Pharming Group N.V. has announced a recommended public cash offer to acquire Abliva AB, a biotechnology company focused on mitochondrial disease treatments, for SEK 0.45 per share in cash. The total transaction is valued at approximately US$66.1 million. This acquisition aims to strengthen Pharming's late-stage pipeline with Abliva's lead product, KL1333, currently in a pivotal clinical trial for primary mitochondrial disease (PMD).
KL1333: A Potential First-in-Disease Treatment
Abliva's KL1333 is under development for adult patients with genetically confirmed PMD, specifically those with mitochondrial DNA (mtDNA) mutations experiencing consistent, debilitating fatigue and muscle weakness (myopathy). This patient population often faces reduced life expectancy. KL1333, a regulator of the essential co-enzymes NAD+ and NADH, has demonstrated positive clinical effects in an earlier Phase 1b study. An interim analysis of the ongoing pivotal FALCON trial also showed promising differences over placebo in alternate primary efficacy endpoints.
Clinical Trial and Regulatory Status
The FALCON study is a Phase 2, global, randomized, placebo-controlled trial evaluating the safety and efficacy of KL1333. It involves 180 patients with mitochondrial DNA mutations, randomized 3:2 to receive KL1333 (50mg-100mg) or placebo twice daily for 48 weeks. The trial's primary endpoints assess consistent fatigue (using the PROMIS® Fatigue Mitochondrial Disease Short Form) and myopathy (using the 30-second Sit-to-Stand test). KL1333 has already received Fast Track designation in the U.S. and Orphan Drug Designation for PMD in both the U.S. and EU.
Strategic Rationale and Financial Details
Pharming intends to fund the acquisition using existing cash reserves, with no external funding required. The company anticipates covering the costs to complete the pivotal trial with positive cash flows from its existing business. According to Sijmen de Vries, CEO of Pharming, KL1333 has blockbuster potential in the U.S. alone and can significantly change Pharming’s future growth trajectory. The acquisition aligns with Pharming's vision to become a leading global rare disease company, with a U.S. launch of KL1333 expected in 2028.
Market and Shareholder Support
KL1333 targets a significant addressable patient population, with over 30,000 patients diagnosed with mtDNA mitochondrial disease in the U.S., EU4 (France, Germany, Italy, Spain), and the UK. Pharming has secured acceptance undertakings from the three largest shareholders of Abliva, accounting for 49.82% of Abliva's outstanding shares. The Board of Directors of Abliva has unanimously recommended that shareholders accept the offer, further supported by a fairness opinion from PwC.
The acceptance period for the offer is expected to commence around January 16, 2025, and expire around February 7, 2025, pending customary regulatory approvals.
