Two M&A transactions and two partnering deals brightened the generally slow month of August for rare disease therapeutics financings and deals as global economic conditions continued to make raising capital difficult, according to data gathered by Global Genes and Dealforma.

Total public and private equity and debt of rare disease focused therapeutics developers reached $10 billion year-to-date through the end of August, almost 42 percent below the amount raised by these companies during the same period in 2021. That compares with $42.5 billion raised by all therapeutics developers through public and private equity and debt deals, a drop of 31 percent compared to last year.

Venture funding of rare disease focused drug developers is down 39 percent year-to-date compared to the same period in 2021, according to data gathered by Global Genes and Dealforma. Only two companies received funding in August: IDRx raised $122 million in a series A round to advance its purpose-built precision combination therapies to treat rare cancers; and Swiss startup Vector BioPharma, which launched with $30 million to develop a precision gene delivery platform.

While public equity and debt raises by rare disease therapeutics developers is off by 20 percent compared to the same period in 2021, four companies tapped the markets to raise capital to fund their operations and extend their cash runways: Mirum Pharmaceuticals raised $92 million; Fulcrum Therapeutics raised $75 million; and Poseida Therapeutics raised $70 million—all three through secondary offerings of common stock, while MeiraGTx raised $75 million through a $100 million term loan agreement with affiliates of Perceptive Advisors.  Pfizer stepped up with $5.4 billion to acquire Global Blood Therapeutics, paying a 102 percent premium to strengthen its rare hematology portfolio with the approved drug Oxbryta to treat sickle cell anemia, and a clinical stage pipeline that Pfizer says give it the potential for an SCD franchise that could achieve combined worldwide peak sales of more than $3 billion.

Amgen beefed up its rare autoimmune diseases portfolio with the acquisition of ChemoCentryx for $3.7 billion. The deal gives Amgen Tavneos, an oral selective complement component 5a receptor inhibitor that was approved in the United States in October 2021 as an adjunctive treatment for adult patients with severe active ANCA-associated vasculitis in combination with standard therapy. ChemoCentryx is also developing Tavneos for the treatment of patients with C3 glomerulopathy, severe hidradenitis suppurativa and lupus nephritis, and has three early-stage drug candidates that target chemoattractant receptors in other inflammatory diseases and an oral checkpoint inhibitor for cancer.

Two potentially lucrative partnering deals raised the total potential deal values of rare disease focused deals to $25.6 billion year to date, 5.3 percent ahead of the same period in 2021.

In early August, Poseida Therapeutics entered into a broad strategic collaboration and license agreement with Roche to develop multiple, off-the-shelf, CAR-T therapies directed to hematologic malignancies, most of which are rare. Poseida received $110 million upfront and is eligible to receive research, development, launch, and net sales milestones and other payments potentially up to $6 billion in aggregate value, as well as tiered net sales royalties into the low double digits, across multiple programs. For a subset of both the Poseida portfolio programs licensed or optioned to Roche and future collaboration programs, Poseida will conduct the phase 1 studies and manufacture clinical materials before transitioning the programs to Roche for further development and commercialization.

Kiniksa Pharmaceuticals entered into a global license agreement with Roche and Genentech for the rights to develop and commercialize vixarelimab, a fully human monoclonal antibody targeting oncostatin M receptor beta, which Genentech will develop in fibrosis, where oncostatin M (OSM)-mediated pathogenesis is thought to be an important pathway for intervention in multiple fibrotic indications. Kiniksa received $100 million in upfront and near-term payments, and is eligible to receive up to approximately $600 million in certain clinical, regulatory, and sales-based milestones, before fulfilling upstream financial obligations. Kiniksa is also eligible to receive royalties on annual net sales. Genentech will obtain rights for the development and commercialization of vixarelimab.

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