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.

On May 12, 2021 Epigenomics AG (FSE: ECX, OTCQX: EPGNY, the "Company") reported financial results (IFRS, unaudited) for the first three months of 2021 (Press release, Epigenomics, MAY 12, 2021, View Source [SID1234579751]).

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Financial key figures

Product revenue in the first quarter 2021 decreased from EUR 239 thousand to EUR 106 thousand compared to the prior-year period due to lower product revenues in the United States and Europe. In the current pandemic situation, many patients eligible for screening continue to postpone their screenings.
Research and development costs decreased from EUR 1,603 thousand to EUR 737 thousand.
Selling, general and administrative costs diminished from EUR 1,992 thousand to EUR 1,601 thousand.
EBITDA before share-based payment expenses improved to EUR -985 thousand in the reporting period vs. EUR -2,641 thousand in the same period as the previous year
The net loss for the period was EUR -1,201 thousand (Q1 2020: EUR -2,982 thousand); the net loss per share decreased significantly from EUR 0.55 to EUR 0.20 compared to the same period of the previous year.
Cash consumption decreased to EUR 1,401 thousand in Q1 2021 (Q1 2020: EUR 3,284 thousand).
As of March 31, 2021, the Company had cash and cash equivalents (including marketable securities) of EUR 7,446 thousand (December 31, 2020: EUR 4,527 thousand).
Strategic options

Following the negative reimbursement decision by the CMS in January 2021, the management is considering various strategic options. As announced at the end of March, the Company is evaluating, among other things, a potential sale of the Company by way of a share deal (public takeover) or asset deal to one or more investors as part of an M&A transaction. For this purpose, a corresponding sales process has been set up and the Company is in discussions with several potential parties. The Company has mandated a leading international investment bank as advisor in connection with the sale process.

Major events after the end of the reporting period

The first conversion period for the mandatory convertible bond issued in January 2021, ran from April 1 to April 14, 2021. Bonds with a nominal value of EUR 4,357,606.00 have been converted into 3,961,460 new shares (ISIN DE000A3H2184). Accordingly, the number of issued shares increased from 5,891,230 to 9,852,690 no-par value registered shares of the Company. The Company’s share capital increased correspondingly to EUR 9,852,690.00. The outstanding portion of the mandatory convertible bond 2021/2024 therefore has a nominal value of EUR 1,142,394.00.
In addition, on April 27, 2021, the Executive Board of Epigenomics AG resolved, with approval of the Supervisory Board, to increase the company’s share capital through a rights issue using the Authorized Capital 2020/II. The share capital shall be increased from currently EUR 9,852,690.00 by up to EUR 1,970,537.00 to up to EUR 11,823,227.00 by issuing up to 1,970,537 new registered no par value shares of the Company against cash contributions. The subscription price for the new shares was set at EUR 1.10. The subscription ratio is 5:1. This means for each five existing shares of the Company, a subscription right for one new share will be allocated. Deutsche Balaton Aktiengesellschaft, with 22.59% the largest shareholder of Epigenomics AG, has already announced that it will participate in the capital increase. The capital increase serves the purpose of improving the Company’s liquidity position ahead of upcoming important strategic decisions.
Outlook 2021

Revenue

The Company confirms its outlook for fiscal year 2021 and continues to expect revenue within the range of EUR 0.4 million to EUR 1.0 million. If the NCD decision made by CMS is successfully appealed or reversed in 2021, a change in the revenue guidance would be made.
EBITDA / Cash consumption

For EBITDA before share-based payment expenses, Epigenomics forecasts a range of EUR -7.0 million to EUR -9.0 million. Based on the Company’s 2021 business plan, cash consumption is expected to be in line with the EBITDA forecast (before share-based payment expenses).
Further Information

The 2020 Q1 interim statement (unaudited) is available on the Epigenomics’ website: View Source

Conference call for analysts and investors

Epigenomics AG will host a conference call for analysts and investors today at 5.00 pm (CET) / 11.00 am (EDT). The webcast can be accessed on the Company’s website: View Source

The dial-in numbers for the conference call are:

Participants are asked to dial in 10 minutes prior to the start of the conference call and to register using the link above.

An audio replay of the conference call will be provided on the Epigenomics’ website subsequently.

On May 12, 2021 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that two abstracts for CA-4948, a novel, small molecule IRAK4 inhibitor, have been accepted for oral and poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress (EHA) (Free EHA Whitepaper), which will be held virtually from June 9-17, 2021 (Press release, Curis, MAY 12, 2021, View Source [SID1234579777]). The abstracts include updated data from a February data-cut for its ongoing open-label, single arm, Phase 1/2 study of CA-4948 in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS).

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"We are very pleased to report this clinical update on our first-in-class IRAK4 kinase inhibitor, CA-4948, as an anticancer agent for patients with acute myeloid leukemia and myelodysplastic syndromes for whom multiple prior lines of therapy have been unsuccessful," said James Dentzer, President and Chief Executive Officer of Curis. "The clinical data published in the abstract this morning are consistent with our preliminary findings reported late last year showing that CA-4948 has, in addition to encouraging safety characteristics, clear potential to reduce leukemic blasts in late-line patients, along with early signs of hematologic recovery. We look forward to providing updated safety, pharmacodynamic, and efficacy data, as well as data from additional patients and nonclinical combination synergy data at EHA (Free EHA Whitepaper) next month."

Key findings from a cutoff date of February 8, 2021 in 15 patients (8 MDS and 7 AML) include:

Bone marrow blast reductions observed at all tested doses in 8 of 9 (89%) evaluable patients (at least one malignancy assessment following first cycle) with elevated blast counts at baseline
Objective responses observed included 1 patient experiencing a full hematologic recovery complete response, 1 CRi with negative minimal residual disease, and 2 bone marrow CRs
All 3 patients with SF3B1 or U2AF1 spliceosome mutation achieved marrow CR or better
All patients with objective responses also saw signs of hematologic recovery
Details of the presentations are as follows:

Oral Presentation:

Title: A Phase 1, Dose Escalation Trial with Novel Oral IRAK4 Inhibitor CA-4948 in Patients with Acute Myelogenous Leukemia or Myelodysplastic Syndrome – Interim Report
Author: Guillermo Garcia-Manero, MD, MD Anderson Cancer Center
Session Name: 10. Myelodysplastic syndromes – Clinical
Presentation Time: Friday, June 11, 2021, 09:00 CEST (3:00 am ET)
Q&A Session: Wednesday, June 16, 2021, 17:00 CEST (11:00 am ET)
Poster Presentation

Title: IRAK4 Inhibitor CA-4948 Potentiates Antitumor Effects of Azacitidine and Venetoclax in Human Acute Myeloid Leukemia
Session Name: 01. Acute lymphoblastic leukemia – Biology & Translational Research
Session Date & Time: Friday, June 11, 2021, 09:00 CEST (3:00 am ET)
Virtual KOL Event

Virtual event to be hosted Friday, June 11 at 8:00 am ET, featuring Dr. Guillermo Garcia-Manero, Chief of the Section of Myelodysplastic Syndromes within the Department of Leukemia at The University of Texas MD Anderson Cancer Center
Discussion of the EHA (Free EHA Whitepaper) presentation data from the ongoing Phase 1/2 study of CA-4948 in patients with acute myeloid leukemia and myelodysplastic syndromes
A live webcast of the presentation will be available under "Events & Presentations" in the Investors section of the Company’s website at www.curis.com. A replay of the webcast will be available on the Curis website for 90 days following the event.
Additional meeting information can be found on the EHA (Free EHA Whitepaper) website at www.ehaweb.org/congress. Each presentation will also be available under "Events and Presentations" in the Investors section of the Company’s website at www.curis.com

About CA-4948

CA-4948 is an IRAK4 kinase inhibitor and IRAK4 plays an essential role in the toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) signaling pathways, which are frequently dysregulated in patients with AML and MDS. Third parties have recently discovered that the long form of IRAK4 (IRAK4-L) is oncogenic and preferentially expressed in over half of patients with AML and MDS. The overexpression of IRAK4-L is believed to be driven by a variety of factors, including specific spliceosome mutations such as SF3B1 and U2AF1.