1stOncology™: Cancer Intelligence Service – Page 9945 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Marker Therapeutics Reports Third Quarter 2020 Operating and Financial Results

On November 9, 2020 Marker Therapeutics, Inc. (Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported financial results for the third quarter ended September 30, 2020 (Press release, TapImmune, NOV 9, 2020, View Source [SID1234570386]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

"This quarter, our Company reached a significant milestone by initiating our first Marker-sponsored study—a Phase 2 trial of zelenoleucel or MT-401, our lead MultiTAA-specific T cell product candidate for the treatment of post-transplant acute myeloid leukemia," said Peter L. Hoang, President & CEO of Marker Therapeutics. "We have enrolled the first patient in the safety lead-in portion of the trial, and are in the process of scheduling the donor in order to manufacture the product."

Continued Mr. Hoang: "During this unprecedented time, we have made significant progress by enrolling additional clinical sites for our AML trial, advancing our manufacturing process, reducing production time by 50% and improving the potency of our MT-401 product, and entering the final phase of the construction of our new in-house cGMP manufacturing facility. I am extremely proud of the dedication and resolve that our team has shown during these challenging months. I want to acknowledge their hard work across the organization that went into achieving these milestones."

PROGRAM UPDATES

MT-401: Multi-Antigen Targeted (MultiTAA)-Specific T Cell Product Candidate for AML

Phase 2 AML Trial
The Company initiated the safety lead-in portion of its Phase 2 study of zelenoleucel (MT-401) in patients with acute myeloid leukemia (AML) following an allogeneic stem cell transplant in both the adjuvant and active disease settings. The Company anticipates treating the first patient by Q1 2021. The safety lead-in is expected to enroll a total of six patients: three of which will be treated with MT-401 manufactured with a legacy reagent, and the remaining three to be treated with study drug manufactured with a new reagent from an alternate supplier.

Marker has activated four clinical sites and is in the start-up phase with additional clinical sites to enroll patients for the safety lead-in portion of the AML trial. The Company has also received commitments from additional clinical sites to participate in the Phase 2 AML trial following the safety lead-in phase and anticipates activating a total of approximately 20 sites.

The study remains on partial clinical hold pending the review of final data and subsequent acceptance of certificates of analysis for the new reagent by the U.S. FDA. The Company received the remaining reagent from the alternate supplier in Q3 2020 and is currently conducting the comparability analyses between the previous and new reagents, as required by FDA. Marker intends to submit all required data to FDA by Q1 2021 to enable removal of the partial clinical hold.

Over the past year, the Company has continued to streamline and simplify the MT-401 manufacturing process. The technical improvements include a 50% reduction in manufacturing time, a 95% reduction in the number of required operator interventions, and significant improvement in the consistency and reproducibility of the manufacturing process, while yielding a significant increase in the number of T cells available for patient administration. The Company expects the new process to yield a measurably improved product, with superior T cell phenotype and antigen specificity as compared to the original process. The new process improvements have been updated in the CMC section of the IND and will be used for all patients in the Marker AML clinical trial.

BUSINESS UPDATES

Construction of the cGMP manufacturing facility has entered its final phase. The facility, located in Houston near the George Bush Intercontinental Airport, will be used to support the manufacture of study drug for Marker’s Phase 2 AML trial and for future hematological and solid tumor trials, in addition to the potential commercialization of any approved products. The construction is expected to be completed by year-end with clinical activities to be initiated in the first half of 2021.
THIRD QUARTER 2020 FINANCIAL RESULTS

Cash Position and Guidance: At September 30, 2020, Marker had cash and cash equivalents of $27.0 million. The Company raised $2.2 million through the previously executed $30 million common stock purchase agreement with Aspire Capital Fund, LLC. The remaining $27.8 million available to Marker from Aspire Capital along with current cash available, funds operations into Q1 2022.

R&D Expenses: Research and development expenses were $4.8 million for the quarter ended September 30, 2020, compared to $3.1 million for the quarter ended September 30, 2019.

G&A Expenses: General and administrative expenses were $2.6 million for the quarter ended September 30, 2020, compared to $2.5 million for the quarter ended September 30, 2019.

Net Loss: Marker reported a net loss of $7.4 million for the quarter ended September 30, 2020, compared to a net loss of $5.5 million for the quarter ended September 30, 2019.

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its third quarter 2020 financial results and provide a corporate update today at 5:00 PM EST.

The webcast will be accessible in the Investors section of the Company’s website at markertherapeutics.com. Individuals can participate in the conference call by dialing 877-407-8913 (domestic) or 201-689-8201 (international) and referring to the "Marker Therapeutics Third Quarter 2020 Earnings Call."

The archived webcast will be available for replay on the Marker website following the event.

ChemoCentryx Reports Third Quarter 2020 Financial Results and Recent Highlights

On November 9, 2020 ChemoCentryx, Inc., (Nasdaq: CCXI), reported financial results for the third quarter ended September 30, 2020 and provided an overview of the Company’s recent corporate highlights (Press release, ChemoCentryx, NOV 9, 2020, View Source [SID1234570385]).

"A powerful current of progress propels our enterprise, as exemplified by the recent FDA acceptance for review of our NDA for avacopan for the treatment of ANCA-associated vasculitis," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "We ready ourselves for the bright prospect of changing the treatment paradigm in ANCA vasculitis, while also pursuing the rest of our avacopan pipeline-in-a-drug strategy. To that end, we expect to announce topline data from avacopan in C3G by the end of this year, and we are also on track to start clinical development of avacopan in lupus nephritis and initiate a pivotal Phase III trial of avacopan in patients with severe hidradenitis suppurativa in 2021. Beyond that — let’s not forget — our orally-administered small molecule checkpoint inhibitor CCX559, designed to be a next generation cancer treatment, is also slated to enter the clinic in the first half of 2021."

Dr. Schall continued, "The marked progress that we have generated in recent quarters at ChemoCentryx continues to drive us toward our goal of becoming a fully-integrated company that provides new kinds of therapies for the most critical diseases. History shows that our unique discovery and development platform has generated not just avacopan but multiple drug candidates for the highest need indications. We will always follow where the science leads, continuing to focus on the areas of the greatest need and greatest promise for patients, and also with the greatest potential reward for our shareholders."

Key Highlights

In September, the FDA accepted for review the Company’s New Drug Application (NDA) for avacopan in the treatment of ANCA-associated vasculitis and set July 7, 2021 as the Prescription Drug User Fee Act (PDUFA) goal date. The NDA included data from the global, Phase III ADVOCATE trial.
In November, the Company’s Kidney Health Alliance partner Vifor Fresenius Medical Care Renal Pharma announced that their marketing authorization application (MAA) for avacopan in the treatment of ANCA-associated vasculitis has been validated by the European Medicines Agency (EMA). Approval is expected in the second half of 2021.
The results of the Phase III ADVOCATE trial of avacopan for the treatment of ANCA-Associated Vasculitis were shared in oral presentations at Kidney Week 2020, the annual meeting of the American Society of Nephrology (ASN), and ACR Convergence 2020, the annual meeting of the American College of Rheumatology (ACR).
In October, the Company announced topline data from the randomized, double-blind, placebo-controlled, multi-center Phase II AURORA clinical trial of avacopan for the treatment of the chronic disabling skin disease Hidradenitis Suppurativa (HS) in patients with moderate or severe disease. Avacopan at 30 mg BID demonstrated a statistically significant higher response than placebo in the pre-specified Hurley Stage III (severe) HS patients and the Company plans to advance avacopan into Phase III development for the treatment of severe HS.
The Company expects to announce topline data from the ACCOLADE trial of avacopan for patients with C3 Glomerulopathy (C3G) by year end 2020.
The Company remains on track to initiate clinical studies of avacopan in lupus nephritis and its orally administered checkpoint inhibitor, CCX559, for cancer in the first half of 2021.
Third Quarter 2020 Financial Results

Revenue was $5.1 million for the third quarter of 2020, compared to $10.6 million for the same period in 2019. Revenue is recognized based on actual costs incurred as a percentage of total budgeted costs as the Company completes its performance obligations under its alliance agreements. The decrease from 2019 to 2020 was primarily attributable to lower costs incurred in 2020 due to the completion of the avacopan ADVOCATE Phase III pivotal trial.

Research and development expenses were $18.6 million for the third quarter of 2020, compared to $18.1 million for the same period in 2019. The increase from 2019 to 2020 was primarily attributable to professional fees associated with the preparation of the NDA submission for avacopan for the treatment of ANCA vasculitis and higher research and drug discovery expenses, including those tied to the advancement of CCX559, the Company’s orally administered checkpoint inhibitor. These increases were partially offset by lower expenses due to the completion of the avacopan ADVOCATE Phase III pivotal trial and the CCX140 LUMINA-1 Phase II clinical trial in 2019.

General and administrative expenses were $10.4 million for the third quarter of 2020, compared to $6.1 million for the same period in 2019. The increase from 2019 to 2020 was primarily due to higher employee-related expenses, including those associated with our commercialization planning efforts, and higher professional fees.

Net loss for the third quarter of 2020 was $24.1 million, compared to net loss of $12.9 million for the same period in 2019.

Total shares outstanding at September 30, 2020 were approximately 69.1 million shares.

Cash, cash equivalents and investments totaled $485.8 million at September 30, 2020 and the Company projects to end 2020 with cash and investments in excess of $460 million.

Conference Call and Webcast

The Company will host a conference call and webcast today, November 9, 2020 at 5:00 p.m. Eastern Time / 2:00 p.m. Pacific Time. To participate by telephone, please dial (877) 303-8028 (Domestic) or (760) 536-5167 (International). The conference ID number is 7147318. A live and archived audio webcast can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. The archived webcast will remain available on the Company’s website for fourteen (14) days following the conference call.

TG Therapeutics Provides Business Update and Reports Third Quarter 2020 Financial Results

On November 9, 2020 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the third quarter ended September 30, 2020 and recent company developments (Press release, TG Therapeutics, NOV 9, 2020, View Source [SID1234570384]).

Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "This has been a very exciting few months for TG especially sharing the first ever data from the UNITY-CLL Phase 3 trial last week showing that the trial met its primary endpoint of improvement in progression-free survival, as well as data from the UNITY-NHL trial which supported our NDA submission for umbralisib monotherapy. These data sets add to the growing body of evidence suggesting that umbralisib has a differentiated safety profile and support our long-term vision of U2 as a potential backbone for future combination therapies." Mr. Weiss continued, "With PDUFA goal dates in February 2021 and June 2021 now set for our umbralisib NDA for the treatment of relapsed/refractory MZL and FL, respectively, our team is hard at work ensuring we are prepared for a successful launch in these indications. With a healthy balance sheet which includes a proforma cash position of approximately $325 million as of September 30, 2020, we are focused on preparing a BLA/NDA submission for U2 in CLL and importantly delivering on our remaining milestones for the year including topline results from our ULTIMATE I & II Phase 3 trials of ublituximab in MS."

Recent Developments and Highlights

ASH 2020 Presentations:
• Four abstracts have been accepted for presentation at the upcoming 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, to be held virtually December 5 – 8, 2020, including:
• Results from the UNITY-NHL Phase 2 marginal zone lymphoma (MZL) and follicular lymphoma (FL) umbralisib monotherapy cohorts
• Results from the UNITY-CLL Phase 3 trial evaluating the combination of umbralisib and ublituximab (U2) in patients with treatment naïve and relapsed/refractory chronic lymphocytic leukemia (CLL)
• Two triple therapy presentations, one evaluating the combination of U2 plus venetoclax in CLL, and a another evaluating the combination of U2 plus TG-1701, our BTK inhibitor, in patients with B-cell malignancies
• Abstracts were made available last week and a call was held with leading investigators from our trials to review the data included in these abstracts. A replay from this call is available on our corporate website at View Source

UNITY-NHL: Umbralisib Monotherapy Marginal Zone Lymphoma & Follicular Lymphoma Cohorts
• In July 2020, we announced the publication of preclinical data describing the unique immunomodulatory effects of umbralisib in Blood Advances, a Journal of the American Society of Hematology (ASH) (Free ASH Whitepaper).
• In August 2020, the U.S Food and Drug Administration (FDA) accepted the Company’s New Drug Application (NDA) for umbralisib as a treatment for patients with previously treated MZL and FL. The NDA was based primarily on data from the umbralisib monotherapy MZL and FL cohorts of the UNITY-NHL Phase 2b trial. The MZL indication, under Breakthrough Therapy Designation (BTD), has been accepted for Priority Review and has a Prescription Drug User Fee Act (PDUFA) goal date of February 15, 2021. The FL indication has been accepted for standard review with a PDUFA goal date of June 15, 2021.

UNITY-CLL: Ublituximab and Umbralisib (U2) in Chronic Lymphocytic Leukemia
• In October, the FDA granted Fast Track Designation to the combination of ublituximab and umbralisib (U2) for the treatment of adult patients with CLL, which could potentially expedite the development and regulatory review of U2. The application for Fast Track was based on data from the UNITY-CLL Phase 3 Study.

Key Objectives for Remainder of 2020 and Early 2021

Report topline results from the Phase 3 ULTIMATE I & II trials in Multiple Sclerosis
Present full data from the UNITY-CLL Phase 3 trial and from the FL and MZL single agent umbralisib cohorts of the UNITY-NHL trial at ASH (Free ASH Whitepaper) 2020 as well as data from our triple therapy combinations of U2 plus venetoclax and U2 plus 1701, our BTK inhibitor
Target an NDA/Biologics Licensing Application (BLA) submission of U2 for the treatment of patients with CLL (including both previously untreated and relapsed/refractory patients)
Complete enrollment in ULTRA-V Phase 2b trial
Continue to advance our early pipeline candidates including our anti-PD-L1 monoclonal antibody, cosibelimab (TG-1501), our covalently-bound Bruton’s Tyrosine Kinase (BTK) inhibitor, TG-1701, and our anti-CD47/CD19 bispecific antibody, TG-1801

Financial Results for the Three and Nine Months Ended September 30, 2020

R&D Expenses: Other research and development (R&D) expense (not including non-cash compensation) was $45.8 million and $114.8 million for the three and nine months ended September 30, 2020, respectively, compared to $56.5 million and $118.8 million for the three and nine months ended September 30, 2019, respectively. The decrease in R&D is primarily attributable to a decrease in manufacturing costs for ublituximab and umbralisib, offset by an increase in milestone payments made during the three and nine months ended September 30, 2020. We expect our R&D expenses to decrease during the remainder of 2020 as costs associated with our main pivotal clinical trials continue to decline, partially offset by expenses associated with the expected NDA/BLA filing for U2 in CLL.

G&A Expenses: Other general and administrative (G&A) expense (not including non-cash compensation) was $11.6 million and $25.4 million for the three and nine months ended September 30 2020, respectively, as compared to $2.3 million and $6.6 million for the three and nine months ended September 30, 2019, respectively. The increase in other G&A expenses is primarily due to increased personnel and other general and administrative costs, associated with preparations for a potential commercial launch. We expect G&A expenses to increase modestly during the remainder of 2020 in preparation for potential launch.

Net Loss: Net loss was $87.2 million and $191.2 million for the three and nine months ended September 30, 2020, respectively, compared to a net loss of $61.9 million and $133.3 million for the three and nine months ended September 30, 2019, respectively. The net loss for the nine months ended September 30, 2020 included approximately $15 million of one-time milestone expenses related to our license agreements. Excluding non-cash compensation, the net loss for the three and nine months ended September 30, 2020 was approximately $58.8 million and $144.4 million, respectively, compared to a net loss of $59.9 million and $127.6 million for the three and nine months ended September 30, 2019, respectively.

Cash Position and Financial Guidance: Cash and cash equivalents were $254.2 million as of September 30, 2020. Pro forma cash, cash equivalents and investment securities as of September 30, 2020 are approximately $328 million, after giving effect to $74.0 million of net proceeds from the utilization of the Company’s ATM sales facility during the fourth quarter of 2020. The Company believes its cash and cash equivalents on hand as of September 30, 2020, along with the additional capital raised in the fourth quarter of 2020, will be sufficient to fund the Company’s planned operations well into 2022.

EUSA Pharma and BeiGene Announce Acceptance of a Biologics License Application
for QARZIBA®▼ (Dinutuximab Beta) in China

On November 9, 2020 EUSA Pharma (UK) Limited and BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160) reported that the Biologics License Application (BLA) for QARZIBA▼ (dinutuximab beta) was accepted by the China National Medical Products Administration (NMPA) and granted priority review. Dinutuximab beta is a targeted immunotherapy approved by the European Medicines Agency (EMA) for the treatment of high-risk neuroblastoma in patients aged 12 months and above who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma with or without residual disease (Press release, BeiGene, NOV 9, 2020, View Source [SID1234570383]). High-risk neuroblastoma is an aggressive neoplasm and the most common childhood solid tumour that originates outside of the brain. Dinutuximab beta is listed in the first batch of New Drugs in Urgent Clinical Need Marketed Overseas by the NMPA.

"Dinutuximab beta represents an important biologic, which is already available to patients with high-risk neuroblastoma in Europe," commented Xiaobin Wu, Ph.D., General Manager of China and President of BeiGene. "For pediatric patients fighting this disease in China, we are hopeful that dinutuximab beta will soon be available as a new treatment option. Our collaboration with EUSA and the progress thus far demonstrate our joint commitment to bringing high-quality therapies to the people who need them."

Lee Morley, Chief Executive Officer of EUSA Pharma, said, "This milestone brings us and BeiGene closer to delivering on our promise of bringing innovative cancer and rare disease therapies to patients around the world. We look forward to working with BeiGene and the NMPA to potentially make dinutuximab beta available in China."

About QARZIBA▼ (dinutuximab beta)

QARZIBA▼ is a monoclonal antibody that is specifically directed against the carbohydrate moiety of disialoganglioside 2 (GD2), which is overexpressed on neuroblastoma cells. Dinutuximab beta was approved by the European Commission in 2017 and is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilised by other suitable measures. In patients with a history of relapsed/refractory disease and in patients who have not achieved a complete response after first line therapy, dinutuximab beta should be combined with interleukin-2 (IL-2).

ATHERSYS REPORTS THIRD QUARTER 2020 RESULTS AND PROVIDES CORPORATE UPDATE

On November 9, 2020 Athersys, Inc. (NASDAQ: ATHX) reported its financial results for the three months ended September 30, 2020 and provided a corporate update (Press release, Athersys, NOV 9, 2020, View Source [SID1234570382]).

Highlights of the third quarter of 2020 and recent events include:

Received Regenerative Medicine Advanced Therapy (RMAT) designation for MultiStem from the U.S. Food and Drug Administration (FDA) for the treatment of acute respiratory distress syndrome (ARDS), a designation enabling additional interactions with the FDA that are focused on expediting development;
Commenced patient screening for the Phase 2 clinical trial led by The University of Texas Health Science Center at Houston (UTHealth) evaluating MultiStem Administration for Trauma Related Inflammation and Complications (MATRICS-1) in patients at Memorial Hermann-Texas Medical Center, a leading Level 1 Trauma center;
Further advanced the MACOVIA Phase 2/3 trial evaluating MultiStem administration to patients with COVID-19 induced ARDS and advanced preparations to potentially expand the study to include a broader range of patients with ARDS, including from influenza and other pathogens;
Advanced the MASTERS-2 ischemic stroke study, reactivating all clinical sites previously impacted by COVID-19 operational disruptions, and adding new sites to the study;
Received notification that HEALIOS K.K. (Healios), our Japanese partner, completed enrollment of its COVID-19 induced ARDS patient cohort in its ONE-BRIDGE study. Healios has previously stated it intends to complete enrollment of the entire ONE-BRIDGE study and the TREASURE ischemic stroke study by around the end of the year;
Advanced our partnering negotiations regarding MultiStem for potential commercialization in Europe and other regions of interest;
Continued key initiatives for establishing commercialization readiness, including supply chain and logistics, process development, manufacturing, branding and other key areas;
Recognized net loss of $22.5 million, or $0.11 net loss per share, for the quarter ended September 30, 2020; and
Ended the third quarter with $61.7 million of cash and cash equivalents.
"Despite the continuing chaos created by the ongoing COVID-19 pandemic, we made excellent progress this quarter. Our clinical programs and ongoing partnering negotiations continue to advance, and our efforts to establish commercial readiness in several key areas have also made good progress," commented Dr. Gil Van Bokkelen, Chairman and CEO of Athersys. "We have maintained a strong financial position while also continuing to build key capabilities that would support our planned transition into commercialization following regulatory approval of our innovative therapies.

"Completing a high value partnership for Europe and potentially other geographies is an important near-term priority, and we are focused on achieving that goal, which will be a key event in the continued evolution of the company," concluded Dr. Van Bokkelen.

Third Quarter Results

There were revenues of $0.1 million for the three months ended September 30, 2020 compared to revenues of negative $0.4 million for the three months ended September 30, 2019, primarily related to our collaboration with Healios. Our collaboration revenues currently fluctuate from period to period based on the delivery of goods and services under our arrangement with Healios.

Research and development expenses increased to $18.5 million for the three months ended September 30, 2020 from $8.9 million for the comparable period in 2019. The $9.6 million net increase is primarily associated with increases in clinical trial and manufacturing process development costs of $6.9 million, research supplies of $1.4 million, and personnel and stock compensation costs of $0.9 million. Our clinical development, clinical manufacturing and manufacturing process development expenses vary over time based on the timing and stage of clinical trials underway, manufacturing campaigns for clinical trials and manufacturing process development projects.

General and administrative expenses increased to $3.7 million for the three months ended September 30, 2020 compared to $3.0 million in the comparable period in 2019. The $0.7 million increase was primarily due to increased personnel and stock compensation costs, outside services and consulting costs.

Net loss for the third quarter of 2020 was $22.5 million compared to a net loss of $12.0 million in the third quarter of 2019. The difference primarily results from the above variances.

During the nine months ended September 30, 2020, net cash used in operating activities was $44.5 million compared to $25.2 million in the nine months ended September 30, 2019. At September 30, 2020, we had $61.7 million in cash and cash equivalents, compared to $35.0 million at December 31, 2019.

Conference Call

Gil Van Bokkelen, Chairman and Chief Executive Officer, Ivor Macleod, Chief Financial Officer, and Karen Hunady, Director of Corporate Communications and Investor Relations will host a conference call today to review the results as follows:

Date

November 9, 2020

Time

4:30 p.m. (Eastern Time)

Live webcast registration

Webcast link

Phone registration

View Source

We encourage shareholders to listen using the webcast link above. If you would like to dial in using the phone to ask a question, please register for the conference call ahead of time using the call registration link above. Once registered, you will receive the toll-free number, a direct entry passcode and a registrant ID.