On May 12, 2021 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported that Fred Schwarzer, Chief Executive Officer, will participate in a fireside chat at the 2021 RBC Capital Markets Global Healthcare Conference on May 19, 2021 at 1:55 p.m. EDT (Press release, IGM Biosciences, MAY 12, 2021, View Source [SID1234579784]). The conference will be held in a virtual meeting format.

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A live webcast of the event will be available on the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days following the presentation.

On May 12, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported financial results and business highlights for the first quarter of 2021 (Press release, Hookipa Pharma, MAY 12, 2021, View Source [SID1234579783]).

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"We had a strong start to the year as we drive our pipeline forward to deliver a new class of arenavirus-based immunotherapeutics. As we shared at AACR (Free AACR Whitepaper), one initial dose of our lead oncology candidates, HB-201 or HB-202, each induced a robust increase in antigen-specific T cells, including an increase of up to 8% of antigen-specific circulating CD8+ T cells, in people with advanced Human Papillomavirus 16-positive (HPV16+) cancers," said Joern Aldag, Chief Executive Officer at HOOKIPA. "We believe these data are impressive, and they are consistent with the pre-clinical data published in Cell Reports Medicine in March. Both data sets highlight the potential of our engineered arenavirus platform to redefine success in cancer immunotherapy. As our clinical programs progress, we’re excited about the oral abstract presentation at ASCO (Free ASCO Whitepaper) (#2502) and other future data presentations at upcoming conferences."

Program Highlights

In April 2021, HOOKIPA announced positive preliminary Phase 1 immunogenicity data for its lead oncology candidates, HB-201 and HB-202, for the treatment of advanced HPV16+ cancers, reinforcing the promising anti-tumor activity reported from the Phase 1/2 clinical trial in December 2020. The preliminary immunogenicity data demonstrated a robust increase in HPV16+-specific T cells, including an increase of up to 8% of antigen-specific circulating CD8+ T cells, after one dose of HB-201 or HB-202. Early HB-201 monotherapy data also highlighted immune system activation of increasing interferon-gamma and other immune stimulatory cytokines with a single dose. The data were presented at a late-breaker poster session at the virtual American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

In March, Cell Reports Medicine published pre-clinical data on HOOKIPA’s arenaviral therapeutics. The peer-reviewed article showed that intravenous HB-201 administration induced single digit percentage of antigen-specific CD8+ T cells, while alternating administration of HB-201 and HB-202 induced a potent CD8+ T cell response, exceeded 50% of the circulating T cell pool. The two-vector cancer therapeutic approach also resulted in tumor cures and long-term immunity in a pre-clinical setting.

HOOKIPA’s prophylactic Cytomegalovirus, or CMV, vaccine candidate, HB-101, continued to enroll patients awaiting kidney transplantation in a Phase 2 clinical trial. We expect to conclude trial enrollment in mid-2021 and to report additional safety, immunogenicity, and efficacy data from evaluable patients in the second half of 2021.
Upcoming Milestones

Oral abstract presentation at ASCO (Free ASCO Whitepaper) (#2502 at 3:00pm EDT on June 7): First report of the safety/tolerability and preliminary antitumor activity of HB-201 and HB-202, an arenavirus-based cancer immunotherapy, in patients with HPV16+ cancers
Initial HB-201/HB-202 Phase 1/2 efficacy data in HPV16+ cancers in mid-2021
Additional HB-101 CMV Phase 2 efficacy data in H2 2021
Advancing our HB-300 to IND for the treatment of metastatic prostate cancer
HBV and HIV collaboration with Gilead Sciences advancing towards clinical studies
First Quarter 2021 Financial Results

Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of March 31, 2021 was $128.1 million compared to $143.2 million as of December 31, 2020. The decrease was primarily attributable to cash used in operating activities.

Revenue was $5.3 million for the three months ended March 31, 2021, and $3.7 million for the three months ended March 31, 2020. The increase was primarily due to higher cost reimbursements received under the Collaboration Agreement with Gilead and the recognition of cost reimbursements initially recognized as deferred revenue.

Research and Development Expenses: HOOKIPA’s research and development expenses were $20.2 million for the three months ended March 31, 2021, compared to $11.5 million for the three months ended March 31, 2020.

The primary drivers of the increase in direct research expenses were an increase in clinical trial expenses of $1.5 million and an increase in manufacturing and quality control expenses of $4.6 million.

The increase was mainly due to the progress in our HB-201 and HB-202 clinical trial, the increased patient recruitment in our HB-201 and HB-202 clinical trial, monitoring and testing activities and manufacturing and quality control work in preparation of a further extension of the trial. Manufacturing and quality control expenses were also driven by the progress towards clinical development in our Gilead partnered programs. This increase in HB-201/HB-202 and Gilead related direct expenses was partially offset by a decrease in direct costs related to our HB-101 program due to slower patient recruitment as a result of the COVID pandemic.

The increase in internal research and development expenses was mainly due to an increase of personnel-related research and development expenses, resulting primarily from a higher headcount, while stock-based compensation expenses included in personnel-related research and development expenses decreased. In addition, an increase in facility related costs and an increase in other internal costs contributed to the overall increase in internal research and development expenses.

General and Administrative Expenses: General and administrative expenses for the three months ended March 31, 2021 were $4.3 million, compared to $4.6 million for the three months ended March 31, 2020. The decrease was primarily due to a decrease in personnel-related expenses, partially offset by an increase in professional and consulting fees. The decrease in personnel-related expenses resulted from decreased stock compensation expenses, partially offset by increased salaries and a growth in headcount in our general and administrative functions.

Net Loss: HOOKIPA’s net loss was $17.2 million for the three months ended March 31, 2021 compared to a net loss of $10.9 million for the three months ended March 31, 2020. This increase was due to an increase in research and development expenses, partially offset by an increase in revenues from collaboration and licensing, a decrease in general and administrative expenses, and an increase in grant income.

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.

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