Nanobiotix Announces Full Results From Completed Phase 1 Study Evaluating JNJ-1900 (NBTXR3) in Pancreatic Cancer

On May 5, 2025 NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-stage clinical biotechnology company pioneering nanotherapeutic approaches to improve treatment outcomes for patients with cancer, reported the presentation of full results from the completed dose escalation and dose expansion phases of a Phase 1 study evaluating JNJ-1900 (NBTXR3) in patients with locally advanced or borderline resectable pancreatic cancer (Press release, Nanobiotix, MAY 5, 2025, View Source [SID1234652514]). The study, conducted by The University of Texas MD Anderson Cancer Center ("MD Anderson"), was presented by principal investigator Dr. Eugene Koay at the 2025 Annual Meeting of the European Society for Radiotherapy and Oncology (ESTRO 2025).

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Pancreatic ductal adenocarcinoma (PDAC) remains one of the most lethal malignancies, driven by aggressive tumor biology and limited responsiveness to standard therapies. For patients with locally advanced ("LAPC") or borderline resectable ("BRPC") disease, the current standard-of-care ("SOC")—induction chemotherapy followed by chemoradiation—rarely delivers curative outcomes, underscoring the need for novel treatment approaches.

"Patients with locally advanced or borderline resectable pancreatic cancer face a particularly urgent unmet need for therapeutic innovation that can provide a meaningful survival benefit with an acceptable safety profile," said Eugene Koay, MD, PhD, Associate Professor of Radiation Oncology at MD Anderson. "We are encouraged by the results from the completed cohorts and look forward to the continued evaluation of JNJ-1900 (NBTXR3) in combination with standard-of-care chemoradiation after induction chemotherapy."

PRESENTATION #E25-2265: NANORAY Pancreas: A Phase 1 Study of NBTXR3 (JNJ-1900) Activated by Radiotherapy for Locally Advanced or Borderline Resectable Pancreatic Cancer (LAPC or BRPC)
Koay EJ, Liu S, Guerrero P, Stokes E, Katz MHG, Ikoma N, Snyder RA, Tzeng CD, Overman MJ, Pant S, Wolff RA, Javle M, Holliday EB, Ludmir EB, Das P, Noticewala S, Koong AC, Tamm EP, Bhutani M

This MD Anderson-sponsored Phase 1 study evaluated the potential of radiotherapy("RT")-activated JNJ-1900 (NBTXR3) activated by radiation therapy (45 Gy in 15 fractions) to overcome inherent radioresistance in patients with LAPC or BRPC. The majority of patients in the study (20/22) were diagnosed with locally advanced, unresectable disease (LAPC). For clarity, patients with LAPC or BRPC are traditionally treated with induction chemotherapy followed by concurrent chemoradiation. The treatment regimen in the completed dose escalation and dose expansion parts of this Phase 1 study replaced concurrent chemoradiation with RT-activated JNJ-1900 (NBTXR3) after induction chemotherapy.

Key Results:

Favorable safety profile and injection feasibility were observed (n=22)
Median overall survival ("mOS"): 23 months from diagnosis [95% CI; 17 months – not reached]
For context, an MD Anderson historical review of 144 patients with LAPC treated at the same center showed a mOS of 19.2 months. Patients in the historical review received induction chemotherapy followed by RT with or without concurrent or maintenance chemotherapy (80% received RT with concurrent chemotherapy)
Median local progression-free survival ("mLPFS"): 13.3 months from completion of radiation
Two LAPC patients achieved R0 surgical resection
Exploratory Biomarker Analyses:

Of the 20 patients for whom circulating Tumor Mutational Burden (cTMB) data was available, a notable proportion (40%; 8/20) exhibited increased cTMB, and investigators observed an association between increased cTMB and improved LPFS and OS
Normalization of CA19-9, a surrogate for overall survival benefit, was observed in 59% of patients (11/22) and was associated with longer survival in the study.
For context, an MD Anderson historical review of 243 patients with LAPC treated at the same center showed normalization of CA19-9 in approximately 17% of patients treated with the standard of care who had elevated CA19-9 levels at diagnosis
Based on the safety and preliminary efficacy findings, investigators concluded that further evaluation of JNJ-1900 (NBTXR3) is warranted in a randomized study.

"Our collaboration with MD Anderson has always been driven by a shared commitment to exploring bold new approaches for patients with high unmet need," said Louis Kayitalire, MD, Chief Medical Officer at Nanobiotix. "Given the extremely poor survival rates in LAPC and BRPC, the results from this Phase 1 study give us confidence in the potential of JNJ-1900 (NBTXR3) to serve as a meaningful addition to the treatment landscape. We are particularly excited about the potential to further enhance outcomes through combination of JNJ-1900 (NBTXR3) with SOC chemoradiation in the study’s new active cohort, and we look forward to advancing this program in pancreatic cancer."

MD Anderson received FDA clearance to expand the study to include a new cohort that combines of JNJ-1900 (NBTXR3) with SOC concurrent chemoradiation after induction chemotherapy. The first patient in the new cohort has been injected, and recruitment is ongoing.

Moleculin Bolsters Annamycin Intellectual Property Portfolio with Granting of Two New U.S. Patents

On May 5, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viruses, reported the U.S. Patent and Trademark Office (USPTO) has granted two additional U.S. patents with claims covering Annamycin. U.S. patent number 12,257,261 titled, "Preparation of Preliposomal Annamycin Lyophilizate", has claims covering methods of making liposomal Annamycin and U.S. patent 12,257,262 titled "Method of Reconstituting Liposomal Annamycin", has claims covering methods of making liposomal Annamycin suspension (Press release, Moleculin, MAY 5, 2025, View Source [SID1234652511]). Both patents have a base patent term currently extending until June 2040, subject to adjustment for delays in prosecution and extension to account for time required to fulfill requirements for regulatory approval. Moleculin has additional patent applications related to Annamycin pending in the U.S., Europe and in major jurisdictions worldwide.

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Annamycin, Moleculin’s novel drug candidate, is being positioned to become the first anthracycline demonstrating a lack of cardiotoxicity to be approved and is currently being developed for the treatment of acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases (STS lung mets). Additional preclinical studies performed at a world-renowned cancer center indicate Annamycin may be a potential treatment for many other types of cancers. The new chemical entity uses a unique lipid-based delivery technology and has shown the potential to be used in a wide range of cancers.

Wally Klemp, Chairman and CEO of Moleculin, said, "We remain focused on expanding our intellectual property portfolio for Annamycin. Following the issuance of two U.S. patents in 2024, these new patents enhance the exclusivity of Annamycin, bringing to four the total number of U.S. patents related to Annamycin, in addition to the European patents granted. Based on the data seen to date and feedback we continue to receive from clinicians and patients, we are dedicated to advancing this important and much needed treatment option forward. We continue to make solid progress in our ongoing pivotal, adaptive Phase 3 MIRACLE trial and remain on track to report initial data in the second half of 2025."

Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory AML, in addition to Orphan Drug Designation for the treatment of STS lung mets. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA).

TG Therapeutics Reports First Quarter 2025 Financial Results and Raises BRIUMVI Full Year Revenue Guidance

On May 5, 2025 TG Therapeutics, Inc. (NASDAQ: TGTX) (the Company or TG Therapeutics) reported its financial results for the first quarter of 2025, along with recent company developments and provided an update on 2025 revenue guidance (Press release, TG Therapeutics, MAY 5, 2025, View Source [SID1234652510]).

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Michael S. Weiss, the Company’s Chairman and Chief Executive Officer, stated, "2025 is off to a strong start, and I’m incredibly proud of the progress made thus far. Our performance in the first quarter, including BRIUMVI’s $119.7 million in U.S. net sales, demonstrates the growing confidence in our treatment and the increasing adoption by healthcare providers. This momentum, combined with the positive feedback we’re hearing from both patients and clinicians, reinforces our belief that we can achieve our long-term goal of BRIUMVI becoming the number one prescribed anti-CD20 treatment based on dynamic market share." Mr. Weiss continued, "In addition to our ongoing BRIUMVI commercialization efforts, we are continuing to invest in innovation, including simplifying the BRIUMVI treatment regimen, advancing subcutaneous BRIUMVI, and developing azer-cel for progressive MS. All with a focus on developing treatment options designed to improve the lives of individuals living with MS."

Recent Highlights & Developments

BRIUMVI (ublituximab-xiiy) Commercialization

BRIUMVI U.S. net product revenue of $119.7 million for the first quarter of 2025, representing approximately 137% growth over the same period last year

Expansion of commercialization outside of the U.S. with our partner, Neuraxpharm, with BRIUMVI now commercially available in additional countries in the European Union, United Kingdom and Switzerland

BRIUMVI Data

Presented five-year data from the open-label extension study of the ULTIMATE I & II Phase 3 clinical trials evaluating BRIUMVI in adult patients with relapsing forms of multiple sclerosis (RMS) which demonstrated that 92% of patients were free from disability progression after five years of treatment, an annualized relapse rate of 0.02 during year 5 of treatment (equivalent to one relapse occurring every fifty years of patient treatment), and an overall safety profile that remained consistent, with no new safety signals emerging over 5 years of continuous treatment and no observed cases of progressive multifocal leukoencephalopathy (PML) to date.

Presented data from the ENHANCE Phase 3b clinical trial evaluating alternative dosing strategies for BRIUMVI in patients with RMS which demonstrated that:


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A single 600 mg BRIUMVI infusion on Day 1 was well tolerated in individuals with RMS regardless of prior treatment or B-cell depletion status


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Rapid 30-minute BRIUMVI infusions were well tolerated in patients with RMS

Published two articles in medical journals:


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"Switching to Ublituximab from Prior anti-CD20 Monoclonal Antibody Therapy: A Case Report Series", published in Frontiers in Immunology, demonstrating a retrospective case series of seven individuals with multiple sclerosis (MS) treated in private practice or at an MS clinic who switched to ublituximab from a different anti-CD20 monoclonal antibody therapy due to efficacy or tolerability concerns, published in Frontiers in Immunology


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"The Evolution of Anti-CD20 Treatment in Multiple Sclerosis", published in CNS DRUGS, demonstrating the differentiating characteristics within the anti-CD20 monoclonal antibody class used to treat MS, published in CNS DRUGS.

Pipeline Status

Phase 1 clinical trial evaluating subcutaneous BRIUMVI in patients with RMS remains ongoing

Phase 1 clinical trial evaluating BRIUMVI in patients with Myasthenia Gravis (MG) remains ongoing

Phase 1 clinical trial evaluating azercabtagene zapreleucel (azer-cel) in patients with primary progressive multiple sclerosis is now open for enrollment

2025 Financial Guidance

Raises BRIUMVI U.S. net product revenue target to $560 million for the full year 2025 (prior guidance of $525 million for full year 2025)

Raises total global revenue target to $575 million for the full year 2025 (prior guidance of $540 million for full year 2025)

Second quarter 2025 BRIUMVI U.S. net product revenue target of $135 million Full year 2025 target operating expense of approximately $300 million (excluding non-cash compensation and cost of goods sold)

2025 Development Pipeline Anticipated Milestones

Commence pivotal program of subcutaneous BRIUMVI

Commence a pivotal program based on the improved dosing regimens evaluated in the ENHANCE trial, with the goal of enhancing the patient experience on intravenous BRIUMVI

Continue enrollment of participants into the ongoing clinical trial evaluating BRIUMVI in autoimmune diseases outside of MS

Continue enrollment of participants into the Phase 1 clinical trial evaluating azer-cel for the treatment of autoimmune diseases, beginning with progressive forms of MS

Present updated data at major medical conferences throughout the year

Financial Results for First Quarter 2025

Product Revenue, Net: Product revenue, net was approximately $119.7 million for the three months ended March 31, 2025, compared to $50.5 million for the three months ended March 31, 2024. Product revenue, net for both the three months ended March 31, 2025 and March 31, 2024, consisted of net product sales of BRIUMVI in the United States.

License, Milestone, Royalty and Other Revenue: License, milestone, royalty and other revenue was approximately $1.2 million for the three months ended March 31, 2025, compared to $13.0 million for the three months ended March 31, 2024. License, milestone, royalty and other revenue for the three months ended March 31, 2024 is predominantly comprised of a $12.5 milestone payment under the commercialization agreement with Neuraxpharm for the first key market commercial launch of BRIUMVI in the European Union.

R&D Expenses: Total research and development (R&D) expense was $46.4 million for the three months ended March 31, 2025, compared to $32.7 million for the three months ended March 31, 2024. The increase in R&D expense for the three months ended March 31, 2025, was primarily attributable to manufacturing expenses incurred in connection with our ublituximab subcutaneous development work during the period.

SG&A Expenses: Total selling, general and administrative (SG&A) expense was $50.3 million for the three months ended March 31, 2025, compared to $34.6 million for the three months ended March 31, 2024. The increase was primarily due to other selling, general and administrative costs, including marketing, personnel and consultants, associated with the commercialization of BRIUMVI for the three months ended March 31, 2025.

Net Income (Loss): Net income was $5.1 million for the three months ended March 31, 2025, compared to a net loss of $10.7 million for the three months ended March 31, 2024.

Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $276.2 million as of March 31, 2025. We anticipate that our cash, cash equivalents and investment securities as of March 31, 2025, combined with the projected revenues from BRIUMVI, will be sufficient to fund our business based on our current operating plan.CONFERENCE CALL INFORMATION
The Company will host a conference call today, May 5, 2025, at 8:30 AM ET, to discuss the Company’s financial results from first quarter 2025.

To participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for a period of 30 days after the call.

ABOUT BRIUMVI (ublituximab-xiiy) 150 mg/6 mL Injection for IV
BRIUMVI is a novel monoclonal antibody that targets a unique epitope on CD20-expressing B-cells. Targeting CD20 using monoclonal antibodies has proven to be an important therapeutic approach for the management of autoimmune disorders, such as RMS. BRIUMVI is uniquely designed to lack certain sugar molecules normally expressed on the antibody. Removal of these sugar molecules, a process called glycoengineering, allows for efficient B-cell depletion at low doses.

BRIUMVI is indicated in the U.S. for the treatment of adults with RMS, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease and in the EU and UK for the treatment of adult patients with RMS with active disease defined by clinical or imaging features.

A list of authorized specialty distributors can be found at www.briumvi.com.

IMPORTANT SAFETY INFORMATION

Contraindications: BRIUMVI is contraindicated in patients with:

Active Hepatitis B Virus infection

A history of life-threatening infusion reaction to BRIUMVI

WARNINGS AND PRECAUTIONS

Infusion Reactions: BRIUMVI can cause infusion reactions, which can include pyrexia, chills, headache, influenza-like illness, tachycardia, nausea, throat irritation, erythema, and an anaphylactic reaction. In MS clinical trials, the incidence of infusion reactions in BRIUMVI-treated patients who received infusion reaction-limiting premedication prior to each infusion was 48%, with the highest incidence within 24 hours of the first infusion. 0.6% of BRIUMVI-treated patients experienced infusion reactions that were serious, some requiring hospitalization.

Observe treated patients for infusion reactions during the infusion and for at least one hour after the completion of the first two infusions unless infusion reaction and/or hypersensitivity has been observed in association with the current or any prior infusion. Inform patients that infusion reactions can occur up to 24 hours after the infusion. Administer the recommended pre-medication to reduce the frequency and severity of infusion reactions. If life-threatening, stop the infusion immediately, permanently discontinue BRIUMVI, and administer appropriate supportive treatment. Less severe infusion reactions may involve temporarily stopping the infusion, reducing the infusion rate, and/or administering symptomatic treatment.

Infections: Serious, life-threatening or fatal, bacterial and viral infections have been reported in BRIUMVI-treated patients. In MS clinical trials, the overall rate of infections in BRIUMVI-treated patients was 56% compared to 54% in teriflunomide-treated patients. The rate of serious infections was 5% compared to 3% respectively. There were 3 infection-related deaths in BRIUMVI-treated patients. The most common infections in BRIUMVI-treated patients included upper respiratory tract infection (45%) and urinary tract infection (10%). Delay BRIUMVI administration in patients with an active infection until the infection is resolved.

Consider the potential for increased immunosuppressive effects when initiating BRIUMVI after immunosuppressive therapy or initiating an immunosuppressive therapy after BRIUMVI.

Hepatitis B Virus (HBV) Reactivation: HBV reactivation occurred in an MS patient treated with BRIUMVI in clinical trials. Fulminant hepatitis, hepatic failure, and death caused by HBV reactivation have occurred in patients treated with anti-CD20 antibodies. Perform HBV screening in all patients before initiation of treatment with BRIUMVI. Do not start treatment with BRIUMVI in patients with active HBV confirmed by positive results for HB surface antigen (HBsAg) and anti-HB tests. For patients who are negative for HBsAg and positive for HB core antibody [HBcAb+] or are carriers of HBV [HBsAg+], consult a liver disease expert before starting and during treatment.

Progressive Multifocal Leukoencephalopathy (PML): Although no cases of PML have occurred in BRIUMVI-treated MS patients, JC virus infection resulting in PML has been observed in patients treated with other anti-CD20 antibodies and other MS therapies.

If PML is suspected, withhold BRIUMVI and perform an appropriate diagnostic evaluation. Typical symptoms associated with PML are diverse, progress over days to weeks, and include progressive weakness on one side of the body or clumsiness of limbs, disturbance of vision, and changes in thinking, memory, and orientation leading to confusion and personality changes.

MRI findings may be apparent before clinical signs or symptoms; monitoring for signs consistent with PML may be useful. Further investigate suspicious findings to allow for an early diagnosis of PML, if present. Following discontinuation of another MS medication associated with PML, lower PML-related mortality and morbidity have been reported in patients who were initially asymptomatic at diagnosis compared to patients who had characteristic clinical signs and symptoms at diagnosis.

If PML is confirmed, treatment with BRIUMVI should be discontinued.

Vaccinations: Administer all immunizations according to immunization guidelines: for live or live-attenuated vaccines, at least 4 weeks and, whenever possible, at least 2 weeks prior to initiation of BRIUMVI for non-live vaccines. BRIUMVI may interfere with the effectiveness of non-live vaccines. The safety of immunization with live or live-attenuated vaccines during or following administration of BRIUMVI has not been studied. Vaccination with live virus vaccines is not recommended during treatment and until B-cell repletion.

Vaccination of Infants Born to Mothers Treated with BRIUMVI During Pregnancy: In infants of mothers exposed to BRIUMVI during pregnancy, assess B-cell counts prior to administration of live or live-attenuated vaccines as measured by CD19+ B-cells. Depletion of B-cells in these infants may increase the risks from live or live-attenuated vaccines. Inactivated or non-live vaccines may be administered prior to B-cell recovery. Assessment of vaccine immune responses, including consultation with a qualified specialist, should be considered to determine whether a protective immune response was mounted.

Fetal Risk: Based on data from animal studies, BRIUMVI may cause fetal harm when administered to a pregnant woman. Transient peripheral B-cell depletion and lymphocytopenia have been reported in infants born to mothers exposed to other anti-CD20 B-cell depleting antibodies during pregnancy. A pregnancy test is recommended in females of reproductive potential prior to each infusion. Advise females of reproductive potential to use effective contraception during BRIUMVI treatment and for 6 months after the last dose.

Reduction in Immunoglobulins: As expected with any B-cell depleting therapy, decreased immunoglobulin levels were observed. Decrease in immunoglobulin M (IgM) was reported in 0.6% of BRIUMVI-treated patients compared to none of the patients treated with teriflunomide in RMS clinical trials. Monitor the levels of quantitative serum immunoglobulins during treatment, especially in patients with opportunistic or recurrent infections, and after discontinuation of therapy, until B-cell repletion. Consider discontinuing BRIUMVI therapy if a patient with low immunoglobulins develops a serious opportunistic infection or recurrent infections, or if prolonged hypogammaglobulinemia requires treatment with intravenous immunoglobulins.

Most Common Adverse Reactions: The most common adverse reactions in RMS trials (incidence of at least 10%) were infusion reactions and upper respiratory tract infections.

Physicians, pharmacists, or other healthcare professionals with questions about BRIUMVI should visit www.briumvi.com.

The full Summary of Product Characteristics approved in the European Union (EU) for BRIUMVI can be found here Briumvi | European Medicines Agency (europa.eu).

ABOUT BRIUMVI PATIENT SUPPORT in the U.S.
BRIUMVI Patient Support is a flexible program designed by TG Therapeutics to support U.S. patients through their treatment journey in a way that works best for them. More information about the BRIUMVI Patient Support program can be accessed at www.briumvipatientsupport.com.

ABOUT MULTIPLE SCLEROSIS
Relapsing multiple sclerosis (RMS) is a chronic demyelinating disease of the central nervous system (CNS) and includes people with relapsing-remitting multiple sclerosis (RRMS) and people with secondary progressive multiple sclerosis (SPMS) who continue to experience relapses. RRMS is the most common form of multiple sclerosis (MS) and is characterized by episodes of new or worsening signs or symptoms (relapses) followed by periods of recovery. It is estimated that nearly 1 million people are living with MS in the United States and approximately 85% are initially diagnosed with RRMS.1,2 The majority of people who are diagnosed with RRMS will eventually transition to SPMS, in which they experience steadily worsening disability over time. Worldwide, more than 2.3 million people have a diagnosis of MS.

ImmunityBio Requests an Urgent Meeting With FDA to Address the Change in the Agency’s Unambiguous Guidance on Jan 2025 to Submit a sBLA for NMIBC BCG Unresponsive Papillary Disease, Following an Inconsistent Refusal to File Letter on May 2, 2025

On May 5, 2025 ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, reported that the Company received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) for the supplemental biologics license application (sBLA) for use of ANKTIVA plus Bacillus Calmette-Guerin (BCG) in BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) for the indication of papillary disease (Press release, ImmunityBio, MAY 5, 2025, View Source [SID1234652509]). This RTF letter was received despite reaching unanimous guidance and encouragement at the in-person January 2025 meeting from the leadership of the Agency, including from CBER, CDER and OCE to submit this sBLA. At this meeting all key decision makers were specifically asked and unanimously confirmed that ImmunityBio should submit the sBLA as soon as possible based on the data in the single-arm trial. Relying on this unanimous guidance the Company submitted the sBLA in March 2025. The Company has already requested an urgent meeting to resolve the inconsistencies between the directives provided at the January Meeting and receipt of the RTF letter.

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ANKTIVA was approved by the FDA in 2024 with BCG for the treatment of BCG unresponsive NMIBC with Papillary tumors with CIS (Cohort A). In the same clinical trial (QUILT-3.032) long term results of patients with Papillary tumors without CIS (Cohort B), was submitted as a sBLA. The RTF letter referenced herein does not impact this prior approval of CIS +/- Papillary in BCG unresponsive patients.

The Agency leaders present at the January meeting unambiguously invited the Company to expeditiously submit an sBLA for the NMIBC papillary indication based on the strength of the clinical response and long-term duration of follow-up data of the Papillary without CIS cohort of the QUILT 3.032 study. Data was also presented of the long term follow-up Phase 1 results which demonstrated Complete Response and Disease Free Survival in both CIS and Papillary disease, with patients in on-going cystectomy free state at 10 years. In addition disease-specific overall survival rate of 99% at 12 months and 96% at 36 months, in the BCG unresponsive patients with Papillary disease without CIS was discussed. To our knowledge, this is the most durable data to date in the Papillary setting for bladder preservation.

In March 2025, the Company completed its submission to the FDA of the sBLA for the use of ANKTIVA plus BCG in BCG-unresponsive NMIBC in the papillary indication. On May 2, 2025, and notwithstanding the FDA’s invitation to submit the sBLA articulated by its leadership at the January Meeting, the FDA delivered an RTF letter. The Company together with its consultants who attended the January 2025 meeting were shocked by this inconsistent response and have requested an urgent meeting with the Agency to resolve this issue.

"Our commitment to NMIBC patients in the papillary indication and our belief in ANKTIVA’s potential based on the strength of the clinical response and long duration of 5-year follow-up remains unchanged, despite our receipt of a refusal to file letter regarding our supplemental BLA," said Dr. Patrick Soon-Shiong, the Company’s Founder, Executive Chairman and Global Chief Scientific and Medical Officer. "We are fully determined to work with the FDA as quickly as possible, including having already requested a Type A meeting, to explore the best path forward. We would also welcome an FDA Advisory Committee meeting as part of the regulatory process going forward. We presented our data at the recent 2025 American Urological Association (AUA) meeting and ANKTIVA+BCG was considered best in class and best in disease by the thought leaders in attendance, when compared to all the therapies currently approved or in development. Patients with BCG Unresponsive Papillary disease, face a life changing and life-threatening prospect of a total radical cystectomy, as well as the danger of the disease progressing from non-muscle invasive to muscle invasion with consequent progression and mortality. With the data presented of 99% disease specific survival at 12 months and over 82% patients not requiring bladder removal, it is essential that these patients be provided this treatment option, especially with the safety profile of ANKTIVA+BCG and the already approved indication of Papillary disease with CIS. The Company presented these data to the Agency in person in January and the Agency leaders present unanimously encouraged the company to expeditiously submit a supplemental BLA for this indication."

Dr. Soon-Shiong further stated: "The Agency must explain the confounding inconsistency of approving ANKTIVA+BCG for patients with Papillary with CIS disease, while refusing to file the sBLA for patients with Papillary without CIS disease—even though both groups were part of the same trial, in the same high-risk population of BCG-unresponsive non-muscle invasive bladder cancer. In both cases, patients received the same surgical procedure for the Papillary component, the same therapy at the same dose, with the same excellent tolerability and meaningful clinical benefit: over 82% bladder-sparing and over 96% disease-specific survival at 36 months, as shown in Figure 1. On behalf of patients facing a potential loss of a vital organ and high risk of progression of disease, I urge the Agency to reconsider and act now."

"I actively participated in the January 2025 meeting at which the leadership of the Agency present at that meeting unanimously supported and encouraged ImmunityBio to submit results from the single arm trial, QUILT 3.032 as a supplemental BLA because of the high-risk of progression and metastasis these patients with BCG unresponsiveness face. The consensus was reached by all present, including me, because of the lack of satisfactory alternatives in this desperately ill population at high-risk of losing their bladder – a life threatening and life changing procedure. Thus, I was startled to learn the ImmunityBio had received a ‘Refuse to File’ letter which is in my opinion rife with regulatory inaccuracies. I recommend that the company seek a rapid meeting with the Agency to resolve this issue and minimize the delay and threat to well-being of patients who could benefit significantly from avoiding a cystectomy," said Dr. Rachel Sherman, former FDA Principal Deputy Commissioner, who pioneered single arm trials at the height of the HIV epidemic, and was responsible for developing the expedited pathways at the Agency to address life threatening and serious diseases on behalf of the American public during her 30 year career at the agency. "Furthermore, it is incomprehensible to me that the FDA refuses to file a supplemental BLA, stating the study is not sufficient to support a regulatory review, when it has already approved a product based on that very same study in essentially the same indication and population. As stated above, these patients are suffering from a disease with a high risk of morbidity and mortality in the very short term – no delay should be tolerated," added Dr. Sherman.

About the QUILT-3.032 Study

QUILT-3.032 (NCT03022825) is a Phase II/III, open-label, single-arm, multicenter study of intravesical BCG plus ANKTIVA or ANKTIVA only in patients with BCG unresponsive high grade non-muscle invasive bladder cancer (NMIBC). All participants receive BCG plus ANKTIVA (Cohorts A and B) or ANKTIVA only (Cohort C) via a urinary catheter in the bladder for 6 consecutive weeks (initial induction treatment period). After the first disease assessment, eligible patients receive either a 3-week maintenance course or a 6-week re-induction course (second treatment period) at Month 3. Eligible patients will continue to receive maintenance treatment in the third treatment period at Months 6, 9, 12, and 18. Eligible patients have the option to receive maintenance treatment in the fourth treatment period at Months 24, 30, and 36. The study duration is 60 months.

Cohort A (N=100) includes patients with histologically confirmed BCG-unresponsive NMIBC CIS with or without papillary disease. The primary endpoint is biopsy-confirmed complete response (CR) rate at any time. Secondary endpoints include duration of CR, progression-free survival (PFS), time to cystectomy, safety and overall survival. FDA Approved this indication in 2024 in which eligibility included patients with Papillary disease.

Cohort B (N=80) enrolled participants with histologically confirmed BCG-unresponsive high-grade Papillary disease without CIS. The primary endpoint is a response of a disease-free rate at 12 months. Secondary endpoints include disease-free survival DFS and disease-specific survival, PFS, time to cystectomy, safety and overall survival.

About ANKTIVA

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response.

ANKTIVA is a first-in-class IL-15 receptor agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and drives the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones. The proliferation of the trifecta of these immune killing cells and the activation of trained immune memory results in immunogenic cell death, inducing a state of equilibrium with durable complete responses. ANKTIVA has improved pharmacokinetic properties, longer persistence in lymphoid tissues, and enhanced anti-tumor activity compared to native, non-complexed IL-15 in-vivo.

ANKTIVA was approved by the FDA in 2024 with BCG for the treatment of adult patients with BCG-unresponsive non-muscle invasive bladder cancer with CIS with or without papillary tumors. For more information, visit ImmunityBio.com (Founder’s Vision) and Anktiva.com.

Immuneering Reports First Quarter 2025 Financial Results and Provides Business Updates

On May 5, 2025 Immuneering Corporation (Nasdaq: IMRX), a clinical-stage oncology company seeking to develop and commercialize more effective and better tolerated therapies for cancer patients, reported financial results for the first quarter ended March 31, 2025, and provided several business updates (Press release, Immuneering, MAY 5, 2025, View Source [SID1234652508]).

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"In Q1, we showed that IMM-1-104 can drive exceptional efficacy for patients with pancreatic cancer, including a third-line monotherapy patient with over 13 months progression-free survival, a second-line monotherapy patient with a confirmed partial response, and a first-line combination therapy patient with a confirmed complete response. These outcomes are particularly striking given the excellent tolerability observed with IMM-1-104. In January, we reported encouraging overall response rates and disease control rates for IMM-1-104 in combination with chemotherapy in first-line pancreatic cancer. We are excited to share survival data in a larger group of patients in the coming weeks," said Ben Zeskind, Ph.D., Co-founder and Chief Executive Officer of Immuneering.

"Our corporate progress in Q1 has matched the pace of our clinical progress. We announced a clinical supply agreement with Regeneron, expanded our cash runway into 2026, and hired an outstanding Chief Medical Officer as we plan for the initiation of a Phase 3 trial in first-line pancreatic cancer patients," Zeskind concluded.

Corporate Highlights

Pancreatic Cancer Patient Passes 13-month Mark on IMM-1-104 Monotherapy: In April, Immuneering provided an update on a Phase 1 pancreatic cancer patient in the third-line setting who has been receiving IMM-1-104 monotherapy for more than 13 months and is still on treatment. The patient – who previously experienced disease progression on first-line FOLFIRINOX and second-line Gem/Cis/nab-Pac – has been on IMM-1-104 monotherapy at 240 mg once daily and maintained stable disease including a RECIST SLD change of -28% and a 91% reduction in peak CA 19-9 levels. Treatment continued to be well tolerated by the patient, with approximately 16% weight gain.
Dr. Igor Matushansky Named as Chief Medical Officer: In March, Immuneering announced that Igor Matushansky, MD, PhD, an industry veteran with extensive global oncology drug development expertise and experience in clinical treatment of cancer patients, has been appointed the Company’s Chief Medical Officer. In this role, Dr. Matushansky is directing Immuneering’s clinical activities and is providing medical and operational leadership for the company’s development programs, including the ongoing Phase 2a trial of IMM-1-104 in pancreatic cancer, lung cancer, and melanoma, and plans to initiate a pivotal Phase 3 clinical trial in pancreatic cancer.
Clinical Trial Supply Agreement Announced with Regeneron for Libtayo (cemiplimab): In February, Immuneering announced a clinical trial supply agreement with Regeneron Pharmaceuticals for its anti-PD-1 therapy, Libtayo. The agreement supports the evaluation of IMM-1-104, in combination with Libtayo in patients with unresectable or metastatic RAS-mutant non-small cell lung cancer (NSCLC).
Provided a Positive Data Update from Three Pancreatic Cancer Arms of Ongoing Phase 2a Trial of IMM-1-104: In January, Immuneering announced positive data updates from three pancreatic cancer arms of its ongoing Phase 2a trial of lead program IMM-1-104 and confirmed plans to expand the Phase 2a trial to include additional combination arms.
Near-Term Milestone Expectations

IMM-1-104

Initial progression-free survival data from the IMM-1-104 Phase 2a trial expected in the second quarter of 2025.
Additional IMM-1-104 combination arms in planning.
Initiation of Phase 3 trial of IMM-1-104+mGnP in first-line pancreatic cancer planned for 2026.
First Quarter 2025 Financial Highlights

Cash Position: Cash and cash equivalents as of March 31, 2025, were $35.9 million, compared with $36.1 million as of December 31, 2024.
Research and Development (R&D) Expenses: R&D expenses for the first quarter of 2025 were $11.5 million compared with $11.2 million for the first quarter of 2024. The increase in R&D expenses was primarily attributable to higher clinical costs related to the Company’s lead program.
General and Administrative (G&A) Expenses: G&A expenses for the first quarter of 2025 were $4.0 million compared with $4.1 million for the first quarter of 2024. The decrease in G&A was primarily attributable to a decrease in the Company’s employee-related costs in connection with the general and administrative functions supporting the business.
Net Loss: Net loss attributable to common stockholders was $15.0 million, or $0.42 per share, for the first quarter ended March 31, 2025, compared to $14.3 million, or $0.49 per share, for the first quarter ended March 31, 2024.
2025 Financial Guidance

Based on cash and cash equivalents, as of March 31, 2025, and current operating plans, the Company expects its cash runway to be sufficient to fund operations into 2026.