Clinigen bets on ALS to revitalise Aldesleukin with new orphan drug designation, says GlobalData

Findings from Phase II trials of Clinigen’s Proleukin (aldesleukin) have suggested an overall shift toward immunosuppression in amyotrophic lateral sclerosis (ALS) patients who were given low doses of aldesleukin without any serious side effects. This shows promise for the use of Proleukin as a new ALS therapy, and will allow Clinigen to revitalize Proleukin by exploring its potential to enter this new therapy area, says GlobalData, a leading data and analytics company.

According to GlobalData, Clinigen is currently investigating Proleukin in eight ongoing and three planned clinical trials in various therapy areas across the US and EU to explore its modulatory effects on regulatory T-cells (Tregs) in other conditions such as multiple sclerosis, ulcerative colitis, systemic lupus erythematosus and type 1 diabetes. In its latest development, the drug’s new orphan drug status for the treatment of amyotrophic lateral sclerosis (ALS) will allow Clinigen to revitalize Proleukin by exploring its potential to enter this new therapy area.

Sarah Elsayed, Pharma Analyst at GlobalData, comments: “Clinigen will be able to capitalize on the incentives of the orphan drug status, including seven years of marketing exclusivity following its launch in the US, as well as a partial tax credit for clinical trial costs and fee waivers. If Proleukin is approved for ALS, it will be prescribed in combination with the only two treatments currently approved in the ALS market: Sanofi’s Rilutek (riluzole) and Mitsubishi’s Radicava (edaravone).

“Due to the lack of curative treatments, and the fatal course of the disease, any late-stage pipeline that demonstrates any slight benefits for ALS patients in clinical trials is expected to receive FDA approval. According to GlobalData, global sales in the ALS market is expected to grow to $1.2bn by 2029, driven by the launch of multiple late-stage pipeline drugs.”

The efficacy and safety of aldesleukin is currently being assessed in Phase IIb clinical trial MIROCALS in Europe. Involving 300 newly diagnosed ALS patients, the trial has an expected completion date of Q3 2021. Previous studies have shown that aldesleukin is well tolerated and immunologically effective in participants with ALS, which encouraged further clinical investigation on slowing ALS progression.

Elsayed continues: “The findings from the previous Phase II clinical trial IMODALS in France were encouraging. There was a significant increase in Treg numbers and frequency, as opposed to patients who were given placebo in the same trial. Additionally, aldesleukin was well-tolerated in ALS patients with no serious adverse events.”

The MIROCALS trial design aims to explore the effectiveness of recently identified disease biomarkers and research into developing future blood or spinal fluid biomarkers that could improve ALS diagnosis and drive future ALS market growth. Other pressing unmet needs remain in the ALS treatment landscape due to the lack of curative drugs that can halt or delay disease progression.

Elsayed adds: “The hope remains that Clinigen’s movements in this space will encourage more companies to invest in ALS clinical trials and uncover new drugs that could prolong the survival of patients. As such, there are significant remaining opportunities for pharmaceutical companies to target this major unmet need in the near future.”

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