Biomarin (BMRN) recently delivered solid Q3:21 total revenue of $409 million — a bit below the consensus estimate of $435 million as orders for Vimizim and Naglazyme are concentrated in the first half, notes John McCamant, biotech sector specialist and editor of The Medical Technology Stock Letter.

However, the EU Voxzogo launch is underway with indications pointing to rather strong patient demand that have surpassed the Street’s expectations. Up next for Voxzogo is the FDA action date (Nov 20), where a positive outcome would set BMRN up for its largest product launch to date. 

Another important upcoming regulatory decision for BMRN will be Roctavian where we expect two-year Phase III data in Q1:22 followed by a US regulatory resubmission in Q2:22. 

At the upcoming R&D day (Nov 30), the early stage pipeline will also be in focus, including updates on Voxzogo in additional indications, BMN331 (hereditary angioedema gene therapy), BMN255 in chronic renal diseases, and BMN351 in Duchenne muscular dystrophy.

An upcoming R&D day is planned for November 30 to highlight the early pipeline:

  1. BMN-255 (small molecule for subset of chronic renal disease).
  2. IND for BMN255 is active and dosing is underway in subjects.
  3. BMN-331 (HAE gene therapy): The IND has been allowed and the first patient is expected to be enrolled near YE21.
  4. DiNA-001: Preclinical studies evaluating the therapy for MYBPC3 hypertrophic cardiomyopathy are underway – BMRN expects to select a candidate vector in 2021.
  5. BMN-351 (DMD antisense therapy): IND-enabling studies demonstrated dystrophin protein expression levels between 30% and 50% of normal levels in a DMD mouse model treated for at least 13 weeks. BMRN is on track to file an IND in 1H22

The company has been in active discussions with the agency on next steps after the recent clinical trial hold for BMN307 (PKU gene therapy) due to interim preclinical observations from a 52-week PKU mouse model.

Biomarin is investigating the preclinical findings and evidence of liver tumors from integration of portions of AAV vector into the genome. Management noted it believes the requests from the agency are straightforward and can be addressed relatively easily.

Importantly, management reiterated its view that there is no read-through to the rest of the firm's gene therapy portfolio and specifically no impact to Roctavian’s potential approval, currently under review with the EMA and FDA submission based on two-year data expected in Q2:22. BMRN is a buy under $100 with a new target price of $150.

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