On January 26, 2015 AMATSIGROUP, the leading French supplier of services devoted to the development of both human and veterinary pharmaceutical products, reported the acquisition of Belgian company SEPS Pharma, which provides drug product development services, encompassing preformulation and formulation development, analytical drug product development, process development and clinical trial manufacturing (Press release, Amatsigroup, JAN 26, 2015, View Source [SID1234520755]). Founded in 2007 and located in Ghent, Belgium, SEPS Pharma counts a team of 30 highly-qualified people specialized in the development of innovative oral (liquid and solid dosage forms), inhalable and injectable formulations with acknowledged expertise in enhancing the bioavailability and controlling the drug release. With over 25 clients in Europe, US and Japan, the Belgian company recorded a 2014 revenue of €5 million.

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On January 26, 2015 Provectus Biopharmaceuticals reported that it has received notification of allowance from the Chinese Patent Office for its patent application protecting the synthetic process used to produce the small molecule Rose Bengal, the active pharmaceutical ingredient (API) in PV-10, the Company’s lead oncology drug candidate (Press release Provectus Pharmaceuticals, JAN 26, 2015, http://www.pvct.com/pressrelease.html?article=20150126.1 [SID:1234501388]).

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The pending Chinese patent covers the same process as the one granted by the US Patent Office in September 2013, as U.S. Patent 8,530,675, "Process for the Synthesis of 4,5,6,7-tetrachloro-3′,6′-dihydroxy-2′,4′,5′,7′-tetraiodo-3H-spiro[isobenzofuran-1,9-xanthen]-3-one (Rose Bengal) and Related Xanthenes." The application details a new process for the manufacture of Rose Bengal and related iodinated xanthenes in high purity. The allowed claims cover the process under which pharmaceutical grade Rose Bengal and related xanthenes are produced, reducing the formation of certain previously unknown transhalogenated impurities that currently exist in commercial grade Rose Bengal in uncontrolled amounts. The requirement to identify and control related substances is in accordance with International Conference on Harmonisation (ICH) guidelines for manufacture of API suitable for phase 3 clinical trial material and commercial pharmaceutical use. Once issued later this year, the patent is expected to provide protection for Rose Bengal API to 2031 and covers any hypothetical process that controls the amount of transhalogenated impurities in Rose Bengal through the awarded Jepson style claims.

Eric Wachter, CTO of Provectus, stated, "The issuance of this patent will enhance the protection of our novel synthesis process for the manufacture of Rose Bengal covering the entire Chinese market. As we prepare to begin our phase 3 clinical trial for intralesional PV-10 as a treatment for melanoma and as we discuss with Chinese interests licensing PV-10 for other indications, it is important that we defend our intellectual property in this way. We are pleased that the Chinese authorities have been so helpful in this, and we will continue to protect our stockholders’ interests in this way as we seek out partners globally to further develop our product line."

On January 23, 2015 The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for Jakavi (ruxolitinib) for the treatment of adult patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea (Press release Novartis, JAN 23, 2015, View Source [SID:1234501375]). If approved in the EU, ruxolitinib could provide the first targeted treatment option for these patients.

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PV is a chronic, incurable blood cancer associated with an overproduction of blood cells that can cause serious cardiovascular complications, such as stroke and heart attack[1]. In PV, patients with resistance to or intolerance of hydroxyurea are considered to have uncontrolled disease, which is typically defined as hematocrit levels greater than 45%, elevated white blood cell count and/or platelet count, and may be accompanied by debilitating symptoms and/or enlarged spleen[1],[3],[4]. Elevated white blood cell count and hematocrit are also associated with an increased risk of blood clots[5].

"This positive CHMP opinion is encouraging news for patients with polycythemia vera who need effective treatment options," said Alessandro Riva, MD, Global Head, Novartis Oncology Development and Medical Affairs. "If approved, ruxolitinib will be the first-ever targeted therapy for polycythemia vera in the EU, a positive step forward for the rare blood cancer community in Europe and a major development in Novartis’ continued commitment to help patients with high unmet needs."

In the EU, the European Commission generally follows the recommendations of the CHMP and delivers its final decision within three months of the CHMP recommendation. The decision will be applicable to all 28 EU member states plus Iceland, Norway and Liechtenstein. Global regulatory applications for ruxolitinib in PV are currently ongoing, and further regulatory filings are under review by health authorities. Ruxolitinib, which is marketed in the US by Incyte Corporation as Jakafi, received approval in December 2014 from the US Food and Drug Administration (FDA) for the treatment of patients with PV who have had an inadequate response to or are intolerant of hydroxyurea.

The CHMP recommendation was based on results from the pivotal Phase III RESPONSE clinical trial demonstrating that a significantly greater proportion of patients achieved the composite primary endpoint of hematocrit control (volume percentage of red blood cells in whole blood) without use of phlebotomy (a procedure to remove blood from the body to reduce the concentration of red blood cells) and spleen size reduction when treated with ruxolitinib compared to best available therapy (21% compared to 1%, respectively; p<0.0001)[1],[2]. In addition, a greater proportion of patients in the ruxolitinib treatment arm achieved complete hematologic remission, as defined by the modified 2009 European LeukemiaNet (ELN) criteria, when compared to the standard therapy arm (24% compared to 9%, respectively; p=0.003)[2]. The data also showed more patients treated with ruxolitinib had a durable primary response at week 48 compared to patients treated with standard therapy (19% compared to 1%, respectively; (p<0.0001)[2].

Overall, ruxolitinib was well tolerated, and non-hematologic adverse events (AEs) were consistent with those previously seen in ruxolitinib studies in PV and myelofibrosis[2],[6],[7]. Within the first 32 weeks of treatment, the most common Grade 3 or 4 hematologic AEs in the ruxolitinib treatment arm were anemia (1.8%) and thrombocytopenia (5.5%)[2]. The most common non-hematologic AEs were dizziness (15.5%), constipation (8.2%) and herpes zoster (6.4%)[2]. The three most frequent non-hematological laboratory abnormalities (any Grade) were hypercholesterolemia (30.0%), raised alanine aminotransferase (22.7%) and raised aspartate aminotransferase (20.9%), which were mainly Grade 1 and 2[2].

On January 23, 2015 Celgene reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for ABRAXANE (paclitaxel formulated as albumin-bound nanoparticles, or nab-paclitaxel) in combination with carboplatin for the first-line treatment of non-small cell lung cancer in adult patients who are not candidates for potentially curative surgery and/or radiation therapy (Press release Celgene, JAN 23, 2015, View Source [SID:1234501378]).

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Lung cancer is the fourth most commonly diagnosed cancer in both men and women, however it is the leading cause of cancer-related mortality in Europe. Non-small cell lung cancer (NSCLC) is the most common form of lung cancer, accounting for 85 to 90% of all cases. The predominant cause of lung cancer is cigarette smoking, although environmental and occupational factors also can cause the cancer. Treatment options generally include systemic chemotherapy or protein kinase inhibitors. In the most advanced cases, only the symptoms of the disease can be managed; there is a clear need for innovative new medicines for the treatment of lung cancer.

"Progress in lung cancer will come first from early diagnosis with patients presenting promptly with symptoms and second, with new drugs that are well tolerated and improve on current therapies. Incremental steps can lead to a meaningful impact on patients and society, given the frequency and aggressiveness of lung cancer," says Dr. Mary O’Brien, Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust, UK. "The positive CHMP opinion for ABRAXANE in combination with carboplatin for the treatment of adult patients with NSCLC is a significant step toward bringing a new treatment option to patients in Europe. The clinical data show patients had a significant positive response rate to the treatment, combined with an established safety profile. The therapy has also shown a significant response benefit for a subset of patients with squamous cell lung cancer, where there have been limited treatment advances in recent years."

The positive CHMP opinion was based on the results of a multicenter, randomized, open-label study including 1,052 chemotherapy-naive patients with Stage IIIb/IV non-small cell lung cancer. The study compared ABRAXANE in combination with carboplatin versus solvent-based paclitaxel in combination with carboplatin as first-line treatment in patients with advanced non-small cell lung cancer. The primary efficacy endpoint, overall response rate, was significantly higher for patients in the ABRAXANE/carboplatin arm at 33%, compared with patients in the control arm, at 25%. The most common adverse reactions

(≥ 20%) of ABRAXANE in combination with carboplatin for NSCLC were anaemia, neutropenia, thrombocytopenia, peripheral neuropathy, nausea, and fatigue.

Tuomo Pätsi, President of Celgene in Europe, the Middle East and Africa (EMEA), said, "The positive CHMP opinion is the first opportunity for Celgene to play a role in helping patients with NSCLC have access to an important treatment option in Europe. The anticipated European Commission decision would be the third approved indication for ABRAXANE, underscoring the value of this medicine. We are committed to ensuring that patients who need ABRAXANE will gain access to it once approved by the European Commission."

The CHMP reviews applications for all 28 member states in the European Union (EU), as well as Norway, Liechtenstein and Iceland. The European Commission, which generally follows the recommendation of the CHMP, is expected to make its final decision within approximately two months. If approval is granted, detailed conditions for the use of this product will be described in the Summary of Product Characteristics (SmPC), which will be published in the revised European Public Assessment Report (EPAR).

ABRAXANE is not currently indicated for the treatment of metastatic NSCLC in the European Union.

On January 23, 2015 Progenics Pharmaceuticals reported that it has dosed the first subject in the resumed pivotal Phase 2 clinical study of Azedra in patients with malignant pheochromocytoma and paraganglioma (Press release Progenics Pharmaceuticals, JAN 23, 2015, View Source [SID:1234501379]). The trial is being conducted under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA). Progenics acquired Azedra, a novel targeted cancer radiotherapy, in conjunction with its 2013 acquisition of Molecular Insight Pharmaceuticals (MIP).

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"Relaunching this study is a very important milestone for patients with metastatic and/or recurrent pheochromocytoma/paraganglioma," said Dr. Daniel Pryma, Associate Professor of Radiology and Clinical Director of Nuclear Medicine and Molecular Imaging at the University of Pennsylvania, a current investigator and lead investigator on the original trial. "This treatment has been generally well tolerated and the data emerging from the trial to date has been encouraging. I am pleased to be resuming this study and feel that Azedra could represent the best treatment in malignant pheochromocytoma/paraganglioma in the future."

The study is designed to evaluate the efficacy and safety of the administration of two therapeutic doses of Azedra in patients with malignant relapsed/refractory pheochromocytoma or paraganglioma, ultra-orphan cancers with limited treatment options. The primary objective of the study is to determine the clinical benefit of Azedra based on the proportion of study participants with a reduction of all antihypertensive medication by at least 50% for at least six months. The SPA requires that 25% of 58 evaluable patients achieve the primary endpoint.

In late 2010, MIP suspended enrollment in the trial to seek additional funding. The trial has now resumed to fulfill enrollment requirements under the SPA. The trial has treated 41 patients and 32% of those patients have achieved the primary endpoint. The most common adverse events observed have been gastroenterological and hematologic disorders.

"As a targeted cancer treatment, Azedra has the potential to improve outcomes in patients suffering from pheochromocytoma and paraganglioma," stated Mark Baker, CEO of Progenics. "We are focused on successfully completing this pivotal trial and look forward to completing patient enrollment by the end of 2015, building on the promising data seen to date, and advancing this promising candidate toward the marketplace."

Azedra has received Orphan Drug and Fast Track designations from the FDA.