Synthetic Biologics(US:TOVX)2025-11-12 13:16Oncology Focus - The company transitioned its strategic focus to oncology following the acquisition of Theriva Biologics in March 2022, developing the oncolytic adenovirus platform for cancer treatment [122]. - The lead product candidate, VCN-01, is currently in a Phase 2b clinical study for pancreatic cancer and has been used in Phase 1 studies for retinoblastoma and other solid tumors [122]. - The VIRAGE Phase 2b trial for metastatic pancreatic cancer involved 112 patients, comparing standard chemotherapy with and without VCN-01 [135]. - Patients receiving VCN-01 + GA showed improved overall survival (OS) of 14.8 months compared to 11.6 months for GA alone, with a hazard ratio (HR) of 0.44 (P=0.046) [136]. - The Phase 2b VIRAGE trial achieved target enrollment of 92 evaluable patients, with 36 patients receiving their second doses of intravenous VCN-01, which were well tolerated [146]. - VCN-01 has been administered to 142 patients across multiple Phase 1 clinical trials and the Phase 2b VIRAGE trial, targeting various cancers including pancreatic cancer and retinoblastoma [142]. - 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 [149]. - The Independent Data Monitoring Committee found that VCN-01 was well tolerated in combination with standard-of-care chemotherapy, with adverse events consistent with prior clinical trials [153]. - The company received Rare Pediatric Drug Designation for VCN-01 for the treatment of retinoblastoma, which may lead to a Priority Review Voucher if approved by September 30, 2026 [160]. - The European Medicines Agency granted Orphan Medicinal Product Designation to VCN-01 for the treatment of retinoblastoma [161]. - VCN-01 demonstrated enhanced anti-tumor effects in human pancreatic cancer xenograft-bearing mice when combined with liposomal irinotecan [148]. - The Phase 2b trial of VCN-01 in combination with standard-of-care chemotherapy is being conducted at approximately 17 sites in the US and EU [145]. - The company plans to conduct a stand-alone Phase 3 study of VCN-01 with gemcitabine/nab-paclitaxel based on FDA guidance [151]. - The Phase 1 trial of VCN-01 combined with durvalumab enrolled 20 patients, with a median of 4 prior lines of therapy, showing promising results in both concomitant and sequential administration schedules [164]. - In the concomitant schedule, overall survival (OS) was 10.4 months, while in the sequential schedule, OS was 15.5 months at a dose of 3.3×10^12 viral particles (vp) and 17.3 months at 1×10^13 vp [168]. - 66.6% of patients receiving huCART-meso after VCN-01 showed tumor shrinkage, indicating a potential benefit of the combination therapy [167]. - The study of VCN-01 in patients with high-grade brain tumors aims to confirm the presence of the treatment in resected specimens, potentially paving the way for larger trials [170]. - VCN-01 showed sustained blood levels and increased serum hyaluronidase levels for over six weeks, indicating biological activity in patients [168]. - The clinical trial of huCART-meso cells in combination with VCN-01 demonstrated safety consistent with expectations from monotherapy studies [167]. - VCN-01 combined with liposomal irinotecan showed significant tumor growth inhibition in pancreatic cancer models [189][195]. - VCN-11, a next-generation oncolytic adenovirus, exhibited 450 times more cytotoxicity in tumor cells compared to normal cells [191]. Financial Performance - The company implemented a workforce reduction of approximately 32%, or seven employees, to focus resources on business development and clinical trial planning, expecting to save approximately $1.8 million annually [125]. - The company raised approximately $4.4 million from the exercise of existing warrants, with net proceeds expected to be used for working capital [127]. - The company plans to extend its cash runway into the first quarter of 2027 through cost reductions and capital raised from the ATM Sales Agreement [125]. - General and administrative expenses decreased by 18% to $1.9 million for the three months ended September 30, 2025, from $2.3 million for the same period in 2024 [209]. - Research and development expenses decreased by 7% to $2.6 million for the three months ended September 30, 2025, from approximately $2.7 million for the same period in 2024 [210]. - Total research and development expenses for the nine months ended September 30, 2025, were $7.5 million, a decrease of 18% from approximately $9.1 million for the same period in 2024 [217]. - The net loss for the three months ended September 30, 2025, was $4.4 million, or ($0.45) per common share, compared to a net loss of $7.7 million, or ($6.81) per common share for the same period in 2024 [215]. - The net loss attributable to common stockholders for the nine months ended September 30, 2025, was approximately $21.7 million, or ($3.38) per share, compared to a net loss of approximately $21.2 million, or ($24.47) per share for the same period in 2024 [222]. - As of September 30, 2025, the accumulated deficit was $356.7 million, with expectations of continued losses due to reliance on successful phase 3 clinical trials and FDA approvals [223]. - Cash and cash equivalents totaled $7.5 million as of September 30, 2025, a decrease of $4.1 million from December 31, 2024, but increased to $15.5 million in early November 2025 due to net proceeds from sales [224]. - Cash used in operating activities was $13.8 million for the nine months ended September 30, 2025, compared to $12.2 million for the same period in 2024, primarily for the development of VCN-01 [235]. - Cash provided by financing activities for the nine months ended September 30, 2025, included $1.8 million from research and development tax credits and $6.7 million from the sale of Common Stock [237]. - The company has an obligation to pay up to $70.2 million in additional consideration under the VCN Purchase Agreement, with $6.8 million paid to date [229]. - Future clinical trials are expected to require larger cash expenditures, and the company currently lacks committed sources of financing for these trials [227]. - The company anticipates needing additional funding to continue the development of product candidates and to maintain operations beyond early 2027 [232]. - There is substantial doubt about the company's ability to continue as a going concern without additional capital or strategic actions [225]. Research and Development - The company is exploring value creation options for its previous GI disease assets, SYN-004 and SYN-020, including out-licensing or partnering [123]. - The Phase 1b/2a clinical trial of SYN-004 (ribaxamase) aims to evaluate safety and tolerability in up to 36 adult allogeneic hematopoietic cell transplant (HCT) recipients [177]. - Positive outcomes from the Data and Safety Monitoring Committee review allowed the study to proceed to the second cohort, which involves administering SYN-004 with piperacillin/tazobactam [178]. - The SYN-004 program targets prevention of antibiotic-mediated microbiome damage, potentially reducing the incidence of Clostridioides difficile infection (CDI) and acute graft-versus-host disease (aGVHD) [173]. - The company is actively maintaining and building its patent portfolio to support its gastrointestinal and microbiome-focused pipeline [172]. - SYN-004 (ribaxamase) Phase 1b/2a trial safety and pharmacokinetic data presented at major conferences in 2023 [179][180]. - SYN-020 production cost anticipated to be a few hundred dollars per gram, significantly lower than the current market price of $10,000 per gram [182]. - SYN-020 demonstrated a favorable safety profile in Phase 1 studies, with no serious adverse events reported [185]. - The company is exploring strategic opportunities for SYN-004 and SYN-020 assets, including out-licensing or partnerships [188]. - Enrollment for the third cohort of the SYN-004 trial is contingent on obtaining grant funding or finding a partner [180]. - Research and development expenses directly related to VCN-01 were $1.4 million for the three months ended September 30, 2024, compared to $1.37 million for the same period in 2025 [212]. - Total direct research and development costs for the nine months ended September 30, 2025, were $4.89 million, down from $5.77 million for the same period in 2024 [218]. - The fair value of in-process R&D was written down from $19.8 million to $18.6 million, resulting in a $1.3 million impairment charge during the nine months ended September 30, 2024 [220]. - The charge related to stock-based compensation expense was $269,000 for the nine months ended September 30, 2025, compared to $335,000 for the same period in 2024 [216]. - Contingent consideration liabilities increased by $9.2 million due to the achievement of primary survival and safety endpoints in the VIRAGE Phase 2b clinical trial of VCN-01 [216]. - Theriva Biologics holds over 135 U.S. and foreign patents, supporting its various programs [197]. - SYN-004 and SYN-020 programs are backed by strong intellectual property, with patent terms extending to at least 2035 and 2038-2040 respectively [198][199]. - THERICEL program aims to enhance the efficiency and reduce the cost of VCN-01 manufacturing for clinical trials [194]. Corporate Governance - The company filed a prospectus supplement to offer and sell up to $2,534,352 of common stock, with sales made through an at-the-market offering [124]. - The Board approved an amendment to the 2020 Stock Incentive Plan, increasing the number of shares available for grant from 2,500,000 to 4,500,000 [128]. - The company does not recognize Right of Use (ROU) assets or lease liabilities for short-term leases with original terms of 12 months or less [240]. - As of September 30, 2025, the company did not have any material finance leases [240]. - ROU assets and liabilities are recognized at the commencement date based on the present value of lease payments over the lease term [239].