On May 16, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to creating novel solutions that treat urothelial and specialty cancers, reported that highlights new data on real-world experience utilizing the antegrade approach via nephrostomy tube for administration of JELMYTO (mitomycin) for pyelocalyceal solution (Press release, UroGen Pharma, MAY 16, 2022, View Source [SID1234614633]). This data adds to a growing body of evidence on the safety and efficacy profile of the antegrade method of administration for JELMYTO. These data were presented during a podium presentation at the 2022 American Urological Association (AUA) annual meeting in New Orleans, Louisiana.

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"JELMYTO is efficacious as a chemoablative agent in adult patients with low grade upper tract urothelial cancer, and while it’s FDA approved for both antegrade and retrograde administration, prior reports are limited to the retrograde experience," said Kyle Rose, MD, Urologic Oncology Fellow at Moffitt Cancer Center in Tampa, Fla., and study investigator. "These data provide additional evidence that instillation via a nephrostomy tube is an effective instillation method with a safety profile that offers an encouraging option to appropriate patients."

Dr. Rose presented the abstract Antegrade Administration of Reverse Thermal Mytomycin Gel for Primary Chemoablation of Upper Tract Carcinoma via Percutaneous Nephrostomy Tube: A Multi-Institutional Real-World Experience (Abstract PD58-06) during a podium presentation at the AUA annual meeting on Monday, May 16.

"All 71 patients in the Phase 3 OLYMPUS trial utilized the retrograde approach to administer JELMYTO, therefore we are pleased to see real-world evidence that supports the utilization of the antegrade approach giving physicians and patients more options to administer JELMYTO based on their preference and experience," said Mark Schoenberg, MD, Chief Medical Officer, UroGen.

About This Study

The real-world data from this retrospective analysis was pooled from Moffitt Cancer Center, Tampa, FL; University of Missouri School of Medicine, Columbia, MO; The University of Texas MD Anderson Cancer Center, Houston, TX; and Mayo Clinic, Rochester, MN.

Twenty-six patients received nephrostomy tube administration of JELMYTO, six patients (23%) had solitary kidneys. Nine patients (35%) went on to receive at least one dose of maintenance therapy. Ureteral stenosis occurred in four patients (15%). Other adverse events included fatigue (27%), flank pain (19%), urinary tract infection (12%), sepsis (8%) and hematuria (8%). No patients had impaired renal function during follow-up and no deaths occurred.

Thirteen patients (50%) exhibited a complete response at post-induction ureteroscopy while 12 patients (46%) had a partial response. One patient experienced progression to invasive disease and required a nephroureterectomy. At a median follow-up of seven months (IQR 3-9) post-induction, no patients who experienced a complete response recurred.

The limitations of this study include the retrospective nature, small sample size, and pooled reporting of results. There is a need for larger studies with longer follow-up to study more conclusively any potential advantages of antegrade JELMYTO administration when compared to retrograde instillation.

About the Pivotal OLYMPUS Study

OLYMPUS (Optimized DeLiverY of Mitomycin for Primary UTUC Study) was an open-label, single-arm Phase 3 clinical study of UGN-101 JELMYTO (mitomycin) for pyelocalyceal solution, to evaluate the safety, tolerability and tumor ablative effect of JELMYTO in patients with low-grade Upper Tract Urothelial Cancer UTUC (LG UTUC). Seventy-one patients were treated at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of JELMYTO administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine Complete Response (CR), the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer and for cause biopsy. Patients who achieved a CR at the PDE timepoint were eligible for the maintenance phase of the trial, during which they could receive monthly maintenance instillations for up to 12 months and were assessed to determine the durability of response with JELMYTO.

In the OLYMPUS study, data was generated for the retrograde administration of JELMYTO. In that study population ureteric obstruction was reported in 58% (n=41) of patients receiving JELMYTO, including 17% (n=12) of patients who experienced Grade 3 obstruction.

About LG UTUC

LG UTUC is a rare disease managed by endoscopic methods and radical nephroureterectomy. Endoscopic resection and laser ablation attempt to preserve the kidney, though there is a high risk of recurrence that may eventually necessitate removal of the kidney. Although kidney removal is the gold standard for treatment of high-grade UTUC, it may be over-treatment in LG UTUC, as kidney removal offers similar five-year survival as kidney-sparing procedures but is associated with significant morbidity. JELMYTO is efficacious as a primary chemoablative therapy in patients with LG UTUC.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for primary chemoablative treatment of LG UTUC in adults. It is recommended for primary treatment of biopsy-proven LG UTUC in patients deemed appropriate candidates for renal-sparing therapy. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

APPROVED USE FOR JELMYTO

JELMYTO is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO.
Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose.

Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.

are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.

You are encouraged to report negative side effects of prescription drugs to the U.S. Food and Drug Administration. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

On May 16, 2022 Cullinan Oncology, Inc. (Nasdaq: CGEM), a biopharmaceutical company focused on developing a diversified pipeline of targeted therapies for patients with cancer, reported on recent and upcoming business highlights and announced its financial results for the first quarter ended March 31, 2022 (Press release, Cullinan Oncology, MAY 16, 2022, View Source [SID1234614649]).

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"We made excellent progress in the first quarter of 2022, substantially advancing our lead asset, CLN-081, and continuing to progress our diversified pipeline," said Nadim Ahmed, Chief Executive Officer of Cullinan Oncology. "Our March clinical and regulatory update for CLN-081 further affirmed its differentiated profile, and we are looking forward to sharing an additional clinical update in an oral presentation at the upcoming ASCO (Free ASCO Whitepaper) meeting. We also announced a U.S. co-development and co-commercialization agreement for CLN-081 with Taiho, the original innovators of the molecule, and an ideal partner with whom to advance this asset into later stage development and commercialization. The proceeds from this transaction will also allow us to accelerate and expand the development of our diverse pipeline as well as discover and obtain promising new oncology therapeutic candidates."

Mr. Ahmed continued, "We are also pleased to continue the progression of our pipeline programs. Dosing in the Phase I trials of CLN-619 and CLN-049 was initiated in December 2021 and the studies are continuing on track to deliver initial data readouts by mid-2023. We are also on track to file INDs for two additional programs, CLN-617 and CLN-978, in the first half of next year. Importantly, we are in an even stronger financial position to continue advancing our strategically built pipeline of diversified assets, each with the potential to be the first or best in their class. We ended the first quarter of 2022 with approximately $410 million in cash and investments2, which, when added to proceeds from the Taiho deal, aggregates to $685 million, and extends our cash runway through 2026."

Portfolio Highlights

CLN-081 (Pearl): Cullinan announced the simultaneous sale of Cullinan Pearl and a U.S. co-development and co-commercialization collaboration of CLN-081 with Taiho Pharmaceutical Co., Ltd. (Taiho). Cullinan will receive $275 million upfront and is eligible for an additional $130 million tied to EGFR exon20 NSCLC regulatory milestones, in addition to sharing 50/50 in the future potential U.S. profits and losses for CLN-081. In January, Cullinan announced that CLN-081 was granted Breakthrough Therapy Designation (BTD) for the treatment of non-small cell lung cancer (NSCLC) patients harboring epidermal growth factor (EGFR) exon 20 insertion mutations who have previously received platinum-based systemic chemotherapy. In March, Cullinan reported a clinical and regulatory update for CLN-081, which included an improved 41% confirmed overall response rate at 100mg BID and a continued favorable safety and tolerability profile.
CLN-049 (Florentine): In December 2021, patient dosing was initiated in a first-in-human clinical trial evaluating CLN-049 in patients with relapsed/refractory AML and MDS. Initial clinical data are expected by mid-2023. CLN-049 is a FLT3/CD3-bispecific T cell-engaging antibody in an IgG format for the treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). CLN-049 targets the extracellular domain of FLT3, regardless of mutant or wild type expression.
CLN-619 (MICA): In December 2021, patient dosing was initiated in a first-in-human clinical trial evaluating CLN-619 alone and in combination with pembrolizumab in patients with advanced tumors. Initial clinical data are expected by mid-2023. CLN-619 is a first-in-class monoclonal antibody that stabilizes MICA/MICB on the tumor cell surface to promote an antitumor response via activation of both natural killer (NK) cells and certain T cells, with broad therapeutic potential across multiple cancer indications.
Preclinical Portfolio: Continued advancement of five additional oncology programs with the following highlights:
Preclinical data across five distinct programs were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting in April, including CLN-049, CLN-619, CLN-617 (Amber), CLN-978 (NexGem) and Opal.
Cullinan Oncology continues to advance two programs through Investigational New Drug (IND)-enabling studies: CLN-617, a cytokine fusion protein uniquely combining IL-12 and IL-2 with a collagen binding domain for retention in the tumor microenvironment (TME), and CLN-978, a novel CD19/CD3-bispecific construct with extended serum half-life and high potency against target cells expressing very low levels of CD19. Cullinan expects to submit INDs for both programs by the end of the first half of 2023.
First Quarter 2022 Financial Results

Cash Position: Cash and investments2 were $410 million as of March 31, 2022. Including the $275 million upfront payment related to the Taiho transaction, we expect to have $685 million in cash and investments2. As a result of the transaction and based on current operating plans, we expect to have cash runway through 2026, compared to our previous guidance of through 2024.
R&D Expenses: Research and development (R&D) expenses were $24.5 million for the first quarter of 2022, compared to $20.9 million for the fourth quarter of 2021. R&D expenses for each of the first quarter of 2022 and fourth quarter of 2021 included $2.6 million of equity-based compensation expenses. The increase in R&D expenses is primarily related to expanded clinical and chemistry, manufacturing, and controls (CMC) activity for CLN-081 and CLN-619, IND-enabling activities for CLN-617, and discovery and development of our preclinical programs.
G&A Expenses: General and administrative (G&A) expenses were $8.1 million for the first quarter of 2022, compared to $13.5 million for the fourth quarter of 2021. G&A expenses in the first quarter of 2022 and fourth quarter of 2021 included $3.8 million and $9.6 million of equity-based compensation expenses, respectively. The decrease in G&A expenses was primarily driven by a decrease in equity-based compensation expenses, partially offset by a $0.3 million increase in professional services.
Net Loss: The Company’s net loss (before items attributable to noncontrolling interest) was $12.9 million for the first quarter of 2022 compared to $34.2 million for the fourth quarter of 2021. The decrease in net loss was primarily related income tax benefits recorded for the expected utilization of current quarter losses and the release of valuation allowance of certain historical tax losses against the expected gain on the sale of Cullinan Pearl, as well as a decrease in equity-based compensation expenses, which were partially offset by increases in R&D costs to advance our portfolio.

On May 16, 2022 ​Imago BioSciences, Inc. ("Imago") (Nasdaq: IMGO), a clinical stage biopharmaceutical company discovering and developing new medicines for the treatment of myeloproliferative neoplasms (MPNs), reported that Hugh Young Rienhoff, Jr., M.D., Imago’s Chief Executive Officer, will participate in three upcoming investor conferences (Press release, Imago BioSciences, MAY 16, 2022, View Source [SID1234614665]).

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Details of the events are as follows:

HC Wainwright Global Investment Conference. Dr. Rienhoff will participate in a virtual presentation that will be available on demand beginning on Tuesday, May 24, 2022 at 7:00 am ET.

Cowen Oncology Innovation Summit. Dr. Rienhoff will present in an analyst led fireside chat on Thursday, June 2, 2022 at 3:00 pm ET.

Jefferies Healthcare Conference. Dr. Rienhoff will present on Wednesday, June 8, 2022 at 9:30 am ET.

Interested parties can access the live webcasts for these conferences from the Investor Relations section of the company’s website at www.imagobio.com. The webcast replays will be available after the conclusion of each conference.

On May 16, 2022 Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx plant cell-based protein expression system, reported financial results for the first quarter ended March 31, 2022 and provided a business update on recent corporate and clinical developments (Press release, Protalix, MAY 16, 2022, View Source [SID1234614708]).

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"We are proud of the great progress Protalix has made throughout the first quarter in advancing our Fabry disease candidate PRX-102," said Dror Bashan, Protalix’s President and Chief Executive Officer. "The announcement of compelling results from both the BRIGHT and BALANCE phase III trials marks a significant milestone, as we have now completed three phase III studies supporting the planned BLA resubmission to the U.S. Food and Drug Administration later this year. As we work toward potential approval and commercialization, we remain focused on our mission of bringing this important treatment option to patients with Fabry disease, while continuing to advance our early stage pipeline. We are grateful for the unwavering dedication of Chiesi Farmaceutici S.p.A, or Chiesi, our development and commercialization partner, and our other external partners and team members and look forward to building on our momentum throughout the rest of 2022."

2022 First Quarter and Recent Business Highlights

Regulatory Advancements

●As previously announced, on February 24, 2022, the Company, together with Chiesi, announced the submission and subsequent validation of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for PRX-102 for the treatment of adults with Fabry disease. The MAA included final data from the Company’s phase III BRIDGE clinical trial (1 mg/kg every other week) and the phase III BRIGHT clinical trial (2 mg/kg every four weeks); 12-month interim data from the Company’s phase III BALANCE clinical trial (1 mg/kg every other week); and final data from the Company’s phase I/II clinical trial data from naïve/untreated patients, including the extension study related thereto (1 mg/kg every other week).
Clinical Advancements

●On April 4, 2022, the Company, together with Chiesi, announced positive top-line results from its phase III BALANCE clinical trial, a 24-month, randomized, double-blind, active control study designed to evaluate the efficacy and safety of 1 mg/kg of PRX-102 administered every other week compared to agalsidase beta in patients previously treated with agalsidase beta for at least one year. The study met its primary endpoint and demonstrated that PRX-102 was statistically non-inferior to agalsidase beta, as measured by estimated glomerular filtration rate (eGFR) slope. In addition,
results showed a favorable tolerability profile for PRX-102, consistent with results from the Company’s prior trials.
●On March 18, 2022, the Company, together with Chiesi, announced positive final results from its phase III BRIGHT clinical trial, a multicenter, multinational open-label, switch-over study designed to evaluate the safety, efficacy and pharmacokinetics of treatment with 2 mg/kg of PRX-102 administered every four weeks for 52 weeks (a total of 14 infusions) in adult patients previously treated with a commercially available enzyme replacement therapy (ERT) (Fabrazyme or Replagal). Results of the BRIGHT study indicate that 2 mg/kg of PRX-102 administered by intravenous infusion every four weeks was well tolerated, and Fabry disease assessed by eGFR slope and plasma lyso-Gb3 was stable throughout PRX-102 treatment in adult Fabry patients.
First Quarter 2022 Financial Highlights

●The Company recorded revenues from selling goods of $9.0 million for the three months ended March 31, 2022, an increase of $4.5 million, or 100%, compared to revenues of $4.5 million for the same period of 2021. The increase of $5.4 million in sales to Brazil, resulting from timing differences, was partially offset by a decrease of $1.1 million in sales to Pfizer Inc.
●Revenue from licenses and R&D services for the three months ended March 31, 2022 were $7.1 million, compared to $6.8 million for the same period in 2021. Revenue from license and R&D services are recognized, mainly, in connection with the Company’s license and supply agreements with Chiesi.
●Cost of goods sold for the three months ended March 31, 2022 was $6.0 million, an increase of $1.2 million, or 25%, compared to cost of goods sold of $4.8 million for the same period in 2021. The increase in cost of goods sold was primarily the result of higher sales.
●Research and development expenses, for the three months ended March 31, 2022 were $8.8 million, an increase of $1.7 million, or 24%, compared to $7.1 million for the same period in 2021. The increase is primarily the result of subcontractors costs related to the completion of our phase III clinical trials of PRX-102 and maintaining our related extension studies.
●Selling, general and administrative expenses were $3.2 million for the three months ended March 31, 2022, an increase of $0.1 million, or 3% from $3.1 million for the same period in 2021.
●Financial expenses, net were $0.4 million for the three months ended March 31, 2022, a decrease of $1.4 million, or 78%, compared to $1.8 million for the same period in 2021. The decrease resulted primarily from lower interest and debt amortization costs due to a decrease in our outstanding notes from an aggregate principal amount of $57.92 million 2021 Notes to an aggregate principal amount of $28.75 million 2024 Notes.
●Cash, cash equivalents and short-term bank deposits were approximately $32.9 million at March 31, 2022.
●Net loss for the three months ended March 31, 2022 was approximately $2.3 million, or $0.05 per share, basic and diluted, compared to a net loss of approximately $5.5 million, or $0.14 per share, basic and diluted, for the same period in 2021.
Conference Call and Webcast Information

The Company will host a conference call today, May 16, 2022, at 8:30 a.m. Eastern Daylight Time, to review the corporate and clinical developments, which will also be available by webcast. To participate in the conference call, please dial the following numbers prior to the start of the call:

Please access the websites at least 15 minutes ahead of the conference to register, download and install any necessary audio software.

The conference call will be available for replay for two weeks on the Events Calendar of the Investors section of the Company’s website, at the above link.

On May 16, 2022 Celsion Corporation (NASDAQ: CLSN), a clinical-stage drug-development company focused on DNA-mediated immunotherapy and next-generation vaccines, reported financial results for the quarter ended March 31, 2022, and provided an update on its clinical development program of GEN-1, a DNA-based interleukin-12 (IL-12) immunotherapy in Phase II clinical development for the treatment of advanced-stage ovarian cancer, and its preclinical studies of PLACCINE, a proprietary, multivalent DNA plasmid technology utilizing synthetic, non-viral delivery vectors, being evaluated in proof of concept studies for superiority over the current generation of nucleic acid vaccines (Press release, Celsion, MAY 16, 2022, View Source [SID1234614585]).

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"Patient enrollment in OVATION 2, our randomized Phase II study of advanced ovarian cancer patients, continues to show momentum with 85% of patients enrolled as of May 15, 2022, and full enrollment expected to be completed by the third quarter of 2022. We remain encouraged by surrogate endpoints like surgical resection scores among patients being treated at the 100 mg/m² dose cohort in the study. Early reports from the first thirty-nine patients who have undergone interval debulking surgery showed a 27% improvement in the surgical resection (R0) rate in the GEN-1 treatment arm over the control arm. A complete R0 is a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer.

"We have presented results from preclinical studies which showed our next-generation PLACCINE DNA vaccine can target two different viral variants, demonstrating immunogenicity as determined by the levels of IgG, neutralizing antibodies, and T-cell responses. Our research shows that our multivalent PLACCINE vaccine was equally effective against two different variants of the COVID-19 virus while the commercial mRNA comparison vaccine appeared to have lost some activity against the newer variant," added Mr. Tardugno. "Our goal is to demonstrate PLACCINE’s superiority with respect to the quality of immune response against SARS-CoV-2 variants, including a longer duration of immune response and a stable product at commercially favorable storage temperatures when compared to commercially available mRNA vaccines. To that end, we have engaged BIOQUAL, Inc., a preclinical testing contract research organization, to conduct a non-human primate (NHP) challenge study of our first of many vaccine candidates as protection against SARS-CoV-2. The first inoculation of NHP subjects occurred the last week of March with the goal of generating important data to inform human clinical studies by mid-June 2022."

Recent Developments

GEN-1 Immunotherapy

Data Safety Monitoring Board’s Unanimous Recommendation to Continue Dosing Patients in the Phase II Portion of the OVATION 2 Study with GEN-1 in Advanced Ovarian Cancer. In February 2022, the Company announced that following a pre-planned interim safety review of eighty-one as treated patients randomized in the Phase I/II OVATION 2 Study with GEN-1 in advanced (Stage III/IV) ovarian cancer, the Data Safety Monitoring Board (DSMB) unanimously recommended that the OVATION 2 Study continue treating patients with the dose of 100 mg/m2. The DSMB also determined that safety is satisfactory with an acceptable risk/benefit, and that patients tolerate GEN-1 during a course of treatment that lasts up to six months. No dose-limiting toxicities were reported.

Findings from the Use of a Synthetic Control Arm to Estimate Treatment Effect in Phase Ib dose-escalating OVATION I Study presented at 2022 AACR (Free AACR Whitepaper) Annual Meeting. In April 2022 Celsion demonstrated its commitment to innovation in clinical research. The Company and the premier global data management CRO, Medidata, announced findings on the use of a Synthetic Control ArmⓇ (SCA) in a completed Phase Ib dose-escalating study of GEN-1 in the neoadjuvant treatment of patients with Stage III/IV ovarian cancer at the Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper). In a poster presentation entitled "Phase IB trial efficacy estimates via a clinical trial synthetic control arm," which took place on Monday April 11, 2022 from 9 AM through 12:00 PM EST, the research team’s findings demonstrated how comparing patients from a single-arm trial can help enhance understanding of treatment effects in advance of randomized trials, inform drug development and trial design, and increase the scientific value of early phase trials.

A Synthetic Control Arm is a type of external control and is formed by carefully matching patients treated with a new investigational therapy to anonymized clinical trial patients from Medidata’s extensive repository of historical clinical trials using baseline demographic and disease characteristics. Using this advanced statistical methodology, Celsion and Medidata found that progression-free survival was prolonged for the patients treated with the investigational therapy GEN-1 along with standard of care chemotherapy in the OVATION 1 Study compared to well-balanced historic control patients treated with the same standard of care chemotherapy alone (Hazard Ratio=0.53, 95% Confidence Interval (0.16, 1.73). This larger than expected effect size led to a decrease in the number of planned patients for Celsion’s subsequent Phase II trial and was used in support of Fast Track Designation from the U.S. Food and Drug Administration (FDA) received in February 2021.

Vaccine Initiative

Proof of Concept Vaccine Candidate Advanced to Non-Human Primate Challenge Study Against SARS-CoV-2. In January 2022, the Company announced it had engaged BIOQUAL, Inc., a preclinical testing contract research organization, to conduct a non-human primate (NHP) challenge study with Celsion’s DNA-based approach for a SARS-CoV-2 vaccine. The NHP pilot study follows the generation of encouraging murine data and will evaluate the Company’s vaccine formulations for safety, immunogenicity and protection against SARS-CoV-2.

In completed preclinical studies, Celsion demonstrated safe and efficient immune responses including IgG response, neutralizing antibodies and T-cell responses that parallel the activity of commercial vaccines following intramuscular (IM) administration of novel vaccine compositions expressing a single viral antigen. In addition, vector development has shown promise of neutralizing activity against a range of SARS-CoV-2 variants. Celsion’s novel DNA-based vaccines are based on a simple intramuscular injection that does not require viral encapsulation or special equipment for administration. Ongoing directional and technical guidance from our Vaccine Advisory Board, which is comprised of leaders in commercial vaccine development, virology, vector engineering and drug development, has been invaluable as we approach this critical advancement in our platform development program. We expect NHP studies to begin during the second quarter of 2022 with the goal of generating important data to inform human clinical studies.

PLACCINE Vaccine Platform Technology Highlighted During Oral Presentation at the World Vaccine Congress. In April 2022, the Company presented its PLACCINE platform technology at the World Vaccine Congress which took place in Washington D.C. In an oral presentation during a Session on Cancer and Immunotherapy, Dr. Khursheed Anwer, Celsion’s Chief Science Officer, highlighted the Company’s technology platform in his presentation entitled: "Novel DNA Approaches for Cancer Immunotherapies and Multivalent Infectious Disease Vaccines." PLACCINE is one of three platform technologies Celsion has for a range of therapeutics in oncology and immunotherapy. A copy of Dr. Anwer’s presentation is available on the investor portion of the Celsion website under Scientific Presentations.

PLACCINE is demonstrating the potential to be a platform for a range of infectious disease that provides for rapid design capability for targeting two or more different variants of a single virus in one vaccine. There is a clear public health need for vaccines today that address more than one strain of viruses, like COVID-19, which have fast evolving variant capability. Murine model data has thus far been encouraging and suggests that the Company’s approach provides not only flexibility, but also the potential for efficacy comparable to benchmark COVID-19 commercial vaccines with durability to protect expected to be greater than 6 months.

In the murine model, our multivalent PLACCINE vaccine targeted against two different variants showed to be immunogenic as determined by the levels of IgG, neutralizing antibodies, and T-cell responses. Additionally, our multivalent vaccine was equally effective against two different variants of the COVID-19 virus while the commercial mRNA vaccine appeared to have lost some activity against the newer variant. The Company continues to evaluate our technology and look forward to the results from our ongoing proof-of-concept non-human primate study evaluating our PLACCINE vaccine against the challenge from live SARS-CoV-2 virus in the second quarter, with durability results available in the second half of this year.

Corporate Developments

Issuance and Redemption of Convertible Redeemable Preferred Stock. In January 2022, the Company announced that it had entered into a securities purchase agreement with certain institutional investors to purchase Series A and B convertible redeemable preferred stock for approximately $28.5 million in gross proceeds. The Series A and Series B preferred stock permitted the holders thereof to vote together with the holders of the Company’s common stock on a proposal to effectuate a reverse stock split of the Company’s common stock at a special meeting of Company stockholders held on February 24, 2022. The Series A and Series B preferred stock were not permitted to vote on any other matters.

The holders of the Series A and Series B preferred stock had the right to redeem their shares of preferred stock for cash at 105% of the stated value after the Company’s stockholders’ approval of the reverse stock split. On March 3, 2022, the Company redeemed for cash at a price equal to 105% of the $300 stated value per share all of its Preferred Stock. As a result, all shares of the Preferred Stock have been retired and are no longer outstanding and Celsion’s only class of outstanding stock is its common stock, par value $0.01 per share. Each share of common stock entitles the holder to one vote.

Received $1.4 Million in Non-Dilutive Funding from the Sale of its New Jersey State Net Operating Losses, with an Additional $3.5 Million Expected in 2022 – 2023. In February 2022, the Company announced it has received $1.4 million in net cash proceeds from the sale of approximately $1.5 million of its unused New Jersey net operating losses (NOLs). The NOL sales cover the tax year 2020 and are administered through the New Jersey Economic Development Authority’s (NJEDA) Technology Business Tax Certificate Transfer Program. The Company plans to sell an additional $3.5 million of unused New Jersey NOLs available to the Company under the program over the next 2 years. The Technology Business Tax Certificate Transfer Program administered by the NJEDA enables qualified companies to sell up to $20 million of their unused New Jersey net operating losses and R&D tax credits to unaffiliated, profit-generating corporate taxpayers in the state of New Jersey. The economic development program is designed to allow technology and biotechnology companies with NOLs to turn their tax losses and credits into cash proceeds to fund more R&D, expand its workforce, and cover other allowable expenditures.

Strengthened Balance Sheet Through Registered Direct Offering of Common Shares totaling $7.0 Million in Gross Proceeds Priced At-The-Market under NASDAQ Rules. On April 8, 2022, the Company announced the closing of a registered direct offering of 1,328,274 shares of common stock at a purchase price of $5.27 per share, resulting in gross proceeds of $7.0 million, before deducting placement agents’ fees and expenses. Celsion intends to use the net proceeds for general corporate purposes, including research and development activities, capital expenditures and working capital.

Financial Results for the Three Months Ended March 31, 2022

Celsion reported a net loss for the first quarter of 2022 of $10.5 million ($1.82 per share) compared with a net loss of $5.7 million ($3.31 per share) in 2021. Operating expenses were $6.0 million for the first quarter in 2022, which represented a $0.5 million (8%) increase from $5.5 million for the first quarter of 2021.

Net cash used for operating activities was $8.0 million for the first quarter of 2022, compared with $4.7 million for the comparable prior-year period. This increase is attributable to higher non-operating expenses (interest expense related to the one-time sale and subsequent redemption of $30 million convertible, redeemable preferred stock during the first quarter of 2022). The Company had $47.3 million in cash, investments and restricted cash as of March 31, 2021. Combined with $6.5 million of net proceeds received from the sale of equity in a registered direct offering that closed on April 8, 2022, along with $3.5 million in future planned sales of the Company’s State of New Jersey NOLs, the Company believes it has sufficient capital resources to fund its operations into the second quarter of 2025.

Research and development expenses were $3.1 million for the first quarter of 2022, an increase of $0.5 million or 20% from $2.6 million for the comparable period in 2021. R&D costs associated with the development of GEN-1 to support the OVATION 2 Study as well as development of the PLACCINE DNA vaccine technology platform increased to $1.9 million in the first quarter of 2022 compared to $1.4 million in the same three-month period of 2021. Costs associated with the OPTIMA Study were $0.1 million in the first quarter of 2022 and 2021. In July 2020, the Company unblinded the OPTIMA Study at the recommendation of the DMC to halt the study due to futility. Other clinical and regulatory costs were $0.8 million in the first quarter of 2022 and $0.6 million the first quarter of 2021. CMC costs decreased to $0.3 million in the first quarter of 2022 compared to $0.5 million in in the first quarter of 2021 due to the discontinuation of the ThermoDox clinical development program in primary liver cancer.

General and administrative expenses were $2.9 million in each of the first quarters of 2022 and 2021. Lower non-cash stock compensation expense of $0.4 million was offset by higher salaries and benefits, higher professional fees (largely legal fees to defend various suits filed after the announcement in July 2020 of the OPTIMA Phase III clinical results) and higher premiums for directors’ and officers’ insurance in the first quarter of 2022 when compared to same prior year period.

Other non-operating expenses increased to $4.6 million in the first quarter of 2022 compared to $0.3 million in the comparable prior year. Interest expense increased by $4.5 million resulting from the sale and subsequent redemption of $30 million of Series A & B convertible redeemable preferred stock during the first quarter of 2022.

Conference Call

The Company is hosting a conference call to provide a business update, discuss first quarter 2022 financial results and answer questions at 11:00 a.m. EDT today. To participate in the call, interested parties may dial 1-888-394-8218 (Toll-Free/North America) or +1-323-794-2588 (International/Toll) and ask for the Celsion Corporation First Quarter 2022 Earnings Call (Conference Code: 7615593) to register ten minutes before the call is scheduled to begin. The call will also be broadcast live on the internet at www.celsion.com. The call will be archived for replay on Monday, May 16, 2022, and will remain available until May 30, 2022. The replay can be accessed at +1-719-457-0820 or 1-888-203-1112 using Conference ID: 7615593. An audio replay of the call will also be available on the Company’s website, www.celsion.com, for 90 days after 2:00 p.m. EDT Monday, May 16, 2022.