On May 20, 2019 Clovis Oncology, Inc. (NASDAQ: CLVS) reported data demonstrating that patients with recurrent ovarian cancer who received Rubraca in the Phase 3 ARIEL3 study had longer periods of quality-adjusted time without clinically relevant symptoms as compared to patients who received placebo, and that Rubraca maintenance treatment continued to provide significant benefit in progression-free survival when weighted with patients’ perceptions of their wellbeing (Press release, Clovis Oncology, MAY 20, 2019, View Source [SID1234536486]). The presentation today at the International Society for Pharmacoeconomics and Outcomes (ISPOR) Annual Meeting in New Orleans highlights post-hoc evaluations of quality-adjusted time without symptoms or toxicity (Q-TWiST) and quality-adjusted PFS (QA-PFS) from the randomized, placebo-controlled study of Rubraca▼(rucaparib) for the maintenance treatment of patients with recurrent ovarian cancer. To the company’s knowledge, this is the first time that Q-TWiST data will be presented in the maintenance therapy setting in recurrent ovarian cancer.

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"The goal of maintenance therapy for recurrent ovarian cancer is to delay disease progression without compromising a patient’s quality of life," said Robert L. Coleman, MD, professor of Department of Gynecologic Oncology and Reproductive Medicine, The University of Texas MD Anderson Cancer Center. "The findings from QA-PFS and Q-TWiST analyses of ARIEL3 data demonstrate that the clinical benefits of rucaparib as maintenance therapy were retained across all trial populations. These results further support the positive benefit-risk profile of rucaparib in ARIEL3, including the absence of a detrimental effect on patient-centered outcomes across all treatment cohorts in the study."

During the study, patients were asked to complete EuroQol’s five-dimension, three-level (EQ-5D-3L) questionnaire at screening, on day 1 of each treatment cycle, at the treatment discontinuation visit, and at the 28-day follow-up visit. QA-PFS and Q-TWiST analyses were calculated using EQ-5D-3L data as described in the poster. Patient-centered outcomes were examined in the intent-to-treat (ITT) population (all randomized patients), in patients with a BRCA mutation, and in subgroups of patients with BRCA wild-type carcinomas based on loss of heterozygosity (LOH) status (BRCA wild type/LOH high; BRCA wild type/LOH low; and BRCA wild type/LOH indeterminate).

Results from the study include:

Mean QA-PFS was significantly longer in the Rubraca arm than placebo arm (12.02 vs 5.74 months) for the ITT population and for patients with a BRCA mutation (15.28 vs. 5.92 months)
In patients with a BRCA wild-type carcinoma, mean QA-PFS was longer in the Rubraca arm than the placebo arm regardless of LOH status
Mean Q-TWiST analysis using all grade ≥3 treatment-emergent adverse events (TEAEs) was significantly longer in the rucaparib arm than placebo for the ITT population (13.32 vs 6.44 months) and for patients with a BRCA mutation (16.42 vs 6.70 months)
In the analyses using TEAEs of interest (grade ≥2 TEAEs of nausea, vomiting, fatigue, and asthenia), Q-TWiST was also longer in the rucaparib arm than placebo for the ITT population (13.16 vs 6.40 months) and for patients with BRCA mutation (16.24 vs 6.68 months)
In patients with BRCA wild-type carcinoma, longer Q-TWiST was also observed in the Rubraca treated arm, regardless of LOH status
"It is very important to evaluate patients’ perceptions of their well-being during any type of treatment, particularly in the maintenance therapy setting in which patients may be on therapy for an extended period of time," said Professor Jonathan Ledermann, M.D., Professor of Medical Oncology, UCL Cancer Institute and UCL Hospitals, London. "These QA-PFS and Q-TWiST data from ARIEL3 integrate the patient perspective over the course of follow-up until progression to reflect their overall experience over time and suggests that the toxicity which may occur with rucaparib doesn’t outweigh its clinical benefit as maintenance treatment for women with recurrent ovarian cancer."

The Rubraca ISPOR poster will be available online at View Source as of the time it is presented at the meeting.

About Quality-Adjusted PFS (QA-PFS) and Quality-Adjusted Time Without Symptoms or Toxicity (Q-TWiST)

QA-PFS represents the duration of survival without disease progression, adjusted for the value the patient placed on their health status – i.e. it is a survival measure that adjusts for patient perceptions of treatment toxicity and any associated detrimental effects.
Q-TWiST results were derived by subtracting from the survival endpoint all time in which patients experienced treatment toxicity or disease symptoms, and then multiplying this area under the curve by a patient-derived utility value. Q-TWiST therefore adds additional value not available in a traditional TWiST analysis because it reflects patients’ perceptions of the impact of toxicity on the survival endpoint.
About the ARIEL3 Clinical Trial

The ARIEL3 pivotal study of rucaparib is a confirmatory randomized, double-blind study comparing the effects of rucaparib against placebo to evaluate whether rucaparib given as a maintenance treatment to platinum-sensitive ovarian cancer patients can extend the period of time for which the disease is controlled after a complete or partial response to platinum-based chemotherapy. The study enrolled 564 patients with high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer. To be eligible for the study, participants had to have received at least two prior platinum-based treatment regimens, been sensitive to the penultimate platinum regimen, and achieved a complete or partial response to their most recent platinum-based regimen. There were no genomic selection criteria for this study. Trial participants were randomized 2:1 to receive 600 milligrams of rucaparib twice daily (BID) or placebo. The study achieved its primary endpoint of improved PFS by investigator review in each of three populations. PFS was also improved in the rucaparib group compared with placebo by independent review, a key secondary endpoint, in all three populations. In addition, rucaparib improved objective response rate vs placebo among evaluable trial participants in all three study populations.

About Rubraca (rucaparib)

Rucaparib is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3 being developed in multiple tumor types, including ovarian and metastatic castration-resistant prostate cancers, as monotherapy, and in combination with other anti-cancer agents. Exploratory studies in other tumor types are also underway.

Rubraca U.S. FDA Approved Indications

Rubraca is indicated as monotherapy for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy.

Rubraca is indicated as monotherapy for the treatment of adult patients with deleterious BRCA mutations (germline and/or somatic) associated epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more chemotherapies and selected for therapy based on an FDA-approved companion diagnostic for Rubraca.

Select Important Safety Information

Myelodysplastic Syndrome (MDS)/Acute Myeloid Leukemia (AML) occur uncommonly in patients treated with Rubraca and are potentially fatal adverse reactions. In approximately 1100 treated patients, MDS/AML occurred in 12 patients (1.1%), including those in long-term follow-up. Of these, five occurred during treatment or during the 28-day safety follow-up (0.5%). The duration of Rubraca treatment prior to the diagnosis of MDS/AML ranged from 1 month to approximately 28 months. The cases were typical of secondary MDS/cancer therapy-related AML; in all cases, patients had received previous platinum-containing regimens and/or other DNA-damaging agents. Do not start Rubraca until patients have recovered from hematological toxicity caused by previous chemotherapy (≤ Grade 1).

Monitor complete blood counts for cytopenia at baseline and monthly thereafter for clinically significant changes during treatment. For prolonged hematological toxicities (> 4 weeks), interrupt Rubraca or reduce dose (see Dosage and Administration [2.2] in full Prescribing Information) and monitor blood counts weekly until recovery. If the levels have not recovered to Grade 1 or less after 4 weeks, or if MDS/AML is suspected, refer the patient to a hematologist for further investigations, including bone marrow analysis and blood sample cytogenetic analysis. If MDS/AML is confirmed, discontinue Rubraca.

Based on its mechanism of action and findings from animal studies, Rubraca can cause fetal harm when administered to a pregnant woman. Apprise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for 6 months following the last dose of Rubraca.

Most common adverse reactions in ARIEL3 (≥ 20%; Grade 1–4) were nausea (76%), fatigue/asthenia (73%), abdominal pain/distention (46%), rash (43%), dysgeusia (40%), anemia (39%), AST/ALT elevation (38%), constipation (37%), vomiting (37%), diarrhea (32%), thrombocytopenia (29%), nasopharyngitis/upper respiratory tract infection (29%), stomatitis (28%), decreased appetite (23%) and neutropenia (20%).

Most common laboratory abnormalities in ARIEL3 (≥ 25%; Grade 1–4) were increase in creatinine (98%), decrease in hemoglobin (88%), increase in cholesterol (84%), increase in alanine aminotransferase (ALT) (73%), increase in aspartate aminotransferase (AST) (61%), decrease in platelets (44%), decrease in leukocytes (44%), decrease in neutrophils (38%), increase in alkaline phosphatase (37%) and decrease in lymphocytes (29%).

Most common adverse reactions in Study 10 and ARIEL2 (≥ 20%; Grade 1–4) were nausea (77%), asthenia/fatigue (77%), vomiting (46%), anemia (44%), constipation (40%), dysgeusia (39%), decreased appetite (39%), diarrhea (34%), abdominal pain (32%), dyspnea (21%) and thrombocytopenia (21%).

Most common laboratory abnormalities in Study 10 and ARIEL2 (≥ 35%; Grade 1–4) were increase in creatinine (92%), increase in alanine aminotransferase (ALT) (74%), increase in aspartate aminotransferase (AST) (73%), decrease in hemoglobin (67%), decrease in lymphocytes (45%), increase in cholesterol (40%), decrease in platelets (39%) and decrease in absolute neutrophil count (35%).

Co-administration of Rubraca can increase the systemic exposure of CYP1A2, CYP3A, CYP2C9, or CYP2C19 substrates, which may increase the risk of toxicities of these drugs. Adjust dosage of CYP1A2, CYP3A, CYP2C9, or CYP2C19 substrates, if clinically indicated. If co-administration with warfarin (a CYP2C9 substrate) cannot be avoided, consider increasing frequency of international normalized ratio (INR) monitoring. Because of the potential for serious adverse reactions in breast-fed children from Rubraca, advise lactating women not to breastfeed during treatment with Rubraca and for 2 weeks after the last dose. You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Clovis Oncology, Inc. at 1-844-258-7662.

Click here for full Prescribing Information and additional Important Safety Information.

Rubraca▼ (rucaparib) EU Authorized Use and Important Safety Information

Rucaparib is indicated as monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy.

Rucaparib is indicated as monotherapy treatment of adult patients with platinum sensitive, relapsed or progressive, BRCA mutated (germline and/or somatic), high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have been treated with two or more prior lines of platinum-based chemotherapy, and who are unable to tolerate further platinum-based chemotherapy.

Summary warnings and precautions: Haematological toxicity: Patients should not start Rubraca until they have recovered from haematological toxicities caused by previous chemotherapy (≤ CTCAE Grade 1). Complete blood count testing prior to starting treatment with Rubraca and monthly thereafter is advised. Rubraca should be interrupted or dose reduced and blood counts monitored weekly until recovery for the management of low blood counts. Myelodysplastic syndrome/acute myeloid leukaemia (MDS/AML): If MDS/AML is suspected, the patient should be referred to a haematologist for further investigation. If MDS/AML is confirmed, Rubraca should be discontinued. Photosensitivity: Patients should avoid spending time in direct sunlight as they may burn more easily. When outdoors, patients should wear protective clothing and sunscreen with SPF of 50 or greater. Gastrointestinal toxicities: Low grade (CTCAE Grade 1 or 2) nausea and vomiting may be managed with dose reduction or interruption. Additionally, antiemetics may be considered for treatment or prophylaxis.

Click here to access the current Summary of Product Characteristics. Healthcare professionals should report any suspected adverse reactions via their national reporting systems.

On May 20, 2019 SpringWorks Therapeutics, a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported that the first patient has been dosed in the Phase 3 "DeFi" (Desmoid/Fibromatosis) trial evaluating nirogacestat, an oral, selective, small molecule gamma-secretase inhibitor, in adult patients with progressing desmoid tumors (Press release, SpringWorks Therapeutics, MAY 20, 2019, View Source [SID1234536485]).

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Desmoid tumors are rare and often debilitating and disfiguring soft-tissue tumors that can aggressively invade surrounding healthy tissues, including joints, muscles, blood vessels, nerves and internal organs. Depending on their size and location, desmoid tumors can cause significant morbidities, including severe pain, disfigurement, internal bleeding, debilitating loss of range of motion, and, in rare cases, desmoid tumors can be fatal.1 It is estimated that 1,000 to 1,500 new cases of desmoid tumors are diagnosed each year in the United States.2,3 There are currently no therapies approved by the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors.

"For patients living with these debilitating tumors, the DeFi trial marks a meaningful step forward in what we hope will result in the first approved treatment for this underserved patient community," said Saqib Islam, Chief Executive Officer of SpringWorks Therapeutics. "I am grateful for the persistence of our partners, colleagues and the patient advocacy community, whose collective efforts made it possible for SpringWorks to advance nirogacestat into our Phase 3 DeFi trial. We look forward to enrolling patients to confirm the clinical benefits of nirogacestat for people with desmoid tumors."

In June 2018, the FDA granted Orphan Drug designation for nirogacestat for the treatment of desmoid tumors, and in November 2018, the FDA granted Fast Track designation for nirogacestat for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.

About the DeFi Trial

The DeFi trial is a global, randomized, double-blind, placebo-controlled Phase 3 trial to evaluate the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors. The study will enroll approximately 100 adult patients, who will receive 150 mg of nirogacestat or placebo twice daily. Key eligibility criteria include tumor progression by ≥20% as measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1) within 12 months prior to the first dose of study treatment.

The primary endpoint is progression-free survival (PFS), defined as the time from randomization until the date of assessment of progression or death by any cause using RECIST 1.1. Secondary endpoints include safety and tolerability measures, as well as overall response rate, tumor volume changes as assessed by MRI, and changes in baseline in patient-reported outcomes.

More information about the DeFi trial is available at www.clinicaltrials.gov under the identifier NCT03785964.

About Desmoid Tumors

Desmoid tumors (also referred to as aggressive fibromatosis or desmoid-type fibromatosis) are rare and often debilitating and disfiguring soft tissue tumors characterized by a growth pattern that can aggressively invade surrounding healthy tissues, including joints, muscles, blood vessels, nerves and internal organs. While they can arise in any part of the body, the most common sites are the upper and lower extremities, abdominal walls, thoracic areas, and the head and neck. The severity of desmoid tumors and associated symptoms varies based on their size, location and the aggressiveness of the growth pattern. Desmoid tumors can cause significant morbidities, including severe pain, disfigurement, internal bleeding, debilitating loss of range of motion, and, in rare cases, desmoid tumors can be fatal.1

Desmoid tumors can affect children and adults, and are more commonly diagnosed in young adults between 20-30 years of age, with a two-to-three-fold predominance in females.1,4 It is estimated that desmoid tumors affect 2 to 5 per million people worldwide, and that there are 1,000 to 1,500 new cases diagnosed per year in the United States.2,3

Historically, desmoid tumors were treated with surgical resection or in severe cases, amputation, but even with these interventions, high rates of tumor regrowth have been observed.5 There are currently no FDA-approved therapies for the treatment of desmoid tumors.

About Nirogacestat

Nirogacestat is an investigational, oral, selective, small molecule gamma-secretase inhibitor. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

Nirogacestat has been investigated in 24 patients with desmoid tumors across Phase 1 and Phase 2 clinical trials. In these studies, treatment with nirogacestat demonstrated 100% disease control rate as measured by RECIST criteria, and median PFS was not reached by the time of publication in either trial due to lack of tumor progression events. Nirogacestat also showed an encouraging tolerability profile in these earlier studies, with many patients remaining on treatment for years and only one patient in the combined trials discontinuing due to an adverse event. The most common adverse events in prior studies were diarrhea, skin disorders and hypophosphatemia.

On May 20, 2019 Oragenics, Inc. (NYSE American: OGEN), a leader in the development of new antibiotics against infectious diseases and effective treatments for oral mucositis ("OM"), reported development updates regarding the Company’s lead compound for OM, AG013 (Press release, Oragenics, MAY 20, 2019, View Source [SID1234536484]). These developments include the enrollment of over 80 patients in the Company’s Phase 2 clinical of AG013 and, in addition, the World Health Organization has provided the Company with the generic name of dapatifagene navolactibac for the AG013 compound.

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"We continue to increase the number of clinical sites across the globe with 48 of the 61 identified clinical sites actively enrolling patients for our Phase 2 clinical trial of AG013," stated Alan Joslyn, Ph.D., President and Chief Executive Officer of Oragenics, Inc. "In the meantime, we are pleased to have an agreed upon generic designation for AG013, which will now also be referred to as dapatifagene navolactibac, to allow us a more specific way of referring to the compound moving forward. We anticipate providing further updates as developments warrant."

The ongoing Phase 2 trial is a double-blind, placebo-controlled, two-arm, multi-center trial, in which approximately 200 patients will be randomized in a 1:1 ratio to receive either dapatifagene navolactibac or placebo. The purpose of the study (NCT03234465) is to evaluate the safety, tolerability and efficacy of topically administered dapatifagene navolactibac compared to placebo for reducing the incidence and severity of OM in patients undergoing traditional chemoradiation for the treatment of head and neck cancer. Key measures include duration, time to development, and overall incidence of OM (using a World Health Organization scale) during the active treatment phase, which begins from the start of chemoradiation therapy and ends two weeks following its completion.

Dapatifagene navolactibac, which has been granted Fast Track designation with the U.S. Food and Drug Administration and orphan drug status in Europe, is an Intrexon Actobiotics therapeutic candidate formulated to deliver the therapeutic molecule, human Trefoil Factor 1, to the mucosal tissues in the oral cavity in a convenient oral rinsing solution. Trefoil Factors are a class of peptides involved in the protection of gastrointestinal tissues against mucosal damage and play an important role in these tissues’ subsequent regeneration. The compound was designed by the Company’s strategic partner, Intrexon Actobiotics NV, a wholly-owned subsidiary of Intrexon Corporation (NYSE: XON) whereby Oragenics, Inc. holds an exclusive world-wide license.

On May 20, 2019 Veracyte, Inc. (Nasdaq: VCYT), a leading genomic diagnostics company, reported new data demonstrating that its next-generation Percepta Genomic Sequencing Classifier (GSC) provides expanded lung cancer risk information that can further guide next steps for patients with lung nodules (Press release, Veracyte, MAY 20, 2019, View Source [SID1234536483]).

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Researchers prospectively validated the Percepta GSC on 412 patients with lung nodules who had inconclusive results following bronchoscopy, a common nonsurgical procedure to diagnose lung cancer. Among patients with an "intermediate" pre-test risk of cancer – the group that accounts for the majority of lung nodules – the Percepta GSC demonstrated high accuracy when it down-classified patients to "low risk" for cancer (negative predictive value of 91 percent). The test also had a positive predictive value (PPV) of 65 percent when it up-classified patients to "high risk" for cancer. The American College of Chest Physicians recommends patients with a cancer risk of 65 percent or greater undergo surgical treatment.

The new findings will be shared this evening at a company event being held during the American Thoracic Society 2019 International Conference(ATS 2019) in Dallas.

"Determining whether lung nodules are benign or cancerous is often difficult, which can lead to unnecessary invasive procedures and treatment delays," said Giulia C. Kennedy, Ph.D., chief scientific and medical officer of Veracyte. "With its ability to both down-classify and up-classify patients with inconclusive lung nodules, the Percepta GSC should help physicians avoid invasive biopsies in patients who are at low risk of lung cancer, while helping to guide intervention steps for those at high risk."

The Percepta GSC utilizes novel "field of injury" science – which identifies genomic changes associated with lung cancer in current or former smokers using a simple brushing of the person’s airway. The test is also Veracyte’s third clinical classifier to be developed on the company’s novel RNA whole-transcriptome sequencing and machine learning platform. Veracyte plans to begin making the Percepta GSC available to physicians by the middle of 2019.

"We are excited to unveil our Percepta GSC, which we believe will improve diagnosis and treatment decisions for patients undergoing evaluation for lung cancer," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "Additionally, we believe that moving the Percepta classifier to our RNA whole-transcriptome sequencing platform – together with our Afirma and Envisia classifiers – will provide operational efficiencies and a robust foundation for continued innovation. This includes our development of the first nasal swab test for early lung cancer detection."

Lung cancer is the leading cause of cancer deaths worldwide. In the United States, lung cancer causes more than 154,000 deaths each year – more than the next three most prevalent cancers combined. Because lung cancer is difficult to diagnose before it has metastasized, only 16 percent of cases are detected at an early stage, when the disease is more treatable. Lung cancer’s five-year survival rate is only 18 percent, much lower than that of other common cancers. Approximately 80 percent of lung cancer deaths are caused by smoking. Veracyte estimates that approximately 350,000 bronchoscopies are currently performed each year to evaluate suspicious lung nodules for cancer and that up to 70 percent of these produce inconclusive results.

Also during the ATS 2019 conference, Kevin Flaherty, M.D., of the University of Michigan Health System, will present clinical utility data demonstrating that Veracyte’s Envisia Genomic Classifier improves diagnosis of idiopathic pulmonary fibrosis (Session D12, Abstract A5837). These data were published online in The Lancet Respiratory Medicine on April 1, 2019.

On May 20, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that ELZONRIS (tagraxofusp) clinical data in blastic plasmacytoid dendritic cell neoplasm (BPDCN), chronic myelomonocytic leukemia (CMML), and myelofibrosis (MF) have been selected for poster presentations at the upcoming 24thCongress of the European Hematology Association (EHA) (Free EHA Whitepaper), to be held from June 13-16, 2019, in Amsterdam (Press release, Stemline Therapeutics, MAY 20, 2019, View Source [SID1234536482]).

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Details on the presentations are as follows:

Results from Ongoing Phase 1/2 Clinical Trial of Tagraxofusp (SL-401) in Patients with Relapsed/Refractory Chronic Myelomonocytic Leukemia (CMML)

• Abstract: PF672
• Presenter: Mrinal M. Patnaik, MBBS; Mayo Clinic
• Date: Friday, June 14
• Time: 5:30-7:00 PM CET
Results from Ongoing Phase 1/2 Clinical Trial of Tagraxofusp (SL-401) in Patients with Intermediate, or High Risk, Relapsed/Refractory Myelofibrosis (MF)

• Abstract: PF668
• Presenter: Naveen Pemmaraju, MD; The University of Texas MD Anderson Cancer Center
• Date: Friday, June 14
• Time: 5:30-7:00 PM CET
Results of Pivotal Phase 2 Clinical Trial of Tagraxofusp (SL-401) in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

• Abstract: PS1063
• Presenter: Naveen Pemmaraju, MD; The University of Texas MD Anderson Cancer Center
• Date: Saturday, June 15
• Time: 5:30-7:00 PM CET
Please visit the BPDCN disease awareness booth (#1351) during the 24thCongress of EHA (Free EHA Whitepaper).

About ELZONRIS
ELZONRIS (tagraxofusp-erzs), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About CD123
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.