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2024 saw significant clinical successes and failures in biopharma, with Vertex, BMS, and Alnylam achieving notable wins, while AbbVie, Sage Therapeutics, Pfizer, and Gilead faced setbacks in neurological and other indications. Despite high failure rates in drug trials, particularly in CNS diseases, some companies advanced new treatments, while others faced challenges in their pipelines.

Hyunji Kim, a breast cancer patient, is in a clinical trial for LCB84, an antibody-drug conjugate, which has stabilized her liver metastasis. Dr. Yuan Yuan describes antibody-drug conjugates as designer drugs targeting specific cancer cells. Over 150 such conjugates are in trials, with Kadcyla, Enhertu, and Trodelvy approved for breast cancer. The field offers hope for chemotherapy-resistant diseases, with ongoing research exploring various targets and payloads.

Recent advancements in sequencing targeted therapies are reshaping HR-positive, HER2-negative metastatic breast cancer management, emphasizing the critical role of NGS in personalizing treatment decisions. Key factors include considering CDK 4/6 inhibitors, biomarker assessment, and sequencing new therapies like inavolisib and capivasertib. Trials such as SOLAR-1, INAVO120, and CAPItello-291 highlight improved progression-free survival and tolerable adverse effects. NGS is crucial for identifying PIK3CA/AKT mutations to determine optimal frontline and second-line treatments.

Gilead Sciences invested $20 million in an exclusive option and license agreement with Tubulis to develop novel antibody-drug conjugate therapies for solid tumors, potentially totaling up to $415 million. The partnership aims to leverage Tubulis' Tubutecan platform for ultra-stable ADCs, following Gilead's recent setbacks with Trodelvy in bladder and lung cancer trials.

Abingworth seeks to raise up to $1.5 billion for biopharma clinical development in return for royalties, aiming to support up to eight late-stage trials. This follows earlier deals with Gilead and Teva, where Abingworth funded clinical studies in exchange for fixed payments and royalties on sales.

Kelun-Biotech's sacituzumab tirumotecan (sac-TMT) receives NMPA approval in China for triple-negative breast cancer, competing with Gilead's Trodelvy. The drug, also under review for EGFR-mutant NSCLC, is partnered with MSD, potentially worth up to $1.4 billion. Phase 3 study results showed a 47% reduction in death risk.

Troldelvy (sacituzumab govitecan), a triple-negative breast cancer treatment, faces re-deliberation by HIRA’s PREC after Gilead Sciences Korea submitted an additional financial sharing plan. The drug, which passed CDRC in 2023, failed to meet ICER thresholds but has garnered significant attention as a breakthrough treatment. Over 100,000 petitions support its reimbursement, reflecting high hopes among patients and medical professionals.

David H. Aggen, MD, PhD, discusses the challenge of personalizing frontline treatment for urothelial cancer patients living over 2 years, highlighting the FDA approvals of enfortumab vedotin-ejfv plus pembrolizumab and nivolumab plus cisplatin and gemcitabine. Aggen emphasizes the need for biomarker research to guide treatment selection and mitigate toxicity, noting the potential use of circulating tumor DNA (ctDNA) to tailor therapy.

IDEAYA Biosciences appoints Stu Dorman as Chief Commercial Officer, leveraging his 20+ years of oncology commercialization experience from Gilead Sciences and Bristol Myers Squibb, including successful launches of Trodelvy® and Opdivo®.