PTC’s Phase III Data Sets Up PKU Competition With BioMarin

 
• By Joseph Haas
  
Generate a summary of this article
Listen to this article
Beta

Phase III data for PTC’s sepiapterin shows statistically significant reduction of blood phenylalanine levels in patients with phenylketonuria. BioMarin has the only two approved drugs for PKU.

Rare disease
PTC hopes to bring a competitor to BioMarin's PKU drugs to market • Source: Shutterstock

Rare disease-focused PTC Therapeutics, Inc. unveiled Phase III data for its phenylketonuria (PKU) candidate sepiapterin (PTC923) on 17 May that looks capable of positioning the oral synthetic drug to compete with BioMarin Pharmaceutical Inc.’s two approved PKU therapies. But, while PTC highlighted data from a subset of more severe PKU patients, at least one analyst questioned whether the sample size would be large enough to win sepiapterin a broad label indication.

In a 98-patient readout from the Phase III APHENITY trial, PTC reported a mean 63% reduction in phenylalanine (Phe) at...

Scrip Podcast

Keep up with the top pharma business news of the week. On the go.

Scrip brings you a weekly bulletin of top picks.

SpotifyApple PodcastsWebsite

More from Clinical Trials

Sciwind Eyes Differentiation For Ecnoglutide In Highly Competitive Arena

 
• By Xu Hu

Chinese firm Sciwind has revealed additional Phase III findings for its promising once-weekly GLP-1 agonist ecnoglutide, just one asset in a burgeoning Chinese sector that could see the emergence of new rivals to current global leaders in obesity and diabetes.

Fosun ALK/ROS1 Inhibitor Shows 1L Promise In Advanced NSCLC

 
• By Xu Hu

Interim results from the Phase III REMARK study presented at WCLC showed the Chinese company’s second-generation ALK/ROS1 inhibitor met its primary endpoint as a first-line therapy in ALK-positive advanced NSCLC.

Sponsored By:

Nancy Tests SPARC-931-part

The article discusses the impact of upcoming patent expirations in oncology biologics on biosimilar developers, highlighting the opportunities in Antibody-Drug Conjugates (ADCs) and Bispecific Antibodies (BsAbs). It explores the challenges posed by the structural complexities of these biologics, necessitating new bioanalytical tools and manufacturing processes. The evolving regulatory landscape, with contributions from EMA, FDA, and WHO, offers flexible pathways but also presents challenges. The article underscores the potential for biosimilars to generate significant cost savings and enhance patient access to cancer treatments over the next decade.

Fosun ALK/ROS1 Inhibitor Shows 1L Promise In Advanced NSCLC

Interim results from the Phase III REMARK study presented at WCLC showed the Chinese company’s second-generation ALK/ROS1 inhibitor met its primary endpoint as a first-line therapy in ALK-positive advanced NSCLC.

More from R&D

Scrip-R&D-Premium: ‘Article’ article that includes different Composer content

 
• By Nabil, Olga Zviezdicheva, Reed Miller, Mary Jo Laffler, Ian Schofield, João Andrés-Ruíz de la Fontaine-LeBlanc, Chloë Anastasía-Stojanović del Castillo-Ribeirão, and Nabil

To make your document look professionally produced, Word provides header, footer, cover page, and text box designs that complement each other. For example, you can add a matching cover page, header, and sidebar. Click Insert and then choose the elements you want from the different galleries.

Spotlight On RDCs, AOCs As Leading Novel Bioconjugates[Updated]

Pipelines in bioconjugates continue to develop, from ADCs to XDCs, with radionuclide drug conjugates (RDCs) and antibody-oligonucleotide conjugates (AOCs) leading the expansion.

Fosun ALK/ROS1 Inhibitor Shows 1L Promise In Advanced NSCLC

Interim results from the Phase III REMARK study presented at WCLC showed the Chinese company’s second-generation ALK/ROS1 inhibitor met its primary endpoint as a first-line therapy in ALK-positive advanced NSCLC.