A Quantitative Systems Pharmacology Software Platform for Biologics: reduce costs and accelerate development of innovative biotherapeutics

 描述（由申请人提供）：药物开发是一项非常漫长和昂贵的工作。新药的失败率超过95%。因此，成功的药物必须弥补这些失败的成本。因此，处方药价格以惊人的速度上涨，而且没有停止的迹象。成功的药物需要覆盖失败的药物，这也意味着制药公司主要将资源投入到追求候选药物上，这些候选药物具有足够大的人群，使公司能够获得投资回报。因此，只影响一小部分人口的疾病没有像肿瘤学、心血管学或免疫学那样得到足够的研究。目前的做法通常涉及采用为小分子研究开发的建模技术，并试图使其适应生物制剂。然而，这种方法往往不能为科学家提供关于可行性和最佳药物特性的预测，导致浪费精力追求具有潜在价值的线索。 没有机会通过临床试验，也没有机会得到付款人的报销。Applied BioMath开发了一些工具，可以解决药物开发过程中的高价值问题。通过将定量系统药理学技术与高性能计算和复杂的数学算法相结合，我们已经证明了在进入临床前几年预测最佳药物特性的能力。在过去的两年里，我们一直为制药和生物技术公司提供服务，好评如潮。我们还收到了有关许可我们软件的询问。该项目将资助我们的专有算法和工具集开发成一个稳定的标准化软件平台，该平台可以自动验证GLP，用于生物制剂开发其内部系统药理学模型。在其核心，我们的工具集是建立在Kronecker Bio，一个开源的生物物理计算引擎，由我们的创始人之一，同时追求他的生物工程博士学位与计算机科学和人工智能实验室从马萨诸塞州理工学院开发。这个强大的平台目前以其原始形式在制药行业中使用，但由于缺乏可用性，质量控制和GLP验证，其采用受到限制。该项目将侧重于应用程序和表示层，使底层计算功能易于访问，利用和理解，因此资本需求低于典型的软件开发项目。实现我们构建这个软件平台的目标只是第一步。接下来是对生物制剂领域的一致推动，我们目前正在通过我们的服务提供播种，并获得作为一家按时提供高价值的公司的声誉。我们已经完成了第二轮融资，两轮之间共筹集了180万美元。这笔赠款，加上额外的筹款，将确保我们能够推出我们的工具，并协助制药公司提供最好的生物制剂，满足未满足的医疗需求，加快时间轴，为患者提供更好的生活质量。更好，更快，更便宜的药物...真的是一场双赢的比赛。

 DESCRIPTION (provided by applicant): Drug development is a very lengthy and expensive undertaking. Failure rate for novel drugs exceeds 95%. Therefore, successful drugs must cover the costs of these failures. As such, prescription drug prices have escalated at an alarming rate and show no signs of stopping. The need for successful drugs to cover failures also means that pharmaceutical companies primarily devote resources to pursuing drug candidates that have a large enough population to allow the company to earn a return on its investment. Thus, diseases that affect only a small portion of the populace are not investigated nearly as much as, say, oncology, cardiovascular or immunology. Current practice usually involves taking modeling techniques developed for small molecule research and trying to adapt them to biologics. However, this approach, more often than not, does not provide the scientist with predictions around feasibility and optimal drug properties, resulting in wasted effort pursuing leads that have no chance of making it through clinical trials, or to be reimbursed by payors. Applied BioMath has developed tools that address high value questions in the middle of the drug development pipeline. By coupling quantitative systems pharmacology techniques with high performance computing and sophisticated mathematical algorithms, we have proven an ability to predict optimal drug properties years before entering the clinic. For the past two years we have been offering our services to pharma and biotechs alike, to rave reviews. We have also been approached with inquiries to license our software. This project will fund the development of our proprietary algorithms and toolsets into a stable, standardized software platform, that can be automatically validated for GLP, for by biologics to develop their internal systems pharmacology models. At its heart, our toolsets are built on Kronecker Bio, an open source biophysical computational engine co­developed by one of our Founders while pursuing his PhD in Biological Engineering with the Computer Science and Artificial Intelligence Lab from the Massachusetts Institute of Technology. This robust platform is currently in use, in its raw form, in the pharmaceutical industry but is limited in its adoption due to its lack of usability, quality contro and GLP validation. This project will focus on the application and presentation layer, allowing the underlying computational functionality to be easily accessed, utilized and understood, so capital requirements are less than a typical software development project. Achieving our goal of building this software platform is only the first step. What follows is a concerted push into the biologics segment, which we are currently seeding through our services offering and gaining a reputation as a firm that delivers high value on time. We have completed our second round of fundraising, raising a total of $1.8m between both rounds. This grant, plus the additional fundraising, will ensure that we are able to roll out our tools and assist drug companies in delivering best­in­class biologics, that meet unmet medical need, on an accelerated timeline to provide patients with a better quality of life. Better, faster, cheaper drugs... truly a win­win­in.