Eli Lilly cuts FY19 EPS view to $5.55-$5.65 from $5.90-$6.00, consensus $5.77

Cuts FY19 revenue view to $25.1B-$25.6B from $25.3B-$25.8B, consensus $25.27B.

https://thefly.com/landingPageNews.php?id=2859779

Capricor Therapeutics resumes dosing of enrolled patients in HOPE-2 trial

Capricor Therapeutics (NASDAQ: CAPR), a clinical-stage biotechnology company, today announced that it has resumed per protocol dosing of patients already enrolled in its HOPE-2 clinical trial of CAP-1002, the company’s novel cell therapy candidate to treat Duchenne muscular dystrophy. Approximately 20 young men and boys in advanced stages of Duchenne muscular dystrophy have already been enrolled in the randomized, double-blind, placebo-controlled trial to date. Capricor had put a voluntary hold on dosing in December after a patient in the HOPE-2 trial had a serious adverse event in the form of anaphylaxis. The investigation suggested the patient may have been allergic to something contained in the investigational product, including an excipient, or inactive ingredient, in the formulation. To reduce the risk of future events, Capricor initiated a pre-medication strategy commonly used by physicians to prevent and treat allergic reactions. The U.S. Food and Drug Administration and the Data and Safety Monitoring Board have granted permission to resume enrollment in the HOPE-2 study. The HOPE-2 trial is studying the safety and effectiveness of CAP-1002 in older Duchenne patients who are not currently eligible for gene therapy clinical trials. Enrollment of new patients will depend on various factors but will not commence until additional funding is secured. To reduce expenses and better align resources and personnel on the company’s core lead programs, Capricor has reduced its staff by 21 full-time employees. The reduction in operating expenses is expected to extend the company’s cash, cash equivalents and marketable securities into late 2019. Additionally, Capricor is exploring strategic alternatives, with respect to one or more of its product candidates.

https://thefly.com/landingPageNews.php?id=2859819

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.
https://thefly.com/landingPageNews.php?id=2859857

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