On September 8, 2020 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, reported that Aoife Brennan, M.B. Ch.B., Synlogic’s president and chief executive officer, and other members of the executive team will present at the following virtual banking conferences (Press release, Synlogic, SEP 8, 2020, View Source [SID1234564739]):

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HC Wainwright 22nd Annual Global Investor Conference: The Company will present at 12:00 noon on Tuesday, September 15, 2020.
Oppenheimer Fall Healthcare Life Sciences & Med Tech Summit: The Company will present at 4.10 pm on Wednesday, September 23, 2020
Jefferies IBD Therapeutics Summit: The Company will present at a time to be determined on Wednesday, September 23, 2020.
These are virtual events. A live webcast of the presentations can be accessed under "Event Calendar" in the Investors & Media section of the Company’s website. An archived copy of the webcast will be available on the Synlogic website for approximately 30 days after the event.

On September 8, 2020 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported first positive data from the Phase 1 clinical trial evaluating the intravenous administration of TG6002 in patients with advanced gastrointestinal tumors (Press release, Transgene, SEP 8, 2020, View Source [SID1234564738]).

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The trial aims at confirming the good tolerability of ascending doses of intravenous TG6002 and at providing the first translational data from this novel route of administration.

The independent safety review committee met and recommended that the trial be continued and that the dose level be increased to a 3×109 pfu in the absence of dose-limiting toxicity of TG6002 at the 109 pfu dose.
The first translational data show that, when administered intravenously, TG6002 circulates transiently in the patient’s bloodstream and induces the production of 5-FU at therapeutic doses. The production of 5-FU results from the expression of the FCU1 gene, integrated into the TG6002 genome, as the virus replicates selectively in the tumor cells.
TG6002 has been engineered to combine the oncolytic and immunogenic actions of the virus with the intra-tumoral production of 5-FU, a chemotherapeutic agent. This production is achieved by converting the pro-drug 5-FC (administered orally) into 5-FU, which is expected to reach high concentrations within the tumor. This mechanism of action is based on the expression of the proprietary FCU1 gene that has been integrated with the genome of TG6002.

"This is the first clinical study demonstrating that Transgene’s Vaccinia Virus from the Invir.IO platform is able to reach the tumor when administered intravenously. Importantly, through the production of 5-FU at therapeutic doses, we have also demonstrated the functionality of the FCU1 transgene integrated into the genome of TG6002. These positive initial translational data, combined with a satisfactory tolerability profile, have allowed us to continue this Phase 1 clinical trial and evaluate higher doses of this promising new drug candidate," said Dr. Maud Brandely, MD, PhD, Chief Medical Officer of Transgene.

With this clinical trial, Transgene aims to demonstrate the benefits of intravenous administration of next generation oncolytic viruses such as TG6002 and the candidates derived from the Invir.IO platform, in order to extend the use of these therapies to many solid tumors, including gastrointestinal cancers. This broad utility contrasts with first-generation oncolytic viruses that have only been approved to be given via intra-tumoral administration, which restricts their use to easily accessible tumors.

TG6002 is also being evaluated in another Phase 1/2a clinical trial where it is being given by Intrahepatic Artery Infusion in patients with advanced colorectal cancer with liver metastases (CRLM). In order to respond to the Covid-19 pandemic, the trial’s clinical site had temporarily suspended patient inclusion; recruitment resumed in early September 2020.

About the trial

This trial is a single-arm open-label Phase 1/2 trial evaluating the safety and tolerability of multiple ascending doses of TG6002 administered intravenously in combination with oral 5-FC, a non-cytotoxic pro-drug that can be converted in 5-FU, its active metabolite. Based on the safety profile of TG6002, several dose levels have been added to the initial Phase 1 clinical protocol. At the end of this Phase 1 part, Phase 2 patients will receive the recommended dose of TG6002. The trial has safety as primary endpoint for the Phase 1 part and efficacy for the Phase 2 part. The trial also evaluates pharmacokinetic properties and biodistribution of TG6002, along with immune modulation of the tumor micro-environment. This European study will enroll up to 40 patients suffering from advanced gastrointestinal carcinomas who have failed and/or are intolerant to standard therapeutic options in the Phase 1 part. Patients with colon cancer and liver metastases will be enrolled in the phase 2 part.

Dr. Philippe Cassier, M.D., PhD, head of the early-phase trials unit at Centre Léon Bérard (Lyon, France) is the principal investigator of the trial.

About TG6002

TG6002 has been engineered to directly kill cancer cells (oncolysis), to enable the production of a chemotherapy agent (5-FU) within the tumor, and to elicit an immune response by the body against the tumor cells. In preclinical experiments, TG6002 has been shown to induce the shrinkage of the primary tumor as well as the regression of distant metastases1.

The production of 5-FU directly in the tumor aims at achieving a better anti-tumor effect with limited chemotherapy-induced side effects.

­ TG6002 induces the production of 5-FU in the cancer cells it has infected, by enabling the local conversion of the pro-drug 5-FC (administered orally) into 5-FU. 5-FU is a common chemotherapy for patients with CRLM. This mechanism of action is based on the expression of the proprietary FCU1 gene that has been integrated with the genome of TG6002, as the virus replicates selectively in the tumor cells.
­ 5-FU is associated with side effects that can lead to treatment discontinuation. With TG6002, 5-FU is produced within the tumor where it is expected to be present at a high concentration level in contrast to the very low levels anticipated in the rest of the patient’s body.

On September 8, 2020 Innovation Pharmaceuticals (OTCQB:IPIX) ("the Company"), a clinical stage biopharmaceutical company, reported the publication of independent research, in Oncology Reports, a leading oncology journal, supporting the therapeutic potential of Kevetrin, the Company’s p53-modulating anti-cancer drug candidate, in treating Acute Myeloid Leukemia (AML) (Press release, Innovation Pharmaceuticals, SEP 8, 2020, View Source [SID1234564737]). AML accounts for approximately one-quarter to one-third of all leukemias worldwide and has a 5-year survival rate of only 24 percent.

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In the recently published Kevetrin AML research, linked below, Kevetrin treatment was shown to arrest cell growth and cause cell death (apoptosis) in AML cells. Multiple p53 targets, several cellular processes and oncogenic pathways were also shown to be positively impacted by Kevetrin. Kevetrin was further shown to exhibit preferential cytotoxic activity against leukemia blast cells, while largely not affecting the immune microenvironment, supporting a less toxic drug profile.

The researchers concluded: "These findings suggest that Kevetrin may be a promising therapeutic option for patients with both wild-type and TP53-mutant AML."

"These compelling research findings, by independent researchers, confirm Kevetrin’s treatment potential in AML—a serious blood cancer in great need of novel treatments," commented Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. "A Phase 2a clinical trial of Kevetrin in late-stage platinum-resistant ovarian cancer revealed intra-tumor p53 modulation. The Company is planning to formulate Kevetrin for oral delivery, leveraging drug pharmacokinetics and enhancing ease of use. These new published findings provide valuable insights into Kevetrin’s anti-cancer properties."

As a gene-based therapy, should Kevetrin demonstrate efficacy in clinical testing, its regulatory pathway to approval might be expedited. The Food and Drug Administration (FDA) has signaled strong support for development of gene therapies. Two gene therapies for cancers of the blood (Kymriah and Yescarta) were approved for genetically-driven diseases based primarily on results from mid-stage, single-arm, open-label clinical trials. FDA’s efficacy determination for Kymriah was based on 92 evaluable patients, and 108 evaluable patients for Yescarta.

The Company will keep shareholders apprised of developments in our Kevetrin program. In separate news, the Company will be submitting a briefing package shortly to the FDA in preparation for our planned clinical study of Brilacidin for COVID-19.

· Napolitano R, et al. "Kevetrin Induces Apoptosis in TP53 Wild‑Type and Mutant Acute Myeloid Leukemia Cells." Oncol Rep. 2020 Oct; 44(4): 1561–1573.
View Source
View Sourcepdf/or-44-04-1561.pdf (pdf)

About Kevetrin and p53

Kevetrin is a small molecule that has demonstrated the potential of becoming a breakthrough cancer treatment by modulating p53, a protein frequently referred to as the "Guardian of the Genome" due to its critical role in controlling cell mutations. In a majority of cancers, the p53 pathway is mutated, preventing the body from performing its natural anti-tumor functions. Kevetrin, by intravenous dosing, has successfully completed a Phase 1 clinical trial in advanced solid tumors and a Phase 2a clinical trial in late-stage ovarian cancer. The company plans on completing oral toxicology studies and changing the dosing to an oral formulation (tablet or capsule), subject to available financial resources. The Food and Drug Administration (FDA) has awarded Orphan Drug status for Kevetrin in ovarian cancer, pancreatic cancer, and retinoblastoma, qualifying it for developmental incentives and market exclusivities upon any future regulatory approval. The FDA also has granted Kevetrin Rare Pediatric Disease designation for childhood retinoblastoma.

On September 8, 2020 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform, ImmTOR, reported that Selecta’s Chief Executive Officer, Carsten Brunn, Ph.D., will participate virtually in the following investor conferences in September (Press release, Selecta Biosciences, SEP 8, 2020, View Source [SID1234564735]):

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H.C. Wainwright & Co. 22nd Annual Global Investment Conference 2020
Date: Monday, September 14, 2020
Presentation Time: 3 p.m. ET

Cantor Fitzgerald Virtual Global Healthcare Conference
Date: Wednesday, September 16, 2020
Presentation Time: 1:20 p.m. ET

Live webcasts will be available in the Investors & Media section of the company’s website at www.selectabio.com.

On September 8, 2020 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported an update on the OVATION 2 Study with GEN-1 in advanced ovarian cancer patients (Press release, Celsion, SEP 8, 2020, View Source [SID1234564734]). Celsion also announced it has entered into a common stock purchase agreement for the issuance and sale, from time to time, of up to $26 million of shares of common stock with Lincoln Park Capital Fund, LLC (LPC), a Chicago-based institutional investor.

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Stock Purchase Agreement

In connection with the execution of the purchase agreement, LPC made an initial purchase of $1 million of common stock at $1.00 per share, representing a significant premium to the current market price.

Under the terms of the new purchase agreement with LPC, Celsion has the right at its sole discretion, but not the obligation, to sell to LPC up to $26 million worth of shares (including the $1 million initially purchased) over the 36-month term of the agreement, subject to certain conditions. There are no upper limits to the price per share LPC may pay to purchase the shares, and the purchase price of the shares will be based on the prevailing market prices at the time of each sale to LPC. Celsion controls the timing and amount of any future sales of its stock to LPC.

There are no warrants, derivatives, financial or business covenants associated with the agreement, and LPC has agreed not to cause or engage in any direct or indirect short selling or hedging of Celsion’s common stock. Celsion may terminate the purchase agreement at any time, at its discretion, without any cost or penalty. In consideration for LPC entering into the purchase agreement, Celsion issued shares of its common stock to LPC as a fee for LPC’s obligation to purchase shares at the Company’s discretion.

"The LPC financing, along with the anticipated sale of New Jersey net operating losses later this year, the repayment and restructure of our long-term debt with Horizon announced last week and our current cash position of approximately $20 million, will help us reach a number of value-rich events including full enrollment of the OVATION 2 Study," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "These funds will also allow us to complete the definitive analysis of the OPTIMA Study, our Phase III trial with ThermoDox in primary liver cancer. We continue to follow patients for overall survival, and to compile data and CT scans to submit to the National Institutes of Health for their review and perspective," Mr. Tardugno added.

Celsion intends to use any net proceeds from the sale of its common stock to LPC to advance its product pipeline and for general corporate purposes. Additional information regarding the purchase agreement with LPC is available in the Current Report on Form 8-K that Celsion will file with the Securities and Exchange Commission.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy any shares of common stock, nor shall there be any sale of shares of common stock in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction.

OPTIMA Study Update

Celsion continues to follow patients for overall survival (OS) in its Phase III study of ThermoDox for newly diagnosed hepatocellular carcinoma (HCC) patients, noting that the unexpected and marginally crossed futility boundary, suggested by the Kaplan-Meier analysis at the second interim analysis on July 9, 2020, may be associated with a data maturity issue. The Company has hired independent statisticians to further evaluate the trial data, the statistical plan, and the hypothesis generating data from the earlier HEAT Study, as well as, supplying data to the NIH for independent analysis and recommendation. The Company expects to announce its plans for the OPTIMA Study before year end.

OVATION 2 Study Update

Enrollment in the Phase II portion of the Phase I/II OVATION 2 Study has been accelerated with 22 patients enrolled thus far at 13 sites in the U.S. The Company expects 12 additional sites in the U.S. and Canada to begin enrolling patients by the end of the year, and now expects full enrollment of 118 patients to be completed by the end of the second quarter of 2021, a full quarter earlier than previously anticipated.

GEN-1 was designed using TheraPlas, Celsion’s proprietary, synthetic, non-viral nanoparticle delivery system platform. It is an interleukin-12 (IL-12) DNA plasmid vector associated with a non-viral nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein.

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

Because OVATION 2 is an open-label study, the Company intends to provide clinical updates throughout the course of treatment, including response rates and surgical resection scores.

Dr. Nicolas Borys, Celsion’s chief medical officer, said, "We are pleased with the pace of enrollment in this trial, and attribute the high level of interest among investigators and patients to the impressive data generated from prior studies. In particular, data from the Phase I portion of the OVATION 2 Study that showed successful tumor resections, with seven out of eight patients (87.5%) in the GEN-1 treatment arm having a complete tumor resection (R0), which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed. The NACT-only treatment arm had an R0 resection rate of 50%."

"In addition, investigators have expressed confidence in the role of IL-12 and GEN-1’s safety profile. The Data Safety Monitoring Board recommended that the Phase II portion of the OVATION 2 Study proceed with a GEN-1 dose of 100 mg/m², and with more than 17 doses over more than six-months of treatment. We look forward to providing tumor response data and surgical results as they become available throughout the course of treatment. In addition, we believe that PFS as an endpoint bodes well for study success," Dr. Borys added.