PhaseRx Receives Orphan Drug Designation from FDA for PRX-OTC for the Treatment of Ornithine Transcarbamylase Deficiency

SEATTLE, Nov. 28, 2016  -- PhaseRx, Inc. (PZRX), a biopharmaceutical company developing mRNA treatments for life-threatening inherited liver diseases in children, today announced that its lead candidate, PRX-OTC, which is being developed for the treatment of ornithine transcarbamylase deficiency (OTCD), has received orphan drug designation by the U.S. Food and Drug Administration (FDA).
 
OTCD is a rare liver disorder caused by an inherited single-gene deficiency that results in hyperammonemia (elevated ammonia in the blood), and can lead to irreversible neurological impairment, coma and death. PRX-OTC is an intracellular enzyme replacement therapy (i-ERT) designed to replace the missing or defective enzyme in patients with OTCD, thereby correcting the disease. PRX-OTC has shown therapeutic potential in a preclinical model of OTCD, including lowering of blood ammonia and survival of 100% of treated mice.
Read more: PhaseRx Inc ( PZRX )

Anavex Life Sciences Announces Data on 41-Week Treatment of ANAVEX 2-73 for Patients with Alzheimer’s Disease − Investigational Treatment suggests to curb Cognitive and Functional Decline

NEW YORK, Nov. 22, 2016  -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (AVXL) today announced a positive 41-week update from its Phase 2a study in mild-to-moderate Alzheimer’s disease (AD) patients for ANAVEX 2-73, which targets cellular homeostasis.
 
At 41 weeks, Alzheimer’s patients taking a daily oral dose of ANAVEX 2-73 in the exploratory, not yet dose optimized Phase 2a clinical trial, showed a stabilization of cognitive and functional measures. This data of stabilization is promising since Alzheimer’s disease is a progressive disease where current therapeutics are only able to temporarily slow the worsening of dementia symptoms and not stop the disease from progressing.
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FDA Confirms a 510(k) De Novo Path for EyeGate’s Device, the Ocular Bandage Gel, Following Pre-Submission Meeting

WALTHAM, Mass., Nov. 17, 2016 -- EyeGate Pharmaceuticals, Inc. (EYEG) (“EyeGate” or the “Company”), a clinical-stage specialty pharmaceutical company that focuses on developing and commercializing products for treating diseases and disorders of the eye, today announced that, following a pre-submission meeting with the U.S. Food & Drug Administration (“FDA”), it plans to pursue U.S. regulatory clearance of its EyeGate Ocular Bandage Gel (“EyeGate OBG”), via the De Novo 510(k) pathway. EyeGate OBG is the lead product candidate from the Company’s cross-linked, thiolated carboxymethyl hyaluronic acid (CMHA-S) platform. The Company plans to release top-line results by year-end, from its initial pilot study evaluating the ability of EyeGate OBG to accelerate ocular surface re-epithelialization following photorefractive keratectomy (“PRK”).
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ProMetic's PBI-4050 Demonstrates Early Evidence of Efficacy in its Idiopathic Pulmonary Fibrosis ("IPF") Phase 2 Clinical Trial

Early evidence of efficacy of PBI-4050 alone and also in combination with one of the commercially available IPF drugs
PBI-4050 shown to be very well tolerated whether used alone or in combination with nintedanib or pirfenidone
 
LAVAL, QC, Nov. 17, 2016 - ProMetic Life Sciences Inc. (TSX:PLI.TO - News) (OTCQX:PFSCF - News) ("ProMetic" or the "Corporation") announced today positive interim results from its Open Label Phase 2 clinical trial in patients suffering from idiopathic pulmonary fibrosis ("IPF"). In addition to demonstrating that PBI-4050 is safe and well tolerated in patients suffering from IPF, the objective of this study was to provide early evidence of clinical benefits of PBI-4050 treatment whether used alone or in addition to either nintedanib or pirfenidone. Forty patients are enrolled in the study in 6 sites across Canada. At this time, the Corporation is reporting on the first 30 patients that have completed their 12 weeks of treatment.
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Cellectar Biosciences Announces Successful Conjugation of Multiple Pierre Fabre Cytotoxic Compounds to PDC Delivery Platform; Initiates In Vivo Studies for Solid Tumors

MADISON, Wis., Nov. 17, 2016  -- Cellectar Biosciences, Inc. (CLRB) (the “company”), an oncology-focused, clinical stage biotechnology company, today announces that following the successful conjugation of multiple, natural product cytotoxic molecules developed by Pierre Fabre to Cellectar’s PDC delivery platform, it has initiated in vivo studies for a variety of solid tumors.
 
Harnessing a selection of linkers to attach the cytotoxic molecules to the PDC platform, the company has constructed a series of novel compounds specifically designed for improved tumor targeting.  This research collaboration falls under the company’s CLR CTX programs, geared to convert non-targeted cytotoxic agents into targeted cancer treatments when combined with Cellectar’s proprietary delivery system.  The drug’s targeting enhancement is designed to further improve efficacy and reduce adverse events.
Read more: Cellectar Biosciences ( CLRB )