On May 12,, 2021 Epizyme, Inc. (Nasdaq: EPZM), a fully integrated, commercial-stage biopharmaceutical company developing and delivering novel epigenetic therapies, reported that new preclinical and clinical data will be presented at upcoming medical meetings in June (Press release, Epizyme, MAY 12, 2021, View Source [SID1234579825]).

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"We look forward to sharing our preclinical data at EHA (Free EHA Whitepaper) that provide the therapeutic rationale for targeting SETD2 in multiple myeloma and are so pleased that it has been chosen for an oral presentation. These data are the basis for moving our SETD2 inhibitor program forward and, as we have previously reported, we remain on track for our planned IND submission in mid-2021 and plan to initiate a first-in-human clinical trial by year end," said Dr. Jeffery Kutok, Epizyme’s Chief Scientific Officer. "As our SETD2 inhibitor program began to take shape, we were struck by the many parallels we saw with our EZH2 inhibitor, TAZVERIK (tazemetostat). SETD2 is a histone methyltransferase, like EHZ2, and plays multiple key roles in cellular processes and cancer. We’re excited about the potential of SETD2 inhibition in several settings, including high-risk t(4;14) myeloma, and more broadly in myeloma without the t(4;14) mutation, as well as other B-cell malignancies. We see potential as a monotherapy, as well as in combination with existing and emerging therapies in myeloma, with preclinical data also showing synergy with tazemetostat."

"We are excited to share safety and preliminary activity data related to the EZH-301 confirmatory study of tazemetostat in Epithelioid Sarcoma (ES) at ASCO (Free ASCO Whitepaper), a key milestone toward our goal of demonstrating the benefits of tazemetostat to patients in earlier lines of treatment for ES and Follicular Lymphoma by exploring combinations with standard of care therapies," said Dr. Shefali Agarwal, Executive Vice President and Chief Medical and Development Officer at Epizyme. "As we outlined during our recent Vision Call, we plan to further explore tazemetostat as both monotherapy and in combinations across multiple hematologic and solid tumor cancers in clinical studies, which are on track to initiate later this year."

Details of the presentations are listed below:

EHA Oral Presentation

Title: Discovery of a selective inhibitor of the SETD2 histone methyltransferase with potent in vitro and in vivo activity in preclinical models of multiple myeloma
Session: Basic and translational myeloma research
Presenter: Dr. Jennifer Totman
Abstract Code: S176
Live Q&A: Monday, June 14; 2021 8:45-9:30 p.m. CEST/2:45-3:30 p.m. EDT.

The EHA (Free EHA Whitepaper) abstracts are available at View Source All oral and poster presentations will be available on the EHA (Free EHA Whitepaper) website on Friday, June 11, 2021 at 9:00 a.m. CEST/3:00 a.m. EDT.

ASCO Poster Presentation

Title: Results of the phase 1b soft-tissue sarcoma (STS) portion of the global randomized, double-blind, placebo-controlled study of tazemetostat (TAZ) plus doxorubicin (DOX) as frontline therapy for advanced epithelioid sarcoma (ES)
Session: Sarcoma
Presenter: Sant P. Chawla, M.D.
Abstract No.: 11563

The ASCO (Free ASCO Whitepaper) abstracts are available at View Source All oral and poster presentations will be available on the ASCO (Free ASCO Whitepaper) website on Friday, June 4, 2021 at 9:00 a.m. EDT.

About TAZVERIK (tazemetostat)
TAZVERIK is a methyltransferase inhibitor indicated for the treatment of:

Adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.
Adult patients with relapsed or refractory follicular lymphoma whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least two prior systemic therapies.
Adult patients with relapsed or refractory follicular lymphoma who have no satisfactory alternative treatment options.
These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

The most common (≥20%) adverse reactions in patients with epithelioid sarcoma are pain, fatigue, nausea, decreased appetite, vomiting and constipation. The most common (≥20%) adverse reactions in patients with follicular lymphoma are fatigue, upper respiratory tract infection, musculoskeletal pain, nausea and abdominal pain.

On May 12, 2021 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) reported two abstracts detailing updated data from the ongoing CTX001 clinical trials have been accepted for presentation during the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress (Press release, CRISPR Therapeutics, MAY 12, 2021, View Source [SID1234579776]).

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Abstract #EP736 entitled "CTX001 for Sickle Cell Disease: Safety and Efficacy Results from the Ongoing CLIMB SCD-121 Study of Autologous Crispr-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells," will be made available on the virtual platform as an e-poster Friday, June 11 at 9:00 CEST. The abstract posted online today includes data on patients with severe sickle cell disease with more than 3 months of follow-up, as of the interim data cut on January 28, 2021. Data will be updated and information on additional patients will be included for the congress.

Abstract #EP733 entitled "CTX001 for Transfusion-Dependent Β-Thalassemia: Safety and Efficacy Results from the Ongoing CLIMB Thal-111 Study of Autologous Crispr-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells," will be made available on the virtual platform as an e-poster Friday, June 11 at 9:00 CEST. The abstract posted online today includes data on patients with transfusion-dependent beta thalassemia (TDT) with more than 3 months of follow-up, including patients with the most severe genotypes, as of the interim data cut on January 21, 2021. Data will be updated and information on additional patients will be included for the congress.

The accepted abstracts are now available online on the EHA (Free EHA Whitepaper) website.

CTX001 is being investigated in two ongoing clinical trials as a potential one-time curative therapy for patients suffering from TDT and severe SCD.

About CTX001
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published as a Brief Report in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About CLIMB-111
The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121
The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-131
This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.

About the Gene-Editing Process in These Trials
Patients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patient’s cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the Vertex-CRISPR Collaboration
Vertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics. This amendment is subject to customary closing conditions and clearances, including clearance under the Hart-Scott Rodino Antitrust Improvements Act.

On May 12, 2021 Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplants to more patients, reported positive preliminary results from its Phase 2 clinical trial of MGTA-145 plus plerixafor in patients with multiple myeloma, which were accepted for presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress, to be held virtually June 9-17, 2021 (Press release, Magenta Therapeutics, MAY 12, 2021, View Source [SID1234579792]). Magenta also provided initial direction regarding the acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) patients it expects to evaluate in its planned clinical trial with MGTA-117.

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Multiple Myeloma Phase 2 Clinical Trial

The investigator-initiated, 25-patient Phase 2 open-label clinical trial, ongoing at Stanford University School of Medicine, is designed to evaluate the ability of MGTA-145, in combination with plerixafor, to mobilize and collect stem cells for autologous stem cell transplant in patients with multiple myeloma.

Preliminary results:

All patients (10/10) met the primary endpoint of mobilization and collection of 2 million CD34+ stem cells per kg in up to two days of same-day mobilization and apheresis. Nine of 10 patients achieved the primary endpoint in a single day.
The median number of stem cells collected in one day was 5.4 million CD34+ stem cells per kg.
Patients could also be successfully mobilized and apheresed on a second day, if needed, based upon protocol requirements. The median number of stem cells collected on day 1 and 2 (if needed) was 7.1 million CD34+ stem cells per kg in all patients. Current standard of care with G-CSF regimens require a minimum of five days of dosing to initiate stem cell collection.
All transplanted patients (6/6) successfully engrafted, with median recovery of neutrophils after 12 days and platelets after 17 days, which are within transplant expectations in multiple myeloma.
The CD34+ stem cells collected were enriched for CD90 expression (31% of CD34 cells were CD34+CD90+), a stem cell population associated with durable engraftment, which is three-fold greater than observed with G-CSF historically.
The regimen of MGTA-145 and plerixafor was well tolerated. Acute, transient, MGTA-145-related grade 1 bone or musculoskeletal pain was observed in 40% of patients following MGTA-145 infusion.
This study has broad and clinically representative inclusion criteria and is enrolling patients that represent the general transplant eligible population of patients with multiple myeloma, some of whom may have additional risk factors that may impact stem cell collection. Risk factors can include myeloma-directed therapies that are known to impact stem cell collection, previous malignancy treated with chemotherapy and/or radiation and other co-morbid conditions. Mobilization agents may be less effective in patients with multiple risk factors.

"These preliminary results are encouraging, given the expected mobilization challenges present in the multiple myeloma patient population," said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta Therapeutics. "A patient’s ability to mobilize is highly contingent on a variety of risk factors, which is particularly relevant for blood cancer patients. These initial results provide insight into MGTA-145 plus plerixafor’s ability to improve the approach to mobilization and collection, and its potential to be a first-line mobilization drug in this and other disease areas."

"Based on this initial data set, MGTA-145 combined with plerixafor has shown promising results for rapid stem cell mobilization in patients with multiple myeloma," said John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta Therapeutics.

This study is being conducted at Stanford University School of Medicine and is led by Surbhi Sidana, M.D., Assistant Professor of Medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy at Stanford Medicine.

EHA Poster Presentation (June 11, 2021)
Title: Phase 2 Study of MGTA-145 + Plerixafor for Rapid and Reliable Hematopoietic Stem Cell (HSC) Mobilization for Autologous Stem Cell Transplant in Multiple Myeloma
Author: Surbhi Sidana, M.D., Assistant Professor of Medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy, Stanford University School of Medicine
Date/Time: E-posters to be available in the EHA (Free EHA Whitepaper) Congress virtual platform Friday, June 11 at 3:00am EDT / 9:00am CEST.

This trial continues to enroll patients and Magenta expects to report additional data at the EHA (Free EHA Whitepaper) Congress, as well as at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held virtually June 4-8, 2021.

Conference Call & Webcast Details

The Company will host a conference call and webcast today at 4:30pm EDT to review the preliminary results from the multiple myeloma Phase 2 clinical trial. The live webcast of the conference call may be accessed by visiting the "Events & Presentations" page in the Investors & Media section of the Magenta Therapeutics website at View Source The live teleconference may be accessed by dialing (866) 688-5232 (domestic) or (409) 217-8328 (international) and entering conference ID: 7273937. An archived version of the call will be available on the website for 90 days.

Planned MGTA-117 Clinical Study

Magenta is on track to file an Investigational New Drug (IND) application for MGTA-117, a potential first-in-class drug for targeted patient conditioning, in June 2021. In preparation for the filing, Magenta has finalized its proposed clinical trial study design which incorporates FDA feedback from pre-IND communications. Magenta anticipates starting the Phase 1/2 dose escalation study by evaluating the safety, pharmacokinetics and pharmacodynamics of MGTA-117 as a single agent in a relapsed/refractory AML and MDS patient population. Magenta will also monitor for anti-tumor activity in this patient subset, which is a population that is not traditionally eligible for stem cell transplant. Magenta expects to work with the FDA on an ongoing basis to transition the study to transplant-eligible patients after adequate data related to the safety, pharmacokinetics and pharmacodynamics of MGTA-117 have been collected in the relapsed/ AML and MDS patient population.

On May 12, 2021 NANOBIOTIX (Euronext : NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-clinical stage biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported that Laurent Levy, chief executive officer, will present at the UBS Global Healthcare Virtual Conference on Monday, May 24, 2021 at 7:00 am Eastern Time (Press release, Nanobiotix, MAY 12, 2021, View Source [SID1234579826]).

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A live webcast of the presentation may be accessed by visiting the events section of the company’s website at www.nanobiotix.com. A replay of the webcast will be available shortly after the conclusion of the presentation and will be archived on the company’s website.

On May 12, 2021 GENFIT (Nasdaq and Euronext: GNFT), a late-stage biopharmaceutical company dedicated to improving the lives of patients with metabolic and liver diseases, reported its cash position as of March 31, 2021 and revenues for the first three months of 2021 (Press release, Genfit, MAY 12, 2021, https://ir.genfit.com/news-releases/news-release-details/genfit-reports-first-quarter-2021-financial-information [SID1234579851]).

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Cash position

As of March 31, 2021, the Company’s cash and cash equivalents amounted to €108.9 million compared with €252.0 million as of March 31, 2020 and €171.0 million as of December 31, 2020.

The decrease in cash and cash equivalents between December 31, 2020 and March 31, 2021 takes into account the Company’s partial buyback of its convertible bonds (OCEANEs) in January 2021 for an amount of €47.5 million as well as the expenses related to the convertible bond renegotiation (financial advisors, legal counsel fees, costs related to holding the shareholder and bondholder meetings, etc) which totaled €2.9 million tax included, and for which a significant portion was already paid at March 31, 2021.

Revenues
Revenues for the first three months of 2021 amounted to €1 thousand compared to €102 thousand for the same period in 2020. Revenues for the first three months of 2020 mainly consisted of revenues from services provided to Terns Pharmaceuticals pursuant to the collaboration and license agreement in relation to their clinical trials.

Reminder

On September 30, 2020, GENFIT announced its plan to reduce its cash burn by 50% by 2022 compared to the cash burn before the publication of the RESOLVE-IT Phase 3 data readout.

The Company reiterates its goal to reduce the cash burn rate from €110 million annually before our Phase 3 data, to approximately €45 million annually, beginning in 2022. 2021 will be a transition year with a cash burn of approximately €75 million (excluding the partial OCEANEs buyback transaction for €47.48 million in cash1) mainly due to the residual expenses related to the termination of the RESOLVE-IT clinical trial, and to costs associated with the workforce reduction plan.