On May 12, 2021 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, reported that two poster presentations of new clinical data and analyses related to imetelstat, the Company’s first-in-class telomerase inhibitor, will be made at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress meeting to be held virtually from June 9 – 17 (Press release, Geron, MAY 12, 2021, View Source [SID1234579781]). The abstracts for the posters are available on the EHA (Free EHA Whitepaper) website at www.ehaweb.org. Both posters will be published on the EHA (Free EHA Whitepaper) Virtual Congress platform on June 11, 2021.

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"We are pleased that the EHA (Free EHA Whitepaper) accepted both of our abstracts which allows us to present what we believe to be imetelstat’s compelling potential to become a leading treatment for lower risk MDS and MF patients," said Aleksandra Rizo, M.D., Ph.D., Geron’s Chief Medical Officer. "The new data and analyses from our Phase 2 IMbark and IMerge trials continue to highlight imetelstat’s disease-modifying activity and potential to achieve remarkable clinical benefits, including durable transfusion independence in lower risk MDS patients and improvement in MF patients’ overall survival. We look forward to the presentations and remain confident that imetelstat, with its unique telomerase inhibition mechanism of action, is a highly differentiated treatment that can positively impact patients."

Abstract Title: Efficacy of Imetelstat is Independent of Molecular Subtypes in Heavily Transfused Non-Del(5q) Lower Risk MDS (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESA)
Abstract Code: EP910

The abstract reports new data and analyses of the clinical efficacy of imetelstat in molecularly defined patient subtypes from the IMerge Phase 2 clinical trial in transfusion dependent, non-del(5q) lower risk myelodysplastic syndromes (MDS) patients who are relapsed or refractory to ESAs. Clinical responses were analyzed across multiple molecularly defined subgroups based on cytogenetic and mutation profiles. The abstract concluded that imetelstat demonstrated clinical efficacy across different molecularly defined subgroups, including patients with poor prognosis.

Abstract Title: Imetelstat Demonstrates an Acceptable Safety Profile in Myeloid Malignancies
Abstract Code: EP1106

The abstract describes new analyses of safety data from the Phase 2 IMbark and IMerge trials to further characterize hematologic and non-hematologic adverse events (AEs). Based on these analyses, the abstract concluded that imetelstat-related cytopenias observed in the trials were on-target effects based on the selective reduction of malignant cells through telomerase inhibition. Also, these cytopenias were of short duration, reversible and with limited clinical consequences when managed with the dose modification guidelines in the respective trial protocols. The difference in toxicity profiles between the two trials could be attributed to the different disease pathologies (proliferation vs. dysplasia) of patients with myelofibrosis or myelodysplastic syndromes.

In accordance with EHA (Free EHA Whitepaper) policies, abstracts submitted to the EHA (Free EHA Whitepaper) Annual Congress are embargoed from the time of submission. To be eligible for presentation at the EHA (Free EHA Whitepaper) Annual Congress, any additional data or information to be presented at the EHA (Free EHA Whitepaper) Annual Congress may not be made public before the posters are published. The posters will be available on Geron’s website at www.geron.com/r-d/publications after June 11, 2021.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Data from Phase 2 clinical trials provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in hematologic myeloid malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.

On May 12, 2021 Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported financial results for the quarter ended March 31, 2021 and provided an update on corporate progress (Press release, Exicure, MAY 12, 2021, View Source [SID1234579780]).

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"Exicure is off to a strong start in 2021, as we continue to advance our clinical and pre-clinical programs and add depth to our Board of Directors and management team," commented Dr. David Giljohann, Exicure’s Chief Executive Officer. "In the first quarter, cavrotolimod (AST-008) was granted two Fast Track designations and an Orphan Drug designation from the U.S. Food and Drug Administration. We also strengthened our Board of Directors with the appointments of Mr. James Sulat, Dr. Elizabeth Garofalo, and Mr. Andrew Sassine, and added expertise to our management team with the additions of Ms. Rebecca Peterson, VP, Investor Relations & Corporate Communications and Ms. Sarah Longoria, VP, Human Resources. We believe we are well positioned to execute on upcoming clinical milestones and operational goals for the remainder of 2021."

Pipeline Highlights

Immuno-Oncology – Cavrotolimod (AST-008)

The U.S. Food and Drug Administration (FDA) granted two Fast Track designations for cavrotolimod in January 2021:
Cavrotolimod in combination with anti-programmed death (PD)-1 or anti-PD ligand 1 (PD-L1) therapy for the treatment of patients with locally advanced or metastatic Merkel cell carcinoma (MCC), refractory to prior anti-PD-(L)1 blockade.
Cavrotolimod in combination with anti-PD-1 therapy for the treatment of patients with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC), refractory to prior anti-PD-1 blockade.
The FDA granted Orphan Drug designation for cavrotolimod for the treatment of patients with MCC in March 2021.
The Phase 1b/2 clinical trial of intra-tumoral cavrotolimod in combination with approved checkpoint inhibitors pembrolizumab or cemiplimab, for the treatment of patients with advanced or metastatic MCC or CSCC, is open and actively enrolling patients:
In the first quarter of 2021, the Company continued enrolling patients in the Phase 2 dose expansion stage of the Phase 1b/2 clinical trial.
As of April 22, 2021, 20 clinical trial sites are open for enrollment and seven additional sites are pending activation. The Company plans to open up to 30 sites for the Phase 2 stage of the clinical trial.
The Company expects to provide interim results from the Phase 2 portion of the clinical trial in mid-2021, and we expect to report overall response rate (ORR) results in the first half of 2022.
Neurology – XCUR-FXN

The Company hosted a virtual R&D Day in January 2021 to discuss progress within its neurology pipeline, which included an overview of the Company’s XCUR-FXN path to clinical validation for the treatment of Friedreich’s Ataxia (FA).
During the first quarter of 2021, the Company continued to advance preclinical and IND-enabling studies of XCUR-FXN in FA and disclosed encouraging preclinical mouse data, demonstrating significant upregulation of XCUR-FXN components in the brain and spinal cord. The Company believes these data support the potential for XCUR-FXN as a disease-modifying treatment for the disease.
The Company is on track with prior guidance to submit an IND for XCUR-FXN in FA by year-end 2021 and expects to initiate a first-in-patient Phase 1b clinical trial of XCUR-FXN in FA in the first half of 2022.
First Quarter Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and short-term investments were $67.4 million as of March 31, 2021 compared to $82.1 million as of December 31, 2020.

Research and Development (R&D) Expenses: R&D expenses were $10.3 million for the quarter ended March 31, 2021, compared to $6.1 million for the quarter ended March 31, 2020. The Company has increased full-time headcount in R&D from 36 at March 31, 2020 to 54 at March 31, 2021. The increase in R&D expense reflects this increased headcount and the related increase in R&D activities, in addition to increased clinical trial activities.

General and Administrative Expenses: General and administrative expenses were $2.9 million for the quarter ended March 31, 2021, compared to $2.6 million for the quarter ended March 31, 2020. This increase is primarily due to costs related to new hires needed to grow the Company as it evolves.

Net Loss: Exicure had a net loss of $12.5 million for the quarter ended March 31, 2021 compared to net income of $1.2 million for the quarter ended March 31, 2020. The increase in net loss was primarily driven by lower revenue associated with Exicure’s collaboration with AbbVie as well as higher R&D costs to advance our pipeline.

Cash Runway Guidance: The Company believes that, based on its current operating plans and estimates of future expenses, as of the date of this press release, its existing cash, cash equivalents and short-term investments will be sufficient to fund its operations for at least the next 12 months.

About Friedreich’s Ataxia (FA)

FA is a rare, degenerative, life-shortening neuro-muscular disorder that affects children and adults, and involves the loss of strength and coordination usually leading to wheelchair use, diminished vision, hearing and speech, scoliosis, increased risk of diabetes, and a life-threatening heart condition. There are currently no FDA-approved treatments. The Company estimates that approximately 13,000 patients across the United States, Europe, Canada and Australia are affected by FA.

On May 12, 2021 Delcath Systems, Inc. (NASDAQ: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported business highlights and financial results for the first quarter ended March 31, 2021 (Press release, Delcath Systems, MAY 12, 2021, View Source [SID1234579779]).

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Recent Business Highlights

During and since the first quarter the company:

Reported positive preliminary results from the FOCUS Clinical Trial (NCT02678572) for Patients with Hepatic Dominant Ocular Melanoma treated with HEPZATO KIT based on an analysis of currently evaluable patients. The preliminary analysis included 87% of treated patients and final results are expected later in the year.
The primary endpoint, overall response rate (ORR), exceeded the prespecified threshold for success by a large enough margin to ensure that final results based on 100% of patients will be positive.
Both prespecified ORR and Progression Free Survival comparative analyses versus the best alternative care arm demonstrated a statistically significant improvement.
The safety profile was consistent with the safety profile of CHEMOSAT treatment described in previous European single-center and multi-center publications with no new safety signals observed in this patient population and no treatment related deaths on study.
Announced that the United Kingdom’s National Institute for Health and Care Excellence, has updated its guidance for the Delcath CHEMOSAT Hepatic Delivery System for Melphalan (CHEMOSAT) in the treatment of patients with metastases in the liver from Ocular Melanoma. Under this designation, private insurance may be more likely to fund treatment with CHEMOSAT, some regional funding may be more accessible, and a process is now available to seek national reimbursement.
Announced an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting to be held virtually June 4-8, 2021.
"The recently released preliminary results from the FOCUS trial strongly indicates that HEPZATO’s benefit risk ratio is a significant improvement versus an earlier generation of Delcath’s proprietary percutaneous hepatic perfusion system," said Gerard Michel, CEO of Delcath. "We look forward to continued progress in the balance of the year, as we prepare both to file an NDA in early 2022 and expand the development of HEPZATO into additional areas of high unmet need."

First Quarter 2021 Financial Results:

Income Statement Highlights.

Product revenue for the three months ended March 31, 2021 was approximately $0.4 million, compared to $0.3 million for the prior year period from our sales of CHEMOSAT procedures in Europe. Selling, general and administrative expenses were approximately $3.3 million compared to $2.3 million in the prior year quarter. Research and development expenses for the quarter were $3.7 million compared to $3.0 million in the prior year quarter. Total operating expenses for the quarter were $7.0 million compared with $5.3 million in the prior year quarter. First quarter expenses included approximately $2.2 million of stock option expense compared to no material stock option expense in the prior year quarter.

We recorded a net loss for the three months ended March 31, 2021, of $6.8 million, compared to a net income of $7.9 million for the same period in 2020

Balance Sheet Highlights.

At March 31, 2021, we had cash, cash equivalents and restricted cash totaling $26.7 million, as compared to cash, cash equivalents and restricted cash totaling $4.7 million at March 31, 2020. During the three months ended March 31, 2021 and March 31, 2020, we used $4.6 million and $5.2 million, respectively, of cash in our operating activities.

Conference Call Information

To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call.

On May 12, 2021 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported its financial results for the first quarter ended March 31, 2021 (Press release, Curis, MAY 12, 2021, View Source [SID1234579778]).

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"The first quarter of 2021 saw continued momentum for our pipeline of next generation targeted cancer therapies designed to meaningfully improve and extend patients’ lives. We continued to make important progress with CA-4948, our first-in-class, small molecule inhibitor of IRAK4, now in three clinical trials after expanding into one new study earlier this year with the Phase 2 LUCAS IST for patients with lower-risk MDS, as well as expanding our previous Phase 1/2 study in patients with relapsed/refractory (R/R) NHL to include the combination of CA-4948 plus ibrutinib. We were also very pleased to announce that CA-4948 was granted Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of AML and MDS, highlighting the unique potential of our IRAK4 program," said James Dentzer, President and Chief Executive Officer of Curis. "We are especially excited about the AML/MDS data published today in the EHA (Free EHA Whitepaper) abstract, and we look forward to providing additional data from this study in the oral presentation at EHA (Free EHA Whitepaper) next month."

Mr. Dentzer added, "We are also pleased with the continuing dose escalation in our ongoing Phase 1 study of CI-8993, our first-in-class monoclonal anti-VISTA antibody for the treatment of patients with R/R solid tumors and look forward to providing initial data from this study later this year."

First Quarter 2021 and Recent Operational Highlights

Precision oncology, CA-4948 (IRAK4 Inhibitor; Aurigene collaboration):

Today, EHA (Free EHA Whitepaper) released the Curis abstract reporting interim data from the ongoing Phase 1/2 study of CA-4948 in patients with R/R AML and MDS. The data are from 15 patients as of February 8, 2021 (the cut-off date) and are consistent with previously announced findings, including marrow blast reductions observed at all tested doses in 8 of 9 (89%) evaluable patients with elevated blast counts at baseline, with 1 patient experiencing a full hematologic recovery complete response, 1 complete remission with incomplete hematologic recovery (CRi) with negative minimal residual disease, and 2 bone marrow complete responses (CRs).
All 3 patients presenting with SF3B1 or U2AF1 spliceosome mutations achieved marrow CR or better
All patients with objective responses also saw signs of hematologic recovery
Updated safety, pharmacodynamic, and efficacy data, as well as data from additional trial participants, will be featured in an oral presentation at EHA (Free EHA Whitepaper) on Friday, June 11 at 9:00 am CEST (3:00 am EDT).
Curis updated that the 500mg BID dosing regimen in the AML/MDS study has exceeded the maximum tolerated dose according to protocol guidelines. Two patients in the cohort were observed to have dose-limiting toxicities, one of whom had Grade 3 rhabdomyolysis and the other experienced Grade 3 syncope. Both AEs resolved after discontinuation of dosing. Current enrollment is exploring lower dose levels to determine the appropriate recommended phase 2 dose (RP2D).
Also today, Curis reported non-clinical data to be presented in a poster at EHA (Free EHA Whitepaper) demonstrating synergistic antitumor activity of CA-4948 in combination with azacitidine and venetoclax in leukemia cells, providing supportive rationale for evaluation of the combinations in a clinical setting for AML and MDS patients.
The Phase 1/2 study of CA-4948 in AML and MDS was expanded to include both a combination dose escalation and a monotherapy dose expansion:
Combination dose escalation, which will start at 200mg BID, will include two cohorts:
1) CA-4948 + azacitidine, for patients with AML or MDS who are naïve to hypomethylating agents (HMA)
2) CA-4948 + venetoclax, for patients with AML or MDS after first line therapy who are naïve to venetoclax
Monotherapy dose expansion, which will begin after the RP2D is determined, will include four cohorts:
1) MDS patients, R/R to HMA, with spliceosome mutations
2) MDS patients, R/R to HMA, without spliceosome mutations
3) R/R AML patients with FLT3-ITD mutation
4) R/R AML patients with FLT3 WT
In April 2021, Curis announced that the U.S. Food and Drug Administration (FDA) had granted Orphan Drug designation for CA-4948 for the treatment of AML and for treatment of MDS.
Also in April, Curis presented updated data on a potentially predictive biomarker demonstrating target engagement in a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021.
In February, Curis announced the dosing of the first patient in its Phase 1/2 combination study of CA-4948 plus ibrutinib, for the treatment of patients with R/R NHL or other hematologic malignancies. In preclinical models, CA-4948 demonstrated synergistic anti-cancer activity when combined with a potent BTK inhibitor such as ibrutinib. Curis expects to report initial data from this study in the second half of 2021.
Earlier in February, Curis announced the initiation of the investigator-sponsored Phase 2 LUCAS study of CA-4948 for the treatment of anemia in patients with very low, low, or intermediate-risk MDS. The study is expected to start recruitment in the second quarter of 2021.
Immuno-oncology, CI-8993 (anti-VISTA antibody; ImmuNext collaboration):

Curis continues to enroll patients in the ongoing Phase 1 dose escalation study of its first-in-class monoclonal anti-VISTA antibody for the treatment of R/R solid tumors and expects to report initial clinical data in the second half of 2021.
Upcoming 2021 Planned Milestones

Report additional clinical data at the EHA (Free EHA Whitepaper) 2021 Virtual Congress from the Phase 1/2 monotherapy study of CA-4948 in patients with AML and MDS, including patients with spliceosome mutations that result in aberrant splicing of oncogenic IRAK4-L.
Initiate dosing in the Phase 1/2 combination study of CA-4948 plus azacitidine and CA-4948 plus venetoclax in patients with R/R AML and MDS.
In the second half of 2021, report initial data from the ongoing Phase 1/2 combination study of CA-4948 plus ibrutinib in patients with R/R NHL.
In the second half of 2021, report initial data from the ongoing Phase 1 monotherapy study of CI-8993 for the treatment of R/R solid tumors.
First Quarter 2021 Financial Results

For the first quarter of 2021, Curis reported a net loss of $9.9 million or $0.11 per share on both a basic and diluted basis, as compared to a net loss of $9.7 million, or $0.28 per share on both a basic and diluted basis for the same period in 2020.

Revenues for the first quarter of 2021 and 2020 were $2.2 million and $2.7 million, respectively.

Operating expenses for the first quarter of 2021 were $11.0 million, as compared to $11.2 million for the same period in 2020, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.1 million for the first quarter of 2021 and 2020.

Research and Development Expenses. Research and development expenses were $6.8 million for the first quarter of 2021 as compared to $7.5 million for the same period in 2020. The decrease in direct research and development expenses for the quarter is primarily attributable to the upfront license fee expense from our option and license agreement with ImmuNext that occurred during the first quarter of 2020. These costs were partially offset by a $0.3 million increase in employee related costs.

General and Administrative Expenses. General and administrative expenses were $4.1 million for the first quarter of 2021, as compared to $3.6 million for the same period in 2020. The increase in general administrative expense was driven primarily by higher costs for stock-based compensation and professional and consulting services, partially offset by lower legal services costs during the three months ended March 31, 2021.

Other Expense, Net. For the first quarter of 2021 and 2020, net other expense was $1.1 million and $1.2 million, respectively. Net other expense primarily consisted of imputed interest expense related to future royalty payments.

As of March 31, 2021, Curis’s cash, cash equivalents and investments totaled $168.4 million, and there were approximately 91.5 million shares of common stock outstanding. Curis expects that its existing cash, cash equivalents and investments should enable it to maintain its planned operations into 2024.

Conference Call Information

Curis management will host a conference call today, May 12, 2021, at 4:30 p.m. ET, to discuss these financial results, as well as provide a corporate update.

To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412-317-5252 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.

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.