On May 21, 2018 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported that it has commenced a Phase 2 clinical trial to evaluate the efficacy of VGX-3100 in adult men and women with human papilloma virus (HPV)-related anal dysplasia (Press release, Inovio, MAY 21, 2018, View Source;The-No-1-Sexually-Transmitted-Disease/default.aspx [SID1234526823]). Recruitment is ongoing for patients who are HIV-negative with histologically confirmed anal or perianal high-grade squamous intraepithelial lesions (HSIL) associated with HPV-16 and/or HPV-18. The study is planning to enroll approximately 24 patients and will administer at least three doses of VGX-3100.

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Anal HSIL or dysplasia is the precursor to anal cancer, which is estimated to cause more than 1,100 deaths in the United States in 2018. Currently, the only treatments for anal dysplasia consist of surgical excision, electro-cautery or laser therapy, but more than 50% of those treated with these current treatments experience recurrence of the disease.

Dr. Céline Bouchard, FRCSC, gynecologist, colposcopist and Coordinating Principal Investigator, said, "The large burden of disease rests in the general population. As more than 70% of anal cancers in the United States occur among HIV-negative men and women, a non-surgical immunotherapy to eradicate precancerous perianal and/or anal lesions caused by HPV types 16 or 18 infection would represent a major breakthrough for the treatment of this disease and the prevention of anal cancer."

Dr. J. Joseph Kim, Inovio’s President and CEO, said, "Inovio is boldly targeting the world’s No. 1 sexually transmitted disease – the HPV virus – across the continuum of conditions with our treatment that has demonstrated efficacy eliminating disease and clearing the underlying HPV infection. This compact Phase 2 efficacy trial represents an important step towards Inovio’s aim to develop a comprehensive immunotherapeutic solution to HPV-related diseases, especially given that anal dysplasia remains an underdiagnosed condition which needs better treatment options. Inovio is rapidly moving on a path to become the ‘go-to’ immunotherapeutic solution provider for all diseases caused by HPV, including cervical, vulvar and anal precancers and with our partner MedImmune/AstraZeneca for HPV-related cancers."

This open-label, multi-center Phase 2 study is designed to evaluate the safety and efficacy of VGX-3100 administered by intramuscular (IM) injection w CELLECTRA delivery system in adult men and women who are human immunodeficiency virus (HIV) negative with HSIL associated with HPV-16 and/or HPV-18. Prior study results utilizing Inovio’s VGX-3100 immunotherapy, which is also in a Phase 3 trial evaluating treatment for cervical dysplasia, supported expanding treatment indications for patients associated with HPV-16 and/or HPV-18. In a Phase 2 trial for cervical dysplasia, VGX-3100 demonstrated a systemic response (vs. localized surgery) and clearance of cervical lesions and eliminating the underlying HPV infection in many patients. For additional information about the study, please visit www.clinicaltrials.gov (search identifier NCT03499795).

About VGX-3100

VGX-3100 is a DNA-based immunotherapy under Phase 3 investigation for the treatment of HPV-16 and HPV-18 infection and precancerous lesions of the cervix. Inovio is in open-label Phase 2 clinical trials evaluating its efficacy for treating HPV-related vulvar and anal precancers. VGX-3100 has the potential to be the first approved treatment for HPV infection of the cervix and the first non-surgical treatment for precancerous cervical lesions. VGX-3100 works by stimulating a specific immune response to HPV-16 and HPV-18, which targets the infection and causes destruction of precancerous cells. In a randomized, double-blind, placebo-controlled phase 2b study in 167 adult women with histologically documented HPV 16/18 cervical HSIL (CIN2/3), treatment with VGX-3100 resulted in a statistically significantly greater decrease in cervical HSIL and clearance of HPV infection vs. placebo. The most common side effect was injection site pain, and no serious adverse events were reported. VGX-3100 utilizes the patient’s own immune system to clear HPV-16 and HPV-18 infection and precancerous lesions without the increased risks associated with surgery, such as loss of reproductive health and negative psychosocial impacts.

About Inovio’s DNA Immunotherapy Technology Platform

Inovio is advancing the medical potential of a unique class of immunotherapy technology. Its DNA-based platform, which is the foundation for all of Inovio’s products, including VGX-3100, is unique in its ability to leverage the body’s naturally existing mechanisms to generate robust, highly targeted immune responses to prevent and treat disease – and to do so in the body without harmful side effects. Its SynCon immunotherapy design and CELLECTRA delivery transform novel genetic blueprints into functional antibody and killer T cell responses. Inovio was the first to report the activation – in the body – of significant, antigen-specific functional T cells correlated to statistically significant efficacy in a placebo-controlled, randomized, double-blind Phase 2b clinical trial (HPV-related precancer), with a very favorable safety profile. These data were published in The Lancet and independently described as a "major breakthrough" in the field by U.S. National Cancer Institute scientists. Inovio has achieved significant antigen-specific immune responses against multiple diseases and is advancing a growing pipeline of cancer and infectious disease immunotherapies and vaccines.

On May 21, 2018 PharmaCyte Biotech (OTCQB: PMCB) reported it has reached the proverbial "home stretch" regarding its efforts to submit an Investigational New Drug Application (IND) to the U.S. FDA (Press release, PharmaCyte Biotech, MAY 21, 2018, View Source [SID1234526825]). It’s an IND that has been eagerly anticipated by the company’s shareholders since PharmaCyte met with the FDA in early 2017, and it would lay out PharmaCyte’s planned Phase 2b clinical trial for the treatment of locally advanced, non-metastatic, inoperable pancreatic cancer or LAPC.

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The key to submitting the IND is and always has been satisfying regulatory requirements. In short, PharmaCyte must generate the necessary data that the FDA requires for any treatment—but especially a treatment that is deemed a "biologic" by the FDA and comprised of living cells that would be placed inside a human’s body.

PharmaCyte just announced that it has completed and passed 29 different tests from its Master Cell Bank (MCB), which is made up of cells that will be used in its planned clinical trial in patients with LAPC. The successful completion of these tests is the final hurdle before encapsulation and testing of the encapsulated cells can begin at Austrianova.

Vials of cells from the MCB have already been supplied to the company’s partner, Austrianova, for encapsulation. Austrianova will now be charged with completing the encapsulation process using PharmaCyte’s signature live-cell encapsulation technology, Cell-in-a-Box, to create the thousands of capsules that will be necessary to conduct its planned clinical trial. Each capsule will contain 10,000 live cells, and then each syringe will be filled with 300 capsules—making up the finished product that will be sent to clinical trial sites.

The encapsulation process will be followed by a battery of tests that will generate the necessary data to satisfy regulatory requirements and should complete what has been a long, meticulous process and allow PharmaCyte to finally submit it’s IND to the FDA.

In addition to the 29 successful tests that PharmaCyte announced last week, the company has stated that a number of additional tests have also been a success (See www.PharmaCyte.com/news). This is all very good news for shareholders who have been clamoring for the start of a clinical trial, and it should bode well for a company that is trying to win the FDA’s approval when it does submit its IND.

PharmaCyte’s goal of starting a Phase 2b clinical trial to meet an unmet medical need in the treatment of patients with LAPC is a remarkable milestone for such a small company, and because their first ever trip inside the clinic involves presenting the FDA with a biologic treatment, the sheer complexity of this journey explains why the process has been so time consuming. PharmaCyte’s treatment for LAPC utilizes genetically engineered live human cells that produce a particularly potent cytochrome P450 enzyme that is able to activate the chemotherapy prodrug ifosfamide.

As previously mentioned, these cells are encapsulated using the Cell-in-a-Box technology, and the tiny, pinhead-sized capsules are implanted near the cancerous tumor so that a high local concentration of the cancer-killing ifosfamide metabolite is produced near the tumor.

PharmaCyte’s treatment is not a single molecule drug. It’s not a drug at all actually. And because the treatment is made up of live human cells that are responsible for activating an already FDA-approved chemotherapy drug, the FDA expects every single cell to act exactly the same way, every single time, in every single test that PharmaCyte is required to conduct.

The FDA simply wants to know that the capsules and the cells that live inside them will remain exactly the same at all times when they are eventually placed inside the human body. And unfortunately there is no short cut when it comes to satisfying regulatory requirements.

The unmet medical need that PharmaCyte expects to address in its clinical trial is for those patients who no longer see any benefit from using the preferred standard of care, Abraxane combined with gemcitabine, or FOLFIRINOX, another combination chemotherapy that is increasingly being used in the U.S. as the preferred standard of care. These patients must have tumors that no longer respond to these combination chemotherapies after they’ve been on the treatment for a period of between 4 and 6 months.

The good news for PharmaCyte’s patient shareholders is that the company has reached the "end of the line" in what has been a very long process. But, when it comes to the FDA and success in a clinical trial—especially for a small company like PharmaCyte—there is only one true shot at getting it right.

Stock Market Media Group will be interviewing PharmaCyte’s CEO, Kenneth L. Waggoner, and its Chief Scientific Officer, Prof. Dr. Walter H. Günzburg, to discuss the IND, encapsulation, testing, preparations for the upcoming planned clinical trial, among other topics. The radio-style interview will be released and announced publicly via a press release within the next 2 weeks.

On May 21, 2018 GRAIL, Inc., a healthcare company whose mission is to detect cancer early, when it can be cured, reported it has raised USD$300 million in an oversubscribed Series C financing (Press release, Grail, MAY 21, 2018, View Source [SID1234526850]). The financing is led by Ally Bridge Group, co-led by Hillhouse Capital Group and 6 Dimensions Capital, and includes Blue Pool Capital, China Merchant Securities International, CRF Investment, HuangPu River Capital (HPR), ICBC International, Sequoia Capital China, and WuXi NextCODE.

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Since early 2016, GRAIL has raised more than $1.5 billion in three rounds of equity financing to pursue its vision of transforming the way cancer is diagnosed and reducing global cancer mortality. The Series C funding will add to the company’s balance sheet and support ongoing development and validation of products for the early detection of cancer in GRAIL’s clinical research program.

"GRAIL has continued to execute at a rapid pace towards its goal of saving lives through early detection of cancer. We have enrolled more than 73,000 participants in our population-scale clinical studies, CCGA and STRIVE, and are on-track to complete enrollment in both studies this year," said Jennifer Cook, Chief Executive Officer of GRAIL. "We recently reported data supporting the potential for development of a highly specific and sensitive blood test, and are now continuing our development programs to optimize and validate a product for early detection of multiple cancer types."

"We are fortunate to partner with new international investors who share our vision of delivering early detection products to people globally," said Ken Drazan, President of GRAIL. "Many of our new investors have a focus in Asia, which we believe is a natural fit as we plan to grow our capabilities and operations in the region, following the planned launch of our first product for early detection of nasopharyngeal cancer in Hong Kong this year."

"We are very impressed with the scientific, clinical, and software engineering achievements the team at GRAIL has made in just over two years. Our significant investment in GRAIL aligns well with Ally Bridge’s strong focus on investing in some of the world’s most innovative life science technologies and enhancing value-creation across geographies," said Frank Yu, Founder and Chief Executive Officer, Ally Bridge Group.

On May 21, 2018 Verastem, Inc. (NASDAQ: VSTM) (Verastem Oncology or the Company), focused on developing and commercializing drugs to improve the survival and quality of life of cancer patients, reported that Jonathan Pachter, PhD, the Company’s Chief Scientific Officer, will give an oral presentation and moderate a roundtable discussion at the 3rd Annual Advances in Immuno-Oncology Congress being held May 24-25, 2018 in London, UK (Press release, Verastem, MAY 21, 2018, View Source;p=RssLanding&cat=news&id=2350086 [SID1234526826]).

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"The data that will be presented at the Immuno-Oncology Congress demonstrate the unique potential of duvelisib, as a dual inhibitor of PI3K-delta and PI3K-gamma, to enhance the efficacy of immune checkpoint and co-stimulatory antibodies in preclinical models of both hematological malignancies and solid tumors," said Dr. Pachter. "These results support continued research and lend particular importance as we move toward the commercialization of duvelisib, Verastem’s lead candidate an oral, dual inhibitor of PI3K-delta and PI3K-gamma. The duvelisib New Drug Application (NDA) is currently under review by the U.S. Food and Drug Administration (FDA) for the treatment of patients with relapsed or refractory CLL/SLL, and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma. I will also give an update on the scientific rationale and clinical progress of our FAK inhibitor defactinib in combination with PD-1 and PD-L1 inhibitors in solid tumors."

Details for the presentation and round table discussion at the Congress are as follows:

Oral Presentation Title: Immunological Effects of Clinical Stage FAK & PI3K-Delta/Gamma Inhibitors
Session: Translational Immuno-Oncology
Date and time: Thursday, May 24, 2018 at 5:40 – 6:10 PM BST

Round Table Discussion Title: Novel Checkpoint Pathways & Strategies for Combined Modality Treatment
Date and time: Friday, May 25, 2018 at 7:30 – 8:00 AM BST

A copy of the oral presentation will be available following the presentation.

About Duvelisib
Duvelisib is a first-in-class investigational oral, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B- and T-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib was evaluated in late- and mid-stage extension trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL),4 and DYNAMO, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary endpoints. Verastem Oncology’s New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL, and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL) was accepted for filing by the U.S. Food and Drug Administration (FDA), granted Priority Review and assigned a target action date of October 5, 2018. Duvelisib is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

About Defactinib
Defactinib is an investigational inhibitor of focal adhesion kinase (FAK), a non-receptor tyrosine kinase that mediates oncogenic signaling in response to cellular adhesion and growth factors.7 Based on the multi-faceted roles of FAK, defactinib is used to treat cancer through modulation of the tumor microenvironment and enhancement of anti-tumor immunity.8,9 Defactinib is currently being evaluated in three separate clinical collaborations in combination with immunotherapeutic agents for the treatment of several different cancer types including pancreatic cancer, ovarian cancer, non-small cell lung cancer (NSCLC), and mesothelioma. These studies are combination clinical trials with pembrolizumab and avelumab from Merck & Co. and Pfizer/Merck KGaA, respectively.10,11,12 Information about these and additional clinical trials evaluating the safety and efficacy of defactinib can be found on www.clinicaltrials.gov.

On May 21, 2018 Immedica Group reported that it has entered into an exclusive supply and distribution agreement with German based Oncoscience GmbH regarding the distribution, marketing and sale of CIMAher (Nimotuzumab) in the Nordics (Press release, Immedica Pharma, MAY 21, 2018, View Source [SID1234555254]). Under the agreement, Medical Need AB gains the rights to distribute CIMAher in Denmark, Finland, Iceland, Norway and Sweden, and will initially do so on a named patient basis in the territories.

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Tomas Gloveus, the Head of Marketing and Sales at Medical Need AB said, "CIMAher addresses a significant unmet medical need for many patients with severe cancers, who are currently left to treatment alternatives with severe side-effects and we look forward to making CIMAher available in the Nordic countries."

Dr. Dirk Reuter, the Managing Director of Oncoscience GmbH added, "The niche focus combined with their capabilities within oncology, makes Medical Need a very suitable partner for us in the Nordics, and we look forward to working together to improve the access of CIMAher in the Nordic region."

Nimotuzumab is a well-established monoclonal antibody within oncology that targets the epidermal growth factor receptor (EGFR) on cancer cells and inhibits their signalling pathway which stops cancer cells from growing and proliferating. To date, Nimotuzumab has been administered to more than 65,000 patients globally and has undergone 82 clinical studies. Nimotuzumab is already approved in 25 countries around the world for a variety of cancers, such as head & neck cancer, oesophageal cancer, highly malignant brain tumours and pancreatic cancer.

Nimotuzumab has an EU orphan designation for the treatment of pancreatic cancer and glioma and has been used in EU under named patient basis, following special individual approvals from a national regulatory authority as it does not yet hold a EU marketing authorization.

About CIMAher (Nimotuzumab)
Nimotuzumab is a humanized monoclonal antibody that targets the epidermal growth factor receptor (EGFR). Nimotuzumab’s capacity to bind to EGFR is strongly dependant on cell receptor density and it’s bivalent binding mechanism. EGRF is overexpressed in many cancer cells as compared to normal tissue and nimotuzumabs bivalent binding mechanism results in more specific bonds and therefore, potentially increases anti-tumor activity with decreased toxicity in normal tissue (i.e. skin and healthy organ toxicity).

Nimotuzumab is currently available as a registered treatment in 25 countries around the world for a variety of cancers, including head & neck cancers in advanced stages, nasopharyngeal carcinoma, oesophageal cancer, high-malignant glial cancers (glioblastoma multiforme and anaplastic astrocytoma), and locally advanced or metastatic pancreatic cancer (adenocarcinoma). Nimotuzumab has also been approved in a number of countries for high-grade gliomas in children and adolescents (newly diagnosed, recurrent and refractory).