VerImmune Awarded ~$470K CDMRP Grant to Drive Innovation in ViP-Based Therapeutics for Advanced Melanoma

On April 29, 2025 VerImmune, Inc. ("VerImmune"), a biotechnology company developing innovative Virus-inspired Particle (ViP) modalities, reported it has been awarded a ~$470,000 USD research grant (MRP Idea Award) from the U.S. Department of Defense (DoD) Melanoma Research Program (MRP), managed by the office of Congressionally Directed Medical Research Programs (CDMRP) (Press release, VerImmune, APR 29, 2025, View Source [SID1234652326]).

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The award will support the development of novel candidates based on VerImmune’s ViP technology for the treatment of advanced or rare melanoma cancers.

The MRP received 136 compliant applications and recommended funding for only nine, placing VerImmune’s application in the top 6.6% of submissions.

"Receiving this award from such a highly competitive pool clearly demonstrates the innovation and groundbreaking work we are doing at VerImmune to develop therapies for patients with unmet medical needs," said Joshua Wang, Founder and CEO of VerImmune, Inc. "We are committed to advancing our ViP platform to address critical healthcare challenges and look forward to utilizing this funding to drive our efforts in melanoma and other cancers."

Tonix Pharmaceuticals Presented Preclinical Data on Gastric Cancer Models at the American Association for Cancer Research (AACR) 2025 Annual Meeting

On April 29, 2025 Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, reported data in a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2025 Annual Meeting, held April 25-30, 2025, in Chicago, IL (Press release, TONIX Pharmaceuticals, APR 29, 2025, View Source [SID1234652325]). A copy of the Company’s presentation is available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com. The presentation titled, "TFF2-mediated CXCR4 partial agonism outperforms CXCR4 antagonism in reducing murine gastric cancer by suppressing PMN-MDSC generation," demonstrated positive data in gastric cancer animal models. In the AACR (Free AACR Whitepaper) presentation, a fusion protein of murine trefoil factor family member 2- murine serum albumin (mTFF2-MSA) was studied. Tonix is developing human TFF2-human serum albumin (hTFF2-HAS) as TNX-1700.

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"The combination therapy of mTFF2-MSA with anti-PD1 treatment shows promise in reducing immunosuppression in the tumor microenvironment (TME) in animal models," said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. "We are excited to develop TNX-1700 (TFF2-HAS) as the lead program in our immuno-oncology pipeline, by testing potential dosing strategies, and establishing potential clinical biomarkers through preclinical models."

Immunosuppressive neutrophils, also known as polymorphonuclear myeloid-derived suppressor cells (PMN-MDSCs), are a major component in solid tumors that significantly hinder anti-tumor activity1,2. Despite being short-lived, their continuous replenishment from the bone marrow sustains their potent immunosuppression in the TME3. Stromal cells in the TME promote immunosuppression by recruiting MDSCs via secretion of CXCL12. Trefoil Factor 2 (TFF2), a secreted peptide of the trefoil factor family, has displayed activity as a partial agonist of CXCR44,5. Data presented in the poster demonstrated that TFF2-MSA selectively reduces immunosuppressive neutrophils and cancer-driven granulopoiesis. Treatment with TFF2-MSA, in combination with an anti-PD1 antibody, induced robust anti-tumoral CD8+ T cell responses, inhibiting tumor invasion. TFF2 reduction correlated with elevated PMN-MDSCs in gastric cancer patients, highlighting the potential negative correlation between TFF2 and PMN-MDSCs levels.

About Trefoil Factor Family Member 2 (TFF2)

Human TFF2 is a secreted protein, encoded by the TFF2 gene in humans, that is expressed in gastrointestinal mucosa where it functions to protect and repair mucosa. TFF2 is also expressed at low levels in splenic immune cells and is now appreciated to have intravascular roles in the spleen and in the tumor microenvironment. In gastric cancer, TFF2 is epigenetically silenced, and TFF2 is suggested to be protective against cancer development through several mechanisms. Tonix is developing TNX-1700 (rTFF2-HSA) for the treatment of gastric and colon cancers under a license from Columbia University. The inventor of the core technology at Columbia is Dr. Timothy Wang, who is an expert in the molecular mechanisms of carcinogenesis whose research has focused on the carcinogenic role of inflammation in modulating stem cell functions. Dr. Wang demonstrated that knocking out the mTFF2 gene in mice leads to faster tumor growth and that overexpression of TFF2 markedly suppresses tumor growth by curtailing the homing, differentiation, and expansion of MDSCs to allow activation of cancer-killing CD8+ T cells. He went on to show that a novel engineered form of recombinant murine TFF2 (mTFF2-CTP) had an extended half-life in vivo and was able to suppress MDSCs and tumor growth in an animal model of colorectal cancer. Later, he showed in gastric cancer models that suppressing MDSCs using chemotherapy enhances the effectiveness of anti-PD1 therapy and significantly reduces tumor growth. Dr. Wang proposed the concept of employing rTFF2 in combination with other therapies in cancer prevention and early treatment.

Regeneron Reports First Quarter 2025 Financial and Operating Results

On April 29, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the first quarter of 2025 and provided a business update (Press release, Regeneron, APR 29, 2025, View Source [SID1234652324]).

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"Regeneron has one of the most exciting pipelines in the industry, with unmatched diversity, scientific distinction, and potential to help millions of patients," said Leonard S. Schleifer, M.D., Ph.D., Board co-Chair, President and Chief Executive Officer of Regeneron. "We are laser focused on fulfilling the promise of this pipeline by advancing our clinical programs, and expect several important data readouts this year. We are also working to ensure our four blockbuster medicines reach even more patients who could benefit, with year-to-date progress including regulatory approvals for Dupixent in CSU in the U.S. and COPD in Japan."

Financial Highlights

($ in millions, except per share data) Q1 2025 Q1 2024 % Change
Total revenues $ 3,029 $ 3,145 (4 %)
GAAP net income $ 809 $ 722 12 %
GAAP net income per share – diluted $ 7.27 $ 6.27 16 %
Non-GAAP net income(a) $ 928 $ 1,116 (17 %)
Non-GAAP net income per share – diluted(a) $ 8.22 $ 9.55 (14 %)

"We have made meaningful progress across our pipeline so far in 2025, including four regulatory approvals, nine regulatory submissions, and are poised to report pivotal or proof-of-concept data across programs in immunology, oncology, hematology, internal medicine, and rare diseases later this year," said Christopher Fenimore, Executive Vice President, Finance and Chief Financial Officer of Regeneron. "We continue to deploy capital with the goal of maximizing long-term shareholder value, with a focus on internal investment in our research, development, and commercial capabilities, along with returning capital directly to our shareholders through opportunistic share repurchases and our dividend program, which we initiated earlier this year."

Business Highlights

Key Pipeline Progress
Regeneron has approximately 45 product candidates in clinical development, including a number of marketed products for which it is investigating additional indications. Updates from the clinical pipeline include:

Dupixent (dupilumab)

In April 2025, the U.S. Food and Drug Administration (FDA) approved Dupixent for the treatment of adults and adolescents aged 12 years and older with CSU who remain symptomatic despite antihistamine treatment.
In March 2025, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Dupixent for the treatment of patients with COPD.
The Company and Sanofi presented positive results from the pivotal Phase 2/3 trial in adults with moderate-to-severe bullous pemphigoid at the 2025 American Academy of Dermatology (AAD) Annual Meeting. In February 2025, the FDA accepted for priority review the supplemental Biologics License Application (sBLA) for Dupixent in bullous pemphigoid, with a target action date of June 20, 2025. A regulatory application has also been submitted in the European Union (EU).

EYLEA HD (aflibercept) 8 mg

In April 2025, the FDA accepted for priority review an sBLA for both the treatment of macular edema following RVO, and broadening the dosing schedule to include every 4-week (monthly) dosing across approved indications. The FDA target action date is August 19, 2025, following the use of a priority review voucher.
The FDA issued a Complete Response Letter (CRL) for the EYLEA HD pre-filled syringe on April 23, 2025. The Company held several teleconferences with the FDA to better understand the contents of the CRL, and believes the key outstanding issue relates to a question posed by the FDA to a third-party component supplier. This component supplier has expeditiously responded to FDA requests for information. The CRL did not identify any issues with the safety or efficacy of EYLEA HD, the usability of the device, proposed labelling, or pre-approval inspection findings.
In April 2025, the FDA issued a CRL regarding the sBLA for the addition of extended dosing intervals. The FDA indicated that the submitted data did not support extended dosing intervals greater than every 16 weeks. The Company is evaluating the FDA’s decision.
The Company presented positive results from the Phase 3 QUASAR trial for the treatment of patients with macular edema following RVO, including those with central, branch, and hemiretinal vein occlusions, at the Angiogenesis, Exudation, and Degeneration (Angiogenesis) 2025 annual meeting.
The Company announced positive three-year (156-week) results from an extension study of the Phase 3 PULSAR trial in patients with wet age-related macular degeneration (wAMD). Similar to the three-year results for the pivotal PHOTON trial in diabetic macular edema (DME), the longer-term wAMD data demonstrated the vast majority of patients who entered the extension study sustained the visual gains and anatomic improvements achieved by the end of the second year, while also achieving substantially longer treatment intervals. The results were presented at the Angiogenesis 2025 annual meeting.

Oncology Programs

The Company submitted an sBLA to the FDA and a regulatory application in the EU for Libtayo (cemiplimab) in adjuvant CSCC based upon data from a Phase 3 trial which demonstrated that adjuvant treatment with Libtayo was the first and only immunotherapy that led to a statistically significant and clinically meaningful improvement in the primary endpoint of disease-free survival (DFS) in patients with high-risk CSCC after surgery.
The FDA accepted for review the resubmission of the BLA for odronextamab, a bispecific antibody targeting CD20 and CD3, in relapsed/refractory (R/R) follicular lymphoma, with a target action date of July 30, 2025.
The FDA accepted for review the resubmission of the BLA for linvoseltamab, a bispecific antibody targeting BCMA and CD3, in R/R multiple myeloma, with a target action date of July 10, 2025.
The European Commission (EC) granted conditional marketing approval of Lynozyfic (linvoseltamab) to treat adults with R/R multiple myeloma. The indication is specific to those who have received at least three prior therapies including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy.
Enrollment was completed in a Phase 3 confirmatory trial (LINKER-MM3) for linvoseltamab in R/R multiple myeloma.

Other Programs

The Company presented updated data from the Phase 1/2 trial of DB-OTO, an AAV-based gene therapy, in children with profound genetic hearing loss due to variants of the otoferlin (OTOF) gene at the Association for Research in Otolaryngology’s (ARO) 48th Annual MidWinter Meeting. The data showed that 11 out of 12 children treated demonstrated a notable response, with improved hearing at various decibel hearing levels. This included updated results from the first child dosed, who received treatment at 10 months of age and at 48 weeks post-treatment showed continued near-normal levels of hearing, as well as formal speech perception improvements between 48- and 72-weeks post-treatment.
A Phase 3 study for itepekimab, an antibody to IL-33, in chronic rhinosinusitis with nasal polyposis (CRSwNP) was initiated.
A Phase 2 study for REGN5381, an agonist antibody to NPR1, in uncontrolled hypertension was initiated.
A Phase 2 study for REGN7544, an antagonist antibody to NPR1, in sepsis-induced hypotension was initiated.
The FDA granted Orphan Drug designation for mibavademab, an agonist antibody to leptin receptor (LEPR), in generalized lipodystrophy; a Phase 3 study is ongoing.

Corporate Updates

The Company reached resolution of its patent infringement litigation related to Biocon’s EYLEA (aflibercept) Injection 2 mg biosimilar product. The settlement precludes Biocon from launching its biosimilar product in the United States until the second half of 2026. All intellectual property-related litigation with Biocon in the United States has been dismissed.
The Company announced a 10-year agreement with FUJIFILM Diosynth Biotechnologies (Fujifilm) to manufacture and supply Regeneron’s commercial bulk drug product at Fujifilm’s North Carolina campus. The agreement is anticipated to nearly double the Company’s large-scale manufacturing capacity in the United States.
First Quarter 2025 Financial Results

Revenues

($ in millions) Q1 2025 Q1 2024 % Change
Net product sales:
EYLEA HD – U.S. $ 307 $ 200 54 %
EYLEA – U.S. 736 1,202 (39 %)
Total EYLEA HD and EYLEA – U.S. 1,043 1,402 (26 %)
Libtayo – U.S. 192 159 21 %
Libtayo – ROW** 93 105 (11 %)
Total Libtayo – Global 285 264 8 %
Praluent – U.S. 57 70 (19 %)
Evkeeza – U.S. 31 24 29 %
Inmazeb – ROW — 1 (100 %)
Total net product sales 1,416 1,761 (20 %)

Collaboration revenue:
Sanofi 1,183 910 30 %
Bayer 344 356 (3 %)
Other 4 1 *
Other revenue 82 117 (30 %)
Total revenues $ 3,029 $ 3,145 (4 %)

* Percentage not meaningful
** Rest of world (ROW)

Net product sales of EYLEA HD increased in the first quarter of 2025, compared to the first quarter of 2024, primarily due to higher sales volumes.

Net product sales of EYLEA in the first quarter of 2025, compared to the first quarter of 2024, were negatively impacted by (i) lower volume as a result of continued competitive pressures (as described below), loss in market share to compounded bevacizumab due to patient affordability constraints, and the continued transition of patients to EYLEA HD, and (ii) a lower net selling price.

In addition, total EYLEA and EYLEA HD net product sales were negatively impacted by lower wholesaler inventory levels at the end of the first quarter of 2025 compared to the end of the fourth quarter of 2024. Total EYLEA and EYLEA HD net product sales decreased 30% on a sequential basis; however, physician unit demand decreased sequentially by 11%.

EYLEA net product sales have been, and are likely to continue to be, negatively impacted by increased competition from other anti-VEGF products, including biosimilars, as well as the transition of patients from EYLEA to EYLEA HD. In addition, if independent not-for-profit patient assistance funds that provide copay assistance are unable to support eligible patients, this will likely have a continued negative impact on patient affordability resulting in lower utilization of higher-cost anti-VEGF agents.

Sanofi collaboration revenue increased in the first quarter of 2025, compared to the first quarter of 2024, due to an increase in the Company’s share of profits from the commercialization of antibodies, which were $1.018 billion and $804 million in the first quarter of 2025 and 2024, respectively. The change in the Company’s share of profits from commercialization of antibodies was driven by higher profits associated with an increase in Dupixent sales.

Refer to Table 4 for a summary of collaboration revenue.

Operating Expenses

GAAP %
Change
Non-GAAP(a) %
Change

($ in millions) Q1 2025 Q1 2024 Q1 2025 Q1 2024
Research and development (R&D) $ 1,327 $ 1,248 6 % $ 1,186 $ 1,122 6 %
Acquired in-process research and development (IPR&D) $ 12 $ 7 71 % * * n/a
Selling, general, and administrative (SG&A) $ 633 $ 689 (8 %) $ 537 $ 584 (8 %)
Cost of goods sold (COGS) $ 266 $ 240 11 % $ 217 $ 196 11 %
Gross margin on net product sales(c) 81% 86% 85% 89%
Cost of collaboration and contract manufacturing (COCM) $ 199 $ 193 3 % * * n/a
Other operating expense (income), net $ — $ 15 (100 %) * $ — — %

* GAAP and non-GAAP amounts are equivalent as no non-GAAP adjustments have been recorded

GAAP and non-GAAP R&D expenses increased in the first quarter of 2025, compared to the first quarter of 2024, driven by the advancement of the Company’s clinical pipeline and higher personnel-related costs.
GAAP and non-GAAP gross margin on net product sales was adversely impacted by higher inventory write-offs and reserves in the first quarter of 2025 compared to the first quarter of 2024.
Other Financial Information

GAAP other income (expense), net included the recognition of net unrealized gains on equity securities of $140 million in the first quarter of 2025, compared to $196 million of net unrealized losses in the first quarter of 2024.

In the first quarter of 2025, the Company’s GAAP effective tax rate (ETR) was 10.6%, compared to (3.0%) in the first quarter of 2024. The GAAP ETR increased in the first quarter of 2025, compared to the first quarter of 2024, due to lower tax benefits from less stock option exercises. In the first quarter of 2025, the non-GAAP ETR was 11.6%, compared to 6.1% in the first quarter of 2024.

A reconciliation of the Company’s GAAP to non-GAAP results is included in Table 3 of this press release.

Capital Allocation

In February 2025, the Company’s board of directors authorized a new share repurchase program to repurchase up to an additional $3.0 billion of the Company’s common stock. During the first quarter of 2025, the Company repurchased shares of its common stock and recorded the cost of the shares, or $1.052 billion, as Treasury Stock. As of March 31, 2025, an aggregate of $3.874 billion remained available for share repurchases under the Company’s share repurchase programs.

In April 2025, the Company’s board of directors declared a cash dividend of $0.88 per share on the Company’s common stock and Class A stock, payable on June 6, 2025 to shareholders of record as of May 20, 2025.

Rakovina Therapeutics Showcases Preclinical Results of Novel AI-Discovered Cancer Therapies at AACR 2025

On April 29, 2025 Rakovina Therapeutics Inc. (TSX-V: RKV) (FSE: 7JO), a biopharmaceutical company advancing next-generation cancer therapies through artificial intelligence (AI)-powered drug discovery, reported the presentation of new preclinical data from two of its lead programs at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 (Press release, Rakovina Therapeutics, APR 29, 2025, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-showcases-preclinical-results-of-novel-ai-discovered-cancer-therapies-at-aacr-2025 [SID1234652322]).

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The first presentation highlighted Rakovina’s PARP1-selective inhibitor program. Using the proprietary Deep Docking and generative platforms , Rakovina previously screened more than four billion potential drug-like molecules to identify a shortlist of top candidates. These compounds were synthesized and evaluated in Rakovina’s laboratory facilities at the University of British Columbia.

Data presented at AACR (Free AACR Whitepaper) demonstrated a new class of PARP1 inhibitors with significantly improved metabolic stability, including the lowest in vitro clearance rates and the longest half-life compared to other candidates currently in development. Early animal studies revealed strong plasma exposure and a promising pharmacokinetic profile suggestive of central nervous system (CNS) penetration — a potentially significant advantage in treating brain-involved malignancies.

The second presentation showcased progress from Rakovina’s ATR-specific inhibitor program, developed in partnership with Variational AI. Researchers identified a focused set of lead candidates predicted to be highly potent and selective against ATR, a key DNA damage response target. These candidates are also designed with the potential to cross the blood-brain barrier, an important feature for addressing cancers affecting the CNS. The top candidates are currently being synthesized for laboratory validation.

"Our ability to screen billions of molecules and advance top candidates to in vitro and in vivo validation within months — rather than years — exemplifies the transformative power of AI in drug discovery," said Jeffrey Bacha, Executive Chairman of Rakovina Therapeutics. "This acceleration gives us the opportunity to significantly reduce the time and cost required to bring new life-saving cancer treatments to patients."

The results presented at AACR (Free AACR Whitepaper) reinforce the robustness of Rakovina’s AI-enabled discovery platform. Moving forward, Rakovina will leverage these findings to further refine and train its AI models, with the goal of advancing best-in-class lead candidates for both the PARP1 and ATR programs into clinical development. The Company intends to collaborate with pharmaceutical partners to accelerate the path to the clinic and deliver novel therapies to patients in need.

Pasithea Therapeutics Announces Completion of Enrollment and Initial Dosing of Patients in Cohort 6 from its Phase 1 Trial of PAS-004 in Advanced Cancer Patients

On April 29, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, for the treatment of neurofibromatosis type 1 (NF1) and other MAPK pathway driven cancer indications, reported the Company has completed enrollment and initial dosing of three subjects in Cohort 6 with 30 mg capsules of PAS-004 (Press release, Pasithea Therapeutics, APR 29, 2025, View Source [SID1234652321]).

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"We are pleased to have recruited, enrolled and commenced dosing of the initial three subjects in Cohort 6 more rapidly than anticipated and we currently expect to complete enrollment of all patients in the trial by the end of 2025," said Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea.

The ongoing Phase 1 clinical trial is a multi-center, open-label, dose escalation 3+3 study design to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of PAS-004 in patients with MAPK pathway driven advanced solid tumors with a documented RAS, NF1 or RAF mutation or patients who have failed BRAF/MEK inhibition (NCT06299839).