Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
FDA to outline new pathway for patient-specific gene therapies
The successful use of a custom CRISPR therapy to treat baby Kyle Patrick “KJ” Muldoon Jr.’s rare metabolic disorder is prompting the FDA to create a new approval pathway for highly individualized treatments. Vinay Prasad, Director of the agency’s Center for Biologics Evaluation and Research, said the forthcoming “plausible mechanism pathway” will be outlined soon in the New England Journal of Medicine and could allow approval after trials with as few as five patients, according to Endpoints News. The initiative follows growing demand for bespoke therapies and comes alongside the FDA’s new Rare Disease Evidence Principles and its National Priority Voucher pilot program. – Bree Foster
Eli Lilly launches TuneLab to give biotechs access to AI drug discovery models
Eli Lilly has launched TuneLab, an artificial intelligence (AI) platform that gives biotechnology companies access to drug discovery models built on more than $1 billion worth of proprietary research data. The system, trained on decades of Lilly’s preclinical, safety, and drug disposition datasets covering hundreds of thousands of molecules, is designed to help smaller biotechs accelerate drug development without directly exposing their own data. Partners provide training data in exchange for access, with the platform using federated learning to protect confidentiality. TuneLab is part of Lilly’s Catalyze360 initiative, which also offers venture funding, lab space, and development expertise to early-stage firms. The effort could ease barriers for smaller drug developers that lack the resources to build large-scale AI systems. – Andrea Corona
Rapport’s epilepsy drug sends stock to all-time high
On Monday, Rapport Therapeutics announced results from their Phase 2a trial of RAP-219, a first-in-class anti-epilepsy drug that targets the AMPA (alpha-amino-3-hydroxyl-5-methyl-4-isoxazole-propionic acid) receptor. The trial included 30 patients with focal onset seizures — which originate in one specific area of the brain — who also had an implanted responsive neurostimulation system (RNS). Across eight weeks, patients showed a 77.8 percent reduction in seizures, and 24 percent even reached complete seizure freedom for the duration of the study. Following the announcement, Rapport’s stock rose up 160 percent. “This trial represents the first time a novel antiseizure medication was evaluated in focal seizure patients using the RNS system with an objective biomarker of seizure activity. The magnitude of the reduction in clinical seizure frequency seen in this trial, and the corroboration of the clinical activity from the objective biomarker, give me confidence that a medication like RAP-219 has the potential to be a highly effective [anti-seizure medication] for drug-resistant focal seizure patients,” said Jacqueline French, a neurologist and principal investigator of the study at NYU Langone Health's Comprehensive Epilepsy Center. – Allison Whitten
Novartis adds innovative therapy to fight heart disease
Novartis will acquire Tourmaline Bio in a $1.4 billion deal, strengthening its cardiovascular portfolio with the addition of pacibekitug, a late-stage anti-IL-6 (interleukin-6) monoclonal antibody. Pacibekitug is designed to target systemic inflammation, a key driver of atherosclerotic cardiovascular disease (ASCVD), and recently delivered encouraging Phase 2 results which reduced hs-CRP levels — a key marker of inflammation — by up to 86 percent with safety comparable to placebo. “With no widely adopted anti-inflammatory therapies currently available for cardiovascular risk reduction, pacibekitug represents a potential breakthrough in addressing residual inflammatory risk in ASCVD," said Shreeram Aradhye, President, Development and Chief Medical Officer at Novartis. Designed for low-volume, subcutaneous injection as infrequently as once every three months, pacibekitug could help address a major unmet need in a disease that remains a leading cause of death worldwide. Novartis will pay $48 per share for all outstanding Tourmaline stock, with the transaction expected to close in the fourth quarter of 2025 pending regulatory approvals. – Bree Foster
Novo Nordisk to cut 9,000 jobs in transformation plan to reinvest for growth
Novo Nordisk, the Danish drugmaker behind blockbuster obesity and diabetes treatments, will eliminate about 9,000 positions worldwide, including 5,000 in Denmark, in a restructuring designed to simplify its organization and cut costs after years of rapid expansion. The company expects the job cuts to generate around 8 billion Danish krone (DKK; $1.2 billion) in annual savings by 2026, which will be redirected toward research, commercial activities, and manufacturing for its diabetes and obesity businesses. The overhaul will carry one-off charges of about DKK 8 billion this year, prompting Novo Nordisk to lower its 2025 operating profit growth forecast to four to 10 percent at constant exchange rates, down from 10–16 percent. – Andrea Corona
Eli Lilly’s blood cancer drug could become first-line treatment
In the Phase 3 BRUIN CLL-313 trial, Eli Lilly’s Jaypirica was shown to improve progression-free survival in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who had not yet received treatment. Their drug is the first and only reversible Bruton tyrosine kinase (BTK) inhibitor available on the market, and it was approved in 2023 to treat CLL/SLL patients with at least two prior lines of therapy. Lilly called the new results “one of the most compelling effect sizes ever observed for a single agent BTK inhibitor in a front-line CLL study,” and reported that they plan to seek label expansions for Jaypirica to be used as a first-line treatment. – Allison Whitten
