BPGbio Inc

BOSTON--( BUSINESS WIRE )-- BPGbio, Inc. , a leading biology-first, AI-powered clinical-stage biopharma company focused on mitochondrial biology and protein homeostasis, today announced that it will co-present two pivotal studies with Stanford Medicine’s Department of Neurology at the Society for Neuro-Oncology (SNO) 2024 Annual Meeting, taking place November 21-24, 2024, in Houston, Texas. The presentations will highlight BPM31510 and BRG399—potential glioblastoma multiforme (GBM) therapies that were identified by the company’s proprietary NAi Interrogative Biology® Platform , a causal AI-powered system designed to reveal hidden cause-and-effect relationships within patient biology. The first study, titled “Optimizing brain cancer therapy: balancing tumor eradication and normal tissue preservation with BPM31510,” explores BPM31510’s superior ability to preserve healthy cells compared to standard cancer treatments in mouse, rat, and human glioblastoma cell lines. The study demonstrates BPM31510’s ability to effectively target and diminish cancer cells while selectively allowing the growth of healthy brain cells, an effect that standard treatments fail to achieve. Researchers also showed that the BPM31510 significantly improved survival in glioma implanted mice and rats compared to the control group, mirroring the results from the ongoing phase 2b trial on BPM31510 for GBM and highlighting BPM31510's potential in brain cancer therapy. The second study, titled “Improved anti-glioblastoma efficacy by BRG399, a novel oral microtubule binding agent,” presents findings on BRG399’s anti-cancer activity. BRG399, a novel pan-cancer therapy, works by disrupting the cell division process in both solid and liquid tumors. BRG399 was also found to induce an immune memory response, protecting against tumor recurrence. The study confirmed that BRG399 can cross into the brain, target tumor cells, and improve survival in rat glioma models. “Standard cancer treatments that focus solely on destroying tumors often harm healthy tissue in the process,” said Seema Nagpal, M.D. , principal investigator of the BPM31510 GBM phase 2b trial and Clinical Professor of Neurology and Neurological Sciences at Stanford Medicine. “These new treatments are taking novel targeted approaches on glioma cancer cells and we are excited to share the encouraging results from our studies with our peers and industry researchers.” “Our ongoing clinical and preclinical trials for BPM31510 and BRG399 are continuously deepening our understanding of these promising therapies,” said Niven R. Narain, Ph.D. , President and CEO of BPGbio. “By putting biology first, our NAi Platform has enabled us to optimize every aspect of these therapies, from dosing and timing to patient selection and potential indication expansion opportunities.” Poster Presentation Details: Optimizing brain cancer therapy: balancing tumor eradication and normal tissue preservation with BPM31510 Date and Time: November 22, 2024, 7:30 p.m. - 9:30 p.m. CST Location: Hall B3, George R. Brown Convention Center, Houston, Texas Presenter: Abbas Khojasteh, Ph.D. Abstract Number: DDDR-13 Improved anti-glioblastoma efficacy by BRG399, a novel oral microtubule binding agent Date and Time: November 22, 2024, 7:30 p.m. - 9:30 p.m. CST Location: Hall B3, George R. Brown Convention Center, Houston, Texas Presenter: Stephane Gesta, Ph.D. Abstract Number: DDDR-14 About BPM31510 BPM31510IV is BPGbio’s lead candidate in late-stage development for aggressive solid tumors such as glioblastoma multiforme (GBM) and pancreatic cancer . Other topical and oral formulations of the investigational agent are also being developed as a potential treatment for several rare diseases. The compound has demonstrated a tolerable safety profile and shown potential clinical benefits across multiple disease indications. Validated by BPGbio’s NAi Interrogative Biology platform , BPM31510 induces a hallmark shift in the tumor microenvironment (TME) by modulating mitochondrial oxidative phosphorylation in aggressive tumors, leading to cancer cell death. In many mitochondrial diseases, restoring CoQ 10 levels can overcome the effect of mutations in genes that lead to mitochondrial dysfunction. BPM31510 has been granted Orphan Drug Designation by the FDA for GBM, pancreatic cancer, and epidermolysis bullosa (EB), as well as Rare Pediatric Disease Designation for primary CoQ10 deficiency and EB. About BRG399 BRG399 is a BPGbio-developed candidate being studied for its therapeutic potential as a treatment for solid and liquid tumor cancers as well as diseases associated with inflammation. This experimental drug, a first-in-class, anti-mitotic agent with broad-spectrum anti-cancer activity and favorable pharmacological properties for clinical testing, is being designed for oral delivery. BRG399 is leading the new oncology drug pipeline for BPGbio which includes drug candidates uniquely targeting E2 enzymes. About BPGbio BPGbio is a leading biology-first AI-powered clinical stage biopharma focused on mitochondrial biology and protein homeostasis. The company has a deep pipeline of AI-developed therapeutics spanning oncology, rare disease and neurology, including several in late-stage clinical trials. BPGbio’s novel approach is underpinned by NAi , its proprietary Interrogative Biology Platform, protected by over 400 US and international patents; one of the world’s largest clinically annotated non-governmental biobanks with longitudinal samples; and exclusive access to the most powerful supercomputer in the world. With these tools, BPGbio is redefining how patient biology can be modeled using bespoke Bayesian AI specifically designed for solving large-scale biology challenges. Headquartered in greater Boston, the company is at the forefront of a new era in medicine, combining biology, multi-modal data, and AI to transform the way we understand, diagnose, and treat disease. For more information, visit www.bpgbio.com .

BOSTON--( BUSINESS WIRE )-- BPGbio, Inc., a leading biology-first, AI-powered clinical-stage biopharma company focused on mitochondrial biology and protein homeostasis, today announced that it will present three scientific posters at the Society for Immunotherapy of Cancer (SITC) 2024 Annual Meeting, taking place November 6-10, 2024, in Houston, Texas. The posters will highlight the latest research on the company’s drug candidates BPM31510 and BRG399 in the immuno-oncology space, providing further validation and paving the way for new potential therapeutic strategies in cancer treatment. In the first study, titled “The Anti-tumor Response of BPM31510 is Associated with Immune Cell Regulation in the Tumor Microenvironment,” researchers showed that BPM31510 significantly increases reactive oxygen species (ROS) levels in cancer cells, leading to cell death across multiple cancer types. They also identified that, BPM31510 can boost the activity of cytotoxic tumor-infiltrating lymphocytes and reduce markers of T cell exhaustion. This dual action gives it the potential to be especially effective in treating "immunologically cold" tumors, such as glioblastoma and pancreatic cancer. The second study, titled “BRG399, a Novel Oral Microtubule Binding Agent, Induces Tumor Regression and Immune Memory in an Orthotopic Glioblastoma Rat Model,” found that BRG399 treatment leads to glioblastoma tumor regression, with 100% survival in treated rats. BRG399 also induces an immune memory response, preventing the recurrence of tumors when surviving rats are re-challenged with glioma cancer cells. The third study, titled, “BRG399, a Novel Oral Microtubule Binding Agent, Exhibits Immune-Modulatory Properties Enhancing Anti-Tumor Responses,” showed that BRG399 alters the immune cell composition within the tumor microenvironment and blood. BRG399 also reduced markers of T cell exhaustion, suggesting that it can reinvigorate immune responses against tumors. These findings suggest that BRG399 should be further investigated as a potential component of cancer therapy, particularly when combined with immunotherapies, as it can both kill cancer cells and enhance immune activity. “These compounds – BPM31510 and BRG399 – push the boundaries of what's possible in cancer therapy, showing that we can not only attack the tumor but also empower the body's immune system to keep fighting long after the treatment ends,” said Stephane Gesta, Ph.D. , VP, Discovery and Translational Biology at BPGbio. “As we advance BRG399 through preclinical trials and approach the completion of BPM31510's Phase 2b study, we will continue leveraging our NAi Interrogative Biology Platform to gain additional insight for exploring new therapeutic opportunities for other diseases.” Poster Presentation Details: The Anti-Tumor Response of BPM31510 Is Associated with Immune Cell Regulation in the Tumor Microenvironment Date and Time: November 8, 2024, 1:00 p.m. CST Location: Exhibit Halls A B, George R. Brown Convention Center, Houston, Texas Presenter: Maria-Dorothea Nastke, Ph.D. Abstract Number: 1312 BRG399, a Novel Oral Microtubule Binding Agent, Induces Tumor Regression and Immune Memory in an Orthotopic Glioblastoma Rat Model Date and Time: November 9, 2024, 2:00 p.m. CST Location: Exhibit Halls A B, George R. Brown Convention Center, Houston, Texas Presenter: Maria-Dorothea Nastke, Ph.D. Abstract Number: 1313 BRG399, a Novel Oral Microtubule-Binding Agent, Exhibits Immune-Modulatory Properties Enhancing Anti-Tumor Responses Date and Time: November 9, 2024, 3:00 p.m. CST Location: Exhibit Halls A B, George R. Brown Convention Center, Houston, Texas Presenter: Kaila Bennett, Ph.D. Abstract Number: 1284 About BPM31510 BPM31510 is BPGbio’s lead candidate in late-stage development for glioblastoma multiforme (GBM) and pancreatic cancer . The compound has demonstrated a tolerable safety profile and shown potential clinical benefit in both populations. The mechanism of action of BPM31510 was first validated by data from BPGbio’s NAi Interrogative Biology platform , which suggested that there is a hallmark shift in the tumor microenvironment (TME) induced by BPM31510 which modulates mitochondrial oxidative phosphorylation in highly aggressive tumors. BPGbio has received FDA's Rare Pediatric Disease Designation for BPM31510IV for primary CoQ10 deficiency and BPM31510T for Epidermolysis Bullosa (EB) . About BRG399 BRG399 is a BPGbio-developed candidate being studied for its therapeutic potential as a treatment for solid and liquid tumor cancers as well as diseases associated with neutrophil-driven inflammation. This experimental drug, a first-in-class, anti-tubulin agent with broad-spectrum anti-cancer activity and favorable pharmacological properties including oral bioavailability for clinical testing. BRG399 is leading the new oncology drug pipeline for BPGbio among other drug candidates which uniquely target the colchicine binding pocket in tubulin. About BPGbio BPGbio is a leading biology-first AI-powered clinical stage biopharma focused on mitochondrial biology and protein homeostasis. The company has a deep pipeline of AI-developed therapeutics spanning oncology, rare disease and neurology, including several in late-stage clinical trials. BPGbio’s novel approach is underpinned by NAi , its proprietary Interrogative Biology Platform, protected by over 400 US and international patents; one of the world’s largest clinically annotated non-governmental biobanks with longitudinal samples; and exclusive access to the most powerful supercomputer in the world. With these tools, BPGbio is redefining how patient biology can be modeled using bespoke causal AI specifically designed for solving large-scale biology challenges. Headquartered in greater Boston, the company is at the forefront of a new era in medicine, combining biology, multi-modal data, and AI to transform the way we understand, diagnose, and treat diseases. For more information, visit www.bpgbio.com .

全球药物批准/研发动态 01 全球新药批准情况 根据药渡数据统计分析，本次统计周期（2024.10.26-11.01）全球（不含中国）共有8个新药获批上市。其中，BLA批准2个，新适应症批准4个，新制剂批准2个。与上个统计周期相比，本次增加3个批准新药。 10月28日，贝海生物宣布其自主研发的新药BH009（商品名：BEIZRAY®）正式获得美国FDA上市批准，主要用于乳腺癌、非小细胞肺癌、头颈部鳞癌、前列腺癌以及胃癌的治疗。在临床试验中，BEIZRAY降低了多西他赛特有的血液毒性，整体提高了药物临床获益风险比，为晚期实体瘤患者提供了更优的治疗选择。BEIZRAY作为多西他赛的创新剂型，无论从产品处方工艺、产品特征，还是临床安全性，均具有显著的创新性和临床优势。 10月31日，基石药业宣布MHRA已批准舒格利单抗联合含铂化疗用于无EGFR敏感突变，或无ALK，ROS1，RET基因组肿瘤变异的转移性非小细胞肺癌（NSCLC）成人患者的一线治疗。III期临床研究结果显示，舒格利单抗联合化疗，可显著延长初治转移性NSCLC患者的无进展生存期和总生存期。舒格利单抗不仅可以阻断PD-1/PD-L1相互作用，还能通过介导PD-L1表达阳性的肿瘤细胞与肿瘤相关巨噬细胞（TAMs），在诱导抗体依赖性细胞吞噬（ADCP）的同时不损害效应性T细胞，展现出了潜在同类最优的疗效和安全性。 全球（不含中国）新药批准情况（部分） 02 全球新药申报进展 根据药渡数据统计分析，本次统计周期（2024.10.26-11.01）全球（不含中国）共有7个新药申报上市。其中，NDA申报3个，BLA申报4个。与上次统计周期相比，本次增加1个NDA/BLA申报。 10月30日，复宏汉霖与Organon共同宣布在研PROLIA/XGEVA（地舒单抗）生物类似药HLX14的生物制品许可申请获FDA受理。目前地舒单抗已在多个国家和地区获批用于如骨折高风险的绝经后妇女的骨质疏松症等。此次递交主要基于一系列的头对头比对研究，包括质量对比研究和两项临床研究。 NDA/BLA申报（部分） 根据药渡数据统计分析，本次统计周期（2024.10.26-11.01）全球（不含中国）共有4个药物获监管机构特殊资格认定。与上次统计周期相比，本次增加4个获监管机构特殊资格认定的药物。 10月30日，东诚药业宣布下属公司蓝纳成在研产品177Lu-LNC1004注射液获得FDA授予的快速通道资格。该产品用于治疗接受过一种及以上酪氨酸激酶抑制剂的转移性碘-131难治性分化型甲状腺癌患者。177Lu-LNC1004注射液是一种靶向成纤维细胞活化蛋白的放射性体内治疗药物，在多个肿瘤模型中显示出明显的肿瘤高摄取和更长的滞留时间，能够在较低的剂量下达到显著的肿瘤抑制作用。 10月31日，BPGbio宣布FDA已授予BPM31510T罕见儿科疾病资格认定，适应症为大疱性表皮松解症。BPM31510T开发用于支持EB患者的伤口愈合。通过影响伤口愈合中涉及的关键通路（包括炎症、细胞增殖和组织重塑），BPM31510T已被证明可以支持跨多种亚型的EB伤口护理。在成功的临床前和I期研究之后，BPGbio正在与美国DEBRA合作，将BPM31510T推进到II/III期试验，预计将于2025年开始。 监管机构特殊资格认定药物 03 全球新药研发进展 根据药渡数据统计分析，本次统计周期（2024.10.26-11.01）全球（不含中国）新药临床研发状态更新共计56条，涉及肿瘤、血液和淋巴疾病、心血管疾病、神经疾病以及免疫系统疾病等共计12个领域。 其中，精神疾病领域临床进展更新居各领域之首，为14条：涉及化学药7条，生物药5条，细胞疗法2条。 10月28日，Arcus Biosciences公布HIF-2α抑制剂Casdatifan（AB521）在临床I/Ib期试验ARC-20的积极数据。分析显示，在100mg扩展队列中，转移性透明细胞肾细胞癌（ccRCC）患者的主要疾病进展率仅为19%，疾病控制率（DCR）高达81%。在剂量递增部分（20mg至200mg），各剂量的安全性表现相当，未观察到剂量限制性毒性，且在最高每日150mg剂量下仍未达到最大耐受剂量。 10月31日，Roche在CTAD大会上公布其在研抗体Trontinemab用于治疗轻度至中度阿尔茨海默病患者的Ib/IIa期试验积极结果。分析显示，在28周时大多数接受较高剂量的患者大脑中的淀粉样蛋白斑块几乎全部清除，两种较高剂量（1.8mg/kg与3.6mg/kg）的Trontinemab在第12周时便导致患者大脑淀粉样蛋白水平迅速且显著地降低。 全球新药研发进展详情（部分） 04 全球医药交易事件 本次统计周期（2024.10.26-11.01）全球（含中国）医药交易时间共计31起，涉及药物权益转让、公司并购等多起交易事件。 全球医药交易时间汇总表（部分） 国内药物批准/研发动态 01 国内新药批准情况 根据药渡数据统计分析，本次统计周期（2024.10.26-11.01）国内共有2个新药获NMPA批准上市。其中，NDA批准2个。与上次统计周期相比，本次减少3个NMPA批准新药。 10月22日，礼来中国今日宣布，其非共价（可逆）BTK抑制剂捷帕力®（匹妥布替100毫克和50毫克片剂）获得NMPA批准，单药适用于既往接受过至少两种系统性治疗（含布鲁顿氏酪氨酸激酶[BTK]抑制剂）的复发或难治性套细胞淋巴瘤（MCL）成人患者。数据显示，在既往接受过BTK抑制剂治疗的复发或难治性MCL患者中，IRC评估的ORR为56.7%，中位DoR为17.6个月，中位PFS为7.4个月，中位OS为23.5个月，且耐受性良好。 10月28日，NMPA宣布批准新通药物全资子公司葛蓝新通制药申报的1类创新药甲磺酸普雷福韦片上市申请，用于治疗成人慢性乙型肝炎。这是一种核苷类药物，通过抑制病毒DNA聚合酶以阻止病毒持续复制。试验数据显示，普雷福韦片抑制乙肝病毒DNA复制率、乙肝e抗原（HBeAg）转阴率及肝功复常率等疗效指标与当前一线用药对比的疗效相当，未观察到核苷（酸）类药物常见的对肾脏、骨骼的不良影响，其安全性更佳，对血脂影响更低，长期用药所导致的心脑血管疾病隐患更小。 国内新药批准情况 02 国内新药临床默示许可进展 根据药渡数据统计分析，本次统计周期（2024.10.26-11.01）国内共有31个新药获临床默示许可，涉及54个受理号。其中，化学药17个，治疗用生物制品14个。与上次统计周期相比，本次增加27个临床默示许可获批受理号。 本周国内新药临床默示许可进展（部分） 03 国内新药申报进展 根据药渡数据统计分析，本次统计周期（2024.10.26-11.01）国内共有4个新药申报上市，涉及4个受理号。其中，化药1个，治疗用生物制品3个。与上个统计周期相比，本次增加1个新药申报上市受理号。 国内新药申报上市情况（部分） 根据药渡数据统计分析，本次统计周期（2024.10.26-11.01）国内共有36个新药申报临床，涉及54个受理号。其中，化学药17个，治疗用生物制品18个，预防用生物制品1个。与上次统计周期相比，本次增加4个临床申报受理号。 国内新药临床申报情况（部分） 根据药渡数据统计分析，本次统计周期（2024.10.26-11.01）国内共有1个药物获NMPA特殊资格认定。其中，化学药1个。与上次统计周期相比，本次减少4个获监管机构特殊资格认定的药物。 11月1日，CDE官网最新公示，由艾司林科（苏州）生物医药有限公司申报的AL01211胶囊拟纳入突破性治疗品种，针对适应症为法布雷病。这是AceLink Therapeutics在研的一款口服非脑渗透性GCS（葡萄糖神经酰胺合酶）抑制剂。研究表明，AL01211显示出剂量依赖性的PK和PD效应，AL01211暴露与疾病生物标志物减少之间存在明确的相关性。在30mg剂量水平下，血浆葡萄糖神经酰胺和三己糖酰基鞘脂醇（Globotriaosylceramide）分别比基线水平降低了78%和52%，支持AL01211的进一步临床开发。此外，该产品总体安全、耐受性良好，没有严重的不良事件，且具有优秀的效力（个位数纳摩尔半抑制浓度IC50）、高选择性和可支持每日一次口服的药物特性。 国内监管机构特殊资格认定药物 04 国内新药研发进展 根据药渡数据统计分析，本次统计周期（2024.10.26-11.01）国内新药临床研发状态更新共计9条，涉及肿瘤、血液和淋巴疾病、精神疾病、皮肤病和感染共计5个领域。其中，化学药3条，生物药5条，疫苗1条。与上次统计周期相比，本次增加3条国内新药临床研发状态更新。 10月28日，康方生物宣布其自主研发的靶向IL-17的单克隆抗体古莫奇单抗（AK111）治疗中重度斑块型银屑病的关键III期研究（AK111-301）继2023年达到主要终点后，已于近期完成研究并获得优异的长期疗效和安全性结果。古莫奇单抗是与人IL-17A结合的IgG1单克隆抗体，特异性和高亲和力结合人IL-17A，阻断IL-17介导的信号传导，由此发挥治疗银屑病的作用。古莫奇单抗单药治疗第52周时，PASI75应答率近100%，sPGA0/1应答率近90%，PASI90应答率超90%。 10月30日，中国药物临床试验登记与信息公示平台官网近期公示显示，恒瑞医药已经启动了一项中国III期临床研究，该研究旨在评价HRS-5965胶囊治疗阵发性睡眠性血红蛋白尿患者的有效性和安全性。HRS-5965胶囊靶向因子B，可通过抑制补体系统的过度激活，抑制补体系统介导的溶血。临床前研究显示其在补体介导的溶血模型中起到了显著的抑制作用，安全性良好。 国内新药研发进展（部分） 05 国内新药研发领域政策法规动态 国家药监局关于适用《M12：药物相互作用研究》国际人用药品注册技术协调会指导原则及问答文件的公告（2024年第130号） 为推动药品注册技术标准与国际接轨，经研究，国家药品监督管理局决定适用《M12：药物相互作用研究》国际人用药品注册技术协调会指导原则及问答文件（以下简称M12指导原则及问答文件）。现将有关事项公告如下： 一、自2024年10月29日起开始的相关研究（以试验记录时间点为准），均适用M12指导原则及问答文件。 二、相关技术指导原则可在国家药品监督管理局药品审评中心网站查询。国家药品监督管理局药品审评中心负责本公告实施过程中的相关技术指导工作。特此公告。 06 国内新药研发领域热点新闻 恒瑞医药海外征战的“英雄史诗” 奔赴全球市场，已成为恒瑞医药的发展主线。 正如恒瑞医药副总经理张连山此前在接受媒体采访时表示，“海外战略上我们有所调整，现在每个产品都愿意寻求合作。” 从去年达成5个跨境license-out交易，到今年成功运用NewCo模式将GLP-1产品组合以总价超60亿美元授权出海，恒瑞的BD出海策略愈加成熟。 近期，更是有媒体报道称，恒瑞聘请了强生BD老将Jens Bitsch-Norhave担任“全球发展事务负责人”，也就是海外BD高管。更多资讯，请阅读原文 创新药对外BD模式正在升级迭代 缺医少药的时代，离我们并没有多远。 曾几何时，我们迟迟无法用到国外早已上市的成熟药，多少人遗憾离世；曾几何时，我们必须付出高昂的代价才能获得国外特效药，多少人因病返贫。在那个时代，进口药尚且稀缺，更遑论产自中国的创新药能够“出海”。但时过境迁，今日的中国创新药已不可同日而语，一笔笔天价交易彰显着中国正在走近世界舞台的中央，并且，在大规模对外交易中，一些更符合中国药企利益的BD模式也已经衍生出来了。更多资讯，请阅读原文 咨询、合作请联系作者 小D有话说 为方便读者阅读及保存，我们将周报原文整理成了PDF版本，如需获取全文，可点击最上方蓝字，在药渡Daily公众号后台回复“1107周报”，即可下载。