Akash Tewari

Cory Kasimov

Olivia Brayer

Paul Matteis

Jessica Fye

Konstantinos Biliouris

Philip Nadeau

Salveen Richter

Adam Ferrari

Alexandria Hammond

Allison Bratzel

Andrea Park

Christopher Raymond

Eliana Merle

Ellen Horste

Gena Wang

Huidong Wang

Jasmine Fels

Jason Gerberry

Joseph Schwartz

Kostas Biliouris

Mohit Bansal

Sean Laaman

Tommie Reerink

Alex Hammond

Ellie Merle

John Wang

Julian Pino

Sadia Rehman

Vikram Purohit

- RIBOMIC, Inc. announced positive Phase 2 clinical trial results for **Umedaptanib Pegol** in pediatric patients with Achondroplasia (ACH), demonstrating an increased annualized growth rate of up to **+5.0 cm/year** in Cohort 2.  **[1048477_dd3202eb6eee4266948ce04f1532dacf_0]** 
- The results for some patients in Cohort 2, with increases of +5.0 cm/year and +2.0 cm/year, surpassed the average growth rate of **+1.7 cm/year** for the currently approved drug Voxzogo®.  **[1048477_dd3202eb6eee4266948ce04f1532dacf_0]** 
- No safety concerns were identified during the Phase 2 study, and Umedaptanib Pegol successfully demonstrated proof-of-concept for ACH treatment.  **[1048477_dd3202eb6eee4266948ce04f1532dacf_0]** **[1048477_dd3202eb6eee4266948ce04f1532dacf_1]** 
- RIBOMIC plans to initiate a Phase 3 study in **Q1 FY2026**, aiming for completion in **FY2027**, with regulatory approval targeted by the **end of FY2028**.  **[1048477_dd3202eb6eee4266948ce04f1532dacf_1]**

RIBOMIC Reports Positive Phase 2 Results for Umedaptanib Pegol in Achondroplasia

- BioMarin Pharmaceutical Inc. expects to report **estimated acquired In-Process Research and Development (IPR&D) charges** for the **third quarter of 2025**.  **[1048477_0001048477-25-000101_bmrn-20251007.htm:1]** 
- These pre-tax charges are estimated at **approximately $221 million**.  **[1048477_0001048477-25-000101_bmrn-20251007.htm:1]** 
- The charges are projected to impact both **GAAP Diluted EPS and Non-GAAP Diluted EPS by approximately $1.10**.  **[1048477_0001048477-25-000101_bmrn-20251007.htm:1]** 
- These charges are related to the previously announced **acquisition of Inozyme Pharma, Inc. on July 1, 2025**.  **[1048477_0001048477-25-000101_bmrn-20251007.htm:1]** 
- The reported financial results for the quarter ended September 30, 2025, are **preliminary and unaudited**, subject to BioMarin's financial statement closing procedures.  **[1048477_0001048477-25-000101_bmrn-20251007.htm:1]**

BioMarin Pharmaceutical Inc. Announces Estimated Q3 2025 Acquisition-Related Charges

- RIBOMIC, Inc. announced positive **Phase 2 Cohort 2 results** for umedaptanib pegol in pediatric patients (ages 5-14) with achondroplasia (ACH).  **[1048477_0c07077da6e949fd93008aa1d02714ed_0]** 
- The trial demonstrated an increase in the annualized growth rate of up to **+5.0 cm/year** with biweekly administration.  **[1048477_0c07077da6e949fd93008aa1d02714ed_0]** 
- In Cohort 2, **5 out of 6 subjects** showed increased height growth rates, with two subjects achieving significant increases of **+5.0 cm/year** and **+2.0 cm/year**, which exceeded the average growth rate of **+1.7 cm/year** for BioMarin's approved drug, Voxzogo®.  **[1048477_0c07077da6e949fd93008aa1d02714ed_0]** 
- The results established **proof-of-concept (POC)** for umedaptanib pegol as an ACH treatment, with no safety concerns observed throughout the Phase 2 clinical trial.  **[1048477_0c07077da6e949fd93008aa1d02714ed_0]** 
- RIBOMIC plans to initiate a **Phase 3 clinical trial** in **Q1 FY2026** and aims for regulatory approval by the **end of FY2028**, leveraging its orphan drug designation in Japan.  **[1048477_0c07077da6e949fd93008aa1d02714ed_1]**

RIBOMIC Announces Positive Phase 2 Results for Umedaptanib Pegol in Achondroplasia

- BioMarin Pharmaceutical Inc. is prioritizing its **BMN 333 program** for achondroplasia, with a combined Phase 2/3 study planned to start in patients in the **first half of next year**, aiming for superiority over Voxzogo.  **[1048477_tx_379960_0]** **[1048477_tx_379960_4]** **[1048477_tx_379960_5]** 
- The company expects to release **hypochondroplasia data for Voxzogo** and **BMN 401 (ENPP1 deficiency) ENERGY3 study data** in the **first half of next year** and **first quarter of next year**, respectively.  **[1048477_tx_379960_0]** **[1048477_tx_379960_7]** 
- BioMarin is also filing for **Palynziq in adolescents** in the U.S. and Europe, representing a significant opportunity to expand the label.  **[1048477_tx_379960_0]** 
- The company is actively pursuing **business development opportunities** for both early and late-stage assets, leveraging its **cash position** and focusing on genetically defined conditions, skeletal conditions, and enzyme replacement therapies.  **[1048477_tx_379960_0]** **[1048477_tx_379960_1]**

BioMarin Pharmaceutical Inc. Discusses Key R&D Priorities and Pipeline Progress

BIOMARIN PHARMACEUTICAL (BMRN)