Four years after being spun out of Potentia Pharmaceuticals, Apellis Pharmaceuticals said today it will acquire Potentia and intellectual property rights to its APL-2. The complement inhibitor compound is a preclinical candidate whose first clinical trial in dry age-related macular degeneration (AMD) is set for next year. The value of the acquisition was not disclosed.

A next-generation inhibitor of the class of compstatin derivatives, APL-2 is in late preclinical development in ophthalmology and is expected to enter Phase II clinical testing in patients with AMD by the middle of 2015, Apellis said. The company is also testing APL-2 in several Phase I clinical trials, having received the FDA’s Orphan Drug Designation to treat paroxysmal nocturnal hemoglobinuria (PNH).

Apellis said it will seek to further explore the interaction between complement and adaptive immunity in several indications. In addition to PNH, these include AMD, chronic obstructive pulmonary disease (COPD), as well as graft-versus-host disease (GvHD), ABO-incompatible transplantation, periodontitis, and ischemia reperfusion injury.

APL-2 inhibits complement at the levels of complement factor C3, thus blocking all downstream effector pathways of the complement cascade. Complement inhibition is the only mechanism thus far to show reductions in the growth of dry AMD, according to the company. APL-2 has the same mechanism of action as Potentia's original drug compound but has a significantly improved half-life in the eye.

“We have learned much about complement since our first venture in this area a decade ago, and have great hopes that complement inhibition will be the first effective treatment for patients with dry AMD,” Apellis CEO Cedric Francois, M.D., Ph.D., said in a statement.

Apellis was founded in 2010 as a spin-off of Potentia Pharmaceuticals, to continue the development of therapies in non-ocular diseases such as severe asthma and COPD. Apellis developed APL-2, a form of APL-1 with a longer clearance half-life in the body, in order to target systemic diseases such as paroxysmal nocturnal hemoglobinuria (PNH).

Potentia was launched in 2003—two years after winning the 2001 Harvard Business School Business Plan Competition—to commercialize APL-1 (also called POT-4), a derivative of the cyclic peptide Compstatin, discovered at the University of Pennsylvania. Compstatin and its derivatives bind to and inhibit complement activation at the level of complement factor C3, a keystone of innate or nonspecific immunity that is harmful when activated inappropriately.

Potentia licensed its ocular program to Alcon in 2009 for an undisclosed amount, in a deal that allowed Alcon to acquire Potentia based on achievement of milestones and Alcon interest in developing APL-1/POT-4. That partnership ended last year, when Potentia entered into an agreement with Apellis to develop complement inhibitors for ocular diseases.

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