Axsome Therapeutics reaches agreement under SPA for MOMENTUM Phase 3 trial

Axsome Therapeutics has reached agreement with the FDA under a special protocol assessment, or SPA, for the design, endpoints and statistical approach of the planned MOMENTUM Phase 3 trial of AXS-07 in the acute treatment of migraine. MOMENTUM will enroll only patients with a history of inadequate response to prior migraine treatments. The SPA provides agreement that the MOMENTUM trial design adequately addresses objectives that, if met, will support the regulatory submission for approval of AXS-07 for the indication of acute treatment of migraine in adults with or without aura. Based on FDA feedback during this SPA process, Axsome believes that only one Phase 3 trial may be needed for the approval of AXS-07. Axsome anticipates starting the MOMENTUM trial in Q1, with topline results expected within approximately one year of trial initiation.
https://thefly.com/landingPageNews.php?id=2859851

Marinus Pharmaceuticals initiated at Leerink

Marinus Pharmaceuticals initiated with an Outperform at Leerink. Leerink initiated Marinus Pharmaceuticals with an Outperform and $10 price target.
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Vanda files complaint against FDA seeking to lift clinical hold on study

Vanda Pharmaceuticals announced last night that it has filed a complaint against the FDA requesting that the court lift a partial clinical hold the agency “illegally imposed prohibiting Vanda from studying a new drug in humans for more than 12 weeks without conducting unnecessary and unethical animal studies.” The company explained, “Vanda has taken this legal action as it works through the FDA development process for tradipitant, a potential treatment for several human conditions including gastroparesis. During the course of drug development, animal studies are routinely conducted to identify potential toxicities in humans. FDA guidance documents outline the types of animal studies, including the appropriate species and duration, that are recommended to provide sufficient evidence of safety before a drug is studied in humans. However, the recommendations in FDA guidance documents are not legally binding on either the FDA or drug developers. If a company submits information to the FDA to show that further study in humans would be safe based on different information, the FDA is supposed to evaluate the company’s proposal and make a case-specific, science-based determination as to whether it agrees. In Vanda’s case, the FDA did not do so. It has instead treated a non-binding recommendation that nine-month non-rodent toxicity studies should be conducted before a drug is studied in humans for longer than three months as a non-negotiable requirement. Solely because Vanda has refused to conduct this study, which usually involves young beagles as the test subjects, each of which must be “sacrificed” to permit evaluation of the animal’s tissues, the FDA has placed a partial clinical hold on Vanda’s studies of tradipitant. Tradipitant studies can proceed up to 12 weeks duration. The FDA has imposed this partial hold without providing any specific scientific justification. As a matter of law, the FDA is not permitted to do that. As a result, Vanda has sued in federal court for judicial relief. ”
https://thefly.com/landingPageNews.php?id=2859867

Fate Therapeutics announces FDA clearance for FT516 IND

Fate Therapeutics announced that the FDA has allowed its investigational new drug, or IND, application for FT516, the company’s off-the-shelf natural killer, or NK, cell product candidate derived from a clonal master induced pluripotent stem cell, or iPSC, line engineered to express a novel CD16 Fc receptor. FT516 is the first-ever cell therapy derived from a genetically engineered pluripotent stem cell cleared for clinical testing in the world, and is the company’s second off-the-shelf, iPSC-derived NK cell product candidate cleared for clinical investigation by the FDA within the past two months. The company intends to initiate clinical testing of FT516 in patients with certain relapsed/refractory hematologic malignancies, including acute myelogenous leukemia, or AML, as a monotherapy, non-Hodgkin’s lymphoma, or NHL, in combination with rituximab, and multiple myeloma, or MM, in combination with elotuzumab. CD16 is naturally expressed on NK cells and mediates antibody-dependent cellular cytotoxicity, or ADCC, a potent immune mechanism through which NK cells can recognize, bind and kill antibody-coated cancer cells. ADCC is an underlying mechanism associated with the clinical efficacy of many monoclonal antibodies that are approved for the treatment of various cancers, including hematologic malignancies and solid tumors. The expression of CD16 on NK cells can undergo considerable down-regulation in cancer patients, which significantly inhibits the immune system’s anti-tumor response. FT516 incorporates a novel CD16 Fc receptor, which has been modified to prevent its down-regulation and to augment its binding to tumor-targeting antibodies, for enhanced ADCC. The initial clinical study of FT516 is intended to assess the safety and tolerability of three weekly doses for the treatment of certain relapsed/refractory hematologic malignancies. The study includes three independent, dose-escalating treatment arms: monotherapy for AML; combination with rituximab for NHL; and combination with elotuzumab, plus pomalidomide and dexamethasone, for MM. All subjects will receive low-dose conditioning chemotherapy consisting of cyclophosphamide and fludarabine and cytokine support with IL-2. Subjects are eligible to receive a second treatment cycle following an initial 28-day safety assessment.

https://thefly.com/landingPageNews.php?id=2859950

Centene price target lowered to $83 from $165 at Oppenheimer

Centene price target lowered to $83 from $165 at Oppenheimer. Oppenheimer analyst Michael lowered his price target for Centene to $83 from $165 to reflect split. The analyst notes that the company’s Q4 results were solid, and its outlook remains strong, given the growth and stability of the ACA business, and the potential for $500M in savings over many years from the Centene Forward initiative. Given the strong prospects and attractive valuation, he reiterates an Outperform rating on the shares.
https://thefly.com/landingPageNews.php?id=2859995

Vertex downgraded to Hold at Maxim on valuation

Maxim analyst Jason McCarthy downgraded Vertex to Hold from Buy after its better than expected Q4 results. The analyst notes that 2018 marks another “great year” for the company’s CF business, with revenue of $868M topping consensus while rising 11% from last year. McCarthy adds that he still expects the franchise to grow amid Symdeko launch ramps in U.S. and Europe, but sees the valuation on the stock being potentially stretched.
https://thefly.com/landingPageNews.php?id=2859999

Jefferies highlights Karyopharm, Reata as stocks to focus on in 2019

Jefferies analyst Maury highlights Karyopharm Therapeutics (KPTI) and Reata Pharmaceuticals (RETA) as two stocks to focus on in 2019. Karyopharm has important regulatory updates upcoming with an expected FDA panel for selinexor in penta-refractory multiple myeloma and then an FDA action date scheduled for April 6, Raycroft tells investors in a research note. And Reata is expected to report initial, awaited focal segmental glomerulosclerosis data in the first half of 2019 and then two pivotal readouts in the second half in Alport syndrome and Friedreich’s ataxia, adds the analyst. He believes the current share weakness offers an “appealing entry point” for Karyopharm.

https://thefly.com/landingPageNews.php?id=2860008