Rare disease drugmaker Alexion Pharmaceuticals announced two separate rare disease drug development collaborations, one with Danish biotech Zealand Pharma and the other with Swedish biotech Affibody.

Alexion said it will collaborate with Zealand to discover and develop novel peptide therapies for complement-mediated diseases. The companies believe peptides offer a number of advantages, including being highly selective and potent, allowing low dosage volumes for ease of administration, and having the potential to treat a broad range of complement-mediated diseases.

“We know that uncontrolled activation of the complement pathway is responsible for many devastating diseases, and through the development of the first two complement inhibitors – Soliris and Ultomiris– we have shown the transformative impact of C5 inhibition on several of these diseases,” said John Orloff, executive vice president and head of research and development at Alexion.

Under the terms of their agreement, the companies will jointly discover and develop subcutaneously delivered peptide therapies directed to up to four complement pathway targets. Zealand will lead the joint discovery and research efforts through the preclinical stage, and Alexion will lead development efforts beginning with IND filing and phase 1 studies. Alexion will pay Zealand $25 million upfront and will make a $15 million equity investment in the company at a premium to its market price.

Alexion will have exclusive worldwide licenses, as well as development and commercial rights, for up to four targets within the complement pathway.

For the lead target, the agreement provides the potential for development-related milestones of up to $115 million, as well as up to $495 million in sales-related milestones, plus royalties. Each of the three subsequent targets can be selected for an option fee of $15 million and has the potential for additional development milestones, sales milestones, and royalty payments.

In the second deal, Alexion is teaming up with Affibody to co-develop ABY-039 for rare Immunoglobulin G (IgG)-mediated autoimmune diseases. Currently in phase 1 development, ABY-039 is a bivalent antibody-mimetic that targets the neonatal Fc receptor (FcRn). ABY-039 has been specifically designed for a longer half-life to allow for the ease and convenience of administration of the drug.

“We believe there is significant opportunity to transform patient care with FcRn-targeted therapies and are thrilled to add a second clinical-stage anti-FcRn medicine to our pipeline with this collaboration,” said Alexion’s Orloff. “While clinical development is still early, we are excited by ABY-039’s potential to be an optimal subcutaneous therapy across a number of IgG-mediated diseases, providing patients with the possibility of a convenient self-administered treatment option.”

ABY-039 is currently being evaluated for its safety, tolerability, pharmacokinetics, and pharmacodynamics that will aid in dose selection for future studies. The companies are assessing potential indications for future development.

Under the terms of their agreement, Alexion will pay Affibody $25 million upfront, with the potential for additional development- and sales-based milestones of up to $625 million, plus royalties. Alexion will lead joint clinical development of ABY-039 and commercialization activities. Affibody has the option to co-promote ABY-039 in the United States and will lead clinical development for an undisclosed indication.

Photo: John Orloff, executive vice president and head of research and development at Alexion.