As Priovant works toward approval of potential blockbuster brepocitinib as a treatment for dermatomyositis, the North Carolina biotech has presented promising results for the TYK2/JAK1 inhibitor in another rare skin condition.
A phase 2 study evaluating brepocitinib in patients with cutaneous sarcoidosis (CS) has become the first industry-sponsored, placebo-controlled trial to generate a positive readout in the indication, according to Priovant. And, based on the results, Priovant plans to initiate a phase 3 study this year, after consultation with the FDA, the four-year-old company said.
Analysts at Leerink Partners deemed the results “exceptional” and said the findings “substantially” exceeded expectations, according to a Feb. 6 note.
The Beacon trial enrolled 31 patients, randomizing them at a rate of 3:2:2 to receive a daily 45-mg oral dose of brepocitinib, a 15-mg daily dose or placebo for 16 weeks. The 45-mg arm achieved a 100% response rate in multiple endpoints.
While patients in the placebo group achieved close to no benefit, those in the 15-mg arm “improved considerably, with numerically similar improvement to the 45 mg arm on lower-bar endpoints,” Priovant said. There also was evidence of dose-dependent benefit seen on higher-bar endpoints, the biotech added.
On the Cutaneous Sarcoidosis Activity and Morphology Instrument Activity (CSAMI-A) score, brepocitinib 45 mg achieved a 22.3-point mean improvement at Week 16 compared to a 0.7-point improvement in the placebo arm, with statistically significant separation of treatment effect observed as early as Week 4. All brepocitinib 45-mg patients achieved a clinically meaningful 10-point improvement.
The CSAMI-A scores range from 0 to 165, evaluating the severity of skin lesions in sarcoidosis.
Another measurement tool used in the trial was the Investigator’s Global Assessment, which measures overall disease severity in dermatological studies, with scores ranging from 0 (clear) to 5 (very severe). In Beacon, 69% of the patients in the 45-mg arm achieved a two-point improvement to a score of 0 or 1 (almost clear).
Misha Rosenbach, M.D., the director of the CS program at the Hospital of the University of Pennsylvania, called the study a “watershed moment for the sarcoidosis field.”
“This is an incredible milestone for a historically neglected disease,” Rosenbach added. “The study drug showed a clear difference in patients who received the medication compared to placebo, both from the patient and the physician perspective, and appeared to be well tolerated. This is the sort of data you dream of seeing when you look at trial results.”
CS is an inflammatory skin disease affecting approximately 40,000 adults in the U.S. CS lesions are often profoundly disfiguring, Priovant said, leading to substantial psychosocial distress. With no treatments approved for CS, many patients rely on off-label corticosteroids, immunosuppressants and biologics, which provide limited efficacy and can become toxic.
This is another significant step in the progression of brepocitinib, which scored last year in a phase 3 trial of patients with dermatomyositis. The study showed brepocitinib to be a best-in-class treatment, according to TD Cowan, and Priovant has submitted a new drug approval request to the FDA. The company also is running a phase 3 trial of brepocitinib for patients with the inflammatory eye condition noninfectious uveitis.
Priovant was created in 2022 through a partnership between Pfizer and Roivant Sciences to advance brepocitinib and the TYK2 inhibitor ropsacitinib. The New York City pharma giant holds a 25% stake in the new company. Evaluate has forecast sales of brepocitinib to reach $2.3 billion by 2032.
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