Fri, Jun. 19, 5:36 PM
Fri, Jun. 12, 9:55 AM
- Initial results from an ongoing Phase 1/2 study evaluating Alnylam Pharmaceuticals' (NASDAQ:ALNY) ALN-CC5 for complement-mediated diseases showed that a single subcutaneous dose of ALN-CC5 in 12 healthy volunteers resulted in a knockdown of serum C5 of up to 96% and achieved inhibition of serum complement activity of up to 92%. The data were presented at the 20th Congress of the European Hematology Association in Vienna, Austria.
- ALN-CC5 is a first-in-class C5 synthesis inhibitor under development for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). Phase 2 enrollment is expected to commence by the end of the year.
- PNH is a rare, chronic debilitating disorder characterized by the breakdown of red blood cells by the complement system with releases of hemoglobin in the urine. Sufferers have dark-colored urine in the morning. It is caused by a defect in the formation of surface proteins on red blood cells. The only approved drug to treat the disorder is Alexion Pharmaceuticals' (ALXN +1.1%) blockbuster Soliris (eculizumab) which generated $2.2B in sales in 2014. It is the world's second most expensive drug (after uniQure's Glybera).
- C5 (fifth component of complement) plays a key role in inflammation and cell killing processes. The complement system is part of the innate immune system that helps (complements) it to clear pathogens.
- Under its 2014 global alliance with Sanofi's (SNY -2.6%) Genzyme, Alnylam retains the rights to ALN-CC5 in North America and Western Europe while Genzyme owns the rights in the rest of the world. Genzyme, under certain circumstances, can opt-in to co-develop and co-commercialize product candidates which could include global product rights.
Thu, Jun. 11, 8:10 AM
- Responding to Alnylam's (NASDAQ:ALNY) lawsuit accusing it of misappropriating trade secrets, Dicerna Pharmaceuticals (NASDAQ:DRNA) says the case is without merit and it will vigorously defend itself in the matter.
- The company says that earlier this year Alnylam was concerned about certain aspects of Dicerna's GalNAc delivery technologies. Dicerna conducted an internal investigation and confirmed that no Merck/Alnylam confidential information had been used, a finding that it communicated to Alnylam. It also says that many of the insinuations in Alnylam's complaint are either false or based on unfounded speculation.
- Previously: Alnylam sues Dicerna Pharma over trade secrets (June 10)
Wed, Jun. 10, 4:35 PM
- Alnylam Pharmaceuticals (NASDAQ:ALNY) files a lawsuit in Massachusetts against Dicerna Pharmaceuticals (NASDAQ:DRNA) accusing it of misappropriating trade secrets Alnylam acquired via its buyout of Sirna Therapeutics from Merck in January 2014.
- The suit accuses Dicerna of obtaining its trade secrets by hiring six scientists from Merck who were involved in RNAi work, despite each of them signing a confidentiality agreement. It alleges that Dicerna's subsequent product candidates and delivery technologies are "strikingly similar" to Sirna's/Merck's. It also alleges that certain scientists hired by Dicerna were seen taking material out of the Merck facility prior to their departure.
Mon, Jun. 8, 7:21 AM
Wed, May 20, 7:46 AM
- The FDA designates Alnylam's (NASDAQ:ALNY) revusiran an Orphan Drug for the treatment of transthyretin-mediated amyloidosis, a genetic disorder characterized by the buildup of proteins called amyloid in the body's organs and tissues. It is caused by mutations in the TTR gene which provides instructions for producing transthyretin, a protein that transports vitamin A and thyroxine throughout the body. The buildup of beta amyloid in the brain is associated with Alzheimer's disease, a well-known example of the condition.
- Revusiran, an RNAi therapeutic that employs small interfering RNA (siRNA) targeting wild-type and all mutant forms of TTR, utilizes the company's proprietary GalNAc-siRNA conjugate delivery platform which enables subcutaneous (injection under the surface of the skin) delivery of the therapeutic.
- Among the benefits of Orphan Drug status is a seven-year period of market exclusivity for the indication, if approved.
Fri, May 15, 5:38 PM
Fri, May 8, 10:18 AM
- Alnylam Pharmaceuticals (ALNY +2.6%) Q1 results: Revenues: $18.5M (+122.9%); R&D Expense: $58M (+32.4%); SG&A: $12.7M (+42.7%); Operating Loss: ($52.2M) (+80.6%); Net Loss: ($50.8M) (+79.8%); Loss Per Share: ($0.62) (+83.2%); Quick Assets: $1,450.8M (+64.5%).
- 2015 Guidance: Cash, Cash Equivalents and Total Marketable Securities: > $1.2B.
Thu, May 7, 4:01 PM
Wed, May 6, 5:35 PM
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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.
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
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