Stoke Therapeutics, Inc. STOK announced that the FDA has granted a Breakthrough Therapy designation to its investigational candidate, zorevunersen (STK-001), for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy.
Shares of the company were up 12.1% on Dec. 4 following the announcement of the news.
The FDA grants breakthrough therapy designation to expedite the development and review of therapies for severe or life-threatening diseases where preliminary clinical evidence shows that the therapy may provide substantial improvements over available treatments.
Zorevunersen is a novel proprietary antisense oligonucleotide with the potential to become the first disease-modifying therapy to address the genetic cause of Dravet syndrome.
Year to date, shares of Stoke Therapeutics have rallied 143.5% against the industry’s decline of 8.3%.
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More on STOK's Development Plans for Zorevunersen
Zorevunersen is currently being evaluated in phase I/IIa and open-label extension (OLE) studies for the treatment of Dravet syndrome with a confirmed mutation, not associated with gain-of-function, in the SCN1A gene.
Data from the studies have shown that treatment with zorevunersen led to substantial and sustained reductions in seizure frequency and continuous improvements in multiple measures of cognition and behavior.
Per management, these effects were observed on top of the best anti-seizure medicines, the current standard of care.
Treatment with zorevunersen was generally well tolerated across the studies.
Stoke Therapeutics is currently discussing the path for a global phase III registrational study of zorevunersen in Dravet syndrome with the FDA and other regulatory agencies worldwide.
An update on the phase III registrational study on zorevunersen is expected by 2024-end.
STOK's Zacks Rank & Other Key Picks
Stoke Therapeutics currently carries a Zacks Rank #2 (Buy).
Some other top-ranked stocks from the biotech sector are Immunocore Holdings plc IMCR, Spero Therapeutics, Inc. SPRO and Castle Biosciences, Inc. CSTL, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Immunocore’s 2024 loss per share have narrowed from $1.79 to 94 cents. Loss per share estimates for 2025 have narrowed from $2.35 to $1.57 during the same time. Year to date, shares of IMCR have declined 53.6%.
IMCR’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 25.57%.
In the past 60 days, estimates for Spero Therapeutics’ 2024 loss per share have narrowed from $1.59 to $1.29. Loss per share estimates for 2025 have narrowed from $1.54 to 79 cents during the same time. Year to date, shares of SPRO have declined 19.7%.
SPRO’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 94.42%.
In the past 60 days, estimates for Castle Biosciences’ 2024 loss per share have narrowed from 58 cents to 8 cents. Loss per share estimates for 2025 have narrowed from $2.13 to $1.88 during the same time. Year to date, shares of CSTL have surged 47.6%.
CSTL’s earnings beat estimates in each of the trailing four quarters, the average surprise being 172.72%.
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Spero Therapeutics, Inc. (SPRO) : Free Stock Analysis Report
Stoke Therapeutics, Inc. (STOK) : Free Stock Analysis Report
Castle Biosciences, Inc. (CSTL) : Free Stock Analysis Report
Immunocore Holdings PLC Sponsored ADR (IMCR) : Free Stock Analysis Report
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