Mesoblast Explained

Mesoblast Limited is an Australian regenerative medicine company. It seeks to provide treatments for inflammatory ailments, cardiovascular disease, and back pain. The company is led by Silviu Itescu, who founded the company in 2004.^[1]

History

In Mesoblast's annual report from August 2010, the company first announced positive results of preclinical trials showing radiographic and pathologic disc regeneration with MPC-06-ID. Mesoblast said they were in the process of completing an Investigational New Drug (IND) submission to the United States FDA to commence Phase 2 clinical trials.^[2] In December 2010, Mesoblast entered into an agreement with US-based Cephalon to develop and commercialize novel adult Mesenchymal Precursor Stem Cell (MPC) therapeutics for degenerative conditions of the cardiovascular and central nervous systems.^[3]

In September 2011, Mesoblast entered into an agreement with Swiss-based Lonza Group. Under the agreement, Lonza will manufacture the stem cells for Mesoblast's MPC cell products. Mesoblast will also have exclusive access to Lonza's Cell Therapy facilities in Singapore for the manufacture of allogeneic cell therapy.^[4]

Osiris sold its MSC drug and patents to Mesoblast Ltd. in 2012.^[5] In March 2014, Circulation Journal published the results of a clinical trial using MPCs as adjunctive therapy for patients with a Ventricular Assist Device.^[6] In May 2014, Mesoblast announced it would receive incentives from the Singapore Economic Development Board (EDB) for activities in Singapore related to manufacturing operations, product development, and commercialization.^[7]

On February 10, 2015, Mesoblast was granted a patent by the United States Patent and Trademark Office (USPTO) covering its proprietary Mesenchymal Precursor Cell (MPC) technology for use in the treatment of degenerated intervertebral discs. In June 2015, Mesoblast received $5.8 million from the Australian Government for Research and Development (R&D) activities conducted during the 2014 financial year. The funds were provided to Mesoblast under the Government's R&D Tax Incentive Program.^[8]

In February 2016, Mesoblast's Licensee (JCR Pharmaceuticals Co., Ltd.) sold its first allogeneic cell product for the treatment of acute graft versus host disease (aGVHD) in children and adults in Japan. TEMCELL is the first allogeneic cell therapy to be fully approved in Japan.^[9] In December 2016, Mesoblast and Mallinckrodt Pharmaceuticals entered into an agreement to exclusively negotiate a commercial and development partnership for MPC-06-ID in the treatment of chronic low back pain due to disc degeneration.^[10]

Research

Mesenchymal lineage cells

Mesoblast products are largely derived from its proprietary mesenchymal lineage cells.^[11] This includes Mesenchymal Precursor Cells (MPCs) and Mesenchymal Stem Cells (MSCs).

Focus areas

• Back pain: Mesoblast's investigational product candidate MPC-06-ID is being developed.^[12]
• Rheumatoid Arthritis: On 24 March 2016 US patent 9,265,796 was granted for the use of Mesenchymal Precursor Cells (MPCs)for the treatment or prevention of rheumatic conditions, including rheumatoid arthritis, osteoarthritis, psoriatic arthritis, ankylosing spondylitis, sacroiliitis, enteric arthritis, and reactive arthritis.^{[13] [14]}
• Crohn's disease: A trial was announced to evaluate Prochymal in moderate to severe Crohn's Disease.^[15]
• Diabetic nephropathy: Mesoblast presented the results of a trial involving product candidate MPC-300-IV at the late-breaking scientific sessions of the 75th annual meeting of the American Diabetes Association.^[16]
• COVID-19 Acute Respiratory Distress Symptoms: In 2020, Ryoncil was tested on late-stage, ventilator-assisted COVID-19 patients with Acute Respiratory Distress Syndrome in the United States.^{[17] [18]}

Criticism

Mesoblast's solution for back pain came under heavy criticism from competitor Regenexx in August 2015. Dr. Chris Centeno said the trial results were "spit shined" with confusing and misleading language, press releases for phases 1 and 2 did not include images of MRIs to prove that discs were regenerated and expressed doubts about maintaining cell quality while mass-producing cells. He also said the host's immune system removes the injected stem cells.^[19]

Financial performance

Mesoblast is listed as MSB on the Australian Securities Exchange (ASX), as MESO on the NASDAQ, and as MEOBF on the ATC Markets.

The company reported its financial results in November 2020.^[20] As of September 30, 2020:

• Cash on hand was $108.1 million
• Net loss before tax $25.3 million
• Cash outflows for Q1 FY21 were $24.5 million.

Notes and References

1. Web site: BRW Rich 200 list 2014: 136. Silviu Itescu. BRW. 13 June 2015. 10 May 2015. https://web.archive.org/web/20150510152039/http://www.brw.com.au/p/lists/rich-200/2014/brw_rich_list_silviu_itescu_CMbcXpy9iWiDL37dc0XC0N. dead.
2. Web site: Mesoblast document . 27 September 2015 . 27 January 2014 . https://web.archive.org/web/20140127090623/http://ir.mesoblast.com/DocumentDownload.ashx?item=WH9llnmHhUKiM63h75M58w . dead .
3. Cephalon and Mesoblast Enter Into Strategic Alliance to Develop... -- FRAZER, Pa. and MELBOURNE, Australia, Dec. 7, 2010 /PRNewswire-FirstCall/ --. Mesoblast Limited . 7 December 2010.
4. Web site: Mesoblast and Lonza partner to mass-produce stem cells. www.labonline.com.au. 28 March 2024.
5. Waltz. Emily. December 1, 2013. Mesoblast acquires Osiris' stem cell business. Nature Biotechnology. en. 31. 12. 1061 . 10.1038/nbt1213-1061. 32036070. 1546-1696. free.
6. 10.1161/CIRCULATIONAHA.113.007412 . Ascheim . D.D. . Mesenchymal Precursor Cells as Adjunctive Therapy in Recipients of Contemporary LVADs . Circulation . 129. 22 . 1–52 . 2014 . 24682346 . 4243683 .
7. Mesoblast Accelerates Plans For Commercialization Manufacturing Operations In Singapore. Mesoblast. GlobeNewswire News Room. 28 May 2014.
8. http://ir.mesoblast.com/DownloadFile.axd?file=/Report/ComNews/20150605/01631222.pdf Report
9. http://investorsmedia.mesoblast.com/phoenix.zhtml?c=187006&p=irol-news&nyo=0 Mesoblast document
10. Web site: ASX Announcements | Mesoblast. 28 March 2024. 9 November 2018. https://web.archive.org/web/20181109140338/http://investorsmedia.mesoblast.com/phoenix.zhtml?c=187006&p=irol-asxnews&nyo=0. dead.
11. Web site: Ondek, Mesoblast and Sirtex investors roll the biotech dice | the Australian . 2014-11-07 . 2015-01-17 . https://web.archive.org/web/20150117055910/http://www.theaustralian.com.au/business/the-deal-magazine/ondek-mesoblast-and-sirtex-investors-roll-the-biotech-dice/story-e6frgabx-1227090406491?nk=3e172135863d3fb12cd9774780650fcb . dead .
12. Web site: News Announcements. Mesoblast. 15 February 2015. 15 February 2015. https://web.archive.org/web/20150215220325/http://www.mesoblast.com/news-and-media/news-announcements. dead.
13. Web site: KEY UNITED STATES PATENT GRANTED FOR CELL-BASED TREATMENT OF RHEUMATOID ARTHRITIS AND OTHER RHEUMATIC CONDITIONS . 24 Mar 2016 . ASX .
14. US. 9265796. Patent. Methods of treating or preventing rheumatic disease. 2014-09-18. 2012-07-04. 2011-07-04. Peter Ghosh. Silviu Itescu. MESOBLAST, INC..
15. Web site: Crohn's Disease. Mesoblast. Mesoblast. 8 November 2014. 8 November 2014. https://web.archive.org/web/20141108051033/http://www.mesoblast.com/products/immunologic-inflammatory/crohns-disease. dead.
16. http://ir.mesoblast.com/DownloadFile.axd?file=/Report/ComNews/20150609/01631584.pdf Report
17. News: Sheikh. Knvul. Thomas. Katie. May 8, 2020. More Coronavirus Vaccines and Treatments Move Toward Human Trials. en-US. The New York Times. 2020-08-13. 0362-4331.
18. Web site: Knapp. Alex. May 2, 2020. Clinical Trials Of Mesoblast's Stem Cell Treatment For COVID-19 Set To Begin Soon. 2020-08-13. Forbes. en.
19. Web site: Did the Mesoblast Stem Cell Disc Trial Succeed or Fail? -Regenexx®. 5 August 2015.
20. http://investorsmedia.mesoblast.com/static-files/dcb7d445-8492-4f68-b8fb-f0cbb3c87685 Financial Results for First Quarter Ended September 30, 2020