5-HT2C receptor

欢迎关注凯莱英药闻 （收集周期：9.9-9.20，国内部分为首次申请临床、首次申请上市、首次获批上市的创新药）    国内创新药IND汇总 1、百济神州：BGB-58067片 作用机制：PRMT5抑制剂 适应症：肿瘤 9月10日，百济神州的BGB-58067片的临床试验申请（IND）获CDE受理。BGB-58067是一款PRMT5抑制剂，具有高效力和高选择性，且具有脑渗透性，有望用于非小细胞肺癌和消化道肿瘤等实体瘤。PRMT5 是一种表观遗传酶，参与多种生理过程，包括转录调控、RNA 代谢、核糖体生物合成和细胞周期调控。已有证据表明，PRMT5过度表达可能在多种癌症中起着关键作用，包括B 和T 细胞淋巴瘤、转移性黑色素瘤、神经母细胞瘤、胶质母细胞瘤、生殖细胞瘤、卵巢癌、乳腺癌等。 2、普利药业：注射用PL002 作用机制：—— 适应症：肝癌 9月10日，普利药业的注射用PL002的IND获CDE受理。PL002是一款荧光/磁共振双模态造影剂；双模态造影剂的荧光成像能力可用于肝癌术中荧光导航，而磁共振增强能力可用于在术前判断肝脏代谢能力，推测肿瘤及肝脏对造影剂的摄取情况，提高肿瘤诊断精度，降低术前规划难度。相较于吲哚菁绿，PL002采用独特的钆络合物与荧光分子共价键结合的分子结构设计，这种设计能够让医生在术前通过磁共振成像确定造影剂在病灶中的聚集情况，进而针对患者个体情况来对手术时间进行规划。同时，由于PL002体内更为稳定，同等条件下，在荷瘤小鼠模型中荧光成像效果维持时间长于现有荧光造影剂，具有更宽的手术窗口。 3、广为医药：GW117片 作用机制：MT1/2激动剂;5-HT2C receptor拮抗剂 适应症：抑郁症 9月10日，广为医药的GW117片的IND获CDE受理。GW117是一款抗抑郁药，具有改善情绪心境、调节生物节律、且具有突出安全性。一项治疗成人抑郁症的II期临床试验显示，治疗8周时，采用GW117的各给药组HAM-D17 /MADRS总分较基线的减分值、各给药组HAM-D17/MADRS有效率，均优于安慰剂组。此外，在抑郁情形更严重（HAM-D17基线≥25分）、抑郁核心症状更明显的人群中，GW117各给药组在主要及次要终点指标上，与安慰剂组相比均呈现统计学意义（P＜0.05）。此外，在安全性及依从度方面，GW117各给药组与安慰剂组相当甚至优于安慰剂组。 4、映恩生物：注射用DB-1419 作用机制：靶向PDL1/B7-H3双抗 适应症：肿瘤 9月11日，映恩生物的注射用DB-1419的IND获CDE受理。DB-1419是一款潜在同类首创的B7-H3/PD-L1双抗ADC药物，采用了新型结合和内吞机制，相较于传统ADC疗效更卓越，且能有效克服因单靶点表达下降导致的耐药性问题。临床前研究表明，DB-1419能够杀伤癌细胞并进行T细胞活化，在免疫重构模型中，DB-1419展现出比单特异性B7-H3 ADC更强的肿瘤生长抑制活性。 5、嘉因生物：EXG110注射液 作用机制：—— 适应症：法布雷病 9月12日，嘉因生物的EXG110注射液的IND获CDE受理，拟用于治疗法布雷病。EXG110是一款在研的AAV基因疗法，以重组AVV为载体将治疗基因GLA靶向递送到肝脏和肌肉细胞，使正常GLA基因在肝脏和肌肉细胞中特异、持续表达；此外，产生的α-半乳糖苷酶 A（α-Gal A）分泌到血液循环中，其他细胞可以通过受体摄取。通过开发EXG110一次静脉注射制剂，有望实现高效、持久的疗效。 6、万春医药：普那布林注射用浓溶液 作用机制：GEF-H1激活剂 适应症：中性粒细胞减少症 9月13日，万春医药的普那布林注射用浓溶液的IND获CDE受理。普那布林是一款选择性免疫调节剂，通过促使树突状细胞(DC)成熟，加强抗原递呈作用，以激活抗原特异性T细胞，从而增强机体的免疫功能。该药物可诱导巨噬细胞极化并提高M1型/M2型肿瘤相关巨噬细胞的比值，从而进一步增强了肿瘤微环境中免疫细胞的抗肿瘤活性。研究显示，在多西他赛的基础上加用普那布林，使二线治疗后的PFS显著延长，改善了疾病控制时间；无论是客观缓解率（ORR）、无进展生存（PFS）和总生存（OS）数据，都达到具有统计学意义的阳性结果。此外，普那布林可以促进造血干细胞和祖细胞（HSPC）成熟，在骨髓功能保护方面也显示出卓越的临床疗效，与多西他赛联用进一步降低了化疗毒性、发挥保护中性粒细胞的作用。 7、仁景生物：RG002注射液 作用机制：—— 适应症：肿瘤 9月13日，仁景生物的RG002注射液的IND获CDE受理。RG002是仁景生物开发的一款mRNA疫苗，采用的递送系统递送效率高、体内安全性优异。临床前研究表明，RG002单药能诱导强烈的特异性细胞免疫和肿瘤细胞杀伤作用，有效抑制HPV相关肿瘤生长；与免疫检查点抑制剂等抗肿瘤药物联用表现出良好的协同、增效作用。2023年11月，该疫苗的IND获FDA受理，用于治疗人乳头瘤病毒（HPV）16型和/或18型相关的2级或3级宫颈上皮内瘤变（CIN2/3），是全球首个被批准开展用于治疗HPV 16型和/或18型相关的CIN2/3临床试验的mRNA-LNP治疗性疫苗。 8、瑞宏迪医药：外用RGL-2102 作用机制：—— 适应症：肿瘤 9月13日，瑞宏迪医药的外用RGL-2102的IND获CDE受理。RGL-2102是公司自主研发的由脂质体 (LNP) 包载的mRNA，给药后可在体内翻译得到目的人源蛋白，促进下肢缺血性疾病患者血管新生，建立侧枝循环，改善缺血区域的血流灌注。目前，公司建立了具有自主知识产权的、完善的mRNA纳米脂质体的研发体系，以及符合法规要求的中试生产车间，具备mRNA原液和LNP制剂成品的生产能力。 9、必贝特医药：BEBT-507注射液 作用机制：—— 适应症：真性红细胞增多症 9月14日，必贝特医药的BEBT-507注射液的IND获CDE受理。BEBT-507是公司首款siRNA药物，旨在通过调节体内铁水平，治疗真性红细胞增多症等铁代谢异常疾病。临床前研究结果显示，BEBT-507能长期高效地抑制靶点活性，在动物模型中表现出显著和持久的药效以及良好的安全性。必贝特医药专注肿瘤、自身免疫性疾病、代谢性疾病等重大疾病领域，已经建立了一体化的、具有全球知识产权的小核酸药物研发平台，涵盖siRNA药物的设计与筛选、化学修饰、体内外药效评估、药代动力学和安全评价体系等多个环节，并成功建立了靶向肝脏的GalNA siRNA偶联GSOC和GDOC递送系统、靶向肾脏和中枢神经系统（CNS）等不同器官的多肽siRNA偶联（POC）递送系统，持续开发临床急需的全球首创药物（First-in-Class）和针对未满足临床需求的创新药物。 10、特科罗生物：TDM-180935软膏 作用机制：JAK1/Tyk2抑制剂 适应症：特应性皮炎/湿疹 9月19日，特科罗生物的TDM-180935软膏的IND获CDE受理。TDM-180935是一个高效的JAK1/Tyk2小分子抑制剂；临床前研究显示：功能性细胞测试表明，TDM-180935能有效抑制与导致AD有关的角质细胞以及T细胞介导的致病通路。在多种动物疾病模型的试验中，TDM-180935显示出有效性，且非常适合外用涂抹。在大鼠和小型猪实验中，TDM-180935显示出良好的安全性和毒代动力学特征。I期临床试验显示，外用TDM-180935显示出很好的耐受性，没有发生与药物相关的安全性问题；采用不同剂量TDM-180935每天涂药1次，连续使用4周，没有出现或仅有个别受试者出现极低的系统性药物暴露。 11、Mirum Pharmaceuticals：Volixibat胶囊 作用机制：ISBT抑制剂 适应症：胆管炎 9月19日，Mirum 公司的Volixibat胶囊的IND获CDE受理。Volixibat是一种口服、吸收率极低的药物，旨在选择性抑制回肠胆汁酸转运体(IBAT)；该药物通过抑制 IBAT 来阻断胆汁酸的循环，从而减少全身和肝脏中的胆汁酸，为成人胆汁淤积性疾病的治疗提供一种新方法。2b 期中期分析结果显示，在原发性胆汁性胆管炎 (PBC) 和原发性硬化性胆管炎 (PSC) 患者中，瘙痒症状相较于安慰剂有统计学显著改善（-3.82，p<0.0001），75%服用Volixibat的患者血清胆汁酸减少超过 50%；此外，与安慰剂相比，服用Volixibat 16 周后疲劳症状有显著改善。在安全性上，研究未观察到新的安全信号，最常见的不良事件是腹泻（77%），所有病例均为轻度至中度，且大多为短暂性；肝脏生物标志物没有出现有临床意义的变化。 12、柯君医药：注射用苯磺酸CG-0255 作用机制：P2Y12抑制剂 适应症：脑卒中 9月19日，柯君医药的注射用苯磺酸CG-0255的IND获CDE受理。CG-0255是基于氯吡格雷活性成分设计的巯基前药，既可以口服，也可以静脉注射。和氯吡格雷通过CYP多步氧化代谢形成活性成分途径不一样，CG-0255通过脂酶一步水解代谢，可以高效、快速地在体内转化为活性成分。根据2024欧洲心脏病学会年会（ESC）披露的数据，药代动力学和药效学结果显示：（1）给药后24小时IPA离散系数CV：CG-0255 2 mg组与氯吡格雷300 mg：11% vs 116%。（2）起效时间：注射剂型<15分钟，口服剂型<30分钟。（3）血小板聚集抑制率在高剂量组注射和口服组都接近100%。其中口服剂型2 mg低剂量组的血小板聚集抑制率约40%，和氯吡格雷300 mg组药效相当。（4）CG-0255不经过CYP代谢，减少了药物之间的相互作用。（5）CG-0255前药设计高效，只需氯吡格雷剂量的1%或者更低，即可达到相似的血小板聚集抑制率。（6）注射剂型可以一次推注给药，不需要持续滴注，且药效持续超过一天。    国内创新药NDA汇总 1、禾元生物：植物源重组人血清白蛋白注射液 作用机制：重组albumin 适应症：低蛋白血症 9月12日，禾元生物的植物源重组人血清白蛋白注射液的上市申请获CDE受理并纳入优先审评审批程序。植物源重组人血清白蛋白注射液是公司利用国际先进的植物分子医药技术研发生产；由于我国人血清白蛋白完全从血浆提取，因血浆短缺，长期依赖进口；本产品获批后有望拜托进口依赖。禾元生物是一家专门从事植物源重组蛋白表达技术研究与产品开发的国家高新技术企业，拥有技术力量雄厚的研发队伍和完备先进的蛋白纯化工艺研究设施，具有一系列独立自主核心技术和知识产权保护；目前已建立了“一个独特植物表达体系、两个技术平台”，即水稻胚乳细胞蛋白质表达平台（OryzHiExp）和蛋白质纯化平台（OryzPur）。 2、恒瑞医药：注射用瑞康曲妥珠单抗 作用机制：靶向HER2 ADC 适应症：非小细胞肺癌 9月14日，恒瑞医药的注射用瑞康曲妥珠单抗（SHR-A1811）的上市申请获CDE受理。SHR-A1811是一款靶向HER2的ADC药物，利用抗体特异性识别并结合肿瘤细胞后，精准释放携带的细胞毒性药物，实现了对肿瘤细胞的精准打击与高效清除。一项在HER2过表达、扩增或突变的晚期NSCLC受试者中的安全性、耐受性、药代动力学及有效性的I/II期临床研究显示，全剂量组患者中位无进展生存期（mPFS）约9.5个月，经研究者评估确认的客观缓解率（ORR）为38.1%，疾病控制率（DCR）高达90%以上，中位缓解持续时间（DoR）为10.3个月；4.8mg/kg剂量组中位PFS为8.4个月，超过40%的患者在接受治疗后达到了疾病缓解，ORR为41.9%，DCR为95.3%，中位DoR为13.7个月，充分证明了SHR-A1811在HER2突变肺癌治疗中的潜力和价值。 3、奥默/仙琚：奥美克松钠注射液 作用机制：—— 适应症：肌松药 9月18日，奥默/仙琚共同开发的奥美克松钠注射液的上市申请获CDE受理。奥美克松钠是新一代靶向性肌松拮抗剂，拟用于逆转罗库溴铵诱导的神经肌肉传导阻滞作用。与阳性对照药舒更葡糖钠头对头比较的两项III期临床试验结果显示：奥美克松钠疗效明确，3分钟内可以快速拮抗中度和深度的神经肌肉传导阻滞；药效稳定性更好，再箭毒化和肌松残余发生率更低，与阳性对照药舒更葡糖钠相比具有显著的优势（P<0.05)。同时，奥美克松钠在特别关注的不良反应发生率，如过敏反应、心动过缓、喉痉挛/支气管痉挛等方面显著降低，与阳性对照药舒更葡糖钠相比具有显著的优势（P<0.05)。 4、恒瑞医药：瑞拉芙普-α注射液 作用机制：抗PD-L1/TGF-βRII双功能融合蛋白 适应症：胃及胃食管结合部腺癌 9月20日，恒瑞医药的瑞拉芙普-α注射液的上市申请获CDE受理，用于与氟尿嘧啶类和铂类药物联合一线治疗局部晚期不可切除、复发或转移性胃及胃食管结合部腺癌。瑞拉芙普-α是一款抗PD-L1/TGF-βRII双功能融合蛋白，可以促进效应性T细胞的活化，同时还可有效改善肿瘤微环境中的免疫调节作用，最终有效促进免疫系统对于肿瘤细胞的杀伤。 2020年11月，恒瑞医药与韩国DONG-A ST公司（以下简称“东亚公司”）达成协议，以1.3927亿美元交易总额将SHR-1701项目许可给韩国东亚公司，同时获得韩国东亚公司的销售分成。韩国东亚公司将获得SHR-1701在韩国的独家临床开发、生产和市场销售的权利，被许可进行研发和销售SHR-1701用于所有人类疾病。 近期，公司在2024年欧洲肿瘤内科学会（ESMO）大会公布了SHR-1701联合化疗对比安慰剂联合化疗用于HER2阴性胃或胃食管结合部腺癌(G/GEJA)晚期一线的3期临床研究数据，结果显示：（1）PD-L1 CPS≥5人群中，SHR-1701组和安慰剂组的中位生存期(mOS)分别为16.8个月和10.4个月，达到统计学差异。（2）ITT人群中，SHR-1701组和安慰剂组mOS分别为15.8个月和11.2个月，达到统计学差异。（3）PD-L1 CPS≥5人群中，SHR-1701组和安慰剂组的mPFS分别为7.6个月和5.5个月。SHR-1701组和安慰剂组确认的ORR分别为56.5%和32.7%，差异为23.8%。DoR分别为10.2个月和5.1个月。（4）ITT人群中，SHR-1701组和安慰剂组的mPFS分别为7.0个月和5.5个月。SHR-1701组和安慰剂组确认的ORR分别为53.4%和32.8%，差异为20.6%。DoR分别为8.5个月和5.3个月。    国内创新药上市获批 1、大冢制药：泊那替尼片 作用机制：Bcr-Abl抑制剂 适应症：白血病 9月9日，大冢制药的泊那替尼片的上市申请获国家药品监督管理局（NMPA）批准，用于：（1）既往用药耐药或不耐受的慢性髓性白血病（CML）；（2）复发或难治性费城染色体阳性（Ph+）急性淋巴细胞白血病（ALL）；（3）T315I阳性CML或T315I Ph+ ALL。泊那替尼（Ponatinib）是一款第三代Bcr-Abl抑制剂，对T315I突变型Bcr-Abl激酶具有高效特异性抑制作用，可有效解决因激酶突变（T315I）引起的耐药性。该药物于2012年12月获FDA批准用于对酪氨酸激酶抑制药耐药或不能耐受的慢性髓性白血病（CML）患者。2024 年3 月，FDA 加速批准Ponatinib（商品名：Iclusig）的补充新药申请（sNDA），即联合化疗一线治疗新诊断费城Ph+ ALL成年患者；研究显示，Ponatinib 联合低强度化疗实现了更高的微小残留病（MRD）阴性完全缓解率，达到了主要终点，同时也没有观察到新的安全信号。 2、石药/康诺亚：司普奇拜单抗注射液 作用机制：IL-4Rα单抗 适应症：特应性皮炎 9月12日，石药/康诺亚共同开发的司普奇拜单抗注射液获NMPA批准上市，用于治疗外用药控制不佳或不适合外用药治疗的成人中重度特应性皮炎。司普奇拜单抗是一款靶向IL-4Rα单抗，通过阻断IL-4和IL-13与IL-4Rα受体的结合，抑制IL-4和IL-13引起的下游炎症因子的释放、蛋白表达及炎症细胞活性，治疗炎症反应过度诱发的特应性皮炎（AD）。此外，公司还在探索其用于青少年AD、结节性痒疹、哮喘、慢性阻塞性肺病等多个适应症。 该药物获批是基于一项在国内迄今为止，已完成的皮肤科受试者样本量最大的随机对照试验研究，结果显示：（1）首剂单药治疗1天，即可快速缓解瘙痒症状；（2）治疗2周，对全身各部位皮损均有强效改善作用；单药治疗52周，可实现持续改善皮损的“双9可达”治疗目标——即超过九成患者实现湿疹面积和严重指数程度改善75%以上（EASI-75），近八成患者实现湿疹面积和严重指数程度改善90%以上（EASI-90）。（3）单药治疗52周，PP-NRS较基线下降≥4应答率高达67.3%，（4）单药治疗DLQI、POEM评分较基线变化率持续改善，显著提高患者生活质量。（5）治疗52周复发率仅0.9%，停药8周复发率仅0.9%，结膜炎发生率仅5.3%。 全球新药III期临床汇总 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

LA JOLLA, Calif.--( BUSINESS WIRE )-- Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced that the FDA has granted Rare Pediatric Disease designation and Orphan Drug designation for its investigational drug bexicaserin for the treatment of Dravet syndrome. “ We are thrilled to receive both Rare Pediatric Disease and Orphan Drug designations for bexicaserin in Dravet syndrome. As the first company to receive Breakthrough Therapy designation (BTD) for the treatment of seizures associated with Developmental and Epileptic Encephalopathies (DEEs), we appreciate the regulatory support that BTD provides as we continue on our journey to study bexicaserin as a potential treatment option for DEE patients with significant unmet medical needs. Receiving Orphan Drug designation in Dravet syndrome provides us with further regulatory support as we progress the development of bexicaserin. Additionally, the granting of the Rare Pediatric Disease designation provides us with an opportunity to receive a Priority Review Voucher which can be used for priority review of another Longboard compound or monetized,” stated Dr. Randall Kaye, Longboard’s Chief Medical Officer. “ We appreciate the FDA’s ongoing support and look forward to initiating our global Phase 3 DEEp program, starting with DEEp SEA in Dravet syndrome in the coming weeks.” About Rare Pediatric Disease Designation and Priority Review Vouchers (PRVs) The Rare Pediatric Disease program aims to incentivize drug development for rare pediatric diseases. Under this program, a sponsor who receives an approval for a drug or biological product for a rare pediatric disease may qualify for a Priority Review Voucher (PRV) that can be redeemed to receive priority review for a different product. The sponsor may also transfer or sell the PRV to another sponsor. About Orphan Drug Designation Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. The FDA has authority to grant Orphan Drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan Drug designation qualifies sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees, and the potential for seven years of market exclusivity after approval. ABOUT LONGBOARD PHARMACEUTICALS Longboard Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases. Longboard is working to advance a portfolio of centrally acting product candidates designed to be highly selective for specific G protein-coupled receptors (GPCRs). Longboard’s small molecule product candidates are based on more than 20 years of GPCR research. Longboard plans to advance bexicaserin (LP352), an oral, centrally acting 5-hydroxytryptamine 2C (5-HT2C) receptor superagonist, with no observed impact on 5-HT2B and 5-HT2A receptor subtypes, into a global Phase 3 program. Earlier this year, Longboard reported positive topline data from a Phase 1b/2a clinical trial (the PACIFIC Study) evaluating bexicaserin in participants ages 12 to 65 years old with Developmental and Epileptic Encephalopathies (DEEs), including Lennox-Gastaut syndrome, Dravet syndrome and other DEEs. Longboard is also evaluating LP659, an oral, centrally acting, sphingosine-1-phosphate (S1P) receptor subtypes 1 and 5 modulator, which is in development for the potential treatment of rare neuroinflammatory conditions. Longboard recently completed a Phase 1 single-ascending dose (SAD) clinical trial for LP659 in healthy volunteers. Bexicaserin and LP659 are investigational compounds that are not approved for marketing by the FDA or any other regulatory authority. FORWARD-LOOKING STATEMENTS Certain statements in this press release are forward-looking statements that involve a number of risks and uncertainties. In some cases, you can identify forward-looking statements by words such as “focus”, “potential”, “opportunity”, “can”, “look forward”, “aim”, “may”, “working to”, “designed to”, “plan”, or the negative, plural or other tenses of these words, references to future dates or time periods, or other comparable language, and they may include, without limitation, statements about the following: Longboard’s product candidates and programs (including bexicaserin’s potential, development, advancement into a global Phase 3 program, and Breakthrough Therapy, Rare Pediatric Disease and Orphan Drug designations and their potential benefits, including eligibility for a Priority Review Voucher (PRV)), plans, focus and work. For such statements, Longboard claims the protection of the Private Securities Litigation Reform Act of 1995. Actual events or results may differ materially from Longboard’s expectations. Factors that could cause actual results to differ materially from those stated or implied by Longboard’s forward-looking statements include, but are not limited to, the following: the rare pediatric disease PRV program will begin to sunset after September 30, 2024, and if the program is not reauthorized, we will only be eligible to receive a PRV if bexicaserin receives marketing approval by September 30, 2026; the standards for Breakthrough Therapy, Rare Pediatric Disease and Orphan Drug designations are not the same as the standard for drug approval, the clinical evidence supporting Breakthrough Therapy designation is preliminary, and not all drugs designated as Breakthrough Therapies ultimately will be shown to have substantial improvement over available therapies; the FDA may later decide to rescind a designation if it determines the designation is no longer supported by subsequent data; Longboard’s product candidates are in a lengthy research and development process, the timing, manner and outcome of research, development and regulatory review is uncertain, and Longboard’s product candidates, including bexicaserin and LP659, may not advance in research or development or be approved for marketing; results of clinical trials and other studies are subject to different interpretations and may not be predictive of future results; topline or interim data may not accurately reflect the complete results of a particular study or trial and remain subject to audit, and final data may differ materially from topline or interim data; enrolling participants in clinical trials is competitive and challenging; risks related to unexpected or unfavorable new data; nonclinical and clinical data is voluminous and detailed, and regulatory agencies may interpret or weigh the importance of data differently and reach different conclusions than Longboard or others, request additional information, have additional recommendations or change their guidance or requirements before or after approval; risks related to Longboard’s limited operating history, financial position and need for additional capital; Longboard will need additional managerial and financial resources to advance all of its programs, and you and others may not agree with the manner Longboard allocates its resources; risks related to the development and commercialization of Longboard’s product candidates; risks related to relying on licenses or collaborative arrangements; other risks related to Longboard’s dependence on third parties; competition; product liability or other litigation or disagreements with others; government and third-party payor actions, including relating to reimbursement and pricing; risks related to regulatory compliance; and risks related to Longboard’s and third parties’ intellectual property rights. Additional factors that could cause actual results to differ materially from those stated or implied by Longboard’s forward-looking statements are disclosed in Longboard’s filings with the Securities and Exchange Commission (SEC). These forward-looking statements represent Longboard’s judgment as of the time of this release. Longboard disclaims any intent or obligation to update these forward-looking statements, other than as may be required under applicable law.

Bright Minds Biosciences announced the initiation of Phase 2 clinical trial of BMB-101 in a group of drug-resistant epilepsies and will discuss the details of the BREAKTHROUGH clinical trial on September 25th. The event will feature Epilepsy KOLs to discuss the unmet needs for epilepsy treatment. BMB-101 is the first 5-HT2C agonist in clinical studies designed to exclusively target therapeutic pathways (via G-protein) without receptor desensitization. NEW YORK, Sept. 19, 2024 (GLOBE NEWSWIRE) -- Bright Minds Biosciences Inc. (NASDAQ / CSE: DRUG) announces the Key Opinion Leader (KOL) event with leading experts in epilepsy research and treatment. The event will include Dr. Dennis Dlugos, Dr. Joe Sullivan, and Dr. Jo Sourbron, who will provide critical insights into the evolving landscape of drug-resistant seizures and the unmet needs in epilepsy care. These thought leaders will explore the scientific innovations driving the Phase 2 BREAKTHROUGH clinical trial design announced last week and highlight the potential for novel treatments. Featured KOL speakers Dr. Dennis J. Dlugos , MD, MSCE, is a professor of neurology and pediatrics at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine. He is the Director of the Section of Clinical Neurophysiology and the Epilepsy/Clinical Neurophysiology Fellowship. He holds the Tristram C. Colket, Jr. Endowed Chair in Pediatric Neurology. Dr. Dlugos is a member of the American Academy of Pediatrics, the American Academy of Neurology, the American Epilepsy Society, the American Clinical Neurophysiology Society, and the Child Neurology Society. Dr. Dlugos is a frequent lecturer locally and at national medical meetings on topics related to pediatric neurology, epilepsy, and electroencephalograms. He has published papers in journals such as Neurology, Epilepsia, Archives of Neurology, Pediatric Neurology, and Journal of Child Neurology . Dr. Joseph Sullivan , a pediatric neurologist, serves as director of the UCSF Pediatric Epilepsy Center, where he specializes in evaluating and caring for children with epilepsy, particularly refractory epilepsy (in which medications fail to control seizures). He has special interests in Dravet syndrome and PCDH19-related epilepsy (two genetic forms of the condition) as well as evaluating children for epilepsy surgery. Dr. Sullivan is a member of the American Epilepsy Society and serves on boards for several organizations. His roles include chair of the PCDH19 Alliance’s scientific advisory board, member of the Epilepsy Foundation of Northern California’s board of directors, and member of the Dravet Syndrome Foundation’s medical advisory board and board of directors. Dr. Jo Sourbron , MD, PhD, MPharm, is a physician scientist with a clinical practice at the University Hospitals of Ghent (UZ Ghent, Belgium) and a postdoctoral researcher at the University of Leuven (UZ/KU Leuven, Belgium). Dr. Sourbron has a special interest in genetic forms of epilepsy and clinical trials for drug-resistant forms of epilepsy. He has been a preclinical researcher for over a decade, focused on finding novel serotonergic (5-HT) compounds for drug-resistant epilepsies. Dr. Sourbron has explored the efficacy and safety of a variety of anti epileptic drugs including, 5-HT2 agonists like fenfluramine and lorcaserin in various animal models. For the past five years, he has been particularly involved in research involving epilepsy with eyelid myoclonia (EEM) with prominent photic induction (Sunflower syndrome). Concomitantly, he was involved in the pilot clinical trial of fenfluramine in Sunflower syndrome patients. Finally, he participated in several other clinical trials for drug-resistant epilepsies, such as cannabidiol in the treatment of drug-resistant epileptic disorders. IMPORTANT INFORMATION: Registration Details: Advance registration is required to participate in the webcast. If you cannot join live, a replay will be available. Register at Livestorm . Q&A Instructions: To ask a question during the live Q&A session, please submit your request through the portal below the webcast player when you register or send an email to info@brightmindsbio.com . About BMB-101 BMB-101 is a novel scaffold 5-HT2C Gq-protein biased agonist developed using structure-based drug design. It was explicitly designed for chronic treatment of neurological disorders where tolerance and drug resistance are common issues. Biased agonism at the 5-HT2C receptor is one of its key features and adds another layer of functional selectivity within a well-validated target. BMB-101 works exclusively via the Gq-protein signaling pathway and avoids beta-arrestin activation, which is crucial to minimize the risk of receptor desensitization and tolerance development. This provides a novel mechanism, anti-epileptic drug designed to provide sustained seizure relief in hard-to-treat patient populations. In preclinical studies, BMB-101 has demonstrated efficacy in animal models of Dravet Syndrome and numerous models of generalized seizures. In Phase 1 clinical studies, BMB-101 was given to 64 healthy volunteers in a Single Ascending Dose (SAD), Multiple Ascending Dose (MAD) and food-effects study. BMB-101 was demonstrated to be safe and well tolerated at all doses. No Serious Adverse Events (SAEs) were observed, and Adverse Events (AEs) were mild in nature and in line with on-target effects for serotonergic drugs. An extensive target-engagement study was conducted using both fluid biomarkers (transient prolactin release) and physical biomarkers (Quantitative Electroencephalogram, qEEG). Both methods confirmed robust central target engagement. A qEEG signature typical for anti-epileptic drugs was observed, with a selective depression of EEG power at frequencies observed during epileptic seizures. Furthermore, a potentiation of frontal gamma-power was observed in this study which could indicate the potential for improved cognition. About Bright Minds Biosciences Bright Minds Biosciences is a biotechnology company developing innovative treatments for patients with neurological and psychiatric disorders. Our pipeline includes novel compounds targeting key receptors in the brain to address conditions with high unmet medical need, including epilepsy, depression, and other CNS disorders. Bright Minds is focused on delivering breakthrough therapies that can transform patients’ lives. Bright Minds Biosciences has developed a unique platform of highly selective serotonergic agonists exhibiting selectivity at different serotonergic receptors. This has provided a rich portfolio of NCE programs within neurology and psychiatry. Forward-Looking Statements This news release contains “forward-looking information”. Often, but not always, forward-looking statements can be identified by the use of words such as “plans”, “expects”, “is expected”, “budget”, “scheduled”, “estimates”, “forecasts”, “intends”, “anticipates”, or “believes” or variations (including negative variations) of such words and phrases, or state that certain actions, events or results “may”, “could”, “would”, “might” or “will” be taken, occur or be achieved. Forward-looking statements in this news release include design, progress, and completion of the BREAKTHROUGH Study, future clinical development of BMB-101, and future intended use or therapeutic benefit of BMB-101 to treat refractory epilepsy disorders. A variety of factors, including known and unknown risks, many of which are beyond our control, could cause actual results to differ materially from the forward-looking information in this news release. These factors include the company’s financial position and operational runway, regulatory risk to operating in the pharmaceutical industry, and inaccuracies related to the assumption made by management relating to general availability of resources required to operate the studies noted in this news release. Additional risk factors can also be found in the Company’s public filings under the Company’s SEDAR+ pro Forward-looking statements contained herein are made as of the date of this news release and the Company disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or results or otherwise. There can be no assurance that forward-looking statements will prove to be accurate, as actual results and future events could differ materially from those anticipated in such statements. The Company undertakes no obligation to update forward-looking statements if circumstances, management’s estimates or opinions should change, except as required by securities legislation. Accordingly, the reader is cautioned not to place undue reliance on forward-looking statements. The Canadian Securities Exchange has neither approved nor disapproved the information contained herein and does not accept responsibility for the adequacy or accuracy of this news release. Contact Information Alex Vasilkevich Chief Operating Officer Bright Minds Biosciences Inc. Phone: (414)7316422 Email: alex@brightmindsbio.com Website: