Fortress Biotech(FBIO) - 2020 Q4 - Annual Report

Part I Business Fortress Biotech operates as a diversified biopharmaceutical company, acquiring, developing, and commercializing products through its parent operations and partner companies - Fortress Biotech's business model involves acquiring, developing, and commercializing pharmaceutical products through its own operations, majority-owned subsidiaries, and significant minority ownership positions in founded entities[20] - The company has established collaborations with prominent research institutions such as Fred Hutchinson Cancer Research Center, St Jude Children's Research Hospital, and Dana-Farber Cancer Institute[20] - Fortress utilizes a variety of strategic arrangements to fund research and development, including joint ventures, out-licensings, and both public and private financings, with three partner companies currently publicly traded[20] Product Portfolio The company's portfolio spans commercialized dermatology products, late-stage candidates like IV Tramadol and CUTX-101, and a robust early-stage and preclinical pipeline focused on CAR T and gene therapies Commercialized Dermatology Products (via Journey Medical) | Product | Indication | | :--- | :--- | | Ximino® | Inflammatory lesions of non-nodular moderate to severe acne vulgaris | | Targadox® | Adjunctive therapy for severe acne | | Exelderm® | Treatment of tinea infection (ringworm, jock itch) | | Ceracade® | Dry skin conditions and dermatitis | | Luxamend® | Management of superficial wounds, abrasions, and burns | | Accutane® | Severe recalcitrant nodular acne | Key Late-Stage Product Candidates | Candidate | Partner | Indication | Status | | :--- | :--- | :--- | :--- | | IV Tramadol | Avenue | Post-operative acute pain | NDA resubmitted to FDA; PDUFA date April 12, 2021 | | CUTX-101 | Cyprium | Menkes Disease | Positive top-line results; FDA Breakthrough Therapy Designation; Rolling NDA submission planned for H2 2021 | | MB-107 / MB-207 | Mustang | XSCID (Bubble Boy Disease) | Registrational Phase 2 trial for MB-107 initiated; IND for MB-207 trial planned for Q2 2021 | | Cosibelimab | Checkpoint | mCSCC and NSCLC | Registration-enabling trial ongoing; Top-line results expected H2 2021 | | CAEL-101 | Caelum | AL Amyloidosis | Two Phase 3 studies initiated and actively enrolling | - The early-stage pipeline is heavily focused on oncology, particularly CAR T therapies targeting CD123 (MB-102), IL13Rα2 (MB-101), CS1 (MB-104), CD20 (MB-106), HER2 (MB-103), and PSCA (MB-105) for various cancers including BPDCN, glioblastoma, multiple myeloma, and prostate cancer[38][39][43] - Preclinical development includes AAV-ATP7A gene therapy for Menkes disease, AVTS-001 gene therapy for complement-mediated diseases, and the ONCOlogues platform using peptic nucleic acids (PNAs) to target genetically driven cancers[52][53][60] Competition Fortress and its partners face intense competition from larger pharmaceutical and biotechnology firms, with significant generic competition posing a threat to its commercialized dermatology products - The company competes with large pharmaceutical and biotechnology enterprises that have significantly greater financial, product development, manufacturing, and marketing resources[63] - In the dermatology sector, major competitors include Galderma, Vyne Therapeutics, Sol-Gel, Almirall, and Sun Pharma, with competition based on quality, efficacy, market acceptance, and price[63] - The company faces significant risk from generic competition, which typically leads to intense price competition and loss of market share for branded products once market exclusivity is lost[65] Government Regulation The company operates under extensive FDA regulation, requiring lengthy and costly preclinical and clinical development, adherence to manufacturing and clinical practices, and navigating complex reimbursement policies - The FDA approval process for a new pharmaceutical product generally includes preclinical lab and animal studies, submission of an Investigational New Drug (IND) application, and performance of adequate and well-controlled human clinical trials (Phase 1, 2, and 3)[67][69] - Manufacturing facilities must comply with current Good Manufacturing Practices (CGMPs), and clinical trials must adhere to Good Clinical Practices (GCPs), with non-compliance potentially leading to sanctions or withdrawal of approval[67] - The Orphan Drug Act provides incentives, including seven years of market exclusivity, for products developed for rare diseases affecting fewer than 200,000 people in the U.S[76] - Sales of approved products are dependent on reimbursement from third-party payors, including government programs and private insurers, with healthcare reforms like the Affordable Care Act (ACA) potentially impacting coverage and pricing[80] Corporate Information As of December 31, 2020, Fortress and its partner companies employed 111 full-time personnel, led by an experienced executive team, with principal offices in New York, NY - As of December 31, 2020, the company and its partner companies employed 111 full-time personnel[82] Executive Officers (as of Dec 31, 2020) | Name | Age | Position | | :--- | :--- | :--- | | Lindsay A Rosenwald, M.D. | 65 | Chairman of the Board of Directors, President and Chief Executive Officer | | Robyn M Hunter | 59 | Chief Financial Officer | | George Avgerinos, Ph.D. | 67 | Senior Vice President, Biologics Operations | | Michael S Weiss | 54 | Executive Vice Chairman, Strategic Development | Risk Factors The company faces substantial risks including high drug development failure rates, significant financing needs, reliance on limited revenue streams, complex business structure, dependence on third parties, intellectual property challenges, and commercialization uncertainties Risks Inherent in Drug Development Most product candidates are in early development, facing high risks of failure in clinical trials, regulatory approval delays, or being outcompeted by more advanced treatments - Most product candidates are in early development and may never be successfully developed or commercialized due to the high-risk, time-intensive, and costly nature of pharmaceutical development and regulation[91] - The FDA and other regulatory agencies may delay, limit, or refuse approval for numerous reasons, including disagreements with trial design, insufficient safety or efficacy data, or unsatisfactory manufacturing processes[93] - Competitors with greater resources may develop treatments more quickly or effectively, which could eliminate the commercial opportunity for Fortress's product candidates[97] Risks Pertaining to the Need for and Impact of Existing and Additional Financing Activities The company has a history of operating losses and an accumulated deficit, requiring substantial additional funding for R&D and commercialization, with existing debt covenants limiting financial flexibility - The company has a history of operating losses, reporting a loss from continuing operations of $103.0 million in 2020 and an accumulated deficit of $482.8 million as of December 31, 2020[104] - The company's $60.0 million senior secured credit agreement with Oaktree contains restrictive covenants on actions like incurring debt, making investments, and paying dividends, which could inhibit strategic transactions[104] - Substantial additional funding is needed for R&D programs, and failure to raise capital when needed may force delays, curtailment, or elimination of programs and commercialization efforts[108] Risks Pertaining to Our Existing Revenue Stream from Journey Medical Corporation Operating income heavily relies on Journey Medical's dermatology product sales, which are vulnerable to manufacturing issues, competition, generic threats, and adverse changes in healthcare reimbursement policies - The vast majority of operating income is expected to come from the sale of dermatology products through Journey Medical, and any setback with these products could significantly impair operating results[111] - Most of Journey's sales are from products without patent protection, making them vulnerable to generic competition, which could lead to intense price competition and loss of market share[111][112] - Market acceptance and sales depend on adequate coverage and reimbursement from third-party payors, and legislative changes, such as those related to the ACA, and payor policies could negatively affect profitability[114][118][122] Risks Pertaining to our Business Strategy, Structure and Organization The company's strategy involves complex collaborations and potential divestitures, carrying execution risks, potential financial liabilities from subsidiary guarantees, and conflicts of interest due to overlapping management roles - The merger of partner company Avenue with InvaGen is at risk, as InvaGen has asserted a Material Adverse Effect due to COVID-19's impact and issues with the proposed product label for IV Tramadol, potentially allowing InvaGen to avoid closing the deal[127] - Fortress acts as an indemnitor for certain obligations of its subsidiaries, such as a potential indemnification obligation of up to $35.0 million to InvaGen related to the Avenue merger agreement[129] - The company's growth depends on acquiring or in-licensing new products, which entails risks such as exposure to unknown liabilities, integration challenges, and competition for attractive assets[129] - Certain officers and directors serve in similar roles at partner companies and related parties, which could create conflicts of interest regarding corporate opportunities and transactions[131] Risks Pertaining to Reliance on Third Parties Fortress is highly dependent on third-party manufacturers and clinical research organizations, introducing significant operational risks related to compliance, supply disruption, and data reliability - The company relies predominantly on third-party manufacturers for preclinical, clinical, and commercial product supplies, and any failure by these manufacturers to comply with CGMP could disrupt supply and impair commercialization[137] - The company relies on third-party CROs and clinical investigators to conduct its clinical trials, and poor performance or non-compliance by these parties could delay trials or render data unreliable[142] - This reliance on third parties reduces control over manufacturing and development activities but does not relieve Fortress of its responsibilities or potential liabilities under regulations like GCP and GLP[142] Risks Pertaining to Intellectual Property and Potential Disputes with Licensors Thereof Success hinges on robust intellectual property protection, but patents are uncertain and vulnerable to challenges, with in-licensed IP exposing the company to potential disputes with licensors and infringement claims - The company's ability to successfully commercialize products depends on obtaining and maintaining sufficient patent protection, but the patent process is uncertain and issued patents may be challenged or invalidated[146] - The company is exposed to litigation risk if it is sued for infringing third-party intellectual property rights, which could be costly and result in having to obtain licenses or abandon product candidates[150] - Since most product candidates are based on in-licensed IP, any dispute with licensors regarding the terms of license agreements could adversely affect the ability to develop and commercialize the applicable products[153] Risks Pertaining to the Commercialization of Product Candidates Even with regulatory approval, product candidates face significant commercialization risks including limited market acceptance, substantial product liability exposure, and ongoing regulatory oversight that could lead to market withdrawal - Approved products may not gain market acceptance from physicians, patients, and payors, which would limit revenue, as acceptance depends on factors like efficacy, safety, cost, and advantages over alternatives[159] - The company faces potential product liability claims, which could lead to substantial financial liability and limit commercialization, a notable risk for products like Accutane, which has a history of litigation[161][163] - Approved products remain subject to ongoing regulatory requirements and review, and failure to comply or the discovery of new problems could result in restrictions, recalls, or withdrawal of approval[163] Risks Pertaining to Legislation and Regulation Affecting the Biopharmaceutical and Other Industries The company's business is subject to unpredictable changes in government regulation and healthcare policy, including drug pricing initiatives and complex anti-kickback laws, with non-compliance leading to severe penalties - The company cannot predict the impact of future legislation or administrative action, particularly regarding drug pricing, which could be affected by policies from the new presidential administration[169][171] - Relationships with customers and payors are subject to anti-kickback, false claims, and other healthcare laws, and violations can lead to criminal sanctions, civil penalties, and exclusion from government healthcare programs[171] General Risks The company faces general business risks such as the adverse effects of public health crises like COVID-19, challenges in retaining key personnel, stock price volatility, and potential limitations on using net operating loss carryforwards - The COVID-19 pandemic could adversely affect clinical trials by causing delays in site initiation and patient enrollment, disrupting supply chains, and diverting healthcare resources[176] - The company's success depends on retaining key executive, scientific, and technical personnel in a highly competitive market[181][183] - The ability to use pre-change Net Operating Losses (NOLs) to offset future income may be limited under Section 382 of the Internal Revenue Code if an "ownership change" occurs[189] - The company has never paid cash dividends on its Common Stock and does not intend to in the near future, so capital appreciation is the sole source of potential gain for common stockholders[192] Unresolved Staff Comments The company reports no unresolved staff comments from the Securities and Exchange Commission - None[196] Properties Fortress Biotech's principal executive offices are in New York, NY, with additional leased office space in Waltham, MA, and Scottsdale, AZ for Journey Medical, while Mustang Bio operates a cell therapy manufacturing facility in Worcester, MA - The company's principal executive offices are at 2 Gansevoort Street, New York, NY, under a 15-year lease, with portions subleased to related party TGTX[197] - Partner company Mustang Bio leases a 27,043 sq ft manufacturing facility in Worcester, MA, for producing personalized CAR T and gene therapies[199] - Partner company Journey Medical leases office space in Scottsdale, AZ, with a lease term extending to December 2022[198] Legal Proceedings Fortress Biotech is a defendant in a securities class action lawsuit regarding IV Tramadol's NDA and faces potential legal proceedings with Cipla and InvaGen concerning the Avenue merger - A securities class action complaint was filed against the company and two of its officers in November 2020, alleging false and/or misleading statements related to Avenue Therapeutics' NDA for IV Tramadol during the class period of December 11, 2019, to October 9, 2020[201] - The company notes a potential for legal proceedings arising from claims by Cipla and InvaGen that the conditions for the second closing of the Avenue merger cannot be met[201] Mine Safety Disclosures This item is not applicable to the company - Not applicable[201] Part II Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities The company's Common Stock and 9.375% Series A Preferred Stock are listed on NASDAQ, with no cash dividends paid on common stock as future earnings are retained for business development - The company's Common Stock trades on the NASDAQ Capital Market under the symbol "FBIO"[203] - The 9.375% Series A Cumulative Redeemable Perpetual Preferred Stock trades on the NASDAQ Capital Market under the symbol "FBIOP"[203] - The company has never paid cash dividends on its Common Stock and does not intend to in the near future, while dividends on the Series A Preferred Stock are paid monthly at a rate of 9.375% per annum[207] Selected Consolidated Financial Data This item is not applicable to the company - Not applicable[208] Management's Discussion and Analysis of Financial Condition and Results of Operations Management discusses the company's financial performance, liquidity, and recent operational events, highlighting increased net revenue, continued operating losses, and strong cash position from recent financings Results of Operations For 2020, net revenue increased by 24% to $45.6 million, driven by dermatology product sales, while the loss from operations improved to $94.3 million due to decreased R&D expenses, despite a rise in SG&A Consolidated Results of Operations (in thousands) | | Year Ended Dec 31, 2020 | Year Ended Dec 31, 2019 | Change $ | Change % | | :--- | :--- | :--- | :--- | :--- | | Net revenue | $45,599 | $36,629 | $8,970 | 24% | | Product revenue, net | $44,531 | $34,921 | $9,610 | 28% | | Total operating expenses | $139,869 | $147,448 | ($7,579) | (5)% | | Cost of goods sold | $14,594 | $10,532 | $4,062 | 39% | | Research and development | $61,275 | $75,236 | ($13,961) | (19)% | | R&D – licenses acquired | $2,834 | $6,090 | ($3,256) | (53)% | | Selling, general and administrative | $61,166 | $55,590 | $5,576 | 10% | | Loss from operations | ($94,270) | ($110,819) | $16,549 | (15)% | | Net loss | ($102,985) | ($101,660) | ($1,325) | 1% | | Net loss attributable to common stockholders | ($46,526) | ($39,960) | ($6,566) | 16% | - The decrease in R&D spending was primarily driven by lower clinical trial costs at Avenue following the completion of studies in 2019, and reduced manufacturing costs for cosibelimab at Checkpoint[242] - The increase in SG&A expenses was mainly due to higher headcount-related costs at Fortress and Checkpoint, and increased sales and marketing costs at JMC for its expanded product portfolio[244] Liquidity and Capital Resources As of December 31, 2020, the company had $233.4 million in cash and equivalents, with financing activities providing $172.4 million, sufficient for the next twelve months but requiring additional long-term capital - The company held cash and cash equivalents of $233.4 million as of December 31, 2020[251] Consolidated Cash Flow Summary (in thousands) | | Year Ended Dec 31, 2020 | Year Ended Dec 31, 2019 | | :--- | :--- | :--- | | Net cash used in operating activities | ($83,682) | ($94,961) | | Net cash (used in)/provided by investing activities | ($7,164) | $20,097 | | Net cash provided by financing activities | $172,410 | $146,714 | - In August 2020, the company entered into a $60 million senior secured credit agreement with Oaktree to refinance existing indebtedness[231] - The company raised significant capital through its At-The-Market (ATM) programs, issuing approximately 17.4 million shares of common stock for gross proceeds of $47.5 million in 2020[251] - Management believes current cash is sufficient to fund operations for at least the next 12 months, but additional capital will be needed for long-term development plans[253] Quantitative and Qualitative Disclosures About Market Risk This item is not applicable to the company - Not applicable[253] Financial Statements and Supplementary Data This section indicates that the company's consolidated financial statements and related notes are provided, beginning on page F-1 of the Annual Report on Form 10-K - The required information is set forth in the consolidated financial statements and notes beginning at page F-1 of the report[253] Changes in and Disagreements with Accountants on Accounting and Financial Disclosure The company reports no changes in or disagreements with its accountants on accounting and financial disclosure - None[253] Controls and Procedures Management concluded that the company's disclosure controls and procedures and internal control over financial reporting were effective as of December 31, 2020, with no material changes during the quarter - Management concluded that as of December 31, 2020, the company's disclosure controls and procedures were effective[257] - Based on an assessment using the COSO framework, management concluded that the company's internal control over financial reporting was effective as of December 31, 2020[258] Other Information The company reports no other information for this item - None[260] Part III Directors, Executive Officers and Corporate Governance Information regarding the company's directors, executive officers, and corporate governance practices is incorporated by reference from the 2021 Proxy Statement - Information is incorporated by reference from the Proxy Statement for the 2021 Annual Meeting of Stockholders[262] Executive Compensation Information regarding executive compensation is incorporated by reference from the company's 2021 Proxy Statement - Information is incorporated by reference from the Proxy Statement for the 2021 Annual Meeting of Stockholders[263] Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters Information regarding security ownership and equity compensation plans is incorporated by reference from the company's 2021 Proxy Statement - Information is incorporated by reference from the Proxy Statement for the 2021 Annual Meeting of Stockholders[263] Certain Relationships and Related Transactions, and Director Independence Information regarding certain relationships, related-party transactions, and director independence is incorporated by reference from the company's 2021 Proxy Statement - Information is incorporated by reference from the Proxy Statement for the 2021 Annual Meeting of Stockholders[263] Principal Accounting Fees and Services Information regarding principal accounting fees and services is incorporated by reference from the company's 2021 Proxy Statement - Information is incorporated by reference from the Proxy Statement for the 2021 Annual Meeting of Stockholders[263] Part IV Exhibits, Financial Statement Schedules This section lists the financial statements, financial statement schedules, and exhibits filed as part of the Form 10-K, including corporate governance documents and material agreements - The consolidated financial statements, including the Report of Independent Registered Public Accounting Firm, are filed as part of this report[265][267] - A comprehensive list of exhibits is provided, including corporate governance documents, material agreements such as the Oaktree Credit Agreement, and various stock incentive plans[269][270] Financial Statements The audited consolidated financial statements for 2020 and 2019 are presented with an unqualified auditor's opinion, highlighting increased assets and continued net losses, with detailed disclosures in the notes - The independent auditor, BDO USA, LLP, issued an unqualified opinion on the consolidated financial statements[275] - The auditor identified the accounting for the $60.0 million Oaktree Note and related warrants as a critical audit matter due to its complexity, including the assessment of potential embedded derivatives[277][279] Consolidated Balance Sheet Highlights (in thousands) | | Dec 31, 2020 | Dec 31, 2019 | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $233,351 | $136,858 | | Total current assets | $261,571 | $156,252 | | Total assets | $328,834 | $226,422 | | Liabilities & Equity | | | | Total current liabilities | $47,959 | $45,616 | | Notes payable, long-term (net) | $51,677 | $77,436 | | Total liabilities | $131,835 | $153,890 | | Total stockholders' equity | $196,999 | $72,532 | Consolidated Statement of Operations Highlights (in thousands) | | Year Ended Dec 31, 2020 | Year Ended Dec 31, 2019 | | :--- | :--- | :--- | | Net revenue | $45,599 | $36,629 | | Loss from operations | ($94,270) | ($110,819) | | Net loss | ($102,985) | ($101,660) | | Net loss attributable to common stockholders | ($46,526) | ($39,960) | | Net loss per common share - basic and diluted | ($1.43) | ($1.86) |