Before we dig in, we wanted to flag our recent webcast ft. Chris Senyek that had our illustrative US projections of total # of 1) diagnosed cases 2) deaths and case peak: (April-May) due to COVID-19 that’s generated a lot of positive feedback.
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Over the last few days we’ve been getting a fair amount of Q’s on HCQ in COVID-19, especially after updated data from the French Gautret group this weekend (we have our reservations on trusting anything from that group at this point).
Today BMRN just posted another ph.3b trial, (primary completion Dec 2021) for Valrox that we wanted to quickly flag. This open-label trial will study the efficacy of prophy steroids & Valrox at a 6E13 dose. Additionally, the trial will study the safety including liver inflammation and generation of anti-F8 antibodies.
We want to be very clear: our report today is NOT meant to make light of the seriousness of COVID-19. In fact, our work illustrates how important it is to follow smart practices such as social distancing & not “reopen” the country too soon (our base scenario assumes a “lockdown” through April& 1H May). Smart policy decisions over the next few weeks will absolutely save lives.
Today at 10:30AM EST we will be doing a live webcast on the therapeutic approaches being worked on to fight the Coronavirus, including what's 'hype' and what's 'reality' + the impt. catalysts to track going forward.
Yesterday (3/16/20), an article came out of China that noted Chinese researchers have completed some clinical studies of Favirpiravir (an antiviral) which showed that treated pts recovered from COVID-19 more quickly, and their lung conditions were better improved than the control group.
BMRN just reported inline Q419 results + 2020 guidance, here’s what stood out to us
As we head into Q120 – we’ve started getting more Q’s on Valrox & street expectations, with some investors playing for a better than expected launch after mgmt’s bullish commentary at JPM (please reach out if you’d like to discuss).
FIXX just announced preliminary data from its ph1/2 (potentially registrational) trial for PKU Gene Therapy. For those interested in digging into both BMRN & FIXX’s competing programs please check out our deep dive. We believe that PKU remains an interesting target biologically for GT & will become a larger debate for BMRN’s stock overtime
BMRN'S vosoritide Ph.3 read out positively + showed a stat. sig 1.6 cm/yr improvement in pbo-adj. ΔAGV after 1-yr of tx and a generally well tolerated safety profile...this result is generally what we and other investors expected and is a net positive for BMRN.
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