Abstract
Artificial intelligence (AI) has the potential to improve many areas of medicine, with drug development as a key focus. Many companies are now working to apply the power of AI to design better drugs, but six companies are leading the race with candidates already in clinical trials.
Over the last few years, advances in AI, particularly in image and language processing, have vastly expanded the applications it can be used for. It has the potential to make precision medicine a reality including improving imaging and diagnostics, population health, and allowing the development of more targeted drugs.
The use of AI can make drug development quicker, cheaper, and more efficient. Large libraries of potential drug candidates can be scanned for disease targets in the blink of an eye using these tools. The medical literature can also be analyzed to make sure past mistakes are not repeated and the most suitable patients are recruited for clinical trials to improve the possibility of the drug in question reaching the market.
Use of AI can also reduce the need for extensive preclinical trials in animals and speed up the time it takes for a potential drug candidate to reach the clinic, something that can be crucial for conditions where no treatment currently exists. This also has the advantage of reducing development costs, meaning pharma companies may be more likely to proceed along the development pathway.
The huge potential of this area has led to the founding of many new companies that are using AI in different ways to bring new drugs to the clinic. However, only a few have actually succeeded in reaching clinical trials.
Here are six companies using AI to develop new drugs who have already progressed to clinical trials and are leading the race to have the first AI-developed drug on the market.
Founded in 2012, Exscientia was one of the first companies to apply AI technology to drug discovery. It is one of the leading players in this area and has a focus on quickly identifying and optimizing drug candidates that are most likely to succeed in clinical trials.
The firm raised $451M in an initial public offering on the Nasdaq and accompanying private placement in October 2021, with more than $470M raised overall, and has many partnerships with big pharma and the biotech industry, namely Evotec, Bristol-Myers Squibb, Bayer and Sanofi who it signed a $4.7M deal to develop oncology and immunology treatments with earlier this year.
The company's first AI-designed drug candidate, to treat obsessive-compulsive disorder, began human testing in a phase I trial in 2020 in collaboration with Sumitomo Dainippon Pharma. It took Exscientia 12 months to bring the candidate to clinical trials rather than the average industry time of around five years. More recently, the company has also tested a potential cancer treatment, developed with Evotec, in healthy volunteers at Phase I. The treatment is aimed at solid tumours exhibiting high adenosine signatures and should move into the next stage of development in cancer patients later this year. A second neuropsychiatric compound to treat Alzheimer's disease psychosis, also developed in collaboration with Sumitomo Dainippon Pharma, started phase I trials last year.
Recursion Pharmaceuticals was founded in 2013 and already has 4 drug candidates in clinical trials. It uses sophisticated lab robotics and automation to carry out as many as 1.5 million experiments a week for diseases that can be modelled on the cellular level. It uses neural networks and other computational techniques to link these with other databases of chemical and biological information and extract relevant information, for example, using phenotype analysis or ‘phenomics'. As part of its data collection and research, Recursion is creating a map of human cellular biology to help improve its processes in future.
Recursion has a focus on rare disease and has two drug candidates, for cerebral cavernous malformation and neurofibromatosis type 2, undergoing phase II clinical trials and two candidates, for familial adenomatous polyposis and GM2 gangliosidosis, at phase I. It also has a number of compounds being tested in preclinical trials.
The company launched on the Nasdaq last year with more than $500M in proceeds after its terms were bumped up several times. Similar to Exscientia, it also has several big pharma collaborations notably with Roche and Genentech, aiming to focus on neuroscience and oncology targets, and an older partnership with Bayer focusing on developing drugs for fibrotic diseases.
Similar to Recursion, Benevolent AI was founded in 2013. The company has an AI platform, but also its own pharmaceutical team to develop potential assets. The company began life as Stratified Medicine before changing its name in 2016.
Like the other companies on the list, Benevolent is developing drugs in a range of areas including inflammatory bowel diseases, atopic dermatitis, cancer and the non-alcoholic liver disease NASH, among others. It has an atopic dermatitis drug in phase I/II clinical trials and was also instrumental in identifying a treatment for COVID-19 during the pandemic. This treatment, the rheumatoid arthritis drug baricitinib, improves and speeds up the recovery of hospitalized patients with COVID-19 and recently achieved approval by the FDA for treatment of hospitalized adults with the infection.
BenevolentAI has its own drug candidates, but also has various partnerships including with AstraZeneca and Novartis. The company technically reached ‘unicorn' status in 2018 when it achieved a valuation of just over $1.7B after a big fundraising. However, the company's valuation suffered significantly when UK-based Neil Woodford, who had been a major investor, was forced to wind up his fund in 2019, though the valuation has since improved.
In December last year the company merged with Odyssey Acquisition, an Amsterdam-listed special-purpose acquisition company (SPAC) in order to list on the Euronext Amsterdam stock exchange. This deal is the largest European SPAC merger so far and one of the largest Euronext Amsterdam biotech listings ever. Since the merger, the company valuation is almost back to 2018 levels at $1.5B
Insilico Medicine was founded in 2014 and has developed a machine learning model called Generative Tensorial Reinforcement Learning (GENTRL), a type of generative adversarial network (GAN) that uses neural networks to search for new drug targets. Unlike other companies in this field, it is using AI to develop the entire drug discovery pipeline.
In December last year it announced its first AI-designed and discovered drug candidate had reached clinical trials in less than three years. The drug candidate is designed to target idiopathic pulmonary fibrosis, a rare lung disease with a poor prognosis. Other targets not yet at the clinical trial stage include inflammatory bowel disease, cancer and COVID-19, among others, and this month the company announced its PandaOmics engine had revealed 28 potential new targets to treat amyotrophic lateral sclerosis or ALS.
The company announced in June it had raised $60M in Series D funding, bringing its total funding to approximately $366.3M. Insilico has multiple academic partnerships and is also working with 4B Technologies on ALS treatments and with Fosun Pharma on an immune-oncology candidate.
Relay therapeutics was founded in 2016 and had a primary focus on developing precision oncology drugs for previously ‘undrug-gable' targets. It uses a three-way approach combining structural biology work to assess protein motion, genomic data and AI. The company has two candidate drugs in early clinical trials, as well as others at the preclinical stage. The most advanced candidate is an oral, small molecule, selective inhibitor of FGFR2, a receptor tyrosine kinase that is altered in some cancers. The second is an allosteric, pan-mutant (H1047X, E542X and E545X) and isoform-selective PI3Kα inhibitor – PI3Kα is the most frequently mutated kinase in cancer.
Relay listed on the Nasdaq in 2020 and has so far raised approximately $870M in total across all funding rounds. Last year it acquired ZebiAI for $85M to bring in complementary expertise on DNA-encoded libraries (DELs), groups of small molecules tagged with unique DNA sequences, to add to Relay's own expertise at analyzing protein motion.
The company has an ongoing partnership with Roche's Genentech to develop another cancer drug, a SHP2 inhibitor, and has also collaborated with EQRx.
Valo Health (previously Integral Health) is the newest company on this list. Founded in 2019 it came out of stealth mode in September 2020. Supported by Flagship Pioneering, the VC firm that backed Moderna, it's CEO is Flagship general partner and serial entrepreneur Davis Berry.
The company has an end-to-end system and uses its Opal Computational Platform to translate data into potential drugs for development. It is working across a range of therapy areas including: cardiovascular, metabolic, renal, oncology, and neurodegenerative disease.
The company has raised more than $460M to date and completed two major acquisitions in its first year: Numerate, acquired in September 2019, and assets from Forma Therapeutics, acquired in March 2020. Numerate previously created a platform with more than 30,000 models and 70 trillion molecules, which came with the acquisition. From Forma, Valo gained research and development compounds and libraries, a global intellectual property portfolio, and two early discovery labs as well as a skilled team.
Valo already has two in-licensed drug candidates in phase II clinical trials, as well as many in development. One clinical candidate is a drug for diabetic retinopathy and the other is a treatment to improve cardiovascular outcomes (specifically left ventricular dysfunction) after myocardial infarction.
Last year the company announced plans to go public via a SPAC deal backed by Khosla Ventures, but it fell through in November due to “current market conditions” according to the two companies. This year Valo announced a big partnership with Charles River Laboratories to provide its clients with access to AI-guided drug design. It also recently announced plans to acquire Tara Biosystems, developers of a 3D heart tissue modelling platform.