Collagen Diseases

CLEVELAND, Oct. 29, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that the Company has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or Agency) for prademagene zamikeracel (pz-cel), its investigational autologous cell-based gene therapy, as a potential new treatment for patients with recessive dystrophic epidermolysis bullosa (RDEB). “We have worked closely with the FDA in preparing for the pz-cel BLA resubmission and thank the Agency for their ongoing guidance,” said Vish Seshadri, Chief Executive Officer of Abeona. “We have incorporated the Agency’s feedback and are confident that our resubmission package addresses all the Chemistry Manufacturing and Controls items identified in the Complete Response Letter, including observations from the completed Pre-License Inspection of our manufacturing facility. Upon acceptance of the BLA, we look forward to assisting the FDA in the completion of its review.” The BLA resubmission follows the Company’s Type A meeting in August 2024, where Abeona aligned with the FDA on the content of the resubmission, including additional information to satisfy all Chemistry Manufacturing and Controls (CMC) requirements outlined in the Complete Response Letter (CRL) received in April 2024. In the CRL, the FDA required that certain additional information needed to satisfy CMC requirements be provided before the application could be approved. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel. The BLA resubmission is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study (NCT04227106) and a Phase 1/2a study (NCT01263379). Upon acceptance of the BLA resubmission, Abeona expects the FDA to set a new Prescription Drug User Fee Act (PDUFA) target action date for pz-cel. The review period for a resubmitted BLA is either two or six months from the resubmission date depending on the FDA's classification of a resubmission as Class 1 or Class 2, respectively. The FDA’s decision on acceptance of a BLA resubmission is typically made within 14 calendar days following the resubmission. About prademagene zamikeracel (pz-cel) Prademagene zamikeracel (pz-cel), Abeona’s investigational autologous, COL7A1 gene therapy, is currently being developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disease caused by a mutation in both copies of the COL7A1 gene. As a result of this defect, cells are unable to express functional collagen VII protein which is needed to form anchoring fibrils that bond the epidermis to the dermis. Lack of anchoring fibrils leads to fragile skin that blisters easily and patients suffer from years of painful wounds, itch and increased risk of infection and squamous cell carcinoma. Pz-cel is made from patients' own skin cells that are genetically corrected with a functional COL7A1 gene integrated into the skin cells’ genome to express collagen VII. These gene-corrected cells are expanded to form keratinocyte sheets to cover wound areas in a single surgical application. Pz-cel has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA. About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features 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 www.abeonatherapeutics.com. 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.

Plus, news about Astellas, Clover, ElevateBio, Monopar, Idorsia and Neurocrine Biosciences: Vera Therapeutics plans $300M offering: The California biotech is selling shares after presenting Phase 2b data for an experimental drug called atacicept over the weekend. The company shared 96-week data for patients with immunoglobulin A nephropathy. — Kyle LaHucik Abeona Therapeutics resubmits rare skin disease therapy: Back in April, the FDA rejected Abeona’s cell therapy, called pz-cel, requesting more information about manufacturing. Abeona is developing pz-cel for recessive dystrophic epidermolysis bullosa, a rare genetic skin disease. — Lei Lei Wu Astellas yanks Izervay’s submission in Europe: The company said it withdrew its marketing authorization application for Izervay, a C5 protein inhibitor for geographic atrophy secondary to age-related macular degeneration. Astellas did not detail feedback from the Committee for Medicinal Products for Human Use, but said it is still “confident” in Izervay’s risk-benefit profile and will continue to engage with regulators in Europe and elsewhere. There are currently no treatments approved for GA secondary to AMD outside of the US, where Izervay was greenlit in August of last year. — Ayisha Sharma Clover releases early RSV vaccine data: The Shanghai-based biotech said its bivalent vaccine candidate, SCB-1019, produced “comparable” levels of RSV neutralizing antibodies to GSK’s Arexvy at 28 days in a Phase 1 test. Based on these data, Clover plans to advance the asset into studies focused on re-vaccination next year. — Ayisha Sharma ElevateBio lays off 4%: The genetic medicines company, which employs about 500 people, is ending its preclinical cellular engineering work, a spokesperson told Endpoints News on Monday. The Waltham, MA-based biotech laid off about 13% of its staff in October 2023. It’s one of the most well-funded private biopharma startups after having raised about $1.25 billion, including its $401 million Series D last year. Partnerships with companies like Novo Nordisk, Moderna, Kyverna and Affini-T are unaffected, the spokesperson confirmed. — Kyle LaHucik Monopar Therapeutics prices $19.2M offering: The Chicago-area biotech sold shares to Janus Henderson, RA Capital and others. A few days ago, Monopar licensed a Wilson disease drug candidate from AstraZeneca’s Alexion. — Kyle LaHucik Idorsia ends work with Neurocrine Biosciences: The companies were developing Idorsia’s T-type calcium channel blocker for patients with a rare form of pediatric epilepsy. The treatment failed a Phase 2 study in 2022, and further analysis led to the decision to stop work. — Jaimy Lee

欢迎关注凯莱英药闻 近日，Vivoryon Therapeutics在2024 年美国肾脏学会 (ASN) 上，公布了varoglutamstat的最新分析数据，显示与安慰剂相比，在有肾病风险以及患有糖尿病肾病 (DKD)人群中具有统计学和临床意义的肾小球滤过率（eGFR）  改善，且持续改善高于基线，表明肾脏功能可能开始恢复。 Varoglutamstat 是一款口服小分子谷氨酰肽环转移酶（QPCT）抑制剂；2021年6月，先声药业曾与Vivoryon达成战略性区域许可合作，获得在大中华区开发和商业化包括varoglutamstat（SIM0801）在内两款药物的权益。 关于最新研究数据 在这项2b 期多中心、随机、双盲、安慰剂对照、平行组剂量探索研究中，受试者分别给予每天两次，每次 300 mg或 600 mg的varoglutamstat或安慰剂，治疗持续时间为 48-96 周。在有肾病风险的老年人中，研究显示： 与安慰剂相比，varoglutamstat组的肾功能参数eGFR改善 3.4 mL/min/年（p<0.0001），具有统计学和临床意义的改善；在糖尿病患者亚组的改善约为 8.2 mL/min/年（p=0.02）。 其它几项敏感性分析结果保持一致，包括使用 CKD-EPI 2021 公式测定肌酐和胱抑素-C。 在安全性上，varoglutamstat组具有良好的安全性和耐受性，没有蛋白尿增加的迹象；两年研究期间没有发生不良的肾脏影响，且在总人群和糖尿病亚组中的肾脏和代谢系统不良事件中没有观察到有意义的差异。 关于Varoglutamstat Varoglutamstat具有治疗炎症和纤维化疾病、神经退行性疾病、癌症等疾病的潜力。 公司最初旨在评估varoglutamstat治疗阿尔茨海默病 (AD)的潜力，已在多项不同的临床试验中进行了研究。根据公司在2024年3月公布的数据，VIVIAD研究未达到其主要终点，也未显示出认知能力随时间推移而发生变化的显著统计学差异。此外，该研究未达到测量认知能力、日常生活活动工具问卷和脑电图全局θ功率的关键次要终点。 尽管 VIVIAD 中的患者是根据 AD 状态而非肾功能水平进行选择的，但其中许多人由于年龄和/或合并症而导致肾功能下降，分析显示varoglutamstat 对关键肾功能终点 (eGFR)的改善具有统计学意义，且促炎细胞因子 pE-CCL2 显著降低；促使公司计划针对3b/4 期 DKD 患者进行安慰剂对照的 2 期研究。 此外，公司还计划针对其他罕见/孤儿肾脏疾病（Alport 综合征、法布里病）适应症进行临床前研究。 关于QPCT/QPCTL药物 由于QPCT可催化具有神经毒性的N3pE淀粉样蛋白生成，N3pE与AD患者大脑中聚合成斑块的β淀粉样多肽有关，也会对AD其他病理因素产生不良影响。因此，包括Vivoryon等公司最早企图通过抑制QPCT来阻止N3pE淀粉样蛋白的形成，具有预防神经元损伤的潜力。 此外，QPCTL同样是潜在的癌症靶点。研究显示，QPCT在肾癌舒尼替尼耐药中所发挥的功能，下调QPCT 可以促进肾癌细胞对舒尼替尼的敏感性；此外，QPCT启动子区域的甲基化程度及QPCT的表达水平，亦可以作为预测肾癌舒尼替尼敏感性的生物学标志物。证明了QPCT 有望成为逆转肾癌舒尼替尼耐药发生的治疗新靶点，进一步提高肾癌靶向治疗的效果。 英矽智能和复星医药合作开发的重点项目ISM8207，在体内临床前研究中显示出抗肿瘤的功效，且表现出良好的药代动力学特征和安全性；此外，该药物不仅具有较高的肿瘤渗透性，还能降低血液学毒副作用避免抗原沉没。 据不完全统计，目前在研的QPCT/QPCTL药物有十种。 关于Vivoryon Vivoryon 是一家临床阶段生物技术公司，专注于开发创新的小分子药物。公司利用其在翻译后修饰方面的深厚专业知识来开发药物，以调节在疾病环境中发生改变的蛋白质的活性和稳定性。目前，公司已建立了一系列口服小分子抑制剂，用于治疗各种适应症，包括阿尔茨海默病、炎症和纤维化疾病（包括肾脏疾病）和癌症。 关于先声药业 先声药业成立于 1995 年，是一家创新与研发驱动的制药公司，聚焦神经科学、抗肿瘤、自身免疫及抗感染领域。 公司以自主研发及协同创新双轮驱动，与多家创新企业、科研院校建立战略合作伙伴关系。据不完全统计，近年来发生三十余笔交易。 参考资料 1、赵堂亮.DNA甲基化调控的QPCT在肾癌舒尼替尼耐药中的作用及机制研究[D].中国人民解放军海军军医大学,2019. 2、公司官网 3、太平洋证券 4、生物制药小编 感谢关注、转发，转载授权、加行业交流群，请加管理员微信号“hxsjjf1618”。 “在看”点一下