Learning a Lesson With Our Own Skin
Abeona is a company selling Zevaskyn therapy – hope for patients with a rare, devastating skin disease – recessive dystrophic epidermolysis bullosa (RDEB). It is one of the last speculative stocks we still own. Excited about the promise of cell and gene therapy we bought it in 2021 when our investment style was still taking shape. We would not be buying it today, but we are not yet cutting substantial losses as we want to learn this lesson fully – commercialization story is about to deliver its outcome.
RDEB is caused by genetic mutation. As a result, skin lacks "glue" that is holding layers together and tears at the slightest friction. Patients develop chronic, excruciating wounds over their bodies. Many die young from infection. The standard care is Vyjuvek gel, but it requires indefinite weekly dosing. It is better suited for smaller wounds. Large, chronic, non-healing wounds that define the disease remain a source of suffering.
Abeona's Zevaskyn provides durable wound healing for up to eight years after a single application. A second one will be needed in the patient's lifetime. Both will cost 6.2 million dollars in total. It still makes sense economically, because Vyjuvek costs tens of millions cumulatively in a lifetime. We understand that reimbursement authorization in the U.S. is 100% – health insurance is willing to cover.
Abeona has already identified 100 eligible patients. Management think in the U.S. alone there are 750 people that will need Zevaskyn therapy. That is 4.6 billion dollars in potential revenue on the cards, not counting any international expansion. Even at a valuation of 0.66 billion, which is the price we paid for our shares, there is still a possibility for a multibagger return.
Abeona has manufacturing capacity to treat six patients per month and targets to expand it to ten, which for now seems like a hard limit. If the company can serve more than three patients a month – roughly 40 a year – it crosses into profit zone, according to CEO Vishwas Seshadri. We have taken note, and if it is not the case by the start of 2027 – we will exit the position. The clock is ticking, because Zevaskyn was approved April 2025, and nearly a year since, as of the start of 2026, only two commercial patients were treated amid delays. We think the company will have enough cash to run to the middle of 2027 if profitability is not reached.
CEO Seshadri has some six million dollars of wealth kept in Abeona shares, but he is trimming his share, not buying. He and his Chief Commercial Officer Madhav Vasanthavada both have experience in launching complicated therapy, logistically similar to Zevaskyn. They led Breyanzi (liso-cel) at Celgene-BMS.
Zevaskyn takes patients' own skin cells, transduces them ex vivo with functional genes. It then expands them into credit-card-sized sheets. Those sheets are surgically grafted onto wound sites. The corrected cells integrate permanently into the genome and start producing functional collagen needed to hold skin together. Every batch is manufactured for one specific patient. The sheets have an 85-hour shelf life once released. Patients must come to one of only four qualified centers and stay hospitalized for at least five days. The journey from consultation to treatment takes four months. To sum up, margin for error is thin, but the path to profitability is visible at relatively modest volumes.
Any delays and manufacturing failures will sink Abeona. It will prove that autologous cell therapy commercialization is too complex, too slow, and too expensive for a small biotech. A similar case of Bluebird Bio already exists. It had three comparable approved therapies and failed to achieve viable commercial scale. The uncertainty over the future of Abeona is very high. But we have committed only a fraction of our portfolio to the company. We are ready to see how the case plays out soon to learn the lesson of investing in biotech with our skin, so we never repeat the same mistake again.