Data from Galera Therapeutics’ 223-Patient Phase 2b Clinical Trial of GC4419 Presented at MASCC/ISOO 2018 Annual Meeting

On June 29, 2018 Galera Therapeutics, Inc., a clinical-stage biotechnology company focused on the development of drugs targeting oxygen metabolic pathways with the potential to transform cancer radiotherapy, reported data from the Phase 2b clinical trial evaluating GC4419, a highly selective and potent small molecule dismutase mimetic, were presented today during an oral session at the Multinational Association of Supportive Care in Cancer (MASCC) and the International Society of Oral Oncology (ISOO) 2018 Annual Meeting in Vienna, Austria (Press release, Galera Therapeutics, JUN 29, 2018, View Source [SID1234527597]).

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Trial investigator Carryn Anderson, M.D., Radiation Oncologist, University of Iowa Hospitals and Clinics, was awarded the MASCC Steven M. Grunberg Memorial Award, which recognizes the author of the highest-ranking abstract for excellent scientific achievement in supportive care in cancer. Dr. Anderson also delivered the Annual Steven M. Grunberg Memorial Lecture.

"I’m honored that my peers have recognized the clinically meaningful GC4419 data with this prestigious award," said Dr. Anderson. "GC4419 demonstrated the ability to significantly decrease the duration and incidence of severe oral mucositis, highlighting its potential to be an important new adjunct medication for head and neck cancer patients undergoing radiation therapy. There are currently no approved therapies to prevent or mitigate this common and painful side effect of cancer treatment."

"We’re delighted that Dr. Anderson has been commended with the Steven M. Grunberg Memorial Award for the GC4419 data," said Mel Sorensen, M.D., President and CEO of Galera. "Earlier this month, our Phase 2b clinical trial data were also selected to be part of the Best of ASCO (Free ASCO Whitepaper) program. These distinctions from academia speak to the significance of the data and provide further validation of GC4419’s promise to address a serious unmet need. We look forward to advancing GC4419 into the next phase of development."

About the GC4419 Phase 2b Data

The 223-patient, double blind, randomized, placebo-controlled trial evaluated the safety of GC4419 and its ability to reduce the duration of radiation-induced severe oral mucositis (SOM) in patients with locally advanced squamous cell head and neck cancer receiving seven weeks of radiation therapy plus cisplatin. Approximately 70 percent of patients receiving chemoradiotherapy develop SOM, as defined by the World Health Organization as Grade 3 or 4, which is the most debilitating side effect of the radiotherapy.

Patients in the trial were treated with either 30 mg or 90 mg of GC4419 or placebo by infusion on the days they received their radiation treatment. Patients were randomized to one of the three treatment groups (1:1:1) and the trial recruited patients in both the United States and Canada. GC4419 exhibited a safety profile comparable to placebo in the two treatment groups, and was well tolerated. In the trial’s intent-to-treat population, the 90 mg dose of GC4419 met the primary endpoint, demonstrating a statistically significant (p = 0.024) 92 percent reduction in the median duration of SOM from 19 days to 1.5 days.

In the 90 mg arm, GC4419 also demonstrated a clinically meaningful effect in pre-specified secondary endpoints (incidence and severity of SOM). GC4419 achieved a 34 percent reduction through completion of radiation (p = 0.009), and a 36 percent reduction through 60 Gy of radiation (p = 0.010), in the overall incidence of SOM, and reduced the severity of patients’ OM by 47 percent (p = 0.045).

About Oral Mucositis

Oral mucositis (OM) is a painful and problematic complication during cancer treatment, especially radiation therapy, caused by excessive superoxide generated during treatment that breaks down epithelial cells that line the mouth. Patients suffering from OM experience severe pain, inflammation, ulceration and bleeding of the mouth.

In the United States, more than 50 percent of patients with cancer receive radiotherapy at some time in their treatment. In patients with head and neck cancer, radiotherapy is a mainstay of treatment and approximately 70 percent of patients receiving chemoradiotherapy develop severe oral mucositis (SOM) as defined by the World Health Organization as Grade 3 or 4, which is the most debilitating side effect of the radiotherapy.

SOM can adversely affect cancer treatment outcomes by causing interruptions in radiotherapy, which may compromise the otherwise good prognosis for tumor control in many of these patients. SOM may also inhibit patients’ ability to eat solid food or even drink liquids, and can cause serious infections. Further, the costs of managing these side effects are substantial, particularly when hospitalization and/or surgical placement of PEG tubes to maintain nutrition and hydration are required. There is currently no drug approved to prevent or treat SOM in patients with head and neck cancer.

About GC4419

GC4419 is a highly selective and potent small molecule dismutase mimetic that closely mimics the activity of human superoxide dismutase enzymes. GC4419 works to reduce elevated levels of superoxide caused by radiation therapy by rapidly converting superoxide to hydrogen peroxide and oxygen. Left untreated, elevated superoxide can damage noncancerous tissues and lead to debilitating side effects, including oral mucositis (OM), which can limit the anti-tumor efficacy of radiation therapy. Conversion of elevated superoxide to hydrogen peroxide, which is selectively more toxic to cancer cells, can also enhance the effect of radiation on tumors, particularly with stereotactic body radiation therapy (SBRT), which produces high levels of superoxide.

GC4419 has been studied in patients with head and neck cancer, GC4419’s lead indication, for its ability to reduce the incidence and duration of radiation-induced severe oral mucositis (SOM). Results from Galera’s 223-patient, double blind, randomized, placebo-controlled Phase 2b clinical trial demonstrated GC4419’s ability to dramatically reduce the duration of SOM from 19 days to 1.5 days (92 percent), the incidence of SOM through completion of radiation by 34 percent and the severity of patients’ OM by 47 percent, while demonstrating acceptable safety when added to a standard radiotherapy regimen. In addition, in multiple preclinical studies, GC4419 demonstrated an increased tumor response to radiation therapy while preventing toxicity in normal tissue.

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to GC4419 for the reduction of the duration, incidence and severity of SOM induced by radiation therapy with or without systemic therapy. The FDA also granted Fast Track designation to GC4419 for the reduction of the severity and incidence of radiation and chemotherapy-induced OM.

Combination Therapy BRAFTOVI (encorafenib) and MEKTOVI (binimetinib) for the Treatment of BRAF-mutant Melanoma, Available for Order at Biologics, Inc.

On June 29, 2018 Biologics, Inc., a McKesson Specialty Health oncology, neurology and complex care pharmacy services company, has been selected by Array BioPharma, Inc. to be in the limited distribution network for the combination therapy BRAFTOVITM (encorafenib) and MEKTOVI (binimetinib) for the treatment of patients with unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation, as detected by a U.S. Food and Drug Administration (FDA) approved test (Press release, Biologics, JUN 29, 2018, View Source [SID1234527519]).

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"Advanced BRAF-mutant melanoma is a serious and deadly type of skin cancer. Nearly half of melanoma patients who are diagnosed with the cancer after it has already spread have the BRAF mutation," said Brandon Tom, VP, Commercial Services, McKesson Specialty Health. "BRAFTOVI and MEKTOVI represent an important new targeted treatment, and we are looking forward to helping to bring this new option to the metastatic melanoma community."

BRAFTOVI and MEKTOVI were approved by the FDA on June 27, 2018 based on clinical data from the Phase 3 COLUMBUS trial, published in the Lancet Oncology.1 More information about these products can be found at www.braftovimektovi.com.

Metastatic melanoma is the most life-threatening type of skin cancer and is associated with low survival rates. 2,3 Approximately half of the estimated 200,000 new cases of melanoma diagnosed worldwide each year have BRAF mutations, a key target in the treatment of metastatic melanoma. 2,4,5

Biologics’ oncology specialty pharmacy is committed to providing patient-centric care throughout the patient’s cancer journey. The company is recognized across the industry for its high level of customer service and its innovative high-touch patient care model that supports patients with a multidisciplinary care team. Each team includes a pharmacist with in-depth knowledge of oncology pharmaceuticals, an experienced oncology nurse and a financial counselor who is familiar with various financial assistance programs and organizations that help cancer patients. This highly skilled care team works together to develop individualized care plans that address each patient’s unique clinical, financial and emotional needs and that streamline communication back to the treating provider, empowering high-quality care and optimal outcomes.

Physicians may submit prescriptions to Biologics via phone (800-850-4306), fax (800-823-4506) or eScribe. For electronic prescribing systems, physicians may search for Biologics within their EMR system.

FDA Lifts Clinical Hold so MYC Inhibitor APTO-253 Can Return to Phase 1b Trial In Patients With Hematologic Cancers

On June 29, 2018 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS) reported that the U.S. Food and Drug Administration (FDA) has notified the company that it has lifted the clinical hold on APTO-253, Aptose’s investigational drug for hematologic cancers (Press release, Aptose Biosciences, JUN 29, 2018, View Source;p=RssLanding&cat=news&id=2356603 [SID1234527515]). APTO-253 is the only known clinical-stage molecule that has the potential to directly inhibit expression of the MYC oncogene, shown to be a causative factor in many malignancies, including acute myeloid leukemia (AML).

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Up to fifteen clinical centers are expected to participate in the Phase 1b trial, and the screening and dosing will resume as soon as practicable for patients with relapsed or refractory AML or with high risk myelodysplastic syndromes (MDS). Recent data also highlight the role of MYC gene dysregulation in B-cell malignancies1, and Aptose hopes to pursue this patient population in the coming months.

The Phase 1b trial of APTO-253 had been placed on clinical hold as a consequence of an event that occurred at a clinical site with the infusion procedure. Ultimately, a root cause investigation determined that the event resulted from chemistry and manufacturing based issues, all of which were incorporated into a Chemistry, Manufacturing and Control (CMC) amendment to the Investigational New Drug (IND) application.

"We are eager to return APTO-253 back into the clinic," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "Our understanding of this molecule has evolved dramatically, and we are excited to deliver a MYC gene expression inhibitor to patients with debilitating hematologic malignancies."

About the Study

The Phase 1b, multicenter, open-label, dose-escalation clinical trial of APTO-253 is designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamic responses and efficacy of APTO-253 as a single agent and determine the recommended Phase 2 dose. APTO-253 will be administered once weekly, over a 28-day cycle. The dose escalation cohort of the study could potentially enroll up to 20 patients with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS). The study is designed to then transition, as appropriate, to single-agent expansion cohorts in AML and MDS.

MYC dysregulation is a common driver in many malignancies, making it an attractive therapeutic target. Repression of MYC expression by bromodomain (BET) inhibitors has proven effective at triggering apoptosis in leukemia cells; however, inhibition of bromodomain proteins can cause severe toxicities and myelosuppression due to the presence of bromodomain proteins on all active genes. Unlike BET inhibitors and other cancer chemotherapies, APTO-253 acts through a distinct targeted mechanism. As a first in class, small molecule MYC inhibitor, APTO-253 may be particularly appropriate for the management of patients having AML and other hematologic malignancies with compromised bone marrow function. Earlier this month, Aptose announced the publication of preclinical data further elucidating the mechanism of action of APTO-253 (here).

About APTO-253

APTO-253 is a clinical-stage small molecule targeted therapeutic agent that inhibits expression of the MYC oncogene, leading to cell cycle arrest and programmed cell death (apoptosis) in human-derived solid tumor and hematologic cancer cells. The MYC oncogene is overexpressed in hematologic cancers, including acute myeloid leukemia (AML). Aptose researchers have reported the ability of APTO-253 to induce cell death, or apoptosis, in multiple blood cancer cell lines including AML, as well as in vitro synergy with various classes of conventional approved and investigational therapies for AML or myelodysplastic syndromes (MDS). New findings reveal that APTO-253 might also serve certain solid tumor patients with BRCA1/2 mutations, but without causing toxicity to the normal bone marrow functions.

Vyxeos™ Receives Positive CHMP Opinion for Treatment of Certain Types of High-Risk Acute Myeloid Leukaemia

On June 29, 2018 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending marketing authorisation of Vyxeos 44 mg/100 mg powder for concentrate for solution for infusion for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) (Press release, Jazz Pharmaceuticals, JUN 29, 2018, View Source;p=RssLanding&cat=news&id=2356615 [SID1234527512]). Vyxeos is an advanced liposomal formulation that delivers a fixed-ratio (1:5) of daunorubicin and cytarabine.

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"Jazz is committed to bringing new and clinically meaningful treatment options to patients on a global basis, and we now look forward to bringing Vyxeos to adults with AML in the European Union," said Allen Yang, M.D., Ph.D., vice president, hematology/oncology therapeutic area head, and acting chief medical officer at Jazz Pharmaceuticals. "If approved by the European Commission, Vyxeos will become the first chemotherapy treatment option specifically for European patients with therapy-related AML or AML with myelodysplasia-related changes."

The Marketing Authorisation Application (MAA) for Vyxeos includes clinical data from five studies, including the pivotal Phase 3 study. Data from the Phase 3 study, which met its primary endpoint, were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2016.

Jazz Pharmaceuticals filed a MAA for Vyxeos in November 2017 after the CHMP granted accelerated assessment, which is designed to reduce the review timeline for products of major interest for public health and therapeutic innovation. The positive opinion from the CHMP will be reviewed by the European Commission, which has the authority to approve medicines in all European Union Member States, Iceland, Norway and Liechtenstein.

About Vyxeos
Vyxeos 44 mg/100 mg powder for concentrate for solution for infusion is an advanced liposomal formulation that delivers a fixed-ratio (1:5) of daunorubicin and cytarabine to the bone marrow that has been shown to have synergistic effects at killing leukaemia cells in vitro and in animal models. Vyxeos is the first product developed with the company’s proprietary CombiPlex platform, which enables the design and rapid evaluation of various combinations of therapies. Vyxeos received Orphan Drug Designation by the European Commission in January 2012 and by the U.S. Food and Drug Administration (FDA) in September 2008 for the treatment of AML. Vyxeos received Promising Innovative Medicine (PIM) designation from the Medicines and Healthcare Products Regulatory Agency in the United Kingdom. Vyxeos received U.S. FDA approval and orphan drug exclusivity on August 3, 2017 for the treatment of adults with newly-diagnosed t-AML or AML-MRC.

About CombiPlex
The CombiPlex proprietary technology enables the design and rapid evaluation of various combinations of therapies to deliver enhanced anti-cancer activity. The CombiPlex technology seeks to identify the most synergistic ratio of drugs in vitro and fix this ratio in a nano-scale delivery complex that maintains the synergistic combination after administration. CombiPlex utilizes two proprietary nano-scale delivery platforms: liposomes to control the release and distribution of water-soluble drugs and drugs that are both water- and fat-soluble (amphipathic), and nanoparticles to control the release and distribution of non-water-soluble (hydrophobic) drugs.

About AML
Acute myeloid leukaemia (AML) is a blood cancer that begins in the bone marrow, which produces most of the body’s new blood cells.1 AML cells crowd out healthy cells and move aggressively into the bloodstream to spread cancer to other parts of the body.2 The median age at diagnosis is 68 years old, with rising age associated with a progressively worsening prognosis.3-4 There is also a reduced tolerance for intensive chemotherapy as patients age.5 Patients with t-AML or AML-MRC have few treatment options and some of the lowest survival rates compared to people with other forms of leukaemia.6-7 A hematopoietic stem cell transplant (HSCT) may be a curative treatment option for patients.8

Chi-Med to Announce 2018 Half-Year Financial Results

On June 29, 2018 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) reported that it will be announcing its interim results for the six months ended June 30, 2018 on Friday, July 27, 2018 at 7:00 am British Summer Time (BST) (Press release, Hutchison China MediTech, JUN 29, 2018, View Source [SID1234527511]).

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An analyst presentation will be held at 9:00 am BST (4:00 pm Hong Kong Time) on the same day at Citigate Dewe Rogerson, 3 London Wall Buildings, London, EC2M 5SY, UK, which will be webcast via the company website at www.chi-med.com/investors/event-information/. The presentation will be available to download before the analyst presentation begins.

For North America based analysts and investors, Chi-Med will also host a conference call with Q&A at 9:00 am Eastern Daylight Time (2:00 pm BST).

Details of the analyst presentation and conference call dial-in will be provided in the financial results announcement. A replay will also be available on the website shortly after each event.