Australian-based regenerative medicine company Mesoblast has expanded access to its compassionate use programme in the US of its investigational candidate to include the treatment of children infected with COVID-19.
The drug, an allogeneic mesenchymal stem cell (MSC) product candidate dubbed remestemcel-L, will now be available for children between the ages of two months and 15 years old infected with COVID-19 who experience cardiovascular and other complications of multisystem inflammatory syndrome, thanks to the new expanded access protocol.
Expanded access is a potential pathway for patients who have an immediate, life-threatening condition to access an investigational medical product for treatment outside clinical trials. Mesoblast filed the protocol with the US Food and Drug Administration (FDA) under its existing investigational new drug application, to allow clinicians to access remestemcel-L for an intermediate-size patient population.
Multisystem inflammatory syndrome is a potentially life-threatening complication of COVID-19 observed in otherwise healthy children and adolescents, and is characterised by massive simultaneous inflammation of multiple critical organs and their vasculature.
In around 50% of these cases, inflammation is associated with significant cardiovascular complications that directly involve the heart muscle and can lead to decreased cardiac function.
Remestemcel-L is comprised of culture-expanded mesenchymal stem cells taken from the bone marrow of an unrelated donor, and is administered in a series of intravenous infusions. It is believed that the therapy has immunomodulatory properties that could counteract the inflammatory processes seen in a number of diseases, including COVID-19.
The drug does this by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.
A host of candidates are being investigated to regulate the overactive inflammatory side effects associated with some cases of COVID-19, including Abivax’s experimental anti-inflammatory med ABX464.
Last week, Abivax initiated a phase 2b/3 study of ABX464, with the main goal of measuring the potential of the treatment to limit viral replication and decrease the severe inflammation that leads to acute respiratory distress syndrome (ARDS) seen in COVID019 patients.
Another anti-inflammatory drug – dexamethasone – also demonstrated impressive efficacy against COVID-19 in recent results from a large-scale study led by the University of Oxford. Dexamethasone is already approved to reduce inflammation in a number of diseases and conditions, including arthritis, asthma and certain skin conditions.
The news of the expanded access protocol caused Mesoblast shares to jump by as much as 18.1% yesterday, settling to a 9.1% gain by the close on Monday.