R&D Leading to Supported Progress for Treating Acute Myeloid Leukemia (AML)

PALM BEACH, Florida, Feb. 5, 2020 /PRNewswire/ -- Acute Myeloid Leukemia (AML) research has seen great progress with more researchers are now studying the causes, diagnosis, and treatment of Acute myeloid leukemia (AML) at many medical centers, university hospitals, and other institutions. Acute myeloid leukemia (AML) is the second most common type of leukemia diagnosed in adults and children. The American Cancer Society's estimates for ALM in the United States, for 2019, indicates that the number of new cases of acute myeloid leukemia (AML) will be around 21,450, most of whom will be the be adults. In terms of mortality, the society has estimated that there will be around 10,920 deaths due to AML, in the country. This statistic shows that there is a huge number of people that are prone to suffer from acute myeloid leukemia, which may directly impact the growth of the AML market in the United States. Active biotech and pharma companies in the markets this week include Moleculin Biotech, Inc. (NASDAQ: MBRX), La Jolla Pharmaceutical Company (NASDAQ: LJPC), Insmed Incorporated (NASDAQ: INSM), Teva Pharmaceutical Industries Limited (NYSE: TEVA), Puma Biotechnology, Inc. (NASDAQ: PBYI).

There has been advancing progress in understanding how changes in the 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. Perhaps even more important, this knowledge is now being used to help develop newer targeted therapies against AML. The global acute myeloid leukemia market was valued at USD 701.6 million in 2018, and is estimated to be valued at USD 1,539.99 million in 2024, witnessing a CAGR of 14.0%. The key factors propelling the growth of this market are high Incidence and prevalence of acute myeloid leukemia, advancements in pharmacology and molecular biology to promote drug development, and increasing investments in R&D by the pharmaceutical companies, reports an industry insider, Mordor Intelligence.

Moleculin Biotech, Inc. (NASDAQ:MBRX) BREAKING NEWS: Moleculin to Seek Accelerated FDA Approval and Plans for Pivotal Phase 2 AML Trial - Moleculin Biotech, a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, today announced it intends to discuss with the FDA and EMA (European Medicines Agency) plans to conduct a single arm Phase 2 trial that would serve as the basis for accelerated approval of Liposomal Annamycin ("Annamycin") to treat relapsed or refractory acute myeloid leukemia ("AML"). This will follow the establishment of a recommended Phase 2 dose ("RP2D") in the Company's ongoing Phase 1 dose escalation trial in Europe. The FDA has already granted Annamycin Fast Track status and Orphan Drug Designation for AML. FDA grants Fast Track designation to drugs intended to treat serious conditions that demonstrate the potential to address unmet medical needs, which can include providing efficacy comparable to available therapy while avoiding toxicity associated with the existing treatment. The benefits of Fast Track include FDA actions to expedite development and review, including "rolling review," where the agency reviews portions of a marketing application before the complete application is submitted.

The announcement follows continuing positive results from Moleculin's open label, single arm Phase 1 trials in AML patients in Europe and the US. Most recently, the US Phase 1 study met its primary objective of demonstrating the safety of Annamycin at a dose that was cumulatively at or below the lifetime maximum anthracycline dose. Those results are consistent with results achieved with the parallel Phase 1 study being conducted in Europe, which has demonstrated the safety of escalating doses of Annamycin in AML patients, including doses that significantly exceed the maximum lifetime dose of anthracyclines imposed in the US. In both trials, the primary endpoints are aimed at demonstrating the product's safety, primarily the lack of cardiovascular risk. This is a key characteristic that, if borne out, could significantly differentiate Annamycin from other anthracyclines, which generally are well-known to have treatment-limiting cardiotoxicity.

Based on these results, Moleculin will continue to focus the Company's efforts on the European trial to establish an RP2D. Once that is complete, the Company intends to enter discussions with the FDA and EMA about conducting a single arm Phase 2 study that would be the pivotal trial supporting US and European approval of Annamycin for relapsed or refractory AML.

Walter Klemp, Chairman and CEO of Moleculin commented, "We believe relying upon the European trial to establish an RP2D is the fastest and most efficient way to reach a pivotal Phase 2 trial. Recruitment in Europe has been faster than in the US and the trial is progressing well. The US Phase 1 trial was designed to demonstrate that Annamycin is indeed non-cardiotoxic when delivered to patients at or below the lifetime maximum anthracycline dose, and it has served that purpose. Beyond that, we have now treated 9 patients in the European trial above the lifetime maximum, also without any evidence of cardiotoxicity."

The U.S. trial met its primary endpoint, demonstrating the safety of Annamycin in AML patients, most importantly the absence of cardiotoxicity (potential damage to the heart), as determined by echocardiograms, as well as cardiac health biomarkers, principally blood troponin levels. Based on testing to date, no patients in either the US or European trial have exhibited evidence of cardiotoxicity. Additionally, there were no unexpected serious adverse events (SAE) and no dose limiting toxicities (DLT) at any dose tested. Although a primary objective of the Phase 1 trial was to evaluate safety, the study also gathered data to support a preliminary assessment of the product's efficacy. Among other things, the study recorded complete response (CR), partial response (PR), event-free survival (EFS), overall survival (OS; Kaplan-Meier), and time to and duration of remission/response. The Company reported efficacy in 33% of the US patients, even though the drug was dosed at what was expected to be sub-therapeutic levels. The evidence of efficacy consisted of 1 patient who achieved a "morphologically leukemia-free state," which the protocol defined as a CR with incomplete recovery of platelets or neutrophils, and another patient who had a substantial remission of leukemia cutis (a somewhat rare leukemia symptom), from diffuse to 3 lesions. Read this and more news for MBRX at: https://www.financialnewsmedia.com/news-mbrx/ 

Other recent developments in the biotech industry include: 

La Jolla Pharmaceutical Company (NASDAQ: LJPC) recently announced preliminary GIAPREZA™ (angiotensin II) net sales for the three and twelve months ended December 31, 2019. For the three months ended December 31, 2019, preliminary GIAPREZA net sales were $7.2 million, up 71% from the three months ended December 31, 2018 and up 26% from the three months ended September 30, 2019. Vials of GIAPREZA shipped from distributors to hospitals (hospital demand) grew 74% for the three months ended December 31, 2019 as compared to the three months ended December 31, 2018 and 18% as compared to the three months ended September 30, 2019. For the twelve months ended December 31, 2019, preliminary GIAPREZA net sales were $23.1 million, up 129% from the twelve months ended December 31, 2018. La Jolla announced the commercial availability of GIAPREZA in the U.S. in March 2018.

Insmed Incorporated (NASDAQ: INSM) recently announced positive top-line results from its global, randomized, double-blind placebo-controlled Phase 2 WILLOW study evaluating the efficacy, safety, and pharmacokinetics of INS1007 administered once daily in adults with non-cystic fibrosis bronchiectasis (NCFBE). INS1007 is a novel, oral, selective, reversible inhibitor of dipeptidyl peptidase 1 (DPP1). 

The WILLOW study met its primary endpoint of time to first pulmonary exacerbation over the 24-week treatment period for both the 10 mg and 25 mg dosage groups of INS1007 compared to placebo (p=0.027, p=0.044, respectively). In addition, treatment with INS1007 resulted in a reduction in the frequency of pulmonary exacerbations, a key secondary endpoint, versus placebo. Specifically, patients treated with INS1007 experienced a 36% reduction in the 10 mg arm (p=0.041) and a 25% reduction in the 25 mg arm (p=0.167) versus placebo. Change in concentration of active neutrophil elastase (NE) in sputum versus placebo from baseline to the end of the treatment period was also statistically significant (p=0.034 for 10 mg, p=0.021 for 25 mg).

Teva Pharmaceutical Industries Limited (NYSE: TEVA) recently announced positive results from two Phase 2/3 trials evaluating AJOVY^® (fremanezumab) in patients in Japan. AJOVY is under development in Japan by Otsuka Pharmaceutical Co., Ltd. (Otsuka) as part of a May 2017 exclusive license agreement for development and sales of AJOVY in Japan.

"This study adds to the wealth of positive AJOVY data we have in patients globally," said Joshua M. Cohen, MD, MPH, FAHS, Global Medical Lead for Migraine & Headache at Teva. "The annual prevalence of migraine in Japan is 8.4% of adults,¹ so we are pleased to be one step closer to bringing AJOVY to patients in Japan who could benefit from a preventive treatment."

Puma Biotechnology, Inc. (NASDAQ: PBYI) closed up over 231.59% on Tuesday at $10.58/share trading over 4.3 Million shares by the market close.  Its average volume is slightly over 1.7 million shares/day.  Puma Biotechnology, Inc. is a biopharmaceutical company with a focus on the development and commercialization of innovative products to enhance cancer care. Puma in-licenses the global development and commercialization rights to PB272 (neratinib, oral), PB272 (neratinib, intravenous) and PB357. Neratinib, oral was approved by the U.S. Food and Drug Administration in July 2017 for the extended adjuvant treatment of adult patients with early stage HER2-overexpressed/amplified breast cancer, following adjuvant trastuzumab-based therapy and is marketed in the United States as NERLYNX® (neratinib) tablets. NERLYNX was granted marketing authorization by the European Commission in August 2018 for the extended adjuvant treatment of adult patients with early stage hormone receptor-positive HER2-overexpressed/amplified breast cancer and who are less than one year from completion of prior adjuvant trastuzumab-based therapy. NERLYNX is a registered trademark of Puma Biotechnology, Inc.

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