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.

On March 29, 2022 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a late clinical stage biopharmaceutical company focused on helping patients fight cancer with oncolytic viral immunotherapies, reported financial results for the fourth quarter and year ended December 31, 2021 and provided a corporate update (Press release, Candel Therapeutics, MAR 29, 2022, View Source [SID1234611131]).

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"2021 was a year of significant progress for Candel, filled with important accomplishments in development of our late-stage pipeline of novel oncolytic viral immunotherapies for cancer treatment. We strengthened our organization for future growth with our successful initial public offering, by meeting key clinical trial accrual milestones and by enhancing our talent pool," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. "Today, we are well positioned for major catalysts over the next 12 months, namely, multiple data readouts across our product candidates and advancement of our pipeline of promising therapeutics with potential to treat various solid tumors, including lung, brain, pancreatic and prostate cancer. We look forward to delivering on our mission to develop oncolytic viral immunotherapies aimed at tipping the balance in favor of the patient’s immune system to fight cancer."

Fourth Quarter 2021 & Recent Highlights

Presented Candel’s novel oncolytic viral immunotherapies at several preeminent industry conferences:
October 2021: Cambridge Healthtech Institute’s 9th Annual Immuno-Oncology Summit
October 2021: Hanson Wade 6th Annual Oncolytic Virotherapy Summit
October 2021: 28th Annual Prostate Cancer Foundation Scientific Retreat
November 2021: 13th International Oncolytic Virus Conference Meeting
November 2021: Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting
Patrick Y. Wen, MD, principal investigator, presented initial safety data on CAN-2409 in combination with nivolumab from Candel’s Phase 1 clinical trial in high-grade glioma at the 26th Annual Meeting of the Society for Neuro-Oncology in November 2021.
Established a collaboration with Partnership for Accelerating Cancer Therapies and the Cancer Immune Monitoring and Analysis Centers – Cancer Immunologic Data Commons to profile the biomarker response to a combination of CAN-2409 and valacyclovir, in combination with anti-PD-1 and PD-L1 immune checkpoint inhibitors in patients with non-small cell lung cancer (NSCLC) in December 2021.
Enhanced executive leadership with the promotion of Francesca Barone, MD, PhD to Chief Scientific Officer and appointment of Seshu Tyagarajan, PhD, RAC as Chief Technical and Development Officer in 2022.
Strengthened cash position with non-dilutive debt financing with Silicon Valley Bank (SVB) in February 2022.
Appointed Mace Rothenberg, MD, prior Chief Medical Officer of Pfizer, as Senior Advisor to the CEO.
Key Upcoming Milestones

The Company expects to present initial data from an ongoing Phase 2 clinical trial of CAN-2409 and valacyclovir combined with PD-1 or PD-L1 targeting agents in patients with NSCLC in June 2022.
In the fourth quarter of 2022, the Company expects to present data from two high grade glioma clinical trials – a Phase 1b clinical trial of CAN-2409 in combination with nivolumab (Opdivo) combined with standard of care first line treatment and a Phase 1 clinical trial of CAN-3110 in recurrent high-grade glioma.
Financial Results for the Year and Fourth Quarter Ended December 31, 2021

Cash Position: Cash and cash equivalents as of December 31, 2021 were $82.6 million, compared to $35.1 million as of December 31, 2020. The increase was due to receipt of $71.3 million of net proceeds from the Company’s successful initial public offering (IPO) of common stock in August 2021. In addition, the Company utilized $22.2 million in the year ended December 31, 2021 to fund operating activities. Subsequent to December 31, 2021, the Company entered into a term loan agreement with SVB and borrowed $20.0 million. Based on current plans and assumptions, the Company expects that its existing cash and cash equivalents, including the $20 million in term loan proceeds, will be sufficient to fund its operations into the fourth quarter of 2023.

Research & Development Expenses: Research and development expenses were $3.9 million and $15.2 million for the three- and twelve-month periods ended December 31, 2021, respectively, as compared to $3.5 million and $8.8 million for the comparable periods of 2020. The increases were primarily due to increased personnel-related costs, including stock-based compensation, for additional headcount to support the ongoing clinical trials for Candel’s product candidates as well as increased clinical development costs related to our clinical trial sites and the cost of treatment and follow-up on patients. Excluding stock-based compensation expense of ($810,000) and $1.2 million for the three- and twelve-month periods ended December 31, 2021, respectively, research and development expenses for the three- and twelve-month periods ended December 31, 2021, were $4.7 million and $14.0 million, respectively.

General and Administrative Expenses: General and administrative expenses were $3.9 million and $10.7 million for the three- and twelve-month periods ended December 31, 2021, respectively, as compared to $2.6 million and $5.2 million for the comparable periods of 2020. The increases were primarily due to increased personnel-related costs, including stock-based compensation, for additional headcount required to support the growth of the Company and the transition to a public company, an increase in professional fees associated with Candel’s preparation for the IPO completed in August 2021 and costs associated with operating as a public company. Excluding stock-based compensation expense of $364,000 and $1.7 million for the three- and twelve-month periods ended December 31, 2021, respectively, general and administrative expenses for the three- and twelve-month periods ended December 31, 2021, were $3.5 million and $9.0 million, respectively.

Total Operating Expenses: Total operating expenses were $7.8 million and $25.9 million for the three- and twelve-month periods ended December 31, 2021, respectively, as compared to $6.2 million and $13.9 million for the comparable periods of 2020. The increases were primarily due to increased personnel-related costs, including stock-based compensation, for additional headcount required to support the growth of the Company and the transition to a public company, an increase in professional fees associated with Candel’s preparation for the IPO completed in August 2021, an increase in costs associated with operating as a public company and increased clinical development costs. Excluding stock-based compensation expense of ($446,000) and $2.9 million for the three- and twelve-month periods ended December 31, 2021, respectively, total operating expenses for the three- and twelve-month periods ended December 31, 2021, were $8.2 million and $23.0 million, respectively.

Net Loss: Net (income) loss was ($1.6 million) and $36.1 million for the three- and twelve-month periods ended December 31, 2021, respectively, as compared to a net loss of $10.5 million and $17.7 million for the comparable periods of 2020. The net (income) loss for the three- and twelve-month periods ended December 31, 2021, includes a noncash credit and charge of ($9.2 million) and $11.4 million, respectively, for the change in the fair value of the Company’s warrant liability and stock-based compensation expense of ($446,000) and $3.0 million, respectively. Excluding the noncash charges for the change in the warrant liability and the stock-based compensation, the net loss for the three- and twelve-month periods ended December 31, 2021, was $8.0 million and $21.7 million, respectively.