Jerusalem, Israel, February 2nd 2020 – Immunity Pharma, a clinical-stage neurology-focused biopharmaceutical company, announced today that its novel ALS targeting drug IPL344 was granted an orphan drug designation by both the FDA (US Food and Drug Administration) and the EMA (European Medicines Agency).
The Orphan Drug Designation programs provide incentives for pharmaceutical companies to develop treatments for serious diseases and disorders that are rare and affect fewer than 200,000 people in the U.S and 5 in 10,000 in Europe. It provides 10 years protection against competing sales of the same drug in Europe and 7 years in the US, and guaranties a centralized regulatory process in Europe.
The regulators confirmed the ALS population size that amounts to about 80 thousand in those two territories combined. The ‘Public Summary of Opinion’ on orphan designation for IPL344 by the EMA states: “The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with ALS, because early data showed that the disease progressed more slowly in patients treated with the medicine”
IPL344 is currently in a phase 2a clinical trial in Israel. The home-care based treatment is administered by family members and home caregivers, and has so far demonstrated a good safety profile. After the completion of the ongoing Phase 2a study, Immunity Pharma prepares to perform a confirmatory phase 2b study in the US and Europe.
“Obtaining orphan drug designation for IPL344 from both regulatory agencies – in the US and in Europe – is an important milestone for Immunity Pharma. It supports our global regulatory and drug development strategy.” said Ron Pfeifer, Immunity Pharma’s chairman of the board. “We are especially pleased with EMA’s opinion about our preliminary clinical results. It ratifies the clinical potential of IPL344. This solid support from a regulatory agency strengthens our resolve to bring this drug to the market as soon as possible for the benefit of ALS patients.”
“The acceptance of orphan drug designation will accelerate our efforts to start a confirmatory multicentre trial in the US and EU with IPL344, in ALS patients.” said Eran Ovadia, Immunity Pharma’s CEO. “While pleased with the regulators’ recognition, our main efforts now are dedicated to forming this study aimed to reproducing and confirming the preliminary results of treatment with IPL344 that indicate the significant clinical potential of IPL344”
IPL344 is Immunity Pharma’s lead drug candidate. IPL344 was discovered in the Weizmann institute of Science, Israel, at Prof. Irun Cohen's Laboratory. IPL344 is being developed as an intravenous injection for the treatment of ALS and is currently in a Phase 1/2a clinical trial in ALS patients. IPL344 activates the PI3K-Akt signaling pathway in a variety of cells, including neurons, inducing pro-survival and anti-inflammatory processes.
About Immunity Pharma
Immunity Pharma Ltd. (IPL) is a privately-held clinical-stage neurology-focused biopharmaceutical company that develops therapies for neurodegenerative diseases, with an initial focus on Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. IPL’s drugs are biologically active peptides that stimulate therapeutic cell-signaling processes which are down-regulated in neurodegenerative diseases. These drugs mitigate progression of neurodegenerative diseases by inducing survival-supporting processes, including reduction of inflammation and reduction of programed cell death (apoptosis).
For more information please visit www.immunitypharma.com