Spinogenix’s SPG302 Oral Therapy Named Orphan Drug by FDA

The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to Spinogenix’s investigational oral molecule SPG302 for treating amyotrophic lateral sclerosis (ALS).

Orphan drug status is granted by the FDA to encourage the development of therapies for conditions that affect fewer than 200,000 people in the U.S. It provides Spinogenix with certain benefits and incentives, such as assistance in clinical trial design, tax credits, and fee waivers, as well as seven years of marketing exclusivity should the medication be given regulatory approval.

“Receiving orphan drug designation from the FDA for the treatment of ALS is an important milestone,” Stella Sarraf, PhD, founding CEO at Spinogenix, said in press release.

The company also received positive feedback from the FDA concerning its clinical development program for SPG302. In a meeting preceding a potential clearance for clinical testing — called a pre-investigational new drug meeting — the regulatory agency agreed with Spinogenix’s overall clinical program for SPG302.

That program includes a first-in-human Phase 1/2 clinical trial involving ALS patients, followed by a pivotal Phase 2/3 trial.

Design of the pivotal trial, which is expected to support an application for SPG302’s approval, will be based on the safety and pharmacokinetics data observed in the Phase 1/2 trial. Pharmacokinetics refers to how a medicine is processed within the body, from its absorption, distribution, and metabolism until excretion.

“We are also pleased with the guidance from the FDA that now provides us with a clear clinical plan to rapidly advance our first-in-class drug to help ALS patients,” Sarraf said.

SPG302 is an orally administered small molecule that restores lost synapses — the point of contact between two nerve cells, where they transmit chemical messages to communicate.

Called a spinogenic compound, the therapy is designed to increase these connections between nerve cells, which has led to improvements in motor and cognitive function in animal models of neurodegenerative diseases, the company said.

Of note, the candidate therapy is able to penetrate the blood brain barrier — a highly selective membrane that prevents most molecules from reaching the brain and spinal cord and thus limiting their effectiveness.

Spinogenix expects to work with Mass General Hospital, in Boston, as it moves toward the trial phase with SPG302.

“We are thrilled that the FDA granted [orphan drug designation] to Spinogenix for their novel drug SPG302 and are excited to collaborate with them as they advance toward the clinic,” said Merit Cudkowicz, MD, director of the hospital’s Sean M. Healey and AMG Center for ALS.

The U.S. Department of Defense recently awarded Spinogenix a research grant further test the candidate therapy for ALS. The project will first use induced pluripotent stem cells (iPSCs) from ALS patients and healthy controls, and then move to animal models of the disease in a later stage.

iPSCs are stem cells generated from mature cells, either from the skin or blood, which are programmed to become stem cells and can then originate different cell types, including nerve cells, depending on the chemical cue provided.