Trizell Ltd. announces Phase 3 pivotal study of interferon alfa-2b gene therapy in malignant pleural mesothelioma

On March 20, 2019 Trizell Ltd (Trizell) reported that it has opened a Phase 3 Study of its novel gene therapy TR002, an adenovirus-mediated interferon alfa 2b, in patients with malignant pleural mesothelioma (MPM) who have failed first-line standard of care chemotherapy (Press release, Trizell, MAR 20, 2019, View Source [SID1234561809]). TR002 is given to trigger the pleiotropic anti-tumour effects of interferon, a naturally occurring protein the body uses to fight cancer, and is followed by gemcitabine chemotherapy. This study will include sites in the United States, Europe, Australia, and Russia.

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Malignant pleural mesothelioma is an aggressive and devastating form of cancer affecting the outer (pleural) membrane of the lungs. The disease is usually triggered by inhalation of asbestos fibres that slowly make their way from the alveoli to the outer surface of the lung in the pleural cavity. The fibres then induce a cancer which grows on the outside of the lung into the pleural space, eventually invading surrounding thoracic tissues leading to death. There is a clear association between occupational or environmental asbestos and mineral fibre exposure and MPM development.
A previous Phase 2 study of TR002 at the Abramson Cancer Center included 40 MPM patients who were newly diagnosed or failed standard chemotherapy (Pemetrexed/ Platin) showed an overall disease control rate of 87.5%. The second line treatment cohort showed an almost doubling of median survival time compared to historical study controls (17 months vs. 9 months) with approximately 25% of patients living at least 2 years and approximately 20% at least 3 years.

MPM prognosis is poor and following failure of standard of care first line treatment, median life expectancy is around 9 months, with very few cases surviving past 18 months. With a prevalence of around 200,000 cases worldwide, there are around 3000 and 5000 new cases diagnosed every year in the EU and US alone. Dust from the September 11th attacks in New York is predicted to cause a rise in the number of future cases in the US, although the extent of the future rise is currently unclear.
"This is an exciting trial. The results that we noted in our previous study showed significant prolongation of life expectancy and particularly so for about 25 percent of these refractory patients who have gone on to live two and in some cases three years and more," said Daniel H. Sterman, MD, Director of the Multidisciplinary Pulmonary Oncology Program at NYU Langone Health. "We will work hard to get this potentially ground-breaking clinical trial completed."

The Phase 3 study is an open-label, randomized, parallel group study in up to approximately 300 patients who have failed chemotherapy. TR002 is given by catheter directly into the pleural cavity as a single dose of 3 x 1011 viral particles. Gemcitabine chemotherapy commences 14 days later and continues until disease progression.

"It is very exciting to see a therapeutic approach developed at the University of Pennsylvania being moved to an international, randomized trial, as it is a career milestone for any academic researcher to see their work tested in this way," said Steven M. Albelda, MD, William Maul Measey Professor of Medicine, Perelman School of Medicine, University of Pennsylvania.

TR002 was pioneered by Drs. Albelda and Sterman, and by Dr. Evan W. Alley, MD, PhD, whose current appointment is at Cleveland Clinic Florida but who was involved in the trial during his time at Penn.

About TR002
TR002 (nadofaragene firadenovec) is an investigational gene therapy consisting of an adenovirus containing the gene interferon alfa-2b. It is administered by catheter into the pleural cavity, where the virus enters the cells lining the pleural cavity. Inside the cells, the virus breaks down leaving the active gene to do its work. The internal gene/DNA machinery of the cells picks up the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach turns the patient’s own pleural cavity cells into multiple interferon microfactories, enhancing the body’s natural defences against the cancer.

About Malignant Pleural Mesothelioma
Malignant pleural mesothelioma is usually triggered by inhalation of asbestos fibres. These slowly make their way from the alveoli to the outer surface of the lung in the pleural cavity where they induce a cancer which grows on the outside of the lung into the pleural space, eventually invading surrounding thoracic tissues leading to death. Prognosis is poor and following failure of standard of care first-line treatment, median life expectancy is around 9 months, with very few cases surviving past 18 months.

WHO estimates that 43,000 people die of MPM each year; annually in North America, Western Europe, Japan, and Australia, there are 10,000 cases each year1. With the passage of the Clean Air Act, asbestos use was banned in the US in 19702, and the incidence of mesothelioma in the US was predicted to steadily decline. However, dust from the September 11th attacks in New York is predicted to cause a rise in the number of future cases although the extent of the future rise is currently unclear3. In contrast, asbestos use continued in Australia until 2003 and in Europe until 2005. As a result, the incidence of mesothelioma in European countries continues to rise and is projected to peak in 2020 and may account for as many as 250,000 European deaths in the next 35 years. In 2020, it is estimated over 18,000 Australian cases will be diagnosed4.

Diffusion Pharmaceuticals Reports 2018 Financial Results and Provides Business Update

On March 20, 2019 Diffusion Pharmaceuticals Inc. (Nasdaq: DFFN), a cutting-edge biotechnology company developing new treatments for life-threatening medical conditions by improving the body’s ability to bring oxygen to the areas where it is needed most, reported financial results for the year ended December 31, 2018 and provided a business update (Press release, Diffusion Pharmaceuticals, MAR 20, 2019, View Source [SID1234534500]).

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2018 was marked by significant developments related to the Company’s lead drug candidate, trans sodium crocetinate (TSC), for the treatment of stroke and cancer. During the third quarter Diffusion received approval from the U.S. Food and Drug Administration (FDA) to enroll patients in an ambulance-based Phase 2 clinical trial testing TSC for the treatment of acute stroke, while the in-ambulance trial design was presented at several important medical conferences. The trial, named PHAST-TSC (Pre-Hospital Administration of Stroke Therapy-TSC), will involve 23 hospitals across urban, suburban and rural areas in Los Angeles County and Central Virginia, working closely with approximately 150 emergency medical transport groups. PHAST-TSC will be led by researchers at the University of California Los Angeles (UCLA) and the University of Virginia (UVA). Diffusion is working to engage local ambulance companies and expects the first patients to be treated in the coming months. Results for the trial will potentially be available in just under two years, subject to Diffusion receiving the necessary funding.

The Company continues to screen and enroll patients in its Phase 3 INTACT (INvestigation of TSC Against Cancerous Tumors) program, using TSC to treat inoperable glioblastoma multiforme (GBM) brain cancer. In Phase 2 testing TSC demonstrated a nearly four-fold improvement in overall survival at two years for the subset of inoperable GBM patients compared with the control group of GBM patients.

Commenting on 2018 and plans for 2019, David Kalergis, chairman and chief executive officer of Diffusion, said, "In the coming weeks we expect to complete enrollment in the initial dose-escalation cohort of the INTACT trial. In addition, we expect to begin enrollment in PHAST-TSC during the second quarter of 2019. We continue to be excited about the potential for TSC to bring new hope to patients with life-threatening unmet medical needs and making TSC a commercial success.

Several patents were allowed and issued during the course of 2018, strengthening the Company’s intellectual property portfolio around broad uses of TSC in hypoxic conditions such as stroke, and as a treatment for solid cancerous tumors in conjunction with radiation and chemotherapy. Of note, a U.S. patent was issued for TSC in conjunction with tissue plasminogen activator (tPA) for the treatment of stroke. tPA is the only FDA-approved therapeutic for this indication. At the end of 2018, the company had 56 issued patents in the U.S. and abroad.

2018 Financial Results

Diffusion had cash and cash equivalents of $8.0 million as of December 31, 2018. The Company believes its cash and cash equivalents are sufficient to fund operations into July 2019.

Diffusion recognized $5.8 million in research and development expenses during 2018, compared with $5.1 million during 2017. This increase was primarily attributable to a $1.7 million increase in Phase 3 GBM trial expenses and to a $0.2 million increase in salary and wages expenses, partially offset by a $1.2 million decrease in manufacturing and other costs.

General and administrative expenses were $6.2 million during 2018, which were flat compared with 2017. Salaries and wages expense increased by $0.6 million, which was offset by a $0.6 million decrease in professional fees.

The Company recognized a non-cash goodwill impairment charge of $6.9 million during the year ended December 31, 2018 as a result of a sustained decrease in our market capitalization during the second half of 2018. There was no such charge in 2017.

Net cash used in operating activities for 2018 was $10.8 million, compared with $12.3 million during 2017

Enterome announces research collaboration with renowned US Cancer Institute focused on new microbiome-derived cancer immunotherapy

On March 20, 2019 ENTEROME SA, a clinical-stage biopharmaceutical company leveraging its unique knowledge of the key functional and molecular interactions between the gut microbiome and the human body to develop targeted therapeutics, reported that it has entered into a research and development collaboration with Dana-Farber Cancer Institute (Boston, MA) to evaluate and develop gut microbiome-derived antigens as potential cancer immunotherapies (Press release, Enterome, MAR 20, 2019, View Source [SID1234534522]).

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Enterome is developing an innovative approach to cancer immunotherapy, based on the concept of "molecular mimicry," whereby microbiome-derived bacterial antigens show molecular similarity with Tumor-associated Antigens (TAAs) and Tumor-specific Neoantigens (TSNAs). Based on this similarity, bacterial antigens ("onco-mimics") mimic key tumor antigens that are highly expressed by tumors to trigger tumor-specific cytotoxic T cell immune responses.

Enterome has developed a discovery platform to identify such onco-mimics from the human gut microbiome and has advanced EO2401 as its first clinical candidate. EO2401 comprises several microbiome-derived antigens that mimic antigens highly expressed by brain tumors and is targeting first clinical trials in 2019 as a potential new immunotherapy for recurrent glioblastoma multiforme (GBM) for which no curative treatments exist.

The collaboration brings together Enterome’s ability to identify potential TAAs and TSNAs as well as to generate bacterial onco-mimics for the selected TAAs and TSNAs with the complementary translational expertise from the research groups at Dana-Farber Cancer Institute led by Dr. David Reardon at the Center for Neuro-Oncology and Dr. Paul Kirschmeier at the Belfer Center for Applied Cancer Science. Drs. Reardon’s and Kirschner’s groups are focused on driving innovative research programs and clinical trials to improve cure rates in patients with brain and spinal cancers, with a particular focus on immunotherapies.

Through this collaboration Enterome will have access to knowledge, scientific expertise and preclinical models at Dana-Farber Cancer Institute that are intended to support the in vivo validation and further development of Enterome’s immunotherapy approach for the treatment of cancer.

"We are delighted to have signed this collaboration with one of the most esteemed cancer research institutions in the US," said Pierre Belichard, CEO of Enterome. "Drs. Reardon and Kirschmeier are accomplished and renowned clinician-scientists in their respective fields and their groups have pioneered novel approaches to cancer therapy, particularly in the brain. We believe that this collaboration will be highly productive in further validating our onco-mimic immunotherapy approach and enable us to develop our pipeline of new candidates for multiple cancer indications."

FDA confirms RhoVac’s application for a pre-IND meeting

On March 20, 2019 RhoVac AB (publ) ("RhoVac") reported that FDA (US Food and Drug Administration) has accepted RhoVac’s application for a pre-IND meeting, which is focused on the clinical development program with the drug candidate RV001 (Press release, RhoVac, MAR 20, 2019, View Source [SID1234534503]).

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RhoVac’s next step in the clinical development of the drug candidate RV001 is expected to start in mid-2019. Clinical development is going to be run primarily in Europe, but part of the company’s strategy is to run some of the clinical development in the US.

Therefore, RhoVac has submitted an application for a pre-IND meeting with the FDA to get answers and recommendations on specific issues related to the continued clinical development. A pre-IND meeting with the FDA is very similar to EMA’s Scientific Advice procedure, which was conducted by RhoVac in spring of 2018.

FDA has now confirmed RhoVac’s application following which the company has submitted the required Briefing Package. The evaluation of the FDA will be based on this package. RhoVac is expected to receive FDA’s comments on RV001 project by end of April 2019.

CEO Anders Ljungqvist comments
-It is very exciting that we now have the opportunity to receive FDA’s feedback and comments on RV001 project. The company’s drug candidate, RV001, is aimed at treating metastatic cancer in an earlier phase of prostate cancer development than existing treatments currently do. Therefore, it is important that both FDA and RhoVac have a common understanding of the concept of the treatment under development.

For more information, please contact:
Anders Ljungqvist – CEO, RhoVac AB
Phone: +45 4083 2365
E-mail: [email protected]

Xynomic Pharmaceuticals, Inc. Will Present at ASCO 2019 Annual Meeting, Will Sponsor an EU Investigator Meeting for Potentially Pivotal Kidney Cancer Trial, and Appoints Interim Chief Accounting Officer

On March 20, 2019 Xynomic Pharmaceuticals, Inc. ("Xynomic"), a clinical stage US-China oncology drug development company, and Bison Capital Acquisition Corporation (Nasdaq: BCAC), jointly reported that Xynomic and its collaborators will present at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") 2019 Annual Meeting to be held in Chicago from May 31, 2019 – June 4, 2019 (Press release, Xynomic Pharmaceuticals, MAR 20, 2019, View Source [SID1234534943]). The presentation will show Phase 1 data of XP-105 (also known as BI 860585), Xynomic’s Phase 2 ready, ATP-competitive, third generation mTORC1/2 Inhibitor, used alone or in combination with exemestane or paclitaxel in patients with advanced solid tumors. XP-105 is a potent dual inhibitor designed to overcome mTORC1 inhibition resistance. An oral presentation titled "A Dose Escalation Pharmacokinetic (PK) and Pharmacodynamic (PD) Study of mTORC1/2 Inhibitor XP-105 (BI 860585) as Monotherapy and in Combination with Exemestane or Paclitaxel in Patients (pts) with Advanced Solid Tumors" will be presented by Dr. Filippo G. De Braud, a lead investigator at Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy. This Phase 1 trial enrolled a total of 90 patients; 41 were in Arm A where XP-105 was used as a monotherapy (the "monotherapy arm"), 25 were in Arm B where XP-105 was used in combination with exemestane (the "exemestane combination arm"), and 24 were in Arm C where XP-105 was used in combination with paclitaxel (the "paclitaxel combination arm"). Combination regimens showed higher activity as compared to monotherapy. In the monotherapy arm, stable disease ("SD") was reported in 8 pts (20%), with a median duration of 11 months. In the exemestane combination arm, 4 (16%) partial responses ("PR") were reported. In the paclitaxel combination arm, 1 complete response ("CR") and 4 PRs were reported (OR rate 21%). Disease control rate (CR/PR/SD) was 20%, 28%, and 58% in the monotherapy, XP-105/exemestane, and XP-105/paclitaxel arms, respectively. In the XP-105/paclitaxel combination the most frequent drug-related adverse events ("AEs") were diarrhea and fatigue (58.3% each), hyperglycaemia (54.2%), and anaemia (50%). Grade ≥3 AEs were hyperglycaemia, fatigue, diarrhea, anaemia, and leukopenia.

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In addition, Xynomic will sponsor an investigator meeting on Friday, March 22, 2019 in Athens, Greece. The investigator meeting will bring together European Union ("EU") physicians, care providers and clinical research coordinators. The participants are from current and prospective clinical trial sites in the potentially pivotal Phase 3 clinical trial of testing Xynomic’s abexinostat, in combination with pazopanib, against renal cell carcinoma. The participants will be presented with trial design, protocol, and study management as a part of the preparation of the trial start and patient recruitment.

Furthermore, Xynomic’s board of directors has appointed Ms. Jinwei Coco Kou as the Interim Chief Accounting Officer. Ms. Kou is responsible for overseeing all accounting functions such as ledger accounts, financial statements, and cost control systems. Before joining Xynomic, Ms. Kou had extensive experience in internal controls, multinational operations and corporate finance of high-tech companies. From 2017-2018, Ms. Kou was the Chief Financial Officer at Salion Food Condiment Company Limited (a company approved for listing by the Hong Kong Stock Exchange). From 2008-2016, she was a Managing Director at Marcum Bernstein & Pinchuk LLP, one of the largest independent public accounting and advisory services firms in the United States, with multinational offices. From 2005-2008, Ms. Kou worked for Deloitte Touche Tohmatsu. Ms. Kou is a CPA in both the United States and China. Ms. Kou holds a Bachelor degree in Economics majoring in Finance, and a Master’s degree in Economics majoring in Risk Management and Insurance, both from Peking University. Ms. Kou holds an Executive MBA degree jointly granted by Columbia Business School, London Business School and Hong Kong University Business School.