The BioPharm Brief: Incentives, Immunity, and Iron

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry.

First, rare disease biotech investment is getting a boost in the US following renewal of the FDA’s Priority Review Voucher program. The vouchers, which can be sold or transferred, have recently commanded prices of $150 million to $200 million, creating a significant financial incentive for companies developing treatments for rare diseases. SynaptixBio says the UK is falling behind because it lacks a comparable incentive.

In Parkinson disease, AC Immune’s investigational immunotherapy ACI-7104 has received FDA fast track designation and IND clearance. The therapy is designed to generate antibodies against aggregated alpha-synuclein, and the company is expanding its phase 2 study into the US.

And in polycythemia vera, Silence Therapeutics reported positive phase 2 results for divesiran, an siRNA therapy targeting TMPRSS6. Eighty-eight percent of patients receiving divesiran achieved a clinical response, compared with 19% on placebo. The company plans to begin phase 3 development in 2027.

The numbers to remember today are $150 million, 88%, and one FDA fast track designation.

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Key takeaways:

• PRVs can drive investment in rare disease programs by creating a valuable financial incentive.
• Fast track can accelerate development as AC Immune advances its Parkinson disease immunotherapy into phase 2.
• siRNA shows promise in polycythemia vera, with divesiran producing significantly higher response rates than placebo.