Clinical Trials for Acute Myeloid Leukemia (AML) Drug Therapy Advancing with Encouraging Results

Palm Beach, FL –October 22, 2019 – There is much research activity in the acute myeloid leukemia (AML) the sector. The American Cancer Society’s (ACS) estimates for leukemia in the United States for 2019 predict that there will be 61,780 new cases of leukemia (all kinds) and 22,840 deaths from leukemia (all kinds) and about 21,450 new cases of acute myeloid leukemia and about 10,920 deaths from AML. Most will be in adults.  Although it is classified as an orphan’s disease, many researchers are now studying the causes, diagnosis, and treatment of acute myeloid leukemia (AML) at many medical centers, university hospitals, and other institutions. The results in treating leukemia are encouraging according to recent clinical trials published in the New England Journal of Medicine (NEJM) and The Lancet Oncology.  Currently, most patients with AML are treated with standard chemotherapy. But recent scientific advances have revealed that there are many forms of the disease, each with different specific genetic changes that may affect cancer growth and treatment.  As such, there has been increasing research into the development of new targeted therapies that inhibit specific mutations found in AML. These therapies are especially important for patients who are unable to tolerate chemotherapy, or whose cancer has relapsed after standard treatment.    Active biotech and pharma companies in the markets this week include Moleculin Biotech, Inc. (NASDAQ: MBRX), Clovis Oncology, Inc. (NASDAQ: CLVS), Seattle Genetics, Inc. (NASDAQ: SGEN), ACADIA Pharmaceuticals Inc. (NASDAQ: ACAD), Agios Pharmaceuticals, Inc. (NASDAQ: AGIO).


The American Cancer Society added that: “There has been great progress in understanding how changes in the DNA (genes) inside normal bone marrow cells can cause them to develop into leukemia cells. A greater understanding of the gene changes that often occur in AML is providing insight into why these cells become abnormal. As researchers have found more of these changes, it is becoming clear that there are many types of AML. Each might have different gene changes that affect how the leukemia will progress and which treatments might be most helpful. Doctors are now learning how to use these changes to help them determine a person’s outlook and if they should receive more or less intensive treatment.  Perhaps even more important, this knowledge is now being used to help develop newer targeted therapies against AML. While chemotherapy, is still the main treatment for most types of AML, the effectiveness of chemo may be limited in some cases because the leukemia cells become resistant to it over time. Researchers are now looking at ways to prevent or reverse this resistance by using other drugs along with chemo. They are also looking at combining chemo with a number of newer types of drugs to see if this might work better.”


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 the expansion of Annamycin production commitments in response to management’s assessment of positive AML clinical trial activity and the potential expansion of indications for use to include lung-localized tumors.  The purchase commitment arranged through Davos Pharmaceuticals includes moving final production of Annamycin to a larger-scale suite within BSP Pharmaceuticals S.p.A. (“BSP”) in Latina, Italy.  Until now, BSP has been producing the clinical supplies of Annamycin in a smaller pilot-scale suite.


“Our clinical trials of Annamycin in relapsed and refractory acute myeloid leukemia (“AML”) have been going better than expected, as it now appears we will be able to reach a higher maximum tolerable dose than what was established in previous clinical trials,” commented Walter Klemp, Moleculin’s Chairman and CEO.  “That not only increases our chances for improved patient outcomes, it places a higher demand on drug supply.  Coupled with the recent discovery in animal models that Annamycin may be well suited to treat lung-localized tumors because of its ability in such models to accumulate in the lungs at nearly 6 times the level of the current standard of care anthracycline, we are clearly going to need more drug.”


Dr. Donald Picker, Moleculin’s Chief Science Officer added: “With management’s view of success in clinic and the expectation of wider demand resulting from additional clinical trials in lung-localized tumors, it’s time for Moleculin to start preparing for an eventual drug approval process.  That requires the development and validation of commercial scale methods and this move with BSP marks the beginning of that process.  In addition to increasing the scale of clinical supply production, we will be working with BSP and with our manufacturer of API to develop the commercial scale synthesis and drug production methods we will need to ultimately prepare for New Drug Approval.    Read this and more news for MBRX at     


Other recent developments in the biotech industry include:


Clovis Oncology, Inc. (NASDAQ: CLVS) this month announced that the National Institute for Health and Care Excellence (NICE) has recommended that women with relapsed ovarian cancer in England have access to rucaparib through the Cancer Drugs Fund (CDF).1 Rucaparib is available for use within the CDF as an option for the maintenance treatment of relapsed, platinum-sensitive high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer that has responded to platinum-based chemotherapy in adults, based on the conditions outlined in the managed access agreement.


“Ovacome welcomes the availability of rucaparib via the CDF as an option for maintenance treatment of platinum-sensitive relapsed high grade serous epithelial ovarian cancer regardless of BRCA status or line of treatment in the relapsed maintenance setting,” said Victoria Clare, CEO of Ovacome, a United Kingdom ovarian cancer charity focused on providing support to anyone affected by ovarian cancer. “It is vital that the expansion of available maintenance options continues as maintenance treatments extend the time between chemotherapies. Many women with relapsed ovarian cancer know that they are facing a future of managing their disease as a chronic illness.”


Seattle Genetics, Inc. (NASDAQ: SGEN) recently announced positive topline results from the HER2CLIMB trial, a randomized, double-blind, placebo-controlled, active comparator pivotal trial evaluating tucatinib. The trial compared tucatinib in combination with trastuzumab and capecitabine to trastuzumab and capecitabine alone in patients with locally advanced unresectable or metastatic HER2-positive breast cancer. Patients had previously received trastuzumab, pertuzumab and ado-trastuzumab emtansine (T-DM1), and 47 percent of the patients enrolled in the trial had brain metastases at the time of enrollment. Tucatinib is an oral, small molecule tyrosine kinase inhibitor (TKI) that is highly selective for HER2.


The trial met the primary endpoint of progression-free survival (PFS), showing that the addition of tucatinib was superior to trastuzumab and capecitabine alone, with a 46 percent reduction in the risk of disease progression or death (hazard ratio (HR)=0.54 (95% Confidence Interval (CI): 0.42, 0.71); p<0.00001). The trial also met the two key secondary endpoints at interim analysis. The tucatinib arm demonstrated an improvement in overall survival, with a 34 percent reduction in the risk of death (HR=0.66 (95% CI: 0.50, 0.88); p=0.0048) compared to trastuzumab and capecitabine alone. For patients with brain metastases at baseline, the tucatinib arm also demonstrated superior PFS, with a 52 percent reduction in the risk of disease progression or death compared to those who received trastuzumab and capecitabine alone (HR=0.48 (95% CI: 0.34, 0.69); p<0.00001).


ACADIA Pharmaceuticals Inc. (NASDAQ: ACAD) recently announced that based on secondary analyses from its Phase 2 CLARITY study, adjunctive pimavanserin showed the potential to improve symptoms of sexual dysfunction experienced by patients with major depressive disorder (MDD). These additional data were presented in the poster, “Improvement of Sexual Function Observed During Treatment of Major Depressive Disorder With Adjunctive Pimavanserin” at the 2019 Psych Congress, October 3-6 in San Diego, California.


The CLARITY study was a 10-week, double-blind, placebo-controlled, two-stage sequential parallel comparison design (SPCD) study, which evaluated the efficacy, safety, and tolerability of pimavanserin as an adjunctive treatment for MDD in patients who have had an inadequate response to SSRI or SNRI therapy. In the study, pimavanserin met the overall primary endpoint, the key secondary endpoint, and seven of the eleven pre-specified additional secondary endpoints, including the Massachusetts General Hospital Sexual Functioning Index (MGH-SFI) (nominal p=0.0003)1. In addition, in Stage 1, the all-inclusive, parallel design portion of the study (n=207), adjunctive pimavanserin showed significant improvement relative to placebo on mean MGH-SFI scores from baseline after five weeks of treatment (nominal p=0.0002; effect size=0.614).


Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) recently presented detailed data from the phase III ClarIDHy study on its leukemia drug Tibsovo (ivosidenib), which is being evaluated in previously treated patients with IDH1 mutant cholangiocarcinoma, also called bile-duct cancer.


In the study, the median progression-free survival (PFS) was 2.7 months for patients who received Tibsovo compared to 1.4 months in the placebo arm. Importantly, patients who were treated with Tibsovo had a 6-month and 12-month PFS rate of 32% and 22%, respectively, while none of the patients in the placebo arm was free from disease progression for more than six months.


Tibsovo also reduced the risk of disease progression or death by 63%. In the Tibsovo arm, partial response was achieved by 2% of patients and 51% of patients had stable disease while in the placebo arm, 28% of patients had stable disease.


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