The last major healthcare conference of the year is the ASH hematology conference and will be held from December 7-10, 2019.
Below are the publicly traded companies presenting at the event next weekend.
ARQL = B. Riley FBR analyst Mayank Mamtani resumed coverage of ArQule with a Buy rating and $14 price target. The company will present ARQ 531 B-cell malignancies which is in Phase 1 clinical trials at ASH on December 9, 2019 at 6pm EST.
AUTL = CEO said, “We are excited about the opportunity to share data updates at ASH on AUTO1 in ALL in three oral presentations, as well as an oral presentation on AUTO3 in DLBCL. We are also looking forward to presenting data on our other hematological clinical programs at ASH, and non-clinical data on our lead solid tumor program AUTO6NG at SITC,” stated Dr. Christian Itin, chairman and chief executive officer of Autolus. “This quarter we have made significant operational progress, delivering cell products from our new manufacturing operations at the Cell and Gene Therapy Catapult and further strengthening our management team. Supported by a strong balance sheet, our key focus is on moving AUTO1 into our first pivotal clinical program in adult patients with ALL.”
On November 13, 2019, Autolus Therapeutics plc (Nasdaq: AUTL) and Noile-Immune Biotech, Inc., Tokyo, Japan, announced that they have entered into a license agreement whereby Autolus will have the right to develop CAR T cell therapies incorporating Noile-Immune’s PRIME (proliferation-inducing and migration-enhancing) technology secreting both IL-7 and CCL19. The PRIME technology is designed to improve proliferation and trafficking into solid tumors of both engineered CAR T cells as well as the patient’s own T cells.
Through the license agreement with Noile-Immune, Autolus has gained the right to incorporate the PRIME technology in its CAR T cell programs against a limited number of cancer targets. Autolus will make an upfront cash payment and milestone payments to Noile‑Immune, and Noile-Immune is also entitled to receive royalties on net sales of resulting products. Additional terms of the agreement were not disclosed.
“We have an emerging pipeline of CAR T cell products targeting solid tumors and are excited to add the Noile-Immune PRIME technology to our existing suite of T cell programming modules,” said Dr. Christian Itin, chairman and chief executive officer of Autolus. “We believe the PRIME technology will help enhance the anti-tumor potential of CAR T cells and expand our ability to design and develop transformational CAR T therapies in a variety of solid tumor indications.”
“Autolus is a leader in the CAR T field, and we are very pleased that they have selected our PRIME technology for their solid tumor pipeline,” said Hidenobu Ishizaki, M.D., Ph.D., president & chief executive officer of Noile-Immune. “This agreement provides further endorsement of our technologies, which were invented by Dr. Koji Tamada, our scientific founder. We look forward to continuing to contribute to, and develop, therapies that have the potential to transform the lives of patients.”
The company will present ARQ 531 B-cell malignancies which is in Phase 1 clinical trials at ASH on December 9, 2019 at 6pm EST.
BLUE = Will have two major presentations at ASH next week. The company will present its phase 3 LentiGlobin – HGB-207 Northstar-2 non-β0/β0 transfusion-dependent thalassemia (TDT) clinical trial data at ASH December 9, 2019, 6pm. The company will also be presenting its phase 1 bb21217 Multiple Myeloma data at ASH December 9, 2019, 6:45 p.m. ET.
On November 26, 2019, SVB Leerink analyst Mani Foroohar upgraded Bluebird Bio to Outperform from Market Perform with a $119 price target.
On November 19, 2019, Evercore ISI analyst Joshua Schimmer downgraded Bluebird Bio to In Line from Outperform with a price target of $86, down from $172. The analyst said Bluebird continues to spend heavily on SG&A and R&D and is steering its lentivirus platform more towards the crowded and challenging cancer therapeutics field and away from traditional monogenic diseases.
On November 12, 2019, Bluebird Bio (BLUE) and Forty Seven (FTSV) announced that they have entered into a research collaboration to pursue clinical proof-of-concept for Forty Seven’s novel antibody-based conditioning regimen, FSI-174 plus magrolimab, with bluebird’s ex vivo lentiviral vector hematopoietic stem cell, or LVV HSC, gene therapy platform. This collaboration will focus on a conditioning approach aimed to deliver reduced toxicity and will initially target diseases that have the potential to be corrected with transplantation of autologous gene-modified blood-forming stem cells. If successful, the new conditioning regimen could allow for more patients to undergo gene therapy. Under the terms of the agreement, Bluebird bio will provide its ex vivo LVV HSC gene therapy platform and Forty Seven will contribute its antibody-based conditioning regimen for the collaboration.
On November 4, 2019, Wedbush analyst David Nierengarten upgraded Bluebird Bio to Outperform from Neutral with a price target of $124, down from $131, saying he thinks shares are reflecting significant negativity regarding the Zynteglo launch at current levels. While the analyst says he “shares the concerns,” he feels negative Zynteglo sentiment is “more than accounted for” in the current valuation. Nierengarten recommends getting off the sidelines for both valuation and near-term data catalysts.
On October 31, 2019, Piper Jaffray analyst Tyler Van Buren lowered his price target on Bluebird Bio to $85 after its Q3 earnings miss, stating that while he is eager to see its KarMMa dataset for ide-cel/bb2121 by year-end, he sees expectations of median progression free survival of 11.8 months as already relatively high. With ide-cel BLA submission having been delayed a few months from 2019-end to the first half of 2020, the analyst says it is unlikely to see any substantial revenues from the program next year. With added concerns about Bluebird Bio’s launch of Zynteglo in the E.U., Van Buren is keeping his Neutral rating on the stock.
The company said, “In October, bluebird bio announced that the European Medicines Agency approved the refined commercial drug product manufacturing specifications for ZYNTEGLO, a one-time gene therapy for patients 12 years and older with transfusion-dependent beta-thalassemia who do not have a beta0/beta0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen-matched related HSC donor is not available. With this update, apceth is in the final stages of preparing to manufacture ZYNTEGLO for commercial use. The company continues to proceed with discussions on value-based payment agreements and Qualified Treatment Center contracts and expects to treat the first commercial patient in early 2020.”
BMY = Will present data from its phase 1 liso-cel (JCAR017) – TRANSCEND NHL-001
r/r B cell non-Hodgkin lymphoma cancer trial at ASH on December 7, 2019, 8:45 a.m ET.
On November 27, 2019, Gabelli analyst Kevin Kedra initiated coverage of Bristol-Myers Squibb CVR (BMY-R) with a Buy rating and private market value estimate of $3.50. CVR holders will receive a $9.00 payout if all three of the FDA approval milestones are met, Kedra tells investors in a research note. The analyst sees an 80% probability for the ozanimod milestone, an 86% probability for JCAR017, and 68% probability for bb2121. He recommends the Bristol-Myers CVR as an attractive risk/reward opportunity with multiple near-term catalysts.
On November 25, 2019, in an interview on CNBC’s Mad Money, Bristol-Myers (BMY) CEO Giovanni Caforio said: We have a great pipeline and great people… We are positioned to succeed… Our position in cell therapy is one of the great programs we have right now… We recently announced a partnership with Fitbit (FIT)… We think we can grow through technology… The next frontier for growth is the early stage of cancer… We take a responsible approach to drug pricing, but we see a need for change in the system…Renewing our portfolio was really the rationale for acquiring Celgene (CELG).”
BPMC = The company will release data from its phase 2 Avapritinib BLU-285 – PIONEER trial for indolent and smoldering systemic mastocytosis (SM). The data will be released at ASH on December 8, 2019, at 6pm ET.
On November 6, 2019, Raymond James analyst Dane Leone upgraded Blueprint Medicines to Strong Buy from Outperform with a price target of $112, up from $106, following the company’s R&D update in NYC. In a research note to investors, Leone says the event supported key tenets to the thesis around systemic mastocytosis, or SM, and specifically provided clinical follow-up for the indolent systemic mastocytosis group that had been treated within the EXPLORER study, a discussion with a SM KOL that provided feedback on clinical experience with avapritinib, and a first look at a next gen D816V targeted drug that will submit an IND during 1H20.
On November 5, 2019, Blueprint Medicines reports Q3 EPS ($1.93), consensus ($2.18). Reports Q3 revenue $9.1M, consensus $2.59M. As of September 30 cash, cash equivalents and investments were $594.5M, as compared to $494.0M as of December 31, 2018. “As we prepare to launch our first medicine and submit multiple additional marketing applications next year, today we are unveiling our next wave of internally discovered research and clinical-stage precision therapies with the potential to deliver durable clinical benefits to additional patient populations,” said the company. “By fully leveraging our integrated research capabilities and reinvesting insights from our ongoing clinical programs, we continue to build a powerful research engine with the potential to deliver transformative treatment advances to patients as well as rapid and sustainable growth to Blueprint Medicines.”
CNST = The company will be releasing phase 2 data from its CPI-0610 and ruxolitinib myelofibrosis trial at ASH on December 9, 2019, at 11:15 AM.
On November 21, 2019, the FDA granted Constellation Pharmaceuticals orphan status for its treatment of myelofibrosis.
On November 7, 2019 Jefferies analyst Maury Raycroft reiterated a Buy rating on Constellation Pharmaceuticals and raised his price target on the shares to $40 from $17, saying that Constellation’s Phase 2 ‘0610 myelofibrosis data showed activity in 2L and 1L, which were “better than expected.” At the annual ASH meeting, the company will have more 2L and 1L data, the analyst noted. Raycroft added that, provided data at ASH continue to be in-line with the abstract cut, Constellation should have a “productive” conversation with regulators on pivotal paths and next steps.
DTIL = Will release Phase 1/2 interim data due at ASH on December 9, 2019 at 6:00 p.m. ET. The data is from its trial for PBCAR0191, relapsed or refractory B-cell precursor acute lymphoblastic leukemia and R/R non-hodgkin lymphoma.
“We are excited to share initial clinical data from Precision’s PBCAR0191 program at ASH, which we believe demonstrate the potential of our differentiated approach to the development of allogeneic CAR T therapies,” said Chris Heery, MD, Chief Medical Officer of Precision BioSciences. “These data bring the reality of a true off-the-shelf CAR T therapy a step closer for patients in need of new and improved treatment options. We remain committed to the wider goal of improving access to cellular therapies for patients with advanced NHL and ALL, and we are optimistic, based on these initial findings, that we may be able to help meet this need. While preliminary and from a limited number of patients, the safety profile, in vivo cell expansion and early evidence of clinical activity we have demonstrated at our lowest dose level with PBCAR0191 in the absence of biologic lymphodepletion is very encouraging. We look forward to sharing updated results from patients treated at Dose Levels 1 and 2 at ASH.”
On November 13, 2019, Precision BioSciences (DTIL) announced that Kevin Buehler, former CEO of Alcon Laboratories (ALC), has been appointed to its Board of Directors, which took effect following the company’s third quarter board meeting held on November 8, 2019.
On November 12, 2019, Precision BioSciences reported Q3 EPS (41c), consensus (50c). Reported Q3 revenue $4.9M, consensus $6.21M. “We reached a transformative moment for Precision BioSciences this quarter; it is very exciting to report initial data from our first clinical trial with a product candidate that leverages our unique approach to allogeneic CAR T therapy,” commented Matt Kane, Chief Executive Officer and Co-Founder of Precision BioSciences. “These data bring the reality of a true off-the-shelf CAR T therapy a step closer for patients in need of new and improved treatment options. While preliminary and from a limited number of patients, the safety profile, in vivo cell expansion and early evidence of clinical activity we have demonstrated with PBCAR0191 in the absence of biologic lymphodepletion is very encouraging and gives us confidence in the approach we have taken to allogeneic CAR T. We are looking forward to sharing updated results from patients treated at both Dose Level 1 and Dose Level 2 at ASH. The team at Precision is committed to advancing our pipeline of differentiated CAR T product candidates as rapidly as possible to bring these potentially transformative therapies to patients.”
REGN = To present phase 1 REGN1979 Follicular Lymphoma data at ASH on December 9, 2019, 4PM.
On November 27, 2019, Regeneron Pharmaceuticals announced that the New England Journal of Medicine, NEJM, published results from the randomized controlled PALM trial showing that Regeneron’s REGN-EB3 and another investigational agent provided the highest overall survival rates among four investigational treatments for Ebola virus disease. Monoclonal antibody treatments REGN-EB3, mAb114 and ZMapp and the small molecule antiviral agent remdesivir were given to a trial population of 681 patients who had Ebola during the ongoing outbreak in the Democratic Republic of the Congo, DRC. REGN-EB3, a triple-antibody cocktail, demonstrated superior efficacy compared to the ZMapp control arm across multiple measures, including the primary endpoint of mortality at day 28, 33.5% with REGN-EB3 versus 51.3 percent with ZMapp, p=0.002, and secondary endpoint of reduction of the number of days until the Ebola virus was no longer detected in the bloodstream.
On November 12, 2019, SunTrust analyst Robyn Karnauskas initiated coverage of Regeneron with a Hold rating and $360 price target. The analyst contends that the company’s base business is “baked into the stock”, noting that its “evolving oncology platform” is key to further upside. Karnauskas adds that while Regeneron’s Dupixent sales continue to grow, the possibility of biosimilar entry and Part B overhang around its flagship drug EYLEA could cap potential upside on the stock.
On November 6, 2019, Citi analyst Mohit Bansal upgraded Regeneron Pharmaceuticals to Buy from Neutral with a price target of $420, up from $340. The Libtayo data in 2020 and continuous Dupixent growth will make it easy to keep owning Regeneron shares through near-term Eylea competition, Bansal tells investors in a research note. The analyst remains bullish on Dupixent and sees $10B in peak sales versus the current run rate of $2.5B. With Libtayo’s profile “looking very similar to Keytruda,” the analyst feels “incrementally confident” and likes Regeneron’s risk/reward profile.
SGMO = The company will present data from its ST-400 beta-thalassemia phase 1/2 trial at ASH on December 9, 2019, 6pm ET.
On November 26, 2019, Sangamo Therapeutics announced the appointment of James R. Meyers, an accomplished pharmaceutical executive with three decades of commercial experience, to the Company’s Board of Directors.
“We are pleased that Jim has joined the Sangamo Board of Directors,” said Sandy Macrae, Chief Executive Officer of Sangamo. “We believe that Jim’s organizational development experience will provide great benefit to Sangamo as we continue to grow, and that his commercial, public policy, and pricing and access expertise will support our later stage programs through to registration and help get our medicines to patients in need.”
Mr. Meyers has over 30 years of commercial leadership experience in the biotechnology industry and worked at Gilead from 1996 until his retirement in February 2018. Most recently, he served as Gilead’s Executive Vice President of Worldwide Commercial Operations, where he was responsible for global commercial activities, including pricing and market access in North America, Europe, Middle East, Australia and Japan. At Gilead, he successfully led several important product launches in the HIV and hepatitis C therapeutic areas. Prior to Gilead, Mr. Meyers held positions of increasing responsibility with Zeneca Pharmaceuticals and Astra USA. He currently serves on the Board of Directors of two public companies, Arbutus Biopharma Corp. and CytomX Therapeutics, and remains an active advisor to several major biopharmaceutical companies. Mr. Meyers holds a B.S. in Economics from Boston College.
On November 19, 2019, Sangamo announced that the United Kingdom’s Medicines Healthcare Products Regulatory Agency, or MHRA, has granted authorization of the first-in-human clinical trial to evaluate a Chimeric Antigen Receptor Regulatory T Cell, or CAR-Treg, therapy. TX200 is being studied for the prevention of immune-mediated rejection following HLA-A2 mismatched kidney transplantation for end-stage renal disease, or ESRD. Sangamo expects to open the first clinical sites for the STEADFAST study in 2020.
On November 6, 2019, Sangamo reported Q3 EPS (24c), consensus (34c). Reported Q3 revenue $22M, consensus $12.16M.
“We continue to prioritize and progress our clinical development programs, as demonstrated by the accepted ASH poster presentations for our two most advanced programs, SB-525 hemophilia A gene therapy and ST-400 beta thalassemia cell therapy. Patients are currently being screened for enrollment into the clinical study evaluating ST-920, our wholly owned Fabry disease gene therapy, and we expect to enroll a first patient by the end of the year. We have also recently submitted a CTA for the clinical trial of our CAR-Treg, TX200, in mismatched renal transplantation,” said Sandy Macrae, CEO of Sangamo. “As it is important that we continue to articulate our drug development, research, and partnership strategies, we will host a Sangamo R&D day in New York on December 17, 2019. At this meeting, we will provide updates across our various genomic medicine programs, offer our perspective on the clinical data at ASH, share improvements across our technology platforms, and provide an overview of the manufacturing strategy to support our clinical and commercial supply.”
SNSS = Will report data from its Vecabrutinib (SNS-062) Advanced B-Cell Malignancies phase 1/2 trial at ASH on December 8, 2019, 6pm ET.
“We continue to advance our Phase 1b/2 trial of vecabrutinib in patients with B-cell malignancies with the goal of identifying our recommended dose and studying vecabrutinib in defined patient populations in the Phase 2 portion of the study. We are currently in the 400mg cohort as the safety and pharmacokinetic data to date support continued dose escalation,” said Dayton Misfeldt, Interim Chief Executive Officer of Sunesis. “As announced last week, we will be presenting a poster at the ASH Annual Meeting in December that provides an update on the Phase 1b/2 trial of vecabrutinib, and we look forward to sharing the new findings. We also recently presented exciting preclinical pharmacology data for our first-in-class PDK1 inhibitor, SNS-510, at the AACR-NCI-EORTC conference. The equity offering we completed in July strengthened our cash position and extends our runway through key milestones for the Company.”
XLRN = With BMY, will present phase 2 data from their luspatercept myelofibrosis trial at ASH on December 9, 2019, at 7:00 a.m. EST.
On November 8, 2019, Celgene Corporation (CELG) and Acceleron Pharma (XLRN) announced the FDA approved REBLOZYL for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions. Relblozyl was approved by the FDA for transfusion dependent betathalassemia weeks ahead of its December 4 action date, and the label is “clean and broader” than expected as it includes “all transfusion dependent” beta thalassemia patients, Piper Jaffray analyst Danielle Brill tells investors in a research note. The analyst views the early approval as a signal of the FDA’s confidence in Relblozyls’s profile. She also sees “positive readthrough” for myelodysplastic syndromes, saying today’s approval should mitigate concerns about a potential panel meeting ahead of approval in the indication. Brill reiterates an Overweight rating on Acceleron Pharma with a $73 price target.
On November 11, 2019, H.C. Wainwright analyst Edward White raised his price target for Acceleron Pharma to $88 from $68 after the FDA approved Reblozyl for the treatment of anemia in adults with beta thalassemia who require regular red blood cell transfusions. The analyst sees no surprises in the Reblozyl label and reiterates a Buy rating on the shares.
The company said, “With the FDA’s decision on the luspatercept regulatory submission for the treatment of beta-thalassemia expected in less than one month, we are close to achieving a major company milestone-the potential approval for the first Acceleron-discovered medicine. Alongside our global collaboration partner, Celgene, we are preparing for luspatercept’s potential commercial launch in the U.S. We also await the FDA’s decision on our BLA for the MDS indication in April 2020 and the European Medicines Agency’s decision on the MAA for both indications, which is expected in the second half of 2020. We continue to advance our ongoing clinical trials in first-line lower-risk MDS-, non-transfusion-dependent beta-thalassemia- and myelofibrosis-associated anemia. In addition, we are looking forward to presenting luspatercept updates on our Phase 3 MEDALIST and BELIEVE trials, as well as interim results from the ongoing Phase 2 myelofibrosis trial at the upcoming ASH meeting in December.”