Wed, Sep. 9, 9:17 AM
Mon, Aug. 10, 5:19 PM
Sun, May 10, 7:06 AM
- The World Health Organization has declared Liberia free of Ebola, marking the end of a national outbreak that infected as many as 400 new victims a week at its peak.
- Liberia has now gone 42 days - twice Ebola's maximum incubation period - since the burial of its last confirmed patient without discovering a new case.
- The disease is still spreading in Sierra Leone and Guinea, though at a slower pace. According to WHO statistics, more than 11,000 people have died from the virus, with about half of them in Liberia.
- Ebola-related stocks: JNJ, GSK, HEB, BCRX, NLNK, NNVC, INO, TKMR, OTCQB:GOVX, SRPT, CMRX, PLX, NSPH, LAKE, APT, VSR, SMED.
Thu, May 7, 5:43 PM
Tue, Apr. 21, 11:34 AM
- Sanofi's (SNY +0.4%) Genzyme unit initiates a Phase 2a clinical trial evaluating the pharmacodynamics of GZ/SAR402671, a new once-daily oral therapy for Fabry disease, a rare lysosomal storage disorder that results in the buildup of a type of fat, globotriasylceramide (GL-3), in the body's cells caused by a deficiency in a certain enzyme. It affects ~one in 50K males, predominantly, or ~10K total people worldwide.
- The primary endpoint of the nine-patient 26-week study is the reduction in accumulated GL-3 from the skin capillary endothelium. After its completion, all patients will have the option to enroll in an extension study.
- Genzyme's current offering for Fabry, Fabrazyme (agalsidase beta), replaces the deficient enzyme, alpha-galactosidase A. The FDA approved the intravenously-administered therapy in April 2003. It generated over $500M in sales for Sanofi last year. Annual therapy costs ~$200K per patient.
- Related tickers: (FOLD)(SHPG +1.8%)(PLX +1%)
Thu, Mar. 19, 7:41 AM
- Amicus Therapeutics (NASDAQ:FOLD) jumps 20% premarket on higher-than-normal volume in response to its announcement of accelerated schedules for its migalastat regulatory applications for the treatment of Fabry disease in the U.S. and Europe.
- Based on a Type C (late development stage) meeting with the FDA, the company intends to submit its New Drug Application (NDA) in H2. The agency agreed to review the application under its Accelerated Approval pathway. A Phase 4 (post-approval study) will be required so several protocol designs were discussed.
- Based on a meeting with the European Commission (EC), Amicus intends to submit its Marketing Authorization Application (MAA) in Q2 instead of mid-year. The company will request Accelerated Assessment of the MAA, which Rapporteurs (EC representatives assigned to provide guidance to application sponsors) indicated support for.
- Amicus will host a conference call this morning at 8:00 am ET to discuss its global regulatory strategy for migalastat monotherapy for Fabry disease.
- Related tickers: (NYSE:SNY) (NASDAQ:SHPG) (NYSEMKT:PLX)
Thu, Mar. 12, 4:50 PM
Thu, Feb. 12, 9:27 AM
- Thinly-traded micro cap Protalix BioTherapeutics (NYSEMKT:PLX) is up 7% premarket on above average volume in response its announcement of additional positive interim data from its Phase 1/2 dose-ranging study of the recombinant human alpha-Galactosidase-A enzyme, PRX-102, for the treatment of Fabry disease. The results will be presented this morning at the Lysosomal Disease Network WORLD Symposium in Orlando, FL.
- In the trial, 11 male and 7 female treatment-naive patients were enrolled across three dosing cohorts of 0.2 mg/kg, 1 mg/kg and 2 mg/kg. Intravenous infusions were administered every two weeks, with a six-month efficacy follow-up period.
- PRX-102 has a circulatory half-life of ~60 hours and an area under the curve (AUC) of ~70K ng/mL*hour for the 0.2 mg/kg dose, both substantially higher than currently approved enzyme replacement therapies.
- Patients in the 0.2 mg/kg cohort demonstrated stable cardiac and kidney function with favorable trends after six months. The leading causes of death of Fabry sufferers are cardiovascular disease and renal failure.
- Patient enrollment was completed earlier this month. All patients that completed the trial (11/12) elected to continue to receive PRX-102 in an open-label extension study. Protalix expects to report interim results for the 1 mg/kg cohort in Q3 and complete top-line results from all dosing cohorts in Q4.
Thu, Jan. 8, 9:15 AM
Mon, Jan. 5, 12:48 PM
Mon, Jan. 5, 9:30 AM
- Protalix BioTherapeutics (NYSEMKT:PLX) jumps 10% premarket on increased volume in response to its announcement of a plan to accelerate its growth by focusing on products with clinically superior profiles including PRX-102 for Fabry disease, PRX-106 for inflammatory bowel disease, PRX-110 for cystic fibrosis and PRX-112 for Gaucher disease.
Mon, Jan. 5, 9:10 AM
Nov. 21, 2014, 8:04 AM
- The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopts a positive opinion supporting approval of Celgene's (NASDAQ:CELG) Otezla (apremilast) for two indications: 1. the treatment of adult patients with moderate-to-severe plaque psoriasis who failed to respond to, or who are contraindicated for or are intolerant to, other systemic therapy including cyclosporine, methotrexate or psoralen and ultraviolet-A light (PUVA); 2. alone or in combination with Disease Modifying Antirheumatic Drugs (DMARDs) for the treatment of active psoriatic arthritis (PsA) in adult patients who have had an inadequate response or who have been intolerant to a prior DMARD therapy. A final decision from the European Commission usually takes about 60 days.
- Apremilast is a PDE4 inhibitor specific for cyclic AMP (cAMP). PDE4 inhibition results in increased cAMP levels which is thought to indirectly modulate the production of inflammatory mediators.
- The FDA approved Otezla in March.
- Previously: FDA approves Otezla
- Previously: Otezla label expansion cleared in the U.S.
- Related tickers: (NYSE:PFE) (NYSE:SNY) (OTCQB:SNYNF) (NYSEMKT:PLX) (NASDAQ:SHPG)
Nov. 21, 2014, 7:38 AM
- The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopts a positive opinion supporting approval for Genzyme's (NYSE:SNY) (OTCQB:SNYNF) Cerdelga (eliglustat) capsules for the treatment of certain adults with Gaucher disease type 1. A final decision by the European Commission usually takes about 60 days.
- The FDA approved Cerdelga in August. It was the first oral therapy approved as a first-line treatment for Gaucher.
- Previously: FDA approves new drug for Gaucher's disease
- Related tickers: (NYSE:PFE) (NYSEMKT:PLX) (NASDAQ:SHPG)
Nov. 20, 2014, 11:03 AM
- The Securities and Exchange Commission suspends trading in thinly-traded nano caps Bravo Enterprises (OTCQB:OGNG), Immunotech Labs (OTCPK:IMMB), Myriad Interactive Media (MYRY) and Wholehealth Products (OTCPK:GWPC) citing a lack of publicly available information about the firms' operations.
- The SEC can suspend trading in a stock for 10 days and prohibit broker-dealers from soliciting buyers and sellers until certain reporting requirements are met.
- Ebola Small Cap Index (based on trading behavior): (OTCQB:AEMD -7%)(HEB +1.4%)(TKMR +0.2%)(NLNK +5.4%)(NNVC +2.9%)(INO +5%)(OTCQB:GOVX -2.1%)(SRPT +3.5%)(CMRX -1.3%)(PLX -0.9%)(NSPH -1.4%)(BCRX +0.4%)(LAKE -1.9%)(APT -2.4%)(VSR)(SMED +0.7%)
Nov. 17, 2014, 12:06 PM
- Nano cap Aethlon Medical (OTCQB:AEMD +29.8%) jumps on massive turnover of 10M shares in response to its report of the successful treatment of a critically-ill Ebola patient in Germany with its Hemopurifier bio-filtration device. In a post-treatment analysis, the device captured 242M Ebola viruses. The patient is expected to make a full recovery.
- The Hemopurifier targets the broad-spectrum elimination of viruses and immunosuppressive proteins from the blood.
- The stock has more than tripled since mid-October when the first treatment with the Hemopurifier was reported.
- Some of the members of the trading behavior-characterized Ebola Small Cap Index have completed their round trips. (HEB +1.2%)(TKMR +5.1%)(NLNK +2.5%)(NNVC -3.8%)(INO -8.5%)(OTCQB:GOVX)(SRPT -0.7%)(CMRX +4.4%)(PLX -1%)(NSPH -0.2%)(BCRX +0.7%)(LAKE +1.3%)(APT +0.2%)(VSR -0.7%)(SMED -2.7%)
PLX vs. ETF Alternatives
Protalix BioTherapeutics Inc is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins based on itsproprietary ProCellEx protein expression system, or ProCellEx.
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