On March 27, 2026 Monopar Therapeutics Inc. ("Monopar," the "Company," "we") (Nasdaq: MNPR), a clinical-stage biopharmaceutical company developing innovative treatments for patients with unmet medical needs, reported the fourth quarter and full-year 2025 financial results and provided a summary of recent developments.

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"2025 was a productive year for Monopar, marked by multiple ALXN1840 data presentations, an important publication, a strengthened balance sheet and continued progress toward a planned New Drug Application submission for ALXN1840 in Wilson disease," said Chandler Robinson, MD, Chief Executive Officer of Monopar. "We also recently strengthened our leadership team with the addition of Susan Rodriguez as Chief Commercial and Strategy Officer as we prepare for the potential launch of ALXN1840. We are grateful to the Wilson disease patients and their families whose experiences have informed our efforts to advance ALXN1840."

Recent Program Developments

ALXN1840 – NDA Submission Planned for Mid-2026 for Wilson Disease

Wilson disease is a rare genetic disorder characterized by impaired copper elimination, resulting in toxic accumulation in organs such as the liver and brain. ALXN1840 binds and mobilizes copper and has a novel mechanism of action as an albumin tripartite complex ("ATC") activator that differentiates it from currently available first-line therapies.

Based on recent interactions with the U.S. Food and Drug Administration ("FDA"), Monopar plans to submit a New Drug Application ("NDA") for ALXN1840 in mid-2026.

ALXN1840 updates:

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EASL 2025: Presented pooled long-term efficacy and safety data (n=255; median treatment duration 2.63 years), with additional safety data (n=266) supporting a favorable safety profile, as a late-breaking abstract

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ANA 2025: Presented data demonstrating long-term neurological benefit; the abstract was selected for oral and poster presentation and designated an "Abstract of Distinction"

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Journal of Hepatology / AASLD 2025: Reported statistically significant improvement in copper balance, with sustained improvement in daily copper balance driven by increased fecal copper excretion

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EL-PFDD: Attended externally led patient-focused drug development ("EL-PFDD") meeting with the FDA on January 29, 2026. During the meeting, patients and caregivers described the burden of Wilson disease, shared their experience with the currently available treatments, and highlighted the urgent need for additional treatment options

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Upcoming 2026 presentations: Abstracts accepted for presentation at EASL 2026 and the American Academy of Neurology ("AAN") 2026 Annual Meeting, including:

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Tiomolybdate choline stabilizes liver disease and improves neurological symptoms as well as quality of life in treatment-experienced Wilson disease patients (EASL 2026 oral presentation)

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Greater clinical benefit with tiomolybdate choline versus standard-of-care in neurologic Wilson disease patients in the Phase 3 FoCus Trial (AAN 2026 late-breaking oral and poster presentation)

MNPR-101 Radiopharmaceutical Programs

MNPR-101-Zr (zirconium-89), MNPR-101-Lu (lutetium-177), and MNPR-101-Ac (actinium-225) target the urokinase plasminogen activator receptor ("uPAR"), which is expressed in multiple aggressive cancers, including triple-negative breast, colorectal, and pancreatic cancers.

MNPR-101 platform update:
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Ongoing Phase 1 clinical activity in Australia for MNPR-101-Zr and MNPR-101-Lu

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Investigational new drug ("IND") clearance received for MNPR-101-Lu to initiate a Phase 1 clinical trial in the US

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FDA-authorized physician-sponsored Expanded Access Program at Excel Diagnostics and Nuclear Oncology Center ("EDNOC") in Houston, Texas

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Preclinical development of MNPR-101-Ac

Financings

In 2025, Monopar strengthened its balance sheet through the following financing activities:

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Completed an underwritten public offering generating approximately $91.9 million, after a concurrent repurchase of common stock but before offering expenses

Results for the Fourth Quarter and Year Ended December 31, 2025, Compared to the Fourth Quarter and Year Ended December 31, 2024

Cash and Net Loss

Cash, cash equivalents and short-term investments as of December 31, 2025, were $140.4 million.

Monopar expects its current funds to support operations through at least December 31, 2027, including: (1) regulatory and potential commercial activities for ALXN1840; (2) continued development of MNPR-101 programs; and (3) internal research and development.

Net loss for the fourth quarter of 2025 was $5.2 million, or $0.61 per share, compared to $10.9 million, or $2.23 per share, for the fourth quarter of 2024.

Net loss for the year ended December 31, 2025, was $13.7 million, or $1.85 per share, compared to $15.6 million, or $4.11 per share, for the year ended December 31, 2024.

Research and Development ("R&D") Expenses

R&D expenses for the fourth quarter of 2025 were $3.9 million compared to $9.9 million for the fourth quarter of 2024. The decrease was primarily due to the absence of one-time expenses incurred in connection with the in-licensing of ALXN1840 in 2024, partially offset by higher R&D personnel expenses (driven by increased headcount and compensation), increased clinical material and manufacturing costs for the ALXN1840 program, and higher other R&D expenses.

R&D expenses for the year ended December 31, 2025, were $9.9 million compared to $13.0 million for the year ended December 31, 2024. The decrease was primarily due to the absence of one-time expenses incurred in connection with the in-licensing of ALXN1840 in 2024, as well as lower radiopharmaceutical clinical trial costs reflecting a shift in focus following the in-licensing, partially offset by higher R&D personnel expenses (driven by increased headcount and compensation), increased clinical material and manufacturing costs for the ALXN1840 program, and higher other R&D expenses.

General and Administrative ("G&A") Expenses

G&A expenses for the fourth quarter of 2025 were $2.2 million compared to $1.2 million for the fourth quarter of 2024. The increase was primarily due to higher Board of Directors (the "Board") and G&A personnel expenses (including stock-based compensation and bonuses), higher patent legal fees, and other increases in G&A expenses.

G&A expenses for the year ended December 31, 2025, were $6.8 million compared to $3.2 million for the year ended December 31, 2024. The increase was primarily due to higher Board and G&A personnel expenses (including stock-based compensation and bonuses), higher patent legal fees, and other increases in G&A expenses.

(Press release, Monopar Therapeutics, MAR 27, 2026, View Source [SID1234663979])

On March 27, 2026 Intensity Therapeutics, Inc. ("Intensity" or "the Company") (Nasdaq: INTS), a late-stage clinical biotechnology company focused on the discovery and development of novel intratumoral cancer therapies that are designed to kill tumors and increase immune system recognition of cancers using its proprietary non-covalent conjugation technology, reported 2025 year-end financial results and highlights, and provides a corporate update.

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Corporate Update

INVINCIBLE-4 Study: Phase 2 randomized open-label, multicenter study to analyze the clinical activity, safety, and tolerability of INT230-6 given before administration of the SOC treatment in patients with early-stage, operable triple negative breast cancer and SOC alone.
In March 2026, the Company reported the following:
•Preliminary observations of the INVINCIBLE-4 Study showed that five (5) out of seven (7) patients (71.4%) who received INT230-6 prior to SOC ("Cohort A") achieved a pathological complete response ("pCR") whereas two (2) out of six (6) (33%) patients in the SOC arm alone ("Cohort B") achieved a pCR, with one patient still to be evaluated.
•Forty-four percent (44%) fewer grade 3 or higher adverse events were observed in Cohort A compared to Cohort B.
•A protocol amendment was submitted to Swissmedic, Switzerland’s regulatory authority, and the Switzerland Ethics Committee to resume enrollment. Full approval to resume enrollment was granted on March 26, 2026.
The Company expects presentation of more detailed results for the seven (7) Cohort A patients at a future oncology conference.

INVINCIBLE-3 Study: Phase 3 open-label, randomized study testing INT230-6 as monotherapy compared to the SOC drugs in second- and third-line treatment for specific soft tissue sarcoma subtypes.
In March 2025, the Company paused new site activations and patient enrollments due to funding constraints. Before this pause, the trial had enrolled 21 patients. The Company continues to treat patients enrolled in this study, maintain the database, conduct pharmacovigilance, and conduct other study-related activities in cooperation with its third-party contract research organizations at significantly reduced ongoing costs during this pause. The Company has prioritized reinitiating patient enrollment and site activations during 2026 once sufficient funding is obtained.

IT-01 Study Manuscript Publication
In October 2025, the Company reported that eBioMedicine, a Lancet Discovery Science journal, published the Company’s phase 1/2 IT-01 clinical study manuscript, "Safety and Efficacy of Intratumourally Administered INT230-6 in Adult Patients with Advanced Solid Tumours: Results from an Open-Label Phase 1/2 Dose Escalation Study," for the treatment of metastatic or refractory cancers. The manuscript included the following data results:

•In heavily pretreated patients with advanced disease having over 20 different types of cancer who had progressed following multiple prior lines of therapy, intratumoral INT230-6 achieved:
◦A disease control rate of 75% (48/64 patients) and median overall survival ("mOS") of 11.9 months; these results compare favorably in phase 1/2 studies that historically reported an mOS of 4 to 7 months
◦In a metastatic sarcoma subset population receiving only INT230-6, the median overall survival was 21.3 months
•In an exploratory analysis comparing patients receiving INT230-6 at a total dose (in mL) that treated greater than 40% of the patient’s total tumor burden ("TTB") compared to those treated with less than 40% of their TTB, the:
◦Disease control rate was 83.3% (40/48) compared to 50% (8/16)
◦Median overall survival was 18.7 months (95% CI: 11.5–23.5) compared to 3.1 months (95% CI: 1.6–5.9) with a hazard ratio (HR) of 0.17 (95% CI: 0.081–0.342); P<0.0001
◦Improved survival was consistent across a range of low to high tumor burden and tumor sizes
•Approximately 20% of patients in the >40% group had uninjected tumors shrink, abscopal effects
•Fifteen of 64 patients survived for more than 21 months
•INT230-6 induced a qualitative decrease in proliferating cancer cells in injected tumors and a qualitative increase in activated T-cells infiltrating the tumor microenvironment
•No dose-limiting toxicities were reported among 64 monotherapy patients; seven patients had a grade 3 (10.9%) with no grade 4 or 5 treatment-related adverse events
•Pharmacokinetic results showed that greater than 95% of the active cytotoxic agents remained in the injected tumors

Cash and Cash Runway
In 2025, the Company raised over $20 million in gross proceeds through two public offerings, one registered direct offering, and ATM issuances. These successful capital-raising efforts strengthened Intensity’s balance sheet with cash and cash equivalents of $11.9 million as of December 31, 2025, and extended its current operating runway into the second quarter of 2027.

Lewis H. Bender, Founder, President, and CEO, stated, "Our data published in the Lancet’s eBioMedicine journal for the treatment of metastatic disease, and the results reported on the safety and efficacy in the INVINCIBLE-4 study were promising and unique for a locally-delivered oncology drug. With the capital raised in 2025 and an unused $60 million ATM facility in place, we intend to resume patient enrollment in both studies as soon as possible. The American Cancer Society ("ACS") estimates that roughly 6,400 more deaths occurred in 2025 than in 2024, a trend that continues every year. Intensity remains committed to helping patients live longer, healthier lives with less toxicity. The ACS data supports the conclusion that today’s cancer treatments have many limitations, and that the unmet medical need for new ideas and better cancer therapies remains as strong as ever."

2025 Year End Financial Results

Research and development expenses were $6.8 million for the year ended December 31, 2025, compared to $10.5 million for the same period in 2024. Clinical trial expenses decreased $2.8 million primarily due to lower INVINCIBLE-3 Study costs. In March 2025, the Company paused new site activations and patient enrollments in the INVINCIBLE-3 Study due to funding constraints. Prior to this pause, the trial had enrolled 21 patients. The Company will continue to treat all patients enrolled in this study in cooperation with our third-party contract research organizations during this pause, and the Company plans to restart site activations and patient enrollment as soon as possible. Contract manufacturing costs declined by $0.6 million, as there were no new manufacturing batches of INT230-6 in 2025, along with $0.5 million of lower stock-based compensation. These decreases were partially offset by $0.3 million of bonus accruals for 2025 compared to zero bonus accruals for 2024.

General and administrative expenses were $5.2 million for the year ended December 31, 2025, compared to $6.1 million for the same period in 2024. Stock-based compensation decreased $0.6 million in 2025, and legal, audit, consulting, insurance and other general & administrative costs decreased due to cost efficiencies and less corporate development activity compared to the prior year period. These decreases were partially offset by $0.5 million of bonus accruals for 2025 compared to zero bonus accruals for 2024.

Overall, net loss was $11.6 million for the year ended December 31, 2025, compared to a net loss of $16.3 million for the year ended December 31, 2024.

As of December 31, 2025, cash and cash equivalents totaled $11.9 million.

About INT230-6
INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug consists of two proven, potent anti-cancer agents, cisplatin and vinblastine sulfate, and a diffusion and cell penetration enhancer molecule ("SHAO") that non-covalently conjugates to the two payload drugs, facilitating the dispersion of potent cytotoxic drugs throughout tumors and allowing the active agents to diffuse into cancer cells. These agents remain in the tumor, resulting in a favorable safety profile. In addition to local disease control and direct tumor killing, INT230-6 causes a release of a bolus of neoantigens specific to the malignancy, leading to immune system engagement and systemic anti-tumor effects. Importantly, these effects are mediated without immunosuppression, which often occurs with systemic chemotherapy.

(Press release, Intensity Therapeutics, MAR 27, 2026, View Source [SID1234663978])

On March 27, 2026 Instil Bio, Inc. ("Instil") (Nasdaq: TIL), a biotechnology company focused on identifying and advancing innovative therapeutics, reported its fourth quarter and full year 2025 financial results and provided a corporate update.

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Recent Highlights:

In January 2026, Axion Bio, Inc., a wholly owned subsidiary of Instil, discontinued clinical development of AXN-2510 and entered into a termination agreement with ImmuneOnco Biopharmaceuticals (Shanghai) Inc. to terminate the license and collaboration agreement for AXN-2510 and AXN-27M.
Following this decision, Instil will focus on its next phase of strategic development through external innovation and disciplined capital deployment.
The company is pursuing potential acquisitions and in-licensing opportunities that could provide access to promising novel therapeutic candidates. Instil is evaluating opportunities across several therapeutic areas.
"We have been taking important steps to sharpen Instil’s strategic focus and position the company for its next phase of strategic development," said Bronson Crouch, Chief Executive Officer, Instil. "We are focused on identifying high-quality opportunities that can position Instil for long-term value creation. Our priority is to leverage our balance sheet to pursue acquisitions and in-licensing opportunities that can drive meaningful shareholder value. We believe this disciplined approach to capital deployment can allow us to build a differentiated pipeline and advance innovative therapies for patients with serious diseases."

There can be no assurance that any transaction will result from these efforts, nor as to the timing of any such outcome. Instil does not intend to provide further updates unless and until a specific transaction is approved or disclosure is otherwise deemed appropriate.

Fourth Quarter and Full Year 2025 Financial and Operating Results:

As of December 31, 2025, Instil had $76.3 million in total cash, cash equivalents, restricted cash and marketable securities, which consisted of $6.6 million in cash and cash equivalents, $0.2 million in restricted cash and $69.5 million in marketable securities, compared to $115.1 million in total cash, cash equivalents, restricted cash and marketable securities, which consisted of $8.8 million in cash and cash equivalents, $1.8 million in restricted cash, and $104.5 million in marketable securities, as of December 31, 2024. Instil expects that its cash, cash equivalents, restricted cash and marketable securities as of December 31, 2025 will enable it to fund its current operating plan beyond 2027.

In-process research and development expenses were nil and $10.0 million for the fourth quarter and full year ended December 31, 2025, respectively, compared to nil and $10.0 million for the fourth quarter and full year ended December 31, 2024.

Research and development expenses were $3.5 million and $24.7 million for the fourth quarter and full year ended December 31, 2025, respectively, compared to $1.1 million and $11.8 million for the fourth quarter and full year ended December 31, 2024, respectively.

General and administrative expenses were $6.1 million and $27.2 million for the fourth quarter and full year ended December 31, 2025, respectively, compared to $10.4 million and $44.2 million for the fourth quarter and full year ended December 31, 2024, respectively.

Restructuring and impairment charges were nil and $16.6 million for the fourth quarter and full year ended December 31, 2025, respectively, compared to $0.3 million and $7.5 million for the fourth quarter and full year ended December 31, 2024, respectively.

Net loss per share, basic and diluted was $1.21 and $10.70 for the fourth quarter and full year ended December 31, 2025, respectively, compared to $1.82 and $11.39 for the fourth quarter and full year ended December 31, 2024, respectively. Non-GAAP net loss per share, basic and diluted was $0.97 and $6.91 for the fourth quarter and full year ended December 31, 2025, respectively, compared to $1.08 and $7.59 for the fourth quarter and full year ended December 31, 2024, respectively.

(Press release, Instil Bio, MAR 27, 2026, View Source [SID1234663977])

On March 27, 2026 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") and MSD K.K. (Headquarters: Tokyo, Representative Director: Prashant Nikam, "MSD"), a subsidiary of Merck & Co., Inc., Rahway, NJ, USA, reported that an application for LENVIMA (lenvatinib), an orally available multiple receptor tyrosine kinase inhibitor (TKI) discovered by Eisai, has been submitted in Japan for the additional dosage and administration in combination with WELIREG (belzutifan), the first-in-class oral hypoxiainducible factor-2 alpha (HIF-2α) inhibitor from MSD, for the treatment of unresectable or metastatic renal cell carcinoma that has progressed after chemotherapy.

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This application is based on the results of the Phase 3 LITESPARK-011 trial evaluating the dual regimen of LENVIMA plus WELIREG for the treatment of patients with advanced renal cell carcinoma (RCC) whose disease progressed on or after treatment with anti-programmed death receptor-1 (PD-1)/ programmed death-ligand 1 (PD-L1) therapy. The data from this trial were presented at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (GU) Cancers Symposium in February 2026. At a pre-specified interim analysis with a median follow-up of 29.0 months (range, 19.3-49.2), the LENVIMA plus WELIREG combination therapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS), one of the primary endpoints, reducing the risk of disease progression or death by 30% (HR=0.70 [95% CI, 0.59-0.84]; p=0.00007) compared to cabozantinib. The safety profile of this combination was consistent with those reported for each agent administered as monotherapy, and no new safety signals were identified.

In 2022, approximately 435,000 people worldwide were newly diagnosed with kidney cancer, and about 156,000 people died from the disease. 1 In Japan, it is estimated that roughly 21,000 people were newly diagnosed and about 7,000 died in 2022.2 RCC accounts for approximately 85% of kidney cancers3, and the five-year survival rates for patients with stage III and IV RCC have been reported as 63%–78% and 27%–28%4, respectively, indicating that the disease still has a high unmet medical needs.

LENVIMA is approved in combination with KEYTRUDA (pembrolizumab) in Japan for the first-line treatment of unresectable or metastatic RCC. WELIREG is approved in Japan for the treatment of unresectable or metastatic RCC that has progressed following cancer chemotherapy. Additionally, supplemental New Drug Applications (sNDA) for the LENVIMA and WELIREG combination therapy for the treatment of adult patients with advanced RCC with a clear cell component following a PD-1 or PDL1 inhibitor has been accepted by the U.S. Food and Drug Administration (FDA), with a PDUFA (Prescription Drug User Fee Act) target action date set for October 4, 2026.

Eisai and MSD have been collaborating through the provision of information on LENVIMA in Japan since October 2018, and will work together to expedite the maximization of contribution to patients with cancer.

About LENVIMA (lenvatinib)

LENVIMA, discovered and developed by Eisai, is an orally available multiple receptor tyrosine kinase inhibitor that inhibits the kinase activities of vascular endothelial growth factor (VEGF) receptors VEGFR1 (FLT1), VEGFR2 (KDR), and VEGFR3 (FLT4). LENVIMA inhibits other kinases that have been implicated in pathogenic angiogenesis, tumor growth, and cancer progression in addition to their normal cellular functions, including fibroblast growth factor (FGF) receptors FGFR1- 4, the platelet derived growth factor receptor alpha (PDGFRα), KIT, and RET. In syngeneic mouse tumor models, LENVIMA decreased tumor-associated macrophages, increased activated cytotoxic T cells, and demonstrated greater antitumor activity in combination with an anti-PD-1 monoclonal antibody compared to either treatment alone. LENVIMA has been approved for the indications below.

Thyroid cancer
– Indication as monotherapy
(Approved mainly in Japan, the United States, Europe, China and Asia)
Japan: Unresectable thyroid cancer
The United States: The treatment of patients with locally recurrent or metastatic, progressive, radioiodine-refractory differentiated thyroid cancer (DTC)
Europe: The treatment of adult patients with progressive, locally advanced or metastatic, differentiated (papillary/follicular/Hürthle cell) thyroid carcinoma (DTC), refractory to radioactive iodine (RAI)

Hepatocellular carcinoma
– Indication as monotherapy
(Approved mainly in Japan, the United States, Europe, China and Asia)
Japan: Unresectable hepatocellular carcinoma
The United States: The first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)
Europe: The treatment of adult patients with advanced or unresectable hepatocellular carcinoma (HCC) who have received no prior systemic therapy
– Indication in combination with KEYTRUDA (generic name: pembrolizumab) and transarterial chemoembolization (Approved in China)

Thymic carcinoma
– Indication as monotherapy (Approved in Japan)
Japan: Unresectable thymic carcinoma

Renal cell carcinoma (In Europe other than the United Kingdom, the agent was launched under the brand name Kisplyx)
– Indication in combination with everolimus
(Approved mainly in the United States, Europe and Asia) The United States: The treatment of adult patients with advanced renal cell carcinoma (RCC) following one prior anti-angiogenic therapy
Europe: The treatment of adult patients with advanced renal cell carcinoma following one prior vascular endothelial growth factor (VEGF) targeted therapy
– Indication in combination with KEYTRUDA
(Approved mainly in Japan, the United States, Europe and Asia)
Japan: Radically unresectable or metastatic renal cell carcinoma
The United States: The first-line treatment of adult patients with advanced renal cell carcinoma
Europe: The first-line treatment of adult patients with advanced renal cell carcinoma

Endometrial carcinoma
– Indication in combination with KEYTRUDA
(Approved mainly in Japan, the United States, Europe and Asia)
Japan: Unresectable, advanced or recurrent endometrial carcinoma that progressed after cancer chemotherapy
The United States: The treatment of patients with advanced endometrial carcinoma that is pMMR or not microsatellite instability-high (MSI-H), as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation
Europe: The treatment of adult patients with advanced or recurrent endometrial carcinoma (EC) who have disease progression on or following prior treatment with a platinum-containing therapy in any setting and are not candidates for curative surgery.

About WELIREG (belzutifan)

WELIREG, Merck & Co., Inc., Rahway, NJ, USA’s, known as MSD outside of the United States and Canada, first-in-class hypoxia-inducible factor 2 alpha (HIF-2α) inhibitor, is an orally administered small-molecule designed to reduce transcription and expression of HIF-2α target genes associated with cellular proliferation, angiogenesis and tumor growth. By inhibiting HIF-2α signaling, WELIREG aims to disrupt key pathways certain tumors may use to adapt to low-oxygen conditions, including those that help promote abnormal blood vessel formation and support tumor survival. WELIREG has demonstrated antitumor activity in certain von Hippel-Lindau (VHL) diseaseassociated tumors, renal cell carcinoma and in pheochromocytoma or paraganglioma. As part of a broader clinical program, Merck & Co., Inc., Rahway, NJ, USA continues to research WELIREG monotherapy and combination approaches for people with genitourinary, breast and gynecologic cancers across a range of treatment settings to further define where HIF-2α inhibition may provide clinical benefit and to better understand which patients are most likely to respond. WELIREG has been approved in Japan for the treatment of certain von Hippel–Lindau (VHL) disease–associated tumors, as well as for unresectable or metastatic renal cell carcinoma that has progressed after chemotherapy.

LITESPARK-011 Results

Data from LITESPARK-011 (ClinicalTrials.gov, NCT04586231) were presented at the ASCO (Free ASCO Whitepaper) GU Symposium held in February 2026. LITESPARK-011 is a randomized, open-label Phase 3 trial (ClinicalTrials.gov, NCT04586231) evaluating WELIREG in combination with LENVIMA compared to cabozantinib for the treatment of patients with advanced clear cell RCC that has progressed on or after anti-PD-1/L1 therapy. The dual primary endpoints are progression-free survival (PFS) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) as assessed by blinded independent central review (BICR), and overall survival (OS). Secondary endpoints include objective response rate (ORR) per RECIST v1.1 as assessed by BICR, duration of response (DOR) per RECIST v1.1 as assessed by BICR, and safety. The trial enrolled 747 patients who were randomized to receive WELIREG (120 mg orally once daily) plus LENVIMA (20 mg orally once daily) or cabozantinib (60 mg orally once daily).

At a pre-specified second interim analysis with a median follow-up of 29.0 months (range, 19.3- 49.2), WELIREG plus LENVIMA demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of PFS, reducing the risk of disease progression or death by 30% (HR=0.70 [95% CI, 0.59-0.84]; p=0.00007) compared to cabozantinib. For WELIREG plus LENVIMA, the median PFS was 14.8 months (95% CI, 11.2-16.6) versus 10.7 months (95% CI, 9.2-11.1) for cabozantinib. A trend toward improvement in overall survival (OS), the trial’s other primary endpoint, was also observed for WELIREG plus LENVIMA (HR=0.85 [95% CI, 0.68-1.05]; p=0.06075). The median OS was 34.9 months (95% CI, 27.5-NR) for WELIREG plus LENVIMA versus 27.6 months (95% CI, 24.0-31.4) for cabozantinib. The trial is continuing, and OS will be evaluated at a subsequent analysis per the clinical trial protocol. Regarding secondary endpoints, at the first interim analysis with a median follow-up of 19.6 months (range, 9.9-39.8), WELIREG plus LENVIMA met ORR, demonstrating a statistically significant improvement compared to cabozantinib. A confirmed ORR of 52.6% (95% CI, 47.3-57.7) was observed for WELIREG plus LENVIMA versus 39.6% (95% CI, 34.6-44.8) for cabozantinib. At the second interim analysis with a median follow-up of 29.0 months, the median DOR was 23.0 months (95% CI, 2.0-44.3+) for WELIREG plus LENVIMA versus 12.3 months (95% CI, 1.8+-35.9+) for cabozantinib. WELIREG plus LENVIMA was administered to 370 patients and cabozantinib was administered to 371 patients. Grade ≥3 treatment-related adverse events (TRAEs) occurred in 71.6% of patients receiving WELIREG plus LENVIMA versus 65.8% of patients receiving cabozantinib. Adverse events led to treatment discontinuation in 11.1% of patients receiving WELIREG plus LENVIMA and in 11.3% of patients receiving cabozantinib, respectively. Serious adverse events were observed in 51.6% of patients receiving WELIREG plus LENVIMA versus 43.9% of patients receiving cabozantinib, and AEs led to death in 5.4% of patients (two were treatment-related: thrombotic microangiopathy [n=1] and pneumonitis [n=1]) versus 3.2% (one was treatment-related: hemoptysis [n=1]) of patients, respectively.

(Press release, Eisai, MAR 27, 2026, View Source [SID1234663976])

On March 27, 2026 Autolus Therapeutics plc (Nasdaq: AUTL), a commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, reported its operational and financial results for the fourth quarter and full year ended December 31, 2025.

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"Autolus had a strong first year of launch of AUCATZYL in the US building a market leading position in adult patients with relapsed or refractory B-ALL and demonstrating strong commercial execution, including reliable, high-quality product delivery with consistent turn-around time. Parallel to the launch, the ROCCA consortium collected real world data from approximately 60% of the commercial patients treated with AUCATZYL. Recently reported data confirm a high level of clinical activity without inducing high grade CRS and only 3% of patients experiencing high grade ICANS. We expect this positive customer experience will be a key driver for the future growth of AUCATZYL," said Dr. Christian Itin, Chief Executive Officer of Autolus.

Dr. Itin continued, "Our focus in 2026 will be on driving adoption of AUCATZYL in the US, launching in the UK and expanding the utility of obe-cel in additional indications while leveraging our established commercial and manufacturing capabilities. Based on regulatory feedback we are executing two compact pivotal studies: CATULUS in pediatric r/r B-ALL and LUMINA in severe lupus nephritis patients. In addition, we are exploring the utility of obe-cel in progressive MS patients in the Phase 1 BOBCAT study. Clinical data updates are planned for long-term follow up of the Phase 1 CARLYSLE data in severe SLE patients, initial clinical experience in light chain amyloidosis from the ALARIC study with AUTO8 and initial data from the BOBCAT study by the end of 2026."

Product and Pipeline Updates:

AUCATZYL Launch
Autolus reported net product revenue of $23.3 million* for the three months ended December 31, 2025, and $74.3 million* for the year ended December 31, 2025, driven by U.S. sales.
Following a successful National Institute for Health and Care Excellence (NICE) evaluation, AUCATZYL launched in the UK in January 2026 and is now available under routine commissioning.
Data from the ROCCA (Real-World Outcomes Collaborative for CAR T in Adult ALL) consortium database evaluating patient characteristics, toxicity and response after real-world administration of AUCATZYL was presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2025 and the TANDEM meeting in February 2026. Real-world data show consistency in both safety and efficacy compared to the FELIX clinical trial that was the basis for regulatory approvals. The ROCCA Consortium registry covers approximately 60% of U.S. commercial patients at a data cutoff of January 5, 2026.
Obe-cel data in pediatric r/r B-ALL
Preliminary data from the CATULUS Phase 1 trial of obe-cel in pediatric relapsed or refractory (r/r) B-ALL patients were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2025. Obe-cel demonstrated high remission rates in pediatric patients with high-risk r/r B-ALL with overall response rate (ORR) of 95.5%. Low rates of high-grade cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were observed, consistent with obe-cel’s adult safety profile. The Phase 2 portion of the trial is underway and Autolus expects to report data at the end of 2027.
In October 2025, the U.S. Food and Drug Administration (FDA) granted regenerative medicine advanced therapy (RMAT) designation to obe-cel for the treatment of pediatric patients with r/r B-ALL. The RMAT designation is a program created under the 21st Century Cures Act to accelerate development and regulatory review of regenerative medicine therapies, including cell therapies, intended to treat serious or life-threatening diseases.

Obe-cel in lupus nephritis

Data from the ongoing Phase 1 CARLYSLE trial in patients with severe refractory systemic lupus erythematosus (srSLE) were reported at the American College of Rheumatology (ACR) Convergence 2025 and the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. All patients show deep B-cell depletion after infusion, suggesting an immune reset. No ICANS or high-grade CRS were observed in the nine patients evaluable for safety.
Data support progressing obe-cel as a treatment for LN and 50 million cells was selected as the recommended Phase 2 dose.
Autolus has previously aligned with the US Food and Drug Administration (FDA) on a Phase 2 trial design in LN and potential registrational path to approval. The LUMINA trial is now enrolling and the Company expects to report data in 2028.
Obe-cel in progressive multiple sclerosis
Autolus has advanced obe-cel into initial clinical development to explore treatment in progressive MS. The first patient in the BOBCAT trial was dosed in October 2025. The Phase 1 trial, expected to include up to 18 adult patients, will determine the safety, tolerability, and preliminary efficacy of obe-cel in participants with refractory progressive forms of MS. The Company expects to report initial data from the trial at the end of 2026 and full data in 2027.
AUTO8 in AL-Amyloidosis
The first patient was dosed in the Phase 1 ALARIC trial evaluating AUTO8 in light-chain amyloidosis and initial data is expected to be reported at the end of 2026.

Q4 2025 Operational Updates:

In the fourth quarter of 2025, Autolus initiated an overall manufacturing life cycle plan to facilitate additional cost reductions and gross margin improvements as the Company plans to expand obe-cel into new indications and pursue larger market opportunities. The initiatives are focused on 1) optimizing the Company’s current manufacturing operating model; 2) enhancing automation opportunities for the Company’s existing manufacturing process; and 3) developing a next-generation manufacturing platform with a step change in the cost and capacity profile. The Company plans to provide a detailed update on these plans in mid-2026.

Outlook:
For 2026, the Company continues to project AUCATZYL net product revenue of between $120 million to $135 million.

Increasing patient numbers in 2026 are expected to improve manufacturing plant utilization and together with operational efficiencies, Autolus expects a shift to positive gross margin in 2026.

Based on current operating plans, including anticipated AUCATZYL net revenues, Autolus expects that its current and projected cash, cash equivalents and marketable securities will be sufficient to fund the Company’s operations into Q4 2027.

Summary of Anticipated News Flow:

Longer-term follow up data from CARLYSLE trial
Year End 2026
Initial clinical data from BOBCAT Phase 1 trial in progressive MS Year End 2026
Initial clinical data from ALARIC Phase 1 trial in AL amyloidosis
(UCL collaboration) Year End 2026
BOBCAT trial progressive MS Phase 1 full data 2027
CATULUS trial pediatric Phase 2 data Year End 2027
LUMINA trial LN Phase 2 data 2028

ALL: acute lymphoblastic leukemia
SLE: systemic lupus erythematosus
LN: lupus nephritis
MS: multiple sclerosis
ALA: light-chain amyloidosis

Virtual Investor Event: Spotlight on Acute Lymphoblastic Leukemia (ALL) Program
April 8, 2026
1:00pm EDT / 6:00pm BST
A live webcast of the event will be available on the investor relations section of the Autolus website: View Source

Financial Results for the Quarter Ended December 31, 2025

Product revenue, net for the three months ended December 31, 2025, was $23.3 million*.

Cost of sales increased from $11.4 million to $25.3 million for the three months ended December 31, 2025, compared to the same period in 2024. This increase was primarily due to product sales in Q4 2025 and to the timing of commercial manufacturing activity expenses upon FDA approval of AUCATZYL in November 2024. Additionally, cost of sales in Q4 2025 includes cancelled orders in the period, patient access program product, inventory reserves and write-offs and third-party royalties for certain technology licenses.

Research and development expenses increased to $35.6 million from $30.8 million for the three months ended December 31, 2025, compared to the same period in 2024. This change was primarily due to an increase in research and development activities including clinical trial costs and a reduction in the UK R&D tax credit, partially offset by commercial manufacturing-related employee and infrastructure costs shifting to cost of sales and inventory.

Selling, general and administrative expenses increased to $35.8 million from $33.7 million for the three months ended December 31, 2025, compared to the same period in 2024. This increase was primarily due to salaries and other employment-related costs, driven by increased headcount supporting commercialization activities.

Loss from operations for the three months ended December 31, 2025, was $72.5 million, as compared to $75.9 million for the same period in 2024.

Net loss was $90.3 million for the three months ended December 31, 2025, compared to $27.6 million for the same period in 2024. Basic and diluted net loss per ordinary share for the three months ended December 31, 2025, totaled $(0.34), compared to basic and diluted net loss per ordinary share of $(0.10) for the same period in 2024.

Cash, cash equivalents and marketable securities at December 31, 2025, totaled $300.7 million, as compared to $588.0 million at December 31, 2024. The decrease was primarily driven by net cash used in operating activities and impacted by a delayed cash receipt of approximately $18.6 million in the Company’s 2023 R&D tax credit expected from the UK HMRC.

(Press release, Autolus, MAR 27, 2026, View Source [SID1234663974])