Silviu Itescu, Mesoblast CEO
Novartis offered up $25 million in cash last November for the rights to Mesoblast’s potential cell therapy for Covid-19. But after a Phase III flunk, the Big Pharma’s now getting cold feet — and Mesoblast’s stock is suffering big time.
Mesoblast shares $MESO tanked more than 17% on Tuesday morning after announcing that Novartis has walked out on its partnership for remestemcel-L. And because the termination occurred before closing, Mesoblast missed out on upfront payments of $25 million cash and a $25 million equity investment.
The reason? Subpar data, according to Novartis.
‘After assessing additional data, the totality of evidence from the Phase III trial is not sufficient to continue with the collaboration agreement. No safety concerns have been identified,’ the company said in a statement to Endpoints News.
Novartis was drawn to remestemcel-L after an open-label compassionate use program suggested an 83% survival rate in patients on ventilators who were treated with the experimental cell therapy last March. Then in December, Mesoblast cut a 300-person Phase III trial short after a data safety monitoring board concluded the therapy was ‘not likely’ to reach its primary endpoint, a 43% reduction in mortality at 30 days.
That October, the FDA issued a complete response letter for the same candidate in a different indication, pediatric acute graft-versus-host disease, over issues with trial design. Mesoblast had submitted its application on the basis of one single-arm, open-label study rather than a randomized trial, even though the drug demonstrated a statistically significant benefit in its primary endpoint against the historical control rate.
The company also suffered a miss for its second candidate, rexlemestrocel-L, in heart failure last December. While the experimental cell therapy did not significantly reduce hospitalizations over placebo in individuals with recurring heart failure, Mesoblast says the program managed to hit secondaries in mortality-rate reduction for earlier stages of the disease.
US investor group SurgCenter Development threw Mesoblast a lifeline in March, leading a $110 million private placement.
Despite the setbacks, Mesoblast still thinks there’s a path forward for remestemcel-L in acute respiratory distress syndrome due to Covid-19.
‘The observed mortality reduction with remestemcel-L in patients aged under 65 in the completed COVID ARDS trial, despite having missed the primary endpoint, is considered by Mesoblast to be a sufficiently strong signal to support pursuing an emergency use authorization (EUA), the most direct path to market,’ the company said in a statement.
Mesoblast was unavailable for comment as of press time, but Endpoints will update the story as it develops. The company said in a statement that it’s preparing to initiate another Phase III trial that may support an EUA in Covid-associated ARDS.
‘Variants including Omicron present a growing threat due to increased infectivity and immune evasion from vaccines and monoclonal antibodies, increasing the urgent need for therapeutics to prevent the likely high mortality of those progressing to ICU and ARDS,’ the company said.
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Sensor-based technology for clinical trial data collection represents the latest medical paradigm shift. There are more than 700 clinical studies involving wearable devices currently underway in the United States. A study from Intel IT projects their inclusion in clinical trials will surge to 70% by 2025.
Apps, biosensors and patient-centered technologies increase visibility of comprehensive patient data. Pharma leaders anticipate the benefits of wearables to include better data (58%), faster results (33%) and lower trial costs (10%).
When Bristol Myers Squibb celebrated the approval of ozanimod — branded Zeposia — in ulcerative colitis earlier this year, the company touted the first gastrointestinal indication for an S1P receptor modulator.
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Rob Califf, the famous cardiologist from Duke University, is likely to return to the top of the FDA, this time under the Biden administration.
At his confirmation hearing Tuesday, Democrats and Republicans on the Senate health committee offered their support for Califf, with Chair Patty Murray (D-WA) stressing the need for an experienced leader, like Califf, who can ensure that science comes first.
Aamir Malik, Pfizer chief business innovation officer
Pfizer made a big splash in the M&A space Monday, announcing a $6.7 billion buyout of Arena Pharmaceuticals to chase Bristol Myers Squibb in the S1P race. But company execs suggested the company isn’t finished bringing on new assets.
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A Boston-based provider of lab space is tripling its footprint with the addition of a West Coast campus.
SmartLabs, a company with labs in three different neighborhoods in the Boston area, will open a new research and manufacturing center that will be located in the heart of the South San Francisco biotech corridor. The site will support end-to-end drug development and include 500L manufacturing bioreactors that can support allogeneic and autologous cell therapies.
As the investments in cell and gene therapy manufacturing continue to grow across the world, Cincinnati Children’s Hospital and CTI Clinical Trial & Consulting Services have entered a $100 million agreement to produce clinical material locally.
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Tillman Gerngross, Adagio CEO
Since the pandemic began in the spring of 2020, Tillman Gerngross has raised more than half a billion dollars for his startup Adagio and their promise to develop a so-called broadly neutralizing coronavirus antibody that could snare any variant.
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Harpreet Singh, Immatics CEO (Credit: Allogeneic Cell Therapies Summit)
Just a few weeks after offering a positive readout on its first early clinical-stage offering, the transatlantic biotech Immatics is back with news that the research crowd around Rupert Vessey at Bristol Myers Squibb has anted up $150 million in cash to get on at the ground floor with one of their still-preclinical efforts.
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https://endpts.com/mesoblast-shares-tank-after-novartis-abandons-a-partnership-for-the-biotechs-potential-covid-19-treatment/