On January 16, 2019 Nexus Pharmaceuticals Inc. reported the U.S. Food and Drug Administration (FDA) approval of Busulfan Injection, the company’s AP-rated therapeutic equivalent for Busulfex 60 mg/10 mL (6 mg/1 mL) (Press release, Nexus Pharma, JAN 16, 2019, View Source [SID1234532683]).

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"The FDA approval of Busulfan continues to expand our portfolio of difficult to manufacture injectables, in addition to becoming our second oncology generic drug," said Omair Ahmed, Chief Commercial Officer, Nexus Pharmaceuticals. "Busulfan is currently on American Society of Health System Pharmacists drug shortage list. The addition of Busulfan will continue to show our commitment to providing patients and clinicians with access to affordable generic alternatives to critical need medicines that have a history of shortage."

Busulfan is expected to launch in the United States shortly in cartons of eight single-dose vials, each containing 60 mg of busulfan in 10 mL of clear sterile solution. It is Latex and Preservative free.

About Busulfan Injection

Busulfan is indicated for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia.

On January 16, 2019 Phoenix Molecular Designs (PhoenixMD), a privately-held biotechnology company designing precise cancer therapeutics by targeting essential kinases, reported that it has entered into a collaboration with Roche to develop a diagnostic (CDx) in triple-negative breast cancer (TNBC) (Press release, PhoenixMD, JAN 16, 2019, View Source [SID1234553816]). The Roche CDx identifies RSK2 activation in human tumors. In cancer, the PDK-1 and MAPK pathways converge on RSK2 to activate it, moving it from the cytoplasm into the nucleus. Measuring nuclear RSK2 signifies activation and abundance of this emerging drug target.

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Complementing its diagnostic efforts, PhoenixMD has also developed PMD-026, which is the first orally available small molecule inhibitor that targets RSK2, a prime drug target in multiple cancers. The leading focus for PhoenixMD is in the treatment of triple-negative breast cancer (TNBC) given the companies’ core expertise in developing breast cancer therapeutics.

The diagnostic assay developed through the Roche/PhoenixMD collaboration will relay information on how active the RSK2 pathway is in TNBC and other cancers. Preliminary data indicates that 80 percent of cases (52/65 TNBC cases) express activated RSK2. More broadly, researchers have investigated more than 300 biopsies and found that RSK2 is activated in 65 percent of tumors from a study of 13 different tumor types. RSK2 was also detected in breast cancer metastases using this method. Over the coming months, Roche will establish a CAP/CLIA certified protocol as a gateway into clinical tumor analyses. In upcoming clinical trials, PhoenixMD will further refine the precision of the RSK2 CDx in identifying patients that may ultimately benefit from PMD-026. In the near term, PhoenixMD expects to file an IND for PMD-026 and initiate a Phase I/Ib study in women with TNBC.

"By working together, PhoenixMD and Roche are at the forefront of innovation in TNBC, the most deadly breast cancer type with no approved therapies. Creating our diagnostic assay and identifying disease biomarkers, such as RSK2 for TNBC, will dramatically reduce the development time needed to create targeted drugs and will improve a drug’s chance of advancing through clinical trials," said Dr. Sandra E. Dunn, CEO of PhoenixMD. "The top-line data generated from our CDx is encouraging, and we look forward to applying these learnings to identify TNBC patients that may benefit from PMD-026 in our upcoming Phase I/Ib study."

About Triple Negative Breast Cancer (TNBC) and RSK Kinases

Approximately 400,000 cases of TNBC are diagnosed every year worldwide and it is one of the most difficult breast cancer subtypes to treat due to lack of effective, targeted therapies. TNBC also claims the lives of young women more than any other type of breast cancer due to a lack of understanding around the therapeutic bullseye. It is also a very heterogeneous disease, therefore a common denominator across TNBC types was necessary to identify the bullseye. Through genome-wide screens, RSK was identified as the prime target for TNBC by scientists at PhoenixMD. Currently, there are no approved targeted therapies available for TNBC.

There are four types of RSK involved in cancer, known as RSK1-4, and each type has a unique role in the development of the disease. RSK1 is responsible for cancer cell invasion and is an important driver in the spread of cancer. RSK2 controls cancer cell growth, and RSK3 and RSK4 are associated with drug resistance.

RSK1 and RSK2 have been proven critical to the survival of patients with TNBC. Over 90 percent of primary TNBC cases express high levels of RSK1 and RSK2. Inhibiting RSK2 eliminates TNBC cells completely, including cancer stem cells, which give rise to cancer recurrence. PhoenixMD, with its novel, targeted approach, is focused on creating patented cancer RSK inhibitors and companion diagnostics for cancer indications – initially in breast cancer – with the potential to treat blood, brain, ovarian, lung, skin, prostate, colon, head and neck cancers.

While there are currently no approved targeted therapies for TNBC, several drugs are involved in research studies and clinical trials. PhoenixMD is addressing this unmet medical need through a novel, targeted approach by inhibiting critical kinases, such as RSK1-4, a group of highly conserved Ser/Thr kinases that promote cell proliferation, growth, motility and survival. For this target, PhoenixMD developed PMD-026, a first-in-class, specific RSK inhibitor that blocks downstream signaling of RSK and induces apoptosis.

On January 16, 2019 QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) reported that the Japanese Pharmaceuticals and Medical Device Agency (PMDA) has approved the therascreen EGFR RGQ PCR Kit to allow its use as a companion diagnostic with Pfizer’s VIZIMPRO (dacomitinib) for EGFR gene mutation-positive, inoperable or recurrent non-small cell lung cancer (Press release, Qiagen, JAN 16, 2019, View Source [SID1234532684]). The therascreen EGFR RGQ PCR Kit is registered in more than 40 countries globally. This marks the first companion diagnostic approval for QIAGEN in Japan.

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"As precision medicine becomes the standard of care in oncology, we are pleased to provide benefits to more lung cancer patients with our clinically proven therascreen EGFR RGQ PCR Kit. Our collaboration with Pfizer has made great strides already and will continue to improve personalized healthcare for patients around the world," said Jonathan Arnold, Vice President, Head of Oncology and Precision Diagnostics for QIAGEN. "In addition to detecting a comprehensive panel of EGFR mutations, the therascreen EGFR kit offers laboratories an efficient workflow on the Rotor-Gene Q MDx, the real-time PCR module in our widely-used QIAsymphony family of instruments."

QIAGEN is a pioneer in Personalized Healthcare and the global leader in collaborations with pharmaceutical and biotechnology companies to co-develop companion diagnostics, which detect genetic abnormalities to provide insights that guide clinical decision-making on the use of drugs in diseases such as cancer. QIAGEN has an unmatched depth and breadth of technologies from NGS to PCR for companion diagnostic development and has been the market leader in companion diagnostics working under master collaboration agreements with more than 25 pharmaceutical companies developing companion diagnostic tests for their drug candidates. For more details surrounding QIAGEN’s companion diagnostics and their claims please visit www.qiagen.com. For additional information on Pfizer’s VIZIMPRO (dacomitinib) please visit www.pfizer.com.

On January 15, 2019 Kitov Pharma (NASDAQ/TASE: KTOV), an innovative pharmaceutical company, reported new findings from its ongoing collaboration with researchers from the Hebrew University of Jerusalem (Press release, Kitov Pharmaceuticals , JAN 15, 2019, View Source [SID1234532669]). The data reveal NT219’s high affinity and selective binding to its target proteins.

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Hadas Reuveni, Ph.D., Chief Technology Officer at Kitov’s subsidiary, TyrNovo Ltd., in collaboration with Dr. Galia Blum and Dr. Ofra Moshel from the Hebrew University demonstrated that NT219 binds directly to Insulin Receptor Substrates (IRS) 1/2 and to the Signal Transducer and Activator of Transcription 3 (STAT3), both known modulators of tumor survival, metastasis and drug resistance. Data showed that a short exposure of cancerous cells to NT219 was sufficient to trigger irreversible shutdown of these pathways, resulting in a long-term anti-cancer effect.

Based on these latest findings, Kitov and Yissum, the Technology Transfer company of the Hebrew University of Jerusalem, have extended their collaboration agreement in order to deepen the understanding of NT219’s efficacy in overcoming tumors’ resistance to immunotherapy.

As previously reported (Reuveni et al, Cancer Research, 2013) upon binding to IRS1/2 a three-step mechanism is activated. IRS1/2 dissociates from the cell membrane, undergoes serine phosphorylation which prevents rebinding to the receptor, and is finally degraded by the proteasome. This sequence of events leads to the blockage of AKT – a major cancer cell survival pathway.

"We are excited about the new data which demonstrate NT219’s unique mechanism of action. NT219, a new and promising concept in cancer therapy, is designed to prevent, reverse, and delay resistance to anti-cancer drugs. We are developing NT219 as a drug to be used in combination with other therapies that has a potential to overcome cancer drug resistance and to boost the efficacy of numerous oncology drugs on the market today," stated Kitov CEO, Isaac Israel.

About NT219

NT219 is a small molecule that presents a new concept in cancer therapy by promoting the degradation and inhibiting the phosphorylation of two oncology-related signal transducers, Insulin Receptor Substrates (IRS) 1/2 and signal transducer and activator of transcription 3 (STAT3), respectively. While targeted anti-cancer drugs inhibit the "ON" signal, NT219 activates the "OFF" switch, extensively blocking major oncogenic pathways. In pre-clinical trials, NT219, in combination with several approved cancer drugs, displayed potent anti-tumor effects and increased survival in various cancers, including sarcoma, melanoma, pancreatic, lung, head & neck, prostate and colon cancers, by preventing the tumors from developing drug resistance and reversing resistance after it had been acquired. NT219 is licensed from Yissum, the technology transfer company of the Hebrew University of Jerusalem

On January 15, 2019 Oasmia Pharmaceutical AB (NASDAQ: OASM) reported that the European Medicines Agency (EMA) has validated a type II variation application to add efficacy data to the Apealea product information (Press release, Oasmia, JAN 15, 2019, View Source [SID1234556566]). Validation of the application confirms that the submission is complete and that the EMA assessment process begins. Based on the EMAs procedural timelines, Oasmia anticipates an opinion from the Committee for Medicinal Products for Human Use (CHMP) by end of Q1 or beginning of Q2 2019.

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The application is based on subpopulation data (n=599) from the OAS-07OVA study and aims to provide treating physicians with efficacy data for Apealea in combination with carboplatin for the approved indication; adult patients with first relapse of platinum-sensitive epithelial ovarian cancer, primary peritoneal cancer and fallopian tube cancer. The current efficacy data describes the population in OAS-07OVA that also included patients with several disease relapses (n=789).

About the subpopulation analysis submitted as a type II variation:

The marketing authorisation approval for Apealea in the EEA was based on study OAS 07OVA conducted on 789 randomized patients with disease relapse. The results of the subpopulation analysis of patients experiencing their first relapse were based on 301 patients (Apealea arm) and 298 patients (Comparator arm) being part of the OAS-07OVA study. The results show that there is a statistically significant advantage for Apealea with regards to progression-free survival.

Key efficacy results in patients with their first relapse in the intention-to-treat population from the pivotal randomized clinical trial OAS-07OVA

Progression-free survival (PFS)
(N=301) Overall survival (OS)
(N=298)
Hazard ratio, HR1
(95% CI) 0.80
(0.66-0.97) 0.98
(0.79-1.21)

Apealea2 CrEL-paclitaxel2 Apealea2 CrEL-paclitaxel2
Median, months
(95% CI) 10.3
(10.1-11.1) 10.0
(9.6-10.2) 24.7
(21.9-28.0) 23.4
(20.5-26.7)
1A longer PFS or OS for Apealea compared to Cremophor-EL (CrEL)-formulated paclitaxel is indicated by a HR less than 1.0.
2In combination with carboplatin.

About epithelial ovarian cancer

Ovarian cancer is the seventh most common cancer in women. Approximately 239,000 women are annually diagnosed with ovarian cancer globally and 152,000 dies from the disease. Epithelial ovarian cancer is the most common form and accounts for about 90% of ovarian cancers. The disease is often diagnosed at an advanced staged since it has no symptoms at early stages. The five-year survival rate (i.e. survival of patients with ovarian cancer compared to survival in the general population at the same age) for ovarian cancer has been estimated to 38% in Europe. During 2018, approximately 68,000 women will be diagnosed with ovarian cancer in Europe and 45,000 are predicted to die from the disease. Carboplatin and paclitaxel are common chemotherapy drugs for treatment of ovarian cancer and are often used in combination.

About Apealea

Apealea is a Cremophor- and albumin-free formulation of the well-known cytostatic paclitaxel combined with Oasmia’s excipient technology XR17. Paclitaxel is one of the most widely used anticancer substances and is included in the standard treatment of a variety of cancers such as lung cancer, breast cancer and ovarian cancer. Apealea consists of a freeze-dried powder, which is dissolved in conventional solutions for infusion.