Industry News

Addex Therapeutics, a leading company pioneering allosteric modulation-based drug discovery and development, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to dipraglurant for the treatment of levodopa-induced dyskinesia associated with Parkinson’s disease (PD-LID). Orphan drug status provides Addex with a number of benefits including reduced development costs and seven years US market exclusivity from launch. Dipraglurant is a novel small molecule inhibitor of the metabotropic glutamate receptor 5 (mGluR5) that has successfully completed Phase II proof-of-concept testing in Parkinson's disease (PD) patients suffering from debilitating levodopa-induced dyskinesia (LID), an indication that has no approved treatment options. Addex is currently conducting an mGlu5 receptor occupancy clinical trial and is preparing to start a Phase III pivotal trial in PD-LID.

NANOBIOTIX, a late clinical-stage nanomedicine company pioneering novel approaches for the local treatment of cancer, announces the US Food and Drug Administration (FDA) has approved the Company’s Investigational New Drug (IND) application on the 30th of December. This allows Nanobiotix to launch its first clinical study in the US for its lead product NBTXR3 in prostate cancer, a new and very significant indication.

Merck KGaA, Darmstadt, Germany, Pfizer and Syndax Pharmaceuticals, Inc. announced that they have entered into a collaboration agreement to evaluate avelumab*, an investigational fully human anti-PD-L1 IgG1 monoclonal antibody, in combination with Syndax’s entinostat, an investigational oral small molecule that targets immune regulatory cells (myeloid-derived suppressor cells and regulatory T-cells), in patients with heavily pre-treated, recurrent ovarian cancer. Avelumab is currently under clinical investigation across a broad range of tumor types by the alliance between Merck KGaA, Darmstadt, Germany, and Pfizer. This is an exclusive agreement between the alliance and Syndax to study the combination of these two investigational agents in ovarian cancer. Syndax will be responsible for conducting the Phase Ib/II clinical trial in ovarian cancer.

NeuroDerm Ltd., a clinical stage pharmaceutical company developing drugs for CNS disorders, announced the start of patient enrollment in a first efficacy trial of ND0612H, the company’s continuously administered subcutaneous levodopa/carbidopa solution. This treatment is intended to be an alternative to current treatments for patients with advanced Parkinson’s disease that require surgical intervention.

Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet needs, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for GBT440 for the treatment of patients with sickle cell disease (SCD). GBT440 is being developed as a potentially disease-modifying therapy for SCD.

Minerva Neurosciences, Inc., a clinical-stage biopharmaceutical company focused on the development of therapies to treat CNS disorders, announced that the US Food and Drug Administration (FDA) has accepted the company's Investigational New Drug (IND) application for MIN-101, a first-in-class 5-HT2a and sigma2 antagonist in clinical development for the treatment of schizophrenia.

Pluristem Therapeutics Inc.,  a leading developer of placenta-based cell therapy products, announced that the U.S. Food and Drug Administration has granted the Company’s PLX-PAD cells Orphan Drug Designation in the treatment of severe preeclampsia. Preeclampsia is among the most common medical complications of pregnancy and a leading cause of premature births, stillbirths and neonatal and maternal deaths. There is no cure except delivery. Due to high risks to the mother, women diagnosed with severe preeclampsia are usually delivered promptly, even if the baby will be born prematurely and may suffer permanent disabilities as a result. Severe preeclampsia occurs in approximately 1% of pregnancies in Western countries.

Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, “Eisai”) announced today that regarding the acquisition of Chinese generic pharmaceutical company Liaoning TianYi Biological Pharmaceutical Co., Ltd. (Location: Benxi, Liaoning Province, China) previously announced on November 27, 2015, the conditions of transfer have been arranged, including receiving a new business license from the regulatory authority in China, and the acquisition has been completed.

BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has notified the Company that they had not yet completed their review process and would be unable to take an action by the Prescription Drug User Fee Act (PDUFA) action date for KyndrisaTM (drisapersen) of December 27, 2015, and anticipate taking action in early January 2016.

Faron Pharmaceuticals Ltd (Faron), the clinical stage biopharmaceutical company, has announced the enrollment of the first patient in the phase III INTEREST clinical programme for Traumakine for the treatment of acute respiratory distress syndrome (ARDS). Faron is a drug discovery and development company focused on creating novel treatments for medical conditions with significant unmet needs.