What's your position on ?
Why are you ish?
You voted ish on Vote again
Posts appear on the My Feed page of subscribers to this ticker
Aug. 5, 2015, 5:35 PM
- ABTL, ACAD, AHT, AIRM, AL, ALEX, ALNY, AMBR, AMRN, AMRS, ANAC, ANAD, ANET, ASEI, ASYS, ATHX, BBG, BEAT, BIO, BITA, BOJA, BRKS, BRS, CECO, CENX, CERS, CLVS, CPST, CSOD, CUB, CUBE, CVT, CYTX, DIOD, DMD, DRYS, EAC, ED, EFC, EGN, EGOV, EGY, ELON, EOG, ERII, ESPR, EVC, FLDM, FLTX, FPRX, FXCM, FXEN, GALE, GEOS, GNMK, GSBD, GXP, HNSN, HTGC, HUBS, IMI, IMPV, INWK, IRG, JMBA, KTOS, LGF, MAIN, MDVN, MHK, MNST, NDLS, NEWR, NFG, NPTN, NUAN, NUS, NVDA, OLED, ONTY, ORIG, OSTK, OUT, PACD, PETX, PLNR, PODD, POST, PRO, RBA, RBCN, RIGP, RJET, RMAX, RPTP, RRMS, SAAS, SEM, SEMG, SFM, SHO, SKUL, SPPI, SRC, SREV, SSRI, STMP, SWIR, TCRD, TCX, TEAR, TNGO, TPC, TRMR, TRUE, TRXC, TSRO, TWOU, UBNT, VRNS, VSAT, WAIR, WIFI, WING, XOMA, XOXO, ZNGA
Jul. 27, 2015, 11:05 AM
- Alnylam (ALNY -3.8%) commences a Phase 1/2 study assessing its RNAi therapeutic targeting alpha-1 antitrypsin, ALN-AAT, for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease). Initial data are expected in early 2016.
- The primary endpoint is safety as measured by the number of adverse events through days 70, 154 and 224. Secondary endpoints are the pharmacokinetic profile up to days 21, 105 and 161 and the effect of ALN-AAT on serum AAT protein levels through days 70, 154 and 224. The estimated study completion date is April 2017.
- ALN-AAT, administered via subcutaneous injection, utilizes the company's ESC-GalNAc-siRNA conjugate delivery technology.
- Alpha-1 antitrypsin (AAT) deficiency is an autosomal disorder that causes disease in the lungs and liver. Most (95%) people with AAT-deficiency carry two copies (homozygous) of an abnormal allele called PiZZ which expresses a protein called Z-AAT. The misfolding of mutant Z-AAT interferes with its normal release into the bloodstream causing it to accumulate in liver cells, leading to liver injury, fibrosis, cirrhosis and liver cancer. There are ~120K people in the U.S. and Europe with the PiZZ mutation. About 12K have the associated liver pathology. The only treatment options currently available for AAT-deficient patients is supportive care and, for those with advanced cirrhosis, liver transplantation.
Jul. 20, 2015, 10:07 AM
- Alnylam Pharmaceuticals (ALNY -0.9%) commences the Phase 3 open-label extension study of its RNAi therapeutic targeting transthyretin (TTR), patisiran, for the treatment of TTR-mediated amyloidosis in patients with Familial Amyloidotic Polyneuropathy (FAP). All patients who complete the randomized, double-blind Phase 3 study, APOLLO, will be eligible to enroll in the extension phase, called APOLLO-OLE.
- The primary endpoint of APOLLO is the change from baseline in modified Neuropathy Impairment Score (mNIS+7) over 18 months. The estimated study completion date is May 2017.
- FAP is an inherited disorder caused by mutations in the TTR gene. Sufferers produce variant TTR protein that results in the buildup of TTR amyloid deposits in the tissues, mainly in the nerves and heart. This eventually leads to organ damage and clinical symptoms similar to those of senile systemic amyloidosis.
Jun. 19, 2015, 5:36 PM
Jun. 12, 2015, 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.
Jun. 11, 2015, 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)
Jun. 10, 2015, 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.
Jun. 8, 2015, 7:21 AM
May 20, 2015, 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.
May 15, 2015, 5:38 PM
May 8, 2015, 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.
May 7, 2015, 4:01 PM
- Alnylam Pharmaceuticals (NASDAQ:ALNY): Q1 EPS of -$0.62 beats by $0.04.
- Revenue of $18.54M (+123.9% Y/Y) beats by $3.19M.
May 6, 2015, 5:35 PM
- ABTL, ACAD, ACET, ADVS, AGO, AHS, AHT, AIRM, AL, ALIM, ALJ, ALNY, ALSK, AMBR, AMH, ASYS, AVNW, BCEI, BEBE, BIOS, BKD, CA, CBS, CDXS, CERN, CNAT, CORT, CROX, CUTR, CYBR, DATA, DIOD, DXPE, EBS, ECPG, ECYT, ED, EGY, ENOC, ENV, ERII, ESPR, EVC, EZPW, FLDM, FRT, FTD, FUEL, GALE, GDOT, GEOS, GST, GXP, HTGC, IMPV, INWK, JAZZ, JCOM, JMBA, KEYW, KTOS, LBTYA, MAIN, MCHP, MDRX, MDVN, MED, MHK, MNST, MOH, MTD, MTRX, NGVC, NHI, NOG, NUAN, NVAX, NVDA, OLED, OMED, OUTR, PCTY, PETX, PLNR, POST, PRO, PSIX, RBA, RBCN, RIGL, RJET, RMAX, RMTI, RPTP, RRMS, SAAS, SEMG, SFM, SGMS, SLW, SPPI, SRC, SREV, SSNI, STMP, SWIR, TCX, TEAR, TGI, THOR, TLLP, TNGO, TPC, TRMR, TRUE, TWOU, UBNT, UEPS, WAIR, WIFI, XOMA, XOXO, YUME, ZNGA
Apr. 21, 2015, 9:22 AM
Apr. 21, 2015, 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.
Mar. 5, 2015, 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.
Other News & PR