realfast95
5 years ago
Sanofi enzyme replacement therapy shows mixed results in Pompe study
Jun. 16, 2020 8:24 AM ET|About: Sanofi (SNY)|By: Douglas W. House, SA News Editor
Sanofi (NASDAQ:SNY) announces positive results from a Phase 3 clinical trial, COMET, evaluating avalglucosidase alfa, an enzyme replacement therapy, in patients with late-onset Pompe disease, an inherited disorder characterized by the buildup of glycogen in the body's cells due to the dysfunction of a certain enzyme.
The study met the primary endpoint demonstrating non-inferiority (no worse than) to alglucosidase alfa (Lumizyme) as measured by the change in respiratory function but it failed to demonstrate superiority, the other primary objective.
Since superiority was not shown, statistical testing of the secondary endpoints was not done.
The safety profiles were comparable.
Development is ongoing.
realfast95
5 years ago
Amicus Therapeutics (FOLD) is seeing some action in the analyst community this week. Yesterday, both JP Morgan and H.C. Wainwright ($20 price target) reissued Buy ratings on this rare disease concern. Today, Citigroup does the same with $15 price target. Here is the commentary from Citi's analyst
The analyst attributes the weakness yesterday in shares of Amicus Therapeutics to Sanofi's (SNY) press release indicating that the company will release Phase 3 data for avalglucosidase alfa at a virtual science session on June 16. This is a key event for Amicus as this neoGAA would compete with its investigational drug AT-GAA, Bansal tells investors in a research note. Given the company issued a press release about hosting such an event, investors are likely putting a high probability to neoGAA's success, hence the weakness in Amicus shares, says the analyst. However, he sees enough differences between the respective Phase 3 programs that it is not easy to compare neoGAA data with AT-GAA on an apples to apples basis. Further, Amicus does not get much credit for AT-GAA in current valuation as fair value of the company is $10 per share on Galafold alone".
https://seekingalpha.com/instablog/498952-bret-jensen/5458158-biotech-analyst-run-down-for-tuesday
Glider549
5 years ago
Amicus was able to achieve positive interim results from a phase 1/2 study using AAV-CLN6 gene therapy to treat patients with Batten disease.
Another gene therapy, known as AT-GAA, is currently being explored in a phase 3 study to treat patients with Pompe disease; enrollment completion is expected end of 2019.
The 6-month and 24-month data using AT-GAA to treat patients with Pompe disease are expected to be presented at the World Muscle Society October 1-5, 2019.
Amicus Therapeutics had $575.7 million in cash as of June 30, 2019, with enough cash to fund its operations into 2021.
Kylef
9 years ago
Did u Stay in that one for today?
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