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▎WuXi AppTec Content Team Editor On January 21, 2022, Alnylam Pharmaceuticals announced positive results from a Phase 3 trial of its investigational RNAi therapy vutrisiran
.
After meeting its primary endpoint at month 9, the trial met all of its secondary endpoints at month 18, hereditary transthyretin amyloidosis polyneuropathy (hATTR-PN) compared with placebo in adults Patients showed significant improvements in multiple measures including neuropathic impairment, quality of life (QoL), gait speed, nutritional status, and overall disability
.
In addition, the reduction of serum TTR in patients in the vutrisiran group met the non-inferiority criteria compared to another approved RNAi therapy, patisiran
.
Vutrisiran has been granted Fast Track designation by the US FDA with a PDUFA target date of April 14, 2022
.
hATTR amyloidosis is an inherited, progressive, debilitating and fatal disease caused by mutations in the TTR gene
.
The TTR protein is mainly produced in the liver and is normally a carrier of vitamin
A.
Variations in the TTR gene lead to abnormal amyloid accumulation and damage to body organs and tissues, such as peripheral nerves and the heart, resulting in intractable peripheral sensory-motor neuropathy, autonomic neuropathy and/or cardiomyopathy, among other disease manifestations
.
hATTR amyloidosis has a significant unmet medical need, with patients having a median survival of only 4.
7 years after diagnosis
.
Vutrisiran is an investigational subcutaneous RNAi therapeutic being developed for the treatment of ATTR amyloidosis, including hATTR and wild-type ATTR amyloidosis
.
It blocks wild-type and variant transthyretin production by targeting and silencing specific mRNAs
.
Vutrisiran is designed based on Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc conjugated delivery platform with increased potency and metabolic stability, potentially allowing for infrequent subcutaneous injections
.
Data from previous studies suggest that quarterly administration of vutrisiran to patients may help reduce deposits in tissues, promote clearance of TTR amyloid deposits, and hopefully restore function in these tissues
.
▲Introduction of Alnylam’s ATTR amyloidosis product pipeline (Image source: Alnylam official website) The Phase 3 clinical trial enrolled 164 patients with hATTR-PN, who were randomized in a 3:1 ratio to receive 25 mg vutrisiran every three months (n = 122) subcutaneously, or 0.
3 mg/kg patisiran intravenously every 3 weeks (n=42) for 18 months, and compared the results obtained with vutrisiran in another phase 3 clinical trial evaluating the efficacy of patisiran compared to the placebo group
.
Trial results at month 18 are shown below: The Vutrisiran group (n=122) had a mean decrease from baseline in the Modified Neuropathic Impairment Score (mNIS+7) of 0.
46 points compared to a mean increase of 28.
09 points in the placebo group (n=77).
48% of patients in the Vutrisiran group showed an improvement in mNIS+7 compared to 4% in the placebo group
.
Compared with a mean increase of 19.
8 points in the placebo group, the Norfolk QoL-DN score, which assessed quality of life, decreased by an average of 1.
2 points from baseline in the vutrisiran group; 57% of patients in the vutrisiran group experienced an improvement in quality of life from baseline, compared with 10% in the placebo group
.
Image source: Alnylam official website Compared with the placebo group, patients in the Vutrisiran group showed statistically significant improvements in measures of gait speed (10-MWT), nutritional status (mBMI) and disability (R-ODS)
.
The Vutrisiran group achieved rapid and sustained reductions in serum TTR levels, with a mean reduction of 88% from baseline, meeting the criteria for non-inferiority relative to the patisiran control group
.
In addition to meeting the trial's secondary endpoints, patients in the vutrisiran arm also demonstrated improvements in exploratory cardiac endpoints compared with placebo, continuing to support vutrisiran's potential to reduce cardiac amyloid burden and improve cardiac disease in patients
.
Ms.
Rena Nassr Denoncourt, TTR program leader at Alnylam, said: "The trial data show that vutrisiran's improvement in neuropathic impairment and quality of life observed at 9 months was maintained to 18 months, the treatment effect increased over time, and the safety The characteristics are encouraging
.
In addition, new data show that the majority of patients have reduced cardiac technetium uptake compared to baseline, indicating the possibility of amyloid regression
.
" Reference: [1] Alnylam Presents Positive 18-Month Results from HELIOS -A Phase 3 Study of Investigational Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy.
Retrieved January 21, 2022, from https://investors.
alnylam.
com/press-release?id=26396 Global biomedical health research progress
.
This article is for information exchange purposes only, and the views expressed in this article do not represent WuXi AppTec's position, nor do they represent WuXi AppTec's support or opposition to the views expressed in this article
.
This article is also not a treatment plan recommendation
.
For guidance on treatment options, please visit a regular hospital
.
.
After meeting its primary endpoint at month 9, the trial met all of its secondary endpoints at month 18, hereditary transthyretin amyloidosis polyneuropathy (hATTR-PN) compared with placebo in adults Patients showed significant improvements in multiple measures including neuropathic impairment, quality of life (QoL), gait speed, nutritional status, and overall disability
.
In addition, the reduction of serum TTR in patients in the vutrisiran group met the non-inferiority criteria compared to another approved RNAi therapy, patisiran
.
Vutrisiran has been granted Fast Track designation by the US FDA with a PDUFA target date of April 14, 2022
.
hATTR amyloidosis is an inherited, progressive, debilitating and fatal disease caused by mutations in the TTR gene
.
The TTR protein is mainly produced in the liver and is normally a carrier of vitamin
A.
Variations in the TTR gene lead to abnormal amyloid accumulation and damage to body organs and tissues, such as peripheral nerves and the heart, resulting in intractable peripheral sensory-motor neuropathy, autonomic neuropathy and/or cardiomyopathy, among other disease manifestations
.
hATTR amyloidosis has a significant unmet medical need, with patients having a median survival of only 4.
7 years after diagnosis
.
Vutrisiran is an investigational subcutaneous RNAi therapeutic being developed for the treatment of ATTR amyloidosis, including hATTR and wild-type ATTR amyloidosis
.
It blocks wild-type and variant transthyretin production by targeting and silencing specific mRNAs
.
Vutrisiran is designed based on Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc conjugated delivery platform with increased potency and metabolic stability, potentially allowing for infrequent subcutaneous injections
.
Data from previous studies suggest that quarterly administration of vutrisiran to patients may help reduce deposits in tissues, promote clearance of TTR amyloid deposits, and hopefully restore function in these tissues
.
▲Introduction of Alnylam’s ATTR amyloidosis product pipeline (Image source: Alnylam official website) The Phase 3 clinical trial enrolled 164 patients with hATTR-PN, who were randomized in a 3:1 ratio to receive 25 mg vutrisiran every three months (n = 122) subcutaneously, or 0.
3 mg/kg patisiran intravenously every 3 weeks (n=42) for 18 months, and compared the results obtained with vutrisiran in another phase 3 clinical trial evaluating the efficacy of patisiran compared to the placebo group
.
Trial results at month 18 are shown below: The Vutrisiran group (n=122) had a mean decrease from baseline in the Modified Neuropathic Impairment Score (mNIS+7) of 0.
46 points compared to a mean increase of 28.
09 points in the placebo group (n=77).
48% of patients in the Vutrisiran group showed an improvement in mNIS+7 compared to 4% in the placebo group
.
Compared with a mean increase of 19.
8 points in the placebo group, the Norfolk QoL-DN score, which assessed quality of life, decreased by an average of 1.
2 points from baseline in the vutrisiran group; 57% of patients in the vutrisiran group experienced an improvement in quality of life from baseline, compared with 10% in the placebo group
.
Image source: Alnylam official website Compared with the placebo group, patients in the Vutrisiran group showed statistically significant improvements in measures of gait speed (10-MWT), nutritional status (mBMI) and disability (R-ODS)
.
The Vutrisiran group achieved rapid and sustained reductions in serum TTR levels, with a mean reduction of 88% from baseline, meeting the criteria for non-inferiority relative to the patisiran control group
.
In addition to meeting the trial's secondary endpoints, patients in the vutrisiran arm also demonstrated improvements in exploratory cardiac endpoints compared with placebo, continuing to support vutrisiran's potential to reduce cardiac amyloid burden and improve cardiac disease in patients
.
Ms.
Rena Nassr Denoncourt, TTR program leader at Alnylam, said: "The trial data show that vutrisiran's improvement in neuropathic impairment and quality of life observed at 9 months was maintained to 18 months, the treatment effect increased over time, and the safety The characteristics are encouraging
.
In addition, new data show that the majority of patients have reduced cardiac technetium uptake compared to baseline, indicating the possibility of amyloid regression
.
" Reference: [1] Alnylam Presents Positive 18-Month Results from HELIOS -A Phase 3 Study of Investigational Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy.
Retrieved January 21, 2022, from https://investors.
alnylam.
com/press-release?id=26396 Global biomedical health research progress
.
This article is for information exchange purposes only, and the views expressed in this article do not represent WuXi AppTec's position, nor do they represent WuXi AppTec's support or opposition to the views expressed in this article
.
This article is also not a treatment plan recommendation
.
For guidance on treatment options, please visit a regular hospital
.
