Otsuka Submits New Drug Application in Japan for Bempedoic Acid in the Treatment of Hypercholesterolemia
Esperion (NASDAQ: ESPR) announced that Otsuka Pharmaceutical has submitted a New Drug Application in Japan for bempedoic acid to treat hypercholesterolemia. The Japanese Phase 3 trial, involving 96 patients, demonstrated significant efficacy with a -25.25% change in LDL-C levels compared to -3.46% in the placebo group after 12 weeks. The drug showed consistent safety profile with previous trials and no serious adverse events. This treatment targets patients who have insufficient response to statins or cannot tolerate them. Bempedoic acid works by inhibiting ATP citrate lyase in the liver's cholesterol synthesis pathway and is already marketed in the US and Europe.
Esperion (NASDAQ: ESPR) ha annunciato che Otsuka Pharmaceutical ha presentato una Richiesta di Nuovo Farmaco in Giappone per bempedoico per trattare l'ipercolesterolemia. Lo studio clinico di Fase 3 in Giappone, che ha coinvolto 96 pazienti, ha dimostrato un'efficacia significativa con una variazione del -25,25% nei livelli di LDL-C rispetto al -3,46% nel gruppo placebo dopo 12 settimane. Il farmaco ha mostrato un profilo di sicurezza coerente con studi precedenti e senza eventi avversi gravi. Questo trattamento è rivolto a pazienti che non rispondono adeguatamente alle statine o che non possono tollerarle. Il bempedoico agisce inibendo la ATP citrato liasi nella via di sintesi del colesterolo nel fegato ed è già commercializzato negli Stati Uniti e in Europa.
Esperion (NASDAQ: ESPR) anunció que Otsuka Pharmaceutical ha presentado una Solicitud de Nuevo Medicamento en Japón para ácido bempedoico para tratar la hipercolesterolemia. El ensayo clínico de Fase 3 en Japón, que involucró a 96 pacientes, demostró una eficacia significativa con un cambio de -25,25% en los niveles de LDL-C en comparación con -3,46% en el grupo de placebo después de 12 semanas. El medicamento mostró un perfil de seguridad coherente con ensayos anteriores y sin eventos adversos graves. Este tratamiento está dirigido a pacientes que tienen una respuesta insuficiente a las estatinas o que no pueden tolerarlas. El ácido bempedoico actúa inhibiendo la ATP citrato liasa en la vía de síntesis de colesterol en el hígado y ya se comercializa en los EE. UU. y Europa.
Esperion (NASDAQ: ESPR)은 오츠카 제약이 일본에서 벡페도산에 대한 신약 신청서를 제출했다고 발표했습니다. 96명의 환자가 참여한 일본의 3상 시험은 12주 후 위약군에 비해 LDL-C 수치가 -25.25% 변화하는 유의한 효능을 보여주었습니다. 이 약물은 기존 시험과 일관된 안전성 프로필을 나타내었고 중대한 부작용은 없었습니다. 이 치료는 스타틴에 충분한 반응을 보이지 않거나 이를 견딜 수 없는 환자들을 대상으로 합니다. 벡페도산은 간의 콜레스테롤 합성 경로에서 ATP 시트레이트 리아제를 억제하여 작용하며, 이미 미국과 유럽에서 판매되고 있습니다.
Esperion (NASDAQ: ESPR) a annoncé qu'Otsuka Pharmaceutical a soumis une Demande d'Autorisation de Mise sur le Marché au Japon pour l'acide bempedoïque afin de traiter l'hypercholestérolémie. L'essai clinique de Phase 3 au Japon, impliquant 96 patients, a démontré une efficacité significative avec un changement de -25,25% des niveaux de LDL-C par rapport à -3,46% dans le groupe placebo après 12 semaines. Le médicament a montré un profil de sécurité cohérent avec les essais précédents et aucun événement indésirable grave. Ce traitement cible les patients qui ont une réponse insuffisante aux statines ou qui ne peuvent pas les tolérer. L'acide bempedoïque agit en inhibant l'ATP citrate lyase dans la voie de synthèse du cholestérol dans le foie et est déjà commercialisé aux États-Unis et en Europe.
Esperion (NASDAQ: ESPR) gab bekannt, dass Otsuka Pharmaceutical einen Antrag auf Zulassung eines neuen Arzneimittels in Japan für Bempedoinsäure zur Behandlung von Hypercholesterinämie eingereicht hat. Die japanische Phase-3-Studie, an der 96 Patienten beteiligt waren, zeigte eine signifikante Wirksamkeit mit einer -25,25%igen Veränderung der LDL-C-Werte im Vergleich zu -3,46% in der Placebo-Gruppe nach 12 Wochen. Das Medikament zeigte ein konsistentes Sicherheitsprofil im Vergleich zu vorherigen Studien und keine schwerwiegenden Nebenwirkungen. Diese Behandlung richtet sich an Patienten, die unzureichend auf Statine ansprechen oder diese nicht vertragen können. Bempedoinsäure wirkt, indem sie die ATP-Zitratlyase im Cholesterinsyntheseweg der Leber hemmt und ist bereits in den USA und Europa auf dem Markt.
- Phase 3 trial showed statistically significant LDL-C reduction (-25.25% vs -3.46% placebo)
- NDA submission in Japan expands potential market reach
- No serious adverse events reported in Japanese trial
- Addresses unmet medical need for statin-intolerant patients
- Relatively small trial size (96 patients) could raise questions about broader applicability
- Multiple safety warnings including hyperuricemia and tendon rupture risks
- Various adverse reactions reported in trials including liver enzyme elevation
Insights
The NDA submission in Japan represents a significant milestone for Esperion's global expansion. The Phase 3 trial results showing a
The Japanese market presents a substantial commercial opportunity, with approximately
This regulatory milestone could significantly impact Esperion's revenue stream through potential milestone payments and future royalties from Otsuka. Japan represents the world's third-largest pharmaceutical market, offering substantial commercial potential. The 2020 licensing agreement with Otsuka provides validation of bempedoic acid's market value and reduces Esperion's commercialization risks in the region.
The positive trial results and safety profile increase the probability of regulatory success, though investors should note that approval timelines in Japan typically range from 9-12 months. A successful launch could help strengthen Esperion's financial position and global market presence.
ANN ARBOR, Mich., Nov. 26, 2024 (GLOBE NEWSWIRE) -- Esperion (NASDAQ: ESPR) today announced that Otsuka Pharmaceutical Co., Ltd. (Otsuka) has submitted a New Drug Application (NDA) to the Japanese Ministry of Health, Labour and Welfare for the manufacture and sale of bempedoic acid in Japan for the treatment of hypercholesterolemia and familial hypercholesterolemia.
Bempedoic acid has a novel mechanism of action that inhibits a cholesterol synthesis pathway by acting on ATP (adenosine triphosphate) citrate lyase, a citrate-degrading enzyme in the liver. Bempedoic acid is marketed for the treatment of hypercholesterolemia in several regions around the world, including the United States and Europe. In 2020, Otsuka acquired exclusive development and commercialization rights for bempedoic acid in Japan from Esperion and is currently developing it domestically.
The Japanese Phase 3 trial was conducted as a placebo-controlled, randomized, multicenter, double-blind, parallel-group comparative study, in 96 patients with high LDL cholesterol and in whom statins have insufficient effect or cannot be tolerated. Trial participants were administered either 180 mg of bempedoic acid or a placebo, orally, once a day, for 12 weeks to evaluate the efficacy and safety of bempedoic acid. In the preliminary results, the percentage change from baseline in LDL-C at Week 12, the primary endpoint, was -25.25 percent in the group receiving bempedoic acid group and -3.46 percent in the placebo group, demonstrating positive outcomes with statistical significance compared to placebo (p<0.001). Furthermore, the safety and tolerability of bempedoic acid were consistent with findings from previous trials, and no serious adverse events were observed.
Some patients with hypercholesterolemia are unable to achieve their target values even when taking statins (insufficient response to statins), or they are unable to continue taking statins due to the occurrence of adverse events associated with statin use (statin intolerance). This drug candidate in Japan is expected to become a new treatment option for hypercholesterolemic patients with insufficient response to statins or statin intolerance.
IMPORTANT SAFETY INFORMATION
NEXLIZET and NEXLETOL are contraindicated in patients with a prior hypersensitivity to bempedoic acid or ezetimibe or any of the excipients. Serious hypersensitivity reactions including anaphylaxis, angioedema, rash, and urticaria have been reported.
Hyperuricemia: Bempedoic acid, a component of NEXLIZET and NEXLETOL, may increase blood uric acid levels, which may lead to gout. Hyperuricemia may occur early in treatment and persist throughout treatment, returning to baseline following discontinuation of treatment. Assess uric acid levels periodically as clinically indicated. Monitor for signs and symptoms of hyperuricemia, and initiate treatment with urate-lowering drugs as appropriate.
Tendon Rupture: Bempedoic acid, a component of NEXLIZET and NEXLETOL, is associated with an increased risk of tendon rupture or injury. Tendon rupture may occur more frequently in patients over 60 years of age, in those taking corticosteroid or fluoroquinolone drugs, in patients with renal failure, and in patients with previous tendon disorders. Discontinue NEXLIZET or NEXLETOL at the first sign of tendon rupture. Consider alternative therapy in patients who have a history of tendon disorders or tendon rupture.
The most common adverse reactions in the primary hyperlipidemia trials of bempedoic acid, a component of NEXLIZET and NEXLETOL, in ≥
Adverse reactions reported in ≥
In the primary hyperlipidemia trials of NEXLIZET, the most commonly reported adverse reactions (incidence ≥
The most common adverse reactions in the cardiovascular outcomes trial for bempedoic acid, a component of NEXLIZET and NEXLETOL, at an incidence of ≥
Discontinue NEXLIZET or NEXLETOL when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. Because of the potential for serious adverse reactions in a breast-fed infant, breastfeeding is not recommended during treatment with NEXLIZET or NEXLETOL.
Report pregnancies to Esperion Therapeutics, Inc. Adverse Event reporting line at 1-833-377-7633.
Please see full Prescribing Information for NEXLIZET and NEXLETOL.
Esperion Therapeutics
At Esperion, we discover, develop, and commercialize innovative medicines to help improve outcomes for patients with or at risk for cardiovascular and cardiometabolic diseases. The status quo is not meeting the health needs of millions of people with high cholesterol – that is why our team of passionate industry leaders is breaking through the barriers that prevent patients from reaching their goals. Providers are moving toward reducing LDL-cholesterol levels as low as possible, as soon as possible; we provide the next steps to help get patients there. Because when it comes to high cholesterol, getting to goal is not optional. It is our life’s work. For more information, visit esperion.com and esperionscience.com and follow us on X at twitter.com/EsperionInc.
Forward-Looking Statements
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Esperion Contact Information:
Investors:
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FAQ
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