Form 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
October 1, 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): ¨
On October 1, 2015, the poster attached hereto as Exhibit 99.1 regarding the final data from the Phase 2
pediatric trial of TransCon Growth Hormone of Ascendis Pharma A/S (the “Company”) will be made available at the 2015 Annual Meeting of the European Society for Paediatric Endocrinology being held in Hall 8 of the Fira Gran Via Congress
Centre in Barcelona. The poster will be presented by Professor Pierre Chatelain, M.D., former Chairman of the College of Pediatrics at the Université Claude Bernard Lyon 1, Professor Emeritus of Pediatrics, and Coordinating Investigator of
the Phase 2 pediatric study of TransCon Growth Hormone, from 12:45 pm to 2:30 pm Central European Time on October 2, 2015 during a session titled “GH and IGF Treatment”.
The furnishing of the attached poster is not an admission as to the materiality of any information therein. The information contained in the poster 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.
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Ascendis Pharma A/S |
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| Date: October 1, 2015 |
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By: |
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/s/ Michael Wolff Jensen
Michael Wolff Jensen Senior Vice President, General
Counsel |
EXHIBIT INDEX
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| Exhibit No. |
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Description |
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| 99.1 |
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Company Poster. |
EX-99.1
Exhibit 99.1
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| A Phase 2, six-month, randomized, active-controlled, safety and efficacy
study of TransCon hGH compared to daily hGH in children with
Growth Hormone Deficiency (GHD) |
| Pierre Chatelain1, MD; Oleg Malievsky2, MD; Klaudziya Radziuk3, MD; Ganna Senatorova4, MD;
Michael Beckert5, MD |
| 1University Claude Bernard, Lyon, France, 2Bashkir State Medical University, Ufa, Russia, 32nd Children City Clinic,
Minsk, Belarus,
4Kharkiv National Medical University, Kharkiv, Ukraine, 5Ascendis Pharma A/S,
on behalf of the TransCon hGH study group
This study was sponsored by Ascendis Pharma A/S. |
Background
TransCon hGH is a long-acting prodrug of recombinant human Growth Hormone (rhGH) that releases fully active unmodified rhGH into the blood compartment. In
Phase 1 and Phase 2 AGHD studies, TransCon hGH was shown to:
| 1) |
Be safe and well tolerated, |
| 2) |
Be suitable for a once-weekly dosing regimen, |
| 3) |
Provide a pharmacokinetic (PK) hGH and pharmacodynamic (PD) IGF-I response comparable to daily hGH treatment throughout the dosing period. |
This pediatric Phase 2 clinical study was designed to investigate the safety, efficacy, pharmacokinetics and pharmacodynamics of TransCon hGH compared to
daily hGH over a treatment period of six months. Topline data of the full analysis set are reported in this poster.
Figure 1: The TransCon hGH prodrug consists of hGH transiently bound to a polyethylene glycol carrier molecule via a
TransCon linker. The released hGH is unmodified, and designed to maintain the same mode of action and distribution in the body as daily hGH.
Objectives
The objective of this study is to
investigate
| 1) |
Safety and Tolerability, |
| 2) |
Pharmacokinetics and Pharmacodynamics, |
| 3) |
Efficacy of TransCon hGH |
in children with Growth Hormone Deficiency.
Design and Methods
Pre-pubertal, treatment naïve GHD children received s.c. injections of one of three once-weekly TransCon hGH doses (0.14, 0.21 and 0.30 mg rhGH/kg/week)
or daily hGH (Genotropin®; 0.03 mg rhGH/kg/day = 0.21 mg rhGH/kg/week) over a six-month treatment period, in a randomized Phase 2 study. GHD diagnoses were established in accordance with
international consensus guidelines.
Demographics
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| Mean + SD |
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All subjects |
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0.14 mg
rhGH/
kg/week
TransCon
hGH |
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0.21 mg
rhGH/
kg/week
TransCon
hGH |
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0.30 mg
rhGH/
kg/week
TransCon
hGH |
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0.03 mg
rhGH/
kg/day
Genotropin® |
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| # Subjects |
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53 |
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12 |
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14 |
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14 |
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13 |
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| Age (years) Baseline |
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8.0
(2.5 |
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8.2
(2.9 |
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8.4
(2.1 |
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7.5
(2.8 |
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7.7
(2.5 |
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| Height SDS |
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-3.1
(0.9 |
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-3.1
(1.1 |
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-2.8
(0.4 |
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-3.2
(1.0 |
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-3.3
(1.1 |
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| GH Stimulation Test *
[ng/mL] (Screening) |
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5.0
(2.8 |
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5.1
(3.2 |
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5.2
(2.6 |
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4.4
(2.8 |
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5.2
(3.1 |
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| IGF-I SDS |
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-2.2
(0.8 |
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-2.0
(0.7 |
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-2.0
(0.8 |
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-2.2
(0.7 |
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-2.5
(0.9 |
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* The higher peak of the two performed GH stimulation tests was used for calculation of the mean.
Results - Growth
Annualized height velocities among the three once-weekly TransCon hGH doses ranged from 11.9 cm for the 0.14 mg rhGH/kg/week dose to 13.9 cm for the 0.30 mg
rhGH/kg/week dose, which were comparable to 11.6 cm for the active comparator, daily injections of Genotropin® at a cumulated dose of 0.21 mg rhGH/kg/week.
Figure 2: Annualized Height Velocity (Mean + SD) of full dataset of 53 patients after 6 months of treatment.
Results - PK/PD
A full PK/PD profile was established in week 13. Maximum hGH blood concentration is comparable between equivalent weekly doses of TransCon Growth Hormone and
daily hGH (Figure 3). IGF-I levels (SDS) increased dose-proportionally and was normalized for all dose groups (Figure 4) following dosing of the three TransCon Growth Hormone dose levels.
Figure 3: hGH levels for TransCon hGH (0.21 mg rhGH/kg/week) and daily hGH (0.21 mg rhGH/kg/week).
Figure 4: IGF-I SDS levels increased dose-proportionally and was normalized for all dosing groups following dosing of the
three TransCon hGH dose levels.
Safety
No
safety concerns were observed. Injection site reactions have generally been mild and similar to what is expected with daily hGH injections, with no nodule formation or lipoatrophy noted.
Conclusion
The results of this Phase 2 study in
pediatric patients with GHD confirms the safety, tolerability and the suitability of TransCon hGH for once-weekly dosing. An equivalent dose-level to daily hGH demonstrated numerically higher growth rates compared to daily hGH treatment. No
drug-related SAEs occurred, no lipoatrophy or nodule formation was seen. IGF-I changes suggest a dose response and levels are in the expected range.
Participating Investigators:
M. Omar Abdou, MD; M. Aryayev, MD;
G. Balazs, MD; P. Barat, MD; T. Battelino, MD; I. Ben-Skowronek, MD; E. V. Bolshova, MD; P. Chatelain, MD; S. Darcan, MD; H. Hassan Elsedfy, MD; E. Erhardt, MD; C. Georgescu, MD; M. Mamdouh Hassan, MD; V. Iotova, MD; S. Koloušková, MD; O.
Malievskiy, MD; O. Marginean, MD; A. Mazur, MD; E. Mikhailova, MD; V. Mogos, MD; K. Mohnike, MD; A. Muzsnai, MD; I. Pascanu, MD; V. Peretyatko, MD; V. Peterkova, MD; R. Pfäffle, MD; G. Senatorova, MD; A. El Sharkawy, MD; Z. Siklar, MD; J.
Skorodok, MD; J. Škvor, MD; M. Szalecki, MD; K. Radziuk, MD; Z. Vajda, MD; E. Vlachopapadopolou, MD; J. Weill, MD; N. Zelinska, MD; K. Ziora, MD
