SPG302 for ALS Receives Orphan Drug Designation


In early June 2021, SPG302, an investigational therapy for patients with amyotrophic lateral sclerosis (ALS), received Orphan Drug designation from the FDA. According to ALS News Today, the treatment was developed by Spinogenix, whose mission is to create novel and beneficial therapies for neurodegenerative conditions.


So what is SPG302? A Spinogenix press release describes SPG302 as:

an orally bioavailable, blood-brain barrier penetrating small molecule. Its mode of action regenerates lost synapses and has demonstrated improvements in cognitive and motor behaviors in multiple animal models of neurodegenerative disorders.

The therapy’s ability to move through the blood-brain barrier (BBB) offers a more targeted and effective treatment option. Additionally, the therapy works to rebuild or regenerate synapses. By doing so, SPG302 rebuilds connections between nerve cells to improve cognitive and motor function.

SPG302 Orphan Drug Designation

Orphan Drug designation is granted by the FDA for the development of drugs and biologics intended to treat rare and life-threatening conditions. In this case, “rare” conditions are those affecting under 200,000 Americans. Once a drug receives Orphan Drug status, the drug developer also receives a set of benefits. For example, Spinogenix now receives tax credits and fee waivers, FDA regulatory assistance, and 7 years of market exclusivity (once SPG302 is approved for use).

Further Drug Development

Outside of receiving Orphan Drug designation for SPG302, Spinogenix also received a number of other updates from the FDA. First, Spinogenix received approval to move forward with a Phase 1/2 clinical trial evaluating SPG302 for ALS. Following this trial, and not counting any potential issues that may arise, Spinogenix will proceed to a Phase 2/3 trial.

Additionally, Spinogenix was given a research grant from the Department of Defense to study its therapy using induced pluripotent stem cells (iPSCs).

Amyotrophic Lateral Sclerosis (ALS)

Sometimes known as “locked-in syndrome” or “Lou-Gehrig’s syndrome,” amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease. In most cases, doctors do not know what causes ALS. However, some believe that certain toxin exposure could play a role. Around 5-10% of ALS diagnoses are familial, which means there is an underlying genetic cause. ALS is characterized by the death or degeneration of nerve cells, particularly in the brain, brain stem, and spinal cord. As these nerve cells die, muscles weaken and waste. Patients experience a loss of voluntary movement and control, preventing them from living independently. Typically, ALS affects men more than women; it also affects older individuals more than younger individuals. Unfortunately, ALS is fatal. While patients can live for extended periods following diagnosis, the typical survival rate is between 3-5 years following diagnosis.

Symptoms vary. In initial stages, symptoms may hardly be noticeable. However, as ALS progresses, the following symptoms could appear:

  • Frequent tripping and falling
  • Changes in speech, such as slurring or slowed speech
  • Psychological distress (depression, anxiety)
  • Difficulty performing small movements or normal activities, such as holding objects or walking
  • Leg, feet, hand, and ankle weakness
  • Inappropriate crying, yawning, or laughing
  • Muscle twitching and cramping
  • Difficulty speaking and swallowing
  • Poor posture
  • Progressive inability to move muscles
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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