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SRPT

Sarepta

$146.98

4.17 (2.92%)

06:50
10/04/18
10/04
06:50
10/04/18
06:50

Sarepta gene therapy showed functional improvements across all measures

Sarepta Therapeutics announced yesterday that at the 23rd International Congress of the World Muscle Society in Mendoza, Argentina, Jerry Mendell, M.D., of Nationwide Children's Hospital presented updated results from its gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne muscular dystrophy. Dr. Mendell presented the following updated data on the four patients enrolled in the study: All patients showed robust expression of transduced micro-dystrophin, which is properly localized to the muscle sarcolemma, as measured by immunohistochemistry. Mean gene expression for the study, as measured by percentage of micro-dystrophin positive fibers was 81.2% and the mean intensity of the fibers was 96.0% compared to normal control. All post-treatment biopsies showed robust levels of micro-dystrophin as measured by Western blot, with a mean of 74.3% compared to normal utilizing Sarepta's method, or 95.8% compared to normal pursuant to Nationwide Children's quantification of Sarepta's method that adjusts for fat and fibrotic tissue. Gene expression for the fourth patient was robust, as follows: As measured by immunohistochemistry, micro-dystrophin positive fibers was 96.2% and the mean intensity of the fibers was 160.0% compared to normal control. As measured by Western blot, patient 4 showed robust levels of micro-dystrophin, with a mean of 182.7% compared to normal utilizing Sarepta's method, or 222% compared to normal pursuant to Nationwide Children's quantification of Sarepta's method that adjusts for fat and fibrotic tissue. In all patients, expression of micro-dystrophin was associated with significant expression and up-regulation of the dystrophin-associated protein complex, an additional indication of functionality of dystrophin. All patients showed significant decreases of serum creatine kinase levels at last measure, with a mean reduction of CK of over 78% from baseline. Doug Ingram, Sarepta's president and CEO, said, "The encouraging results that we previously saw and reinforced in the fourth patient strengthen our resolve to rapidly move to a confirming trial and, assuming successful, to bring this therapy to the Duchenne community around the world with a sense of urgency."

SRPT Sarepta
$146.98

4.17 (2.92%)

10/03/18
JMPS
10/03/18
NO CHANGE
Target $270
JMPS
Outperform
JMP looking for continued dystrophin expression in Sarepta gene therapy update
JMP Securities analyst Liisa Bayko noted that Sarepta is scheduled to give an update on its Phase 1/2a study of a micro-dystrophin gene therapy at the World Muscle Society meeting and see hopes to see about 40% micro-dystrophin expression in the fourth patient and at least one vector copy per nucleus, which would be in line with the first three patients. She is also looking for details of the functional benefits that were previously acknowledged, said Bayko, who keeps an Outperform rating and $270 price target on Sarepta shares.
10/01/18
CANT
10/01/18
INITIATION
Target $217
CANT
Overweight
Sarepta initiated with an Overweight at Cantor Fitzgerald
Cantor Fitzgerald analyst Alethia Young started Sarepta Therapeutics with an Overweight rating and $217 price target.
09/26/18
RBCM
09/26/18
NO CHANGE
Target $200
RBCM
Outperform
Sarepta price target raised to $200 from $187 at RBC Capital
RBC Capital analyst Brian Abrahams raised his price target on Sarepta to $200 and kept his Outperform rating. The analyst says his discussions with the company's senior management have left him "even more impressed with their commercial and clinical execution, strong focus on maintaining competitive positioning in development and manufacturing, and commitment to cement their gene therapy leadership role". Abrahams also anticipates a "favorable supportive data" for the microdystrophin gene therapy program at next week's WMS event, with the pivotal plans for the program remaining on track toward FDA feedback later this year and dosing before year-end.
09/24/18
BTIG
09/24/18
NO CHANGE
Target $190
BTIG
Buy
Sarepta one step closer to gene therapy trial with FDA lifting hold, says BTIG
BTIG analyst Timothy Chiang said the FDA lifting its clinical hold on Sarepta's micro-dystrophin gene therapy program for the treatment of Duchenne Muscular Dystrophy moves the company one step closer to beginning its registrational study in 24 patients. The analyst, who expects additional biomarker data to be presented in early October on the four patients that have been dosed with its AAVrh74 micro-dystrophin gene therapy program, maintains a Buy rating and $190 price target on Sarepta shares.

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