On June 24, 2022 Kyowa Kirin Co., Ltd. (TSE: 4151, Kyowa Kirin) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended that CRYSVITA (burosumab) be approved for the treatment of FGF23-related hypophosphataemia in Tumour-Induced Osteomalacia (TIO) associated with phosphaturic mesenchymal tumours (PMTs) that cannot be curatively resected or localised in children and adolescents aged 1 to 17 years and in adults (Press release, Kyowa Hakko Kirin, JUN 24, 2022, View Source [SID1234616252]).1 CRYSVITA is also already licensed for use in the rare disease X-Linked Hypophosphataemia (XLH), for children and adolescents between 1 and 17 years of age with radiographic evidence of bone disease, and in adults.2

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Also known as oncogenic osteomalacia, TIO is an acquired disorder caused by typically small, slow-growing, benign PMTs.3,4 It is a rare condition with less than 1000 cases reported in the medical literature,4 which mainly affects adults and with a mean onset age of 40 – 45 years.3,5 TIO is associated with progressive and debilitating musculoskeletal deficits,6,7 ultimately having a detrimental impact on ability to perform daily activities, as well as on physical and social wellbeing.8

A cure for TIO can be achieved with complete surgical resection of the causative tumour(s), however, surgical resection is not always possible due to the location and difficulty in detecting tumours.3,5 TIO may recur and persist following incomplete or unsuccessful surgical resection.9

If approved by the European Commission (EC), CRYSVITA will be the first biologic treatment available to European patients with TIO, which blocks the action of FGF23, restoring phosphate homeostasis.2,10

"Being diagnosed with a rare condition, like TIO, does present many challenges for patients and treating physicians, including the diagnostic process and the current lack of specific therapies. The unmet need for people living with TIO is clear, so this positive CHMP opinion is a very important step forward for those who cannot be cured by tumour removal and for the healthcare professionals supporting them," said Professor Ralf Oheim, Department of Osteology and Biomechanics, University Medical Center Hamburg.

"For people diagnosed with TIO in Europe, we are a step closer to being able to deliver the first biologic treatment for those who cannot undergo surgical removal of tumours," said Abdul Mullick, President of Kyowa Kirin International. "This positive CHMP opinion is a much welcomed milestone for CRYSVITA, which is currently approved for use in Europe in adults and children with X-Linked Hypophosphataemia. I’m proud that Kyowa Kirin International is helping those living with TIO, as part of our extensive work in supporting those with rare diseases gain access to life changing therapies for their diseases."

Administered by a subcutaneous injection, CRYSVITA is a recombinant fully human monoclonal antibody that binds to and inhibits the activity of FGF23, restoring phosphate homeostasis.2 The efficacy and safety of CRYSVITA have been demonstrated in two Phase II clinical trials published in the disease area of TIO.11,12 CRYSVITA has been approved for clinical use in X-Linked Hypophosphataemia (XLH) across the European Union (EU) and Great Britain (GB) since 2018, and in this indication is presently approved for use in children and adolescents aged 1 and 17 years of age with radiographic evidence of bone disease, and in adults.2

The EC will review the CHMP recommendation and a final decision on the expansion of the recommended use for CRYSVITA for the treatment of FGF23-related hypophosphataemia in TIO associated with phosphaturic mesenchymal tumours (PMTs) that cannot be curatively resected or localised in children and adolescents aged 1 to 17 years and in adults is expected in the coming months. This means that use of CRYSVITA in this TIO indication is not currently approved in the EU or GB.

▼This medicinal product is subject to additional monitoring.

About Tumour-Induced Osteomalacia (TIO)
TIO is characterised by chronic hypophosphataemia caused by tumour(s) secreting excess fibroblast growth factor 23 (FGF23),3 which can lead to issues such as decreased intestinal phosphate absorption and compromised vitamin D activation.3,4

The most common signs and symptoms include bone pain, difficulty walking, pathological fractures, height loss and muscle weakness.6 In TIO, muscle weakness and pain severely interfere with physical functioning, including standing up without assistance, walking and ability to work.8 The pain in TIO also severely interferes with mood and moderately interferes with enjoyment of life for those living with it.8

TIO diagnosis is often missed and/or delayed and testing serum phosphate levels is important for diagnosis.3 The only cure in TIO is complete removal of the causative tumour(s).3 Pharmacological treatment should be considered in TIO cases where tumour(s) cannot be curatively resected or localised.3 Restoring phosphate homeostasis is essential to improve the health of people living with TIO.3

About CRYSVITA (burosumab) in TIO
CRYSVITA (burosumab) was created and developed by Kyowa Kirin and is a recombinant fully human monoclonal antibody that binds to and inhibits the activity of FGF23.2 CRYSVITA blocks the action of FGF23, restoring phosphate homeostasis.2

The efficacy and safety of CRYSVITA have been demonstrated in two Phase 2 clinical trials published in the disease area of TIO.11,12 CRYSVITA was well-tolerated and demonstrated an acceptable safety profile.11,12

CRYSVITA is presently indicated for the treatment of XLH in children and adolescents aged 1 to 17 years with radiographic evidence of bone disease, and in adults.2 If approved by the European Commission, CRYSVITA would be indicated for the treatment of FGF23-related hypophosphataemia in TIO associated with PMTs that cannot be curatively resected or localised in children and adolescents aged 1 to 17 years and in adults.1 CRYSVITA is given as a subcutaneous injection, every 4 weeks in adults and every 2 weeks in children and adolescents aged 1 to 17 years.2

CRYSVITA is currently approved for use in the treatment of TIO in a number of countries, including the United States13 and Japan.14

Kyowa Kirin and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE: Ultragenyx) have been collaborating in the development and commercialisation of CRYSVITA globally, based on the collaboration and licence agreement between Kyowa Kirin and Ultragenyx.

On June 24, 2022 The Janssen Pharmaceutical Companies of Johnson & Johnson reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending approval of a new treatment option with IMBRUVICA (ibrutinib) in an oral fixed-duration combination with venetoclax (I+V) for adults with previously untreated chronic lymphocytic leukaemia (CLL) (Press release, Johnson & Johnson, JUN 24, 2022, View Source [SID1234616251]).

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Outcomes for patients with CLL have improved in the last decade with the advent of oral therapies that target the underlying disease biology.3 This provides the opportunity to combine these novel treatments for an effective and convenient approach that results in deep responses with time-limited therapy.1 If approved, I+V will be the first all-oral, once daily, fixed-duration combination treatment with a Bruton’s tyrosine kinase (BTK) inhibitor for first-line treatment of patients with CLL.

"With this innovative treatment regimen, healthcare professionals would have the flexibility to use ibrutinib either in a fixed-duration combination or as a continuous therapy, helping them to better tailor frontline CLL therapy based on patients’ individual needs," said Edmond Chan, MBChB, M.D. (Res), EMEA Therapeutic Area Lead Haematology, Janssen-Cilag Limited. "This recommendation brings us one step closer to European Commission (EC) approval and to providing patients with an all-oral, once daily, fixed-duration regimen, which until this point has not been available with the BTK inhibitor class of treatments."

The CHMP positive opinion is supported by data from the pivotal Phase 3 GLOW study (NCT03462719), which demonstrated that I+V was superior to chlorambucil-obinutuzumab with respect to the primary endpoint, progression-free survival (PFS), in elderly or unfit patients with CLL (PFS hazard ratio [HR]: 0.216; 95 percent confidence interval [CI], 0.131 to 0.357; P<0.001).1 It is also supported by the fixed-duration cohort of the Phase 2 CAPTIVATE study (NCT02910583) which evaluated I+V in 159 patients with previously untreated CLL who were 70 years or younger, including patients with high-risk CLL disease.2

Data from these studies were recently published in NEJM Evidence, and Blood, respectively,1,4 and featured as oral presentations at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Congress. Secondary analyses from GLOW, with additional study follow-up, were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2021 Annual Meeting, and additional data from the CAPTIVATE study including clinical outcomes at three years and evidence of immune restoration post-treatment were recently presented at the EHA (Free EHA Whitepaper) 2022 Congress.

Updated data for both studies showed the safety profile of the I+V regimen was consistent with known safety profiles of ibrutinib and venetoclax.1,4 In the GLOW study, the most common treatment-emergent adverse events (TEAEs) were diarrhoea (50.9 percent) and neutropenia (41.5 percent) in the ibrutinib-venetoclax arm, and neutropenia (58.1 percent) and infusion-related reactions (29.5 percent) in the chlorambucil-obinutuzumab arm.1 TEAEs of Grade 3 or greater occurred in 80 (75.5 percent) and 73 (69.5 percent) of patients in the ibrutinib-venetoclax and chlorambucil-obinutuzumab arms, respectively.1 In the CAPTIVATE fixed-duration cohort, the most common TEAEs were diarrhoea (62 percent), nausea (43 percent), neutropenia (42 percent), and arthralgia (33 percent) and primarily were Grade 1 or 2 in severity.4 The most common Grade 3/4 AEs were neutropenia (33 percent), hypertension (6 percent), and decreased neutrophil count (5 percent).4

"The promising data from GLOW and CAPTIVATE reinforce the distinct and complementary modes of action between ibrutinib and venetoclax, and the potential of this combination regimen to provide treatment-free remissions for patients," said Craig Tendler, M.D., Global Head of Late Development, Diagnostics & Medical Affairs, Hematology & Oncology, Janssen Research & Development, LLC. "The positive CHMP opinion for I+V is testament to our commitment and continued leadership in developing innovative and convenient treatment regimens that may help improve outcomes for people living with complex blood cancers like CLL."

#ENDS#

About Ibrutinib
Ibrutinib is a once-daily oral medication that is jointly developed and commercialised by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company.5 Ibrutinib blocks the Bruton’s tyrosine kinase (BTK) protein, which is needed by normal and abnormal B-cells, including specific cancer cells, to multiply and spread.6 By blocking BTK, ibrutinib may help move abnormal B-cells out of their nourishing environments and inhibits their proliferation.7

Ibrutinib is approved in more than 100 countries and has been used to treat more than 250,000 patients worldwide.8 There are more than 50 company-sponsored clinical trials, including 18 Phase 3 studies, over 11 years evaluating the efficacy and safety of ibrutinib.1,9 In October 2021, ibrutinib was added to the World Health Organization’s Model Lists of Essential Medicines (EML), which refer to medicines that address global health priorities and which should be available and affordable for all.10

Ibrutinib was first approved by the European Commission (EC) in 2014, and approved indications to date include:1

As a single agent or in combination with rituximab or obinutuzumab for the treatment of adult patients with previously untreated CLL
As a single agent or in combination with bendamustine and rituximab (BR) for the treatment of adult patients with CLL who have received at least one prior therapy
As a single agent for the treatment of adult patients with relapsed or refractory (RR) mantle cell lymphoma (MCL)
As a single agent for the treatment of adult patients with Waldenström’s macroglobulinaemia (WM) who have received at least one prior therapy, or in first line treatment for patients unsuitable for chemo-immunotherapy, and in combination with rituximab for the treatment of adult patients with WM
For a full list of side effects and information on dosage and administration, contraindications and other precautions when using ibrutinib please refer to the Summary of Product Characteristics for further information.

About the CAPTIVATE study
The Phase 2 CAPTIVATE study evaluated previously untreated adult patients with CLL who were 70 years or younger, including patients with high-risk disease, in two cohorts: an MRD-guided cohort (N=164; median age, 58 years) and a fixed-duration cohort (N=159; median age, 60 years).4 Patients in the fixed-duration cohort received 3 cycles of ibrutinib lead-in then 12 cycles of ibrutinib plus venetoclax (oral ibrutinib [420 mg/d]; oral venetoclax [5-week ramp-up to 400 mg/d]) and the primary endpoint was complete response (CR) rate.4

About the GLOW study
The Phase 3 GLOW study (N=211; median age, 71 years) is a randomised, open-label trial which evaluated the efficacy and safety of first-line, fixed-duration I+V vs. Clb+O in elderly patients (≥65 years of age) with CLL/SLL, or patients ages 18-64 with a cumulative illness rating scale (CIRS) score of greater than six or creatinine clearance less than 70 mL/min, without del(17p) or known TP53 mutations.1 Patients in the study were randomised to receive either I+V (n= 106) or Clb+O (n=105).1

About Chronic Lymphocytic Leukaemia
Chronic lymphocytic leukaemia (CLL) is typically a slow-growing blood cancer of the white blood cells.11 The overall incidence of CLL in Europe is approximately 4.92 cases per 100,000 persons per year and is about 1.5 times more common in men than in women.12 CLL is predominantly a disease of the elderly, with a median age of 72 years at diagnosis.13

While patient outcomes have dramatically improved in the last few decades, the disease is still characterised by consecutive episodes of disease progression and the need for therapy.3 Patients are often prescribed multiple lines of therapy as they relapse or become resistant to treatments.14

On June 23, 2022 Turning Point Therapeutics, Inc. (the "Company") reported that it entered into a strategic collaboration agreement (the "Agreement") with The University of Texas M.D. Anderson Cancer Center ("MD Anderson") (Filing, 8-K, Turning Point Therapeutics, JUN 24, 2022, View Source [SID1234616249]). Under the terms of the Agreement, the Company will provide funding and support for preclinical and clinical studies to be conducted by MD Anderson in several solid tumors, including non-small cell lung cancers, gastrointestinal malignancies and endocrine cancers (the "Studies").

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The initial focus of the Studies will be the Company’s lead drug candidate, repotrectinib, a next-generation kinase inhibitor targeting the ROS1 and NTRK oncogenic drivers of non-small cell lung cancer and advanced solid tumors that is currently being studied in a registrational Phase 1/2 study (TRIDENT-1). The Studies will also include two additional drug candidates, elzovantinib (TPX-0022), a kinase inhibitor targeting MET, CSF1R and SRC, which is being studied in a Phase 1 trial of patients with advanced solid tumors harboring genetic alterations in MET (SHIELD-1), and TPX-0131, a next-generation ALK inhibitor, which is being studied in a Phase 1/2 trial of previously treated patients with ALK-positive advanced or metastatic non-small cell lung cancer (FORGE-1).

Pursuant to the Agreement, the Company has agreed to provide total funding in an amount of $10.0 million for the performance of the Studies, such funding to be provided in increments of $1.0 million on a twice-yearly basis. The Studies will be overseen by a joint steering committee, which will provide technical, scientific, clinical and regulatory guidance, discuss and approve study budgets, and monitor the progress of the Studies.

Under the terms of the Agreement, all Inventions will be solely owned by the Company, except with respect to the use of Proprietary Materials of MD Anderson that does not involve the use of the Company’s Study Drug or Proprietary Materials (each as defined in the Agreement).

The term of the Agreement runs five years or until the Studies are completed, whichever is later, unless terminated earlier in accordance with the terms set forth in the Agreement.

The foregoing description of the material terms of the Agreement does not purport to be complete and is qualified in its entirety by reference to the complete text of the Agreement, a copy of which the Company intends to file, with confidential terms redacted, with the Securities and Exchange Commission as an exhibit to the Company’s Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2022.

On June 24, 2022 TOLREMO therapeutics AG reported that Dr. Karl Schumacher, MD, MHBA, has joined the company as Chief Medical Officer (CMO). Dr. Schumacher will lead the clinical and medical programs of TOLREMO in support of the company’s lead asset TT125-802, that is expected to enter the clinic in early 2023 (Press release, TOLREMO, JUN 24, 2022, View Source [SID1234616248]). He will help in the further advancement of TOLREMO’s platform, addressing the unmet medical need of non-genetic cancer drug resistance.

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«We are delighted to have Karl join TOLREMO’s executive management team in this exciting phase for the company. With more than 10 years of clinical development experience in oncology focusing on combination therapies and drug resistance, he will add tremendous value to our drug development programs» said Dr. Stefanie Flückiger-Mangual, CEO and co-founder of TOLREMO.

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Dr. Schumacher practiced surgical oncology for several years before joining the life sciences industry. Prior to joining TOLREMO, he held various roles in the pharmaceutical industry focusing on drug combinations to prevent resistance to targeted therapies in multiple cancer types. He has been instrumental to the clinical development of targeted and innovative anti-cancer therapies for NSCLC, breast cancer and melanoma. He joins TOLREMO from Merck KGaA where he was Vice President Clinical Development for the MET inhibitor tepotinib (Tepmetkoâ) and the DNA damage response (DDR) inhibitor portfolio.

«I am excited to join TOLREMO at this pivotal phase of the company’s development. I am looking forward to applying my clinical expertise with drug combinations and cancer drug resistance to the company’s clinical development. I am convinced that TT125-802 has a high potential to be developed as a versatile combination partner to a number of targeted therapies» said Dr. Karl Schumacher.

On June 24, 2022 Quest Diagnostics Incorporated (NYSE: DGX), the world’s leading provider of diagnostic information services, reported that it will report second quarter 2022 financial results on Thursday, July 21, 2022, before the market opens (Press release, Quest Diagnostics, JUN 24, 2022, View Source,-2022 [SID1234616247]). It will hold its quarterly conference call to discuss the results beginning at 8:30 a.m. Eastern Time on that day.

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The conference call can be accessed by dialing 888-455-0391 within the U.S. and Canada, or 773-756-0467 internationally, using the passcode: "7895081." The earnings release and live webcast will be posted on www.QuestDiagnostics.com/investor. The company suggests participants dial in approximately 10 minutes before the call.

A replay of the call may be accessed online at www.QuestDiagnostics.com/investor or by phone at 888-566-0439 for domestic callers or 203-369-3045 for international callers; no passcode is required. Telephone replays will be available from approximately 10:30 a.m. Eastern Time on July 21, 2022 until midnight Eastern Time on August 4, 2022.

Anyone listening to the call is encouraged to read the company’s periodic reports on file with the Securities and Exchange Commission, including the discussion of risk factors and historical results of operations and financial condition in those reports.