Orphan Rare Diseases R&D Advancements Increase Novel Therapeutic Options for Drug Development Pipeline

Wednesday, January 10, 2018 Drug News
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PALM BEACH, Florida, January 10, 2018 /PRNewswire/ --

Rare conditions

are also known as Orphan diseases which are generally categorized as chronic, degenerative and life-threatening in nature and normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders, which
are extremely life-threatening such as Acute Myeloid Leukemia (AML). Treatment and prognosis in Acute Myeloid Leukemia is strongly influenced by a patient's age and cytogenetic profile while the majority of diagnosed patients are of the age 65 or over. Current developmental pipelines are forging ahead with Research and Development studies to address the therapeutic needs in the market, along with identifying and developing significant targeted treatments. According to the latest market report published by Credence Research, Inc. - Acute Myeloid Leukemia (AML) Therapeutics Market - Growth, Future Prospects and Competitive Analysis, 2017 - 2025 - the global acute myeloid leukemia therapeutics market was valued at US$404.3 Million in 2016, and is expected to reach US$1.025 Billion by 2025, expanding at a CAGR of 10.9% from 2017 to 2025. Active in the biotech and pharma industry today includes: Moleculin Biotech, Inc. (NASDAQ: MBRX), Global Blood Therapeutics Inc. (NASDAQ: GBT), Pain Therapeutics Inc. (NASDAQ: PTIE), TEVA Pharmaceuticals Industries Limited (NYSE: TEVA), Diffusion Pharmaceuticas Inc. (NASDAQ: DFFN).

Moleculin Biotech, Inc. (NASDAQ: MBRX), a clinical stage pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the MD Anderson Cancer Center ("MD Anderson"), today announced it has expanded the Company's development pipeline for the treatment of acute myeloid leukemia ("AML") with an immuno-stimulating STAT3 inhibitor. "Leading experts in the treatment of AML, Dr. Jorge Cortes and Dr. Sanjay Awasthi have now asked us to expand our clinical research to include WP1066, our immuno-stimulating agent and STAT3 inhibitor, to increase therapeutic options for AML patients," commented Walter Klemp, Chairman and CEO of Moleculin. "This would potentially be complementary and synergistic with Annamycin and existing first line treatments and could position us as a leader in the advancement of leukemia treatments." Read this and more news for Moleculin Biotech at: http://www.marketnewsupdates.com/news/mbrx.html   Dr. Sanjay Awasthi, Professor of the Department of Internal Medicine, Division of Hematology & Oncology, Texas Tech University Health Sciences Center and Medical Director, Southwest Cancer Center Lubbock, Texas, added, "The apparent ability in pre-clinical trials of WP1066 to stimulate the patient's natural immune response and simultaneously inducing tumor cell death by inhibiting the activated form of STAT3 is highly promising and unique. Importantly, based on current scientific findings, such properties should be extremely valuable in developing improved treatments for AML patients and expanding their therapeutic options. "We are clearly excited to see what Moleculin's Annamycin can do for relapsed or refractory AML patients in the Company's recently launched clinical trial," continued Dr. Awasthi, "thus given the potential for an even broader arsenal of AML drugs, we are encouraging Moleculin to expand their AML clinical research to include this novel immuno-stimulating STAT3 inhibitor drug candidate." Another noted AML expert, Dr. Jorge Cortes, is also encouraging Moleculin's clinical expansion, commenting: "AML appears to be associated with a significant increase in the activation of STAT3 and many of us in the AML clinical community have been eager to test the ability of a STAT3 inhibitor to treat AML patients. Part of the difficulty in pursuing this path has been finding a safe and effective STAT3 inhibitor and, if successful, WP1066 may have finally opened this pathway." In other biotech developments in the markets: 

Global Blood Therapeutics Inc. (NASDAQ: GBT) closed up over 19% on Tuesday at $47.15 trading over 4.2 million shares by the market close and was also up slightly in afterhours trading. Global Blood Therapeutics announced on Tuesday that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to voxelotor (previously called GBT440) for the treatment of sickle cell disease (SCD). Voxelotor is being developed as a disease-modifying therapy for SCD and previously received European Medicines Agency (EMA) Priority Medicines (PRIME) designation for the treatment of SCD. "The FDA's decision to grant voxelotor the first Breakthrough Therapy designation for the treatment of sickle cell disease reflects a recognition of the promising efficacy and safety data we have collected to date for this investigational drug, as well as an acknowledgement of the overwhelming need for major advances over available therapies in the treatment of SCD patients," said Ted W. Love, president and chief executive officer of GBT. "This designation is another significant milestone for GBT as we work to expedite the development of voxelotor."

Pain Therapeutics Inc. (NASDAQ: PTIE) closed up over 100% on Tuesday at $9.04 trading over 31 million shares by the market close and was up over 3% in afterhours trading. Pain Therapeutics announced positive results from a human abuse potential study of its late-stage drug candidate, REMOXY. Study results indicate that in non-dependent, recreational opioid users, nasal administration of REMOXY resulted in significantly lower abuse potential compared to immediate-release (IR) oxycodone. All study subjects reported reduced 'Drug Liking' 'Take Drug Again' and 'Drug High' for REMOXY compared to oxycodone IR. In addition, nasal administration of REMOXY showed lower exposure to oxycodone, lower peak concentrations (Cmax) and longer time to peak drug concentration (Tmax) against comparator drugs, suggesting comparatively lower abuse potential. "We believe these data indicate REMOXY may have limited nasal abuse potential relative to comparator drugs," said Remi Barbier, President & CEO. "We have now successfully completed all studies necessary to resubmit the REMOXY NDA to the FDA, and plan to do so shortly."

Teva Pharmaceutical Industries Limited (NYSE: TEVA) announced this week that its subsidiary, Teva Pharmaceuticals International GmbH., has signed a global license agreement with Alder BioPharmaceuticals. The agreement validates Teva's IP and resolves Alder's opposition to Teva's European Patent No. 1957106 B1, with respect to anti-calcitonin gene-related peptide (CGRP) antibodies and methods for their use. It also provides Alder with clarity for its ongoing plans in the field.Under the terms of the agreement, Alder has received a non-exclusive license to Teva's anti-CGRP antibodies patent portfolio to develop, manufacture and commercialize eptinezumab in the U.S. and worldwide, excluding Japan and Korea.

Diffusion Pharmaceuticas Inc. (NASDAQ: DFFN) closed Tuesday over 40% at $1.73 trading over 8.1 million shares by the market close. The company recently announced that a Phase 3 clinical trial using its lead small molecule trans sodium crocetinate ("TSC") in patients with newly-diagnosed inoperable glioblastoma multiforme ("GBM") brain cancer, is now open for enrollment. The trial, which has been named INTACT (INvestigating Tsc Against Cancerous Tumors), follows a previous Phase 2 GBM study in which the inoperable patient subgroup showed a nearly four-fold increase in survival compared with historical controls when TSC was added to their treatment regimen (40% alive at two years vs. 10.4%). TSC's innovative mechanism of action affects the tumor micro-environment, making treatment-resistant cancer cells more susceptible to the tumor-killing power of conventional radiation therapy ("RT") and chemotherapy (temozolomide) by re-oxygenation of the hypoxic portion of the tumor. The Company believes that a largely intact GBM tumor vasculature with limited surgical resection is conducive to TSC's tumor re-oxygenation properties, and that this contributed to the survival increase in the Phase 2 GBM inoperable patient subgroup.

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