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PTC Therapeutics(PTCT) - 2024 Q1 - Earnings Call Transcript
2024-04-26 03:52
Financial Data and Key Metrics Changes - Total revenue for the first quarter was $210 million, with DMD franchise revenue of $161 million, reflecting strong commercial performance [5][20][42] - Non-GAAP R&D expenses decreased to $107 million from $180 million year-over-year, indicating strategic portfolio prioritization [21] - Cash, cash equivalents, and marketable securities totaled $885 million as of March 31, 2024, up from $877 million at the end of 2023, providing a strong balance sheet for future initiatives [43] Business Line Data and Key Metrics Changes - Translarna generated $104 million in revenue during the quarter, while Emflaza net product revenue was $57 million, demonstrating effective brand protection strategies [37][42] - The global customer-facing team delivered $178 million in revenue from five marketed products, with a focus on growth and diversification [15] Market Data and Key Metrics Changes - The company is expanding its geographic presence in Latin America and the Middle East, with ongoing regulatory filings and approvals anticipated for products like Tegsedi and Waylivra [19] - The European market continues to be a significant contributor to Translarna revenue, accounting for approximately 45% of total revenue, consistent with the previous year [80] Company Strategy and Development Direction - The company plans to submit the NDA for sepiapterin in mid-2024 and for vatiquinone in late 2024, aiming to address significant unmet needs in the PKU and DMD markets [13][33] - The strategy includes maximizing Translarna revenue in Europe while preparing for the U.S. launch of Upstaza, the first approved gene therapy for AADC patients [39] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving all planned regulatory and clinical milestones for 2024, highlighting a strong start to the year [12][35] - There is significant anticipation for the potential availability of Translarna in the U.S. market, with positive feedback from physicians and patients regarding its efficacy [54][80] Other Important Information - The company is actively working on regulatory submissions in Brazil and Japan for sepiapterin, with a focus on meeting the needs of PKU patients globally [38] - The CARDINAL study for utreloxastat in ALS patients is on schedule to report top-line results in the fourth quarter of 2024, with expectations for positive outcomes [14] Q&A Session Summary Question: What work has been done around Europe, Japan, and Brazil regarding upcoming filings? - The company has established commercial infrastructure in these regions and is actively engaging with treatment centers and patient communities to prepare for launches [24] Question: How much did the EU contribute to Translarna revenue this quarter? - The contribution from the EU is consistent with the first quarter of last year, representing around 45% of total revenue [80] Question: Can you provide insights on reimbursement progress in Europe for AADC gene therapy? - The company is seeing favorable pricing and reimbursement negotiations in Europe, which are expected to carry over into the U.S. market upon launch [84] Question: What are the expectations for the efficacy of the Huntington study? - The company aims for a statistically significant benefit on the ALSFRS scale, with a clinically meaningful effect defined as a 2.5 point difference between treatment and placebo groups [104] Question: What is the status of Translarna in Europe? - The European Commission has not yet adopted the CHMP opinion, and the company is actively ensuring patient access to Translarna while awaiting further updates [88]
PTC Therapeutics(PTCT) - 2024 Q1 - Quarterly Report
2024-04-25 20:36
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 March 31, 2024 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 (Stat ...
PTC Therapeutics(PTCT) - 2024 Q1 - Quarterly Results
2024-04-25 20:10
Exhibit 99.1 PTC Therapeutics Provides Corporate Update and Reports First Quarter 2024 Financial Results WARREN, N.J., April 25, 2024 – PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ending March 31, 2024. "We are off to a strong start in 2024, with outstanding commercial performance and achievement of all planned clinical and regulatory milestones for the first quarter," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutic ...
PTC Therapeutics(PTCT) - 2023 Q4 - Earnings Call Transcript
2024-03-01 03:48
PTC Therapeutics, Inc. (NASDAQ:PTCT) Q4 2023 Earnings Conference Call February 28, 2024 4:30 PM ET Company Participants Ron Aldridge - Senior Director, IR Matthew Klein - CEO Eric Pauwels - CBO Kylie O'Keefe - CCO Pierre Gravier - CFO Conference Call Participants Brooke Schuster - William Blair Rick Miller - Cantor Fitzgerald Eric Joseph - JPMorgan Jeff Hung - Morgan Stanley Brian Abrahams - RBC Capital Markets Peyton Bohnsack - TD Cowen David Lebowitz - Citi Gena Wang - Barclays Paul Choi - Goldman Sachs J ...
PTC Therapeutics(PTCT) - 2023 Q4 - Annual Report
2024-02-29 21:36
Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended: December 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number: 001-35969 PTC THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 04-3416587 (State or other jurisdiction of incorporati ...
PTC Therapeutics(PTCT) - 2023 Q3 - Earnings Call Transcript
2023-10-29 10:11
PTC Therapeutics, Inc. (NASDAQ:PTCT) Q3 2023 Earnings Conference Call February 8, 2023 9:00 AM ET Company Participants Jane Hanlon - Associate Director, Investor Relations Matthew Klein - Chief Executive Officer Eric Pauwels - Chief Business Officer Kylie O'Keefe - Chief Commercial Officer Pierre Gravier - Chief Financial Officer Conference Call Participants Eric Joseph - J.P. Morgan Kristen Kluska - Cantor Fitzgerald Samantha Corwin - William Blair David Lebowitz - Citi Brian Abrahams - RBC Capital Markets ...
PTC Therapeutics(PTCT) - 2023 Q3 - Quarterly Report
2023-10-26 20:34
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 September 30, 2023 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) (State or other juris ...
PTC Therapeutics(PTCT) - 2023 Q2 - Earnings Call Transcript
2023-08-04 04:05
Financial Data and Key Metrics Changes - The company reported total revenue of $214 million for the second quarter of 2023, representing a 29% increase compared to the same quarter in 2022 [46] - The DMD franchise revenue totaled $162 million, reflecting a 21% year-over-year growth [55][75] - Non-GAAP R&D expenses were $170 million for the second quarter of 2023, up from $143 million in the same quarter of 2022 [90] Business Line Data and Key Metrics Changes - Translarna generated $96 million in revenue, a 25% increase over the same quarter in 2022 [84] - Emflaza achieved net product revenue of $66 million, representing a 16% growth compared to the second quarter of 2022 [85] - The APHENITY trial for sepiapterin in PKU patients met its primary endpoint with a 63% reduction in blood phenylalanine levels [77] Market Data and Key Metrics Changes - Evrysdi's global revenue for the second quarter was approximately $380 million, with PTC earning $37 million in royalties [63] - The company expects continued growth in the Latin American and Eastern European markets, despite unpredictability in large government orders [28][56] Company Strategy and Development Direction - The company is focused on launching sepiapterin in the US and Europe, with expectations of a $1 billion-plus global commercial opportunity [49][88] - A strategic portfolio review has led to a reduction in operational expenses, with anticipated savings of approximately $150 million in 2024 [76] - The company plans to submit an NDA for sepiapterin in the fourth quarter of 2023 following a pre-NDA meeting with the FDA [77] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving the revenue guidance of $940 million to $1 billion for 2023, which would represent a year-over-year growth of 34% to 43% [75][116] - The company remains optimistic about the potential for Translarna to convert from conditional to standard marketing authorization in Europe [11][52] - Management highlighted the importance of the upright stability subscale in the mFARS scale as a predictor of time to loss of ambulation in Friedreich ataxia [120] Other Important Information - The company has approximately $338 million in cash, cash equivalents, and marketable securities as of June 30, 2023 [117] - The company is well-positioned for multiple product launches, leveraging its existing infrastructure and expertise [123] Q&A Session Summary Question: Clarity on CHMP opinion for Translarna - Management explained that the initial timing for the CHMP opinion was pushed to the third quarter due to additional questions during the review process [10] Question: Capital deployment strategy with $330 million in cash - Management indicated that they are well-capitalized to support upcoming product launches and maintain a robust discovery and development infrastructure [13] Question: Main questions for the PKU pre-NDA meeting - The focus will be on the structure of the NDA and ensuring alignment with the FDA on clinical and safety data [18] Question: Potential decline in Duchenne revenue guidance - Management acknowledged the unpredictability of large orders but expressed confidence in achieving growth despite potential fluctuations [30] Question: Overlap in physician communities for Huntington's disease - Management noted the recognition of the splicing platform's power and the importance of leveraging learnings from previous product launches [20]
PTC Therapeutics(PTCT) - 2023 Q2 - Quarterly Report
2023-08-03 20:34
Financial Performance - Translarna generated $96.5 million in net sales during the quarter ended June 30, 2023[214]. - Emflaza recognized $65.7 million in net sales for the same quarter[214]. - Net product revenues for the three months ended June 30, 2023, were $174.6 million, an increase of $30.9 million, or 21%, from $143.7 million for the same period in 2022, driven by sales of Translarna and Emflaza[278]. - Net product sales for the three months ended June 30, 2023, were $108.9 million outside the U.S., up from $86.9 million in 2022, with Translarna contributing $96.5 million[248]. - Net product sales in the United States for the six months ended June 30, 2023, were $120.3 million, compared to $105.4 million in 2022, solely from Emflaza sales[250]. - Net product revenues for the six months ended June 30, 2023, were $362.1 million, an increase of $88.6 million, or 32%, from $273.5 million for the same period in 2022, driven by Translarna and Emflaza sales[294]. - Royalty revenue increased to $36.9 million for the three months ended June 30, 2023, up $15.0 million, or 69%, from $21.8 million in the same period of 2022, primarily due to higher Evrysdi sales[279]. - Royalty revenue for the six months ended June 30, 2023, was $67.7 million, an increase of $27.0 million, or 66%, from $40.7 million for the same period in 2022, due to higher Evrysdi sales[297]. Research and Development - Sepiapterin achieved a 63% reduction in blood Phe levels in the overall primary analysis population during its Phase 3 trial[228]. - PTC518 demonstrated a mean 30% reduction in mutant HTT levels at the 10mg dose level in its Phase 2 study[226]. - Vatiquinone did not meet its primary endpoint in the Phase 3 trial for Friedreich ataxia, but showed significant benefits in secondary endpoints and was well tolerated[229]. - The Phase 2/3 trial of vatiquinone for children with mitochondrial disease associated seizures was deprioritized after failing to achieve its primary endpoint[229]. - Utreloxastat was well tolerated in a Phase 1 trial and a Phase 2 trial for amyotrophic lateral sclerosis is ongoing[229]. - Unesbulin is in a registration-directed Phase 2/3 trial for leiomyosarcoma, with plans for a similar trial for diffuse intrinsic pontine glioma[230]. - Research and development expenses for the six months ended June 30, 2023, totaled $380.998 million, an increase from $297.341 million in 2022[268]. - Research and development expenses were $185.9 million for the three months ended June 30, 2023, an increase of $28.6 million, or 18%, from $157.3 million for the same period in 2022, related to the advancement of the clinical pipeline[285]. Regulatory and Market Authorization - Upstaza received marketing authorization in the EEA for patients 18 months and older in July 2022[221]. - Evrysdi was approved by the FDA for SMA treatment in August 2020 and received a label expansion for infants under two months in May 2022[225]. - Translarna's marketing authorization in the EEA is subject to annual review and renewal, with a decision expected in Q3 2023[215]. - The company is preparing to submit a BLA for Upstaza to the FDA in Q3 2023[223]. - The conditional marketing authorization for Translarna is pending the completion of a Type II variation process[217]. - The company anticipates submitting a BLA to the FDA for Upstaza in Q3 2023 and an NDA for sepiapterin in Q4 2023, pending FDA feedback[327]. Financial Position and Cash Flow - As of June 30, 2023, the company reported a net loss of $337.8 million, compared to a net loss of $278.8 million for the same period in 2022[241]. - The company has an accumulated deficit of $2,994.8 million as of June 30, 2023[241]. - Cash, cash equivalents, and marketable securities totaled $337.9 million as of June 30, 2023[321]. - Net cash used in operating activities was $43.6 million for the six months ended June 30, 2023, compared to $152.6 million for the same period in 2022[324]. - Net cash used in investing activities was $52.7 million for the six months ended June 30, 2023, compared to a net cash provided of $121.3 million for the same period in 2022[325]. Strategic Initiatives and Workforce - The company announced an 8% workforce reduction in May 2023 as part of a strategic pipeline prioritization[232]. - The company is focused on maintaining orphan exclusivity in the U.S. for Emflaza and completing post-marketing requirements imposed by regulatory agencies[337]. - The company is advancing its splicing, metabolic, Bio-e, and oncology programs, with ongoing studies for maintaining authorizations and label extensions[337]. - The company is exploring collaborations, including with Roche and the SMA Foundation, to obtain research funding and achieve milestones[337]. Risks and Future Outlook - The company has never been profitable and will need to generate significant revenues to achieve and sustain profitability, requiring substantial additional funding for continuing operations[341]. - The company anticipates financing cash needs primarily through equity offerings, debt financings, collaborations, and grants, with potential dilution of shareholder ownership interest[341]. - The company faces risks related to unexpected decreases in revenue or increases in expenses due to the COVID-19 pandemic or other contagious disease outbreaks[337]. - The company must satisfy obligations under the Blackstone Credit Agreement and the indentures governing the 2026 Convertible Notes, with annual cash interest payments of $4.3 million required[336].
PTC Therapeutics (PTCT) Update / Briefing Transcript
2023-07-19 17:02
Summary of PTC Therapeutics PKU Deep Dive Conference Call Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on innovative therapies for rare disorders, with a robust commercial portfolio of six marketed products, five of which are global, and one generating collaboration and royalty revenue [5][6] - The company aims to achieve total revenue guidance of $940 million to $1 billion for 2023, including $545 million to $565 million from DMD revenue [6] Product Focus: Sepiaterin for PKU - The primary focus of the call is on sepiaterin, a treatment for phenylketonuria (PKU) [8] - The Phase 3 APHINITY trial showed a 63% reduction in blood phenylalanine levels in the overall primary analysis population and 69% in classical PKU patients, with a p-value of less than 0.0001, indicating statistical significance [9][10] - 84% of patients achieved target phenylalanine levels below 360 micromoles per liter, and 93% of adolescents and adults achieved levels below 600 micromoles per liter [10] - Sepiaterin was well tolerated with no serious adverse events reported [9] Clinical Insights - PKU is caused by variants in the PAH gene, leading to impaired PAH function and elevated phenylalanine levels, which can have neurological consequences [17][19] - Current treatments, Kuvan and Palynziq, have limitations, including low response rates and safety concerns, highlighting the need for new therapies like sepiaterin [30][32][39] - Sepiaterin offers mechanistic advantages, including better absorption and bioavailability compared to existing treatments, and has shown potential to treat a broader patient population, including those with classical PKU [35][39] Unmet Needs and Market Opportunity - Less than 10% of diagnosed PKU patients are well-controlled on Kuvan, with around 70% of patients trialed on Kuvan not responding adequately [41] - The patient journey begins with newborn screening, and the need for lifelong dietary restrictions significantly impacts quality of life, leading to social isolation and mental health issues [42][43][44] - The potential market opportunity for sepiaterin is estimated to be between $1 billion and $1.5 billion, targeting 15% to 30% of the global PKU population [62][73] Commercial Strategy - PTC Therapeutics has established relationships with key opinion leaders and treatment centers, positioning itself for a successful launch of sepiaterin [63][68] - The company plans to differentiate sepiaterin through its data package, mechanistic advantages, and support for the PKU community [72] - The launch strategy includes targeting therapy-naive patients, those who have failed Kuvan, and patients inadequately controlled on existing therapies [61][70] Conclusion - Sepiaterin represents a significant advancement in the treatment of PKU, with strong clinical data supporting its efficacy and safety [39][60] - The company is preparing for regulatory submissions and anticipates a successful commercial launch, aiming to address the substantial unmet needs in the PKU patient population [73]