On October 30, 2018 Pulse Biosciences, Inc. (Nasdaq: PLSE), a novel medical therapy company bringing to market its proprietary Nano-Pulse Stimulation (NPS) platform, reported recent corporate developments and financial results for the three- and nine-month periods ended September 30, 2018 (Press release, Pulse Biosciences, OCT 30, 2018, View Source [SID1234530396]).

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Recent Developments

Completion of enrollment and patient treatments in the Company’s NPS clinical study for the treatment of Sebaceous Hyperplasia (SH)

Study enrollment and patient treatments were completed in the Company’s multi-center study to evaluate the safety and efficacy of NPS for the treatment of Sebaceous Hyperplasia, a common but difficult-to-treat facial lesion.

Preliminary data on the first 79 of 226 (35%) treated lesions indicate excellent safety and efficacy results to date with no adverse events and over 95% of treated lesions rated as clear or mostly clear after 60-day follow-up.

Patient follow-up visits scheduled to be completed during the fourth quarter with study data available by the end of 2018.

Initiation of a clinical feasibility study using NPS to treat patients with cutaneous warts.

First patient enrollment and treatment in the Company’s feasibility study that includes up to 20 subjects at the prestigious Scripps Clinic in San Diego, CA. Patient enrollment commenced October 2018. Treatment and follow-up are expected to be completed during the first quarter of 2019.

Continued enrollment in NPS Basal Cell Carcinoma (BCC) Biomarker Study

First patient enrolled and treated in the Company’s multi-center "treat and resect" study evaluating local lesion effect and immune response changes to NPS. Study enrollment is progressing and is expected to be completed by the end of 2018 with data available during the first quarter of 2019.

Clinical introduction of the CellFX System, the Company’s next generation Nano-Pulse Stimulation system.

Designed with commercial intent for office, outpatient, or hospital setting;

Simple and intuitive system design suitable across multiple clinical applications;

Integrated networking capability for a per-click revenue model; and

Single-patient-use applicator available with a variety of tip sizes for different applications.

"We’re pleased with the continued and significant progress we’ve made and are making towards bringing our NPS platform closer to commercialization," said Darrin Uecker, Pulse Biosciences’ President and Chief Executive Officer. "Our data continues to demonstrate safety and efficacy allowing us the opportunity to move forward with our plans to target the cash paying aesthetic dermatology market while we continue to make progress in longer-term opportunities."

Financial Highlights

Cash, cash equivalents, and investments totaled $21.0 million at September 30, 2018, compared to $38.1 million at December 31, 2017. Cash use totaled $6.5 million for the third quarter of 2018 compared to cash use of $3.9 million for the fourth quarter of 2017. Cash use for 2018 is currently anticipated to total approximately $24 million.

Operating expenses for the three-month period ended September 30, 2018 totaled $10.9 million, compared to $7.5 million for the three-month period ended September 30, 2017. Operating expenses for the three-month period ended September 30, 2018 included non-cash stock-based compensation of $3.4 million, compared to non-cash stock-based compensation of $3.4 million for the three-month period ended September 30, 2017.

Operating expenses for the nine-month period ended September 30, 2018 totaled $28.9 million, compared to $17.0 million for the nine-month period ended September 30, 2017. Operating expenses for the nine-month period ended September 30, 2018 included non-cash stock-based compensation of $10.0 million, compared to non-cash stock-based compensation of $6.4 million for the nine-month period ended September 30, 2017.

Conference Call Details

Pulse Biosciences will host an investor call on October 30, 2018, at 1:30 p.m. PDT / 4:30 p.m. EDT. The telephone dial-in number for the call is (844) 494-0190 (U.S. toll-free) or (508) 637-5580 (international) using Conference ID 2098843. Listeners will also be able to access the call via webcast available on the Investors section of the Company’s website at www.pulsebiosciences.com.

On October 30, 2018 UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, with a focus on uro-oncology, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to the Company’s lead product candidate, UGN-101, (mitomycin gel) for instillation (Press release, UroGen Pharma, OCT 30, 2018, View Source [SID1234530412]). UGN-101 is currently in Phase 3 development for the treatment of patients with low-grade upper tract urothelial cancer (LG UTUC). Breakthrough Therapy Designation is designed to expedite the development and review of new drugs to treat serious or life-threatening conditions, so patients may have access to therapies through FDA approval as soon as possible. The FDA previously granted both Orphan Drug and Fast Track designations to UGN-101 for the treatment of LG UTUC.

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"We are very excited about receiving the Breakthrough Therapy Designation for UGN-101 and the potential to deliver this far less invasive, organ-sparing therapy option to patients," said Ron Bentsur, Chief Executive Officer of UroGen. "We look forward to working with the FDA as we prepare to initiate a rolling submission of the UGN-101 New Drug Application (NDA) later this year, with the potential to become the first drug ever approved as frontline treatment of LG UTUC."

"UGN-101 was developed to provide an effective alternative to current treatment options, that avoids the risks of surgery, anesthesia, and the deleterious effects of kidney removal," said Mark Schoenberg, M.D., Chief Medical Officer of UroGen. "The Breakthrough Therapy Designation confirms that UGN-101 represents a novel and effective approach to treat this devastating disease, and we look forward to close collaboration with the FDA as we bring this potentially transformative therapy to patients with LG UTUC as quickly as possible."

In the United States, approximately 6,000 to 8,000 patients present with new or recurrent LG UTUC every year1, and nearly 14,500 people are currently living with low-grade LG UTUC. LG UTUC is a rare malignant tumor of the cells lining the urinary tract. It most commonly presents in the elderly who also suffer from comorbid conditions such as hypertension, diabetes, obesity and the metabolic syndrome. There is a clear unmet medical need to provide effective, organ-sparing therapy for these patients because the current standard of care imposes significant burdens on both patients and the healthcare system. Patients diagnosed with LG UTUC typically face either complete removal of the kidney and/or partial removal of the ureter. In selected patients who present with a limited tumor burden, repetitive endoscopic tumor resection is employed when feasible. These interventions are surgical in nature and require anesthesia; and these procedures are associated with the typical risks for this patient population, including bleeding, infection, injury to adjacent organs, and the potential long-term morbidity associated with kidney removal. Due to the anatomy and physiology of the upper urinary tract and renal pelvis, organ-sparing endoscopic tumor resection is often challenging, leading to high rates of recurrence. Although the administration of water-based drug solutions has been used following surgery to treat patients with LG UTUC, evidence of the therapeutic benefit of this approach is lacking and none of these products have been approved for frontline therapy. Continuous urine flow and the inability of the upper urinary tract to retain a liquid volume under normal circumstances results in limited exposure of target tissue to aqueous medications. No therapeutic agent has ever been approved to treat LG UTUC.

The criteria for Breakthrough Therapy Designation require preliminary clinical evidence that demonstrates that use of the drug may result in substantial improvement on at least one clinically significant endpoint over available therapy. The Breakthrough Therapy Designation for UGN-101 is supported by data from the ongoing Phase 3 OLYMPUS clinical trial of UGN-101 for the non-surgical treatment of LG UTUC. Results from an interim analysis presented in May, 2018 showed a complete response (CR) rate of 59 percent (20 out of the interim analysis intent to treat population of 34 patients) who were evaluated for primary disease evaluation (PDE, or the primary endpoint). In addition, 15 percent (five of 34 patients) achieved a partial response. At the time of the interim analysis presentation, of the 20 patients who achieved a CR, 13 patients had reached three-month follow-up, and all remained in CR. Four of these 13 patients had reached six-month follow-up and one of the 13 patients had reached nine-month follow-up, and all remained in CR. UGN-101 appeared to be well-tolerated with most treatment-emergent adverse events characterized as mild or moderate and transient.

About UGN-101

UGN-101 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of low-grade (LG) upper tract urothelial cancer (UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-101 is designed to enable longer exposure of mitomycin to the urinary tract tissue, thereby potentially enabling the treatment of tumors by non-surgical means. UGN-101 is delivered to patients using standard intravesical catheters.

On October 29, 2018 Significant developments in cancer research were presented by NantHealth (NASDAQ: NH) and NantOmics at the ESMO (Free ESMO Whitepaper) conference in Munich, Germany this week (Press release, NantHealth, OCT 29, 2018, View Source;p=RssLanding&cat=news&id=2373856 [SID1234530256]). NantHealth, a leader in breakthrough cancer research and solutions to improve patient care and lower healthcare costs, in conjunction with NantOmics, a leader in molecular diagnostic testing, conducted one oral presentation and four papers, including a few demonstrating the promise of the company’s Liquid GPS, a blood-based molecular test that provides oncologists with a powerful tool for non-invasive tumor profiling and quantitative monitoring of treatment response. ESMO (Free ESMO Whitepaper) is the world’s second largest cancer symposium where researchers and clinicians come together to study the latest breakthroughs in treatment.

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"We continue to be on the cutting edge of cancer research and that, coupled with the advancements we’re making in all areas of cancer diagnostics, treatment plans and provider solutions, are all important steps in supporting physicians and patients," said Dr. Sandeep Reddy, Chief Medical Officer, NantHealth. "Our researchers and scientists are working very hard to tackle some of the toughest challenges in cancer research and sharing them as quickly as possible. Our Liquid GPS tumor profiling is dramatically changing the way that physicians identify and treat different types of cancer."

The oral presentation, Gene mutation status in circulating tumor DNA (ctDNA) and first-line FOLFOXIRI plus bevacizumab (bev) in metastatic colorectal cancer (mCRC) harboring RAS mutation, authored by Y. Sunakawa, et al, includes investigational data suggesting that circulating DNA markers may predict outcomes in mCRC and can guide treatment decisions for these patients.

Additional papers were reviewed showing breakthroughs in immune oncology, including:

Title: Differential expression of PD-L1 and immune biomarkers by age: Decreased expression in pediatric/AYA patients with advanced cancer
Author: Dr. Omid Hamid, et al
Description: Whole exome and RNA sequencing of 1,467 patients highlights younger patients (age < 24) with advanced cancer appear to have lower levels of CTLA4 and PD-L1 expression.
Key Takeaway: Immune checkpoint drugs have not yet been approved in pediatric and adolescent/young adult patients. Large dataset results show it may be necessary to employ new combinations to be used in younger patients such as Nant’s Cancer Vaccine, immunotherapy that combines the delivery of metronomic, low-dose chemotherapy and radiation therapy with natural killer (NK) cell-based technology to enhance patient’s immune system response against cancer cells.

Title: PD-L1 expression is strongly associated with TIGIT, FOXP3 and LAG3 across advanced cancers, but not OX40, TIM3 and IDO
Author: Dr. Sumanta K. Pal, et al
Description: RNA sequencing reveals immune checkpoint gene expression was not significantly different in TMB high versus low groups. In patients demonstrating the highest PD-L1 expression, higher expression of CTLA4, TIGIT, FOXP3 and LAG3 were also observed.
Key Takeaway: While researchers are focused on TMB as a more promising biomarker for Immuno-Oncology (IO) therapy than PDL1, this research shows PDL1 may be a marker of generalized tumor inflammation and could be the reason those tumors respond to IO drugs.

Title: Predicting survival benefit of capecitabine plus cisplatin in patients with metastatic gastric cancer using quantitative proteomics
Author: D. Yan, et al
Description: Targeted proteomic analysis of metabolic enzymes required for intracellular activation of the chemotherapeutic drug capecitabine identifies specific enzymes and expression cutoffs which are significantly associated with extended survival in metastatic gastric cancer patients given this drug regimen.
Key Takeaway: Clinical tissue analysis of specific proteomic biomarkers could be used to better personalize the usage of chemotherapeutic agents.

Title: Development and validation of neuroendocrine tumor marker panel in small biopsies using multiplexed mass spectrometry
Author: S. Thyparambil, et al
Description: Immunohistochemical analysis of neuroendocrine tumor markers is performed on only a small subset of cancer. In spite of the approval of several new agents with specific activity against neuroendocrine tumors. A multiplexed clinical tissue proteomics assay was developed to add analysis of neuroendocrine markers into a larger diagnostic panel in order to better identify candidates for neuroendocrine specific therapy.
Key Takeaway: A three protein targeted proteomics assay can identify neuroendocrine tumors with high sensitivity and specificity. The expanded use of this technology may efficiently identify patients for optimal therapy.

On October 29, 2018 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that it will release its third quarter 2018 financial results on Monday, November 5, after the close of the U.S. financial markets (Press release, Syndax, OCT 29, 2018, View Source [SID1234530322]).

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In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET on Monday, November 5, to discuss the Company’s financial results and provide a general business update.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 8397904
Domestic Dial-in Number: 855-251-6663
International Dial-in Number: 281-542-4259
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.

On october 29, 2018 LSK BioPharma (LSKB, Company) reported it has completed patient enrollment in its pivotal Phase 3 trial, ANGEL, which is evaluating the efficacy and safety of rivoceranib plus Best Supportive Care (BSC) compared to placebo plus BSC in patients with advanced or metastatic gastric cancer (Press release, LSK BioPharma, OCT 29, 2018, View Source [SID1234530520]). The ANGEL study is designed to support approval by the U.S Food and Drug Administration (FDA), the European Medicines Agency (EMA), Japanese Pharmaceuticals and Medical Devices Agency (PMDA), Korean Ministry of Food and Drug Safety (MFDS) and Taiwan Food and Drug Administration (TFDA).

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"Completion of enrollment in the ANGEL study is an important achievement and a critical milestone for our gastric cancer program and we appreciate all of the support from patients and investigators in accomplishing this global development milestone" said Dr. Sung Chul Kim, LSKB President, "Although rivoceranib has been approved in China, there is still no oral small-molecule angiogenesis inhibitor approved outside of China for gastric cancer. We believe it will be the first such drug for these patients worldwide."

The Company will now focus on follow-up of enrolled patients and preparation of the data for analysis with an expectation that the top-line, unblinded efficacy and safety data will be reported in the second half of 2019. The Company further expects to submit a New Drug Application for rivoceranib with the FDA in late 2019.

About the ANGEL Study

The ANGEL study is a prospective, randomized, double-blinded, placebo-controlled, multinational, multicenter, parallel-group, phase III study to evaluate the efficacy and safety of rivoceranib plus Best Supportive Care (BSC) compared to placebo plus BSC in patients with advanced or metastatic gastric cancer. A total of 459 patients have been randomized in the ANGEL study in 12 countries (in USA, EU, EEC, Japan, Korea and Taiwan). Details of the ANGEL Study can be found at the following link: View Source

About Rivoceranib (Apatinib)
Rivoceranib is the first successful oral small-molecule angiogenesis inhibitor in gastric cancer. Rivoceranib acts by inhibiting angiogenesis, a critical process in cancer growth and proliferation. Specifically, rivoceranib selectively inhibits VEGFR-2 which mediates the primary pathway for tumor-mediated angiogenesis. It was approved in China (advanced gastric cancer, Dec 2014) where it is marketed by the Chinese-territory license-holder Jiangsu Hengrui Medicine Co., Ltd. LSK BioPharma holds the global rights (ex-China). The Company is currently conducting a global (12 countries including US, Japan, Korea, Italy, Germany, and Russia) Phase 3 clinical trial of rivoceranib in advanced or metastatic gastric cancer patients. Rivoceranib has been clinically tested in over 1,000 patients worldwide and has demonstrated efficacy in numerous cancers including gastric cancer, CRC, HCC, NSCLC, esophageal cancer, thyroid cancer, mesothelioma, and neuroendocrine tumors. It has also shown potential to significantly improve clinical outcomes in combination with chemotherapeutics and immunotherapy, as well as for maintenance therapy. LSKB has received notification designating rivoceranib as an orphan medicinal product for the treatment of gastric cancer from the European Commission in the European Union, the US FDA, as well as the MFDS in South Korea.