- Agreement between Ascendis Pharma A/S and ST is for the
exclusive distribution and commercialisation of three Ascendis
Pharma endocrinology therapies
- Two of the therapies are already internationally approved,
the third is in development following successful Phase 2
data
- ST's exclusive distribution agreement covers Australia, New
Zealand, Singapore,
Malaysia, Brunei, Thailand and Vietnam
SINGAPORE, Jan. 7, 2024
/PRNewswire/ -- Independent biopharmaceutical
company Specialised Therapeutics Asia Pte Ltd (ST) has added
three new endocrinology therapies to its specialist portfolio,
following an exclusive distribution agreement with Danish company
Ascendis Pharma A/S (NASDAQ: ASND).
Under the terms of the agreement, ST will commercialise Ascendis
Pharma's weekly injectable paediatric human growth hormone
treatment SKYTROFATM (lonapegsomatropin),
hypoparathyroidism treatment YORVIPATHTM
(palopegteriparatide) and investigational achondroplasia therapy
TransCon™ CNP (navepegritide).
The agreement spans ST's key regions of Australia, New
Zealand, Singapore,
Malaysia, Brunei, Thailand, and Vietnam.
Two of the products included in this agreement are already
internationally approved:
- Once-weekly SKYTROFA is a human growth hormone (hGH)
approved in the United States
for the treatment of paediatric patients aged >1 years
weighing >11.5 kg with growth failure due to inadequate
secretion of endogenous growth hormone (GH)1 and
in the European Union for growth failure in children and
adolescents aged from 3 to 18 years due to insufficient endogenous
growth hormone secretion (growth hormone deficiency
[GHD]).2
- YORVIPATH is a first-in-class parathyroid hormone
(PTH) replacement therapy to treat chronic
hypoparathyroidism, a rare and potentially serious condition
where the body produces no or abnormally low levels of
PTH. It is approved in the European Union for
the treatment of adults with chronic
hypoparathyroidism.3
The third product – TransCon CNP – is in development by Ascendis
Pharma for the treatment of achondroplasia (ACH), the most common
genetic form of skeletal dysplasia and resulting disproportionate
short stature, following successful Phase 2 trial
results.4
Australian endocrinologist Dr Veronica
Preda noted that YORVIPATH would be the first specialist
therapeutic option for Australian patients living with
hypoparathyroidism.
"Hypoparathyroidism can seriously impact quality of life and has
potentially life-threatening consequences," Dr Preda said.
"To have an option that is able to treat the underlying cause of
the disease, moving beyond standard oral calcium and active Vitamin
D, is a great step forward."
Announcing the partnership, ST Chief Executive Officer
Carlo Montagner said this agreement
was an important company milestone, signalling ST's expansion into
both endocrinology and paediatric medicine.
Mr Montagner commented: "We are delighted to have been selected
as Ascendis Pharma's exclusive partner for commercialising their
portfolio in Oceania and these South-East
Asia countries and look forward to launching these critical
endocrinology products in our regions as soon as possible.
"All three products are valuable inclusions to our broad
therapeutic pipeline and our international business, as we continue
to leverage our substantial experience commercialising specialist
medicines across multiple regions.
"We look forward to working with endocrinologists across our
territories to make these endocrine therapies available to all
eligible patients who may benefit."
Ascendis Pharma Executive Vice President and Chief Commercial
Officer Camilla Harder Hartvig said
ST had been selected to launch the endocrinology portfolio in these
countries based on its strong track record commercialising
specialist products in multiple regions.
"We are delighted to partner with Specialised Therapeutics to
broaden the reach of our endocrinology rare disease portfolio,
contributing to our shared goal of making a meaningful difference
for patients facing unmet medical needs," she said.
About Specialised Therapeutics
Founded in 2007, Specialised Therapeutics is the region's
largest independent specialty pharmaceutical company, providing new
therapies and technologies to patients in Australia, New
Zealand and across Southeast
Asia. Headquartered in Singapore, ST partners with global
pharmaceutical, biotech and diagnostic companies to bring novel
healthcare opportunities to patients who are impacted by a range of
diseases. ST has built a strong track record of success, navigating
complex regulatory, reimbursement and commercialisation
environments in its diverse regions. The ST mission is to provide
specialty therapies where there is an unmet need. The company's
broad therapeutic portfolio currently includes novel agents in
oncology, haematology, CNS, neurology, endocrinology, ophthalmology
and supportive care, although it is not confined to these areas. ST
is a member of the World Orphan Drug Alliance (WODA).
Additional information: www.stbiopharma.com
About Ascendis Pharma
Ascendis Pharma is applying its innovative TransCon technology
platform to build a leading, fully integrated biopharma company
focused on making a meaningful difference in patients' lives.
Guided by its core values of patients, science and passion, the
company uses its TransCon technologies to create new and
potentially best-in-class therapies. Please visit
https://ascendispharma.com to learn more.
About SKYTROFA (lonapegsomatropin)
SKYTROFA™ (lonapegsomatropin, developed as
TransCon™ hGH) is a prodrug of somatropin, designed to provide
sustained release of unmodified somatropin. The unmodified, unbound
somatropin released from lonapegsomatropin has the same 191 amino
acid sequence and size as endogenous GH.5 TransCon hGH
is approved and marketed as SKYTROFA (lonapegsomatropin-tcgd)
in the United
States1 and as
SKYTROFA (lonapegsomatropin) in the European
Union2 as a once-weekly treatment for children and
adolescents with GHD.
SKYTROFA single-use, prefilled cartridges are manufactured in
nine dosage strengths, allowing for convenient dosing flexibility.
They are designed for use only with the SKYTROFA Auto-Injector
and may be stored at room temperature for up to six months. The
recommended dose of SKYTROFA for treatment-naïve patients and
patients switching from daily somatropin is 0.24 mg/kg body
weight, administered once weekly.1,2 The dose may be
adjusted based on the child's weight and insulin-like growth
factor-1 standard deviation score (IGF-1 SDS).2
TransCon hGH was studied in over 300 children with GHD across
the Phase 3 program, which consisted of the Height
Trial5 (for treatment-naïve patients), the Flight
Trial6 (for treatment-experienced patients), and the
Enlighten Trial7 (a long-term extension trial). Patients
who completed the Height or Flight Trials were able to continue in
Enlighten, with some on lonapegsomatropin treatment for over four
years.
Ascendis Pharma is also conducting the ongoing global Phase 3
Foresight Trial of TransCon hGH in adults with GHD.8
About Paediatric Growth Hormone Deficiency
Paediatric GHD is a serious orphan disease caused when the
pituitary gland does not produce enough GH. Children with GHD are
not only characterised by short stature; they also may experience
metabolic abnormalities, psychosocial challenges, and an overall
poor quality of life. For decades, the standard of care for GHD has
been a daily subcutaneous injection of hGH to improve growth and
overall endocrine health.
About YORVIPATH (palopegteriparatide)
YORVIPATH™ (palopegteriparatide, developed as
TransCon™ PTH) is a once-daily prodrug with sustained release of
active PTH approved by the European Union as a PTH replacement
therapy for the treatment of adults with chronic
hypoparathyroidism. Treatment should be initiated and monitored by
physicians or qualified healthcare professionals experienced in the
diagnosis and management of patients with
hypoparathyroidism.3
TransCon PTH met all primary and key secondary endpoints in the
Phase 3 Pathway Trial, demonstrating a response rate of 78.7%
compared to 4.8% for control (p-value <0.0001) for the primary
composite endpoint, and statistically significant improvements
compared to control on all key secondary endpoints, which included
measures evaluating patient-reported disease symptoms and
impacts.9
About Hypoparathyroidism
Hypoparathyroidism is an endocrine disease caused by
insufficient levels of PTH, the primary regulator of
calcium/phosphate balance in the body, acting directly on bone and
kidneys and indirectly on intestines. Hypoparathyroidism is
considered chronic if it persists >6 months following
surgery. Individuals with hypoparathyroidism may experience a
range of severe and potentially life-threatening short-term and
long-term complications, including neuromuscular irritability,
renal complications, extra-skeletal calcifications, and cognitive
impairment.9
About TransCon CNP (navepegritide)
TransCon™ CNP (navepegritide) is an investigational long-acting
prodrug of C-type natriuretic peptide (CNP), designed
to provide continuous exposure of CNP at safe, therapeutic
levels, via a single, weekly subcutaneous dose, for the treatment
of children with ACH.4
The Phase 2 Accomplish Trial, a randomised, double-blind,
placebo-controlled, dose-escalation trial evaluating the safety and
efficacy of once-weekly TransCon CNP compared to placebo in
prepubertal children with ACH aged 2 to 10 years old, met its
primary objectives, and demonstrated that TransCon CNP at 100
µg/kg/week was superior to placebo for the primary efficacy
endpoint of annualised growth velocity (AGV) at 52
weeks4.
All 57 randomised children completed the blinded portion of
Accomplish and are currently continuing in the open label
extension at the 100 μg/kg/week dose4. Ascendis
Pharma recently confirmed that these 57 clinical trial patients
have all completed one year of treatment with TransCon CNP at 100
µg/kg/week, and announced that TransCon CNP is the first
investigational product to demonstrate improvements in
health-related quality of life and disease impacts in children with
ACH.10
About Achondroplasia
Achondroplasia is the most common genetic form of skeletal
dysplasia and resulting disproportionate short stature, caused by a
genetic mutation in the fibroblast growth factor receptor 3
(FGFR3). This leads to an imbalance between the stimulatory and
inhibitory signaling pathways involved in regulating bone growth.
People living with ACH may experience serious complications and
comorbidities due to inhibited skeletal development. Complications
may include sleep apnoea and respiratory problems, chronic back and
leg pain from lower spine impingement, and sudden infant death from
compression of the brain stem. Chronic ear infections due to
eustachian tube problems can lead to hearing loss and speech delay.
Children with ACH may also experience social and emotional
challenges.
References
- SKYTROFA (lonapegsomatropin-tcgd) US Prescribing
Information.
- SKYTROFA (lonapegsomatropin) EU
Summary of Product Characteristics.
- YORVIPATH (palopegteriparatide) EU
Summary of Product Characteristics.
- Savarirayan, R, Hoemschemeyer DG,
Ljungberg M, et al., Lancet.
2023;65:1–10.
- Thornton PS, Maniatis AK, Aghajanova
E, et al., J Clin Endocrinol Metab.
2021;106(11):3184–3195.
- Maniatis AK, Nadgir U, Saenger
P, et al., Horm Res
Paediatr. 2022;95(3):233–243.
- Maniatis AK, Casella SJ, Nadgir
UM, et al. J Clin Endocrinol Metab.
2022;107(7): e2680–e2689.
- Foresight Trial (NCT05171855),
https://clinicaltrials.gov/study/NCT05171855.
- Khan AA, Rubin MR, Schwarz P, et
al. J Bone Miner Res.
2023;38(1):14–25.
- Ascendis Pharma A/S, Significant Health and Quality of
Life Improvements Achieved in Children with Achondroplasia
Treated for One Year with TransCon™ CNP
(Navepegritide) at 100
µg/kg/week, Press Release, 20 December 2023,
https://investors.ascendispharma.com/news-releases/news-release-details/significant-health-and-quality-life-improvements-achieved.
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SOURCE Specialised Therapeutics