The American Society of Hematology (ASH) hosted its 61st annual meeting, and one of the biggest biotech events, earlier this week in Orlando, Florida. This conference is usually a big catalyst for biotechs working with blood cancer treatments and other blood-related diseases. A number of companies present results from their studies in this field, and investors cheer or boo these stocks in response.
Wedbush to an in-depth look at some of the major names at the conference in its most recent report and gave some insight on to what may be the next step for the companies.
Argenx S.E. (NASDAQ: ARGX) presented an updated follow-up from the cusatuzumab Phase 1 dose-escalation trial in patients with newly diagnosed acute myeloid leukemia (AML) who are unfit for intensive chemotherapy. In the study, there was a deepening of response from 92% (11/12) to 100% (12/12), as an additional patient achieved a partial response — interestingly at about 10 months — while the remaining patients included eight with complete response. Wedbush has an Outperform rating with a $179 price target for the stock.
Bluebird Bio Inc. (NASDAQ: BLUE) reported results from the pivotal Phase 2 study of idecabtagene vicleucel in relapsed/refractory multiple myeloma. Patients across all dose levels showed an objective response rate of 73.4% (94/128). Topline data are in line with prior results and expectations.
Separately, the 206 data update for LentiGlobin in sickle cell patients was encouraging. Patients continued to express high levels of T87Q hemoglobin that led to improvements in disease markers, and importantly, in near elimination of vaso-occlusive crises and acute chest syndrome events. Wedbush has an Outperform rating with a $124 price target.
Blueprint Medicines Corp. (NASDAQ: BPMC) presented initial Phase 2 data from its Pioneer study of avapritinib in patients with indolent systemic mastocytosis. The topline data showed avapritinib hitting relevant biomarker endpoints with demonstrations of sustained reductions in tryptase levels (a measure of mast cell burden) at all three dose levels evaluated. Wedbush has an Outperform rating with a $113 price target.
Epizyme Inc. (NASDAQ: EPZM) gave an update on tazemetostat. Wedbush has an Outperform rating with a $21 price target. The boutique brokerage firm detailed in its report:
Responses confirmed by central review in follicular lymphoma (FL) studies, we believe the response rates (69% in EZH2 mutant and 35% for wild type) and durability (13.8 and 11.1 months PFS) support approval. While we expect some volatility around the ODAC panel on December 18, we expect the drug will be approved in epithelioid sarcoma (PDUFA on January 23, 2020). Importantly, that approval will allow for the company to get drug on formularies for health care plans that will speed the eventual launch of taz in FL so there could be upside to our initial estimates. Epizyme plans to submit the NDA for taz in FL later this month. Also, from the presentation, 18% of patients continued treatment past progression of disease which could provide upside to our estimates.
Fate Therapeutics Inc. (NASDAQ: FATE) presented updated data from its Phase 1 lead induced-pluripotent-stem-cell-derived natural killer cell candidates FT500 and FT516. Overall Wedbush was encouraged by the favorable safety and tolerability profile of the cell products, their notable lack of immunogenicity, and early signs of anti-tumor activity. Wedbush has an Outperform rating with a $24 price target.
Magenta Therapeutics Inc. (NASDAQ: MGTA) presented updated Phase 1 healthy volunteer data of stem cell mobilization agent MGTA-145, which showed not only one-day mobilization in real-world healthy volunteers, with a relatively broad age range, but the stem cells were also of high quality and at levels well exceeding the lower limits for transplantation. MGTA-145 treated subjects experienced mostly grade 1 back pain, but of short duration. Wedbush has an Outperform rating with a $22 price target.