April 29, 2011
– Comments (6)
It might be a good time to buy a few REGN shares. REGN shares are currently one of the larger positions of my "fund" (see here). There are currently 510 REGN shares in the fund with break-even of around $51.67. REGN is at around $52.00 now.
a recent "pitch" by zzlangerhans.
I'm trying something new here. Normally I don't care to green thumb stocks that have doubled in the last few months. I just figure I missed the boat and I move on to companies that haven't broken out yet. But in the case of Regeneron, there appears to be room to run.I've gone red on Regeneron before, figuring that the market cap had outstripped potential, and I was lucky to get away with it. The company has been on a major hot streak with clinical trials and has been progressing rapidly to the BLA stage. VEGF-Trap Eye is looking really good for first pass approval for wet AMD after strong results from the VIEW1 and VIEW2 phase III trials last November. The PDUFA is August 20. VEGF-Trap Eye has also shown positive results in the COPERNICUS trial for central retinal vein occlusion and results of the similar GALILEO trial are expected later in H1 2011. Thus far, the company has been very consistent in providing positive results from the second of paired trials after the first has been positive. VEGF-Trap Eye has also begun the VIVID-DME phase III trial in diabetic macular edema, another potential billion dollar indication, as well as a phase III trial in Asia for choroidal neovascularization of the retina.In February Regeneron's other compound in clinical development, rilonacept, showed positive results in the PRE-SURGE2 confirmatory trial in prophylaxis of gout attacks. With two phase III trials showing 72% and 80% reduction in gout attacks, a BLA for rilonacept may be submitted as early as mid-2011 with the resultant potential for two billion-dollar drugs on the market by 2012.A potential fly in the ointment is the performance of VEGF-Trap as an anti-neoplastic agent. The VANILLA trial in pancreatic cancer and the VITAL trial in NSCLC were failures. Topline data from VELOUR in colorectal cancer and interim data from VENICE in prostate cancer should be released by mid-2011. There is always a chance that the company may take advantage of their rising share price to raise cash, although with 627M in liquid assets after October's dilutive financing there is no obvious need for this.It still isn't my style to jump on the bandwagon of a rising star like this. On the other hand, if the failure I expect from the remaining cancer trials of VEGF-TRAP comes before the PDUFA for wet AMD and the share price reacts disproportionately, I may look into a long investment or calls.
some recent "recommendations".
XOMAINSM(D)BCRX DYAXRIGLALIMALTHNKTRDSCOLGNDSVNTMNTA MorphoSysZIOPALNYEXASIDRATKMR
Ranibizumab and Bevacizumab for Neovascular Age-Related Macular Degeneration
Bevacizumab versus Ranibizumab — The Verdict
Roche Holding’s Avastin Treatment Effective for Blindness in Study
Have you found any estimate on the cost of VEGF Trap-Eye treatments? I haven't been able to track it down...
An interesting sidebar is PDLI...they receive royalty payments from Avastin and Lucentis. It appears highly probable that net payments will decrease going forward with the Avastin news. This might impact the "margin of safety" in the investment. : )
Have you found any estimate on the cost of VEGF Trap-Eye treatments?
Discovery Labs' KL4 Surfactant Aerosolization Program Data to be Presented at the Pediatric Academic Societies Annual Meeting
The presentations at the upcoming PAS congress, internationally recognized as the most relevant medical meeting dedicated to pediatric research, are as follows:
Aerosolized KL4 Surfactant Dose-Response in the Spontaneously Breathing CPAP-Supported Preterm Lamb; Wolfson, et al.
Novel Ventilator Circuit Adaptor for Combined Delivery of CPAP and Aerosolized Drugs to Premature Infants; Mazela, et al.
Aerosolized KL4 Surfactant Improves Gas Exchange and Survival in Spontaneously Breathing Piglets With HCl Induced Acute Lung Injury; Lampland, et al.
Aerosolized KL4 Surfactant Dose-Response in the Spontaneously Breathing CPAP-Supported Preterm LambMarla R. Wolfson, Jichuan Wu, Terrence L. Hubert, Jan Mazela, Timothy J. Gregory, Russell G. Clayton, Thomas H. Shaffer. Physiology, Pediatrics, and Medicine; Temple Lung Center, Temple Univ Sch Med, Phila., PA; Discovery Laboratories, Inc., Warrington, PA; Poznan University of Medical Sciences, Poznan, Poland; Nemours Lung Research Ct, A.I. duPont Hosp for Child, Wilmington, DE.BACKGROUND: Previously, we have successfully demonstrated a therapeutic effect of an aerosolized, peptide-containing, synthetic surfactant in spontaneously breathing CPAP-supported preterm lambs. As a next developmental step, dose-ranging studies are required to define the dose that produces the optimal physiologic and biomarker responses in this model as well as to guide clinical expectation and management.OBJECTIVE: To evaluate dose-responses to an aerosolized, peptide-containing, synthetic surfactant (KL4 surfactant), using lung mechanics, histomorphology and lung inflammation biomarkers in spontaneously breathing, CPAP-supported preterm lambs.DESIGN/METHODS: Following Cesarean section, lambs (n = 20; 130-132 days gestation) were anesthetized, instrumented, delivered, supported with 100% oxygen, CPAP, and caffeine then quasi-randomized to receive CPAP alone (controls) or CPAP plus aerosolized KL4 surfactant for up to 10, 20, 30 or 90 min using a novel capillary aerosol generator. Cardiopulmonary parameters were monitored for 4 hrs. Lung IL-8 and histomorphometry were measured.RESULTS: By 4 hrs. and in comparison to controls, lambs treated with CPAP plus aerosolized KL4 surfactant demonstrated a dose-dependent increase in compliance and decrease in lung IL-8; marked differences occurred with the 20 min dose and there were little further differences between the 20, 30 and 90 min. doses. Relative to controls, PaO2 was greater following the 10, 20 and 30 min. doses and trended towards control values with the 90 min. dose. Independent of the duration of aerosolized surfactant, gross inspection and lung histomorphometry demonstrated greater and more homogenous expansion compared to controls.CONCLUSIONS: Relative to treatment with CPAP alone, aerosolized KL4 surfactant improved gas exchange, pulmonary mechanics, lung structure integrity, and reduced lung inflammation in a dose-dependent manner in preterm lambs. These observations provide preliminary guidance for titrating dosing strategies designed to optimize peri-dosing functional responses and lung protective biomarker outcomes for the management of neonatal respiratory distress syndrome.(Discovery Laboratories, Inc; DURIP-ONR; NIH P20 RR 020173).Funded by: Discovery Laboratories, Inc.Session: Poster Session: Neonatal Pulmonology (10:00 AM - 2:00 PM)Date/Time: Tuesday, May 3, 2011 - 10:00 AM
Novel Ventilator Circuit Adaptor for Combined Delivery of CPAP and Aerosolized Drugs to Premature InfantsJan Mazela, Timothy J. Gregory, Christopher Henderson, Jan Wenstrup, Thomas H. Shaffer, Marla R. Wolfson. Discovery Laboratories, Inc., Warrington, PA; Neonatology, Poznan Univ of Med Sciences, Poznan, Poland; Nemours Lung Research Center, AI du Pont Hospital, Wilmington, DE; Physiology & Pediatrics, Temple Univ Sch of Med, Philadelphia, PA.BACKGROUND: Aerosolized drugs have been used for several decades. However, there is a paucity of data supporting efficacy of aerosols in the neonatal population treated with CPAP. Series of novel concentric, low dead space CPAP adaptors that connect the nebulizer/aerosol tube with the inspiratory/expiratory circuit arms and patient interface were developed. They direct undiluted aerosol to the patient and maintain CPAP throughout the breathing cycle.OBJECTIVE: To test novel CPAP adaptors designed to optimize delivery of aerosolized drugs to neonates.DESIGN/METHODS: To measure the effect of the adaptors on dilution, different O2 concentrations (100% O2 for aerosol flow path and 21% O2 for the CPAP flow path) and an O2 analyzer were used to determine the dilution factor. Adaptors were tested at different CPAP flows (4, 6, 8, 10, & 12 L/min), and different steady state with inspiratory flows (IF:∼1 & 3 L/min), CPAP at 5 cm H2O. Flow resistance was measured by manometry and pneumotachography to establish operational characteristic of the novel adaptors at two dynamic flow conditions (∼1 & 3 L/min).RESULTS: Of the three adaptors tested, CPAP adaptor 1 demonstrated the lowest dilution at the highest IF tested without increasing expiratory resistance.
CONCLUSIONS: A novel CPAP adaptor may reduce dilution of the aerosol by ventilator bias flow without increasing resistance above what is observed for a standard Y connector when combined with CPAP, thus improving inhaled drug delivery.
Session: Poster Session: Neonatal Pulmonology (10:00 AM - 2:00 PM)Date/Time: Tuesday, May 3, 2011 - 10:00 AM
Aerosolized KL4 Surfactant Improves Gas Exchange and Survival in Spontaneously Breathing Piglets with HCl Induced Acute Lung InjuryAndrea Lampland, Patricia Meyers, Marla Wolfson, Christopher Henderson, Timothy Gregory, Cathy Worwa, Brenda Plumm, Mark C Mammel. Pediatrics - Neonatology, University of Minnesota, Minneapolis, MN, United States; Infant Diagnostic & Research Center, Children's Hospitals and Clinics of Minnesota, St. Paul, MN, United States; Physiology, Temple University, Philadelphia, PA, United States; Discovery Laboratories, Inc., Warrington, PA, United States.
BACKGROUND: Surfactant therapy may be a useful treatment for acute lung injury (ALI) (Pediatric Pulmonol 2010;68:782). Use of aerosolized surfactant in ALI has not been investigated.OBJECTIVE: Evaluate aerosolized surfactant (KL4; lucinactant, Discovery Laboratories, Inc., Warrington, PA) in treating piglets with HCl-induced ALI.DESIGN/METHODS: ALI was induced in spontaneously breathing piglets with intratracheal 0.2N HCl until PaO2 was ≤ 350 torr at FIO2 1.0. Piglets were randomized to receive 5.8 ml/kg (175 mg/kg) of endotracheal KL4 (ET KL4) with extubation to CPAP; aerosolized KL4 (AERO KL4; 60 minutes; 22.5 mg/min) while on CPAP; or CPAP alone (CPAP/control). Piglets were monitored for 3 hours; blood gases obtained every 30 minutes. Lung tissue was analyzed for IL-8 and total protein by porcine-specific ELISA and Bradford with group differences analyzed by ANOVA. Physiologic data were analyzed using 2 way ANOVA with Tukey LSD post-hoc testing for p values <0.05.RESULTS: Both ET KL4 and AERO KL4 produced higher PaO2s (p<0.001) and improved survival (p<0.05) compared to CPAP. AERO KL4 was as effective as ET KL4, and produced the highest final PaO2 (p<0.05). IL-8/total protein ratios were lower in AERO KL4 versus CPAP (p<0.03).[table1][figure1]CONCLUSIONS: In spontaneously breathing piglets with ALI, AERO KL4 and ET KL4 resulted in better gas exchange and survival. AERO KL4 was as effective as ET KL4, and produced the highest final PaO2. AERO KL4 treated piglets had lower IL-8/total protein ratios than CPAP, suggesting less lung inflammation. This is the first successful use of aerosolized surfactant in an animal model of ALI.Session: Platform Session: Late Breaker Abstract Session: Neonatology (8:00 AM - 10:00 AM)Date/Time: Monday, May 2, 2011 - 9:00 AM
what DSCO is currently doing ...
The Company believes that a key remaining step to potentially gain FDA marketing approval for Surfaxin is to satisfy the FDA as to the final validation of its fetal rabbit biological activity test (“BAT”), an important quality control release and stability test for Surfaxin. The Company has been conducting a comprehensive preclinical program in this regard. The Company has previously optimized the BAT and its final validation is intended to satisfy the FDA with respect to the ability of the optimized BAT to adequately reflect the biological activity of Surfaxin throughout its shelf life and to discriminate biologically active from inactive Surfaxin drug product. The comprehensive preclinical program will also provide data that will be used to gain the FDA’s agreement on final acceptance criteria, with respect to biological activity as assessed by the BAT, for release and ongoing stability of Surfaxin drug product. The comprehensive preclinical program also calls for multiple Surfaxin batches to be used to demonstrate concordance between the BAT and the well-established preterm lamb model of RDS by performing a series of prospectively-designed, side-by-side preclinical studies (i.e., concordance studies). Data from the preterm lamb model and BAT concordance studies are intended to support final BAT validation and to demonstrate comparability of drug product used in the Phase 3 clinical program with Surfaxin drug product to be manufactured for commercial use.
I agree with you that some of these abstracts being presented are good signs. The major reason I've kept my shares of DSCO, besides stubborness, is that they've gotten govt. funding for their research and still are definitely producing studies. At least some people at DSCO are definitely working hard to get the foundation needed for approval.
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