A quartet of companies on May 2 announced three complete response letters (CRLs) that left them scrambling to get back on the path to approval. Hutchmed Ltd.'s surufatinib met with word from the U.S. FDA that two positive phase III studies in China and a bridging study in the U.S. would not support approval of the drug for pancreatic and extra-pancreatic neuroendocrine tumors. A multi-regional clinical trial in the U.S. is needed, the regulator said, brushing aside China-only studies when seeking a U.S. approval.
After August 2021 news from the U.S. FDA of deficiencies in the NDA that Axsome Therapeutics Inc. submitted for AXS-05 in major depressive disorder (MDD), word was especially welcome April 19 that the firm has come to an agreement with the agency with regard to post-marketing requirements. Meanwhile, another player in MDD, Relmada Therapeutics Inc., is due to report phase III data with REL-1017 (esmethadone) around the middle of this year.
Shares of Axsome Therapeutics Inc. (NASDAQ:AXSM) hit a 52-week low on Aug. 9, falling 46.5% to close at $24.37, after the FDA said deficiencies in an NDA in major depressive disorder for its drug, AXS-05 (dextromethorphan + bupropion), would "preclude labeling discussions" for now.
Although 14 of the 17 stocks that make up BioWorld’s Neurological Disease Index (NDI) dropped in value over the last month, the index is still ahead by about 9% this year, with a huge surge by Prothena Corp. plc leading the way.
The significant risks and high costs associated with neurological R&D has tended to keep companies and investors on the sidelines over the past few years. However, thanks to research progress and the development of new technologies, business development and investing in the space is heating up once again.
In a relatively light news flow for the group last month, the gainers and decliners in the BioWorld Neurological Diseases index canceled themselves out during August, with the result that the price-weighted index closed relatively flat and, despite briefly moving into positive territory in July, its value is down 4% for the year.
With regard to agitation in Alzheimer’s disease (AD), doctors “are in a very difficult position right now,” said Axsome Therapeutics Inc. CEO Herriot Tabuteau. That’s because the products currently used off-label are antipsychotics – all of which carry an FDA black box warning “specifically against their use in elderly patients with dementia, including AD,” since they double the risk of stroke and of mortality.
With positive top-line data reported for Axsome Therapeutics Inc.’s oral migraine candidate, AXS-07, in its INTERCEPT trial, the company and the candidate have notched two successful phase III trials in little more than three months.
Investors warmed to biopharma company equities, particularly in the final quarter of the year, with the BioWorld Biopharmaceutical Index increasing 23% in value during this period, helping the group climb to a respectable 14% for the year after being underwater from April through to September.
Fueled by new positive data, Axsome Therapeutics Inc. plans to seek approval during the second half of 2020 for its dextromethorphan/bupropion modulated delivery tablet, AXS-05, an oral NMDA receptor antagonist to treat major depressive disorder.
