On September 28, 2021 The University of Texas MD Anderson Cancer Center and BostonGene Corporation, a biomedical software company committed to defining optimal precision medicine-based therapies for cancer patients, reported a strategic alliance to advance the development and clinical integration of multiplatform biomarker signatures (Press release, MD Anderson, SEP 28, 2021, View Source [SID1234590415]).

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The alliance brings together BostonGene’s innovative computational platform and CLIA-certified and CAP-accredited high complexity molecular laboratory with the clinical and translational research expertise of MD Anderson, leveraging scientific discovery, analytical advancement and powerful computing to develop clinically applicable solutions for patients with cancer. BostonGene’s computational platform evaluates myriad molecular and immune-based parameters to discover correlations between tumor genomics, a patient’s immune system and the effectiveness of new and emerging treatments. This agreement expands upon existing research collaborations between BostonGene and multiple MD Anderson researchers.

"As we gain deeper insights into the drivers behind each cancer, actionable biomarkers are increasingly important to inform clinical decisions and provide individualized treatments," said Funda Meric-Bernstam, M.D., Chair of Investigational Cancer Therapeutics at MD Anderson. "Through our collaboration with BostonGene, we will utilize multi-parameter discovery, big data analytics and computational support to advance and validate novel diagnostic and therapeutic biomarkers that we hope will guide physicians to deliver optimal care to each patient."

Under the terms of the agreement, BostonGene and MD Anderson will collaborate with the goal of translating unique research findings into multiple clinically actionable tests. BostonGene and MD Anderson expect to validate promising clinical biomarker targets through retrospective and prospective MD Anderson investigator-sponsored trials for patients with common and rare cancers. MD Anderson is eligible to receive certain payments based on the achievement of certain clinical milestones and commercial sales. As part of the research alliance, MD Anderson and BostonGene will work together on clinical utility studies designed to support incorporation of BostonGene testing into national guidelines and adoption as a national standard of care.

"Implementing cutting-edge technologies is a critical component in MD Anderson’s approach to improving care for our patients. This agreement with BostonGene further demonstrates our commitment to advancing the adoption of precision medicine through focused and strategic collaborations," said Ferran Prat, PhD., J.D., Senior Vice President for Research Administration and Industry Relationships at MD Anderson.

"We are proud to collaborate with MD Anderson in the pursuit of identifying optimal treatment options for cancer patients," said Andrew Feinberg, President and CEO of BostonGene. "This new agreement reinforces our combined commitment to transforming and improving patient care."

In addition to the research alliance, BostonGene recently entered into a laboratory services agreement with MD Anderson to offer BostonGene Tumor Portrait Tests, available to physicians to order through the electronic medical records system for patients meeting test requirements.

Disclosures
MD Anderson has an institutional conflict of interest with BostonGene through this strategic alliance and is implementing an Institutional Conflict of Interest Management and Monitoring Plan for all research related to this agreement.

On September 28, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase"), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, reported that Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase, will participate in a fireside chat at the virtual Chardan 5th Annual Genetic Medicines Conference being held October 4 – 5 (Press release, NeuBase Therapeutics, SEP 28, 2021, View Source [SID1234590450]).

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Chardan 5th Annual Genetic Medicines Conference
Date: Tuesday, October 5th
Time: 11:30 a.m. ET
Location: Webcast link or at the company’s website

On September 27, 2021 Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, reported the financial results for Nicox SA and its subsidiaries (the "Nicox Group") for the six months ending June 30, 2021 and provided an update on recent events as well as progress on key programs (Press release, NicOx, SEP 27, 2021, View Source [SID1234590282]).

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First Half 2021 Financial Highlights
Net revenue1 for the first half of 2021 was €1.3 million (including €1.2 million in royalty revenue) versus €2.4 million (including €1.4 million in royalty revenue) for the first half of 2020. Operating expenses for the first half of 2021 were €13.3 million compared to €10.2 million for the first half of 2020.

The Nicox Group recorded a net loss of €11.7 million for the six months ended June 30, 2021, compared to a net loss of €14.6 million for the same period in 2020.
As of June 30, 2021, the Nicox Group had cash and cash equivalents of €36.5 million, as compared with €42.0 million at March 31, 2021 and €47.2 million at December 31, 2020. The Company is financed for at least 12 months, based on the development of NCX 470 alone.
As of June 30, 2021, the Nicox Group had financial debt of €18.0 million consisting of €16.0 million in the form of a bond financing agreement with Kreos Capital signed in January 2019 and a €2 million credit agreement guaranteed by the French State and granted in August 2020 in the context of the COVID-19 pandemic. Details of the bond financing agreement can be found in the Press Release of January 29, 2021.
Recent Events
The Mississippi Phase 2b clinical trial of NCX 4251 ophthalmic suspension 0.1%, evaluated against placebo, while not meeting the pre-defined blepharitis primary endpoint, demonstrated statistically significant results on blepharitis signs and symptoms when measured as change from baseline. NCX 4251 was found to be safe and well-tolerated over 14 days’ treatment, with no serious adverse events (see Press Release of September 23, 2021).
VYZULTA (latanoprostene bunod ophthalmic solution), 0.024%, recently received regulatory approval in Brazil, Jordan and Singapore. Further approvals and launches of VYZULTA will now be reported by our global partner Bausch + Lomb on a quarterly basis.
José Boyer has retired as Interim Head of R&D, although he will continue to advise the Company as a consultant. We would like to thank him for his commitment and contribution to Nicox.
Fera’s resubmitted application for Orphan Drug Designation (ODD) for naproxcinod in sickle-cell disease has been refused by the U.S. Food and Drug Administration (FDA), and Fera is currently seeking to discuss the next steps with the FDA. Fera continues to review other undisclosed therapeutic indications for naproxcinod.
Key Programs Updates
NCX 470: The New Drug Application submission timing to the U.S. FDA remains in line with our expectations, driven by the end of 2023 date for results from the ongoing Denali Phase 3 clinical In the first Phase 3 clinical trial, Mont Blanc, 494 out of 670 patients (74%) had been randomized as of September 20, and 398 of those patients have completed the 3-month efficacy evaluation. Given recruitment continues to be impacted by COVID-19, we are prudently moving the expectation for Mont Blanc trial top-line results to Q1 2023 (previously Q2 2022). The Phase 3 program, evaluating NCX 470 for lowering of intraocular pressure in patients with open-angle glaucoma or ocular hypertension, is expected to support NDA submissions in the U.S. and China, and will also provide data for countries accepting the same package for approval.
NCX 4251: Following the results of the Phase 2b Mississippi trial, the Company plans to meet with the U.S. FDA in early 2022 to discuss next steps in the development of this innovative product candidate for blepharitis.
ZERVIATETM in China: A Phase 3 clinical trial intended to support an application for regulatory approval in China, conducted and financed by our partner Ocumension, is ongoing.
Only figures at December 31, 2020 are audited, all other figures of this press release are non-audited.

Notes
Net revenue consists of revenue from collaborations less royalty payments which we refer to as net profit from collaborations in the condensed consolidated statements of profit or loss for the six-month period ended June 30, 2021.

On September 27, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company using its ARCUS genome editing platform to develop allogeneic CAR T and in vivo gene editing therapies, reported that Michael Amoroso has been named as the Company’s President and Chief Executive Officer and a member of the Precision BioSciences Board of Directors, in each case effective October 15, 2021 (Press release, Precision Biosciences, SEP 27, 2021, View Source [SID1234590299]). Mr. Amoroso will succeed Matt Kane, Co-Founder, President and Chief Executive Officer, who is expected to serve as an advisor to the Company to help ensure a smooth transition.

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Mr. Amoroso is a biotech CEO with significant operational experience leading organizations focused on cell and gene therapies. He brings extensive experience leading teams across research, clinical development, regulatory and medical affairs, and commercial operations, with direct operational experience in the U.S. and major world markets with a particular focus on oncology drugs, including CAR T cell therapies for hematologic malignancies.

"We are very pleased that Michael will lead the Precision BioSciences team as we embark on the next phase of the company’s growth and seek to maximize the opportunities for our ARCUS platform," said Kevin Buehler, Chair of Precision BioSciences Board of Directors. "Michael’s deep commercial and operational expertise in hematology, oncology, and gene therapy is an excellent fit for Precision and very relevant as we develop our strategy for late-stage clinical development and ultimately aim for commercialization. We believe that Michael’s priorities and motivations align well with the culture at Precision BioSciences and position him to successfully lead the Precision team as we pursue novel treatments to overcome cancer and cure genetic diseases."

Mr. Amoroso is currently President and Chief Executive Officer of Abeona Therapeutics, Inc., a fully-integrated gene and cell therapy company. Prior to this role, Mr. Amoroso served as Abeona’s Chief Commercial Officer and Chief Operating Officer, responsible for overseeing the operational management of Abeona, including research and clinical development, regulatory, medical, commercial, corporate affairs, and business development. Previously, Mr. Amoroso was the Senior Vice President and Chief Commercial Officer at Kite Pharma, Inc. He held various senior level executive positions at leading biopharmaceutical companies, including Eisai Inc., Celgene Corporation, and began his biopharmaceutical career at Aventis (now Sanofi S.A.). Mr. Amoroso earned his Executive M.B.A. in Management from the Stern School of Business, New York University, and his B.A. in Biological Sciences, summa cum laude, from Rider University.

"I am excited to join Precision BioSciences as its next President and Chief Executive Officer and to advance Precision’s novel genome editing platform, ARCUS, in two cutting edge sectors of biotech, allogeneic CAR T therapy and in vivo gene editing. I look forward to working alongside co-founders Derek Jantz, Chief Scientific Officer and Jeff Smith, Chief Technology Officer, and the team of Precisioneers who are driving the development of our potentially breakthrough treatments with their perseverance and continuous technical innovation. I’m particularly optimistic about the potential opportunity to bring novel cell therapies to patients who suffer from hematologic malignancies, and about the prospects of filing three Investigational New Drug/Clinical Trial applications in the next three years for our gene editing treatments," commented Mr. Amoroso.

Mr. Buehler added, "Over the last 15 years, Matt has played a critical role in the formation, development, and growth of Precision BioSciences. The Board of Directors sincerely thank him for his commitment to the company and the significant accomplishments made by the Precision team under his leadership."

On September 27, 2021 CytoDyn Inc. (OTCQB: CYDY) ("CytoDyn" or the "Company"), a late-stage biotechnology company developing leronlimab, a CCR5 antagonist with the potential for multiple therapeutic indications, reported that Nader Pourhassan, Ph.D., President and Chief Executive Officer and Scott Kelly, M.D., Chairman, Chief Medical Officer and Head of Business Development of CytoDyn will provide a comprehensive business update at the upcoming Emerging Growth Conference (Press release, CytoDyn, SEP 27, 2021, View Source [SID1234590334]). CytoDyn will present for 20 minutes, and 40 minutes will be dedicated to live questions and answers.

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Interested participants are encouraged to login early prior to the start of the event. This is a livestream presentation, and a link will also be posted on CytoDyn’s website approximately 48 hours after the presentation. The conference sponsor provides corporate visibility services to CytoDyn for a fee.

About Leronlimab

The U.S. Food and Drug Administration (FDA) granted CytoDyn Fast Track designation to explore two potential indications using leronlimab to treat Human Immunodeficiency Virus (HIV) and metastatic cancer. The first indication is combination therapy with HAART for HIV-infected patients, and the second is for metastatic triple-negative breast cancer (mTNBC). Leronlimab is an investigational humanized IgG4 mAb that binds to CCR5, a cellular receptor important in HIV infection, tumor metastases, and other diseases, including nonalcoholic steatohepatitis (NASH). Leronlimab has been studied in 16 clinical trials involving more than 1,200 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab combined with HIV standard care in patients with multi-drug resistance to current available classes of HIV drugs).

Leronlimab, among various potential applications, is a viral-entry inhibitor in HIV/AIDS. It binds to CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab does not work on other strains of HIV (for example X4), however, R5 is the most dominant strain of HIV. Five clinical trials have demonstrated leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent with fewer side effects and less frequent dosing requirements than currently used daily drug therapies. Cancer research has shown CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control (for example, through angiogenesis). Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 97% in a murine xenograft model. As a result, CytoDyn is conducting two clinical trials, one, a Phase 2 in mTNBC, which was granted Fast Track designation by the FDA in 2019, and a second, a Phase 2, basket trial which encompasses 22 different solid tumor cancers.

The CCR5 receptor plays a central role in modulating immune cell trafficking to sites of inflammation. After completing two clinical trials with COVID-19 patients (a Phase 2 and a Phase 3), CytoDyn initiated a Phase 2 investigative trial for post-acute sequelae of SARS COV-2 (PASC), also known as COVID-19 Long-Haulers. This trial evaluated the effect of leronlimab on clinical symptoms and laboratory biomarkers to further understand the pathophysiology of PASC. It is currently estimated that between 10-30% of those infected with COVID-19 develop long-term sequelae. Common symptoms include fatigue, cognitive impairment, sleep disorders, and shortness of breath. CytoDyn plans to pursue clinical trials to evaluate leronlimab’s effect on immunological dysregulation in other post-viral syndromes, including myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS).

CytoDyn is also conducting a Phase 2 clinical trial for NASH to evaluate the effect of leronlimab on liver steatosis and fibrosis. Pre-clinical studies revealed a significant reduction in NAFLD and a reduction in liver fibrosis using leronlimab. There are currently no FDA approved treatments for NASH, which is a leading cause of liver transplant. About 30 to 40 percent of adults in the U.S. live with NAFLD, and 3 to 12 percent of adults in the U.S. live with NASH. There have been no strong safety signals identified in patients administered leronlimab in multiple disease spectrums, including patients with HIV, COVID-19, and oncology.