On March 22, 2024 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported its financial results for the fiscal year ended December 31, 2023 (Press release, Moleculin, MAR 22, 2024, View Source [SID1234641389]). As previously announced, the Company will host a conference call and live audio webcast, Monday, March 25, 2024, at 8:30 AM ET (details below), issuing a separate clinical trial update press release just prior.

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"Over the course of 2023 we established a significant growing body of positive clinical and encouraging safety data for Annamycin that continues to strengthen our confidence in the potential of our next generation, non-cardiotoxic chemotherapy in the treatment landscape for hard-to-treat cancers and viruses," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Building off of our momentum, we are laser focused on driving our priority pipeline programs toward key data milestones in the near term and believe we are well-positioned to achieve clinical and regulatory milestones that will translate into significant value creation for all of our stakeholders."

Recent Highlights

Announced 2023 year-end Annamycin clinical trials preliminary data and 2024 expectations for multiple data readouts and transition to pivotal Phase 2B/3;
Closed a registered direct offering priced at-the-market under Nasdaq rules with a single healthcare-focused institutional investor and certain of the Company’s executive officers, a board member and concurrent private placement for gross proceeds of $4.5 million before deducting the placement agent’s fees and other estimated offering expenses payable by the Company pushing the Company’s cash runway into the fourth quarter of 2024; and
Presented positive interim data from the Company’s ongoing European Phase 1B/2 clinical trial evaluating Annamycin for the treatment of AML (MB-106) to key opinion leaders and current investigators at a meeting held in conjunction with the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting and Exposition (ASH) (Free ASH Whitepaper) in San Diego, CA.
Summary of Financial Results for the Full Year 2023

Research and development (R&D) expenses were $19.5 million and $19.0 million for the years ended December 31, 2023 and 2022, respectively. The increase in R&D of $0.5 million is mainly related to the $1.5 million WPD sublicense termination, which enabled the reacquisition of the Company’s intellectual property rights in certain territories, including parts of the European Union. This was offset by $1.0 million in costs related to the timing of costs incurred for clinical trials and sponsored research.

General and administrative (G&A) expenses were $10.0 million and $11.5 million for the years ended December 31, 2023 and 2022, respectively. The decrease in G&A of $1.5 million was mainly attributable to a decrease in regulatory and legal services, and consulting & advisory fees.

The net loss for the year ended December 31, 2023 was $29.8 million, which included a non-cash loss of $1.0 million on warrants in 2023 as compared to a gain of $1.3 million in the prior year and approximately $2.0 million of stock-based compensation expense in 2023 as compared to $2.3 million in 2022.

As of December 31, 2023, the Company had cash and cash equivalents of $23.6 million. The Company believes that this cash is sufficient to meet its projected operating requirements into the fourth quarter of 2024.

Conference Call and Webcast

Moleculin management will host its quarterly conference call and webcast for investors, analysts, and other interested parties Monday, March 25, 2024, at 8:30 AM ET.

Interested participants and investors may access the conference call by dialing (877) 407-0832 (domestic) or (201) 689-8433 (international) and referencing the Moleculin Biotech Conference Call. The live webcast will be accessible on the Events page of the Investors section of the Moleculin website, moleculin.com, and will be archived for 90 days.

On March 22, 2024 LIXTE Biotechnology Holdings, Inc. ("LIXTE" or the "Company") (Nasdaq: LIXT and LIXTW), a clinical-stage pharmaceutical company developing a new class of cancer therapy to enhance chemotherapy and immunotherapy, reported an update on the Company’s progress with its proprietary compound, LB-100 (Press release, Lixte Biotechnology, MAR 22, 2024, View Source [SID1234641386]).

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"Clinical trials are underway at leading cancer centers in the United States and Spain using LB-100 in combination with chemotherapy and immunotherapy that we are hopeful will provide new and effective treatment options for patients with lung, ovarian and sarcoma cancers," said Bas van der Baan, LIXTE’s President and Chief Executive Officer.

Clinical trials are taking place at the University of Texas MD Anderson Cancer (ovarian clear cell carcinoma); City of Hope Cancer Center and the Sarah Cannon Research Institute (small cell lung cancer); and the Spanish Sarcoma Group (advanced soft tissue sarcoma).

Click here for a brief video overview by LIXTE’s Chief Executive Officer.

LIXTE recently announced the following significant developments:

■ The dosing of the first patient in a Phase 1b/2 clinical trial to assess whether adding LB-100 to GSK’s dostarlimab-gxly will enhance the effectiveness of immunotherapy in the treatment of ovarian clear cell carcinoma (OCCC). The clinical trial was initiated by, and is being conducted at, the University of Texas MD Anderson Cancer Center. LIXTE is providing LB-100, and GSK is providing dostarlimab-gxly and financial support.

● The signing of an exclusive patent license agreement with the National Institute of Neurological Disorders and Stroke and the National Cancer Institute, each an Institute of the National Institutes of Health (NIH). LIXTE has licensed NIH’s intellectual property rights under a Cooperative Research and Development Agreement that, focused on LB-100 promoting anti-cancer activity alone and in combination with immune checkpoint inhibitors.

● On February 28, 2024, René Bernards, PhD, a leader in the field of molecular carcinogenesis and Senior Staff Scientist at the Netherlands Cancer Institute, presented new pre-clinical data at the Joint Conference of European and American Associations for Cancer Research in Dublin, Ireland on how stress imposed onto colon cancer cells by LB-100 drives cancer cells to evolve to less cancerous behavior, underscoring a unique mechanism of action of LB-100. Dr. Bernards is a member of the Board of Directors of LIXTE.

On March 22, 2024, Lineage Cell Therapeutics, Inc. (the "Company" or "Lineage"), reported to have entered into a sales agreement (the "Sales Agreement") with B. Riley Securities, Inc. (the "Sales Agent"), under which the Company may offer and sell its common shares from time to time through the Sales Agent as the Company’s sales agent (Press release, Lineage Cell Therapeutics, MAR 22, 2024, View Source [SID1234641384]). Sales of the Company’s common shares through the Sales Agent, if any, will be made by any method permitted by law deemed to be an "at the market offering" as defined in Rule 415(a)(4) under the Securities Act of 1933, as amended (the "Securities Act"), including without limitation sales made directly on the NYSE American or any other existing trading market for the common shares. The Sales Agent will use commercially reasonable efforts to sell the Company’s common shares from time to time, based on instructions from the Company (including any price, time or size limits or other parameters or conditions the Company may impose). The Company will pay the Sales Agent a commission of up to 3.0% of the aggregate gross proceeds from the sales of common shares sold through the Sales Agent under the Sales Agreement. The Company also provided the Sales Agent with customary indemnification and contribution rights.

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The Company is not obligated to make any sales of common shares under the Sales Agreement. The offering of the Company’s common shares under the Sales Agreement will terminate upon the termination of the Sales Agreement in accordance with its terms.

The foregoing description of the Sales Agreement is not complete and is qualified in its entirety by reference to the full text of the Sales Agreement, a copy of which is filed as an exhibit to this report and is incorporated herein by reference. This report also incorporates by reference the Sales Agreement into the registration statement on Form S-3 described below.

The Company’s common shares are being offered and sold pursuant to the Company’s effective shelf registration statement on Form S-3 and an accompanying prospectus (Registration Statement No. 333- 254167) declared effective by the U.S. Securities and Exchange Commission (the "SEC") on March 19, 2021 (the "March 2021 Registration Statement") and pursuant to a prospectus supplement dated March 22, 2024. On March 7, 2024, the Company filed with the SEC a shelf registration statement on Form S-3 and an accompanying prospectus (Registration Statement No. 333-277758) (the "New Registration Statement"). Under Rule 415(a)(5) of the Securities Act, the Company’s securities registered under the March 2021 Registration Statement may be offered and sold only if not more than three years have elapsed since the initial effective date of such registration statement, provided, however, that because the New Registration Statement has been filed pursuant to Rule 415(a)(5) of the Securities Act and the New Registration Statement is not an automatic shelf registration statement, (i) securities covered by the March 2021 Registration Statement may continue to be offered and sold until the earlier of the effective date of the New Registration Statement or 180 days after the third anniversary of the initial effective date of the March 2021 Registration Statement; and (ii) a continuous offering of securities covered by the March 2021 Registration Statement that commenced within three years of the initial effective date of the March 2021 Registration Statement may continue until the effective date of the New Registration Statement if such offering is permitted under the New Registration Statement.

A copy of the opinion of Sheppard Mullin Richter & Hampton LLP regarding the common shares to be sold under the Sales Agreement is filed as an exhibit to this report.

On March 22, 2024 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 fourth quarter and full year 2023 (Press release, Hookipa Pharma, MAR 22, 2024, View Source [SID1234641382]).

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"It was a defining year for HOOKIPA as we witnessed the powerful potential of our novel arenaviral immunotherapies in action. Our HB-200 program delivered potentially best-in-class T cell activation and clinical activity, as this program, in combination with pembrolizumab, showed doubled objective response rate compared to historical standard of care treatment alone," said Joern Aldag, Chief Executive Officer of HOOKIPA. "Our progress last year has positioned us to execute in a meaningful way in 2024. Our data enable us to potentially bring a drug to patients who need new treatment options. We are excited for what is ahead for HOOKIPA, and we look forward to sharing more very soon."

Business Highlights and Recent Developments

Oncology

HB-200 in combination with pembrolizumab: positive preliminary Phase 2 data in patients with recurrent/metastatic HPV16+ head and neck cancers in the first line setting. Data was initially presented in May 2023 and additional patient data was provided in October at the European Society for Medical Oncology Congress 2023.
Data from the ongoing study (NCT04180215), showed a 42 percent objective response rate for 19 evaluable checkpoint inhibitor (CPI)-naïve patients treated with HB-200 in combination with pembrolizumab. These data represent a doubling of the historical response rate (19 percent) reported for pembrolizumab alone.
HOOKIPA is preparing to start a randomized Phase 2/3 trial of HB-200 in combination with pembrolizumab in the 1st-line setting for patients with recurrent/metastatic HPV16+ head and neck cancers.

HOOKIPA’s HB-700 program is a novel arenaviral immunotherapy for KRAS-mutated cancers, including the five mutations that are the primary causes of lung, pancreatic and colon cancers.
On January 25, 2024, the Company received notification from Roche of their decision to terminate the collaboration and licensing agreement for HOOKIPA’s HB-700 program. To date, HOOKIPA has met all go-forward criteria under the agreement and remains eligible for a final milestone payment associated with IND submission. HB-700 is on-track to an expected IND application filing by April 2024.
Effective April 25, 2024, HOOKIPA will regain full control of the associated intellectual property portfolio and have full collaboration and licensing rights for this program.

HOOKIPA enrolled the dose escalation cohorts in the Phase 1/2 study of HB-300 (NCT05553639) for the treatment of advanced prostate cancer.
The Study Safety Committee deemed that HB-300 was generally safe and well-tolerated in both dose escalation cohorts.
Initial analysis of target antigen-specific T cell responses—using direct ELISPOT without pre-expansion of T cells—in ten patients between dose level 1 (N=5) and dose level 2 (N=5) indicated a 15- to 26-fold increase of target antigen specific T cells in 30 percent of patients (3/10).
In line with its previously announced strategy to prioritize the development of HB-200, the Company has terminated the Phase 1/2 study of HB-300 and will utilize the associated capital and resources for the advancement of its HB-200 program. HOOKIPA will keep the IND open to allow the potential for further development of this program in the future.
HOOKIPA will publish the final data at a scientific conference, once complete.
Infectious Disease

In August, The Journal of Infectious Disease, published peer-reviewed preclinical data on HB-400, an investigational therapeutic vaccine for chronic hepatitis B (HBV). The data show that HB-400 induced robust, HBV-specific T cell and antibody responses in non-human primates and cleared detectable serum HBV antigens in a mouse model for chronic HBV infection, with near elimination of detectable HBV antigen positive hepatocytes in the liver.
HB-400 (NCT05770895) is currently being evaluated in a Phase 1 trial and is one of two independent development programs in HOOKIPA’s collaboration and license agreement with Gilead Sciences, Inc. Gilead is solely responsible for further development and commercialization of the HBV product candidate.
In November, HOOKIPA’s HB-500 program, an investigational therapeutic vaccine for the treatment of human immunodeficiency virus (HIV), also partnered with Gilead, received FDA clearance of its IND application.
Also in November, Nature Partner Journals Vaccines, published peer-reviewed preclinical data for the program. Data show that HB-500 was well tolerated and generated robust, high-quality and durable immune responses (antigen-specific T cells and antibodies) in non-human primates; and the arenaviral therapeutic vaccination significantly reduced SIV viral load and clinical illness in those animals compared to placebo.
HOOKIPA expects to initiate a Phase 1 clinical study of HB-500 in people with HIV in the second quarter of 2024. Under the collaboration agreement with Gilead, HOOKIPA is eligible for a milestone payment upon dosing the first patient in this study.
Corporate and Financial Updates

Corporate Highlights

The Company added two new Board Members in 2023: Malte Peters, M.D., in January and Terry Coelho in April.
Mark Winderlich, Ph.D., will join the Company on April 1, 2024, as Chief Development Officer to lead HOOKIPA’s clinical research and development organization.
On January 29, 2024, HOOKIPA provided an update on its business priorities and oncology partnership programs. The Company announced that it will focus its resources in two strategic areas: (1) prioritize the clinical development of a randomized trial for its HB-200 program and (2) its two Gilead-partnered infectious disease cure programs for hepatitis B and human immunodeficiency virus. As part of its strategic refocus, HOOKIPA will pause development activities related to HB-300 and most of its preclinical research activities. The Company also announced that it will regain full control of the intellectual property portfolio and will have full collaboration and licensing rights to the HB-700 program for KRAS-mutated cancers from Roche effective April 25, 2024.
Financial Highlights

In January 2023, HOOKIPA achieved a $5.0 million milestone payment under its HB-400 collaboration agreement with Gilead. The success-based milestone payment reflected the Company’s completion and delivery of a regulatory support package for Gilead’s Phase 1 clinical trial.
In February 2023, HOOKIPA achieved a $10.0 million milestone payment under its HB-700 collaboration agreement with Roche. The success-based milestone payment reflected the start of the HB-700 manufacturing process to support a Phase 1 clinical trial.
In June 2023, the Company completed a $50.0 million public offering of common stock and non-voting convertible preferred stock. The financing was completed in parallel with the initial Phase 2 data release for HB-200.
In December 2023, the Company issued 15 million shares of common stock for approximately $21.25 million, at a price of $1.4167 per share, to Gilead under an amended stock purchase agreement between the companies. HOOKIPA has the right, subject to certain terms and conditions, to sell an additional approximately $8.75 million of common stock to Gilead as pro-rata participation in potential future equity raises.
Anticipated Catalysts & Milestones

Program Indication Upcoming Anticipated Catalysts
Oncology Programs
HB-200 HPV16+ HNSCC
Additional Phase 2 first-line data for HB-200 in combination with pembrolizumab (2Q 2024)
Announcement of pivotal trial design post FDA-feedback (2Q 2024)
Pivotal study start 2024
HB-700 KRAS
IND submission, milestone payment (April 2024)
Publication of preclinical data (2Q 2024)

Infectious Disease Programs: Gilead-Partnered
HB-400 HBV
Gilead-led: Phase 1b actively enrolling
Next milestone: Initiation of Phase 2 (timing determined by Gilead)
HB-500 HIV
Initiation of Phase 1 trial; first patient dosed and associated milestone payment (2Q 2024)

Fourth Quarter and Full Year 2023 Financial Results

Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of December 31, 2023 was $117.5 million compared to $113.4 million as of December 31, 2022. The increase was primarily attributable to funds resulting from the follow-on financing in June 2023 and the Gilead stock purchase in December 2023, partly offset by cash used in operating activities.

Revenue: Revenue was $7.4 million and $20.1 million for the three and twelve months ended December 31, 2023, respectively, compared to $7.8 million and $14.2 million for the same periods in 2022. The increase for the twelve months ended December 31, 2023 was primarily due to higher partial recognition of the upfront and milestone payments under the Gilead and Roche collaborations and cost reimbursements for activities related to the preparation of a first human trial under the Roche collaboration, partially offset by lower cost reimbursements received under the Restated Gilead collaboration agreement. The decrease for the three months ended December 31, 2023 was primarily due to a $5.0 million research milestone that was recognized upon achievement related to the Gilead collaboration agreement, partially offset by higher partial recognition of the upfront and milestone payments under the Gilead and Roche collaborations.

Research and Development Expenses: HOOKIPA’s research and development expenses were $21.2 million and $86.4 million for the three and twelve months ended December 31, 2023, respectively, compared to $17.6 million and $68.6 million for the same periods in 2022. The primary drivers of the increase in research and development expenses were higher clinical study expenses for the HB-200 program and increased spending for the HB-700 program.

General and Administrative Expenses: General and administrative expenses amounted to $4.4 million and $18.6 million for the three and twelve months ended December 31, 2023, respectively, compared to $3.8 million and $18.8 million for the same periods in 2022.

Impairment Expenses: Impairment expenses amounted to $12.8 million for the three and twelve months ended December 31, 2023, respectively, and resulted from asset write-offs related to the Company’s GMP manufacturing facility project, that was discontinued as part of the Company’s strategic refocus and rebalance of its cost structure.

Net Loss: HOOKIPA’s net loss was $24.8 million and $81.6 million for the three and twelve months ended December 31, 2023, respectively, compared to $12.3 million and $64.9 million for the same periods in 2022. This increase was primarily due to an increase in research and development expenses and the impairment of the GMP manufacturing facility project.

On March 22, 2024 Gilead Sciences, Inc. (Nasdaq: GILD) reported the completion of the previously announced transaction to acquire CymaBay Therapeutics, Inc. (Nasdaq: CBAY) for approximately $4.3 billion in total equity value (Filing, 8-K, Gilead Sciences, MAR 22, 2024, View Source [SID1234641381]). The addition of CymaBay’s investigational lead product candidate, seladelpar for the treatment of primary biliary cholangitis (PBC) including pruritus, complements Gilead’s existing liver portfolio and aligns with its long-standing commitment to bringing transformational medicines to patients.

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"The acquisition of CymaBay brings us a potential best in disease therapy that could transform the treatment landscape for people with primary biliary cholangitis," said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences. "I want to thank the CymaBay team for their efforts and commitment to addressing this high unmet need. We look forward to advancing seladelpar and building on Gilead’s more than 20-year legacy of treating and curing liver disease on a global scale."

On February 12, 2024, Gilead and CymaBay announced that CymaBay, Gilead and Pacific Merger Sub, Inc., a wholly owned subsidiary of Gilead ("Purchaser"), had signed a definitive merger agreement pursuant to which a tender offer would be made. Pursuant to the merger agreement, Gilead and Purchaser commenced a tender offer on February 23, 2024, to acquire all outstanding shares of CymaBay at a price of $32.50 per share. On March 22, 2024, Gilead successfully completed the tender offer for all outstanding shares of common stock of CymaBay and accepted for payment all shares validly tendered and not withdrawn as of the expiration time of the tender offer, and Gilead will promptly pay for such shares, which shares represented approximately 77.3% of CymaBay’s outstanding shares (not including 5,095,996 shares delivered through Notices of Guaranteed Delivery, representing approximately 4.2% of the shares outstanding). Pursuant to the terms of the merger agreement, Purchaser merged with and into CymaBay on March 22, 2024. All outstanding shares of common stock of CymaBay, other than (i) shares owned by Gilead, Purchaser or any of Gilead’s direct or indirect wholly owned subsidiaries, (ii) shares owned by CymaBay, (iii) shares irrevocably accepted by Purchaser for purchase pursuant to the tender offer and (iv) shares held by CymaBay stockholders who properly demand appraisal for their shares under Delaware law, were cancelled and converted into the right to receive cash equal to the $32.50 price per share.

As a result of the completion of the merger, CymaBay has become a wholly owned subsidiary of Gilead and the common stock of CymaBay will no longer be listed for trading on the Nasdaq Global Select Market, which is expected to take effect as of the close of market on March 22, 2024.

This transaction is expected to be accounted for as an asset acquisition and reduce Gilead’s GAAP and non-GAAP 2024 EPS by approximately $3.10 – $3.20. Reflecting acquisition costs, associated operating expenses and lower interest income, we expect this transaction to reduce Gilead’s GAAP and non-GAAP 2024 EPS by approximately $3.35 – $3.45 relative to the full year 2024 guidance shared on February 6, 2024.

Seladelpar is an investigational product that has not been approved for use anywhere globally, and its safety and efficacy have not been established.