Cynata Therapeutics (ASX:CYP) has today seen its share price jump 8% in morning trade as the company announces its receipt of Investigational New Drug (IND) status attached to its Phase 2 trial approvals from the US medical regulator.
The IND approval awards Cynata’s product CYP-001 regulatory prioritisation within the FDA, ultimately meaning that if successful, the company will receive full approvals ahead of competitor products.
CYP-001 is a treatment designed to treat patients presenting with steroid-resistant aGvHD, a condition which affects recipients of bone marrow and stem cell transplants where the patient’s immune system rejects the new cells introduced into the body, a condition which is a leading cause of death in such recipients.
The approval of IND status for CYP-001 from the FDA comes ahead of the company’s Phase 2 Trials, which will be the first US trials using CYP-001.
The trial itself will involve some 50 patients with high risk aGvHD at clinical centers in a number of countries, including Australia alongside the US. Participants will be randomised to receive either CYP-001 or a placebo, in addition to corticosteroids.
The company is seeking to assess the efficacy of CYP-001 across 28 days by measuring Overall Response Rate to the treatment at the conclusion of the month-long study.
The trial is expected to commence in the second half of 2022, results will be published in early 2024.
Cynata remains active in conducting negotiations with study centres and still must win receipt of “relevant ethics [and] administrative approvals.”
With that said, company management is confident the study will go ahead as planned.
“A cleared IND represents a very significant achievement for any pharmaceutical or biotech company and underpins a major valuation catalyst,” Cynata CEO Dr. Ross Macdoanld commented.
“We…will continue to leverage the momentum in our negotiations with study centres with an expectation to commence a US trial in aGvHD by the end of the year.”
The US represents the largest healthcare market in the world; CYP-001 is part of the company’s larger ‘Cymerus’ product platform. Cynata specialises in stem cell treatments.
As advertised on the company's website, "The Cymerus technology [allows] economic manufacture of cell therapy products, including mesenchymal stem cells (MSCs), at commercial scale."
Get the latest news and media direct to your inbox