Patient Enrollment Commenced in Pivotal Phase 3 Trial of Rexlemestrocel-L for Chronic Low Back Pain
Mesoblast (Nasdaq:MESO; ASX:MSB) has commenced enrollment for its confirmatory Phase 3 trial of rexlemestrocel-L in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease. The trial, aligned with FDA guidelines, involves 300 patients across multiple U.S. sites. The primary endpoint is pain reduction at 12 months, with secondary measures including improved quality of life, function, and reduced opioid usage.
The FDA has granted rexlemestrocel-L Regenerative Medicine Advanced Therapy (RMAT) designation, providing benefits such as rolling review and priority review eligibility. Dr. Alan Miller, a trial investigator, describes the therapy as potentially groundbreaking for the low back pain population.
Mesoblast (Nasdaq:MESO; ASX:MSB) ha avviato l'arruolamento per il suo studio di conferma di fase 3 su rexlemestrocel-L in pazienti con dolore lombare cronico (CLBP) causato da malattia degenerativa infiammatoria del disco. Lo studio, conforme alle linee guida della FDA, prevede 300 pazienti in diversi centri negli Stati Uniti. L'obiettivo principale è la riduzione del dolore a 12 mesi, con misure secondarie che includono un miglioramento della qualità della vita, della funzionalità e una riduzione dell'uso di oppioidi.
La FDA ha conferito a rexlemestrocel-L la designazione di Terapia Avanzata di Medicina Rigenerativa (RMAT), offrendo vantaggi come la revisione continua e l'idoneità per la revisione prioritaria. Il dott. Alan Miller, un investigatore dello studio, descrive la terapia come potenzialmente rivoluzionaria per la popolazione con dolore lombare.
Mesoblast (Nasdaq:MESO; ASX:MSB) ha iniciado el reclutamiento para su ensayo de confirmación de fase 3 de rexlemestrocel-L en pacientes con dolor lumbar crónico (CLBP) debido a enfermedad degenerativa inflamatoria del disco. El ensayo, alineado con las pautas de la FDA, involucra 300 pacientes en múltiples sitios de EE. UU. El objetivo primario es la reducción del dolor a los 12 meses, con medidas secundarias que incluyen la mejora de la calidad de vida, la función y la reducción del uso de opioides.
La FDA ha otorgado a rexlemestrocel-L la designación de Terapia Avanzada en Medicina Regenerativa (RMAT), que ofrece beneficios como revisión continua y elegibilidad para revisión prioritaria. El Dr. Alan Miller, un investigador del ensayo, describe la terapia como potencialmente revolucionaria para la población con dolor lumbar.
메조블라스트(Mesoblast)(Nasdaq:MESO; ASX:MSB)는 환자 모집을 시작했습니다 상 confirmatory 3상 시험을 rexlemestrocel-L에 대해 만성 요통 (CLBP)으로 인해 염증성 퇴행성 디스크 질환을 가진. 이 시험은 FDA 지침에 따라 진행되며, 300명의 환자를 여러 미국 사이트에서 모집할 예정입니다. 주요 목표는 12개월 동안의 통증 감소이며, 2차 측정으로는 삶의 질 개선, 기능 향상 및 오피오이드 사용 감소가 포함됩니다.
FDA는 rexlemestrocel-L에게 재생 의학 고급 치료(RMAT) 지정을 부여했습니다, 연속 검토 및 우선 검토 자격과 같은 혜택을 제공합니다. 임상 시험 조사자인 앨런 밀러 박사는 이 치료법이 요통 환자에게 혁신적일 가능성이 있다고 설명합니다.
Mesoblast (Nasdaq:MESO; ASX:MSB) a commencé le recrutement pour son essai de phase 3 de confirmation sur le rexlemestrocel-L chez des patients souffrant de douleurs lombaires chroniques (CLBP) dues à une maladie dégénérative discale inflammatoire. L'essai, conforme aux directives de la FDA, implique 300 patients dans plusieurs sites aux États-Unis. Le principal objectif est la réduction de la douleur après 12 mois, avec des mesures secondaires comprenant l'amélioration de la qualité de vie, de la fonction et la réduction de l'utilisation des opioïdes.
La FDA a accordé à rexlemestrocel-L la désignation de Thérapie Avancée en Médecine Régénérative (RMAT), offrant des avantages tels que l'examen continu et l'éligibilité pour un examen prioritaire. Le Dr Alan Miller, un investigateur de l'essai, décrit cette thérapie comme potentiellement révolutionnaire pour la population souffrant de douleurs lombaires.
Mesoblast (Nasdaq:MESO; ASX:MSB) hat die Rekrutierung für seine bestätigende Phase-3-Studie zu rexlemestrocel-L bei Patienten mit chronischen Rückenschmerzen (CLBP) aufgrund von entzündlicher degenerativer Bandscheibenerkrankung begonnen. Die Studie, die den Richtlinien der FDA entspricht, umfasst 300 Patienten an mehreren Standorten in den USA. Der Primärendpunkt ist die Schmerzlinderung nach 12 Monaten, wobei sekundäre Maßnahmen die Verbesserung der Lebensqualität, der Funktion und die Reduzierung des Opioidgebrauchs umfassen.
Die FDA hat rexlemestrocel-L die Bezeichnung als Regenerative Medizin Fortschrittliche Therapie (RMAT) gewährte, die Vorteile wie die laufende Überprüfung und die Berechtigung zur Prioritätsüberprüfung bietet. Dr. Alan Miller, ein Prüfungsleiter, beschreibt die Therapie als potenziell bahnbrechend für die Bevölkerung mit Rückenschmerzen.
- Commencement of patient enrollment in pivotal Phase 3 trial for chronic low back pain treatment
- FDA alignment on trial design and primary endpoint
- Regenerative Medicine Advanced Therapy (RMAT) designation granted by FDA
- Potential for priority review and rolling review of Biologics License Application (BLA)
- None.
Insights
Mesoblast's announcement of the commencement of patient enrollment in their pivotal Phase 3 trial for rexlemestrocel-L is highly significant in the medical research field. The designation of rexlemestrocel-L as a Regenerative Medicine Advanced Therapy (RMAT) by the FDA underscores the potential impact of this therapy on chronic low back pain treatment. The RMAT designation, with its benefits of rolling review and eligibility for priority review, expedites the developmental timeline, potentially leading to faster patient access if the trial results are positive.
From a research perspective, the focus on a 12-month primary endpoint of pain reduction and secondary measures such as quality of life, function improvement and reduced opioid usage is comprehensive. It addresses key patient outcomes that can transform the management of chronic low back pain, a condition with limited effective long-term treatments currently. The randomized, placebo-controlled design of the trial enhances the credibility and reliability of the findings, important for future approval and clinical adoption.
The initiation of a Phase 3 trial for rexlemestrocel-L is pivotal for Mesoblast from a financial standpoint. Successful progression through this phase could significantly increase the company's valuation and market confidence. The RMAT designation augments this potential by facilitating a more efficient review process, which can lead to earlier market entry and revenue generation.
Investors should note that this trial's success could open substantial market opportunities. Chronic low back pain affects a large population and a new, effective treatment could capture significant market share, especially if it proves to reduce opioid dependency, aligning with growing public health initiatives against opioid misuse. The market for non-opioid pain management solutions is expanding and a successful trial could position Mesoblast as a leader in this sector.
From a market research perspective, the commencement of the Phase 3 trial for rexlemestrocel-L is a critical development. The chronic pain management market is vast and highly competitive and a new entrant with RMAT designation can disrupt the current landscape. The trial's outcomes could significantly influence market dynamics, particularly in the segment focused on non-opioid pain relief.
Additionally, the approval of rexlemestrocel-L could align with broader industry trends towards regenerative medicine and biologics, which are gaining traction due to their potential for long-term efficacy and reduced side effects. The successful development of this therapy could enhance Mesoblast's competitive positioning and open doors for further innovations in regenerative treatments for other inflammatory conditions.
NEW YORK, July 21, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, announced today that the confirmatory Phase 3 trial of its allogeneic, immunoselected, and industrially manufactured stromal cell product rexlemestrocel-L in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease of less than five years duration has commenced enrollment at multiple sites across the United States.
The United States Food and Drug Administration (FDA) has previously confirmed alignment with Mesoblast on the design of the 300-patient randomized, placebo-controlled trial and the 12-month primary endpoint of pain reduction as an approvable indication. Key secondary measures include improvement in quality of life, function, and reduced opioid usage.
FDA has designated rexlemestrocel-L a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of chronic low back pain. RMAT designation provides all the benefits of Breakthrough and Fast Track designations, including rolling review and eligibility for priority review on filing of a Biologics License Application (BLA).
“This therapy has the potential to be groundbreaking and life changing for the low back pain population,” said Dr. Alan Miller, MD, trial investigator at Coastal Health Specialty Care in Jacksonville, Florida.
Mesoblast Chief Medical Officer Dr. Eric Rose said “We are very excited to be actively enrolling our pivotal trial of rexlemestrocel-L across multiple sites and look forward to confirming the durable pain reduction previously observed in the first Phase 3 trial. There is a significant need for a safe, effective, and durable treatment in patients with CLBP and degenerative disc disease, in particular one that reduces or eliminates opioid use.”
About Chronic Low Back Pain
Back pain is the leading cause of disability in Americans under 45 years,1 with an annual prevalence in the general US adult population of 10
About Mesoblast
Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of late-stage product candidates which respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.
Mesoblast has a strong and extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets. The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.
Mesoblast is developing product candidates for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid refractory acute graft versus host disease, and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for advanced chronic heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by Mesoblast’s licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.
Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast
References / Footnotes
- American Academy of Pain Medicine - Get the Facts on Pain. The American Academy of Pain Medicine. http://www.painmed.org/patientcenter/facts-on-pain/ Accessed on June 28, 2017.
- Urits I, Burshtein A, Sharma M, et al. Low Back Pain, a Comprehensive Review: Pathophysiology, Diagnosis, and Treatment. Current Pain and Headache Reports. 2019;23(3):1-10. doi:10.1007/s11916-019-0757-1.
- Navigant: Commercial Assessment for a Proprietary Cell-Based Therapy for DDD in the U.S. and the EU3 – August 2014.
- Decision Resources: Chronic Pain December 2015.
Forward-Looking Statements
This press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals (including any future decision that the FDA may make on the BLA for remestemcel-L for pediatric patients with SR-aGVHD), manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.
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For more information, please contact:
| Corporate Communications / Investors | Media |
| Paul Hughes | BlueDot Media |
| T: +61 3 9639 6036 | Steve Dabkowski |
| E: investors@mesoblast.com | T: +61 419 880 486 |
| E: steve@bluedot.net.au |
FAQ
What is the purpose of Mesoblast's Phase 3 trial for rexlemestrocel-L (MESO)?
How many patients are enrolled in Mesoblast's Phase 3 trial for chronic low back pain (MESO)?
What is the significance of the RMAT designation for Mesoblast's rexlemestrocel-L (MESO)?
What are the secondary measures in Mesoblast's Phase 3 trial for chronic low back pain (MESO)?
