Bridget Hoyt

The treatment offers a non-surgical option for patients who may not be candidates for repeat procedures.

The U.S. Food and Drug Administration has approved UGN-102 (mitomycin intravesical solution) for the treatment of adult patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).

The drug’s efficacy was based on data from the phase 3 ENVISION trial, which evaluated 240 enrolled patients with low-grade NMIBC recurring following previous transurethral resection, or surgical removal, of bladder tumor (TURBT).

Out of 223 evaluable patients, 78% had a 

, which, in this case, indicates that patients had no cancer left in the bladder according to an internal visual analysis called cystoscopy and a microscope urine evaluation called urine cytology, along with a biopsy if needed. Among those who responded, 79% remained cancer-free for at least one year.

To be part of the study, patients needed to meet at least one of the following: have multiple tumors, have a single tumor larger than 3 cm or have a cancer recurrence within one year.

The most common side effects that patients experienced were pain or burning with urination (dysuria), urinary tract infections, blood in the urine, fatigue and changes in lab values like kidney function or blood counts.

Serious side effects occurred in 12% of patients. Less than 1% of participants experienced urinary retention or urethral narrowing. One patient died from heart failure during the study, although it is not currently public information whether the death was caused by UGN-102.

Previous data from the phase 3 ATLAS trial showed that UGN-102 may also benefit patients regardless of whether they had TURBT. In that study, newly diagnosed patients who received UGN-102 had a 15-month disease-free survival rate of about 77%. Patients with recurrent disease had a 63% disease-free rate over the same period.

Complete response rates remained high at one year in both groups—nearly 88% in newly diagnosed patients and about 69% in those with recurrent cancer.

The most common treatment-related side effects in ATLAS included dysuria, frequent urination, urgent urination, and nighttime urination.

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The FDA has granted approval to UGN-102 for use in patients with low-grade intermediate-risk non-muscle-invasive bladder cancer.

FDA Approves UGN-102 For Some With Bladder Cancer

The FDA grants fast track status to PHST001 for advanced platinum-resistant and platinum-sensitive ovarian cancer.

The U.S. Food and Drug Administration (FDA) has granted fast track designation to PHST001 for the single-agent treatment of patients with advanced platinum-resistant ovarian cancer or in combination with chemotherapy in platinum-sensitive ovarian cancer, according to a news release from Pheast Therapeutics.

PHST001 is an experimental antibody therapy that may help the immune system detect and attack solid tumors by targeting the CD24 protein.

“Receiving fast track designation from the FDA reinforces the [possibility] of CD24 as a next-generation immuno-oncology target and highlights the potential of PHST001 to address a critical unmet need in the treatment of ovarian cancer,” Dr. Raphaël Rousseau, chief medical officer of Pheast Therapeutics, said in the news release. “We are committed to advancing PHST001 through the clinic and accelerating its development for cancer patients in urgent need of more effective treatments.”

The FDA’s fast track program helps speed the development and review of new therapies for serious conditions with unmet needs. Companies granted fast track status can have more frequent FDA interactions during clinical trials and may qualify for faster approval or priority review if they meet certain criteria.

The multicenter, open-label phase 1 study of PHST001 is enrolling up to 80 patients with advanced relapsed or refractory solid tumors. It aims to evaluate the safety and tolerability of PHST001 and determine the recommended phase 2 dose, with secondary goals to assess pharmacokinetics and early anti-tumor activity.

In Texas, two centers are actively recruiting: MD Anderson Cancer Center in Houston and South Texas Accelerated Research Therapeutics (START) in San Antonio. Additional locations in California, Connecticut, Illinois, Massachusetts, Michigan, North Carolina and Tennessee are to be included in the future of the study.

CD24 is a protein found on the surface of many cancer cells that helps tumors avoid being destroyed by the immune system. It does this by binding to Siglec-10, a receptor on immune cells called macrophages, which normally would clear away harmful cells. This interaction prevents macrophages from attacking the cancer.

CD24 was found to be an important “checkpoint” in the immune system by Dr. Amira Barkal, founder of Pheast Therapeutics, and her colleagues in Dr. Irving Weissman’s lab at Stanford University.

PHST001 is an investigational antibody designed to block the CD24 checkpoint. By inhibiting CD24, PHST001 helps restore macrophages’ ability to engulf and destroy cancer cells, sparking a stronger immune response. CD24 is found at high levels in several cancers, including ovarian cancer and triple-negative breast cancer, where its presence is linked to worse outcomes. PHST001 is engineered to be a leading treatment in its class, aiming to overcome immune suppression within tumors.

What is Platinum Sensitive Ovarian Cancer

Cancer that responds to treatment with platinum-based drugs, such as cisplatin and carboplatin, is called “platinum sensitive,” according to the National Cancer Institute. Some cancers that return after treatment may also be platinum sensitive — for example, ovarian cancer that comes back 6 or more months after platinum therapy. Platinum-resistant ovarian cancer, however, shows a resistance to platinum-based therapies and may return within 6 months. Knowing if cancer is platinum sensitive helps guide further treatment.

Ovarian cancer develops in the tissues of the ovaries, female reproductive glands that produce eggs. Most ovarian cancers are ovarian epithelial cancers, which start in the surface cells of the ovary, or malignant germ cell tumors, which begin in egg cells. Fallopian tube cancer and primary peritoneal cancer are similar to ovarian epithelial cancer and are treated the same way.

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The FDA has granted fast track designation to PHST001, an investigational antibody therapy targeting CD24, for treating advanced platinum-resistant ovarian cancer and in combination with chemotherapy for platinum-sensitive ovarian cancer. PHST001 aims to enhance immune response by inhibiting CD24, a protein that helps tumors evade immune destruction. A phase 1 study is underway to assess its safety and efficacy. CD24 is a significant immune checkpoint, and PHST001 could address unmet needs in ovarian cancer treatment. Fast track status facilitates accelerated development and review of promising therapies.

The FDA grants fast track designation to PHST001, an antibody targeting CD24, for advanced platinum-resistant and platinum-sensitive ovarian cancer.

FDA Grants Fast Track Status to PHST001 for Ovarian Cancer Treatment 

The FDA granted orphan drug designation to MVdeltaC, a measles virus-based immunotherapy, for pleural mesothelioma, supporting its future development.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to MVdeltaC for the treatment of patients with pleural mesothelioma, a rare and aggressive cancer with high unmet medical need and limited therapeutic options, according to a news release from Oncovita, the developer of the drug.

“Receiving orphan drug designation from the FDA for MVdeltaC marks a major milestone for Oncovita and validates our approach of harnessing the potential of measles vaccine viruses to treat solid tumors, particularly rare and devastating cancers such as pleural mesothelioma,” said Stéphane Altaba, CEO of Oncovita, in the news release. “This regulatory support strengthens our strategy to advance innovative immunotherapies as we prepare to enter clinical development with MVdeltaC by 2026.”

The FDA grants orphan drug designation to encourage therapies for rare diseases that affect fewer than 200,000 people annually in the U.S. This designation grants developers perks including waived fees for FDA applications, as many as seven years of market exclusivity following approval, and tax credits toward qualifying clinical trials.

“With this designation, Oncovita is now well-positioned to enter the U.S. market with its modified attenuated measles virus for the treatment of pleural mesothelioma. This recognition highlights the promise of this novel approach against one of the most aggressive cancers in medicine,” Dr. Stéphane Champiat, head of medical affairs at Oncovita, added in the news release.

Champiat is also an associate professor for the Department of Investigational Cancer Therapeuticsat The University of Texas MD Anderson Cancer Center, Houston, Texas

MVdeltaC is an experimental immunotherapy that uses a weakened, modified measles virus designed to target and destroy tumor cells while also activating the immune system. With this treatment, Oncovita aims to improve outcomes in advanced solid tumors such as pleural mesothelioma by combining direct cancer cell destruction with immune stimulation.

Pleural mesothelioma is a rare, aggressive cancer of the lung lining, most often caused by long-term asbestos exposure. About 3,000 people are diagnosed annually in the U.S. Prognosis is generally poor, especially when the disease recurs or stops responding to treatment, and options remain limited.

MVdeltaC showed strong immune-stimulating and tumor-killing activity in preclinical studies, according to a previous news release from Oncovita published in 2023. Based on these findings, a first-in-human trial is planned for patients with solid tumors, with particular interest in those whose disease no longer responds to checkpoint inhibitors.

Researchers created a modified version of the measles virus called MVdeltaC by removing one of its genes linked to illness. They tested MVdeltaC in lab studies on more than 40 types of human cancer cells, including mesothelioma, lung, bladder, ovarian, cervical and liver cancers. The vaccine was also tested in several animal models, including mice implanted with human tumors and mice with intact immune systems. Scientists studied how the virus works using immune cells taken from people.

In preclinical studies, MVdeltaC showed strong anti-tumor activity both in vitro and in vivo. It was two to three times more potent than standard measles virus in killing human tumor cell lines and was active in over 70% of those tested. In mice with human mesothelioma, a single low dose significantly reduced tumor size, and in patient-derived xenograft models, weekly MVdeltaC dosing controlled tumor growth in mesothelioma and bladder cancer.

In immunocompetent mice, three intratumoral injections led to tumor regression or complete disappearance in 70% of animals, with long-term immune protection shown by resistance to tumor re-challenge. Ex vivo experiments confirmed MVdeltaC triggered a strong immune response, including activation of dendritic cells, phagocytosis of tumor cells and presentation of tumor antigens to T cells. The therapy also produced high levels of defective interfering genomes, which may drive its immune-stimulating effects through the RIG-I pathway.

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The FDA has granted orphan drug designation to MVdeltaC, an experimental immunotherapy for pleural mesothelioma, developed by Oncovita. This designation supports therapies for rare diseases, offering benefits like market exclusivity and tax credits. MVdeltaC utilizes a modified measles virus to target tumor cells and stimulate the immune system, showing promising preclinical results in various cancer models. The drug demonstrated significant tumor-killing activity and immune response activation, with plans for a first-in-human trial targeting solid tumors, particularly those unresponsive to checkpoint inhibitors, by 2026.

MVdeltaC Earns Orphan Drug Status for Pleural Mesothelioma

Iopofosine-131 received FDA breakthrough status for relapsed or refractory Waldenstrom macroglobulinemia, showing strong response rates in early trials.

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to iopofosine-131, a potential first-in-class cancer-targeting agent, for the treatment of relapsed or refractory Waldenstrom macroglobulinemia (r/r WM), according to a news release from Cellectar Biosciences.

“The FDA’s breakthrough therapy designation underscores the potential of iopofosine I-131 as it may offer substantial improvement on at least one clinically significant endpoint over available therapies to address the substantial unmet medical need in this life-threatening cancer,” said James Caruso, president and chief executive officer of Cellectar, in the news release. “With robust clinical data, a favorable safety profile, expedited review designations in the United States and Europe and a compelling commercial market potential, we believe iopofosine I-131 represents an attractive candidate for potential collaborations or partners seeking impactful innovation and accelerated development pathways.”

Results from the phase 2 CLOVER WaM study showed an overall response rate of 83.6% and a major response rate (MRR) of 58.2%, exceeding the study’s primary endpoint of a 20% MRR. These data were shared at a podium presentation at the 66th American Society of Hematology Annual Meeting in December 2024 by Dr. Sikander Ailawadhi, professor of medicine at Mayo Clinic.

The FDA previously granted iopofosine-131 both fast track and orphan drug designations. In Europe, the European Medicines Agency (EMA) granted orphan drug designation as well as PRIME designation for r/r WM.

Cellectar has also submitted a comprehensive data package to the EMA, including preclinical, regulatory and manufacturing data, along with safety and efficacy findings from the CLOVER WaM phase 2b study. The EMA will evaluate whether the evidence is sufficient for Cellectar to pursue conditional marketing authorization. A recommendation from the agency regarding whether to submit a marketing authorization application is expected in late July 2025.

Iopofosine I-131 utilizes a phospholipid ether as a radioconjugate monotherapy.

Cellectar’s products currently being evaluated by health agencies includes iopofosine I-131, a phospholipid drug conjugate (PDC) delivering iodine-131; CLR 121225, an actinium-225–based agent in development for solid tumors such as pancreatic cancer; and CLR 121125, an iodine-125–based radiotherapeutic being explored in solid tumors like triple-negative breast, lung and colorectal cancers. The company is also advancing proprietary preclinical PDC chemotherapy candidates and several partnered programs.

Iopofosine I-131 is also being studied in phase 2b trials for relapsed or refractory multiple myeloma and central nervous system lymphoma, as well as in the phase 1b CLOVER-2 trial for pediatric patients with high-grade gliomas. If approved for the pediatric indication, Cellectar may receive a pediatric review voucher from the FDA.

Altogether, the drug has received six orphan drug, four rare pediatric disease and two fast track designations from the FDA across multiple indications.

What Are WM and Breakthrough Therapy Designation?

WM, the most common subtype of lymphoplasmacytic lymphoma, remains incurable with current therapies, according to the International Waldenstrom’s Macroglobulinemia Foundation. Limited approved treatments highlight the need for novel therapies with unique mechanisms of action.

Breakthrough therapy designation is an FDA program designed to speed the development and review of drugs for serious or life-threatening conditions. It is granted when early clinical evidence indicates that the therapy may significantly improve at least one meaningful endpoint over available treatments. This designation provides greater interaction with the FDA and may support a six-month priority review of a future new drug application.

Iopofosine-131, a novel cancer-targeting agent, has received breakthrough therapy designation from the FDA for relapsed or refractory Waldenstrom macroglobulinemia (r/r WM). This designation highlights its potential to improve treatment outcomes over existing therapies. The phase 2 CLOVER WaM study demonstrated an 83.6% overall response rate and a 58.2% major response rate, surpassing the primary endpoint. Iopofosine-131 has also received fast track and orphan drug designations in the U.S. and Europe. Cellectar is pursuing further regulatory approvals and exploring additional indications for this promising agent.

Bridget Hoyt

FDA Approves UGN-102 For Some With Bladder Cancer

FDA Grants Fast Track Status to PHST001 for Ovarian Cancer Treatment

MVdeltaC Earns Orphan Drug Status for Pleural Mesothelioma

Iopofosine-131 Earns FDA Breakthrough for Waldenstrom Macroglobulinemia

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