Before its unfortunate drop in quality in its last few seasons, the medical drama House was one of my favorite shows, with its title character, a cranky, possibly drug-addicted doctor solving diagnostic riddles and saving lives with Sherlockian brilliance, and quite often breaking every rule in the book to do it. One episode in particular had House facing off with billionaire investor Vogel, who had thwarted his attempt to enroll a very sick patient in a clinical trial without mentioning to the trial investigator that she wouldn’t fit the criteria, namely having just undergone a C-section. They have an impassioned exchange:...
After enduring a social media firestorm this week vilifying the firm for not providing its antiviral candidate brincidofovir to a dying 7-year-old boy on a compassionate use basis, Chimerix Inc. found a way to get the drug to Josh Hardy without setting a potentially dangerous ethical precedent.
Patients with spinal muscular atrophy (SMA) and their families, who rejoiced two weeks ago when Isis Pharmaceuticals Inc. posted positive phase Ia/IIb data with its antisense candidate, got even better news Monday: French biopharma Trophos SA scored positive pivotal results with neuroprotective candidate olesoxime, putting it on track to file what could become the first treatment specifically for SMA.
After a three-week respite – giving IPO-weary investment bankers and industry analysts a chance to catch up after the rapid succession of pricings in early February – Aquinox Pharmaceuticals Inc. and Recro Pharma Inc. priced initial public offerings Friday as expected, and both made solid if not stunning debuts despite the mystery biopharma sell-off of late last week.
The bad news is that Xoma Corp. said its interleukin-1 beta (IL-1 beta) antibody gevokizumab failed to offer compelling enough data in a phase II study in erosive osteoarthritis (EOA) to warrant phase III investment in that indication, sending the company’s shares down 23 percent Wednesday.
Two more biopharmas joined the initial public offering (IPO) queue late last week – Scynexis Inc. aiming to raise up to $55 million for its fungal infection pipeline and cancer firm Ignyta Inc. filing to raise up to $40 million – and, after two quiet weeks on the IPO front, two firms are expected to make their debuts this week.
On the heels of disclosing a planned 2014 R&D investment hike of more than 60 percent over last year, Compugen Ltd. took the opportunity to pad its coffers with a $63 million public offering.
Scholar Rock Inc. CEO Nagesh Mahanthappa’s previous biopharma experiences include stints at RNAi powerhouse Alnylam Pharmaceuticals Inc. and privately held Avila Therapeutics Inc., which was snapped up by Celgene Corp. in a potential $935 million deal in 2012, and he is the first to admit that “those are high standards.”
Barely a month after Celladon Corp. priced its modest initial public offering (IPO), the San Diego-based firm saw its shares jump 25.5 percent Monday on news of an option deal with French pharma firm Servier based on SERCA2b modulators for metabolic disease.
As Biomarin Pharmaceutical Inc. preps for U.S. commercial launch of recently approved mucopolysaccharidosis Type IVA drug Vimizim (elosulfase alfa) and awaits a decision expected Friday from the European Committee for Medicinal Products for Human Use (CHMP), investors have focused largely on the high pricing for Vimizim – about $380,000 per year – and the expected hike in R&D expenses for 2014.
