On December 1, 2020 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that management will participate in two upcoming virtual conferences (Press release, BioMarin, DEC 1, 2020, View Source [SID1234572064]). An audio webcast of the presentations will be available live. You can access the webcast at: View Source An archived version of the remarks will also be available through the Company’s website for a limited time following the conference.

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On December 1, 2020 Elevar Therapeutics, Inc. ("Elevar"), a fully integrated biopharmaceutical company built on the promise of elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, and Tanner Pharma Group ("Tanner"), a global provider of integrated specialty access solutions, reported that they have launched a global named patient program to facilitate access to Apealea (paclitaxel micellar) on a named patient basis in areas outside of the United States (U.S.) and Middle East North Africa (MENA) where Apealea is not yet commercially available (Press release, Elevar Therapeutics, DEC 1, 2020, View Source [SID1234572062]). Apealea has been approved by the European regulatory authorities for use in combination with carboplatin for the treatment of adult patients with first relapse of platinum-sensitive epithelial ovarian cancer, primary peritoneal cancer and fallopian tube cancer.

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A named patient program is a mechanism through which physicians can legally and ethically prescribe investigational or approved drugs for patients prior to their commercial availability. Under the terms of the agreement, Tanner is the exclusive supplier of Apealea to healthcare providers outside of the U.S. and MENA on a named patient basis in countries where Apealea is not yet commercially available.

Healthcare professionals can obtain details about the Apealea Access Program by contacting Tanner at:

About Apealea (paclitaxel micellar)

Apealea is a patented, water-soluble, intravenously injectable, non-Cremophor based formulation of paclitaxel. Paclitaxel is a well-known chemotherapy agent used to treat breast, ovarian, lung, bladder, prostate, melanoma, and esophageal cancer, as well as other types of solid tumor cancers. Cremophor EL, is a toxic formulation vehicle used for various poorly-water soluble drugs, including the anticancer agent paclitaxel and is associated with allergic reactions. Apealea received market authorization by the European Commission in November 2018, making it Europe’s first non-Cremophor EL formulation of paclitaxel approved for use in ovarian cancer.

In a pivotal clinical study comparing Apealea to Cremophor-containing Taxol, Apealea preserved activity of paclitaxel with similar overall survival and progression-free survival rates with a comparable safety and tolerability profile despite higher drug load. Less pre-treatment, shorter infusion times, and reduction of Cremophor EL-associated side effects were also observed.

On December 1, 2020 Menarini Ricerche, the R&D division of the Menarini Group, reported today the positive results of the pharmacodynamic assay demonstrating target engagement in the dose escalation part of the DIAMOND-01 trial (CLI24-001; clinicaltrials.gov identifier NCT03008187), a study investigating SEL24/MEN1703, a first-in-class, orally available, dual PIM/FLT3 inhibitor as single agent in Acute Myeloid Leukemia (AML) (Press release, Menarini, DEC 1, 2020, View Source [SID1234572061]).

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The poster entitled "SEL24/MEN1703 provides PIM/FLT3 Downstream Pathway Inhibition in Acute Myeloid Leukemia (AML) Blast Cells: Results of the Pharmacodynamic (PD) Assay in the Dose Escalation Part of First-in-Human DIAMOND Trial" will be presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, which will take place virtually on December 5-8.

"We are pleased with the preliminary, positive results observed with SEL24/MEN1703, a PIM/FLT3 inhibitor under investigation for the treatment of AML. As outlined in our ASH (Free ASH Whitepaper) poster presentation, the dose escalation phase of the DIAMOND-01 trial showed that SEL24/MEN1703 has a manageable safety profile and results in a meaningful target engagement in peripheral blood and bone marrow blast cells from patients treated with SEL24/MEN1703," said Andrea Pellacani, General Manager of Menarini Ricerche. "We look forward to continuing our investigation of SEL24/MEN1703 as a potential new treatment for this aggressive and hard-to-treat cancer, as part of our commitment to develop effective innovative therapies that can make a difference in the lives of cancer patients."

DIAMOND-01 is the First-in-Human, Phase I/II dose escalation and cohort expansion trial of SEL24/MEN1703, in-licensed by Menarini from Ryvu Therapeutics, in AML. The study has completed the dose escalation part showing a manageable safety profile up to the recommended dose of 125 mg/day, with initial evidence of anti-leukemic activity in a single agent setting.

The objective of the pharmacodynamic assessment was to investigate the degree of target engagement achieved at different doses of SEL24/MEN1703, by measuring the phosphorylation of S6 (pS6), a downstream effector of the PIM/FLT3 signaling pathway. In addition, the correlation between pS6 levels and the anti-leukemic effect of SEL24/MEN1703 was assessed in samples collected from patients enrolled in the dose escalation part of the DIAMOND-01 trial. The quantitative assessment of pS6 at a single-cell level was performed both on peripheral blood (PB) and bone marrow (BM) blast cells samples.

The results of this assay confirmed that meaningful target engagement was achieved, both in PB and BM blast cells, in patients treated with SEL24/MEN1703 at 100 mg/day (one dose level below the recommended dose) and at 125 mg/day. Moreover, preliminary data suggest that the PIM/FLT3 pathway inhibition might be associated with blast count reduction, particularly in those patients showing high phosphorylation of S6 at baseline.

We will continue to measure target engagement with this assay in the cohort expansion part of the DIAMOND-01 trial, which is currently recruiting patients with relapsed or refractory AML in both the EU and the US.

About MEN1703

SEL24/MEN1703 is a first-in-class, orally available, dual PIM/FLT3 inhibitor in-licensed by Menarini from Ryvu Therapeutics. It is an investigational compound, not approved for use by regulatory authorities, currently being evaluated in the DIAMOND-01 trial (CLI24-001; clinicaltrials.gov identifier NCT03008187) for the treatment of Acute Myeloid Leukemia.

On December 1, 2020 Dragonfly Therapeutics, Inc. ("Dragonfly"), reported that Merck, known as MSD outside the United States and Canada, has licensed its first TriNKET immunotherapy candidate from Dragonfly (Press release, Dragonfly Therapeutics, DEC 1, 2020, View Source [SID1234572060]). Merck and Dragonfly’s collaboration, initially focused on a number of solid tumor targets, began in October 2018. Earlier this year, the companies expanded their collaboration with a multi-target agreement to develop and commercialize additional natural killer ("NK") cell engager immunotherapies in oncology, infectious disease and immune disorders.

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"Merck is a powerful world leader in drug development across a wide number of therapeutic areas and continues to be a strong scientific collaborator," said Bill Haney, co-founder and CEO of Dragonfly Therapeutics. "We are delighted that Merck has exercised its option for this first immunotherapy candidate from our collaboration, and excited by the progress we are making together on bringing Dragonfly’s TriNKET technology to targets across a broader set of diseases."

Under the agreement Merck has exercised its option to license exclusive worldwide intellectual property rights on its first immunotherapy candidate developed using the TriNKET technology platform and Dragonfly has received an undisclosed payment associated with this milestone.

On December 1, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that it will host a virtual investor and analyst event to discuss the Company’s pipeline of clinical programs and highlights from the data presentations being given at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2020 Annual Meeting (Press release, Karyopharm, DEC 1, 2020, View Source [SID1234572058]). This Karyopharm-sponsored event is scheduled for Tuesday, December 8, 2020 from 1:00 – 2:30 p.m. ET.

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The Karyopharm management team will be joined by a group of recognized multiple myeloma, diffuse large B-cell Lymphoma and leukemia experts to provide additional external context and participate in the Q&A portion of the call.

To access the event, please dial (877) 870-4263 (local) or (412) 317-0790 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.