Yesterday, 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
Nov. 5, 2014, 4:19 PM| Comment!
Nov. 5, 2014, 12:52 PM
- Alnylam (ALNY -0.5%) completes the enrollment of 26 patients in its Phase 2 study of revusiran (ALN-TTRsc) for the treatment of transthyretin (TTR) cardiac amyloidosis. Each subject received a five-week course of treatment.
- The Phase 2 open-label extension study is now open for enrollment. The trial will assess the safety and tolerability of long-term dosing of revusiran for up to two years. The company plans to report clinical data about once per year with the initial report expected in 2015.
- A Phase 3 trial evaluating revusiran in TTR cardiac amyloidosis is on track to start later this year.
Nov. 4, 2014, 5:35 PM
- ABTL, ACAS, ACLS, ALNY, ALSK, ANDE, AR, AREX, ASH, ATO, ATSG, AVG, AWK, BALT, BCOR, BIOS, BKD, BREW, BWC, CBS, CDE, CF, CLR, CODI, CPE, CSOD, CTL, CVG, CXO, DATA, DEPO, DK, DPM, DRYS, DVR, DYN, ECOL, ECYT, EFC, ENS, ENSG, ETE, ETP, EXAR, EXXI, FMI, FNV, G, GNW, GPOR, GSM, HOLX, HTCH, IL, INWK, IO, JCOM, KGC, KW, LBTYA, LHCG, LPSN, MBI, MCHX, MCP, MDR, MDWD, MED, MHLD, MIDD, MRIN, MTDR, MTRX, MUSA, MWE, NDLS, NLY, NVAX, NWSA, OILT, ORA, ORIG, OSUR, PAA, PLNR, PMT, PODD, POWR, PPO, PRGN, PRU, PTC, QCOM, QEP, QUAD, RGP, RNDY, RUTH, SB, SBY, SCTY, SD, SKUL, SLF, SSNI, SSRI, STMP, SUSP, SWIR, SWM, SXL, SYMC, SZYM, TCAP, TEAR, TEG, TERP, THOR, TNGO, TPC, TRAK, TROX, TRUE, TS, TSLA, TSRO, TTEC, TXTR, UHAL, VVUS, WBMD, WFM, WMC, WMGI, WR, WTI, WWWW, XPO, Z
Oct. 13, 2014, 12:46 PM
Oct. 13, 2014, 9:15 AM
Oct. 13, 2014, 8:38 AM
- In a Phase 2 open-label extension study, Alnylam's (NASDAQ:ALNY) investigational RNAi therapeutic, patisiran (ALN-TTR02), demonstrated effectiveness as a treatment for patients with familial amyloidotic polyneuropathy (FAP) who have transthyretin (TTR)-mediated amyloidosis.
- The mean decrease in modified Neuropathy Impairment Score (mNIS+7) at six months in 19 patients was 0.95 points. The compares favorably with a historical 7 - 10 point increase in untreated patients.
- Patients treated with patisiran achieved a sustained mean serum TTR knockdown at the 80% target level for over nine months and as high as 89.6% between doses.
- The ongoing open-label extension study is designed to evaluate the long-term safety and tolerability of patisiran administration.
- The mNIS+7 measurement is the primary endpoint of the company's Phase 3 trial of patisiran in FAP patients.
- Shares are up 13% premarket on good volume.
Sep. 15, 2014, 11:22 AM
- Alnylam (ALNY -2.3%) hires three new executives. It appoints Karen Anderson as SVP, Chief HR Officer. She joins the firm from Biogen where she was VP, HR for R&D and Corporate Development.
- It appoints Laurie Keating as SVP, General Counsel and Secretary. She joins the company from Millennium where she was SVP, General Counsel and Secretary.
- It appoints Marko Kozul, M.D. as VP, Strategy. He joins the firm from Leerink Partners where he was a senior biotech analyst.
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