Supernus Announces Webcast of Investor Day on April 16, 2019

Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system diseases, today announced that it will host and webcast an Investor Day on Tuesday, April 16, 2019.

During the meeting, members of Supernus’ senior management team will provide a detailed discussion of the Company’s clinical programs, including SPN-810 for the treatment of Impulsive Aggression in ADHD and results of its Phase III clinical program of SPN-812 for the treatment of ADHD, and an assessment of their market opportunity. In addition, key thought leaders will share their perspectives on the current treatment paradigms, unmet medical needs, and the Company’s clinical development programs.

Presentation Date: Tuesday, April 16, 2019
Time: 8:00 a.m. ET (registration); 8:30 a.m. to 12:00 p.m. ET (presentation)
Place: Lotte New York Palace, New York City

To register to attend the event please contact Brandon.Weiner@Westwicke.com or (443) 450-4190.

A live webcast of the presentation can be accessed by visiting ‘Events & Presentations’ in the Investors section on the Company’s website at www.supernus.com. An archived replay of the webcast will be available on the Company’s website.

https://www.marketscreener.com/SUPERNUS-PHARMACEUTICALS-10523839/news/Supernus-Announces-Webcast-of-Investor-Day-on-April-16-2019-28332990/

Esperion Publishes Bempedoic Acid Study 3 Results

– Study 3 Demonstrated Bempedoic Acid Significantly Lowered LDL-Cholesterol and Reduced hsCRP in Patients Considered Statin Intolerant –– Over 24-Weeks, Bempedoic Acid Was Observed to be Safe, Well-Tolerated, and the Muscle-Related Adverse Event Rate Did Not Differ from Placebo –– Bempedoic Acid Is an Oral, Once-daily ATP Citrate Lyase (ACL) Inhibitor that Reduces Cholesterol and Fatty Acid Synthesis in the Liver –– Research by Ulrich Laufs, MD, PhD published in the Journal of the American Heart Association –

Esperion (NASDAQ:ESPR) announced today that results from the 345 patient, 24-week, Phase 3, double-blind, placebo-controlled study of bempedoic acid (CLEAR Serenity, also known as Study 3) were published today in the Journal of the American Heart Association (JAHA). Bempedoic acid is being developed as a complementary, cost-effective, convenient, once-daily, oral therapy for the treatment of patients with elevated low-density lipoprotein cholesterol (LDL-C).  Bempedoic acid and the bempedoic acid / ezetimibe combination tablet new drug applications have been submitted to the United States Food and Drug Administration, as well as are under regulatory review for marketing authorization by the European Medicines Agency.

Study 3 evaluated the efficacy, safety, and tolerability of bempedoic acid 180 mg versus placebo in 345 patients with hypercholesterolemia and considered statin intolerant (history of intolerance to at least two statins).

The JAHA publication highlights results from the primary efficacy endpoint of LDL-C lowering at 12-weeks and key secondary endpoints of safety and tolerability over 24-weeks, including that bempedoic acid (BA):

• significantly lowered LDL-cholesterol by 21 percent;
• significantly reduced high-sensitivity C-reactive protein (hsCRP), an important marker of the underlying inflammation associated with cardiovascular disease, by 25 percent;
• was observed to be safe and well-tolerated;
• did not induce muscle symptoms in statin intolerant patients compared with placebo (BA 12.8% vs placebo 16.2%);
• showed an adverse event rate of 64.1% compared with a placebo event rate of 56.8%, a serious adverse events rate of 6.0% compared with a placebo event rate of 3.6%, with no serious adverse events considered related to study medication;
• showed numerically fewer new-onset or worsening of diabetes compared with the placebo group (BA 2.1% vs 4.5% placebo)

https://www.marketscreener.com/ESPERION-THERAPEUTICS-INC-13479951/news/Esperion-Announces-Publication-in-The-Journal-of-the-American-Heart-Association-of-Bempedoic-Acid-St-28332989/

Guardant Health says draft of LCD may expand Guardant360 Medicare coverage

On March 28, Guardant Health announced that Palmetto GBA, a Medicare Administrative Contractor, has posted a draft local coverage determination, LCD, that, if implemented, would expand Medicare coverage of the company’s Guardant360 assay from advanced non-small cell lung cancer, NSCLC, to over a dozen advanced solid tumor cancer types with guideline-recommended genomic targets.The draft LCD would apply to advanced cancer patients who are covered by Medicare for next-generation sequencing of tumor tissue, but have insufficient or unavailable tissue samples and are candidates for FDA-approved treatment that is recommended by the guidelines of the National Comprehensive Cancer Network as category 1 or 2A. These cases may comprise a significant percentage of advanced cancers, especially those that have spread to the deep viscera, bone, or brain. Palmetto GBA is expected to finalize and implement the draft LCD after soliciting public comments. There can be no assurances that Palmetto GBA will finalize and implement the draft LCD.
https://thefly.com/landingPageNews.php?id=2887033

Alector, AbbVie Initiate Phase 1 for Treatment of Patients with Alzheimer’s

Alector, Inc. (Nasdaq:ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, a novel therapeutic approach for the treatment of neurodegeneration, today announced the initiation of the first-in-human Phase 1 trial for AL003, called the INTERCEPT study. The study is initially investigating the safety profile, pharmacodynamics and target engagement of AL003 in healthy volunteers. AL003 is the company’s second product candidate being developed for the treatment of patients with Alzheimer’s disease in collaboration with its partner AbbVie.

Alector is focused on identifying and functionally repairing genetic mutations that cause dysfunction of the brain immune system leading to neurodegeneration. Genetic mutations in the SIGLEC 3 receptor, which is expressed on the brain’s microglia cells, were shown to increase the risk of developing Alzheimer’s disease by inhibiting the beneficial activity of the brain’s immune system.

“We designed AL003 to functionally counteract the SIGLEC 3 risk allele,” said Arnon Rosenthal, Ph.D., co-founder and chief executive officer of Alector. “AL003, which blocks the function of SIGLEC 3, an inhibitory receptor on the microglia, conceptually acts in a similar manner to PD1 inhibitors in oncology, which block the function of an inhibitory receptor on T cells. Blocking SIGLEC 3 will enable a more robust brain immune system that can better address multiple disease-causing pathologies. and we expect this will lead to slowing or stopping of disease progression in patients with Alzheimer’s disease.”

https://www.nasdaq.com/press-release/alector-initiates-phase-1-trial-of-al003-for-the-treatment-of-patients–with-alzheimers-disease-20190401-01196

Aveo, Biodesix report ‘positive’ results from ficlatuzumab-cytarabine trial

AVEO Oncology and Biodesix, Inc. announced results from an investigator-sponsored Phase Ib expansion cohort of ficlatuzumab, AVEO’s potent hepatocyte growth factor inhibitory antibody product candidate, in combination with cytarabine in patients with relapsed and refractory acute myeloid leukemia. The results were presented in a poster session at the American Association for Cancer Research 2019 Annual Meeting. The presentation, titled, “CyFi: Results from a phase Ib expansion cohort of ficlatuzumab combined with high-dose cytarabine in patients with high risk relapsed or refractory acute myeloid leukemia” is available in the Publications & Presentations section of AVEO’s website. Elevated serum HGF level is an adverse prognostic factor associated with worse survival in AML and other cancers. Pre-clinical models have shown that myeloid blasts produce HGF and that blocking the HGF/c-Met pathway sensitizes blasts to cell death. The Phase Ib trial, which was funded by Gateway for Cancer Research and is being conducted at the UCSF Medical Center under the direction of Charalambos Andreadis, M.D., Associate Professor of Clinical Medicine, Director, Clinical Research Support Office, UCSF Helen Diller Family Comprehensive Cancer Center, was designed to assess the safety, tolerability and preliminary efficacy of ficlatuzumab with cytarabine in AML patients who are refractory to first line therapy or have relapsed within one year of induction, a population known to have poor outcomes. The maximally tolerated dose was 20 mg/kg of ficlatuzumab on day 1 followed by 2 g/m2 cytarabine daily on days 2-7. Of 12 patients who received ficlatuzumab and cytarabine at the maximally tolerated dose, one of whom was non-evaluable, 6 achieved a complete response. Of 18 patients enrolled in the study, 17 were evaluable and 9 achieved a CR. The most frequent grade 3/4 treatment emergent adverse events observed were febrile neutropenia, LFT abnormalities, and electrolyte disturbance. There was one death from sepsis and multi-organ failure that was determined to be disease related, and one patient withdrew from the study due to grade 4 gastrointestinal bleed, determined to be likely ficlatuzumab related. scRNA sequencing identified a TNF alpha and IFN gamma inflammatory signature that correlated with response to ficlatuzumab at count recovery.
https://thefly.com/landingPageNews.php?id=2886987

Apyx pulls application for premarket notification 510k submission to FDA

Apyx Medical Corporation announced it has voluntarily withdrawn its application for premarket notification 510k regulatory clearance of J-Plasma/Renuvion for use in dermal resurfacing procedures. The company will continue to work with the U.S. Food and Drug Administration relative to the development of a new 510k submission. At the present time, the company cannot provide a timeline for resubmission but intends to do so after further discussions with the Agency.
https://thefly.com/landingPageNews.php?id=2886991

RA Pharma: Phase 2 zilucoplan trial data selected for presentation

Ra Pharmaceuticals, Inc. announced that data from its Phase 2 clinical trial of zilucoplan for the treatment of generalized myasthenia gravis have been selected for an Emerging Science dual oral and poster presentation at the 2019 American Academy of Neurology Annual Meeting in Philadelphia, PA, from May 4 to 10, 2019. The presentation will feature data from the Phase 2, randomized, double-blind, placebo-controlled clinical trial, as well as data from the open-label, long-term extension study.

https://thefly.com/landingPageNews.php?id=2886994