On December 21, 2020 Equillium, Inc. (Nasdaq: EQ) a clinical-stage biotechnology company developing itolizumab to treat severe autoimmune and inflammatory disorders, reported the appointment of Dolca Thomas, M.D., as its executive vice president of research and development and chief medical officer (Press release, Equillium, DEC 21, 2020, View Source [SID1234573158]). Dr. Thomas joins Equillium from Principia Biopharma (recently acquired by Sanofi) where she was chief medical officer focused on developing treatments for immune-mediated diseases.

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"Equillium has made tremendous progress in 2020 and is now at a critical juncture as we begin to strategically outline more advanced development of itolizumab," said Bruce Steel, chief executive officer at Equillium. "Dolca’s significant track record of success and broad experience in executing late-stage programs, specifically in immunology, comes to Equillium at an important time. With several key readouts expected over the next twelve months, as well as interactions with the U.S. Food and Drug Administration that will help guide the future of our lead program in acute graft versus host disease, her expertise will come to bear immediately. I’d also like to take this opportunity to thank Dr. Krishna Polu, our departing chief medical officer, who contributed significantly to advancing our clinical programs and building our experienced research and development team; we wish Krishna well as he transitions to a new role in venture capital."

"I’m thrilled to join Equillium at such an exciting time and to advance the development of itolizumab, a highly novel drug targeting the CD6-ALCAM co-stimulatory signaling pathway. Modulating this biology may potentially have therapeutic effect in a number of immuno-inflammatory diseases beyond the current pipeline," said Dr. Thomas. "I look forward to guiding itolizumab’s path to registration in acute graft versus host disease, leading our clinical research efforts in lupus/lupus nephritis and uncontrolled asthma, and building a pipeline where we can have the most profound effect on the lives of patients."

Dr. Thomas brings almost two decades of industry and medical experience with strategic and operational responsibility for clinical development, pharmacovigilance, safety and medical affairs of approximately two dozen pharmaceutical product candidates. Prior to her position as chief medical officer at Principia, Dr. Thomas was vice president and global head of translational medicine for immunology, inflammation, and infectious disease at Roche, where she was responsible for advancing multiple product candidates through clinical development. Prior to Roche, Dr. Thomas held roles of increasing responsibility at Pfizer, including vice president of clinical development and clinical immunophenotyping, and vice president and chief development officer of the biosimilars research and development unit where she was responsible for all stages of development of multiple assets. Dr. Thomas began her industry career at Bristol-Myers Squibb as director of global clinical development in immunology, where she was involved in the development and approval of belatacept, a novel therapeutic targeting the co-stimulatory pathway CD28.

Dr. Thomas received her medical degree from Cornell University and completed her residency in internal medicine, in addition to her post-doctoral training in nephrology and transplantation, at New York-Presbyterian Hospital, Weill Cornell Medical Center.

"Supporting the momentum we have achieved, we are continuing to build the company," continued Mr. Steel. "I am pleased to announce that we have recently added Michael Son, Ph.D., as vice president of regulatory affairs, Nelson Lugo as vice president of manufacturing, and Michael Moore as vice president of investor relations and corporate communications. Their leadership has already begun to pay dividends and we look forward to their continued support as we rapidly transition to later-stage clinical development."

Dr. Son joins Equillium from Allergan where he served as global regulatory affairs lead. At Equillium, Dr. Son will be responsible for a global regulatory strategy and execution across Equillium’s development programs to support regulatory approvals.

Mr. Lugo previously directed technical services and commercial contract manufacturing operations for U.S. and international drug substance and drug product process operations at AstraZeneca, and was vice president of manufacturing at Nielsen Biosciences. At Equillium, Mr. Lugo will oversee the CMC (chemistry manufacturing and controls), clinical production and commercial manufacturing operations.

Mr. Moore comes to Equillium with over 20 years of experience in investor relations and corporate communications, representing all niches of life sciences, at every stage of development. At Equillium, Mr. Moore will be responsible for leading the company’s communications strategy, corporate messaging and ongoing external communications with the financial community and other stakeholders.

On December 21, 2020 Legend Biotech Corporation (NASDAQ: LEGN) ("Legend Biotech"), a global clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, reported the initiation of a rolling submission of a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for ciltacabtagene autoleucel (cilta-cel), an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell therapy, for the treatment of adults with relapsed and/or refractory multiple myeloma (Press release, Legend Biotech, DEC 21, 2020, View Source [SID1234573179]).

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The submission is based on results from the pivotal Phase 1b/2 CARTITUDE-1 study which evaluated the efficacy and safety of cilta-cel in the treatment of patients with relapsed and/or refractory multiple myeloma.1 The latest data from the study were recently presented (Abstract #177) at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

"Initiation of the BLA submission is an important milestone in advancing this therapy for patients with multiple myeloma who are heavily pretreated and in need of treatment options," said Ying Huang, PhD, CEO and CFO of Legend Biotech. "Together with our collaborator Janssen, we look forward to working with the FDA to fulfill this unmet medical need with the goal of making this breakthrough treatment available to patients and healthcare providers in the future."

Based on this submission, Legend Biotech also announced, according to the terms and conditions of an agreement with Janssen Biotech, Inc. (Janssen), achievement of a $75M milestone payment relating to the clinical development of cilta-cel. Janssen, Legend Biotech’s collaboration partner, initiated the submission of the BLA for cilta-cel. The FDA previously granted Breakthrough Therapy Designation (BTD) for cilta-cel and has agreed to a rolling review of the BLA in which completed portions of the application will be submitted and reviewed on an ongoing basis.

About CARTITUDE-1

CARTITUDE-1 (NCT03548207) is an ongoing Phase 1b/2, open-label, multicenter study evaluating the safety and efficacy of cilta-cel in adults with relapsed and/or refractory multiple myeloma, 99 percent of whom were refractory to the last line of treatment; 88 percent of whom were triple-class refractory (to at least 1 immunomodulatory drug [IMiD], 1 proteasome inhibitor [PI] and 1 anti-CD38 antibody).1

The primary objective of the Phase 1b portion of the study was to characterize the safety and confirm the dose of cilta-cel, informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2). The Phase 2 portion further evaluated the efficacy of cilta-cel with overall response rate as the primary endpoint.1

About Ciltacabtagene autoleucel (cilta-cel)

Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy, formerly identified as JNJ-4528 outside of China and LCAR-B38M CAR-T cells in China, that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed and/or refractory multiple myeloma and in earlier lines of treatment. The design consists of a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies. In December 2017, Legend Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. to develop and commercialize cilta-cel.

In addition to a Breakthrough Therapy Designation (BTD) granted in the U.S. in December 2019, cilta-cel received a PRIority MEdicines (PRiME) designation from the European Commission in April 2019, and a BTD in China in August 2020. In addition, Orphan Drug Designation was granted for cilta-cel by the U.S. FDA in February 2019, and by the European Commission in February 2020.

About Multiple Myeloma

Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.2 Although treatment may result in remission, unfortunately, patients will most likely relapse. 3 Relapsed myeloma is when the disease has returned after a period of initial, partial or complete remission and does not meet the definition of being refractory.4 Refractory multiple myeloma is when a patient’s disease is non-responsive or progresses within 60 days of their last therapy.5,6 While some patients with multiple myeloma have no symptoms until later stages of the disease, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.7 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and few treatment options.8

On December 21, 2020 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported receipt of U.S. Food and Drug Administration (FDA) permission for a Phase 1 study to proceed under the Company’s Investigational New Drug application (IND) for ON 123300, a proprietary, differentiated, first-in-class multi-kinase inhibitor (Press release, Onconova, DEC 21, 2020, View Source [SID1234573159]).

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"We are grateful to receive this timely, favorable response from the FDA to initiate a Phase 1 trial with ON 123300," said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova. "We are advancing the process to secure Institutional Review Board approval, and affirm our expectation that the first patient will be enrolled during the first half of 2021."

The Phase 1 trial will be conducted in the U.S. and will assess the safety, tolerability and pharmacokinetics of ON 123300 administered orally as monotherapy at increasing doses starting at 40 mg daily or higher for consecutive 28-day cycles. The trial will enroll patients with relapsed/refractory advanced cancer, including but not limited to patients with HR+ HER2- metastatic breast cancer with clinical resistance to approved second-generation CDK4/6 inhibitors. Once the dose escalation phase of the trial is completed and the recommended Phase 2 dose is established, additional HR+ HER2- postmenopausal metastatic breast cancer patients resistant to approved second-generation CDK4/6 inhibitors will be enrolled. Additional patient cohorts are under consideration, including but not limited to patients diagnosed with advanced colorectal cancer, and non-Hodgkin’s lymphoma, in particular mantle cell lymphoma.

The design of this U.S. Phase 1 trial differs from the ongoing study with ON 123300 in China conducted by the Company’s partner HanX Biopharmaceuticals, Inc., which is dosing patients daily for 21 days. The HanX trial has enrolled four patients to date, has opened the second dosing cohort and is expected to continue enrolling patients with advanced cancer at two sites until the recommended Phase 2 dose is identified. Notably, of the three currently approved CDK4/6 inhibitors, two are approved for dosing in 21-day cycles and one is approved for dosing in a 28-day cycle. All three are blockbuster drugs marketed in HR+ HER2– metastatic breast cancer by well-known pharmaceutical companies, and all of these approved therapies require concomitant treatment with an aromatase inhibitor.

"Based on its differentiated mechanism of action, we believe that ON 123300 presents an innovative approach to study advanced cancers including in HR+ HER2- metastatic breast cancer that is or has become resistant to commercial CDK4/6 inhibitors. Beyond metastatic breast cancer, we believe that ON 123300 may present a novel approach to treating other cancers including mantle cell lymphoma, multiple myeloma, advanced colorectal cancer and hepatocellular carcinoma, as well as inoperable glioblastoma based on preclinical studies suggesting ON 123300 crosses the blood-brain barrier," added Richard Woodman, M.D, Chief Medical Officer.

About ON 123300

Onconova’s lead pipeline compound is the novel small molecule ON 123300, a proprietary, first-in-class multi-kinase inhibitor targeting tumor-driving kinases including CDK4/6 and ARK5. ON 123300 is reported to simultaneously inhibit both cell cycle and cellular energy metabolism through CDK4/6 and ARK5, respectively, and in vitro has been shown to be cytotoxic to cancer cells (killing the cancer cells). The current commercial CDK inhibitors are reported to be cytostatic (inhibiting the growth of cancer cells). With its differentiated mechanism of action, ON 123300 may present an innovative approach for treating solid tumors and hematologic malignancies that are refractory to or have become resistant to other CDK4/6 inhibitors. Based on experiments in preclinical models, ON 123300 exhibits single-agent cytotoxicity, and may have utility for certain types of cancers including breast cancer, non-Hodgkin’s lymphoma including mantle cell lymphoma, multiple myeloma, colorectal cancer, hepatocellular carcinoma, and inoperable glioblastoma.

On December 21, 2020 Exicure, Inc. (Nasdaq: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) constructs, reported that the U.S. Patent and Trademark Office has issued two new patents, No.10,792,251 (the ‘251 patent) and No. 10,837,018 (the ‘018 patent), and allowed U.S. patent application 14/907,430 (the ‘430 application), further strengthening the Company’s intellectual property position and coverage for the Company’s therapeutic product candidate, cavrotolimod (Press release, Exicure, DEC 21, 2020, View Source [SID1234573180]).

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The ‘018 patent and the ‘430 application, both titled, "Spherical Nucleic Acid-based Constructs as Immunostimulatory Agents for Prophylactic and Therapeutic Use," covers nanoparticles with a corona of CpG oligonucleotides and their methods for treating cancer.

The ‘251 patent, jointly owned by the Company and Northwestern University, titled, "Liposomal Particles, Methods of Making Same and Uses Thereof," is directed to liposomal nanoparticles with toll-like receptor 9 (TLR9) agonist oligonucleotides and methods for treating cancer.

"The two issued patents and the soon-to-be-issued patent demonstrate our commitment to developing cavrotolimod, which is currently in a Phase 2 clinical trial for the potential treatment of Merkel cell carcinoma and cutaneous squamous cell carcinoma," said David Giljohann, Chief Executive Officer of Exicure. "We are pleased with cavrotolimod’s development to date and excited about its potential to address unmet need for patients living with these rare forms of skin cancers."

On December 21, 2020 Propanc Biopharma, Inc. (OTC: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that the first granted patent for an important patent family (i.e., a set of patents filed in various countries to protect an invention) was received by the Company from the Australian Patent Office (Press release, Propanc, DEC 21, 2020, View Source [SID1234573160]). The granted patent protects proprietary claims, capturing methods and uses for pancreatic proenzymes to treat cancer, specifically, by targeting and eradicating cancer stem cells ("CSCs"). CSCs represent only a small fraction of the cancer cells within a tumor and can remain dormant for extended periods of time, thereby evading standard treatments like chemotherapy and radiotherapy that target dividing cells. Consequently, a priority for improving cancer treatment and reducing risk of cancer relapse is to develop new strategies that selectively target CSC eradication whilst sparing normal stem cells. This continues to be the focus of ongoing research, as the Company’s lead product candidate, PRP, advances towards clinical trials for the treatment of patients with advanced solid tumors.

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The granted patent, citing the novel CSC treatment method, is one of our four patent families, consisting of 65 patents either in force, or pending, and is the first to be granted covering a method of minimizing the progression of cancer in a patient by administering a therapeutically effective amount of the two proenzymes, trypsinogen and chymotrypsinogen, thereby preventing metastatic, or spreading cancer in the patient. As a result, examination of patent applications in a number of other jurisdictions can be expedited where the Australian claims will be utilized for supplementary examination.

"The advancement of this patent to grant status in Australia is a significant step forward for our intellectual property portfolio and represents a novel therapeutic approach for the treatment and prevention of metastatic cancer by targeting and eradicating cancer stem cells, which is the main cause of death for sufferers," said James Nathanielsz, Propanc’s Chief Executive Officer. "It is especially important to continue to expand and grow our intellectual property portfolio as we advance PRP to clinical trials."

"Our main point of difference from other CSC therapies is the ability of our technology to differentiate cancer stem cells back towards normal cell behavior, so they die naturally, rather than directly killing CSCs. This way, we selectively target CSCs, leaving healthy stem cells alone, which means PRP is less toxic compared to standard treatment approaches," said Dr Julian Kenyon, Propanc’s Chief Scientific Officer. "Expanding our intellectual property portfolio by patenting new inventions using a world first, novel proenzyme treatment approach to target CSCs builds confidence that we are on track with our research."