Latest news with #RDEB
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
01-07-2025
- Business
- Yahoo
Abeona Therapeutics® Announces New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
CLEVELAND, July 01, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced it has granted equity awards to new non-executive employees who joined the Company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4). On June 30, 2025, the Compensation Committee of Abeona's Board of Directors granted restricted stock equity awards as a material inducement to employment to 21 individuals hired by Abeona, which equity awards relate to, in the aggregate, up to 48,715 restricted shares of Abeona common stock. One-third of the shares subject to such restricted stock awards will vest yearly on each anniversary of the Grant Date, such that the shares subject to such restricted stock awards granted to each employee will be fully vested on the third anniversary of the Grant Date, in each case, subject to each employee's continued employment with Abeona on the applicable vesting dates. About Abeona Therapeutics Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona's ZEVASKYN™ (prademagene zamikeracel) is the first and only autologous cell-based gene therapy for the treatment of wounds in adults and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). The Company's fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio serves as the manufacturing site for ZEVASKYN commercial production. The Company's development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit ZEVASKYN™, Abeona Assist™, Abeona Therapeutics®, and their related logos are trademarks of Abeona Therapeutics Inc. Forward-Looking Statements This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as 'may,' 'will,' 'believe,' 'anticipate,' 'expect,' 'intend,' 'potential,' and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, the timing and outcome of the FDA's review of our BLA resubmission for pz-cel; the FDA's grant of a Priority Review Voucher upon pz-cel approval; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company's most recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws. CONTACT: Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics ir@ 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
03-06-2025
- Business
- Yahoo
Traws Pharma Announces Publication of Compelling Efficacy data in RDEB SCC Patients Treated with Legacy Oncology Drug Rigosertib
NEWTOWN, Pa., June 03, 2025 (GLOBE NEWSWIRE) -- Traws Pharma, Inc. (NASDAQ: TRAW) ('Traws Pharma', 'Traws' or 'the Company'), a clinical-stage biopharmaceutical company developing novel therapies to target critical threats to human health from respiratory viral diseases, today announced the publication of key clinical efficacy data for rigosertib, a legacy Traws Pharma oncology asset for which development and commercialization partners are being actively sought, in patients with RDEB SCC. The paper, published in the British Journal of Dermatology1, details the first clinical trial of any experimental cancer therapeutic in this rare and complicated monogenic disease. The results indicated an overall response rate of 80%, with complete responses in 50% of evaluable patients. 'These data indicate that rigosertib is a potential treatment for cutaneous SCC in RDEB patients, where there is a substantial unmet need and no approved therapies. The aggressive course of this disease is inadequately addressed by current treatment regimens, which produce limited response rates of mostly short duration,' said Victor Moyo, MD, Chief Medical Officer Oncology, Traws Pharma. 'We are excited to report the compelling efficacy and tolerability profile of rigosertib in this devastating, difficult to treat disease, and thank the patients, sponsors and investigators for their commitment to this program,' commented Iain Dukes, MA, DPhil, Interim CEO, Traws Pharma. 'Rigosertib is available for further development and commercialization, and we are committed to finding an appropriate partner to advance this important medicine to approval.' About RDEB-associated SCC RDEB is caused by insufficient expression of type VII collagen, which is responsible for anchoring the skin's inner layer to its outer layer. This leads to extreme skin fragility as well as chronic blistering and wound formation with recurrent infections in RDEB patients, many of whom go on to develop metastatic squamous cell carcinoma driven by overexpression of polo-like kinase 1 (PLK-1). RDEB-associated SCC tumors show a highly aggressive and early metastasizing course that makes them the primary cause of death for these patients, with a cumulative risk of death of 70% and 78.7% by ages 45 and 55, respectively2,3. RDEB-associated SCC can appear in pediatric patients or in young adults. Currently available treatments such as targeted therapies and conventional chemo- and/or radiotherapy have demonstrated limited response rates and poor durability in RDEB-associated SCC2,4. Abbreviations: RDEB SCC, Recessive Dystrophic Epidermolysis Bullosa Associated Locally Advanced or Metastatic Squamous Cell Carcinoma References Martin Laimer, Andrew P South, Elisabeth Mayr, Sophie Kitzmueller, Lauren Banner, Michael Alexander, Linda Hosler, Henry Yang, Matthew Parris, Meena Arora, Georg Zimmermann, Gregor Schweighofer-Zwink, Johann W Bauer, Neda Nikbakht, Efficacy and Safety of Rigosertib in Patients with Recessive Dystrophic Epidermolysis Bullosa Associated Advanced/Metastatic Cutaneous Squamous Cell Carcinoma, British Journal of Dermatology, 2025;, ljaf205, Mellerio et al. Br J Dermatol. 2016 Jan; 174(1):56-67. doi: 10.1111/bjd.14104. Fine et al. J Am Acad Dermatol. 2009 Feb; 60(2):203-11. doi: 10.1016/ Stratigos et al. Eur J Cancer. 2020 Mar;128:83-102. doi: 10.1016/ About Rigosertib Rigosertib is a small molecule kinase inhibitor (including PLK-1). The compound is being evaluated in a series of investigator sponsored studies including as a monotherapy for potential use in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC) (NCT03786237, NCT04177498), using both oral and IV formulations. Rigosertib has also been evaluated in combination with other agents, including checkpoint inhibitors, for solid tumors including non-small cell lung cancer, and metastatic melanoma. About Traws Pharma, Inc. Traws Pharma is a clinical stage biopharmaceutical company dedicated to developing novel therapies to target critical threats to human health in respiratory viral diseases. Traws integrates antiviral drug development, medical intelligence and regulatory strategy to meet real world challenges in the treatment of viral diseases. We are advancing novel investigational oral small molecule antiviral agents that have potent activity against difficult to treat or resistant virus strains that threaten human health: bird flu and seasonal influenza, and COVID-19/Long COVID. Tivoxavir marboxil is in development as a single dose treatment for bird flu and seasonal influenza, targeting the influenza cap-dependent endonuclease (CEN). Ratutrelvir is in development as a ritonavir-independent COVID treatment, targeting the Main protease (Mpro or 3CL protease). Traws is actively seeking development and commercialization partners for its legacy clinical oncology programs, rigosertib and narazaciclib. More details can be found on Traws' website at Follow our progress on our website or on LinkedIn. Forward-Looking Statements Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties including statements regarding the Company, its business and product candidates, including the potential opportunity, benefits and the regulatory plans for its rigosertib oncology program. The Company has attempted to identify forward-looking statements by terminology including 'believes', 'estimates', 'anticipates', 'expects', 'plans', 'intends', 'may', 'could', 'might', 'will', 'should', 'preliminary', 'encouraging', 'approximately' or other words that convey uncertainty of future events or outcomes. Although Traws believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including Traws' ability to identify and enter into development and/or commercialization agreements with potential partners for its legacy rigosertib oncology program, the success and timing of Traws' clinical trials, the efficacy of rigosertib as a treatment for cutaneous SCC in RDEB patients, market conditions, regulatory requirements, changes in government regulation, and those discussed under the heading 'Risk Factors' in Traws' filings with the U.S. Securities and Exchange Commission (SEC). Any forward-looking statements contained in this release speak only as of its date. Traws undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events, except to the extent required by law. Traws Pharma ContactNora BrennanTraws Pharma, Investor Contact:Bruce Mackle LifeSci Advisors, LLC646-889-1200bmackle@
Medscape
03-06-2025
- Business
- Medscape
Rigosertib Shows Promise in EB-Associated Skin Cancer
Rigosertib, a polo-like kinase 1 (PLK1) inhibitor, demonstrated antitumor efficacy in patients with recessive dystrophic epidermolysis bullosa (RDEB) and advanced squamous cell carcinoma (SCC), with an objective response achieved in four of five patients. METHODOLOGY: Researchers conducted an open-label, single-arm phase 2 study in Austria and the United States of five patients with advanced, treatment-refractory RDEB-SCC who had failed prior standard of care and were then treated with oral or intravenous (IV) rigosertib. Participants (median age, 23 years) received either oral rigosertib (560 mg twice daily on days 1-21 of each 28-day cycle for 13 cycles) or IV rigosertib (1800 mg/24 h as a 72-h continuous infusion on days 1-3 of each 14-day cycle for eight cycles, then on days 1-3 of 28-day cycles until 1 year of treatment). Two patients received IV treatment, two received oral treatment, and one received a combination of both. The primary outcome was the objective response rate and safety. Quality of life was a secondary outcome. TAKEAWAY: Two patients achieved complete a compete response, and two others had partial responses. The fifth patient discontinued treatment early because of unrelated complications. Rigosertib was generally well tolerated. Most adverse events were urinary-related (cystitis, hematuria) and manageable with dose adjustments or symptomatic treatment. IN PRACTICE: 'These initial results indicate rigosertib as a promising drug therapy for RDEB-SCC where there is a substantial unmet need and no approved therapies,' the study authors wrote. The study, they noted, was the 'first clinical trial of any experimental cancer therapeutic in this rare and complicated monogenic disease.' SOURCE: The study was led by Martin Laimer, Department of Dermatology and Allergology and EB House Austria, University Hospital of the Paracelsus Medical University, Salzburg, Austria, and was published online on May 29 in British Journal of Dermatology . LIMITATIONS: Usefulness of imaging techniques in this study was limited, particularly because of the chronic inflammatory response in lesional skin that is inherent to RDEB. DISCLOSURES: The study was funded by Debra International through Debra Austria, Debra of America, Cure EB, and Debra UK. Laimer and three other authors reported receiving consulting fees and grants from Onconova Therapeutics, Inc. Two authors declared being employees of Onconova, which provided the study drug.
Associated Press
02-06-2025
- Business
- Associated Press
Abeona Therapeutics® Announces New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
CLEVELAND, June 02, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced it has granted equity awards to new non-executive employees who joined the Company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4). On May 31, 2025, the Compensation Committee of Abeona's Board of Directors granted restricted stock equity awards as a material inducement to employment to five individuals hired by Abeona, which equity awards relate to, in the aggregate, up to 11,500 restricted shares of Abeona common stock. One-third of the shares subject to such restricted stock awards will vest yearly on each anniversary of the Grant Date, such that the shares subject to such restricted stock awards granted to each employee will be fully vested on the third anniversary of the Grant Date, in each case, subject to each employee's continued employment with Abeona on the applicable vesting dates. About Abeona Therapeutics Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona's ZEVASKYN™ (prademagene zamikeracel) is the first and only autologous cell-based gene therapy for the treatment of wounds in adults and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). The Company's fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio serves as the manufacturing site for ZEVASKYN commercial production. The Company's development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit ZEVASKYNTM, Abeona AssistTM, Abeona Therapeutics®, and their related logos are trademarks of Abeona Therapeutics Inc. Forward-Looking Statements Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics [email protected]
