Read the full announcement in PDF
Read the full announcement in PDF
Final Results Presented in Late-Breaking Session
at Society for Vascular Surgery 2019 Vascular Annual Meeting
NATIONAL HARBOR, Md., June 15, 2019 (GLOBE NEWSWIRE) — Silk Road Medical, Inc. (Nasdaq: SILK), a company focused on reducing the risk of stroke and its devastating impact, today announced positive final results for the company’s ROADSTER-2 post-marketing study evaluating real world use of the ENROUTE® Neuroprotection and Stent Systems in TransCarotid Artery Revascularization (TCAR) procedures.
In the late-breaking session at the Society for Vascular Surgery 2019 Vascular Annual Meeting (VAM), Dr. Vikram Kashyap, Chief of Vascular Surgery and Endovascular Therapy at University Hospitals Case Medical Center (Cleveland, OH) and National Co-Principal Investigator of ROADSTER-2, reported that the study demonstrated compelling patient outcomes with low stroke and combined stroke and death rates of 0.6% and 0.8%, respectively, in 632 high surgical risk patients enrolled across 42 sites. Seventy percent (70%) of patients enrolled in the study were from physicians new to TCAR.
“The results of ROADSTER-2 continue to demonstrate the safety, effectiveness and clinical advantages of TCAR, especially given that a majority of the TCAR procedures were performed by a broad group of physicians with no previous TCAR experience,” Dr. Kashyap said. “The study results highlight the short learning curve of the TCAR procedure and its remarkable consistency and reproducibility, and, I believe, will further encourage physicians to broadly adopt the TCAR procedure.”
Designed as a follow-on study to the pivotal ROADSTER trial, ROADSTER-2 is a prospective, multi-center study designed to assess the real-world usage of the ENROUTE Transcarotid Stent when used with the ENROUTE Transcarotid Neuroprotection System by physicians of varying experience with the TCAR procedure. The study met its primary endpoint of procedural success, defined as acute device and technical success in the absence of stroke, death or myocardial infarction (MI) at 30 days, at 97.9%.
Significant findings from the study showed TCAR to have low rates of 30-day major adverse events, including:
In addition, ROADSTER-2 showed lower rates of acute (1.3%) and permanent (0.5%) cranial nerve injury than is typically observed for patients receiving carotid endarterectomy (CEA), the current standard of care.
“The mounting clinical evidence base demonstrates the compelling patient benefits of TCAR, which we believe will further support physician confidence and adoption. The data from ROADSTER-2, taken together with recent updated results from the TCAR Surveillance Project, supports the case for TCAR as the standard of care in high surgical risk patients,” said Erica Rogers, Silk Road Medical’s Chief Executive Officer.
About TCAR with the ENROUTE Transcarotid Neuroprotection and Stent System
TCAR (TransCarotid Artery Revascularization) is a clinically proven procedure combining surgical principles of neuroprotection with minimally invasive endovascular techniques to treat blockages in the carotid artery at risk of causing a stroke. The ENROUTE Transcarotid Stent is intended to be used in conjunction with the ENROUTE Transcarotid Neuroprotection System (NPS) during the TCAR procedure. The ENROUTE Transcarotid NPS is a first in class device used to directly access the common carotid artery and initiate high rate temporary blood flow reversal to protect the brain from stroke while delivering and implanting the ENROUTE Transcarotid Stent.
About Silk Road Medical
Silk Road Medical, Inc. is a medical device company located in Sunnyvale, California, that is focused on reducing the risk of stroke and its devastating impact. The company has pioneered a new approach for the treatment of carotid artery disease called TransCarotid Artery Revascularization (TCAR). TCAR is a clinically proven procedure combining surgical principles of neuroprotection with minimally invasive endovascular techniques to treat blockages in the carotid artery at risk of causing a stroke.
ENROUTE is a registered trademark of Silk Road Medical, Inc.
Lynn Lewis or Carrie Mendivil
SOUTH SAN FRANCISCO, Calif., June 15, 2019 (GLOBE NEWSWIRE) — Sunesis Pharmaceuticals, Inc. (Nasdaq: SNSS) today announced the presentation of results from the Company’s Phase 1b/2 clinical trial of its non-covalent BTK inhibitor vecabrutinib in adults with relapsed/refractory chronic lymphocytic leukemia (CLL) and other B-cell malignancies. The results are being presented today, June 15, from 5:30-7:00 p.m. CET in a poster session titled “Chronic lymphocytic leukemia and related disorders – Clinical” at the 24th Congress of the European Hematology Association (EHA) in Amsterdam. The poster, titled “Preliminary Results of a Phase 1b/2 Dose-Escalation and Cohort-Expansion Study of the Noncovalent, Reversible Bruton’s Tyrosine Kinase Inhibitor (BTKi) Vecabrutinib in B-Cell Malignancies,” Abstract No. PS1148, is available at www.sunesis.com.
“We are encouraged by the data presented today demonstrating vecabrutinib’s well-tolerated safety profile and evidence of clinical activity in CLL and other B-cell malignances,” said Dayton Misfeldt, Interim Chief Executive Officer of Sunesis. “Importantly, vecabrutinib’s median steady-state trough concentrations continue to increase with dose and are approaching levels expected to provide consistent BTK inhibition and greater clinical activity. We are currently dosing patients in the 200mg cohort and momentum in the trial continues as reflected by the robust pace of enrollment we’ve seen this year. We look forward to sharing data from the additional cohorts as we complete the Phase 1b and proceed to Phase 2 later this year.”
Preliminary data reported today were available from 23 patients treated in the trial thus far. These included 19 CLL patients, two mantle cell lymphoma (MCL) patients, and two Waldenstrom Macroglobulinemia (WM) patients. Patients had received an average of 4 lines of prior therapy, and all patients had progressed on prior BTKi therapy. 61% of CLL patients enrolled had a BTK C481 mutation as of the data cutoff for the poster.
The poster builds vecabrutinib’s profile in four key areas:
About Sunesis Pharmaceuticals
Sunesis is a biopharmaceutical company developing new targeted therapeutics for the treatment of hematologic and solid cancers. Sunesis has built an experienced drug development organization committed to improving the lives of people with cancer. The Company is focused on advancing its novel kinase inhibitor pipeline, with an emphasis on its oral non-covalent BTK inhibitor vecabrutinib. Vecabrutinib is currently being evaluated in a Phase 1b/2 study in adults with chronic lymphocytic leukemia and other B-cell malignancies that have progressed after prior therapies. The Company’s proprietary PDK1 inhibitor SNS-510 is in preclinical development. PDK1 is a master kinase that activates other kinases important to cell growth and survival including members of the AKT, PKC, RSK, and SGK families. Sunesis is exploring strategic alternatives for vosaroxin, a late-stage investigational product for relapsed or refractory AML. Sunesis also has an interest in the pan-RAF inhibitor TAK-580 which is licensed to Takeda. TAK-580 is in a clinical trial for pediatric low-grade glioma.
For additional information on Sunesis, please visit www.sunesis.com.
SUNESIS and the logos are trademarks of Sunesis Pharmaceuticals, Inc.
This press release contains forward-looking statements, including statements related to Sunesis’ continued development of vecabrutinib (SNS-062), including the timing of the Phase 1b/2 trial of vecabrutinib,the therapeutic potential of vecabrutinib, and the further development and potential of its kinase inhibitor pipeline. Words such as “believe”, “expect,” “likely,” “look forward” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Sunesis’ current expectations. Forward-looking statements involve risks and uncertainties. Sunesis’ actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, the risk related to the timing or conduct of Sunesis’ clinical trials, including the vecabrutinib Phase 1b/2 trial, the risk that Sunesis’ clinical or preclinical studies for vecabrutinib, SNS-510 or other product candidate may not demonstrate safety or efficacy or lead to regulatory approval, the risk that data to date and trends may not be predictive of future data or results, that Sunesis’ development activities for vecabrutinib or SNS-510 could be otherwise halted or significantly delayed for various reasons, that Sunesis may not be able to receive regulatory approval of vecabrutinib, or SNS-510 in the U.S. or Europe, and risks related to Sunesis’ ability to raise the capital that it believes to be accessible and is required to fully finance the development and commercialization of vecabrutinib, SNS-510 and other product candidates. These and other risk factors are discussed under “Risk Factors” and elsewhere in Sunesis’ Quarterly Report on Form 10-Q for the quarter ended March 31, 2019 and Sunesis’ other filings with the Securities and Exchange Commission. Sunesis expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein reflect any change in Sunesis’ expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.
|Investor and Media Inquiries:
Sunesis Pharmaceuticals Inc.
IND-enabling activities initiated for EDIT-301, a potentially best-in-class experimental medicine for the treatment of sickle cell disease and beta-thalassemia
CAMBRIDGE, Mass., June 15, 2019 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced results from a follow-up study to assess two different CRISPR genome editing strategies, one targeting the BCL11A erythroid enhancer (BC11Ae) and one targeting the beta-globin locus, for the treatment of sickle cell disease and beta-thalassemia. The Company reported the data at the 24th Congress of the European Hematology Association in Amsterdam.
In this study, NBSGW mice received an infusion of human CD34+ cells which had been edited either at the BCL11Ae or at the beta-globin locus. In vivo-derived erythroid cells from BCL11Ae-edited CD34+ hematopoietic stem/progenitor cells had reduced total indels and increased non-productive indels as compared to other tested lineages, a phenomenon not observed with beta-globin locus editing. Additionally, further optimization of nuclease and guide RNA combinations led to fetal hemoglobin expression of approximately 40 percent in the beta-globin locus-edited erythroid cells. Based on the data, Editas Medicine has initiated IND-enabling activities for EDIT-301, an experimental CRISPR medicine designed to durably treat sickle cell disease and beta-thalassemia by editing the beta-globin locus.
“We are encouraged by these pre-clinical results demonstrating cells edited at the beta-globin locus repopulated all lineages of the blood system including, importantly, the red blood cell precursors and the high percentage of fetal hemoglobin expression. Editing at this site continues to meet our preclinical goals for making a medicine including robust, long-term induction of fetal hemoglobin and maintenance of normal hematopoietic stem/progenitor cell function,” said Charles Albright, Ph.D., Chief Scientific Officer, Editas Medicine. “Our program is on track towards the clinic, and we have started our IND-enabling activities as we look to develop a best-in-class medicine for the treatment of sickle cell disease and beta-thalassemia.”
About Editas Medicine
As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit www.editasmedicine.com.
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.
CRESTWOOD, Ky. and WALTHAM Mass., June 15, 2019 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals Inc. (Nasdaq:APLS), a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, today announced updated data from its Phase 2 PLAUDIT study of APL-2 in patients with autoimmune hemolytic anemia (AIHA), including cold agglutinin disease (CAD) and warm antibody autoimmune hemolytic anemia (wAIHA). Data from the PLAUDIT trial will be presented in an oral presentation today at the 24th Annual Congress of the European Hematology Association (EHA), held in Amsterdam, the Netherlands.
In the ongoing PLAUDIT study, 13 patients with CAD have been enrolled to receive subcutaneous APL-2 treatment, of which 10 patients have been on APL-2 for at least 168 days. The trial has also enrolled 11 patients with wAIHA, 8 of which were Direct Antiglobulin Test (DAT) C3+ (C3+ wAIHA); 5 of the C3+ wAIHA patients have been on APL-2 for at least 168 days.
“Patients with autoimmune hemolytic anemias on APL-2 have shown improvement both in important hematologic measures and in quality of life scores, particularly in patients with CAD,” said Federico Grossi, CMO, Apellis. “These data provide a clear path to move forward with a Phase 3 trial of APL-2 in patients with CAD, which we anticipate commencing in early 2020. We have also seen hematologic improvements in patients with wAIHA receiving treatment with APL-2 when there is significant involvement of the complement pathway. Apellis will continue to evaluate the potential of APL-2 for patients with C3+ wAIHA.”
Data will be presented by Bruno Fattizzo, Consultant Hematologist at Fondazione IRCCS Ca’ Granda Policlinico Hospital, Milan, Italy. The presentation will be made available through the “Our Scientific Publications & Presentations” page of the Apellis website at: https://apellis.com/publication-presentation.html.
“This trial demonstrates that APL-2 has the potential to significantly help patients with autoimmune hemolytic anemias when there is complement involvement,” said Dr. Fattizzo. “APL-2 represents a promising potential therapy for these patients, the majority of whom have no access to approved treatments.”
Oral Presentation: Inhibition of C3 with APL-2 controls haemolysis and increases haemoglobin levels in subjects with autoimmune haemolytic anaemia (AIHA)
Session Name: Hemolytic anemias: From diagnostics to treatment
Date: Saturday, June 15
Presentation Time: 4:00 – 4:15 PM CEST
Location: RAI Amsterdam at Europaplein 24, Amsterdam, the Netherlands, Hall G102
Cold Agglutinin Disease (CAD)
Of the 10 patients who reached Day 168:
Two heavily transfusion dependent patients did not respond to APL-2 and left the study at Day 56 and 108 respectively. The remaining transfusion dependent patients (n=4) did not require any transfusions during maintenance treatment with APL-2 (i.e. after one week of starting APL-2 therapy). One previously non-transfusion dependent patient received an on-study transfusion prior to APL-2 steady-state (within one week of commencement of dosing). One patient is still participating in the study and has not yet reached Day 168.
C3+ Warm Antibody Autoimmune Hemolytic Anemia (C3+ wAIHA)
Of the 5 patients with C3+ wAIHA who reached Day 168:
Two of the eight enrolled C3+ wAIHA patients left the study due to lack of response, and one of the eight enrolled C3+ wAIHA subjects is still participating in the study and has not yet reached Day 168. The three enrolled patients who were not DAT C3+ did not show a meaningful response and two have left the study.
In both the CAD and C3+ wAIHA populations, APL-2 was generally well tolerated; no serious adverse events related to APL-2 were reported in the PLAUDIT trial.
Cold Agglutinin Disease (CAD)
Cold Agglutinin Disease (CAD) is a severe, chronic rare autoimmune disorder caused by pathogenic Immunoglobulin M (IgM) antibodies that react with red blood cells (RBCs) at temperatures below 30oC and leads to agglutination of the RBCs. Agglutinated RBCs activate a part of the body’s immune system called the complement system leading to destruction of the RBCs. The disease is often characterized by chronic anemia, severe fatigue, and an increased risk of life-threatening events such as stroke. There are an estimated 10,000 CAD patients across the United States and Europe. There are currently no approved therapies for CAD.
Warm autoimmune hemolytic anemia (wAIHA)
Warm autoimmune hemolytic anemia (wAIHA) is a rare autoimmune disorder caused by pathogenic Immunoglobulin G (IgG) antibodies that react with RBCs and can activate the complement system leading to the premature destruction of RBCs at normal body temperature. The disease is often characterized by profound, and potentially life-threatening anemia and other acute complications, including severe and life-threatening hemolysis, severe weakness, enlarged spleen or liver, rapid heart rate, chest pain, heart failure and fainting. There are estimated to be more than 30,000 wAIHA patients across the United States and Europe. C3+ wAIHA has been estimated to represent as much as two thirds of the total wAIHA population. There are currently no approved treatments for wAIHA.
APL-2, an investigational drug, is designed to inhibit the complement cascade centrally at C3 and may have the potential to treat a wide range of complement-mediated diseases more effectively than is possible with partial inhibitors of complement. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation (classical, lectin, and alternative). Apellis is currently evaluating APL-2 in clinical studies in patients with geographic atrophy, in patients with paroxysmal nocturnal hemoglobinuria (PNH) who are being treated with eculizumab or who are naïve to complement inhibitor treatment, in patients with AIHA and in patients with C3G and other glomerular diseases. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.
About APL-2 in Hematologic Diseases
Apellis is currently evaluating APL-2 in PEGASUS, a Phase 3 trial for patients with PNH as well as in two Phase 1b trials (PHAROAH and PADDOCK) for systemic administration. Previously reported interim data from these Phase 1b trials showed improvements in lactate dehydrogenase and hemoglobin levels in patients who are suboptimal responders to eculizumab and untreated patients, respectively. Apellis is also testing APL-2 in a Phase 2 open-label trial assessing the safety, tolerability, efficacy, and PK of multiple subcutaneous doses of APL-2 administered daily in patients with wAIHA or CAD. In this trial to date, APL-2 has shown the potential to improve hemoglobin, reticulocytes, bilirubin and lactate dehydrogenase levels.
Apellis Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds for the treatment of a broad range of life-threatening or debilitating autoimmune diseases based upon complement immunotherapy through the inhibition of the complement system at the level of C3. Apellis is the first company to advance chronic therapy with a C3 inhibitor into clinical trials. For additional information about Apellis and APL-2, please visit http://www.apellis.com.
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the implications of preliminary clinical data. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether the Company’s clinical trials will be fully enrolled and completed when anticipated; whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; whether results obtained in preclinical studies and clinical trials will be indicative of results that will be generated in future clinical trials; whether APL-2 will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of such clinical trials will warrant regulatory submissions and whether APL-2 will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, wAIHA or any other indication; whether, if Apellis’ products receive approval, they will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 7, 2019 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
1D. Cella et al. “Combining Anchor and Distribution-Based Methods to Derive Minimal Clinically Important Differences on the Functional Assessment of Cancer Therapy (FACT) Anemia and Fatigue Scales” Journal of Pain and Symptom Management Vol. 24 No. 6 December 2002
CONTACT: Media Contact: Liza Sullivan firstname.lastname@example.org 617.482.0042 (office) 917.981.7494 (mobile) Investor Contact: Alex Kane email@example.com 212.301.7218 (office) 929.400.2691 (mobile)
— 5F9 Well-Tolerated in Combination with Rituximab —
— Durable Responses Observed in DLBCL and Indolent Lymphoma; Median Duration of Response Not Yet Reached —
— Overall Response Rate (ORR) of 36% Observed in DLBCL and ORR of 61% Observed in Indolent Lymphoma —
— FDA Feedback Suggests Single-Arm Pivotal Trial May Support Registration of 5F9 in Combination with Rituximab in Heavily Pre-Treated DLBCL Patients, Including Those Ineligible for CAR-T Therapy —
— Forty Seven to Host Conference Call and Webcast at 8:00 am ET on Monday, June 17, 2019 —
MENLO PARK, Calif., June 15, 2019 (GLOBE NEWSWIRE) — Forty Seven, Inc., a clinical-stage, immuno-oncology company focused on developing therapies to activate macrophages in the fight against cancer, today announced updated data from its ongoing Phase 1b/2 clinical trial evaluating 5F9 in combination with rituximab for the treatment of relapsed/refractory non-Hodgkin’s lymphoma (r/r NHL), including diffuse large B-cell lymphoma (DLBCL) and indolent lymphoma. The data will be presented in an oral session at the 24th Congress of the European Hematology Association (EHA) in Amsterdam, Netherlands. Also at EHA, Forty Seven will present data from its ongoing Phase 1b clinical trial evaluating 5F9 as a monotherapy and in combination with azacitidine for the treatment of myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), which were previously presented at the 2019 American Society for Clinical Oncology (ASCO) Annual Meeting.
“Despite advancements in the treatment of newly-diagnosed NHL, there are very limited options for patients with advanced forms of the disease, who have failed multiple lines of treatment or are ineligible for existing therapies due to advanced age, numerous co-morbidities, and/or the diagnosis of aggressive, proliferating disease,” said Justin Kline, M.D., University of Chicago, Department of Medicine, an investigator for the clinical trial. “In treating these patients, our goal is to achieve durable responses and stave off the rapid disease progression that can limit overall survival to a matter of months. As such, I am encouraged by the updated data for 5F9 in combination with rituximab, which showed consistent clinical benefit across a range of patient populations — including patients who are heavily pre-treated, ineligible for CAR-T therapy or suffering from primary refractory disease — as well as durable responses in both DLBCL and FL. I am eager to continue evaluating this combination as a well-tolerated, chemotherapy-free regimen, with the potential to induce long-term remissions even in the sickest patients.”
“Today’s announcement marks a significant milestone in our development of 5F9 for the treatment of r/r NHL,” said Chris Takimoto, M.D., Ph.D., F.A.C.P., Chief Medical Officer of Forty Seven, Inc. “The design of our Phase 1b/2 clinical trial allowed us to continue to explore the clinical benefit of 5F9 in combination with rituximab in patients with DLBCL and indolent lymphoma, while also expanding into a subset of older, sicker DLBCL patients who have been deemed ineligible for CAR-T therapy. We are particularly gratified to see meaningful activity in this newly-defined population, which has never before been evaluated in clinical trials, and for whom there are few, if any, effective treatment options available. Importantly, following recent interactions with the U.S. Food and Drug Administration (FDA), we believe we may be able to pursue approval in a heavily pre-treated population, including DLBCL patients who have failed at least two prior lines of therapy or who are ineligible for CAR-T therapy, with a single-arm pivotal study serving as the basis for our BLA filing. These developments bring us closer to achieving our vision of establishing 5F9 as a cornerstone immunotherapy for the treatment of a broad range of cancers.”
Data from the Ongoing Phase 1b/2 Clinical Trial in r/r NHL
Forty Seven’s Phase 1b/2 trial, which is being funded in part by the Leukemia and Lymphoma Society (LLS) through the Therapy Acceleration Program® (TAP), is designed to evaluate 5F9 in combination with rituximab in patients with r/r B-cell NHL, who have failed standard-of-care therapies. All patients received a 1 mg/kg priming dose of 5F9 to mitigate on-target anemia. Patients in the Phase 1b portion of the trial were treated with 5F9 maintenance doses of 10 to 45 mg/kg, and patients in the Phase 2 portion of the trial were treated with 5F9 doses of either 30 or 45 mg/kg. All patients were also administered full doses of rituximab.
As of the data cutoff of May 2019, 115 patients had been treated in the Phase 1b/2 trial, including 70 patients with DLBCL, 41 patients with follicular lymphoma (FL) and four patients with marginal zone lymphoma (MZL). The median number of prior therapies across all patients was three (range one to 10), and 85% of all patients were refractory to a prior rituximab-containing regimen, with 59% of DLBCL patients having primary refractory disease. Additionally, 42 of the 47 DLBCL patients enrolled in the Phase 2 portion of the trial were ineligible for CAR-T therapy (89%).
As of the data cutoff, 5F9 was well-tolerated in combination with rituximab. Adverse events (AEs) were consistent with prior clinical experience. Most AEs were Grade 1 or Grade 2, and the most commonly-reported AEs were expected CD47-mechanism-based effects on red blood cells, which led to a temporary and reversible anemia, and infusion-site reactions. No autoimmune-related AEs were observed, nor were any significant late safety signals observed in patients treated with 5F9 for up to 24 months. No maximum tolerated dose was reached with up to 45 mg/kg of 5F9 dosing. Eight out of 115 patients discontinued treatment due to an AE (7%).
Clinical Activity Data
As of the data cutoff, 97 patients were evaluable for response assessment, including 21 relapsed/refractory DLBCL patients who were treated in the Phase 1b portion of the study, 38 DLBCL patients who were treated in the Phase 2 portion of the study and 38 indolent lymphoma patients (35 patients with FL and three patients with MZL).
|Best Overall Response||Phase 1b
N = 21 (%)
N = 38 (%)
|≥ 3 Prior Lines of Therapy
N = 39 (%)
|Primary refractory disease or
relapsed/refractory to ≥ 2 prior
lines of therapy
relapsed/refractory to ≥ 2
prior lines of therapy and
ineligible for CAR-T
|Subgroup analysis of
combined Phase 1b and
Phase 2 Data
|ORR||10 (48)||11 (29)||15 (38)|
|CR||7 (33)||2 (5)||7 (18)|
|PR||3 (14)||9 (24)||8 (20)|
|SD||4 (19)||3 (8)||4 (10)|
Among patients treated in the Phase 1b portion of the trial, the median duration of response has not been reached, with a median follow-up of over 13.8 months. This includes one patient who has remained in a durable complete response (CR) for more than 24 months.
|Phase 1b + 2
FL N = 35; MZL N = 3 (%)
|Relapsed/refractory to ≥ 2 prior lines of therapy|
Among patients treated in the Phase 1b portion of the trial, the median duration of response has not been reached with a median follow-up of over 21 months. This includes the patient who has remained in a durable CR for more than 28 months.
Additionally, 5F9 tumor penetrance was evaluated at 30 and 45 mg/kg as a key pharmacodynamic endpoint. Data show that the 30 mg/kg maintenance dose of 5F9 saturated the tumor microenvironment similarly to 45 mg/kg, with similar efficacy. As a result, a 30 mg/kg maintenance dose of 5F9 was selected as the recommended dose for use in future clinical studies.
Clinical Development Plans for 5F9 in r/r NHL
Based on feedback from a Type C meeting with the FDA in May 2019, Forty Seven believes that data from a single arm pivotal study evaluating ORR and durability may be sufficient to support the registration of 5F9 in combination with rituximab in patients with r/r DLBCL who have failed at least two prior lines of therapy, including those who have been deemed CAR-T ineligible. The Company is currently finalizing the operational components of the proposed registrational study, including details on trial design and chemistry, manufacturing and controls (CMC), and will provide a detailed update in the second half of 2019.
In parallel, the Company will continue ongoing efforts to evaluate 5F9 as part of additional combination regimens for patients with DLBCL, including in patients in earlier lines of treatment. This includes the planned triplet regimen of 5F9, rituximab and atezolizumab, which Forty Seven is evaluating in collaboration with Genentech, and the planned triplet regimen of 5F9, rituximab and acalabrutinib, which Forty Seven is evaluating in collaboration with Acerta Pharma, in addition to potential studies of 5F9 in combination with other targeted antibodies.
Forty Seven will also evaluate opportunities to advance 5F9 in combination with rituximab for patients with indolent lymphoma.
Data from the Ongoing Phase 1b Clinical Trial in MDS and AML
As Forty Seven previously presented at the 2019 ASCO Annual Meeting in June 2019, data from the ongoing Phase 1b clinical trial of 5F9 in MDS and AML showed an ORR of 100% and a CR rate of 55% in higher-risk MDS patients treated with 5F9 in combination with azacitidine and an ORR of 64% and a CR/complete response with incomplete blood count recovery (CRi) rate of 50% in untreated AML patients treated with 5F9 in combination with azacitidine. In addition, the combination was well-tolerated, with no evidence of increased toxicities compared to azacitidine alone.
Based on the favorable safety profile and encouraging clinical activity observed in this Phase 1b clinical trial, expansion cohorts have been initiated in patients with both higher-risk MDS and untreated AML with 5F9 in combination with azacitidine. In addition, based on feedback from a Type B meeting with the FDA in May 2019, Forty Seven believes that data from a single pivotal study may be sufficient to support the registration of 5F9 in combination with azacitidine in patients with untreated, higher-risk MDS. The Company is finalizing the details of the proposed registrational study, and will provide a detailed update in the second half of 2019.
Investor Conference Call and Webcast Information
Forty Seven will host a live conference call and webcast on Monday, June 17, 2019 at 8:00 a.m. ET to review the clinical data presented at EHA. The conference call may be accessed by (866) 953-0780 (domestic) or (630) 652-5854 (international), and by referring to conference ID 3236239. A webcast of the conference call will also be in the Investors section of the Forty Seven website at www.fortyseveninc.com. The archived webcast will be available approximately two hours after the conference call and will be available for 30 days following the call.
5F9 is a monoclonal antibody against CD47 that is designed to interfere with recognition of CD47 by the SIRPα receptor on macrophages, thus blocking the “don’t eat me” signal used by cancer cells to avoid being ingested by macrophages. Forty Seven, Inc. is initially developing 5F9, an investigational medicine, for the treatment of patients with solid tumors, acute myeloid leukemia, non-Hodgkin’s lymphoma and colorectal cancer. 5F9 has been granted Fast Track designation by the U.S. Food and Drug Administration for the treatment of relapsed or refractory diffuse large B-cell lymphoma and follicular lymphoma, two forms of B-cell non-Hodgkin’s lymphoma, and Orphan Drug designation by the U.S. Food and Drug Administration and European Medicines Agency for the treatment of acute myeloid leukemia.
About Forty Seven Inc.
Forty Seven, Inc. is a clinical-stage immuno-oncology company that is developing therapies targeting cancer immune evasion pathways based on technology licensed from Stanford University. Forty Seven’s lead program, 5F9, is a monoclonal antibody against the CD47 receptor, a “don’t eat me” signal that cancer cells commandeer to avoid being ingested by macrophages. This antibody is currently being evaluated in multiple clinical studies in patients with solid tumors, myelodysplastic syndrome, acute myeloid leukemia, non-Hodgkin’s lymphoma, ovarian cancer and colorectal carcinoma.
Forward Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “may,” “will,” “expect,” “anticipate,” “estimate,” “intend,” “potential,” “believe” and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. These statements include those related to the overall advancement of 5F9 in clinical trials; the potential of 5F9 as a monotherapy and in combination with azacitidine for the treatment of MDS and AML; the potential of 5F9 as a tolerable treatment option for patients with MDS and AML as a monotherapy and in combination; the sufficiency of the Company’s single arm pivotal trial design and results from such trial for FDA registration of 5F9 in combination with azacitidine for the treatment of patients with untreated higher-risk MDS; Forty Seven’s plans to continue development of 5F9 as a monotherapy and in combination with azacitidine; the potential of 5F9 in combination with rituximab for the treatment of r/r NHL, including DLBCL, and indolent lymphoma; the sufficiency of the Company’s single arm pivotal trial design evaluating ORR and durability and results from such trial for FDA registration of 5F9 in combination with rituximab in patients with r/r DLBCL; and Forty Seven’s plans to continue development of 5F9 in triplet combination with rituximab and atezolizumab and acalabrutinib, as well as in combination with other targeted antibodies.
Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. The product candidates that Forty Seven develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all. In addition, clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release. Such product candidates may not be beneficial to patients or successfully commercialized. The failure to meet expectations with respect to any of the foregoing matters may have a negative effect on Forty Seven’s stock price. Additional information concerning these and other risk factors affecting Forty Seven’s business can be found in Forty Seven’s periodic filings with the Securities and Exchange Commission at www.sec.gov. These forward-looking statements are not guarantees of future performance and speak only as of the date hereof, and, except as required by law, Forty Seven disclaims any obligation to update these forward-looking statements to reflect future events or circumstances.
For more information please visit www.fortyseveninc.com or contact firstname.lastname@example.org.
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WAYNE, Pa., June 14, 2019 (GLOBE NEWSWIRE) — Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a physician-led biopharmaceutical company focused on immuno-inflammatory and dermatological diseases, today announced that management will present a company overview at the 2019 JMP Securities Life Sciences Conference on Thursday, June 20, 2019 at 11:30 AM ET at the St. Regis Hotel in New York, New York.
A live audio webcast of the presentation may be accessed through the Company’s web site, www.aclaristx.com, on the ‘Events’ section. An archived version of the presentation will be available for 30 days.
About Aclaris Therapeutics, Inc.
Aclaris Therapeutics, Inc. is a physician-led biopharmaceutical company committed to addressing the needs of people with immuno-inflammatory and dermatological diseases who lack satisfactory treatment options. The company’s diverse and multi-stage portfolio includes two FDA-approved medicines, one late-stage investigational medicine, and a pipeline powered by a robust R&D engine exploring protein kinase regulation. Aclaris Therapeutics’ active development programs focus on areas where significant treatment gaps exist, such as common warts, alopecia areata, and vitiligo. For additional information, please visit www.aclaristx.com and follow Aclaris on LinkedIn or Twitter @aclaristx.
Michael Tung, M.D.
Senior Vice President
Corporate Strategy/Investor Relations
[Updated Release to Correct Date of Presentation]
BETHLEHEM, Pa., June 14, 2019 (GLOBE NEWSWIRE) — OraSure Technologies, Inc. (NASDAQ: OSUR), a leader in point-of-care diagnostic tests and specimen collection devices, today announced that Roberto Cuca, Chief Financial Officer, will speak to the investment community at the Raymond James Life Sciences and MedTech Conference. The conference will be simultaneously webcast over the Internet.
Mr. Cuca is scheduled to speak on Wednesday, June 19, 2019, at approximately 3:35 PM Eastern Time (12:35 PM Pacific Time). Interested investors can access the live webcast of the presentation by going to OraSure Technologies’ web site, www.orasure.com and clicking on the Investors link. A replay of the webcast will be available on OraSure Technologies’ web site for seven days. Alternatively, you can access the live webcast of the presentation via the following link: http://wsw.com/webcast/rj115/osur/
About OraSure Technologies
OraSure Technologies is empowering the global community to improve health and wellness by providing access to accurate essential information. OraSure is a leader in the development, manufacture and distribution of point-of-care diagnostic tests, molecular collection devices and other technologies designed to detect or diagnose critical medical conditions. Its first-to-market, innovative products include rapid tests for the detection of antibodies to HIV and Hepatitis C (HCV) on the OraQuick® platform, sample self-collection and stabilization products for molecular applications, and oral fluid laboratory tests for detecting various drugs of abuse. Together with its wholly-owned subsidiaries (DNA Genotek, CoreBiome and Novosanis), OraSure provides its customers with value-added, end-to-end solutions that encompass tools, diagnostics and services. OraSure’s portfolio of products is sold globally to various clinical laboratories, hospitals, clinics, community-based organizations and other public health organizations, research institutions, distributors, government agencies, physicians’ offices, commercial and industrial entities and consumers. For more information on OraSure Technologies, please visit www.orasure.com.
|Roberto Cuca||Jeanne Mell|
|Chief Financial Officer||VP Corporate Communications|
Final regulatory framework positions Emerald for further growth
VANCOUVER, British Columbia, June 14, 2019 (GLOBE NEWSWIRE) — Emerald Health Therapeutics, Inc. (“Emerald”) (TSXV: EMH; OTCQX: EMHTF) is pleased that today the Government of Canada released the final regulatory framework for ingestibles and edibles.
“The clarity of the final regulatory framework provides is important for Emerald in bringing our new product categories such as vape pens, ingestibles and edibles to market,” said Allan Rewak, VP Communications and Stakeholder Relations. “We applaud the government for providing this certainty today.”
The regulatory framework announced by Health Canada prohibits product forms that could be appealing to children and youth, combine alcohol or nicotine with cannabis and restrict production of infused cannabis products to dedicated licenced cannabis facilities approved by Health Canada.
“We see today’s announcement as a positive step forward and a validation of our plan,” said Dr. Avtar Dhillon, Emerald’s Executive Chairman and President. “In anticipation of the restrictions placed on production for infused cannabis products, we worked with our strategic partner the Factors Group to fully segment their Kelowna plant into a separate processing facility. As a result of this early action, I am pleased to say that the Factors Group is working to complete construction in the near term, with the final evidence package to be submitted to Health Canada shortly thereafter.”
Emerald’s partnership with the Factors Group is anticipated to greatly assist the Company in overcoming processing bottlenecks in the Canadian marketplace for extraction and ingestible processing due to the restrictions on allowable production facilities. Once fully operational, the Factors Group will be capable of converting up to 1 million kg of biomass into value added products, beginning with soft-gels.
The regulations announced today will come into force on October 17th, 2019, which when combined with the statutory 60-day notification period for new product forms, will allow Emerald to introduce new ingestible product categories to market in the December – January timeframe.
About Emerald Health Therapeutics
Emerald Health Therapeutics, Inc. is a Canadian licensed producer of cannabis. Its 50%-owned Pure Sunfarms joint venture in BC is licensed and fully planted in the first of its two 1.1 million square foot greenhouses. The capacity of each greenhouse is estimated to exceed 75,000 kg of cannabis annually. Emerald’s Verdélite operation in Saint Eustache, Québec is completing the build-out of its 88,000 square foot indoor cultivation facility and is scaling up production. Emerald has contracted for approximately 1,200 acres of hemp in 2019 to 2022 with the objective of extracting low-cost cannabidiol. Emerald has secured exclusive strategic partnerships for large scale extraction and softgel encapsulation, as well as for proprietary technology to enhance cannabinoid bioavailability. Its team is highly experienced in life sciences, product development, large-scale agri-business, and marketing, and is focused on developing proprietary, value-added cannabis products for medical and adult-use customers.
Emerald is part of the Emerald Group, which represents a broad array of companies focused on developing pharmaceutical, botanical, and nutraceutical products aimed at providing wellness and medical benefits by interacting with the human body’s endocannabinoid system.
Please visit www.emeraldhealth.ca for more information or contact:
Rob Hill, Chief Financial Officer
(800) 757 3536 Ext. # 5
Emerald Media Relations
Emerald Investor Relations
(800) 757 3536 Ext. #5
Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.
Cautionary Note Regarding Forward-Looking Statements: Certain statements made in this press release that are not historical facts are forward-looking statements and are subject to important risks, uncertainties and assumptions, both general and specific, which give rise to the possibility that actual results or events could differ materially from our expectations expressed in or implied by such forward-looking statements. Such statements include projected job creation figures at our operating facilities; production and processing capacity of various facilities; expansion of facilities; and anticipated production costs.
We cannot guarantee that any forward-looking statement will materialize, and readers are cautioned not to place undue reliance on these forward-looking statements. These forward-looking statements involve risks and uncertainties related to, among other things, failure to obtain regulatory approvals; failure to obtain necessary financing; results of production and sale activities; results of scientific research; regulatory changes; changes in prices and costs of inputs; demand for labour; demand for products; as well as the risk factors described in the Company’s annual information form and other regulatory filings. The forward-looking statements contained in this press release represent our expectations as of the date hereof. Forward-looking statements are presented for the purpose of providing information about management’s current expectations and plans and allowing investors and others to obtain a better understanding of our anticipated operating environment. Readers are cautioned that such information may not be appropriate for other purposes. The Company undertakes no obligations to update or revise such statements to reflect new circumstances or unanticipated events as they occur, unless required by applicable law.
NEW YORK and MELBOURNE, Australia, June 14, 2019 (GLOBE NEWSWIRE) — Mesoblast Limited (NASDAQ: MESO; ASX: MSB), global leader in cellular medicines for inflammatory diseases, today announced that it will host a virtual symposium on Monday, June 17 to discuss Mesoblast’s product candidate, remestemcel-L, in steroid refractory acute graft versus host disease (aGVHD) in children, and to include Dr. Susan E. Prockop, a key opinion leader with significant expertise and experience in treating pediatric patients with this life threatening disease. Dr. Prockop is a pediatric oncologist specializing in bone marrow and stem cell transplantation at Memorial Sloan Kettering Cancer Center in New York. She also served as an investigator in Mesoblast’s recent remestemecel-L study in pediatric patients with steroid refractory aGVHD.
The webcast event will feature a presentation by Dr. Prockop who will address the current management and treatment options for patients with aGVHD. She will also discuss her experiences with remestemcel-L in patients with steroid refractory aGVHD. Mesoblast senior management will discuss the potential market and commercial opportunities for remestemcel-L should it receive approval from the US Food and Drug Administration (FDA). The Company has agreement from the FDA for submission of a rolling Biologics License Application (BLA) for remestemcel-L in the treatment of aGVHD in children, for which the Company filed the first component of in May of 2019.
Interested parties may access the event on Monday, June 17 at 9:00am EDT (11:00 pm AEST) by dialing (866) 939-3921 (US); 1 800 507 265 (Australia) or 0808 238 9578 (UK) using the confirmation code 48733893.
About Steroid-refractory Acute Graft Versus Host Disease
Steroid-refractory acute graft versus host disease (aGVHD) is a life-threatening complication of a bone marrow transplant in patients primarily being treated for blood cancers. There are more than 30,000 allogeneic bone marrow transplants performed globally, with 20-25% occurring in children. Currently, there are no approved products for aGVHD in children outside Japan, where Mesoblast licensee JCR Pharmaceuticals markets TEMCELL®1 HS Inj. for both children and adults with aGVHD.
Mesoblast Limited (ASX:MSB; Nasdaq:MESO) has leveraged its proprietary technology platform to establish a broad portfolio of late-stage allogeneic (off-the-shelf) product candidates with three product candidates in Phase 3 trials – acute graft versus host disease, chronic heart failure and chronic low back pain due to degenerative disc disease. Through a proprietary process, Mesoblast selects rare mesenchymal lineage precursor and stem cells from the bone marrow of healthy adults and creates master cell banks, which can be industrially expanded to produce thousands of doses from each donor without the need for tissue matching. Mesoblast has facilities in Melbourne, New York, Singapore and Texas and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). www.mesoblast.com
1. TEMCELL® HS Inj. is a registered trademark of JCR Pharmaceuticals Co. Ltd.
This announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about the timing, progress and results of Mesoblast’s preclinical and clinical studies; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies; the timing or likelihood of regulatory filings and approvals; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.
For further information, please contact:
T: +61 3 9639 6036
T: +1 212 880 2060