On March 12, 2020 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported corporate updates and financial results for the fourth quarter and fiscal year ended December 31, 2019 (Press release, CymaBay Therapeutics, MAR 12, 2020, View Source [SID1234555479]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Since announcing the decision to halt the development of seladelpar last November, we have been focused on two parallel initiatives: an investigation of the unexpected histologic findings identified by study pathologists in the Phase 2b study of seladelpar in NASH, and an evaluation of strategic alternatives to maximize shareholder value," said Sujal Shah, President and CEO of CymaBay. "Our investigation includes several key activities that will be essential for us to understand the nature and significance of the findings and have the requisite follow-up dialogue with the FDA which we are planning for before the end of the second quarter."

Shah continued, "In parallel to this investigation, our executive team and board have been focused on a comprehensive evaluation of strategic alternatives and cost-cutting initiatives. While we remain committed to completing the investigation, we believe these efforts are prudent in order to make decisions expeditiously once we gain needed clarity on the potential path forward for seladelpar."

Recent Business Highlights

The development of seladelpar was halted in all indications after consulting with expert liver pathologists and hepatologists and in consideration of patient safety. The FDA agreed with this decision, formally placed the seladelpar program on clinical hold for all indications, and subsequently provided input on plans to further investigate the situation. Since then, CymaBay has commenced an in-depth review of the Phase 2b NASH findings. This investigation includes three activities intended to confirm and subsequently understand the significance of the findings identified by study pathologists:
First, a comprehensive collection and review of data including patient demographics, medical history, concomitant medications and a broad set of biochemical markers.
Second, a blinded, independent review of baseline and end of treatment biopsies by several, world-renowned liver pathologists. The independent pathology review will include an accepted pathology scoring framework, known as the Ishak Modified HAI scoring system, to quantitatively characterize features of histology present in our patient population both at baseline and at end-of-treatment. Among these features includes a scoring for the presence and severity of interface hepatitis which is not quantified in the existing framework for scoring NASH pathology.
Third, a formal pathology and clinical hepatology review panel meeting that CymaBay anticipates convening in the middle of the second quarter during which experts will review all information gathered to provide a consensus and independent determination of the role of seladelpar in the Phase 2b NASH findings. This panel will allow for a properly informed dialogue with FDA regarding seladelpar development.

Completed reading of the Phase 2b NASH end-of-treatment biopsies by study pathologists. Preliminary results reported below are for the 152 patients out of the 181 patients enrolled in the study with paired biopsies at entry and end-of-study:
Phase 2b Preliminary Topline Results

Resolution of NASH defined as patients having a NAFLD Activity Score (NAS) of 0 or 1 for lobular inflammation and 0 for hepatocellular ballooning.
Patients that did not have end-of-study biopsies are not included in reported histology endpoints.
Measured changes from baseline to end-of-treatment in liver enzymes including ALT, AST, GGT and ALP, resembled the pattern of meaningful reductions previously reported at week 12.
Implemented cost containment and restructuring program following the decision to place the seladelpar program on hold in order to minimize expenses and conserve capital. As part of this program, CymaBay froze hiring, significantly scaled-back future procurement plans, reduced its work force by more than 60% and scaled down or cancelled many existing contracts for goods and services. The size of the Board of Directors has also been decreased from nine to five seats.

As a result of these actions, CymaBay recorded a $5.1 million restructuring charge during the fourth quarter which includes $2.9 million of employee severance costs, $0.9 million of non-cash stock-based compensation expense associated with the acceleration of stock options of certain terminated employees, and $1.3 million of charges associated with the termination of certain contract manufacturing agreements.

Held $190.9 million in cash, cash equivalents and short-term investments at December 31, 2019.
Fourth Quarter and Year Ended December 31, 2019 Financial Results

Research and development expenses for the three and twelve months ended December 31, 2019 were $20.9 million and $83.8 million, respectively. This compared to R&D expenses of $16.4 million and $58.1 million for the three and twelve months ended December 31, 2018, respectively. Prior to the decision to halt development of seladelpar in November 2019, research and development expenses in the fourth quarter and twelve months ended 2019 were generally higher than in the corresponding periods in 2018 due to expanding clinical trial activities related to the PBC Phase 3 clinical trial, PSC Phase 2 clinical trial, and other NDA-enabling studies.

General and administrative expenses for the three and twelve months ended December 31, 2019 were $4.5 million and $19.2 million, respectively. This compared to $4.2 million and $14.4 million for the three and twelve months ended December 31, 2018, respectively. Prior to the decision to halt development of seladelpar, G&A expenses in the fourth quarter and twelve months ended 2019 were higher than in the corresponding periods in 2018 as a result of higher labor costs and other administrative expenses necessary to support expanding development activities.

Net loss for the three and twelve months ended December 31, 2019 was $29.4 million, or ($0.43) per diluted share, and $102.8 million, or ($1.53) per diluted share, respectively. This compared to net loss of $19.4 million, or ($0.32) per diluted share, and $72.5 million, or ($1.26) per diluted share, in the three and twelve months ended December 31, 2018, respectively. Net loss was higher largely due to increases in operating expenses, including restructuring charges.
Conference Call Details

CymaBay will host a conference call today at 4:30 p.m. ET to discuss fourth quarter and fiscal year end 2019 financial results and provide a business update. To access the live conference call, please dial 855-327-6837 from the U.S. and Canada, or 631-891-4304 internationally, Conference ID# 10008868. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

On March 12, 2020 Marker Therapeutics, Inc. (Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported financial results for the full year ended December 31, 2019 (Press release, TapImmune, MAR 12, 2020, View Source [SID1234555494]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"With a clear path forward for our Phase 2 trial in AML patients with our novel T cell therapy, and the cash resources needed to advance our studies, 2020 is shaping up to be a busy and productive year for our Company," said Peter L. Hoang, President & CEO of Marker Therapeutics. "Based on promising results observed with our MultiTAA T cell therapy across various forms of cancer in investigator-sponsored trials, we are also evaluating opportunities for additional Marker-sponsored trials."

PROGRAM UPDATES

Multi-Antigen Targeted (MultiTAA) T Cell Therapies

Marker Prepares to Initiate Phase 2 AML Trial

In February 2020, the Company announced an updated study protocol for its Phase 2 clinical trial of MultiTAA T cell therapy in post-allogeneic hematopoietic stem cell transplant patients with acute myeloid leukemia (AML) in both the adjuvant and active disease setting. Under an amended trial design, the U.S. Food and Drug Administration (FDA) has permitted the trial to move forward with the safety lead-in. During the second half of 2020, Marker expects to complete enrollment of the first three patients and to submit the information required by the FDA to lift a partial clinical hold during the second half of 2020. The Company does not currently expect the partial clinical hold to significantly impact site or patient enrollment.

Investigator-Sponsored Trials with MultiTAA T Cell Therapy Continue to Generate Positive Results

Marker previously reported interim data from an ongoing Phase 1/2 clinical trial of MultiTAA T cell therapy for the treatment of patients with pancreatic adenocarcinoma being conducted by its partners at the Baylor College of Medicine (BCM). In this trial, the modified T cells exhibited activity against both targeted tumor-associated antigens (TAA) and non-targeted TAAs, indicating induction of antigen spreading. To date, there has not been any cytokine release syndrome or neurotoxicity observed in this trial.

T Cell-Based Vaccines

Phase 2 Triple Negative Breast Cancer Trial Progressing

Marker’s T cell-based vaccine program in triple negative breast cancer has delivered the following results as of September 30, 2019:

·Based on a preliminary analysis of 34 patients enrolled in the triple negative breast cancer trial, 31 patients showed meaningful immune response to vaccine treatment;

·Of 80 patients treated at 11 clinical sites, 16 have shown disease progression following treatment with TPIV200.

Phase 2 Platinum-Sensitive Advanced Ovarian Cancer Trial

·As previously announced, Marker has discontinued the development of TPIV200 in patients with platinum-sensitive advanced ovarian cancer based on an unblinded review of interim results from the trial conducted by the Data Safety Monitoring Board (DSMB). While the DSMB did not express safety concerns, Marker elected to discontinue the trial as it did not meet the threshold for probability of clinical benefit based upon the Company’s pre-specified criteria.

FINANCING UPDATE

·On March 2, 2020, Marker announced that the Company entered into a Common Stock Purchase Agreement of up to $30 million with Aspire Capital Fund, LLC, a Chicago-based institutional investor and long-term Marker shareholder.

FULL YEAR 2019 FINANCIAL RESULTS

Cash Position and Guidance: At December 31, 2019, Marker had cash and cash equivalents of $43.9 million. The Company believes that the financial flexibility provided by the Aspire transaction will enable the cash runway to extend beyond the second quarter of 2021.

R&D Expenses: Research and development expenses were $12.8 million for the year ended December 31, 2019 compared to $8.0 million for the year ended December 31, 2018. The increase was primarily attributable to increases in personnel-related expenses relating to the build-up of Marker’s internal infrastructure, an increase in clinical consulting and professional expenses relating to preparation of the AML trial, an increase in process development expenses, offset by a decrease in clinical trial expenses due to the stages of ongoing clinical trials and the decreased number of active patients in such trials.

G&A Expenses: General and administrative expenses were $10.0 million for the year ended December 31, 2019, compared to $24.4 million for the year ended December 31, 2018. The decrease was primarily attributable to a decrease of $12.8 million in stock-based compensation expenses due to executive stock option grants issued in fiscal year 2018, as well as a decrease in merger-related expenses during fiscal year 2019, offset by increased expenses in headcount-related and legal and other professional expenses.

Net Loss: Marker reported a net loss of $21.4 million for the year ended December 31, 2019, compared to a net loss of $148.0 million for the year ended December 31, 2018.

On March 12, 2020 Celyad (Euronext Brussels and Paris, and Nasdaq: CYAD), a clinical-stage biopharmaceutical company focused on the development of CAR-T cell-based therapies, reported that the Company will report full year 2019 financial and operating results on the evening of Tuesday, March 24, 2020 (Press release, Celyad, MAR 12, 2020, View Source [SID1234555510]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Following the press release, Celyad management will host a conference call on Wednesday, March 25 at 1 p.m. CET / 8 a.m. ET to discuss full year 2019 results and provide an update on the Company’s recent progress and upcoming milestones.

Participants may access the conference call by dialing +44 (0) 2071 928501. The conference ID for the call is 1392585. Alternatively, participants may access the conference call by dialing the following local numbers: Belgium 02 401 70 35, France 01 76 72 89 28, Netherlands 020 7188527 and United States 1 917 720 0181.

To access the subsequent archived recording, visit the "Events & Webcasts" section of the Celyad website.

Additionally, in consideration of growing concerns over the spread of coronavirus disease, COVID-19, the Company has decided to postpone its upcoming Research & Development Day, previously scheduled for March 18th, until later in the year.

On March 12, 2020 Kitov Pharma Ltd. ("Kitov") (NASDAQ/TASE: KTOV), a clinical-stage company advancing first-in-class therapies to overcome tumor immune evasion and drug resistance, reported pricing of a public offering of 20,000,000 units at a price to the public of $0.30 per unit, for gross proceeds of $6.0 million, before deducting placement agent fees and other offering expenses payable by Kitov (Press release, Kitov Pharmaceuticals , MAR 12, 2020, View Source [SID1234555545]). Each unit contains one American Depositary Shares ("ADS") (or ADS equivalent) and one warrant to purchase one ADS. Each ADS represents one ordinary share, no par value, of Kitov. The ADSs (or ADS equivalents) and warrants included in the units can only be purchased together in this offering, but will be issued separately and will be immediately separable upon issuance.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The warrants will have an exercise price of $0.325 per ADS and will be exercisable at any time after the date of issuance and will expire five years from the date of issuance.

The closing of the sale of the securities is expected to take place on or about March 16, 2020, subject to satisfaction of customary closing conditions.

Kitov intends to use the net proceeds of this offering to fund the development of its oncology drug candidates, acquisition of new assets and for general working capital purposes.

The offering is being made under an effective registration statement on Form F-1 (File No. 333-235729) filed with the Securities and Exchange Commission (the "SEC") and declared effective on March 11, 2020. The offering is being made only by means of a prospectus forming part of the effective registration statement. The final terms of the offering will be disclosed in a final prospectus to be filed with the SEC and made available on the SEC’s website at www.sec.gov. When available, electronic copies of the final prospectus relating to the offering may also be obtained by contacting H.C. Wainwright & Co., 430 Park Avenue, New York, NY 10022, by telephone at (646) 975-6996 or by email at [email protected]. This press release shall not constitute an offer to sell or a solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

On March 12, 2020 The National Foundation for Cancer Research (NFCR)reported that it has recently been approved by the U.S. Food and Drug Administration (FDA) for the initiation of human clinical trials. The regulatory body accepted the Investigational New Drug (IND) application for ST-001, a unique intravenous (IV) nano-delivery system of the off-patent synthetic chemical compound fenretinide, for the experimental treatment of patients with T-cell non-Hodgkin’s lymphoma.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Fenretinide is a small-molecule which past studies have shown to be both safe and have cancer mitigating qualities. However, heretofore it has not been approved as a cancer therapeutic, largely due to the challenges of delivering it in adequate doses to tumor cells.

The molecule is highly hydrophobic, meaning poorly soluble in water. Therefore, the clinical use of fenretinide as a cancer therapeutic by itself is limited—that is, unless delivered to cancer cells in a manner via which the molecule’s insolubility will no longer be problematic. Existing technology has not yet solved this problem.

However, funded in part by an NFCR grant, a unique nanoparticle-based IV delivery system has been developed which shows promise in being able to address the insolubility challenge and administer therapeutically effective high doses of fenretinide. Phase I clinical trials for its safety are expected to begin later this year at the Rush University Medical Center in Chicago.

NFCR’s translational research support helped advance the cancer treatment technology, developed by scientists including the U.S. National Cancer Institute’s Ralph Parchment, Ph.D., to this crucial IND acceptance stage. Additionally, optimism for the prospect of the upcoming trials’ successful outcome is justified by earlier research on fenretinide conducted and published by multiple scientists, including Dartmouth College’s Michael Sporn, M.D., a former NFCR project director, showing the drug to be safe in humans after long periods of use.

"The acceptance by the FDA of ST-001’s IND application underscores the importance of research—basic and translational—in the battle against not only lymphoma, but all forms of cancer," said NFCR President and CEO Sujuan Ba, Ph.D. "It’s only novel research that can result in game-changing treatments for patients."