Untitled - The Medicines Company

Transcription

34TH ANNUAL JP MORGAN HEALTHCARE CONFERENCE
Clive Meanwell
Chief Executive Officer
Legal notices
Forward-looking statements
Statements contained in these slides
about The Medicines Company (the
“Company”), the Company’s
products and product candidates,
clinical trial results, regulatory
submissions, product or indication
launches, the Company’s future
financial and operating results, and
future opportunities for the
Company, that are not purely
historical, and all other statements
that are not purely historical, may be
deemed to be forward-looking
statements for purposes of the safe
harbor provisions under The Private
Securities Litigation Reform Act of
1995. Without limiting the foregoing,
the words “believes," “anticipates,"
“plans," “expects," “intends,"
“potential," “estimates," “outlook” and
similar expressions are intended to
identify forward-looking statements.
There are a number of important
factors that could cause actual
results or events to differ materially
from those indicated by such
3
forward looking statements,
including: the extent of the
commercial success of our
products; the Company's ability to
develop its global operations and
penetrate foreign markets; whether
the Company’s patent and other
litigation is resolved in a timely and
satisfactory manner; whether the
results of preclinical studies or early
clinical trials will be indicative of the
results of later clinical trials; whether
the Company’s product candidates
will advance in the clinical trials
process on a timely basis or at all;
whether the clinical trial results will
warrant submission of applications
for regulatory approval; whether the
Company will make regulatory
submissions for product candidates
on a timely basis or at all; whether the
Company’s product candidates will
receive approvals from regulatory
agencies on a timely basis or at all;
whether the Company’s ongoing and
planned commercial launches will be
successful; whether physicians,
patients and other key decision
makers will accept clinical trial
results, whether we can successfully
enter into strategic partnerships and
such other factors as are set forth in
the risk factors detailed from time to
time in the Company’s periodic
reports filed with the Securities and
Exchange Commission (“SEC”)
including, without limitation, the risk
factors detailed in the Company’s
Form 10-Q filed with the SEC on
November 9, 2015, which are
incorporated herein by reference.
The Company specifically disclaims
any obligation to update these
forward-looking statements. You
are cautioned not to place undue
reliance on these forward-looking
statements, which speak only as of
the date of this presentation. All
forward-looking statements are
qualified in their entirety by this
cautionary statement.
©The Medicines Company 2016
Current situation
Valuable portfolio addressing large markets
Cardiovascular
disease
Marketed Kengreal™
Angiomax®
Phase III
Proof-ofconcept
Preclinical
Resistant
infections
Anesthesia and
surgery
Orbactiv®
Minocin IV®
Ionsys®
Recothrom®
Cleviprex®
Raplixa™
Argatroban RTU
Preveleak™
1
Carbavance®
PCSK9si
MDCO-216
ABP-700
BLI program
1: 12/18/15 announced divestment (purchase) agreement with subsidiaries of
Mallinckrodt plc for a total potential consideration of up to $410 million
5
Current situation
Valuable portfolio addressing large markets
Fundamental analysis of riskadjusted asset valuations
—
—
—
2 year cash burn
Carbavance
Wide range of value
Significant short-term cash
requirements
Evident conglomerate discount
Orbactiv
MDCO-216
Ionsys
PCSK9 si
(range)
Kengreal
ABP700
Cleviprex
Size of circle proportional to risk-adjusted NPV
Probability of success-adjusted NPV calculated with
allocation of corporate overhead to each asset
proportionate to lifetime total expense for that asset
6
Minocin
-10
-5
0
5
Years from US launch
Strategy
Unlocking value
Focus on highest value assets & activities
• Develop four potential blockbusters
• Launch five hospital products
• Secure non-dilutive sources of capital
• Manage cash in disciplined manner
7
Develop four potential blockbusters
Key programs
1. PCSK9 synthesis inhibitor for LDL-C lowering
2. MDCO-216 for coronary plaque regression
3. ABP700 for intravenous sedation & anesthesia
4. Carbavance for Gram (-) CRE infections
8
PCSK9 synthesis inhibitor
Potential blockbuster for dyslipidemia
PCSK9 RNA interference
Small volume sc injection
Clamped knockdown of PCSK9
(up to 89%) & LDL-C (79%)
6 month duration at 300 mg
Aligns Dx – Rx cycle: potential
adherence advantages
PCSK9 synthesis inhibitor is an investigational agent not approved for commercial use in any market
9
Market opportunity in U.S.1
~74 million dyslipidemia
patients, 2014
~13 million initial target
population
Anticipated expansion
with CVOT data
US only
Lipid lowering therapy
~43.1 M
Primary
Prevention
~19.2 M
Secondary
Prevention
~23.9 M
Statin
intolerant
~4.4M
High Risk
(LDL-C >70
mg/mL)
~7.82 M
FH
~0.65M2
1. Decision Resources, 2014, AHA 2015 and NHANES
2. Familial Hypercholesterolemia; US Census Data
10
Orion development program
Anticipated development sequence
2015
2016
2017
2018
2019
2020
Anticipated non-critical path
Phase I LDL-C lowering
Anticipated critical path
CMC Development (scale up, formulation, device, and supply)
Phase III preparation
Non-clinical long-term toxicology (including repro and carc)
Phase II including HoFH & MAbs
Phase III LDL lowering
NDA/MAA process
Potential outcomes study
Timelines are estimates based on current assumptions
11
ORION 1 clinical trial in ASCVD
Multi-center phase II study
—
N = 480 patients
—
High CV risk and elevated LDLC on therapy
—
—
—
Placebo-controlled, doubleblind, randomized
Primary endpoint is % change
LDL-C
Canada, Germany,
Netherlands, UK, US
Day of study
Dose in mg
(patient-n)
1
30
60
90
120
150
180
210
Q 6 months cohort
Placebo (60)
200 (60)
300 (60)
500 (60)

IA
Primary
endpoint
day 180
Q 3 months cohort
Placebo (60)
100 (60)
200 (60)
300 mg (60)

12

MDCO-216 for coronary plaque regression
Potential blockbuster for ASCVD
Rationale
—
ApoA1 acts via ABCA1 which
mediates cholesterol efflux1,2
—
Loss of function increases risk of CV
disease3
—
Gain of function reduces risk of CV
disease & atherosclerosis2,3,4
—
ApoA1-Milano induces plaque
regression in rabbits and humans5,6
Cholesterol efflux is the first step in
reverse cholesterol transport2
1.
2.
3.
4.
5.
6.
Schmitz & Langmann. Curr. Opin. Lipidol. 2000; 12: 129–40.
Duffy et al. Circulation 2006;113:1140–1150
Bodzioch et al. Nature Genetics 1999; 22: 347–51.
Rohatgi et al. N Engl J Med. 2014. 371:2383-93
Ibanez et al. Atherosclerosis. 2012; 220:72-7
Nissen SE et al. JAMA. 2003;290:2292-2300
MDCO-216 is an investigational agent not approved for commercial use in any market
13
Atheroma Volume Regression
Change in Percentage Atheroma Volume
Previous investigators1
demonstrated significant
atheroma regression in ACS
patients after only 5-weeks
of treatment
0.5
0.14
0.0
-0.5
-0.73
-1.0
-1.06
p=0.02
-1.29
p=0.03
-1.5
Placebo
(N=11)
15mg/kg
(N=21)
45mg/kg
(N=15)
Combined
(N=36)
1. Nissen SE et al. JAMA. 2003;290:2292-2300
14
Using an improved compound
(MDCO216) a recently
published phase I study1
demonstrated:
Absolute change from baseline in ABCA1 efflux:
CAD Patients
Cohort 6 (10
mg/kg)
Cohort 7 (20
mg/kg)
Cohort 8 (30
mg/kg)
Cohort 9 (40
mg/kg)
16
14
—
—
—
Profoundly increased ABCA1mediated cholesterol efflux
ApoA1-Milano phenotype (low
HDL-C & raised TG)
Well tolerated
Change of ABCA1
(% efflux/4h)
12
10
8
6
4
2
0
-2
-4
0 0.5 2
4
8
24
48
144
696
Time (hr)
Kallend et al. Eur Heart J. published online Dec 10th 2015
15
Milano-Pilot
Multi-center study
—
N = 120 patients (interim at 40 and 80)
—
Qualifying acute coronary syndrome event within 14 days
—
Placebo-controlled, double-blind, randomized trial
—
5 weekly doses of MDCO-216 at 20 mg/kg vs placebo
—
Primary endpoint is % atheroma volume (PAV) change on intravascular
ultrasound
—
Canada, Netherlands, Hungary, Czech Republic, Poland, US
16
Managing dyslipidemia
MDCO-216 and PCSK9si complementarity
Continuum of care – aiming for significant mitigation of risk
Patients
with CV
risk factors
requiring
therapy
ASCVD
ACS event
US
900,000
MDCO 216
Reduce risk
associated with
vulnerable plaque
PCSK9 si
Reduce risk of
secondary events
PCSK9 si
Reduce risk of
primary ASCVD
events
US
74 million
MDCO-216 and PCSK9 si are investigational agents not approved for commercial use in any market
17
Develop potential blockbusters
ABP700 intravenous sedation | anesthesia
Substantial unmet medical need & opportunity
~184 million US & EU procedures
Unique attributes of ABP700 demonstrated in
phase I program
—
Rapid onset and offset of effect
—
Minimal respiratory depression
—
No adrenal suppression
—
Water soluble, no pain on injection
Phase II program in 2016
Minimum
sedation/anxiolysis
Moderate/sedation
analgesia (previously
conscious sedation)
“MAC” - deep
sedation/analgesia
General anesthesia
ABP-700 is an investigational agent not approved for commercial use in any market.
18
Carbavance for gram- CRE infections
Gram (-) MDR infection rates rising worldwide
Organism (no. tested)
Resistanceb
E. coli (5,118)
Klebsiella spp (3,219)
USA
China
Europe
LATAMb
ESBL
12.8 (+)
73.8 (+)
19.4 (+)
37.1 (+)
CRE
0.4 (+)
2.1 (+)
0.1 (NC)
0.0 (NC)
ESBL
14.6 (NC)
44.2 (+)
43.4 (+)
50.7 (NC)
CRE
4.4 (NC)
4.3 (+)
8.7 (+)
9.7 (+)
Five year trends (2007–2011): “+” = increasing rate; “-” = decreasing rate; and NC = no significant
change. LATAM=Latin America
ESBL=extended spectrum β-lactamases; and CRE=carbapenem-resistant
JMI Laboratories from the SENTRY antimicrobial surveillance program.
CRE infection mortality rate 25-40%
Carbavance is meropenem, plus RPX7009
Phase III trials recruiting
RPX7002
Cyclic boronic acid
compound
Potent inhibitory
activity against Class
A and C serine betalactamases
Pharmacology
optimized for
carbapenems
CARBAVANCE™ is an investigational agent not approved for commercial use in any market
19
Carbavance phase III nearing completion
TANGO I
TANGO II
Patients
Complicated UTI and AP
CRE suspected cUTI, HABP,
VABP and/or bacteremia
Design
Randomized 1:1
Double blind
Randomized 2:1
Open label
Comparator
Piperacillin/Tazobactam
Best available therapy
Total patients
550 (previously 850)
Up to 150
Study sites
~140
~60
FDA/EMA guidance
Yes
Yes
Supported in part under contract with the Biomedical Advanced Research and Development Authority (BARDA).
CARBAVANCE™ is an investigational agent not approved for commercial use in any market
20
Carbavance for gram- CRE infections
Estimated initial market opportunity ~1 million patients1
Resistance strains expected
to be susceptible2
Enterobacteriaceae
to Carbavance:
infections worldwide
~90% US and
~7.5 million
~80% EU
Target
Population
~700,000
Carbapenem
resistant
~750,000
Pseudomonal
infections worldwide
~900,000
Carbapenem
resistant
~270,000
Target
Population
~250,000
1. Decision Resources; Company primary and secondary research.
2.In vitro data do not necessarily correlate to clinical efficacy.
Carbavance® is an investigational agent not approved for commercial use in any market
21
Unlocking value
Five hospital products in launch phase
Key programs
1. Kengreal® for percutaneous coronary intervention
2. Cleviprex® for acute severe hypertension
3. Orbactiv® for G+ infections including MRSA
4. Minocin IV® for resistant Acinetobacter infections
5. Ionsys® for post-operative pain
22
Cardiovascular hospital product launches
Kengreal® and Cleviprex®
Kengreal®
—
For patients undergoing PCI who have not
received an oral P2Y12 inhibitor or in whom the
oral P2Y12 inhibitor preload is not feasible or
desirable
Cleviprex®
—
23
For the reduction in blood pressure when oral
therapy is not feasible or not desirable
Cardiovascular hospital product launches
Kengreal® and Cleviprex®
Kengreal®
― Formulary acceptance
>90%
– C-Code effective Jan 1st
2016
Cleviprex®
– 36% (Q/Q) growth in
formulary wins
Formulary approvals
# cumulative approvals, US
400
Cleviprex
New accounts
# cumulative new accounts, US
Kengreal
350
350
300
250
250
200
200
150
150
100
100
50
0
0
Q1
Q2
Q3
2015
24
Kengreal
300
50
– 76% (Q/Q) growth in
new account wins
Cleviprex
Q4
Q1
Q2
Q3
Q4
2015
Antibacterial hospital product launches
Orbactiv®
Orbactiv®
—
Lipoglycopeptide for ABSSSI
—
Gram + organisms including MRSA
—
Single dose administration (1200 mg)
—
Rapid concentration dependent bacteriocidal activity
25
Vancomycin MIC = 1 ug/ml
Oritavancin MIC = 0.06-0.12 ug/ml
Bacterial burden (log CFU/mL)
Bacterial burden (log CFU/ml)
Antibacterial hospital product launches
Orbactiv®
Progression of formularies, accounts and sales
ORBACTIV® Formulary adoption
# cumulative approvals, US
400
300
200
100
0
Q1
Q2
Q3
2015
Q4
ORBACTIV® accounts ordering
# cumulative new accounts, US
ORBACTIV® Sales
# treatments, US
700
600
500
400
300
200
100
0
1,200
1,000
800
600
400
200
0
Q1
Q2
2015
Q3
Q4
Q1
Q2
Q3
Q4
2015
Permanent J-code for hospital outpatient and freestanding infusion centers - reimbursement
effective 1st January 2016 at ASP plus 6%
26
Surgical care hospital product launch
Ionsys® for post-operative pain
Market of >50 million patients worldwide
Ionsys®
—
Drug-device system containing fentanyl given transdermally under patient self-control
—
Approved by FDA and EC in 2H 2015 for short term
treatment of post-operative pain in adults
—
Advantages over existing opioid administration for caregivers and patients
27
Advantages over existing opioid administration for
care-givers and patients
More nurses favored Ionsys® on
ease-of-care satisfaction over IV
PCA
75% of nurses rated Ionsys®
more highly than IV PCA for
time-efficiency
More Ionsys® patients reported
no interference with mobility
compared to IV PCA
% of nurse responses
% of nurse responses
% of patients reporting no
mobility interference
Neutral
Morphine
IV PCA
30
95
Neutral
8
17
[VA
LUE
]
53 Ionsys®
17
Morphine
IV PCA
75
Ionsys®
Ionsys®
28
Morphine IV
PCA
Value proposition
—
—
—
29
PCA medication errors
cost $6,943 per event
PCA device errors cost
$7,281 per event
Process efficiency of
Ionsys® reduces nursing
time 30-40 minutes per
patient
Cost type
Patient
outcomes:
Safety
Process
improvement
outcomes
Disposable
and capital
equipment
efficiencies
Cost with
Ionsys®
Cost with
IV PCA
Ionsys®
savings
~$1100-1150 ~$1350-1400
~$275
~$25
~$75
~$50
$0
~$85
~$85
Total savings
~$410
Restructuring the firm
Divestment of surgical hemostat products
—
Transaction with subsidiaries of Mallinckrodt plc announced
on 18 December
—
Total potential consideration of up to $410 million
Initial payment of ~$175 million
Additional milestones $235 million
—
Pending HSR review, expected to close in Q1 2016
—
Strategic rationale
Inflow of non-dilutive capital
Reduction of launch costs
Focus on core programs
30
Main policies
Anticipated news flow for 2016
Series of key events throughout the year
Anticipated event
Timeframe

Start phase II for PCSK9 si
January

Start phase II for ABP-700
1H

Interim data from Milano-Pilot study for MDCO-216
1H

Data from phase III for Carbavance
2H

Data from phase II for PCSK9 si
Q4

Initiate phase III for PCSK9 si
Q4

Progression of hospital launch products
Quarterly
31
Strategy
Unlocking value
Focused on highest value assets & activities
• Developing four potential blockbusters
• Launching five hospital products
• Securing non-dilutive sources of capital
• News flow anticipated to drive significant value
32

Untitled - The Medicines Company

Transcription

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