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.
Shares of San Diego-based Cidara Therapeutics Inc. (NASDAQ:CDTX) enjoyed a skyward ride, closing Tuesday at $2.35, up 67 cents, or 40%, on word of a deal with Mundipharma International Ltd., of Cambridge, U.K., to develop and commercialize rezafungin for invasive fungal infections. "Historically, [antifungal] pricing has been much stronger in the EU, so that is an important opportunity for us and now for Mundipharma as well," Cidara CEO Jeffrey Stein said.
Merus N.V.'s second-quarter earnings report included an important update on phase I MCLA-117, which Jefferies analyst Maury Raycroft said could be a best-in-class "game-changer" for the treatment of acute myeloid leukemia (AML) – a candidate that "alone is a reason to own the stock," in his view.
Newark, Calif.-based Protagonist Therapeutics Inc.'s third-quarter earnings call included an update on hepcidin mimetic PTG-300 in beta-thalassemia, but other prospects in the pipeline are starting to excite investors as well.
