Several milestones have been reached or exceeded . . .
1. The submission of the "unaudited interim report on the 6 month dog inhalation study" to the FDA not only clears the way for Arikace, but sets up a cornerstone safety datapoint for a liposome masterfile submission later on. The submission of the unaudited report starts opens the door for the CRO to submit an important abstract for scientific presentation. Look for that abstract to appear in the next 4-6 months.
2. The statement that INSM will be adding regulatory people in the near future to augment US, Canadian and European submissions for drug approvals was important information. The addition of the marketing and manufacturing personnel already facilitates ramp-up and preparation for future sales.
3. Several future opportunities on R&D, which must be pursued low key because of the need to focus on current completion of the clinical trials on Arikace and marketing:
a. Potential for Increased Efficacy and with Low Drug Toxicity (localized lung distribution only)
b. High-Efficiency Drug Encapsulation (aqueous in center, lipid in capsule of liposome)
c. Endogenous Lipid Excipients (liposome acts as a carrier)
d. For Other APIs
We believe that our liposomal technology can be used for the successful delivery of other low molecular weight products as well as high molecular weight compounds such as peptides, proteins and genes. Our unique lipid-based delivery systems are not dependent on the inhalation device and can be designed to be administered either as a nebulized aerosol spray or as a dry powder. (this is the big future - carrying biodegradable substances into the lung for direct action on the immune system, smooth muscle of conducting airways, etc.)
As WL said, these potential uses offer "partnering opportunities."
4. As for Iplex and ALS, the comment on PCUT is not weighty because PCUT is non-profit. So WL's statement about that relationship in the future strongly suggests that the best case scenario is to outlicense to Shire after Premacure.