On April 20, 2017 Celsion Corporation (NASDAQ:CLSN), an oncology drug development company, reported an update on its OVATION Study, a Phase Ib dose escalating clinical trial combining GEN-1, the Company’s DNA-based immunotherapy, with the standard of care for the treatment of newly-diagnosed patients with advanced (stage III/IV) ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery (Press release, Celsion, APR 20, 2017, View Source [SID1234518637]). GEN-1 is an IL-12 DNA plasmid vector formulated as a nanoparticle in a non-viral delivery system to cause the sustained local production and secretion of the Interleukin-12 (IL-12) protein loco-regionally to the tumor site. Schedule your 30 min Free 1stOncology Demo! The Company announced that an abstract for the OVATION Study has been accepted for presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2017 Annual Meeting, which will take place from June 2-6 at McCormick Place in Chicago, IL.
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
The abstract, entitled "Phase 1 study of the safety and activity of formulated IL-12 plasmid administered intraperitoneally in combination with neoadjuvant chemotherapy in patients with newly diagnosed advanced stage ovarian cancer," will be presented in a poster presentation session on Saturday, June 3rd from 1:15 PM to 4:45 PM by Dr. Premal H. Thaker, Associate Professor, Obstetrics and Gynecology Division of Gynecologic Oncology, Washington University in St. Louis School of Medicine.
The presentation will summarize clinical findings and translational data from all available patients treated in the trial. The translational data will provide further insight into GEN-1’s mechanism of action through the evaluation of dose-related changes in the tumor and peritoneal immune cell population, as well as through the peritoneal cytokine levels.
Celsion has previously reported highly encouraging interim data from the first twelve patients who have completed treatment in the OVATION Study. GEN-1 plus standard chemotherapy produced excellent results, with no dose limiting toxicities and promising dose dependent efficacy signals which appear to correlate well with successful surgical outcomes as summarized below:
Of the twelve patients treated, one patient demonstrated a complete response, eight (8) patients demonstrated a partial response and three (3) patients demonstrated stable disease, as measured by RECIST criteria. This translates to a 100% disease control rate ("DCR") and a 75% objective response rate ("ORR").
Eleven patients had successful resections of their tumors, with six (6) patients (55%) having an R0 resection, which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed, and four (4) patients (36%) having a R1 resection, indicating microscopic residual tumor.
One patient demonstrated a pathological complete response (pCR). pCRs are typically seen in less than 7% of patients receiving neoadjuvant chemotherapy followed by surgical resection, and have been associated with a median overall survival (OS) of 72 months, which is more than three years longer than those who do not experience a pCR.
All patients experienced a dramatic decrease in their CA-125 protein levels as of their most recent study visit. CA-125 is used to monitor certain cancers during and after treatment. CA-125 is present in greater concentrations in ovarian cancer cells than in other cells.
"We have now completed enrollment of our OVATION Study in newly diagnosed ovarian cancer patients, one goal of which is to determine GEN-1’s activity in combination with standard chemotherapy. The previously reported remarkable, unexpected surgical outcomes among all patients completing the prescribed eight weekly treatments reinforce our confidence in the promise of GEN-1’s ability to work safely and effectively in advanced ovarian cancer," said Michael H. Tardugno, Celsion’s chairman, president and CEO. "We are looking forward to Dr. Thaker’s report of comprehensive findings at the upcoming ASCO (Free ASCO Whitepaper) meeting and to learning more about the utility of our gene-based immunotherapy approach as this important study matures."
OVATION Study Design
The Phase Ib trial will evaluate weekly intraperitoneal dosing of GEN-1 in combination with neoadjuvant chemotherapy, the standard of care for patients newly diagnosed with ovarian cancer. Concurrently with neoadjuvant chemotherapy, enrolled patients will receive escalating weekly doses of GEN-1, from levels beginning at 36mg/m², to 47mg/m², 61mg/m² and 79mg/m² weekly for 8 treatments in total, with interval debulking surgery to follow. The regimen will primarily be evaluated for its safety and tolerability.
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 in patients with peritoneally metastasized ovarian cancer, and recently completed a Phase Ib trial of GEN-1 in combination with PEGylated doxorubicin in patients with platinum-resistant ovarian cancer.
Spherix Announces Economic Interest Acquired in New Patent Portfolio
On April 19, 2017 Spherix Incorporated (Nasdaq: SPEX) an intellectual property development company committed to the fostering of technology and monetization of intellectual property, reported that it has acquired an economic interest in a new patent portfolio that is largely unlicensed to any third party (Press release, Spherix, APR 19, 2017, View Source [SID1234538993]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
Spherix and its partner Equitable IP Corporation have acquired an economic interest and the right to monetize a valuable portfolio in the fiber optic technology space as part of their ongoing patent monetization efforts. The newly acquired portfolio consists of approximately 112 patents and is largely unlicensed.
Anthony Hayes, Chief Executive Officer of Spherix, stated, "By working with Equitable IP Corporation, we’ve expanded the number of Equitable’s assets that can be monetized to now over 400 patents. The addition of these new patents to Equitable’s portfolio increases our potential revenue generation. This is part of our continued efforts to increase value for our shareholders."
CytRx Announces FDA Agreement on Regulatory Pathway to Approval for Aldoxorubicin in Soft Tissue Sarcomas
On April 19, 2017 CytRx Corporation (NASDAQ: CYTR) reported the U.S. Food and Drug Administration (FDA) has reached an agreement with CytRx on preparations for a New Drug Application (NDA) submission for aldoxorubicin in soft tissue sarcomas (STS). STS remains a high unmet medical need (Press release, CytRx, APR 19, 2017, View Source [SID1234518622]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"We are very pleased to have achieved clarity from the FDA regarding CytRx’s soft tissue sarcoma program," said Daniel Levitt, MD, Ph.D., Chief Operating Officer and Chief Medical Officer. "The FDA agreed that CytRx could use the application pathway for its filing that has been successfully used previously by the oncology drugs Abraxane, Doxil and Onivyde. Our interaction with the FDA was part of a continued collaborative and productive relationship with the Agency. We look forward to providing the study reports and analysis that can lead to the approval of aldoxorubicin for the treatment of patients with soft tissue sarcomas."
The Company’s goal is to submit a rolling NDA under section 505(b)(2) to the FDA for soft tissue sarcomas in the last quarter of 2017. CytRx also plans to discuss with the European Medicines Agency (EMA) a path to filing a Marketing Authorization Application (MAA). The commercial launch of aldoxorubicin is still projected for 2018 in the United States. Aldoxorubicin has received Orphan Drug Designation by the FDA for the treatment of STS. Orphan designation provides several benefits including seven years of market exclusivity after approval, certain R&D related tax credits and protocol assistance by the FDA. European regulators granted aldoxorubicin Orphan Medicinal Product Designation for STS which confers ten years of market exclusivity among other benefits.
The proposed product label would include the treatment of soft tissue sarcomas. New data could allow future use of aldoxorubicin in neoadjuvant (pre-surgery) settings, as well as a replacement for doxorubicin in combinations. CytRx is also working on a market expansion strategy which could include other indications for aldoxorubicin including combinations with other chemotherapeutics and immunotherapies.
CytRx is under confidentiality agreements with a number of companies for a commercial partnership for the marketing of aldoxorubicin. The Company believes those active discussions may be further advanced by this latest news.
About a 505(b)(2) New Drug Application
A new drug application (NDA) under the Food and Drug Administration’s (FDA) section 505(b)(2) is for a new drug containing similar active ingredients as a previously approved drug. According to the publication Regulatory Focus, a drug reviewed under 505(b)(2) represents a modified version of a previously approved product that requires additional clinical and nonclinical studies, other than bioavailability/bioequivalence studies, to demonstrate safety and efficacy. Such an application differs from a typical NDA in that the sponsor can rely on, at least in part, the FDA’s findings of safety and/or effectiveness for a previously approved reference drug.
About the Phase 2b and Phase 3 Clinical Trials
The Phase 2b trial involved 123 patients at 31 sites. Patients with advanced soft tissue sarcomas were randomized 2:1 to receive either 350 mg/m2 of aldoxorubicin (83 patients) or 75 mg/m2 of doxorubicin (40 patients) every 3 weeks for up to 6 cycles. The trial was designed to compare aldoxorubicin directly with doxorubicin.
The randomized, controlled Phase 3 trial enrolled a total of 433 patients at 79 clinical sites. Patients with metastatic, locally advanced or unresectable soft tissue sarcomas who had either not responded to, or who had progressed following treatment with one or more systemic regimens of non-adjuvant chemotherapy were randomized 1:1 to be treated with aldoxorubicin or the investigator’s choice of an approved chemotherapeutic regimen, including doxorubicin, ifosfamide, dacarbazine, pazopanib (Votrient), or gemcitabine plus docetaxel.
About Soft Tissue Sarcoma
Soft tissue sarcoma is a cancer occurring in muscle, fat, blood vessels, tendons, fibrous tissues and connective tissue. It can arise anywhere in the body at any age. STS remains a high unmet medical need because of the difficulty in treating the more than 50 types of this aggressive cancer. According to the American Cancer Society, in 2016 more than 12,300 new cases were diagnosed in the U.S. and approximately 5,000 Americans died from this disease. In addition, approximately 40,000 new cases and 13,000 deaths in the U.S. and Europe are part of a growing underserved market.
About Aldoxorubicin
Aldoxorubicin is a rationally engineered cytotoxic which combines doxorubicin, a widely used chemotherapeutic agent, with a novel linker molecule that binds directly and specifically to circulating albumin, the most abundant protein in the bloodstream. Protein-hungry tumors concentrate albumin, which facilitates the delivery of the linker molecule with the attached doxorubicin to tumor sites. In the acidic environment of the tumor, but not the neutral environment of healthy tissues, doxorubicin is released. Typically, doxorubicin is delivered systemically and is highly toxic, which limits its dose to a level below its maximum therapeutic benefit. Doxorubicin also is associated with many side effects, especially the potential for damage to heart muscle at cumulative doses greater than 450 mg/m2. Using this acid-sensitive linker technology, aldoxorubicin delivers greater doses of doxorubicin (3 ½ to 4 times). To date, there has been no evidence of clinically significant effects of aldoxorubicin on heart muscle, even at cumulative doses of the drug well in excess of 5,000 mg/m2. Aldoxorubicin is the first-ever single agent to show superiority over doxorubicin in a randomized clinical trial in first-line STS.
CRT Pioneer Fund, Cancer Research UK and NCI collaborate to boost research against the ‘undruggable’ RAS
On April 19, 2017 Cancer Research UK and the Cancer Research Technology Pioneer Fund (CPF)* reported that they have committed £2.5 million in collaboration with the National Cancer Institute (NCI) in the US to tackle one of the toughest challenges in cancer that has thwarted researchers for more than 30 years (Press release, Cancer Research Technology, 19 19, 2017, View Source [SID1234523166]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
Scientists will develop and test promising new molecules for targeting RAS, one of the most common driving mutations in aggressive, hard to treat cancers including pancreatic and lung cancer.
Scientists at the NCI in Frederick, Maryland, USA will work with the Drug Discovery Unit at the Cancer Research UK Beatson Institute** in Glasgow, Scotland to develop gold standard tests to analyse these novel RAS inhibitors.
This new collaboration links up with the NCI’s RAS Initiative*** which brings scientists together from around the globe to help develop drugs targeting the faulty protein. Launched in 2013, the initiative has established a hub of expertise that supports the international community in developments that could have huge clinical benefit.
The CRT Pioneer Fund, managed by Sixth Element Capital, will be responsible for the commercial exploitation of compounds that arise from the collaboration.
For decades, scientists have been attempting to target RAS,**** but with little success. This is because RAS lacks an obvious site on its surface for potential drug molecules to fit into and inhibit its signalling.
Dr Martin Drysdale, head of the Drug Discovery Unit at the Cancer Research UK Beatson Institute, said: "Our team is determined to challenge the dogma that RAS is ‘undruggable.’ This collaboration is our biggest yet and will double our resource targeting RAS. We are excited to be joining forces with the NCI in their pioneering RAS Initiative."
"Instead of scientists working and thinking in isolation, the NCI has created a research hub to pull together all the best science and expertise. My team is looking forward to contributing and working with Dr Frank McCormick, who leads the RAS Initiative and who has been at the forefront of cancer science for many years."
Dr Frank McCormick, who directs the research efforts of the RAS Initiative at the Frederick National Laboratory for Cancer Research, sponsored by the NCI, said: "We’re making progress in our understanding of how RAS proteins function at the molecular level and how they form signalling complexes in membranes. New technologies and tools mean we can now analyse these proteins in ways that were not possible a few years ago, and can now test new ways of blocking RAS function."
Dr Iain Foulkes, chief executive officer of Cancer Research Technology and executive director of research and innovation at Cancer Research UK, said: "It’s crucial that we unite the brightest minds across the globe. This international collaboration and investment could herald a new era in targeting RAS.
"We hope to develop these small molecules to pave the way for potential drugs in the future. Our aim is to work alongside industry to ensure any progress makes its way into clinical trials."
Dr Robert James, Managing Partner at Sixth Element Capital, said: "The CRT Pioneer Fund was established to invest in outstanding science that has the potential to benefit patients on a global scale. We are delighted to have catalysed this relationship which has created an opportunity to make real progress in discovering drugs against RAS, one of the most important oncogenes in cancer."
VLP Therapeutics Receives Notice of Allowance of Key U.S. composition of matter Patent for immune checkpoint PD-1/PD-L1/PD-L2 inserted alphavirus VLP
On April 19, 2017 VLP Therapeutics, LLC. ("VLP"), a biotechnology company focusing on the research and development of therapeutic and preventative vaccines and antibody agents, reported that it successfully received a notice of allowance from the U.S. Patent and Trademark Office for the composition of matter patent to cover immune checkpoint PD-1/PD-L1/PD-L2 inserted alphavirus VLP (Press release, VLP Therapeutics, APR 19, 2017, View Source [SID1234519826]). The patent will be issued on May 2nd. The patent protects key composition of matter of VLP’s proprietary i-α virus like particles platform technology, and the pharmaceutical composition and vaccine for use in the treatment of cancer. Utilizing the platform technology covered by this patent and other patent estates, VLP is currently focused on developing preventative and therapeutic cancer vaccines as well as next generation of targeted antibody agents. Schedule your 30 min Free 1stOncology Demo! "We are delighted to have achieved this milestone which provides strong protection for our immune checkpoint PD-1/PD-L1/PD-L2 inserted alphavirus VLP based on our platform technology. We strongly believe that this technology represents next generation cancer therapies that use the body’s own immune system to fight cancer with less side effects than current therapies." said Chief Executive Officer, Dr. Wataru Akahata. "Our candidate products have already been shown to be very effective in multiple cancer types in animals, which confirms my belief that this technology has the potential to treat a number of different cancers with high unmet medical needs. More people can benefit from our new cancer vaccine. This patent is another important element in our intellectual property strategy that we believe will allow us to fully exploit the fundamental value of the ongoing research and development. With this patent, as well as additional patents we are actively prosecuting, we are set to move forward with our R&D programs rapidly," he continued.
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
About i-α VLP Technology
The immune system is biologically designed to protect us against disease. The human immune system detects foreign objects such as viruses, bacteria or abnormal self-tissues (like cancer cells) in the body, and not only tries to eliminate these entities but also "memorizes" them so it can protect the body from these objects in the future. Vaccines utilize the immune system to protect us from various diseases.
Traditional vaccines are made using live viruses, which, though rare, can cause serious safety issues. Unlike traditional vaccines, VLP’s novel, proprietary platform technology was created by utilizing virus like particles. Virus like particles are identical to the authentic native viruses in their shapes, but do not carry the genetic material of native viruses. Without genetic material, these particles cannot replicate themselves. This means that when virus like particles are presented within our bodies, our immune system will recognize the particles as foreign objects, triggering effective immune responses, but will not cause side effects associated with the native virus due to the absence of genetic material.
Utilizing these virus like particles, VLP Therapeutics developed a proprietary, plug-and-play platform called inserted alphavirus virus-like particle (i-αVLP) using the Chikungunya virus (CHIKV) VLP. Through this adaptable platform, foreign antigens can be inserted into two specific sites of the envelope protein on the surface of i-αVLP. With 240 copies of envelope protein per CHIKV VLP, each i-αVLP can display a tremendous 480 copies of an inserted antigen. This highly symmetrical, icosahedral dense array of antigens has been shown to induce very strong immune responses, resulting in its superior efficacy. Additionally, unlike traditional vaccines, virus like particles themselves are non-replicative, as described above, because they do not carry any genetic material, and therefore are safe, having been used to make the FDA-approved vaccines for Hepatitis B virus and human papillomavirus. VLP Therapeutics has established a method to efficiently produce i-αVLPs which can be scaled for commercial production.
About VLP Therapeutics
VLP Therapeutics, LLC (VLP) was established in 2012 by seasoned biopharmaceutical veterans with the mission to develop innovative medical treatments which can transform traditional vaccines and targeted antibody therapies to address global unmet medical needs. Its vision is to combat 21st century global public health problems through its revolutionary next generation i-αVLP technology platform. VLP is currently developing preventative and therapeutic vaccines as well as next generation targeted antibody agents to treat cancer, infectious diseases, such as Malaria, Dengue Fever and Zika virus-based diseases, autoimmune and neurological diseases.