We only get one shot! LOL unless you have to provide all of the below because you know a penny stock fraud lawyer pretending to run a Biotech will tell you anything that keeps his paychecks coming.
· Provide additional sequencing data and genetic stability studies;
· Conduct a stability study on the final formulated drug product as well as the cells from the Master Cell Bank;
· Evaluate the compatibility of the delivery devices (i.e., the prefilled syringe and microcatheter) with the drug product;
· Provide additional detailed description of the manufacturing process;
· Provide additional product release specifications for the encapsulated cells;
· Demonstrate comparability between the 1st and 2nd generation products and ensure adequate and consistent product performance and safety between the two generations;
· Conduct a biocompatibility assessment using the final finished capsules after the entire drug product manufacturing process (but without cells);
· Address insufficiencies in Chemistry, Manufacturing and Controls information in the cross-referenced Drug Master File;
· Conduct an additional nonclinical study to assess the safety, activity and distribution of the drug product;
· Revise the Investigators Brochure to include any additional preclinical studies conducted in response to the clinical hold and remove any statements not supported by the data; and
· Provide data from a new pig study.
The FDA also requested that we address several issues not related to the clinical hold in an amendment to the IND, including:
· Provide a Certificate of Analysis for pc3/2B1 plasmid that includes tests for assessing purity, safety, and potency;
· Perform qualification studies for the drug substance filling step to ensure that the product remains sterile and stable during the filling process;
· Submit an updated batch analysis for the drug product for the specific lot that will be used for manufacturing all future drug product;
· Provide additional details for the methodology for the Resorufin (CYP2B1) potency and the PrestoBlue cell metabolic assays;
· Provide a few examples of common microcatheters that fit the specifications in the Angiography Procedure Manual;
· Clarify the language in the Pharmacy Manual regarding proper use of the syringe fill with the drug product; and
· Provide a discussion with data for the potential for cellular and humoral immune reactivity against the heterologous rat CYP2B1 protein and potential for induction of autoimmune-mediated toxicities in the Company’s study population.
There has been no further communication with the FDA regarding the clinical hold
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