Insilico Medicine, a pioneer in generative artificial intelligence (AI) for drug discovery, is transforming the pharmaceutical landscape with cutting-edge technology that accelerates drug development and enhances therapeutic efficacy.
The company’s business strategy includes selling to other pharmaceutical companies both its generative AI drug discovery software and the investigational drug candidates it designs and assesses with that software. Ten of the top 20 pharmaceutical companies use Insilico Medicine's software, as well as many academic centers.
One of the newest Insilico Medicine deals is a January 2025 agreement for more than $550 million in total value to exclusively license to Stemline Therapeutics, Inc., a wholly-owned subsidiary of the Menarini Group, global rights to develop and commercialize a preclinical small molecule targeting a range of solid tumor cancers. Insilico Medicine developed the molecule using Chemistry42, its generative AI-chemistry engine.
Insilico Medicine’s pipeline is also delivering multiple proofs of the advantages that AI brings to innovation in healthcare. With 22 preclinical drug candidates delivered since 2021 and several programs in clinical trials, 10 have garnered IND approvals. One of the most recent Insilico Medicine pipeline milestones is the dosing of the first patient with ISM633, a novel AI-generated pan-TEAD inhibitor for the treatment of mesothelioma and other solid tumors.
Insilico Medicine, at 10, Leads AI-Drug Discovery
Founded in 2014, Insilico Medicine has an impressive track record of success. Insilico Medicine is also a Cure Collaboration Resident company, with additional offices around the world.
Alex Zhavoronkov, PhD, is the founder and CEO of Insilico Medicine. He confidently believes his company is among the very top generative AI companies worldwide for drug discovery, if not the #1 or #2 slot.
He has good reason. Insilico Medicine’s clinical development includes multiple programs in clinical trials. For one program, a phase 2 clinical trial was completed in China and a phase 2 study is ongoing in the United States. The drug, ISM001-055, is designed to treat patients with idiopathic pulmonary fibrosis (IPF) — a serious lung disease in which the tissue surrounding air sacs becomes stiff and thick, making breathing progressively more difficult.
"Generative artificial intelligence can produce higher-quality therapeutic assets that are likely to be safer and more efficacious for patients than drugs discovered using traditional approaches," said Zhavoronkov. "It's not only faster and better, but there's also most likely going to be a higher probability of success."
Why Insilico Medicine is a company to watch
In an interview with Cure, Zhavoronkov noted several reasons why Insilico Medicine is a company to watch:
Insilico Medicine is an end-to-end generative AI-driven biotech company.
"We go from end to end, utilizing generative artificial intelligence that we developed ourselves," explained Zhavoronkov. "And it's traceable through academic publications."
The company publishes an academic paper about every 10 days. The most influential paper in 2024, published in Nature Biotechnology in March, describes step by step how the Insilico Medicine team discovered a novel fibrosis target and generated a new molecule that it took all the way to phase 2 clinical trials.
The company has been in the AI field longer than most others.
Zhavoronkov described a highly cited Nature Biotechnology paper he published in 2019 — several years before ChatGPT — describing their technology called generative tensorial reinforcement learning.
"The paper showed that we can generate small molecules with the desired properties and take them all the way into mouse studies in 46 days. That was a speed record," said Zhavoronkov. "A lot of people criticized it in the early days, but they're not criticizing us anymore. They're trying to catch up."
They have a global presence.
Insilico Medicine is a Cure Collaboration Residency company, which is one of seven of the company's global locations, and Deerfield Management is one of Insilico's elite investors.
Insilico Medicine's revenues continue to rise.
The company's revenues increased from 2021 to 2023 — rising from $4.5 million in 2021 to more than $30 million in 2022, over $51 million in 2023 and $85 million in 2024. Insilico Medicine had previously sold one drug candidate to another company for close to $1 billion, with $80 million cash upfront. Another sold for close to half a billion dollars, with $12 million cash upfront.
Insilico Medicine has a dedicated focus on diseases associated with aging, which will rise in incidence as the population ages.
The average age of people diagnosed with IPF is 65. If Insilico Medicine can obtain approval of its drug candidate for this fibrotic disease, it should be possible to expand its indications to other fibrotic diseases of aging, such as chronic kidney disease — which is far more common around the world.
"If you're going to conduct a clinical study for chronic kidney disease, it's much more expensive than going after IPF. But if you can demonstrate a really clear mechanism and a very strong antifibrotic, you can do an indication expansion and go after other diseases," Zhavoronkov maintained. "If we show efficacy in IPF, we should be able to show efficacy in multiple other forms of fibrosis, and possibly other age-related diseases."
The company is committed to sustainability.
"If we are to live on this planet, we need to invest in a more sustainable future," asserted Zhavoronkov. Insilico created a Gen AI consortium to develop and promote open-source technologies for environmental sustainability. The consortium is using AI to enhance carbon dioxide capture, hydrogen storage, agrochemistry and lubricant production.
Zhavoronkov compliments Deerfield Management and its commitment to fuel biotechnology. "They are the elite of the elite investors," he contended. "We love being in their building. Deerfield created the Cure Experience, which gives you a premium location in New York as well as a platform to become whatever you want to be. They create and incubate companies and invest in public markets. I think being part of the Cure Experience and the Deerfield ecosystem should be the goal of every biotech."