1stOncology™: Cancer Intelligence Service – Page 199 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Genprex Collaborators Present Positive Preclinical Data on Diabetes Gene Therapy for Type 2 Diabetes at the 2026 American Society of Gene and Cell Therapy Annual Meeting

On May 14, 2026 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that its research collaborators presented positive preclinical data on the Company’s diabetes gene therapy drug candidate at the 2026 American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting. The collaborators presented preclinical data demonstrating that the diabetes gene therapy (Pdx1/MafA gene therapy, PM or GPX-002) can reverse hyperglycemia in Type 2 diabetic (T2D) mouse models.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

"We are pleased to have our collaborators present this promising preclinical data in Type 2 diabetes at the ASGCT (Free ASGCT Whitepaper) Annual Meeting," said Ryan Confer, President and Chief Executive Officer at Genprex. "We believe this preclinical data is a pivotal step toward a transformative treatment for Type 2 diabetes by directly addressing beta-cell dysfunction. Our therapy holds the potential to offer long-term glycemic control, moving beyond symptomatic management to fundamentally address the disease for diabetic patients."

The featured Genprex-supported abstract and poster at the 2026 ASGCT (Free ASGCT Whitepaper) Annual Meeting:

Title: "Pancreatic Delivery of AAV-Pdx1/MafA Reverses Hyperglycemia in a Preclinical Model of Type 2 Diabetes"

Abstract ID: 2419

Topic: Gene-Based Therapies in Pre-Clinical Models of Genetic Disease

Poster Presentation Date: Wednesday, May 13, 2026

Poster Presentation Time: 5-6:30 p.m. ET

In this study, eight-week-old male C57BL/6 mice were maintained on a regular diet (RD) or high fat diet (HFD) for 24 weeks. HFD mice then either remained unoperated or underwent retrograde infusion into the pancreatic duct of adeno-associated virus (AAV-8) encoding Pdx1 and MafA (PM) cassettes under the CMV promoter (global–islet cell targeting) or the rat insulin promoter (RIP) (β-cell–specific targeting) or received a control virus. The diet remained unchanged after surgery. At two and/or four weeks after surgery, researchers performed intraperitoneal glucose tolerance testing (IPGTT), insulin tolerance testing (ITT), glucose-stimulated insulin secretion (GSIS), calculated HOMA-IR and assessed glucagon secretion. Mice were then euthanized for pancreatic histology, quantification of β- and α-cell mass, electron microscopy (EM), and islets were isolated for ex-vivo glucose-stimulated insulin secretion (GSIS) and single-cell RNA sequencing. The results at four weeks showed major improvements in the control of diabetes.

At four weeks after surgery, ex-vivo GSIS showed that islets isolated from HFD+CMV-PM-GFP treated mice had insulin secretion similar to islets from RD mice, and both groups had increased insulin secretion

compared to islets from the control HFD groups, indicating improved β-cell function with PM treatment.

Similarly, and importantly, treatment of HFD mice with RIP-PM-GFP, which selectively targets β-cells,

reversed hyperglycemia and improved ex-vivo GSIS. In addition, EM imaging showed that PM treatment in HFD mice increased the number of total and mature insulin granules and decreased the number of immature insulin granules compared with HFD controls. Furthermore, transcriptomic pseudotime analysis demonstrated a shift in β-cells from an immature state toward a more mature state after PM treatment.

PM gene therapy reverses hyperglycemia, likely in large part by specifically enhancing β-cell

function and maturation. This approach is technically translatable to humans using endoscopic retrograde

cholangiopancreatography to deliver PM gene therapy to the pancreas.

To view the poster presented at the 2026 ASGCT (Free ASGCT Whitepaper) Annual Meeting, please visit Genprex’s website here.

(Press release, Genprex, MAY 14, 2026, View Source [SID1234665704])

Fortress Biotech Reports First Quarter 2026 Financial Results and Recent Corporate Highlights

On May 14, 2026 Fortress Biotech, Inc. (Nasdaq: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty income, reported financial results and recent corporate highlights for the first quarter ended March 31, 2026.

Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "The first quarter of 2026 marked a pivotal period for Fortress, highlighted by significant execution across our portfolio and meaningful progress in enhancing long‑term shareholder value. The FDA approval of ZYCUBO for Menkes disease and the subsequent monetization of Cyprium’s PRV for $205 million represent important validation of our business model. We deployed a portion of these proceeds to strengthen our balance sheet through debt reduction, lowering our outstanding principal with Oaktree to $15.0 million. We also continued to expand our pipeline through business development, including Avenue’s acquisition of ATX‑04 from Duke University, a clinically validated program with the potential to address significant unmet need in Pompe disease."

Dr. Rosenwald added, "Looking ahead, we expect to generate increasing royalty revenue from ZYCUBO and UNLOXCYT, along with potential milestone payments across our portfolio. In parallel, AstraZeneca’s regulatory submissions in the EU and Japan for anselamimab (formerly known as CAEL-101), underscore the continued optionality within our partnered assets for potential future sales milestones for Fortress and approval milestones in the U.S. We have a diversified portfolio of commercial, late‑stage, and development‑stage programs and Fortress is well positioned to advance strategic initiatives and drive long‑term value for our shareholders."

Recent Corporate Highlights1:

Regulatory and Monetization Updates

● ZYCUBO Approved for Menkes Disease; Cyprium Sold PRV for $205 Million. In January 2026, the FDA approved ZYCUBO (copper histidinate, formerly known as CUTX-101) for the treatment of Menkes disease in pediatric patients. A PRV was issued at approval and transferred to Cyprium under its agreement with Sentynl Therapeutics, Inc. ("Sentynl"). In March 2026, Cyprium closed the sale of the PRV for gross proceeds of $205 million. Cyprium is also eligible to receive tiered royalties on net sales of ZYCUBO and up to approximately $128 million in aggregate sales milestones from Sentynl.

o In connection with the sale of the PRV, Cyprium redeemed all outstanding shares of its 9.375% Perpetual Preferred Stock pursuant to the previously disclosed terms of such securities.
● Checkpoint Acquired by Sun Pharma; Fortress Establishes Long-Term Royalty Stream. In May 2025, Fortress’ subsidiary, Checkpoint, was acquired by Sun Pharmaceutical Industries, Inc. (together with its subsidiaries and/or associated companies, "Sun Pharma"). Pursuant to the acquisition, Fortress received ~$28 million upfront, with the potential for an additional contingent value right payment of up to $4.8 million and a 2.5% royalty on future net sales of UNLOXCYT (cosibelimab-ipdl). UNLOXCYT was approved by the FDA in December 2024 to treat metastatic or locally advanced cutaneous squamous cell carcinoma ("cSCC") in patients who are not candidates for curative surgery or radiation and was commercially launched in January 2026.

Commercial Portfolio Updates

● Journey Medical Expands Payer Access for Emrosi. At the end of March 2025, our partner company Journey Medical Corporation ("Journey Medical") commercially launched Emrosi (40mg Minocycline Hydrochloride Modified-Release Capsules, consisting of 10mg immediate release and 30mg extended release pellets), also known as DFD-29, for inflammatory lesions of rosacea. Emrosi was approved by the FDA in November 2024 and is available by prescription at specialty pharmacy chains. In April 2026, Journey Medical announced that it secured a contract with a third major group purchasing organization (GPO) for Emrosi. As such, payer access for Emrosi expanded to over 150 million commercial lives as of April 1, 2026, which equates to approximately 85% of all commercial lives in the United States that have access to Emrosi. Journey Medical reported net product revenues of $15.9 million for the first quarter of 2026, compared to net product revenues of $13.1 million for the first quarter ended March 31, 2025.
● Royalties. In the first quarter of 2026, Cyprium recognized $0.1 million in royalty revenue on net sales of ZYCUBO.

Clinical Updates

● Phase 3 CARES Results for Anselamimab (CAEL-101); Regulatory Submission of Prespecified Subgroup Analysis Planned. In July 2025, AstraZeneca announced that anselamimab (formerly known as CAEL-101) did not achieve statistical significance for the primary endpoint in its Phase III Cardiac Amyloid Reaching for Extended Survival ("CARES") clinical program for Mayo stages IIIa and IIIb AL amyloidosis patients. However, the drug showed clinically meaningful improvement in a prespecified subgroup and was well tolerated. AstraZeneca indicated that the company plans to submit the prespecified subgroup analysis from the CARES trials to regulatory authorities and disclosed regulatory submissions in the EU and Japan.

General Corporate:

● In March 2026, Fortress made aggregate prepayments on its loan with Oaktree, including a prepayment in connection with the sale of the PRV, reducing the outstanding principal balance to $15.0 million.
● In February 2026, Avenue entered into an exclusive worldwide license agreement with Duke University to acquire patent and know-how rights pertaining to ATX-04 (clenbuterol), a well-characterized small-molecule β2-adrenergic agonist, in clinical development for the treatment of Pompe disease. ATX-04 is a selective β2-adrenergic agonist with human proof-of-concept data demonstrating improved muscle function and enhanced response to enzyme replacement therapy. Avenue anticipates meeting with the FDA in 2026 to discuss and align on the design of a potential single pivotal trial for ATX-04 for Pompe disease.

Financial Results:

● As of March 31, 2026, Fortress’ consolidated cash and cash equivalents totaled $255.8 million, compared to $79.4 million as of December 31, 2025, an increase of $176.5 million during the quarter.
● Fortress’ consolidated cash and cash equivalents totaling $255.8 million as of March 31, 2026, includes $209.9 million attributable to Fortress and the private subsidiaries, $2.4 million attributable to Avenue, $16.3 million attributable to Mustang Bio and $27.2 million attributable to Journey Medical.
o Fortress’ consolidated cash and cash equivalents totaled $79.4 million as of December 31, 2025, and includes $35.2 million attributable to Fortress and private subsidiaries, $2.9 million attributable to Avenue, $17.3 million attributable to Mustang and $24.1 million attributable to Journey Medical.

● Fortress’ consolidated net revenue totaled $16.0 million for the first quarter ended March 31, 2026, of which $15.9 million is generated from Journey Medical’s marketed dermatology products. This compares to consolidated revenue totaling $13.1 million for the first quarter of 2025.
● Consolidated research and development expenses totaled $0.5 million for the first quarter ended March 31, 2026, compared to $3.9 million for the first quarter ended March 31, 2025.
● Consolidated selling, general and administrative costs were $15.9 million for the first quarter ended March 31, 2026, compared to $25.7 million for the first quarter ended March 31, 2025.
● Consolidated net income attributable to common stockholders was $108.4 million, or $3.44 per share (basic) and $2.82 per share (diluted), for the first quarter ended March 31, 2026, compared to net loss attributable to common stockholders of $(12.7) million, or $(0.48) per share basic and diluted for the first quarter ended March 31, 2025.

(Press release, Fortress Biotech, MAY 14, 2026, View Source [SID1234665703])

CORMEDIX THERAPEUTICS REPORTS FIRST QUARTER 2026 FINANCIAL RESULTS AND PROVIDES BUSINESS UPDATE

On May 14, 2026 CorMedix Therapeutics (Nasdaq: CRMD) reported financial results for the first quarter ended March 31, 2026 and provided an update on its business.

Recent Corporate Highlights:

CorMedix announces $127.4 million of net revenue for the first quarter of 2026, reflecting strong first quarter execution and positive underlying demand trends. The Company also recognized net income of $38.6 million and adjusted EBITDA of $70.0 million.(1) Basic and fully diluted EPS were $0.48 and $0.43 per share, respectively, for the quarter
DefenCath (taurolidine and heparin) sales contributed $97.5 million of net revenue in the quarter, bolstered by higher utilization of DefenCath by outpatient dialysis customers as well as a non-recurring $9.0 million favorable change in estimate related to certain sales allowances. The acquired Melinta portfolio contributed $29.9 million, reflecting typical, first quarter purchasing patterns for the anti-infective portfolio.
CorMedix updates previously established guidance for 2026 net revenue and adjusted EBITDA. The Company increases full-year 2026 net revenue guidance to a range of $325 to $345 million, and full-year adjusted EBITDA guidance to a range of $115 to $135 million.
On April 27, 2026, CorMedix announced positive Phase III topline results from the global ReSPECT clinical trial evaluating REZZAYO (rezafungin for injection) for prophylaxis of invasive fungal diseases in adult patients undergoing allogeneic hematopoietic stem cell transplantation. CorMedix is actively working together with its global partner to prepare for FDA submission of the sNDA, expected in the second half of this year, and targeting for a potential commercial launch for the expanded indication in 2027.
The ongoing Phase 3 study of taurolidine/heparin catheter lock solution in TPN patients continues to enroll patients and is currently trending to completion in 2028. The Company is taking appropriate steps to accelerate enrollment trajectory, including the opening of new study sites and the submission of a protocol amendment to FDA, which, if approved, would remove certain exclusion criteria and broaden patient enrollment.
Cash and short-term investments, excluding restricted cash, at March 31, 2026 totaled $178.1 million.
Joseph Todisco, CorMedix Chairman & CEO, commented, "CorMedix has entered 2026 with strong momentum across all areas of our business. DefenCath continues to exceed expectations despite pending TDAPA expiration and demonstrates strong underlying utilization demand. In addition, we are advancing a pipeline of late-stage opportunities, including REZZAYO for prophylaxis, which we expect will meaningfully expand our long-term revenue opportunity. Lastly, we have delivered significant profitability and cash generation, allowing us to reinvest in growth, as well as shareholder value creation through stock repurchases, while preserving financial flexibility for new business development initiatives."

(1) Adjusted EBITDA is a non-GAAP financial measure and excludes non-cash items such as depreciation, amortization, stock-based compensation, interest and other income and expense, taxes and certain non-recurring items. See "Non-GAAP Financial Measures" on the following pages for additional information regarding the use of EBITDA and Adjusted EBITDA and a reconciliation to the most comparable GAAP measure.

First Quarter 2026 Financial Highlights

For the first quarter of 2026, CorMedix recorded $127.4 million in net revenue, comprised of $97.5 million in sales of DefenCath and $29.9 million in revenue associated with the acquired Melinta portfolio, an increase from $39.1 million in net revenue in the comparable period of 2025. DefenCath sales in the quarter were positively impacted by a non-recurring $9.0 million change in estimate related to certain sales allowances. DefenCath sales increased year over year largely due to the onboarding of a large dialysis organization in mid-2025. As the Melinta acquisition occurred in August 2025, the first quarter of 2025 included net revenue from only sales of DefenCath

Total operating expenses in the first quarter of 2026 were $41.5 million, compared with $17.4 million in the first quarter of 2025, an increase of approximately 139%. The increase of $24.1 million over the prior period was driven primarily by the contribution of operating expenses from the Melinta acquisition for the full quarter and reflects the larger combined company.

Research and development (R&D) expenses in the first quarter of 2026 were $7.2 million, compared with $3.2 million for the same period in 2025. The increase in R&D was primarily due to an increase in personnel and clinical trial services in support of the ongoing clinical programs, including pediatric studies for several of our brands and the continued investment in the development of DefenCath for the TPN indication.

Selling and marketing expense increased approximately 180% to $12.5 million in first quarter of 2026 from $4.5 million in the first quarter of 2025. The increase was primarily due to higher personnel cost associated with the larger product portfolio and related marketing programs.

General and administrative expenses increased approximately 124% to $21.7 million in the first quarter of 2026 from $9.7 million in the first quarter of 2025. The increase was primarily attributable to higher costs associated with operating as a combined company following the acquisition, including higher personnel, information technology, branded prescription drug, legal and facilities costs.

CorMedix recorded net income of $38.6 million, or $0.48 and $0.43 per basic and diluted share, respectively, in the first quarter of 2026, compared with net income of $20.6 million, or $0.32 and $0.30 per basic and diluted share, respectively, in the first quarter of 2025. Also for the first quarter of 2026, CorMedix reported adjusted EBITDA of $70.0 million, compared to adjusted EBITDA of $23.6 million in the first quarter of 2025.

The Company reported cash and cash equivalents of $178.1 million at March 31, 2026, excluding restricted cash. The Company believes that it has sufficient resources to fund operations for at least twelve months from the issuance of the Company’s Quarterly Report on Form 10-Q.

Conference Call Information

The management team of CorMedix will host a conference call and webcast today, May 14, 2026, at 8:30AM Eastern Time, to discuss recent corporate developments and financial results. Call details and dial-in information are as follows:

May 14, 2026 @ 8:30am ET

Domestic: 1-844-676-2922

International: 1-412-634-6840

Webcast: Webcast Link

(Press release, CorMedix, MAY 14, 2026, View Source [SID1234665701])

Cellectar Biosciences Reports First Quarter 2026 Financial Results and Provides Corporate Updates

On May 14, 2026 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery and development of drugs for the treatment of cancer, reported financial results for the quarter ended March 31, 2026, and provided a corporate update.

"The first part of 2026 was a pivotal period for Cellectar as we executed across our pipeline and capital strategies to position the company for value creation," said James Caruso, president and chief executive officer of Cellectar. "With the support of industry-leading healthcare focused investors, we successfully completed a financing of up to $140 million, providing the necessary resources to advance iopofosine through key U.S. regulatory milestones and potential commercialization. The recently reported positive 12-month follow-on data from our CLOVER WaM study reinforce our confidence that iopofosine can provide meaningful patient benefits and meet regulatory expectations, supporting our plans to initiate a Phase 3 confirmatory study and file for accelerated approval with the FDA," Mr. Caruso continued.

"In parallel, we expanded our radio-conjugate pipeline with the enrollment of the first patients in our Phase 1b study of CLR 125 in triple negative breast cancer, a challenging solid tumor cancer with a substantial unmet medical need. Together, these advances underscore the strength of our radiopharmaceutical platform and potential to deliver meaningful new treatment options to patients battling a variety of difficult-to-treat cancers," concluded Mr. Caruso.

First Quarter 2026 and Recent Corporate Highlights

Iopofosine I 131, the company’s Phospholipid Drug Conjugate (PDC) designed to provide targeted delivery of iodine-131 (radioisotope)
Reported positive 12-month follow-up data from all patients in the Phase 2b CLOVER WaM study evaluating iopofosine I 131 in relapsed/refractory Waldenström Macroglobulinemia, which demonstrated strong and consistent efficacy in both BTKi-exposed and BTKi-refractory patients. The minimum 12-month follow-up data aligns with the expectations set by the U.S. Food and Drug Administration (FDA) and positions the company for accelerated approval submission and the initiation of the confirmatory study.
Notably, the primary and secondary endpoints were both achieved in the protocol study population (n=55), with 61.8% achieving a major response rate (MRR) and a median duration of response (DoR) of 17.8 months. Additional data points included:
Overall response rate (ORR): 83.6%
Median progression-free survival (PFS): 13.5 months
Very good partial response/complete response rate (VGPR/CR): 14.5%
Disease control rate (DCR):98.2
Selected to present data from the CLOVER WaM study of iopofosine I 131 in r/r WM patients at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO) (Free ASCO Whitepaper) taking place May 29 – June 2, 2026. The poster presentation will highlight efficacy results from a subset of patients treated with iopofosine I 131 immediately post-Bruton Tyrosine Kinase inhibitor (BTKi) therapy. Details of the poster presentation are as follows:
Title: "Iopofosine I-131 after BTK inhibitors in Waldenström macroglobulinemia: CLOVER-WaM subgroup efficacy and safety"
Poster: 592
Date/Time: June 1, 2026, 9:00 AM – 12:00pm CDT
Presenter: Jarrod Longcor
Advancing plans to initiate a Phase 3 confirmatory trial of iopofosine I 131 as a treatment for WM and file for accelerated approval with the U.S. FDA in alignment with the FDA requirements. This Phase 3 study will be a comparator, randomized controlled study with approximately 100 WM patients per arm, with full patient enrollment projected within 18-24 months of the first patient admitted to the study.
Continuing to work with the European Medicines Agency (EMA) to file for a Conditional Marketing Approval (CMA) for iopofosine I 131 as a treatment option for post-BTKi refractory patients with WM.
CLR 121125 (CLR 125), an iodine-125 Auger-emitting program targeted for solid tumors
Announced the enrollment of the first patient in the Phase 1b trial evaluating CLR 125 in refractory triple negative breast cancer (TNBC). The Company anticipates activating additional study sites throughout the second quarter and will provide dosimetry, safety and efficacy updates in the second quarter and throughout the balance of 2026.
Corporate
In May 2026, the Company entered into a securities purchase agreement with certain institutional investors to issue and sell an aggregate of approximately $35 million upfront and up to $105 million of milestone-based securities in a registered direct offering of common stock and a concurrent private placement of common stock, pre-funded warrants and milestone-based warrants.
The oversubscribed financing was led by Nantahala Capital, with participation from Balyasny Asset Management, Caligan Partners, Janus Henderson Investors, SilverArc Capital Management and other dedicated healthcare funds. In connection with the Offering, Andrew Gu of Nantahala Capital Management, LLC will join Cellectar’s Board of Directors.
2026 Financial Highlights

Cash and Cash Equivalents: As of March 31, 2026, the company had cash and cash equivalents of $8.3 million, compared to $13.2 million as of December 31, 2025, which does not reflect net proceeds of approximately $31 million from the May 2026 offering. The company believes its cash balance as of March 31, 2026, along with funds from the May 2026 financing, are adequate to fund its budgeted operations into the second quarter of 2027, including the initiation costs for the iopofosine I 131 confirmatory study in WM.
Research and Development Expenses: R&D expenses for the three months ended March 31, 2026, were approximately $3.0 million, compared to approximately $3.4 million for the three months ended March 31, 2025. The overall decrease was primarily a result of reduced clinical and preclinical study costs, partially offset by increased spending for product manufacturing processes.
General and Administrative Expenses: G&A expenses for the three months ended March 31, 2026, were approximately $2.8 million, compared to approximately $3.0 million for the same period in 2025. The decrease was primarily a result of reduced personnel costs.
Net Loss: The net loss attributable to common stockholders for the three months ended March 31, 2026, was $5.7 million, or $1.33 per share, compared to $6.6 million, or $4.30 per share, in the three months ended March 31, 2025.
Conference Call & Webcast Details
Cellectar management will host a conference call and webcast today, May 14, 2026, at 8:30 AM Eastern Time to discuss these results and answer questions. Stockholders and other interested parties may participate in the conference call by dialing 1-800-717-1738. A live webcast of the conference call can be accessed in the "Events & Presentations" section of Cellectar’s website at www.cellectar.com. A recording of the webcast will be available and archived on the Company’s website for approximately 90 days.

(Press release, Cellectar Biosciences, MAY 14, 2026, View Source [SID1234665700])

Celcuity Inc. Reports Release of First Quarter 2026 Financial Results and Provides Corporate Update

On May 14, 2026 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company focused on the development of targeted therapies for the treatment of multiple solid tumor indications, reported financial results for the first quarter ended March 31, 2026 and other recent business developments.

"With positive results in both cohorts of the pivotal VIKTORIA-1 study, we believe gedatolisib regimens have the potential to advance the standard of care in the second-line setting for a significant number of patients with HR+/HER2- advanced breast cancer, regardless of PIK3CA status," said Brian Sullivan, CEO and co-founder of Celcuity. "We are on track to launch gedatolisib commercially in anticipation of its potential FDA approval in the third quarter of 2026, and we look forward to bringing this important therapy to physicians treating patients with advanced breast cancer."

Mr. Sullivan added, "Our positive Phase 3 results, combined with our promising Phase 1b clinical trial results in treatment-naive late-stage patients, provide a strong scientific rationale to evaluate gedatolisib combinations as first-line therapy. By expanding our VIKTORIA-2 study to enable evaluation of treatment-naive patients who have endocrine-sensitive breast cancer, we are positioning gedatolisib regimens to potentially be available for nearly all patients in the first-line setting, irrespective of their endocrine sensitivity or PIK3CA status."

First Quarter 2026 Business Highlights and Other Recent Developments

● Celcuity reported positive topline results from the PIK3CA mutant-type ("MT") cohort of the Phase 3 VIKTORIA-1 clinical trial evaluating gedatolisib in combination with fulvestrant with or without palbociclib in patients with hormone receptor positive ("HR+"), human epidermal growth factor receptor 2 negative ("HER2-") ("HR+/HER2-"), PIK3CA MT locally advanced or metastatic breast cancer ("ABC").

○ The primary efficacy analysis of gedatolisib combined with fulvestrant and palbociclib ("gedatolisib triplet") demonstrated a statistically significant and clinically meaningful improvement in progression-free survival ("PFS") compared with alpelisib, a PI3Kα inhibitor, and fulvestrant.

○ The secondary endpoint comparing gedatolisib in combination with fulvestrant ("gedatolisib doublet") versus alpelisib plus fulvestrant, which was not part of the primary efficacy analysis in the hierarchical order, also demonstrated a statistically significant and clinically meaningful improvement in PFS.

○ Both gedatolisib regimens were generally well tolerated, with manageable safety profiles, and no new safety signals.

○ Detailed data for the gedatolisib triplet and doublet regimens will be presented in a late-breaking abstract ("LBA") oral session on June 2, 2026, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual Meeting in Chicago, Illinois.

○ Celcuity intends to submit these data to the FDA in the third quarter as a supplemental New Drug Application ("sNDA") and to submit VIKTORIA-1 data to other regulatory authorities outside the U.S. following the sNDA submission.

● The Phase 3 VIKTORIA-2 clinical trial now includes two studies, Study 1 and Study 2, each with independent statistical analysis plans that include primary endpoints for their respective intent-to-treat populations. Study 1, which is ongoing, is evaluating the efficacy and safety of gedatolisib in combination with palbociclib and fulvestrant in approximately 440 patients with endocrine-resistant HR+/HER2- ABC. Study 2, which was added in conjunction with a VIKTORIA-2 protocol amendment, is evaluating the efficacy and safety of gedatolisib in combination with palbociclib and letrozole in approximately 740 patients with treatment-naive endocrine-sensitive HR+/HER2- ABC. Eligible patients include those whose cancer relapsed or progressed 12 months or more after completion of adjuvant endocrine therapy, or those with de novo metastatic disease without prior endocrine therapy exposure. Approximately 60,000 adults are newly diagnosed each year in the United States with endocrine-sensitive HR+/HER2- ABC.1

● To support its long-term lifecycle development plan, Celcuity submitted its first patent application to the United States Patent and Trademark Office ("USPTO") for a subcutaneous formulation of gedatolisib that would enable a patient to receive gedatolisib as an injection as an alternative to an infusion. Development of the subcutaneous gedatolisib formulation is ongoing with the goal of demonstrating clinical equivalence to the current intravenous formulation of gedatolisib. The subcutaneous formulation is aimed to support potential future indications for gedatolisib regimens that may result in duration of treatment periods greater than several years.

● In January 2026, the FDA accepted the submission of Celcuity’s New Drug Application ("NDA") for gedatolisib in HR+/HER2- PIK3CA wild-type ("WT") ABC. The FDA granted Priority Review and assigned a Prescription Drug User Fee Act ("PDUFA") goal date of July 17, 2026.

First Quarter 2026 Financial Results

Unless otherwise stated, all comparisons are for the first quarter ended March 31, 2026, compared to the first quarter ended March 31, 2025.

Net loss for the first quarter of 2026 was $52.8 million, or $0.97 per share, compared to a net loss of $37.0 million, or $0.86 per share, for the first quarter of 2025. Non-GAAP adjusted net loss for the first quarter of 2026 was $46.8 million, or $0.86 per share, compared to non-GAAP adjusted net loss of $34.7 million, or $0.81 per share, for the first quarter of 2025. Non-GAAP adjusted net loss excludes stock-based compensation expense, non-cash interest expense, and non-cash interest income. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States ("GAAP") to non-GAAP financial measures, please see the financial tables at the end of this press release.

Total operating expenses were $50.5 million for the first quarter of 2026, compared to $36.1 million for the first quarter of 2025.

Research and development ("R&D") expenses were $33.1 million for the first quarter of 2026, compared to $29.8 million for the prior year period. The $3.3 million increase in R&D expenses was primarily due to a $3.0 million increase in employee-related and consulting expenses. The remaining increase was primarily due to a $5.4 million increase in manufacturing and other costs, partially offset by a $5.1 million decrease in clinical trial costs, which was primarily driven by decreased costs for the VIKTORIA-1 Phase 3 clinical trial.

Selling, general and administrative ("SG&A") expenses were $17.4 million for the first quarter of 2026, compared to $6.3 million for the prior year period. The $11.1 million increase in SG&A expenses was primarily due to an $8.7 million increase in employee-related and consulting expenses, of which $6.6 million was due to commercial headcount additions and other launch-related activities, and a $2.4 million increase primarily due to software costs, professional fees and other costs.

Net cash used in operating activities for the first quarter of 2026 was $55.1 million, compared to $35.9 million for the prior year period. Cash, cash equivalents and short-term investments were $387.1 million at the end of the first quarter of 2026. We expect cash, cash equivalents, investments and drawdowns on our debt facility to finance our operations through 2027.

Webcast and Conference Call Information

To participate in the teleconference, domestic callers should dial 1-800-717-1738 and international callers should dial 1-646-307-1865.

A live webcast presentation can also be accessed using this weblink: View Source;tp_key=2f73ec65ba. A replay of the webcast will be available on the Celcuity website following the live event.

(Press release, Celcuity, MAY 14, 2026, View Source [SID1234665699])