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."

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."

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."