MC2 Biotek

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Introducing PADciclo™ - because tolerability matters

Ophthalmology Innovation Summit, October 16th 2014

PAD™ Technology -‐ releasing the full poten1al of API’s

2014



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Click to edit Master title style MC2 Biotek Group at a glance §  Privately held Danish company

§  Major investor is family behind Danfoss Group (USD 6 bn) §  Long term commitment – flexible collabora1on models

§  PAD™ Technology – releasing full poten;al of API’s §  Key to a successful drug is a label recognized by payers and clinicians as clinically superior

§  Focus: Clinically superior PAD™ versions of high-‐value marketed topical drugs §  PADciclo™ Dry eye (phase II star1ng up) §  PADcombo™ Psoriasis (phase II ongoing) §  Atopic derma11s (phase I, Q1 2015) §  Other PAD™ topical projects in pre-‐clinical development

§  Operates through a network based business model §  Company loca1ons in Copenhagen and London §  Project teams of highly reputed experts in the US and EU §  Strategic collabora1ons with academia and hospitals



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Click to edit Master title style PADciclo™- need for 2nd generation CsA eye drop Ra;onale Challenge Solu;on by PADciclo™

Restasis® is twice daily, it s1ngs and has sub-‐op1mal label – yet a blockbuster

Need for improved CsA product in the US

Need for approved CsA products in EU

Reduce s1nging

(CsA will always s1ng in some pa1ents)

§ Reduce surfactant level ~30 fold § Reduce concentra1on of CsA

Once daily dosing § CsA penetra1on ~4 fold be]er than Restasis®

Get best in class claim for “treatment” § Novel clinical strategy



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Click to edit Master title style PAD™ droplets are robust with low level of surfactants



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>1,000 patients treated with PADciclo™ 0.06% in the UK

Collabora;on with Moorfields Pharmaceu;cals (Moorfields Eye Hospital) §  Available in the UK since April 2012 as a 0.06% preserva1ve free unlicensed Special §  Repeated sales to >300 hospitals, clinics, wholesalers and pharmacies §  More than 1,000 pa1ents have been treated



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MEH analysis of 129 patient files is very encouraging

Safety: §  PADciclo™ 0.06% is safe §  No decrease in visual acuity, no effect on intraocular pressure and no vital signs

Efficacy: §  Qualita1ve data reported by pa1ents and ophthalmologists show improvement of clinical

status of dry eye pa1ents (Be]er, Stable, Worse)

Ophthalmologists Pa;ents BePer 66% 69% Stable 34% 27% Worse 0% 4%

Data from dry eye pa1ent files with sufficient data



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PADciclo™ is preferred by patients at MEH

PADciclo 0.6% CsA survey (n=23) conducted by Moorfields Eye Hospital (Feb-‐April 2013) Author: Kris1n Chapman (Formulary & An1bio1c Pharmacist)



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Click to edit Master title style PADciclo™ is well tolerated and increases ocular delivery

Ocular PK data support once daily ins;lla;on at strengths of 0.03% and 0.06% w/w CsA §  PK studies in rabbits show superior ocular

exposure of PADciclo™ compared to Restasis®

§  PADciclo™ once daily is sufficient for saturated delivery to cornea and conjunc1va

0

1

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PADciclo 0.06%, 1x

PADciclo 0.06%, 2x

PADciclo 0.1%, 1x

PADciclo 0.1%, 2x

0.05% Restasis

Fold Exposure over Restasis (AU

C 0-‐24h) Cornea

Conjunc1va

Ocular Exposure rela;ve to Restasis®

PADciclo is safe and very well tolerated up to 6 months §  Rabbit safety and tolerability studies conducted at 0.1% strength with 2-‐4 1mes daily dosing §  No treatment or administra1on-‐related effects §  Systemic exposure of ciclosporin is very low



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Large PADciclo™ phase II trial is starting up PADciclo™ has the required poten;al

§  Excellent pre-‐clinical data

§  Large scale manufacture in unit dose secured

§  Once daily dosing and high tolerability are key for pa1ent compliance

§  Experience from >1,000 pa1ents in the UK and 129 pa1ent files

§  Outcome of Scien1fic Advice mee1ngs support our clinical strategy

Fast to market and best in class



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Opportuni;es using PAD™ vehicle: §  Reformulate high value marketed drugs + LCM §  Release the full poten1al of API’s from first formula1on

PAD™ Technology - release the full potential of API’s

§  The right vehicle can provide the twist that makes a superior product

§  PAD™ Technology does not impose addi1onal risk -‐ only pharmacopeial excipients

§  Key differen1a1ng features of the PAD™ Technology:

Penetra1on Solubility Stability

Compliance Tolerability Patented



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Click to edit Master title style We welcome collaboration

Thank you for your attention

MC2 Biotek

Healthcare