On February 13, 2025 RenovoRx, Inc. ("RenovoRx" or the "Company") (Nasdaq: RNXT), a life sciences company developing novel targeted oncology therapies and commercializing RenovoCath, an innovative, FDA-cleared device, reported its recent abstract presentation at the 2025 Society of Interventional Oncology Annual Conference ("SIO 2025") in Las Vegas, Nevada (Press release, Renovorx, FEB 13, 2025, View Source [SID1234650273]). The abstract published promising pre-clinical data from the use of RenovoRx’s patented Trans-Arterial Micro-Perfusion (TAMP) therapy platform, which aims to optimize local and targeted drug delivery in difficult-to-treat cancers.

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The abstract, titled "Micro-CT imaging following intra-arterial delivery of a radiopaque silicone polymer using a double-balloon occlusion catheter in pigs: a model to analyze tissue penetration via the trans-arterial micro perfusion (TAMP) technique," was presented at SIO 2025 on February 3, 2025 by Paula Novelli, MD, of the University of Pittsburgh Medical Center.

The results of the study support a more optimized drug delivery method for tumors that suffer from limited blood supply and poor drug penetration. The TAMP technique was evaluated in a porcine (pig) model utilizing micro-CT imaging for precise monitoring of drug penetration into tissue.

The pre-clinical data shows that drug delivery with the patented, FDA-cleared dual balloon RenovoCath device via TAMP potentially may improve localized drug delivery by achieving greater drug penetration in the microvasculature near the target tumor. The data shows that, by using RenovoCath, researchers were able to isolate sections of the blood vessel through the adjustment of the distance between the occlusion balloons, thereby excluding any off-target side branches. Researchers were able to confirm the level of penetration into the targeted area utilizing micro-CT imaging. The penetration achieved appears to be consistent with a more optimized drug delivery. The observed effect is expected to be even stronger if radiation is applied beforehand.

"This study highlights that TAMP is an encouraging approach for improving localized drug delivery to difficult-to-treat tumor areas. By understanding how the infusion medium penetrates tissue, techniques can be refined for better clinical outcomes, especially for patients with cancers like pancreatic adenocarcinoma," said Dr. Ramtin Agah, RenovoRx’s Founder, Chairman of the Board and Chief Medical Officer and one of the study’s authors. "We look forward to continued research in hypovascular tumors using minimally invasive procedures and are also working on a radiation sub-study. With continued investigation, we believe TAMP has the potential to revolutionize how interventional oncologists approach cancer treatment, offering a more targeted, effective solution for challenging cases."

The procedure in this study utilized a porcine model, where the RenovoCath device was effectively employed to deliver a technique involved inflating the catheter balloons in the splenic artery, the superior mesenteric artery and renal artery. This approach enabled precise side branch exclusion in both vascular regions, ensuring targeted and controlled intervention, allowing for extensive filling of the microvessels with Microfil (a micro-CT imaging following an injection of a radiopaque silicone rubber imaging reagent) in the perivascular space. In comparison, the control group, where balloons in the splenic arteries were left deflated, showed minimal Microfil filling beyond the main artery, as confirmed by micro-CT imaging. These striking differences highlight the potential of this technique to drive forward the development of targeted therapies, especially for complex cancers like pancreatic adenocarcinoma.

"We are excited to highlight this pre-clinical data abstract presented at SIO 2025. Including procedures performed as part of clinical trials, intra-arterial drug delivery by RenovoCath has been used in over 500 procedures by oncologists and interventional radiologists over the past several years," said Shaun Bagai, RenovoRx CEO.

Mr. Bagai added, "Based on recent positive feedback we have received from medical practitioners, we have launched an effort to commercialize RenovoCath as a stand-alone device within its FDA-cleared indications for use in temporary vessel occlusion in applications including arteriography, preoperative occlusion, and chemotherapeutic drug infusion."

Additionally, the TAMP therapy platform was the topic of the SIO 2025 panel discussion "Breaking Barriers in Drug Delivery: Trans-Arterial Micro-Perfusion," where key interventional oncologists including Dr. Novelli, Khashayar Farsad, MD, PhD, Oregon Health and Science University, and David Sperling, MD, FSIR, Columbia University Irving Medical Center discussed their experience with the therapy platform and the RenovoCath device.

About RenovoCath

Based on its FDA clearance, RenovoCath is intended for the isolation of blood flow and delivery of fluids, including diagnostic and/or therapeutic agents, to selected sites in the peripheral vascular system. RenovoCath is also indicated for temporary vessel occlusion in applications including arteriography, preoperative occlusion, and chemotherapeutic drug infusion. For further information regarding our RenovoCath Instructions for Use ("IFU"), please see: IFU-10004-Rev.-F-Universal-IFU.pdf.

On February 13, 2025 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported a new collaboration with the Institute for Follicular Lymphoma Innovation (IFLI), a global, non-profit, private foundation dedicated to accelerating the development of innovative treatment options for patients with follicular lymphoma (FL) (Press release, Tempus, FEB 13, 2025, View Source [SID1234650274]). The collaboration aims to develop and make available a real-world multimodal, deidentified FL data library in Lens – Tempus’ data analytics platform – through which researchers may derive AI-driven insights to accelerate the development of FL treatments in an effort to improve patient outcomes.

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IFLI is committed to supporting advances in understanding the biology of FL patients who are at high risk of cancer progression and enhancing therapeutic development to address critical unmet needs in the management of this disease. The collaboration will initially focus on prospectively generating multimodal FL data, particularly for POD24 patients who experience disease progression within 24 months of treatment, through a Tempus-sponsored study.

The availability of a follicular lymphoma data library has the potential to improve researchers’ understanding of the disease’s biology. Providing researchers with access to robust multimodal data supports their work in uncovering critical insights into the heterogeneity of FL, which may lead to the development of more targeted precision medicines. A comprehensive understanding of the disease is crucial for tailoring treatments to individual patient profiles, enhancing outcomes, and advancing the fight against this complex disease.

"This collaboration exemplifies the power of combining IFLI’s specialized focus on FL with Tempus’ cutting-edge AI-enabled solutions," said Kate Sasser, PhD, Chief Scientific Officer at Tempus. "We are excited to work with IFLI and explore a new way of working with foundations to advance research. By leveraging our combined expertise and reach, we aim to create a centralized and dynamic data library that enables researchers to better understand FL."

"IFLI is thrilled to announce our collaboration with Tempus, chosen for their exceptional leadership in the field. Together, we aim to create a robust, de-identified FL database, accelerating research and drug development to ultimately find a cure for the FL patient community," said David McCullagh, Managing Director at IFLI. " We believe Tempus is uniquely positioned to develop this comprehensive FL database, making it accessible to professionals in the field to combat this disease effectively."

On February 13, 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 tumors and viruses, reported it has received U.S. Food and Drug Administration ("FDA") feedback and guidance on its IND amendment that has allowed a reduction in the size of its Phase 3 pivotal trial protocol 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) (MB-108) (Press release, Moleculin, FEB 13, 2025, View Source [SID1234650256]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US, Europe and the Middle East.

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"Given the importance of our Miracle trial as a pivotal registration clinical trial, we were extremely pleased to receive detailed follow up from the FDA regarding our IND amendment to enable the trial. While all of the major aspects of the trial remain unchanged, guidance from FDA recommended an alteration to the statistical plan that will allow us to reduce the size of Part B of our trial by approximately 10%. Moreover, the nature of the feedback helps us move forward quickly to open sites in the US, in addition to the sites we are expecting to open in Europe and the Middle East. All of this supports the pursuit of an accelerated timeline for new drug approval," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

Mr. Klemp added, "The spirit of collaboration surrounding the Miracle trial is, frankly, inspiring. From investigating clinicians to regulatory authorities around the world, people are starting to realize the potential significance of approving the first-ever non-cardiotoxic anthracycline. Annamycin’s approval in AML alone would bring the potential to save thousands of lives every year, and the opportunity to eliminate the threat of cardiotoxicity in a wide range of tumors equates to as much as 20 times that in long term potential. It’s critical to remember that nearly half of all cancers are treated with an anthracycline and 60% of all childhood cancers. One of the things that drives us every day is our belief that no child should ever have to be subjected to a cardiotoxic anthracycline once Annamycin is approved."

The MIRACLE study, 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 amended protocol 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). This early unblinding will yield 30 subjects with Annamycin (190mg/m2 and 230/m2) and HiDAC and 15 subjects 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 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 February 13, 2025 CareDx, Inc. (Nasdaq: CDNA) – The Transplant Company – a leading precision medicine company focused on the discovery, development, and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers – reported new AlloHeme data presented at the 2025 Tandem Meetings, Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held February 12-15, 2025 in Honolulu, Hawaii (Press release, CareDx, FEB 13, 2025, View Source [SID1234650275]).

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In an oral presentation, a one-year interim analysis of the ACROBAT prospective, multi-center study showed that AlloHeme is an accurate and sensitive test for monitoring relapse after allogeneic stem cell transplantation in Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS). By measuring small changes in mixed chimerism, AlloHeme demonstrated excellent performance characteristics with a hazard ratio (HR) of 40.5 (p<0.001) for relapse, and a clinically meaningful median lead time to relapse of 36 days. The analysis included one-year interim follow-up data from a cohort of 229 patients with AML and MDS from 11 stem cell transplant centers across the U.S.

"We are extremely pleased with the results of the ACROBAT study which demonstrates that AlloHeme is highly accurate in predicting risk of relapse in patients who have undergone a hematopoietic cell transplant," said Dr. Monzr M. Al Malki, Associate Professor, and Director of Unrelated Donor Bone Marrow Transplant Program at City of Hope National Medical Center. "This study gets us one step closer to having a highly reliable molecular biomarker that enables us to assess the status of the stem cell engraftment and predict risk of relapse."

CareDx’s AlloHeme is an NGS-based test that has been shown to be more sensitive than the current standard of care methods for monitoring engraftment and relapse post-allogeneic hemopoietic stem cell transplantation for hematologic malignancies.

"The interim results of the ACROBAT study build upon our growth strategy to expand into hematology oncology. With AlloHeme, we can detect relapse after allogeneic stem cell transplantation prior to conventional methods, giving clinicians the significant lead time they need to intervene sooner," said Marica Grskovic, PhD, CareDx Chief Strategy Officer. "We are very pleased with these results demonstrating the high sensitivity of the AlloHeme assay and its selection for an oral presentation at the Tandem Meetings given the significant impact it may have on patient outcomes through earlier detection of malignancy recurrence."

On February 13, 2025 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2024 (Press release, Agios Pharmaceuticals, FEB 13, 2025, View Source [SID1234650239]).

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"Agios had a transformative year in 2024, continuing to successfully deliver on all priorities. Our PYRUKYND franchise is poised for multi-billion-dollar potential, driven by the key milestones we achieved last year, including filing for regulatory approval in thalassemia across four markets and completing enrollment in our Phase 3 RISE UP study for sickle cell disease," said Brian Goff, chief executive officer at Agios. "Backed by a strong balance sheet and a highly experienced team, Agios is focused on maximizing the potential PYRUKYND launches in thalassemia and sickle cell disease in 2025 and 2026, respectively, while advancing and diversifying our key pipeline programs and strategically deploying our capital to drive long-term growth. We are well positioned to bring significant value for shareholders, healthcare professionals and patients, as we build towards a breakout year in 2025."

Fourth Quarter 2024 and Recent Highlights

•PYRUKYND Revenues: Generated $10.7 million in net revenue for the fourth quarter of 2024, a 20 percent increase from the third quarter of 2024, primarily driven by year-end stocking and adjustments to certain revenue reserves. A total of 223 unique patients have completed prescription enrollment forms, representing an increase of 6 percent over the third quarter of 2024. A total of 130 patients are on PYRUKYND therapy, inclusive of new prescriptions and continued therapy.
•Thalassemia:
◦Presented positive results from the ENERGIZE-T Phase 3 randomized clinical trial evaluating mitapivat versus placebo in adults with transfusion-dependent alpha- or beta-thalassemia at the 66ᵗʰ American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2024) in December 2024.
◦Based on the favorable benefit-risk profile observed in both the ENERGIZE and ENERGIZE-T Phase 3 studies, filed regulatory applications for mitapivat (PYRUKYND) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia with the U.S., European Union, Kingdom of Saudi Arabia and United Arab Emirates health authorities.
◦The U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The review classification for this application is Standard and the Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025.
•Sickle Cell Disease:
◦Completed enrollment of the Phase 3 RISE UP study evaluating mitapivat for the treatment of sickle cell disease patients who are 16 years of age or older. This Phase 3 study enrolled more than 200 patients worldwide.
◦European Commission adopted a positive decision for the designation of mitapivat as an orphan medicinal product for the treatment of sickle cell disease.
◦Presented Phase 1 clinical results on tebapivat (AG-946) in patients with sickle cell disease at ASH (Free ASH Whitepaper) 2024.
•Pediatric Pyruvate Kinase (PK) Deficiency:
◦Reported in a separate press release today, positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused.
•Lower-risk myelodysplastic syndromes (LR-MDS):
◦Initiated patient enrollment in the Phase 2b study of tebapivat in LR-MDS.
◦FDA granted orphan drug designation to tebapivat for the treatment of MDS.
•Medical Congresses: Presented 16 abstracts highlighting new data on mitapivat and tebapivat at ASH (Free ASH Whitepaper) 2024.
•Corporate: David Schenkein, M.D., has informed the company that he will step down from Agios’ Board of Directors, effective February 28, 2025, to devote more time to his other commitments. He will continue to serve as a strategic advisor to Agios’ Leadership Team, concentrating on advancing the company’s clinical development programs.

Key Upcoming Milestones & Priorities

Agios expects to achieve the following key milestones in 2025:
•Thalassemia: Receive FDA regulatory decision for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia (PDUFA goal date is September 7, 2025).
•Sickle Cell Disease: Announce topline results from the Phase 3 RISE UP study of mitapivat in sickle cell disease in late 2025, with a potential U.S. commercial launch in 2026. Additionally, begin patient enrollment for the Phase 2 study of tebapivat in sickle cell disease in mid-2025.
•LR-MDS: Complete patient enrollment in the Phase 2b study of tebapivat for LR-MDS in late 2025.
•Early-Stage Pipeline: File an Investigational New Drug Application for AG-236, a siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera, in mid-2025.

Fourth Quarter 2024 Financial Results

Revenue: Net product revenue from sales of PYRUKYND for the fourth quarter of 2024 was $10.7 million, compared to $7.1 million for the fourth quarter of 2023, and $36.5 million for the year ended December 31, 2024, compared to $26.8 million for the year ended December 31, 2023.

Cost of Sales: Cost of sales for the fourth quarter of 2024 was $1.3 million and $4.2 million for the full year ended December 31, 2024.

Research and Development (R&D) Expenses: R&D expenses were $82.8 million for the fourth quarter of 2024, compared to $77.5 million for the fourth quarter of 2023, and $301.3 million for the full year ended December 31, 2024, compared to $295.5 million for the full year ended December 31, 2023.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $51.7 million for the fourth quarter of 2024 compared to $35.3 million for the fourth quarter of 2023, and $156.8 million for the full year ended December 31, 2024, compared to $119.9 million for the full year ended December 31, 2023. The year-over-year increase was primarily attributable to an increase in commercial-related activities as the company prepares for the potential approval of PYRUKYND in thalassemia.

Net Income (Loss): Net loss was $96.5 million for the fourth quarter of 2024 compared to a net loss of $95.9 million for the fourth quarter of 2023 and net income was $673.7 million for the year ended December 31, 2024, compared to a net loss of $352.1 million for the year ended December 31, 2023.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of December 31, 2024, were $1.5 billion compared to $806.4 million as of December 31, 2023. This increase reflects payments from the royalty monetization for vorasidenib and a milestone payment from Servier for vorasidenib approval received in the third quarter of 2024. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND launches in thalassemia and sickle cell disease, advance existing programs, and to opportunistically expand its pipeline through both internally and externally discovered assets.

Conference Call Information

Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s fourth quarter 2024 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.