Biotech Brief: FDA Fast Tracking Innovative Breakthrough Therapies For Cancer Patients

Palm Beach, FL – April 18, 2019 – A recent headline from a report released by Friends of Cancer Research, made the following eye-opening statement: “ONE OF THE BIGGEST TREATMENT BREAKTHROUGHS IN THE PAST FIVE YEARS WASN’T A DRUG, BUT A LAW.” The article was looking at the effects of the “Advancing Breakthrough Therapies for Patients Act” a law that enables the FDA to work closely with the drug developer/sponsor and puts the treatment on a much faster track. The article continued: “A new drug may be designated as a breakthrough therapy by the Food and Drug Administration (FDA) if it is intended to treat a serious or life-threatening disease and preliminary clinical evidence suggests it provides a substantial improvement over existing therapies. Once the breakthrough therapy designation is requested by the drug sponsor, the FDA and sponsor work together to determine the most efficient path forward.  As of March 27, 2019 the FDA has approved 132 breakthrough therapy designated products and… that there have been 766 total requests for the designation with 292 designations granted.”   Active biotech and pharma companies in the markets this week include Moleculin Biotech, Inc. (NASDAQ:MBRX), T Bio-Path Holdings, Inc. (NASDAQ: BPTH), Pfizer Inc. (NYSE: PFE), Wave Life Sciences Ltd. (NASDAQ: WVE), Zosano Pharma Corporation (NASDAQ: ZSAN).


The article continued: “As knowledge about complex diseases such as cancer… has expanded, researchers have been able to develop increasingly precise methods of treatment. Targeted therapies, a variety of drugs that target specific molecular pathways, allow physicians and researchers to identify patients highly likely to respond to treatment. Frequently, these new drugs show major clinical activity and significant improvement over currently available treatment early in their development. In cases where new drugs show so much promise, and particularly in cases where existing treatment options are limited, the traditional multi-phase, sequential drug development process may not be appropriate… A new drug may be designated as a breakthrough therapy if it is intended to treat a serious or life-threatening disease and preliminary clinical evidence suggests it provides a substantial improvement over existing therapies.”


Moleculin Biotech, Inc. (NASDAQ:MBRX) BREAKING NEWS:  Moleculin Biotech, a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, today announced that the U.S. Food and Drug Administration (“FDA”) has approved its request for Fast Track Designation for its drug, Annamycin, for the treatment of relapsed or refractory acute myeloid leukemia (“AML”).


“We are thrilled that Annamycin has been granted Fast Track Designation,” commented Walter Klemp, Moleculin’s Chairman and CEO. “Not only does this make us eligible for accelerated approval and priority review, but it serves as an important validation of the significant unmet need we are trying to address. Currently, Annamycin is in separate Phase I/II trials in the U.S. and Europe for the treatment of AML and the Company has recently announced positive interim top line data.”


A drug that receives Fast Track designation is eligible for some or all of the following:


  • More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
  • More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
  • Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
  • Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA


Read this and more news for MBRX at    


Other recent developments in the biotech industry include:


Bio-Path Holdings, Inc. (NASDAQ: BPTH) a biotechnology company leveraging its proprietary DNAbilize® antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, recently announced that data from pre-clinical studies supporting the potential of BP1003, a novel liposome-incorporated STAT3 oligodeoxynucleotide inhibitor, for the treatment of pancreatic cancer, non-small cell lung cancer (NSCLC) and acute myelogenous leukemia (AML) were presented in a poster at the American Association for Cancer Research (AACR) Annual Meeting 2019 in Atlanta, GA.


The poster, entitled “BP1003, a Novel Liposome-Incorporated STAT3 Antisense Oligodeoxynucleotide Inhibitor,” was presented by Ana Tari Ashizawa, Ph.D., Vice President of Research and Development at Bio-Path.


Pfizer Inc. (NYSE: PFE) recently announced that the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) to expand the indications for IBRANCE®(palbociclib) in combination with an aromatase inhibitor or fulvestrant to include men with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer. The approval is based on data from electronic health records and postmarketing reports of the real-world use of IBRANCE in male patients sourced from three databases: IQVIA Insurance database, Flatiron Health Breast Cancer database and the Pfizer global safety database.


Wave Life Sciences Ltd. (NASDAQ: WVE) a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, recently announced the final results from its Phase 1 clinical trial of investigational suvodirsen (WVE-210201) in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The company also announced the design of its planned Phase 2/3 clinical trial of suvodirsen in DMD, DYSTANCE 51. The Phase 1 data and DYSTANCE 51 details will be presented today at the 2019 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida.


“We are delighted that suvodirsen’s Phase 1 results demonstrate a favorable safety and tolerability profile that allow us to proceed rapidly into Phase 2/3 clinical development with doses we expect to be within the therapeutic window. Later this year, we expect to report interim efficacy data from the ongoing open-label extension study of suvodirsen and intend to use those data as an important component of a submission for accelerated approval in the United States,” said Michael Panzara, MD, MPH, Chief Medical Officer of Wave Life Sciences. “We truly appreciate the boys and families that made this trial possible.”


Zosano Pharma Corporation (NASDAQ: ZSAN), a clinical-stage biopharmaceutical company, recently announced the peer-reviewed publication of a review and analysis of recently-completed trials using newly FDA-recommended endpoints in the acute treatment of migraine.  The review article, led by Stanford Assistant Professor of Neurology, Nada Hindiyeh, MD., was published in Headache, titled “Review of Acute Treatment of Migraine Trial Results With the New FDA Endpoints: Design Implications for Future Trials.”


In this analysis, researchers reviewed eight published trials designed in accordance with the Guidance for Industry, “Migraine: Developing Drugs for Acute Treatment”, finalized in February 2018, that recommended using co-primary endpoints of pain freedom and freedom from most bothersome symptom (MBS) at 2-hours post-treatment. Using the placebo response rates from these various trials, the authors were able to calculate required sample sizes for future trials and compare these calculations to the sample sizes actually used in completed trials. Included in this analysis was Zosano’s ZOTRIP trial, in which 41.5% of subjects in the QTRYPTA 3.8mg arm achieved pain freedom at two hours and 68.3% achieved freedom from their MBS at two hours, as compared to 14.3% and 42.9% respectively in the placebo group.


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