STOK stock is fading higher on December 31, 2019, after the company was recently added to the NASDAQ Biotechnology Index.

On December 23, 2019, Stoke Therapeutics, Inc. (Nasdaq:STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases by precisely upregulating protein expression, announced that it has been selected for addition to the NASDAQ Biotechnology Index (Nasdaq:NBI). This addition is effective prior to market open today.

The NASDAQ Biotechnology Index tracks the performance of a set of NASDAQ-listed securities that are classified as either biotechnology or pharmaceutical according to the Industry Classification Benchmark. Selected companies must meet eligibility requirements, including minimum market capitalization, average daily trading volume and seasoning as a public company, among other criteria. The NASDAQ Biotechnology Index is re-ranked annually and forms the basis for a number of Exchange Traded Funds (ETFs), including the iShares NASDAQ Biotechnology ETF.

On December 17, 2019, Wedbush analyst Laura Chico initiated coverage of Stoke Therapeutics with an Outperform rating and $31 price target.

On December 8, 2019, Stoke Therapeutics (Nasdaq: STOK) presented new preclinical data on STK-001, a potential new disease-modifying medicine for the treatment of Dravet syndrome. Data from studies in non-human primates (NHP) showed STK-001 distributed throughout the brain and achieved target engagement and increased Nav1.1 protein expression throughout the cortex after a single intrathecal injection. Safety findings showed STK-001 to be well-tolerated at the two intrathecal dose levels studied. These data were presented today in a poster session at the American Epilepsy Society (AES) Annual Meeting in Baltimore.

“The effects of Dravet syndrome go beyond seizures and often include cognitive regression or developmental stagnation, ataxia, speech impairment and sleep disturbances. The disease is believed to affect multiple areas of the brain, with the cerebral cortex playing a particularly important role,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “These new data are encouraging because they show the ability of STK-001 to broadly distribute in the brain and to elicit target engagement and increased Nav1.1 throughout the cortex. These results will be included in our planned IND submission and provide additional confidence in our clinical plans for STK-001.”

Dravet syndrome is a severe and progressive form of genetic epilepsy that affects approximately 35,000 people in the United States, Canada, Japan, Germany, France and the United Kingdom. Approximately 85% of Dravet syndrome cases are caused by spontaneous, heterozygous mutations in the SCN1A gene, resulting in 50% of normal Nav1.1 protein expression.

Stoke selected two dose levels of STK-001 for this non-GLP study in order to evaluate safety, brain biodistribution, target engagement and Nav1.1 protein expression. On day 1, treatment-naïve cynomolgus monkeys were administered a single, bolus intrathecal lumbar (IT-L) injection at one of two dose levels of STK-001. After dosing, the animals underwent standard clinical and neurological observation, and blood samples were collected. STK-001 concentration level, gene expression, and protein expression were assessed in the brain on day 3 and on day 29.

The following are highlights from today’s poster presentation:

  • Brain tissue exposure to STK-001 was observed on day 3 and day 29. In the high dose group, exposure of STK-001 was observed in all brain regions examined, except pons and thalamus. STK-001 levels in cortical brain regions were generally higher than in deeper structures and were also increased from day 3 to day 29.
  • Nav1.1 protein levels were observed to increase up to 3-fold in some regions of the cortex on day 29 in the high dose group. No or marginal changes in Nav1.1 protein levels were observed on day 29 in the low dose group, or on day 3 in either dose group.
  • Significant target engagement (SCN1A expression) was observed on day 29 throughout the cortex and the limbic lobe in the high dose group. No or marginal change in SCN1A levels in brain tissues were observed at the low dose of STK-001, or on day 3 in either dose group.
  • A favorable safety profile was demonstrated for STK-001 at both dose levels with no change in neurological or physical measures, even in animals that overexpressed Nav1.1 protein above wild type levels.
  • Stoke plans to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration in early 2020 and, subject to acceptance of the IND, plans to initiate a Phase 1/2 single-ascending dose study in children and adolescents with Dravet syndrome in the first half of 2020.
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