Race Receives Governance Approval for Extramedullary AML & MDS Human Trial

On May 12, 2022 Race Oncology Limited ("Race") reported it has received Research Governance Office (RGO) approval from the Calvary Mater Newcastle Hospital for its open label clinical trial of Zantrene (bisantrene dihydrochloride) in patients with extramedullary Acute Myeloid Leukaemia (AML) or high-risk Myelodysplastic Syndrome (MDS). Human ethics approval for this trial has been granted (ASX Announcement: 5 April 2022) (Press release, Race Oncology, MAY 12, 2022, View Source [SID1234614468]).

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Representatives of the Race Oncology clinical team, the contract research organisation Paraxel, and associated clinical teams of the Calvary Mater Hospital are scheduled to meet for site initiation and training on the May 31, 2022. Completion of this site training will enable the first patient to be recruited into the trial.

This open label Phase 1 trial with a dose expansion Phase 2 stage will recruit up to 60 patients with extramedullary AML or MDS using a two-stratum (arm) design at trial sites in Australia and Europe (ASX Announcement: November 1, 2021).

JCRI – ABTS and USMI to Present a Two-year Follow-up of Phase I Clinical Trial Using Cold Atmospheric Plasma for the Treatment of Solid Tumors at Israeli Society of Surgical Oncology

On May 12, 2022 Jerome Canady Research Institute for Advanced and Biological Technological Sciences (JCRI-ABTS) and US Medical Innovations, LLC (USMI) reported that Jerome Canady MD, Chief Science Officer at JCRI-ABTS and a Surgical Oncologist at Holy Cross Hospitals Silver Spring/Germantown, MD will present the results of a Two-year follow up, "Phase I Clinical Trial of Canady Helios Cold Atmospheric Plasma (CHCP) Treatment for Patients with Advanced Stage IV Metastatic and Recurrent Solid Tumors: A Novel Potential 4th Treatment Arm for Cancer" at The Biannual Conference of the Israeli Society of Surgical Oncology (ISSO), May 11-13, 2022 in Haifa, Israel (Press release, JCRI-ABTS, MAY 12, 2022, View Source;ABTS-and-USMI-to-Present-a-Two-year-Follow-up-of-Phase-I-Clinical-Trial-Using-Cold-Atmospheric-Plasma-for-the-Treatment-of-Solid-Tumors-at-Israeli-Society-of-Surgical-Oncology [SID1234614439]).

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Dr. Canady stated, "Metastatic and recurrent Stage IV solid tumors have a poor overall survival (OS) despite the advancement of surgery, chemotherapy, radiation, and immunotherapy. The CHCP Trial was a Phase I, multiple-center, open labelled, prospective controlled trial which enrolled eligible subjects undergoing surgery and intra-operative CHCP treatment. Between March 2020 to April 2021, twenty (20) patients were recruited from Rush University Medical Center Chicago, Illinois and Sheba Medical Center Tel HaShomer, Israel. Patients received intra-operative CHCP treatment at the operative site after the tumor was removed. The primary end point was safety, and the secondary end point was to demonstrate ablation and slowing down tumor growth in cancer patients without damaging surrounding normal biological tissue."

Patients from 26 to 85 years of age (mean age 59) were enrolled in the study. Physiological data was recorded throughout surgery (i.e. blood pressure, pulse, body temperature, End Tidal CO2, and oxygen saturation which demonstrated no significant changes (p > 0.05) during the intra-operative CHCP treatment period). There were no adverse events related to CHCP.

As of May 12, 2022, four patients died of their disease at 3, 4, 8 and 10 months. Kaplan-Meier survival analysis showed that interim OS rate at 26 months was 80.0% (95% confidence interval [CI], 64.3 – 99.6%). Median survival was 14.5 months. Histology of the surgical margins revealed cancer cell death and no damage to normal tissue. Ex vivo culture confirmed reduction of cancer cell growth.

CHCP treatment in combination with surgery for high-risk stage IV solid tumors is safe, induces tumor death and slows down tumor growth in vitro, ex vivo and in cancer patients without damaging non-cancerous tissue. CHCP is potentially a 4th Treatment Arm for Solid Tumor Cancers. (ClinicalTrials.gov identifier: NCT04267575.)

The FDA Phase One Clinical trial was completed April 15, 2021, at Rush University Medical Center (Chicago, IL) and Sheba Medical Center-Tel HaShomer (Ramat Gan, Israel). The Principal Investigators were Professor Steven Gitelis, M.D., and Professor Aviram Nissan, M.D. respectively.

REHEVA BIOSCIENCES ANNOUNCES FIRST PATIENT ENROLLED IN RH324 PHASE I SAFETY TRIAL

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Gracell Biotechnologies to Present Clinical Data on BCMA/CD19 Dual-targeting CAR-T GC012F in RRMM and B-NHL and CD19/CD7 Dual-directed Allogeneic CAR-T GC502 in B-ALL at EHA2022 Congress

On May 12, 2022 Gracell Biotechnologies Inc. ("Gracell" or the "Company",NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, reported the details of three abstracts that it will present at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress (EHA2022 Congress), being held from June 9 – June 12 in Vienna, Austria (Press release, Gracell Biotechnologies, MAY 12, 2022, View Source [SID1234614436]). The abstracts highlight the clinical data from ongoing investigator-initiated trials (IITs) of BCMA/CD19 dual-targeting FasTCAR candidate GC012F in two indications of B-cell non-hodgkin’s lymphoma (B-NHL) and relapsed/refractory multiple myeloma (RRMM), and allogeneic TruUCAR candidate GC502 in B-cell acute lymphoblastic leukemia (B-ALL).

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"We are very excited to share our data for both our FasTCAR candidate GC012F in two indications of RRMM and B-NHL, and allogeneic TruUCAR candidate GC502 in B-ALL at the EHA (Free EHA Whitepaper)2022 Congress," said Dr. Martina Sersch, Chief Medical Officer of Gracell. "The new data, including the expanded indication of GC012F into B-NHL, demonstrates the potential of our platforms and provides further validation. The clinical data of BCMA/CD19 dual-targeting GC012F in the treatment of B-NHL shows promising early results, along with benefits of the next-day manufacturing enabled by the FasTCAR platform. The CD19/CD7 dual-directed CAR-T therapy GC502 is our second allogeneic candidate on our TruUCAR platform, demonstrating the potential wide applicability of the TruUCAR design."

BCMA/CD19 Dual-Targeting FasTCAR-T GC012F for the Treatment of B-NHL

GC012F is an autologous CAR-T therapeutic candidate dual-targeting B cell maturation antigen (BCMA) and CD19. It is developed using Gracell’s proprietary FasTCAR platform which enables next-day manufacturing, and is currently being evaluated in IITs in China including in RRMM and B-NHL. GC012F is the first BCMA/CD19 dual-targeting CAR-T in human trials for B-NHL.

Gracell will present the early results of the first-in-human phase 1 IIT in China evaluating the safety and tolerability of GC012F in B-NHL patients. Three patients who had received a median of two prior lines of therapy were enrolled, all of which presented with bulky disease. As of the February 22, 2022 data cutoff date, the enrolled patients had received one single infusion of GC012F at three different doses of 3.7×104 cells/kg and 2-3×105 cells/kg.

All three patients had achieved a complete response (CR) confirmed by PET- CT at day 28 after GC012F infusion. At 3-month follow-up, both of the two assessable patients had ongoing response. No dose-limiting toxicities were observed and no immune effector cell-associated neurotoxicity syndrome (ICANS) were observed. CRS presented as Grade 1 in two patients and Grade 3 in one patient (duration of two days) with no Grade 4 or 5 events.

Details of the presentation are as follows:

Abstract title: First-in-human study of CD19/BCMA dual-targeting FasTCAR-T GC012F for patients with relapsed/refractory B-cell non-Hodgkin’s lymphoma
Session title: Poster session
Presentation time: Friday, June 10 from 4:30 – 5:45 PM CEST
BCMA/CD19 Dual-Targeting FasTCAR-T GC012F for the Treatment of RRMM

Gracell will also present as an oral abstract presentation the updated results from the first-in-human IIT evaluating GC012F for the treatment of RRMM patients. This data is currently under embargo and will be published on the EHA (Free EHA Whitepaper)2022 Hybrid Congress website on Thursday, May 26 concurrently with ASCO (Free ASCO Whitepaper).

Details of the presentation are as follows:

Abstract title: Updated results of a multicenter first-in-human study of BCMA/CD19 dual-targeting FasTCAR-T GC012F for patients with relapsed/refractory multiple myeloma (RRMM)
Session title: Relapsed/refractory myeloma: BCMA-directed therapies
Presentation time: Sunday, June 12 from 11:30 AM – 12:45 PM CEST
Presentation location: Hall A2-A3
CD19/CD7 Dual-directed Allogeneic TruUCAR-T GC502 for the Treatment of B-ALL

GC502 leverages the novel dual-directed CAR design of Gracell’s proprietary TruUCAR platform, designed to generate high-quality allogeneic CAR-T cell therapies that can be administered "off-the-shelf" at lower cost and with faster patient’s access. TruUCAR-enabled GC502 utilizes the dual-directed CAR design with one CAR targeting CD19 on malignant cells and a second CAR targeting CD7 to suppress host-versus-graft rejection. An enhancer molecule is embedded in the basic construct of TruUCAR to enhance proliferation of TruUCAR T cells.

Between September 2021 and January 2022, four r/r B-ALL patients were enrolled and treated in an open-label, non-randomized, prospective IIT in China in two different dose levels and with two different formulations. Patients were heavily pretreated, and all had previously received either autologous or donor derived CD19 or CD19/CD22 targeted CAR-T therapy. As of the January 28, 2022 data cutoff date, all four patients had received a single dose of GC502, including one patient at dose level 1 (DL1) 1.0×107 cells/kg and three patients at dose level 2 (DL2) 1.5×107 cells/kg. Patients received a Flu/Cy based lymphodepletion regimen prior to treatment with GC502.

Three of four patients achieved minimal residual disease negative complete response or complete response with incomplete count recovery (MRD- CR/CRi), and one patient achieved a partial response at month one and subsequently received allogeneic hematopoietic stem-cell transplantation (allo-HSCT) on day 39.

Cytokine release syndrome (CRS) presented as Grade 2 and Grade 3 with no Grade 4 or 5 events. No immune effector cell-associated neurotoxicity syndrome (ICANS) or acute graft-versus-host disease (aGvHD) were observed.

Details of the presentation are as follows:

Abstract title: Early results of a safety and efficacy study of allogeneic TruUCARTM GC502 in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (R/R B-ALL)
Session title: Poster session
Presentation time: Friday, June 10 from 4:30 – 5:45 PM CEST
For more information about the EHA (Free EHA Whitepaper)2022 Hybrid Congress, visit www.ehaweb.org.

About GC012F

GC012F is a FasTCAR-enabled dual-targeting CAR-T product candidate that is currently being evaluated in IIT studies in China for the treatment of multiple myeloma and B-cell non-Hodgkin’s lymphoma. GC012F simultaneously targets CD19 and BCMA to drive fast, deep and durable responses, which can potentially improve efficacy and reduce relapse in multiple myeloma and B-NHL patients.

About B-NHL

Non-Hodgkin’s lymphoma (NHL) is a group of blood cancers that developed from lymphocytes, most commonly derived from B cells (B-NHL). Globally, approximately 510,000 patients are diagnosed with NHL every year with about 80,470 patients expected to be diagnosed with NHL in the United States in 2022[1]. B-NHL accounts for approximately 85% of NHL diagnoses.

[1] Data source: American Cancer Society

About GC502

GC502 is a TruUCAR-enabled CD19/CD7 dual-directed, off-the-shelf allogeneic CAR-T product candidate that is being studied in an ongoing Phase 1 IIT in China for the treatment of B-cell malignancies. GC502 is manufactured using T cells from non-human leukocyte antigen (HLA) matched healthy donors. An enhancer molecule is embedded in the basic construct of TruUCAR to enhance proliferation of TruUCAR T cells. Optimized for CD19/CD7 dual-CAR functionality and in vivo durability, GC502 has demonstrated robust anti-tumor effects with potential to suppress host versus graft (HvG) rejection in preclinical models.

About B-ALL

Acute lymphoblastic leukemia (ALL) is a type of blood cancer characterized by proliferation of immature lymphocytes in the bone marrow, which can involve either T lymphocytes (T-ALL), or B lymphocytes (B-ALL). Globally, approximately 64,000 patients are diagnosed with ALL every year with an estimated 6,660 new cases to be diagnosed in the United States in 2022[2]. B-ALL accounts for 75% of ALL diagnoses in adults.

[2] Data source: American Cancer Society

About FasTCAR

CAR-T cells manufactured on Gracell’s proprietary FasTCAR platform appear younger, less exhausted and show enhanced proliferation, persistence, bone marrow migration and tumor cell clearance activities as demonstrated in preclinical studies. With next day manufacturing, FasTCAR is able to significantly improve cell production efficiency which may result in meaningful cost savings, and, together with fast turnaround time, enables enhanced accessibility of cell therapies for cancer patients.

About TruUCAR

TruUCAR is Gracell’s proprietary technology platform and is designed to generate CAR-T cell therapies from high quality allogeneic T cells that can be administered "off-the-shelf" at lower cost and with improved accessibility of cell therapies for cancer patients. With differentiated design enabled by gene editing, TruUCAR is designed to control HvG as well as GvHD without the need for being co-administered with additional strong immunosuppressant after conventional lymphodepletion. The novel dual-CAR design allows tumor antigen-CAR moiety to target malignant cells, while the CD7 CAR moiety is designed to suppress rejection (HvG response) of allogeneic CAR-T cells by host T and NK cells (HvG).

Jazz Pharmaceuticals to Present Data Showcasing Clinical Advancements Across Oncology Portfolio at 2022 ASCO and EHA Meetings

On May 12, 2022 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the Company and its partners will present seven abstracts at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting from June 3 – June 7, 2022, and eight abstracts at the 27th Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) from June 9 –12, 2022. Research findings to be presented include data on Rylaze/JZP458, Zepzelca (lurbinectedin), Defitelio (defibrotide sodium) and Vyxeos/Vyxeos Liposomal (daunorubicin and cytarabine), also known as JZP351 (Press release, Jazz Pharmaceuticals, MAY 12, 2022, View Source [SID1234614434]).

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"As part of Jazz’s commitment to explore potential new applications of our oncology medicines to address critical needs, we continue to advance programs that could impact difficult-to-treat therapeutic areas," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "Our growing, early-stage pipeline, combined with ongoing Jazz-sponsored and partner research across our portfolio, is making significant progress when it comes to addressing unmet patient needs in cancers that have historically lacked scientific advancements."

Highlights from Jazz and its investigational sponsors at the congresses feature data for our medicines across a range of solid tumors and hematological malignancies, including:

An oral presentation at ASCO (Free ASCO Whitepaper) featuring results from Cohort 1 of a Phase 2/3 trial conducted with the Children’s Oncology Group, evaluating the efficacy and safety of Rylaze administered intramuscularly (IM) on a Monday/Wednesday/Friday dosing schedule, for patients living with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL).
Two poster presentations for JZP351 including preliminary results from Arm B (JZP351 in combination with midostaurin) from the Phase 1b V-FAST trial in adults with previously untreated FLT3-mutated acute myeloid leukemia (AML) and data from the Phase 1b trial evaluating lower-intensity JZP351 + venetoclax in adults with newly diagnosed AML who are unfit for intensive chemotherapy. These data will be presented at both ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper).
Four poster presentations at ASCO (Free ASCO Whitepaper) evaluating Zepzelca in a range of small cell lung cancer (SCLC) settings, both as a monotherapy and in combination with other therapies, and in BRCA1/2-associated metastatic breast cancer.
The Jazz-supported presentations at the 2022 ASCO (Free ASCO Whitepaper) Annual Meeting are:

Rylaze Presentations

Presentation Title

Author

Presentation Details

Efficacy and safety of intramuscular (IM) recombinant Erwinia asparaginase in acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL): The Children’s Oncology Group (COG) AALL1931 study

Maese L, et al.

Type: Oral presentation

Session: Oral Abstract Session/ Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant

Date: June 7 at 10:45 a.m. EDT

Abstract number: 7001

Abstract Link

Zepzelca Presentations

Presentation Title

Author

Presentation Details

Analysis of patients with relapsed small cell lung cancer (SCLC) receiving single-agent lurbinectedin in the phase 3 ATLANTIS trial

Navarro A, et al.

Type: Poster

Session: Lung Cancer – Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers

Date: June 6 at 9:00 a.m. EDT

Abstract number: 8524

Abstract link

Efficacy and safety of lurbinectedin as second-line therapy in Chinese patients with small cell lung cancer: Preliminary results of a phase 1 study

Cheng Y, et al.

Type: Poster

Session: Lung Cancer – Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers

Date: June 6 at 9:00 a.m. EDT

Abstract number: 8580

Abstract link

A phase 1/2 trial of lurbinectedin (L) in combination with pembrolizumab (P) in relapsed small cell lung cancer (SCLC): The LUPER study

Calles Blanco A, et al.

Type: Poster

Session: Lung Cancer – Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers

Date: June 6 at 9:00 a.m. EDT

Abstract number: 8581

Abstract link

Lurbinectedin in patients with pretreated BRCA1/2-associated metastatic breast cancer: Results from a phase II basket study

Boni V, et al.

Type: Poster

Session: Breast Cancer – Metastatic

Date: June 6 at 9:00 a.m. EDT

Abstract number: 1092

Abstract link

Vyxeos Presentations

Presentation Title

Author

Presentation Details

V-FAST master trial: Preliminary results of treatment with CPX-351 plus midostaurin in adults with newly diagnosed FLT3-mutated acute myeloid leukemia

McCloskey J, et al.

Type: Poster

Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant

Date: June 4 at 9:00 a.m. EDT

Abstract number: 7043

Abstract link

Lower-intensity CPX-351 + venetoclax for patients with newly diagnosed AML who are unfit for intensive chemotherapy

Uy G.L. et al.

Type: Poster

Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant

Date: June 4 at 9:00 a.m. EDT

Abstract number: 7031

Abstract link

The Jazz-supported presentations at the EHA (Free EHA Whitepaper) 27th Congress are:

Vyxeos Liposomal Presentations

Presentation Title

Author

Presentation Details

Lower-intensity CPX-351 + venetoclax for patients with newly diagnosed acute myeloid leukemia who are unfit for intensive chemotherapy

Uy L. G. et al.

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P515

Abstract link

CPX-351 treatment for acute myeloid leukemia in England: Real-world outcomes in adults aged <60 years versus >60 years

Legg A. et al.

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P513

Abstract link

V-FAST master trial: Preliminary results of treatment with CPX-351 plus midostaurin in adults with newly diagnosed FLT3-mutated acute myeloid leukemia

McCloskey J, et al

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P514

Abstract link

Real life experience using front-line CPX-351 for therapy-related and AML-MRC: results from the Spanish PETHEMA registry (IST)

Bernal T, et al

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P508

Abstract link

A randomised comparison of CPX-351 and FLAG-Ida in high risk acute myeloid leukaemia. Results from the NCRI AML19 trial

Russel N, et al

Type: Oral presentation

Date: June 11

Abstract number: S128

Abstract link

Defitelio Presentations

Presentation Title

Author

Presentation Details

Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) after autologogous hematopoietic cell transplantion (HCT): Outcomes of defibrotide-treated adult patients from the DefiFrance study

Mohty M, et al

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P1355

Abstract link

A systematic literature review (SLR) of the manifestations of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) after hematopoietic cell transplant (HCT) in adults versus children

Angus J, et al.

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P1354

Abstract link

Asparaginase Presentations

Presentation Title

Author

Presentation Details

Phase 1 trial of pegcrisantaspase in combination with venetoclax in adults with relapsed or refractory acute myeloid leukemia (R/R AML) – Safety, efficacy and PK/PD in the first two cohorts

Liu Y, et al

Type: Poster

Date: June 10 at 16:30 CEST

Abstract number: P527

Abstract link

All ASCO (Free ASCO Whitepaper) virtual poster presentations and poster discussion presentations will be available on-demand to registered participants for 180 days beginning June 3, 2022. EHA (Free EHA Whitepaper) presentations will be available on-demand beginning June 20, 2022.

About Zepzelca (lurbinectedin)
Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and eventual cell death.2

Zepzelca for injection 4 mg is a prescription medicine used to treat adults with a kind of lung cancer called small cell lung cancer that has spread to other parts of the body (metastatic) and who have received treatment with chemotherapy that contains platinum, and it did not work or is no longer working. Zepzelca is approved based on response rate and how long the response lasted. Additional studies will further evaluate the benefit of Zepzelca for this use.

Important Safety Information for ZEPZELCA

Before receiving ZEPZELCA, tell your healthcare provider about all of your medical conditions, including if you:

have liver or kidney problems.
are pregnant or plan to become pregnant. ZEPZELCA can harm your unborn baby.
Females who are able to become pregnant:
Your healthcare provider should do a pregnancy test before you start treatment with ZEPZELCA.
You should use effective birth control (contraception) during treatment with and for 6 months after your final dose of ZEPZELCA.
Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with ZEPZELCA.
Males with female partners who are able to become pregnant should use effective birth control during treatment with and for 4 months after your final dose of ZEPZELCA.

Are breastfeeding or plan to breastfeed. It is not known if ZEPZELCA passes into your breastmilk. Do not breastfeed during treatment with ZEPZELCA and for 2 weeks after your final dose of ZEPZELCA. Talk to your healthcare provider about the best way to feed your baby during treatment with ZEPZELCA.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Certain other medicines may affect how ZEPZELCA works.

What should I avoid while using ZEPZELCA?

Avoid eating or drinking grapefruit, or products that contain grapefruit juice during treatment with ZEPZELCA.

ZEPZELCA can cause serious side effects, including:

Low blood cell counts. Low blood counts including low neutrophil counts (neutropenia) and low platelet counts (thrombocytopenia) are common with ZEPZELCA, and can also be severe. Some people with low white blood cell counts may get fever, or an infection throughout the body (sepsis), that can cause death. Your healthcare provider should do blood tests before you receive each treatment with ZEPZELCA to check your blood cell counts.
Tell your healthcare provider right away if you develop:

fever or any other signs of infection
unusual bruising or bleeding
tiredness
pale colored skin
Liver problems. Increased liver function tests are common with ZEPZELCA, and can also be severe. Your healthcare provider should do blood tests to check your liver function before you start and during treatment with ZEPZELCA.
Tell your healthcare provider right away if you develop symptoms of liver problems including:

loss of appetite 
nausea or vomiting 
pain on the right side of your stomach area (abdomen)
Your healthcare provider may temporarily stop treatment, lower your dose, or permanently stop ZEPZELCA if you develop low blood cell counts or liver problems during treatment with ZEPZELCA.

The most common side effects of ZEPZELCA include:

tiredness
low white and red blood cell counts
increased kidney function blood test (creatinine)
increased liver function blood tests
increased blood sugar (glucose)
nausea
decreased appetite
muscle and joint (musculoskeletal) pain
low level of albumin in the blood
constipation
trouble breathing
low levels of sodium and magnesium in the blood
vomiting
cough
diarrhea
These are not all of the possible side effects of ZEPZELCA.

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.

More information about Zepzelca, including Full Prescribing Information and Patient Information, is available here.

ZEPZELCA is a trademark of PharmaMar, S.A. used by Jazz Pharmaceuticals under license.

About RYLAZE (asparaginase erwinia chrysanthemi (recombinant)-rywn)
RYLAZE, also known as JZP458, is approved in the U.S. for use as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients one month or older who have developed hypersensitivity to E. coli-derived asparaginase. RYLAZE has orphan drug designation for the treatment of ALL/LBL in the United States. RYLAZE is a recombinant erwinia asparaginase that uses a novel Pseudomonas fluorescens expression platform. JZP458 was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in October 2019 for the treatment of this patient population. RYLAZE was approved as part of the Real-Time Oncology Review program, an initiative of the FDA’s Oncology Center of Excellence designed for efficient delivery of safe and effective cancer treatments to patients.

The full U.S. Prescribing Information for RYLAZE is available here.

Important Safety Information for Rylaze

RYLAZE should not be given to people who have had:

Serious allergic reactions to RYLAZE
Serious swelling of the pancreas (stomach pain), serious blood clots, or serious bleeding during previous asparaginase treatment
RYLAZE may cause serious side effects, including:

Allergic reactions (a feeling of tightness in your throat, unusual swelling/redness in your throat and/or tongue, or trouble breathing), some of which may be life-threatening
Swelling of the pancreas (stomach pain)
Blood clots (may have a headache or pain in leg, arm, or chest)
Bleeding
Liver problems
Contact your doctor immediately if any of these side effects occur.

Some of the most common side effects with RYLAZE include: liver problems, nausea, bone and muscle pain, tiredness, infection, headache, fever, allergic reactions, fever with low white blood cell count, decreased appetite, mouth swelling (sometimes with sores), bleeding, and too much sugar in the blood.

RYLAZE can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Females of reproductive potential should use effective contraception (other than oral contraceptives) during treatment and for 3 months following the final dose. Do not breastfeed while receiving RYLAZE and for 1 week after the final dose.

Tell your healthcare provider if there are any side effects that are bothersome or that do not go away.

These are not all the possible side effects of RYLAZE. For more information, ask your healthcare provider.

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 (1-800-332-1088).

About Vyxeos/Vyxeos Liposomal (daunorubicin and cytarabine), also known as JZP351
Vyxeos is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor.

In the U.S., Vyxeos 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.1

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

In Europe, Vyxeos Liposomal (daunorubicin/cytarabine) is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Backed by a robust clinical development program including Phase 3 data, Vyxeos is currently approved in more than 30 countries, and Jazz continues to work with regulatory authorities worldwide to bring this innovative therapy to appropriate patients.

The full Summary of Product Characteristics of Vyxeos Liposomal in Europe is available here.

Important Safety Information for VYXEOS/VYXEOS LIPOSOMAL
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 Defitelio (defibrotide sodium)
In the U.S., Defitelio (defibrotide sodium) injection 80mg/mL received U.S. Food and Drug Administration (FDA) marketing approval on March 30, 2016, and it is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT) and is the first and only FDA-approved therapy for patients with this rare, potentially fatal complication. Defitelio is not approved for the prevention of VOD.

Please see full Prescribing Information for Defitelio in the United States.

In Europe, defibrotide is marketed under the name Defitelio ▼ (defibrotide). In October 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients after HSCT therapy. In Europe, Defitelio is indicated in patients over one month of age. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy.

▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system found under section 4.8 of the SmPC.
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The full Summary of Product Characteristics of Defitelio in Europe is available here.

Important Safety Information for Defitelio
Defitelio should not be given to patients who are:

Currently taking anticoagulants or fibrinolytics
Allergic to Defitelio or any of its ingredients
Defitelio may increase the risk of bleeding in patients with VOD and should not be given to patients with active bleeding. During treatment with Defitelio, patients should be monitored for signs of bleeding. In the event that bleeding occurs during treatment with Defitelio, treatment should be temporarily or permanently stopped. Patients should tell the doctor right away about any signs or symptoms of hemorrhage such as unusual bleeding, easy bruising, blood in urine or stool, headache, confusion, slurred speech, or altered vision.

Defitelio may cause allergic reactions including anaphylaxis. Patients who develop signs and symptoms of anaphylaxis such as trouble breathing, severe itching, skin rash or hives, or swelling of the face, lips, mouth or tongue should seek medical attention immediately.

The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds.