FDA Approves Precigen's Papzimeos as Pfizer's Sickle Cell Drug Falters

In a week of significant developments for the pharmaceutical industry, the FDA granted approval to a groundbreaking immunotherapy while a major sickle cell disease treatment failed to meet its primary endpoint in a late-stage trial.

Precigen's Papzimeos Gains FDA Approval

The U.S. Food and Drug Administration has given full approval to Precigen's Papzimeos, a first-of-its-kind immunotherapy for recurrent respiratory papillomatosis (RRP). This rare and potentially life-threatening condition is caused by persistent HPV infections. The approval was based on study results showing that over half of the patients treated with Papzimeos did not require surgery within a year of therapy.

Dr. Vinay Prasad, who recently rejoined the FDA as the director of the Center for Biologics Evaluation and Research, made a notable statement regarding the approval. He described it as evidence that "randomized trials are not always needed to approve medical products." This comment has been interpreted by some analysts, including Jennifer Kim of Cantor Fitzgerald, as a potentially reassuring sign for biotech investors concerned about stricter regulatory standards under Prasad's leadership.

Pfizer's Sickle Cell Drug Fails in Phase 3 Trial

In contrast to Precigen's success, Pfizer announced that its experimental drug for sickle cell disease, inclacumab, failed to meet its goal in a Phase 3 study. The drug, which Pfizer acquired through its 2022 buyout of Global Blood Therapeutics, did not significantly reduce the pain crises experienced by sickle cell patients compared to a placebo.

This setback follows Pfizer's decision last year to withdraw another sickle cell drug, Oxbryta, from the market due to safety concerns. Oxbryta was also obtained through the Global Blood Therapeutics acquisition. Pfizer has stated that it will share analyses of the inclacumab data with the scientific and patient community in due course and plans to provide updates on Oxbryta and a third Global Blood drug, the experimental osivelotor, when available.

Industry Collaborations and Strategic Moves

Amid these clinical developments, the pharmaceutical industry continues to see strategic collaborations and corporate restructuring:

• Eli Lilly has entered into a collaboration with biotechnology startup Superluminal Medicines to develop new drugs for cardiometabolic diseases and obesity. The partnership aims to discover and advance small molecule medicines targeting undisclosed G protein-coupled receptors (GPCRs). Lilly will receive exclusive rights to the compounds resulting from this collaboration, with Superluminal potentially earning up to $1.3 billion in total payouts.

• Autolus Therapeutics is delaying the European launch of its leukemia cell therapy, Aucatzyl, to evaluate pricing and market entry opportunities in certain countries. The company has put the German launch on hold and does not anticipate any EU sales of Aucatzyl in 2025 and 2026. In the UK, where Aucatzyl has been approved, a government cost-effectiveness monitor has initially decided not to recommend payment for the therapy.

• Generation Bio has revealed promising preclinical results for its nucleic acid delivery technology targeting T cells. However, the company has also announced a strategic review that could result in a sale or merger, citing potential difficulties in raising funds to advance the technology to human trials. As part of this review, Generation Bio plans to lay off approximately 90% of its workforce, including all research and development staff, by the end of October.

These developments underscore the dynamic nature of the pharmaceutical industry, where breakthrough approvals, clinical setbacks, and strategic realignments continue to shape the landscape of drug development and patient care.