Appendix 4C Quarterly Activity Report for Quarter Ended June 30, 2024
Mesoblast (Nasdaq:MESO; ASX:MSB) provided highlights of its activities for Q4 2024 ended June 30. Key points include:
1. Ryoncil BLA resubmission for SR-aGVHD treatment in children accepted by FDA, with a decision expected by January 7, 2025.
2. Phase 3 trial for rexlemestrocel-L in chronic low back pain has commenced enrollment.
3. FDA supports accelerated approval pathway for Revascor in end-stage heart failure patients with LVADs.
4. Cash balance of US$63.0 million as of June 30, 2024, with an additional US$10.0 million available upon Ryoncil approval.
5. 37% reduction in net operating cash spend for Q4 FY2024 compared to Q4 FY2023.
6. Successful implementation of cost containment plan, including voluntary salary reductions for directors.
Mesoblast (Nasdaq:MESO; ASX:MSB) ha fornito i punti salienti delle sue attività per il Q4 2024 conclusosi il 30 giugno. I punti chiave includono:
1. Ri-sottomissione del BLA di Ryoncil per il trattamento della SR-aGVHD nei bambini accettata dalla FDA, con una decisione prevista entro il 7 gennaio 2025.
2. È iniziato il reclutamento per il trial di Fase 3 per rexlemestrocel-L nel trattamento del dolore lombare cronico.
3. La FDA sostiene la procedura di approvazione accelerata per Revascor nei pazienti con insufficienza cardiaca terminale dotati di LVAD.
4. Saldo di cassa di 63 milioni di dollari statunitensi al 30 giugno 2024, con ulteriori 10 milioni di dollari disponibili in caso di approvazione di Ryoncil.
5. Riduzione del 37% nella spesa netta operativa di cassa per il Q4 FY2024 rispetto al Q4 FY2023.
6. Implementazione riuscita del piano di contenimento dei costi, inclusi tagli salariali volontari per i direttori.
Mesoblast (Nasdaq:MESO; ASX:MSB) proporcionó los aspectos más destacados de sus actividades para el Q4 2024 finalizado el 30 de junio. Los puntos clave incluyen:
1. Reenvío del BLA de Ryoncil para el tratamiento de SR-aGVHD en niños aceptado por la FDA, con una decisión esperada para el 7 de enero de 2025.
2. Se ha iniciado el reclutamiento para el ensayo de Fase 3 para rexlemestrocel-L en el dolor lumbar crónico.
3. La FDA apoya la ruta de aprobación acelerada para Revascor en pacientes con insuficiencia cardíaca en etapa terminal con LVADs.
4. Saldo de efectivo de 63 millones de dólares estadounidenses a partir del 30 de junio de 2024, con otros 10 millones de dólares disponibles tras la aprobación de Ryoncil.
5. Reducción del 37% en el gasto operativo neto en efectivo para el Q4 FY2024 en comparación con el Q4 FY2023.
6. Implementación exitosa del plan de contención de costos, incluidos recortes salariales voluntarios para los directores.
Mesoblast (Nasdaq:MESO; ASX:MSB)는 2024년 4분기 활동의 주요 내용을 6월 30일 기준으로 제공했습니다. 주요 내용은 다음과 같습니다:
1. Ryoncil의 BLA 재제출이 아동의 SR-aGVHD 치료를 위해 FDA에 의해 수용되었으며, 결정은 2025년 1월 7일로 예상됩니다.
2. 만성 요통에 대한 rexlemestrocel-L의 3상 시험이 등록을 시작했습니다.
3. FDA는 LVAD가 장착된 말기 심부전 환자에 대한 Revascor의 가속 승인 경로를 지원합니다.
4. 2024년 6월 30일 기준으로 현금 잔고는 6,300만 달러이며, Ryoncil 승인 시 1,000만 달러가 추가로 제공됩니다.
5. FY2024 4분기 동안 FY2023 4분기 대비 37%의 순 운영 현금 지출 감소가 있었습니다.
6. 이사들을 위한 자발적인 급여 삭감을 포함한 비용 절감 계획의 성공적인 시행.
Mesoblast (Nasdaq:MESO; ASX:MSB) a fourni un aperçu de ses activités pour le Q4 2024, qui s'est terminé le 30 juin. Les points clés incluent :
1. Réexamen du BLA de Ryoncil pour le traitement de la SR-aGVHD chez les enfants accepté par la FDA, avec une décision attendue d'ici le 7 janvier 2025.
2. Le procès de Phase 3 pour rexlemestrocel-L dans le traitement des douleurs lombaires chroniques a commencé le recrutement.
3. La FDA soutient la voie d'approbation accélérée pour Revascor chez les patients en insuffisance cardiaque terminale équipés de LVAD.
4. Solde de trésorerie de 63 millions de dollars américains au 30 juin 2024, avec un montant supplémentaire de 10 millions de dollars disponible après l'approbation de Ryoncil.
5. Réduction de 37% des dépenses nettes d'exploitation de trésorerie pour le Q4 FY2024 par rapport au Q4 FY2023.
6. Mise en œuvre réussie du plan de maîtrise des coûts, y compris des réductions salariales volontaires pour les directeurs.
Mesoblast (Nasdaq:MESO; ASX:MSB) hat die Höhepunkte seiner Aktivitäten für das Q4 2024, das am 30. Juni endete, bereitgestellt. Die Hauptpunkte sind:
1. Wiedereinreichung des BLA von Ryoncil für die Behandlung von SR-aGVHD bei Kindern wurde von der FDA akzeptiert, eine Entscheidung wird bis zum 7. Januar 2025 erwartet.
2. Die Phase-3-Studie für rexlemestrocel-L bei chronischen Rückenschmerzen hat mit der Rekrutierung begonnen.
3. Die FDA unterstützt den beschleunigten Genehmigungsweg für Revascor bei Patienten mit terminaler Herzinsuffizienz mit LVADs.
4. Bargeldbestand von 63 Millionen US-Dollar zum 30. Juni 2024, mit weiteren 10 Millionen US-Dollar, die im Falle der Genehmigung von Ryoncil verfügbar sind.
5. 37% Rückgang der Nettooperationen-Ausgaben für das Q4 FY2024 im Vergleich zum Q4 FY2023.
6. Erfolgreiche Umsetzung des Kostenkontrollplans, einschließlich freiwilliger Gehaltskürzungen für Direktoren.
- Ryoncil BLA resubmission accepted by FDA for review, with potential approval by January 7, 2025
- Phase 3 trial for rexlemestrocel-L in chronic low back pain has started enrollment
- FDA supports accelerated approval pathway for Revascor in end-stage heart failure patients with LVADs
- 37% reduction in net operating cash spend for Q4 FY2024 compared to Q4 FY2023
- 23% reduction in net operating cash spend for FY2024 compared to FY2023
- Successful implementation of cost containment plan, reducing expenses
- Net operating cash spend of US$10.2 million for Q4 FY2024, indicating ongoing cash burn
Mesoblast's Q4 FY2024 report reveals significant progress in its product pipeline, particularly for Ryoncil and Revascor. The company's cash balance of
The potential FDA approval of Ryoncil for SR-aGVHD in children by January 7, 2025, could be a significant catalyst for Mesoblast's revenue growth. The company's strategic approach to commercial launch preparation, balancing prudent cash management with progress on key programs, is commendable.
However, investors should note that Mesoblast is still in a pre-revenue stage for its lead candidates. The success of Ryoncil's launch and the progress of other pipeline products will be important for the company's long-term financial health. The reduction in executive compensation and deferral of director fees indicate a commitment to cost containment, which is positive for cash preservation in this critical phase.
Mesoblast's progress with Ryoncil (remestemcel-L) for SR-aGVHD in children is promising. The FDA's acceptance of the BLA resubmission as a complete response and the Priority Review designation underscore the potential significance of this treatment. The anticipated decision by January 7, 2025, could be a pivotal moment for both patients and the company.
The advancement of rexlemestrocel-L (Revascor) for chronic heart failure and chronic low back pain demonstrates Mesoblast's diversified approach to cellular medicines. The FDA's support for an accelerated approval pathway for Revascor in end-stage heart failure patients with LVADs is particularly noteworthy, as it could expedite the product's path to market.
The RMAT designations for both rexlemestrocel-L indications highlight the FDA's recognition of these therapies' potential to address unmet medical needs. However, it's important to remember that regulatory success doesn't guarantee commercial success. The company will need to navigate the challenges of market adoption and reimbursement for these novel cellular therapies.
Mesoblast's focus on inflammatory diseases positions it in a growing market segment. The potential approval of Ryoncil for SR-aGVHD in children addresses a critical unmet need, which could lead to rapid adoption if approved. However, the company will face challenges in educating healthcare providers and securing favorable reimbursement terms for this novel therapy.
The chronic low back pain market, targeted by rexlemestrocel-L, represents a substantial opportunity given the high prevalence and effective treatments available. The FDA's alignment on the trial design and primary endpoint of pain reduction is encouraging for potential future approval.
In the heart failure space, Revascor's potential to reduce inflammation and improve outcomes in end-stage patients could differentiate it in a competitive market. The reduction in major adverse cardiac events (MACE) observed in the Phase 3 trial for ischemic HFrEF patients is particularly promising.
Mesoblast's ability to execute its go-to-market strategy for Ryoncil and advance its pipeline will be critical for investor confidence. The company's cost containment efforts and strategic focus on key programs demonstrate a pragmatic approach to resource allocation, which is essential given the capital-intensive nature of bringing cellular therapies to market.
Ryoncil BLA Submission Under FDA Review for Approval in Children with SR-aGVHD
NEW YORK, July 30, 2024 (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 fourth quarter ended June 30, 2024.
Mesoblast Chief Executive Silviu Itescu said: “We are very pleased with the strong relationship we have built with FDA across our product pipeline and the positive outcomes over the past six months.”
“Our BLA resubmission for approval of Ryoncil® (remestemcel-L) in the treatment of children with acute graft versus host disease (SR-aGVHD) was accepted as a complete response, we received feedback from FDA on the potential accelerated approval pathway for Revascor® (rexlemestrocel-L) in end-stage heart failure patients, and our confirmatory Phase 3 trial in inflammatory back pain is actively enrolling with a primary endpoint of pain reduction aligned with FDA.”
“We are executing on our go-to-market plan to bring Ryoncil to the many children suffering with the devastating disease of acute GVHD. I look forward to an activity update at our full year financials and investor call on August 28th 6.30pm EDT (August 29th 8.30am AEST).”
KEY HIGHLIGHTS
Graft versus Host Disease – Pending Decision on FDA Approval
- FDA informed Mesoblast at the end of March 2024 that, following additional consideration, the available clinical data from the Phase 3 study MSB-GVHD001 appears sufficient to support submission of the proposed BLA for remestemcel-L (RYONCIL) for treatment of pediatric patients with SR-aGVHD.
- Mesoblast resubmitted its BLA for approval of RYONCIL on July 8, 2024, addressing remaining CMC (Chemistry, Manufacturing, and Controls) items in the August 2023 Complete Response Letter (CRL).
- FDA accepted the BLA resubmission within two weeks, considering it to be a complete response.
- Mesoblast anticipates a decision prior to or on the FDA’s Prescription Drug User Fee Act (PDUFA) goal date of January 7, 2025.
- FDA has already conducted the Pre-License Inspection (PLI) of the manufacturing process for RYONCIL in May 2023 and this did not result in the issuance of any Form 483.
- RYONCIL is being reviewed under Priority Review, a designation given to drugs that treat a serious condition and provide a significant improvement in safety or effectiveness over existing treatments.
Chronic Inflammatory Low Back Pain – Phase 3 Program
- The confirmatory Phase 3 trial of Mesoblast’s second generation 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.
- 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).
Chronic Heart Failure with Reduced Ejection Fraction (HFrEF) and Persistent Inflammation
- FDA informed Mesoblast that it supports an accelerated approval pathway for its second generation allogeneic, immunoselected, and industrially manufactured stromal cell product rexlemestrocel-L (Revascor®), for patients with end-stage ischemic heart failure with reduced ejection fraction (HFrEF) and a left ventricular assist device (LVAD).
- In these patients, a single administration of REVASCOR reduced inflammation, strengthened left ventricular function, reduced right ventricular failure, and reduced hospitalizations.
- REVASCOR has also reduced major adverse cardiac events (MACE) (cardiovascular death, heart attacks and strokes) in a completed Phase 3 trial in ischemic HFrEF patients with NYHA class II /III disease and inflammation.
- Mesoblast has received RMAT designation for rexlemestrocel-L in the treatment of end-stage heart failure in LVAD patients and intends to meet with FDA to discuss data presentation, timing and FDA expectations for an accelerated approval filing in these patients.
FINANCIAL REPORT
We will take a measured approach to preparing for the commercial launch of RYONCIL for treatment of children with SR-aGVHD in anticipation of potential FDA BLA approval and ensure prudent cash management. The successful implementation of the cost containment plan from August 2023 and the re-prioritization of projects has enabled us to reduce cash expenditure whilst still making significant strides forward on key programs as outlined above. We continue to work on corporate and strategic initiatives to access commercial distribution channels and optimize our balance sheet.
Fourth Quarter and Full Year Results
- Cash balance at June 30, 2024 is US
$63.0 million , with additional US$10.0 million available from an existing facility on FDA approval of RYONCIL. - Net operating cash spend of US
$10.2 million for the fourth quarter FY2024. 37% (US$6.0 million ) reduction in net operating cash spend for the fourth quarter FY2024 versus the prior comparative quarter in FY2023.23% (US$14.8 million ) reduction in net operating cash spend in FY2024 compared to FY2023.
Cost containment strategy achieved
On completion of the FY2024 financial year we are pleased to report the results of the successful cost containment plan announced in August 2023 as follows:
Other
Fees to Non-Executive Directors were nil, consulting payments to Non-Executive Director were US
A copy of the Appendix 4C – Quarterly Cash Flow Report for the fourth quarter FY2024 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 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
- 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 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 |
| 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 | |
A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/ea78d212-3108-47c8-805d-fd80206fd7c4
FAQ
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