Manufacturing pathogen inactivated platelet lysate to treat corneal inflammation

DESCRIPTION provided by applicant Ocular graft versus host disease O GVHD is a particularly severe and debilitating complication of allogeneic hematopoietic stem cell transplantation HSCT O GVHD develops in of patients with chronic GVHD Currently Restasis cyclosporine A is the only FDA approved prescription topical medication to treat O GVHD In Restasis had sales of just under $ billion for the treatment dry eye however the therapeutic efficacy of Restasis is only and its use is associated with a rate of adverse events This lack of robust clinical effectiveness demonstrates that alternative therapies are needed Many HSCT patients who fail Restasis therapy have found relief from using autologous serum formulated into topical eye drops autologous serum tears AST Unfortunately the lack of standardization of AST preparations which are often prepared in doctorandapos s offices or pharmacies and challenges with its storage and administration have limited widespread adoption of this product In order to address the drawbacks of AST we have developed a proprietary method to manufacture a promising alternative a standardized platelet lysate preparation using pooled platelet collections phPL from qualified blood donors Elate OcularTM Ocular phPL We believe Elate OcularTM will be superior to Restasis or AST for treatment of patients with O GVHD and other forms of dry eye since Elate OcularTM is enriched compared to serum for a number of growth factors that promote corneal healing has robust anti inflammatory actions that can reduce ocular inflammation in patients with GVHD and possesses bacteriostatic properties This STTR Phase I application is submitted by Cambium Medical Technologies LLC a start up company founded by faculty members from Emory University School of Medicine who developed Ocular phPL Emory will be the sub contracting research organization for this application Emory University has filed a patent application to protect our proprietary manufacturing process for phPL and Cambium has exclusive rights to this patent and technology The investigators have previously received two INDs from FDA CBER for other applications of phPL IND NCT and IND NCT and we are now seeking to submit a treatment IND for Elate OcularTM and conduct a Phase I II clinical study in HSCT patients with O GVHD In order to support these clinical studies we propose in this application to first further improve and validate the safety and efficacy profiles f Elate OcularTM by i demonstrating feasibility of applying a pathogen inactivation PI process during production of Elate OcularTM ii performing in vitro work on Elate OcularTM PI treatment to compare product compositions and mechanisms of action and iii performing animal toxicology efficacy studies with Elate OcularTM PI treatment in animals with a pre existing inflammatory condition or deficient tear production Successful completion of these aims will allow us to complete our FDA IND application and initiate the Phase I II study for which we will seek Phase II STTR support in order to demonstrate that Elate OcularTM is safe and well tolerated in patients suffering O GVHD morbidity a required step toward future commercialization PUBLIC HEALTH RELEVANCE Many patients with blood cancers are increasingly being treated by allogeneic hematopoietic stem cell transplantation HSCT This type of HSCT is often curative but is not without side effects including debilitating eye problems We propose to perform the FDA required studies necessary to commercialize a new eye drop designed to treat the ocular side effects of allogeneic HSCT