On September 27, 2022 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a pre-commercial-stage biopharmaceutical company focused on oncology, reported its U.S. commercialization plan for APHEXDA (Motixafortide) in stem cell mobilization for autologous bone marrow transplantation for multiple myeloma patients (Press release, BioLineRx, SEP 27, 2022, View Source [SID1234621452]). If approved, the Company intends to commercialize APHEXDA in the U.S. independently in order to accelerate its availability to patients and to maximize the value of this innovative therapeutic candidate. To lead its U.S. operations and drive commercial strategy, the Company has appointed commercial product veteran Holly May to the role of President, BioLineRx USA.

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In the U.S., autologous stem cell transplants for patients with multiple myeloma and other conditions are highly concentrated within academic and regional centers. To support a robust commercial launch, the Company will employ a small and targeted sales force to support outreach to this well-defined community.

"We are excited to announce our plan to commercialize Motixafortide, now known by its FDA approved trade name APHEXDA, independently in the U.S., assuming FDA approval next year," said Philip Serlin, Chief Executive Officer of BioLineRx. "Our approach ensures focused outreach to transplant centers and enhanced value for the Company over other potential commercialization approaches examined. Since the beginning of 2022, we have been advancing key pre-launch activities. Our progress, together with our recent financings, puts us in an ideal position to efficiently build the additional infrastructure and targeted sales team to ensure the rapid uptake of APHEXDA."

"Our independent market research suggests that the U.S. market for mobilization agents used in stem cell transplants is approximately $360 million annually and growing," said Holly May, President, BioLineRx USA. "Given the totality of clinical and pharmacoeconomic data that we have compiled to date, we believe APHEXDA, if approved, can quickly become part of a new standard of care, allowing us to capture a significant share of this opportunity. Our U.S. commercial team is actively engaged in launch preparedness and excited about the potential of bringing this important therapeutic candidate to patients."

The Company recently announced that it submitted its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Motixafortide in stem cell mobilization for autologous bone marrow transplantation for multiple myeloma patients.

The NDA submission is based on the overwhelmingly positive top-line results from BioLineRx’s GENESIS Phase 3 trial of Motixafortide on top of G-CSF (versus placebo on top of G-CSF) in stem cell mobilization for autologous bone marrow transplantation in multiple myeloma patients. The study met all primary and secondary endpoints with a very high degree of statistical significance (p<0.0001). The combination was also found to be safe and well tolerated.

Investor and Key Opinion Leader Webinar
BioLineRx will provide more detail around its U.S. launch plans during an Investor and Key Opinion Leader Webinar that is occurring tomorrow, Wednesday, September 28, at 9:00 a.m. EDT.

The webinar will feature a presentation by Dr. John F. DiPersio, Chief of the Division of Oncology, Washington University School of Medicine, St. Louis, and lead investigator of BioLineRx’s GENESIS Phase 3 clinical study (the basis for the Company’s recently submitted New Drug Application), who will highlight the unmet need and current treatment landscape for the mobilization of stem cells (SCM) for multiple myeloma patients undergoing autologous stem cell transplantation.

Following Dr. DiPersio’s presentation, Lissa Gray, RN, who heads BioLineRx’s patient advocacy program, will moderate a panel discussion between an apheresis nurse, a multiple myeloma patient who underwent apheresis, and her caretaker, to provide a detailed assessment of the current treatment experience.

Holly May, President of BioLineRx USA, will then provide insight into the SCM market opportunity, as well as expand upon the Company’s plans to commercialize APHEXDA independently in the U.S., if approved.

Interested parties may register for the webinar here.

A replay of the webinar will be available on the Company’s Investor Relations page approximately two hours after the event’s completion. The webinar replay will be available until October 31, 2022.

About the GENESIS Trial
The GENESIS trial (NCT03246529) was initiated in December 2017. GENESIS was a randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of Motixafortide and G-CSF, compared to placebo and G-CSF, for the mobilization of hematopoietic stem-cells for autologous transplantation in multiple myeloma patients. The primary objective of the study was to demonstrate that only one dose of Motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize ≥ 6 million CD34+ cells in up to two apheresis sessions. A key secondary objective of the study was to demonstrate that only one dose of Motixafortide on top of G-CSF is superior to G-CSF alone in the ability to mobilize ≥ 6 million CD34+ cells in only one apheresis session. In this regard, ~90% of patients in the GENESIS study went directly to transplantation after mobilizing the optimal number of stem cells following only one administration of Motixafortide on top of G-CSF and in only one apheresis session, compared to less than 10% of those receiving G-CSF alone. Additional objectives included time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters.

About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that affects some white blood cells called plasma cells, which are found in the bone marrow. When damaged, these plasma cells rapidly spread and replace normal cells in the bone marrow with tumors. In 2022, it is estimated that more than 34,000 people will be diagnosed with multiple myeloma, and more than 12,000 people will die from the disease in the U.S. While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.

About Autologous Stem Cell Transplantation
Autologous stem cell transplantation (ASCT) is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma. In the U.S., nearly 15,000 ASCTs are performed each year with the majority in patients with multiple myeloma. The current standard of care includes the administration of 5-8 daily doses of granulocyte colony stimulating factor (G-CSF), with or without 1-4 doses of plerixafor, and the performance of 1-4 apheresis sessions. For patients unable to mobilize sufficient numbers of cells for harvesting during this primary mobilization phase, rescue therapy is carried out, consisting of 1-4 additional doses of plerixafor on top of G-CSF, and the performance of an additional number of apheresis sessions as necessary. In light of this, an agent with superior mobilization activity may significantly reduce the mobilization and harvesting burden and associated risks of the ASCT process and lead to significant clinical and resource benefits.

On September 27, 2022 Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare tumors and aggressive cancers reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for AL102 for the treatment of progressing desmoid tumors. AL102 is a potent, selective, oral gamma-secretase inhibitor (Press release, Ayala Pharmaceuticals, SEP 27, 2022, View Source [SID1234621451]).

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"We are pleased to receive FDA Fast Track designation for AL102 in progressing desmoid tumors, which we believe reinforces the large unmet medical need for patients with this serious disease. This designation holds important advantages that may expedite the development and regulatory review of AL102," said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. "We are very encouraged by the emerging body of clinical data supporting AL102 and, if approved, believe that this product could have a meaningful impact on patients’ lives."

The FDA grants Fast Track designation to facilitate development and expedite the review of therapies with the potential to treat a serious condition where there is an unmet medical need. A therapeutic that receives Fast Track designation can benefit from early and frequent communication with the agency, in addition to a rolling submission of the marketing application, with potential pathways for expedited approval that have the objective of getting important new therapies to patients more quickly.

AL102 is being evaluated in the ongoing RINGSIDE pivotal Phase 2/3 clinical trial in desmoid tumors. Positive interim results from Part A, the Phase 2 segment of this study, were presented at ESMO (Free ESMO Whitepaper) 2022, showing efficacy across all cohorts, with early tumor responses that deepened over time. AL102 was well tolerated. The company has initiated Part B of RINGSIDE (Phase 3), and is enrolling patients in an open label extension study.

About the RINGSIDE study
The RINGSIDE pivotal Phase 2/3 study is a randomized global multi-center trial. Part A of the study is evaluating the efficacy, safety, tolerability, and tumor volume by MRI after 16 weeks of AL102 in patients with desmoid tumors. It enrolled 42 patients and is evaluating 3 doses of AL102. Patients who participated in Part A are eligible to enroll into an open-label extension study at the Part B selected dose of 1.2 mg daily, and long-term efficacy and safety will be monitored.

Part B of the study, the Phase 3 segment, has been initiated. This is a double-blind, placebo-controlled segment enrolling up to 156 patients with progressive disease, comparing AL102 at 1.2 mg once daily to placebo. The primary endpoint for Part B is progression-free survival (PFS) with secondary endpoints including objective response rate (ORR), duration of response (DOR), tumor volume reduction, and patient-reported Quality of Life (QOL) measures. For more information on the RINGSIDE Phase 2/3 study with AL102 for the treatment of desmoid tumors, please visit ClinicalTrials.gov and reference Identifier NCT04871282 (RINGSIDE).

About Desmoid Tumors
Desmoid tumors also called aggressive fibromatosis or desmoid-type fibromatosis, are rare connective tissue tumors that typically arise in the upper and lower extremities, abdominal wall, head and neck area, mesenteric root, and chest wall with the potential to arise in additional parts of the body. Desmoid tumors do not metastasize, but often aggressively infiltrate neurovascular structures and vital organs. People living with desmoid tumors are often limited in their daily life due to chronic pain, functional deficits, general decrease in their quality of life and organ dysfunction. Desmoid tumors have an annual incidence of approximately 1,700 patients in the United States and typically occur in patients between the ages of 15 and 60 years. They are most commonly diagnosed in young adults between 30-40 years of age and are more prevalent in females. Today, surgery is no longer regarded as the cornerstone treatment of desmoid tumors due to a high rate of recurrence post-surgery and there are currently no FDA-approved systemic therapies for the treatment of unresectable, recurrent or progressive desmoid tumors.

On September 27, 2022 Avid Bioservices, Inc. (NASDAQ:CDMO), a dedicated biologics contract development and manufacturing organization (CDMO) working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, reported that the company will participate in the RBC Global CDMO Conference (Press release, Avid Bioservices, SEP 27, 2022, View Source [SID1234621450]). Nick Green, president and chief executive officer, will be the featured speaker in a fireside chat at the conference, which will take place October 3-4, 2022.

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Details of the company’s participation are as follows:

RBC Global CDMO Conference
Conference Date: October 3-4, 2022
Fireside Chat Time/Date: 12:15 – 12:45 p.m. Eastern on Monday, October 3, 2022
Format: Virtual conference; webcast available
To listen to the webcast of the RBC fireside chat please visit: View Source

On September 27, 2022 Race Oncology Limited ("Race") reported that it has contracted the Monash Fragment Platform (MFP) at Monash University to complete a fragment-based screening program aimed at discovering novel drugs that inhibit the m6 A RNA demethylases FTO and ALKBH5 (Press release, Race Oncology, SEP 27, 2022, View Source [SID1234621447]). Eminent medicinal chemist and Director of the MFP, Professor Martin Scanlon, will lead the project.

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All intellectual property developed in the project will be owned exclusively by Race.

RNA Epitranscriptomics Important scientific discoveries made over the last decade have identified dysregulation (loss of control) of RNA epigenetics (epitranscriptomics) as a key driver of cancer and other complex diseases1. Two of the major players in this dynamic regulatory system are the Fatso/FaT and Obesity associated (FTO) and ALKBH5 proteins. FTO and ALKBH5 are the only m6 A RNA demethylases found in humans and are major global regulators of the m6 A RNA levels in cells2.

Changes in the expression of FTO or ALKBH5 has a profound impact on cancer growth, spread and resistance to treatment. Inhibiting FTO or ALKBH5 activity is able to kill or slow the growth of a wide range of cancers, including leukaemia, breast, lung, ovarian, gastric, brain, melanoma, pancreatic, kidney and many more. Race Oncology’s most advanced asset Zantrene (bisantrene dihydrochloride) is a potent inhibitor of FTO (IC50 142nM)3 and is the only m6 A RNA demethylase inhibitor and RNA epitranscriptomic drug in the clinic.

NMR Fragment Based Drug Screening To build on our success in the RNA epitranscriptomics space, Race is aiming to discover new, potent and selective inhibitors of FTO and ALKBH5 for use in cancer and other indications. To advance this program, Race has contracted the Monash Fragment Platform to complete a fragment screening campaign using the latest techniques in Race Oncology Ltd ABN 61 149 318 749 Registered office: L36, 1 Macquarie Place, Sydney NSW 2000 www.raceoncology.com nuclear magnetic resonance spectroscopy (NMR). In this approach, a library of diverse small molecules (‘fragments’) will be tested by NMR for their ability to bind to the FTO and ALKBH5 proteins. Molecules that are found to bind to the proteins will be transformed into drug leads and ultimately clinical drug candidates in a follow-up medicinal chemistry campaign. This screening program will start immediately with results reported over the coming 12 months. The total cost of the project is $286,786 and is expected to be eligible for the ATO 43.5% R&D Tax Rebate.

Race Principal Scientist, Professor Mike Kelso said: "Race is extremely pleased to be working with Professor Scanlon and his experienced team on this important project. The discovery and patenting of new FTO and ALKBH5 inhibitors will greatly strengthen Race’s drug development pipeline and add valuable assets to our expanding IP portfolio. Drawing on my career in medicinal chemistry, I relish the downstream challenge of evolving molecules discovered in this campaign into clinic-ready FTO and ALKBH5 inhibitors as innovative new treatments for cancer and other diseases." Professor Scanlon said: "Developing next-generation medicines requires outstanding discovery science. The Monash Fragment Platform was established to help researchers accelerate this long and complex journey. We are delighted to be working with Race to use our established Fragment-Based Screening technology to identify novel inhibitors for FTO and ALKBH5."

Race Chief Executive Officer, Phil Lynch said: "Addition of new FTO inhibitors to Race’s drug pipeline creates major value as it grows Race ‘Beyond Zantrene’ and positions us as a world leading RNA epitranscriptomics pharma company." 1. Sun, T., Wu, R. & Ming, L. The role of m6A RNA methylation in cancer. Biomed Pharmacother 112, 108613 (2019). 2. You, Y. et al. Recent Advances of m6A Demethylases Inhibitors and Their Biological Functions in Human Diseases. Int J Mol Sci 23, 5815 (2022). 3. Su R et al. Targeting FTO Suppresses Cancer Stem Cell Maintenance and Immune Evasion. Cancer Cell 38:1–18 (2020).-ENDSAbout Professor Martin Scanlon and the Monash Fragment Platform The Monash Fragment Platform (MFP) is part of the Monash University Technology Research Platform Network (MTRP). The aim of MFP is to facilitate access to the transformative technology of Fragment-Based Drug Design through its multidisciplinary technology network. MFP supports Australian academic groups and pharma companies to accelerate their drug discovery R&D programs using a fee-for-service model. Martin Scanlon is Scientific Director of the MFP and Professor of Medicinal Chemistry at the Monash Institute of Pharmaceutical Sciences. He has published over 100 papers in highranking international journals, and has led more than 30 successful fragment-based discovery campaigns at MFP for academic and industry clients since its inception in 2015.

On September 27, 2022 Quest Diagnostics Incorporated (NYSE: DGX), the world’s leading provider of diagnostic information services, reported that it will report third quarter 2022 financial results on Thursday, October 20, 2022, before the market opens (Press release, Quest Diagnostics, SEP 27, 2022, View Source,-2022 [SID1234621446]). It will hold its quarterly conference call to discuss the results beginning at 8:30 a.m. Eastern Time on that day.

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The conference call can be accessed by dialing 888-455-0391 within the U.S. and Canada, or 773-756-0467 internationally, using the passcode: "7895081." The earnings release and live webcast will be posted on www.QuestDiagnostics.com/investor. The company suggests participants dial in approximately 10 minutes before the call.

A replay of the call may be accessed online at www.QuestDiagnostics.com/investor or by phone at 888-566-0462 for domestic callers or 203-369-3609 for international callers; no passcode is required. Telephone replays will be available from approximately 10:30 a.m. Eastern Time on October 20, 2022 until midnight Eastern Time on November 3, 2022.

Anyone listening to the call is encouraged to read the company’s periodic reports on file with the Securities and Exchange Commission, including the discussion of risk factors and historical results of operations and financial condition in those reports.