Word from Immix Biopharma Inc. of updated data due with NXC-201 brought to the forefront an ongoing push by drug developers to come up with a treatment for AL amyloidosis. Immix has the only CAR T therapy in the works for the disease, and the principal investigator in the Nexicart-1 phase Ib/IIa effort is slated to speak May 19 during the annual meeting of the American Society of Gene & Cell Therapy.
Eli Lilly and Co.’s complete response letter (CRL) from the U.S. FDA relating to accelerated approval of Alzheimer’s disease (AD) candidate donanemab set off a round of speculation regarding not only what the move might mean for the pharma giant but also for others in the embattled therapeutic space and beyond. The answer, if you believe analysts: not much.
With new therapeutics under FDA review for migraine, Alzheimer’s disease and seizures, and several rare disease gene therapies in development, stocks included in BioWorld’s Neurological Diseases Index are tracking 6.09% ahead for the year.
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.
Amid a mix of controversy and clinical progress, the Alzheimer’s Association International Conference 2021 kicked off July 26 in Denver and online, delivering both new clinical data and insights into a key form of dementia affecting as many as 35 million people worldwide and growing. While interest in Biogen Inc.'s recently approved Aduhelm (aducanumab) remained high, new data on investigational therapies from Synaptogenix Inc. and Inmune Bio Inc. also drew attention, weighing on shares of both companies. New epidemiological research also revealed the first evidence that reducing air pollution may improve cognitive function and reduce dementia risk.
Novo Nordisk A/S has acquired a clinical-stage humanized monoclonal antibody and transthyretin amyloidosis (ATTR) program in a deal that could bring Prothena Corp. plc potential development and sales milestone payments of up to $1.2 billion. Also in the agreement, Dublin-based Prothena brings in $100 million in up-front and near-term clinical milestones payments as Novo gets full worldwide rights to the intellectual property and related rights of Prothena’s ATTR amyloidosis business and pipeline.
By granting accelerated approval for Biogen Inc.’s Aduhelm (aducanumab) for Alzheimer’s disease (AD), the FDA is “essentially confirming that the beta-amyloid hypothesis has been validated,” Mizuho analyst Salim Syed said in an alert to investors. Shares of Cambridge, Mass.-based Biogen (NASDAQ:BIIB) closed $395.85, up $109.71, or 38.3%, as Wall Street hailed the first new AD therapy to reach the market since 2003.
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.
Almost three years after announcing it would quit development of its humanized immunoglobulin G1 candidate, birtamimab, in light chain (AL) amyloidosis, Prothena Corp. plc is revving the program back up with a new focus, sending shares (NASADQ:PRTA) 30.5% higher to close at $14.35 on Feb. 2.
Yumanity Therapeutics Inc. said that Merck & Co. Inc. is making a big bet on its nascent neurodegenerative disease pipeline, licensing exclusive rights to programs addressing amyotrophic lateral sclerosis (ALS) and a rare type of dementia in a deal with up to $500 million in potential milestone payments. The collaboration also includes an up-front payment, money toward a series C financing and potential royalties on net sales of the licensed programs, though the value of those elements wasn't disclosed.