On July 7, 2025 PanTher Therapeutics ("PanTher" or the "Company"), a clinical-stage company redefining cancer treatment with therapeutics administered continuously and exclusively at the tumor site, reported that the first patient has been treated with PTM-101 in a Phase 1b clinical trial in pancreatic ductal adenocarcinoma (PDAC) (Press release, PanTher Therapeutics, JUL 7, 2025, View Source [SID1234654271]). PTM-101 is the most advanced product candidate within PanTher’s portfolio of innovative formulations for continuous, high-dose, localized drug administration directly to the site of the tumor.

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"The start of this second clinical trial of PTM-101 is an exciting milestone in our journey to transcend the dosing limitations of today’s cancer treatments by reimagining how chemotherapy is delivered," said Laura Indolfi, Ph.D., Chief Executive Officer and Co-founder of PanTher Therapeutics. "Powerful cancer drugs exist but their toxicity lowers maximum dose and limits dosing frequency — leaving too many opportunities for cancers to continue spreading while patients grapple with debilitating side effects. Our investigational formulations are designed to circumvent the toxic effects of systemic chemotherapy while retaining a much higher dose of the drug exclusively at the tumor, with the goal of shrinking difficult-to-treat tumors and extending patients’ lives."

PTM-101 is a polymeric thin film formulation of paclitaxel, a well-established chemotherapy drug, designed to deliver a sustained (~6 weeks) high dose to the tumor site with little to no systemic exposure. A previous first-in-human Phase 1 study (ACTRN12621000881831) of PTM-101 at the 100 mg dose level, combined with standard of care chemotherapy in borderline resectable and locally advanced PDAC, reported promising tumor shrinkage and a favorable safety profile. No paclitaxel was detected systemically at any time. The study additionally demonstrated PTM-101’s ability to fit into current PDAC treatment protocols and deliver potential therapeutic benefit early in the clinical paradigm, beginning weeks before intravenous chemotherapy.

The ongoing dose escalation and expansion Phase 1b study (NCT06673017) is assessing safety, tolerability, and anti-tumor activity of PTM-101 at two higher dose levels when combined with standard of care neoadjuvant chemotherapy (FOLFIRINOX) in patients with borderline resectable or locally advanced PDAC. The first patient was dosed at Virginia Mason Medical Center in Seattle, Washington, under Drs. Vince Picozzi and William "Scott" Helton. In addition to Virginia Mason, trial enrollment is presently ongoing at Northwell Health Zuckerberg Cancer Center in Lake Success, New York; Hoag Memorial Hospital Presbyterian in Newport Beach, California; and the Barbara Ann Karmanos Cancer Institute in Detroit, Michigan. This non-randomized, open-label study plans to enroll approximately 30 treatment-naïve patients across multiple clinical sites in the U.S.

"PTM-101 is a novel, innovative approach to treating the primary pancreatic tumor," said Vince Picozzi, M.D., a medical oncologist and a principal investigator of the Phase 1b clinical trial. "Doing so successfully is the first step towards curative therapy."

Studies have shown that only about 1% of systemically delivered chemotherapy reaches the tumor, with the remaining 99% of drug producing toxic effects on off-target tissues — including neutropenia, hair loss, nausea and vomiting, and peripheral neuropathy. Additionally, pancreatic cancer is notoriously poorly vascularized, making it extremely difficult for systemic chemotherapy to reach therapeutic levels at the tumor site.

"Targeted cancer treatment is an area of immense interest for oncology," said Scott Helton, M.D., a pancreatic surgeon in Seattle, Washington. "The ability of PTM-101 to integrate into our current PDAC care pathway is promising, offering the possibility of transforming a diagnostic step into the start of therapy, weeks before the patient can begin intravenous chemotherapy."

PanTher is additionally developing polymeric drug formulations for the treatment of a range of other solid tumor types.

About PTM-101

PanTher’s most advanced product candidate, PTM-101, is an absorbable thin film formulation of paclitaxel for non-metastatic pancreatic cancer. PTM-101 is designed to deliver continuous, long-term, high-dose chemotherapy to the tumor with little to no systemic exposure. The product, laparoscopically implanted at the tumor site, easily integrates with common minimally-invasive procedures used in staging pancreatic cancer. PTM-101 is currently being evaluated in a Phase 1b clinical trial (NCT06673017) with support from the Cancer Prevention & Research Institute of Texas (CPRIT) DP220066.

On July 7, 2025 FUJIFILM Pharmaceuticals U.S.A., Inc., drug development center and a leading provider of Contract Development and Manufacturing Organization (CDMO) services for drug delivery system (DDS) technologies, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Fujifilm’s FF-10832 — an investigational liposomal formulation of gemcitabine — for the treatment of biliary tract cancer (BTC) (Press release, Fujifilm, JUL 7, 2025, View Source [SID1234654270]). Phase 1 study (NCT03440450) results presented at ASCO (Free ASCO Whitepaper) 2025 suggest FF-10832 is well tolerated and has anti-tumor activity in patients with advanced BTC. FF-10832 is currently being evaluated in phase 2a studies (NCT05318573) as monotherapy or in combination with pembrolizumab for the treatment of solid tumors in the U.S.

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The FDA grants orphan drug designation to support the development of therapies for rare diseases that affect fewer than 200,000 people in the U.S.1 Small patient populations can limit preclinical research and slow clinical trials, stalling progress in therapeutic development for rare diseases. The designation supports research and clinical development by providing 7-years marketing exclusivity and other financial incentives.

There are approximately 16,000 new cases of BTC in the U.S per year.2 Most (≥70%) patients present with unresectable or metastatic disease at diagnosis, and current treatments of surgery, chemotherapy, and radiation show limited efficacy in advanced stages. High recurrence (50–70%) and low 5-year survival rates (4–13%) highlight the urgent need for new and effective treatments.

Gemcitabine has been a key component of BTC treatment since its approval in the 1990s, with all major first line regimens currently containing gemcitabine.3 FF-10832’s novel liposomal formulation of gemcitabine for intravenous administration is designed to enhance anti-tumor activity by prolonging plasma half-life and improving targeted delivery to tumors.

"BTCs are rare but aggressive malignancies associated with a poor prognosis and limited treatment options," said Susumu Shimoyama, president, FUJIFILM Pharmaceuticals U.S.A., Inc. "Receiving orphan drug designation highlights the significant unmet medical need that still remains and supports development of FF-10832 for patients with BTC who have few satisfactory options."

FF-10832 is manufactured by FUJIFILM Toyama Chemical, providing seamless CDMO end-to-end services for full integration from formulation development to GMP manufacturing. Fujifilm’s investigational drug candidates FF-10502 and FF-10850 have also been granted orphan drug designations for cholangiocarcinoma and Merkel cell carcinoma, respectively.

On July 7, 2025 Sirtex Medical ("Sirtex"), a leading manufacturer of interventional oncology solutions, reported that the U.S. Food and Drug Administration (FDA) approved SIR-Spheres Y-90 resin microspheres for the treatment of unresectable hepatocellular carcinoma (HCC) in the United States (Press release, Sirtex Medical, JUL 7, 2025, View Source [SID1234654269]). With this approval, SIR-Spheres is the only radioembolization therapy approved for the treatment of both metastatic colorectal cancer (mCRC) of the liver and HCC in the U.S.

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HCC is the most common form of liver cancer in adults in the U.S., according to the American Cancer Society. Radioembolization–commonly referred to as selective internal radiation therapy (SIRT)–with SIR-Spheres uses personalized dosimetry to deliver the optimal dose of radiation directly to tumors in patients with HCC. This approval gives clinicians expanded flexibility in selecting a liver-directed therapy that aligns with patient-specific needs and treatment goals.

"The expanded indication makes SIR-Spheres the only Y-90 treatment approved in the U.S. for both HCC and mCRC," said Matt Schmidt, CEO of Sirtex. "This milestone reflects our ongoing commitment to delivering flexible, personalized therapies—with multiple dose options available daily—that empowers physicians to treat patients when and where it works best."

This regulatory milestone is supported by results from the DOORwaY90 study, a prospective, multicenter, open-label clinical trial evaluating the safety and efficacy of SIR-Spheres in treating HCC. The study enrolled 100 patients across 18 U.S. centers, with 65 patients included in the interim primary efficacy cohort. DOORwaY90 met its prespecified co-primary endpoints, demonstrating a best overall response rate (ORR) of 98.5% as assessed by independent central review. All evaluable patients demonstrated a response, indicating a 100% local tumor control rate. Additionally, the median duration of response (DoR) exceeded 300 days. These findings highlight SIR-Spheres as a highly effective liver-directed therapy with a favorable safety profile.

"This study moves the field of radioembolization forward with reproducible dosimetry outcomes and a strong safety profile linked to very positive clinical results," said Dr. Armeen Mahvash, Interventional Radiologist at MD Anderson Cancer Center and Co-Principal Investigator of the DOORwaY90 Study. "This will give multidisciplinary care teams the confidence to recommend SIR-Spheres for HCC treatment."

On July 7, 2025 Lutris Pharma, a clinical stage biopharmaceutical company focused on improving anti-cancer therapies by reducing cutaneous dose limiting toxicities, reported the presentation of results from its double-blind, placebo-controlled phase 2 randomized clinical trial of lead compound, LUT014 gel (Press release, Lutris Pharma, JUL 7, 2025, View Source [SID1234654268]). The topically-applied novel B-Raf inhibitor is optimized for paradoxical MAPK activation, for use by patients treated with epidermal growth factor receptor (EGFR) inhibitor therapy who develop dose-limiting acneiform rash. The clinical data was presented by Dr. Ofer Purim, Head of Gastrointestinal Malignancy Unit at the Helmsley Cancer Center, Shaare Zedek Medical Center, Jerusalem, Israel, in an oral presentation, entitled, "A double-blind placebo-controlled randomized phase 2 clinical trial to assess the efficacy of a topical BRAF inhibitor for acneiform rash toxicities from anti-EGFR therapies," during a Proffered Paper Session at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Gastrointestinal Cancers Congress 2025.

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"EGFR inhibitors, like cetuximab and panitumumab, are critical components of cancer therapy, but their effectiveness is often compromised by skin toxicities that can lead to dose modifications or treatment discontinuation," said Benjamin W. Corn, M.D., Chief Medical Officer of Lutris Pharma. "LUT014 is designed to mitigate these toxicities by restoring downstream signaling in skin cells disrupted by EGFR inhibition. In our clinical trial, the high dose of LUT014 demonstrated statistically significant efficacy in improving acneiform rash across both cetuximab and panitumumab-treated patients. Additionally, encouraging results were observed in the open-label extension, where even the low dose of LUT014 showed meaningful efficacy, with up to 69% of patients in the per-protocol population achieving improvement. LUT014 was also well tolerated, with fewer and mostly mild adverse events compared to placebo. We believe these results underscore LUT014’s potential to be a key therapeutic, offering significant benefits to patients by enabling them to remain on optimal anti-EGFR treatment without interruption."

"This is the first placebo-controlled, randomized trial to demonstrate both efficacy and safety of a treatment for anti-EGFR-induced acneiform rash, a major advancement in our effort to become the first company to provide a therapeutic that addresses the dose-limiting side effects of cancer treatments," added Sumant Ramchandra, M.D., Ph.D., Chief Executive Officer of Lutris Pharma. "Following its initial recognition at the AACR (Free AACR Whitepaper) Annual Meeting, the selection of our LUT014 data for oral presentation at the ESMO (Free ESMO Whitepaper) Gastrointestinal Cancers Congress further validates the clinical importance and growing momentum behind our lead program. Importantly, patients who respond best to EGFR inhibitor therapy are often the ones who experience the most severe cutaneous side effects, underscoring the urgent need for an effective solution in this underserved market. Our successful $30 million financing in January 2025 has enabled us to accelerate development of LUT014 and advance toward commercialization. Currently, there is no accepted standard of care or approved drugs for EGFR inhibitor associated cutaneous toxicity. We are hopeful that the ongoing development of LUT014 will be able to provide for this significant unmet medical need. As such, Lutris is well-positioned to help patients remain on life-saving cancer treatments, enhancing outcomes and dramatically improving quality of life."

The trial enrolled 118 colorectal cancer patients from 23 clinical sites, all of whom had developed grade 2 or non-infected grade 3 acneiform rash while receiving cetuximab or panitumumab. Participants were randomized in a 1:1:1 ratio to receive either LUT014 gel 0.03%, LUT014 gel 0.1%, or a placebo gel. The gel was applied daily for 28 days.

The primary endpoint was the proportion of patients who achieved treatment success, measured by an improvement of at least one grade in Common Terminology Criteria for Adverse Events (CTCAE) scoring or an improvement of at least 5 points in the Functional Assessment of Cancer Therapy (FACT)-EGFRI-18 HRQoL skin-specific assessment. The study employed both an intention-to-treat (ITT) analysis and a Per-Protocol (PP) analysis (i.e., patients who dropped out or did not discontinue their EGFR inhibitor for reasons unrelated to the rash, such as disease progression were excluded from the analysis). Sample size calculation was based on an expected treatment success of 20% for the placebo group and 50% for one of the treatment groups. A total of 117 patients were required for a two group ꭕ2 test with a 0.05 two-sided significance and 80% power.

Efficacy is shown in the table below. The high dose of LUT014 demonstrated statistically significant rates of success improving acneiform rash in both the cetuximab and panitumumab ITT groups, compared to placebo. Additionally, patients randomized to LUT014 gel had lower rates of interruption of cetuximab or panitumumab therapy due to acneiform rash.

N

Success Rate

P value

Panitumumab

High dose LUT014 (0.1%)

28

64.3 %

0.028

Low dose LUT014 (0.03%)

21

47.6 %

0.321

Placebo

27

33.3 %

Cetuximab

High dose LUT014 (0.1%)

11

81.8 %

0.021

Low dose LUT014 (0.03%)

19

47.4 %

0.448

Placebo

12

33.3 %

About EGFR Inhibitor-Induced Rash
EGFR is a receptor on the surface of cells which is expressed in many normal epithelial tissues, including skin. The EGFR signaling pathway is one of the key pathways that regulate growth, survival, proliferation, and differentiation of cells. B-Raf is a protein encoded by the BRAF gene and is a downstream effector component of the EGFR signaling pathway. EGFR has been shown to be over-activated in various human cancers, including colorectal, lung, head and neck, urinary bladder, pancreatic and breast cancers, eliciting downstream phosphorylation and activation of the MAP Kinase pathway.

EGFR inhibitors can block the EGFR signal responsible for cell growth. Among the various types of pharmacological therapies for cancer, EGFR inhibitors are increasingly being used both as primary therapy as well as in patients who have progressed on prior chemotherapy treatments. Although effective as anti-cancer therapy leading to tumor shrinkage, EGFR inhibitors have many adverse reactions associated with their use. The majority of patients treated with EGFR inhibitors will experience adverse dermatological side effects typically manifested as a papulopustular skin rash, also known as acneiform lesions, which can impact quality of life and affect adherence to therapy.

About LUT014
LUT014 is a novel B-Raf inhibitor which is applied topically to the skin. When the B-Raf protein is mutated, as is the case in some human cancers such as melanoma, blocking this pathway leads to apoptosis of the cells and tumor shrinkage. However, when the same pathway is blocked in normal, non-mutated cells, the opposite happens: the MAPK pathway is activated, and cells start growing. This phenomenon is recognized as the paradoxical effect of B-Raf Inhibitors. LUT014 harnesses the paradoxical effect of B-Raf Inhibitors in order to enhance cell proliferation and balance cell destruction, typical to radiation dermatitis.

On July 7, 2025 Myosin Therapeutics, a clinical-stage biotechnology company advancing first-in-class therapies targeting molecular motor proteins, reported the publication of two major papers in the journal Cell that establish non-muscle myosin II (NMII) as a druggable target with broad therapeutic implications (Press release, Myosin Therapeutics, JUL 7, 2025, View Source [SID1234654267]).

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The first article in Cell, "Development of Clinically Viable Non-Muscle Myosin II Small Molecule Inhibitors," details the optimization and development of a new pharmacological class of small molecule inhibitors of the enzyme NMII, an actin-binding motor protein that regulates cellular functions such as division, synaptic remodeling, and immune evasion. The study describes how an iterative structure-based approach overcame historical challenges to yield brain-penetrant small molecules with high specificity and excellent tolerability.

The work originated in the lab of neuroscientist Courtney Miller, Ph.D., who teamed up with medicinal chemist, Theodore Kamenecka, Ph.D., and structural biologist, Patrick Griffin, Ph.D. With support from the National Institute of Neurological Disorders and Stroke (NINDS/NIH) Blueprint Neurotherapeutics Program and National Institute for Drug Abuse (NIDA), the three led a program to develop and bring NMII inhibitors to the clinic. The team identified MT-110 as a clinical candidate for the treatment of stimulant use disorder. MT-110 and MT-125, another NMII inhibitor being pursued in oncology, serves as the cornerstone of the company they co-founded, Myosin Therapeutics.

The potential of MT-125 in glioblastoma (GBM) is detailed in the second Cell article, "MT-125 Inhibits Non-Muscle Myosin IIA and IIB and Prolongs Survival in Glioblastoma." GBM is an extremely aggressive, treatment-resistant brain cancer. The team collaborated with Mayo Clinic scientist and neuro-oncologist Steven Rosenfeld, M.D., Ph.D. The research was supported by NINDS and the National Cancer Institute (NCI).

In preclinical GBM models, MT-125 demonstrated robust monotherapy activity, as well as synergy with radiation and FDA-approved oncology therapeutics at suppressing tumor growth and extending survival. The publication provides detailed mechanistic insight into these therapeutic actions and informs MT-125’s clinical development strategy.

"As a clinician treating glioblastoma my entire career, collaboration with Myosin Therapeutics is meaningful," said Dr. Rosenfeld. "There’s great promise that our research will translate into improved outcomes for patients who urgently need them."

Miller said," The work detailed in these articles establishes NMII inhibitors as a new therapeutic class and we’re passionate about using them to tackle big health challenges, starting with glioblastoma and addiction."