Latest news with #Zevaskyn
Medscape
04-07-2025
- Health
- Medscape
RDEB: Large Wounds Healed With Genetically Corrected Grafts
In a recently published phase 3 trial, credit card-sized cultured skin grafts corrected for the COL7A1 mutation that causes recessive dystrophic epidermolysis bullosa (RDEB) and enabled most patients to achieve at least 50% reductions in the size of large chronic wounds, with an overall mean pain score reduction of more than 2 points at week 24. In April 2025, prademagene zamikeracel (Zevaskyn, Abeona Therapeutics) became the first FDA-approved cell-based genetic therapy when it was approved for the treatment of wounds in adult and pediatric patients with RDEB. It is the first commercially available RDEB treatment to demonstrate sustained wound healing and pain reduction for large, chronic RDEB wounds, according to investigators. 'These wounds are the most terrible and difficult to treat in our patients,' the study's lead principal investigator Jean Y. Tang, MD , PhD, professor of dermatology at Stanford University School of Medicine in Stanford, California, said in an interview. 'To have a therapy using the patient's cells to suture on, hopefully close their wounds, and reduce their pain is monumental.' Jean Y. Tang, MD , PhD For the VIITAL trial, published online on June 23 in The Lancet , Tang and colleagues enrolled 11 patients with clinically and genetically confirmed RDEB (median age, 21 years) and no evidence of immune response to type VII collagen. To reduce the likelihood of immunogenicity, only patients with the amino-terminal NC1 fragment of type VII collagen could enroll. Investigators selected 43 wounds of at least 6 months' duration measuring at least 20 cm2 for treatment and compared these results against standard care for 43 randomly assigned control wounds matched for size, chronicity, and location. Grafting Process Using 8-mm punch biopsies from unaffected skin, investigators transduced isolated keratinocytes with a retrovirus carrying the full-length human COL7A1 gene, then used those keratinocytes to culture up to 12 40 cm2 sheets of autologous keratinocytes per patient. After 25 days, surgeons sutured up to six sheets of prademagene zamikeracel per patient, with each procedure taking 3-4 hours. To minimize pressure and friction, patients remained hospitalized with no changes of nonadhesive contact dressings for 7 days postsurgery. Images of wounds randomly assigned to prademagene zamikeracel or control at baseline, surgery, and week 24. Investigator assessments showed that 24 weeks posttreatment, 81% of treated patients achieved at least 50% healing from baseline vs 16% of control wounds ( P < .0001). Mean pain reduction from baseline (measured with the Wong-Baker Faces scale within 3 hours after dressing change) was 3.07 among treated patients vs 0.90 for control wounds ( P = .0002). Also at week 24, 16% of treated wounds achieved complete healing, with a 2.0-point decrease in itch severity from baseline. The corresponding figures for control wounds were 0 (healing) and 0.5 (itch). In the past 3 years, the FDA and the European Medicines Agency also have approved topical beremagene geperpavec (Vyjuvek) and birch triterpenes (Filsuvez) for dystrophic EB. However, wrote Tang and colleagues, the wounds treated with these therapies were mostly less than 20 cm2, and both treatments require repeated application. Nor did they improve pain or itchin clinical trials, added Tang. Having the first permanent gene correction for RDEB is very exciting, said Amy Paller, MS, MD, professor and chair of Dermatology and professor of pediatrics at Northwestern University, Chicago. She was not involved with the phase 3 study but will run the first of several specialized centers where prademagene zamikeracel will be applied. Amy Paller, MS, MD 'This is the first instance in our field where a gene has been corrected for grafting and is commercially available,' Paller said. 'It's something that we and our patients dreamed about for genetic skin disorders.' Logistics and Labor Performing the treatment is logistically complex and 'incredibly labor-intensive,' Paller said. The process requires rushing biopsies to Abeona's good manufacturing practice facility in Cleveland, where over the next few weeks, the keratinocytes are grown out, corrected, expanded markedly, and quality tested. 'It's a very expensive procedure with many moving parts,' she said. Accordingly, Paller plans to start with three patients from her own practice, beginning in August. Additionally, she is consulting with other interested families in the Midwest and will soon expand outreach to her other patients. 'I want experience with the process in patients I have known for years before grafting additional patients,' she explained. Prademagene zamikeracel's retroviral component may provoke discussion. Tang explained, 'We take the biopsy from the patient's skin, grow their keratinocyte skin cells, and use a retrovirus containing wild-type collagen VII to introduce that into the patient's skin cells. There's always a theoretical concern of retroviruses maybe hitting off-target genes, but so far, we and others haven't seen that.' In a phase 1/2a study, investigators followed seven patients treated with what was then known as EB-101 for a mean of 5.9 years. There were no serious adverse events related to treatment, with no gene therapy-related cutaneous or extracutaneous malignancies or evidence of systemic replication-competent retrovirus infections in serum samples from patients. The beauty of grafting skin, Paller added, is that development of a tumor — while unexpected — would be easily visible and biopsied, just as dermatologists now biopsy for suspected squamous cell carcinoma, a feared complication related to the scarred skin in patients with RDEB. Treated patients will require a long-term commitment to surveillance, she said, with a low threshold for considering biopsy if a change suggesting carcinoma is seen. The FDA recommends that manufacturers of genetic products follow patients for 15 years posttreatment. Clinical and Research Implications Although the phase 3 study showed the utility of correcting genetically defective collagen VII in treating RDEB, said Tang, the cell therapy approach could prove useful for additional genetic skin diseases such as ichthyosis and Gorlin syndrome. Paller said she hopes that junctional EB will be the next candidate for gene-corrected grafts. However, she added, with more extensive clinical experience and cost reductions over time, grafting of gene-corrected skin could be considered to improve focal areas in other forms of EB and genetic skin disorders. For the near term, Paller said she also hopes that insurers will not block access to the other approved RDEB treatments for patients who undergo prademagene zamikeracel treatment. 'I trust that that won't happen because these patients are so needy,' she said. To help patients access treatment, Abeona offers the Abeona Assist program, which helps patients understand their insurance benefits and financial assistance options and provides travel and logistical assistance. 'As far as I'm concerned,' said Paller, 'each patient with EB should have everything at our disposal to help — this is such a horrible disease. If I can graft a 12 credit card-sized area and then keep them going with tricks for other areas, I'll be very happy.' The study was funded by Abeona Therapeutics, which developed prademagene zamikeracel, which also conducted data analysis and employs several study co-authors. Tang is listed on the prademagene zamikeracel patent, which is licensed by Stanford University to Abeona, but she receives no royalties. Additionally, Tang has consulted on EB-related therapeutics for BridgeBio and Fibroderm. Paller served on the VIITAL data safety monitoring board and has consulted for Chiesi, Krystal, and Castle Creek Biosciences.
Yahoo
13-05-2025
- Business
- Yahoo
Abeona secures cash runway with $155m priority review voucher sale
US-based Abeona Therapeutics has signed a deal to sell its priority review voucher for $155m, two weeks after picking up approval in the US for its first commercial product. The biotech company was eligible for the voucher upon securing a rare paediatric disease designation. A voucher was subsequently given to the biotech after winning approval from the US Food and Drug Administration (FDA) for gene therapy Zevaskyn (prademagene zamikeracel) late last month. Zevaskyn is a treatment for the rare disease recessive dystrophic epidermolysis bullosa (RDEB). Abeona did not disclose the voucher's buyer, saying only it had entered a definitive asset purchase agreement. Investors seemed happy with Abeona's decision, with the company's stock, listed on the Nasdaq exchange, peaking 16.8% higher in trading yesterday, from $5.27 a share at close on Friday (9 May) to a high of $6.16. The company's stock later settled at $5.60 a share at market close – a 6.26% increase on the previous trading day. This spike coincides with stock increases across the pharmaceutical sector on Monday after US President Donald Trump announced he would sign an executive order to bring down US drug prices. Abeona has a market cap of $273.27m. Priority review vouchers are awarded to pharmaceutical companies developing drugs for rare paediatric diseases, neglected tropical diseases, or material threat medical countermeasures – markets that companies are hesitant to enter due to weak financial performance. Biotechs can use the vouchers to slash the FDA review time of any drug application from the usual ten months to six months. This can be done in a more lucrative market, facilitating a recuperation of funds spent developing the original product. Alternatively, companies can sell their voucher for a quick cash injection – the route opted by Abeona. The company did not reveal how it plans to use the funds, with CEO Joe Vazzano commenting only that the $155m means the company has 'sufficient cash for more than two years of operating expenses without the need for capital infusion.' Vazzano added that cash projections neither account for sales of Zevaskyn, which has a planned commercial rollout in Q3 2025. It is likely, therefore, that at least part of the funds raised from the voucher sale will go towards the gene therapy's launch. Zevaskyn will be priced at $3.1m a dose and is a one-time treatment. Analysis by GlobalData's Pharma Intelligence Centre forecasts sales of $366m by 2031. GlobalData is the parent company of Pharmaceutical Technology. The figure achieved by Abeona in its voucher sale tallies with other recent deals. Zevra sold a voucher in February for $150m, also to an undisclosed party. In November 2024, PTC Therapeutics sold a voucher to an unnamed buyer for $150m. The highest fee for a voucher still stands at $350m, when AbbVie bought one from United Therapeutics in 2015. Though down from these heights, recent transactions are still a step up from a relatively stable $100m selling price average seen over the past seven years, perhaps a reflection that vouchers now hold more value in an uncertain US funding landscape. Rare disease development has hit rocky waters amid mass layoffs across US health agencies by the Trump administration. A big setback could arise from the failure to renew the priority review voucher programme for paediatric rare diseases, which expired, in part, due to shifting political priorities. This has cast a blanket of precariousness over future rare disease pipelines. "Abeona secures cash runway with $155m priority review voucher sale" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
13-05-2025
- Business
- Yahoo
Abeona sells speedy drug review voucher for $155M
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Abeona Therapeutics has quickly cashed in on a recent drug approval, agreeing on Monday to sell a so-called priority review voucher awarded by the Food and Drug Administration to an undisclosed buyer for $155 million. Abeona earned the voucher two weeks ago, when the FDA cleared a cell therapy called Zevaskyn for a form of epidermoloysis bullosa, a rare skin condition. That approval, Abeona's first, was critical for its future prospects, as the company is counting on drug sales to help it break even financially next year. Like many other small drugmakers, Abeona is struggling with a depressed stock price that makes it difficult to raise equity. The voucher Abeona won is one tool it can use to bolster its cash holdings. These vouchers help speed up drug reviews and are regularly sold for $100 million or more. On a conference call last month discussing Zevaskyn's approval, company executives expressed urgency in completing a deal, given the uncertainty about the future of the FDA program governing these regulatory fast passes. On that call, CEO Vishwas Seshadri noted how the last four priority review voucher sales each totaled $150 million or more, and the company anticipated interest to 'remain strong.' Since November, Zevra Therapeutics, Acadia Pharmaceuticals and PTC Therapeutics all sold vouchers for $150 million, while Ipsen got slightly more, at $158 million, in a deal last August. 'We're confident that there is demand, and we will prioritize speed over any other further price optimization,' Seshadri said. In a statement, Abeona Chief Financial Officer Joe Vazzano said the voucher sale leaves the company with enough cash to operate for more than two years without the need for additional funding or accounting for any Zevaskyn sales. Abeona expects to become profitable 'in early 2026,' according to Vazzano. Abeona's cell therapy should be available in the third quarter. The company is competing for market share with Krystal Biotech, which sells a topical gel for a form of epidermolysis bullosa that's administered weekly. Zevaskyn, by comparison, is a one-time treatment made from a person's own skin cells. Analysts at the investment firm Jefferies have estimated Zevaskyn could generate $460 million in yearly sales at its peak. Abeona executives have said to expect gradual growth in uptake, with 10 to 15 patients likely treated this year and an acceleration afterwards. Abeona shares climbed 10% in early trading Monday, to about $6 apiece. Recommended Reading Acadia sells speedy drug review voucher for $150M
Forbes
02-05-2025
- Health
- Forbes
The Prototype: These Bacteria Can Generate Electricity
In this week's edition of The Prototype, we look at quantum computing for image recognition, a gene therapy using a patient's own skin cells, electricity-generating bacteria, using physics to cook perfect pasta and more. You can sign up to get The Prototype in your inbox here. Years ago, scientists found that certain kinds of bacteria appear to breathe by generating electricity, rather than taking in oxygen, but how they did so was a mystery. A new study published in the journal Cell identifies how this happens: the microbes are using a group of chemicals called naphthoquinones in a manner similar to the way a battery discharges electricity. This is what enables bacteria to thrive in oxygen-deprived areas like deep sea vents. Armed with this knowledge, it may be possible for scientists to adapt these bacteria for use in a wide variety of applications, such as wastewater treatment or developing bioelectronic sensors. It could even be used as a method to turn atmospheric carbon dioxide into useful chemicals–a sustainability twofer. Stay tuned. CEO Vishwas Seshdari Abeona Therapeutics Around 750 people in the United States have a rare genetic condition called recessive dystrophic epidermolysis bullosa (RDEB). People with this disease aren't able to correctly produce a particular type of collagen essential for the skin, causing excessive blistering and slowing the healing of wounds dramatically–if they heal at all. Earlier this week, the FDA approved a new treatment for this condition called Zevaskyn, which was developed by gene therapy company Abeona Therapeutics. The product is manufactured using a patient's own skin cells, which have been genetically modified to produce the right kind of collagen. Those cells are then formed into sheets that can be grafted to a patient at the site of a wound. In a clinical trial, 81% of the wounds treated with Zevaskyn showed significant healing after six months, compared to just 16% in patients using current therapies. What's more, the healing appears to be durable, showing long term improvements at follow-ups conducted years later. Patients getting the therapy also reported significantly less pain. Abeona's CEO Vishwas Seshdari told me that with the approval, the company should be ready to start treating patients by July, starting with a manufacturing cadence of being able to treat about six patients a month. The price per treatment is $3.1 million, with patients expected to receive one or two in their lifetime, and Seshdari said that it's already working on reimbursement arrangements with payers to ensure those who need it can get it. The company is also expanding its manufacturing capability with an eye to being able to treat 10 patients a month by the first half of next year. 'We can't wait to bring this therapy to patients,' he said. Quantum software company BlueQubit has figured out how to use quantum computing to power image-classifying AI. Working with the Honda Research Institute, it developed three different ways to encode images so they can be manipulated by quantum computing hardware. They were then used both by regular computers using quantum algorithms as well as quantum computers manufactured by IBM. The company found that one of the encoding methods enabled quantum-powered AI to classify images with about 94% accuracy, which is comparable to what can be achieved by classical computers. That said, current methods of classifying images are still faster. But by developing a way to do image classifying on quantum hardware, this research sets the stage for this type of application to be used when quantum computers have scaled to the point where they're regularly out-performing regular computers. On Monday morning, 27 satellites for Amazon's Project Kuiper were launched into orbit. CEO Andy Jassy confirmed on social media that day that all of them were operational. The company intends to eventually have over 3,200 satellites in orbit, with a goal of providing broadband internet access around the globe. In my other newsletter, InnovationRx, Amy Feldman and I looked at a patent fight over the world's top-selling drug, new breakthroughs from the American Association for Cancer Research, a dataset to better understand Parkinson's, the relationship between microplastics and heart disease and more. Waymo is partnering with Toyota to design a new platform for autonomous cars and trucks with an aim to develop them for personal use. Scientists have discovered a massive, glowing molecular cloud just 300 light years from our solar system. This vast cloud of gas and dust, which has been dubbed Eos, is primarily composed of hydrogen and will evaporate in around 6 million years. A team of researchers has developed a soft exoskeleton called the MyoStep, which is made of lightweight materials and can help kids with cerebral palsy walk and play. Starbucks has 3D-printed its latest store, a 1400 square foot, drive-thru only facility in Brownsville, Texas. A genetically modified probiotic could remove mercury from seafood after you eat it. The gut microbe was developed by researchers at UCLA and UC San Diego, and was shown to reduce the amount of mercury passing into the brain and fetuses of mice who were fed a diet of fish. Cacio e pepe is a deceptively simple Italian dish with only three ingredients - pasta, black pepper and pecorino romano cheese, which combined make a rich and creamy dish. Or, if you're like me, a clumpy mess. But a team of physicists have figured out how to do it flawlessly every time, and published their results in the journal Physics of Fluids. The key is to add powdered starch to the water before cooking the pasta, which ensures there's enough of it to allow the cheese to blend. It's also important to make sure the water cools before you add the cheese, blending it in, then heating the resulting sauce slowly to avoid clumps. If you're looking for a dish to make this weekend, give it a try. I've been greatly enjoying Guy Ritchie's series MobLand on Paramount+. It's an organized crime drama set in London that primarily follows Tom Hardy's Harra Da Souza, who works as chief enforcer for his boss, played by Pierce Brosnan. The show itself is well-executed and entertaining, but it's elevated by Helen Mirren clearly having a ball playing a Lady MacBeth-esque crime boss's wife. I definitely recommend it.
Yahoo
30-04-2025
- Business
- Yahoo
FDA approves Abeona's $3.1m cell therapy for rare skin disease
Abeona Therapeutics has secured US Food and Drug Administration (FDA) approval for Zevaskyn (prademagene zamikeracel), a gene-corrected cell therapy designed to treat recessive dystrophic epidermolysis bullosa (RDEB). The approval follows years of clinical development and regulatory setbacks, and positions Abeona as the second company to bring a therapy for this condition to the US market. Also known as pz-cel, Zevaskyn is indicated for both paediatric and adult patients with RDEB. The therapy is administered as credit card-sized sheets of skin made from the patient's own keratinocytes, which are harvested, genetically modified to express the functional COL7A1 gene, and surgically applied to chronic wounds. The COL7A1 gene encodes a type of collagen essential for anchoring skin layers – something patients with RDEB lack due to genetic mutations. The FDA had previously rejected an application from Abeona last year, citing outstanding manufacturing and quality control (QC) concerns. Following a complete response (CR) letter and further chemistry, manufacturing and controls (CMC) submissions, the agency granted approval based on results from a pivotal Phase III study (NCT04227106). Despite a partial clinical hold in 2019, the study met both co-primary endpoints of wound healing and pain reduction, with no serious treatment-related adverse events (AEs) reported. The company also provided confirmatory evidence from a Phase I/IIa trial (NCT01263379), which supported the durability of healing after a single application. Zevaskyn is priced at $3.1m, placing it among the most expensive therapies in the US but in line with other rare disease gene and cell therapies. Abeona expects to make the product available commercially in Q3 2025. The company raised $35m in late 2022 after announcing the Phase III data, followed by a $25m financing round in July 2024 to support manufacturing and launch readiness. Krystal Biotech's Vyjuvek (beremagene geperpavec), a topical gene therapy gel for DEB, was approved in 2023 and generated $290.5m in revenue in 2024. Unlike Zevaskyn, which is applied once through surgery, Vyjuvek is dosed weekly and costs around $631,000 per patient per year. Vyjuvek also delivers a functional COL7A1 gene, though via a re-dosable, non-integrating viral vector. In the 29 April announcement, Abeona's CEO Vish Seshadri, said: 'We have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic and prone to infection. Through a single surgical application, Zevaskyn can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal Phase III study.' In connection with the approval, Abeona received a rare paediatric disease priority review voucher (PRV), which the company said it plans to monetise. The PRV programme, which grants expedited FDA review for a future drug application, is set to expire soon unless renewed by Congress. Rare disease advocates have warned that the programme's lapse, combined with existing FDA resource constraints, could deter future investment in paediatric rare disease research and development. "FDA approves Abeona's $3.1m cell therapy for rare skin disease" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
