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6-K

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

 

FORM 6-K

 

 

REPORT OF FOREIGN PRIVATE ISSUER

PURSUANT TO SECTION 13a-16 OR 15d-16

UNDER THE SECURITIES EXCHANGE ACT OF 1934

May 13, 2015

 

 

Commission File Number: 001-36815

 

 

Ascendis Pharma A/S

(Exact Name of Registrant as Specified in Its Charter)

 

 

Tuborg Boulevard 12

DK-2900 Hellerup

Denmark

(Address of principal executive offices)

 

 

Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F.

Form 20-F  x            Form 40-F  ¨

Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1):  ¨

Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7):  ¨

 

 

 

Representatives of Ascendis Pharma A/S (the “Company”) plan to present the information in the presentation attached hereto as Exhibit 99.1 on May 13, 2015 at the Bank of America Merrill Lynch 2015 Health Care Conference.

The furnishing of the attached presentation is not an admission as to the materiality of any information therein. The information contained in the presentation is summary information that is intended to be considered in the context of more complete information included in the Company’s filings with the Securities and Exchange Commission (the “SEC”) and other public announcements that the Company has made and may make from time to time by press release or otherwise. The Company undertakes no duty or obligation to update or revise the information contained in this report, although it may do so from time to time as its management believes is appropriate. Any such updating may be made through the filing or furnishing of other reports or documents with the SEC, through press releases or through other public disclosures.

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

Ascendis Pharma A/S
Date: May 13, 2015 By:

/s/ Thomas P. Soloway

Thomas P. Soloway
Senior Vice President, Chief Financial Officer

EXHIBIT INDEX

 

Exhibit
No.

   

Description

99.1    Company Presentation.
EX-99.1
ASCENDIS PHARMA
Jan Mikkelsen, President and CEO
Company Presentation, Q2 2015
1
Q2 2015
Exhibit 99.1

CAUTIONARY NOTE ON FORWARD LOOKING STATEMENTS:
2
Q2 2015
This
presentation
contains
forward-looking
statements.
All
statements
other
than
statements
of
historical
facts
contained
in
this
presentation,
such
as
statements
regarding
our
future
results
of
operations
and
financial
position,
business
strategy,
prospective
products,
availability
of
funding,
clinical
trial
results
(including
the
timing
of
data
from
our
ongoing
Phase
2
pediatric
study
of
TransCon
hGH),
product
approvals
and
regulatory
pathways,
collaborations,
timing
and
likelihood
of
success,
plans
and
objectives
of
management
for
future
operations,
and
future
results
of
current
and
anticipated
products,
are
forward-looking
statements.
These
forward-looking
statements
are
based
on
our
current
expectations
and
beliefs,
as
well
as
assumptions
concerning
future
events.
These
statements
involve
known
and
unknown
risks,
uncertainties
and
other
factors
that
could
cause
our
actual
results
to
differ
materially
from
the
results
discussed
in
the
forward-looking
statements.
These
risks,
uncertainties
and
other
factors
are
more
fully
described
in
our
reports
filed
with
or
submitted
to
the
Securities
and
Exchange
Commission,
including,
without
limitation,
our
most
recent
Annual
Report
on
Form
20-F,
particularly
in
the
sections
titled
“Risk
Factors”
and
“Management’s
Discussion
and
Analysis
of
Financial
Condition
and
Results
of
Operations.”
In
light
of
the
significant
uncertainties
in
our
forward-looking
statements,
you
should
not
place
undue
reliance
on
these
statements
or
regard
these
statements
as
a
representation
or
warranty
by
us
or
any
other
person
that
we
will
achieve
our
objectives
and
plans
in
any
specified
timeframe,
or
at
all.
Any
forward-looking
statement
made
by
us
in
this
presentation
speaks
only
as
of
the
date
of
this
presentation
and
represents
our
estimates
and
assumptions
only
as
of
the
date
of
this
presentation.
Except
as
required
by
law,
we
assume
no
obligation
to
update
these
statements
publicly,
whether
as
a
result
of
new
information,
future
events
or
otherwise
after
the
date
of
this
presentation.
This
presentation
concerns
product
candidates
that
are
under
clinical
investigation
and
which
have
not
yet
been
approved
for
marketing
by
the
U.S.
Food
and
Drug
Administration,
European
Medicines
Agency
or
other
foreign
regulatory
authorities.
These
product
candidates
are
currently
limited
by
U.S.
Federal
law
to
investigational
use,
and
no
representations
are
made
as
to
their
safety
or
effectiveness
for
the
purposes
for
which
they
are
being
investigated.
Ascendis
is
a
trademark
that
we
use
in
this
presentation.
Any
other
trademarks
appearing
in
this
presentation
are
the
property
of
their
respective
holders.

INVESTMENT HIGHLIGHTS
*USD/EUR=1.076 as of March 31, 2015
TransCon technology is applied to create long-acting prodrugs with best-in-class profiles
Internal programs targeting multi-billion dollar orphan indications:
-
Growth disorders and pulmonary arterial hypertension
Capital to complete Phase 3 study of TransCon Growth Hormone
March
31
,
2015
cash
balance:
~€153
million*
High-value collaborations with Large Cap Pharma in their key strategic therapeutic areas
-
Roche Genentech in ophthalmology
-
Sanofi in diabetes
Existing and future collaborations are a potential source of significant revenue
-
Approximately €202 million* in development and regulatory milestones for three product candidates
-
Eligible for royalties from mid-single to low teen digit percentages from ophthalmology collaboration
All TransCon products eligible for new composition of matter IP
TransCon technology is broadly applicable with no third-party royalty or milestone payment
obligations
Technology &
Pipeline
Strong
Balance Sheet
Partners
Partner
Revenue
Intellectual
Property
3
Q2 2015
st

EXPERIENCED LEADERSHIP TEAM
4
Jan Mikkelsen, Director, President and CEO
LifeCycle Pharma (now known as Veloxis), Maxygen,
Profound Pharma, Novo Nordisk
Martin Auster, MD, SVP & CBO
United Therapeutics, Wachovia, GLG Partners
Michael Wolff Jensen, LLM,
Chairman, SVP & General Counsel
Dong Energy, Veloxis, Genmab
Grethe
Rasmussen,
PhD,
SVP
Development
Maxygen, Novo Nordisk
Dr. Harald Rau, SVP & CSO
Complex Biosystems
Thomas P. Soloway, SVP & CFO
Transcept, Montreux Equity Partners
Lotte Sønderbjerg, SVP & CAO
Veloxis, Acadia, Novo Nordisk
Management
Michael Wolff Jensen, LLM,
Chairman, SVP & General Counsel
Dong Energy, Veloxis, Genmab
Albert Cha, MD, PhD
Vivo Capital, Managing Partner
Edwin de Graaf
Gilde Healthcare Partners, Managing Partner
James Healy, MD, PhD
Sofinnova Ventures, General Partner
Michael Mayer
TechnoStart, Managing Partner
Jan Mikkelsen, President and CEO
LifeCycle Pharma (now known as Veloxis), Maxygen,
Profound Pharma, Novo Nordisk
Martin Olin
Symphogen A/S, CFO
Jonathan Silverstein, JD
Orbimed, Co-Head of Global Private Equity
Rafaèle Tordjman, MD, PhD
Sofinnova Partners, Managing Partner
Board of Directors
Q2 2015

TRANSCON TECHNOLOGY
Predictable self-cleaving
of TransCon linker
Parent
drug
Inactive long-acting prodrug 
Unmodified active parent drug released
Long-acting prodrugs with predictable release of unmodified parent drugs
Parent drugs can be proteins, peptides or small molecules
Predictable release of parent drug supporting up to half-yearly administration
TransCon linker release dependent only on pH and temperature
Enables systemic or localized drug exposure depending upon choice of TransCon carrier
Same mode-of-action as parent drug molecule
Potential
to
improve
development
success
rate
when
incorporating
approved
parent
drug
TransCon carrier
TransCon linker
Parent
drug
Parent
drug
5
Q2 2015

BROAD, BALANCED AND DIVERSE PIPELINE
Product Candidate
Primary Indication
Approved
Parent
Drug
Stage of Development
Market Size
Worldwide
Commercial
Rights
TransCon
Growth Hormone
Pediatric Growth
hormone deficiency
Phase 2
(Topline data expected mid-2015)
>
$3
billion
Adult Growth hormone
deficiency
Phase 2 completed
TransCon Treprostinil
Pulmonary Arterial
Hypertension
Phase 1
>
$1
billion
TransCon Ranibizumab
Ophthalmology
Preclinical
>
$5
billion
TransCon Peptide
Diabetes
Preclinical
n/a
6
Q2 2015

TransCon hGH: Once-weekly growth hormone
7
Q2 2015

ONCE-WEEKLY TRANSCON GROWTH HORMONE
The daily hGH market is a $3+ billion orphan opportunity
Current therapies require daily injections, which can result in poor compliance and
suboptimal treatment response
1
TransCon Growth Hormone is a long-acting prodrug designed to release
unmodified growth hormone in the bloodstream
Maintains the same mode-of-action as daily hGH
Efficacy, safety, tolerability and immunogenic profile comparable to daily hGH
Phase 2 study in adults with growth hormone deficiency: Comparable dose
response and tolerability to daily hGH therapy
Phase 2 study in children with growth hormone deficiency ongoing
1
PLoS ONE 2011, 6(1), e16223
8
Q2 2015

Global sales of daily hGH products exceeded $3 billion in 2013
The daily hGH market is fragmented and undifferentiated
Novo Nordisk, Pfizer, Eli Lilly, Sandoz, Merck KGaA and Roche account for approximately
95% of volume market share
Pediatric indications comprise up to 90% of the market
Indications for growth hormone treatment include:
Growth Hormone Deficiency (GHD)
Turner Syndrome (TS)
Idiopathic short stature (ISS)
Prader-Willi Syndrome (PWS)
Small for Gestational Age (SGA)
GROWTH HORMONE MARKET
Ascendis market research
9
Q2 2015

POOR COMPLIANCE REDUCES TREATMENT OUTCOMES
Poor compliance with daily growth hormone therapy is associated with reduced
height velocity and impaired quality of life
Two
out
of
three
of
the
patients
miss
more
than
one
injection
on
average
per
week
Reduced frequency of administration is associated with better compliance
Once-weekly TransCon Growth Hormone may improve compliance and overall
treatment outcomes
1
PLoS
ONE
2011,
6(1),
e16223
2
Clinical
Therapeutics,
2008,
30(2),307
Doses missed
per week
Higher
compliance
Improved growth
response
10
Q2 2015
1
2

TRANSCON hGH: LONG-ACTING UNMODIFIED GROWTH HORMONE
Q2 2015
11
TransCon Growth Hormone is designed to provide the efficacy, safety and
tolerability of daily hGH with once-weekly dosing
Height
Velocity
Metabolic
effects
Quality
of Life
Safety
Injection
Device
Daily hGH treatment characteristics
TransCon hGH prodrug
releases unmodified hGH
First-year height velocity
>10
cm
Reduced adiposity and improved
cardiovascular health
Improved
Safe and well-tolerated
Essentially pain-free
<
1.0
mL
per
injection
Convenient and easy to use

TRANSCON hGH: OVERVIEW OF COMPLETED STUDIES
Dose proportional response in hGH and IGF-I levels
hGH & IGF-I Cmax and exposure comparable between
equivalent weekly doses of TransCon hGH and daily hGH
No treatment-emergent anti-hGH antibody formation
Transient and generally mild injection site reactions
comparable to daily hGH
PK and PD data support once-weekly administration
Phase 1
First in man
Phase 1
High dose
enabling
Phase 2
Adult GHD
12
Q2 2015

PHASE 2 STUDY IN GHD CHILDREN ONGOING
Six-month Phase 2 study in GHD children of TransCon hGH vs. daily hGH
-
Multicenter, randomized, open label, active-controlled, parallel-group study investigating the safety,
tolerability and efficacy in pre-pubertal children with GHD (n=53)
-
Efficacy endpoint: 6-month mean height velocity
-
Enrolled children meet internationally recognized guidelines, similar enrollment criteria expected in
Phase 3 design
-
Doses of 0.14 mg, 0.21 mg and 0.30 mg hGH/kg/week administered once-weekly versus daily hGH
equivalent to 0.21 mg hGH/kg/week
-
Study being conducted across Europe and North Africa
Positive interim update reported December 2014 (n=25)
Fully enrolled: n=53
Top-line study results anticipated mid-2015
13
Q2 2015

Phase 2 pediatric study patient demographics are comparable to U.S. and EU
studies of daily and long-acting growth hormone products
PATIENT DEMOGRAPHICS COMPARABLE TO OTHER hGH STUDIES
Q2 2015
14
VRS-317: Versartis corporate presentation Jan. 2015
Lagova: OPKO presentation ENDO 2014
Norditropin: JCEM 2002 87: 90-98
* Ascendis analysis using weighted average of cohorts 1-4
TransCon hGH
Weekly
VRS-317
Semi-monthly
Lagova*
Weekly
Norditropin
Daily
Key territory
Europe
United States
Europe
United States
Number of subjects
53
64
50
104
Age
7.8
7.8
6.1
7.8
Height SDS
-3.1
-2.5
-3.9
-3.0
GH stimulation test
5.0
5.4
3.9
4.9
IGF-I SDS
-2.2
-1.7
-2.1
-2.8

TRANSCON hGH PRODUCES ROBUST GROWTH COMPARABLE TO DAILY hGH
TransCon hGH compared to
Genotropin
®
control arm
Same weekly dose
Phase 2 pediatric study 6-month interim results
15
Q2 2015

DOSE PROPORTIONAL IGF-I ELEVATION INTO THE NORMAL RANGE
Transient
point
values
of
IGF-I
SDS
>
+2
have
been
observed
in
a
small
number
of
patients
and
only
in
the
high-dose
treatment
arm
Phase 2 pediatric study 6-month interim results
16
Q2 2015

COMPARABLE hGH LEVELS FOR TRANSCON hGH AND DAILY hGH
Maximum hGH blood concentration is comparable between equivalent
weekly
doses of TransCon hGH and daily hGH (0.21 mg/kg/week)
Dose-proportionality observed for TransCon hGH across dose range
Phase 2 pediatric study 6-month interim results
17
Q2 2015

SAFETY COMPARABLE BETWEEN TRANSCON hGH AND DAILY hGH
No Serious Adverse Events (SAEs) related to study drug
Adverse events consistent with hGH therapy observed and not different between
cohorts
Low immunogenicity comparable to daily hGH
Transient injection site reactions comparable to daily hGH
-
>950
TransCon
hGH
injections
administered
in
the
Phase
2
pediatric
study
-
No reports of lipoatrophy or nodule formation
Phase 2 pediatric study 6-month interim results
18
Q2 2015

TRANSCON hGH MAY IMPROVE REAL-WORLD TREATMENT OUTCOMES
Phase 2 interim results demonstrate improved height velocity compared to age-matched historical
controls (KIGS database)
Improved height velocity translates into improved adult height
Sources: Ranke JCEM 2010 KIGS database/CDC/Company analysis
TransCon hGH 6-month HV vs. KIGS 12-month HV
No statistical comparison conducted between the two studies
Genotropin
(KIGS)
Prescribed
0.22
mg/kg/wk
TransCon
hGH
0.21
mg/kg/wk
19
Q2 2015

LONG-ACTING GROWTH HORMONE PROGRAMS
20
Ascendis
Versartis
OPKO
Novo
Height velocity
11.9
-
14.5
cm
As daily hGH
8.7 cm
No active control
12.2-13.6 cm
As daily hGH
No data
Mode of action: Unmodified hGH
comparable to daily hGH
Yes
No
No
No
Growth hormone blood levels
comparable to daily hGH
Yes
No
(Supra-physiological)
No
(Supra-physiological)
No
(Supra-physiological)
Injection volume
<1
mL
for
most
GHD
patients
Yes
No
Yes
Yes
Injection frequency
Weekly
Every two weeks
Weekly
Weekly
Source: Versartis, OPKO, Novo
Q2 2015

SUMMARY TRANSCON GROWTH HORMONE
Efficacy, safety profile and mode-of-action comparable to daily hGH with
once-weekly administration
TransCon hGH has the potential to improve real-world treatment outcomes
One Phase 3 study in GHD children may enable approval for several indications
based on 505(b)(2) strategy
Clinical milestones:
–
Phase 2 pediatric study: Top-line data expected mid-2015
–
Phase 3 pediatric study: Planned initiation of 12-month height velocity trial in mid-2016
Strong IP position with patent life extending until 2035
21
Q2 2015

TransCon Treprostinil: Once-daily administration
22
Q2 2015

ONCE-DAILY TRANSCON TREPROSTINIL
Global sales of prostacyclin therapy for Pulmonary Arterial Hypertension (PAH)
represented
a
~$1.2
billion
orphan
drug
market
in
2013
TransCon Treprostinil is being designed as a long-acting prodrug to be inactive
and continuously release unmodified treprostinil after administration
To maintain same mode-of-action as treprostinil and preserves the benefits of continuous exposure
To address the administrative burden that prevents patient and physician acceptance of currently
approved prostacyclin therapies
Phase 1 proof-of-concept study completed
Demonstrated predictable, extended release of unmodified treprostinil supporting
infusion-like pharmacokinetics
Injection-site tolerability of TransCon Treprostinil did not meet the criteria defined in the
target product profile
New product formulations are currently being explored to improve
injection site
tolerability
23
Q2 2015

Investment Highlights
24
Q2 2015

INVESTMENT HIGHLIGHTS
Q2 2015
25
Internal pipeline focused on developing best-in-class products for orphan markets
TransCon Growth Hormone: Phase 2 pediatric study top-line results expected mid 2015
High-value partnerships in place
Roche Genentech in ophthalmology and Sanofi in diabetes
Broadly applicable and proven TransCon technology platform
Validated with proteins, peptides and small molecules
Strong financial position
Capitalized to complete Phase 3 study of TransCon Growth Hormone