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Investing.com - PTC Therapeutics (NASDAQ:PTCT), a $4.1 billion market cap biotech company with an EXCELLENT financial health score according to InvestingPro, received a Complete Response Letter (CRL) from the FDA for vatiquinone in Friedreich’s ataxia (FA), the company announced Tuesday. The FDA stated that substantial evidence of efficacy was not demonstrated and that an additional adequate and well-controlled study would be needed to support NDA resubmission.
The regulatory setback aligns with previous FDA feedback from a Type C meeting in 2023, where the agency indicated a confirmatory study would likely be needed despite acknowledging the value of upright stability as a clinically meaningful endpoint. Similarly, the European Medicines Agency had previously indicated the MOVE-FA data would not be sufficient for conditional authorization. Despite these challenges, PTC (NASDAQ:PTC) maintains strong financials with a healthy current ratio of 3.62 and impressive revenue growth of 96% over the last twelve months.
PTC Therapeutics had hoped additional analyses of the MOVE-FA data would provide sufficient confirmatory evidence for the FDA, but these efforts proved inadequate. The company plans to meet with the FDA to discuss potential steps to address the issues raised in the CRL.
During its August 7 earnings call, PTC Therapeutics had disclosed that its late-cycle meeting with the FDA in July focused on evidence evaluation, noting that upright stability was not a pre-specified primary endpoint. The company maintained that upright stability is the most relevant measurement of efficacy in ambulatory, pediatric and adolescent FA patients.
Leerink Partners reiterated a Market Perform rating and $54.00 price target on PTC Therapeutics following the announcement. The firm noted that vatiquinone was not a large driver of value for PTC Therapeutics, contributing only approximately $1 to their valuation of the company. Trading at a P/E ratio of 6.4, InvestingPro analysis suggests the stock is currently fairly valued. Get access to 8 additional ProTips and comprehensive financial analysis through InvestingPro’s detailed research reports, available for over 1,400 US stocks.
In other recent news, PTC Therapeutics received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) concerning its New Drug Application for vatiquinone, a treatment aimed at Friedreich’s ataxia. The FDA indicated that PTC Therapeutics had not demonstrated substantial evidence of efficacy and requested an additional well-controlled study before reconsidering the application. Following this regulatory setback, Jefferies lowered its price target for PTC Therapeutics to $63 while maintaining a Buy rating. Similarly, BofA Securities adjusted its price target to $76, also retaining a Buy rating. Despite the FDA’s rejection, Truist Securities kept its Buy rating and $86 price target, viewing the CRL as a "clearing event" before upcoming product launches and regulatory updates. These developments reflect a cautious but optimistic outlook from analysts regarding the company’s future prospects amidst the current challenges.
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