On March 16, 2022 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company developing therapeutics designed to treat diseases in areas of high unmet need, reported financial results for the year ended December 31, 2021, and provided a corporate update (Press release, Synthetic Biologics, MAR 16, 2022, View Source [SID1234610277]).

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Recent Developments:

VCN acquisition: Synthetic Biologics completed the acquisition of privately held VCN Biosciences, developer of a novel oncolytic adenovirus (OV) platform designed for intravenous (IV), intravitreal (IVit) and intratumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system.
Pipeline updates: The acquisition of VCN Biosciences transformed Synthetic Biologics’ pipeline with the addition of VCN’s lead clinical-stage drug candidate, VCN-01, as well as preclinical stage VCN-11, which incorporates a proprietary albumin binding domain in the virus shell with the potential to extend the effectiveness of the therapy.
In February of 2022, VCN-01 received Orphan Drug Designation (ODD) from the U.S. FDA for the treatment of retinoblastoma; VCN-01 has previously been granted ODD by the EMA for the treatment of pancreatic cancer.
Regulatory agency recognition builds upon the encouraging clinical data generated to date; VCN-01 has been evaluated in 72 patients across four Phase 1 clinical trials, including patients with pancreatic cancer, head and neck squamous cell carcinoma, colorectal cancer, and retinoblastoma.
Enrollment progressed in the Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic hematopoietic cell transplant (HCT) recipients for the prevention of acute graft-versus-host-disease; and topline data for this cohort are expected in H1 2022.
Completed dosing and follow-up in a Phase 1, placebo-controlled, multiple ascending dose study of SYN-020 in healthy volunteers; SYN-020 was very well tolerated at all doses and patient samples are undergoing pharmacokinetic and pharmacodynamic analyses.
Anticipated Milestones:

VCN-01

Initiation of VCN-01 dosing in an investigator sponsored study of brain tumors at the University of Leeds (H1 2022).
Initiation of VCN-01 dosing in combination with mesothelin-directed CAR-T cells for pancreatic and ovarian cancer in an investigator sponsored study at the University of Pennsylvania (H1 2022).
Initiation of a Phase 2 study of VCN-01 in combination with standard-of-care chemotherapy (gemcitabine/nab-paclitaxel) as a first line therapy in newly diagnosed metastatic PDAC patients (H2 2022).
Initiation of a Phase 2/3 pivotal trial of VCN-01 as either an adjunct to chemotherapy or a potential rescue therapy in pediatric patients with advanced retinoblastoma (early 2023).
VCN-11

Evaluating CMOs for GMP manufacture of VCN-11.
Preclinical studies characterizing VCN-11 are on-going.
SYN-004

Data read out from the first cohort of the SYN-004 study in allo-HCT patients (H1 2022).
SYN-020

Top-line data from the multiple ascending dose study of SYN-020 in healthy volunteers (H1 2022).
Planning for the initiation of a Phase 2a study of SYN-020 (H2 2022).
"2021 was a monumental year for the Company, setting the stage for continued progress and a number of important upcoming milestones in 2022," said Steven A. Shallcross, Chief Executive Officer of Synthetic Biologics. "The recent acquisition of VCN Biosciences positions us at the forefront of oncolytic virus development. VCN’s systemically administered, selectively replicating adenoviruses are designed to break down the tumor stroma, which hides the tumor from the patient’s immune system. In turn, this improves the anti-tumor effect of the oncolytic virus, as well as chemotherapies and immuno-oncology products."

Mr. Shallcross continued, "We are poised for another exciting year ahead as we anticipate the initiation of multiple international studies, including a Phase 2 clinical trial of intravenous VCN-01 in combination with standard-of-care chemotherapy as a first line therapy in newly-diagnosed metastatic PDAC patients, as well as a Phase 2/3 pivotal trial of intravitreal VCN-01 as either an adjunct to chemotherapy or a potential rescue therapy in pediatric patients with advanced retinoblastoma. We do not plan any clinical activities in Eastern Europe; however, we recognize that the war in the Ukraine may have follow-on effects globally that could adversely impact the cost and conduct of our international clinical trials. We will seek to mitigate this impact where possible. In parallel with our development of VCN-01, we continue to advance the clinical development of both SYN-004 and SYN-020 and look forward to providing further updates on key upcoming milestones. We remain well-capitalized through the end of 2023 and highly encouraged by the potential for each of our clinical programs."

Year Ended December 31, 2021 Financial Results
General and administrative expenses increased to $6.5 million for the year ended December 31, 2021, from $5.0 million for the year ended December 31, 2020. This increase of 28.7% is primarily composed of increased consulting and legal costs related to the VCN acquisition, higher insurance costs, audit fees, and public relations expenses. The charge relating to stock-based compensation expense was $0.3 million for the year ended December 31, 2021, compared to $0.3 million for the year ended December 31, 2020.

Research and development expenses increased to $7.8 million for the year ended December 31, 2021, from $5.1 million for the year ended December 31, 2020. This increase of 53% is primarily the result of increased clinical trial expenses as we continued dosing patients in the Phase 1b/2a clinical trial of SYN-004, the dosing of healthy volunteers in the SAD and MAD Phase 1 clinical trials for SYN-020, and by higher indirect program costs for the year ended December 31, 2021, including an increase in manufacturing costs for SYN-020. We anticipate research and development expense to increase as our ongoing clinical trials continue to enroll patients and new patients are enrolled in the VCN-01 clinical trials. Research and development expenses also included a charge relating to non-cash stock-based compensation expense of $76,000 for the year ended December 31, 2021, compared to $66,000 for the year ended December 31, 2020.

Other income was $6,000 for the year ended December 31, 2021, compared to other income of $44,000 for the year ended December 31, 2020. Other income for the year ended December 31, 2021 and 2020 is primarily composed of interest income from investments.

Cash and cash equivalents totaled $67.3 million as of December 31, 2021, an increase of $61.1 million from December 31, 2020.

For further details on Synthetic Biologics’ financial results refer to the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, as filed with the Securities and Exchange Commission.

Conference Call

Synthetic Biologics will host a conference call at 4:30 p.m. ET today to review year end 2021 operational highlights and financial results. Individuals may participate in the live call via telephone by dialing (877) 451-6152 (domestic) or (201) 389-0879 (international) and using the conference ID: 13727535. Participants are asked to dial in 15 minutes before the start of the call to register. Investors and the public can access the live and archived webcast of this call via the "News & Media" section of the company’s website, View Source, under "Events" or by clicking here, for 90 days after the call.