Alnylam Pharmaceuticals, Inc. (ALNY) - NASDAQ
  • Wed, Feb. 10, 5:35 PM
  • Mon, Feb. 1, 12:48 PM
    • Alnylam (ALNY -2.7%) completes enrollment in its Phase 3 clinical trial assessing RNAi therapeutic patisiran for the treatment of transthyretin (TT)-mediated amyloidosis in patients with familial amyloidotic polyneuropathy (FAP), a rare inherited disorder characterized by abnormal protein aggregation in tissues and organs. It affects ~50K people worldwide.
    • According to, the estimated final data collection date for the primary endpoint is January 2017. The estimated study completion date is May 2017. The company does not intend to perform an interim analysis. If the results are positive, it plans to file regulatory applications in the U.S. and Europe in late 2017.
    • In a mid-stage study, treatment with patisiran knocked serum TTR protein levels down as much as 96%.
    • Previously: Alnylam's patisiran shows promise of halting neuropathy progression in patients with genetic disorder (April 21, 2015)
    | Mon, Feb. 1, 12:48 PM
  • Fri, Jan. 22, 8:23 AM
    • Credit Suisse analysts have been very busy lately with a long list of initiations: Acceleron Pharma (NASDAQ:XLRN) Outperform with $50 (47% upside) price target; BioMarin Pharmaceutical (NASDAQ:BMRN) Outperform with $110 (36% upside) price target; Amgen (NASDAQ:AMGN) Outperform with $205 (34% upside) price target.
    • Celgene (NASDAQ:CELG) Outperform with $149 (41% upside) price target; Coherus Biosciences (NASDAQ:CHRS) Outperform with $25 (79% upside) price target; Gilead Sciences (NASDAQ:GILD) Outperform with $125 (40% upside) price target.
    • Regeneron Pharmaceuticals (NASDAQ:REGN) Neutral with $552 (19% upside) price target; Vertex Pharmaceuticals (NASDAQ:VRTX) Outperform with $151 (59% upside) price target; Alnylam Pharmaceuticals (NASDAQ:ALNY) Outperform with $154 (117% upside) price target.
    • Alexion Pharmaceuticals (NASDAQ:ALXN) Neutral rating with $201 (29% upside) price target; Biogen (NASDAQ:BIIB) Neutral with $322 (23% upside) price target; XenoPort (NASDAQ:XNPT) Underperform with $4 (16% downside risk) price target.
    • PTC Therapeutics (NASDAQ:PTCT) Outperform with $36 (33% upside) price target; Mesoblast Limited (NASDAQ:MESO) Outperform with $10 (92% upside) price target and Incyte (NASDAQ:INCY) Outperform with $110 (49% upside) price target.
    | Fri, Jan. 22, 8:23 AM | 3 Comments
  • Dec. 18, 2015, 6:11 PM
    • Michael Bonney, an existing Alnylam Pharma (NASDAQ:ALNY) board member and until this year the CEO of Cubist Pharma, has been named Alnylam's chairman, effective Jan. 1.
    • Bonney replaces founding investor John Clarke, who has been chairman for the last 13 years. Clarke will remain on the board.
    • Alnylam also states David Pyott, the former chairman/CEO of Allergan, is joining the board.
    | Dec. 18, 2015, 6:11 PM
  • Nov. 11, 2015, 1:00 PM
    • Alnylam Pharmaceuticals (ALNY +1.1%) and development partner The Medicines Company (MDCO -0.8%) announce positive results from their ongoing Phase 1 clinical trial evaluating ALN-PCSsc for the potential treatment of high cholesterol. The data were presented at the American Heart Association Scientific Sessions in Orlando, FL.
    • As previously reported, subcutaneous administration of ALN-PCSsc reduced LDL-C (bad cholesterol) up to 83% (average maximum: 59 - 69%). New data showed the effects were highly durable and could support a twice/year dosing regimen, significantly longer than Amgen's (AMGN +0.6%) Repatha (evolocumab), dosed every two weeks or once/month, and Sanofi (SNY +0.3%)/Regeneron's (REGN +1%) Praluent (alirocumab), dosed every two weeks.
    • Specifically, the maximum PCSK9 knockdown was 89% (average 80.3 - 84.3%) and the maximum LDL-C reduction was up to 78% (average 54.3 - 64.3%) after a single injection of ALN-PCSsc. At day 180, LDL-C reduction was as high as 53% (average 47%) in the 300 mg cohort. No clinically significant drug-related adverse events were seen.
    • In the multiple dose cohorts (n=45), the maximum PCSK9 knockdown and LDL-C reduction were 94% (av. 86.9 - 90.1%) and 83% (av. 59.0 - 69.8%), respectively. At day 208, the LDL-C reduction was as high as 60% (av. 44.4%). In clinical trials, the mean LDL-C reductions observed for Repatha and Praluent were 64% (week 12) and 58% (week 24), respectively.
    • Both Repatha and Praluent bind to PCSK9 in the blood. ALN-PCSsc turns off PCSK9 synthesis in the liver.
    • The Medicines Company plans to initiate a Phase 2 study by year end with a Phase 3 trial to follow in 2017.
    • The companies will host a conference call this afternoon at 4:30 pm ET to discuss the data.
    | Nov. 11, 2015, 1:00 PM | 6 Comments
  • Nov. 9, 2015, 10:44 AM
    • Alnylam Pharmaceuticals (ALNY +4.8%) Q3 results: Revenues: $6.3M (-42.7%); R&D Expense: $68.6M (+48.2%); SG&A: $16M (+61.6%); Operating Loss: ($78.3M) (-73.2%); Net Loss: ($76.8M) (-74.5%); Loss Per Share: ($0.91) (-56.9%); Quick Assets: $1,336.3M (+51.5%).
    • No guidance given.
    | Nov. 9, 2015, 10:44 AM
  • Nov. 9, 2015, 7:11 AM
    • Alnylam Pharmaceuticals (NASDAQ:ALNY): Q3 EPS of -$0.91 in-line.
    • Revenue of $6.32M (-42.4% Y/Y) misses by $1.23M.
    | Nov. 9, 2015, 7:11 AM
  • Nov. 8, 2015, 5:30 PM
  • Nov. 4, 2015, 12:48 PM
    • Johnson & Johnson (JNJ) acquires privately held Novira Therapeutics, a clinical stage biopharmaceutical firm developing therapies to cure chronic hepatitis B infection within one year of treatment, for an undisclosed sum. The transaction should close this quarter.
    • Novira's lead product candidate is NVR 3-778, a orally available, small molecule, direct-acting antiviral that inhibits the HBV core or capsid protein. HBV core is an attractive drug target since it is plays a key role in viral replication and survival. NVR 3-778 disrupts the HBV life cycle by inducing the assembly of defective capsids. According to the company, its inhibitors, when used in combination with current standard-of-care drugs (nucleosides and interferon), should deliver greater and faster suppression of viral DNA and new virus production.
    • Selected HBV-related tickers: (ARWR +2.1%)(GILD -1%)(CTRV -3.2%)(ABUS +2.6%)(DVAX -1.8%)(MRK +0.7%)(SNY -0.8%)(GSK +0.1%)(ISIS +2.1%)(ALNY +3.1%)(BMY -0.6%)
    | Nov. 4, 2015, 12:48 PM | 11 Comments
  • Oct. 1, 2015, 9:33 AM
    • Sanofi's (NYSE:SNY) Genzyme elects to opt into Alnylam's (NASDAQ:ALNY) investigational ALN-AT3 hemophilia program for development and commercialization in territories outside of the U.S. and Western Europe. This is the first product from Alnylam's Genetic Medicines pipeline that Genzyme has elected to pursue since the formation of their global alliance in January 2014, although it represents Genzyme's third opt-in overall (revusiran and patisiran).
    • Under the terms of the alliance, Genzyme will fund 20% of the global development costs of ALN-AT3 beginning January 1 since it is considered a "regional" program.
    • ALN-AT3 is a subcutaneously administered RNAi therapeutic under investigation for the treatment of hemophilia and other rare bleeding disorders. Its mechanism of action is the knockdown of antithrombin, a naturally occurring anticoagulant.
    | Oct. 1, 2015, 9:33 AM
  • Sep. 15, 2015, 11:35 AM
    • The FDA approves privately-held Octapharma's NUWIQ, Antihemophilic Factor (Recombinant), an intravenous therapy for adults and children with hemophilia A. The approval includes on-demand treatment and control of bleeding episodes, routine prophylaxis to reduce the frequency of bleeding episodes and perioperative management of bleeding.
    • NUWIQ is the first B-domain-deleted recombinant Factor VIII derived from a human cell line, not chemically modified or fused with another protein, for the treatment of hemophilia A, which affects ~16K Americans.
    • NUWIQ was cleared in Europe in August 2014.
    • Related tickers: (BAX +0.3%)(BXLT -0.5%)(OTCQX:RHHBY +0.6%)(SGMO +2.4%) (OTCPK:CHGCY) (OPK +2.3%)(ALNY)(BIIB +0.5%)(OTCPK:BAYRY +0.4%)
    | Sep. 15, 2015, 11:35 AM | 6 Comments
  • Sep. 15, 2015, 9:03 AM
    • A Phase 1 study assessing Alnylam Pharmaceuticals' (NASDAQ:ALNY) investigational RNAi therapeutic, ALN-AS1, in patients with a rare liver condition called acute hepatic porphyria (AIP) showed encouraging results from just one subcutaneous injection, reducing two toxic substances 82% and 93%, respectively. The company is now progressing to Part B of the study in which patients will receive monthly injections. A third stage, Part C, will commence in early 2016 that will assess ALN-AS1 in AIP patients suffering from recurrent attacks.
    • Hepatic porphyria is caused by a deficiency in an enzyme involved in the heme (iron) biosynthesis pathway called porphobilinogen deaminase (PBGD). This leads to the accumulation of two toxic heme intermediates called aminolevulinic acid (ALA) and porphobilinogen (PBG). AIP episodes are characterized by severe abdominal pain, neuropathy and, in severe cases, cardiovascular instability, paralysis and respiratory failure. In the study, a single injection of ALN-AS1 reduced ALA up to 82% and PBG up to 93%.
    • The study participants carry the genetic mutation for AIP and are characterized as asymptomatic "high excreters" (ASHE) who have elevated levels 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. Inhibiting ALAS1 reduces the accumulation of ALA and PBG.
    | Sep. 15, 2015, 9:03 AM
  • Sep. 8, 2015, 10:40 AM
    • Based on encouraging preclinical data, Alnylam Pharmaceuticals (ALNY +2.2%) plans to initiate a Phase 1 clinical trial in early 2016 to investigate its RNAi therapeutic, ALN-GO1, for the treatment of primary hyperoxaluria type 1 (PH1), an ultra-rare liver disorder characterized by the overproduction of oxalate which leads to the accumulation of calcium oxalate kidney stones, irreparable kidney damage and end stage renal disease by the age of 30. It affects less than 1,000 Americans. There are no approved therapies for the condition.
    • ALN-GO1 targets the enzyme glycolate oxidase, referred to as hydroxyacid oxidase 1 (HAO1). In animal models, ALN-GO1 silenced up to 99% of the HAO1 mRNA and reduced up to 98% of mean urinary oxalate.
    • Dicerna Pharmaceuticals (DRNA +2.7%) is also developing a similar candidate, DCR-PH1. Its Phase 1 study should commence in the next few months.
    • Previously: Dicerna Pharma submits IND for DCR-PH1 for the treatment of rare liver disorder (Sept. 2)
    | Sep. 8, 2015, 10:40 AM
  • Aug. 31, 2015, 9:16 AM
    | Aug. 31, 2015, 9:16 AM
  • Aug. 31, 2015, 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.
    | Aug. 31, 2015, 7:53 AM | 8 Comments
  • Aug. 26, 2015, 6:57 PM
    • Biotech analysts at Piper Jaffray say they are buyers on weakness of four biotech names - Vertex Pharma (NASDAQ:VRTX), Novavax (NASDAQ:NVAX), Alnylam Pharma (NASDAQ:ALNY) and Arrowhead Research (NASDAQ:ARWR) - that boast strong enough balance sheets to make it through upcoming value-driving events.
    • The firm notes VRTX holds more than $1B in cash, and expects a dramatic increase in treatable cystic fibrosis patients with the newly approved Orkambi, which will help drive revenue growth and a return to near-term profitability.
    • Jaffray says it has increased confidence in NVAX following positive Phase 2 data in its RSV-F in elderly subjects; the company ended the quarter with $315M in cash, which can fund "at least one" pivotal RSV trial.
    • ALNY ended the recent quarter with $1.4B in cash, with key patisiran and revusiran Phase II in OLE updates expected in Q4.
    • On ARWR, the firm looks to an expected September update of Phase IIa HEPARC 2001 data.
    | Aug. 26, 2015, 6:57 PM | 14 Comments
Company Description
Alnylam Pharmaceuticals, Inc. is a biopharmaceutical company, which discovery, development and commercialization of RNAi therapeutics. It is the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases.... More
Sector: Healthcare
Industry: Biotechnology
Country: United States