Tuesday, July 16, 2024

Therapy Setbacks and Advances: AstraZeneca’s Truqap, Sanofi’s New Partnership, and More

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AstraZeneca faced a setback with its new breast cancer therapy, Truqap. The Capitello-290 trial, which evaluated Truqap in combination with chemotherapy for treating “triple-negative” breast cancer, did not show an improvement in overall survival compared to a placebo plus chemotherapy. Currently, Truqap is approved for hormone-receptor-positive, HER2-negative breast cancer with specific genetic alterations and achieved $50 million in sales in the first quarter of 2024. AstraZeneca remains hopeful, with upcoming data expected from a trial involving prostate cancer later this year.

In other news, Sanofi has entered into a significant partnership with Belharra Therapeutics, a biotechnology startup, to develop small-molecule drugs for inflammatory diseases. This collaboration will see Belharra receive $40 million in upfront and near-term payments to kickstart the deal. Belharra will utilize its “next-generation chemoproteomics” technology to identify potential immune drug candidates. This alliance marks Belharra’s second major partnership since its launch last year, which was initiated with $130 million in funding and a research agreement with Genentech.

Meanwhile, Taysha Gene Therapies announced promising results from a Phase 1/2 trial of their gene therapy, TSHA-102, for treating Rett syndrome, a rare neurodevelopmental disorder. The trial included two adult and two pediatric patients, with one adult achieving the milestone of sitting up unassisted for the first time in over a decade. Other participants also showed symptomatic improvements. Taysha plans to proceed with testing higher doses of TSHA-102 in future trials.

FDA Backs Regenxbio’s Gene Therapy for Hunter Syndrome Amid Biotech Advances

Regenxbio received a favorable response from the Food and Drug Administration (FDA) regarding its accelerated approval application for a gene therapy aimed at treating Hunter syndrome, a neurodegenerative disease. The FDA supports Regenxbio’s plan to use a neurological protein as a surrogate marker likely to predict clinical benefit and has discussed the design of a confirmatory trial scheduled to commence next year. Regenxbio plans to start submitting its application in the third quarter of this year. If approved, RGX-121 would be the first wholly-owned treatment by Regenxbio to reach the market.

These developments highlight the dynamic nature of the biotech and pharmaceutical industries, with significant advancements and challenges emerging regularly. Sanofi’s partnership with Belharra underscores the ongoing interest and investment in innovative drug discovery platforms, particularly in the realm of inflammatory diseases. Belharra’s use of advanced chemoproteomics is expected to enhance the identification and development of new therapeutic candidates, potentially accelerating the drug discovery process.

AstraZeneca’s experience with Truqap illustrates the complexities and uncertainties inherent in cancer drug development. Despite the disappointing results in the Capitello-290 trial, the company’s continued efforts to explore Truqap’s efficacy in other cancer types demonstrate its commitment to expanding the drug’s therapeutic potential. The forthcoming data from the prostate cancer trial will be crucial in determining the future of Truqap.


Promising Gene Therapies for Rett Syndrome and Hunter Syndrome Highlight Biotech Advances

The promising preliminary results from Taysha Gene Therapies’ TSHA-102 for Rett syndrome bring hope to patients with this debilitating condition. The observed improvements in motor function among trial participants suggest that gene therapy could offer a viable treatment option for Rett syndrome, pending further validation in larger and more comprehensive trials.

Regenxbio’s progress with its gene therapy for Hunter syndrome reflects the FDA’s willingness to support innovative treatment approaches through accelerated approval pathways. By endorsing the use of surrogate markers and collaborating on trial design, the FDA is facilitating the development and potential approval of therapies that address significant unmet medical needs. RGX-121, if approved, could provide a much-needed treatment option for patients with Hunter syndrome and represent a milestone for Regenxbio.

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In summary, the recent updates from Sanofi, AstraZeneca, Taysha Gene Therapies, and Regenxbio highlight the ongoing advancements and challenges within the pharmaceutical and biotechnology sectors. These developments not only showcase the potential of innovative technologies and therapies but also underscore the importance of rigorous clinical testing and regulatory support in bringing new treatments to market. As these companies continue to push the boundaries of medical science, their efforts promise to deliver new hope and improved outcomes for patients worldwide.


Resource: Global News Wire, June 18, 2024

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