Clinical Trials in Gene Sequencing Reveal Novel Mutations Aiding in New Treatments For Acute Myeloid Leukemia

Palm Beach, FL –October 29, 2019 – Acute myeloid leukemia (AML), is the most common of the four major types of leukemia. Without treatment, AML is often deadly within a few months. Many of the symptoms of the disease are nonspecific and could include symptoms related to anemia, unexplained bleeding or bruising, weight loss, unexplained neurologic complaints like headache, and a marked reduction in CBC cell counts. This is a cancer that is rarely seen before age 45 with the average age of onset around 65. Treatment for AML can vary based on a number of factors, but in general the first-line therapy consists of induction therapy.  The American Cancer Society (ACS) estimates that about two-thirds of patients who undergo standard induction therapy will achieve remission. About half of patients who achieve remission and undergo consolidation will achieve long-term remission. While those are pretty good odds for remission compared to many other types of cancer, it does leave many who need further options for treatment. A search on shows high interest in the continuation of research for additional therapies for AML.  Active biotech and pharma companies in the markets this week include Moleculin Biotech, Inc. (NASDAQ: MBRX), Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), Rigel Pharmaceuticals, Inc. (NASDAQ: RIGL), Nektar Therapeutics (NASDAQ: NKTR), BioDelivery Sciences International, Inc. (NASDAQ: BDSI).


With new drug approvals and hundreds of clinical trials underway in the treatment of AML, it is apparent that there is quite a bit of interest in producing new treatment options for AML. This brings new hope to patients who cannot receive traditional therapies, or who have relapsed despite receiving traditional therapy already. It will be interesting to monitor the impact new therapies will have on the market considering the high cost of treatment with the new therapies and the relatively high success of lower cost traditional chemotherapy for those eligible for treatment.


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, announced the presentation of a poster at the AACR-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference today in Boston, MA.  The poster, entitled “Dose and Schedule-Dependent Efficacy of Liposomal Annamycin in Pre-clinical Models of Acute Myeloid Leukemia,” presents data documenting the high activity of Annamycin against AML, including in vitro studies in a panel of human AML cell lines, as well as in vivo studies in both human and murine AML models developed under the Company’s sponsored research agreement with MD Anderson Cancer Center.


“This study highlights an important new finding,” commented Walter Klemp, Moleculin’s Chairman and CEO.  “We’ve known for some time that Annamycin is effective in AML animal models and the activity that we believe is coming from our current AML clinical trials seems to correlate with this.  But what’s new here is the observation that Annamycin may also be more effective than other drugs due to its high uptake and effectiveness in eliminating AML cells localized in different organs.  Additional important observations made with these studies indicates that the long-term exposure of healthy mice (at least 12 doses so far) to a highly efficacious dose of 4 mg/kg administered weekly is not toxic and that even two weekly doses of 4 mg/kg are producing a significant increase in survival. And, because Annamycin is designed to be non-cardiotoxic, this extended dosing regimen may prove to be feasible and beneficial in humans.  This potentially opens the door for expanded and improved dosing regimens in future clinical trials.”


Quoting from the accepted abstract: “In vivo studies confirmed anti-tumor efficacy of Annamycin in both human and murine AML models. Based on bioluminescence imaging, the liposomal formulation of the drug significantly delayed AML progression in the human OCL-AML3/NSG model at 4 mg/kg with once weekly dosing. Similarly, significant dose-dependent reduction of peripheral blood AML blasts was observed in the murine AML-Turq2 model, and this reduction was strongly correlated with prolongation of animal survival. The median survival of mice receiving four doses of L-Ann once a week at 4 mg/ml was 37 days while mice receiving vehicle lived only 14 days (p=0.0002). Different doses and administration schedules of [Annamycin] were tested in an effort to maximize survival benefits.  In summary [Annamycin] is effective in AML, demonstrating significant activity in both in vitro and in vivo mouse models with a distinct pattern of intracellular uptake and organ distribution using a once a week schedule. This suggests that [Annamycin] with this profile, including a lack of cardiotoxicity and activity against [doxorubicin] resistant tumors, may be an advantageous approach in the treatment of AML.”   Read this and more news for MBRX at     


Other recent developments in the biotech industry include:


According to the Leukemia & Lymphoma Society (LLS) the many chromosomal and genetic abnormalities in AML make treating AML challenging. There is a need to identify these genetic variations and customize treatment options based on the genetic characteristics of the leukemia cells. Newer techniques in gene sequencing have revealed novel mutations that may be involved in the development of AML. This information will help researchers develop new targeted therapies, tailored to each patient’s disease. Recent trials demonstrate that testing for gene mutations may improve treatment options. Additional testing will be needed to confirm these findings.


LLS said: “Researchers are trying to find more effective and safer treatments for AML. Researchers are studying new drugs, and they are also studying the use of existing drugs given in different doses and on different schedules. In the last 10 years, improvements in overall survival of AML patients has been driven by using older chemotherapy drugs more effectively. Researchers are continuing to modify and reformulate traditional chemotherapy drugs to improve overall survival. They are also looking at combining chemotherapy with newer types of drugs to see if this approach may work to increase patient survival.”


Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a clinical-stage biopharmaceutical company addressing key mechanisms of tumor drug resistance, this week presented data from its ongoing Phase 1b/2 clinical study of rebastinib, an oral TIE2 kinase inhibitor, in combination with paclitaxel at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston. In addition, the Company also presented data from preclinical studies of DCC-3116, a potential first-in-class autophagy inhibitor to treat mutant RAS cancers.


“Both of these datasets highlight the broad applicability of Deciphera’s kinase switch control platform and our potential to address unmet needs in oncology,” said Matthew L. Sherman, M.D., Executive Vice President and Chief Medical Officer of Deciphera. “We look forward to continuing Part 2 of our Phase 1b/2 study of rebastinib in combination with paclitaxel with the insights garnered from Part 1 of the study. We also look forward to advancing the IND-enabling studies for DCC-3116.”


Rigel Pharmaceuticals, Inc. (NASDAQ: RIGL) recently announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has adopted a positive trend vote on the Marketing Authorization Application (MAA) for fostamatinib disodium hexahydrate (fostamatinib). The indication for the positive trend vote is for the treatment of chronic immune thrombocytopenia in adult patients who are refractory to other treatments.


“During the EMA review process for fostamatinib in adult chronic ITP, we have had very constructive interactions with the committee,” said Raul Rodriguez, president and CEO of Rigel. “We are pleased with this positive trend vote from the CHMP this week, which brings us one step closer to potentially providing a new therapeutic option for a patient population that has a clear unmet clinical need.”


Nektar Therapeutics (NASDAQ: NKTR) recently announced the initiation of a first-in-human, Phase 1 clinical study evaluating NKTR-255, an interleukin-15 (IL-15) receptor agonist, as monotherapy for patients with relapsed or refractory non-Hodgkin lymphoma (NHL) or multiple myeloma (MM). The study will also combine NKTR-255 with multiple targeted antibodies, that function through an antibody-dependent cell-mediated cytotoxicity (ADCC) mechanism, to evaluate the safety and efficacy in adults with relapsed or refractory MM. NKTR-255 is designed to activate the IL-15 pathway and expand functionally superior natural killer (NK) cells and promote the survival and expansion of memory CD8+ T cells without inducing suppressive regulatory T cells.


“We are excited to launch the first-in-human clinical study of NKTR-255, which has shown promising and substantial anti-tumor activity in our preclinical studies,” said Wei Lin, M.D., Senior Vice President and Head of Development at Nektar Therapeutics. “By increasing the number and activity of NK cells, NKTR-255 has the potential to enhance the host’s tumor-fighting response, both as a single agent and in combination with tumor-targeting antibodies, including daratumumab and rituximab.”


BioDelivery Sciences International, Inc. (NASDAQ: BDSI), a specialty pharmaceutical company dedicated to patients living with chronic conditions, this month announced that a major PBM will begin providing improved patient access to BELBUCA® (buprenorphine buccal film), CIII, and Symproic® (naldemedine) tablets 0.2 mg beginning October 1, 2019, with full plan adoption by January 1, 2020.  This will enable approximately 14 million covered lives within both commercial and health exchange plans to access BELBUCA as either the preferred or preferred exclusive buprenorphine product within their respective plans and Symproic as the preferred exclusive product within its class.  The addition of this large national PBM brings the total number of commercial lives with preferred access to BELBUCA to more than 104 million (out of more than 160 million with coverage) and more than 76 million overall covered lives for Symproic.



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