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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 successf

Key Takeaway: Mesoblast Limited provided an update on its product candidates at the Bell Potter Emerging Leaders Conference. The company is preparing to resubmit its Biologics License Application (BLA) for Ryoncil, targeting treatment for steroid-refractory acute graft versus host disease, with potential FDA approval expected in the latter half of 2024. Additionally, Mesoblast is advancing its Phase 3 programs for heart failure and chronic low back pain, with feedback from the FDA indicating a possible accelerated approval process for its heart failure product. The company holds a strong intellectual property position in the US, with patents extending through 2043.
Price reaction · baseline $7.38 (2024-06-03 close) · hit after-hours · clean, no other MESO news in the window
day 0 close · peak
+2.7%

Market Sentiment Analysis

POSITIVE FACTORS

  • Expected resubmission of BLA for Ryoncil in Q2 2024.
  • Phase 3 programs for heart failure and back pain progressing.
  • Potential FDA accelerated approval pathway for heart failure product.
  • Strong intellectual property protection valid until 2043.

CONCERNS & RISKS

  • Future performance is uncertain, and actual results may vary.
  • Risk of FDA rejection for BLA resubmission.
  • Potential market withdrawal of products due to safety concerns.
  • Dependence on regulatory approvals could delay commercialization.

BiopharmaWatch Analysis

From our catalyst data and publicly available data · not financial advice
Best trade, last catalyst
+91%
120-day peak, hindsight
Typical move
31.6%
average across 4 past catalysts
Lead asset
Prochymal
Phase 2 · Pulmonary Disease, Chronic Obstructive

Full Press Release Details

{00876702} MESOBLAST CORPORATE PRESENTATION AT INVESTOR CONFERENCE Melbourne, Australia; June 3 and New York, USA; June 2, 2024: Mesoblast Limited (ASX:MSB; Nasdaq:MESO), Chief Executive Silviu Itescu provided a corporate update at the Bell Potter Emerging Leaders Conference. Dr. Itescu reiterated that the Company expects to file this quarter the Biologics License Application (BLA) resubmission with the United States Food and Drug Administration (FDA) for its lead product candidate Ryoncil (remestemcel-L) in the treatment of steroid-refractory acute graft versus host disease, with potential approval in the second half of CY2024. Dr. Itescu additionally highlighted the heart failure and back pain programs in Phase 3, and the recent feedback from FDA regarding an accelerated approval pathway for the heart failure product Revascor (rexlemestrocel). The presentation also highlighted Mesoblast's strong United States intellectual property position, with granted patents through 2032 covering mesenchymal stem cell compositions of matter, irrespective of source, and their broad uses, including for GVHD. On RYONCIL approval, the granted patents may be extended up to 2037. Additional patent filings may extend the commercial barrier to entry through 2043. 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 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: Exhibit 99.1
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

Frequently Asked Questions

What is the purpose of Mesoblast's BLA resubmission?

Mesoblast plans to resubmit the Biologics License Application for Ryoncil to treat steroid-refractory acute graft versus host disease.

What key products are highlighted in Mesoblast's update?

Mesoblast is focused on Ryoncil for GVHD and Revascor for heart failure and back pain.

How long is Mesoblast's intellectual property protection?

Mesoblast's intellectual property is protected through at least 2041, extending to 2043 with additional filings.

Where are Mesoblast's operational locations?

Mesoblast has operations in Australia, the United States, and Singapore.

What types of conditions do Mesoblast's therapies target?

Their therapies target severe inflammatory diseases, including GVHD and chronic heart failure.

Last updated: Jun 4, 2024