On March 30, 2021 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the U.S. Food and Drug Administration (FDA) approved a revised label for Vyxeos (daunorubicin and cytarabine) to include a new indication to treat newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in pediatric patients aged one year and older (Press release, Jazz Pharmaceuticals, MAR 30, 2021, View Source [SID1234577388]). The approval of Vyxeos for this indication is supported by safety data from two single-arm trials: AAML1421, conducted by the Children’s Oncology Group (COG) and CPX-MA-1201, conducted by Cincinnati Children’s Hospital (CCH) and evidence of effectiveness from an adequate and well-controlled study in adults.

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"At Jazz Pharmaceuticals, we believe all patients living with complex conditions deserve solutions, and work diligently to expand the science behind our therapies to ensure the greatest number of patients can benefit from our medicines," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development and chief medical officer of Jazz Pharmaceuticals. "While pediatric patients represent a relatively small percentage of total AML patients, there is a critical need for more effective therapies in this setting. With the expansion of the Vyxeos label to include the pediatric population, Jazz demonstrates our continued commitment to broadening our cancer research and focusing on the people for whom we can have the greatest impact."

Safety and pharmacokinetics of Vyxeos in children and young adults were established in two clinical studies that enrolled patients with AML or relapsed/refractory hematologic malignancies. Thirty-eight pediatric patients aged one to 21 years of age with AML in first relapse were enrolled in the Phase 1/2 AAML1421 study conducted by COG, and 27 patients aged one to 19 years with relapsed/refractory hematologic malignancies were enrolled in the Phase 1 CPX-MA-1201 study conducted by CCH. Both studies found no differences in the safety profile based on age. 1 The use of Vyxeos for this indication is supported by evidence of effectiveness from study CPX351-301 in adult patients.

Vyxeos has a Boxed Warning as it cannot be substituted with other daunorubicin and/or cytarabine-containing products. In the Phase 3 study, the most common adverse reactions (incidence ≥ 25%) were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders and vomiting.2

"The expansion of the Vyxeos label to include children is a welcome and necessary advancement in support of some of our most vulnerable patients," said Dr. Edward Anders Kolb, M.D., director of the Center for Cancer and Blood Disorders at Nemours/Alfred I. DuPont Hospital for Children and chair of myeloid disease committee at COG. "Jazz has been a wonderful partner in pediatric drug development and we are grateful for the continued work being done to provide safe and effective therapies for children."

About Vyxeos (daunorubicin and cytarabine)
Vyxeos is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor, that is indicated for the treatment of newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in adults and pediatric patients 1 year and older. For more information about Vyxeos in the United States, please visit View Source

More information about Vyxeos, including Full Prescribing Information, BOXED Warning and Medication Guide, is available here.

Important Safety Information
WARNING: VYXEOS has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute VYXEOS for other daunorubicin and/or cytarabine-containing products.

VYXEOS should not be given to patients who have a history of serious allergic reaction to daunorubicin, cytarabine, or any of its ingredients.

VYXEOS can cause a severe decrease in blood cells (red and white blood cells and cells that prevent bleeding, called platelets) which can result in serious infection or bleeding and possibly lead to death. Your doctor will monitor your blood counts during treatment with VYXEOS. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding.

VYXEOS can cause heart-related side effects. Tell your doctor about any history of heart disease, radiation to the chest, or previous chemotherapy. Inform your doctor if you develop symptoms of heart failure such as:

shortness of breath or trouble breathing
swelling or fluid retention, especially in the feet, ankles, or legs
unusual tiredness
VYXEOS may cause allergic reactions including anaphylaxis. Seek immediate medical attention if you develop signs and symptoms of anaphylaxis such as:

trouble breathing
severe itching
skin rash or hives
swelling of the face, lips, mouth, or tongue
VYXEOS contains copper and may cause copper overload in patients with Wilson’s disease or other copper-processing disorders.

VYXEOS can damage the skin if it leaks out of the vein. Tell your doctor right away if you experience symptoms of burning, stinging, or blisters and skin sores at the injection site.

VYXEOS can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Do not breastfeed while receiving VYXEOS. Females and males of reproductive potential should use effective contraception during treatment and for 6 months following the last dose of VYXEOS.

The most common side effects were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting.

Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Jazz Pharmaceuticals at 1-800-520-5568.

About AML
Acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets.3 It can sometimes spread to other parts of the body including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles.4 AML is a relatively rare disease representing 1.1 percent of all new cancer cases.5 It is estimated that more than 19,500 people will be diagnosed with AML in the United States this year with the potential for more than 11,000 people to die from the disease.6 The median age at diagnosis is 68 years old,6 with rising age associated with a progressively worsening prognosis.7 AML in children makes up a small portion of the overall AML population (4.5% occurs in patients < 20 years old). Further, t-AML and AML-MRC in pediatric AML are very rare subtypes of this group accompanied by poor prognosis.5 There is also a reduced tolerance for intensive chemotherapy as patients age.8 AML has the lowest survival rate of any other form of leukemia.5 Patients with newly diagnosed therapy-related AML or AML with myelodysplasia-related changes may have a particularly poor prognosis.9-11 A hematopoietic stem cell transplant may be a curative treatment option for patients.12

On March 30, 2021 Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, reported that the Center for Drug Evaluation (CDE) of China’s Nation Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) for Parsaclisib (IBI376) for the treatment of patients with relapsed/refractory follicular lymphoma (FL) (Press release, Innovent Biologics, MAR 30, 2021, View Source [SID1234577387]). IBI376 is a phosphatidylinositol 3-kinase delta (PI3Kδ) inhibitor originally discovered by Incyte. Innovent owns the rights to develop and commercialize Parsaclisib in greater China.

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NMPA grants Breakthrough Therapy Designation to new medicines that are intended to treat serious conditions and where clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint. The BTD designation for Parsaclisib is based on the results observed in an ongoing Phase 2 study for the treatment of adults with relapsed or refractory FL being conducted in China (CTR2019239). Parsaclisib is currently in Phase 2 pivotal trials for the treatment of adults with relapsed or refractory follicular or with marginal zone lymphoma.

In clinical studies, Parsaclisib has demonstrated potent and rapid anti-lymphoma activity with promising safety, efficacy, and persistence. Preliminary clinical results of the CITADEL-203 study in relapsed or refractory FL presented at the 62st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) held in 2020 highlighted the safety profile, efficacy, and the durability of response of Parsaclisib (Abstract 2935). Study results showed that in the dosing group (N=95), Parsaclisib monotherapy achieved an objective response rate of 75%, with median PFS of 15.8 months in relapsed or refractory FL patients.

Dr. Hui ZHOU, Vice President of Medical Science and Strategy Oncology of Innovent, stated: "The breakthrough therapy designation from NMPA indicated that Parsaclisib possesses great potential in treating relapsed or refractory follicular lymphoma and we are hopeful that this product can help benefit more patients in the future."

About Parsaclisib (IBI376)

Parsaclisib is an investigational novel oral inhibitor of phosphatidylinositol 3-kinase delta (PI3Kδ) isoforms. PI3Kδ is an important anticancer target implicated in malignant B-cell growth, survival and proliferation which has demonstrated potency and selectivity in preclinical studies and has potential therapeutic utility in the treatment of patients with hematologic malignancies such as lymphoma. Parsaclisib is currently under evaluation as a monotherapy in several ongoing Phase 2 trials as treatment for non-Hodgkin lymphomas (follicular, marginal zone and mantle cell); and autoimmune hemolytic anemia. Pivotal trials of Parsaclisib in combination with ruxolitinib for the treatment of patients with myelofibrosis are also underway; and there are plans to initiate a trial to evaluate Parsaclisib in combination with tafasitamab for non-Hodgkin lymphoma (NHL) and chronic lymphocytic leukemia (CLL).

In December 2018, Innovent and Incyte entered into a strategic collaboration for three clinical-stage product candidates discovered and developed by Incyte, including Parsaclisib (PI3Kδ inhibitor). Under the terms of the agreement, Innovent has received the rights to develop and commercialize Parsaclisib and two other assets in Mainland China, Hong Kong, Macau and Taiwan.

About Follicular Lymphoma

Follicular lymphoma (FL) is a B-cell cancer that originates from the uncontrolled division of specific types of B-cells known as centrocytes and centroblasts. Although it is classified as indolent lymphoma, and the current immunochemotherapy has achieved good efficacy, it still often relapses following by aggressive diseases, which may lead to death within 1 to 2 years. There is an unmet medical need for treatment options for recurrent or refractory follicular lymphoma.

About Breakthrough Therapy Designation

In recent years, in order to encourage innovation to address unmet clinical needs, China has established a rapid drug review and approval pathway. A Breakthrough Therapy Designation (BTD) is intended to facilitate and expedite development and review of an investigational drug to treat serious disease or condition when preliminary clinical evidence indicates that the drug has demonstrated substantial improvement over current therapies. The BTD will not only qualify a drug candidate to receive status for rapid review by the CDE, but also allow the sponsor to obtain timely advice and communications from the CDE to accelerate the approval and launch in order to address the unmet clinical needs of patients at an accelerated pace.

On March 30, 2021 ImmuneOncia Therapeutics, Inc., a clinical-stage, immuno-oncology company in South Korea, and 3D Medicines, Inc., a China-based biopharmaceutical company developing next-generation immuno-oncology drugs, reported an exclusive license agreement for the development, manufacture and commercialization of IMC-002, ImmuneOncia’s monoclonal antibody against CD47 (Press release, ImmuneOncia Therapeutics, MAR 30, 2021, View Source [SID1234577386]). The agreement includes uses of IMC-002 for oncology indication as a monotherapy or combination agent in the Territory of Greater China (Mainland China, Hong Kong, Macau, and Taiwan). ImmuneOncia will retain rights of IMC-002 in the rest of the world including the United States, European Union, and Japan.

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Under the terms of the agreement, ImmuneOncia will receive an upfront payment of $8 million from 3D Medicines. Additionally, ImmuneOncia is eligible to receive up to $462.5 million upon the achievements of all future development and commercial milestones, plus tiered royalties up to double-digits on annual net sales of IMC-002 in Greater China. In exchange, 3D Medicines will receive rights to develop, manufacture and commercialize IMC-002 in Greater China. 3D Medicines is planning to file IND to NMPA in China this year.

"3D Medicines is an established leader in oncology, with a track record of successfully developing in-licensed oncology programs." said Yun Jeong Song, Chief Executive Officer of ImmuneOncia. "We are confident that 3D Medicines is the ideal partner as we enter into our collaboration and look forward to accelerating our delivery of IMC-002 to patients in Greater China."

"We are very pleased to enter into this exclusive collaboration with ImmuneOncia" said John Gong, M.D., Ph.D., Chairman and Chief Executive Officer of 3D Medicines. "We believe that IMC-002, used in combination with existing standard of care therapeutics or Envafolimab, an innovative subcutaneous PD-L1 antibody which we have just filed for marketing approval in China, could alter the treatment paradigm across various tumor types."

YAFO Capital (Shanghai) Co. Ltd. acted as financial advisor on this transaction for ImmuneOncia.

About IMC-002

IMC-002 is a fully human IgG4 monoclonal antibody designed to block the CD47–SIRPα interaction in order to promote the phagocytosis of cancer cells by macrophages. According to its non-clinical results, it binds to human CD47 with an optimal affinity that maximizes efficacy without binding to RBCs or causing anemia which is often seen in other CD47 blocking agents under development. For more information about the Phase 1 clinical trial, visit clinicaltrials.gov, identifier number NCT04306224.

On March 30, 2021 Everest Medicines (HKEX 1952.HK), a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products that address critical unmet medical needs for patients in Greater China and other parts of Asia, reported that the China National Medical Products Administration (NMPA) approved its Clinical Trial Application (CTA) for a Phase 2 basket trial of Trodelvy (sacituzumab govitecan-hziy) in a variety of cancers with high TROP-2 expression (Press release, Everest Medicines, MAR 30, 2021, View Source [SID1234577385]).

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The Phase 2 single arm, multiple-cohorts basket trial will evaluate sacituzumab govitecan-hziy in 180 patients with relapse/refractory esophageal squamous cell carcinoma, gastric cancer, and cervical cancer at selected sites in China. The incidence of these indications is higher in China/Asia than Western countries, and there are very limited treatment options in later line settings, represent a significant unmet medical need in China and Asia.

"As of 2019, the incidence of cancers with TROP-2 expression was more than 3.5 million, accounting for approximately 78% of all cancer occurrences in China," said Yang Shi, Chief Medical Officer for Oncology at Everest Medicines. "Sacituzumab govetican-hziy’s unique TROP-2 directed antibody and topoisomerase inhibitor drug conjugate mechanism of action, along with its robust set of data in other TROP-2 expressing cancers suggest that it may be effective in a broad range of tumors. We look forward to advancing this important basket study as we work to expand potential indications of this novel therapy across a variety of cancers with high unmet medical need."

About Trodelvy (sacituzumab govitecan-hziy)

Trodelvy (sacituzumab govitecan-hziy) is a first-in-class, antibody-drug conjugate (ADC) directed at TROP-2, a membrane antigen that is over-expressed in many common epithelial cancers. It is indicated in the U.S. for the treatment of adult patients with metastatic triple-negative breast cancer (mTNBC) who have received at least two prior therapies for metastatic disease and was granted accelerated approval by the U.S. Food and Drug Administration for this patient population in April 2020, based on overall response rate and duration of response results in a Phase 1/2 study.

Under a licensing agreement with Gilead Sciences, Inc., Everest Medicines has exclusive rights to develop, register, and commercialize sacituzumab govitecan-hziy for all cancer indications in Greater China, South Korea, and certain Southeast Asian countries.

On March 30, 2021 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company enabled by its proprietary synthetic lethality approach to the discovery and development of novel therapeutics, reported it will host a virtual Investor Day on Thursday, April 8, 2021, from 10:30 a.m. to 12:00 p.m. Eastern Time (Press release, Repare Therapeutics, MAR 30, 2021, View Source [SID1234577382]). Repare Therapeutics’ executive management team will be joined by two distinguished physicians:

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Carol Aghajanian, MD, Chief, Gynecologic Medical Oncology Service, Professor of Medicine, Weill Cornell Medical College, Memorial Sloan Kettering Cancer Center, New York, NY
Timothy Yap, MBBS, PhD, FRCP, Medical Director, Institute for Applied Cancer Science, Associate Professor, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, MD Anderson Cancer Center, Houston, TX
The event will focus on RP-6306, a potent and selective inhibitor of a novel target that is synthetic lethal with CCNE-1 amplification. The Company expects to begin a Phase 1 clinical trial in the second quarter of 2021, one quarter earlier than prior guidance.

To access the event virtual event, please dial (833) 638-9655 (U.S. and Canada) or (602) 585-9856 (international) at least 10 minutes prior to the start time and refer to conference ID 1093819. A live video webcast will be available in the Investor section of the Company’s website at View Source A webcast replay will also be available on the corporate website at the conclusion of the call.

About RP-6306

RP-6306 is the result of Repare’s proprietary drug discovery program for tumors with genetic alterations characterized by CCNE1 amplification, which typically do not respond well to platinum or PARP inhibitor treatment. Through Repare’s SNIPRx screen campaign for targets that are SL with CCNE1 amplification, the Company has identified and validated a novel SL gene that is believed to have the characteristics of a therapeutic target. Subsequently, the Company developed novel and selective inhibitors against the target that have repeatedly demonstrated compelling anti-tumor activity and announced the advancement of a clinical candidate for this potential first-in-class program. Repare anticipates initiating a Phase 1 clinical trial for RP-6306 in the second quarter of 2021. This trial is expected to enroll patients suffering from recurrent tumors characterized by CCNE1 amplification and other genomic alterations predicted to be sensitive to RP-6306. The primary objective of the trial is to establish the recommended Phase 2 dose and schedule for RP-6306 for further studies as monotherapy and to assess preliminary safety in patients. An additional trial is planned to evaluate combination with approved anticancer agents.