A development deal with Biogen Inc. could eventually bring City Therapeutics Inc. about $1 billion in milestone payments. Privately held City Therapeutics is getting $46 million in the deal. That includes $16 million as an up-front payment and an investment of $30 million in exchange for a City Therapeutics convertible note. City Therapeutics plans to develop at RNAi trigger molecule and combine it with Biogen’s drug delivery capabilities. The two companies are focused on a sole target that mediates central nervous system indications. Biogen is handling IND-enabling duties, global clinical development, regulatory submissions and commercialization.

Grin’s radiprodil heads to phase III with $570M deal, $140M series D

A new version of a drug that failed in a phase II for neuropathic pain 15 years ago has attracted $140 million in series D funds, plus an ex-North American deal worth up to $570 million, for Grin Therapeutics Inc. The deal, signed with Rome’s Angelini Pharma SpA, involves commercial rights worldwide, except for the U.S., Canada and Mexico, for Grin’s radiprodil, a negative allosteric modulator that selectively targets the GluN2B subunit of the N-methyl-D-aspartate receptor. Grin plans to move the product into a phase III trial for GRIN-related neurodevelopmental disorder in the third quarter of 2025. Angelini is making a $65 million investment in the series D, and paying $50 million up front for the radiprodil rights, with the promise of up to $520 million in development, regulatory and sales milestone payments, plus tiered royalties on global sales and payments from any future sublicenses outside of Europe. Existing Grin investor Blackstone Life Sciences contributed the other $75 million to the series D round.

Patient death taints Rocket phase II in Danon disease

Shares of Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) were trading at $2.68, down $3.60, or 57%, after an update related to RP-A501, its gene therapy for Danon disease. A patient in the phase II pivotal trial of the compound, which delivers a LAMP2B gene to heart cells, turned up an unexpected serious adverse event that involved complications related to a capillary leak syndrome and later died from further problems. The U.S. FDA has placed the study on clinical hold, Rocket said, as CEO Gaurav Shah called the loss “deeply tragic” and said the firm is committed to understanding what happened “objectively and neutrally.”

Prothena sinks on phase III miss in amyloidosis

History has repeated itself for Prothena Corp. plc, which has reported a second phase III miss for birtamimab in the treatment of light chain amyloidosis. Announcing the trial failure, the company said it is planning “a substantial reduction” of its organization. The phase III Affirm trial did not meet the endpoint of time to all-cause mortality, or the secondary endpoints of the six-minute walk test distance, or the physical component score.

GC Genome plans $30M IPO to diversify genomic products, suppliers

GC Genome Corp. priced a Kosdaq offering of 4 million shares at ₩10,500 per share May 27, entailing a gross ₩42 billion (US$30.5 million) raise in early June. The Yongin, South Korea-based genomics subsidiary of GC Biopharma Corp. fixed its shares price to the top of its ₩9,000 to ₩15,000 price band May 27 after conducting demand forecasting on domestic and international institutional investors from May 19 to May 23. GC Genome reported a competitive subscription rate of 547.47-to-1.

Axsome's solriamfetol shows mixed results amid 13 phase III wins in April

BioWorld recorded 151 clinical trial updates from phases I through III in April 2025, a drop from 197 in March. The month saw 13 successful phase III readouts, alongside four failed trials and two with mixed results.

Holiday notice

BioWorld's offices were closed in observance of Memorial Day in the U.S. No issue was published Monday, May 26.

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