Stemline Therapeutics to Present at the Cowen and Company 39th Annual Health Care Conference

On March 6, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that Ivan Bergstein, M.D., Stemline’s CEO, will present at the Cowen and Company 39th Annual Health Care Conference on Monday, March 11, 2019 at 4:50 PM ET at the Marriott Copley Place in Boston, MA (Press release, Stemline Therapeutics, MAR 6, 2019, https://ir.stemline.com/news-releases/news-release-details/stemline-therapeutics-present-cowen-and-company-39th-annual [SID1234534013]). A live webcast of the presentation can be viewed on the company’s website at www.stemline.com.

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About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, and other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About ELZONRIS
ELZONRIS (tagraxofusp), a CD123-directed cytotoxin, was approved by the Food and Drug Administration (FDA) on December 21, 2018 for the treatment of adult and pediatric patients, two years and older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In November 2018, the European Medicines Agency (EMA) granted ELZONRIS accelerated assessment to the marketing authorization application (MAA), which was submitted to, and validated by, the EMA in January 2019. ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

Ophthotech to Present at the Cowen and Company 39th Annual Health Care Conference

On March 6, 2019 Ophthotech Corporation (Nasdaq: OPHT) reported that Kourous A. Rezaei, M.D., Chief Medical Officer of Ophthotech, will present an overview of the Company at the Cowen and Company 39th Annual Health Care Conference in Boston, MA, on Wednesday, March 13, 2019 at 9:20 a.m. Eastern Time (Press release, Ophthotech, MAR 6, 2019, https://investors.ophthotech.com/news-releases/news-release-details/ophthotech-present-cowen-and-company-39th-annual-health-care [SID1234534011]).

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Investors and the general public are invited to listen to a live webcast of the presentation on the Events & Presentations section on the Ophthotech website at www.ophthotech.com. An archived replay of the webcast will be available on the website for 14 days after the conference.

Protalix BioTherapeutics to Hold Full-Year 2018 Financial Results and Corporate Update Conference Call on March 14, 2019

On March 6, 2019 Protalix BioTherapeutics, Inc. (NYSE American:PLX, TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx, reported that it will report full-year 2018 financial results and provide a corporate update on Thursday, March 14, 2019 at 8:30 am ET (Press release, Protalix, MAR 6, 2019, View Source;p=RssLanding&cat=news&id=2390302 [SID1234534008]).

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To participate in the conference call, please dial the following numbers prior to the start of the call: United States: +1 (844) 358-6760; International: +1 (478) 219-0004. Conference ID number 9583103.

The conference call will also be broadcast live and available for replay for two weeks on the Company’s website, www.protalix.com, in the Events Calendar of the Investors section. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.

GlycoMimetics Reports Fourth Quarter and Year-End 2018 Results

On March 6, 2019 GlycoMimetics, Inc. (Nasdaq:GLYC) reported its financial results for the year and fourth quarter ended December 31, 2018, highlighted recent company achievements and commented on milestone achievements anticipated in 2019 (Press release, GlycoMimetics, MAR 6, 2019, View Source [SID1234534007]). Cash and cash equivalents at December 31, 2018 were $209.9 million.

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"In 2018, GlycoMimetics delivered achievements on many fronts: in clinical development, preclinical research and discovery and in financial resource management. We advanced our comprehensive clinical program for uproleselan that, if successful, could position uproleselan as a foundational therapy across the spectrum of AML. We believe this accomplishment reflects the enthusiasm of clinicians who have seen the Phase 1/2 uproleselan data at top oncology congresses and uproleselan’s impact on patient outcomes in clinical trials. In terms of our discovery and preclinical research during 2018, our team, utilizing our specialized chemistry expertise, produced new drug candidates to expand our pipeline opportunities into indications that go beyond sickle cell disease and hematologic cancers. Looking forward to 2019, we are in a strong financial position to pursue the clinical and preclinical opportunities ahead. Importantly, we look to the rivipansel top-line readout expected late in the second quarter that could represent both the first potential commercial success from our pipeline and a key financial resource going forward," noted Rachel King, Chief Executive Officer.

2018 and Recent Highlights

The GlycoMimetics-sponsored pivotal Phase 3 trial of uproleselan in relapsed/refractory AML enrolled its first patient; multiple investigative sites have now been initiated; work continues to expand to clinical sites across the US, Europe, Canada and Australia
The National Cancer Institute (NCI) collaborative study of uproleselan in newly diagnosed patients fit for chemotherapy has opened and is recruiting patients at multiple sites
Planning continues for the collaborative Haemato Oncology Foundation for Adults in the Netherlands (HOVON) European study of uproleselan in newly diagnosed patients unfit for chemotherapy with a goal of trial initiation in 2019
At the ASH (Free ASH Whitepaper) Annual Meeting in December 2018, key new data on clinical outcomes from the Phase 1/2 relapsed/refractory AML trial of uproleselan underscored opportunities to position this drug candidate, if approved, as a potential foundational therapy across the spectrum of AML
Our Japanese patent for uproleselan was granted in August 2018, complementing patents already issued in the United States and Europe
Preclinical data for several existing and new pipeline programs, including GMI-1687 and GMI-1757, were presented at key scientific meetings, including AACR (Free AACR Whitepaper) in March 2018 and ASH (Free ASH Whitepaper) in December 2018
GlycoMimetics’ collaborator Pfizer advised that top-line results of the rivipansel Phase 3 clinical trial would be announced by late second quarter 2019
Scott Jackson, veteran biopharma executive, joined the Board of Directors
Chairman of the Board, M. James Barrett, Ph.D., GlycoMimetics’ founding venture investor and John Magnani, Ph.D., GlycoMimetics’ Co-founder and Chief Scientific Officer, notified the company that they will not run for reelection to the Board. At the Annual Meeting of Stockholders on May 17, 2019, the Board Chair position will be taken by Tim Pearson, a GlycoMimetics Director since 2014, and until recently, Chief Financial Officer and Executive Vice President for TESARO, Inc., a publicly held oncology-focused biopharmaceutical company recently acquired by GlaxoSmithKline
Fourth Quarter and Year-end 2018 Financial Results:

Cash position: As of December 31, 2018, GlycoMimetics had cash and cash equivalents of $209.9 million as compared to $123.9 million as of December 31, 2017. In March 2018, the Company completed a public offering of 8,050,000 shares of common stock yielding net proceeds of $128.4 million.
R&D Expenses: The Company’s research and development expenses increased to $12.0 million for the quarter ended December 31, 2018 as compared to $6.7 million for the fourth quarter of 2017. Research and development expenses increased by $16.0 million to $40.1 million for the year ended December 31, 2018, from $24.1 million in the year ended December 31, 2017. These increases were primarily the result of higher manufacturing costs to scale up production of uproleselan clinical supplies for the Company’s Phase 3 clinical trial and for clinical trials conducted by or in collaboration with third parties. Personnel-related and stock-based compensation increased due to an increase in clinical headcount.
G&A Expenses: The Company’s general and administrative expenses increased to $2.9 million for the quarter ended December 31, 2018 as compared to $2.8 million for the fourth quarter of 2017. General and administrative expenses for the year ended December 31, 2018 increased to $11.4 million as compared to $9.8 million in the prior year. These increases were primarily due an increase in legal and patent expenses as well as labor-related costs and stock-based compensation expense. Patent expenses were higher due to an increase in the number of patent applications filed. Personnel-related and stock-based compensation expenses increased due to additional headcount in 2018, annual salary adjustments and annual stock option awards granted in the first quarter of 2018.
Shares Outstanding: Shares of common stock outstanding as of December 31, 2018 were 43,160,751.
The company will host a conference call and webcast today at 8:30 a.m. ET. The dial-in number for the conference call is (844) 413-7154 for domestic participants and (216) 562-0466 for international participants, with participant code 5072004. A webcast replay will be available via the "Investors" tab on the GlycoMimetics website for 30 days following the call. A dial-in phone replay will be available for 24 hours after the close of the call by dialing (855) 859-2056 for domestic participants and (404) 537-3406 for international participants, participant code 5072004.

About Uproleselan (GMI-1271)

uproleselan (yoo’ pro le’ sel an) is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed/refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better than expected remission rates and overall survival compared to historical controls, which have been derived from results from third party clinical trials evaluating standard chemotherapy, as well as lower than expected induction-related mortality rates. Treatment in these patient populations was generally well tolerated, with fewer than expected adverse effects. The U.S. Food and Drug Administration (FDA) has granted uproleselan Breakthrough Therapy Designation for the treatment of adult AML patients with relapsed/refractory (R/R) disease. GlycoMimetics is implementing a comprehensive development program across the clinical spectrum of AML. This includes the company-sponsored Phase 3 trial in R/R AML that is currently enrolling patients and two consortia-sponsored trials in newly diagnosed patients. One consortium trial is being sponsored by the NCI and will enroll newly diagnosed patients fit for intensive chemotherapy. The other trial is sponsored by the HOVON group in Europe and will enroll newly diagnosed patients unfit for intensive chemotherapy.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules that keep cancer cells in the bone marrow. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers.

About Rivipansel

rivipansel, the most advanced drug candidate in the GlycoMimetics pipeline, is a glycomimetic drug candidate that acts as a pan-selectin antagonist, meaning it binds to all three members of the selectin family – E-, P- and L-selectin. The first potential indication for rivipansel is vaso-occlusive crisis (VOC) of sickle cell disease (SCD), one of the most severe complications of SCD which can result in acute ischemic organ injury at one or more sites. By reducing cell adhesion, activation and inflammation that are believed to contribute to reduced blood flow through the microvasculature during VOC, GlycoMimetics believes that rivipansel could be the first drug to interrupt the underlying cause of VOC, thereby potentially enabling patients to leave the hospital more quickly. Pfizer is conducting a Phase 3 clinical trial for rivipansel in SCD.

Ayala Pharmaceuticals to Present Preclinical Data at AACR for AL101, a Pan-Notch Inhibitor Being Evaluated for Adenoid Cystic Carcinoma

On March 6, 2019 Ayala Pharmaceuticals, Inc., a clinical-stage company developing medicines for cancers that are genetically defined, reported that new data for AL101 – a gamma secretase inhibitor currently being evaluated as a potential therapy for adenoid cystic carcinoma (ACC) with Notch activating mutations – will be presented at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Atlanta, Ga (Press release, Ayala Pharmaceuticals, MAR 6, 2019, View Source [SID1234534003]).

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The details of the poster presentation are as follows:

Title: AL101 mediated tumor inhibition in Notch mutated ACC PDX models
Date and Time: Wednesday April 3, 2019, 8 a.m. – 12 p.m. EDT
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 19
Poster Board Number: 8
Permanent Abstract Number: 4885

The preclinical data that will be presented show that AL101 monotherapy has a significant effect on tumor growth inhibition in a mouse model using implanted ACC patient-derived xenograft (PDX) tumors. The effect of AL101 was only seen in PDX tumors with Notch activating mutations and not on PDX tumors without such mutations. These data support the clinical development of AL101 as a targeted monotherapy for ACC tumors bearing Notch activating mutations.

"This proof-of-concept study in genetically-defined ACC PDX models validates our patient selection strategy in the ongoing ACCURACY Phase 2 clinical trial, testing AL101 in people with ACC tumors bearing Notch activating mutations," said Roni Mamluk, Ph.D., Chief Executive Officer at Ayala Pharmaceuticals. "These encouraging results bring us one step closer to potentially helping people suffering from ACC, an underserved cancer with no standard of care."

In a previous Phase 1 all-comers solid tumor study, two ACC patients were treated with AL101, the first had partial response and the second had prolonged stable disease. This study did not apply patient selection for Notch activating mutations; however, it was confirmed that both patients had tumors with activating Notch mutations. These results promoted further evaluation of the effect of AL101 in tumors that harbor/lack Notch activated mutations.

Additionally, prior research revealed that genomic alterations in the Notch pathway yield a subset of ACC patients with a distinct phenotype and a more aggressive course of disease than their wild type counterparts. In addition to Notch 1 mutations, mutations in Notch 2 and 4 have been reported in ACC.

About AL101
AL101 is a gamma secretase inhibitor developed as a Notch inhibitor for oncology indications. Notch signaling pathway plays an important role in tumorigenesis in several solid and hematological malignancies. Upon ligand binding of the Notch receptor, an important step in the activation of Notch receptors is cleavage by gamma secretase, which frees the Notch intracellular signaling domain.

AL101 is currently in Phase 2 for adenoid cystic carcinoma patients with tumor bearing Notch activating mutations (ACCURACY). For additional information about AL101 ACCURACY clinical trial, please to www.clinicaltrials.gov. Interested patients and physicians can contact Ayala Medical for more information at: [email protected] or call refer +1-857-444-0553.