On December 10, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported data from ELZONRIS (tagraxofusp) Phase 1/2 clinical trials in myelofibrosis (oral presentation) and multiple myeloma, at the 2019 ASH (Free ASH Whitepaper) annual meeting (Press release, Stemline Therapeutics, DEC 10, 2019, View Source [SID1234552195]). The presentations are now available on the Stemline website, www.stemline.com, under the Scientific Presentations tab. Highlights of results are presented below.

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Myelofibrosis: Clinical Trial of ELZONRIS in Patients with Intermediate or High-Risk, Relapsed/Refractory Myelofibrosis (MF) – Oral presentation

ELZONRIS demonstrated efficacy (spleen size reductions and total symptom score [TSS] reductions) with a predictable and manageable safety profile, including in patients with poor prognostic factors, such as thrombocytopenia, CMML-type features/monocytosis, and clonal evolution
45% (9/20) of patients had symptom burden reduction, including 3 with symptom response per IWG-MRT 2013 MF response criteria
53% (8/15) of patients with baseline splenomegaly >= 5cm experienced a spleen size reduction; 20% (3/15) had a reduction >35% (specifically 100%, 47% and 46% reductions)
° 100% (5/5) of patients with CMML-type features/monocytosis experienced a spleen size reduction, and 40% (2/5) of these patients had a reduction >35%
° 64% (7/11) of patients with thrombocytopenia (platelet count < 100×109/L) experienced a spleen size reduction, and 18% (2/11) had a reduction >35%; 60% (3/5) of patients with thrombocytopenia (platelet count < 50×109/L) experienced a spleen size reduction, and 20% (1/5) had a reduction >35%

Most common treatment related adverse events (TRAEs) include alanine aminotransferase increase, headache, hypoalbuminemia (all 17%), anemia, and thrombocytopenia (both 14%)
° There was one case of capillary leak syndrome (CLS), which was grade 3
43% overall survival (OS) at 18 months; 29% OS at 2 years
Next Steps

Expansion of Phase 2 trial to include additional sites is underway
Patient enrollment and follow up continues to inform design of potential registration-directed trial of ELZONRIS in patients with relapsed/refractory MF, including poor prognosis patients with:
° Thrombocytopenia, and/or
° Monocytosis (CD123-overexpressed subsets)
Other areas of evaluation in MF

An additional ASH (Free ASH Whitepaper) presentation demonstrated that ELZONRIS, either alone or in combination with ruxolitinib, had preclinical activity against primary MF patient samples, including those in accelerated phase and with high molecular risk profiles where current therapeutic options beyond ruxolitinib are limited and CD123 expression is evident. These data support further evaluation of tagraxofusp in MF, not only as single agent but potentially in combination with Jak inhibitors.
Multiple Myeloma: Clinical Trial of ELZONRIS in Patients with Relapsed/Refractory Multiple Myeloma (MM)

ELZONRIS, in combination with pomalidomide (POM) and dexamethasone (DEX), demonstrated activity and a predictable and manageable safety profile in heavily pretreated patients with relapsed/refractory MM
56% of patients (5/9) who received ELZONRIS in combination with POM+DEX had partial responses (PRs)
° Notably, these patients also had decreases in their plasmacytoid dendritic cells (pDCs), a cell type implicated in myeloma growth and aggressiveness, and the cell of origin of blastic plasmacytoid dendritic cell neoplasm (BPDCN)
Given these clinical data, ELZONRIS may offer a novel mechanism of action and future therapeutic option for patients with MM
Next Steps

Given these encouraging early results, potential avenues for further ELZONRIS development are being assessed, including evaluating additional patients with this and/or potentially other regimens including in combination with daratumumab or novel agents such as XPO1 inhibitors. Moreover, the clinical knockdown of pDCs in MM patients may also be relevant with respect to other diseases, such as chronic myelomonocytic leukemia (CMML), MF and certain acute myeloid leukemia (AML) patient subsets, where pDCs have been similarly implicated in the aggressiveness of these malignancies.
About ELZONRIS
ELZONRIS (tagraxofusp), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

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, as well as other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About CD123
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

On December 10, 2019 Spherix Incorporated (Nasdaq: SPEX) reported that it has closed the CBM BioPharma, Inc. transaction and is working to develop these cancer drugs (Press release, Spherix, DEC 10, 2019, View Source [SID1234552194]).

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Building on its successful investment in Hoth Therapeutics, Inc., Spherix now owns a diverse portfolio of early stage small-molecule anti-cancer therapeutics. The Spherix platform contains patented technology from leading universities and researchers and we seek to develop our innovative drugs through strong partnership with world renowned institutions, such as The University of Texas at Austin and Wake Forest University. The Company’s diverse pipeline of therapeutics includes therapies for pancreatic cancer, acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). Its pancreatic treatment has shown positive preclinical results for inhibiting pancreatic tumor growth in clinically relevant transgenic mouse models. The drug has also demonstrated the potential to overcome tumor cell resistance to current chemotherapeutic drugs. The Company’s AML drug is a next generation targeted therapeutic designed to overcome multiple resistance mechanisms observed with the current standard of care. In addition, Spherix is continually seeking to grow its pipeline to treat unmet medical needs in oncology.

Anthony Hayes, CEO of Spherix, stated, "The acquisition of these assets is yet another step in the transformation of Spherix into a diversified biotechnology company. These are exciting drugs and we intend to provide additional information about these drugs and the development plan moving forward. As the deal has evolved, we have successfully improved the terms for Spherix shareholders, we thank our shareholders for their support during this process."

On December 10, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX) ("Onconova" or the "Company"), a Phase 3-stage biopharmaceutical company discovering and developing novel products to treat cancer, with an initial focus on myelodysplastic syndromes (MDS), reported the closing of its previously announced registered direct offering of 14,326,648 shares of its common stock and warrants to purchase up to 7,163,324 shares of its common stock, at a combined purchase price of $0.349 per share and associated warrant, for aggregate gross proceeds of approximately $5.0 million (Press release, Onconova, DEC 10, 2019, View Source [SID1234552192]). The registered direct offering priced at-the-market under Nasdaq rules.

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H.C. Wainwright & Co. acted as the exclusive placement agent for the offering.

The warrants issued in the offering have an exercise price of $0.287 per share and exercise period commencing immediately upon issuance and a term of five (5) years.

The Company intends to use the net proceeds from the offering for working capital and general corporate purposes, including advancing preparations for a planned New Drug Application (NDA) filing to the FDA for intravenous rigosertib in second-line higher-risk MDS in 2020. The Company surpassed 90% of the required enrollment of the INSPIRE Trial in November 2019 and anticipates reporting topline data in the first half of 2020, following full enrollment and reaching the number of required survival events. With the additional proceeds from the offering and the proceeds from the recent warrant exercise, the Company believes that it has the sufficient funds to extend operations and ongoing trials late into the third quarter of 2020.

The securities described above were offered and sold by the Company pursuant to a "shelf" registration statement on Form S-3 (Registration No. 333-221684), including a base prospectus, previously filed with and declared effective by the Securities and Exchange Commission (the "SEC") on December 28, 2017. The offering of the securities was made only by means of a prospectus supplement that forms a part of the registration statement. A final prospectus supplement and an accompanying base prospectus relating to the registered direct offering was filed with the SEC and is available on the SEC’s website located at View Source Electronic copies of the prospectus supplement and the accompanying base prospectus may also be obtained by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at 646-975-6996 or e-mail at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On December 10, 2019 Galera Therapeutics, Inc. (Nasdaq: GRTX), a clinical-stage biopharmaceutical company focused on developing and commercializing a pipeline of novel, proprietary therapeutics that have the potential to transform radiotherapy in cancer, reported financial results for the third quarter ended September 30, 2019, and highlighted recent corporate accomplishments (Press release, Galera Therapeutics, DEC 10, 2019, View Source [SID1234552191]).

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"2019 has been a momentous year for Galera, culminating in the closing of our IPO last month," said Mel Sorensen, M.D., President and CEO of Galera. "We look forward to continuing to advance our clinical development of GC4419 and GC4711, which we believe could change the management of radiation therapy by both protecting normal tissue and improving the effectiveness of radiation."

Third Quarter 2019 and Recent Corporate Highlights

In November 2019, completed an initial public offering of common stock and raised net proceeds of approximately $58.1 million, after deducting the underwriting discounts and other offering expenses, through the sale of 5,445,690 common shares, including shares sold pursuant to the partial exercise of the underwriters’ option to purchase additional shares in December 2019, at a public offering price of $12.00 per share.

Continued enrollment in the Phase 3 ROMAN clinical trial of GC4419 for the treatment of severe oral mucositis (SOM) in patients with locally advanced head and neck cancer receiving radiotherapy with enrollment expected to be completed by the second half of 2020. Galera anticipates reporting topline data in the first half of 2021.

Continued enrollment in the pilot Phase 1b/2a safety and anti-cancer efficacy clinical trial of GC4419 in patients with locally advanced pancreatic cancer with topline data expected in the second half of 2020.

In December 2019, full results from the 223-patient randomized, double-blind Phase 2b clinical trial of GC4419 for the treatment of SOM in patients with locally advanced head and neck cancer receiving radiotherapy were published in the Journal of Clinical Oncology, a journal of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). The paper, titled, "Phase IIb, Randomized, Double-Blind Trial of GC4419 Versus Placebo to Reduce Severe Oral Mucositis Due to Concurrent Radiotherapy and Cisplatin For Head and Neck Cancer," reinforces the potential of GC4419 to address a serious unmet need for a therapy to reduce the incidence and severity of radiation-induced SOM, for which there is currently no FDA-approved drug.

In October 2019, announced final data from the two-year tumor outcomes follow up of patients enrolled in the Phase 2b clinical trial of GC4419 for the treatment of SOM in patients with locally advanced head and neck cancer receiving radiotherapy. At both the one-year interim assessment and final two-year mark, tumor outcomes were maintained across all four measures – overall survival, progression-free survival, locoregional control and metastasis-free survival – in both GC4419 dose groups (30 mg and 90 mg) compared to placebo.

In October 2019, strengthened Galera’s leadership team with the appointment of Christopher Degnan as Chief Financial Officer.

Third Quarter Financial Highlights

Research and development expenses were $11.0 million in the third quarter of 2019, compared to $4.2 million for the same period in 2018. The increase was primarily attributable to GC4419 and GC4711 development costs. Galera initiated the Phase 3 ROMAN trial in October 2018, began chronic toxicology studies of GC4419 to support registration, and initiated a Phase 1 clinical trial and additional toxicology studies of GC4711.

General and administrative expenses were $1.8 million in the third quarter of 2019, compared to $1.2 million for the same period in 2018. The increase was primarily the result of employee-related costs from increased headcount.

Galera reported a net loss of $(13.4) million, or $(51.43) per share, for the third quarter of 2019, compared to a net loss of $(5.2) million, or $(22.35) per share, for the same period in 2018.

As of September 30, 2019, Galera had cash, cash equivalents, and short-term investments of $67.9 million. Galera expects that its existing cash, cash equivalents and short-term investments, together with the net proceeds from the IPO and assumed payments from Clarus in the amount of $40 million upon the achievement of the two remaining specified clinical milestones in the ROMAN trial, will enable Galera to fund its operating expenses and capital expenditure requirements into 2022.

On December 10, 2019 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported new in vivo preclinical data for FT819, its first off-the-shelf, iPSC-derived chimeric antigen receptor (CAR) T-cell product candidate, at the 61stAmerican Society of Hematology (ASH) (Free ASH Whitepaper) Meeting and Exposition in Orlando, Florida (Press release, Fate Therapeutics, DEC 10, 2019, View Source [SID1234552190]).

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FT819 is derived from a clonal master engineered induced pluripotent stem cell (iPSC) line with complete elimination of T-cell receptor (TCR) expression and a novel 1XX CAR targeting CD19 inserted into the T-cell receptor alpha constant (TRAC) locus. The cell product candidate is being developed under a collaboration with Memorial Sloan Kettering Cancer Center (MSK) led by Michel Sadelain, M.D., Ph.D. The Company has now selected a single engineered iPSC clone, and generated and fully-characterized the master engineered iPSC bank for GMP production of FT819.

"CAR T-cell therapy continues to deliver remarkable outcomes for patients with hematologic malignancies, and next-generation approaches are needed to enable broad and timely patient access and reduce the cost and complexity of therapy," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "With early evidence of clinical activity for our off-the-shelf, iPSC-derived NK cell programs, we are excited to lead in bringing next-generation CAR T-cell therapies to patients and plan to submit an IND for FT819 in the first half of 2020."

The Company’s iPSC product platform unites stem cell biology and precision genetic engineering to create renewable master engineered iPSC lines that can be repeatedly used to mass produce cancer-fighting immune cells, replacing the high production costs, weeks of manufacturing time, and complex engineering processes required for current-generation CAR T-cell immunotherapies with an off-the-shelf product that has the potential to reach many more patients.

At ASH (Free ASH Whitepaper), scientists from the Company and MSK presented new in vivo preclinical data demonstrating that FT819 exhibits durable tumor control and extended survival. In a stringent xenograft model of disseminated lymphoblastic leukemia, FT819 demonstrated enhanced tumor clearance and control of leukemia as compared to primary CAR19 T cells. At Day 35 following administration, a bone marrow assessment showed that FT819 persisted and continued to demonstrate tumor clearance, whereas primary CAR T cells, while persisting, were not able to control tumor growth. Over the past twelve months, the collaboration team has worked to optimize its processes for making T cells from iPSCs, and has now shown the production of pure T-lymphocytes consisting of both CD8+ and CD4+ T cells having a global gene expression profile that is highly-similar to primary T cells based on a principal component analysis.

As proof-of-principle for the unique advantages arising from selecting a single engineered iPSC clone for the production of CAR T-cell therapy, the scientists assessed 747 clones after engineering a pool of cells using CRISPR. It was found that only about 2% of clones met the Company’s standards for overall quality including containing both bi-allelic disruption of the TCR, proper insertion of the CAR into the TRAC locus without random transgene integrations, and no evidence of off-target genomic modifications or translocations. The Company selected the top-performing clone for generation of the master engineered iPSC bank for GMP production of FT819.

Fate Therapeutics has exclusively licensed from MSK foundational intellectual property covering the production and composition of iPSC-derived T cells. In August, the Company announced that the U.S. Patent and Trademark Office issued U.S. Patent No. 10,370,452 covering compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell, including an iPSC. The foundational patent, which expires in 2034, is owned by MSK and is licensed exclusively to Fate Therapeutics for all human therapeutic uses.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 250 issued patents and 150 pending patent applications.