22:29 , Mar 21, 2018 |  BC Extra  |  Company News

Weather delay for achondroplasia meeting

FDA postponed until further notice the March 22 joint meeting of the Pediatric Advisory Committee (PAC) and Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) due to inclement weather. The committees are slated to discuss drug...
21:45 , May 12, 2017 |  BioCentury  |  Strategy

Vertical vision

Shire plc’s deal with Parion Sciences Inc. is a step toward building vertically in ophthalmics, much like it has done in hereditary angioedema and hemophilia. On May 1, Shire gained exclusive, worldwide rights to P-321...
22:23 , Apr 6, 2017 |  BC Innovations  |  Targets & Mechanisms

Competing for growth

With little in the clinical pipeline for achondroplasia beyond BioMarin Pharmaceutical Inc.’s late-stage vosoritide, startups Therachon AG and BioClin Therapeutics Inc. are rethinking how to tackle the disease. By targeting FGFR3 directly, the two companies...
23:02 , Dec 14, 2016 |  BC Week In Review  |  Clinical News

Vosoritide: Ph III started

BioMarin began a double-blind, placebo-controlled, international Phase III trial to evaluate daily vosoritide for 52 weeks in about 110 patients ages 5-14. Patients who complete the trial may be eligible to enroll in an open-label...
01:17 , Apr 21, 2016 |  BC Extra  |  Clinical News

BioMarin updates plans for three late-stage programs

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) gave R&D updates on three late-stage programs: achondroplasia therapy vosoritide ( BMN 111), phenylketonuria (PKU) treatment pegvaliase ( BMN 165), and cerliponase alfa ( BMN 190) to treat late-infantile neuronal ceroid...
07:00 , Mar 14, 2016 |  BC Week In Review  |  Company News

Chugai, BioMarin deal

Chugai granted BioMarin U.S. and Japanese rights to Chugai’s C-type natriuretic peptide ( CNP; NPPC) patent, which covers BioMarin’s achondroplasia candidate vosoritide. The analog of CNP is in Phase II testing to...
08:00 , Jan 11, 2016 |  BioCentury  |  Finance

Sowing season

Stephen Hansen, Associate Editor   BUYSIDE VIEW XXIV While the biotech sector's three-year rocket ride isn't likely to continue in 2016, fund managers are still anticipating a slew of high-profile milestones in the New Year. This year...
07:00 , Oct 5, 2015 |  BioCentury  |  Finance

Back to the bone

In one of his first investments since joining OrbiMed Advisors in January, Stephen Squinto is returning to a space he knows well - biologics for an Orphan bone disease. The result was OrbiMed leading last...
07:00 , Jul 13, 2015 |  BioCentury  |  Finance

Big news

The performance of big cap biotechs has lagged that of companies one tier down, but the second half includes a host of milestones that could put the bellwethers on a steeper upward trajectory. Buysiders...
07:00 , Jun 22, 2015 |  BC Week In Review  |  Clinical News

Vosoritide: Phase II data

An open-label, dose-escalation, international Phase II trial in 26 patients ages 5-14 with achondroplasia showed that once-daily 7.5 and 15 ug/kg subcutaneous BMN 111 improved mean growth velocity by 1.3 and 2 cm/year, respectively, from...