SERES THERAPEUTICS REPORTS FIRST QUARTER 2025 FINANCIAL RESULTS AND
PROVIDES BUSINESS UPDATES

On May 7, 2025 Seres Therapeutics, Inc. (Nasdaq: MCRB), (Seres or the Company), a leading live biotherapeutics company, reported first quarter 2025 financial results and provided business updates (Press release, Seres Therapeutics, MAY 7, 2025, View Source [SID1234652650]).

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"Advancing SER-155 through the next stage of development remains our top corporate priority, and we are making significant progress toward initiating the next clinical study," said Eric Shaff, President and Chief Executive Officer of Seres. "The clinical results generated to date underscore the potential of SER-155 to redefine the standard of care for allo-HSCT recipients and other vulnerable patients at risk of bloodstream infections— addressing a significant unmet medical need and representing substantial commercial opportunities. At the recent European Society for Blood and Marrow Transplantation conference, we obtained encouraging feedback from European transplant experts, consistent with the supportive views of U.S. based physicians, who highlighted that preventing BSIs remains a major unmet need in this patient population and expressed enthusiasm for both the safety and efficacy results reported in the SER-155 Phase 1b study. Notably, European clinicians also communicated their interest in participating in the further development of SER-155."

Mr. Shaff continued: "Guided by recent constructive FDA feedback and our aim to rapidly obtain clear and actionable SER-155 clinical results, we are preparing for a well-powered, placebo-controlled Phase 2 study, with a planned interim analysis. The efficacy and safety results of a successful Phase 2 study could support the design of, and advancement into, a subsequent single Phase 3 study for approval. We intend to submit a Phase 2 study protocol to the FDA in the coming weeks while we continue to actively engage in discussions seeking a partner to provide financial and other support and who shares our vision to realize the clinical and commercial value of SER-155."

Seres’ partnership discussions are focused on supporting SER-155 clinical advancement to reduce the risk of bloodstream infections, including life-threatening and anti-microbial resistance (AMR) infections, in medically vulnerable patient populations. The Company believes that SER-155 and other cultivated live biotherapeutic candidates could be developed in additional patient populations beyond allo-HSCT, including autologous-HSCT patients, cancer patients with neutropenia, CAR-T recipients, individuals with chronic liver disease, solid organ transplant recipients, as well as patients in the intensive care unit and long-term acute care facilities.

Recent Highlights

SER-155 and Bloodstream Infection Prevention


Based on recent US Food and Drug Administration (FDA) feedback and strategic considerations, Seres plans to advance SER-155 into a Phase 2 study that provides a time and capital-efficient path to obtaining clinical results and could support a subsequent Phase 3 study for product registration. Seres has selected a contract research organization, is planning for study start-up activities, and is manufacturing clinical trial material to support study initiation. The Company expects to submit a Phase 2 protocol to the FDA in the coming weeks. As previously noted, the Company requires additional capital to support the SER-155 Phase 2 study.


The Phase 2 study is expected to target 248 participants and incorporate an adaptive design and an interim data analysis when approximately half of the enrolled participants have reached the primary endpoint. The Company expects to obtain the interim clinical results within 12 months following study initiation which it believes will facilitate timely engagement with the FDA on the design of a Phase 3 study and inform development in adjacent medically vulnerable patient populations.


In April 2025, Seres presented SER-155 Phase 1b clinical and exploratory biomarker results at the 5th annual meeting of the European Society for Blood and Marrow Transplantation (EBMT). The Company’s presented poster was recognized by the EBMT scientific organizing committee and obtained the Best Clinical Poster Award.


In February 2025, clinical and exploratory biomarker results from Seres’ biotherapeutic programs were presented as a poster at the 2025 Tandem Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and Center for International Blood and Marrow Transplant Research (CIBMTR). SER-155 Phase 1b clinical study data were also featured in an oral presentation in the Best Abstracts in Infectious Diseases track at the Tandem meeting.

Development of Biotherapeutics for the Treatment of Inflammatory and Immune Diseases


In January 2025, Seres reported exploratory translational biomarker data from its SER-155 Phase 1b study which provided evidence supporting the intended therapeutic mechanisms, including promotion of intestinal epithelial barrier integrity to reduce the potential of bacterial translocation into the bloodstream, and reduction of systemic inflammatory responses. These results reinforce previously reported promising SER-155 clinical study efficacy and safety data.


The epithelial barrier and systemic inflammatory response data also further support the potential to develop Seres’ live biotherapeutics to address inflammatory and immune diseases, including ulcerative colitis and Crohn’s disease.


In May 2025, Seres presented data at the Digestive Disease Week (DDW) conference highlighting: (1) preclinical and clinical data that enable identification of patients with a disease etiology linked to the gastrointestinal microbiome, and (2) the identification of microbiome-based biomarkers that are predictive of response and suitable for patient selection and stratification in clinical trials. The Company’s poster, entitled "Candidate Biomarkers of Microbiome Disruption for Patient Selection or Stratification in Clinical Trials of Microbiome Therapies in Ulcerative Colitis" received a Poster of Distinction award in the Microbiome and Microbial Therapies subgroup. The Company believes that the data generated suggest that live biotherapeutics could provide a novel modality that could benefit patients living with gut-related inflammatory and immune diseases that are not effectively addressed today. Furthermore, research indicates that specific patient subpopulations optimally suited for biotherapeutic-based treatments may be identifiable. Seres’ research has been supported by the Crohn’s & Colitis Disease Foundation.


Seres is exploring options to advance the development of its investigational live biotherapeutics in inflammatory and immune diseases, including ulcerative colitis and Crohn’s disease.

Recent Corporate Update


On April 21, 2025, Seres effected a 1-for-20 reverse stock split of its common stock. Trading of Seres common stock on The Nasdaq Global Select Market commenced on a split-adjusted basis on April 22, 2025. On May 6, 2025, the Company was notified by the Nasdaq Listing Qualifications staff that it has regained compliance with the Nasdaq Bid Price Requirement.

Anticipated Upcoming Milestones and Events


Provide updates regarding SER-155 partnership discussions


Submit to FDA a protocol for a SER-155 Phase 2 study in allo-HSCT in the coming weeks


Present additional SER-155 clinical results at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 conference taking place from May 30 to June 3, 2025


Expecting receipt of the $25 million (less approximately $1.5 million in employment related obligations) installment payment from Nestlé in July 2025

Financial Results

The Company has classified all historical operating results for the VOWST business within discontinued operations in the consolidated statements of operations for the comparative period presented (3 months ended March 31, 2024). There is no ongoing activity in the current period related to discontinued operations.


Seres reported net income from continuing operations of $32.7 million for the first quarter of 2025, as compared to a net loss from continuing operations of $32.9 million for the same period in 2024. The net income in 2025 is primarily driven by the previously announced $50.0 million installment payment received from Nestlé in January, consistent with the Company fulfilling its transition obligations.


Research and development (R&D) expenses for the first quarter of 2025 were $11.8 million, compared with $19.5 million for the first quarter of 2024. The decrease in R&D expenses was primarily driven by lower personnel expenses, a decrease in platform investments, and a decrease in expenses related to SER-155 Phase 1b study.


General and administrative (G&A) expenses for the first quarter of 2025 were $11.9 million, compared with $14.9 million for the first quarter of 2024. The decrease in G&A expenses was primarily a result of lower personnel and contractor expenses and other cost management activities.


Manufacturing services expenses were $3.5 million for the first quarter of 2025. These costs relate to the provision of manufacturing services under the transition services agreement with Nestlé. The associated reimbursement received from Nestlé related to these expenses is recognized in other (expense) income, net.


On April 21, 2025, the Company effected a 1-for-20 reverse stock split of its common stock. The reverse stock split had no impact on the number of authorized shares or the par value of preferred and common stock. All share and per share amounts in the financial statements have been retroactively adjusted, for all periods presented, to give effect to the reverse stock split.

Cash Runway

As of March 31, 2025, Seres had $58.8 million in cash and cash equivalents. Based on the Company’s current cash, the anticipated second installment payment to be received from Nestlé in July 2025, transaction-related obligations and current operating plans, the Company expects to fund operations into the first quarter of 2026.

Conference Call Information

Seres’ management will host a conference call today, May 7, 2025, at 8:30 a.m. ET. The conference call may be accessed by calling 1-800-715-9871 (international callers dial 1-646-307-1963) and referencing the conference ID number 4618787. To join the live webcast, please visit the "Investors and News" section of the Seres website at www.serestherapeutics.com. A webcast replay will be available on the Seres website beginning approximately two hours after the event and will be archived for at least 21 days.

About SER-155

SER-155 is an investigational, oral, live biotherapeutic designed to decolonize gastrointestinal (GI) pathogens, improve epithelial barrier integrity, and induce immune homeostasis, to prevent bacterial bloodstream infections, including those that can harbor antimicrobial resistance (AMR), as well as other pathogen associated negative clinical outcomes, in patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).

SER-155 has been evaluated in a Phase 1b placebo-controlled study in patients undergoing allo-HSCT, which demonstrated a significant reduction in both bacterial bloodstream infections (BSIs) (77% relative risk reduction) and systemic antibiotic exposure, as well as lower incidence of febrile neutropenia. SER-155 has received Breakthrough Therapy designation for the reduction of bloodstream infections in adults undergoing allo-HCST and Fast Track designation for reducing the risk of infection and graft-versus-host disease in patients undergoing allo-HCST. The early development of the program was supported by Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X), a global non-profit partnership accelerating antibacterial products to address drug-resistant bacteria.

PAQ Therapeutics Announces $39 Million Series B Financing and Initiates Phase 1 Trial to Advance Novel Approach Addressing KRAS-Driven Cancers with High Unmet Need

On May 7, 2025 PAQ Therapeutics, a biotechnology company developing best- and first-in-class KRAS degraders for patients with lethal cancers lacking effective treatment options, reported the completion of its $39 million Series B funding (Press release, PAQ Therapeutics, MAY 7, 2025, View Source [SID1234652669]). The Series B round was co-led by Bayland Capital and MRL Ventures Fund (MRLV), with participation from Johnson & Johnson Innovation – JJDC, Inc. (JJDC), LAV Fund, BioTrack Capital, and existing investor Sherpa Health Partners.

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The capital raised will fund PAQ’s next stage of clinical development for the company’s lead asset PT0253, a potent and selective degrader of KRAS G12D, a known driver for a range of solid tumors. PT0253 has demonstrated best-in-class potential based on preclinical comparison to existing agents currently in clinical development targeting the same KRAS mutant. In Q1 2025, the first patient was dosed in a Phase 1 study to assess its safety and tolerability.

"The successful completion of our Series B funding and rapid enrollment of our Phase 1 trial in the US for PT0253 mark significant steps forward in our clinical development efforts," said Nan Ji, PhD, PAQ’s co-founder, President, and CEO. "The strong support from our investors validates the potential of our innovative approach. We now turn our focus to executing a robust clinical development plan while continuing to develop a differentiated pipeline of KRAS degraders."

The funds will also support the advancement of the company’s second asset through IND-enabling studies.

"PAQ represents a compelling opportunity to develop transformative therapies for oncology populations with high unmet need," said Olga Danilchanka, Partner, MRLV, the therapeutics-focused corporate venture arm of Merck & Co. "Their innovative efforts advancing the targeted protein degradation modality aligns with our commitment to backing scientifically rigorous teams that tackle pressing medical challenges. We’re confident in PAQ’s ability to unlock the full potential of KRAS degraders and are proud to support their journey toward achieving clinical impact."

"The team at PAQ is at the forefront of an innovative approach to treat some of the most underserved patient populations in oncology," said Yuexing Su, Founding Partner, Bayland Capital. "We were attracted to PAQ based on its scientific approach, preclinical data, and experienced leadership team. We believe there is tremendous potential for KRAS degraders and are excited to be part of PAQ’s next stage of growth."

PAQ Therapeutics was founded in 2020 and completed a $30M Series A financing in 2021. Over the last three years, the PAQ team gained proprietary understanding of KRAS biology, which guided medicinal chemistry to identify KRAS degraders. These programs have the potential to address key limitations of clinical-stage KRAS inhibitors, offering more effective and durable treatments.

Novo Nordisk’s sales increased by 19% in Danish kroner and by 18% at constant exchange rates to DKK 78.1 billion in the first three months of 2025

On May 7, 2025 Novo Nordisk reported the company’s sales increased by 19% in Danish kroner and by 18% at constant exchange rates to DKK 78.1 billion in the first three months of 2025 (Press release, Novo Nordisk, MAY 7, 2025, View Source [SID1234654078]).

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BeiGene Announces First Quarter 2025 Financial Results and Business Updates

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.

Syndax Announces Publication of Pivotal Revumenib Data in Relapsed or Refractory mNPM1 Acute Myeloid Leukemia in the Journal Blood

On May 7, 2025 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported that data from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib, a first-in-class menin inhibitor, in patients with relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML) have been published in Blood (Press release, Syndax, MAY 7, 2025, View Source [SID1234652651]).

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"We are thrilled to publish the first positive pivotal dataset in patients with an NPM1 mutation, the most common genetic alteration observed in AML," said Neil Gallagher, M.D., Ph.D., President, Head of Research and Development at Syndax. "These important data support the safety and efficacy of revumenib in relapsed or refractory mNPM1 AML and serve as the foundation for the sNDA we submitted to the FDA under its RTOR program."

"These are impressive, landmark results which underscore the potential for revumenib to meaningfully advance the standard of care for patients with R/R mNPM1 AML, a difficult to treat disease with a poor prognosis," said Martha L. Arellano, M.D., Professor of Hematology and Oncology, Winship Cancer Institute of Emory University, and Principal Investigator in the AUGMENT-101 trial. "In the older, heavily pretreated population enrolled in this trial, it is very encouraging to observe that nearly 50% of patients achieved an overall response and that revumenib was generally well-tolerated. These findings add to the growing body of evidence supporting revumenib’s paradigm-changing potential as treatment for a significant subset of acute leukemias."

In November 2024, revumenib was FDA approved as Revuforj for the treatment of R/R acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older. In April 2025, Syndax completed the submission of a supplemental New Drug Application (sNDA) for revumenib in R/R mNPM1 AML under the FDA’s Real-Time Oncology Review (RTOR) program. The sNDA is supported by the positive pivotal data reported from the Phase 2 portion of the AUGMENT-101 trial.

About the published data

The publication entitled "Menin inhibition with revumenib for NPM1-mutated (NPM1m) relapsed or refractory acute myeloid leukemia: AUGMENT-101" reports results from patients with R/R mNPM1 AML who received revumenib in the pivotal Phase 2 portion of the AUGMENT-101 trial.

The protocol-defined efficacy-evaluable population for the primary analysis included the first 64 adult patients with R/R mNPM1 AML. In this efficacy population, the median age was 65 (range: 19, 84). Patients were heavily pretreated with 36% having received three or more prior lines of therapy and 22% having received four or more prior lines of therapy (median prior lines: 2). 75% of patients were previously treated with venetoclax.

The study met the primary efficacy endpoint with a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% (15/64; 95% confidence interval [CI]: 14%-36%; one-sided p-value =0.0014). The median time to first CR+CRh was 2.76 months (range: 1.8-8.8) and the median duration of CR+CRh was 4.7 months (95% CI: 1.2-8.2). Responses were observed across the various subgroups assessed, including patients with multiple prior lines of therapy, prior venetoclax exposure, prior HSCT, and prior FLT3 inhibitor use.

The overall response rate (ORR)1 was 47% (30/64). Of the responders, 17% (5/30) proceeded to hematopoietic stem cell transplant (HSCT) while in remission, with three patients resuming revumenib after HSCT. The duration of therapy with revumenib post-transplant ranged from 2 to 60 weeks.

In this single-arm trial, the median overall survival (OS) observed was 4.0 months (95% CI: 2.5-7.2) among all 64 adults in the primary efficacy analysis based on the Kaplan-Meier estimate. The median OS observed was 23.3 months (95% CI: 7.2-not reached) among the 15 CR+CRh responders based on the Kaplan-Meier estimate.

The safety population included 84 adult and pediatric patients with R/R mNPM1 AML. The safety profile observed with revumenib in this population was consistent with previously reported data. Treatment-emergent serious adverse events that occurred in ≥5% of patients included: febrile neutropenia (21%), differentiation syndrome (13%), sepsis (13%), pneumonia (8%), anemia (7%), and QTc prolongation (7%). TEAEs leading to dose reduction or treatment discontinuations occurred in 12% (10/84) and 30% (25/84) of patients, respectively.

About Mutant NPM1 (mNPM1) Acute Myeloid Leukemia (AML)

Mutations in the NPM1 gene are the most common genetic alteration in adult AML and are observed in approximately 30% of cases. Patients with relapsed or refractory mNPM1 AML have a poor prognosis and high unmet need. Similar to KMT2A-rearranged acute leukemia, mNPM1 AML is highly dependent on the expression of specific developmental genes shown to be negatively impacted by inhibitors of the menin-KMT2A interaction. mNPM1 AML is routinely diagnosed through currently available screening techniques. There are currently no approved targeted therapies for mNPM1 AML.

About Revuforj (revumenib)

Revuforj (revumenib) is an oral, first-in-class, selective menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older.

Revumenib is in development for the treatment of R/R acute myeloid leukemia (AML) with a nucleophosmin 1 mutation (mNPM1). Positive pivotal data from the AUGMENT-101 trial in this population with revumenib as a monotherapy were recently reported and the Company submitted a supplemental NDA for revumenib in R/R mNPM1 AML in April 2025. Additionally, multiple trials of revumenib in combination with standard-of-care agents in mNPM1 AML or KMT2A-rearranged acute leukemia are ongoing or planned across the treatment landscape, including in newly diagnosed patients.

Revumenib was previously granted Orphan Drug Designation for the treatment of AML, ALL and acute leukemias of ambiguous lineage (ALAL) by the U.S. FDA and for the treatment of AML by the European Commission. The U.S. FDA also granted Fast Track designation to revumenib for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation and Breakthrough Therapy Designation for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation syndrome: Revuforj can cause fatal or life-threatening differentiation syndrome (DS). Symptoms of DS, including those seen in patients treated with Revuforj, include fever, dyspnea, hypoxia, peripheral edema, pleuropericardial effusion, acute renal failure, and/or hypotension. In clinical trials, DS occurred in 39 (29%) of 135 patients treated with Revuforj. DS was Grade 3 or 4 in 13% of patients and fatal in one. The median time to onset was 10 days (range 3-41 days). Some patients experienced more than 1 DS event. Treatment interruption was required for 7% of patients, and treatment was withdrawn for 1%.

Reduce the white blood cell count to less than 25 Gi/L prior to starting Revuforj. If DS is suspected, immediately initiate treatment with systemic corticosteroids (e.g., dexamethasone 10-mg IV every 12 hours in adults or dexamethasone 0.25-mg/kg/dose IV every 12 hours in pediatric patients weighing less than 40 kg) for a minimum of 3 days and until resolution of signs and symptoms. Institute supportive measures and hemodynamic monitoring until improvement. Interrupt Revuforj if severe signs and/or symptoms persist for more than 48 hours after initiation of systemic corticosteroids, or earlier if life-threatening symptoms occur such as pulmonary symptoms requiring ventilator support. Restart steroids promptly if DS recurs after tapering corticosteroids.

QTc interval prolongation: In the clinical trials, QTc interval prolongation was reported as an adverse reaction in 39 (29%) of 135 patients treated with Revuforj. QTc interval prolongation was Grade 3 in 12% of patients. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 8%, and the increase from baseline QTcF was greater than 60 msec in 18%. Revuforj dose reduction was required for 5% of patients due to QTc interval prolongation. QTc prolongation occurred in 16% of the 31 patients less than 17 years old, 33% of the 88 patients 17 years to less than 65 years old, and in 50% of the 16 patients 65 years or older.

Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to treatment with Revuforj. Perform an electrocardiogram (ECG) prior to initiation of Revuforj, and do not initiate Revuforj in patients with QTcF >450 msec. Perform an ECG at least once weekly for the first 4 weeks and at least monthly thereafter. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation.

Interrupt Revuforj if QTcF increases >480 msec and <500 msec, and restart Revuforj at the same dose twice daily after the QTcF interval returns to ≤480 msec
Interrupt Revuforj if QTcF increases >500 msec or by >60 msec from baseline, and restart Revuforj twice daily at the lower-dose level after the QTcF interval returns to ≤480 msec
Permanently discontinue Revuforj in patients with ventricular arrhythmias and in those who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia.
Embryo-fetal toxicity: Revuforj can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with Revuforj and for 4 months after the last dose of Revuforj.

ADVERSE REACTIONS

Fatal adverse reactions occurred in 4 (3%) patients who received Revuforj, including 2 with differentiation syndrome, 1 with hemorrhage, and 1 with sudden death.

Serious adverse reactions were reported in 99 (73%) patients. The most frequent serious adverse reactions (≥5%) were infection (24%), febrile neutropenia (19%), bacterial infection (17%), differentiation syndrome (12%), hemorrhage (9%), and thrombosis (5%).

The most common adverse reactions (≥20%) including laboratory abnormalities, were hemorrhage (53%), nausea (51%), phosphate increased (50%), musculoskeletal pain (42%), infection (41%), aspartate aminotransferase increased (37%), febrile neutropenia (35%), alanine aminotransferase increased (33%), parathyroid hormone intact increased (33%), bacterial infection (31%), diarrhea (30%), differentiation syndrome (29%), electrocardiogram QT prolonged (29%), phosphate decreased (25%), triglycerides increased (25%), potassium decreased (24%), decreased appetite (24%), constipation (23%), edema (23%), viral infection (23%), fatigue (22%), and alkaline phosphatase increased (21%).

DRUG INTERACTIONS

Drug interactions can occur when Revuforj is concomitantly used with:

Strong CYP3A4 inhibitors: reduce Revuforj dose
Strong or moderate CYP3A4 inducers: avoid concomitant use with Revuforj
QTc-prolonging drugs: avoid concomitant use with Revuforj. If concomitant use is unavoidable, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold Revuforj if the QTc interval is >480 msec. Restart Revuforj after the QTc interval returns to ≤480 msec.
SPECIFIC POPULATIONS

Lactation: advise lactating women not to breastfeed during treatment with Revuforj and for 1 week after the last dose.

Pregnancy and testing: Revuforj can cause fetal harm when administered to a pregnant woman. Verify pregnancy status in females of reproductive potential within 7 days prior to initiating Revuforj.

Pediatric: monitor bone growth and development in pediatric patients.

Geriatric: compared to younger patients, the incidences of QTc prolongation and edema were higher in patients 65 years and older.

Infertility: based on findings in animals, Revuforj may impair fertility. The effects on fertility were reversible.

To report SUSPECTED ADVERSE REACTIONS, contact Syndax Pharmaceuticals at 1-888-539-3REV or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Full Prescribing Information, including BOXED WARNING.