Precision medicine is an approach to tailoring treatments based on people’s genetic makeup, lifestyles and environment. These targeted interventions can tackle diseases like cancer, neurological as well as metabolic diseases. The global market value of the precision medicine industry, encompassing many companies, crossed 70 billion last year, and is expected to hit $175 billion in the next seven years.
Here are seven biopharma companies that could potentially contribute to this rise in the precision medicine market value in the coming years, as they are focused on getting their clinical candidates to the market soon.
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More than 50 million people currently live with dementia. To treat dementia and diseases that affect cognitive function, various biotechs are attempting to create safe and effective drugs. Clinical-stage company AC Immune, which is based in Switzerland, has a compelling precision medicine pipeline that aims to tackle neurodegenerative diseases. This encompasses measures to diagnose, treat and prevent cognitive decline due to neurodegeneration.
The company’s Tau-PET tracer PI-2620, is a diagnostic tool that is currently in phase 3 studies. It has been designed to bind to the tau protein, which is linked to Alzheimer’s disease progression. The multi-million dollar company has numerous candidates undergoing phase 2 studies. This includes the Abeta vaccine, which can generate antibodies that target misfolded amyloid beta to prevent accumulation and curb the formation of amyloid plaques, a hallmark pathology of Alzheimer’s. A trial is also being conducted to test the candidate in people with Down Syndrome, who are at the risk of developing Alzheimer’s disease. The results of the trial are predicted to be released next year.
Moreover, AC Immune’s monoclonal antibody crenezumab, which also targets misfolded Abeta, is being examined in a prevention trial in Colombia.
The biotech is also focused on treating Parkinson’s disease, for which it has developed an alpha synuclein vaccine that is being investigated in the clinic. Alpha synuclein is a protein that is associated with Parkinson’s disease.
And that’s not all. The company has a few preclinical candidates that aim to address neurodegenerative diseases as well. These comprise diagnostic biomarkers, biologic drug candidates and small molecules.
California-based precision medicine company Alto Neuroscience secured $45 million in a series C funding round last month, which it will use to support the clinical development of its central nervous system (CNS) candidates.
This includes its three drug candidates for depression and post-traumatic stress disorder (PTSD), which are all being evaluated in phase 2 trials. Each of these drugs is designed to target brain processes that improve three different functions – cognition, emotion and regulation, sleep and activity.
Now, its candidate ALTO-300 has reaped promising results, as it “led to a significantly greater improvement in depression symptoms in a biomarker-characterized patient population,” according to a press release released last week. The study lasted for eight weeks, where potential predictive biomarkers were used to test the efficacy and safety of the drug. It was also found that more biomarker-characterized patients achieved a clinical response rather than patients without the biomarker.
For this, the biotech has created a biomarker platform that is powered by artificial intelligence. Its Precision Psychiatry Platform aims to drive drug development with the help of biomarkers, a potentially efficient method to assess brain function.
Based in the biotech hub of California, Biomea Fusion is immersed in the discovery and development of small molecules to treat people with genetically defined cancers and metabolic diseases. The company’s covalent small molecules are designed to form a bond with a target protein.
Its drug candidate BMF-219 is currently being investigated for type 2 diabetes. The candidate also received U.S. Food and Drug Administration (FDA) clearance for its investigational new drug (IND) application for type 1 diabetes recently, which allowed it to proceed onto phase 2 trials as well. The candidate is a menin inhibitor that is capable of regenerating insulin-producing beta cells. The candidate will be evaluated for safety, efficacy, and durability in patients with type 1 diabetes.
But that’s not all that BMF-219 is committed to treating. The candidate is being tested in a number of cancer indications like leukemia, lymphoma, myeloma, and non-small cell lung cancer, among others, all of which are in the phase 1 stage. It works by blocking the interaction of the protein menin, and restricts activity from cancer-driving genes.
BMF-219 showed signs of clinical activity, and it was able to sustain complete responses with minimal residual disease negativity (MRD-neg) in patients with acute myeloid leukemia (AML), according to findings that it presented at 2023 ASH.
Part of the biotech scene in Singapore, the company Engine Biosciences is focused on drug development programs based on precision medicine. It raised $27 million in October, for advancing its oncology programs that are in their discovery phase.
These include candidates to treat a range of cancers including ovarian, liver, colorectal, prostate and breast cancer, among others. One of its candidates targets a protein kinase called PKMYT1, which has been found to contribute to tumor progression.
The company also has more than 15 biomarker programs that are in the clinical stage, at present. Along with precision oncology, the company has also identified therapeutic targets in the field of neurology.
The company’s NetMAPPR platform mines through the database that combines machine learning and biology to identify potential synthetic lethal connections that are then progressed through the pipeline. Engine Biosciences has also curated a library of gene connections with the help of its platform.
A pipeline centered around rare disease therapeutics, ReCode Therapeutics has developed mRNA-based treatments to tackle primary ciliary dyskinesia and cystic fibrosis.
Primary ciliary dyskinesia is a condition caused by genetic mutations, where the hairline cilia present in the lungs, nose and ears are affected, leading to mucus buildup and infection because of the impaired cilia’s inability to remove pollutants. The company’s candidate, which targets the DNAI1 gene, aims to treat the disease, and is presently in phase 1/2 trials. It also has two candidates in their discovery phases that target PCD gene 2 and PCD gene 3, to treat primary ciliary dyskinesia.
Its cystic fibrosis treatment is an inhaled disease-modifying mRNA-based investigational therapy that addresses a fraction of the patient population who have nonsense mutations and do not respond to drugs like CFTR modulators, which are typically administered to treat the disease.
Recently, the precision medicine company closed a $260 million series B round, with which it will continue to develop its candidates, as well as expand its Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform. With the SORT LNP platform, the company is developing its gene correction therapeutics pipeline. The SORT LNPs help package the drugs, and aid in drug delivery.
U.S.-based Scorpion Therapeutics is attempting to find ways to selectively target and strike against cancer. Since its inception in 2020, the company has been committed to a precision medicine approach to treat cancer.
By identifying the specific cancer drivers in patients, it aims to pick out the treatment strategy that works best for each individual. Through computational biology techniques and human tumor genomics databases, it creates small molecules that are target-specific. Scorpion Therapeutics also uses tools like CRISPR to identify drug targets that have not been successfully targeted before.
The company has two candidates that are in phase 1 studies. STX-478 is an oral PI3Kα inhibitor, which is being developed to treat breast, gynecological, and head and neck cancers with the PI3Kα mutation. The candidate has expressed anti-tumor activity in preclinical studies.
The other candidate in phase 1 is STX-721. Its purpose is to selectively target exon 20 insertion mutations in EGFR, an oncogene in non-small cell lung cancer. Previously, in xenograft models, the candidate has shown promise in potentially treating cancer. But how the phase 1 trial pans out, we are yet to find out.
American company SpringWorks Therapeutics specializes in targeted oncology. Its gamma secretase inhibitor called nirogacestat has received orphan drug designation, fast track and breakthrough therapy designations from the FDA for treating adult desmoid tumors.
Desmoid tumors are rare, noncancerous growths that occur in the connective tissue. Nirogacestat works by inhibiting gamma secretase, an enzyme that aids in activating a signaling protein called Notch. As it is thought that these proteins drive desmoid tumor growth since they are present in high amounts in the tumors, suppressing the production of Notch protein can impede tumor growth. The candidate is also being investigated as a monotherapy in phase 2 trials for pediatric desmoid tumors as well as ovarian granulosa cell tumors – a kind of rare ovarian cancer for which there are no FDA-approved therapies, at present. It is also being assessed in combination with a number of other therapies, for the treatment of relapsed or refractory multiple myeloma.
As part of the biotech’s precision oncology approach to study investigational drugs to address biomarker-defined metastatic cancers, the company is also developing an MEK inhibitor, mirdametinib, for treating various cancers including NF1-associated plexiform neurofibromas, for which it is in a phase 2b trial. It has also partnered with global pharmaceutical company BeiGene, to treat solid tumors with mirdametinib in combination with the RAF inhibitor lifirafenib. Furthermore, the biotech has other inhibitors that it is developing in the mix. The company closed a public offering worth more than $300 million last week.
Precision medicine and big pharmaceutical companies
Among the big leagues in biopharma, Novartis is one of the multinational companies that is invested in precision medicine. The company has created next-generation sequencing tests along with molecular diagnostics company Genoptix. The diagnostic test helps identify specific mutations present in patients, which in turn helps select the drug that would work best based on the patient’s genetic makeup.
Pfizer is another multinational that is also leveraging precision medicine in drug discovery. This includes research in biomarkers and imaging tools. Similarly, AstraZeneca is focused on treating asthma and metabolic disorders with the help of precision medicine. It is doing so by identifying drug targets and biomarkers, and developing diagnostic tests that can help guide patients through treatment options.
Other contenders include Amgen, Roche, Bristol Myers Squibb and even BioNTech, which recently partnered with the U.K. government to provide personalized cancer therapies for up to 10,000 patients by the end of 2030. This will see BioNTech’s clinical programs for personalized mRNA cancer immunotherapies advance further. The company intends to design and set clinical trials in motion for its precision medicine candidates soon.