On April 23, 2018 Pfizer Inc. (NYSE:PFE) reported that the European Commission has approved MYLOTARG (gemtuzumab ozogamicin) in combination with daunorubicin and cytarabine for the treatment of patients age 15 years and above with previously untreated, de novo, CD33-positive acute myeloid leukemia (AML), except acute promyelocytic leukemia (APL) (Press release, Pfizer, APR 23, 2018, View Source [SID1234525590]). MYLOTARG is the first and only AML therapy approved in the European Union (EU) that targets CD33, an antigen expressed on AML cells in up to 90% of patients.1,2,3

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"The marketing authorization of MYLOTARG provides a much-needed treatment option offering renewed hope for many acute myeloid leukemia patients in Europe," said Andreas Penk, M.D., regional president, Pfizer Oncology. "In clinical trials, the addition of MYLOTARG to standard chemotherapy resulted in deeper, more durable remission, thus providing an additional treatment option with the potential to prevent relapse for these patients."

AML is a rapidly progressing, life-threatening blood and bone marrow cancer.4 If left untreated, patients with AML will die within months, if not weeks, of their disease. AML is the most common type of acute leukemia in adults and accounts for approximately 80% of all cases of acute leukemia.5About 16,800 people are expected to be newly diagnosed with AML in Europe annually.6 The goal of AML treatment is for the patient to achieve a complete, prolonged remission. Longer periods of remission prior to relapse are associated with better long-term outcomes for patients. Thus, medicines that delay the time until the disease comes back and extend life can provide meaningful clinical benefit.

"I am thrilled that MYLOTARG will be available soon in Europe as a first-line treatment for patients with acute myeloid leukemia," said Doctor Sylvie Castaigne, Professeur des Universités, Université de Versailles – Saint Quentin, Praticien Hospitalier, Centre Hospitalier de Versailles, and lead investigator of the ALFA-0701 study. "This important milestone is a result of close collaboration between Pfizer and clinical investigators around the world, particularly the ALFA investigators in France, who believed in the promise of this therapy. We thank all of the investigators, nurses and patients who participated in these studies."

The European Commission’s approval of MYLOTARG was based on data from an investigator-led, Phase 3 randomized, open-label study (ALFA-0701) in previously untreated, de novo patients. MYLOTARG received approval by the U.S. Food and Drug Administration in September 2017 for adults with newly diagnosed CD33-positive acute myeloid leukemia (AML), and adults and children 2 years and older with relapsed or refractory CD33-positive AML.

Pfizer is advancing a broad range of therapies that leverage multiple pathways and mechanisms of action (MOAs) to address acute and chronic leukemias, myeloproliferative disorders and lymphomas. Pfizer currently has four marketed therapies for hematologic cancers worldwide as well as several therapies in clinical development. Pfizer is also forging collaborations with a diversity of industry, academic and community partners to study multiple paths to advancing treatment. By working together, Pfizer and its partners aim to overcome the challenges of hematologic cancers and deliver meaningful benefits to patients.

Indication for MYLOTARG (gemtuzumab ozogamicin) in the EU

MYLOTARG is approved in combination with daunorubicin and cytarabine for the treatment of patients age 15 and above with previously untreated, de novo, CD33-positive acute myeloid leukemia (AML), except acute promyelocytic leukemia (APL).

IMPORTANT MYLOTARG (gemtuzumab ozogamicin) SAFETY INFORMATION in the EU

The overall safety profile of MYLOTARG is based on data from patients with acute myeloid leukemia from the combination therapy study ALFA-0701, monotherapy studies, and from post-marketing experience.

Hepatotoxicity, including life-threatening, and sometimes fatal hepatic failure and VOD/SOS have been reported in patients treated with MYLOTARG. Other special warnings and precautions include myelosuppression and infusion-related reactions.

In the combination therapy study ALFA-0701, clinically relevant serious adverse reactions were hepatotoxicity, including VOD/SOS (3.8%), hemorrhage (9.9%), severe infection (41.2%), and tumour lysis syndrome (1.5%). In monotherapy studies, clinically relevant serious adverse reactions also included infusion related reactions (2.5%), thrombocytopenia (21.7%), and neutropenia (34.3%).

The most common adverse reactions (> 30%) in the combination therapy study were hemorrhage and infection. In monotherapy studies the most common adverse reactions (> 30%) included pyrexia, nausea, infection, chills, hemorrhage, vomiting, thrombocytopenia, fatigue, headache, stomatitis, diarrhea, abdominal pain, and neutropenia.

The most frequent (≥ 1%) adverse reactions that led to permanent discontinuation in the combination therapy study were thrombocytopenia, VOD, hemorrhage and infection. The most frequent (≥ 1%) adverse reactions that led to permanent discontinuation in monotherapy studies were infection, hemorrhage, multi-organ failure, and VOD.

The EU Summary of Product Characteristics (SmPC) will be available at View Source

About MYLOTARG (gemtuzumab ozogamicin)

MYLOTARG is an antibody-drug conjugate (ADC) composed of the cytotoxic agent calicheamicin, attached to a monoclonal antibody (mAB) targeting CD33, an antigen expressed on the surface of myeloblasts in up to 90 percent of AML patients.1,2,3When MYLOTARG binds to the CD33 antigen on the cell surface it is absorbed into the cell and calicheamicin is released causing cell death.2,3

MYLOTARG was approved by the U.S. Food and Drug Administration in September 2017 for adults with newly diagnosed CD33-positive acute myeloid leukemia (AML), and adults and children 2 years and older with relapsed or refractory CD33-positive AML.

MYLOTARG originates from a collaboration between Pfizer and Celltech, now UCB. Pfizer has sole responsibility for all manufacturing, clinical development and commercialization activities for this molecule.

Pfizer also collaborated with SFJ Pharmaceuticals Group on the registrational program for MYLOTARG.

On April 23, 2018 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported that it will report first quarter financial results after the Nasdaq market closes on Monday, April 30, 2018 (Press release, Neurocrine Biosciences, APR 23, 2018, View Source;p=RssLanding&cat=news&id=2344001 [SID1234525589]). Neurocrine will then host a conference call and webcast to discuss its financial results and provide a Company update that day at 2:00 p.m. Pacific Time (5:00 p.m. Eastern Time).

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Participants can access the live conference call by dialing 877-876-9176 (US) or 785-424-1667 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine’s website under Investors at View Source A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for one month.

On April 23, 2018 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology, reported that two scientific abstracts have been accepted for presentation at the 21st Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper), taking place May 16-19, 2018, in Chicago (Press release, Intellia Therapeutics, APR 23, 2018, View Source [SID1234525587]).

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The first presentation will share information relating to Intellia’s lead in vivo program, for transthyretin amyloidosis. The data being presented include results from an ongoing collaboration with researchers at the University of Porto in Portugal. The second presentation will focus on Intellia’s ongoing research collaboration with IRCCS Ospedale San Raffaele in Italy to develop CRISPR-edited, T-cell therapies to address hard-to-treat cancers. Intellia will also participate in an education session on RNA therapeutics.

Details of Intellia’s presentations are as follows:

"Rescue of Amyloid Deposition Phenotype after Single-Treatment CRISPR/Cas9 Gene Editing in a Humanized Mouse Model of TTR Amyloidosis"
Session: Neurologic Diseases (Including Ophthalmic and Auditory Diseases) I
Session date/time:Wed., May 16, 2018, 5:30-7:30 p.m. CT
Location: Stevens Salon C, D
"Hunting WT1-Specific T-Cell Receptors for TCR Gene Editing for Acute Myeloid Leukemia"
Session: Cancer – Immunotherapy, Cancer Vaccines I
Session date/time:Wed., May 16, 2018, 5:30-7:30 p.m. CT
Location: Stevens Salon C, D
"Lipid Nanoparticle-Based RNA Delivery: At the Intersection of Chemistry and Immunology"
Presenter: Jonathan Finn, Ph.D., executive director, Platform Biology
Session: Education Session 401 – RNA Therapeutics
Presentation date/time:Sat., May 19, 2018, 9-9:30 a.m. CT
Location: Salon A-1
In addition, the following Intellia collaborators will highlight aspects of their research efforts with the Company:

"Clinical Gene Editing Programs"
Presenter: Beverly Davidson, Ph.D., chief scientific strategy officer, Children’s Hospital of Philadelphia (CHOP); director, Raymond G. Perelman Center for Cellular and Molecular Therapeutics, CHOP; Arthur V. Meigs Chair in Pediatrics, CHOP; and professor, Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania
Session: Pre-Meeting Program – Gene Editing Workshop
Presentation date/time:Tue., May 15, 11 a.m.-12 p.m. CT
Location: Continental B
"TCR Gene Transfer and TCR Gene Editing"
Presenter: Chiara Bonini, M.D., Ph.D., full professor, Università Vita-Salute San Raffaele; deputy director, Division of Immunology, Transplantation and Infectious Diseases; and head, Experimental Hematology Unit, Ospedale San Raffaele, Italy
Session: 100 Immune Responses to Cell and Gene Therapies, Mechanisms, Biomarkers and Therapeutic Interventions
Presentation date/time:Wed., May 16, 2018, 8-8:30 a.m. CT
Location: International Ballroom North
Abstracts will become available on the ASGCT (Free ASGCT Whitepaper) website on Mon., April 30th, at 11 a.m. CT.

On April 23, 2018 Diplomat Pharmacy, Inc. (NYSE: DPLO), reported it will release its first-quarter 2018 operating results after market close Monday, May 7, with a conference call to follow at 5 p.m. ET (Press release, Diplomat Speciality Pharmacy, APR 23, 2018, View Source [SID1234525586]).

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Shareholders and interested participants can listen to a live broadcast by calling 833.286.5805 (647.689.4450 for international callers) and entering participant code 5178318, starting about 15 minutes before the call. A live webcast of the conference call will be available on the investor relations section of Diplomat’s website at ir.diplomat.is. The site will host an audio recording and supplemental investor information for 90 days.

On April 23, 2018 Castle Biosciences, Inc., a provider of molecular diagnostics to improve cancer treatment decisions, reported presentation of results demonstrating that the use of the DecisionDx-Melanoma gene expression profile (GEP) test with American Joint Committee on Cancer (AJCC) staging can improve accuracy of recurrence and metastasis risk for patients with localized cutaneous melanoma (Press release, Castle Biosciences, APR 23, 2018, View Source [SID1234525585]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The study titled, "Improved identification of high-risk, Stage I-II cutaneous melanomas with the combination of American Joint Committee on Cancer staging and a 31-gene expression profile test result," was presented as a poster at the 3rd Annual Real World Dermatology for Physician Assistants and Nurse Practitioners conference, held April 20-22, 2018 in Orlando, Florida.

"In this large Stage I-II melanoma study population, the DecisionDx-Melanoma test improved risk prediction when used in combination with AJCC risk assessment and showed independent prognostic value," commented study co-author Robert W. Cook, PhD, Vice President, Medical Affairs and R&D at Castle Biosciences. "Providing this information for risk assessment is important to help guide patient management decisions for improved patient outcomes."

Study Background

For patients with cutaneous melanoma, accurate assessment of recurrence risk is important to guide management plans including imaging based surveillance, follow-up frequency and sentinel lymph node biopsy recommendations that can lead to early detection of metastatic disease. National guidelines suggest surveillance plans based on AJCC stage, with Stage I-IIA considered low risk and Stage IIB-IV considered high risk. The DecisionDx-Melanoma GEP test accurately and independently predicts risk of recurrence and metastasis, classifying patients as Class 1A (lowest risk) Class 1B/2A (lower or intermediate risk) or Class 2B (highest risk).

This study of 485 Stage I and II patients from a multicenter cohort with long-term outcomes assessed the use of AJCC staging combined with results from the DecisionDx-Melanoma GEP test to improve recurrence risk prediction.

Key Study Findings:

Patients who were classified as high risk by both AJCC staging (Stage IIB-C) and the GEP test (Class 2B) had significantly lower 5-year recurrence-free survival (RFS 33.4%), distant metastasis-free survival (DMFS 49.5%) and melanoma specific survival (MSS 86.7%) compared to those identified as low risk by both methods (RFS 96.1%, DMFS 97.3%, MSS 99.6%, p<0.0001 for all comparisons).
Importantly, patients who were assessed as low risk using AJCC staging (Stage I-IIA) but high risk using the GEP test also demonstrated significantly worse outcomes (RFS 60.9%, DMFS 75.8%, MSS 85.9%) compared to patients who were assessed as low risk using both methods (p<0.0001 for all comparisons).
Multivariate Cox regression analysis indicated that both GEP high risk and AJCC high risk were significant and independent predictors of RFS (GEP HR 6.8; AJCC HR 2.98, p<0.0001, both groups) and DMFS (GEP HR 8.5; AJCC HR 2.5, p<0.001, both groups). For MSS, GEP Class 2B was the only significant predictor (GEP HR 43.8, p<0.001; AJCC HR 1.04, p<0.94).
Clinical Impact of DecisionDx-Melanoma Test

A second poster titled, "Clinical impact of a 31-gene expression profile test for cutaneous melanoma patients: a review of clinical utility studies," was also presented at the conference.

The poster highlights key findings across 5 published clinical utility studies, including:

In prospective and retrospective multicenter clinical utility studies, the inclusion of the DecisionDx-Melanoma test in risk assessment resulted in significant differences in follow-up and surveillance when comparing low- and high-risk patients.
Findings across multiple clinical impact studies show that incorporation of the GEP test consistently impacts clinical management decisions for approximately 1 in 2 patients tested.
Use of the DecisionDx-Melanoma GEP test in combination with conventional staging methods can help develop a more efficient and individualized follow-up plan based on clinical factors and tumor biology.
About DecisionDx-Melanoma
The DecisionDx-Melanoma test uses tumor biology to predict individual risk of melanoma recurrence and sentinel lymph node positivity independent of traditional factors. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in three multicenter studies that have included 690 patients and have demonstrated consistent results. Performance has also been confirmed in four prospective studies including 702 patients. The consistent high performance and accuracy demonstrated in these studies, which combined have included over 1,300 patients, provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results. Prediction of the likelihood of sentinel lymph node positivity has also been validated in two prospective multicenter studies which included over 1,400 patients. Clinical impact has been demonstrated in multicenter and single-center studies showing that test results impact clinical management decisions for one of every two patients tested. More information about the test and disease can be found at www.SkinMelanoma.com.