On August 29, 2017 Augustus BioTarget reported that An early-stage drug development company in South Carolina has launched an online fundraising campaign to study the ancient spice curcumin in combination with an FDA-approved Immune Checkpoint Inhibitor to fight colon cancer (Press release, Augustus BioTarget, AUG 29, 2017, http://augustusbiotarget.com/wp-content/uploads/2017/08/Press-Release-ABT-August-29-2017.pdf [SID1234556207]).

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Within five days of launch, the company has met 78% of its funding goal on Experiment.com, an online science crowdfunding platform dedicated to scientific research. https://experiment.com/curcumincancerproject

A group of scientists working in the US at Augustus BioTarget, Inc., and in Germany at Rodos Biotarget, GmbH, developed a unique nanocarrier drug delivery system, the CLR-TargoSphere, into which curcumin was encapsulated. This will be studied in mice with colon cancer at the Charles River Labs in North Carolina.

"Our aim is to resurrect the immune response against cancer with targeted curcumin," said Michael Scolaro, founder of Augustus BioTarget and Chief Investigator. "Curcumin is known to possess significant anti-inflammatory and anti-neoplastic properties. But because it is very poorly absorbed and distributed in the body, it has been impractical for clinical use. Delivering curcumin directly to immune cells that are immobilized by cancers promises to help restore anticancer activity.

"Using curcumin in medicine may not secure huge capital investments the way a new iPad does," he added. ‘But with the latest advancements in immune system targeting, its use to fight cancer is now within reach."

On August 28, 2018 the U.S. Food and Drug Administration reported that it took decisive action to prevent the use of a potentially dangerous and unproven treatment belonging to StemImmune Inc. in San Diego, California, and administered to patients at the California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, California (Press release, US FDA, AUG 28, 2017, View Source [SID1234526938]). On behalf of the FDA, on Friday, Aug. 25, 2017 the U.S. Marshals Service seized five vials of Vaccinia Virus Vaccine (Live) – a vaccine that is reserved only for people at high risk for smallpox, such as some members of the military. Each of the vials originally contained 100 doses of the vaccine, and although one vial was partially used, four of the vials were intact.

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As the vaccine is not commercially available, the FDA has serious concerns about how StemImmune obtained the product for use as part of an unapproved and potentially dangerous treatment. The FDA is actively investigating the circumstances by which StemImmune came to possess the vaccine.

"Speaking as a cancer survivor, I know all too well the fear and anxiety the diagnosis of cancer can have on a patient and their loved ones and how tempting it can be to believe the audacious but ultimately hollow claims made by these kinds of unscrupulous clinics or others selling so-called cures," said FDA Commissioner Scott Gottlieb, M.D. "The FDA will not allow deceitful actors to take advantage of vulnerable patients by purporting to have treatments or cures for serious diseases without any proof that they actually work. I especially won’t allow cases such as this one to go unchallenged, where we have good medical reasons to believe these purported treatments can actually harm patients and make their conditions worse."

The seizure comes after recent FDA inspections at StemImmune Inc. and the California Stem Cell Treatment Centers confirmed that the vaccine was used to create an unapproved stem cell product (a combination of excess amounts of vaccine and stromal vascular fraction – stem cells derived from body fat), which was then administered to cancer patients with potentially compromised immune systems and for whom the vaccine posed a potential for harm, including myocarditis and pericarditis (inflammation and swelling of the heart and surrounding tissues). The unproven and potentially dangerous treatment was being injected intravenously and directly into patients’ tumors.

Serious health problems, including those that are life-threatening, can also occur in unvaccinated people who are accidentally infected with the vaccinia virus by being in close contact with someone who has recently received the vaccine. In particular, unvaccinated people who are pregnant, or have problems with their heart or immune system, or have skin problems like eczema, dermatitis, psoriasis and have close contact with a vaccine recipient are at an increased risk for inflammation and swelling of the heart and surrounding tissues if they become infected with the vaccine virus, either by being vaccinated or by being in close contact with a person who was vaccinated.

"I’ve directed the agency to vigorously investigate these kinds of unscrupulous clinics using the full range of our tools, be it regulatory enforcement or criminal investigations. Our actions today should also be a warning to others who may be doing similar harm, we will take action to ensure Americans are not put at unnecessary risk," Gottlieb added. "I also urge health care providers, patients and consumers to report these kinds of activities or any adverse events associated with these unproven treatments to the agency through MedWatch."

Health care professionals and consumers should report any adverse events related to treatments received at California Stem Cell Treatment Center to the FDA’s MedWatch Adverse Event Reporting program. To file a report, use the MedWatch Online Voluntary Reporting Form. The completed form can be submitted online or via fax to 1-800-FDA-0178.

The U.S. Department of Justice filed the seizure complaint, on behalf of the FDA, in the U.S. District Court for the Central District of California.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

On August 23, 2017 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) and Nippon Shinyaku Co., Ltd. (TOKYO: 4516) reported that Chugai filed a new drug application to the Ministry of Health, Labour and Welfare (MHLW), for glycoengineered type II anti-CD20 monoclonal antibody, obinutuzumab (genetical recombination) which was co-developed by the two companies for the treatment of "CD20-positive B-cell follicular lymphoma (FL)" in Japan (Press release, Chugai, AUG 23, 2017, View Source [SID1234520310]).

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"Combination therapy of rituximab and chemotherapy has been used as the standard treatment for CD20-positive B-cell FL for a long time. As a new treatment option, Obinutuzumab was confirmed to be more beneficial than the conventional treatment with rituximab for the treatment of FL," said Dr. Yasushi Ito, Chugai’s Senior Vice President, Head of Project & Lifecycle Management Unit. "We are committed to deliver obinutuzumab to patients as early as possible to contribute to the access to better treatments in Japan."

Dr. Kazuya Mori, Nippon Shinyaku’s Corporate Officer, Head of R&D Administration Div. said "I am very glad that a new drug application for our co-developed product, obinutuzumab, was filed. By adding this new product to our lineup in the area of hematologic malignancies, on which we are focusing, we will make utmost efforts to meet the demands of the clinical setting and contribute to the treatment of patients."

This filing was based on the results of the GALLIUM study, a global phase III clinical study conducted by Roche and participating from Japan and other several clinical studies.
The GALLIUM study is a global Phase III open-label, multi-center, randomized two-arm study that evaluated the efficacy and safety of obinutuzumab plus chemotherapy followed by obinutuzumab alone (obinutuzumab arm) compared with rituximab plus chemotherapy followed by rituximab alone (rituximab arm) in 1,401 patients with previously untreated CD20-positive advanced non-Hodgkin’s lymphoma. The primary endpoint of the study was investigator-assessed progression free survival (PFS) in 1,202 patients with FL. The secondary endpoints were PFS assessed by an independent review committee (IRC), overall survival (OS), safety, and other endpoints.

With respect to the primary endpoint, as the median PFS was not reached, the results showed that obinutuzumab arm reduced the risk of disease progression, recurrence or death in patients with FL by 34 percent (HR=0.66, 95% CI: 0.51-0.85, p=0.0012, stratified log-rank test, Data cut-off: January 31, 2016) compared to rituximab arm. Concerning secondary endpoints, the median PFS assessed by the IRC was not reached as well, the risk of disease progression, recurrence or deaths decreased by 29% (HR=0.71, 95% CI: 0.54-0.93], p=0.0138, stratified log-rank test, Data cut-off: January 31, 2016). The median OS did not reach in both arms due to small numbers of events. As for safety, the adverse events (AEs) expressed in both arms in the GALLIUM study were consistent with previous reports. The Grade 3 or higher AEs which was observed in 5% or more frequently for obinutuzumab arm than rituximab arm was neutropenia (43.9% for obinutuzumab arm vs. 37.9% for rituximab arm).

Obinutuzumab is a glycoengineered type II anti-CD20 monoclonal antibody designed to attach to CD20, a protein expressed on certain B cells, but not on stem cells or plasma cells, same as rituximab which is recommended as a treatment of non-Hodgkin’s lymphoma in treatment guidelines in Japan and overseas. Obinutuzumab is designed to attack and destroy targeted B-cells both directly and together with the body’s immune system.

In Japan, the prevalence of malignant lymphoma was reported to be approximately 27,000 and the number of deaths due to the disease was reported to be approximately 11,000 in 20121, 2). Since the cases of Hodgkin’s lymphoma is reported to account for approximately 8 to 10% of the cases of malignant lymphoma in Japan3), the prevalence of non-Hodgkin’s lymphoma is estimated to be approximately 24,000 and the number of deaths from this condition is estimated to be approximately 10,000. The prevalence of and deaths from malignant lymphoma tend to increase in recent years1, 2), and a same tendency is seen in patients with non-Hodgkin’s lymphoma.

Chugai and Nippon Shinyaku will work for the early approval to provide obinutuzumab as a new treatment option for patients with CD20-positive B-cell follicular lymphoma and medical professionals.

On August 21, 2017 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of disease-driving genes, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SY-1425 for the treatment of acute myeloid leukemia (AML) (Press release, Syros Pharmaceuticals, AUG 21, 2017, View Source [SID1234520301]). SY-1425, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently in a Phase 2 clinical trial in genomically defined subsets of patients with AML and myelodysplastic syndrome (MDS).

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"Treatment of AML remains a significant unmet medical need, with many patients lacking adequate therapeutic options," said David A. Roth, M.D., Syros’ Chief Medical Officer. "We believe that SY-1425 may provide a meaningful benefit for subsets of AML patients whose disease is driven by abnormally high expression of the RARA or IRF8 genes. Receiving orphan drug designation is an important regulatory milestone in the development of SY-1425. We’re pleased with the continued progress of the ongoing Phase 2 clinical trial, and we look forward to presenting initial clinical data in the fourth quarter of this year."

The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

Using its gene control platform, Syros discovered subsets of AML and MDS patients with super-enhancers associated with RARA or IRF8. Syros identified proprietary biomarkers related to these super-enhancers. These super-enhancers are believed to drive overexpression of the RARA or IRF8 genes, locking cells in an immature, undifferentiated and proliferative state, leading to disease. In preclinical studies, SY-1425 promoted differentiation of AML cells with high RARA or IRF8 expression and inhibited tumor growth and prolonged survival in patient-derived xenograft models of AML with high RARA expression. Syros estimates that about one-third of AML and MDS patients have either the RARA or IRF8 biomarker, or both.

The ongoing Phase 2 clinical trial of SY-1425 is assessing the safety and efficacy of SY-1425 as a single agent in four AML and MDS patient populations, as well as in combination with azacitidine, a standard-of-care therapy, in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy. All patients in the trial are prospectively selected using biomarkers for high expression of RARA or IRF8. Additional details about the trial can be found using the identifier NCT02807558 at www.clinicaltrials.gov.

On August 21, 2017 Blueprint Medicines Corporation (NASDAQ: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, reported that updated data from its ongoing Phase 1 clinical trial evaluating BLU-554 in patients with advanced hepatocellular carcinoma (HCC) will be presented in oral presentations at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress on September 10, 2017 in Madrid, Spain and at the 11th International Liver Cancer Association (ILCA) Annual Conference on September 17, 2017 in Seoul, South Korea (Press release, Blueprint Medicines, AUG 21, 2017, View Source;p=irol-newsArticle&ID=2294896 [SID1234520303]). BLU-554 is a potent and highly selective inhibitor of FGFR4.

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At ESMO (Free ESMO Whitepaper) and ILCA, Blueprint Medicines anticipates presenting updated safety and clinical activity data from its ongoing Phase 1 clinical trial of BLU-554. As of the most recent data cutoff date of August 18, 2017, 38 patients with FGFR4 pathway activation, as indicated by FGF19 overexpression, were evaluable for clinical activity, and an objective response rate of 16 percent (95 percent confidence interval 6-31 percent) was observed in this population.

"The preliminary BLU-554 data announced today continue to provide support for selective FGFR4 inhibition as a novel targeted treatment approach in biomarker-selected patients with hepatocellular carcinoma," said Andy Boral, M.D., Ph.D., Chief Medical Officer of Blueprint Medicines. "As we evaluate the results for BLU-554 in the context of historical data for currently approved therapies showing response rates of less than 10 percent, we are encouraged that radiographic tumor reductions were observed in a refractory population with progressive disease and few or no remaining therapeutic options. We look forward to sharing a comprehensive update on the ongoing BLU-554 clinical trial, as well as the path forward for further development, in September."

Details of the presentations are as follows:

2017 ESMO (Free ESMO Whitepaper) Congress

Presentation Title: Phase 1 Safety and Clinical Activity of BLU-554 in Advanced Hepatocellular Carcinoma (HCC)
Session Title: Developmental Therapeutics
Date & Time: Sunday, September 10, 2017 from 5:30 – 5:45 p.m. CET (11:30 – 11:45 a.m. ET)
Abstract ID: 365O
Location: Cordoba Auditorium, IFEMA, Feria de Madrid

11th ILCA Annual Conference

Presentation Title: Clinical Activity Of BLU-554, A Potent, Highly-Selective FGFR4 Inhibitor In Advanced Hepatocellular Carcinoma (HCC) With FGFR4 Pathway Activation
Session Title: General Session 5
Date & Time: Sunday, September 17, 2017 from 11:30 a.m. – 1:00 p.m. KST (Saturday, September 16, 2017 from 10:30 p.m. – 12:00 a.m. ET)
Abstract No: O-032
Location: Grand Hyatt Seoul