On April 2, 2025 Senhwa Biosciences, Inc. (TPEx: 6492), a new drug development company focusing on first-in-class therapeutics for oncology, rare diseases, and infectious diseases, reported that the completion of Clinical Study Report (CSR) for Phase 1/Dose Expansion Trial of Silmitasertib (CX-4945) in the Treatment of Basal Cell Carcinoma(BCC), with positive data outcomes (Press release, Senhwa Biosciences, APR 2, 2025, View Source [SID1234651768]). The study included patients who had relapsed after standard therapies and had no other treatment options. Among them, three patients experienced over 30% tumor reduction (PR), and two patients had a progression-free survival (PFS) exceeding 21 months. CX-4945 significantly prolonged survival in advanced cancer patients, marking a major milestone for both the patients and Senhwa.

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Compared to current first- and second-line therapies, CX-4945 demonstrated superior tolerability and disease control potential. Therefore, Senhwa will actively pursue licensing possibilities while carefully evaluating CX-4945’s potential as a monotherapy or in combination with the second-line immunotherapy Libtayo. The company aims to explore further indications and potential as a first-line treatment, offering better options for patients.

Clinical Trial Overview

The trial enrolled 25 patients with locally advanced BCC (laBCC, 20 patients) and metastatic BCC (mBCC, 5 patients). The primary objective was to determine the recommended Phase 2 dose (RP2D) and optimal dosing regimen for CX-4945.

Among 22 patients eligible for efficacy analysis:

Three laBCC patients achieved partial response (PR).
Ten patients had stable disease (SD), including 2 mBCC and 8 laBCC patients.
Disease control rates:
mBCC patients: 80% (4 patients with complete/partial response or stable disease).
laBCC patients: 65% (11 patients with complete/partial response or stable disease).
Further data analysis showed that:

Median progression-free survival (PFS):
laBCC: 9.2 months
mBCC: 3.7 months
Median duration of disease control (DDC):
laBCC: 10.3 months
mBCC: 7.5 months
CX-4945 exhibited promising anti-tumor efficacy and disease stabilization potential in this indication. Additionally, CX-4945 has a lower study drug discontinuation rate of 24% due to AEs, compared to first-line treatments including Vismodegib and Sonidegib, suggesting superior tolerability.

Future Prospects

This trial is particularly noteworthy as it evaluated CX-4945 monotherapy in patients who had already failed first-line HHIs. Notably, 27.3% (6 patients) had also failed second-line PD-1 inhibitors such as Libtayo or Keytruda.

Among the enrolled patients, two advanced-stage patients achieved a progression-free survival (PFS) of over 21 months, lasting 653 days and 667 days, respectively. Notably, the primary lesion of the former patient was almost undetectable by visual inspection.

Given the significant potential of CX-4945 as a monotherapy, Senhwa is now actively pursuing regional licensing to accelerate commercialization through strategic partnerships, ultimately benefiting more patients.

On April 1, 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 first patient has been dosed in its Phase 3 pivotal trial evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML) (Press release, Moleculin, APR 1, 2025, View Source [SID1234651718]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, Europe and the Middle East.

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"The start of patient dosing represents a huge milestone for Moleculin and importantly, the AML community," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Our team remains focused on bringing clinical sites online in the U.S., Europe and Middle East and enrolling patients to build on this momentum. With the progress made to date, we expect to unblind preliminary data from the first 45 subjects in the second half of this year, a near-term, potentially value-driving milestone that will provide key insight as we continue to advance Annamycin’s development towards approval."

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B portion will be combined with the Phase 3 portion for purposes of measuring its primary endpoint. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.

The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding will yield 30 subjects treated with Annamycin (190mg/m2 and 230/m2) and HiDAC and 15 subjects treated with just HiDAC. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756.

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

On April 1, 2025 Hengrui Pharmaceuticals reported the 5-year long-term follow-up data from the CameL-sq study-a randomized, double-blind, multicenter, phase III trial that evaluated the efficacy and safety of camrelizumab vs. placebo in combination with carboplatin and paclitaxel as first-line treatment of advanced squamous non-small cell lung cancer (sq-NSCLC) with negative driver genes (Press release, Hengrui Pharmaceuticals, APR 1, 2025, View Source;year-survival-of-patients-with-advanced-squamous-lung-cancer—a-potential–paradigm-shift-in-clinical-outcome-for-this-difficult-to-treat-population-302416809.html [SID1234651734]). The results show that the 5-year overall survival (OS) rate for patients treated with camrelizumab plus chemotherapy was 27.8%, indicating that more than one-quarter of the patients survived beyond five years., and a 15.3% improvement over the placebo-chemotherapy group (12.5%). Additionally, camrelizumab significantly reduced the risk of mortality by 43%, enabling more patients to achieve long-term survival.

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The five-year survival rate serves as a critical indicator for assessing potential clinical cure in oncology. China’s Healthy China 2030 initiative has established an ambitious target to increase the overall five-year survival rate for cancer patients by 15%. Epidemiologic data from the National Cancer Center consistently identify lung cancer as the malignancy with the highest incidence and mortality rates across both genders. However, early symptoms of lung cancer are frequently overlooked, resulting in most patients being diagnosed at an advanced stage. In the era dominated by traditional chemotherapy, the five-year survival rate for patients with advanced lung cancer was below 10%. Among histologic subtypes, squamous NSCLC presents unique challenges as it does not benefit from targeted therapies, leading to relatively poor survival outcomes. With the advent of immunotherapy drugs, the prognosis for squamous NSCLC has significantly improved; however, numerous unmet clinical needs remain.

Camrelizumab is a humanized IgG4 monoclonal antibody independently developed by Hengrui Pharmaceuticals. With high binding affinity to PD-1, it has been shown to significantly improve the overall survival of patients with various solid tumors, including lung cancer, liver cancer, esophageal cancer, and nasopharyngeal cancer. At the 2024 ELCC, the long-term follow-up data from the CameL study were presented, highlighting the efficacy of camrelizumab in combination with chemotherapy as a first-line treatment for advanced non-squamous non-small cell lung cancer. The findings revealed that approximately one-third of the patients achieved a survival duration exceeding five years.

The significant clinical benefits of camrelizumab plus chemotherapy as demonstrated by the CameL-sq study ( an increase of 15.3% in the five year survival rate compared to the control group), represent a meaningful advancement toward the goals outlined in the Healthy China 2030 blueprint for cancer prevention and control. This breakthrough represents another significant contribution from Hengrui Pharmaceuticals, a leading innovator in China’s pharmaceutical sector, to prolong and improve the lives of cancer patients worldwide.

On April 1, 2025 US NCI reported new form of tumor infiltrating lymphocyte (TIL) therapy, a form of personalized cancer immunotherapy, dramatically improved the treatment’s effectiveness in patients with metastatic gastrointestinal cancersExit Disclaimer, according to results of a clinical trial led by researchers at the National Institutes of Health (NIH) (Press release, US NCI, APR 1, 2025, View Source [SID1234651719]). The findings, published April 1, 2025, in Nature Medicine, offer hope that this therapy could be used to treat a variety of solid tumors, which has so far eluded researchers developing cell-based therapies.

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This form of therapy involves identifying and selecting immune cells (TILs) that are found in the tumor that specifically recognize and attack a patient’s tumor cells. Next, scientists grow those TILs into large quantities in the laboratory before they are finally administered to the patient.

Patients in the clinical trial, who had a variety of gastrointestinal tumors, also received the immune checkpoint inhibitor pembrolizumab (Keytruda) to help further boost their immune response. The result was nearly 24% of patients treated with selected TILs plus pembrolizumab had a substantial reduction in the size of their tumors, compared with 7.7% of patients who received selected TILs without pembrolizumab. Patients treated with TILs that had not been selected for anti-tumor activity had no tumor shrinkage.

"We’re seeing the first extension of cellular therapy with TILs into the common solid cancers," said Steven A. Rosenberg, M.D., Ph.D., the study’s lead investigator at NIH’s National Cancer Institute. "We see a little crack in the solid wall of cancer by using cell-based immunotherapy for the common solid cancers, and we think we have ways to open that crack even further."

The clinical trial included 91 patients with metastatic gastrointestinal cancers—including esophageal, stomach, pancreatic, colon, and rectal cancers—that had worsened despite a median of four prior treatment regimens. In the pilot phase of the trial, 18 patients were treated with TILs that had not been selected for anti-tumor activity, and there were no objective responses (tumor shrinkage of at least 30% is considered an objective response). In the second phase, 39 patients were treated with selected TIL therapy, and three (7.7%) had objective responses.

In the third phase, 34 patients received pembrolizumab immediately before selected TIL therapy to prevent the newly introduced immune cells from becoming inactivated by the patient’s own immune system. This group had the best response, with 8 of 34 (23.5%) patients experiencing an objective response. All 91 patients had also received standard chemotherapy and high-dose interleukin-2 before the TIL therapy.

In the trial’s second and third phases, objective responses were seen in multiple types of gastrointestinal cancers, including cancers of the colon, rectum, pancreas, and bile duct. Responses lasted between 8 months and more than 5.8 years in the group that received selected TIL therapy alone, and between 4 months and 3.5 years in the group that received selected TIL therapy and pembrolizumab. Serious side effects occurred in 30% of patients treated with selected TILs.

The researchers are now developing methods to identify TILs that recognize multiple, specific proteins within a tumor, known as neoantigens, to help increase the number of patients who respond to selected TIL therapy with pembrolizumab.

TIL therapy, developed in the late 1980s by Dr. Rosenberg and his colleagues at NIH, uses an individual’s own TILs to fight their tumor cells. Last year, the Food and Drug Administration approved the first TIL therapy for a solid cancer, lifileucel (Amtagvi), for treating advanced melanoma.

On April 1, 2025 RenovoRx, Inc. ("RenovoRx" or the "Company") (Nasdaq: RNXT), a life sciences company developing innovative targeted oncology therapies and commercializing RenovoCath, a novel, FDA-cleared drug-delivery device, reported its financial results and business updates for the fourth quarter and full year ended December 31, 2024 (Press release, Renovorx, APR 1, 2025, View Source [SID1234651735]).

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"The fourth quarter of 2024 marks a significant milestone in our company history as we have generated our first revenue from sales of our proprietary RenovoCath device on a standalone basis, with expectations for meaningful revenue growth going forward. Importantly, we believe our current commercial strategy can be accomplished without a material increase in our capital expenditures, regardless of whether we self-commercialize or choose to partner with a larger organization and use their existing sales force and channels. Further, we believe that the gross proceeds from our February 2025 public offering of $12.1 million together with our cash on hand of $7.2 million at year end fully funds our current operational plan as we look to scale RenovoCath revenues and progress our Phase III TIGeR-PaC clinical trial towards key milestones. Moreover, we expect that growing RenovoCath revenues will reduce our burn rate as we prudently deploy our cash on hand to drive shareholder value," said Shaun Bagai, CEO of RenovoRx.

"Based on our internal analysis with current clinical applications, we believe that our initial total addressable market (TAM) for RenovoCath represents an estimated $400 million peak annual U.S. sales opportunity. Beyond this, there are expansion opportunities across other indications that could create the potential for a several billion-dollar U.S. TAM for RenovoCath over time," continued Mr. Bagai. "We expect revenue to increase to the low six figure range for the first quarter of 2025 followed by sequential quarter-over-quarter increases for the remainder of the year."

RenovoCath Commercialization Update

In December 2024, RenovoRx received first commercial purchase orders for its RenovoCath device, which resulted in revenue generation of approximately $43,000 for the fourth quarter of 2024.

RenovoRx is seeing strong organic demand for RenovoCath. More than ten medical institutions have initiated the process for RenovoCath purchase orders, and purchase orders have already been received from several esteemed, high volume National Cancer Institute-designated centers. Additionally, utilization of RenovoCath devices by initial customers has led to repeat purchase orders. Moreover, RenovoRx believes the twenty cancer centers that have used RenovoCath as part of its TIGeR-PaC trial could also be potential customers for RenovoCath after completion of TIGeR-PaC enrollment later this year.

RenovoRx has identified its initial target market for RenovoCath to be approximately $400 million in peak annual U.S. sales, based on the Company’s internal assumptions1. Moreover, expansion opportunities across other clinical indications could create a several billion-dollar total addressable market potential for RenovoCath over time. It’s management’s belief that the Company can achieve meaningful market penetration with a small commercial team targeting the top 200 high-volume treatment centers.

Importantly, there is a current reimbursement code with the Centers for Medicare and Medicaid Services covering procedures utilizing specialty pressure-mediated delivery catheters, which creates incentives for hospitals to adopt more advanced technology, like RenovoCath.

Ongoing Pivotal Phase III TIGeR-PaC Clinical Trial Update

During the fourth quarter 2024, RenovoRx added several additional renowned clinical oncology sites to participate in the TIGeR-PaC study. The initiation of these sites allows for new patient enrollment at the Sarah Cannon Research Institute Oncology Partners in Nashville, TN and at the Northwell Health Cancer Institute Clinical Site in New Hyde Park, NY which are key additions to the number of clinical sites to support RenovoRx’s path to completing patient enrollment for the trial. RenovoRx is continuing to target additional clinical oncology sites, with the expectation that the study will achieve full enrollment during 2025.

The current protocol and statistical analysis plan for the TIGeR-PaC trial requires 114 randomized patients, with 86 events (deaths) necessary to complete the final analysis. As of March 28, 2025, 90 patients have been randomized with 50 events having occurred. A second interim analysis will be triggered by the 52nd event.

The timing required to analyze the data after the 52nd event is expected to take several months and includes a full review with recommendations by the TIGeR-PaC Data Monitoring Committee. RenovoRx currently anticipates the 52nd event to occur during the second quarter of 2025. The key recommendation from the Data Monitoring Committee on whether or not to continue the study based on the data reviewed is expected to be announced in the second half of 2025.

Fireside Chat Strategic Update

RenovoRx will host a fireside chat with Shaun Bagai, Chief Executive Officer, on Thursday, April 3, 2025, at 12:00 p.m. ET. During the event, Mr. Bagai will discuss the momentum of RenovoRx’s commercialization efforts for its RenovoCath device, including an update on initial revenues generated, and continued progress on the ongoing Phase III TIGeR-PaC clinical trial. Additionally, one of RenovoCath’s initial customers, Gregory Tiesi, MD, FACS, FSSO, Medical Director of Hepatobiliary Surgery, Division of Surgical Oncology, Hackensack Meridian Jersey Shore University Medical Center, will join Mr. Bagai. He will share his insights on the Trans-Arterial Micro-Perfusion (TAMP) therapy platform and its impact on patient care. Hackensack Meridian Jersey Shore University Medical Center began using the RenovoCath device with oncology patients in December 2024.

Fireside Chat Details:
Date: Thursday, April 3, 2025
Time: 12:00 p.m. ET
Webcast: View Source

A question and answer session will occur at the end of the call, and a link to the recording of this presentation will be available on RenovoRx’s Investor Relations website after the event.