1stOncology™: Cancer Intelligence Service – Page 14480 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

National Institute for Health and Care Excellence (NICE) Recommends Jazz Pharmaceuticals’ Vyxeos® (Daunorubicin and Cytarabine) for Adults with Specific Types of Secondary Acute Myeloid Leukaemia (AML)

On November 8, 2018 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the National Institute for Health and Care Excellence (NICE) has published a Final Appraisal Determination (FAD) recommending Vyxeos 44 mg/100 mg powder for concentrate for solution for infusion for routine use on the National Health Service (NHS) in England and Wales for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC)1 – two types of secondary AML (Press release, Jazz Pharmaceuticals, NOV 8, 2018, View Source;p=RssLanding&cat=news&id=2376093 [SID1234530972]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

Schedule Your 30 min Free Demo!

"This is the first new chemotherapy in forty years for adults with specific types of newly diagnosed secondary AML, a particularly aggressive cancer that typically affects older people and has a high mortality rate," said Dr. Nigel Russell, Professor of Haematology, Faculty of Medicine & Health Sciences at the University of Nottingham. "I am pleased that NICE has recognised the value of this medicine for adults with secondary AML. In time, it is expected to become the standard of care for this specific group of older AML patients."

Patients diagnosed with t-AML or AML-MRC have a very poor prognosis and have the poorest survival of all AML diagnostic subgroups.2,3 Thus they are classified as having high risk disease because of these poor outcomes. In the UK, the expected number of these high-risk AML cases is 680 per year.4 Its incidence increases with age and accounts for approximately 25% of all AML cases in the UK.4

"A diagnosis of acute myeloid leukaemia can have a huge emotional impact on the lives of patients, as well as their family and friends," said Zack Pemberton-Whiteley, Patient Advocacy Director at Leukaemia Care. "We welcome the decision to recommend this treatment for adults with high-risk AML. Leukaemia Care has been working with NICE to enable patients to access this important new medicine, where options have been previously limited."

"Jazz is delighted that Vyxeos will now be made routinely available on the NHS in England and Wales as people with therapy-related AML or AML with myelodysplasia-related changes have had limited treatment options until now," said Iain McGill, senior vice president, Europe and Rest of World at Jazz Pharmaceuticals. "We believe that it is a meaningful medicine for patients with this rapidly progressing and life-threatening blood cancer."

Vyxeos is an advanced liposomal formulation that delivers a synergistic molar ratio of daunorubicin and cytarabine. It is the first chemotherapy to demonstrate a significant overall survival advantage versus the current treatment standard, 7+3 chemotherapy, in a Phase 3 study of older adult patients with newly diagnosed t-AML or AML-MRC,5 and the first chemotherapy treatment option specifically for people who have these types of high-risk AML.

Notes to Editors

About Vyxeos
It is an advanced liposomal formulation that has been shown in vitro to deliver a synergistic combination of daunorubicin and cytarabine to leukaemia cells for a prolonged period of time. Based on data in animals, the liposomes accumulate and persist in high concentration in the bone marrow, where they are preferentially taken up intact by leukaemia cells.6 It is the first product developed with the company’s proprietary CombiPlex platform, which enables the consideration and design of various combinations of therapies.

It received Orphan Drug Designation (ODD) by the European Commission in January 2012 with retention of the ODD reaffirmed in July 2018 following assessment by the Committee for Orphan Medicinal Products (COMP), and by the U.S. Food and Drug Administration (FDA) in September 2008 for the treatment of AML. It received Promising Innovative Medicine (PIM) designation from the Medicines and Healthcare Products Regulatory Agency in the United Kingdom. In August 2018, the European Commission approved it for the treatment of adults with newly diagnosed t-AML or AML-MRC in all European Union Member States, as well as Iceland, Norway and Liechtenstein.

View the Summary of Product Characteristics here.

About CombiPlex
The CombiPlex proprietary technology enables the design and rapid evaluation of various combinations of therapies to deliver enhanced anti-cancer activity. The CombiPlex technology seeks to identify the most synergistic ratio of drugs in vitro and fix this ratio in a nano-scale delivery complex that maintains the synergistic combination after administration. CombiPlex utilizes two proprietary nano-scale delivery platforms: liposomes to control the release and distribution of water-soluble drugs and drugs that are both water- and fat-soluble (amphipathic), and nanoparticles to control the release and distribution of non-water-soluble (hydrophobic) drugs.

About AML
Acute myeloid leukaemia (AML) is a blood cancer that begins in the bone marrow, which produces most of the body’s new blood cells. AML cells crowd out healthy cells and move into the bloodstream to spread cancer to other parts of the body.7 The median age at diagnosis is 72 years old, with rising age associated with a progressively worsening prognosis.4 There is also a reduced tolerance for intensive chemotherapy as patients age.8 Patients with t-AML or AML-MRC have the lowest survival rates compared with people with other forms of leukaemia.2,3 A haematopoietic stem cell transplant (HSCT) may be a curative treatment option for patients.9

Intensity Therapeutics Highlights INT230-6 Data in Advanced Solid Tumors at Society for Immunotherapy of Cancer’s (SITC) 33rd Annual Meeting

On November 8, 2018 Intensity Therapeutics, Inc., a clinical-stage biotechnology company developing proprietary immune cell-activating cancer treatments, reported additional data from a Phase 1/2 clinical study of INT230-6, the Company’s novel lead product candidate designed for direct intratumoral injection, and preclinical research highlighting the Company’s proprietary DfuseRxSM technology were presented in a poster session at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 33rd Annual Meeting in Washington, D.C (Press release, Intensity Therapeutics, NOV 8, 2018, View Source [SID1234530971]). INT230-6 is comprised of two proven, potent anti-cancer agents and a unique molecule that causes rapid drug dispersion throughout tumors and diffusion into cancer cells.

The presenting author and a study investigator, Anthony Olszanski, MD, Vice Chair of the Department of Hematology/Oncology and Director of the Phase 1 Developmental Therapeutics Program at Fox Chase Cancer Center, said, "We are pleased to share post-treatment imaging of a patient with heavily pretreated cutaneous squamous cell carcinoma who had two deep tumors in the upper arm. Following treatment with INT230-6, scans revealed increased tumor necrosis. In addition, a patient with a chordoma achieved excellent tumor reduction, with some non-injected lesions also decreasing in size, following treatment with INT230-6 as monotherapy, even when administered at low doses. These encouraging results, in addition to a very promising safety profile, support the continued evaluation of INT230-6 in patients with advanced solid tumors."

INT230-6 was discovered using Intensity’s DfuseRxSM technology platform. Preclinical research demonstrated the enhanced ability of INT230-6 to saturate murine pancreatic cancer tumors, compared to the drug alone.

"Our preclinical animal data of INT230-6 shows its ability to disperse and saturate a tumor with drug when administered at the proper dose-to-tumor volume ratio," said Lewis H. Bender, Founder and CEO of Intensity Therapeutics. "When dosing intratumorally, proper drug dispersion is critical in order to treat the entire tumor effectively, especially in regions away from the blood vessels that are hypoxic. Data also show that our highly efficient tumor-killing product, INT230-6, induces a strong adaptive immune response to attack untreated lesions and metastases."

Ian B. Walters, MD, Chief Medical Officer of Intensity, added, "The latest data from Intensity’s clinical study evaluating INT230-6 in patients with different types of solid tumors continues to indicate that INT230-6 can be safely injected, even into deep tumors. The vast majority of the active components of INT230-6 stay inside the tumor, and most reports are limited to mild to moderate local discomfort from the injection."

Intensity plans to add more North American clinical sites, as well as international sites, to the Phase 1/2 clinical study of INT230-6. The Company also plans to move into combination arms with an anti-PD-1 antibody and begin Phase 2 expansion cohorts in specific tumor types next year.

About INT230-6

INT230-6, Intensity’s lead product candidate designed for direct intratumoral injection, is comprised of two proven, potent anti-cancer agents and a penetration enhancer molecule that helps disperse the drugs throughout tumors and diffuse into cancer cells. INT230-6 is being evaluated in a Phase 1/2 clinical study (NCT03058289) in patients with various advanced solid tumors. In preclinical studies, INT230-6 eradicated tumors by a combination of direct tumor kill and recruitment of dendritic cells to the tumor micro-environment that induced anti-cancer T-cell activation. Treatment with INT230-6 in in vivo models of severe cancer resulted in substantial improvement in overall survival compared to standard therapies. Further, INT230-6 provided complete responder animals with long-term, durable protection from multiple re-inoculations of the initial cancer and resistance to other cancers. In mouse models, INT230-6 has shown strong synergy with checkpoint blockage, including anti-PD-1 and anti-CTLA4 antibodies. INT230-6 was discovered from Intensity’s DfuseRxSM platform.

About the Phase 1/2 Clinical Study

INT230-6 is being evaluated in a Phase 1/2 clinical study in patients with different types of advanced solid tumor malignancies. The study’s primary objective is to assess the safety and tolerability of multiple intratumoral doses of INT230-6. Secondary assessments are the measurement of injected and bystander tumor responses, and determination of the systemic pharmacokinetic profile of multiple doses of INT230-6’s drug substances after single and then multiple intratumoral injections. Exploratory analysis will characterize patient outcome, as well as evaluate various tumor and anti-tumor immune response biomarkers that may correlate with response. The study includes several adaptive components that will allow for adjustments in patient groups, dosing schedule and dose volumes administered. Data will be used to assess the progression free and overall survival in patients receiving INT230-6. For more information, please visit www.clinicaltrials.gov (NCT03058289).

Spectrum Pharmaceuticals Reports Third Quarter 2018 Pipeline Update and Financial Results

On November 8, 2018 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, reported financial results for the three-month period ended September 30, 2018 (Press release, Spectrum Pharmaceuticals, NOV 8, 2018, View Source [SID1234530968]).

"In Q3 at the World Conference on Lung Cancer, MD Anderson presented poziotinib interim data in a heavily pre-treated population with exon 20 mutations, which demonstrated strong anti-tumor activity against this difficult-to-treat mutation," said Joe Turgeon, President and Chief Executive Officer of Spectrum Pharmaceuticals. "We also actively expanded the poziotinib clinical program on multiple fronts including the initiation of two first-line cohorts in our pivotal ZENITH20 trial and the opening of study sites in Europe. Closing out the year, we expect to receive a response on the poziotinib BTD request and file a BLA with the FDA for our novel G-CSF, ROLONTIS."

Clinical Program Overview:

Poziotinib, an irreversible tyrosine kinase inhibitor targeting EGFR and HER2 mutations:

Updated interim data from the MD Anderson Phase 2 trial in heavily pre-treated, non-small cell lung cancer (NSCLC) patients with exon 20 mutations were presented at the World Conference on Lung Cancer in September.
In evaluable patients with EGFR exon 20 mutations, the confirmed overall response rate (ORR) was 43% and disease control rate was 90%. Median progression free survival (PFS) was 5.5 months (ITT).
In evaluable patients with HER2 exon 20 mutations, the confirmed overall response rate (ORR) was 42% and disease control rate was 83%. Median progression free survival (PFS) was 5.1 months (ITT).
EGFR-related toxicities (including rash, diarrhea, and paronychia) were manageable and required dose reductions in 60% of patients. Discontinuation due to poor tolerance was rare (approximately 3% of patients).
Spectrum submitted a request for Breakthrough Therapy Designation for poziotinib in previously treated metastatic NSCLC with EGFR exon 20 mutations and expects a response from the FDA by the end of 2018.
Spectrum’s Phase 2 ZENITH20 trial studying poziotinib in NSCLC patients with EGFR or HER2 exon 20 insertion mutations is well underway and enrolling in four distinct cohorts.
First-line cohorts in both EGFR and HER2 were initiated in the third quarter of 2018.
Enrollment in the EGFR, previously treated cohort is expected to be completed by the first quarter of 2019.
ROLONTIS (eflapegrastim), a novel long-acting G-CSF:

Spectrum had a positive pre-BLA meeting with the FDA in the third quarter of 2018 and expects to file for a BLA in the fourth quarter of 2018.
Data from the RECOVER Phase 3 study will be presented in a poster session at the San Antonio Breast Cancer Symposium in early December 2018.
Financial Guidance

Spectrum is refining its full year 2018 revenue guidance and is now between $100-$110 million, revised from $95-$115 million. Additionally, Spectrum currently anticipates that its current cash and marketable securities will be sufficient to fund operations into 2020.

Three-Month Period Ended September 30, 2018 (All numbers are approximate)

GAAP Results

Total product sales were $24.6 million in the third quarter of 2018. Product sales in the third quarter included: FOLOTYN (pralatrexate injection) net sales of $11.3 million, EVOMELA (melphalan) for injection net sales of $6.9 million, BELEODAQ (belinostat) for injection net sales of $3.2 million, ZEVALIN (ibritumomab tiuxetan) net sales of $1.5 million, MARQIBO (vinCRIStine sulfate LIPOSOME injection) net sales of $1.1 million, and FUSILEV (levoleucovorin) net sales of $0.6 million.

Spectrum recorded net loss of $68.7 million, or $0.66 loss per basic and diluted share, in the three-month period ended September 30, 2018, compared to net loss of $18.3 million, or $0.22 loss per basic and diluted share, in the comparable period in 2017. Total research and development expenses were $21.1 million in the quarter, as compared to $13.8 million in the same period in 2017. Selling, general and administrative expenses were $19.8 million in the quarter, compared to $18.5 million in the same period in 2017.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $17.5 million, or $0.17 loss per basic and diluted share, in the three-month period ended September 30, 2018, compared to non-GAAP net loss of $9.2 million, or $0.11 loss per basic and diluted share, in the comparable period in 2017. Non-GAAP research and development expenses were $20.2 million, as compared to $13.2 million in the same period of 2017. Non-GAAP selling, general and administrative expenses were $16.6 million, as compared to $16.1 million in the same period in 2017.

Conference Call

Thursday, November 8, 2018 @ 4:30 p.m. Eastern/1:30 p.m. Pacific

Domestic: (877) 837-3910, Conference ID# 3075918

International: (973) 796-5077, Conference ID# 3075918
This conference call will also be webcast. Listeners may access the webcast, which will be available on the investor relations page of Spectrum Pharmaceuticals’ website: www.sppirx.com on November 8, 2018 at 4:30 p.m. Eastern/1:30 p.m. Pacific.

Castle Biosciences to Present at the 2018 Canaccord Genuity Medical Technologies & Diagnostics Forum

On November 8, 2018 Castle Biosciences, Inc., the skin cancer diagnostics company providing molecular diagnostics to improve cancer management decisions, reported that Derek Maetzold, President and CEO, will present at the Canaccord Genuity Medical Technologies & Diagnostics Forum on Thursday, November 15th, 2018 at 3:00 p.m. in New York City (Press release, Castle Biosciences, NOV 8, 2018, View Source [SID1234530967]).

Caladrius Biosciences Reports 2018 Third Quarter Financial Results

On November 8, 2018 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a late-stage therapeutics development biopharmaceutical company with multiple technology platforms targeting select cardiovascular indications and autoimmune disease, reported its financial results for the three ended September 30, 2018, and provides a business update (Press release, Caladrius Biosciences, NOV 8, 2018, View Source [SID1234530965]).

Highlights of the 2018 third quarter and early fourth quarter include:

Continued enrollment in a Phase 2 (SAKIGAKE designated and eligible for early conditional approval) clinical trial in Japan of CLBS12 for the treatment of no-option critical limb ischemia ("CLI"), including completion of enrollment of the five patient Buerger’s disease cohort;

Continued enrollment in a Phase 2 clinical trial using the CD34 cell therapy CLBS14-CMD for the treatment of coronary microvascular dysfunction ("CMD");

Continued follow-up analysis of The Sanford Project: T-Rex Study Phase 2 clinical trial of CLBS03 in type 1 diabetes after completing enrollment and reporting six-month results on 50% of trial subjects in the first quarter of 2018 that concluded the treatment is well-tolerated and non-futile for therapeutic effect; and

Conducted a Type B meeting with the US Food and Drug Administration ("FDA") under the provisions of the RMAT designation for CLBS14-RfA for the treatment of refractory angina to define remaining steps to registration.

"We are pleased with the advancement of our clinical programs during the third quarter. We maintain our previous guidance regarding development milestones and continue to demonstrate efficient management of cash spend," stated Dr. David J. Mazzo, President and CEO. "The fully-enrolled T-Rex Study of CLBS03 in type 1 diabetes remains on track for top-line data in early 2019. Our programs studying our CD34 cell therapy platform for CMD here in the United States and CLI in Japan continue to enroll with a target for top-line data in the second half of 2019 and early 2020, respectively.

"We are also pleased to announce that we recently completed our Type B meeting with the FDA pertaining to CLBS14-RfA for the treatment of refractory angina. This meeting was conducted to obtain FDA guidance and to define the remaining requirements for registration of this product under the terms of its RMAT designation. We believe that the meeting was both collaborative and positive and our assessment of the conversation is that FDA is demonstrating maximum flexibility afforded under the RMAT designation as we work together to establish the development steps necessary to bring this product to registration. We will be working with FDA to finalize the next development steps and to formalize the minutes of the meeting. We look forward to providing further information once these actions are completed."

Third Quarter Financial Highlights

Research and development expenses for the third quarter of 2018 were $1.7 million, a 47% decrease compared with $3.2 million for the third quarter of 2017. The current quarter expenses were principally comprised of costs in our ischemic repair programs for CLBS12 and CLBS14-CMD, as well as initial planning for our CLBS14-RfA

program. Conversely, the prior year quarter expenses were primarily focused on our T-Rex study for CLBS03, which completed enrollment in December 2017 and is now in the follow-up phase of the study.

General and administrative expenses for the third quarter of 2018 were $2.1 million, a 30% decrease compared with $2.9 million for the third quarter of 2017, due to lower general and administrative headcount and corporate-related activities compared with the prior year period.

The net loss from continuing operations for the third quarter of 2018 was $3.5 million, or $0.36 per share, compared with $3.5 million, or $0.38 per share, for the third quarter of 2017.

Nine Month Financial Highlights

Research and development expenses for the nine months ended September 30, 2018 were $6.1 million, a 46% decrease compared with $11.2 million for the nine months of 2017. The current year expenses were principally comprised of costs in our ischemic repair programs for CLBS12 and CLBS14-CMD as well as initial planning for our CLBS14-RfA program. Conversely, the prior year expenses were primarily focused on our T-Rex study for CLBS03, which completed enrollment in December 2017 and is now in the follow-up phase of the study.

General and administrative expenses for the nine months ended September 30, 2018 were $7.1 million, a 22% decrease compared with $9.1 million for the nine months of 2017. The decrease was due to lower general and administrative headcount and corporate-related activities compared with the prior year period, along with the sale of our counter-flow centrifugation system to Hitachi in the second quarter of 2018, which resulted in a one-time $1.4 million gain included in general and administrative expenses.

The net loss from continuing operations for the nine months ended September 30, 2018 was $12.6 million, or $1.31 per share, compared with $25.8 million, or $1.37 per share, for the nine months of 2017.

Balance Sheet Highlights

As of September 30, 2018, Caladrius had cash, cash equivalents and marketable securities of $46.1 million, compared with $60.1 million as of December 31, 2017. Based on existing programs and projections, the Company continues to remain confident that its cash balances and additional grant funding, along with continued disciplined expense management, will allow it to fund its current business plan beyond 2019.

Conference Call

Caladrius management will host a conference call for the investment community today beginning at 4:30 p.m. Eastern time to review financial results, provide a Company update and answer questions.

Stockholders and other interested parties may participate in the conference call by dialing (866) 595-8403 (domestic), or (706) 758-9979 (international), and providing conference ID: 9490459. The call will also be broadcast live on the Internet via the Company’s website at www.caladrius.com/investors/news-events.

For those unable to participate on the live conference call, a replay will be available through November 15, 2018, and can be accessed by dialing (855) 859-2056 or (404) 537-3406. All listeners should provide the following replay access code: 9490459.

The webcast replay will be archived on the Company’s website for 90 days at www.caladrius.com.