Clinical FDA partial hold stands as Avidity shows evidence of...

FDA partial hold stands as Avidity shows evidence of the first targeting RNA into muscle

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Avidity Biosciences has hinted that the latest phase 1/2 data of the first targeted delivery of RNA into muscle has been successful. The FDA (Food and Drug Administration) clinical partial hold did not stop the company from showing more interest in the siRNA therapy AOC 1001.

Avidity Biosciences is a decade-old biotech company that is headquartered in California, U.S. The company is working on new ideas and methods surrounding a new class of oligonucleotide-based treatments to cater to the rare disease community’s needs.

The latest details on Avidity’s lead myotonic dystrophy type 1 treatment were revealed which revived interest in the potential of the medicine- while also outlining the drawbacks. The company stated that it is in the process of resolving the partial hold that was placed in September and is working as fast as possible. Avidity also stated that it is working towards the conclusion of a side effect investigation. An update on the partial hold is expected by the end of the first quarter of next year.

However, the company was eager to promote the potential of AOC 100. Avidity was optimistic as it stressed its oligonucleotide-based platform’s ability to carry RNA directly into the muscle by flaunting biomarker data in 19 patients and safety data in 38 patients.

Arthur A. Levin, the Chief Scientific Officer (CSO) of Avidity Biosciences said, “Utilizing our AOC platform technology, we have demonstrated for the first time ever the successful targeted delivery of siRNA to muscle in humans, a major breakthrough for the field of RNA therapeutics.”

DMPK (myotonic dystrophy protein kinase) mRNA was significantly reduced in all patients treated with AOC 1001 while the average reduction was 45% according to the company. Among the mentioned patients, two groups were created, one of which received two doses of 2 mg/kg while the other received a single dose of 1 mg/kg. However, Avidity did not keep separate results for the two groups. The company did not provide the data for 4 mg/kg and 8 mg/kg doses.

Despite not showing the data for 4 mg/kg doses, the investor presentation disclosed that 50% of the patients evaluated faced an adverse event, 77% of which were those treated with 4 mg/kg doses. The unfavorable event in the 4 mg/kg dose group patients is said to be the cause of the partial hold. Another adverse event was also noticed in the 2 mg/kg dose group which was linked to opioid pain medication following elective surgery, according to the company.

While the company tries to uphold optimism and relevance, the corporate standing is moving towards oligonucleotide-based treatments. A multi-billion dollar research and licensing agreement was inked between British giant GlaxoSmithKline (GSK) and Wave Life Sciences, through which GSK got one of the greatest assets with a vision of evolving eight more programs.

Avidity is still planning to combine data from the MARINA trial in the coming year and progressing with two additional clinical-stage programs.

Avidity Biosciences has hinted that the latest phase 1/2 data of the first targeted delivery of RNA into muscle has been successful. The FDA (Food and Drug Administration) clinical partial hold did not stop the company from showing more interest in the siRNA therapy AOC 1001.

Avidity Biosciences is a decade-old biotech company that is headquartered in California, U.S. The company is working on new ideas and methods surrounding a new class of oligonucleotide-based treatments to cater to the rare disease community’s needs.

The latest details on Avidity’s lead myotonic dystrophy type 1 treatment were revealed which revived interest in the potential of the medicine- while also outlining the drawbacks. The company stated that it is in the process of resolving the partial hold that was placed in September and is working as fast as possible. Avidity also stated that it is working towards the conclusion of a side effect investigation. An update on the partial hold is expected by the end of the first quarter of next year.

However, the company was eager to promote the potential of AOC 100. Avidity was optimistic as it stressed its oligonucleotide-based platform’s ability to carry RNA directly into the muscle by flaunting biomarker data in 19 patients and safety data in 38 patients.

Arthur A. Levin, the Chief Scientific Officer (CSO) of Avidity Biosciences said, “Utilizing our AOC platform technology, we have demonstrated for the first time ever the successful targeted delivery of siRNA to muscle in humans, a major breakthrough for the field of RNA therapeutics.”

DMPK (myotonic dystrophy protein kinase) mRNA was significantly reduced in all patients treated with AOC 1001 while the average reduction was 45% according to the company. Among the mentioned patients, two groups were created, one of which received two doses of 2 mg/kg while the other received a single dose of 1 mg/kg. However, Avidity did not keep separate results for the two groups. The company did not provide the data for 4 mg/kg and 8 mg/kg doses.

Despite not showing the data for 4 mg/kg doses, the investor presentation disclosed that 50% of the patients evaluated faced an adverse event, 77% of which were those treated with 4 mg/kg doses. The unfavorable event in the 4 mg/kg dose group patients is said to be the cause of the partial hold. Another adverse event was also noticed in the 2 mg/kg dose group which was linked to opioid pain medication following elective surgery, according to the company.

While the company tries to uphold optimism and relevance, the corporate standing is moving towards oligonucleotide-based treatments. A multi-billion dollar research and licensing agreement was inked between British giant GlaxoSmithKline (GSK) and Wave Life Sciences, through which GSK got one of the greatest assets with a vision of evolving eight more programs.

Avidity is still planning to combine data from the MARINA trial in the coming year and progressing with two additional clinical-stage programs.

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