On March 19, 2026 Relmada Therapeutics, Inc. (Nasdaq: RLMD, "Relmada" or the "Company"), a clinical-stage biotechnology company advancing innovative therapies for oncology and central nervous system disorders, reported audited financial results for the fourth quarter and full year ended December 31, 2025 and provided a corporate update highlighting significant progress across its pipeline.

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"This has truly been a transformational year for Relmada, marked by significant progress with our lead program NDV-01," said Sergio Traversa, Chief Executive Officer of Relmada Therapeutics. "Our recently reported 12-month data for NDV-01 demonstrated durable complete responses with a favorable safety profile, reinforcing the program’s potential to become a best-in-class therapy for patients with non-muscle invasive bladder cancer. With a successful $160 million PIPE financing and regulatory alignment with the FDA on two registrational pathways, we believe that we are well positioned to advance NDV-01 into the Phase 3 RESCUE program in mid-2026. Our team is now focused on executing this plan and initiating the RESCUE registrational program as we work to bring NDV-01 to patients as efficiently as possible."

"NDV-01’s compelling efficacy, durability, and favorable safety profile, combined with operational ease-of-use are the cornerstone of its differentiated product profile and best-in-class potential," said Raj S. Pruthi, MD, Chief Medical Officer-Oncology of Relmada Therapeutics. "We continue to be encouraged by the high response rates and durable clinical benefit observed through 12 months, including in the BCG-unresponsive population, alongside a favorable safety profile with no ≥ Grade 3 treatment-related adverse events and no treatment-related discontinuations. Our clinical program builds on the urologic oncology community’s comfort with conventional Gem/Doce’s efficacy and safety profile with a sustained release product that could provide physicians and patients with a streamlined, less than 5-minute in-office procedure. These results reinforce our confidence as we advance NDV-01 into the Phase 3 RESCUE registrational program in mid-2026."

Highlights of the 12-month follow-up data from the ongoing Phase 2 study of NDV-01:

In the 12-month follow-up of the Phase 2a study (March 9, 2026 Company press release) treatment with NDV-01 produced:

Durable 76% complete response (CR) rate at 12 months with 95% CR rate at any time in high-risk NMIBC
BCG-unresponsive patients achieved an 80% CR rate at 12 months and 94% CR rate at any time
No patient had progression to muscle-invasive disease, and no patient underwent a radical cystectomy
Favorable overall tolerability – no ≥ Grade 3 treatment-related adverse events and no treatment-related discontinuations or dose interruptions.
Phase 3 RESCUE Registrational Pathways:

As previously disclosed in the Company’s January 12, 2026 regulatory update, Relmada has received written feedback from the U.S. Food and Drug Administration (FDA) confirming alignment on two registrational development pathways for NDV-01, including study design, patient populations and primary endpoints.

Registrational Pathway 1 – An open label randomized controlled trial in intermediate-risk NMIBC of adjuvant therapy following TURBT (NDV-01 vs. observation). There are no approved treatments for adjuvant intermediate risk NMIBC, which we estimate affect ~75,000 patients/year in the U.S. The primary endpoint of the study is disease free survival (DFS).

Registration Pathway 2 – A single-arm trial in second line (2L) BCG-unresponsive NMIBC with carcinoma in situ (CIS) patients who are currently refractory to approved or developmental therapies. Patients with BCG-unresponsive NMIBC with CIS who fail first line (1L) therapies, which we estimate to affect ~5,000 patients/year in the U.S., have few, if any, effective treatment alternatives to radical cystectomy. The primary endpoint of the study is complete response (CR) rate at any time.

Expected Upcoming Relmada Milestones:

NDV-01 United States IND clearance – Mid-2026
NDV-01 Phase 3 RESCUE Program initiation – Mid-2026
Sepranolone Phase 2 initiation in Prader-Willi syndrome – Mid-2026
Initial 3-month NDV-01 data from Phase 3 2L BCG-unresponsive study expected by YE 2026
Financial Results

Fourth Quarter 2025 Financial Results

Research and development expense for the three months ended December 31, 2025, totaled $8.1 million, compared to $11.0 million for the three months ended December 31, 2024, a decrease of $2.9 million. The decrease was primarily driven by a decrease in study costs associated with the completion of two Phase 3 trials for REL-1017, partially offset by increased costs related to the start-up the Phase 3 NDV-01 trials and Phase 2b sepranolone study and additional R&D personnel.
General and administrative expense for the three months ended December 31, 2025, totaled $12.3 million compared to $8.1 million for the three months ended December 31, 2024, an increase of approximately $4.2 million. The increase was primarily driven by an increase in compensation costs partially offset by a decrease in stock based compensation costs.
Net cash used in operating activities for the three months ended December 31, 2025, totaled $14.6 million compared to $8.8 million for the three months ended December 31, 2024.
The net loss for the three months ended December 31, 2025, was $19.9 million, or $0.27 per basic and diluted share, compared with a net loss of $18.6 million, or $0.62 per basic and diluted share, for the three months ended December 31, 2024.
Twelve Month Ended December 31, 2025 Financial Results

Research and development (R&D) expense for the 12 months ended December 31, 2025, totaled $26.9 million, compared to $46.2 million for the 12 months ended December 31, 2024, a decrease of $19.3 million. The decrease was primarily driven by a decrease in study costs associated with completion and conclusion of two Phase 3 trials for REL-1017, partially offset by increased costs related to the acquisition of NDV-01 and sepranolone, as well as the start-up of the Phase 3 NDV-01 trials and Phase 2b sepranolone study.
General and administrative (G&A) expense for the 12 months ended December 31, 2025, totaled $32.2 million compared to $37.7 million for the 12 months ended December 31, 2024, a decrease of approximately $5.5 million. The decrease was primarily driven by a decrease in stock-based compensation expense and lower professional fees, partially offset by an increase in personnel-related costs.
Net cash used in operating activities for the 12 months ended December 31, 2025, totaled $45.8 million compared to $51.8 million for the 12 months ended December 31, 2024.
The net loss for the 12 months ended December 31, 2025, was $57.4 million, or $1.45 per basic and diluted share, compared with a net loss of $80.0 million, or $2.65 per basic and diluted share, for the 12 months ended December 31, 2024.
The Company’s cash balance of $93.0 million in cash, cash equivalents, and short-term investments, includes net proceeds of approximately $94 million from an underwritten stock offering announced November 5, 2025. This compares to cash, cash equivalents, and short-term investments of approximately $44.9 million at December 31, 2024.
On March 9, 2026, the Company announced a private financing with gross proceeds of $160 million. This financing, along with the cash, cash equivalents, and short-term investments as of December 31, 2025, is expected to provide sufficient resources to fund Company operations through 2029, including completion of the Phase 3 NDV-01 RESCUE program.
The Company had 104,890,223 shares outstanding, as of March 16, 2026
Conference Call and Webcast Information:
Relmada will host a conference call and webcast today at 4:30 PM ET to discuss recent business progress and financial results.

Conference Call and Webcast Information:

Date: Thursday, March 19, 2026 at 4:30 PM ET
Participant Dial-in (US): 1-877-407-0792
Participant Dial-in (International): 1-201-689-8263
Webcast Access: Click Here
A replay of the webcast will be available in the Investors section of the Relmada website at View Source

About NDV-01

NDV-01 is a sustained-release, intravesical formulation of gemcitabine and docetaxel (Gem/Doce), in development for the treatment of non-muscle invasive bladder cancer. It is designed to enable Gem/Doce bladder retention and gradual drug release over 10 days. The formulation creates a soft matrix that enhances local exposure while minimizing systemic toxicity. The NDV-01 formulation is ready to use, convenient to administer in-office in less than 5 minutes and does not require anesthesia or specialized equipment. It is protected by patents through 2038.

About the Phase 2 Study

The Phase 2 study (NCT06663137) is an open-label, single-arm, single-center study evaluating the safety and efficacy of NDV-01 in patients with high-grade non-muscle invasive bladder cancer (HG-NMIBC). Patients are treated with NDV-01 in a biweekly induction phase, followed by monthly maintenance for up to one year, with regular assessments via cystoscopy, cytology, and biopsy, as indicated. The primary efficacy endpoints are safety and complete response rate (CRR) at 12 months, and secondary efficacy endpoints are duration of response (DOR) and event free survival (EFS).

About NMIBC

NMIBC represents 75-80% of all bladder cancer cases and is associated with high recurrence (50 – 80% over 5 years). With over 744,000 prevalent cases in the U.S. and limited treatment options, the market opportunity is significant. High-grade BCG-unresponsive disease represents one of the most difficult-to-treat NMIBC subtypes, with limited bladder-sparing options. Intermediate-risk NMIBC in the adjuvant setting has no currently approved therapies. NDV-01 has the potential to serve as a frontline or salvage therapy and could be applicable across multiple NMIBC subtypes.

About Sepranolone and GABA Modulation

Sepranolone, a synthetic isoallopregnanolone, selectively modulates GABAA receptors by antagonizing allopregnanolone (ALLO), without disrupting GABA signaling. It targets disorders linked to excess GABAergic activity such as Prader-Willi syndrome, Tourette syndrome, and Obsessive-Compulsive Disorder (OCD). More than 335 patients have been treated with sepranolone in clinical trials to date, with an excellent safety profile.

About Prader-Willi Syndrome (PWS)

PWS is a rare genetic disorder caused by chromosomal deletions on chromosome 15, leading to neurodevelopmental and behavioral complications. Global prevalence is estimated to be 350,000-400,000 patients. Current treatments address symptoms but do not modify the underlying neurobehavioral pathology.

(Press release, Relmada Therapeutics, MAR 19, 2026, View Source [SID1234663754])

On March 19, 2026 Oncolytics Biotech Inc. (Nasdaq: ONCY) ("Oncolytics" or the "Company"), a clinical-stage immunotherapy company developing pelareorep, reported two abstracts across distinct tumor types were accepted for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting 2026, at the San Diego Convention Center from April 17-22, 2026. Pelareorep is an investigational, systemically delivered immunotherapy that has been shown to activate innate immune-sensing pathways via double‑stranded RNA signaling, driving interferon production, dendritic cell activation, and cytotoxic T‑cell priming.

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"These new translational findings strengthen our understanding of pelareorep as a systemic immune‑priming agent that reshapes the tumor microenvironment," said Jared Kelly, Chief Executive Officer of Oncolytics. "The coordinated immune activation and tertiary lymphoid structure formation we are observing support pelareorep as a foundational backbone for combination therapy across multiple tumor types, including RAS-driven cancers. RAS mutations are especially prevalent in deadly cancers, including roughly 95% of pancreatic cancers and 45% of colorectal cancers. As we advance registrational programs in gastrointestinal cancers, we believe pelareorep will enhance immunotherapy activity and potentially help patients who did not respond initially."

Biomarker data from cohort 1 of the Phase 1/2 GOBLET trial in advanced pancreatic cancer suggest pelareorep plus atezolizumab and gemcitabine/nab-paclitaxel can shift tumors toward a more immune-active state. Patients with an immune activation signature after four weeks were more likely to achieve durable clinical benefit, supporting a potential biomarker. Pancreatic cancer continues to pose a significant unmet clinical need, with approximately 510,000 new cases reported globally each year.1

Additionally, new first-of-its-kind data from AWARE-1 show pelareorep can drive coordinated anti-tumor immune responses, including tertiary lymphoid structure ("TLS") formation, helping make immunologically cold tumors more susceptible to immunotherapy. This could include patients who have failed checkpoint inhibitors, representing a significant portion of the patient population and a meaningful unmet need in oncology.

Pelareorep has also been shown to stimulate RAS-specific T-cell clones in gastrointestinal cancers, which may enhance its ability to target RAS-mutated tumor cells. The Company will present more data on this topic at an upcoming scientific meeting.

Together, the data support pelareorep as a novel immune‑priming strategy capable of enhancing the activity of multiple therapeutic classes, including checkpoint inhibitors, targeted therapies, chemotherapy, and emerging immuno‑oncology modalities.

Abstract: Pelareorep combined with atezolizumab and chemotherapy shows immune conversion activity in advanced pancreatic cancer: Biomarker results of cohort 1 of the GOBLET trial

New biomarker data from cohort 1 of the Phase 1/2 GOBLET study demonstrated that pelareorep in combination with atezolizumab, gemcitabine, and nab-paclitaxel led to meaningful immune activation in patients with metastatic pancreatic ductal adenocarcinoma ("mPDAC").

An exploratory analysis integrating routine serum markers, a 172-protein circulating panel, selected T-cell receptor sequencing, and clinical outcomes found early on-treatment changes showed increases in adaptive immune and cytotoxicity-associated proteins, including interferon signaling and CD8+/NK-related markers, alongside decreases in tumor/stroma-associated proteins. A 14-protein signature linked to oncolytic activity stratified patients at Week 4 into ‘hot’ vs. ‘cold’ immune phenotypes relative to baseline. Patients demonstrating immune activation signatures experienced longer median progression-free survival (7.5 months) compared with patients who did not exhibit similar immune responses (5.6 months).

As previously reported, the combination of pelareorep, atezolizumab, gemcitabine, and nab-paclitaxel demonstrated a 62% objective response rate in first-line mPDAC patients, more than double the response rates historically observed with chemotherapy alone.

Abstract: Imaging mass cytometry of pelareorep treated early breast cancer samples reveals developing tertiary lymphoid structures

AWARE-1 is a window-of-opportunity study evaluating the effects of pelareorep in early-stage breast cancer. In biopsies from Cohort 2 (n=8), cellular neighborhood analysis identified immune-cell clusters, including cytotoxic T cells, dendritic cells, T helper cells, activated T helper cells, B cells, differentiated B cells, and M2 macrophages. Interactions between these cells developed into TLS in select patients. TLS function as localized immune hubs that facilitate antigen presentation, T‑cell activation, and sustained anti‑tumor immune responses. Published studies link TLS formation with improved responses to immunotherapy.

These findings support pelareorep’s potential to expand cytotoxic T cells, activate dendritic cells, and promote TLS formation, helping to convert immunologically ‘cold’ tumors into immune-responsive ‘hot’ tumors.

About GOBLET
The GOBLET (Gastrointestinal tumOrs exploring the treatment comBinations with the oncolytic reovirus peLarEorep and anTi-PD-L1) study is a phase 1/2 multiple indication study in advanced or metastatic gastrointestinal tumors. The study is being conducted at 17 centers in Germany and is being managed by AIO-Studien-gGmbH. The co-primary endpoints of the study are objective response rate and/or disease control rate and safety. Key secondary and exploratory endpoints include additional efficacy assessments and evaluation of potential biomarkers. The study comprises five treatment groups:
1.Pelareorep in combination with atezolizumab, gemcitabine, and nab-paclitaxel in 1st line advanced/metastatic pancreatic cancer patients;
2.Pelareorep in combination with atezolizumab in 1st line MSI (microsatellite instability)-high metastatic colorectal cancer patients;

3.Pelareorep in combination with atezolizumab and TAS-102 in 3rd line metastatic colorectal cancer patients
4.Pelareorep in combination with atezolizumab in 2nd line advanced and unresectable anal cancer patients; and
5.Pelareorep in combination with mFOLFIRINOX with and without atezolizumab in newly diagnosed metastatic PDAC patients.
About AIO
AIO-Studien-gGmbH (AIO) emerged from the study center of the medical oncology working group within the German Cancer Society (DKG). AIO operates with a non-profit purpose of promoting science and research with a focus on medical oncology. Since its foundation, AIO has become a successful sponsor and study management company and has established itself both nationally and internationally.

About AWARE-1
AWARE-1 was an open-label window-of-opportunity study in early-stage breast cancer. The study combined pelareorep, without or with atezolizumab, and standard of care therapy according to breast cancer subtype. Tumor tissue was collected from patients as part of their initial breast cancer diagnosis, again on day three following initial treatment, and finally at three weeks following treatment, on the day their tumor is surgically resected. Key objectives of the study were to confirm that pelareorep is acting as a novel immunotherapy, to evaluate potential synergy between pelareorep and checkpoint blockade, and to collect biomarker data. The primary endpoint of the translational study was overall CelTIL score (a measurement of cellularity and tumor-infiltrating lymphocytes that is associated with favorable clinical outcomes). Secondary endpoints for the study included safety and tumor and blood-based biomarkers. The combination of pelareorep, letrozole, and atezolizumab resulted in 60% of patients experiencing 30% or greater increases in their CelTIL score.

(Press release, Oncolytics Biotech, MAR 19, 2026, View Source [SID1234663752])

On March 19, 2026 NuCana plc (NASDAQ: NCNA) ("NuCana" or the "Company") reported financial results for the fourth quarter and year ended December 31, 2025 and provided an update on its clinical development program with its two lead anti-cancer medicines.

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"We are excited to enter 2026 with significant momentum as we continue to advance the development of our ProTide pipeline with the aim to deliver significantly improved treatment outcomes for patients with cancer," said Hugh S. Griffith, NuCana’s Chief Executive Officer. "In late 2025, we presented compelling data from our Phase 2 NuTide:701 study at the annual European Society for Medical Oncology Immuno-Oncology Congress, evaluating NUC-7738 in combination with pembrolizumab in patients with PD-1 inhibitor-resistant metastatic melanoma. These data demonstrated a favorable safety profile and evidence of clinical activity, including two partial responses, one of which was confirmed, and multiple cases of stable disease, including one patient whose disease converted to a complete metabolic response with no detectable active disease."

Mr. Griffith continued, "We expect to complete enrollment in the Phase 2 NuTide:701 expansion study in the first half of 2026, with final data expected later this year. We also plan to obtain regulatory guidance from the U.S. Food and Drug Administration regarding a potential registrational pathway for NUC-7738 in melanoma. In parallel, we are evaluating additional indications and combination strategies to further explore the therapeutic potential of NUC-7738."

Mr. Griffith concluded, "Earlier this year we appointed Theresa Bruce as our Chief Operating Officer. Ms. Bruce brings over 25 years of oncology research and development experience, and her operational leadership will support the continued advancement of our pipeline. Based on our current operating plan, we expect our existing cash resources to fund operations into 2029, positioning us to execute on our key anticipated clinical and regulatory objectives."

2026 Anticipated Milestones

NUC-7738
Complete patient recruitment in the Phase 2 expansion study (NuTide:701) evaluating NUC-7738 in combination with pembrolizumab in patients with PD-1 resistant melanoma;
Announce final data from the Phase 2 expansion study (NuTide:701) of NUC-7738 in combination with pembrolizumab in patients with PD-1 resistant melanoma;
Obtain regulatory guidance from the U.S. Food and Drug Administration regarding a potential registrational strategy for NUC-7738 in melanoma; and
Advance evaluation of additional indications and combination strategies.
NUC-3373
Evaluate optimal combinations and indications to inform potential future clinical studies of NUC-3373.

Fourth Quarter and Year-End 2025 Financial Highlights and Cash Position

As at December 31, 2025, NuCana had cash and cash equivalents of £24.3 million compared to £25.2 million at September 30, 2025 and £6.7 million at December 31, 2024.

In May 2025, NuCana completed a financing, raising £9.6 million in gross proceeds, £5.2 million upfront and £4.4 million from the exercise of warrants, before expenses and commissions.

Subsequently, in July 2025, NuCana raised, through the at-the-market program, £19.0 million in gross proceeds before expenses and commissions. On July 21, 2025, having raised the full amount of capital required, NuCana announced it had successfully canceled all remaining Series A Warrants issued in the May 2025 financing, in exchange for payments totaling $3.6 million. This initiative fully eliminated all overhanging rights from the May 2025 financing.

NuCana anticipates its cash and cash equivalents at December 31, 2025 will be sufficient to fund its planned operations into 2029.

NuCana reported a net loss of £2.5 million for the quarter ended December 31, 2025, as compared to a net loss of £0.7 million for the quarter ended December 31, 2024. Basic and diluted loss per ordinary share was £0.00 for the quarter ended December 31, 2025, as compared to a loss per ordinary share of £0.01 for the comparable quarter ended December 31, 2024.

NuCana reported a net loss of £29.4 million for the year ended December 31, 2025, as compared to a net loss of £19.0 million for the year ended December 31, 2024. The net loss for the year ended December 31, 2025 included the following non-cash or non-recurring items:

Finance expense of £12.6 million (2024: £nil) relating to the non-cash loss on fair value revaluation of the warrants issued in the May 2025 financing;
Professional fees of £1.4 million (2024: £nil) related to the issue of warrants; and
Share-based payment expenses of £10.0 million (2024: £1.6 million); partly offset by
Total other income of £2.7 million (2024: £nil).
Basic and diluted loss per ordinary share was £0.00 for the year ended December 31, 2025, as compared to a loss per ordinary share of £0.26 for the year ended December 31, 2024.

(Press release, Nucana, MAR 19, 2026, View Source [SID1234663751])

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])