Venturing into the unknown: how pharma is embracing neuroscience R&D after decades of failure

After the blockbuster neuroscience breakthroughs of the late 1990s (Eli Lilly’s Prozac and Pfizer’s Zoloft to name two), the last two decades have been characterized by?high-profile clinical trial failures and the shuttering of pharma neuroscience pipelines. Companies like?AstraZeneca,?Pfizer,?Bristol-Myers Squibb,?GSK, and?Amgen?all cut neuroscience R&D programs or left the field completely.

However, some?big players have been returning to the space with large investments, making big bets on entire portfolios and billion-dollar deals. In 2020, in a deal worth up to $1.2 billion,?Eli Lilly?acquired Disarm Therapeutics and its portfolio of therapies for peripheral neuropathy and other neurodegenerative conditions, like amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS).?GSK?returned to neuroscience in 2021 through a collaboration with Alector, worth $2.2 billion, to develop drugs for Alzheimer’s and Parkinson’s.

The resurgence of funding in neuroscience has been brought about by some?notable successes in the field, including approvals for Biogen’s?Spinraza?in 2016, followed by Novartis’ gene therapy?Zolgensma?in 2019, the first available treatments for Spinal Muscular Atrophy (SMA). Though?short-lived, Biogen had another win in 2021 when their controversial drug?Aduhelm?became the first disease-modifying treatment available for Alzheimer’s.

While risks of costly clinical trial failures remain,?advances in gene therapy?mean that neurodegenerative diseases linked to a single gene, like SMA or Huntington’s, may become easier to treat.?Advances in drug transporters, like Denali Therapeutics’?Transport Vehicle platform, may also improve the ability to deliver therapeutics to the brain. The FDA, recognizing?the high unmet need in neuroscience, has also taken steps to promote innovation and increase treatment options. In 2018, it released new?guidance for approaching drug development?within five neurological diseases. It has also become more receptive to the?use of surrogate endpoints in neuroscience clinical trials, as evidenced by Aduhelm’s?accelerated approval.

The breadth of the neuroscience field could equate to?millions of patients, with that number increasing as populations age and later-onset conditions become more common. It is therefore no surprise that in the last few years?venture capital funding has been pouring into neuroscience, opening the door for even more investments and collaborations from big pharma players. Neumora, a biotech using data science to develop targeted therapies for neurological conditions, closed one of the largest ever biopharmaceutical?Series A funding rounds?in 2021, earning $500 million.

But all of this renewed enthusiasm begs the question: After the failures that plagued the last twenty years of neuroscience drug development, is pharma’s newfound risk tolerance in the field now here to stay??And is this the decade that the risk finally pays off?

Author: Brynna Hoggard, Eradigm Neurology and Immunology Expert Circle