On March 19, 2026 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported financial results for the year ended December 31, 2025 and provided a clinical update highlighting rapid progress toward a major upcoming milestone in its pivotal MIRACLE trial of Annamycin in combination with cytarabine for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML).

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"With the MIRACLE trial rapidly progressing and the first MIRACLE interim readout now clearly within reach, we believe 2026 will be a defining year for Moleculin," said Walter Klemp, Chairman and CEO of Moleculin. "The encouraging blinded efficacy data seen so far, particularly in a pretreated population that includes venetoclax failures, reinforces our belief that Annamycin has the potential to represent a significant advancement in AML therapy."

Recent Highlights

Reported (n=30) MIRACLE Trial delivered 40% preliminary blinded CRc rate;
Received a new independent assessment for the absence of cardiotoxicity in subjects treated with Annamycin, bringing the total number of Annamycin treated subjects reviewed by its independent expert to 90;
Entered a new Annamycin research collaboration focused on glioblastoma multiforme (GBM) with CIC biomaGUNE to evaluate intra arterial delivery of Annamycin in preclinical models;
Announced positive results from a Phase 1 clinical trial of WP1066 in pediatric recurrent malignant brain tumors, showing safety and signs of antitumor immune responses and supporting potential progression to Phase 2;
Announced a grant-funded preclinical research agreement with the University of North Carolina at Chapel Hill to evaluate Annamycin for pancreatic cancer treatment; and
Announced a targeted new investigator-initiated Phase 1B/2 study of Annamycin in third-line pancreatic cancer with Atlantic Health, based on preclinical data suggesting activity in advanced pancreatic tumors.
Clinical Development Update

Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML)

The Company is currently evaluating Annamycin in combination with cytarabine (Ara-C), collectively referred to as AnnAraC, in its pivotal Phase 3 "MIRACLE" trial for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (R/R AML) following induction therapy. The MIRACLE trial is a global, adaptive Phase 2B/3 clinical study being conducted across the United States, Europe, and other international sites.

The study’s adaptive design will integrate data from the ongoing Phase 2B portion (i.e., Part A) with the eventual Phase 3 portion (i.e., Part B). In Part A, approximately 75 to 90 subjects are randomized in a 1:1:1 ratio to receive high-dose cytarabine (HiDAC) combined with either placebo, 190 mg/m² Annamycin, or 230 mg/m² Annamycin. These Annamycin doses were selected based on recommendations from the FDA during the Company’s End-of-Phase 2 meeting. The protocol allows for early unblinding of data at 45 subjects, enabling an initial readout of approximately 30 subjects treated with Annamycin plus cytarabine and 15 subjects in the control arm receiving cytarabine plus placebo. This initial unblinding is expected in mid-2026, following completion of treatment for the first 45 patients.

In February 2026, Moleculin reported a blinded preliminary composite complete remission (CRc) rate of 40% in the MIRACLE trial’s first 30 subjects treated and with blinded, preliminary efficacy data. This CRc rate is comprised of a complete remission (CR) rate of 30% and complete remission with partial hematological recovery (CRh) of 10%. MIRACLE is randomizing 1:1:1 of the three different arms described above. This includes the control arm of cytarabine plus placebo. Even with the control arm included, these preliminary, blinded results substantially outperform historical outcomes for CR with cytarabine alone in the treatment of R/R AML. Of particular note is that roughly 35% of the subjects treated to date are relapsed or refractory (R/R) from a venetoclax regimen, a subject population that is generally considered among the most challenging to address with 2nd line therapies. The subjects treated to date presented with a high degree of genetic markers that are considered predictive of poor treatment response. These efficacy rates were observed across six different countries.

For Part B of the trial, 222 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative. Data from the control arm and the optimum dose of Annamycin chosen in the Phase 2B portion of the trial will be combined with the data for the Phase 3 portion of the trial in determining efficacy and safety for the trial.

For additional information, the MIRACLE trial is registered on ClinicalTrials.gov under identifier NCT06788756.

Expected Milestones for Annamycin AML Development Program

Q1 2026: MIRACLE – 45th subject treated with unblinding of data for 45 subjects expected in mid-2026
Q3 2026: MIRACLE – 90 subjects treated and unblinding thereafter
2H 2026: MIRACLE – Start of Part B
2H 2026: Atlantic Health pancreatic cancer clinical trial begins
2027: Begin recruitment of 3rd line AML subjects
2027: Begin pediatric AML clinical study
2028: End recruitment of Part B
2028: Primary efficacy data for MIRACLE 2nd line subjects
2028: Begin submission of a Rolling New Drug Application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE
Soft Tissue Sarcoma (STS Lung Metastases) Program

Moleculin’s Phase 1B/2 trial of Annamycin for soft tissue sarcoma lung metastases demonstrated:

Median overall survival of 13.5 months
Achieved in a median seventh-line therapy population
These results compare favorably with historical outcomes in earlier-line therapies.
Pancreatic Cancer Program

An investigator-initiated Phase 1B/2 study evaluating Annamycin for third-line pancreatic cancer is expected to begin in 2026.

Preclinical research suggests Annamycin may have particular activity in pancreatic tumors due to its ability to concentrate in pancreatic tissue.

WP1066 Program

The Company’s STAT3-inhibiting immunomodulator WP1066 continues to advance through investigator-initiated clinical studies targeting glioblastoma and pediatric brain tumors.

Moleculin expects several important milestones in the coming months.

Summary of Financial Results for the Full Year 2025

Research and development (R&D) expense was $15.9 million and $17.7 million for the years ended December 31, 2025 and 2024, respectively. The decrease in R&D of $1.8 million is primarily attributable to a reduction in sponsored research activities during the current year compared to the prior year.

General and administrative (G&A) expenses and depreciation and amortization expenses were $9.2 million and $8.9 million for the years ended December 31, 2025 and 2024, respectively. The increase in G&A of $0.3 million was primarily attributable to higher regulatory and legal services, as well as increased consulting and investor relations expenses.

As of December 31, 2025, the Company had cash and cash equivalents of $8.9 million and prepaid expenses and other current assets of $0.8 million. The Company also had $3.5 million of accounts payable and $3.3 million of accrued expenses and other current liabilities. A significant portion of the accounts payable and accrued expenses were due to work performed in relation to our preclinical activities and our clinical trials. Management believes that cash resources as of December 31, 2025, along with $8.3 million in gross proceeds received via financing activities in the first quarter of 2026 will be sufficient to fund the Company’s planned operations into the third quarter of 2026.

(Press release, Moleculin, MAR 19, 2026, View Source [SID1234663750])

On March 19, 2026 Mereo BioPharma Group plc (NASDAQ: MREO) ("Mereo" or the "Company"), a clinical-stage biopharmaceutical company focused on rare diseases, reported financial results for the full year ended December 31, 2025, and provided an update on recent corporate developments.

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"In collaboration with our partner Ultragenyx, we have analysed a significant part of the data from the Phase 3 Orbit and Cosmic studies of setrusumab in osteogenesis imperfecta and continue to develop our understanding of the fracture data and the patient reported outcomes (PROs), especially in patients aged 2–18 years old. We believe that these data, which include pre-specified sub-groups and ad hoc analyses, may provide the basis for engagement with the regulatory agencies," said Denise Scots-Knight, Chief Executive Officer of Mereo. "There are no FDA or EMA therapies approved specifically for OI and although bisphosphonates are used to improved bone mineral density, it remains a high unmet need. Setrusumab has demonstrated statistically significant improvements in bone mineral density as well as compelling reductions in vertebral fractures and statistically significant improvements in PROs of disease pain and daily activity in pediatric and teenage patients. We look forward to providing updates on these efforts as we progress with next steps. Alongside this, our partnering discussions around alvelestat in AATD-LD are continuing to advance on multiple fronts and our partner āshibio has indicated that it plans to initiate a Phase 2 trial of vantictumab in osteopetrosis in the second half of this year. Following our cost reductions and delays to investment in manufacturing and pre-commercial activities for setrusumab, our revised financial runway into mid-2027 enables us to potentially deliver on several key milestones during 2026."

Setrusumab (UX143)

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As announced on December 29, 2025, the Phase 3 Orbit and Cosmic studies of setrusumab in OI did not achieve statistical significance against the primary endpoints of reduction in annualized clinical fracture rate compared to placebo or bisphosphonates, respectively.
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Both studies achieved high statistical significance against the key secondary endpoint of improvement in bone mineral density versus a placebo or versus a bisphosphonate (standard of care) comparator.
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The improvement in bone mineral density in the Cosmic study was associated with a decreased rate of fracture in this younger, more highly fracturing patient population, although this was not statistically significant.
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Additionally, the studies demonstrated reductions in vertebral fractures, which are a key contributor to pain and disability in this patient population and are known to drive treatment decisions, as well as improvements in patient-reported outcomes of disease severity, pain / comfort and daily activities which were statistically significant in the Orbit study in pediatrics and teens.
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The safety profile of setrusumab was consistent with that observed in prior studies.
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Further analyses of the data from both studies, including patient subgroups, are ongoing ahead of any planned interactions with the regulatory agencies.

Alvelestat (MPH-966)

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The site feasibility work for initiation of the global Phase 3 pivotal study has been completed.
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Based on previous discussions with the FDA and EMA, Mereo anticipates a single Phase 3 trial enrolling approximately 220 early- and late-stage AATD-LD patients evaluating alvelestat over an 18-month treatment period will support regulatory submissions in both the U.S. and Europe.
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The primary efficacy endpoint for potential U.S. approval will be the St. George’s Respiratory Questionnaire (SGRQ) Total Score, with lung density measured by CT scan serving as the primary endpoint for potential European regulatory approval.
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Mereo continues to be in active discussions with potential partners for the Phase 3 development and commercialization of alvelestat.
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Additionally, the Company has designed a potential Phase 2b study of alvelestat in bronchiectasis to further support the ongoing partnering discussions.

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The proposed study would enroll approximately 250 patients, randomized 1:1:1 to receive one of two alvelestat doses with standard of care or placebo with standard of care, with a primary efficacy endpoint of change in exacerbation rate at six months.
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The proposed trial design is intended to substantially de-risk potential Phase 3 development, as exacerbations are a required confirmatory endpoint in registrational trials.

Vantictumab (OMP18R5)

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The Company outlicensed vantictumab for autosomal dominant osteopetrosis Type 2 (ADO2) to āshibio, Inc. whilst retaining European rights.
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āshibio is responsible for funding the global program and, following regulatory discussions, plans to initiate a Phase 2 study in the second half of 2026.
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Vantictumab was previously investigated in Phase 1a/b oncology trials in around 100 patients with biomarker evidence of potential impact on osteoclast function and high bone turnover, which led to fragility fractures in some patients.
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āshibio reported promising pre-clinical data at ASBMR 2025 in an ADO2 mouse model, in which vantictumab significantly decreased areal bone mineral density and rescued the bone phenotype in this model.
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ADO2 is an inherited metabolic bone disorder characterized by impaired osteoclast function, with no currently approved therapies. This impaired osteoclast function results in the growth of dense, brittle bones leading to multiple fractures, osteomyelitis, bone pain, low blood counts and significant morbidity.

Full Year 2025 Financial Results

Total research and development ("R&D") expenses decreased by $3.2 million from $20.9 million in 2024 to $17.8 million in 2025. The decrease was primarily due to reductions in R&D expenses for alvelestat and etigilimab of $7.7 million and $1.0 million, respectively, partially offset by an increase of $5.7 million in R&D expenses for setrusumab. The reductions in program expenses for alvelestat was primarily due to the completion of the activities undertaken in preparation for the potential Phase 3 study, including drug formulation and manufacturing, in the year ended December 31, 2024. The increase in program expenses for setrusumab was primarily driven by amounts due under the manufacturing and supply agreement with our partner, Ultragenyx, as well as ongoing activities we undertake related to real-world evidence programs and medical affairs activities in Europe. These are in addition to costs we incur in relation to our collaboration with Ultragenyx, who fund the global development of the program, including input into development, regulatory and manufacturing plans.

General and administrative expenses decreased by $3.4 million from $26.4 million in 2024 to $23.0 million in 2025. The decrease was due to a lower accrual for annual cash bonuses of $1.2 million, along with a reduction in professional fees.

Net loss for the full year ended December 31, 2025, was $41.9 million, compared to $43.3 million during 2024, primarily reflecting an operating loss of $40.1 million and a foreign currency translation loss of $6.3 million, partially offset by interest income of $2.2 million and a benefit from research and development tax credit of $1.9 million.

As of December 31, 2025, the Company had cash and cash equivalents of $41.0 million, compared to $69.8 million as of December 31, 2024. The Company’s expects, based on current operational plans, that its existing cash and cash equivalents balance will enable it to fund its operating expenses, and capital expenditure requirements into mid-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 December 31, 2025 were 795,658,504. Total ADS equivalents as of December 31, 2025 were 159,131,700, with each ADS representing five ordinary shares of the Company.

(Press release, Mereo BioPharma, MAR 19, 2026, View Source [SID1234663749])

On March 19, 2026 Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the "Company"), a clinical-stage biopharmaceutical company focused on its Phase III clinical trial, FLAMINGO-01, which is evaluating Fast Track designated GLSI-100, an immunotherapy to prevent breast cancer recurrences, reported the initiation of new clinical sites in the US.

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The Phase III clinical trial has recently been activated at City of Hope, one of the largest and most advanced cancer research and treatment organizations in the United States – specifically at its network sites in Los Angeles and Orange counties, Arizona, Atlanta, and Illinois. Principal investigator at City of Hope, Hope S. Rugo, M.D., continues her participation on the Steering Committee. Her prior site at University of California San Francisco, where she is Professor Emeritus, is still participating in the study.

Dr. Rugo is division chief of breast medical oncology and a professor in the Department of Medical Oncology & Therapeutics Research at City of Hope. She also serves as director of the Women’s Cancers Program for City of Hope’s national network of cancer centers. A world-renowned expert in breast cancer and clinical trial design and execution, Dr. Rugo oversees women’s cancer research initiatives and clinical care at City of Hope. She is focused on expanding breast cancer clinical trials, advancing translational research, and standardizing care to improve patient outcomes. She is deeply committed to improving access to innovative new therapies for breast cancer patients everywhere and takes a compassionate, collaborative approach in her work.

Dr. Rugo has been directly involved in numerous projects that have established new standards of care for breast cancer. She has served on the steering committees of multiple clinical trials that led to the approval of agents such as PARP inhibitors, CDK4/6 inhibitors, PI3K inhibitors, checkpoint inhibitors, and antibody-drug conjugates, among others. Additionally, Dr. Rugo has led several studies aimed at minimizing therapy-related toxicity. She also served as co-chair of the Triple Negative Working Group of the Translational Breast Cancer Research Consortium, where she spearheaded groundbreaking multicenter clinical trials in collaboration with researchers, pharmaceutical companies, and clinical providers. As a physician-scholar with more than 500 peer-reviewed publications, Dr. Rugo served on the editorial board of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Education Committee, which included co-chairing the creation of new guidelines for the hormonal treatment of metastatic breast cancer.

Dr. Rugo commented, "I am excited to open this important trial across our City of Hope sites, expanding treatment opportunities for our patients with high-risk early stage HER2 positive breast cancer. It is truly an honor to now represent City of Hope on the Steering Committee. This study aligns with City of Hope’s interest in and the importance of harnessing the host immune system to reduce the risk of breast cancer recurrence."

Last year City of Hope launched a national clinical trials model that includes clinical sites across the United States in order to accelerate cancer research.

Dr. Jaye Thompson, VP Clinical and Regulatory Affairs, commented, "We are honored to have Dr. Rugo, a globally recognized leader in breast cancer, now participating in FLAMINGO-01 through her leadership at City of Hope. Her site’s expertise and commitment to advancing patient care, which I personally experienced when training these sites, is invaluable as we continue to study GLSI-100. The four City of Hope locations also strengthen our study footprint in some regions of the US where we were not previously covered, providing the study with access to additional population centers."

CEO Snehal Patel commented, "We started working with Dr. Rugo when she was the breast cancer leader at UCSF and joined our Phase III study and Steering Committee. We quickly benefited from her recommendations as the study was starting up and expanding into Europe. Her extensive experience in developing novel therapies, while minimizing toxicities, will guide us through the development of GLSI-100 as an effective and safe vaccine to prevent metastatic breast cancer in high-risk breast cancer survivors. With the addition of these new sites at City of Hope, planned expansion of US Oncology/Sarah Cannon sites, addition of sites in new countries, including potentially the United Kingdom and Canada, the total sites participating in FLAMINGO-01 could increase from the current 160 sites to up to 190-200 sites."

About FLAMINGO-01 Open Label Phase III Data

More than 1,000 patients have been screened with a current screen rate of approximately 800 patients per year. The 250 patient non-HLA-A*02 arm is now fully enrolled, where all patients received GLSI-100, which is 5 times more treated patients and recurrence rate data than the approximately 50 patients treated in the Phase IIb trial. The Primary Immunization Series (PIS), which includes the first 6 GLSI-100 injections over the first 6 months and is required to reach peak protection, is followed by 5 booster injections given every 6 months to prolong the immune response, thereby providing longer-term protection.

In the non-HLA-A*02 arm, a preliminary analysis of recurrence rates after the PIS is completed shows an approximately 70-80% reduction in recurrence rate.
This observation is trending similarly to the Phase IIb trial results and hazard ratio where HLA-A*02 patients were treated and where breast cancer recurrences were reduced up to 80% compared to a 20-50% reduction in recurrence rate by other approved products.
The immune response at baseline prior to any GLSI-100 treatment, the increasing immune response during the PIS, and the safety profile of non-HLA-A*02 patients is trending similarly to the HLA-A*02 arms of FLAMINGO-01 and to the Phase IIb study.

Analysis of the open label data from FLAMINGO-01 has been conducted in a manner that maintains the study blind. The open label recurrence rate, immune response, and safety data is based on the patients enrolled to date in FLAMINGO-01 and the data provided by the clinical sites so far, which is not completed or fully reviewed, and is thus preliminary. While comparing any preliminary FLAMINGO-01 data to the Phase IIb clinical trial data may be possible, these preliminary results are not a prediction of future results, and the results at the end of the study may differ.

About GLSI-100 Phase IIb Study

In the prospective, randomized, single-blinded, placebo-controlled, multi-center (16 sites led by MD Anderson Cancer Center) Phase IIb clinical trial of HLA-A*02 breast cancer patients, 46 HER2/neu 3+ over-expressor patients were treated with GLSI-100, and 50 placebo patients were treated with GM-CSF alone. After 5 years of follow-up, there was an 80% or greater reduction in cancer recurrences in the HER2/neu 3+ patients who were treated with GLSI-100, followed, and remained disease free over the first 6 months, which we believe is the time required to reach peak immunity and thus maximum efficacy and protection. The Phase IIb results can be summarized as follows:

80% or greater reduction in metastatic breast cancer recurrence rate over 5 years of follow-up with a peak immune response at 6 months and well-tolerated safety profile.
The PIS elicited a potent immune response as measured by local skin tests and immunological assays.

About FLAMINGO-01 and GLSI-100

FLAMINGO-01 (NCT05232916) is a Phase III clinical trial designed to evaluate the safety and efficacy of Fast Track designated GLSI-100 (GP2 + GM-CSF) in HER2 positive breast cancer patients who had residual disease or high-risk pathologic complete response at surgery and who have completed both neoadjuvant and postoperative adjuvant trastuzumab based treatment. The trial is led by Baylor College of Medicine and currently includes US and European clinical sites from university-based hospitals and academic and cooperative networks with plans to open up to 150 sites globally. In the double-blinded arms of the Phase III trial, approximately 500 HLA-A*02 patients are planned to be randomized to GLSI-100 or placebo, and up to 250 patients of other HLA types are planned to be treated with GLSI-100 in a third arm. The trial has been designed to detect a hazard ratio of 0.3 in invasive breast cancer-free survival, where 28 events will be required. An interim analysis for superiority and futility will be conducted when at least half of those events, 14, have occurred. This sample size provides 80% power if the annual rate of events in placebo-treated subjects is 2.4% or greater.

For more information on FLAMINGO-01, please visit the Company’s website here and clinicaltrials.gov here. Contact information and an interactive map of the majority of participating clinical sites can be viewed under the "Contacts and Locations" section. Please note that the interactive map is not viewable on mobile screens. Related questions and participation interest can be emailed to: [email protected]

About Breast Cancer and HER2/neu Positivity

One in eight U.S. women will develop invasive breast cancer over her lifetime, with approximately 300,000 new breast cancer patients and 4 million breast cancer survivors. HER2 (human epidermal growth factor receptor 2) protein is a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or over-expressor) levels.

(Press release, Greenwich LifeSciences, MAR 19, 2026, View Source [SID1234663748])

On March 19, 2026 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 and FLT3 inhibitor, reported its business update and financial results for the quarter ended December 31, 2025.

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Operational Highlights

TakeAim Lymphoma

Lakshmi Nayak, MD, Director of the Center for CNS Lymphoma at Dana-Farber Cancer Institute in Boston, presented a poster with updated clinical data in Primary CNS Lymphoma (PCNSL) at the 30th Annual Meeting of the Society for Neuro-Oncology (SNO) in November 2025. The poster, titled Preliminary Safety and Efficacy of Emavusertib (CA-4948) in Combination with Ibrutinib in Relapsed/Refractory Primary Central Nervous System Lymphoma Patients, showed results for 24 patients who have received treatment with emavusertib and ibrutinib, a BTK inhibitor (BTKi), for at least 1 cycle (28 days):
5 of 5 BTKi-naïve patients achieved objective response (100% ORR)
7 of 19 BTKi-experienced patients achieved objective response (37% ORR)
Median treatment duration for the 12 responders was 123 days (44 – 798 days)
9 additional patients were unable to complete 1 cycle of treatment; reasons for discontinuing treatment included adverse events unrelated to treatment, disease progression, and transition to hospice care.
Cecilia Merrigan, DNP, Mayo Clinic in Rochester, presented a poster with initial clinical data in Secondary CNS Lymphoma (SCNSL) at the 30th Annual Meeting of the Society for Neuro-Oncology (SNO) in November 2025. The poster, titled Promising Efficacy Signal in Secondary CNS Lymphoma Patients Treated with Emavusertib and Ibrutinib, showed results for 2 patients who had previously progressed on a BTKi. Adding emavusertib to their ibrutinib treatment resulted in 1 complete response (CR) and 1 stable disease (SD) with a 38% reduction in disease burden.
Curis continues to enroll PCNSL patients in the Company’s TakeAim Lymphoma study of emavusertib in combination with ibrutinib which, as a result of discussions with FDA and EMA, is intended to support filings for accelerated approval in PCNSL in the US and Europe. Emavusertib has been granted orphan drug designation by both FDA and EMA in PCNSL.
TakeAim CLL

Curis initiated an open label Phase 2 clinical study of emavusertib in combination with zanubrutinib, a BTKi, in patients with Chronic Lymphocytic Leukemia (CLL). The goal of combining emavusertib with a BTKi is to enable a dual blockade of NF-kB, a key driver of disease in CLL and NHL, by inhibiting both the TLR and BCR pathways. The current standard of care is BTKi, which blocks the BCR pathway and can deliver high response rates, though typically only partial responses. Previous clinical studies have shown that adding emavusertib, which blocks the TLR pathway, to a BTKi regimen can enable patients with NHL to achieve deeper responses, including complete remission or undetectable minimal residual disease (MRD) and the potential for time-limited treatment, outcomes which represent the potential for a paradigm shift in the management of CLL.
AML

Christina Papayannidis, MD, IRCCS Azienda Ospedaliero Universitaria di Bologna, presented a poster with initial clinical data in frontline AML at the 67th ASH (Free ASH Whitepaper) Annual Meeting in December. The AML triplet study (CA-4948-104) is evaluating the addition of emavusertib to the combination of azacitidine and venetoclax (aza-ven) in AML patients who have achieved complete remission on aza-ven but remain MRD-positive (MRD+). The first two cohorts in the study evaluate patients who received emavusertib for either 7 or 14 days in a 28-day cycle, in addition to their aza-ven treatment. The poster, titled Preliminary Pharmacokinetic and MRD Results from AML Patients Treated with 7- and 14-Day Dosing Schedule of Emavusertib added to Combination Therapy with Azacitidine and Venetoclax, showed results for 4 patients in the 7-day cohort and 6 patients in the 14-day cohort:
8 patients had central MRD samples
5 of 8 patients (62.5%) achieved MRD conversion (MRD+ to undetectable).
Corporate

On January 9, 2026, the Company announced the closing of a private placement (the "January 2026 PIPE Financing") with gross proceeds of up to $80.8 million, including initial gross proceeds of approximately $20.2 million with three series of warrants (A, B, and C) which can be exercised for up to $20.2 million each according to the terms and conditions of the financing agreement. All three series of warrants are currently exercisable at $0.75 per share and have the following termination conditions:

Series A warrants terminate on January 8, 2031
Series B warrants terminate 30 days after the Company announces dosing of the fifth patient in the Phase 2 clinical trial in CLL, subject to conditions defined in the financing agreement
Series C warrants terminate on January 8, 2027.
Fourth Quarter 2025 Financial Results

For the year ended December 31, 2025, Curis reported a net loss of $7.6 million, or $0.58 per share on both a basic and diluted basis, as compared to a net loss of $43.4 million, or $6.88 per share on both a basic and diluted basis in 2024. For the fourth quarter of 2025, Curis reported net income of $19.4 million or $1.23 per share on both a basic and diluted basis as compared to a net loss of $9.6 million or $1.25 on both a basic and diluted basis for the same period in 2024.

Revenues, net were $9.4 million and $10.9 million for the years ended December 31, 2025 and 2024, respectively. Revenues are comprised of royalty revenues related to Genentech and Roche’s net sales of Erivedge. Revenues were $1.1 million and $3.3 million for the fourth quarters of 2025 and 2024, respectively. As previously announced, on November 6, 2025, the Company sold to TPC Investments Royalty LLC, a limited liability company managed by Oberland, its interest in Curis Royalty LLC. The sale included the Erivedge intellectual property, other assets associated with Erivedge and the License Agreement with Genentech ("Erivedge"), in exchange for upfront consideration of $2.5 million and a release of the Company’s liability related to sale of future royalties to Oberland. In connection with such transaction, the Company transferred to Curis Royalty all rights to Curis Technology, Inventions and Joint Patents (each as defined in the License Agreement) and assigned the Company’s rights, duties and obligations under the License Agreement to Curis Royalty. Following the sale, the Company is no longer entitled to revenues under the License Agreement.

Research and development expenses were $28.3 million and $38.6 million for the years ended December 31, 2025 and 2024, respectively. The decrease was primarily attributable to lower employee-related, clinical, manufacturing and consulting costs. Research and development expenses were $5.8 million and $9.0 million for the fourth quarters of 2025 and 2024, respectively.

General and administrative expenses were $14.0 million and $16.8 million for the years ended December 31, 2025 and 2024, respectively. The decrease was primarily attributable to lower employee-related and legal costs. General and administrative expenses were $2.9 million and $3.4 million for the fourth quarters of 2025 and 2024, respectively.

Gain on release of liability related to sale of future royalties associated with sale of assets was the result of the sale of Erivedge. In the fourth quarter 2025, the Company recognized a non-cash $27.2 million gain and the liability related to sale of future royalties was extinguished.

Other expense, net was $1.9 million for the year ended December 31, 2025 and other income, net was $1.2 million for the year ended December 31, 2024. The increase was partially attributable to an increase in expense related to the sale of future royalties and a decrease in interest income. Other expense, net was $0.3 million and $0.6 million for the fourth quarters of 2025 and 2024, respectively.

As of December 31, 2025, Curis’s cash and cash equivalents totaled $5.1 million, and the Company had approximately 12.9 million shares of common stock outstanding.

Cash Runway Guidance

Curis believes its cash and cash equivalents as of December 31, 2025, together with initial gross proceeds of $20.2 million received in January 2026 and expected gross proceeds of up to an additional $20.2 million from the exercise of the January 2026 PIPE Financing Series B Warrants upon the public announcement of dosing the 5th CLL patient in our TakeAim CLL study expected later this year, should enable the Company’s planned operations into the second half of 2027.

Conference Call Information

Curis management will host a conference call today, March 19, 2026, at 4:30 p.m. ET, to discuss the business update and these financial results.

To access the live conference call, please dial (800)-836-8184 from the United States or (646)-357-8785 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed here on the Curis website in the Investors section.

(Press release, Curis, MAR 19, 2026, View Source [SID1234663747])

On March 19, 2026 Congruence Therapeutics, a clinical-stage, computationally-driven biotechnology company building a unique pipeline of pharmacological correctors for diseases of protein misfolding, including MC4R-deficient (MC4R-d) genetic obesity, GBA1-driven Parkinson’s disease and Alpha-1 antitrypsin (A1AT) deficiency, reported the closing of a US $39.5 million financing.

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The financing was co-led by new investor Dimension and existing investor OrbiMed. Additional investors included Amplitude Ventures, FSTQ, Lumira, Investissement Quebec, BDC Capital’s Thrive Venture Fund, Driehaus, and Silver Arc.

"Our mission is to translate biology, chemistry and protein structural insights into medicines that address disease at its molecular origin," said Dr. Clarissa Desjardins, co-founder and CEO of Congruence Therapeutics. "In addition to CGX-926, we are advancing multiple proprietary and collaborative programs. We thank our new and existing investors for their continued support."

Proceeds from the financing will support the Phase 1/1b study of CGX-926, the Company’s lead program for MC4R-deficient genetic obesity, in healthy subjects and patients with MC4R-deficiency. Congruence also plans to complete IND-enabling activities for two additional development candidates targeting GBA1-driven Parkinson’s Disease and Alpha-1 antitrypsin deficiency, with the goal of filing a CTA/IND for both programs in early 2027.

"Leveraging cutting edge molecular dynamics and machine learning, Congruence has built a best-in-class platform to unlock an exciting modality of medicines in small molecule correctors," said Zavain Dar, Founder & Managing Partner, Dimension. "In short order Clarissa and team have demonstrated technology and scientific leadership, joined with strong execution and capital efficiency. This is the promise of technologically enabled biotech. We’re thrilled to join as CGX-926 enters the clinic and we see the translation of the platform’s unique capabilities."

Congruence also continues to advance its R&D collaborations, including a recently expanded multi-target partnership with Ono Pharmaceuticals in which Congruence is leading the effort to discover small molecules for targets spanning the therapeutic areas of oncology, neurology, and immunology. The Company is separately advancing another collaboration with an undisclosed global pharmaceutical company on a difficult to drug metabolic target.

About Revenir Drug Discovery Platform

Revenir, Congruence’s proprietary computational drug discovery platform, captures the dynamic biophysical changes of proteins in different functional states, offering unique insights into protein function and their modulation. By examining surface features and a spectrum of biophysical descriptors across an ensemble of protein conformers, Revenir predicts small molecule induced modulation of underlying physiologic protein states. This platform-driven strategy underpins Congruence’s growing pipeline of first-in-class and best-in-class programs directed to genetically validated targets implicated in conditions associated with significant unmet medical need, supporting a readily scalable and repeatable value creation paradigm.

(Press release, Congruence Therapeutics, MAR 19, 2026, View Source [SID1234663746])