“Third time’s the charm,” the old saying goes, but for Abeona Therapeutics (NASDAQ: ABEO), the second time around proved successful for the company’s first-ever pipeline candidate to make it through clinical trials.
The FDA approved Abeona’s Zevaskyn
(prademagene zamikeracel) as the first (and so far only) cell-based gene therapy to be authorized for recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue skin disorder caused by mutations in the COL7A1 gene.
RDEB patients lack functional Type VII collagen, resulting in extremely fragile skin that blisters easily, leading to painful chronic wounds that often cover more than 30%, and sometimes up to 80%, of a patient’s body surface, as well as increased infection risk and elevated risk of squamous cell cancer. Until recently, treatment options have been limited to wound care and pain management rather than addressing the underlying genetic cause.
Zevaskyn consists of a patient’s own keratinocytes, skin cells that are genetically modified to produce functional Type VII collagen. Zevaskyn gene-modified cellular sheets are applied via surgery to a patient’s wounded areas, with a manufacturing run able to supply up to 12 credit card-sized sheets that can be joined together in a single application of the therapy to cover a large area or applied to multiple wounds.
“It’s almost like an application of tiles to cover a large area. And if you have a smaller area, one or two, or three sheets can cover that. So there’s flexibility of how many sheets you use, and it can be done in one sitting,” Abeona president and CEO Vishwas (Vish) Seshadri, PhD, told GEN Edge.
Zevaskyn won FDA approval upon Abeona’s second filing of a biologics license application (BLA) for the therapy. The first BLA was rejected last year, through an FDA Complete Response Letter citing chemistry, manufacturing, and controls (CMC) concerns related to Zevaskyn, but not questioning its safety and efficacy as shown in clinical data.
“They [the FDA] probably didn’t anticipate that level of complexity in our product, and were not prepared for it. That’s what caused them to give us a complete response letter and give us those items to address,” Seshadri said. “And we did exactly that, which is why we got the approval.”
The FDA based its approval on positive data from the Phase III VIITAL trial (NCT04227106) showing that across 43 large and chronic wounds treated with a single application of Zevaskyn, 81% of the wounds had healed by ≥50% after six months, compared to 16% of wounds receiving standard care. Fewer than 5% of patients experienced side effects such as procedural pain or itch.
In approving Zevaskyn, the FDA also granted Abeona a Rare Pediatric Disease Priority Review Voucher (PRV), which the company plans to sell.
Investors responded to the FDA’s approval of Zevaskyn with a buying surge that sent Abeona’s stock price soaring 30% over two days, from $5.30 to $6.56 Wednesday, when it scored its 52-week high of $7.08—then up to $6.87 Thursday, before sliding 4.5% Friday to $6.56.
“We saw risk in approval, getting a PRV, pricing, and pot’l for label surprises, and the update seemed (+)ve [positive] to us across the board,” Maury Raycroft, PhD, equity analyst with Jefferies, and four colleagues wrote April 29 in a research note. Raycroft raised his firm’s 12-month price target on Abeona shares 21%, from $14 to $17.
Raycroft projected Zevaskyn will achieve $108 million in revenue next year, its first full year on the market. “I think that’s pretty good,” Seshadri said of the estimate, which is based on treatment of 45 patients.
The company told investors in March that it estimated peak-year annual U.S. revenue for the therapy of more than $500 million.
That’s more than double the $290.5 million generated last year by Krystal Biotech’s marketed therapy Vyjuvek® (beremagene geperpavec-svdt)—the third highest sales of any gene therapy in 2024, according to an updated A-List to be published by GEN.
Unlike Zevaskyn, Vyjuvek is a gel applied weekly. Vyjuvek is a Herpes-simplex virus type 1 (HSV-1) vector-based gene therapy that won FDA approval in 2023 to treat wounds in patients six months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. RDEB is a severe form of DEB.
Among clinical candidates for RDEB is allo-APZ2-CVU, a first-in-class intravenous stem cell therapy that uses ABCB5-positive mesenchymal stem cells (MSCs) to treat patients with severe immune and inflammation-driven diseases with high unmet medical need. Developed by Rheacell, allo-APZ2-CVU is under study in a Phase III trial (NCT05838092).
Last year, Phoenicis Therapeutics studied PTW-002, an antisense oligonucleotide (AON)-mediated RNA regulation therapy in a Phase I/II trial (NCT05529134) in patients with RDEB or dominant DEB due to mutation(s) in exon 73 of the COL7A1 gene. The company’s website has since been taken online, and no results have been posted for the trial.
Abeona plans to slowly but steadily increase the availability of Zevaskyn starting in the third quarter, when it expects to treat the first patient. Ten to 12 patients are expected to be treated this year. If 12 patients are treated, Abeona stands to make $28.4 million in revenue, Raycroft estimated.
The company will make Zevaskyn available through five Qualified Treatment Centers (QTCs) expected to be established by the third quarter. The sites will be disclosed once activated.
Manufacturing will be ramped up to six Zevaskyn treatments per month by the end of 2025, then up to 10 per month in the first half of 2026. Abeona will commercially manufacture Zevaskyn at its fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, where the company is headquartered.
“We’re starting with an ability to supply about four units a month. When we launch, which is in July, we have said that we anticipate treating the first patient and ramping up that capacity. In the first half of 2026, we should hit that 10-per-month cadence,” Seshadri said. “Revenues will be driven not just by the demand, how many patients there are out there seeking treatment. It’s actually more of how much we can supply.”
Zevaskyn carries a list price or “wholesale acquisition cost” of $3.1 million per one-time treatment, making it one of the priciest gene therapies ever approved. The most expensive is Lenmeldy
(atidarsagene autotemcel), a treatment for early-onset metachromatic leukodystrophy, which is list-priced at $4.25 million and marketed by Kyowa Kirin-owned Orchard Therapeutics.
Seshadri defended the list price by saying it needed to be viewed in context of:
“There is no cure. They live in constant pain all their lives. And we’re trying to make a big difference to their quality of life,” Seshadri said.
Abeona said it plans to broaden access to Zevaskyn through its Abeona Assist
program, which is designed to offer personalized support, including helping patients understand their insurance benefits and financial assistance options, and providing travel and logistical assistance for eligible patients.
Healthcare providers will offer Zevaskyn through value-based agreements with payers that tie reimbursement to patient care outcomes rather than volume of patients treated. Seshadri said government payers will be able to hammer out value-based reimbursement agreements now that Zevasky has attained FDA approval, while the company has had talks with several private insurers: “Nobody has blinked at our pricing. They see the value, so there’s going to be coverage for these patients.”
“We’re going to make sure that whether you’re a patient that’s covered by private commercial insurance or Medicaid, or even the small subset of patients that may have Medicare for disability, we’re going to make sure that their access is going to be available,” Seshadri said. “We are standing behind our product.”
That means if a patient comes for repeat treatment of a wound, payers receive rebates from the company.
Because Zevaskyn had previously been designated a rare pediatric disease drug, it was grandfathered under the agency’s Rare Pediatric Disease PRV program, despite it starting to sunset on December 20, 2024, after Congress failed to agree on a renewal.
As a result of the sunset, the average value of PRVs has skyrocketed from the $100 million cited in a study published last year to $150 million.
“We believe that we should be able to achieve that kind of sale, but it’s always going to be a tradeoff with how quickly we want to do it, because even those that sold for 150, sometimes they started significantly lower, or the companies held off selling for months,” Seshadri observed.
The 2024 study found individual vouchers had sold for between $67.5 million and $350 million.
“We have to monetize the PRV rather quickly. But what we are confident about is that second quarter-ish of 2026, we should be a profitable entity,” Seshadri said. “If we monetize the PRV quickly, it will bridge us to profitability.”
Seshadri said Abeona can use the money it would generate from a PRV sale, since its cash runway is only enough to take the company into 2026. Abeona finished 2024 with available cash, cash equivalents, short-term investments, and restricted cash of $98.1 million, up 87% from $52.6 million as of December 31, 2023.
Abeona finished 2024 with a net loss of $63.7 million, 17.5% above the company’s $54.2 million net loss for 2023. Abeona recorded no revenue last year and only $3.5 million in 2023.
Abeona has a workforce of “150-plus” that is expected to expand as the company grows its manufacturing, particularly quality assurance/quality control (QA/QC) staff.
“Mostly manufacturing personnel and technical operations are going to increase. Basically, everything that needs to support the scale-up of Zevaskyn supply,” Seshadri said.
The faster it can scale up Zevaskyn supply, the better able Abeona will be to maximize two opportunities—treating RBED patients with the first one-time treatment available for the disorder, and advancing rare disease genetic medicines, according to Paula Cannon, PhD, president of the American Society of Gene + Cell Therapy (ASGCT) for 2024–2025.
“This landmark approval represents a significant breakthrough for patients suffering from this devastating skin disorder,” said Cannon, who is also a distinguished professor of molecular microbiology & immunology at the University of Southern California (USC)’s Keck School of Medicine. “This approval not only brings hope to those living with RDEB but also represents significant progress in general for gene-corrected cell therapies for rare genetic disorders.”
The post StockWatch: Second Time’s the Charm for Abeona’s Gene Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.
The FDA approved Abeona’s Zevaskyn
RDEB patients lack functional Type VII collagen, resulting in extremely fragile skin that blisters easily, leading to painful chronic wounds that often cover more than 30%, and sometimes up to 80%, of a patient’s body surface, as well as increased infection risk and elevated risk of squamous cell cancer. Until recently, treatment options have been limited to wound care and pain management rather than addressing the underlying genetic cause.
Zevaskyn consists of a patient’s own keratinocytes, skin cells that are genetically modified to produce functional Type VII collagen. Zevaskyn gene-modified cellular sheets are applied via surgery to a patient’s wounded areas, with a manufacturing run able to supply up to 12 credit card-sized sheets that can be joined together in a single application of the therapy to cover a large area or applied to multiple wounds.
“Application of tiles”
“It’s almost like an application of tiles to cover a large area. And if you have a smaller area, one or two, or three sheets can cover that. So there’s flexibility of how many sheets you use, and it can be done in one sitting,” Abeona president and CEO Vishwas (Vish) Seshadri, PhD, told GEN Edge.
Zevaskyn won FDA approval upon Abeona’s second filing of a biologics license application (BLA) for the therapy. The first BLA was rejected last year, through an FDA Complete Response Letter citing chemistry, manufacturing, and controls (CMC) concerns related to Zevaskyn, but not questioning its safety and efficacy as shown in clinical data.
“They [the FDA] probably didn’t anticipate that level of complexity in our product, and were not prepared for it. That’s what caused them to give us a complete response letter and give us those items to address,” Seshadri said. “And we did exactly that, which is why we got the approval.”
The FDA based its approval on positive data from the Phase III VIITAL trial (NCT04227106) showing that across 43 large and chronic wounds treated with a single application of Zevaskyn, 81% of the wounds had healed by ≥50% after six months, compared to 16% of wounds receiving standard care. Fewer than 5% of patients experienced side effects such as procedural pain or itch.
In approving Zevaskyn, the FDA also granted Abeona a Rare Pediatric Disease Priority Review Voucher (PRV), which the company plans to sell.
Investors responded to the FDA’s approval of Zevaskyn with a buying surge that sent Abeona’s stock price soaring 30% over two days, from $5.30 to $6.56 Wednesday, when it scored its 52-week high of $7.08—then up to $6.87 Thursday, before sliding 4.5% Friday to $6.56.
“We saw risk in approval, getting a PRV, pricing, and pot’l for label surprises, and the update seemed (+)ve [positive] to us across the board,” Maury Raycroft, PhD, equity analyst with Jefferies, and four colleagues wrote April 29 in a research note. Raycroft raised his firm’s 12-month price target on Abeona shares 21%, from $14 to $17.
$108M revenue projected for 2026
Raycroft projected Zevaskyn will achieve $108 million in revenue next year, its first full year on the market. “I think that’s pretty good,” Seshadri said of the estimate, which is based on treatment of 45 patients.
The company told investors in March that it estimated peak-year annual U.S. revenue for the therapy of more than $500 million.
That’s more than double the $290.5 million generated last year by Krystal Biotech’s marketed therapy Vyjuvek® (beremagene geperpavec-svdt)—the third highest sales of any gene therapy in 2024, according to an updated A-List to be published by GEN.
Unlike Zevaskyn, Vyjuvek is a gel applied weekly. Vyjuvek is a Herpes-simplex virus type 1 (HSV-1) vector-based gene therapy that won FDA approval in 2023 to treat wounds in patients six months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. RDEB is a severe form of DEB.
Among clinical candidates for RDEB is allo-APZ2-CVU, a first-in-class intravenous stem cell therapy that uses ABCB5-positive mesenchymal stem cells (MSCs) to treat patients with severe immune and inflammation-driven diseases with high unmet medical need. Developed by Rheacell, allo-APZ2-CVU is under study in a Phase III trial (NCT05838092).
Last year, Phoenicis Therapeutics studied PTW-002, an antisense oligonucleotide (AON)-mediated RNA regulation therapy in a Phase I/II trial (NCT05529134) in patients with RDEB or dominant DEB due to mutation(s) in exon 73 of the COL7A1 gene. The company’s website has since been taken online, and no results have been posted for the trial.
First Zevaskyn patient set for Q3
Abeona plans to slowly but steadily increase the availability of Zevaskyn starting in the third quarter, when it expects to treat the first patient. Ten to 12 patients are expected to be treated this year. If 12 patients are treated, Abeona stands to make $28.4 million in revenue, Raycroft estimated.
The company will make Zevaskyn available through five Qualified Treatment Centers (QTCs) expected to be established by the third quarter. The sites will be disclosed once activated.
Manufacturing will be ramped up to six Zevaskyn treatments per month by the end of 2025, then up to 10 per month in the first half of 2026. Abeona will commercially manufacture Zevaskyn at its fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, where the company is headquartered.
“We’re starting with an ability to supply about four units a month. When we launch, which is in July, we have said that we anticipate treating the first patient and ramping up that capacity. In the first half of 2026, we should hit that 10-per-month cadence,” Seshadri said. “Revenues will be driven not just by the demand, how many patients there are out there seeking treatment. It’s actually more of how much we can supply.”
$3.1M price tag
Zevaskyn carries a list price or “wholesale acquisition cost” of $3.1 million per one-time treatment, making it one of the priciest gene therapies ever approved. The most expensive is Lenmeldy
Seshadri defended the list price by saying it needed to be viewed in context of:
- Current cost of care, which can run about $1 million a year for RDEB patients, many of whom die in their 30s or 40s
- The cost of other gene therapies
- The lack of other treatment options
“There is no cure. They live in constant pain all their lives. And we’re trying to make a big difference to their quality of life,” Seshadri said.
Abeona said it plans to broaden access to Zevaskyn through its Abeona Assist
Healthcare providers will offer Zevaskyn through value-based agreements with payers that tie reimbursement to patient care outcomes rather than volume of patients treated. Seshadri said government payers will be able to hammer out value-based reimbursement agreements now that Zevasky has attained FDA approval, while the company has had talks with several private insurers: “Nobody has blinked at our pricing. They see the value, so there’s going to be coverage for these patients.”
“We’re going to make sure that whether you’re a patient that’s covered by private commercial insurance or Medicaid, or even the small subset of patients that may have Medicare for disability, we’re going to make sure that their access is going to be available,” Seshadri said. “We are standing behind our product.”
That means if a patient comes for repeat treatment of a wound, payers receive rebates from the company.
Because Zevaskyn had previously been designated a rare pediatric disease drug, it was grandfathered under the agency’s Rare Pediatric Disease PRV program, despite it starting to sunset on December 20, 2024, after Congress failed to agree on a renewal.
Skyrocketing voucher values
As a result of the sunset, the average value of PRVs has skyrocketed from the $100 million cited in a study published last year to $150 million.
“We believe that we should be able to achieve that kind of sale, but it’s always going to be a tradeoff with how quickly we want to do it, because even those that sold for 150, sometimes they started significantly lower, or the companies held off selling for months,” Seshadri observed.
The 2024 study found individual vouchers had sold for between $67.5 million and $350 million.
“We have to monetize the PRV rather quickly. But what we are confident about is that second quarter-ish of 2026, we should be a profitable entity,” Seshadri said. “If we monetize the PRV quickly, it will bridge us to profitability.”
Seshadri said Abeona can use the money it would generate from a PRV sale, since its cash runway is only enough to take the company into 2026. Abeona finished 2024 with available cash, cash equivalents, short-term investments, and restricted cash of $98.1 million, up 87% from $52.6 million as of December 31, 2023.
Abeona finished 2024 with a net loss of $63.7 million, 17.5% above the company’s $54.2 million net loss for 2023. Abeona recorded no revenue last year and only $3.5 million in 2023.
Abeona has a workforce of “150-plus” that is expected to expand as the company grows its manufacturing, particularly quality assurance/quality control (QA/QC) staff.
“Mostly manufacturing personnel and technical operations are going to increase. Basically, everything that needs to support the scale-up of Zevaskyn supply,” Seshadri said.
The faster it can scale up Zevaskyn supply, the better able Abeona will be to maximize two opportunities—treating RBED patients with the first one-time treatment available for the disorder, and advancing rare disease genetic medicines, according to Paula Cannon, PhD, president of the American Society of Gene + Cell Therapy (ASGCT) for 2024–2025.
“This landmark approval represents a significant breakthrough for patients suffering from this devastating skin disorder,” said Cannon, who is also a distinguished professor of molecular microbiology & immunology at the University of Southern California (USC)’s Keck School of Medicine. “This approval not only brings hope to those living with RDEB but also represents significant progress in general for gene-corrected cell therapies for rare genetic disorders.”
Leaders and laggards
- Ardelyx (NASDAQ: ARDX) shares skidded 24.5% from $5.47 to $4.13 on Friday, after the company’s stock was downgraded from “Strong Buy” to “Outperform” by Raymond James, which sliced its price target on the company’s shares 15%, from $13 to $11. Raymond James reacted to Ardelyx announcing first-quarter results that missed analyst expectations. Ardelyx finished Q1 with a $41.1 million net loss or $(0.17) per share vs. a net loss of $26.5 million or $(0.11) per share in Q1 2024, reflecting increased costs associated with ongoing commercialization of tenapanor, marketed as Ibsrela® for adult irritable bowel syndrome with constipation and as Xphozah® for chronic kidney disease in adults on dialysis. Revenues jumped 61% from $46 million to $74.1 million, which the company said reflected increased net product sales and licensing revenue.
- Immunic (NASDAQ: IMUX) shares yo-yoed this week, tumbling 23% from $1.28 to 99 cents Tuesday, then rising 18% to $1.17 Wednesday after the company reported topline data that investors initially considered disappointing from its Phase II CALLIPER trial (NCT05054140) assessing its multiple sclerosis (MS) drug vidofludimus calcium (IMU-838). Immunic said vidofludimus calcium treatment resulted in a 5% improvement in annualized rate of percent brain volume change vs. placebo after two years of treatment, a “modest benefit” on the exploratory primary MRI endpoint. The drug was also shown to reduce the annualized rate of thalamic brain volume loss by 20% in patients with progressive MS vs. placebo, and reduce by 30% the relative risk of 24-week confirmed disability worsening events in the 152-patient primary progressive multiple sclerosis (PPMS) study population vs. placebo.
- Organon (NYSE: OGN) shares nosedived 27% from $12.93 to $9.45 after the company all but wiped out its quarterly dividend 93%, from 28 cents per share to 2 cents per share. The move is expected to redirect $200 million into debt reduction. “We have reset our capital allocation priorities to accelerate progress towards deleveraging, enabling a path to achieve a net leverage ratio of below 4.0x by year-end,” Organon CEO Kevin Ali stated. “Below 4.0x” means less than four times earnings before interest, taxes, depreciation, and amortization (EBITDA). “By deleveraging more rapidly, we will continue to strengthen the future prospects of the company,” Ali added.” The dividend is payable June 12 to stockholders of record at the close of business on May 12. Organon focuses on women’s health, biosimilars, and established medicines across a range of therapeutic areas.
- Regulus Therapeutics (NASDAQ: RGLS) shares more than doubled, rocketing 137% from $3.37 to $7.98 Wednesday after Novartis (SIX Swiss: NOVN:SW) announced it committed up to $1.7 billion to acquire the microRNA therapeutics developer focused on autosomal dominant polycystic kidney disease (ADPKD), the most common genetic cause of renal failure worldwide. The deal would bolster the buyer’s cardiovascular, renal, and metabolic therapeutic area with Regulus’ lead asset arabursen, a next-generation, miR-17-targeting oligonucleotide that recently completed a Phase Ib multiple-ascending dose clinical trial. Novartis agreed to pay Regulus $800 million upfront, and up to $900 million tied to achieving an undisclosed regulatory milestone. Novartis shares on the SIX Swiss Exchange dipped 0.45% from CHF 94.21 ($113.95) to CHF 93.79 ($113.44).
The post StockWatch: Second Time’s the Charm for Abeona’s Gene Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.