CellProthera has reported a positive pre-investigational new drug (IND) meeting with the US Food and Drug Administration (FDA), as a pivotal Phase III trial for the company’s cell therapy for myocardial infarction inches closer.
France-headquartered CellProthera said the US agency aligned with plans for the trial design, which will include a two-year follow-up. The trial will evaluate the therapy’s ability to prevent subsequent heart diseases.
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In a press conference in London, UK, the company’s CEO Matthieu de Kalbermatten said that the open-label, randomised trial will enrol around 300 patients or more and will contain a composite primary effectiveness endpoint.
De Kalbermatten disclosed to Medical Device Network that the company will pursue Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, though put no timeline on it.
The RMAT designation programme is intended to expedite the development and review of regenerative therapies to treat, modify, reverse, or cure a serious condition. The RMAT tag also permits the use of surrogate endpoints in clinical trials studying the therapy, which can lead to accelerated approval.
CellProthera has already conducted a Phase II trial of its one-time cell therapy product – called ProtheraCytes – with a six-month follow-up.
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By GlobalDataProtheraCytes is derived from CD34+ cells harvested from the peripheral blood of patients. CD34+ cells are a type of stem cell that is adept at stimulating new blood vessel formation.
CellProthera has developed technology that allows larger quantities of cells to be implanted back into the patient. The company also uses an intramyocardial injection route, which facilitates more successful cell grafting. De Kalbermatten said CellProthera’s platform can inject 50 million cells directly into the heart tissue, more than other approaches such as intracoronary catheters.
An average of 800,000 people in the US have a heart attack every year. Whilst the majority respond well to current treatments, a minority – around 5%-10% who experience a severe infarction – do not recover cardiovascular health and enter long-term heart failure. This patient group would either need to take regular pharmacological interventions or receive a device implant, with a last resort being a heart transplant.
De Kalbermatten explained to Medical Device Network that the biotech is targeting this population with its autologous product that regenerates tissue and revascularises the heart. Whilst the cell therapy will only be suitable for a subset of myocardial infarction patients, the market opportunity is still relatively large, comprising around 50,000-100,000 patients every year. De Kalbermatten added that “this is not an orphan disease”.
As part of the meeting with the FDA, the agency reviewed clinical data from the Phase II EXCELLENT study completed this year. The trial demonstrated that, in combination with standard-of-care, ProtheraCytes improved left ventricular (LV) volumes and viability of myocardial segments at six months following treatment. The company said the therapy showed “strong and promising [efficacy] signals that will be confirmed through long-term follow-up”.
Whilst cell and gene therapies present a unique way of treating diseases, scalability hurdles remain. Because these types of therapies are developed for each patient, manufacturing costs can be high, leading to costly list prices of products.
