Author: [email protected]

ZIOPHARM Oncology Announces FDA Acceptance of IND for CD33-Specific CAR-T Cell Therapy Targeting Relapsed/Refractory Acute Myeloid Leukemia

On May 2, 2017 ZIOPHARM Oncology, Inc. (Nasdaq: ZIOP), a biopharmaceutical company focused on new immunotherapies, reported that an investigator-initiated Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a Phase 1 trial infusing the Company’s CD33-specific CAR+ T therapy for relapsed or refractory acute myeloid leukemia (AML) is now active, with the first patient to be enrolled in the study expected to begin treatment in the third quarter of 2017 (Press release, Ziopharm, MAY 2, 2017, View Source [SID1234518785]). The CD33-specific CAR+ T cells incorporate a kill switch designed to eliminate the modified T cells under potential adverse safety conditions.

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!

"Relapsed AML is an aggressive disease with very poor outcomes," said William G. Wierda, M.D., Ph.D., Professor and Center Medical Director, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center and principal investigator for the CD33 study. "In vivo preclinical animal studies have demonstrated that these CAR-T cells targeting CD33 exhibit specific killing of AML cells, eliminating disease burden, and significantly enhancing survival compared to controls, and I look forward to evaluating the safety and effectiveness of this gene therapy for patients with AML."

Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM added, "CAR-T cells expressing CD33 have shown promise in preclinical studies, but to-date, there has been limited experience in humans, representing a significant white space for us in treating AML. We look forward to seeing the positive preclinical results with our CD33-specific CAR-T cells translate to the clinic for relapsed/refractory AML patients who have far too few treatment options. In parallel with this Phase 1 study, we have also begun preclinical studies to evaluate rapid non-viral manufacturing of CAR+ T CD33-specific therapy for treatment of AML under point-of-care."

AML is a rapidly progressing cancer of the blood and bone marrow characterized by uncontrolled proliferation of immature blast cells with multiple associated gene mutations. The American Cancer Society estimates that there were approximately 20,000 new cases of AML and over 10,000 patient deaths from AML in the United States in 2016. A majority of AML patients relapse or present with refractory disease and have overall poor prognosis.
This will be the second CAR target for genetically modified T cells to be studied at The University of Texas MD Anderson Cancer Center under the research and development agreement among ZIOPHARM, Intrexon Corporation (NYSE:XON), and MD Anderson.

Pipeline Review Check

Preclincal investigations are ongoing in combining BI1361849/CV9202 with checkpoint inhibitor or PD1 after chemo-radiation in NSCLC (Company Pipeline, CureVac, MAY 2, 2017, View Source [SID1234518776]).

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!


UCL’S CANCER IMMUNOLOGY GROUP PUBLISHES ON THE RELEVANCE OF CD25 AS A TARGET IN CANCER

On May 2, 2017 Tusk Therapeutics, an immuno-oncology company, reported that its collaborators, Dr. Sergio Quezada and Professor Karl Peggs of the Cancer Immunology group at University College London (UCL), published work in Immunity, a peer-reviewed medical journal, on targeting CD25 with an optimised antibody for the treatment of cancer (Press release, Cancer Research Technology, MAY 2, 2017, View Source [SID1234523498]).

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!

The paper entitled, Fc-Optimized Anti-CD25 Depletes Tumor-Infiltrating Regulatory T Cells and Synergizes with PD-1 Blockade to Eradicate Established Tumor, emphasises the relevance of CD25 as a target and discusses the promise of using an optimised antibody, targeting CD25, for the treatment of cancer in future combination approaches in immuno-oncology.

Tusk Therapeutics, Cancer Research Technology (Cancer Research UK’s commercial arm) and UCL announced in February 2017 that they have an exclusive licensing and collaboration deal aimed at progressing Dr. Quezada’s findings into the clinic.

Luc Dochez, CEO of Tusk Therapeutics, said: "We are excited to be working with Cancer Research UK and Dr. Quezada and Prof. Karl Peggs. The recently published pre-clinical data shows great potential for therapeutic approaches targeting CD25 and Tusk is committed to translating these findings into the clinic."

Dr. Quezada commented: "The recently published results demonstrate the relevance of CD25 as a therapeutic target and I believe that it shows a clear rationale for progressing it into a therapeutic setting."

The paper can be accessed here.

Magenta Therapeutics Advances Stem Cell Transplantation Strategy with $50 Million Series B Financing, Licensing of Clinical-Stage Stem Cell Expansion Program and Strategic Partnership with Be The Match BioTherapies

On May 2, 2017 Magenta Therapeutics, a biotechnology company developing therapies to improve and expand the use of curative stem cell transplantation for more patients, reported rapid progress in advancing the company’s strategic vision, including the completion of a $50 million Series B financing; in-licensing a clinical-stage program from Novartis to support the use of stem cell transplantation in a variety of disease settings; and a strategic partnership with Be The Match BioTherapiesSM, an organization offering solutions for delivering autologous and allogeneic cellular therapies (Press release, Magenta Therapeutics, MAY 2, 2017, View Source [SID1234520733]).

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!

The financing announced today is intended to fuel development of innovative product candidates across multiple aspects of transplantation medicine, including more precise preparation of patients, stem cell harvesting and stem cell expansion. The Series B round, which was oversubscribed, was led by GV (formerly Google Ventures), with participation from all existing investors, including Atlas Venture, Third Rock Ventures, Partners Innovation Fund and Access Industries. The financing also included Casdin Capital and other crossover investors, as well as Be The Match BioTherapies, a subsidiary of National Marrow Donor Program (NMDP)/Be The Match, the world’s leading organization focused on saving lives through bone marrow and umbilical cord blood transplantation.

"Magenta has quickly established itself as a nexus of innovation in stem cell science, catalyzing interest in this area of medicine with the recognition that improvements will have profound impact on patients," said Jason Gardner, D. Phil., chief executive officer, president and cofounder of Magenta Therapeutics. "We aspire to accelerate products that could unleash the potential of transplantation to more patients, including those with autoimmune diseases, genetic blood disorders and cancer. The resounding interest in Magenta from such a high-quality set of investors is a testament to our solid progress since launch, including building a world-class team and a robust pipeline, and generating promising early data."

MGTA-456: Investigational Product Addressing Significant Unmet Need in Stem Cell Transplant
The clinical-stage program in-licensed by Magenta from Novartis, MGTA-456 (formerly HSC835), aims to expand the number of cord blood stem cells used in transplants to achieve superior clinical outcomes compared to standard transplant procedures, and to enable more patients to benefit from a transplant. Under this agreement, Magenta gains rights to use MGTA-456 in selected applications and will develop MGTA-456 in multiple diseases, including immune and blood diseases.

Early results published in Science[1] demonstrated the ability of MGTA-456 to significantly increase the number of umbilical cord blood stem cells. Clinical results reported in Cell Stem Cell[2] demonstrated that this approach yielded an increased expansion of stem cells.

John E. Wagner, M.D., executive medical director of the Bone Marrow Transplantation Program at the University of Minnesota and the study’s lead author, stated: "MGTA-456 markedly shortens time to recovery, addressing one of the most significant challenges in stem cell transplantation today. MGTA-456 achieved a remarkable increase in the number of blood-forming stem cells, greater than that observed by all other methods that have been tested to date. This product has the potential to further improve cord blood transplant outcomes."

Be The Match BioTherapies Strategic Partnership Agreement
Magenta and Be The Match BioTherapies also announced today that in addition to the equity investment, the two organizations have initiated a collaboration to support their shared goals of improving transplant medicine. Magenta and Be The Match BioTherapies will explore opportunities to work together across all of Magenta’s research efforts, from discovery through clinical development. Under this agreement, Magenta may leverage Be The Match BioTherapies’ capabilities, including its cell therapy delivery platform, industry relationships, clinical trial design and management, and patient outcomes data derived from the NMDP/Be The Match, which operates the largest and most diverse marrow registry in the world. NMDP/Be The Match has a network of more than 486 organizations that support marrow transplant worldwide, including 178 transplant centers in the United States and more than 45 international donor centers and cooperative registries.

"We are proud to have made our first equity investment as an organization in Magenta Therapeutics, and we share a vision to improve and advance the use of curative stem cell transplantation for patients with a wide range of diseases," said Amy Ronneberg, president of Be The Match Biotherapies.

Myriad Genetics Reports Fiscal Third-Quarter 2017 Financial Results

On May 2, 2017 Myriad Genetics, Inc. (NASDAQ:MYGN), a global leader in molecular diagnostics and personalized medicine, reported financial results for its fiscal third-quarter 2017, provided an update on recent business highlights and updated its fiscal year 2017 financial guidance (Press release, Myriad Genetics, MAY 2, 2017, View Source [SID1234518811]).

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!

"We were very encouraged to see sequential growth in hereditary cancer testing volumes for the second consecutive quarter," said Mark C. Capone, president and CEO, Myriad Genetics. "Coupled with meaningful sequential volume growth in all of our major pipeline tests including GeneSight, Vectra DA, Prolaris, and EndoPredict, we believe we are rapidly approaching an important inflection in our business where our new products will drive accelerated revenue growth and profitability."

Financial Highlights

The following table summarizes the financial results and product revenue for our fiscal third-quarter 2017:
Revenue
Fiscal Third-Quarter
($ in millions) 2017 2016 %
Change
Molecular diagnostic testing revenue

Hereditary cancer testing revenue $ 140.8 $ 156.3 (10 %)

GeneSight testing revenue 23.9 NA NM

Vectra DA testing revenue 11.2 12.3 (9 %)

Prolaris testing revenue 3.4 5.2* (35 %)

EndoPredict testing revenue 2.3 1.1 109 %

Other testing revenue 3.6 2.5 44 %

Total molecular diagnostic testing revenue 185.2 177.4 4 %

Pharmaceutical and clinical service revenue 11.7 13.1 (11 %)

Total Revenue $ 196.9 $ 190.5 3 %

Income Statement
Fiscal Third-Quarter
($ in millions) 2017 2016 %
Change
Total Revenue $ 196.9 $ 190.5 3 %

Gross Profit 152.6 150.3 2 %
Gross Margin 77.5 % 78.9 %

Operating Expenses 139.7 107.7 30 %

Operating Income 12.9 42.6 (70 %)
Operating Margin 6.6 % 22.4 %

Adjusted Operating Income 24.0 45.8 (48 %)
Adjusted Operating Margin 12.2 % 24.0 %

Net Income 4.2 34.5 (88 %)

Diluted EPS 0.06 0.47 (87 %)

Adjusted EPS $ 0.27 $ 0.41 (34 %)
* Included Medicare retrospective payments
Business Highlights

• myRisk Hereditary Cancer

Hereditary cancer volumes grew on a sequential basis for the second consecutive quarter.
A publication in The Oncologist by researchers at Northwestern University compared 4,250 variants from ClinVar to those from Myriad Genetics. In the study, only 73 percent of the classifications in ClinVar were consistent with Myriad classifications with 27 percent discordant. In addition, it was shown that Myriad could definitely classify up to 60 percent of the variants of uncertain significance from other laboratories.

• GeneSight

Volume grew 44 percent year-over-year to more than 60,000 tests performed in the fiscal third-quarter.
Completed enrollment ahead of schedule in a 1,200 patient clinical utility study evaluating GeneSight in patients with treatment resistant depression. The company anticipates top line data by the end of calendar year 2017.
Published data in Clinical Therapeutics which evaluated 2,168 patients whose treatment was either congruent or non-congruent with the GeneSight test result which demonstrated health savings of $3,998 for primary care physicians and $1,308 for patients treated by psychiatrists after paying for the cost of the test.
Completed a payer demonstration project using the Optum healthcare informatics platform from United Health that demonstrated substantial cost savings associated with the use of GeneSight. Initiated similar demonstration projects with Humana and HealthCore, a subsidiary of Anthem Blue Cross Blue Shield.
Launched a highly successful pilot sales program for GeneSight in the preventive care market with the average sales territory already generating a 300 sample annual run rate.

• Vectra DA

Volumes increased five percent sequentially with approximately 38,500 tests performed.
Creaky Joints, a leading advocacy group for arthritis patients added Vectra DA to its professional guidelines. This builds upon the recent addition of Vectra DA to the United Rheumatology guidelines, a physician guideline body comprising approximately 10 percent of practicing rheumatologists.

• Prolaris

Volumes grew 17 percent year-over-year and nine percent sequentially with approximately 5,100 tests ordered in the third quarter.
The comment period ended on a draft local coverage determination from Palmetto GBA for favorable-intermediate patients, a new indication that would represent a market expansion of approximately 30,000 patients per year in the United States. Prolaris is the only test to receive proposed Medicare coverage in this patient population.
At the upcoming American Urology Association meeting, Myriad will be presenting a 767 patient study that demonstrated the ability of Prolaris to predict metastases from biopsy samples with a high degree of statistical significance.

• EndoPredict

Revenues grew 109 percent year-over-year to $2.3 million in the fiscal third-quarter.
Launched EndoPredict in the United States at the end of the fiscal third-quarter.
In aggregate, Myriad has now received positive coverage decisions from payers in the United States representing 83 million lives.

• myPath Melanoma

Myriad’s third clinical validation study, which demonstrated myPath Melanoma was able to differentiate melanoma from benign nevi with 95 percent diagnostic accuracy, was published in Cancer Epidemiology.
Myriad has submitted its reimbursement dossier for myPath Melanoma to Medicare and private payers.

• Companion Diagnostics

AstraZeneca announced that olaparib met its primary endpoint in BRCA positive, HER2- metastatic breast cancer in the OlympiAD study, demonstrating a statistically significant benefit in progression free survival. This represents a potential 60,000 patient per year market for BRACAnalysis CDx as a companion diagnostic.
Myriad signed a research collaboration with BeiGene which is a global pharmaceutical company developing the PARP inhibitor BGB-290 in the United States.
Signed a commercial collaboration with Clovis Oncology to perform BRACAnalysis CDx testing. Myriad is now performing companion diagnostic testing for every major company developing a PARP inhibitor.
Submitted our regulatory filing in Japan for BRACAnalysis CDx as the companion diagnostic for Lynparza in conjunction with our collaboration with AstraZeneca.

• International

International revenue grew 41 percent year-over-year and comprised five percent of total revenue in the fiscal third-quarter.
International EndoPredict revenue grew 109 percent year-over-year, largely as a result of recent French and German reimbursement.
Fiscal Year 2017 and Fiscal Fourth-Quarter 2017 Financial Guidance
Below is a table summarizing Myriad’s updated fiscal year 2017 and fiscal fourth-quarter 2017 financial guidance:

Revenue GAAP Diluted
Earnings Per
Share Adjusted
Earnings Per
Share
Fiscal Year 2017 $763-$765
million $0.23-$0.25 $1.01-$1.03

Fiscal Fourth-Quarter 2017 $192-$194
million $0.11-$0.13 $0.26-$0.28
These projections are forward-looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release. The Company will provide further details on its business outlook during the conference call today to discuss the fiscal third-quarter financial results, fiscal year 2017, and fiscal fourth-quarter 2017 financial guidance.