1stOncology™: Cancer Intelligence Service – Page 12799 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Rigel to Present Poster Highlighting TAVALISSE at the Virtual Edition of the 25th European Hematology Association (EHA) Annual Congress

On June 11, 2020 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported that data related to TAVALISSE (fostamatinib disodium hexahydrate) tablets will be highlighted in a presentation at the Virtual Edition of the 25th EHA (Free EHA Whitepaper) Annual Congress taking place June 11-21, 2020 (Press release, Rigel, JUN 11, 2020, View Source [SID1234561019]). The presentation will be made available on the event’s website at www.ehaweb.org/congress/ on Friday, June 12 at 8:30 am CEST.

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!

In this previously presented post-hoc analysis, 32 patients received TAVALISSE as a second-line therapy, and 78% (25/32) achieved ≥1 platelet count of ≥50,000/µL (without rescue therapy). Adverse events were manageable and consistent with those previously reported with fostamatinib.

Fostamatinib disodium hexahydrate is an oral drug designed to inhibit spleen tyrosine kinase (SYK), a key signaling component of the body’s immune process that leads to platelet destruction in immune thrombocytopenia (ITP) and proposed red blood cell destruction in warm autoimmune hemolytic anemia (wAIHA). Fostamatinib is commercially available in the U.S. under the brand name TAVALISSE (fostamatinib disodium hexahydrate) tablets and is the first and only SYK inhibitor indicated for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to a previous treatment. TAVALISSE is currently being investigated in a Phase 3 trial for the treatment of wAIHA, a rare, serious blood disorder for which there are no approved therapies.

Poster Presentation
Abstract #EP1625
Second-line Therapy for Immune Thrombocytopenia with the Spleen Tyrosine Kinase Inhibitor Fostamatinib
Presenter: Dr. Nichola Cooper
Session Topic: 32. Platelets Disorders

About ITP
In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with this disease may live with an increased risk of severe bleeding events that can result in serious medical complications or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPO-RAs) and splenectomy. However, not all patients respond to existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder in which the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 45,000 adult patients in the U.S. and can be a severe, debilitating disease. To date, there are no disease-targeted therapies approved for AIHA, despite the unmet medical need that exists for these patients. Warm AIHA (wAIHA), the most common form of AIHA, is characterized by the presence of antibodies that react with the red blood cell surface at body temperature.

About TAVALISSE
Indication
TAVALISSE (fostamatinib disodium hexahydrate) tablets is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Important Safety Information
Warnings and Precautions

Hypertension can occur with TAVALISSE treatment. Patients with pre-existing hypertension may be more susceptible to the hypertensive effects. Monitor blood pressure every 2 weeks until stable, then monthly, and adjust or initiate antihypertensive therapy for blood pressure control maintenance during therapy. If increased blood pressure persists, TAVALISSE interruption, reduction, or discontinuation may be required.
Elevated liver function tests (LFTs), mainly ALT and AST, can occur with TAVALISSE. Monitor LFTs monthly during treatment. If ALT or AST increase to >3 x upper limit of normal, manage hepatotoxicity using TAVALISSE interruption, reduction, or discontinuation.
Diarrhea occurred in 31% of patients and severe diarrhea occurred in 1% of patients treated with TAVALISSE. Monitor patients for the development of diarrhea and manage using supportive care measures early after the onset of symptoms. If diarrhea becomes severe (≥Grade 3), interrupt, reduce dose or discontinue TAVALISSE.
Neutropenia occurred in 6% of patients treated with TAVALISSE; febrile neutropenia occurred in 1% of patients. Monitor the ANC monthly and for infection during treatment. Manage toxicity with TAVALISSE interruption, reduction, or discontinuation.
TAVALISSE can cause fetal harm when administered to pregnant women. Advise pregnant women the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose. Verify pregnancy status prior to initiating TAVALISSE. It is unknown if TAVALISSE or its metabolite is present in human milk. Because of the potential for serious adverse reactions in a breastfed child, advise a lactating woman not to breastfeed during TAVALISSE treatment and for at least 1 month after the last dose.
Drug Interactions

Concomitant use of TAVALISSE with strong CYP3A4 inhibitors increases exposure to the major active metabolite of TAVALISSE (R406), which may increase the risk of adverse reactions. Monitor for toxicities that may require a reduction in TAVALISSE dose.
It is not recommended to use TAVALISSE with strong CYP3A4 inducers, as concomitant use reduces exposure to R406.
Concomitant use of TAVALISSE may increase concentrations of some CYP3A4 substrate drugs and may require a dose reduction of the CYP3A4 substrate drug.
Concomitant use of TAVALISSE may increase concentrations of BCRP substrate drugs (eg, rosuvastatin) and P-Glycoprotein (P-gp) substrate drugs (eg, digoxin), which may require a dose reduction of the BCRP and P-gp substrate drug.
Adverse Reactions

Serious adverse drug reactions in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis, which occurred in 1% of TAVALISSE patients. In addition, severe adverse reactions occurred including dyspnea and hypertension (both 2%), neutropenia, arthralgia, chest pain, diarrhea, dizziness, nephrolithiasis, pain in extremity, toothache, syncope, and hypoxia (all 1%).
Common adverse reactions (≥5% and more common than placebo) from FIT-1 and FIT-2 included: diarrhea, hypertension, nausea, dizziness, ALT and AST increased, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.

CNS Announces the FDA has Granted Orphan Drug Designation for Brain Cancer Drug Berubicin

On June 11, 2020 CNS Pharmaceuticals, Inc., (Nasdaq: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for its lead product Berubicin for the treatment of malignant gliomas (Press release, CNS Pharmaceuticals, JUN 11, 2020, View Source [SID1234561018]).

"We are pleased to receive Orphan Drug Designation for Berubicin, our lead candidate. The designation provides Berubicin with a special status that can accelerate its development to treat malignant gliomas, and provides CNS with the potential for market exclusivity upon the drug’s approval," stated John Climaco, CEO of CNS Pharmaceuticals. "In the Phase 1 trial of Berubicin to treat glioblastoma, one of the world’s most aggressive cancers, under a prior developer, 44% of the patients demonstrated a significant improvement in progression free survival, and one patient experienced a complete response. We look forward to continuing to execute on our strategic plan and initiating a Phase II trial evaluating the effect of Berubicin on patients with glioblastoma later this year."

Chief Medical Officer of CNS, Dr. Sandra Silberman, stated, "We are excited to continue to drive the development of Berubicin and work towards addressing a critical unmet medical need. Glioblastoma currently has a dismal survival rate of only 14.6 months from its diagnosis. We believe Berubicin, which based on limited clinical data appears to be the first anthracycline to cross over the blood brain barrier in adults, provides a potentially novel therapy for the treatment of malignant gliomas."

The FDA grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States at any given time. Due to small patient numbers, treatment for these rare diseases would not be considered economically feasible without government programs to support their economic viability. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

About Berubicin

Berubicin is an anthracycline, a class of anticancer agents that are among the most powerful chemotherapy drugs and effective against more types of cancer than any other class of chemotherapeutic agents. Anthracyclines are designed to utilize natural processes to induce deoxyribonucleic acid (DNA) damage in targeted cancer cells by interfering with the action of topoisomerase II, a critical enzyme enabling cell proliferation. Berubicin treatment of brain cancer patients appeared to demonstrate positive responses that include one durable complete response in a Phase 1 human clinical trial conducted by Reata.

AVM Biotechnology Awarded SBIR Phase I Grant from National Cancer Institute

On June 11, 2020 AVM Biotechnology reported that it has been awarded a National Cancer Institute Small Business Innovation Research (SBIR) Grant from the National Institutes of Health (NIH) to study the use of their lead molecule AVM0703 as a preconditioning agent to allow safe and efficient delivery of therapeutic immune cells for cancer treatment (Press release, AVM Biotechnology, JUN 11, 2020, View Source [SID1234561017]). This novel solution could offer clinical advantages to any cell-based immunotherapy, improving access to potentially life-saving therapies by all cancer patients, including those too frail to receive chemotherapy.

Cellular immunotherapy has the potential to become a crucial solution for cancer. However, toxic chemotherapy is currently required as preconditioning treatment to impair graft rejection and maintain therapeutic cells in the bloodstream where they can target cancer cells. Chemotherapy is hardly tolerated by frail cancer patients, and it fuels the side effects of immunotherapy such as toxic cytokine releases (CRS) and neuroedemas. AVM0703, which could be easily administered to increase efficient delivery of adoptive cellular immunotherapy, induces safe lymphodepletion in only 24 hours, sparing platelets, stem cells and red blood cells in animal models. AVM0703 can safely deplete monocytes, known to be a key inducer of CRS. CRS toxicities occur as frequently as 90% with half of them determined as severe. Severe CRS complications can be life threatening if not treated in a timely manner. Unlike chemotherapy, AVM0703 could safely deplete monocytes reducing the risk of CRS and making cellular immunotherapy accessible to high-risk individuals.

This Phase I grant will be used to validate the efficacy and safety of preconditioning by AVM0703 in an established tumor mouse models of multiple myeloma (MM). The proposal was regarded as very significant in addressing a clinical need of better ways for improving efficacy and reducing toxicity of cellular therapies. Moreover, preconditioning using AVM0703 was seen as well supported by a good rationale and strong preliminary data. AVM0703 mode of action could offer an exemplary preconditioning regimen.

Named one of the top 10 best Biotech and Pharma companies to keep your eye on in 2019 by Mirror Review Online Magazine, AVM Biotechnology was founded in 2008 by Dr. Theresa Deisher, Ph.D. With over 30 years of successful pharmaceutical research experience and holding over 47 patents, Dr. Deisher leads a team of scientists dedicated to changing what a diagnosis of cancer, autoimmunity, or chronic infectious disease means to patients and their loved ones. AVM received FDA IND approval in April 2020 to test AVM0703 for treatment of relapsed/refractory lymphoid malignancies. AVM’s passion is to deliver drugs that work rapidly and that are safe, effective, and affordable, to treat serious worldwide illness like cancer, autoimmunity, and life-altering infectious disease. They develop products that improve outcomes without additional suffering because they believe that side effects from treatments of cancers or infections should never be worse than the diseases themselves.

AVM is the only company to receive homologous use designation for a patient’s own bone marrow used for an indication outside of blood disorders. Additionally, in 2019 AVM Biotechnology was awarded an SBIR grant for targeted lympho-ablation as an alternative to cure Type I Diabetes by the National Institute of Diabetes and Digestive and Kidney Disease (NIDDK).

This award was granted by the National Institutes of Health under Award Number R43CA246896. The content of this press release is solely the responsibility of the author and does not necessarily represent the official views of the NIH.

Accellix, the Cell Therapy QC Company Raises $9.5 Million Series D Round

On June 11, 2020 Accellix, the Cell Therapy QC company, reported a Series D financing round of $9.5 million led by bioMérieux, a world leader in the field of in vitro diagnostics for over 55 years (Press release, Accellix, JUN 11, 2020, View Source [SID1234561016]). All existing major investors participated in this raise and have also agreed to convert their pre-existing convertible note in the amount of $8.5 million, bringing the company’s total funding in this round to $18 million. This latest round of funding will be used to further expand the market reach of the Accellix system for Cell Therapy QC, enabling its customers greater access to the expertise, scale, resources, and technology needed to effectively assess the quality of life saving immunotherapies.

"Companies developing cellular therapies in the area of immuno-oncology and other therapeutic areas understand the need to replace costly and complicated flow cytometry based procedures with an easy to use sample-to-answer system. Being recognized by bioMérieux for our achievements to-date and having access to their vast array of resources and expertise, will help us serve our existing customers while attracting new ones," said Nir Nimrodi, Accellix Chairman and CEO.

"The Accellix platform eliminates extensive training and complex operational procedures by automating inherent manual complex flow cytometry processes. It provides accurate and immediate flow cytometry results at the point of need, removing the requirement of sending samples out to a central lab. We have been searching for such a platform that will complement our Industry-leading Microbial Quality Control offering and it is our intention to work with Accellix on enabling them to advance their market reach and service and support capabilities," stated Michael Reynier, bioMérieux Vice President, Healthcare Industries Business Area. "Testing a precious cell therapy product for sterility and for quality, swiftly, accurately and reproducibly, will improve patients’ access to the lifesaving cellular therapies that are currently being developed."

City of Hope signs licensing agreement with Scopus BioPharma Inc. to develop novel, targeted immuno-oncology gene therapy

On June 11, 2020 City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, reported that it has signed an exclusive, worldwide licensing agreement with Scopus BioPharma Inc., a biopharmaceutical company focused on novel therapeutics for serious diseases with significant unmet medical needs (Press release, City of Hope, JUN 11, 2020, View Source [SID1234561015]). Scopus will further develop and plans to commercialize a City of Hope first-in-class, targeted immuno-oncology gene therapy.

A first in-human phase 1 clinical trial for B cell lymphoma patients that uses the licensed gene therapy drug, CpG-STAT3siRNA, a STAT3 inhibitor, is expected to commence at City of Hope in the second half of this year.

Growing evidence links B cell non-Hodgkin lymphomas to persistent activation of STAT3, a gene that drives tumor cell growth and anti-tumor immune suppression. The STAT3 inhibitor is a highly selective and targeted therapy that silences the activity of the STAT3 gene by way of RNA interference. It also stimulates the TLR9 receptors to activate the body’s immune defense to recognize and kill cancer cells.

In preclinical testing at City of Hope, the STAT3 inhibitor has successfully reduced growth and metastasis of various preclinical tumor models, including melanoma, and colon and bladder cancers, as well as leukemia and lymphoma.

City of Hope’s Hua Yu, Ph.D., Billy and Audrey L. Wilder Professor in Tumor Immunotherapy, associate chair/professor in the Department of Immuno-Oncology, and co-leader of the Cancer Immunotherapeutics Program, and Marcin Kortylewski, Ph.D., associate professor in the Department of Immuno-Oncology, who are both leading experts in the role of STAT3 in tumor angiogenesis and tumor immune evasion and in oligonucleotide-based cancer immunotherapies, developed the STAT3 inhibitor. The strategy was developed based on seminal discoveries by Yu’s team defining the key role of STAT3 in cancer cell survival and immune tolerance, combined with pioneering work by Kortylewski’s team on STAT3 targeting using TLR9-targeted delivery of siRNA oligonucleotide therapeutics into immune cells.

"City of Hope is proud to work with Scopus to bring this long-anticipated STAT3 drug to our clinical trial patients," Yu said. "STAT3 is critical for the survival and metastasis of cancer cells, and for suppressing anti-tumor immune responses. If City of Hope and Scopus can develop the first therapy against STAT3, many cancer patients will benefit from this lifesaving drug."

"Our laboratories were the first to demonstrate that successful cancer immunotherapy needs to be two-step since TLR9 immunostimulation is only effective when STAT3 in the tumor microenvironment is no longer active," Kortylewski added. "It is exciting to see this technology approaching clinical application with a strong ally in biopharma."

Both an academic medical center and a drug development powerhouse, City of Hope is known for creating the technology used in the development of human synthetic insulin and numerous breakthrough cancer drugs. Its unique research and development hybrid of the academic and commercial creates an infrastructure that enables City of Hope researchers to submit an average of 50 investigational new drug applications to the U.S. Food and Drug Administration each year. The institution currently holds more than 450 patent families.

"Scopus is extremely excited about licensing such a promising gene therapy drug with a robust intellectual property portfolio, compelling preclinical efficacy and safety profile, and a plan to enter phase 1 this year," said Joshua R. Lamstein, Scopus co-chairman. "We are privileged to have the opportunity to work with City of Hope, a globally recognized research institution and cancer center. We are looking forward to working with Drs. Yu and Kortylewski, who are among the preeminent researchers in immuno-oncology."