Towards Understanding & Reversing Motor Neurone Disease

Vision
Dual-acting, Retinoic Acid Receptor-Modulators as lead potential drugs for developing a rational and effective new treatment regime for ALS.
The Problem
The number of ALS cases across the globe will increase from 222,801 in 2015 to 376,674 in 2040, representing an increase of 69%.
Investment
We seek partners and investors for the journey towards understanding, treating and potentially reversing neurodegenerative diseases.

RAR-M Therapeutics is a drug development company working towards understanding, treating and potentially reversing neurodegenerative diseases.

Our primary interests in motor neurone (Amyotrophic Lateral Sclerosis or ALS) disease through a novel dual-acting lead candidate, which may also be valuable in treating the closely linked with frontotemporal dementia (FTD) disease. 

With novel intellectual property in this area, RAR-M is raising significant early stage investment to take the lead candidate through the preclinical stages and on into clinical trials.

RAR-M’s strategy involves initially targeting ALS (and FTD) as an orphan indication and major unmet need, with the longer term view of developing global blockbusters for Alzheimer’s, Parkinson’s and other neurodegenerative and related diseases.

RAR-M Therapeutics is also actively looking at a wider scientific understanding of the diseases, their causes and links between the different types of clearly related diseases.

Our novel drugs are not only being developed to try and improve the quality of life of ALS suffers, but are also being used as biological probes for developing a deeper understanding of the causation in ALS.

The Problem

Although ALS is relatively rare, the socioeconomic significance of the disease is considerable.

0
million

Americans

Currently 5.2 million Americans (1.6% of the population) suffering from Alzheimer’s

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%

Global ALS Increase

The number of ALS cases will increase from 222,801 to 376,674 in 2040

$
0
billion

Cost Increase by 2021

The cost of Alzheimer’s treatments is set to expand to $3.2 billion by 2021

The number of ALS cases across the globe will increase from 222,801 in 2015 to 376,674 in 2040, representing an increase of 69%. The worldwide annual incidence of ALS is about 1.9 per 100,000, with uniform rates in Caucasian populations and lower rates in African, Asian and Hispanic populations. The number of ALS cases is projected to move away from developed countries towards more developing nations.

Current Treatments

As of December 2019, Riluzole (Rilutek, Sanofi-Aventis) and Radicava (Edavarone, Mitsubishi Tanabe) are the only FDA approved drugs on the market. The former only has a modest effect on prolonging life in ALS patients (for up to 2-3 months when taken for a 18-month duration), while the latter also has a modest impact and costs $148,000 per year.

Additionally, Radicava is reported to negatively affect patients’ overall quality-of-life. Riluzole is generally well tolerated, but has common side-effects like asthenia, nausea, gastrointestinal upset and abnormal liver function.

More broadly, the estimated market size for Alzheimer’s treatments suggests a market size of between $4-9 billion pa in 2019. There are currently 5.2 million Americans (1.6% of the population) and a comparable proportion of the global population suffering from Alzheimer’s.

No new drug-based treatments have been approved in more than a decade. Parkinson’s Disease is the second most common neurodegenerative disease with approximately 1.2 million people in the United States affected by 2030. The cost is set to expand to $3.2 billion by 2021. According to analysts game-changing treatments will not be available for the foreseeable future.

Vision

ALS and FTD represent a severe unmet medical need.

These diseases have a tragic impact upon patients and impact the lives of carers and society as a whole. Together with novel therapies, there is profound need for a deeper understanding of the underlying causes of ALS, as with all other neurodegenerative diseases.

We neither fully understand the links between these diseases, nor their root cause(s) and, even worse, suffer from a complete lack of effective treatments for all of them.

Our vision is to employ new, dual-acting, retinoic acid receptor modulators (RAR-M) as lead potential drugs for developing a rational and effective new treatment regime for ALS and meeting that therapeutic need.

In the longer term, we will use the lead candidate to investigate and characterise its impact on the underlying mechanisms of ALS and potentially use this knowledge to target the wider range of neurodegenerative diseases.

Our core development team have been working together as part of a close scientific collaboration for a number of years.

Learn more about our background,
people and key papers

About
Supported by

Investment Opportunities

We are currently seeking supportive partners and investors for the development journey ahead, to complete preclinical work (est. completion April 2021) and Phase 1 trials (est. completion December 2021).

If you are interested in investment opportunities please contact us

Key Partners