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].

 



New Treatment for Rare Cancer Cholangiocarcinoma Approved in Australia

  • Available to treat adults with locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement  

    .. PEMAZYRE® (pemigatinib) is available via a co-pay access program in Australia

 

Melbourne, Australia, 15 September 2022: A NEW targeted therapy to treat a rare bile duct cancer called cholangiocarcinoma has been approved for use in Australia.

PEMAZYRE® (pemigatinib) has been provisionally approved by the Therapeutic Goods Administration (TGA) “for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that has progressed after at least one prior line of systemic therapy. The decision to approve this indication has been made on the basis of overall response rate (ORR) and duration of response (DOR).  Continued approval of this indication depends on verification and description of benefit in confirmatory trial(s).” 1

This means it will be available to Australian patients with cholangiocarcinoma who have been tested for an abnormal gene change in their tumour called an FGFR2 fusion or rearrangement – a defect that can drive cancer growth.

This defect is estimated to be present in around 15% of patients diagnosed with cholangiocarcinoma.2

Australian oncologist Dr David Goldstein said the approval of PEMAZYRE was “a significant step forward” for Australian patients who are diagnosed with this rare and aggressive cancer.

He commented: “This TGA approval of a targeted therapy heralds an era of precision medicine in this rare cancer.

“The study underpinning this approval, FIGHT 202, saw clinical outcomes you would not expect from previous experience after initial treatment ceases to be effective.

“This is one of only a few second-line therapies to offer serious real benefits to patients and will be very focused upon the right tumour type.

“Up until now, we have had some chemotherapies that were effective, but only in a minority of patients.

“This TGA approval is a positive first step. Subsequent reimbursement via the Pharmaceutical Benefits Scheme is really the only way forward to translate this scientific knowledge of targeting a specific tumour type into everyday clinical practice.”

PEMAZYRE, developed by Incyte (NASDAQ:INCY) and partnered with independent pharmaceutical company Specialised Therapeutics (ST) for commercialisation in Australia, New Zealand and Singapore, belongs to a class of drugs called kinase inhibitors and works by blocking the abnormal FGFR2 protein in bile duct tumour cells and preventing cell growth.

The TGA approval follows PEMAZYRE’s approval in the United States, Europe, Great Britain, Canada and Japan.

While PEMAZYRE is not yet reimbursed in Australia, it is currently being made available to eligible patients via a co-pay access program.

ST CEO Carlo Montagner said PEMAZYRE was a “wonderful inclusion” to the company’s portfolio of rare cancer therapies and was synergistic with its mission of providing therapies to patient populations where there is a high unmet medical need.

He said: “We look forward to providing this new highly-targeted treatment to eligible patients with cholangiocarcinoma who have limited therapy options.”

PEMAZYRE’s approval is based on a clinical trial known as the FIGHT-202 study – a Phase 2 investigation evaluating the safety and efficacy of PEMZYRE in adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGFR2 fusion or rearrangement. 2

In the primary analysis of 108 patients enrolled in the trial, treatment with PEMAZYRE resulted in an objective response rate of 37% with 3.7% of patients having a confirmed complete response and 33.3% having a confirmed partial response. The disease control rate was 82.2%.1

The safety analysis, including 147 patients, demonstrated that PEMAZYRE was generally well tolerated.

The most common adverse reactions were hyperphosphataemia: includes hyperphosphataemia and increased blood phosphorous (60.5%), alopecia (49.7%), diarrhoea (46.9%), nail toxicity (44.9%), fatigue (43.5%), nausea (41.5%), dysgeusia (40.8%), stomatitis (37.4%), constipation (36.7%), dry mouth (34.0%), dry eye (27.9%), arthralgia (25.9%), hypophosphataemia (23.1%), dry skin (21.8%) and palmar-plantar erythrodysaesthesia syndrome (16.3%).1

 

Ends.

  

About Specialised Therapeutics

Headquartered in Singapore, Specialised Therapeutics (ST) is an international biopharmaceutical company providing new specialist 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 PEMAZYRE®

PEMAZYRE (pemigatinib) a fibroblast growth factor receptor (FGFR) inhibitor, has provisional approval in Australia for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that has progressed after at least one prior line of systemic therapy. The decision to approve this indication has been made on the basis of overall response rate (ORR) and duration of response (DOR). Continued approval of this indication depends on verification and description of benefit in confirmatory trial(s).

PEMAZYRE is marketed by Incyte in the United States, Europe and Japan. Incyte has established various license or distribution agreements for Pemazyre in certain geographies and retains all other rights to develop and commercialize pemigatinib outside of the United States.

PEMAZYRE® is a trademark of Incyte Corporation.

 

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. PEMAZYRE (pemigatinib) Product Information Australia
  2. Abou-Alfa et al. Lancet Oncol 2020;21:671-684