On November 28, 2018 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that nine abstracts, including two oral presentations, relating to the company’s hematology/oncology portfolio were accepted for the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego from December 1-4 (Press release, Jazz Pharmaceuticals, NOV 28, 2018, View Source;p=RssLanding&cat=news&id=2378498 [SID1234531666]).

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"We look forward to showcasing our commitment to the hematology/oncology community at ASH (Free ASH Whitepaper) by advancing the science and addressing the clinical needs of patients with blood cancers and with complications of stem cell transplantation," said Allen Yang, M.D., Ph.D., head of clinical development and acting chief medical officer of Jazz Pharmaceuticals. "We’ve made great progress in the last year with the initiation of new clinical trials for Vyxeos and Defitelio, the marketing authorization of Vyxeos in the European Union, and a collaboration with MD Anderson Cancer Center to evaluate potential treatment options for hematologic malignancies."

Jazz Pharmaceuticals data at the 2018 ASH (Free ASH Whitepaper) Annual Meeting will highlight the following:
Results from a post-hoc analysis from the pivotal Phase 3 randomized trial of Vyxeos (daunorubicin and cytarabine) liposome for injection, also known as the molecule name CPX-351, in a subgroup of older patients with newly diagnosed high risk (secondary) AML with myelodysplasia-related changes (AML-MRC) that evaluated the efficacy of Vyxeos compared to conventional 7+3 chemotherapy on overall survival and remission rates as well as the safety profile in older adults with AML-MRC. Additionally, data will be presented from an exploratory analysis which evaluated the impact of hematopoietic cell transplantation (HCT) on survival in patients treated with Vyxeos compared to 7+3 in the Phase 3 trial.

Vyxeos abstracts include:

Efficacy and Safety of CPX-351 versus 7+3 in a Subgroup of Older Patients with Newly Diagnosed Acute Myeloid Leukemia with Myelodysplasia-Related Changes (AML-MRC) Enrolled in a Phase 3 Study [Abstract #1425; Saturday, December 1, 6:15 PM – 8:15 PM PST]
Population Pharmacokinetic (PK)/Pharmacodynamic (PD) Modeling of Myelosuppression in Patients with Hematologic Malignancies for CPX-351 and Standard-of-Care 7+3 Therapy [Abstract #4037; Monday, December 3, 6:00 PM – 8:00 PM PST]
The Impact of Hematopoietic Cell Transplantation on Survival: An Exploratory Analysis of a Phase 3 Study of CPX-351 versus 7+3 in Older Patients with Newly Diagnosed, High-Risk/Secondary AML [Abstract #2706; Sunday, December 2, 6:00 PM – 8:00 PM PST]
Final Safety and Efficacy Results from the CPX-351 Early Access Program (EAP) for Older Patients with High-Risk/Secondary Acute Myeloid Leukemia (sAML) [Abstract #1434; Saturday, December 1, 6:15 PM – 8:15 PM PST]
Impact of Post–Hematopoietic Cell Transplant (HCT) Survival on Cost-effectiveness of CPX-351 versus 7+3 in the Treatment of Therapy-Related AML or AML-MRC in the United States (online only)
A Phase I/Pilot Study of CPX-351 [Daunorubicin and Cytarabine Liposome for Injection (Vyxeos)] for Children, Adolescents and Young Adults with Recurrent or Refractory Acute Leukemia [Abstract #336; Sunday, December 2, 10:45 AM PST(oral presentation)]
In addition, data from an expanded access program and post-hoc analyses of clinical trials for Defitelio (defibrotide sodium) will be presented, including an oral presentation of a pooled analysis of survival based on timing of initiation in adults with Veno-Occlusive Disease/ Sinusoidal Obstruction Syndrome (VOC/SOS) following hematopoietic stem cell transplant (HSCT).

Defitelio abstracts include:

Incidence of Post-Hematopoietic Stem Cell Transplantation (HSCT) Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Without Hyperbilirubinemia at Diagnosis and Efficacy of Defibrotide in an Expanded-Access Program [Abstract #2080; Saturday, December 1, 6:15 PM – 8:15 PM PST]
A Pooled Analysis of Survival by Defibrotide Timing of Initiation in Adults with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Following Hematopoietic Stem Cell Transplant (HSCT) [Abstract #815; Monday, December 3, 3:45 PM PST (oral presentation)]
Cost-Effectiveness of Defibrotide for the Treatment of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) with Multi-Organ Dysfunction (MOD) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Canada [Abstract #4702; Monday, December 3, 6:00 PM – 8:00 PM PST]
About Vyxeos
Vyxeos (daunorubicin and cytarabine) is a liposome formulation of a fixed combination of daunorubicin and cytarabine for intravenous infusion, and is approved for the treatment of two types of secondary AML in adult patients, newly diagnosed therapy-related acute myeloid leukaemia (t-AML) and AML with myelodysplasia-related changes (AML-MRC). Vyxeos is the first product developed with the company’s proprietary CombiPlex platform, which enables the design and rapid evaluation of various combinations of therapies. Vyxeos received U.S. FDA approval and orphan drug exclusivity in August 2017 and EU EMA marketing authorization in August 2018. Vyxeos received Orphan Drug Designation for the treatment of AML by the U.S. FDA in September 2008 and by the European Commission in January 2012 (with retention of the designation reaffirmed in July 2018). Vyxeos received Promising Innovative Medicine (PIM) designation from the Medicines and Healthcare Products Regulatory Agency in the United Kingdom.

Important Safety Information
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.

Please see full Prescribing Information for Vyxeos including BOXED Warning, and visit www.Vyxeos.com for additional information.

About Defitelio
In the U.S., Defitelio (defibrotide sodium) injection 80mg/mL received U.S. FDA marketing approval on March 30, 2016 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.

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 undergoing HSCT therapy. It is the first and only approved treatment in Europe for severe VOD. 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. (View Source)

Important Safety Information

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.

Please see full Prescribing Information for Defitelio and visit www.Defitelio.com for additional information.

On November 28, 2018 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported that the U.S. Patent and Trademark Office (USPTO) has issued a new patent protecting the Company’s lead drug candidate, tipifarnib, a potent and selective farnesyl transferase that is currently being studied in multiple solid tumor and hematologic indications, including a registration-directed trial in HRAS mutant head and neck squamous cell carcinoma (HNSCC) and a Phase 2 trial in peripheral T-cell lymphoma (PTCL) (Press release, Kura Oncology, NOV 28, 2018, View Source [SID1234531664]).

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U.S. Patent No. 10,137,121, "Methods of Treating Cancer with Farnesyltransferase Inhibitors," includes multiple claims directed to the use of tipifarnib as a method of treating patients with angioimmunoblastic T-cell lymphoma (AITL), an aggressive form of T-cell lymphoma. The newly issued patent has an expiration date of November 2037, excluding any possible patent term extension. Kura continues to pursue U.S. and foreign patent protection in this and other indications.

"The issuance of this new patent is an important achievement for Kura and reflects our ability to expand the breadth and depth of tipifarnib’s development opportunities," said Troy Wilson, Ph.D., President and CEO of Kura Oncology. "This patent comes just six months after the USPTO issued us a patent for the use of tipifarnib as method of treating patients with certain CXCL12-expressing cancers, further strengthening our intellectual property protection for tipifarnib based on genetically defined patient populations and disease indications."

Kura is evaluating, on a prospective basis, the role of the CXCL12 pathway and markers of bone marrow homing as potential biomarkers of clinical activity for tipifarnib in hematologic malignancies. The Company’s ongoing Phase 2 trial of tipifarnib in PTCL is enrolling patients into two expansion cohorts. The first cohort is defined by histology and includes patients with AITL. The second cohort is defined by genetics and includes patients with PTCL not otherwise specified (NOS) who have the absence of a single nucleotide variation in the 3’ untranslated region of the CXCL12 gene. The Company estimates that the combined addressable populations of patients with AITL and CXCL12+ account for approximately 40% of all PTCL cases.

Kura plans to report preliminary data from both expansion cohorts in its Phase 2 trial of tipifarnib at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego on Sunday, December 2, 2018. A copy of the poster will be available on the Company’s website at www.kuraoncology.com following presentation at the meeting.

About Tipifarnib

Kura Oncology’s lead candidate, tipifarnib, is an inhibitor of farnesylation, a key cell signaling process implicated in cancer initiation and development. Tipifarnib was previously studied in more than 5,000 cancer patients and showed compelling and durable anti-cancer activity in certain patient subsets with a manageable side effect profile. Leveraging advances in next-generation sequencing as well as emerging information about cancer genetics and tumor biology, the Company is seeking to identify those patients most likely to benefit from tipifarnib. Based on positive results from a Phase 2 clinical trial in HRAS mutant HNSCC and feedback from the U.S. Food and Drug Administration, Kura recently initiated a global, registration-directed trial of tipifarnib in patients with recurrent or metastatic HRAS mutant HNSCC.

On November 28, 2018 Aclaris Therapeutics, Inc. (NASDAQ:ACRS), a dermatologist-led biopharmaceutical company committed to identifying, developing, and commercializing innovative therapies to address significant unmet needs in aesthetic and medical dermatology and immunology, reported that management will attend the following conferences (Press release, Aclaris Therapeutics, NOV 28, 2018, View Source [SID1234531663]):

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Management will host investor meetings at the 2018 Citi Global Healthcare in New York, NY on Wednesday, December 5, 2018.
Dr. Neal Walker, President and Chief Executive Officer, will present at the 12th Annual Leerink POLARxPRESS Conference in New York, NY on Tuesday, December 11, 2018.

On November 28, 2018 the U.S. Food and Drug Administration approved Truxima (rituximab-abbs) as the first biosimilar to Rituxan (rituximab) for the treatment of adult patients with CD20-positive, B-cell non-Hodgkin’s lymphoma (NHL) to be used as a single agent or in combination with chemotherapy. Truxima is the first biosimiliar to be approved in the U.S. for the treatment of non-Hodgkin’s lymphoma.

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"As part of the FDA’s Biosimilars Action Plan we’re advancing new policies to make the development of biosimilars more efficient and to enable more opportunities for biosimilar manufacturers to make these products commercially successful and competitive. Our goal is to promote competition that can expand patient access to important medicines," said FDA Commissioner Scott Gottlieb, M.D. "The Truxima approval is our third biosimiliar approval in the past month. The growing pipeline of biosimilars is encouraging. We’re seeing more biosimilar drugs gain market share as this industry matures. We’ll continue to make sure biosimilar medications are evaluated efficiently through a process that makes certain that these new medicines meet the FDA’s rigorous standards for approval."

Truxima is indicated for the treatment of adult patients with:

Relapsed or refractory, low grade or follicular, CD20-positive B-cell NHL as a single agent;
Previously untreated follicular, CD20-positive, B-cell NHL in combination with first line chemotherapy and, in patients achieving a complete or partial response to a rituximab product in combination with chemotherapy, as single-agent maintenance therapy; and
Non-progressing (including stable disease), low-grade, CD20­ positive, B-cell NHL as a single agent after first-line cyclophosphamide, vincristine and prednisone (CVP) chemotherapy.

Biological products are generally large, complex molecules and may be produced through biotechnology in a living system, such as a microorganism, plant cell or animal cell. A biosimilar is a biological product that is approved based on data showing that it is highly similar to a biological product already approved by the FDA (reference product) and has no clinically meaningful differences in terms of safety, purity and potency (i.e., safety and effectiveness) from the reference product, in addition to meeting other criteria specified by law.

The FDA’s approval of Truxima is based on a review of evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic data, clinical immunogenicity data, and other clinical data that demonstrates Truxima is biosimilar to Rituxan. Truxima has been approved as a biosimilar, not as an interchangeable product.

The most common side effects of Truxima are infusion reactions, fever, abnormally low level of lymphocytes in the blood (lymphopenia), chills, infection and weakness (asthenia). Health care providers are advised to monitor patients for tumor lysis syndrome (a complication of treatment where tumor cells are killed off at the same time and released into the bloodstream), cardiac adverse reactions, damage to kidneys (renal toxicity), and bowel obstruction and perforation. Patients should not receive vaccinations while in treatment. Women who are pregnant or breastfeeding should not take Truxima because it may cause harm to a developing fetus or newborn baby.

Like Rituxan, the labeling for Truxima contains a Boxed Warning to alert health care professionals and patients about increased risks of the following: fatal infusion reactions, severe skin and mouth reactions, some with fatal outcomes; Hepatitis B virus reactivation, that may cause serious liver problems including liver failure and death; and Progressive Multifocal Leukoencephalopathy, a rare, serious brain infection that can result in severe disability or death. This product must be dispensed with a patient Medication Guide that provides important information about the drug’s uses and risks.

The FDA granted approval of Truxima to Celltrion. Rituxan was approved in November 1997 and is manufactured by Genentech.

With the Truxima approval, the FDA has approved 15 biosimiliars. On Oct. 30, 2018, the FDA approved Hyrimoz (adalimumab-adaz), from Sandoz, as a biosimilar to Humira (adalimumab) and on Nov. 2, 2018, the FDA approved Udenyca (pegfilgrastim-cbqv) from Coherus, as a biosimilar to Neulasta (pegfilgrastim).

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

On November 28, 2018 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a company focused on the development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell ("CAR T") technology and gene therapies for rare diseases, reported that additional safety and efficacy Phase 1 data evaluating MB-102 (CD123 CAR) in relapsed or refractory acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN) have been selected for an oral presentation at the AACR (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy (Press release, Mustang Bio, NOV 28, 2018, View Source [SID1234531653]). The presentation will take place on November 30, 2018, in Miami Beach, Florida.

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Martina Sersch, M.D., Ph.D., Chief Medical Officer of Mustang, said, "We are excited about the additional data demonstrating MB-102’s (CD123 CAR T) potential to treat patients with AML and BPDCN. Initial safety and tolerability data as well as preliminary data on efficacy are very promising in a population with high unmet medical need. Based on the Phase 1 data, we expect to submit an IND filing for MB-102 and look forward to initiating a multicenter Phase 1/2 clinical trial in 2019 in patients with AML, BPDCN and high-risk myelodysplastic syndrome."

Lihua Elizabeth Budde, M.D., Ph.D., assistant professor in the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope and principal investigator for the Phase 1 trial, said, "There is increased expression of CD123 on AML blasts, leukemic stem cells and BPDCN cells compared to normal hematopoietic stem cells, and it is therefore a promising target for cellular immunotherapy. We remain encouraged by interim data showing MB-102’s potential to treat BPDCN and AML and continue to evaluate MB-102’s clinical benefits in our ongoing Phase 1 clinical trial."

Details of the oral presentation are as follows:

Title: CD123CAR Displays Clinical Activity in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Safety and Efficacy Results from a Phase 1 Study
Special Session: Novel Targets, Pathways and Tools
Date and Time: Friday, November 30, 2018; 8:00 AM – 9:30 AM ET
Location: Loews Miami Beach Hotel
Presenter: Elizabeth Lihua Budde, M.D., Ph.D., City of Hope, Duarte, CA

For more information, please visit: View Source;ItemID=733&DetailItemID=733.

Key Efficacy and Safety Findings
This single center, first-in-human Phase 1 dose-escalation clinical trial is evaluating the safety and activity of escalating doses of MB-102 in patients with relapsed or refractory AML (cohort 1) and BPDCN (cohort 2). Patients receive a single dose of MB-102 with an option for a second infusion if they continue to meet safety and eligibility criteria and still have CD123+ disease. To date, 18 patients have been enrolled and nine have been treated (seven with AML, two with BPDCN).

In the AML cohort, all seven patients had at least one prior allogeneic stem cell transplant and median of 4 (range: 4-10) prior lines of therapy. Two patients were treated at dose level 1 (50M CAR+ T). Trial investigators reported that one achieved a morphologic leukemic-free state (MLFS) that lasted 70 days. This patient received a second infusion three months later, with blast reduction from 77.9% to 0.9% (flow cytometry) after 35 days. Five patients received dose level 2 (200M CAR+ T). One patient achieved complete remission (CR) with incomplete count recovery at day 28, and one patient achieved MLFS that improved to CR at day 84. The remaining three patients had stable disease.

In the BPDCN cohort, two patients were treated with 100M CAR+ T and tolerated the treatment well, with no grade 3 or above treatment-related toxicities. One patient achieved CR with no evidence of disease in the bone marrow and skin at day 28.

Investigators found MB-102 infusions of up to 200M CAR T cells were safe. All toxicities observed to date were reversible and manageable. No patient developed grade 3 or above cytokine release syndrome or neurotoxicity. There were no treatment-related dose-limiting toxicities and no treatment-related cytopenias longer than 12 weeks. One patient with prior cutaneous graft-versus-host disease developed grade 1 rash five weeks after CAR T infusion, which resolved after clinical management. Peak of T cell expansion occurred within the first 14 days. Investigators did not observe any CD123-loss leukemic variants.

About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of the myeloid line of blood cells characterized by rapid growth of abnormal white blood cells that accumulate in the bone marrow. Although AML is rare, there are approximately 20,000 new cases in the U.S. each year and 10,000 deaths. Current treatment of relapsed or refractory AML with chemotherapy or hematopoietic stem cell transplantation is associated with low rates of complete response and considerable complications.

CD123 is overexpressed on AML blasts and leukemic stem cell-enriched cell subpopulations compared to normal hematopoietic stem cells and myeloid progenitors, making CD123 an attractive target for T cell-based adoptive immunotherapy.

About Blastic Plasmacytoid Dendritic Cell Neoplasm
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and incurable blood cancer with a median survival of less than 18 months and no standard of care. High levels of CD123 expression is one of the diagnostic hallmarks of BPDCN, making CD123 an attractive target for T cell-based adoptive immunotherapy.

About MB-102 (CD123 CAR T)
MB-102 (CD123 CAR T) is a CAR T cell therapy that is produced by engineering patient T cells to recognize and eliminate CD123-expressing tumors. CD123 is widely expressed on bone marrow cells of patients with MDS, as well as in hematologic malignancies including AML, B cell acute lymphoblastic leukemia, hairy cell leukemia, BPDCN, chronic myeloid leukemia and Hodgkin’s lymphoma.

In the first-in-human clinical trial at City of Hope (NCT02159495), MB-102 has demonstrated complete responses at low doses in AML and BPDCN without dose-limiting toxicities, as reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2017. Dose escalation continues at City of Hope in both indications.