Specialised Therapeutics Expands Partnership with Incyte to Include Two Additional Therapies for Hard-to-Treat Conditions

Singapore 11 June 2025: Independent biopharmaceutical company Specialised Therapeutics (ST) is pleased to announce the expansion of its existing supply and distribution agreement with Incyte Biosciences International Sàrl, the Swiss-based affiliate of Incyte (NASDAQ:INCY), to launch and distribute two additional medicines from its oncology portfolio in Australia, New Zealand and Singapore, with an option to add further countries in the Asia-Pacific region. The expanded agreement will see new therapies axatilimab (registered as Niktimvo^® in the United States) and retifanlimab (registered as Zynyz^® in the U.S. and European Union) added to the current partnered portfolio of Minjuvi^® (tafasitamab) and Pemazyre^® (pemigatinib).

Under the terms of the expanded agreement, Incyte will be responsible for the development, manufacture and supply of both axatilimab and retifanlimab to the region, while ST will have responsibility for regulatory, distribution and local marketing and medical affairs related activities.

ST Chief Executive Officer, Mr Carlo Montagner, welcomed the expansion of the partnership agreement with Incyte, a leading global biopharmaceutical company, and the opportunity to bring these important medicines to eligible patients in the local region.

“Specialised Therapeutics has partnered with Incyte since 2021 and we are delighted to be extending this successful partnership,” he said. “As part of our initial agreement, our teams have worked collaboratively to commercialise Minjuvi, which has been approved for use in Australia, and Pemazyre, which has been approved in Australia and Singapore, and we look forward to expanding the portfolio to help more patients with rare and hard-to-treat conditions.”

“The addition of axatilimab and retifanlimab to the partnership agreement with Incyte recognises our strong track record of working with local stakeholders to bring innovative medicines to patients where unmet medical needs persist, despite the availability of existing treatments,” said Mr Montagner. “Our focus will be on seeking regulatory and reimbursement approval for all four medicines in the region, to ensure equitable access to these treatments are available for patients as soon as practicable.”

ST anticipates submitting axatilimab and retifanlimab for local regulatory and reimbursement approval in 2025.

Incyte CEO, Mr Hervé Hoppenot said the expanded partnership agreement reflected the synergies between the two companies, with a shared goal of improving outcomes for patients with unmet medical needs.

“We are pleased to extend our partnership with ST to include axatilimab and retifanlimab,” he said. “ST has already demonstrated its ability to navigate complex regional regulatory pathways for Minjuvi and Pemazyre, and we look forward to continuing our work together to make these new therapies available for the oncology community in the Asia-Pacific region.”

Axatilimab is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody. It was approved by the US Food and Drug Administration (FDA) in August 2024 as a treatment for adults and children with chronic graft-versus-host disease (GVHD) who have received at least two prior treatments (systemic therapy) and require additional treatment.¹ Chronic GVHD usually occurs 3 months after a transplant – typically haematopoietic stem cell or bone marrow transplantation, but occasionally also solid organ transplants – where the donor cells (“graft”) attack the graft recipient’s cells (“host”).² Chronic GVHD can affect all organs, but commonly impacts the skin, mouth, eyes, lungs, stomach, bowel, and liver.^2,3 The Phase II clinical trial for axatilimab involved 79 patients from 13 countries, including Australia and Singapore.⁴

Retifanlimab is an intravenous immune checkpoint (PD-1) inhibitor that has been approved in the US in combination with carboplatin and paclitaxel (platinum-based chemotherapy) for the first-line treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC) and as a single agent for the treatment of adult patients with locally recurrent or metastatic SCAC with disease progression or intolerance to platinum-based chemotherapy.⁵ Retifanlimab is also approved in the US⁶ and Europe⁷for the treatment of adults with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC). MCC is a rare and aggressive type of skin cancer, that is often difficult to diagnose due to the lack of specific features to distinguish it from other common skin cancers.⁸ Australia has the highest incidence of MCC in the world.⁹ The pivotal Phase II clinical trial of retifanlimab in MCC enrolled 101 chemotherapy-naïve patients from 12 countries, including Australia.¹⁰ In addition to MCC, retifanlimab, in combination with chemotherapy, is currently being investigated as a potential therapeutic option in other hard-to-treat cancers, including metastatic non-small cell lung cancer (NSCLC).

Ends.

Further enquiries:

Tania Jayesuria, Director, TJ Health Comms | +61 404 094 744 | tania.jayesuria@outlook.com

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 can be found at www.stbiopharma.com

About axatilimab (Niktimvo^®)

Axatilimab-csfr (registered as Niktimvo^® in the United States) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the US for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and paediatric patients weighing at least 40 kg (88.2 lbs).

In 2016, Syndax licensed exclusive worldwide rights to develop and commercialise axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialisation license agreement for axatilimab in chronic GVHD and any future indications.

Axatilimab is being studied in frontline combination trials in chronic GVHD – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774) are underway. Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

Niktimvo is a trademark of Incyte.
All other trademarks are the property of their respective owners.

About retifanlimab (Zynyz^®)

Retifanlimab-dlwr (registered as Zynyz^®in the United States and European Union) is a humanized monoclonal antibody targeting programmed death receptor-1 (PD-1), indicated in combination with carboplatin and paclitaxel (platinum-based chemotherapy) for the first-line treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC) and as a single agent for the treatment of adult patients with locally recurrent or metastatic SCAC with disease progression or intolerance to platinum-based chemotherapy in the U.S.

Zynyz is also indicated for the treatment of adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC) in the US. This indication is approved under accelerated approval based on tumour response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Zynyz is marketed by Incyte in the US. In 2017, Incyte entered into an exclusive collaboration and license agreement with MacroGenics, Inc. for global rights to retifanlimab.

Zynyz is a registered trademark of Incyte.

References:

US FDA. FDA approves axatilimab-csfr for chronic graft-versus-host disease, 14 Aug 2024. Available at: https://www.fda.gov/‌ drugs/resources-information-approved-drugs/fda-approves-axatilimab-csfr-chronic-graft-versus-host-disease [Accessed Mar 2025].
The Australasian College of Dermatologists. A-Z of Skin: Graft Versus Host Disease, Nov 2020. Available at: https://www.dermcoll.edu.au/wp-content/uploads/2022/03/Graft-Versus-Host-Disease-A-Z-of-Skin.pdf [Accessed Mar 2025].
Leukaemia Foundation Australia. Factsheet: Graft versus host disease, 24 May 2024. Available at: https://www.leukaemia.org.au/wp-content/uploads/2022/11/Factsheet_Graft-Versus-Host-Disease_Leukaemia-Foundation.pdf [Accessed Mar 2025].
gov. A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD) (AGAVE-201); NCT04710576. Available at: https://clinicaltrials.gov/study/NCT04710576 [Accessed Mar 2025].
FDA approves retifanlimab-dlwr with carboplatin and paclitaxel and as a single agent for squamous cell carcinoma of the anal canal. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-retifanlimab-dlwr-carboplatin-and-paclitaxel-and-single-agent-squamous-cell-carcinoma. [Accessed May 2025]
US FDA. FDA grants accelerated approval to retifanlimab-dlwr for metastatic or recurrent locally advanced Merkel cell carcinoma, 22 Mar 2023. Available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-retifanlimab-dlwr-metastatic-or-recurrent-locally-advanced-merkel [Accessed Mar 2025].
European Medicines Agency (EMA). Zynyz (retifanlimab): Marketing Authorisation in the European Union (EU), 19 Apr 2024. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/zynyz [Accessed Mar 2025].
NeuroEndocrine Cancer Australia. Merkel Cell Carcinoma: The Facts, 19 Nov 2023. Available at: https://neuroendocrine.‌ org.au/what-are-nets/merkel-cell-carcinoma/ [Accessed Mar 2025].
The Australasian College of Dermatologists. A-Z of Skin: Merkel cell carcinoma, Oct 2024. Available at: https://www.dermcoll.edu.au/‌ atoz/merkel-cell-carcinoma/ [Accessed Mar 2025].
Grignani G, et al. Updated Results From POD1UM-201: A Phase 2 Study of Retifanlimab in Patients With Advanced or Metastatic Merkel Cell Carcinoma. Presented at the European Society for Medical Oncology Congress 2023, Madrid, Spain; 20-24 October 2023.

New Therapy for Children with Growth Hormone Deficiency Approved in Australia

SKYTROFA^® ® (lonapegsomatropin) is now registered by the Therapeutic Goods Administration (TGA) for the treatment of paediatric growth hormone deficiency.¹

Singapore 30 May 2025:

Independent biopharmaceutical company Specialised Therapeutics (ST) is pleased to announce the Australian registration of SKYTROFA® (lonapegsomatropin) as a new once-weekly injectable therapy for paediatric growth hormone deficiency (GHD).2 The Therapeutic Goods Administration (TGA) has approved SKYTROFA as a treatment for “growth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion”.^1,2

GHD is a rare disease affecting around 2-3/10,000 Australians, with approximately 2,000 children thought to be living with the condition.³ It occurs when the pituitary gland, located at the base of the brain, does not produce an adequate level of growth hormone, which is essential for promoting healthy growth in children.³ In children, GHD is typically characterised by slow height growth, resulting in short stature, and may also include delayed puberty, impaired hair growth and headaches.³ The goal of treatment for GHD is to restore normal levels of growth hormone in the body, reduce physical symptoms, enhance metabolic health and improve quality of life.³

ST Chief Executive Officer, Mr Carlo Montagner, said the TGA approval of SKYTROFA demonstrated the company’s ongoing mission to support patients with rare diseases in Australia and the Asia-Pacific region.

“We are delighted to have secured TGA registration of SKYTROFA for eligible Australian children with growth hormone deficiency. Beyond short stature, children and adolescents with the condition may experience considerable physical and psychosocial impacts on their daily life, such as poor concentration, decreased strength or muscle development, fatigue, and reduced quality of life.⁴

“This announcement also represents a significant milestone for Specialised Therapeutics, marking the second endocrinology therapy we have successfully registered in Australia, following the approval of YORVIPATH^® (palopegteriparatide) earlier this year,” said Mr Montagner.

SKYTROFA is being registered in Australia by ST, under an exclusive distribution agreement with biopharmaceutical company Ascendis Pharma A/S that covers Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam. The Australian registration of SKYTROFA follows approvals issued to Ascendis Pharma by the United States Food and Drug Administration (US FDA)5 in August 2021 and the European Medicines Agency (EMA)⁶ in January 2022.

Developed using Ascendis Pharma’s proprietary TransConTM platform, SKYTROFA is delivered as a subcutaneous injection, available in various strengths.² The starting dose depends on the patient’s body weight and is then adjusted individually by the treating endocrinologist based on the patient’s response to treatment.²

Ascendis Pharma’s innovative TransCon™ technology platform is designed to create new therapies with the potential to optimise therapeutic effect, including improving treatment efficacy, safety and dosing frequency.⁷ TransCon™ molecules have three components: an unmodified parent drug, an inert TransCon™ carrier that protects it, and a TransCon™ linker that temporarily binds the two.⁷

SKYTROFA is a prodrug of somatropin administered once weekly, designed to provide sustained release of active, unmodified somatropin.⁶ This allows the medicine to slowly release unmodified growth hormone into the body over the course of one week, removing the need for daily injections.

SKYTROFA consists of three components: the parent drug somatropin, an inert methyloxypolyethlene glycol carrier (mPEG), and a proprietary TransCon™ linker that transiently binds the other two elements.^8,9 While bound, the carrier inactivates the somatropin and shields it from renal excretion and receptor-mediated clearance.¹⁰ Following injection, autocleavage of the linker occurs under physiologic conditions, and SKYTROFA releases fully active, unmodified somatropin in a predictable manner.¹⁰

The TGA registration of SKYTROFA was based on the results of Ascendis Pharma’s three pivotal Phase 3 clinical trials, heiGHt, fliGHt and enliGHten, which collectively treated more than 300 paediatric patients diagnosed with GHD, including from Australia.^2,8,11,12

SKYTROFA was generally well-tolerated across all three clinical trials. The most commonly reported adverse events include viral infections, fever, cough, nausea and vomiting, haemorrhage, diarrhoea, abdominal pain, arthralgia, arthritis, and increased blood phosphate levels.

PBS Information: SKYTROFA is not listed on the Pharmaceutical Benefits Scheme (PBS).

Ends.

Further enquiries:

Tania Jayesuria, Director, TJ Health Comms | +61 404 094 744 | tania.jayesuria@outlook.com

About Specialised Therapeutics

Additional information can be found at www.stbiopharma.com

About Ascendis Pharma

Ascendis Pharma is a global biopharmaceutical company focused on applying its innovative TransCon™ technology platform to make a meaningful difference for patients. Guided by core values of Patients, Science, and Passion, and following its algorithm for product innovation, Ascendis Pharma applies TransCon™ to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit: ascendispharma.com to learn more.

About Paediatric Growth Hormone Deficiency

Paediatric growth hormone deficiency (GHD) is a serious orphan disease caused when the pituitary gland does not produce enough growth hormone. Physiological levels of growth hormone are required for overall endocrine health and development of healthy bone, muscle, and adipose tissue. Children with GHD are characterised by short stature and may also 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 human growth hormone (hGH) to improve growth and overall endocrine health.

About SKYTROFA (lonapegsomatropin)

Once-weekly SKYTROFA 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 growth hormone.⁸

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 children with growth hormone deficiency and those switching from daily somatropin is 0.24 mg/kg body weight, administered once weekly.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

SKYTROFA was studied in over 300 children with GHD across the Phase 3 program, which consisted of the HeiGHt Trial⁸ (for treatment-naïve patients), the FliGHt Trial¹¹ (for treatment-experienced patients), and the EnliGHten Trial¹² (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 open-label portion of the global Phase 3 ForesiGHt Trial of SKYTROFA in adults with GHD.

Ascendis^®, TransCon™ and SKYTROFA^® are trademarks owned by the Ascendis Pharma group (NASDAQ: ASND).

About TransCon™ Technologies

TransCon refers to “transient conjugation”. Ascendis Pharma’s innovative technology platform to create new therapies designed to potentially optimise therapeutic effect, including improving efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert TransCon carrier that protects it, and a TransCon linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g. pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied to a broad variety of therapeutics, such as antibodies, antibody fragments, proteins, peptides, or small molecules, and can be designed for systemic or local administration.

References:

Therapeutic Goods Australian Register of Therapeutic Goods (ARTG). Available at: https://www.tga.gov.au/resources/artg?keywords=skytrofa. [Accessed May 2025].
SKYTROFA (lonapegsomatropin) Product Information,
Australian Pituitary Factsheet: A Guide To Growth Hormone Deficiency, 2023. Available at: https://pituitary.asn.au/wp- content/uploads/2023/09/APF-Growth-Hormone-Deficiency-Fact-Sheet-2023_Digital-Final.pdf [Accessed May 2025].
Brod M et Qual Life Res. 2017 Jul;26(7):1673-1686.
US Food and Drug Administration (FDA). Drug Approval Package: SKYTROFA. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/761177Orig1s000TOC.cfm [Accessed May 2025].
European Medicines Agency (EMA). Skytrofa (previously Lonapegsomatropin Ascendis Pharma), January. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/skytrofa [Accessed May 2025].
Ascendis Pharma A/S. TransCon: A powerful technology platform central to our innovative Available at: https://ascendispharma.com/technology/ [Accessed May 2025].
Thornton PS, et J Clin Endocrinol Metab. 2021 Oct;106(11):3184-3195.
SKYTROFA^® [package insert]. Palo Alto, CA: Ascendis Pharma,
Sprogøe K, et Endocr Connect. 2017;6(8):R171-r181.
Maniatis AK, et Horm Res Paediatr. 2022 Mar;95(3):233-243.
Maniatis AK, et J Clin Endocrinol Metab. 2022 Jun;107(7):e2680-e2689.

First and Only Treatment for Chronic Hypoparathyroidism Approved in Australia

YORVIPATH^® (palopegteriparatide) is now registered by the Therapeutic Goods Administration (TGA) for the treatment of chronic hypoparathyroidism in adults¹
First-in-class novel PTH replacement therapy
Australia becomes the first country to obtain Marketing Authorisation for YORVIPATH since FDA approval

Singapore 21 February 2025:

Independent biopharmaceutical company Specialised Therapeutics (ST) welcomes the registration of YORVIPATH^® (palopegteriparatide) by the Therapeutic Goods Administration (TGA), “for the treatment of chronic hypoparathyroidism in adults”.^1,2 YORVIPATH was granted an Orphan Drug Designation and assessed through the TGA’s Priority Review pathway.^3,4 It is the first and only medicine to be listed on the Australian Register of Therapeutic Goods (ARTG) for the treatment of chronic hypoparathyroidism.

Hypoparathyroidism is a rare, complex endocrine disease, affecting an estimated 6.4-37 per 100,000 people globally.^5,6 It is an endocrine disorder in which the production of parathyroid hormone (PTH) by the parathyroid glands is abnormally low or absent, causing low levels of calcium (hypocalcaemia) and high levels of phosphorous (hyperphosphataemia) in the blood.^7,8 This impacts proper functioning of nerves and muscles, leading to weakness, muscle spasms or cramps, headaches, hair loss, numbness or tingling, and memory problems.^8,9Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extraskeletal calcifications, and cognitive impairment.¹⁰

Leading Australian Endocrinologist and Head of the School of Clinical Sciences at Monash Health, Professor Peter Ebeling AO, applauded the TGA approval of YORVIPATH in addressing an unmet medical need for Australian adults diagnosed with chronic hypoparathyroidism.

“Until now, the only treatment option for adults with chronic hypoparathyroidism has been conventional therapy with calcium and active vitamin D supplementation to keep blood calcium levels normal,” said Professor Ebeling. “While these treatments help to manage the symptoms of hypoparathyroidism, they do not address the underlying deficiency of PTH and contribute to the significant pill burden for patients.”

“The lack of effective therapeutic options has been an urgent medical need in this area, and the local approval of YORVIPATH represents an important advance in the treatment of chronic hypoparathyroidism in Australia,” said Professor Ebeling.

YORVIPATH is a first-in-class PTH replacement therapy. A prodrug of parathyroid hormone (PTH [1-34]), YORVIPATH is administered subcutaneously once daily, with sustained release of active PTH designed to provide PTH levels in the physiological range for 24 hours/day.¹¹

YORVIPATH’s approval in Australia was supported by the results of Ascendis Pharma’s Phase 3 PaTHway trial, a 26-week randomised, double-blind, placebo-controlled study with a 156-week open-label extension, published in the Journal of Bone and Mineral Research.¹² Involving 84 adults with chronic hypoparathyroidism, the clinical trial demonstrated the efficacy, safety and tolerability of YORVIPATH as a once-daily PTH replacement therapy.

Data from the pivotal trial showed 93% (57/61) of YORVIPATH-treated patients achieved independence from both active vitamin D and therapeutic doses of elemental calcium, which was associated with a clinically meaningful reduction in their daily pill burden.¹²

The safety analysis demonstrated YORVIPATH was generally well-tolerated, with the most frequently reported adverse reactions being injection site reactions (21.6%), headache (18.7%), and paraesthesia (13.7%).²

In addition to the significant short-term and long-term medical complications of chronic hypoparathyroidism, individuals diagnosed with the condition also experience reduced health-related quality of life, including the negative impact of symptoms on their daily life and work productivity.¹²

Chief Executive Officer of the Australian Thyroid Foundation (ATF), Beverley Garside, acknowledged the significance of the TGA approval in offering adults living with chronic hypoparathyroidism the opportunity to improve their health and wellbeing. However, she reinforced that this is not the time for complacency, as it is just the first step towards securing subsidised access to an important new treatment option for this community.

“As the leading national voice for good thyroid health in Australia, the ATF recognises the importance of the TGA registration of YORVIPATH as a new treatment option for the hypoparathyroid community. While this is positive news, it is only the first step,” said Ms Garside. “For adults diagnosed with chronic hypoparathyroidism to truly benefit from this new treatment, it is essential they have the option of subsidised and affordable access to YORVIPATH through the Pharmaceutical Benefits Scheme as quickly as possible.”

YORVIPATH is being made available in Australia by Specialised Therapeutics (ST), under an exclusive distribution agreement with global biopharmaceutical company Ascendis Pharma A/S that covers Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam. The Australian registration of YORVIPATH follows approvals issued to Ascendis Pharma by the United States Food and Drug Administration (US FDA)¹³ in August 2024 and the European Medicines Agency (EMA)¹⁴ in November 2023.

“The Australian approval of YORVIPATH reflects our values and dedication to applying science to help address the significant unmet medical needs expressed by the hypoparathyroidism community,” said Roy Khoury, Vice President, Head of International Markets at Ascendis Pharma. “We are pleased to partner with Specialised Therapeutics to broaden access to this innovative therapy, based on our TransCon technology, which offers a new approach to the treatment of this often-debilitating rare disease.”

ST Chief Executive Officer, Mr Carlo Montagner, said the approval of YORVIPATH was a meaningful milestone, but the company would not stop until it achieved reimbursement for eligible Australians with hypoparathyroidism through the Pharmaceutical Benefits Scheme (PBS).

“We are proud to have brought the first and only TGA-registered treatment for chronic hypoparathyroidism to Australians living with the condition so rapidly following the recent FDA and EMA approvals,” said Mr Montagner. “Chronic hypoparathyroidism has considerable and life-long wide-ranging impacts on these individuals and we are committed to working with the local hypoparathyroidism community and the Australian Government to ensure YORVIPATH is accessible to eligible patients through the PBS at the earliest opportunity.”

YORVIPATH will be reviewed by the Pharmaceutical Benefits Advisory Committee (PBAC) at its first meeting of the year, to be held from 12-14 March 2025. YORVIPATH will be made available for prescribing in Australia once it has been listed on the PBS.

PBS Information: YORVIPATH is not listed on the Pharmaceutical Benefits Scheme (PBS).

Ends.

Further enquiries:

Tania Jayesuria, Director, TJ Health Comms | +61 404 094 744 | tania.jayesuria@outlook.com

About Specialised Therapeutics

Additional information can be found at 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. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit: ascendispharma.com to learn more.

About Hypoparathyroidism¹⁰

Hypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidneys and indirectly on the intestines. 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. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%), while other etiologies include autoimmune and idiopathic causes.

About PaTHway¹²

The PaTHway trial was a double-blind, placebo-controlled, 26-week, Phase 3 study that assessed the efficacy and safety of PTH replacement therapy with palopegteriparatide for individuals with hypoparathyroidism. Participants (n=84) were randomised 3:1 to once-daily palopegteriparatide (initially 18 μg/d) or placebo, both co-administered with conventional therapy.

The study drug and conventional therapy were titrated according to a dosing algorithm guided by serum calcium. The composite primary efficacy endpoint was the proportion of participants at week 26 who achieved normal albumin-adjusted serum calcium levels (8.3–10.6 mg/dL), independence from conventional therapy (requiring no active vitamin D and ≤600 mg/d of calcium), and no increase in study drug over 4 weeks before week 26. At week 26, 79% (48/61) of participants treated with palopegteriparatide versus 5% (1/21) with placebo met the composite primary efficacy endpoint (p < 0.0001). Additionally, 93% (57/61) of participants treated with palopegteriparatide achieved independence from conventional therapy. Palopegteriparatide treatment normalised mean 24-hour urine calcium. Overall, 82% (50/61) treated with palopegteriparatide and 100% (21/21) with placebo experienced adverse events; most were mild (46%) or moderate (46%). No study drug-related withdrawals occurred.

About YORVIPATH^® (palopegteriparatide)

YORVIPATH^® (palopegteriparatide, developed as TransCon™ PTH) is a once-daily prodrug providing sustained release of active PTH. It was approved by the European Union as a parathyroid hormone (PTH) replacement therapy for the treatment of adults with chronic hypoparathyroidism¹⁵ and the United States for the treatment of hypoparathyroidism in adults^13. Treatment should be initiated and monitored by physicians or qualified healthcare professionals experienced in the diagnosis and management of patients with hypoparathyroidism.

Yorvipath^® is a registered trademark of Ascendis Pharma A/S (NASDAQ: ASND).

References:

Therapeutic Goods Administration. Australian Register of Therapeutic Goods (ARTG). Available at: https://www.tga.gov.au/australian-register-therapeutic-goods. [Accessed Dec 2024].
YORVIPATH (palopegteriparatide) AU Product Information.
Therapeutic Goods Administration. Prescription medicine determinations and designations: Notice for palopegteriparatide (Specialised Therapeutics Pharma Pty Ltd) – Orphan drug designation. Available at: https://www.tga.gov.au/resources/designations-determinations/notice-palopegteriparatide-specialised-therapeutics-pharma-pty-ltd. [Accessed Dec 2024].
Therapeutic Goods Administration. Prescription medicine determinations and designations: Notice for palopegteriparatide (Specialised Therapeutics Pharma Pty Ltd) – Priority review. Available at: https://www.tga.gov.au/resources/designations-determinations/notice-palopegteriparatide-specialised-therapeutics-pharma-pty-ltd-0. [Accessed Dec 2024].
Siggelkow H, Pacaud D, et al. Clin Endocrinol (Oxf). 2020;92(2).
Bjornsdottir S, Sikjaer T, et al. J Bone Miner Res. 2022;37(12).
Australian Thyroid Foundation. Thyroid Facts: Parathyroid Glands – Hypoparathyroidism. Available at: https://thyroidfoundation.org.au/Parathyroid-Glands [Accessed Dec 2024].
National Organization for Rare Disorders. Disease Overview: Hypoparathyroidism – Symptoms, Causes, Treatment, 14 August 2024. Available at: https://rarediseases.org/rare-diseases/hypoparathyroidism/ [Accessed Dec 2024].
Endocrine Society. Endocrine Library: Patient Resources; Hypoparathyroidism, 24 January 2022. Available at: https://www.endocrine.org/patient-engagement/endocrine-library/hypoparathyroidism [Accessed Dec 2024].
Bilezikian JP. J Clin Endocrinol Metab. 2020;105(6):1722-1736. doi:10.1210/clinem/dgaa113.
YORVIPATH (palopegteriparatide) US Prescribing Information.
Khan AA, Bilezikian JP, Cusano NE, et al. J Bone Miner Res. 2023;38(1).
US Food and Drug Administration. FDA approves new drug for hypoparathyroidism, a rare disorder, 9 August 2024. Available at: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-drug-hypoparathyroidism-rare-disorder [Accessed Dec 2024].
European Medicines Agency. Yorvipath: Palopegteriparatide, 17 November 2023. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/yorvipath [Accessed Dec 2024].
YORVIPATH (palopegteriparatide).EU Summary of Product Characteristics.

Specialised Therapeutics Signs Exclusive Agreement with Ascendis Pharma A/S for Distribution and Commercialisation of Three Endocrinology Therapies in Australia and Select South-East Asia Countries

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 8 January 2024: 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 SKYTROFA^TM (lonapegsomatropin), hypoparathyroidism treatment YORVIPATH^TM (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.

Ends.

Further enquiries:

ST Senior Manager Communications and Corporate Affairs, Emma Power

+61 419 419 525 or email epower@stbiopharma.com

About Specialised Therapeutics

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.

Reference

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.

Specialised Therapeutics Signs Exclusive License Agreement with CanariaBio for New Ovarian Cancer Therapy

First ovarian cancer therapy for ST oncology portfolio
Phase 2 study demonstrated oregovomab in combination with chemotherapy improved progression free survival by ~30 months compared to chemotherapy alone¹
Phase 3 results expected in 2025
Exclusive license for AU, NZ, Singapore, Malaysia, Brunei, Thailand and Vietnam

Singapore and Seoul, South Korea, 13 October 2023: Independent biopharmaceutical company Specialised Therapeutics Asia Pte Ltd (ST) has signed a license deal with Korea-based CanariaBio Inc., acquiring the exclusive license to a new monoclonal antibody therapy for patients with ovarian cancer in Australia, New Zealand and in select Southeast Asian countries.

The therapy, known as oregovomab, is currently in a pivotal phase III international clinical trial known as the FLORA-5 study.² This investigation is examining oregovomab in combination with chemotherapy agents carboplatin and paclitaxel for patients with advanced ovarian cancer.

Under the terms of the arrangement, ST will be responsible for all commercial, medical, regulatory and distribution activities for oregovomab in its key territories of Australia, New Zealand, Singapore, Thailand, Vietnam, Brunei and Malaysia. CanariaBio will be responsible for the manufacture and supply of oregovomab to ST.

Announcing the partnership, ST Chief Executive Officer Carlo Montagner said he was pleased CanariaBio had selected ST as a partner for this highly promising therapy.

“ST has a portfolio of anti-cancer therapies targeting multiple solid tumours with the exception of ovarian cancer, and now oregovomab becomes our first ovarian cancer agent,” Mr Montagner said.

“Despite great advances in recent years, there remains a high unmet need in all our regions to treat this patient population. We look forward to working closely with our new partners at CanariaBio and pending the results of the pivotal Phase III registration study, making oregovomab available to eligible patients.”

CanariaBio Chairman and CEO Michael Na said the company had selected ST for its regional expertise and strong track record commercialising oncology products. Carlo Montagner (Oct 11, 2023 12:11 GMT+11)

“Formalising this agreement is a pivotal moment for our program. This collaboration is more than just a deal – it’s a shared commitment as we develop novel therapies to address unmet medical needs. At CanariaBio, we’ve always believed in the transformative power of partnerships, and teaming up with ST reinforces this belief.” Oregovomab works by targeting and binding specifically to a surface protein known as CA-125 found on the surface of ovarian cancer cells, then activating the patient’s own immune system to respond.³

In the Phase 2 study, the addition of oregovomab to chemotherapy yielded a median progression-free survival of 41.8 months compared with 12.2 months with standard chemotherapy alone (HR, 0.46, P=0.0027). The overall survival hazard ratio was 0.35.1 The Phase 3 FLORA-5 study is fully enrolled and ongoing. Final results are expected in 2025.

Ends.

Further enquiries:

ST Senior Manager Communications and Corporate Affairs Emma Power

+61 419 419 525 or email epower@stbiopharma.com

CanariaBio Communications Manager Jacquelyn Choi

+82 6925 2177 or via email jacquelyn@canariabio.com

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, ophthalmology and supportive care, although it is not confined to these areas. Additional information: www.stbiopharma.com

About CanariaBio Inc. CanariaBio Inc. is a clinical-stage biopharmaceutical company dedicated to the development and commercialization of innovative cancer biotherapeutics. CanariaBio’s technology platform includes a portfolio of tumor antigen-specific monoclonal antibodies targeting CA-125, MUC1, PSA, and HER2/neu.

About Oregovomab

Oregovomab is a murine monoclonal antibody directed to the tumor-associated antigen CA-125 that stimulates a host cytotoxic immune response against tumor cells expressing CA-125, a biomarker commonly found in ovarian cancer (OC). In a randomized Phase 2 clinical trial, oregovomab demonstrated a significant improvement in progression-free and overall survival in advanced OC treatment when administered simultaneously with first-line chemotherapy. This promising schedule is currently being investigated in a Phase 3 trial.

About FLORA-5 Phase 3 Study

The Phase 3 clinical trial called FLORA-5/GOG-3035, is a double-blind, placebo-controlled, multicentre clinical study comparing the safety and efficacy of oregovomab versus placebo when administered in combination with specific cycles of a standard six-cycle chemotherapy regimen (paclitaxel and carboplatin) for the treatment of newly diagnosed patients with advanced epithelial ovarian, fallopian tube or peritoneal carcinoma, in conjunction with optimal debulking surgical resection. The primary and secondary endpoints, for both the adjuvant and neoadjuvant cohorts of this trial, are progression free survival and overall survival, respectively. The FLORA-5 trial is being conducted in collaboration with the Gynecologic Oncology Group Foundation in the US and IQVIA (a clinical research organization). Greater China area clinical trials are conducted in collaboration with OncoVent, a Shenzhen Hepalink Pharmaceuticals Group Company in China, which is also the commercialization license holder of oregovomab for China. Information on the clinical trial can be found on www.clinicaltrials.gov

References:

Brewer M, et al. Gynecol Oncol. 2020.156(3):523-529
ClinicalTrials.gov NCT04498117
www.FLORA-5.com. Last accessed October 2023.

World Orphan Drug Alliance Welcomes Specialised Therapeutics

SEA’s largest independent specialty pharma company Specialised Therapeutics (ST) has joined global pharma consortium committed to collaborating to provide new specialist medicines for rare diseases

ST to represent WODA in Australia, New Zealand and across Southeast Asia (ANZSEA)

World Orphan Drug Alliance (WODA) now spans 152 countries.

WODA offers a ‘one-stop’ solution for biotech companies seeking to commercialise products across global markets.

Dubai, Moscow, Ljubljana, Sao Paulo, Zurich, Shanghai, Singapore, September 8, 2023 – Independent biopharmaceutical company Specialised Therapeutics (ST) has joined World Orphan Drug Alliance (WODA), an international consortium of pharmaceutical companies, established to improve patient access to new treatments for rare diseases.

WODA operates by identifying biotech and pharmaceutical companies with new therapies for treating rare diseases that may not be available to patients in many regions and providing these companies with the opportunity to commercialise their novel therapies in member countries.

WODA Chairman Patrick Jordan commented: “It’s truly inspiring and exciting to witness the alliance’s expansion. WODA’s commercial presence has now extended to an impressive number of 152 markets covered by nine like-minded pharmaceutical companies, providing extensive global outreach with a local focus. Our members are experienced and high-performance commercialisation companies, each being an expert in their own region.”

He added: “Through seamless collaboration among our members, we provide our partners with a single platform for full commercialisation of medicines, tailored to both partner and product needs.”

Specialised Therapeutics CEO Carlo Montagner said WODA’s mission to address the unmet medical needs of local communities with novel therapies strongly aligned with ST’s vision.

“We firmly believe that patients in our regions should have access to the same innovative treatments as patients have in larger markets like the US and Europe,” Mr. Montagner commented.

“We now look forward to working with our WODA peers to ensure timely and equitable access to new therapies that may improve outcomes. I am confident that our WODA membership will further expand our capabilities, enabling us to provide additional therapies where there is an unmet need.”

###

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, ophthalmology and supportive care, although it is not confined to these areas.

Additional information: www.stbiopharma.com

About WODA

The World Orphan Drug Alliance (WODA) is a global alliance of commercial distributors focused on providing access to treatments for rare diseases and specialty medicines in complex and underserved markets around the world. WODA aims to provide comprehensive support to pharmaceutical and biotech companies with rare disease, oncology, and highly specialized therapeutics portfolio, starting from Named Patient Programs through to full commercialization.

Additional information: www.woda-alliance.com

About other WODA members

EffRx Pharmaceuticals is a Switzerland based company focused on the late-stage development and commercialization of prescription medications for niche and orphan indications.
Medis based in Slovenia is the commercialization partner of choice for innovative pharmaceutical and biotech companies seeking strong business growth in Central and Eastern Europe.
Orpharm is a Moscow based full-service distributor covering Russia and the Commonwealth of Independent States (CIS).
OrphanDC based in Brazil acts as a partner for biotech companies in Latin America. They focus on supporting their clients from the clinical development stage throughout the product lifecycle.
Vector Pharma is a Dubai based full-service distributor covering Middle East, North Africa and Turkey.
The Greater China region is covered by RareStone Group, which aims to become the leading company supporting the rare disease community in China.
Founded in 1921, CTS is among Israel’s leading pharmaceutical companies and is well-known for its strong capabilities in local access, distribution, and marketing of high-end therapies.
Path Pharma is a full-service distributor in Greece, Cyprus, and Malta. Founded by industry experts, Path Pharma has expertise in local market access, medical support, and marketing and sales, with focus on rare diseases, highly specialized therapeutics, and oncology.

Media contact:

Emma Power

+65 3158 9940, +61 419 149 525

epower@stbiopharma.com

Tina Vojnovic

tina.vojnovic@woda-alliance.com

+386 41 744 735

Specialised Therapeutics Acquires Commercialisation Rights to New Oral MND Therapy

Singapore and Tilburg, Netherlands, August 28 2023: Independent biopharmaceutical company Specialised Therapeutics Asia Pte Ltd (ST) will partner with Netherlands based biotechnology company Treeway BV to commercialise a new therapy to treat Amyotrophic Lateral Sclerosis (ALS) – the most common form of Motor Neurone Disease (MND) – in Australia and New Zealand.

The therapy is known as TW001 and is a unique oral formulation of edaravone which works by reducing the oxidative damage associated with neuron death in ALS.¹ TW001 is currently being evaluated in the pivotal ADORE phase III registration study at almost 40 global sites.²

Australian neurologist Associate Professor Susan Mathers said around 2000 people were living with MND at any one time in Australia, and an oral therapy like edaravone presented the opportunity for patients to be managed at home.

Associate Professor Mathers commented: “Better disease modifying therapies are urgently needed to slow and potentially halt this disease. Oral therapies like edaravone present the opportunity for a simple to manage therapy which can be taken at home and monitored through each person’s local health care provider.”

And key patient advocacy body MND Australia is also welcoming the potential for this new oral treatment option.

Executive Director, Research Gethin Thomas commented: “Oral edaravone would complement the recent approval of intravenous edaravone in Australia and broaden the patient base able to access treatment.”

Under the terms of the licensing agreement, ST will be responsible for all marketing, regulatory and distribution activities of TW001 for ALS/MND in Australia and New Zealand.
Announcing the partnership, ST Chief Executive Officer Carlo Montagner said TW001 was the first central nervous system (CNS) therapy to be included in the company’s therapeutic portfolio and the arrangement was further endorsement of ST’s regional capabilities and focus on making available in this region unique therapies that would otherwise not be accessible.

“We are delighted to partner with Treeway as this promising treatment progresses through the final stages of the pivotal global registration ADORE study,” he said. “We look forward to working with the wider MND community, who are determined to access new therapies to treat this terrible disease. “While there is still no cure for MND, we remain hopeful that new therapies such as TW001 may help to slow disease progression and improve outcomes.”

Treeway CEO Inez de Greef commented: “This important therapy has shown very encouraging results in all studies to date. We look forward to further results from the ADORE study and then working with ST to make our therapy available for all eligible patients in Australia and New Zealand who may benefit. ST is focussing on bringing new therapies to the market for diseases with a high medical need and therefore ST fits well as a licensing partner for Treeway.”

Ends.

About Specialised Therapeutics

Headquartered in Singapore, Specialised Therapeutics Asia Pte Ltd (ST) is an international biopharmaceutical company established to commercialise new therapies and technologies to patients throughout Southeast Asia, as well as in Australia and New Zealand. ST and its regional affiliates collaborate with leading global pharmaceutical and diagnostic companies to bring novel, innovative and life-changing healthcare solutions to patients affected by a range of diseases. Its mission is to provide therapies where there is an unmet need. The company’s broad therapeutic portfolio currently includes novel agents in oncology, haematology, neurology, ophthalmology and supportive care. Additional information can be found at www.stbiopharma.com.

About Treeway

Treeway is a clinical-stage biotechnology company with a mission to develop therapies to cure ALS and other neurodegenerative diseases. Founded in 2012 by two ALS patients, Treeway is commited to developing the neurodegenerative disease drugs of tomorrow. Treeway’s research and development portfolio has a strong focus on ALS and Alzheimer’s Disease and is continuously looking to expand the therapeutic targets within the neurodegenerative diseases arena.

Treeway, Therapy development inspired by patients

www.treeway.nl

About ALS³

Amyotrophic Lateral Sclerosis (ALS), the most frequent motor neuron disease, is a progressive neurodegenerative disease of motor neurons in the brain and spinal cord, resulting in progressive paralysis, with death typically within 2 to 5 years of diagnosis.

ALS is a rare disease that typically occurs in people between 40-70 years old, slightly more men than women. It is caused by a multitude of factors: 10-15% of cases may have a genetic/family link, while 85-90% are considered sporadic, with no known cause.

References:

1. AlzForum Foundation Inc. https://www.alzforum.org/therapeutics/edaravone
2. Clinicaltrials.gov ADORE Study (NCT05178810)
3. Masrori and Van Damme; Amyotrophic lateral sclerosis: a clinical review. European Journal of Neurology 2020, 27: 1918– 1929

Further Enquiries:

Aster van Oordt
Treeway Communication Manager
Email: info@treeway.nl

Emma Power
Specialised Therapeutics Communications Manager
M: +61 419 149 525
epower@stbiopharma.com

Global Sarcoma Therapy Now Listed on Pharmaceutical Benefits Scheme

YONDELIS^® (trabectedin) now PBS listed for Australian patients

Listing described as “wonderful news” for patients living with rare lipo and leiomyo sarcomas

YONDELIS^® (trabectedin) demonstrates 45% reduction in risk of disease progression or death versus dacarbazine¹

Singapore, 31 July 2023: AUSTRALIAN cancer patients who have been diagnosed with rare soft tissue sarcomas will now have affordable access to a global therapy shown to improve survival, following its listing on the Pharmaceutical Benefits Scheme (PBS).

The therapy YONDELIS^® (trabectedin) is a novel anti-tumour agent originally derived from the sea squirt and will be available to eligible patients on the PBS from August 1.

It is used extensively around the world and has been shown to improve progression-free survival for patients with liposarcoma and leiomyosarcoma when used after anthracycline-based therapy.¹

Until today, some patients have paid up to $50,000 to access YONDELIS treatment.

News of the PBS listing is being welcomed by oncologists and the Australian sarcoma community, who say it will alleviate cost of treatment pressures for those patients whose disease has progressed.

Medical oncologist and Scientific Advisory Committee member and Lead of the ANZSA National Sarcoma Database Dr Susie Bae, said YONDELIS has been available in Europe since 2007 for patients with advanced soft tissue sarcoma, and Australian patients had waited many years for reimbursed access.

“This milestone means patients don’t need to worry about not being able to afford or miss out on an active drug that can potentially buy precious time with their loved ones, by providing disease control and keeping symptoms at bay for longer,” Dr Bae said.

Melbourne patient advocate and mother of two Karen Lurati – herself diagnosed with liposarcoma six years ago – said this listing provided new hope for other patients.

“A PBS listing for YONDELIS is so exciting for those people who may not have been able to afford the treatment before,” she said. “Rare cancers don’t often get (Government) funding or attention. To now have this therapy on the PBS is great progress.

“Patients often feel that they have to go overseas and spend enormous amounts of money on treatments that may not be available in Australia. This can be frustrating and financially crippling. So, for patients to have access to a global therapy in their own country is wonderful news.”

And Rare Cancers Australia (RCA) Chief Executive Richard Vines said he was “delighted with this outcome”, describing the listing as “great news” for patients living with an L-sarcoma.

“For too long, sarcoma patients have been unable to access all therapies which may provide benefit,” he said. Today’s announcement means they can access a PBS funded medicine instead of having to try and find tens of thousands of dollars – if not more – to self-fund a treatment that may give them more time.”

YONDELIS is marketed in Australia by independent pharmaceutical company Specialised Therapeutics, under an exclusive license arrangement with international partner PharmaMar.

ST Chief Executive Officer Carlo Montagner said the PBS listing was a significant milestone for the company.

He commented: “We acquired the YONDELIS rights in 2019 following requests from key oncology groups and doctors, who had been importing the product at great cost and with complex logistics for those patients diagnosed with these rare cancers.

“This PBS listing is the culmination of a substantive effort by our team together with the oncology community to achieve full regulatory approval and a PBS listing.

“We look forward to continuing our work with the sarcoma community.”

Ends.

Further Inquiries can be directed to ST Communications Manager Emma Power via email epower@stbiopharma.com or on +61 419 149 525.

About Specialised Therapeutics Asia
Headquartered in Singapore, Specialised Therapeutics (ST) is an international biopharmaceutical company established to commercialise new therapies and technologies to patients in Australia, New Zealand and across South-East Asia. ST and its regional affiliates collaborate with leading global pharmaceutical and diagnostic companies to bring novel, innovative and life-changing healthcare solutions to patients affected by a range of diseases. Our mission is to provide therapies that would otherwise not be available to communities in our regions. The company’s broad therapeutic portfolio currently includes novel agents in oncology, haematology, neurology, ophthalmology and supportive care. Additional information can be found at www.stbiopharma.com

About YONDELIS^® (trabectedin)

YONDELIS^® (trabectedin) is a novel, multimodal, synthetically produced antitumor agent, originally derived from the sea squirt, Ecteinascidia turbinata. The anti-cancer medicine works by preventing tumor cells from multiplying and is approved in 76 countries in North America, Europe, South America and Asia for the treatment of advanced soft-tissue sarcomas as a single-agent, and in 69 countries for relapsed ovarian in combination with doxorubicin HCl liposome injection.

The approval was based on the results of a pivotal phase 3, randomised, open-label controlled study which evaluated YONDELIS versus dacarbazine in over 500 patients with unresectable or metastatic liposarcoma (LPS) or leiomyosarcoma (LMS) previously treated with an anthracycline and at least one additional chemotherapy regimen. LPS and LMS are subtypes of soft tissue sarcoma (STS) and represent more than 35% of all STS cases.³

The median progression-free survival (PFS) among the YONDELIS treatment group was 4.2 months compared to 1.5 months in the dacarbazine treatment group, representing a 45% reduction in the risk of disease progression or death with YONDELIS (HR=0.55; 95% CI: 0.44 – 0.70; p<0.001).¹

Among the 340 patients who received YONDELIS and were included in the safety analysis in the randomised trial, the most common (≥20%) adverse reactions were nausea (73%), fatigue (67%), vomiting (44%), constipation (36%), decreased appetite (34%), diarrhoea (34%), dyspnoea (25%), peripheral oedema (24%) and headache (23%). The most common (≥20%) laboratory abnormalities were neutropenia (49%), increased alanine transaminase (ALT) (45%), anaemia (39%), increased aspartate aminotransferase (AST) (35%), thrombocytopaenia (30%) and increased blood alkaline phosphatase (20%).¹

About Soft Tissue Sarcoma

Soft tissue sarcoma is a rare type of cancer that forms as a painless lump (tumour) in any one of the soft tissues connecting all the organs and body structures – including fat, muscle, nerves, deep skin tissue, blood vessels and the tissue surrounding joints (synovial tissue). Soft tissue sarcomas commonly develop in the thigh, shoulder and pelvis and may sometimes develop in the abdomen or chest.⁶

Metastatic or locally advanced STS is generally considered incurable, with the mainstay of treatment being systemic chemotherapy. For some patients with limited disease burden however, long-term remission can be achieved through a multimodality approach involving medical, surgical and radiation therapy.⁴

About PharmaMar

PharmaMar is a biopharmaceutical company focused on the research and development of new oncology treatments, whose mission is to improve the healthcare outcomes of patients afflicted by serious diseases with our innovative medicines. The Company is inspired by the sea, driven by science, and motivated by patients with serious diseases to improve their lives by delivering novel medicines to them. PharmaMar intends to continue to be the world leader in marine medicinal discovery, development and innovation.

PharmaMar has developed and now commercializes Yondelis^® in Europe by itself, as well as Zepzelca^® (lurbinectedin), in the US; and Aplidin^® (plitidepsin), in Australia, with different partners. In addition, it has a pipeline of drug candidates and a robust R&D oncology program. PharmaMar has other clinical-stage programs under development for several types of solid cancers: lurbinectedin, ecubectedin, PM534 and PM54. It also has a preclinical and clinical program in virology. Headquartered in Madrid (Spain), PharmaMar has subsidiaries in Germany, France, Italy, Belgium, Austria, Switzerland and The United States. PharmaMar also wholly owns Sylentis, a company dedicated to researching therapeutic applications of gene silencing (RNAi). To learn more about PharmaMar, please visit us at www.pharmamar.com.

References

Demetri G. et al. J Clin Oncol. 2016; 34(8): 786-793
Australian Institute of Health and Welfare data on file 2022
Toro JR, et al. Int J Cancer. 2006; 119:2922-2930
Cancer Council Victoria Fact Sheet – Soft tissue sarcoma. Available at https://www.cancervic.org.au/cancer-information/types-of-cancer/soft_tissue_cancers/soft-tissue-cancers-overview.html

Lymphoma Therapy Now Approved for Australian Patients with Diffuse Large B-cell Lymphoma

MINJUVI^® (tafasitamab) provisionally approved by Therapeutic Goods Administration¹

Recent five-year follow-up data from Phase 2 L-MIND investigation showed patients treated with MINJUVI had prolonged, durable responses²

Singapore, 28 June 2023: Independent biopharmaceutical company Specialised Therapeutics (ST) is pleased to announce that a new therapy to treat the most common type of non-Hodgkin lymphoma in adults – diffuse large B-cell lymphoma – is now approved for use in Australia.

The Therapeutic Goods Administration (TGA) has provisionally approved MINJUVI^® (tafasitamab) “in combination with lenalidomide followed by MINJUVI monotherapy for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT)”.¹

Australian lymphoma specialist and current chair of the Australasian Lymphoma Alliance, Professor Chan Cheah, said the MINJUVI approval was a great step forward for patients who had been diagnosed with DLBCL and relapsed, as the MINJUVI regimen provides an opportunity for longer-term disease management.

“I think it is great news for patients,” Professor Cheah said. “We do have chemotherapy options and we cure about two-thirds of patients using that approach. Unfortunately, a substantial proportion of patients either don’t respond to chemotherapy, or the disease comes back after chemotherapy, and they need better treatments.”

MINJUVI, a CD19-targeting immunotherapy that works by attaching to a protein on the surface of B-cell lymphoma cells, stimulating an immune response against the lymphoma, is also approved in the United States [as Monjuvi^® (tafasitamab-cxix)], Great Britain, Canada, Europe and other countries.

Professor Cheah added: “Access to novel immune therapies like MINJUVI is really important for Australian patients. Apart from CAR-T cell therapies – and these are only applicable to a certain proportion of patients with DLBCL – there have been no novel therapies for relapsed DLBCL approved in Australia. MINJUVI has a favourable side effect profile and (combined with lenalidomide) has demonstrated a high response rate in patients with relapsed disease. We now need to see it listed on the Pharmaceutical Benefits Scheme.”

MINJUVI has been approved via a provisional regulatory pathway, with the TGA participating in the Modified Project Orbis initiative to accelerate availability to Australian patients. The approval was based on data from the Phase 2 L-MIND study, an open label, multi-center single arm study which evaluated its safety and efficacy in combination with lenalidomide as a treatment for patients with relapsed or refractory DLBCL who were not eligible for ASCT.^1,3

Continued approval for this indication depends on verification and description of clinical benefit in the confirmatory Phase 3 frontMIND study which has completed enrollment.⁴

Recently, five-year follow up data were presented which showed that MINJUVI plus lenalidomide followed by MINJUVI monotherapy provided prolonged, durable responses in adult patients with relapsed or refractory DLBCL. The overall response rate (ORR) was 57.5% with a complete response (CR) observed in 41.2% of patients, and a partial response (PR) in 16.2% of patients. The median overall survival was 33.5 months and median progression-free survival (PFS) was 11.6 months.² The most common adverse reactions with MINJUVI are infections (73%), neutropenia (51%), asthenia (40%), anaemia (36%), diarrhoea (36%), thrombocytopenia (31%), cough (26%), oedema peripheral (24%), pyrexia (24%), decreased appetite (22%). The most common serious adverse reactions were infection (26%) including pneumonia (7%), and febrile neutropenia (6%).¹

ST Chief Executive Officer Mr. Carlo Montagner said securing TGA approval was a key regulatory milestone for the company, noting that the therapy was synergistic with the company’s mission to provide therapies that addressed unmet needs in rare patient populations.

“We are delighted to successfully register MINJUVI for Australian patients and look forward to working with the lymphoma community to ensure it is available at the earliest opportunity,” he said.

ST markets MINJUVI under an exclusive distribution arrangement with international partner Incyte (NASDAQ: INCY).

Ends.

About Specialised Therapeutics Asia

Headquartered in Singapore, Specialised Therapeutics Asia Pte Ltd (STA) is an international biopharmaceutical company established to commercialise new therapies and technologies to patients throughout Southeast Asia, as well as in Australia and New Zealand. ST and its regional affiliates collaborate with leading global pharmaceutical and diagnostic companies to bring novel, innovative and life-changing healthcare solutions to patients affected by a range of diseases. Its mission is to provide therapies where there is an unmet need. The company’s broad therapeutic portfolio currently includes novel agents in oncology, haematology, neurology, ophthalmology and supportive care.

Additional information can be found at www.stbiopharma.com

About Diffuse Large B-cell Lymphoma (DLBCL)

DLBCL is the most common type of non-Hodgkin lymphoma in adults worldwide⁵, characterised by rapidly growing masses of malignant B-cells in the lymph nodes, spleen, liver, bone marrow or other organs. It is an aggressive disease with about 40% of patients not responding to initial therapy or relapsing thereafter⁵, leading to a high medical need for new, effective therapies⁶, especially for patients who are not eligible for an autologous stem cell transplant in this setting.

About L-MIND

The L-MIND trial was a single arm, open-label Phase 2 study (NCT02399085) investigating the combination of tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma who had at least one, but no more than three, prior lines of therapy, including an anti-CD20 targeting therapy (e.g., rituximab), who were not eligible for high-dose chemotherapy or refused subsequent autologous stem cell transplant. The study’s primary endpoint was overall response rate. Secondary outcome measures included duration of response, progression-free survival and overall survival. In May 2019, the study reached its primary completion. For more information about L-MIND, visit https://clinicaltrials.gov/ct2/show/NCT02399085.

About MINJUVI^® (tafasitamab-cxix)

Tafasitamab is a humanized Fc-modified CD19 targeting immunotherapy. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb^® engineered Fc domain, which is intended to lead to a significant potentiation of Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP), thus aiming to improve a key mechanism of tumor cell killing.

MINJUVI known as Monjuvi^® (tafasitamab-cxix) in the United States is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved underaccelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In Europe, Minjuvi^® (tafasitamab) received conditional marketing authorization in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT).

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in several ongoing combination trials.

Monjuvi^® and Minjuvi^® are registered trademarks of MorphoSys AG. Tafasitamab is co-marketed by Incyte and MorphoSys under the brand name Monjuvi^® in the U.S., and marketed by Incyte under the brand name Minjuvi^® in Europe and Canada.

XmAb^® is a trademark of Xencor, Inc.

References:

Minjuvi® (tafasitamab). Product Information, Australia.
Minjuvi® (tafasitamab). Oral Abstract # CT022 Five-year follow-up of Phase 2 L-MIND study announced at the American Association for Cancer Research (AACR) Annual Meeting 2023 in Orlando, Florida.
Salles, G. et al. (2020) ‘Tafasitamab plus lenalidomide in relapsed or refractory diffuse large B-cell lymphoma (L-mind): A multicentre, prospective, single-arm, phase 2 study’, The Lancet Oncology, 21(7), pp. 978–988. doi:10.1016/s1470-2045(20)30225-4.
gov/study/NCT04824092?term=frontMIND&intr=tafasitamab&rank=1
Sarkozy C, et al. Management of relapsed/refractory DLBCL. Best Practice Research & Clinical Haematology. 2018 31:209–16. doi.org/10.1016/j.beha.2018.07.014.
Skrabek P, et al. Emerging therapies for the treatment of relapsed or refractory diffuse large B cell lymphoma. Current Oncology. 2019 26(4): 253–265. doi.org/10.3747/co.26.5421.

New Therapy for Rare Gastrointestinal Stromal Tumours Approved in Singapore

Singapore’s Health Sciences Authority (HSA) has approved QINLOCK^® (ripretinib) for the treatment of patients with 4^th line GIST

QINLOCK significantly reduced the risk of disease progression or death by 85% and showed clinically meaningful overall survival in the INVICTUS Phase 3 Study^1,2

Singapore, 8 May 2023: Independent biopharmaceutical company Specialised Therapeutics Asia (ST) is pleased to announce that a new therapy to treat rare gastrointestinal stromal tumours (GIST) shown to improve survival has been approved for use in Singapore.

The therapy, QINLOCK (ripretinib) is now approved by the Health Sciences Authority (HSA) “for the treatment of adult patients with advanced gastrointestinal stromal tumours (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib, sunitinib, and regorafenib”.

Singapore-based senior consultant in medical oncology Dr Richard Quek said QINLOCK represented a major treatment advancement for patients with advanced GIST.

“Since 2013, despite multiple attempts and studies, no therapy was shown to be effective for 4^th line GIST patients whose cancers have progressed on existing treatment, until the discovery of QINLOCK,” Dr Quek said.

In the pivotal INVICTUS study that led to QINLOCK’s approval, QINLOCK was shown to significantly delay cancer progression.

“This approval in Singapore clearly provides an opportunity for us to improve the outcomes of our GIST patients who are refractory to the current existing treatment.”

QINLOCK is an oral medication used to treat GIST in people who have received at least three prior treatments. It belongs to a drug class called tyrosine kinase inhibitors and works by blocking specific tumour proliferation pathways.²

A pivotal Phase 3 clinical trial of QINLOCK – the INVICTUS study – demonstrated that QINLOCK was able to significantly reduce the risk of disease progression by 85% (hazard ratio of 0.15, p<0.0001) with a median progression-free survival of 6.3 months in patients administered QINLOCK, compared to 1.0 month in the placebo arm.¹ QINLOCK was associated with clinically meaningful overall survival of 15.1 months vs 6.6 months and reduced the risk of death by 64% (hazard ratio of 0.36). The objective response rate by Blinded Independent Central Review using modified Response Evaluation Criteria in Solid Tumors (RECIST) was 9.4% with QINLOCK vs 0.0% with placebo (p=0.0504).^1,3

In addition, in a long-term follow up analysis of the INVICTUS trial, patients in the QINLOCK arm demonstrated a median overall survival of 18.2 months compared to 6.3 months in the placebo arm and reduced the risk of death by 59% (hazard ratio of 0.41).The objective response rate was 11.8% with QINLOCK vs 0.0% with placebo.³

ST Chief Executive Officer Carlo Montagner said the Singapore approval followed the recent approval of QINLOCK in New Zealand, as well as regulatory and reimbursement approval in Australia.

“Achieving these critical regulatory milestones is testament to the dedication of our regulatory teams to make QINLOCK available to all eligible patients in Singapore who are impacted by this rare gastrointestinal cancer.”

ST commercialises QINLOCK in Singapore under an exclusive distribution agreement from US based Deciphera Pharmaceuticals.

Further Inquiries can be directed to ST Senior Manager Communications and Corporate Affairs Emma Power on + 65 31589910 epower@stbiopharma.com

About GIST

Gastrointestinal stromal tumour (GIST) is a cancer affecting the digestive tract or nearby structures within the abdomen, most often presenting in the stomach or small intestine. GIST growth usually begins in the connective tissue in the wall of the affected organ and grows outwards. The common location of GIST is in the stomach (50 to 60%) and small intestines (30 to 40%) but can occur in any site in the digestive system. Other possible GIST sites are the oesophagus, rectum, and colon. GIST cases are rare and estimated to cause between 0.1% and 3% of GI cancer. The risk of GIST diagnosis increases with age, with GIST incidence peaking among people in their fifties and sixties.⁴

About QINLOCK (ripretinib)

QINLOCK is a switch-control tyrosine kinase inhibitor that was engineered to broadly inhibit KIT and PDGFRA mutated kinases by using a dual mechanism of action that regulates the kinase switch pocket and activation loop. QINLOCK inhibits primary and secondary KIT mutations in exons 9, 11, 13, 14, 17, and 18 involved in GIST, as well as the primary exon 17 D816V mutation. QINLOCK also inhibits primary PDGFRA mutations in exons 12, 14, and 18, including the exon 18 D842V mutation, involved in a subset of GIST.^5,6

About Specialised Therapeutics

Headquartered in Singapore, Specialised Therapeutics (ST) is an international biopharmaceutical company established to commercialise new therapies and technologies to patients in Australia, New Zealand and across South-East Asia. ST and its regional affiliates collaborate with leading global pharmaceutical and diagnostic companies to bring novel, innovative and life-changing healthcare solutions to patients affected by a range of diseases. Our mission is to provide therapies that would otherwise not be available to communities in our regions. The company’s broad therapeutic portfolio currently includes novel agents in oncology, haematology, neurology, ophthalmology and supportive care. Additional information can be found at www.stbiopharma.com

About the INVICTUS Phase 3 Study

INVICTUS is a Phase 3 randomised, double-blind, placebo-controlled, international, multicenter clinical study evaluating the safety, tolerability, and efficacy of QINLOCK compared to placebo in patients with advanced GIST whose previous therapies have included at least imatinib, sunitinib, and regorafenib. Patients were randomized 2:1 to either 150 mg of QINLOCK once daily (n=85) or placebo (n=44). The primary efficacy endpoint was progression-free survival (PFS) as determined by independent radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST). The median PFS in the study was 6.3 months in the QINLOCK arm compared to 1.0 month in the placebo arm and significantly reduced the risk of disease progression or death by 85% (hazard ratio of 0.15, p<0.0001) compared to placebo.¹ Secondary endpoints included Objective Response Rate (ORR) as determined by independent radiologic review using modified RECIST and Overall Survival (OS). QINLOCK demonstrated an ORR of 9.4% compared with 0% for placebo (p=0.0504), which was not statistically significant.¹ QINLOCK demonstrated a median OS of 15.1 months compared to 6.6 months in the placebo arm and reduced the risk of death by 64% (hazard ratio of 0.36).¹ In a long-term follow up of 19 months after the primary analysis, QINLOCK also demonstrated a median OS of 18.2 months compared to 6.3 months in the placebo arm and reduced the risk of death by 59% (hazard ratio of 0.41).³The most common (>2%) grade 3 or 4 treatment related adverse events in the QINLOCK group included lipase increase (5%), hypertension (4%), fatigue (2%), and hypophosphataemia (2%); and in the placebo group, anaemia (7%), fatigue (2%), diarrhoea (2%), decreased appetite (2%), dehydration (2%), hyperkalaemia (2%), acute kidney injury (2%), and pulmonary oedema (2%).^1,4

References

Blay JY, Serrano C, Heinrich MC et al. Ripretinib in patients with advanced gastrointestinal stromal tumours (INVICTUS): A double-blind, randomised, placebo-controlled, phase 3 trial. Lancet Oncol 2020; 21:923–934.

QINLOCK (ripretinib) HSA Approved Product Information, April 2023
von Mehren M, Heinrich M, George S, et al. Ripretinib as ≥4th‑line treatment in patients with advanced gastrointestinal stromal tumour (GIST): Long-term update from the phase 3 INVICTUS study. Poster presented at: 2021 European Society for Medical Oncology Virtual Meeting; September 16‑21, 2021.

GI Cancer Institute Australia https://gicancer.org.au/cancer/gastro-intestinal-stromal-tumour-gist/#cancer-explanation

Smith BD, Kaufman MD, Lu WP et al. Ripretinib (DCC-2618) is a switch control kinase inhibitor of a broad spectrum of oncogenic and drug-resistant KIT and PDGFRA variants. Cancer Cell 2019; 35(5):738–751.
Bauer S, Heinrich MC, George S et al. Clinical Activity of Ripretinib in Patients with Advanced Gastrointestinal Stromal Tumor Harboring Heterogeneous KIT/PDGFRA Mutations in the Phase III INVICTUS Study. Clin Cancer Res. 2021;27(23): 6333-6342.