PHILADELPHIA – Compared to oncology, chronic diseases such as cardiovascular disease, diabetes and dementia represent much more substantial costs to the overall U.S. health care system, yet investment and R&D innovation in those areas has been on a steady decline over the past decade or so, according to research compiled by the Biotechnology Innovation Organization (BIO).
Tocagen Inc.'s disclosure that the independent data monitoring board for its ongoing phase III trial in recurrent high-grade glioma (HGG) recommended the study continue without modification following a planned interim analysis drew mixed reactions from analysts and sent shares of the San Diego-based company (NASDAQ:TOCA) falling 38% Wednesday.
Shares of Solid Biosciences Inc. took it on the chin again, after the company reported a serious adverse event (SAE) from the second cohort of its phase I/II Ignite DMD study testing gene therapy SGT-001 in Duchenne muscular dystrophy. The latest setback comes three months after Cambridge, Mass.-based Solid disclosed disappointing data from the first patient cohort and a year after the FDA lifted a clinical hold placed on the same trial after the first patient treated suffered an SAE.
Shares of Catalyst Pharmaceuticals Inc. tumbled 40% Tuesday on word that the FDA had cleared Ruzurgi (amifampridine) for treating pediatric patients with Lambert-Eaton myasthenic syndrome (LEMS). Catalyst, of Coral Gables, Fla., currently markets its LEMS treatment, Firdapse (amifampridine phosphate), for adult patients but has come under congressional scrutiny for its hefty $375,000 annual price tag, particularly since the active drug was once available to patients for free.
VIENNA – After nearly three days of partnering meetings and sessions touting the latest therapeutic advances such as cell and gene therapy and the microbiome, along with presentations depicting the unprecedented level of capital flowing into the sector, it would be easy to think the biopharma industry had reached optimal capacity. But as the BIO Europe Spring meeting came to a close Wednesday, Evotec AG CEO and Austria native Werner Lanthaler suggested that progress and innovation "aren't happening as fast as they should."
VIENNA – Data flowing out of the BIO Europe Spring conference have underscored the significance of U.S. in the global biopharma market. During Monday's plenary session, figures showed U.S. venture capital reached a whopping $12.4 billion in 2018, compared to $2.6 billion and $2.4 billion in Europe and Asia, respectively, while Nasdaq remains the listing of choice. During a panel Tuesday, moderator Johannes Roebers, founder and CEO of Cilatus Biopharma Consulting AG, also pointed to an innovation edge, with U.S.-headquartered firms developing nearly half – 46.5 percent – of biologic molecules currently in development globally. The lead is even greater when looking at new modalities such as gene and cell therapy, with U.S. firms developing 54 percent.
VIENNA – There's a new term gaining attention in the health care sector. Defined as the "constellation of chronic noncommunicable diseases of aging," comprising cardiovascular disease, diabetes, obesity, cancer and neurodegenerative disease, "metabesity" represents a clear opportunity in the quest to extend healthspan, at least if biopharma can succeed in getting investors, partners and regulators on board.
Hailing it as the "first new mechanism of action in decades" to treat major depressive disorder (MDD), specifically treatment-resistant depression, Johnson & Johnson's Janssen unit disclosed the FDA's approval of Spravato (esketamine), a drug that works on the N-methyl-D-aspartate (NMDA) receptor. While a win for Janssen, the agency's nod might portend even better for other NMDA-targeting drugs that can be administered without all of Spravato's restrictions.
Novavax Inc. execs tried to shift the focus to promising secondary and exploratory endpoint data showing efficacy of its Resvax respiratory syncytial virus (RSV) vaccine on the severe impacts of RSV infection, but Wall Street refused to be diverted Thursday, sending company shares (NASDAQ:NVAX) to a 52-week low on news of the primary endpoint miss in the phase III Prepare maternal immunization study.
NEW YORK – Contrary to what the flurry of recent headlines might suggest, FDA approvals of the first gene and cell therapies didn't lead to the invention of value-based pricing. The model has circulated in the U.S. health care system over the years in various forms – risk-sharing arrangements and managed entry agreements, for example – but the introduction of potentially curative treatments, combined with accelerated approvals based on limited durability data, is putting the pressure on companies to reconsider how they view product launches and reimbursement.
