药物研发

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5年20亿,神经与肿瘤药物研发全国重点实验室发布“求索计划”
Xin Jing Bao· 2025-03-31 03:43
新京报讯(记者王卡拉)在3月29日-3月30日举行的神经与肿瘤药物研发全国重点实验室协同创新会议上, 神经与肿瘤药物研发全国重点实验室发布"先声全重求索计划",预计5年累计投入20亿元,聚焦神经与 肿瘤领域十大科学难题,支持"从0到1"的颠覆性创新研究。 "求索计划"由11位中国科学院及工程院院士组成科学指导委员会,为项目进行方向性规划,鼓励非共识 性创新、多学科交叉融合、综合运用新兴技术。 "求索计划"主要针对十大研究方向:从根本上逆转脑卒中高死亡率、高致残率的治疗手段;精准治疗神 经退行性疾病的潜在靶点发现、验证和药物研发;有望大幅提升当前肿瘤治疗效果的靶点(或组合)发 现、验证和药物研发验证;RNA干扰/mRNA技术在神经退行性疾病、肿瘤和感染等领域的治疗性应 用;新一代细胞治疗的临床应用;新一代基因编辑等相关技术的临床应用;新一代递送技术的临床应 用;抗衰老相关机制的临床应用;基于临床数据,AI技术手段应用于靶点发现、验证;AI技术手段应 用于药物研发临床有效性和安全性预测。 "求索,顾名思义就是要探索未知、攻克难题、寻找答案,我们要凝聚一群生命科学领域、年轻的求索 者,为了患者和临床医生的期待,探索 ...
Cognition Therapeutics(CGTX) - 2024 Q4 - Earnings Call Transcript
2025-03-20 17:15
Financial Data and Key Metrics Changes - The company reported a net loss of $34 million or $0.86 per basic and diluted share for the year ended December 31, 2024, compared to a net loss of $25.8 million or $0.86 per basic and diluted share for 2023 [23] - Cash and cash equivalents as of December 31, 2024, were approximately $25 million, with total obligated grant funds remaining from the NIA at $50 million [20][21] - Research and development expenses increased to $41.7 million for the year ended December 31, 2024, from $37.2 million for 2023, primarily due to higher costs associated with completing two Phase II trials [22] Business Line Data and Key Metrics Changes - The company decided to conclude its Phase II dry AMD study before completion, reallocating resources entirely to Alzheimer's and DLB programs [11][19] - The decision to conclude the dry AMD study was based on strategic prioritization rather than safety concerns, as the analysis indicated potential efficacy [12][13] Market Data and Key Metrics Changes - The company is actively pursuing partnerships for funding and development in the Alzheimer's and DLB space, indicating strong interest from biotech and pharma players [15][35] - The company received a 6-month grace period to regain compliance with NASDAQ's minimum bid requirement, needing to maintain a stock price above $1 for 10 consecutive days before September 8, 2025 [24] Company Strategy and Development Direction - The focus remains on advancing zervimesine for Alzheimer's disease and DLB, with plans to submit final study documents to the FDA for two different end of Phase II meetings [10][14] - The company is preparing for Phase III readiness and has developed a novel chemical process for zervimesine manufacturing, with provisional patent applications filed [17][18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in regaining NASDAQ compliance and highlighted upcoming milestones that could drive stock value [24][60] - The company is committed to overcoming financing challenges and is focused on delivering multiple clinical milestones to create long-term shareholder value [60] Other Important Information - The company has built strong relationships with potential partners and is evaluating all options for financing clinical development efforts [15][16] - The publication process for SHIMMER data is underway, with expectations for release in the coming months [37] Q&A Session Summary Question: Thoughts on tau cutoff threshold for end of Phase II FDA meeting - Management plans to enrich participants in the next study for those with lower tau, similar to the SHINE study, but has not finalized the specific cutoff [29][30] Question: Feedback from investigative physicians on SHIMMER data - Management received excellent feedback from KOLs and neurologists, indicating a strong need for zervimesine and appreciation for its safety profile [35] Question: Competitive landscape in DLB and potential approvability - Management is confident that neuropsychiatric symptoms will be of interest to the FDA, and they plan to propose specific outcome measures during the FDA meeting [49][50] Question: Outlook for accelerated approval in Europe vs. the US - The company plans to follow a traditional pathway for approval, as the accelerated pathway has shown mixed results in the past [52] Question: NIH funding outlook and potential for new grants - The company has a remaining balance of $50 million from NIH funding, which is expected to support ongoing trials, but anticipates lower probability for new grant funding as it progresses to Phase III [54][56]
DiaMedica Therapeutics(DMAC) - 2024 Q4 - Earnings Call Transcript
2025-03-18 14:27
Financial Data and Key Metrics Changes - As of December 31, 2024, the company reported total combined cash and investments of $44.1 million, down from $52.9 million at the end of 2023, indicating a decrease due to cash used for operations [34] - Current liabilities increased to $5.4 million from $2.8 million, while working capital decreased to $39.2 million from $50.9 million [34] - Net cash used in operating activities for the full year 2024 was $22.1 million, compared to $18.7 million in 2023, driven by increased net loss and advance deposit funds to vendors [35] Business Line Data and Key Metrics Changes - Research and development expenses rose to $19.1 million for the year ended December 31, 2024, up from $13.1 million in the prior year, primarily due to the continuation of the ReMEDy2 clinical trial and increased manufacturing activities [36] - General and administrative expenses decreased to $7.6 million from $8.2 million, attributed to reduced legal fees and lower insurance premiums, partially offset by increased personnel costs [38] Market Data and Key Metrics Changes - The company activated 30 clinical sites for the ReMEDy2 trial, which is expected to generate a steady stream of enrollments [16] - The protocol for the stroke program was updated to allow DM199 to be stored at refrigerated temperatures, expanding the eligible patient population [17] Company Strategy and Development Direction - The company is focused on advancing its clinical programs for DM199 in both stroke and preeclampsia, aiming to provide treatment options for conditions currently lacking effective therapies [31][78] - The strategy includes expanding the ReMEDy2 trial globally and increasing site activation and enrollment activities [37] Management's Comments on Operating Environment and Future Outlook - Management acknowledged challenges in site engagement and enrollment due to lower staffing levels in research units post-COVID, but expressed optimism about increasing enrollment rates in 2025 [22][24] - The company anticipates that its current cash and investments will provide a runway into Q3 of 2026, indicating a stable financial outlook for the near term [36] Other Important Information - The independent Data Safety Monitoring Board reviewed safety data and concluded that the ReMEDy2 trial should continue without modification [27] - A recent publication analyzed the mechanism of action of DM199, providing scientific insight into its potential benefits for acute ischemic stroke patients [28] Q&A Session Summary Question: How many of the activated sites are among the top 15 identified? - The majority of the top 15 sites are activated, with many currently enrolling patients [43] Question: What data did the DSMB review for safety? - The DSMB had access to the entire database of patient data up until a specific cutoff date [45] Question: What are the expectations around enrollment rates for the interim analysis? - The company anticipates doubling the number of enrolling sites and targeting sites that can enroll about one to two patients per month [55] Question: Has the amended statistical analysis plan been finalized with the FDA? - Yes, the amended statistical analysis plan has been finalized with the FDA [74]
科技+投资!机器人成券商策略会上“最靓的仔”
券商中国· 2025-03-14 01:36
3月13日,华泰证券、西南证券、华西证券均选在这一天召开春季策略会。 正如华泰证券机构业务委员会主席梁红在致辞中提到的,当前国际变局和科技进步正以前所未有的速度冲击传统秩序,中国 资产正在开启价值重估的大幕。 在此背景下,科技成为各家研究所的核心议题,不管是主会场还是分论坛,均以大篇幅多角度对科技产业逻辑进行剖析和解 读。值得一提的是,三家券商还不约而同在策略会现场安排了机器人、机器狗的表演及互动环节,生动演示了何为"科技+投 资"的深度融合。 券商策略会开卷机器人 券商扎堆召开春季策略会,宇树科技或许成为最大赢家。券商中国记者发现,3月13日在深圳、杭州、重庆三城,同步召开的 三家券商春季策略会,竟都安排了宇树科技的机器人及机器狗作为"特邀嘉宾",在现场与参会者零距离亲密互动。 华泰证券策略会会场外,参会者与机器人握手 华西证券策略会会场外,参会者观看机器狗表演 与此同时,各家券商策略会主题也基本聚焦以AI为代表的科技产业。例如,华西证券在3月13日上午的主论坛中,邀请了人工 智能相关行业的创始人,讲述AICG技术的商业应用以及AI眼镜的未来图景。 而华泰证券两天共6场分论坛中,仅科技主题论坛就有三场,分别 ...