On September 15, 2022 Specialised Therapeutics Asia reported A NEW targeted therapy to treat a rare bile duct cancer called cholangiocarcinoma has been approved for use in Australia (Press release, Specialised Therapeutics Asia, SEP 15, 2022, View Source [SID1234619602]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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

On September 15, 2022 Medtronic plc (the "Company") ( NYSE:MDT) reported that its wholly-owned subsidiary, Medtronic Global Holdings S.C.A. ("Medtronic Luxco"), has priced an offering (the "Offering") of €500,000,000 principal amount of 2.625% senior notes due 2025, €1,000,000,000 principal amount of 3.000% senior notes due 2028, €1,000,000,000 principal amount of 3.125% senior notes due 2031 and €1,000,000,000 principal amount of 3.375% senior notes due 2034 (collectively, the "Notes") (Press release, Medtronic, SEP 15, 2022, View Source [SID1234619601]). All of Medtronic Luxco’s obligations under the Notes will be fully and unconditionally guaranteed by the Company and Medtronic, Inc., a wholly-owned indirect subsidiary of Medtronic Luxco, on a senior unsecured basis.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The net proceeds of the Offering are expected to be used to repay at maturity Medtronic Luxco’s outstanding 0.00% Senior Notes due 2022, 0.375% Senior Notes due 2023 and 0.00% Senior Notes due 2023 and for general corporate purposes. While Medtronic Luxco may elect at a later date to repay, redeem or repurchase such notes prior to maturity, it currently has no intention to repay, redeem or repurchase such notes prior to maturity. The Offering is expected to close on September 21, 2022, subject to customary closing conditions. The joint book-running managers for the Offering are Barclays Bank PLC, BofA Securities Europe SA, Citigroup Global Markets Limited and HSBC Continental Europe.

The Offering is being made only by means of a prospectus dated February 28, 2020 and prospectus supplement (together, the "Prospectus"). You may get these documents for free by visiting EDGAR on the Securities and Exchange Commission website at www.sec.gov. Alternatively, copies of the Prospectus for the Offering may be obtained by contacting Barclays Bank PLC, toll-free at +1-888-603-5847, BofA Securities Europe SA, at +33(0) 1 8770 0000, Citigroup Global Markets Limited, toll-free at +1-800-831-9146 and HSBC Continental Europe, at +1-866-811-8049.

On September 15, 2022 J INTS BIO reported the poster presentation of its novel, orally administered 4th generation EGFR-TKI ‘JIN-A02’ at the ESMO (Free ESMO Whitepaper) 2022, that showed high potency of JIN-A02 against both cis and trans isomers of C797S mutation (Press release, J INTS BIO, SEP 15, 2022, View Source;esmo-2022-301625927.html [SID1234619600]). This year’s edition of the European Society for Medical Oncology Congress (ESMO 2022) was held in Paris, France from 9th to 13th September.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Professor Cho, Byoung Chul, poster presentation of further preclinical data of its Novel Oral 4th Generation EGFR-TKI ‘JIN-A02’ at the 2022 European Society for Medical Oncology Congress in Paris, France (ESMO 2022)
Professor Cho, Byoung Chul, poster presentation of further preclinical data of its Novel Oral 4th Generation EGFR-TKI ‘JIN-A02’ at the 2022 European Society for Medical Oncology Congress in Paris, France (ESMO 2022)
Even though EGFR-TKI have improved treatment outcomes of patients with EGFR mutant NSCLC, resistance inevitably emerges with disease progression and often with CNS metastasis. C797S mutation is one of the most common on-target resistance mutation after the use of 3rd generation TKIs such as Osimertinib. ‘JIN-A02’ is a novel 4th generation EGFR-TKI, which is highly selective and potent against C797S double and triple mutations, with high BBB penetrance and intracranial efficacy.

The allelic context in which this C797S mutation is acquired, cis or trans isomers, have significant implications for treatment outcomes. This is especially so when C797S positive tumor are in cis form together with T790M mutation. In such situations, there are no available treatments.

J INTS BIO believes that JIN-A02 will be pivotal in the treatment of patients with EGFR mutant NSCLC harboring C797S double or triple mutations, regardless of allelic context. And the company is expecting the Phase I/IIa clinical study for JIN-A02 to start recruitment before the end of this year.

On September 15, 2022 Caravan Biologix, Inc., a developer of novel cell-derived vesicle (CDV) therapeutics targeting specific cancers and gene therapy-related diseases, reported that it has entered into an agreement with Seoul-based MDimune Inc. to apply their nanovesicle technology to enhance the effects of chimeric antigen receptor-expressing natural killer (CAR-NK) cells on various solid tumor cancers. Research findings should provide important insights as Caravan continues to advance the development of its proprietary Mini-CAR platform for stand-alone therapeutics and whole-cell CAR co-therapies (Press release, Caravan Biologix, SEP 15, 2022, View Source [SID1234619599]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are pleased to establish this collaboration with MDimune, a developer of bio-extrusion and delivery," stated Tom Malcolm, Ph.D., CSO and Founder of Caravan Biologix. "Findings from this collaboration will support our mini-CAR-NK CDV program, which focuses on treating primary liver carcinomas and other human diseases."

Seung Wook Oh, Ph.D., Chief Scientific Officer of MDimune added, "We are thrilled to launch this collaboration with Caravan Biologix. By joining the advanced mini-CAR-NK technology of Caravan and the enormous therapeutic potential of our vesicle technology, we hope to be able to demonstrate that our BioDrone platform can significantly escalate the therapeutic promise of current CAR-T/NK-based therapies."

"The signing of this agreement with Caravan Biologix is another important corporate milestone for MDimune. I am very pleased that our highly innovative and detail-oriented analytical approach to produce the highest quality of cell-derived vesicles at low cost has convinced Caravan Biologix to work with us on such an important endeavor to effectively treat patients with solid tumor cancers. It is clear that new solutions are needed to properly address the complexities associated with traditional whole cell CAR therapeutics," stated Dr. Oh.

On September 15, 2022 Onco360, the nation’s leading independent Specialty Pharmacy, reported that it has been selected by Servier Pharmaceuticals to be a specialty pharmacy partner for TIBSOVO (ivosidenib), which is an isocitrate dehydrogenase-1 (IDH1) inhibitor indicated for patients with a susceptible IDH1 mutation as detected by an FDA-approved test with newly diagnosed acute myeloid leukemia (AML) in combination with azacitidine or as monotherapy in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy, relapsed or refractory AML, or locally advanced or metastatic cholangiocarcinoma that has been previously treated (Press release, Onco360, SEP 15, 2022, View Source [SID1234619597]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Onco360 is honored to partner with Servier Pharmaceuticals and become a specialty pharmacy provider for TIBSOVO patients," said Benito Fernandez, Chief Commercial Officer, Onco360. "We are dedicated to supporting the highly specialized needs of patients battling IDH1-mutant AML or cholangiocarcinoma across the United States."

According to the National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) program, it is estimated that 20,050 new cases of AML will be diagnosed in 2022 in the United States with a corresponding 11,540 deaths in 2022. Approximately 6-9% of AML cases are found to have IDH1 mutations. The median age at the time of initial AML diagnosis is 68 years. The five-year overall survival for AML patients is only 30.5%.1,2

According to the American Cancer Society, approximately 8,000 patients are diagnosed with cholangiocarcinoma, a bile duct cancer, on an annual basis in the United States. Approximately 13% of cholangiocarcinoma cases are found to have IDH1 mutations. The average age at the time of initial cholangiocarcinoma diagnosis is 70-72 years old dependent upon tumor location. The five-year overall survival for cholangiocarcinoma patients, regardless of cancer stage, is only 9-10% dependent upon tumor location.3,4

TIBSOVO is commercialized by Servier Pharmaceuticals. Please see the full prescribing information for TIBSOVO at Tibsovopro.com.