Pharming pays $66M for Abliva and its 'blockbuster potential' mitochondrial disease med

Pharming is paying $66.1 million to acquire Swedish biotech Abliva and its phase 2-stage med that the Dutch drugmaker believes has blockbuster potential with a type of mitochondrial disease.

At the center of the deal is KL1333, a regulator of the co-enzymes NAD and NADH, which Abliva is evaluating in a phase 2 study in adults with genetically confirmed primary mitochondrial disease with mitochondrial DNA mutations.

Affected patients experience consistent, debilitating fatigue and muscle weakness along with reduced life expectancy, Pharming explained in a Dec. 15 release. The Netherlands-based drugmaker is eyeing a market of around 30,000 patients in the U.S., France, Germany, Italy, Spain and the U.K. alone.

The med has shown positive clinical effects in a phase 1b study, while an interim analysis of the ongoing phase 2 trial in July has “demonstrated promising differences over placebo in both alternate primary efficacy endpoints,” Pharming said in the release.

“We believe KL1333 has blockbuster potential in the U.S. alone and can significantly change Pharming’s future growth trajectory,” CEO Sijmen de Vries, M.D., said in the release. “The acquisition of Abliva would further strengthen our clinical pipeline with the addition of a therapy, with U.S. launch expected in 2028, aligning with our vision to become a leading global rare disease company.”

The deal will see Pharming Technologies, a subsidiary of Pharming, offer Abliva’s shareholders 0.45 Swedish krona in cash per share in Abliva, equivalent to a sale value of around $66.1 million.

Pharming currently markets the angioedema attack treatment Ruconest and the PI3Kδ syndrome med Joenja. The company ended September with $173.3 million in cash and securities, and de Vries said Pharming would fund the Abliva acquisition “using existing cash, and anticipate covering costs to complete the pivotal trial with positive cash flows from our existing business.”

Pharming made no secret of the fact it was on the hunt for deals. Announcing its third-quarter earnings in October, the company said it had a “continued focus on potential acquisitions and in-licensing of clinical stage opportunities in rare diseases.”

There are currently no approved drugs for mitochondrial diseases, although gene editing biotech Cellectis teamed up with newly formed company Primera Therapeutics last year in a $750 million partnership to work on therapies for these conditions. Dutch biotech Khondrion is also working on its own candidate, despite a phase 2 miss.