On August 12, 2021 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, reported financial results for the second quarter ended June 30, 2021, and provided an update on its clinical and preclinical programs (Press release, Synlogic, AUG 12, 2021, View Source [SID1234586421]).

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"We are executing across our co-lead metabolic programs and advancing towards proof of concept readouts of our Synthetic Biotic medicines for the treatment of Phenylketonuria and Enteric Hyperoxaluria," said Aoife Brennan, M.B. Ch.B., Synlogic’s President and Chief Executive Officer. "With a next-generation strain in a Phase 1 study for the treatment of Phenylketonuria, a strategic collaboration in place to expand our IBD pipeline and an advancing pre-clinical pipeline of metabolic disease programs, we have a robust set of potential therapies that could provide meaningful benefit to patients. We look forward to communicating results and next steps over the coming months."

Quarter Highlights

The Metabolic Portfolio:

Proof of concept data of SYNB1618 for the treatment of Phenylketonuria (PKU) anticipated in second half of 2021, Phase 1 study of SYNB1934 initiated.

The SynPheny-1 Phase 2 trial of SYNB1618 continues to progress.
SynPheny-1 is designed to evaluate plasma phenylalanine (Phe) lowering of a solid oral formulation of SYNB1618 in adult PKU patients who do not benefit from, or do not tolerate, existing therapies.
In July, the Company initiated a Phase 1 study of SYNB1934, a next-generation strain designed for the treatment of PKU, to evaluate safety, tolerability and head-to-head comparison of Phe-consumption biomarkers between SYNB1934 and SYNB1618.
SYNB1934, an evolved strain of SYNB1618 in the PKU portfolio, has the potential to provide increased benefit to patients living with PKU.
Preclinical in vivo and in vitro studies demonstrated a greater than 2-fold improvement in the ability of SYNB1934 to consume and break down Phe compared to SYNB1618.
Papers published in the journals Nature Metabolism and Communications Biology detail findings from a first-in-human study of SYNB1618 and the development of a mechanistic model to predict the function of Synthetic Biotic medicines in healthy volunteers and PKU patients.
Data from the first-in-human study of SYNB1618 showed dose-responsive, non-saturated increases in gastrointestinal consumption of Phe by SYNB1618.
These data add to the growing body of scientific research demonstrating the therapeutic potential of Synthetic Biotic medicines for the treatment of PKU.
SYNB1618 and SYNB1934 are orally administered Synthetic Biotic medicines being developed as potential treatments for PKU. They are intended to address the needs of patients of all age groups through the consumption of Phe in the gastrointestinal (GI) tract, which has the potential to lower blood Phe levels and enable the consumption of more natural protein in the diet.

Proof of concept data of SYNB8802 for the treatment of Enteric Hyperoxaluria anticipated in second half of 2021.

SYNB8802 demonstrated proof of mechanism in Part A of an ongoing Phase 1 trial, with evidence of urinary oxalate lowering in a Dietary Hyperoxaluria model in healthy volunteers given a high oxalate diet.
Urinary oxalate lowering by SYNB8802 was robust and dose-dependent.
The 3e11 dose is undergoing evaluation in Part B of the study in patients with Enteric Hyperoxaluria.
This dose was well-tolerated and resulted in a 28.6% (90% CI: -42.4 to -11.6) reduction in urinary oxalate as measured by a change from baseline compared to placebo.
Part B of the study is continuing with the evaluation of SYNB8802 in patients with Enteric Hyperoxaluria secondary to Roux-en-Y gastric bypass surgery.
Data on the development of SYNB8802 was presented at the Synthetic Biology: Engineering, Evolution & Design (SEED) conference in June 2021.
SYNB8802 is an orally administered Synthetic Biotic medicine being developed as a potential treatment for Enteric Hyperoxaluria. SYNB8802 is designed to consume oxalate in the GI tract to prevent the increased absorption of oxalate in Enteric Hyperoxaluria patients.

Enteric Hyperoxaluria results in dangerously high urinary oxalate levels causing progressive kidney damage, kidney stone formation, and nephrocalcinosis. Enteric Hyperoxaluria has no approved treatment options. Approximately 100,000 patients in the US suffer from chronic and recurrent kidney stones as a result of severe Enteric Hyperoxaluria.

The Immunomodulation Portfolio:

Progression of SYNB1891 in combination arm dosing with PD-L1 checkpoint inhibitor in Phase 1 study in patients with advanced solid tumors or lymphoma.

SYNB1891 is currently being evaluated in a Phase 1 study that has two parts: Part A is a monotherapy arm that has enrolled six dose cohorts to date. Part B is a combination arm with SYNB1891 and the PD-L1 checkpoint inhibitor atezolizumab that has enrolled two dose cohorts to date.
The study is ongoing. Mature combination therapy data is expected by the end of the year.
SYNB1891 is an investigational drug for the intra-tumoral treatment of solid tumors and lymphoma, composed of an engineered Synthetic Biotic strain of E. coli Nissle that produces cyclic di-AMP (CDA), a stimulator of the STING (STimulator of INterferon Genes) pathway.

Advancement of preclinical programs in Inflammatory Bowel Disease.

In June, Synlogic and Roche entered into a research collaboration agreement for the discovery of a novel Synthetic Biotic medicine for the treatment of inflammatory bowel disease (IBD). Under the terms of the agreement, Synlogic and Roche will collaborate to develop a Synthetic Biotic medicine addressing an undisclosed novel target in IBD.
Data on novel Synthetic Biotic approaches for the treatment of IBD was presented at Digestive Disease Week (DDW) in May 2021.
Corporate Update:

Synlogic strengthens Balance Sheet and advances synthetic biology capabilities.

In April, Synlogic completed an underwritten public offering of 11.5 million shares, resulting in net proceeds to Synlogic of approximately $32.6 million.
Synlogic and Ginkgo Bioworks continue to advance their long-term strategic platform collaboration that provides expanded synthetic biology capabilities to Synlogic with multiple undisclosed metabolic programs now in preclinical stages of development. Additional information on these programs will be provided over the course of the year.
Second Quarter 2021 Financial Results

As of June 30, 2021, Synlogic had cash, cash equivalents, and short-term investments of $115.5 million.

For the three months ended June 30, 2021, Synlogic reported a consolidated net loss of $14.5 million, or $0.28 per share, compared to a consolidated net loss of $15.5 million, or $0.44 per share, for the corresponding period in 2020.

Research and development expenses were $10.7 million for the three months ended June 30, 2021 compared to $12.9 million for the corresponding period in 2020.

General and administrative expenses for the three months ended June 30, 2021 were $4.1 million compared to $3.5 million for the corresponding period in 2020.

Revenue was $0.2 million for the three months ended June 30, 2021, compared to $0.4 million for the corresponding period in 2020. Revenue for the three months ended June 30, 2021 was due to the collaboration with Roche, for the discovery of a novel Synthetic Biotic medicine for treatment of inflammatory bowel disease (IBD). Under the terms of the agreement, Synlogic and Roche will collaborate to develop a Synthetic Biotic medicine addressing an undisclosed novel target in IBD. Revenue for the three months ended June 30, 2020 was due to the prior collaboration with AbbVie to develop Synthetic Biotic medicines for the treatment of inflammatory bowel disease, which was terminated in May 2020.

Financial Outlook

Based upon its current operating plan and balance sheet as of June 30, 2021 Synlogic expects to have sufficient cash to be able to fund the base operating plan into the second half of 2023.

Conference Call & Webcast Information

Synlogic will host a conference call and live webcast at 8:30 a.m. ET today, Thursday, August 12, 2021. To access the live webcast, please visit the "Event Calendar" page within the Investors and Media section of the Synlogic website. Investors may listen to the call by dialing +1 (844) 815-2882 from locations in the United States or +1 (213) 660-0926 from outside the United States. The conference ID number is 7586239. A replay will be available for 30 days on the Investors and Media section of the Synlogic website.

On August 12, 2021 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported corporate updates and financial results for the second quarter ended June 30, 2021 (Press release, CymaBay Therapeutics, AUG 12, 2021, View Source [SID1234586437]).

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In July 2021, CymaBay announced a development funding agreement with Abingworth, pursuant to which CymaBay will receive up to $100 million of funding for seladelpar development costs, of which $75 million will be received in three installments over approximately six months. CymaBay has an option to receive an additional $25 million within approximately two months of the completion of enrollment of CymaBay’s Phase 3 RESPONSE clinical trial. In exchange, CymaBay will make fixed payments spread over a six-year period based on regulatory approval in the U.S. or the E.U. after the first such regulatory approval is obtained, as well as pay fixed and capped sales milestones based on U.S. product sales. CymaBay has the ability to accelerate payment at a reduced amount upon regulatory approval and in the event of a change of control of CymaBay. CymaBay retains upside potential for seladelpar in the U.S. along with full worldwide commercial rights.

CymaBay also continued to make progress conducting the development program for seladelpar in primary biliary cholangitis (PBC). Additional clinical sites were activated in North America and Europe during the second quarter in RESPONSE, a global Phase 3 registrational study evaluating seladelpar in patients with PBC, and in ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional safety data to support registration. Enrollment of patients in both of these studies continued through the second quarter and is expected to increase as more sites are activated through the rest of 2021.

Sujal Shah, President and CEO of CymaBay, stated, "In addition to adding experienced talent across the functional areas vital to getting seladelpar to patients, we were thrilled to have secured the additional funding needed to complete Phase 3 development of seladelpar in PBC through a non-dilutive, risk-sharing funding agreement with Abingworth. Abingworth has had a long history of funding innovative companies in life sciences and shares our belief that seladelpar has the opportunity to meaningfully improve the care of patients with PBC. This strategic funding agreement further strengthens our balance sheet and allows us to maintain our dedicated focus on completing development of seladelpar to deliver value to patients and shareholders."

Recent Corporate Highlights

Executed a non-dilutive financing transaction with Abingworth for the development of seladelpar in PBC. Under the terms of the agreement, CymaBay will receive up to $100 million of funding for seladelpar development costs, of which $75 million will be received in three installments over approximately six months. CymaBay has an option to receive an additional $25 million within approximately two months of the completion of enrollment of CymaBay’s Phase 3 RESPONSE clinical trial.
Conducted enrollment activities for RESPONSE, a 52-week, placebo-controlled, randomized, global, Phase 3 registrational study evaluating the safety and efficacy of seladelpar in patients with PBC. This study is targeting enrollment of 180 patients who have an inadequate response to, or intolerance to, ursodeoxycholic acid, in a 2:1 randomization to oral, once daily seladelpar 10 mg or placebo. The primary outcome measure is the responder rate at 52 weeks. A responder is defined as a patient who achieves an alkaline phosphatase level < 1.67 times the upper limit of normal with at least a 15% decrease from baseline and has a normal level of total bilirubin. Additional key outcomes of efficacy will compare the rate of normalization of alkaline phosphatase at 52 weeks and the level of pruritus at 6-months for patients with moderate to severe pruritus at baseline assessed by a numerical rating scale recorded with an electronic diary.
Conducted enrollment activities for ASSURE, an open-label, long-term study of seladelpar in patients with PBC intended to collect additional long-term safety data to support registration.
Initiated enrollment in a Phase 2a proof-of-pharmacology study to evaluate the potential for MBX-2982, a GPR119 agonist, to prevent hypoglycemia in patients with type 1 diabetes (T1D). The study is being conducted by the AdventHealth Translational Research Institute in Orlando, Florida and fully funded by The Leona M. and Harry B. Helmsley Charitable Trust with CymaBay retaining full rights to MBX-2982.
Completed a Phase 1 single ascending dose and multiple asending dose study of CB-0406, a non-agonist ligand of PPARg that attenuates the expression of inflammatory genes. While the study showed CB-0406 had improved pharmacokinetics versus those historically achieved with the pro-drug arhalofenate, CB-0406’s safety profile did not support continued development as a result of the occurrence of a small number of reversible cases of thrombocytopenia at higher doses.

Held $106.1 million in cash, cash equivalents and short-term investments as of June 30, 2021. We believe that cash and investments, along with the initial $75 million funding raised through the development funding agreement with Abingworth in July 2021 are sufficient to fund CymaBay’s current operating plan into 2023.

Due to the ongoing effects of the global coronavirus pandemic, CymaBay continues to conduct its operations remotely for all employees, which has allowed business activities to continue as seamlessly as possible. The recent emergence of the Delta variant has led to uncertainty regarding the duration and effects that the pandemic will have on future operating milestones. CymaBay continues to closely monitor pandemic developments and their associated risks to the business, including the conduct of its clinical development of seladelpar, and will continue to take actions to mitigate them where possible. Further, all CymaBay’s actions will continue to be guided by a commitment to ensuring the health and safety of its employees as well as patients enrolled in its clinical studies.
Second Quarter and Six Months Ended June 30, 2021 Financial Results

Research and development expenses for the three months ended June 30, 2021 and 2020 were $16.7 million and $7.9 million, respectively. Research and development expenses for the six months ended June 30, 2021 and 2020 were $29.1 million and $17.5 million, respectively. Research and development expenses in the three and six months ended June 30, 2021 were higher than the corresponding periods in 2020 primarily due to an increase in clinical trial activities following our resumption of clinical development of seladelpar in PBC in late 2020. In particular, cost increases were primarily driven by the enrollment activities associated with RESPONSE and ASSURE, our two active global late-stage clinical trials in PBC. In the three and six months ended June 30, 2020, costs incurred were primarily associated with the termination and shutdown of our Phase 3 PBC, Phase 2b NASH, and Phase 2 PSC clinical trials, and other studies, after the seladelpar development program was placed on hold from November 2019 through July 2020.

General and administrative expenses for the three months ended June 30, 2021 and 2020 were $6.5 million and $3.2 million, respectively. General and administrative expenses for the six months ended June 30, 2021 and 2020 were $11.8 million and $7.6 million, respectively. General and administrative expenses in the three and six months ended June 30, 2021 were higher than the corresponding periods in 2020 due to higher employee compensation associated with the hiring of additional personnel and an increase in consulting and other expenses upon resumption of development of seladelpar in the second half of 2020.

Net loss for the three months ended June 30, 2021 and 2020 was $23.2 million and $10.7 million, or ($0.34) and ($0.16) per diluted share, respectively. Net loss for the six months ended June 30, 2021 and 2020 was $40.8 million and $23.8 million, or ($0.59) and ($0.35) per diluted share, respectively. Net loss was higher largely due to increases in clinical operating expenses, which were incurred following the resumption of our clinical development of seladelpar in PBC during the second half of 2020. We expect our operating expenses to increase in 2021 as we continue to execute on our clinical development plans.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss second quarter 2021 financial results and provide a business update. To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID# 13720877. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

On August 12, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported its financial results for the quarter ended June 30, 2021 and provided a business update (Press release, Phio Pharmaceuticals, AUG 12, 2021, View Source [SID1234586455]).

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"The first half of 2021 was just the start of what promises to be an exciting period in our development of the INTASYL enabled immunotherapy compounds across our pipeline. Over the past several months, we have generated positive new preclinical data from different studies that support the initiation of two first-in-human studies of our lead asset PH-762, an INTASYL compound that targets the checkpoint protein PD-1, in cancer patients. Looking ahead, we are finalizing the studies required for the regulatory submissions for each program and expect to be in a position to initiate both studies in the first half of 2022," said Dr. Gerrit Dispersyn, President and CEO of Phio. "Overall, we are very excited by the overwhelmingly positive data generated by our pipeline of INTASYL based product candidates. This data shows that the INTASYL platform is a valuable alternative to other direct therapeutic approaches, but can also be used to improve cell based immunotherapy products."

Quarter in Review and Recent Corporate Updates

Presented a continuous stream of positive data from preclinical studies exploring the flexibility and application of INTASYL in the field of immuno-oncology at leading scientific conferences held during the second quarter of 2021:
Announced positive new data at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting that provide further evidence on the utility of the INTASYL self-delivering RNAi therapy platform to target multiple proteins and provide evidence of the synergy of the Company’s pipeline products in the field of immuno-oncology. These in vivo data showed that INTASYL specifically dual-targeting BRD4 and PD-1 elicited complete tumor responses in an in vivo hepatoma model, and significantly outperformed the efficacy of small molecule and antibody treatments towards the same targets.
Announced positive in vivo data at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) showing that PH-762 can reprogram HER2-targeted CAR-T cells (HER2CART) and significantly enhance their antitumor efficacy in solid tumors, compared to untreated HER2CART cells.
Announced new in vivo data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 showing that intratumoral (IT) treatment with PD-1 targeting INTASYL (mPH-762) inhibits tumor growth in a dose dependent fashion in both PD-1 responsive and refractory models.
Upcoming Pipeline Milestones

Expect to initiate a first-in-human clinical study on the use of PH-762 using direct drug therapy (IT) administration for patients with advanced melanoma in the first quarter of 2022.
Expect to initiate a first-in-human clinical study on the use of PH-762 and tumor infiltrating lymphocytes (TILs) in adoptive cell therapy (ACT) in patients with advanced melanoma in the second quarter of 2022.
Additional data publications on the Company’s pipeline programs.
Financial Results

Cash Position

At June 30, 2021, the Company had cash of $29.4 million as compared with $14.2 million at December 31, 2020. The Company expects its current cash will be sufficient to fund currently planned operations to the second quarter of 2023.

Research and Development Expenses

Research and development expenses were approximately $1.7 million for the quarter ended June 30, 2021, compared to approximately $0.8 million for the quarter ended June 30, 2020. The increase is primarily due to manufacturing costs and fees for the required preclinical studies in support of the Company’s planned clinical trials for PH-762 as compared to the same period in the prior year. The Company expects its research and development expenses to continue to increase in support of, and as the Company commences its clinical trial activities with PH-762.

General and Administrative Expenses

General and administrative expenses were approximately $1.0 million for the quarter ended June 30, 2021, compared to approximately $0.9 million for the quarter ended June 30, 2020. The increase is primarily due to an increase in stock-based compensation expense as no equity awards were granted in the prior year period.

Net Loss

Net loss was $2.7 million, or $0.20 per share, for the quarter ended June 30, 2021, compared with $1.7 million, or $0.34 per share, for the quarter ended June 30, 2020. The increase in net loss was primarily attributable to the increase in research and development expenses related to the Company’s preclinical activities in preparation for the start of its clinical trials with PH-762, as described above. The change in net loss per share was primarily due to an increase in the number of shares outstanding as a result of the Company’s capital raise activities as compared to the prior year period.

On August 12, 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 and six months ended June 30, 2021, and provided an update on clinical development programs with GEN-1, a DNA-based interleukin-12 (IL-12) immunotherapy in Phase II clinical development for the treatment of advanced-stage ovarian cancer (Stage III/IV), and ThermoDox, a proprietary heat-activated liposomal encapsulation of doxorubicin under investigator-sponsored development for several cancer indications (Press release, Celsion, AUG 12, 2021, View Source [SID1234586472]). 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.

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"GEN-1, our oncology-focused immunotherapy, continues to show encouraging resection results at the 100 mg/m² dose cohort in the Phase II OVATION 2 Study. These results are consistent with those reported from our earlier Phase I trials in advanced-stage ovarian cancer. In July 2021, the Data Safety Monitoring Board (DSMB) unanimously recommended that the OVATION 2 Study continue treating patients with the 100 mg/m2 dose. The DSMB also determined that safety was satisfactory with an acceptable risk/benefit and no dose-limiting toxicities," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "These findings were supported with positive R0 surgical resection scores from the first 36 patients with interval debulking surgery. Of those, 80% treated with GEN-1 at a dose of 100 mg/m² plus NACT had a complete tumor resection (R0), which indicates a microscopically margin-negative resection with no gross or microscopic tumor remaining in the tumor bed, compared with 56% of patients in the control arm having R0 resections.

"In recent weeks, Celsion strengthened its capabilities in vaccine development with the expansion of our Vaccine Advisory Board," continued Mr. Tardugno. "Drs. Dan H. Barouch and Luke D. Handke joined Drs. Britt A. Glaunsinger and Xinzhen Yang on the VAB, which was formed during the first quarter of this year. We’re delighted to assemble a group of well-known and distinguished scientists to advise management’s work in developing next-generation vaccines directed to COVID-19 and its variants, and other infectious diseases for which there are few or no preventive options."

Mr. Tardugno concluded, "Results of the OVATION 1 Study recently published in the Journal of Clinical Research showed complete/near complete chemotherapy response scores (CRS) of 50% in the two highest doses of GEN-1, compared with 28% from a major publication evaluating CRS scoring. CRS is a three-tier standardized scoring system for histological tumor regression into complete/near complete (CRS 3), partial (CRS 2) and no/minimal (CRS 1) response based on omental examination. While not an FDA recognized endpoint, like R0 resection rates, CRS 3 is believed to be a predictor of progression-free survival."

Recent Developments

GEN-1 Immunotherapy

DSMB Recommends GEN-1 to Continue Dosing Patients in the Phase II Portion of the OVATION 2 Study in 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 additional cycles of chemotherapy to treat any residual tumor.

In July 2021, the Company announced that following a pre-planned interim safety review of 55 as-treated patients randomized in the Phase I/II OVATION 2 Study, the 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 can tolerate up to 17 doses of GEN-1 during a course of treatment that lasts up to six months. No dose-limiting toxicities were reported.

More than 50% of the Projected 110 Patients Have Been Enrolled in the OVATION 2 Study. Interim clinical data from the first 36 patients who have undergone interval debulking surgery are as follows:

20 patients were treated with GEN-1 at a dose of 100 mg/m² plus NACT, with 16 out of 20 patients (80%) having a complete tumor resection (R0).
16 patients were treated with NACT only, with 9 out of 16 patients (56%) having R0 resections.
When combining these results with the surgical resection rates observed in the Company’s prior Phase Ib dose-escalation trial (the OVATION 1 Study), a population of patients with inclusion criteria identical to the OVATION 2 Study, the data reflect the strong dose-dependent efficacy of adding GEN-1 to NACT.
% Patients with R0 Resections
0, 36, 47 mg/m² of GEN-1 plus NACT n=22 50 %
61, 79, 100 mg/m² of GEN-1 plus NACT n=28 82 %
The objective response rate (ORR) as measured by Response Evaluation Criteria in Solid Tumors (RECIST) criteria for the 16 patients treated with NACT only were comparable, as expected, to the 20 patients treated with GEN-1 at a dose of 100 mg/m² plus NACT, with both groups demonstrating an approximate 80% ORR.
Poster on Phase I/II OVATION 2 Study Presented at the Society of Gynecologic Oncology Virtual Annual Meeting on Women’s Cancer. In April 2021, the Company announced that a poster highlighting its ongoing OVATION 2 Study was presented at the Virtual Annual Meeting on Women’s Cancer, sponsored by the Society of Gynecologic Oncology. The poster, titled "A Phase I/II Study Evaluating Intraperitoneal GEN-1 in Combination with Neoadjuvant Chemotherapy [NACT] in Patients with Newly Diagnosed Advanced Epithelial Ovarian Cancer (EOC)," can be viewed here. The poster was presented by Premal Thaker, M.D., Study Chair of the OVATION 2 Study and Professor of Obstetrics and Gynecology, Director of Gynecological Oncology Clinical Research, Division of Gynecologic Oncology, Washington University School of Medicine.

The poster describes the OVATION 2 Study, which 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 (NACT + GEN-1) with the control arm (NACT).

Celsion announced in the first quarter of 2021 that GEN-1 had received Fast Track designation from the U.S. Food and Drug Administration. 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.

Vaccine Initiative

Vaccine Advisory Board Expanded. In July 2021, the Company announced the addition of Dan H. Barouch, M.D., Ph.D. and Luke D. Handke, Ph.D. to its Vaccine Advisory Board (VAB). They join Britt A. Glaunsinger, Ph.D. and Xinzhen Yang, M.D., Ph.D. on the VAB, which was formed in February 2021.

Dr. Barouch is the principal investigator at the Barouch Laboratory, Director of the Center for Virology and Vaccine Research at Beth Israel Deaconess Medical Center and William Bosworth Castle Professor of Medicine at Harvard Medical School. In addition, he is a key participant in the Bill & Melinda Gates Foundation Collaboration for AIDS Vaccine Discovery, the National Institutes of Health Martin Delaney HIV-1 Cure Collaboratory and the Ragon Institute of MGH, MIT and Harvard. Dr. Barouch and his team were instrumental in developing the vector, a variant of an adenovirus called Ad26, that was used to make single-dose vaccines for HIV, tuberculosis and Zika, and ultimately, in conjunction with Johnson & Johnson researchers, SARS-CoV-2. He has authored numerous peer-reviewed articles.

Dr. Handke is a highly skilled molecular biologist and microbiologist with a decade of pharmaceutical industry experience including nine years with Pfizer’s Vaccine Research and Early Development Unit. At Pfizer he served as molecular biology lead on an early phase viral vaccine program and was the lead reviewer of data sources and literature citations for licensure application for the Trumenba meningococcal group B vaccine in the U.S. and in Europe. He began his career in vaccine research at Wyeth. He is co-author and co-inventor on various patent applications for a protein-based RSV vaccine and a SARS-CoV-2 detection assay and authored 10 peer-reviewed publications including six as first author. Dr. Handke is currently a Senior Scientist at the University of Nebraska Medical Center in Omaha. In addition to serving on the VAB, Dr. Handke will provide consulting services to Celsion in connection with its vaccine development program, which involves DNA-based vectors in combination with proprietary non-viral cellular delivery agents. He also will advise Celsion as it advances this program into human clinical studies.

ThermoDox

Subsidiary Established to Manage Investigator-Sponsored Development of ThermoDox. In June 2021, the Company announced that its new wholly owned subsidiary, Celsion GmbH, will manage all current and future investigator-sponsored development of ThermoDox. Andreas Voss, M.D., a leading oncology researcher, has been named Managing Director of Celsion GmbH and will step down from Celsion’s board of directors later this year to head the subsidiary, which is based in Zug, Switzerland.

Establishing Celsion GmbH allows Celsion’s management to focus solely on GEN-1 and PLACCINE, its nucleic acid vaccine platform. In addition to clinical and regulatory advice, Celsion’s ongoing investment in ThermoDox will be limited to providing clinical drug supply and modest financial support. ThermoDox is currently under investigator-sponsored development for several cancer indications.

Commencement of Enrollment in Phase 1 Study with ThermoDox and Focused Ultrasound in Pancreatic Cancer. In July 2021, Celsion GmbH announced the commencement of enrollment in Oxford University’s Phase I PanDox study with ThermoDox in conjunction with focused ultrasound in patients with pancreatic cancer.

This investigator-led study sponsored by the University of Oxford and supported by the National Institute for Health Research (NIHR) Oxford Biomedical Research Centre has now received ethics, MHRA and institutional R&D approval to commence (ClinicalTrials.gov Identifier: NCT04852367). PanDox is being carried out as a multi-disciplinary collaboration between Celsion, the Oxford University Institute of Biomedical Engineering, the Oncology Clinical Trials Office and the Oxford University Hospitals NHS Foundation Trust. Prof. Mark Middleton, M.D., Head of the Department of Oncology at the University of Oxford, is the chief clinical investigator and Prof. Constantin Coussios, FREng, Ph.D., Director of the Institute of Biomedical Engineering, is the lead scientific investigator.

The primary endpoint of the two-arm 18-subject PanDox study is enhanced uptake of doxorubicin in pancreatic tumors using ThermoDox and focused ultrasound (FUS), compared with systemic delivery of free doxorubicin. ThermoDox will be administered intravenously in 12 patients with non-resectable pancreatic ductal adenocarcinoma and locally activated by focused ultrasound-mediated hyperthermia. This will be compared with conventional systemic delivery of doxorubicin without FUS in 6 patients. Secondary endpoints include:

Comparing radiologically assessed tumor activity and response with ThermoDox and FUS to free drug alone.
Examining the impact on patient symptoms of ThermoDox plus FUS.
Assessing the safety profile of both FUS and ThermoDox.
The PanDox study is expected to be completed by December 2022 and is similar in design to Oxford’s 10-patient TARDOX study, which demonstrated that ThermoDox plus focused ultrasound increased doxorubicin tumor concentrations by up to 10-fold and enhanced nuclear drug uptake in patients with liver tumors. The findings of the TARDOX study are published in Lancet Oncology (Lyon et al., 2018) and Radiology (Gray et al., 2019).

Corporate Developments

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

Received $1.85 Million in Non-Dilutive Funding from the Sale of New Jersey State Net Operating Losses. In May 2021, the Company announced it received $1.85 million of net cash proceeds from the sale of approximately $2.0 million of its unused New Jersey net operating losses (NOLs). The NOL sales cover the 2019 tax year and are administered through the New Jersey Economic Development Authority’s (NJEDA) Technology Business Tax Certificate Transfer (NOL) Program. Additional sales of $5.0 million of unused New Jersey NOLs are anticipated in 2022 – 2024, which will further increase Celsion’s cash position on a non-dilutive basis.

New $10.0 Million Strategic Loan Facility with Silicon Valley Bank. In June 2021, the Company announced it entered into a $10.0 million loan facility with Silicon Valley Bank (SVB). Celsion immediately used $6.0 million from this facility to retire all outstanding indebtedness with Horizon Technology Finance Corporation. The remaining $4.0 million will be available to be drawn down up to 12 months after closing and will be used to fund the advancement of the Company’s product pipeline, including GEN-1 for the treatment of newly diagnosed advanced ovarian cancer, as well as other strategic initiatives intended to broaden its product pipeline. The funding is in the form of money market secured indebtedness bearing interest at a calculated WSJ Prime-based variable rate (currently 3.25%). Payments under the loan agreement are interest only for the first 24 months after loan closing, followed by a 24-month amortization period of principal and interest through the scheduled maturity date.

Appointment of Two New Directors to the Celsion Board. In June 2021, the Company announced the appointment of Stacy R. Lindborg, Ph.D. and Christine A. Pellizzari to Celsion’s Board of Directors.

Dr. Lindborg brings to Celsion more than 25 years of pharmaceutical industry experience with a particular focus on R&D, executive management and strategy. She has worked with biologics, small molecules and cell therapies to address a broad range of diseases and disorders, including multiple orphan drug products, along with extensive experience in early-stage development having taken molecules from first-in-man studies into the clinic through approval and launch. Dr. Lindborg is a graduate of Baylor University where she received a Ph.D. and M.A. in statistics and a B.A. in psychology with a minor in mathematics. A prolific researcher, she has authored more than 50 abstracts, 200 presentations and 40 manuscripts that have been published in peer-reviewed journals. She serves on several industry advisory boards related to statistics and biotechnology.

Ms. Pellizzari is Chief Legal Officer of Science 37, a developer of a leading decentralized clinical trial operating system where she has global responsibility for both legal and quality. Ms. Pellizzari brings more than 20 years of leadership in the global pharmaceutical industry to Celsion. Immediately prior to joining Science 37, Ms. Pellizzari was Chief Legal Officer of Insmed Incorporated, a global biopharmaceutical company dedicated to transforming the lives of patients with serious and rare diseases. At Insmed, Ms. Pellizzari had global responsibility for legal and government affairs including corporate governance, regulatory compliance, contracting, alliance management, clinical trial oversight, labor and employment, litigation management and intellectual property strategy and portfolio management. Ms. Pellizzari received a J.D. from the University of Colorado School of Law and a B.A. from the University of Massachusetts (Amherst). She is a member of Executive Women in Bio, Women Corporate Directors, National Association of Corporate Directors, Association of Corporate Counsel, Society for Corporate Governance and National Association of Stock Plan Professionals.

Second Quarter Financial Results

For the quarter ended June 30, 2021, Celsion reported a net loss of $5.4 million ($0.06 per share), compared with a net loss of $5.3 million ($0.18 per share) in the same period of 2020. Operating expenses were $5.2 million in the second quarter of 2021, which represented a $0.3 million (6%) increase from $4.9 million in the same period of 2020.

The Company ended the second quarter of 2021 with $64.5 million in cash, investment securities, restricted cash and accrued interest receivable. Coupled with future sales of unused New Jersey NOL’s, the Company believes it has sufficient capital resources to fund its operations through 2024.

Research and development (R&D) expenses decreased $0.4 million to $2.6 million in the second quarter of 2021 from $3.0 million in the second quarter of 2020. R&D costs associated with the development of GEN-1 to support the OVATION 2 Study as well as development of the PLACCINE DNA technology platform increased to $1.4 million in the second quarter of 2021, compared with $0.9 million in the same period of 2021. Clinical development costs for the Phase III OPTIMA Study decreased $0.4 million to $0.2 million in the second quarter of 2021, compared with $0.6 million in the second quarter of 2020, due to the discontinuation of this 556-patient trial in the first quarter of 2021. Other costs related to clinical supplies and regulatory support for the Company’s clinical development programs decreased to $0.9 million in the current quarter from $1.5 million in the second quarter of 2020, largely driven by higher manufacturing costs for GEN-1 clinical supplies for the Phase II portion of the OVATION 2 Study offset by lower regulatory and manufacturing costs related to the discontinued OPTIMA Study.

General and administrative expenses were $2.6 million in the second quarter of 2021, compared with $1.9 million in the same period of 2020. The $0.7 million increase was primarily attributable to higher non-cash stock-compensation expense ($0.1 million), an increase in legal and professional fees ($0.4 million) and an increase in Directors’ and Officers’ insurance premiums ($0.1 million) incurred during the second quarter of 2021.

In connection with the Company’s venture debt facility with Horizon Technology Finance Corporation entered in late June 2018, the Company incurred interest expense of $0.2 million during the second quarter of 2021. This compares with interest expense of $0.3 million in the comparable prior-year period. In June 2021, the Company entered into a new $10.0 million loan facility with SVB, with a portion of the proceeds used to retire all outstanding indebtedness with Horizon. The Company recognized a $0.2 million loss on this debt extinguishment.

Six Month Financial Results

For the six months ended June 30, 2021, the Company reported a net loss of $11.1 million ($0.15 per share), compared with a net loss of $10.4 million ($0.37 per share) in the same period of 2020. Operating expenses were $10.7 million during the first six months of 2021, which represented a $0.9 million (10%) decrease from $9.8 million in the same period of 2020.

Net cash used for operating activities was $7.3 million in the first six months of 2021, compared with $7.9 million in the same period in 2020. This was in line with the Company’s projected cash utilization for 2021 of approximately $17 million, or an average of approximately $4.25 million per quarter. Cash provided by financing activities was $54.8 million during the first six months of 2021 resulting from equity offerings in January 2021 and April 2021, and proceeds from the $10 million loan facility with SVB in June 2021 and the sale of the Company’s unused New Jersey NOLs in May 2021.

Research and development expenses decreased $0.8 million to $5.2 million in the first half of 2021 from $6.0 million in the first half of 2020. R&D costs associated with the development of GEN-1 to support the OVATION 2 Study as well as development of the PLACCINE DNA technology platform increased to $2.8 million in the first half of 2021, compared with $2.1 million in the comparable six-month period in 2020. Costs for the Phase III OPTIMA Study decreased $0.9 million to $0.4 million in the first half of 2020, compared with $1.3 million in the first half of 2020, due to the discontinuation of this trial in the first quarter of 2021. Other costs related to clinical supplies and regulatory support for the Company’s clinical development programs decreased $0.6 million in the first half of 2021, compared with the same prior-year period due to lower regulatory and manufacturing costs for the discontinued Phase III OPTIMA Study.

General and administrative expenses were $5.5 million in the first half of 2021, compared with $3.7 million in the same period of 2020. The $1.8 million increase was primarily attributable to higher non-cash stock-compensation expense ($0.8 million), an increase in legal and professional fees ($0.7 million) and an increase in Directors’ and Officers’ insurance premiums ($0.2 million) incurred during the six months ended June 30, 2021.

Other expenses during the first half of 2021 included a non-cash charge of $0.1 million for the change in valuation of the earn-out milestone liability for the GEN-1 ovarian product candidate, compared with a non-cash charge of $0.3 million during the comparable prior-year period. In connection with the Company’s venture debt facility with Horizon entered in late June 2018, the Company incurred interest expense of $0.4 million during the first six months of 2021, compared with $0.7 million during the same six-month period in 2020.

Second Quarter Conference Call

The Company will host a conference call to provide a business update, discuss its second quarter 2021 financial results and answer questions at 11:00 a.m. EDT today. To participate in the call, interested parties may dial 1-800-353-6461 (Toll-Free/North America) or 1-334-323-0501 (International/Toll) 10 minutes before the call is scheduled to begin, and ask for the Celsion Corporation Second Quarter 2021 Earnings Call (Conference Code: 2901622). The call will also be broadcast live on the internet at www.celsion.com. The call will be archived for replay through August 26, 2021. The replay can be accessed at 1-719-457-0820 or 1-888-203-1112 using Conference ID: 290622. 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 Thursday, August 12, 2021.

On August 12, 2021 INmune Bio, Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, reported that Raymond J. Tesi, MD, President and CEO, will present at the Canaccord Genuity 41st Annual Growth Conference being held August 10 – 12 (Press release, INmune Bio, AUG 12, 2021, View Source [SID1234586511]).

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Canaccord Genuity 41st Annual Growth Conference

Presentation Date: Wednesday, August 11, 2021
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Webcast: View Source

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