|Bid||6.88 x 1300|
|Ask||7.08 x 900|
|Day's Range||6.80 - 7.28|
|52 Week Range||3.76 - 15.02|
|Beta (5Y Monthly)||1.31|
|PE Ratio (TTM)||N/A|
|Earnings Date||Nov 03, 2020|
|Forward Dividend & Yield||N/A (N/A)|
|1y Target Est||14.56|
The FDA bestows a Regenerative Medicine Advanced Therapy status on Orchard Therapeutics' (ORTX) investigational gene therapy, OTL-200, for treating early-onset metachromatic leukodystrophy. Shares up.
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 Jan. 13) * Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) * Atea Pharmaceuticals Inc (NASDAQ: AVIR) * Bioanalytical Systems, Inc. (NASDAQ: BASI) * Bioatla Inc (NASDAQ: BCAB) * Biodesix Inc (NASDAQ: BDSX) (announced a collaboration with HiberCell for the development of an enzyme-linked immunosorbent assay as a companion diagnostic in future registrational trials in breast cancer for Imprime PGG programs) * BIOLASE Inc (NASDAQ: BIOL) * C4 Therapeutics Inc (NASDAQ: CCCC) * Cassava Sciences Inc (NASDAQ: SAVA) * Celcuity Inc (NASDAQ: CELC) * CareDx Inc (NASDAQ: CDNA) * Celsius Holdings, Inc. (NASDAQ: CELH) * DermTech Inc (NASDAQ: DMTK) (announced inclusion of non-invasive genomic patch testing for melanoma in National Comprehensive Cancer Network program) * Dyne Therapeutics Inc (NASDAQ: DYN) * Edap Tms SA (NASDAQ: EDAP) * ESSA Pharma Inc (NASDAQ: EPIX) - announced a clinical collaboration with Johnson & Johnson (NYSE: JNJ) unit for prostate cancer study * Genetron Holdings Ltd - ADR (NASDAQ: GTH) * Genmab 10 Sponsored ADR Ord Shs (NASDAQ: GMAB) (announced achievement of $40 million milestone payment due from AbbVie for diffuse large B-cell lymphoma study) * Glaukos Corp (NYSE: GKOS) (announced positive Phase 2b data for iDose TR sustained-release travoprost implant) * Guardant Health Inc (NASDAQ: GH) * Halozyme Therapeutics, Inc. (NASDAQ: HALO) * Inari Medical Inc (NASDAQ: NARI) (reacted to preliminary fourth-quarter results) * Insulet Corporation (NASDAQ: PODD) * Kaleido Biosciences Inc (NASDAQ: KLDO) * Kodiak Sciences Inc (NASDAQ: KOD) * Laboratory Corp. of America Holdings (NYSE: LH) * Mediwound Ltd (NASDAQ: MDWD) * Natera Inc (NASDAQ: NTRA) * Neuronetics Inc (NASDAQ: STIM) (reacted to preliminary fourth-quarter results) * Novocure Ltd (NASDAQ: NVCR) * Pacira Biosciences Inc (NASDAQ: PCRX) * Renalytix AI PLC (NASDAQ: RNLX) * SAGE Therapeutics Inc (NASDAQ: SAGE) - Biogen Inc (NASDAQ: BIIB) disclosed a 10.7% stake in the company * Shockwave Medical Inc (NASDAQ: SWAV) * Supernus Pharmaceuticals Inc (NASDAQ: SUPN) * Syneos Health Inc (NASDAQ: SYNH) * TFF Pharmaceuticals Inc (NASDAQ: TFFP) * TG Therapeutics Inc common stock (NASDAQ: TGTX) * Vericel Corp (NASDAQ: VCEL) * X T L Biopharmaceuticals Ltd (NASDAQ: XTLB) * Zai Lab Ltd - ADR (NASDAQ: ZLAB)Down In The Dumps None of the biotech/medical device/diagnostics shares hit 52-week lows in the session.Stocks In Focus Alexion Halts Late-Stage COVID-19 Study In Patients Requiring Mechanical Ventilation Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) announced its decision to pause further enrollment in the global Phase 3 study of Ultomiris in adults with severe COVID-19 requiring mechanical ventilation.This decision is based on the recommendation of an independent data monitoring committee following the review of data from a pre-specified interim analysis. The IDMC recommended that additional enrollment be paused pending further analysis of the data due to lack of efficacy when Ultomiris was added to best supportive care, compared to best supportive care alone.NantKwest Reports Positive Results For Combo Therapy In Advanced Pancreatic Cancer Nantkwest Inc (NASDAQ: NK) and Immunity Bio announced early interim results from the pivotal QUILT 88 Phase 2 study trial that is evaluating standard-of-care chemotherapy versus standard-of-care chemotherapy in combination with PD-L1 t-haNK, Anktiva and aldoxorubicin, showing median survival rates more than doubled that of the historic rate in patients with advanced metastatic pancreatic cancer for whom no other FDA-approved treatment exists.The company noted that primary endpoint of overall survival was achieved, with 15 out of 18 (83%) of patients enrolled with second-line or greater pancreatic cancer remaining alive to date.Orchard's Gene Therapy Designated As Regenerative Medicine Advanced Therapy Orchard Therapeutics PLC - ADR (NASDAQ: ORTX) said the FDA granted a regenerative medicine advanced therapy designation to OTL-200, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of early onset metachromatic leukodystrophy.The stock was adding 6.74% premarket to $6.65.Related Link: The Week Ahead In Biotech (Jan 10-16): Expect Stocks To Move As J.P. Morgan Healthcare Conference Kicks Off Preannouncements Apyx Medical Corp (NASDAQ: APYX) preannounced fourth-quarter revenue of $10.9 million to $11.3 million, representing a 30%-35% year-over-year increase. The company guided to full-year revenue of $27.1 million to $27.5 million. Analysts, on average, estimated revenues of $8.09 million for the fourth quarter and $24.35 million for the full year.The stock rallied 24.30% to $9.72 in after-hours session.Organogenesis Holdings Inc (NASDAQ: ORGO) said it expects fourth-quarter revenue of $104.6 million to $106 million, up 40%-42% year-over-year, and full-year net revenue of $336.1 million to $337.5 million, up about 29%.This notably exceeded the consensus estimates that call for revenue of $80.84 million for the quarter and $312.34 million for the year.Separately, the company announced the commencement of a pivotal Phase 3 clinical trial evaluating the safety and efficacy of ReNu, a cryopreserved amniotic suspension allograft, for the management of symptoms associated with knee osteoarthritis.The stock was surging 32.15% premarket Thursday to $9.70.Integra Lifesciences Holdings Corp (NASDAQ: IART) preannounced fourth-quarter revenue of $387 million to $389 million, a decline of 1.6%-2.1%. For the full year, the company expects revenue in a range of $1.37 billion to $1.372 billion, representing a decrease of 9.7% to 9.6%.Analysts, on average, estimate revenues of $389.05 million for the quarter and $1.37 billion for the year.The stock was slipping 0.96% to $65.02 in premarket trading Thursday.Offerings Evoke Pharma Inc (NASDAQ: EVOK) said it has priced an underwritten public offering of 5 million shares of its common stock at $2.50 per share to raise gross proceeds of $12.5 million.The stock was up 1.83% premarket at $2.78.Aldeyra Therapeutics Inc (NASDAQ: ALDX) priced an underwritten public offering of 6.842 million shares of its common stock at $9.50 per share. The gross proceeds from the offering are expected to be approximately $65 million.The company anticipates using the net proceeds from the offering for the continued development of its lead compound reproxalap, and its other product candidates, as well as for working capital and other general corporate purposes.The stock was up 5.01% premarket at $10.68.Related Link: Attention Biotech Investors: Mark Your Calendar For January PDUFA Dates See more from Benzinga * Click here for options trades from Benzinga * The Daily Biotech Pulse: Bausch Health Issues Positive Pre-Announcement, Acorda Streamlines Operations * The Daily Biotech Pulse: GSK, Vir To Start COVID-19 Antibody Study, Novartis In-Licenses BeiGene's Cancer Drug, Earnings Preannouncements Continue(C) 2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Interactions with FDA anticipated by mid-2021 to determine the BLA filing strategy for OTL-200BOSTON and LONDON, Jan. 14, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OTL-200, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of early-onset metachromatic leukodystrophy (MLD). In late 2020, the FDA cleared the company’s Investigational New Drug (IND) application for OTL-200, and the therapy also recently was approved in the European Union (EU) under the brand name, LibmeldyTM. “Receipt of RMAT designation for OTL-200 underscores both the severe nature of MLD and the transformative potential of the therapy for young patients suffering from this devastating, fatal neurodegenerative condition,” said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. “Alongside our open IND, RMAT designation provides an opportunity for enhanced interactions with the FDA to determine the optimal path to submit a Biologics License Application (BLA) for OTL-200 in the U.S.”Established under the 21st Century Cures Act, the RMAT designation program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse or cure a serious condition. The FDA granted Orchard RMAT designation for OTL-200 based on data submitted on 39 patients, including 9 patients from the U.S., who have received OTL-200 as part of clinical studies and compassionate use programs conducted at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. This data set includes post-treatment follow-up data of up to eight years in the earliest treated patients in these programs.“We look forward to continued engagement with the FDA in the coming months to discuss the comprehensive data set we have already collected in the OTL-200 clinical development program and agree on the potential next steps on the regulatory path to approval for this innovative gene therapy,” said Anne Dupraz, chief regulatory officer at Orchard.About Libmeldy / OTL-200 Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the European Medicines Agency (EMA) website. Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein, and Norway. OTL-200 is an investigational therapy in the U.S.Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit www.orchard-tx.com, and follow us on Twitter and LinkedIn.Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchard’s investor relations website and may include additional social media channels. The contents of Orchard’s website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchard’s strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchard’s business strategy and goals, the therapeutic potential of Libmeldy (OTL-200), the likelihood that data from clinical trials will support further clinical development and regulatory approval of OTL-200, and the outcome of planned FDA interactions regarding the potential approval pathway for OTL-200. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchard’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of OTL-200, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that OTL-200 or any one or more of Orchard’s product candidates will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchard’s ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the delay of any of Orchard’s regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchard’s product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchard’s ability to commercialize OTL-200, if approved, or Libmeldy in the EU; the risk that the market opportunity for Libmeldy, or any of Orchard’s product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchard’s business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchard’s quarterly report on Form 10-Q for the quarter ended September 30, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchard’s views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.ContactsInvestors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.comMedia Christine Harrison Vice President, Corporate Affairs +1 202-415-0137 firstname.lastname@example.org