Palm Beach, FL –December 4, 2019 – So it is not surprising that there is a lot of money and research resources being devoted to find new therapies for Acute Myeloid Leukemia (AML), many of which are in the targeted drugs arena. AML is the second most common type of leukemia diagnosed in adults and children. Even given all the research in new treatments, it is still often deadly within a few months. With all those research resources focused on AML, reports have projected that revenues in the market will continue to rise. A report from Mordor Intelligence states: “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.” Active biotech and pharma companies in the markets this week include Moleculin Biotech, Inc. (NASDAQ: MBRX), Audentes Therapeutics, Inc. (NASDAQ: BOLD) Outlook Therapeutics, Inc. (NASDAQ: OTLK), Axsome Therapeutics, Inc. (NASDAQ: AXSM), uniQure N.V. (NASDAQ: QURE).
The American Cancer Society’s estimates for leukemia in the United States, for 2019, indicates that approximately 61,780 new cases of leukemia and 22,840 deaths from leukemia are expected to occur in the country. Among them, the number of new cases of acute myeloid leukemia (AML) will be around 21,450, from which most of the population will 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.
Moleculin Biotech, Inc. (NASDAQ:MBRX) BREAKING NEWS: Moleculin Announces Additional Positive Interim Results from Phase 1/2 Clinical Studies of Annamycin in Acute Myeloid Leukemia – Moleculin Biotech, a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, today announced additional positive interim safety and efficacy data from one of the Company’s two ongoing open label, single arm Phase 1/2 studies of Annamycin for the treatment of relapsed or refractory acute myeloid leukemia (“AML”).
The Phase 1 portion of these clinical trials, which are described in more detail later in this press release, is designed to establish the safety of Annamycin and to determine the Recommended Phase 2 Dose to be used in the Phase 2 portion of the trials. While the Primary Endpoint of the Phase 1 portion is safety, a Secondary Endpoint is the assessment of efficacy generally defined as an improvement in bone marrow biopsy results sufficient to qualify patients for a potentially curative bone marrow transplant. The Company cautions not to place undue reliance on interim results.
The third cohort in Poland receiving a single dose of 180 mg/m2 in the Phase 1 dose escalation portion of the trial was completed with no adverse events and the trial will continue to the next cohort of 210 mg/m2. In the US trial, one patient has completed treatment in the second cohort at 120 mg/m2. This brings the total number of patients treated and evaluated at or above 120 mg/m2 to 10. An additional patient in the US has begun treatment at 120 mg/m2 but has yet to complete post-treatment evaluation. The interim results for these 10 patients are 1 CRi (defined as a complete response with incomplete recovery of white blood cells and/or platelets) and 2 partial responses (“PRs” or where bone marrow blasts are reduced 50% and to below 25%). One additional patient was bridged to bone marrow transplant (“BT”) based on a sufficient reduction in bone marrow blasts, bringing the total to 4 out of 10 patients at or above 120 mg/m2 who have demonstrated efficacy.
In the latest cohort in Poland, 1 of the 3 patients treated at 180 mg/m2 had a PR sufficient to qualify for a potentially curative bone marrow transplant. The results for all 3 patients were reviewed by the Safety Review Committee, which determined that no drug-related adverse events were observed that would prevent advancing the trial to the next higher dose level of 210 mg/m2. To date in the European trial, only one adverse event related to Annamycin has been reported; a patient experienced grade 2 mucositis (which resolved to grade 1 within 2 days). In the Company’s parallel US clinical trial, one new patient (the first of cohort #2) achieved a “morphologically leukemia free state” or MLFS, which also constitutes a CRi, after receiving a single dose of 120 mg/m2.
We refer to Annamycin as a “next generation anthracycline,” because it is designed to provide enhanced therapeutic benefits when compared with traditional anthracyclines (like doxorubicin) while reducing the potential for unwanted cardiotoxicity, or damage to the heart. This design intent has previously been validated with preclinical toxicology studies in animal models (as required by FDA) demonstrating Annamycin has little to no cardiotoxicity when compared with doxorubicin. Of the 14 patients treated thus far in both trials, none has shown any evidence of cardiotoxicity. This includes 7 patients in Poland who were treated at levels above the US maximum allowable cumulative anthracycline dose level (550 mg/m2), a limitation not imposed on our trial in Europe. If upheld in further studies, this lack of toxicity could be an important differentiator between Annamycin and the currently approved anthracyclines, for which cardiotoxicity is a well-known treatment limitation. Read this and more news for MBRX at: https://financialnewsmedia.com/news-mbrx/
Other recent developments in the biotech industry include:
Audentes Therapeutics, Inc. (NASDAQ: BOLD) and Astellas Pharma Inc. recently announced that they have entered into a definitive agreement for Astellas to acquire Audentes at a price of US$60.00 per share in cash, representing a total equity value of approximately US$3 billion.
“Recent scientific and technological advances in genetic medicine have advanced the potential to deliver unprecedented and sustained value to patients, and even to curing diseases with a single intervention,” said Kenji Yasukawa, President and CEO, Astellas. “Audentes has developed a robust pipeline of promising product candidates which are complementary to our existing pipeline, including its lead program AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM). By joining together with Audentes’ talented team, we are establishing a leading position in the field of gene therapy with the goal of addressing the unmet needs of patients living with serious, rare diseases.”
Outlook Therapeutics, Inc. (NASDAQ: OTLK) recently announced that it has received agreement from the U.S. Food and Drug Administration (“FDA”) on three Special Protocol Assessments (“SPAs”) for three additional registration clinical trials for its ongoing Phase 3 program for ONS-5010, an investigational ophthalmic formulation of bevacizumab. The agreements reached with the FDA on these SPAs cover the protocols for NORSE 4, a registration clinical trial to treat BRVO, and NORSE 5 and NORSE 6, two registration clinical trials to treat DME. Outlook Therapeutics intends to initiate NORSE 4, 5 and 6 in 2020.
“We are very pleased to reach these agreements with the FDA so that we can continue to build clinical data for additional retinal indications beyond wet AMD for ONS-5010,” said Lawrence A. Kenyon, President, Chief Executive Officer and Chief Financial Officer. “Anti-VEGF therapy has been the standard of care for years within the retinal community for wet AMD, DME and BRVO, and we hope that ONS-5010 will become the first FDA-approved bevacizumab indicated for these diseases. An on-label bevacizumab would enable clinicians to treat their retinal patients with a well-established therapy, and FDA approval would ensure sterility, potency and accurate, safe syringes for intravitreal injections.”
Axsome Therapeutics, Inc. (NASDAQ: AXSM) recently announced that AXS-12 (reboxetine) met the prespecified primary endpoint and significantly reduced the number of cataplexy attacks as compared to placebo in patients with narcolepsy in the CONCERT Phase 2 trial. AXS-12 also significantly reduced excessive daytime sleepiness (EDS), and improved cognitive function, sleep quality and sleep-related symptoms.
Narcolepsy is a debilitating, neurological condition characterized by EDS and cataplexy, a sudden loss of muscle tone triggered by strong emotions. AXS-12 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of narcolepsy.
uniQure N.V. (NASDAQ: QURE) recently announced that the U.S. Patent and Trademark Office has issued U.S. Patent Number 10,465,180. This patent covers adeno-associated virus vectors comprising a nucleic acid encoding FIX-Padua, promoter sequences, transcription termination and control elements. This patent is uniQure’s third U.S patent, along with the previously issued U.S. Patent Number 9,982,248 and U.S. Patent Number 9,249,405, in a patent family that uniQure acquired in 2017 from the inventor, Professor Paolo Simioni, a renowned hemophilia expert at the University of Padua, Italy.
“uniQure continues to build a significant intellectual property portfolio, including its FIX-Padua patents,” said David Cerveny, chief legal officer at uniQure. “These latest claims broaden the scope of uniQure’s FIX-Padua patent protection as they relate to any AAV comprising a nucleic acid encoding FIX-Padua together with promoter sequences, transcription termination and control elements. We believe this patent strengthens our intellectual property portfolio and adds further protection to the use of the FIX-Padua in AAV gene therapy to treat hemophilia B.”
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