On March 6, 2025 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company" or "Plus Therapeutics"), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Rhenium (186Re) Obisbemeda for the treatment of leptomeningeal metastases (LM) in patients with lung cancer (Press release, Plus Therapeutics, MAR 6, 2025, View Source [SID1234650984]).

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"Receiving Orphan Drug Designation for Rhenium (186Re) Obisbemeda marks a significant milestone in our efforts to develop a much-needed therapy for lung cancer patients with leptomeningeal metastases," said Mike Rosol, Ph.D., Plus Therapeutics Chief Development Officer. "These patients currently have limited treatment options, and the growing incidence of LM in lung cancer underscores the urgency for new therapies. This designation, in combination with our previously granted Fast Track designation, strengthens our pathway toward delivering an innovative, targeted radiotherapeutic solution for this highly underserved patient population."

The FDA grants ODD status to an investigational drug or biologic intended to prevent, diagnose, or treat a rare disease or condition affecting fewer than 200,000 people in the United States. ODD provides certain benefits to drug developers, including seven potential years of market exclusivity, tax credits for qualified clinical trials, and exemptions from significant regulatory fees, including the Prescription Drug User Fee Act (PDUFA) charge of $4.3 million in 2025 and the Pediatric Research Equity Act (PREA) requirements.

This milestone follows the recent completion of the ReSPECT-LM Phase 1 single-dose trial, which established the recommended Phase 2 dose (RP2D). The Company is now advancing a Phase 2 single-dose expansion trial and a Phase 1 multiple-dose trial while actively engaging the FDA to define the optimal pivotal trial strategy.

Additional details on the ReSPECT-LM trial can be found here.

About Leptomeningeal Metastases (LM)

LM is a rare complication of cancer in which the primary cancer spreads to the cerebrospinal fluid (CSF) and leptomeninges surrounding the brain and spinal cord. All malignancies originating from solid tumors, primary brain tumors, or hematological malignancies have this LM complication potential with breast cancer as the most common cancer linked to LM, with 3-5% of breast cancer patients developing LM. Additionally, lung cancer, GI cancers and melanoma can also spread to the CSF and result in LM. LM occurs in approximately 5% of people with cancer and is usually terminal with 1-year and 2-year survival of just 7% and 3%, respectively. The incidence of LM is on the rise, partly because cancer patients are living longer and partly because many standard chemotherapies cannot reach sufficient concentrations in the spinal fluid to kill the tumor cells, yet there are no FDA-approved therapies specifically for LM patients, who often succumb to this complication within weeks to several months, if untreated.

About Rhenium (186Re) obisbemeda
Rhenium (186Re) obisbemeda is a novel injectable radiotherapy specifically formulated to deliver highly targeted high dose radiation in CNS tumors in a safe, effective and convenient manner to optimize patient outcomes. Rhenium (186Re) obisbemeda has the potential to reduce risks and improve outcomes for CNS cancer patients, versus currently approved therapies, with a more targeted and potent radiation dose. Rhenium-186 is an ideal radioisotope for CNS therapeutic applications due to its short half-life, beta energy for destroying cancerous tissue and gamma energy for live imaging. Rhenium (186Re) obisbemeda is being evaluated for the treatment of recurrent glioblastoma and leptomeningeal metastases in the ReSPECT-GBM and ReSPECT-LM clinical trials. ReSPECT-GBM is supported by an award from the National Cancer Institute (NCI), part of the U.S. National Institutes of Health (NIH), and ReSPECT-LM is funded by a three-year $17.6M grant by the Cancer Prevention & Research Institute of Texas (CPRIT).

On March 6, 2025 Cidara Therapeutics, Inc. (Nasdaq: CDTX) (the Company), a biotechnology company using its proprietary Cloudbreak platform to develop drug-Fc conjugate (DFC) immunotherapies, reported financial results for the fourth quarter and full year ended December 31, 2024 and provided recent business updates (Press release, Cidara Therapeutics, MAR 6, 2025, View Source [SID1234650980]).

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"2024 was a transformational year for Cidara as we reacquired rights to the CD388 program, our long-acting, universal influenza drug, and raised $240.0 million in April to conduct the 5,000 subject Phase 2b NAVIGATE trial evaluating CD388 for the single-dose prevention of seasonal influenza. We raised an additional $105.0 million with new and existing investors in November to support our continued development efforts," said Jeffrey Stein, Ph.D., president and chief executive officer of Cidara. "Based on these collective achievements and the promising CD388 clinical data generated to date, we believe we are well-positioned to continue advancing CD388 as a potential long-acting, universal influenza preventative. We look forward to continuing this momentum in 2025 and sharing additional important milestones and inflection points on our clinical programs throughout the year."

Recent Corporate Highlights

Completed enrollment of Phase 2b NAVIGATE trial evaluating CD388 for prevention of seasonal influenza. In December 2024, Cidara announced that it had reached full planned enrollment of at least 5,000 subjects in the Phase 2b NAVIGATE trial across clinical sites in the U.S. and UK. The randomized, double-blind, controlled Phase 2b trial is designed to evaluate the efficacy and safety of CD388, the Company’s DFC for the pre-exposure prophylaxis of seasonal influenza. Three CD388 dose groups and one placebo group were randomized in a 1:1:1:1 ratio and administered CD388 at the beginning of the 2024-25 influenza season. Subjects will be followed for the remainder of the 2024-25 influenza season to monitor for breakthrough cases. Rates of laboratory and clinically confirmed influenza will be compared between subjects receiving the various single doses of CD388 or placebo.

Potential early analysis of efficacy data in the first half of 2025. Given the severity of the 2024-25 flu season, we may consider a potential early analysis of efficacy data from the ongoing CD388 Phase 2b NAVIGATE study in the first half of 2025. This would potentially enable the initiation of a Phase 3 study during the 2025-26 Northern Hemisphere influenza season.

Closed $105.0 million private placement. In November 2024, Cidara entered into a securities purchase agreement with certain investors and raised $105.0 million in gross proceeds. The private placement was led by new investor, Venrock Healthcare Capital Partners, with significant participation by new and existing life sciences-focused investors, including RA Capital Management, TCGX, BVF Partners LP, Vivo Capital, Spruce Street Capital, Adage Capital Partners LP, and Checkpoint Capital.

Significantly expanded Equity Research Coverage. Guggenheim (Seamus Fernandez), Cantor (Eric Schmidt) and RBC (Gregory Renza) initiated coverage between November 2024 and January 2025 with Buy, Overweight and Outperform ratings, respectively.
Appointed Frank Karbe as Chief Financial Officer. In February 2025, Cidara announced the appointment of Frank Karbe as Chief Financial Officer. Mr. Karbe brings more than 25 years of leadership experience in the biopharma industry transitioning companies from research and development (R&D) to commercialization.

Fourth Quarter and Full Year 2024 Financial Results

Cash, cash equivalents and restricted cash totaled $196.2 million as of December 31, 2024, compared with $35.8 million as of December 31, 2023.

Collaboration revenue totaled zero and $1.3 million for the three months and full year ended December 31, 2024, respectively, compared to $2.8 million and $23.3 million for the same periods in 2023.

Collaboration revenue for the years ended December 31, 2024 and 2023 related to the achievement of milestones and ongoing R&D and clinical supply services provided to J&J Innovative Medicine, previously Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen), under our license and collaboration agreement with Janssen (the Janssen Collaboration Agreement). The Janssen Collaboration Agreement was terminated upon the effectiveness of our license and technology transfer agreement with Janssen (the Janssen License Agreement) on April 24, 2024, pursuant to which we re-acquired the rights to CD388.
Acquired in-process research and development expenses were $84.9 million for year ended December 31, 2024 and related to an upfront payment of $85.0 million paid to Janssen under the Janssen License Agreement, on April 24, 2024, plus $0.4 million in direct transaction costs, offset by a settlement gain of $0.5 million to settle the preexisting Janssen Collaboration Agreement relationship.

R&D expenses were $46.9 million and $71.9 million for the three months and full year ended December 31, 2024, respectively, compared to $8.0 million and $36.8 million for the same periods in 2023. The increase in R&D expenses is primarily due to higher expenses associated with our CD388 Phase 2b NAVIGATE study and higher personnel costs, including $1.2 million for severance payments and employee benefits incurred related to a reduction in force, offset by lower nonclinical expenses associated with our Cloudbreak platform.

General and administrative (G&A) expenses were $7.3 million and $20.6 million for the three months and full year ended December 31, 2024, respectively, compared to $3.4 million and $13.6 million for the same periods in 2023. The increase in G&A expenses is primarily due to higher audit fees and legal costs associated with our corporate transactions during 2024, as well as higher personnel costs.

On April 24, 2024, we entered into an asset purchase agreement with Napp Pharmaceutical Group Limited (Napp), an affiliate of Mundipharma Medical Company, pursuant to which we sold to Napp all of our rezafungin assets and related contracts. We completed all conditions of the sale on April 24, 2024 and classified the sale of rezafungin as discontinued operations. Accordingly, we have separately reported the financial results of rezafungin as discontinued operations in the consolidated statements of operations and comprehensive loss for all periods presented. Income from discontinued operations was $0.1 million and $0.5 million for the three months and full year ended December 31, 2024, respectively, compared to $5.0 million and $2.1 million for the same periods in 2023.
Net loss was $52.3 million and $169.8 million for the three months and full year ended December 31, 2024, respectively, compared to $3.2 million and $22.9 million for the same periods in 2023.

As previously reported, on January 1, 2025, Renovaro, Inc., a Delaware corporation ("Renovaro"), entered into binding letter of intent (the "LOI") with Predictive Oncology Inc., a Delaware corporation ("Predictive Oncology"), with respect to the proposed acquisition of all of the capital stock of Predictive Oncology by Renovaro (the "Transaction"). On February 28, 2025, Renovaro entered into an extension agreement with Predictive Oncology (the "Extension Agreement"), pursuant to which the parties amended the LOI to (i) eliminate Renovaro’s obligation to acquire certain shares of Predictive Oncology’s common stock and (ii) extend the outside termination date of the LOI from February 28, 2025 to March 31, 2025 (Filing, Renovaro Biosciences, MAR 6, 2025, View Source [SID1234650973]). Additionally, pursuant to the Extension Agreement, Renovaro acquired 467,290 shares of Predictive Oncology’s common stock for an aggregate purchase price of $500,000 and agreed to purchase an additional 901,298 shares of Predictive Oncology common stock for an aggregate of $964,389 upon, and subject to, the execution of a definitive agreement in respect of the Transaction.

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The foregoing description of the Extension Agreement is only a summary and is qualified in its entirety by reference to the complete text of the Extension Agreement, which is filed as Exhibit 10.1 to this Current Report on Form 8-K and incorporated by reference in this Item 1.01.

Additional Information and Where to Find It:

This communication may be deemed to relate to a proposed acquisition of Predictive Oncology by Renovaro. In connection with the proposed acquisition, Renovaro and Predictive Oncology intend to file relevant materials with the Securities and Exchange Commission (SEC), including a Registration Statement on Form S-4 to be filed by Renovaro that will include a preliminary proxy statement of Predictive Oncology and also constitute a prospectus with respect to the shares of equity securities of Renovaro to be issued in the proposed transaction. The information in the preliminary proxy statement/prospectus will not be complete and may be changed. Predictive Oncology will deliver the definitive proxy statement to its stockholders as required by applicable law. This communication is not a substitute for any prospectus, proxy statement or any other document that may be filed with the SEC in connection with the proposed business combination.

INVESTORS AND SECURITY HOLDERS ARE URGED TO READ THE DEFINITIVE PROXY STATEMENT/PROSPECTUS AND OTHER DOCUMENTS FILED WITH THE SEC CAREFULLY AND IN THEIR ENTIRETY WHEN THEY BECOME AVAILABLE BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED TRANSACTION.

Investors and security holders will be able to obtain these materials (when they are available) and other documents filed with the SEC free of charge at the SEC’s website, www.sec.gov. Copies of documents filed with the SEC by Renovaro (when they become available) may be obtained free of charge at Renovaro’s website at renovarogroup.com. Copies of documents filed with the SEC by Predictive Oncology (when they become available) may be obtained free of charge on Predictive Oncology’s website at predictive-oncology.com.

Participants in the Solicitation:

Predictive Oncology and its directors, executive officers and certain other members of management and employees may be deemed to be participants in the solicitation of proxies in respect of the proposed transaction. Information regarding these persons who may, under the rules of the SEC, be considered participants in the solicitation of Predictive Oncology stockholders in connection with the proposed transaction and their interests in the transaction will be set forth in the proxy statement/prospectus described above filed with the SEC. Additional information regarding Predictive Oncology’s executive officers and directors is included in Predictive Oncology’s annual report on Form 10-K for the year ended December 31, 2023 filed with the SEC on March 28, 2024 and Predictive Oncology’s proxy statement for its 2024 annual meeting of stockholders filed with the SEC on November 27, 2024. These documents may be obtained free of charge at the SEC’s website, www.sec.gov, or Predictive Oncology’s website, predictive-oncology.com.

On March 6, 2025 RAPT Therapeutics, Inc. (Nasdaq: RAPT) ("RAPT" or the "Company") is a clinical-stage immunology-based biopharmaceutical company focused on discovering, developing and commercializing novel therapies for patients living with inflammatory and immunological diseases, reported financial results for the fourth quarter and year ended December 31, 2024 (Press release, RAPT Therapeutics, MAR 6, 2025, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-reports-fourth-quarter-and-full-year-2024 [SID1234650972]).

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"Our focus for 2025 will be on advancing development of RPT904, a novel, potential best-in-class option to treat the large and underserved population of patients suffering from food allergy and chronic spontaneous urticaria," said Brian Wong, President and CEO of RAPT. "We believe RPT904 can be a differentiated product to treat these diseases by targeting IgE, an approach validated by omalizumab. We expect to initiate a Phase 2b clinical trial for RPT904 in food allergy in the second half of 2025 and await clinical data later this year from our partner Jemincare to guide our development strategy in CSU."

Financial Results for the Fourth Quarter and Year Ended December 31, 2024

Fourth Quarter Ended December 31, 2024

Net loss for the fourth quarter of 2024 was $53.2 million, compared to $30.9 million for the fourth quarter of 2023.

Research and development expenses for the fourth quarter of 2024 were $46.5 million, compared to $26.8 million for the same period in 2023. The increase in research and development expenses was primarily due to the $35.0 million upfront license fee for RPT904, partially offset by lower development costs related to zelnecirnon, tivumecirnon and early-stage programs, as well as decreased expenses for personnel, professional services, non-cash stock-based compensation and lab supplies.

General and administrative expenses for the fourth quarter of 2024 were $8.0 million, compared to $6.5 million for the same period in 2023. The increase in general and administrative expenses was primarily due to increases in expenses for professional services, non-cash stock-based compensation, personnel and facilities.

In December 2024, the Company entered into a license agreement with Shanghai Jemincare Pharmaceutical Co., Ltd. ("Jemincare"), a company incorporated in the People’s Republic of China, under which the Company obtained exclusive rights to RPT904 throughout the world, excluding mainland China, Hong Kong, Macau and Taiwan. As consideration for those rights, the Company paid a $35.0 million upfront license fee and could pay up to $672.5 million in additional milestone payments, as well as tiered royalty payments (at percentages ranging from high single-digit to low double-digit) on future net sales.

Also in December 2024, the Company sold through a private placement to a select group of accredited investors 100,000,000 shares of common stock at a price of $0.85 per share and pre-funded warrants to purchase 76,452,000 shares of common stock at a purchase price of $0.8499 per pre-funded warrant, resulting in net proceeds of $143.0 million after deducting offering expenses.

Year Ended December 31, 2024

Net loss for the year ended December 31, 2024 was $129.9 million, compared to $116.8 million for the same period in 2023.

Research and development expenses for the year ended December 31, 2024 were $107.2 million, compared to $101.0 million for the same period in 2023. The increase in research and development expenses was primarily due to the $35.0 million upfront license fee for RPT904 and an increase in non-cash stock-based compensation expense, partially offset by lower development costs related to zelnecirnon, tivumecirnon and early-stage programs, as well as decreased expenses for personnel, professional services and lab supplies.

General and administrative expenses for the year ended December 31, 2024 were $28.9 million, compared to $26.1 million for the same period in 2023. The increase in general and administrative expenses was primarily due to increased expenses for non-cash stock-based compensation, consultants, personnel, and facilities, partially offset by decrease in expenses for insurance premiums.

As of December 31, 2024, the Company had cash and cash equivalents and marketable securities of $231.1 million.

On March 6, 2025 NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class, and best-in-class therapies to treat cancer, reported a business update and announced full year 2024 financial results (Press release, NextCure, MAR 6, 2025, View Source [SID1234650971]).

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"In 2024 we reprioritized our resources to advance our antibody-drug conjugate ("ADC") program and recently completed cohort 1 of the Phase 1 study evaluating LNCB74 as a potential therapeutic for treating multiple cancers. We look forward to additional progress in 2025, including initiating backfill cohorts in the second half of the year," said Michael Richman, NextCure’s president and CEO.

Business Highlights and Near-Term Milestones

LNCB74 (B7-H4 ADC)

● Presented preclinical data from LNCB74 (B7-H4 ADC) at the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in November 2024 to highlight its potential as a therapeutic for treating multiple solid tumor indications.
● The U.S. Food and Drug Administration accepted an Investigational New Drug (IND) application in December 2024.
● Dosed our first patient in January 2025 in the Phase 1 trial, clearing cohort 1 in February 2025 and currently dosing cohort 2.
● We plan to initiate backfill cohorts in the second half of 2025.

Preclinical Non-Oncology Programs Seeking Partnering

● Preclinical data for NC181 (ApoE4), a humanized antibody for the treatment of Alzheimer’s disease, has demonstrated amyloid clearance, prevention of amyloid deposition, plaque clearance and reduced neuroinflammation.

● Preclinical data for NC605 (Siglec-15), a humanized antibody for the treatment of osteogenesis Imperfecta (OI), has demonstrated that NC605 treatment reduced bone loss and enhanced bone quality in mice with OI.
● Both programs could lead to IND filings within 12 to 18 months if financial support from partners or third parties is secured.

Financial Results for Full Year Ended December 31, 2024

● Cash, cash equivalents, and marketable securities as of December 31, 2024 were $68.6 million as compared to $108.3 million as of December 31, 2023. The decrease of $39.7 million was primarily due to cash used to fund operations. We expect current financial resources to fund operating expenses and capital expenditures into the second half of 2026.
● Research and development expenses were $41.5 million for the full year ended December 31, 2024, as compared to $47.9 million for the full year ended December 31, 2023. Higher net costs on the LNCB74 program were more than offset by lower costs on other programs and preclinical development and lower personnel-related costs.
● General and administrative expenses were $15.7 million for the full year ended December 31, 2024, as compared to $19.7 million for the full year ended December 31, 2023. The decrease of $4.0 million was primarily related to lower payroll, lower stock compensation expense and lower insurance costs.
● Net loss was $55.7 million for the full year ended December 31, 2024, as compared to a net loss of $62.7 million for the full year ended December 31, 2023.