CS1 receives orphan medicinal product status for PAH in Europe
Cereno Scientific to announce top-line data from Phase 2 trial soon
The European Commission has granted orphan medicinal product designation to Cereno Scientific’s CS1 for the treatment of people with pulmonary arterial hypertension (PAH).
This designation is given to medications for life-threatening or chronically debilitating diseases, affecting up to five per 10,000 individuals in the European Union (EU), or treatments whose marketing likely does not generate sufficient returns to justify the investment in development. The designation comes with certain incentives, including scientific advice and 10 years of market exclusivity if the medication is ultimately approved.
The EU status complements the orphan drug designation granted by the U.S. Food and Drug Administration (FDA) in 2020, “enhancing the protection and value of CS1 in these major markets,” Sten R. Sörensen, Cereno’s CEO, said in a company press release. “These market exclusivities, alongside our robust patent portfolio, are integral to our commercial strategy for CS1.”
The treatment’s safety, tolerability, pharmacological properties, as well as preliminary efficacy, are being tested in adults with PAH in the Phase 2 CS1-003 trial (NCT05224531). The trial stopped recruiting patients about two months ago after its clinical steering committee concluded that enough data had been collected to assess next steps. Top-line data are expected soon.
CS1 is reformulation of anti-seizure medication valproic acid
CS1 is a reformulation of valproic acid, an anti-seizure medication, that works as an epigenetic modulator, meaning it makes changes to how genes are read, but not to the DNA sequence itself. It’s designed to block histone deacetylase, an enzyme that makes DNA less accessible for the production of proteins.
The therapy is reported to have pressure-relieving, anti-inflammatory, anti-scarring, and anti-blood-clotting properties, which are thought to have therapeutic benefits in PAH. The disease is characterized by the narrowing of pulmonary arteries that cause high blood pressure in lung arteries, potentially leading to right heart failure.
Preliminary data from the first patient who completed the Phase 2 trial showed that treatment for about three months reduced pulmonary hypertension by 30%, while cardiac output, which is the amount of blood pumped by the heart in one minute, increased by 20%. In addition, the patient’s physical function reached nearly normal levels. In several other patients, CS1 was also found to reduce pulmonary pressure in a clinically meaningful way.
Early this year, the FDA approved an expanded access program (compassionate use) for CS1, as an extension of the Phase 2 trial. This program enables patients who have completed the trial to continue on CS1 and will allow Cereno to collect long-term safety and efficacy data. The first patient under this program was dosed recently.
Having such data may help the company in regulatory discussions and in planning a potential Phase 2/3 clinical study.
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