Future clinical trials of targeted therapies for pemphigus

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1 Future clinical trials of targeted therapies for pemphigus Aimee S. Payne, MD, PhD University of Pennsylvania September 17, 2017 Conflicts of interest: Founder of Tycho Therapeu9cs, focused on targeted therapies for pemphigus; inventor on a patent licensed to Novar9s for immunotherapy of autoimmune diseases including P/P

2 Precision medicine for pemphigus therapy Cancer therapy X Pemphigus therapy Prednisone Mycophenolate Azathioprine Methotrexate X

3 Strategies for targeted therapy in pemphigus Pep;de immunotherapy for tolerance induc;on in pemphigus vulgaris Targeted B cell deple;on in pemphigus vulgaris (CAAR- T)

4 Why all the a7en8on on pemphigus vulgaris? Pemphigus is considered one of the best, if not the best, defined autoimmune diseases in humans The desmoglein ELISA is % sensi;ve and specific for the diagnosis of pemphigus, which means that almost everyone with a pemphigus has a posi;ve test, and almost no one with a posi;ve test doesn t have pemphigus Pemphigus vulgaris represents 85% of all pemphigus cases, whereas pemphigus foliaceus is only 15% Pemphigoid is a bit more complicated because the an;body alone isn t necessarily enough to cause disease, you also need inflamma;on or other signals to develop blisters

5 Tolerance induc8on using pep8de- coupled cells htp:// Collect cells from a mouse with experimental mul;ple sclerosis Coat the cells with small parts of the protein targeted in disease Inject the coated cells back into the mouse disease resolves

6 Tolerance induc8on using pep8de coupled cells htp:// htps:// Collect cells from a human with mul;ple sclerosis Coat the cells with small parts of the protein targeted in disease Inject the coated cells back into the pa;ent Safety No adverse events related to therapy (1 pa;ent with metallic taste) 4 flares of disease in 10 pa;ents: Two within 1 month of treatment in pa;ents with severe disease Two 4 months aaer treatment Efficacy 4 of 10 pa;ents showed reduced protein- specific T cell responses

7 Tolerance induc8on using pep8de coupled cells: how does it work? htps:// pep;de- cells cause T cell anergy pep;de- cells cause dendri;c cells to tolerize Stand down! Induc;on of regulatory T cells T cells send signal: Make an;bodies! B cells respond

8 Tolerance induc8on using pep8de coupled cells: how does it work? htps:// pep;de- cells cause T cell anergy pep;de- cells cause dendri;c cells to tolerize Stand down! Induc;on of regulatory T cells T cells send signal: Make an;bodies! B cells respond

9 Cures of B cell leukemia with CAR- T cells Phase I/IIa trial 30 pa;ents: 90% complete remission 73% relapse- free Major toxici;es observed: Permanent B cell deple;on cytokine release syndrome Maude et al, NEJM 2014, 371:1507 ELIANA trial (NCT ): 82% CR in refractory leukemia Grupp et al, ASH #221, 2016 Ac?vate T cells Engineer them to express CARs Make millions more Nat. Rev. Clin. Oncol. 2013

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11 Targeted B cell deple8on with CAAR- T cells htp://science.sciencemag.org/content/early/2016/06/29/science.aaf6756 B cells: all have CD19 on their surface, only a few have an an;- desmoglein an;body

12 Targeted B cell deple8on with CAAR- T cells htp://science.sciencemag.org/content/early/2016/06/29/science.aaf6756 An;- CD19 B cells: all have CD19 on their surface, only a few have an an;- desmoglein an;body

13 Targeted B cell deple8on with CAAR- T cells htp://science.sciencemag.org/content/early/2016/06/29/science.aaf6756 An;- CD19 Desmoglein B cells: all have CD19 on their surface, only a few have an an;- desmoglein an;body

14 CAAR T cells cure pemphigus in mouse models htp://science.sciencemag.org/content/early/2016/06/29/science.aaf6756 Dsg3 CAAR control Day 5 Day 12

15 Poten8al advantages of CAAR T cells htp://science.sciencemag.org/content/early/2016/06/29/science.aaf6756 CAAR T cells: Specifically kill an;- Dsg3 B cells while sparing other B cells, without detectable off- target toxicity Offer the poten;al for long- term cure of disease May not be suscep;ble to side effects seen with CAR- T therapy of cancer given that pemphigus B cells are rare Could theore;cally be applied to any an;body- mediated disease Next steps: meet the FDA requirements to launch a clinical trial of CAAR- Ts in pemphigus

16 How soon to clinical trials? (1-3 years) Pep;de immunotherapy for tolerance induc;on in pemphigus vulgaris Targeted B cell deple;on in pemphigus vulgaris (CAAR- T)

17 An9gen- specific therapies for pemphigus vulgaris Innova;ve therapeu;c strategies are on the horizon for pemphigus If proven successful in pemphigus vulgaris, therapies for other forms of P/P, as well as other autoimmune diseases, may follow There will be challenges in designing trials for rare diseases when the cost of therapy is currently high Clinical trials are ul;mately the only way to know whether therapy is safe and effec;ve in humans

18 Acknowledgments Penn Dermatology Christoph Ellebrecht Jinmin Lee Eun- Jung Choi Michael Cho Xuming Mao John Seykora Arben Nace George Cotsarelis Mike Milone, MD/PhD Christoph Ellebrecht, MD Jinmin Lee, PhD Penn Pathology Michael Milone Vijay Bhoj Selene Nunez- Cruz Masayuki Amagai (Keio University) Jun amagami Antonio Lanzavecchia (ETH Zurich) Giovanni Di Zenzo

19 Targe8ng inflamma8on in bullous pemphigoid Autoan;bodies bind skin Ac;va;on of complement Release of chemicals to bring in immune cells (IL- 17, X eotaxin, X others) ixekizumab ber;limumab Inflammatory cells release damaging enzymes or chemicals Skin blistering htp://link.springer.com/ar;cle/ %2fs y

20 Benefits and risks in ongoing pemphigoid trials RITUXIMAB Ixekizumab Already FDA- approved for psoriasis No clinical data on efficacy in BP, but epidemiologic studies have shown that psoriasis is more common in BP pa;ents (5x more common in those and 43x more common in those under 60 years of age), sugges;ng poten;al common causes Risks = injec;on site reac;ons, upper respiratory or fungal infec;ons Ber;limumab Preliminary data in a few BP pa;ents shows an 84% improvement in disease ac;vity Side effect profile unclear, but eosinophil blockade is generally well tolerated, mainly a risk of upper respiratory infec;ons

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