Rare Disease Therapeutics Financings Slow but M&A and Partnering Deals Abound in November

Marie Daghlian

While financings for public and private companies slowed in November, M&A and Partnering deals remained strong among companies developing rare disease therapeutics, according to data gathered by Dealforma and Global Genes.

Nine acquisitions and asset purchase transactions involving rare disease assets and companies, seven of which had disclosed deal values, raised M&A in the sector to $61 billion since the beginning of the year, up 221.5 percent from the values during the same period in 2020.

The most significant deal of note was Novo Nordisk’s push into the rare disease space with the $3.3 billion acquisition of Dicerna Pharmaceuticals, a premium of 80 percent to Dicerna’s closing price ahead of the deal. The companies were two years into a research collaboration when Novo decided to buy Dicerna outright. Their research collaboration focused on discovering and developing RNAi therapies using Dicerna’s proprietary GalXC RNAi platform technology and encompassed the exploration of more than 30 liver cell targets with the potential to deliver multiple clinical candidates for disorders including chronic liver disease, non-alcoholic steatohepatitis, type 2 diabetes, obesity, and rare diseases.

Partnering deals were also plentiful in November, with 25 announced collaborations and licensing deals, nine of which disclosed total potential deal values totaling a combined $4 billion, with $300 million of that paid at signing. Rare disease therapeutic partnering potential deal values are up 38 percent year to date compared to the same period in 2020, while the money paid at signing is up 23.5 percent compared to 2020 signing values.

The largest deal, valued at $2.5 billion but with other details not disclosed, was BridgeBio Pharma and Helsinn Group’s strategic collaboration to co–develop and co-commercialize a potentially first-in-class inhibitor designed to target glutathione peroxidase 4 (GPX4) with the hope of providing an effective new therapy for patients with difficult-to-treat tumors.

The collaboration for BridgeBio’s GPX4 inhibitor was established as part of a new non-exclusive collaboration framework between BridgeBio and Helsinn allowing the companies to propose co-development and co-commercialization opportunities for preclinical precision oncology programs. It builds on an earlier global collaboration and licensing agreement that BridgeBio and Helsinn Group’s affiliates, Helsinn Healthcare and Helsinn Therapeutics, entered into in March 2021.

For each program that the parties agree to pursue, they will share global development responsibilities under an agreed cost split. Helsinn will have exclusive manufacturing and commercial rights to the programs under the agreement, with BridgeBio receiving a profit share on U.S. sales and tiered royalties on ex-U.S. sales.

Although venture financings of rare disease therapeutics companies have been strong across the board in 2021 at $8.8 billion year to end of November and up 35 percent compared to the same period last year, it has gone down as a percent of total financings, comprising 23 percent so far this year compared to 30 percent of total capital raised at the end of November 2020. Still, privately held rare disease drug developers added $540 million to their coffers in the month.