The Problem:

Millions of patients are suffering while existing treatments could help them

Today, less than 25% of the 18.5K recognized diseases in the world have FDA-approved treatments, affecting hundreds of millions of people. While half of these diseases without treatments are considered rare diseases, the combined magnitude of rare diseases is enormous, impacting over 300M people globally. The severity is devastating: 50-80% of people with rare diseases are children, 30% of whom die before age 5. And then there are undertreated diseases — diseases with expensive, burdensome or inaccessible treatments that need better cures. For example, Alzheimer’s, pancreatic cancer, and ALS have approved treatments, but they don’t substantially extend lives.

We know many of these diseases could be treated with many of the 4,000 FDA-approved drugs—at less than 1% the cost of developing a new drug. For instance, an inflammatory disease and a certain type of cancer might share common mechanisms in the body (e.g., related proteins and genes responsible for the disease) and thus may be able to be treated with the same drug.

But systemic barriers prevent this from happening. It’s just not profitable to pursue a new use for an existing drug, especially for rare diseases and the 80% of drugs that are already generic and inexpensive. It’s both expensive to do the trials and companies can’t recoup those costs in sales. Until recently, it has also been logistically impossible to scan across the world’s knowledge of all drugs and all diseases to find matches. So the system focuses on new drugs for profitable diseases, and no one has taken responsibility for systematically uncovering these hidden treatments. Until now. We’re addressing each of the system’s barriers to find the best matches to save and improve lives.  

 

Our Approach: Bringing together AI and medical science to unlock the highest impact cures

We are pioneering a new approach called computational pharmacophenomics to interrogate the world’s biomedical knowledge to find and advance the most promising opportunities across all drugs and all diseases. 

We turn traditional drug repurposing on its head: instead of starting with a specific drug or disease of focus, we make predictions looking across all 4k approved drugs and all 18k recognized diseases to identify the highest potential opportunities. This means that every single disease is in our platform and we are constantly evaluating all opportunities to find the very best ones (note: while emailing us about a specific disease will not speed up our ability to find a treatment for that disease, please do send us drug repurposing ideas that you’ve come across so we can evaluate them next to our AI predictions).

We use multiple AI-powered approaches to create a ranked list of repurposing opportunities:

Knowledge graph-based approaches

We integrate over 100 biomedical datasets, including drug mechanisms, gene interactions, and disease pathways, into an AI-readable map of human biology - a knowledge graph. Our predictive models ingest this map and use it to predict new connections between drugs and diseases

LLM-based approaches

Using best-in-class LLMs, we define reasoning chains that think the same way that our Medical team think, use agentic workflows to pressure test each statement, and then surface those conclusions to answer the question “Could this drug treat this disease?”

Real world evidence-based approaches

We use real world medical data, including prescriptions and medical records, to identify links between already prescribed drugs and new diseases that might not be obvious to a doctor on the ground, but that an AI model can surface in seconds.

In addition to repurposed treatments identified through our AI platform, we also encourage the submission of drug repurposing ideas from patients, physicians, and researchers. 

 

We use human expertise to evaluate and prioritize opportunities that have been highly ranked by our AI methods

Focusing on diseases where the unmet need is highest, we review top-ranked repurposing opportunities. For opportunities with promise, rounds of review and discussion occur, ensuring that we understand the patients and their journey, the medicine and its usage, the existing data, and how Every Cure can help. To do this, we work with expert physicians, disease and patient organizations, payors, and regulators, considering the entire landscape of stakeholders. 

Every promising repurposed treatment is evaluated by an independent Scientific Advisory Council (SAC) for possible endorsement as a program. Currently, we have 6 SAC-endorsed programs.

 

We select programs that we know have high likelihood of success, potential for patient impact, and a feasible path to reaching patients in a timely and resource efficient manner. We aim for the greatest impact possible per dollar spent so we can have the greatest impact with the resources available to us. We do not select repurposing opportunities to advance based on disease prevalence. No disease is too rare. 

 

 

We classify each of our repurposing programs based on what is needed to get the treatment to patients in need: 

Frontier Explorer

This opportunity needs more laboratory studies (e.g., testing on cells) in order to progress to clinical studies

Clinical Gem

This opportunity needs more clinical evidence (e.g., a clinical trial) in order to progress to patients

Unsung Hero

This opportunity has sufficient evidence, but is not being used by all patients who can benefit - it needs help to be approved, put into guidelines, or simply talked about more

We generate the evidence needed to get high-potential opportunities to patients

We partner with disease organizations from the very beginning and work with clinical research organizations, academic labs, research institutes, patient advocacy organizations, and anyone we need to generate the pre-clinical and clinical evidence required to confirm the safety and efficacy of repurposing opportunities. This might include in-silico simulations, lab experiments, clinical trials, or any other research required to get pairs to patients.

When we validate repurposing opportunities, we maximize the uptake of these opportunities by patients

We know that a published study or even a successful trial, is often not enough to get treatments to all patients who can benefit from them. Every Cure works to not only identify repurposing opportunities, but to ensure that every patient possible is benefiting from them. That can mean:

  • Working to expand a drug’s approval with the FDA or other regulatory bodies
  • Attending medical conferences to ensure all doctors possible are aware of new treatments 
  • Working with disease and patient organizations to update treatment guidelines
  • Running marketing campaigns to reach patients directly 

We work on every validated repurposing opportunity in our portfolio until we’re sure that we’ve reached as many patients as possible! 

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