The Neuroscience Revival: A Growth Area for Investors in 2023
On the heels of Biogen’s 2022 success with Aduhelm in 2021 and lecanemab in early 2023, the neuroscience space has become increasingly crowded with drug developers hoping to replicate the company’s landmark achievements.
Drug development in neurodegenerative disease poses unique challenges. Historically low investment, lack of policy focused on promoting drug approvals, poor understanding of the causes of neurodegenerative disorders and high profile drug failures have all contributed to neglect by risk-averse investors.
In 2023, however, a neuroscience revival appears to be taking place. Encouraged by the success of Biogen and others, biotechs are flocking to join the race to develop the next treatment that could be life-changing for those living with progressive, ultimately fatal neurodegenerative diseases.
According to clinicaltrials.gov, there are 583 clinical trials currently recruiting in Alzheimer’s disease, 573 recruiting in Parkinson’s disease, 42 in Huntington’s disease, 444 recruiting in Schizophrenia and 193 recruiting patients in ALS. Experts believe that combination treatment will be the ultimate answer to managing – and then curing – neurodegenerative disease, since the way each disease progresses in each individual patient can vary widely.
We took a closer look at companies developing treatments in the five major neurodegenerative diseases:
Alzheimer’s disease (AD)
More than six million Americans are living with AD and new treatments are urgently needed to improve quality of daily living for individuals and families whose lives are impacted. Longeveron (Nasdaq: LGVN) expects to deliver top line results from its Phase 2a CLEAR MIND trial for AD by the end of 2023. Lomecel-BTM is a cellular therapy in which allogeneic Medicinal Signaling Cells have a therapeutic effect through secretion of bioactive molecules and direct cell-to-cell contact at the site of inflamed and damaged tissue.
Stocks to watch in AD:
Intra-Cellular Therapies (Nasdaq: ITCI) with lumateperone,
Axsome (Nasdaq: AXSM) with AXS-05 for AD agitation,
Cassava Sciences (Nasdaq: SAVA) with Simufilam, and
INmune Bio (Nasdaq: INMB) with XPRO™ for mild AD.
Parkinson’s disease (PD)
500,000 Americans have been diagnosed with PD, but experts estimate the true total patient population to be closer to one million. Annovis Bio (NYSE: ANVS) dosed the first patient in a Phase 3 trial evaluating buntanetap as a treatment for PD in August 2022. Buntanetap is a potentially ground-breaking therapy designed to inhibit the production of multiple neurotoxic proteins associated with several neurodegenerative conditions. The company expects to provide an interim data analysis from the study in the second quarter of 2023. A previous Phase 2 study demonstrated statistically significant improvements in PD patients’ motor function and coding speed.
Stocks to watch in PD:
BioVie (Nasdaq: BIVI) with NE3107 which aims to improve symptoms by reducing inflammation,
Anavex Life Sciences (Nasdaq: AVXL) with blarcamesine for PD dementia, and
Denali Therapeutics (Nasdaq: DNLI) which is working with Biogen (Nasdaq: BIIB) to develop DNL151, aimed at patients with certain gene mutations.
Huntington’s disease (HD)
HD is a rare disorder affecting approximately 30,000 people in the United States. Symptoms commonly appear between 30-50 years of age and are characterized by progressive movement, cognitive, and psychiatric symptoms. Neurocrine Biosciences, Inc. (Nasdaq: NBIX) announced statistically significant improvement in chorea (involuntary movement of limbs or facial muscles) associated with HD in a Phase 3 study of valbenazine. Improvements were seen as early as Week 2 of treatment. The company’s Prescription Drug User Fee Act (PDUFA) target date is set for August 2023.
Stocks to watch in HD:
Sage Therapeutics (Nasdaq: SAGE) which has received EMA Fast Track designation for SAGE-718 for cognitive impairment in HD, and
UniQure (Nasdaq: QURE) with the gene-silencing AMT-130.
Schizophrenia
Approximately 21 million worldwide are living with schizophrenia, a serious mental disorder that impairs daily functioning, and can be disabling. Karuna Therapeutics (Nasdaq: KRTX) is on track to submit a New Drug Application (NDA) to the FDA in mid-2023 for KarXT, which addresses schizophrenia symptoms without the common side effects seen with current treatment options.
Stocks to watch in schizophrenia:
Acadia Pharmaceuticals (Nasdaq: ACAD) which expects to deliver Phase 3 results evaluating pimavanserin in 2023/2024, and
Reviva Pharmaceuticals (Nasdaq: RVPH) which expects topline data for pivotal Phase 3 trial evaluating brilaroxazine in mid-2023.
ALS
BrainStorm Cell Therapeutics (Nasdaq: BCLI) is developing NurOwn® (autologous MSC-NTF cellular therapy) as a treatment to slow or stabilize disease progression in ALS. The company has submitted a Biologics License Application and expects to meet with the FDA in 2023.
Stocks to watch in ALS:
NeuroSense Therapeutics (Nasdaq: NRSN) which has enrolled 80% of patients in its Phase 2b trial of PrimeC, to regulate microRNA synthesis, reduce neuroinflammation, and influence iron accumulation,
Athira Pharma’s (Nasdaq: ATHA) with ATH-1105 to to promote cell regeneration, provide neuroprotection, and
Coya Therapeutics (Nasdaq: COYA) with the early-stage COYA 302.
