We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies, revised Privacy Policy and Terms of Service.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Solid Biosciences (SLDB) Announces DMD Study Data, Stock Down
Read MoreHide Full Article
Solid Biosciences Inc. (SLDB - Free Report) announced interim data from its ongoing phase I/II study — IGNITE DMD — evaluating its lead gene therapy candidate, SGT-001, in patients with Duchenne muscular dystrophy (DMD). Data from the study provided evidence that treatment with the candidate can have potential benefit in patients.
The study is evaluating two doses of the candidate — 5E13 vg/kg (low dose) and 2E14 vg/kg (high dose) — in patients aged five to 14 years, at baseline. Data from six patients showed that treatment with the low dose achieved a mean improvement of 1.0 point from baseline while the high dose achieved a mean improvement of 0.3 point in North Star Ambulatory Assessment (NSAA) scores, following one year of treatment. Patients in the untreated control cohort showed a mean decline of 4 points in NSAA score from baseline in the same period.
Moreover, data showed a mean increase in improvement of 37 meters and 49.7 meters in the 6-Minute Walk Test (6MWT) distance for the low dose and high dose cohorts, respectively, after one year of treatment. Improvements for both doses were above the generally accepted minimally clinically important difference of 30 meters. Patients in the low-dose and high-dose cohorts exhibited mean improvements of 3.9% and 16.7%, respectively, from baseline to one year during pulmonary function tests. Meanwhile, creatine kinase, whose elevated levels may suggest muscular dystrophy, declined approximately 50% in patients receiving the high dose while it increased approximately 166% in the low-dose group, after one year of treatment.
Meanwhile, we note that two other companies — Sarepta Therapeutics (SRPT - Free Report) and Pfizer (PFE - Free Report) — are developing gene therapies for treating DMD that have made encouraging progress with their respective candidates. While Sarepta is evaluating 2E14 vg/kg of its candidate, SRP-9001, Pfizer is evaluating two doses (1E14 vg/kg and 3E14 vg/kg) of its candidate, PF-06939926. Solid Biosciences’ high dose may be comparable to the doses of these two competing companies.
Data provided by Sarepta and Pfizer have shown that treatment with SRP-9001 and PF-06939926 led to mean improvements of 1.7 and 3.5 points, respectively, in NSAA scores. Both candidates beat the improvements achieved by either dose of SGT-001. Please note that direct comparison of different study results is difficult due to different study designs and patient selection procedures.
Moreover, the IGNITE DMD study data showed a lower mean improvement in NSAA scores for the higher dose versus the lower dose. However, creatine kinase improved for the high dose but deteriorated in the low-dose cohort.
These divergences in IGNITE DMD study data along with seemingly lower improvements in NSAA scores compared to competing companies, presumably, led to a 10.6% decline in shares of Solid Biosciences in pre-market trading on Mar 16. However, the company’s shares have gained 25.5% so far this year compared with the industry’s growth of 3.4%.
Meanwhile, 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. Wave Life Sciences (WVE - Free Report) is another company developing gene therapy for DMD.
In addition to the stocks discussed above, would you like to know about our 10 best buy-and-hold tickers for the entirety of 2021?
Last year's 2020Zacks Top 10 Stocks portfolio returned gains as high as +386.8%. Now a brand-new portfolio has been handpicked from over 4,000 companies covered by the Zacks Rank. Don’t miss your chance to get in on these long-term buys.
Image: Bigstock
Solid Biosciences (SLDB) Announces DMD Study Data, Stock Down
Solid Biosciences Inc. (SLDB - Free Report) announced interim data from its ongoing phase I/II study — IGNITE DMD — evaluating its lead gene therapy candidate, SGT-001, in patients with Duchenne muscular dystrophy (DMD). Data from the study provided evidence that treatment with the candidate can have potential benefit in patients.
The study is evaluating two doses of the candidate — 5E13 vg/kg (low dose) and 2E14 vg/kg (high dose) — in patients aged five to 14 years, at baseline. Data from six patients showed that treatment with the low dose achieved a mean improvement of 1.0 point from baseline while the high dose achieved a mean improvement of 0.3 point in North Star Ambulatory Assessment (NSAA) scores, following one year of treatment. Patients in the untreated control cohort showed a mean decline of 4 points in NSAA score from baseline in the same period.
Moreover, data showed a mean increase in improvement of 37 meters and 49.7 meters in the 6-Minute Walk Test (6MWT) distance for the low dose and high dose cohorts, respectively, after one year of treatment. Improvements for both doses were above the generally accepted minimally clinically important difference of 30 meters. Patients in the low-dose and high-dose cohorts exhibited mean improvements of 3.9% and 16.7%, respectively, from baseline to one year during pulmonary function tests. Meanwhile, creatine kinase, whose elevated levels may suggest muscular dystrophy, declined approximately 50% in patients receiving the high dose while it increased approximately 166% in the low-dose group, after one year of treatment.
Meanwhile, we note that two other companies — Sarepta Therapeutics (SRPT - Free Report) and Pfizer (PFE - Free Report) — are developing gene therapies for treating DMD that have made encouraging progress with their respective candidates. While Sarepta is evaluating 2E14 vg/kg of its candidate, SRP-9001, Pfizer is evaluating two doses (1E14 vg/kg and 3E14 vg/kg) of its candidate, PF-06939926. Solid Biosciences’ high dose may be comparable to the doses of these two competing companies.
Data provided by Sarepta and Pfizer have shown that treatment with SRP-9001 and PF-06939926 led to mean improvements of 1.7 and 3.5 points, respectively, in NSAA scores. Both candidates beat the improvements achieved by either dose of SGT-001. Please note that direct comparison of different study results is difficult due to different study designs and patient selection procedures.
Moreover, the IGNITE DMD study data showed a lower mean improvement in NSAA scores for the higher dose versus the lower dose. However, creatine kinase improved for the high dose but deteriorated in the low-dose cohort.
These divergences in IGNITE DMD study data along with seemingly lower improvements in NSAA scores compared to competing companies, presumably, led to a 10.6% decline in shares of Solid Biosciences in pre-market trading on Mar 16. However, the company’s shares have gained 25.5% so far this year compared with the industry’s growth of 3.4%.
Meanwhile, 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. Wave Life Sciences (WVE - Free Report) is another company developing gene therapy for DMD.
Solid Biosciences Inc. Price
Solid Biosciences Inc. price | Solid Biosciences Inc. Quote
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.
Zacks Top 10 Stocks for 2021
In addition to the stocks discussed above, would you like to know about our 10 best buy-and-hold tickers for the entirety of 2021?
Last year's 2020Zacks Top 10 Stocks portfolio returned gains as high as +386.8%. Now a brand-new portfolio has been handpicked from over 4,000 companies covered by the Zacks Rank. Don’t miss your chance to get in on these long-term buys.
AccessZacks Top 10 Stocks for 2021 today >>