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MIRM, INCY Report Positive Rare Disease Data as FDA Begins NDA Review
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Key Takeaways
MIRM/INCY reported positive phase II data showing zilurgisertib met the primary endpoint in FOP patients.
The investigational therapy reduced new HO lesion formation by 81% versus placebo and met key secondary goals.
MIRM and INCY's zilurgisertib NDA received FDA Priority Review, with a decision due by Sept. 26, 2026.
Mirum Pharmaceuticals (MIRM - Free Report) and Incyte Corporation (INCY - Free Report) have reported positive pivotal phase II results from Cohort 1 of the PROGRESS study evaluating zilurgisertib, an investigational oral ALK2 inhibitor, in adolescent and adult patients with fibrodysplasia ossificans progressiva (FOP). The data showed that the investigational oral therapy significantly reduced new heterotopic ossification (HO) lesion formation, meeting the study’s primary endpoint. Key secondary endpoints, including lesion volume measures and disease flare activity, were also met, supporting zilurgisertib’s potential as a new treatment option for the ultra-rare disease.
FOP is a rare genetic disorder in which muscle, tendons and other soft tissues progressively transform into bone, causing irreversible loss of mobility and severe disability over time. Based on the positive pivotal study findings, the FDA has accepted the new drug application (NDA) for zilurgisertib for FOP and granted Priority Review status. The agency is expected to deliver its decision regarding the NDA by Sept. 26, 2026.
A filing accepted under the FDA’s Priority Review pathway reduces the review period to six months from the standard 10 months. This status is awarded to marketing applications for medicines intended to treat serious conditions and that, if approved, would offer a substantial improvement in safety, effectiveness, prevention, or diagnosis of such conditions. Mirum licensed worldwide development and commercialization rights to the candidate from Incyte.
Year to date, MIRM and INCY’s shares have risen 25.7% and 9.9%, respectively, against the industry’s 1.4% decline.
Image Source: Zacks Investment Research
MIRM/INCY's Pivotal Phase II FOP Study Data in Details
The pivotal phase II PROGRESS study is evaluating the safety and efficacy of zilurgisertib in FOP patients. Cohort 1 enrolled 63 patients aged 12 years and older, who were randomized equally to receive either zilurgisertib 100 mg once daily or placebo for 24 weeks, followed by an open-label extension period. At the time of analysis, 61 patients had available 48-week whole-body CT scan data.
The primary endpoint assessed the proportion of patients developing new HO lesions at Week 24. Results showed that only one patient (3.1%) in the zilurgisertib arm developed a new lesion compared with five patients (16.7%) in the placebo group, representing an 81% reduction. The treatment demonstrated consistent benefits across several secondary efficacy measures.
Mirum and Incyte reported particularly strong effects on new lesion burden. Patients receiving zilurgisertib experienced a 99.9% reduction in the total volume of newly formed HO lesions compared with placebo at Week 24. The mean volume of new lesions was nearly eliminated in the treatment arm, highlighting the drug's ability to suppress abnormal bone formation. Treated patients also developed fewer new lesions overall compared with placebo recipients.
The study also showed favorable effects on overall disease burden. Patients receiving zilurgisertib experienced a reduction in total HO lesion volume by Week 24, whereas total lesion volume increased among placebo-treated patients. Annualized flare activity was likewise lower in the treatment arm, with patients receiving zilurgisertib reporting fewer new disease flares than those receiving placebo.
Results from the open-label extension demonstrated sustained benefit through Week 48. No new HO lesions were observed among patients who continued receiving zilurgisertib, and no new lesions were reported in patients who crossed over from placebo to active treatment after Week 24. Total HO lesion volume continued to decline in both groups through Week 48, while flare activity remained low over the extended treatment period.
The safety profile remained favorable throughout the placebo-controlled portion of the study. Most adverse events were mild to moderate in severity, and no patients discontinued treatment or required dose reductions because of adverse events.
With positive pivotal data now in hand and Priority Review underway, Mirum and Incyte are moving closer to potentially bringing zilurgisertib to patients with FOP, a population with limited treatment options and significant unmet medical need.
Over the past 60 days, estimates for Liquidia Corporation’s 2026 EPS have increased from $1.50 to $2.97. Over the same period, EPS estimates for 2027 have also increased from $2.91 to $4.81. LQDA shares have rallied 106.7% year to date.
Liquidia Corporation’searnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
The estimate for Immunocore’s 2026 EPS is currently pegged at 6 cents. In the past 60 days, the estimates for its 2027 EPS have increased from 24 cents to 87 cents. IMCR shares have lost 16.5% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 46.66%.
View the Zacks #1 Rank List
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MIRM, INCY Report Positive Rare Disease Data as FDA Begins NDA Review
Key Takeaways
Mirum Pharmaceuticals (MIRM - Free Report) and Incyte Corporation (INCY - Free Report) have reported positive pivotal phase II results from Cohort 1 of the PROGRESS study evaluating zilurgisertib, an investigational oral ALK2 inhibitor, in adolescent and adult patients with fibrodysplasia ossificans progressiva (FOP). The data showed that the investigational oral therapy significantly reduced new heterotopic ossification (HO) lesion formation, meeting the study’s primary endpoint. Key secondary endpoints, including lesion volume measures and disease flare activity, were also met, supporting zilurgisertib’s potential as a new treatment option for the ultra-rare disease.
FOP is a rare genetic disorder in which muscle, tendons and other soft tissues progressively transform into bone, causing irreversible loss of mobility and severe disability over time. Based on the positive pivotal study findings, the FDA has accepted the new drug application (NDA) for zilurgisertib for FOP and granted Priority Review status. The agency is expected to deliver its decision regarding the NDA by Sept. 26, 2026.
A filing accepted under the FDA’s Priority Review pathway reduces the review period to six months from the standard 10 months. This status is awarded to marketing applications for medicines intended to treat serious conditions and that, if approved, would offer a substantial improvement in safety, effectiveness, prevention, or diagnosis of such conditions. Mirum licensed worldwide development and commercialization rights to the candidate from Incyte.
Year to date, MIRM and INCY’s shares have risen 25.7% and 9.9%, respectively, against the industry’s 1.4% decline.
Image Source: Zacks Investment Research
MIRM/INCY's Pivotal Phase II FOP Study Data in Details
The pivotal phase II PROGRESS study is evaluating the safety and efficacy of zilurgisertib in FOP patients. Cohort 1 enrolled 63 patients aged 12 years and older, who were randomized equally to receive either zilurgisertib 100 mg once daily or placebo for 24 weeks, followed by an open-label extension period. At the time of analysis, 61 patients had available 48-week whole-body CT scan data.
The primary endpoint assessed the proportion of patients developing new HO lesions at Week 24. Results showed that only one patient (3.1%) in the zilurgisertib arm developed a new lesion compared with five patients (16.7%) in the placebo group, representing an 81% reduction. The treatment demonstrated consistent benefits across several secondary efficacy measures.
Mirum Pharmaceuticals, Inc. Price and Consensus
Mirum Pharmaceuticals, Inc. price-consensus-chart | Mirum Pharmaceuticals, Inc. Quote
Mirum and Incyte reported particularly strong effects on new lesion burden. Patients receiving zilurgisertib experienced a 99.9% reduction in the total volume of newly formed HO lesions compared with placebo at Week 24. The mean volume of new lesions was nearly eliminated in the treatment arm, highlighting the drug's ability to suppress abnormal bone formation. Treated patients also developed fewer new lesions overall compared with placebo recipients.
The study also showed favorable effects on overall disease burden. Patients receiving zilurgisertib experienced a reduction in total HO lesion volume by Week 24, whereas total lesion volume increased among placebo-treated patients. Annualized flare activity was likewise lower in the treatment arm, with patients receiving zilurgisertib reporting fewer new disease flares than those receiving placebo.
Results from the open-label extension demonstrated sustained benefit through Week 48. No new HO lesions were observed among patients who continued receiving zilurgisertib, and no new lesions were reported in patients who crossed over from placebo to active treatment after Week 24. Total HO lesion volume continued to decline in both groups through Week 48, while flare activity remained low over the extended treatment period.
The safety profile remained favorable throughout the placebo-controlled portion of the study. Most adverse events were mild to moderate in severity, and no patients discontinued treatment or required dose reductions because of adverse events.
With positive pivotal data now in hand and Priority Review underway, Mirum and Incyte are moving closer to potentially bringing zilurgisertib to patients with FOP, a population with limited treatment options and significant unmet medical need.
Incyte Corporation Price and Consensus
Incyte Corporation price-consensus-chart | Incyte Corporation Quote
MIRM/INCY’s Zacks Rank and Stocks to Consider
Mirum and Incyte currently carry a Zacks Rank #3 (Hold) each.
Some better-ranked stocks in the biotech sector are Liquidia Corporation (LQDA - Free Report) and Immunocore (IMCR - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Liquidia Corporation’s 2026 EPS have increased from $1.50 to $2.97. Over the same period, EPS estimates for 2027 have also increased from $2.91 to $4.81. LQDA shares have rallied 106.7% year to date.
Liquidia Corporation’searnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
The estimate for Immunocore’s 2026 EPS is currently pegged at 6 cents. In the past 60 days, the estimates for its 2027 EPS have increased from 24 cents to 87 cents. IMCR shares have lost 16.5% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 46.66%.