Raising Capital Remained Tough for Rare Disease Therapeutics Drug Developers in July

Marie Daghlian

Through the end of July, rare disease drug developers raised a total $9.5 billion in equity and debt in 2022, a 41 percent decline compared to the same time period in 2021, according to data gathered by Dealforma and Global Genes.

That was slightly better than the 43 percent drop in public and private capital raised by all drug developers year-to-date during the same period in 2021.

A handful of rare disease focused companies completed venture financings in July, including Camp4 Therapeutics, which raised $100 million in a series B round to advance its regulatory RNA (regRNA) programs and accelerate expansion of its platform, and Vicinitas, which launched with $65 million in a series A round to advance a targeted protein stabilization platform to develop therapeutics for cancer and genetic disorders.

Though public financings are down 21 percent, companies can still raise money on the heels of positive clinical data. Just days after announcing positive date from a mid-stage trial of it experimental therapy PLN-74809 in patients with idiopathic pulmonary fibrosis, Pliant Therapeutics raising $230.0 million in a public offering of common stock.

There was little M&A activity in the rare disease therapeutics space in July, with potential deal values down 28 percent year-to-date compared to the same period in 2021. The biggest deals involved the selling of royalty streams for immediate cash. Ultragenyx Pharmaceutical sold 30 percent of its royalty interest from Kyowa Kirin on the future sales of Crysvita in the Unites States and Canada to Omers, one of Canada’s largest defined benefit pension plans, for $500 million. And Blueprint Medicines sold its royalties receivable from Gavreto net sales by Roche outside the United States except for China for $175 million in upfront cash and up to $165 million in potential milestone payments based on future sales.

Biobucks played a large part in rare disease partnering deals, one area that was on par with 2021—up 3.4 percent in total potential deal values, a deal value at signing fell 56 percent. Avista Therapeutics partnership with Roche is a good example. The companies will work together to develop novel AAV gene therapy vectors for ocular diseases. Roche has the right to evaluate and license novel capsids from Avista, and will be responsible for conducting preclinical, clinical, and commercialization activities for gene therapy programs using these novel capsids, which will be distinct from Avista’s internal pipeline. Roche is paying Avista $7.5 million upfront, with potential research and milestone payments that may exceed $1 billion.