Mereo BioPharma Provides Update on Lead Clinical Programs
Mereo BioPharma (NASDAQ: MREO) provided updates on its lead clinical programs. The Phase 3 Orbit Study of setrusumab for Osteogenesis Imperfecta (OI) is progressing toward a second interim analysis expected in mid-2025, with potential final analysis in Q4 2025. The Cosmic study, evaluating setrusumab against bisphosphonate therapy in younger patients, continues in parallel.
Alvelestat, their treatment for alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD), received a positive opinion from EMA's Committee for Orphan Medicinal Products, with final European Commission decision expected in Q1 2025. The drug previously received FDA Orphan Drug and Fast Track Designations.
The company confirmed its current cash position will fund operations into 2027, supporting key development milestones through 2025.
Mereo BioPharma (NASDAQ: MREO) ha fornito aggiornamenti sui suoi principali programmi clinici. Lo Studio Orbit di Fase 3 su setrusumab per l'Osteogenesi Imperfetta (OI) sta procedendo verso una seconda analisi intermedia prevista per metà 2025, con un'analisi finale potenziale nel Q4 2025. Lo studio Cosmic, che valuta il setrusumab rispetto alla terapia con bisfosfonati nei pazienti più giovani, continua in parallelo.
Alvelestat, il loro trattamento per la malattia polmonare associata a deficienza di alfa-1 antitripsina (AATD-LD), ha ricevuto un parere positivo dal Comitato per i Medicinali Orfani dell'EMA, con la decisione finale della Commissione Europea attesa nel Q1 2025. Il farmaco ha precedentemente ricevuto le designazioni di Farmaco Orfano e Fast Track dalla FDA.
L'azienda ha confermato che la sua attuale posizione di liquidità finanzierà le operazioni fino al 2027, supportando i principali traguardi di sviluppo fino al 2025.
Mereo BioPharma (NASDAQ: MREO) proporcionó actualizaciones sobre sus principales programas clínicos. El Estudio Orbit de Fase 3 sobre setrusumab para la Osteogénesis Imperfecta (OI) avanza hacia un segundo análisis intermedio que se espera para mediados de 2025, con un análisis final potencial en el cuarto trimestre de 2025. El estudio Cosmic, que evalúa setrusumab en comparación con la terapia con bisfosfonatos en pacientes más jóvenes, continúa en paralelo.
Alvelestat, su tratamiento para la enfermedad pulmonar asociada a la deficiencia de alfa-1 antitripsina (AATD-LD), recibió un dictamen positivo del Comité de Medicamentos Huérfanos de la EMA, con la decisión final de la Comisión Europea esperada para el primer trimestre de 2025. El medicamento previamente recibió designaciones de Medicamento Huérfano y de Ruta Rápida de la FDA.
La empresa confirmó que su posición de efectivo actual financiará las operaciones hasta 2027, apoyando los hitos clave de desarrollo hasta 2025.
Mereo BioPharma (NASDAQ: MREO)는 주요 임상 프로그램에 대한 업데이트를 제공했습니다. 세트루수맙(setrusumab)에 대한 3상 Orbit 연구가 골형성부전증(OI)을 대상으로 하여 2025년 중반에 예상되는 두 번째 중간 분석을 향해 진행 중이며, 2025년 4분기에 최종 분석이 가능할 수 있습니다. 젊은 환자를 대상으로 세트루수맙과 이옴산염 치료를 비교하는 Cosmic 연구가 병행하여 진행 중입니다.
알벨라세탐(Alvelestat), 알파-1 항트립신 결핍에 관련된 폐 질환(AATD-LD) 치료제는 EMA의 고아 의약품 위원회로부터 긍정적인 의견을 받았으며, 최종 유럽 위원회의 결정은 2025년 1분기에 예상됩니다. 이 약물은 이전에 FDA에서 고아 약물 및 패스트 트랙 지정을 받았습니다.
회사는 현재 현금 보유량이 2027년까지 운영을 지원하며 2025년까지 주요 개발 이정표를 지원할 것이라고 확인했습니다.
Mereo BioPharma (NASDAQ: MREO) a fourni des mises à jour sur ses principaux programmes cliniques. L'étude Orbit de phase 3 sur setrusumab pour l'ostéogenèse imparfaite (OI) progresse vers une seconde analyse intermédiaire attendue mi-2025, avec une analyse finale potentielle au quatrième trimestre 2025. L'étude Cosmic, qui évalue le setrusumab par rapport à la thérapie aux bisphosphonates chez les patients plus jeunes, se poursuit en parallèle.
Alvelestat, leur traitement pour la maladie pulmonaire associée à un déficit en alpha-1-antitrypsine (AATD-LD), a reçu un avis favorable du Comité pour les Médicaments Orphelins de l'EMA, avec une décision finale de la Commission Européenne attendue au premier trimestre 2025. Le médicament a précédemment reçu les désignations de Médicament Orphelin et de Voie Accélérée par la FDA.
L'entreprise a confirmé que sa position actuelle de liquidité financera ses opérations jusqu'en 2027, soutenant les principales étapes de développement jusqu'en 2025.
Mereo BioPharma (NASDAQ: MREO) hat Aktualisierungen zu seinen führenden klinischen Programmen bereitgestellt. Die Phase-3-Studie Orbit zu setrusumab für Osteogenesis Imperfecta (OI) schreitet auf eine zweite Zwischenanalyse zu, die für Mitte 2025 erwartet wird, mit einer möglichen finalen Analyse im vierten Quartal 2025. Die Cosmic-Studie, die setrusumab mit Bisphosphonattherapie bei jüngeren Patienten vergleicht, wird parallel fortgeführt.
Alvelestat, ihre Behandlung für die Lungenkrankheit im Zusammenhang mit alpha-1-Antitrypsinmangel (AATD-LD), erhielt eine positive Stellungnahme des Ausschusses für Orphan-Arzneimittel der EMA, mit einer endgültigen Entscheidung der Europäischen Kommission, die im ersten Quartal 2025 erwartet wird. Das Medikament erhielt zuvor von der FDA die Auszeichnungen für ein Waisenmedikament und Fast Track.
Das Unternehmen bestätigte, dass seine aktuelle Liquiditätsposition die Betriebe bis 2027 finanzieren wird, und unterstützt wichtige Entwicklungsmeilensteine bis 2025.
- Phase 3 Orbit Study of setrusumab progressing with key analysis milestones in 2025
- Positive EMA opinion received for Alvelestat's European Orphan Designation
- Strong cash position extending operations into 2027
- Potential 10-year marketing exclusivity in EU upon Alvelestat approval
- None.
Insights
The clinical development updates for Mereo BioPharma's two lead programs represent significant milestones in their advancement. The Phase 3 Orbit study for setrusumab is approaching a important second interim analysis that could validate its potential in treating Osteogenesis Imperfecta. The parallel Cosmic study in younger patients (2-7 years) provides complementary data that could support a broader label indication.
The positive COMP opinion for alvelestat's Orphan Designation in Europe adds to its existing FDA Orphan Drug and Fast Track Designations. This regulatory pathway optimization could accelerate market access and provide 10 years of marketing exclusivity in Europe, significantly enhancing the commercial potential.
The company's
From a market perspective, these developments strengthen Mereo's position in the rare disease space. The Orphan Designation for alvelestat unlocks valuable commercial benefits including:
- Market exclusivity protection
- Fee reductions for regulatory activities
- Enhanced pricing power during reimbursement negotiations
With a
The regulatory developments for alvelestat are particularly noteworthy given the unmet need in AATD-LD treatment. The positive COMP opinion signals recognition of alvelestat's potential therapeutic value, especially for earlier-stage patients currently ineligible for augmentation therapy in many countries. This could expand the addressable patient population and improve access to treatment. The multi-regional regulatory strategy (US/EU) demonstrates a comprehensive approach to market access, potentially accelerating the path to commercialization in key markets.
Orbit Phase 3 study of setrusumab in osteogenesis imperfecta continuing to planned second interim analysis, expected in mid-2025
Alvelestat, for Alpha-1 Antitrypsin Deficiency-associated Lung Disease, receives positive EMA opinion on European Orphan Designation Application; European Commission expected to issue final decision in first quarter 2025
LONDON, Jan. 12, 2025 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today provided an update on its lead clinical programs, setrusumab, a monoclonal antibody in Phase 3 clinical development for the treatment of Osteogenesis Imperfecta (OI) through a partnership with Ultragenyx Pharmaceutical, Inc. (Ultragenyx) and alvelestat, an oral neutrophil elastase inhibitor being studied for the treatment of alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD). The Company also reiterated its previous cash runway guidance that its current cash and cash equivalents are expected to fund operations into 2027, through multiple key inflection points.
“Based on the highly promising data from completed studies of setrusumab in OI, including the Phase 2 portion of the Orbit Study, we remain confident in the potential of setrusumab to become the standard-of-care in OI. We look forward to the second interim analysis expected mid-year as we continue our launch readiness activities in the key European markets,” said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo BioPharma. “Additionally, EU Orphan Designation, which follows the granting of both Orphan Drug and Fast Track Designations from the FDA in the U.S., is another important milestone in our ongoing partnering process and our efforts to bring alvelestat to patients worldwide, including earlier stage patients who are not currently eligible for augmentation therapy in many countries. With our cash runway into 2027, we continue to be in a strong position to execute on our key milestones through 2025.”
Setrusumab (UX143)
As announced by the Company’s partner, Ultragenyx, the Phase 3 Orbit Study of setrusumab in OI is continuing to dose patients and progressing towards the planned second interim analysis expected in mid-2025, with a potential final analysis in the fourth quarter of 2025. Additionally, treatment is continuing in Cosmic, an open-label Phase 3 study evaluating setrusumab against intravenous bisphosphonate therapy in patients aged 2 to <7 years. Data from the Cosmic study will be evaluated in parallel with the Orbit interim and final analyses.
Alvelestat (MPH-966)
The European Medicines Agency (EMA)’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on the Company’s application for Orphan Designation for alvelestat. The COMP recommendation has been provided to the European Commission, which is expected to issue a final decision on the Orphan Designation in the first quarter of 2025. Alvelestat previously received Orphan Drug Designation and Fast Track Designation from the U.S. FDA in 2021 and 2022, respectively.
European Orphan Designation is awarded to therapeutic candidates targeting the treatment, prevention or diagnosis of life-threatening or chronically debilitating diseases with a prevalence of fewer than 5 in 10,000 people in the European Union which provide a significant benefit over available therapies, or for which no approved therapies exist. Therapeutics receiving EU Orphan Designation are eligible for ten years of marketing exclusivity upon approval, as well as fee reductions for various centralized activities including the Marketing Authorization Application, inspections and protocol assistance. Individual EU Member States also provide specific incentives to support the development, review and availability of Orphan Medicinal Products at the time of HTA evaluations and Pricing and Reimbursement negotiations.
About Mereo BioPharma
Mereo BioPharma is a biopharmaceutical company focused on the development of innovative therapeutics for rare diseases. The Company has two rare disease product candidates: setrusumab for the treatment of osteogenesis imperfecta (OI); and alvelestat, primarily for the treatment of severe alpha-1 antitrypsin deficiency-associated lung disease (AATD-LD). The Company’s partner, Ultragenyx Pharmaceutical, Inc., has completed enrollment in the Phase 3 portion of a pivotal Phase 2/3 study in pediatrics and young adults (5 to 25 years old) for setrusumab in OI and in the Phase 3 study in pediatric patients (2 to <7 years old) in the first half of 2024. The partnership with Ultragenyx includes potential additional milestone payments of up to
Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties. All statements other than statements of historical fact contained herein are forward-looking statements within the meaning of Section 27A of the United States Securities Act of 1933, as amended, and Section 21E of the United States Securities Exchange Act of 1934, as amended. Forward-looking statements usually relate to future events and anticipated revenues, earnings, cash flows or other aspects of our operations or operating results. Forward-looking statements are often identified by the words “believe,” “expect,” “anticipate,” “plan,” “intend,” “foresee,” “should,” “would,” “could,” “may,” “estimate,” “outlook” and similar expressions, including the negative thereof. The absence of these words, however, does not mean that the statements are not forward-looking. These forward-looking statements are based on the Company’s current expectations, beliefs and assumptions concerning future developments and business conditions and their potential effect on the Company. While management believes that these forward-looking statements are reasonable as and when made, there can be no assurance that future developments affecting the Company will be those that it anticipates.
All of the Company’s forward-looking statements involve known and unknown risks and uncertainties some of which are significant or beyond its control and assumptions that could cause actual results to differ materially from the Company’s historical experience and its present expectations or projections. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical development process; the Company’s reliance on third parties to conduct and provide funding for its clinical trials; the Company’s dependence on enrollment of patients in its clinical trials; and the Company’s dependence on its key executives. You should carefully consider the foregoing factors and the other risks and uncertainties that affect the Company’s business, including those described in the “Risk Factors” section of its Annual Report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in the Company’s subsequent filings with the Securities and Exchange Commission. The Company wishes to caution you not to place undue reliance on any forward-looking statements, which speak only as of the date hereof. The Company undertakes no obligation to publicly update or revise any of our forward-looking statements after the date they are made, whether as a result of new information, future events or otherwise, except to the extent required by law.
Mereo BioPharma Contacts: | ||
Mereo | +44 (0)333 023 7300 | |
Denise Scots-Knight, Chief Executive Officer | ||
Christine Fox, Chief Financial Officer | ||
Burns McClellan (Investor Relations Adviser to Mereo) | +01 646 930 4406 | |
Lee Roth | ||
Investors | investors@mereobiopharma.com |
FAQ
When is Mereo BioPharma's (MREO) second interim analysis for the setrusumab Phase 3 Orbit Study expected?
What regulatory designations has MREO's alvelestat received for AATD-LD treatment?
How long will MREO's current cash reserves last?
What benefits does European Orphan Designation provide for MREO's alvelestat?