Our approach
We're on a mission to get new drug treatments into the hands of rare disease patients. Quickly. Safely. Cost-effectively.
Rethinking rare disease
We accelerate drug development by strategically acquiring clinical-stage assets with the potential for approval in rare disease indications where there is no current drug treatment.
Bringing stalled drugs to market
Our core business focuses on bringing rare disease drugs to market. Quickly. Safely. And cost-effectively.
With 95% of rare diseases having no approved treatment, it is clear that existing approaches aren't enough. Mainstream drug development costs are high and promising drug candidates can be left on the shelf; rare diseases regulatory incentives can provide new opportunities for them.
At the current rate of development, it is estimated that it will take over a century before every rare disease has an approved drug treatment.
So, we are using our expertise and deep understanding of rare diseases to take a different approach.
Using our in-house asset evaluator, we identify, acquire, develop, and commercialise drug candidates that have already generated safety and efficacy data that show potential for regulatory approval in a rare disease indication where there is no current approved drug treatment.
We reinvigorate these assets by leveraging our knowledge of rare disease drug development and regulatory pathways, establishing relevant clinical study endpoints and engaging with patients from the start. Rare disease regulatory pathways can enable approvals based on Phase 2 study results. This is how we hope to bring new drug therapies to market faster and more cost-efficiently than mainstream drug development.
Our growing drug development pipeline focuses on indications where there is no existing, approved drug treatment.