Healx has raised $47 million in a Series C round co-led by Silicon Valley-based R42 Group and Atomico with participation from new and existing investors including Balderton, Jonathan Milner, Global Brain, btov, Ayana Capital, o2h, and VU Venture Partners. In conjunction with the financing, Stanford Medicine adjunct professor Ronjon Nag, PhD, founder of R42 Group and 2024 Silicon Valley Hall of Fame AI inductee will join Healx’s board..

Healx在硅谷R42集团和Atomico共同领导的C轮融资中筹集了4700万美元，新的和现有的投资者包括Balderton、Jonathan Milner、Global Brain、btov、Ayana Capital、o2h和VU Venture Partners。除此之外，斯坦福医学副教授Ronjon Nag博士、R42集团创始人和2024硅谷名人堂AI入选者将加入Healx董事会。。

The funds will help the company accomplish its goal of using AI to identify new treatments for rare diseases. Healx said that the proceeds of the financing will be used to advance its pipeline of medicines in rare oncology, renal, and neurodevelopmental disorders. This includes advancing its lead program HLX-1502 through a Phase II clinical trial for the treatment of neurofibromatosis type 1 (NF1).

这些资金将帮助该公司实现其使用人工智能识别罕见疾病新疗法的目标。希克斯表示，融资所得将用于推进罕见肿瘤学、肾脏和神经发育障碍的药物管道。这包括通过治疗1型神经纤维瘤病（NF1）的II期临床试验推进其主要计划HLX-1502。

Tim Guilliams, PhD, Healx’s co-founder and CEO, said that the company intends to begin testing HLX-1502 in patients by December 2024. .

Healx的联合创始人兼首席执行官蒂姆·吉利亚姆博士表示，该公司打算在2024年12月之前开始在患者中测试HLX-1502。

Healx has been cleared by the FDA to move forward with a Phase II trial that will test its drug in adults with NF1 and inoperable plexiform neurofibroma.

Healx已被FDA批准进行II期临床试验，该试验将在患有NF1和无法手术的丛状神经纤维瘤的成年人中测试其药物。

HLX-1502 is a tablet taken orally that Healx claims works differently from other NFI treatments. The drug has received orphan drug and rare pediatric disease designations from the FDA for treating NF1. The company has also signed an investment agreement with its long-term research partner, Children’s Tumor Foundation (CTF).

HLX-1502是一种口服片剂，Healx声称其作用与其他NFI治疗不同。该药物已被FDA指定用于治疗NF1的孤儿药和罕见儿科疾病。该公司还与其长期研究合作伙伴儿童肿瘤基金会（CTF）签署了投资协议。

Under the terms of the agreement, Healx will receive milestone-driven payments from CTF that it will use to move its drug programs forward including HLX-1502..

根据协议条款，Healx将从CTF获得里程碑式的付款，用于推进包括HLX-1502在内的药物计划。。

Tim Guilliams, PhD,Healx Co-founder and CEO

Tim Guilliams博士，Healx联合创始人兼首席执行官

Several of those drugs will target a few of the approximately 10,000 rare diseases, about 95% of which currently don’t have an approved treatment. Guilliams said that Healx opted to focus on rare diseases because of this unmet need. Aside from the commercial opportunity, “there’s the ability to make a breakthrough and find something new that’s really going to be impactful for the patient community and the families,” he told GEN. .

其中一些药物将针对大约10000种罕见疾病中的一些，其中约95%目前尚未获得批准的治疗。Guilliams说，由于这种未满足的需求，Healx选择专注于罕见疾病。除了商业机会之外，“还有能力取得突破，找到对患者社区和家庭真正有影响的新东西，”他告诉GEN。

Healx is confident that the approach used by its machine learning solution, HealNet, can help with finding effective candidates for rare diseases. “Broadly it’s really understanding disease biology in a different way, in a more complex way,”  Guilliams explained. Specifically, it defines “complex patterns and signatures around disease” as well as “complex drug patterns and signatures that basically reverse the disease signature into a healthy one.” These definitions are based on a combination of data types including information from transcriptomic, metabolomic, and phenomic experiments.  .

Healx相信其机器学习解决方案HealNet使用的方法可以帮助寻找罕见疾病的有效候选者。“从广义上讲，它真正以一种不同的方式，以更复杂的方式理解疾病生物学，”吉利亚姆解释道。具体而言，它定义了“疾病周围的复杂模式和特征”以及“基本上将疾病特征逆转为健康特征的复杂药物模式和特征”。这些定义基于数据类型的组合，包括来自转录组学，代谢组学和表型实验的信息。。

The system then predicts drug candidates that are most likely to target and reverse the disease signature, and Healx scientists test them in the lab. “The first wave AI technology that we built was around a biomedical rare disease knowledge graph. That was basically based on the Google search model,” Guilliams said.

然后，该系统预测最有可能靶向和逆转疾病特征的候选药物，Healx科学家在实验室对其进行测试。“我们建立的第一波人工智能技术是围绕生物医学罕见疾病知识图。这基本上是基于谷歌搜索模型，”吉利亚姆说。

Now with the advent of generative AI and large language models, Healx is looking to leverage the technology to enhance its platform further using some of the funds raised in the Series C. Their plans include generating more data to better understand rare disease biology as well as using techniques such as retrieval augmented generation to try to fill in gaps in their knowledge about diseases. .

现在，随着生成人工智能和大型语言模型的出现，Healx希望利用C系列中筹集的一些资金，利用这项技术进一步增强其平台。他们的计划包括生成更多数据以更好地了解罕见疾病生物学，以及使用检索增强生成等技术，试图填补他们对疾病知识的空白。。

At the moment, “drug discovery is largely focused on a target-based paradigm [and] in certain cases it works,” he said. However, that approach may not work for most rare diseases whose biology “is still poorly understood. And then if you look at more complex diseases, maybe your single target will not work to … modulate the disease biology.” This is why Healx approaches drug discovery for rare diseases in “a target agnostic manner” from the very beginning. .

目前，“药物发现主要集中在基于目标的范例上，在某些情况下它是有效的，”他说。然而，这种方法可能不适用于生物学“仍然知之甚少”的大多数罕见疾病。然后，如果你研究更复杂的疾病，可能你的单一目标无法……调节疾病生物学。”这就是为什么Healx从一开始就以“目标不可知的方式”对罕见疾病进行药物发现的原因。。

Testing HLX-1502 in patients

在患者中测试HLX-1502

Affecting approximately 1 in 2,500 individuals, NF1 is a rare genetic disorder that is associated with a predisposition to develop multiple benign and malignant tumors. Two notable types of tumors associated with NF1 are plexiform neurofibromas and cutaneous neurofibromas.

NF1是一种罕见的遗传疾病，约每2500个人中就有1个人受到影响，与易患多种良性和恶性肿瘤有关。与NF1相关的两种显着类型的肿瘤是丛状神经纤维瘤和皮肤神经纤维瘤。

Plexiform neurofibromas grow aggressively along nerves which often leads to significant morbidity and greater risk of malignant transformation. These tumors can cause functional impairments, disfigurement, and pain, and require multidisciplinary management. Currently, a single treatment option is available for some children with plexiform neurofibromas.

丛状神经纤维瘤沿着神经积极生长，这通常导致显着的发病率和更大的恶性转化风险。这些肿瘤可能导致功能障碍，毁容和疼痛，需要多学科管理。目前，一些患有丛状神经纤维瘤的儿童可以使用单一的治疗选择。

But these treatments have tolerability and safety concerns including GI, heart, eye, and skin toxicity. Cutaneous neurofibromas, for their part, are benign tumors but they can cause significant symptoms. There are currently no approved treatments for this subtype. .

但这些治疗具有耐受性和安全性问题，包括胃肠道，心脏，眼睛和皮肤毒性。皮肤神经纤维瘤本身是良性肿瘤，但可引起明显症状。目前尚无针对该亚型的批准治疗方法。。

Currently, chemotherapies seem to be the treatment approach of choice for the pharma companies that are working on therapies for NF1. So far, AstraZeneca’s Koselugo (selumetinib), a MEK inhibitor, has been approved for the treatment of NF1 patients in 32 countries including the United States and China, and is in clinical trials for other tumor types that occur in NF2-related schwannomatosis and other types of schwannomatosis..

目前，化学疗法似乎是正在研究NF1疗法的制药公司的首选治疗方法。到目前为止，阿斯利康（AstraZeneca）的Koselugo（selumetinib）是一种MEK抑制剂，已被批准用于治疗包括美国和中国在内的32个国家的NF1患者，并且正在对NF2相关神经鞘瘤病和其他类型神经鞘瘤病中发生的其他肿瘤类型进行临床试验。。

But chemotherapies have significant side effects. If Healx’s drug is successful, the company claims it will have a much better safety profile than existing inhibitors, which is good since the drug is intended for chronic use.

但是化学疗法有明显的副作用。如果Healx的药物成功，该公司声称它将比现有的抑制剂具有更好的安全性，这是好的，因为该药物旨在长期使用。

And early data about the molecule is promising. There is documented safety data on the compound from previous studies in healthy volunteers. Results from those studies also describe properties about the compound that indicate a high affinity for nerve cells.

。先前对健康志愿者的研究记录了该化合物的安全性数据。这些研究的结果还描述了该化合物对神经细胞具有高亲和力的特性。

In NF1 cases, HLX-1502 works by modulating mitochondrial function, through various pathways, to control tumor growth. It is also drawn to signals from the environment around nerve cells with tumors, including higher levels of oxidative stress. “It’s a small molecule with very few side effects and shown preclinically to be exceptionally effective at reducing tumor growth and taking out the tumor cells,” Guilliams said. .

在NF1病例中，HLX-1502通过各种途径调节线粒体功能来控制肿瘤生长。它还被来自患有肿瘤的神经细胞周围环境的信号所吸引，包括更高水平的氧化应激。“这是一种副作用很小的小分子，临床前已证明在减少肿瘤生长和清除肿瘤细胞方面非常有效，”吉利亚姆说。。

Healx launched its HLX-1502 program in 2019 and plans to dose its first of 20 patients participating in the trial later this year. They’ll be evaluating the participants for tumor reduction along the nerve in response to the drug as the clinical trial endpoint.

Healx于2019年启动了其HLX-1502计划，并计划在今年晚些时候为参与该试验的20名患者中的第一名患者提供剂量。作为临床试验终点，他们将评估参与者对该药物的神经肿瘤减少反应。

For Guillams, AI was critical for helping his team “analyze things quickly, match drugs, and patterns, and then select the best one with the best properties” but so was its partnership with CTF. “They really helped us accelerate things further [and] they also invested in the program themselves.” Healx hopes to wrap up the trial in the first half of 2026. .

对于Guillams来说，AI对于帮助他的团队“快速分析事物，匹配药物和模式，然后选择具有最佳特性的最佳药物”至关重要，但它与CTF的合作关系也是如此。“他们确实帮助我们进一步加快了速度，而且他们自己也对该项目进行了投资。”希克斯希望在2026年上半年完成试验。

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