On September 24, 2020 Nucleix, a liquid biopsy company revolutionizing cancer treatment by detecting the disease earlier, reported a published independent review of Bladder EpiCheck studies which showed high diagnostic accuracy and high performance for surveillance of disease recurrence in patients with non-muscle invasive bladder cancer (NMIBC) (Press release, Nucleix, SEP 24, 2020, View Source [SID1234565586]). The publication, titled "The Bladder EpiCheck Test as a Non-Invasive Tool Based on the Identification of DNA Methylation in Bladder Cancer Cells in the Urine: A Review of Published Evidence," was published in the International Journal of Molecular Sciences.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The review analyzed data from five prospective, blinded, single-arm cohort studies of Bladder EpiCheck for NMIBC detection. The review focused on data from populations of patients in surveillance for histologically proven NMIBC. Notably, Bladder EpiCheck showed a high sensitivity and negative predictive value (NPV) in several studies and a higher sensitivity and NPV than cytology in all studies. Authors concluded Bladder EpiCheck had the greatest diagnostic power in patients with high-grade NMIBC, and it could be used as a monitoring tool to extend time between cystoscopies.

"Patients with NMIBC are at high risk for recurrence, making surveillance critical to identify recurrence, progression, or localization in different areas of the urinary tract. However, quarterly cystoscopies are invasive and painful for patients, which places a high burden on them and on healthcare systems," said Aharona Shuali, M.D., vice president of medical at Nucleix. "We are pleased that this review of Bladder EpiCheck demonstrates how the test’s high sensitivity and NPV in detection of high-grade disease makes it a meaningful option to safely reduce frequency of cystoscopies."

The review noted that some positive cases of Bladder EpiCheck with negative cystoscopy and positive/negative cytology results, seem to present DNA methylation changes. These ‘false-positive’ cases of Bladder EpiCheck could identify a genetic high-risk population that may relapse in the near future and deserves close clinical attention. Bladder EpiCheck negative cases could be based on a more stable methylation pattern, which authors propose could represent a population of high-risk bladder cancer patients with an inferior risk of progression.

"We need a test with high accuracy for NMIBC, especially for high-grade NMIBC, to help avoid unnecessary cystoscopies," said Mariangela Mancini, M.D., consultant urological surgeon at the University of Padua in Padua, Italy, and lead author of the study. "The high performance demonstrated by the current evidence, combined with Bladder EpiCheck’s non-invasive nature, could make it an invaluable tool in the future for follow-up of bladder cancer patients. Furthermore, having a safe alternative for follow-up that does not require patients to come to the hospital, could be even more valuable during the COVID-19 pandemic."

About Bladder EpiCheck

Bladder EpiCheck provides patients and clinicians with a simple, objective urine test to detect recurrence of bladder tumors. The test analyzes subtle disease-specific changes in DNA methylation markers, allowing for the detection of 92% of the high-risk (non Ta-LG) cancers. Bladder EpiCheck demonstrated negative predictive value (NPV) of 99% for high-risk cancer, meaning that when receiving a negative Bladder EpiCheck result, there is 99% chance that no high-risk cancer is present1. Bladder EpiCheck is intended for use as a noninvasive method for monitoring of tumor recurrence in conjunction with cystoscopy in patients previously diagnosed with bladder cancer. Bladder EpiCheck is CE-marked and available in Europe. The test is not available for sale in the United States.

On September 24, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported participation at the following upcoming conferences (Press release, Rocket Pharmaceuticals, SEP 24, 2020, View Source [SID1234565585]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

SVB Leerink CybeRx Series: Rare Diseases & Genetic Medicines
Thursday, October 1, 2020
Chardan’s 4th Annual Genetic Medicines Conference
Gaurav Shah, M.D., President and CEO, is scheduled to participate in a fireside chat on Tuesday, October 6, 2020, at 2:30 p.m. Eastern Time.
A live audio webcast of the presentation will be available on the Investors section of the company’s website, www.rocketpharma.com. A replay of the presentation will be archived on the Rocket website following the conferences.

On September 24, 2020 Monte Rosa Therapeutics, a biotechnology company focused on developing precision medicines to degrade disease-causing proteins, reported the closing of a Series B financing of $96 million (Press release, Monte Rosa Therapeutics, SEP 24, 2020, View Source [SID1234565584]). This financing will enable Monte Rosa to accelerate the growth of its pipeline, advance development candidates into the clinic and bolster its platform capabilities to rationally design and develop small molecule degraders (also known as molecular glues) that hijack the body’s innate ability to degrade proteins. Through this approach Monte Rosa Therapeutics will eradicate undruggable proteins that cause or drive the progression of genomically defined diseases intractable to standard care, including cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Series B financing was led by Aisling Capital with participation from founding investor Versant Ventures and existing investor New Enterprise Associates. Additional new investors included HBM Healthcare Investments, Cormorant Asset Management, GV, Amzak Health, Casdin Capital, Sixty Degree Capital and Cambridge Asset Management.

"We are pleased to have the support of this strong group of investors who share our vision for driving a next-generation drug discovery approach that captures the promise of expanding the field of protein degradation into a broader array of diseases," said Markus Warmuth, M.D., CEO of Monte Rosa. "With this financing, we are now well positioned to broadly develop our integrated drug discovery platform and advance multiple new therapies toward clinical development."

The company has made robust progress since it was launched by Ridgeline, Versant’s Discovery Engine based in the Technologiepark Basel, in collaboration with The Institute of Cancer Research (ICR), London, and Cancer Research UK, with academic co-founders Professor Ian Collins, Ph.D., Head of Chemistry at ICR – who continues to lead an active collaboration with Monte Rosa – and former ICR Professor Rajesh Chopra, M.D., both thought leaders in the field of protein degradation.

In connection with the Series B round, the company also announced that Andrew Schiff, M.D., of Aisling Capital, and Chandra P. Leo, M.D., of HBM Partners, will join its Board of Directors. They join existing board members Dr. Warmuth; Brad Bolzon, Ph.D., and Alex Mayweg, Ph.D., of Versant; Ali Behbahani, M.D., of NEA; and Kim Blackwell, M.D., of Tempus Labs, Inc.

"Many approved drugs have been developed to inhibit a protein’s function. Monte Rosa’s goal is to eliminate the protein altogether," said Dr. Schiff. "This investment will continue to support the experienced team led by Markus and will enable the company to target known drivers of cancers and other diseases that are currently unaddressed with conventional methods."

"Monte Rosa has built one of the largest and highly focused libraries of small molecule protein degraders that target critical disease drivers," said Dr. Mayweg, Chair of the Board of Directors. "The company is now poised to bring forward multiple drug candidates for some of the most difficult-to-treat cancers and other diseases."

On September 24, 2020 TriSalus Life Sciences (TriSalus), an emerging immuno-oncology company committed to transforming outcomes for patients with solid tumors, reported its therapeutic clinical development strategy following the successful acquisition of SD-101, an investigational IND-ready immunotherapy, from Dynavax Technologies on August 3, 2020 (Press release, TriSalus Life Sciences, SEP 24, 2020, View Source [SID1234565583]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

SD-101 is an investigational toll-like receptor 9 (TLR9) agonist that has been tested in Phase 2 clinical trials for advanced cutaneous melanoma and head and neck cancer, evaluating efficacy, safety, and the ability to increase responsiveness to checkpoint inhibitors in PD-L1 negative tumors. By combining SD-101 with TriSalus’ proprietary FDA cleared, drug delivery technologies, TriSalus will deliver the agent into the local vasculature of solid tumors. The Company intends to begin evaluating SD-101 in patients with uveal melanoma liver metastases followed by testing in patients with pancreatic ductal adenocarcinoma and colorectal cancer liver metastases. In addition, a separate program for locally advanced pancreatic ductal adenocarcinoma is in progress.

Mats Wahlstrom, Chairman of TriSalus Life Sciences, commented, "Our strategy is to develop a portfolio of immuno-oncology therapeutics and innovative infusion technologies to solve the two key reasons for poor outcomes in pancreatic and liver solid tumors ─ immunosuppression and low therapeutic index. The recent acquisition of SD-101 is a strong start to our therapeutics portfolio strategy. We plan to deploy multiple approaches with SD-101 by testing it clinically in a range of liver and pancreatic cancers for which few options currently exist."

Mary Szela, Chief Executive Officer and President of TriSalus Life Sciences, added, "SD-101 represents an important component of the clinical program, studying the safety and effectiveness on activation of the immune system to help attack solid tumors. Building on the acquisition of our first therapeutic candidate, we will seek other immuno-oncology products which could benefit from being delivered directly into the disease site through acquisitions, licenses, and partnerships."

Steven Katz, MD, Chief Medical Advisor of TriSalus and Chairman of its Scientific Advisory Board, said, "As an investigational TLR9 agonist, SD-101 has shown promising immunostimulatory capabilities that promote anti-tumor activity in Phase 1b/2 clinical studies.1 By using Pressure-Enabled Drug Delivery, (PEDD), for intravascular, regional, rather than systemic delivery, our intent is to increase SD-101’s therapeutic index. Our clinical development program will initially evaluate SD-101 delivered with PEDD for treatment of uveal melanoma liver metastases. Concurrently, we will develop three additional programs assessing a combination of SD-101 and other immuno-oncology agents delivered with PEDD."

Richard Carvajal, MD, Director of Experimental Therapeutics and Director of the Melanoma Service at Columbia University Medical Center, concluded, "Based on the biology of uveal melanoma and the clinical results in cutaneous melanoma, delivery of TLR9 agonists into the liver has been quite challenging. I am excited about studying the combination of TriSalus’ delivery technology with SD-101 in uveal melanoma liver metastases."

The Company reported that it will seek a meeting with the Food and Drug Administration to share study designs and clinical plans for the initiation of human clinical studies in uveal melanoma liver metastases, the most common primary intraocular malignancy in adults, representing approximately 85% of all ocular melanomas.2

TriSalus was formed to investigate treatments to help stimulate the immune system to overcome immunosuppression by delivering a combination of immuno-oncology therapies directly to the site of disease. Trisalus is researching this multi-pronged approach to reprogram the immunosuppressive tumor microenvironment, harness the power of tumor killing agents, and deliver these therapies directly to the tumor through its Pressure-Enabled Drug Delivery (PEDD) approach. The Company has identified validated, IND-ready targets to acquire through license agreements, collaborations, or joint ventures. TriSalus is initially focused on the goal of successfully treating intractable solid tumors including uveal melanoma liver metastases and pancreatic cancer.

About SD-101

SD-101 is an investigational proprietary short sequence of synthetic deoxyribonucleic acid (DNA) which binds to the Toll-Like receptor 9 (TLR9) found on suppressive immune cells including myeloid-derived suppressor cells and antigen presenting cells.3 SD-101 has been evaluated in numerous clinical studies to assess safety and efficacy. Investigational studies suggest responsiveness to checkpoint inhibitors in PD-L1 negative tumors as well as inducing an influx of cytotoxic T cells and interferon gamma production.1

SD-101 will be evaluated in multiple visceral organ tumor types to assess its safety and activity via PEDD as well as in combination with other immunotherapies and modalities. For more information on SD-101 clinical trials that are currently recruiting patients, please visit www.clinicaltrials.gov.

About Pressure-Enabled Drug Delivery (PEDD)

Pressure-Enabled Drug Delivery (PEDD) approach with SmartValve technology features a self-expanding, nonocclusive, one-way valve which can infuse therapeutics into solid tumor vasculature 4 The FDA cleared SmartValve devices have been shown to deliver more therapy into the tumor while preventing embolic reflux5.

On September 24, 2020 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or the "Company") reported that its Board of Directors has approved a $25 million accelerated share repurchase ("ASR") transaction with JPMorgan Chase Bank, National Association ("JP Morgan") as part of the Company’s existing $160 million share repurchase program (Press release, Eagle Pharmaceuticals, SEP 24, 2020, View Source [SID1234565582]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The specific number of shares to be repurchased pursuant to the ASR is based on the average of the daily volume weighted average share prices of the Company’s common stock, less a discount, during the term of the ASR program. Based on yesterday’s closing price, the $25 million ASR would represent approximately 5% of the Company’s basic outstanding shares. Upon completion of the ASR, Eagle will have bought back a total of approximately $205 million of its stock since its IPO in 2014.

"The $25 million ASR reflects our ongoing confidence in our pipeline and continued earnings potential. With multiple opportunities to expand our RYANODEX franchise, the anticipated near-term launch of vasopressin, and three potential oncology launches in 2022 including PEMFEXY, fulvestrant and SM-88, along with the recent approval received by our Japanese marketing partner, SymBio, for its ready-to-dilute TREAKISYM product, we remain committed to building shareholder value," stated Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals.

Under the terms of the agreement, Eagle will pay $25 million to JP Morgan on September 24, 2020, and receive 505,817 shares, representing approximately 80% of the notional amount of the ASR, based on the closing price of $39.54 on September 23, 2020. Upon settlement of the ASR, the final number of shares repurchased will be trued up based on the average of the daily volume weighted average share prices of the Company’s common stock, less a discount, during the term of the accelerated share repurchase program. Eagle expects the ASR to be completed in the fourth quarter of 2020. As of September 23, 2020, the Company had 13.5 million common shares outstanding.

The Company intends to use cash on hand to fund the ASR program. As of June 30, 2020, cash and cash equivalents were $108.2 million, net accounts receivable was approximately $46.8 million, and debt was $37 million.