US FDA Clears IND for Cynata's Phase 2 Clinical Trial of CYP-001 in GvHD

27 May 2022 | Friday | News

Cynata Therapeutics Limited (ASX: "CYP" or "Cynata"), a clinical-stage biotechnology company specialising in cell therapeutics, is delighted to announce that the United States (US) Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for Cynata's Phase 2 clinical trial of CYP-001[1], Cynata's lead product, in patients with acute graft versus host disease (aGvHD).
Image Source : Public Domain

Image Source : Public Domain

Key Highlights:

  • US FDA has cleared Cynata's IND application for a Phase 2 clinical trial of CYP-001 in patients with aGvHD – a major milestone and value catalyst for the Company
  • Phase 2 clinical study in aGvHD expected to commence subsequent to customary and satisfactory completion of negotiations with study centres and receipt of relevant ethics/administrative approvals
  • Transformative event for Cynata as it provides a gateway in the USA to potential further clinical targets and is a critical validation step for Cynata's ongoing commercial partnering activities

Dr Kilian Kelly, Cynata's Chief Operating Officer, said:
"Clearance of our IND application confirms that the FDA is satisfied with both the clinical and preclinical data as well as the manufacturing and quality control data on our product that we submitted in support of this application. This achievement represents a hugely important milestone in the development of CYP-001 and our Cymerus platform. We had previously completed several positive interactions with the FDA, as well as securing Orphan Drug Designation for CYP-001 in 2018.  However, the planned Phase 2 aGvHD trial will be our first clinical trial conducted in the US, which is the largest healthcare market worldwide.  We look forward to commencing this Phase 2 study, which aims to build on the very encouraging Phase 1 clinical trial results.[2]"

Dr Ross Macdonald, Cynata's Chief Executive Officer, said:
"A cleared IND represents a very significant achievement for any pharmaceutical or biotech company and underpins a major valuation catalyst; I would like to thank our shareholders for their patience during this process. We are delighted to have crossed this threshold and 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 proposed Phase 2 clinical trial will seek to recruit approximately 60 patients with high risk aGvHD, at clinical centres in a number of countries, including the US and Australia. Participants will be randomised to receive either CYP-001 or placebo, in addition to corticosteroids. The primary objective of the trial is to assess efficacy of CYP-001 in subjects with high risk aGvHD by Overall Response Rate (ORR) at Day 28. The trial is expected to commence enrolment by later this year, with results of the primary evaluation expected in early 2024.

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