Five impactful biotech companies to know in New York

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Biotech companies in New York

New York – the place where people of all backgrounds come with hopes and dreams of making a better life for themselves – is just recently becoming a life sciences powerhouse, attracting many biotech companies, after a $1 billion public investment in the industry from the City of New York. 

This initiative, called LifeSci NYC, will invest $450 million to spur new research, $600 million to construct new labs and incubator spaces, and $20 million to build a pipeline of diverse talent. In doing so, the initiative aims to create 40,000 jobs, unlock 10 million square feet of wet- and dry-lab space, and spur the launch of 1,000 new companies.

Additionally, in 2017, New York City Economic Development Corporation (NYCEDC), which aims to encourage growth in New York City’s five boroughs, launched the LifeSci NYC Internship Program which prepares NYC undergraduate and graduate students for careers in the field, and builds a direct talent pipeline for the life sciences industry. 

Needless to say, because of this, there are now several biotech companies making their mark in The Big Apple, each with their own innovative ideas.

So, with everything in place to make New York a major U.S. biotech hub, let’s take a look (in alphabetical order) at five companies in New York looking to make the most of this opportunity. 

Table of contents

    Axsome Therapeutics 

    With a focus on CNS conditions that have currently limited treatment options, Axsome Therapeutics is targeting diseases such as depression, Alzheimer’s disease agitation, migraine, narcolepsy, and fibromyalgia. 

    In Just last year, the U.S. Food and Drug Administration (FDA) approved Axsome’s drug Auvelity (dextromethorphan HBr -bupropion HCl), which became the first and only rapid-acting oral medicine approved for the treatment of major depressive disorder (MDD), with labeling of statistically significant antidepressant efficacy compared to placebo starting at one week. Furthermore, the company also has another approved product in Sunosi (solriamfetol), which it acquired from Jazz Pharmaceuticals in 2022, for the treatment of excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea. 

    Auvelity, under the investigational name AXS-05, has also received a breakthrough therapy designation from the FDA for the treatment of Alzheimer’s disease agitation. And, towards the end of last year, it cleared a phase 3 trial for the same indication, after meeting both primary and secondary endpoints, showing it was better than placebo at delaying the time of agitation relapse in patients with Alzheimer’s. 

    Furthermore, the New York-based biotech company is developing a candidate called AXS-12, which contains the active ingredient reboxetine, for the treatment of the sleep disorder narcolepsy. Reboxetine was actually approved in regions of Europe for forms of depression in the late 1990s, but was rejected in the U.S. in the early 2000s. Originally belonging to Pfizer, Axsome then in-licensed the reboxetine clinical and nonclinical data from the pharma giant in 2020. AXS-12 is now in phase 3 of development. 

    Celmatix

    As a pre-clinical stage women’s health company, Celmatix’s aim is to transform lives through better ovarian health, citing the fact that one in three women have conditions caused by poor ovarian health, many of which are still being treated with hormonal interventions introduced more than 50 years ago. To help it discover new therapies, the company has developed a proprietary multi-omic ovarian health platform. 

    The New York-based biotech company recently announced that it had identified promising early leads in its work to develop the world’s first oral follicle-stimulating hormone receptor (FSHR) – which is an integral component of the endocrine axis that regulates gonadal function and fertility – agonist drug, which could revolutionize fertility treatments. The early data suggests that Celmatix’s compounds are able to successfully address the challenge of stimulating the FSH receptor without also stimulating the thyroid hormone receptor. 

    Celmatix also has partnerships with several organizations, including Evotec, Bayer and the Bill & Melinda Gates Foundation. As part of its partnership with Evotec, the company is providing novel polycystic ovary syndrome (PCOS) targets to Bayer to support their mission to bring new treatments to women facing this debilitating disease. The five-year collaboration is expected to yield multiple clinical candidates, with Bayer contributing to discovery efforts and leading clinical development and commercialization. 

    Intra-Cellular Therapies

    Intra-Cellular Therapies is on a mission to deliver innovative treatments to improve the lives of individuals with neuropsychiatric and neurologic disorders, such as schizophrenia, bipolar depression, Parkinson’s disease, and behavioral disturbances associated with Alzheimer’s disease. The company was founded on Dr. Paul Greengard’s Nobel Prize-winning research, which uncovered how therapies affect the inner workings of cells in the body.  

    Intra-Cellular Therapies has received approval from the FDA for its medication lumateperone – sold under the brand name Caplyta – for the treatment of schizophrenia (approved in 2019), and for bipolar I and II depression (approved in 2021). Caplyta is an atypical antipsychotic, whereby the mechanism of action is unknown. However, the efficacy of the drug could be mediated through a combination of antagonist activity at central serotonin 5-HT2A receptors – which are widely distributed in the central nervous system (CNS), particularly in the brain region essential for learning and cognition – and postsynaptic antagonist activity at central dopamine D2 receptors – which play a key role in regulating the activity of dopamine neurons. 

    The New York biotech company’s pipeline also includes a novel molecular entity called ITI-1284 – intended for agitation and psychosis in patients with Alzheimer’s disease, as well as generalized anxiety disorder – PDE inhibitors – for Parkinson’s disease and cancer – and another novel compound called ITI-333 – to treat opioid use disorder, and pain and mood disorders.

    Kallyope

    New York-based biotech company Kallyope is pioneering the science and understanding of gut and gut-brain biology, harnessing the gut-brain axis to try and develop groundbreaking medicines for metabolic conditions, gastrointestinal disease, and neurological disorders. To achieve this, the company has a purpose-built therapeutic platform called Klarity, which the company claims brings together the broadest set of integrated end-to-end technologies for the discovery and translation of gut-brain biology in the biopharma landscape today.

    Kallyope currently has a diverse portfolio of novel, oral small-molecule therapies across the aforementioned disease areas. In fact, the company recently initiated a phase 2 trial evaluating two new agents – K-757 and K-833 – for the treatment of both obesity and type 2 diabetes. The trial aims to demonstrate the weight loss efficacy, safety, and tolerability of these novel agents, which are oral nutrient receptor agonists that enhance the body’s natural metabolic signals to stimulate the secretion of multiple appetite-suppressing satiety (the state of feeling full) hormones – including GLP-1 (a hormone mimicked by many new obesity drugs to suppress appetite) – and several other well-validated satiety hormones, such as peptide YY (PYY) and cholecystokinin (CCK). They are currently the only known oral nutrient receptor agonists being studied for the treatment of obesity. 

    Additionally, in May, Kallyope also received an $8.2 million grant from the Gates Foundation to identify promising bioactive compounds for improving maternal, newborn, and children’s health in the epicenters of the global food and nutrition crisis. 

    MeiraGTx

    Based in New York, biotech MeiraGTx is a clinical-stage gene therapy company developing potentially curative treatments for patients suffering from a range of serious diseases. It currently has six programs in clinical development, including four ocular indications, a salivary gland condition, and a Parkinson’s disease program. The company says that its initial focus on these disease areas is based on a significant unmet need in these areas, along with the high potential gene therapy has to provide a meaningful clinical benefit. 

    MeiraGTx’s ocular portfolio consists of programs for the treatment of x-linked retinitis pigmentosa, RPE65-associated retinal dystrophy, achromatopsia, as well as RDH12-associated retinal dystrophy and age-related macular degeneration. Meanwhile, its portfolio for neurodegenerative disease includes Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and Alzheimer’s disease, and its salivary gland therapeutic area includes the development of treatments for radiation-induced xerostomia and Sjogren’s syndrome.

    The company has several partnerships with big pharma, including a deal with Janssen, which includes the development of a gene therapy treatment for the degenerative eye disease x-linked retinitis pigmentosa. This is MeiraGTx’s lead program, and has yielded positive results in an ongoing phase 1/2 clinical trial, with data demonstrating statistically significant improvement in retinal sensitivity and vision-guided mobility. 

    New York: emerging as a leading hub for biotech companies

    Earlier this year, as part of New York’s LifeSciNYC initiative, it was announced that a new $20 million center for sustainability-focused biotech will be coming to the Brooklyn Navy Yard in New York. The 50,000-square-foot innovation space is expected to open in 2025, and will be the first-ever commercial hub for sustainable biotechnology in the U.S..

    The aim of this investment is to help position New York as the nation’s leading life sciences market, while also producing new technologies to address climate change and advance the city’s carbon neutrality goals. The center will also create more than 400 jobs, and support a new generation of scientists, innovators, and entrepreneurs through office space, research labs, and events and programming space.

    The commitment to developing the life sciences industry in New York – from the mayor and those in charge – is extremely clear, and it will inevitably lead to more and more companies entering the biotech space in the City of Dreams in the coming years.

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