U.S. markets closed
  • S&P 500

    3,638.35
    +8.70 (+0.24%)
     
  • Dow 30

    29,910.37
    +37.90 (+0.13%)
     
  • Nasdaq

    12,205.85
    +111.44 (+0.92%)
     
  • Russell 2000

    1,855.27
    +10.25 (+0.56%)
     
  • Crude Oil

    45.53
    -0.18 (-0.39%)
     
  • Gold

    1,788.10
    -23.10 (-1.28%)
     
  • Silver

    22.64
    -0.81 (-3.44%)
     
  • EUR/USD

    1.1970
    +0.0057 (+0.4788%)
     
  • 10-Yr Bond

    0.8420
    -0.0360 (-4.10%)
     
  • Vix

    20.84
    -0.41 (-1.93%)
     
  • GBP/USD

    1.3314
    -0.0042 (-0.3169%)
     
  • USD/JPY

    104.0850
    -0.1650 (-0.1583%)
     
  • BTC-USD

    17,717.93
    +532.58 (+3.10%)
     
  • CMC Crypto 200

    333.27
    -4.23 (-1.25%)
     
  • FTSE 100

    6,367.58
    +4.65 (+0.07%)
     
  • Nikkei 225

    26,644.71
    +107.40 (+0.40%)
     

Ionis' Alexander Disease Candidate Gets Orphan Drug Status

Zacks Equity Research
·2 min read

Ionis Pharmaceuticals, Inc. IONS announced that the FDA has granted an orphan drug designation to its investigational pipeline candidate ION373, which is being developed for the treatment of people with Alexander disease, a rare and severe neurodegenerative disease.

The FDA’s orphan drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. This designation also makes the company entitled to certain other benefits including tax credits related to clinical trial expenses and an exemption from administrative fees. Upon potential approval, the status will also make ION373 eligible for seven years of marketing exclusivity in the United States.

Notably, in February 2020, the European Medicines Agency also granted an orphan drug tag to ION373 for treating Alexander disease, which is said to affect around 500 people in the EU.

Per the press release, the debilitating Alexander disease is estimated to occur in about one of one million births. Developed by Ionis, ION373 is an investigational antisense medicine, designed to stop the mutated gene from producing excess glial fibrillary acidic protein (GFAP), which causes the disease.

Meanwhile, Ionis is developing several investigational medicines to address various neurological diseases. Apart from ION373, the company is developing ION716 for treating Prion disease, ION283 for treating Lafora disease and ION363 for amyotrophic lateral sclerosis (ALS).

We note that Ionis also has many other pipeline candidates in phase II development, which it is developing in partnership with Biogen BIIB, Akcea AKCA, Roche RHHBY or on its own. The company now focuses on its wholly-owned pipeline to boost growth. It plans to conduct six pivotal studies by this year-end. It also intends to file 10 or more new drug applications through the end of 2025, possibly leading to multiple new medicines.

These Stocks Are Poised to Soar Past the Pandemic

The COVID-19 outbreak has shifted consumer behavior dramatically, and a handful of high-tech companies have stepped up to keep America running. Right now, investors in these companies have a shot at serious profits. For example, Zoom jumped 108.5% in less than 4 months while most other stocks were sinking.

Our research shows that 5 cutting-edge stocks could skyrocket from the exponential increase in demand for “stay at home” technologies. This could be one of the biggest buying opportunities of this decade, especially for those who get in early.

See the 5 high-tech stocks now>>


Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
 
Biogen Inc. (BIIB) : Free Stock Analysis Report
 
Roche Holding AG (RHHBY) : Free Stock Analysis Report
 
Ionis Pharmaceuticals, Inc. (IONS) : Free Stock Analysis Report
 
Akcea Therapeutics, Inc. (AKCA) : Free Stock Analysis Report
 
To read this article on Zacks.com click here.
 
Zacks Investment Research