At the end of March 2022, ALS News Today announced that IFB-088, an experimental treatment for amyotrophic lateral sclerosis (ALS), earned Orphan Drug designation from the FDA. The treatment was developed by biotechnology company InFlectis BioScience and is currently considered to be the company’s lead compound. 

In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases. Rare diseases are conditions affecting under 200,000 Americans. As a benefit, InFlectis BioScience also receives increased regulatory assistance, fee waivers, tax credits, and 7 years of market exclusivity upon approval. 

About IFB-088

So what exactly is IFB-088? InFlectis BioScience explains that IFB-088 is also called Sephin-1, and:

selectively inhibits PPP1R15A/PP1c phosphatase complex, which in turn maintains an active Integrated Stress Response (ISR).

Prior research suggests that ISR helps to maintain the protein and cellular balance within the body. As a result, ISR stops cells from dying. This is especially important in conditions like ALS, where cellular stress is believed to cause the buildup of toxic proteins and, as a result, cell death. Research has shown that IFB-088 is generally safer and more well-tolerated than other similar therapies, while operating on the same level of efficaciousness. 

Moving forward, Inflectis BioScience hopes to begin a Phase 2 clinical trial to evaluate the safety, efficacy, and tolerability of IFB-088 for those with bulbar-onset ALS. 

What is Amyotrophic Lateral Sclerosis (ALS)?

Also called Lou Gehrig’s disease, amyotrophic lateral sclerosis (ALS) is a progressive motor neuron disease which causes progressive nerve cell degeneration in the brain, spinal cord, and brain stem. About 5-10% of cases are familial (inherited), with 90-95% of cases being sporadic (and of unknown cause). Typically, ALS manifests between the ages of 40-70. It is more common in males than in females. Symptoms vary, but can include:

• Frequent tripping and falling
• Arm, leg, and hand weakness
• Progressive muscle weakness
• Difficulty performing small movements, walking, speaking, or swallowing
• Poor posture
• Slowed or slurred speech
• Muscle cramping and twitching
• Persistent fatigue
• Periods of uncontrolled laughing or crying
• Muscle paralysis