Biomarin Pharmaceutical (BMRN) Stock News

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) will present positive data from the CANOPY clinical program evaluating VOXZOGO® (vosoritide) at the 16th International Skeletal Dysplasia Society meeting. The data shows:

• Meaningful improvements in health-related quality of life for children with achondroplasia
• Increased bone length while maintaining bone strength
• Sustained growth gains in children with hypochondroplasia, Noonan syndrome, and genetic variants associated with idiopathic short stature

VOXZOGO is becoming the standard of care in achondroplasia, with proven effects on growth velocity, safety profile, and positive impact on proportionality and quality of life. The drug shows promise in treating other genetic skeletal conditions, with ongoing research demonstrating efficacy and safety consistent with its profile in achondroplasia.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) hosted an Investor Day, unveiling a new corporate strategy and financial guidance for 2027. The company aims to deliver sustained value creation through a restructured organization built around three business units: Enzyme Therapies, Skeletal Conditions, and ROCTAVIAN®. BioMarin plans to implement a $500 million cost transformation program to support Non-GAAP Operating margin targets.

Key financial projections include:

• Total Revenues of $4 billion by 2027
• Non-GAAP Operating Margin in the low-to-mid 40%s starting with 40% in 2026
• Operating Cash flow of $1.25 billion+ by 2027

The company reaffirmed its 2024 guidance, projecting Total Revenues of $2.750B to $2.825B and Non-GAAP Operating Margin of 26% to 27%.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is hosting its Investor Day on September 4, 2024, in New York, starting at 10:30 a.m. Eastern Time. The event, led by President and CEO Alexander Hardy, will showcase the company's new strategic priorities aimed at delivering significant value creation.

Key presentations include:

• BioMarin's new corporate strategy for sustained value creation
• Financial strategy for high growth and superior returns
• Innovation strategy for a sustainable pipeline of high-impact medicines
• Commercial growth strategy, focusing on enzyme therapies and leadership in achondroplasia

The event will be accessible virtually, with a live webcast, replay, and presentation slides available on BioMarin's Investor Relations website for at least 12 months.

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.