Mon, Jun. 15, 6:52 PM
- Relatively recent IPO Avalanche Biotechnologies (NASDAQ:AAVL) craters 40% after hours on robust volume in response to its announcement of top-line results from a Phase 2a study assessing its lead product candidate, AVA-101, in patients with wet age-related macular degeneration (wet AMD). The primary endpoint of the trial was safety, but secondary efficacy measures have apparently disappointed investors despite the study not being powered to show statistical significance.
- The trial enrolled 32 wet AMD patients at least 55 years old. They were randomized to receive AVA-101 (n=21) or control (n=11). Both arms received two ranibizumab injections [Roche's (OTCQX:RHHBY) Lucentis] at day 0 and week 4. Almost all (n=29/32) had received prior anti-VEGF therapy with a median of 10 prior injections.
- The mean change in best corrected visual acuity (BCVA) from baseline in the AVA-101 cohort was +2.2 letters compared to -9.3 letters for control. In addition, 42.9% of patients (n=9/21) receiving AVA-101 improved or maintained stable vision with two or fewer rescue injections compared to 9.1% (n=1/11) for control. BCVA improvement of at least 10 letters with no more than two rescue injections occurred in 23.8% of the AVA-101 arm versus 0% in control.
- The modest average letter gain for the AVA-101 arm is substantially less than published studies of Eylea and Lucentis, which have demonstrated mean improvements in BCVA scores of 18.9 letters and 14.2 letters, respectively. If the difference persists in subsequent studies, then Regeneron will be less likely to take advantage of its right of first negotiation for the rights to AVA-101.
- The value proposition of AVA-101, a gene therapy that contains a gene that encodes a naturally occurring anti-VEGF protein, is a single subretinal injection compared intravitreal (inside the eye) injections as frequent as once per month for Regeneron's (NASDAQ:REGN) Eylea and Roche's Lucentis.
Wed, Jun. 10, 3:58 PM| Wed, Jun. 10, 3:58 PM | 7 Comments
Wed, Jun. 10, 10:25 AM
- Esperion Therapeutics (ESPR -24%) drops on a 3x surge in volume as investors apparently perceive more modest prospects for the company's cholesterol-lowering drug candidate, ETC-1002, after yesterday's positive Ad Comm vote for Regeneron (REGN -4.9%) and Sanofi's (SNY +0.7%) Praluent (alirocumab), which is now poised for approval sometime this summer.
- UBS analyst Andrew Peters remains undeterred. He maintains his Buy rating and $140 price target.
- Previously: Esperion cholesterol-lowering drug candidate successful in Phase 2 study (March 17)
Wed, Jun. 10, 8:41 AM
- Regeneron Pharmaceuticals (NASDAQ:REGN) slumps 4% premarket on light volume in response to yesterday's Ad Comm vote on its cholesterol-lowering med Praluent (alirocumab), co-developed with Sanofi (NYSE:SNY).
- The committee voted 13-3 in favor of approval, but only in combination with a statin or as monotherapy in patients who cannot tolerate statins. Ideally, the vote would have supported the use of alirocumab as unconditional monotherapy.
- SNY is up a fraction premarket on modest volume.
Tue, Jun. 9, 6:51 PM
- Regeneron (NASDAQ:REGN) has reopened for trading, up 1.1% to $532, after an FDA Ad Comm ruling in favor of its and Sanofi's Praluent (alirocumab) cholesterol-lowering drug.
- Shares had been halted since just before 7 a.m.
- The vote was 13-3. On Wednesday, the committee turns its attention to Amgen's (NASDAQ:AMGN) drug evolocumab.
- Sanofi estimates that 11M Americans might qualify for the drugs, which could run up to $10K/year in cost. Amgen estimates that 8M Americans might qualify.
Tue, Jun. 9, 5:10 PM
- The FDA's Endocrinologic and Metabolic Drugs Advisory Committee votes 13-3 recommending approval of Praluent (NYSE:SNY) (NASDAQ:REGN) (alirocumab).
- Previously: Ad Comm approaches for Sanofi/Regeneron cholesterol-lowering med (June 5)
- Previously: Regeneron's alirocumab shows durable effect in lowering "bad" cholesterol in high risk patients (March 16)
Tue, Jun. 9, 7:20 AM
Fri, Jun. 5, 11:02 AM
- The FDA's Endocrinologic and Metabolic Drugs Advisory Committee meets Tuesday, June 9 to discuss the Biologics License Application (BLA) submitted by Sanofi (SNY -2.7%) seeing approval for Praluent (alirocumab) for the treatment of adult patients with high cholesterol (hypercholesterolemia) or mixed dyslipidemia to reduce low-density lipoprotein cholesterol (LDL-C) as monotherapy or in combination with a statin. Sanofi and Regeneron Pharmaceuticals (REGN +1.9%) co-developed Praluent.
- FDA Briefing doc
- Sanofi/Regeneron briefing doc
- Addendum to Sanofi/Regeneron briefing doc
- The committee will review Amgen's (AMGN +0.1%) BLA for Repatha on Wednesday, June 10 (briefing docs not yet posted).
Fri, Jun. 5, 9:23 AM
- Amgen (NASDAQ:AMGN) announces the completion of enrollment of ~27,500 patients in the Phase 3 study, called FOURIER, assessing whether treatment with Repatha (evolocumab) reduces the risk of cardiovascular (CV) events in patients with high cholesterol and clinically relevant CV disease. The trial will compare Repatha in combination with statin therapy to placebo plus statin therapy. Results are expected no later than 2017.
- Evolocumab is an investigational fully human monoclonal antibody that inhibits convertase subtilisin/kexin type 9 (PCSK9), a protein that reduces the liver's ability to remove low-density lipoprotein cholesterol (LDL-C) from the blood.
- PCSK9 inhibitors are posed to dominate the post-statin cholesterol-lowering market. Amgen is neck-and-neck with Regeneron (NASDAQ:REGN) and Sanofi's (NYSE:SNY) Praluent (alirocumab). Europe's CHMP recommended Repatha for approval several weeks ago. A final decision by the European Commission usually takes ~60 days. The EMA accepted the MAA for Praluent in January 2015. An FDA advisory committee will meet on June 9 to discuss Praluent's BLA and June 10 to discuss Rapatha's. The PDUFA date for Repatha is August 27 while Praluent's is July 24.
Tue, May 26, 9:21 AM
- The FDA approves privately-held Boehringer Ingelheim's once-daily Stiolto Respimat (tiotropium bromide and olodaterol) Inhalation Spray for the maintenance treatment of chronic obstructive pulmonary disease (COPD), including chronic bronchitis and/or emphysema. It is not cleared for the treatment of asthma or acute deterioration of COPD.
- Tiotropium, a long-acting anticholinergic, is the active ingredient in Spiriva, the most prescribed COPD maintenance treatment worldwide. Olodaterol, a long-acting beta2-agonist that complements the efficacy of Spiriva, has a fast onset of action that improves airflow in five minutes after dosing.
- COPD-related tickers: (NASDAQ:TBPH) (NYSE:GSK) (NASDAQ:THRX) (NYSE:AZN) (NYSE:NVS) (NASDAQ:REGN) (NYSE:SNY) (NYSE:TEVA) (OTCQX:RHHBY) (NASDAQ:AQXP)
Thu, May 21, 9:30 AM
- Alongside Goldman's list of 50 stocks appearing most as top holdings at hedge funds is its list of the 50 top shorts.
- New additions this quarter: Baxter Intl (NYSE:BAX), UPS, Marriott (NASDAQ:MAR), NextEra (NYSE:NEE), Ford (NYSE:F), National Oilwell Varco (NYSE:NOV), McDonald's (NYSE:MCD), M&T Bank (NYSE:MTB), CenturyLink (NYSE:CTL), Amgen (NASDAQ:AMGN), Pioneer Natural (NYSE:PXD), Duke Energy (NYSE:DUK), Seagate (NASDAQ:STX), AbbVie (NYSE:ABBV), Cisco (NASDAQ:CSCO).
- The full list (in order of $ value of short interest): AT&T (NYSE:T), Disney (NYSE:DIS), IBM, Verizon (NYSE:VZ), Intel (NASDAQ:INTC), Kinder Morgan (NYSE:KMI), Exxon (NYSE:XOM), Pfizer (NYSE:PFE), J&J (NYSE:JNJ), Deere (NYSE:DE), Caterpillar (NYSE:CAT), Exelon (NYSE:EXC), GE, Boeing (NYSE:BA), Halliburton (NYSE:HAL), Fox (NASDAQ:FOXA), Comcast (NASDAQ:CMCSA), UTX, Regeneron (NASDAQ:REGN), Merck (NYSE:MRK), salesforce.com (NYSE:CRM), AbbVie (ABBV), Conoco (NYSE:COP), Wal-Mart (NYSE:WMT), Eli Lilly (NYSE:LLY), Celgene (NASDAQ:CELG), Schlumberger (NYSE:SLB), AutoZone (NYSE:AZO), Wells Fargo (NYSE:WFC), Emerson (NYSE:EMR), McDonald's (MCD), Reynolds (NYSE:RAI), Target (NYSE:TGT), Accenture (NYSE:ACN), Coca-Cola (NYSE:KO).
Thu, May 21, 7:51 AM
- A Phase 3 clinical trial, called SARIL-RA-TARGET, evaluating the investigational fully human IL-6 receptor antibody sarilumab in patients with rheumatoid arthritis (RA) who were inadequate responders to or intolerant of TNF-alpha inhibitors (TNF-IR) achieved its co-primary endpoints. The study compared two subcutaneous doses of sarilumab versus placebo, added to non-biologic disease-modifying anti-rheumatic (DMARD) therapy (e.g., methotrexate) in 546 patients.
- Subjects were randomized in three treatments groups that self-administered either sarilumab 200 mg, 150 mg or placebo every other week, in addition to DMARD therapy.
- The co-primary endpoints were: 1) proportion of patients with improvement in signs and symptoms of RA measured by ACR20 score (American College of Rheumatology score of 20% improvement in symptoms) at week 24 and 2) improvement in physical function measured by change from baseline in the Health Assessment Question-Disability Index at week 12.
- The results in the three treatment arms (200 mg, 150 mg, placebo) relative to the first endpoint were 61%, 56% and 34%, respectively. The specific results for the second endpoint are not reported but both test arms showed clinically relevant and statistically significant improvements compared to placebo (p>0.001).
- Detailed results from the study, along with results from two other trials, SARIL-RA-EASY and SARIL-RA-ASCERTAIN, will be presented at future medical conferences.
- Sarilumab, developed with Regeneron Pharmaceuticals' (NASDAQ:REGN) VelocImmune antibody technology, binds to IL-6, a protein that plays a key role in inflammation. It is being co-developed with Sanofi (NYSE:SNY) under their collaboration that began in 2007.
Wed, May 20, 1:14 PM
- Thinly traded nano cap Lpath (LPTN -79.5%) craters on a massive 78x surge in volume in response to its announcement that its lead product candidate, iSONEP, failed to achieve its primary and key secondary efficacy endpoints in a Phase 2 trial in patients with wet age-related macular degeneration (wet AMD) who had not responded adequately to anti-vascular endothelial growth factor (VEGF) therapies such as Roche's (OTCQX:RHHBY -0.4%) Lucentis (ranibuzumab) or Avastin (bevacizumab) or Regeneron's (REGN +1.4%) Eylea (aflibercept).
- Patients treated with iSONEP, either alone or as an adjunct, did not show any statistically significant improvement in visual acuity.
- SVP and Chief Development Officer Dario Paggiarino, M.D., says, "This trial was designed to evaluate the activity of iSONEP in wet AMD patients that had previously received at least three prior injections of an anti-VEGF agent and had not responded well. While the primary endpoint of the trial was not met, we will be conducting a complete analysis of the data, including additional anatomical endpoints, to better understand the results from each arm of the trial."
- iSONEP is a monoclonal antibody that binds to a bioactive lipid called sphingosine-1-phosphate, a major regulator of vascular systems.
- Final results will be presented in November at the American Academy of Ophthalmology meeting in Las Vegas, NV.
- Related ticker: (PFE +0.4%)
Mon, May 11, 5:22 PM
- Ophthotech (NASDAQ:OPHT) completes enrollment in its first Phase 3 trial assessing Fovista (anti-PDGF BB pegylated aptamer) in combination with Roche's (OTCQX:RHHBY) Lucentis (ranibizumab) compared to Lucentis alone as monotherapy in patients with wet age-related macular degeneration (wet-AMD).
- Patient enrollment in a second Phase 3 study assessing Fovista + Lucentis compared to Lucentis alone should be completed by the end of Q3. Data readout from the first two studies should happen in 2016.
- A third Phase 3 study evaluating Fovista in combination with Regeneron's (NASDAQ:REGN) Eylea (aflibercept) or Roche's Avastin (bevacizumab) compared to Eylea or Avastin alone should reach its enrollment target in the same time frame as the other two studies, although the expected time from today is unclear.
- Total enrollment in the three trials will be ~1,866 across 225 sites worldwide.
- The company's objective with Fovista is to be agnostic relative to the choice of anti-VEGF agent co-administered with it.
Thu, May 7, 8:04 AM
- Regeneron Pharmaceuticals (NASDAQ:REGN) Q1 results ($M): Revenues: 869.6 (+39.0%); Net Product Sales: 544.6 (+50.3%); Sanofi Collaboration Revenue: 173.4 (+28.4%); Bayer Healthcare Collaboration Revenue: 123.8 (-1.2%); Technology Licensing/Other: 27.8 (+270.7%); Operating Income: 283.6 (+48.0%); Net Income: 76.0 (+11.3%); EPS: 0.66 (+8.2%); Quick Assets: 1,225.5 (-10.0%).
- Eylea U.S. sales: 541 (+50.7%); ex-U.S. sales: 292 (+33.9%).
- 2015 Guidance: Eylea U.S. net product sales growth: 30 - 35% from 25 - 30%.
Thu, May 7, 6:31 AM
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