On February 8, 2021 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that the Agencja Badań Medycznych (The Medical Research Agency) a Polish state agency responsible for development of scientific research in the field of medical and health sciences, awarded a grant equivalent to $1.5 million USD to the Maria Sklodowska-Curie National Research Institute to fund a Phase 1B/2 clinical trial of Annamycin for the treatment of soft tissue sarcoma (STS) lung metastases (Press release, Moleculin, FEB 8, 2021, View Source [SID1234574727]). The grant-funded clinical trial will be led by Prof. Piotr Rutkowski, MD, PhD, Head of Department of Soft Tissue/Bone Sarcoma and Melanoma at the Maria Sklodowska-Curie National Research Institute of Oncology in Warsaw, Poland.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Prof. Piotr Rutkowski will be assisted in part, by WPD Pharmaceuticals in Poland, a licensee of Annamycin, who will provide support in preparation for and conduct of the clinical trial, which is expected to begin this year. As a part of the collaboration between Moleculin and Prof. Rutkowski, Moleculin will be supplying the drug product necessary for the clinical trial, but Moleculin will not participate in conducting the clinical trial. This trial is independent from and will be in addition to the US clinical trial Moleculin is planning to conduct with Annamycin in sarcoma lung metastases.

"There is a significant unmet need for improved treatments for patients with sarcoma lung metastases," commented Prof. Piotr Rutkowski of Maria Sklodowska-Curie National Research Institute of Oncology, "so we are excited to begin this trial. Although this is considered a rare disease, there are no other clinical trials of this kind currently active in Poland, so it’s a tremendous opportunity for our patients."

"Prof. Rutkowski’s trial is a key element in a collaboration between teams in the US and Poland," added Walter Klemp, Chairman and CEO of Moleculin. "We are hopeful that data from the US and Poland can be combined to identify the potential for Annamycin to treat lung metastases."

Soft tissue sarcomas are the most common form of sarcoma, accounting for an estimated 130,000 incident cases per year worldwide. While many sarcomas can be addressed through surgical removal, it is estimated that as many 20% to 50% of STS sarcomas will eventually metastasize to the lungs, where treatment can become more challenging.

Once metastasized to the lungs, if tumors cannot be surgically removed, the primary chemotherapy regimen is the anthracycline doxorubicin (also known as Adriamycin). While 10% to 30% of patients with sarcoma lung metastases may initially respond to doxorubicin, most will relapse leaving the majority of these patients without an alternative chemotherapy. Treatment options are further limited because of the inherent cardiotoxicity of currently approved anthracyclines, including doxorubicin, which limits the amount of anthracycline that can be given to patients.

Annamycin is a "next generation" anthracycline that has recently been shown in animal models to accumulate in the lungs at up to 34 times the level of doxorubicin. Importantly, Annamycin has also demonstrated a lack of cardiotoxicity in recently conducted human clinical trials for the treatment of acute myeloid leukemia, so the use of Annamycin may not face the same dose limitations imposed on doxorubicin.

On February 8, 2021 Elevar Therapeutics, Inc. ("Elevar"), a fully integrated biopharmaceutical company built on the promise of elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, reported that the U.S. Food and Drug Administration (FDA) has granted rivoceranib (apatinib) with orphan drug designation for the treatment of adenoid cystic carcinoma (ACC), a rare form of cancer that most commonly develops in the salivary glands or other regions of the head and neck (Press release, LSK BioPharma, FEB 8, 2021, View Source [SID1234574725]). Approximately 1,200 new cases of ACC are diagnosed each year in the United States.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The FDA’s Office of Orphan Drug Products grants orphan status to medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S.

"We are pleased by the FDA’s recognition of the critical need to develop treatments for adenoid cystic carcinoma (ACC) given the progressive nature of the disease,’ said Alex Kim, chief executive officer of Elevar. "The orphan drug designation provides further momentum for the rivoceranib development program, which we are committed to advancing as quickly as possible for patients in need."

ACC is considered a slow growing but relentless cancer that is characterized by nerve invasion and metastases. Although good local control is usually achieved by resection of the primary tumor and adjuvant radiation therapy, more than half of patients eventually have recurrent and/or metastatic disease. Currently, no curative treatments are available for these patients which underscores the need for effective new therapies.

"With no approved treatments for adenoid cystic carcinoma (ACC), a significant unmet need remains for therapies that slow or stop this relentless disease," said Mark Gelder, M.D., chief medical officer of Elevar. "The orphan drug designation from the FDA reinforces the urgency of Elevar’s work with rivoceranib in ACC and our commitment to improving treatment experiences and outcomes for patients who have limited or inadequate therapeutic options."

In November 2020, Elevar announced that the Company met its initial 55 patient enrollment target for its Phase 2, open-label, multicenter, clinical trial of rivoceranib in patients with recurrent or metastatic ACC one year ahead of schedule. The Company also announced increasing the target size of the trial to approximately 72 patients to further explore the potential clinical benefit of rivoceranib in this patient population.

About Rivoceranib (apatinib)

Rivoceranib is the first small-molecule tyrosine kinase inhibitor (TKI) to be approved in gastric cancer (China, Dec 2014). It has been granted Orphan Drug designation in the U.S., Europe and South Korea and has been clinically tested in over 1,000 patients worldwide. Rivoceranib acts by inhibiting angiogenesis, a critical process in cancer growth and proliferation. Specifically, rivoceranib potently and selectively inhibits VEGFR-2 which mediates the primary pathway for tumor-mediated angiogenesis. As a best-in-class therapeutic known for its safety and tolerability, Elevar believes rivoceranib has the potential to significantly improve clinical outcomes in combination with chemotherapeutics and immunotherapy, as well as for maintenance therapy. Elevar Therapeutics is developing rivoceranib for the treatment of patients with gastric cancer, colorectal cancer, hepatocellular carcinoma, and adenoid cystic carcinoma.

On February, 2021 Blue Earth Diagnostics, a Bracco company and recognized leader in the development and commercialization of innovative PET radiopharmaceuticals, reported presentations on Axumin (fluciclovine F 18) at the upcoming ASCO (Free ASCO Whitepaper) 2021 Genitourinary Cancers Symposium (ASCO GU), from February 11 to 13, 2021, to be held in a virtual format (Press release, Blue Earth Diagnostics, FEB 8, 2021, View Source [SID1234574724]). Details of selected oral poster presentations are listed below.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

NOTE: Axumin (fluciclovine F 18) injection is FDA-approved for PET imaging in men with suspected prostate cancer recurrence based on elevated blood prostate specific antigen (PSA) levels following prior treatment. Axumin is a registered trademark of Blue Earth Diagnostics, Ltd., or its related companies. All other marks are the property of their respective owners.

Highlighted Axumin (Fluciclovine F 18) Scientific Presentations

All ASCO (Free ASCO Whitepaper) GU presentations are available beginning Thursday, February 11, 2021, at 8 a.m. ET.

Track: Prostate Cancer – Advanced

Title: Diagnostic utility of (18)f-fluciclovine positron emission tomography (FACBC) in biochemically recurrent (BCR) prostate cancer (PCa) based on prior primary treatment modality for localized disease and the impact of FACBC findings on treatment selection.

Author(s) Marina N. Sharifi, Petra Lovrec, Jens C. Eickhoff, Aria Kenarsary, David F. Jarrard, John Floberg, Steve Y. Cho, Christos Kyriakopoulos, Hamid Emamekhoo

Session: Poster session

Abstract: 34

Track: Prostate Cancer – Advanced

Title: Use of 18F-fluciclovine PET/CT (FluPET) for prostate cancer (PC): Initial results from a prospective registry at a tertiary academic center.

Author(s): Risa L. Wong, Heather H. Cheng, Sarah K. Holt, Nathan Conrad, Hannah Loesch, Sara Fernandez, Rameen Sahoo, Zoya Bauer, Andrea Toulouse, Petros Grivas, Todd Yezefski, Kenneth J. Russell, Jonathan L. Wright, Michael T. Schweizer, Robert B. Montgomery, Jean H. Lee, Delphine L. Chen, Jing Zeng, Daniel W. Lin, Evan Y. Yu

Session: Poster session

Abstract: 29

Track: Trials in Progress – Advanced Prostate Cancer

Title: 18F-fluciclovine positron emission tomography (PET) in metastatic castration-resistant prostate cancer (mCRPC) treated with abiraterone acetate.

Author(s): Pedro C. Barata, Charlotte Manogue, Ellen Jaeger, Malcolm Light, Patrick Cotogno, Elisa M. Ledet, Janeiro Goffin, Jodi L. Layton, Brian E. Lewis, A. O. Sartor

Session: Poster session

Abstract: TPS171

Blue Earth Diagnostics invites participants at the ASCO (Free ASCO Whitepaper) 2021 Genitourinary Cancers Symposium (ASCO GU) to attend the presentations above and to visit Blue Earth Diagnostics’ commercial exhibit in the Virtual Exhibit hall. For full session details and scientific presentation listings, please see the ASCO (Free ASCO Whitepaper) GU online program here.

INDICATION AND IMPORTANT SAFETY INFORMATION ABOUT AXUMIN
INDICATION

Axumin (fluciclovine F 18) injection is indicated for positron emission tomography (PET) imaging in men with suspected prostate cancer recurrence based on elevated blood prostate specific antigen (PSA) levels following prior treatment.

IMPORTANT SAFETY INFORMATION

Image interpretation errors can occur with Axumin PET imaging. A negative image does not rule out recurrent prostate cancer and a positive image does not confirm its presence. The performance of Axumin seems to be affected by PSA levels. Axumin uptake may occur with other cancers and benign prostatic hypertrophy in primary prostate cancer. Clinical correlation, which may include histopathological evaluation, is recommended.
Hypersensitivity reactions, including anaphylaxis, may occur in patients who receive Axumin. Emergency resuscitation equipment and personnel should be immediately available.
Axumin use contributes to a patient’s overall long-term cumulative radiation exposure, which is associated with an increased risk of cancer. Safe handling practices should be used to minimize radiation exposure to the patient and health care providers.
Adverse reactions were reported in ≤ 1% of subjects during clinical studies with Axumin. The most common adverse reactions were injection site pain, injection site erythema and dysgeusia.
To report suspected adverse reactions to Axumin, call 1-855-AXUMIN1 (1-855-298-6461) or contact FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

On February 8, 2021 Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, reported that it will issue financial results for its fiscal quarter ended December 31, 2020 on Thursday, February 11, 2021 (Press release, Anavex Life Sciences, FEB 8, 2021, View Source [SID1234574722]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Management will host a conference call on Thursday, February 11, 2021 at 4:30 pm Eastern Time to review financial results and provide an update on its clinical programs and corporate highlights. Following management’s formal remarks, there will be a question-and-answer session with equity analysts.

Conference Call / Webcast Information:

The live webcast of the conference call can be accessed online at View Source

To join the conference call, live via telephone, interested parties within the U.S. should dial, toll-free, 1 (866) 451-7964 and international callers should dial 1 (847) 944-7134. Please use confirmation number 50097999, followed by the pound sign (#).

A replay of the conference call will also be available on www.anavex.com.

On February 8, 2021 Novartis reported that asciminib – a novel investigational treatment specifically targeting the ABL myristoyl pocket (STAMP) – has been granted Breakthrough Therapy designation (BTD) by the US Food and Drug Administration (FDA) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs) (Press release, Novartis, FEB 8, 2021, View Source [SID1234574721]). Asciminib was also granted BTD for the treatment of adult patients with Ph+ CML in CP harboring the T315I mutation.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Despite tremendous advances in CML treatment over the past few decades, some of these pre-treated patients struggle to meet treatment goals due to resistance and intolerance18-23.
With few remaining treatment options, patients in later lines of care may be at risk of progression3-9.

These FDA designations, which may allow for an expedited development and review of asciminib, were based on:

The pivotal, Phase III ASCEMBL trial, where asciminib was compared to Bosulif (bosutinib)* in patients with Ph+ CML in CP previously treated with two or more TKIs1,2

A Phase I trial that included patients with Ph+ CML, some of them harboring the T315I mutation24
Data from these trials were shared at the 2020 Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), and details on positive findings can be found here.

The FDA previously granted Fast Track designation to asciminib, and Novartis plans for a submission in the first half of 2021 for review under the FDA Oncology Center of Excellence Real-Time Oncology Review program.

About asciminib (ABL001)
Asciminib (ABL001) is an investigational treatment specifically targeting the ABL myristoyl pocket (STAMP)11-17. As a STAMP inhibitor, asciminib is being studied in patients with chronic myeloid leukemia (CML) who experience resistance or intolerance to two or more tyrosine-kinase inhibitors (TKIs), and in several clinical trials in hopes of helping patients across multiple treatment lines of CML11-17, 25-32.

About ASCEMBL
ASCEMBL is the first head-to-head clinical trial in chronic myeloid leukemia using a second-generation tyrosine-kinase inhibitor (TKI) as a comparator. As a Phase III, multicenter, open-label, randomized study, ASCEMBL was designed to evaluate superiority in major molecular response rate at 24 weeks of the oral investigational treatment asciminib (ABL001) versus bosutinib in patients with Philadelphia-chromosome positive CML in chronic phase previously treated with two or more TKIs2. Patients with failure or intolerance to the most recently administered TKI therapy were included in the trial2.