Yesterday, 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 ($1M) in Germany. It will be sold for a one-time payment because it is too difficult to measure how well it works (this reasoning will undoubtedly pop up again).
- Previously: UniQure sets price record with Glybera (Nov. 26, 2014)
- Related tickers: (CELG +1.3%)(AMGN -0.2%)(BIIB +0.5%)(GILD +0.1%)(MRK -0.3%)(GSK -0.2%)(AZN +0.9%)(LLY +0.3%)(NVS +0.1%)(PFE -0.5%)(JNJ -0.3%)(OTCQX:RHHBY +1.4%)(BMY -0.4%)(ABT)(BAX -1.1%)
Mon, Feb. 9, 12:21 PM
- The following are healthcare companies whose after-tax margins have expanded the past three quarters and trade at least 500K shares per day:
- (ACOR -0.7%)(BCR -1.5%)(BIIB -0.2%)(CAH -1.2%)(CPHD +1.3%)(EVHC -1.6%)(ILMN +0.7%)(JAZZ -1.7%)(LCI +7.3%)(MNK -0.5%)(SHPG -2.9%)(SYK -0.2%)(TMO -0.7%)(UTHR +1.4%)(VRX).
- This is not a list of "Buy" recommendations, but rather a targeted list of firms that may be suitable for more in-depth research.
Fri, Jan. 30, 12:31 PM
- Opko Health (OPK +3.5%) submits an Investigational New Drug Application (IND) to the FDA for clearance to commence a Phase 2a study evaluating its long-acting Orphan Drug-designated coagulation Factor VIIa (Factor VIIa-CTP) for the treatment of bleeding episodes in patients with hemophilia A or B with inhibitors to Factor VII or Factor IX.
- Currently, Factor VIIa therapy is administered intravenously which requires multiple infusions, due to its short half-life, to treat a bleeding episode.
- The longer duration of action of Opko's Factor VIIa-CTP is based on the naturally occurring peptide, C-terminal peptide, of the beta chain of human chorionic gonadotropin (hCG).
- Opko acquired the rights to the product when it bought Prolor Biotech.
- Previously: Opko completes its acquisition of Prolor Biotech (Aug. 29, 2013)
- Related tickers: (BAX +0.4%)(SGMO +0.6%)(ALNY +0.5%)(BIIB +11.4%)(OTCPK:BAYRY -1.4%)
Fri, Jan. 30, 9:12 AM| 4 Comments
Thu, Jan. 29, 4:32 PM
Thu, Jan. 29, 4:30 PM
- Biogen Idec (NASDAQ:BIIB) Q4 results ($M): Total Revenues: 2.640.7 (+34.3%); Net Product Sales: 2,287.0 (+42.3%); Joint Business: 304.5 (+13.0%); Royalty: 31.4 (-48.2%); Corporate Partner: 17.8 (-38.0%).
- Key Product Sales: Avonex: 736.0 (-2.1%); Tysabri: 483.9 (+13.4%); Tecfidera: 916.0 (+81.1%); Fampyra: 18.5 (+6.9%); Fumaderm: 14.4 (+2.1%); Plegridy: 41.1; Alprolix: 40.3; Eloctate: 36.8.
- COGS: 297.3 (+15.0%); R&D Expense: 500.1 (+18.5%); SG&A Expense: 573.6 (+9.7%); Net Income: 883.5 (+93.2%); EPS: 3.74 (+94.8%); Quick Assets: 1,845.4 (+50.9%).
- Gross Profit: 2,343.4 (+37.3%); COGS: 11.3% (-14.4%); Gross Margin: 88.7% (+2.2%); Non-GAAP Operating Earnings: 1,269.7 (+66.6%); Operating Earnings Yield: 48.1% (+24.0%); Net Earnings Yield: 33.5% (+43.8%).
- Net income growth the past four quarters: 12.5%, 47.4%, 75.7%, 93.2%.
- 2015 Guidance: Revenue growth: 14 - 16%; Non-GAAP EPS: $16.60 - 17.00; EPS: $15.45 - 15.85.
Wed, Jan. 28, 5:35 PM
- ABAX, ALGN, AMZN, AVNW, BCR, BIIB, BRCM, BXP, CB, COHR, CORT, CPHD, CPSI, CTCT, DECK, EFII, ELX, ELY, EMN, EPAY, FICO, GDOT, GIMO, GOOG, HA, HBI, HLIT, INVN, ISBC, IXYS, JDSU, LEG, MCHP, MITK, MTW, N, NATI, NEU, NFG, NGVC, PCCC, PFG, PFPT, PKI, PMCS, QLGC, RHI, RVBD, SFG, SWI, SYNA, TFSL, TUES, UIS, V, VR, WERN
Mon, Jan. 26, 5:57 PM
Wed, Jan. 14, 12:20 PM
- Isis Pharmaceuticals (ISIS -1.1%) earns a $7M milestone payment from Biogen Idec (BIIB -0.6%) related to the advancement of the ongoing open-label extension study of ISIS-SMNrx in children with spinal muscular atrophy.
- Patients in this stage have completed the dosing in previous trials. They are receiving a 12 mg dose of ISIS-SMNrx every six months for the duration of the study.
Wed, Jan. 14, 9:13 AM
- Express Scripts' (NASDAQ:ESRX) success in containing the prices of new HCV therapies by pitting Gilead Sciences (NASDAQ:GILD) against AbbVie (NYSE:ABBV) is sending shock waves through the biotech/pharma industry. Most of the big players slumped yesterday as investors fear that ESRX's tactics will spread to other high cost areas like cancer and cholesterol.
- At JPM15 yesterday, CEO George Paz said that the new cholesterol-lowering post-statin PCSK9 inhibitors will be the next big opportunity to pit drug firms against each other in order to drive costs down. FDA clearance of the first two therapies, one from Amgen (NASDAQ:AMGN) and the other from Regeneron Pharmaceuticals (NASDAQ:REGN) (NYSE:SNY), is expected to occur at about the same time.
- The prices of emerging cancer therapies will also be in payers' crosshairs. Amgen's Blincyto, for example, costs $178,000 per standard course of treatment while Merck's (NYSE:MRK) Keytruda is close behind at $150,000.
- Related tickers: (NYSE:AZN) (NYSE:GSK) (NYSE:LLY) (ABBV) (NYSE:PFE) (NASDAQ:BIIB) (NYSE:ABT) (NASDAQ:CELG) (NYSE:BMY) (OTCQX:RHHBY) (NYSE:JNJ) (NYSE:NVS) (OTCPK:BAYRY)
Sun, Jan. 11, 10:14 PM
- In the context of accelerating the growth of its pain portfolio, Biogen (NASDAQ:BIIB) acquires Cambridge-based Convergence Pharmaceuticals, a clinical-stage biotech whose technology platform is based on ion channel electrophysiology and ion channel medicinal chemistry. Biogen is particularly interested in CNV1014802, a small molecule state-dependent sodium channel blocker, currently being developed for the treatment of neuropathies like lumbosacral radiculopathy (sciatica) and trigeminal neuralgia.
- Under the terms of the acquisition agreement, Biogen will pay Convergence shareholders an upfront payment of $200M and up to $475M in milestones. The transaction is expected to close this quarter.
Thu, Jan. 8, 11:10 AM
- A Phase 2 trial assessing Biogen Idec's (BIIB -1.9%) monoclonal antibody anti-LINGO-1 for the treatment of acute optic neuritis (AON) achieved its primary endpoint of recovery of optic nerve latency compared to placebo but showed no effect on the secondary endpoints of change in thickness of the retinal layers, visual function and low contrast letter acuity.
- The trial was designed to study the ability of ant-LINGO-1 to enable repair of an optic nerve lesion via axonal remyelination following the onset of a first episode of AON. The condition is considered a good clinical model to assess the hypothesized mechanisms of action of anti-LINGO-1, remyelination and neuroprotection.
- The study measured the effects of remyelination by measuring the latency of nerve conduction between the retina and the brain's visual cortex using full field evoked potential (FF-VEP) at Week 24. Anti-LINGO-1 demonstrated a 34% improvement compared to placebo. The results were statistically significant (p=0.0504) by a nose.
Dec. 23, 2014, 4:21 PM
Dec. 2, 2014, 12:26 PM
- Traders jumping into Biogen (BIIB +5.8%) this morning after the company's announcement of preliminary results for BIIB037 will have to wait a while to see if Phase 3 results demonstrate efficacy in Alzheimer's disease. Top-line data will not be available until 2018 - 2019 according to Ben Levishon.
- Bulls are already prognosticating that BIIB037 is a $5B - 10B opportunity and may add as much as $21 to the company's share price. Bears are skeptical in light of the consistent failures of plaque inhibitors to prove long-term improvements in cognition.
- Previously: Biogen up on Alzheimer's results
Dec. 2, 2014, 11:32 AM
- Biogen (BIIB +5.3%) jumps on double normal volume in response to its disclosure of interim data at an investors conference from an early-stage study of BIIB037 that demonstrated encouraging results. BIIB037 is designed to clear amyloid plaques from the brain. The study results indicated that it reduced amyloid levels in the brain in both a dose- and time-dependent way. It also demonstrated a positive effect on cognition after 54 weeks.
- The company says it will move aggressively into later-stage trials.
- Alzheimer's disease has been a tough nut to crack for drug makers. Pfizer, J&J and Eli Lilly have all experienced clinical trial failures, for example. Biogen did not issue a press release on its results because it was primarily a safety study.
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