Top Tweets for #st920
The FDA accepts eGFR slope as a clinically relevant surrogate for Fabry Disease — the European EMA requires proof that it results in real events. For this reason, #ST920 approval will first be sought from the FDA and later, with mature data, from the EMA in Europe.
The European Medicines Agency (EMA) has given a positive opinion on #ST920 (Isaralgagene Civaparvovec), a gene therapy developed by $SGMO for the treatment of Fabry disease.
EMA has granted ST920 both orphan drug status and PRIME (PRIority MEdicines) designation. PRIME status is intended for therapies that address significant unmet medical needs and provides companies with early and enhanced scientific support to accelerate development and approval. In the ongoing Phase 1/2 study (STAAR), ST920 demonstrated a favorable safety profile. Most adverse events were mild to moderate; there were no serious adverse events that led to discontinuation of the study. In addition, a sustained increase in α-galactosidase-A enzyme activity was observed, indicating potential clinical efficacy.
Indirect evidence of potential pain relief after treatment with $SGMO s #ST920
Some results from the #STAAR study suggest potential improvements with regard to pain issues: Patients showed significant improvements in the SF-36 questionnaires, particularly in the areas of physical health, vitality, and general well-being. Such improvements could indicate a reduction in pain or better pain management.
A significant decrease in the Mainz Severity Score Index (FOS-MSSI) was observed, suggesting an overall reduction in disease burden.
Kidney function remained stable or improved, suggesting effective control of disease progression.
Fabry disease can have a huge impact on the quality of life of those affected:
"Death of bone tissue a rare, painful #Fabry complication: Case report"
https://t.co/MkWJ0j7Tdv
...all important parameters going in the same direction: Gene therapy is effective and improves both objective (eGFR) and subjective parameters (pain). Accelerated approval of #ST920 is coming! $SGMO
#Fabry $SGMO #ST920 #Isavec #STAAR
An interim analysis (data cut-off: September 12, 2024) showed a positive average annual eGFR increase of +3.061 mL/min/1.73 m² per year in a cohort of 23 patients who were followed for at least 12 months. This indicates a significant improvement in kidney function. An earlier interim analysis (cut-off: September 19, 2023) showed nearly stable kidney function in 13 patients with an eGFR increase of -0.915, indicating a slowing of the typical functional decline in FD. The improvement in the average eGFR from 13 to 23 patients who were followed up for AT LEAST 12 months suggests that the dramatic improvement in kidney function only becomes apparent over time (>12 months). If 10 additional study participants are included in the next analysis, then, depending on their study duration, a lower average eGFR value than in the interim analysis is possible, especially if the eGFR values are consolidated after exactly 12 months. It is important that the eGFR values remain positive over time, thus fulfilling the requirements for accelerated approval.
cut-off: September 19, 2023:
https://t.co/fQMfiUTscj
cut-off: September 12, 2024
https://t.co/ro5vYaP2F6
Improved diagnostics increase the potential for $SGMO Gene therapy #ST920
"Using AI offers ways to improve Fabry diagnosis, care: Study - Artificial intelligence tech may reduce misdiagnoses, per scientists"
https://t.co/nBJBDwR0Ns
@yusufhameed In terms of upfront payment, milestones for NDA submission and approval, I expect a total of at least $300m for $SGMO.
#Fabry #Isaralgagene_civaparvovec #ST920
@yusufhameed Regarding $SGMO / #ST920 I will say it again: #Isavec will win prizes, such as the Prix Galien. This is a prestigious international award for outstanding innovations in pharmaceutical research and medicine. Mark my words!
I stick to the facts:
There is a life-shortening (approx. 20y) indication (FD), in which the previous medications (ERT) only slow down the deterioration (kidney and heart function). Now there is a $SGMO gene therapy (#Isavec/#ST920), of which the interim data has convinced the drug regulatory authorities (FDA and EMA; several FDA members attended a EMA meeting!) to such an extent that a phase 1/2 study (STAAR) is being approved as a registration study and a phase 3 study is not required for approval. A potential licensee thus has no development costs for a ready-to-use gene therapy (usually up to $1b) that also has hardly any side effects. Every pharmaceutical company doing gene therapy research is a potential takeover candidate, because it is hard to imagine a better therapy outcome. For Fabry patients, there are many advantages (single application, improvement of kidney and heart function), but also from an overall economic point of view: an economic benefit is very likely despite the expected high price of the gene therapy (> $1m). Isavec comes very close to a cure!
@Quantumup1 $SGMO The Phase 3 initiation of #isaralgagene #civaparvovec ( #ST920 ) gene therapy in adults with #Fabry disease is of HIGHEST IMPORTANCE as based on the approved study details for example (single-arm trial) has a good chance to result in a Partnering. How naïve is #BOFA ?
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