Profile

Insilico Medicine, a global clinical-stage biotechnology company powered by generative AI, connects biology, chemistry, and clinical trial analysis using next-generation AI systems. The company has developed AI platforms that utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques for novel target discovery and generating novel molecular structures with desired properties. The platform has been licensed to over 40 pharma companies and has been used internally to produce a diversified pipeline of 31 programs for 29 drug targets — including cancer, fibrosis, immunity, central nervous system diseases, infectious diseases, autoimmune diseases, and aging-related diseases. Six of these programs have reached clinical stages. 

The Company’s lead drug, designed to treat the chronic, and often fatal, lung condition, idiopathic pulmonary fibrosis, is the first drug designed by generative AI for an AI-discovered target to reach Phase II trials with patients – a major milestone for the industry. The company just published a paper in Nature Biotechnology that tells the story of this lead AI drug. (https://www.nature.com/articles/s41587-024-02143-0) 

A number of small molecule therapeutics produced by Insilico’s AI platform have been licensed by pharma partners, including a $1.2b deal with Sanofi to develop drugs for up to 6 targets; an $80m deal with Exelixis for a potential best-in-class USP1 inhibitor for BRCA-mutated tumors; and a deal worth up to $500m with Menarini for a KAT6 cancer inhibitor to treat breast cancer. 

In addition to Insilico’s partnerships with pharma, the company also partners with many academic researchers to advance its technology and find new cures. For example, an international research team – including from Johns Hopkins, Harvard, 4B Technologies and the University of Zurich – along with the organization Answer ALS – used Insilico’s AI target discovery engine, PandaOmics, to identify 28 new targets for ALS. Three of those targets have approved and marketed drugs for other diseases. 4B Technologies is now launching an investigator-initiated trial with ALS patients for one of these drugs – and has completed enrollment. From discovery to clinical trials with patients in just two years. And Insilico is also working closely with Igor Stagljar’s lab at University of Toronto, combining the company’s Pharma.AI platform with his live cell assays to develop drugs for “undruggable” cancer targets.

Customer Insights

“We are delighted to enter a collaboration with Insilico that harnesses the power of generative AI as a leader in the field, to explore a promising new treatment approach and potentially unlock transformative new cancer therapies,” said Elcin Barker Ergun, CEO of the Menarini Group.

“We believe preclinical data on ISM3091’s potent anti-tumor activity, tolerability, and pharmacokinetics set the compound apart from competing USP1 inhibitors and make it an important addition to Exelixis’ growing clinical-stage pipeline,” said Dana Aftab, Ph.D., Executive Vice President, Discovery and Translational Research and Chief Scientific Officer, Exelixis.  “Following the FDA’s clearance of Insilico’s IND earlier this spring, we’re looking forward to accelerating phase 1 trial enrollment.”

“We are extremely impressed by the expert team of scientists at Insilico and the sophistication of the technology and chemistry,” said Andy Stamford, PhD, Vice President of Chemistry at Deerfield Discovery and Development at Deerfield Management. “We believe the platform has the potential to broadly impact drug discovery and break ground on novel targets across multiple disease indications.”

Key Executives

• Alex Zhavoronkov, PhD, Founder and CEO


• Feng Ren, PhD, Co-Founder and Chief Scientific Officer


• Michelle Chen, Chief Business Officer, Alex Aliper, PhD, President


• Sujata Rao, MD, Chief Medical Officer


• Petrina Kamya, PhD, Global Head of AI Platforms

To view their executive page, click here.

Board Members

• Alex Zhavoronkov, PhD, Founder and CEO


• Min Fang, Non-executive Director


• Kan Chen, PhD, Non-executive Director


• Feng Ren, PhD, Executive Director, Co-CEO, Chief Scientific Officer

To view their executive page, click here.

Investors

The company has raised over $400 million in funding from investors including B Capital Group, Warburg Pincus, BHR Partners, Deerfield, and BOLD Capital.

Key Milestones

Insilico Medicine is a pioneer in using generative AI for drug discovery and development. The Company first described the concept of using generative AI for the design of novel molecules in a peer-reviewed journal in 2016. Then, Insilico developed and validated multiple approaches and features for its generative adversarial network (GAN)-based AI platform and integrated those algorithms into the commercially available Pharma.AI platform, which includes generative biology, chemistry, and medicine and has been used to produce a robust pipeline of promising therapeutic assets in multiple disease areas, including fibrosis, cancer, immunology and aging-related disease, a number of which have been licensed. Since 2021, Insilico has nominated 18 preclinical candidates in its comprehensive portfolio of over 30 assets and has advanced six pipelines to the clinical stage. In March 2024, the Company published a paper in Nature Biotechnology that discloses the raw experimental data and the preclinical and clinical evaluation of its lead drug – a potentially first-in-class TNIK inhibitor for the treatment of idiopathic pulmonary fibrosis discovered and designed using generative AI currently in Phase II trials with patients – the first such drug to reach this milestone.

News and Press Releases

• May 2024: Insilico Medicine introduces nach0: A one-stop LLM for chemical and biomedical tasks (Read).


• May 2024: Insilico Medicine completes first-in-patient dose for its novel QPCTL inhibitor for solid tumors in collaboration with Fosun Pharma (Read).


• May 2024: Insilico Medicine publishes CDK8/19 novel inhibitor powered by generative chemistry platform to treat multiple cancers (Read).

To view their full press release, click here.

Competitors

Competitors include: BenevolentAI, Exscientia, Insitro, Recursion Pharmaceuticals, Relay Therapeutics, Schrödinger, Verge Genomics, and Valo Health.

Click here for a comprehensive analysis.

Corporate Responsibility

One year ago, Insilico Medicine opened the largest AI research and development center in the Middle East – the Insilico Medicine Generative AI & Quantum Computing R&D Center, located in the UAE which features a team of around 70 biotechnology experts from around the world. Now, as part of a new “Generative AI for Sustainability” vision, Insilico’s platform is also being used to develop more sustainable chemicals, fuels, and materials in partnership with Syngenta, Luberef and others. Learn more, click here.