On March 26, 2018 Bayer reported that its collaboration partner Loxo Oncology, Inc., (NASDAQ: LOXO) has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for larotrectinib for the treatment of adult and pediatric patients with locally advanced or metastatic solid tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion (Press release, Bayer, MAR 26, 2018, View Source [SID1234525481]). The rolling submission was initiated in December 2017. NTRK gene fusions are genetic alterations present across a wide range of tumors resulting in uncontrolled tropomyosin receptor kinase (TRK) signaling and tumor growth.

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"This NDA submission in the U.S. marks an important milestone in bringing us one step closer to providing larotrectinib as a potential treatment option for patients with TRK fusion cancer," said Scott Fields, MD, Bayer’s senior vice president and head of Oncology Development at Bayer’s Pharmaceutical Division. "NTRK gene fusions, while rare, are present in various pediatric and adult cancers. We are committed to working with the FDA and the oncology community to bring larotrectinib to patients as soon as possible."

Bayer and Loxo Oncology are jointly developing larotrectinib, an investigational compound being studied globally for the treatment of patients across a wide range of cancers that harbor a NTRK gene fusion. Bayer plans to submit a Marketing Authorization Application (MAA) in the European Union in 2018.

About Larotrectinib (LOXO-101)
Larotrectinib (LOXO-101) is an investigational oral and selective drug in clinical development for the treatment of patients across a wide range of cancers that harbor a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. Growing research suggests that the NTRK genes, which encode for tropomyosin receptor kinases (TRKs), can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body.

Larotrectinib has been granted Breakthrough Therapy Designation, Rare Pediatric Disease Designation and Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration. For additional information about the larotrectinib clinical trials, please refer to www.clinicaltrials.gov or visit www.loxooncologytrials.com. Larotrectinib has not been approved by the U.S. Food and Drug Administration, the European Medicines Agency or any other health authority.

In November 2017, Bayer and Loxo Oncology entered into an exclusive global collaboration for the development and commercialization of larotrectinib and LOXO-195, a next-generation TRK inhibitor in clinical development. Bayer and Loxo Oncology will jointly develop the two products with Loxo Oncology leading the ongoing clinical studies as well as the filing in the U.S., and Bayer leading ex-U.S. regulatory activities and worldwide commercial activities. In the U.S., Bayer and Loxo Oncology will co-promote the products.

About Oncology at Bayer
Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments. The oncology franchise at Bayer now includes four oncology products and several other compounds in various stages of clinical development. Together, these products reflect the company’s approach to research, which prioritizes targets and pathways with the potential to impact the way that cancer is treated.

RXi Pharmaceuticals has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, RXi Pharmaceuticals, 2018, MAR 26, 2018, View Source [SID1234525021]).

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On March 26, 2018 Cancer Genetics, Inc. (Nasdaq:CGIX), a leader in enabling precision medicine for oncology through molecular markers and diagnostics, reported that it will release its financial results for the fourth quarter and full year ended December 31, 2017 on Monday, April 2, 2018 (Press release, Cancer Genetics, MAR 26, 2018, View Source [SID1234524985]). The Company will hold a conference call at 4:30 PM Eastern on Monday, April 2, 2018 to discuss the financial results and provide an update on its strategic direction and key organizational improvements being made by the Company.

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CONFERENCE CALL & WEBCAST
Monday, April 2, 2018, 4:30 p.m. Eastern Time
Domestic: 888-394-8218
International: 323-701-0225
Conference ID: 7874980
Webcast: View Source
Replay – Available through April 16, 2018
Domestic: 844-512-2921
International: 412-317-6671
Conference ID: 7874980

On March 26, 2018 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported that new clinical safety data from the expansion phase of a Phase 2 clinical trial at the Bone Marrow Failure Disease Scientific Symposium, held in Rockville, Maryland, on March 22-23, 2018 (Press release, Onconova, MAR 26, 2018, View Source [SID1234524993]).

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Oral rigosertib has been developed as a single agent and in combination with azacitidine. Previous studies have demonstrated that Low-Risk (LR) MDS patients with intermittent oral rigosertib treatment at a dose of 560 mg BID show a transfusion independence rate (TI), as defined by the IWG 2006 criteria, of 44% (Raza, et al, Blood 2017 130:1689). Oral rigosertib in combination with AZA is being studied in patients with Higher-risk (HR) MDS. Initial results of the Phase 2 study with oral rigosertib (840 mg /day 3 out of 4 weeks) in combination with azacitidine in patients with MDS demonstrated an overall response rate of 76%; 62% in patients following hypomethylating agent (HMA) failure; and 85% in HMA naïve patients (Navada et al, EHA (Free EHA Whitepaper), 2017). In both single agent and combination studies, oral rigosertib has been associated with hematuria in a subset of patients which has been shown to be dose and administration scheme dependent (Garcia-Manero G, Blood 2016 128:2011). The results reported here are from a dose exploration study in HR MDS patients with an increased oral rigosertib dose (1120 mg/day 3 out of 4 weeks) and focus on the impact of risk-mitigation strategies in minimizing the incidence of urinary adverse events (UAEs); including hematuria. The mitigation strategies included prescribing the second dose of rigosertib earlier in the day and encouraging bladder emptying at bedtime.

The reported incidence of hematuria of any grade with single agent azacitidine is 6.3%, including 2.3% grade 3 and 4 events (per product insert). In the combination trial of oral rigosertib (total dose of 840 mg/day 3 out of 4 weeks) and azacitidine, the incidence of hematuria was 48%, with grade 3 or grade4 AEs of 12%. In the new study, in 37 patients studied with oral rigosertib (total dose of 1120 mg/day 3 out of 4 weeks) and azacitidine employing prophylactic risk-mitigating strategies to minimize hematuria, a significantly lower incidence of grade 1 & 2 hematuria (11%), and no grade 3 or 4 hematuria have been seen to date.

Guillermo Garcia-Manero, M.D, Professor and Chief of the MDS Section at the MD Anderson Cancer Center, who presented the new results, commented, "Choices are very limited for higher risk MDS, with two HMAs as the only drugs approved by the Health Authorities for these patients. There is an urgent need to develop novel approaches, including combination therapies that can improve the outcomes in patients who require an HMA. The previously presented studies of the combination regimen of oral rigosertib with azacitidine have demonstrated impressive evidence of efficacy in HMA naïve and HMA refractory patients with higher-risk MDS. Since the success of a combination therapy is greatly influenced by the safety and tolerability of the regimen, the new results of improved tolerability are of great importance for the proposed pivotal study of this combination. The ability to ensure longer duration of treatment without interruption or dose reduction due to an acceptable safety profile can ensure optimal benefit for patients. We look forward to participating in the planned Phase 3 study of this novel approach, which combines two agents with distinct mechanisms of action for the potential benefit of frontline MDS patients."

Dose optimization and risk mitigation strategies undertaken specifically to minimize UAEs associated with oral rigosertib in combination with azacitidine have resulted, to date, in a decrease in frequency of hematuria from 48% to 11% and elimination of any serious grade 3 events. Minimization of AEs permits patients to continue on treatment to optimize the potential benefit. Reduction in incidence of hematuria also enables the continued study of oral rigosertib in LR-MDS, based on the promising TI Rate previously reported.

A copy of the poster is available by visiting the Scientific Presentations section of Onconova’s website.

On March 26, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it has filed a U.S. patent application with the United States Patent and Trademark Office (USPTO) to protect its therapy to treat cancerous tumors, including the therapy that will be used in its upcoming clinical trial in locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC) (Press release, PharmaCyte Biotech, MAR 26, 2018, View Source [SID1234525389]). PharmaCyte has also filed a Patent Cooperation Treaty (PCT) application, which includes protection of its technology in about 150 different countries outside the United States. Both the U.S. and PCT applications claim a priority date from a U.S. provisional patent application PharmaCyte filed in March of last year.

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The patent applications specifically include methods of treating many types of solid cancerous tumors, such as those of the pancreas, liver, breast and colon, using the live-cell encapsulation of genetically modified human cells that overexpress a form of the cytochrome P450 enzyme system normally found in the liver. These cells are encapsulated using the Cell-in-a-Box technology. Together with low doses of oxazaphosphorines, such as ifosfamide, the encapsulated cells comprise PharmaCyte’s therapy for cancerous tumors. The patent application also includes using PharmaCyte’s platform technology with cyclophosphamide, another chemotherapy drug that must be activated by the cytochrome P450 enzyme system. PharmaCyte believes these technologies will be beneficial to patients who no longer respond to standard chemotherapies, such as gemcitabine and Abraxane.

These new applications, if granted, will provide protection for PharmaCyte’s technology for 20 years – until March 2038. "By filing these patent applications, we are continuing the process of pursuing patent protection for 20 years to protect our therapy for all forms of solid malignant tumors. This is particularly important to the company as we are taking steps to embark upon a clinical trial in LAPC," said PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner.
PharmaCyte’s pancreatic cancer therapy was designated an orphan drug and listed in the official registry of medicinal products for rare diseases by the U.S. Food and Drug Administration (FDA) on December 17, 2014. Orphan drug exclusivity would provide marketing exclusivity for PharmaCyte’s pancreatic cancer therapy in the U.S. for 7 years after market approval by the FDA. Similarly, PharmaCyte has orphan drug status in the European Union (EU) for its pancreatic cancer therapy, which provides 10 years of marketing exclusivity in all countries in the EU following approval by the European Medicines Agency (EMA).

In addition, the Biologics Price Competition and Innovation Act (BPCIA), which was enacted as part of the Affordable Care Act in 2010, establishes a period of 12 years of "data exclusivity" for reference products to preserve incentives for future innovation. Under this framework, data exclusivity protects the data in the innovator’s regulatory application by prohibiting others, for a period of 12 years, from gaining FDA approval based in part on reliance on or reference to the innovator’s data in a biosimilar application. PharmaCyte’s 12-year exclusivity will begin as soon as the FDA approves the company’s first Cell-in-a-Box-based therapy.

Mr. Waggoner concluded by stating, "While these patent applications should make our investors feel assured about the protection of our pancreatic cancer therapy, they should understand that if our therapy receives FDA approval, the orphan drug designation in the U.S. and the EU, together with the BPCIA data exclusivity that may be awarded, will give us substantial marketing exclusivity for our pancreatic cancer therapy. These patent applications should be viewed as an opportunity to dramatically broaden PharmaCyte’s ability to protect its unique therapy for all malignant solid tumors for the next 20 years