This March, Mesoblast once again submitted additional clinical trial data to the FDA. The FDA stated that Mesoblast’s Phase 3 therapy data is sufficient to support a resubmission of the Biologics License Application (BLA) for remestemcel-L treatment of pediatric patients with steroid-refractory acute graft-versus-host disease (SR-aGVHD). This announcement sent ripples through the entire stem cell industry, boosting morale across the board.

Mesoblast, an Australian-based company, has emerged as a dark horse in the booming stem-cell industry, boldly making its mark in the promising U.S. market. Over the past 19 years, Mesoblast has navigated every challenge a biotech company might face—experiencing both soaring highs and challenging lows, facing waves of praise as well as criticism, all while stumbling forward yet remaining steadfastly hopeful.

In December 2004, founder Silviu Itescu established Mesoblast and successfully led the company’s listing on the Australian Securities Exchange (ASX), raising A$20 million in funding.

At that time, Australia was not exactly fertile ground for the biotech industry. Yet Mesoblast emerged as a dark horse, boldly challenging what seemed to be the ceiling of Australia's biotechnology sector. As one observer put it, "Mesoblast has fundamentally disrupted the strategic thinking and mindset of everyone in Australia's life sciences field. The company has already secured FDA approval for at least four IND applications—something no other Australian firm has ever achieved before."

Mesoblast's decision to establish roots in Australia was driven by unavoidable challenges. At the time, the U.S. capital markets were largely dismissive of stem-cell therapies. The market had yet to fully recover, and venture capital firms still hadn’t recognized the true value of stem cells.

However, Mesoblast has been incredibly fortunate. In 2008, research on in vitro-induced pluripotent stem cells was ranked as the number one and number two major scientific breakthroughs by the world-renowned journals Nature and Science, respectively. Moreover, in 2007 and 2012, stem cell research earned the Nobel Prizes in Physiology or Medicine—first in Physiology and later in Medicine. As the popularity of stem cell therapies continues to soar, Mesoblast is now benefiting from this growing momentum.

The concept of off-the-shelf stem cell therapy, combined with Itescu's operational expertise, propelled Mesoblast's stock price steadily upward from 2004 to 2011. Although no products have yet been launched, the company has already delivered a 10-fold return for its founding shareholders.

During this period, both capital markets and major pharmaceutical companies are brimming with confidence in stem cells.

In 2013, Mesoblast acquired the Prochymal product from Osiris Therapeutics (Maryland, USA) for the treatment of acute graft-versus-host disease (aGVHD). Over the past decade-plus, Mesoblast has essentially been doubling down—agonizingly focused—on the Prochymal product, later rebranded as Remestemcel-L.

This product is an allogeneic mesenchymal stem cell therapy derived from bone marrow–derived mesenchymal stem cells that have been expanded through in vitro culture. It is intended for the treatment of aGVHD in pediatric patients aged 12 years and younger. GVHD carries a high mortality rate and typically occurs in transplant recipients whose tissue matches are only partially compatible. It often leads to severe damage affecting organs such as the skin, liver, and digestive tract. Currently, clinical treatment usually relies on hormone-based medications, but these therapies often show limited effectiveness.

Founded in 1992, Osiris specializes in bone marrow-derived mesenchymal stem cells and has become a leader in driving the direction of biotechnology and pharmaceutical innovation in the United States. The company name "Osiris" is inspired by the ancient Egyptian god of the underworld, who presided over the judgment of souls after death—determining whether one could achieve eternal life. This reflects the profound and transformative potential of stem cells to redefine the future of life itself. Osiris’s flagship product, Prochymal, has lived up to expectations, delivering remarkable results and positioning itself as a strong contender to claim global leadership. From its groundbreaking product concept and impressive clinical progress to its promising market outlook, Prochymal continues to inspire Mesoblast with limitless possibilities for the future.

In 2012, Prochymal successfully launched in Canada and New Zealand, administered via intravenous infusion for pediatric patients with GVHD. This drug became the world’s first approved allogeneic bone marrow-derived mesenchymal stem cell therapy—and also the first stem cell treatment ever approved specifically for the treatment of GVHD.

In February 2016, Prochymal received approval for marketing in Japan under the brand name Temcell, thanks to the joint efforts of Mesoblast and JCR Pharmaceutics. JCR secured exclusive sales rights for Prochymal in Japan. According to pricing set by Japan's pharmaceutical regulatory authority, cell companies receive a medical reimbursement of $7,079 per bag, while a full treatment course typically ranges from $110,000 to $170,000.

“Prochymal is sure to win!” With this unwavering belief, Mesoblast made its U.S. stock market debut on the Nasdaq, officially stepping onto the global stage. Yet, during this very period, U.S. biotech stocks were entering a bubble—marked by the failed IPO of cell therapy company BioCardia and the shattered dreams surrounding StemCells. It was against this backdrop that Mesoblast faced its truest challenge yet.

Mesoblast has poured the majority of its resources into its flagship product, Remestemcel-L, and submitted New Drug Applications to the FDA for clinical approval in both 2019 and 2023—but both applications were subsequently rejected by the FDA.

In May 2019, Mesoblast submitted its first application to the U.S. FDA. The submission included data from at least three clinical trials involving 458 participants, which demonstrated both high overall response rates and impressive patient survival outcomes. However, the FDA rejected the application, citing as a key reason that… The FDA has questioned Mesoblast's ability to consistently produce effective doses of Remestemcel-L.

In March 2023, Mesoblast submitted its second marketing application to the U.S. FDA. This time, the dossier included long-term survival data spanning at least four years, as well as results from high-risk patient populations. Five months later, the FDA once again rejected the marketing application for Remestemcel-L, emphasizing that Issues in efficacy assessment and studies involving adult populations.

It's still too early to tell whether Mesoblast can pull off a remarkable turnaround in the third quarter of this year, boosting confidence among industry players and investors. We believe the key to Mesoblast securing approval lies in… Was the potency assay and pharmaceutical issue—both repeatedly raised by the FDA—successfully resolved?

In 2012, Mesoblast’s founder, Silviu Itescu, told the media during an interview: "It will take about three years for our product to reach the market." Today, nearly four of those three-year periods have passed, yet Mesoblast still hasn’t seen the launch of its first commercialized product.

However, Mesoblast still deserves everyone's respect. Amidst the broader market downturn, the company has remained steadfast, forging its own path where none seemed to exist before.