Medicenna to Present at Raymond James Human Healthcare Innovation Conference

On June 11, 2020 Medicenna Therapeutics Corp. ("Medicenna" or the "Company") (TSX: MDNA) (OTCQB: MDNAF), a clinical stage immuno-oncology company, reported that Dr. Fahar Merchant, President, CEO and Chairman of the Board of Medicenna, will present a corporate overview at the Raymond James 2020 Human Healthcare Innovation Conference on Thursday, June 18th, 2020, at 1:40 PM ET (Press release, Medicenna Therapeutics, JUN 11, 2020, View Source [SID1234561021]).

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A live webcast of the presentation may be accessed at View Source A replay of the presentation will be available after the event by visiting the Investor Relations section of Medicenna’s website at View Source

Kymera Therapeutics to Disclose IRAKIMiD Degrader Program and Present Preclinical Data Demonstrating Potent Immunomodulatory and Antitumor Activity for its Novel STAT3 Degraders in Immuno-oncology

On June 11, 2020 Kymera Therapeutics, Inc., a biotechnology company pioneering targeted protein degradation to invent breakthrough protein degrader medicines for patients, reported that it will present preclinical data on its potent and highly selective STAT3 degraders as well as the first data from its novel IRAKIMiD degraders combining IRAK4 and IMiD substrate degradation (Press release, Kymera Therapeutics, JUN 11, 2020, View Source [SID1234561020]). Data will be shared in two separate presentations (Abstracts #10165 and #4349, respectively) during the 2020 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II on Monday, June 22 at 9:00 AM EDT.

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STAT3 is an attractive but elusive target known to regulate genes implicated in oncogenesis, tumor immune evasion, inflammation and fibrosis. In cancer, STAT3 has been shown to drive tumor growth and promote an immunosuppressive tumor microenvironment (TME). Kymera has previously reported the ability of its highly selective STAT3 degraders to achieve tumor regression in mouse xenograft models of STAT3-dependent hematologic malignancies. The company will present preclinical data demonstrating that its STAT3 degraders downregulated immune checkpoint signals on tumor cells and positively modulated composition and activity of immune cells in the TME, leading to in vivo antitumor activity in a solid tumor model refractory compared to standard anti-PD-1/L1 immunotherapy. These findings demonstrate the potential for STAT3 degraders to drive antitumor responses through both tumor cell-intrinsic and -extrinsic immunomodulation as well as direct antitumor effects.

Kymera will also present the first data from its potent IRAKIMiD degraders in development for the treatment of MYD88-mutant lymphomas, which constitute approximately one quarter of all diffuse large B-cell lymphomas (DLBCL). IRAKIMiDs are novel heterobifunctional degraders that target degradation of both IRAK4 and IMiD substrates with a single small molecule. While IRAK4 degradation alone offers a viable therapeutic approach that we believe is superior to IRAK4 kinase inhibition, targeting two complementary pathways in lymphoma biology addresses potential tumor escape mechanisms and shows synergistic activity in MYD88-mutant lymphomas compared to IRAK4 degraders or IMiDs alone. In fact, IRAKIMiDs demonstrated improved cell death and breadth of activity relative to IMiDs or IRAK4-selective degraders, and drove strong in vivo tumor regressions in multiple models of MYD88-mutant B cell lymphoma that Kymera believes are superior to what has been observed in preclinical studies with other agents such as BTK inhibitors and IMiDs.

"Our STAT3 and IRAKIMiD programs exemplify the tremendous potential of targeted protein degradation in oncology, allowing us to inhibit well-validated, high impact disease pathways through targets like STAT3 previously considered undruggable, and through selective multi-targeting of IRAK4 and IMiD substrates with a single degrader," said Jared Gollob, MD, Chief Medical Officer. "The dual effects of STAT3 degraders on both tumor cells and the tumor microenvironment, as well as the dual effects of IRAKIMiDs on multiple pathways involved in tumor cell growth and survival in MYD88-mutant lymphomas, drive robust antitumor responses in preclinical models that support progression of both programs into the clinic in 2021."

AACR Study Highlights

ABSTRACT #10165 / POSTER #LB-088, "A STAT3 selective targeted protein degrader decreases the immune-suppressive tumor microenvironment and drives antitumor activity in preclinical models," presented by Fred Csibi, PhD, Associate Director, Oncology Biology at Kymera Therapeutics.

KTX-201 (formerly known as KYM-003) is a potent and highly selective STAT3 degrader with activity in immune and tumor cells.
Degradation of STAT3 with KTX-201 in both immune and tumor cells reversed expression of genes that contribute to immune suppression.
KTX-201 treatment resulted in reversal of immunosuppression in an in vitro non-small cell lung cancer model as well as antitumor activity in a mouse colorectal cancer model resistant to immune checkpoint inhibitors.
ABSTRACT #4349 / POSTER #5222, "Degraders targeting both IRAK4 and IMiD substrates show combinatorial effects leading to broader activity with durable and complete regressions in MYD88 mutant lymphoma xenografts in vivo," presented by Duncan H. Walker, PhD, VP of Oncology at Kymera Therapeutics.

IRAK4 degradation, but not IRAK4 kinase inhibition, showed additive and synergistic activity with IMiDs in vitro; IRAKIMiDs, which combine these activities in a single molecule, showed potent in vitro and in vivo activity.
Degradation of both IRAK4 and IMiD substrates correlated with in vitro cell growth inhibition, consistent with a requirement for both activities for cell death.
Regressions observed in xenograft models of MYD88-mutant lymphoma were associated with degradation of both IRAK4 and IMiD substrates, consistent with the dual-targeting activities of these molecules.

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.

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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]).

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"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.

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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.