On February 25, 2021 Break Through Cancer (www.breakthroughcancer.org) reported its formal launch as a public foundation designed to find new solutions to the most intractable challenges in cancer (Press release, Break Through Cancer, FEB 25, 2021, View Source [SID1234575797]). The foundation is being launched with an extraordinary challenge pledge of $250 million from Mr. and Mrs. William H. Goodwin, Jr. and their family, and the estate of William Hunter Goodwin, III. This represents one of the largest gifts ever in support of cancer research. Led by Dr. Tyler Jacks, Founding Director of the Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology, the David H. Koch Professor of Biology and Co-director of the Ludwig Center for Molecular Oncology, Break Through Cancer will fund and support collaborative research teams drawn from several of the country’s top cancer centers.

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Multidisciplinary research teams will be selected from across five participating institutions: Dana-Farber Cancer Institute, the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, The University of Texas MD Anderson Cancer Center, Memorial Sloan Kettering Cancer Center, and the Koch Institute for Integrative Cancer Research at MIT.

Break Through Cancer will focus on historically highly challenging cancer types, including pancreatic cancer, ovarian cancer, glioblastoma, and acute myelogenous leukemia (AML), for its initial programs, aided by the guidance of a scientific advisory board of cancer experts from outside the participating institutions. Teams will receive substantial funding to bring new approaches and new thinking as rapidly as possible to the clinical challenges of cancer.

"Break Through Cancer’s model builds on the outstanding efforts of the broader cancer research community and presents the potential for major advances in our shared fight against these intractable cancers," said Dr. Jacks, President of Break Through Cancer. "Our tagline, "collaborating for cures," captures our collective goal to empower many of the brightest, most dedicated minds in cancer research and to maximize the capabilities of these highly respected institutions. In the future, we look forward to partnering with the broader philanthropic, biotech, and pharmaceutical communities to expand the impact of Break Through Cancer further still."

The organization is supported by a board that includes leaders from each of the participating institutions, with William G. Nelson, V, MD, PhD, the Marion I. Knott Professor of Oncology and Director of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, serving as Chairman.

"We owe a debt of gratitude to the Goodwin family and the late Hunter Goodwin, whose vision and generosity made this powerful collaboration possible," said Dr. Nelson, Break Through Cancer’s Chairman. "Their contributions have been key both financially and conceptually, as we worked together to create a research model that will provide a compelling advantage in the search for cures. We are fortunate to have such strong commitments from all of the parties involved."

"We realize there are no guarantees, yet we believe this effort to fight cancer, particularly with collaborative research, has a realistic probability of success," said Bill Goodwin. "We want to help people have better lives. And we sincerely hope that by being public with our support, we will inspire others to support this incredible effort."

On February 25, 2021 Charles River Laboratories International, Inc. (NYSE: CRL) reported a strategic partnership with Kibur Medical to offer exclusive access to its implantable microdevice (IMD) for in vivo preclinical oncology studies (Press release, Charles River Laboratories, FEB 25, 2021, View Source [SID1234575796]).

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Kibur’s Unique Technology

The IMD provides an opportunity to transform how researchers may assess efficacy of oncology compounds in vivo as both individual and combination therapies. Kibur’s microdevice technology can hold up to 20 different compounds for local administration, which allow it to:

Measure interactions between disease tissue and microdoses of therapies to predict the optimal drug regimen;
Rapidly test multiple combinations at once in vivo in most solid tumors​;
Dose therapies directly into disease tissue, allowing for investigative studies of early-stage compounds where pharmacokinetic properties are poorly understood.
Data-Driven Decisions

According to a study in Biostatistics, oncology drugs have a 97% failure rate in clinical trials. Together with Charles River’s leading expertise in early-stage preclinical testing, researchers can utilize the Kibur technology to perform in vivo testing of multiple doses and multiple combinations of oncology or immune-oncology therapies in small cohorts of patient-derived xenograft (PDX), cell-line derived or syngeneic models. Additionally, Kibur’s IMD provides both a platform to observe synergies of drug combinations and a time and cost-sensitive testing solution for extending indications of existing oncology drugs.

Excised tumor tissue is analyzed via multiplex immuno-histochemistry, immune cell readouts, spatial transcriptomics and MALDI-TOF mass spectrometry providing for rich datasets in short cycle times and thereby improved decision making early in the preclinical process.

Approved Quotes

"We are proud to partner with Kibur Medical to help advance our oncology service offering. The development of cancer therapies is extremely nuanced, and Kibur’s technology provides clients with important data on drug efficacy to help inform their preclinical programs." –Birgit Girshick, Corporate Executive Vice President, Discovery and Safety Assessment, Biologics Testing Solutions, and Avian Vaccine Services, Charles River
"When looking for a strategic partner, we knew we wanted an organization with global reach and a strong scientific bench. Charles River’s longstanding reputation as an industry leader will help deliver our technology to the widest possible network, providing a huge opportunity to help develop the next generation of cancer therapeutics." – Oliver Jonas, PhD, Scientific Founder, Kibur Medical

On February 25, 2021 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company") a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported its financial results and business highlights for the fourth quarter and full year ended December 31, 2020 (Press release, Cyclacel, FEB 25, 2021, View Source [SID1234575777]).

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The Company’s net loss applicable to common shareholders for the three months and year ended December 31, 2020 was $6.6 million and $12.4 million, respectively. As of December 31, 2020, cash and cash equivalents totaled $33.4 million. Additional proceeds of $4.3 million were received from the exercise of warrants after December 31, 2020. Based on current spending, the proforma cash on hand of $37.7 million provides the Company with sufficient resources to fund planned operations including research and development through early 2023.

"During 2020 we have reported on fadraciclib’s oral bioavailability and evidence of durable anticancer activity," said Spiro Rombotis, President and Chief Executive Officer. "Independent evidence supporting the rationale for dual inhibition of CDK2 and CDK9 cancer pathways, the targets of fadraciclib, were published in peer-reviewed communications. Meanwhile our team, led by Dr. Mark Kirschbaum, our newly appointed CMO, has prepared a streamlined clinical development strategy to evaluate oral fadraciclib in multi-cohort, Phase 1b/2, registration-directed, studies for both solid and liquid cancers. A similar trial design will be applied to the development of CYC140, our selective PLK1 inhibitor, supported by extensive preclinical data demonstrating CYC140’s antimitotic mechanism and broad therapeutic potential in both solid and liquid cancers. We look forward to providing further details of our clinical development plans and our progress with fadraciclib and CYC140 to drive shareholder value."

Key 2020 Highlights

Corporate

Appointed Mark Kirschbaum, M.D. as Senior Vice President and Chief Medical Officer. Dr. Kirschbaum is a highly experienced hematologist/oncologist with over 30 years of experience in molecular medicine, new drug development, clinical trial design and patient care. Most recently, Dr. Kirschbaum served as Vice President, Hematology/Oncology at ArQule Inc.

Appointed two new Directors to strengthen and broaden our Board’s skill base:

Brian Schwartz, M.D. has wide-ranging experience as a drug development expert in the biopharmaceutical industry primarily in oncology, hematology, and rare diseases. Brian was formerly Senior Vice President, Head of Research & Development and Chief Medical Officer of ArQule Inc., which was acquired by Merck & Co. in 2020 for $2.7 billion. Dr. Schwartz is a member of the Company’s Science & Technology Committee.

Karin L. Walker brings over 30 years of extensive finance experience in biopharmaceuticals, including public biotechnology and technology companies. Ms. Walker currently serves as the Chief Accounting Officer of Prothena Corporation plc. Ms. Walker has been appointed as Chair of the Audit Committee.

Raised approximately $25 million in net cash in two equity financings, including a strategic investment by Acorn Bioventures of $6.9 million, net. An additional $8.8 million of proceeds have been received through warrant exercises ($4.3 million of which after year end).
Clinical studies

Reported data from a Phase 1 study of fadraciclib as a single agent at the Plenary Session of the 32nd EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) (ENA) Symposium:
Radiographically confirmed partial response (PR) after a month and a half on i.v. fadraciclib in a patient with MCL1-amplified endometrial cancer, who failed seven lines of prior therapy and is continuing treatment for more than 18 months with 96% reduction in target tumor lesions.
High bioequivalence observed in five patients treated with oral fadraciclib.
Enrolled 19 patients with relapsed or refractory AML/MDS and CLL receiving i.v. fadraciclib in combination with venetoclax with evidence of antileukemic activity.
Enrolled seven patients evaluating i.v. CYC140 in patients with advanced leukemias.
Enrolled 12 patients with relapsed or refractory AML/MDS in a Phase 1/2 study evaluating an oral regimen of sapacitabine in combination with venetoclax.
Announced a peer-reviewed publication describing the discovery of fadraciclib in PLOS ONE. Authored by scientists from Cyclacel and The Institute of Cancer Research, London, the publication shows that targeting of CDK2 and CDK9 holds broad therapeutic potential.
More information on our clinical trials can be found here.

Key Business Objectives for 2021

First patient dosed with oral fadraciclib in Phase 1b/2 advanced solid tumor and leukemia studies
First patient dosed with oral CYC140 in Phase 1/2 advanced solid tumor and leukemia studies
Manufacture clinical supplies of oral fadraciclib and oral CYC140 for registration-enabling studies
Data on safety and antileukemic activity from the i.v. fadraciclib-venetoclax Phase 1 study in relapsed/refractory AML and CLL
Data from the sapacitabine-venetoclax Phase 1/2 study in relapsed/refractory AML or MDS
Initial data from the i.v. CYC140 Phase 1 First-in-Human study in patients with advanced leukemias
Data from the Phase 1b/2 IST of sapacitabine-olaparib combination in patients with BRCA mutant metastatic breast cancer when reported by the investigators.
Financial Highlights

As of December 31, 2020, cash and cash equivalents totaled $33.4 million, compared to $11.9 million as of December 31, 2019. The increase of $21.5 million was primarily due to $29.5 million of net cash provided by financing activities, offset by net cash used in operating activities of $7.9 million and $0.1 million of net cash used in investing activities.

Research and development expenses were $1.4 million and $4.8 million for the three months and year ended December 31, 2020 as compared to $1.4 million and $4.7 million for the same periods in 2019. Research and development expenses relating to the transcriptional regulation, CDK inhibitor program with fadraciclib increased by $0.5 million from $3.1 million for the year ended December 31, 2019 to $3.6 million for the year ended December 31, 2020, as the clinical evaluation of fadraciclib progressed. Research and development expenses relating to CYC140 decreased by $0.1 million from $0.7 million for the year ended December 31, 2019 to $0.6 million for the year ended December 31, 2020, primarily as a result of a reduction in expenditures associated with drug supply manufacturing which were not required in 2020. Research and development expenses relating to other research and development decreased by $0.1 million from $0.4 million for the year ended December 31, 2019 to $0.3 million for the year ended December 31, 2020, due to a reduction in consultancy costs.

General and administrative expenses for the three months and year ended December 31, 2020 were $1.7 million and $5.9 million respectively, compared to $1.4 million and $5.0 million for the same periods of the previous year.

Total other income, net for the three months and year ended December 31, 2020 were $14,000 expense and $1.0 million income, compared to $41,000 and $0.6 million income for the same periods of the previous year. The increase of $0.4 million for the year ended December 31, 2020 is primarily related to income received under an Asset Purchase Agreement with ThermoFisher Scientific.

United Kingdom research & development tax credits were $0.4 million and $1.2 million for the three months and year ended December 31, 2020 as compared to $0.4 million and $1.3 million for the same periods in 2019.

Net loss for the three months and year ended December 31, 2020 were $2.8 million and $8.4 million compared to $2.3 million and $7.8 million for the same periods in 2019.

The Company raised net proceeds of approximately $29.7 million during 2020, from agreements to sell securities and warrant conversions. An additional $4.3 million in proceeds from warrant conversions was received after year end.

The Company estimates that proforma cash resources, including proceeds of recent warrant exercises after December 31, 2020, of $37.7 million, will fund currently planned programs through early 2023.

On February 25, 2021 Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the fourth quarter and full year 2020. Net loss for the fourth quarter was $43.9 million or $0.62 per share and the net loss for the year 2020 was $127.3 million or $1.97 per share (Press release, Cytokinetics, FEB 25, 2021, View Source [SID1234575776]). Net loss for the fourth quarter of 2019 was $30.6 million or $0.52 per share and net loss for the year 2019 was $121.7 million or $2.11 per share. Cash, cash equivalents and investments totaled $501.0 million at December 31, 2020.

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"In the fourth quarter, we were pleased to present the results of GALACTIC-HF which demonstrated a positive effect on the primary composite endpoint of cardiovascular death or heart failure events in patients receiving standard of care plus omecamtiv mecarbil, with potentially larger treatment effects in patients with increasingly lower ejection fractions. In the next few weeks, we plan to discuss the results of GALACTIC-HF with FDA as may inform a potential registration path," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "In addition, we recently progressed REDWOOD-HCM to Cohort 2 following a positive interim analysis and we completed enrollment promptly afterwards. Results from both cohorts are expected mid-year. We believe that we are well positioned for what may be a transformational year for the company as we approach potential commercialization of our first medicine for patients with heart failure and we hope to advance two other programs into pivotal clinical trials."

Q4 and Recent Highlights

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)

Results from GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure), the Phase 3 clinical trial of omecamtiv mecarbil, were presented and published online.

GALACTIC-HF demonstrated a statistically significant effect of treatment with omecamtiv mecarbil to reduce risk of the primary composite endpoint of cardiovascular (CV) death or heart failure events (heart failure hospitalization and other urgent treatment for heart failure) compared to placebo in patients treated with standard of care.

No reduction in the secondary endpoint of time to CV death was observed and no other secondary endpoints were met in accordance with the prespecified statistical analysis.

Adverse events and treatment discontinuation of study drug were balanced between the treatment arms. In general, the overall rates of myocardial ischemia, ventricular arrhythmias and death were similar between treatment and placebo groups.

The effect of omecamtiv mecarbil was consistent across most prespecified subgroups and with a potentially greater treatment effect suggested in patients with a lower left ventricular ejection fraction (LVEF).

Supplemental analyses from GALACTIC-HF were presented that demonstrated a greater treatment effect of omecamtiv mecarbil in patients with lower LVEF as well as characteristics that may indicate advanced heart failure, such as being hospitalized within the last 3 months, higher N-terminal-pro brain natriuretic peptide levels and lower blood pressures.

Continued conduct of METEORIC-HF (Multicenter Exercise Tolerance Evaluation of Omecamtiv Mecarbil Related to Increased Contractility in Heart Failure), the second Phase 3 trial of omecamtiv mecarbil.

Presented findings from analyses of claims data and electronic health records related to heart failure, including analyses of the high spending and unmet need, underscoring the growing economic burden of this disease.

Conducted market research with physicians and payors and continued other commercial planning activities for omecamtiv mecarbil.
CK-3828136 (CK-136 (formerly referred to as AMG 594), cardiac troponin activator)

Analyzed data from the completed Phase 1 study of CK-136 conducted by Amgen to inform next steps in its development.
CK-3773274 (CK-274, cardiac myosin inhibitor)

Progressed REDWOOD-HCM (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM), the Phase 2 clinical trial designed to determine the safety and tolerability of CK-274 in patients with obstructive hypertrophic cardiomyopathy (oHCM), to Cohort 2 following the conduct of an interim analysis of data from Cohort 1. In December, we opened Cohort 2 in REDWOOD-HCM to screening and it completed patient enrollment in February.

The interim analysis of data from Cohort 1 showed that patients experienced substantial reductions in the average resting left ventricular outflow tract gradient (LVOT-G) as well as the post-Valsalva LVOT-G. These clinically relevant decreases in pressure gradients were achieved with only modest decreases in average LVEF; there were no dose interruptions due to LVEF falling below 50%. Pharmacokinetic data were similar to data from Phase 1.

Safety and tolerability data were supportive of continued dose escalation with no serious adverse events attributed to study treatment reported by the investigators.

Received approval of IND submitted in China for conduct of a Phase 1 study of CK-274 under the License and Collaboration Agreement between Cytokinetics and Ji Xing Pharmaceuticals Limited.

Presented preclinical data for CK-274 showing that it reduced contractility and left ventricular outflow tract peak pressure gradient in cats with naturally occurring HCM and left ventricular outflow tract obstruction.
CK-3772271 (CK-271, second cardiac myosin inhibitor)

Presented preclinical data for CK-271 demonstrating that, in the Dahl/Salt sensitive rat model of heart failure with preserved ejection fraction (HFpEF), CK-271 attenuated the development of fibrosis and diastolic dysfunction.

Completed the planned Phase 1, single-dose pharmacokinetic evaluation and tolerability assessments of CK-271 in healthy volunteers and determined it to be suitable for further development. We are evaluating its potential for further development in connection with plans to conduct a broad development program for our cardiac myosin inhibitor(s) in HCM and other indications.
Skeletal Muscle Program

reldesemtiv (next-generation fast skeletal muscle troponin activator (FSTA))

Additional data from FORTITUDE-ALS, the Phase 2 clinical trial of reldesemtiv in patients with ALS, were presented showing that the effect of reldesemtiv was more apparent in faster progressing patients, supporting the rationale and design of COURAGE-ALS, the planned Phase 3 clinical trial of reldesemtiv in patients with ALS.

The design of COURAGE-ALS was also presented, and we conducted readiness activities in preparation for the potential start of the trial.
Pre-Clinical Development and Ongoing Research

Continued to develop new chemical entities and to conduct IND enabling studies with the expectation of our potentially advancing 1-2 potential drug candidates in development.

Continued research in collaboration with Astellas directed to the discovery of next-generation skeletal muscle activators under a joint research program extended through March 31, 2021.

Continued research activities directed to our other muscle biology research programs which we expect to continue in 2021.
Corporate

Announced we will regain worldwide rights to develop and commercialize omecamtiv mecarbil and CK-136 in May 2021.

Named Nancy Wysenski and Muna Bhanji to the company’s Board of Directors.

Received $85 million upon the closing of the sale of a royalty on mavacamten, being developed by Bristol-Myers Squibb Company (formerly by MyoKardia, Inc.), to RTW Royalty Holdings Designated Activity Company.
2021 Corporate Milestones

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)

Plan to meet with FDA to discuss GALACTIC-HF in Q1 2021.

Expect enrollment in METEORIC-HF to be completed in 1H 2021.

Develop a "go-to-market" strategy and potential commercial launch plan in 1H 2021.
CK-3773274 (CK-274, cardiac myosin inhibitor)

Expect a Phase 1 study of CK-274 in China, conducted under the License and Collaboration Agreement between Cytokinetics and Ji Xing Pharmaceuticals Limited, to start in Q1 2021.

Expect to begin an open label extension study for patients who complete REDWOOD-HCM in Q2 2021.

Expect to announce results from both cohorts in REDWOOD-HCM by mid-year 2021.

Plan to engage regulatory authorities regarding a potential registration path in 2H 2021.

Expect to begin a potential Phase 3 clinical trial of CK-274 by the end of 2021.
Skeletal Muscle Program

reldesemtiv (next-generation fast skeletal muscle troponin activator (FSTA))

We expect to conduct start-up activities for COURAGE-ALS in 2021 and may open the trial to enrollment in 2H 2021, subject to our plans relating to advancing omecamtiv mecarbil towards commercialization and CK-274 to a potential Phase 3 clinical trial in patients with oHCM.
Financials

Revenues for the three and twelve months ended December 31, 2020 were $6.7 million and $55.8 million, respectively, compared to $5.2 million and $26.9 million for the corresponding periods in 2019. The increase in revenues for the year ended December 31, 2020 was primarily due to $36.5 million of license revenue recognized in the third quarter 2020 for the RTW transactions.

Research and development expenses for the three and twelve months ended December 31, 2020 increased to $29.2 million and $97.0 million, respectively, compared to $18.3 million and $86.1 million for the same periods in 2019, respectively, due primarily to increased spending for our cardiac myosin inhibitor programs and increased spending on readiness for reldesemtiv offset by a study that concluded on reldesemtiv in 2019.

General and administrative expenses for the three and twelve months ended December 31, 2020 increased to $13.9 million and $52.8 million from $10.6 million and $39.6 million in 2019 due primarily to an increase in personnel related costs including stock-based compensation and higher outside spending for commercial readiness.

2021 Financial Guidance

The company reported financial guidance for 2021. The company anticipates revenue will be in the range of $23 to $28 million, operating expenses will be in the range of $195 to $205 million, and net cash utilization will be approximately $160 to 170 million. Our current cash balance of $501 million represents more than two years of forward cash based on our projected operating expenses and this net cash utilization range. The net cash utilization range includes approximately $35 million of non-recurring, building construction and related costs; it also includes receipt of a potential $45 million from RTW Royalty Holdings Designated Activity Company in exchange for a low single digit royalty on CK-274 in connection with the funding agreement signed in July 2020, subject to conditions for payment being fulfilled. Should we not exercise our option to receive the $45 million, we expect our net cash utilization range will be increased. We expect to revise our financial guidance mid-year once we finalize strategies and potential commercial launch plans for omecamtiv mecarbil. Significant additional expenses may arise from our executing on those strategies and plans that are not included in the current financial guidance.

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will review the company’s fourth quarter 2020 results via a webcast and conference call today at 4:30 PM Eastern Time. The webcast can be accessed through the Investors & Media section of the Cytokinetics website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or +1 (706) 679-3078 (international) and typing in the passcode 6555297.

An archived replay of the webcast will be available via Cytokinetics’ website until March 11, 2021. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or +1 (404) 537-3406 (international) and typing in the passcode 6555297 from February 25, 2021 at 7:30 PM Eastern Time until March 11, 2021.

On February 25, 2021 Celsion Corporation (NASDAQ: CLSN), a clinical-stage development company focused on DNA-based immunotherapy and next-generation vaccines, reported an update on its Phase I/II OVATION 2 Study with GEN-1 in patients with advanced ovarian cancer, including interim observations (Press release, Celsion, FEB 25, 2021, View Source [SID1234575744]). GEN-1 is Celsion’s DNA-mediated interleukin-12 (IL-12) immunotherapy designed using TheraPlas, its proprietary, synthetic, non-viral nanoparticle delivery system platform.

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The OVATION 2 Study combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).

To date, the Company has enrolled approximately one-third, or 34 patients, of the anticipated 110 patients to be enrolled into the OVATION 2 Study, of which 20 are in the treatment arm and 14 are in the control. Currently, 27 patients have had their interval debulking surgery with the following results:

12 of 15, or 80%, of patients treated with GEN-1 had a R0 resection, which indicates a microscopically margin-negative complete resection in which no gross or microscopic tumor remains in the tumor bed.
7 of 12 patients, or 58%, of patients in the control arm had an R0 resection.
This interim data represents a 38% improvement in R0 resection rates for GEN-1- patients compared with control arm patients and is consistent with the reported improvement in resection scores noted in the encouraging Phase I OVATION I Study, the manuscript of which has been submitted for peer review publication.
"As the goal for surgical debulking is to eliminate microscopic disease, more ovarian cancer patients require neoadjuvant chemotherapy. However, little progress has been made in adding additional efficacious immunotherapy agents to standard neoadjuvant chemotherapy," said Premal H. Thaker, M.D., MSc., Professor in Gynecologic Oncology and Director of Gynecologic Oncology Clinical Research at Washington University School of Medicine in St. Louis and lead Principal Investigator for the OVATION 2 Study. "The results seen to date in the OVATION 2 Study are exciting and impactful for ovarian cancer patients."

The Company further reports that 22 clinical sites in the U.S. and Canada have been initiated, with three more sites expected to be added by the end of the first quarter. Clinical investigators met in early February in a virtual meeting and expressed excitement about the potential for GEN-1 to treat advanced ovarian cancer and, despite the challenges and earlier delays posed by the COVID-19 pandemic, they remain committed to completing enrollment in the study during the second half of 2021.

Commenting on the interim patient reports, Dr. Nick Borys, chief medical officer of Celsion, said, "We are gratified that such a high proportion of GEN-1 patients had no residual disease at the time of their debulking surgery. These results are consistent with what we observed in our Phase I (OVATION I) study. This is great news for the patients and the surgeons in our study as a R0 resection suggests a good clinical outcome. We are following our patients carefully to see how well they do long term. The OVATION 2 investigators remain enthusiastic about the potential of GEN-1 to treat late-stage ovarian cancer, which currently has few treatment options."

Celsion announced earlier this week that GEN-1 had received Fast Track designation from the U.S. Food and Drug Administration (FDA). This designation is intended to facilitate the development and expedite the regulatory review of drugs to treat serious conditions and fill an unmet medical need. According to the FDA, a Fast Track Drug must show some advantage over available therapy, including:

Showing superior effectiveness, effect on serious outcomes or improved effect on serious outcomes
Avoiding serious side effects of an available therapy
Decreasing a clinically significant toxicity of an available therapy that is common and causes discontinuation of treatment
About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy or a combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer, and recently completed a Phase Ib dose-escalation trial (OVATION 1 Study) of GEN-1 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer.