Biomarin Pharmaceutical Inc Stock Price, News & Analysis

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced that its Executive Vice President and Chief Financial Officer, Brian Mueller, will be presenting at the 22nd Annual Morgan Stanley Global Healthcare Conference. The presentation is scheduled for Thursday, September 5, 2024, at 7:45 AM PT / 10:45 AM ET in New York, NY.

Investors and interested parties can access a live audio webcast of the presentation through the company's website at https://investors.biomarin.com/. For those unable to attend or listen live, an archived version of the remarks will be available on the company's website for a time following the conference.

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announces key leadership changes in R&D and Business Development. Greg Friberg, M.D., joins as EVP, Chief Research & Development Officer on Sept. 30, succeeding Hank Fuchs, M.D., who retires after 15 years. James Sabry, M.D., Ph.D., becomes EVP, Chief Business Officer on Oct. 7. Dr. Friberg, from Amgen, brings extensive experience in clinical development and lifecycle management. Dr. Sabry, from Roche and Genentech, is known for his expertise in business development, having led 1,200 transactions.

Dr. Fuchs, retiring after overseeing the development of five new medicines, will remain in an advisory capacity until March 3, 2025. Brinda Balakrishnan, M.D., Ph.D., Chief Corporate Strategy and Business Development Officer, is leaving the company on Oct. 1 after eight years of service.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced its upcoming Investor Day scheduled for September 4, 2024, in New York City. The event, running from 10:30 am to 1:00 pm Eastern Time, will be accessible via live webcast. Key highlights include:

• Presentations from executive management team
• Focus on BioMarin's pipeline and strategic plans
• Insights into growth opportunities
• Discussion of long-term financial outlook

Investors and analysts can access the webcast and presentation materials through the company's investor relations website. A replay will be available shortly after the event concludes, providing an opportunity for those unable to attend live to catch up on the important updates shared during the Investor Day.

BioMarin reported a strong financial performance for Q2 2024, with total revenues reaching $712 million, a 20% increase year-over-year (Y/Y). The company also raised its full-year 2024 guidance. Key drivers include strong global demand for VOXZOGO, which saw a 73% Y/Y increase in the number of children receiving treatment, and robust performance of enzyme therapies. GAAP diluted EPS for the quarter was $0.55, a 90% Y/Y increase, while Non-GAAP diluted EPS was $0.96, up 78% Y/Y.

VOXZOGO contributions were significant, with nearly 900 new patient starts in the first half of 2024. BioMarin's enzyme therapies also showed strong growth, partially offset by lower revenues from KUVAN due to generic competition. GAAP net income for Q2 2024 increased by $51.2 million to $107.2 million. Non-GAAP income rose by $83.7 million to $188.9 million. Financial guidance for 2024 was updated to reflect these strong results.

BioMarin continued to make progress in its clinical programs and strategic priorities, including its supply chain efforts and development of new indications for VOXZOGO.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced a strategic shift for its hemophilia A treatment, ROCTAVIAN®. The company will focus commercial operations on the United States, Germany, and Italy, where the medicine is approved and reimbursed. This move aims to reduce annual direct ROCTAVIAN expenses to approximately $60 million by 2025, with the goal of achieving profitability for the product by the end of that year.

Key changes include:

• Reducing investments in development and manufacturing
• Focusing on markets where ROCTAVIAN is approved and reimbursed
• Placing the gene therapy manufacturing facility in an idle state
• Discontinuing enrollment in new clinical development programs
• Continuing support for patients already treated with ROCTAVIAN

BioMarin remains committed to ROCTAVIAN as an important option for people with severe hemophilia A, citing its potential for years of bleed control after a single treatment.

The FDA has approved BioMarin's supplemental Biologics License Application (sBLA) for BRINEURA® (cerliponase alfa) to treat children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), including those under 3 years, regardless of symptom presence. Previously, BRINEURA was only indicated for symptomatic children aged 3 and above. The approval is based on data from Study 190-203, a Phase 2 trial that showed BRINEURA slows motor function decline and delays disease onset in children aged 1-6, including those under 3. In this study, none of the treated children under 3 experienced a significant motor decline, compared to 61% of untreated children in a natural history cohort. The safety profile for children under 3 is consistent with the known safety profile of the drug. This expanded indication emphasizes the importance of early diagnosis and treatment for CLN2 disease.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) has announced that it will host a conference call and webcast on Monday, August 5, 2024, at 4:30 p.m. ET to discuss its second quarter 2024 financial results and provide a general business update. The call will be hosted by Alexander Hardy, President and Chief Executive Officer of BioMarin.

Participants can join the call using the following details:

• U.S. / Canada Dial-in: 888-596-4144
• International Dial-in: 646-968-2525
• Conference Call ID: 1816377

BioMarin announced new data from its Phase 2 111-205 study showing that children with achondroplasia treated with VOXZOGO® exhibited significant increases in bone length while maintaining bone strength over five years. The results will be presented at the International Conference on Children's Bone Health in June 2024. Additional Phase 3 data reveal VOXZOGO's positive impacts on proportionality and health-related quality of life. Separately, research on hypochondroplasia highlights higher comorbidity rates, underscoring unmet medical needs. BioMarin is advancing clinical trials for hypochondroplasia, idiopathic short stature, and other growth conditions.

BioMarin presented four-year data from its Phase 3 study on ROCTAVIAN® at the ISTH 2024 Congress, showing long-term safety and efficacy in treating severe hemophilia A. The study revealed that 73.6% of patients had zero treated bleeds in year four, with stable FVIII levels. Mean FVIII activity was 27.1 and 16.1 IU/dL by OSA and CSA assays, respectively. Additionally, there was a notable improvement in health-related quality of life (HRQoL), with a 6.2 point increase in Haemo-QOL-A Total Score. Phase 2 results also showed seven-year bleed control for a majority of patients.