Chimerix, Inc. (NASDAQ: CMRX), announced its investigational broad-spectrum antiviral brincidofovir has been selected as one of two investigational agents to be evaluated in a clinical study in patients with confirmed Ebola Virus Disease in west Africa. Chimerix and the University of Oxford are in the process of finalizing a definitive agreement for supplying brincidofovir for the planned clinical trial.

With funding provided by the Wellcome Trust, the trial will be led by the University of Oxford on behalf of the International Severe Acute Respiratory and Emerging Infection Consortium (ISARIC), with operational support in west Africa provided by Medecins Sans Frontieres (MSF). The World Health Organization (WHO) and local health authorities are also participating in this clinical research. The west African trials will require the review and authorization of local health ministers and ethics boards prior to commencement.

"During what is perhaps the greatest acute public health crisis of our lifetimes, we want to recognize the profound sense of urgency and myriad scientific, medical, and clinical development issues being addressed together with multiple federal and international partners. We look forward to finalizing the trial arrangements with ISARIC as soon as possible to allow a better understanding of the potential benefit that brincidofovir could have in this Ebola Virus Disease outbreak," said M. Michelle Berrey, M.D., M.P.H., President and Chief Executive Officer of Chimerix.

In October, the U.S. Food and Drug Administration (FDA) authorized a Phase 2 (Study 205) single-arm study to evaluate the safety and antiviral activity of brincidofovir in subjects with confirmed Ebola Virus Disease.

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) and AstraZeneca (AZN) announced a strategic alliance to discover and develop novel delivery methods for antisense oligonucleotides. The new delivery approaches seek to target the desired tissue more effectively. The agreement builds on an existing collaboration between AstraZeneca and Isis Pharmaceuticals, a leader in the field of antisense, and supports AstraZeneca's research and development capabilities in the area of antisense oligonucleotide-based therapeutics and RNA biology. Initial project areas will be oncology and cardiovascular and metabolic diseases (CVMD).

Antisense oligonucleotides are short, single strands of DNA or RNA molecules. Rather than modulating the activity of already-formed proteins, antisense oligonucleotides act before proteins are produced at the level of messenger RNA in the cell, thus opening up new opportunities for therapeutic intervention. The new delivery methods will aim to enhance the access of antisense oligonucleotides into specific organs and cells. The methods build on Isis Pharmaceuticals' successful Ligand Conjugation Antisense (LICA) technology. The first example of this technology being Isis' GalNac-conjugated antisense oligonucleotides targeting liver hepatocytes, which lowers the therapeutic dose needed for liver targets by approximately 10-fold.

Under the terms of the agreement, each party will fund its own contribution and commit investigators to the collaboration. In line with AstraZeneca's open innovation approach, the companies will work together on an agreed programme and share rights to the results. Isis can apply learnings from this collaboration broadly across its antisense technology platform and AstraZeneca can similarly apply learnings across its broader RNA-based, small molecule and antibody research and development activities.

Isis Pharmaceuticals and AstraZeneca entered into a collaboration, development and license agreement in 2012 in oncology, subsequently expanded in 2013 to include CVMD. Under the 2012 agreement, one of the molecules being developed is AZD9150 (ISIS-STAT3Rx), a first-in-human, first-in-class, antisense oligonucleotide inhibitor of STAT3, which is being developed as an immunomodulatory agent in combination with MEDI4736, AstraZeneca's investigational anti-PD-L1 immune checkpoint inhibitor. A second product of that collaboration, an antisense oncology compound targeting the androgen receptor AZD5312 (ISIS-ARRx), is in Phase I trials. The new delivery collaboration announced today builds on this existing relationship and is expected to also bring benefits to these programs.

"This exciting collaboration very much supports AstraZeneca's research and development in the area of RNA-based therapeutics. If successful, we'll have a way to selectivity modulate therapeutic targets in specific cell types that are intractable to small molecules and antibodies. This could lead to a number of ground breaking drugs for both oncology and cardiovascular and metabolic diseases," said Susan Galbraith, Head of the Oncology Innovative Medicines Unit, AstraZeneca.

Brett Monia, Senior Vice President of Antisense Drug Discovery, Isis Pharmaceuticals said, "The collaboration expansion announced today builds upon an already successful agreement between Isis and AstraZeneca. Together, we have advanced ISIS-STAT3Rx and ISIS-ARRx in clinical development, both of which are being evaluated in patients with cancer. This opportunity also complements our internal efforts to expand the use of our technology and develop drugs with broad therapeutic applicability."

"RNA molecules play an increasingly important role in our research portfolio. We are delighted to be expanding our existing, strong collaboration with Isis Pharmaceuticals, who are leading players in RNA biology, with the aim of improving the delivery of antisense oligonucleotides to specific cardiovascular and metabolic tissue targets," said Marcus Schindler, Head of the CVMD Innovative Medicines Unit, AstraZeneca.