NATIONAL INSTITUTES OF HEALTH

Aristotle's early animal dissections evolved into standard drug development practices by the late 1930s, leading to the 1938 Federal Food, Drug, and Cosmetic Act. Despite animal testing's contributions to medicine, 95% of drugs fail post-testing. Thomas Hartung's research at Johns Hopkins Center for Alternatives to Animal Testing advocates for AI and organoid cultures to replace animal tests, with some success. The FDA Modernization Act of 2023 no longer requires animal testing before clinical trials, and collaborations like CAAT's with the FDA aim to advance nonanimal testing methods. However, challenges remain in replacing animal models entirely due to technological limitations and systemic resistance.

Spark Biomedical partners with Battelle and Hazelden Betty Ford to develop an AI algorithm for detecting opioid withdrawal symptoms and cravings, funded by a $318,875 SBIR grant from NIDA. Phase I focuses on data collection and AI algorithm development, while Phase II aims to integrate the algorithm with Spark's FDA-cleared tAN® device, Sparrow® Ascent, for closed-loop neurostimulation treatment.

The FDA approved Johnson & Johnson’s Tremfya for treating adults with moderately to severely active ulcerative colitis, marking the first fully-human, dual-acting monoclonal antibody targeting IL-23 and CD64. Tremfya showed significant clinical and endoscopic improvements, with 50% and 45% of patients achieving clinical remission at 200 mg and 100 mg doses, respectively, compared to 19% in the placebo group. Common adverse events included respiratory tract infections, injection site reactions, and arthralgia.

Researchers at Baylor College of Medicine identified two B cell abnormalities in triple negative breast cancer patients, which could serve as blood biomarkers for predicting response to chemotherapy and immunotherapy. TiBA-0 patients showed no B cell changes, while TiBA-1 and TiBA-2 exhibited reduced and immature B cells, respectively, leading to poorer treatment responses. These systemic immune changes can be detected via a blood draw, potentially aiding in patient stratification for tailored treatments.

The ALS Bucket Challenge, now 10 years old, continues to raise awareness and funds for ALS research. The VCU Health ALS Clinic, the largest in Virginia, has tripled in size and is involved in major research studies. Since the challenge, three FDA-approved medications for ALS have been developed, and awareness has significantly increased. The clinic's care plan includes multidisciplinary care, access to therapies, and clinical research. The VCU Health ALS Clinic aims to expand its research and diagnostic programs, continuing the fight until a cure is found.

Sinopia Biosciences awarded $2.2M Phase II SBIR grant from NIDCR to advance oral mucositis program using LEADS® platform, combining AI/ML and high-throughput screening for therapeutic target identification.

Aura Biosciences' new therapy, bel-sar, for early-stage choroidal melanoma shows promise in Phase II trials, potentially offering a vision-preserving alternative to radiation therapy. The treatment uses virus-drug conjugates activated by light to target tumors, with results indicating high efficacy and safety. Aura plans to advance to Phase III trials and explore broader cancer applications.

Ractigen Therapeutics receives FDA orphan drug designation for RAG-18, a saRNA for DMD/Becker muscular dystrophy. Biogen's nusinersen shows higher dose efficacy in SMA. Cartesian Therapeutics initiates Descartes-15 CAR-T phase 1 trial for r/r multiple myeloma. Imugene's azer-cel CAR-T produces complete responses in r/r diffuse large B cell lymphoma. Novartis licenses Voyager Therapeutics' capsid for neurological target. BridgeBio Pharma's BBP-812 receives FDA RMAT designation for Canavan disease. Genprex plans to spin off its diabetes program into NewCo. NCI awards TransCode $2 million for TTX-MC138 RNA therapy in advanced solid tumors.

Kintara Therapeutics provides corporate and REM-001 clinical study updates, including a merger agreement with TuHURA Biosciences and a special stockholder meeting on September 20, 2024. The REM-001 study for cutaneous metastatic breast cancer has enrolled four of ten needed patients, with no safety issues identified.