On December 9, 2017 Amphivena Therapeutics Inc., a privately held biotechnology company developing AMV564, a CD33/CD3 T cell redirector for the treatment of Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), reported that it will present in an oral presentation at the 59th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) preclinical data that demonstrate that treatment with AMV564 selectively depletes myeloid-derived suppressor cells (MDSCs) in bone marrow cells from patients with MDS with resultant reactivation of T lymphocytes (Press release, Amphivena Therapeutics, DEC 9, 2017, View Source [SID1234522520]). AMV564-induced restoration of immune homeostasis was accompanied by a significant improvement in hematopoiesis. AMV564 is a CD33/CD3 bivalent bispecific antibody that binds both CD33 and CD3 with strong avidity and results in T-cell directed lysis of CD33-expressing myeloid cells.

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"These preclinical data provide a strong rationale for clinical investigation of this innovative approach in patients with MDS, who have limited treatment options today. The data also underscore an opportunity to develop AMV564 for patients with other malignancies where MDSCs have been shown to contribute to the immunosuppressive tumor microenvironment," said Eric J. Feldman, M.D., Amphivena’s Senior Vice President, Clinical Development.

Alan List, M.D., President and CEO of Moffitt Cancer Center, who presented on behalf of the investigators, said, "AMV564 eliminated CD33+ MDSCs in a dose-dependent manner and restored critical aspects of immune homeostasis. In addition, proliferation of CD4+ and CD8+ T cells more than doubled with AMV564 treatment as compared to baseline; IFN-γ production, as measured by gene expression, markedly increased in AMV564-treated cells. AMV564-directed elimination of MDSCs was associated with decreased DNA damage in CD34+ stem cells and improved colony-forming capacity. Finally, the presentation concluded, AMV564 and anti-PD-1 treatment are synergistic for T-cell activation."

Amphivena plans to launch a Phase 1 clinical study in patients with MDS in early 2018. Currently, the company is conducting a Phase 1 clinical study of AMV564 in relapsed or refractory AML and is also exploring the utility of AMV564 in solid tumors.

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 7, 2017 Sandoz, a division of Novartis and the global leader in biosimilars, reported data demonstrating the pharmacokinetics (PK), pharmacodynamics (PD), safety and immunogenicity of proposed biosimilar pegfilgrastim as compared to the reference biologic, Neulasta*[1] (Press release, Novartis, DEC 7, 2017, View Source [SID1234522450]). The Phase I study, conducted in healthy volunteers, confirmed that Sandoz biosimilar pegfilgrastim matches the reference biologic in terms of PK, PD, safety and immunogenicity profiles[1]. The data were presented during the 2017 San Antonio Breast Cancer Symposium.

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"At Sandoz, we are committed to developing high-quality biosimilar and generic medicines that provide the oncology community with treatment options to help manage their patients," said Mark Levick, MD PhD, Global Head of Development, Biopharmaceuticals, Sandoz, "And it starts with following the science. These findings add to the totality of evidence supporting our proposed biosimilar pegfilgrastim."

Sandoz biosimilar pegfilgrastim is currently under review by the European Medicines Agency (EMA) for use in the same indication as the reference biologic. Pegfilgrastim is a long-acting formulation of filgrastim (granulocyte colony-stimulating factor [G-CSF])[2].

About the study
Study participants were randomized to receive a single 6 mg subcutaneous injection of biosimilar pegfilgrastim or reference medicine on Day 0[1]. After dosing, study participants underwent a 4-week assessment period followed by a 8-week washout period before crossing over to receive the other medicine, and were assessed for a further 4-weeks[1].

The results demonstrated that Sandoz proposed biosimilar pegfilgrastim matched the reference medicine in the PK and PD comparisons as primary endpoints, in terms of absolute neutrophil count (maximum effect attributed to study medication) (95% CI: [0.97, 1.02]) and maximum serum concentration of study medication after administration (90% CI: [1.03, 1.19])[1]. Secondary endpoints of safety and immunogenicity were comparable between both groups[1].

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved generic or biosimilar products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Neither can there be any guarantee that, if approved, such generic or biosimilar products will be approved for all indications included in the reference product’s label. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; the particular prescribing preferences of physicians and patients; competition in general, including potential approval of additional generic or biosimilar versions of such products; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; litigation outcomes, including intellectual property disputes or other legal efforts to prevent or limit Sandoz from selling its products; general economic and industry conditions, including the effects of the persistently weak economic and financial environment in many countries; safety, quality or manufacturing issues, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

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.

On December 8, 2017 TG Therapeutics, Inc. (NASDAQ:TGTX), reported the schedule of data presentations for the Company’s lead compounds, TGR-1202, (umbralisib), the Company’s once-daily PI3K delta inhibitor, and TG-1101 (ublituximab), the Company’s novel glycoengineered anti-CD20 monoclonal antibody, at the upcoming 59th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, being held December 9-12, 2017, at the Georgia World Congress Center in Atlanta, Georgia (Press release, TG Therapeutics, DEC 8, 2017, View Source [SID1234522474]).

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Poster presentations at the ASH (Free ASH Whitepaper) 2017 meeting include the following:

Sunday December 10, 2017:

Title: Phase I/II Study of Pembrolizumab in Combination with Ublituximab (TG-1101) and Umbralisib (TGR-1202) in Patients with Relapsed/Refractory CLL
• Abstract Number: 3010
• Session: 642. CLL: Therapy, excluding Transplantation: Poster II
• Date and Time: Sunday, December 10, 2017; 6:00 PM – 8:00 PM ET
• Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
• Presenter: Anthony R. Mato, MD, University of Pennsylvania, Philadelphia, PA

Title: Umbralisib/TGR-1202 as a Novel Dual PI3K/CK1 Inhibitor Has a Unique Therapeutic Role in Silencing Oncogenes in Aggressive Lymphomas
• Abstract Number: 2809
• Session: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Agents: Poster II
• Date and Time: Sunday, December 10, 2017; 6:00 PM – 8:00 PM ET
• Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
• Presenter: Ipsita Pal, PhD, Columbia University Medical Center, New York, NY

Title: Differential Regulation of T Cells By PI3K Delta Inhibitors in a CLL Murine Model
• Abstract Number: 3009
• Session: 642. CLL: Therapy, excluding Transplantation: Poster II
• Date and Time: Sunday, December 10, 2017; 6:00 PM – 8:00 PM ET
• Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
• Presenter: Kamira K. Maharaj, BS, Moffit Cancer Center, Tampa, FL
Monday, December 11, 2017:

Title: An Integrated Safety Analysis of the Next Generation PI3Kδ Inhibitor Umbralisib (TGR-1202) in Patients with Relapsed/Refractory Lymphoid Malignancies
• Abstract Number: 4037
• Session: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies: Poster III
• Date and Time: Monday, December 11, 2017; 6:00 PM – 8:00 PM ET
• Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
• Presenter: Matthew S. Davids, MD, Dana Farber Cancer Institute, Boston, MA

Title: KI Intolerance Study: A Phase 2 Study to Assess the Safety and Efficacy of Umbralisib (TGR-1202) in Patients with Chronic Lymphocytic Leukemia (CLL) Who Are Intolerant to Prior BTK or PI3K-δ Inhibitor Therapy
• Abstract Number: 4314
• Session: 642. CLL: Therapy, excluding Transplantation: Poster III
• Date and Time: Monday, December 11, 2017; 6:00 PM – 8:00 PM ET
• Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
• Presenter: Anthony R. Mato, MD, University of Pennsylvania, Philadelphia, PA

Title: PI3K-Delta Inhibitors Induce Primary Monocyte Cytotoxicity but Do Not Alter Monocyte Differentiation
• Abstract Number: 4284
• Session: 641. CLL: Biology and Pathophysiology, excluding Therapy: Poster III
• Date and Time: Monday, December 11, 2017; 6:00 PM – 8:00 PM ET
• Location: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2
• Presenter: Daphne Friedman, MD, Durham VA/Duke University Medical Center, Durham, NC
The above referenced abstracts can be viewed online through the ASH (Free ASH Whitepaper) meeting website at www.hematology.org. Following each presentation, the data presented will be available on the Publications page of the Company’s website at www.tgtherapeutics.com.

TG THERAPEUTICS INVESTOR & ANALYST EVENT

TG Therapeutics will also host a reception on Sunday, December 10, 2017 beginning at 8:00pm ET with featured presentations beginning promptly at 8:15pm ET. The event will take place at the Ritz Carlton Atlanta (Downtown) in the Salon I/II Room on the lower level. This event will be webcast live and will be available on the Events page, located within the Investors & Media section of the Company’s website at www.tgtherapeutics.com, as well as archived for future review. This event will also be broadcast via conference call. To access the conference line, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), and reference Conference Title: TG Therapeutics December 2017 Investor & Analyst Event.