On May 17, 2022 CytRx Corporation (OTCQB: CYTR) ("CytRx" or the "Company"), a biopharmaceutical innovator focused on research and development of life-saving cancer therapeutics, reported to highlight the strategic purchase of arimoclomol by KemPharm, Inc. ("KemPharm") (Press release, CytRx, MAY 17, 2022, View Source [SID1234614766]).

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KemPharm is a specialty biopharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system ("CNS") diseases. KemPharm has announced a definitive agreement with Orphazyme A/S ("Orphazyme") to acquire arimoclomol, a first-in-class heat shock protein potentiator being developed as a treatment for Niemann-Pick disease type C ("NPC"). NPC is a rare neurodegenerative disease characterized by abnormal accumulation of cholesterol and lipids in neuronal tissues. Under the terms of the agreement, KemPharm will purchase substantially all of the assets and operations of Orphazyme, including arimoclomol, for a cash payment of USD $12.8 million. The transaction is expected to close on or before June 1, 2022.

CytRx licensed arimoclomol to Orphazyme for milestones and royalties on sales, and CytRx expects the terms of its license with Orphazyme to remain intact as the arimoclomol asset transfers to KemPharm.

In the press release issued by KemPharm, Travis Mickle, Ph.D., President and Chief Executive Officer of KemPharm stated, "The acquisition of arimoclomol aligns perfectly with our strategy to build KemPharm’s value via the advancement and commercialization of novel treatments that address rare CNS conditions, including our lead clinical candidate, KP1077 in idiopathic hypersomnia. We have carefully evaluated the CRL issued by the FDA and the minutes from the subsequent Type A meeting, as well as the data that has been generated from the development work performed to date. We believe the efficacy signal for arimoclomol in NPC is convincing and that there is a viable regulatory path that could enable a successful NDA resubmission. KemPharm has significant experience with challenging regulatory situations, having successfully led or participated in three FDA product approvals, two of which followed an initial CRL. We welcome the opportunity to work with the FDA on the resubmission of the NDA for arimoclomol in NPC, which we expect to file as early as the first quarter of 2023."

Stephen Snowdy, PhD, Chief Executive Officer of CytRx commented, "CytRx is very pleased to see that arimoclomol will enjoy the support and resources of KemPharm, and that KemPharm sees value in the human trials data collected on arimoclomol through the course of 10 Phase 1, four Phase 2 and three pivotal Phase 2/3 trials. We are especially pleased to see that KemPharm, with its experience in challenging regulatory submissions, plans to re-submit arimoclomol to the FDA as early as Q1 2023. With a mean age of death of 13 years, patients desperately need a solution to Niemann-Pick disease type C."

On May 17, 2022 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported its participation in the H.C. Wainwright Global Investment Conference 2022 (Press release, MEI Pharma, MAY 17, 2022, View Source [SID1234614765]). The company will present a company overview and business update available for on-demand listening starting Tuesday, May 24, 2022, 7:00 a.m. Eastern Time that will be archived for 90 days.

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The presentation can be accessed via the Events & Presentations page of the Investors section of MEI Pharma’s website at View Source An archived replay of the webcast will be available on MEI Pharma’s website for at least 30 days after the live event concludes.

On May 17, 2022 Imugene Limited (ASX:IMU), a clinical stage immuno-oncology company, and City of Hope, one of the largest cancer research and treatment organizations in the United States, reported that the first patient was dosed in a Phase 1 clinical trial evaluating the safety of novel cancer-killing virus CF33-hNIS VAXINIA when used in people with advanced solid tumors (Press release, City of Hope, MAY 17, 2022, View Source [SID1234614764]). The City of Hope-developed oncolytic virus has been shown to shrink colon, lung, breast, ovarian and pancreatic cancer tumors in preclinical laboratory and animal models.

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"Our previous research demonstrated that oncolytic viruses can stimulate the immune system to respond to and kill cancer, as well as stimulate the immune system to be more responsive to other immunotherapies, including checkpoint inhibitors," said Daneng Li, M.D., principal investigator and assistant professor of City of Hope’s Department of Medical Oncology & Therapeutics Research. "Now is the time to further enhance the power of immunotherapy, and we believe CF33-hNIS has the potential to improve outcomes for our patients in their battle with cancer."

Oncolytic virus therapy is made possible once viruses found in nature are genetically modified to infect, replicate in and kill cancer cells, while sparing healthy cells. While immune checkpoint inhibitors have been effective in certain cancers, patients often relapse and eventually stop responding to or develop resistance to this type of treatment. Early research shows oncolytic viruses can prime a person’s immune system and increase the level of PD-L1 in tumors, making immunotherapy more effective against cancer.

The multicenter Phase 1 trial will start by delivering a low dose of CF33-hNIS to cancer patients with metastatic or advanced solid tumors who have had at least two prior lines of standard of care treatment. The investigational treatment will be delivered either as an injection directly into tumors or intravenously.

Once patients in the single therapy group have been treated with the lowest doses of CF33-hNIS and acceptable safety has been demonstrated, certain new study participants will receive the experimental oncolytic virus in combination with the immunotherapy pembrolizumab, an engineered antibody that improves the immune system’s ability to fight cancer-causing cells. The study aims to recruit 100 patients across approximately 10 trial sites in the United States and Australia.

City of Hope exclusively licensed patent rights covering CF33 to Imugene Limited, a company developing novel therapies that activate the immune system against cancer. Imugene has given CF33-hNIS the name Vaxinia.

"Interestingly, the same characteristics that eventually make cancer cells resistant to chemotherapy or radiation treatment actually enhance the success of oncolytic viruses, such as CF33-hNIS," said Yuman Fong, M.D., the Sangiacomo Family Chair in Surgical Oncology at City of Hope and the key developer of the genetically modified virus. "We are hoping to harness the promise of viralogy and immunotherapy for the treatment of a wide variety of deadly cancers."

The clinical trial is titled "A Phase I, Dose Escalation Safety and Tolerability Study of VAXINIA (CF33-hNIS), Administered Intratumorally or Intravenously as a Monotherapy or in Combination with Pembrolizumab in Adult Patients with Metastatic or Advanced Solid Tumours (MAST)." The trial is anticipated to run for approximately 24 months and is funded from existing budgets and resources.

The U.S. component of the Phase 1 trial is conducted under the U.S. Food and Drug Administration (FDA) investigational new drug (IND) process following FDA IND clearance in December 2021. Site activation and patient recruitment is proceeding.

The first clinical institution in the U.S. to receive ethics approval is City of Hope, a world-renowned cancer research and treatment organization in Los Angeles. Additional clinical sites will be opened across the U.S. in 2022.

Imugene M.D. and CEO Leslie Chong said, "The dosing of the first patient in our VAXINIA study is a significant milestone for Imugene and clinicians faced with the challenge of treatment for metastatic advanced solid tumours. Professor Yuman Fong and the City of Hope team have provided outstanding research. In addition to the positive preclinical results, we’re incredibly eager to unlock the potential of VAXINIA and the oncolytic virotherapy platform."

About Daneng Li, M.D.

Daneng Li, M.D., is an assistant professor in the Department of Medical Oncology & Therapeutics Research at City of Hope who specializes in treating neuroendocrine tumors, liver tumors and gastrointestinal cancers. Li currently leads City of Hope’s liver tumors program and is also the co-director of the Neuroendocrine Tumour Program at City of Hope.

Li earned his undergraduate degree from The Ohio State University in Columbus, Ohio, where he graduated summa cum laude. He then went on to receive his medical doctorate from Weill Cornell Medical College in New York before pursuing an internship and residency in internal medicine at New York-Presbyterian Hospital/Weill Cornell Medical Center. He completed a hematology/oncology fellowship at Memorial Sloan-Kettering Cancer Center in New York City.

Board certified in internal medicine and medical oncology, Li serves on several national committees focused on his specialty tumor types. He has authored many publications in peer-reviewed literature and has presented his work nationally.

On May 17, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, reported that company management will present at the upcoming H.C. Wainwright Global Life Sciences Conference (Press release, Gamida Cell, MAY 17, 2022, View Source [SID1234614762]). A pre-recorded presentation will become available to registered conference attendees on May 24, 2022 at 7:00 a.m. ET. Management will discuss 2022 catalysts and potential milestones including the U.S. launch opportunity for omidubicel upon potential U.S. Food and Drug Administration approval, accelerating the development of its first-in-class NAM-enabled natural killer (NK) cell therapy candidate, GDA-201, as a potential new approach for patients with follicular and diffuse large B-cell lymphomas, and expansion of its NAM-enabled cell therapy pipeline with multiple next-generation, genetically engineered NK cells.

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A webcast of the presentation will be available on the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com, and will be available for at least 14 days following the event.

About Omidubicel

Omidubicel is an advanced cell therapy candidate under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the U.S. FDA and has also received Orphan Drug Designation in the U.S. and EU. Gamida Cell has completed an international, multi-center, randomized Phase 3 study (NCT0273029) evaluating the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing allogeneic bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. That study achieved its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in a patient’s recovery from a stem cell transplant. The Phase 3 study also achieved its secondary endpoints of reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. Gamida Cell initiated a rolling BLA submission for omidubicel in the first quarter of 2022 with full BLA submission on track for the second quarter of 2022. In 2019, approximately 8,000 patients who were 12 years old and up with hematologic malignancies underwent an allogeneic stem cell transplant. Unfortunately it is estimated that another 1,200 patients were eligible for transplant but could not find a donor source. Omidubicel has the opportunity, upon FDA approval to improve outcomes for patients based on transplanter feedback and increase access for patients to get to transplant. Omidubicel has the potential to treat approximately 2000 – 2500 patients each year in the U.S. For more information about omidubicel, please visit View Source

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About GDA-201

Gamida Cell applied the capabilities of its nicotinamide (NAM)-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy candidate for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells. There are approximately 40,000 patients with relapsed/refractory lymphoma in the E.U.5 and U.S. which is the patient population that will be studied in the GDA-201 Phase 1/2 clinical trial.

For more information about GDA-201, please visit View Source For more information on the Phase 1/2 clinical trial of GDA-201, please visit www.clinicaltrials.gov.

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About NAM Technology

Our NAM-enabling technology, supported by positive Phase 3 data, is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (Nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

On May 17, 2022 Gilead Sciences, Inc. (Nasdaq: GILD) and Kite, a Gilead Company, reported that more than 20 abstracts, including two oral presentations and four poster discussions, will be presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Gilead Sciences, MAY 17, 2022, View Source [SID1234614761]). These data highlight promising targets across diverse tumor types and blood cancers, including hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) metastatic breast cancer, metastatic triple-negative breast cancer (TNBC), large B-cell lymphoma (LBCL), myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and mantle cell lymphoma (MCL).

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"Gilead has built a diverse oncology pipeline guided by our strategic framework, with a focus on depth and breadth to address the greatest gaps in care for people with overlooked, underserved and difficult-to-treat cancers," said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences. "Anchored by our Trop-2 directed antibody-drug conjugate, CAR T-cell therapies and our investigational anti-CD47 antibody, these data reinforce the potential of our transformative science across multiple cancers."

Driving Scientific Innovation in Breast Cancers with High Unmet Need

A late-breaking presentation on the TROPiCS-02 study will examine the investigational use of Gilead’s antibody-drug conjugate in heavily pre-treated patients with HR+/HER2- unresectable locally advanced or metastatic breast cancer, a population with significantly limited treatment options following endocrine resistance. The final data from the landmark ASCENT study in second-line metastatic TNBC will also be presented.

Advancing an Industry-Leading CAR T-cell Therapy Portfolio

New analyses of the Phase 3 ZUMA-7 trial will highlight results in patients aged 65+, as well as data on pre-treatment tumor burden characteristics and clinical outcomes for the first and only CAR T-cell therapy approved for initial treatment of relapsed/refractory LBCL. Additional presentations include real-world outcomes by race and ethnicity for Kite’s CAR T-cell therapy in LBCL and longer-term data from the ZUMA-2 and ZUMA-3 studies assessing the durability of response to Kite’s CAR T-cell therapy in relapsed/refractory MCL and relapsed/refractory adult ALL, respectively.

"At Kite, our singular focus is on developing cell therapies to treat and potentially cure cancers," said Frank Neumann, MD, PhD, Kite’s Global Head of Clinical Development. "Our data at ASCO (Free ASCO Whitepaper) span multiple types of blood cancer and stages of treatment, reinforcing how our therapies are changing the way cancer is treated."

Furthering Potential Treatment Approaches in MDS and AML

Results of our Phase 1b study evaluating an investigational anti-CD47 antibody in combination with azacitidine in high-risk MDS and in TP53-mutant AML will be presented. MDS and AML are blood cancers which have seen limited therapeutic advancements in the past decade, and Gilead data being presented at ASCO (Free ASCO Whitepaper) will reinforce the potential of harnessing the innate immunity of macrophages.

Summary of Presentations

Accepted abstracts at the 2022 ASCO (Free ASCO Whitepaper) Annual Meeting include (all times CDT):

Abstract Disposition

Abstract Title

Breast Cancer

Abstract #LBA1001 (Oral Session)

Saturday, June 4

1:27 PM

Primary Results from TROPiCS-02: A Randomized Phase 3 Study of Sacituzumab Govitecan (SG) versus Treatment of Physician’s Choice (TPC) in Patients with Hormone Receptor-Positive/HER2-Negative (HR+/HER2-) Advanced Breast Cancer

Abstract #1071

Monday, June 6

8:00 AM

Sacituzumab Govitecan (SG) versus Treatment of Physician’s Choice (TPC) in Patients with Previously Treated Metastatic Triple-Negative Breast Cancer (mTNBC): Final Results from the Phase 3 ASCENT Study

Abstract #1076

Monday, June 6

8:00 AM

Exposure-Response Analyses of Sacituzumab Govitecan (SG) Efficacy and Safety in Patients with Metastatic Triple-Negative Breast Cancer (mTNBC)

Abstract #1075

Monday, June 6

8:00 AM

Real-World Treatment Patterns and Outcomes among 2nd Line (2L) and 3rd Line (3L) Metastatic Triple-Negative Breast Cancer (mTNBC) Patients in England Using the Cancer Analysis System (CAS)

Myelodysplastic Syndrome

Abstract #7017 (Poster Discussion)

Saturday, June 4

1:15 PM

Magrolimab in Combination with Azacitidine for Untreated Higher-Risk Myelodysplastic Syndromes (HR-MDS): 5F9005 Phase 1b Study Results

Abstract #7054

Saturday, June 4

8:00 AM

Impact of Magrolimab Treatment in Combination with Azacitidine on Red Blood Cells in Higher-Risk Myelodysplastic Syndrome (HR-MDS) Patients

ePublication #e19062

Clinical Outcomes Associated with Azacitidine Monotherapy for Treatment-Naïve Higher-Risk Myelodysplastic Syndrome: A Systematic Literature Review and Meta-Analysis

Acute Myeloid Leukemia

Abstract #7020 (Poster Discussion)

Saturday, June 4

1:15 PM

Tolerability and Efficacy of the First-in-Class Anti-CD47 Antibody Magrolimab Combined with Azacitidine in Frontline TP53m AML Patients: Phase 1b Results

ePublication #e19020

Treatment Outcomes for Newly Diagnosed, Untreated TP53-Mutated Acute Myeloid Leukemia: A Systematic Review and Meta-Analysis

B-cell Lymphomas

Abstract #7571 (Oral Session)

Friday, June 3

3:12 PM

Real-World Outcomes of Axicabtagene Ciloleucel (Axi-Cel) for the Treatment of Large B-cell Lymphoma (LBCL) by Race and Ethnicity

Abstract #7548

Saturday, June 4

8:00 AM

Clinical and Patient-Reported Outcomes (PROs) in a Phase 3, Randomized, Open-Label Study Evaluating Axicabtagene Ciloleucel (Axi-Cel) versus Standard-of-Care (SOC) Therapy in Elderly Patients with Relapsed/Refractory (R/R) Large B-cell Lymphoma (LBCL) (ZUMA-7)

Abstract #7565

Saturday, June 4

8:00 AM

Association of Pretreatment (Pretx) Tumor Characteristics and Clinical Outcomes Following Second-Line (2L) Axicabtagene Ciloleucel (Axi-Cel) versus Standard of Care (SOC) in Patients with Relapsed/Refractory (R/R) Large B-cell Lymphoma (LBCL)

Abstract #7567

Saturday, June 4

8:00 AM

Axicabtagene Ciloleucel (Axi-Cel) in Combination with Rituximab (Rtx) for the Treatment of Refractory Large B-cell Lymphoma (R-LBCL): Outcomes of the Phase 2 ZUMA-14 Study

Abstract #7555

Saturday, June 4

8:00 AM

Quality-Adjusted Time without Symptoms or Toxicities (Q-Twist) Analysis of ZUMA-7, a Randomized Controlled Trial of Axicabtagene Ciloleucel versus Standard of Care for Second-Line Large B-cell Lymphoma

Abstract #TPS7579

Saturday, June 4

8:00 AM

KITE-363: A Phase 1 Study of an Autologous Anti-CD19/CD20 Chimeric Antigen Receptor (CAR) T-Cell Therapy in Patients with Relapsed/Refractory (R/R) B-cell Lymphoma (BCL)

ePublication #e19558

Patient Preferences for Second-Line Treatment Options in Diffuse Large B-cell Lymphoma: A Discrete Choice Experiment

Mantle Cell Lymphoma

Abstract #7518 (Poster Discussion)

Saturday, June 4

3:00 PM

Three-Year Follow-Up of Outcomes with KTE-X19 in Patients with Relapsed/Refractory Mantle Cell Lymphoma in ZUMA-2

Acute Lymphoblastic Leukemia

Abstract #7010 (Poster Discussion)

Saturday, June 4

1:15 PM

Two-Year Follow-Up of KTE-X19, an Anti-CD19 Chimeric Antigen Receptor (CAR) T-Cell Therapy, in Adult Patients with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (R/R B-ALL) in ZUMA-3

Non-Small Cell Lung Cancer

Abstract #TPS9149

Monday, June 6

8:00 AM

EVOKE-01: A Phase 3 Study of Sacituzumab Govitecan (SG) versus Docetaxel in Patients with Non-Small Cell Lung Cancer (NSCLC) Progressing On or After Platinum-Based Chemotherapy and Checkpoint Inhibitors

Abstract #TPS9146

Monday, June 6

8:00 AM

EVOKE-02: A Phase 2 Study of Sacituzumab Govitecan Plus Pembrolizumab with or without Platinum Chemotherapy in First-Line Metastatic Non-Small Cell Lung Cancer (NSCLC)

Advanced Solid Tumors

Abstract #2566

Sunday, June 5

8:00 AM

Phase 1b Study of GS-3583, A Novel FLT3 Agonist Fc Fusion Protein, in Patients with