On July 27, 2020 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that its partner, Austrianova, has submitted a Drug Master File (DMF) to the U.S. Food and Drug Administration (FDA) (Press release, PharmaCyte Biotech, JUL 27, 2020, View Source [SID1234562408]). The DMF provides all confidential and detailed information covering the production of the CypCaps final product, which was produced by Austrianova and will be used in PharmaCyte’s planned clinical trial in locally advanced, inoperable pancreatic cancer (LAPC).

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PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said, "We’ve reached an exciting milestone at PharmaCyte that has us on the precipice of reaching our long-awaited goal of submitting an Investigational New Drug Application (IND). We’re elated to announce that our partner, Austrianova, submitted a DMF with the FDA for the production of our Cell-in-a-Box encapsulated cell product CypCaps. This DMF is an important and significant event since it is the last prerequisite for the formal FDA application process. It will support and now facilitate the submission of our IND."

Austrianova’s Chief Technical Officer, Walter H. Gunzburg stated, "The DMF filing is a key event for both PharmaCyte and Austrianova since it will provide the basis for many Cell-in-a-Box products in addition to CypCaps. The DMF is by its nature a comprehensive document compiled from many months of in-house work and past historical data and was compiled with the support and advice of an external consultant."

A DMF is submitted to the FDA to provide detailed information about facilities, processes and materials used in the manufacturing, processing and packaging of human drugs and biologics. It is a prerequisite to securing approval and commercialization and ensures confidentiality of proprietary information related to the Active Pharmaceutical Ingredient (API). The information contained in a DMF is used to support, among other applications, an IND.

The DMF requirements are complex and specific, encompassing every detail involved with the manufacture of the API – from raw materials to analytical methods, process development and optimization. The scrutiny goes all the way back to the starting materials used in the API.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced, inoperable pancreatic cancer, we encourage you to watch the company’s documentary video complete with medical animations at: View Source

On July 27, 2020 Celsion Corporation (NASDAQ: CLSN), an oncology focused drug-development company, reported the randomization of the first two patients in the Phase II portion of the Phase I/II OVATION 2 Study with GEN-1 in advanced ovarian cancer (Press release, Celsion, JUL 27, 2020, View Source [SID1234562407]). The Company anticipates completing enrollment of up to 118 patients in the third quarter of 2021. Because this is an open-label study, the Company intends to provide clinical updates throughout the course of treatment including response rates and surgical resection scores.

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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).

"During the first half of 2020, we reported 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%," said Dr. Nicolas Borys, Celsion’s Chief Medical Officer. "The Company engaged Medidata/Acorn to independently evaluate our data using a propensity matched synthetic control arm (SCA) with results from the Company’s previously completed Phase I studies. The results suggested that GEN-1 nearly doubled progression-free survival (PFS) in these patients. These findings are not statistically significant, however, due to the small number of patients, but nonetheless are encouraging and supportive of our current Phase II study."

"This past May we reported that 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," said Michael H. Tardugno, Chairman, President and Chief Executive Officer of Celsion. "Patients will undergo up to 6 months of immunostimulatory GEN-1 treatment. Based on results from earlier studies, we believe this regimen holds great potential to alter the current treatment paradigm, and in doing so improve survival for ovarian cancer patients, whose prognosis is generally poor. Our investigators are among the leading researchers in ovarian cancer. Together, we are delighted to have begun enrolling patients in the Study and look to achieve our goal of providing new novel therapeutic options to patients with this difficult-to-treat cancer."

In March 2020 the Company announced the European Medicines Agency Committee for Orphan Medicinal Products recommended that GEN-1 be designated as an orphan medicinal product for the treatment of ovarian cancer. GEN-1 previously received orphan designation from the U.S. Food and Drug Administration for the treatment of ovarian cancer. This designation by the EMA provides 10 years of exclusivity once approved for marketing.

About GEN-1 Immunotherapy

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

On July 27, 2020 INOVIO (NASDAQ: INO) reported that second quarter 2020 financial results will be released after the market close on August 10, 2020 (Press release, Inovio, JUL 27, 2020, View Source [SID1234562406]). Following the release, the Company will host a live conference call and webcast at 4:30 p.m. ET to discuss financial results and provide a general business update, including the company’s ongoing vaccine developments for COVID-19.

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A live and archived version of the audio presentation will be available online at View Source This is a listen-only event but will include a live Q&A with analysts.

Telephone replay will be available approximately one hour after the call at 877-344-7529 (US toll free) or 412-317-0088 (international toll) using replay access code 10146894.

On July 27, 2020 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that it will host a call with a key opinion leader (KOL) and members of the Oncternal management team to discuss the current treatment landscape of mantle cell lymphoma (MCL) and updates on the use of cirmtuzumab to treat patients with MCL on Wednesday, July 29, 2020 at 1:30 p.m. Eastern Time (Press release, Oncternal Therapeutics, JUL 27, 2020, View Source [SID1234562405]).

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The call will feature a scientific presentation on the current treatment landscape and unmet medical need in treating patients with MCL by Michael Wang, MD, professor of Lymphoma & Myeloma at The University of Texas MD Anderson Cancer Center. Dr. Wang, a consultant to Oncternal, will be available to answer questions at the conclusion of the call.

Oncternal’s management team will also discuss the recently announced updates to the clinical program of cirmtuzumab, a first-in-class humanized monoclonal antibody that binds with high affinity to a biologically important epitope on ROR1 (Receptor-tyrosine kinase-like Orphan Receptor 1). Cirmtuzumab is currently in a Phase 1/2 clinical trial in combination with ibrutinib for treating patients with relapsed/refractory MCL and patients with CLL. After recently announced positive data in MCL presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Annual Meeting in May, the Company is amending the study to increase enrollment of MCL patients in the Phase 2 expansion cohort to at least 20 patients and to allow the enrollment of patients with a broader range of prior BTK inhibitor treatments.

To register for the call, click here.

About Cirmtuzumab

Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of MCL or CLL, in a collaboration with the University of California San Diego School of Medicine and the California Institute for Regenerative Medicine (CIRM). In addition, an investigator-initiated Phase 1 clinical trial of cirmtuzumab in combination with paclitaxel for women with HER2-negative metastatic breast cancer is being conducted at the UC San Diego School of Medicine.

ROR1 is a potentially attractive target for cancer therapy because it is an onco-embryonic antigen – not usually expressed on adult cells, and its expression confers a survival and fitness advantage when reactivated and expressed by tumor cells. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to specifically targeting ROR1 expressing tumors. This led to the development of cirmtuzumab, that binds this critical epitope of ROR1, which is highly expressed on many different cancers but not on normal tissues. Preclinical data showed that when cirmtuzumab bound to ROR1, it blocked Wnt5a signaling, inhibited tumor cell proliferation, migration and survival, and induced differentiation of the tumor cells. The FDA has granted Orphan Drug Designations to cirmtuzumab for the treatment of MCL and CLL/small lymphocytic lymphoma. Cirmtuzumab is in clinical development and has not been approved by the FDA for any indication.

On July 27, 2020 Mateon Therapeutics (OTC.QB: MATN) reported the closing of the 1st tranche of financing related to the Mateon operations and spinoff of its EdgePoint AI, Inc. ("EdgePoint", a Delaware Corporation and a division of Mateon) (Press release, Mateon Therapeutics, JUL 27, 2020, View Source [SID1234562404]).

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JH Darbie & Co., Inc. is acting as the exclusive placement agent for the Offering. The Offering consists of a minimum of 40 units on a best effort all or none basis and a maximum of up to 100 units on a best effort basis at a price per unit of $50,000 (the "Units"). This initial closing is for the sale of 40 Units. Each Unit allows the Unit holder to purchase 25,000 shares of the Common Stock of EdgePoint (Mateon’s artificial intelligence ("AI") division) and one note issued by Mateon (the "Note"). Each Note is convertible into up to 25,000 shares of EdgePoint’s Common Stock (conversion price $1.00 per share) or up to 138,889 shares of Mateon’s Common Stock (conversion price $0.18 per share). Each Unit also consists of 100,000 warrants (the "Warrants"), 50,000 Warrants, each to purchase one share of EdgePoint’s Common Stock at $1.00 per share, and 50,000 Warrants, each to purchase one share of Mateon’s Common Stock at $0.20 per share.

The full exercise of the warrants will bring in an additional $2.0 million for EdgePoint and $0.4 million for Mateon. Mateon will be registering, with the Securities and Exchange Commission, for the spinoff of EdgePoint, as a publicly traded AI powered blockchain technology company. Early investors in EdgePoint include Silicon Valley veteran Balaji Baktha.

"The spinoff of EdgePoint AI is part of our continuing effort at unlocking the value within Mateon," said Dr. Vuong Trieu, CEO of Mateon. "This financing establishes a strong investor base for EdgePoint AI in preparation for its IPO as a fully independent AI technology company. Recent IPOs of AI technology companies been highly successful and we will be working diligently to ensure the success of EdgePoint AI."

This press release does not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein in any manner. There shall not be any offer, solicitation of an offer to buy, or sale of securities in any state or jurisdiction in which such an offering, solicitation, or sale would be unlawful prior to registration or qualification in accordance with the securities laws of any such state or jurisdiction.