On February 23, 2021, Odonate Therapeutics, Inc. (the "Company") reported that entered into an Open Market Sale AgreementSM (the "Sale Agreement") with Jefferies LLC (the "Agent"), pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering price of up to $100,000,000, from time to time, in "at the market" offerings through the Agent (Filing, 8-K, Odonate Therapeutics, FEB 23, 2021, View Source [SID1234575447]). Sales of the shares of common stock, if any, will be made at prevailing market prices at the time of sale, or as otherwise agreed with the Agent. The Agent will receive a commission from the Company of 3.0% of the gross proceeds of any shares of common stock sold under the Sale Agreement.

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The Company is not obligated to sell, and the Agent is not obligated to buy or sell, any shares of common stock under the Sale Agreement. No assurance can be given that the Company will sell any shares of common stock under the Sale Agreement, or, if it does, as to the price or amount of shares of common stock that it sells or the dates when such sales will take place.

In the Sale Agreement, the Company agreed to indemnify the Agent against certain liabilities, including under the Securities Act of 1933, as amended, or to contribute payments that the Agent may be required to make because of such liabilities.

The shares of common stock sold pursuant to the Sale Agreement will be offered pursuant to a shelf registration statement on Form S-3 (File No. 333‑233990), which became effective on October 18, 2019. The Company filed a prospectus supplement with the U.S. Securities and Exchange Commission on February 23, 2021 in connection with the offer and sale of shares of the Company’s common stock pursuant to the Sale Agreement.

A copy of the Sale Agreement is attached as Exhibit 1.1 hereto and is incorporated herein by reference. The foregoing description of the Sale Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Sale Agreement.

On February 23, 2021 Leidos (NYSE:LDOS), a FORTUNE 500 science and technology leader, reported it has been named to Ethisphere Institute’s annual list of the World’s Most Ethical Companies (Press release, Leidos, FEB 23, 2021, View Source [SID1234575446]). Ethisphere, a global leader in defining and advancing the standards of ethical business practices recognized Leidos for the fourth consecutive year. Ethisphere recognized 135 honorees spanning 22 countries and 47 industries.

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"Our commitment to maintaining the highest ethical standards is core to our culture at Leidos," said Leidos Chairman and Chief Executive Officer Roger Krone. "Our 39,000 employees across the world embody this value as we strive to deliver a safer, healthier, and more efficient world. We are proud to receive this recognition, and will work to maintain it for years to come."

"Continuing to make the World’s Most Ethical Companies list year after year is a significant achievement, and is a testament to our employees’ pledge to do what is right every day," said Leidos Chief Ethics and Compliance Officer Michele Brown. "Our commitment to ethics and integrity guides every decision and reinforces the value we bring to our customers."

The World’s Most Ethical Companies assessment is based on Ethisphere’s Ethics Quotient framework. It includes more than 200 questions on culture, environmental and social practices, ethics and compliance activities, governance, diversity and other initiatives to support a strong value chain. The process captures and codifies the leading practices of organizations across industries and around the globe.

"While addressing the tough challenges of 2020, we saw companies lead – above all other institutions – on earning the trust of stakeholders through resilience and a commitment to ethics and integrity," said Ethisphere CEO, Timothy Erblich. "As one of the World’s Most Ethical Companies honorees, Leidos continues to demonstrate an unwavering commitment to the highest values and positively impacting the communities it serves."

On February 23, 2021 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics, reported fourth quarter and full year 2020 financial results and provided a business update (Press release, Rubius Therapeutics, FEB 23, 2021, View Source [SID1234575445]).

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"Last year was one of strong execution for Rubius Therapeutics as we advanced our clinical trials in oncology and strengthened our in-house manufacturing capabilities," said Pablo J. Cagnoni, M.D., president and chief executive officer of Rubius Therapeutics. "2021 is set to be an important year in which we plan to further advance our programs and report the clinical results from the Phase 1 trial of RTX-240 in advanced solid tumors early in the year. We are encouraged by the initial key clinical takeaways we presented in January, showing the ability of RTX-240 to stimulate innate and adaptive immune responses in advanced solid tumors. We are continuing to enroll patients in our trials for RTX-240 in relapsed/refractory acute myeloid leukemia and solid tumors and screen patients for the RTX-321 trial in advanced HPV 16-positive cancers."

Fourth Quarter and Full Year Highlights

RTX-240 Phase 1/2 Clinical Program for Advanced Solid Tumors or Relapsed/Refractory Acute Myeloid Leukemia (AML)

Escalated the dose and continue to enroll patients in the relapsed/refractory AML arm of the Phase 1 RTX-240 clinical trial
At the J.P. Morgan Healthcare Conference in January 2021, the Company presented key takeaways from 5 cohorts (n=14) of the Phase 1/2 RTX-240 solid tumor clinical trial, showing activation and expansion of both NK and T cells, indicating the ability of RTX-240 to stimulate innate and adaptive immune responses. The key takeaways from the initial data were:
◦ No treatment-related Grade 3 or Grade 4 adverse events and no dose limiting toxicities observed (n=14)
◦ All patients showed activation of NK or T cells or both cell types (n=14)
◦ In the majority of patients (n=8), all of the following were observed across dose levels:
• Activation of NK cells, activation of T cells, expansion of NK cells and expansion of T cells
As more patients are enrolled and data mature, the Company expects to disclose additional clinical results in early 2021 and plans to present the data at a medical conference
By showing that RTX-240 stimulates innate and adaptive immune responses, Rubius Therapeutics believes that the full data set from the Phase 1 clinical trial will validate its RED PLATFORM and potentially de-risk clinical development of its oncology pipeline, in particular for RTX-321. RTX-321 has an encouraging preclinical data package and expresses combinations of agonists on the cell surface, similar to RTX-240.

RTX-321 Artificial Antigen-Presenting Cell (aAPC) Development Program for Human Papillomavirus (HPV) 16-Positive Cancers

Phase 1 clinical trial of RTX-321 is screening for HLA-A*02:01-positive patients with advanced HPV 16-positive cancers, including cervical cancer, head and neck cancer and anal cancer
Achievements from Rubius Fully Owned Manufacturing Site

Continue to provide consistent cGMP supply for the two Phase 1 arms in the ongoing RTX-240 trial and Phase 1 RTX-321 clinical trial
Achieved increases in productivity and liquid in-vial shelf life for RTX-240
Continously met red blood cell identity and target product profile criteria for clinical supply for RTX-240
Introduced frozen drug substance for the first time as part of the IND application for RTX-321, resulting in a truly off-the-shelf cellular therapy with a potential shelf life of up to several years
Preclinical Data 2020 Summary
The Company presented preclinical oncology data for RTX-240 and RTX-321 at the following conferences:

Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting;
Federation of Clinical Immunology Societies (FOCIS) Virtual Annual Meeting;
American Association for Cancer Research (AACR) (Free AACR Whitepaper) Tumor Immunology Conference; and
American Society of Gene & Cell Therapy 23rd Annual Meeting
Anticipated 2021 Catalysts and Operational Objectives

Present additional clinical data from the Phase 1 RTX-240 solid tumor trial in early 2021
Continue to enroll patients in the second Phase 1 arm of the RTX-240 clinical trial in patients with relapsed/refractory AML
Dose the first patient in the RTX-321 clinical trial in HPV 16-positive tumors
Continue to produce cGMP material for the RTX-240 and RTX-321 clinical trials
Present an integrated clinical program for RTX-240, including plans for expansion cohorts, and plans for the Company’s oncology pipeline
About RTX-240
RTX-240, Rubius Therapeutics’ lead oncology program, is an allogeneic, off-the-shelf cellular therapy product candidate that is engineered to simultaneously present hundreds of thousands of copies of the costimulatory molecule 4-1BB ligand (4-1BBL) and IL-15TP (trans-presentation of IL-15 on IL-15Rα) in their native forms. RTX-240 is designed to broadly stimulate the immune system by activating and expanding both NK and memory T cells to generate a potent anti-tumor response.

About RTX-321
RTX-321, the Company’s second oncology program, is an allogeneic, off-the-shelf aAPC therapy product candidate that is engineered to induce a tumor-specific immune response by expanding antigen-specific T cells. RTX-321 expresses hundreds of thousands of copies of an HPV peptide antigen bound to major histocompatibility complex class I proteins, the costimulatory molecule 4-1BBL and the cytokine IL-12 on the cell surface to mimic human T cell-APC interactions.

Fourth Quarter 2020 Financial Results
Net loss for the fourth quarter of 2020 was $40.5 million or $0.50 per common share, compared to $44.5 million or $0.56 per common share in the fourth quarter of 2019.

In the fourth quarter of 2020, Rubius invested $25.6 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $30.5 million in the fourth quarter of 2019. This year-over-year decrease was driven primarily by a $6.2 million reduction in costs following the deprioritization of our rare disease pipeline in March 2020. The decrease in rare disease program expenses was offset by $8.4 million of incremental costs to advance our lead cancer programs, including RTX-240 and RTX-321, which were principally related to costs incurred for our Phase 1/2 clinical trial of RTX-240 for the treatment of solid tumors, including clinical CRO and internal manufacturing costs, as well as to costs incurred for IND-enabling activities and clinical startup costs for RTX-321. Additionally, R&D expenses not allocated to programs were reduced by $7.1 million driven primarily by a shift in manufacturing activities towards the technical development and production of clinical supply for our oncology programs and the shift in discovery activities from the development of clinical candidates towards work to enable more advanced programs.

G&A expenses were $14.1 million during the fourth quarter of 2020, as compared to $14.9 million for the fourth quarter of 2019. The lower costs were principally driven by a reduction in stock-based compensation expense.

Full Year 2020 Financial Results
Net loss for the full year 2020 was $167.7 million or $2.08 per common share, compared to $163.5 million or $2.08 per common share for the full year 2019.

For the full year 2020, Rubius invested $116.1 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $112.4 million for the full year 2019. The year-over-year increase was driven by $36.6 million of incremental costs to advance our lead cancer programs, including RTX-240 and RTX-321, which were principally related to costs incurred for our Phase 1/2 clinical trial of RTX-240 for the treatment of solid tumors, including clinical CRO and internal manufacturing costs, as well as to costs incurred for preclinical, IND-enabling activities and clinical startup costs for RTX-321. The increase in cancer program costs was offset by a $19.0 million decrease in expenses related to our rare disease pipeline following the deprioritization of these programs in March 2020. Additionally, costs not allocated to programs were reduced by $13.9 million driven primarily by a shift in manufacturing activities towards the technical development and production of clinical supply for our oncology programs and the shift in discovery activities from the development of clinical candidates towards work to enable more advanced programs.

G&A expenses were $50.3 million during the twelve months of 2020, as compared to $57.2 million for the same period in 2019. The lower costs were principally driven by a reduction in stock-based compensation expense.

Cash Position
As of December 31, 2020, cash, cash equivalents and investments were $176.3 million as compared to $283.3 million as of December 31, 2019, providing Rubius with a cash runway into the first quarter of 2022. The Company has the ability to extend runway into the middle of 2022 by scaling back certain activities. During 2020, the Company used $127.8 million of cash to fund operations and $5.5 million to fund capital expenditures, consisting of payments for assets purchased in 2019 related to our manufacturing facility in Rhode Island, as well as computer and laboratory equipment purchased in 2020 for both of our locations. In addition, during 2020 the Company drew down the third and final tranche of $25.0 million pursuant to its $75.0 million loan agreement with Solar Capital.

On February 23, 2021 Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) ("Kiniksa"), a biopharmaceutical company with a pipeline of assets designed to modulate immunological pathways across a spectrum of diseases, reported fourth quarter and full-year 2020 financial results and corporate and pipeline activity (Press release, Kiniksa Pharmaceuticals, FEB 23, 2021, View Source [SID1234575440]).

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"Kiniksa delivered outstanding performance in 2020, with encouraging clinical data across our pipeline as well as Breakthrough Therapy designation or Orphan Drug designation for rilonacept, mavrilimumab and vixarelimab," said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. "Building on this momentum, we expect to launch rilonacept in recurrent pericarditis, pending FDA approval, in the first half of 2021. We also anticipate final Phase 1 data for KPL-404, our anti-CD40 program, in which data generated to-date support the best-in-class potential of this molecule and the opportunity for its evaluation in multiple devastating autoimmune diseases. Further, we expect data from the fully-enrolled mavrilimumab Phase 2 clinical trial in severe COVID-19 in the first half of this year. With these significant advances, it is important to highlight we are well capitalized with cash reserves of approximately $323 million."

Pipeline Activity
Rilonacept (IL-1α and IL-1β cytokine trap)

Kiniksa is preparing for the commercial launch of rilonacept in recurrent pericarditis in the first half of 2021, if approved by the U.S. Food and Drug Administration (FDA).
The FDA accepted the supplemental Biologics License Application (sBLA) for rilonacept in recurrent pericarditis with priority review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 21, 2021.
Upon approval in recurrent pericarditis, Kiniksa will commence sales and distribution of rilonacept for the approved indications in the United States, including cryopyrin-associated periodic syndromes (CAPS), and deficiency of IL-1 receptor antagonist (DIRA), and evenly split profits with Regeneron Pharmaceuticals, Inc. (Regeneron).
Kiniksa is building commercial competencies in-house, including sales operations, value and access, and sales and marketing teams. In addition, the company is generating evidence on unmet need and disease burden, building disease awareness with payers, physicians, and advocacy groups, and establishing core capabilities such as distribution, patient services and data management.
Kiniksa achieved a regulatory milestone in the fourth quarter of 2020, which triggered a $7.5 million payment to Regeneron. The company is obligated to make a regulatory milestone payment of $20 million through the potential approval of rilonacept in recurrent pericarditis.
Mavrilimumab (monoclonal antibody inhibitor targeting GM-CSFRα)

Kiniksa expects to provide next steps for mavrilimumab, including for giant cell arteritis (GCA), in the first half of 2021.
Kiniksa is conducting a Phase 2/3 clinical trial of mavrilimumab in severe COVID-19 pneumonia and hyperinflammation.
Kiniksa completed enrollment in the Phase 2 portion of the clinical trial and expects data in the first half of 2021.
Kiniksa is enrolling patients in the Phase 3 portion of the clinical trial.
Vixarelimab (monoclonal antibody inhibitor of signaling through OSMRβ)

Kiniksa is conducting a Phase 2b dose-ranging clinical trial of vixarelimab in prurigo nodularis.
The Phase 2b clinical trial is expected to enroll approximately 180 patients experiencing severe pruritus. Patients will be randomized to receive vixarelimab or placebo subcutaneously once-monthly.
The primary efficacy endpoint is the percent change from baseline in the weekly-average Worst-Itch Numeric Rating Scale (WI-NRS) at Week 16.
Key secondary efficacy endpoints include the proportion of patients achieving a greater-than-or-equal-to 4-point weekly-average WI-NRS reduction at Week 16 and the proportion of patients achieving a 0/1 score (clear/almost clear) on the prurigo nodularis-investigator’s global assessment (PN-IGA) at Week 16.
KPL-404 (monoclonal antibody inhibitor of signaling between CD40 and CD40L)

Kiniksa expects final data and safety follow-up from all cohorts of the single-ascending-dose Phase 1 clinical trial of KPL-404 in the first half of 2021.
Preliminary Phase 1 data showed full receptor occupancy through Day 29 at the 3 mg/kg intravenous dose.
This data corresponded with complete suppression of the T-cell Dependent Antibody Response (TDAR) to the novel antigen keyhole limpet hemocyanin (KLH) through Day 29.
KPL-404 is a monoclonal antibody inhibitor designed to disrupt CD40-CD40 ligand (CD40L) signaling, a well-known pathway that plays a critical role in regulating B cell proliferation and T cell activation as well as antibody production.
Dysregulation of the CD40-CD40L pathway has been implicated in a broad range of autoimmune diseases including rheumatoid arthritis, Sjogren’s syndrome, Graves’ disease, systemic lupus erythematosus and the prevention of solid organ transplant graft rejection.
Financial Results

Net loss for the fourth quarter of 2020 was $53.7 million, compared to a net loss of $31.8 million for the fourth quarter of 2019. Net loss for the full-year 2020 was $161.4 million, compared to a net loss of $161.9 million for the full-year 2019.
Total operating expenses for the fourth quarter of 2020 were $52.9 million, compared to $32.6 million for the fourth quarter of 2019. Total operating expenses for the full-year 2020 were $157.4 million, compared to $170.0 million for the full-year 2019.
Non-cash, share-based compensation expense for the fourth quarter of 2020 was $6.3 million, compared to $5.0 million for the fourth quarter of 2019. Non-cash, share-based compensation expense for the full-year 2020 was $20.9 million, compared to $15.1 million for the full-year 2019.
Total operating expenses for the fourth quarter and full-year 2020 included a $7.5 million payment made to Regeneron related to our achievement of a regulatory milestone.
As of December 31, 2020, the company had cash, cash equivalents and short-term investments of $323.5 million and no debt.
Financial Guidance

Kiniksa expects that its cash, cash equivalents and short-term investments will fund its current operating plan into 2023.

On February 23 2021 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported that Stephen S. Yoder, President and Chief Executive Officer of Pieris, is scheduled to participate in a GI Oncology and Pancreatic Cancer panel discussion at the Cowen 41st Annual Healthcare Conference on Tuesday, March 2, 2021 at 9:50 AM EST (Press release, Pieris Pharmaceuticals, FEB 23, 2021, View Source [SID1234575439]).

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