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 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies and cytokines for the treatment of cancer and autoimmune diseases, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided a review of recent business and clinical highlights (Press release, Xencor, FEB 23, 2021, View Source [SID1234575461]).

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"Throughout 2020, we advanced multiple clinical programs, introduced several new XmAb bispecific technologies, and saw the progress of many partnered programs, including U.S. regulatory approval for tafasitamab, the second antibody with XmAb technology to achieve commercialization. Internally, early-stage clinical data have guided our decisions to advance several candidates into new studies scheduled for 2021. These include a potentially registrational study to evaluate the chemotherapy-free, triple combination of plamotamab, tafasitamab and lenalidomide for patients with relapsed or refractory DLBCL and a study of XmAb717 in patients with prostate cancer, a population with high unmet need and in whom we have seen encouraging activity to date," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "To continue enriching our pipeline, we have entered into new collaborations with partners who identify novel tumor targets that we can engineer XmAb bispecific antibodies against. This includes our recently started partnership with Janssen Biotech, where we are engineering CD28 bispecific antibodies against a prostate tumor target and can also access their leading prostate cancer therapeutics portfolio for combination clinical studies with our growing prostate cancer pipeline."

Dr. Dahiyat added, "Looking ahead, we will continue to present maturing data from our clinical-stage programs and soon expect to initiate a Phase 1 study for XmAb564, our wholly owned IL-2 cytokine that we engineered to preferentially activate regulatory T cells, an emerging mechanism for treating patients with autoimmune diseases. In addition, we plan to submit an IND by year-end for XmAb819, an ENPP3 x CD3 bispecific antibody for renal cell carcinoma. Notably, this program is engineered with our multi-valent XmAb 2+1 format, which enables CD3 bispecific antibodies with greater tumor selectivity and against an expanded set of novel tumor antigens."

Recent Business and Clinical Highlights

Plamotamab (CD20 x CD3): In November 2020, the Company entered into a clinical collaboration with MorphoSys AG and Incyte Corporation to investigate the chemotherapy-free triple combination of plamotamab, tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), first-line DLBCL and relapsed or refractory follicular lymphoma. Plamotamab, which redirects T cells to tumors, and tafasitamab, a CD19-directed XmAb antibody, combine powerful and distinct immune pathways, and this collaboration is designed to generate new clinical insights and accelerate development timelines for the program. MorphoSys and Incyte will provide tafasitamab for the studies, which Xencor will sponsor and fund. Tafasitamab is co-commercialized in the U.S. by MorphoSys and Incyte and is marketed as Monjuvi. Monjuvi, the second product with XmAb technology to be approved for commercial marketing, was approved by the U.S. FDA in July 2020.
XmAb717 (PD-1 x CTLA-4): In November 2020, the Company presented updated interim data from the Phase 1 study of XmAb717 (formerly XmAb20717) in patients with multiple types of advanced solid tumors at the SITC (Free SITC Whitepaper) Annual Meeting. XmAb717 was generally well-tolerated. As of the September 2020 data cutoff, a complete response was observed in a patient with melanoma, and partial responses were observed in multiple tumor types, including melanoma, renal cell carcinoma (RCC), non-small cell lung cancer (NSCLC), castration-resistant prostate cancer (CRPC) and ovarian cancer. In the first half of 2021, the Company plans to initiate a Phase 1b study of XmAb717 for patients with certain molecular subtypes of CRPC, as a monotherapy or in combination depending on the subtype, as these patients represent a high unmet medical need.
Vibecotamab (CD123 x CD3): In December 2020, the Company presented updated data from the Phase 1 study of vibecotamab in patients with relapsed or refractory acute myeloid leukemia (AML) at the ASH (Free ASH Whitepaper) Annual Meeting. The most common adverse event was cytokine release syndrome, the majority of which was observed in the first dose, and it was generally manageable with premedication. The efficacy and biomarker analyses indicated that responses appeared to be associated with lower baseline disease burden. The Company is continuing the dose escalation study and is reviewing data with Novartis in planning additional studies of vibecotamab.
XmAb564 (IL2-Fc Cytokine): XmAb564 (formerly XmAb27564) is a wholly owned, engineered IL2-Fc fusion that the Company is developing for the treatment of patients with autoimmune diseases. In January 2021, an investigational new drug (IND) application for XmAb564 was allowed by the FDA, and the Company plans to initiate a Phase 1 study in healthy volunteers in early 2021.
Select New Collaborations and Progress Across Partnered Programs

Janssen Biotech: In November 2020, Xencor entered into an agreement with Janssen Biotech, Inc., focused on the discovery of XmAb bispecific antibodies against CD28, an immune co-stimulatory receptor on T cells, and an undisclosed prostate tumor target, for the potential treatment of patients with prostate cancer. Additionally, the Company has a right to access select, predefined agents from Janssen’s portfolio of clinical-stage drug candidates and commercialized medicines to evaluate potential combination therapies in prostate cancer with agents in the Company’s own pipeline, subject to some limitations. Janssen has the same right with Xencor’s portfolio to evaluate potential combination therapies in prostate cancer. The Company received a $50 million upfront payment from Janssen and is eligible for future potential milestone and royalty payments.
MD Anderson: In December 2020, Xencor entered into a new agreement with The University of Texas MD Anderson Cancer Center to develop novel CD3 bispecific antibody therapeutics for the potential treatment of patients with cancer. MD Anderson will work to identify and develop promising antibodies, and the Company will apply its XmAb Fc bispecific technology to create therapeutic candidates. MD Anderson will then conduct and fund all preclinical activities to advance candidates toward clinical studies. Xencor has certain exclusive options to license worldwide rights to develop and commercialize potential new medicines arising from the collaboration.
Viridian Therapeutics: In December 2020, Xencor entered into a technology license agreement with MiRagen Therapeutics, Inc., which received a non-exclusive license to Xtend Fc technology and an exclusive license to apply Xtend Fc technology to antibodies targeting IGF-1R. MiRagen subsequently changed its name to Viridian Therapeutics, Inc. The Company received an upfront payment of common stock valued at $6 million.
In November 2020, Xencor entered into a product license agreement with a newly formed, privately held biotechnology company which received the exclusive worldwide rights to develop and commercialize three preclinical-stage Fc-engineered drug candidates for autoimmune disease—XmAb6755, XPro9523 and XmAb10717—programs incorporating an Xtend Fc Domain, a Cytotoxic Fc Domain, or both. The Company received a 15% equity interest in the company and is eligible to receive royalties on net sales of approved products in the mid-single digit to mid-teen percentage range.
Ultomiris (Alexion): Alexion’s Ultomiris uses Xtend Fc technology for longer half-life, and it has received marketing authorizations from regulatory agencies in the U.S., Europe and Japan for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) and for patients with atypical hemolytic uremic syndrome (aHUS). Alexion is also evaluating Ultomiris in a broad late-stage development program across many indications in neurology and nephrology. In 2020, the Company earned $16.2 million in royalties and, in the fourth quarter of 2020, received a $10 million sales-based milestone payment from Alexion.
Monjuvi is a registered trademark of MorphoSys AG. Ultomiris is a registered trademark of Alexion Pharmaceuticals, Inc.

Fourth Quarter and Full Year Ended December 31, 2020 Financial Results

Cash, cash equivalents and marketable securities totaled $604.0 million as of December 31, 2020, compared to $601.3 million on December 31, 2019. During the year, the Company received upfront payments, milestone payments and royalties from partners of $165 million, which offset spending on operations and resulted in an increase in the year-end cash balance.

Revenues for the fourth quarter ended December 31, 2020 were $41.9 million, compared to $3.5 million for the same period in 2019. Revenues for full year 2020 were $122.7 million, compared to $156.7 million in 2019. Revenues for the three-month period ended December 31, 2020 were earned primarily from the licensing of XmAb technologies and drug candidates and a sales-based milestone payment from Alexion, compared to revenues from the same period in 2019, which were primarily Alexion royalties. Total revenues earned in 2020 included royalties and milestones from the MorphoSys and Alexion agreements and the licensing of XmAb technologies and drug candidates, compared to revenue earned from Xencor’s Genentech and Astellas collaborations in 2019.

Research and development expenditures for the fourth quarter ended December 31, 2020 were $47.9 million, compared to $27.3 million for the same period in 2019. Research and development expenditures were $169.8 million for the full year ended December 31, 2020, compared to $118.6 million in 2019. Research and development spending for the fourth quarter and full year ended December 31, 2020 was greater than expenditures incurred over the comparable periods in 2019, primarily due to increased spending on Xencor’s bispecific antibody and cytokine candidates and technologies.

General and administrative expenses for the fourth quarter ended December 31, 2020 were $7.6 million, compared to $6.7 million in the same period in 2019. General and administrative expenses were $29.7 million in the full year 2020, compared to $24.3 million in 2019. Additional general and administrative spending for the full year ended December 31, 2020 over the comparable period in 2019 reflects increased staffing, professional expenses and spending on intellectual property.

Non-cash, share based compensation expense for the year ended December 31, 2020 was $31.6 million, compared to $31.9 million for the year ended December 31, 2019.

Net loss for the fourth quarter ended December 31, 2020 was $13.7 million, or $(0.24) on a fully diluted per share basis, compared to a net loss of $26.9 million, or $(0.47) on a fully diluted per share basis, for the same period in 2019. For the full year ended December 31, 2020, net loss was $69.3 million, or $(1.21) on a fully diluted per share basis, compared to a net income of $26.9 million, or $0.46 on a fully diluted per share basis, for the full year ended December 31, 2019. The lower net loss reported for the three months ended December 31, 2020 compared to the same period in 2019 is primarily due to higher revenue reported in the three months ended December 31, 2020, while the net loss reported for 2020 compared to the net income reported for 2019 is primarily due to higher research and development expenses and lower licensing and milestone revenue reported in 2020.

The total shares outstanding were 57,873,444 as of December 31, 2020, compared to 56,902,301 as of December 31, 2019.

Financial Guidance

Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations into 2024. Xencor expects to end 2021 with between $425 million and $475 million in cash, cash equivalents and marketable securities.

Conference Call and Webcast

Xencor will host a conference call today at 4:30 p.m. ET (1:30 p.m. PT) to discuss these fourth quarter and full year 2020 financial results and provide a corporate update.

The live call may be accessed by dialing (877) 359-9508 for domestic callers or +1 (224) 357-2393 for international callers and referencing conference ID number 1163598. A live webcast of the conference call will be available online from the Investors section of the Company’s website at www.xencor.com. The webcast will be archived on the company’s website for 30 days.

On February 23, 2021 TG Therapeutics, Inc. (NASDAQ: TGTX) reported the publication of final results from the Phase 3 GENUINE trial evaluating ublituximab, the Company’s investigational glycoengineered anti-CD20 monoclonal antibody, in combination with ibrutinib, in patients with relapsed or refractory high-risk chronic lymphocytic leukemia (CLL), in The Lancet Haematology (Press release, TG Therapeutics, FEB 23, 2021, View Source [SID1234575477]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer stated, "The Phase 3 data published yesterday, and previously presented, demonstrated that the addition of ublituximab to ibrutinib significantly improved overall response rate, complete response rate as well as prolonged progression-free survival. Significant unmet need still exists within the CLL landscape, and patients with high-risk relapsed or refractory CLL progress more rapidly than those without high-risk cytogenetics. The outcome of the GENUINE study is therefore very encouraging, and we believe these data are supportive of pursuing combination strategies with ublituximab for high-risk CLL patients." Mr. Weiss continued, "We look forward to bringing ublituximab to market as soon as possible as we pursue completion of a BLA submission with the FDA in the first half of 2021 for the combination of ublituximab plus umbralisib for patients with CLL."

Jeff P. Sharman, MD, Director of Research at Willamette Valley Cancer Institute and Medical Director of Hematology Research for The US Oncology Network and Study Chair for the GENUINE trial stated, "The utility of adding anti-CD20 therapy in combination with BTK inhibitors, such as ibrutinib, has long been unclear with prior studies using rituximab having failed to demonstrate an improvement in long-term outcomes. These results published from the GENUINE study are encouraging and may suggest that next generation anti-CD20 antibodies could have value in combination approaches to treating CLL."

The manuscript includes data from 126 patients with relapsed or refractory high-risk CLL who were randomized on study, of which 117 received at least one dose of treatment and were included in safety analyses, with 59 receiving ublituximab plus ibrutinib and 58 receiving ibrutinib monotherapy. Ibrutinib was given orally daily at 420 mg for all cycles. Ublituximab was given intravenously in 28-day cycles with up to 150 mg on day 1, 750 mg on day 2, and 900 mg on days 8 and 15 of cycle 1, and continuing at 900 mg on day 1 of cycles 2 through 6. Beyond cycle 6, ublituximab was given at 900 mg every 3 months. Ublituximab and ibrutinib were continued until unacceptable toxicity, disease progression, or withdrawn consent. The primary endpoint was independent review committee (IRC) assessed overall response rate (ORR) per iwCLL 2008 criteria. Key highlights from this manuscript include:

The IRC-assessed ORR among treated patients was 90% (53 of 59) in the ublituximab-ibrutinib arm and 69% (40 of 58) in the ibrutinib arm (p=0.0060), with a CR/CRi rate of 20% (12 of 59) and 5% (3 of 58), respectively (p=0.024).
After a median follow-up of 41.6 months, median IRC-assessed progression-free survival (PFS) in all treated patients was not reached in the ublituximab-ibrutinib group (95% CI, not estimable [NE]) after 15 PFS events and 35.9 months (95% CI, 17·0-NE) in the ibrutinib group after 25 PFS events (hazard ratio [HR], 0.46; 95% CI, 0.24-0.87).
The most common grade 3/4 adverse events in the ublituximab-ibrutinib group and the ibrutinib group were neutropenia (19%; 12%), anaemia (8%; 9%), and diarrhea (10%; 5%).
These data are described further in the manuscript entitled, "A Phase 3, Randomized Trial of Ublituximab Plus Ibrutinib for Patients With Relapsed/Refractory High-Risk Chronic Lymphocytic Leukaemia," which was published [online yesterday] in The Lancet Haematology. The online version of the article can be accessed at https://www.thelancet.com/journals/lanhae/home.

On February 23, 2021 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the company will webcast its corporate presentation at the Cowen 41st Annual Healthcare conference (Press release, Jazz Pharmaceuticals, FEB 23, 2021, https://www.prnewswire.com/news-releases/jazz-pharmaceuticals-announces-participation-at-cowen-41st-annual-healthcare-conference-301232868.html [SID1234575493]).

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Bruce Cozadd, chairman and chief executive officer, will provide an overview of the company and a business and financial update on Tuesday, March 2, 2021, at 12:50 p.m. EST / 5:50 p.m. GMT.

A live audio webcast of each presentation may be accessed from the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com. Please connect to the website prior to the start of each presentation to ensure adequate time for any software downloads that may be necessary to listen to the webcast.

An archive of each webcast will be available for at least one week following each presentation on the Investors section of the company’s website at www.jazzpharmaceuticals.com.

On February 23, 2021 Graviton Bioscience Corporation ("Graviton"), a privately held early-stage drug development company founded by Dr. Samuel Waksal, and Beijing Tide Pharmaceutical Co., Ltd. (Beijing Tide) a subsidiary of Sino Biopharmaceutical Limited (HKG:1177), reported the signing of an exclusive license agreement for TDI01 (Press release, Graviton Bioscience, FEB 23, 2021, View Source [SID1234576629]). The agreement grants development and commercialization rights for TDI01 to Graviton in all territories, excluding China. Under the terms of the agreement, Graviton will make an upfront payment and additional amounts for development, regulatory and sales milestone payments for all programs, as well as royalties on net sales and an option for a revenue-sharing arrangement for certain developed products. The aggregate amount of upfront, development, regulatory and sales milestone payments is up to USD 517.5 million.

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TDI01 is a targeted inhibitor of Rho/Rho containing protein kinase 2 (ROCK2). In 2020, an Investigational New Drug application for evaluation of TDI01 in fibrosis was cleared by the FDA, and a clinical trial application for the treatment of fibrosis was filed and accepted by the Center for Drug Evaluation of the People’s Republic of China. A Phase I clinical trial of the drug candidate in idiopathic pulmonary fibrosis is currently ongoing in the United States. Graviton expects to develop this novel therapeutic for the treatment of various serious diseases, including exploration of TDI01’s ability to penetrate the central nervous system, and the translation of efficacy demonstrated in certain preclinical cancer models as well as models of certain viral diseases.

"There remains a tremendous unmet need for novel, safe and effective therapies against a broad spectrum of diseases globally," said Dr. Waksal, Founder of Graviton. "Having pioneered the field in targeting ROCK2 mediated disease, I believe that the extraordinarily high selectivity and potency engineered by Beijing Tide in developing TDI01 offers great potential in advancing the next-generation of ROCK2 therapeutics. We look forward to moving TDI01 into different clinical studies in the U.S. as quickly as possible, and to begin elucidating this potential in the coming quarters."

"TDI01 is the first innovative small molecule in the clinical phase developed by Beijing Tide, and it shows great potential in treating a variety of diseases," said Sino Biopharmaceutical Limited’s Chairlady Theresa Tse. "We believe that collaborating with the experienced scientific team at Graviton would not only maximize the value of TDI01, but also accelerate the clinical development in both China and US. We look forward to initiating multiple clinical studies for different indications very soon."