On May 13, 2025 Atossa Therapeutics, Inc. (Nasdaq: ATOS) (Atossa or the Company), a clinical-stage biopharmaceutical company developing innovative medicines for breast cancer, reported its financial results for the first quarter ended March 31, 2025 and provided an update on recent company developments (Press release, Atossa Therapeutics, MAY 13, 2025, View Source [SID1234652996]).

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First Quarter 2025 Highlights:

Announced Strategic Decision to Pursue Metastatic Breast Cancer Indication: Atossa announced plans to target metastatic breast cancer as its lead program for (Z)-endoxifen. The decision reflects its commitment to addressing the persistent unmet medical need in metastatic breast cancer and the potential for a more streamlined regulatory pathway to deliver (Z)-endoxifen to these patients. Current treatment options for metastatic breast cancer often provide limited durability of response and substantial side effects. In previous clinical trials, (Z)-endoxifen has been shown to be well-tolerated as a selective estrogen receptor modulator (SERM), which Atossa believes supports its potential to fill this critical gap in treatment.
Significantly Strengthened (Z)-endoxifen Patent Portfolio with Three New U.S. Patents: Atossa continued to bolster the intellectual property portfolio of (Z)-endoxifen with the grant of three new U.S. patents covering 31 claims directed at: sustained release compositions of (Z)-endoxifen (U.S. Patent No. 12,201,591); enteric oral formulations of (Z)-endoxifen and salts thereof as well as their use in treating hormone-dependent breast and reproductive tract disorders (U.S. Patent No. 12,275,684); and 58 claims covering (Z)-endoxifen formulations, including various levels of purity and stability as well as methods of using those formulations (U.S. Patent No. 12,281,056). Atossa’s robust patent portfolio now encompasses more than 200 patent claims related to (Z)-endoxifen formulations and their clinical applications.
"Our focus remains firmly on advancing (Z)-endoxifen as a next-generation therapy for breast cancer patients across the full spectrum of care—including a strategic emphasis on metastatic breast cancer, where therapeutic innovation is urgently needed," said Steven Quay, M.D., Ph.D., President and Chief Executive Officer of Atossa. "Across multiple clinical trials involving hundreds of patients, (Z)-endoxifen has consistently demonstrated strong tolerability and therapeutic versatility, which we believe shows its potential as a therapy for breast cancer from early-stage disease to more advanced stages. We are committed to unlocking the full potential of (Z)-endoxifen for patients while delivering value to our shareholders. A cornerstone of this strategy is the robust intellectual property portfolio we are building in an effort to protect our programs globally. As we look ahead to the remainder of 2025 and beyond, we are energized by the many opportunities to position (Z)-endoxifen as a potentially safer, more effective endocrine therapy for breast cancer patients worldwide."

Comparison of Three Months Ended March 31, 2025 and 2024

Revenue and Cost of Revenue. For the three months ended March 31, 2025 and 2024, we had no source of revenue and no associated cost of revenue.

Operating Expenses. Total operating expenses were $7.4 million for the three months ended March 31, 2025, which was an increase of $0.4 million, from the three months ended March 31, 2024 of $7.0 million. Factors contributing to the increased operating expenses in the three months ended March 31, 2025 are explained below.

Research & Development (R&D) Expenses. The following table provides a breakdown of major categories within R&D expenses for the three months ended March 31, 2025 and 2024, together with the dollar change and percentage change in those categories (dollars in thousands):

For the Three Months Ended March 31,

2025

2024

Increase (Decrease)

% Increase (Decrease)

Research and Development Expenses

Clinical and pre-clinical trials

$

2,747

$

2,884

$

(137)

(5) %

Compensation

880

626

254

41 %

Professional fees and other

530

238

292

123 %

Research and Development Expenses Total

$

4,157

$

3,748

$

409

11 %

Clinical and pre-clinical trial expenses decreased $0.1 million for the three months ended March 31, 2025, compared to the three months ended March 31, 2024, due to a slight decrease in spend related to our (Z)-endoxifen trials, including drug development costs.
The increase in R&D compensation expenses of $0.3 million for the three months ended March 31, 2025, compared to the three months ended March 31, 2024 was due to an increase in headcount.
The increase in R&D professional fees and other of $0.3 million was due to an increase in spending on regulatory consulting services.
General and Administrative (G&A) Expenses. The following table provides a breakdown of major categories within G&A expenses for the three months ended March 31, 2025 and 2024, together with the dollar change and percentage change in those categories (dollars in thousands):

For the Three Months Ended March 31,

2025

2024

Increase (Decrease)

% Increase (Decrease)

General and Administrative Expenses

Compensation

$

1,462

$

1,325

$

137

10 %

Professional fees and other

1,614

1,680

(66)

(4) %

Insurance

181

227

(46)

(20) %

General and Administrative Expenses Total

$

3,257

$

3,232

$

25

1 %

The increase in G&A compensation expenses of $0.1 million for the three months ended March 31, 2025, compared to the three months ended March 31, 2024 was due to an increase in headcount quarter over quarter.
Interest Income. Interest income was $0.7 million for the three months ended March 31, 2025, a decrease of $0.4 million from interest income of $1.1 million for the three months ended March 31, 2024. The decrease was due to a decrease in the balance in our money market account.

About (Z)-Endoxifen
(Z)-endoxifen is a highly potent SERM with demonstrated ability to inhibit—and potentially degrade—estrogen receptors. It has shown activity even in tumors that have developed resistance to other endocrine therapies. Beyond its anti-estrogenic properties, (Z)-endoxifen also targets protein kinase C beta 1 (PKCβ1), an oncogenic signaling protein, at clinically achievable blood levels. Importantly, (Z)-endoxifen seems to deliver comparable or superior bone-protective effects relative to tamoxifen, while exhibiting minimal or no endometrial proliferative activity—which we believe addresses key limitations of current standard-of-care therapies. Atossa is developing a proprietary oral formulation of (Z)-endoxifen that is enteric-coated to bypass stomach acid, which would otherwise convert the active (Z)-isomer to its inactive (E)-form. We believe this innovation allows for optimal bioavailability and therapeutic integrity. Clinical studies have shown Atossa’s (Z)-endoxifen to be well tolerated in both healthy women and those with breast cancer. Atossa is prioritizing the development of (Z)-endoxifen for the treatment of metastatic breast cancer, where novel therapeutic options are urgently needed. The compound is currently being evaluated in three Phase 2 trials: one in women with ductal carcinoma in situ (DCIS) and two in women with estrogen receptor positive (ER+) / human epidermal growth factor receptor 2 negative (HER2-) breast cancer, including the EVANGELINE study and an I-SPY study. Atossa’s (Z)-endoxifen program is supported by a growing global intellectual property portfolio, including three recently issued U.S. patents and numerous pending applications worldwide.

On May 13, 2025 Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, reported the presentation of preclinical data demonstrating the potential and versatility of its proprietary RSwitch technology to enable the fine-tuning of transgene levels in gene therapy applications with orally administered small molecules specifically built to meet the needs of each indication with a range of pharmacokinetic properties (Press release, Rgenta Therapeutics, MAY 13, 2025, View Source [SID1234652995]). The data will be presented at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting, which will be held from May 13 -17th, 2025, in New Orleans, LA.

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"The data presented at this year’s ASGCT (Free ASGCT Whitepaper) meeting provide evidence to support the advantages of our RSwitch technology which is designed to deliver precise temporal control of therapeutic transgene expression over a wide dynamic range and is applicable to a variety of gene and cell therapy applications," said Travis Wager, Ph.D., co-founder and chief scientific officer. "While there are existing methods to control transgene expression in gene therapy, they typically rely on engineered transcription factors and repurposed pharmacology, are not specific to the switching system, and consequently do not provide particularly tight control of the transgene’s expression. Rgenta has developed a platform for imparting gene therapy vectors with selective, dose-dependent regulation by orally bioavailable small molecules, RDrugs. Aided by massively parallel sequence library screening and deep learning-driven design, we have built RDrugs to meet the needs of each indication with a range of pharmacokinetic properties enabling direct control, via RSwitch, of specific pre-mRNA splicing events needed for the regulated production of the therapeutic payload."

In a poster titled RSwitch Enabled Gene Therapy to Fine Tune Frataxin Expression for the Treatment of Friedreich’s Ataxia, Rgenta scientists presented data demonstrating the ability of RSwitches to enable dose-dependent control with up to 4000-fold induction of gene expression by a specific RDrug. RSwitches were able to control gene expression in both high and low strength promotors. Data were also presented from an RSwitch-regulated AAV gene therapy designed to express frataxin (FXN) for the potential treatment of Friedreich’s Ataxia (FA). FA is a progressive neurodegenerative disease caused by a genetic deficiency of FXN, which is a small, nuclear-encoded mitochondrial protein believed to act as an iron chaperone or iron storage protein. While constitutive replacement of FXN by AAV vectors has shown promise in animal models, multiple studies have demonstrated the cardiotoxic effects of unregulated FXN overexpression making this gene therapy a good candidate for regulation of gene expression by a system such as RSwitch. The data demonstrate that an RSwitch-regulated FA expressing AAV-gene therapy achieves dose dependent expression of FXN in the heart with RDrug and human endogenous FXN levels in mice at low RDrug levels. A second test system demonstrated the ability of Rgenta’s RSwitch and corresponding brain penetrant RDrug to regulate expression of a transgene in the brain.

"Our proprietary RSwitch technology provides an example of the power of Rgenta’s RNA-targeted small molecule platform and represents a potentially game-changing tool for the development of regulatable gene and cell therapies that could improve the safety of these medicines," Simon Xi, Ph.D., cofounder and chief executive officer of Rgenta. "Rgenta’s internal pipeline is focused on the application of our platform to inhibit disease driving therapeutic targets, such as MYB for the treatment of solid tumors, including adenoid cystic carcinoma and colorectal cancer, and hematological cancers such as acute myeloid leukemia, and PMS1 for the potential treatment of repeat expansion diseases such as Huntington’s disease. We see the value of our RSwitch technology as a potential partnering opportunity with companies developing gene and cell therapies to improve the clinical success of these gene replacement therapies for difficult to treat diseases such as FA."

About RSwitch
RSwitch is a proprietary regulatable gene therapy system that enables oral, small molecule drug control of transgene levels in gene and cell therapy applications. RSwitch encodes a "dimmer" switch that makes the expression of transgene dependent on the administration of an oral small molecule drug (RDrug) that controls the system. Only when the drug is administered is the system activated. Furthermore, the level of gene expression is dependent on how much drug is administered. This precise gene control has the potential to enable fine control of the expression of a therapeutic protein. Rgenta has demonstrated the RSwitch system’s feasibility in vitro and in vivo, achieving dose-dependent expression of reporter transgenes following small molecule administration. RSwitch technology offers versatile control across multiple gene and cell therapy applications, and the company is actively exploring strategic partnerships.

On May 13, 2025 Altimmune, Inc. (Nasdaq: ALT), a late clinical-stage biopharmaceutical company developing novel peptide-based therapeutics for liver and cardiometabolic diseases, reported financial results for the first quarter ended March 31, 2025, and provided a business update (Press release, Altimmune, MAY 13, 2025, View Source [SID1234652993]).

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"The first quarter of 2025 was productive for Altimmune as the Company approaches a number of important milestones," said Vipin K. Garg, Ph.D., President and Chief Executive Officer of Altimmune. "The readout of our IMPACT Phase 2b trial of pemvidutide in MASH is on-track for the second quarter of 2025. We believe that achieving statistical significance on MASH resolution and fibrosis improvement at only 24 weeks, coupled with clinically meaningful weight loss, would position pemvidutide as the best-in-class therapeutic candidate for the treatment of MASH."

Dr. Garg continued, "At our recent R&D Day event we unveiled two additional indications for pemvidutide in AUD and ALD, and our intent to initiate Phase 2 clinical trials in these indications in Q2 and Q3, respectively. AUD and ALD are conditions of significant unmet medical need with limited treatment options. We remain committed to developing pemvidutide for treatment of liver and cardiometabolic diseases that leverage its differentiated clinical profile."

Recent Highlights and Anticipated Milestones

MASH

Top-line data from the IMPACT Phase 2b trial of pemvidutide in biopsy-confirmed F2/F3 MASH expected in Q2 2025
Top-line data is expected to include rates of MASH resolution and fibrosis improvement, weight loss, non-invasive tests, and data on safety and tolerability.
A total of 212 participants were randomized, exceeding the 190 originally planned.
If successful, pemvidutide would be the first investigational therapy in MASH to achieve statistical significance in both MASH resolution and fibrosis improvement, as well as demonstrate meaningful weight loss, after only 24 weeks of treatment.
Altimmune presented new data at the EASL International Liver Congress 2025, including a follow-on analysis of the Company’s Phase 1b trial in MASLD using the MASH Resolution Index (MASHResInd).
Developed by Dr. Rohit Loomba, Professor of Medicine and Chief of Gastroenterology and Hepatology at the University of California San Diego, MASHResInd is a non-invasive measure that has been highly predictive of MASH resolution.
The analyses indicated that after 24 weeks of treatment, the proportion of participants receiving pemvidutide achieving MASHResInd responses exceeded 90%. These findings indicate that high rates of MASH resolution may be observed in the upcoming IMPACT Phase 2b MASH trial readout.
Additional Indications for Pemvidutide: AUD and ALD

During Altimmune’s R&D Day, the Company announced the development of pemvidutide in two additional indications: AUD and ALD
AUD and ALD are characterized by large patient populations with significant unmet medical need and very few treatment options.
Investigational New Drug (IND) applications were cleared by the FDA in the first quarter of 2025. The Phase 2 trials in AUD and ALD are expected to initiate in the second and third quarters of 2025, respectively.
Preclinical data and data from other clinical trials support the potential of pemvidutide to reduce alcohol consumption, improve liver health, and provide the added benefit of meaningful weight loss.
Corporate Update

The Company entered into a $100 million credit facility with Hercules Capital, with an initial $15 million tranche funded at closing. An additional $25 million is available in 2025 at Altimmune’s option, subject to the achievement of certain clinical and financial milestones. The remaining $60 million is available beginning in 2026, with $15 million subject to the achievement of certain clinical and financial milestones and up to $45 million available subject to approval of Hercules. The credit facility significantly increases Altimmune’s financial strength and flexibility on attractive terms.
Financial Results for the Three Months Ended March 31, 2025

Altimmune reported cash, cash equivalents and short-term investments totaling $150 million on March 31, 2025.
Research and development expenses were $15.8 million for the three months ended March 31, 2025, compared to $21.5 million in the same period in 2024, the decrease resulting from timing of clinical trial costs. The expenses for the quarter ended March 31, 2025, included $9.2 million in direct costs related to pemvidutide development activities.
General and administrative expenses were $6.0 million for the three months ended March 31, 2025, compared to $5.3 million in the same period in 2024. The increase was primarily due to a $0.5 million increase in stock compensation and other labor-related expenses.
Interest income was $1.5 million for the three months ended March 31, 2025, compared to $2.4 million for the same period in 2024.
Net loss for the three months ended March 31, 2025, was $19.6 million, or $0.26 net loss per share, compared to a net loss of $24.4 million, or $0.34 net loss per share, in the same period in 2024.
Conference Call Information:

Date: May 13, 2025
Time: 8:30 a.m. Eastern Time
Webcast: To listen, the conference call will be webcast live on Altimmune’s Investor Relations website at View Source
Dial-in: To participate or dial-in, register here to receive the dial-in numbers and unique PIN to access the call.

Following the conclusion of the call, the webcast will be available for replay on the Investor Relations (IR) page of the Company’s website at www.altimmune.com. The Company has used, and intends to continue to use, the IR portion of its website as a means of disclosing material non-public information and for complying with disclosure obligations under Regulation FD.

About Pemvidutide

Pemvidutide is a novel, investigational, peptide-based 1:1 GLP-1/glucagon dual receptor agonist in development for the treatment of MASH, obesity, Alcohol Use Disorder (AUD) and Alcohol Liver Disease (ALD). Activation of the GLP-1 and glucagon receptors is believed to mimic the complementary effects of diet and exercise on weight loss, with GLP-1 suppressing appetite and glucagon increasing energy expenditure. Glucagon is also recognized as having direct effects on hepatic fat metabolism, which is believed to lead to rapid reductions in levels of liver fat and serum lipids. In clinical trials to date, once-weekly pemvidutide has demonstrated compelling weight loss with class-leading lean mass preservation, and robust reductions in triglycerides, LDL cholesterol, liver fat content and blood pressure. The U.S. FDA has granted Fast Track designation to pemvidutide for the treatment of MASH. Pemvidutide completed the MOMENTUM Phase 2 obesity trial in 2024 and is being studied in the ongoing IMPACT Phase 2b MASH trial with top line results expected in late June 2025. IND applications in AUD and ALD have received FDA clearance with Phase 2 trials scheduled to commence in Q2 and Q3 2025, respectively.

On May 13, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported business updates and announced financial results for the first quarter ended March 31, 2025 (Press release, Verastem, MAY 13, 2025, View Source [SID1234652992]).

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"In the first quarter of 2025, we continued to make progress with our pipeline programs by exercising our option early to license VS-7375 from our partner GenFleet Therapeutics, completing enrollment in the initial cohorts in our RAMP 205 clinical trial in first-line metastatic pancreatic cancer, and continuing enrollment in the triplet combination in our RAMP 203 clinical trial in advanced KRAS G12C mutant non-small cell lung cancer," said Dan Paterson, president and chief executive officer of Verastem Oncology. "With a strengthened financial position, we are looking forward to a transformational second quarter with the FDA approval and launch of AVMAPKI FAKZYNJA CO-PACK for KRAS-mutated recurrent low-grade serous ovarian cancer, our plans to initiate a Phase 1/2a study in the U.S. for VS-7375, our potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, in mid-2025, and share updated data for both VS-7375 and RAMP 205 at ASCO (Free ASCO Whitepaper)."

First Quarter 2025 and Recent Updates

Avutometinib and Defactinib Combination in Low-Grade Serous Ovarian Cancer (LGSOC)

Announced the U.S. Food and Drug Administration (FDA) approved AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025, in advance of PDUFA action date of June 30, 2025.
Initiation of the commercial execution of the AVMAPKI FAKZYNJA CO-PACK launch in the U.S.
AVMAPKI FAKZYNJA CO-PACK is now available through a specialty distribution network in the U.S.
A support program for patients prescribed AVMAPKI FAKZYNJA CO-PACK, called Verastem Cares, is now available.
Submitted request for NCCN guideline inclusion.
Shared multiple oral and poster presentations at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 on April 25-30, highlighting the exploration of the mechanisms by which the Company’s FAK inhibitor increases the anti-tumor efficacy of avutometinib.
Multiple abstracts were selected for oral and poster presentations at the Society of Gynecologic Oncology (SGO) 2025 Annual Meeting on Women’s Cancer on March 14-17 in Seattle. These presentations included an oral presentation of additional analyses from the Phase 2 RAMP 201 trial of avutometinib and defactinib combination with recurrent LGSOC and an oral presentation of interim results from a Phase 2 Investigator-Sponsored Trial evaluating avutometinib plus defactinib in advanced or recurrent gynecologic mesonephric cancer.
Key Milestones Expected for 2025:

Primary analysis from both the FRAME and RAMP 201 clinical trials anticipated to be published in H1 2025.
Complete enrollment for the international Phase 3 confirmatory RAMP 301 clinical trial for patients with recurrent LGSOC regardless of KRAS mutation status by the end of 2025.
Report initial data from the RAMP 201J Phase 2 clinical trial being conducted in Japan with JGOG in H2 2025.
Continue to advance the regulatory pathway in Japan and Europe.
RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in First-Line Metastatic Pancreatic Cancer

Completed enrollment of 60 patients in the dose-level evaluation phase of RAMP 205 study in Q1; follow-up continues.
In March 2025, announced several updates to the trial, including the addition of a new dose level "0" to evaluate the doses of avutometinib and defactinib used in LGSOC and expanding all dose levels to 12 patients each, including six additional patients to dose level "1", where 5/6 patients reported an objective response (83% cORR) at the ASCO (Free ASCO Whitepaper) 2024 annual meeting.
Key Milestones Expected for 2025:

Plan to report additional data when ASCO (Free ASCO Whitepaper) abstracts are live on May 22, 2025.
Select the recommended Phase 2 Dose (RP2D) for trial expansion in H1 2025.
RAMP 203: Avutometinib Plus Defactinib in Combination with a KRAS G12C Inhibitor in Non-Small Cell Lung Cancer (NSCLC)

Completed enrollment in the KRAS G12C inhibitor prior-treated Stage 1 Part B doublet cohort in Q1 2025.
Completed enrollment in the planned dose level evaluation cohorts for the triplet combination in Q1 2025.
Key Milestones Expected for 2025:

Present an interim update of both doublet and triplet data at a medical meeting in H2 2025.
VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor, in Advanced Solid Tumors

Verastem announced in April 2025 that the FDA had cleared the Company’s Investigational New Drug (IND) application for VS-7375, enabling a Phase 1/2a trial in advanced solid tumors in the U.S.
Shared a presentation at the AACR (Free AACR Whitepaper) Annual Meeting 2025, highlighting that VS-7375 was found to be more potent than other KRAS G12D inhibitors in preclinical models.
GenFleet announced on Feb. 28, 2025, that it had dosed the first patient in the Phase 2 portion of the trial in China.
Verastem announced on January 14, 2025, that it had exercised its option early to license GFH375 (VS-7375) from partner GenFleet Therapeutics. In addition, the Company announced preliminary clinical data from the Phase 1 dose-escalation study conducted by GenFleet in China. In the study, VS-7375 demonstrated oral bioavailability, with no DLTs across six dose levels, and partial responses were achieved among multiple patients with both pancreatic and lung cancers.
Key Milestones Expected for 2025:

Initiate a Phase 1/2a trial in the U.S. by mid-2025.
GenFleet to share clinical data from the Phase 1 study of VS-7375 in an oral presentation at ASCO (Free ASCO Whitepaper) on Monday, June 2, 2025.
Upcoming Presentations

ASCO Annual Meeting
The meeting will be held from May 30 to June 3, 2025, in Chicago, IL, and abstracts are under embargo until May 22, 2025, at 5:00 pm EDT.

Title: A First-in-Human Phase I/II Study of GFH375, a Highly Selective and Potent Oral KRAS G12D Inhibitor in Patients with KRAS G12D Mutant Advanced Solid Tumors

Abstract Number: 3013
Session: Rapid Oral Abstract Sessions: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Date/Time: Monday, June 2, 2025 from 8:00 am to 9:30 am CDT
Title: Avutometinib/defactinib and gemcitabine/nab-paclitaxel combination in first-line metastatic pancreatic ductal adenocarcinoma: Updated safety and efficacy of a phase 1b/2 study (RAMP 205)

Abstract Number: e16043
Accepted for inclusion in the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting Proceedings, Journal of Clinical Oncology supplement. The abstract is under embargo until May 22, and the Company will be reporting additional data then.
ESMO Gynaecological Cancers Congress 2025
The meeting will be held from June 19 to 21, 2025, in Vienna, Austria, and the abstract is under embargo until June 16, 2025.

Title: Blood ctDNA vs tumor tissue screening for the detection of KRAS mutations in low-grade serous ovarian cancer
Abstract Number: 276
Date/Time: Thursday, June 19, from 2:00 to 3:30 pm EDT
Corporate Updates

In April 2025, Verastem strengthened its balance sheet by raising gross proceeds of approximately $75 million in a private placement of 3.4 million shares of its common stock and 7.3 million pre-funded warrants to purchase 7.3 million shares of its common stock.
First Quarter 2025 Financial Results

Verastem Oncology ended the first quarter of 2025 with cash, cash equivalents and investments of $117.6 million. On a pro forma basis, taking into account the $75 million of gross proceeds raised in a private placement in April, cash and cash equivalents were $192.6 million as of March 31, 2025.

Total operating expenses for the three months ended March 31, 2025 (the "2025 Quarter") were $44.2 million, inclusive of $6.8 million of one-time charges, compared to $28.1 million for the three months ended March 31, 2024 (the "2024 Quarter").

Research & development expenses for the 2025 Quarter were $29.2 million, compared to $17.7 million for the 2024 Quarter. The increase of $11.5 million, or 65.0%, was primarily related to the option exercise fee related to the GenFleet G12D program, increased contract research organization costs, and increased drug substance and drug product costs.

Selling, general & administrative expenses for the 2025 Quarter were $15.0 million, compared to $10.4 million for the 2024 Quarter. The increase of $4.6 million, or 44.2%, was primarily related to additional costs in anticipation of a potential launch of avutometinib and defactinib in KRAS mt LGSOC, increased personnel costs, including non-cash stock compensation, and one-time financing costs associated with the note purchase agreement.

Net loss for the 2025 Quarter was $52.1 million, or $0.96 per share (basic and diluted), compared to $33.9 million, or $1.26 per share for the 2024 Quarter.

For the 2025 Quarter, non-GAAP adjusted net loss was $42.9 million, or $0.79 per share (diluted) compared to non-GAAP adjusted net loss of $26.2 million, or $0.98 per share (diluted), for the 2024 Quarter. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.

Use of Non-GAAP Financial Measures

To supplement Verastem Oncology’s condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), the Company uses the following non-GAAP financial measures in this press release: non-GAAP adjusted net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company’s GAAP financial statements, because it provides greater transparency and period-over- period comparability with respect to the Company’s operating performance and can enhance investors’ ability to identify operating trends in the Company’s business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company’s operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months ended March 31, 2025 and 2024 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

About AVMAPKI and FAKZYNJA Combination Therapy

AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.

The U.S. Food and Drug Administration (FDA) approved AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is also evaluating avutometinib in combination with defactinib and other agents as a potential treatment for patients with advanced pancreatic cancer (RAMP 205; NCT05669482) and advanced KRAS G12C mutant non-small cell lung cancer (RAMP 203; NCT05074810). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.

AVMAPKI FAKZYNJA CO-PACK U.S. Indication

Indication
AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.

This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Important Safety Information

Warnings and Precautions

Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.
Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.
Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.
Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.
Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.
Adverse Reactions
The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.

Drug Interactions

Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.
Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.
Use in Specific Populations

Lactation: Advise not to breastfeed.
Fertility: May impair fertility in males and females.
Click here for full Prescribing Information.

About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor

VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 is the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. Verastem announced in April 2025 that the U.S. Investigational New Drug (IND) application for VS-7375 was cleared and plans to initiate a Phase 1/2a clinical trial in mid-2025. GenFleet’s IND for VS-7375 (known as GFH375 in China) was approved in China in June 2024, and the first patient was dosed in a Phase 1/2 study in July 2024.

On May 13, 2025 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel radioimmunotherapy and antibody-based therapeutic products for the treatment of cancer, reported financial results for the first quarter ended March 31, 2025 (Press release, Y-mAbs Therapeutics, MAY 13, 2025, View Source [SID1234652991]).

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"We closed the first quarter of 2025 demonstrating solid DANYELZA net product revenue, advancement of our novel SADA PRIT platform and programs, and prudent operational spending," said Michael Rossi, President and Chief Executive Officer. "We were pleased to have dosed the first patient in our Trial 1201 where our innovative approach to pretargeted radioimmunotherapy has the potential to improve outcomes for patients in the high-risk population with relapsed/refractory non-Hodgkin Lymphoma. Starting in the first quarter of 2025 we began operating as two separate business units, DANYELZA and Radiopharmaceuticals, and in doing so, our reporting highlights DANYELZA’s segment profit in addition to the resource investments we are making to advance our radioimmunotherapy platform. We look forward to sharing updates on our radiopharmaceutical business strategy, including Part A clinical data from Trial 1001, new optimization data, and new planned target programs and anticipated timelines, during our virtual Radiopharmaceutical R&D update on May 28th."

Recent Corporate Highlights

● On May 7, 2025, Y-mAbs announced that naxitamab-gqgk (DANYELZA) has been recommended by the National Comprehensive Cancer Network ("NCCN") Clinical Practice Guidelines in Oncology (NCCN Guidelines) as a NCCN Category 2A treatment option for high-risk neuroblastoma.
● First patient has been dosed in the Company’s CD38-SADA Phase 1 clinical trial (Trial 1201) evaluating Y-mAbs’ Self-Assembly and Disassembly ("SADA") Pretargeted Radioimmunotherapy ("PRIT") platform for the treatment of patients with relapsed or refractory non-Hodgkin Lymphoma (r/r NHL). The patient was administered both the first protein dose and the 177Lu-DOTA imaging dose. Trial 1201 is a dose-escalation, open-label, single-arm, multi-center trial investigating the safety and tolerability of the CD38-SADA: 177Lu-DOTA Drug Complex in r/r NHL.
o Trial 1201 is designed to investigate the pretargeted delivery of the CD38-SADA protein that binds with high affinity to lymphoma cells, followed by the administration of a radioactive 177Lu-DOTA payload to selectively target the tumor-bound CD38-SADA molecules while minimizing radiation to normal tissues. Part A of the clinical trial is CD38-SADA dose escalation with fixed 177Lu-DOTA payload doses to explore the optimal CD38-SADA protein dose and interval between the SADA protein administration and the payload. The primary endpoints of Part A include tumor imaging and occurrence of dose limiting toxicities ("DLT") in the DLT evaluation period.

● The Company presented preclinical and translational pharmacokinetics (PK) data of CD38-SADA in a poster at the 2025 American Association of Cancer Research ("AACR") Annual Meeting on April 27, 2025 in Chicago, IL. The poster titled "Preclinical and translational pharmacokinetic (PK) modeling of the self-assembling and disassembling (SADA) bispecific fusion protein CD38-SADA for first-in-human (FIH) pretargeted radioimmunotherapy (PRIT)" characterized the plasma concentrations of CD38-SADA in animal models over time and a range of doses. Utilizing in vitro binding kinetic parameters and PK data generated from three studies in mice, the study characterized the concentration- and time-dependent equilibrium between CD38-SADA tetramers and monomers. Using these data, Y-mAbs conducted a series of appropriately scaled human PK simulations, which informed the design and initial dosing regimen of Trial 1201, the Company’s first-in-human Phase 1 clinical trial (Trial 1201) in patients with r/r NHL.
● Y-mabs plans to host a virtual Radiopharmaceutical R&D update on Wednesday, May 28, 2025 where the Company will discuss:
o Part A clinical data from ongoing Phase 1 GD2-SADA clinical trial (Trial 1001), including pharmacokinetic and dosimetry data;
o Updates around the Company’s nonclinical optimization studies for the GD2-SADA asset and plans for clinical implementation; and
o Radiopharmaceutical pipeline strategy, including new planned target programs and anticipated timelines.
● Following the business realignment strategy announced in January 2025, the Company is now organized into two business units: DANYELZA and Radiopharmaceuticals. The Company’s business units are focused on different products and platforms. They are managed separately as each business unit requires different research and development, marketing and other operational investments. Their segment profit/(loss) from operations include certain non-cash costs.
First Quarter 2025 Key Highlights

● Enhanced collaboration with SciClone and other distribution partners with new commercial programs introduced in distribution partners’ territories.
● Continued commercial success with the named patient program for DANYELZA in Turkey with partner INPHARMUS (formerly named TRPharm İlaç Sanayi Ticaret A.Ş. and TRPharm FZ-LLC) and expansion of agreement into new markets.

Financial Results

Revenues

Total revenues for the quarter ended March 31, 2025 were $20.9 million, which was a 5% increase over the $19.9 million of total revenues for the quarter ended March 31, 2024, primarily driven by a $6.7 million increase in Ex-U.S. DANYELZA revenue, partially offset by a $5.2 million decrease in U.S. DANYELZA revenue and $0.5 million decrease related to license revenue recognized in the three months ended March 31, 2024. Total DANYELZA net product revenues for the quarter ended March 31, 2025 were $20.9 million, which was an 8% increase over $19.4 million total DANYELZA net product revenues for the quarter ended March 31, 2024.

The Company’s U.S. DANYELZA net product revenues for the quarter ended March 31, 2025 were $13.4 million, representing a decrease of 28% from the same period in 2024. The decline in the U.S. DANYELZA net product revenues was driven by enrollments in clinical studies and market dynamics.

The Company’s Ex-U.S. DANYELZA net product revenues for the quarter ended March 31, 2025 were $7.5 million, representing an increase of $6.7 million from the same period in 2024. The increase in the Ex-U.S. DANYELZA net product revenues was driven by a $3.8 million increase in net product revenue in Western Asia, where the named patient program launched in late 2024, and increased net product sales in the Eastern Asia, where a new marketing initiative program was introduced in late 2024, and Latin America regions.

As of March 31, 2025, Y-mAbs had delivered DANYELZA to 70 centers across the U.S. since initial launch, with one new account added in the U.S. in the first quarter 2025. During the quarter ended March 31, 2025, approximately 72% of the vials sold in the U.S. were sold outside of Memorial Sloan Kettering Cancer Center ("MSK"), compared to 64% in the fourth quarter ended December 31, 2024.

There was no license revenue for the quarter ended March 31, 2025. During the quarter ended March 31, 2024, the Company had license revenues of $0.5 million, which included license revenue from the Latin America distribution partner, Adium, related to price approval for DANYELZA in Brazil from the Brazilian Medicines Market Regulation Chamber.

Cost of Goods Sold

Cost of goods sold was $3.0 million and $2.1 million for the three months ended March 31, 2025 and 2024, respectively. The increase in cost of goods sold in the three months ended March 31, 2025 compared to the same period in 2024, was driven by increased volumes in Ex-U.S. regions which carry a lower gross margin.

Gross Profit

Gross profit stayed consistent at $17.9 million and $17.8 million for the three months ended March 31, 2025 and 2024, respectively.

Gross margins are 86% and 89% for the three months ended March 31, 2025 and 2024, respectively. Gross margin from total revenues decreased in the three months ended March 31, 2025, which was mainly attributable to the decreased U.S. net product revenues, which are at higher margins compared to our Ex-U.S. regions.

Operating Costs and Expenses

Research and Development

Research and development expenses were $11.4 million and $13.3 million for the three months ended March 31, 2025 and 2024, respectively. The $1.9 million decrease in research and development expenses was primarily attributable to a decrease of $0.7 million in clinical trials due to the timing of completion in the Company’s GD2-SADA program, investment in its ongoing SADA PRIT programs and a $0.9 million decrease in personnel and stock-based compensation costs, partially offset by $0.6 million increase in outsourced manufacturing for investment in the Company’s naxitamab program.

Selling, General, and Administrative

Selling, general, and administrative expenses were $13.1 million for the three months ended March 31, 2025, as compared to $11.4 million for the three months ended March 31, 2024. The $1.7 million increase in selling, general, and administrative expenses was primarily attributable to a $0.8 million increase in personnel and stock-based compensation costs, a $0.5 million charge related to the business realignment expense and $0.4 million in legal expenses recorded in the three months ended March 31, 2025.

Interest and Other Income

Interest and other income for the three months ended March 31, 2025 was $1.4 million compared to $0.4 million for the three months ended March 31, 2024. Interest and other income increased by $1.0 million primarily due to a $1.3 million increase in foreign currency transaction gains, partially offset by a $0.3 million decrease in interest earned from money market fund investments.

Net Loss

Y-mAbs reported a net loss for the quarter ended March 31, 2025, of $5.2 million, or ($0.12) per basic and diluted share, compared to a net loss of $6.6 million, or ($0.15) per basic and diluted share, for the quarter ended March 31, 2024. The decrease in net loss for the quarter ended March 31, 2025 was primarily driven by increased total revenues and increased foreign currency transactional gains, partially offset by increased operating costs and expenses.

Cash and Cash Equivalents

As of March 31, 2025, Y-mAbs had approximately $60.3 million in cash and cash equivalents which, together with anticipated DANYELZA product revenues, is expected to support operations as currently planned into 2027. The Company is currently operating below its anticipated cash investment guidance for the full year 2025. This estimate reflects the Company’s current business plan that is supported by assumptions that may prove to be inaccurate, such that YmAbs could use its available capital resources sooner than it currently expects. The Company continues its efforts to be capital efficient in its operations.

2025 Financial Guidance

Management reiterates its guidance for the full year 2025:

● Anticipated Total Revenues expected to be between $75 million and $90 million;
● Anticipated Total Operating Costs and Expenses, excluding cost of goods sold, expected to be between $116 million and $121 million (Total Operating Costs and Expenses including anticipated cost of goods sold of between $13 million and $15 million is anticipated to be between $129 million and $134 million);
● Anticipated Total Annual Cash Investment expected to be between $25 million and $30 million; and
● Cash and Cash Equivalents anticipated to be sufficient to fund operations as currently planned into 2027.
Management announces its guidance for the second quarter 2025:

● The Company anticipates Total Revenues to be between $17 million and $19 million.
Webcast and Conference Call

Y-mAbs will host a conference call on Tuesday, May 13, 2025, at 8:00 a.m. ET. To listen to the live webcast, please use this link. Prior to the call and webcast, a slide presentation pertaining to the Company’s quarterly earnings will be made available on the Investor Relations section of the Y-mAbs website, www.ymabs.com, shortly before the call begins.