Part I [Business](index=12&type=section&id=Item%201.%20Business) PTC Therapeutics is a global biopharmaceutical company focused on rare disorders, with a commercial portfolio led by its DMD franchise and a diversified pipeline across multiple therapeutic platforms - The company's strategy focuses on leveraging its scientific expertise and global commercial infrastructure to discover, develop, and commercialize treatments for rare diseases with high unmet medical needs[31](index=31&type=chunk) Key Commercial Products Overview | Product | Indication | 2022 Net Sales/Revenue | Key Markets/Status | | :--- | :--- | :--- | :--- | | **Translarna™** | nonsense mutation DMD (nmDMD) | $288.6 million | Approved in EEA, Russia, Brazil. Investigational in the U.S. | | **Emflaza®** | Duchenne muscular dystrophy (DMD) | $218.3 million | Approved in the U.S. | | **Upstaza™** | AADC deficiency | Revenue recognition started in 2022 | Approved in EEA & UK. BLA submission to FDA planned for H1 2023. | | **Evrysdi®** | Spinal Muscular Atrophy (SMA) | Royalty-generating | Marketed by Roche; PTC receives tiered royalties. | - PTC maintains a diversified development pipeline across multiple technology platforms: - **Splicing Platform:** Includes PTC518 for Huntington's disease (Phase 2 data expected Q2 2023) - **Bio-e Platform:** Vatiquinone for mitochondrial disease and Friedreich ataxia (Phase 2/3 results expected Q2 2023) - **Metabolic Platform:** Sepiapterin for PKU (Phase 3 results expected May 2023) - **Gene Therapy Platform:** Includes assets for Friedreich ataxia and Angelman syndrome - **Oncology Platform:** Unesbulin for leiomyosarcoma (Phase 2/3 ongoing)[36](index=36&type=chunk)[37](index=37&type=chunk) [Global Commercial Footprint](index=16&type=section&id=Item%201.%20Business-Global%20Commercial%20Footprint) PTC's commercial portfolio is anchored by its Global DMD Franchise, complemented by the newly launched Upstaza, Latin American rights, and Evrysdi royalties - Translarna's marketing authorization in the EEA is conditional, subject to annual renewal and the submission of results from Study 041. A Type II variation to convert to standard marketing authorization was submitted in September 2022, with a CHMP opinion expected in H1 2023[42](index=42&type=chunk)[44](index=44&type=chunk) - Upstaza™, a gene therapy for AADC deficiency, was approved by the European Commission in July 2022 and in the UK in November 2022. The company plans to submit a Biologics License Application (BLA) to the U.S. FDA in the first half of 2023[54](index=54&type=chunk)[68](index=68&type=chunk) - PTC began commercial sales of Tegsedi® for hATTR amyloidosis and Waylivra® for FCS in Brazil in 2022, leveraging its commercialization rights in Latin America and the Caribbean[73](index=73&type=chunk)[78](index=78&type=chunk) - Evrysdi® for SMA, developed in collaboration with Roche, is a key revenue driver through royalties. It has received broad approval, including a label expansion in the U.S. for infants under two months old in May 2022[81](index=81&type=chunk) [Diversified Development Pipeline](index=28&type=section&id=Item%201.%20Business-Diversified%20Development%20Pipeline) PTC's diversified pipeline features multiple late-stage clinical programs across five platforms, with key data readouts anticipated in 2023 - The Splicing Platform's lead candidate, PTC518 for Huntington's disease, is in a Phase 2 study. Data from the initial 12-week phase is expected in **Q2 2023**. U.S. enrollment is currently paused pending additional data requested by the FDA[87](index=87&type=chunk) - The Bio-e Platform has two registration-directed trials for vatiquinone, with results for both mitochondrial disease associated seizures (MIT-E trial) and Friedreich ataxia (MOVE-FA trial) anticipated in **Q2 2023**[93](index=93&type=chunk)[94](index=94&type=chunk) - The Metabolic Platform's lead asset, sepiapterin, is in a Phase 3 trial for PKU. Results from Part 2 of the trial are now expected in **May 2023** due to over-enrollment[98](index=98&type=chunk) - The Oncology Platform is advancing unesbulin, with a registration-directed Phase 2/3 trial for leiomyosarcoma (LMS) ongoing and a similar trial for diffuse intrinsic pontine glioma (DIPG) expected to initiate in **Q4 2023**[100](index=100&type=chunk)[101](index=101&type=chunk) [Collaborations, Acquisitions, and Intellectual Property](index=50&type=section&id=Item%201.%20Business-Collaborations%2C%20Acquisitions%2C%20and%20Intellectual%20Property) PTC leverages strategic collaborations, manages significant contingent payment obligations from acquisitions, and maintains a patent portfolio with key expirations in 2024 and beyond - The collaboration with Roche for the SMA program entitles PTC to tiered royalties (**8-16%**) on worldwide net sales of Evrysdi and potential sales milestones up to **$250.0 million**. In July 2020, PTC sold **42.933%** of its royalty rights to Royalty Pharma for **$650.0 million**[173](index=173&type=chunk)[174](index=174&type=chunk)[175](index=175&type=chunk) - The company has significant ongoing contingent payment obligations from past acquisitions, including up to **$311.0 million** in regulatory milestones for the Agilis acquisition and up to **$217.5 million** in development/regulatory milestones for the Censa acquisition[216](index=216&type=chunk)[222](index=222&type=chunk) - As of January 31, 2023, PTC's patent portfolio included **143 active U.S. patents**. Key patents for ataluren (Translarna) expire in **2024** (composition of matter) and **2026-2027** (methods of use). For Emflaza, the company relies on Orphan Drug Act exclusivity, which expires in **February 2024** for patients 5+ and **June 2026** for patients 2-5[224](index=224&type=chunk)[227](index=227&type=chunk)[608](index=608&type=chunk) [Manufacturing, Commercial Matters, and Competition](index=68&type=section&id=Item%201.%20Business-Manufacturing%2C%20Commercial%20Matters%2C%20and%20Competition) PTC relies on third-party manufacturing, manages global commercial sales subject to distributor concentration and country-specific pricing, and faces significant competition across its product portfolio - The company relies on third parties for manufacturing, packaging, and distribution of its products. It has a single source for some raw materials and for Emflaza drug substance, creating potential supply chain risks[236](index=236&type=chunk)[237](index=237&type=chunk)[241](index=241&type=chunk) - PTC operates a biologics manufacturing facility in Hopewell, NJ, to produce clinical materials for some gene therapy candidates and to provide manufacturing services for external customers[249](index=249&type=chunk)[250](index=250&type=chunk) - In 2022, two distributors each accounted for over **10%** of net product sales. Revenue from certain countries, like Brazil and Russia, is subject to fluctuations due to centralized group purchasing orders for multiple months of therapy[256](index=256&type=chunk)[258](index=258&type=chunk) - The company faces significant competition for its DMD franchise from Sarepta's approved exon-skipping therapies and multiple gene therapies in development from Pfizer, Solid Biosciences, and Sarepta[277](index=277&type=chunk)[508](index=508&type=chunk) [Government Regulation](index=80&type=section&id=Item%201.%20Business-Government%20Regulation) PTC operates under extensive global regulatory oversight, utilizing expedited pathways like Orphan Drug Designation, and faces significant pricing and reimbursement challenges, including the impact of the Inflation Reduction Act - The FDA and EMA provide several expedited review programs for serious conditions, including Fast Track, Priority Review, and Accelerated Approval. PTC has utilized these pathways for its rare disease portfolio[299](index=299&type=chunk)[300](index=300&type=chunk)[301](index=301&type=chunk) - The company has received Orphan Drug Designation for multiple products, including Translarna, Emflaza, and Upstaza. This provides market exclusivity for **seven years** in the U.S. and **ten years** in the EU, which is critical for products with limited or no patent protection like Emflaza[334](index=334&type=chunk)[358](index=358&type=chunk) - In the EU, Translarna has a conditional marketing authorization, which requires annual renewal based on a reassessment of the benefit-risk balance by the EMA[368](index=368&type=chunk) - The company is subject to complex pharmaceutical pricing and reimbursement regulations globally. In the U.S., this includes Medicaid Drug Rebate Program, 340B pricing, and new price negotiation provisions under the Inflation Reduction Act of 2022 (IRA). In the EU, pricing is subject to governmental control on a country-by-country basis[390](index=390&type=chunk)[391](index=391&type=chunk)[645](index=645&type=chunk) [Risk Factors](index=128&type=section&id=Item%201A.%20Risk%20Factors) PTC faces significant risks in product commercialization, pipeline development, financial sustainability, regulatory approvals, and intellectual property protection, including conditional authorizations and intense competition - **Commercialization & Regulatory Risk:** A primary risk is the potential failure to renew the annual conditional marketing authorization for Translarna in the EEA, which is contingent on the EMA's reassessment of its benefit-risk profile and the results of Study 041. Loss of this authorization would materially harm revenue[477](index=477&type=chunk)[479](index=479&type=chunk)[480](index=480&type=chunk) - **Financial Risk:** The company has a history of significant operating losses (**$2.66 billion** accumulated deficit as of Dec 31, 2022) and expects to continue incurring substantial expenses. Future profitability is not guaranteed and depends on successful commercialization and pipeline advancement[539](index=539&type=chunk) - **Pipeline Development Risk:** There is a substantial risk of delays or failures in clinical trials and in obtaining regulatory approvals for product candidates. The FDA has previously disagreed with the company's interpretation of Translarna data, and securing approval for new candidates like Upstaza involves significant uncertainty[436](index=436&type=chunk)[439](index=439&type=chunk)[442](index=442&type=chunk) - **Competition & Market Access Risk:** The company faces intense competition from larger pharmaceutical companies with greater resources. Furthermore, its products are subject to unfavorable pricing regulations, third-party reimbursement challenges, and healthcare reform initiatives like the Inflation Reduction Act, which could limit profitability[506](index=506&type=chunk)[518](index=518&type=chunk) [Properties](index=119&type=section&id=Item%202.%20Properties) PTC Therapeutics primarily leases its operational facilities, including principal research and office spaces in South Plainfield, NJ, and other significant sites in New Jersey and internationally - The company's key properties are leased, not owned. Major facilities include: - **South Plainfield, NJ:** ~**126,000 sq. ft.** (Principal research/office) - **Bridgewater, NJ:** ~**103,000 sq. ft.** (Lab/office) - **Hopewell, NJ:** ~**220,500 sq. ft.** (Office/manufacturing/lab) - **Warren, NJ:** ~**360,000 sq. ft.** (Shell condition space for future development)[773](index=773&type=chunk) [Legal Proceedings](index=119&type=section&id=Item%203.%20Legal%20Proceedings) The company is not currently aware of any material legal proceedings against it or its property - As of the filing date, there are no material legal proceedings against the company[774](index=774&type=chunk) Part II [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=120&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) PTC Therapeutics reported significant revenue growth in FY2022 driven by product and royalty sales, but increased operating expenses led to a wider net loss, with current liquidity deemed sufficient for the next 12 months Financial Performance Summary (2022 vs. 2021) | Metric | FY 2022 | FY 2021 | Change | Change (%) | | :--- | :--- | :--- | :--- | :--- | | **Total Revenues** | **$698.8M** | **$538.6M** | **+$160.2M** | **+29.7%** | | Net Product Revenue | $535.2M | $428.9M | +$106.3M | +24.8% | | Royalty Revenue | $113.5M | $54.6M | +$58.9M | +107.9% | | **Total Operating Expenses** | **$1,146.2M** | **$913.0M** | **+$233.2M** | **+25.5%** | | R&D Expense | $651.5M | $540.7M | +$110.8M | +20.5% | | SG&A Expense | $326.0M | $285.8M | +$40.2M | +14.1% | | **Net Loss** | **($559.0M)** | **($523.9M)** | **-$35.1M** | **+6.7%** | - The **25%** increase in net product revenue was driven by a **22%** rise in Translarna sales (**$288.6 million**) due to geographic expansion and a **17%** increase in Emflaza sales (**$218.3 million**) from new patient prescriptions and high compliance[856](index=856&type=chunk) - Research and development expenses increased by **$110.8 million** (**20%**) in 2022, primarily due to increased investment in research programs and the advancement of the clinical pipeline across multiple platforms[863](index=863&type=chunk) - As of December 31, 2022, the company had **$410.7 million** in cash, cash equivalents, and marketable securities. Management believes this is sufficient to fund operations and capital expenditures for at least the next **12 months**[908](index=908&type=chunk)[915](index=915&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=142&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) PTC is exposed to market risks from interest rate fluctuations on its investments and foreign currency exchange rates from international operations, but does not currently use hedging instruments - The primary market risk is interest rate sensitivity on its **$410.7 million** portfolio of cash, cash equivalents, and short-term investments. A hypothetical **10%** increase in interest rates is not expected to have a significant impact on the fair value of these securities[936](index=936&type=chunk)[937](index=937&type=chunk) - The company has exposure to foreign currency fluctuations (British Pound, Euro, Brazilian Real, Swiss Franc, Russian Ruble) from its international operations. For FY 2022, it recognized net realized foreign currency transaction losses of **$19.6 million**[939](index=939&type=chunk) - The company does not currently engage in hedging activities for interest rate or foreign currency risks but will evaluate the use of derivatives as needed[938](index=938&type=chunk)[939](index=939&type=chunk) [Financial Statements and Supplementary Data](index=144&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) The 2022 consolidated financial statements show total assets of **$1.71 billion**, a stockholders' deficit of **$347.1 million**, a net loss of **$559.0 million**, and an unqualified audit opinion Consolidated Balance Sheet Highlights (as of Dec 31, 2022) | Account | Amount (in thousands) | | :--- | :--- | | **Assets** | | | Cash, cash equivalents, and marketable securities | $410,705 | | Total Current Assets | $693,785 | | Intangible assets, net | $705,891 | | **Total Assets** | **$1,705,619** | | **Liabilities & Equity** | | | Total Current Liabilities | $406,236 | | Long-term debt | $571,722 | | Liability for sale of future royalties | $757,886 | | **Total Liabilities** | **$2,052,705** | | **Total Stockholders' (Deficit)** | **($347,086)** | Consolidated Statement of Operations (Year Ended Dec 31, 2022) | Line Item | Amount (in thousands) | | :--- | :--- | | Total Revenues | $698,801 | | Total Operating Expenses | $1,146,210 | | Loss from Operations | ($447,409) | | **Net Loss** | **($559,017)** | | **Net Loss Per Share** | **($7.79)** | Consolidated Statement of Cash Flows (Year Ended Dec 31, 2022) | Cash Flow Activity | Amount (in thousands) | | :--- | :--- | | Net cash used in operating activities | ($356,654) | | Net cash provided by investing activities | $290,181 | | Net cash provided by financing activities | $167,952 | | **Net increase in cash and cash equivalents** | **$98,707** | [Controls and Procedures](index=202&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded that disclosure controls and internal control over financial reporting were effective as of December 31, 2022, with an unqualified audit opinion from Ernst & Young LLP - Management, including the CEO and CFO, evaluated and concluded that the company's disclosure controls and procedures were effective as of December 31, 2022[1252](index=1252&type=chunk) - Based on the COSO 2013 framework, management assessed and concluded that the company's internal control over financial reporting was effective as of December 31, 2022[1255](index=1255&type=chunk) - Ernst & Young LLP, the independent auditor, provided an unqualified opinion on the effectiveness of the company's internal control over financial reporting as of December 31, 2022[1257](index=1257&type=chunk)[1260](index=1260&type=chunk) Part III [Directors, Executive Officers, Corporate Governance, Compensation, and Principal Accountant Fees](index=206&type=section&id=Items%2010-14) Information for Items 10-14, covering directors, executive compensation, and related matters, is incorporated by reference from the 2023 Proxy Statement - The information for Directors, Executive Officers, Corporate Governance (Item 10), Executive Compensation (Item 11), Security Ownership (Item 12), Certain Relationships and Related Transactions (Item 13), and Principal Accountant Fees and Services (Item 14) is incorporated by reference from the registrant's 2023 Proxy Statement[1272](index=1272&type=chunk)[1274](index=1274&type=chunk)[1275](index=1275&type=chunk)[1276](index=1276&type=chunk)[1277](index=1277&type=chunk) Part IV [Exhibits and Financial Statement Schedules](index=207&type=section&id=Item%2015.%20Exhibits%20and%20Financial%20Statement%20Schedules) This section lists financial statements from Item 8 and provides a comprehensive index of all exhibits filed with the Form 10-K, including key agreements and certifications - This item references the financial statements contained in Item 8 and provides a comprehensive Exhibit Index listing all required filings under Item 601 of Regulation S-K[1279](index=1279&type=chunk)[1280](index=1280&type=chunk)

PTC Therapeutics, Inc. (NASDAQ:PTCT) Q3 2022 Results Conference Call October 27, 2022 4:30 PM ET Company Participants Kylie O'Keefe - SVP, Head of Global Commercial and Corporate Strategy Stuart Peltz - Chief Executive Officer Matthew Klein - Chief Operating Officer Eric Pauwels - Chief Business Officer Emily Hill - Chief Financial Officer Conference Call Participants Kristen Kluska - Cantor Fitzgerald Eric Joseph - JPMorgan Joseph Thome - Cowen Alexander Xenakis - Truist Raju Prasad - William Blair Gena Wa ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) Commission file number: 001-35969 PTC Therapeutics, Inc. (Exact name of registrant as specified in its charter) (State or other jurisdiction of incorporation or organization) (I.R.S. Employer Identification No.) 100 Corporate Court South Plainfield, NJ 07080 (Address of principal executive offices) (Zip Code) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 ...

PTC 2022 Corporate Presentation September 2022 Forward-Looking Statements 2 This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements with respect to guidance relating to 2022 total revenue, 2022 DMD franchise net product revenue, 2022 operating expenditure guidance and future revenue guidance and s ...

PTC Therapeutics (NASDAQ:PTCT) Q2 2022 Earnings Conference Call August 4, 2022 4:30 PM ET Company Participants Kylie O'Keefe - Senior Vice President, Head of Global Commercial & Corporate Strategy Stuart Peltz - Chief Executive Officer Matthew Klein - Chief Operating Officer Eric Pauwels - Chief Business Officer Emily Hill - Chief Financial Officer Conference Call Participants Eric Joseph - JPMorgan Raju Prasad - William Blair Kristen Kluska - Cantor Fitzgerald Joe Thome - Cowen & Company Tazeen Ahmad - Ban ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-35969 PTC Therapeutics, Inc. (Exact name of registrant as specified in its charter) Delaware 04-3416587 (State ...

Translarna™ (Ataluren) Study 041 Topline Results 21st June 2022 Forward looking statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the commercialization of its products and product candidates ...

PTC 2022 Corporate Presentation May 2022 Forward Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements with respect to guidance relating to 2022 total net product revenue, 2022 DMD franchise net product revenue, 2022 operating expenditure guidance and future revenue guidance a ...

Start Time: 16:30 January 1, 0000 5:36 PM ET PTC Therapeutics, Inc. (NASDAQ:PTCT) Q1 2022 Earnings Conference Call May 03, 2022, 16:30 PM ET Company Participants Stuart Peltz - Founder and CEO Matthew Klein - COO Eric Pauwels - Chief Business Officer Emily Hill - CFO Kylie O’Keefe - SVP, Global Commercial and Corporate Strategy Conference Call Participants Eric Joseph - JPMorgan Rick Krause - Cantor Fitzgerald Brian Abrahams - RBC Capital Markets Joseph Thome - Cowen and Company Gena Wang - Barclays Robyn K ...

Financial Performance - Translarna generated $79.2 million in net sales during the quarter ended March 31, 2022[213]. - Emflaza achieved $48.6 million in net sales for the same quarter[213]. - For the three months ended March 31, 2022, net product sales outside of the United States were $81.2 million, compared to $47.8 million for the same period in 2021, representing a 70.0% increase[246]. - Net product sales in the United States for the same period were $48.6 million, up from $43.5 million in 2021, indicating an 11.8% growth[246]. - Net product revenue for the three months ended March 31, 2022, was $129.8 million, an increase of $38.6 million, or 42%, from $91.3 million for the same period in 2021[267]. - Translarna net product revenues were $79.2 million for the three months ended March 31, 2022, an increase of $32.7 million, or 70%, compared to $46.5 million for the same period in 2021[267]. - Emflaza net product revenues were $48.6 million for the three months ended March 31, 2022, an increase of $5.1 million, or 12%, compared to $43.5 million for the same period in 2021[267]. - Royalty revenue for the three months ended March 31, 2022, was $18.9 million, an increase of $12.2 million, or over 100%, from $6.7 million for the same period in 2021[269]. Research and Development - The company anticipates reporting results from the placebo-controlled trial of Study 041 by the end of Q2 2022[214]. - The company initiated a Phase 2 study of PTC518 for Huntington's disease in Q1 2022, expecting initial results by the end of 2022[222]. - A registration-directed Phase 3 trial for PTC923 for phenylketonuria was initiated in Q3 2021, with results expected by the end of 2022[227]. - The company completed a Phase 1 trial for PTC857, which was well-tolerated, and initiated a Phase 2 trial for amyotrophic lateral sclerosis in Q1 2022[226]. - The company expects to incur ongoing research and development expenses for its products and product candidates, including studies for emvododstat for COVID-19 and other therapeutic areas[238]. - Research and development expenses for the three months ended March 31, 2022, were $140.1 million, an increase of $5.6 million, or 4%, from $134.5 million for the same period in 2021[274]. Regulatory and Market Access - The marketing authorization for Translarna in the EEA is subject to annual review and renewal, with a renewal request submitted in February 2022[214]. - The company expects a regulatory decision on Waylivra for familial partial lipodystrophy in the second half of 2022[220]. - The company’s ability to generate revenue from Translarna is dependent on maintaining marketing authorizations and securing market access in various territories[283]. - The company plans to submit a BLA to the FDA for PTC-AADC in the third quarter of 2022[294]. Expenses and Financial Obligations - The company had an accumulated deficit of $2,224.7 million as of March 31, 2022, with a net loss of $126.7 million for the three months ended March 31, 2022[236]. - Selling, general and administrative expenses were $73.3 million for the three months ended March 31, 2022, an increase of $12.2 million, or 20%, from $61.1 million for the same period in 2021[275]. - Interest expense, net was $23.5 million for the three months ended March 31, 2022, an increase of $4.4 million, or 23%, from $19.2 million for the same period in 2021[277]. - The company expects selling, general and administrative expenses to increase in future periods due to ongoing commercialization efforts[261]. - The company anticipates significant expenses related to commercialization efforts in various regions, including the United States and EEA, and ongoing research and development costs[294]. - The company expects to pay $70.0 million to former equityholders of Agilis upon achieving certain milestones in 2022 related to PTC-AADC[306]. - The company may need to obtain substantial additional funding to sustain operations and achieve profitability[308]. Cash Flow and Financing - As of March 31, 2022, the company had cash, cash equivalents, and marketable securities totaling $587.8 million[290]. - Net cash used in operating activities was $97.4 million for the three months ended March 31, 2022, compared to $100.2 million for the same period in 2021[291]. - The company incurred net cash provided by investing activities of $49.0 million for the three months ended March 31, 2022, primarily due to net sales and redemption of marketable securities[292]. - The company closed a private offering of $150.0 million in aggregate principal amount of 3.00% convertible senior notes due 2022, receiving net proceeds of approximately $145.4 million after expenses[285]. - The company believes its cash flows from product sales and existing cash will be sufficient to fund operating expenses for at least the next twelve months[298]. Impact of COVID-19 - The COVID-19 pandemic has not significantly affected revenue generation, but may impact future revenue due to operational challenges[210]. Product Pipeline - The company has a pipeline of product candidates focused on multiple therapeutic areas, including rare diseases and oncology[230]. - The company may seek to expand its product pipeline through in-licensing or acquiring rights to new products, which could increase future capital requirements[239]. - The company expects to incur substantial costs in connection with regulatory submissions and commercialization efforts for its products[294].