Pipeline & Platform
Using existing approved drug actives, off-the-shelf metered dose inhaler (MDI) components, well established manufacturing processes, and our innovative proprietary particle platform, we have developed MDI combination products that improve upon the consistency, stability, safety and efficacy of existing respiratory therapeutics.
Our lead product, PT003, is a combination therapy of glycopyrrolate, a long-acting muscarinic antagonist (LAMA) and formoterol, a long acting β2-agonist (LABA). Currently we have a comprehensive clinical development program under way. We have completed a Phase 1 safety and PK study in healthy volunteers with PT003 and plan to conduct Phase 2b clinical safety and efficacy studies in 2010 and 2011 to rapidly advance PT003 toward Phase 3 trial initiation in 2012.
We also have generated extensive formulation stability and product performance data with multiple actives to demonstrate conformance to the most stringent regulatory expectations, with no interaction between glycopyrrolate and formoterol in the canister over long periods of storage. We have scaled up and demonstrated excellent control over key processes for powder production and filling of MDI suspensions. Furthermore, the particle platform allows formulation of monotherapy products at 3 mcg or less.
Expected Patient Benefits
Pearl's MDIs have the potential to deliver drugs equally well across a broad class of highly potent actives, both in combination products and in monotherapies. Pearl’s MDIs are superior to many existing MDIs due to their higher lung dose, lower oropharyngeal deposition and consistent performance. These qualities are expected to result in important benefits over existing therapies for patients with mild, moderate and severe COPD and asthma, including:
- More uniform delivery of drug throughout the lungs for more complete therapeutic coverage of the airways
- Less drug deposited in the mouth and throat, which should decrease local side effects such as thrush, dysphonia, or dry mouth
- Lower systemic side effects
- Consistent dose of highly potent drugs delivered to the lungs with each actuation
- No drug-drug interaction in combination products, hence consistent combined dose delivery throughout the patient use period
- Enhanced compliance due to ease of use and familiarity with the MDI dosage form, more reproducible dosing due to the stable suspensions and slower moving ("soft mist") aerosol plume, and hence potentially improved efficacy and therapeutic benefit
- Potentially greater nocturnal control of disease with BID (twice-daily) dosing
- Higher proportion of patient response and increase in inspiratory capacity (ability to exercise/mobilize)