Preclinical Assessment of Investigational Cellular and Gene Therapy Projects

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1 Preclinical Assessment of Investigational Cellular and Gene Therapy Projects Doug Padley, MT (ASCP) Mayo Clinic, Rochester, MN ISCT Regional Meeting September MFMER slide-1

2 Objectives Review Draft FDA Guidance: Preclinical Assessment of Investigational Cellular and Gene Therapy Products Relate experiences with pre-clinical assessments of cells and tissue engineered products Review benefits and pitfalls of mouse models to elucidate mechanisms of action MFMER slide-2

3 Guidance for Industry Preclinical Assessment of Investigational Cellular and Gene Therapy Products (Draft) US FDA, November, MFMER slide-3

4 Disclaimers This draft guidance, when finalized, will represent the Food and Drug Administration s (FDA s) current thinking on this topic. It does not create or confer any rights for or on any person and does not operate to bind FDA or the public. You can use an alternative approach if the approach satisfies the requirements of the applicable statutes and regulations 2013 MFMER slide-4

5 Refers to or summarizes guidance 2013 MFMER slide-5

6 Preclinical Assessment of Investigational CGT Products Introduction Background Preclinical Study Considerations Recommendations for CT Products Recommendations for GT Products Recommendations for Therapeutic Vaccines 2013 MFMER slide-6

7 Introduction To provide recommendations on the type of pre-clinical information needed to support clinical trials of cell and gene therapy (CGT) products Cell therapies Gene therapies Therapeutic vaccines Xenotransplantation Certain device-biologic combos Doesn t apply to: 361 HCTP/s Devices, CDER and OBRR reviewed products, preventive vaccines 2013 MFMER slide-7

8 Introduction Traditional approaches for typical drugs are often not appropriate. OCTGT uses flexible, science driven review to address safety. Considers biology of product and clinical indication. Incorporates the basic principles of toxicology studies MFMER slide-8

9 Preclinical Program Should: Establish biological plausibility Identify active dose levels, escalation and overall regimen Provide support for chosen route of administration Support trial eligibility criteria Identify physiologic parameters to guide monitoring Identify public health risks 2013 MFMER slide-9

10 Recommendations: Product The clinical product should be used for preclinical studies Not always possible Rationale and differences should be explained in IND submission Lot release testing in preclinical studies should be as close as possible to clinical products 2013 MFMER slide-10

11 Cell-Coated Platinum Coils for the Treatment of Intracranial Aneurysms Platinum coils are a standard treatment Known aneurysm recurrence rate Coating coils with cells before delivery could facilitate repair of vessel wall Preliminary animal study Human cell coated coil for experimental aneurysm in rabbits 2013 MFMER slide-11

12 Cell-Coated Platinum Coils for the Treatment of Intracranial Aneurysms Human cells appeared to properly localize Extravascular inflammation Rabbits were not immunosuppressed IND strategy: Next studies - autologous rabbit cells in the same model Same culture procedures as for human manufacturing 2013 MFMER slide-12

13 Recommendations: Animal Species Selection Biological response should be similar to humans Consider anatomy, viral replication, immune tolerance, compatibility with patient delivery system More than one species might be required In-Vivo tests to help identify ideal animal species Rationale and summary part of IND submission 2013 MFMER slide-13

14 Selection of Animal Models of disease/injury Same models from basic research may benefit preclinical studies Might be preferable to healthy animals to assess mechanism and safety Possible identification of biomarkers to monitor clinical trials 2013 MFMER slide-14

15 Limitations of Animal Models of Disease/injury Inherent variability of the model. Limited historical/baseline data for the model. Technical limitations with the physiological and anatomical constraints of the model. Animal care issues Limited fidelity in modeling human pathophysiology of the disease/injury of interest MFMER slide-15

16 Recommendations: Animal Model of Disease In the IND submission describe: Similarities and differences between animal model and human disease Effect of animal disease on the pharm/tox of the product Effect of animal disease on the efficacy of the product Tiered approach Pilot studies with CGT product Help select animal model for later studies 2013 MFMER slide-16

17 Mesenchymal Stem Cells for ALS Commonly studied animal model is SOD-1 knockout mouse Only the hereditary form of ALS in humans is associated with SOD-mutation 5-10% of ALS cases Animal model may not be applicable for 90% of clinical patients 2013 MFMER slide-17

18 MSC for ALS Animal models not predictive for human trials IND strategy Safety of cells Safety of route of administration Reference published human studies Risk/benefit equation 2013 MFMER slide-18

19 Risk Benefit Assessment Benefit Good pre-clinical data MOA and dose defined Biomarkers of efficacy Prior clinical experience Poor prognosis without Rx Limited other Rx Risk Less clinical experience Known toxicity Undefined MOA Pediatric population 2013 MFMER slide-19

20 Proof of Concept Studies Benefit side of the Risk/Benefit relationship Establish the rationale and feasibility of the proposed clinical trial Help define the dose range and timing Optimize the Route of Administration Help characterize the mechanism of action Can be combination of in vitro and in vivo studies 2013 MFMER slide-20

21 Toxicology Studies Adequate numbers and randomization Appropriate controls Bracketed dosing Dosing schedule modeled on clinical protocol Clinical route of administration Including delivery device where applicable Assessments for standard acute and chronic effects Assessments for product specific expected toxicities 2013 MFMER slide-21

22 Good Laboratory Practices GLP required for toxicity studies per 21 CFR part 58 Recognition that isn t always possible Rationale needs to be included in IND submission 2013 MFMER slide-22

23 Data in Support of Clinical Trials- Mayo Solution Not GLP or GMP, but a defined and established system for data supporting IND and IDE submissions. A voluntary opt-in system where membership requires participation in the entire process. DSCT is overseen by a group individuals interested in standardizing and providing the best data in support of clinical trials MFMER slide-23

24 Data in Support of Clinical Trials The Human Cellular Therapy Laboratory performs few animal studies independently Partner with numerous research laboratories DSCT provides templates and standardization for reports from diverse investigators Consistent report formats Can be used for multiple INDs 2013 MFMER slide-24

25 Reduction, Refinement and Replacement Refers to Animal Welfare Act of 1975 Reduction by use of a single species and by non terminal studies when justified Refinements such as incorporation of pain management and non-terminal imaging Replacement by use of in-vitro studies when available 2013 MFMER slide-25

26 MSC for Multisystem Atrophy (MSA) Degenerative neurologic disorder of autonomic system Median survival ~9 yrs IND strategy Cross reference ALS IND Same cells and ROA Refer to published safety study of MSC for MSA Different ROA than our proposed IND No additional pre-clinical studies required 2013 MFMER slide-26

27 Product Development for Later Phase Clinical Trials As the CGT product changes through early trials, consider additional preclinical studies to address any outstanding issues Manufacturing Dose Patient population Consult with OCTGT to ensure seamless product development MFMER slide-27

28 Preclinical Study Reports Separate report required for each in vitro and in vivo safety study Include Protocol + all amendments Detailed description of study design Animals, doses, route of delivery, controls Complete data set for all parameters measured Tabular data for all animals Analysis and interpretation of results 2013 MFMER slide-28

29 Cellular Therapy Products Two broad categories Stem Cell Derived Variable capacity for self renewal Able to differentiate into variety of cell types Fate is determined mainly by milieu post administration Mature/Functionally Differentiated No self renewing or differentiation 2013 MFMER slide-29

30 Animal Species/models Considerations Ability to access the anatomic site for administration Ability to deliver specific dose to target site Immunodeficient animals for long term assessment 2013 MFMER slide-30

31 Immunologic Tolerance How do you test a product if the animal rejects it? Immunosuppression Gene modified immunodeficient animals Humanized animals Immune privileged site Combination Analogous animal product 2013 MFMER slide-31

32 Using an Analogous Animal Product Need to demonstrate comparability to product Sample harvest Cell identification and culture Growth kinetics Phenotype and function Final product formulation Storage and stability 2013 MFMER slide-32

33 Study Design: Overall Cell phenotype Cell source Ex vivo manipulation Minimal effective dose Limiting dose Cell fate Possibility of host immune response Local and systemic toxicities 2013 MFMER slide-33

34 Study Design: Safety Administration site reactions Inflammatory response Host immune response Migration away from administration site Ectopic tissue formation Abnormal proliferation Tumorigenicity 2013 MFMER slide-34

35 % Positive % Recovery % Viable Cell Loss 100 Recovery 100 Viability Cell Loss e6 cells 25.0e6 cells Cell dose 50.0e6 cells 75 Pre 10.0e6 cells 25.0e6 cells 50.0e6 cells Post-culture e6 cells 25.0e6 cells Cell dose 50.0e6 cells Phenotype MSC delivered through injection catheter at clinical doses and proposed rate of administration 0 CD90 CD73 CD105 CD44 HLA-ABC CD14 CD45 HLA-DR 2013 MFMER slide-35

36 Study Design: Product Fate Survival/engraftment affected by: Biocompatibility Route of Administration Genetic relationship of the host Immune status Timing Distribution Typical pharmacokinetics don t often apply Imaging, immunohistochemistry, PCR, karyotype Differentiation and integration Tumorigenicity 2013 MFMER slide-36

37 Study Design: Product Fate Differentiation and Integration Tumorigenicity 2013 MFMER slide-37

38 CT Products with Scaffolds Cells Scaffolds Biocompatibility Cell seeding Study groups Varying cell density and loading Biologic responsiveness Dose response and durability Safety 2013 MFMER slide-38

39 Stem Cell Fistua Plug in Perianal Crohn s Disease Fistula plug is standard surgical treatment Clinical trial using MSC coated fistula plug IND strategy Demonstrate MSC coating of plug Refer to published reports of safety of MSC for Crohn s Refer to safety record of plug, clinically available device Risk mitigation strategy MSCs stay on plug despite trypsin Plug can be surgically removed in necessary 2013 MFMER slide-39

40 Stem Cell Fistua Plug in Perianal Crohn s Disease Submitted preclinical in vitro studies No animal studies required for IND to proceed 2013 MFMER slide-40

41 Conclusions Draft Guidance is a good road map to follow for successful IND submission FDA encourages communication Risk based approach Develop cooperative relationship with groups performing animal pre-clinical studies They may need some guidance if they re new to the IND process 2013 MFMER slide-41

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