Junior Biotech Utilizes AI To Shorten Drug Discovery Timelines, Ultimately Saving Lives

The urgent need for innovative technologies to accelerate the development of life-saving cancer therapies has never been more critical. With millions of lives and billions of dollars at stake, the potential to shorten drug development timelines is paramount. DNA-damage response (DDR)-based therapies, which target cancer cells' repair mechanisms, are emerging as a highly promising approach. When combined with artificial intelligence (AI), the potential for breakthrough discoveries in this field is immense. 

DDRs are naturally occurring mechanisms that detect and repair DNA damage within our cells. Many cancers harbor a defect in these natural repair mechanisms allowing mutations to accumulate and grow into life-threatening cancer. A stunning 75% of solid tumors harbor a DDR defect.

Recognizing this opportunity, Rakovina Therapeutics Inc. (TSX-Venture: RKV), a Canadian biotechnology company, has strategically focused on the intersection of AI and DDR-based cancer drug development. Recent high-profile industry transactions lend credence to Rakovina's approach, positioning it as a key player in the future of cancer treatment and perhaps important partner to industry leaders today.

Deep Docking AI

Founded in 2021 by a close-knit team led by serial biotech entrepreneur Jeffrey Bacha and University of British Columbia (UBC) Prof. Mads Daugaard, Rakovina Therapeutics has quickly gained attention for its innovative approach to cancer therapy. The company has recently secured exclusive access to the Deep Docking AI Platform, developed by UBC Prof. Artem Cherkasov, allowing the Company to rapidly analyze billions of molecular structures to identify potential drug candidates targeting DDR mechanisms, common in many cancers.

Deep Docking, a computational modeling technique, is transforming drug discovery by enabling the rapid evaluation of vast numbers of compounds. This deep learning (DL) method outperforms traditional machine learning, allowing AI machines to screen billions of drug candidates in months rather than years. The platform’s ability to accelerate virtual screening up to 100-fold and its remarkable 6,000-fold enrichment in candidate molecules showcase its potential to significantly speed up the discovery of new cancer therapies.

One quote from Dr. Cherkasov summarizes the unprecedented speed of the platform:

“The team behind Deep Docking partnered with Nvidia, Dell and UBC Advanced Research Computing (ARC) to identify potential COVID-19 therapeutics from large libraries. Even with a supercomputer, it would take years to screen 40 billion compounds with traditional methods; with Deep Docking™ we did it in 20 days. It is a paradigm shift.” (https://strategicplan.ubc.ca/ai-technology-enables-screening-of-billions-of-compounds-to-identify-potential-covid-19-drugs/) 

Rakovina’s partnership with Dr. Cherkasov grants the company exclusive rights to all novel drug candidates generated through the Deep Docking AI Platform. To that point, sub $10 million market cap Rakovina stands at the forefront of DDR-based therapies, an area with an estimated $18 billion addressable market by 2030. This massive multi-billion addressable market gives the company almost unheard-of upside potential if all goes to plan. The company is screening a vast database of over 7 billion drug candidate molecules continuously screened with Deep Docking, providing crucial structure-activity relationship (SAR) data for drug development.

Further Differentiated

A key differentiator for Rakovina is its established in-house laboratory infrastructure, developed in partnership with UBC. This infrastructure is essential for validating AI-identified compounds and advancing them to human clinical trials through pharmaceutical partnerships. The seamless integration between AI computations and wet lab operations sets Rakovina apart, enabling a more efficient path from discovery to clinical development.

Rakovina's current focus is on developing advanced PARP inhibitors, a type of DDR-based therapy, with the potential to reduce side effects and improve treatment efficacy. First-generation PARP inhibitors have already seen commercial success, generating $2.3 billion annually for treating certain cancers. However, these inhibitors are limited by side effects and are ineffective against cancers that spread to the brain. Rakovina is working on next-generation PARP inhibitors that are selective for PARP-1, offering the potential to treat cancers with few, if any, meaningful therapeutic options.

The strategic move towards AI-driven DDR-based therapies is further validated by recent industry transactions, namely:

• Merck (NYSE: MRK) signed a license agreement with Hengrui for its preclinical PARP inhibitor in a deal valued at up to $1.5 billion, including $161 million upfront. 
• Roche (OTC: RHHBY) entered into a $1.2 billion license agreement with Repare Therapeutics for its preclinical ATR inhibitor, which included $125 million upfront. 
• Novartis (NYSE: NVS) penned an even earlier stage deal, teaming up with Artios in a deal worth up to $1.3 billion, including $20 million upfront, to collaborate on discovering new DDR-based therapies.

These high-value deals underscore the market’s recognition of the potential for DDR-based therapies to transform cancer treatment, further bolstering Rakovina's strategic direction.

The Pipeline

Rakovina has identified some of its top candidates and is moving them forward towards pre-clinical studies. As shown by the aforementioned industry activity, even drugs in the discovery stage can grab the attention (and investment) of some of the world’s top drugmakers. 

Smart money understands the catalyzation potential as evidenced by 37.3% of the company’s stock being held by a combination of insiders and privately held Edison Oncology Holding Corp.

The Team

Rakovina’s leadership team reads like something straight from a blue chip, not a company with a CDN$6 million market cap. The company is led by Jeffrey Bacha, who has more than 25 years of experience as founder and executive of multiple companies across the health sector such as Kintara Therapeutics (NASDAQ: KTRA), XBiotech, Inc. (NASDAQ: XBIT), Inimex Pharmaceuticals and Inflazyme Corp.

Mads Daugaard, PhD, serves as the company’s President and Chief Scientific Officer. He is recognized as a world leader in translational cancer research with expertise in DNA-damage response mechanisms and therapeutics targeting DNA integrity. 

Dr. Cherkasov sits on Rakovina’s Scientific Advisory Board and is also the company’s Senior AI & Medicinal Chemistry Advisor. He has co-authored 200+ research papers, filed 80+ patents, and licensed 8 drug candidates to major companies, amongst his list of accolades.

Also on the SAB is Dr. Leonard Post and Prof. Petra Hamerlik, Dr. Post has 35+ years of drug development and leadership experience in the pharmaceutical and biotechnology industry spanning companies of all sizes. He was previously Chief Scientific Officer of BioMarin (NASDAQ: BMRN), Senior Vice President of research and development for Onyx Pharmaceuticals, acquired by Amgen (NASDAQ: AMGN) for $10.4 billion in 2013 and Vice President of Discovery Research for Parke-Davis Pharmaceuticals. Prof. Hamerlik is the former Principal Scientist and CNS Cancer Bioscience Lead at AstraZeneca (NASDAQ: AZN) where she contributed to the development of brain-penetrant DNA repair inhibitors for targeting primary brain tumors and brain metastases

This is merely a sampling of the quality of the people directing Rakovina. See the whole team here: https://www.rakovinatherapeutics.com/wp-content/uploads/2024/08/Rakovina_Investor-Presentation_Q3Aug2024-PDF36.pdf. 

Expedite the Process, Minimize the Risk

Rakovina's business model is designed to capitalize on AI-driven drug discovery while minimizing the risks associated with traditional drug development. The company's compound screening and lead optimization phase, typically taking three years, can now be reduced to just three to four months, thanks to AI. 

This accelerated timeline is critical, as patients cannot afford to wait years for life-saving treatments. By focusing on DDR-based therapies and leveraging AI, Rakovina is well-positioned to bring new cancer treatments to market faster than its competitors or partner with a pharma that aims to capitalize on R&D optimization, ultimately saving lives and reducing the financial burden of cancer treatment.

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