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Novartis drug PKC412 (midostaurin) receives Breakthrough Therapy designation from the FDA for newly-diagnosed FLT3-mutated acute myeloid leukemia (AML)

On February 19, 2016 Novartis reported that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to PKC412 (midostaurin)(Press release, Novartis, FEB 19, 2016, View Source [SID:1234509099]). PKC412 (midostaurin) is an investigational treatment for adults with newly-diagnosed AML who are FLT3 mutation-positive, as detected by an FDA-approved test, and who are eligible to receive standard induction and consolidation chemotherapy.

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The Breakthrough Therapy designation for PKC412 (midostaurin) is primarily based upon the positive results from the Phase III RATIFY (CALGB 10603) clinical trial. This study was conducted in partnership with the Alliance for Clinical Trials in Oncology and presented during a plenary session at the 57th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting[4].

Patients who received PKC412 (midostaurin) and standard induction and consolidation chemotherapy experienced a significant improvement in overall survival (OS) (hazard ratio = 0.77, P = 0.0074) compared to those who received standard induction and consolidation chemotherapy alone[4]. The median OS for patients in the PKC412 (midostaurin) treatment group was 74.7 months (95% confidence interval [CI]: 31.7, not attained), versus 25.6 months (95% CI: 18.6, 42.9) for patients in the placebo group[4]. No statistically significant differences were observed in the overall rate of grade 3 or higher hematologic and non-hematologic adverse events in the PKC412 (midostaurin) treatment group versus the placebo group[4]. A total of 37 deaths were reported, with no difference in treatment-related deaths observed between groups[4].

"For more than 25 years, medical developments have been limited for AML patients and the chemotherapy treatment strategy has essentially remained unchanged," said Alessandro Riva, MD, Global Head, Novartis Oncology Development and Medical Affairs. "We look forward to working closely with the FDA to bring PKC412 (midostaurin), the first potential AML targeted therapy, to patients as quickly as possible."

According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of new medicines that treat serious or life-threatening conditions, if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint. The designation includes all of the Fast Track program features, as well as more intensive FDA guidance on an efficient drug development program[5].

This designation adds to the growing number granted to Novartis by the FDA, illustrating the company’s continued commitment to developing innovative therapies for diseases with a significant unmet medical need.

In the US, about 20,000 people were diagnosed with AML in 2015, the majority of whom were adults[6]. According to the latest research, approximately one-third of AML patients also harbor a FLT3 gene mutation[7], which is associated with worse outcomes and shorter survival than in those without the mutation[8]. PKC412 (midostaurin) is the first drug targeting FLT3 to demonstrate an overall survival benefit in AML[4].

Since PKC412 (midostaurin) is investigational at this time and is expected to be submitted for FDA approval, Novartis opened a Global Individual Patient Program (compassionate use program) and a US Expanded Treatment Protocol (ETP) to enable PKC412 (midostaurin) access. Patients 18 years of age and older with newly-diagnosed FLT3-mutated AML and able to receive standard induction and consolidation therapy will be considered.

In order to help identify patients who may have a FLT3 mutation and potentially benefit from treatment with PKC412 (midostaurin), Novartis is collaborating with Invivoscribe Technologies, Inc. who is leading regulatory submissions for a companion diagnostic.

About acute myeloid leukemia (AML) and the FLT3 mutation
AML is an aggressive cancer of the blood and bone marrow[9]. It prevents white blood cells from maturing, causing an accumulation of "blasts" which do not allow room for the normal blood cells[9]. AML is the most common acute leukemia in adults, but also has the lowest survival rate[1]. AML accounts for approximately 25% of all adult leukemias worldwide, with the highest incidence rates occurring in the United States, Europe and Australia[1].

Mutations in specific genes are found in many cases of AML, and biomarker testing is considered standard of care for newly-diagnosed patients to help determine the best possible treatment option[7]. FMS-like tyrosine kinase-3 (FLT3) is a receptor tyrosine kinase, a type of cell-surface receptor, which plays a role in the proliferation, or increase, in the number of certain blood cells[10].

About PKC412 (midostaurin)
PKC412 (midostaurin) is an investigational, oral, multi-targeted kinase inhibitor in development for the treatment of patients with AML with a FLT3 mutation. The safety and efficacy profile has not been fully established. There is no guarantee that PKC412 (midostaurin) will become commercially available.

PKC412 (midostaurin) is also being investigated for the treatment of aggressive systemic mastocytosis/mast cell leukemia.

BioGenerator Invests in New Immunotherapy Company, Unleash. Company will open operations in St. Louis.

On February 18, 2016 BioGenerator, an evergreen investor that creates, grows and invests in promising companies and entrepreneurs, reported that it has made an investment from its Concept Fund into Unleash Immuno Oncolytics, a company focused on treatments of cancer (Press release, Unleash Immuno Oncolytics, FEB 18, 2016, View Source [SID1234540112]). Unleash is an immune-oncology company developing virus immunotherapy products to treat cancer. The company was developed based on collaborations between Dr. David Curiel, Director, Biologic Therapeutics Center at Washington University School of Medicine and Dr. Osvaldo Podhajcer, Director of the Argentinian Consortium of Genomic Technology and is licensing technology from Leloir Institute. As part of the funding, Unleash will set up headquarters in St. Louis and operate from CIC in the @4240 building.

BioGenerator is the investment arm of BioSTL, a nonprofit civic organization building St. Louis’ innovation ecosystem and which in the last two years has added a focus of recruiting international companies to the region. "Through our GlobalSTL initiative we are leveraging St. Louis’ scientific and business strengths to attract cutting-edge technologies and talent that enrich our region," explained Donn Rubin, president and CEO of BioSTL. "Unleash’s decision to locate in St. Louis is a testament to our world-class strengths in medicine and human health." "Unleash is based on technology from two leaders in the field of oncology and has promising applications," said Charlie Bolten, vice president of BioGenerator. "Immunotherapy is a quickly evolving area of human health and BioGenerator is pleased to be one of the first investors in a company at the cutting edge of research."

Other investors in the round include Axia Ventures, a Latin American investment and support organization. Unleash will use the initial investment to fund IND-enabling tasks including cGMP manufacturing, toxicology and bio-distribution studies for its oncolytic immunotherapy product.

¨We are pleased with BioGenerator’s role as lead investor in this round and establishing Unleash in Saint Louis," said Daniel Katzman, CEO of Unleash. "We are now eager to advance Unleash’s virus immunotherapy products to the clinic."

Unleash Immuno Oncolytics Enters License Agreement with Leloir Institute to Develop Immuno-Oncology Products for Cancer Treatment, Gets Established in Saint Louis

On February 18, 2016 Unleash Immuno Oncolytics, an immune-oncology company developing oncolytic virus immunotherapy products to treat cancer, reported that the company has entered a license agreement with INIS Biotech, the tech transfer arm of Fundación Instituto Leloir, and has established operations in Saint Louis, Missouri, USA (Press release, Unleash Immuno Oncolytics, FEB 18, 2016, View Source [SID1234540111]).

"Immuno-oncology is a very promising field and we are excited to enter this space that could potentially bring truly breakthrough therapies for cancer treatment. Unleash is privileged to license this technology from the Leloir Institute, to leverage this outstanding science to develop innovative immune-oncology therapies" said Unleash’s CEO, Daniel Katzman.

The research work of Dr. Osvaldo Podhajcer´s group led to the development of Unleash’s leading product – UIO-512, an oncolytic virus designed to target both malignant cells and tumor-associated stroma cells. "The stroma, composed by non-malignant cancer-associated fibroblasts, endothelial and immune cells, provides nutrients, structure and helps the malignant cells to grow and disseminate. Cancer-associated stroma cells may account for up to 90% of the tumor mass and are not being specifically and are not targeted by any other current therapies. Thus, targeting the stroma cells is a significant paradigm shift over existing cancer treatments" explained Dr. Podhjacer – Chief, Laboratory of Molecular and Cellular Therapy (Fundación Instituto Leloir, Buenos Aires), Superior Researcher (CONICET) ), Director of the Argentinian Consortium of Genomic Technology.

"UIO-512’s viral replication is triggered by a triple hybrid promoter that combines the stroma-associated SPARC gene promoter and motifs responsive to tumor microenvironment conditions such as inflammation and hypoxia. Unleash’s patented technology is unique in that it attacks the entire tumor mass, not only the malignant cells themselves but also the stromal cells that support cancer dissemination" said Dr. David T. Curiel – Unleash’s Scientific Advisory Board Chairman and Director of the Biologic Therapeutics Center, Washington University School of Medicine.

Unleash was formed by Axia Ventures Company Builder. ¨We are proud to have created an exciting biotechnology start-up company born with the vision of a global world-class scale¨, said Lisandro Bril, Managing Partner of Axia Ventures.

Unleash will establish its operations in Saint Louis. ¨Saint Louis’ growing biotechnology ecosystem is happy to welcome Unleash. We are excited to be the lead investor in Unleash’s seed round of financing", said Charles Bolten, Vice President of BioGenerator, a biotechnology fund and incubator based in Saint Louis, Missouri.

6-K – Report of foreign issuer [Rules 13a-16 and 15d-16]

On February 18, 2016 Rosetta Genomics Ltd. (NASDAQ: ROSG), a leading developer and provider of microRNA-based and other molecular diagnostic testing services, conditional approval status for RosettaGX Reveal, its novel microRNA classifier for the diagnosis of indeterminate thyroid Fine Needle Aspirate (FNA) smears from the New York State Department of Health (NYSDOH) under the Company’s Molecular Oncology permit (Filing, 6-K, Rosetta Genomics, FEB 18, 2016, View Source [SID:1234509216]). RosettaGX Reveal is the only molecular test in the thyroid market that has been validated in a multicenter, international, blinded study using convenient, routinely prepared cytology slides.

The assay is CLIA certified, but New York requires an additional license from the NYSDOH for CLIA-certified tests to be offered to patients in the state. With this conditional approval, RosettaGX Reveal is now available in all 50 states. In making the assay available pending final approval, the NYSDOH requires the Company to provide any additional information that it may request within 60 business days.

"We are very pleased to have approval to market this important cancer diagnostic for the benefit of physicians and patients in the vast New York market," stated Kenneth A. Berlin, President and Chief Executive Officer of Rosetta Genomics. "It is estimated that nearly 550,000 FNAs are performed on thyroid nodules each year in the U.S. and that approximately 740,000 are performed annually in Europe. Interpretation of FNA samples is not always straightforward, leading to an indeterminate result in up to 30% of the samples. Many patients with indeterminate results undergo surgery as a precaution despite the fact that up to 80% of these cases are benign. This exposes patients to unnecessary surgical risk and costs the healthcare system hundreds of millions of dollars. Through an analysis of our validation study data, we believe we can help prevent up to 75% of unnecessary thyroid surgeries."

"In addition to this expanded geographic access, recent managed care contracting initiatives have resulted in covered lives for RosettaGX Reveal exceeding 150 million in the U.S. These increases in geographic and health insurance access, along with the potential health economic benefits the RosettaGX Reveal assay can bring by avoiding unnecessary surgeries, should enhance adoption into a market valued at more than $350 million annually in the U.S. alone. This market is one that continues to see accelerating penetration of molecular classifiers such as RosettaGX Reveal, which we expect to continue over the next several years," added Mr. Berlin.

AbbVie Declares Quarterly Dividend

On February 18, 2016 The board of directors of AbbVie Inc. (NYSE: ABBV) reported a quarterly cash dividend of $0.57 per share (Press release, AbbVie, FEB 18, 2016, View Source;p=RssLanding&cat=news&id=2140604 [SID:1234509123]).

The cash dividend is payable May 16, 2016 to stockholders of record at the close of business on April 15, 2016.

Since the company’s inception in 2013, AbbVie has increased its dividend by more than 42 percent. AbbVie is a member of the S&P Dividend Aristocrats Index, which tracks companies that have annually increased their dividend for at least 25 consecutive years.