On December 11, 2019 Exicure, Inc. (NASDAQ:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported an update from its Phase 1b/2 trial with AST-008 in patients with solid tumors (Press release, Exicure, DEC 11, 2019, View Source [SID1234552266]). AST-008 is an investigational SNA consisting of toll-like receptor 9 (TLR9) agonists designed for immuno-oncology application, and is being evaluated in combination with pembrolizumab in patients with solid tumors.

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"We’re very pleased with the preliminary data generated so far in our ongoing Phase 1b/2 trial and we look forward to enrolling patients in the Phase 2 portion of the trial," said Dr. David Giljohann, CEO of Exicure. "Based on these results, we are considering adding additional cohorts to the trial in other cancers where AST-008 may have benefit," concluded Dr. Giljohann.

The primary objective of the dose escalation portion of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AST-008 alone and in combination with pembrolizumab, and to produce a recommended Phase 2 dose. Fourteen patients have been enrolled and dosed with AST-008. No treatment-related serious adverse events or dose-limiting toxicities have been observed. The fifth and final dose escalation cohort is now open and enrolling.

The study has enrolled five melanoma patients, four Merkel cell carcinoma (MCC) patients, two cutaneous squamous cell carcinoma patients, two head and neck squamous cell carcinoma patients, and one mucosal melanoma patient. Prior to enrolling, most patients had progressive disease on anti-PD-(L)1 antibodies.

Available data show:

AST-008 administration, alone or in combination with pembrolizumab, produced cytokine and chemokine expression and immune cell activation in patient blood indicative of desired immune activation.
Of the 4 MCC patients, one patient, which had previously progressed on anti-PD-1 antibody therapy, has confirmed stable disease with decreased target lesion diameters for a period in excess of twelve weeks, while a second MCC patient experienced a target lesion complete response and a confirmed overall partial response longer than 24 weeks.
Nine patients had progressive disease, two patients have not yet been evaluated and one is not evaluable.
"The initial results are highly encouraging and warrant expansion of the trial, and I’m looking forward to participating," stated Dr. Steven O’Day, Executive Director of the John Wayne Cancer Institute and Cancer Clinic, and a principal investigator on the study.

Detailed results are expected to be presented at major upcoming oncology meetings. Based on these early results, showing positive biomarker data and initial tumor responses, Exicure anticipates enrolling MCC patients, which have previously failed anti PD-1/PD-L1 therapy, in its Phase 2 study during the first quarter of 2020.

On December 11, 2019 Eli Lilly and Company (NYSE: LLY) reported the opening of the LIBRETTO-431 clinical trial [NCT04194944] for selpercatinib, also known as LOXO-292, for treatment-naïve RET fusion-positive non-small cell lung cancer (NSCLC) patients (Press release, Eli Lilly, DEC 11, 2019, View Source [SID1234552245]). Enrolled trial participants will be randomized to receive either selpercatinib or platinum-based (carboplatin or cisplatin) and pemetrexed therapy with or without pembrolizumab as initial treatment of their advanced or metastatic RET fusion-positive NSCLC.

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"Given the remarkable results of the LIBRETTO-001 trial, I am excited to open this important Phase 3 trial of selpercatinib, a highly selective and potent molecule that has previously demonstrated sustained responses with a well-tolerated safety profile," said Professor Ben Solomon, principal investigator at the Peter MacCallum Cancer Centre in Melbourne Australia. "This trial endeavors to generate outcome data that place patients with RET fusions alongside those with EGFR mutations and ALK fusions, as driver-positive populations that should be treated with targeted therapies in the first-line setting, rather than chemoimmunotherapy."

"This is an important milestone in the journey to further demonstrate the benefit of selpercatinib and the potential for people living with advanced or metastatic RET fusion-positive non-small cell lung cancer in the first-line setting against the current standard of care," said Anne White, president of Lilly Oncology. "Launching a trial of this size underscores the importance of now including RET fusions in the paradigm of genomic testing in lung cancer."

Trial Background
LIBRETTO-431 is a randomized Phase 3 clinical trial of patients with treatment-naïve RET fusion-positive NSCLC. The trial will enroll 400 patients with advanced or metastatic RET fusion-positive NSCLC who have received no prior systemic therapy for metastatic disease. Enrolled trial participants will be randomized 1:1 to receive either selpercatinib or platinum-based (carboplatin or cisplatin) and pemetrexed therapy with or without pembrolizumab as initial treatment of their advanced or metastatic RET fusion-positive NSCLC. RET fusions may be identified using local testing. This trial’s primary endpoint is progression-free survival (PFS) and secondary endpoints include overall survival (OS), overall response rate (ORR), duration of response (DoR), and intracranial ORR. For patients randomized to the control arm, crossover is allowed at progression.

About Selpercatinib (LOXO-292)
Selpercatinib, also known as LOXO-292, is a highly selective and potent, oral investigational new medicine in clinical development for the treatment of patients with cancers that harbor abnormalities in the rearranged during transfection (RET) kinase. RET fusions and mutations occur across multiple tumor types with varying frequency. Selpercatinib was designed to inhibit native RET signaling as well as anticipated acquired resistance mechanisms.

Selpercatinib has received breakthrough designations in RET fusion-positive NSCLC, RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancers.

About RET-Altered Cancers
Genomic alterations in RET kinase, which include fusions and activating point mutations, lead to overactive RET signaling and uncontrolled cell growth. RET fusions have been identified in approximately 2 percent of non-small cell lung cancer, 10-20 percent of papillary and other thyroid cancers and a subset of other cancers. Activating RET point mutations account for approximately 60 percent of MTC. RET fusion cancers and RET-mutant MTC are primarily dependent on this single activated kinase for their proliferation and survival. This dependency, often referred to as "oncogene addiction," renders such tumors highly susceptible to small molecule inhibitors targeting RET.

On December 11, 2019 Novigenix SA, a leading Immuno-Transcriptomics company that develops and commercializes solutions for early cancer detection and precision medicine, reported the development of a new immune cell type specific Immuno-Transcriptomic signature for the detection of colorectal cancer (CRC) in blood (Press release, Novigenix, DEC 11, 2019, View Source [SID1234552267]). The data were presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Immuno-Oncology Congress (11-14 December, Geneva, Switzerland).

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Immune cells play a key role in cancer progression. Their response to the tumor can promote or prevent tumor growth and can be used to increase the diagnostic power of a cancer detection test. In this study, RNA signatures specific for different immune cell types (neutrophils, monocyte, T cells, CD4, CD8, B cells, NK cells) were compiled from published data and tested on the RNA-seq transcriptome profiles of 561 peripheral blood mononuclear cells (PBMC) samples from 218 healthy donors, 189 CRC, 115 advanced adenomas and 39 other cancers patients. The signature analysis was performed on Novigenix’s LITOseek sequencing platform.

Myeloid cell (monocyte and neutrophil) RNA signatures were strongly upregulated in CRC compared to control samples (p<0.01), whereas the T-cell signature was significantly downregulated (p<0.01). The specificity of the different RNA signatures was demonstrated by their correlation with the respective cell count, performed with traditional method.

"These are very encouraging data on the utility of immune cell type specific RNA signature as biomarkers for CRC detection in blood," said Prof. Dr. G. Dorta from the Department of Gastroenterology and Hepatology at CHUV in Lausanne, Switzerland. "The correlation of the cell type specific RNA signature with traditional cell counting as well as the CRC discriminant power could be very valuable for the development of an early CRC detection test."

"This newly developed cell type specific RNA signature demonstrated strong differentiation power between CRC and other cancers," said Dr. Jan Groen, CEO of Novigenix. "Therefore, we are evaluating how a combination of cell type specific RNA signatures with a CRC specific profile could be incorporated into a test for the early diagnosis of colon cancer."

About LITOseek

Novigenix’s Blood Immuno-Transcriptomic sequence platform, LITOseek, analyzes the gene expression modifications (mRNA signatures) induced by the host response to various triggers, such as the onset of cancer. Disease specific algorithms are developed combining the mRNA signature with clinical and medical parameters. The combination of mathematical models with machine learning and collection of new data enables the continuous improvement of the predictive and adaptive algorithms.

On December 11, 2019 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported the appointment of Shehnaaz Suliman, M.D., MBA, M.Phil., as the company’s president and chief operating officer (Press release, Alector, DEC 11, 2019, View Source [SID1234552251]). In this new role, Dr. Suliman will oversee Alector’s day-to-day operations, including preclinical development, program management, and strategic and administrative functions. Dr. Suliman will report to Arnon Rosenthal, Ph.D., chief executive officer of Alector.

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"Shehnaaz’s background and expertise in drug development, strategy, portfolio management, and operational leadership uniquely position her to bring greater integration to our operations. Her strong track record of building and leading teams that can support the development of life-changing medicines comes at a key inflection point for Alector as we prepare to launch our first registrational trial in 2020," said Dr. Rosenthal. "Shehnaaz’s core leadership strengths will be invaluable to our entire organization, and we look forward to leveraging her insights as a thought partner and leader at Alector."

"Neurodegeneration affects millions of patients and families worldwide, and I am proud to be supporting Alector’s mission to find a cure for these diseases. As the leader in immuno-neurology, Alector has made substantial progress in rapidly advancing a broad clinical portfolio of novel and innovative programs," said Dr. Suliman. "As we advance our lead programs into late-stage clinical development and continue to progress our research pipeline, I look forward to partnering with the team to further our vision for finding cures for neurodegenerative diseases. I am excited to be joining the company at this important time and look forward to helping Alector execute our objectives and scale our organization effectively."

Dr. Suliman brings over 20 years of business development, drug development, strategic and operational expertise, and executive leadership skills to Alector. She joins Alector from Theravance Biopharma, where she served as senior vice president, corporate development and strategy, a position she held from July 2017 to March 2019. Prior to Theravance, Dr. Suliman worked for Genentech as a group leader and project team leader in the R&D Portfolio Management and Operations Group from September 2010 to May 2015 where she led and oversaw a number of neuroscience development programs. She was also vice president and global therapeutic head, Roche Partnering from June 2015 to July 2017. Dr. Suliman held various management roles of increasing responsibility at Gilead Sciences between January 2005 and September 2010. Before Gilead, Dr. Suliman was an investment banker with Lehman Brothers and Petkevich & Partners, advising public and private companies on buy- and sell-side transactions. She is a member of the board of directors of Ultragenyx Pharmaceutical, a biopharmaceutical company, and 10X Genomics, a life science technology company. Dr. Suliman received her M.D. from the University of Cape Town Medical School, South Africa, and holds an MBA, with distinction, and M.Phil. in development studies from Oxford University, where she was a Rhodes Scholar.

On December 11, 2019 Phoenix Biotechnology, Inc. ("Phoenix" or the "Company" www.phoenixbiotechnology.com) reported that it has completed the private placement of 1,373,400 shares of common stock for gross proceeds of $4,120,200 (Press release, Phoenix Biotechnology, DEC 11, 2019, View Source [SID1234552268]).

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The gross proceeds of the offering are expected to be used for general corporate purposes including funding Phoenix’s continuing research at The University of Texas M.D. Anderson Cancer Center, University of Nebraska and University of L’Aquila (Italy); filing and prosecuting the Company’s U.S. and international patent portfolio; and paying general and administrative expenses.

Robert A. Newman, Ph.D., President and Chief Science Officer, said, "We are grateful to have received significant new resources for our continued research efforts. The ongoing research of our lead drug against glioblastoma appears to be very promising. In addition, we are proud of the research recently published of our drug’s efficacy against HTLV-1, a virus that affects millions of people with devastating declines in their quality of life. All of our work is aimed at providing hope for those afflicted with serious diseases for which there are few, if any, meaningful therapeutic options. The funds recently raised will assist us in continuing this important work."

The shares of common stock have not been registered under the Securities Act of 1933, as amended, and may not be offered or sold except pursuant to an exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and applicable state securities laws. This news release does not constitute an offer to sell or the solicitation of an offer to buy any security and shall not constitute an offer, solicitation or sale in any jurisdiction in which it would be unlawful.