On November 7, 2016 TG Therapeutics, Inc. (NASDAQ:TGTX) reported its financial results for the third quarter ended September 30, 2016 and recent company developments (Press release, TG Therapeutics, NOV 7, 2016, View Source [SID1234516778]).

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Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer, stated, "The third quarter was a pivotal one for the company, particularly with the amendment of the GENUINE Phase 3 trial that will enable the rapid conclusion of the study while also maintaining the possibility for accelerated approval for TG-1101 in combination with ibrutinib. Importantly, with GENUINE enrollment wrapping up, we can dedicate our resources and focus to our proprietary UNITY program, which we believe offers the potential for highly active, well-tolerated therapy with certain pricing advantages over competitors. As a result, we see our value proposition rising as the concern around pharmaceutical pricing continues to grow." Mr. Weiss continued, "We see important value creating milestones over the next 12 months, including completion of enrollment of GENUINE expected by year end 2016 to be followed by top line data in the first half of 2017. During the course of 2017, we should also report early data from our UNITY-DLBCL study and our MS pivotal program should take full form supported by B-cell depletion data from our ongoing Phase 2 study in RRMS. With all these events lining up, we believe 2017 will be our most impactful year to date."

Third Quarter and Recent Highlights

ASH 2016: The Company looks forward to the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting where data presentations will include three oral presentations and three poster presentations. All clinical presentations will highlight the safety and efficacy for combinations of TGR-1202 with novel targeted agents including oral presentations with TGR-1202 in combination with ibrutinib and TGR-1202 in combination with ruxolitinib.

TGR-1202 Preclinical Differentiation: An October publication in Blood presented preclinical data describing the synergy of TGR-1202 with carfilzomib and the unique effects of the combination to silence c-Myc in various preclinical lymphoma and myeloma models. In addition, the publication described TGR-1202’s unique complimentary mechanism of inhibiting the protein kinase casein kinase-1 (CK1) epsilon, which may contribute to the silencing of c-Myc and explain TGR-1202’s clinical activity in aggressive lymphoma.

TGR-1202 + Carfilzomib: Based on the preclinical work on TGR-1202 published in Blood , a Phase 1/2 study of TGR-1202 and Carfilzomib in patients with relapsed or refractory lymphoma was launched.

GENUINE Study Amendment: The Company amended the ongoing GENUINE Phase 3 Clinical Trial by revising the primary endpoint to Overall Response Rate (ORR), with enrollment expected to be completed before year end 2016 and top-line data available in first half of 2017. The study is well powered to detect a difference in ORR, which, if successful, would potentially support a filing for accelerated approval.

Clinical Data Presentation of TG-1101 in NMO: During the 32nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis, the Company presented clinical data from the Phase Ib study of TG-1101 in patients with NMO with TG-1101 demonstrating rapid and effective depletion of B-cells during acute NMO relapse. The Company has an on-going Phase 2 study of TG-1101 in multiple sclerosis.

Orphan Drug Designations: The Company received Orphan Drug Designation for TGR-1202 for treatment of Chronic Lymphocytic Leukemia and for TG-1101 for treatment of Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD).

UNITY-DLBCL: Patient enrollment began for the registration-directed UNITY-DLBCL Phase 2b clinical study evaluating TG-1101 and TGR-1202 in combination compared to TGR-1202 monotherapy in patients with advanced relapsed/refractory DLBCL.
Key Remaining 2016 Milestones

Complete enrollment into the GENUINE Phase 3 clinical trial
Aggressively enroll into the Company’s registration directed trials, including the UNITY-CLL Phase 3, and the UNITY-DLBCL Phase 2b
Continue enrollment into the Phase 2 clinical trial in Multiple Sclerosis
Present clinical data from a variety of Phase 1 and 2 clinical trials at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2016, held in San Diego, CA
Financial Results for the Third Quarter 2016

Cash Position: Cash, cash equivalents, investment securities, and interest receivable were $60.7 million as of September 30, 2016.

R&D Expenses: Research and development (R&D) expenses were $21.8 million and $46.9 million for the three and nine months ended September 30, 2016, respectively, compared to $11.6 million and $32.5 million for the three and nine months ended September 30, 2015, respectively. Included in research and development expenses for the three and nine months ended September 30, 2016, are $10.2 million and $17.9 million, respectively, of manufacturing and CMC expenses for Phase 3 clinical trials and potential commercialization. The increase in R&D expenses for both the three and nine months ended September 30, 2016, is primarily due to the ongoing clinical development programs and related manufacturing costs for TG-1101 and TGR-1202.

G&A Expenses: General and administrative (G&A) expenses were $3.2 million and $8.1 million for the three and nine months ended September 30, 2016, respectively, as compared to $2.3 million and $13.2 million for the three and nine months ended September 30, 2015, respectively. The period-over-period decrease in G&A expenses from the nine months ended September 30, 2015 relates primarily to non-cash compensation expenses related to equity incentive grants recognized during 2015. G&A expenses for the three months ended September 30, 2016 remained relatively flat compared to the second quarter of 2015, and we expect G&A expenses to remain relatively constant through the fourth quarter of 2016.

Net Loss: Net loss was $24.8 million and $54.6 million for the three and nine months ended September 30, 2016, respectively, compared to a net loss of $13.7 million and $45.3 million for the three and nine months ended September 30, 2015, respectively.

Financial Guidance: The Company believes its cash, cash equivalents, investment securities, and interest receivable of $60.7 million as of September 30, 2016 will be sufficient to fund the Company’s planned operations into the first half of 2018.

On November 7, 2016 DelMar Pharmaceuticals, Inc. (NASDAQ: DMPI) ("DelMar" and the "Company"), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, reported that the Company and its research collaborators have presented data at two scientific conferences hosted by the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (Press release, DelMar Pharmaceuticals, NOV 7, 2016, View Source [SID1234516371]).

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AACR DNA Repair: Tumor Development & Therapeutic Response Conference. On Friday, November 4, 2016, DelMar and its collaborators from the University of British Columbia Prostate Cancer Research Center presented an abstract entitled: "Dissecting the Molecular Mechanism of Dianhydrogalactitol (VAL-083) in Cancer Treatment."
AACR New Horizons in Cancer Research: Delivering Cures through Cancer Research. Also, on Friday, November 4, 2016, DelMar and its collaborators from the University of Texas MD Anderson Cancer Center and the University of British Columbia presented an abstract entitled: "Assessment of dianhydrogalactitol in the treatment of relapsed or refractory non-small cell lung cancer."
The presentations can be accessed on DelMar’s website at View Source

Jeffrey Bacha, DelMar’s chairman & CEO, stated, "We continue to make great strides in understanding how VAL-083 targets cancer cells."

The data demonstrate that, upon treatment, VAL-083 rapidly forms cross-links on the DNA of cancer cells leading to cell cycle arrest in the S/G2 phase and lethal double-strand DNA breaks. This mechanism is distinct from other alkylating agents used in the treatment of cancer such as temozolomide, nitrosoureas or platinum-based chemotherapy. Because VAL-083’s mechanism differs from the others it is not subject to the same resistance mechanisms and therefore may be used to treat patients whose tumors are resistant to other therapies.

"We are leveraging clinical validation from prior NCI-sponsored research with a modern understanding of VAL-083’s unique anti-cancer mechanism to diversify our product development portfolio into new indications such as lung and ovarian cancer," added Mr. Bacha.

About VAL-083
VAL-083 is a "first-in-class," small-molecule chemotherapeutic. In more than 40 Phase I and II clinical studies sponsored by the U.S. National Cancer Institute, VAL-083 demonstrated clinical activity against a range of cancers including lung, brain, cervical, ovarian tumors and leukemia both as a single-agent and in combination with other treatments.

VAL-083 has received an orphan drug designation in Europe for the treatment of malignant gliomas and the U.S. FDA Office of Orphan Products has granted an orphan designation to VAL-083 for the treatment of glioma, medulloblastoma and ovarian cancer.

The Company has completed a successful end of Phase II meeting with the US FDA and plans to advance VAL-083 into a pivotal clinical trial for GBM patients following bevacizumab failure. DelMar presented data from its Phase I/II clinical trial in refractory GBM at the 2016 American Association of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual meeting demonstrating that the median survival of 22 patients receiving an assumed therapeutic dose of VAL-083 (≥20mg/m2) was 8.35 months following bevacizumab (Avastin) failure compared to published literature where survival of approximately two to five months has been reported.

DelMar’s advanced development program will feature a single multi-center randomized Phase III study measuring survival outcomes compared to a "physicians’ choice" control, which, if successful, would serve as the basis for a New Drug Application (NDA) submission for VAL-083. The control arm will consist of a limited number of salvage chemotherapies currently utilized in the treatment of Avastin-failed GBM. The final pivotal trial design will be confirmed with the FDA following further discussions with the Company’s clinical advisors.

In addition to the pivotal trial, DelMar also plans to initiate two separate Phase II clinical trials in earlier-stage GBM patients.

In collaboration with the University of Texas MD Anderson Cancer Center: A non-comparative, biomarker-driven, Phase II study to determine if treatment of MGMT-unmethylated recurrent GBM with VAL-083 or CCNU improves overall survival at 9 months, compared to historical control in bevacizumab naïve patients. (clinicaltrials.gov identifier: NCT02717962)
In collaboration with Sun-Yat Sen University and Guangxi Wuzhou Pharmaceutical (Group) Co.: A single arm Phase II clinical trial to confirm the tolerability of DelMar’s dosing regimen in combination with radiotherapy (XRT) and to explore the activity of VAL-083 in newly diagnosed MGMT-unmethylated GBM patients whose tumors are known to express high levels of MGMT.
DelMar believes that data from these clinical trials, if successful, will form the basis of a new paradigm in the treatment for all GBM patients who fail, or whose tumors exhibit features that make them unlikely to respond to currently available chemotherapy.

In addition to its clinical research in GBM, DelMar believes that its research supports a unique mechanism of action for VAL-083 and that these data support the potential of VAL-083 as a new chemotherapy that may offer improved outcomes in the treatment of GBM and other solid tumors in patients whose tumors have failed or exhibit features that make them resistant to or unlikely to respond to current standard-of-care chemotherapy.

The company and its collaborators from the University of Texas MD Anderson Cancer Center recently presented data at the 11th Biennial Ovarian Cancer Research Symposium demonstrating that VAL-083 was able to overcome cisplatin-resistance in ovarian cancer cell lines with known p53 mutations and displays synergy with both cisplatin and AstraZeneca’s PARP inhibitor Olaparib against ovarian cancer in vitro.

On November 7, 2016 Cascadian Therapeutics (NASDAQ: CASC), a clinical-stage biopharmaceutical company, reported third quarter highlights and reported financial results for the quarter ended September 30, 2016 (Press release, Cascadian Therapeutics, NOV 7, 2016, View Source [SID1234516374]).

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"We continue to make strides on the clinical development front as we advance tucatinib in combination for patients with metastatic HER2+ breast cancer, including those with and without brain metastases," said Scott Myers, CEO of Cascadian Therapeutics. "There is significant need for improved therapies in this disease, and we continue to be impressed by the favorable safety profile observed in the tucatinib combination studies to date when compared with other HER2 therapies, as well as its potential to treat systemic disease and positively impact brain metastases."
Mr. Myers added, "We are looking forward to the presentation of updated clinical data from our ongoing Phase 1b ‘Triplet’ study of tucatinib plus capecitabine and trastuzumab at the San Antonio Breast Cancer Symposium in December, as well as updating everyone on our clinical and regulatory plans later this quarter."
THIRD QUARTER AND RECENT HIGHLIGHTS
Clinical Development

• HER2CLIMB, continues to be on track with enrollment of Phase 2 tucatinib (ONT-380) combination trial. This randomized, double-blind, placebo-controlled study is evaluating the safety and efficacy of tucatinib versus placebo in combination with capecitabine and trastuzumab in late stage HER2+ breast cancer patients, with and without brain metastases, who have previously been treated with a taxane, trastuzumab, pertuzumab and T-DM1. Tucatinib, which has Fast Track designation from the FDA in this setting, is expected to enroll approximately 180 patients at sites in the U.S., Canada and select countries in Western Europe. Previously reported results from the ongoing Phase 1b study of the tucatinib "Triplet" combination demonstrated promising systemic activity, a favorable safety profile and activity against brain metastases, which represents a significant unmet medical need.

• Data presented at ESMO (Free ESMO Whitepaper) 2016 show early evidence of activity of tucatinib combination therapy in patients with cutaneous HER2+ metastases. In a poster presentation, Dr. Alison Conlin, study author and Medical Oncologist, Providence Cancer Center, Portland, OR, presented data on eight patients with
LOGO

HER2+ metastases to the skin who received the maximum tolerated tucatinib dose in combination with capecitabine and/or trastuzumab from the Company’s Phase 1b combination study. Patients had previously received a median of six lines of drug therapy. Responses observed in skin lesions in these patients included one complete response, four partial responses and three patients with stable disease, including one partial response of a patient receiving tucatinib and trastuzumab only.

• Continue to advance novel Chk1 cell cycle inhibitor with goal of conducting IND-enabling studies. Preclinical research has shown that select Cascadian Chk1 inhibitors display cellular potency in in vitro models against Chk1, are active against a diverse range of cancer cell lines, and demonstrate synergistic activity in combination with certain chemotherapeutic drugs.
Corporate Update

• Cascadian’s board of directors has called a Special Stockholder Meeting to vote on a reverse split of the Company’s common stock and a reduction of the authorized common stock. The objective is to make the stock accessible to a wider range of institutional investors, benefiting all stockholders, and ensure that the necessary financial structure is in place to execute product development and other necessary corporate initiatives. The stockholder vote on the proposal will be held on November 18, 2016. For details, see the proxy statement at www.sec.gov/edgar.

THIRD QUARTER 2016 FINANCIAL HIGHLIGHTS

• Cash, cash equivalents and investments totaled $71.6 million as of September 30, 2016, compared to $56.4 million at December 31, 2015, an increase of $15.2 million, or 27.0%. The increase was primarily the result of net proceeds of $43.3 million from the Company’s June 2016 financing offset by cash used to fund operations of $28.1 million.

•
Net loss attributable to common stockholders for the three months ended September 30, 2016 was $11.8 million, or $0.09 per basic and diluted share, compared with a net loss attributable to common stockholders of $4.6 million, or $0.05 per basic and diluted share, for the comparable period in 2015. The increase in net loss attributable to common stockholders for the quarter was primarily due to increases in research and development expenses of $2.2 million primarily due to greater activity related to the development of the Company’s product candidates and increases in general and administrative expenses of $1.4 million primarily due to expenses related to the Company’s Retention Payment Plan and higher professional fees associated with legal and regulatory

compliance. The Company also recognized a non-cash $1.0 million deemed dividend due to the beneficial conversion feature on the Series D convertible preferred stock. In addition, the increase in net loss attributable to common stockholders was due to lower non-cash income from the change in the fair value of the Company’s warrant liability, which was zero for the three months ended September 30, 2016 compared to $2.6 million for the three months ended September 30, 2015. The change in the fair value of warrant liability was due to the expiration of September 2010 warrants, which expired in October 2015.

• Net loss attributable to common stockholders for the nine months ended September 30, 2016 was $49.8 million, or $0.46 per basic and diluted share, compared with a net loss attributable to common stockholders of $23.5 million, or $0.24 per basic and diluted share, for the comparable period in 2015. The increase in net loss attributable to common stockholders for the nine months ended September 30, 2016 was primarily due to the intangible asset impairment charge of $19.7 million during the nine months ended September 30, 2016, which was the result of the mutual termination of the STC.UNM agreement. In addition, the increase in net loss attributable to common stockholders was due to increases in research and development expenses of $3.4 million primarily due to greater activity related to the development of the Company’s product candidates, and increases in general and administrative expenses of $7.5 million primarily related to the retirement and separation agreement that Cascadian entered into with its former chief executive officer in January 2016, expenses related to the Company’s Retention Payment Plan and higher professional fees associated with legal and regulatory compliance. The Company also recognized a non-cash $2.6 million deemed dividend due to the beneficial conversion feature on the Series D convertible preferred stock. The increase in the net loss attributable to common stockholders was partially offset by a $6.9 million tax benefit during the nine months ended September 30, 2016.

Financial Guidance
Cascadian Therapeutics believes the following financial guidance to be correct as of the date provided. Cascadian Therapeutics is providing this guidance as a convenience to investors and assumes no obligation to update it.
Cascadian Therapeutics currently expects cash used in operations in 2016 to be approximately $38.0 million to $40.0 million. With cash, cash equivalents and investments of $71.6 million as of September 30, 2016, Cascadian Therapeutics estimates that its cash, cash-equivalents and investments will be sufficient to fund operations for at least the next 12 months.

On November 7, 2016 Heat Biologics, Inc. (Nasdaq:HTBX), a leader in the development of gp96-based immunotherapies that activate a patient’s immune system to fight cancer, reported that it will present a poster on its ComPACT platform at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, in National Harbor, Maryland, on Friday, November 11th .

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The details are:

Title: Gp96-Ig/Costimulator (OX40L, ICOSL, or 4-IBBL) Combination Vaccine Improves T-cell Priming and Enhances Immunity, Memory and Tumor Elimination

Date and Time: November 11, 2016, 6:15-7:30 PM

Poster Number: 211

On November 7, 2016 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that eleven abstracts, including four oral presentations, supporting the company’s hematology and oncology portfolio will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in San Diego, California, December 3-6, 2016 (Press release, Jazz Pharmaceuticals, NOV 7, 2016, View Source;p=RssLanding&cat=news&id=2220278 [SID1234516378]).

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"The data presentations at ASH (Free ASH Whitepaper) reflect our efforts in advancing our diversified pipeline of programs in hematology and oncology, including rare blood disorders such as acute lymphoblastic leukemia (ALL) and AML, and in complications of hematopoietic stem-cell transplantation (HSCT) such as hepatic VOD," said Karen Smith, M.D., Ph.D., global head of research and development and chief medical officer at Jazz Pharmaceuticals. "Of note, we look forward to sharing a post-hoc sub-analysis of Phase 3 survival data following allogeneic HSCT in older high-risk AML patients that compares CPX-351, also known as Vyxeos, with the standard of care."

The following oral and poster presentations focusing on Defitelio (defibrotide sodium) injection, Erwinaze (asparaginase Erwinia chrysanthemi) and CPX-351(cytarabine and daunorubicin liposome injection) will be presented at ASH (Free ASH Whitepaper).

Defitelio Related Oral and Poster Presentations

Presentation Title
Author
Presentation Number / Date / Time / Location
Timing of Initiation of Defibrotide Post-Diagnosis of Hepatic Veno-Occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome (SOS) Post-Hematopoietic Stem Cell Transplantation (HSCT): Exploratory Age-Group Analysis From an Expanded Access Study

Grupp S, et al.
Oral Presentation 66:
– December 3; 8:45 AM (PT); Manchester Grand Hyatt San Diego, Grand Hall B
– Session 721: Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Post-Transplant Complications
Impact on Outcomes of Baseline Bilirubin in Patients with Hepatic VOD/SOS Receiving Defibrotide Treatment: A Post-Hoc Analysis

Richardson P, et al.
Poster Presentation 2213:
– December 3; 5:30-7:30 PM (PT); San Diego Convention Center, Hall GH
– Session 721: Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Poster I
Treatment of Hepatic VOD/SOS Post-HSCT in Patients With Acute Leukemias: A Subgroup Analysis From the Defibrotide Expanded-Access Program
Richardson P, et al.
Poster Presentation 3412:
– December 4; 6:00-8:00 PM (PT); San Diego Convention Center, Hall GH
– Session 721: Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Poster II
VOD Related Abstract

Abstract Title
Author
Details
Stratification of Allogeneic HSCT Patients by Risk of Developing VOD: A Model for Assigning a Risk Score

Strouse C, et al.
Oral Presentation 983:
– December 5; 3:45 PM (PT); Manchester Grand Hyatt San Diego, Grand Hall B
Session 721: Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Post-Transplant Complications II
Diagnosis of VOD/SOS With or Without Multi-Organ Dysfunction (MOD) After HSCT: Analysis of a Multicenter Chart Review
Doede T, et al.
Online Only Publication;
Abstract 5756
Erwinaze Related Oral and Poster Presentations

Presentation Title
Author
Presentation Date / Time / Location
Population Pharmacokinetic Modeling of Intravenous Asparaginase Erwinia Chrysanthemi: Impact of Varied Infusion Rates on Exposure

Zomorodi K, et al.
Poster Presentation 1631:
– December 3; 5:30-7:30 PM (PT), San Diego Convention Center, Hall GH
– 614: Acute Lymphoblastic Leukemia: Therapy, excluding Transplantation: Poster I
CPX-351 Related Oral and Poster Presentations

Presentation Title
Author
Presentation Date / Time / Location
Analysis of Efficacy by Age for Patients Aged 60–75 With Untreated Secondary Acute Myeloid Leukemia (AML) Treated With CPX-351 Liposome Injection Versus Conventional Cytarabine and Daunorubicin in a Phase III Trial
Medeiros B, et al.
Oral Presentation 902:
– December 5; 3:00 PM (PT); Marriot Marquis San Diego Marina, San Diego Ballroom AB
– Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Clinical Trials of Novel Drugs and Combinations in AML
Survival Following Allogeneic Hematopoietic Cell Transplantation in Older High-Risk Acute Myeloid Leukemia Patients Initially Treated With CPX-351 Liposome Injection Versus Standard Cytarabine and Daunorubicin: Subgroup Analysis of a Large Phase III Trial
Lancet J, et al.
Oral Presentation 906:
– December 5; 4:00 PM (PT); Marriot Marquis San Diego Marina, San Diego Ballroom AB
– Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Clinical Trials of Novel Drugs and Combinations in AML
Enhanced Cytarabine and Daunorubicin Population Pharmacokinetics When Administered as CPX-351: A Novel Liposomal Formulation Not Requiring Dose Reduction for Mild Renal or Hepatic Dysfunction

Nikanjam M, et al.
Poster Presentation 3955:
– December 5; 6:00 – 8:00 PM (PT); San Diego Convention Center, Hall GH
– Session 604: Molecular Pharmacology and Drug Resistance in Myeloid Diseases: Poster III

AML Related Poster

Presentation Title
Author
Presentation Number / Date / Time / Location
Burden of Acute Myeloid Leukemia (AML) Among Older Newly-Diagnosed Patients
Sacks N, et al.
Poster Presentation 4780:
– December 5; 6:00 – 8:00 PM (PT); San Diego Convention Center, Hall GH
– Session 904: Outcomes Research—Malignant Conditions: Poster III
Additionally, one Jazz-sponsored Investigator Initiated Research poster presentation focusing on CPX-351 as an investigational agent for the treatment of AML will be presented at ASH (Free ASH Whitepaper).

Presentation Title
Author
Presentation Number / Date / Time / Location
CPX-351 for the Treatment of High-Risk Patients (pts) With Acute Myeloid Leukemia (AML)
Assi, R, et al.

Poster Presentation 4047:
– December 5; 6:00 – 8:00 PM (PT); San Diego Convention Center, Hall GH
– Session 616: Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III
Full details of the ASH (Free ASH Whitepaper) 2016 annual meeting can be found here (View Source) and abstracts can be found here (View Source).

About Defitelio1
In the U.S., Defitelio (defibrotide sodium) injection 80mg/mL received 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.

Defitelio is contraindicated in patients currently taking anticoagulants or fibrinolytics and in patients who are allergic to Defitelio or any of its ingredients. Defitelio may increase the risk of bleeding and should be withheld or stopped if significant bleeding occurs. Patients should be monitored for allergic reactions, especially if there is a history of previous exposure to Defitelio. The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds.

Please see full Prescribing Information for Defitelio. (View Source)

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)

About VOD
HSCT is an aggressive, potentially curative procedure to treat patients with malignant and non-cancerous hematologic disorders such as leukemia, lymphoma and aplastic anemia, and congenital immunodeficiency and autoimmune disorders.2 VOD is a rare complication of HSCT, which occurs in approximately 9-14% of HSCT patients.3,4 Hepatic VOD, also known as SOS, is an early and life-threatening complication affecting the sinusoidal endothelial cells of the liver, which can typically occur within the first 21 days following HSCT.4,5 Hepatic VOD progresses to multi-organ dysfunction in approximately 30-50% of cases.5 VOD with multi-organ dysfunction (MOD) is associated with an overall mortality (death) rate of 84%.3 MOD is characterized by the presence of renal or pulmonary dysfunction.6,7 VOD is often characterized by sudden weight gain, hepatomegaly (abnormally enlarged liver), and elevated bilirubin.6,7

About Erwinaze
Erwinaze (asparaginase Erwinia chrysanthemi) is currently approved in the U.S. for administration via intramuscular injection or via intravenous infusion in conjunction with chemotherapy. It is indicated as a component of a multi-agent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia (ALL) who have developed hypersensitivity to E. coli-derived asparaginase.8 Erwinaze is derived from the bacterium Erwinia chrysanthemi and is therefore immunologically distinct from E. coli-derived asparaginase and suitable for patients with hypersensitivity to E. coli-derived treatments9. Outside of the U.S., Erwinaze is sold under the name Erwinase. Please consult local labeling for product information specific to your country.

Erwinaze is contraindicated in patients who have had serious allergic reactions to Erwinaze, or had serious swelling of the pancreas, serious blood clots, or serious bleeding with past L-asparaginase treatment. Erwinaze should be discontinued if any of the following occur: serious allergic reactions, including a feeling of tightness in the throat, unusual swelling/redness in the throat and/or tongue, or trouble bleeding; or severe inflammation of the pancreas. Glucose intolerance has been reported, which in some cases may be irreversible. If blood clots of bleeding occur, discontinue Erwinaze until symptoms resolve. The most common side effects of Erwinaze are allergic reactions, too much sugar in the blood, fever, swelling of the pancreas, local reactions (swelling, rash, etc. where the needle entered the skin), vomiting, nausea, blood clots, liver problems, stomach pain/discomfort, and diarrhea. Please see full Prescribing Information for Erwinaze. (View Source)

About Vyxeos (CPX-351)
CPX-351 (cytarabine and daunorubicin liposome injection) is an investigational product being evaluated for the treatment of AML and is a combination of cytarabine and daunorubicin encapsulated within a nano-scale liposome at a 5:1 molar ratio. The proposed trade name, Vyxeos, is conditionally approved by the FDA and is subject to confirmation upon approval of the NDA. CPX-351 was granted orphan drug status by the FDA and the European Commission for the treatment of acute myeloid leukemia. CPX-351 was granted Breakthrough Therapy Designation for the treatment of adults with therapy-related AML or AML with myelodysplasia-related changes and was also granted Fast Track designation by the FDA for the treatment of older patients with secondary AML. On October 3, 2016 Jazz announced the initiation of a rolling submission of a New Drug Application (NDA) to the FDA, seeking marketing approval of CPX-351 for the treatment of AML.

About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a rapidly progressing and life-threatening blood cancer that rises in frequency with age.16 The American Cancer Society estimates that there will be 19,950 new cases of AML and 10,430 deaths from AML in the U.S. in 2016.10 In the European Union, the number of new cases is estimated to be 20,100 in 2016.11

The median age at diagnosis is 67 and with rising age there is progressive worsening of prognosis.10,12 Advancing age is associated with increasing risk of specific chromosomal/mutational changes and risk of pre-malignant marrow disorders which give rise to more aggressive and less responsive forms of AML.13,14 As patients age there is also reduced tolerance for intensive chemotherapy.15 As a consequence, advances in supportive care, intensive chemotherapy, and bone marrow transplantation have primarily benefitted younger patients with approximately one third of patients 18-60 years of age achieving cure.13,15 Older patients have not achieved higher rates of cure or improved upon a 5-year survival rate of 10-20% in spite of 40 years of research.15,16