Stimulated Development of Orphan Drug Designations For Rare Diseases and Anti-Cancer Drug Candidates Intensifies

Palm Beach, FL – (December 6, 2018) – The FDA is committed to doing what is can to stimulate the development of more products by improving the consistency and efficiency fueling biotech and pharma companies in the pharmaceutical industry forge ahead seeking positive results of clinical trials towards new treatments for various rare diseases, including numerous forms of cancer. Treatments for rare diseases are often given a designation by the FDA known as “orphan”. According to the Quintiles IMS Institute, there are over 7,000 rare diseases that affect 25 to 30 million adults, but there are only treatments for roughly 5% of those diseases. This wide gap between rare diseases and available treatments presents an enormous opportunity and has leaders in big pharma hustling to develop innovative solutions to assist those in the greatest need. From 2008 to 2017, 53% of orphan drug marketing approvals were in one of two therapeutic areas that were also common for granted designations: oncology (42.5%) and hematology (10.8%), according to the report. There were 27 different therapeutic areas overall, with seven of those areas having 10 or more approved orphan drugs. Active biotech and pharma companies in the markets this week include Moleculin Biotech, Inc. (NASDAQ:MBRX), Oragenics Inc. (NYSE:OGEN), Clovis Oncology Inc. (NASDAQ:CLVS), Novelion Therapeutics Inc. (NASDAQ:NVLN), AstraZenaca PLC (NYSE:AZN).


Moleculin Biotech, Inc., (NASDAQ:MBRX) BREAKING NEWS:  Moleculin Biotech, a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, today announced it has filed a request with the U.S. Food and Drug Administration (“FDA”) for Orphan Drug Status for its drug candidate WP1066.


“Clinical progress with WP1066 has been encouraging,” commented Walter Klemp, Moleculin’s Chairman and CEO.  “Given its potential to address rare and difficult to treat cancers, including glioblastoma, we believe WP1066 is well positioned to qualify for Orphan Drug Status.”


The FDA grants orphan drug designation to drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.    Read this and more news for MBRX at   


Other recent developments in the biotech industry include:


Oragenics Inc. (NYSE American:OGEN) came to a close up 6.25% on Tuesday with more than 4.8 million shares traded. The company recently announced the publication of a research paper titled “Blueprints for the rational design of therapeutic mutacin 1140 variants,” in Chemical Biology & Drug Design (CBDD). The study, which can be accessed via focused on structure-activity relationships of the lantibiotic Mutacin 1140 (MU1140) that expand the “drugability” of the MU1140 pipeline. Lantibiotics are a novel class of peptide antibacterial compounds naturally produced by a variety of Gram-positive bacterial strains to attack competing bacteria. The outcomes from the study enabled investigators, Kers et. al., to conceptualize variants of MU1140 that eliminated the commonly found limitations in drug manufacturing that has made the development of lantibiotics challenging, while also improving efficacy, solubility, stability and longer half-life, as demonstrated in biological systems. This is the first study that proposes that a therapeutic variant of MU1140 would require multiple substitutions in order to present all of the desired properties necessary to justify further development.


Clovis Oncology Inc. (NASDAQGS:CLVS) closed Tuesday up 4.77% with a volume north of 11.3 million. Earlier this week, Clovis announced after opposition proceedings at The Hague, Netherlands, that the European Patent Office upheld claims of European Patent 2534153 in amended form covering certain crystalline forms of rucaparib camsylate, including rucaparib S-camsylate Form A, the crystalline form in Rubraca. In its oral decision announced at the hearing, the Opposition Division upheld claims, narrowed from the originally granted patent, to certain crystalline forms of rucaparib camsylate. These forms include, but are not limited to, the commercial product. The European Opposition Division found patentability of the claimed forms based on the inventiveness of these crystalline forms and a constellation of unexpected properties. The European patent was opposed by two opponents. Clovis and/or either opponent have an opportunity to appeal the decision of the European Opposition Division within two months of the written decision, which is expected in the next few months. If appealed, all claims in the originally granted patent will remain in force until the Technical Board of Appeal issues its decision.


Novelion Therapeutics Inc. (NASDAQGS:NVLN) came to a close Tuesday up 32.65% with over 17.4 million shares traded on the day. The company recently announced that General Counsel, Benjamin Harshbarger, has been appointed as interim CEO, reporting to Novelion’s Executive Chair, Mark Corrigan, M.D., effective immediately. Jeffrey Hackman resigned as interim CEO to pursue another opportunity and will stay with the Company in a transitional role until November 30, 2018. Dr. Corrigan commented, “In the past year, we have made substantial progress in the turnaround of Novelion, and on behalf of the Board of Directors, I’d like to thank Jeff for his dedication to this mission. Importantly, Ben has played a critical role in these initiatives, including the recent loan facility secured by our operating subsidiary, Aegerion Pharmaceuticals, Inc. (“Aegerion”), which we believe positions Aegerion to achieve a more comprehensive capital restructuring – our primary near-term goal. Ben is a long-tenured and trusted leader among Novelion’s employees, and under his guidance we are well-positioned to continue the positive momentum towards fixing our capital structure and driving growth.”


AstraZenaca PLC (NYSE:AZN) closed Tuesday up sligthly with more than 5.9 million shares traded. Last week, the company presented 27 abstracts, including six oral presentations, at the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition in San Diego, CA. New data include presentations on CALQUENCE® (acalabrutinib) and LUMOXITI™ (moxetumomab pasudotox-tdfk), as well as research findings from AstraZeneca’s early pipeline, across a variety of blood cancers. Dave Fredrickson, Executive Vice President, Head of Oncology Business Unit, said: “In less than a year, we have launched two innovative medicines to treat blood cancers. At this year’s ASH, we will continue our momentum by presenting new results from two important trials of CALQUENCE in mantle cell lymphoma and chronic lymphocytic leukemia, and further showcase our broad pipeline with data from our novel MCL1 and CDK9 inhibitors.”


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