Financial Performance - The net loss for the three months ended March 31, 2025, was $4.3 million, or ($1.55) per common share, compared to a net loss of $5.2 million, or ($7.53) per common share for the same period in 2024[229]. - Cash and cash equivalents totaled $10.0 million as of March 31, 2025, a decrease of $1.6 million from December 31, 2024[231]. - The accumulated deficit was $339.3 million as of March 31, 2025, with expectations of continued losses in the foreseeable future[230]. - Cash used in operating activities was $4.8 million during the three months ended March 31, 2025, compared to $4.9 million for the same period in 2024[236]. - Total research and development expenses for the three months ended March 31, 2025, were $2.968 million, down from $3.459 million in 2024[227]. - Research and development expenses decreased to $3.0 million for the three months ended March 31, 2025, from approximately $3.5 million for the same period in 2024, a decrease of 14%[225]. - General and administrative expenses decreased to $1.4 million for the three months ended March 31, 2025, from $1.9 million for the same period in 2024, a decrease of 25%[224]. - Cash provided by financing activities during the three months ended March 31, 2025, included $1.7 million received for the research and development tax credit[238]. - As of early May 2025, cash position was $14.1 million, expected to fund operations into the first quarter of 2026[232]. - Future clinical trials for VCN-01, SYN-004, and SYN-020 will require significant financing, and the company does not currently have committed sources of funding[235]. Clinical Trials and Product Development - The lead product candidate, VCN-01, is currently in a Phase 2b clinical study for pancreatic cancer, with positive topline outcomes indicating a median overall survival (OS) of 10.8 months for patients receiving VCN-01 plus standard-of-care chemotherapy compared to 8.6 months for those receiving standard care alone[145][149]. - In the VIRAGE trial, patients receiving two doses of VCN-01 showed a median OS of 14.8 months compared to 11.6 months for those receiving standard care, suggesting a significant benefit from the additional dose[146]. - The Phase 2b clinical trial of VCN-01 in combination with nab-paclitaxel and gemcitabine is ongoing, with additional studies evaluating VCN-01 in other cancer types[154]. - The Phase 2b clinical trial VIRAGE for VCN-01 in combination with gemcitabine and nab-paclitaxel has enrolled 92 evaluable patients, achieving the target enrollment by September 23, 2024[157]. - Topline data for the VIRAGE Phase 2b clinical trial is anticipated for Q2 2025[157]. - The FDA granted Fast Track Designation to VCN-01 in combination with gemcitabine and nab-paclitaxel to improve progression-free survival and overall survival in patients with metastatic pancreatic adenocarcinoma on May 23, 2024[161]. - An Independent Data Monitoring Committee confirmed that VCN-01 was well tolerated in the Phase 2b trial, with adverse events consistent with prior trials and less frequent after the second dose[164]. - The clinical trial for VCN-01 in retinoblastoma showed positive topline data, with no dose-limiting toxicities and treatment-related adverse events primarily Grade 1 or 2[165][166]. - The Phase 1 trial combining VCN-01 with durvalumab showed well-tolerated results, with sustained blood levels of VCN-01 and increased tumor inflammation markers[173]. - The ongoing studies suggest that VCN-01 may enhance the potential antitumor effects of co-administered huCART-meso cells in patients with unresectable or metastatic pancreatic adenocarcinoma[176]. - The Phase 1 trial of huCART-meso cells combined with VCN-01 showed overall survival (OS) of 10.4 months in the concomitant cohort and 15.5 months in the sequential cohort at a dose of 3.3×10^12 viral particles (vp), with OS reaching 17.3 months at a dose of 1×10^13 vp[178]. - 61.1% of patients (11 out of 18) were alive for more than 12 months, indicating a promising trend in disease stabilization[178]. - VCN-01 demonstrated biological activity with replication observed and increased serum hyaluronidase levels maintained for over six weeks[178]. - The safety profile of VCN-01 was consistent with expectations from monotherapy studies, with 66.6% of patients showing tumor shrinkage when receiving huCART-meso after VCN-01[179]. - A new next-generation mesothelin-specific CAR-T is being developed, which may be evaluated in combination with VCN-01 in future clinical trials[180]. - The Phase 1 clinical study of SYN-004 (ribaxamase) aims to prevent antibiotic-mediated microbiome damage and is currently in a randomized, double-blinded, placebo-controlled trial with 36 evaluable adult allogeneic HCT recipients[186][187]. - SYN-020, a recombinant version of bovine Intestinal Alkaline Phosphatase, is being developed to mitigate intestinal damage caused by radiation therapy, with production costs anticipated to be significantly lower than current market rates[192]. - The Phase 1 clinical study of SYN-020 demonstrated a favorable safety profile with no serious adverse events reported, and it was well tolerated across all dose levels[196]. - The company has received a study-may-proceed letter from the FDA for SYN-020, allowing for further clinical development[195]. - Phase 1 data from SAD and MAD studies support the development of SYN-020 for multiple clinical indications, including NAFLD and aging-related diseases[198]. Funding and Grants - The company completed a public offering on May 8, 2025, raising approximately $7.5 million through the sale of 1,990,900 shares of Common Stock and warrants[134]. - The company received €2.28 million (approximately $2.54 million) from the Spanish government's National Knowledge Transfer Program to support the THERICEL project, which includes a €1.3 million loan and a €0.95 million grant[139]. - The company received $1.7 million from the 2023 Research and Development rebate program sponsored by the Spanish government[138]. Intellectual Property and Licensing - The company is exploring value creation options for its previous assets, SYN-004 and SYN-020, including out-licensing or partnering[133]. - The ongoing clinical trials and product developments are supported by a growing intellectual property portfolio, with efforts to maintain and build patent applications[183]. - The company has over 130 U.S. and foreign patents and over 65 patents pending, supporting its various programs[208]. - The SYN-004 program is supported by patents that will expire in at least 2031, covering compositions of matter and uses of beta-lactamases[209]. - The VCN-01 and VCN-11 programs are supported by patents that provide coverage for engineered oncolytic adenoviruses, with some patents expiring as late as 2037[211]. - The company entered into an agreement with Massachusetts General Hospital for an exclusive license related to IAP technology, which expired unexercised on July 1, 2024[197]. Manufacturing and Development - The THERICEL program is advancing a proprietary A549 suspension cell line to enhance the efficiency and reduce the cost of manufacturing VCN-01 for clinical trials[205]. - Preclinical data indicated that the ABD-containing virus VCN-11 induced 450 times more cytotoxicity in tumor cells compared to normal cells, demonstrating a low toxicity profile[203].

Recent Highlights and Anticipated Milestones Exhibit 99.1 Theriva™ Biologics Reports First Quarter 2025 Operational Highlights and Financial Results - VCN-01 Achieves Primary Efficacy and Safety Endpoints for Pancreatic Ductal Adenocarcinoma in VIRAGE Phase 2b Clinical Trial - - Closed a public offering on May 8, 2025, raising the Company's cash balance and extending its cash runway into the first quarter of 2026 - Rockville, MD, May 14, 2025 – Theriva™ Biologics (NYSE American: TOVX), a diversified clinica ...

Core Viewpoint - Theriva Biologics has successfully closed a public offering, raising approximately $7.5 million to support its cancer therapeutics development and other corporate purposes [1][2]. Group 1: Offering Details - The public offering consisted of 6,818,180 shares of common stock and warrants to purchase an equal number of shares at a combined price of $1.10 per share [1]. - The warrants are exercisable immediately and will expire five years from the issuance date [1]. - The offering was conducted under a registration statement that was declared effective by the SEC on May 7, 2025 [3]. Group 2: Use of Proceeds - The net proceeds from the offering will primarily be used for working capital, general corporate purposes, research and development, and manufacturing scale-up [2]. - The company may also consider investing in or acquiring other products, businesses, or technologies, although no commitments currently exist [2]. Group 3: Company Overview - Theriva Biologics is a clinical-stage company focused on developing therapeutics for cancer and related diseases, with a particular emphasis on high unmet medical needs [5]. - The company's lead candidates include VCN-01, SYN-004, and SYN-020, targeting various aspects of cancer treatment and gastrointestinal health [5].

Core Viewpoint - Theriva Biologics announced a public offering of up to 6,818,180 shares of common stock and warrants, aiming to raise approximately $7.5 million for working capital and R&D purposes [1][2]. Group 1: Offering Details - The offering price is set at $1.10 per share and accompanying warrant, with warrants exercisable immediately and expiring five years from issuance [1]. - The closing of the offering is expected on or about May 8, 2025, pending customary closing conditions [2]. - A.G.P./Alliance Global Partners is acting as the sole placement agent for the offering [3]. Group 2: Use of Proceeds - The net proceeds from the offering will primarily be used for working capital and general corporate purposes, including research and development and manufacturing scale-up [2]. - The company may also consider using a portion of the proceeds for investments or acquisitions, although no commitments currently exist [2]. Group 3: Company Overview - Theriva Biologics is a clinical-stage company focused on developing therapeutics for cancer and related diseases, with a particular emphasis on high unmet medical needs [6]. - The company's lead candidates include VCN-01, SYN-004, and SYN-020, targeting various aspects of cancer treatment and microbiome protection [6].

Core Insights - Theriva Biologics announced positive topline outcomes from the VIRAGE Phase 2b clinical trial for VCN-01 in combination with standard-of-care chemotherapy for metastatic pancreatic ductal adenocarcinoma (PDAC) patients [1][3] Company Overview - Theriva Biologics is a diversified clinical-stage company focused on developing therapeutics for cancer and related diseases, with its lead product candidate being VCN-01, an oncolytic adenovirus [10][11] Clinical Trial Results - The VIRAGE trial involved 96 newly-diagnosed metastatic PDAC patients, showing that patients receiving 2 doses of VCN-01 plus chemotherapy had a median overall survival (OS) of 14.8 months compared to 11.6 months for those receiving chemotherapy alone [2] - In the primary endpoint analysis, patients treated with VCN-01 followed by chemotherapy had a median OS of 10.8 months, while those receiving only chemotherapy had a median OS of 8.6 months [5] - The trial demonstrated increased progression-free survival (PFS) and duration of response (DoR) in the VCN-01 treatment group, with median PFS of 7.0 months compared to 4.6 months for the control group [5] Safety Profile - VCN-01 was well-tolerated, with adverse events being transient and reversible, consistent with prior clinical trials [3][4] - The most common adverse events included pyrexia, flu-like illness, and elevated transaminases, which were less frequent after the second dose of VCN-01 [3] Future Directions - The positive data from the VIRAGE trial is expected to facilitate engagement with industry partners and support the design of a Phase 3 confirmatory trial [3][4]

Core Viewpoint - Theriva Biologics is presenting interim blinded safety and pharmacokinetic data for SYN-004 at the ESCMID Global Congress, focusing on its potential to prevent acute graft-versus-host disease in allogeneic hematopoietic cell transplant recipients [1][3]. Group 1: Clinical Trial Details - The ongoing Phase 1b/2a clinical trial is randomized, double-blinded, and placebo-controlled, evaluating the safety and tolerability of oral SYN-004 in allogeneic HCT recipients receiving IV antibiotics [3]. - The trial is being conducted at Washington University School of Medicine and aims to enroll up to 36 participants across three cohorts, each receiving different IV beta-lactam antibiotics [3]. - Safety and pharmacokinetic data are reviewed by an independent Data and Safety Monitoring Committee to determine the progression to the next cohort [3]. Group 2: About SYN-004 - SYN-004 (ribaxamase) is designed to degrade certain IV beta-lactam antibiotics in the gastrointestinal tract, maintaining gut microbiome balance and preventing Clostridioides difficile infection and acute graft-versus-host disease [4][6]. - A previous Phase 2b clinical trial with 412 patients showed that SYN-004 protected the gut microbiome from antibiotic-mediated dysbiosis, leading to better maintenance and recovery of the microbiome [4]. Group 3: Company Overview - Theriva Biologics is a diversified clinical-stage company focused on developing therapeutics for cancer and related diseases, with a subsidiary working on an oncolytic adenovirus platform [5][6]. - The company's lead candidates include VCN-01, SYN-004, and SYN-020, targeting various aspects of cancer treatment and microbiome protection [6].

Company Overview - Theriva™ Biologics is a diversified clinical-stage company focused on developing therapeutics for cancer and related diseases in areas of high unmet need [3] - The company is advancing a new oncolytic adenovirus platform for intravenous, intravitreal, and antitumoral delivery to enhance tumor cell death and improve access to co-administered cancer therapies [3] Lead Candidates - The lead candidates include: - VCN-01, an oncolytic adenovirus that selectively replicates within tumor cells and degrades the tumor stroma barrier [3] - SYN-004 (ribaxamase), designed to degrade certain IV beta-lactam antibiotics in the gastrointestinal tract to prevent microbiome damage and reduce acute graft-versus-host disease in transplant recipients [3] - SYN-020, a recombinant oral formulation of intestinal alkaline phosphatase intended to treat local GI and systemic diseases [3] Upcoming Conference Participation - Theriva's management will present at the Cancer Advocacy Group of Louisiana NeauxCancer 2025 Conference from March 27th-29th, 2025 [1] - A company presentation is scheduled for the Innovation track on March 28th at 9:00 am, highlighting emerging biotech and healthcare companies in oncology [2] - Management will be available for one-on-one meetings with interested investors during the conference [2]

Oncology Focus and Product Development - The company transitioned its strategic focus to oncology following the acquisition of Theriva Biologics, S.L. in March 2022, emphasizing the development of oncolytic adenovirus platforms for cancer treatment[23] - The first product candidate, VCN-01, has been administered to 142 patients across multiple Phase 1 clinical trials and the Phase 2 VIRAGE trial, targeting various cancers including pancreatic cancer and head and neck squamous cell carcinoma[33] - VCN-01 is designed to degrade hyaluronan in the tumor stroma, facilitating better penetration of the virus and other therapeutic agents into tumors[30] - The oncology platform is based on oncolytic virotherapy, which utilizes viruses to selectively kill tumor cells and stimulate an anti-tumor immune response[27] - The development of oncolytic viruses has faced challenges, and the company acknowledges that its research and development efforts may not succeed in bringing successful products to market[13] Financial Position and Capital Needs - The company currently has no products approved for commercial sale and has identified material weaknesses in its internal controls, indicating a need for improved operational stability[13] - The company expects to incur significant operating and capital expenditures and will need to raise additional capital, which may be dilutive to stockholders[13] - As of December 31, 2024, the accumulated deficit totaled approximately $335.0 million on a consolidated basis[188] - During the year ended December 31, 2024, operating activities used net cash of approximately $16.9 million, with cash and cash equivalents at $11.6 million[190] - The company expects its current cash will fund operations into the third quarter of 2025 but will not be sufficient for the next twelve months[188] - The company has a significant need for additional capital to operate its business and may face delays or reductions in development programs if funding is not secured[190] - The company may need to raise additional capital through equity or debt securities, which could dilute current stockholders' ownership[204] Clinical Trial Results and Regulatory Designations - The overall response rate (ORR) for patients treated with intravenous VCN-01 in combination with chemotherapy was 50%[43] - Median progression-free survival (PFS) for patients receiving VCN-01 was 6.7 months, and median overall survival (OS) was 13.5 months[43] - The FDA granted Fast Track Designation to VCN-01 in combination with gemcitabine and nab-paclitaxel to improve PFS and OS in metastatic PDAC patients[49] - VCN-01 received Orphan Drug designation from the FDA for retinoblastoma treatment in February 2022[54] - The European Medicines Agency granted Orphan Medicinal Product Designation to VCN-01 for retinoblastoma treatment on October 11, 2024[60] Challenges in Commercialization - The company has no marketing, sales, or distribution organization and lacks experience in marketing products, which could hinder commercialization efforts[13] - The company relies on third parties for product manufacturing and may face risks related to supply chain disruptions, which could impact its operations[18] - The company faces potential delays in regulatory approvals due to the complexity and novelty of its products, which could adversely affect its business[141] - The company must conduct post-approval trials to confirm clinical benefits for drugs that receive accelerated approval, with the risk of market withdrawal if these trials do not demonstrate efficacy[150] Research and Development Efforts - The budgetary plan for VCN's research and development programs, including VCN-01 clinical trials, is approximately $27.8 million[190] - The company intends to focus capital on VCN-01 clinical trials and does not plan to provide further funding for SYN-004 development internally[190] - The Phase 1 clinical study of SYN-020 demonstrated a favorable safety profile with no serious adverse events reported among 24 subjects[94] - The VCN-X program is developing next-generation oncolytic adenoviruses with enhanced tumor targeting capabilities, leveraging the Albumin Shield technology[100] Strategic Partnerships and Collaborations - The company is exploring value creation options for its previous GI disease assets, SYN-004 and SYN-020, including out-licensing or partnering[23] - The collaboration with Sant Joan De Déu Hospital includes a payment of €500,000 (approximately $534,000) for trial results and an additional €320,000 (approximately $340,000) upon completion of a pivotal study[118] - The company has entered into a Clinical Trial Agreement with Washington University to conduct a Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients[87] Market and Competitive Landscape - The company faces intense competition from well-established pharmaceutical and biotechnology companies with greater resources[171] - The overall incidence rate of CDI in the United States is estimated at 121.2 cases per 100,000 persons, translating to approximately 500,000 patients annually[76] - CDI infections are estimated to cost the U.S. healthcare system $5 billion per year, primarily due to increased hospitalization and length of stay[76]

Clinical Trials and FDA Designations - Enrollment completed in the VIRAGE Phase 2b clinical trial of VCN-01 for metastatic pancreatic ductal adenocarcinoma (PDAC), with topline data expected in Q2 2025[1] - FDA granted Fast Track designation for VCN-01 in combination with standard chemotherapy, aiming to improve progression-free survival and overall survival in metastatic PDAC patients[3] - The company aims to request an End-of-Phase 2 meeting with the FDA before the end of 2025 to discuss the proposed Phase 3 study for VCN-01[14] - Anticipated increase in research and development expenses as the company completes the VIRAGE Phase 2 trial and plans for a Phase 3 trial of VCN-01 in PDAC[10] Financial Performance - Cash and cash equivalents totaled $11.6 million as of December 31, 2024, down from $23.2 million as of December 31, 2023[12] - General and administrative expenses increased by 4% to $7.4 million for the year ended December 31, 2024, compared to $7.1 million in 2023[9] - Research and development expenses decreased by 16% to $12.0 million for the year ended December 31, 2024, down from $14.3 million in 2023[10] - Other income was $693,000 for the year ended December 31, 2024, compared to $1.4 million in 2023, primarily due to a decrease in interest income[11] - Total operating costs and expenses increased from $21,431 million in 2023 to $26,346 million in 2024, an increase of approximately 23%[19] - Net loss attributable to common stockholders rose from $18,349 million in 2023 to $25,653 million in 2024, reflecting an increase of about 40%[19] - Net loss per share increased from $28.48 in 2023 to $19.03 in 2024, indicating a worsening financial position[19] - Total comprehensive loss increased from $17,638 million in 2023 to $26,863 million in 2024, reflecting a worsening overall financial performance[19] Assets and Equity - Total assets decreased from $55,219 million in 2023 to $35,352 million in 2024, a decline of approximately 36%[17] - Current assets fell from $27,403 million in 2023 to $16,281 million in 2024, representing a decrease of about 41%[17] - Total stockholders' equity decreased from $36,963 million in 2023 to $19,067 million in 2024, a decline of about 48%[17] - The weighted average number of shares outstanding increased from 644,282 in 2023 to 1,348,126 in 2024, indicating a significant dilution of shares[19] - Goodwill impairment of $5,594 million was recorded in 2024, with no impairment reported in 2023[19] Funding and Tax Credits - The company received a total of €2.28 million (approximately $2.54 million) in manufacturing funding from the Spanish government's National Knowledge Transfer Program[7] - The company recognized a tax credit receivable of $1.8 million from the Spanish government for R&D expenses incurred in Spain[8]

Core Insights - Theriva Biologics has completed enrollment in the VIRAGE Phase 2b clinical trial for VCN-01 targeting metastatic pancreatic ductal adenocarcinoma (PDAC), with topline data expected in Q2 2025 [1][2] - The development of VCN-01 has been supported by the FDA's Fast Track designation for PDAC and Rare Pediatric Disease designation for retinoblastoma [1][4] - The company has received guidance from the FDA and EMA regarding the design of a potential Phase 3 trial for VCN-01 in combination with standard chemotherapy for metastatic PDAC [1][4] Clinical Development Updates - The VIRAGE trial achieved its target enrollment of 92 evaluable patients, and the outcomes of a second Data Monitoring Committee review of safety data are anticipated in Q2 2025 [4] - Positive topline data from a Phase 1 trial of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma was announced in April 2024 [4] - VCN-01 received Rare Pediatric Drug Designation from the FDA and Orphan Medicinal Product Designation from the European Commission for retinoblastoma treatment [4] Financial Performance - For the year ended December 31, 2024, general and administrative expenses increased to $7.4 million, a 4% rise from $7.1 million in 2023 [8] - Research and development expenses decreased to $12.0 million, down 16% from $14.3 million in 2023, primarily due to lower clinical trial expenses [9] - The company reported a net loss of $25.7 million for the year ended December 31, 2024, compared to a net loss of $18.3 million in 2023 [18] Cash Position - As of December 31, 2024, cash and cash equivalents totaled $11.6 million, a decrease from $23.2 million as of December 31, 2023 [11] - The company recognized a tax credit receivable of $3.2 million from the Spanish government for R&D expenses [7][11] Business Development - Theriva Biologics was awarded €2.28 million (approximately $2.54 million) in manufacturing funding from the Spanish government's National Knowledge Transfer Program [6] - The company is focusing on advancing its clinical development of VCN-01 while seeking grant funding or partnerships for the SYN-004 development program [5]