On November 17, 2020 UroGen Pharma Ltd. (Nasdaq: URGN) reported final topline results from the single-arm, open-label OPTIMA II Phase 2b trial evaluating the efficacy and safety of investigational UGN-102 (mitomycin) for intravesical solution in patients with low-grade intermediate risk non-muscle invasive bladder cancer (LG IR-NMIBC) (Press release, UroGen Pharma, NOV 17, 2020, View Source [SID1234571273]). As previously reported, 65% (41/63) of patients receiving UGN-102 achieved a complete response (CR) three months after the start of therapy. In this subset of patients, duration of response at nine months (12-months from start of therapy) was estimated by Kaplan-Meier analysis to be 72.5%. Median duration of response was not reached. The Company expects to initiate a Phase 3 study evaluating UGN-102 versus current standard of care by the end of the year.

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Treatment with UGN-102 was generally well tolerated and the safety profile was consistent with previously reported results. In the OPTIMA II trial, the majority of the most common adverse events (≥ 10%) were reported as mild to moderate in severity and include dysuria, urinary frequency, hematuria, urinary urgency, urinary tract infection and fatigue. No treatment related serious adverse events were reported.

"The current approach to treating patients diagnosed with low-grade intermediate risk non-muscle invasive bladder cancer is surgery. In most cases, the cancer comes back and repetitive surgical intervention is required. This puts a tremendous burden on patients and their families and can even be life-threatening," said Andrea Maddox-Smith, Chief Executive Officer, Bladder Cancer Advocacy Network. "Being able to provide patients with an alternative, non-surgical treatment option that is effective, well-tolerated and durable, would greatly benefit those in the patient community."

Patients with LG IR-NMIBC are chronically relapsing and currently, their only treatment option is repeated transurethral resection of bladder tumors (TURBT). Some patients require multiple TURBT surgeries per year, which can lead to increased morbidity and risks associated with repetitive anesthesia. It is estimated that 80,000 people in the U.S. are treated annually for LG IR-NMIBC. This includes newly diagnosed patients and patients who have a recurrence after surgery.

"We are extremely encouraged by the OPTIMA II data and believe UGN-102 has the potential to provide a safe, durable, outpatient treatment alternative for low-grade intermediate risk non-muscle invasive bladder cancer," said Dr. Mark Schoenberg, Chief Medical Officer at UroGen. "We look forward to the expected initiation of our Phase 3 trial this year and further exploring the potential of our innovative technology in advancing new treatments for specialty cancers and urologic diseases."

The Company anticipates submitting the data for presentation at an upcoming medical meeting as well as potential publication in a peer-reviewed journal.

About the Phase 2b OPTIMA II Trial

OPTIMA II (OPTimized Instillation of Mitomycin for Bladder Cancer Treatment) is an open-label, single-arm, multi-center Phase 2b clinical trial of investigational agent UGN-102 (mitomycin) for intravesical solution to evaluate its safety and efficacy in patients with low-grade non-muscle invasive bladder cancer (LG NMIBC) at intermediate risk of recurrence. Intermediate risk is defined as one or two of the following: multiple tumors, solitary tumor >3 cm, or recurrence (≥ 1 occurrence of LG NMIBC within one year of the current diagnosis). Patients were to receive six weekly intravesical instillations of 75 mg UGN-102 in an office setting. The chemoablative effect of UGN-102 was assessed three months after initiation of study treatment with complete response (CR) defined as a negative endoscopic examination, negative cytology, and when indicated, a negative for-cause biopsy. Patients achieving CR were followed quarterly to 12 months after initiation of study treatment to evaluate safety, efficacy, and durability.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an investigational drug formulation of mitomycin in Phase 2b development for the treatment of low-grade intermediate risk non-muscle invasive bladder cancer. Utilizing the RTGel Technology Platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter. The Company reported topline interim results from the Phase 2b OPTIMA II trial in May 2020 and intends to begin a Phase 3 study to further investigate UGN-102 in the treatment of this condition by year end 2020.

On November 17, 2020 Zimmer Biomet Holdings, Inc. (NYSE and SIX: ZBH), a global leader in musculoskeletal care, reported that President and CEO Bryan Hanson and Executive Vice President and CFO Suky Upadhyay will be presenting at the 2020 Evercore ISI HealthCONx Conference on Tuesday, December 1, 2020, at 10:30 a.m. Eastern Time (Press release, Zimmer Holdings, NOV 17, 2020, https://www.prnewswire.com/news-releases/zimmer-biomet-to-present-at-2020-evercore-isi-healthconx-conference-and-piper-sandler-32nd-annual-virtual-healthcare-conference-301173015.html [SID1234571296]). Additionally, Executive Vice President and CFO Suky Upadhyay will be presenting at the Piper Sandler 32nd Annual Virtual Healthcare Conference Wednesday, December 2, 2020, at 11:00 a.m. Eastern Time.

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Live webcasts of the presentations can be accessed via Zimmer Biomet’s Investor Relations website at https://investor.zimmerbiomet.com. The webcasts will be archived for replay following the conference.

On November 17, 2020 NANOBIOTIX (Euronext: NANO – ISIN: FR0011341205 – the "Company") (Paris:NANO), a clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, reported that the United States Food and Drug Administration (FDA) has provided ‘Safe to Proceed’ notifications for two additional trials in its ongoing clinical collaboration with The University of Texas MD Anderson Cancer Center (MD Anderson) (Press release, Nanobiotix, NOV 17, 2020, View Source [SID1234571172]). These trials were co-developed with Nanobiotix and MD Anderson is the sponsor and executor.

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Significant Unmet Needs and Opportunity in Cancer Immunotherapy

Cancer immunotherapies such as immune checkpoint inhibitors (ICIs) have shown promising clinical outcomes over the past two decades; and are often used for patients with advanced cancers once other therapies have reached the end of their effectiveness. However, the vast majority of patients only receive a temporary benefit or no benefit from ICIs, as they either develop resistance to the treatment during the course of therapy or are non-responsive to the treatment altogether (only 15%-20% of patients respond, according to published data). These barriers present a significant unmet need to improve the efficacy ICIs and expand their potentially curative benefits to more patients with advanced cancers.

Combining ICIs with radiation therapy is emerging as a valuable strategy to "prime" an immune response and thereby increase the response rate, however the efficacy of radiation therapy is limited by toxicities related to the exposure of healthy tissues.

NBTXR3 is injected one time, directly into solid tumors. The product candidate is designed to increase the energy deposit from radiation therapy within the target tumor and subsequently increase the tumor-killing effect without increasing toxicity in surrounding healthy tissue. Pre-clinical and clinical data also suggest that NBTXR3 activated by radiation therapy can prime the immune system, creating an anti-tumor immune response that produces both local and systemic effects.

A Phase II Study of NBTXR3 Activated by Radiation and Combined with Pembrolizumab for Patients with Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma with Limited PD-L1 Expression or Refractory to PD-1 Blockade

This MD Anderson trial is an open label, two cohort, non-randomized phase II study. The primary objective of the study is to evaluate tumor response of NBTXR3 activated by radiation therapy in combination with pembrolizumab in patients with recurrent or metastatic (R/M) head and neck squamous cell carcinoma (HNSCC).

The population includes patients with inoperable R/M HNSCC of the oropharynx, oral cavity, hypopharynx, larynx or neck. Patients could be anti-PD-1/L1 naïve or refractory. Up to 60 patients may be treated, with up to 40 in the first cohort and up to 20 in the second cohort. The first cohort will include anti-PD-1/L1 naïve patients with a combined positive score (CPS) between greater than or equal to 1% and less than 20%. The second cohort will include anti-PD-1/L1 refractory patients irrespective of PD-L1 expression.

A Phase II Study of Reirradiation with NBTXR3 in Patients with Inoperable Locoregional Recurrent Head and Neck Squamous Cell Carcinoma

This MD Anderson trial is an open label, two cohort, non-randomized phase II study. The primary objectives of the study are: (i) to estimate progression-free survival (PFS) and the early clinical benefit in patients treated with NBTXR3 activated by SBRT re-irradiation, with concurrent pembrolizumab; (ii) to assess the safety profile and estimate the early clinical benefit of NBTXR3 activated by a reduced dose of IMRT or IMPT re-irradiation with concurrent pembrolizumab.

The population includes patients with inoperable, locoregional recurrent head and neck squamous cell carcinoma (HNSCC) or second primary HNSCC, previously treated with definitive radiation therapy and without radiographic evidence of metastases. Patients could be anti-PD-1/L1 naïve or non-responders. Up to 80 patients may be treated, with up to 60 in the SBRT cohort and up to 20 in the IMRT/IMPT cohort.

About NBTXR3
NBTXR3 is a novel, potentially first-in-class radioenhancer composed of functionalized hafnium oxide nanoparticles that is administered via one-time intra-tumoral injection and activated by radiation therapy. The primary mode of action (MoA) of NBTXR3 is designed to generate increased cellular destruction when activated by radiation therapy without increasing damage to healthy tissues. Subsequently, this cellular destruction also triggers an adaptive immune response.

NBTXR3 is being evaluated in locally advanced head and neck squamous cell carcinoma (HNSCC) of the oral cavity or oropharynx in elderly patients unable to receive chemotherapy or cetuximab with limited therapeutic options. Promising results have been observed in the phase I trial regarding local control. In the United States, the Company has started the regulatory process to commence a phase III clinical trial in locally advanced head and neck cancers. In February 2020, the United States Food and Drug Administration granted the regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced head and neck squamous cell cancer who are not eligible for platinum-based chemotherapy.

Nanobiotix is also running an Immuno-Oncology development program. The Company has launched a Phase I clinical trial of NBTXR3 activated by radiotherapy in combination with anti-PD-1 checkpoint inhibitors in locoregional recurrent (LRR) or recurrent and metastatic (R/M) HNSCC amenable to re-irradiation of the HN and lung or liver metastases (mets) from any primary cancer eligible for anti-PD-1 therapy.

Other ongoing NBTXR3 trials are treating patients with hepatocellular carcinoma (HCC) or liver metastases, locally advanced or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with concurrent chemotherapy, and pancreatic cancer. The Company is also engaged in a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center to further expand the NBTXR3 development program.

On November 17, 2020 xCures Inc. reported that it is launching xACCESS – a new registration module for their AI-assisted clinical study platform (Press release, xCures, NOV 17, 2020, View Source [SID1234571196]). The module is used to enroll cancer patients and empower them, and their physicians, to make informed decisions and learn about access to treatment programs. The xCures platform uses a Master Observational Registry to efficiently run a wide variety of oncology studies and decentralized trials, prospectively generating real-world evidence.

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"xACCESS is xCures’ first patient-centric technology module, onboarding patients directly to our sponsored studies and partner programs," stated Mika Newton, xCures’ CEO. "This is an important milestone on our journey to make the cancer treatment ecosystem work better for patients and facilitating access to the best treatment options."

The xACCESS registration module enables rapid scaling by eliminating bottlenecks in eConsenting and acquiring medical records, providing benefits to patients, physicians, partners, payers, sponsors, and investors. The resulting Real-World Evidence (RWE) supports the assessment of safety, efficacy, and utility of investigational and approved cancer therapies.

The xACCESS module will be used for all clinical studies and programs available on the xCures platform, including the recently announced Compassionate Use program for ulixertinib (BVD-523), currently enrolling patients with MAPK pathway aberrant cancer.

On November 17, 2020 PierianDx, the leading clinical genomics informatics company and Bench International, a leading global executive search firm, jointly announce the appointment of Mark McDonough as Chief Executive Officer (CEO) and member of the Board of Directors of PierianDx, effective immediately (Press release, PierianDx, NOV 17, 2020, View Source [SID1234571242]). In his new role, McDonough will help fulfill the company’s unique value proposition of enabling precision medicine through its best-in-class variant interpretation knowledgebase, integrated infrastructure, and clinical genomics reporting for both cancer and inherited disease. McDonough succeeds Chairman of the Board and Interim CEO, Joe Boorady, who remains as Chairman of the PierianDx Board of Directors.

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"Mark commands a deep understanding of the diagnostics market, and we are delighted to welcome him to the PierianDx family during this exciting time in the company’s development," says Joe Boorady. "Mark will drive company execution on its IVD strategy and solidify its leadership position in the global cancer and hereditary disease diagnostics market."

Mark McDonough is a global technology business executive with nearly 30 years of experience. His personal mission is to impact change in the field of healthcare in a compassionate yet resolute manner, leading by example. He most recently served as CEO and board member for Immunis.AI, where he secured funding and developed a top flight team within six months to launch a novel noninvasive liquid biopsy immunogenomics assay for prostate cancer patients on ‘active surveillance’. Prior to this, McDonough served as President, CEO and board member of CombiMatrix from 2012 through 2018, orchestrating its sale to Invitae in November 2017. Under his leadership, CombiMatrix experienced 14 consecutive quarters of record growth. Earlier in his career, he served in various sales leadership roles within the clinical laboratory industry and developed his leadership skills as a Navigator and Communications Officer in the US Navy.

"An established, respected and trusted leader in the genomics industry, Mark is the perfect CEO for PierianDx," said DeeDee DeMan, Chairman and CEO of Bench International. "He has a remarkable track record of developing teams, fundraising and directing corporate strategy and growth."

"I am excited to join PierianDx and lead this talented team committed to delivering tools to our partners and customers that enable precision medicine adoption at scale," said Mark McDonough. "We believe advanced genomic testing should be democratized and available to all at the highest quality and lowest cost. I look forward to helping propel the company as we expand our market leadership and serve more patients through our partners and customers."