Ultragenyx (RARE) Posts Data From Osteogenesis Imperfecta Study
Ultragenyx Pharmaceuticals RARE, along with partner Mereo BioPharma MREO, announced positive data from the mid-stage of setrusumab for patients with osteogenesis imperfecta. Shares of RARE and MREO were up almost 5% and 12%, respectively, after market hours on the news.
The phase II dose-selection portion of the phase II/III, Orbit study data revealed that the drug candidate setrusumab rapidly induces bone production in patients affected by osteogenesis imperfecta (OI).
The study included 24 patients who were randomized to receive different doses of setrusumab. The results showed that treatment with the drug candidate leads to a significant improvement in bone mineral density (BMD) by three months and statistically major increase in serum P1NP levels, a bone formation marker.
The patients in the 20 mg/kg cohort experienced a mean serum P1NP increase of 57% from baseline over the first month, representing a significant improvement in bone formation.
Notably, the increase in serum P1NP levels in pediatric and adolescent patients was approximately eight times higher than in adult OI patients, highlighting the potential benefits for younger patients with developing bones.
Treatment with setrusumab for three months resulted in a substantial increase in lumbar spine BMD from baseline, with a 9.4% increase in the 20 mg/kg cohort. This indicated a substantial improvement in bone health.
Ultragenyx’s shares have risen 10.6% year to date against the industry’s 7.7% decline.
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OI is a group of genetic disorders that impacts bone metabolism. It is characterized by increased bone fragility with a high risk of fractures. The condition affects approximately 60,000 people in the developed world. Currently, there are no approved treatments for this debilitating condition.
The positive results from the phase II portion of the study provided valuable data for dose selection. RARE has started patient screening for the phase III portion of the study.
The phase III portion aims to enroll approximately 195 patients across 12 countries. The study will evaluate the effect of setrusumab versus placebo on the fracture rate in pediatric and young adult patients with OI.
The positive outcomes from the phase II portion support the selection of the 20 mg/kg dose for the phase III program.
Ultragenyx Pharmaceutical Inc. Price and Consensus
Ultragenyx Pharmaceutical Inc. price-consensus-chart | Ultragenyx Pharmaceutical Inc. Quote
Zacks Rank and Stocks to Consider
Currently, Ultragenyx carries a Zacks Rank #3 (Hold).
A couple of better-ranked stocks in the biotech sector are Akero Therapeutics AKRO and ADMA Biologics, Inc. ADMA, both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Loss per share estimate for Akero Therapeutics has narrowed from $3.46 to $2.78 for 2023 in the past 90 days. Shares of Akero Therapeutics have nosedived 5.1% year to date.
AKRO’s earnings beat estimates in three of the trailing four quarters and missed the mark in one, delivering an average surprise of 7.96%.
Loss per share estimate for ADMA Biologics has narrowed from 19 cents to 9 cents for 2023 in the past 90 days. In the year so far, shares of ADMA Biologics have risen 4.4%.
ADMA’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 19.13%.
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