XM은(는) 미국 국적의 시민에게 서비스를 제공하지 않습니다.

Sarepta surges as investors cheer expanded use of gene therapy



<html xmlns="http://www.w3.org/1999/xhtml"><head><title>UPDATE 2-Sarepta surges as investors cheer expanded use of gene therapy</title></head><body>

Adds background on FDA decision in paragraph 4, CEO quote in paragraphs 8 & 9, analyst quote in paragraph 12 & 13

By Mariam Sunny and Bhanvi Satija

June 21 (Reuters) -Sarepta Therapeutics SRPT.O shares surged as much as 40% to a more than three-year high on Friday as an expanded use approval cemented its dominant position in the gene therapy market for Duchenne muscular dystrophy (DMD).

At current levels of $163.88, stock was set to add nearly $4 billion to the company's market value if gains hold.

On Thursday, the U.S. FDA granted traditional approval for the therapy, Elevidys, in patients four years and above who can walk, as well as an accelerated approval for those who cannot.

The health regulator's expanded approval was based on the therapy meeting its secondary goals of improvement in factors like time taken by patients to rise from the floor and a 10-meter walk or run, even though it had failed to meet its primary goal of improving motor functions as assessed by a scoring system.

The FDA decision was the best case scenario predicted by analysts, with BMO Capital Market's Kostas Biliouris estimating that Elevidys could be given to about 13,000 patients, or nearly 90% of U.S. patients.

Given the high demand and acceptable safety, analysts flagged manufacturing concerns and insurance coverage for newly added patients as limiting factors.

The company is working on a new so-called suspension process for commercial production of Elevidys, which could be more beneficial as the therapy moves to a broader outside U.S. population, CEO Doug Ingram said in a conference call with analysts and investors.

"We've already done engineering runs up to 2,000 liters... and it will give us not only more materials down the road years from now, but also significantly reduce the cost of goods," Ingram added.

The company uses Catalent CTLT.N and Thermo Fisher Scientific TMO.N as contract manufacturers for its gene therapy.

Elevidys is among the most expensive treatments in the world with a list price of $3.2 million, and Ingram said on Friday the company does not intend to modify the price.

Sales of the therapy came in at $200.4 million in 2023. BMO's Biliouris said those sales showed that Sarepta had "done an amazing job".

"Commercially, they have been perhaps more successful than any other gene therapy, with the only exception being Zolgensma from Novartis," Biliouris said in an interview ahead of the approval.

Biliouris now expects Elevidys to be the "dominant" DMD gene therapy with no near-term competition until later than 2027.

DMD is an inherited progressive muscle-wasting disorder that affects an estimated one-in-3,500 male births worldwide, according to the U.S.-based National Organization for Rare Disorders.

The decision also follows concerns around an "uphill battle" to secure traditional approval after the therapy failed to meet the main goal in a key confirmatory late-stage trial.

The global treatment market for DMD - including gene therapies and other drugs - is expected to grow to $11.47 billion by 2034, according to ResearchandMarkets.com.

RegenxBio RGNX.O is testing a rival gene therapy in early- to mid-stage, while Pfizer's PFE.N treatment recently failed in a late-stage study.


Sarepta stock performance https://reut.rs/3Vtofsa


Reporting by Mariam Sunny, Bhanvi Satija and Leroy Leo in Bengaluru; Editing by Sriraj Kalluvila and Shailesh Kuber

</body></html>

면책조항: XM Group 회사는 체결 전용 서비스와 온라인 거래 플랫폼에 대한 접근을 제공하여, 개인이 웹사이트에서 또는 웹사이트를 통해 이용 가능한 콘텐츠를 보거나 사용할 수 있도록 허용합니다. 이에 대해 변경하거나 확장할 의도는 없습니다. 이러한 접근 및 사용에는 다음 사항이 항상 적용됩니다: (i) 이용 약관, (ii) 위험 경고, (iii) 완전 면책조항. 따라서, 이러한 콘텐츠는 일반적인 정보에 불과합니다. 특히, 온라인 거래 플랫폼의 콘텐츠는 금융 시장에서의 거래에 대한 권유나 제안이 아닙니다. 금융 시장에서의 거래는 자본에 상당한 위험을 수반합니다.

온라인 거래 플랫폼에 공개된 모든 자료는 교육/정보 목적으로만 제공되며, 금융, 투자세 또는 거래 조언 및 권고, 거래 가격 기록, 금융 상품 또는 원치 않는 금융 프로모션의 거래 제안 또는 권유를 포함하지 않으며, 포함해서도 안됩니다.

이 웹사이트에 포함된 모든 의견, 뉴스, 리서치, 분석, 가격, 기타 정보 또는 제3자 사이트에 대한 링크와 같이 XM이 준비하는 콘텐츠 뿐만 아니라, 제3자 콘텐츠는 일반 시장 논평으로서 "현재" 기준으로 제공되며, 투자 조언으로 여겨지지 않습니다. 모든 콘텐츠가 투자 리서치로 해석되는 경우, 투자 리서치의 독립성을 촉진하기 위해 고안된 법적 요건에 따라 콘텐츠가 의도되지 않았으며, 준비되지 않았다는 점을 인지하고 동의해야 합니다. 따라서, 관련 법률 및 규정에 따른 마케팅 커뮤니케이션이라고 간주됩니다. 여기에서 접근할 수 있는 앞서 언급한 정보에 대한 비독립 투자 리서치 및 위험 경고 알림을 읽고, 이해하시기 바랍니다.

리스크 경고: 고객님의 자본이 위험에 노출 될 수 있습니다. 레버리지 상품은 모든 분들에게 적합하지 않을수 있습니다. 당사의 리스크 공시를 참고하시기 바랍니다.