Satellite Companies

Clinical | SatCo | Preclinical

Therapeutic Area

Phase

Neuro
Drug
Target
Partner
Indication
P1
P2
P3
R
C

ATL1102

VLA-4

ATL

Duchenne Muscular Dystrophy

Generation 2.0 antisense drug

Indication*:

Duchenne Muscular Dystrophy (DMD)

Description/Summary:

For more information, please visit the Antisense Therapeutics (ATL) website.
* Safety and efficacy have not been evaluated by any regulatory authorities for the indications described
 
 
Severe & Rare

Our severe and rare disease franchise is the largest franchise in our pipeline. We believe that our antisense technology could offer effective therapies for patients with severe and rare diseases and neurological disorders that are life-threatening or fatal and for which there are limited treatment options. According to the National Institutes of Health, or NIH, there are approximately 5,000 to 8,000 rare diseases, many life-threatening or fatal. Unfortunately, patients with many of these severe and rare diseases have few effective therapies available. Since most severe and rare diseases are genetic or have a genetic component, parents often pass the disease to their children, creating a legacy of the disease and resulting in profound effects on the family. IONIS-SMNRx, the most advanced neurological drug in our pipeline, is now in two Phase 3 studies for the treatment of infants and children with SMA.

We are discovering and developing antisense drugs to treat severe and rare and neurological diseases for which there is a need for new treatment options. We have established strategic alliances in drug development areas that are high risk or in which our partners have significant expertise and resources to allow us to expand our drug discovery and development efforts beyond what we would choose to do internally. For example, our strategic partnerships with Biogen Idec and Roche have supported advancing five drugs for the treatment of neuromuscular or neurological diseases in our pipeline.

Due to the severe nature of these diseases and the lack of available treatments, there is an opportunity for more flexible and efficient development paths to the market. This means that, in some cases, the studies necessary for us to demonstrate proof-of-concept with a particular drug may also be the studies that complete our marketing registration package, thereby providing us with a relatively rapid path to market for potential new treatments for devastating and often fatal diseases.

Drug
Target
Partner
Indication
P1
P2
P3
R
C

CAMLIGOTM (alicaforsen)

ICAM-1

Atlantic

Pouchitis

Antisense drug

Indication*:

Pouchitis

Description/Summary:

For more information, please visit the Atlantic Healthcare website.

Selected Publications

  1. Barish, C.F. (2005) Alicaforsen therapy in inflammatory bowel disease. Expert Opin Biol Ther. 5, 1387-1391.
  2. Miner, P. et al. (2004) An enema formulation of alicaforsen, an antisense inhibitor of intercellular adhesion molecule-1, in the treatment of chronic, unremitting pouchitis. Aliment Pharmacol Ther. 19, 281-286.
  3. Philpott, J.R. & Miner, P.B. Jr. (2008) Antisense inhibition of ICAM-1 expression as therapy provides insight into basic inflammatory pathways through early experiences in IBD. Expert Opin Biol Ther. 8, 1627-1632.
  4. van Deventer, S.J. et al. (2006) A phase II dose ranging, double-blind, placebo-controlled study of alicaforsen enema in subjects with acute exacerbation of mild to moderate left-sided ulcerative colitis. Aliment Pharmacol Ther. 23, 1415-1425.
* Safety and efficacy have not been evaluated by any regulatory authorities for the indications described Back to top
 
 
 

RG-012

miR-21

Regulus

Alport Syndrome

Anti-miR oligonucleotide

Indication*:

Alport Syndrome

Description/Summary:

For more information, please visit the Regulus website.

Selected Publications

  1. Gomez, I.G. et al. (2005) Anti-microRNA-21 oligonucleotides prevent Alport nephropathy progression by stimulating metabolic pathways. J. Clin. Invest. Epub 2014 Nov 21.
  2. Kruegel, J., Rubel, D. and Gross, O. (2004) Alport syndrome--insights from basic and clinical research. Nat. Rev. Nephrol. 9, 170-178.
* Safety and efficacy have not been evaluated by any regulatory authorities for the indications described
 
 

RGLS4326

miR-17

Regulus

ADPKD

Anti-miR oligonucleotide

Indication*:

Autosomal dominant polycystic kidney disease (ADPKD)

Description/Summary:

For more information, please visit the Regulus website.
* Safety and efficacy have not been evaluated by any regulatory authorities for the indications described
 
Other

The broad applicability of our antisense technology allows us to create promising drugs and we have successfully developed novel drugs designed to treat many different diseases. In therapeutic areas that are outside of our core areas of development, we have licensed our drugs to highly focused satellite companies that have the specific expertise and resources to continue developing the drugs. Together with our partners we continue to advance drugs in clinical development that are outside of our core therapeutic areas, such as the ocular and antiviral drugs we and GSK are developing under our preferred partner collaboration.

Drug
Target
Partner
Indication
P1
P2
P3
R
C

ZEMDRITM (plazomicin)

Aminoglycoside

Achaogen

Complicated Urinary Tract Infections (cUTI)

Aminoglycoside

Indication*:

Complicated urinary tract infections (cUTI), including pyelonephritis, due to certain Enterobacteriaceae

Description/Summary:

For more information, please visit the ZEMDRI website.

Selected Publications

  1. Armstrong, E.S. & Miller, G.H. (2010) Combating evolution with intelligent design: the neoglycoside ACHN-490. Curr Opin Microbiol. 13, 565-573.
  2. Galani, I. et al. (2012) Activity of plazomicin (ACHN-490) against MDR clinical isolates of Klebsiella pneumoniae, Escherichia coli, and Enterobacter spp. from Athens, Greece. J Chemother. 24, 191-194.
  3. Walkty, A. et al. (2014) In vitro activity of plazomicin against 5,015 gram-negative and gram-positive clinical isolates obtained from patients in canadian hospitals as part of the CANWARD study, 2011-2012. Antimicrob Agents Chemother. 58, 2554-2563.
* Safety and efficacy have not been evaluated by any regulatory authorities for the indications describedBack to top