After BioMarin (BMRN) and PTC Therapeutics (PTCT) hosted a symposium on "Advances in Duchenne Muscular Dystrophy Natural History and Biomarkers," Oppenheimer says that the seminar helped lower the risk posed by the results of a study of PTC's Translarna drug. The firm views the recent weakness in PTC as a buying opportunity, and recommends owning the stock ahead of the release of data on Translarna in October. It keeps a $155 price target and Outperform rating on PTC.

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Canaccord analyst Whitney Ijem lowered the firm's price target on BioMarin to $89 from $91 and keeps a Hold rating on the shares. The firm noted investor focus remains on the bigger announcements/greater clarity that is expected at the company's September investor day rahter than the day's headline surrounding Roctavian struggles and the company's trimmed pipeline expenses.

Reports Q1 revenue $210M, consensus $169.7M. "We are off to a strong start in 2024, with outstanding commercial performance and achievement of all planned clinical and regulatory milestones for the first quarter," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "We remain on track to achieve the many planned 2024 clinical and regulatory milestones, including global regulatory submissions for sepiapterin."

Scotiabank raised the firm's price target on BioMarin to $85 from $83 and keeps a Sector Perform rating on the shares following model adjustments. While the company's enzyme business remains stable, in-line Voxzogo results and a struggling Roctavian franchise has led the firm to maintain its rating on the stock.

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Consensus for FY24 EPS is $1.65. Still sees FY24 revenue $2.7B-$2.8B, consensus $2.77B.

Reports Q1 revenue $649M, consensus $651.79M. "During the quarter, execution across our business led to double digit revenue growth, on a constant currency basis, and an 18% increase in non-GAAP diluted EPS. At the same time, we made rapid progress on advancing our strategic priorities for the year, including accelerating and maximizing the VOXZOGO opportunity, focusing R&D on the most productive assets, and increasing profitability," said CEO Alexander Hardy. "We were pleased with the outcome of our strategic R&D asset review, resulting in the acceleration and prioritization of the most potentially impactful medicines for patients...Quarterly results were driven by strong demand for VOXZOGO, the only approved treatment for children with achondroplasia, and solid contributions from our established enzyme products...Our registration-enabling plans with VOXZOGO in hypochondroplasia, and ongoing discussions with health authorities to align on development plans for idiopathic short stature and pathway conditions are on-track, with all three studies expected to begin enrollment this year."