Item 7.01 Regulation FD Disclosure.
BioMarin Pharmaceutical Inc. ("BioMarin" or the "Company") is providing the
following information due to the number of individuals required to be notified
of the following matter consistent with BioMarin's regulatory obligations and to
ensure consistent notification to security holders.
On August 19, 2022, BioMarin was notified that a participant in its
valoctocogene roxaparvovec ("BMN 270") phase 3 study was diagnosed with B-cell
acute lymphoblastic leukemia (B-ALL). Based on BioMarin's assessment of the case
to date, including initial genetic testing of the leukemic cells, BioMarin
believes at this time that this cancer is unrelated to BMN 270. The overall rate
of all cancers observed in all BMN 270 trial patients (2 in approximately 400
patient years of observation) appears consistent with expected rates of cancer
in persons with Hemophilia.
Consistent with the Company's commitment to investigate any report of cancer in
its BMN 270 trial, BioMarin promptly began a comprehensive assessment of the
case. The preliminary results of genetic testing performed at the site revealed
that 85% of leukemic cells carried a Philadelphia-like (Ph-like) chromosomal
translocation, which is a well-known driver-mutation in leukemic cells. Further,
testing on leukemic cells enriched to 90% purity from peripheral blood carried
negligible levels of BMN 270 vector DNA (less than 1 copy per 500 cells). These
negligible, near background levels of BMN 270 indicate that BMN 270 is not
clonally expanding in these leukemic cells. Additional genomic analyses are
underway, which BioMarin expects to confirm the absence of BMN 270 vector
integration events contributing to leukemic growth, as well as to provide
further insights into the underlying genetic etiology of this case.
Consistent with the Company's commitments to regulatory authorities, BioMarin
submitted an IND safety report to the U.S. Food and Drug Agency ("FDA") and
communicated these findings to international health authorities on September 2,
2022. No authority has requested a hold on any trial at this time. Currently,
all trials of BMN 270 are ongoing, including additional enrollments, with no
modifications, and BioMarin does not expect to make any modifications in the
future due to this event. The Company's independent Data Safety Monitoring Board
agreed that this case did not require any modification to BioMarin's ongoing
trials at this time. BioMarin remains on track to resubmit the Biologics License
Application for BMN 270 to the FDA by the end of this month.
Forward-Looking Statements
This current report on Form 8-K contains forward-looking statements about the
business prospects of BioMarin, including, without limitation, BioMarin's belief
that the BMN 270 study patient's cancer is unrelated to BMN 270, that BioMarin
expects additional genomic analyses to confirm the absence of BMN 270 vector
integration events contributing to leukemic growth, BioMarin's expectation that
it will not make any modifications to its ongoing BMN 270 trials due to this
event, and that BioMarin remains on track to resubmit the Biologics License
Application for BMN 270 to the FDA by the end of this month.
These forward-looking statements are predictions and involve risks and
uncertainties such that actual results may differ materially from these
statements. These risks and uncertainties include, among others: results of the
ongoing additional genomic analyses of the leukemic cells, results and timing of
current and planned preclinical studies and clinical trials, and the actions of
regulatory authorities on (i) BioMarin's assessment that the BMN 270 study
patient's cancer is unrelated to BMN 270, (ii) BioMarin's ability to continue
such preclinical studies and clinical trials and (iii) the timing of such
preclinical studies and clinical trials, and the release of data from those
trials; the content and timing of decisions by the FDA, the European Commission
and other regulatory authorities concerning BMN 270; and those factors detailed
in BioMarin's filings with the Securities and Exchange Commission ("SEC"),
including, without limitation, the factors contained under the caption "Risk
Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended
June 30, 2022 as such factors may be updated by any subsequent reports.
Stockholders are urged not to place undue reliance on forward-looking
statements, which speak only as of the date hereof. BioMarin is under no
obligation, and expressly disclaims any obligation to update or alter any
forward-looking statement, whether as a result of new information, future events
or otherwise.
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