Ionis expands partnership with Sobi to include olezarsen commercialization outside the U.S.
Ionis Pharmaceuticals (NASDAQ: IONS) has expanded its partnership with Sobi, granting exclusive rights to commercialize olezarsen outside the U.S., Canada, and China for treating familial chylomicronemia syndrome (FCS) and severely elevated triglycerides.
Key highlights:
- TRYNGOLZA™ (olezarsen) received FDA approval in December 2024 as the first treatment for FCS in the U.S.
- European Medicines Agency (EMA) review is ongoing with potential approval expected this year
- Ionis will receive an upfront payment, milestone-based payments, and tiered royalties up to mid-20% on annual net sales
- Sobi will handle regulatory submissions and commercialization in their designated territories
Three Phase 3 clinical trials (CORE, CORE2, and ESSENCE) are evaluating olezarsen for severe hypertriglyceridemia (sHTG), with data expected in mid-2025 and second half of 2025.
Ionis Pharmaceuticals (NASDAQ: IONS) ha ampliato la sua partnership con Sobi, concedendo diritti esclusivi per commercializzare olezarsen al di fuori degli Stati Uniti, del Canada e della Cina per il trattamento della sindrome da chilomicronemia familiare (FCS) e dei livelli di trigliceridi severamente elevati.
Principali punti salienti:
- TRYNGOLZA™ (olezarsen) ha ricevuto l'approvazione della FDA nel dicembre 2024 come primo trattamento per la FCS negli Stati Uniti.
- La revisione da parte dell'Agenzia Europea dei Medicinali (EMA) è in corso, con un'approvazione potenziale attesa per quest'anno.
- Ionis riceverà un pagamento iniziale, pagamenti legati a traguardi e royalty su base scalare fino al 20% sulle vendite nette annuali.
- Sobi si occuperà delle presentazioni regolatorie e della commercializzazione nei territori designati.
Tre studi clinici di Fase 3 (CORE, CORE2 ed ESSENCE) stanno valutando olezarsen per l'ipertrigliceridemia severa (sHTG), con dati attesi a metà 2025 e nella seconda metà del 2025.
Ionis Pharmaceuticals (NASDAQ: IONS) ha ampliado su asociación con Sobi, otorgando derechos exclusivos para comercializar olezarsen fuera de EE. UU., Canadá y China para el tratamiento del síndrome de quilomicronemia familiar (FCS) y niveles de triglicéridos severamente elevados.
Puntos clave:
- TRYNGOLZA™ (olezarsen) recibió la aprobación de la FDA en diciembre de 2024 como el primer tratamiento para la FCS en EE. UU.
- La revisión por parte de la Agencia Europea de Medicamentos (EMA) está en curso, con una aprobación potencial esperada para este año.
- Ionis recibirá un pago inicial, pagos basados en hitos y regalías escalonadas de hasta el 20% sobre las ventas netas anuales.
- Sobi se encargará de las presentaciones regulatorias y la comercialización en sus territorios designados.
Tres ensayos clínicos de Fase 3 (CORE, CORE2 y ESSENCE) están evaluando olezarsen para la hipertrigliceridemia severa (sHTG), con datos esperados a mediados de 2025 y en la segunda mitad de 2025.
아이오니스 제약 (NASDAQ: IONS)는 소비(Sobi)와의 파트너십을 확대하여 미국, 캐나다 및 중국 외부에서 가족성 킬로미크론혈증 증후군(FCS) 및 심각하게 높은 트리글리세리드 치료를 위한 올레자르센(olezarsen)의 상업화 독점 권한을 부여했습니다.
주요 하이라이트:
- TRYNGOLZA™ (olezarsen)는 2024년 12월 미국에서 FCS에 대한 첫 번째 치료제로 FDA 승인을 받았습니다.
- 유럽 의약청(EMA)의 검토가 진행 중이며, 올해 승인될 가능성이 있습니다.
- 아이오니스는 선불금, 이정표 기반 지급금 및 연간 순매출의 최대 20%에 해당하는 단계적 로열티를 받을 것입니다.
- 소비는 지정된 지역에서 규제 제출 및 상업화를 담당할 것입니다.
세 가지 3상 임상 시험(CORE, CORE2 및 ESSENCE)이 심각한 고트리글리세리드혈증(sHTG)에 대한 올레자르센을 평가하고 있으며, 2025년 중반과 하반기에 데이터가 예상됩니다.
Ionis Pharmaceuticals (NASDAQ: IONS) a élargi son partenariat avec Sobi, accordant des droits exclusifs pour commercialiser olezarsen en dehors des États-Unis, du Canada et de la Chine pour traiter le syndrome de chylomicronémie familiale (FCS) et des triglycérides sévèrement élevés.
Points clés :
- TRYNGOLZA™ (olezarsen) a reçu l'approbation de la FDA en décembre 2024 en tant que premier traitement pour la FCS aux États-Unis.
- La révision par l'Agence européenne des médicaments (EMA) est en cours, avec une approbation potentielle attendue cette année.
- Ionis recevra un paiement initial, des paiements basés sur des jalons et des redevances échelonnées allant jusqu'à 20 % sur les ventes nettes annuelles.
- Sobi s'occupera des soumissions réglementaires et de la commercialisation dans ses territoires désignés.
Trois essais cliniques de phase 3 (CORE, CORE2 et ESSENCE) évaluent olezarsen pour l'hypertriglycéridémie sévère (sHTG), avec des données attendues à la mi-2025 et dans la seconde moitié de 2025.
Ionis Pharmaceuticals (NASDAQ: IONS) hat seine Partnerschaft mit Sobi erweitert und exklusive Rechte zur Vermarktung von olezarsen außerhalb der USA, Kanada und China zur Behandlung des familiären Chylomikronämie-Syndroms (FCS) und stark erhöhten Triglyceridwerten eingeräumt.
Wichtige Highlights:
- TRYNGOLZA™ (olezarsen) erhielt im Dezember 2024 die FDA-Zulassung als erste Behandlung für FCS in den USA.
- Die Überprüfung durch die Europäische Arzneimittel-Agentur (EMA) ist im Gange, eine mögliche Genehmigung wird in diesem Jahr erwartet.
- Ionis wird eine Vorauszahlung, milestone-basierte Zahlungen und gestaffelte Lizenzgebühren von bis zu 20 % auf den jährlichen Nettoumsatz erhalten.
- Sobi wird die regulatorischen Einreichungen und die Vermarktung in den zugewiesenen Gebieten übernehmen.
Drei klinische Phase-3-Studien (CORE, CORE2 und ESSENCE) evaluieren olezarsen bei schwerer Hypertriglyceridämie (sHTG), wobei Daten für Mitte 2025 und die zweite Hälfte von 2025 erwartet werden.
- First-mover advantage with FDA approval for TRYNGOLZA as the only FCS treatment in U.S.
- Expanded market reach through Sobi's presence in 30+ countries
- Revenue potential through upfront payment, milestones, and up to mid-20% royalties
- Leverages Sobi's existing European FCS market expertise and distribution channels
- Giving up commercialization rights in multiple markets could limit long-term revenue potential
- Success depends on pending EMA approval and Phase 3 trial results
Insights
This licensing agreement between Ionis and Sobi represents a strategically sound commercialization approach for olezarsen. By partnering with Sobi for ex-U.S. markets, Ionis gains several advantages without sacrificing significant economics.
The deal structure includes an upfront payment, milestone payments, and notably, tiered royalties reaching the mid-20% range - relatively high for pharmaceutical licensing agreements, indicating Ionis maintained favorable economics while offloading international commercial infrastructure costs. This arrangement allows Ionis to focus its commercial resources on the U.S. market where it retains full rights to TRYNGOLZA.
Sobi brings established infrastructure in over 30 countries and existing relationships with the FCS treatment community through their commercialization of Waylivra in Europe. This foundation should enable efficient market entry for olezarsen following anticipated EMA approval later this year.
The timing of this agreement is particularly strategic with three Phase 3 trials (CORE, CORE2, and ESSENCE) for severe hypertriglyceridemia reading out in 2025. If successful, these trials would expand olezarsen's addressable market dramatically beyond the rare FCS population. Securing global commercial channels ahead of these potential label expansions demonstrates forward-thinking planning by Ionis management.
This partnership effectively balances Ionis's resource allocation while maintaining strong economic participation in global markets, creating a favorable outlook for the global commercialization strategy of olezarsen.
This licensing deal significantly enhances olezarsen's global accessibility for patients with conditions characterized by elevated triglycerides. The agreement creates a particularly compelling solution for FCS patients, who suffer from a rare genetic disorder with severely treatment options.
Familial chylomicronemia syndrome affects approximately 1-2 people per million worldwide and leads to extremely high triglyceride levels, potentially life-threatening pancreatitis, and chronic symptoms that dramatically impact quality of life. TRYNGOLZA (olezarsen) received FDA approval in December 2024 as the first and only treatment for FCS in the U.S.
By leveraging Sobi's established rare disease commercialization infrastructure, particularly their experience marketing Waylivra for FCS in Europe, this partnership ensures rapid accessibility following anticipated EMA approval this year. For rare disease treatments, specialized infrastructure and patient identification capabilities are critical success factors - Sobi brings these existing capabilities to the partnership.
The ongoing Phase 3 program for severe hypertriglyceridemia (sHTG) represents an even more substantial market opportunity. While FCS is ultra-rare, sHTG affects a considerably larger population that could significantly benefit from triglyceride reduction therapy.
This partnership effectively bridges the gap between therapeutic innovation and patient access in underserved markets, potentially transforming care for patients with these challenging metabolic conditions across multiple geographies.
– Agreement enables olezarsen to reach people living with conditions associated with elevated triglycerides across the world –
– Ionis will continue to independently commercialize TRYNGOLZA™ (olezarsen) in the
Olezarsen is currently under review by the European Medicines Agency (EMA) with a potential approval anticipated for the treatment of FCS this year. Sobi will be responsible for future regulatory submissions and commercialization in ex-
“We look forward to deepening our long-standing partnership with Sobi and our shared commitment to improving the lives of people living with conditions marked by severely elevated triglycerides,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “This agreement enables us to advance our goal of making olezarsen available to patients globally, while focusing our independent commercialization efforts in the
Sobi® (STO: SOBI) brings deep commercialization expertise for both rare and more prevalent conditions, with operations in more than 30 countries, and is committed to bringing olezarsen to as many global patients as possible. As Ionis’ current European commercialization partner for Waylivra® (volanesorsen), which is the only medicine approved for FCS in
"We are thrilled to continue our collaboration with Ionis and bring this potential new medicine to patients if approved," said Guido Oelkers, chief executive officer, Sobi. "This will enable us to further leverage our combined strengths and to continue to deliver our commitment to provide innovative medicines that transform the lives of people with rare and debilitating diseases."
In addition to FCS, Ionis is evaluating olezarsen for the treatment of severe hypertriglyceridemia (sHTG) in three Phase 3 clinical trials – CORE, CORE2 and ESSENCE. Data from ESSENCE are expected mid-2025 and data from CORE and CORE2 are expected in the second half of 2025.
About Familial Chylomicronemia Syndrome (FCS)
FCS is a rare, genetic disease characterized by extremely elevated triglyceride levels. It is caused by impaired function of the enzyme lipoprotein lipase (LPL). Because of limited LPL production or function, people with FCS cannot effectively break down chylomicrons, lipoprotein particles that are
About Severe Hypertriglyceridemia (sHTG)
sHTG is a disease categorized by triglyceride levels of 500 mg/dL and above. It develops due to primary (genetic) and secondary causes including diet and lifestyle, other medical conditions and certain medications. More than three million people are currently estimated to live with sHTG in the
Olezarsen has not been reviewed or approved for the treatment of severe hypertriglyceridemia (sHTG) by any regulatory authority.
About TRYNGOLZA™ (olezarsen)
TRYNGOLZA™ (olezarsen) was approved by the
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred.
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur.
ADVERSE REACTIONS
The most common adverse reactions (incidence >
Please see full Prescribing Information for TRYNGOLZA.
About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology, and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram.
Ionis Forward-Looking Statements
This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of TRYNGOLZA™ (olezarsen), Ionis' technologies and other products in development. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended Dec. 31, 2024, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at www.Ionis.com.
Ionis Pharmaceuticals® and TRYNGOLZA™ are trademarks of Ionis Pharmaceuticals, Inc.
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Source: Ionis Pharmaceuticals, Inc.