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Stemline Announces Seven Presentations, Including Oral Presentation of Updated SL-401 Phase 2 BPDCN Data, at the 2016 ASH Meeting this Weekend

On December 2, 2016 Stemline Therapeutics, Inc. (Nasdaq:STML) reported that SL-401, a novel targeted therapeutic directed to CD123, will be featured in seven presentations, including three oral presentations, at the 2016 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being held December 3-6, 2016 at the San Diego Convention Center in San Diego, CA (Press release, Stemline Therapeutics, DEC 2, 2016, View Source [SID1234516890]).

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Investigators will deliver an oral presentation on updated clinical data from the SL-401 Phase 2 trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN). Additional presentations include early clinical data from ongoing SL-401 clinical trials in patients with acute myeloid leukemia (AML) in remission with high relapse risk (selected for oral presentation), high-risk myeloproliferative neoplasms (MPN), and relapsed/refractory multiple myeloma. Preclinical data to be presented include an oral presentation of SL-401 activity against cancer stem cells (CSCs) of AML and myelodysplastic syndrome (MDS), as well as SL-401 activity against CSCs of myeloma and SL-401 efficacy in combination with SL-801, a novel XPO1 inhibitor, against myeloma and other malignancies.

Details on the presentations are listed below. Additionally, presentations will be available on the Stemline website, Scientific Presentations tab, after their delivery.

Details on the presentations are as follows:

SL-401 – BPDCN (Clinical) – Oral Presentation
Title: Results from Phase 2 Trial Ongoing Expansion Stage of SL-401 in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Presenter: Naveen Pemmaraju, MD; MD Anderson Cancer Center
Abstract: 342
Session: 613. Acute Myeloid Leukemia: Clinical Studies: Optimizing Current AML Therapy
Date/Time: Sunday, December 4, 2016 10:45 AM PT
Location: Marriott Marquis San Diego Marina, Pacific Ballroom

SL-401 – AML in CR with MRD (Clinical) – Oral Presentation
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: 215
Session: 613. Acute Myeloid Leukemia: Clinical Studies: Innovations in Induction Therapy
Date/Time: Saturday, December 3, 2016 5:00 PM PT
Location: Marriott Marquis San Diego Marina, San Diego Ballroom AB

SL-401 – Myeloproliferative neoplasms (Clinical)
Title: Results from Ongoing Phase 2 Trial of SL-401 in Patients with Advanced, High-Risk Myeloproliferative Neoplasms Including Chronic Myelomonocytic Leukemia
Presenter: Mrinal Patnaik, MBBS; Mayo Clinic
Abstract: 4245
Session: 634. Myeloproliferative Syndromes: Clinical: Poster III
Date/Time: Monday, December 5, 2016 6:00 PM – 8:00 PM PT
Location: San Diego Convention Center, Hall GH

SL-401 — Multiple myeloma (Clinical)
Title: Results from Ongoing Phase 1/2 Trial of SL-401 in Combination with Pomalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma
Presenter: Myo Htut, MD; City of Hope
Abstract: 5696
Date/Time: Thursday, December 1, 2016 publication release
Location: Published online on ASH (Free ASH Whitepaper) abstract website

SL-401 – AML and MDS cancer stem cells – Oral Presentation
Title: SL-401 Mediates Potent Cytotoxicity Against CD123+ AML and MDS with Excess Blasts and Demonstrates Therapeutic Benefit in PDX Model
Presenter: Rajeswaran Mani, PhD; Ohio State University
Abstract: 580
Session: 604. Molecular Pharmacology and Drug Resistance in Myeloid Diseases: Targeting Leukemia-Initiating Cells
Date/Time: Monday, December 5, 2016 ?7:45 AM PT
Location: San Diego Convention Center, Room 24

SL-401 in combination with SL-801 — Multiple myeloma and other malignancies
Title: SL-401, a Targeted Therapy Directed to the Interleukin-3 Receptor (CD123), and SL-801, a Reversible Inhibitor of Exportin-1 (XPO1), Display Synergistic Anti-Tumor Activity Against Hematologic Malignancies in Vitro
Presenter: Janice Chen, PhD; Stemline
Abstract: 4724
Session: 802. Chemical Biology and Experimental Therapeutics: Poster III
Date/Time: Monday, December 5, 2016 6:00 PM – 8:00 PM PT
Location: San Diego Convention Center, Hall GH

SL-401 – Multiple myeloma
Title: SL-401, a Novel IL-3Rα/CD123—Directed Agent Targets Stem-like Cells in Multiple Myeloma
Presenter: Arghya Ray, PhD; Dana-Farber Cancer Institute
Abstract: 4463
Session: 652. Myeloma: Pathophysiology and Pre-Clinical Studies, excluding Therapy: Poster III
Date/Time: Monday, December 5, 2016 6:00 PM – 8:00 PM PT
Location: San Diego Convention Center, Hall GH

Late-Breaking Oral Plenary Presentation of a Novel Entrectinib Combination Regimen at the 2016 EORTC-NCI-AACR Annual Meeting

On December 2, 2016 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported data examining the combination of entrectinib and trametinib in overcoming resistance to TRK inhibition during a late-breaking oral plenary presentation (during the Exceptional Response and Expected Resistance Session) at the 2016 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) (ENA) Molecular Targets and Cancer Therapeutics Symposium in Munich, Germany (Press release, Ignyta, DEC 2, 2016, View Source [SID1234516880]). Entrectinib is the company’s orally available, CNS-penetrant tyrosine kinase inhibitor, targeting tumors that harbor TRK, ROS1 or ALK fusions, and is currently in a registration-enabling Phase 2 clinical trial known as STARTRK-2.

"This presentation is a great example of the successful translation of preclinical observations into clinical practice for the benefit of patients," said Alexander Drilon, M.D., of Memorial Sloan Kettering Cancer Center, who presented the data on behalf of the abstract authors at ENA. "The patient experience described in today’s presentation highlights the ability of entrectinib, in combination with a MEK inhibitor, to overcome potential treatment resistance that has been described for TRK inhibitors."

Data presented describe results from a single patient protocol designed to allow co-administration of entrectinib and trametinib, a commercially-approved MEK inhibitor. The patient, diagnosed with mammary analog secretory carcinoma (or MASC) with an NTRK3 fusion—~90-100% of MASC cases have TRK fusions—and previously treated with multiple surgeries, radiation, vinorelbine, carboplatin/paclitaxel, doxorubicin and crizotinib, experienced a rapid and confirmed partial response (89% reduction) with single-agent entrectinib treatment and remained on therapy for nine months. However, during the course of therapy, the patient’s tumor developed a solvent front point mutation, the predicted mechanism of resistance to first generation TRK inhibitors and analogous to a common mechanism of resistance for other TKIs against other fusion targets.

Based on both in vitro and in vivo data developed by Ignyta, indicating that entrectinib plus a MEK inhibitor could overcome such TRK inhibitor resistance, a single patient protocol was created, reviewed by the FDA and implemented to allow for dose escalation of entrectinib in combination with trametinib. While on the combination, all drug-related adverse events (AEs) were grade 1 or 2, and no new AEs specific to the combination were encountered. The patient achieved a 22% reduction in tumor volume and remained on the combination regimen for nearly seven months.

"This single-patient protocol is an excellent example of our vision at Ignyta, leveraging the molecular alterations responsible for cancer’s growth to design rational treatment strategies for patients in their fight against cancer," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "In addition to STARTRK-2, our global Phase 2 basket trial of entrectinib in multiple tumor histologies and STARTRK-NG, our Phase 1/1b trial of entrectinib in pediatric patients with solid tumors, we look forward to further exploration of entrectinib in combination with a MEK inhibitor in a Phase 1/1b clinical trial anticipated to initiate in the second half of 2017."

Calithera Biosciences Announces CB-839 Preclinical Data Selected for Presentation at the 58th American Society of Hematology Annual Meeting

On December 2, 2016 Calithera Biosciences, Inc. (Nasdaq:CALA), a clinical stage biotechnology company focused on the discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer, reported that preclinical data for its lead drug candidate CB-839, the company’s novel, orally bioavailable glutaminase inhibitor, will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which is being held from December 3-6, 2016 in San Diego, California (Press release, Calithera Biosciences, DEC 2, 2016, View Source;p=RssLanding&cat=news&id=2227168 [SID1234516878]). Data to be presented include the following two poster presentations:

Glutaminase Inhibitor CB-839 Enhances Proteasome Inhibitor Sensitivity in Multiple Myeloma Cells
Presenter: Ravyn Thompson, Nathan Dolloff, Medical University of South Carolina
Oral and Poster Abstracts Session 652: Myeloma: Pathophysiology and Preclinical Studies, Hall GH, Abstract 3294
Sunday, December 4, 2016, 6:00 p.m.-8:00 p.m. PT
Gls Inhibitor CB-839 Modulates Cellular Metabolism in AML and Potently Suppresses AML Cell Growth When Combined with 5-Azacitidine
Presenter: Tianyu Cai, Marina Konopleva, The University of Texas, MD Anderson Cancer Center
Oral and Poster Abstracts Session 616: Acute Myeloid Leukemia: Novel Therapy, Hall GH, Abstract 4064
Monday, December 5, 2016, 6:00 p.m.-8:00 p.m. PT

PIQUR Receives Orphan Drug Designation from FDA for PQR309 in PCNSL

On December 2, 2016 PIQUR Therapeutics AG, a Swiss clinical-stage pharmaceutical company, reported that
the U.S. Food & Drug Administration (FDA) has granted orphan drug designation to PIQUR’s lead compound PQR309 for the treatment of primary central nervous system lymphoma (PCNSL) (Press release, PIQUR Therapeutics, DEC 2, 2016, View Source [SID1234527272]).

PCNSL is a very rare and aggressive form of lymphoma involving brain and its linings, eyes or spinal cord. The disease affects less than 1 person in 100,000 in the USA with approximately 7,500 new cases reported annually in the USA, Europe and Japan. Treatment options available for PCNSL are limited, and the prognosis with current therapies is poor.

"We are very pleased to receive FDA orphan drug designation for PQR309 in PCNSL. This is an important regulatory milestone for the company and a significant step towards the clinical advancement of PQR309," said Dr. Ruggero Della Bitta, Chief Medical Officer of PIQUR. "This represents an important step towards addressing a high unmet medical need and bringing a potential treatment to those with this rare and life-threatening disease."

The Orphan Drug Designation Program, administered by the FDA’s Office of Orphan Products Development, is intended to encourage companies to develop therapeutics for diseases that affect fewer than 200,000 people in the USA. The designation provides the company several benefits and incentives, including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, as well as a seven-year period of market exclusivity upon regulatory product approval.

About PQR309
PIQUR’s lead compound, PQR309, is an oral, brain-penetrant, dual inhibitor of the PI3K/mTOR pathway, which is activated in 60 – 80% of human cancers. Unlike most of its competitors, PQR309 crosses the blood-brain barrier, expanding its use to malignant diseases involving the brain. Preclinical and Phase 1 studies have shown PQR309 to have a favorable safety, tolerability and pharmacokinetic profile. In addition, PQR309 has shown both preclinical
activity in various tumor models and clinical activity in Phase 1 and 2 studies.

PQR309 is currently being investigated in five Phase 1 and 2 clinical studies in advanced solid tumors (NCT02483858), relapsed or refractory lymphoma (NCT02249429), relapsed or refractory PCNSL (NCT02669511) and progressive glioblastoma multiforme (NCT02850744). In addition, the PIQHASSO Phase 1/2b study investigates PQR309 in combination with Eisai’s Eribulin in metastatic HER2-negative and triple-negative breast cancer (NCT02723877).

Amgen And Allergan Submit Biosimilar Marketing Authorization Application To European Medicines Agency For ABP 215, A Biosimilar Candidate To Bevacizumab

On December 2, 2016 Amgen (NASDAQ:AMGN) and Allergan plc. (NYSE:AGN) reported the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for ABP 215, a biosimilar candidate to Avastin (bevacizumab) (Press release, Amgen, DEC 2, 2016, View Source [SID1234516874]). The companies believe this submission is the first bevacizumab biosimilar application submitted to the EMA.

"The submission of ABP 215 to the EMA is an important milestone as Amgen seeks to expand our oncology portfolio," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "Amgen is committed to delivering medicines to patients worldwide and ABP 215 has the potential to offer an additional high-quality therapeutic option for patients diagnosed with cancer."

"The EMA submission of ABP 215 marks an important step forward in providing a potential therapeutic option for patients diagnosed with cancer. Allergan is committed to globally developing high-quality therapies in critical disease areas," said David Nicholson, Chief Research and Development Officer at Allergan.

ABP 215 is a biosimilar candidate to bevacizumab, a recombinant immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that binds to vascular endothelial growth factor (VEGF) and inhibits the interaction of VEGF with its receptors, VEGF receptor-1 and VEGF receptor-2, thus inhibiting establishment of new blood vessels necessary for the maintenance and growth of solid tumors.

The MAA submission includes analytical, pharmacokinetic and clinical data, as well as pharmacology and toxicology data. The Phase 3 comparative efficacy, safety and immunogenicity study was conducted in adult patients with non-squamous non-small cell lung cancer (NSCLC). The Phase 3 study confirmed no clinically meaningful difference to bevacizumab in terms of efficacy, safety and immunogenicity.

About ABP 215
ABP 215 is being developed as a biosimilar to bevacizumab, which is approved in the U.S., EU and other regions for the treatment of patients with unresectable, locally advanced, recurrent or metastatic non-squamous NSCLC as well as metastatic carcinoma of the colon or rectum; metastatic renal cell carcinoma; and other region-specific indications.