Thu, Jul. 7, 10:38 AM
- The first patient has been dosed in a Phase 2 clinical trial assessing Protalix BioTherapeutics' (PLX +1.6%) AIR DNase (formerly PRX-110) for the treatment of cystic fibrosis (CF). The 28-day switch-over study involves 15 CF patients previously treated with Roche's (OTCQX:RHHBY) Pulmozyme (dornase alfa). The primary endpoint is the change from baseline in lung function as measured by forced expiratory volume in one second (FEV1) and forced vital capacity (FVC). Top-line data should be available around year end.
- According to the company, AIR DNase is a proprietary plant cell-expressed recombinant form of a human enzyme called deoxyribonuclease 1 (DNase 1) that cleaves extracellular DNA and thins the thick mucus that accumulates in the lungs of CF sufferers. It is designed to be more effective than Pulmozyme by being more resistant to actin inhibition, which restricts DNase I activity. Actin is a protein that plays a role in a wide range of cellular processes.
Mon, May 9, 7:16 AM
Fri, Apr. 8, 11:05 AM
- MacroGenics (MGNX +3.1%) initiated with Buy rating and $35 price target by BTIG Research.
- Viking Therapeutics (VKTX -39.4%) initiated with Buy rating and $6 price target by H.C. Wainwright.
- Pfizer (PFE -0.3%) initiated with an Overweight rating and $38 price target by JP Morgan.
- Medivation (MDVN -0.1%) initiated with Buy rating and $56 price target by SunTrust Robinson Humphrey.
- Eyegate Pharmaceuticals (EYEG +6.5%) initiated with Buy rating and $10 price target by Noble Financial.
- Eiger Biopharmaceuticals (EIGR +0.5%) initiated with Buy rating and $35 price target by Jefferies.
- Vertex Pharmaceuticals (VRTX +0.3%) initiated with Market Perform rating and a $90 price target by BMO Capital.
- SocGen has been busy with initiations on six large cap pharmas: Buy: Merck (MRK +0.1%); Hold: Johnson & Johnson (JNJ -0.4%), Eli Lilly (LLY -0.3%) and Pfizer; Sell: Bristol-Myers Squibb (BMY -0.4%) and AbbVie (ABBV -0.9%).
- Tokai Pharmaceuticals (TKAI -6.7%) initiated with Buy rating and $16 price target by Bank of America. Initiated with Buy rating by Janney Capital.
- Cellectis (CLLS -0.1%) initiated with Buy rating and $45 price target by Ladenburg Thalmann.
- Novartis (NVS +0.5%) initiated with Hold rating by Argus Research.
- TherapeuticsMD (TXMD) initiated with Buy rating and $10 price target by Goldman Sachs.
- Protalix (PLX -4.2%) initiated with Buy rating and $3.50 price target by Rodman & Renshaw.
- Raptor Pharmaceuticals (RPTP -0.4%) upgraded to Outperform from Market Perform by JMP Securities.
- Ironwood Pharmaceuticals (IRWD -0.6%) upgraded to Outperform from Market Perform by Cowen & Company. Price target raised to $16 from $12.
- Intercept Pharmaceuticals (ICPT -6%) upgraded to Outperform from Market Perform by Wells Fargo.
- Edwards Lifesciences (EW -0.1%) upgraded to Buy from Neutral with $115 price target by BTIG Research.
- Express Scripts (ESRX +0.7%) upgraded to Outperform from Market Perform by Cowen & Company. Price target raised to $80 from $76.
- BIND Therapeutics (BIND -7%) downgraded to Market Perform from Outperform by Cowen & Company.
- Allergan (AGN -0.5%) downgraded to Neutral from Buy by Mizuho Securities. Downgraded to Neutral from Positive by Susquehanna. Price target lowered to $275 from $345.
- Bruker (BRKR +0.2%) downgraded to Market Perform from Outperform by Leerink Swann. Price target is $27.
Tue, Mar. 8, 1:44 PM
Thu, Mar. 3, 10:22 AM
- Interim data from a Phase 1/2 dose-ranging study showed Protalix BioTherapeutics' (PLX +6%) PRX-102 reduced the levels of a key fat in the kidneys of Fabry disease patients. The results will be presented today at the Lysosomal Disease Network 12th Annual WORLDSymposium 2016 in San Diego, CA.
- The study enrolled 18 treatment-naive Fabry patients across three dosing cohorts (0.2 mg/kg, 1.0 mg/kg and 2.0 mg/kg). Subjects received an IV infusion of PRX-102 every two weeks. Efficacy was assessed at Months 6 and 12.
- Results from kidney biopsies in the 0.2 mg/kg and 1.0 mg/kg cohorts showed a mean reduction of a fat called Gb3 was 75.5% and 86.5%, respectively. No data were provided for the 2.0 mg/kg cohort. Gb3 or globotriaosylceramide, accumulates in the brain, heart and kidneys of Fabry sufferers. Cardiovascular disease and kidney failure are the leading causes of death in Fabry patients.
- Additional data showed consistent cardiac and renal function from baseline to Month 6 and 12 in both the 0.2 mg/kg and 1.0 mg/kg dosage groups.
- PRX-102 is chemically modified version of a recombinant therapeutic enzyme called alpha-galactosidase A.
- Fabry disease is an inherited disorder caused by mutations in the gene that codes for alpha-galactosidase A. It is characterized by the buildup of Gb3 in the body's cells.
- The company plans to initiate two Phase 3 studies that will support an NDA filing. The first should commence in Q2.
- Previously: Protalix Bio to initiate two late-stage studies of lead product candidate in Fabry disease in early 2016 (Nov. 16, 2015)
Dec. 10, 2015, 2:59 AM
- New Ebola cases have surfaced in Liberia, the worst hit of all Ebola-affected nations, suggesting that the fight against the disease could take months, or even years, to fully eliminate.
- Recent scientific studies and case reports indicate the Ebola virus can persist in survivors' bodily fluids months longer than previously thought.
- Liberia has already been declared Ebola-free (42 days pass without a new episode of hemorrhagic fever) twice this year, only to see new cases appear.
- Previously: Sierra Leone declared free of Ebola (Nov. 08 2015)
- Ebola-related stocks: JNJ, GSK, HEB, BCRX, NLNK, NNVC, INO, OTCQB:GOVX, SRPT, CMRX, PLX, NSPH, LAKE, APT, VSR, SMED
Nov. 16, 2015, 1:18 PM
- Based on feedback from the FDA at an End-of-Phase 2 meeting, Protalix BioTherapeutics (PLX +3.8%) intends to proceed with two Phase 3 trials assessing lead product candidate PRX-102 for the treatment of Fabry disease, an inherited disorder characterized by the buildup of a type of fat in the body's cells.
- One trial will be a randomized, multicenter, placebo-controlled study assessing the 1 mg/kg dose of PRX-102 in treatment-naive Fabry patients. The company believes that a small sample size will be sufficient to achieve statistical significance with a study duration of only six months. The primary endpoint will be the effect on gastrointestinal symptoms.
- The other trial will be a head-to-head superiority study comparing PRX-102 to Sanofi's (SNY -0.3%) Fabrazyme (agalsidase beta). The primary endpoint will be an improvement in eGFR (estimated Glomerular Filtration Rate), a measure of kidney function/damage.
- The company believes that no additional non-clinical studies are required to support a Biologics License Application (BLA). More details on the design of the trials will be provided after the study protocols have been approved.
- Both trials should commence in early 2016.
- PRX-102 is chemically modified version of a recombinant therapeutic enzyme called alpha-Galactosidase-A protein.
Nov. 9, 2015, 5:40 PM
- Protalix BioTherapeutics (NYSEMKT:PLX): Q3 EPS of -$0.04 beats by $0.04.
- Revenue of $4.3M (+79.2% Y/Y) misses by $0.7M.
Oct. 19, 2015, 10:46 AM
- 12-month data from a Phase 1/2 clinical trial assessing Protalix BioTherapeutics' (PLX) PRX-102 in patients with Fabry disease showed positive responses in two key metrics. The three-arm dose-ranging study evaluated 0.2 mg/kg, 1 mg/kg and 2 mg/kg infusions of PRX-102 administered every two weeks for one year in 18 treatment-naive subjects.
- Patients receiving the lowest dose of 0.2 mg/kg showed stable kidney function as measured by estimated Glomerular filtration rate (eGFR). Fabry sufferers are characterized by a deterioration in eGFR over time.
- Patients in the 0.2 mg/kg arm also showed continuous and durable reductions in lyso-Gb3 levels of up to almost 62%. Lyso-Gb3, a biomarker, dramatically increases and accumulates in the plasma of Fabry patients. In addition, they showed a continuous reduction and durable improvement in Mainz Severity Score Index (MSSI), a tool used to evaluate Fabry disease status.
- The company has scheduled an end-of-Phase 2 meeting with the FDA for November. A Phase 3 study is on tap for early 2016.
- PRX-102 is chemically modified version of a recombinant therapeutic enzyme called alpha-Galactosidase-A protein.
Oct. 13, 2015, 9:16 AM
Sep. 9, 2015, 9:17 AM
Aug. 10, 2015, 5:19 PM
- Protalix BioTherapeutics (NYSEMKT:PLX): Q2 EPS of -$0.05 beats by $0.03.
- Revenue of $3.8M (+57.0% Y/Y) misses by $2.6M.
May 10, 2015, 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.
May 7, 2015, 5:43 PM
- Protalix BioTherapeutics (NYSEMKT:PLX): Q1 EPS of -$0.06 beats by $0.02.
- Revenue of $4.4M (-34.3% Y/Y) misses by $3.8M.
Apr. 21, 2015, 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%)
Mar. 19, 2015, 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)
Protalix Biotherapeutics, Inc. engages in the development and commercialization of recombinant therapeutic proteins based on ProCellEx plant cell based expression system. It offers Taliglucerase alfa injections under the brand name Elelyso, an enzyme replacement therapy for Gaucher disease. Its... More
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