On March 23, 2026 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it obtained regulatory approval from the Ministry of Health, Labour and Welfare (MHLW) for the additional indication of the combination therapy of anti-cancer agent / anti-CD20/CD3 humanized bispecific monoclonal antibody Lunsumio subcutaneous injection 5mg and 45mg [generic name: mosunetuzumab (genetical recombination)] (hereafter, Lunsumio) and anti-cancer agent/antimicrotubule-binding anti-CD79b monoclonal antibody Polivy intravenous infusion 30 mg and 140 mg [generic name: polatuzumab vedotin (genetical recombination)] (hereafter, Polivy) for the treatment of relapsed or refractory large B-cell lymphoma. This marks the first approval in the world for the combination therapy of Lunsumio and Polivy for this indication.

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"For patients with relapsed or refractory large B-cell lymphoma, treatment options remain limited and unmet medical needs persist. The Lunsumio and Polivy combination therapy achieved responses in approximately 70% of patients and showed a 59% reduction in the risk of disease progression or death compared to the chemotherapy control arm. We are committed to delivering this treatment to patients as quickly as possible by providing timely information to healthcare professionals and promoting appropriate use," said Dr. Osamu Okuda, Chugai’s President and CEO.

This approval is based on the results of the global, multi-center, randomized Phase III SUNMO study evaluating the efficacy and safety of the combination therapy compared to the regimen of rituximab, gemcitabine, and oxaliplatin (R-GemOx; not approved in Japan) in patients with relapsed or refractory large B-cell lymphoma who are not eligible for autologous hematopoietic stem cell transplantation.

In the interim analysis of the study, the objective response rate (ORR), a primary endpoint assessed by an independent review committee, was 69.7% (95% CI: 60.7-77.8) in the Lunsumio and Polivy combination therapy group and 44.1% (95% CI: 31.2-57.6) in the R-GemOx group, with a between-group difference of 25.7% (97.5% CI: 7.4-43.9). At the time of the primary analysis, the progression-free survival (PFS), also a primary endpoint assessed by an independent review committee, was 11.5 months (95% CI: 5.6-18) in the Lunsumio and Polivy combination therapy group and 3.8 months (95% CI: 2.9-4.1) in the R-GemOx group, demonstrating a 59% reduction in the risk of disease progression or death.

The safety profile of the combination therapy of Lunsumio and Polivy was consistent with the known profiles of each agent in their respective individual studies. Adverse events were observed in 131 of 135 patients (97.0%) in the Lunsumio and Polivy combination therapy group and in 61 of 64 patients (95.3%) in the R-GemOx group. The main adverse events in the Lunsumio and Polivy combination therapy group included injection site reactions in 71 patients (52.6%), neutropenia in 62 patients (45.9%), anemia in 41 patients (30.4%), and cytokine release syndrome in 35 patients (25.9%), among others.1

Approval Information (Lunsumio) *Relevant sections only, with modifications underlined

Product name: "LUNSUMIO subcutaneous injection 5mg" and "LUNSUMIO subcutaneous injection 45mg"

Generic name: mosunetuzumab (genetical recombination)

Indications:
The following relapsed or refractory large B-cell lymphomas:

Diffuse large B-cell lymphoma
High-grade B-cell lymphoma

Relapsed or refractory follicular lymphoma

Dosage and administration:
〈Relapsed or refractory large B-cell lymphoma (diffuse large B-cell lymphoma, high-grade B-cell lymphoma) and relapsed or refractory follicular lymphoma (Grade 3B)〉
In combination with polatuzumab vedotin (genetical recombination), mosunetuzumab (genetical recombination) is administered subcutaneously to adults in 21-day cycles.
In the first cycle, 5 mg is administered on Day 1, followed by 45 mg on Days 8 and 15, and from the second cycle onward, 45 mg is administered on Day 1 for up to 8 cycles.

Approval Information (Polivy) *Relevant sections only, with modifications underlined

Product name: "Polivy intravenous infusion 30 mg" and "Polivy intravenous infusion 140 mg"

Generic name: polatuzumab vedotin (genetical recombination)

Indications:
The following large B-cell lymphomas:

Diffuse large B-cell lymphoma
High-grade B-cell lymphoma

Relapsed or refractory follicular lymphoma

Precautions concerning indications:

This drug should be administered to patients diagnosed with Grade 3B by a pathologist with sufficient experience.

Dosage and administration:
In combination with other anti-cancer agents, polatuzumab vedotin (genetical recombination) is usually administered to adults as an intravenous infusion at a dose of 1.8 mg/kg (body weight) once every three weeks for a total of six doses. The initial dose is infused over 90 minutes, and if tolerated well, the infusion time for subsequent doses may be shortened to 30 minutes. The dose may be reduced as appropriate depending on the patient’s condition.

[Reference]

Roche’s Lunsumio and Polivy combination significantly prolongs remission for people with relapsed or refractory large B-cell lymphoma (Press release from Roche issued on June 20, 2025)
View Source

About the SUNMO study

The SUNMO [NCT05171647] study is a multinational, multicenter, randomized Phase III trial that targets patients with relapsed or refractory large B-cell lymphoma who are not eligible for autologous hematopoietic stem cell transplantation, and evaluates the combination therapy of subcutaneously administered Lunsumio (mosunetuzumab) and intravenously administered Polivy (polatuzumab vedotin) in comparison with the R-GemOx regimen [Rituxan (rituximab), gemcitabine, and oxaliplatin]. The primary endpoints are progression-free survival and objective response rate, and the secondary endpoints include overall survival, duration of objective response, complete response rate, duration of complete response, safety and tolerability, and patient-reported outcomes (PROs: Patient Reported Outcomes).

About Lunsumio (mosunetuzumab)

Lunsumio is a T-cell-engaging bispecific antibody designed to target CD3 on T cells and CD20 on B cells. Lunsumio is expected to activate immunity mediated by cytotoxic T cells and exert antitumor effects against tumor cells expressing CD20. Lunsumio has been approved in 65 countries worldwide. Clinical studies are currently underway in follicular lymphoma (second line and untreated settings). In December 2025, in addition to the intravenous formulation, Lunsumio obtained manufacturing and marketing approval for a new subcutaneous formulation.

About Polivy (polatuzumab vedotin)

Polivy is a first-in-class anti-CD79b antibody-drug conjugate (ADC). The CD79b protein is expressed in the majority of B cells, an immune cell impacted in some types of non-Hodgkin lymphoma (NHL), making it a promising target for the development of new therapies. Polivy binds to cancer cells such as those expressing CD79b and destroys these B cells through the delivery of an anti-cancer agent, which is thought to minimize the effects on normal cells. Polivy is being developed by Roche and is currently being investigated for the treatment of several types of NHL.

About Large B-cell lymphoma (LBCL)

LBCL consists primarily of diffuse large B-cell lymphoma (DLBCL), which is the most common subtype of non-Hodgkin lymphoma (NHL) affecting B-cell lymphocytes, a type of white blood cell. DLBCL is the most common form of aggressive NHL and accounts for approximately 80%2 of LBCL cases. LBCL also includes high-grade B-cell lymphoma (HGBL), which is considered to be even more aggressive and to have a poorer prognosis than DLBCL. Although patients generally respond to frontline therapy, up to 40%3 experience relapse or become refractory, and in such cases, treatment options for salvage therapy are limited.

Trademarks used or mentioned in this release are protected by law.

(Press release, Chugai, MAR 23, 2026, View Source [SID1234663822])

On March 23, 2026 GlycoNex, Inc. (4168, hereinafter referred to as GNX), a clinical-stage biotechnology company focused on the development of glycan-directed cancer immunotherapies, reported that it has entered into a collaboration agreement with Nippon Kayaku Co., Ltd. ("Nippon Kayaku") to advance GNX201-ADC, a next-generation antibody-drug conjugate (ADC) candidate for the treatment of solid tumors. Under the agreement, the two companies will jointly support preclinical development activities for GNX201-ADC, leveraging their respective expertise in antibody engineering and oncology drug development.

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The collaboration will center on research and development activities for GNX201-ADC including optimization of the ADC construct and generation of preclinical data to support regulatory submissions. GNX201-ADC is designed as a protease-activated ADC that remains stable in circulation and is selectively activated in the tumor microenvironment, enabling targeted delivery of its cytotoxic payload while minimizing effects on normal tissues. This differentiated pro-antibody activation strategy is intended to improve therapeutic index and reduce on-target toxicity compared with conventional ADC approaches. Initial development efforts will focus on solid tumors characterized by high levels of glycan antigens.

"GlycoNex has long been dedicated to advancing anti-glycan antibodies and tumor glycobiology, and ADCs represent a natural extension of that scientific focus," said Dr. Mei-Chun Yang, CEO of GlycoNex. "We are honored to collaborate with Nippon Kayaku to combine our complementary R&D strengths, clinical development capabilities, and regulatory expertise to accelerate GNX201-ADC and advance the program toward clinical studies."

GNX201-ADC leverages GlycoNex’s "Antibody Lock" technology, incorporating a protease-activated pro-ADC that is "turned on" when the masking peptide is removed in the tumor microenvironment and turned off with the masked domain when in circulation. This selective activation allows GNX201-ADC to target tumors without harming healthy tissue. The design aims to reduce the risk of on-target toxicity and associated adverse effects observed with conventional ADC approaches.

"We are pleased to partner with GlycoNex on GNX201-ADC, a program that brings a differentiated pro-antibody activation concept into an ADC modality," said Akira Masuda, General Manager of Pharmaceutical Research Laboratories of Nippon Kayaku. "By combining GlycoNex’s expertise in tumor-associated glycan targeting with Nippon Kayaku’s experience in oncology-related pharmaceuticals, we aim to advance preclinical development and generate the data needed to support future clinical evaluation and treatment options for patients."

This collaboration further strengthens GlycoNex’s growing ADC pipeline and reflects its broader strategy of leveraging glycan-directed antibody technologies to develop novel precision oncology therapies. For Nippon Kayaku, the partnership expands its oncology research portfolio through access to a differentiated targeting modality with potential application across multiple solid tumor indications.

(Press release, GlycoNex, MAR 23, 2026, View Source [SID1234663841])

On March 23, 2026 GSK plc (LSE/NYSE: GSK) reported that risvutatug rezetecan (or Ris-Rez, for short), a B7-H3-targeted antibody-drug conjugate (ADC), has received Orphan Drug Designation (ODD) from Japan’s Ministry of Health, Labour and Welfare for the treatment of small-cell lung cancer (SCLC). The ODD was supported by preliminary clinical data showing durable responses in patients with extensive-stage SCLC (ES-SCLC) who were treated with Ris-Rez in the phase I ARTEMIS-001 clinical trial.1 This is the sixth regulatory designation for Ris-Rez, which is being developed in a range of solid tumours, including lung, prostate and colorectal cancers.

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Lung cancer is the second most common cancer in Japan, and SCLC makes up 10-15% of cases.2,3 Of patients with SCLC, 70% have ES-SCLC, meaning the cancer has spread throughout one or both lungs and/or to other parts of the body.4 ES-SCLC is an aggressive and difficult-to-treat cancer with limited treatment options and poor long-term survival. Most patients with ES-SCLC relapse after initial treatment and the median overall survival with standard-of-care for these patients is approximately 8 months.5

About Ris-Rez
Ris-Rez is a novel investigational B7-H3-targeted antibody-drug conjugate composed of a fully human anti-B7-H3 monoclonal antibody covalently linked to a topoisomerase inhibitor payload. GSK acquired exclusive worldwide rights (excluding China’s mainland, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to progress clinical development and commercialisation of Ris-Rez. GSK’s global phase III trial (NCT07099898) for Ris-Rez in relapsed ES-SCLC began in August 2025.

Regulatory designations received for Ris-Rez to date include ODDs from the US Food and Drug Administration (FDA) in SCLC and the European Medicines Agency (EMA) in a category of cancer that includes SCLC, called pulmonary neuroendocrine carcinoma; Priority Medicines (PRIME) Designation from the EMA for relapsed or refractory ES-SCLC; and Breakthrough Therapy Designations for relapsed or refractory ES-SCLC and relapsed or refractory osteosarcoma from the US FDA.

(Press release, GlaxoSmithKline, MAR 23, 2026, View Source [SID1234663823])

On March 23, 2026 WuXi AppTec (stock code: 603259.SH / 2359.HK), a leading global pharmaceutical CRDMO (Contract Research, Development, and Manufacturing Organization), reported financial results for the full year ended December 31, 2025 ("Reporting Period"):

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Total revenue reached RMB 45.46 billion, up 15.8% YoY. Revenue from Continuing Operations reached RMB 43.42 billion, up 21.4% YoY.
Adjusted non-IFRS gross profit margin up 6.6pts YoY to 48.2%.
Adjusted non-IFRS net profit attributable to the owners of the Company up 41.3% YoY to RMB 14.96 billion; adjusted non-IFRS net profit margin up 5.9pts YoY to 32.9%; adjusted non-IFRS diluted EPS up 41.4% YoY to RMB 5.16.
Net profit attributable to the owners of the Company[1] up 102.6% YoY to RMB 19.15 billion; diluted EPS up 102.8% YoY to RMB 6.61.
With continuous capacity expansion to better meet customer demand, backlog for Continuing Operations reached RMB 58.00 billion as of year-end, up 28.8% YoY.
Adjusted operating cash flow[2] up 39.1% YoY to RMB 16.67 billion, driven by sustained business growth and enhanced operational efficiency and financial management.
Sustained and steady business growth driven by our unique, fully integrated Contract Research, Development and Manufacturing Organization (CRDMO) platform. Guided by "follow the molecule" and "win the molecule" strategies, WuXi Chemistry’s small molecule CRDMO pipeline continues to efficiently convert and capture high-quality molecules, delivering sustained business growth. In 2025, we added 839 new molecules to the small molecule Development and Manufacturing (D&M) pipeline, bringing the total to 3,452 molecules as of year-end. Notably, commercial and phase III projects increased by 22 during the year.
Accelerating global expansion, capacity construction and capability development. In 2025, Changzhou, Taixing and Jinshan API sites successfully passed FDA on-site inspections with no single observation. By year-end, our total reactor volume of small molecule APIs has reached over 4,000kL, while total reactor volume of Solid Phase Peptide Synthesizers has reached over 100,000L.
Driving sustainability, embracing initiatives, with sustained recognition by leading global ratings. In 2025, we achieved our first MSCI "AAA" and CDP Climate Change "A" ratings, maintained CDP Water Security "A" and EcoVadis "Gold" ratings. Meanwhile, our near-term GHG emissions reduction targets have been successfully validated by SBTi. As a committed UNGC participant and PSCI Supplier Partner, we actively embrace global initiatives and are dedicated to integrating sustainability into our business strategy and operations.
Unwavering commitment to safeguarding customers’ IP and adhering to the highest standards of quality & compliance. In 2025, the Company completed 741 quality audits and inspections conducted by global customers, regulatory authorities and independent third parties, as well as 60 information security audits by global customers, all with no critical findings. Currently, 20 of our main sites are ISO/IEC 27001 certified, covering all main sites in China.
[1] Net profit attributable to the owners of the Company is prepared in accordance with China Accounting Standards for Business Enterprises (CAS).

[2] Adjusted operating cash flow and adjusted free cash flow exclude income tax payments related to significant transactions (i.e., partial equity sales of WuXi XDC and the sale of the China-based clinical research service businesses) disclosed in the Company’s announcements.

2026 Outlook

With customers’ ongoing demand for enabling services, our CRDMO business model and management execution, the Company is confident to sustain rapid business growth. We expect total revenue to reach RMB 51.3-53.0 billion in 2026, with Continuing Operations revenue growing 18-22% YoY.

By continuously driving quality growth, realizing scale efficiency and enhancing operational excellence, while proactively managing new capacity ramp-up and FX challenges, we are confident in maintaining a stable and resilient adjusted non-IFRS NPM in 2026.

2026 capex is expected to reach RMB 6.5-7.5 billion. Along with business growth and efficiency improvements, adjusted free cash flow[2] is expected to reach RMB 10.5-11.5 billion.

While accelerating global capacity and capability enhancement, we remain committed to rewarding shareholders. We propose a cash dividend distribution plan totaling a record RMB 5.7 billion in 2026.

To invest in talents for long-term shared growth, we propose to launch the 2026 H-share Incentive Trust Plan, which will grant no more than HKD1.5 billion H-shares upon achieving RMB 51.3 billion revenue in 2026, and an additional HKD1.0 billion H-shares upon reaching RMB 53.0 billion and above. Underlying H-shares will be purchased in the open market at prevailing market prices, with no dilution to existing shareholders.

Management Comment

Dr. Ge Li, Chairman and CEO of WuXi AppTec, said, "In 2025, WuXi AppTec achieved record performance with strong growth in revenue, profit and cash flow. Meanwhile, our backlog for Continuing Operations reached RMB 58.0 billion – a 28.8% YoY increase, demonstrating the strength of our unique CRDMO business model and the exceptional execution of our global team."

"Entering 2026, with a sharpened focus on our core CRDMO strategy, we are accelerating the growth of our global capabilities and capacity, further improving production and operational efficiency, and delivering greater value for customers and shareholders. For 2026, we expect total revenue to reach RMB 51.3-53.0 billion, reflecting rapid growth of 18-22% YoY in Continuing Operations revenue, and adjusted free cash flow of RMB 10.5-11.5 billion."

"For 25 years, WuXi AppTec has been dedicated to lowering barriers to R&D and advancing healthcare innovation worldwide. Staying true to our founding aspiration, we will remain committed to ‘doing the right thing and doing it right’, enabling our partners to deliver life-saving therapies to patients in need, and advancing our vision that ‘every drug can be made and every disease can be treated’."

Business Performance by Segment

WuXi Chemistry: CRDMO Business Model Drives Continuous Growth; 2025 Revenue Up 25.5% YoY, with TIDES Revenue Up 96.0% YoY
WuXi Chemistry’s 2025 revenue reached RMB 36.47 billion, up 25.5% YoY. With continued optimization of production processes and improvements in capacity efficiency driven by the growth of late-stage clinical and commercial projects, 2025 adjusted non-IFRS gross profit margin of WuXi Chemistry steadily improved 5.9pts YoY to 52.3%.
Small molecule drug discovery service ("R") continues to generate downstream opportunities. In 2025, we successfully synthesized and delivered more than 420,000 new compounds to global customers. Meanwhile, 310 molecules were converted from R to D phase. Guided by our "follow-the-customer" and "follow-the-molecule" strategies, we have built trusted partnerships that underpin the sustainable growth of our CRDMO business.
Small molecule D&M service remains strong.
i. The small molecule CDMO pipeline continued to expand. In 2025, small molecule D&M revenue rose 11.4% YoY to RMB 19.92 billion. We added 839 new molecules to the small molecule D&M pipeline. As of year-end, our pipeline reached 3,452 molecules, including 83 commercial projects, 91 in phase III, 377 in phase II and 2,901 in phase I and pre-clinical stages. Notably, commercial and phase III projects increased by 22 in 2025.
ii. We continued to build small molecule capacity. In 2025, our Changzhou, Taixing and Jinshan API sites successfully passed FDA on-site inspections with no single observation. By year-end, total reactor volume of small molecule APIs reached over 4,000kL.
TIDES business (oligo and peptides) sustains rapid growth.
i. With the sequential ramp-up of new capacity released in 2024, 2025 TIDES revenue grew 96.0% YoY to RMB 11.37 billion. As of year-end, TIDES backlog increased 20.2% YoY.
ii. TIDES D&M customers grew 25% YoY, while the number molecules grew 45% YoY.
iii. In September 2025, we completed Taixing peptide capacity construction ahead of schedule. The Company’s total reactor volume of Solid Phase Peptide Synthesizers has reached over 100,000L.
WuXi Testing[3]: Strengthening Differentiated Capabilities and Operational Management; 2025 Revenue Back to Positive YoY Growth of 4.7%, Drug Safety Evaluation Services Maintained Leading Position
In 2025, WuXi Testing revenue resumed positive growth, up 4.7% YoY to RMB 4.04 billion. Of which, drug safety evaluation services revenue grew 4.6% YoY, maintaining an industry-leading position in the Asia-Pacific region.
Due to market impact, WuXi Testing’s 2025 adjusted non-IFRS gross profit margin declined YoY as pricing gradually reflected in revenue through backlog conversion, yet continued to improve sequentially each quarter driven by differentiated capabilities and enhanced operational management.
The Company is committed to actively enabling customers in global licensing deals. New modality business continued to develop, with revenue contribution increasing to more than 30% in 2025, while the Company maintained its leading position in nucleic acids, conjugates, multispecific antibodies and peptides, etc.
The Company continued to advance automation. DMPK successfully launched its proprietary all-in-one compound identification software, enhancing efficiency in spectral interpretation and metabolite identification for nucleic acids and peptides by 83%.
The facilities in Suzhou and Shanghai successfully passed multiple inspections by FDA, OECD, NMPA and PMDA.
[3] As disclosed in the 2025 Annual Report, WuXi Testing refers to Continuing Operations only (not including clinical research service businesses); historical data has been adjusted accordingly.

WuXi Biology: Continues to Follow the Science & Generate Downstream Opportunities; 2025 Revenue Back to Positive YoY growth of 5.2%, In Vivo & In Vitro Synergies and New Modalities Drove Growth
WuXi Biology follows the science and strategically builds differentiated capabilities of drug discovery in emerging areas. It actively expands global business and efficiently generates downstream opportunities for the CRDMO model by continuously contributing more than 20% of the Company’s new customers.
We efficiently enable our global customers through integrated in vitro & in vivo drug discovery capabilities, cross-regional collaboration and end-to-end solutions in emerging areas. 2025 WuXi Biology revenue resumed positive growth, up 5.2% YoY to RMB 2.68 billion.
Due to market pricing impact, 2025 adjusted non-IFRS gross profit margin of WuXi Biology was down 1.9pts to 36.9%. WuXi Biology closely follows market dynamics and maintains flexible pricing strategy, maximizing its value in generating downstream opportunities.
We achieved rapid revenue growth driven by accelerated progress in integrated in vitro screening and enhanced in vivo pharmacology capabilities. Non-oncology business maintained a competitive edge, serving as a key growth contributor.
New modality business continues to drive growth, with revenue contribution increasing to more than 30% in 2025, supported by rapid new customer expansion in nucleic acids, antibody conjugates and peptides, etc.
This release provides a summary of the results and does not intend to provide a complete statement relating to the Company, its securities, or any relevant matters herein that a recipient may need in order to evaluate the Company. For additional information, please refer to the WuXi AppTec 2025 Annual Results Presentation and 2025 Annual Report disclosed on the Company’s official website, as well as the Company’s disclosure documents and information on the Shanghai Stock Exchange, the Stock Exchange of Hong Kong Limited website. Investors are advised to exercise caution and be aware of the investment risks in trading Company shares.

Net profit attributable to the owners of the Company is prepared in accordance with China Accounting Standards for Business Enterprises (CAS), in currency of RMB. All other financial information disclosed in this press release is prepared in accordance with the International Financial Reporting Standards Accounting Standards ("IFRS"), in currency of RMB.

(Press release, WuXi AppTec, MAR 23, 2026, View Source [SID1234663843])

On March 23, 2026 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported that the 45th subject has been enrolled in its pivotal Phase 2B/3 MIRACLE trial evaluating Annamycin in combination with cytarabine (AnnAraC) for the treatment of adult subjects with relapsed or refractory acute myeloid leukemia (R/R AML). This milestone triggers the final phase of preparation for the trial’s interim 45 subject data unblinding, which remains on track for mid-2026 and represents a potentially defining inflection point for the Company.

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"We believe this upcoming data readout represents the most important milestone in our history to date," said Walter Klemp, Chairman and CEO of Moleculin. "This is a critical achievement for Moleculin and, more importantly, for patients facing relapsed or refractory AML. Reaching the 45 subject mark brings us to the threshold of our first meaningful look at MIRACLE data. Based on what we believe to be encouraging blinded results announced in February, we believe we are building real momentum toward what could be a transformative outcome. Our Phase 2 AML MB-106 clinical trial generated greater efficacy than any drug ever approved for relapsed or refractory AML, and it contributed to the more than 100 patients treated to date without any associated cardiotoxicity. What remains now is to demonstrate that a larger Phase 3 trial can produce results that will support new drug approval. The upcoming unblinding of the first 45 subjects should be a strong indicator of that potential. A true next-generation anthracycline has long been sought by medical science and would rightly be considered a gamechanger, not just for AML but for a wide range of cancers. We now appear to be on the threshold of finding out whether this can become a reality."

Moleculin Biotech continues to advance toward a defining milestone with its ongoing MIRACLE trial, a global, adaptive Phase 2B/3 study designed to evaluate AnnAraC across eight countries to date. The trial protocol provides for early unblinding of data after the first 45 subjects complete treatment, which is expected to yield an initial dataset of approximately 30 patients treated with AnnAraC (at two different dose levels) and 15 patients in the control arm receiving cytarabine plus placebo. This upcoming interim readout is anticipated to provide critical insights into efficacy, safety, and dose optimization as the study progresses toward its Phase 3 portion. Enrollment continues in parallel with the 45th subject unblinding as the Company advances to 90 total subjects thereby concluding Part A of MIRACLE, expected in Q3 2026, with the complete unblinding of Part A thereafter.

Encouraging early signals have already emerged from this difficult-to-treat patient population. In February 2026, Moleculin reported a preliminary blinded composite complete remission rate of 40% among the first 30 patients enrolled in the MIRACLE trial, consisting of 30% complete remission and 10% complete remission with partial hematologic recovery. These findings are particularly notable given that approximately 35% of subjects had previously failed venetoclax-based therapies, and many exhibited adverse genetic markers typically associated with poor outcomes. Additionally, the multinational nature of the trial underscores consistent activity across diverse clinical settings. Even with the inclusion of the control arm, these preliminary blinded aggregate outcomes compare favorably to historical remission rates associated with cytarabine alone in relapsed or refractory acute myeloid leukemia.

The MIRACLE trial’s adaptive design is intended to support a streamlined global registration pathway by integrating data from its Phase 2B portion into the planned Phase 3 portion, in alignment with regulatory guidance, including FDA Project Optimus principles. Following the interim readout, enrollment is expected to continue toward 90 patients to enable a second unblinding, with the Phase 3 portion of the study commencing once the optimum dose is determined. The program remains on track toward a potential accelerated approval pathway based solely on the Complete Remission primary endpoint.

Progress in the MIRACLE trial also reflects broader momentum across Moleculin’s oncology pipeline, which includes ongoing and planned studies targeting pancreatic cancer, brain tumors, and soft tissue sarcoma. With dosing of the 45th subject now complete, the Company is entering a catalyst-rich period leading up to the anticipated mid-2026 interim data readout, a milestone that could significantly influence the future development of Annamycin in the treatment of acute myeloid leukemia.

For additional information on the MIRACLE trial, please visit ClinicalTrials.gov and reference Identifier: NCT06788756.

(Press release, Moleculin, MAR 23, 2026, View Source [SID1234663824])