On December 18 2017 Galera Therapeutics, Inc. reported positive results from its Phase 2b clinical trial for its lead drug candidate, GC4419 for severe oral mucositis (SOM) in patients with head and neck cancer receiving chemoradiation (Press release, Galera Therapeutics, DEC 18, 2017, View Source [SID1234522697]). In the intent-to-treat population, the 90 milligram (mg) dose of GC4419 met its primary endpoint, demonstrating a highly significant (p=0.024), clinically meaningful and dose-dependent reduction in the duration of SOM as defined by the World Health Organization as Grade 3 or 4. In the pre-specified secondary endpoints, GC4419 demonstrated a consistent effect across all efficacy measures.

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"We are excited by these results and believe these are robust data in this patient population. Up to 70 percent of patients with head and neck cancer receiving radiotherapy experience severe oral mucositis, and GC4419 has the potential to become an important treatment in a therapeutic area that has not seen meaningful innovation," said J. Mel Sorensen, M.D., Galera Therapeutics, President and Chief Executive Officer. "The results of this trial validate the superoxide dismutase mimetic mechanism of action of GC4419, demonstrating its role in reducing the side effects of radiation therapy. We look forward to discussing the results of this trial and our next steps with the FDA."

The 223-patient, double blind, randomized, placebo-controlled trial treated patients with head and neck cancer 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. The primary outcome measure was the duration of SOM experienced by patients scheduled to receive seven weeks of radiation therapy plus cisplatin.

GC4419 was well tolerated and the frequency of treatment-related side effects was comparable across all treatment arms in the trial.

"Oral mucositis is a common and debilitating side effect of chemoradiation to treat head and neck cancer. Oral mucositis can severely and negatively impact quality of life, treatment outcomes and recovery, as many patients are unable to eat or drink for several weeks," said Carryn Anderson, M.D., Head and Neck Radiation Oncologist, University of Iowa. "New treatment options are urgently needed to reduce or eliminate this problem, and the Phase 2b data on GC4419 suggests a new approach to mitigate oral mucositis in patients with head and neck cancer by preventing the damaging effects of radiation in normal tissue."

About Oral Mucositis

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 as defined by the World Health Organization as Grade 3 or 4, which is the most debilitating side effect of the radiotherapy. Oral mucositis can also adversely affect cancer treatment outcomes. In addition, patients with severe OM suffer significant pain, may be unable to eat solid food or even drink liquids, and may develop 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. Importantly, severe OM may result in interruptions in radiotherapy, which can compromise the otherwise good prognosis for tumor control in many of these patients. There is currently no drug approved to prevent or treat severe OM in patients with head and neck cancer.

On December 18, 2017 Innovation Pharmaceuticals, (IPIX) ("the Company"), a clinical stage biopharmaceutical company, reported an update on the developmental plan for Brilacidin, its first-in-class defensin-mimetic drug candidate, for the prevention and treatment of Oral Mucositis (OM) (Press release, Innovation Pharmaceuticals, DEC 18, 2017, View Source [SID1234522691]). On December 11, 2017, the Company released topline data from a Phase 2 trial. The study met its primary endpoint of reducing the incidence of severe OM experienced by patients receiving chemoradiation for treatment of Head and Neck Cancer (HNC).

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Oral Mucositis is a disease which is largely unfamiliar to the general public, many of whom haven’t witnessed the harsh side effects of OM first hand— namely extreme difficulty swallowing and painful sores in the mouth that can be so intense that cancer treatment must be stopped and a feeding tube inserted. There are currently no approved drugs for the prevention of severe OM in patients receiving chemoradiation treatment of HNC. Moreover, OM is a consequence of therapy for an array of cancers, not just HNC, where the incidence rate is particularly high.

Significant value inflection point for shareholders

Innovation Pharmaceuticals’ Brilacidin is now anchored in OM, representing a value inflection point for shareholders, as further supported by its lead positioning in an untapped $1 Billion market. "Anchored" is an industry term indicating a drug has delivered a meaningful response in a clinical setting for a specific disease along with good apparent safety and toleration. As such, Brilacidin-OM now represents a significant asset to the company. The next steps will be aimed at defining the most appropriate development plan to complete its evaluation prior to submission for marketing approval. This will involve consultation with the FDA and other health authorities worldwide, once full efficacy and safety endpoint data are available from the recent clinical trial.

Towards commercial planning, the Company has begun exploring potential unit dose drug product packaging in the form of a sachet. Sachets, which people are very familiar with and use on an almost daily basis (e.g., sugar packets, artificial sweeteners) increasingly are being developed as a novel means of patient-friendly, drug delivery. Manufacturing plans for drug substance appropriate for late phase testing and eventual market introduction are also underway.

As there are currently no approved drugs for prevention of severe OM in patients receiving chemoradiation treatment of HNC, management opinion is that Brilacidin-OM has the potential to rapidly assume a lead position after market introduction. This perspective is shared by multiple Pharma companies, potential partners the Company currently is in active discussions with, equally interested in the continued development and eventual market introduction of Brilacidin-OM worldwide.

"I am extremely proud that our team has achieved this very important milestone in cancer care and I feel very optimistic for the future. There is an urgent need for a drug such as Brilacidin to help cancer patients facing the threat of OM. We will be pursuing every option available to us toward providing these patients with a new therapeutic option," commented Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. "I have spoken with family members of patients dealing with OM and to hear the pain and suffering is simply heart-wrenching. I envision a day where doctors will have a viable option for preventing and treating severe OM and if Brilacidin-OM could be that drug, it would be a considerable accomplishment for the Company, our shareholders and the entire oncology industry."

About Brilacidin

Brilacidin is Innovation Pharmaceuticals’ lead drug candidate in its defensin mimetic franchise. Modeled after Host Defense Proteins (HDPs), the "front-line" of defense in the immune system, it is a small, non-peptidic, synthetic molecule that kills pathogens swiftly and thoroughly. Just as importantly, Brilacidin also functions in a robust immunomodulatory capacity, lessening inflammation and promoting healing. Due to its unique properties, the Company is studying Brilacidin’s effect on Oral Mucositis (under Fast Track designation) and on Ulcerative Proctitis / Proctosigmoiditis (UP/UPS) in Phase 2 trials. Additional trials of Brilacidin are planned in other conditions, including: Atopic Dermatitis, Hidradenitis Suppurativa and Acne. Brilacidin is also being developed under FDA’s Qualified Infectious Disease Product (QIDP) designation as an antibacterial product for Acute Bacterial Skin and Skin Structure Infection (ABSSSI)—qualifying it for Fast Track and possible Priority FDA Review and an extra 5 years of United States market exclusivity upon drug approval.

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About Brilacidin-OM

Innovation Pharmaceutical’s first-in-class immunomodulatory drug candidate, Brilacidin, targets the prevention of severe Oral Mucositis (OM)—a common and debilitating side-effect of receiving chemoradiation therapy—in Head and Neck Cancer. Each year, OM affects hundreds of thousands of patients worldwide. Only a limited number of OM treatments are available, most of which are palliative in nature. A Phase 2 randomized, placebo-controlled clinical trial of Brilacidin-OM (see NCT02324335) has been recently completed in which topline results demonstrate a reduced rate of severe OM (WHO Grade >3) in patients treated with Brilacidin-OM compared to those on placebo.

About Oral Mucositis

Oral Mucositis (OM) is a frequent, painful and debilitating complication of chemoradiation. Head and Neck Cancer (HNC) patients—comprising an estimated 65,000 newly diagnosed cases in the U.S. alone in 2017, and an estimated 700,000 worldwide (source:GLOBOCAN)—are at the greatest risk of developing OM (a 90 to 100 percent rate of occurrence). By 2030, the global incidence of HNC cases is expected to exceed 1 million per year. Moreover, between 25 and 60 percent of cancer patients, regardless of cancer type, also will experience OM. Characterized by inflammation and ulceration, patients suffering from OM are often unable to speak and eat (requiring the insertion of a feeding tube) and are more susceptible to infections, with severe cases leading to hospitalization at increased treatment costs of up to $25,000. There currently are no approved medications for the prevention of OM in the HNC population, with only limited palliative care options available. Worldwide, the potential marketfor OM is expected to exceed $1 billion in the next few years.

On December 18, 2017 Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, reported that the first patient in its Phase 1/2 dose escalation and dose expansion clinical trial (see www.clinicaltrials.gov, identifier NCT03340883) has been dosed at Virginia Cancer Specialists in Fairfax, Virginia, the first site in this multi-center trial (Press release, Aduro Biotech, DEC 18, 2017, View Source;p=RssLanding&cat=news&id=2323132 [SID1234522686]). The study is designed to evaluate the safety and activity of BION-1301, a humanized anti-APRIL (A PRoliferation-Inducing Ligand) antibody, for adults with relapsed or refractory multiple myeloma.

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"Neutralizing APRIL is a differentiated approach in the treatment of multiple myeloma, and it has been shown to inhibit tumor growth and overcome drug resistance in preclinical studies," said Natalie Sacks, M.D., chief medical officer of Aduro Biotech. "BION-1301 is our wholly-owned novel antibody that has been shown in non-clinical studies to fully block the APRIL-induced signaling cascade at a critical juncture, and we are eager to further characterize its potential activity in the clinic. Until there is a cure for multiple myeloma which remains a debilitating disease, alternative treatments are needed."

Recently, two datasets on anti-APRIL were presented at the 59TH American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta, Georgia. Data presented by Yu-Tzu Tai, Ph.D., of the Dana Farber Cancer Institute in an oral session at ASH (Free ASH Whitepaper) demonstrate that BION-1301 and its parental antibody blocked APRIL binding to both its receptors BCMA and TACI. Blocking APRIL with BION-1301 (or its parental antibody) not only inhibited proliferation and survival of multiple myeloma cells but it also alleviated drug resistance and immune suppression in preclinical models and cell culture, leading to enhanced anti-BCMA and daratumumab-mediated myeloma cell killing. Additionally, Dr. Tai and her colleagues demonstrated that regulatory T cells in the blood and bone marrow expressed TACI, and that APRIL blockade inhibited their function. Also, John Dulos, Ph.D., director and project team leader at Aduro Biotech, and his team conducted preclinical pharmacokinetic and pharmacodynamic studies indicating BION-1301 was well tolerated and binding of APRIL in preclinical models resulted in decreased IgA, IgG and IgM production in a dose-dependent fashion.

"We know that APRIL sets off a cascade of events that hinders the immune response to multiple myeloma cells, helping the disease escape immune attack. The data presented at ASH (Free ASH Whitepaper) indicate that blocking APRIL by targeting BCMA alone may not be sufficient for the treatment of multiple myeloma, as APRIL critically modulates regulatory T cell function via TACI, not BCMA," said Dr. Tai. "Importantly, BION-1301, as a fully-blocking monoclonal antibody, may have unique efficacy by inhibiting APRIL-induced proliferation of immune system components linked to multiple myeloma progression."

About Phase 1/2 Trial
The Phase 1/2 multi-center, open-label study is designed to evaluate the safety and activity of BION-1301 in patients with relapsed or refractory multiple myeloma whose disease has progressed after at least 3 prior systemic therapies, including immunomodulatory drugs (IMiDs), proteasome inhibitors, chemotherapies, or monoclonal antibodies. The Phase 1 part of the study will evaluate the safety, pharmacokinetics and pharmacodynamics of escalating doses of BION-1301 administered once every two weeks in a 28-day cycle. Once the recommended Phase 2 dose is determined, the Phase 2 part of the study will begin. It will assess safety and preliminary activity of BION-1301 at the selected dose, with a primary activity endpoint of objective response rate.

About APRIL and BION-1301
APRIL (A PRoliferation-Inducing Ligand) is a member of the tumor necrosis factor (TNF) superfamily and is primarily secreted by bone marrow and/or myeloid cells. APRIL is overproduced in patients with multiple myeloma and binds to BCMA (B cell maturation antigen) and TACI (Transmembrane Activator and CAML Interactor) to stimulate a wide variety of responses that promote multiple myeloma growth and suppress the immune system so that the tumor cells are allowed to proliferate. BION-1301 is a humanized anti-APRIL antibody that has been shown in preclinical studies to effectively neutralize APRIL, eliminate malignant cells and reduce resistance to therapy in models of multiple myeloma. In addition to multiple myeloma, APRIL’s role in other cancers and in B cell dependent autoimmune and inflammatory diseases indicate that BION-1301 may also be useful in treating chronic lymphocytic leukemia, colorectal cancer and Berger’s disease (caused by IgA antibody deposits in the kidneys).

About Multiple Myeloma
Lymphocytes (B cells and T cells) are the primary cell types within the immune system that work together to fight infection and disease. As B cells respond to normal infection in the body, they mature and change into plasma cells, which in turn make antibodies that help the body attack infection. While lymphocytes circulate throughout the body, plasma cells remain primarily in the bone marrow. Multiple myeloma is a blood cancer that occurs when malignant plasma cells proliferate uncontrollably. Approximately 50,000 new cases of multiple myeloma are expected to be diagnosed in the United States and Europe each year. While many new therapies have become available in recent years, multiple myeloma remains incurable and significant unmet needs exist among patients who relapse following, are resistant to, or cannot tolerate currently available agents.

On December 18, 2017 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology, reported that it has named John Leonard, M.D., President and Chief Executive Officer (CEO), effective January 1, 2018 (Press release, Intellia Therapeutics, DEC 18, 2017, View Source [SID1234522683]). Dr. Leonard succeeds Nessan Bermingham, Ph.D., Intellia’s founding President and CEO, who is returning to the venture capital industry. In recognition of the company’s growth and expanding pipeline, the Board of Directors and Dr. Bermingham agreed that Dr. Leonard should lead the Company as it progresses toward clinical development, given his experience in successfully developing biopharmaceutical products and leading large scientific organizations.

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Dr. Leonard joined Intellia in 2014 as the Company was being formed. He initially served as Intellia’s Chief Medical Officer and as a member of its Board of Directors, and together with Dr. Bermingham, set the strategic direction of the Company. In 2016, Dr. Leonard became Intellia’s Executive Vice President of Research & Development (R&D). Prior to Intellia, Dr. Leonard was Chief Scientific Officer and Senior Vice President of Research and Development at AbbVie, Inc., a global biopharmaceutical company, which was spun off from Abbott Laboratories in 2013. At Abbott, Dr. Leonard served as Senior Vice President of Global Pharmaceutical Research & Development from 2008 to 2012.

His combined tenure at Abbott and AbbVie spanned 22 years from 1992, until his retirement from AbbVie in 2013. Dr. Leonard was responsible for the development of the groundbreaking HIV protease inhibitors Norvir and Kaletra, which led to new treatment paradigms for HIV/AIDS.

He also led AbbVie’s HCV programs, laid the foundation for its Oncology effort and guided the development of HUMIRA, the all-time, top-selling pharmaceutical product worldwide.

"After nearly four years of building Intellia and this exceptional team, the Company is now poised to begin development of CRISPR/Cas9-based therapies," says Nessan Bermingham, Ph.D. "At this stage, Intellia requires a CEO with a track record of successful drug development. John was the first to join me in starting Intellia, and was an ideal partner because of his unmatched R&D expertise and biopharmaceutical leadership experience. As I return to my roots in biotech venture capital, I am confident that Intellia, the science and our employees are in great hands."

"With more than a 30-year career in biopharmaceutical R&D, John is well recognized as a premier R&D leader, having developed many breakthrough medicines that turned into life-improving therapies for patients. As Intellia progresses towards clinical development, we are fortunate that he will lead the company," says Intellia’s Chairman Perry Karsen. "We thank Nessan for his passion and leadership in taking Intellia from an idea through preclinical science, initial public offering and the path to the clinic. As Intellia’s founder, Nessan had great vision for what was possible with the CRISPR/Cas9 technology and its application in human therapeutics."

"Intellia has made significant progress in advancing the CRISPR/Cas9 technology and in applying it in our pre-clinical programs, as well as in those of our partners Novartis and Regeneron," says John Leonard, M.D. "As we enter the next phase, our ambition and business strategy remains the same. We will build on Nessan’s vision, and advance our mission of developing curative genome editing treatments that can positively transform the lives of people living with severe and life-threatening diseases."

On December 18, 2017 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, reported an update on its Phase II clinical trial with drug candidate Namodenoson (CF102) in the treatment of advanced hepatocellular carcinoma (HCC). Ongoing close observation of enrolled subjects indicates a potentially favorable drug safety profile (Press release, Can-Fite BioPharma, DEC 18, 2017, View Source [SID1234522681]). The Company previously announced in August that it had successfully completed enrollment of 78 patients.

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The global Phase II study is being conducted in the U.S., Europe and Israel. Patients with advanced HCC, Child Pugh B, who failed Nexavar (sorafenib) as a first line treatment are treated twice daily with 25 mg of oral Namodenoson or placebo using a 2:1 randomization. The primary endpoint of the Phase II study is Overall Survival (OS). Secondary endpoints include Progression Free Survival (PFS), safety, and the relationship between outcomes and A3AR expression.

Although the trial remains blinded to the Company, accumulated safety data to date indicates a potentially favorable drug safety profile without hepatotoxicity and possible positive clinical effects. There are now subjects treated for more than one year and in some cases, two years. To date, 15 subjects have completed at least 12 cycles of treatment (each cycle is 28 days of treatment) of which two completed 24 cycles. The Company anticipates data release to occur in 2H2018.

Can-Fite CEO Dr. Pnina Fishman commented, "We are pleased with the progress so far in our clinical trial for Namodenoson for the treatment of advanced HCC, the third leading cause of cancer deaths worldwide, and look forward to data release later in 2018."

Can-Fite received Orphan Drug Designation for Namodenoson in Europe and the U.S., as well as Fast Track Status in the U.S. as a second line treatment for HCC.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is being evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.