Biomarin Pharmaceutical Inc Stock Price, News & Analysis

BioMarin Pharmaceutical announced positive results from the Phase 3 extension study of vosoritide, a treatment for children with achondroplasia. Following two years of treatment, children showed maintained growth acceleration with a cumulative height gain of 3.52 cm compared to untreated subjects. The treatment was well tolerated, with 93% of participants remaining on it. Regulatory applications for vosoritide are under review by the FDA and EMA, with potential approval dates in August and the second half of 2021, respectively. This could mark the first therapy for achondroplasia in these regions.

BioMarin Pharmaceutical (NASDAQ: BMRN) announced its participation in two upcoming virtual investor conferences. An audio webcast of the presentations will be available for live access and later archived on the company's website. BioMarin focuses on developing innovative therapies for life-threatening rare disorders, boasting a portfolio that includes six commercialized products along with several clinical and pre-clinical candidates. For further details, please visit www.biomarin.com.

BioMarin Pharmaceutical (BMRN) announced a preclinical collaboration with Deep Genomics on November 17, 2020, to enhance drug discovery using AI technology. The partnership focuses on identifying oligonucleotide drug candidates for four rare diseases with significant unmet needs. Financial terms remain undisclosed, but Deep Genomics will receive an upfront payment and potential development milestones. This collaboration aims to leverage AI for faster identification and validation of therapeutic candidates, aiming to expedite BioMarin's development pipeline in rare disease treatments.

BioMarin Pharmaceutical (NASDAQ: BMRN) announced its participation in three upcoming virtual investor conferences on November 12, 2020. Management will be presenting, and an audio webcast will be available live on their website. Archived versions of the presentations will be accessible for a limited time post-conference. BioMarin focuses on developing innovative therapies for serious rare disorders and has a strong portfolio with six commercialized products and ongoing clinical candidates. Further details can be found on their official website.

BioMarin Pharmaceutical (NASDAQ:BMRN) has announced the expansion of its clinical program for vosoritide with two new Phase 2 studies targeting children with achondroplasia and other genetic forms of short stature. The first study will assess the safety of vosoritide in infants at risk of life-threatening foramen magnum compression. The second, an investigator-initiated study by Children's National Hospital, will explore vosoritide’s effects on growth in selected genetic short stature patients. Both studies aim to enroll a significant number of participants, aiming to enhance treatment outcomes.

BioMarin Pharmaceutical reported Q3 2020 financial results with total revenues of $476.8 million, up by 3% from Q3 2019. Net product revenues increased by 2% to $460.7 million. The company achieved a substantial GAAP net income of $784.8 million, largely due to a $800.8 million tax benefit related to IP transfer. However, revenues from products like Vimizim and Naglazyme declined. Despite COVID-19 challenges affecting revenues, BioMarin remains optimistic about upcoming regulatory filings for its gene therapies, valoctocogene roxaparvovec and vosoritide. Cash reserves stood at $1.77 billion.

BioMarin Pharmaceutical (NASDAQ: BMRN) announced that its CEO, Jean-Jacques Bienaimé, and CFO, Brian Mueller, will participate in the Credit Suisse 29th Annual Virtual Healthcare Conference on November 10, 2020, at 12:30 PM ET. An audio webcast will be available live and can be accessed here. An archived version will also be accessible on the company's website afterward. BioMarin develops innovative therapies for serious rare disorders and has six commercialized products along with several candidates in the pipeline.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced that the FDA has accepted its New Drug Application (NDA) for vosoritide, a treatment for achondroplasia, marking the first marketing application for this condition in the U.S. The PDUFA action date is set for Aug. 20, 2021. The company believes that the outcomes from its prior clinical trials support the application's validity. Additionally, BioMarin is nearing completion of enrollment for a Phase 2 study involving infants with achondroplasia, focusing on safety and height Z-scores.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will host a conference call and webcast on November 5, 2020, at 4:30 p.m. ET to discuss its third quarter 2020 financial results and provide a business update. The call can be accessed via U.S. and international dial-in numbers, with a conference ID of 3291898. A replay will be available for one week after the call. BioMarin focuses on innovative therapies for serious rare genetic diseases, offering six commercial products and various clinical candidates.