08/11/2026 | Press release | Distributed by Public on 08/11/2026 06:16
MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS.
The following discussion should be read in conjunction with our unaudited condensed consolidated financial statements and notes thereto included in this Quarterly Report on Form 10-Q, and our audited consolidated financial statements and notes thereto for the year ended December 31, 2025 included in our 2025 Form 10-K. This discussion contains forward-looking statements reflecting our current expectations that involve risks and uncertainties. See "Note Regarding Forward-Looking Statements" for a discussion of the uncertainties, risks and assumptions associated with these statements. Our actual results and the timing of events could differ materially from those expressed or implied by the forward-looking statements due to important factors and risks including, but not limited to, those set forth below under "Risk Factors" and elsewhere herein, and those identified under Part I, Item 1A of our 2025 Form 10-K.
Overview
We are a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. As a result of our acquisition of Theriva Biologics, S.L. ("VCN", formerly named VCN Biosciences, S.L.) (the "Acquisition") in March 2022, we transitioned our strategic focus to oncology through the development of VCN's new oncolytic adenovirus platform designed for intravenous and intravitreal delivery to trigger tumor cell death, to improve access of co-administered cancer therapies to the tumor, and to promote a robust and sustained anti-tumor response by the patient's immune system. Our lead product candidate, VCN-01 (zabilugene almadenorepvec), is a clinical stage oncolytic human adenovirus that is modified for tumor-selective replication and to express an enzyme, PH20 hyaluronidase. VCN-01 has been evaluated in a Phase 2b clinical study for the treatment of pancreatic cancer ("VIRAGE"), a Phase 1 clinical study for the treatment of retinoblastoma, as well as various other Phase 1 clinical studies for the treatment of other solid tumors including head and neck squamous cell carcinoma.
VCN-01 has been administered to 144 patients in multiple Company- and Investigator-sponsored Phase 1 clinical trials and the Phase 2b VIRAGE trial, including patients with pancreatic cancer (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian carcinoma (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). VCN-01 has also been made available for compassionate use in retinoblastoma patients, and 2 patients have been treated in this program.
Prior to the Acquisition, our focus was on developing therapeutics designed to treat gastrointestinal (GI) diseases which included our clinical development candidates: (1) SYN-004 (ribaxamase) which is designed to degrade certain commonly used intravenous (IV) beta-lactam antibiotics within the GI tract to prevent microbiome damage, thereby preventing overgrowth and infection by pathogenic organisms such as Clostridioides difficile infection (CDI) and vancomycin resistant Enterococci (VRE), and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients, and (2) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases, which we have out-licensed as described below.
Additionally, as part of our strategic transformation into an oncology focused company, we are exploring value creation options for our SYN-004 asset, including out-licensing or partnering as we do not intend to continue clinical trial activities or further develop SYN-004 without receipt of grant funding or funding through a partnership or other collaboration.
Financial Developments
During the six months ended June 30, 2026, we sold 10,204,319 shares of our Common Stock pursuant to the Amended and Restated At The Market Issuance Sales Agreement, dated February 9, 2021, as amended by Amendment No. 1 thereto, dated May 3, 2021, as further amended by Amendment No. 2 thereto, dated May 2, 2024 (the "ATM Sales Agreement") and received net proceeds of approximately $2.3 million. On January 22, 2026, we received $1.6 million for the 2024 Research and Development rebate program sponsored by the Spanish government. The program provides for reimbursement of certain expenses incurred in research and development efforts we incur in Spain. The reimbursements can be through either tax credits or direct refunds.
Our Current Product Pipeline
*Based on management's current beliefs and expectations
aGVHD acute graft-versus-host disease. allo-HCT allogeneic hematopoietic cell transplant. IV intravenous. IVit intravitreal.
¹†Phase 1b/2a study remains open, but enrollment paused after completing 2 of 3 scheduled cohorts; conduct of the third cohort is contingent on grant funding or a partnership.
Recent Clinical Developments
Phase 2 Trial of intravenous VCN-01 with nab-paclitaxel plus gemcitabine in patients with PDAC
On March 23, 2026, we announced a positive End-of-Phase 2 Meeting with U.S. FDA Regarding the Design of a Phase 3 Trial of VCN-01 in Metastatic Pancreatic Ductal Adenocarcinoma.
The FDA provided general agreement with our proposed design for a Phase 3 clinical trial, which closely tracks the design of the successful VIRAGE Phase 2 trial. As announced in 2025, the VIRAGE trial met its primary endpoints, with metastatic PDAC patients receiving VCN-01 with SoC chemotherapy having improved overall survival (OS), progression free survival (PFS) and Duration of Response (DoR) compared to SoC chemotherapy alone. Greater improvements in OS and PFS were observed in patients who received two doses of VCN-01, leading Theriva to plan the Phase 3 trial to include repeat dosing and an adaptive design aimed to optimize the trial's timelines and outcomes.
Consistent with scientific advice previously received from the Committee for Medicinal Products for Human Use (CHMP) of the EMA, the FDA advised that a potential biologics licensing application (BLA) for VCN-01 in metastatic PDAC could be supported by our proposed Phase 3 clinical trial (if successful) comprising a single, high-quality, randomized, double-blinded, study comparing VCN-01 plus gemcitabine/nab-paclitaxel SoC to gemcitabine/nab-paclitaxel SoC plus placebo. The FDA further agreed on the proposed dosing of VCN-01 and gemcitabine/nab-paclitaxel in repeated "macrocycles" (enabling more than 2 doses of VCN-01 to be administered in the Phase 3 trial), the proposed inclusion/exclusion criteria, the primary endpoint (overall survival), key secondary endpoints (including progression free survival), and the use of an adaptive design. The FDA also clarified statistical expectations regarding the proposed interim analyses and the quality of data required for potential sample size re-estimation or a demonstration of early efficacy.
On April 17, 2026, we announced the presentation of additional data from the VIRAGE Phase 2b Clinical Trial at American Association for Cancer Research (AACR) 2026 Annual Meeting. Tumor response, biomarker, and subgroup analyses from the VIRAGE Phase 2b clinical trial support a VCN-01 immune-mediated mode of action and demonstrate improved outcomes in VCN-01 treated patients across multiple subgroups, including patients with liver metastases were presented in a poster session by Dr. Manuel Hidalgo (NYU Langone Health Perlmutter Cancer Center, New York) in San Diego, California on April 20, 2026.
On July 7, 2026, we announced that the Spanish Agency of Medicines and Medical Devices ("AEMPS") has authorized us to initiate the VIRAGE2 clinical trial, entitled "A Phase IIa, single-arm, single-center, open-label, proof-of-concept trial evaluating increased
frequency dosing of zabilugene almadenorepvec (VCN-01) in combination with gemcitabine/nab-paclitaxel in patients with newly-diagnosed metastatic pancreatic cancer".
The VIRAGE2 trial builds on the results of the 112-patient VIRAGE Phase 2b clinical trial evaluating VCN-01 in treatment naïve metastatic pancreatic ductal adenocarcinoma ("PDAC") patients receiving gemcitabine/nab-paclitaxel standard-of-care ("SoC") chemotherapy. In the VIRAGE trial, patients who received 2 doses of VCN-01 administered 3 months apart had significantly improved overall survival, progression free survival, and duration of response compared to patients treated with only one dose of VCN-01 or with SoC chemotherapy alone. As previously reported, both the EMA and the FDA recognized the improved survival in the group treated with 2 doses of VCN-01, and raised the possibility of more frequent repeated dosing of VCN-01 in combination with SoC chemotherapy to potentially improve clinical outcomes. The VIRAGE2 trial is designed to evaluate the feasibility of administering at least 3 doses of VCN-01 given 2 months apart in combination with SoC chemotherapy. Results from this trial will inform the VCN-01 dosing regimen for potential evaluation in a future pivotal Phase 3 clinical trial.
Phase 1 Trial of intravenous VCN-01 in Combination with Durvalumab in Subjects with Recurrent/ Metastatic SCCHN
On June 11, 2026, we announced that clinical and translational results from VCN-01's Phase 1 clinical trial in Head & Neck Squamous Cell Carcinoma ("HNSCC") were recently published on-line first in the journal Clinical Cancer Research.
The trial enrolled 20 adult patients with refractory or metastatic HNSCC, whose disease progressed despite previous therapies, including anti-PD-(L)1 immune checkpoint inhibitors. Six patients were enrolled into the concomitant Arm I LD of the study and were administered IV low dose VCN-01 (3.3E12 virus particles; LD) four hours prior to a fixed IV dose of durvalumab (1500 mg/q4w). Eight patients were enrolled into the sequential Arm II LD of the study, receiving low dose IV VCN-01 14 days prior to IV durvalumab administration. An additional six patients were entered into Arm II HD, receiving high dose IV VCN-01 (1.0E13 virus particles; HD) 14 days prior to IV durvalumab administration.
| ● | Median progression-free survival (PFS) was 1.6 months in Arm I LD, 3.7 months in Arm II LD, and 2.1 months in Arm II HD. |
| ● | Median overall survival (OS) was 10.3 months in Arm I LD, 15.5 months in Arm II LD, and 17.3 months in Arm II HD. |
| ● | Circulating levels of the stroma-degrading hyaluronidase enzyme PH20 (expressed during selective VCN-01 intratumoral replication) increased significantly after VCN-01 administration in all tested patients, peaking on day 3-8 for most patients and detectable until day 28 in 11 of 12 patients. |
| ● | Similarly, VCN-01 viral genome levels detected in patient blood exhibited an initial peak immediately following administration and a secondary peak on day 3-8, consistent with continued viral replication in tumors followed by a return of virus to circulation. |
| ● | Upregulation of CD8 and IDO was observed in tumor biopsy samples, implying increased tumor infiltration with activated cytotoxic T cells - historically associated with increased HNSCC patient survival. Diminished levels of FoxP3, CD25, and CTLA4 were also observed, consistent with a reduction in tumor Tregs and inhibition of tumor immunosuppression. |
| ● | Tumor biopsies revealed upregulation of PD-1 and PD-L1 in most patients following VCN-01 administration that correlated with patient survival, suggesting that immune system activity and heightened PD-L1 expression in tumors contributed to the improved outcomes from VCN-01 and durvalumab combination. |
| ● | Transcriptomic and radiomic analyses showed changes in the extracellular matrix and increased tumor perfusion. |
The collective pharmacokinetic, tissue biopsy, radiomic and transcriptomic results support the proposed VCN-01 stroma-degrading and immune enhancing modes-of-action, resensitizing refractory tumors to durvalumab. As described in the publication, the prolonged OS observed with sequential delivery of VCN-01 followed by durvalumab suggests a potential clinical benefit in this heavily pretreated HNSCC population, These findings support further clinical development of VCN-01 with immune checkpoint inhibitors or other immune modulating anticancer therapies in HNSCC and potentially other cancer indications.
Intravitreal VCN-01 with topotecan for the treatment of patients with retinoblastoma
As previously reported, an investigator-sponsored Phase 1 study of VCN-01 in refractory retinoblastoma patients facing imminent enucleation was completed in the first half of 2024. Patients received two intravitreal administrations of VCN-01 on days 1 and 15. Based on the study results, it was concluded that VCN-01 was well tolerated and demonstrated an acceptable adverse event profile. Three patients presented a complete response. The safety and clinical outcomes of the Phase 1 study of VCN-01 were presented by the Principal Investigator (the "PI") of the study, Dr. Jaume Català-Mora, Pediatric Ophthalmologist, Sant Joan de Déu-Barcelona Children's Hospital, on February 3, 2026 at an invited session at the 41st Asia-Pacific Academy of Ophthalmology (APAO) Congress in Hong Kong (China).
We have been granted Rare Pediatric Drug Designation ("RPDD") for VCN-01 for the treatment of retinoblastoma. If a Biologics License Application (BLA) for VCN-01 for the treatment of retinoblastoma is approved by the FDA by September 30, 2029, we may be eligible to receive a Priority Review Voucher, which can be monetized. VCN-01 has also received orphan drug designation from the FDA and Orphan Medicinal Product Designation from the European Commission for the treatment of retinoblastoma.
In the first quarter of 2026, we made VCN-01 available to investigators at Hospital Sant Joan de Déu, Barcelona, for compassionate use in treating patients with retinoblastoma. Two patients have been treated with intravitreal VCN-01 in combination with intravitreal topotecan and patients are being followed by the treating physicians. We expect that outcomes from these compassionate use patients will provide valuable information on the feasibility and tolerability of this combination for use in a potential Phase 2/3 clinical trial.
As previously disclosed, preclinical data demonstrate potential antitumor synergy between VCN-01 and topoisomerase I inhibitors, such as topotecan. We believe that intravitreal coadministration of VCN-01 with topotecan may provide a new treatment option for children with retinoblastoma with vitreous seeds that are refractory/resistant to the use of current intravitreal chemotherapy, which remains an unmet medical need in patients with this ultra rare patient population. Extensive discussions with clinicians and key opinion leaders have enabled the design a Phase 2/3 clinical trial protocol for the VCN-01 + topotecan combination in this ultra rare patient population, and we plan to discuss the proposed protocol with the FDA in Q3 2026.
We anticipate that the cost for the Phase 2/3 trial would be approximately $9 million (including chemistry, manufacturing and controls). If a protocol is ultimately submitted to, and agreed by, the FDA, we expect the first patient to be enrolled in January 2027, with rolling BLA submissions expected to be made in 2029 (if successful), targeting potential approval of the BLA prior to the current legislative deadline of September 30, 2029 to be eligible to receive a Priority Review Voucher. We have revised our estimate of the Phase 2/3 trial cost downward from our estimate of $12 million, as disclosed in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, based on a reduced requirement for clinical drug product manufacture , and have adjusted the anticipated first-patient enrollment from December 2026 to January 2027.
Critical Accounting Estimates
The preparation of our consolidated financial statements in accordance with accounting principles generally accepted in the United States of America ("U.S. GAAP") which requires the use of estimates, judgments and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the consolidated financial statements, and the reported amounts of expenses in the periods presented. We believe that the accounting estimates employed are appropriate and resulting balances are reasonable; however, due to inherent uncertainties in making estimates, actual results may differ from the original estimates, requiring adjustments to these balances in future periods.
There are accounting policies, each of which requires significant judgments and estimates on the part of management, that we believe are significant to the presentation of our consolidated financial statements. The most significant accounting estimates relate to valuation of IPR&D and contingent consideration.
IPR&D
IPR&D assets are considered to be indefinite-lived until the completion or abandonment of the associated research and development projects. IPR&D assets represent the fair value assigned to technologies that we acquire, which at the time of acquisition have not reached technological feasibility and have no alternative future use. IPR&D is capitalized at its fair value as an indefinite-lived intangible asset, and any development costs incurred after the acquisition are expensed as incurred. During the period that the assets are considered indefinite-lived, they are tested for impairment on an annual basis, or more frequently if we become aware of any events occurring or
changes in circumstances that indicate that the fair value of the IPR&D assets are less than their carrying amounts. If and when development is complete, which generally occurs upon regulatory approval and the ability to commercialize products associated with the IPR&D assets, these assets are then deemed definite-lived and are amortized based on their estimated useful lives at that point in time. If development is terminated or abandoned, we may have a full or partial impairment charge related to the IPR&D assets, calculated as the excess of carrying value of the IPR&D assets over fair value.
We conduct an impairment test of IPR&D on an annual basis as of October 1 of each year and will also conduct tests if events occur or circumstances change that would, more likely than not, reduce our fair value below our net equity value.
Contingent Consideration
Consideration paid in a business combination may include potential future payments that are contingent upon the acquired business achieving certain milestones in the future ("contingent consideration"). Contingent consideration liabilities are measured at their estimated fair value as of the date of acquisition, with subsequent changes in fair value recorded in the consolidated statements of operations. We estimate the fair value of the contingent consideration as of the acquisition date using the estimated future cash outflows based on the probability of meeting future milestones. The milestone payments will be made upon the achievement of clinical and commercialization milestones. Subsequent to the date of acquisition, we reassess the actual consideration earned and the probability-weighted future earn-out payments at each balance sheet date. Any adjustment to the contingent consideration liability will be recorded in the consolidated statements of operations. Contingent consideration liabilities expected to be settled within 12 months after the balance sheet date are presented in current liabilities, with the non-current portion recorded under long term liabilities in the consolidated balance sheets.
Results of Operations
Three Months Ended June 30, 2026 and 2025
General and Administrative Expenses
General and administrative expenses decreased to $2.0 million for the three months ended June 30, 2026, from $11.2 million for the three months ended June 30, 2025. This decrease of 82% is primarily comprised of the prior year increase in fair value of the contingent consideration adjustment of $9.2 million due to the VIRAGE Phase 2b clinical trial of VCN-01 in PDAC achieving its primary survival and safety endpoints, offset set by current year increase in legal fees. The charge related to stock-based compensation expense was $110,000 for the three months ended June 30, 2026, compared to $97,000 for the three months ended June 30, 2025.
Research and Development Expenses
Research and development expenses decreased to $1.3 million for the three months ended June 30, 2026, from approximately $2.0 million for the three months ended June 30, 2025. This decrease of 35% is primarily the result of lower indirect cost related to compensation expense and lower direct clinical trial expenses related to our Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients and lower expenses related to SYN-020, offset by higher direct expenses related to VCN-01 manufacturing activities and expenses associated with the planning for the Phase 2a study in metastatic PDAC patients evaluating more frequent VCN-01 dosing for a longer period. We anticipate research and development expense to decrease in the near future until we commence additional clinical trials as we focus on regulatory interactions regarding a proposed pivotal clinical trial of VCN-01 in retinoblastoma, conduct exploratory VCN-01 manufacturing scale-up activities, and continue limited preclinical studies supporting VCN-01 and VCN-12, the first candidate from our VCN-X discovery program. The charge related to stock-based compensation expense was $25,000 for the three months ended June 30, 2026, compared to $76,000 for the three months ended June 30, 2025.
The following table sets forth our research and development expenses directly related to our product candidates for the three months ended June 30, 2026 and 2025. These direct expenses were external costs associated with preclinical studies and clinical trials. Indirect research and development expenses related to employee costs, facilities, stock-based compensation and research and development support services that are not directly allocated to specific product candidates.
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June 30, |
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June 30, |
||
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Therapeutic Areas |
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2026 |
|
2025 |
||
|
VCN-01 (zabilugene almadenorepvec) |
|
$ |
895 |
|
$ |
780 |
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SYN-004 (ribaxamase) |
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29 |
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|
105 |
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SYN-020 |
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- |
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|
89 |
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Other therapeutic areas |
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120 |
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|
119 |
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|
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|
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Total direct costs |
|
1,044 |
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|
1,093 |
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Total indirect costs |
|
224 |
|
|
860 |
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|
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|
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|
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|
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Total Research and Development |
|
$ |
1,268 |
|
$ |
1,953 |
Other Income/Expense
Other income was $78,000 for the three months ended June 30, 2026, compared to other income of $74,000 for the three months ended June 30, 2025. Other income for the three months ended June 30, 2026 is comprised of interest income of $79,000 and an exchange loss of $1,000. Other income for the three months ended June 30, 2025 is comprised of interest income of $54,000 and an exchange gain of $20,000.
Net Loss
Our net loss for the three months ended June 30, 2026 was $3.2 million, or ($0.07) per common share, compared to $13.1 million, or ($1.93) per common share, for the three months ended June 30, 2025.
Six Months Ended June 30, 2026 and 2025
General and Administrative Expenses
General and administrative expenses decreased to $4.1 million for the six months ended June 30, 2026, from $12.6 million for the six months ended June 30, 2025. This decrease of 68% is primarily comprised of the of the prior year increase in fair value of the contingent consideration adjustment of $9.2 million due to the VIRAGE Phase 2b clinical trial of VCN-01 in PDAC achieving its primary survival and safety endpoints, offset set by current year increase in legal fees. The charge related to stock-based compensation expense was $220,000 for the six months ended June 30, 2026, compared to $151,000 for the six months ended June 30, 2025.
Research and Development Expenses
Research and development expenses decreased to $1.6 million for the six months ended June 30, 2026, from approximately $4.9 million for the six months ended June 30, 2025. This decrease of 67% is primarily the result of lower direct clinical trial expenses related to our VIRAGE Phase 2b clinical trial of VCN-01 in PDAC, lower indirect cost related to decreased VCN-01 manufacturing costs and lower clinical trial expenses related to our Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, offset by higher patent expenses related to SYN-020. We anticipate research and development expense to decrease in the near future until we commence additional clinical trials as we focus on regulatory interactions regarding a proposed pivotal clinical trial of VCN-01 in retinoblastoma, conduct exploratory VCN-01 manufacturing scale-up activities, and continue limited preclinical studies supporting VCN-01 and VCN-12, the first candidate from our VCN-X discovery program. The charge related to stock-based compensation expense was $49,000 for the six months ended June 30, 2026, compared to $122,000 related to stock-based compensation expense for the six months ended June 30, 2025.
The following table sets forth our research and development expenses directly related to our product candidates for the six months ended June 30, 2026 and 2025. These direct expenses were external costs associated with preclinical studies and clinical trials. Indirect research and development expenses related to employee costs, facilities, stock-based compensation and research and development support services that are not directly allocated to specific product candidates.
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June 30, |
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June 30, |
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Therapeutic Areas |
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2026 |
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2025 |
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VCN-01 (zabilugene almadenorepvec) |
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$ |
872 |
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$ |
2,726 |
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SYN-004 (ribaxamase) |
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68 |
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167 |
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SYN-020 |
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73 |
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150 |
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Other therapeutic areas |
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235 |
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205 |
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Total direct costs |
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1,248 |
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3,248 |
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Total indirect costs |
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375 |
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1,673 |
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Total Research and Development |
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$ |
1,623 |
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$ |
4,921 |
Other Income/Expense
Other income was $161,000 for the six months ended June 30, 2026 compared to other income of $167,000 for the six months ended June 30, 2025. Other income for the six months ended June 30, 2026 is primarily comprised of interest income of $161,000. Other income for the six months ended June 30, 2025 is primarily comprised of interest income of $150,000 and an exchange gain of $17,000.
Net Loss Attributable to Common Stockholders
Our net loss attributable to common stockholders was approximately $5.3 million, or ($0.12) per basic and diluted common share for the six months ended June 30, 2026, compared to a net loss of approximately $17.4 million, or ($3.64) per basic common share and diluted common share for the six months ended June 30, 2025.
Liquidity and Capital Resources
Historically, we have financed our operations primarily through public and private sales of our securities, and we expect to continue to seek and obtain additional capital in a similar manner. During the year ended December 31, 2025, our primary sources of cash were the approximately $6.8 million in net proceeds received from sales of our Common Stock under the ATM Sales Agreement, approximately $3.9 million in net proceeds from the exercise of existing warrants by holders pursuant to a warrant inducement agreement, the $1.7 million received for the Research and Development rebate program, $1.4 million for the THERICEL project loan from the National Knowledge Transfer Program of the Spanish government's Ministry of Science and, in May 2025, we closed our May 2025 Offering of 6,818,180 shares of Common Stock (or pre-funded warrants in lieu thereof) in combination with accompanying common stock purchase warrants to purchase an aggregate of 6,818,180 shares of our Common Stock for gross proceeds of $7.5 million (net proceeds of $6.7 million, after deducting underwriting discounts and expenses). During the six months ended June 30, 2026, the primary source of cash was from the $300,000 up-front payment received for the out-licensing agreement of SYN-020, $1.6 million received for the Research and Development rebate program and $2.3 million in net proceeds received from sales of our Common Stock under the ATM Sales Agreement.
Under the terms of the license agreement we entered into with Rasayana Therapeutics, Inc. ("Rasayana") on February 18, 2026 (the "Rasayana License Agreement"), pursuant to which we granted Rasayana an exclusive worldwide license with the right to grant sublicenses to Research, Develop, Manufacture and Commercialize (as such terms are defined in the Rasayana License Agreement) any Product (as such term is defined in the Rasayana License Agreement), which includes SYN-020, an oral formulation of the recombinant intestinal alkaline phosphatase enzyme, comprising, containing, or covered by the Licensed IP (as such term is defined in the Rasayana License Agreement) and/or devised, developed, or produced using the Licensed IP, we received an upfront payment of $300,000 and we are entitled to receive from Rasayana: (i) development milestone payments of up to an aggregate of $16,000,000; (ii) sales milestone payments of up to an aggregate of $22,000,000 upon achievement of certain development and net sales milestones with respect to Products; and (iii) during the Royalty Term (as such term is defined in the Rasayana License Agreement), tiered royalties ranging from low to mid single digits on net sales of a Product. We will also be entitled to receive a certain percentage of any Sublicense Revenue (as such term is defined in the Rasayana License Agreement) received by Rasayana or its affiliates. However, we do not anticipate receiving any funds due pursuant to the Rasayana License Agreement within the next twelve months.
As of June 30, 2026, we have a significant accumulated deficit, and with the exception of the three months ended June 30, 2010 and the three months ended December 31, 2017, we have experienced significant losses and incurred negative cash flows since inception. We have incurred an accumulated deficit of $364 million as of June 30, 2026, and expect to continue to incur losses in the foreseeable future
with the recognition of revenue being contingent on successful phase 3 clinical trials and requisite approvals by the FDA or foreign equivalents and the success of our licensee.
Our cash and cash equivalents totaled $11.3 million as of June 30, 2026, a decrease of $1.7 million from December 31, 2025. During the year ended December 31, 2025 and six months ended June 30, 2026, the primary use of cash was for working capital requirements and operating activities, which resulted in a net loss of $23.7 million and $5.3 million for the year ended December 31, 2025 and the six months ended June 30, 2026, respectively.
With our cash position of $9.6 million as of early August 2026, we believe we will be able to fund our operations into the first quarter of 2027; however, the current cash will only be sufficient to conduct certain limited clinical, regulatory, manufacturing, and preclinical activities as described herein and our cash runway could differ materially from our expectations based on various factors, many of which are out of our control. We continue to experience operating losses and face significant uncertainties related to our business model, market conditions, clinical trial outcomes, FDA review timelines and strategic initiatives. These factors raise substantial doubt about our ability to continue as a going concern beyond the next twelve months without additional capital or other strategic actions. Management has developed plans intended to mitigate these uncertainties, including pursuing strategic collaborations, securing additional financing, prioritizing key development programs and pursuing other strategic alternatives, that may include a business combination, merger or reverse merger. There can be no assurance that such actions will be sufficient to alleviate the going concern uncertainty.
We are devoting substantial time and resources to the strategic review. Despite devoting significant efforts to identify and evaluate potential strategic alternatives, there can be no assurance that this strategic review process will result in us pursuing any transaction or that any transaction, if pursued, will be completed on attractive terms or at all. We have not set a definitive timeline for completion of this strategic review process, and our board of directors has not approved a definitive course of action. Additionally, there can be no assurances that any particular course of action, business arrangement or transaction, or series of transactions, will be pursued, successfully consummated or lead to increased stockholder value.
Based on our current plans, we expect that our cash and cash equivalents will be sufficient to cover overhead costs, commence an approved Phase 2a study evaluating more frequent VCN-01 dosing for a longer period, exploratory VCN-01 manufacturing scale-up activities, regulatory interactions regarding a proposed pivotal clinical trial of VCN-01 in retinoblastoma, and limited preclinical studies supporting VCN-01 and VCN-12, the first candidate from our VCN-X discovery program. We believe that the cash will also be sufficient to fund our committed obligations under the terms of the Share Purchase Agreement entered into in connection with the Acquisition (the "Purchase Agreement"), however, payment of the $5.0 million owed to Grifols Innovation and New Technologies Limited will significantly deplete our cash and cash equivalents, which could materially and adversely affect our liquidity and limit our ability to fund operations or meet other financial obligations. Our current cash will not be sufficient for additional trials of VCN-01 (other than the Phase 2a study evaluating more frequent VCN-01 dosing for a longer period), or additional trials of SYN-004 (ribaxamase), or to complete the last cohort of the Phase 1b/2a clinical trial of SYN-004, which are expected to require significant cash expenditures. Following the completion of our ongoing Phase 1 and Phase 2b clinical trials for VCN-01, and limited preclinical studies supporting VCN-01 and our discovery initiatives, we will need to obtain additional funds for future clinical trials. We anticipate that our future clinical trials will be much larger in size and require larger cash expenditures than the aforementioned clinical programs. We do not have any committed sources of financing for future clinical trials at this time, and it is uncertain whether additional funding will be available when we need it on terms that will be acceptable to us, or at all. Management believes its plan, which is focused on the advancement of VCN-01, will allow us to meet our financial obligations, further advance key products, and maintain our planned operations. Based upon our current available funding and our focus on our clinical development of VCN-01 we do not anticipate that we will fund the last cohort of the Phase 1b/2a clinical trial of SYN-004 and enrollment in this cohort will not commence unless we obtain grant funding, or find a licensee or partner for the SYN-004 development program. However, the amount of additional capital needed by us will also depend upon the costs to advance our VCN-01 clinical programs.
We are actively pursuing additional equity or debt financing opportunities, in the form of either a private placement or a public offering and have been engaged in ongoing discussions with strategic institutional investors and investment banks with respect to such possible offerings as well as other strategic alternatives. Potential sources of financing that we are pursuing include strategic relationships, licensing arrangements, public or private sales of our equity or debt and other sources. Such additional financing opportunities might not be available to us when and if needed, on acceptable terms or at all. We may attempt to utilize the ATM Sales Agreement or seek to raise additional capital in other financing transactions, neither of which is guaranteed. We cannot assure that we will meet the requirements for use of the ATM Sales Agreement especially in light of the fact that we are currently limited by rules of the SEC as to the number of shares of Common Stock that we can sell pursuant to the ATM Sales Agreement due to the market value of our Common Stock held by non-affiliates. Even if we meet the requirements for use of the ATM Sales Agreement, there can be no assurance that we
will be able to raise funds through the sale of shares of Common Stock through the ATM Sales Agreement. Additionally, we may seek to access the public or private equity markets when conditions are favorable due to our long-term capital requirements. If we are not able to obtain additional capital (which is not assured at this time), our long-term business plan may not be accomplished, and we may be forced to cease certain development activities. More specifically, the completion of any later stage clinical trial will require significant financing or a significant partnership.
If we raise funds by selling additional shares of Common Stock or other securities convertible into Common Stock, the ownership interest of our existing stockholders will be diluted. If we are not able to obtain funding for future clinical trials when needed, we will not be able to carry out our business plan and we will be forced to delay the initiation of future clinical trials until such time as we obtain adequate financing or we are able to secure a strategic collaboration and may need to abandon some of our development programs, cease operations, sell or otherwise liquidate our assets or reorganize the Company, or complete a combination of the foregoing.
Our ability to continue as a going concern is dependent upon our ability to obtain additional equity or debt financing, attain further operating efficiencies, reduce expenditures, and, ultimately, to generate revenue. Our notes to the consolidated financial statements included in this Quarterly Report contain an explanatory paragraph referring to our recurring and continuing losses from operations and expressing substantial doubt in our ability to continue as a going concern without additional capital becoming available. We cannot provide any assurance that we will be able to obtain the required funding to achieve our current business plan, obtain the required regulatory approvals for our product candidates or complete additional corporate partnering or acquisition transactions in order to commercialize such product candidates once regulatory approval is received. If we fail to obtain additional funding for our clinical trials, whether through the sale of securities or a partner or collaborator, and otherwise when needed, we will not be able to execute our business plan as planned and will be forced to cease certain development activities (including initiation of planned clinical trials) until funding is received or we are able to secure a strategic collaboration and may need to abandon some of our development programs, cease operations, sell or otherwise liquidate our assets or reorganize the Company, engage in a strategic alternative or complete a combination of the foregoing. There can be no assurance that we will be able to successfully effect any of the foregoing.
Cash Flows
The following table summarizes our cash flows for the periods presented (in thousands):
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|
|
|
|
|
|
|
|
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Six Months Ended June 30, |
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|
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2026 |
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2025 |
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Cash used in operating activities |
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$ |
(5,541) |
|
$ |
(9,468) |
|
Cash used in investing activities |
|
(3) |
|
(16) |
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Cash provided by financing activities |
|
3,852 |
|
9,883 |
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Effects of exchange rate changes on cash and cash equivalents |
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|
(29) |
|
|
62 |
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Net increase (decrease) in cash |
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(1,721) |
|
461 |
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Cash, cash equivalents and restricted cash, beginning of period |
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13,102 |
|
11,705 |
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Cash, cash equivalents and restricted cash, end of period |
|
$ |
11,381 |
|
$ |
12,166 |
Cash Used in Operating Activities
Net cash used in operating activities was $5.5 million and $9.5 million during the six months ended June 30, 2026 and 2025, respectively, which was primarily due to the use of funds in our operations related to the development of VCN-01. Cash used in operating activities for the six months ended June 30, 2026 decreased compared to the same period in 2025 due primarily to prior year contingent consideration adjustment, and lower research and development expenses due to completion of trials offset by higher legal and investor relation costs, which led to a decrease in net loss.
Cash Used in Investing Activities
Cash used in investing activities during the six months ended June 30, 2026 and 2025 was $3,000 and $16,000, respectively, for equipment purchases.
Cash Provided by Financing Activities
Cash provided by financing activities during the six months ended June 30, 2026 included at the market offering proceeds of $2.3 million from sales of 10,204,319 shares of our Common Stock under the ATM Sales Agreement, and $1.6 million received for the research and development tax credit offset by payments of loans payable of $60,000. Cash provided by financing activities during the six months ended June 30, 2025 included $1.8 million received for the research and development tax credit, $1.4 million in loan proceeds from the THERICEL project loan and $6.9 million in net proceeds from the sale of Common Stock, offset by payments of loans in the amount of $67,000.
Off-Balance Sheet Arrangements
During the three months ended June 30, 2026, we did not have, and we do not currently have, any off-balance sheet arrangements, as defined under SEC rules.
Contractual Obligations
Leases
At the inception of a contract we determine if the arrangement is, or contains, a lease. Right of use ("ROU") assets represent our right to use an underlying asset for the lease term and lease liabilities represent our obligation to make lease payments arising from the lease. ROU assets and liabilities are recognized at the commencement date based on the present value of lease payments over the lease term.
We have made certain accounting policy elections whereby we (i) do not recognize ROU assets or lease liabilities for short-term leases (those with original terms of 12-months or less) and (ii) combine lease and non-lease elements of our operating leases. As of June 30, 2026, we did not have any material finance leases.