Navigation Links
Isis Initates Phase 2/3 Study Of ISIS-TTR Rx And Earns $7.5 Million Milestone Payment From GlaxoSmithKline
Date:2/19/2013

CARLSBAD, Calif., Feb. 19, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $7.5 million milestone payment from GlaxoSmithKline (GSK) related to the initiation of a Phase 2/3 clinical study for ISIS-TTRRx.  ISIS-TTRRx is an antisense drug in development with GlaxoSmithKline (GSK) for the treatment of transthyretin (TTR) amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.  Isis and GSK recently amended the clinical development plan and financial terms relating to ISIS-TTRRx to support this registration-directed Phase 2/3 clinical study on ISIS-TTRRx.

"TTR amyloidosis is a severe and rare genetic disease that leads to death.  TTR amyloidosis affects approximately 50,000 patients worldwide and current treatments are limited.  Patients with familial amyloid polyneuropathy, or FAP, experience TTR build up in their peripheral nerves and experience the loss of motor functions, such as walking," said Merrill D. Benson , M.D., Professor of Medical Genetics at Indiana University.  "By inhibiting the production of TTR protein, ISIS-TTRRx could offer an alternative approach to treating patients with FAP.  Furthermore, a drug that can reduce TTR levels could potentially slow or arrest progression of this terrible disease."

The Phase 2/3 study of ISIS-TTRRx is a randomized, double-blinded, placebo-controlled, international study designed to support an application for marketing approval of ISIS-TTRRx in patients with FAP.  The fifteen month study will enroll approximately 200 patients randomized 2:1 to receive 300 mg/week of ISIS-TTRRx or placebo and will measure the effects of ISIS-TTRRx on neurological dysfunction and on quality-of-life.  The United States Food and Drug administration granted ISIS-TTRRx fast track designation and orphan drug status for the treatment of FAP.

"The rapid development of ISIS-TTRRx from a research-stage program to a drug in late-stage clinical development in just over two years represents the strong commitment of both teams to bring this novel drug to patients who are in need of new therapeutic options," said B. Lynne Parshall , chief operating officer at Isis.  "The encouraging data from our Phase 1 study, in which our drug was well tolerated and produced significant reductions in TTR protein, supported the advancement of ISIS-TTRRx directly into this registration-directed Phase 2/3 study.  While we will continue to manage the clinical development of ISIS-TTRRx, we will benefit from GSK's late-stage clinical development, regulatory and commercial expertise."

ISIS-TTRRx is part of the Isis-GSK strategic alliance to develop RNA therapeutics for rare and infectious diseases.  For the initiation of the Phase 2/3 study, Isis will receive a $7.5 million milestone payment and is eligible to earn an additional $50 million in pre-licensing milestone payments to support the Phase 2/3 study of ISIS-TTRRx­.  In addition, Isis is eligible to receive regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTRRx.

ABOUT ISIS-TTRRx
Transthyretin amyloidosis is a genetic disease in which the patient inherits a mutant gene that produces a misfolded form of TTR, which progressively accumulates in tissues, impairing their function.  In patients with transthyretin amyloidosis, both the mutant and normal forms of TTR can build up as fibrils in tissues, including heart, peripheral nerves, and the gastrointestinal tract.  The presence of TTR aggregates interferes with the normal functions of these tissues, and as the TTR protein aggregates enlarge more tissue damage occurs and the disease worsens.  There are two common types of transthyretin amyloidosis, familial amyloid cardiomyopathy, or FAC, which affects more than 40,000 patients worldwide, and FAP, which affects more than 10,000 patients worldwide.  Patients with FAC have TTR build up in the heart muscle and succumb to heart failure five to six years after symptom onset.  Patients with FAP have TTR build up in peripheral nerve tissue leading to the loss of nerve function and wasting.  ISIS-TTRRx is an investigational drug that is designed to inhibit the production of all forms of TTR, and could offer an alternative approach to treat all types of transthyretin-related amyloidosis. 

ABOUT ISIS PHARMACEUTICALS, INC.
Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners.  Isis' broad pipeline consists of 28 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, and cancer.  Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO™, in the United States for the treatment of patients with HoFH.  Genzyme is also pursuing marketing approval of KYNAMRO in other markets, including Europe.  Isis' patents provide strong and extensive protection for its drugs and technology.  Additional information about Isis is available at www.isispharm.com.

ISIS PHARMACEUTICALS' FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding Isis' collaboration with GlaxoSmithKline, the discovery, development and potential of drugs for severe and rare diseases, and the development, activity, therapeutic potential and safety of ISIS-TTRRx. Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of KYNAMRO, is a forward-looking statement and should be considered an at-risk statement.  Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs.  Isis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements.  Although Isis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis.  As a result, you are cautioned not to rely on these forward-looking statements.  These and other risks concerning Isis' programs are described in additional detail in Isis' annual report on Form 10-K for the year ended December 31, 2011 and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.

In this press release, unless the context requires otherwise, "Isis," "Company," "we," "our," and "us" refers to Isis Pharmaceuticals and its subsidiaries.

Isis Pharmaceuticals® is a registered trademark of Isis Pharmaceuticals, Inc.  KYNAMRO™ is a trademark of Genzyme Corporation.


'/>"/>
SOURCE Isis Pharmaceuticals, Inc.
Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. Edison Pharmaceuticals & Bambino Gesu Childrens Hospital Announce Initiation of EPI-743 Phase 2 Cobalamin C Deficiency Syndrome Clinical Trial
2. Neurim Pharmaceuticals Announces Positive Phase 2 Clinical Trial Results of Piromelatine for the Treatment of Insomnia
3. NW Bio Takes Next Steps In Moving Forward With Its Phase III Brain Cancer Trial In Europe
4. Ferring to Highlight Phase 3 Data for Cervical Ripening Candidate at Society for Maternal-Fetal Medicine Annual Meeting
5. RNL BIO filed Phase II/III Clinical Trial with KFDA to Treat Cerebral Palsy Using Autologous Fat Derived Stem Cells
6. Lilly Discontinues Phase 3 Rheumatoid Arthritis Program for Tabalumab Based on Efficacy Results
7. Matrixx Initiatives Withdraws Acquisition Offer for ProPhase Labs
8. Cytos VLP Platform is Basis for Phase 1 Clinical Trial with Anti-IgE Vaccine
9. Vanda Announces Phase IIb/III Clinical Study In Major Depressive Disorder Did Not Meet The Primary Endpoint
10. Targovax AS Announces First Patient Included in Phase I/II Clinical Study With the Therapeutic Cancer Vaccine TG01 in Operable Pancreatic Cancer
11. Cell Therapeutics Announces Data Safety and Monitoring Board Recommendation to Continue the GOG-0212 Phase 3 Clinical Trial of OPAXIOTM as Maintenance Therapy in Ovarian Cancer
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/9/2017)... 9, 2017 Zimmer Biomet Holdings, Inc. (NYSE ... today announced it has earned a spot on ... The Company was ranked among 500 U.S. employers as ... Healthcare Equipment and Services. The annual ... anonymous, independent survey of over 30,000 employees across 25 ...
(Date:5/9/2017)... 2017  Semler Scientific, Inc. (OTCQB: SMLR), an ... improve the clinical effectiveness and efficiency of healthcare ... quarter ended March 31, 2017. ... to identify when preventive care options are appropriate, ... heart attacks or strokes occur," said Doug ...
(Date:5/6/2017)... , May 5, 2017   Provista , a proven ... than 200,000 customers, today announced Jim Cunniff as ... of executive and business experience to Provista, including most recently ... in California . He assumed his new ... is a great fit for Provista," says Jody Hatcher ...
Breaking Medicine Technology:
(Date:5/26/2017)... ... 2017 , ... After raising nearly $30,000 on Kickstarter , about three-times ... at a discounted crowdfunding price on Indiegogo . , “Along with creating an ... to bring a fidget toy to the market that was made of superior quality ...
(Date:5/26/2017)... ... May 26, 2017 , ... “When the Stars Lead Home”: a poignant ... of published author Laura Weigel Douglas, an avid reader who lives in the Pacific ... sometimes feels like Green Hills Adventure Camp. She couldn’t be more grateful. , Twelve-year ...
(Date:5/26/2017)... (PRWEB) , ... May 26, 2017 , ... Water damage ... Lee, New Jersey School District had left education officials with a number of critical ... replacement of the flooring had to be accomplished with little or no disruption to ...
(Date:5/24/2017)... ... 24, 2017 , ... NucleusHealth ™, advancing clinical practice ... Food and Drug Administration (FDA) 510(k) clearance for its Nucleus.io ... for medical image management. At the core is patented streaming technology that provides ...
(Date:5/24/2017)... ... May 24, 2017 , ... Myers Jackson is well ... the ability to sell luxury homes anywhere on the planet. The luxury home market ... from Hattiesburg to Houston city-scapes. A quick search of “11 Spyglass Hill Auction will ...
Breaking Medicine News(10 mins):