|Bid||300.40 x 0|
|Ask||300.50 x 0|
|Day's Range||293.05 - 300.75|
|52 Week Range||265.75 - 357.85|
|Beta (5Y Monthly)||0.22|
|PE Ratio (TTM)||18.18|
|Earnings Date||Feb 04, 2021|
|Forward Dividend & Yield||9.10 (3.01%)|
|Ex-Dividend Date||Mar 18, 2021|
|1y Target Est||259.86|
Roche Holding AG (OTCMKTS: RHHBY) has reported positive data from the dose-finding Part 1 FIREFISH Phase 2/3 study of Evrysdi (risdiplam) in infants with symptomatic Type 1 spinal muscular atrophy (SMA). Evrysdi helps in the survival of motor neuron 2 (SMN2) splicing modifier designed for treating SMA by boosting SMN protein production. The protein is vital to maintaining healthy motor neurons and movement. According to the latest data from 21 infant participants, Evrysdi treatment at 12 months helped 90% of infants survive without needing permanent ventilation, and 33% sit without support for at least five seconds. Furthermore, it raised the survival levels of SMN protein by a median 1.9-fold from baseline in the high-dose group at 12 months. In the low- and high-dose cohorts, no infant lost the ability to swallow over 12 months, and 86% were able to feed orally, either exclusively or in combination with a feeding tube at 12 months. Price Action: RHHBF is up 1.03% $344 in market trading hours on the last check Thursday. See more from BenzingaClick here for options trades from BenzingaOpthea Outlines OPT-302 Pivotal Study Designs In Chronic Eye DisorderRoche's Faricimab On Par With Rival Regeneron's Aflibercept In Four Late-Stage Studies In Vision Loss Disorder© 2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Here's a roundup of top developments in the biotech space over the last 24 hours: Scaling The Peaks (Biotech Stocks Hitting 52-week Highs Feb. 24) Apyx Medical Corp (NASDAQ: APYX) bluebird bio Inc (NASDAQ: BLUE) (reacted to fourth-quarter results) Five Prime Therapeutics Inc (NASDAQ: FPRX) Gemini Therapeutics Inc (NASDAQ: GMTX) Halozyme Therapeutics, Inc. (NASDAQ: HALO) (announced fourth-quarter results) Horizon Therapeutics PLC (NASDAQ: HZNP) (reacted to fourth-quarter results) Jazz Pharmaceuticals PLC (NASDAQ: JAZZ) Krystal Biotech Inc (NASDAQ: KRYS) MannKind Corporation (NASDAQ: MNKD) (reacted to positive analyst action) Orthofix Medical Inc (NASDAQ: OFIX) Pliant Therapeutics Inc (NASDAQ: PLRX) PRA Health Sciences Inc (NASDAQ: PRAH) - announced a deal to be bought by ICON PLC (NASDAQ: ICLR) Solid Biosciences Inc (NASDAQ: SLDB) Vanda Pharmaceuticals Inc. (NASDAQ: VNDA) Xencor Inc (NASDAQ: XNCR) (reacted to fourth-quarter results) Xenon Pharmaceuticals Inc (NASDAQ: XENE) Down In The Dumps (Biotech Stocks Hitting 52-week Lows Feb. 24) Acutus Medical Inc (NASDAQ: AFIB) Opthea Spon American Depositary Receipts Representing 8 Ord Shs (NASDAQ: OPT) (announced finalization of study design and start up activities for Phase 3 study of OPT-302 in wet age-related macular degeneration) Stocks In Focus Selecta Announces Publication Of Positive Preclinical Data For ImmTOR Platform In Gene Therapy Selecta Biosciences Inc (NASDAQ: SELB) announced the peer-reviewed publication of a study investigating the effects of the co-administration of ImmTOR nanoparticles to adeno-associated viral vectors on transgene expression in mice. The data, published in a Science Advances paper, demonstrated that the addition of ImmTOR nanoparticles to AAV vectors has the potential enhance the efficacy, safety and durability of gene therapies by mediating more efficient transgene expression at the first dose and by enabling vector redosing by preventing the formation of capsid-specific antibodies. The stock was up 9.66% premarket at $4.54. Karuna Says Positive Phase 2 Data For Schizophrenia Drug Published In NEJM PureTech Health's ADR Representing 10 Ord Shs (NASDAQ: PRTC) founded entity Karuna Therapeutics Inc (NASDAQ: KRTX) announced publication in the New England Journal of Medicine of positive results from the EMERGENT-1 Phase 2 clinical trial evaluating KarXT for the treatment of schizophrenia. KarXT demonstrated statistically significant and clinically meaningful improvements in primary and key secondary efficacy measures compared with placebo. Incidences of somnolence, weight gain and extrapyramidal symptoms were similar in KarXT and placebo treatment groups, the company said. Roche Announces Publication Of Positive Results For Evrysdi In Infants With Type 1 Spinal Muscular Dystrophy Roche Holdings AG Basel ADR Common Stock (OTC: RHHBY) said NEJM has published Evrysdi data from the dose finding Part 1 of the pivotal FIREFISH study in infants with symptomatic Type 1 spinal muscular atrophy. The data showed that treatment with Evrysdi at 12 months helped 90% of these infants survive without permanent ventilation and 33% sit without support for at least 5 seconds, which is not normally seen in the natural course of the disease. The study also found that treatment with Evrysdi increased the levels of survival of motor neuron protein by a median 1.9-fold from baseline in the high-dose cohort at 12 months. In August, the FDA approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older. Xencor To Collaborate With UCLA To Develop Therapeutic Antibodies Xencor announced an agreement with UCLA to develop novel therapeutic antibodies, pairing novel targets proposed by scientists at UCLA and utilizing Xencor's modular suite of XmAb technology platforms. Xencor and UCLA said they have established a streamlined framework to select promising biology, perform collaborative research and license intellectual property. Related Link: The Week Ahead In Biotech: J&J Vaccine Adcom Meeting, Sarepta FDA Decision And Plenty Of Earnings Moderna Higher Amid Multiple Vaccine Updates, Q4 Earnings Moderna Inc (NASDAQ: MRNA) said it has completed manufacturing of clinical trial material for its variant-specific vaccine candidate mRNA-1273.351 in the SARS-CoV-2 variant known as B.1.351 that was first identified in South Africa. The company said it has shipped doses to the National Institutes of Health for a Phase 1 clinical trial that will be led and funded by the NIH's National Institute of Allergy and Infectious Diseases. Additionally, the company said it is contemplating pursuing two strategies against these variants: evaluating booster doses of mRNA-1273 and evaluating mRNA-1273.351 and mRNA-1273.211 as a primary vaccination series for those who are seronegative. Separately, the company said it is making new capital investments to increase capacity at its owned and partnered manufacturing facilities, which it expects will increase global 2022 capacity to approximately 1.4 billion doses of its COVID-19 vaccine, assuming a 100 μg dose. The stock was up 4.08% to $150.70 premarket Thursday. AbbVie Receives Label Expansion For Rheumatoid Arthritis Drug AbbVie Inc (NYSE: ABBV) announced FDA approval for Humira in the treatment of moderately to severely active ulcerative colitis in pediatric patients 5 years of age and older. Altimmune Commences Phase 1 Study Of Intranasal COVID-19 Vaccine Altimmune Inc (NASDAQ: ALT) said it has commenced enrollment in a Phase 1 clinical trial of AdCOVID, a single-dose intranasal COVID-19 vaccine candidate. AdCOVID is an adenovirus-vector vaccine designed to stimulate a broad immune response including both systemic immunity and local immunity in the nasal cavity and respiratory tract. The stock was up 5.21% to $20.60 in premarket trading. Chimerix Announces Positive 2/3 Data For DTSTAT In COVID-19 Patients Chimerix Inc (NASDAQ: CMRX) reported positive topline results today from the first cohort of its Phase 2/3 study in hospitalized patients with acute lung injury and COVID-19 infection. "The promising results of this first cohort of 12 COVID-19 ALI patients provides clinical support for DSTAT's proposed mechanism of action and suggest it may accelerate recovery from ALI and mitigate thrombotic events in COVID-19 patients," said Mike Sherman, CEO of Chimerix. Merck to Buy Pandion For About $1.85B Merck & Co., Inc. (NYSE: MRK) announced a definitive agreement to buy Pandion Therapeutics Inc (NASDAQ: PAND), a clinical-stage biotech developing therapies for patients living with autoimmune diseases, for $60 per share in cash. This represents an approximate total equity value of $1.85 billion. View more earnings on IBB In premarket trading Thursday, Pandion shares were up 131.8% to $59.41 and Merck was adding 0.28% to $74.78. Pfizer/BioNTech To Test Additional Booster Dose, Variant-Specific Vaccine Pfizer Inc. (NYSE: PFE) and BioNTech SE – ADR (NASDAQ: BNTX) announced Thursday the beginning of an evaluation of the safety and immunogenicity of a third dose of the Pfizer-BioNTech COVID-19 vaccine BNT162b2 to understand the effect of a booster on immunity against COVID-19 caused by circulating and potentially newly emerging SARS-CoV-2 variants. The study is part of the clinical development strategy to determine the effectiveness of a third dose against evolving variants. Separately, in order to be prepared for any potential future strain changes, Pfizer and BioNTech said they are in ongoing discussions with regulatory authorities regarding a registration-enabling clinical study to evaluate a variant-specific vaccine with a modified mRNA sequence. Earnings Tandem Diabetes Care Inc (NASDAQ: TNDM) said its fourth-quarter revenue climbed about 55% to $168.07 million. The company's earnings per share climbed from 4 cents to 22 cents, in line with estimates. The company issued above-consensus revenue guidance for fiscal year 2021. The stock was up 12.55% at $102.76 premarket Thursday. Global Blood Therapeutics Inc's (NASDAQ: GBT) fourth-quarter net product sales climbed sharply from $2.11 million in 2019 to $41.3 million in 2020. The loss per share narrowed from $1.59 to $1, while analysts expected a narrower loss of 94 cents per share. In premarket trading Thursday, the stock was slipping 5.16% to $46.65. CytomX Therapeutics Inc (NASDAQ: CTMX) said its fourth-quarter revenue increased from $57.49 million in 2019 to $100.36 million in 2020. The net loss per share narrowed from $2.26 to 71 cents. The stock was advancing 5.6% to $8.49 in premarket trading Thursday. Guardant Health Inc (NASDAQ: GH) reported a wider-than-expected loss for the fourth quarter despite a 25% year-over-year increase in revenue. The stock was down 6.31% to $152 in premarket trading Thursday. Offerings Inflarx NV (NASDAQ: IFRX) said it has commenced an underwritten public offering of common shares of the company. For each common share purchased, an investor will also receive a warrant to purchase a common share of the company. The company said it intends to use the net proceeds from the offering primarily for R&D expenses and for working capital and general corporate purposes. The stock was down 5.11% premarket at $5.01. AVITA Medical Inc (NASDAQ: RCEL) priced its previously announced underwritten registered public offering of 2.795 million shares of its common stock at $21.50 per share. All of the shares of common stock to be sold in the offering are being offered by the company. AVITA Medical expects to close the offering on or about March 1. The stock was down 10.16% premarket Thursday at $22.55. Adamas Pharmaceuticals Inc (NASDAQ: ADMS) priced its underwritten public offering of 12.5 million shares of its common stock at a price of $4.40 per share. All of the shares in the offering are being sold by the company. The gross proceeds of the offering are expected to be $55 million. The stock was down 2.75% premarket at $4.60. On The Radar PDUFA Dates The FDA is scheduled to rule on Sarepta Therapeutics Inc's (NASDAQ: SRPT) NDA for casimersen, a phosphorodiamidate morpholino oligomer engineered to treat patients with Duchenne muscular dystrophy who have genetic mutations that are amenable to skipping exon 45 of the dystrophin gene. The PDUFA goal date is Thursday, Feb. 25. Clinical Readouts ACTRIMS Presentation: Clene Inc. (NASDAQ: CLNN): updated interim Phase 2 data from the REPAIR-MS study evaluating CNM-Au8 in relapsing multiple sclerosis and updated blinded interim Phase 2 data from the VISIONARY-MS study of CNM-Au8 in chronic optic neuropathy. Earnings Chimerix Inc (NASDAQ: CMRX) (before the market open) Concert Pharmaceuticals Inc (NASDAQ: CNCE) (before the market open) Kala Pharmaceuticals Inc (NASDAQ: KALA) (before the market open) Jounce Therapeutics Inc (NASDAQ: JNCE) (before the market open) Intellia Therapeutics Inc (NASDAQ: NTLA) (before the market open) Moderna (before the market open) Radius Health Inc (NASDAQ: RDUS) (before the market open) Insmed Incorporated (NASDAQ: INSM) (before the market open) Intercept Pharmaceuticals Inc (NASDAQ: ICPT) (before the market open) Editas Medicine Inc (NASDAQ: EDIT) (before the market open) Mediwound Ltd (NASDAQ: MDWD) (before the market open) Novocure Ltd (NASDAQ: NVCR) (before the market open) Glaukos Corp (NYSE: GKOS) (before the market open) BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) (before the market open) Pacira Biosciences Inc (NASDAQ: PCRX) (before the market open) Provention Bio Inc (NASDAQ: PRVB) (before the market open) Albireo Pharma Inc (NASDAQ: ALBO) (before the market open) Amarin Corporation plc (NASDAQ: AMRN) (before the market open) Adaptimmune Therapeutics PLC – ADR (NASDAQ: ADAP) (before the market open) Akebia Therapeutics Inc (NASDAQ: AKBA) (before the market open) Agios Pharmaceuticals Inc (NASDAQ: AGIO) (before the market open) Aerie Pharmaceuticals Inc (NASDAQ: AERI) (after the close) Apellis Pharmaceuticals Inc (NASDAQ: APLS) (after the close) Acceleron Pharma Inc (NASDAQ: XLRN) (after the close) Allogene Therapeutics Inc (NASDAQ: ALLO) (after the close) Puma Biotechnology Inc (NASDAQ: PBYI) (after the close) BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (after the close) Caladrius Biosciences Inc (NASDAQ: CLBS) (after the close) Iovance Biotherapeutics Inc (NASDAQ: IOVA) (after the close) Intra-Cellular Therapies Inc (NASDAQ: ITCI) (after the close) Nektar Therapeutics (NASDAQ: NKTR) (after the close) Natera Inc (NASDAQ: NTRA) (after the close) ICU Medical Inc (NASDAQ: ICUI) (after the close) Zogenix, Inc. (NASDAQ: ZGNX) (after the close) NuVasive, Inc. (NASDAQ: NUVA) (after the close) ZIOPHARM Oncology Inc. (NASDAQ: ZIOP) (after the close) PRA Health Sciences Inc (NASDAQ: PRAH) (after the close) Endo International PLC (NASDAQ: ENDP) (after the close) Lantheus Holdings Inc (NASDAQ: LNTH) (after the close) TRACON Pharmaceuticals Inc (NASDAQ: TCON) (after the close) PTC Therapeutics, Inc. (NASDAQ: PTCT) (after the close) LeMaitre Vascular Inc (NASDAQ: LMAT) (after the close) Voyager Therapeutics Inc (NASDAQ: VYGR) (after the close) Natus Medical Inc (NASDAQ: NTUS) (after the close) Cerus Corporation (NASDAQ: CERS) (after the close) Dynavax Technologies Corporation (NASDAQ: DVAX) (after the close) Cytokinetics, Inc. (NASDAQ: CYTK) (after the close) Collegium Pharmaceutical Inc (NASDAQ: COLL) (after the close) GenMark Diagnostics, Inc (NASDAQ: GNMK) (after the close) Dicerna Pharmaceuticals Inc (NASDAQ: DRNA) (after the close) Vir Biotechnology Inc (NASDAQ: VIR) Related Link: Attention Biotech Investors: Mark Your Calendar For February PDUFA Dates See more from BenzingaClick here for options trades from BenzingaThe Daily Biotech Pulse: Bausch Calls Truce With Icahn, Pfizer's Brain Inflammation Vaccine Gets Priority Review, BriaCell IPOThe Daily Biotech Pulse: Aridis To Study Antibody Cocktail For Coronavirus Variants, Revance & Protalix Data Readouts© 2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
PHILADELPHIA, Feb. 25, 2021 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the appointment of Cynthia Pussinen as chief technical officer (CTO). Cynthia has more than 25 years of experience in the biopharma industry, leading teams in manufacturing, quality, supply chain, formulation development and process sciences. As CTO, she will oversee Spark’s manufacturing and technical operations, including pipeline process development, preclinical and clinical manufacturing, and the commercial manufacturing and distribution of LUXTURNA® (voretigene neparvovec-rzyl). “As Spark looks to the future of gene therapy and our growing research and development portfolio, bringing products through the clinical trial process and successfully to market requires both breadth and depth in scaling technical development and operations,” said Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics. “We are excited to welcome Cynthia to Spark, as she brings both the expertise and leadership needed to be successful in this role. We are confident she will be an excellent addition to our management team as we prepare to bring forward the next generation of gene therapies to patients.” Previously, Cynthia served as global vice president and general manager, Life Sciences and Specialty Chemicals, for Honeywell International, Inc., where she developed and executed a comprehensive strategy designed for significant, sustained growth and innovation across multiple verticals including process automation and artificial intelligence (AI). “Gene therapies represent the present and future of medicine, and it is an honor to join Spark, the proven gene therapy leader that is building on its successes to unlock the full potential of gene therapy for patients with inherited diseases,” said Pussinen. “I admire the Spark team’s passion for the important role they play in delivering innovative gene therapies to patients, and I look forward to joining the company and making an impact on those patients’ lives.” Prior to her role at Honeywell, she was the executive vice president, Technical Development, Operations and Supply Chain for Actinium Pharmaceuticals, Inc. Cynthia also worked at Ipsen Biosciences, the U.S. subsidiary of Ipsen, serving as president and, prior to that, vice president of Technical Operations and Development. She previously spent more than 18 years at Pfizer in a variety of roles including portfolio and project management, logistics and supply chain, pharmaceutical sciences and as a scientist in medicinal chemistry. Cynthia holds a Master of Science in R&D management from Rensselaer Polytechnic Institute and a Bachelor of Science in chemistry, with a minor in engineering, from the University of Connecticut. About Spark Therapeutics At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We currently have four programs in clinical trials. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit www.sparktx.com, and follow us on Twitter and LinkedIn. Media Contact:Kevin Giordanokevin.email@example.com(215) 294-9942