On September 2, 2025 Hoth Therapeutics, Inc. (NASDAQ: HOTH), a clinical-stage biopharmaceutical innovator, reported combined positive findings from multiple preclinical programs evaluating its precision antisense candidate HT-KIT, including compelling anti-tumor efficacy, a clean safety profile, and new GLP-validated bioanalytical results that exceeded internationally recognized regulatory thresholds (Press release, Hoth Therapeutics, SEP 2, 2025, View Source [SID1234655665]).

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Preclinical Efficacy & Safety Highlights

Rapid Tumor Kill: HT-KIT triggered significant tumor cell death in preclinical models of gastrointestinal stromal tumors (GIST) and systemic mastocytosis as early as 24 hours post-treatment, with statistically significant tumor shrinkage observed by day 8.
Strong KIT Suppression: In vitro, HT-KIT achieved over 80% knockdown of KIT expression, the oncogenic driver in multiple aggressive cancers.
Clean Safety Profile: Multi-dose in vivo studies confirmed no off-target toxicity across critical organs including liver, kidneys, spleen, bone marrow, and thymus.
Dose-Dependent Biological Signal: A preclinical safety study demonstrated proportional liver engagement (increase from 1.11g to 1.32g at 3.0 mg/kg) with zero gross pathology at any organ site.
GLP-Validated Bioanalytical Results

The study, conducted by Altasciences Company, Inc. under OECD, FDA, and EMA GLP standards, demonstrated that HT-KIT meets or exceeds strict bioanalytical benchmarks:

Regulatory-Grade Validation: All calibration curve, quality control, and dilution integrity requirements passed with high reproducibility.
Superior Data Integrity: 90.5% of Incurred Sample Reanalysis (ISR) values fell within ±30%, well above the 66.7% regulatory minimum.
Extended Stability: HT-KIT remained stable in serum for 37 days at -80°C, surpassing the validated 28-day stability period, with further studies ongoing.
Flawless Compliance: No protocol or SOP deviations impacted study reliability.
"These results combine a rare and powerful story — tumor kill within 24 hours, clean safety across all systems, and GLP-validated reproducibility beyond regulatory standards," said Robb Knie, CEO of Hoth Therapeutics. "We believe HT-KIT has the potential to transform outcomes in KIT-driven cancers, and these milestones accelerate our path toward IND submission and first-in-human trials."

Next Steps

Hoth expects to integrate this bioanalytical data into its formal GLP toxicology package and is preparing for an IND.

About HT-KIT

HT-KIT is a precision antisense oligonucleotide designed to silence mutant KIT mRNA, an oncogenic driver in gastrointestinal stromal tumors (GIST), systemic mastocytosis, and certain leukemias. By targeting KIT expression at the genetic level, HT-KIT seeks to overcome resistance to tyrosine kinase inhibitors while minimizing systemic side effects.

On September 2, 2025 Agilent Technologies Inc. (NYSE: A) reported that its MMR IHC Panel pharmDx (Dako Omnis) has received class C companion diagnostic (CDx) certification under EU in vitro diagnostic regulation (IVDR)1 as a CDx test for colorectal cancer (Press release, Agilent, SEP 2, 2025, View Source [SID1234655681]). MMR IHC Panel pharmDx (Dako Omnis) is indicated as an aid to identify mismatch repair (MMR) deficient CRC patients eligible for treatment with OPDIVO (nivolumab) in combination with YERVOY (ipilimumab). MMR IHC Panel pharmDx (Dako Omnis) is approved for exclusive use with the Agilent Dako Omnis automated staining solution.

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The MMR pathway corrects DNA replication errors to maintain genomic stability2. Dysfunction in key MMR proteins (MLH1, PMS2, MSH2, and MSH6) causes MMR deficiency (dMMR), leading to elevated mutations, tumorigenesis, and neoantigen accumulation—features that make dMMR tumors more responsive to immunotherapy due to enhanced immune recognition3.

MMR IHC Panel pharmDx (Dako Omnis) is an immunohistochemical panel specifically developed and validated to detect the loss of function of any of the four MMR proteins in formalin-fixed paraffin-embedded colorectal cancer tissue. Agilent’s panel is the only companion diagnostic IHC panel IVDR approved to diagnose colorectal cancer patients eligible for treatment with OPDIVO in combination with YERVOY.

Nina Green, vice-president and general manager of Agilent’s Clinical Diagnostics Division remarked: "The approval of MMR IHC Panel pharmDx will provide physicians in Europe with critical information to inform treatment decisions for patients with mismatch repair deficiency (dMMR). This endorsement underscores Agilent’s leadership in the development of companion diagnostics for groundbreaking therapies."

Agilent collaborated with Bristol Myers Squibb Company to develop the MMR IHC Panel pharmDx (Dako Omnis).

OPDIVO and YERVOY are registered trademarks of Bristol-Myers Squibb Company.

On September 2, 2025 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported a global licensing and collaboration agreement with Novartis for ARO-SNCA, Arrowhead’s preclinical stage siRNA therapy against alpha-synuclein for the treatment of synucleinopathies, such as Parkinson’s Disease, and for other additional collaboration targets that will utilize Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform (Press release, Arrowhead Research Corporation, SEP 2, 2025, View Source [SID1234655634]). Upon closing, Arrowhead will receive $200 million as an upfront payment and is eligible to receive up to $2 billion in potential milestone payments plus royalties on commercial sales.

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"With a robust neuroscience pipeline and clear commitment to neurodegenerative diseases and genetic medicines, Novartis is a compelling partner for Arrowhead in the CNS space," said Christopher Anzalone, Ph.D., President and CEO at Arrowhead. "Our TRiMTM platform has generated impressive preclinical results demonstrating delivery to CNS, including distribution to deep brain regions, after subcutaneous administration. The potential translation of these results in upcoming clinical trials would represent an important leap forward for neurodegenerative diseases and gene targets in the CNS that have been historically difficult to address. We look forward to working with Novartis to bring ARO-SNCA for the treatment of synucleinopathies, such as Parkinson’s Disease, into clinical trials as soon as possible and to collaborate on additional programs in the future."

Fiona Marshall, Ph.D., President of Biomedical Research at Novartis added, "Neurodegenerative conditions such as Parkinson’s Disease affect millions of patients worldwide. Novartis aspires to transform the lives of patients and families living with these diseases, by advancing medicines that significantly alter the course of the disease. We believe that one way to effectively target core drivers in Parkinson’s and other neurodegenerative diseases requires completely novel approaches to deliver RNA medicines to the brain. We see Arrowhead’s TRiMTM technology as having great potential to achieve the type of widespread and effective delivery in key brain structures that will be necessary to see the full benefit of RNA medicines in neurodegeneration."

Summary of License and Collaboration Agreement
Under the terms of the agreement, Novartis will receive an exclusive worldwide license to research, develop, manufacture, and commercialize ARO-SNCA, a preclinical stage program that utilizes Arrowhead’s TRiMTM platform for subcutaneous administration and delivery to the CNS designed to target the gene that encodes the alpha-synuclein protein as a potential treatment for patients with Parkinson’s Disease, and other synucleinopathies. Novartis will select additional collaboration targets outside of Arrowhead’s current pipeline to be developed using the TRiMTM platform.
For all licensed programs under the agreement, Arrowhead will conduct and complete preclinical research activities necessary to enable a clinical trial application (CTA) filing. Novartis will then assume sole control over development, manufacturing, medical affairs, and commercialization activities.

Summary Financial Terms
Upon closing, Novartis will make a $200 million upfront payment to Arrowhead. Arrowhead is also eligible to receive development, regulatory, and sales milestone payments of up to $2 billion. Arrowhead is further eligible to receive tiered royalties on commercial sales up to the low double digits.
The transaction is expected to close in the second half of 2025, subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions.
Gibson, Dunn & Crutcher LLP is serving as legal advisor to Arrowhead.

On September 2, 2025 Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies, reported its participation in the following investor conference (Press release, Nkarta, SEP 2, 2025, View Source [SID1234655651]):

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H.C. Wainwright 27th Annual Global Investment Conference
September 9, 2025
8:30 a.m. ET – fireside chat

A simultaneous webcast of the event will be available on the Investors section of Nkarta’s website, www.nkartatx.com, and a replay will be archived on the website for approximately 90 days.

On September 2, 2025 Whitehawk Therapeutics, Inc. (Nasdaq: WHWK), an oncology therapeutics company applying advanced technologies to established tumor biology to efficiently deliver improved ADC cancer treatments, reported that the Company’s president and CEO, Dave Lennon, PhD, will participate in a fireside chat during the following upcoming investor conferences in New York City, NY (Press release, Whitehawk Therapeutics, SEP 2, 2025, View Source [SID1234655668]):

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Morgan Stanley 23rd Annual Global Healthcare Conference on Monday, September 8, 2025. Fireside chat at 4:50 PM ET.

H.C. Wainwright 27th Annual Global Investment Conference on Tuesday, September 9, 2025. Fireside chat at 3:30 PM ET.
A live webcast of the fireside chat events can be accessed by visiting the Whitehawk Therapeutics IR website. These will be available for replay for approximately 30 days following the event.