The Future for Disease Modifying Therapies for Lysosomal Disorders

Pharmaceutical Executive previously covered FDA’s efforts to expand the range of data allowed in clinical trials, including the use of the Bayesian method. This style of data collection allows for clinical trial data to be examined alongside existing, relevant data.

This expansion of data is especially important for clinical trials in any rare disease space that have limited patient populations. While clinical trials in this area still present unique difficulties, regulatory changes have provided solutions to issues related to limited data-sets.

Pharmaceutical Executive spoke with Lisa Bollinger, chief medical officer at Polaryx. The clinical-stage biotech focuses on treatments for rare, pediatric lysosomal storage disorders. Due to its focus, Polaryx has been able to update its trial design thanks to the previously mentioned regulatory updates.

Bollinger discusses the regulatory landscape in this area, along with the style of trial design that the company is utilizing and some of the unique challenges they still face.

Pharmaceutical Executive: What is the future of disease modifying therapies for lysosomal storage disorders?
Lisa Bollinger: The future looks bright. It's a very difficult and complex disease to treat, it's hard to address all the issues. So, when we look at lysosomal storage diseases, we know that there are more than 70 different lysosomal storage diseases, and each of them is driven by a single gene mutation.

However those single gene mutations also have multiple forms of those mutations for that single gene being mutated. How do you address the different aspects?

For example, you could have an enzyme that just doesn't work, you could have an enzyme that's not produced at all, or you could have an enzyme that's produced, but there's a protein missing in a cell membrane that doesn't allow that enzyme to get across that membrane.

It's more than just solving one single problem. So, what you need to do is think about each of those gene cell mutations, you have to come up with a modifying drug that fixes that particular single gene.

With PLX 200, our drug works high enough up in the cascade that we do think that we may see a treatment effect across different lysosomal storage diseases, but that's why we must study each different one.

They are different diseases, and so having a single disease modifying therapy is really difficult. It's hard to hit more than just one of these diseases.