==Sarepta Therapeutics (SRPT) : expanded FDA approval of ELEVIDYS to Duchenne Muscular Dystrophy patients

 

Sarepta Therapeutics announces expanded FDA approval of ELEVIDYS to Duchenne Muscular Dystrophy patients

• FDA grants traditional approval to ELEVIDYS for ambulatory Duchenne patients. FDA grants accelerated approval to ELEVIDYS for non-ambulatory Duchenne patients.
• Consistent with the accelerated approval pathway, Sarepta has committed to conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of ELEVIDYS in patients with Duchenne muscular dystrophy who are non-ambulatory. ENVISION (Study SRP-9001-303), a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne, is underway and intended to serve as this postmarketing requirement.

Unusual Options Activity Tue 9/26/23

Stocks seeing volatility buying (bullish call buying/bearish put buying):

Calls:

• ZIM Oct 12.50 calls are seeing interest with 50 contracts trading vs. open int of 3345, pushing implied vol up around 16 points to ~210.87%
  
• GME Dec 80.00 calls are seeing interest with 65 contracts trading vs. open int of 7260, pushing implied vol up around 29 points to ~142.78%. The Co will report its next earnings in December

Puts:

• RUM Oct 8.00 puts are active with 20 contracts trading vs. open int of 2970, pushing implied vol down around 51 points to ~148.61%
  
• SRPT Jan 75.00 puts are active with 190 contracts trading vs. open int of 555, pushing implied vol down around 6 points to ~124.22%. The Co will report its next earnings in late October/early November

Sentiment: The CBOE Put/Call ratio is currently: 1.02, VIX: (18.53, +1.63, +9.65%).
October 20 is options expiration -- the last day to trade October equity options.

Unusual Options Activity Tue 9/19/23

Stocks seeing volatility buying (bullish call buying/bearish put buying):

Calls:

• LCID Jan 85 calls are seeing interest with 170 contracts trading vs. open int of 9890, pushing implied vol up around 39 points to ~172.74%. The Co is expected to report its next earnings in November
  
• TVTX Oct 20 calls are seeing interest with 40 contracts trading vs. open int of 590, pushing implied vol up around 16 points to ~171.31%. The Co is expected to report its next earnings in late October/early November

Puts:

• ACRS Dec 7.5 puts are active with 260 contracts trading vs. open int of 1790, pushing implied vol down around 76 points to ~324.41%. The Co announced results from Phase 1 Multiple Ascending Dose Trial of ATI-2138, an Investigational Oral Covalent ITK/JAK3 Inhibitor yesterday
  
• SRPT Nov 75 puts are active with 130 contracts trading vs. open int of 1860, pushing implied vol down around 14 points to ~145.24%. The Co is expected to report its next earnings in late October/early November

Sentiment: The CBOE Put/Call ratio is currently: 0.91, VIX: (14.23, +0.23, +1.64%).
October 20 is options expiration -- the last day to trade October equity options.

-= Sarepta Therapeutics (SRPT) : Disappointing gene-therapy study results

 

Sarepta Therapeutics announces top-line results for Part 1 of Study 102; primary functional endpoint did not achieve statistical significance

Sarepta Therapeutics today announced top-line results from Part 1 of Study SRP-9001-102 (Study 102), an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy, and tolerability of a single dose of SRP-9001 in 41 patients with Duchenne muscular dystrophy.

• SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.

At 12 weeks post-treatment compared to baseline, the study met its primary biological endpoint of micro-dystrophin protein expression.

• Participants who received SRP-9001 had mean micro-dystrophin expression of 28.1%, as measured by western blot.
• Accompanying secondary biological endpoints including vector genome copies per nucleus, percent positive fibers, intensity, and reduction in creatine kinase (exploratory) were also met.

In the primary functional endpoint, SRP-9001-treated participants showed an increase in NSAA total score compared to placebo at 48 weeks; however, the difference was not statistically significant.

• At every time point measured, the cohort of SRP-9001 treated participants outperformed the placebo group, and, at 48 weeks, participants in the treatment group demonstrated a statistically significant increase of 1.7 points in NSAA total score compared to baseline, while participants in the placebo group saw an increase of 0.9 points on the NSAA total score compared to baseline, which was not statistically significant.

In the pre-specified analysis by age-group, by which the randomization was stratified, participants aged 4-5 years at time of treatment with SRP-9001 demonstrated a statistically significant improvement in NSAA total score versus the age-matched placebo cohort, achieving a 4.3-point improvement on NSAA at 48 weeks post-treatment from baseline.

The results from Study 102 reinforce the "favorable" safety and tolerability profile of SRP-9001, with no new safety signals identified.

-=Sarepta Therapeutics (SRPT) : positive results with Duchenne Muscular Dystrophy drug

Sarepta Therapeutics announces that at its first R&D Day, Jerry Mendell, M.D. presented positive preliminary results from the first three children dosed in the Phase 1/2a gene therapy micro-dystrophin trial to treat patients with Duchenne Muscular Dystrophy 

At the Company's R&D Day, Jerry Mendell, M.D. of Nationwide Children's Hospital presented positive preliminary results from its Phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne muscular dystrophy (DMD). Dr. Mendell presented the following preliminary data on the first three 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, as measured by percentage of micro-dystrophin positive fibers was 76.2% and the mean intensity of the fibers was 74.5% compared to normal control.
• All post-treatment biopsies showed robust levels of micro-dystrophin as measured by Western blot, with a mean of 38.2% compared to normal utilizing Sarepta's method, or 53.7% compared to normal pursuant to Nationwide Children's quantification of Sarepta's method that adjusts for fat and fibrotic tissue.
• A mean of 1.6 vector copies per cell nucleus was measured in patients, consistent with the high micro-dystrophin expression levels observed.
• All patients showed significant decreases of serum creatine kinase (CK) levels, with a mean reduction of CK of over 87% at Day 60. CK is an enzyme associated with muscle damage and patients with DMD uniformly exhibit high levels of CK. Indeed, significantly elevated CK is often used as a preliminary diagnosis tool for DMD, which is then followed by confirmatory genetic testing.
• No serious adverse events (SAEs) were observed in the study. Two patients had elevated gamma-glutamyl transferase (GGT) that resolved with increased steroids within a week and returned to baseline levels. There were no other significant laboratory findings. Patients had transient nausea generally during the first week of therapy coincident with increased steroid dosing.
• Peers: PTCT -6% and SLDB +16%.

-=Sarepta Therapeutics (SRPT) reported earnings on Thur 1 March 2018 (a/h)

CAMBRIDGE, Mass. (AP) _ Sarepta Therapeutics Inc. (SRPT) on Thursday reported a loss of $24 million in its fourth quarter.

On a per-share basis, the Cambridge, Massachusetts-based company said it had a loss of 37 cents.

Sales of Sarepta's Exondys 51, a drug to treat Duchenne muscular dystrophy, came in at $57.3 million, in line with the company's pre-announcement in January, RBC analyst Brian Abrahams said in a note to clients. Research and development expenses of $44.4 million were also in line, he said.

"The company ended the quarter with $1.1 billion cash/equivalents, which we estimate will be sufficient to sustain operations along with revenues for the foreseeable future," he said.

Sarepta also reiterated its 2018 guidance for $295 million to $305 million in Exondys 51 sales, which would represent about 100% year-over-year growth. Abrahams notes this supports a launch trajectory capable of reaching $1.2 billion in peak sales.

Sarepta also seeks to have Exondys 51 approved in Europe, though analysts were split on whether officials would approve the drug. Sarepta is slated to present its argument in late April, Piper Jaffray analyst Edward Tenthoff said in a note. A positive opinion would offer upside, he said.

But it wasn't Exondys 51 that had Sarepta stock popping Friday. Analysts are particularly excited about the potential for golodirsen, a drug being developed to treat about 8% of Duchenne muscular dystrophy patients. Sarepta recently met with the U.S.  Food and Drug Administration to discuss golodirsen.

Golodirsen is being tested in a pivotal trial dubbed Essence. That trial should finish enrolling in April and have data in about two years, Needham analyst Chad Messer said in a note. The trial could possibly serve as a conformational study for golodirsen.

Sarepta is also working on three investigational drugs including two gene therapies. By midyear, the firm plans to report preliminary data from both gene therapy studies, Leerink analyst Michael Schmidt said in his note to clients. These "may provide a glimpse into Sarepta's future."

=Sarepta Therapeutics (SRPT) : positive early/mid-stage clinical trial results in DMD

Sarepta Therapeutics announces positive results in its study evaluating gene expression, dystrophin production, and dystrophin localization in patients with duchenne muscular dystrophy (dmd) amenable to skipping exon 53 treated with Golodirsen (SRP-4053) -- Study achieved statistical significance on all primary and secondary biological endpoints

Co announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen in 25 boys with confirmed deletions of the DMD gene amenable to skipping exon 53. The study comprised two parts. In Part 1, 12 patients were randomized to receive a dose titration of golodirsen (8 patients) or placebo (4 patients). At the end of Part 1 (dose titration), all 12 patients continued on golodirsen and an additional 13 patients started golodirsen (Part 2). In Part 2, all 25 patients were treated for an additional 48 weeks at the time of muscle biopsy. The analysis included biopsies of the bicep muscle at baseline and on-treatment at the Part 2 Week 48 time point. All 25 participants displayed an increase in skipping exon 53 (p < 0.001) over baseline levels, representing a 100 percent response rate as measured by RT-PCR and demonstrating proof of mechanism. Mean dystrophin protein increased to 1.019 percent of normal compared to a mean baseline of 0.095 percent of normal (p < 0.001) as measured by Western blot, the primary biological endpoint in the study, representing a 10.7 fold increase from baseline. The study also showed a statistically significant increase in dystrophin immunofluorescence as measured by immunohistochemistry (IHC), the secondary biological endpoint in the study, confirming sarcolemma-associated protein expression and distribution.

"These data demonstrate statistically significant exon skipping, dystrophin production and localization, which further validate the broad application of our exon-skipping platform and aligns with our strategic imperative to expand and improve the treatment choices for the majority of patients with DMD," said Douglas Ingram, Sarepta Therapeutics' president and chief executive officer.

The full biological results from the 4053-101 study will be presented at an upcoming medical meeting or scientific conference. Golodirsen is one of the investigational candidates currently being evaluated in the ESSENCE study, a global, randomized double-blind, placebo-controlled study evaluating efficacy and safety in patients amenable to skipping exons 45 or 53.

=Sarepta Therapeutics (SRPT) reported earnings on Wed 19 July 2017 (a/h)

Sarepta Therapeutics (SRPT) stock bolted late Wednesday after the biotech smashed Wall Street views for second-quarter sales and reported narrowing losses just a day after it settled a patent dispute with BioMarin Pharmaceuticals (BMRN).

For the second quarter ended June 30, Sarepta reported adjusted losses of 46 cents a share on revenue of $35 million, compared with losses of $1.19 per share and no revenue in the year-ago quarter. Both metrics topped the consensus for a 92-cent loss and $22 million in sales.

The biotech also boosted its 2017 guidance to $125 million to $130 million after, earlier in the day, announcing a program to expand access to its only approved drug, Exondys 51, a treatment for patients with Duchenne muscular dystrophy.

Patients in North America, South America and Europe will benefit from the program which, Leerink analyst Joseph Schwartz said earlier Wednesday, could add meaningful revenue to Sarepta's top line in 2017. Schwartz has an outperform rating on Sarepta stock.