On November 5, 2020 Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP), reported its financial results for the third quarter ended September 30, 2020 and provided a corporate update. The Company will host a conference call and webcast today at 4:30 pm ET (Press release, Ziopharm, NOV 5, 2020, View Source [SID1234570049]).

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"During the third quarter, we again made progress in all three programs and strengthened the Board of Directors, Scientific Advisory Board and Executive Team," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer. "Ziopharm is on track to file an IND for the first TCR-T trial early next year and the NCI is taking steps to begin the phase 2 Sleeping Beauty TCR-T trial under their direction. We are pleased by Eden BioCell’s steps to submit an IND for the RPM CAR-T trial in Taiwan to infuse T cells the day after gene transfer and heartened by the initial reports we are receiving regarding the first patients dosed by Eden BioCell and partners under compassionate use. Later this month, we will share data from our Controlled IL-12 program at the 2020 Society for Neuro-Oncology Annual Meeting."

Recent Corporate Highlights

Sleeping Beauty TCR-T Program

Personalized and Library TCR-T Clinical Trials with MD Anderson Cancer Center. During the third quarter, Ziopharm further expanded its library of T-cell receptors (TCRs) targeting shared neoantigens in hotspots for use with the Sleeping Beauty platform. The Company remains on track to file an Investigational New Drug (IND) application with the FDA in the first quarter of next year, seeking clearance to begin its TCR-T trial utilizing allogeneic TCRs from its library. The Company is working closely with MD Anderson, the initial site for this trial, which is expected to commence mid-2021 to treat patients with gynecologic, colorectal, pancreatic, non-small cell lung and cholangiocarcinoma cancers. The Company continues with its planning for a clinical trial evaluating its Personalized TCR-T platform, which will be initiated at MD Anderson after the Library TCR-T trial.

NCI Phase 2 Personalized TCR-T Trial. This phase 2 study, under the direction of Steven Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the National Cancer Institute (NCI), and his team, is a first-in-human, non-viral TCR-T study that will enroll patients with a range of solid tumors. The NCI controls the timing of patient treatment for this trial, which is now estimated for next year. During the quarter, data regarding the engineering runs conducted by Ziopharm were successfully transferred to the NCI and the manufacturing process was authenticated by their team and is being validated in their GMP facility. Despite this, the NCI has recently informed Ziopharm that enrollment to the trial will additionally be delayed by regulatory requirements being implemented by the National Institute of Health as well as the impact of COVID-19. The ongoing pandemic led to the depletion of patients available to be treated on TCR-T trials at the NCI due to disease progression while the facility was largely shut down and has added time needed to accrue new patients. As previously mentioned, the NCI has been proactively screening patients for neoantigens and TCRs to render them eligible for the trial (NCT0402436).
Sleeping Beauty CAR-T Program

Eden BioCell CAR-T Study. The Company’s joint venture partner, Eden BioCell, has commenced filing of an IND for a clinical trial in Taiwan to assess patient-derived (autologous) CD19-specific membrane bound IL-15 (mbIL15) CAR-T cells, produced using our Rapid Personalized Manufacturing (RPM) platform. The team expects the filing to be complete before year end, as planned. In addition, Eden BioCell and partners have dosed several patients with relapsed CD19+ malignancies under compassionate use, infusing autologous CAR-T the day after gene transfer per RPM. They report initial data showing the presence of infused T cells, measured weeks after infusion, in peripheral blood and bone marrow. Preliminary observations appear to indicate that mbIL15 supports the manufacturing of CAR-T under RPM which can be safely infused without unexpected toxicities. Additional follow-up is underway in Asia.

Ziopharm CAR-T Study. The Company’s clinical collaborators at MD Anderson are actively screening and evaluating patients for enrollment in the phase 1 clinical trial infusing donor-derived (allogeneic) CD19-specific CAR-T therapies produced using RPM. Up to 24 patients with CD19+ leukemias and lymphomas who have relapsed after allogeneic bone marrow transplantation will be enrolled in this investigator-initiated trial (NCT03579888).
Controlled IL-12 Program

SNO 2020. Three abstracts detailing clinical data and observations of Controlled IL-12 have been accepted for presentation at the upcoming 2020 Society for Neuro-Oncology (SNO) virtual annual meeting next month. Updates to be presented on Controlled IL-12 in DIPG (NCT03330197) and combination studies of Controlled IL-12 with Opdivo as a phase 1 trial (NCT03636477) and with Libtayo as a phase 2 trial (NCT04006119) in recurrent glioblastoma (rGBM).

Pediatric Trial. In the Company’s phase 1/2 study of Controlled IL-12 for the treatment of pediatric brain tumors, all three clinical sites are now active. The trial is designed to evaluate the safety and tolerability of a single intratumoral injection of Ad-RTS-hIL-12 with up to 14 days of oral veledimex in children with diffuse intrinsic pontine glioma (DIPG). Up to 12 patients with DIPG may be enrolled in phase 1 of the study, which is being conducted at: Lurie Children’s Hospital, the Dana-Farber Cancer Institute and the University of California in San Francisco. The FDA recently granted a Rare Pediatric Disease Designation to Controlled IL-12 for the investigational treatment of DIPG.
Operational

Expanded Board of Directors. During the third quarter, Ziopharm announced the appointment of two new members of the Company’s Board of Directors. Biotech entrepreneur James Huang, Managing Partner at Kleiner Perkins Caufield & Byers China, was added to the Board in July, and in September, Kevin Buchi, biotech industry veteran and former CEO of Cephalon joined the Board. Directors will continue to actively review the Board membership, in coordination with a retained national search firm, to ensure the skills and experience of directors support the progress and future prospects of the business.

Scientific Advisory Board. Following the appointment of immunotherapy pioneer, Carl June, M.D., as Chairman of Ziopharm’s Scientific Advisory Board (SAB), the Company announced population of the SAB in September with the addition of Adi Barzel, Ph.D., Gavin Dunn, M.D., Ph.D., Matthew Porteus, M.D., Ph.D., and Kole Roybal, Ph.D. The SAB will provide strategic counsel to guide the efficient development of Ziopharm’s innovative technologies and pipeline of immunotherapies.

Executive Leadership. Ziopharm is pleased to welcome former Gilead executive Adam Levy, Ph.D., M.B.A., as EVP, Investor Relations and Corporate Communications. Most recently, Dr. Levy was Executive Director and Head, Corporate Strategy and Investor Relations for Gilead Sciences. Previously, Dr. Levy was VP, Corporate Strategy for Alexion and Executive Director, Corporate Strategy for Bristol-Myers Squibb. He had prior leadership positions with Novartis and McKinsey & Company. Adam holds a Ph.D. in Molecular Biology from the University of Illinois and an MBA in Finance and Strategy from Northwestern University Kellogg School of Management.
Third Quarter 2020 Financial Results

Research and development expenses were $14.0 million for the third quarter of 2020, compared to $8.6 million for the third quarter of 2019, primarily reflecting increased manufacturing activity and headcount.

General and administrative expenses were $6.4 million for the third quarter of 2020, compared to $4.8 million for the third quarter of 2019. The increase in general and administrative expenses for the third quarter of 2020 is primarily due to increased headcount, legal costs associated with its expanded patent portfolio and facility costs.

Net loss for the third quarter of 2020, was $20.3 million, or $(0.10) per share, compared to a net loss of $74.0 million, or $(0.43) per share, for the third quarter of 2019 (which reflected a $60.8 million, $(0.36) per share, non-cash charge for an inducement warrant).

Cash and cash equivalents, as of September 30, 2020 were $135.5 million.

Additionally, a prepayment of approximately $11.4 million remains for work to be conducted by the Company at MD Anderson under the Company’s research and development agreements.
Conference Call and Webcast
Ziopharm will host a conference call and webcast for the investment community today, November 5, 2020, at 4:30 p.m. ET. The conference call can be accessed by dialing 1-800-920-9723 (U.S. and Canada) or 1-212-231-2932 (international). The passcode for the conference call is 21971110. To access the live webcast or the subsequent archived recording, click here or visit the "Investors" section of the Ziopharm website at www.ziopharm.com. The webcast will be recorded and available for replay on the company’s website for two weeks.

On November 5, 2020 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer and autoimmune diseases, reported financial results for the third quarter ended September 30, 2020 and provided a review of recent business and clinical highlights (Press release, Xencor, NOV 5, 2020, View Source [SID1234570048]).

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"In 2020, we have reported advances for multiple programs in our clinical pipeline. Monjuvi became the second drug with XmAb Fc technology to receive marketing approval in the United States, and we reported initial clinical results from multiple programs across our portfolio of XmAb bispecific antibodies in oncology. In the coming weeks, we will present additional clinical data from some of these programs, including updated results from the Phase 1 studies of XmAb20717 at SITC (Free SITC Whitepaper), and vibecotamab at ASH (Free ASH Whitepaper)," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "We also continue to leverage our protein engineering expertise to rapidly generate drug candidates with our XmAb bispecific Fc domains, in order to advance a range of innovative technologies and molecules, and at SITC (Free SITC Whitepaper) next week, we will be presenting new preclinical data from our B7-H3 x CD28 and PD-1 x TGFβR2 bispecific antibody programs, as well as our IL-12-Fc program."

Dr. Dahiyat continued, "Looking ahead to next year, we are on track to initiate a Phase 1 study of XmAb27564, our IL-2 cytokine for autoimmune disease, in healthy volunteers in early 2021. Subject to potential COVID-19 impacts, we also plan to advance clinical studies across all our CD3 programs including vibecotamab, plamotamab, tidutamab and XmAb30819, which is our first XmAb 2+1 bispecific antibody and targets the underexplored tumor target ENPP3."

COVID-19 Business Update

Clinical Studies: The pandemic did not significantly disrupt patient enrollment to Xencor’s six ongoing clinical studies during the third quarter of 2020. Manufacturers that provide the Company’s drug supply are currently experiencing critical shortages of material used in their manufacturing processes. Xencor has sufficient supplies of drug material to continue conducting ongoing studies without interruption; however, delays are expected in the development timelines for the preclinical XmAb30819 program. Timelines for additional early-stage programs and ongoing clinical programs could be affected if the supply interruption extends longer than current estimates.

Workforce and Research Operations: During the third quarter, Xencor continued to require non-laboratory employees to work remotely.

Licensing and Partnerships: Xencor is monitoring potential impacts to partnership revenues, which are primarily milestone payments and royalties. There was no impact during the third quarter as the Company earned revenue from its partners and collaborators including Alexion, MorphoSys and Omeros. If the pandemic affects the sales or clinical and regulatory progress of partnered programs, Xencor’s revenue could be adversely affected in the future.

Recent Business and Clinical Highlights

Tidutamab (SSTR2 x CD3): In October, the Company presented initial dose-escalation data from the ongoing Phase 1 study in patients with neuroendocrine tumors (NET). Tidutamab was generally well tolerated at the recommended dose identified for the expansion portion of the study, a 0.3 mcg/kg priming dose and subsequent 1.0 mcg/kg repeated doses. Peripheral blood biomarkers indicated tidutamab induced acute and sustained T-cell activation at this dose, and a dose-dependent increase in proliferation and activation markers of CD8+ T cells was observed, which is consistent with tidutamab’s mechanism of action. The best overall response was stable disease in the analysis to describe clinical activity (n=14), and the median duration of treatment was approximately seven months. Completion of enrollment and longer follow-up are required to evaluate progression-free survival and the clinical utility of tidutamab for patients with NET. Xencor plans to initiate an additional clinical study in patients with Merkel cell carcinoma and small cell lung cancer, SSTR2-expressing tumor types known to be responsive to immunotherapy, in early 2021.

New Clinical Collaboration: In September, Xencor and MD Anderson entered a strategic collaboration to design and execute new investigator-sponsored clinical studies with Xencor’s portfolio of XmAb drug candidates, including novel bispecific antibodies and engineered cytokines. Xencor is committing $10 million in funding and supporting these studies over an initial five-year term.

Select Internal Programs: Xencor will present updated data from the vibecotamab and XmAb20717 programs, as well as data from several preclinical-stage programs, at upcoming scientific and medical meetings.

● Vibecotamab (CD123 x CD3): Updated results from the ongoing Phase 1 study in patients with acute myeloid leukemia will be presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020. Patient enrollment continues in this study, and Xencor plans to initiate additional clinical studies evaluating vibecotamab in 2021.

● Plamotamab (CD20 x CD3): Patient enrollment continues in the Phase 1 study in non-Hodgkin lymphoma and chronic lymphocytic leukemia, with planned expansion cohorts expected to open in 2021. In addition, operational preparation for a Phase 2 monotherapy trial in diffuse large B-cell lymphoma (DLBCL) is underway, as well as for a Phase 2 combination therapy study.

● XmAb20717 (PD-1 x CTLA-4): Updated results from the ongoing Phase 1 study in patients with advanced solid tumors will be presented at the 35th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) in November 2020. The study is currently enrolling patients with renal cell carcinoma to an expansion cohort and continues to enroll patients in additional dose-escalation cohorts. Expansion cohorts for patients with melanoma, advanced non-small cell lung cancer, prostate cancer, and other cancers without approved checkpoint therapies are fully enrolled.

● New data from three preclinical-stage programs, including the IL-12-Fc cytokine program, the CD28 bispecific antibody platform, and the PD-1 x TGFβR2 bispecific antibody program, will also be presented at the SITC (Free SITC Whitepaper) Annual Meeting in November 2020.

Select Partnered Programs: Xencor’s partners expand the use of XmAb technology by providing late-stage development capabilities, successful track records of developing or commercializing programs or have programs for potential combination with Xencor’s bispecific antibody or cytokine drug candidates. Additionally, the plug-and-play nature of XmAb technologies enables selective access for licensees with limited effort or resources by Xencor.

● Monjuvi (MorphoSys): Monjuvi is a CD19-directed cytolytic antibody indicated in combination with lenalidomide as a second-line treatment option for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). Tafasitamab, which was engineered with an XmAb Cytotoxic Fc Domain, was created at Xencor and is the second product with Xencor’s XmAb technology to be approved by the FDA. Upon Monjuvi’s approval in July, Xencor earned a $25 million milestone payment from MorphoSys under the license agreement between the companies and is eligible to receive royalties on worldwide net sales in the high-single to low-double digit percent range, as well as additional development, regulatory and sales milestone payments. The European Marketing Authorization Application for tafasitamab is currently under review, and MorphoSys expects a decision in the second half of 2021.

● Ultomiris (Alexion): Alexion’s Ultomiris uses Xtend technology for longer half-life. In September, Japan’s Ministry of Health, Labour and Welfare approved Ultomiris for adults and children with atypical hemolytic uremic syndrome (aHUS). Ultomiris previously 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 in the U.S. and Europe for aHUS. In addition to evaluating Ultomiris in a broad late-stage development program, Alexion is conducting a randomized, controlled Phase 3 study in adults with COVID-19 who are hospitalized with severe pneumonia or acute respiratory distress syndrome. Xencor is eligible to receive additional sales-based milestone payments and a low single-digit royalty on net sales of Ultomiris.

● VIR-7831 and VIR-7832 (Vir Biotechnology): Vir has non-exclusive access to Xencor’s Xtend Fc technology to extend the half-life of VIR-7831 and VIR-7832, novel antibodies that Vir is investigating as potential treatments for patients with COVID-19, as well as prophylactic use against infection from the virus. Vir has commenced a Phase 3 clinical study of VIR-7831 for the early treatment of COVID-19 patients who are at high risk of hospitalization; Vir plans to initiate a clinical study of VIR-7832 in the near future. Xencor is eligible to receive royalties on the net sales of approved products in the mid-single digit percent range.

● Omeros Corporation: In August, Xencor entered into a technology license agreement providing Omeros a non-exclusive license to its Xtend Fc technology, an exclusive license to apply Xtend technology to an identified antibody and options to apply Xtend technology to three additional antibodies. Omeros is responsible for all development and commercialization activities for all target candidates. Pursuant to these licenses, the Company received an upfront payment of $5.0 million and is eligible to receive development, regulatory and sales milestone payments for each product incorporating the selected antibodies. In addition, the Company is eligible to receive a royalty in the mid-single digit percent range on net sales of commercialized products.

Monjuvi is a registered trademark of MorphoSys AG. Ultomiris is a registered trademark of Alexion Pharmaceuticals, Inc.

Third Quarter Ended September 30, 2020 Financial Results

Cash, cash equivalents and marketable and equity securities totaled $582.9 million at September 30, 2020, compared to $601.3 million at December 31, 2019. The decrease reflects cash used to fund operating activities in the first nine months of 2020, offset by total proceeds of $89.1 million received in upfront payments, milestone payments and royalties from licensing agreements.

Total revenue for the third quarter ended September 30, 2020 was $35.4 million, compared to $21.8 million for the same period in 2019. Revenues in the third quarter included milestone revenue from MorphoSys, licensing revenue from Omeros and royalty revenue from Alexion, compared to revenues from the same period in 2019, which primarily reflects milestone revenue from the Alexion, Amgen and Novartis collaborations. Total revenue for the nine months ended September 30, 2020 was $80.8 million, compared to $153.2 million for the same period in 2019. Revenues for the nine-month period in 2020 include royalty revenue from Alexion, milestone revenue from MorphoSys, and licensing revenue from Gilead, Aimmune and Omeros, compared to licensing and collaboration revenue from Genentech and Astellas and milestone revenue from the Alexion, Amgen and Novartis collaborations in 2019.

Research and development expenditures for the third quarter ended September 30, 2020 were $44.5 million, compared to $29.8 million for the same period in 2019. Total research and development expenses for the nine months ended September 30, 2020 were $121.9 million, compared to $91.3 million for the same period in 2019. Additional spending on research and development expenses for the third quarter and first nine months of 2020 over amounts for the same periods in 2019 is primarily due to increased spending on clinical programs, including plamotamab and XmAb20717, and the IL-2-Fc cytokine development program, XmAb27564.

General and administrative expenses for the third quarter ended September 30, 2020 were $7.6 million, compared to $6.3 million in the same period in 2019. Total general and administrative expenses for the nine months ended September 30, 2020 were $22.1 million, compared to $17.5 million for the same period in 2019. Additional spending on general and administrative expenses for the third quarter and first nine months of 2020 over amounts for the same periods in 2019 is primarily due to increased compensation costs related to additional general and administrative staffing and spending on intellectual property, including patents and licensing costs.

Non-cash, stock-based compensation expense for the nine months ended September 30, 2020 was $23.1 million, compared to $ 24.7 million for same period in 2019.

Net loss for the third quarter ended September 30, 2020 was $12.6 million, or $(0.22) on a fully diluted per share basis, compared to net loss of $10.2 million, or $(0.18) on a fully diluted per share basis, for the same period in 2019. The higher net loss reported for third quarter of 2020 compared to the same period in 2019 is primarily due to increased research and development spending over increased revenue earned during the period. For the nine months ended September 30, 2020, net loss was $55.6 million, or $(0.97) on a fully diluted per share basis, compared to net income of $53.8 million, or $0.92 on a fully diluted per share basis, for the same period in 2019. The net loss reported for the nine months ended September 30, 2020 compared to net income reported for the same period in 2019 is primarily due to higher

collaboration and licensing revenue reported in 2019 compared to 2020 and increased spending on research and development programs in 2020 over 2019 amounts.

The total shares outstanding were 57,374,937 as of September 30, 2020, compared to 56,714,788 as of September 30, 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 2020 with between $525 million and $575 million in cash, cash equivalents and marketable and equity securities.

Conference Call and Webcast

Xencor will host a conference call today at 4:30 p.m. EST (1:30 p.m. PST) to discuss these third quarter 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 7307079. A live webcast of the conference call will be available online from the Investors section of Xencor’s website at www.xencor.com. The webcast will be archived on Xencor’s website for 30 days.

On November 5, 2020 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today reported financial results for the third quarter ended September 30, 2020, including sales of TAVALISSE (fostamatinib disodium hexahydrate) tablets, for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment (Press release, Rigel, NOV 5, 2020, View Source [SID1234570047]).

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"Our team has done an excellent job advancing our key value drivers while adapting to the widespread changes in the current global environment," said Raul Rodriguez, Rigel’s president and CEO. "We have continued to grow our TAVALISSE franchise with third quarter sales increasing 39% year-over-year and our global Phase 3 clinical trial for warm AIHA having enrolled over 60% of our patient goal. Additionally, exploration of fostamatinib’s potential in COVID-19 is rapidly expanding with our Phase 3 clinical trial launching this quarter and enrollment ongoing in the Phase 2 trials sponsored by the NIH/NHLBI and Imperial College London."

Business Update

Rigel’s FORWARD study, a Phase 3 pivotal trial of TAVALISSE in warm autoimmune hemolytic anemia (AIHA), has enrolled 57 of the 90 patients targeted for enrollment. The trial currently has over 90 clinical trial sites established across 22 countries.

Rigel plans to launch a Phase 3 clinical trial to evaluate the safety and efficacy of fostamatinib in hospitalized COVID-19 patients without respiratory failure that have certain high-risk prognostic factors. This multi-center, double-blind, placebo-controlled, adaptive design study is expected to enroll over 300 evaluable patients that will be randomly assigned to either fostamatinib plus standard of care (SOC) or matched placebo plus SOC (1:1). Treatment will be administered orally twice daily for 14 days. There will be a follow-up period to day 60. The primary endpoint of this study is the proportion of subjects who progress to severe/critical disease within 29 days.

The Phase 2 clinical trial sponsored by the NIH/NHLBI, in collaboration with Inova Health System, to evaluate the safety of fostamatinib for the treatment of hospitalized COVID-19 patients has enrolled 9 patients. This multi-center, double-blind, placebo-controlled study will randomly assign fostamatinib plus SOC or matched placebo plus SOC (1:1) to approximately 60 evaluable patients. Treatment will be administered orally twice daily for 14 days. There will be a follow-up period to day 60. The primary endpoint of this study is cumulative incidence of serious adverse events (SAE) through day 29. The trial also includes multiple secondary endpoints designed to assess the early efficacy and clinically relevant endpoints of disease course.

The Imperial College London-sponsored Phase 2 clinical trial to evaluate the efficacy of fostamatinib for the treatment of COVID-19 pneumonia has begun enrolling patients. The study is a two-stage, open label, controlled clinical trial with patients randomized (1:1:1) to fostamatinib plus SOC, ruxolitinib plus SOC, or SOC. Treatment will be administered twice daily for 14 days and patients will receive a follow-up assessment at day 14 and day 28 after the first dose. The primary endpoint of this study is progression from mild to severe COVID-19 pneumonia within 14 days in hospitalized patients.

Rigel recently launched FORTE, an observational study to further evaluate TAVALISSE as a second-line treatment for adult chronic ITP. The study goal is to generate additional data on patient quality of life and financial expenditures relative to the healthcare of ITP patients. Along with the post-hoc data analysis of its Phase 3 clinical program, Rigel plans to use this study to further increase and enhance its database of TAVALISSE in early line treatment of adult chronic ITP patients.

Financial Update

For the third quarter of 2020, Rigel reported a net loss of $14.2 million, or $0.08 per share, compared to a net loss of $11.5 million, $0.07 per share, in the same period of 2019.

In the third quarter of 2020, total revenues were $18.4 million, consisting of $16.3 million in net product sales and $2.1 million in contract revenues from collaborations for the achievement of a milestone in accordance with the amended license and collaboration agreement with Daiichi-Sankyo. Net product sales increased by 39% from $11.7 million in the third quarter of 2019. The decrease in contract revenues from collaborations in the third quarter of 2020 from $9.1 million in the same period of 2019 was due to developmental and commercial milestones from our various collaborative partners in 2019, partially offset by the milestone in the third quarter of 2020 from Daiichi-Sankyo as noted above.

Rigel reported total costs and expenses of $32.2 million in the third quarter of 2020, compared to $32.9 million in the same period of 2019. The decrease in total costs and expenses was primarily due to decreases to the timing of certain commercial-related activities due to the COVID-19 pandemic, partially offset by the increases in personnel-related costs and increased use of consultants.

For the nine months ended September 30, 2020, Rigel reported a net loss of $10.5 million, or $0.06 per share, compared to a net loss of $49.7 million, or $0.30 per share, in the same period of 2019.

Rigel reported total revenues of $90.2 million for the nine months ended September 30, 2020, compared to $43.9 million in the same period of 2019. Total revenues for the nine months ended September 30, 2020 consisted of $43.9 million in net product sales and $46.2 million in contract revenues from collaborations. The increase in contract revenues from collaborations related to revenue from the upfront fee previously received in 2019, as well as the milestone payment received from Grifols in the first quarter of 2020 upon EC approval of the MAA for fostamatinib in Europe and the $2.1 million in contract revenues for achievement of a milestone in accordance with the amended license and collaboration agreement with Daiichi-Sankyo, partially offset by the developmental and commercial milestones from our various collaborative partners in 2019.

Total costs and expenses for the nine months ended September 30, 2020 were $100.3 million, compared to $95.6 million in the same period of 2019. The increase in total costs and expenses was primarily related to increases in research and development cost for the on-going Phase 3 trial in warm AIHA, Phase 1 trial in RIP 1 inhibitor program and Phase 1 trial in IRAK 1/4 inhibitor program, partially offset by decreases in stock-based compensation expense and various third-party costs.

As of September 30, 2020, Rigel had cash, cash equivalents and short-term investments of $72.8 million, compared to $98.1 million as of December 31, 2019.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time

Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing 1-800-954-0603 (domestic) or 1-415-226-5355 (international). The conference call and accompanying slides will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

About ITP

In patients with ITP (immune thrombocytopenia), the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP include fatigue, excessive bruising and bleeding. People suffering with chronic ITP may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. In addition to fostamatinib, current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs) and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA

Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 45,000 adult patients in the U.S. and can be a severe, debilitating disease. Warm AIHA (wAIHA), the most common form of AIHA, is characterized by the presence of antibodies that react with the red blood cell surface at body temperature. To date, there are no disease-targeted therapies approved for AIHA, despite the unmet medical need that exists for these patients.

About Coronavirus Disease 2019 (COVID-19) & SYK-Signaling

COVID-19 is the infectious disease caused by Severe Acute Respiratory Syndrome Coronavirus-2 (SARS-CoV-2). SARS-CoV-2 primarily infects the upper and lower respiratory tract and can lead to acute respiratory distress syndrome (ARDS). Additionally, some patients develop other organ dysfunction including myocardial injury, acute kidney injury, shock resulting in endothelial dysfunction and subsequently micro and macrovascular thrombosis.1 Much of the underlying pathology of SARS-CoV-2 is thought to be secondary to a hyperinflammatory immune response associated with increased risk of thrombosis.2

Spleen tyrosine kinase (SYK) is involved in the intracellular signaling pathways of many different immune cells. Therefore, SYK inhibition may improve outcomes in patients with COVID-19 via inhibition of key Fc gamma receptor (FcγR) and c-type lectin receptor (CLR) mediated drivers of pathology, such as inflammatory cytokine release by monocytes and macrophages, production of neutrophil extracellular traps (NETs) by neutrophils, and platelet aggregation.3,4,5 Furthermore, SYK inhibition in neutrophils and platelets may lead to decreased thromboinflammation, alleviating organ dysfunction in critically ill patients with COVID-19.

About TAVALISSE

Indication

TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information

Warnings and Precautions

·Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.

·Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.

·Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.

·Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.

·TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.

Drug Interactions

·Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.

·It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.

·Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.

·Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.

Adverse Reactions

·Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).

·Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.

On November 5, 2020 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported business and financial highlights for the third quarter ended September 30, 2020 (Press release, Celldex Therapeutics, NOV 5, 2020, View Source [SID1234570046]).

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"In the third quarter, building on the compelling CDX-0159 data presented at EAACI, we initiated two Phase 1b studies in chronic spontaneous urticaria and chronic inducible urticaria which will provide the foundation for several important data readouts in 2021," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "We also advanced the first candidate from our bispecific program, CDX-527, into the clinic in solid tumors and continued to expand the ongoing CDX-1140 program, adding a combination cohort with chemotherapy in pancreatic cancer.

In addition, we are completing our work prioritizing opportunities to expand development of CDX-0159 into additional therapeutic areas. Our analysis supports that there are multiple mast cell driven indications that have the potential to be dramatically impacted by an agent that targets the root cause of the disease—the mast cell itself—and we remain on track to start a third study in a mast cell disease setting next summer. We are closing out a busy, successful year and look forward to continuing this momentum in 2021," concluded Marucci.

Recent Pipeline Highlights

While Celldex’s clinical development programs have not been significantly, negatively impacted by COVID-19 to date, the Company continues to carefully monitor the evolving situation closely across all development programs and work to minimize potential impact/disruptions.

CDX-0159—a humanized monoclonal antibody developed by Celldex that binds the KIT receptor with high specificity and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells, which mediate inflammatory responses such as hypersensitivity and allergic reactions. KIT signaling controls the differentiation, tissue recruitment, survival and activity of mast cells. Results from a Phase 1a dose escalation study of CDX-0159 were featured in a late breaking presentation in June at the EAACI Annual Congress 2020. CDX-0159 demonstrated a favorable safety profile as well as profound and durable reductions of plasma tryptase, indicative of systemic mast cell ablation.

Celldex initiated dosing in a Phase 1b multi-center study of CDX-0159 in chronic spontaneous urticaria (CSU) in October. This study is a randomized, double-blind, placebo-controlled clinical trial designed to assess the safety of multiple ascending doses of CDX-0159 in up to 40 patients with CSU who remain symptomatic despite treatment with antihistamines. Secondary and exploratory objectives include pharmacokinetic and pharmacodynamic assessments, including measurement of tryptase and stem cell factor levels and clinical activity outcomes (impact on urticaria symptoms, disease control, clinical response) as well as quality of life assessments. Results from the study are expected in the second half of 2021.

Patient screening has begun in a second Phase 1b study in chronic inducible urticaria (CIndU). This study, which is being conducted by Dr. Marcus Maurer, Professor of Dermatology and Allergy and Director of Research at the Department of Dermatology and Allergy at the Allergie-Centrum-Charité of the Charité – Universitätsmedizin in Berlin, is exploring cold-induced urticaria and symptomatic dermographism (scratch-induced urticaria). The initiation of the study was slowed by the need to incorporate, align and obtain approval on COVID-19 screening and management protocols with the responsible Ethics Committee and German Health Authority. The Company plans to present data from the study at the end of the first quarter.

Celldex is also exploring additional mast cell driven diseases for potential future development, including mast cell activation syndromes, asthma, allergic conditions and mast cell driven gastrointestinal disorders.
CDX-1140—a potent CD40 human agonist antibody developed by Celldex that the Company believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile.

In the Phase 1 dose escalation study of CDX-1140 in patients with recurrent, locally advanced or metastatic solid tumors and B cell lymphomas, both the monotherapy and combination with CDX-301 dose escalation portions of the trial are complete with an identified maximum tolerated dose (MTD) and recommended dose of CDX-1140 at 1.5 mg/kg—one of the highest systemic dose levels in the CD40 agonist class. Expansion cohorts are actively recruiting including:

CDX-1140 with KEYTRUDA (pembrolizumab) in patients who have progressed on checkpoint therapy; and,

A combination of CDX-1140 with standard of care chemotherapy in first line metastatic pancreatic cancer.
Updated interim data from the ongoing Phase 1 study has been accepted for poster presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 35th Anniversary Annual Meeting & Pre-Conference Programs (SITC 2020) and will include updated data from the monotherapy and CDX-301 combination cohorts focusing on the MTD level (1.5 mg/kg), as well as preliminary safety data from the combination cohort with pembrolizumab.
CDX-527—the first candidate developed by Celldex from its bispecific platform which utilizes the Company’s proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway.

In August, Celldex initiated a Phase 1 dose escalation study in up to ~90 patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy to be followed by tumor-specific expansion cohorts. The study is designed to determine the MTD during a dose escalation phase and to recommend a dose level for further study in the subsequent expansion phase. The expansion is designed to further evaluate the tolerability, and biologic and anti-tumor effects of selected dose level(s) of CDX-527 in specific tumor types. Initial data from the Phase 1 study are anticipated in the first half of 2021.

The CDX-527 Phase 1 study design will be presented as a clinical trial in progress poster at SITC (Free SITC Whitepaper) 2020.
In addition to the CDX-1140 and CDX-527 presentations, the Company will also present preclinical data from its Axl discovery program at SITC (Free SITC Whitepaper) 2020. Axl is a member of the TAM (Tyro3/Axl/MerTK) family of receptor tyrosine kinases and a negative regulator of innate immunity.

Third Quarter 2020 Financial Highlights and 2020 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2020 were $199.6 million compared to $206.9 million as of June 30, 2020. The decrease was primarily driven by third quarter cash used in operating activities of $11.9 million, partially offset by net proceeds of $4.3 million from sales of common stock under Celldex’s Controlled Equity OfferingSM agreement with Cantor. At September 30, 2020, Celldex had 39.6 million shares outstanding.

Revenues: Total revenue was $0.7 million in the third quarter of 2020 and $3.6 million for the nine months ended September 30, 2020, compared to $0.5 million and $2.7 million for the comparable periods in 2019. The increase in revenue was primarily due to the $1.8 million milestone payment from Rockefeller University related to Celldex’s manufacturing and development services agreement, partially offset by a decrease in services performed under the Company’s manufacturing and research and development agreement with Duke University.

R&D Expenses: Research and development (R&D) expenses were $10.7 million in the third quarter of 2020 and $32.1 million for the nine months ended September 30, 2020, compared to $11.1 million and $32.3 million for the comparable periods in 2019. The decrease in R&D expense was primarily due to a decrease in contract research and stock-based compensation expenses, partially offset by an increase in clinical trials and contract manufacturing expenses.

G&A Expenses: General and administrative (G&A) expenses were $3.6 million in the third quarter of 2020 and $10.8 million for the nine months ended September 30, 2020, compared to $3.4 million and $12.2 million for the comparable periods in 2019. The decrease in G&A expenses was primarily due to a decrease in stock-based compensation and facility expenses.

Intangible Asset Impairment: The $3.5 million non-cash impairment charge recorded during the second quarter of 2020 was due to the discontinuation of the CDX-3379 program.

Changes in Fair Value Remeasurement of Contingent Consideration: The $0.7 million loss on fair value remeasurement of contingent consideration recorded during the third quarter of 2020 and the $4.2 million gain on fair value remeasurement of contingent consideration recorded during the nine months ended September 30, 2020 were primarily due to updated assumptions for CDX-3379 related milestones due to the discontinuation of the CDX-3379 program in the second quarter of 2020, changes in discount rates and the passage of time.

Net Loss: Net loss was $14.2 million, or ($0.36) per share, for the third quarter of 2020, and $37.9 million, or ($1.44) per share, for the nine months ended September 30, 2020, compared to a net loss of $11.4 million, or ($0.75) per share, for the third quarter of 2019 and $40.4 million, or ($2.92) per share, for the nine months ended September 30, 2019.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at September 30, 2020 are sufficient to meet estimated working capital requirements and fund planned operations through 2023.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ USA.

On November 5, 2020 Navidea Biopharmaceuticals, Inc. (NYSE American: NAVB) ("Navidea" or the "Company"), a company focused on the development of precision immunodiagnostic agents and immunotherapeutics, reported it will host a conference call and webcast on Thursday, November 12, 2020 at 5:00 p.m. (EST) to discuss financial results and corporate developments for the third quarter ended September 30, 2020 (Press release, Navidea Biopharmaceuticals, NOV 5, 2020, View Source [SID1234570045]).

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Jed Latkin, Chief Executive Officer, Dr. Michael Rosol, Chief Medical Officer, and Erika Eves, Senior Director of Finance and Administration, will host the call and webcast to discuss the financial results and provide an update on recent developments and clinical progress. Management will be available to answer questions live immediately following the earnings announcement and prepared remarks portion of the call.

To participate in the call and webcast, please refer to the information below:

Event: Q3 2020 Earnings and Business Update Conference Call

Date: Thursday, November 12, 2020

Time: 5:00 p.m. (EST)

Webcast Link: View Source

A live audio webcast of the conference call will also be available on the investor relations page of Navidea’s corporate website at www.navidea.com. In addition, the recorded conference call can be replayed and will be available for 90 days following the call on Navidea’s website.