BioMarin (BMRN) PT Trimmed at Wedbush Following Briefing Docs; Sees Positive Read-Thru to SRPT, None to PTCT

Wedbush analyst Heather Behanna lowered her price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to $125.00 (from $137.00) following today's FDA briefing documents. The firm maintained a Neutral rating.

Behanna said the briefing docs for drisapersen efficacy were in line with their expectation. Meanwhile, the FDA has unleashed its view point on BioMarin's drisapersen for DMD including the focus on potential unblinding of studies due to injection site reactions. "Things that were new to us include analysis that boys in the 6 mg/kg continuous arm of the one successful 24-week Phase II study may have been healthier than the other cohorts, confounding data even further. The other interesting fact, we believe, is that the open-label study of 12 boys enrolled patients who had unusually well-preserved function at baseline, suggesting, in our view, that the performance of these boys could be unrelated to drug; we note that two boys in this study lost ambulation after one year of treatment."

The analyst said the lack of incorporation of FDA feedback is apparent.

On the read-through to Sarepta Therapeutics (NASDAQ: SRPT), the analyst said:" In our view, the focus on dystrophin in the briefing documents (and lack of dystrophin production for drisapersen) is a good sign for Sarepta, as the agency appears to be focused on biomarkers for accelerated approval as is the risk/reward for eteplirsen considering its benign profile to date. In our view, the adherence to FDA guidance with regard to natural history controls and confirmatory studies is a plus for Sarepta. We believe approval of eteplirsen is not a slam dunk at this time; however, we see the overall prospects as positive for Sarepta's approach and its panel in January."

On the read-through to PTC Therapeutics (NASDAQ: PTCT), the analyst said: "We do not see a huge read-through from these documents to PTC's ataluren. We believe FDA focus on secondary endpoints is a positive for PTC, as it highlights the body of data that support a drug effect, in contrast to drisapersen data where this is not the case. We note FDA is looking at each individual study for drisapersen to see if they support the others -- if this is done for PTC it would put less focus on the meta-analysis; however, in contrast to drisapersen's data, we believe the two ataluren studies support each other and a drug effect. The one negative read-through is the agency's focus on dystrophin; as a reminder, PTC has limited dystrophin data and did not have biopsies collected in its pivotal trials."

Commenting on what it all means, the analyst said: "We do not see a huge read-through from these documents to PTC's ataluren. We believe FDA focus on secondary endpoints is a positive for PTC, as it highlights the body of data that support a drug effect, in contrast to drisapersen data where this is not the case. We note FDA is looking at each individual study for drisapersen to see if they support the others -- if this is done for PTC it would put less focus on the meta-analysis; however, in contrast to drisapersen's data, we believe the two ataluren studies support each other and a drug effect. The one negative read-through is the agency's focus on dystrophin; as a reminder, PTC has limited dystrophin data and did not have biopsies collected in its pivotal trials."

For an analyst ratings summary and ratings history on BioMarin Pharmaceutical Inc. click here. For more ratings news on BioMarin Pharmaceutical Inc. click here.

Shares of BioMarin Pharmaceutical Inc. closed at $103.11 yesterday.

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