Will the green shoots in 2024 lead to big change for rare diseases?

Everyone seems to agree it’s been a tough time in biotech and drug discovery, but the general consensus from the beginning of the year is positive.  

BioCentury are reporting an increasing pace of significant mergers and acquisitions, with only 2x  $1bn+ deals in Q3 last year, but an incredible 9x in Q4.  

Evaluate Pharma agrees the outlook is encouraging. While its 2024 Preview Report focuses on the largest commercial potential in what it calls a “pretty dynamic” market, it’s noteworthy that they are shining a spotlight on some pending approvals for rare diseases, such as sickle cell and lupus.  

Indeed, Sanofi just acquired Inhibrx for around $2.2bn, adding a phase two asset for a rare genetic disease to its portfolio.  

And that’s just an example of a trend the FT is reporting on. As pharma companies struggle to fill their pipelines ahead of some significant patent expiries in the coming years – mergers and acquisitions look to be the way forward. And what’s important in the current climate is having good clinical data – “investors remain cautious about backing earlier-stage companies”, reports Nicholas Megaw from the FT.  

We are delighted to see that rare disease drugs are in play. At Ambrose, we have two assets in the wings with phase 2 clinical data, and with our specialist insights into regulatory stimuli for rare diseases – we believe we are well positioned in the current market. But we’re not just relying our expertise, we have partnered with BioPhy, who have a remarkable 80% accuracy rate for predicting clinical trial successes – to make sure that we focus on the right assets. 

Just 5% of rare diseases have an FDA-approved treatment, and it’s great to see some options coming through the pipeline. But while the options are scattered in the industry, rare disease is our focus.