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 ...

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 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 Viewpoint - The approval of Itvisma by Novartis marks a significant advancement in gene therapy for spinal muscular atrophy (SMA), with a wholesale price of $2.59 million, highlighting the growing market for high-cost life-saving drugs and the potential for gene therapy to become a mainstream treatment option [4][5][10]. Gene Therapy Market Dynamics - The global gene therapy market is at a pivotal point, expected to grow at an annual rate of over 20% in the next 5-10 years, driven by technological advancements, clearer regulatory pathways, and innovative payment models [5][12]. - Itvisma is the first gene therapy approved for a broader patient demographic with SMA, emphasizing its rarity and the high barriers to entry in the market due to substantial R&D investments [5][8]. Pricing Strategy and Market Comparison - Novartis' pricing strategy for Itvisma is informed by the $9.4 billion total R&D investment for Zolgensma, which was previously priced at $2.1 million [7][8]. - The pricing of Itvisma and Zolgensma significantly exceeds estimates from industry observers, indicating a disconnect between market expectations and pharmaceutical pricing strategies [7][8]. Clinical Efficacy and Safety - Itvisma's approval is based on robust clinical trial data demonstrating significant improvements in motor function for SMA patients, with a favorable safety profile [9][10]. - The treatment offers a one-time administration approach, reducing the burden of ongoing therapies that are common with existing SMA treatments [9][10]. Competitive Landscape - Itvisma competes directly with Spinraza, the first approved SMA treatment, which has undergone significant price reductions in markets like China after being included in insurance coverage [10][11]. - The gene therapy market is witnessing increased competition as more companies invest in gene editing and delivery systems, necessitating continuous innovation and differentiation [5][12]. Future Growth and Investment Trends - The gene therapy market is projected to reach $64.64 billion by 2033, with a compound annual growth rate of 27.6% from 2024 to 2033, driven by technological breakthroughs and accelerated regulatory approvals [12][13]. - Major pharmaceutical companies are actively acquiring biotech firms to enhance their gene therapy portfolios, indicating strong investor interest and market potential [13][14]. Challenges Ahead - Despite the promising outlook, gene therapy faces challenges such as high production costs, short shelf life of viral vectors, and the need for a balance between innovation incentives and drug accessibility [16][17]. - The industry is also grappling with the complexities of developing therapies for common diseases, where competition is fierce and clinical efficacy must be convincingly demonstrated [14][16].

Core Insights - The approval of Itvisma by the FDA marks a significant advancement in gene therapy for spinal muscular atrophy (SMA), targeting patients aged two and older with specific genetic mutations [1][2] - Itvisma's wholesale price is set at $2.59 million, making it one of the most expensive drugs on the market, and it is the first gene therapy approved for a broader patient demographic [1][3] - The global gene therapy market is expected to grow at an annual rate of over 20% in the next 5-10 years, driven by technological advancements and regulatory clarity [2][8] Drug Pricing and Market Strategy - Novartis has justified the high pricing of Zolgensma, its previous gene therapy, citing a total R&D investment of $9.4 billion, including an $8.7 billion acquisition of AveXis [3][4] - The pricing strategy for Zolgensma has been significantly higher than industry estimates, indicating a potential trend for future gene therapies [3][4] - Itvisma's pricing reflects a calculated commercial strategy, aiming to establish a precedent for high-cost life-saving treatments [3][4] Competitive Landscape - Itvisma competes directly with Spinraza, the first targeted SMA treatment, which has undergone significant price reductions in China after being included in the national insurance directory [6][7] - The gene therapy market is characterized by high R&D costs and significant barriers to entry, allowing early entrants to maintain pricing power and market exclusivity [2][7] - The market is expected to evolve towards platform-based and diversified pipelines as more companies enter the gene editing and delivery systems space [2][8] Market Growth and Investment - The global gene therapy market is projected to reach $9.03 billion in 2024 and grow to $64.64 billion by 2033, with a compound annual growth rate of 27.6% [8] - Major pharmaceutical companies are actively investing in gene therapy, with significant acquisitions and partnerships to enhance their portfolios [9][10] - The increasing number of approved gene therapy products and the rapid pace of regulatory approvals are expected to drive market expansion [8][10] Challenges and Future Directions - The development of gene therapies faces challenges such as high costs, short shelf life, and production capacity limitations [11][12] - The industry is exploring second and third-generation gene therapies to overcome the limitations of current technologies, such as AAV delivery systems [11][12] - Balancing innovation incentives with drug accessibility remains a long-term issue for the industry [11][12]

Core Insights - The approval of Itvisma by the FDA marks a significant advancement in gene therapy for spinal muscular atrophy (SMA), targeting patients aged two and older with specific genetic mutations [1][2] - Itvisma's wholesale price is set at $2.59 million, making it one of the most expensive drugs on the market, and it is positioned as a test for the market's tolerance for high-priced life-saving medications [1][3] - The global gene therapy market is expected to grow at an annual rate of over 20% in the next 5-10 years, driven by technological advancements and regulatory clarity [2][8] Company Insights - Novartis has invested heavily in gene therapy, with Zolgensma's development costs reaching $9.4 billion, including the acquisition of AveXis for $8.7 billion [3] - Zolgensma's pricing strategy has set a precedent in the industry, with its initial sales reaching $160 million in the first quarter post-launch and $1.37 billion in 2022 [6] - Itvisma is designed for a one-time administration, providing a functional human SMN1 gene to improve motor function, which could reduce the treatment burden compared to existing therapies [5][6] Industry Insights - The gene therapy market is projected to reach $9.03 billion in 2024 and grow to $64.64 billion by 2033, with a compound annual growth rate of 27.6% [8] - Major pharmaceutical companies are actively investing in gene therapy, with significant acquisitions such as Sanofi's $11.6 billion purchase of Bioverativ and Roche's $4.3 billion acquisition of Spark Therapeutics [9] - The industry faces challenges including high production costs, short shelf life of viral vectors, and the need for a balance between innovation incentives and drug accessibility [10][11]

Core Viewpoint - The approval of Itvisma by Novartis marks a significant advancement in gene therapy for spinal muscular atrophy (SMA), with a price tag of $2.59 million, indicating the market's tolerance for high-cost life-saving drugs [1][2]. Group 1: Gene Therapy Market Dynamics - The global gene therapy market is at a turning point, expected to grow at an annual rate of over 20% in the next 5-10 years, driven by technological advancements, clearer regulatory pathways, and innovative payment models [2]. - The success of Zolgensma has validated the commercial viability of one-time curative treatments for single-gene hereditary diseases, setting a benchmark for future gene therapies in various fields [2]. - Currently, only three SMA drugs have been approved globally, with Itvisma being the only gene therapy product, highlighting its rarity and the high barriers to entry in the market [2]. Group 2: Pricing and Cost Justification - Novartis disclosed that the total R&D investment for Zolgensma reached $9.4 billion, including an $8.7 billion acquisition of AveXis [4]. - The pricing strategy for Zolgensma was significantly higher than industry estimates, with a suggested price range of $1.5 million to $5 million per patient, compared to UBS's estimate of $2 million and ICER's range of $310,000 to $1.5 million [4]. - The high price of Itvisma reflects a calculated commercial strategy, as the company aims to establish a strong market presence despite the high upfront costs associated with gene therapy development [2][4]. Group 3: Clinical Efficacy and Safety - Itvisma's approval was based on the results of the pivotal Phase 3 study STEER, which showed statistically significant improvements in motor function among treated patients, with effects sustained over a 52-week follow-up [5][6]. - The most common adverse events reported in the studies included upper respiratory infections and fever, indicating a consistent safety profile [5][6]. Group 4: Competitive Landscape - Itvisma competes directly with Spinraza, the first targeted SMA treatment, which has undergone significant price reductions in markets like China after being included in insurance coverage [7]. - The gene therapy market is expected to see explosive growth, with projections indicating a market size of $9.03 billion in 2024, increasing to $64.64 billion by 2033, driven by innovations in gene editing technologies and accelerated regulatory approvals [9]. Group 5: Industry Challenges and Future Outlook - The gene therapy sector faces challenges such as high production costs, short shelf life of viral vectors, and the need for stringent quality control, which can limit supply and increase prices [13]. - Balancing innovation incentives with drug accessibility remains a long-term issue for the industry, as the future of gene therapy will depend on its ability to reach a broader patient population beyond clinical settings [13].

Core Insights - The approval of Itvisma by the FDA marks a significant advancement in gene therapy for spinal muscular atrophy (SMA), targeting patients aged two and older with specific genetic mutations [1][2] - Itvisma's wholesale price is set at $2.59 million, making it one of the most expensive drugs on the market, and it is the first gene therapy approved for a broader patient demographic [1][3] - The global gene therapy market is expected to grow at an annual rate of over 20% in the next 5-10 years, driven by technological advancements and regulatory clarity [2][8] Pricing and Market Dynamics - The pricing strategy for Itvisma is influenced by the high R&D costs associated with gene therapies, with Zolgensma's development costing $9.4 billion [3][4] - The market for SMA treatments is currently limited, with only three approved drugs, highlighting Itvisma's unique position as a gene therapy product [2][4] - The competitive landscape is evolving, with companies focusing on gene editing and delivery systems, which may lead to increased competition and innovation [2][10] Clinical Efficacy and Safety - Itvisma's approval is based on significant improvements in motor function observed in clinical trials, with consistent safety profiles reported [4][5] - The treatment aims to provide a one-time solution by delivering a functional SMN1 gene, potentially reducing the need for ongoing therapies [5][6] Market Potential and Competition - The gene therapy market is projected to reach $90.3 billion by 2024, with a compound annual growth rate of 27.6% expected through 2033 [8] - Major pharmaceutical companies are actively investing in gene therapy, as evidenced by significant acquisitions and partnerships in the sector [9][10] - Itvisma will compete directly with Spinraza, the first SMA treatment, which has undergone significant price adjustments to improve accessibility [6][7] Future Outlook - The success of Itvisma could pave the way for further advancements in gene therapy, particularly for rare diseases, while also raising questions about drug pricing and accessibility [12][13] - Ongoing challenges include high production costs and the need for innovative delivery systems to enhance the efficacy and safety of gene therapies [11][12]

Core Viewpoint - The approval of Itvisma, a gene therapy priced at approximately $2.59 million, marks a significant milestone in the gene therapy market, raising questions about the sustainability of high-priced treatments and their accessibility for patients [1][12]. Gene Therapy Market Overview - The global gene therapy market is at a turning point, expected to grow at an annual rate of over 20% in the next 5-10 years, driven by technological advancements, clearer regulatory pathways, and innovative payment models [2][11]. - The market size is projected to reach $90.3 billion by 2024, increasing to $115.2 billion by 2025, and potentially reaching $646.4 billion by 2033, with a compound annual growth rate of 27.6% [7]. Itvisma and Competitive Landscape - Itvisma is the first gene therapy approved for a broader patient population with spinal muscular atrophy (SMA), highlighting its rarity and the high barriers to entry in the market [2][4]. - Itvisma's approval is based on significant clinical trial results showing improvements in motor function, with a consistent safety profile [4][5]. - The competitive landscape includes Spinraza, another SMA treatment, which has seen significant price reductions in markets like China, indicating the potential for price adjustments in response to market dynamics [6][12]. Pricing and Economic Considerations - The pricing strategy for Itvisma reflects the high R&D costs associated with gene therapies, with Zolgensma's development costing $9.4 billion [3][4]. - The debate over the "reasonable" price range for gene therapies continues, with various estimates significantly lower than the actual market prices [3][4]. Accessibility and Payment Models - The high cost of gene therapies raises concerns about patient accessibility, prompting discussions on innovative payment models, such as installment payments linked to treatment outcomes [12][13]. - The need for regulatory frameworks to balance innovation incentives with drug accessibility remains a critical issue for the industry [11][12]. Future Outlook - The gene therapy sector is expected to expand beyond rare diseases into more common conditions, driven by advancements in gene editing technologies and increasing regulatory approvals [11][12]. - Companies are focusing on production scalability and cost control to make therapies more affordable, which is essential for broader market penetration [12][13].

Summary of Ionis Pharmaceuticals Conference Call Company Overview - **Company**: Ionis Pharmaceuticals - **Industry**: Biotechnology, specifically focusing on genetic medicines targeting RNA for therapeutics - **Recent Achievements**: Launched two FDA-approved medicines independently in the U.S. in 2025: Tryngolza for Familial Chylomicronemia Syndrome (FCS) and Donidalorsen for hereditary angioedema [2][3] Core Points and Arguments - **Successful Product Launches**: - Tryngolza, the first FDA-approved medicine for FCS, launched successfully in 2025 [2] - Donidalorsen launched as a prophylactic treatment for hereditary angioedema [2] - **Pipeline Progress**: - Announced positive phase three data for severe hypertriglyceridemia and a neurology drug for Alexander disease [3][4] - Anticipating five phase three readouts and two to three FDA approvals in the upcoming year [4] - **Market Opportunity**: - Severe hypertriglyceridemia affects millions in the U.S., with a focus on patients at high risk for acute pancreatitis [7][9] - Plans to target both severely at-risk patients and those with mildly elevated triglycerides [10][11] - **Combination Therapy Potential**: - Exploring combination therapies for managing triglycerides and other cardiovascular risk factors [12][13] - Collaboration with AstraZeneca to combine treatments for TTR cardiomyopathy [14][17] Additional Important Insights - **Self-Administration Preference**: - Patients prefer self-administered treatments, which could enhance market penetration, especially in community settings [20][21] - **Regulatory and Market Dynamics**: - Anticipated inclusion in treatment guidelines for severe hypertriglyceridemia, with a significant patient population of over 3 million in the U.S. [40] - **Spinraza Update**: - Ionis is developing a next-generation Spinraza with once-per-year dosing, expected to re-emerge as a leading treatment for spinal muscular atrophy (SMA) [32][33] - Economic terms for the next-gen product are more favorable, with royalties in the mid-20% range compared to mid-teens for the current compound [34] Conclusion - Ionis Pharmaceuticals is positioned for significant growth with its innovative therapies targeting severe hypertriglyceridemia and SMA. The company is leveraging its unique RNA-targeting platform to address unmet medical needs and expand its market presence through strategic partnerships and a focus on patient convenience.