Earendil Labs and WuXi Biologics Enter Strategic Collaboration to Accelerate Development and Manufacturing of Bispecific/Multispecific Antibodies and ADCs

On March 12, 2026 Earendil Labs, a global leader in AI-driven research and development of next-generation biologics therapeutics, and WuXi Biologics (2269.HK), a global leading Contract Research, Development and Manufacturing Organization (CRDMO), reported the signing of a strategic collaboration agreement on the development and manufacturing of multiple novel bispecific and multispecific antibodies and ADC candidates in Earendil Labs’ pipeline targeting autoimmune disease, cancer, and other diseases.

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Under the agreement, WuXi Biologics will provide end-to-end biologics development and manufacturing services, including cell line development, process and bioassay development, drug product formulation development, and GMP manufacturing. The collaboration is designed to accelerate regulatory timelines, enhance CMC execution reliability, and support scalable global clinical development across Earendil Labs’ programs.

WuXi Biologics has built a strong track record in developing complex biologics, particularly its technology strengths in bispecific and multispecific antibodies and ADCs. By 2025, a total of 945 projects were advancing on the company’s integrated CRDMO platform, with bispecific and multispecific antibodies representing one of the fastest‑expanding categories. Leveraging next‑generation technology platforms such as the WuXia TrueSite targeted‑integration CHO cell line platform and the WuXiHigh high‑throughput formulation platform—together with expanded capabilities for high‑dose and subcutaneous delivery, including the Hyaluronidase Co‑Formulation platform and large‑volume device solutions—the company provides development pathways designed to increase efficiency, strengthen product consistency, and enable seamless scale‑up from early research through commercial manufacturing.

Jian Peng, PhD, CEO of Earendil Labs, said: "Partnering with WuXi Biologics enables us to advance our pipeline with greater speed, precision, and CMC excellence. By integrating Earendil Labs’ AI-driven protein‑engineering capabilities with WuXi Biologics’ deep expertise in complex biologics as well as its proven end‑to‑end development and manufacturing capabilities, we can more efficiently advance our pipeline toward clinical milestones to deliver meaningful clinical benefit and transform patient treatment."

Zhenping Zhu, MD, PhD, President & co-CEO of Earendil Labs, added: "Earendil Labs has built a robust and highly competitive pipeline within the past three years. This partnership with WuXi Biologics strengthens our ability to translate AI-driven innovation into high-quality clinical and commercial assets. WuXi Biologics’ proven expertise in complex biologics and global development makes them a strong strategic partner as we build a differentiated, multi-asset therapeutic pipeline for the treatment of various human diseases."

Chris Chen, PhD, CEO of WuXi Biologics, commented, "We are delighted to enter this strategic collaboration with Earendil Labs, a company distinguished by its strong scientific leadership and innovative approach to advancing next‑generation immunotherapies. By leveraging our proven expertise across complex biologics—including bispecific and multispecific antibodies as well as ADCs—and our industry‑leading technology platforms across drug research, development and manufacturing, we aim to accelerate the advancement of Earendil Labs’ innovative pipeline. Through this collaboration, we are poised to accelerate development, ensure robust and scalable manufacturing, and ultimately bring transformative therapies to patients worldwide."

(Press release, Earendil Labs, MAR 12, 2026, View Source [SID1234663515])

Aminex Therapeutics Receives Second FDA Orphan Drug Designation for AMXT 1501 for Malignant Glioma Including DIPG – a Highly Aggressive Childhood Brain Cancer

On March 12, 2026 Aminex Therapeutics, Inc., a clinical-stage biotechnology company focused on developing a novel metabolic-targeted therapy to treat adult and pediatric cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AMXT 1501 in combination with difluoromethylornithine (DFMO) for the treatment of malignant glioma, including diffuse intrinsic pontine glioma (DIPG). This is the company’s second Orphan Drug Designation, following the ODD granted in October 2025 for neuroblastoma.

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"This second Orphan Drug Designation is a powerful validation of AMXT 1501’s potential to make a meaningful difference for patients facing some of the most devastating cancers," said Mark Burns, PhD, Chief Scientific Officer and President of Aminex Therapeutics. "DIPG is heartbreaking — children diagnosed with this brain tumor have virtually no effective treatment options and very little time. We are committed to changing that."

DIPG is an aggressive brainstem tumor that primarily strikes children aged 5 to 10, with a median survival of less than 12 months from diagnosis. There is currently no cure.

For Families of Children with DIPG and Other High-Risk Cancers: A Trial Is Now Enrolling

The Beat Childhood Cancer Research Consortium at Penn State College of Medicine, in partnership with Aminex, is actively enrolling patients in a national Phase 1/2 clinical trial of AMXT 1501 plus DFMO in pediatric patients with DIPG, neuroblastoma, sarcomas and other high-risk childhood cancers. The trial is planned to open at 50 clinics nationwide and enroll patients. For more information, visit ClinicalTrials.gov (NCT06465199) to learn more.

AMXT 1501 is also being evaluated in adults with solid tumors including breast cancer and metastatic melanoma in an active Phase 1b/2 multicenter trial (NCT07287917.)

The FDA’s Orphan Drug Designation provides Aminex with development incentives including tax credits, fee waivers, and seven years of market exclusivity upon potential approval.

(Press release, Aminex Therapeutics, MAR 12, 2026, View Source;a-highly-aggressive-childhood-brain-cancer-302711779.html [SID1234663514])

Plus Therapeutics Reports 2025 Results, Business Progress and 2026 Anticipated Milestones for REYOBIQ™ Clinical Program and CNSide® Commercial Rollout

On March 12, 2026 Plus Therapeutics, Inc. (Nasdaq: PSTV) ("Plus" or the "Company"), a healthcare company developing and commercializing precision diagnostics and radiopharmaceuticals for central nervous system (CNS) cancers, reported financial results for the fourth quarter and year ended December 31, 2025 and provides an overview of recent and upcoming business highlights.

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"Our team remains highly focused on achieving our 2026 targets," said Marc H. Hedrick, M.D., Plus Therapeutics President and Chief Executive Officer. "Specifically, our top priority goals are CNSide commercial scale-up and REYOBIQ pivotal trial readiness; we also are looking aggressively for ways to over achieve where possible."

Q4 2025 AND RECENT HIGHLIGHTS

Corporate

Completed an upsized public offering generating $15 million in gross proceeds, extending the Company’s projected cash runway and supporting CNSide commercialization and advancement of two ongoing Phase 2 clinical programs
REYOBIQ Development

Secured American Medical Association Category III CPT reimbursement code for convection-enhanced delivery with REYOBIQ, unlocking market access and growth potential of REYOBIQ therapy in recurrent glioblastoma and pediatric brain cancer
Incorporating constructive Type B meeting feedback from the FDA to help accelerate clinical development timelines and facilitate submission of application for the approval of REYOBIQ for patients with leptomeningeal metastases (LM)
Highlighted three REYOBIQ clinical data presentations at the World Federation of Neuro-Oncology Societies/Society for Neuro-Oncology (WFNOS/SNO), building upon body of real world clinical experience with REYOBIQ in both primary and metastatic CNS cancers that continue to show promising safety profile and signs of efficacy
CNSide CSF Assay Platform

Expanded CNSide laboratory licensing to 49 of 50 U.S. states, having recently added Pennsylvania and California; enables access to CNSide Tumor Cell Enumeration (TCE) test to approximately 95% of the U.S. population
Continued to expand the CNSide Diagnostics team to support national test adoption
Announced second of planned national coverage agreements with Humana effective October 29, 2025. Combined with UnitedHealthcare national coverage agreement, CNSide CSF laboratory test policy coverage now reaches approximately 67 million people throughout the U.S.
Full Year 2025 FINANCIAL RESULTS

The Company’s cash and investments balance was $13.1 million on December 31, 2025 compared to $3.6 million on December 31, 2024
Recognized $5.2 million in grant revenue in the year ending December 31, 2025 and $5.8 million for the year ending December 31, 2024, which in both periods represents CPRIT’s share of the costs incurred for the advancement of our REYOBIQ development for the treatment of patients with LM
Total operating loss for the year ending December 31, 2025 was $15.3 million versus $14.7 million for the year ending December 31, 2024, with the increase primarily attributed to expansion of the CNSide operations team
Net loss for the year ending December 31, 2025 was $22.4 million, or $(0.29) per basic share versus $13.0 million, or $(1.95) per basic share, for the year ending December 31, 2024. The change in the net loss for the year ended December 31 was primarily due to change in fair value of derivative instruments from the Q1 2025 financings
Anticipated MILESTONES and OUTLOOK for 2026

REYOBIQ clinical program:

Define optimal dose/interval for REYOBIQ in the ReSPECT-LM Phase 2 trial; anticipate reporting data in Q3 2026
Completing enrollment in the ReSPECT-GBM Phase 2 trial for glioblastoma and conduct an End of Phase meeting with FDA with the goal of aligning on pivotal trial design. Data expected in Q4 2026
Complete commercial manufacturing scale up for REYOBIQ
Begin enrollment in the ReSPECT-PBC pediatric brain cancer Phase 1 trial
The Company expects research and development expenditures to increase in 2026 compared to 2025, due to increased costs for the ReSPECT-LM clinical trial, manufacturing scale up for REYOBIQ commercial and approval trial drug availability, and initial patient enrollments in the ReSPECT-PBC clinical trial, together with expansion of CNSide research and development teams.

CNSide commercial roll out:

Expand U.S. commercial payer coverage to >150 million covered lives
Secure Medicare coverage pathway
Achieve > 1,250 annualized test orders
Launch additional CSF tumor characterization assays to expand the CNSide platform
The Company expects general and administrative expenditures to increase in 2026 as compared to 2025 due to expanded CNSide commercial operations team (including sales, customer service and laboratory operations), such that the goal is for CNSide Diagnostics to breakeven by 2027.

About Leptomeningeal metastases (LM)
Leptomeningeal metastases (LM) are a rare but severe complication of advanced cancer, affecting the fluid-lined structures of the central nervous system. LM occurs in approximately 5% of patients with metastatic cancer, with breast cancer, lung cancer, and melanoma being the most common sources. Median survival is typically 2-6 months, and effective treatment options are limited, highlighting the urgent need for novel therapies.

About REYOBIQ (rhenium Re186 obisbemeda)
REYOBIQ (rhenium Re186 obisbemeda) is a novel injectable radiotherapy specifically formulated to deliver direct targeted high dose radiation in CNS tumors in a safe, effective, and convenient manner to optimize patient outcomes. REYOBIQ has the potential to reduce off target risks and improve outcomes for CNS cancer patients, versus currently approved therapies, with a more targeted and potent radiation dose. Rhenium-186 is an ideal radioisotope for CNS therapeutic applications due to its short half-life, beta energy for destroying cancerous tissue, and gamma energy for real-time imaging. REYOBIQ is being evaluated for the treatment of recurrent glioblastoma, leptomeningeal metastases, and pediatric brain cancer in the ReSPECT-GBM, ReSPECT-LM, and ReSPECT-PBC clinical trials. ReSPECT-GBM is supported by an award from the National Cancer Institute (NCI), part of the U.S. National Institutes of Health (NIH), and ReSPECT-LM is funded by a three-year $17.6M grant by the Cancer Prevention & Research Institute of Texas (CPRIT). The Company’s ReSPECT-PBC clinical trial for pediatric brain cancer is supported by a $3 million grant from the U.S. Department of Defense’s Peer Reviewed Cancer Research Program.

(Press release, Plus Therapeutics, MAR 12, 2026, View Source [SID1234663512])

Molecular Partners Reports Highlights and Financial Results for Full Year 2025

On March 12, 2026 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a novel class of custom-built protein drugs known as DARPin therapeutics ("Molecular Partners" or the "Company"), reported its corporate highlights and audited financial results for the full year 2025, as well as the publication of its 2025 Annual Report.

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"We are making significant progress in targeted alpha radiotherapy, as our first Radio-DARPin candidate, MP0712 targeting DLL3, enters clinical development. Early imaging and dosimetry results support the ongoing Phase 1/2a study for MP0712, which aims to address critical needs in lung cancer treatment," said Patrick Amstutz, Ph.D., CEO of Molecular Partners. He added: "We are strengthening our expertise with a strong scientific advisory board, chaired by Prof. Ken Herrmann, to support our growing Radio-DARPin pipeline, with MP0726 moving towards imaging and new candidates being selected. Our solid finances position us well for future growth."

Research & Development Highlights

MP0712 & Radio-DARPin Pipeline

The MP0712 Phase 1/2a trial has started (ClinicalTrials.gov: NCT07278479) and recruitment is open. MP0712 is the Company’s lead Radio-DARPin Therapy (RDT) targeting the tumor-associated protein delta-like ligand 3 (DLL3) and carrying the therapeutic payload 212Pb. It is being developed with Molecular Partners’ strategic partner Orano Med, a pioneer in targeted alpha therapy, for the treatment of patients with small cell lung cancer (SCLC) and other neuroendocrine cancers. The Phase 1/2a study is a multi-center study in the US, with the objectives to assess safety and determine a recommended Phase 2 dose for MP0712. The study contains a pre-treatment imaging and dosimetry step with 203Pb-labeled MP0712. The Company expects initial clinical data from this study in 2026.

Molecular Partners and the NuMeRI team presented first patient imaging and dosimetry data on MP0712 at the 8th Theranostics World Congress (TWC) in January 2026. The data from five evaluable patients with various DLL3-expressing cancers, including small cell lung, urothelial, and other neuroendocrine cancers, were generated with MP0712 carrying the diagnostic isotope 203Pb under the leadership of Dr. Mike Sathekge as part of a Named Patient Access Program under the legal framework for compassionate care in South Africa (also referred to as Section 21 of the Medicines and Related Substances Act). The images show specific uptake as well as robust accumulation of MP0712 in tumor lesions, with limited uptake in healthy tissues, as intended, confirming the initial data on MP0712 presented at the Targeted Radiopharmaceuticals (TRP) Summit Europe 2025. MP0712 is half-life engineered to promote tumor uptake over time via the DLL3 internalization and replenishment mechanism. The biodistribution and dosimetry extrapolations are supportive of the Phase 1/2a study design and of the clinical development plans of MP0712 carrying the therapeutic isotope 212Pb for patients with small cell lung cancer (SCLC) and other DLL3-expressing neuroendocrine cancers.

The Company’s second RDT program MP0726, co-developed with Orano Med, targets mesothelin (MSLN), a tumor target overexpressed across several cancers with high unmet need, such as ovarian cancer. Molecular Partners has developed Radio-DARPins able to selectively bind to membrane-bound MSLN without being impacted by shed MSLN – a mechanism which has hampered the development of other MSLN-targeting therapeutics. The Company presented preclinical data on MP0726 at the 2025 Annual Meeting of the Society of Nuclear Medicine and Molecular Imaging (SNMMI). The Company is planning to progress several Radio-DARPin programs towards first-in-human imaging, including MP0726.

For its growing Radio-DARPin pipeline and based on the first-in-human Radio-DARPin data presented at TWC 2026 indicating that Radio-DARPins may be suitable vectors for alpha-emitting isotopes, including 212Pb and also the longer-lived 225Ac, Molecular Partners is evaluating various radio-nuclides moving forward to tailor Radio-DARPin candidates to patient needs – matching vector and isotope properties with target and disease biology. Molecular Partners entered a non-exclusive development agreement with Eckert & Ziegler for targeted alpha radio-therapeutics, thereby enabling the potential of Radio-DARPins for a range of therapeutic isotopes, including 225Ac, and advancing the Company’s wholly owned pipeline. The Company plans to present pre-clinical data on Radio-DARPins suitability with multiple isotopes at the 3rd Global Radiopharmaceuticals Development Summit in March 2026 in Shanghai, China.

In December 2025 Molecular Partners announced the formation of a scientific advisory board (SAB) to accelerate the development of its targeted radiotherapeutics. The SAB, chaired by well-known nuclear medicine expert Prof. Ken Herrmann, will be instrumental in guiding Molecular Partners strategic direction as it transitions and evolves from early clinical validation to full clinical development of its targeted alpha radiotherapies.

Molecular Partners’ Radio-DARPins are designed as ideal vectors for precise delivery of potent alpha-emitting isotopes to tumor lesions with the potential to unlock a broad range of solid tumor targets for radiopharmaceuticals.

MP0317 (tumor-localized CD40 agonist)

An investigator-initiated, proof-of-concept Phase 2 study of MP0317 combined with standard-of-care (SoC) for the treatment of patients with advanced cholangiocarcinoma is now open with two sites activated (NCT07036380) and patient dosing ongoing. The study is a randomized, multicenter study in France and aims to recruit 75 patients (with a 2to1 design, including 50 patients in the experimental arm, and 25 in the control arm). The objective of the study is to assess the clinical benefit of MP0317 combined with SoC comprising the immunotherapy durvalumab, an anti-PD-L1 checkpoint inhibitor, plus gemcitabine-cisplatin-based chemotherapy, compared to SoC alone. The tumor microenvironment (TME) is known to play a crucial role in cholangiocarcinoma development and treatment resistance. MP0317 is hypothesized to lead to immune-mediated reshaping of the TME, thereby improving the 12-month progression-free survival rate of patients compared to those treated with SoC only.

MP0317 is designed to activate immune cells specifically within the TME by anchoring to fibroblast activation protein (FAP), which is expressed in high amounts in the stroma of various solid tumors, including cholangiocarcinoma. The Company completed a Phase 1 dose-escalation study of MP0317 in patients with advanced solid tumors in 2024 (NCT05098405; 46 patients treated across 9 dose levels). Comprehensive biomarker analyses from this trial showed tumor-localized CD40 activation and remodeling of the TME. CD40 is an attractive target for cancer immunotherapy due to its strong immune-stimulatory activity. Molecular Partners believes that MP0317’s tumor-localized approach has the potential to deliver superior efficacy with fewer side effects compared to systemic CD40 agonists.

MP0533 (multispecific T cell engager)

MP0533 is currently being evaluated in a Phase 1/2a clinical trial for relapsed/refractory acute myeloid leukemia (AML) and myelodysplastic syndrome/AML (NCT05673057).

Data presented at the 2025 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting showed that densified dosing appears tolerable, and leads to markedly improved serum exposure in cycle 1, along with antitumor activity, in particular in patients with low bone marrow blast count at baseline. Cohort 10 is currently dosing patients and an update on this study is expected in H1 2026.

Molecular Partners plans to support the exploration of MP0533 in combination, both in patients with relapsed/refractory disease as well as in front-line, and has been approached by several consortia expressing interest in conducting such studies. The Company is actively engaging with key opinion leaders and regulators to shape the next phase of development, and anticipates updating the clinical plan for MP0533 in H1 2026.

MP0533 is a novel tetra-specific T cell-engaging DARPin designed for selective and broad killing of AML cells in a mutation-agnostic manner. MP0533’s mode of action enables T cell-mediated killing of AML cells – which commonly co-express at least two of the three targeted antigens (CD33, CD123, CD70) – while preserving a therapeutic window that minimizes damage to healthy cells, which normally express one or none of the targets.

Switch-DARPin Platform (next-generation immune cell engagers)

Molecular Partners designed a logic-gated Switch-DARPin T-cell engager (TCE) to achieve conditional tumor-localized immune activation targeting MSLN and epithelial cell adhesion molecule (EpCAM), which are highly co-expressed in ovarian cancer and other solid tumors. The Switch-DARPin TCE is designed to unmask the CD3-engaging DARPin ("Switch" on) and to activate T cells only upon binding to both MSLN and EpCAM. Co-engagement of CD2 leads to sustained T-cell activation and cytotoxic capacity, enabling the development of potent TCEs with an improved therapeutic window. This Switch-DARPin is half-life extended through a Fc domain, which broadens the Company’s capabilities in half-life engineering modalities.

Based on the encouraging pre-clinical data presented at the 2025 Annual Meetings of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) and the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), the Company intends to nominate a lead Switch-DARPin candidate for development in H1 2026 and will provide an update on the program at AACR (Free AACR Whitepaper) 2026.

Corporate Governance & Leadership Highlights

Molecular Partners appointed Martin Steegmaier, Ph.D., as Chief Scientific Officer (CSO) and member of its Executive Committee, effective October 1, 2025. Martin brings a wealth of experience in oncology drug development, having previously contributed to the advancement of several innovative cancer therapies at major biotech and pharmaceutical companies.

In 2025 Molecular Partners performed a strategic review of its operations and headcount, with the objectives of increased efficiency in the organization and to sharpen the focus on advancing its clinical assets.

In April 2025, all motions proposed by the Board of Directors at the Annual General Meeting were approved by the shareholders of the Company by a wide majority.

2025 Financial Highlights

In the financial year ended December 31, 2025, Molecular Partners recognized no revenue (2024: CHF 5.0 million) and incurred total operating expenses of CHF 58.1 million (2024: CHF 66.2 million). This led to an operating loss of CHF 58.1 million for 2025 (2024: Operating loss of CHF 61.2 million).

The net financial loss recorded in 2025 was CHF 3.5 million, compared to a net financial gain of CHF 7.2 million in 2024. This resulted in a 2025 net loss of CHF 61.7 million (2024: Net result of CHF 54.0 million).

The net cash used in operating activities in 2025 was CHF 51.3 million (2024: Net cash used in operating activities CHF 59.2 million). Including short-term time deposits, the cash and cash equivalents position decreased by CHF 56.3 million as compared to year-end 2024, to CHF 93.1 million as of December 31, 2025 (December 31, 2024: CHF 149.4 million). Total shareholders’ equity stood at CHF 80.3 million as of December 31, 2025, a decrease of CHF 61.3 million (December 31, 2024: CHF 141.6 million).

The Company’s cash and cash equivalents and short-term time deposits were CHF 93.1 million as of December 31, 2025, and based on current operating assumptions, will be sufficient to fund its operating expenses and capital expenditure requirements until 2028.

The Company’s balance sheet remained debt-free as of December 31, 2025. As of December 31, 2025, the Company employed 134.0 FTE (full-time equivalents). About 81% of the employees are employed in R&D-related functions.

Key figures as of December 31, 2025

Key Financials (CHF million, except per share, FTE data) FY 2025 FY 2024 Change
Total revenues and other income — 5.0 (5.0 )
R&D expenses (40.2 ) (48.6 ) 8.4
SG&A expenses (15.2 ) (17.6 ) 2.4
Restructuring expenses (2.7 ) — (2.7 )
Total operating expenses (incl depr. & amort.) (58.1 ) (66.2 ) 7.9
Operating result (58.1 ) (61.2 ) 3.1
Net finance result (3.5 ) 7.2 (10.7 )
Net result (61.7 ) (54.0 ) (7.6 )
Basic net result per share (in CHF) (1.65 ) (1.59 ) 0.30
Net cash (used in) from operating activities (51.3 ) (59.2 ) 7.9
Cash & cash equivalents (incl. short-term time deposits) 93.1 149.4 (56.3 )
Total shareholders’ equity 80.3 141.6 (61.3 )
Number of total FTE 134.0 158.5 (24.5 )

Financial outlook 2026

For the full year 2026, at constant exchange rates, the Company expects total operating expenses of CHF 45-55 million, of which around CHF 6 million will be non-cash effective costs for share-based payments, IFRS pension accounting and depreciation.

Documentation

This press release, the Company’s Annual Report on Form 20-F for the year ended December 31, 2025 to be filed with the U.S. Securities and Exchange Commission (SEC), and the Company’s annual report 2025 will be made available through www.molecularpartners.com under the investor section after 10.00 pm CET (4.00 pm EST) on March 12, 2026.

Financial calendar

April 14, 2026 Annual General Meeting
May 12, 2026 Interim Management Statement Q1 2026
August 25, 2026 Half-year results 2026 (unaudited)
October 29, 2026 Interim Management Statement Q3 2026

The latest timing of the above events can always be viewed on the investor section of the website.

(Press release, Molecular Partners, MAR 12, 2026, View Source [SID1234663511])

Theriva™ Biologics Reports Full-Year 2025 Operational Highlights and Financial Results

On March 12, 2026 Theriva Biologics, Inc. (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported financial results for the full year ended December 31, 2025, and provided a corporate update.

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"We are very pleased to have completed the licensing of our versatile Phase 2-ready asset SYN-020 to Rasayana Therapeutics, executing on our plan to derive value from our GI portfolio while we remain focused on the advancement of our oncology pipeline," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We continue to advance our lead oncolytic virus candidate VCN-01 towards pivotal clinical development in multiple indications of high unmet need. With the recent feedback from the EMA, we have further clarity on dosing regimen, protocol and overall design for our proposed Phase 3 trial in pancreatic ductal adenocarcinoma (PDAC). An End-of-Phase 2 meeting with the FDA is planned for the first half of 2026 to finalize our design for a multinational pivotal Phase 3 trial in PDAC, aiming to provide patients with a novel therapy for this difficult to treat solid tumor that has a high mortality rate. Additional interactions with the FDA and EMA are planned for 2026 to refine the design of a potential Phase 2/3 trial for retinoblastoma, for which VCN-01 has received Orphan Drug and Rare Pediatric Disease designation. We continue to engage in potential partnership discussions for the additional innovative drug candidates in our portfolio."

Recent Highlights and Anticipated Milestones

VCN-01

Metastatic Pancreatic Ductal Adenocarcinoma (PDAC):

As recently announced, Theriva received positive scientific advice from the EMA on the design of a Phase 3 trial in PDAC. The EMA provided overall agreement with the proposed Phase 3 clinical trial of VCN-01 in combination with gemcitabine/nab-paclitaxel for the first-line treatment of metastatic PDAC, including sample size, repeated dosing of VCN-01, and an adaptive design to potentially optimize trial timelines and outcomes. Specific advice included agreement on proposed inclusion/exclusion criteria, primary endpoint (overall survival), secondary endpoints (including progression free survival, duration of response, and patient reported outcomes).
An End-of-Phase 2 meeting with the FDA is planned for 1H 2026, aiming to finalize the design of a pivotal multinational Phase 3 clinical trial in PDAC.
Additional analysis from the VIRAGE Phase 2b study has been accepted for a poster presentation at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) meeting in San Diego, CA, April 17-22, 2026.
Retinoblastoma:

Further discussions are planned with both the FDA and EMA in 2026 to align on the protocol design, including target population and endpoints, for a Phase 2/3 trial in retinoblastoma, an indication for which VCN-01 has been granted Orphan Drug and Rare Pediatric Disease designation.
Safety and clinical outcomes from the Phase 1 study of VCN-01 in refractory retinoblastoma patients were recently presented at the 41st Asia-Pacific Academy of Ophthalmology (APAO) Congress.
SYN-020

On February 18, 2026, Theriva announced that it granted Rasayana Therapeutics, Inc. an exclusive, worldwide license, with the right to sublicense, to develop and commercialize SYN-020 (recombinant bovine intestinal alkaline phosphatase) for therapeutic and diagnostic use. Theriva received a $300,000 up-front payment at signing and is eligible for up to $16M in development and regulatory milestones, tiered single digit royalties on net product sales, and up to $22M in milestones payable upon achievement of certain annual aggregate net sales.
SYN-020 was well tolerated in Phase 1 clinical studies and is now poised to enter Phase 2 clinical testing. Rasayana will assume responsibility and costs for future clinical development and commercialization.
Full-Year Ended December 31, 2025 Financial Results

General and administrative expenses increased to $15.4 million for the year ended December 31, 2025, from $7.4 million for the year ended December 31, 2024. This increase of 109% is primarily comprised of the contingent consideration adjustment of $9.0 million due to the VIRAGE Phase 2b clinical trial of VCN-01 (zabilugene almadenorepvec) in PDAC achieving its primary survival and safety endpoints, offset by a decrease in compensation costs, investor relations costs, consulting fees, and lower director and officer insurance. The charge relating to stock-based compensation expense was $379,000 for the year ended December 31, 2025, compared to $438,000 for the year ended December 31, 2024.

Research and development expenses decreased to $8.6 million for the year ended December 31, 2025, from $12.0 million for the year ended December 31, 2024. This decrease of 28% is primarily the result of lower clinical trial expenses related to the completion of our VIRAGE Phase 2b clinical trial of VCN-01 (zabilugene almadenorepvec) in PDAC, lower clinical trial expenses related to our Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients and lower indirect cost related to compensation, offset by higher patent expenses related to SYN-020. We anticipate research and development expense to decrease in 2026 as a result of the workforce reduction plan that was implemented on September 30, 2025, the completion of our VIRAGE Phase 2b clinical trial of VCN-01 and our focus on regulatory interactions around potential pivotal clinical trials of VCN-01 in PDAC and retinoblastoma, the planning for VCN-01 manufacturing scale-up activities, a potential Phase 2a study evaluating VCN-01 dosing frequency, and continuing to support our other preclinical and discovery initiatives. In addition, pursuant to the terms of the Rasayana License Agreement that we entered into in February 2026, we will not continue to conduct research and development activities with respect to SYN-020, and do not expect to incur material expenditures in connection therewith since Rasayana is now responsible for all such expenditures including patent expenses. Research and development expenses also include a charge relating to non-cash stock-based compensation expense of $281,000 for the year ended December 31, 2025, compared to $233,000 for the year ended December 31, 2024.

Cash and cash equivalents totaled $13.1 million as of December 31, 2025, compared to $11.6 million as of December 31, 2024. The Company’s current cash of approximately $15.2 million at February 26, 2026 will allow it to fund operations into the first quarter of 2027, including overhead costs, close out of the VIRAGE Phase 2b clinical trial, a potential Phase 2a study evaluating VCN-01 dosing frequency, exploratory VCN-01 (zabilugene almadenorepvec) manufacturing scale-up activities, regulatory interactions regarding proposed VCN-01 clinical trials in PDAC and retinoblastoma, and preclinical studies supporting VCN-01 and VCN-12, the first candidate from our VCN-X discovery program.

The audited financial statements for the year ended December 31, 2025 included in the Company’s Annual Report on Form 10-K contain an audit opinion from the Company’s independent registered public accounting firm that includes an explanatory paragraph related to the Company’s ability to continue as a going concern.

About VCN-01

VCN-01 (zabilugene almadenorepvec) is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to 142 patients to date in clinical trials of different cancers, including pancreatic ductal adenocarcinoma (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.

About SYN-020

SYN-020 is a recombinant bovine intestinal alkaline phosphatase (IAP) produced under cGMP conditions and formulated for oral delivery to the small intestine. SYN-020 is designed to reduce fat absorption and intestinal inflammation, tighten the gut barrier to mitigate leaky gut, and promote a healthy microbiome. These complementary modes of action mean SYN-020 has the potential to address multiple metabolic and inflammatory disorders and diseases associated with aging. Despite its broad therapeutic potential, a key hurdle to commercialization has been the high cost of IAP manufacture. Theriva has overcome this hurdle and has developed a process to produce SYN-020 at a scale and cost viable for clinical and commercial development. In February 2026, Theriva granted to Rasayana Therapeutics, Inc. an exclusive, worldwide license, with the right to sublicense, to develop and commercialize SYN-020 (recombinant bovine intestinal alkaline phosphatase) for therapeutic and diagnostic use.

(Press release, Theriva Biologics, MAR 12, 2026, View Source [SID1234663510])