Veda sells ALNY 83.02: Importantly, ALN-AS1 has been generally well tolerated, with no clinically significant drug-related adverse events reported to date.

Further, ALN-AS1 was found to be generally well tolerated with no clinically significant drug-related adverse events to date. In addition, through analysis of exosomal mRNA preparations from serum and urine, it was demonstrated that ALN-AS1 treatment resulted in potent, dose-dependent, and durable silencing of ALAS1 mRNA in liver. Inhibiting ALAS1 reduces the accumulation of ALA and PBG. ALN-AS1 targets the enzyme aminolevulinic acid synthase 1 (ALAS1), also involved in the heme biosynthesis pathway but upstream from PBGD. A third stage, Part C, will commence in early 2016 that will assess ALN-AS1 in AIP patients suffering from recurrent attacks.


Dean:
Unless Alnylam's drug reports continued efficacy in late-stage trials and the company prices its drug lower than competitors, there is an increased likelihood that the company will fail against the bigger players in the market.

Emiko:
The two drugs await results of larger cardiovascular trials to gain a wider approval and consequently, higher eventual sales.

Danelle:
It must also be noted that Praluent and Repatha have been approved on a restricted basis for hard-to-treat patient groups only on account of a lack of evidence establishing their benefit in lowering risk of cardiovascular diseases.

Dann:
Chances are PBMs will cover the cholesterol drug that offers the lowest price, in a bid to initiate a price war in the cholesterol market similar to the one initiated in hepatitis C space earlier.

Latrice:
PCSK9 inhibitors are due to be priced over $14,000 a year, before discounts, which has already attracted much criticism from health insurers and pharmacy-benefit managers (PBMs), as standard statins cost no more than $50 per month.

Denise:
CEO Clive Meanwell, ALN-PCSsc will be cheaper to develop than PCSK9 inhibitors, however, its eventual price tag is yet to be seen.

Belkis:
Jefferies analysts have estimated peak sales for ALN-PCSsc at $980 million, after a 50% risk discount as the drug is still early-stage.

Estrella:
In contrast, the single-dose average LDL cut of Praluent in Phase 3 studies ranged from 36-50%, while Repatha was approved at a 60% mean drop.

Theda:
In a trial consisting of 69 patients, a 300 mg dose of the drug helped lower low-density lipoprotein (LDL) level to an average of 44% in 140 days.

Ewa:
ALN-PCSsc uses a technique called RNA interference (RNAi) to prevent PCSK9 protein from being produced in the body, instead of inhibiting once it has been made.

Alnylam Pharmaceuticals (NASDAQ:ALNY)
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