On May 14, 2021 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported financial results for the first quarter ended March 31, 2021 (Press release, Leap Therapeutics, MAY 14, 2021, View Source [SID1234580014]).

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!

Leap First Quarter Highlights:

Completed enrollment for first-line patient cohort in the DisTinGuish study, a clinical trial evaluating Leap’s anti-Dickkopf-1 (DKK1) antibody, DKN-01, in combination with tislelizumab, BeiGene Ltd.’s anti-PD-1 antibody, with or without chemotherapy, in patients with gastric or gastroesophageal junction cancer (G/GEJ)
Presented updated clinical data from the Phase 2 study of DKN-01 as a monotherapy and in combination with paclitaxel in patients with advanced gynecological malignancies at the Society of Gynecologic Oncology (SGO) 2021 Virtual Annual Meeting on Women’s Cancer
Announced partnership to use a clinically validated tumor expression assay utilizing RNAscope and image analysis with Flagship Biosciences for patient enrollment
"We’re off to a strong start this year as we’ve continued to advance our understanding of DKN-01 and the potential role it can play as both a monotherapy or in combination with existing treatments in multiple DKK1 biomarker defined cancer indications," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "The completion of enrollment of the first-line patient cohort in the DisTinGuish study brings us one step closer to an important milestone for us with our partner BeiGene, anticipated later this year."

DKN-01 Development Update

DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the DKK1 protein. DKK1 modulates the Wnt/Beta-catenin and PI3kinase/AKT signaling pathways, which have an important role in tumor cell signaling and in mediating an immuno-suppressive tumor microenvironment through enhancing the activity of myeloid-derived suppressor cells and downregulating NK cell ligands on tumor cells.

Leap Announced Completion of Enrollment in First-Line Cohort in the DisTinGuish Study of DKN-01 plus Tislelizumab and Chemotherapy in Gastric Cancer – In April 2021, Leap announced the completion of enrollment for the first-line patient cohort in the DisTinGuish study (NCT04363801), a clinical trial evaluating DKN-01 in combination with tislelizumab, BeiGene Ltd.’s anti-PD-1 antibody, with or without chemotherapy, in patients with G/GEJ. The study, which is being conducted in two parts in the United States and the Republic of Korea, enrolled 25 patients with first-line G/GEJ cancer and will enroll up to 48 patients with second-line G/GEJ cancer whose tumors express high levels of DKK1. Initial data is expected in the second half of 2021. Leap is conducting this combination study as part of an exclusive option and license agreement with BeiGene for the development of DKN-01 in Asia (excluding Japan), Australia, and New Zealand.

Leap Presented Final Data for DKN-01 in Gynecologic Cancers – At the SGO 2021 Virtual Annual Meeting on Women’s Cancer, Leap presented the final data from the study of DKN-01 as a monotherapy or in combination with paclitaxel in groups composed of epithelial endometrial cancer (EEC), epithelial ovarian cancer (EOC), or carcinosarcoma (MMMT) patients. The key findings from the study were:

EEC patients and patients with Wnt activating mutations express higher levels of DKK1: EEC patients expressed higher levels of DKK1 and had a higher frequency of Wnt activating mutations than patients with EOC. Within EEC, patients with endometrioid histology had higher DKK1 expression than those with non-endometrioid histology. Patients whose tumors had Wnt activating mutations expressed 14.4 times higher levels of DKK1.

DKN-01 has enhanced activity in patients whose tumors express high levels of DKK1: In the group of 22 EEC patients treated with DKN-01 monotherapy for whom DKK1 expression data was available, patients with DKK1-high tumors (n=7) had greater ORR (14% vs. 0%), DCR (57% vs. 7%), and median PFS (3.0 months vs. 1.8 months [HR 0.39; 95% CI: 0.14, 1.1]) compared to patients with DKK1-low tumors (n=15). Additionally, seven patients did not have DKK1 expression results available, of whom one had a complete response (14%) and five (72%) had a best response of stable disease, including three patients with Wnt activating mutations. In the group of 24 EEC patients treated with DKN-01 plus paclitaxel, 72% of whom had received three or more prior systemic therapies, DKK1-high patients (n=11) had improved median PFS (5.4 months vs. 1.8 months [HR 0.34; 95% CI: 0.12, 0.97]) compared to DKK1-low patients (n=9). Four patients did not have DKK1 expression data available.

Presented DKK1 Biomarker Assay Validation Data – At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021, Leap and its clinical laboratory partner, Flagship Biosciences, presented data on the validation of a DKK1 RNAscope chromogenic in situ hybridization (CISH) assay and digital image analysis solution. Leap and Flagship have demonstrated that the DKK1 RNAscope assay and accompanying digital image analysis solution is specific, sensitive, accurate and reproducible according to Clinical Laboratory Improvements Amendments (CLIA) guidelines. The assay is currently being used to prospectively identify G/GEJ patients with elevated tumoral expression of DKK1 in the ongoing DisTinGuish clinical trial.
Selected First Quarter 2021 Financial Results

Net Loss was $9.1 million for the first quarter 2021, compared to $7.2 million for the same period in 2020. This increase was primarily due to increased development activity for the DKN-01 program and an increase in headcount and compensation expense as the Company has grown throughout the year.

License revenues for each of the first quarter 2021 and 2020 were $0.4 million, and relate to the BeiGene Agreement for the development and commercialization of DKN-01 in Asia (excluding Japan), Australia, and New Zealand. The BeiGene Agreement became effective on January 3, 2020.

Research and development expenses were $6.8 million for the first quarter 2021, compared to $4.6 million for the same period in 2020. The increase of $2.2 million in research and development expenses was primarily due to an increase of $0.8 million in payroll and other related expenses due to an increase in headcount of our research and development full time employees, an increase of $0.6 million in manufacturing costs related to clinical trial material and an increase of $0.8 million in clinical trial costs due to timing of patient enrollment.

General and administrative expenses were $2.7 million for the first quarter 2021, compared to $2.2 million for the same period in 2020. The increase of $0.5 million in general and administrative expenses was due to a $0.3 million increase in payroll and other related expenses during the three months ended March 31, 2021 as compared to the same period in 2020 and a $0.2 million increase in professional fees primarily due to increased recruiting and information technology costs.

Cash and cash equivalents totaled $43.5 million at March 31, 2021. Research and development incentive receivables totaled $0.02 million at March 31, 2021.

On May 14, 2021 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, reported financial results for the first quarter ended March 31, 2021 (Press release, Forma Therapeutics, MAY 14, 2021, View Source [SID1234580013]). The company also highlighted recent progress and upcoming milestones for its pipeline programs.

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!

"During the first quarter, we successfully completed the multiple ascending dose portion of our Phase 1 trial in sickle cell disease, and despite challenges from the COVID-19 pandemic also began enrolling patients in the Phase 2/3 trial of FT-4202, called The Hibiscus Study, as well as in the Phase 1 trial of FT-7051 for metastatic castration resistant prostate cancer," said Frank Lee, President and Chief Executive Officer of Forma. "We look forward to sharing additional pipeline results over the course of 2021 in our mission to transform the lives of people living with rare hematologic diseases and cancers."

Key Business and Clinical Highlights

PKR Program in Sickle Cell Disease (SCD):

MAD cohorts completed with approximately 71% of participants achieving hemoglobin increase ≥ 1 g/dL from baseline, and improvement across markers of RBC health. Doubling the dose of FT-4202 to 600 mg daily for 14 days compared to the previous 300 mg cohort was well-tolerated with no dose-limiting toxicities or treatment-related adverse events observed. Improvements in hematologic (hemoglobin and reticulocytes) and hemolytic (bilirubin and lactate dehydrogenase) parameters were comparable to that observed with the 300 mg dose, with best response typically observed at the end of the 14-day treatment period. In the combined cohorts, 10 of 14 (71%) patients on FT-4202 achieved a hemoglobin increase ≥ 1 g/dL from baseline to Day 14. Improvement in RBC health was evidenced by increased sickle RBC survival and reduced intravascular hemolysis in patients with SCD based on a reduction in reticulocytes, bilirubin and LDH levels.
Patient enrollment began in Phase 2/3 registrational trial, the Hibiscus Study. The Phase 2/3 Hibiscus Study is currently enrolling people living with SCD. This adaptive, randomized, placebo-controlled, double-blind, multi-center trial is expected to enroll approximately 344 adults and adolescents with SCD. FT-4202 doses of 200mg and 400mg administered once-daily are being evaluated in the Phase 2 portion of the trial. Primary endpoints in the Phase 3 portion of the trial are hemoglobin response rate at week 24 (increase of > 1 g/dL from baseline), intended to support accelerated approval, and annualized vaso-occlusive crisis rate during the 52-week blinded treatment period, which if positive is expected to support full approval.
CPB/p300 Program in Prostate Cancer:

FT-7051 Phase 1 clinical trial initiated for the treatment of mCRPC. In January 2021, Forma announced that the first patient was dosed in the ongoing Phase 1 clinical trial evaluating FT-7051 for the treatment of mCRPC. The trial is a multicenter, open-label evaluation of the safety and tolerability, preliminary anti-tumor activity (prostate specific antigen (PSA) and radiographic responses), and pharmacokinetics/pharmacodynamics (PK/PD) of FT-7051 in men with mCRPC who have progressed despite prior therapy with at least one anti-androgen therapy. The trial will include genetic mutation analysis to identify the basis of resistance to standard-of-care and will also evaluate expression of the AR-v7 splice variant, for which there are no approved therapies. The trial utilizes an adaptive trial design, intended to accelerate the escalation to potentially therapeutic doses and yield important safety information, as well as to identify biomarkers of clinical benefit such as PSA response.
IDH1 Program in AML and Glioma:

Olutasidenib NDA preparation for R/R AML. With the conclusion of the Phase 2 R/R AML trial, Forma has begun preparing an NDA for submission to the U.S. Food and Drug Administration (FDA).
Upcoming Milestones

Presentation of updated Phase 1 FT-4202 results in SCD. A poster presentation on FT-4202 in SCD is scheduled for the EHA (Free EHA Whitepaper) Virtual Congress taking place June 9-17, 2021. The presentation will include combined unblinded data from the two-week MAD cohorts as well as initial OLE results to date. In addition, full results from the MAD dose cohorts and the OLE are expected to be presented at a scientific congress in late 2021.
Initial Phase 1 clinical results from FT-7051 in mCRPC anticipated later this year. This adaptive trial is assessing multiple doses of FT-7051 with dose escalation dependent upon safety and tolerability. Initial results anticipated in the fourth quarter of 2021 may include safety/tolerability, PK/PD results and preliminary biomarker data.
Olutasidenib results presentation in R/R AML. Phase 2 registrational results of olutasidenib in R/R AML will be presented at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting taking place from June 4-8, 2021, and the EHA (Free EHA Whitepaper) Virtual Congress taking place June 9-17, 2021.
Possibility of COVID-19 impact remains. The COVID-19 pandemic remains a factor in the successful completion of these milestones. Many clinical trials across the biopharma industry, including ours, have been impacted by the COVID-19 pandemic, with clinical trial sites implementing new policies in response to COVID-19, resulting in potential delays to enrollment of clinical trials or changes in the ability to access sites participating in clinical trials.
Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $603.7 million as of March 31, 2021, as compared to $645.6 million as of December 31, 2020. Current cash runway is projected through the third quarter of 2024.
Research and Development (R&D) Expenses: R&D expenses were $26.3 million for the quarter ended March 31, 2021, compared to $23.2 million for the quarter ended March 31, 2020. The increase was primarily attributable to increases in FT-4202 development expenses, partially offset by reduced spending on olutasidenib development.
General and Administrative (G&A) Expenses: G&A expenses were $9.9 million for the quarter ended March 31, 2021, compared to $8.9 million for the quarter ended March 31, 2020. The increase in general and administrative expense was primarily attributable to stock compensation expense and insurance, partially offset by a reduction in professional fees.
Net Income/Loss: Net loss was $36.0 million for the quarter ended March 31, 2021, compared to net income of $11.2 million for the quarter ended March 31, 2020.
Forma will conduct a conference call and webcast May 14th at 8 a.m. Eastern Daylight Time (EDT) to discuss first quarter 2021 results and business update. The call can be accessed by dialing (833) 301-1146 in the U.S., and (914) 987-7386 internationally, with conference ID 8597396.

On May 14, 2021 Dr. Reddy’s Laboratories Ltd. (BSE: 500124 | NSE: DRREDDY | NYSE: RDY | NSEIFSC: DRREDDY) reported its consolidated financial results for the fourth quarter and full year ended March 31, 2021 (Press release, Dr Reddy’s, MAY 14, 2021, View Source [SID1234580010])
. The information mentioned in this release is on the basis of consolidated financial statements under International Financial Reporting Standards (IFRS).

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!

COVID portfolio We continue to play our role in the fight against Covid-19 by acting proactively to bring multiple preventive and curative treatment options, including a vaccine. Some of our major Covid-19 products are:

Sputnik V vaccine: The trials demonstrated efficacy @ 91.6%, consistent safety and immunogenicity results. In April, 2021 we received Emergency Use Authorization (EUA) for the vaccine. We have launched it today and the first dose of the vaccine was administered. Our priority is to ensure widest reach in the shortest possible time.

Remdesivir: We launched it in India and have ramped up our supplies to meet with the higher demand due to surge of the COVID cases in India.

Avigan (Favipiravir): We are selling it in India and few other markets. We are conducting phase 3 trials in North America for outpatient setting with mild to moderate symptoms. 2-deoxy-D-glucose (2-DG): We developed it in collaboration with DRDO lab. Received EUA as adjunct therapy for hospitalized moderate to severe Covid-19 patients.

Other Covid drugs: We are also working on Molnupiravir, Baricitinib and several other covid drugs for treatment ranging from mild to severe conditions. Revenue Analysis [Q4 and full year FY21] Global Generics (GG)
 Revenues from GG segment at Rs. 154.4 billion higher by 12% over FY20, on account of growth across all our markets. We witnessed double digit growth in Europe and India during the year.
Q4 revenue at Rs. 38.7 billion, YoY growth of 6% and QoQ decline of 5%. The YoY growth was driven by branded markets (India and emerging markets), Europe partly offset by decline in NAG. QoQ decline was on account of branded markets & Europe. North America 
Revenues from North America Generics for the year at Rs. 70.5 billion, YoY growth of 9%. The year was benefited by new launches, scale up of existing products and a favorable forex rate, which was partially offset by price erosion.
 Revenues for Q4 at Rs. 17.5 billion, YoY decline of 3% and QoQ growth of 1%. The YoY decline was primarily on account of higher volumes during Q4 last year due to COVID-19 related stocking up and price erosion. The QoQ growth was driven by volume traction in our base business and new product launches partly offset by price erosion.
 During this quarter, we launched 6 new products – Vigabatrin tablets (CGT status granted), Febuxostat tablets, Capecitabine tablets, Fluphenazine Hydrochloride tablets, Lansoprazole OD tablets and Abiraterone Acetate in Canada.
As of 31st March 2021, cumulatively 95 generic filings are pending for approval with the USFDA (92 ANDAs and 3 NDAs under 505(b)(2) route). Out of the pending ANDAs, 47 are Para IVs, and we believe 23 have ‘First to File’ status. Europe
 Revenues from Europe for the year at Rs. 15.4 billion. YoY growth of 32%, primarily on account of volume traction in base business, new product launches across our markets including newer markets of France, Italy and Spain and favorable fo ex, which was partially offset by price erosion. 7
 Revenues for Q4 at Rs. 4.0 billion, YoY growth of 15% and QoQ decline of 5%. QoQ decline was on account of lower volumes in our base business and price erosion which was partly offset by new products launched during the quarter. India
 Revenues from India for the year at Rs. 33.4 billion. Year-on-year growth of 15%, driven by revenues from the acquired business of Wockhardt and contribution from new product launches.
 Revenues for Q4 at Rs. 8.4 billion, YoY growth of 23%, QoQ decline of 12%. QoQ decline was led by reduction in covid drugs sales and seasonality. Emerging Markets
 Revenues from Emerging Markets for the year at Rs. 35.1 billion, growth of 7%.-Revenues from Russia for the year at Rs. 15.8 billion, YoY decline of 6%. The decline was primarily driven by adverse forex and lower volumes of some of our key molecules.-Revenues from other CIS countries and Romania for the year at Rs. 7.4 billion, YoY growth of 15%. Growth was on account of increase in volumes and new launches.-Revenues from Rest of World (RoW) territories for the year at Rs. 11.8 billion, YoY growth of 25%. Growth primarily on account of new launches and volume traction in key products, partially impacted by price erosion in certain markets.
 Revenues for the quarter are Rs. 8.8 billion, YoY growth of 10%, QoQ decline of 8%.-Revenues for Russia for the Q4 at Rs. 4.0 billion, YoY growth of 3%, QoQ decline of 11%.-Revenues from other CIS countries and Romania for the quarter are Rs. 1.9 billion, YoY growth of 7%, QoQ decline of 11%.-Revenues from Rest of World (RoW) territories for this quarter are Rs. 2.9 billion, YoY growth of 24%, QoQ decline of 1%. Pharmaceutical Services and Active Ingredients (PSAI)

Revenues from PSAI at Rs. 32.0 billion. Year-on-year growth of 24% driven by new products, increase in volumes of key products of API business and favorable forex partially offset by price erosion.
 Revenues for Q4 at Rs. 7.9 billion, YoY growth of 10% and QoQ growth of 13%.
 During the year, we have filed 14 DMFs in the US. Proprietary Products (PP) & Others  Revenues from PP & Others for the year at Rs. 3.3 billion, YoY decline of 69%. FY20 was higher due to income from sale of the US and select territory rights for two of Neurology franchise products pertaining to PP.  Revenues for Q4 are Rs. 632 million. 8 Income Statement Highlights:  Gross profit margin for the year at 54.3%, an increase of ~50 bps over previous year. The increase was driven by a better product mix and increased leverage from manufacturing overheads. This was partly offset by price erosion, lower export incentives and benefit from PP out-licensing income in FY 20. Gross profit margin for GG and PSAI business segments are at 59.0% and 29.5% respectively. 
Gross profit margin for the Q4 at 53.7% (GG: 57.9%, PSAI: 31.7%).-YoY gross margin increased by ~220 bps, primarily due to a better product mix and increased leverage from manufacturing overheads, partly offset by price erosion and lower export benefits-QoQ gross margin declined by ~10 bps.  Selling, general & administrative (SG&A) expenses for FY21 at Rs. 54.6 billion, an increase of 9% on a YoY basis. This increase was primarily due to incremental costs post the integration of the acquired divisions from Wockhardt in this year and increased freight expenses. SG&A expenses for Q4 at Rs. 14.3 billion, YoY increase of 17% and QoQ decline of 1%. SG&A as a % to sales for the full year remained in line with FY20.  Impairment charge at Rs. 6.8 billion in FY21, which were taken considering the triggers which occurred during the year.  Research & development (R&D) expenses in FY21 at Rs. 16.5 billion. As % to Revenues – FY21: 8.7% | FY20: 8.8%. R&D expenses for Q4 at Rs. 4.1 billion, as % to revenues stood at 8.7%. Our focus continues on building a healthy pipeline of new products across our markets including development of products pertaining to COVID-19 treatment.  Other operating income for the year at Rs. 982 million compared to Rs. 4.3 billion in FY20. Previous year included Rs. 3.5 billion received from Celgene pursuant to a settlement agreement in Canada.  Net Finance income for the year at Rs. 1.7 billion compared to Rs. 1.5 billion in FY20. The increase is primarily on account of higher foreign exchange gain in current year as compared to FY20. Net finance income in Q4 is Rs. 0.3 billion.  Profit before Tax for the year at Rs. 28.3 billion, YoY growth of 57%. Profit before Tax for Q4 is at Rs. 8.1 billion. 

Profit after Tax for the year at Rs. 19.1 billion and for Q4 at Rs. 5.5 billion. The tax rate in FY21 is higher due to non-recognition of deferred tax asset (DTA) on losses arising out of impairment. It was lower in FY20 due to recognition of deferred tax asset (DTA) on losses arising out of impairment, recognition of MAT credit, and others in line with the requirements of accounting standards.  Diluted earnings per share for the year is Rs. 115.14. Diluted earnings per share for Q4 is Rs. 33.29. Other Highlights:  EBITDA for FY21 at Rs. 47.5 billion and the EBITDA margin is 25.0%. EBITDA for Q4 FY21 is at 11.3 billion and the EBITDA margin in 24.0%.  Capital expenditure for FY21 is at Rs. 9.7 billion. Capital expenditure for Q4 FY21 is at Rs. 2.9 billion.  Free cash-flow at Rs. 24.6 billion before acquisitions. Free cash-flow for Q4 FY21 at Rs. 7.9 billion.  Net cash surplus for the company is at Rs. 7.5 billion as on March 31, 2021. Consequently, net debt to equity ratio is (0.04).  The Board has recommended payment of a dividend of Rs. 25 per equity share of face value Rs 5/-each (500% of face value) for the year ended March 31, 2021 subject to approval of members. 9 Earnings Call Details (05:30 pm IST, 08:00 am EDT, May 14, 2021) The management of the Company will host an earnings call to discuss the Company’s financial performance and answer any questions from the participants.

On May 14, 2021 Celsion Corporation (NASDAQ: CLSN), a clinical-stage drug development company focused on DNA-based immunotherapy and next-generation vaccines, reported financial results for the three months ended March 31, 2021, and provided an update on clinical development programs with GEN-1, a DNA-based interleukin-12 (IL-12) immunotherapy in Phase I/II clinical development for the localized treatment of ovarian cancer, and ThermoDox, a proprietary heat-activated liposomal encapsulation of doxorubicin under investigator-sponsored development for several cancer indications (Press release, Celsion, MAY 14, 2021, View Source [SID1234580006]). In addition, Celsion has two feasibility-stage platform technologies for the development of novel nucleic acid-based immunotherapies and next-generation vaccines for infectious diseases.

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!

"Celsion entered 2021 with a focus on a novel DNA-based immunotherapy for ovarian cancer and an initiative for next-generation vaccines with the potential to immunize against a broad range of infectious agents, including coronaviruses. Our platform technologies, TheraPlas and PlaCCine, are rich with promise for a pipeline of product candidates that have potential to address difficult and unaddressed diseases. During the first quarter, the Company extended its cash operating runway through 2024 and strengthened our balance sheet with the successful execution of its financing strategy by raising more than $58 million in gross proceeds from two well placed, registered direct offerings, sales under our at-the-market (ATM) equity facility, warrant exercises and the sale of our New Jersey State NOLs," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer.

"Our Phase I/II OVATION 2 Study is more than 40% enrolled. With 23 clinical sites activated, encouraging trial data to date and the strong commitment of our clinical investigators, we hope to complete enrollment before the end of 2021. Initial data at the 100 mg/m² dose cohort appear to be consistent with the directionally impressive results reported from our Phase Ib dose-escalating OVATION I Study in ovarian cancer. In the OVATION 2 study of 28 patients who completed interval debulking surgery, 81% of those treated with GEN-1 had an R0 resection, compared with 58% of control patients, a 41% improvement."

Mr. Tardugno added, "During the first quarter of 2021, we announced an initiative to focus our considerable DNA plasmid experience and competencies on DNA vaccine development, an approach we believe may represent an advance in nucleic acid immunotherapy. Leveraging our clinical-stage TheraPlas platform, we envision a vaccine characterized by a single-plasmid DNA with multiple coding regions. Celsion’s plasmid vectors currently in development are designed to promote multiple antigens that are expressed by a single pathogen in combination with a potent immune modifier such as IL-12. IL-12 is the active ingredient in our GEN-1 product candidate. We are well positioned with a capital structure sufficient to support our planned R&D and clinical programs through transformative milestones. In doing so, we look to create significant value for our shareholders, patients and the medical community."

Recent Developments

GEN-1 Immunotherapy

Announced Encouraging Interim Clinical Update on Phase I/II OVATION 2 Study with GEN-1 in Patients with Advanced Ovarian Cancer. In February 2021, the Company provided an update on its Phase I/II OVATION 2 Study with GEN-1 in patients with advanced ovarian cancer. 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 disease progression and improve overall survival. To date, the Company has enrolled more than 40% of the anticipated 110 patients to be enrolled into the OVATION 2 Study. Currently, 28 patients have had their interval debulking surgery with the following results:

● 13 of 16 patients, or 81%, 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;
● Seven of 12 patients, or 58%, in the control arm had an R0 resection; and,
● These interim data represent a 41% 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 1 Study, the manuscript of which has been submitted for peer-review publication.
Received FDA Fast Track Designation for GEN-1 in Advanced Ovarian Cancer. In February 2021, the Company announced receipt of Fast Track designation from the U.S. Food and Drug Administration (FDA) for GEN-1. Fast Track 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
Vaccine Initiative

Filed Provisional U.S. Patent Application for a Broad Range of Next-Generation DNA Vaccines. In January 2021, the Company announced the filing of a provisional U.S. patent application for a novel DNA-based, investigational vaccine for preventing or treating infections from a broad range of infectious agents, including coronaviruses, using its PLACCINE DNA vaccine technology platform. The provisional patent covers a family of novel composition of multi-cistronic vectors and polymeric nanoparticles that comprise the PLACCINE DNA vaccine platform technology for preventing or treating infectious agents that have the potential for global pandemics, including the SARS-CoV-2 virus and its variants, using the Company’s technology platform. Celsion’s vaccine approach is designed to optimize the quality of the immune response dictating the efficiency of pathogen clearance and patient recovery. Celsion has taken a multivalent approach in an effort to generate an even more robust immune response that not only results in a strong neutralizing antibody response, but also a more robust and durable T-cell response. Delivered with Celsion’s synthetic polymeric system, the proprietary DNA plasmid is protected from degradation and its cellular uptake is facilitated.

PLACCINE is a natural extension of the Company’s synthetic, non-viral TheraPlas delivery technology currently in a Phase II trial for the treatment of late-stage ovarian cancer with GEN-1. Celsion’s proprietary multifunctional DNA vaccine technology concept is built on the flexible PLACCINE technology platform that is amenable to rapidly responding to the SARS-CoV-2 virus, as well as possible future mutations of SARS-CoV-2, other future pandemics, emerging bioterrorism threats and novel infectious diseases. Celsion’s extensive experience with TheraPlas suggests that the PLACCINE-based nanoparticles are stable at storage temperatures of 4oC to 25oC, making vaccines developed on this platform suitable for broad worldwide distribution.

Formed Vaccine Advisory Board. In February 2021, the Company announced the formation of a Vaccine Advisory Board and the appointment of its first two members:

● Britt A. Glaunsinger, Ph.D., Professor, Virology & Molecular Biology, Howard Hughes Medical Institute, University of California, Berkeley; and
● Xinzhen Yang, M.D., Ph.D., Independent Professional Consultant for the Gerson Lehman Group and former Director of Viral Vaccines / Program Lead of the HCMV Vaccine Program at Pfizer Inc.
Corporate Developments

Strengthened Balance Sheet Through Two Registered Direct Offerings of Common Shares Totaling $50 Million in Gross Proceeds.

● On January 26, 2021, the Company announced the closing of a registered direct offering of 25,925,925 shares of common stock at a purchase price of $1.35 per share, priced at-the-market under Nasdaq rules, resulting in net proceeds of $32.6 million after deducting placement agents’ fees but before expenses payable by the Company.
● On April 5, 2021, the Company announced the closing of a registered direct offering of 11,538,462 shares of common stock at a purchase price of $1.30 per share, resulting in net proceeds of $13.9 million, after deducting placement agents’ fees but before expenses payable by the Company.
Celsion intends to use the net proceeds for general corporate purposes, including research and development activities, capital expenditures and working capital.

Received $2.0 Million Allocation Through the New Jersey Technology Business Tax Certificate Transfer (NOL) Program, with an Additional $5.0 Million Expected in 2021-2023. In February 2021, the Company received approval from the New Jersey Economic Development Authority’s (NJEDA) Technology Business Tax Certificate Transfer (NOL) program to sell $2.0 million of its unused New Jersey net operating losses (NOLs) for the tax years 2018 and 2019. The NOLs are typically sold at a small, single-digit discount to qualified companies with operations in New Jersey. As a result, the Company was able to transfer this credit and receive approximately $1.85 million of net cash proceeds in May 2021. An additional $5.0 million allocation of NOL sales will be available to the Company during 2021-2023.

Participated in Two Investor Events. In March 2021, the Company held one-on-one meetings with investors during the Virtual 33rd Annual Roth Conference. A webcast of Celsion’s presentation was pre-recorded and is available on the Company’s website. In April 2021, Company management participated in Alliance Global Partners’ (AGP) Virtual Series. Michael H. Tardugno and Dr. Kursheed Anwar were interviewed in a "Fireside Chat" by Matt Cross, Senior Biotech Research Analyst at AGP. The discussion focused on the Company’s lead product, GEN-1and on its PLACCINE vaccine development platform. Requests to listen to a replay can be made by emailing [email protected].

Financial Results for the Three Months Ended March 31, 2021

Celsion reported a net loss for the first quarter of 2021 of $5.7 million ($0.09 per share) compared with a net loss of $5.1 million ($0.20 per share) for the year-ago quarter. Operating expenses were $5.5 million for the first quarter of 2021, which represented a $0.6 million or 13% increase from $4.9 million for the first quarter of 2020.

Research and development expenses were $2.6 million for the first quarter of 2021, a decrease of $0.5 million or 16% from $3.1 million for the comparable period in 2020. Clinical development costs for the Phase III OPTIMA Study decreased to $0.1 million for the first quarter of 2021, compared with $0.7 million for the prior-year quarter. In July 2020, the Company unblinded the OPTIMA Study and at the recommendation of the Data Monitoring Committee halted the study due to futility. R&D costs associated with development of GEN-1 to support the OVATION 2 Study as well as development of the PLACCINE DNA vaccine technology platform increased to $1.0 million for the first quarter of 2021, compared with $0.9 million for the same period of 2020. Other costs related to the Company’s clinical development programs decreased by $0.2 million for the first quarter of 2021 compared with 2020, due to lower regulatory and manufacturing costs for the ThermoDox development program.

General and administrative expenses were $2.9 million for the first quarter of 2021, compared with $1.8 million for the first quarter of 2020. This increase is primarily attributable to higher non-cash stock compensation expense of $0.8 million, an increase in professional fees of $0.2 million and an increase in premiums for directors’ and officers’ insurance.

The Company had $54.6 million in cash and cash equivalents, short-term investments and a receivable on the sale of NOLs as of March 31, 2021. Combined with $15 million of gross proceeds received from the sale of equity in a registered direct offering that closed on April 5, 2021 along with future planned sales of the Company’s State of New Jersey NOLs, the Company believes it has sufficient capital resources to fund its operations through 2024.

Net cash used for operating activities was $4.7 million for the first quarter of 2021, compared with $5.0 million for the comparable prior-year period. Total cash provided by financing activities was approximately $40.5 million during the first quarter of 2021, resulting from $39.0 million of net proceeds from sales of common stock and $1.5 million from the exercise of common stock warrants. The Company raised an additional $13.9 million in net proceeds from sales of common stock during the second quarter of 2021. The Company recognized a $1.85 million income tax benefit resulting from the sale of its New Jersey NOLs during the fourth quarter of 2020. Net proceeds from this sale are expected to be received in the second quarter of 2021. The Company has approximately $5.0 million in future tax benefits remaining under the NJEDA Technology Business Tax Certificate Transfer program for future years.

Conference Call

The Company is hosting a conference call at 10:00 a.m. EDT today to provide a business update, discuss first quarter 2021 financial results and answer questions. To participate in the call, please dial 1-800-353-6461 (Toll-Free/North America) or 1-334-323-0501 (International/Toll) and ask for the Celsion Corporation first quarter 2021 Earnings Call (Conference Code: 8053366). The call will also be broadcast live on the internet at www.celsion.com. The call will be archived for replay on Friday, May 14, 2021 and will remain available until May 28, 2021. The replay can be accessed at 1-888-203-1112 or 1-719-457-0820 using Conference ID: 8053366. An audio replay of the call will also be available on the Company’s website, www.celsion.com, for 90 days beginning 2:00 p.m. EDT Friday, May 14, 2021.

On May 14, 2021 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery and development of drugs for the treatment of cancer, reported the company will participate and be available for 1×1 meetings at the following upcoming conference (Press release, Cellectar Biosciences, MAY 14, 2021, View Source [SID1234580005]):

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!

Oppenheimer Rare & Orphan Disease Summit
Date: May 21, 2021
Time: On demand presentation for registered participants
Available for 1×1 meetings
Webcast: To register, click on the link HERE
A replay of the presentation will be available on the Events page of the company website.