FDA grants broad orphan drug status to PTX, shoots share price up almost 30%

Clinical state oncology company Prescient Therapeutics (ASX: PTX) has received the US FDA’s Orphan Drug Designation for its PTX-100 drug, used for cancer treatment. Previously, the Company received ODD for peripheral T-cell lymphomas (PTCL) in 2022. Following that, it applied for ODD for cutaneous T-cell lymphomas (CTCL), and it was granted a blanket designation for all of its TCLs. How about that!

The U.S. Food and Drug Administration’s (FDA) orphan drug designation is a status granted to drugs and biologics that are intended to treat rare diseases or conditions that affect fewer than 200,000 people in the United States. If a drug receives orphan drug designation, it may be eligible for several incentives, including tax credits for clinical research costs, waived or reduced fees for FDA services, and seven years of market exclusivity if the drug is ultimately approved for the rare disease indication.

Prescient Managing Director and CEO, Steven Yatomi-Clarke, shared, “Prescient is delighted to be granted this Orphan Drug Designation by the FDA, and is pleasantly surprised for the granting of the designation that is broader than our request. This now confers the certainty of 7 years of market exclusivity for PTX-100 in a broader range of diseases with unmet or poorly met clinical need. We look forward to sharing updates on the PTX-100 trial shortly.” 

PTX-100 blocks the growth of a cancer enzyme, geranylgeranyl transferase-1 (GGT-1). Its uniqueness—or “orphanness”—rests on the fact that it is the only medicine in the world that can specifically block a pathway called RhoA, which is important for cancer cell growth. 

TCLs are a type of cancer that occurs when some white blood cells grow too much. There are different types of TCLs, and they all need better treatment, especially when cancer comes back after treatment or doesn’t go away with treatment. The orphan designation includes all types of TCLs. The current focus of a research study is on treating TCLs that come back or don’t go away with treatment.

Globally-renowned lymphoma expert, Professor H. Miles Prince, AM is leading the PTX-100 Phase 1b study which focuses on relapsed and refractory TCLs. An update on the trial is due imminently. 

The benefits of an Orphan Drug Designation are considerable and include guaranteed market exclusivity of seven years from granting of regulatory approval. It also includes a waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs, which had a value of over $4.7 million in 2022. 

The designation will allow Prescient to benefit from incentives that can assist the development of PTX-100. This new win complements the Company’s 868.5% increase in revenue to $137k in H1 FY23. However, its losses increased, too, to $2.8 million. 

The FDA approval has infused shareholders with optimism, with its share price increasing by 28.9%. This could be the turnaround point for the Company’s financials.