On December 27, 2024 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported that it has entered into a binding term sheet for a proposed merger with Oqory, Inc., a privately-held, clinical-stage company developing ADCs for the treatment of multiple oncology indications (Press release, Vincerx Pharma, DEC 27, 2024, View Source [SID1234649332]).

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Upon completion of the proposed merger, Oqory, Inc. will merge with Vincerx Pharma, Inc. Post-closing, Oqory equity holders are expected to own approximately 95% of the combined entity, while Vincerx equity holders will hold about 5%. The transaction includes a minimum fully diluted equity value of $13.66 million for existing Vincerx stockholders at closing and, as a condition to the closing of the merger, completion of a concurrent offering of Vincerx equity securities of at least $20 million. Additionally, Oqory-designated investors will provide interim financing to Vincerx of $1.5 million in two tranches, approximately $1,000,000 of which was funded today through the issuance of common stock and pre-funded warrants along with accompanying common stock warrants and approximately $500,000 of which will be funded on or prior to January 31, 2025. The merger is subject to customary closing conditions, including due diligence, regulatory approvals, negotiation of a definitive merger agreement, stockholder approval from both parties, completion of the minimum $20 million financing, and the continued listing of Vincerx’s common stock on Nasdaq.

Vincerx is also implementing additional streamlining and cost-control measures, including a workforce reduction, as it pursues due diligence and transaction-related work. As part of this workforce reduction, Dr. Ahmed Hamdy, Chairman and Chief Executive Officer (CEO), has stepped down as CEO but will remain as Chairman. Dr. Raquel Izumi has stepped down as President and Chief Operations Officer and taken over as Acting CEO in a consulting capacity. Alexander Seelenberger has stepped down as Chief Financial Officer, and Kevin Hass, the Company’s Vice President and Controller, has taken over as Acting Chief Financial Officer. Mr. Seelenberger has agreed to provide ongoing assistance in a consulting capacity to assist the Company as it pursues its strategic efforts.

"This strategic transaction highlights Vincerx’s commitment to develop ADCs with improved safety profiles that allow patients to thrive on—rather than endure—their cancer therapies," said Raquel Izumi, Ph.D., Acting Chief Executive Officer. "Oqory’s anti-TROP2 ADC has shown favorable efficacy and safety in the clinic. Among approximately 150 treated patients, results include an 83% overall response rate and 100% disease control rate in first-line triple-negative breast cancer (TNBC; n=30). Unlike other TROP2 ADCs in Phase 3, no cases of interstitial lung disease or Grade 3 and above stomatitis have been reported. Oqory’s Phase 3 studies of OQY-3258 are ongoing to confirm these promising findings."

About OQY-3258 (also known as ESG401)
OQY-3258 is Oqory’s anti-TROP2 ADC with an optimized enzyme-dependent linker technology and an SN-38 payload with established efficacy and manageable side effect profile. OQY-3258 has completed Phase 1/2 development in over 150 patients with solid tumors, including metastatic HR+/HER2- and triple-negative breast cancer. OQY-3258 has shown efficacy in these patients, including reduction of brain metastasis and responses in heavily pretreated patients. To date, OQY-3258 has exhibited a differentiated safety profile vs. Trodelvy and other TROP2 ADCs in Phase 3 development. Notably, no interstitial lung disease or ocular surface events have been observed. Gastrointestinal effects have been mild and mainly Grade 1/2. Neutropenia and leukopenia have been the major AEs, which were manageable and did not result in discontinuation of study drug. OQY-3258 is being evaluated in a Phase 3 study as first-line treatment in patients with unresectable recurrent or metastatic triple-negative breast cancer (NCT06732323) and in a Phase 3 study in patients with unresectable locally advanced or metastatic HR+/HER2- breast cancer (NCT06383767).

On December 27, 2024 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical-stage biotechnology company that develops therapeutics using its INTASYL siRNA gene silencing technology to make the body’s immune cells more effective in killing cancer cells, reported that it has entered into definitive agreements for the purchase and sale of an aggregate of 240,000 shares of its common stock at a purchase price of $2.00 per share in a registered direct offering priced at-the-market under Nasdaq rules (Press release, Phio Pharmaceuticals, DEC 27, 2024, View Source [SID1234649331]). In addition, in a concurrent private placement, the Company will issue unregistered warrants to purchase up to 240,000 shares of common stock. The warrants will have an exercise price of $2.00 per share, will be exercisable upon issuance and expire five years following the date of issuance. The closing of the offering is expected to occur on or about December 24, 2024, subject to the satisfaction of customary closing conditions.

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

The aggregate gross proceeds to the Company from the offering are expected to be $0.48 million, before deducting the placement agent fees and other offering expenses payable by the Company. The Company currently intends to use the net proceeds from the offering for working capital and other general corporate purposes.

The shares of common stock (but not the warrants issued in the private placement or the shares of common stock underlying such warrants) are being offered by the Company pursuant to a "shelf" registration statement on Form S-3 (File No. 333-279557) filed with the Securities and Exchange Commission ("SEC") on May 20, 2024 and became effective on July 1, 2024. The registered direct offering of the shares of common stock is being made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. The prospectus supplement and the accompanying prospectus relating to the shares of common stock being offered in the registered direct offering will be filed with the SEC and be available at the SEC’s website at www.sec.gov. Electronic copies of the prospectus supplement and the accompanying prospectus relating to the registered direct offering may also be obtained, when available, by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by telephone at (212) 856-5711 or e-mail at [email protected].

The warrants described above are being issued in a concurrent private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Securities Act"), and Regulation D promulgated thereunder and, along with the shares of common stock underlying the warrants, have not been registered under the Securities Act, or applicable state securities laws. Accordingly, the warrants and underlying shares of common stock may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, 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 December 27, 2024 Outlook Therapeutics, Inc. (Nasdaq: OTLK), a biopharmaceutical company that achieved regulatory approval in the European Union and the United Kingdom earlier this year for the first authorized use of an ophthalmic formulation of bevacizumab for the treatment of wet age-related macular degeneration (wet AMD), reported financial results for fiscal year 2024 and provided a corporate update (Press release, Outlook Therapeutics, DEC 27, 2024, View Source [SID1234649330]).

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"Over the course of the past year, our team has continued to execute and progress the development of ONS-5010/LYTENAVA in Europe and the United States. Following the receipt of our first positive reimbursement decision worldwide for LYTENAVA from NICE in the United Kingdom, our team continues to make preparations for commercial launch in the UK and Germany, which is expected in the first half of calendar 2025," commented Lawrence Kenyon, Chief Financial Officer and Interim Chief Executive Officer of Outlook Therapeutics. "We expect to receive the month 3 NORSE EIGHT efficacy data in January 2025 and are continuing preparations for the planned resubmission of our BLA in the first quarter of calendar 2025. We believe that 2025 holds significant opportunity for Outlook Therapeutics and we remain confident in the potential of ONS-5010/LYTENAVA to provide a meaningful impact globally for the treatment of wet AMD."

Upcoming Anticipated Milestones

Final efficacy data from NORSE EIGHT expected in January 2025;
Resubmission of the ONS-5010 BLA targeted for Q1 CY2025;
Initial commercial launches in Europe planned to commence in first half of CY2025; and
Potential for US FDA approval of ONS-5010 in second half of CY2025.
ONS-5010 / LYTENAVA (bevacizumab-vikg) Clinical and Regulatory Update

In May 2024, the European Commission granted Marketing Authorization for LYTENAVA (bevacizumab gamma) for the treatment of wet AMD in the EU. Additionally, in July 2024, the UK Medicines and Healthcare products Regulatory Agency (MHRA) granted Marketing Authorization for LYTENAVA (bevacizumab gamma) for the same indication in the UK. In December 2024, the National Institute for Health and Care Excellence (NICE) recommended LYTENAVA (bevacizumab gamma) as an option for the treatment of wet AMD. Plans for a potential 2025 launch in the UK and Germany are ongoing. Outlook Therapeutics remains confident that ONS-5010/ LYTENAVA is an important therapy for the treatment of wet AMD in place of off-label repackaged bevacizumab that has not received regulatory approval for use in retina diseases such as wet AMD.

Previously, the Company reported that in the NORSE EIGHT trial, ONS-5010 did not meet the pre-specified non-inferiority endpoint at week 8 set forth in the special protocol assessment (SPA) with the U.S. Food and Drug Administration (FDA). However, the preliminary data from the trial demonstrated an improvement in vision and the presence of biologic activity, as well as a continued favorable safety profile for ONS-5010. Analysis of the data is ongoing as the month 3 data from NORSE EIGHT is being collected, which is expected to be available in January 2025. Upon receipt of the full month 3 efficacy and safety results for NORSE EIGHT, Outlook Therapeutics plans to resubmit the BLA for ONS-5010 in the first quarter of calendar 2025.

LYTENAVA (bevacizumab gamma) is the first and only authorized ophthalmic formulation of bevacizumab for use in treating wet AMD in adults in the EU and UK and has an initial 10 years of market exclusivity. Authorization may also be sought in other European countries, Japan, and elsewhere. As part of a multi-year planning process, Outlook Therapeutics entered into a strategic collaboration with Cencora (formerly AmerisourceBergen) to support the commercial launch of LYTENAVA globally following regulatory approvals. The collaboration and integrated approach is designed to support market access and efficient distribution of LYTENAVA to benefit all stakeholders, including retina specialists, providers and patients.

In the EU and the UK and other regions outside of the US, Outlook Therapeutics is planning to commercialize LYTENAVA (bevacizumab gamma) directly and is also assessing potential licensing and partnering options. Additionally, if approved by the FDA, Outlook Therapeutics plans to commercialize ONS-5010/LYTENAVA (bevacizumab-vikg) directly in the US.

Financial Highlights for the 2024 Fiscal Year Ended September 30, 2024

For the fiscal year ended September 30, 2024, Outlook Therapeutics reported a net loss of $75.4 million, or $4.06 per basic and diluted share, compared to a net loss of $59.0 million, or $4.72 per basic and diluted share, for the prior fiscal year.

As of September 30, 2024, Outlook Therapeutics had cash and cash equivalents of $14.9 million.

About ONS-5010 / LYTENAVA (bevacizumab-vikg, bevacizumab gamma)

ONS-5010/LYTENAVA is an ophthalmic formulation of bevacizumab for the treatment of wet AMD. LYTENAVA (bevacizumab gamma) is the subject of a centralized Marketing Authorization granted by the European Commission in the European Union (EU) and Marketing Authorization granted by the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom (UK) for the treatment of wet age-related macular degeneration (wet AMD).

In the United States, ONS-5010/LYTENAVA (bevacizumab-vikg) is investigational and is being evaluated in an ongoing non-inferiority study for the treatment of wet AMD.

Bevacizumab-vikg (bevacizumab gamma in the EU and UK) is a recombinant humanized monoclonal antibody (mAb) that selectively binds with high affinity to all isoforms of human vascular endothelial growth factor (VEGF) and neutralizes VEGF’s biologic activity through a steric blocking of the binding of VEGF to its receptors Flt-1 (VEGFR-1) and KDR (VEGFR-2) on the surface of endothelial cells. Following intravitreal injection, the binding of bevacizumab to VEGF prevents the interaction of VEGF with its receptors on the surface of endothelial cells, reducing endothelial cell proliferation, vascular leakage, and new blood vessel formation in the retina.

On December 27, 2024 Citius Pharmaceuticals, Inc. ("Citius Pharma" or the "Company") (Nasdaq: CTXR), a biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products reported business and financial results for the fiscal full year ended September 30, 2024 (Press release, Citius Pharmaceuticals, DEC 27, 2024, View Source [SID1234649328]).

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Fiscal Full Year 2024 Business Highlights and Subsequent Developments

- Achieved U.S. Food and Drug Administration (FDA) approval of LYMPHIR (denileukin diftitox-cxdl), an immunotherapy for the treatment of adults with relapsed or refractory cutaneous T-cell lymphoma (CTCL);

- Advanced manufacturing, marketing and sales activities in preparation for commercial launch of LYMPHIR in the first half of 2025;

- Completed the merger of Citius Pharma’s oncology subsidiary with TenX Keane to form Citius Oncology, Inc., a standalone publicly traded company which began trading on the Nasdaq exchange under the ticker symbol CTOR on August 13, 2024;

- Supported two investigator-initiated trials to explore LYMPHIR’s potential as an immuno-oncology combination therapy being conducted at the University of Pittsburgh Medical Center and the University of Minnesota;

- Shared interim trial results with the clinical community at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference (SITC) (Free SITC Whitepaper) of University of Pittsburgh Medical Center’s Phase I trial of LYMPHIR with checkpoint inhibitor pembrolizumab; and,

- Met primary and secondary endpoints in the Phase 3 Pivotal Trial of Mino-Lok, demonstrating a statistically significant improvement in time to catheter failure of infected catheters compared to other physician-selected anti-infective lock solutions.

Financial Highlights

- Cash and cash equivalents of $3.3 million as of September 30, 2024;

- R&D expenses were $11.9 million for the full year ended September 30, 2024, compared to $14.8 million for the full year ended September 30, 2023;

- G&A expenses were $18.2 million for the full year ended September 30, 2024, compared to $15.3 million for the full year ended September 30, 2023;

- Stock-based compensation expense was $11.8 million for the full year ended September 30, 2024, compared to $6.6 million for the full year ended September 30, 2023; and,

- Net loss was $39.4 million, or ($5.97) per share for the full year ended September 30, 2024 compared to a net loss of $32.5 million, or ($5.57) per share for the full year ended September 30, 2023.

"In fiscal year 2024 we drove tremendous progress in our pipeline. It was a transformative year, marked by our first FDA approval and significant clinical milestones. The approval of LYMPHIR and the positive Phase 3 results for Mino-Lok underscore our commitment to developing innovative therapies. Our team successfully responded to FDA comments related to the biologics license application for LYMPHIR and ultimately gained FDA approval. Productive engagement with the FDA regarding the positive results of our Phase 3 Mino-Lok trial and Phase 2 Halo-Lido trial clarified our next steps for both programs. We anticipate continued engagement with the agency in the coming year and look forward to their guidance. Additionally, we are exploring strategic partnerships and licensing opportunities to maximize the potential of our portfolio and bring these important therapies to market efficiently," stated Leonard Mazur, Chairman and CEO of Citius Pharma.

"Looking ahead, our priorities for fiscal year 2025 include launching LYMPHIR through our majority-owned subsidiary, Citius Oncology, driving the clinical and regulatory strategies for Mino-Lok and Halo-Lido, fortifying our financial position, and applying a disciplined approach to resource allocation. We expect to launch LYMPHIR in the first half of 2025 and distribute CTOR shares to Citius Pharma shareholders by the end of the year, pending favorable market conditions. Our goal remains to deliver value for patients, healthcare providers, and shareholders. With a clear vision and a strong team, we are well-positioned to execute on our mission of bringing innovative therapies to market," added Mazur.

Full Year 2024 Financial Results:

Liquidity

As of September 30, 2024, the Company had $3.3 million in cash and cash equivalents.

As of September 30, 2024, the Company had 7,247,243 common shares outstanding, as adjusted for the 1-for-25 reverse stock split of the Company’s common stock, effected on November 25, 2024.

During the year ended September 30, 2024, the Company received net proceeds of $13.8 million from the issuance of equity. The Company expects to raise additional capital to support operations.

Research and Development (R&D) Expenses

R&D expenses were $11.9 million for the full year ended September 30, 2024, compared to $14.8 million for the full year ended September 30, 2023. The decrease in R&D expenses primarily reflects the completion of the Halo-Lido trial and completion of activities related to the regulatory resubmission for LYMPHIR, offset by shutdown costs associated with the end of the Phase 3 trial for Mino-Lok.

We expect research and development expenses to decrease in fiscal year 2025 as we continue to focus on the commercialization of LYMPHIR through our majority-owned subsidiary, Citius Oncology and because we have completed the Phase 3 trial for Mino-Lok.

General and Administrative (G&A) Expenses

G&A expenses were $18.2 million for the full year ended September 30, 2024, compared to $15.3 million for the full year ended September 30, 2023. The increase was primarily due to costs associated with pre-launch and market research activities associated with LYMPHIR. General and administrative expenses consist primarily of compensation costs, professional fees for legal, regulatory, accounting and corporate development services, and investor relations expenses.

Stock-based Compensation Expense

For the full year ended September 30, 2024, stock-based compensation expense was $11.8 million as compared to $6.6 million for the prior year. The increase of $5.2 million is largely due to the grant of options under the Citius Oncology stock plan. Stock-based compensation expense under the Citius Oncology stock plan was $7.5 million during the year ended September 30, 2024, compared to $2.0 million for the year ended September 30, 2023, as the plan was initiated in July 2023. For the years ended September 30, 2024 and 2023, stock-based compensation expense also includes $47,547 and $130,382, respectively, for the NoveCite stock option plan. In fiscal years 2023 and 2024, we granted options to our new employees and additional options to other employees, our directors, and consultants.

Net loss

Net loss was $39.4 million, or ($5.97) per share for the year ended September 30, 2024, compared to a net loss of $32.5 million, or ($5.57) per share for the year ended September 30, 2023, as adjusted for the reverse stock split. The increase in net loss reflects an increase in operating expense of $5.3 million offset by a decrease of $1.6 million in other income. Operating expense increased due to increases in stock-based compensation and general and administrative expenses, which were offset by decreased research and development expense.

On December 27, 2024 Chugai Pharmaceutical Co., Ltd, reported that it has obtained regulatory approval from the Ministry of Health, Labour and Welfare (MHLW) for "LUNSUMIO for intravenous infusion 1mg" and "LUNSUMIO for intravenous infusion 30mg" (generic name: mosunetuzumab (genetical recombination)) (hereafter, LUNSUMIO), antineoplastic agent / anti-CD20/CD3 bispecific antibody for the treatment of patients with relapsed or refractory (R/R) follicular lymphoma (FL) who have received two or more prior standard therapies (Press release, Chugai, DEC 27, 2024, View Source [SID1234649326]). LUNSUMIO is a T-cell engaging bispecific antibody targeting CD20/CD3, offering a new treatment option with high response rates and the potential for durable remission. The treatment duration is set at approximately six months or one year, depending on the patient’s response to treatment.

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"R/R FL is a difficult-to-cure disease that repeatedly relapses, and there is a need for new treatment options. LUNSUMIO is expected to provide durable remission with monotherapy, and with a predetermined treatment duration based on each patient’s response to therapy, which can help reduce the burden of treatment on patients. We are proceeding with preparations for the launch to make this drug available for treatment as soon as possible," said Dr. Osamu Okuda, Chugai’s President and CEO.

This approval is based on the results of a Japanese Phase I study with an expansion cohort (FLMOON-1 study) conducted in patients with R/R FL who had received two or more prior standard therapies, as well as an overseas Phase I/II clinical trial conducted by Roche in the same patient population. In both studies, the efficacy and safety of this drug were evaluated as a monotherapy.

The FLMOON-1 study was conducted on 19 Japanese patients with R/R FL who had previously received two or more prior standard therapies. The complete response rate (CRR), which was the primary endpoint as assessed by an independent review facility (IRF), was 68.4% (90% confidence interval: 47.0-85.3%). The most frequent adverse reactions were lymphocyte count decrease, cytokine release syndrome, alanine aminotransferase increase, neutrophil count decrease, aspartate aminotransferase increase, and infusion-related reactions.

The overseas Phase I/II study was conducted on 90 patients with R/R FL who had previously received two or more standard therapies. The primary endpoint, CRR as assessed by an IRF, was 57.8% (95% confidence interval: 46.9-68.1%). The most frequent adverse reactions were cytokine release syndrome, fever, fatigue, pruritus, neutropenia, and hypophosphatemia.

[Approval Information]

Product name: "LUNSUMIO for intravenous infusion 1mg" and "LUNSUMIO for intravenous infusion 30mg"

Generic name: mosunetuzumab (genetical recombination)

Indications: relapsed or refractory follicular lymphoma

Precautions concerning indications:

Treatment with this drug should be targeted at patients who have failed to respond to or have relapsed after two or more standard therapies, including an anti-CD20 monoclonal antibody product.
This drug should be administered to patients diagnosed with Grade 1-3A by a pathologist with sufficient experience.
Dosage and administration:
For adults, the usual dosage of mosunetuzumab (genetically modified) is administered as an intravenous infusion in 21-day cycles as follows:
Cycle 1: 1 mg on Day 1, 2 mg on Day 8, and 60 mg on Day 15
Cycle 2: 60 mg on Day 1
Cycles 3-8: 30 mg on Day 1 of each cycle
After 8 cycles, treatment should be discontinued for patients who achieve a complete response. For patients with stable disease or partial response, treatment may be continued for up to a total of 17 cycles.