Calliditas Therapeutics AB · Market opportunity Annual market opportunity over $2bn. Calliditas...

Calliditas Therapeutics AB

Pareto Securities Health Care Conference 2019September 5, 2019

Renée Aguiar-Lucander, CEO

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.

2Calliditas Therapeutics September 2019

Out licensing deal for China ($121m) closed in Q2, validating market potential in IgAN for Nefecon

Nasdaq Stockholm listed (ticker CALTX) specialty pharmaceutical company (market cap SEK 2.2bn)

Cash position end Q2 2019: $56m (SEK 534m)

Key shareholders include: Industrifonden, B.V.F, B Julander, Gladiator, AFA Insurance

NefIgArd pivotal Phase 3 study in IgAN on plan, expected readout in H2 2020


$121m (SEK 1.1bn) out licensing deal for Greater China validates future commercial potential

Investment Summary Calliditas

Novel treatment of IgA nephropathy (IgAN) with potential disease modifying effect targeting the origin of the disease

Strong cash position fully finances ongoing clinical development portfolio

Ongoing pivotal clinical Phase 3 study NefIgArd replicates successful Phase 2b

Additional potential for pipeline development: orphan liver indications and targeted in-licensing

Significant unmet medical need with no approved drugs. US annual market opportunity of around $2bn. Combined opportunity in China and Europe of at least equal size.

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4September 2019Calliditas Therapeutics

Our lead indication: IgA nephropathy – large unmet medical need

PROFILE ESTIMATED PREVALENCE

Genetic predisposition – required but not sufficientEnvironmental, bacterial, dietary triggers

Normally diagnosed in ages 20-30sMore prevalent in men than in womenUp to 50% at risk of ESRD within 10-20 years

130,000-150,000

200,000

600,000-800,000

MAI

N M

ARKE

TS5

March 2019

Calliditas Therapeutics September 2019

Wordwide Nefecon Market Potential3

ESRD causes considerable costs to society• Average annual cost per person in the US

$70,000-$200,0001

• Average cost per kidney transplant $414,8002 $4bn

ESTIMATED DIAGNOSED PATIENTS

Source: 1) Calliditas estimate based on third party research commissioned by the Company. 2) Milliman, Milliman research report:2017 U.S organ and tissue and transplant cost estimation and discussion. 3) 50% of patients diagnosed being candidates for treatment & recent third party (IQVIA) research commissioned by the Company

Disease origin and progression – predominant theory

September 2018Calliditas Therapeutics 6

IgA nephropathyCluster complexesImmune responseSugar deficient IgAPeyer’s patches1 2 3 4

In patients there is an increase in a subclass of immunoglobulin molecules (“IgA”) which lack a specific sugar modification

The sugar-deficient IgA molecules trigger an immune response resulting in formation of antibody clusters

As the clusters enter the circulation, they form even larger complexes that eventually lodge in the kidneys

Deposits of immune complexes result in inflammation, necrosis and destruction of the kidneys’ filtration apparatus

Lymphoid tissue; Peyer’s patches in the distal part of the small intestines produces IgA antibodies

Source: Suzuki et al, J Am Soc Nephrol 2011;22(10):1795-803; Novak et al, Curr Opin Nephrol Hypertens 2013; 22(3):287-94; Novak et al, Kidney Dis (Basel). 2015; 1(1):8-18.

Nefecon offers local treatment at origin of disease

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Designed to provide a locally restricted and highly concentrated release of budesonide to Peyer’s patches for maximum effect

16mg oral dose

Unique two-step release profile

PH-governed delayed disintegration of the capsule

Pulse-like exposure throughout the iIeum

Unique targeted release profile

September 2019Calliditas Therapeutics

Nefecon offers local treatment at origin of disease

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Oral, once daily administration

Budesonide – proven to be safe and effective

Minimize systemic side effects 90% first pass liver metabolism

Orphan protection in the US and the EU

Strong product protection for Nefecon

10 years market exclusivity in the EU

7 years market exclusivity in the US

Formulation patent 2029 expiry

Drug product based on known active ingredient

September 2019Company Presentation

Triple coated beads for sustained release

Polymer coatingSeal coatingActive coatingCore particle

Enteric coat for delayed release

Capsule

Successful Phase 2b trial

Primary endpoint: Reduction in proteinuria

2.7%

-27.3%-21.5%

Placebo Nefecon(16 mg)

Nefecon(8 mg)

% ∆

UP

CR

-9.8%

0.6%

-0.9%

Placebo Nefecon(16 mg)

Nefecon(8 mg)

% ∆

eG

FR

Secondary endpoint:Stabilization of eGFR

P (16mg)=0.0026P (8mg) = 0.0064

P (16mg)<0.01P (8mg)<0.03

Only placebo controlled, randomized Phase 2b Study in IgANto reach Primary Endpoint and show renal protection Conclusion – Efficacious and safe drug profile

Efficacy Pan European; 150 patients, 10 countries, 62 sites Highly statistically significant UPCR (proteinuria)

reduction compared to placebo - 9 months treatment (p=0.0066)

Highly statistically significant eGFR stabilization compared to placebo – 9 months treatment (p=0.0026)

Safety and tolerability Medication-related adverse effects were transient

and mainly mild (77%) to moderate (22%) No metabolic adverse events (hypertension,

diabetes, weight gain) No severe infections Benign safety profile of 16 mg Nefecon

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-4,7 ml/min/1.73m2

Calliditas Therapeutics September 2019Study results published in The Lancet, 2017

Ongoing clinical Phase 3 study NefIgArd to confirm Phase 2b results

9 months on treatment

Nefecon Phase 3 design – NefIgArd

→ Phase 3 study design replicates successful Ph2b

→ Fixed 16mg Nefecon once daily oral dose

→ Reduction of proteinuria: Accelerated approval/market access

→ Full approval on 2 year eGFR based endpoint – 360 patients

Key highlights


Topline readout: 200 patients

Nefecon – Poised to be first approved treatment of IgAN

→FPI Nov 2018 →140 clinical sites

recruiting in 19 countries

→Top line read-out on first 200 patients estimated H2 2020

→Filing with regulatory agencies for accelerated / conditional approval Q1 2021

→ Market launch Q1 2022

→ Readout for full approval mid 2022


• Company sponsored statistical framework, Inker et al 2016, basis for FDA surrogate marker acceptance

• Potential for full approval based on proteinuria, if reduction is substantial, however 505b2 pathway assumed

• Read out on loss of kidney function over two years (eGFR) for validation of surrogate marker H2 2022

2018 2019 2020 2021 2022

Additional open chronic dosing study with Nefecon

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Preliminary Outline:

Global study with around 100 patients Leveraging existing sites and networks Inclusion criteria different from ongoing study Focus: safety and stabilization of eGFR Provide additional support for disease modification

Planned start in Q1 2020 Duration 12-18 months depending on feedback from health authorities Starting dose 16mg, step down to maintenance dose

Enables additional data capture related to safety & maintenance treatment


Commercialization strategy for IgAN in the US

March 2019

Commercial strategy Target IgA nephropathy patients at risk of

progressing to ESRD (up to 50%)

Earlier stage treatment to prevent progression and preserve kidney function

Chronic / intermittent dosing

Targeting of therapeutically focused nephrologists - hub and spoke structure

Collaborate with patient organizations

Need for relatively small sales and marketing organization

Company managed US commercialization

Significant unmet medical need

Calliditas Therapeutics September 2019 13

Calliditas TherapeuticsMarket opportunity Annual market opportunity over $2bn

Indicative pricing from market

research $55,000-85,000 per treatment cycle1

Positioning Backbone treatment potential - first approved medication for IgAN

Severe side effects of existing off label treatments

Orphan drug

Specialist target market

Disease modifying potential –delay or avoid dialysis / transplantation

Source: 1 Third party (IQVIA) research commissioned by the Company

Out-licensing of Nefecon to Greater China


Partnering deal with Everest Medicines

Well funded, highly experienced biopharma company with a broad pipeline of late stage clinical programs for China

Total deal value of $121m, with $15m upfront payment

Everest Medicines responsible for the development and commercialization in the territory

Potential to be the first approved medication for IgAN in China, targeting a significant unmet medical need

Market opportunity

Greater China has a population of approx. 1.4bn people, which implies that the total IgAN population is very sizeable

Assuming that biopsies have been carried out fairly regularly for at least the last 10 years, it would indicate a diagnosed population today of around 600,000 – 800,000

As biopsies are still limited to large hospitals, the actual patient population should be significantly larger, likely at least 2x

Source: 1) Am J Nephrol 2009;30:268-273

Commercialization strategy for IgAN in EU and China

March 2019

Commercial strategy

One, or several commercial partners Orphan status in the EU Market landscape mapping work

initiated to assess potential

Partnership signed in June 2019 with Everest Medicines

Potential to join global NefIgArd trial Near term approval path for Chinese

market

Partner driven commercialization


Market opportunity

~$1bn

Indicative pricing To be provided post results from market study

Positioning Potential to be first approved treatment for IgAN – backbone treatment

Partner driven commercialization

~$1bn

Partner assessment

Partner assessment

Calliditas Pipeline


(As per September 1, 2019)

Candidate IndicationResearch/Preclinical Phase 1 Phase 2 Phase 3 Market Approval / phase 4

Completed

Nefecon IgA Nephropathy

- Chronic dosing

- Roll over study, intermittent treatment

- Transplant study

- Single course

Agreed /Ongoing

Planned

Primary Biliary Cholangitis

Autoimmune HepatitisPartner Asset1

1 In-licensed from Dr. Falk Pharma

Pipeline Indications - Autoimmune Hepatitis (AIH)

September 2019Corporate Presentation 17Source: 1) Feld et al, Hepatology 2005;42:53-62, Sahebjam and Vierling, Front Med. 2015 Jun;9(2):187-219. 2) Sahebjam and Vierling, Front Med 2015; 9(2):187-219. 3) Czaja, Expert Opin Drug Saf. 2008;7(3):319-33; Czaja, Liver Int 2009;29(6):816-23; Manns et al, Hepatology 2010;51(6):2193-213 (AASLD 2010 AIH Guideline).

The disease

Rare, orphan, chronic inflammation of the liver with unknown cause

Leads at variable rates to cirrhosis with complications like portal hypertension, liver failure and liver cancer

Medical need

Estimated prevalence

60,000 – 80,000

Annual US incidence 0.1 – 1.9

per 100,000

Currently no products approved in the US: SoC treatment includes immunosuppression with systemic steroids (prednisone) alone or in combination with azathioprine

Up to 80% of treated patients report steroid related side effects after 2 years and 15% discontinue due to drug related adverse events

Calliditas estimates the intolerance and relapse segments together comprise 35-40% of the total population, approx. 25,000 patients

Calliditas’ strategic in-licensing for AIH

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In-licensing agreement with Dr. Falk Pharma for US market

Maximizes opportunity to accelerate time to approval and US market access in AIH Creates optionality and flexibility for product portfolio in the US Orphan indication with market exclusivity and reimbursement No approved treatments €1.5m upfront payment, total deal value of €40m

Preparation for FDA interaction initiated FDA interaction planned for Q1 2020


Pipeline Indications - Primary Biliary Cholangitis (PBC)

Calliditas Therapeutics September 2019 19Source: 1) Lindor et al, Hepatology. 2009 Jul;50(1):291-308,EASL PBC Clinical Practice Gudielines, Journal of Hepatology 2017; 67:145–172. 2) Nguyen et al, Best Pract Res Clin Gastroenterol 2010; 24(5): 647–654. 3) Kim et al, Gastroenterology 2000;119:1631–1636). 4) EASL PBC Clinical Practice Gudielines, Journal of Hepatology 2017; 67:145–172. 5) Company estimate based on prevalence reported by Kim et al Gastroenterology 2000; 119(6):1631-6. 6) Company estimate based on prevalence reported by Kim et al Gastroenterology 2000; 119(6):1631-6 and Nguyen et al, Best Pract Res Clin Gastroenterol 2010; 24(5):647-54.

The disease

A progressive chronic autoimmune disease of the liver

The bile ducts are destroyed by inflammatory processes, bile accumulates in the liver causing an increase in the liver volume (cholestasis)

If untreated, the active liver tissue is destroyed and replaced by fibrous tissue, cirrhosis and liver transplant

Medical need

Ursodeoxycholic acid (UDCA) and obeticholic acid (Ocaliva) are the only FDA-approved medical treatments for PBC3

No targeted anti-inflammatory therapy is registered in the US or Europe

Previous trials indicate that corticosteroids may alleviate symptoms and improve biochemical and histologic findings4

Estimated prevalence

140,0005

Annual US incidence 0.3 – 5.8

per 100,0002

Primary Biliary Cholangitis competition


Standard of careUDCA

Nefecontargeted budesonide

FXR agonistsOCALIVA (Intercept)

Cilofexor (P2, Gilead)*Tropifexor (Novartis)*

EDP305 (P2, Enanta)**

Anti-fibroticsAldafermin (P2, NGM Biopharma)*

Setanaxib (P2, Genkyotex)*

PPAR agonistsBezafibrate (P3, Acad*; Intercept)

Elafibranor (P2, Genfit)*Seladelpar (P3, Cymabay)**

Saroglitazar (P2, Zydus Cadila)***

Primary BiliaryCholangitis

* Completed. ** Primary completion 2020. *** Primary completion 2019Public sources

Unique position addressing local autoimmune reactions

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Going forward: focus on Nefecon program & project pipeline

H1 2018

• IPO raising $82m on Nasdaq OMX

Ongoing updates regarding commercial strategy and plans

H2 2018 H1 2019 H2 2019 H1 2020 H2 2020 2021 2022

• NEFIGARD first patient in

• Application for ODD for second indication submitted

• Application for ODD for third indication submitted

• Filing of Pediatric Investigational Plan submitted to EMA

• Approval of ODD designation for second indication

• Approval of ODD designation for third indication

• EMA meeting to discuss surrogate marker

• 200 patients recruited

• In-licensing of additional product

• Out-licensing of major territory rights

• FDA interaction enhancing Part B design

• Clinical trial initiation of chronic dosing study with Nefecon

• EMA decision regarding pediatric pathway

• FDA meeting regarding regulatory pathway for AIH indication

• Initiation of roll-over study for re-treatment

• Top line read out for 200 patients

• Study fully recruited

• Initiation of roll-over study

• Filing with regulatory agencies for market approval

• Open study results of chronic / repeat dosing trials

• Commercial launch of Nefecon in H1

• Analysis based on 360 patients for validation of surrogate marker around mid 2022


Strong cash position fully finances ongoing clinical development

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Cash position $56m (SEK 534m) end of Q2

Additional $21.5m (SEK 210m) raised in Q3, attracting specialist US investor California-based BVF Capital Partners L.P. took a significant position Incremental funds enable initiation of additional study with Nefecon

$15m (SEK 140m) paid from China deal in Q3

Financed well beyond NDA filing

Ability to pursue additional Phase 3 supporting trials, as well as pursue attractive in-licensing options selectively


$121m (SEK 1.1bn) out licensing deal for Greater China validates future commercial potential

Investment Summary Calliditas

Novel treatment of IgA nephropathy (IgAN) with potential disease modifying effect targeting the origin of the disease

Strong cash position fully finances ongoing clinical development portfolio

Ongoing pivotal clinical Phase 3 study NefIgArd replicates successful Phase 2b

Additional potential for pipeline development: orphan liver indications and targeted in-licensing

Significant unmet medical need with no approved drugs. US annual market opportunity of around $2bn. Combined opportunity in China and Europe of at least equal size.

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