Tue, Sep. 6, 7:11 AM
- Sangamo BioSciences(NASDAQ:SGMO) announces that the FDA has granted orphan drug designation to SB-FIX, the company's zinc finger nuclease ("ZFN")-mediated genome editing product candidate for the treatment of hemophilia B.
- Sangamo expects to initiate a Phase 1/2 clinical study (SB-FIX-1501) in adult subjects with the disease in 2016.
- Management commentt: "By enabling targeted integration of a therapeutic factor 9 gene, our ZFN-mediated genome editing approach may offer hemophilia B patients a therapeutic option that has potential advantages over conventional gene therapy approaches. We will enroll adult hemophilia patients into our first clinical trial, however, our goal is to move into pediatric patients, a population we believe could particularly benefit from a treatment that has the potential to provide lifelong expression of therapeutic levels of Factor IX protein."
- Source: Press Release
Thu, Aug. 4, 12:55 PM
Thu, Aug. 4, 11:00 AM
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- Losers: SGMO -25%. TDOC -21%. INOV -17%. KOOL -13%. SEAS -10%. QUIK -9%. IMGN -9%. CF -8%. AMRN -8%. TRIP -8%. CRC -6%. USCR -6%.
Wed, Aug. 3, 7:06 PM
- After a healthy climb over the past two weeks (of more than 25%), Sangamo BioSciences (NASDAQ:SGMO) has dropped 21.9% and falling after hours in the wake of a Q2 earnings report featuring a net loss that doubled Y/Y and revenues that fell off to $3.7M.
- The bulk of those sales came from collaboration agreements that Sangamo has with Biogen and Shire; the drop of 55.7% is largely due to amending its Shire deal last Q3, which returned rights to hemophilia programs to Sangamo.
- The company's in transition from a research-focused organization to a clinical-stage product development company "capable of building a versatile portfolio of therapeutics based on both our proprietary zinc finger nuclease and gene therapy platforms," says CEO Sandy Macrae, who joined this summer from Takeda Pharmaceuticals.
- Research and development expense came to $19.5M vs. a year-ago $15.6M. Total operating expenses had grown nearly 50%, to $30.5M.
- Sangamo has cash, equivalents, marketable securities and interest receivable of $172.6M.
- Press Release
Wed, Aug. 3, 5:35 PM
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Thu, Jul. 28, 11:32 AM
- Long-term follow-up data from a 16-subject Phase 1/2 clinical trial assessing Roche's (OTCQX:RHHBY) investigational biologic emicizumab in patients with severe hemophilia A show encouraging safety and prophylactic efficacy regardless of the presence of factor VIII inhibitors. The results were presented at the World Federation of Hemophilia 2016 World Congress in Orlando, FL.
- The data show patients who received once-weekly subcutaneous injections of either 0.3,1.0 or 3.0 mg/kg/week of emicizumab with median follow-up periods of 32.6 months, 27.0 months and 21.4 months, respectively, experienced a sustained decrease of 95% in annualized bleeding rate (ABR), irrespective of their inhibitor status and prior treatment regimen. The ABRs for the three cohorts were 1.4, 0.2 and 0.0, respectively.
- The product candidate's safety profile was consistent with what was previously reported in the Phase 1 study. No thromboembolic (blood clot that breaks loose and clogs another vessel) adverse events or clinically significant laboratory abnormalities were observed.
- Emicizumab is an bispecific antibody engineered to bind to both factors IXa and X, replacing the function of the missing factor VIII, thereby improving clotting function and preventing spontaneous bleeding. It was created by Chugai Pharmaceutical Co. and is being co-developed by Roche. Future clinical trials will explore less frequent dosing schedules.
- Related tickers: (OTC:BIOVF)(NASDAQ:BIIB)(BXLT)(OTCPK:BAYRY)(NASDAQ:OPK)(NYSE:SNY)(NASDAQ:ALNY)(NASDAQ:SGMO)(NASDAQ:CBIO)(NASDAQ:BMRN)
Wed, Jun. 1, 7:48 AM
- Sangamo BioSciences (NASDAQ:SGMO) President & CEO Edward Lanphier retires from the day-to-day leadership of the company. He will continue to serve on the board and will assume the chairmanship immediately following the annual stockholders meeting on June 14, succeeding current Chairman William Ringo.
- Succeeding Mr. Lanphier as President & CEO is Alexander "Sandy" Macrae, M.B., Ch.B., Ph.D., MRCP. He joins the firm from Takeda Pharmaceuticals where he was Global Medical Officer.
Thu, May 26, 5:09 PM
- The FDA approves CSL Behring's AFSTYLA [Antihemophilic Factor (Recombinant) Single Chain], its long-acting recombinant factor VIII single-chain therapy for the treatment of adults and children with hemophilia A.
- AFSTYLA is indicated for routine prophylaxis to reduce the frequency of bleeding episodes with dosing of two-to-three times per week, on-demand treatment and control of bleeding episodes and the perioperative management of bleeding.
- Market launch will commence this summer.
- Hemophilia A-related tickers: (NYSE:BXLT)(OTCPK:BAYRY)(NASDAQ:BMRN)(NYSE:OPK)(NYSE:SNY)(NASDAQ:ALNY)(NASDAQ:BIIB)(NASDAQ:SGMO)(OTCQX:RHHBY)(NASDAQ:CBIO)
Mon, May 16, 12:21 PM
- Novo Nordisk (NVO +1.1%) submits its Biologics License Application (BLA) to the FDA seeking approval of its long-acting factor IX, nonacog beta pegol, for the treatment of patients with hemophilia B. The glycopegylated recombinant factor IX has a 5x longer half-life than standard factor IX products which enables once-weekly dosing.
- Related tickers: (BIIB +1.3%)(DMTX +8%)(QURE +2.9%)(SGMO -3.3%)(BAX +0.4%)
Fri, May 13, 7:36 AM
- The European Commission approves Biogen (NASDAQ:BIIB) and collaboration partner Swedish Orphan Biovitrum AB's (OTC:BIOVF) Alprolix (Coagulation Factor IX [Recombinant], Fc Fusion Protein) for the treatment of hemophilia B. It is administered every 7-10 days so it provides prolonged protection against bleeding episodes as well as on-demand treatment.
- The data supporting the marketing application was generated in two Phase 3 trials, B-LONG, in adults and adolescents, and Kids B-LONG, in children under age 12.
- Swedish Orphan has final development and commercialization rights in Europe, North Africa, Russia and most of the Middle East. Biogen has commercialization rights in North America and the rest of the world ex-Swedish Orphan territory.
- Alprolix in now approved in the EU, U.S., Canada, Japan, Australia, New Zealand and certain other countries.
- Previously: Biogen's hemophilia B med successful in Phase 3 trial in children (Feb. 27, 2015)
- Related tickers: (NYSE:NVO)(NASDAQ:DMTX)(NASDAQ:QURE)(NASDAQ:SGMO)(NYSE:BAX)
Wed, May 11, 12:03 PM
- The European Commission approves CSL Behring's Orphan Drug-tagged IDELVION [Coagulation Factor IX (Recombinant), Albumin Fusion Protein] for the treatment and prophylaxis of bleeding in patients of all ages with hemophilia B (factor IX deficiency). The labeling includes routine prophylaxis to prevent/reduce the frequency of bleeding episodes, on-demand control and perioperative management of bleeding. Market launch will commence in the next few months. Orphan Drug status means that IDELVION has a 10-year period of market exclusivity in the EU for the indication.
- IDELVION's principal value proposition is 14-day dosing. This is achieved via the maintaining of high levels of factor IX activity, above 5% over 14 days at 75 IU/kg, via the fusion with recombinant albumin.
- The FDA approved IDELVION in early March.
- Related tickers: (BIIB -0.8%)(NVO -0.1%)(DMTX -0.8%)(QURE -1.2%)(SGMO -0.2%)(BAX +1.9%)
Mon, May 2, 4:08 PM
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