NEW THERAPY TO TREAT RARE GASTROINTESTINAL STROMAL TUMOUR APPROVED FOR NEW ZEALAND PATIENTS

On January 19, 2023 Independent biopharmaceutical company Specialised Therapeutics Asia (ST) is pleased to reported that a new therapy to treat rare gastrointestinal stromal tumour (GIST) shown to improve survival has now been approved in New Zealand (Press release, Specialised Therapeutics Australia, JAN 19, 2023, View Source [SID1234626403]).

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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."

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

10x Genomics to Report Fourth Quarter and Full Year 2022 Financial Results on February 15, 2023

On January 19, 2023 10x Genomics (Nasdaq: TXG) reported it will report financial results for the fourth quarter and full year ended December 31, 2022 after market close on Wednesday, February 15, 2023 (Press release, 10x Genomics, JAN 19, 2023, View Source [SID1234626402]). The company’s management will webcast a corresponding conference call beginning at 1:30 p.m. Pacific Time / 4:30 p.m. Eastern Time to discuss its results, business developments and outlook.

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Live audio of the webcast will be available on the "Investors" section of the company website at: www.10xgenomics.com. The webcast will be archived and available for replay for at least 45 days after the event.

Mirati Announces IND Clearance by U.S. FDA Enabling Phase 1 Initiation for First-in-Class Oral KRASG12D Selective Inhibitor, MRTX1133

On January 19, 2023 Mirati Therapeutics, Inc. (NASDAQ: MRTX), reported the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application of MRTX1133, a potential first-in-class oral KRASG12D selective inhibitor for clinical evaluation (Press release, Mirati, JAN 19, 2023, View Source [SID1234626400]). KRASG12D mutations impact approximately 180,000 patients in the US and Europe, representing approximately a 2.5-fold increase in prevalence compared to KRASG12C mutations. No targeted oncology treatment options currently exist for these patients.

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MRTX1133 is a highly potent investigational inhibitor of the KRASG12D driver mutation and demonstrated selective and reversible inhibition of KRASG12D in both its active and inactive states. In addition, MRTX1133 administration resulted in marked tumor response in preclinical KRASG12D mutated pancreatic cancer models as well as lung and colorectal cancer models. MRTX1133 has demonstrated favorable properties including a low risk for off-target activity and drug interactions and a predicted human half-life of greater than 50 hours.

"The clearance by the FDA to initiate clinical evaluation of MRTX1133, the third program in our KRAS franchise to enter clinical development, is illustrative of the innovative approach to drug discovery and demonstrates the best-in-class capabilities of the Mirati team. This particular mutation has been difficult to target, and we are confident in our novel oral formulation strategy, which we believe will enable near-complete target inhibition over the full dosing interval," said James Christensen, Ph.D. chief scientific officer, Mirati Therapeutics, Inc. "We are thrilled to advance MRTX1133 into clinical development and the potential to positively impact people living with KRASG12D-mutated cancers."

The Phase 1/2 clinical trial will launch in early 2023 with plans for multiple expansion cohorts in pancreatic, colorectal, lung and other KRASG12D tumor types.

Gilead Sciences To Release Fourth Quarter & Full Year 2022 Financial Results On Thursday, February 2, 2023

On January 19, 2023 Gilead Sciences, Inc. (Nasdaq: GILD) reported that its fourth quarter and full year 2022 financial results and full year 2023 financial guidance will be released on Thursday, February 2 after the market closes (Press release, Gilead Sciences, JAN 19, 2023, View Source;Full-Year-2022-Financial-Results-on-Thursday-February-2-2023/default.aspx [SID1234626399]). At 4:30 p.m. Eastern Time that day, Gilead’s management will host a webcast to discuss the company’s fourth quarter and full year 2022 financial results and provide a business update.

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A live webcast will be available on the Investor Relations section of www.gilead.com and will be archived there for one year.

Viracta Therapeutics Announces Orphan Drug Designation Granted by the European Commission to Nana-val for the Treatment of Diffuse Large B-cell Lymphoma

ON January 19, 2023 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company focused on the treatment and prevention of virus-associated cancers that impact patients worldwide, reported that the European Commission (EC) has granted an orphan drug designation (ODD) to Viracta’s all-oral combination product candidate, Nana-val (nanatinostat and valganciclovir), for the treatment of diffuse large B-cell lymphoma (DLBCL). The EC previously granted ODD to Nana-val for the treatment of peripheral T-cell lymphoma (Press release, Viracta Therapeutics, JAN 19, 2023, View Source [SID1234626397]). In addition, Nana-val was previously granted ODD by the U.S. Food and Drug Administration for the treatment of T-cell lymphoma, post-transplant lymphoproliferative disorder, plasmablastic lymphoma, and Epstein-Barr virus-positive (EBV+) DLBCL, not otherwise specified.

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"Nana-val’s sixth orphan drug designation across the U.S. and Europe underscores the high unmet medical need for EBV-positive lymphoma patients worldwide, the broad therapeutic potential of its novel mechanism of action, and the global nature of our clinical and regulatory strategy," said Mark Rothera, President and Chief Executive Officer of Viracta. "This strategy is embodied by our pivotal trial, NAVAL-1, which has more than 60 sites open globally, and cohorts dedicated to multiple subtypes of EBV-positive lymphoma, including DLBCL. Emerging data have shown that EBV-positive DLBCL is a differentiated disease with a significantly lower survival outcome compared to EBV-negative disease. Nana-val has displayed promising preliminary efficacy in EBV-positive DLBCL, leaving it uniquely positioned to treat this underserved patient population."

ODD in the European Union (EU) is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medical Products. To qualify for ODD from the EC, a product candidate must be intended to treat, prevent, or diagnose a life-threatening or chronically debilitating disease that does not affect more than 5 in 10,000 people across the EU. In addition, there must be sufficient clinical or non-clinical data to suggest the product candidate may produce clinically relevant outcomes, and grounds to indicate it can provide a significant benefit over any currently authorized products.

Receiving an ODD from the EC provides companies with certain benefits and incentives including clinical protocol assistance, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees, and ten years of market exclusivity upon receipt of marketing authorization in the EU. The availability of market exclusivity is intended to encourage the development of medicines for rare diseases by protecting them from competition from similar medicines with similar indications, which cannot be marketed during the exclusivity period.

About Nana-val (Nanatinostat and Valganciclovir)

Nanatinostat is an orally available histone deacetylase (HDAC) inhibitor being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which are key to inducing viral genes that are epigenetically silenced in Epstein-Barr virus (EBV)-associated malignancies. Nanatinostat is currently being investigated in combination with the antiviral agent valganciclovir as an all-oral combination therapy, Nana-val, in various subtypes of EBV-associated malignancies. Ongoing trials include a pivotal, global, multicenter, open-label Phase 2 basket trial in multiple subtypes of relapsed/refractory EBV+ lymphoma (NAVAL-1) as well as a multinational Phase 1b/2 trial in patients with EBV+ recurrent or metastatic nasopharyngeal carcinoma and other EBV+ solid tumors.