On May 12, 2021 Sysmex reported that (Press release, Sysmex, MAY 12, 2021, View Source [SID1234580139])

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!

1. Consolidated Results for the Year Ended March 31, 2021
(2) Financial condition
(3) Cash flows

2. Dividend

3. Financial Forecast for the Year Ending March 31, 2022

4. Other Information

(1) Changes in significant consolidated subsidiaries (which resulted in changes in scope of consolidation):
No (2) Changes in accounting policies and accounting estimates

1) Changes in accounting policies required by IFRS:

No 2) Other changes in accounting policies:

No 3) Changes in accounting estimates:

No (3) Number of outstanding stock (common stock)

1) Number of outstanding stock at the end of each fiscal period (including treasury stock): 209,443,232 shares as of Mar. 31, 2021; 209,266,432 shares as of Mar. 31, 2020 2) Number of treasury stock at the end of each fiscal period: 446,876 shares as of Mar. 31, 2021; 446,680 shares as of Mar. 31, 2020 3) Average number of outstanding stock for each period (cumulative): 208,905,283 shares for the year ended Mar. 31, 2021 208,755,623 shares for the year ended Mar. 31, 2020

1. Overview of operating performance

1) Operating performance during the year Future-related information contained in the text below is based on the judgement as of the end of the fiscal period under review. During the fiscal year ended March 31, 2021, economic activity in Japan was in dire straits as a result of the COVID-19 pandemic, which sharply reduced personal consumption and caused corporate earnings to deteriorate. Thereafter, the situation began to improve due to gradual increases in the level of socioeconomic activity and the results of various government policies.

However, this was followed by a resurgence in infections and state of emergency declarations, and the outlook remains unclear. Overseas, increasingly serious COVID-19 outbreaks have prompted major cities to impose lockdowns and regulations against going out, substantially hampering economic activity. Thereafter, various government-imposed financial measures are leading to gradual recovery. That said, with no end to the pandemic in sight, the outlook remains opaque. On the healthcare front, Japan’s medical and healthcare field is expected to remain robust due to an aging society and increasingly diverse health and medical needs, which will drive up demand. Also, the Japanese government is positioning this field as part of an economic growth strategy.

Looking overseas, in developed countries efforts are underway to raise the efficiency of healthcare as populations age. Meanwhile, in emerging markets economic development is prompting an increase in demand for healthcare and the need for healthcare quality and service enhancement. Furthermore, the rapid application of artificial intelligence (AI), information communication technology (ICT) and other leading-edge technologies to the healthcare field are expected to sustain future growth.

The global COVID-19 pandemic has also prompted considerations about healthcare systems and the potential for major changes in the healthcare environment itself. As a result, further opportunities for growth is anticipated. Against this backdrop, Sysmex continued to develop its product portfolio in the hematology field. We launched two automated hematology analyzers in Japan, the XR Series (a next-generation flagship model) and the XQ series (a compact three-part WBC differential model). We will continue with a global sales rollout after receiving regulatory approval in individual countries.

We aim to continue expanding our product portfolio in the hematology field to help optimize operations at testing laboratories based on the regional characteristics and facilities’ needs. In the hemostasis field, we launched CN-6500/CN-3500 automated blood coagulation analyzers in Japan, which offer enhanced efficiency and quality. We renewed our global alliance in hemostasis products with Siemens Healthcare Diagnostics Inc. including review of products and territories, to step up sales of our CN Series automated blood coagulation analyzer and we will continue to offer extensive solutions to customers in regions throughout the world. In the life science field, Sysmex had received health insurance coverage for its gene mutation analysis set for cancer genome profiling (OncoGuide NCC Oncopanel System), becoming the first such system in Japan to receive this coverage. We received approval for partial changes to our manufacturing and marketing approval for this system. it has become possible to detect mutations and copy number alterations of 124 genes, fusions of 13 genes, and microsatellite instability (MSI).

The system will thus provide more detailed genetic information to help doctors decide treatment strategies, including diagnosis and selection of anti-cancer drugs. Medicaroid Corporation, a joint venture between Sysmex and Kawasaki Heavy Industry, Ltd., received manufacturing and marketing approval for hinotori Surgical Robot System, the first made-in-Japan robotic-assisted surgery system. As the global general distributor, Sysmex commenced sales of hinotori to medical institutions in Japan, starting with urology departments. We are working with Medicaroid to obtain regulatory approval overseas, and will begin introducing the system in overseas markets, as well. To help stem the increase in COVID-19 infections, we introduced reagent that can be used in conjunction with HISCL-5000/HISCL-800 automated immunoassay systems to detect the presence of coronavirus antigens that cause COVID-19, as well as reagent that can help in detecting the risk of COVID-19 cases worsening. Sysmex remains committed to contributing toward the research and establishment of diagnosis/treatment methods for COVID-19 by way of diverse testing, including PCR tests, antigen tests, antibody tests and cytokine tests, as well as existing hematology-3-and coagulation tests.

On May 12, 2021 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage biotechnology company leading the development of novel antibody drug conjugates (ADCs) to treat hematological malignancies and solid tumors, reported that results of LOTIS-2, a multicenter, open-label, single-arm Phase 2 clinical trial evaluating the safety and efficacy of single-agent ZYNLONTA in adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) following two or more systemic treatments, have been published online in The Lancet Oncology (Press release, ADC Therapeutics, MAY 12, 2021, View Source [SID1234579762]).

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!

"Patients with relapsed or refractory DLBCL who have been heavily pretreated and have difficult-to-treat disease represent an urgent area of medical need that newly approved ZYNLONTA is now able to address," said Paolo F. Caimi, MD, University Hospitals Cleveland Medical Center and Case Comprehensive Cancer Center, Case Western Reserve University and lead author of The Lancet Oncology paper. "The LOTIS-2 study established that ZYNLONTA demonstrated substantial single-agent activity and produced durable responses with an acceptable safety profile in this patient population."

LOTIS-2 enrolled 145 patients, including those with high-risk characteristics for poor prognosis, such as double-/triple-hit, transformed, and primary refractory DLBCL. Key results include:

Overall response rate (ORR) was 48.3% (70/145 patients), including a 24.1% (35/145 patients) complete response rate and 24.1% (35/145 patients) partial response (PR) rate
Median time to first response, analyzed post-hoc, was 41 days
Median duration of response was 10.3 months (as of the data cut)
Durable responses in high-risk patient groups included 46.2% (6/13 patients) ORR in those who had progression after prior CAR-T therapy, 33.3% (5/15 patients) ORR in double or triple hit and 44.8% (13/29 patients) ORR in transformed DLBCL
ZYNLONTA demonstrated an acceptable safety profile. The most common Grade ≥3 treatment-emergent adverse events were neutropenia (25.5%), thrombocytopenia (17.9%), and increased gamma-glutamyltransferase (16.6%)
"We are proud to have the results of our LOTIS-2 trial published in a prestigious peer-reviewed journal," said Jay Feingold, MD, PhD, Senior Vice President and Chief Medical Officer at ADC Therapeutics. "On the heels of the FDA approval, this further reinforces the value of ZYNLONTA as the first CD19-targeted ADC single-agent treatment for relapsed or refractory DLBCL and the potential for it to become the standard-of-care for 3L+ DLBCL patients in need of new treatment options."

The study can be found on The Lancet Oncology’s website at: http://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(21)00139-X/fulltext.

About ADC Therapeutics

ADC Therapeutics (NYSE: ADCT) is a commercial-stage biotechnology company improving the lives of cancer patients with its next-generation, targeted antibody drug conjugates (ADCs). The Company is advancing its proprietary ADC technology to transform the treatment paradigm for patients with hematologic malignancies and solid tumors.

ADC Therapeutics’ CD19-directed ADC ZYNLONTA (loncastuximab tesirine-lpyl) is approved by the FDA for the treatment of relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy. ZYNLONTA is also in late-stage clinical trials in combination with other agents. Cami (camidanlumab tesirine) is being evaluated in a late-stage clinical trial for relapsed or refractory Hodgkin lymphoma and in a Phase 1b clinical trial for various advanced solid tumors. In addition to ZYNLONTA and Cami, the Company has multiple PBD-based ADCs in ongoing clinical and preclinical development.

ADC Therapeutics is based in Lausanne (Biopôle), Switzerland and has operations in London, the San Francisco Bay Area and New Jersey. For more information, please visit View Source and follow the Company on Twitter and LinkedIn.

ZYNLONTA is a trademark of ADC Therapeutics SA.

About ZYNLONTA (loncastuximab tesirine-lpyl)

ZYNLONTA is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death.

The U.S. Food and Drug Administration (FDA) has approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. This indication is approved by the FDA under accelerated approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

The FDA approval was based on data from LOTIS-2, a large (n=145) Phase 2 multinational, single-arm clinical trial of ZYNLONTA for the treatment of adult patients with r/r DLBCL following two or more prior lines of systemic therapy. Results from the trial demonstrated an overall response rate (ORR) of 48.3% (70/145 patients), which included a complete response (CR) rate of 24.1% (35/145 patients) and a partial response (PR) rate of 24.1% (35/145 patients). Patients had a median time to response of 1.3 months and the median duration of response (mDoR) for the 70 responders was 10.3 months (inclusive of patients who were censored). In a pooled safety population the most common adverse reactions (≥20%) were thrombocytopenia, gamma-glutamyltransferase increased, neutropenia, anemia, hyperglycemia, transaminase elevation, fatigue, hypoalbuminemia, rash, edema, nausea and musculoskeletal pain. In LOTIS-2, the most common (≥10%) grade ≥3 treatment-emergent adverse events were neutropenia (26.2%), thrombocytopenia (17.9%), gamma-glutamyltransferase increased (17.2%) and anemia (10.3%).

ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy.

Important Safety Information

WARNINGS AND PRECAUTIONS

Effusion and Edema

Serious effusion and edema occurred in patients treated with ZYNLONTA. Grade 3 edema occurred in 3% (primarily peripheral edema or ascites) and Grade 3 pleural effusion occurred in 3% and Grade 3 or 4 pericardial effusion occurred in 1%.

Monitor patients for new or worsening edema or effusions. Withhold ZYNLONTA for Grade 2 or greater edema or effusion until the toxicity resolves. Consider diagnostic imaging in patients who develop symptoms of pleural effusion or pericardial effusion, such as new or worsened dyspnea, chest pain, and/or ascites such as swelling in the abdomen and bloating. Institute appropriate medical management for edema or effusions.

Myelosuppression

Treatment with ZYNLONTA can cause serious or severe myelosuppression, including neutropenia, thrombocytopenia, and anemia. Grade 3 or 4 neutropenia occurred in 32%, thrombocytopenia in 20%, and anemia in 12% of patients. Grade 4 neutropenia occurred in 21% and thrombocytopenia in 7% of patients. Febrile neutropenia occurred in 3%.

Monitor complete blood counts throughout treatment. Cytopenias may require interruption, dose reduction, or discontinuation of ZYNLONTA. Consider prophylactic granulocyte colony-stimulating factor administration as applicable.

Infections

Fatal and serious infections, including opportunistic infections, occurred in patients treated with ZYNLONTA. Grade 3 or higher infections occurred in 10% of patients, with fatal infections occurring in 2%. The most frequent Grade ≥3 infections included sepsis and pneumonia.

Monitor for any new or worsening signs or symptoms consistent with infection. For Grade 3 or 4 infection, withhold ZYNLONTA until infection has resolved.

Cutaneous Reactions

Serious cutaneous reactions occurred in patients treated with ZYNLONTA. Grade 3 cutaneous reactions occurred in 4% and included photosensitivity reaction, rash (including exfoliative and maculo-papular), and erythema.

Monitor patients for new or worsening cutaneous reactions, including photosensitivity reactions. Withhold ZYNLONTA for severe (Grade 3) cutaneous reactions until resolution. Advise patients to minimize or avoid exposure to direct natural or artificial sunlight including exposure through glass windows. Instruct patients to protect skin from exposure to sunlight by wearing sun-protective clothing and/or the use of sunscreen products. If a skin reaction or rash develops, dermatologic consultation should be considered.

Embryo-Fetal Toxicity

Based on its mechanism of action, ZYNLONTA can cause embryo-fetal harm when administered to a pregnant woman because it contains a genotoxic compound (SG3199) and affects actively dividing cells.

Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with ZYNLONTA and for 9 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ZYNLONTA and for 6 months after the last dose.

ADVERSE REACTIONS

In a pooled safety population of 215 patients (Phase 1 and LOTIS-2), the most common (>20%) adverse reactions, including laboratory abnormalities, were thrombocytopenia, increased gamma-glutamyltransferase, neutropenia, anemia, hyperglycemia, transaminase elevation, fatigue, hypoalbuminemia, rash, edema, nausea, and musculoskeletal pain.

In LOTIS-2, serious adverse reactions occurred in 28% of patients receiving ZYNLONTA. The most common serious adverse reactions that occurred in ≥2% receiving ZYNLONTA were febrile neutropenia, pneumonia, edema, pleural effusion, and sepsis. Fatal adverse reactions occurred in 1%, due to infection.

Permanent treatment discontinuation due to an adverse reaction of ZYNLONTA occurred in 19% of patients. Adverse reactions resulting in permanent discontinuation of ZYNLONTA in ≥2% were gamma-glutamyltransferase increased, edema, and effusion.

Dose reductions due to an adverse reaction of ZYNLONTA occurred in 8% of patients. Adverse reactions resulting in dose reduction of ZYNLONTA in ≥4% was gamma-glutamyltransferase increased.

Dosage interruptions due to an adverse reaction occurred in 49% of patients receiving ZYNLONTA. Adverse reactions leading to interruption of ZYNLONTA in ≥5% were gamma-glutamyltransferase increased, neutropenia, thrombocytopenia, and edema.

You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to ADC Therapeutics at 1-855-690-0340.

On May 12, 2021 Delcath Systems, Inc. (NASDAQ: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported business highlights and financial results for the first quarter ended March 31, 2021 (Press release, Delcath Systems, MAY 12, 2021, View Source [SID1234579779]).

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!

Recent Business Highlights

During and since the first quarter the company:

Reported positive preliminary results from the FOCUS Clinical Trial (NCT02678572) for Patients with Hepatic Dominant Ocular Melanoma treated with HEPZATO KIT based on an analysis of currently evaluable patients. The preliminary analysis included 87% of treated patients and final results are expected later in the year.
The primary endpoint, overall response rate (ORR), exceeded the prespecified threshold for success by a large enough margin to ensure that final results based on 100% of patients will be positive.
Both prespecified ORR and Progression Free Survival comparative analyses versus the best alternative care arm demonstrated a statistically significant improvement.
The safety profile was consistent with the safety profile of CHEMOSAT treatment described in previous European single-center and multi-center publications with no new safety signals observed in this patient population and no treatment related deaths on study.
Announced that the United Kingdom’s National Institute for Health and Care Excellence, has updated its guidance for the Delcath CHEMOSAT Hepatic Delivery System for Melphalan (CHEMOSAT) in the treatment of patients with metastases in the liver from Ocular Melanoma. Under this designation, private insurance may be more likely to fund treatment with CHEMOSAT, some regional funding may be more accessible, and a process is now available to seek national reimbursement.
Announced an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting to be held virtually June 4-8, 2021.
"The recently released preliminary results from the FOCUS trial strongly indicates that HEPZATO’s benefit risk ratio is a significant improvement versus an earlier generation of Delcath’s proprietary percutaneous hepatic perfusion system," said Gerard Michel, CEO of Delcath. "We look forward to continued progress in the balance of the year, as we prepare both to file an NDA in early 2022 and expand the development of HEPZATO into additional areas of high unmet need."

First Quarter 2021 Financial Results:

Income Statement Highlights.

Product revenue for the three months ended March 31, 2021 was approximately $0.4 million, compared to $0.3 million for the prior year period from our sales of CHEMOSAT procedures in Europe. Selling, general and administrative expenses were approximately $3.3 million compared to $2.3 million in the prior year quarter. Research and development expenses for the quarter were $3.7 million compared to $3.0 million in the prior year quarter. Total operating expenses for the quarter were $7.0 million compared with $5.3 million in the prior year quarter. First quarter expenses included approximately $2.2 million of stock option expense compared to no material stock option expense in the prior year quarter.

We recorded a net loss for the three months ended March 31, 2021, of $6.8 million, compared to a net income of $7.9 million for the same period in 2020

Balance Sheet Highlights.

At March 31, 2021, we had cash, cash equivalents and restricted cash totaling $26.7 million, as compared to cash, cash equivalents and restricted cash totaling $4.7 million at March 31, 2020. During the three months ended March 31, 2021 and March 31, 2020, we used $4.6 million and $5.2 million, respectively, of cash in our operating activities.

Conference Call Information

To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call.

On May 12, 2021 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported its financial results for the quarter ended March 31, 2021 (Press release, Moleculin, MAY 12, 2021, View Source [SID1234579795]). The Company also provided an update on its portfolio of oncology drug candidates for the treatment of highly resistant tumors and viruses.

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!

"Over the course of the first quarter, we continued to make significant progress on multiple fronts. Importantly, we have equipped the Company with the resources to advance our portfolio of drug candidates across a number of oncology indications and viruses. On the clinical front, we have the potential to see up to seven clinical trials this year, including investigator sponsored trials. We believe we are well-positioned to execute our strategy and expect to continue to build momentum and drive shareholder value in the near- and long-term," commented Walter Klemp, Chairman and CEO of Moleculin.

Recent Highlights

Granted Rare Pediatric Disease Designation (RPD) from the U.S. Food and Drug Administration (FDA) to its p-STAT3 inhibitor, WP1066, for the treatment of ependymoma, increasing to a total of four different indications for which a Priority Review Voucher may be granted;
Engaged IQVIA Biotech to manage Moleculin’s effort to begin potential clinical trials of WP1122 for the treatment of COVID-19;
Received Fast Track Designation from the FDA for its drug, Annamycin, for the treatment of soft tissue sarcoma (STS) lung metastases;
Announced a $1.5 million grant was awarded to the Maria Sklodowska-Curie National Research Institute to fund an investigator-initiated Phase 1B/2 clinical trial of Annamycin for the treatment of STS lung metastases;
Successfully closed a public offering, including full exercise of over-allotment option, for gross proceeds of approximately $78.0 million, before deducting underwriting discount and offering expenses;
Announced 100% survival achieved in preclinical study in animals which demonstrated a potentially significant therapeutic benefit of Annamycin against metastatic osteosarcoma; and
Signed an agreement with Catalyst Clinical Research to manage its U.S. clinical trial to study the ability of Annamycin to treat STS that has metastasized to the lungs.
Programs Update

Next Generation Anthracycline – Annamycin

Annamycin is the Company’s "next generation" anthracycline that has recently been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin (the standard of care chemotherapy for STS lung metastases). Importantly, Annamycin has also demonstrated a lack of cardiotoxicity in recently conducted human clinical trials for the treatment of acute myeloid leukemia (AML), so we believe that the use of Annamycin may not face the same usage limitations imposed on doxorubicin. Annamycin is currently in development for the treatment of AML and STS lung metastases.

Upcoming Milestones

2H 2021: Report topline results from ongoing Phase 1/2 study for treatment of AML.
2H 2021: Commence Phase 1/2 study in Europe for the treatment of AML evaluating combination therapy of Annamycin + Ara-C.
2H 2021: Commence Phase 1b/2 clinical trial of Annamycin for the treatment of STS lung metastases in the U.S.
2021: Based on recently announced reimbursement grant awarded in Poland, the Company expects a second Phase 1b/2 clinical trial of Annamycin in sarcoma lung metastases to be primarily investigator-funded in Europe.
First-in-class p-STAT3 inhibitors – WP1066 and WP1220

WP1066 is one of several Immune/Transcription Modulators, designed to stimulate the immune response to tumors by inhibiting the errant activity of Regulatory T-Cells (TRegs) while also inhibiting key oncogenic transcription factors, including p-STAT3 (phosphorylated signal transducer and activator of transcription 3), c-Myc (a cellular signal transducer named after a homologous avian virus called Myelocytomatosis) and HIF-1α (hypoxia inducible factor 1α). These transcription factors are widely sought targets that are believed to contribute to an increase in cell survival and proliferation, and the angiogenesis (coopting vasculature for blood supply), invasion, metastasis and inflammation associated with tumors. They may also play a role in the inability of immune checkpoint inhibitors to affect more resistant tumors. WP1220 is a close analog to WP1066 that the Company has developed as a potential topical therapy for skin-related diseases.

WP1220 is currently being evaluated for the treatment of Cutaneous T-Cell Lymphoma (CTCL) and WP1066 is currently being evaluated for the treatment of pediatric brain tumors, including Diffuse Interstitial Pontine Glioma (DIPG). Additionally, WP1066 + radiation is being evaluated in the treatment of treatment of Glioblastoma Multiforme (GBM).

Upcoming Milestones

2H 2021: Facilitate Phase 1/2 study of WP1066 + radiation for the treatment of GBM.
1H 2022: Facilitate launch of Phase 2 study of WP1066 for the treatment of pediatric brain tumors, including DIPG.
Actively seeking collaboration with a strategic partner in the near term for external funding for the continued development of WP1220 in a Phase 2 clinical trial as a topical therapy for CTCL.
Infectious Disease and Metabolism/Glycosylation Inhibitors- WP1122, WP1096 and WP1097 Portfolio

Moleculin has new compounds designed to target the roles of glycolysis and glycosylation in both cancer and viral diseases. The Company’s lead Metabolism/Glycosylation Inhibitor, WP1122, is a prodrug of 2-DG that appears to improve the drug-like properties of 2-DG by increasing its circulation time and improving tissue/organ distribution. Recent published research has identified that 2-DG has antiviral potential against SARS-CoV-2 in vitro and, based on publicly available information, a recently completed Phase 2 clinical trial by an unrelated company in India has reported efficacy in COVID-19 patients, resulting in the Emergency Use Authorization of 2-DG by the Drugs Controller General of India. Moleculin believes that the improved drug-like properties of WP1122 may allow it to outperform 2-DG in COVID-19 patients and may provide the opportunity for it to become an important drug to potentiate existing therapies, including checkpoint inhibitors. Although the Company has seen superior efficacy for WP1122 over 2-DG against SARS-CoV-2 in vitro, as well as in multiple animal tumor models, WP1122 has yet to be tested in humans so there can be no assurance that this improved preclinical performance will translate into improved clinical outcomes. The Company is also engaged in preclinical development of additional antimetabolites (WP1096 and WP1097) targeting glycolysis and glycosylation.

Upcoming Milestones

2H 2021: Seek to initiate Phase 1a/1b study of WP1122 for the treatment of COVID-19.
2H 2021: Potential to launch Phase 2 pivotal study of WP1122 for the treatment of COVID-19.
2H 2021: File an IND in the U.S. for the treatment of certain cancers such as GBM and pancreatic cancer, with WP1122.
Ongoing preclinical development work in anti-viral indications such as HIV, Zika, and Dengue. IND targeted for 2022.
Summary of Financial Results for First Quarter 2021

Research and development expense was $4.1 million and $3.2 million for the three months ended March 31, 2021 and 2020, respectively. The increase of $0.9 million is mainly related to increased clinical trial activity as described above, increased costs related to sponsored research agreements, and costs related to manufacturing of additional drug product.

General and administrative expense was $1.9 million and $1.8 million for the three months ended March 31, 2021 and 2020, respectively. The increase of $0.1 million is mainly related to an increase in the Company’s insurance, which was offset by a similar decrease in travel expenses.

For the three months ended March 31, 2021 and 2020, the Company reported a net loss of $4.4 million and $1.2 million, respectively, and had net cash flows used in operating activities of $ 3.6 million and $4.3 million, respectively.

The Company ended the quarter with approximately $86.3 million of cash.

On May 12, 2021 Immunocore Holdings Plc (Nasdaq: IMCR), a late-stage biotechnology company pioneering the development of a novel class of T cell receptor (TCR) bispecific immunotherapies designed to treat a broad range of diseases, including cancer, infectious and autoimmune disease, reported its results for the quarter ended March 31, 2021 (Press release, Immunocore, MAY 12, 2021, View Source [SID1234579813]).

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!

Highlights for the quarter included the presentation of the Phase 3 randomized data from the Company’s lead candidate tebentafusp in the plenary clinical trial session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, the launch of a global early access program for tebentafusp, and the successful completion of the Company’s initial public offering resulting in net proceeds of $287 million.

Bahija Jallal, Chief Executive Officer of Immunocore, said: "Tebentafusp has been demonstrated to prolong survival in patients with metastatic uveal melanoma, a cancer that has historically proven insensitive to chemotherapy and immunotherapies. These data, recently presented at AACR (Free AACR Whitepaper), represent the first positive Phase 3 clinical trial for a TCR therapeutic and the first time that a bispecific T cell engager has demonstrated a survival benefit in a solid tumor, representing a significant breakthrough in the field of oncology."

First Quarter 2021 Highlights (including post-period)

Tebentafusp

In April, one oral presentation and three posters on tebentafusp were accepted at the 2021 American Society of Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held virtually from June 4-8, 2021. Per ASCO (Free ASCO Whitepaper)’s Embargo & Release Information, full abstracts will be released to the public on ASCO (Free ASCO Whitepaper)’s Meeting Library at 5:00 p.m. ET on May 19, 2021.

In April, the Company launched a global early access program for tebentafusp in metastatic uveal melanoma (mUM).

In April, the Company’s Phase 3 data of tebentafusp in metastatic uveal melanoma was also the subject of an oral presentation in the Phase 3 clinical trials plenary session at the AACR (Free AACR Whitepaper) Virtual Annual Meeting 2021. Tebentafusp demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) as a first-line treatment in mUM. In the intent-to-treat population, tebentafusp demonstrated a median overall survival of 21.7 months compared to 16.0 months for investigator’s choice and with 73% of patients alive at 1 year for tebentafusp vs. 58% for investigator’s choice. The OS Hazard Ratio (HR) favored tebentafusp, HR=0.51 (95% CI: 0.37, 0.71); p< 0.0001, over investigator’s choice (82% pembrolizumab; 12% ipilimumab; 6% dacarbazine). In addition, tebentafusp resulted in a statistically significant longer PFS. Treatment-related adverse events were manageable and consistent with the proposed mechanism.

In February, tebentafusp was granted Breakthrough Therapy Designation by the U.S. Food & Drug Administration (FDA) for unresectable or metastatic uveal melanoma. Additionally, the European Commission, upon recommendation of the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) awarded tebentafusp Orphan Drug Designation for the treatment of uveal melanoma. Medicines that meet the EMA’s Orphan Drug Designation criteria qualify for several incentives, including 10 years of market exclusivity, protocol assistance, and potentially reduced fees for regulatory activities.

Immunocore will be working to complete its BLA submission to the FDA in the third quarter of 2021, followed by submission of a Marketing Authorization Application to the EMA.

Additional Clinical Programs

IMC-C103C – MAGE-A4

In the first quarter, the Company continued to advance, IMC-C103C, an ImmTAC molecule targeting an HLA-A*02:01 MAGE-A4 antigen, in a first-in-human, Phase 1/2 dose escalation trial in patients with solid tumor cancers including non-small-cell lung cancer (NSCLC), gastric, head and neck, ovarian and synovial sarcoma. The Company plans to report Phase 1 initial data from this trial in the fourth quarter of 2021.

IMC-F106C – PRAME

In the first quarter, the Company continued to advance IMC-F106C, an ImmTAC molecule targeting an HLA-A*02:01 PRAME antigen, in a first-in-human, Phase 1/2 dose escalation trial in patients with multiple solid tumor cancers. PRAME is overexpressed in many solid tumors including NSCLC, SCLC, endometrial, ovarian, and breast cancers. The Company plans to report Phase 1 initial data from this trial in mid-2022.

IMC-I109V – HBV

In the first quarter, the Company continued to advance IMC-I109V, an ImmTAV molecule targeting a conserved Hepatitis B virus (HBV), envelope antigen, in a global Phase 1 single ascending dose trial. The Company plans to initiate dosing mid-year 2021.

Operational Highlights

In February, the Company made key appointments to management and Board of Directors. The appointment of Ralph Torbay as Immunocore’s new Head of Commercial and the appointment of Dr. Roy S. Herbst as a member of the Company’s Board of Directors became effective January 28, 2021. Dr. Herbst served as a member of Immunocore’s Scientific Advisory Board (SAB) and is currently Ensign Professor of Medicine (Medical Oncology), Professor of Pharmacology, Chief of Medical Oncology and Associate Cancer Center Director for Translational Research at Yale Cancer Center and Smilow Cancer Hospital.

In February, the Company completed its initial public offering (IPO) and concurrent private placement. The financing was $312.1 million in aggregate, of which approximately $287 million in net proceeds was from the IPO on Nasdaq of 11,426,280 American Depositary Shares (ADSs), including the exercise in full by the underwriters of their option to purchase an additional 1,490,384 ADSs, at an IPO price of $26.00 per ADS and $15 million from the completion of the concurrent sale of an additional 576,923 ADSs at the initial offering price of $26.00 per ADS, for gross proceeds of approximately $15.0 million, in a private placement to the Bill & Melinda Gates Foundation.

Financial Results

Basic and diluted loss per share was a £0.76 or $1.05 for the quarter ended March 31, 2021 compared to an adjusted to £0.74 for the quarter ended March 31, 2020. Total operating loss for the quarter was £31.9 million or $44.0 million compared to £22.1 million for the same period last year, largely due to an increase in employee costs associated with non-cash share-based payment charges.

For the three months ended March 31, 2021, revenue from collaboration agreements was unchanged at £8.3 million or $11.4 million compared to the same period last year. For the three months ended March 31, 2021, research and development expenses were £19.9 million or $27.4 million, as compared to £20.8 million for the three months ended March 31, 2020. For the quarter, administrative expenses were £20.2 million or $27.8 million compared to £9.6 million for the quarter ended March 31, 2020 including a £7.7 million increase in the non-cash share-based payment charges.

Cash and cash equivalents totaled £313.1 million or approximately $431 million as of March 31, 2021 compared to £68.4 million for the same period last year.