Nasdaq GlobeNewswire

ProQR Presents in vivo Proof of Concept Data for the Axiomer® RNA Editing Platform Technology

Key updates

ProQR to Present at Two Investor Conferences in September

LEIDEN, the Netherlands, Sept. 21, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that the Company will present at two investor conferences during the month of September.

Oxygen Concentrators and Cylinders Market worth over $4.5 billion by 2024: Global Market Insights, Inc.

Ocean View, Delaware, Sept. 21, 2017 (GLOBE NEWSWIRE) --

BioCryst’s RAPIVAB® (peramivir injection) Receives FDA Approval for a Pediatric Indication


RESEARCH TRIANGLE PARK, N.C., Sept. 21, 2017 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) a biotechnology company focused on the development and commercialization of treatments for rare and infectious diseases, announced today that the U.S. Food and Drug Administration (FDA) has approved a supplemental New Drug Application for RAPIVAB (peramivir injection), an intravenous (i.v.) neuraminidase inhibitor, extending its availability for the treatment of acute uncomplicated influenza to pediatric patients 2 years and older who have been symptomatic for no more than two days. The pediatric approval was based on the interim analysis of an ongoing pediatric clinical study. Those results will be presented at the upcoming ID Week 2017 meeting in San Diego.

Amarin to Present at Cantor Global Healthcare Conference

BEDMINSTER, N.J., and DUBLIN, Ireland, Sept. 20, 2017 (GLOBE NEWSWIRE) -- Amarin Corporation plc (NASDAQ:AMRN), a biopharmaceutical company focused on the commercialization and development of therapeutics to improve cardiovascular health, announced today that John F. Thero, Amarin's president and chief executive officer, is scheduled to present a general company update at the Cantor Global Healthcare Conference in New York City on Tuesday, September 26, 2017, at 11:30 a.m. EST.

Arbutus’ LNP Licensee Alnylam Announces Positive Phase 3 Results for LNP-Enabled Patisiran Program

Arbutus’ LNP Technology Further Validated with New Results

Achillion to Present at the 2017 Leerink Partners Rare Disease Roundtable Series

NEW HAVEN, Conn., Sept. 20, 2017 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) announced today that members of the senior management team will provide a corporate update at the 2017 Leerink Partners Rare Disease Roundtable Series on Wednesday, September 27, 2017 at 10:00 a.m. ET at the Lotte New York Palace Hotel in New York City, NY.

Iovance Biotherapeutics, Inc. Announces Public Offering of Common Stock


SAN CARLOS, Calif., Sept. 19, 2017 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ:IOVA), a biotechnology company developing novel cancer immunotherapies based on tumor-infiltrating lymphocyte (TIL) technology, today announced that it intends to offer and sell $50 million of its common stock, subject to market and other conditions, in an underwritten public offering. All of the shares in the offering are to be sold by Iovance. Iovance intends to grant the underwriters a 30-day option to purchase up to $7.5 million of common stock at the public offering price, less the underwriting discounts and commissions. Iovance intends to use the proceeds from this offering to fund its ongoing clinical trials for its current product candidates, including its ongoing Phase 2 clinical trials of LN-144, TIL for treatment of metastatic melanoma, and LN-145, TIL for the treatment of cervical and head and neck cancers to fund activities related to commercial scale-up of the Company’s third party manufacturing operations and for working capital and other general corporate purposes.

Novavax to Present at the Ladenburg Thalmann 2017 Healthcare Conference

GAITHERSBURG, Md., Sept. 19, 2017 (GLOBE NEWSWIRE) -- Novavax, Inc., (Nasdaq:NVAX) today announced the Company will present at the Ladenburg Thalmann 2017 Healthcare Conference.

CEO Robert Clarke Discusses Positive Developments from Partnerships Obtained within the Past Few Months


Lexington, MA.,, Sept. 19, 2017 (GLOBE NEWSWIRE) -- Dr. Robert Clarke, CEO and President of Pulmatrix, Inc. (Nasdaq:PULM), interviews on the Uptick Network Stock Day Podcast with host Everett Jolly. During the first half of the interview, Dr. Clarke discusses the partnership with Vecturato develop Pulmatrix’s drug candidate, PUR0200, for chronic obstructive pulmonary disease (COPD) for the U.S. market and highlights that Vectura and/or its partners will be responsible for all future development costs for the U.S. With a deal structure that includes a technology transfer fee and mid-teen percent revenue share Dr. Clarke explains the program’s trial phases, that both companies benefit from the partnership around a multi-billion dollar US product and future potential combinations, and when the program is expected to finalize in the future.

Novavax Initiates Phase 1/2 Trial of NanoFlu™ Vaccine in Older Adults

  • Recently published NanoFlu™ preclinical data provide strong rationale for Phase 1/2 clinical trial based on robust antibody titers and improved protective responses against both current and drifted strains

  • Data from trial expected by the end of 2017

Cytokinetics Announces Dosing of First Patient in Japan in GALACTIC-HF, Phase 3 Clinical Trial of Omecamtiv Mecarbil in Patients With Heart Failure


SOUTH SAN FRANCISCO, Calif., Sept. 19, 2017 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq:CYTK) today announced that the first patient has been dosed in Japan in GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure), the Phase 3 cardiovascular outcomes clinical trial of omecamtiv mecarbil which is being conducted by Amgen, in collaboration with Cytokinetics.  Coincident with patient dosing, Amgen will make a $10 million milestone payment to Cytokinetics. Omecamtiv mecarbil, a novel investigational cardiac myosin activator that increases cardiac contractility, is being developed by Amgen in collaboration with Cytokinetics for the potential treatment of heart failure.

Madrigal Pharmaceuticals Announces Positive Outcome from Pre-Planned DSMB Safety Review and Completion of Enrollment of Phase 2 Study of MGL-3196 in Patients with Heterozygous Familial Hypercholesterolemia

-- Second DSMB review includes data from both Phase 2 studies in NASH and HeFH --

Akari Therapeutics to Present at the Cantor Fitzgerald Global Healthcare Conference

NEW YORK and LONDON, Sept. 19, 2017 (GLOBE NEWSWIRE) -- Akari Therapeutics (NASDAQ:AKTX), an emerging growth, clinical-stage biopharmaceutical company, is pleased to announce its Chief Executive Officer Dr. David Horn Solomon and Chief Operations Officer Clive Richardson will present at the Cantor Fitzgerald Global Healthcare Conference on Tuesday September 26, 2017 in New York City. The presentation is scheduled for 10:55am.

ProQR Receives Orphan Drug Designation from FDA for Drug Candidate QR-313 for Dystrophic Epidermolysis Bullosa and will Present Data at two Scientific Conferences

Key Updates

Cidara Therapeutics to Present at Two Upcoming Conferences

SAN DIEGO, Sept. 18, 2017 (GLOBE NEWSWIRE) -- Cidara Therapeutics, Inc. (Nasdaq:CDTX), a biotechnology company developing novel anti-infectives including immunotherapies, today announced that Jeffrey Stein, Ph.D., president and chief executive officer of Cidara, is scheduled to present at two upcoming investor conferences.

SENS Research Foundation Announces New Research Program on Developing Monoclonal Antibodies Against Glucosepane With David Spiegel Lab


MOUNTAIN VIEW, Calif., Sept. 18, 2017 (GLOBE NEWSWIRE) -- SENS Research Foundation (SRF) has launched a new research program focused on developing monoclonal antibodies against glucosepane. David A. Spiegel, MD, PhD is Professor of Chemistry and Pharmacology at Yale University, and will be running the project in his laboratory, which focuses on developing new methods and molecules that will facilitate our understanding and treatment of human disease.                                                                                             

Cytokinetics Announces Presentation of Additional Results From COSMIC-HF at the HFSA 21st Annual Scientific Meeting


SOUTH SAN FRANCISCO, Calif., Sept. 18, 2017 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq:CYTK) today announced that additional results from COSMIC-HF (Chronic Oral Study of Myosin Activation to Increase Contractility in Heart Failure), a Phase 2 trial evaluating omecamtiv mecarbil in patients with chronic heart failure, were presented in a Rapid Fire Abstracts Presentation at the 21st Annual Heart Failure Society of America Scientific Meeting in Dallas, Texas. The results, presented by John Teerlink, M.D., Professor of Clinical Medicine at the University of California San Francisco and Director of Heart Failure at the San Francisco Veterans Affairs Medical Centers, suggest that omecamtiv mecarbil may produce similar results with regard to cardiac function, heart rate, biomarkers and adverse events in patients with ischemic and non-ischemic etiology of heart failure. Omecamtiv mecarbil, a novel investigational cardiac myosin activator that increases cardiac contractility, is being developed by Amgen in collaboration with Cytokinetics for the potential treatment of heart failure.

Axsome Therapeutics to Present at Upcoming Investor Conferences

NEW YORK, Sept. 18, 2017 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ:AXSM), a clinical-stage biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced that Herriot Tabuteau, M.D., Axsome’s Chief Executive Officer, will present a corporate overview at the following investor conferences:

BioCryst Pharmaceuticals Announces Full Exercise of Underwriters’ Option to Purchase Additional Shares and Completion of Public Offering of Common Stock


RESEARCH TRIANGLE PARK, N.C., Sept. 15, 2017 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) announced today the completion of an underwritten public offering of 17,864,078 shares of its common stock, including 2,330,097 shares sold pursuant to the exercise in full of the underwriters’ option to purchase additional shares.  The gross proceeds from this offering to BioCryst, including from the shares sold pursuant to the underwriters’ option to purchase additional shares, were $92 million, before deducting underwriting discounts and commissions and other estimated offering expenses payable by BioCryst.  BioCryst expects to use the net proceeds of this offering for general corporate purposes, which may include funding the global launch preparation of BCX7353; expanding our global awareness and commercial and manufacturing efforts; expanding our infrastructure, including commercial and manufacturing, to benefit BCX7353 and the rest of our programs; supporting the initiation of the Phase 3 clinical trial for BCX7353, the long term safety study, and all remaining preclinical and clinical studies to support a new drug application filing for BCX7353; funding an exploratory Phase 2 trial for acute treatment with BCX7353; and advancing our early-stage programs into clinical trials.