Tue, Apr. 21, 9:22 AM
Tue, Apr. 21, 8:47 AMAlnylam's patisiran shows promise of halting neuropathy progression in patients with genetic disorder
- 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.
Thu, Mar. 5, 8:06 AM
- Arrowhead Research (NASDAQ:ARWR) acquires Novartis' (NYSE:NVS) RNA interference (RNAi) R&D portfolio and associated assets, including certain patents and patent applications, an exclusive license to other patents and patent applications owned or controlled by Novartis, the assignment of a third party license and three preclinical product candidates.
- Specific assets include multiple patent families covering RNAi-trigger design rules and modifications that fall outside of competitors' patents which provides Arrowhead freedom to operate for any target or indication, the assignment of Novartis' license from Alnylam (NASDAQ:ALNY) that gives Arrowhead access to Alnylam IP, excluding delivery, covering 30 gene targets and three product candidates with varying amounts of preclinical data.
- Under the terms of the agreement, Arrowhead will pay $10M in cash (includes $7M paid previously) and $25M in ARWR stock within 30 days. Novartis is eligible to receive milestones and single-digit royalties on net sales.
- Arrowhead will host a conference call this morning at 8:30 am to discuss the deal.
Fri, Feb. 27, 9:59 AM
- Biogen Idec (BIIB -0.5%) and collaboration partner Swedish Orphan Biovitrum AB announce positive top-line results from a Phase 3 trial evaluating Alprolix (Coagulation Factor IX [Recombinant], Fc Fusion Protein) in children under age 12 with severe hemophilia B.
- Medical professionals recommend prophylactic treatment in children with severe hemophilia in order to manage and/or prevent bleeding episodes, but frequent administration schedules can be burdensome for patients and caregivers. Alprolix is administered once every 7 - 10 days for routine prophylaxis (prevention).
- In the study, children treated with Alprolix experienced an overall median annualized bleeding rate (ABR) of 1.97, while the median ABR for spontaneous joint bleeds was zero. About one third of the participants had no bleeding episodes at all. Overall, 91.7% of bleeding episodes were controlled by one or two infusions of Alprolix. None of the patients produced inhibitors (antibodies) to Alprolix in the study, which was the most significant potential complication of treatment.
- The European Medicines Agency requires the inclusion of pediatric study data in the initial Marketing Authorization Application (MAA) for a new hemophilia treatment. Interim data were used in the U.S. New Drug Application (NDA).
- Alprolix is the only approved hemophilia B therapy with prolonged circulation in the body. The FDA cleared it in March of last year. It generated $25.3M and $40.3M in sales for Biogen in Q3 and Q4, respectively.
- Related tickers: (BAX +0.3%)(OPK -0.8%)(SGMO -1.9%)(ALNY -1.9%)(ONCE +3%)(PFE -0.2%)(SHPG -1.6%)
Fri, Feb. 13, 9:21 AM
- Alnylam Pharmaceuticals (ALNY +0.5%) Q4 results: Revenues: $24M (+122.2%); R&D Expense: $55.6M (+73.2%); SG&A: $14.2M (+71.1%); Operating Loss: ($45.7M) (-54.4%); Net Loss: ($21.4M) (+34.0%); Loss Per Share: ($0.28) (+45.1%).
- FY2014 results: Revenues: $50.6M (+7.2%); R&D Expense: $190.2M (+68.3%); SG&A: $44.5M (+63.6%); Operating Loss: ($405M) (-336.0%); Net Loss: ($360.4M) (-304.0%); Loss Per Share: ($4.85) (-234.5%); Quick Assets: $881.9M (+151.6%).
- 2015 Guidance: Cash, Cash Equivalents and Total Marketable Securities: > $1.2B.
Thu, Feb. 12, 4:03 PM| Comment!
Wed, Feb. 11, 5:35 PM
Fri, Jan. 30, 12:31 PM
- Opko Health (OPK +3.5%) submits an Investigational New Drug Application (IND) to the FDA for clearance to commence a Phase 2a study evaluating its long-acting Orphan Drug-designated coagulation Factor VIIa (Factor VIIa-CTP) for the treatment of bleeding episodes in patients with hemophilia A or B with inhibitors to Factor VII or Factor IX.
- Currently, Factor VIIa therapy is administered intravenously which requires multiple infusions, due to its short half-life, to treat a bleeding episode.
- The longer duration of action of Opko's Factor VIIa-CTP is based on the naturally occurring peptide, C-terminal peptide, of the beta chain of human chorionic gonadotropin (hCG).
- Opko acquired the rights to the product when it bought Prolor Biotech.
- Previously: Opko completes its acquisition of Prolor Biotech (Aug. 29, 2013)
- Related tickers: (BAX +0.4%)(SGMO +0.6%)(ALNY +0.5%)(BIIB +11.4%)(OTCPK:BAYRY -1.4%)
Wed, Jan. 21, 7:29 AM
Tue, Jan. 20, 7:19 AM
Dec. 23, 2014, 12:45 PM
Dec. 15, 2014, 7:54 AM
- The Q-50 Index houses those companies next-eligible for inclusion into the Nasdaq 100 (NASDAQ:QQQ). Amid the Nasdaq 100's annual changes which added and dropped three from the index, the Q-50's quarterly re-ranking adds and drops eleven.
- Added: Ainylam Pharma (NASDAQ:ALNY), Expedia (NASDAQ:EXPE), F5 Networks (NASDAQ:FFIV), JD.com (NASDAQ:JD), MercadoLibre (NASDAQ:MELI), Maxim Integrated (NASDAQ:MXIM), Old Dominion Freight Line (NASDAQ:ODFL), Shire (NASDAQ:SHPG), Splunk (NASDAQ:SPLK), Stratasys (NASDAQ:SSYS), United Therapeutics (NASDAQ:UTHR).
- Dropped: Avis Budget (NASDAQ:CAR), Cree (NASDAQ:CREE), First Solar (NASDAQ:FSLR), Golar LNG (NASDAQ:GLNG), Methanex (NASDAQ:MEOH), Melco Crown (NASDAQ:MPEL), Nuance (NASDAQ:NUAN), SolarCity (NASDAQ:SCTY). Also dropped are the three Nasdaq 100 additions: American Airlines, Lam Research, and Electronic Arts.
- Source: Press Release
- Previously: Who's in, who's out in annual change to Nasdaq 100 (Dec. 13, 2014)
Dec. 11, 2014, 9:06 AM
- Alnylam Pharmaceuticals (NASDAQ:ALNY) earns a $10M milestone payment from The Medicines Company (NASDAQ:MDCO) by initiating a Phase 1 trial evaluating the safety and tolerability of Alnylam's cholesterol-lowering product candidate ALN-PCSsc in up to 76 volunteers with elevated baseline LDL-C. The U.K.-based study will be conducted in two stages: a single ascending dose and multiple doses. Initial data from the trial should be available by mid-2015.
- ALN-PCSsc is a PCSK9 inhibitor that is delivered via subcutaneous injection. Based on preclinical studies, it has the potential for a once-monthly or once-quarterly dose regimen. In non-human primate studies, the administration of ALN-PCSsc resulted in PCSK9 knockdown of up to 92% and lowering of LDL-C up to 77% without the co-administration of statins.
Dec. 9, 2014, 10:30 AM
- In a modest cohort of patients in a Phase 1 trial assessing Alnylam's (ALNY -2.2%) ALN-AT3, hemophilia subjects (n=4) receiving a once-weekly dose for three weeks of the antithrombin-targeting RNAi therapeutic demonstrated up to a 57% knockdown of antithrombin (AT), an endogenous anti-coagulant.
- In a cohort of healthy volunteers (n=4), the effects of a single dose of ALN-AT3 lasted for ~60 days.
- These are the first clinical data reported for the company's Enhanced Stabilization Chemistry (ESC)-GaINAc conjugate technology.
- EVP and CMO Akshay Vaishnaw, M.D., says, "...these data provide preliminary evidence for a high level of potency for RNAi therapeutics in humans with the improved pharmacologic properties for our ESC-GaINAc conjugate delivery technology. As supported by data we presented earlier this year, this improved potency appears to be due to the increased stabilization chemistry employed with our ESC platform as well as an attenuated nuclease environment in human cells."
- The results were presented at the American Society of Hematology meeting in San Francisco.
Nov. 14, 2014, 7:41 AM
- In a pilot Phase 2 study, Alnylam's (NASDAQ:ALNY) RNAi therapeutic, revusiran, demonstrated a mean maximum knockdown of 87.2% (+/- 9.1%) of serum transthyretin (TTR) in patients with TTR cardiac amyloidosis. The maximum knockdown observed was 98.2%.
- There were similar knockdown effects toward the wild type and mutant TTR protein within V122I patients, the most common genotype associated with inherited forms of TTR cardiac amyloidosis.
- Revusiran utilizes the company's proprietary GaINAc-conjugate delivery platform that enables a subcutaneous delivery of RNAi therapeutics with a wide therapeutic index.
- Alnylam will present the data this weekend at the American Heart Association meeting in Chicago.
Nov. 7, 2014, 5:35 PM
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