PDS Biotechnology(PDSB) - 2019 Q4 - Annual Report

PART I Business Overview PDS Biotechnology Corporation is a clinical-stage biopharmaceutical company developing next-generation cancer immunotherapies using its proprietary Versamune® platform, with lead product PDS0101 showing promising early clinical results and planned Phase II studies - PDS Biotechnology is a clinical-stage biopharmaceutical company leveraging its proprietary Versamune® platform to develop multifunctional cancer immunotherapies[14] - The lead product PDS0101 (Versamune®+HPV antigen) demonstrated strong HPV-specific CD8+ T-cell induction in Phase I clinical trials, with complete regression of pre-cancerous lesions in 6 out of 10 evaluable patients within 1-3 months post-treatment, and no recurrence over a 2-year follow-up period[15][17][56][59] - The Versamune® platform is designed to stimulate both killer and helper T-cell responses and modify the tumor microenvironment, making it an ideal complement to checkpoint inhibitors to enhance their efficacy[14][18] PDS0101 Clinical Development Plan (Initiating H1 2020) | PDS Biotech Product | Indication | Partner | Combination Product | Study Size | | :--- | :--- | :--- | :--- | :--- | | PDS0101 (HPV16) | First-line Recurrent/Metastatic Head and Neck Cancer | Merck & Co. | KEYTRUDA® (Standard of Care) | 96 Subjects, 20 US Sites | | PDS0101 (HPV16) | Advanced HPV-Associated Malignancies | NCI | EMD Serono's M7824 and NHS-IL12 | 29 Subjects, 1 US Site (NCI) | | PDS0101 (HPV16) | Advanced, Locally Advanced Cervical Cancer (Stage IIb-IVa) | Leading Cancer Research Institution | Chemoradiation (Standard of Care) | 35 Subjects, 1 US Site | Versamune® Pipeline (Preclinical Stage) | Product | Indication | Partner | Combination Therapy | Status | | :--- | :--- | :--- | :--- | :--- | | PDS0102 (TARP) | Prostate and Breast Cancer | No Industry Partner | Checkpoint Inhibitor | Preclinical Studies Ongoing | | PDS0103 (MUC-1) | Ovarian, Colorectal, Lung, Breast Cancer | No Industry Partner | Checkpoint Inhibitor | Preclinical Studies Ongoing | | PDS0104 (Melanoma) | Melanoma | No Industry Partner | Checkpoint Inhibitor | Preclinical Studies Ongoing | - The company has not generated any product revenue since its inception in 2005, with operating capital primarily sourced from common stock issuance[26] Company Overview PDS Biotechnology is a clinical-stage biopharmaceutical company leveraging its proprietary Versamune® platform to develop multifunctional cancer immunotherapies - PDS Biotechnology is a clinical-stage biopharmaceutical company leveraging its proprietary Versamune® platform to develop multifunctional cancer immunotherapies[14] - The lead product PDS0101 (Versamune®+HPV antigen) demonstrated strong HPV-specific CD8+ T-cell induction in Phase I clinical trials, with complete regression of pre-cancerous lesions in 6 out of 10 evaluable patients within 1-3 months post-treatment, and no recurrence over a 2-year follow-up period[15][17][56][59] - The company was incorporated as Edge Therapeutics, Inc. on January 22, 2009, and completed a reverse merger with Private PDS on March 15, 2019, subsequently changing its name to PDS Biotechnology Corporation[24][25] - The company has not generated any product revenue since its inception in 2005, with operating capital primarily sourced from common stock issuance[26] Commercial Strategy The company's mission is to develop effective and safe immuno-oncology products across a broad range of cancer types using the Versamune® platform - The company's mission is to develop effective and safe immuno-oncology products covering a broad range of cancer types using the Versamune® platform[29] - Rapidly advance the lead product PDS0101 into three proof-of-concept Phase II clinical studies[36] - Build a Versamune®-based immuno-oncology product pipeline through continued development of PDS0102, PDS0103, and PDS0104 programs[36] - Commercialize proprietary products through partnerships or by establishing targeted sales teams in the US, Canada, and Europe[36] - Maintain high barriers to entry for product candidates and markets through patents and proprietary technology[36] Cancer Immunotherapy There is a significant unmet need in cancer immunotherapy to safely induce sufficient numbers of highly active CD8+ T-cells and modify the tumor microenvironment - There is a significant unmet need in cancer immunotherapy to safely induce sufficient numbers of highly active CD8+ T-cells in vivo and modify the tumor microenvironment to limit its immune tolerance[33] - Company data indicate that the Versamune® platform effectively promotes both key immunotherapeutic properties, leading to potent antigen-specific CD8+ T-cell induction and lesion regression[33] - Induce high levels of tumor-infiltrating CD8+ T-cells in vivo[37] - Further modify the tumor microenvironment by activating complementary immune mechanisms[37] - Provide viable clinical application without significantly increasing combination toxicity compared to single components[37] Limitations of Current Immunotherapies Current immunotherapies face limitations including inefficient antigen uptake, insufficient T-cell activation, and high manufacturing complexity and cost - Inefficient antigen uptake by dendritic cells[40] - Insufficient antigen cross-presentation and killer (CD8+) T-cell priming[41] - Inadequate immune activation, lacking necessary chemokine and cytokine induction[42] - Difficulty overcoming immunosuppressive mechanisms within tumors[43] - Most commercially available immunotherapy formulations have high complexity and cost of manufacturing[44] - Versamune® has demonstrated the ability to promote dendritic cell antigen uptake, present antigens via MHC Class I and II pathways, activate Type I interferon signaling, and modify the tumor microenvironment, while its synthetic lipid nanoparticle manufacturing process is simpler and less costly[40][41][42][43][44][45] Versamune® Platform - Mechanism of Action (MOA) The Versamune® platform utilizes synthetic positively charged lipid nanoparticles to promote efficient dendritic cell antigen uptake and activate Type I interferon signaling - The Versamune® platform is based on synthetic positively charged (cationic) lipids that spontaneously form nanoparticles in aqueous media, sized for efficient dendritic cell uptake[46] - Nanoparticle design and composition mimic viruses, promoting dendritic cell uptake, allowing antigens to enter the cytoplasm for presentation via MHC Class I and II pathways, priming CD8+ killer T-cells and CD4+ helper T-cells[48] - Cationic lipid structure specifically activates the Type I interferon (IFN-1) signaling pathway, inducing cytokines and chemokines necessary for CD8+ T-cell activation and proliferation, with localized induction limiting toxicity[52] - PDS0101 combines Versamune® technology with proprietary HPV 16 antigens, aiming to treat advanced HPV-associated cancers, which affect approximately 43,000 patients diagnosed annually in the US[49][50] - PDS0101's Phase I clinical trial (12 subjects) confirmed potent HPV-specific killer T-cell (CD8+) induction with no dose-limiting toxicities observed, and complete regression of pre-cancerous lesions in 6 out of 10 evaluable patients within 1-3 months[54][56][58][59] - PDS0101's Phase II clinical studies will focus on combination with other therapies like checkpoint inhibitors, immunocytokines, and chemoradiation to address unmet medical needs, with all studies expected to launch in the first half of 2020[61][62][64][66] Other Development Programs The company is expanding its Versamune® pipeline with PDS0102, PDS0103, and PDS0104 targeting various cancers like prostate, breast, colorectal, ovarian, lung, and melanoma - PDS0102 (TARP-Expressing Cancers): Targets prostate and breast cancer, successfully formulated, with clinical study initiation pending; the prostate cancer market is projected to grow to $13.6 billion by 2021[68][69] - PDS0103 (MUC-1 Expressing Cancers): Targets colorectal, breast, ovarian, and lung cancer, based on a novel NCI-licensed MUC-1 agonist antigen; the colorectal cancer market is projected to reach $11 billion by 2025, breast cancer $18.2 billion by 2023, and NSCLC $12.2 billion by 2025[70][71][72][73][74] - PDS0104 (TRP2-Expressing Cancers): Targets melanoma, with preclinical studies showing its ability to overcome immunosuppression, inhibit tumor growth, and demonstrate strong synergy with checkpoint inhibitors; melanoma is the seventh most common cancer in the US[75][76][77] A Summary of the Current State-of-the-art Existing immunotherapies like dendritic cell vaccines, CAR T-cells, and checkpoint inhibitors have limitations in efficacy, safety, and cost for solid tumors - Dendritic Cell Vaccines (e.g., Provenge®): Do not require in vivo antigen targeting, but ex vivo antigen uptake and processing are suboptimal, and they do not address immunosuppressive environments or provide immune activation[79] - CAR T-cell Immunotherapies (e.g., Kymriah®, Yescarta®): Overcome in vivo antigen processing needs and show promise in blood cancers, but are ineffective against solid tumor immunosuppression, can cause severe side effects (cytokine release syndrome), and are costly with complex manufacturing[80][81] - Other Methods (Live Vectors, Antibodies, Electroporation): Can stimulate T-cells and antibodies but often fail to effectively activate necessary immune mechanisms or counteract immunosuppression, leading to suboptimal responses[82] - Checkpoint Inhibitors (e.g., Yervoy®, Keytruda®, Opdivo®): Make cancer cells visible to T-cells by blocking immune checkpoints, but can trigger autoimmune diseases and are effective only in a minority of patients[83][84][85] - Adjuvant-based Cancer Vaccines: Generally well-tolerated, but adjuvants can induce high cytokine levels, leading to severe side effects (cytokine storm)[86][88] Combination Immunotherapy Combination immunotherapy is a leading area of cancer research, with Versamune® positioned as an ideal complement to checkpoint inhibitors due to its T-cell induction and safety profile - Combination immunotherapy represents the latest frontier in cancer research, with over one thousand such clinical studies currently underway, the vast majority involving checkpoint inhibitors[90] - Nivolumab combined with ipilimumab (a checkpoint inhibitor combination) is approved for metastatic melanoma, showing extended survival but with increased toxicity, as nearly 60% of patients experienced severe side effects[91][92] - PDS believes Versamune® is an ideal complement to checkpoint inhibitors due to its ability to promote potent CD8+ T-cell induction, modify the tumor microenvironment, and demonstrate a favorable safety profile[93] - Preclinical studies show that Versamune® combined with Trp2 (PDS0104 prototype) and anti-PD1 treatment exhibited synergistic effects in a B16F10 melanoma model, significantly inhibiting tumor growth and extending survival[94][95][96][98][99] Versamune® Mechanisms of Action (MOA) - Detailed Studies Detailed studies confirm Versamune®'s ability to enhance antigen uptake, presentation, immune activation, and T-cell quality, while modifying the tumor microenvironment - Antigen Uptake: Versamune®'s positive charge and nanoparticle size facilitate efficient dendritic cell antigen uptake, with in vivo studies showing 80% of draining lymph node dendritic cells taking up Versamune® within 4 hours, and very low systemic bioavailability (5-6%)[103][105][108][110] - Antigen Presentation: Versamune® significantly enhances antigen cross-presentation to killer T-cells (CD8+), reducing the required peptide amount by 100-fold in vitro, and promotes in vivo proliferation of both CD8+ and CD4+ T-cells[112][113][115][118][119][120][121](index=121&type=chunk] - Immune Activation: Versamune® specifically activates the Type I interferon signaling pathway, inducing the production of cytokines and chemokines (e.g., CCL2) within lymph nodes, promoting T-cell activation and proliferation while limiting inflammation to the lymph nodes to minimize systemic toxicity[124][125][128][131] - Quality of Induced T-cells: Versamune®-formulated antigen-specific CD8+ T-cells are superior in both quantity and quality compared to other adjuvants, producing a higher proportion of multifunctional T-cells (e.g., IFN-γ, TNF-α, IL-2) with approximately 10-fold greater potency[136][137][138][140][141](index=141&type=chunk] - Modifying the Tumor Microenvironment to Overcome Immunosuppression: Versamune® effectively alters the ratio of effector T-cells to immunosuppressive T-cells within tumors, promoting tumor regression; in TC-1 and B16F10 tumor models, Versamune® demonstrated potent anti-tumor efficacy, achieving complete regression of large tumors even with a single injection[143][144][145][146][148][149][150][153][154][156][158][162][163][168][169] Leadership The company is led by experienced executives and directors in drug discovery, development, and commercialization, supported by scientific experts in vaccine development and oncology - The company is led by executives and directors with extensive experience in drug discovery, development, and commercialization, supported by scientific experts in vaccine development and oncology[170][171] Research and Development Strategy PDS focuses on a low-risk clinical development path through collaborations with oncology and immunology experts, minimizing development and clinical study expenses - PDS focuses on a low-risk path for clinical development and proof-of-concept by collaborating with experts in tumor biology, immunology, and immuno-oncology, thereby reducing development and clinical study expenses[172] - The R&D process includes extensive preclinical studies, toxicology studies, and Phase I human clinical studies to confirm safety and T-cell induction efficacy[173] - Based on successful Phase I clinical studies, the company has established clinical supply agreements and collaborations with leaders in immuno-oncology, including NIH/NCI and Merck & Company, Inc[174] Facilities & Manufacturing and Commercial Scale Up Versamune® products are manufactured using an easily scalable fill-finish process by third-party CMOs, with R&D activities at BioLabs and animal testing outsourced - Products from the Versamune® development platform are manufactured using an easily scalable fill-finish process, and the company does not own or operate cGMP manufacturing facilities, relying on third-party Contract Manufacturing Organizations (CMOs) for preclinical and clinical study materials[175] - R&D activities are conducted at Princeton Innovation Center BioLabs, with animal toxicology and efficacy testing performed through third-party contracts and collaborations to maximize flexibility and minimize operating costs[177] Regulatory Pathway The next step for PDS0101 is Phase II clinical trials, with protocols submitted to the FDA and a Biologics License Application (BLA) pathway anticipated for market approval - The next step for PDS0101 is Phase II clinical trials, with relevant study protocols already submitted to the FDA[179] - The company plans to submit Chemistry, Manufacturing, and Controls (CMC) amendments related to PDS0101 Phase II studies to the FDA in the first half of 2020, to comply with eCTD format requirements and submit cGMP materials[179] - The company expects to seek marketing approval for its product candidates through the Biologics License Application (BLA) pathway under Section 351(a) of the Public Health Service Act (PHSA)[181] Intellectual Property PDS protects its proprietary technology through a portfolio of US and foreign patents, exclusive licenses, and reliance on trade secrets and confidentiality agreements - Patents: As of December 31, 2019, PDS holds 4 granted US patents (expiring 2025-2033) and 6 pending US patent applications (expiring 2033-2037), along with 22 granted foreign patents and 33 pending foreign patent applications (expiring 2031-2034 or later)[181][182][183](index=183&type=chunk] - Licensed Patents: Holds a worldwide exclusive license from Merck & Cie for the Versamune® core component (R)-DOTAP, and a US government license for MUC-1 antigens for future cationic lipid immunotherapies[184][186] - Trade Secrets: Relies on trade secrets and confidentiality agreements to protect proprietary technology and advancements, acknowledging the risk of discovery or misuse by competitors[187][188] Material License Agreements and Research and Development Agreements The company has various agreements, including non-exclusive and exclusive licenses, and collaborative R&D agreements with NIH, Merck, and other institutions for product development - NIH Patent License Agreement: A non-exclusive license for developing therapeutic cancer vaccines combining Versamune® technology for ovarian, breast, colon, and lung cancers, including upfront, annual minimum, sales royalty, and milestone payments[189] - Merck Eprova AG DOTAP Chloride Enantiomers License Agreement: An exclusive license for the worldwide commercialization of (R)-DOTAP and (S)-DOTAP chloride enantiomers to induce immune responses[190] - NCI Cooperative Research and Development Agreement (Prostate Cancer): A five-year agreement (effective February 2016) with NCI to develop Versamune® platform-based immunotherapies for prostate cancer, with PDS providing $0.5 million to $1 million annually[191][193] - University of Kentucky Research Foundation (UKRF) Cost Reimbursement Agreement: An annual agreement (renewed July 2019) for testing preclinical and clinical-stage formulations based on HPV, TARP, MUC-1, and melanoma antigens[194] - MSD International GmbH (Merck) Clinical Trial Collaboration and Supply Agreement: Collaboration for a Phase II clinical study of PDS0101 combined with Keytruda® for recurrent/metastatic head and neck cancer, revised in October 2019 for first-line treatment[195][196] - NCI Cooperative Research and Development Agreement (Advanced HPV-Associated Cancers): A five-year agreement (effective April 2019) with NCI for a Phase II clinical study of PDS0101 combined with M7824 and NHS-IL12 for advanced HPV-associated cancers, with PDS providing $110,000 annually[197][198] - Farmacore Biotechnology Amended and Restated Material Transfer Agreement: Signed in December 2019, for developing novel tuberculosis immunotherapies based on Farmacore tuberculosis antigens and Versamune®[200][201][202](index=202&type=chunk] Competition The biotechnology and pharmaceutical industries are highly competitive, with PDS facing rivals from various entities developing new immunotherapies and advanced technologies - The biotechnology and pharmaceutical industries are highly competitive, with the company facing competition from biotechnology and pharmaceutical companies, academic institutions, government agencies, and research organizations[203] - The company anticipates increased competition as new immunotherapies enter the market and advanced technologies emerge, with products competing on factors such as efficacy, safety, convenience, price, and reimbursement[204] - Key clinical-stage competitors for HPV therapeutic products include Advaxis, Transgene, ISA Pharmaceuticals, and Inovio[206] - Other relevant product development competitors include Etubics, Vaccibody, Admedus, Cel-Sci, Neo-ImmuneTech, Kite Pharma, Immune Design, Dynavax, Bavarian Nordic, Seattle Genetics, and Selecta Biosciences[206] Government Regulation and Product Approval Product development and commercialization in the US are subject to extensive and complex government regulations, including clinical trials, FDA approval, and post-market requirements - US Product Development Process: Involves preclinical testing (GLP), IND submission, human clinical trials (GCP, IRB approval) typically in Phases I, II, and III, potentially requiring Phase IV post-market studies, and manufacturing compliant with cGMP[207][208][209][210][211][212][213][214](index=214&type=chunk] - US Review and Approval Process: Requires BLA submission with PDUFA fees, thorough FDA review potentially involving advisory committees, possible REMS requirements, and pre-approval inspections of manufacturing facilities and clinical trial sites; approval may be delayed, limited, or denied[216][217][218][219][220][221][222][223][224][225](index=225&type=chunk] - Post-Approval Requirements: Approved products remain subject to ongoing FDA regulation, including record-keeping, adverse event reporting, safety and efficacy information updates, product sampling and distribution, promotional and advertising compliance (prohibiting off-label promotion), cGMP compliance, and DSCSA traceability requirements[226][227][228][229][230][231](index=231&type=chunk] - Biologics Regulatory Exclusivity: The BPCIA provides 4 years of data exclusivity and 12 years of market exclusivity for reference biologics; Orphan Drug designation offers 12 years of market exclusivity, tax credits, and BLA user fee waivers; pediatric exclusivity can extend existing exclusivity by 6 months; patent term extensions can be up to 5 years[233][234][235][236][237](index=237&type=chunk] - Other US Healthcare Laws and Compliance Requirements: Subject to federal and state anti-fraud and abuse provisions (e.g., Anti-Kickback Statute, False Claims Act, HIPAA), data privacy and security regulations (e.g., GDPR, CCPA), the federal Physician Payment Sunshine Act, and state laws; violations can lead to criminal prosecution, substantial fines, and exclusion from government programs[239][240][241][242][243][244][245][246][247](index=247&type=chunk] - Coverage, Pricing, and Reimbursement: Post-market product sales depend on coverage and reimbursement levels from third-party payers (federal and state healthcare programs, private managed care providers); reimbursement uncertainty can lead to pricing pressure and limited market access[249][250][251][252](index=252&type=chunk] - US Healthcare Reform: The ACA has multi-faceted impacts on the pharmaceutical industry, including Medicaid drug rebate programs, 340B drug pricing programs, Medicare Part D discounts, annual manufacturer fees, and the Sunshine Act; ongoing healthcare reform and drug pricing legislation may further depress product prices and affect company profitability[254][255][256][257][258][260][261][262](index=262&type=chunk] Foreign Regulation Selling products outside the US requires compliance with numerous and diverse regulatory requirements regarding quality, safety, and efficacy in each country and jurisdiction, and FDA approval does not guarantee foreign approval - Selling products outside the US requires compliance with numerous and diverse regulatory requirements regarding quality, safety, and efficacy in each country and jurisdiction, and FDA approval does not guarantee foreign approval[263] - The EU GDPR (General Data Protection Regulation) imposes strict compliance obligations on the collection and use of personal health data, including cross-border data transfer rules, with violations potentially incurring fines of up to €20 million or 4% of global annual revenue[264] Employees The company's management team possesses extensive experience in drug development, manufacturing, and regulatory affairs, supported by a semi-virtual operating strategy and expert collaborations - The company's management team possesses extensive experience in drug development research, manufacturing, clinical development, and regulatory affairs, employing a semi-virtual operating strategy and collaborating with scientific and clinical experts in cancer immunology, tumor immunology, and gynecologic oncology[265] Legal Proceedings The company may encounter various legal proceedings and claims in its ordinary course of business, but currently faces no material adverse litigation - The company may face various legal proceedings and claims in its ordinary course of business, but currently has no legal proceedings that would materially adversely affect its operations or financial condition[266] Risk Factors The company faces multiple risks including limited operating history, continuous losses, reliance on PDS0101, need for additional capital, and uncertainties in clinical development, regulatory approval, and commercialization - The company has a limited operating history and has not generated product revenue, leading to uncertainty regarding future success and profitability[269][270] - The company has incurred continuous losses since inception and expects to continue generating significant losses, potentially never achieving or maintaining profitability[271][272] - The company is highly dependent on the success of PDS0101, which is still in early clinical development and may not receive regulatory approval or achieve successful commercialization[275][277] - The company requires substantial additional capital to fund operations, and failure to obtain necessary financing may force delays, reductions, or cancellations of development programs[280][281] - Clinical trials are costly, time-consuming, and have uncertain outcomes; failure to demonstrate safety and efficacy will prevent commercialization of PDS0101[283][285] - The company faces intense competition from other biotechnology and pharmaceutical companies, and failure to compete effectively will impact operating results[291][293](index=293&type=chunk] - PDS0101 may cause adverse reactions or other characteristics that could delay or prevent its regulatory approval or limit its market acceptance[294][295](index=295&type=chunk] - The company relies on third parties for clinical trials and some R&D, and poor performance by these third parties could harm the company's business[297][298][299][300](index=300&type=chunk] - The company's intellectual property protection (patents, trade secrets) is uncertain, and it may face infringement lawsuits or risks to patent value[302][305](index=305&type=chunk] - The COVID-19 pandemic may adversely affect the company's operations, clinical trials, and supply chain[308][310](index=310&type=chunk] - US healthcare laws and regulatory requirements (e.g., Anti-Kickback Statute, False Claims Act, HIPAA) are complex and evolving, with non-compliance potentially leading to severe penalties[313][314](index=314&type=chunk] - The company's stock price is expected to be volatile and may decline due to various factors, including clinical trial results, competition, and market sentiment[316][317](index=317&type=chunk] - The company has identified material weaknesses in internal control over financial reporting, which if not effectively remediated, could lead to material misstatements in financial statements and harm investor confidence[320][321][322][323][324][325][326][327][328][329][330][331][332][333][334][335][336][338][339][340][341][342][343][353][354][355][356][357][358][361][362][364][365][366][367][369][370][371][372][373][374][375][376][377][378][379][380][383][384][385][386][387][388][389][390][391][392][393][396][397][398][399][400][401][402][403][404][405][406][407][408][409][410][411][412][413][414][415][417][418][419][420][421][422][423][424][425][426](index=426&type=chunk] Unresolved Staff Comments The company has no unresolved staff comments for this reporting period - No unresolved staff comments[427] Properties The company terminated its previous office lease in August 2019, maintains R&D facilities at BioLabs, and entered a new office sublease in Florham Park, New Jersey, in March 2020 - The company terminated its original office space lease agreement in Berkeley Heights, New Jersey, on August 31, 2019[428] - The company maintains month-to-month leased R&D facilities at Princeton Innovation Center BioLabs[428] - The company entered into a new office space sublease agreement in Florham Park, New Jersey, on March 10, 2020, for approximately 40 months, with an option to extend until October 31, 2027[429] Legal Proceedings The company is subject to claims, legal proceedings, and disputes that arise in the ordinary course of its business - The company is subject to claims, legal proceedings, and disputes that arise from time to time in its ordinary course of business[430] Mine Safety Disclosures Not applicable - Not applicable[431] PART II Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities The company's common stock is listed on Nasdaq Capital Market under "PDSB", with 52 shareholders of record as of March 9, 2020, and no equity securities purchased during the period - The company's common stock is listed on the Nasdaq Capital Market under the trading symbol "PDSB"[434] - As of March 9, 2020, the company had 52 shareholders of record[435] - The company did not purchase any registered equity securities during this reporting period[436] Management's Discussion and Analysis of Financial Condition and Results of Operations This section discusses PDS Biotechnology's financial condition and operating results, highlighting its clinical-stage focus, positive PDS0101 trial results, lack of product revenue, accumulated deficit of $28.9 million as of December 31, 2019, and liquidity plans - PDS Biotechnology is a clinical-stage biopharmaceutical company focused on developing cancer immunotherapies based on the Versamune® platform, aiming to stimulate high levels of antigen-specific killer T-cell responses[439][440] - PDS0101's Phase I clinical trial results were encouraging, confirming potent HPV-specific CD8+ T-cell induction and observing complete regression of pre-cancerous lesions in 6 out of 10 evaluable patients[442] - The company has not generated any product revenue and has incurred net losses every year since its inception, with an accumulated deficit of $28.9 million as of December 31, 2019[447][453] Comparison of Operating Results for 2019 and 2018 (in thousand dollars) | Metric | 2019 | 2018 | Change | Change Percentage | | :--- | :--- | :--- | :--- | :--- | | Research and Development Expenses | $6,100 | $831 | $5,269 | 634% | | General and Administrative Expenses | $10,982 | $2,788 | $8,194 | 294% | | IPR&D Impairment Expense | $2,974 | $0 | $2,974 | 100% | | Lease Termination and Disposal Costs | $979 | $0 | $979 | 100% | | Depreciation and Amortization | $0 | $27 | $(27) | 100% | | Total Operating Expenses | $21,035 | $3,646 | $17,389 | 477% | | Operating Loss | $(21,035) | $(3,646) | $(17,389) | 100% | | Bargain Purchase Gain | $13,335 | $0 | $13,335 | 100% | | Loss on Debt Extinguishment | $0 | $(185) | $185 | 100% | | Interest (Expense), Net | $320 | $(5) | $325 | 100% | | Loss Before Income Taxes | $(7,380) | $(3,836) | $(3,544) | 100% | | Income Tax (Benefit) | $(382) | $0 | $(382) | 92% | | Net Loss and Comprehensive Loss | $(6,998) | $(3,836) | $(3,162) | 82% | Summary of Cash Flows for 2019 and 2018 (in thousand dollars) | Cash Flow Type | 2019 | 2018 | | :--- | :--- | :--- | | Net Cash Used in Operating Activities | $(18,073) | $(1,569) | | Net Cash Provided by Investing Activities | $29,381 | $0 | | Net Cash Provided by Financing Activities | $750 | $1,497 | | Net Increase (Decrease) in Cash | $12,058 | $(72) | | Cash and Cash Equivalents at End of Period | $12,161,739 | $103,695 | - As of December 31, 2019, the company held $12.2 million in cash and cash equivalents; a public offering completed in February 2020 raised an additional $11.9 million in net proceeds, expected to support company operations and R&D programs for at least 12 months[454][493][495] - The company has identified material weaknesses in internal control over financial reporting, including issues in the control environment, risk assessment, information and communication, and control activities, and is actively implementing remediation plans[523][524][525][526][528](index=528&type=chunk] Company Overview and Business Update PDS Biotechnology is a clinical-stage biopharmaceutical company leveraging its proprietary Versamune® platform to develop multifunctional cancer immunotherapies, with encouraging Phase I PDS0101 results and planned Phase II studies - PDS Biotechnology is a clinical-stage biopharmaceutical company leveraging its proprietary Versamune® platform to develop multifunctional cancer immunotherapies, aiming to stimulate high levels of antigen-specific killer T-cell responses[439][440] - PDS0101's Phase I clinical trial results were encouraging, confirming potent HPV-specific CD8+ T-cell induction and observing complete regression of pre-cancerous lesions in 6 out of 10 evaluable patients[442][451] - The company has adjusted its clinical strategy to combine PDS0101 with checkpoint inhibitors and standard of care to address unmet medical needs, with three Phase II clinical studies expected to launch in the first half of 2020[444][445][451] - The company has not generated any product revenue and has incurred net losses every year since its inception; as of December 31, 2019, the accumulated deficit was $28.9 million, with cash and cash equivalents totaling $12.2 million[447][453][454] Corporate Information The company completed a reverse merger with Private PDS on March 15, 2019, and was renamed PDS Biotechnology Corporation, with Private PDS as the accounting acquirer - The company completed a reverse merger with Private PDS on March 15, 2019, and was renamed PDS Biotechnology Corporation, with Private PDS considered the accounting acquirer and its historical financial statements becoming those of the combined company[457][458] Financial Operations Overview The company has no product revenue, with future income expected from R&D payments and licensing, while incurring significant R&D, G&A, and other operating expenses - Revenue: The company has not generated any revenue from commercial product sales, with future revenue potentially derived from R&D payments, license fees, and milestone payments[459] - Research and Development Expenses: Include employee-related costs, license fees, clinical trial material costs, and consulting fees; in 2019, an IPR&D asset impairment of $2.974 million was recognized related to Edge's NEWTON 2 trial[460][461] - General and Administrative Expenses: Primarily include salaries and related benefits for executive, finance, legal, business development, and support functions, as well as travel, audit, tax, and legal professional fees[463][464] - Lease Termination and Disposal Costs: Include $0.7 million in lease termination fees and $0.3 million in office furniture disposal fees[466] - Other Income: Primarily consists of interest income generated from cash and cash equivalents[467][468] Critical Accounting Policies and Significant Judgments and Estimates Key accounting policies involve acquisitions, asset impairment, income taxes, accrued clinical expenses, and equity compensation, all requiring significant management judgment and estimates - Acquisitions: Accounted for using the acquisition method, measuring acquired assets and liabilities at fair value, including the fair value of IPR&D; fair value estimates rely on complex judgments about future events and uncertainties[473][474] - Asset Impairment: All long-lived assets are reviewed for impairment indicators throughout the year, and indefinite-lived intangible assets are tested annually for impairment; an impairment charge is recorded when fair value is below carrying value[475][476] - Income Taxes: Deferred income tax assets and liabilities are recognized using the balance sheet method; deferred tax assets are reduced by a valuation allowance if their realization is not probable; as of December 31, 2019, the company had approximately $79.1 million in federal net operating loss (NOL) carryforwards and $0.7 million in federal R&D tax credit carryforwards, potentially subject to limitations under Section 382 of the Internal Revenue Code[477][479] - Accrued Clinical Expenses: Estimated by reviewing contracts, communicating with personnel, and assessing the level of services performed and associated costs based on available information[480] - Equity Compensation: Fair value of equity option awards for employees, directors, and non-employees is estimated using the Black-Scholes option pricing model and amortized on a straight-line basis over the service period; ASU 2018-07 was adopted on January 1, 2019[481][482] Results of Operations - Comparison of the Years Ended December 31, 2019 and 2018 Operating expenses significantly increased in 2019 compared to 2018, driven by higher R&D and G&A costs, partially offset by a bargain purchase gain from the merger Comparison of Operating Results for 2019 and 2018 (in thousand dollars) | Metric | 2019 | 2018 | Change | Change Percentage | | :--- | :--- | :--- | :--- | :--- | | Research and Development Expenses | $6,100 | $831 | $5,269 | 634% | | General and Administrative Expenses | $10,982 | $2,788 | $8,194 | 294% | | IPR&D Impairment Expense | $2,974 | $0 | $2,974 | 100% | | Lease Termination and Disposal Costs | $979 | $0 | $979 | 100% | | Depreciation and Amortization | $0 | $27 | $(27) | 100% | | Total Operating Expenses | $21,035 | $3,646 | $17,389 | 477% | | Operating Loss | $(21,035) | $(3,646) | $(17,389) | 100% | | Bargain Purchase Gain | $13,335 | $0 | $13,335 | 100% | | Loss on Debt Extinguishment | $0 | $(185) | $185 | 100% | | Interest (Expense), Net | $320 | $(5) | $325 | 100% | | Loss Before Income Taxes | $(7,380) | $(3,836) | $(3,544) | 100% | | Income Tax (Benefit) | $(382) | $0 | $(382) | 92% | | Net Loss and Comprehensive Loss | $(6,998) | $(3,836) | $(3,162) | 82% | - Research and development expenses increased by $5.3 million, primarily due to higher clinical study and internal R&D personnel costs[484] - General and administrative expenses increased by $8.2 million, mainly due to higher personnel costs, non-cash equity compensation, D&O insurance premiums, and legal fees[486][487](index=487&type=chunk] - An IPR&D impairment expense of $2.974 million and lease termination and disposal costs of $0.979 million were recognized in 2019[488][489](index=489&type=chunk] - A bargain purchase gain of $13.3 million resulted from the merger in 2019[490] - Net interest income of $0.3 million in 2019 was primarily from interest earned on cash and cash equivalents[491] Liquidity and Capital Resources As of December 31, 2019, the company had $12.2 million in cash, supplemented by a $11.9 million public offering in February 2020, expected to fund operations for at least 12 months - As of December 31, 2019, the company had $12.2 million in cash and cash equivalents, primarily from $29.1 million in cash acquired from Edge in the merger[492] - In February 2020, the company raised approximately $11.9 million in net proceeds through a public offering[493][498](index=498&type=chunk] - The company expects its existing cash resources, including proceeds from the February 2020 offering, to be sufficient to support operations and R&D programs for at least 12 months[495] - The company plans to meet future operating and capital requirements through equity and/or debt financings, government grant programs, and selective collaborations[496] - In July 2019, the company entered into a common stock purchase agreement with Aspire Capital to sell up to $20 million of common stock over 30 months, but no shares had been sold as of December 31, 2019, other than commitment shares[497] Cash flows Operating cash outflows significantly increased in 2019 due to higher R&D and G&A expenses, while investment activities provided substantial cash from the merger Summary of Cash Flows for 2019 and 2018 (in thousand dollars) | Cash Flow Type | 2019 | 2018 | | :--- | :--- | :--- | | Net Cash Used in Operating Activities | $(18,073) | $(1,569) | | Net Cash Provided by Investing Activities | $29,381 | $0 | | Net Cash Provided by Financing Activities | $750 | $1,497 | | Net Increase (Decrease) in Cash | $12,058 | $(72) | | Cash and Cash Equivalents at End of Period | $12,161,739 | $103,695 | - Net cash used in operating activities increased by $16.5 million in 2019, primarily due to higher R&D costs, general and administrative expenses, and payments for merger-related liabilities[501] - Net cash provided by investing activities was $29.4 million in 2019, primarily from cash acquired in the merger[502] - Net cash provided by financing activities was $0.8 million in 2019, primarily from common stock issuance[503] Operating Capital Requirements The company anticipates continued losses and requires substantial additional funding to support ongoing operations, R&D, and commercialization efforts - The company has not generated product revenue, anticipates continued losses, and will require substantial additional capital to support ongoing operations, R&D, and commercialization[504] - The initiation, progress, timing, costs, and results of planned clinical trials[506] - The outcome, timing, and cost of meeting regulatory requirements of the FDA and other regulatory agencies[506] - The costs of filing, prosecuting, defending, and enforcing patent claims[506] - The effect of competitive technologies and market developments[506] - The costs of establishing sales, marketing, and distribution capabilities[506] - The initiation, progress, timing, and results of commercialization efforts[506] Contractual Obligations and Commitments The company's contractual obligations primarily consist of milestone payments, with no significant non-cancelable purchase commitments as of December 31, 2019 Contractual Obligations as of December 31, 2019 (in thousand dollars) | Contractual Obligation | Total | Less than 1 year | 1-3 years | 3-5 years | More than 5 years | | :--- | :--- | :--- | :--- | :--- | :--- | | Milestone Payments | $550 | $110 | $220 | $220 | $0 | - The company has no significant non-cancelable purchase commitments[511] JOBS Act As an "emerging growth company," PDS has opted out of the extended transition period for new accounting standards but intends to rely on other JOBS Act exemptions - As an "emerging growth company," the company has irrevocably elected not to take advantage of the extended transition period provided by the JOBS Act for complying with new or revised accounting standards and will adopt new standards on the same timeline as other public companies[512] - The company intends to rely on other exemptions in the JOBS Act, such as not providing an auditor attestation report on internal control over financial reporting as required by Section 404(b) of the Sarbanes-Oxley Act[513] Off-balance Sheet Arrangements The company had no off-balance sheet arrangements during the reporting period and currently has none - The company had no off-balance sheet arrangements during the reporting period and currently has none[514] Financial Statements and Supplementary Data This report includes the company's financial statements and supplementary data as of December 31, 2019, with specific indexing provided in Item 15 - This report includes the company's financial statements and supplementary data as of December 31, 2019, with specific indexing provided in Item 15[516] Changes in and Disagreements with Accountants on Accounting and Financial Disclosure There were no changes in accountants or disagreements with accountants on accounting and financial disclosure during this reporting period - No changes in accountants or disagreements with accountants on accounting and financial disclosure[517] Controls and Procedures As of December 31, 2019, the company's disclosure controls and procedures were deemed ineffective due to identified material weaknesses in internal control over financial reporting - As of December 31, 2019, the company's disclosure controls and procedures were deemed ineffective due to material weaknesses in internal control over financial reporting[518] - Management assessed that as of December 31, 2019, the company's internal controls had material weaknesses in four areas: control environment, risk assessment, information and communication, and control activities[521][523][524][525][526](index=526&type=chunk] - These material weaknesses led to non-material error corrections in the consolidated financial statements and their notes for 2018 and 2019[523] - Supplement existing accounting resources and engage external consultants to assist with technical accounting activities[528] - Plan to hire staff with technical accounting expertise and public company experience, including a Chief Financial Officer, as needed[528] - Strengthen review controls, including those for timely identification, capture, and processing of financial information for financial accounting and reporting[528] - Develop and implement a comprehensive and continuous risk assessment process to identify and evaluate risks of material misstatement and ensure financial reporting processes and related internal controls are appropriately designed and implemented[528] Other Information Not applicable - Not applicable[532] PART III Directors, Executive Officers and Corporate Governance The information required for this item is incorporated by reference from the company's proxy statement - The information required for this item is incorporated by reference into this annual report from the proxy statement[536] Executive Compensation The information required for this item is incorporated by reference from the company's proxy statement - The information required for this item is incorporated by reference into this annual report from the proxy statement[537] Securities Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters The information required for this item is incorporated by reference from the company's proxy statement - The information required for this item is incorporated by reference into this annual report from the proxy statement[538] Certain Relationships and Related Transactions and Director Independence The information required for this item is incorporated by reference from the company's proxy statement - The information required for this item is incorporated by reference into this annual report from the proxy statement[539] Principal Accountant Fees and Services The information required for this item is incorporated by reference from the company's proxy statement - The information required for this item is incorporated by reference into this annual report from the proxy statement[540] PART IV Exhibits and Financial Statement Schedules This report includes the required financial statements and exhibits, with financial statement schedules omitted as they are inapplicable, unnecessary, or the information is presented elsewhere - This report contains the required financial statements and exhibits[541] - All financial statement schedules have been omitted because they are inapplicable, not required, or the information is presented in the financial statements or their notes[542] Form 10-K Summary This report does not contain a Form 10-K summary - No Form 10-K summary[543]