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18:05 EST Rocket says Phase 1 trial establishes feasibility of commercial Process B for FA - Rocket Pharmaceuticals announced "encouraging" preliminary results from its Phase 1 trial of commercial-grade RP-L102 "Process B" for Fanconi Anemia, or FA, at the 61st American Society of Hematology Annual Meeting. Results presented in the poster highlight preliminary Phase 1 data from two pediatric patients who were treated with "Process B" RP-L102 prior to the development of severe bone marrow failure and are in ongoing follow-up. Drug product was successfully manufactured using "Process B" optimization, including transduction enhancers, commercial-grade vector and modified cell processing. "Process B" drug product is manufactured to commercial grade standards, allowing for consistent drug product across patients and a vector copy number two to three fold higher than that administered to optimally-treated "Process A" patients. Once transduced, drug product was infused fresh into patients without any prior conditioning regimen. To evaluate transduction efficiency, an analysis of the proportion of the MMC-resistant colony forming cells was conducted. Both patients exhibited early signs of engraftment based on peripheral blood, VCN and/or MMC-resistance. Preliminary phenotypic correction was also apparent in both patients, as evidenced by stabilization or increases in blood cell lineages. No safety or tolerability issues have been reported. "The preliminary data presented at ASH provide evidence regarding the potential of our commercial-grade 'Process B' product in treating FA," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "Treatment with 'Process B' RP-L102 at four to six months post-infusion showed early signs of engraftment and bone marrow restoration. Further, previously declining blood cell counts appear to have been stabilized or even increased within six months of therapy, providing evidence regarding the potential benefits of treatment with a consistent, commercial-grade product. We are looking forward to presenting additional long-term follow-up data from these patients in the first half of 2020."

16:34 EST Eli Lilly presents interim clinical data from LOXO-305 dose escalation trial - Eli Lilly announced interim clinical data from the LOXO-305 global Phase 1/2 BRUIN dose escalation trial. LOXO-305 is an investigational, highly selective, non-covalent Bruton's tyrosine kinase inhibitor. These data were presented at the 2019 American Society of Hematology, or ASH, Annual Meeting. At all doses studied, LOXO-305 delivered objective responses in patients who had received diverse prior therapies and had exhibited varied molecular mechanisms of acquired resistance. "We are excited to report that LOXO-305 is active in patients resistant and intolerant to covalent BTK inhibitors, as well as in patients resistant to BCL2 inhibition," said Anthony Mato, M.D., director of the CLL Program at Memorial Sloan Kettering Cancer Center and the presenting author. "We observed compelling response rates in both CLL and MCL. Interestingly, we reported responses regardless of C481 status, a putative mechanism of resistance to the covalent BTK class. We saw objective responses in dose cohort 1 and have not identified a maximum tolerated dose. These data suggest that LOXO-305 has the required selectivity profile and human target coverage to maximize the full potential of this molecular target, combine well with other agents, and perhaps, even move to earlier lines of therapy." Jacob Van Naarden, chief operating officer of Loxo Oncology at Lilly, added: "Put simply, LOXO-305 has exceeded our expectations. LOXO-305's wide therapeutic index allows us to plan and implement an ambitious comprehensive development program, one that includes combination regimens that exploit the drug's selectivity profile. We look forward to working closely with global regulators to position LOXO-305 in the most appropriate patient populations. It will be exciting to combine Lilly's resources and talent with Loxo's focus and agility for the benefit of this program within Loxo Oncology at Lilly."

16:26 EST uniQure reports 1-year follow-up data from etranacogene dezaparvovec study - uniQure announced updated clinical data on the three patients treated in uniQure's ongoing Phase IIb study of etranacogene dezaparvovec, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. In addition, uniQure presented up to 4 years of follow-up data on the 10 patients in the Phase I/II trial of AMT-060, its first-generation gene therapy for the treatment of hemophilia B. These clinical data are being presented this weekend in poster presentations at the 61st Annual Meeting of the American Society of Hematology, or ASH. "These updated data show that a single administration of etranacogene dezaparvovec has been well-tolerated now out 52 weeks and has increased FIX activity into the therapeutic range for people living with hemophilia B," stated Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan and a principal investigator in the HOPE-B clinical trial. "These data show a full year of meaningful clinical benefit for all three patients in the study, including durable levels of FIX activity with no bleeds, no requirement for infusions of FIX replacement therapy outside of surgery, and no need for immunosuppression." Robert Gut, chief medical officer of uniQure, added: "We are very pleased with these latest results, and continue to believe that etranacogene dezaparvovec has the potential to be the first- and best-in-class gene therapy for patients with hemophilia B. We remain focused on dosing all patients in our ongoing, fully-enrolled HOPE-B pivotal trial, and expect to announce top-line data on our primary endpoint of Factor IX activity by the end of 2020."

12:27 EST Kura Oncology announces updates for lead drug candidate tipifarnib - Kura Oncology announced clinical and regulatory updates for its lead drug candidate, tipifarnib, in angioimmunoblastic T-cell lymphoma, including data from the Company's ongoing Phase 2 clinical trial of tipifarnib in relapsed or refractory peripheral T-cell lymphoma. The updated interim data are being presented during an oral session today at the American Society of Hematology Annual meeting. As of the November 11 data cutoff date, a total of 26 patients with relapsed or refractory AITL were enrolled in all stages of the trial. Among the 20 patients evaluable for efficacy, five achieved a complete response and five achieved a partial response, for an objective response rate of 50% on an evaluable basis and 38% on an intent-to-treat basis. In addition, three patients experienced disease stabilization. Patients had a median of three prior regimens. Next-generation sequencing of 19 available patient biopsies showed that 10 carried C336R/Q386E variants in the killer-cell immunoglobulin-like receptor 3DL2, an immune checkpoint receptor. Four of the 10 patients with KIR3DL2 variants achieved a CR and three achieved a PR, for a CR rate of 40% and an ORR of 70%. This compares to a CR rate of 11% and an ORR of 22% among the nine AITL patients with KIR3DL2 wild type. Tipifarnib was generally well-tolerated in this Phase 2 trial, with adverse events consistent with its known safety profile, Kura said. "We are encouraged by our growing body of data for tipifarnib in AITL," said Antonio Gualberto, Head of Development and Chief Medical Officer of Kura Oncology. "We believe these data support our efforts to expand the development of tipifarnib beyond our initial focus in HRAS mutant solid tumors and could enable registrational strategies in multiple CXCL12-dependent hematologic and solid tumor indications."

10:49 EST Fate Therapeutics announces vivo preclinical data for FT596 - Fate Therapeutics announced new in vivo preclinical data for FT596, its off-the-shelf, multi-antigen targeting natural killer cell product candidate derived from a clonal master engineered induced pluripotent stem cell line. The data were featured during the American Society of Hematology Meeting. New preclinical data showed that FT596 "administered as a monotherapy exhibited durable tumor clearance and extended survival in vivo similar to primary CAR T cells in a humanized mouse model of CD19+ lymphoma. Additionally, when combined with the anti-CD20 monoclonal antibody rituximab, FT596 showed enhanced killing of CD20+ lymphoma cells in vivo as compared to rituximab alone. These data confirm previously presented in vitro findings that demonstrate the unique multi-antigen targeting functionality of FT596, and the product candidate's potential to effectively overcome CD19 antigen escape," the company said. Fate also announced that, in preparation for Phase 1 initiation, it had recently completed GMP production of FT596. In a single small-scale manufacturing campaign, the company produced over 300 cryopreserved, infusion-ready doses of FT596 at a cost of approximately $2,500 per dose.

09:25 EST Autolus announces new data highlighting progress on AUTO3 - Autolus Therapeutics announced new data highlighting progress on AUTO3, the first-in-human bicistronic CD19 and CD22 CAR, in patients with relapsed/refractory diffuse large B-cell lymphoma and pediatric acute lymphoblastic leukemia. The data were presented at the American Society of Hematology Annual Meeting. In the dose escalation phase, 16 patients were treated, with 4 patients dosed at 50 x 106 cells without pembrolizumab; 11 patients were dosed at escalating doses of AUTO3 with pembrolizumab administered at day 14 as follows: 3 at 50 x 106 cells, 4 at 150 x 106 cells, and 4 at 450 x 106 of AUTO3; and 1 patient was dosed with 450 x 106 cells with pembrolizumab administered 1 day before AUTO3 infusion. Fourteen patients were evaluable at one month. AUTO3 was well-tolerated, with no patients experiencing greater than or equal to Grade 3 cytokine release syndrome with primary infusion and 1 of 14 experiencing Grade 3 neurotoxicity that resolved swiftly with steroids, the compamny said. There were no pembrolizumab immune-related toxicities and the majority of grade 3 or higher adverse events were hematological. Across all tested doses 5 patients achieved a complete response, with 4 of 5 complete responses ongoing, the longest at 18 months. All CRs were achieved without need for steroid or tocilizumab-based management of the patients or ICU level care, it added. Among the 10 CAR T-naive patients, at a median follow-up time of 9.7 months, 9 of 10 patients achieved a complete response, and 8 of 10 achieved complete molecular remission by PCR. Estimated overall survival at 12 months was 100%. There are 2 ongoing patients in complete molecular remission at 12 and 15 months post-AUTO3 infusion, respectively. The most common cause of relapse was due to loss of CAR T-cell persistence. The median persistence of CAR-T cells in blood was 170 days.

09:20 EST Ziopharm reports pre-clinical data of Rapid Personalized Manufacture - Ziopharm Oncology announced the presentation of pre-clinical data of Rapid Personalized Manufacture, in the "Adoptive Immunotherapy: Mechanisms and New Approaches" session at the American Society of Hematology Annual Meeting. "These pre-clinical data demonstrate that T cells genetically modified using DNA plasmids from the Sleeping Beauty system to express TCR with membrane bound IL-15 (mbIL15) exhibit anti-tumor effects," said Drew Deniger, leader of Ziopharm's TCR program. "These data build on our approach to reduce the cost and complexity of T-cell therapies. We have previously demonstrated that mbIL15 can be combined with a CD19-specific chimeric antigen receptor (CAR) to shorten the time to generate clinical-grade T cells and an IND is cleared to evaluate this RPM technology."

09:16 EST Karyopharm says four posters will be presented at ASH - Karyopharm Therapeutics announced that four posters relating to Xpovio and eltanexor will be presented at the American Society of Hematology Annual Meeting. The four posters include: updated data from the Kyprolis arm of the Phase 1b/2 Stomp study evaluating selinexor in combination with backbone therapies in patients with relapsed or refractory multiple myeloma; new data from the Revlimid plus selinexor arm of the Stomp study evaluating this combination in patients with newly diagnosed multiple myeloma; encore data highlighting the previously disclosed comparison of patients in the Storm study, Karyopharm's Phase 2b study evaluating Xpovio in patients with heavily pretreated, triple class refractory multiple myeloma, to matched patients from the Mamouth study; and new Phase 1/2 data evaluating eltanexor in patients with higher-risk myelodysplastic syndrome.

09:11 EST Regeneron reports initial data for REGN5458 in myeloma patients - Regeneron Pharmaceuticals announced initial clinical data for REGN5458, a BCMAxCD3 bispecific antibody, in patients with relapsed or refractory multiple myeloma. REGN5458 is designed to bind to BCMA on multiple myeloma cells and the CD3 receptor on T-cells, bridging them together and activating T-cell killing of the cancer cell. Results were presented today at the American Society of Hematology Annual Meeting from the first two dose groups. Patients had a median of seven lines of prior systemic therapy, and all had failed CD38 antibody treatment. Responses were observed in 4 of 7 patients, including 3 of 4 in the 6 mg dose group. In the 6 mg dose group, 2 patients were also minimal residual disease negative, meaning that no cancer cells were detectable in their bone marrow. "We are encouraged to see promising, rapid clinical activity even at the initial two doses of REGN5458 in heavily pretreated patients with multiple myeloma. Two patients achieved the high bar of MRD negativity, and another patient attained a very good partial response despite entering the trial with difficult-to-treat plasmacytomas outside of the bone marrow," said Israel Lowy, Senior Vice President and Head of Clinical and Translational Sciences for Oncology at Regeneron. "We are actively recruiting patients into higher dose groups in this trial and look forward to sharing further results in 2020. In addition, we have also initiated a clinical trial for our second BCMAxCD3 bispecific, REGN5459, which has different binding characteristics."

09:06 EST Blueprint announces initial data from Phase 2 Pioneer trial in mastocytosis - Blueprint Medicines announced initial data from the Phase 2 Pioneer clinical trial of avapritinib in patients with indolent systemic mastocytosis. Initial data from the dose-finding Part 1 of trial showed "rapid and robust reductions" in serum tryptase, a measure of mast cell burden, in patients treated with 25 mg, 50 mg or 100 mg of avapritinib once daily, the company said. All dose levels of avapritinib tested were well-tolerated, and no patients discontinued treatment due to an adverse event, it added.. The results will be presented today in a poster presentation at the American Society of Hematology Annual Meeting. At baseline, all patients had symptomatic disease despite best available therapy. Median Indolent SM Symptom Assessment Form total symptom score was 52. Patients were taking a median of three medications to treat their disease. Mean serum tryptase was 84 micrograms per liter. Across all avapritinib dose cohorts, reductions in serum tryptase were robust, occurred rapidly and were sustained in patients treated up to 30 weeks. The placebo cohort showed no change in serum tryptase at 12 weeks. All doses of avapritinib tested were well-tolerated, and most reported AEs were Grade 1 or 2, according to Blueprint. Across all avapritinib cohorts, five patients had Grade 3 AEs, and no patients had serious AEs. In patients treated with placebo, two patients had Grade 3 AEs, and two patients had serious AEs. There was one Grade 3 cognitive effect reported in the 100 mg cohort. The event resolved following dose modification, and the patient remained on therapy as of the data cutoff date. No patients discontinued treatment due to an AE. Blueprint plans to submit a new drug application to the FDA for avapritinib for the treatment of patients with advanced SM in Q1 of 2020.

08:06 EST AbbVie says post-hoc analysis supports benefit of venetoclax in combination - AbbVie presented long-term data from a post-hoc analysis, which it says further supports the sustained clinical benefit of fixed duration treatment with venetoclax in combination with rituximab in patients with relapsed or refractory chronic lymphocytic leukemia. The updated data from the Phase 3 Murano trial four-year analysis showed that patients with R/R CLL who completed the chemotherapy-free, two-year fixed duration course of venetoclax treatment combination maintained progression-free survival and overall survival, AbbVie said. Patients who completed treatment with the venetoclax combination also achieved higher rates of minimal residual disease-negativity and complete remissions compared to those treated with a standard of care, bendamustine plus rituximab. In the post-hoc analysis, median follow-up for patients who completed two years of treatment with the venetoclax combination without progressive disease was 22 months. By the end of treatment, 64% of patients had achieved MRD-negativity, and 87% of those patients remained free of disease progression two years post-treatment. "These results support the benefits of a fixed duration of treatment with venetoclax to reduce the risk of disease progression or death in patients with chronic lymphocytic leukemia," said Mohammed Zaki, vice president, global head of hematology development at AbbVie. "We remain committed to understanding the full utility of venetoclax combinations and to advancing other clinical development programs with the potential to transform the standards of care for patients with blood cancers."

07:59 EST Orchard to present data from multiple programs at ASH meeting - Orchard Therapeutics said it presenting new registrational data from multiple programs at the American Society of Hematology Annual Meeting. On Sunday, December 8, investigators will describe ongoing clinical progress for two lead development programs in the company's primary immune deficiencies portfolio: OTL-103, an investigational gene therapy in development for the treatment of Wiskott-Aldrich syndrome and OTL-101, an investigational gene therapy in development for the treatment of adenosine deaminase severe combined immunodeficiency. In addition, on Monday, December 9, investigators will deliver an oral presentation featuring updated data from the ongoing clinical proof-of-concept study of OTL-203, an investigational gene therapy in development for the treatment of mucopolysaccharidosis type I.