Traditional drug discovery is fraught with failure and often like trying to find a needle in a haystack. Even once a candidate is identified, the development process takes thousands of man-hours and generally billions of dollars. Artificial Intelligence has the potential to change this paradigm. It can aggregate and synthesize vast amounts of clinical trial data, effectively shortening the drug development timeline. It aids in understanding disease mechanisms, identifying biomarkers, generating data and models, and even discovering novel drug candidates. Additionally, it supports drug design or redesign, runs preclinical experiments, optimizes clinical trial design, and provides insights from real-world data.
While we are not yet living in an era where AI-discovered and developed drugs are widely available to patients – with all the ramifications this would have on financial savings, speed to market, access, and medical breakthroughs – here are five companies attempting to get us there.
Insilico Medicine
Initially an algorithm and software company driven by generative artificial intelligence (AI), Hong Kong-based Insilico made a strong shift towards AI-driven drug discovery in 2020. “Big Pharma, utilizing its traditional model of small molecule chemistry and internal R&D can generally generate four or five preclinical candidates in the same amount of time that our AI-driven approach is able to generate nine,” founder and CEO Alex Zhavoronkov told PharmaBoardroom last year. “Generative AI allows us to rapidly discover targets, formulate the shortest path from the target to the patient, and generate chemistry with the right properties from scratch instead of searching for a compound with those said properties.”
A collaboration with Sanofi that began in 2022, recently yielded an AI-facilitated lead with first-in-class (FIC) potential against an undruggable transcription factor target for treating oncology diseases, enhanced by Insilico Medicine’s AI platform, Pharma.AI and was responsible for what Zhavoronkov called the first AI-designed and discovered candidate to reach Phase II human clinical trials, for which the company has announced positive preliminary results from its Phase IIa clinical trial.
XtalPi
Perhaps China’s leading company in this space, XtalPi was established in 2015 by three postdoctoral physicists at MIT and XtalPi employs AI to predict crystal structures and properties of small-molecule drugs, enhancing drug design efficiency.
The firm has since entered into an AI small molecule drug discovery collaboration with Eli Lilly. “The scale of the chemical space we work with—10 to the power of 60 —makes traditional methods far too slow and inefficient. With AI and quantum physics, we can navigate this vast space quickly and uncover new drug targets that were previously inaccessible. AI allows us to simulate and analyze chemical compounds efficiently, significantly reducing the need for traditional trial and error,” CFO Ronald Tam told PharmaBoardroom.
In recent news, the company entered into a new licensing agreement with Janssen for the use of its proprietary AI biologics discovery platform, XtalFold and saw its share prices soar on the Hong Kong stock exchange after striking a UD 135 million deal with Chinese billionaire Zhu Gongshan’s energy company GCL Group after it began trading in Hong Kong last June.
OWKIN
French biotech OWKIN, founded in 2016, uses AI to identify new drug candidates, de-risk and accelerate clinical trials, and build diagnostic solutions. “AI can be split into two functions, the acceleration of processes like drug discovery and the augmentation of human skills, such as improving clinical trial procedures. There is strong precedence already in drug discovery but the best way for AI to be used in development is growing and better relating to the use of real-world data. Augmentation is trying to better understand the diseases and predict which variations will be resistant to new therapies or how new treatments may react with the patient. Outcome research such as response to targeted therapies are difficult to measure without this tool,” said founder and CEO Thomas Clozel in a PharmaBoardroom interview.
A recent development for the company has been its partnership with AstraZeneca to develop an AI-powered tool designed to prescreen for gBRCA mutations (gBRCAm) in breast cancer directly from digitized pathology slides in order to to speed up and increase access to gBRCS testing that some patients may not be considered for.
Already in 2021, Sanofi made an equity investment of USD 180 million and entered into a strategic collaboration with Owkin to work on drug discovery and development programmes, a partnership which expanded this year to include immunology and will be focused on drug positioning within Sanofi’s immunology therapeutic pipeline.
Exscientia
Exscientia, considered a trailblazer in AI-driven precision medicine, is focused rapidly discovering, designing, and developing optimal drugs through its advanced AI technology. The company pioneered the first functional precision oncology platform, effectively guiding treatment selection and enhancing patient outcomes in prospective interventional clinical studies. This platform has successfully advanced AI-designed small molecules into clinical settings.
The company’s leading candidate, GTAEXS617, is undergoing a Phase 1/2 trial named ELUCIDATE, targeting advanced solid tumors such as head and neck, breast, and non-small cell lung cancers.
Exscientia has established significant partnerships, notably with Sanofi and Merck. In 2017, Exscientia and Sanofi entered a $273 million licensing agreement to discover bispecific small molecule drugs for metabolic diseases. This collaboration expanded in 2022 to include up to 15 small molecule candidates in oncology and immunology. In October 2024, Exscientia announced a $674 million deal with Merck to discover novel small molecule drug candidates across oncology, neuroinflammation, and immunology.
Reflecting on these collaborations, Exscientia’s CEO, David Hallett, stated, “Adding Exscientia’s best-in-class focused precision oncology internal pipeline to Recursion’s first-in-class focused pipeline spanning rare disease, precision oncology and infectious disease is highly complementary as we look to bring treatments to patients faster.”
BenevolentAI
UK-headquartered BenevolentAI is another key player in the AI-driven drug discovery landscape and has been around for over a decade. Benevolent was the first firm to achieve FDA approval for an AI-identified drug in 2022, after the US regulator authorised a new use for Lilly’s Olumiant (baricitinib) for treating COVID-19. Since 2019 it has engaged in a strategic collaboration with AstraZeneca to identify novel targets for chronic kidney disease (CKD) and idiopathic pulmonary fibrosis (IPF).
The last couple of years have been challenging for the company. In May 2023, following a mid-phase clinical trial failure of their lead candidate, BEN-2293, for atopic dermatitis, the company laid off up to 180 employees, reduced its lab footprint, and paused certain programs. In April 2024 it announced further layoffs, cutting 30% of its workforce, closing its U.S. office, and discontinuing its software-as-a-service business to focus on its internal and partnered drug pipeline.
However, the tide has perhaps turned for Benevolent with a USD 594 million partership with German group Merck recently announced. The deal will see Benevolent apply its AI platform to deliver small-molecule drugs across three programmes in oncology, neurology, and immunology. Additionally, two of the company’s co-founders – Ken Mulvany and Michael Brennan – recently returned to Benevolent as executive chairman (Mulvany) and chief strategy and financial officer and executive director (Brennan), perhaps heralding a new era of success.
