Certepetide shown to promote immune cell infiltration into tumors and inhibit metastasis July 10, 2024 -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announces promising preclinical results for its investigational candidate, certepetide (formerly LSTA1). The data, presented in a poster by Dr. Dan G. Duda of Massachusetts G

Sustained exposure to OV329 in preclinical models reduced GABA-aminotransferase (GABA-AT) activity, increased steady state GABA levels in the brain, and induced phasic and tonic inhibition OV329 demonstrated anti-convulsant effects in mice, reducing the severity of status epilepticus and preventing the development of benzodiazepine-resistant seizures OV329 was shown to have a higher potency (as measured by IC50) for the GABA-AT target than published studies of vigabatrin, an FDA-approved GABA-AT

7月10日，中国国家药监局药品审评中心（CDE）官网公示，石药集团巨石生物申报的1.2类新药呼吸道合胞病毒mRNA疫苗获得临床默示许可，用于预防由呼吸道合胞病毒感染引起的下呼吸道疾病。根据石药集团官网公开信息，这是该公司mRNA家族的又一个拳头产品。 呼吸道合胞病毒（RSV）是一种常见的呼吸道病毒，会引起感染者的肺部和呼吸道感染。这种病毒非常普遍，大多数儿童在 2 岁前感染过这种病毒。在成人和老年人以及健康的儿童中，呼吸道合胞病毒的症状较轻。然而，呼吸道合胞病毒可导致一些人群出现严重感染，包括 1岁以内及更小的宝宝、老年人、心肺疾病患者或免疫系统脆弱的人群，需要住院治疗。 mRNA疫苗是科学家们数十年深耕RNA疗法领域的成果之一。2023年，mRNA疫苗科学突破也在2023年获得了诺贝尔奖。当前，mRNA疗法不仅正在被开发用于个性化的癌症治疗，也在更广泛地应用于传染性相关疾病疫苗研发，以及罕见病疗法的开发等。公开资料显示，目前全球范围有两款RSV重组蛋白疫苗获批上市，尚未有RSV mRNA疫苗获批上市。 参考资料： [1]中国国家药监局药品审评中心（CDE）官网.Retrieved

日前，Arcutis Biotherapeutics宣布，美国FDA批准Zoryve（roflumilast，0.15%）乳膏的补充新药申请（sNDA），用于治疗6岁及以上成人和儿童的轻度至中度特应性皮炎（AD）。 该sNDA获得批准的依据是三项3期研究、一项2期剂量范围研究和两项1期药代动力学研究的积极结果。其中INTEGUMENT-1和INTEGUMENT-2是两项相同的平行组、双盲、安慰剂对照3期试验，评估使用每日一次Zoryve（0.15%）乳膏或安慰剂共4周的安全性和有效性，共有1337名6岁及以上的轻度至中度AD患者入组。 INTEGUMENT-1和-2研究均达到了主要终点研究者整体评估（IGA）成功率，定义为在第4周时验证的研究者全球评估-特应性皮炎（vIGA-AD）评分达到清除（0）或几乎清除（1），并从基线改善2个等级（INTEGUMENT-1：Zoryve，32.0% vs安慰剂，15.2%，P<0.0001；INTEGUMENT-2：Zoryve，28.9% vs安慰剂，12.0%，P<0.0001）。在这两项研究中，在第4周时，大约40%的儿童和成人

7月10日，中国国家药监局药品审评中心（CDE）官网公示，明慧基因生物技术有限公司申报的1类创新药抗CD30嵌合抗原受体自体T细胞注射液获批临床，拟开发用于霍奇金淋巴瘤和间变性大细胞淋巴瘤等CD30阳性肿瘤。 CD30存在于多种血液肿瘤中，表达水平较高的有霍奇金淋巴瘤、间变性大细胞淋巴瘤，其他如T细胞淋巴瘤、部分B细胞非霍奇金淋巴瘤等也有这一受体。此前，一些靶向CD30的抗体药物偶联物（ADC）或双特异性抗体已在临床显示出可喜的疗效。 公开资料显示，CD30是CAR-T细胞疗法热门在研靶点之一。除了本次获批临床的这款产品，还有其他靶向CD30的自体CAR-T疗法已经进入临床研究阶段，比如Tessa公司在研的TT11，波睿达生物在研的BRD-01等等。今年4月，《柳叶刀-血液学》还发表一项1期临床研究结果，表明靶向CD30的CAR-T细胞疗法（ATLCAR.CD30）有潜力作为高风险霍奇金淋巴瘤患者在自体HSCT后的巩固治疗。 参考资料： [1]中国国家药监局药品审评中心（CDE）官网.Retrieved July 10，2024, From https://www.cde.org

Encouraging 68% improvement in responder rate results from patients who achieved both the conjunctival staining “sign” and ocular pain “symptom” endpoints from the 240 patient Phase 2 Dry Eye Disease (DED) trial Conjunctival staining and ocular pain represent potential co-primary endpoints to be explored in future trials July 10, 2024 -- OKYO Pharma Limited (NASDAQ: OKYO), a clinical-stage biopharmaceutical company developing innovative ocular therapies for the treatment of inflammatory dry eye

CEO to participate in ARVO SIG panel on oral medications for retinal diseases ZETA-1 Phase 2 clinical trial subset analysis to be presented during the ASRS 42nd Annual Scientific Meeting July 10, 2024 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of patients with retinal and refractive eye disorders, today announced it will participate in the As

July 09, 2024 -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has indicated that it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2, 2024, to review the New Drug Application (NDA) for arimoclomol as an orally delivered, first-in-class treatment for Niemann-Pick disease type C (NPC). According to the notice

– Abiprubart Phase 2b clinical trial in Sjögren’s Disease to evaluate treatment response across biweekly and monthly subcutaneous administrations – – Abiprubart clinical development in Sjögren’s Disease fully funded; company expects to remain cash flow positive on an annual basis – July 09, 2024 -- Kiniksa Pharmaceuticals International, plc (Nasdaq: KNSA) (Kiniksa), a commercial-stage biopharmaceutical company with a pipeline of immune-modulating assets designed to target a spectrum of cardiova

Skyhawk’s SKY-0515 demonstrated dose-dependent huntingtin (HTT) mRNA reduction in healthy volunteers, with 72% reduction at the highest dose tested in the multiple ascending dose study SKY-0515 was generally well tolerated at all doses tested Given these positive topline results, we anticipate dosing in the patient arm of the study in Q3 2024 and initiation of a Phase 2 study early next year July 10, 2024 -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel sma