MIRATI THERAPEUTICS REPORTS YEAR-END 2019 FINANCIAL RESULTS AND RECENT CORPORATE UPDATES

On February 25, 2020 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported financial results for the fourth quarter and full year-ended December 31, 2019 (Press release, Mirati, FEB 25, 2020, View Source [SID1234554732]).

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"In 2019, we advanced MRTX849, our KRAS G12C inhibitor into a Phase 1/2 clinical trial, showing initial promising data, while continuing to move forward our potentially registration-enabling Phase 3 trial of sitravatinib in combination with a checkpoint inhibitor for non-small cell lung cancer (NSCLC)," said Charles M. Baum, M.D., Ph.D., President and Chief Executive Officer at Mirati. "We continue to grow our targeted oncology portfolio, including investigational new drug (IND)-enabling toxicology studies for our lead KRAS G12D candidates. We are also strengthening our organizational capabilities as we prepare the company to further develop and deliver our novel cancer therapies to the patients most in need of treatments."

RECENT CORPORATE UPDATES:

MRTX849 (KRAS G12C Inhibitor)

Began enrolling patients in the single-agent MRTX849 Phase 2 registrational arm of the KRYSTAL trial as 2nd or 3rd line therapy in NSCLC

Initiated both the combinations of MRTX849 and a PD-1 (pembrolizumab) in NSCLC and MRTX849 and an EGFR inhibitor (cetuximab) in colorectal cancer (CRC) under arms of the KRYSTAL trial

KRAS G12D Inhibitor

Advanced lead candidates in our KRAS G12D inhibitor program into potentially IND-enabling toxicology studies.

Sitravatinib

Presented data in two presentations at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 34th Annual Meeting on November 9, 2019.

Interim Phase 2 data for sitravatinib in combination with nivolumab in urothelial carcinoma. As of the cut-off date of October 17, 2019, 22 patients were evaluable for response with at least one radiographic scan. 6/22 evaluable patients achieved a confirmed Complete Response (CR, 1 patient) or Partial Response (PR, 5 patients). 21/22 evaluable patients

achieved a CR, PR or stable disease and 4/6 responding patients had been treated for more than 6 months. The combination has been well-tolerated.

Interim data in the investigator sponsored Phase 1 SNOW trial (sitravatinib and nivolumab in oral cavity cancer window of opportunity study) was announced in a poster presentation. The preliminary data suggest that the combination of neoadjuvant sitravatinib and nivolumab is safe and active in patients with squamous cell carcinoma of the oral cavity who are candidates for resection. As of the data cut-off date of October 9, 2019, 9 patients had been enrolled. Tumor reduction was observed in all eight patients who were eligible for evaluation, including one complete pathological response. All patients received postoperative radiation therapy, and none required postoperative chemotherapy. The median follow-up shown was 31.4 weeks and all patients were alive with no disease recurrence as of the cut-off date. In most patients, treatment with sitravatinib led to a decrease in myeloid-derived suppressor cells and a shift towards M1-type macrophages in the tumor microenvironment, demonstrating the ability of sitravatinib to re-sensitize the tumor microenvironment to immunotherapy.

Presented interim data for sitravatinib in combination with nivolumab in patients with advanced clear cell renal cell carcinoma (aCCRCC) at the recent ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium on February 15, 2020 in an investigator sponsored trial. As of the data cut-off date of January 1, 2020, 38/40 patients with aCCRCC who had progressed following treatment with a VEGF-targeted therapy, were evaluable for response after greater than 12 weeks on therapy:

15/38 (39%) patients achieved a confirmed partial response (PR) including one PR that had improved to an unconfirmed complete response (CR) and 35/38 (92%) patients achieved clinical benefit (combination of stable disease plus PR plus CR). Initial median progression-free survival (PFS) was 10.3 months with median overall survival (OS) had not yet been reached (median follow-up was 17.7 months) with 30/38 patients (79%) still on study as of the data cut-off date. The combination of sitravatinib with nivolumab was well tolerated with manageable adverse events.

Operational Updates

On January 6th, 2020, announced the appointment of Dan Faga as Executive Vice President, Chief Operating Officer and Ben Hickey as Executive Vice President, Chief Commercial Officer.

Ended the year with $415.1 million in cash, cash equivalents, and short-term investments and, in addition, strengthened our balance sheet by completing a public offering of common stock on January 14th, 2020 that provided net proceeds of $324.1 million.
ncial Results for the Fourth Quarter 2019

License and collaboration revenues relate to the Collaboration and License Agreement between the Company and BeiGene, Ltd. ("BeiGene"), dated January 7, 2018. License and collaboration revenues for the three and twelve months ended December 31, 2019 were $0.5 million and $3.3 million, respectively, compared to $3.5 million and $12.9 million for the three and twelve months ended December 31, 2018, respectively. The 2019 revenues relate to revenues earned related to a manufacturing supply services agreement with BeiGene ("MSSA"), and the 2018 revenues relate primarily to the transfer of the license and associated know-how to BeiGene, as well as revenues earned related to the MSSA.

Research and development expenses for the fourth quarter of 2019 were $62.9 million, compared to $26.8 million for the same period in 2018. Research and development expenses for the year ended

December 31, 2019 were $182.9 million, compared to $93.9 million for the same period in 2018. The increase in research and development expenses is due to an increase in expense associated with the development of sitravatinib and MRTX849, as well as an increase in salaries and related expense, including an increase in share-based compensation expense. The Company recognized research and development-related share-based compensation expense of $10.6 million during the fourth quarter of 2019, compared to $2.1 million for the same period in 2018, and $31.0 million during the year ended December 31, 2019, compared to $7.2 million for the same period in 2018.

General and administrative expenses for the fourth quarter of 2019 were $12.2 million, compared to $6.4 million for the same period in 2018. General and administrative expenses for the year ended December 31, 2019 were $42.6 million, compared to $21.7 million for the same period in 2018. The increase is due primarily to an increase in share-based compensation expense and, to a lesser extent, an increase in employee related expense, professional services expense and facilities and insurance expense. The Company recognized general and administrative-related share-based compensation expense of $6.1 million during the fourth quarter of 2019, compared to $2.1 million for the same period in 2018, and $24.5 million during the year ended December 31, 2019, compared to $8.6 million for the same period in 2018.

Net loss for the fourth quarter of 2019 was $72.4 million, or $1.83 per share basic and diluted, compared to net loss of $28.3 million, or $0.87 per share basic and diluted for the same period in 2018. Net loss for the year ended December 31, 2019 was $213.3 million, or $5.69 per share basic and diluted, compared to net loss of $98.4 million, or $3.19 per share basic and diluted for the same period in 2018.

Cash, cash equivalents, and short-term investments were $415.1 million at December 31, 2019, compared to $222.8 million at December 31, 2018. In January 2020, we completed a public offering of common stock that provided net cash proceeds of $324.1 million.

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 non-small cell lung cancer 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.

About Sitravatinib

Sitravatinib is an investigational 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 being evaluated in multiple clinical trials to treat patients who are refractory to prior immune checkpoint inhibitor therapy, including the ongoing potentially registration-enabling Phase 3 trial of sitravatinib in combination with a checkpoint inhibitor in non-small cell lung cancer (NSCLC). In addition, sitravatinib combinations with checkpoint inhibitors are being evaluated in selected checkpoint inhibitor naïve patients.

MannKind Corporation Reports 2019 Fourth Quarter and Full Year Financial Results

On February 25, 2020 MannKind Corporation (NASDAQ:MNKD) reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Mannkind, FEB 25, 2020, View Source [SID1234554731]).

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"We ended 2019 on a strong note with fourth quarter 2019 Afrezza net revenue of $7.8 million, our best quarter to date," said Michael Castagna, Chief Executive Officer of MannKind Corporation. He also noted, "Our collaboration with United Therapeutics on TreT continues to make progress along the clinical and commercial pathways that are expected to set up a filing with the FDA within 12 months."

Fourth Quarter 2019 Results

Total revenues were $16.0 million for the fourth quarter of 2019, reflecting Afrezza net revenue of $7.8 million and collaborations and services revenue of $8.2 million. Afrezza net revenue increased 35% compared to $5.7 million in the fourth quarter of 2018, primarily driven by higher product demand and price. Collaborations and services revenue decreased $2.1 million compared to the fourth quarter of 2018, primarily due to lower revenue from the United Therapeutics research agreement, which was substantially completed by the second quarter of 2019.

On a GAAP basis, Afrezza gross profit was $3.1 million for the fourth quarter of 2019 compared to $0.7 million in the same period in 2018. Afrezza cost of goods sold for the fourth quarter of 2018 included a fee of $2.0 million recorded in connection with the amendment of our insulin supply agreement with Amphastar. As a result, on a non-GAAP basis, gross profit was $2.7 million or 48% gross margin for the fourth quarter of 2018, compared to a GAAP basis gross margin of 40% for the fourth quarter of 2019.

Research and development (R&D) expenses for the fourth quarter of 2019 were $2.0 million compared to $1.1 million for the fourth quarter of 2018. This 82% increase was primarily attributable to an increase of $0.6 million in personnel costs associated with the United Therapeutics research agreement, which was classified as a cost of collaborations and services revenue in 2018, and to an increase of $0.3 million in maintenance costs for our research facility in Danbury, Connecticut.

Selling, general and administrative (SG&A) expenses for the fourth quarter of 2019 were $15.7 million compared to $18.0 million for the fourth quarter of 2018. This decrease of $2.3 million, or 13%, was primarily attributable to a $1.7 million decrease in personnel related costs and a $1.1 million decrease in Afrezza marketing costs, partially offset by an increase of $0.5 million in consulting and professional services costs.

Interest expense on debt for the fourth quarter of 2019 was $2.3 million compared to $1.7 million for the fourth quarter of 2018. This $0.6 million increase, or 35%, was primarily attributable to the net increase in our debt balance as a result of our debt restructuring in the third quarter of 2019.

The net loss for the fourth quarter of 2019 was $14.3 million, or $0.07 per share compared to a $9.8 million net loss in the fourth quarter of 2018 or $0.06 per share. The increase in the net loss of $4.5 million was primarily the result of an increased loss on foreign currency translation of $4.0 million associated with our insulin supply agreement denominated in Euros and of an increase of $0.6 million in interest expense.

Twelve Months Ended December 31, 2019

Total revenues were $63.0 million for the year ended December 31, 2019, reflecting Afrezza net revenue of $25.3 million and collaborations and services revenue of $37.7 million. Afrezza net revenue increased 46% compared to $17.3 million for the year ended December 31, 2018, primarily due to higher product demand, the first sale of Afrezza to our marketing partner in Brazil (Biomm), price increases as well as a more favorable mix of Afrezza cartridges. Collaborations and services revenue increased $27.2 million compared to the full year ended December 31, 2018, which was primarily attributed to the licensing and research agreements with United Therapeutics, both of which began in the fourth quarter of 2018.

On a GAAP basis, Afrezza gross profit was $5.2 million for the year ended December 31, 2019, an improvement of $7.3 million or 347% compared to a gross loss of $2.1 million in the same period in 2018, primarily due to an increase of $8.0 million in net revenue and a $2.2 million decrease in inventory write-offs, partially offset by increased costs due to higher sales and an increase of $0.8 million in fees paid to Amphastar for amendments to our insulin supply agreement. As a result, on a non-GAAP basis, gross profit was $8.0 million, or 32%, for the year ended December 31, 2019 compared to a gross loss of $0.1 million, or 1%, for the full year ended December 31, 2018.

R&D expenses for the year ended December 31, 2019 were $6.9 million compared to $8.7 million for the year ended December 31, 2018. This decrease of $1.8 million, or 21%, was primarily attributable to a $1.7 million decrease in personnel related costs and a $0.9 million decrease in clinical trial spending, partially offset by increased expenses of $0.2 million related to the development of our BluHale inhalation profiling apparatus and increased facility maintenance and equipment repair costs of $0.5 million.

SG&A expenses for the year ended December 31, 2019 were $74.7 million compared to $79.7 million for the year ended December 31, 2018. This decrease of $5.0 million, or 6%, was primarily attributable to a $6.6 million decrease in personnel and employee related costs, decreased consulting costs of $2.5 million and a $1.2 million decrease in stock-based compensation costs, which was partially offset by a $5.6 million increase in costs for television advertising for Afrezza.

Interest income increased by $0.5 million, or 99%, for the year ended December 31, 2019 compared to the year ended December 31, 2018, primarily due to a higher average balance on money market funds and short-term investments.

Interest expense on debt for the year ended December 31, 2019 was $10.9 million compared to $9.4 million for the year ended December 31, 2018. This $1.5 million increase was primarily attributable to a $3.4 million charge realized as a result of achieving a sales milestone in the third quarter of 2019 under our milestone agreement with Deerfield, partially offset by lower interest as a result of the repayment of the Deerfield credit facility.

The net loss for the year ended December 31, 2019 was $51.9 million, or $0.27 per share, compared to $87.0 million net loss for the year ended December 31, 2018, or $0.60 per share. The lower net loss was mainly attributable to a $35.2 million increase in total revenues.

Cash, Cash Equivalents, Restricted Cash and Short Term Investments

Cash, cash equivalents, restricted cash, and short-term investments at December 31, 2019 was $50.2 million compared to $71.7 million at December 31, 2018.

Non-GAAP Measures

Certain financial information contained in this press release is presented on both a reported basis (GAAP) and a non-GAAP basis. Reported results were prepared in accordance with GAAP whereas non-GAAP measures exclude items described in the reconciliation tables below. Non-GAAP financial information is intended to portray the results of our baseline performance, supplement or enhance management, analysts and investors overall understanding of our underlying financial performance and facilitate comparisons among current and past periods. The non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP.

Conference Call

MannKind will host a conference call and presentation webcast to discuss these results today at 9:00 a.m. Eastern Time. To participate in the live call by telephone, please dial (800) 289-0438 or (323) 794-2423 and use the participant passcode: 1233728. Those interested in listening to the conference call live via the Internet may do so by visiting the Company’s website at View Source under News & Events.

A telephone replay of the call will be accessible for approximately 14 days following completion of the call by dialing (844) 512-2921 or (412) 317-6671 and use the participant passcode: 1233728. A replay will also be available on MannKind’s website for 14 days.

MacroGenics Provides Update on Corporate Progress and 2019 Financial Results

On February 25, 2020 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, provided an update on its recent corporate progress and reported financial results for the year ended December 31, 2019 (Press release, MacroGenics, FEB 25, 2020, View Source [SID1234554730]).

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"2019 was an important year for MacroGenics, with the submission of our BLA for margetuximab for HER2-positive breast cancer. We also initiated a registration directed clinical study with margetuximab in combination with checkpoint blockade in advanced HER2-positive gastric cancer," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "During 2020, we anticipate additional key events. Following promising data presented at ASH (Free ASH Whitepaper) in 2019, we plan to define a registration path for flotetuzumab in refractory AML, pending further discussions with FDA. We also look forward to presenting initial clinical data from several of our programs currently in Phase 1, potentially including our two bispecific DART molecules that provide dual checkpoint blockade, MGD013 and MGD019, as well as MGC018, our ADC targeting B7-H3. We look forward to a productive year for MacroGenics in 2020."

Key Pipeline Updates

Recent progress and anticipated events in 2020 related to MacroGenics’ investigational product candidates in clinical development are highlighted below.

Margetuximab is an Fc-engineered, anti-HER2 monoclonal antibody being evaluated for the treatment of patients with advanced HER2-positive cancers.

Metastatic Breast Cancer. In December 2019, MacroGenics submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for margetuximab for the treatment of patients with metastatic HER2-positive breast cancer in combination with chemotherapy. The safety and efficacy results provided in the BLA are primarily from the pivotal Phase 3 SOPHIA study, which is evaluating margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy in patients with HER2-positive metastatic breast cancer who have received prior anti-HER2 therapies. Updated results from the study were presented at the San Antonio Breast Cancer Symposium in December 2019. In February 2020, the BLA was accepted for review by the FDA. MacroGenics expects that there will be a Standard Review process and we anticipate a Prescription Drug User Fee Act (PDUFA) date by the end of 2020. In addition, the Company believes that the FDA will require an Oncologic Drugs Advisory Committee (ODAC) meeting in the second half of 2020.

Separately, in February 2020, MacroGenics’ regional partner in Greater China, Zai Lab Limited (Zai Lab), announced the initiation of a registrational bridging study of margetuximab plus chemotherapy for the treatment of patients with metastatic HER2-positive breast cancer who have received prior anti-HER2 therapies.

Advanced Gastric and Gastroesophageal Junction Cancer. In October 2019, MacroGenics announced the initiation of the Phase 2/3 MAHOGANY study designed to evaluate the combination of margetuximab with anti-PD-1-based therapies as a front-line treatment. Initial safety and efficacy data are expected in the second half of 2020 from Module A of this study, which is evaluating a chemotherapy-free regimen. Module A has been designed to support a potential accelerated approval in the U.S. based on evaluation of objective response rate in a single-arm study.
Flotetuzumab is a bispecific CD123 x CD3 DART molecule being evaluated for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML). At the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2019, MacroGenics presented updated results from patients with AML who are refractory to induction treatment (primary induction failure) in a Phase 1/2 dose expansion study. The Company intends to define a potential registration path in the U.S. for the treatment of patients with primary induction failure AML in the first half of 2020, pending continued discussions with the FDA.

In October 2019, MacroGenics initiated a Phase 1/2 study outside of the U.S. combining flotetuzumab with MGA012, an anti-PD-1 antibody, based on preclinical and translational data that indicate the combination may enhance CD123-directed T cell killing.

MGA012 (INCMGA0012) is an anti-PD-1 monoclonal antibody that has been exclusively licensed to Incyte Corporation. There are currently six registration-directed clinical studies ongoing or planned in 2020 across a broad range of tumor types.

MGD013 is a first-in-class, bispecific PD-1 x LAG-3 DART molecule being evaluated in a Phase 1 dose expansion study. MacroGenics is selecting one or more indications for further development and has submitted data from select cohorts in the ongoing study for presentation at a scientific conference in the first half of 2020. Separately, in February 2020, MacroGenics’ regional partner in Greater China, Zai Lab, announced the initiation of a Phase 1 study of MGD013 in combination with niraparib, a PARP (poly [ADP-ribose] polymerase) inhibitor, for the treatment of patients with advanced gastric or gastroesophageal junction cancer.

Enoblituzumab is an Fc-engineered, anti-B7-H3 monoclonal antibody. In 2020, MacroGenics plans to evaluate the activity of both enoblituzumab plus MGA012 and enoblituzumab plus MGD013 as chemotherapy-free regimens in front-line patients with recurrent and metastatic squamous cell carcinoma of the head and neck (SCCHN) as a lead-in module before proceeding with one of these combinations in a Phase 2/3 study.

MGC018 is an antibody-drug conjugate (ADC) targeting B7-H3 and MGD019 is a bispecific PD-1 x CTLA-4 DART molecule. The Company expects to complete dose escalation for each of these molecules in 2020 and then initiate focused dose expansion studies in select tumor types. In addition, MacroGenics expects to submit data from the dose escalation cohorts for presentation at scientific conferences in 2020.

2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2019 were $215.8 million, compared to $232.9 million as of December 31, 2018.

Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $64.2 million for the year ended December 31, 2019, compared to $60.1 million for the year ended December 31, 2018. This increase was primarily due to the timing of revenue recognition under our collaborative agreements.

R&D Expenses: Research and development expenses were $195.3 million for the year ended December 31, 2019, compared to $190.8 million for the year ended December 31, 2018. This increase was primarily due to continued enrollment in multiple ongoing clinical trials.

G&A Expenses: General and administrative expenses were $46.1 million for the year ended December 31, 2019, compared to $40.5 million for the year ended December 31, 2018. This increase was primarily due to an increase in consulting costs related to market research and other commercial preparation activities.

Net Loss: Net loss was $151.8 million for the year ended December 31, 2019, compared to net loss of $171.5 million for the year ended December 31, 2018.

Shares Outstanding: Shares outstanding as of December 31, 2019 were 48,958,763.

Cash Runway Guidance: MacroGenics anticipates that its cash, cash equivalents and marketable securities as of December 31, 2019, combined with anticipated and potential collaboration payments, will enable it to fund its operations into 2021, assuming the Company’s programs and collaborations advance as currently contemplated. Through the prioritization of programs and ongoing realignment of its resources, MacroGenics is focused on extending its cash runway into 2022.
Conference Call Information

MacroGenics will host a conference call today at 4:30 pm (ET) to discuss financial results for the year ended December 31, 2019 and provide a corporate update. To participate in the conference call, please dial (877) 303-6253 (domestic) or (973) 409-9610 (international) five minutes prior to the start of the call and provide the Conference ID: 5581596.

The listen-only webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of the Company’s website at View Source A replay of the webcast will be available shortly after the conclusion of the call and archived on the Company’s website for 30 days following the call.

Kura Oncology Reports Fourth Quarter and Full Year 2019 Financial Results

On February 25, 2020 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported fourth quarter and full year 2019 financial results and provided a corporate update (Press release, Kura Oncology, FEB 25, 2020, View Source [SID1234554729]).

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"We entered the new year with three wholly owned, clinical-stage drug candidates and a focus on operational execution across our pipeline," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "The conduct of AIM-HN, our registrational-directed trial of tipifarnib in HRAS mutant head and neck squamous cell carcinoma (HNSCC), remains our highest priority. We have also taken a number of steps to better position the company for our next phase of growth, including an increasing investment in pre-commercial activities, expansion of our team of medical science liaisons and the appointment of a Chief Commercial Officer.

"Meanwhile, our growing body of clinical data in T-cell lymphomas, coupled with positive regulatory feedback and the high unmet need for these patients, support our efforts to expand the development of tipifarnib beyond our initial focus in HRAS mutant solid tumors," Dr. Wilson continued. "And we continue to make progress with our emerging drug candidates, KO-947 and KO-539, each with the potential to address genetically defined populations of cancer patients. We believe we are well positioned to advance each of our programs to meaningful inflection points, and we look forward to providing updates on our progress in the year ahead."

Recent Highlights

Strengthened leadership team to support pre-commercial efforts – In January 2020, Kura appointed Kirsten Flowers to the newly created position of Chief Commercial Officer. Ms. Flowers joined from Array Biopharma, where she served as head of commercial operations until the completion of its $11.4 billion acquisition by Pfizer in July 2019. Ms. Flowers was responsible for building and leading the commercial organization that delivered the successful launch of Braftovi + Mektovi for patients with BRAF-mutant melanoma in the U.S. Previously, she held several leadership roles at Pfizer, including U.S. commercial lead for the launches of Ibrance in breast cancer and Inlyta in renal cell carcinoma.

Fast Track designation for tipifarnib in HRAS mutant HNSCC – In December 2019, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to tipifarnib for the treatment of patients with HRAS mutant HNSCC after progression on platinum therapy. Fast Track Designation highlights the potential for tipifarnib to address unmet need for patients with this devastating disease. The designation offers the opportunity for frequent interactions with the FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, as well as eligibility for rolling submission of a New Drug Application.

Robust activity from tipifarnib in advanced T-cell lymphomas – Kura reported updated clinical data from its Phase 2 trial of tipifarnib as a monotherapy in relapsed or refractory nodal T-cell lymphomas, including angioimmunoblastic T-cell lymphoma (AITL). The data, presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2019, showed an objective response rate (ORR) of approximately 50% in heavily pre-treated AITL patients. Patients who carried mutations in the killer-cell immunoglobulin-like receptor (KIR), a CXCL pathway-associated biomarker, achieved an ORR of 70% and a complete response rate of 40%.

Positive feedback from end of Phase 2 meeting with the FDA – Kura is now preparing to initiate a registration-directed trial of tipifarnib in advanced nodal lymphomas of T-follicular helper (TFH) origin, including AITL. This single-arm trial will target enrollment of 128 patients who are relapsed or refractory to at least one prior systemic cytotoxic therapy. The trial has two independent primary objectives, ORR in all patients enrolled and ORR in patients who carry KIR mutations. Each objective can statistically be met independently. Feedback from the Company’s end-of-Phase 2 meeting with the FDA indicate that the trial, as designed, could support a New Drug Application seeking accelerated approval for tipifarnib monotherapy in lymphomas of TFH origin and/or lymphomas of TFH origin with KIR mutations.

New preclinical data support proof-of-concept study in pancreatic cancer – Kura has generated new preclinical data showing tipifarnib’s ability to block CXCL12 production by activated pancreatic stellate cells. In addition, the potential for synergy between tipifarnib and chemotherapy has been observed in preclinical models of pancreatic cancer. The Company believes these preclinical data, coupled with a previously identified association between CXCL12 expression and clinical benefit, support an upcoming proof-of-concept trial of tipifarnib in combination with chemotherapy in the second-line setting for the treatment of advanced pancreatic cancer.

Recommended Phase 2 dose defined for ERK inhibitor KO-947 – Kura believes it has defined a recommended Phase 2 dose for KO-947 as a monotherapy. Pending agreement with the FDA, the Company plans to initiate an expansion cohort to evaluate KO-947 in HNSCC and esophageal squamous cell carcinoma (ESCC) patients with 11q13 amplifications, which are genetically defined populations identified as particularly sensitive to KO-947 as a monotherapy in preclinical models. Recently, a dose-limiting serious adverse drug reaction was observed in a single patient enrolled in the Phase 1 dose-escalation trial of KO-947. Although patients were permitted to remain on therapy, the Company voluntarily paused enrollment, and the FDA placed the trial on a partial clinical hold. Kura is working to lift the hold and resume dosing at the recommended Phase 2 dose with additional safety monitoring in place.

Dose-escalation continues for menin-MLL inhibitor KO-539 –KO-539 is a first-in-class, potent and selective small molecule inhibitor of the menin-MLL protein-protein interaction. Kura commenced a Phase 1/2 clinical trial of KO-539 in relapsed/refractory acute myeloid leukemia (AML) last year and the trial continues in the dose-escalation phase. The Company’s goal is to reach a recommended Phase 2 dose or maximum tolerated dose with the potential to enrich in NPM1-mutant AML and MLL-rearranged genetically defined populations later this year.
Financial Results

Research and development expenses for the fourth quarter of 2019 were $13.5 million, compared to $12.1 million for the fourth quarter of 2018. Research and development expenses for the full year 2019 were $47.8 million, compared to $46.8 million for the prior year.

General and administrative expenses for the fourth quarter of 2019 were $5.5 million, compared to $4.6 million for the fourth quarter of 2018. General and administrative expenses for the full year 2019 were $19.7 million, compared to $16.1 million for the prior year.

Net loss for the fourth quarter of 2019 was $17.9 million, compared to a net loss of $16.1 million for the fourth quarter of 2018. Net loss for the full year 2019 was $63.1 million, compared to a net loss of $60.4 million for the prior year. Net loss for the fourth quarter and full year of 2019 included non-cash, share-based compensation expense of $2.4 million and $9.4 million, respectively, compared to $1.7 million and $8.7 million for the same periods in 2018, respectively.

Cash, cash equivalents and short-term investments totaled $236.9 million as of December 31, 2019, compared with $179.0 million as of December 31, 2018. Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund its current operations into 2022.
Upcoming Milestones

Additional data from Phase 2 trials of tipifarnib in HRAS mutant solid tumors, including HRAS mutant urothelial carcinoma, in 2020

Initiation of a registration-directed trial of tipifarnib in AITL in the second half of 2020

Initiation of a proof-of-concept trial of tipifarnib in pancreatic cancer in the second half of 2020

Additional data from Phase 2 trial of tipifarnib in chronic myelomonocytic leukemia (CMML) in 2020

Potential for full enrollment in the AIM-HN registration-directed trial of tipifarnib in HRAS mutant HNSCC in the first quarter of 2021

Open expansion cohort of patients with HNSCC and ESCC with 11q13 amplifications in Phase 1 trial of KO-947 in 2020

Achievement of a recommended Phase 2 dose in the Phase 1/2 dose-escalation trial of KO-539 by the end of 2020
Conference Call and Webcast

Kura’s management will host a webcast and conference call today at 4:30 p.m. ET / 1:30 p.m. PT today, February 25, 2020, to discuss the financial results for the fourth quarter and full year 2019 and provide a corporate update. The live call may be accessed by dialing (877) 516-3514 for domestic callers and (281) 973-6129 for international callers and entering the conference code: 8077975. A live webcast of the call will be available from the Investors and Media section of the Company’s website at www.kuraoncology.com, and will be archived there for 30 days.

Iovance Biotherapeutics Reports Fourth Quarter and Full-Year 2019 Financial Results and Provides Corporate Update

On February 25, 2020 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies (tumor-infiltrating lymphocyte, TIL and peripheral-blood lymphocyte, PBL), reported fourth quarter and year-end 2019 financial results and provided a corporate update (Press release, Iovance Biotherapeutics, FEB 25, 2020, View Source [SID1234554728]).

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"During 2019 we made tremendous progress in advancing Iovance TIL and PBL products," said Maria Fardis, Ph.D., MBA, Iovance President and Chief Executive Officer. "We conducted two planned pivotal programs for lifileucel in melanoma and LN-145 in cervical, initiated patient dosing in earlier lines of therapy and received clearance from FDA on a new IND to proceed to dose patients with our PBL product, IOV-2001. In anticipation for commercialization of Iovance TIL, we are building our internal manufacturing capability and expanding our commercial team and infrastructure. With the first potential cell therapy in solid tumors, as well as a broad TIL platform, Iovance is poised to become the leader in development, manufacturing, and commercialization of TIL cell therapy for solid tumors."

2019 Highlights and 2020 Updates

Clinical

·Initiation and completion of patient dosing in the pivotal melanoma program, cohort 4 in the C-144-01 trial: Patient dosing in the pivotal cohort 4 of the C-144-01 study was initiated in March 2019 and completed in January 2020, three months ahead of schedule. Iovance intends to submit a biologics license application (BLA) to FDA subsequent to consultation with the agency in 2020.
·Presentation of data for lifileucel in metastatic melanoma and LN-145 for metastatic cervical cancer at major medical meetings: Several clinical data presentations continued to demonstrate efficacy and safety of lifileucel in metastatic melanoma (from C-144-01 study) and LN-145 in recurrent, metastatic, or persistent cervical cancer (from C-145-04 study).
oMelanoma update
·The data for the C-144-01 study in melanoma demonstrated a 36.4% ORR by investigator as presented at ASCO (Free ASCO Whitepaper).1
·In a recent data cut for the C-144-01 study, a median duration of response (DOR) was not reached at 15.5 months of median study follow up (Jan. 2020).2
·Expansion of TIL treatment to earlier lines of therapy for melanoma, head and neck, and non-small cell lung cancer (NSCLC) patients. Patient dosing in the IOV-COM-202 clinical study was initiated in May 2019 to evaluate TIL plus pembrolizumab in patients with immune checkpoint inhibitor naïve melanoma, squamous cell carcinoma of the head and neck (HNSCC), and NSCLC. In the fourth cohort, LN-145 alone is offered to relapsed/refractory NSCLC patients.

Regulatory

·Receipt of Breakthrough Therapy designation (BTD), Fast Track designation and holding End of Phase 2 Meeting for LN-145 in cervical cancer: LN-145 was granted Fast Track and BTD from FDA in recurrent, metastatic or persistent cervical cancer with disease progression on or after chemotherapy. Following an end of Phase 2 meeting, the ongoing C-145-04 clinical study was expanded to dose 75 patients to support registration of LN-145. Additional cohorts in earlier and later line cervical cancer patients were added to study C-145-04 in anticipation of a changing landscape in this indication, specifically, a cohort allowing patients post anti-PD-1 was added.
·Clearance of the investigational new drug (IND) application by FDA and initiation of patient dosing for PBL (IOV-2001) therapy in the first hematologic indication, CLL: An IND application for IOV-2001 was accepted by FDA in the blood cancer indication, relapsed or refractory CLL or small lymphocytic lymphoma (SLL). The first patient was dosed in a Phase 1/2 study (IOV-CLL-01 study).

Manufacturing

·Continuation of high manufacturing success rate: The Gen 2 TIL therapy manufacturing process continues to be robust with a demonstrated success rate, as measured from the receipt of the starting material to the shipment of TIL product, of well over 90 percent in approximately 300 patients.
·Clinical introduction of Gen 3 TIL therapy: A proprietary 16-day, third generation TIL therapy manufacturing process (Gen 3) entered the clinic for initial evaluation in the expanded C-145-03 clinical study in head and neck cancer patients with significant unmet need, for which shorter time to TIL infusion may offer a potential benefit.
·Clinical introduction of PD-1 selected TIL therapy (LN-145-S1): A proprietary, selected TIL product LN-145-S1, aimed at taking further advantage of TIL’s ability to recognize the tumor, is also entering the clinic for initial evaluation in the expanded C-145-03 study in head and neck cancer, and subsequently in a new cohort in the IOV-COM-202 study.

Corporate

·Iovance manufacturing facility on track for commercial production in 2022: Iovance began construction of a state-of-the-art, 136,000 square foot commercial-scale production facility in Philadelphia for its TIL therapies in June 2019. The new facility is expected to be completed by year-end 2021 to support commercial supply in 2022, with capacity to meet demand for thousands of patients in multiple cancers. Iovance expects to invest approximately $85 million over three years for equipment and construction of the facility.
·The National Cancer Institute (NCI), with support from Iovance, received an award for TIL therapy technology transfer: NCI won the Federal Laboratory Consortium’s 2020 Excellence In Technology Transfer Award for "New, First-in-class Immunotherapy, for Treatment of Recurrent, Metastatic Cervical Cancer" for its collaboration with Iovance. Iovance supported NCI’s application for the award and remains a CRADA partner for ongoing research in TIL therapy.3
·New Iovance patents issued: Iovance has been granted or allowed a total of 10 U.S. patents for compositions and methods of treatment in a broad range of cancers related to its 22-day Gen 2 manufacturing process.

Research and Development

·Licensing of novel IL-2 analog (IOV-3001): Iovance obtained a license from Novartis to develop and commercialize an expectedly better IL-2 analog, an antibody cytokine engrafted protein, referred to as IOV-3001, as a targeted and selective IL-2 analog.
·Licensing of TALEN technology to develop gene edited TIL: Under a research collaboration and exclusive worldwide license agreement with Cellectis, Iovance licensed certain Cellectis TALEN technology to develop genetically edited TIL to potentially create more potent therapeutics in several cancer indications. The worldwide exclusive license enables Iovance’s use of TALEN technology addressing multiple gene targets to modify TIL for therapeutic use.

Anticipated 2020 Milestones

·Last patient dosed in the pivotal program of LN-145 for cervical cancer
·Pre-BLA Meeting with U.S. FDA
·Melanoma top-line pivotal data
·BLA submission

Fourth Quarter and Full-Year 2019 Financial Results

Net loss for the fourth quarter ended December 31, 2019, was $63.6 million, or $0.50 per share, compared to a net loss of $32.6 million, or $0.27 per share, for the fourth quarter ended December 31, 2018. Net loss for the full-year ended December 31, 2019, was $197.6 million, or $1.59 per share, compared to a net loss of $123.6 million, or $1.27 per share, for the full-year ended December 31, 2018.

Research and development expenses were $54.2 million for the fourth quarter ended December 31, 2019, an increase of $26.8 million compared to $27.4 million for the fourth quarter ended December 31, 2018. Research and development expenses were $166.0 million for the 12 months ended December 31, 2019, an increase of $66.2 million compared to $99.8 million for the prior year period. The increases in fourth quarter and full year 2019 over the prior year periods were primarily attributable to an increase in costs associated with manufacturing activities and capacity, clinical trials due to higher enrollment, and growth of the internal research and development team.

General and administrative expenses were $10.9 million for the fourth quarter 2019, an increase of $3.4 million compared to $7.5 million for the fourth quarter 2018. General and administrative expenses were $40.8 million for the 12 months ended December 31, 2019, an increase of $12.4 million compared to $28.4 million for the full year ended December 31, 2018. The increases in fourth quarter and full year 2019 over the prior year periods were primarily attributable to growth of the internal general and administrative team, as well as higher intellectual property legal costs and market research activities in preparation for commercialization.

Cash, cash equivalents, short term investments and restricted cash

At December 31, 2019, the company held $312.5 million in cash, cash equivalents, short-term investments and restricted cash compared to $468.5 million at December 31, 2018.

Webcast and Conference Call

Iovance will host a conference call today at 4:30 p.m. ET to discuss fourth quarter and full-year 2019 results and provide a corporate update. The conference call dial-in numbers are 1-844-646-4465 (domestic) or 1-615-247-0257 (international). The conference ID access number for the call is 4693108. The live webcast can be accessed in the Investors section of the company’s website at View Source The archived webcast will be available for a year in the Investors section at www.iovance.com.