With the NDA target date of October approaching for roxadustat, Fibrogen Inc.'s hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) for anemia in chronic kidney disease (CKD), watchers of the space are handicapping the prospects for competitor Akebia Therapeutics Inc., developing vadadustat in the same class.
BOSTON – Panelists at Biopharm America debated strategies for early stage financing as they weighed the importance of a prestigious board, the wisdom of small deals by startups with pharma firms, and the likelihood (or not) that angel investors will help make the case with other backers yet to come.
BOSTON – The gut microbiome and its prospects for drug development have been matters of debate for a while, sharpened by the high-profile phase II failure of Seres Therapeutics Inc.'s candidate, SER-109, in the summer of 2016. A panel at Biopharm America surveyed the space in light of developments since the stumble with that candidate, composed of about 50 species of firmicutes spores derived from stool specimens from healthy donors, against recurrent Clostridium difficile infection.
BOSTON – For investors, immuno-oncology (I-O) is still proving "very hard" to buy into, said Christiana Bardon, portfolio manager at Burrage Capital, during a panel session at Biopharm America. "Honestly it was hard from the get-go. If you think about the original ipilimumab data that came from [Medarex Inc.], it was not obvious that [the drug] worked, right? It was like a head fake, actually. Now we're anxiously looking for the next-gen, but the truth is we've been utterly unsuccessful."
As Wall Street ponders how pricing and reimbursement for gene therapies might shake out, companies in the field march intrepidly on, among them Orchard Therapeutics Inc., which early this month rolled out heartening results from an ongoing proof-of-concept trial evaluating the safety and efficacy of OTL-203 for mucopolysaccharidosis type I (MPS-I), a space that's heating up.
Good news from San Diego-based Acadia Pharmaceuticals Inc.'s phase III trial with Nuplazid (pimavanserin) in dementia-related psychosis (DRP) had analysts trotting out music metaphors in reports about the study called Harmony, while talking already about would-be sales of the drug, a selective serotonin inverse agonist and antagonist that preferentially targets the 5-HT2A receptor.
Findings disclosed from Synlogic Inc.'s phase Ib/IIa trial with SYNB-1020 in hyperammonemia surprised the company and Wall Street, dealing a blow to the compound but leaving in place the Synthetic Biotic platform, which genetically engineers probiotic microbes.
Vertex Pharmaceuticals Inc.'s deal to take over Cambridge, Mass.-based Semma Therapeutics Inc. for $950 million in cash – thereby bringing aboard a potential cure for type 1 diabetes – may have brought to mind another Cambridge player in the space: Sigilon Therapeutics Inc., which inked a whopper deal with Eli Lilly and Co. in April of last year.
CEO Travis Mickle said prodrug-focused Kempharm Inc.'s potential $493 million deal with Gurnet Point Capital (GPC) marks "the end of a long journey to reach a very valuable point" in the story of the company, which has "ended with the same goal it started with" – gaining the best possible partner for attention deficit and hyperactivity disorder (ADHD) prospects KP-415 and KP-484.
