Yesterday, 9:16 AM
Yesterday, 7:53 AM
- The Medicines Company (NASDAQ:MDCO) jumps 20% premarket on average volume in response to development partner Alnylam's (NASDAQ:ALNY) announcement that its investigational RNAi therapeutic, ALN-PCSsc, lowered LDL-C (bad cholesterol) up to 83% with a mean maximum reduction of up to 64% (+-5%) in an early stage study, results comparable to Amgen's (NASDAQ:AMGN) Repatha (evolocumab) and Sanofi (NYSE:SNY) and Regeneron's (NASDAQ:REGN) Praluent (alirocumab). The data were presented at the ESC Congress in London.
- What's notable in this case is the difference in dosing regimens. ALN-PCSsc was administered in one subcutaneous dose that was effective for over 140 days, giving it the potential for once per quarter or twice per year administration. Praluent is dosed once every two weeks and Repatha once every two weeks or once per month at a higher dose.
- ALN-PCSsc turns off PCSK9 synthesis in the liver. This is a different mechanism of action compared to Praluent and Repatha, both of which bind to PCSK9 in the blood.
- The Medicines Company will take the lead in developing ALN-PCSsc under the ORION Program. A Phase 2 study will commence by the end of the year and a Phase 3 trial is planned for 2017. The clinical development will include comparisons to the anti-PCSK9 monoclonal antibodies.
- The companies will host a conference call this morning at 9:30 am ET to discuss the results and their development plan.
Fri, Jun. 19, 5:36 PM
Fri, May 15, 5:38 PM
Tue, Apr. 21, 9:22 AM
Tue, Apr. 21, 8:47 AM
- At the 67th Annual Meeting of the American Academy of Neurology, Alnylam (NASDAQ:ALNY) presented 12-month clinical data from its ongoing Phase 2 open-label extension study of its RNAi therapeutic, patisiran, in development for the treatment of transthyretin-mediated amyloidosis in patients with familial amyloidotic polyneuropathy (FAP), a genetic disorder characterized by abnormal deposits of the protein amyloid in the body's tissues and organs. It is caused by mutations in the TTR gene, which provides instructions for producing transthyretin (TTR).
- The results showed a mean 2.5-point decrease in modified Neuropathy Impairment Score at 12 months in patients who had reached the 12-month endpoint (n=20) at the time of data cutoff. This compares favorably with the 13 - 18-point increase cited in the literature in untreated FAP patients with similar baseline characteristics. Patients treated with patisiran achieved a sustained mean serum TTR knockdown at the 80% target level for ~16 months, with up to an 88% mean knockdown between doses. Researchers believe that TTR knockdown has the potential to halt neuropathy progression in FAP patients.
- 18-month data should be available later this year. The company will host a conference call this morning at 9:00 am ET to discuss the results.
- Shares are up 9% premarket on light volume.
Dec. 23, 2014, 12:45 PM
Nov. 7, 2014, 5:35 PM
Nov. 5, 2014, 4:19 PM
Oct. 13, 2014, 9:15 AM
Aug. 7, 2014, 4:12 PM
Jun. 13, 2014, 11:41 AM
- Shares of Arrowhead Research (ARWR -16.1%) are down on heavy volume in response to the patent award to Alnylam Pharmaceuticals (ALNY -0.2%) expanding its McSwiggen patent estate for RNAi therapeutics. The just-awarded patent (no. 8,618,277) covers the development of RNAi therapeutics for HBV. One of Arrowhead's lead product candidates is ARC-520, an RNAi-based therapeutic for the treatment of chronic HBV infection.
May 12, 2014, 9:26 AM
- Certain traders are apparently swinging from trees this morning as they pop shares of Alnylam (ALNY) 8% on modest volume in premarket action in response to its announcement of a significant reduction in HBV surface antigen in preclinical testing on four monkeys with its RNAi therapeutic ALN-HBV product candidate. The product reduced HBsAg as much as 2.3 log10 compared to 0.5 log10 which is the generally accepted minimum reduction indicating immunologic cure. While encouraging, a colossal amount of work and time remains before any such product will reach the market.
- Alnylam purchased the assets from Merck (MRK) in January for $175M ($25M cash + stock). Merck acquired the assets via its acquisition of Sirna Therapeutics in 2006 for $1.1B. Merck's exit at a significant loss is a clear signal that the road to commercialization will be long and arduous.
- Alnylam hopes to file an IND by the end of 2015.
May 12, 2014, 9:12 AM
Apr. 15, 2014, 12:06 PM
- Shares of RNA interference firm Tekmira Pharmaceuticals (TKMR -14.5%) continue their slide in early trading. Prices have cratered 60% since the 6x run-up peaked at $31.48 on March 13.
- Although Big Pharma has lost much of its enthusiasm for RNA interference, some analysts remain bullish. RBC Capital has a $30 PT on the stock and Maxim Group has $31. Stifel Nicolaus is a bit more modest with $19.
- Most of the firm's 2013 revenue of $15.5M came from its contract with the DOD for the development of an Ebola treatment ($9.8M) and a milestone payment from Alnylam (ALNY -4.5%) ($5M) that was triggered by the start of the Phase 3 clinical trial for ALN-TTR02 (patisiran).
Feb. 24, 2014, 12:04 PM
- "When we first launched coverage of Tekmira (TKMR +8.9%) on June 18, 2013 at $4.80 we modeled our expectations with a high degree of conservatism using a 30% risk rate across the complete model," says analyst Jason Kolbert. "We are now lowering that discount rate from 30% to 15%."
- The new price target of $31 is up from $17 previously.
- The lower discount rate is thanks to progress of Tekmira partners Alnylam (ALNY -1.1%), Marqibo, and Monsanto, and the ever-rising evidence of the viability of SiRNA and the platform required to deliver it. He notes the gains in Isis Pharma last week amid preliminary test data, and the continued rise in Alnylam since Sanofi agreed to pay $700M for access to rare-disease treatments, and took a 12% stake in the company.
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