Appendix 4C Quarterly Activity Report for Quarter Ended December 31, 2024
Mesoblast (MESO) reported key developments for Q2 FY2025 ended December 31, 2024. The company achieved a significant milestone with Ryoncil® becoming the first FDA-approved MSC therapy for steroid-refractory acute GvHD in children. A distribution network has been established with Cencora for efficient product delivery.
The company successfully completed a global private placement raising A$260 million (US$161 million). Net operating cash spend was US$10.1 million, reduced by 18% compared to the previous quarter. Cash on hand was US$38 million, with pro-forma cash after January's raise of approximately US$200 million.
The Phase 3 trial of rexlemestrocel-L for chronic low back pain is actively enrolling patients. Under RMAT designation, Mesoblast plans to discuss accelerated approval filing for end-stage heart failure patients with FDA.
Mesoblast (MESO) ha riportato sviluppi chiave per il secondo trimestre dell'anno fiscale 2025, terminato il 31 dicembre 2024. L'azienda ha raggiunto un traguardo significativo con Ryoncil®, diventando la prima terapia MSC approvata dalla FDA per la GvHD acuta refrattaria agli steroidi nei bambini. È stata stabilita una rete di distribuzione con Cencora per una consegna efficiente dei prodotti.
L'azienda ha completato con successo un collocamento privato globale, raccogliendo A$260 milioni (US$161 milioni). La spesa operativa netta è stata di 10,1 milioni di dollari, ridotta del 18% rispetto al trimestre precedente. La liquidità disponibile era di 38 milioni di dollari, con una liquidità pro-forma dopo la raccolta di gennaio di circa 200 milioni di dollari.
Lo studio di Fase 3 di rexlemestrocel-L per il dolore cronico lombare sta attivamente reclutando pazienti. Sotto la designazione RMAT, Mesoblast prevede di discutere il deposito per l'approvazione accelerata per i pazienti con insufficienza cardiaca in fase terminale con la FDA.
Mesoblast (MESO) reportó desarrollos clave para el segundo trimestre del año fiscal 2025, que finalizó el 31 de diciembre de 2024. La empresa logró un hito importante con Ryoncil®, convirtiéndose en la primera terapia MSC aprobada por la FDA para la GvHD aguda refractaria a esteroides en niños. Se ha establecido una red de distribución con Cencora para una entrega eficiente de productos.
La empresa completó con éxito una colocación privada global, recaudando A$260 millones (US$161 millones). El gasto operativo neto fue de 10,1 millones de dólares, reducido en un 18% en comparación con el trimestre anterior. El efectivo disponible era de 38 millones de dólares, con un efectivo pro forma después de la recaudación de enero de aproximadamente 200 millones de dólares.
El ensayo de Fase 3 de rexlemestrocel-L para el dolor lumbar crónico está reclutando activamente pacientes. Con la designación RMAT, Mesoblast planea discutir el archivo para la aprobación acelerada para pacientes con insuficiencia cardíaca en etapa terminal con la FDA.
Mesoblast (MESO)는 2025 회계연도 2분기(2024년 12월 31일 종료)에 대한 주요 개발 사항을 보고했습니다. 회사는 Ryoncil®이 어린이의 스테로이드 저항성 급성 GvHD에 대한 최초의 FDA 승인 MSC 치료제가 되는 중요한 이정표를 달성했습니다. Cencora와 함께 제품 전달을 위한 효율적인 유통망이 구축되었습니다.
회사는 A$260 million (US$161 million)의 자금을 마감하는 글로벌 사모 공모를 성공적으로 완료했습니다. 순 운영 현금 지출은 10.1백만 달러로, 전분기 대비 18% 감소했습니다. 보유 현금은 3,800만 달러이며, 1월 자금 조달 후의 pro forma 현금은 약 2억 달러입니다.
만성 요통을 위한 rexlemestrocel-L의 3상 시험이 활발히 환자를 모집하고 있습니다. RMAT 지정에 따라, Mesoblast는 FDA와 함께 말기 심부전 환자를 위한 가속 승인 신청에 대해 논의할 계획입니다.
Mesoblast (MESO) a rapporté des développements clés pour le deuxième trimestre de l'exercice 2025, qui s'est terminé le 31 décembre 2024. L'entreprise a atteint une étape significative avec Ryoncil®, devenant la première thérapie MSC approuvée par la FDA pour la GvHD aiguë réfractaire aux stéroïdes chez les enfants. Un réseau de distribution a été établi avec Cencora pour une livraison efficace des produits.
L'entreprise a réussi à réaliser une placement privé mondial, levant A$260 millions (US$161 millions). Les dépenses de fonctionnement nettes étaient de 10,1 millions de dollars, réduites de 18 % par rapport au trimestre précédent. La trésorerie disponible était de 38 millions de dollars, avec une trésorerie pro forma après la levée de fonds de janvier d'environ 200 millions de dollars.
Le procès de Phase 3 de rexlemestrocel-L pour la douleur lombaire chronique recrute activement des patients. Sous la désignation RMAT, Mesoblast prévoit de discuter du dépôt de demande d'approbation accélérée pour les patients en fin de vie avec la FDA.
Mesoblast (MESO) berichtete über wichtige Entwicklungen für das zweite Quartal des Geschäftsjahres 2025, das am 31. Dezember 2024 endete. Das Unternehmen erreichte einen bedeutenden Meilenstein, indem Ryoncil® die erste von der FDA genehmigte MSC-Therapie für steroid-refraktäre akute GvHD bei Kindern wurde. Ein Vertriebsnetzwerk wurde mit Cencora eingerichtet, um eine effiziente Produktlieferung zu gewährleisten.
Das Unternehmen hat erfolgreich eine globale Privatplatzierung abgeschlossen und dabei A$260 Millionen (US$161 Millionen) gesammelt. Der Netto-Betriebsausgabenbetrag betrug 10,1 Millionen US-Dollar und wurde im Vergleich zum vorherigen Quartal um 18% reduziert. Der verfügbare Bargeldbetrag belief sich auf 38 Millionen US-Dollar, mit einem pro-forma Bargeld nach der Kontaktaufnahme im Januar von etwa 200 Millionen US-Dollar.
Die Phase-3-Studie von rexlemestrocel-L bei chronischen Rückenschmerzen rekrutiert aktiv Patienten. Unter RMAT-Anerkennung plant Mesoblast, mit der FDA über die Einreichung eines beschleunigten Genehmigungsantrags für Patienten mit fortgeschrittener Herzinsuffizienz zu sprechen.
- FDA approval of Ryoncil® as first MSC therapy for SR-aGvHD treatment
- Successful completion of A$260M (US$161M) private placement
- 18% reduction in net operating cash spend vs previous quarter
- Commercial inventory manufactured and distribution network established
- Continued cash burn of US$10.1M in quarterly operating expenses
Insights
The Q2 FY2025 results mark a transformative period for Mesoblast, highlighted by three critical developments: FDA approval of Ryoncil®, successful completion of a
The financial position has been substantially strengthened, with pro-forma cash of approximately
Management's alignment with shareholder interests is evident through voluntary fee reductions:
- Non-Executive Directors:
50% fee reduction - Executive Directors:
30% salary reduction
The partnership with Cencora for distribution infrastructure represents a capital-efficient approach to commercialization, avoiding significant upfront infrastructure investments. The company's strategic focus on accelerating the Phase 3 trial for chronic low back pain and pursuing accelerated approval for heart failure applications indicates a well-structured pipeline development strategy backed by newly secured capital.
The FDA approval of Ryoncil® marks a watershed moment in cellular medicine, establishing Mesoblast as the pioneer in MSC therapy with regulatory validation. This approval for steroid-refractory acute GvHD creates a precedent for cellular therapies, potentially streamlining future MSC therapy approvals.
The clinical pipeline shows strategic diversification across high-value indications:
- Chronic low back pain trial expansion suggests confidence in the therapeutic approach
- Heart failure program strengthened by EJHF publication demonstrating survival benefits in high-risk patients
- RMAT designations for both REVASCOR in heart failure and HLHS indicate regulatory support for accelerated development
The publication in EJHF identifying responsive patient populations in heart failure represents important clinical validation, particularly notable for demonstrating improved survival in high-risk NYHA Class II/III patients with ischemic heart failure and inflammation. This precise patient stratification enhances the probability of success in future regulatory submissions.
NEW YORK, Jan. 30, 2025 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided highlights of its recent activities for the second quarter ended December 31, 2024.
ACTIVITY REPORT
- On December 18, 2024, Ryoncil® (remestemcel-L) became the first mesenchymal stromal cell (MSC) therapy approved by U.S. FDA for any indication.
- FDA approved Ryoncil® as the first and only therapy for children aged 2 months and older, including adolescents and teenagers, with steroid-refractory acute graft versus host disease (SR-aGvHD), a life-threatening condition with high mortality rates.
- Commercial inventory has been manufactured and a distribution network has been established using Cencora, a leader in specialty pharmaceutical services and distribution. Cencora will leverage its cryogenic logistics capabilities and state-of-the art cryogenic storage infrastructure to enable the efficient and secure delivery of cryopreserved product to U.S. treatment centers.
- The confirmatory Phase 3 trial of rexlemestrocel-L in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration is actively enrolling and treating patients at multiple sites across the United States; the capital raise concluded this month will facilitate expansion of sites enrolling in the trial and acceleration of patient accrual.
- Under its Regenerative Medicine Advanced Therapy (RMAT) designation Mesoblast intends to meet with FDA to discuss data presentation, timing and FDA expectations for an accelerated approval filing in end-stage heart failure patients.
- In November 2024 a publication in the prestigious peer-reviewed European Journal of Heart Failure (EJHF), reported that a single intramyocardial injection of Revascor® (rexlemestrocel-L), Mesoblast’s second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell therapy, results in improved survival in high-risk NYHA Class II/III patients with ischemic heart failure and inflammation.1 This identifies the HFrEF population that is responsive to REVASCOR and will be the target of a confirmatory trial after accelerated approval, if received.
- FDA granted REVASCOR RMAT designation following submission of results from the randomized controlled trial in children with hypoplastic left heart syndrome (HLHS), a potentially life-threatening congenital heart condition.
FINANCIAL REPORT
Mesoblast successfully completed a global private placement primarily to existing major US, UK, and Australian shareholders raising A
Results for Second Quarter ended December 31, 2024
- Net operating cash spend was US
$10.1 million . - Net operating cash spend for the quarter was reduced by
18% (US$2.2 million ) versus the prior comparative quarter in FY2024. - Cash on hand at the end of the quarter was US
$38 million (A$61 million ) with pro-forma cash after proceeds raised in January of approximately US$200 million (A$322 million ).
Other
Fees to Non-Executive Directors were US
A copy of the Appendix 4C – Quarterly Cash Flow Report for the second quarter FY2025 is available on the investor page of the company’s website www.mesoblast.com.
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 therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform 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’s RYONCIL® (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com.
Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. RYONCIL is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China.
About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications provide commercial protection extending through to at least 2041 in all major markets.
About Mesoblast manufacturing: 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 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
- Perin EC. Et al. Mesenchymal precursor cells reduce mortality and major morbidity in ischaemic heart failure with inflammation: DREAM-HF. Eur J Heart Fail 2024. https://doi.org/10.1002/ejhf.3522
- As required by ASX listing rule 4.7 and reported in Item 6 of the Appendix 4C, reported are the aggregated total payments to related parties being Executive Directors and Non-Executive Directors.
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, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGVHD and any other 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.
Release authorized by the Chief Executive.
For more information, please contact:
| Corporate Communications / Investors | Media – Global |
| Paul Hughes | Allison Worldwide |
| T: +61 3 9639 6036 | Emma Neal |
| E: investors@mesoblast.com | T: +1 603 545 4843 |
| E: emma.neal@allisonworldwide.com | |
| Media – Australia | |
| BlueDot Media | |
| Steve Dabkowski | |
| T: +61 419 880 486 | |
| E: steve@bluedot.net.au |
FAQ
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