Pz-Cel Has Massive Potential For RDEB Patients

2025-04-21 10:00:00 ET

Summary

• Abeona's pz-cel gene therapy shows the most durable wound healing in RDEB patients with no treatment related severe adverse events. There have been no instances of Squamous Cell Carcinoma.
• The FDA's previous Complete Response Letter focused on manufacturing and control issues. Since those questions have been answered thoroughly, an approval is likely by April 29th.
• Pz-cel offers a durable, one-time treatment. This reduces long-term costs compared to VYJUVEK which requires ongoing weekly applications.
• I believe pz-cel will be priced above the floor price of $1.5 million. It could be as high as $2 million per treatment.
• I believe Abeona can reach global peak sales of $1.04 billion. Despite this high potential, the EV is near $0 when you back out the cash & the PRV sale.

RDEB Is Devastating

Recessive Dystrophic Epidermolysis Bullosa (RDEB) is an extremely horrible disease. On average, 30% of the body is covered in chronic wounds (that don’t heal even after years). Some patients have as much as 80% of their body covered in wounds. For context, your palm is 1% of total body surface area. It takes 18 hours per week to change the bandages (2-3 changes per week).

In the control group of Abeona’s ( ABEO ) Phase 3 trial, which I will detail, 0% of wounds completely closed. 50% of generalized severe patients die before the age of 35; there’s a 76% likelihood of death by 40. Dystrophic Epidermolysis Bullosa (DEB) patients spend as much as $996k per year on wound care supplies. Their healthcare costs are 465% greater than that of the average person.

The Cause Of The Disease & Its Addressable Market

DEB is caused by mutations in the collagen type 7 alpha 1 chain. This chain provides instructions to assemble type 7 collagen which is the Velcro that sticks the epidermis and the dermis together. The epidermis is the top and the dermis is the bottom layer of the skin. Without this stabilizing force between the epidermal basement membrane and the dermis, the skin is at risk of blistering and wounds which can’t heal in the worst patients. RDEB is the worse (recessive) version of DEB with the larger wounds that won’t heal....

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