On April 17, 2025 Endevica Bio, a privately held company developing first-in-class peptide drug candidates, reported the dose administration for the first patient in a Phase 2 trial for its experimental drug TCMCB07 (B07) to prevent weight loss in cancer patients undergoing chemotherapy (Press release, Endevica Bio, APR 17, 2025, View Source [SID1234651979]).

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The trial, being conducted in partnership with WuXi Clinical, will include 20 sites and 100 patients who are diagnosed with stage 4 metastatic colorectal cancer.

In the trial, patients are dosed with B07 as they begin chemotherapy and during the first several rounds of chemotherapy. The primary endpoint is preventing weight loss, which can lead to a debilitating condition called cachexia, a life-threatening wasting syndrome associated with chronic diseases, including cancer.

"This marks an important milestone in our commitment in developing a potentially life changing treatment for cachexia," said Russell Potterfield, Chief Executive Officer and Executive Chair of Endevica. "Each trial brings us closer to offering a viable solution for this debilitating disease, and we remain dedicated to making a lasting impact on the lives of those affected."

"We are incredibly excited to have our first patient dosed with B07 in individuals diagnosed with metastatic colorectal cancer who are undergoing chemotherapy," said Dr. Daniel Marks, Chief Medical and Scientific Officer of Endevica Bio. "Since there is no FDA approved therapeutic for cancer cachexia, this trial is a crucial step to provide a therapy for an area of huge unmet clinical need, and we look forward to the results."

In 2024, Endevica Bio completed its Phase 1 clinical trial with preliminary findings supporting its strong safety and efficacy. Last November, the Journal of Clinical Investigation, showed that B07 improved the appetite and preserved lean mass and fat mass in rodent models of cancer and its associated combination chemotherapy. This same study showed the strong potential of B07 to alleviate chemotherapy-induced anorexia and weight loss for millions of patients worldwide.

About TCMCB07
TCMCB07 is a melanocortin‐3/4 antagonist peptide candidate in clinical development for the treatment of cachexia. It is designed to be a first-in-class peptide drug with the ability to cross the blood-brain barrier and act on previously inaccessible target receptors to modulate the body’s behavioral and metabolic response to chronic illness. Pre-clinical animal trial results show significant lean muscle mass retention (e.g., a reversal of the cachectic condition) during administration of the drug. The results are consistent in cachexia arising from many different types of chronic disease.

On April 17, 2025 Researchers with City of Hope, one of the largest and most advanced cancer research and treatment organizations in the U.S. with its National Medical Center named top 5 in the nation for cancer by U.S. News & World Report, reported it will present the latest cancer research at the AACR (Free AACR Whitepaper) Annual Meeting 2025, which will take place April 25 to 30 in Chicago (Press release, City of Hope, APR 17, 2025, View Source [SID1234651980]).

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City of Hope experts will present more than 74 chaired, plenary, educational, minisymposium, poster and other sessions on the use of artificial intelligence (AI), multiomics and other emerging technologies in cancer research, advances in the clinical application of natural killer cells, new clinical trial results, translating research into policy through community engagement and much more.

"The extensive breadth and depth of research being presented at AACR (Free AACR Whitepaper) by City of Hope’s researchers exemplifies our commitment to making hope a reality for all cancer patients," said John D. Carpten, Ph.D., City of Hope’s chief scientific officer, Irell & Manella Cancer Center Director’s Distinguished Chair and Morgan & Helen Chu Director’s Chair of the Beckman Research Institute "We are proud to share our dedication to developing innovative technologies and treatments for improved care through a combination of scientific discovery, clinical ingenuity and compassion."

Chaired sessions

David W. Craig, Ph.D., professor and founding chair of the Department of Integrative Translational Sciences within Beckman Research Institute of City of Hope, is chairing the final plenary session of the conference, "Opportunities in Predicative Oncology." He will deliver opening and closing remarks for the session focused on exploring emerging computational, biological and clinical approaches to learn more about tumors at multiple levels and improve precision medicine. "Opportunities in Predictive Oncology," plenary session PL05, will take place on Wednesday, April 30, 2025, 8 to 10 a.m.

"This session will explore how researchers are working at the forefront of our field and pushing the boundaries of cancer care by using leading-edge technologies to improve how we can target disease on an individual basis," Dr. Craig said. "Combining multifaceted sets of data allows us to better understand treatment resistance, build stronger predictive models and enhance outcomes using precision medicine."

Dr. Craig is also chair of an educational session on integrating AI and data science to gain deeper insights into the causes of cancer and how it progresses. He will present on using a biological analysis approach called multiomics to investigate the unique genetic makeup of different cell populations in solid tumors.

"Accelerating Cancer Research with AI and Data Science: Multi-scale Multi-Modal Integration for Deeper Insights," educational session ED04, is on Friday, April 25, 2025, 4:45 to 6:15 p.m. Dr. Craig’s presentation, "Resolving spatial subclonal genomic heterogeneity using the integration of multi-omic genomic approaches in solid tumors," is the first of the session.

In addition, Nina (Jiarong) Song, a graduate student in Dr. Craig’s lab, will present new data from a team of researchers that highlights the use of AI to integrate digital pathology, genomics and spatial transcriptomics to gain new insights into glioblastoma, triple-negative breast cancer and colorectal cancer progression. Part of the minisymposium MS.BCS01.01 "Artificial Intelligence and Machine Learning for Basic and Translational Research," held on Sunday, April 27, 2025, 3 to 5 p.m., Song’s presentation, "Decoding tumor microenvironment with deep learning: merging spatial transcriptomics and histopathology," is the first talk of the minisymposium.

Michael A. Caligiuri, M.D., former president of City of Hope National Medical Center and professor in the Department of Hematology & Hematopoietic Cell Transplantation, is the chair of two educational sessions. He will introduce a session on advances in the application of natural killer (NK) cells — a type of white blood cell that destroys cancer and can be harnessed as a therapeutic intervention, which is being researched at City of Hope — and present on "Innate immune lymphocytes, including NK cells." The educational session ED57, "Natural Killer Cells: Advances in Basic Biology and Clinical Applications," is on Saturday, April 26, 2025, 10 to 11:30 a.m. Dr. Caligiuri’s presentation is the first of the session.

Dr. Caligiuri is also chair of ED54, "Academic Entrepreneurship: Getting Your Discovery to Patients, Part 1—Liftoff," on Saturday, April 26, 2025, 8 to 9:30 a.m., which will help define the steps required for translating research from the bench to the bedside.

Select presentations by City of Hope scientists and physicians include:

"Managing and predicting toxicities from new and emerging ADCs"

As part of a session on advances in diagnostics and therapeutics, Hope Rugo, M.D., who recently joined City of Hope as director of its Women’s Cancers Program, will talk about new findings in managing toxicities from antibody-drug conjugates. (Advances in Diagnostics and Therapeutics session ADT04: Monday, April 28, 2025, 1:25 to 1:45 p.m.)

Dr. Rugo will also serve as a discussant at the Clinical Trials Plenary Session on Biologics and T-cell Engagers on Tuesday, April 29, 10:15 a.m. to 12:15 p.m.

"Multi-omics analysis of MYC gene and WNT signaling pathway alterations in early-onset colorectal cancer in Hispanic/Latino patients, enhanced with spatial transcriptomics approaches"

Francisco (Paco) Carranza, a postdoctoral scientist in the lab of Enrique Velazquez Villarreal, M.D., Ph.D., M.P.H., M.S., assistant professor in the Department of Integrative Translational Sciences, will present on the investigation of early-onset colorectal cancer in young Hispanic/Latino populations using multi-omics analysis to improve precision medicine in underrepresented populations. (Minisymposium session 3742: Monday, April 28, 2025, 2:35 to 2:50 p.m.)

"PM-AI agent: A conversational artificial intelligence system for precision medicine and advancing health equity through integrative clinical, genomic and social determinants of health data analysis"

Dr. Velazquez Villarreal will also present a poster on the development of a new precision medicine AI tool that addresses key challenges in the integration of clinical, genomic, population-specific variables and social determinants of health to promote better equity in cancer research. (Poster presentation 1115/1: Sunday, April 27, 2025, 2 to 5 p.m.)

"Penpulimab versus placebo in combination with chemotherapy as first-line treatment for recurrent or metastatic nasopharyngeal carcinoma: A global, multicenter, randomized, double-blind, phase 3 trial (AK105-304)"

Medical oncologist Aditya Shreenivas, M.D., M.S. will present new data from phase 3 of a clinical trial testing Penpulimab, a humanized anti-PD-1 monoclonal antibody, in patients with advanced nasopharyngaeal carcinoma. (Clinical trials minisymposium session CT011: Sunday, April 27, 2025, 3:50 to 4 p.m.)

"Overcoming intrinsic mechanisms of cell cycle inhibitor resistance in estrogen receptor-positive (ER+) breast cancer"

Kimya Karimi, a postdoctoral scholar in the lab of Andrea Bild, Ph.D., professor in the Department of Medical Oncology & Therapeutics Research, will present new data from a City of Hope study exploring a combination treatment to overcome primary endocrine therapy resistance in estrogen receptor-positive breast cancer. (Minisymposium session 6383: Tuesday, April 29, 2025, 3:05 to 3:20 p.m.)

"The science and art of community engagement for translating research into cancer-related policy and implementation"

Kimlin Tam Ashing, Ph.D., professor and founding director of the Center of Community Alliance for Research & Education at City of Hope will present during an educational session on building community outreach and engagement partnerships for improved health outcomes. (Educational session ED39: Friday, April 25, 2025, 3:30 to 3:50 p.m.)

Highlighted poster sessions

Jing Qian, a senior research associate in the lab of Dr. Carpten is first author on "Spatial transcriptomics reveals differences in the tumor and immune microenvironment of high-grade serous ovarian cancers with differing responses to immune checkpoint inhibitors." (Late breaking poster session LB076/12: Sunday, April 27, 2025, 2 to 5 p.m.)

Peter Zang, M.D., a hematology and oncology fellow is first author on "Digital spatial profling with GeoMx to identify differential protein expression in Non-Hispanic/Latino and Hispanic/Latino Patients with metastatic hormone sensitive prostate cancer," a poster abstract that outlines work from the lab of senior author Tanya Barauskas Dorff, M.D., professor in the Department of Medical Oncology & Therapeutics Research on the complex interplay between ethnicity and disease biology in prostate cancer. (Poster session 5103/8: Tuesday, April 29, 2025, 2 to 5 p.m.)

A poster presentation by Sydney Grant, a postdoctoral fellow, and Aritro Nath, Ph.D., assistant professor in the Department of Medical Oncology & Therapeutics Research, "Integrating multimodal data with survival-based variational autoencoders to predict recurrence-free survival in breast cancer," highlights a new model the team developed to predict recurrence-free survival in breast cancer patients. (Poster session 5011/15: Tuesday, April 29, 2025, 9 a.m. to noon)

On April 16, 2025 Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, reported sales for the first quarter of 2025 (Press release, Ipsen, APR 16, 2025, View Source [SID1234651945]).

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Q1 2025 % change % change
€m Actual CER1
Oncology 655.0 8.5% 8.0%
Neuroscience 193.5 8.0% 9.6%
Rare Disease 70.3 78.4% 74.6%
Total Sales 918.8 11.7% 11.6%

"Ipsen has delivered a strong start to 2025, building further momentum in the transformation of our company," commented David Loew, Chief Executive Officer, Ipsen. "We continued to execute on our strategy with strong top-line growth and pipeline progression. I am pleased to see the rapid build-up of our Rare Cholestatic Liver disease franchise with two innovate medicines for five indications. 2025 is set to be an important year for Ipsen, with multiple launches underway and several milestones expected across our portfolio."

Full-year guidance

Ipsen is confirming financial guidance for full-year 2025:

Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in March 2025, a limited effect on total sales from currencies is expected.
Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities.
Guidance includes expected negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. It excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions.

Upcoming Milestones

Ipsen anticipates several key milestones across its portfolio in 2025, including:

Cabometyx (CABINET trial) – Regulatory decision in the European Union for advanced pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors (NETs).
fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP).
LANT3 (LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics.
Q1 pipeline progress

The regulatory filing for tovorafenib was accepted by EMA for review in the European Union, marking an important step forward in the development of this potential treatment for pediatric low-grade glioma and reinforcing Ipsen’s commitment to innovation in rare and difficult-to-treat cancers.

Ipsen also initiated the entry in Phase I of IPN01195, a RAF inhibitor, complementing IPN01194, an ERK inhibitor, and tovorafenib, two other assets targeting the MAPK pathway.

Group refinancing

Ipsen announced on March 19th the successful completion of its inaugural Rated Public Bond of €500 million with a coupon of 3.875%, maturing in March 2032. Following the disclosure of the Investment Grade ratings from S&P and Moody’s, this transaction was very well received and largely oversubscribed by a diversified and solid institutional investor base. This transaction is an important component of Ipsen’s refinancing plan which included the successful renewal of €1,5 billion syndicated Revolving Credit Facility, extending Ipsen’s debt maturity profile.

Conference call

A conference call and webcast for investors and analysts will begin today at 2pm CET. Participants can access the call and its details by registering here; webcast details can be found here.

Calendar

Ipsen intends to publish its half-year results on July 31st, 2025.

Notes

All financial figures are in € millions (€m). The performance shown covers the three-month period to 31 March 2025 (Q1 2025, the quarter), compared to the three-month period to 31 March 2024 (Q1 2024), unless stated otherwise.

On April 16, 2025 Oncotelic Therapeutics, Inc (OTCQB:OTLC) ("Oncotelic", the "Company" or "We"), a developer of treatments for rare and orphan indications, including Parkinson’s Disease, pancreatic ductal adenocarcinoma ("PDAC"), diffuse intrinsic pontine glioma ("DIPG"), and various other cancers, reported its financial results for the fiscal year ended December 31, 2024 ("FY 2024"), as compared to the fiscal year ended December 31, 2023 ("FY 2023") (Press release, Oncotelic, APR 16, 2025, View Source [SID1234651963]). The financial results are based on the Annual Report on Form 10-K ("Form 10-K") as filed with the Securities and Exchange Commission ("SEC") on April 15, 2025.

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Highlights for FY 2024 and thereafter:

2024 has been a transformational year for us. We made great progress on multiple fronts and this momentum continues in 2025. We individually discuss each of these areas below:

San Diego Facility

Our joint venture ("JV"), GPM Biotechnology Limited, with Dragon Capital Overseas Limited has continued the development of its nanoparticle pipeline at its facility in San Diego ("Facility") since late 2023 / early 2024. In late 2024, just over a year after breaking ground, the Facility became good manufacturing practices certified and was issued a Drug Manufacturing License by the State of California Department of Public Health and Food and Drug Branch. The Facility is planned to primarily manufacture our drug product, including the clinical trial materials, for the clinical programs for each of our compounds.

Nanoparticle Platform

At the Facility, the JV initially identified 5 compounds, including OT-101, for development as nanoparticles. Subsequently, the JV identified an additional compound, bringing the total to 5 new compounds, not including OT-101. Each of the nanoparticle compounds is designed as a next-generation anticancer agents. We believe that each of these new compounds can potentially lead to significant revenue and value for the JV, which in turn could lead to significant value to the Company due to our investment in the JV. Two of the 6 compounds are in late stages of manufacturing, and the Company plans to file INDs for both before the end of the year. Our proprietary nanoparticle platform enables the development of multiple therapeutic candidates, to address various oncology indications.

Investigational New Drug Filing Platform with Shanghai Medicilon

We recently announced that we entered into an agreement to utilize the proprietary rapid investigational new drug ("IND") platform created and owned by Shanghai Medicilon Inc. ("Medicilon") to file up to 20 new INDs. We, and the JV, plan to utilize it to file the 6 compounds identified utilizing the Medicilon IND platform, among and upto 20 total filings.

OT-101 Clinical Program

The primary candidate of our JV, OT-101, which has previously completed multiple clinical trials, is currently undergoing a Phase 3 trial in pancreatic cancer. OT-101 also completed a phase 1 combination trial with IL-2. This will allow us to pursue OT-101 in combination with various checkpoint inhibitors, such as PD-1 blockers.

Artificial Intelligence Platform

Our artificial intelligence ("AI") team has played an increasingly important role in the development of the Company and the Facility. Our proprietary AI technology has been used by our scientists in writing research papers, development reports, and regulatory documents from the data gathered from our Facility and elsewhere. As announced in December 2024, we are leveraging our AI Platform, "PDAOAI", to allow third party researchers and individuals to utilize our platform in their workflow. Unlike standard AI tools, PDAOAI is specifically designed for pharmaceutical regulatory processes and research documentation, streamlining workflows and potentially accelerating development timelines.

Results of Operations

Below is a presentation of our financial results comparing FY 2024 to FY 2023, and are based on our results published in our Form 10-K filed with the SEC on April 15, 2025.

FY 2024 compared to FY 2023 Financial Results Overview

ONCOTELIC THERAPEUTICS, INC. AND SUBSIDIARIES
CONSOLIDATED STATEMENTS OF OPERATIONS

For the Year Ended December 31,
2024 2023
Service Revenue $ - $ 70,000
Total Revenue - 70,000
Operating expenses:
Research and development $ - $ 61,143
General and administrative 376,013 573,726
Goodwill impairment (See note 2 and 3) 3,200,000 6,083,146
Total operating expenses 3,576,013 6,718,015
Income/(Loss) from operations (3,576,013 ) (6,648,015 )
Other income (expense):
Interest expense, net (857,723 ) (1,044,786 )
Reimbursement for expenses – related party 22,937 72,246
Change in fair value of investment in GMP Bio - 12,706
Change in fair value of derivative on debt (280,402 ) (225,074 )
Loss on debt conversion (88,258 ) (373,142 )
Total other income (expense) (1,203,446 ) (1,558,050 )
Net income (loss) before non-controlling interests (4,779,459 ) (8,206,065 )
Net loss attributable to non-controlling interests (255,527 ) (302,972 )
Net income (loss) attributable to Oncotelic Therapeutics, Inc. $ (4,523,932 ) $ (7,903,093 )
Basic net loss per share attributable to common stock $ (0.01 ) $ (0.02 )
Basic weighted average common stock outstanding 404,396,473 384,075,369

We incurred a lower net loss per basic share of $0.01 for the year ended December 31, 2024 as compared to net loss per basic share of $0.02 for the year ended December 31, 2023. We incurred a significantly lower net loss of approximately $3.4 million between December 31, 2024 and 2023, respectively. The lower net loss was primarily due to lower impairment of goodwill by approximately $2.9 million, lower general and administrative expenses of approximately $0.2 million and lower other expenses, including interest cost, or approximately $0.3 million. All operational costs associated with OT-101 and the nanoparticle platform are substantially covered by the JV, significantly reducing our direct financial burden till such time we make a determination to commence development of our own compounds.

"Our JV is growing rapidly and is moving towards a potential initial public offering, the timing of which is under evaluation. The JV’s product portfolio has the potential to generate significant revenues and value for itself and, consequently, the Company and its shareholders due to our ownership in the JV. That has been the plan for the Company, since we entered into the JV. As reported in our Form 10-K, while we believe that the valuation of the JV has increased, we have opted to revise the fair value of our investment in the JV only upon a triggering event occurring, and justifying the revision to our fair value, such as, but not limited to, a financing, licensing, an IPO, or results of late stage clinical trials such as our Phase 3 pancreatic cancer trial", said Amit Shah, CFO for Oncotelic.

On April 17, 2025 Norgine reported that the Australian Therapeutic Goods Administration (TGA) has approved the registration of IFINWIL (eflornithine) for the treatment of adults and paediatric patients with high-risk neuroblastoma (HRNB), who have responded to prior multiagent, multimodality therapy (Press release, Norgine, APR 16, 2025, View Source [SID1234651964]).

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Neuroblastoma Australia welcomed the news today:

"On behalf of all families of children impacted by neuroblastoma, we welcome the TGA’s decision to approve IFINWIL. We urgently need treatments for children diagnosed with neuroblastoma and this milestone marks a step in the right direction towards a better future for children and their families. We thank the Federal Government for taking action to ensure access and we look forward to continued support for children with aggressive cancers." said Lucy Jones, CEO Neuroblastoma Australia.

High Risk Neuroblastoma is a rare but aggressive form of cancer, predominantly affecting children and most commonly presenting in the first 5 years of life2. Each year in Australia, approximately 50 children are diagnosed with neuroblastoma, with about half of these cases being classified as high risk neuroblastoma3,4. Neuroblastoma originates in the body’s nerve cells (neuroblasts) and typically presents as a primary tumour in the adrenal glands5. It is considered an aggressive tumour because it often spreads to other parts of the body (metastasizes). In most cases, it has spread by the time it is diagnosed5.

"We are committed to improving the lives of children and their families living with high-risk neuroblastoma" said Gus Rudolph, General Manager, Norgine, Australia. "This rare childhood cancer has devastating consequences for those impacted and while more needs to be done to improve treatment outcomes, we would like to recognise the TGA for their work to-date on this approval. Norgine will continue to engage with the relevant stakeholders to bring IFINWIL to patients as quickly as possible."

"This approval, as part of the Project Orbis initiative, represents a vital step forward in ensuring access to innovative cancer treatments for patients around the world," said Dr. David Gillen, Chief Medical Officer, Norgine. "By working collaboratively with international regulatory partners, we are able to help bring promising therapies to paediatric patients sooner – a goal that is especially important when time is critical. We’re proud to support Project Orbis as we strive to expedite access to high-impact oncology medicines."

Receiving the TGA approval is an important milestone on the path to realising sustainable and equitable access. IFINWIL is not currently included on the PBS.

Please refer to the IFINWIL Consumer Medicines Information (CMI) for full safety information on risks, side effects and precautions including the risk of low red blood cells (anaemia), low neutrophils (blood cells that fight infection), low platelets (clotting cells), increase in liver enzymes, and hearing loss or problems balancing.1

Notes to Editors:

About IFINWIL

IFINWIL has been investigated for use as a post maintenance treatment for high-risk neuroblastoma (HRNB) in paediatric patients with no active disease (NAD) / no evidence of disease (NED) after first line multiagent, multimodality therapy.6 IFINWIL is a therapy that blocks an enzyme called ornithine decarboxylase (ODC) responsible for producing polyamines, which are important to tumour growth and development 7.

For more information on IFINWIL, find the CMI here: IFINWIL Consumer Medicine Information (CMI) or the Therapeutic Goods Administration at View Source or speak to your healthcare practitioner.

High-Risk Neuroblastoma (HRNB) Treatment Background

Children diagnosed with HRNB undergo an intense treatment regimen that still leaves them vulnerable to relapse and death.8 Although there have been some improvements in survival, children with high risk neuroblastoma still face a 30% chance of recurrence (relapse) within the first 5 years post maintenance, and have an extremely poor prognosis and low likelihood of long term survival9 (e.g. estimates as low as 15% of patients will live for five years after relapsing).10 Avoiding relapse is key to long-term survival, and until now, there have been no approved therapies for the post maintenance treatment period in major markets outside of the United States11.

About Project Orbis

Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health).

In April 2024, Norgine submitted an application for approval of eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. This milestone supports Norgine’s efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology.