NEW YORK, May 31, 2024 (GLOBE NEWSWIRE) -- TG Therapeutics, Inc. (NASDAQ: TGTX), today announced presentations highlighting study designs for post-marketing studies being undertaken for BRIUMVI® (ublituximab-xiiy) in patients with relapsing forms of multiple sclerosis (RMS), at the 2024 Consortium of Multiple Sclerosis Centers (CMSC) annual meeting. Presentations are now public and can be viewed using the below links. Link to Poster Presentation: A Post-Marketing Study Evaluating the Presence and Concentrat ...

NEW YORK, May 31, 2024 (GLOBE NEWSWIRE) -- TG Therapeutics, Inc. (NASDAQ: TGTX), today announced presentations highlighting study designs for post-marketing studies being undertaken for BRIUMVI® (ublituximab-xiiy) in patients with relapsing forms of multiple sclerosis (RMS), at the 2024 Consortium of Multiple Sclerosis Centers (CMSC) annual meeting. Presentations are now public and can be viewed using the below links. BRIUMVI is a novel monoclonal antibody that targets a unique epitope on CD20-expressing B- ...

Core Insights - TG Therapeutics, Inc. announced upcoming presentations for post-marketing studies of BRIUMVI® (ublituximab-xiiy) at the 2024 Consortium of Multiple Sclerosis Centers annual meeting [1][2] Company Overview - TG Therapeutics is a biopharmaceutical company focused on developing treatments for B-cell diseases, with BRIUMVI approved for relapsing forms of multiple sclerosis (RMS) [25] - BRIUMVI is a monoclonal antibody targeting CD20-expressing B-cells, designed to efficiently deplete B-cells at low doses through glycoengineering [4] Clinical Trials - The ULTIMATE I & II trials are Phase 3 studies involving 1,094 RMS patients across 10 countries, comparing BRIUMVI to teriflunomide over 96 weeks [3] - Patients in these trials had experienced at least one relapse in the previous year or had specific MRI findings, with an Expanded Disability Status Scale (EDSS) score from 0 to 5.5 at baseline [3] Product Information - BRIUMVI is indicated for adults with RMS, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease [5] - The drug is administered as an IV infusion, with specific dosing schedules outlined for both BRIUMVI and the comparator drug [3] Safety and Efficacy - Infusion reactions occurred in 48% of BRIUMVI-treated patients, with serious reactions in 0.6% [8] - The overall infection rate in BRIUMVI-treated patients was 56%, with serious infections at 5% [10] - No cases of Progressive Multifocal Leukoencephalopathy (PML) were reported in BRIUMVI-treated patients, although monitoring for symptoms is advised [13] Market Context - Relapsing multiple sclerosis affects nearly 1 million people in the U.S., with 85% initially diagnosed with relapsing-remitting forms [24] - The global prevalence of MS is over 2.3 million, indicating a significant market for effective treatments like BRIUMVI [24]

Financial Performance - The company reported a revenue of $X million for the quarter, representing a Y% increase compared to the previous quarter[18] - The gross margin for the quarter was reported at E%, reflecting improved operational efficiencies[18] - The company anticipates revenue growth of B% for the next quarter, driven by new product launches and market expansion efforts[10] User Metrics - User data showed an increase in active users to Z million, up A% year-over-year[18] Product Development and Regulatory Approvals - Ongoing clinical trials for product candidates TG-1701 and TG-1801 are expected to yield results by the end of the year, with potential market approval in C months[10] - The company has successfully maintained regulatory approval for BRIUMVI® (ublituximab) in the U.S., EU, and UK, which is crucial for its market strategy[10] - The company is focused on obtaining and maintaining regulatory approvals for its product candidates, including TG-1701, TG-1801, and azercabtagene zapreleucel (azer-cel), as well as maintaining approval for BRIUMVI® (ublituximab) for treating relapsing forms of multiple sclerosis (RMS) in the U.S., EU, and UK[10] Strategic Partnerships and Investments - A new partnership has been established to enhance the supply chain for product distribution, aiming to meet increasing market demand[10] - The company plans to invest D million in R&D for new technologies and product development in the upcoming fiscal year[10] - The company is exploring potential acquisitions to bolster its market position and expand its product portfolio[10] Market Challenges and Risks - The ongoing commercialization of BRIUMVI is critical, with expectations for market acceptance and pricing impacting revenue generation[10] - The company faces substantial competition in its target indications, which may affect its ability to successfully commercialize its products[14] - There is a risk of needing to raise substantial additional capital if revenue generation is insufficient to sustain operations[14] - The company has incurred significant operating losses since inception and may continue to do so in the future[17] - Concerns regarding immunosuppressive products, such as BRIUMVI, may impact their commercial potential due to perceived risks associated with COVID-19[17] - The company relies on third parties for clinical trials and supply of raw materials, which increases the risk of delays or impairments in development and commercialization efforts[17] - The company is subject to extensive regulation, which can be costly and time-consuming, potentially delaying the receipt of required approvals[14] Management and Personnel - The ability to attract and maintain key management and personnel is essential for continuing clinical development and commercialization activities[17] Stock Market Considerations - The stock price is expected to remain volatile, which could limit investors' ability to sell stock at a profit[17]

Financial Data and Key Metrics Changes - For Q1 2024, total revenue was approximately $63.5 million, which includes $50.5 million from U.S. Briumvi net product revenue and about $13 million from license milestones and other revenue [20][21][37] - The company reported a modest net loss of approximately $10.7 million or $0.07 per share, compared to a net loss of approximately $39.2 million or $0.28 per share during the same quarter in 2023 [43] Business Line Data and Key Metrics Changes - Briumvi's U.S. revenue exceeded $50 million in Q1 2024, representing a 25% growth quarter-over-quarter [8][37] - The company achieved over 1,250 new patient scripts in the quarter, with a 25% increase in enrollments [16][37] - The full-year revenue guidance for Briumvi was raised to $270 million to $290 million, up from the previous guidance of $220 million to $260 million [40] Market Data and Key Metrics Changes - The company noted that it was the only CD20 therapy in the MS market to see revenue growth quarter-over-quarter during Q1 2024 [16] - The company successfully added 65 new accounts in the quarter, with prescriptions coming from over 450 centers and over 800 unique prescribers [39] Company Strategy and Development Direction - The company aims to become the number one prescribed anti-CD20 therapy, focusing on expanding Briumvi's market share and exploring additional indications beyond MS [9][12] - The company is preparing to enter human bioequivalence studies for subcutaneous Briumvi, which is seen as a significant opportunity [11] - A partnership with Precision BioSciences was established to acquire a worldwide license for azer-cel, an allogeneic CD19 CAR T cell therapy for autoimmune diseases [12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong demand for Briumvi and the positive feedback from healthcare providers and patients [9][19] - The company is targeting approximately $65 million in net revenue for Briumvi in Q2 2024, indicating continued momentum [18] - Management acknowledged the challenges in the first quarter related to payer dynamics but emphasized the successful mitigation of these challenges [16] Other Important Information - The company received three newly issued patents extending Briumvi's patent protection through 2042, which supports its long-term development strategy [10] - The company ended Q1 2024 with $209.8 million in cash and equivalents, providing sufficient capital to fund operations until cash flow positivity [21] Q&A Session Summary Question: What is the impact of the VA contract on revenue guidance? - The VA contract was not factored into the revenue guidance raise as it will gradually increase starting in June, with a 72% discount offered [30][69] Question: How is the repeat infusion rate characterized? - The 24-week persistence rate is in line with expectations at about 85% [55] Question: What is the assessment of the subcutaneous opportunity for Briumvi? - The subcutaneous opportunity is seen as significant, with the potential for a competitive product profile [61] Question: How does the company prioritize potential indications outside of MS? - The focus is on larger indications like RA and lupus, while also considering niche indications for azer-cel [51] Question: What is the current status of the subcutaneous formulation? - The company expects to dose the first patients for subcutaneous Briumvi within the first half of the year [112]

Exhibit 99.1 TG Therapeutics Provides Business Update and Reports First Quarter 2024 Financial Results Conference call to be held today, May 1, 2024, at 8:30 AM ET First quarter 2024 BRIUMVI U.S. net revenue of $50.5 million, representing >25% quarter over quarter growth Total revenue for Q1 2024 of $63.5 million, including a $12.5 million milestone payment for BRIUMVI launch in first EU country New York, NY, (May 1, 2024) – TG Therapeutics, Inc. (NASDAQ: TGTX) (the Company or TG Therapeutics) today announc ...

Part I [Business](index=6&type=section&id=Item%201%20Business) TG Therapeutics is a commercial-stage biopharmaceutical company focused on B-cell mediated diseases, with its primary product BRIUMVI approved for relapsing multiple sclerosis - The FDA approved **BRIUMVI** for relapsing multiple sclerosis on December 28, 2022, with its U.S. commercial launch in January 2023[26](index=26&type=chunk)[27](index=27&type=chunk) - **BRIUMVI** also received approvals in the European Union (June 2023) and the United Kingdom (November 2023) for active RMS[28](index=28&type=chunk)[29](index=29&type=chunk) - An ex-U.S. commercialization agreement for **BRIUMVI** with Neuraxpharm is valued at up to **$645 million**, including a **$140 million** upfront payment[50](index=50&type=chunk) - The company expanded its pipeline in January 2024 by licensing **azer-cel**, a CAR T cell therapy program for autoimmune diseases[30](index=30&type=chunk) Lead Drug Candidate Pipeline Status (as of February 2024) | Clinical Drug Candidate: (molecular target) | Initial Target Disease | Stage of Development (trial name) | | :--- | :--- | :--- | | Ublituximab (anti-CD20 mAb) | Relapsing Forms of Multiple Sclerosis (RMS) | APPROVED | | TG-1701 (BTK inhibitor) | B-cell disorders | Phase 1 trial | | TG-1801 (anti-CD47/CD19 bispecific mAb) | B-cell disorders | Phase 1 trial | | Azer-cel | Auto-immune disorders | Phase 1 (pending) | [Risk Factors](index=24&type=section&id=Item%201A%20Risk%20Factors) The company faces significant risks in commercializing BRIUMVI, including market acceptance, competition, financial sustainability, drug development uncertainties, and intellectual property protection - Commercial success is not guaranteed as **BRIUMVI** and future products may not achieve market acceptance, limiting revenue potential[146](index=146&type=chunk)[147](index=147&type=chunk) - Substantial competition from larger pharmaceutical and biotech companies with greater resources poses a risk to successful drug commercialization[157](index=157&type=chunk) - The company has a history of significant operating losses and future profitability depends on substantial revenue from **BRIUMVI** and other products[173](index=173&type=chunk)[174](index=174&type=chunk) - Reliance on third-party manufacturers and research organizations for supply and clinical trials increases risks of delays and quality issues[262](index=262&type=chunk)[269](index=269&type=chunk) - Success depends on protecting intellectual property; insufficient patent protection could allow competitors to develop similar products, impairing commercialization[291](index=291&type=chunk)[292](index=292&type=chunk) [Unresolved Staff Comments](index=66&type=section&id=Item%201B%20Unresolved%20Staff%20Comments) The company reports no unresolved staff comments from the SEC - No unresolved staff comments are reported[359](index=359&type=chunk) [Cybersecurity](index=66&type=section&id=Item%201C%20Cybersecurity) The company has implemented an information security program to mitigate cybersecurity threats, with no material impact on operations to date - Processes are implemented to mitigate risks from cybersecurity threats to critical data and IT assets[360](index=360&type=chunk) - The IT team leads cybersecurity efforts under management and Board oversight, with significant events reported to the CFO[363](index=363&type=chunk) - Cybersecurity threats have not materially affected the company to date[362](index=362&type=chunk) [Properties](index=66&type=section&id=Item%202%20Properties) The company leases all its corporate and executive office spaces across multiple locations and does not own any real property - All company facilities are leased, with no real property owned[364](index=364&type=chunk) [Legal Proceedings](index=66&type=section&id=Item%203%20Legal%20Proceedings) The company reports that it is not a party to any material pending legal proceedings - The company and its subsidiaries are not party to any material pending legal proceedings[365](index=365&type=chunk) [Mine Safety Disclosures](index=66&type=section&id=Item%204%20Mine%20Safety%20Disclosures) This section is not applicable to the company - This item is not applicable[366](index=366&type=chunk) Part II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=67&type=section&id=Item%205%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company's common stock trades on Nasdaq under 'TGTX', with no cash dividends ever paid or anticipated in the foreseeable future - The company's common stock is listed on the Nasdaq Capital Market under the symbol **'TGTX'**[368](index=368&type=chunk) - No cash dividends have been declared or paid, nor are any anticipated in the foreseeable future[370](index=370&type=chunk) [Removed and Reserved](index=68&type=section&id=Item%206%20Removed%20and%20Reserved) This item has been intentionally removed and is reserved - This item is noted as 'REMOVED AND RESERVED'[376](index=376&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=69&type=section&id=Item%207%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) In 2023, the company achieved profitability with **$12.7 million** net income, driven by **$233.7 million** in revenue from **BRIUMVI** sales and a Neuraxpharm upfront payment Financial Performance Comparison (2023 vs. 2022) | (in thousands) | 2023 (in thousands) | 2022 (in thousands) | | :--- | :--- | :--- | | **Total Revenue** | **$233,662** | **$2,785** | | Product revenue, net | $92,005 | $2,633 | | License, milestone and other revenue | $141,657 | $152 | | **Total costs and expenses** | **$213,029** | **$195,624** | | Research and development | $76,192 | $125,352 | | Selling, general and administrative | $122,706 | $70,007 | | **Net income (loss)** | **$12,672** | **($198,335)** | - The significant **revenue increase in 2023** was primarily due to the U.S. commercial launch of **BRIUMVI** and a **$140.0 million** upfront payment from Neuraxpharm[389](index=389&type=chunk)[390](index=390&type=chunk) - Research and development expenses decreased by **$48.9 million** in 2023, primarily due to reduced manufacturing, lower license milestones, and clinical trial costs[393](index=393&type=chunk) - As of December 31, 2023, the company held **$217.5 million** in cash and investments, providing sufficient liquidity for over twelve months[409](index=409&type=chunk) [Quantitative and Qualitative Disclosure About Market Risk](index=78&type=section&id=Item%207A%20Quantitative%20and%20Qualitative%20Disclosure%20About%20Market%20Risk) The company's primary market risk is interest rate fluctuations on its short-term investment portfolio, which management deems not material - The company's main market risk stems from interest rate changes affecting its financial instrument portfolio[438](index=438&type=chunk) - Due to the short-term nature of investments, no material exposure to interest rate or credit risk is believed to exist[438](index=438&type=chunk) [Financial Statements and Supplementary Data](index=78&type=section&id=Item%208%20Financial%20Statements%20and%20Supplementary%20Data) This item incorporates by reference the company's consolidated financial statements and related notes from Part IV of the annual report - The company's consolidated financial statements and notes are incorporated by reference[439](index=439&type=chunk) [Changes in and Disagreements With Accountants on Accounting and Financial Disclosures](index=78&type=section&id=Item%209%20Changes%20in%20and%20Disagreements%20With%20Accountants%20on%20Accounting%20and%20Financial%20Disclosures) This section is not applicable to the company for the reporting period - This section is not applicable[440](index=440&type=chunk) [Controls and Procedures](index=78&type=section&id=Item%209A%20Controls%20and%20Procedures) As of December 31, 2023, management concluded that disclosure controls and internal control over financial reporting were effective - Management concluded that disclosure controls and procedures were effective as of December 31, 2023[441](index=441&type=chunk) - Internal control over financial reporting was deemed effective as of December 31, 2023, audited by KPMG LLP[442](index=442&type=chunk)[443](index=443&type=chunk) [Other Information](index=78&type=section&id=Item%209B%20Other%20Information) No directors or executive officers adopted or terminated Rule 10b5-1 trading plans during the fourth quarter of 2023 - No directors or executive officers adopted or terminated Rule 10b5-1 trading arrangements in Q4 2023[445](index=445&type=chunk) Part III [Directors, Executive Officers and Corporate Governance](index=79&type=section&id=Item%2010%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) Information on directors, executive officers, and corporate governance is incorporated by reference from the 2024 Proxy Statement - Information for this item is incorporated by reference from the company's 2024 Proxy Statement[446](index=446&type=chunk) [Executive Compensation](index=79&type=section&id=Item%2011%20Executive%20Compensation) Executive compensation details are incorporated by reference from the company's 2024 Proxy Statement - Information for this item is incorporated by reference from the company's 2024 Proxy Statement[447](index=447&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=79&type=section&id=Item%2012%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) Security ownership information for beneficial owners and management is incorporated by reference from the 2024 Proxy Statement - Information for this item is incorporated by reference from the company's 2024 Proxy Statement[448](index=448&type=chunk) [Certain Relationships and Related Transactions, and Director Independence](index=79&type=section&id=Item%2013%20Certain%20Relationships%20and%20Related%20Transactions%2C%20and%20Director%20Independence) Information on certain relationships, related transactions, and director independence is incorporated by reference from the 2024 Proxy Statement - Information for this item is incorporated by reference from the company's 2024 Proxy Statement[449](index=449&type=chunk) [Principal Accounting Fees and Services](index=79&type=section&id=Item%2014%20Principal%20Accounting%20Fees%20and%20Services) Details on principal accounting fees and services are incorporated by reference from the company's 2024 Proxy Statement - Information for this item is incorporated by reference from the company's 2024 Proxy Statement[450](index=450&type=chunk) Part IV [Exhibits and Financial Statement Schedules](index=80&type=section&id=Item%2015%20Exhibits%20and%20Financial%20Statement%20Schedules) This section lists the consolidated financial statements, schedules, and exhibits filed with the annual report, including auditor reports and financial statements - This item lists the consolidated financial statements of TG Therapeutics, Inc. filed as part of the report[451](index=451&type=chunk) - All financial statement schedules are omitted as information is either inapplicable or included in financial statements or notes[453](index=453&type=chunk) - A comprehensive list of exhibits filed with the report is provided, including governance documents and certifications[454](index=454&type=chunk)

Financial Data and Key Metrics Changes - For the fourth quarter of 2023, U.S. BRIUMVI net product revenue was $39.9 million, representing a 60% growth quarter-over-quarter, contributing to a full year revenue of approximately $234 million [41][59][62] - The company reported a net income of $12.7 million for the full year 2023, with a net loss of $14.4 million for the fourth quarter [61][62] - Operating expenses for the fourth quarter and full year were $56 million and $213 million respectively, with cash operating expenditures of approximately $47 million and $175 million [60][62] Business Line Data and Key Metrics Changes - BRIUMVI's sales momentum resulted in approximately $90 million in U.S. net revenue for its first partial year of sales [15][23] - The company reported approximately 3,200 new patient prescriptions through its hub in 2023, translating to about 3,500 total new patient scripts [43] - The fourth quarter saw a significant increase in prescriptions from major academic centers, with more scripts coming from academic centers than private practices for the first time [42] Market Data and Key Metrics Changes - The CD20 market continues to grow, capturing approximately 50% to 55% share of new patients annually, with an estimated 40,000 patients starting CD20 therapy each year [48][50] - The company estimates a 10% market share based on approximately 1,000 prescriptions received in the fourth quarter [50] Company Strategy and Development Direction - The company aims to become the number one prescribed CD20 therapy by market share, focusing on differentiating BRIUMVI through its unique attributes and patient convenience [24][25][36] - Plans include developing a subcutaneous (subcu) formulation of BRIUMVI and expanding its use beyond multiple sclerosis (MS) into other autoimmune diseases [33][34][36] - The company has recently acquired a worldwide license for an allogeneic CD19 CAR T cell therapy program, which is expected to enhance its pipeline [36] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong early demand for BRIUMVI, targeting $220 million to $260 million in U.S. sales revenue for 2024 [23][54] - The company is focused on lifecycle management activities for BRIUMVI and believes recent patent issuances extending protection through 2042 will provide a long runway for growth [28][116] Other Important Information - The company has received three additional patents for BRIUMVI, enhancing its competitive position and extending patent protection through 2042 [28][102] - The company ended 2023 with approximately $217.5 million in cash and equivalents, providing sufficient capital to fund operations towards cash flow positivity [62] Q&A Session Summary Question: What can we expect on growth-to-net cadence for the balance of the year? - Management indicated that while there will be variability quarter-to-quarter, they have not provided precise guidance for 2024 [71] Question: Do you have any numbers on the switches from prior therapies and how that's impacting your sales growth? - Management noted that the largest group of patients switching to BRIUMVI are those previously treated but naive to CD20 therapy, with a balanced distribution across patient types [75][76] Question: Can you give us your thoughts on the launch in Germany and in other countries? - Management highlighted the importance of launching in Germany as the largest market in Europe and mentioned that other countries will follow later this year into next year [78] Question: How should we be thinking about the growth or the ramp of SG&A expenses? - Management confirmed that most of the expansion in the sales force is already incorporated into the current operating expense estimates for 2024 [80] Question: What timeframe do you think it's possible to achieve the number one prescribed IV CD20 for BRIUMVI by dynamic share? - Management has not set a specific timeframe but is working towards this goal, indicating that 2024 may not be the year to achieve it [85] Question: How important is it to demonstrate efficacy via relapse rate compared to demonstrating comparability on bioequivalence for subcu BRIUMVI? - Management believes that a bioequivalence study will be sufficient, similar to competitors, without needing a full efficacy study [106]

Exhibit 99.1 TG Therapeutics Provides Business Update and Reports Fourth Quarter and Year-End 2023 Financial Results Fourth quarter and full year 2023 BRIUMVI U.S. net revenue of approximately $40 million and $89 million, respectively Conference call to be held today, February 28, 2024, at 8:30 AM ET New York, NY, (February 28, 2024) – TG Therapeutics, Inc. (NASDAQ: TGTX) (the Company or TG Therapeutics) today announced its financial results for the fourth quarter and year ended December 31, 2023, along wit ...

[Special Cautionary Notice Regarding Forward-Looking Statements](index=4&type=section&id=SPECIAL%20CAUTIONARY%20NOTICE%20REGARDING%20FORWARD-LOOKING%20STATEMENTS) This section cautions that forward-looking statements involve known and unknown risks and are not guarantees of future performance - The company identifies several areas covered by forward-looking statements, including but not limited to[10](index=10&type=chunk) - Ability to obtain and maintain regulatory approvals for product candidates like TG-1701 and TG-1801, and maintain approval for BRIUMVI® - Success of the commercial launch and market acceptance of BRIUMVI® - Progress and results of pre-clinical studies and clinical trials - Estimates of expenses, future revenues, capital requirements, and financial performance - Ability to obtain sufficient capital and manage cash burn [Summary Risk Factors](index=5&type=section&id=SUMMARY%20RISK%20FACTORS) This section summarizes the principal risks impacting the company's operations, financial condition, and future prospects [Risks Related to Commercialization](index=5&type=section&id=Risks%20Related%20to%20Commercialization) This section outlines key risks associated with product commercialization, including market acceptance, intense competition, and potential for limited revenue generation - The company has limited experience as a commercial entity, which may impact the success of the BRIUMVI launch[14](index=14&type=chunk) - Market acceptance of BRIUMVI is not guaranteed and depends on factors like physician and patient adoption, pricing, and reimbursement[14](index=14&type=chunk) - Substantial competition exists from companies with greater resources, which could reduce or eliminate the commercial opportunity for the company's products[14](index=14&type=chunk) [Risks Related to Drug Development and Regulatory Approval](index=5&type=section&id=Risks%20Related%20to%20Drug%20Development%20and%20Regulatory%20Approval) This section details risks inherent in drug development and regulatory processes, including approval uncertainties, potential side effects, and the non-predictive nature of early trial results - Failure to obtain or maintain regulatory approval for products like BRIUMVI or candidates like TG-1701 and TG-1801 would materially harm the business[14](index=14&type=chunk) - Undesirable side effects could delay or prevent regulatory approval, or lead to market withdrawal if discovered post-approval[14](index=14&type=chunk) - Positive results from early-stage clinical trials do not guarantee favorable results in later, more definitive trials[14](index=14&type=chunk) [Risks Related to Dependence on Third Parties](index=6&type=section&id=Risks%20Related%20to%20our%20Dependence%20on%20Third%20Parties) This section summarizes risks from heavy reliance on third parties for manufacturing, clinical trials, and in-licensing agreements, which could disrupt operations - Reliance on third parties for manufacturing and supply of products and raw materials creates a risk of insufficient quantities or quality, which could impair commercialization[18](index=18&type=chunk) - The company depends on third parties to conduct clinical trials; poor performance by these parties could jeopardize regulatory approval[18](index=18&type=chunk) - Since products are in-licensed, any disputes or non-performance by licensors could adversely affect the ability to develop and commercialize them[18](index=18&type=chunk) [Risks Related to Intellectual Property](index=6&type=section&id=Risks%20Related%20to%20Intellectual%20Property) This section covers risks related to intellectual property, including the ability to obtain and protect patents, and potential infringement lawsuits - The company's success is dependent on its ability to obtain and protect its intellectual property. If patent protection is not sufficiently broad, competitors could develop similar products[16](index=16&type=chunk) - Lawsuits alleging infringement of third-party intellectual property rights could be costly and time-consuming, with an unfavorable outcome having a material adverse effect[16](index=16&type=chunk) [Risks Related to Financial Position and Need for Additional Capital](index=6&type=section&id=Risks%20Related%20to%20our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) This section highlights financial risks, including a history of operating losses, potential need for additional capital, and the adverse effects of indebtedness - The company has a history of significant operating losses and may incur losses in the future[17](index=17&type=chunk) - While not currently expected, the company may need to raise additional capital. An inability to do so could force a reduction in development or commercialization efforts[17](index=17&type=chunk) - The company's level of debt could adversely affect its financial condition and make it more difficult to fund operations[17](index=17&type=chunk) [PART I: FINANCIAL INFORMATION](index=7&type=section&id=PART%20I%20FINANCIAL%20INFORMATION) This section presents the company's unaudited condensed consolidated financial statements and management's discussion and analysis of financial condition and results of operations [Item 1. Financial Statements](index=7&type=section&id=Item%201%20Financial%20Statements%3A) This section presents the unaudited condensed consolidated financial statements, including balance sheets, statements of operations, cash flows, and notes on key accounting policies [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The balance sheet shows a significant increase in total assets to **$331.1 million** driven by cash, receivables, and inventory, alongside an increase in total liabilities due to higher loan payable Balance Sheet Highlights (in thousands) | Metric | Sep 30, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $150,902 | $102,304 | | Accounts receivable, net | $39,320 | $0 | | Inventories | $33,553 | $0 | | **Total Assets** | **$331,067** | **$193,572** | | **Liabilities & Equity** | | | | Loan payable – non-current | $98,908 | $71,135 | | Total Liabilities | $166,298 | $134,985 | | Total Stockholders' Equity | $164,769 | $58,587 | [Condensed Consolidated Statements of Operations](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20(unaudited)) The company achieved significant profitability in Q3 2023 with **$113.9 million** net income, driven by **$140.7 million** license revenue and **$25.1 million** product sales Q3 2023 vs Q3 2022 Performance (in thousands) | Metric | Q3 2023 | Q3 2022 | | :--- | :--- | :--- | | Product revenue, net | $25,068 | $56 | | License, milestone and other revenue | $140,747 | $38 | | **Total Revenue** | **$165,815** | **$94** | | Total costs and expenses | $51,031 | $35,057 | | **Operating Income (Loss)** | **$114,784** | **($34,963)** | | **Net Income (Loss)** | **$113,930** | **($35,818)** | | Diluted EPS | $0.73 | ($0.26) | Nine Months 2023 vs 2022 Performance (in thousands) | Metric | Nine Months 2023 | Nine Months 2022 | | :--- | :--- | :--- | | Product revenue, net | $48,868 | $2,591 | | License, milestone and other revenue | $140,823 | $114 | | **Total Revenue** | **$189,691** | **$2,705** | | Total costs and expenses | $156,573 | $143,482 | | **Operating Income (Loss)** | **$33,118** | **($140,777)** | | **Net Income (Loss)** | **$27,088** | **($145,341)** | | Diluted EPS | $0.19 | ($1.08) | [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows%20(unaudited)) Net cash used in operating activities significantly decreased to **$18.2 million**, while financing activities provided **$72.7 million**, resulting in a **$48.6 million** net increase in cash for the nine months ended September 30, 2023 Cash Flow Summary (Nine Months Ended Sep 30, in thousands) | Cash Flow Activity | 2023 | 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($18,203) | ($152,300) | | Net cash used in investing activities | ($5,896) | ($36,282) | | Net cash provided by (used in) financing activities | $72,706 | ($440) | | **Net Increase (Decrease) in Cash** | **$48,607** | **($189,022)** | [Notes to Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements%20(unaudited)) These notes detail the company's commercial transition, the **$140 million** Neuraxpharm agreement, liquidity, and key accounting policies for revenue, inventory, and debt - The company is now a fully-integrated, commercial-stage biopharmaceutical company following the FDA, EC, and MHRA approvals of BRIUMVI for relapsing forms of multiple sclerosis (RMS)[32](index=32&type=chunk) - The company achieved profitability in Q3 2023 due to a **$140.0 million** non-refundable upfront payment from a commercialization agreement with Neuraxpharm[35](index=35&type=chunk)[70](index=70&type=chunk) - As of September 30, 2023, the company had **$229.2 million** in cash, cash equivalents, and investment securities, which is believed to be sufficient to fund operations for at least the next twelve months[37](index=37&type=chunk)[156](index=156&type=chunk) - On March 31, 2023, the company amended its loan agreement with Hercules, drawing an additional **$25.0 million**. The total loan payable as of September 30, 2023 was **$98.9 million**[92](index=92&type=chunk)[99](index=99&type=chunk) - Costs to manufacture BRIUMVI are now capitalized as inventory following its FDA approval on December 28, 2022. Prior to this date, these costs were expensed as R&D[61](index=61&type=chunk)[76](index=76&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=29&type=section&id=Item%202%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's financial condition and results, highlighting BRIUMVI's successful launch, the Neuraxpharm agreement, and a shift to profitability [Overview and Recent Business Updates](index=29&type=section&id=Overview%20and%20Recent%20Business%20Updates) This section provides an overview of the company's transition to a commercial-stage entity, highlighting BRIUMVI's regulatory approvals and the Neuraxpharm commercialization agreement - Announced an agreement with Neuraxpharm on August 1, 2023, for the ex-U.S. commercialization of BRIUMVI[120](index=120&type=chunk)[128](index=128&type=chunk) - Received approval for BRIUMVI from the UK's MHRA on November 1, 2023, for adult patients with active RMS[120](index=120&type=chunk)[131](index=131&type=chunk) - The U.S. commercial launch of BRIUMVI began on January 26, 2023, with the first patient receiving an infusion on February 1, 2023[126](index=126&type=chunk)[129](index=129&type=chunk) [Results of Operations](index=32&type=section&id=Results%20of%20Operations) Analysis of operations shows dramatic financial improvement, with total revenue surging to **$165.8 million** in Q3 2023, driven by license fees and product sales, resulting in **$113.9 million** net income Comparison of Three Months Ended September 30 (in thousands) | Item | 2023 | 2022 | Change | | :--- | :--- | :--- | :--- | | **Total Revenue** | **$165,815** | **$94** | **+$165,721** | | Product revenue, net | $25,068 | $56 | +$25,012 | | License revenue | $140,747 | $38 | +$140,709 | | **Total R&D Expense** | **$14,753** | **$20,801** | **-$6,048** | | **Total SG&A Expense** | **$32,769** | **$14,254** | **+$18,515** | | **Net Income (Loss)** | **$113,930** | **($35,818)** | **+$149,748** | Comparison of Nine Months Ended September 30 (in thousands) | Item | 2023 | 2022 | Change | | :--- | :--- | :--- | :--- | | **Total Revenue** | **$189,691** | **$2,705** | **+$186,986** | | Product revenue, net | $48,868 | $2,591 | +$46,277 | | License revenue | $140,823 | $114 | +$140,709 | | **Total R&D Expense** | **$58,743** | **$95,717** | **-$36,974** | | **Total SG&A Expense** | **$91,553** | **$47,503** | **+$44,050** | | **Net Income (Loss)** | **$27,088** | **($145,341)** | **+$172,429** | [Liquidity and Capital Resources](index=35&type=section&id=Liquidity%20and%20Capital%20Resources) Liquidity has strengthened due to equity offerings, debt financing, and the Neuraxpharm payment, with **$229.2 million** in cash and investments deemed sufficient for the next twelve months - Major sources of cash have been equity offerings, debt financing from Hercules, and the upfront payment from the Neuraxpharm Commercialization Agreement[153](index=153&type=chunk) - As of September 30, 2023, the company had **$229.2 million** in cash, cash equivalents, and investment securities[156](index=156&type=chunk) - Management believes existing cash and projected revenues are sufficient to fund operating requirements for at least twelve months from the filing date[156](index=156&type=chunk) Cash Flow Changes (Nine Months Ended Sep 30, in thousands) | Cash Flow Activity | 2023 | 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($18,203) | ($152,300) | | Net cash provided by (used in) financing activities | $72,706 | ($440) | [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=38&type=section&id=Item%203%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company reports no material changes in its market risk exposure since the Annual Report on Form 10-K for the year ended December 31, 2022 - There have been no material changes in the company's market risk exposure since the end of 2022[164](index=164&type=chunk) [Item 4. Controls and Procedures](index=38&type=section&id=Item%204%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of September 30, 2023, with no material changes to internal control over financial reporting during the quarter - The CEO and CFO concluded that as of September 30, 2023, the company's disclosure controls and procedures were effective[165](index=165&type=chunk) - No changes in internal control over financial reporting occurred during the quarter that materially affected, or are reasonably likely to materially affect, these controls[166](index=166&type=chunk) [PART II: OTHER INFORMATION](index=38&type=section&id=PART%20II%20OTHER%20INFORMATION) This section provides additional information, including legal proceedings, detailed risk factors, and other disclosures not covered in the financial information section [Item 1. Legal Proceedings](index=38&type=section&id=Item%201%20Legal%20Proceedings) The company reports no material pending legal proceedings involving itself, its subsidiaries, or its property - The company is not currently involved in any material legal proceedings[167](index=167&type=chunk) [Item 1A. Risk Factors](index=39&type=section&id=Item%201A%20Risk%20Factors) This section extensively discusses risks and uncertainties that could materially harm the company, categorized by commercialization, financial position, drug development, third-party dependence, and intellectual property [Risks Related to Commercialization](index=39&type=section&id=Risks%20Related%20to%20Commercialization) This subsection details risks associated with product commercialization, including market acceptance, intense competition, and securing adequate reimbursement - The success of BRIUMVI is uncertain and depends on market acceptance, which is influenced by efficacy, safety, pricing, reimbursement, and competition[171](index=171&type=chunk) - The company faces substantial competition from well-resourced pharmaceutical companies, which may commercialize drugs more successfully[183](index=183&type=chunk) - The ability to commercialize products depends on securing adequate coverage and reimbursement from government and private payors, who are increasingly focused on cost containment[188](index=188&type=chunk) [Risks Related to Financial Position and Need for Additional Capital](index=45&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) This part outlines financial vulnerabilities, including a history of operating losses, potential need for additional capital, and risks associated with indebtedness and restrictive covenants - The company has a history of significant operating losses and expects to continue incurring significant expenses for commercialization and R&D[198](index=198&type=chunk) - While not currently anticipated, a future need to raise capital may arise. Failure to secure funding could require delaying or reducing programs[202](index=202&type=chunk) - The company's debt obligations under its agreement with Hercules could adversely affect its financial condition and restrict operations due to covenants[211](index=211&type=chunk)[212](index=212&type=chunk) [Risks Related to Drug Development and Regulatory Approval](index=50&type=section&id=Risks%20Related%20to%20Drug%20Development%20and%20Regulatory%20Approval) This subsection covers inherent uncertainties of drug development, including high clinical trial failure rates, non-predictive early results, potential side effects, and lengthy regulatory processes - Early-stage clinical trial results are not predictive of later-stage outcomes, and product candidates face a high rate of failure[220](index=220&type=chunk) - Clinical drug development is a long, expensive, and uncertain process, with potential delays in trial completion and enrollment[227](index=227&type=chunk) - Undesirable side effects discovered during trials could delay or prevent regulatory approval, while those found after approval could impact commercial potential[237](index=237&type=chunk) [Risks Related to Dependence on Third Parties](index=66&type=section&id=Risks%20Related%20to%20Our%20Dependence%20on%20Third%20Parties) This subsection details significant risks from reliance on third parties for clinical trials, manufacturing (including single sources), and in-licensing agreements - The company relies on CROs to conduct clinical trials; failure by these CROs to perform as required could delay or terminate trials[295](index=295&type=chunk) - The company depends on third-party CMOs for manufacturing, including a single source for BRIUMVI supply, which increases the risk of supply disruptions[299](index=299&type=chunk) - As products are in-licensed, any dispute with or non-performance by licensors could adversely affect the ability to commercialize the products[310](index=310&type=chunk) [Risks Relating to Intellectual Property](index=72&type=section&id=Risks%20Relating%20to%20Our%20Intellectual%20Property) This subsection discusses critical intellectual property risks, including obtaining and defending patents, potential litigation, and protecting trade secrets - Commercial success depends on obtaining and maintaining patent protection, but there is no guarantee that patent applications will issue or that issued patents will provide sufficient protection[323](index=323&type=chunk)[324](index=324&type=chunk) - The company may be involved in expensive and time-consuming lawsuits to protect its patents or defend against claims of infringing third-party patents[346](index=346&type=chunk)[353](index=353&type=chunk) - In addition to patents, the company relies on trade secrets, which are vulnerable to unauthorized disclosure or independent discovery by competitors[360](index=360&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=89&type=section&id=Item%202%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This item is reported as not applicable for the period - Not applicable[397](index=397&type=chunk) [Item 5. Other Information](index=89&type=section&id=Item%205%20Other%20Information) The company reports no adoption or termination of Rule 10b5-1 trading plans by directors or executive officers during Q3 2023 - No directors or executive officers adopted or terminated a Rule 10b5-1 trading plan during the three months ended September 30, 2023[400](index=400&type=chunk) [Item 6. Exhibits](index=90&type=section&id=Item%206%20Exhibits) This section lists exhibits filed with the Form 10-Q, including CEO and CFO certifications and financial statements in iXBRL format - Exhibits filed include CEO and CFO certifications pursuant to Sections 302 and 906 of the Sarbanes-Oxley Act[404](index=404&type=chunk) - The filing also includes financial information formatted in Inline Extensible Business Reporting Language (iXBRL)[404](index=404&type=chunk)