Drug repurposing, also known as drug repositioning is an approach to identify a novel medical indication for a drug
Drug discovery companies are realizing that AI that enables the repurposing of current drugs can save time and money, since AI drug repurposing has the potential to offer them enormous business benefits by enabling the analysis of pre-existing massive databases, by accelerating research and development, by saving costs and by promoting effective decision making.
For this reason, AI startups are going after the “viagra effect”, namely the serendipity effect of when you design a drug to be a treatment for cardiac chest pain (and even if you get disappointing results in early trials) you have an interesting side effect: increased erections everywhere.
In particular, since FDA approval on March 27, 1998 Viagra brought in about $1.6 billion in 2016 global sales (Pfizer) and also made many people happy. And despite the fact that it’s hard to beat the legacy of this little blue pill, AI startups for drug repurposing are trying right now through drug repurposing the cure for cancer, the elixir of life or the one pill to cure all.
Are they gonna make it? “Time will explain.”
Acurastem’s (California, US, 2016) iNeuroRx® technology uses living motor neurons and advanced informatics as a tool for the identification and development of novel treatments. So far, they have identified 37 compounds (already approved by FDA for other disorders) that improve nerve cell survival.
On May 25, 2021, AcuraStem received an Amyotrophic Lateral Sclerosis (ALS) award from the Department of Defense, Congressionally Directed Medical Research Programs (CDMRP) totaling $1 million, for it’s drug candidate AS-202 which has potential to be a disease-modifying therapeutic for "sporadic" ALS, to rapidly advance this treatment towards the clinic
AcuraStem has raised a total of $5M.
BioXcel Therapeutics (Connecticut US, 2017) utilises AI to find applications for existing approved drugs or clinically validated candidates, allowing researchers to develop a pipeline of product candidates in immuno-oncology, neuroscience and rare diseases.
On Sept. 15, 2021, BioXcel announced data from its ongoing Phase 1b/2 trial of BXCL701, an oral innate immunity activator, in metastatic castration-resistant prostate carcinoma (mCRPC) in combination with pembrolizumab, with 26% of patients in the adenocarcinoma cohort achieving a composite response and all responders experiencing a decrease in tumor size. The data lay a strong foundation for the broad potential of BXCL701 in combination with pembrolizumab (sold under the brand name Keytruda, a humanized antibody used in cancer immunotherapy that treats melanoma, lung cancer, head and neck cancer, Hodgkin lymphoma and stomach cancer), including for heavily pre-treated mCRPC patients with adenocarcinoma, an aggressive tumor for which there are few available treatment options.
On May 10, 2021, they granted FDA breakthrough therapy designation for BXCL501 (an investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine designed for the treatment of agitation and opioid withdrawal symptoms) for the acute treatment of agitation associated with dementia, schizophrenia and bipolar disorders.
Biovista is a top 20 AI startup (Virginia US, 1999) applying their AI platform (Project Prodigy) to develop their pipeline of repositioned drug candidates in disease areas such as neurodegenerative diseases, epilepsy, oncology and orphan diseases. The firm has made portions of Project Prodigy publicly accessible through their data and hypothesis exploration tool called VIZIT. In June 2020, VIZIT provided answers to over 20,000 unique searches made by scientists and drug developers in over 75 countries.
On Aug. 25, 2020, Bioscience firm applied AI and data science to identify existing drugs Atozet and Cablivi as potentially effective therapies for blood clots and inflammation caused by COVID-19.
Additionally on Sept. 15, 2021, Biovista and Diginova Health Solutions (that empowers proven and enterprise-ready digital health technologies, providing commercial and technical support) announced their deal to deliver AI-driven personalised medicine analytics to over a quarter of a billion patients in Asia. Diginova will offer Biovista Personalised Medicine's market-leading AI Health Shield analytics direct-to-consumer, where the consumers are doctors, patients, their family caregivers, and national/regional health systems.
Delta 4 (Vienna Austria, 2018) uses AI to conduct in silico screening prior to experimental screening. Delta 4 leverages on a proprietary computational analytics platform, combined with straightforward biomedical testing and clinical validation of candidate drugs. Its first therapeutic area is Focal Segmental Glomerulosclerosis (FSGS), a devastating renal disease
with high and urgent medical need.
In late 2020, Delta4 filed four patent applications for compounds and combinations of compounds for the treatment of FSGS and COVID-19. In 2021, drug candidates from both programs have been biologically validated and in-vivo tested and their first FSGS drug candidate is now ready to move into Phase II of clinical testing.
Healx (Cambridge UK, 2014) uses AI to match existing drugs with rare diseases. Their AI platform uses NL processing to extract disease knowledge from published sources and to complement biomedical databases and proprietary curated data. Healx’s data is integrated in the form of the largest, rare disease-focused Knowledge Graph, that shows prioritised hidden and novel connections between drugs and diseases. They collaborate with academic, patient and industry groups: portfolio. Healx’s most advanced project so far is with the US patient group FRAXA Research Foundation (started in 2016), and their drug candidate for fragile X syndrome – a genetic condition that causes a range of developmental problems – is set to enter Phase I/IIa clinical trials later in 2021.
On August 5, 2021, it was announced that Healx will work with Ono Pharmaceutical (one of the largest pharmaceutical companies in Japan) to find multiple new therapeutic indications for Ono’s proprietary assets.
Healx has raised a total of of $67.9M.
Lantern Pharma (Texas US, 2013) is using AI (RADR®, or Response Algorithm for Drug Positioning & Rescue is Lantern’s ML-based platform) to analyse genetic signals and molecular markers for patient response to drugs (small molecules) and is allowing researchers to find clinical uses for validated cancer treatments whose development has been discontinued. RADR uses transcriptomic, genomic and drug sensitivity data: from publicly available databases, from commercial clinical studies and trials, and their proprietary data generated from ex vivo 3D tumor models specific to drug-tumor interactions.
They have analysed so far over 500 million oncology-specific clinical and preclinical data points, more than 144 drug-cancer interactions and over 13,200 patient records from five data bases, one of which is their internal data base. Their long-term objective is to collect and analyse over one billion oncology-specific clinical and preclinical data points to further enhance the prediction power of their platform.
On August 31, 2021, the FDA has granted orphan drug designation to the small molecule and next-generation alkylating agent, LP-184 of Lantern, for the treatment of patients with glioblastoma multiforme (GBM) and other malignant gliomas. LP-184 has been shown to preferentially cause DNA damage in cancer cells that have over-expression of certain biomarker or that harbor mutations in the DNA repair pathway. This news follows the recent announcement of the FDA granting LP-184 ODD for the treatment of pancreatic cancer.
Lantern Pharma has raised a total of $68.7M.
Pharnext (Issy-les-moulineaux France, 2007) is AI-screening and repositioning company and with their platform PLEOTHERAPY™ systemise the identification and development of new synergistic combinations of repositioned drugs for diseases with high unmet medical needs. These new therapeutic entities are called PLEODRUG™.
A new partnership (as of June 2021) between Pharnext and Alpha Blue Ocean will help support the Phase 3 clinical trial PREMIER, which is testing Pharnext’s investigational therapy PXT3003 for Charcot-Marie-Tooth disease type 1A (CMT1A), caused by a genetic mutation that leads to a loss of myelin, the fatty substance that surrounds nerve fibers and enhances nerve cell communication. PXT3003 is a combination of three approved treatments — the muscle relaxant baclofen, naltrexone used to treat opiate and alcohol addiction and the laxative sorbitol — that seeks to reduce PMP22 levels and increase myelin production.
Pharnext has raised a total of $157.6M.
Qrativ (Massachusetts US, 2017) launched by the Mayo Clinic and nference (powered by AI synthesizes the world’s exponentially growing biomedical knowledge — public and proprietary scientific, clinical, regulatory and commercial data sets) uses the Darwin.ai platform. That combines nference’s knowledge synthesis platform with the Mayo Clinic’s medical expertise and clinical data. In this way Qrativ partners can leverage the drug-repurposing platform to search for all potential uses of a drug candidate, including identifying potential rare disease indications and finding subsets of patients who are most likely to respond to a given candidate.
Qrativ has raised a total of $8.3M.
Recursion Pharmaceuticals (Utah US, 2013) top 20 AI pharma utilises AI to conduct experimental biology at scale by testing thousands of compounds (new chemical entities and from a repurposing library) on hundreds of cellular disease models in parallel. They reengineering drug discovery by combining automation, ML and the world’s largest biological image dataset with a highly cross-functional team to discover transformative new treatments.
Their new supercomputer is BioHive-1, that will enable faster training and iteration of their ML algorithms and the ability to train on the totality of their nearly 8 petabytes of relatable biological data. Moreover, Recursion finished the first quarter of 2021 with: 4 clinical stage programs, 4 preclinical programs, 4 late discovery programs, and 25 early discovery programs and an impressive partnership with Bayer (ten programs with possible development and commercial milestone payments of more than USD 100 million per program plus royalties on future sales).
Recursion Pharmaceuticals has raised a total of $465.4M and has a post-money valuation in the range of $1B to $10B as of Sep 10, 2020.
BenevolentAI (London, UK, 2013) is a global leader of AI and computational medicine technologies. While the company is working on its own drug candidates, it also has various partnerships including with the big pharma companies AstraZeneca and Novartis.
Last year hit the news when it discovered a potential treatment for Covid-19 the existing rheumatoid arthritis drug baricitinib (currently marketed by Eli Lilly as Olumiant to treat rheumatoid arthritis in adults), in a process that took just three days to complete and over 370 AAK1 inhibitors were identified. Baricitinib has shown to improve and speed up the recovery of hospitalized patients with Covid-19. Now, the medication has been approved as a COVID-19 treatment in the U.S. and Japan. The European Medicines Agency has also begun evaluating baricitinib for use against the coronavirus.
BenevolentAI has raised a total of $292M.
Korean AI-based drug discovery companies such as Oncocross, Standigm, and Syntekabio have built partnerships with domestic pharmaceutical firms for drug repositioning
Oncocross (Seoul South Korea, 2015) utilises AI to analyse gene expression patterns, allowing researchers to develop biomarkers, repurpose drugs and find novel drugs. The startup aims to increase the number of FDA approved drugs by collaborating with big pharmaceutical companies, both global and Korean (Jeil Pharma, ST Pharma, Korea Pharma, Boehringer Ingelheim and many more), and for this it has developed two AI platforms:
ONCO AI — to screen for optimal drug candidates in preclinical or clinical stages. And
ONCOfind AI — to screen and identify the most suitable biomarker for anti-cancer drug compound candidates.
On March 22 2021, Oncocross signed a memorandum of understanding with Daewoong Pharmaceutical that aims to expand the indications of Enavogliflozin (a drug originally targeting diabetes, repurposed to metabolic, heart, and kidney disease) and DWN12088 (potential as an anticancer drug while developing it as a polygenic risk score (PRS) treatment for intractable fibrosis in the lung, kidney, and skin.
Oncocross has raised a total of 25.5 billion won ($22.6 million).
Standigm (Seoul South Korea, 2015) has proprietary AI platforms encompassing novel target identification to lead generation to generate commercially valuable drug pipelines. Pursuing full-stack, AI-driven industrialising drug discovery, Standigm has achieved the automation of molecular design workflow through DarkMolFactory, and the automation effort has been expanding to the whole drug discovery process on the basis of Standigm AI platforms, including Standigm ASK for target discovery, Standigm BEST for lead design and Standigm Insight for drug repurposing.
In July 2019, Standigm signed an agreement with SK Chemicals (a Korea-based company that is engaged in the manufacture of chemical products) to co-develop therapies to treat non-alcoholic fatty liver and rheumatoid arthritis. In January 2021, the two companies found a new rheumatoid arthritis indication through drug repurposing and SK Chemicals filed a patent. Standigm is also working on an anticancer drug candidate targeting T cells with Hanmi Pharmaceutical (2021), by identifying pre-clinical substances based on AI platform technology.
Syntekabio (2009) is a Korean AI drug development company based on genome big data. Syntekabio is using DeepMatcher, an AI drug development platform that combines new drug development technology and genomic big data technology, such as biomarkers for individual cancer drug screening and disease susceptibility by immunotyping, pharmacogenomic typing, and predicting multi-omics information-based drug adverse effects, etc.
In February 2020, Syntekabio found the possibility of Covid-19 treatment in leukotriene receptor blocker Zafirlukast and platelet aggregation inhibitor Sulfinpyrazone, using its AI solution, “Deep-matcher.” Syntekabio plans to develop a Covid-19 drug STB-R011 by using the two drugs in combination.
Syntekabio has raised a total of $877K
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