Appendix 4C Quarterly Activity Report for Quarter Ended September 30, 2024
Mesoblast (MESO) has filed a Biologics License Application with the FDA for Ryoncil® in treating steroid-refractory acute graft versus host disease, with a decision expected by January 7, 2025. The company secured a strategic financing agreement for up to US$50.0 million in convertible notes upon FDA approval. First quarter FY2025 showed strong financial management with net operating spend of US$10.5 million, down 26% year-over-year. Cash balance stands at US$51.1 million, with additional US$60.0 million available from existing facilities upon Ryoncil approval.
Mesoblast (MESO) ha presentato una domanda di licenza biologica alla FDA per Ryoncil® nel trattamento della malattia del grasso acuto da trapianto contro ospite refrattaria ai corticosteroidi, con una decisione attesa entro il 7 gennaio 2025. L'azienda ha ottenuto un accordo di finanziamento strategico fino a 50,0 milioni di dollari USA in note convertibili, soggetto all'approvazione della FDA. Il primo trimestre dell'anno fiscale 2025 ha mostrato una forte gestione finanziaria con una spesa operativa netta di 10,5 milioni di dollari USA, in calo del 26% rispetto all'anno precedente. Il saldo di cassa ammonta a 51,1 milioni di dollari USA, con ulteriori 60,0 milioni di dollari USA disponibili da strutture esistenti all'approvazione di Ryoncil.
Mesoblast (MESO) ha presentado una Solicitud de Licencia Biológica a la FDA para Ryoncil® en el tratamiento de la enfermedad injerto contra huésped aguda refractaria a esteroides, con una decisión esperada para el 7 de enero de 2025. La empresa aseguró un acuerdo de financiamiento estratégico por hasta 50,0 millones de dólares EE.UU. en notas convertibles, sujeto a la aprobación de la FDA. El primer trimestre del año fiscal 2025 mostró una sólida gestión financiera con un gasto operativo neto de 10,5 millones de dólares EE.UU., una disminución del 26% interanual. El saldo de efectivo se sitúa en 51,1 millones de dólares EE.UU., con otros 60,0 millones de dólares EE.UU. disponibles de instalaciones existentes tras la aprobación de Ryoncil.
Mesoblast (MESO)는 스테로이드에 반응하지 않는 급성 이식편대숙주질환 치료를 위한 Ryoncil®의 생물학적 제품 허가 신청서를 FDA에 제출했으며, 결정은 2025년 1월 7일까지 예상됩니다. 회사는 FDA 승인을 조건으로 최대 5천만 달러의 전환사채에 대한 전략적 자금조달 계약을 체결했습니다. 2025 회계연도 1분기 동안 순 운영 비용이 1천50만 달러로 전년 대비 26% 감소하며 강력한 재무 관리가 이루어졌습니다. 현금 잔고는 5천11만 달러이며, Ryoncil 승인을 받으면 기존 시설로부터 추가로 6천만 달러를 사용할 수 있습니다.
Mesoblast (MESO) a soumis une Demande de Licence Biologique à la FDA pour Ryoncil® dans le traitement de la maladie du greffon contre l'hôte aigu résistante aux stéroïdes, avec une décision attendue pour le 7 janvier 2025. L'entreprise a sécurisé un accord de financement stratégique pouvant aller jusqu'à 50,0 millions de dollars en billets convertibles sous réserve de l'approbation de la FDA. Le premier trimestre de l'exercice 2025 a montré une solide gestion financière avec des dépenses d'exploitation nettes de 10,5 millions de dollars, en baisse de 26 % par rapport à l'année précédente. Le solde de liquidités est de 51,1 millions de dollars, avec 60,0 millions de dollars supplémentaires disponibles grâce à des installations existantes après l'approbation de Ryoncil.
Mesoblast (MESO) hat einen Antrag auf Biologischen Lizenz bei der FDA für Ryoncil® zur Behandlung der steroidgegenständlichen akuten Transplantat-gegen-Wirt-Krankheit eingereicht, mit einer Entscheidung, die bis zum 7. Januar 2025 erwartet wird. Das Unternehmen sicherte sich eine strategische Finanzierungsvereinbarung über bis zu 50,0 Millionen USD in wandelbaren Anleihen, vorbehaltlich der Genehmigung durch die FDA. Im ersten Quartal des Geschäftsjahres 2025 zeigte die finanzielle Verwaltung eine starke Leistung mit einem Nettobetriebsausgaben von 10,5 Millionen USD, was einem Rückgang von 26 % im Vergleich zum Vorjahr entspricht. Der Bargeldbestand beläuft sich auf 51,1 Millionen USD, wobei zusätzlich 60,0 Millionen USD aus bestehenden Einrichtungen nach Genehmigung von Ryoncil verfügbar sind.
- 26% reduction in net operating cash spend vs previous year
- Secured up to US$50.0 million convertible note agreement for Ryoncil launch
- Strong cash position of US$51.1 million with additional US$60.0 million available
- FDA Pre-License Inspection completed without Form 483 issues
- Received Rare Pediatric Disease and Orphan-Drug Designations for Revascor
- Executive Directors took 30% salary reduction
- Non-Executive Directors deferred 50% of cash payments
- Company requires FDA approval to access additional financing
Insights
The quarterly report reveals significant financial and operational developments. Key financial metrics show a cash balance of
The strategic financing arrangement through convertible notes worth up to
Management's voluntary compensation adjustments, including
The BLA resubmission for RYONCIL represents a significant milestone, targeting an unmet medical need in pediatric SR-aGvHD with a PDUFA date of January 7, 2025. The successful Pre-License Inspection without Form 483 issues strengthens the probability of approval.
The company's pipeline shows promising developments, particularly with REVASCOR receiving both Rare Pediatric Disease and Orphan-Drug designations for HLHS treatment. Clinical data showing significant improvements in left ventricular volumes supports its potential efficacy. The accelerated approval pathway for REVASCOR in end-stage heart failure patients with LVADs further diversifies potential revenue streams.
The advancement of the confirmatory Phase 3 trial for chronic low back pain, with FDA-agreed endpoints and RMAT designation, positions the company for potential expansion in the pain management market, particularly targeting opioid reduction.
NEW YORK, Oct. 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 first quarter ended September 30, 2024.
Mesoblast Chief Executive Silviu Itescu said: “We have had an extremely busy and productive quarter starting right from the outset with the filing of our Biologics License Application (BLA) with the United States Food and Drug Administration (FDA) for approval of Ryoncil® (remestemcel-L) in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGvHD). We continue to be engaged in active and ongoing interactions with the agency as part of the review process. We are anticipating a decision prior to or on the FDA’s Prescription Drug User Fee Act (PDUFA) goal date of January 7, 2025.”
“During the period we put in place a strategic financing to ensure that the Company is well capitalized for a commercial launch of RYONCIL. This has been structured as a convertible note subscription agreement with our largest shareholder for issue, at Mesoblast’s sole discretion, up to US
“I look forward to providing an update at our Annual General Meeting (AGM) on November 15th 12.00 noon AEDT (November 14th 8.00pm EST).”
KEY HIGHLIGHTS
Ryoncil® (Remestemcel-L) for Steroid-Refractory Acute Graft Versus Host Disease – Potential FDA Approval
- There are no approved treatments for children under 12 with steroid-refractory acute GvHD, making approval of a safe and effective treatment for this vulnerable population the most urgent need.
- Mesoblast resubmitted its BLA to FDA for approval of RYONCIL on July 8, 2024 and 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.
- Inventory has been manufactured and there is an established supply chain to ensure cryopreserved product is available for delivery to meet the needs of each site immediately post approval, with ability to scale up as necessary going forward.
- We have been working diligently to lay the groundwork for a successful launch of RYONCIL, including hiring select senior positions to implement a targeted commercial strategy since
50% of pediatric transplants are performed at just 15 centers. - Post approval implementation will initially target those centers with greatest experience using the RYONCIL product and highest volume, with staged rollout beyond.
Revascor® (Rexlemestrocel-L) for Pediatric Congenital Heart Disease - Hypoplastic Left Heart Syndrome
- Earlier this year, FDA granted Mesoblast’s second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell product REVASCOR both Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) for treatment of children with hypoplastic left heart syndrome (HLHS), a potentially life-threatening congenital heart condition.
- Results from a blinded, randomized, placebo-controlled prospective trial of REVASCOR conducted in the United States in children with HLHS were published in the December 2023 issue of the peer reviewed The Journal of Thoracic and Cardiovascular Surgery Open (JTCVS Open).1
- A single intramyocardial administration of REVASCOR at the time of staged surgery resulted in the desired outcome of significantly larger increases in left ventricular (LV) end-systolic and end-diastolic volumes over 12 months compared with controls as measured by 3D echocardiography (p=0.009 & p=0.020 respectively).
- These changes are indicative of clinically important growth of the small left ventricle, facilitating the ability to have a successful surgical correction, known as full biventricular (BiV) conversion, which allows for a normal two ventricle circulation.
- Without full BiV conversion the right heart chamber is under excessive strain with increased risk of heart failure, liver cirrhosis, and death.
- RPDD demonstrates that the disease is serious or life-threatening and the manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents, and that the disease is a rare disease or condition.
- On FDA approval of a BLA for REVASCOR for the treatment of HLHS, Mesoblast may be eligible to receive a Priority Review Voucher (PRV) that can be redeemed for any subsequent marketing application or may be sold or transferred to a third party.
- Mesoblast plans to meet with FDA to discuss the clinical data to support regulatory approval for REVASCOR in children with this life-threatening condition.
REVASCOR for Chronic Heart Failure with Reduced Ejection Fraction (HFrEF) and Persistent Inflammation
- In March FDA informed Mesoblast that it supports an accelerated approval pathway for its second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell product REVASCOR, for patients with end-stage ischemic HFrEF kept alive with a left ventricular assist device (LVAD).
- This followed presentation to FDA of the results of two complementary randomized controlled trials of REVASCOR, one in patients with end-stage HFrEF and LVADs and a second in advanced NYHA class II/III HFrEF patients.
- 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, after the meeting on HLHS, to discuss data presentation, timing and FDA expectations for an accelerated approval filing in these patients.
Rexlemestrocel-L for Chronic Low Back Pain associated with Degenerative Disc Disease – Phase 3 Program
- The confirmatory Phase 3 trial of Mesoblast’s second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell product rexlemestrocel-L in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration has commenced enrollment at multiple sites across the United States.
- FDA has previously agreed on the design of this 300-patient randomized, placebo-controlled confirmatory Phase 3 trial, and the 12-month primary endpoint of pain reduction as an approvable indication.
- This endpoint was successfully met in Mesoblast’s first Phase 3 trial.
- Key secondary measures include improvement in quality of life and function.
- A particular focus is on treatment of patients on opioids, since discogenic back pain accounts for approximately
50% of prescription opioid usage in the US. - Significant pain reduction and opioid cessation were observed in Mesoblast’s first Phase 3 trial.
- 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 BLA.
FINANCIAL REPORT
We have continued our disciplined financial management strategy focused on ensuring that we are well-positioned to execute our clinical, manufacturing, and commercialization plans while maintaining conservative fiscal practices. The successful implementation of our cost containment plan over the past 12 months 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.
To ensure that the Company is well capitalized for a commercial launch of RYONCIL, we recently entered into a convertible note subscription agreement with our largest shareholder for issue, at Mesoblast’s sole discretion, up to US
First Quarter Results
- Cash balance at September 30, 2024 is US
$51.1 million , with additional US$60.0 million available from existing financing facilities on RYONCIL approval. - Net operating cash spend of US
$10.5 million for the first quarter FY2025.26% (US$3.7 million ) reduction in net operating cash spend for the first quarter FY2025 versus the prior comparative quarter in FY2024.
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 first 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 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
- Wittenberg RE et al. Prospective randomized controlled trial of the safety and feasibility of a novel mesenchymal precursor cell therapy in hypoplastic left heart syndrome, JTCVS Open Volume 16, Dec 2023.
- 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 (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.
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 | |
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