On February 24, 2021 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company primarily focused on developing proprietary therapeutics designed to extend life or improve the quality of life for cancer patients, reported that Chandler D. Robinson, MD, Chief Executive Officer, is planning to present a Company overview at the following investor conferences in March (Press release, Monopar Therapeutics, FEB 24, 2021, View Source [SID1234575521]):

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H.C. Wainwright Global Life Sciences Conference
The Company’s presentation will be webcast on Tuesday, March 9, 2021 at 7:00 a.m. ET

33rd Annual Roth Conference, Healthcare
The Company’s fireside chat will be webcast on Tuesday, March 16, 2021 at 12:00 p.m. ET

Maxim’s 2021 Emerging Growth Virtual Conference
Presentation time to be determined on Wednesday, March 17, 2021

On February 24, 2021 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, reported business highlights and financial results for the fourth quarter ended December 31, 2020 (Press release, Fate Therapeutics, FEB 24, 2021, View Source [SID1234575537]).

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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

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"2020 was a pivotal year for Fate Therapeutics. We demonstrated the clinical safety and therapeutic activity of engineered iPSC-derived NK cell therapy as patients with relapsed / refractory lymphoma achieved objective responses across our FT516 and FT596 Phase 1 studies. We successfully worked with the FDA to enable clinical investigation of FT538, the first-ever CRISPR-edited, iPSC-derived cell therapy, and FT576, the first-ever cell therapy engineered with four functional anti-tumor modalities, in patients with multiple myeloma. We also made strong progress with our strategic partners, Ono Pharmaceutical and Janssen, in leveraging the unique advantages of our iPSC product platform to advance multiplexed-engineered CAR NK and CAR T-cell product candidates toward clinical development for solid tumors," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We look forward to a promising 2021 where we expect to have clinical read-outs across our programs, treat patients with the first-ever iPSC-derived CAR T-cell therapy, submit IND applications for two iPSC-derived CAR NK cell programs targeting novel antigens in solid tumors, and open our second cGMP manufacturing facility for an additional 40,000 square feet of capacity."

Clinical Programs

FT516 (hnCD16) NK Cell Product Candidate

Reported Positive Interim Clinical Data for B-cell Lymphoma. In December 2020, the Company reported positive interim data from its Phase 1 study of FT516 in combination with rituximab for patients with relapsed / refractory B-cell lymphoma (BCL) who have previously failed or progressed on CD20-targeted monoclonal antibody therapy. As of a November 16, 2020 cutoff date, three patients in the second dose cohort (90 million cells per dose) and one patient in the third dose cohort (300 million cells per dose) had each received two FT516 treatment cycles, each cycle consisting of three days of outpatient lympho-conditioning, one dose of rituximab, and three once-weekly infusions of FT516 with IL-2 cytokine support. Three of four relapsed / refractory patients achieved an objective response, including two complete responses, following the second FT516 treatment cycle. The two-cycle treatment regimen was well-tolerated, supporting the potential to safely administer up to six doses of FT516 in the outpatient setting. No dose-limiting toxicities (DLTs), no FT516-related serious adverse events (AEs), no FT516-related grade 3 or greater AEs, and no events of any grade of cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), or graft-versus-host disease (GvHD) were reported by investigators. In addition, no evidence of anti-product T- or B-cell mediated host-versus-product alloreactivity was detected.
Phase 1 Dose Escalation Ongoing at 900 Million Cells. The Phase 1 clinical trial is designed to assess the safety and determine the maximum dose of FT516 as a monotherapy for the treatment of relapsed / refractory acute myeloid leukemia (AML) and in combination with CD20-targeted monoclonal antibody therapy for the treatment of relapsed / refractory BCL (NCT04023071). Dose escalation is ongoing at 900 million cells per dose in both disease regimens.
FT596 (CAR19 + hnCD16 + IL-15RF) NK Cell Product Candidate

Presented Patient Case Study Demonstrating Clinical Activity in Refractory DLBCL. The case study, which was presented at the 62nd Annual Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2020, described a heavily pre-treated patient with diffuse large B-cell lymphoma (DLBCL) who was enrolled in the first dose cohort (30 million cells) and achieved a partial response following administration of a single dose of FT596 as monotherapy. The patient subsequently received a second, single dose of FT596, which resulted in a deepening response as evidenced by further decreases in both tumor size and metabolic activity. No DLTs, no FT596-related serious AEs, and no events of any grade of CRS, ICANS, or GvHD were reported by the investigator. The patient had previously received seven prior treatment regimens, including five rituximab-containing regimens as well as autologous stem cell transplantation, and was most recently refractory to an experimental natural killer (NK) cell therapy regimen comprised of fludarabine and cyclophosphamide lympho-conditioning followed by ex vivo expanded, donor-derived NK cells, IL-2, and rituximab.
First CLL Patient Treated. The Phase 1 clinical trial is designed to assess the safety and determine the maximum dose of FT596 as a monotherapy and in combination with CD20-targeted monoclonal antibody therapies for the treatment of relapsed / refractory BCL and chronic lymphocytic leukemia (CLL) (NCT04245722). Dose escalation for the treatment of BCL is ongoing in the second dose cohorts of 90 million cells as monotherapy and in combination with rituximab. The first patient with CLL has been treated in the first dose cohort of 30 million cells as monotherapy, and the Company plans to begin enrollment in combination with obinutuzumab upon clearance of the first monotherapy dose cohort.
First Patients Treated in Investigator-initiated Study for Relapse Prevention following HSCT. Investigators from the Masonic Cancer Center, University of Minnesota, are conducting a Phase 1 study of FT596 in combination with rituximab for the prevention of relapse in patients with BCL who have undergone autologous hematopoietic stem cell transplant (HSCT) and are considered high risk for early relapse (NCT04555811). The first patients have been treated in the first dose cohort of 90 million cells.
FT538 (hnCD16 + IL-15RF + CD38KO) NK Cell Product Candidate

First AML Patients Treated. FT538 is the first-ever CRISPR-edited cell therapy derived from a clonal master engineered induced pluripotent stem cell (iPSC) line, and is modified with three functional components to enhance innate immunity. The Phase 1 clinical trial is designed to assess three once-weekly doses of FT538 as a monotherapy for patients with relapsed / refractory AML and in combination with the CD38-targeted monoclonal antibody daratumumab for patients with relapsed / refractory multiple myeloma (NCT04614636). The first patients with AML have been treated in the first dose cohort of 100 million cells per dose.
Second IND Allowed by FDA for AML in Combination with CD38-targeted Monoclonal Antibody. In December 2020, the FDA allowed a second Investigational New Drug (IND) application for the clinical investigation of three once-weekly doses of FT538 in combination with daratumumab for the treatment of relapsed / refractory AML. The Phase 1 clinical trial is sponsored and managed by investigators from the Masonic Cancer Center, University of Minnesota. CD38 expression on leukemic blasts has been observed in a significant number of AML patients, indicating the potential of CD38 as a therapeutic target for AML.
FT576 (CAR-BCMA + hnCD16 + IL-15RF + CD38KO) NK Cell Product Candidate

IND Application Allowed by FDA for Multiple Myeloma. FT576 is an investigational, off-the-shelf, chimeric antigen receptor (CAR) NK cell cancer immunotherapy targeting B-cell maturation antigen (BCMA). FT576 is derived from a clonal master iPSC line engineered with four functional components designed to enable multi-antigen targeting of myeloma cells, augment antibody-dependent cellular cytotoxicity (ADCC), enhance cell persistence and prevent anti-CD38 monoclonal antibody-induced fratricide. In December 2020, the U.S. Food & Drug Administration (FDA) allowed the Company’s IND application for clinical investigation of FT576 in patients with relapsed / refractory multiple myeloma who have failed at least two lines of therapy. The Company is preparing to initiate a Phase 1 clinical trial to assess single-dose and multi-dose treatment regimens of FT576 as monotherapy and in combination with CD38-targeted monoclonal antibody therapy.
Preclinical Programs for Solid Tumors

CAR MICA/B Program Featured in Oral Presentation at ASH (Free ASH Whitepaper). Dr. Kai W. Wucherpfennig, Chair of Cancer Immunology and Virology and Director of the Center for Cancer Immunotherapy Research at Dana-Farber Cancer Institute (DFCI), presented preclinical data highlighting the Company’s development of FT536, a novel CAR NK cell product candidate targeting the alpha-3 domain of the pan-tumor associated stress antigens MICA and MICB. While MICA/B are selectively expressed at high levels on many solid tumors, proteolytic shedding of MICA/B is a prominent mechanism of tumor escape from NK cell-mediated destruction. Several recent publications have shown that targeting the alpha-3 domain strongly inhibits MICA/B shedding, resulting in a substantial increase in the cell surface density of MICA/B and restoration of NK cell-mediated tumor immunity. The Company plans to submit an IND application in the second half of 2021 to initiate a Phase 1 clinical trial of FT536 for the treatment of solid tumors.
CAR B7H3 Program Featured in Oral Presentation at SITC (Free SITC Whitepaper). During an oral session at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in November 2020, preclinical data from the Company’s collaboration with Dr. Jeffrey S. Miller, Professor of Medicine and Deputy Director of the Masonic Comprehensive Cancer Center, University of University of Minnesota, was presented that highlighted the specificity and activity of CAR T cells incorporating a proprietary camelid single-domain antibody fragment targeting B7H3, a pan-tumor associated antigen expressed on a wide range of cancers. The Company is currently incorporating novel CAR constructs targeting B7H3 into multiplexed engineered master iPSC lines for selection of a preclinical development candidate.
Other Corporate Highlights

Preclinical Milestone Reached under iPSC-derived CAR T-Cell Collaboration with Ono Pharmaceutical. In December 2020, the Company and Ono reviewed a preclinical data package for an iPSC-derived CAR T-cell product candidate incorporating Ono’s proprietary antigen binding domain targeting a cancer-specific antigen expressed on certain solid tumors. The Company and Ono elected to continue preclinical development of the iPSC-derived CAR T-cell product candidate under the collaboration, and the Company received a $10 million milestone fee from Ono. Ono maintains an option to develop and commercialize the iPSC-derived CAR T-cell product candidate in all territories of the world, with the Company retaining the option to co-develop and co-commercialize the product candidate in the United States and Europe under a joint arrangement with Ono whereby Fate is eligible to share at least 50% of the profits and losses.
Completed $460 Million Public Offering. In January 2021, the Company completed an underwritten public offering of 5.1 million shares of its common stock priced at $85.50 per share and, in lieu of common stock to certain investors, pre-funded warrants to purchase 0.3 million shares of its common stock priced at $85.499 per pre-funded warrant. Net proceeds to the Company were approximately $432 million.
Fourth Quarter 2020 Financial Results

Cash & Investment Position: Cash, cash equivalents and investments as of December 31, 2020 were $482.9 million. This amount does not include net proceeds to the Company of approximately $432 million from the January 2021 underwritten public offering.
Total Revenue: Revenue was $15.9 million for the fourth quarter of 2020, which was derived from the Company’s collaborations with Janssen and Ono Pharmaceutical.
R&D Expenses: Research and development expenses were $39.0 million for the fourth quarter of 2020, which includes $5.3 million of non-cash stock-based compensation expense.
G&A Expenses: General and administrative expenses were $10.3 million for the fourth quarter of 2020, which includes $3.4 million of non-cash stock-based compensation expense.
Other Expenses: Other expenses, net were $19.7 million, which includes a $20.1 million non-cash charge equal to the fair value change of certain contingent milestone payments that will be owed to Memorial Sloan Kettering Cancer Center upon the Company’s achievement of a specified clinical milestone with an iPSC-derived CAR T-cell product candidate and the subsequent appreciation of the Company’s common stock price per share.
Shares Outstanding: Common shares outstanding were 87.7 million, and preferred shares outstanding were 2.8 million, as of December 31, 2020. Each preferred share is convertible into five common shares. Common shares outstanding does not include 5.4 million common shares, including 0.3 million common shares issuable upon exercise of pre-funded warrants, that were issued in the January 2021 underwritten public offering.
Today’s Conference Call and Webcast
The Company will conduct a conference call today, Wednesday, February 24, 2021 at 5:00 p.m. ET to review financial and operating results for the quarter ended December 31, 2020. In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 6368962. The live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website beginning approximately two hours after the event.

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 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 350 issued patents and 150 pending patent applications.

About FT516
FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies. CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. ADCC is dependent on NK cells maintaining stable and effective expression of CD16, which has been shown to undergo considerable down-regulation in cancer patients. In addition, CD16 occurs in two variants, 158V or 158F, that elicit high or low binding affinity, respectively, to the Fc domain of IgG1 antibodies. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. FT516 is being investigated in an open-label, multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-targeted monoclonal antibodies for the treatment of advanced B-cell lymphoma (NCT04023071). Additionally, FT516 is being investigated in an open-label, multi-dose Phase 1 clinical trial in combination with avelumab for the treatment of advanced solid tumor resistant to anti-PDL1 checkpoint inhibitor therapy (NCT04551885).

About FT596
FT596 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary chimeric antigen receptor (CAR) optimized for NK cell biology that targets B-cell antigen CD19; a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; and an IL-15 receptor fusion (IL-15RF) that augments NK cell activity. In preclinical studies of FT596, the Company has demonstrated that dual activation of the CAR19 and hnCD16 targeting receptors enhances cytotoxic activity, indicating that multi-antigen engagement may elicit a deeper and more durable response. Additionally, in a humanized mouse model of lymphoma, FT596 in combination with the anti-CD20 monoclonal antibody rituximab showed enhanced killing of tumor cells in vivo as compared to rituximab alone. FT596 is being investigated in an open-label, multi-center Phase 1 clinical trial for the treatment of relapsed / refractory B-cell lymphoma as a monotherapy and in combination with rituximab, and for the treatment of relapsed / refractory chronic lymphocytic leukemia (CLL) as a monotherapy and in combination with obinutuzumab (NCT04245722).

About FT538
FT538 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three functional components: a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; an IL-15 receptor fusion (IL-15RF) that augments NK cell activity; and the deletion of the CD38 gene (CD38KO), which promotes persistence and function in high oxidative stress environments. FT538 is designed to enhance innate immunity in cancer patients, where endogenous NK cells are typically diminished in both number and function due to prior treatment regimens and tumor suppressive mechanisms. In preclinical studies, FT538 has shown superior NK cell effector function, as compared to peripheral blood NK cells, with the potential to confer significant anti-tumor activity to patients through multiple mechanisms of action. FT538 is being investigated in an open-label, multi-dose Phase 1 clinical trial for the treatment of acute myeloid leukemia (AML) and in combination with daratumumab, a CD38-targeted monoclonal antibody therapy, for the treatment of multiple myeloma (NCT04614636).

On February 24, 2021 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 2020 financial results and provided a corporate update (Press release, Kura Oncology, FEB 24, 2021, View Source [SID1234575553]).

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"Last year was a transformative one for Kura, and our team continues to make tremendous progress advancing our pipeline of anti-cancer therapeutics," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "In December, we reported encouraging first-in-human data for our menin inhibitor KO-539 in an all-comers population of patients with acute myeloid leukemia (AML). Now, we look forward to obtaining a larger clinical dataset as we move into genetically enriched Phase 1 expansion cohorts comprising NPM1-mutant and KMT2A-rearranged relapsed/refractory AML patients. KO-539 continues to demonstrate a clean safety and tolerability profile, compelling clinical activity and a wide therapeutic window, supporting a potentially best-in-class profile both as a monotherapy and in combination."

"Meanwhile, we were very pleased to receive Breakthrough Therapy Designation from the FDA for tipifarnib," continued Dr. Wilson. "We believe this designation acknowledges both the dire unmet need for patients with recurrent or metastatic HRAS mutant HNSCC and the promise of tipifarnib to provide clinical benefit to patients. Breakthrough Therapy Designation is the latest milestone in our effort to pioneer the use of farnesyl transferase inhibitors to treat patients with cancer. We are also developing a next-generation farnesyl transferase inhibitor, which we intend to direct at innovative biology and larger oncology indications through rational combinations. We have identified multiple advanced lead compounds with superior properties, and we expect to nominate a development candidate for IND-enabling studies in mid-2021. Finally, thanks to a successful public offering in December, we are in a stronger financial position than ever before, and we believe this provides us with sufficient resources to advance our pipeline programs through multiple value-inflection points."

Recent Highlights

First clinical data for menin inhibitor KO-539 presented at ASH (Free ASH Whitepaper) – In December, Kura reported preliminary clinical data from a Phase 1/2 KOMET-001 clinical trial of KO-539 at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. These data were highlighted by single-agent activity in an all-comer population of patients with relapsed or refractory AML, including patients with NPM1 mutations and KMT2A rearrangements. KO-539 also demonstrated a favorable safety and tolerability profile, with no drug discontinuations due to treatment-related adverse events and no evidence of QTc prolongation.

KOMET-001 protocol amendment to include Phase 1 expansion cohorts – Kura is currently evaluating KO-539 in an 800 mg dose cohort in Phase 1 dose escalation, and KO-539 continues to demonstrate compelling clinical activity, encouraging safety and tolerability and a wide therapeutic window. Based on recent feedback from the FDA regarding the registration-enabling design for the KOMET-001 study, the Company is amending the trial protocol to include two Phase 1 expansion cohorts while continuing to evaluate KO-539 in dose escalation. Kura plans to enrich these Phase 1 expansion cohorts with NPM1-mutant and KMT2A-rearranged relapsed/refractory AML patients at doses that have already met the safety threshold to help determine a minimum safe and biologically effective dose. This will enable the Company to further characterize the efficacy of KO-539 in these target populations and better inform a recommended Phase 2 dose. Initiation of the genetically enriched Phase 1 expansion cohorts is expected in mid-2021.

Tipifarnib receives Breakthrough Therapy Designation from FDA – Earlier today, Kura announced that tipifarnib has been granted Breakthrough Therapy Designation by the FDA for the treatment of patients with recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (HNSCC) with variant allele frequency ≥ 20% after disease progression on platinum-based chemotherapy. The Breakthrough Therapy Designation is based upon data from the Company’s Phase 2 clinical trial (RUN-HN), which was recently accepted for publication in an upcoming issue of the Journal of Clinical Oncology. Tipifarnib is currently being evaluated in an ongoing registration-directed clinical trial (AIM-HN) in this indication of high unmet need.

Diagnostic development collaboration with Illumina – Kura recently entered into a strategic collaboration with Illumina to develop a diagnostic in support of Kura’s tipifarnib program. The partnership with Illumina is focused on the development of a next-generation sequencing-based companion diagnostic leveraging the content of TruSight Oncology 500 to detect HRAS mutations in HNSCC.

Initiation of Phase 1/2 study of tipifarnib plus PI3Kα inhibitor in 2H 2021 – In October, Kura reported preclinical data showing compelling activity of tipifarnib when combined with a PI3Kα inhibitor in models of HRAS-dependent and/or PI3K dependent HNSCC. The data, presented at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics, support the Company’s upcoming Phase 1/2 proof-of-concept study of tipifarnib in combination with a PI3Kα inhibitor in advanced or unresectable relapsed/refractory HNSCC harboring PIK3CA mutations or amplifications and/or HRAS overexpression. Kura believes that the total addressable population for tipifarnib may be as high as 50% of HNSCC.
Financial Results

Research and development expenses for the fourth quarter of 2020 were $17.5 million, compared to $13.5 million for the fourth quarter of 2019. Research and development expenses for the full year 2020 were $60.4 million, compared to $47.8 million for the prior year.

General and administrative expenses for the fourth quarter of 2020 were $8.8 million, compared to $5.5 million for the fourth quarter of 2019. General and administrative expenses for the full year 2020 were $31.5 million, compared to $19.7 million for the prior year.

Net loss for the fourth quarter of 2020 was $26.2 million, compared to a net loss of $17.9 million for the fourth quarter of 2019. Net loss for the full year 2020 was $89.6 million, compared to a net loss of $63.1 million for the prior year. Net loss for the fourth quarter and full year of 2020 included non-cash share-based compensation expense of $3.7 million and $12.8 million, respectively, compared to $2.4 million and $9.4 million for the same periods in 2019, respectively.

Cash, cash equivalents and short-term investments totaled $633.3 million as of December 31, 2020, compared with $236.9 million as of December 31, 2019. This includes net proceeds of approximately $324.1 million from a public offering completed in December 2020. Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2024.
Upcoming Milestones

Initiation of genetically enriched Phase 1 expansion cohorts in KOMET-001 in mid-2021

Additional Phase 1 data from KOMET-001 in the second half of 2021

Initiation of a Phase 1/2 proof-of-concept study of tipifarnib in combination with a PI3Kα inhibitor in the second half of 2021

Nomination of a next-generation farnesyl transferase inhibitor Development Candidate in mid-2021
Conference Call and Webcast

Kura’s management will host a webcast and conference call at 8:00 a.m. ET / 5:00 a.m. PT today, February 24, 2021, to discuss the financial results for the fourth quarter and full year 2020 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: 8581798. 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.

On February 24, 2021 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health" or the "Company" or "we") reported its fourth-quarter and full-year 2020 financial results (Press release, Bausch Health, FEB 24, 2021, View Source [SID1234575572]).

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"Despite unprecedented business challenges resulting from the COVID-19 pandemic, I’m proud that Bausch Health finished the year strong and outperformed the high end of our latest revenue guidance range," said Joseph C. Papa, chairman and CEO, Bausch Health. "During the pandemic-related downturn, we focused our efforts on growing market share for key promoted products, carefully managed our expenses and continued to invest in our pipeline for future growth opportunities. We generated cash from operations of more than $1.1 billion, which helped us to repay approximately $900 million of our debt."

"We are continuing to execute on our business recovery from the pandemic, and we are well positioned to benefit from recovery-related tailwinds and capitalize on our key growth drivers and catalysts in 2021 as we remain focused on how best to unlock value in the Company, including the planned spinoff of Bausch + Lomb," continued Mr. Papa.

Executing on Core Businesses and Advancing Pipeline

The Bausch + Lomb/International segment comprised approximately 56% of the Company’s reported revenue in the fourth quarter of 2020
Reported revenue in the Bausch + Lomb/International segment grew nominally compared to the fourth quarter of 2019; organic1,2 revenue in this segment was flat compared to the fourth quarter of 2019
The Bausch + Lomb/International segment comprised approximately 55% of the Company’s reported revenue in 2020
Reported revenue in the Bausch + Lomb/International segment decreased 7% compared to 2019; revenue in this segment declined organically1,2 by 6% compared to 2019
Launched several products in 2020, including:
Bausch + Lomb INFUSE silicone hydrogel (SiHy) daily disposable contact lenses in the United States
BAUSCH + LOMB ULTRA ONE Day SiHy daily disposable contact lenses in Australia, Canada and Hong Kong
SimplifEYE intraocular lens (IOL) delivery system in the United States
Expanded parameters for Biotrue ONEday for Astigmatism daily disposable contact lenses
LuxSmart, the Company’s first Extended Depth of Focus IOL, and LuxGood, a monofocal IOL, in Europe
BAUSCH + LOMB ULTRA monthly silicone hydrogel contact lenses in China in November
Received approval from the U.S. Food and Drug Administration (FDA) for Alaway Preservative Free (ketotifen fumarate) ophthalmic solution, 0.035%, antihistamine eye drops, which launched in February 2021
Entered into multiple licensing and business development agreements, including:
An agreement to acquire an option to purchase all ophthalmology assets of Allegro Ophthalmics, LLC, including global rights for risuteganib (Luminate)3
An exclusive license from Eyenovia, Inc. in the United States and Canada for the development and commercialization of an investigational microdose formulation of atropine ophthalmic solution, which is being investigated for the reduction of pediatric myopia progression in children ages 3-12
An exclusive global license from BHVI for a myopia control contact lens design
Completed enrollment in early 2021 for the first Phase 3 study evaluating NOV034 as a first-in-class investigational drug with a novel mechanism of action to treat the signs and symptoms of dry eye disease associated with Meibomian gland dysfunction, after initiating a second, identical Phase 3 study in November 2020
The Salix segment comprised approximately 24% of the Company’s reported revenue in the fourth quarter of 2020
Reported and organic1,2 revenue in the Salix segment increased by 2% compared to the fourth quarter of 2019
The Salix segment comprised approximately 24% of the Company’s reported revenue in 2020
Reported and organic1,2 revenue in the Salix segment decreased by 6% compared to 2019
Received FDA Orphan Drug Designation for rifaximin for the treatment of sickle cell disease
Announced topline results from a Phase 2 study evaluating an investigative soluble solid dispersion (SSD) formulation of immediate release (IR) rifaximin in combination with the current standard of care therapy for the treatment of overt hepatic encephalopathy. In the study, 40 mg BID of rifaximin SSD IR plus standard of care therapy met the study’s primary endpoint with statistically significantly superior results compared to placebo plus standard of care therapy
The Ortho Dermatologics segment comprised approximately 7% of the Company’s reported revenue in the fourth quarter of 2020
Reported revenue in the Ortho Dermatologics segment increased by 1% compared to the fourth quarter of 2019; revenue in this segment declined organically1,2 by 1% compared to the fourth quarter of 2019
The Ortho Dermatologics segment comprised approximately 7% of the Company’s reported revenue in 2020
Reported revenue in the Ortho Dermatologics segment decreased by 2% compared to 2019; revenue in this segment declined organically1,2 by 3% compared to 2019
Launched ARAZLO (tazarotene) Lotion, 0.045%, in the United States
Received an expanded indication in the United States for JUBLIA (efinaconazole) topical solution, 10%, to treat patients as young as six years old
Since announcing its intention to separate Bausch Health’s eye health business into an independent public company, the Company has continued to make progress toward internal objectives necessary for the spin of Bausch + Lomb, and these internal objectives are anticipated to be achieved by the end of the third quarter of 2021
Released both Bausch Foundation Inaugural Activity Report and the Company’s annual Corporate Social Responsibility report in September 2020
Response to COVID-19 Pandemic
When the COVID-19 pandemic emerged, Bausch Health acted quickly to implement business continuity plans that enabled the Company to ensure the health and well-being of its employees, maintain an uninterrupted availability of its health care products and remain focused on supporting customers and patients around the world. Additionally, Bausch Health donated health care products and supplies, ranging from contact lenses to antiviral medicines, through the Company and the Bausch Foundation.

The Company also continued to advance its relief efforts related to the pandemic by researching its existing medicines to determine if any of its products may offer valuable treatment options. Examples include:

DEXAVEN (dexamethasone phosphate), which received an additional new indication in Poland for the treatment of COVID-19 in adult and adolescent patients (12 years of age and older weighing at least 40 kg) who require oxygen therapy
LUMIFY (brimonidine tartrate ophthalmic solution 0.025%), BESIVANCE (besifloxacin ophthalmic suspension) 0.6% and Opcon-A (pheniramine maleate 0.315% and naphazoline HCI 0.02675% ophthalmic solution) eye drops preserved with benzalkonium chloride, for which investigational in vitro data indicated complete inactivation of COVID-19
VIRAZOLE (Ribavirin for Inhalation Solution, USP), which is being studied in an investigational clinical trial in Canada, Greece, Mexico and Brazil to evaluate its use in combination with standard of care therapy to treat hospitalized adult patients with respiratory distress due to COVID-19
IVEXTERM (ivermectin), which is being studied in Latin America to assess an investigational use in treating patients with mild COVID-19; topline results are expected in the first half of 2021
Debt Management

Repaid debt by approximately $480 million in the fourth quarter of 2020 for a total of approximately $900 million in the full year of 2020 using cash generated from operations and more efficient cash management
Refinanced $3.500 billion of debt in 2020 to extend maturities and provide flexibility
Bausch Health has no debt maturities or mandatory amortization payments until 2024
Resolving Legal Matters
The Company resolved multiple legal matters in 2020, including:

Resolving outstanding intellectual property disputes with Sandoz Inc. regarding XIFAXAN (rifaximin) 550 mg tablets and with Sun Pharmaceutical Industries Ltd. regarding XIFAXAN 200 mg and 550 mg tablets. Salix will maintain market exclusivity for XIFAXAN until 20285
Resolving the legacy investigation by the U.S. Securities and Exchange Commission for $45 million regarding the Company’s former relationship with Philidor Rx Services, LLC and certain accounting practices and disclosures related to the 2014 and 2015 reporting periods. The Company neither denied nor admitted the charges
Resolving the Canadian securities class action litigation for $94 million CAD (approximately $71 million USD), plus settlement administration costs. The Company admits no liability and denies all allegations of wrongdoing whatsoever
Fourth-Quarter and Full-Year 2020 Revenue Performance
Total reported revenues were $2.213 billion for the fourth quarter of 2020, as compared to $2.224 billion in the fourth quarter of 2019, a decrease of $11 million.

Total reported revenues were $8.027 billion for the full year of 2020, as compared to $8.601 billion for the full year of 2019, a decrease of $574 million, or 7%. Revenue was negatively impacted by approximately $740 million in 2020 due to the impact of the COVID-19 pandemic. Excluding the unfavorable impact of foreign exchange of $39 million, the impact of divestitures and discontinuations of $20 million and the impact of an acquisition of $13 million, revenue declined 6% organically1,2 compared to the full year of 2019.

Bausch + Lomb/International Segment
Bausch + Lomb/International segment revenues were $1.242 billion for the fourth quarter of 2020, as compared to $1.238 billion for the fourth quarter of 2019, an increase of $4 million. Excluding the favorable impact of foreign exchange of $9 million and the impact of divestitures and discontinuations of $5 million, the Bausch + Lomb/International segment was flat organically1,2 compared to the fourth quarter of 2019 primarily due to the impact of the COVID-19 pandemic.

Bausch + Lomb/International segment revenues were $4.408 billion for the full year of 2020, as compared to $4.739 billion for the full year of 2019, a decrease of $331 million, or 7%. Excluding the unfavorable impact of foreign exchange of $42 million and the impact of divestitures and discontinuations of $19 million, the Bausch + Lomb/International segment declined organically1,2 by 6% compared to the full year of 2019 primarily due to the impact of the COVID-19 pandemic.

Salix Segment
Salix segment reported and organic1,2 revenues were $527 million for the fourth quarter of 2020, as compared to $517 million for the fourth quarter of 2019, an increase of $10 million, or 2%, primarily driven by increased sales of XIFAXAN and TRULANCE (plecanatide), for which sales grew by 4% and by 33%, respectively, compared to the fourth quarter of 2019. The increase was partially offset by the loss of exclusivity of products in the segment, which negatively impacted revenues by approximately $6 million, and an expected decline for GLUMETZA (metformin hydrochloride), for which revenue declined by $7 million due to reduced net selling prices.

Salix segment revenues were $1.904 billion for the full year of 2020, as compared to $2.022 billion for the full year of 2019, a decrease of $118 million, or 6%. Excluding the impact of an acquisition of $13 million, the Salix segment also declined organically1,2 by 6% compared to the full year of 2019. The decrease in revenue was primarily driven by the loss of exclusivity of products in the segment, which negatively impacted revenues by approximately $109 million; by an expected decline for GLUMETZA, for which revenue declined by $70 million due to reduced net selling prices; and by the impact of the COVID-19 pandemic. The decrease in sales for the full year of 2020 was partially offset by increased sales of XIFAXAN and TRULANCE, for which sales grew by 2% and 49%, respectively, compared to the full year of 2019.

Ortho Dermatologics Segment
Ortho Dermatologics segment revenues were $160 million for the fourth quarter of 2020, as compared to $158 million for the fourth quarter of 2019, an increase of $2 million, or 1%. Excluding the favorable impact of foreign exchange of $3 million, the Ortho Dermatologics segment declined organically1,2 by approximately 1% compared to the fourth quarter of 2019 primarily driven by the loss of exclusivity of products in the segment, which negatively impacted revenues by approximately $9 million, partially offset by sales of the Thermage franchise, which grew by 46% compared to the fourth quarter of 2019.

Ortho Dermatologics segment revenues were $553 million for the full year of 2020, as compared to $565 million for the full year of 2019, a decrease of $12 million, or 2%. Excluding the favorable impact of foreign exchange of $3 million, the Ortho Dermatologics segment declined organically1,2 by approximately 3% compared to the full year of 2019 primarily driven by the loss of exclusivity of products in the segment, which negatively impacted revenues by approximately $37 million, partially offset by sales of the Thermage franchise, which grew by 47% compared to the full year of 2019.

Diversified Products Segment
Diversified Products segment reported and organic1,2 revenues were $284 million for the fourth quarter of 2020, as compared to $311 million for the fourth quarter of 2019, a decrease of $27 million, or 9%. The decrease was primarily attributable to the previously reported loss of exclusivity for a basket of products and the impact of the COVID-19 pandemic.

Diversified Products segment revenues were $1.162 million for the full year of 2020, as compared to $1.275 billion for the full year of 2019, a decrease of $113 million, or 9%. Excluding the impact of divestitures and discontinuations of $1 million, the Diversified Products segment also declined organically1,2 by 9% compared to the full year of 2019. The decrease in revenue was primarily attributable to the previously reported loss of exclusivity for a basket of products and the impact of the COVID-19 pandemic.

Operating Results
Operating loss was $5 million for the fourth quarter of 2020, as compared to operating loss of $1.076 billion for the fourth quarter of 2019, an increase in operating results of $1.071 billion. The increase in operating results was primarily due to the accrual of legal reserves established for the resolution of the U.S. securities litigation, other related actions and ongoing legacy litigation and investigations in the fourth quarter of 2019 and profit protection measures taken to manage and reduce operating expenses during the COVID-19 pandemic, partially offset by decreases in revenues and gross margins primarily due to the impact of the COVID-19 pandemic, as discussed above.

Operating income was $676 million for the full year of 2020, as compared to operating loss of $203 million for the full year of 2019, an increase in operating results of $879 million. The increase in operating results was primarily due to the accrual of legal reserves established for the resolution of the U.S. securities litigation, other related actions and ongoing legacy litigation and investigations in 2019 and profit protection measures taken to manage and reduce operating expenses during the COVID-19 pandemic, partially offset by decreases in revenues and gross margins primarily due to the impact of the COVID-19 pandemic, as discussed above.

Net Loss
Net loss was $153 million for the fourth quarter of 2020, as compared to net loss of $1.516 billion for the fourth quarter of 2019, a favorable change of $1.363 billion. The change was primarily driven by the increase in operating results discussed above and the benefit from income taxes in connection with the release of valuation allowance against deferred income taxes.

Net loss was $560 million for the full year of 2020, as compared to net loss of $1.788 billion for the full year of 2019, a favorable change of $1.228 billion. The change was primarily driven by the increase in operating results discussed above, the benefit from income taxes in connection with the release of valuation allowance against deferred income taxes and the decrease in interest expense.

Adjusted net income (non-GAAP)1 for the fourth quarter of 2020 was $478 million, as compared to $404 million for the fourth quarter of 2019, an increase of $74 million, or 18%.

Adjusted net income (non-GAAP)1 for the full year of 2020 was $1.428 billion, as compared to $1.559 billion for the full year of 2019, a decrease of $131 million, or 8%.

Cash Generated from Operations
The Company generated $394 million of cash from operations (GAAP basis) in the fourth quarter of 2020, as compared to $234 million in the fourth quarter of 2019, an increase of $160 million. The increase in cash from operations was primarily attributed to the timing of payments and receipts in the ordinary course of business and profit protection measures taken to manage and reduce operating expenses during the COVID-19 pandemic, partially offset by payments of $79 million for the resolution of certain legacy litigation and investigations in the fourth quarter of 2020.

The Company generated $1.111 billion of cash from operations (GAAP basis) in 2020, as compared to $1.501 billion in 2019, a decrease of $390 million. The decrease in cash from operations was primarily attributed to lower volumes as a result of the COVID-19 pandemic and $122 million of payments for the resolution of certain legacy litigation and investigations in 2020.7

EPS
GAAP Earnings Per Share (EPS) Diluted for the fourth quarter of 2020 was ($0.43), as compared to ($4.30) for the fourth quarter of 2019. GAAP Earnings Per Share (EPS) Diluted for the full year of 2020 was ($1.58), as compared to ($5.08) for the full year of 2019.

Adjusted EBITDA (non-GAAP)1
Adjusted EBITDA (non-GAAP)1 was $911 million for the fourth quarter of 2020, as compared to $898 million for the fourth quarter of 2019, an increase of $13 million, or 1%.

Adjusted EBITDA (non-GAAP)1 was $3.294 billion for the full year of 2020, as compared to $3.571 billion for the full year of 2019, a decrease of $277 million, or 8%. The decrease was primarily due to impact of the COVID-19 pandemic.

2021 Financial Outlook
Bausch Health provided guidance for the full year of 2021 as follows:

Full-year revenue range of $8.60 – $8.80 billion
Full-year Adjusted EBITDA (non-GAAP) range of $3.40 – $3.55 billion
Other than with respect to GAAP Revenues, the Company only provides guidance on a non-GAAP basis. The Company does not provide a reconciliation of forward-looking Adjusted EBITDA (non-GAAP) to GAAP net income (loss), due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation. In periods where significant acquisitions or divestitures are not expected, the Company believes it might have a basis for forecasting the GAAP equivalent for certain costs, such as amortization, which would otherwise be treated as non-GAAP to calculate projected GAAP net income (loss). However, because other deductions (such as restructuring, gain or loss on extinguishment of debt and litigation and other matters) used to calculate projected net income (loss) vary dramatically based on actual events, the Company is not able to forecast on a GAAP basis with reasonable certainty all deductions needed in order to provide a GAAP calculation of projected net income (loss) at this time. The amount of these deductions may be material and, therefore, could result in projected GAAP net income (loss) being materially less than projected Adjusted EBITDA (non-GAAP). These statements represent forward-looking information and may represent a financial outlook, and actual results may vary. Please see the risks and assumptions referred to in the Forward-looking Statements section of this news release.

Additional Highlights

Bausch Health’s cash, cash equivalents and restricted cash were $1.816 billion8 at Dec. 31, 2020
The Company’s availability under its Revolving Credit Facility was $1.121 billion at Dec. 31, 2020
Basic weighted average shares outstanding for the fourth quarter of 2020 were 355.8 million shares. Diluted weighted average shares outstanding for the fourth quarter of 2020 were 359.0 million shares9
Basic weighted average shares outstanding for the full year of 2020 were 355.0 million shares. Diluted weighted average shares outstanding for the full year of 2020 were 358.2 million shares9

On February 24, 2021, ASLAN Pharmaceuticals Limited (the "Company") reported that entered into a Securities Purchase Agreement (the "Purchase Agreement") with the purchasers named therein (the "Purchasers"), pursuant to which the Company agreed to sell to the Purchasers, in an unregistered offering, an aggregate of 25,568,180 Ordinary Shares, nominal value $0.01 per share ("Ordinary Shares") at a purchase price of $0.704 per share (the "Purchase Price"), which represents the closing sale price of the Company’s American Depositary Shares ("ADSs") on the Nasdaq Global Market on February 24, 2021 of $3.52 per ADS divided by five (the "Private Placement") (Press release, ASLAN Pharmaceuticals, FEB 24, 2021, View Source [SID1234575595]). Each ADS represents five Ordinary Shares. The Private Placement is expected to close on or about February 25, 2021 (the "Closing"), subject to customary closing conditions.

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The Private Placement is expected to result in gross proceeds to the Company of approximately $18 million before deducting offering expenses.

The Company has agreed to file a registration statement with the U.S. Securities and Exchange Commission within 30 days after the Closing to register the Ordinary Shares, which will be represented by ADSs (collectively, the "Securities").

The Company has also agreed, among other things, to indemnify the Purchasers, their partners, members, officers and directors, and each person who controls such Purchasers, from certain liabilities and to pay certain expenses incurred by the Company in connection with the registration of the Securities.

The Private Placement is exempt from registration pursuant to Section 4(a)(2) of the Securities Act of 1933, as amended as a transaction by an issuer not involving a public offering. The Purchasers have agreed to acquire the Ordinary Shares for investment only and not with a view to or for sale in connection with any distribution thereof.