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 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.

Ends.

Further enquiries:

  • ST Senior Manager Communications and Corporate Affairs, Emma Power

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

  1. SKYTROFA (lonapegsomatropin-tcgd) US Prescribing Information.
  2. SKYTROFA (lonapegsomatropin) EU Summary of Product Characteristics.
  3. YORVIPATH (palopegteriparatide) EU Summary of Product Characteristics.
  4. Savarirayan, R, Hoemschemeyer DG, Ljungberg M, et al., Lancet. 2023;65:1–10.
  5. Thornton PS, Maniatis AK, Aghajanova E, et al., J Clin Endocrinol Metab. 2021;106(11):3184–3195.
  6. Maniatis AK, Nadgir U, Saenger P, et al., Horm Res Paediatr. 2022;95(3):233–243.
  7. Maniatis AK, Casella SJ, Nadgir UM, et al. J Clin Endocrinol Metab. 2022;107(7): e2680–e2689.
  8. Foresight Trial (NCT05171855), https://clinicaltrials.gov/study/NCT05171855.
  9. Khan AA, Rubin MR, Schwarz P, et al. J Bone Miner Res. 2023;38(1):14–25.
  10. 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:

  1. Brewer M, et al. Gynecol Oncol. 2020.156(3):523-529
  2. ClinicalTrials.gov NCT04498117
  3. 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 

  1. Demetri G. et al. J Clin Oncol. 2016; 34(8): 786-793
  2. Australian Institute of Health and Welfare data on file 2022
  3. Toro JR, et al. Int J Cancer. 2006; 119:2922-2930
  4. 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:

  1. Minjuvi® (tafasitamab). Product Information, Australia.
  2. 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.
  3. 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.
  4. gov/study/NCT04824092?term=frontMIND&intr=tafasitamab&rank=1
  5. 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.
  6. 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 4th 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 Study1,2

 

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 4th 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).3 The 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 

  1. 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.
  1. QINLOCK (ripretinib) HSA Approved Product Information, April 2023
  2. 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.
  1. GI Cancer Institute Australia https://gicancer.org.au/cancer/gastro-intestinal-stromal-tumour-gist/#cancer-explanation
  1. 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.
  2. 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.

 

 




ST to Commercialise New Anti-PD1 Antibody

Specialised Therapeutics signs partnership agreement with Akeso Inc. and CTTQ-Akeso to commercialise new anti-PD1 antibody in Australia and Southeast Asia

Singapore and Beijing, China, 12 April 2023: Independent biopharmaceutical company Specialised Therapeutics Asia Pte Ltd (ST) will partner with CTTQ-Akeso (Shanghai) Biomed. Tech. Co., Ltd. (CTTQ-Akeso) (jointly established by Akeso, Inc. (9926.HK, Akeso) and Chia Tai Tianqing Pharmaceutical Group Co., Ltd.)to commercialise a new immuno-oncology therapy in Australia, Singapore and across Southeast Asia.

The therapy ANNIKO® (penpulimab) is an anti-PD1 monoclonal antibody currently under regulatory review by the US FDA for nasopharyngeal carcinoma – a difficult to treat form of head and neck cancer.

This follows the FDA granting ANNIKO orphan drug and fast track designations in 2020, as well as a further “breakthrough therapy” designation in March 2021. In addition, ANNIKO was granted a FDA Real-Time Oncology Review (RTOR) in 2021, to accelerate the drug approval process.1,2

ANNIKO has been approved in China for the treatment of adult patients with relapsed or refractory classical Hodgkin’s lymphoma (advanced r/r cHL) who have undergone at least second-line chemotherapy, as well as first-line treatment of locally advanced or metastatic squamous non-small cell lung cancer (sq-NSCLC) in combination with chemotherapy.3

Under the terms of the arrangement, ST will be responsible for all marketing, regulatory and distribution activities in its key regions of Australia, Singapore and across Southeast Asia.

CTTQ-Akeso retains the rights of conducting any development work in relation to ANNIKO and Akeso retains all rights to product manufacture and supply.

Announcing the partnership, ST Chief Executive Officer Carlo Montagner said ANNIKO was the first immuno-oncology drug to be included in the company’s therapeutic portfolio and the arrangement was further endorsement of ST’s regional capabilities.

“ANNIKO will provide a new opportunity for patients in our regions with nasopharyngeal carcinoma – a very difficult-to-treat cancer – to be treated with an immune-based therapy,” he said.

“Nasopharyngeal carcinoma is native to Southeast Asia, affecting between 15 and 50 people in every 100,0004 and with almost 37,000 new cases diagnosed annually in this region.5 Making ANNIKO available for this disease is a high priority.”

Akeso CEO Michelle Xia said the company looked forward to collaborating with ST and providing eligible cancer patients with world-class therapy.

“We trust that ST’s expertise in these regions, navigating complex regulatory channels will ensure our therapy reaches as many eligible patients as possible,” she said.

“We look forward to a successful and mutually beneficial partnership, working together with a shared goal of improving patient outcomes.”

Regulatory activities are currently in progress.

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 Akeso Inc.

Akeso Inc (HKEX: 09926) is a commercial-stage biopharmaceutical company committed to the discovery, development, manufacturing and commercialization of innovative medicines with high unmet medical needs worldwide. Founded in 2012, the company has established a comprehensive in-house drug development platform (ACE Platform) and know-how, including R&D, clinical development, CMC (Chemistry, Manufacturing, and Controls), and commercialization capabilities. With fully integrated multi-functional platform, Akeso is internally working on a robust pipeline of over 30 innovative assets in the fields of cancer, autoimmune disease, inflammation, metabolic disease, and other major therapeutic areas. 17 assets have entered into clinical stage. Akeso has advanced four potential first-in-class bispecific antibody drugs into market or clinical development, including cadonilimab (PD-1 / CTLA-4), ivonescimab (PD-1 / VEGF), PD-1 / LAG-3, and TIGIT / TGF-Beta bispecific antibodies. In June 2022, cadonilimab was approved by the NMPA and became the first commercialized PD-1 based bispecific drug globally. Another Akeso internally discovered and developed oncology product, penpulimab (a PD-1 antibody), was granted marketing approval in China in August 2021. Akeso is listed on the Main Board of the Stock Exchange of Hong Kong Limited.

About CTTQ-Akeso

CTTQ-Akeso (Shanghai) Biomed. Tech. Co., Ltd. (CTTQ-Akeso) is an innovative biomedical company jointly established by Akeso, Inc. (9926.HK) and Chia Tai Tianqing Pharmaceutical Group Co., Ltd. CTTQ-Akeso is focused on the R&D and commercialization of Penpulimab (安尼可®).

About Anniko® (penpulimab)

ANNIKO (penpulimab) is a humanised anti-programmed cell death 1 (PD-1) monoclonal antibody developed by Akeso Biosciences for the treatment of various cancers. ANNIKO is an immunoglobulin G1 monoclonal antibody uniquely engineered to completely eliminate Fcγ receptor binding and Fc-mediated effector functions that can compromise anti-tumour activity. ANNIKO is approved in China for the treatment of adult patients with relapsed or refractory classic Hodgkin’s lymphoma (advanced r/r cHL) who have undergone at least second-line chemotherapy, as well as for Sq-NSCLC. The treatment for 1L sq-NSCLC in combination with chemotherapy has been included as a level II recommendation in the Chinese Society of Clinical Oncology (CSCO) Guidelines. The treatment for advanced r/r cHL has been included as a level I recommendation in the CSCO Guidelines. The treatment for advanced nasopharyngeal carcinoma (NPC) has been included as a level III recommendation in the CSCO Guidelines. ANNIKO is under regulatory review for nasopharyngeal cancer in the US and China. Clinical studies with ANNIKO are underway for various cancers in China and Australia.6

 

About Nasopharyngeal Carcinoma

Nasopharyngeal carcinoma (NPC) is a malignant tumour with poor survival outcomes. The incidence of NPC is rare in western countries but significantly higher in Southeast Asia. The dramatic geographic difference implies a link between NPC and genetic and/or environmental factors. Indeed, consumption of preserved foods, tobacco smoking, exposure to Epstein Barr virus (EBV) and genetic characteristics have been clearly linked with NPC. However, how these factors lead to NPC remains unclear.7

 

References:

  1. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=802020
  2. https://www.akesobio.com/en/media/akeso-news/21-5-24/
  3. Akeso Inc. Penpulimab 安尼可®: Chinese prescribing information. Zhongshan City, 2021.
  4. Mahdavifar N, Ghoncheh M, Mohammadian-Hafshejani A et al. Epidemiology and Inequality in the Incidence and Mortality of Nasopharynx Cancer in Asia. Osong Public Health Res Perspect. 7(6): 360-372, 2016.
  5. Nasopharynx factsheet. Globocan Data 2020. https://gco.iarc.fr/today/data/factsheets/cancers/4-Nasopharynx-fact-sheet.pdf
  6. Dhillon S. Penpulimab: First Approval. Drugs. 81(18):2159-2166, 2021; doi:10.1007/s40265-021-01640-9
  7. World Cancer Research Fund International https://www.wcrf.org/researchwefund/dietary-and-genetic-factors-and-risk-of-nasopharyngeal-cancer-in-south-east-asia



Global Sarcoma Therapy Now Approved for New Zealand Patients

  • Globally regarded soft-tissue sarcoma therapy has been approved by Medsafe for New Zealand patients
  • YONDELIS® (trabectedin) demonstrates 45% reduction in risk of disease progression or death versus dacarbazine1

 

Singapore and Auckland, New Zealand, 17 February 2023: Independent biopharmaceutical company Specialised Therapeutics (ST) is pleased to announce that its portfolio therapy to treat rare soft tissue sarcomas has now been approved in New Zealand.

Medsafe has approved the use of YONDELIS® (trabectedin) “for the treatment of patients with unresectable or metastatic liposarcoma or leiomyosarcoma who received a prior anthracycline-containing regimen”.2

YONDELIS (trabectedin) is used extensively around the world and has been shown to improve progression free survival1.

News of the Medsafe registration has been welcomed by oncologists and the New Zealand sarcoma community, who say it means patients whose disease has progressed will have access to a new line of therapy.

“Sarcoma is a relatively rare cancer and treatment options are limited for those with advanced disease,” said Associate Professor Jayesh Desai, Medical Oncologist and Deputy Chair of the Australia and New Zealand Sarcoma Association (ANZSA)

“We welcome news that this therapy is formally approved for use in New Zealand and look forward to seeing advanced sarcoma patients being provided additional benefit.”

And ANZSA Chief Executive Officer Dr Denise Caruso said: “YONDELIS is an established therapy that has already been used extensively around the world to treat advanced sarcoma patients. We welcome formal registration in New Zealand and expect that all eligible New Zealand patients will be provided access to this global treatment option.” 

YONDELIS is already approved and has been available to patients in the United States since 2015,3 and in Europe since 2007.4 It was approved by Australia’s Therapeutic Goods Administration in 2021.5 

YONDELIS is made available in New Zealand by independent biopharmaceutical company Specialised Therapeutics Asia (ST) under an exclusive license arrangement with international partner PharmaMar SA. 

ST Chief Executive Officer Mr Carlo Montagner commented: “We are pleased to achieve this milestone in New Zealand and look forward to providing patients with sarcoma with another valuable treatment option. 

“We will continue working with the sarcoma community in New Zealand to ensure that YONDELIS is available at the earliest opportunity.” 

 

Ends.

  

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. 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 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 and ecubectedin. 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

 

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 tumour 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 DOXIL®/CAELYX® (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.6 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, 2 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.7 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.7 Leiomyosarcoma (LMS) is the most common subtype of STS, accounting for approximately 20 to 25% of all newly diagnosed cases.7 Common sites for LMS include the abdomen, retroperitoneum, larger blood vessels, and the uterus. It is less common in the extremities compared with other STS subtypes, accounting for 10% to 15% of limb sarcomas.8 Liposarcoma (LPS) accounts for approximately 10 to 15% of cases of STS. Typical sites of origin include the extremities and retroperitoneum.9 

 

Further Enquiries:

Emma Power, Corporate Affairs and Communications Manager, Specialised Therapeutics Asia +65 3158 9940 or +61 419 149 525 or epower@stbiopharma.com 

 

References

  1. Demetri G. et al. J Clin Oncol. 2016; 34(8): 786-793
  2. YONDELIS (trabectedin) Medsafe Approved Data Sheet
  3. YONDELIS (trabectedin) FDA Approved Prescribing Information
  4. YONDELIS (trabectedin) European Medicines Agency Summary of Product Characteristics
  5. YONDELIS (trabectedin) TGA Approved Product Information
  6. Toro JR, et al. Int J Cancer. 2006; 119:2922-2930
  7. 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\
  8. George S, et al. Soft Tissue and Uterine Leiomyosarcoma. J Clin Oncol. 2017; 36: 144-150
  9. 11 Lee ATJ, et al. Clinical and Molecular Spectrum of Liposarcoma. J Clin Oncol. 2018; 36 (2): 151-159.

 




New Therapy to Treat Rare Gastrointestinal Stromal Tumour Approved for New Zealand Patients

  • QINLOCK® (ripretinib) now Medsafe approved for GIST patients in NZ
  • QINLOCK now being considered by PHARMAC for reimbursement
  • Data from the INVICTUS Phase 3 study shows QINLOCK reduces risk of disease progression or death by 85%1, 2

 

Singapore and New Zealand, 20 January 2023: Independent biopharmaceutical company Specialised Therapeutics Asia (ST) is pleased to announce that a new therapy to treat rare gastrointestinal stromal tumour (GIST) shown to improve survival has now been approved in New Zealand.

The therapy, QINLOCK® (ripretinib) has been approved for use by the country’s regulatory agency Medsafe for the treatment of adult patients with GIST who have received prior treatment with three or more kinase inhibitors, including imatinib.

It is currently being made available to eligible patients in New Zealand via a co-pay Access Program while it is considered by PHARMAC for reimbursement.

Cancer specialists said the approval was welcome news for NZ patients diagnosed with GIST.

In a joint statement, medical oncologists Dr Joanna Connor and Dr Clement Korenbaum from the Auckland City Hospital said QINLOCK would provide patients with more treatment options, aligning with international standard of care.

“QINLOCK offers clinically meaningful benefits in progression free and overall survival for patients living with advanced GIST,” they noted. “It offers an option for patients who have progressed on other treatments.”

“We would now support having fully funded access to QINLOCK for all those affected by advanced GIST who meet the pivotal study’s criteria.”

61-year-old Auckland grandfather and sales manager Tom Turrall was diagnosed with GIST earlier this year after being admitted to hospital for what doctors initially believed was a bleeding ulcer.

“The approval of QINLOCK means hope and an opportunity,” Mr Turrall said.

“I want to live to see my grandchildren grow. I want to live to be able to experience growing old with my wife. I want to live to be able to spend some time relaxing and enjoying what we have worked for. The approval of QINLOCK will remove a level of anxiety that I live with every day as it provides an opportunity for an additional treatment option.”

Mr Turrall said it was critical that PHARMAC now funded QINLOCK for patients to ensure all those who are eligible for treatment can afford the therapy.

“PHARMAC funding will mean the financial burden is eased. We consider ourselves the average Kiwi couple. We have worked hard for what we have accumulated and are looking forward to retirement. We are not sure what we will do if we have to fund (any) treatment ourselves.”

QINLOCK belongs to a class of drugs known as tyrosine kinase inhibitors, or TKIs. It is designed to inhibit key enzymes linked to tumour growth. In Australia it has been fully reimbursed on the Pharmaceutical Benefits Scheme since 2021.

A pivotal Phase 3 clinical trial of QINLOCK in patients with advanced GIST – 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 (PFS) of 6.3 months in patients administered QINLOCK, compared to 1.0 month in the placebo arm.1 In addition, in a long-term follow up analysis, patients in the QINLOCK arm demonstrated a median overall survival (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).1, 4

QINLOCK is made available in New Zealand by independent pharmaceutical company Specialised Therapeutics (ST) under an exclusive distribution agreement from US based Deciphera Pharmaceuticals.

ST Chief Executive Officer Carlo Montagner said QINLOCK was currently under review by PHARMAC.

“We are hopeful for a positive outcome by PHARMAC so that patients with advanced GIST in New Zealand have ready access to this important new treatment option.”

 

Ends.

  

About GIST

Gastrointestinal stromal tumor (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. The risk of GIST diagnosis increases with age, with GIST incidence peaking among people in their fifties and sixties.5

 

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.6,7 QINLOCK also inhibits primary PDGFRA mutations in exons 12, 14, and 18, including the exon 18 D842V mutation, involved in a subset of GIST.6,7

 

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. 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 the INVICTUS Phase 3 Study

The INVICTUS Phase 3 study was an international, multicenter, randomised, double-blind, placebo-controlled, trial to evaluate the safety, tolerability, and efficacy of QINLOCK compared to placebo in patients with advanced GIST whose previous therapies have included 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 PFS based on disease assessment by blinded independent central review (BICR) using modified Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria. The median PFS in the study was 6.3 months 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 OS. QINLOCK demonstrated an ORR of 9.4% compared with 0% for placebo (p=0.0504)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).4 The most frequently observed adverse drug reactions (≥25%) in a pooled safety population (n=392) treated with QINLOCK were fatigue, alopecia, nausea, myalgia, constipation, diarrhea, palmar-plantar erythrodysesthesia syndrome (PPES), weight decreased, and vomiting.1

 

Further Enquiries:

Emma Power, Corporate Affairs and Communications Manager, Specialised Therapeutics Asia +65 3158 9940 or +61 419 149 525 or epower@stbiopharma.com 

 

References

  1. Blay J-Y 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
  2. QINLOCK (ripretinib) Medsafe approved Product Datasheet
  3. QINLOCK (ripretinib) PBS Listing https://www.pbs.gov.au/browse/medicine-listing
  4. Von Mehren M et.al. Presented at ESMO 2021 virtual meeting, 16-21 September 2021
  5. GI Cancer Institute Australia https://gicancer.org.au/cancer/gastro-intestinal-stromal-tumour-gist/#cancer-explanation
  6. Smith B 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; 35:738–751.
  7. Bauer S, Heinrich M, et al. Clinical activity of ripretinib in patients with advanced gastrointestinal stromal tumor harboring heterogenous KIT/PDGFRA mutations in the phase 3 INVICTUS study. Clinical Cancer Research [online] September 9, 2021. Available from: https://clincancerres.aacrjournals.org/content/early/2021/09/08/1078-0432.CCR-21-1864 [Last accessed: December 2022].