Pharmaceutical Industry Update: FDA Approval, Acquisitions, and Clinical Trial Results

FDA Actions and Regulatory Developments

The U.S. Food and Drug Administration (FDA) has approved Novartis' Rhapsido (remibrutinib) for the treatment of chronic spontaneous urticaria, a form of persistent hives. This approval marks Rhapsido as the first oral targeted therapy for this condition. The drug belongs to a newer class of BTK inhibitors designed to treat autoimmune diseases rather than cancer.

In a separate development, comments made by George Tidmarsh, head of one of the FDA's main drug review offices, sparked controversy and a significant market reaction. Tidmarsh criticized Aurinia Pharmaceuticals' lupus nephritis drug, Lupkynis, in a social media post that was later retracted. The post, which claimed the drug had "significant toxicity" and hadn't proven a "direct clinical benefit to patients," led to a double-digit decline in Aurinia's share price. Tidmarsh subsequently clarified that his comments did not reflect the views of the FDA or HHS, while Aurinia issued a statement defending Lupkynis' "favorable benefit/risk profile."

Corporate Acquisitions and Restructuring

Halozyme Therapeutics has announced the acquisition of Elektrofi in a deal worth up to $900 million. The acquisition provides Halozyme with microparticle technology that enhances the delivery of biologic medicines through subcutaneous injections. Halozyme is paying $750 million upfront, with potential additional payments of $150 million tied to product approvals.

In response to regulatory feedback, IO Biotech has announced plans to lay off half of its staff. The company had hoped to submit its experimental skin cancer vaccine, Cylembio, for approval based on a narrowly failed Phase 3 trial. However, the FDA has advised against submitting an application and recommended a new trial. IO Biotech will work with the FDA to design the new trial while restructuring to conserve cash, as it only has enough funds to operate through the first quarter of 2026.

Clinical Trial Results and Pipeline Updates

Enanta Pharmaceuticals reported mixed results from a Phase 2 trial of its experimental respiratory syncytial virus treatment, zelicapavir. While the drug missed the trial's primary endpoint of quickly resolving specific respiratory symptoms to "mild," it demonstrated a "clinically meaningful benefit" in a broader set of symptoms among high-risk adults. These results have led to a nearly doubling of Enanta's share price as the company plans to move forward with Phase 3 testing using a revised study design.

In contrast, Kala Bio faced a significant setback as its lead candidate, KPI-012, for persistent corneal defects failed to meet primary and secondary goals in a mid-stage trial. As a result, Kala Bio has discontinued the development of KPI-012 and announced plans to lay off 19 employees, representing more than half of its staff. The company will explore "strategic options" moving forward.