Esperion Aligns with U.S. Food and Drug Administration to Initiate Phase 3 Clinical Trials of Bempedoic Acid in Pediatric Heterozygous and Homozygous Familial Hypercholesterolemia
Esperion (NASDAQ: ESPR) has received FDA alignment to proceed with Phase 3 clinical trials of bempedoic acid, both alone and combined with ezetimibe, for pediatric patients with heterozygous and homozygous familial hypercholesterolemia (HeFH and HoFH).
The company plans to initiate these Phase 3 studies in 2025, following successful completion of Phase 2 clinical study enrollment. The FDA has previously granted orphan drug designation for bempedoic acid in HoFH indication.
This development will allow Esperion to extend its patent protection for an additional six months through June 2031, as part of their lifecycle management plan for bempedoic acid products.
Esperion (NASDAQ: ESPR) ha ricevuto l'allineamento della FDA per procedere con i trial clinici di Fase 3 dell'acido bempedoico, sia da solo che in combinazione con ezetimibe, per pazienti pediatrici affetti da ipercolesterolemia familiare eterozigote e omozigote (HeFH e HoFH).
L'azienda prevede di avviare questi studi di Fase 3 nel 2025, dopo il completamento con successo dell'arruolamento nello studio clinico di Fase 2. La FDA ha precedentemente concesso la designazione di farmaco orfano per l'acido bempedoico nell'indicazione HoFH.
Questo sviluppo consentirà a Esperion di estendere la sua protezione brevettuale per sei mesi aggiuntivi fino a giugno 2031, come parte del loro piano di gestione del ciclo di vita per i prodotti a base di acido bempedoico.
Esperion (NASDAQ: ESPR) ha recibido la alineación de la FDA para proceder con los ensayos clínicos de Fase 3 del ácido bempedoico, tanto solo como en combinación con ezetimibe, para pacientes pediátricos con hipercolesterolemia familiar heterocigota y homocigota (HeFH y HoFH).
La compañía planea iniciar estos estudios de Fase 3 en 2025, tras la finalización exitosa de la inscripción en el estudio clínico de Fase 2. La FDA ha otorgado previamente la designación de medicamento huérfano para el ácido bempedoico en la indicación HoFH.
Este desarrollo permitirá a Esperion extender su protección de patente por seis meses adicionales hasta junio de 2031, como parte de su plan de gestión del ciclo de vida para los productos de ácido bempedoico.
Esperion (NASDAQ: ESPR)는 3상 임상 시험을 진행하기 위해 FDA의 승인을 받았습니다. 이는 단독으로 또는 에제티미브와 병용하여 이형접합 및 동형접합 가족 고콜레스테롤혈증(HeFH 및 HoFH) 환자를 대상으로 합니다.
회사는 2025년에 이러한 3상 연구를 시작할 계획이며, 이는 2상 임상 연구 등록이 성공적으로 완료된 후에 이루어집니다. FDA는 HoFH 적응증에 대해 bempedoic acid의 희귀의약품 지정을 이전에 부여한 바 있습니다.
이번 개발은 Esperion이 bempedoic acid 제품의 생애 주기 관리 계획의 일환으로 2031년 6월까지 추가로 6개월 동안 특허 보호를 연장할 수 있게 해줍니다.
Esperion (NASDAQ: ESPR) a reçu l'alignement de la FDA pour procéder aux essais cliniques de Phase 3 de l'acide bempedoïque, à la fois seul et en combinaison avec l'ézétimibe, pour les patients pédiatriques atteints d'hypercholestérolémie familiale hétérozygote et homozygote (HeFH et HoFH).
L'entreprise prévoit de commencer ces études de Phase 3 en 2025, après avoir réussi à compléter l'inscription à l'étude clinique de Phase 2. La FDA a précédemment accordé la dénomination de médicament orphelin pour l'acide bempedoïque dans l'indication HoFH.
Ce développement permettra à Esperion d'étendre sa protection par brevet de six mois supplémentaires jusqu'en juin 2031, dans le cadre de son plan de gestion du cycle de vie pour les produits à base d'acide bempedoïque.
Esperion (NASDAQ: ESPR) hat die Genehmigung der FDA erhalten, um mit den Phase-3-Studien zu Bempedoinsäure fortzufahren, sowohl allein als auch in Kombination mit Ezetimib, für pädiatrische Patienten mit heterozygoter und homozygoter familiärer Hypercholesterinämie (HeFH und HoFH).
Das Unternehmen plant, diese Phase-3-Studien im Jahr 2025 zu starten, nachdem die Einschreibung für die Phase-2-Studie erfolgreich abgeschlossen wurde. Die FDA hat zuvor die Orphan-Drug-Bezeichnung für Bempedoinsäure bei der HoFH-Indikation erteilt.
Diese Entwicklung ermöglicht es Esperion, den Patentschutz um weitere sechs Monate bis Juni 2031 zu verlängern, als Teil ihres Lebenszyklusmanagementplans für Produkte auf Basis von Bempedoinsäure.
- FDA approval to proceed with Phase 3 trials expands market potential to pediatric patients
- Orphan drug designation secured for HoFH indication
- Patent protection extension through June 2031
- Additional clinical trials will require significant time and resources
- Phase 3 results and eventual FDA approval still uncertain
– Confirms Sufficient Data to Complete Phase 2 Clinical Study Enrollment and Advance to Phase 3 Studies in Both Heterozygous and Homozygous Familial Hypercholesterolemia –
– Establishes Pediatric Path Forward to Start and Complete Phase 3 Trial and Secure Additional Six-Month Patent Extension Through June 2031 –
ANN ARBOR, Mich., March 20, 2025 (GLOBE NEWSWIRE) -- Esperion (NASDAQ: ESPR) today announced that following meetings with the U.S. Food and Drug Administration (FDA), it has gained alignment on a regulatory path forward for initiating Phase 3 studies of bempedoic acid alone and in combination with ezetimibe in pediatric patients with heterozygous and homozygous familial hypercholesterolemia (HeFH and HoFH, respectively). Based on these discussions with the FDA, the Company plans to initiate Phase 3 clinical studies this year. The FDA previously granted orphan drug designation for bempedoic acid in HoFH indication.
“We are delighted to further advance the development of bempedoic acid for children with familial hypercholesterolemia (FH) and are pleased the FDA has indicated that we have adequate data to proceed into Phase 3 clinical trials,” stated Sheldon Koenig, President and CEO of Esperion. “In addition, this alignment supports our commitment to broaden the reach of our bempedoic acid products as part of our lifecycle management plan, providing the opportunity to extend our patent protection for an additional six months for these important therapies,” added Sheldon Koenig.
About CLEAR Path 2 and CLEAR Path 3 Studies
CLEAR Path 2 (in children with HeFH) and CLEAR Path 3 (in children with HoFH) are Phase 3, randomized double-blind, placebo-controlled, multicenter studies to evaluate the efficacy and safety of bempedoic acid with and without concurrent ezetimibe in children with HeFH or HoFH and LDL-C ≥130 mg/dL (3.4 mmol/L) while on protocol defined optimum dose of a statin. Each study is a 52-week design where patients will be randomized 2:1 to treatment or control for 24 weeks, followed by 28 weeks of an open-label extension period.
About Heterozygous Familial Hypercholesterolemia (HeFH) and Homozygous FH (HoFH)
HeFH and HoFH are the two types of FH, a genetic condition that causes high cholesterol from birth and if untreated leads to early, aggressive atherosclerotic cardiovascular disease. HeFH is the more common form occurring in 1 in 250 births and results from inheriting a gene that causes FH from one parent. If left untreated, cardiovascular disease can develop during middle adulthood. HoFH is the rarer form of the disease, an orphan indication, occurring in 1 in 300,000 births and results from inheriting a FH gene from both parents. HoFH results in higher cholesterol levels, which are often more difficult to treat as cardiovascular disease can begin as early as in childhood if untreated. In both HeFH and HoFH, the processing of LDL-C is disrupted due to these gene mutations, which leads to dangerously high levels of LDL-C. Both HeFH and HoFH are underdiagnosed and undertreated but with early detection and continued development of LDL-C therapies for patients starting at an early age, there is hope that patients with FH can control their LDL cholesterol and reduce their risk of cardiovascular disease.
About Esperion Therapeutics
Esperion Therapeutics, Inc. is a commercial stage biopharmaceutical company focused on bringing new medicines to market that address unmet needs of patients and healthcare professionals. The Company developed and is commercializing the only U.S. Food and Drug Administration (FDA) approved oral, once-daily, non-statin medicines for patients who are at risk for cardiovascular disease and are struggling with elevated low density lipoprotein cholesterol (LDL-C). These medications are supported by the nearly 14,000 patient CLEAR Cardiovascular Outcomes Trial. Esperion continues to build on its success with its next generation program which is focused on developing ATP citrate lyase inhibitors (ACLYi). New insights into the structure and function of ACLYi fully enables rational drug design and the opportunity to develop highly potent and specific inhibitors with allosteric mechanisms.
Esperion continues to evolve into a leading global biopharmaceutical company through commercial execution, international partnerships and collaborations and advancement of its pre-clinical pipeline. For more information, visit esperion.com and follow Esperion on LinkedIn and X.
Forward-Looking Statements
This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements regarding marketing strategy and commercialization plans, current and planned operational expenses, future operations, commercial products, clinical development, including the timing, designs and plans for the CLEAR Outcomes study and its results, plans for potential future product candidates, financial condition and outlook, including expected cash runway, and other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “suggest,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions. Any express or implied statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements involve risks and uncertainties that could cause Esperion’s actual results to differ significantly from those projected, including, without limitation, the net sales, profitability, and growth of Esperion’s commercial products, clinical activities and results, supply chain, commercial development and launch plans, the outcomes and anticipated benefits of legal proceedings and settlements, and the risks detailed in Esperion’s filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Esperion disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.
Esperion Contact Information:
Investors:
Alina Venezia
investorrelations@esperion.com
(734) 887-3903
Media:
Tiffany Aldrich
corporateteam@esperion.com
(616) 443-8438
FAQ
When will Esperion (ESPR) begin Phase 3 trials for pediatric familial hypercholesterolemia?
How long will Esperion's (ESPR) patent protection extend for bempedoic acid?
What FDA designations has ESPR's bempedoic acid received for familial hypercholesterolemia?
Which patient populations will ESPR's Phase 3 trials target?
