On May 13, 2025 Olema Pharmaceuticals, Inc. ("Olema" or "Olema Oncology", Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of targeted therapies for breast cancer and beyond, reported financial and operating results for the first quarter ended March 31, 2025 (Press release, Olema Oncology, MAY 13, 2025, View Source [SID1234652984]).

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"During the first quarter, we continued to make important operational progress advancing our pipeline and we enter the second quarter well-positioned across the business," said Sean P. Bohen, M.D., Ph.D., President and Chief Executive Officer of Olema Oncology. "Our focus remains on our pivotal palazestrant program, laying the foundation to successfully initiate OPERA-02 in frontline metastatic breast cancer, while advancing OPERA-01 towards an anticipated top-line readout next year. We were also pleased to present promising new preclinical data at AACR (Free AACR Whitepaper) supporting the use of OP-3136, our potent KAT6 inhibitor, in a number of solid tumor applications beyond breast cancer. Investigator interest in our OP-3136 program remains strong and we are continuing to enroll patients in the Phase 1 study. With a clear strategy and strong balance sheet to support execution against our key priorities, we are working diligently to advance the promise of Olema’s science and striving to change the treatment paradigm for endocrine-driven cancers."

Recent Progress

Disclosed updated median progression-free survival (mPFS) from the ongoing Phase 1b/2 study of palazestrant in combination with cyclin-dependent kinase 4/6 inhibitor (CDK4/6i) ribociclib in patients with estrogen receptor-positive, human epidermal growth factor receptor 2-negative (ER+/HER2-) advanced or metastatic breast cancer at the TD Cowen 45th Annual Health Care Conference in March, including a mPFS of 13.8 months among all patients treated with 120 mg of palazestrant and 600 mg of ribociclib daily (n=56) and 13.1 months in patients previously treated with a CDK4/6i plus an endocrine therapy (n=40) as of a February 18, 2025 cutoff date.
Advanced the pivotal Phase 3 OPERA-01 trial of palazestrant as a monotherapy in second- and third-line (2/3L) ER+/HER2- metastatic breast cancer.
Continued enrollment in the Phase 1 study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of OP-3136 in participants with advanced solid tumors.
Presented new preclinical data for OP-3136 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April, demonstrating anti-tumor activity in pre-clinical in vitro and in vivo ovarian, non-small cell lung, and prostate cancer models, regardless of KAT6A expression status, as well as synergy with standard of care drugs.
Anticipated Upcoming Events

Present trial-in-progress poster, "OPERA-01: A randomized, open-label, phase 3 study of palazestrant (OP-1250) monotherapy vs standard-of-care endocrine therapy for patients with ER+, HER2- advanced breast cancer after endocrine and CDK4/6 inhibitor therapy," at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June; report top-line data from OPERA-01 in 2026.
Initiate the pivotal Phase 3 OPERA-02 trial of palazestrant in combination with ribociclib in frontline metastatic breast cancer in 2025.
Present mature data from the Phase 1b/2 trial of palazestrant in combination with ribociclib at an upcoming medical meeting.
First Quarter 2025 Financial Results
Cash, cash equivalents, and marketable securities as of March 31, 2025, were $392.7 million.

Net loss for the quarter ended March 31, 2025 was $30.4 million, as compared to $31.0 million for the quarter ended March 31, 2024. The decrease in net loss for the first quarter was related to higher interest income earned from marketable securities, primarily offset by increased spending on clinical development and research activities as a result of late-stage clinical trials for palazestrant and the advancement of OP-3136.

GAAP research and development (R&D) expenses were $30.6 million for the quarter ended March 31, 2025, as compared to $29.9 million for the quarter ended March 31, 2024. The increase in R&D expenses was primarily related to increased spending on clinical operations and development-related activities as the Company continues to advance palazestrant through late-stage clinical trials, and clinical operations and development-related activities associated with the advancement of OP-3136, and personnel-related costs, partially offset by a one-time $5 million milestone payment incurred to Aurigene and a decrease in non-cash stock-based compensation expense of $0.1 million.

Non-GAAP R&D expenses were $27.3 million for the quarter ended March 31, 2025, excluding $3.3 million non-cash stock-based compensation expense. Non-GAAP R&D expenses were $26.5 million for the quarter ended March 31, 2024, which included a $5.0 million milestone payment in connection with the Aurigene Agreement and excluded $3.4 million non-cash stock-based compensation expense. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release.

GAAP G&A expenses were $4.2 million for the quarter ended March 31, 2025, as compared to $4.5 million for the quarter ended March 31, 2024. The decrease in G&A expenses was primarily due to a decrease in non-cash stock-based compensation expense of $0.4 million, offset by increased spending on corporate-related costs.

Non-GAAP G&A expenses were $3.2 million for the quarter ended March 31, 2025, excluding $1.1 million non-cash stock-based compensation expense. Non-GAAP G&A expenses were $3.0 million for the quarter ended March 31, 2024, excluding $1.5 million non-cash stock-based compensation expense. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release.

On May 13, 2025 Monopar Therapeutics Inc. ("Monopar" or the "Company") (Nasdaq: MNPR), a clinical‐stage biopharmaceutical company focused on developing innovative treatments for patients with unmet medical needs, reported first quarter 2025 financial results and recent developments (Press release, Monopar Therapeutics, MAY 13, 2025, View Source [SID1234652983]).

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Recent Developments

ALXN1840 for Wilson Disease

On May 7, 2025, Monopar presented long-term efficacy and safety data for ALXN1840 (tiomolybdate choline) at the European Association for the Study of the Liver ("EASL") International Liver Congress 2025, a leading global conference in liver disease.

The data support ALXN1840 as a potential treatment for Wilson disease, a rare genetic disorder that causes toxic copper buildup in organs like the liver and brain. Pooled results from three clinical trials (n=255) showed sustained clinical benefits over a median treatment duration of 2.63 years. Safety data, which included an additional Phase 2 study (n=266), confirmed a favorable safety profile with fewer than 5% of patients experiencing a drug-related serious adverse event ("SAE") and no renal or urinary system SAEs.

Sustained neurological improvement as assessed by the physician as well as the patient using the Unified Wilson Disease Rating Scale ("UWDRS") was observed, as was sustained increased copper mobilization. Patients reported greater convenience and effectiveness when treated with ALXN1840 compared to standard of care, and improvement in the New Wilson Index (a prognostic indicator of the status of the liver) was also observed.

The Company is preparing to submit a New Drug Application ("NDA") to the U.S. Food and Drug Administration ("FDA") in early 2026.

MNPR‐101 for Radiopharmaceutical Use

The Company’s MNPR-101-Zr Phase 1 (imaging and dosimetry) and MNPR-101-Lu (therapeutic) Phase 1a clinical trials in advanced cancers are active and enrolling in Australia. Monopar continues its preclinical work with MNPR-101-Ac (therapeutic) with plans to enter the clinic in the future.

Financial Results for the First Quarter Ended March 31, 2025, Compared to the First Quarter Ended March 31, 2024

Cash and Net Loss

Cash, cash equivalents and investments as of March 31, 2025, were $54.6 million. Monopar expects that its current funds will be sufficient to continue operations at least through December 31, 2026, in order to: (1) assemble a regulatory package and file an NDA for ALXN1840; (2) continue to conduct and conclude its first-in-human imaging and dosimetry clinical trial with MNPR-101-Zr; (3) continue to conduct its first-in-human therapeutic clinical trial of MNPR-101-Lu; (4) advance its preclinical MNPR-101-Ac program into the clinic; and (5) invest in internal research and development projects to expand its radiopharmaceutical and rare disease pipeline.

Net loss for the first quarter of 2025 was $2.6 million or $0.38 per share compared to net loss of $1.6 million or $0.51 per share for the first quarter of 2024.

Research and Development ("R&D") Expenses

R&D expenses for the first quarter of 2025 were $1,643,000, compared to $966,000 for the first quarter of 2024. This represents an increase of $677,000 attributed to (1) a $611,000 increase in R&D personnel expenses including stock-based compensation and (2) a $69,000 increase in clinical trial site activity related to MNPR-101 for radiopharmaceutical use, partially offset by (3) a net decrease of $3,000 in other R&D expenses.

General and Administrative ("G&A") Expenses

G&A expenses for the first quarter of 2025 were $1,578,000, compared to $757,000 for the first quarter of 2024. This represents an increase of $821,000 primarily attributed to (1) a $416,000 increase in Board compensation resulting from the grant of stock options during the quarter ended March 31, 2025 (no stock options were granted during the quarter ended March 31, 2024), (2) a $291,000 increase in G&A personnel expenses including stock-based compensation, (3) a $73,000 increase in legal fees and (4) a $41,000 increase in insurance expenses.

Interest Income

Interest income for the first quarter of 2025 increased by $515,000, compared to the first quarter of 2024. The increase is attributed to interest earned on U.S. Treasury securities and higher bank balances in 2025, resulting from over $55 million of funds raised in the fourth quarter of 2024.

On May 13, 2025 Mereo BioPharma Group plc (NASDAQ: MREO) ("Mereo" or the "Company"), a clinical-stage biopharmaceutical company focused on rare diseases, reported its financial results for the first quarter ended March 31, 2025, and provided recent corporate highlights (Press release, Mereo BioPharma, MAY 13, 2025, View Source [SID1234652982]).

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"As we close out the first quarter of 2025, we continue to anticipate this will be an important, milestone-rich year for Mereo. The Phase 3 Orbit study of setrusumab in osteogenesis imperfecta remains on track to read-out either at the second interim analysis in mid-2025 or at the final analysis in the fourth quarter. We are continuing to invest in the pre-commercial activities for setrusumab to enable a successful launch in our European territory, following potential regulatory approvals," said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo. "Further, alvelestat is now Phase 3 ready and we are finalizing the trial start-up activities to support our ongoing partnering process. Along with our late-stage pipeline, we believe that continued close management of our cash balance will enable us to support our operations into 2027."

First Quarter 2025 Highlights, Recent Developments, and Anticipated Milestones

Setrusumab (UX143)

•
Continued progress in the two global Phase 3 studies led by our partner Ultragenyx:
o
The randomized, placebo-controlled Phase 3 portion of the Orbit study (in patients aged 5 to 25 years) is progressing toward a second interim analysis (IA2) in mid-2025 or a final analysis in the fourth quarter of 2025. All patients have now been on therapy for at least 12 months, conduct of the study is going well and patient safety in the Phase 3 portion of the study is consistent with safety observed in the Phase 2.
o
Patients in the Cosmic study (aged 2 to <7 years) are being treated with either setrusumab or intravenous bisphosphonates (IV-BP) therapy and will be evaluated in parallel with the Orbit interim analysis. If Orbit progresses to full study completion in the fourth quarter of 2025, Cosmic will also continue to a data read-out, to align with the Orbit read-out without spending alpha at the mid-year interim assessment.
•
Continued pre-commercial activities in Europe to support potential launch, including engagement with regulatory/HTA bodies and real-world data collection efforts through the SATURN program.

Alvelestat (MPH-966)

•
In first quarter of 2025, the European Commission granted Orphan Designation to alvelestat for the treatment of alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD). This adds to existing US FDA Orphan Drug and Fast Track designations.
•
The start-up activities for the planned single, global Phase 3 pivotal study are ongoing.
•
The Company remains in discussion with multiple potential development and commercialization partners.

First Quarter 2025 Financial Results

Total research and development ("R&D") expenses decreased by $0.1 million from $4.0 million in the first quarter of 2024 to $3.9 million in the first quarter of 2025. The decrease was primarily due to decreases of $1.2 million and $0.1 million in R&D expenses for alvelestat and etigilimab, offset by an increase of $1.3 million in R&D expenses for setrusumab. The decrease in program expenses for alvelestat was primarily due to undertaking reduced drug formulation and manufacturing activities in preparation for the Phase 3 study in the first quarter of 2025, compared to the first quarter of 2024. The increase in program expenses for setrusumab was primarily driven by amounts due under the manufacturing and supply agreement with our partner, Ultragenyx, ongoing activities related to real-world evidence programs and medical affairs activities in Europe and input into development, regulatory and manufacturing plans with Ultragenyx, who fund the global development of the program pursuant to our license and collaboration agreement.

General and administrative expenses increased by $1.4 million from $5.9 million in the first quarter of 2024 to $7.3 million in the first quarter of 2025. The increase was primarily due to the recognition of a $1.7 million reduction in expenses in the first quarter of 2024 for amounts received from our depository to reimburse certain expenses incurred by us in respect of our ADR program, partially offset by a net decrease in employee-related expenses and professional fees. A reimbursement in respect of our ADR program is anticipated in 2025.

Net loss for the first quarter of 2025 was $12.9 million, compared to $9.0 million during the first quarter of 2024, primarily reflecting an operating loss of $11.2 million and foreign currency translation loss.

As of March 31, 2025, the Company had cash and cash equivalents of $62.5 million, compared to $69.8 million as of December 31, 2024. The Company’s guidance remains unchanged, and it continues to expect, based on current operational plans, that its existing cash and cash equivalents balance will enable it to fund its currently committed clinical trials, operating expenses, and capital expenditure requirements into 2027. This guidance does not include any potential upfront payments associated with a partnership for alvelestat or business development activity around any of the Company’s non-core programs.

Total ordinary shares issued as of March 31, 2025 were 795,001,444. Total ADS equivalents as of March 31, 2025 were 159,000,288, with each ADS representing five ordinary shares of the Company.

On May 13, 2025 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported new research across more than 25 types of cancer and multiple treatment settings from the company’s broad and differentiated portfolio and pipeline will be showcased at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (May 30–June 3) (Press release, Merck & Co, MAY 13, 2025, View Source [SID1234652981]). Data highlight Merck’s commitment to rapidly advance research across multiple tumor types and continue to build on its portfolio of established medicines.

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"The new data we are presenting at ASCO (Free ASCO Whitepaper) spotlight our commitment in oncology as we pioneer novel treatment approaches in our ongoing work to transform cancer care," said Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories. "Merck is fueling the next wave of innovation in cancer through research to help address significant unmet medical needs for even more patients."

Key data being presented on investigational candidates from Merck’s pipeline:

First-time data from the colorectal cancer cohort of the Phase 1 KANDLELIT-001 study evaluating MK-1084, an investigational, oral selective KRAS G12C inhibitor, as monotherapy and in combination therapies for patients with KRAS G12C-mutated advanced colorectal cancer (Abstract #3508; Oral abstract session: Gastrointestinal cancer—colorectal and anal).1
Additional data from the non-small cell lung cancer (NSCLC) cohorts of the Phase 1 KANDLELIT-001 study evaluating MK-1084 as monotherapy and in combination therapies for patients with KRAS G12C-mutated metastatic NSCLC (Abstract #8605; Poster session: Lung cancer—non-small cell metastatic).1
First-time data from the Phase 2/3 waveLINE-003 study evaluating zilovertamab vedotin, an investigational antibody-drug conjugate (ADC) that targets receptor tyrosine kinase-like orphan receptor 1 (ROR1), plus standard of care as a treatment for patients with relapsed or refractory diffuse large B-cell lymphoma (Abstract #7005; Oral abstract session: Hematologic malignancies—lymphoma and chronic lymphocytic leukemia).
Data from the randomized Phase 2 OptiTROP-Lung03 study conducted in China, independently led by Kelun-Biotech, evaluating sacituzumab tirumotecan (sac-TMT), an investigational TROP2-directed ADC, as a treatment for patients with previously treated advanced EGFR-mutated NSCLC (Abstract #8507; Oral abstract session: Lung cancer—non-small cell metastatic).2
Additionally, new or updated findings from Merck’s broad portfolio of cancer medicines will be presented, including longer-term results for KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, and WELIREG (belzutifan), Merck’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, as well as findings in earlier stages of cancer.

Key data being presented on existing medicines in Merck’s portfolio:

Primary results from the first interim analysis of the Phase 3 ASCENT-04/KEYNOTE-D19 trial evaluating KEYTRUDA plus the TROP2-directed ADC sacituzumab govitecan-hziy for patients with previously untreated, locally advanced inoperable or metastatic triple-negative breast cancer will be featured as part of the ASCO (Free ASCO Whitepaper) press program (Abstract #LBA109; Oral abstract session: Breast cancer—metastatic).3
Five-year follow-up results from the Phase 3 KEYNOTE-564 trial evaluating KEYTRUDA monotherapy as an adjuvant treatment for patients with renal cell carcinoma at increased risk of recurrence following nephrectomy or following nephrectomy and resection of metastatic lesions (Abstract #4514; Rapid oral abstract session: Genitourinary cancer—kidney and bladder).
Five-year follow-up data from the Phase 2 LITESPARK-004 trial evaluating WELIREG as a treatment for von Hippel-Lindau (VHL) disease-associated tumors (Abstract #4507; Oral abstract session: Genitourinary cancer—kidney and bladder).
Results from the Phase 1b TROPION-Lung02 trial evaluating KEYTRUDA plus the TROP2-directed ADC datopotamab deruxtecan, with or without platinum-based chemotherapy, as a first-line treatment for patients with advanced NSCLC (Abstract #8501; Oral abstract session: Lung cancer—non-small cell metastatic).4
Data from the 4.5-year follow-up of the Phase 3 KEYNOTE-859 trial evaluating KEYTRUDA plus chemotherapy for the first-line treatment of advanced human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction adenocarcinoma (Abstract #4036; Poster session: Gastrointestinal cancer—gastroesophageal, pancreatic, and hepatobiliary).
Final analysis results from the Phase 3 KEYNOTE-A18 trial evaluating KEYTRUDA in combination with concurrent chemoradiotherapy (CRT) as a treatment for patients with high-risk locally advanced cervical cancer (Abstract #LBA5504; Oral abstract session: Gynecologic cancer).5
Exploratory efficacy analysis results from the Phase 3 KEYNOTE-689 trial evaluating KEYTRUDA as a neoadjuvant treatment for patients with resectable locally advanced head and neck squamous cell carcinoma, then continued as adjuvant treatment in combination with standard of care radiotherapy with or without cisplatin and then as a single agent (Abstract #6012; Rapid oral abstract session: Head and neck cancer).
Merck investor event

Merck will hold an Oncology Investor Event to coincide with the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting on Monday, June 2, 2025, 6 p.m. CT, during which senior management will provide an update on the company’s oncology strategy and program. The event will take place in Chicago, Ill., and will be accessible via webcast. Investors, analysts, members of the media and the general public are invited to listen to a webcast of the presentation via this weblink.

Details on abstracts listed above and additional key abstracts for Merck

Breast cancer

Sacituzumab govitecan (SG) + pembrolizumab (pembro) vs chemotherapy (chemo) + pembro in previously untreated PD-L1-positive advanced triple-negative breast cancer (TNBC): Primary results from the randomized Phase 3 ASCENT-04/KEYNOTE-D19 study. S. Tolaney.3

Abstract #LBA109, Oral abstract session: Breast cancer—metastatic

Sacituzumab tirumotecan (sac-TMT) as first-line treatment for unresectable locally advanced/metastatic triple-negative breast cancer (a/mTNBC): Initial results from the Phase II OptiTROP-Breast05 study. Y. Yin.2

Abstract #1019, Rapid oral abstract session: Breast cancer—metastatic

Gastrointestinal cancers

The KRAS G12C inhibitor MK-1084 for KRAS G12C–mutated advanced colorectal cancer (CRC): Results from KANDLELIT-001. I. Ługowska.1

Abstract #3508, Oral abstract session: Gastrointestinal cancer—colorectal and anal

KEYNOTE-859: 4.5-year median follow-up of pembrolizumab plus chemotherapy for previously untreated advanced HER2-negative gastric or gastroesophageal junction (G/GEJ) adenocarcinoma. S. Rha.

Abstract #4036, Poster session: Gastrointestinal cancer—gastroesophageal, pancreatic, and hepatobiliary

Quality-adjusted time without symptoms of disease or toxicity (Q-TWiST) analysis of pembrolizumab (pembro) versus chemotherapy (chemo) in microsatellite instability–high (MSI-H)/mismatch repair–deficient (dMMR) metastatic colorectal cancer (mCRC) in the KEYNOTE-177 trial. E. Elez.

Abstract #3631, Poster session: Gastrointestinal cancer—colorectal and anal

Genitourinary cancers

Five-year follow-up results from the Phase 3 KEYNOTE-564 study of adjuvant pembrolizumab (pembro) for the treatment of clear cell renal cell carcinoma (ccRCC). N. Haas.

Abstract #4514, Rapid oral abstract session: Genitourinary cancer—kidney and bladder

Hypoxia-inducible factor-2α (HIF-2α) inhibitor belzutifan in von Hippel-Lindau (VHL) disease–associated neoplasms: 5-year follow-up of the phase 2 LITESPARK-004 study. V. Narayan.

Abstract #4507, Oral abstract session: Genitourinary cancer—kidney and bladder

Phase 1b/2 KEYNOTE-365 cohort I: Pembrolizumab (pembro) plus carboplatin and etoposide chemotherapy (chemo) or chemo alone for metastatic neuroendocrine prostate cancer (NEPC). G. Amsberg.

Abstract #5059, Poster session: Genitourinary cancer—prostate, testicular, and penile

Gynecologic cancers

Pembrolizumab with chemoradiotherapy in patients with high-risk locally advanced cervical cancer: Final analysis results of the Phase 3, randomized, double-blind ENGOT-cx11/GOG-3047/KEYNOTE-A18 study. L. Duska.5

Abstract #LBA5504, Oral abstract session: Gynecologic cancer

Head and neck cancer

Neoadjuvant and adjuvant pembrolizumab plus standard of care (SOC) in resectable locally advanced head and neck squamous cell carcinoma (LA HNSCC): Exploratory efficacy analyses of the Phase 3 KEYNOTE-689 study. D. Adkins.

Abstract #6012, Rapid oral abstract session: Head and neck cancer

Hematologic cancers

WaveLINE-003: Phase 2/3 trial of zilovertamab vedotin plus standard of care in relapsed/refractory diffuse large B-cell lymphoma. P. Armand.

Abstract #7005, Oral abstract session: Hematologic malignancies—lymphoma and chronic lymphocytic leukemia

Lung cancer

MK-1084 for KRAS G12C-mutated (mut) metastatic non–small-cell lung cancer (mNSCLC): Results from KANDLELIT-001. A. Sacher.1

Abstract #8605, Poster session: Lung cancer—non-small cell metastatic

Sacituzumab tirumotecan (sac-TMT) in patients (pts) with previously treated advanced EGFR-mutated non-small cell lung cancer (NSCLC): Results from the randomized OptiTROP-Lung03 study. L. Zhang.2

Abstract #8507, Oral abstract session: Lung cancer—non-small cell metastatic

Patritumab deruxtecan (HER3-DXd) in resistant EGFR-mutated (EGFRm) advanced non-small cell lung cancer (NSCLC) after a third-generation EGFR TKI: The Phase 3 HERTHENA-Lung02 study. T. Mok.4

Abstract #8506, Oral abstract session: Lung cancer—non-small cell metastatic

TROPION-Lung02: Datopotamab deruxtecan (Dato-DXd) plus pembrolizumab (pembro) with or without platinum chemotherapy (Pt-CT) as first-line (1L) therapy for advanced non-small cell lung cancer (aNSCLC). B. Levy.4

Abstract #8501, Oral abstract session: Lung cancer—non-small cell metastatic

Sacituzumab tirumotecan (sac-TMT) in patients (pts) with previously treated locally advanced or metastatic (LA/M) non-small cell lung cancer (NSCLC) harboring uncommon EGFR mutations: Preliminary results from a Phase 2 study. L. Zhang.2

Abstract #8615, Poster session: Lung cancer—non-small cell metastatic

Sacituzumab tirumotecan (sac-TMT) in combination with tagitanlimab (anti-PD-L1) in first-line (1L) advanced non-small-cell lung cancer (NSCLC): Non-squamous cohort from the Phase II OptiTROP-Lung01 study. W. Fang.2

Abstract #8529, Poster session: Lung cancer—non-small cell metastatic

Melanoma and other skin cancers

First-line lenvatinib plus pembrolizumab versus placebo plus pembrolizumab in Chinese patients with unresectable or metastatic melanoma: Results from LEAP-003. J. Guo.6

Abstract #9553, Poster session: Melanoma/skin cancers

Multi-tumor

Pembrolizumab monotherapy for melanoma or non-small cell lung cancer in India: Results of the Phase IV KEYNOTE-593 study. S. Limaye.

Abstract #e21518, Publication only: Melanoma/skin cancers

On May 13, 2025 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering, developing, manufacturing and commercializing innovative antibody-based therapeutics for the treatment of cancer, reported an update on its recent corporate progress and announced financial results for the quarter ended March 31, 2025 (Press release, MacroGenics, MAY 13, 2025, View Source [SID1234652980]).

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"In the first quarter, we advanced our innovative pipeline of clinical product candidates. Notably, we dosed the first patient in our Phase 2 LINNET study of lorigerlimab, which will evaluate patients with platinum-resistant ovarian cancer and clear cell gynecologic cancers. We believe lorigerlimab, a differentiated bispecific checkpoint inhibitor, may be uniquely positioned to address the significant unmet need in both indications," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "We are focused on continued clinical execution this year and look forward to sharing our progress, including a clinical update from the LORIKEET Phase 2 study in the second half of 2025."

Updates on Proprietary Investigational Programs

Lorigerlimab is a bispecific, tetravalent PD-1 × CTLA-4 DART molecule designed to enhance CTLA-4 blockade on dual-expressing, tumor-infiltrating lymphocytes compared to a PD-1/CTLA-4 monoclonal antibody (mAb) combination therapy, while maintaining maximal PD-1 blockade on all PD-1-expressing cells.

The LORIKEET Phase 2 trial, a 150-patient randomized study of lorigerlimab in combination with docetaxel vs. docetaxel alone in second-line, chemotherapy-naïve patients with metastatic castration-resistant prostate cancer (mCRPC), is ongoing. The study was fully enrolled in late 2024 and the Company expects to provide a clinical update in the second half of 2025.
The first patient has been dosed in the LINNET Phase 2 study evaluating lorigerlimab as monotherapy in patients with either platinum-resistant ovarian cancer (PROC) or clear cell gynecologic cancer (CCGC). Both indications represent areas of unmet need and historically have been relatively insensitive to traditional anti-PD-1 antibody therapy. The LINNET study’s primary endpoint is objective response rate (ORR), with multiple secondary endpoints, including progression free survival (PFS) and duration of response (DoR). The Company anticipates enrolling up to 40 patients with PROC and up to 20 patients with CCGC in LINNET.
Emerging ADC Pipeline. MacroGenics is developing two clinical and one preclinical antibody-drug conjugate (ADC) molecules that each incorporate a novel, glycan-linked topoisomerase I inhibitor (TOP1i)-based payload developed by the Company’s collaboration partner, Synaffix (a Lonza company). These three ADC candidates are described below.

MGC026 targets B7-H3, an antigen with broad expression across multiple solid tumors and a member of the B7 family of molecules involved in immune regulation. MGC026 is currently being evaluated in a Phase 1 dose escalation study in patients with advanced solid tumors, with dose expansion in selected indications expected to initiate in 2025.

MGC028 targets ADAM9, a member of the ADAM family of multifunctional type 1 transmembrane proteins that play a role in tumorigenesis and cancer progression and is overexpressed in multiple cancers. The Company previously presented encouraging preclinical data showing both potent antitumor activity as well as acceptable safety. The Phase 1 study of MGC028 in patients with advanced solid tumors is ongoing.

MGC030 is a preclinical ADC that targets an undisclosed antigen expressed across several solid tumors. There are currently no approved therapeutics to this target. An Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for MGC030 is planned for 2026.
Updates on Selected Partnered Programs

MGD024 is a next-generation CD123 × CD3 DART molecule. Under an October 2022 exclusive option and collaboration agreement with Gilead Sciences, Inc. (Gilead), MacroGenics continues to enroll patients in a Phase 1 dose escalation study of MGD024 in patients with CD123-positive neoplasms, including acute myeloid leukemia and myelodysplastic syndromes. Gilead has the option to license MGD024 at predefined decision points during the Phase 1 study.
ZYNYZ (retifanlimab-dlwr) is a monoclonal antibody targeting PD-1 that the Company licensed to Incyte Corporation (Incyte) in 2017. In February 2025, Incyte disclosed that its supplemental Biologics License Application (sBLA) for retifanlimab in advanced/metastatic squamous cell carcinoma of the anal canal (SCAC) was filed with the FDA in December 2024, with approval anticipated in the second half of 2025. MacroGenics remains eligible to receive up to $540.0 million in additional development, regulatory and commercial milestones.
TZIELD (teplizumab-mzwv) is a monoclonal antibody targeting CD3 that the Company sold in 2018 to a partner that was subsequently acquired by Sanofi S.A. (Sanofi). In November 2022, TZIELD was approved by U.S. FDA to delay the onset of Stage 3 type 1 diabetes (T1D) in adult and pediatric patients aged 8 years and older with Stage 2 T1D. In April 2025, Sanofi disclosed that they anticipate TZIELD-related regulatory decisions in the E.U. and China in the second half of 2025. MacroGenics remains eligible to receive up to $379.5 million in additional development, regulatory and commercial milestones.
First Quarter 2025 Financial Results

Cash Position: Cash, cash equivalents and marketable securities balance as of March 31, 2025, was $154.1 million, compared to $201.7 million as of December 31, 2024.
Revenue: Total revenue was $13.2 million for the quarter ended March 31, 2025, compared to total revenue of $9.1 million for the quarter ended March 31, 2024. The increase was primarily due to an increase in revenue from collaborative and other agreements and contract manufacturing revenue, offset by a decrease in revenue from net product sales, resulting from the sale of MARGENZA to TerSera Therapeutics, LLC in November 2024.
R&D Expenses: Research and development expenses were $39.7 million for the quarter ended March 31, 2025, compared to $46.0 million for the quarter ended March 31, 2024. The decrease was primarily due to decreased costs related to margetuximab and MGC028, offset by increased costs related to MGC026.
SG&A Expenses: Selling, general and administrative expenses were $10.7 million for the quarter ended March 31, 2025, compared to $14.7 million for the quarter ended March 31, 2024. The decrease was primarily due to lower stock-based compensation expense and reduced professional fees. The reduction in professional fees was largely driven by the cessation of commercialization activities for MARGENZA.
Net Loss: Net loss was $41.0 million for the quarter ended March 31, 2025, compared to net loss of $52.2 million for the quarter ended March 31, 2024.
Shares Outstanding: Shares of common stock outstanding as of March 31, 2025 were 63,090,323.
Cash Runway Guidance: MacroGenics’ cash, cash equivalents and marketable securities balance of $154.1 million as of March 31, 2025, combined with projected and anticipated future payments from its partners, supports the Company’s cash runway into the second half of 2026. The Company’s expected funding needs reflect planned investments in ongoing clinical and preclinical programs. MacroGenics has implemented, and will continue to evaluate and execute, various cost-saving measures that are designed to extend its financial runway while continuing to progress its pipeline.
No Conference Call

The Company will not be hosting a conference call to discuss its financial results or corporate progress for the quarter ended March 31, 2025. The Company may resume its practice of hosting quarterly results conference calls in the future.