On March 29, 2022 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported that Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, will deliver a virtual company presentation at the Guggenheim 3rd Annual Genomic Medicines and Rare Disease Conference on Friday, April 1 at 8:00 a.m. ET. Gaurav Shah, M.D., Chief Executive Officer, will present at the 21st Annual Needham Virtual Healthcare Conference on Monday, April 11 at 10:15 a.m. ET (Press release, Rocket Pharmaceuticals, MAR 29, 2022, View Source [SID1234611138]).

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A live audio webcast of the presentations will be available under "Events" in the Investors section of the Company’s website at View Source The webcast replay will be available on the Rocket website following the conferences.

On March 29, 2022 Thermo Fisher Scientific reported that launched the CE-IVD marked Ion Torrent Genexus Dx Integrated Sequencer, an automated, next-generation sequencing (NGS) platform that delivers results in as little as a single day (Press release, Thermo Fisher Scientific, MAR 29, 2022, View Source [SID1234611136]). Designed for use in clinical laboratories, the fully validated system enables users to perform both diagnostic testing and clinical research on a single instrument.

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"With faster answers, the results can aid clinicians in their patient management decision making which may include therapeutic options."

"Next-generation sequencing has become an essential tool to bring the promise of precision medicine therapies to patients. With the automated, easy-to-use Genexus Dx Integrated Sequencer, any hospital – including regional and community hospitals – can bring NGS in-house, giving clinicians access to timely, comprehensive genomic profiling results," said Garret Hampton, president, clinical next-generation sequencing and oncology at Thermo Fisher Scientific. "With faster answers, the results can aid clinicians in their patient management decision making which may include therapeutic options."

In support of increasing physicians’ access to rapid NGS results, Thermo Fisher is also developing a complete sample-to-report diagnostic workflow and a portfolio of clinically validated assays, including those for comprehensive genomic profiling and hemato-oncology, on the Genexus System.

Thermo Fisher introduced the Ion Torrent Genexus System for research use only in 2019 as the first fully integrated NGS platform that delivers results in as little as 24 hours. The platform’s automated workflow minimizes user intervention and the potential for human error, making NGS accessible for all labs.

For more information on the Ion Torrent Genexus Dx Integrated Sequencer, please visit thermofisher.com/genexusDx.

On March 29, 2022 Australian biotech company Noxopharm (ASX:NOX) reported the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to Veyonda, its lead oncology drug candidate, for use in the treatment of soft tissue sarcoma (Press release, Noxopharm, MAR 29, 2022, View Source [SID1234611135]).

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The ODD program was established by the FDA to encourage development of safe and effective treatments of rare diseases and disorders that affect fewer than 200,000 people in the U.S. annually. Soft tissue sarcomas are rare but often fatal cancers — up to 50% of high-grade sarcoma patients develop metastases and die within 12 months.

"Only four of approximately 360 approved ODDs last year went to Australian companies, which demonstrates the high bar that is being set by the FDA," said Noxopharm CEO and Managing Director Gisela Mautner, M.D.-Ph.D., MPH. "It is pleasing that the Noxopharm application for Orphan Drug Designation was approved so quickly."

Noxopharm recently began its Phase 1 CEP-2 trial at City of Hope in Los Angeles for Veyonda, in combination with the chemotherapy drug doxorubicin, for first-line treatment of soft tissue sarcoma.

The ODD program provides a number of significant benefits, including:

A seven-year period of market exclusivity — the FDA will not approve a subsequent drug for the same use within this timeframe
Waiver of new drug application fees, valued at approximately $2.9 million in 2021
Opportunities for grant funding from the Office of Orphan Products Development
Regulatory guidance and assistance from the FDA for the drug development process
"This designation will allow us to lower current development costs and provide a future competitive and financial advantage of Veyonda," Dr. Mautner said. "With the ODD now secured, my team will be able to focus on moving our preclinical assets along the drug development process, while continuing to deliver on our clinical program plan."

On March 29, 2022 Aptevo Therapeutics Inc. (NASDAQ:APVO), a clinical-stage biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported that a patient with relapsed/refractory acute myeloid leukemia or AML in a monotherapy arm of its on-going Phase 1b trial evaluating adult patients with AML, has received an allogeneic stem cell transplant subsequent to receiving APVO436 and experiencing significant reduction in bone marrow blasts (Press release, Aptevo Therapeutics, MAR 29, 2022, View Source [SID1234611133]). This follows the Company’s previous announcement that a patient receiving combination therapy is also moving to transplant after one cycle of therapy.

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"We are very pleased to report that a refractory secondary AML patient, after receiving APVO436 as monotherapy, experienced a significant reduction in bone marrow blasts, tolerated the treatment well, experienced clinical benefit and was therefore able to proceed to allogeneic transplant. Prior to trial entry, this patient had refractory disease after receiving multiple other lines of therapy and had a very poor prognosis. There were few therapeutic options left with which to fight the disease," said Justin Watts, MD, Associate Professor of Medicine, Chief, Leukemia Section at the University of Miami Sylvester Comprehensive Cancer Center and treating investigator. "Without APVO436, this patient would not have proceeded to transplant, a highly desirable outcome for patients with AML."

APVO436 is currently being evaluated in a Phase 1b expansion trial for adult patients with AML in a multi-center, multi-cohort study of up to 90 patients who will receive either APVO436 in combination with standard of care chemotherapies or as monotherapy. In 2021, the Company announced results from its Phase 1b dose escalation trial in patients with both AML and myelodysplastic syndrome or MDS. Results showed that APVO436 exhibited a favorable safety profile with acceptable tolerability and generally manageable drug-related adverse events. Promising clinical activity was also observed in 11 of 40 patients (27.5%) in the dose escalation part of the study and reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2021. This included two complete remissions in patients with AML and three complete marrow responses in patients with MDS.

"We are excited that a patient receiving monotherapy proceeded to transplant in the expansion trial, further demonstrating APVO436 clinical activity in AML and in support of our findings from the dose escalation part of the trial reported last year, said Dirk Huebner, MD, Senior Medical Advisor at Aptevo. "While our clinical evaluation of APVO436 is still in early stages, the safety profile, overall tolerability, and clinical activity reported to date demonstrate the potential for APVO436 to have meaningful impact on the AML treatment paradigm."

About APVO436

Overexpression of CD123 is the hallmark of many forms of leukemia. Aptevo’s lead proprietary drug candidate, APVO436 is a bispecific CD3xCD123 ADAPTIR that is designed to redirect the immune system of the patient to destroy leukemia cells expressing the target CD123 molecule on their surface. This antibody-like recombinant protein therapeutic is designed to engage both leukemia cells and T-cells of the immune system and bring them closely together to trigger the destruction of leukemia cells. APVO436 has been engineered using Aptevo’s proprietary and enabling bioengineering methods and is designed to reduce the likelihood and severity of cytokine release syndrome (CRS). APVO436 has received orphan drug designation ("orphan status") for AML according to the Orphan Drug Act.

On March 29, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that the U.S. Food and Drug Administration (FDA) has cleared UroGen’s Investigational New Drug (IND) application to begin a novel Phase 1 clinical study of the anti-CTLA-4 immunotherapy UGN-301 (zalifrelimab) in patients with recurrent NMIBC (Press release, UroGen Pharma, MAR 29, 2022, View Source [SID1234611132]). The multi-arm Phase 1 study is expected to start in April and support the development of UGN-301 in high-grade (HG) NMIBC.

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UroGen’s pursuit to harness the power of the immune system to fight cancer begins with UGN-301, which UroGen views as a potential cornerstone of a variety of combination therapies targeting recurrent NMIBC and high-grade cancers. UroGen initially plans to combine UGN-301 with UGN-201, the Company’s proprietary formulation of imiquimod a toll-like receptor 7 (TLR7) agonist, which has demonstrated single-agent activity in high-risk bladder cancer patients.

The novel study design will utilize a Master Protocol that UroGen believes is a more efficient and streamlined approach to development. It will provide more flexibility to add study arms as the trial progresses and increase efficiency and reduces costs. UroGen expects the Master Protocol will allow the Company to more quickly evaluate safety, tolerability and dosing of UGN-301 in combination with additional immunomodulators and chemotherapies, with the goal of developing optimized medicines for patients.

"We are pleased that our IND application was cleared to proceed, and we can begin to explore our innovative approach to meeting the high unmet needs in bladder cancer, especially for patients with high-grade disease," says Mark Schoenberg, Chief Medical Officer, UroGen Pharma. "Intravesical delivery of combination therapies is unique with the goal of improving efficacy while avoiding the toxicities associated with systemic treatment of immunotherapies. Our proprietary technology enables local delivery of treatments, which provide opportunities to pursue several promising drug combinations."

Unmet Needs in Bladder Cancer
Bladder cancers are described as muscle invasive or non-muscle invasive based on whether they have invaded the wall of the bladder. HG NMIBC is associated with an increased risk of recurrence and progression. Approximately 25,000 people are diagnosed with HG NMIBC annually. Transurethral resection of bladder tumor (TURBT) followed by intravesical bacillus Calmette-Guérin (BCG) is currently the standard of care for treatment of HG NMIBC.

HG NMIBC patient response to BCG therapy has long been interpreted as evidence that bladder cancer is sensitive to immunotherapy. Unfortunately, many patients with HG disease do not respond to BCG or relapse following therapy. In these cases, patients have limited therapeutic options and often proceed to bladder removal as a means of forestalling disease progression which has a significant association with cancer specific mortality. UroGen’s proprietary RTGel technology provides a novel method for delivering alternatives to BCG including immunomodulatory molecules such as UGN-301 as well as chemotherapeutic agents and other drugs with diverse molecular characteristics and sizes.