Cynata receives key FDA approval to progress GvHD drug candidate

For the second time in as many days, the US Food and Drug Administration (FDA) has granted important permission to an ASX-listed company in the biotech space.

On this occasion, the FDA granted Cynata Therapeutics (ASX: CYP) with an Orphan Drug Designation for CYP-001, Cynata’s drug candidate aiming to treat acute graft versus host disease (GvHD).

Yesterday, Adherium (ASX: ADR) received permission to market its Smartinhalers across the US, which saw the company’s shares rally by over 45%.

GvHD is a condition that sometimes occurs after bone marrow transplants, whereby the donated bone marrow or peripheral blood stem cells view the recipient’s body as foreign, and the donated cells/bone marrow attack the body following the procedure – essentially as an undesirable immune response that Cynata wants to avoid with its CYP-001 drug candidate.

To progress its drug candidate, Cynata opted to apply for the FDA’s orphan drug designation which means it will be classified as a therapeutic agent used for the prevention, diagnosis or treatment of a rare disease, which is defined as a disease or condition that affects fewer than 200,000 people in the USA.

The “Orphan Drug Designation”, means that Cynata will be eligible for a range of incentives including an extended period of marketing exclusivity, tax credits and FDA fee waivers. More specifically, Cynata will have marketing exclusivity protections for a period of 7 years and will be able to claim back 50% of the qualified clinical research costs for CYP-001 as tax credits.

The grant also positions CYP-001 on the starting line towards becoming fully approved after several clinical trials and efficacy tests as set by the FDA.

If successful, Cynata could see its drug candidate marketed in the largest healthcare market in the world, currently worth around US$2 trillion in the US alone and US$8.7 trillion globally, according to research carried out by Deloitte.

Delving into GvHD and CYP-001 efficacy

CYP-001 is the lead mesenchymal stem cell (MSC) product manufactured using Cynata’s proprietary Cymerus platform manufacturing technology.

Cynata recently announced encouraging data from the first cohort of patients in its ongoing Phase 1 trial of CYP-001 for the treatment of steroid-resistant acute GvHD.

The company said that “no treatment-related serious adverse events or safety concerns have been identified to date,” while among the eight participants in Cohort A (who received the lower dose level of CYP-001), overall survival at Day 100 was 87.5%.

According to Cynata, the overall response rate by Day 100 was 100% (all eight participants showed an improvement in the severity of GvHD by at least one grade compared to baseline) and the complete response rate by Day 100 was 50%, which means GvHD symptoms were completely resolved in 4 out of 8 patients).

Cynata is currently recruiting for its second, higher dose tests and says that recruitment is currently “progressing well”.

“We are delighted that the FDA has seen fit to grant Orphan Drug Designation to CYP-001, in recognition of the potential of this product to address the substantial unmet need associated with GvHD.

This follows on from our successful pre-IND meeting with the FDA last year, and we look forward to building our productive relationship with the FDA over the coming years,” said Dr Kilian Kelly, Vice President of Product Development at Cynata Therapeutics.

Today’s FDA approval helped Cynata’s shares rise by 10c in early morning trade up to $1.45 per share, equating to a gain of almost 8%.