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Calliditas Therapeutics AB Stockholm, June 2019 Rene - PowerPoint PPT Presentation

Calliditas Therapeutics AB Stockholm, June 2019 Rene Aguiar-Lucander, CEO Disclaimer Important information This presentation may contain certain forward-looking statements and opinions. Forward-looking statements are statements that do not

  1. Calliditas Therapeutics AB Stockholm, June 2019 Renée Aguiar-Lucander, CEO

  2. Disclaimer Important information This presentation may contain certain forward-looking statements and opinions. Forward-looking statements are statements that do not relate to historical facts and events and such statements and opinions pertaining to the future that, by example, contain wording such as “believes”, “estimates”, “anticipates”, “expects”, “assumes”, “forecasts”, “intends”, “could”, “will”, “should”, “would”, “according to estimates”, “is of the opinion”, “may”, “plans”, “potential”, “predicts”, “projects”, “to the knowledge of” or similar expressions, which are intended to identify a statement as forward-looking. This applies, in particular, to statements and opinions in this presentation concerning the future financial returns, plans and expectations with respect to the business and management of Calliditas Therapeutics, future growth and profitability and general economic and regulatory environment and other matters affecting Calliditas Therapeutics. Forward-looking statements are based on current estimates and assumptions made according to the best of Calliditas Therapeutics’ knowledge. Such forward-looking statements are subject to risks, uncertainties, and other factors that could cause the actual results, including Calliditas Therapeutics’ cash flow, financial condition and results of operations, to differ materially from the results, or fail to meet expectations expressly or implicitly assumed or described in those statements or to turn out to be less favorable than the results expressly or implicitly assumed or described in those statements. Accordingly, prospective investors and other third parties should not place undue reliance on the forward-looking statements herein. Calliditas Therapeutics can give no assurance regarding the future accuracy of the opinions set forth herein or as to the actual occurrence of any predicted developments. In light of the risks, uncertainties and assumptions associated with forward-looking statements, it is possible that the future events mentioned in this presentation may not occur. Moreover, the forward-looking estimates and forecasts derived from third-party studies referred to in the presentation may prove to be inaccurate. Actual results, performance or events may differ materially from those in such statements due to, without limitation, changes in general economic conditions, in particular economic conditions in the markets on which Calliditas Therapeutics operates, changes affecting interest rate levels, changes affecting currency exchange rates, changes in competition levels and changes in laws and regulations. The information, opinions and forward-looking statements contained in this announcement speak only as at its date, and are subject to change without notice. 2 Calliditas Therapeutics March 2019

  3. A specialty pharmaceutical company based in Stockholm, Sweden Listed on Nasdaq in Sweden in June 2018 – Midcap segment (ticker CALTX) Market cap June 2019: 180 MUSD Cash position end Q1, 2019: 63 MUSD Top 5 Shareholders: Industrifonden, Investinor, B Julander, Gladiator, AFA Insurance Ongoing Phase 3 read out expected for IgAN in 2H 2020; Pipeline: ODD received for AIH and PBC 3 Calliditas Therapeutics March 2019

  4. Investment Summary Calliditas 1 Novel treatment of IgA nephropathy (IgAN) with potential disease modifying effect Clear path to market – FDA acceptance of proteinuria as surrogate marker 2 Mode of action targets the origin of the disease – active in the gut 3 Only successful placebo controlled, randomized Ph2b study in IgA nephropathy (150 4 patients) The ongoing clinical Phase 3 study NEFIGARD replicates Phase 2b 5 6 Additional potential for pipeline development, in-licensing targeting orphan disease 7 Significant unmet medical need with USD 1bn market opportunity, no approved drugs 4 Calliditas Therapeutics March 2019

  5. Our lead indication: IgA nephropathy – large unmet medical need PROFILE ESTIMATED PREVALENCE Genetic predisposition – required but not sufficient. Environmental, bacterial, dietary triggers. 130,000-150,000 MAIN MARKET Normally presents in the 20-30s – more prevalent in men than in women. 200,000 Up to 50% at risk of ESRD within 10-20 years . POTENTIAL MARKET ~2,100,000 OPPORTUNITIES ~190,000 5 Calliditas Therapeutics March 2019

  6. Disease origin and progression – predominant theory 1 2 3 4 Cluster complexes Peyer´s patches Sugar deficient IgA Immune response IgA nephropathy  Lymphoid tissue;  In patients there is  The sugar-deficient  As the clusters enter  Deposits of immune Peyer’s patches in an increase in a IgA molecules the circulation, they complexes result in the distal part of the subclass of trigger an immune form even larger inflammation, small intestines immunoglobulin response resulting in complexes that necrosis and produces IgA molecules (“IgA”) formation of eventually lodge in destruction of the antibodies which lack a specific antibody clusters the kidneys kidneys’ filtration sugar modification apparatus Source: Suzuki et al, J Am Soc Nephrol 2011;22(10):1795-803; Novak et al, Curr 6 March 2019 Calliditas Therapeutics Opin Nephrol Hypertens 2013; 22(3):287-94; Novak et al, Kidney Dis (Basel). 2015; 1(1):8-18.

  7. Nefecon offers local treatment at origin of disease Drug product based on known active ingredient Unique targeted release profile  Active substance is budesonide  Targeted local delivery of potent immunosuppressive agent to Peyer’s patches in the ileum  90% first pass liver metabolism  minimize systemic side effects  Unique two-step release profile  Designed to provide a locally restricted and highly ─ PH-governed delayed disintegration of the capsule concentrated release of budesonide to the Peyer’s ─ Pulse like exposure throughout the Ileum patches for maximum effect Budesonide concentration 7 March 2019 Company Presentation

  8. Successful Phase 2b trial Only Phase 2b Study in IgAN to reach Primary Endpoint Conclusion – Efficacious and safe drug profile Efficacy Large study population – Oral dose taken daily over a nine-  Phase 2b trial of 150 patients demonstrated month period 150 patients clinically relevant reduction in proteinuria Randomized, double-blinded, European study in 62 sites in 10 and GFR stabilization in the treatment arms placebo controlled countries  UPCR reduction compared to placebo - 9 months treatment (p=0.0066)  eGFR stabilization compared to placebo – 9 Primary endpoint: Secondary endpoint: Reduction in proteinuria Stabilization of eGFR months treatment (p=0.0026) P (16mg)<0.01 P (16mg)=0.0026 Safety and tolerability P (8mg)<0.03 P (8mg) = 0.0064 2.7%  Medication-related adverse effects were 0.6% % ∆ eGFR % ∆ UPCR transient and mainly mild (77%) to -0.9% -9.8% -21.5% moderate (22%) -27.3%  No metabolic adverse events (hypertension, diabetes, weight gain) Placebo Nefecon Nefecon  No severe infections Placebo Nefecon Nefecon (16 mg) (8 mg) (16 mg) (8 mg)  Benign safety profile of 16 mg Nefecon 8 Calliditas Therapeutics March 2019

  9. Clinical Phase 3 study NEFIGARD to confirm Phase 2b results Key highlights → Phase 3 study design replicates successful Nefecon Phase 2b design Nefecon Phase 2b design Ph2b → 200 versus previous 150 patients (Ph2b) Run-in → 16mg Nefecon once daily oral dose → Surrogate marker; Proteinuria for market approval Nefecon Phase 3 design – NEFIGARD 9 months on treatment 9 Calliditas Therapeutics March 2019

  10. Endpoint: Proteinuria reduction in IgAN patients → Around 150 → Read out on first → Top line read- → Endpoint: → … with eGFR in clinical sites in 200 patients out estimated Proteinuria post approval 19 countries basis for H2 2020 reduction for follow up → Up to 450 approval and US approval of patients in total market launch Phase 3 study… • Endpoint for Phase 3 study identical to endpoint for Phase 2b: Proteinuria reduction. • Measured in the first 200 patients after nine months of oral treatment – basis for the accelerated approval in the US / conditional approval in Europe • Potential for full approval if proteinuria reduction is substantial, or on basis of planned interim analysis in post approval observational study. Data on kidney function expected around 18 months from top line read out • Convenient, oral medication for broad population with disease modifying potential – avoidance of dialysis • FPI in November 2018 • Company sponsored statistical framework, Inker et al 2016, basis for FDA surrogate marker acceptance 10 Calliditas Therapeutics March 2019

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