Synthetic Biologics(US:TOVX)2025-08-11 12:15Oncology Development - The company is focused on developing therapeutics for cancer and related diseases, transitioning to oncology after the acquisition of Theriva Biologics in March 2022[126]. - The lead product candidate, VCN-01, has been evaluated in a Phase 2b clinical study for pancreatic cancer, showing a median overall survival (OS) of 14.8 months for patients receiving 2 doses compared to 11.6 months for those receiving standard care alone[140]. - VCN-01 has been administered to 142 patients across multiple Phase 1 and Phase 2b clinical trials, targeting various cancers including pancreatic and retinoblastoma[146]. - The Phase 1 study of VCN-01 in retinoblastoma patients showed promising results, with no systemic toxicities and a well-tolerated profile[138]. - The Phase 2b VIRAGE trial demonstrated that VCN-01 plus standard chemotherapy improved progression-free survival (PFS) to 7.0 months compared to 4.6 months for standard care alone[142]. - VCN-01 has received Orphan Drug Designation and Fast Track Designation from the FDA for the treatment of pancreatic cancer, indicating its potential in addressing unmet medical needs[139]. - The Phase 2b clinical trial VIRAGE for VCN-01 in combination with gemcitabine/nab-paclitaxel began dosing patients in January 2023, targeting newly-diagnosed metastatic pancreatic ductal adenocarcinoma (PDAC) patients[149]. - The Independent Data Monitoring Committee (IDMC) confirmed no safety concerns in the ongoing Phase 2b trial, with VCN-01 demonstrating a safety profile consistent with prior trials[151]. - 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 metastatic PDAC patients[153]. - The Phase 1 trial of VCN-01 in combination with durvalumab reported overall survival as 17.3 months for patients receiving the higher dose[166]. - The Phase 1 trial of VCN-01 in combination with huCART-meso cells showed that 66.6% (4 out of 6) patients with measurable disease experienced tumor shrinkage, indicating promising disease stabilization trends[169]. - The ongoing Phase 1 trial of VCN-01 for high-grade brain tumors aims to confirm the presence of VCN-01 in resected surgical specimens, potentially paving the way for larger efficacy trials[171]. - The company is focusing on the development of a next-generation mesothelin-specific CAR-T, potentially in combination with VCN-01 in future trials[170]. - The next-generation oncolytic adenovirus VCN-11 demonstrated 450 times more cytotoxicity in tumor cells compared to normal cells in preclinical studies[192]. Financial Performance - The May 2025 Offering raised approximately $7.5 million through the sale of 1,990,900 shares and warrants, with a combined public offering price of $1.10 per share[129]. - The company has filed a prospectus supplement for the sale of up to 2,534,352 shares of common stock, with a commission rate of up to 3.0% for the sales agent[128]. - General and administrative expenses increased to $11.2 million for Q2 2025, up 662% from $1.5 million in Q2 2024, primarily due to a $9.2 million increase in fair value of contingent consideration related to the VIRAGE Phase 2b clinical trial[209]. - Research and development expenses decreased to $2.0 million for Q2 2025, down 34% from approximately $3.0 million in Q2 2024, attributed to lower clinical trial expenses and manufacturing costs[210]. - The net loss for Q2 2025 was $13.1 million, or ($1.93) per common share, compared to a net loss of $8.3 million, or ($10.72) per common share for Q2 2024[215]. - The accumulated deficit reached $352.4 million as of June 30, 2025, with expectations of continued losses in the foreseeable future[224]. - Cash and cash equivalents totaled $12.1 million as of June 30, 2025, an increase of $0.5 million from December 31, 2024[225]. - The company anticipates needing additional funds for future clinical trials, particularly for larger Phase 3 trials, with no committed sources of financing currently available[226]. - The company raised $6.9 million in net proceeds from a May 2025 offering of 6,818,180 shares of common stock[227]. - As of early August 2025, the company has approximately $9.5 million in cash, which is insufficient to meet its near-term or long-term operational plans[229]. - The net cash used in operating activities for the six months ended June 30, 2025, was $9.5 million, compared to $8.3 million for the same period in 2024, primarily due to the development of VCN-01[232]. - The company expressed substantial doubt about its ability to continue as a going concern without additional capital[229]. - The completion of future Phase 3 and registrational clinical studies will require significant financing or partnerships[230]. 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[127]. - The Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients has completed 2 of 3 cohorts, with positive outcomes leading to the recommendation to proceed to the next cohort[182]. - SYN-020 has demonstrated a favorable safety profile in a Phase 1 clinical study, with no serious adverse events reported and mild treatment-related adverse events[186]. - SYN-020 is anticipated to be produced at a cost of a few hundred dollars per gram at commercial scale, significantly lower than the current market price of $10,000 per gram[184]. - The Phase 1 study of SYN-020 included 32 healthy adult volunteers, with positive safety data indicating it was well-tolerated across all dose levels[186]. - Phase 1 data from SAD and MAD studies support the development of SYN-020 for multiple clinical indications, including NAFLD and diseases associated with aging[188]. - The company is exploring strategic opportunities for SYN-004 and SYN-020 assets, including out-licensing or partnerships[189]. - The THERICEL program is advancing a proprietary A549 suspension cell line to support significant scale-up for VCN-01 manufacturing, expected to reduce costs[194]. - The company has over 135 U.S. and foreign patents and over 50 patents pending, supporting its various programs[196]. Regulatory and Compliance - A Type D meeting with the FDA on December 5, 2024, indicated support for a stand-alone Phase 3 study of VCN-01 with gemcitabine/nab-paclitaxel[155]. - The European Medicines Agency (EMA) provided guidance for a potential Phase 3 study of VCN-01, suggesting it could be supported by positive results from a randomized controlled trial[155]. - The FDA granted Rare Pediatric Drug Designation for VCN-01 for retinoblastoma, potentially allowing for a Priority Review Voucher upon approval[161]. - 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[187]. Goodwill and Liabilities - Goodwill impairment tests are conducted annually, with the last test on October 1, and adjustments made if fair value declines[206]. - Contingent consideration liabilities are measured at estimated fair value at acquisition, with adjustments recorded in the consolidated statements of operations[207]. - Goodwill impairment of $4.0 million was recorded during Q2 2024, reducing the carrying value from $5.5 million to an estimated fair value of $1.5 million[213]. - Other income for Q2 2025 was $74,000, down from $172,000 in Q2 2024, primarily due to a decrease in interest income[214].