Please try another search
Shares of Solid Biosciences Inc. (NASDAQ:SLDB) have gained 70.4% since Aug 14 as investors cheered the amendments made to a gene therapy study protocol evaluating its lead candidate, SGT-001 as a potential treatment for Duchenne muscular dystrophy (“DMD”). The company announced the update in its second-quarter earnings release.
The company is evaluating SGT-001, a novel adeno-associated viral (AAV) vector-mediated gene therapy, in a phase I/II study – IGNITE DMD – for treating DMD by increasing level of dystrophin protein in patients. Per the amended protocol, the company has limited new patient selection up to a weight of 25kg. The company has also removed the matched patient control arm in the second cohort of the study. These changes are expected to expedite the data readout from the IGNITE DMD study. Faster progress of SGT-001 is necessary for growth of Solid Biosciences as several companies are developing gene therapies for treating DMD with Sarepta Therapeutics (NASDAQ:SRPT) leading the space.
The company also stated that it has initiated dosing of the second patient in the higher dose (2E14 vg/kg) cohort in the IGNITE DMD study. It anticipates to announce updated data from the study later this year.
Meanwhile, Solid Biosciences’ shares have declined 67.1% so far this year compared with the industry’s decrease of 2.5%.
The development of SGT-001 for DMD has faced multiple hurdles so far this year. In February, the company announced preliminary data from IGNITE DMD study, which showed that lower dose (5E13 vg/kg) of SGT-001 was unable to achieve expected level of dystrophin protein in patients. Based on this result, the company expedited dose escalation activities to evaluate the higher dose in patients. In March 2018, one patient receiving the lower dose of SGT-001 was hospitalized following an adverse event, which led the FDA to put the study on clinical hold. Investors remain skeptical of the dose escalation as higher dose of the candidate can result in more severe adverse events. However, the company has not reported any such adverse event from the higher as well the lower dose cohort since June 2018. The clinical hold on the study was removed in June 2018.
Meanwhile, Solid Biosciences also announced in its earnings release that it has raised $60 million from a private placement. The company expects its cash resources as of Jun 30, 2019 along with the raised fund to be able to support the company’s operations into the fourth quarter of 2020 from the previously expected timeline of first quarter of 2020.
The company is developing SGT-001 in several early-stage studies for indications other than DMD. Please note that the gene therapy space is getting crowded with several pharma/biotech companies developing their pipeline, targeting multiple indications. Apart from Solid Biosciences and Sarepta, companies developing gene therapy for DMD include Pfizer (NYSE:PFE) , Audentes Therapeutics (NASDAQ:BOLD) , Wave Life Sciences and others.
Zacks Rank
Solid Biosciences currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
The Hottest Tech Mega-Trend of All
Last year, it generated $8 billion in global revenues. By 2020, it's predicted to blast through the roof to $47 billion. Famed investor Mark Cuban says it will produce "the world's first trillionaires," but that should still leave plenty of money for regular investors who make the right trades early.
See Zacks' 3 Best Stocks to Play This Trend >>
If you had been following the S&P 500 closely this past week, it likely would have left you scratching your head if you were trying to align the news with the market action. For...
The Russell 2000 (IWM) has been defending its 50-day MA over the early part of 2024, but the last few days have seen a shift in this support with 'sell' triggers in the MACD and...
Consumer instinct is a wonderful attribute to have and is generally talked about when considering stocks to buy.What Is the “Consumer Instinct”? “Peter Lynch is one of the most...
Are you sure you want to block %USER_NAME%?
By doing so, you and %USER_NAME% will not be able to see any of each other's Investing.com's posts.
%USER_NAME% was successfully added to your Block List
Since you’ve just unblocked this person, you must wait 48 hours before renewing the block.
I feel that this comment is:
Thank You!
Your report has been sent to our moderators for review
Add a Comment
We encourage you to use comments to engage with other users, share your perspective and ask questions of authors and each other. However, in order to maintain the high level of discourse we’ve all come to value and expect, please keep the following criteria in mind:
Enrich the conversation, don’t trash it.
Stay focused and on track. Only post material that’s relevant to the topic being discussed.
Be respectful. Even negative opinions can be framed positively and diplomatically. Avoid profanity, slander or personal attacks directed at an author or another user. Racism, sexism and other forms of discrimination will not be tolerated.
Perpetrators of spam or abuse will be deleted from the site and prohibited from future registration at Investing.com’s discretion.