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Investing.com -- Sarepta Therapeutics Inc. (NASDAQ:SRPT) shares plunged 21.1% in after-hours trading Monday after the company announced its Phase 3 ESSENCE study for ultra-rare disease therapies AMONDYS 45 and VYONDYS 53 failed to achieve statistical significance on its primary endpoint.
The company reported third quarter adjusted earnings of -$0.13 per share, beating analyst estimates of -$0.70, while revenue reached $399.4 million, surpassing the consensus estimate of $337.91 million. Despite the financial outperformance, investors focused on the disappointing clinical trial results.
While the ESSENCE study showed numerical trends favoring treatment versus placebo, the observed difference of 0.05 steps/second did not reach statistical significance (P=0.309) on the primary endpoint measuring 4-step ascend velocity at 96 weeks. The company attributed the underperformance partly to COVID-19 pandemic disruptions during the nine-year study period.
"While the ESSENCE study did not meet statistical significance on its primary endpoint, we believe the results demonstrated a clear treatment effect, showing clinically meaningful functional outcomes for people with Duchenne," said Louise Rodino-Klapac, president of research & development at Sarepta .
The company noted that when excluding data from participants whose double-blind period overlapped with the COVID-19 pandemic, results showed a 30% reduction in disease progression, which Sarepta described as "clinically meaningful."
Third quarter net product revenues totaled $370 million, consisting of $238.5 million from PMO therapies and $131.5 million from ELEVIDYS. The company has strengthened its financial position through debt refinancing and cost restructuring initiatives.
CEO Doug Ingram said, "We look forward to discussing the ESSENCE results and the real-world evidence for AMONDYS 45 and VYONDYS 53 with the FDA." The company plans to meet with regulators to discuss the possibility of converting from accelerated to traditional approval based on the study results and real-world evidence.
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