On May 30, 2018 Mustang Bio, Inc. ("Mustang") (NASDAQ:MBIO), a Fortress Biotech (NASDAQ:FBIO) Company focused on the development of novel immunotherapies based on proprietary chimeric antigen receptor engineered T cell (CAR T) technology, reported the publication of preclinical data demonstrating that glioblastoma (GBM)-targeted CD4+ CAR T cells mediate superior antitumor activity over CD8+ CAR T cells (Press release, Mustang Bio, MAY 30, 2018, View Source [SID1234526941]). The results were published in the May 17, 2018, edition of JCI Insight, a peer-reviewed journal of the American Society for Clinical Investigation. Mustang licensed the IL13Rα2‐specific CAR (MB-101) technology used in this preclinical study from the City of Hope National Medical Center ("City of Hope").

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "Optimizing T cell potency has the potential to enhance the antitumor efficacy of CAR T therapies in challenging solid tumors, which includes engineering the appropriate composition of CD4+ and CD8+ subsets. This important study conducted by our research partner City of Hope demonstrated the superior antitumor effect of CD4+ over CD8+ T cells in glioblastoma models. Optimizing T cell potency is one of many avenues Mustang is exploring to improve CAR T efficacy, and we look forward to the application of this research in the ongoing Phase 1 trial of our MB-101 IL13Rα2‐specific CAR T therapy in patients with glioblastoma."

Dr. Christine Brown, Heritage Provider Network Professor in Immunotherapy and Associate Director of the T Cell Therapeutics Research Laboratory at City of Hope, said, "This study provides important insight into the differences between CD4+ and CD8+ CAR T cells for maintaining killing potency and resisting exhaustion under conditions of high disease burden. These findings are part of our larger efforts to develop more powerful CAR therapies for the treatment of brain tumors."

In the study, City of Hope investigated the antitumor effect of CD4+ and CD8+ CAR T cells targeting the GBM-associated antigen IL-13 receptor α2 (IL13Rα2) in mouse models. Upon stimulation with IL13Rα2+ GBM cells, the CD8+ CAR T cells exhibited robust short-term effector function but became rapidly exhausted. In comparison, CD4+ CAR T cells persisted after tumor challenge and sustained effector potency.

Mixing with CD4+ CAR T cells failed to improve the effector dysfunction of CD8+ CAR T cells, and CD4+ CAR T cell effector potency was weakened when applied with CD8+ CAR T cells. In orthotopic GBM models, CD4+ outperformed CD8+ CAR T cells, specifically with respect to long-term antitumor response. Maintenance of the CD4+ subset was positively correlated with the recursive killing ability of CAR T cell products derived from GBM patients.

On May 30, 2018 Humanigen, Inc. (OTCQB:HGEN), a biopharmaceutical company developing cutting-edge T-cell optimization and oncology treatments, reported that Cameron Durrant, M.D., chairman and CEO, will present a company overview at the 8th Annual LD Micro Invitational on Monday, June 4, 2018, at 4 p.m. PDT (7 p.m. EDT) (Press release, Humanigen, MAY 30, 2018, View Source [SID1234526940]).

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Dr. Durrant will discuss Humanigen’s focus to enhance T-cell therapies in oncology with its proprietary Humaneered monoclonal antibodies, including lead asset lenzilumab as a potential prophylactic therapy to optimize treatment and minimize or prevent neurotoxicity associated with chimeric antigen receptor T-cell (CAR-T) therapy. Lenzilumab is a first-in-class recombinant monoclonal antibody that targets and is an antagonist of soluble granulocyte-macrophage colony-stimulating factor (GM-CSF), an upstream druggable target for inflammatory cascade side effects caused by CAR-T treatments.

A live webcast of the presentation will be available at View Source Following the conference, an archived version of the webcast will be available 120 days for replay on the Humanigen website at View Source

On May 30, 2018 Horizon Pharma plc (NASDAQ:HZNP), reported that the company will participate in the Goldman Sachs 39th Annual Global Healthcare Conference on Tuesday, June 12, 2018 and will present at 10:40 a.m. PT (Press release, Horizon Pharma, MAY 30, 2018, View Source [SID1234526939]).

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The presentation will be webcast live and may be accessed by visiting Horizon’s website at View Source A replay of the webcast will be available for the event.

On May 30, 2018 Kyowa Hakko Kirin Co., Ltd. (Tokyo: 4151 President and COO: Masashi Miyamoto, "Kyowa Hakko Kirin") reported that it has been notified that the U.S. Food and Drug Administration (FDA) is extending its review of the Biologics License Application (BLA) for mogamulizumab for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy (Press release, Kyowa Hakko Kirin, MAY 30, 2018, View Source [SID1234526937]). In a notice received from the FDA, the Prescription Drug User FeeAct (PDUFA) target action date has been extended to September 4, 2018.

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During the review, Kyowa Hakko Kirin submitted additional documentation related to the manufacturing section of the BLA, and FDA subsequently decided that it constituted a major amendment that requiring a 3 month extension to the original target action date, to provide time to complete review of the submission.

The Kyowa Hakko Kirin Group companies strive to contribute to the health and well-being of people around the world by creating new value through the pursuit of advances in life sciences and technologies.

About Mogamulizumab
Mogamulizumab is an investigational humanized monoclonal antibody (mAb) directed against CC chemokine receptor 4 (CCR4), which is frequently expressed on leukemic cells of certain hematologic malignancies including CTCL (cutaneous T-cell lymphoma). Mogamulizumab was produced using Kyowa Hakko Kirin’s proprietary POTELLIGENT platform, which is associated with enhanced antibody-dependent cellular cytotoxicity (ADCC).

In August 2017, the FDA announced that mogamulizumab has been granted Breakthrough Therapy designation status for the treatment of MF and SS in adult patients who have received at least one prior systemic therapy. In late November 2017, the FDA accepted the BLA for filing and granted mogamulizumab Priority Review.

About Mycosis Fungoides (MF) and Sézary Syndrome (SS)
MF and SS are the two most common subtypes of CTCL, a rare type of non-Hodgkin’s lymphoma, which is characterized by localization of malignant T lymphocytes to the skin, and depending on the stage, the disease may involve skin, blood, lymph nodes, and viscera.

On May 29, 2018 Laminar Pharma, a pioneering clinical stage biopharmaceutical company developing a new generation of products modulating metabolism of membrane lipids based on the groundbreaking MLT platform, reported that the FDA has approved an IND for a paediatric trial entitled "a phase I study of 2-hydroxyoleic acid (2OHOA) in pediatric patients with malignant glioma and other advanced solid tumors (Press release, Laminar Pharma, MAY 29, 2018, View Source [SID1234562094])"

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This will be the first clinical study with 2OHOA in children with advanced malignant cancer and will be conducted in collaboration with two leading US paediatric clinical research institutions: Hackensack University Medical Center, in New Jersey and Dana-Farber Cancer Institute in Boston. The main objectives of this study are to determine the safety and tolerability of 2OHOA in paediatric population (under 18 years), to characterize the pharmacokinetic profiles in this population and to assess the preliminary anti-tumour efficacy of the product. The trial follows a standard 3+3 design in the dose escalation phase, where 9 to 18 patients will be recruited in three cohorts, and that will be followed by an expansion cohort with 10 additional patients.

High-grade gliomas (HGG) are relatively rare forms of paediatric brain tumours, constituting only 8–12% of primary central nervous system (CNS) tumours in children. The management of these tumours involves surgical resection to the extent feasible, as well as adjuvant radiation and chemotherapy. Even with these interventions, the prognosis for patients with these tumours is poor, with most patients succumbing to their disease within 12–18 months. The incidence rate of primary malignant and non-malignant brain and CNS tumours in the US in paediatric and adolescent population (0-19 years) is 5.42 cases per 100,000 for a total count of around 23,000 incident tumours per year, of which over 2.500 cases correspond to HGG.

Laminar Pharma is committed to advance the clinical development of this promising product and is excited about the prospect of providing a potential therapeutic alternative for children and adult patients with brain and other aggressive cancers.

The approval by the FDA of the Investigational New Drug (IND) application for this paediatric trial in the US is a significant regulatory milestone for 2OHOA, (re)validating the extensive preclinical and clinical development that Laminar Pharma is carrying out with this innovative product.

A PIIb trial in adult patients with newly-diagnosed glioblastoma (CLINGLIO) with 2OHOA added to the current chemoradiation standard of care for this type of tumours is also planned to open within this year in leading Hospitals in Europe and Israel, following the award of an important H2020 grant by the European Commission to an international consortium lead by Laminar Pharma.