Royalty Pharma will now own 100% of the 8% to 16% Evrysdi royalty following multiple transactions with PTC since 2020NEW YORK, Dec. 29, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) today announced that it has acquired the final portion of PTC Therapeutics’ remaining royalty on Roche’s Evrysdi for $240 million upfront and up to $60 million in sales-based milestones. Evrysdi, marketed by Roche, is an orally administered survival motor neuron-2 (SMN2) splicing modifier for the treatment of spinal ...

NEW YORK, Dec. 29, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) today announced that it has acquired the final portion of PTC Therapeutics’ remaining royalty on Roche’s Evrysdi for $240 million upfront and up to $60 million in sales-based milestones. Evrysdi, marketed by Roche, is an orally administered survival motor neuron-2 (SMN2) splicing modifier for the treatment of spinal muscular atrophy. Evrysdi was approved by the FDA in 2020 and has treated over 21,000 patients worldwide. In 2024, E ...

Core Insights - PTC Therapeutics has experienced a significant increase in investment from Palo Alto Investors, which raised its stake by 456,144 shares, valued at approximately $33.1 million, indicating strong investor confidence in the company's future prospects [1][2][10] Company Overview - PTC Therapeutics specializes in the discovery, development, and commercialization of medicines for rare diseases, supported by a diversified portfolio of marketed products and late-stage pipeline candidates [6] - As of the latest report, PTC's market capitalization stands at $6 billion, with a trailing twelve months (TTM) revenue of $1.8 billion and a net income of $751.7 million, a significant improvement from a net loss of $106.7 million the previous year [4][9] Financial Performance - The company reported $211 million in revenue for the third quarter, alongside a net income of $15.9 million, showcasing a strong turnaround and profitability [7][9] - The stock price of PTC Therapeutics has risen to $74.72, reflecting a 49% increase over the past year, significantly outperforming the S&P 500's 13% gain during the same period [3][4] Investment Insights - Palo Alto Investors now holds 9.8% of its $540.4 million in reportable U.S. equity assets in PTC, making it one of the fund's top holdings, which suggests a belief in the company's sustained momentum despite regulatory uncertainties [3][10] - The launch of Sephience has been identified as a growth catalyst, generating $19.6 million in Q3 sales and showing early traction with 521 start forms in the U.S. and 341 patients on therapy worldwide [9][10] Future Outlook - The key question for long-term investors is whether the late-stage pipeline and Sephience can support sustained revenue growth as legacy products decline, with PTC holding cash reserves of $1.7 billion for flexibility during upcoming regulatory milestones [11]

Financial Data and Key Metrics Changes - The company reported a significant milestone with the successful readout of its phase III trial in October 2024, marking it as the first muscle-targeted treatment for SMA [2][5] - The company has a cash balance of $369 million, which is expected to sustain operations into 2027 [54] Business Line Data and Key Metrics Changes - The primary asset, apitegromab, is positioned to serve patients globally with SMA, with plans for clinical development programs for additional myostatin inhibitors [5][21] - The phase III trial demonstrated a statistically significant improvement in motor function for patients receiving apitegromab alongside SMN-targeted therapies, with a p-value of 0.01 [18] Market Data and Key Metrics Changes - In the U.S., approximately 7,000 patients have received at least one SMN-targeted treatment, with a global total of about 35,000 patients [20] - The combined annual revenue from existing SMN-targeted therapies is approximately $5 billion, indicating a robust market for SMA treatments [21] Company Strategy and Development Direction - The company aims to build a global biotech powerhouse, focusing on rare neuromuscular diseases, starting with SMA and expanding into other indications [3][5] - Plans include a methodical expansion into Europe, Asia-Pacific, and Latin America, targeting a 50-country operating platform [5][32] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the unique approach to myostatin inhibition, which differentiates the company from competitors focusing solely on motor neuron survival [12][24] - The company anticipates a resubmission of its BLA and a potential launch in the U.S. in 2026, following a constructive meeting with the FDA [47][52] Other Important Information - The company is currently addressing manufacturing issues that led to a Complete Response Letter (CRL) from the FDA, with a focus on remediation plans at its drug product manufacturer [41][44] - The company is also advancing its pipeline with SRK-439, a next-generation myostatin inhibitor, expected to enter clinical trials soon [48][50] Q&A Session Summary Question: What is the state of FDA interactions post-CRL? - The company received a CRL due to manufacturing issues at its drug product manufacturer, but management remains optimistic about resolving these issues and expects to resubmit and launch in 2026 [41][47] Question: How does the company plan to execute its commercial strategy? - The company plans to leverage its experience in rare diseases to identify and treat patients already diagnosed with SMA, ensuring a smooth market entry for apitegromab [26][28]

Financial Data and Key Metrics Changes - The company reported a significant milestone with the successful readout of its phase III trial in October 2024, marking it as the first muscle-targeted treatment for SMA [2][5] - The company has a cash balance of $369 million, which is expected to sustain operations into 2027 [54] Business Line Data and Key Metrics Changes - The primary asset, apitegromab, is positioned to serve patients globally with SMA, with plans for clinical development programs for additional myostatin inhibitors [5][21] - The phase III trial demonstrated a statistically significant improvement in motor function, with a p-value of 0.01, indicating the drug's effectiveness when combined with existing SMN-targeted therapies [17][18] Market Data and Key Metrics Changes - In the U.S., approximately 7,000 patients have received at least one SMN-targeted treatment, while globally, the number is around 35,000 [20] - The combined annual revenue from existing SMN-targeted therapies is approximately $5 billion, indicating a robust market for SMA treatments [21] Company Strategy and Development Direction - The company aims to build a global biotech powerhouse, focusing on rare neuromuscular diseases, starting with SMA and expanding into other indications [3][32] - Plans include a methodical expansion into Europe, Asia-Pacific, and Latin America, targeting a multi-billion dollar business model [32][33] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the approval process for apitegromab, viewing the recent CRL as a de-risking event that clarifies the path to approval [52] - The company anticipates launching apitegromab in the U.S. in 2026, with ongoing discussions with the FDA regarding manufacturing compliance [47][45] Other Important Information - The company is also developing SRK-439, a next-generation myostatin inhibitor, which is expected to enter clinical trials soon [48][49] - The management team has been strengthened with experienced leaders from previous successful biotech ventures, enhancing the company's operational capabilities [4][6] Q&A Session Summary Question: What is the state of FDA interactions post-CRL? - The company received a CRL due to manufacturing issues at the drug product facility, but they are working closely with Novo Nordisk to address these concerns and expect to resubmit in 2026 [41][45] Question: How does the company plan to execute its commercial strategy? - The company plans to leverage existing patient networks and funding mechanisms for rare disease therapies to facilitate the launch of apitegromab [26][28]

Financial Data and Key Metrics Changes - The company reported a significant transformation over the past 12 to 14 months, particularly following the positive readout of its phase III trial in October 2024, marking it as the first muscle-targeted treatment for SMA [2][5] - The company has a cash balance of $369 million, which is expected to sustain operations into 2027, with plans to expand its loan facility to support the upcoming launch [50] Business Line Data and Key Metrics Changes - The primary asset, apitegromab, is positioned as a muscle-targeted treatment for SMA, with clinical trials demonstrating statistically significant improvements in motor function when used alongside existing SMN-targeted therapies [16][20] - The company anticipates a multi-billion dollar opportunity for apitegromab, estimating over $2 billion in annual revenue from SMA alone [21] Market Data and Key Metrics Changes - In the U.S., approximately 7,000 patients have received at least one SMN-targeted treatment, with a global total of about 35,000 patients [19] - The existing SMN-targeted therapies generate approximately $5 billion in annual revenues, indicating a robust market for SMA treatments [20] Company Strategy and Development Direction - The company aims to build a global operating platform across 50 countries, focusing on expanding its reach in Europe, Asia-Pacific, and Latin America [5] - Future plans include entering clinical trials for other rare neuromuscular disorders, with indications such as FSHD and DMD being assessed [30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in overcoming the recent CRL issued by the FDA, emphasizing that the remaining issue is solvable and that approval is a matter of when, not if [48][49] - The company is committed to a collaborative approach with the FDA and its manufacturing partners to expedite the approval process for apitegromab [42] Other Important Information - The company has established a world-class executive team to drive its strategic vision, with leadership experienced in building successful biotech companies [3][4] - The company is also developing SRK-439, a next-generation myostatin inhibitor, which is expected to enhance its rare neuromuscular franchise [45][46] Q&A Session Summary Question: What is the state of FDA interactions post-CRL? - The company received a CRL due to manufacturing issues at its drug product manufacturer, but management remains optimistic about resolving these issues and expects to resubmit and launch in 2026 [32][44] Question: How does the company plan to execute its commercial strategy? - The company plans to leverage its existing knowledge of the rare disease market, focusing on patients already diagnosed and receiving treatment, to effectively launch apitegromab [26][27]

Core Insights - Tang Capital Management increased its stake in PTC Therapeutics by 400,000 shares, raising its total holdings to 1.5 million shares valued at $92.1 million as of September 30, reflecting a $38.3 million increase in net position [2][11] - PTC Therapeutics has shown strong commercial momentum with the successful launch of Sephience, contributing $19.6 million in revenue during the third quarter, and the company reported a quarterly net profit of $15.9 million, a significant turnaround from a $106.7 million loss a year earlier [10][11] - The stock price of PTC Therapeutics reached $78.50, marking a 51% increase over the past year, significantly outperforming the S&P 500, which rose by 13% in the same period [3][4] Company Overview - PTC Therapeutics is a mid-cap biopharmaceutical company focused on developing therapies for rare genetic disorders, with a market capitalization of $6.3 billion and a revenue of $1.8 billion over the trailing twelve months [4][6] - The company has a diversified portfolio of approved products and a robust pipeline addressing unmet medical needs in the rare disease sector, including therapies like Translarna, Emflaza, Tegsedi, Waylivra, and Evrysdi [9][6] - PTC Therapeutics generates revenue primarily through the sale and licensing of its pharmaceutical products and strategic collaborations with industry partners, targeting healthcare providers and specialty pharmacies across various regions [9][6] Financial Performance - In the third quarter, PTC Therapeutics reported revenue of $211 million, driven by the Sephience launch and increased royalty income from Evrysdi [11] - The company's net income for the trailing twelve months stands at $751.7 million, indicating a strong financial position [4]

Oral Small Molecule Splicing Platform - PTC pioneered small molecule splicing therapies, with Evrysdi approved in over 100 countries and votoplam in development for Huntington's disease[21] - Small molecules enhance U1 interaction, increasing exon inclusion to generate distinct mRNA transcripts and modulate protein expression, exemplified by votoplam (PTC518)[22, 25, 29, 30] - PTC's PTSeek screening technology, leveraging learnings from SMA and HD programs, enables the discovery of novel sequence-selective 5' splice site enhancers for iExons[48, 96, 98] - PTC has discovered iExons throughout the entire transcriptome, covering a majority of genes, allowing for the targeting of splicing for novel small molecule enhancers[89, 90, 91] Splicing Platform Programs - MSH3 program targets nucleotide repeat disorders like Huntington's disease, with 30-50% MSH3 lowering stalling expansion in a human somatic expansion model[111, 129] - Spinocerebellar Ataxia 3 (SCA3) program aims to lower mutant ATXN3 protein production by inducing exon skipping[132, 141, 142] - Oncology program targets a key protein in cancer biology, with novel splicing modulators selectively inducing mRNA decay[147, 152] - Hemoglobinopathies program targets regulators of fetal hemoglobin (HbF) levels, with novel splicing modulators lowering a validated target of HbF induction[158, 163, 165] Inflammation & Ferroptosis Platform - PTC's inflammation and ferroptosis platform targets specialized enzyme hubs to regulate inflammation, energy production, and oxidative stress[179] - Parkinson's Disease program targets 15-LO to prevent ferroptosis-mediated cell death and alpha-synuclein aggregation[181, 183, 188] - PTC612, a highly selective NLRP3 inhibitor, demonstrates significant reduction in inflammatory cytokines and pathology across multiple preclinical models, with an IC50 of 099 nM in THP-1 cells[225, 227, 234]

Core Insights - PTC Therapeutics, focused on rare disease therapies, experienced a significant insider sale by Director Emma Reeve, who sold 10,000 shares for approximately $795,000, reducing her holdings to 6,666 shares [1][2][6] Company Overview - PTC Therapeutics reported a total revenue of $1.78 billion and a net income of $751.72 million for the trailing twelve months (TTM) [4] - The company's stock price has increased by 92.91% over the past year, indicating strong market performance [4] Transaction Details - The transaction involved the exercise of stock options and immediate sale of shares, with the sale representing 60% of Reeve's direct holdings prior to the transaction [6] - Post-transaction, Reeve's direct ownership decreased to approximately 0.0083% [6] - The shares were sold at a price of $79.50, while the stock was priced at $82.93 shortly after the transaction [6] Market Environment - The one-year total return for PTC Therapeutics was 83% as of the transaction date, reflecting strong share price appreciation leading up to the sale [6] - The company reported a 7% year-over-year revenue growth in the third quarter, reaching $211 million, with royalty revenue from Roche increasing to $70.8 million [10] Product Portfolio - PTC Therapeutics specializes in treatments for rare genetic disorders, including products like Translarna and Emflaza for Duchenne muscular dystrophy, and Tegsedi, Waylivra, and Evrysdi for other rare diseases [7][8] - The company operates a biopharmaceutical business model focused on drug discovery, clinical development, and global commercialization [7][8] Customer Base - Primary customers include healthcare providers, hospitals, and government agencies treating patients with rare diseases, particularly in North America, Europe, and Latin America [8]

Summary of PTC Therapeutics Conference Call Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on rare disease therapies with a robust commercial portfolio of six marketed products [3][4] - The company has launched Safiance, an oral therapy for Phenylketonuria (PKU), which is expected to be a multi-billion dollar opportunity due to significant unmet needs in the PKU patient population [3][4][5] Core Points and Arguments Unmet Need in PKU - Approximately 17,000 individuals in the U.S. and 58,000 worldwide suffer from PKU, with a significant unmet need despite two existing therapies [4][5] - Current therapies do not sufficiently lower phenylalanine levels or allow dietary liberalization, leading to a burdensome diet for patients [5][6] - Safiance offers a differentiated solution with significant efficacy, allowing 97% of patients to liberalize their diet [7][8] Launch and Market Reception - Safiance was approved in July 2025, generating $19.6 million in revenue within the first six weeks of launch, with broad penetration among prescribers [10][27] - Physicians express strong interest in prescribing Safiance to all PKU patients, regardless of their current treatment status [11][18] - The launch strategy involved building relationships with Centers of Excellence and integrating with patient communities [10][12] Market Access and Payer Dynamics - Positive feedback from payers with minimal restrictions on Safiance prescriptions, primarily requiring prior authorization [19][22] - The company has engaged with payers to demonstrate the value of Safiance through clinical data, which has helped avoid step edits in access [21][22] International Expansion - The European launch began in Germany, maintaining a pricing corridor consistent with the U.S. [24][25] - Plans for launches in Japan and Brazil are underway, with expectations of favorable pricing dynamics [25][26] Financial Outlook - Revenue guidance for the year is set at $750 million to $800 million, with expectations that Safiance will drive the company towards profitability [29] - The company anticipates a decrease in operating expenses as expensive trials conclude [29] Additional Important Points Pipeline and Future Developments - PTC is focused on maintaining revenue from its Duchenne Muscular Dystrophy (DMD) franchise despite generic competition, achieving over 70% of peak revenue post-exclusivity [30][31] - Upcoming FDA meetings for Vatiquinone (Friedreich's ataxia) and PTC518 (Huntington's disease) are aimed at discussing potential paths for resubmission and trial designs [34][36] R&D Focus - The company plans to showcase progress on its scientific platforms at an upcoming R&D day, highlighting advancements in its splicing platform and other therapeutic areas [37] This summary encapsulates the key insights from the conference call, focusing on PTC Therapeutics' strategic initiatives, market dynamics, and future outlook.