On Thursday, Citi maintained its Sell rating on PTC Therapeutics (NASDAQ:PTCT) with a price target of $26.00. The firm acknowledged the recent announcement by PTC (NASDAQ:PTC) Therapeutics of its interim Phase 2 PIVOT-HD study results for PTC518. The study showed a dose-dependent reduction of the mutant huntingtin (mHTT) protein at 12 months in both the blood and cerebrospinal fluid at dosages of 5mg and 10mg.
The company's investigational drug, PTC518, indicated early signs of effectiveness, evidenced by positive trends in Total Motor Score (TMS) and Composite Unified Huntington’s Disease Rating Scale (cUHDRS). PTC Therapeutics also reported that PTC518 has been well-tolerated among participants, with no treatment-related spikes in neurofilament light chain (Nfl), a potential biomarker of neuronal damage.
In addition to the clinical trial updates, PTC Therapeutics announced that the FDA has lifted its partial clinical hold on the drug. This development potentially paves the way for the continuation of research and development activities without the previous restrictions.
Furthermore, PTC Therapeutics disclosed a financial transaction involving its retained royalty on Evrysdi, a drug for spinal muscular atrophy. The company has sold half of its Evrysdi royalty to Royalty Pharma for $242 million, reducing its royalty ownership from 19% to 9.5%. This sale provides PTC Therapeutics with an immediate influx of capital, which could be allocated to further its research and development efforts or other strategic initiatives.
In other recent news, PTC Therapeutics, Inc. has experienced a series of noteworthy developments. The U.S. Food and Drug Administration (FDA) has lifted a partial clinical hold on PTC518, an investigational drug for Huntington's disease, following promising interim results from a Phase 2 study. The FDA's decision will allow PTC Therapeutics to continue the drug's development.
Moreover, the company announced that the FDA has accepted its Biologics License Application for Upstaza, a gene therapy candidate for the treatment of aromatic L-amino acid decarboxylase deficiency, and granted it Priority Review.
Furthermore, the European Commission (EC) has decided not to endorse the Committee for Medicinal Products for Human Use's second negative opinion on Translarna, a drug developed by PTC Therapeutics. This decision has led to adjustments in the company's stock ratings and price targets by several firms.
Goldman Sachs maintained a sell rating but raised the price target from $18 to $32. Jefferies also increased its price target for the stock, from $35 to $46, maintaining a buy rating. Raymond James upgraded PTC Therapeutics from underperform to market perform.
These developments reflect the recent progress and challenges faced by PTC Therapeutics in its pursuit of advancing treatments for rare disorders. The company continues to engage with regulatory bodies and remains committed to its drug development programs.
InvestingPro Insights
As PTC Therapeutics (NASDAQ:PTCT) navigates the biopharmaceutical landscape, real-time data from InvestingPro helps us understand the company's financial footing. With a market capitalization of $2.55 billion, PTC Therapeutics shows robust revenue growth of 20.39% over the last twelve months as of Q1 2024, despite a quarterly decline of 4.66% in Q1 2024.
This suggests a dynamic revenue trajectory that potential investors should consider. Moreover, the company's gross profit margin stands at 29.53%, indicating its ability to retain a significant portion of its sales as profit.
From an investment standpoint, two InvestingPro Tips highlight critical aspects: Analysts predict a sales decline in the current year and do not foresee the company turning a profit this year. These insights, coupled with the fact that PTC Therapeutics has not been profitable over the last twelve months and does not pay a dividend, may influence investment decisions.
Still, the company has experienced a strong return over the last three months with a 20.7% price total return, and an even larger price uptick over the last six months at 31.73%, showcasing a positive short-term investment performance.
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