160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap...

28
September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik Tiberg, President & CEO

Transcript of 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap...

Page 1: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

September 8, 2016

Carnegie Small & Mid Cap Seminar 2016

CamurusAdvancing late stage pipeline with high market potential

Fredrik Tiberg, President & CEO

Page 2: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Forward-looking statements

This presentation contains forward-looking statements that provide our expectations or forecasts of future events such as new product developments and regulatory approvals and financial performance. Camurus is providing the following cautionary statement. Such forward-looking statements are subject to risks, uncertainties and inaccurate assumptions. This may cause actual results to differ materially from expectations and it may cause any or all of our forward-looking statements here or in other publications to be wrong. Factors that may affect future results include currency exchange rate fluctuations, delay or failure of development projects, loss or expiry of patents, production problems, unexpected contract, patent, breaches or terminations, government-mandated or market-driven price decreases, introduction of competing products, Camurus‘ ability to successfully market products, exposure to product liability claims and other lawsuits, changes in reimbursement rules and governmental laws and interpretation thereof, and unexpected cost increases. Camurus undertakes no obligation to update forward-looking statements

2

Page 3: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Camurus in brief

• Innovation that delivers− Award-winning FluidCrystal® technology

− Broad, advanced and de-risked development pipeline

− Strategic collaborations with strong partners

• Patient centric product development− Long-acting medications for better treatment

outcomes and quality of life for patients

− Meaningful product differentiation and positive pharmacoeconomic impacts

− Focus on attractive specialty markets and disease areas in need of new solutions

• Entrepreneurial company culture − Agile, passionate, collaborative and

result focused

− 60 employees with 43 in R&D

LISTED ON

NASDAQ STOCKHOLM

3rd DEC, 2015

MARKET CAP

~3.5billion SEK

CASH POSITION

549million SEK

END Q2 2016

3

Page 4: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Building value throughout the medical product life-cycle

Time and cost effective development by combining clinically documented active ingredients with a strong proven technology

Value created

Value Spent

Time

Decreased time to market505(b)(2), hybrid regulatory pathway

0

Value created

Value Spent

Time

Deeper market penetrationBest-in-class treatment potential

0

Value created

Value Spent

Time

Expanding end of cycle salesLong-acting barriers to generics

0

4

Page 5: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Leader in lipid science and formulation technologies

The FluidCrystal® technology is based on functional liquid crystal nanostructures

FluidCrystal® injection depot

FluidCrystal® topical bioadhesive

FluidCrystal® nanoparticleswater lipid 2

lipid 1

H2

L

I2

L2

5

+400PATENTS &

APPLICATIONS

16CLINICAL

TRIALS WITH FC TECHNOLOGY

Page 6: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

FluidCrystal® injection depot for longer lasting treatment effects

Long-acting release with weekly and monthly dosing

Good safety and local tolerability

Easy and convenient dosing

Applicable across substance classes

Standard manufacturing processes

~1400SUBJECTS HAVE

RECEIVED >10,000 INJECTIONS IN

CLINICAL TRIALS

6

Page 7: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Strong collaborations with strategic partners

CAM2029, CAM4071 + other products

• Exclusive, worldwide, collaboration, option and license agreement for CAM2029 and related products

CAM2038

• Exclusive license agreement for North America and option rights in Japan, South Korea, Taiwan and China

• USD 50 million received in upfront, option exercise and development milestones

• USD 700 million in total potential development and sales milestones

• Mid to high single digit % royalties on sales

• USD 20 million in upfront license fee received

• USD 130 million in total potential development and sales milestones

• Mid double digit % royalties on sales

Acromegaly, neuroendocrine tumours and other indications Opioid dependence and pain

Scop

eFi

nanc

ials

Fiel

d

CAM4072

• Worldwide license to use FluidCrystal®Injection depot for setmelanotide

• USD 65 million in potential development and sales milestones

• Mid to mid-high single digit % royalties on sales

“New Hope in The Search for Treatment for Obesity”, August 26, 2016”

Genetic obesity

7

Page 8: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Long-acting release from FluidCrystal® injection depot

FluidCrystal® injection depot Immediate release octreotide (Sandostatin®)

0,01

0,1

1

10

100

1000

0 5 10 15 20 25 30

Pla

sma

conc

entra

tion

(ng/

mL)

Time (days)

FC pasireotide

FC octreotide

FC somatostatin 1-14

0,01

0,1

1

10

100

1000

0 7 14 21 28

Pla

sma

conc

entra

tion

(ng/

mL)

Time (days)

subcutaneous octreotide

Single dose injection at t=0; n=6 (SC); rodent; mean values

8

Page 9: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Solid financial position and increasing investments in late-stage development programs

Key figures, kSEK Q2 2016 H1 2016 Q2 2015 H1 2015

Revenues 25,834 45,714 22,686 81,226

Operating result -25,881 -50,739 -31,645 -30,464

Cash 548,983 136,276

Total assets 660,261 210,922

Equity 603,837 106,803

0

50

100

150

200

250

2012 2013 2014 2015

Revenues

Licensepayments

Milestonepayments

Net sales;services andproducts

50

100

150

200

2012 2013 2014 2015

Operating expenses

Research & dev

Sales & marketing

Administration

9

Page 10: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Advancing pipeline of innovative treatment options

PARTNERS PRODUCT PRECLINICAL PHASE 1/2 PHASE 3 REGISTRATION

CAM2038 Weekly Opioid dependence

CAM2038 Monthly Opioid dependence

CAM2029 Neuroendocrine tumours

CAM2029 Acromegaly

CAM2038 Weekly Chronic pain

CAM2038 Monthly Chronic pain

CAM2032 Prostate cancer

CAM4071 Not disclosed

Page 11: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

PRODUCT EVENT COMMUNICATED

CAM2038 Opioid dependence Enrollment completed in two Phase 3 trials

Positive results from pivotal Phase 2 opioid challenge trial

April, 2016

May, 2016

CAM2038 Chronic pain Enrollment completed in Phase 2 trial in chronic pain patients April, 2016

CAM2029 Acromegaly & NET Positive topline results from Phase 2 trial in acromegaly and NET July, 2016

CAM2032 Prostate cancer Positive results from Phase 2 trial in prostate cancer June, 2016

CAM4071 Not disclosed indication Phase 1 clinical trial completed

New clinical product candidates Decision to initiate clinical development and trials of two new candidates June, 2016

11

Clinical development highlights 2016

Page 12: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Promising product candidates with large potentials

Page 13: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Opioid dependence is a growing global health problem

• About 4 million diagnosed with dependence in the US and in Europe, 15 million globally‒ Largest burden to society of all drugs1

‒ About 2 million in medication assisted treatment in the US and in Europe2,3

‒ 12 dollar saved for each dollar spent on treatment4

• The opioid epidemic‒ 28,647 opioid overdose deaths in the US in 2014,

18,893 from prescription drugs – a fourfold increase in 15 years

‒ Growing concern with prescription drug dependence also in EU

• Limitations of current treatment options‒ Frequent relapses to abuse and limited compliance ‒ Extensive misuse, abuse and diversion ‒ Pediatric exposure‒ Quality of life and mortality‒ Stringent treatment rules and regulations

Deaths of opioid overdoses in the US5

Thousands

Source: 1. UNODC, World Drug Report 2015; 2. SAHMSA, National Survey on Drug Use and Health (NSDUH) – 2014; 3. EMCDD, European Drug Report Trends and Developments 2015; 4. Center for Disease Control & Prevention 2016.; 5 WHO, UNAIDS position paper 2004.

”This epidemic is harming too many Americans and their families. But we know that – and your lives affirm – that treatment works and recovery is possible” said President Barack Obama

President’s Budget includes new mandatory funding to help ensure that all Americans who want treatment can get the help they need”

13

Page 14: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

CAM2038 A paradigm shift in opioid dependence treatment

• Weekly and monthly buprenorphine injections for all phases of opioid maintenance treatment

• Ready to use and easy to inject

• Fast Track designation by FDA for both weekly and monthly products

• Strategic partner for North America in BraeburnPharmaceuticals

• Best-in-class treatment potential

Long-acting release and continuous treatment effectReduced number of administrations from 365 to 52 or 12 doses per yearSafeguard against misuse, abuse and diversionNo risk of accidental pediatric exposure

Sustained blocking of subjective opioid effects

Flexible doses and durations allow individualized therapy in all OMT phases

CAM2038 key attributesCAM2038 overview

14

Page 15: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Plasma buprenorphine for CAM2038 versus sublingual tablets

0

1

10

100

0 7 14 21 28

Pla

sma

BP

N c

once

ntra

tion

(ng/

mL)

Time (days)

Conc (q1w 16 mg obs) Conc (q1w 16 mg pred) Conc (q4w 64 mg obs) Conc (q4w 64 mg obs SD)

Conc (q4w 64 mg pred SS) Conc (SL BPN 8 mg obs) Conc (SL BPN 8 mg pred)

Note: obs = observed, pred = predicted, SD = single dose, SS = steady state, SL = sublingual

15

Page 16: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Clinical program for CAM2038 in opioid dependence

HS-11-426 Phase 1 60 healthy volunteers

HS-13-487 Phase 1 87 healthy volunteers

HS-07-307 Phase 2 41 patients

Trial no. Subjects Key results / Study design Status

Dose proportional extended release further supported by pharmacodynamics results for withdrawal symptoms over time and time to rescue medication

Extended release suited for weekly respective monthly dosing. 6-8 times higher bioavailability. Acceptability of CAM2038 dosing higher than SL tablets.

Extended release of BPN suited for once weekly dosing. Dose proportional exposure. 6-8 times higher bioavailability versus SL BPN tablets

HS-14-478 Phase 2HS-14-549 Phase 2

Opioid challenge study of CAM2038 in opioid dependent patients (US)Repeat dose pharmacokinetic study of CAM2038 in opioid dependent pain patients (US), including injections in different subcutaneous injection sites)

Positive top-line resultsEnrolment completed

HS-11-421 Phase 3

HS-14-499 Phase 3

Double blind, double dummy Phase 3 efficacy trial of CAM2038 versus sublingual buprenorphine (US)Open label Phase 3 long-term safety trial in patients with opioid dependence (EU, US, AUS)

Enrolment completed

Enrolment completed

Good safety and local tolerability forCAM2038, weekly and monthly formulations

7COMPLETED

AND ONGOING CLINICAL TRIALS

+900PEOPLE DOSED WITH CAM2038, WEEKLY AND

MONTHLY PRODUCTS OR

PLACEBO

16

Page 17: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Strategy of own commercialisation of CAM2038 in Europe

Positive market drivers support pricing strategy and reimbursement on European markets

Favourable market

Improved compliance and quality of life

Sizeable socio-economic benefits

On-going paradigm shift

Accessible and concentrated market

Cost efficient roll-out

Rationale Overview of market rights and Camurus’ primary markets

Braeburn exclusive markets Braeburn option right Camurus markets

17

Page 18: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Highly addressable target markets in Europe

Market structure for opioid dependence Physicians willingness to prescribe CAM20381Country# OMT patients % buprenorphine Treatment location

Large markets with known physician demand

77,300

172,513

163,000

94,376

21%

25-30%

66%

15%

Specialised centers and primary health care system

Community health clinics and NHS providers

Specialised centers and GP practices

Servizi Tossicodipendenze (Ser.T.) and private and non-profit organisations

86%N=51

94%N=50

86%N=50

96%N=50

Source. 1. Market access dynamics in opioid addiction, Decision Resources 2015

18

Page 19: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Establishment of our commercial organisation

• Building of the commercial organisation for the anticipated launch of CAM2038 on track

• Internationally experienced leadership teamon board

• Engagement initiated with stakeholders to realise optimum product value‒ Politicians, policy makers, payers, prescribers and

patients

• Fully built out, the European commercial organization will include about 70 to 120 people.

19

2016• EU leadership team• GMs in early reimbursed markets• Pricing, market access, medical

affairs

2017• Regional leadership teams

early reimbursed markets• GMs 2nd wave markets

2018• Regional leadership teams

2nd wave markets• Full key account teams for

CAM2038 launch

Pre-launch activities• Publications• Market research• Policy and education• Health economic outcome research• Market access, price & reimbursement

Page 20: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Chronic pain is another significant market opportunity for CAM2038

• Over 200 million people with chronic pain in Europe and the US

• Global market for chronic pain exceeded 20 billion USD, US market 10.8 billion USD in 20121

• Costs estimate to US society is $560-635 billion annually2, exceeding costs of heart disease, cancer and diabetes.

• Clinical Phase 2 trial under completion

• Phase 3 program in preparation‒ Reuse of data from opioid dependence program

Round-the-clock pain reliefLong acting duration Improved treatment complianceReduced overdose riskMinimal risk of misuse, abuse, and diversion

CAM2038 potential advantagesCAM2038 Chronic pain overview

Source: 1 . Decision Resources; 2. IMS Health data 2015

20

Page 21: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Significant potential in converting Sandostatin® LAR ® patients to CAM2029

CAM2029 for treatment of acromegaly and neuroendocrine tumors

• Acromegaly is a rare, chronic and insidious hormonal disorder‒ Occurs when the pituitary gland produces excess

growth hormone (GH) and insulin-like growth factor-1 (IGF-1)

‒ Current gold-standard medical treatment include somatostatin analogues

• Neuroendocrine tumors (NETs) are rare and malignant neoplasms‒ Somatostatin analogues constitute the current

standard of safe and effective medical therapy for symptom control

‒ Somatostatin analogues also show anti-tumor effects

Overview of acromegaly and NET Strong market growth over 15 years1

1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014

Sandostatin® (Novartis) Somatuline® (Ipsen)(2)

Sandostatin® : 7%

Somatuline® : 16%

(USDm)CAGR 2004-2014:

2015

Source1. Medtrack

21

Page 22: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

CAM2029 A convenient, safe and effective treatment option

• Ready-to-use, long-acting octreotide for treatment of acromegaly and neuroendocrine tumours (NETs)

• Orphan drug designation for acromegaly by EMA

• Exclusive partnership with Novartis –market leader within acromegaly and NETs

• Positive Phase 2 results announced by Novartis and Camurus in July 2016‒ Well maintained control of disease symptoms and

biomarkers in NET and acromegaly patient when switching from Sandostatin® LAR®

‒ Good safety and local tolerability

• Phase 3 preparations ongoing for start in 2017

Easy subcutaneous administration using prefilled syringeSelf-administration option with significant convenience benefits and cost savingsIncreased bioavailability (500%) with potential for enhanced treatment efficacts1Thin needle and small injection volumesRoom temperature stability avoiding cold chain distribution and conditioning before use

CAM2029 key attributesCAM2029 overview

Source: 1. Tiberg F, Roberts J, Cervin C, et al. Br J Clin Pharmacol. 2015;80:460-472.

22

Page 23: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

No reconstitutionCAM2029 10, 20 mg0.5 -1.0 mL/ready-to-use/

no reconditioning/room temperature Based on FluidCrystal® system

Sandostatin® LAR® 10, 20, 30 mg 2.0 mL/reconstitution/

refrigerated/30-60 min reconditioningBased on PLGA microsphere system

Somatuline® Autogel® 60, 90, 120 mg0.2-0.5 mL/ready-to-use/refrigerated

≥ 30 min reconditioningSelf-associated gel

Small volume

Thin needle

≥22G

20G

18G/19G

Subcutaneous(12.5mm)

Intramuscular(40mm)

Deep subcutaneous(20mm)

Note: 1) Illustrative. Final product configuration may be different.

Ready for use with small needle and no need for reconstitution

23

Page 24: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Clinical trials confirm target properties of CAM2029

HS-05-194Phase 1 32 volunteers

HS-07-291Phase 1 95 volunteers

HS-11-411Phase 1 122 volunteers

Trial no. Subjects Key results / Study design Status

Rapid and long-acting release of octreotideand suppression of IGF-1. Dose proportional octreotide exposure with 5 times higher bio-availability compared with Sandostatin LAR 30 mg.

Dose proportional octreotide exposure during repeated dosing of CAM2029 mg.Rapid and long-acting release of octreotide

Rapid and long-acting release of octreotide One month suppression of the growth factor IGF-1.

HS-12-455Phase 2

12 patients in two groups with acromegaly and NETs

Positive results

Phase 3 Two Phase 3 trials of CAM2029 versus active control, Sandostatin® LAR®, in patients with neuroendocrine tumours (NETs) and acromegaly, respectively (Global) 3 trials in preparation

Good safety and local tolerability demonstrated in all trials

Randomised multi-centre study of the pharmacokinetics, pharmacodynamics, efficacy and safety of CAM2029 in two patients groups with acromegaly and neuroendocrine tumours (NET) previously treated with Sandostatin® LAR®

24

Page 25: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Pipeline expansion with new attractive clinical candidates

Inflammation & painCAM2041

DiabetesCAM2046

Cancer supportive careCAM2047

Post operative painCAM2048, CAM2058

Target indicationProject

UndisclosedCAM2043

Lead formulation selected

Formulation development

Lead formulation selectedClinical development start in Q4 2016

Lead formulation selectedClinical development start in Q4 2016

Status

Lead formulation selectedClinical development start in Q3 2017

CAM4072Setmelanotide FC

Genetic obesity (Prader-Willis syndrome, POMC deficiency)

Lead formulation selectedRhythm Inc. preparing for clinical trials

Early phase project evaluations, including three big pharma collaborations

25

Page 26: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

PARTNERS PRODUCT EVENT TIME

CAM2038 Opioid dependence Results Phase 3 efficacy trial

Results Phase 3 long-term trial

NDA and MAA submissions

Q4, 2016

Q1, 2017

Mid-2017

CAM2038 Chronic pain Start Phase 3 trial

Results Phase 2 trial in chronic pain patients

Results Phase 3 efficacy trial

Q3, 2016

Q4, 2016

H2, 2017

CAM2029 Acromegaly & NET Start Phase 3 trials in patients with acromegaly and NETs 2017

New clinical product candidates Start of Phase 1 trial for CAM2047, CAM2048, and CAM2058 Q4, 2016

Early partnerships Start of Phase 1 trial for weekly setmelanotide 2017

26

Significant clinical news flow expected during 2016-17

Page 27: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

Summary

• De-risked portfolio of differentiated late-stage assets‒ Opioid addiction, pain, cancer and acromegaly‒ Attractive multi-billion dollar specialty pharmaceutical markets‒ Concentrated prescriber audiences and active patient advocacy groups

• Strong collaborations with dedicated partners‒ Novartis, Braeburn Pharmaceuticals, Rhythm, Solasia, R-Pharm US‒ Early project collaborations with global pharma companies

• Several levers for pipeline and business expansion‒ New product candidates entering clinical development‒ Growth opportunities via technology platforms

as well as business development

• European commercial organisation‒ Leadership team established‒ Prelaunch activities for CAM2038 initiated

• Solid financial position

27

Page 28: 160908 Camurus presentation Carnegie Small Mid …...September 8, 2016 Carnegie Small & Mid Cap Seminar 2016 Camurus Advancing late stage pipeline with high market potential Fredrik

28