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%)
Thu, Feb. 19, 10:10 AM
- Facing ever-increasing pushback from payers over the high price of new treatments, some biotech firms are working on a new payment model that rewards them for the long-term performance of their drugs. Gene therapy developers, including BioMarin Pharmaceuticals (BMRN +0.4%) and Sangamo BioSciences (SGMO -0.1%), are leading the effort.
- The industry says that a one-time cure, even if priced greater than $1M, saves money over the long term but acknowledges that payers will balk at paying the cost upfront. The general consensus is that an upfront payment over $1M will be unacceptable.
- Under a pay-for-performance scheme, the annuity-like payments would cease if medical testing showed that the therapy was not working. Much work remains to be done, though, before this approach could be implemented. For example, legislation may be required to force insurers to pick up the tab for patients that switch coverage.
- Some proponents of the new scheme say that the payment streams could be securitized and sold to investors, like consumer debt.
- Gene therapies represent the highest priced treatments. Only one has cleared the regulatory hurdle to date, UniQure's (QURE +1.5%) Glybera, which costs 850K euros ($1M) in Germany. It will be sold for a one-time payment because it is too difficult to measure how well it works (this reasoning will undoubtedly pop up again).
- Previously: UniQure sets price record with Glybera (Nov. 26, 2014)
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Tue, Feb. 17, 10:09 AM
Thu, Feb. 12, 5:10 PM
- The Phase 1/2 open label safety and optimal dosing trial evaluating Baxter International's (NYSE:BAX) gene therapy candidate, BAX 335, for the treatment of hemophilia B showed none of the six patients dosed to date developed Factor IX (FIX) inhibitors, although two from the highest dose cohorts exhibited FIX activity of ~10%.
- The trial will enroll up to 16 adult patients with hemophilia B to determine the safety of ascending doses of BAX 335 and the optimal single dose. The primary endpoint is the safety of a single dose of BAX 335 administered intravenously.
- As of the end of 2014, six patients in three dosing cohorts have been treated with evidence of a dose-related response. In addition to the two patients cited earlier, one showed elevated levels of liver enzymes which indicates an immune response. The patient is being treated with corticosteroids, per protocol. An immune response is a serious adverse event since it could lead to serious illness or even death.
- The clinical evaluation of BAX 335 is ongoing. The company is also advancing plans to evaluate the gene therapy technology in hemophilia A.
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%)
Thu, Jan. 29, 7:33 AM
- Baxter International (NYSE:BAX) Q4 results ($M): Total Revenues: 4,472 (+3.5%); U.S.: 1,887 (+6.0%); Intl: 2,585 (+1.7%); BioScience: 1,880 (+8.7%), U.S.: 951 (+6.7%), Intl: 929 (+10.7%); Medical Products: 2,592 (0.0%), U.S.: 936 (+5.2%), Intl: 1,656 (-2.7%).
- BioScience: Hemophilia: 1,055 (+8.5%); BioTherapeutics: 628 (+11.3%); BioSurgery: 197 (+1.5%); Vaccines: 34 (-26.1%).
- Medical Products: Fluid Systems: 822 (+0.4%); Renal: 1,082 (-1.5%); Specialty Pharma: 417 (+2.5%); BioPharma: 271 (+1.5%).
- COGS: 2,248 (-3.4%); R&D Expense: 335 (-15.4%); SG&A Expense: 1,091 (+3.5%); Net Income: 953 (+192.3%); EPS: 1.74 (+194.9%); CF Ops: 1,143 (+5.5%).
- Gross Profit: 2,224 (+11.4%); COGS: 50.3% (-6.6%); Gross Margin: 49.7% (+7.7%); Operating Earnings: 798 (+46.2%); Operating Earnings Yield: 17.8% (+41.3%); Net Earnings Yield: 21.3% (+182.5%).
- Q1 Guidance: Sales Growth: (3 - 4%); EPS ex. items: $0.85 - 0.90; GAAP EPS: $0.76 - 0.81.
Thu, Jan. 29, 7:05 AM
Wed, Jan. 28, 5:30 PM
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Tue, Jan. 20, 10:14 AM
- The FDA approves Baxter International's (BAX -0.1%) Phoxillum Renal Replacement Solutions (BK4/2.5 and B22K4/0) as replacement solutions in patients with acute kidney injury who are undergoing continuous renal replacement therapy (CRRT) to correct electrolyte and acid-base imbalances and, in the case of drug poisoning, when CRRT is used to remove dialyzable substances.
- Phoxillum is the only FDA approved premixed solution containing phosphate. Hypophosphatemia (low blood phosphate) is a common electrolyte problem in patients treated with CRRT.
- The company expects to launch Phoxillum phosphate-containing solutions in Q2.
Thu, Jan. 8, 7:55 PM
- The FDA approves Daiichi-Sankyo's (OTCPK:DSKYF) (OTCPK:DSNKY) Savaysa (edoxaban tablets) to reduce the risk of stroke and dangerous blood clots (systemic embolism) in patients with atrial fibrillation that is not caused by a heart valve problem.
- Edoxaban is an anticoagulant that inhibits factor Xa. It was approved in Japan in 2011 for the prevention of venous thromboembolism in patients undergoing total knee replacement surgery, total hip replacement surgery and hip fracture surgery.
- Anticoagulant-related tickers: (NASDAQ:ISIS) (NYSE:BAX) (NASDAQ:PTLA) (NASDAQ:MSLI) (NYSE:MRK) (NYSE:JNJ) (NASDAQ:MDCO) (NASDAQ:SCLN) (NYSE:PFE) (NYSE:BMY) (NYSE:AZN) (OTCPK:BAYRY)
Dec. 22, 2014, 11:10 AM
- Baxter International (BAX +0.6%) submits a Biologics License Application (BLA) to the FDA for BAX111, a highly-purified recombinant von Willebrand Factor (rVWF), for the treatment of von Willebrand disease, the most common form of inherited bleeding disorder.
- Both the FDA and EMA have designated the product candidate an Orphan Drug for the indication. If approved, it will have a seven-year period of exclusivity in the U.S. and ten years in the European Union.
Dec. 17, 2014, 4:50 PM
Dec. 15, 2014, 10:48 AM
- Baxter International (BAX) sells its Vero cell technology, related assets and its production facility in the Czech Republic to Alachua, FL-based Nanotherapeutics for an undisclosed sum. The transaction includes the assets related to vaccines for H5N1, H1N1, seasonal influenza as well as the investigational programs for Ross River virus, Chikungunya disease and West Nile virus.
- The Vero cell platform is an advanced cell-based technology for vaccines production.
Dec. 1, 2014, 9:51 AM
- Baxter International (BAX -0.4%) submits a biologics license application (BLA) to the FDA for BAX 855, an investigational extended half-life recombinant factor VIII treatment for hemophilia A based on Advate [Antihemophilic Factor (Recombinant)].
- The submission is based on the results from a Phase 3 trial of of 137 previously-treated patients. Patients in the twice-weekly prophylaxis arm experienced a 95% reduction in median annual bleed rate (ABR) compared to the on-demand arm. Also, 96% of bleeding episodes were controlled with one or two infusions.
- The company expects to present additional data from the trial in the coming months. The separate Phase 3 study in patients under the age of 12 with severe hemophilia is ongoing. After it concludes, Baxter expects to file a marketing authorization application (MAA) in Europe sometime in 2016.
Dec. 1, 2014, 8:10 AM
- Pfizer (NYSE:PFE) finalizes its buy of Baxter International's (NYSE:BAX) NeisVac-C (meningitis - MenC) and FSME-IMMUN/TicoVac (tick-borne encephalitis) vaccines and a portion of Baxter's facility in Orth, Austria where the products are made. The $635M deal was announced in late July.
- Previously: Pfizer buys Baxter's vaccine business
Nov. 11, 2014, 9:09 AM
Baxter International Inc is a healthcare company. It develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, infectious diseases, kidney disease, trauma and other chronic conditions.
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