The mRNA expression results indicated that Harderian FXN may play a dynamic role in intracellular Fe of heme required for processing cytochromes and other hemeproteins, also suggesting that the moderate sexual dimorphism present in the HG and gonads could be regulated by androgens, while sexually dimorphic expression of FXN in the female pancreas may be controlled by sex steroids.
Thursday, March 23, 2023
Dimorphic frataxin and its gene regulation by sex steroids in hamsters
Ramos L. (2023). Dimorphic frataxin and its gene regulation by sex steroids in hamsters. Molecular genetics and genomics : MGG, 10.1007/s00438-023-02004-6. Advance online publication. doi:10.1007/s00438-023-02004-6
Skyclarys' Positive Reception By FDA And Medical Community Bolsters Reata's Future
Seeking Alpha. Mar. 23, 2023.
Reata Pharmaceuticals recently received FDA approval for Skyclarys, the first drug approved to treat Friedreich's ataxia (FA) in adults and adolescents aged 16 years and older.
Skyclarys is expected to experience significant utilization among FA patients, with a potential peak annual revenue between $800 million and $1 billion in the US alone.
According to the Friedreich's Ataxia Research Alliance, there are several potential competitors in the pipeline for FA treatment, including one Phase 3 candidate and a few in Phase 2. At first glance, the competitive risk to Reata's Skyclarys seems relatively low, but it's essential to remain cautious as the landscape can change.
PTC Therapeutics' Phase 3 candidate, vatiquinone, failed to meet its Phase 2 study endpoints related to FA. Phase 3 data is expected in Q2 2023. Leriglitazone did not meet endpoints in a Phase 2 proof-of-concept study. The HIV antiviral drug, etravirine, demonstrated increased frataxin levels in FA patient-derived cells but is awaiting Phase 2 clinical trial results. Most other drugs are in Phase 1, while gene therapy is in pre-clinical development.
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