On December 18, 2024 Blueprint Medicines Corporation (Nasdaq: BPMC) reported that Kate Haviland, Chief Executive Officer, will present a corporate overview and 2025 outlook at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 9:00 a.m. PT (12:00 p.m. ET) (Press release, Blueprint Medicines, DEC 18, 2024, View Source [SID1234649202]).

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A live webcast of the presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcast will be archived on Blueprint Medicines’ website for 30 days following the presentation.

On December 18, 2024 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, reported that the Safety Review Committee (SRC) monitoring its Phase 1 clinical trial has unanimously approved opening of the third cohort of patients based on its favorable review of Cohort 2 safety data (Press release, TransCode Therapeutics, DEC 18, 2024, https://www.prnewswire.com/news-releases/transcode-therapeutics-announces-safety-review-committee-approval-of-opening-third-cohort-and-preliminary-results-from-first-cohort-in-phase-1-ttx-mc138-clinical-trial-302334447.html [SID1234649203]). The therapeutic candidate being evaluated, TTX-MC138, is TransCode’s lead candidate designed to inhibit microRNA-10b, or miR-10b, a microRNA critical to the emergence and progression of many metastatic cancers. The dose administered to the third cohort will be approximately double the dose administered to the second cohort.

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Several patients in the first and second cohort remain on study for continued treatment. No significant safety or dose limiting toxicities have been reported. Analysis of Cohort 1 data for pharmacokinetic (PK) and pharmacodynamic (PD) activity is ongoing and to date suggests that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from the previous Phase 0 clinical trial. Specifically, results from Cohort 1 confirmed the Phase 0 observation that TTX-MC138 shows evidence of pharmacodynamic activity in the presence of high baseline expression of miR-10b, reaching a 66% inhibition at 24 hours after infusion. Additionally, the concentration of TTX-MC138 in blood plasma as a function of dose in humans was found to be higher than achieved in nonclinical studies, suggesting a favorable pharmacokinetic profile.

"An SRC is a group of clinicians and experts that oversee patient safety during the conduct of a clinical trial. The SRC determines whether and how a study should proceed, including dose escalation and de-escalation decisions in accordance with the study design. The recommendations of the SRC are used to decide whether a clinical trial should be continued as designed, changed, or terminated," commented Sue Duggan, TransCode’s Senior Vice President of Operations. Duggan added, "Enrollment into the study continues based on the cumulative safety data review. Eligible patients may now be screened and scheduled in Cohort 3 for treatment with the next dose level of TTX-MC138 while preliminary data analysis continues."

About TTX-MC138

TTX-MC138 is a first-in-class therapeutic candidate that targets microRNA-10b, a microRNA widely believed to be a driver of metastatic disease. TransCode’s 2023 Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.

About the Trial

TransCode’s Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study designed to generate critical data to support evaluation of the safety and tolerability of TTX-MC138 in patients with a variety of metastatic solid cancers. While not an endpoint, the trial may provide early evidence of clinical activity of TTX-MC138. The trial comprises an initial dose-escalation phase followed by a dose-expansion phase. The primary objective of the dose-escalation phase is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion phase, the safety, tolerability and anti-tumor activity of TTX-MC138 will be further evaluated in certain tumor types selected based on preliminary results from the dose-escalation phase.

Further information is available at www.clinicaltrials.gov NCT Identifier: (NCT06260774).

On December 18, 2024 Inceptor Bio, a leading innovator in cell therapy, and GRIT Bio, a clinical-stage immunotherapy developer, reported a strategic partnership to advance IB-T101, a potentially best-in-class CAR-T therapy targeting solid tumors (Press release, Inceptor Bio, DEC 18, 2024, View Source [SID1234649204]).

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IB-T101, Inceptor Bio’s autologous CD70 CAR-T program, utilizes the proprietary OUTLAST platform to reprogram T cells for superior stemness, durability, and effector function in the hostile tumor microenvironment. The program targets clear cell Renal Cell Carcinoma (ccRCC), a cancer with significant unmet medical need and over 300,000 new cases annually worldwide.

"We are excited to partner with GRIT Bio to bring IB-T101 into the clinic and address the urgent need for effective solid tumor therapies," said Dr. Matthias Schroff, CEO of Inceptor Bio. "This collaboration highlights the potential of our proprietary OUTLAST platform, and the data generated will play a critical role in advancing the program as we strive to deliver best-in-class therapies for patients."

Dr. Mengyang Chong, Chief Business Officer of GRIT Bio, shared, "Partnering with Inceptor Bio allows us to bring a highly differentiated CAR-T program to patients. IB-T101 has the potential to transform the treatment landscape for solid tumors, and we are eager to contribute our expertise in clinical development and manufacturing to accelerate its progress."

Under the terms of the agreement, Inceptor Bio will grant GRIT Bio an exclusive license for IB-T101 in China. GRIT Bio will oversee development, manufacturing, and commercialization. Inceptor Bio is eligible to receive milestone payments and royalties upon IB-T101 achieving certain response criteria and plans to leverage clinical data from this collaboration to support regulatory submissions and development in other regions.

This partnership represents a critical step in Inceptor Bio’s mission to validate the OUTLAST platform as a best-in-class approach for engineered T cell therapies and bring transformative treatments to patients worldwide.

On December 17, 2024 A2A Pharmaceuticals, Inc. ("A2A" or "the company"), a clinical-stage pioneering biopharmaceutical company focused on developing innovative cancer therapies, reported progress in its clinical studies exploring TACC3 (Transforming Acidic Coiled-Coil 3) PPI inhibition in patients with ovarian cancer, triple-negative breast cancer (TNBC), and endometrial cancer (Press release, A2A Pharmaceuticals, DEC 17, 2024, View Source [SID1234649167]).

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A2A has progressed into cohort 4, an important inflection point in its Phase 1 clinical trial to assess the safety and efficacy of its lead TACC3 inhibitor, AO-252. Early results have shown strong safety profiles with the potential for a wide therapeutic index in these patient populations.

"We are excited about the advancements in our TACC3 program and its potential for patients with challenging cancer diagnoses. The company is continually exploring collaborations and partnerships to accelerate development timelines and enhance patient access to cutting-edge treatments," said Sotirios Stergiopoulos, M.D., CEO of A2A.

"While our current focus with AO-252 is on addressing the unmet needs in ovarian, TNBC, and endometrial cancers, we are equally excited about its potential to benefit patients in additional indications in the near future," added Robbin Frnka, Vice President of Clinical Operations."

The selected cancers are characterized by limited treatment options and high rates of recurrence in patients. A2A’s TACC3 PPI inhibitor is designed to disrupt critical cellular mechanisms involved in cancer cell proliferation, providing a much-needed new avenue of treatment for patients battling these aggressive malignancies.

"This trial represents a promising step forward in delivering therapies that could impact the standard of care for patients," said Alex Spira, M.D., principal investigator of Next Oncology Virginia. "A2A Pharmaceuticals shows dedication to supporting the cancer community by advancing novel therapies that target the underlying biology of tumors."

Beyond these indications, pre-clinical studies suggest A2A’s TACC3 PPI inhibitor may also show efficacy in other solid tumors, including gastric, prostate cancers, and sarcomas, etc. Ongoing research efforts are focused on expanding the indication spectrum and optimizing treatment regimens for diverse cancer types.

On December 17, 2024 HighField Biopharmaceuticals, a clinical stage company using lipid-based therapeutics to treat cancer, diabetes and other diseases, reported that the Center for Drug Evaluation of China’s National Medical Products Administration has granted clearance of the company’s Investigational New Drug (IND) application to begin a clinical trial of HF50 in patients with advanced solid tumor cancers (Press release, HighField Biopharmaceuticals, DEC 17, 2024, View Source [SID1234649187]).

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"This is a highly significant milestone for our company and for patients," said Yuhong Xu, Ph.D., CEO and Scientific Founder of HighField. "HF50 is a T cell Redirecting Antibody Fragment-anchored Liposome, or TRAFsome, with two different antibodies attached; the first binds to T cells and the second binds to tumor cells. It is the first-of-its-kind liposomal T cell engager to emerge from our platform of immunoliposomes, and the first to enter clinical trials."

In addition to converting T cells into an army of cancer destroyers, HF50 also carries a payload that releases in the tumor microenvironment to facilitate the anti-tumor immune reactions.

The design of the TRAFsome T cell engagers builds upon the success of HighField’s LipoADCplexTM platform. The first product from this platform is K1, which contains the anti-HER2 antibody for binding to tumor cells and delivering cancer killing drugs. K1 is in clinical trials and has shown very good safety profiles in cancer patients.

"Once we proved the LipoADCplexTM platform could be safer, cheaper and more effective than ADCs (antibody-drug conjugates), the next logical step is to enlist immune cells with a second antibody to enable immunotherapy against the target cells," Dr. Xu explained. "T cell engagers can be very powerful. But over stimulation may result in high risk of toxicity and T cell exhaustion."

The Phase 1 open-label, dose escalation and dose expansion trial of HF50 will evaluate the safety, tolerability, pharmacokinetic characteristics, immunogenicity, and preliminary efficacy in patients with advanced solid tumors. The trial is expect to begin in 1Q2025.

"As a T cell engager, HF50 has advantages over bispecific antibody constructs and even CAR T cells," Dr. Xu said. "The liposomal T cell engagers can integrate three specific activities. The first is to activate T cells; the second is to direct the T cells to target cells; and the third is to release the payload to facilitate the immune responses."

Dr. Xu added, "The TRAFsomes can be made modularly with low costs. Preclinical data suggested they are much safer and more effective. We can also add different payloads to expand their application to other illnesses such as autoimmune diseases and illnesses related to aging."