After BioMarin’s new leadership conducted a “strategic R&D asset review,” three of its “most productive” assets are being prioritized, leaving four candidates on the wayside.
First up on the discard pile is BMN 331, a gene therapy being assessed in a phase 1/2 trial for patients with hereditary angioedema, a rare inherited swelling condition. The open-label, dose-escalation study was in the recruitment stage, with a primary completion date slated for 2028, according to ClinicalTrials.gov.
Next up is BMN 255, an oral phase 1b program designed to tackle the tricky indication of metabolic dysfunction-associated steatohepatitis (MASH). The trial launched in September 2023 and had an expected completion date of October of this year.
Third on the discontinued list is BMN 355, a preclinical candidate BioMarin publicly rolled out in September 2023. The monoclonal antibody was designed to treat long-QT syndrome types 2 and 3, which is a deficiency that prevents appropriate electrical function in the heart and can lead to abnormal heart rhythms. At the time, BioMarin had planned to launch a global trial for BMN 355 in 2025.
BMN 365, just freshly revealed to the public in September 2023, is the last asset to get the boot. The candidate was targeting plakophilin-2 mutations and arrhythmogenic right ventricular dysplasia/cardiomyopathy. Patients with the condition can experience irregular heartbeat and even sudden death. BioMarin had hoped to administer a replacement gene to form a protein that can help overcome the genetic deficiencies and, if successful, reverse irregular heartbeats, also known as arrhythmia, to restore cardiac function.
None of the axed programs were discontinued because of safety signals, according to an April 25 company release.
That leaves three early- and mid-stage candidates that were chosen by the company “with the combined focus on patient impact and commercial opportunity,” according to the release.
The remaining programs include BMN 333, a preclinical C-type natriuretic peptide (CNP) as a potential treatment for multiple growth disorders.
Also still standing is BMN 349, a phase 1 oral small molecule for alpha-1 antitrypsin deficiency-associated liver disease. Though BioMarin lists the asset as a phase 1 program, the candidate is not yet registered in a study on ClinicalTrials.gov.
Last up is BMN 351, BioMarin's next-gen oligonucleotide for patients with Duchenne muscular dystrophy. Investigational new drug-enabling studies for the therapy wrapped up last year, and a phase 1/2 trial for the asset launched at the beginning of this year. The open-label trial has an estimated enrollment of 18 patients with genetic mutations amenable to exon 51 skipping and a completion date at the end of 2025.
All three prioritized programs “met the highest bar for advancement,” according to BioMarin.
“This approach to delivering innovative therapies is an important part of BioMarin's operational transformation, and we look forward to sharing more details at Investor Day on September 4th,” BioMarin CEO Alexander Hardy, said in the April 25 release. Hardy, the former CEO of Roche’s Genentech, took the reins of BioMarin late last year, succeeding Jean-Jacques Bienaimé after his 18 years at the helm.
In his short time with BioMarin, Hardy has overhauled the company’s pipeline and the leadership team, replacing longtime commercial chief Jeffrey Ajer with Cristin Hubbard, who previously led global product strategy for Roche’s pharma group.
BioMarin’s management team expects the pipeline restructure will save up to $60 million in R&D costs for 2024, offset by up to $20 million that will be spent on accelerating the prioritized programs.
Despite the pipeline reductions, analysts remain bullish.
“We are encouraged to see new management’s initiative to prioritize therapies with the highest commercial potential,” William Blair analysts wrote in an April 25 note. “With a promising growth profile and a renewed focus on profitability, we view BioMarin as a core holding in the sector and rate shares ‘Outperform.’”
William Blair noted the “impressive launch of Voxzogo, the company’s largest approved product to date, and a new CEO focused on expanding margins.”
Voxzogo snagged FDA approval in achondroplasia—a genetic disorder that impairs bone growth and is the most common form of disproportionately short height—in late 2021.
Meanwhile, BioMarin reported $0.8 million in Roctavian revenue for the first quarter of 2024. The one-time gene therapy received FDA approval in June 2023 for adults with severe hemophilia A. However, the launch has been disappointing, with management noting that reimbursement and market access challenges continue to impact the ability of interested patients to receive treatment with Roctavian.
Since opening at $92.69 per share today, BioMarin’s stock has slipped to $87 as of 9:15 a.m. ET.