months ended March 31, 2024.
There was no loss on assets held for sale for the three months ended March 31, 2025, compared to a $5.0 million loss on assets held for sale for the three months ended March 31, 2024.
Net income was $0.5 million for the three months ended March 31, 2025, compared to net loss of $52.7 million for the three months ended March 31, 2024.
Cash, cash equivalents, and marketable securities totaled $173.4 million as of March 31, 2025.
Merger Agreement Details and Path to Completion

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 6.0% of the outstanding shares of 2seventy bio’s common stock entered into tender and support agreements, pursuant to which they agreed to tender all of their shares in the offer.

In light of the announced transaction, 2seventy bio 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). 134 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.

About Abecma

Abecma is being jointly developed and commercialized in the U.S. as part of a Co-Development, Co-Promotion, and Profit Share Agreement between BMS and 2seventy bio. BMS assumes sole responsibility for Abecma drug product manufacturing and commercialization outside of the U.S. The companies’ clinical development program for Abecma includes ongoing clinical studies (KarMMa-2, KarMMa-3) in earlier lines of treatment for patients with multiple myeloma. For more information visit clinicaltrials.gov.

On May 7, 2025 BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), a new type of biopharmaceutical company focused on genetic diseases, reported that members of its management team will participate in a fireside chat at the Bank of America Merrill Lynch Global Healthcare Conference 2025 in Las Vegas, NV on Wednesday, May 14 at 2:20 pm PT (Press release, BridgeBio, MAY 7, 2025, View Source [SID1234652635]).

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To access the live webcast of BridgeBio’s presentation, please visit the "Events" page within the Investors section of the BridgeBio website at View Source A replay of the webcast will be available on the BridgeBio website for 30 days following the event.

On May 7, 2025 BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that will change its name to BeOne Medicines, Ltd., reported financial results and corporate updates from the first quarter 2025 (Press release, BeiGene, MAY 7, 2025, View Source [SID1234652634]).

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"We delivered another exceptional quarter, achieving our first quarter of GAAP profitability with continued global revenue growth. In the U.S., BRUKINSA remains the leader in new chronic lymphocytic leukemia (CLL) patient starts across all lines of therapy, and for the first time has become the overall BTKi market share leader," said John V. Oyler, Co-Founder, Chairman, and CEO of BeiGene. "We’ve made significant strides across our late-stage hematology and solid tumor pipelines, with multiple proof-of-concept readouts expected this year across our broad portfolio of antibody-drug conjugates, multispecific antibodies and targeted protein degraders. With accelerating financial momentum and a diversified global footprint spanning six continents, we are well positioned — as we transition to BeOne Medicines and redomicile to Switzerland — to become one of the world’s most impactful oncology innovators."

First Quarter 2025 Financial Snapshot
(Amounts in thousands of U.S. dollars and unaudited)
Three Months Ended March 31,
2025 2024 % Change
Net product revenues $ 1,108,530 $ 746,918 48 %
Net revenue from collaborations $ 8,749 $ 4,734 85 %
Total revenue $ 1,117,279 $ 751,652 49 %
GAAP income (loss) from operations $ 11,102 $ (261,348) 104 %
Adjusted income (loss) from operations* $ 139,357 $ (147,341) 195 %

GAAP net income (loss) $ 1,270 $ (251,150) 101 %
Adjusted net income (loss)* $ 136,137 $ (145,896) 193 %
GAAP basic EPS per ADS $ 0.01 $ (2.41) 100 %
Adjusted basic EPS per ADS* $ 1.27 $ (1.40) 191 %
GAAP diluted EPS per ADS $ 0.01 $ (2.41) 100 %
Adjusted diluted EPS per ADS* $ 1.22 $ (1.40) 187 %

* For an explanation of our use of non-GAAP financial measures refer to the "Note Regarding Use of Non-GAAP Financial Measures" section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measures, see the table at the end of this press release.

First Quarter 2025 Financial Results
Revenue for the first quarter of 2025 was $1.1 billion, compared to $752 million in the prior-year period driven primarily by growth in BRUKINSA product sales in the U.S. and Europe.
Product Revenue totaled $1.1 billion for the first quarter of 2025 compared to $747 million in the prior-year period. The increase in product revenue was primarily attributable to increased sales of BRUKINSA. The U.S. continued to be the Company’s largest market, with product revenue of $563 million compared to $351 million in the prior-year period. In-licensed products from Amgen and TEVIMBRA also contributed to product revenue growth.
•U.S. sales of BRUKINSA totaled $563 million in the first quarter of 2025, representing growth of 60% over the prior-year period driven primarily by demand, with more than 60% of the quarter-over-quarter growth coming from expanded use in CLL as BRUKINSA continued to gain share as the leader in new patient starts in the U.S. in CLL and all other approved indications; BRUKINSA sales in Europe totaled $116 million in the first quarter of 2025, representing growth of 73% compared to the prior-year period, driven by increased market share across all major European markets, including Germany, Italy, Spain, France and the UK.
•Sales of TEVIMBRA totaled $171 million in the first quarter of 2025, representing growth of 18% compared to the prior-year period.
Gross Margin as a percentage of global product sales for the first quarter of 2025 was 85.1% compared to 83.3% in the prior-year period on a GAAP basis. The gross margin percentage increased due to a proportionally higher sales mix of global BRUKINSA compared to other products in our portfolio. Gross margins also benefited from cost of sales productivity improvements for both BRUKINSA and TEVIMBRA. On an adjusted basis, which does not include depreciation and amortization, gross margin as a percentage of product sales increased to 85.5% for the first quarter of 2025, compared to 83.7% in the prior-year period.
Operating Expenses
The following table summarizes operating expenses for the first quarter of 2025:
GAAP Non-GAAP
(unaudited, in thousands, except percentages) Q1 2025 Q1 2024 % Change Q1 2025 Q1 2024 % Change
Research and development $ 481,887 $ 460,638 5 % $ 421,195 $ 405,440 4 %
Selling, general and administrative $ 459,288 $ 427,427 7 % $ 395,511 $ 372,146 6 %
Total operating expenses $ 941,175 $ 888,065 6 % $ 816,706 $ 777,586 5 %

Research and Development (R&D) Expenses increased for the first quarter of 2025 compared to the prior-year period on both a GAAP and adjusted basis primarily due to advancing preclinical programs into the clinic and early clinical programs into late stage. Upfront fees and milestone payments related to in-process R&D for in-licensed assets totaled nil and $35 million in the first quarter of 2025 and 2024, respectively.
Selling, General and Administrative (SG&A) Expenses increased for the first quarter of 2025 compared to the prior-year period on both a GAAP and adjusted basis due to continued investment in the global commercial expansion of BRUKINSA primarily in the U.S. and Europe. SG&A expenses as a percentage of product sales were 41% for the first quarter of 2025, compared to 57% in the prior-year period.
Net Income/(Loss) and Earnings Per Share
GAAP net income improved for the first quarter of 2025, as compared to the prior-year period loss, primarily attributable to revenue growth and improved operating leverage.
For the first quarter of 2025, both basic and diluted earnings per share was $0.00 per share and $0.01 per American Depositary Share (ADS), respectively, compared to basic loss of $0.19 per share and $2.41 per ADS in the prior-year period.
Cash Provided by Operations for the first quarter of 2025 was $44 million, an increase of $353 million over the prior-year period.
For further details on BeiGene’s First Quarter 2025 Financial Statements, please see BeiGene’s Quarterly Report on Form 10-Q for the first quarter of 2025 filed with the U.S. Securities and Exchange Commission.

Full Year 2025 Guidance
BeiGene is maintaining its full year 2025 revenue and expense guidance. Guidance is summarized below:
FY 20251
Total Revenue $4.9 billion to $5.3 billion
GAAP Operating Expenses (R&D and SG&A) $4.1 billion to $4.4 billion
Additional: GAAP Gross Margin Percentage in mid-80% range
Positive Full Year GAAP Operating Income
Generation of Positive Cash Flow from Operations

1 Does not assume any potential new, material business development activity or unusual/non-recurring items. Assumes January 31, 2025 foreign exchange rates.
BeiGene’s total revenue guidance for full year 2025 of $4.9 billion to $5.3 billion includes expectations for strong revenue growth driven by BRUKINSA’s U.S. leadership position and continued global expansion in both Europe and other important rest of world markets. Gross margin percentage is expected to be in the mid-80% range due to mix and production efficiencies as compared to 2024. BeiGene’s guidance for combined operating expenses on a GAAP basis includes expectations of investment to support growth in both commercial and research at a pace that continues to deliver meaningful operating leverage. Non-GAAP operating expenses, which exclude costs related to share-based compensation, depreciation and amortization expense, are expected to track with GAAP operating expenses, with reconciling items unchanged from existing practice. Operating expense guidance does not assume any potential new, material business development activity or unusual/non-recurring items.
First Quarter Business Highlights
Core Marketed Products
BRUKINSA
•BRUKINSA is now approved in 75 markets globally with 11 new or expanded reimbursements in the quarter, including in Japan, Europe and Brazil.
•Received approval for the addition of Siegfried in Switzerland as an alternate Drug Substance manufacturer by the European Medicines Agency.
TEVIMBRA
•TEVIMBRA is now approved in 46 markets globally with 11 new reimbursements in the quarter, including in the U.S., Europe and China.
•Received U.S. Food and Drug Administration (FDA) approval in combination with platinum-containing chemotherapy for the first-line treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1 (≥1).
•Received FDA approval for 150 mg Q2W and 300 mg Q4W alternate dosing regimens in addition to the already approved 200 mg Q3W dosing.
•Received Japan approval in combination with platinum-containing chemotherapy for the first- and second-line treatment of adult patients with unresectable or metastatic ESCC.
•Received European Commission approval in combination with etoposide and platinum chemotherapy as a first-line treatment for adult patients with extensive-stage small cell lung cancer.

Select Clinical-Stage Programs
Hematology
•Sonrotoclax (BCL2 inhibitor): Continued enrollment of global Phase 2 trial for the treatment of Waldenström’s macroglobulinemia.
•Sonrotoclax BGB-11417-202: Filed in China for the treatment of relapsed/refractory (R/R) CLL.
•Sonrotoclax CELESTIAL-RR MCL BGB-11417-302: Achieved first subject enrolled for Phase 3 trial for the treatment of R/R MCL.
•Sonrotoclax CELESTIAL-TN CLL BGB-11417-301: Achieved last subject enrolled for Phase 3 trial for the treatment of treatment-naïve (TN) CLL.
•BGB-16673 (BTK CDAC): Continued enrollment of potentially registration enabling Phase 2 trial for the treatment of R/R CLL with data readout expected in 2026.
•BGB-16673: Initiated Phase 3 trial compared to physician’s choice (IR/VR/BR) for treatment of R/R CLL.
Lung Cancer
•Tarlatamab (AMG757, DLL3xCD3 BiTE): Announced positive data readout from Phase 3 trial for the treatment of second-line small cell lung cancer in collaboration with Amgen.
•Anti-TIGIT antibody: Discontinued clinical development of ociperlimab as a potential treatment for lung cancer.

Anticipated R&D Milestones
•The Company will hold an Investor R&D Day on June 26 highlighting its emerging breast cancer franchise and broader solid tumor portfolio.
Programs
Milestones
Timing
BRUKINSA
•Tablet formulation: FDA and European Commission approvals.
2H 2025
•MANGROVE trial for TN MCL: Interim analysis of Phase 3 trial.
2H 2025
•MAHOGANY trial for the treatment of R/R follicular lymphoma: Complete enrollment of the FL portion of Phase 3 trial.
2H 2025
TEVIMBRA
•EU approvals for the treatment of:
◦Neoadjuvant/adjuvant non-small cell lung cancer.
1H 2025
◦First-line nasopharyngeal carcinoma.
2H 2025
•Subcutaneous formulation: Initiate Phase 3 trial.
2H 2025
Hematology
•Sonrotoclax in combination with anti-CD20 antibody for the treatment of R/R CLL: First subject enrolled in global Phase 3 trial.
1H 2025
•Sonrotoclax for the treatment of R/R MCL: Data readout of Phase 2 trial and potential global accelerated approval submissions.
2H 2025
•BGB-16673 compared to noncovalent BTK inhibitor pirtobrutinib for the treatment of R/R CLL: Initiate Phase 3 head-to-head trial.
2H 2025
Lung Cancer
•BGB-58067 (PRMT5 inhibitor) and BG-89894 (MAT2A inhibitor): First subject enrolled in combination trial.
2H 2025
Breast and Gynecologic Cancers
•BGB-43395 (CDK4 inhibitor): Proof-of-concept data.
1H 2025
GI Cancers
•Zanidatamab (HER2-bispecific antibody) for the treatment of first-line HER2-positive gastroesophageal adenocarcinoma: Readout of primary progression-free survival data from Phase 3 trial of in collaboration with Zymeworks/Jazz.
2H 2025
Inflammation and Immunology
•BGB-45035 (IRAK4 CDAC): First subject enrolled in Phase 2 trial.
2H 2025
•BGB-45035: Proof-of-concept data for tissue IRAK4 degradation.
2H 2025

Other Highlights
•Received shareholder approval on April 28, 2025, to rename the Company to BeOne Medicines Ltd. and redomicile to Switzerland with the transaction set to close later this year.
•As previously disclosed, announced a U.S. Patent Trademark Office Final Written Decision invalidating all claims of Pharmacyclics LLC’s U.S. Patent No. 11,672,803 that were challenged by BeiGene in a post-grant review (PGR) proceeding.
•Appointed Marcello Damiani as Chief Technology Officer.
Conference Call and Webcast
The Company’s earnings conference call for the first quarter 2025 will be broadcast via webcast at 8:00 a.m. ET on Wednesday, May 7, 2025, and will be accessible through the Investors section of BeiGene’s website, www.beigene.com. Supplemental information in the form of a slide presentation and a replay of the webcast will also be available.

On May 7, 2025 Barinthus Biotherapeutics plc (NASDAQ: BRNS) ("Barinthus Bio," or the "Company"), an immunology and inflammation ("I&I") company focused on developing therapies that promote immune tolerance with curative potential, reported its financial results for the quarter ended March 31, 2025 and provided an overview of the Company’s corporate developments (Press release, Barinthus Biotherapeutics, MAY 7, 2025, View Source [SID1234652633]).

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"2025 has started with a strategic focus on immunological and inflammatory diseases, which includes directing our resources to our highly differentiated lead asset,VTP-1000, using the SNAP-TI platform, being tested in patients with celiac disease. Looking ahead, we remain on track to announce Phase 1 single ascending dose data for the celiac program using VTP-1000 in the third quarter of 2025, and will be initiating the multiple ascending dose part of the Phase 1 clinical trial in the second half of 2025, where we are incorporating a gluten challenge as part of our initial clinical evaluation of efficacy" said Bill Enright, Chief Executive Officer of Barinthus Bio. "Today, we announced multiple data milestones from our viral vector pipeline, including encouraging primary endpoint analyses from the two Phase 2 trials in chronic hepatitis B ("CHB") which we believe strengthen VTP-300’s market positioning as a component of a potential functional cure for CHB."

First Quarter 2025 and Recent Corporate Developments

Clinical Developments
Data from two Phase 2 clinical trials of VTP-300 will be showcased in poster presentations at the European Association for the Study of the Liver ("EASL") Congress 2025, taking place May 7-10, 2025, in Amsterdam, the Netherlands. The presentations include the six-month primary analysis of the Phase 2b clinical trial (HBV003), as well as end-of-study data from the Phase 2a clinical trial (IM-PROVE II, AB-729-202) in partnership with Arbutus Biopharma, both in people with CHB receiving ongoing standard of care nucleos(t)ide analogue ("NUC") therapy.

HBV003 data: VTP-300 and Low-dose Nivolumab
The HBV003 study is evaluating the safety, immunogenicity and disease modifying activity of three different dosing regimens of VTP-300 in combination with low-dose nivolumab ("LDN"), an anti-PD-1 monoclonal antibody. The primary analysis showed;
•In CHB participants with hepatitis B surface antigen ("HBsAg") levels of <200 IU/mL, meaningful reductions in HBsAg (>1 log decline) occurred soon after dosing on Day 29 in all treatment groups and were maintained to Day 169.
•In the two best treatment arms HBsAg declines of ≥1 log at Day 169 were observed in 33% (15/45) of participants with HBsAg ≤200 IU/mL at baseline, and 22% (10/45) of participants achieved HBsAg loss at any timepoint.
•71% (48/68) of participants met the criteria for discontinuation of NUC therapy at day 169; and although NUC discontinuation was optional; two participants who did discontinue NUCs achieved functional cure and one seroconverted to HBsAb positivity.
•Treatment with VTP-300 in combination with LDN was generally well-tolerated, with no serious adverse events reported.

The primary analysis confirms observations from previous interim data, which indicated that stronger responses occurred in participants treated with the combination of VTP-300 and LDN (Groups 1 and 2).

IM-PROVE II data: imdusiran and VTP-300
The IM-PROVE II study is evaluating the combination of imdusiran ("IDR"), Arbutus’ RNAi therapeutic, followed by Barinthus Bio’s T-cell stimulating immunotherapeutic, VTP-300, with or without LDN. The end of study data showed:
•25% (2/8) of participants with starting baseline HBsAg levels less than 1000 IU/mL receiving the combination of IDR, VTP-300 and LDN achieved functional cure.
•3 of 13 participants (23%) receiving IDR+VTP-300+LDN had undetectable HBsAg levels at week 48; all (3/3) of participants with HBsAg loss seroconverted.
•Treatment with IDR and VTP-300 was generally well-tolerated, with no serious adverse events or treatment discontinuations reported.

Corporate Updates
•In January 2025, Barinthus Bio announced a strategic business refocus and restructuring to prioritize immunology and inflammation indications, including antigen-specific immune tolerance. Barinthus Bio will not invest in VTP-300 for chronic hepatitis B beyond the completion of the ongoing Phase 2b HBV003 clinical trial and will seek potential partners to be able to take advantage of its differentiated ability to achieve sustained HBsAg loss and functional cure in patients with low levels of HBsAg. Partners are also being sought for the other assets that are based upon the viral vector platforms.

Upcoming Milestones

Celiac Disease (VTP-1000):
•Single ascending dose data from the Phase 1 AVALON clinical trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of VTP-1000 in adults with celiac disease expected in the third quarter of 2025.
•Initiation of the multiple ascending dose portion of the Phase 1 AVALON clinical trial is expected in the second half of 2025.

Prostate Cancer (VTP-850):
•Topline results from the Phase 1 PCA001 clinical trial evaluating safety and efficacy of VTP-850 in men with rising prostate-specific antigen after definitive local therapy for prostate cancer were received and analysis is ongoing. Data will be used to support partnering efforts for VTP-850.

First Quarter 2025 Financial Highlights
•Cash: As of March 31, 2025, cash, cash equivalents and restricted cash was $100.6 million, compared to $112.4 million as of December 31, 2024. The $11.8 million decrease is a result of the net cash used in operating activities of $14.9 million for the development of our pipeline and ongoing clinical trials, offset by a $3.1 million gain on foreign exchange on cash, cash equivalents and restricted cash. Based on current research and development plans, the Company expects its available resources to fund its operating expenses and capital expenditure requirements into 2027.
•Research and Development Expenses: Research and development expenses were $8.3 million for the three months ended March 31, 2025 compared to $11.1 million for the three months ended March 31, 2024, with the decrease attributable to the stage of clinical development of the pipeline assets, a reduction in preclinical activity and a reduction in workforce when compared to the prior year. The year-on-year research and development expenses per program are outlined in the following table. It is anticipated that research and development expenses related to the legacy programs in infectious disease and oncology will reduce going forward, as the ongoing clinical trials complete, and that research and development expenses related to autoimmune programs will continue or increase, as the clinical development continues.

Three months ended March 31, 2025 Three months ended March 31, 2024 Change
$000
$000
$000
Direct research and development expenses by program:
VTP-1000 Celiac $ 982 $ 1,374 $ (392)
VTP-300 HBV 1,350 1,913 (563)
Other clinical programs1
741 1,767 (1,026)
Other pre-clinical programs 419 784 (365)
Total direct research and development expenses 3,492 5,838 (2,346)
Indirect research and development expenses:
Personnel-related (including share-based compensation)2
3,944 4,335 (391)
Facility related 335 390 (55)
Other indirect costs 519 562 (43)
Total indirect research and development expenses 4,798 5,287 (489)
Total research and development expense $ 8,290 $ 11,125 $ (2,835)

1 This includes expenses relating to the infectious disease and oncology programs; VTP-850 Prostate cancer, VTP-200 HPV, VTP-600 NSCLC (the Phase 1/2a trial is sponsored by Cancer Research UK) and VTP-500 MERS (funded pursuant to an agreement with the Coalition for Epidemic Preparedness Innovations ("CEPI"). Expenses relating to these programs were previously presented separately, but are now aggregated for the prior period comparative.
2 This includes $0.07 million and $0.14 million for the three months ended March 31, 2025 and 2024, respectively, of personnel-related indirect expenses relating to time spent progressing the VTP-500 MERS program, which is funded by CEPI.
•General and Administrative Expenses: General and administrative expenses were $12.6 million in the first quarter of 2025, compared to $6.0 million in 2024. The increase of $6.6 million relates primarily to a loss of $4.4 million on foreign exchange in 2025, compared to a gain of $1.2 million in 2024 due to fluctuations between the pound sterling and the US dollar during the year. The remaining increase is attributable to an increase in depreciation of U.K. assets as a result of the expected closure of the U.K. site, and an increase in personnel costs as a result of the workforce reduction.
•Net Loss: For the first quarter of 2025, the Company generated a net loss attributable to its shareholders of $19.6 million, or $(0.49) per share on both basic and fully diluted bases, compared to a net loss attributable to its shareholders of $15.5 million, or $(0.40) per share on both basic and fully diluted bases for the first quarter of 2024.

On May 6, 2025 Mission Bio, a pioneer in single-cell multi-omics solutions, reported it has partnered with Integrated DNA Technologies (IDT), a global leader in genomics solutions, to introduce an advanced workflow for precise on- and off-target confirmation in gene editing applications (Press release, Mission Bio, MAY 6, 2025, View Source [SID1234652616]). This collaboration integrates IDT’s award-winning rhAmpSeq technology with Mission Bio’s Tapestri Platform to provide a comprehensive, high-resolution approach for assessing genome editing outcomes at the single-cell level.

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Genome editing is crucial for the development of the next wave of cell and gene therapies, enabling precise and multiple genetic modifications. However, ensuring the specificity and efficiency of these edits remains a critical challenge, particularly in cell and gene therapy (CGT) applications. Traditional bulk sequencing methods provide valuable insights into genome editing efficiency, but lack the resolution to capture single-cell heterogeneity, zygosity, and co-occurrence of edits within individual cells.

To address these challenges, Mission Bio and IDT have developed an integrated workflow combining IDT’s rhAmpSeq amplicon-based targeted resequencing chemistry with Mission Bio’s droplet-based single-cell DNA sequencing technology. This novel solution builds on published approaches that researchers have previously used to leverage the platforms. The new, integrated approach enhances the efficiency for obtaining both on-target efficacy assessment and off-target detection in a single-cell context, offering researchers an unprecedented level of accuracy and confidence in genome editing analysis.

In a proof-of-concept study, to be presented at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, the integrated workflow was interrogated using well-characterized cell lines and selected for their extensive orthogonal bulk sequencing data. By targeting loci with known indel variations ranging from single-base pair changes to large insertions and deletions (>2,000 bp), the study demonstrated the robustness of rhAmpSeq chemistry in single-cell applications. The resulting targeted panel proved compatible with both bulk and single-cell assays, ensuring seamless adoption across various research needs.

"Our single-cell multi-omics has been used in combination with bulk sequencing to add specificity that otherwise would not be possible," said Brian Kim, CEO of Mission Bio. "By integrating our capabilities with IDT’s trusted approach to bulk sequencing, we’re adding together multiple layers of genetic insights in a single drop."

The Tapestri Platform’s automated single-cell genome editing analytics pipeline provided a quantitative assessment of editing efficiency, off-target editing levels and zygosity, offering a critical layer of data resolution beyond bulk sequencing. By integrating rhAmpSeq’s advanced primer design with Mission Bio’s single-cell DNA resequencing technology, the new workflow establishes a cost-effective, scalable, and highly customizable solution for genome editing validation in CGT applications.

"Safety is key to unlocking the true promise of CRISPR, and this collaboration with Mission Bio reflects another step forward to advancing the delivery of safer and more efficient genome editing platforms," said Sandy Ottensmann, VP/GM, Gene Writing and Editing at IDT. "By pairing the precision of rhAmpSeq with the single-cell resolution of Tapestri, we aim to support cell and gene therapy developers in driving the future of safe, life-changing therapies to benefit more patients."

Researchers and industry professionals can learn more about this cutting-edge approach at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting this month. Mission Bio and IDT will be presenting a poster detailing the study’s findings, demonstrating the power of integrated single-cell sequencing for genome editing validation.

The poster "Comprehensive On- and Off-target Validation Using Integrated rhAmpSeq and Targeted DNA Resequencing Single-Cell Technology for Gene Editing Applications" will be presented on Thursday, May 15 at 5:30 pm CST.

An oral presentation delivered by Dr. Ayal Hendel of Bar-Ilan University, "Single-Cell Profiling of Genome-Editing Alterations and Functional Outcomes in CRISPR-Engineered Cells," will take place on Saturday, May 17 at 9:00 am CST.

Visit Mission Bio’s Booth #1749 at ASGCT (Free ASGCT Whitepaper) to see how this technology can accelerate your research.

For more information, visit missionbio.com or contact [email protected].