Latest news with #RDEB
Yahoo
2 days ago
- 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
2 days ago
- 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
3 days ago
- 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]
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
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.
