PTC Therapeutics(PTCT)

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PTC Therapeutics (PTCT) Update / Briefing Transcript
2023-05-23 22:02
Summary of PTC Therapeutics (PTCT) Update / Briefing May 23, 2023 Company Overview - **Company**: PTC Therapeutics (PTCT) - **Focus**: Development of therapies for rare diseases, specifically targeting Friedreich ataxia (FA) with the drug vutiquinone Key Points from the Call Study Results - **MoveFA Study**: A 72-week placebo-controlled trial of vutiquinone for Friedreich ataxia patients aged 7 to 21 years [3][4] - **Primary Endpoint**: Change in total modified Friedreich ataxia rating scale (mFARS) score from baseline to week 72; did not achieve statistical significance [7][12] - **Secondary Endpoints**: Notable treatment benefits observed in bulbar function and upright stability subscales with nominal p-values of 0.044 and 0.021 respectively [7][12] - **Fatigue Scale**: Significant improvement noted, as fatigue is a major symptom for FA patients [8][12] - **Completer Analysis**: Showed a 2.31 placebo-corrected difference in mFARS score, indicating a 75% slowing of disease progression over 72 weeks [9][10] Safety Profile - **Adverse Events**: Similar profiles between vutiquinone and placebo groups; common events included falls and gastrointestinal symptoms [11][12] - **Serious Events**: One death in each group, with the vutiquinone-related death attributed to cardiac failure [12] Regulatory and Strategic Decisions - **FDA and EMA Discussions**: Plans to share study results with regulatory agencies to explore potential approval pathways for pediatric FA patients [13] - **Portfolio Prioritization**: Decision to discontinue preclinical gene therapy programs and suspend development for certain gene therapy programs, resulting in a 15% reduction in operational expenses for 2023 [13][45] Financial Outlook - **Revenue Guidance**: No changes to revenue expectations; projected revenue remains between $940 million to $1 billion [41][45] - **Operational Expenses**: Focus on reducing OpEx while maintaining investment in promising R&D programs [75][78] Future Directions - **Open Label Extension Study**: Plans to analyze long-term effects beyond the 72-week study period [28][92] - **Subgroup Analysis**: Noted a larger treatment effect in younger patients (aged 16 and below) with a 2.38 improvement in mFARS score [94] Additional Insights - **Natural History Data**: The study's findings align with existing natural history data, reinforcing the drug's potential to modify disease progression [30][92] - **Regulatory Flexibility**: The company aims to leverage insights from other recent FDA approvals in rare diseases to support their case for vutiquinone [80][81] Conclusion - PTC Therapeutics is navigating a complex landscape in rare disease drug development, with promising data from the MoveFA study despite not meeting the primary endpoint. The company is strategically refocusing its resources and engaging with regulatory bodies to explore potential pathways for approval, particularly for pediatric patients with Friedreich ataxia.
PTC Therapeutics (PTCT) Update / Briefing Transcript
2023-05-17 13:02
Summary of PTC Therapeutics (PTCT) Update / Briefing May 17, 2023 Company Overview - **Company**: PTC Therapeutics (PTCT) - **Product**: Sepiaterin - **Indication**: Treatment of Phenylketonuria (PKU) Key Points Industry and Product Development - **Phase III Trial**: The AFFINITY trial for sepiaterin in patients with fetal ketonuria has shown positive top-line results, meeting the primary endpoint of blood phenylalanine reduction [2][19] - **Efficacy Results**: - Overall mean blood phenylalanine reduction of **63%** in the primary analysis population [2][8] - In classical PKU patients, the mean reduction was **69%** [2][8] - **84%** of patients achieved the target phenylalanine level of less than **360 micromoles per liter** [10] - **93%** of adolescents and adults met the European guideline target of below **600 micromoles per liter** [10] Trial Design and Results - **Trial Structure**: The trial included a run-in phase where patients were treated with sepiaterin for two weeks before randomization [3][4] - **Patient Population**: - Total of **156 patients** screened, with **103 patients** showing a reduction of greater than **30%** in blood phenylalanine levels [4][6] - **98 patients** were randomized for the primary analysis [6] - **Safety Profile**: - No serious adverse events reported; common adverse events included headache and diarrhea, mostly grade one [12][13][78] - Similar rates of treatment-related adverse events between sepiaterin and placebo groups [13][78] Market Opportunity - **Commercial Potential**: - PKU represents a unique commercial opportunity with an estimated **58,000 patients** worldwide [17] - Despite two approved therapies, a large unmet need exists, particularly for therapy-naive patients and those poorly controlled on existing treatments [17][18] - **Regulatory Strategy**: Plans to request pre-submission meetings with regulatory authorities and move forward with NDA and MAA submissions [19] Competitive Landscape - **Comparison with Kuvan**: - In a subset of patients previously treated with Kuvan, sepiaterin showed a **48%** reduction in blood phenylalanine levels compared to their baseline on Kuvan [11][25] - The trial results validate the superior efficacy of sepiaterin over Kuvan, with a higher percentage of patients achieving significant benefits [25][26] Future Directions - **Long-term Studies**: Ongoing long-term open-label studies to assess safety and durability of sepiaterin treatment [14][19] - **Phe Tolerance Protocol**: Preliminary data show that patients can tolerate increased phenylalanine intake while maintaining low blood levels [16][104] Conclusion - The AFFINITY trial results position sepiaterin as a promising treatment for PKU, with strong efficacy and safety data supporting its potential to address significant unmet medical needs in the PKU patient population [19][121]
PTC Therapeutics(PTCT) - 2023 Q1 - Earnings Call Transcript
2023-04-28 05:20
Financial Data and Key Metrics Changes - Total revenue for Q1 2023 reached $220 million, marking a 40% increase compared to Q1 2022, which is the highest quarterly revenue ever for the company [5][27] - The DMD franchise generated $170 million in revenue, reflecting a 33% increase year-over-year [5][24] - Evrysdi contributed $31 million in royalty revenue, with growth driven by both therapy-naive patients and those previously treated with other therapies [5][6] - The company expects total revenue for 2023 to be between $940 million and $1 billion, indicating potential growth of up to 43% year-over-year [6][27] - Non-GAAP SG&A expenses were $73.4 million for Q1 2023, compared to $59.7 million in Q1 2022 [28] Business Line Data and Key Metrics Changes - Translarna net product revenues were $115.1 million, with strong growth across all geographies [27] - Emflaza had net product revenues of $54.6 million, representing a 12% growth compared to Q1 2022 [27] - The DMD franchise is expected to achieve revenue guidance of $545 million to $565 million for 2023, supported by new patient starts and improved payer access [43][53] Market Data and Key Metrics Changes - The company is expanding its geographic presence, particularly in Latin America and Asia, with successful launches of Waylivra in Brazil and ongoing growth in the DMD franchise [23][24] - The company anticipates a strong rollout of Upstaza in Europe, with positive guidance received from NICE in England and Wales for AADC patients aged 18 months and older [45] Company Strategy and Development Direction - The company aims to leverage its strong commercial portfolio to drive growth in neurological and metabolic disorders [22] - A strategic portfolio review and potential operational expense reduction are expected based on study results to be reported in Q2 2023 [6] - The company is focused on expanding its clinical pipeline, with results from four clinical studies anticipated in the second quarter [7][10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving the 2023 revenue guidance, citing strong fundamentals and execution across all geographies [22][26] - The company is optimistic about the upcoming clinical trial results and their potential impact on future product launches and market expansion [21][48] Other Important Information - The company reported cash, cash equivalents, and marketable securities totaling approximately $286.3 million as of March 31, 2023, down from $410.7 million at the end of 2022 [28] - Non-GAAP R&D expenses for Q1 2023 were $179.8 million, reflecting increased investment in research programs and clinical pipeline advancement [48] Q&A Session Summary Question: What has driven new patient starts in the DMD portfolio? - Management highlighted geographic expansion, early diagnosis, and rapid treatment initiation as key drivers of growth in new patient starts [31][53] Question: What are the expectations for the APHENITY trial? - The primary analysis population includes subjects with a greater than 30% reduction in phenylalanine levels, with confidence in achieving significant results based on prior data [90][91] Question: How does the company view the competitive landscape for vatiquinone? - The company is exploring ways to strengthen and extend the patent life for vatiquinone, primarily relying on orphan exclusivity [62] Question: What is the confidence level regarding the MOVE-FA study's statistical significance? - Management believes there is sufficient power to detect differences similar to those observed in previous studies, with confidence in capturing treatment benefits [118] Question: What are the implications of the FDA's queries regarding the BLA submission? - The company anticipates potential delays in the BLA submission to Q3 2023 due to ongoing interactions with the FDA [97]
PTC Therapeutics(PTCT) - 2023 Q1 - Quarterly Report
2023-04-27 20:35
PART I—FINANCIAL INFORMATION This section provides the unaudited financial statements, management's discussion and analysis, market risk disclosures, and controls and procedures for the first quarter of 2023 [Item 1. Financial Statements (unaudited)](index=9&type=section&id=Item%201.%20Financial%20Statements%20(unaudited)) This section presents the unaudited consolidated financial statements for PTC Therapeutics, Inc. as of March 31, 2023, including balance sheets, statements of operations, cash flows, and comprehensive notes [Consolidated Financial Statements](index=9&type=section&id=Consolidated%20Financial%20Statements) The consolidated financial statements show a net loss of **$139.0 million** for Q1 2023, with total assets decreasing to **$1.61 billion** and **$29.5 million** cash used in operations Consolidated Statements of Operations (Q1 2023 vs Q1 2022) | Metric | Three Months Ended March 31, 2023 (in thousands) | Three Months Ended March 31, 2022 (in thousands) | | :--- | :--- | :--- | | **Total revenues** | **$220,382** | **$148,735** | | Net product revenue | $187,557 | $129,832 | | Royalty revenue | $30,831 | $18,896 | | **Total operating expenses** | **$337,997** | **$235,257** | | Research and development | $195,124 | $140,078 | | Selling, general and administrative | $86,914 | $73,271 | | **Loss from operations** | **($117,615)** | **($86,522)** | | **Net loss** | **($138,959)** | **($126,726)** | | **Net loss per share—basic and diluted** | **($1.88)** | **($1.78)** | Consolidated Balance Sheet Highlights | Metric | March 31, 2023 (in thousands) | December 31, 2022 (in thousands) | | :--- | :--- | :--- | | Cash and cash equivalents | $167,495 | $279,834 | | Total current assets | $613,392 | $693,785 | | **Total assets** | **$1,608,839** | **$1,705,619** | | Total current liabilities | $441,634 | $406,236 | | Long-term debt | $572,091 | $571,722 | | **Total liabilities** | **$2,066,400** | **$2,052,705** | | **Total stockholders' deficit** | **($457,561)** | **($347,086)** | Consolidated Statements of Cash Flows Highlights (Q1 2023) | Cash Flow Activity | Three Months Ended March 31, 2023 (in thousands) | | :--- | :--- | | Net cash used in operating activities | ($29,491) | | Net cash used in investing activities | ($28,971) | | Net cash provided by financing activities | $4,094 | | **Net decrease in cash and cash equivalents** | **($62,331)** | [Notes to Consolidated Financial Statements](index=14&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) The notes detail accounting policies, revenue recognition, debt, and commitments, highlighting an accumulated deficit of **$2.8 billion** and reliance on product sales and financing for R&D - The company's portfolio includes commercial products **Translarna** and **Emflaza** for DMD, **Upstaza** for AADC deficiency, and a broad pipeline in neurology, metabolism, and oncology[29](index=29&type=chunk)[30](index=30&type=chunk)[31](index=31&type=chunk) - As of March 31, 2023, the company reported an accumulated deficit of approximately **$2.8 billion**, with current cash and expected product sales deemed sufficient for the next twelve months[44](index=44&type=chunk) Net Product Sales by Geography and Product (Q1 2023 vs Q1 2022) | Category | Q1 2023 (in millions) | Q1 2022 (in millions) | | :--- | :--- | :--- | | **Net Product Sales (U.S.)** | **$54.6** | **$48.6** | | *Emflaza* | *$54.6* | *$48.6* | | **Net Product Sales (ex-U.S.)** | **$133.0** | **$81.2** | | *Translarna* | *$115.1* | *$79.2* | | *Tegsedi, Waylivra, Upstaza* | *$17.9* | *$2.0* | - Royalty revenue from Evrysdi, in collaboration with Roche, increased to **$30.8 million** in Q1 2023 from **$18.9 million** in Q1 2022[72](index=72&type=chunk)[197](index=197&type=chunk) - Significant future commitments include potential milestone payments for acquisitions: up to **$331 million** (development/regulatory) and **$150 million** (sales) to Agilis, up to **$200 million** to BioElectron, and up to **$217.5 million** to Censa for two lead programs[181](index=181&type=chunk)[183](index=183&type=chunk)[184](index=184&type=chunk) - In February 2023, a **$30.0 million** development milestone for the sepiapterin program (Censa acquisition) was triggered, payable primarily in common stock shares[185](index=185&type=chunk) - As of March 31, 2023, the company holds **$283.3 million** in 1.50% convertible senior notes due 2026 and a **$300.0 million** senior secured term loan from Blackstone[152](index=152&type=chunk)[167](index=167&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=72&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses a **44%** increase in net product revenue to **$187.6 million** in Q1 2023, total revenues reaching **$220.4 million**, and a net loss of **$139.0 million**, while affirming sufficient liquidity for the next twelve months [Corporate Updates and Pipeline](index=72&type=section&id=Corporate%20Updates%20and%20Pipeline) PTC reports strong Q1 2023 sales for **Translarna** (**$115.1 million**) and **Emflaza** (**$54.6 million**), with key clinical data readouts for **PTC518**, **vatiquinone**, and **sepiapterin** expected in Q2 2023, and a **Upstaza** BLA submission anticipated in Q3 2023 - The company anticipates an EMA CHMP opinion in Q2 2023 concerning the conversion of **Translarna's** conditional marketing authorization to a standard one[217](index=217&type=chunk) - The BLA submission for **Upstaza** in the U.S. is now projected for Q3 2023, a delay from Q2 2023, due to ongoing FDA bioanalytical data queries[223](index=223&type=chunk)[244](index=244&type=chunk) - Key clinical data readouts are expected in Q2 2023 for **PTC518** (Huntington's disease Phase 2), **vatiquinone** (mitochondrial disease Phase 2/3), and **vatiquinone** (Friedreich ataxia Phase 3)[226](index=226&type=chunk)[229](index=229&type=chunk) - Results from Part 2 of the registration-directed Phase 3 trial for **sepiapterin** for PKU are anticipated in May 2023[230](index=230&type=chunk) [Results of Operations](index=90&type=section&id=Results%20of%20Operations) Total revenues for Q1 2023 increased **48%** to **$220.4 million**, driven by product and royalty revenue growth, while operating expenses rose **44%** to **$338.0 million**, resulting in a net loss of **$139.0 million** Q1 2023 vs. Q1 2022 Financial Results (in thousands) | Line Item | Q1 2023 | Q1 2022 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | **Total Revenues** | **$220,382** | **$148,735** | **$71,647** | **48.2%** | | Net product revenue | $187,557 | $129,832 | $57,725 | 44.5% | | Royalty revenue | $30,831 | $18,896 | $11,935 | 63.2% | | **Total Operating Expenses** | **$337,997** | **$235,257** | **$102,740** | **43.7%** | | R&D Expense | $195,124 | $140,078 | $55,046 | 39.3% | | SG&A Expense | $86,914 | $73,271 | $13,643 | 18.6% | | **Net Loss** | **($138,959)** | **($126,726)** | **($12,233)** | **9.7%** | - The increase in R&D expenses primarily resulted from increased investment in research programs and clinical pipeline advancement, including a **$30.0 million** success-based development milestone for the **sepiapterin** for PKU trial[284](index=284&type=chunk) - The rise in SG&A expenses reflects ongoing investment to support the company's expanding commercial portfolio and activities[285](index=285&type=chunk) - Other income totaled **$10.0 million** compared to an **$11.9 million** expense in the prior year, primarily due to unrealized foreign exchange gains and equity investment gains in Q1 2023[289](index=289&type=chunk) [Liquidity and Capital Resources](index=94&type=section&id=Liquidity%20and%20Capital%20Resources) As of March 31, 2023, the company held **$286.3 million** in cash and equivalents, used **$29.5 million** in operating activities, and believes existing capital is sufficient for the next twelve months despite anticipating continued high expenses - The company held cash, cash equivalents, and marketable securities totaling **$286.3 million** as of March 31, 2023[302](index=302&type=chunk) - The company believes existing cash and expected cash flows from product sales will sufficiently fund operations and capital expenditures for at least the next twelve months[310](index=310&type=chunk) - Significant upcoming cash outlays include a potential **$20.0 million** milestone payment to former Agilis equityholders upon FDA acceptance of the **Upstaza** BLA and **$50.0 million** in potential milestones to former Censa securityholders in 2023[317](index=317&type=chunk) - The company funded a **$50.0 million** reserve account during Q1 2023 as mandated by the Blackstone Credit Agreement[301](index=301&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=103&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company reported no material changes in its market risk profile or management during Q1 2023 compared to its 2022 Annual Report on Form 10-K - No material changes occurred in the company's market risk profile during the first quarter of 2023[321](index=321&type=chunk) [Item 4. Controls and Procedures](index=103&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of March 31, 2023, with no material changes to internal control over financial reporting during the quarter - The CEO and CFO concluded that the company's disclosure controls and procedures were effective at a reasonable assurance level as of March 31, 2023[324](index=324&type=chunk) - No material changes to the company's internal control over financial reporting occurred during the first quarter of 2023[325](index=325&type=chunk) PART II—OTHER INFORMATION This section covers legal proceedings, risk factors, and a list of exhibits filed with the quarterly report [Item 1. Legal Proceedings](index=105&type=section&id=Item%201.%20Legal%20Proceedings) The company is subject to ordinary course legal claims but is not currently aware of any material legal proceedings against it - The company is not currently aware of any material legal proceedings to which it is a party[327](index=327&type=chunk) [Item 1A. Risk Factors](index=105&type=section&id=Item%201A.%20Risk%20Factors) This section refers readers to the comprehensive risk factors detailed in the company's Annual Report on Form 10-K for the year ended December 31, 2022 - The report directs readers to the risk factors detailed in the Annual Report on Form 10-K for the year ended December 31, 2022[328](index=328&type=chunk) [Item 6. Exhibits](index=106&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Quarterly Report on Form 10-Q, including agreements, CEO/CFO certifications, and Inline XBRL documents - Exhibits filed include a consulting agreement, CEO/CFO certifications under Sarbanes-Oxley, and XBRL data files[330](index=330&type=chunk)
PTC Therapeutics(PTCT) - 2022 Q4 - Earnings Call Transcript
2023-02-22 03:35
Financial Data and Key Metrics Changes - The company achieved total revenue of $699 million in 2022, representing a 30% growth over 2021, despite significant foreign exchange headwinds [169] - For Q4 2022, total revenue was $167 million, consisting of $128 million from net product revenue and $40 million from Evrysdi royalty revenue [14][169] - Non-GAAP R&D expenses for Q4 2022 were $175 million, up from $136 million in Q4 2021, reflecting increased investment in research programs [41] Business Line Data and Key Metrics Changes - The DMD franchise generated $507 million in revenue for 2022, a 20% year-over-year growth, with Q4 revenue of $114 million [162] - Translarna achieved $56 million in revenue for Q4 2022, totaling $289 million for the year, marking a 22% growth [12][178] - Emflaza reported Q4 revenue of $58 million, bringing total annual revenue to $218 million, a 17% increase over 2021 [207] Market Data and Key Metrics Changes - The company is expanding its geographic presence in Latin America, the Middle East, and Asia Pacific, with ongoing efforts to launch Upstaza and grow the DMD franchise [12][38] - The company anticipates significant growth in 2023, with revenue guidance set between $940 million to $1 billion, reflecting over 30% year-over-year growth [155] Company Strategy and Development Direction - The company aims to deliver one new therapy every two to three years, focusing on rare diseases by modulating gene and protein expression [168] - The company is preparing for the launch of Upstaza in the US, with a dedicated team focusing on disease awareness and key publications [125] - The company plans to request a Type C meeting with the FDA to discuss the potential path for NDA resubmission for Translarna [160] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong performance and growth trajectory for 2023, highlighting the excitement around upcoming clinical trial readouts [4][118] - The company noted the importance of addressing the unmet medical needs of patients, particularly in the PKU and mitochondrial disease markets [6][50] Other Important Information - The company was awarded the 2023 EURORDIS Black Pearl Company Award for Innovation, recognizing its contributions to rare disease research [154] - The company has initiated additional dosing cohorts in the PTC518 Huntington's disease program, with results expected in the second quarter of 2023 [36] Q&A Session Summary Question: Can you break down revenue components in your full year 2023 revenue guidance? - The company expects revenue guidance of $940 million to $1 billion, reflecting growth in the DMD franchise, Upstaza, and Evrysdi, including a $100 million milestone for Evrysdi [43] Question: Can you clarify the nature of your discussions with the agency on Translarna? - The company had an informal meeting with the FDA and plans to request another Type C meeting to review the totality of data collected [55][160] Question: What is the expected reduction in seizure frequency considered clinically meaningful for the MIT-E study? - The study is powered for a 40% differential reduction between treatment and placebo groups, with a hypothesized reduction of 50% in the active population [65] Question: Can you provide an update on the APHENITY study? - The company reported that 156 subjects passed screening, with 102 achieving greater than 30% reduction in phenylalanine levels, indicating a high responder rate [203] Question: What is the rationale for dose escalation in the Part 2 study? - The company plans to utilize a two-week dose escalation approach to assess the safety and efficacy of higher doses in the study [134]
PTC Therapeutics(PTCT) - 2022 Q4 - Annual Report
2023-02-21 21:50
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(PTCT) - 2022 Q3 - Earnings Call Transcript
2022-10-28 02:15
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 ...
PTC Therapeutics(PTCT) - 2022 Q3 - Quarterly Report
2022-10-27 20:38
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 Therapeutics (PTCT) Investor Presentation - Slideshow
2022-09-16 22:10
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(PTCT) - 2022 Q2 - Earnings Call Transcript
2022-08-07 12:02
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 ...