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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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 Flow Pharma Inc., a San Francisco Bay Area biotechnology company focused on personalized vaccine development, reported that it has selected BGI Americas Inc. to provide the sequencing services required for all FlowVax neoantigen-based personalized therapeutic cancer vaccines under development by Flow Pharma (Press release, Flow Pharma, FEB 28, 2019, View Source [SID1234554003]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The first two Flow Pharma products under development are FlowVax HPV for the treatment of cervical cancer and FlowVax BreastCA for the treatment of breast cancer. FlowVax Breast CA will be a personalized therapeutic vaccine requiring patient-specific gene sequencing.

"The exciting new area of personalized therapeutic cancer vaccines requires rapid, accurate genomic and transcriptomic sequencing. BGI Americas is unique in providing next generation sequencing services using its own unique DNBseq sequencing technology based on combinatorial probe-anchor synthesis (cPAS), linear isothermal rolling-circle replication and DNA nanoball (DNB) technology," said Victor Zhang, Ph.D., CEO of BGI Americas. "BGI has a strong track record supporting the therapeutic and biomarker development of top biopharma and biotech companies. Our DNBseq technology has been validated by leading academic centers and industry users and will deliver strong value in our collaboration with Flow Pharma."

"In order to make a personalized therapeutic cancer vaccine, you have to know the genomic information of the patient’s tumor as well as that of the patient. Speed and accuracy are both critical. Once we have these gene sequences, we can use AI-augmented bioinformatics tools to quickly identify tumor targets for T-cell attack and then rapidly load these targets into a FlowVax vaccine customized for that particular patient." Said Lu Wang, Ph.D., Flow Pharma bioinformatics team leader

"We have spent a decade developing the FlowVax vaccine platform. FlowVax is optimized to simultaneously deliver multiple unmodified peptide targets for killer T-cell attack. Each of the chemical components used in the FlowVax platform are currently part of an FDA approved vaccine or pharmaceutical, simplifying our regulatory pathway," said Charles Herst, Ph.D., Flow Pharma chief science officer. "Personalized FlowVax therapeutic vaccines can be manufactured quickly and efficiently, keeping the cost down and getting the therapy in the hands of treating physicians soon after the diagnosis is made, for maximum efficacy."

"We believe that BGI’s DNBseq NGS platform is the best in class for the rigorous requirements of personalized therapeutic cancer vaccine development. The fact that BGI Americas provides professional and competitive genomic sequencing services along with experience and knowledge in supporting drug development, allows us to become a full-stack company in the space without the time and expense required to bring gene sequencing technology in-house," said Reid Rubsamen, M.D., CEO of Flow Pharma.

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.