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

MBRX, OGEN, AZN

Financialnewsmedia.com News Commentary

PR Newswire

PALM BEACH, Florida, December 6, 2018 /PRNewswire/ --

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:  https://www.financialnewsmedia.com/news-mbrx/

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 https://onlinelibrary.wiley.com/doi/abs/10.1111/cbdd.13365 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."

DISCLAIMER: FN Media Group LLC (FNM), which owns and operates FinancialNewsMedia.com and MarketNewsUpdates.com, is a third party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNM is NOT affiliated in any manner with any company mentioned herein. FNM and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security. FNM's market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities. The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material. All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release. FNM is not liable for any investment decisions by its readers or subscribers. Investors are cautioned that they may lose all or a portion of their investment when investing in stocks.  For current services performed FNM has been compensated forty six hundred dollars for news coverage of the current press releases issued by Moleculin Biotech, Inc. by the company. FNM HOLDS NO SHARES OF ANY COMPANY NAMED IN THIS RELEASE.

This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. "Forward-looking statements" describe future expectations, plans, results, or strategies and are generally preceded by words such as "may", "future", "plan" or "planned", "will" or "should", "expected," "anticipates", "draft", "eventually" or "projected". You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors, and other risks identified in a company's annual report on Form 10-K or 10-KSB and other filings made by such company with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and FNM undertakes no obligation to update such statements.

Contact Information:
email: editor@financialnewsmedia.com  
+1(561)325-8757


SOURCE FinancialNewsMedia.com



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