Panoptes Franz Obermayr CEO Development Pipeline •  Panoptes is developing PP-001 for local treatment in ophthalmology Inﬂammatory diseases Research Intravitreal and oral Pre-clinical Phase 1 Phase 2 Posterior Uveitis topical Anterior/ Uveitis topical Dry Eye (Sjögren) topical Viral Conjunctivitis 2 Unique mode of action by PP-001, a best in class inhibitor of DHODH •  Best in class DHODH inhibitor more than 100 fold more potent than Arava and Aubagio (RA and MS) •  Inhibits proliferative T cells •  Is a potent anti adenoviral agent •  Is a selective nanomolar inhibitor of IL-17, IFN-g and VEGF 3 Proof of Concept in Experimental Autoimmune Uveitis (EAU) – once daily p.o. 2500 EAU/R14 in Lewis rats IL-‐17 9000 2000 8000 7000 1500 IL-‐17 pg/ml IFN-‐gamma pg/ml Clinical uvei4s score INF-‐gamma 1000 500 6000 5000 4000 3000 2000 1000 0 3µM 10µM 30µM PBS 0 PP-‐001 PBS vitreous re-na days post immuniza4on 3µM 10µM 30µM PP-‐001 vitreous re-na sclera sclera Placebo PP-‐001 EAU unpublished results Prof. Wildner 4 Therapeutic use of PP-001 in uveitis - single intravitreal injection Prevention of recurrence in relapsing-remitting EAU/R14 placebo Clinical uvei4s score placebo PP-001 PP-001 Right and leT eye Days post immuniza-on treatment treatment •  PP-001 reduced the number of relapses by >50% •  Parallels real clinical need in patients to prevent relapses 5 Developed an Uveitis animal model for choroidal neovascularization •  Choroidal neovascularization (CNV) is one of the most severe causes of visual impairment in patients with posterior uveitis and is one of major sight threatening mechanism. •  Rats were immunized with specific antigens to induce experimental autoimmune uveitis •  After initial uveitis flare rats develop to a high frequency CNV as a sequel GL ganglion cell layer INL inner nuclear layer ONL outer nuclear layer CH choroid 6 PP-001 inhibits choroid neovascularization Inhibition of CNV in rat eyes EAU Rat model was treated with PP-001 at beginning (day9) or at the peak (day 15) of clinical symptoms of uveitis A: Vehicle control 20x B: PP-001 day 9 20x 7 PP-001 inhibits CNV via VEGF PP-001 inhibits VEGF VEGF is the hallmark effector molecule for neovascularization and angiogenesis and the major target for therapy in wet AMD Peripheral lymphnodes of EAU rats induced by the PDSAg peptide were harvested at day 14 and were assayed for activity on cytokines. 8 PP-001 mode of action in CNV PP-‐001 PP-‐001 Schema-c model for promo-on of CNV taken from Hasegawa et al, Jimmunol; Jan 13, 2013. 9 Science Summary PP-001 •  •  •  POC in EAU rat model for uveitis •  is superior to cyclosporineA in Uveitis •  inhibits choroidal neovascularization via VEGF/IL-17 •  administered locally (intravitreal injections) as maintenance therapy for uveitis •  Excellent tolerability POC in scopolamine mouse dry eye model •  First time proof that topical formulation of PP-001 is efficacious •  Significant reduction of corneal defects, the crucial endpoint in dry eye disease •  Excellent tolerability POC in adenoviral conjunctivitis rabbit model •  Clear dose response with topical formulation comparable with cidofovir •  Excellent local tolerability (draize scale analysis) 10 Summary Panoptes •  Recently closed financing round •  Phase 1b study in 2015 with intravitreal PP-001 •  We are looking for experienced development and commercialization partners in the field 11