Biopharma.com reported sharp contrasts, with a landmark approval for a rare disease therapy alongside a high-profile trial miss, and a series of corporate moves reflecting market access and financing realities.
The FDA granted full approval to Precigen’s Papzimeos, a first-of-its-kind immunotherapy for recurrent respiratory papillomatosis, a rare and potentially life-threatening condition tied to persistent HPV infections. Approval rested on results showing a little more than half of treated patients did not require surgery within a year. Vinay Prasad, newly returned to the FDA as director of the Center for Biologics Evaluation and Research, framed the decision as evidence that “randomized trials are not always needed to approve medical products.” Cantor Fitzgerald’s Jennifer Kim said that stance should reassure investors wary of tighter standards; Precigen’s shares rose on the news.
Pfizer, meanwhile, reported that inclacumab, acquired via its 2022 purchase of Global Blood Therapeutics, failed to meet the primary goal in a Phase 3 sickle cell disease study, not significantly reducing versus placebo the painful crises common in the condition. The company pledged to share analyses “in due course.” The setback follows Pfizer’s 2024 market withdrawal of the GBT-derived sickle cell drug Oxbryta over safety concerns; updates on Oxbryta and the experimental osivelotor are planned when available.
Deal-making continued as Eli Lilly and Superluminal Medicines announced a collaboration to discover and advance small molecule drugs for cardiometabolic diseases and obesity, targeting undisclosed GPCRs. Lilly will receive exclusive rights to resulting compounds, while Superluminal could earn up to $1.3 billion in total payouts, including an upfront payment and an equity investment.
Market access pressures surfaced at Autolus Therapeutics, which is delaying the European launch of its approved leukemia cell therapy Aucatzyl while assessing pricing and feasibility. The Germany launch is on hold, and Autolus does not anticipate EU sales in 2025 or 2026. In the U.K., a cost-effectiveness body has initially declined to fund the therapy, though Autolus aims to find a path to access. Approved by the FDA in November 2024, Aucatzyl generated just under $30 million in U.S. sales in the first half of 2025.
Finally, Generation Bio reported preclinical evidence that its delivery technology can transport nucleic acid payloads into T cells, but initiated a strategic review citing funding constraints, planning to lay off roughly 90% of its workforce, including all R&D staff, by October’s end. Shares climbed 60%, though remain well below 2020 IPO levels.
