On December 8, 2017 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported that the one-year efficacy and safety results from its FIT Phase 3 clinical program of fostamatinib for chronic or persistent immune thrombocytopenia (ITP) will be featured in an oral presentation at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held in Atlanta, GA from December 9 to December 12, 2017 (Press release, Rigel, DEC 8, 2017, View Source;p=RssLanding&cat=news&id=2321827 [SID1234522471]).

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During the presentation, James B. Bussel, M.D., professor emeritus of pediatrics at Weill Cornell Medicine and the principal study investigator on the FIT Phase 3 program who has served as a member of an advisory/scientific board for Rigel Pharmaceuticals, will share an overview of the FIT clinical program.

Oral Presentation Details:
TITLE: Long-Term Maintenance of Platelet Responses in Adult Patients with Persistent/Chronic Immune Thrombocytopenia Treated with Fostamatinib: 1-Year Efficacy and Safety Results
Session Name: 311. Disorders of Platelet Number or Function: ITP: clinical aspects
Session Date: Saturday, December 9, 2017
Session Time: 7:30 AM – 9:00 AM EST
Presentation Time: 8:15 AM EST
Location: Georgia World Congress Center, Bldg B, Lvl 3, B304-B305 (Atlanta, GA)

About Fostamatinib in ITP
Fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, is an investigational drug for adult patients with chronic or persistent immune thrombocytopenia (ITP). The New Drug Application (NDA) for fostamatinib for adult patients with chronic or persistent ITP, which was previously granted Orphan Drug designation, is currently under review by the U.S. Food and Drug Administration with a Prescription Drug User Fee Act (PDUFA) goal date of April 17, 2018. The conditionally approved proprietary name for fostamatinib (as confirmed by the FDA) is TAVALISSETM.

The NDA is supported by data from the FIT Phase 3 clinical program, which was comprised of three studies, two randomized placebo-controlled studies (Studies 047 and 048) and an open-label extension study (Study 049). Together with an initial proof of concept study, the NDA included 163 ITP patients. Across all indications, fostamatinib has been evaluated in over 4,600 subjects. Data from all studies, including preclinical evaluation and drug manufacturing data, were included in the NDA submission.

About ITP
In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with increased risk of severe bleeding events that can result in serious medical complication, or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPOs) and splenectomy. However, not all patients are adequately treated with existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

On December 8, 2017 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, reported that SL-401, a novel targeted therapeutic directed to CD123, will be featured in four presentations at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, to be held December 9-12, 2017 in Atlanta, GA (Press release, Stemline Therapeutics, DEC 8, 2017, View Source [SID1234522473]).

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Details on the presentations are listed below. Presentations will be available on the Stemline website, Scientific Presentations tab, after their delivery, as well as at our Stemline corporate booth (#3122) at ASH (Free ASH Whitepaper) 2017.

SL-401: BPDCN (Clinical)

Title: Results of Pivotal Phase 2 Trial of SL-401 in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Presenter: Naveen Pemmaraju, MD; MD Anderson Cancer Center
Abstract: 1298
Session: 613. Acute Myeloid Leukemia: Clinical Studies: Poster I
Date/Time: Saturday, December 9, 2017 5:30 PM–7:30 PM
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

SL-401: AML in CR with MRD (Clinical)

Title: Results from Ongoing Phase 2 Trial of SL-401 As Consolidation Therapy in Patients with Acute Myeloid Leukemia (AML) in Remission with High Relapse Risk Including Minimal Residual Disease (MRD)
Presenter: Andrew Lane, MD, PhD; Dana-Farber Cancer Institute
Abstract: 2583
Session: 613. Acute Myeloid Leukemia: Clinical Studies: Poster II
Date/Time: Sunday, December 10, 2017 6:00 PM–8:00 PM
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

SL-401: Myeloproliferative neoplasms (Clinical)

Title: Results from Ongoing Phase 2 Trial of SL-401 in Patients with Myeloproliferative Neoplasms Including Chronic Myelomonocytic Leukemia and Primary Myelofibrosis
Presenter: Mrinal Patnaik, MBBS; Mayo Clinic
Abstract: 2908
Session: 634. Myeloproliferative Syndromes: Clinical: Poster II
Date/Time: Sunday, December 10, 2017 6:00 PM–8:00 PM
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

SL-401 + Azacitidine: High risk MDS and elderly AML (Preclinical) – Oral Presentation

Title: Resistance to SL-401 in AML and BPDCN Is Associated with Loss of the Diphthamide Synthesis Pathway Enzyme DPH1 and Is Reversible By Azacitidine
Presenter: Andrew A. Lane, Dana-Farber Cancer Institute
Abstract: 797
Session: 604. Molecular Pharmacology and Drug Resistance in Myeloid Diseases: Novel Therapeutics and Mechanisms of Resistance in Myeloid Disease
Date/Time: Monday, December 11, 2017 5:30 PM
Location: Georgia World Congress Center, Building B, Level 2, B207-B208

About BPDCN
Please visit the BPDCN awareness booth (#3143) at ASH (Free ASH Whitepaper) 2017 and www.bpdcninfo.com.

Enzo Biochem has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Enzo Biochem, 2017, DEC 7, 2017, View Source [SID1234522436]).

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On December 7, 2017 Radius Health, Inc. (Nasdaq:RDUS) reported an update on data from the Phase 1 005 clinical study of elacestrant (RAD1901), an oral selective estrogen receptor degrader (SERD), in patients with estrogen receptor positive (ER+) breast cancer (Press release, Radius, DEC 7, 2017, View Source [SID1234522447]). The data were presented at a Spotlight Presentation (Abstract 1410) during the 2017 San Antonio Breast Cancer Symposium (SABCS). Elacestrant recently received Fast Track designation from the U.S. Food and Drug Administration.

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There are 40 patients that have been treated at the 400 mg dose in the elacestrant Phase I dose escalation and expansion cohorts. All study participants are heavily pretreated ER+, HER2-negative, advanced breast cancer patients that have received a median of three prior lines of systemic therapy and have evaluable advanced or metastatic disease. Of the enrolled patients, 22 patients met the RECIST measurable disease criteria at baseline and there were 6 confirmed partial responses in this group. Elacestrant was well-tolerated with the most common adverse events being low grade nausea, dyspepsia and vomiting.

"It is quite encouraging to see the clinical activity with elacestrant in the heavily pretreated advanced patient population, and further therapeutic development is warranted for patients with hormone receptor positive breast cancer", commented Dr. Aditya Bardia, Director of Precision Medicine and attending physician at Center for Breast Cancer, Massachusetts General Hospital Cancer Center, Harvard Medical School, Boston, MA.

Radius plans to initiate a Phase 2 clinical study of elacestrant monotherapy, a potentially pivotal study, for women with advanced or metastatic ER+/HER2- breast cancer early in 2018.

"Patients cycle through and generally do not repeat treatment regimens, limiting treatment options as their disease advances. We are pleased about the potential to offer patients who have progressed or relapsed during their current standard of care with a new treatment option," said Gary Hattersley, PhD, Chief Scientific Officer. "Radius is committed to developing and to providing breast cancer patients with the next generation of hormonal treatment options, as a single agent and in combination, across all lines of therapy."

An update on data from the elacestrant FES PET 106 Phase I clinical study in the EU was presented yesterday and demonstrated that elacestrant reduces 18F-FES uptake in patients with advanced ER+ breast cancer who progressed on prior endocrine therapy. The reduction in FES uptake supports elacestrant dose selection for further clinical development and was similar in patients harboring mutant or wildtype ESR-1. Three preclinical poster presentations further highlighting and demonstrating elacestrant activity, as a single agent and in combination with other targeted therapies, will be presented tomorrow morning at SABCS.

Following a strategic review, Radius has decided to discontinue further evaluation of elacestrant for vasomotor symptoms and will focus instead on the continued clinical development of the compound as a potential treatment option in breast cancer.

WEBCAST/CONFERENCE CALL

In conjunction with today’s elacestrant Spotlight presentation at SABCS, Radius will host a conference call and live webcast at 8:00 p.m. CT today to discuss the results of the Phase I program as of the cutoff date of October 30, 2017 and provide a company update.

The live webcast titled "Oncology Program Update from San Antonio Breast Cancer Symposium — 2017" will be available at View Source or by visiting the Investors section of Radius’ website at View Source

Conference call information:

Domestic Dial-in Number: (866) 323-7965
International Dial-in Number: (346) 406-0961
Conference ID: 9089206

A replay of the webcast will be available on the company’s website, www.radiuspharm.com in the Investor section under Events and Presentations for 7 days following the live webcast.

About Elacestrant (RAD1901)
Elacestrant is a selective estrogen receptor degrader (SERD), which is being evaluated for potential use as a once daily oral treatment for hormone-receptor positive breast cancer. Elacestrant is currently being investigated for potential use in women with advanced estrogen receptor positive, HER2 negative, breast cancer, the most common form of the disease. Studies completed to date indicate that the compound has the potential for use as a single agent or in combination with other therapies for the treatment of breast cancer.

Additional information on the clinical trial program of elacestrant (RAD1901) is available on www.clinicaltrials.gov.

On December 7, 2017 ERYTECH Pharma (Euronext Paris: ERYP) (Nasdaq: ERYP) ("ERYTECH"), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported topline results from its Phase 2b clinical study evaluating eryaspase (GRASPA) for the treatment of acute myeloid leukemia (AML) (Press release, ERYtech Pharma, DEC 7, 2017, View Source [SID1234522449]).

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The open-label, randomized, multi-center clinical study, evaluated eryaspase in newly diagnosed AML patients over the age of 65 and unfit for intensive chemotherapy. The study enrolled a total of 123 patients at 30 European sites. The median age of the patients was 78 years. Patients were randomized two-to-one to receive eryaspase in combination with low-dose cytarabine (LDAC) versus LDAC alone. The primary endpoint in this proof-of concept study was overall survival (OS). The key secondary endpoints included progression free survival, overall response and toxicity. The study was performed in collaboration with Orphan Europe (Recordati Group), ERYTECH’s partner for the anticipated commercialization of GRASPA for the treatment of ALL and AML in Europe.

The study did not meet its primary endpoint of overall survival (OS). The OS Hazard Ratio (HR) was 1.06 (95% CI; 0.70, 1.61). When adjusting for minor imbalances in the main prognostic factors at baseline (age, karyotype and FAB status), the OS HR was 0.98 (95% CI; 0.64, 1.50). The median number of months on treatment was less then 2 months in both treatment arms. The toxicity profile was acceptable and consistent with previously reported data for eryaspase.

"These data reflect the complexity of this disease, particularly in the older age group." commented Iman El-Hariry, MD, PhD, Chief Medical Officer of ERYTECH. "While we are disappointed with the outcome, we are reassured with the safety profile of eryaspase in these very frail and elderly patients."

Gil Beyen, Chairman and CEO of ERYTECH, added, "Although clearly disappointing, these results do not change our commitment to the development of the eryaspase product candidate. Eryaspase has shown positive safety and efficacy results in the treatment of pancreatic cancer and acute lymphoblastic leukemia and we remain committed to bringing this treatment option to patients in these and potential other indications."

ERYTECH will hold a conference call and webcast on Monday, December 11th at 10:00 am EST to discuss the results of this study. The full dataset will be discussed at a scientific congress in 2018.

Investors and analysts wishing to participate can access the call via the following teleconferencing numbers:

USA: +1 833 8186807 United-Kingdom: +080 00323836
Switzerland: +080 0561782 Germany: +080 01815287
France: +080 5081485 Belgium: +080 073308
Sweden: +020 798505 Finland : +080 0412874
Netherlands: +080 00200089

Password: 6983889

The webcast can be followed live online via the following link:

Webcast Link: View Source

An archive of the webcast will be available for 90 days on the "Webcast" section of the Company’s investor relations site at www.erytech.com.

Additionally, a replay of the call will be available for 7 days. To listen to the replay, please dial:

USA: +1 404 537 3406
Participant Password: 6983889

About acute myeloid leukemia (AML)

AML is a form of acute leukemia or blood cancer that results from the improper maturation of myeloid stem cells leading to the production of myeloblasts. The increasing numbers of abnormal blasts crowd out healthy cells in bone marrow (resulting in infection, anemia, and bleeding) and can spread to other parts of body. With about 40,000 new patients per year in Europe and the United States, AML is the most common type of acute leukemia. Affecting mainly the adult and senior patient population, the median age of patients diagnosed with AML is approximately 67 years, and AML represents one of the highest mortality rates among all type of cancers and an important unmet medical need.