Remember how Ras is a frequently mutated oncogene in solid tumors? Well, it turns out Ras plays a role in those memories, too. In the Jan. 13, 2020, online issue of the Proceedings of the National Academy of Sciences, scientists at the Scripps Research Institute in Juniper, Fla., reported on the discovery that Ras signals through Raf and then Rho kinase to control whether memory is short or long-term.
With many on Wall Street transfixed by the three injectable calcitonin gene-related peptide (CGRP) therapies cleared in the prophylactic migraine market, Satsuma Pharmaceuticals Inc.’s prospects with STS-101 may have gone overlooked, at least until lately.
Shares of Cambridge, Mass.-based Wave Life Sciences Ltd. (NASDAQ:WVE) lost 49.5% of their value, or $7.82, to close at $7.99 as investors learned of top-line data from the ongoing phase Ib/IIa Precision HD2 trial testing WVE-120102 in Huntington’s disease.
For biopharma, 2019 can be described as a terrific year – with a few asterisks. The financial markets were flourishing, with venture capital dollars, in particular, flowing to the sector, while dealmaking reached historic proportions. Meanwhile, scientific breakthroughs led the way as cell and gene therapies gained ground, the first signs of success emerged with new technologies like CRISPR and the long-awaited promise of genomics found its way to the front lines of health care.
Four new U.S. drug approvals, one accelerated for need, have handed a string of year-end victories to five drugmakers, marking an unusually active start to a week full of global holiday celebrations. Daiichi Sankyo Co. Ltd., Astrazeneca plc, Eisai Inc., Intra-Cellular Therapies Inc. and Allergan plc all secured new approvals from the agency. Daiichi's Enhertu (trastuzumab deruxtecan), the subject of a $6.9 billion deal with Astrazeneca, won accelerated approval for the third-line treatment of adults with unresectable or metastatic HER2-positive breast cancer. Eisai's Dayvigo (lemborexant) was approved to treat insomnia. Intra-Cellular's Caplyta (lumateperone) was approved to treat schizophrenia. Allergan’s Ubrelvy (ubrogepant) became the first of a relatively new class of drugs to be approved for the acute treatment of migraine.
The therapeutic value of LSD, the psychedelic muse behind countless books, music and works of visual art, has hit an altogether more prosaic milestone, albeit toward a still far-out end: A phase I study, sponsored by U.K.-based Eleusis Pharmaceuticals Ltd., found low doses safe and well-tolerated, setting the stage for new tests of the approach as a disease-modifying therapy for Alzheimer’s disease (AD).
Digital therapeutics have made great strides in recent years, with Pear Therapeutics Inc. playing a key role. Now, the company has reported the dosing of the first patient in part two of a study assessing Pear-006 to address depressive symptoms in multiple sclerosis (MS).
Machine learning and artificial intelligence (AI) are already being actively used in drug discovery to evaluate potential binding of small-molecule drugs to proteins, but there's potential for the technologies to be used on the development side as well, especially in hard-to-treat diseases such as Alzheimer's disease.
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.
Neurotrauma Sciences LLC and Emory University’s Institute for Drug Development entered a license agreement for patents and technology invented at the school, enlarging a deal the two cut about a year ago.
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