An innovative biotechnology company exploring novel therapeutics for neuroscience indications had developed a promising new chemical entity for the treatment of a severe pediatric genetic disease. However, numerous drug delivery challenges threatened the realization of the Fast Track status designation they had received due to promising early stage results. By leveraging a softgel technology, the customer was able to overcome a number of potential hurdles and expeditiously enter the Phase II/III clinical trial.
The new chemical entity (NCE) itself was a high molecular weight, poorly soluble and poorly permeable BCS Class II compound in a Liquid Filled Hardshell (LFHS) formulation. The original formulation presented a number of challenges including the inability to scale for cGMP clinical material, a reduced therapeutic profile and an inadequate shelf life. In addition, the project had deliverable timeline of 120 days.
The Catalent Solution
Catalent’s expert team partnered with the customer to quickly discover a superior alternative drug delivery solution using RP Scherer Softgel technology that would enable the NCE to take advantage of the Fast track designation. At every stage, Catalent was able to rapidly deliver a faster, better solution.
In addition to enhancements in bioavailability, the softgel formulation provided significant improvements in stability, an increase in dosage, and a reduction in capsule size — all delivered within a 4 month time frame (Figure 2).
The synergy between the companies brought together experience, resources, and innovation to enable this NCE to progress rapidly into Phase II/Phase III, ultimately allowing them to realize the Fast Track designation and bring a better therapy closer to the market.