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.

On May 13, 2025 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class1 targeted and immune-mediated therapeutics to fight cancer, reported financial results for the quarter ended March 31, 2025 and provided a corporate update (Press release, Tempest Therapeutics, MAY 13, 2025, View Source [SID1234652990]).

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"The amezalpat program continues to produce data that reinforce its potential as a cancer therapy, most recently in a presentation at AACR (Free AACR Whitepaper) showing that amezalpat reduced immunosuppression and activated the immune system to attack tumors. We were pleased to present these data that elucidate one part of the amezalpat mechanism of action and support the positive randomized Phase 2 data, including the benefit seen in patients with markers of immune resistance," said Stephen Brady, president and chief executive officer of Tempest. "We are actively engaged in exploring strategic alternatives to advance our promising clinical-stage programs and maximize stockholder value and, given the data, continue to have strong conviction in the potential of our oncology portfolio to drive meaningful impact for patients facing cancer."

Recent Highlights

Amezalpat (TPST-1120) (clinical PPARα antagonist):
Reported new data at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting supporting the immune component of amezalpat’s dual mechanism of action and reinforcing its potential as a novel cancer treatment.
Granted both Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) for amezalpat for the treatment of patients with HCC.
TPST-1495 (clinical dual EP2/4 prostaglandin receptor antagonist):
Granted Orphan Drug designation by the FDA to treat patients with FAP.
Received a "Study May Proceed" letter from the FDA for the Phase 2 trial for the treatment of FAP.
This trial, run by CP-CTNet and financially supported by the NCI’s Division of Cancer Prevention, underscores the urgent need for innovative cancer prevention strategies in high-risk patient populations. The Phase 2 study is expected to begin in 2025.
Corporate:
Announced (i) plans to explore a full range of strategic alternatives to advance the company’s promising clinical-stage programs and maximize stockholder value and (ii) a reduction in force that was completed on April 30, 2025.
Using cash on hand, the company repaid $3.5 million in full satisfaction of Loan and Security Agreement with Oxford Finance LLC in April 2025.

Financial Results

First Quarter 2025

Tempest ended the quarter with $21.5 million in cash and cash equivalents, compared to $30.3 million on December 31, 2024. The decrease was primarily due to cash used in operating activities, offset by proceeds of $1.5 million from the at-the-market offering program.
Net loss and net loss per share for the quarter were $10.9 million and $3.16, respectively, compared to $7.9 million and $4.62, respectively, for the same period in 2024.
Research and development expenses for the quarter were $7.6 million compared to $4.3 million for the same period in 2024. The $3.3 million increase was primarily due to an increase in costs incurred from engaging contract research and manufacturing organizations in preparation for our pivotal Phase 3 trial of amezalpat for the treatment of first-line HCC.
General and administrative expenses for the quarter were $3.3 million compared to $3.6 million for the same period in 2024. The $0.3 million decrease was primarily due to a decrease in consulting services.

On May 13, 2025 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported financial results for the first quarter ended March 31, 2025, and provided a corporate update (Press release, Sellas Life Sciences, MAY 13, 2025, View Source [SID1234652989]).

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"We are very encouraged by the strong momentum across our pipeline," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "The positive overall survival data in cohort 3 from the ongoing Phase 2 trial of SLS009 in r/r AML, showing a median OS that exceeds all historical benchmarks by over 3 times, further underscores the transformative potential of SLS009 for many underserved patients. In parallel, our new preclinical findings demonstrating the ability of SLS009 to overcome TP53-driven resistance, along with the promising clinical efficacy from the ongoing Phase 2, give us renewed optimism for patients across different genetic AML mutations. We look forward to presenting further data on SLS009 at ASCO (Free ASCO Whitepaper), highlighting its preclinical efficacy in ASXL1-mutated colorectal cancer lines. With the full topline Phase 2 data of SLS009 anticipated soon, and the final analysis of our Phase 3 pivotal REGAL trial of GPS in AML expected later this year, we are well-positioned for an exciting and meaningful 2025."

Recent Corporate Highlights:

Announced Positive Overall Survival in Cohort 3 from the Ongoing Phase 2 Trial of SLS009 in r/r AML: The data demonstrated that patients with AML-Myelodysplasia-Related Changes (AML-MRC) achieved a mOS of 8.9 months, while all relapsed or refractory to venetoclax-based regimens patients receiving 30 mg BIW achieved a mOS of 8.8 months, far surpassing the historical benchmark of 2.5 months. In addition, the therapy demonstrated a 67% ORR in patients with AML-MRC and 46% in all evaluable patients, significantly exceeding the targeted 20% ORR. The trial continues with full data and FDA regulatory path feedback expected in 1H 2025.

Presented Preclinical Data Highlighting Efficacy of SLS009 in TP53 Mutated AML at the 2025 AACR (Free AACR Whitepaper) Conference: Preclinical data suggest that SLS009 can induce apoptosis downstream of p53 by targeting critical proteins such as MCL-1 and survivin, regardless of p53 status. Immunoblot analysis reveals near-complete removal of these proteins in treated cells within 8 hours of exposure to SLS009. Furthermore, the treatment reduced TP53-mutated leukemia cell populations by up to 97% in combination with azacitidine–venetoclax, and by up to 80% as monotherapy.

Preclinical Efficacy of SLS009 in ASXL1 Mutated Colorectal Cancer to be Showcased at ASCO (Free ASCO Whitepaper) 2025: The poster, entitled, In vitro efficacy of CDK9 inhibitor tambiciclib (SLS009) in ASXL1 mutated colorectal cancer cell lines, will be presented on Monday, June 2, 2025, 1:30 PM-4:30 PM CDT.

Announced Positive Outcome of Interim Analysis for Phase 3 REGAL Trial of GPS in AML: The interim futility, efficacy, and safety analysis was designed to assess whether the therapy is safe, demonstrates potential efficacy, and merits continuation. The IDMC’s review supports the continuation of the study according to its original protocol. Based on this positive evaluation, GPS has shown preliminary signals of effectiveness, allowing the trial to advance toward completion. Fewer than 50% of enrolled patients were confirmed deceased after the median follow-up of 13.5 months, indicating a median survival of over 13.5 months in the trial vs. a historical median survival of 6 months for conventional therapy, as reported in a similar Phase 2 study. The next and final analysis will be conducted once 80 events (deaths) are reached, further determining the potential of GPS in addressing the needs of AML patients. SELLAS anticipates that 80 events will be reached this year.

Announced Promising Data from Phase 2a Trial of SLS009 in Combination with Zanubrutinib in DLBCL: The trial, conducted and funded by GenFleet Therapeutics (Shanghai), Inc. ("Genfleet"), was an open-label single-arm multicenter Phase 2a study in China evaluating SLS009 in combination with BTK inhibitor, Brukinsa (zanubrutinib) in r/r DLBCL. The results showed an overall response rate (ORR) of 67%, more than double the expected ORR of zanubrutinib alone. Among responders, one achieved complete response (CR), while three had partial response (PR) with target lesion shrinkages of 89%, 78%, and 56%, respectively. As of the last follow-up, after the median of 4.6 (range: 1.4 – 7.4) months follow-up, median overall survival (OS) was not reached, and 6 out of 9 patients were alive. GenFleet will determine the next steps in development around lymphoma as SELLAS’ focus remains on AML and spliceosome–chromatin mutations, including ASXL1 mutations.

PIVOT – Received Preliminary Data for Pediatric Acute Lymphoblastic Leukemia (ALL) Patients Derived Xenografts (PDX): The experiment conducted and funded by the National Institute of Health (NIH) through the PIVOT program included 27 patient-derived ALL tumors from pediatric patients. Tumors were xenografted in mice in two groups: vehicle control arm and SLS009 arm. Mice were treated with a fractionated dose once per week for 6 consecutive weeks. The treatment was well tolerated. For all models, median survival was approximately tripled in the SLS009 arm compared to the vehicle control arm. SLS009 demonstrated delayed progression in 25/27 (93%) models and more than 2 times longer time to progression in 15/27 (56%) of ALL models. In addition, there were complete responses (CR) in 2 models, and in one of the two models, CR was maintained after the treatment had been completed until the end of the study (4 months). Among 7 KMT2A rearranged models, time to progression was extended in all 7 models, and in 6/7 (86%) time to progression was more than doubled.

Raised $25.0 Million of Gross Proceeds from a Registered Direct Offering Priced At-the-Market under Nasdaq Rules: On January 28, 2025, SELLAS announced the closing of a $25 million registered direct offering with a single healthcare-focused institutional investor before deducting placement agent’s fees and related offering expenses. The net proceeds from the offering strengthens the Company’s financial position and will be used for working capital purposes and general corporate procedures, including the purchase of any pending or future acquisitions.

Financial Results for the First Quarter 2025:

R&D Expenses: Research and development expenses for the quarter ended March 31, 2025, were $3.2 million, compared to $5.1 million for the same period in 2024. The $1.9 million decrease was primarily due to decreases in clinical trial expenses, manufacturing costs, and clinical drug supply purchases, and clinical and regulatory consulting costs primarily driven by the completion of enrollment in the REGAL study in the first quarter of 2024.

G&A Expenses: General and administrative expenses for the first quarter of 2025 were $2.9 million, as compared to $4.5 million for the same period in 2024. The $1.6 million decrease was primarily attributable to a decrease in personnel related expenses driven by the initial recognition of a one-time severance charge during the three months ended March 31, 2024, and a decrease in headcount, and decreases in professional fees and other general and administrative expenses.

Net Loss: The net loss was $5.8 million for the first quarter of 2025, or a basic and diluted loss per share of $0.07, as compared to a net loss of $9.6 million for the first quarter of 2024, or a basic and diluted loss per share of $0.21.

Cash Position: As of March 31, 2025, cash and cash equivalents totaled approximately $28.4 million. Subsequent to March 31, 2025, the Company received $4.0 million in proceeds from the exercise of warrants.

On May 13, 2025 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a clinical-stage precision oncology company, reported financial results for the first quarter ended March 31, 2025 (Press release, Repare Therapeutics, MAY 13, 2025, View Source [SID1234652988]).

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"During the first quarter of 2025 we continued our efforts to create long-term value for our shareholders via partnering and by advancing our novel pipeline programs," said Steve Forte, President, Chief Executive Officer and Chief Financial Officer of Repare. "We announced a strategic partnership with DCx Biotherapeutics to out-license our discovery platforms, and we are exploring a full range of strategic alternatives and partnerships across our portfolio. We are well-positioned from an operational and financial standpoint to drive our clinical pipeline to key inflection points and remain on track to report initial data for both the LIONS and POLAR trials in the second half of this year."

First Quarter 2025 and Recent Portfolio Highlights:

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Announced out-licensing of its discovery platforms to DCx Biotherapeutics
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Repare announced it out-licensed its early-stage discovery platforms, including certain platform and program intellectual property, to DCx Biotherapeutics Corporation ("DCx"). In connection with this agreement, Repare will receive upfront and near-term payments totaling $4.0 million, as well as a 9.99% equity position in DCx, including certain dilution protection rights, and is eligible to receive potential future out-licensing, clinical and commercial milestone payments, as well as low single-digit sales royalties for the development of certain products by DCx. Additionally, DCx will retain approximately 20 of Repare’s preclinical research employees.
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RP-3467: Potential best-in-class, oral Polθ ATPase/helicase inhibitor
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Repare is conducting a Phase 1 clinical trial of RP-3467 (POLAR), dosing patients alone and in combination with the poly-ADP ribose polymerase (PARP) inhibitor, olaparib. POLAR is a multicenter, open-label, dose-escalation Phase 1 clinical trial designed to investigate the safety, pharmacokinetics, pharmacodynamics, and preliminary clinical activity of RP-3647 alone or in combination with olaparib in adults with locally advanced or metastatic epithelial ovarian cancer, metastatic breast cancer, metastatic castration-resistant prostate cancer, or pancreatic adenocarcinoma.
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Upcoming expected milestone:

Q3 2025: Topline safety, tolerability and early efficacy data from the POLAR trial in monotherapy and in combination with olaparib.
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RP-1664: First-in-class, oral selective PLK4 Inhibitor
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Repare completed enrolment of 29 patients in its Phase 1 LIONS clinical trial evaluating RP-1664 as a monotherapy in adult and adolescent patients with TRIM37-high solid tumors. LIONS is a first-in-human, multicenter, open-label Phase 1 clinical trial designed to investigate safety, pharmacokinetics, pharmacodynamics and the preliminary efficacy of RP-1664.
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Upcoming expected milestone:

Q4 2025: Initial topline safety, tolerability and early efficacy data from the LIONS trial
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Lunresertib (RP-6306)
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Repare is currently evaluating lunresertib in combination with Debio 0123, a highly selective, brain-penetrant, clinical WEE1 inhibitor, in patients with advanced solid tumors harboring CCNE1 amplification or FBXW7 or PPP2R1A deleterious alterations as part of an ongoing 50/50 cost sharing collaboration with Debiopharm. Repare does not intend to continue to develop lunresertib in any other trials, absent securing a partnership with a development partner.
First Quarter 2025 Financial Results

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Cash, cash equivalents and marketable securities: Cash, cash equivalents and marketable securities as of March 31, 2025 were $124.2 million, as compared to $152.8 million as of December 31, 2024. The Company believes that its cash, cash equivalents, and marketable securities are sufficient to fund its current operational plans through 2027.
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Revenue from collaboration agreements: Revenue from collaboration agreements was nil and $52.4 million for the three months ended March 31, 2025 and 2024, respectively.
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Research and development expense, net of tax credits (Net R&D): Net R&D expenses were$20.3 million and $33.0 million for the three months ended March 31, 2025 and 2024, respectively.
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General and administrative (G&D) expenses: G&A expenses were $7.7 million and $8.6 million for the three months ended March 31, 2025 and 2024, respectively.
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Net (loss) income: Net loss was $30.0 million, or $0.71 per diluted share, and $13.2 million, or $0.30 per diluted share, for the three months ended March 31, 2025 and 2024, respectively.