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Sarepta Navigates Confirmatory Study Funk to Expand DMD Gene Therapy Sarepta Navigates Confirmatory Study Funk to Expand DMD Gene Therapy

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Sarepta SRPT, the intrepid player in gene therapy, has tossed a game changer by submitting a banging efficacy supplement to the biologics license application (BLA) with the FDA. This bold move aims to push the envelope on Elevidys, the first-ever FDA approved gene therapy for Duchenne muscular dystrophy (DMD).

Let’s dive into the wild west of biotech. Sarepta is hanging its hat on seeking full approval for Elevidys to treat all DMD patients, regardless of age and ambulation status. The company is also throwing down a gauntlet by requesting the FDA to grant priority review to the submission.

Previously the FDA had granted accelerated approval to Elevidys in June and boy, oh boy, the plot thickens. This accelerated approval was for treating ambulatory pediatric patients aged between four and five years with DMD. This news definitely did not send the stock price to the moon. Year to date, Sarepta’s shares have lost 13.7% compared with the industry’s 10.2% decline. Ouch!

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The BLA supplement is being hyped up by data from the phase III EMBARK study, announced in October 2023, which teases a twist as it serves as the confirmatory study for Elevidys’ full approval in the DMD indication. This showstopper boasts of achieving statistical significance on all pre-specified key secondary outcomes, boldly indicating that treatment with Elevidys modifies the course of DMD indication.

Thought bubble! Management claims to have chewed the fat with the FDA, who had previously indicated that they are open to reviewing the data for label expansion based on the totality of evidence from the EMBARK study. What a rollercoaster!

But wait, there’s more! Sarepta has developed Elevidys in cahoots with Roche RHHBY. These partners in crime entered into a licensing agreement in 2019 to develop and commercialize Elevidys jointly. This gene therapy criminal duo has put their sights on exclusive rights to launch and commercialize the gene therapy in ex-U.S. markets. These international waters can be treacherous!

To appease the gods of regulatory approval, Sarepta is also pulling a fast one by evaluating the safety and efficacy of Elevidys in the ongoing phase III ENVISION study in non-ambulatory and ambulatory DMD patients. Phew, that’s a lot on their plate!

Sarepta Therapeutics, Inc. Price

 

Sarepta Therapeutics, Inc. Price

Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote

 

Zacks Rank & Stocks to Consider

Well, well, look who’s here! Sarepta currently carries a Zacks Rank #3 (Hold). If you’re betting on the ponies, a couple of better-ranked stocks in the overall healthcare sector include Galapagos GLPG and Novo Nordisk NVO. While Galapagos sports a Zacks Rank #1 (Strong Buy) at present, Novo Nordisk carries a Zacks Rank #2 (Buy). Get your bets in!

In the past 60 days, Galapagos’ estimates for 2023 have improved from a loss of $1.96 per share to 79 cents. During the same period, loss estimates per share for 2024 have narrowed from $3.22 to $1.68. Galapagos’ shares have lost 10.1% in the year-to-date period. Talk about a rollercoaster!

Galapagos’ earnings beat estimates in three of the last four quarters while missing the estimates on one occasion. On average, the company witnessed an average surprise of 91.97%. In the last reported quarter, Galapagos’ earnings beat estimates by 140.78%. That’s quite the showstopper!

In the past 60 days, estimates for Novo Nordisk’s 2023 earnings per share have increased from $2.57 to $2.62. During the same period, the earnings estimates for 2024 have risen from $2.99 to $3.14. Shares of NVO have surged 51.8% in the year-to-date period. Hold onto your hats!

Novo Nordisk’s earnings beat estimates in two of the last four quarters while meeting the mark on one occasion and missing the estimates on another. On average, the company witnessed an average surprise of 0.58%. In the last reported quarter, Novo Nordisk’s earnings beat estimates by 5.80%. What a wild ride!

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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