On February 28, 2019 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported financial results for the fourth quarter and full-year ended December 31, 2018 (Press release, Mirati, FEB 28, 2019, View Source [SID1234533854]).

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"Mirati made great progress in 2018 with significant advances in our sitravatinib and KRAS (MRTX849) programs. We remain focused on both programs as we anticipate enrolling second-line NSCLC patients in our Phase 3 randomized trial with sitravatinib in combination with nivolumab in the first half of 2019. We look forward to seeing data from our collaboration with BeiGene, with initial proof of concept data from the clinical trials studying sitravatinib in combination with tislelizumab, in certain tumor types, expected in the second half of 2019. We’re also excited to see MRTX849 advance in the Phase 1/2 trial toward initial clinical efficacy data this year," said Charles M. Baum, M.D., Ph.D., President and Chief Executive Officer. "We are well funded to execute our plans following a successful financing in January and look forward to a pivotal year for Mirati."

Financial Results for the Fourth Quarter and Full Year 2018

Cash, cash equivalents, and short-term investments were $222.8 million at December 31, 2018, compared to $150.8 million at December 31, 2017. In January 2019, we completed a public equity offering with net proceeds of $107.9 million.

License and collaboration revenues for the fourth quarter of 2018 were $3.5 million and for the year ended December 31, 2018 were $12.9 million. There were no license and collaboration revenues during the same periods of 2017. License and collaboration revenues relate to the Collaboration and License Agreement between the Company and BeiGene, Ltd., which became effective January 7, 2018.

Research and development expenses for the fourth quarter of 2018 were $26.8 million, compared to $15.2 million for the same period in 2017. Research and development expenses for the year ended December 31, 2018 were $93.9 million compared to $58.1 million for the same period in 2017. The increase in research and development expenses for both periods is due to an increase in expense associated with sitravatinib and our KRAS inhibitor program, as well as an increase in salaries and related expense, including an increase in share-based compensation expense. The increase in sitravatinib expense is due to increased costs to support the expansion of existing and new clinical trials and the increase in KRAS inhibitor program expense relates primarily to increased manufacturing expenses for our lead clinical compound, MRTX849. The Company recognized research and development-related share-based compensation expense of $2.1 million during the fourth quarter of 2018 as compared to $0.7 million for same period in 2017 and $7.2 million during the year ended December 31, 2018 as compared to $3.2 million for the same period in 2017.

General and administrative expenses for the fourth quarter of 2018 were $6.4 million, compared to $3.0 million for the same period in 2017. General and administrative expenses for the year ended December 31, 2018 were $21.7 million compared to $13.5 million for the same period of 2017. The increase is due primarily to an increase in share-based compensation expense, and to a lesser extent increases in salaries and related expense, professional and consulting fees and patent filing fees. The Company recognized general and administrative-related share-based compensation expense of $2.1 million during the fourth quarter of 2018 as compared to $0.6 million for same period in 2017 and of $8.6 million during the year ended December 31, 2018 as compared to $3.6 million for the same period in 2017.

Net loss for the fourth quarter of 2018 was $28.3 million, or $0.87 per share basic and diluted, compared to net loss of $17.9 million, or $0.67 per share basic and diluted for the same period in 2017. Net loss for the year ended December 31, 2018 was $98.4 million, or $3.19 per share, compared to $70.4 million, or $2.78 per share, for the same period of 2017.

About Sitravatinib

Sitravatinib is a spectrum-selective kinase inhibitor that potently inhibits receptor tyrosine kinases (RTKs), including TAM family receptors (TYRO3, Axl, Mer), split family receptors (VEGFR2, KIT) and RET. As an immuno-oncology agent, sitravatinib is being evaluated in combination with nivolumab (OPDIVO), an anti-PD-1 checkpoint inhibitor, in patients whose cancers have progressed despite treatment with a checkpoint inhibitor. Sitravatinib’s potent inhibition of TAM and split family RTKs may overcome resistance to checkpoint inhibitor therapy through targeted reversal of an immunosuppressive tumor microenvironment, enhancing antigen-specific T cell response and expanding dendritic cell-dependent antigen presentation.

Sitravatinib is also being evaluated as a single agent in a Phase 1b expansion clinical trial emphasizing enrollment of patients whose tumors harbor specific mutations in the CBL protein. When CBL is inactivated by mutation, multiple RTKs, including TAM, VEGFR2 and KIT, are dysregulated and may act as oncogenic tumor drivers in NSCLC and melanoma. Sitravatinib potently inhibits these RTKs and is being investigated as a treatment option for cancer patients with CBL mutations.

About MRTX849

MRTX849 is an investigational, orally-available small molecule, that is designed to potently and selectively inhibit a form of KRAS which harbors a substitution mutation (G12C). KRAS G12C mutations are present in approximately 14% of NSCLC adenocarcinoma patients, 4% of colorectal cancer patients, and subsets of other types of cancer. Tumors characterized by KRAS G12C mutations are commonly associated with poor prognosis and resistance to therapy, and patients with these mutations have few treatment options. MRTX849 is being evaluated in a Phase 1/2 trial treating patients with molecularly-identified KRAS G12C-positive advanced solid tumors.

On February 28, 2019 Odonate Therapeutics, Inc. (NASDAQ: ODT), a pharmaceutical company dedicated to the development of best-in-class therapeutics that improve and extend the lives of patients with cancer, reported the acceptance for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 of an abstract describing results of preclinical studies of tesetaxel, Odonate’s investigational, orally administered taxane (Press release, Odonate Therapeutics, FEB 28, 2019, View Source [SID1234533870]). These preclinical results indicate that, following oral administration, tesetaxel achieves brain concentrations that exceed concentrations required for tumor killing for a sustained period of time. This is in contrast to paclitaxel and docetaxel, the most commonly prescribed taxanes, which do not substantially cross the blood-brain barrier. New therapies are needed for patients with tumors that have metastasized to the brain from other organs, such as the breast or the lung, as well as patients with primary brain tumors. The AACR (Free AACR Whitepaper) Annual Meeting 2019 will be held March 29 – April 3, 2019 in Atlanta, Georgia. The abstract for this presentation was made publicly available yesterday and can be found on the AACR (Free AACR Whitepaper) Annual Meeting 2019 Itinerary Planner or by clicking the title below.

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Presentation Details:

Abstract 3078 (Poster Board #12): Tesetaxel, a novel, oral taxane, crosses intact blood-brain barrier (BBB) at therapeutically relevant concentrations
Session Category: Experimental and Molecular Therapeutics
Session Title: Novel Antitumor Agents 1
Session Date and Time:Tuesday, April 2, 20198:00 AM – 12:00 PM
Location:Georgia World Congress Center, Exhibit Hall B, Poster Section 14

About Tesetaxel

Tesetaxel is an investigational, orally administered chemotherapy agent that belongs to a class of drugs known as taxanes, which are widely used in the treatment of cancer. Tesetaxel has several pharmacologic properties that make it unique among taxanes, including: oral administration with a low pill burden; a long (~8-day) terminal plasma half-life in humans, enabling the maintenance of adequate drug levels with relatively infrequent dosing; no history of hypersensitivity (allergic) reactions; and significant activity against chemotherapy-resistant tumors. In patients with metastatic breast cancer, tesetaxel was shown to have significant, single-agent antitumor activity in two multicenter, Phase 2 studies.

About CONTESSA

CONTESSA is a multinational, multicenter, randomized, Phase 3 study of tesetaxel, an investigational, orally administered taxane, in patients with locally advanced or metastatic breast cancer (LA/MBC). CONTESSA is comparing tesetaxel dosed orally at 27 mg/m2 on the first day of a 21-day cycle plus a reduced dose of capecitabine (1,650 mg/m2/day dosed orally on days 1-14 of a 21-day cycle) to the approved dose of capecitabine alone (2,500 mg/m2/day dosed orally on days 1-14 of a 21-day cycle) in approximately 600 patients randomized 1:1 with human epidermal growth factor receptor 2 (HER2) negative, hormone receptor (HR) positive LA/MBC previously treated with a taxane in the neoadjuvant or adjuvant setting. Capecitabine is an oral chemotherapy agent that is considered a standard-of-care treatment in LA/MBC. Where indicated, patients must have received endocrine therapy with or without a cyclin-dependent kinase (CDK) 4/6 inhibitor. The primary endpoint is progression-free survival (PFS) assessed by an Independent Radiologic Review Committee (IRC). CONTESSA’s secondary efficacy endpoints are overall survival (OS), objective response rate (ORR) assessed by the IRC and disease control rate (DCR) assessed by the IRC. To learn more, please visit www.contessastudy.com.

On February 28, 2019 Theragnostics is reported we have exclusively licensed worldwide rights to a radiolabelled PARP inhibitor for imaging diagnostics and radionuclide therapies from Memorial Sloan Kettering Cancer Center (MSK), USA, a world leading facility in oncology research and cancer treatment (Press release, Theragnostics, FEB 28, 2019, View Source [SID1234533904]).

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"The agreement with MSK provides for access to an exciting piece of intellectual property aligned with Theragnostics strategy to take diagnostic imaging agents and the equivalent targeted radionuclide therapies into the clinic" said Gareth Smith, Chief Operating Officer of Theragnostics. "What is fantastic about the rPARPi technology is that as well as developing a fluorine-18 PET imaging biomarker for PARP the MSK technology is truly a theragnostic approach enabling us to label with well-established iodine isotopes (iodine-131, iodine-123) as well as allowing the development of novel alpha particle therapies with astatine-211."
Greg Mullen, Chief Executive Officer of Theragnostics added "rPARPi therapy has the prospect of providing significant improvement in cancer patient survival in a number of cancers. There are several approved conventional pharmaceutical PARP inhibitors on the market with new indications being approved in several markets in recent months opening up a multibillion dollar opportunity""

About rPARPi
rPARPi was invented and developed within the lab of Thomas Reiner, PhD, at Memorial Sloan Kettering Cancer Center, New York, where the positron emission tomography (PET) agent [18F]PARPi is currently recruiting patients in a Phase I clinical trial in head and neck cancers. The core structure of the developed rPARPi agents allow for their application as theragnostic agents – they can be labelled with diagnostic isotopes as well as with isotopes to enable cancer therapy.

About PARP
Poly (ADP-ribose) polymerase (PARP) enzymes are involved in normal cellular processes including DNA repair. PARP activity and expression are up-regulated in tumour cells and through the inhibition of PARP it is possible to render cancer cells unable to repair local single-stranded DNA breaks. The inability to repair single strand breaks in DNA results in a subsequent double strand DNA breaks and cell death. PARP inhibitors are a rapidly emerging drug class showing promise as anti-cancer agents in a range of tumour types.

On February 28, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported that the company will present at the Barclays Global Healthcare Conference in Miami Beach, Florida on Tuesday, March 12, 2019 at 8:00 a.m. ET (Press release, BioCryst Pharmaceuticals, FEB 28, 2019, View Source [SID1234533790]).

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Links to a live audio webcast and replay of the presentation may be accessed in the Investors section of BioCryst’s website at http://www.biocryst.com.

On February 28, 2019 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage company developing highly differentiated therapeutics targeting the underlying mechanisms of cancer, reported that preclinical data for CG-806, its first-in-class, highly potent oral small molecule pan-FLT3/pan-BTK inhibitor, and APTO-253, its MYC inhibitor, will be presented in two separate posters at the 2019 AACR (Free AACR Whitepaper) Annual Meeting in Atlanta, GA (Press release, Aptose Biosciences, FEB 28, 2019, View Source [SID1234533805]).

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CG-806 Poster Presentation Details:

CG-806, a pan-FLT3 / pan-BTK inhibitor, demonstrates superior potency against cells from IDH-1 mutant and other non-favorable risk groups of AML patients
Date & Time: Monday, April 1, 2019, 8:00 a.m. – 12:00 p.m.
Session Category: Experimental and Molecular Therapeutics
Session Title: Tyrosine Kinase and Phosphatase Inhibitors 1
Abstract Number: 1323
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 15

APTO-253 Poster Presentation Details:

Resistance to APTO-253 caused by internal deletion and alternate promoter usage of the MYC gene in malignant B cells
Date & Time: Monday, April 1, 2019, 1:00 p.m. – 5:00 p.m.
Session Category: Experimental and Molecular Therapeutics
Session Title: Drug Resistance 3
Abstract Number: 2096
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 15

All abstracts will be available on the AACR (Free AACR Whitepaper) website, www.aacr.org, and published in the 2018 Proceedings of the AACR (Free AACR Whitepaper).

About CG-806
CG-806 is an oral, first-in-class pan-FLT3/pan-BTK multi-cluster kinase inhibitor. This small molecule, in-licensed from CrystalGenomics Inc. in Seoul S. Korea, demonstrates potent inhibition of wild type and all mutant forms of FLT3 (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), cures animals of acute myeloid leukemia (AML) tumors in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with AML. Likewise, CG-806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser (C481S) mutant forms of the BTK enzyme, as well as other oncogenic kinase pathways operative in B cell malignancies, suggesting CG-806 may be developed for various B cell malignancy patients (including CLL, SLL, MCL, DLBCL and others) that are resistant/refractory/intolerant to covalent BTK inhibitors. Because CG-806 targets key kinases/pathways operative in malignancies derived from the bone marrow, it is in development for B cell cancers and AML.

About APTO-253
APTO-253 is a clinical-stage, small molecule, targeted therapeutic agent that inhibits expression of the MYC oncogene, leading to cell cycle arrest and programmed cell death (apoptosis) in human-derived solid tumor and hematologic cancer cells. The MYC oncogene is overexpressed in hematologic cancers, including acute myeloid leukemia (AML). Aptose researchers have reported the ability of APTO-253 to induce cell death, or apoptosis, in multiple blood cancer cell lines including AML, as well as in vitro synergy with various classes of conventional approved and investigational therapies for AML or myelodysplastic syndromes (MDS). New findings reveal that APTO-253 might also serve certain solid tumor patients with BRCA1/2 mutations, but without causing toxicity to the normal bone marrow functions.