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Small molecule drugs are defined as any organic compound with a low molecular weight that is discovered, designed, and developed to prompt a specific biological process in the body. They have been a mainstay of the pharma industry for almost a century, and, still, numerous companies are working to discover and develop novel small molecules that can treat a number of different indications. In this article, we take a look at nine promising small molecule drug discovery companies that have raised funding in the last year.
Table of contents
Ascentage Pharma
- Disease area: Oncology
- Lead candidate: Olverembatinib
- Recent news: Closed a $126.4 million U.S. initial public offering
Ascentage Pharma is discovering and developing small molecule drugs for the treatment of cancer that block or degrade protein targets implicated in cancer. It has active clinical programs targeting all three known classes of key apoptosis regulators that restore programmed cell death, a natural process where cells self-destruct in a controlled manner to maintain tissue homeostasis and eliminate damaged or potentially harmful cells. If this process is dysfunctional, however, it can lead to various diseases, especially cancer.
The company’s most advanced candidate is olverembatinib, which is cleared in China for certain patients with chronic myeloid leukemia, and is in phase 3 trials in multiple countries for the same indication, as well as for two other forms of cancer. Meanwhile, Ascentage also has another drug in development called lisaftoclax, which is in late-stage trials for hematological cancers, including chronic lymphocytic leukemia and acute myeloid leukemia. The company believes that it has the potential to become a “backbone molecule” for combination regimens. The drug is currently under regulatory review in China.
In January 2025, Ascentage – which is already publicly traded in Hong Kong – closed a $126.4 million U.S. initial public offering (IPO). The company will use the proceeds to advance its current crop of candidates.
858 Therapeutics
- Disease areas: Oncology and immunology
- Lead candidate: ETX-19477
- Recent news: Entered into a collaboration with OpenBench to discover small molecule inhibitors
858 Therapeutics is developing a portfolio of small molecule drugs that act on novel therapeutic targets in oncology and immunology. The company says that it leverages its expertise in structure-based drug discovery and translational research to develop differentiated drug candidates. Its lead programs focus on important nodes in cancer biology, including DNA damage repair, innate immunity, and RNA epigenetics.
The company’s lead candidate is a PARG inhibitor called ETX-19477, which is in a phase 1 trial for solid tumors. PARG – which stands for poly(ADP-ribose) glycohydrolase – is an enzyme that plays an essential role in DNA repair. While the better-known poly(ADP-ribose) polymerases (PARPs) detect DNA damage and repair it by adding poly(ADP-ribose) chains to various proteins involved in the repair process, PARG is responsible for removing these chains once the job is done. By inhibiting PARG, the normal cycle of poly(ADP-ribose) metabolism is disrupted, in turn hampering the cells’ ability to repair DNA damage efficiently. Because cancer cells are particularly vulnerable to this disruption, PARG inhibitors can selectively target cancer cells while sparing normal, healthy cells that have more robust repair mechanisms.
In September 2024, the small molecule drug discovery company announced that it had raised $50 million in series B financing. Shortly after, in November, it also said that it had entered into a collaboration with OpenBench to discover small molecule inhibitors using OpenBench’s structure-based machine learning platform.
Iktos
- Disease areas: Inflammatory and autoimmune diseases, oncology, and obesity
- Lead program: MTHFD2 inhibitor candidate
- Recent news: Secured a €2.5 million ($2.7 million) grant from the EIC Accelerator
As an artificial intelligence (AI) drug discovery company, Iktos is making use of AI and robotics synthesis automation for drug discovery and design, so that it can rapidly identify suitable small molecules for drug development. The company has multiple different technologies to help with the discovery process, which have helped to form the company’s current preclinical pipeline focused on inflammatory and autoimmune diseases, oncology, and obesity. Its lead program revolves around MTHFD2 inhibitors, which have the potential to treat various inflammatory conditions with an improved safety profile compared to JAK1 inhibitors. The company said that preclinical candidate nomination for this program is expected by some time this year.
In March 2023, Iktos announced that it had raised €15.5 million ($16.4 million) in a series A round, enabling it to further develop its AI and drug discovery capabilities and expand its existing SaaS software offering, as well as launch Iktos Robotics – an end-to-end drug discovery platform that combines AI and automation of chemical synthesis to significantly accelerate drug discovery timelines.
Meanwhile, most recently, in February this year, Iktos received a €2.5 million ($2.7 million) grant from the European Innovation Council (EIC) Accelerator to advance its AI and robotics technology. Additionally, in January, the company entered into a small molecule AI drug discovery collaboration with Cube Biotech that will leverage Iktos’s AI platform and Cube Biotech’s advanced protein technologies to develop novel agonists of the Amylin Receptor.
Inductive Bio
- Technology: AI platform that dramatically accelerates compound optimization
- Recent partnership: Drug optimization collaboration with Nested Therapeutics
- Recent news: Raised $25 million in series A funding
Inductive Bio has developed a collaborative AI platform that dramatically accelerates compound optimization, as one of the biggest bottlenecks in the preclinical drug discovery process is the enormous amount of time and money needed to balance a drug’s potency with the critical properties of absorption, distribution, metabolism, excretion, and toxicity (ADMET).
The company’s approach centers around a pre-competitive data consortium, where multiple companies contribute anonymized data in a secure IP-protected environment, in turn creating a foundational dataset that allows AI models to learn from thousands of real-world drug discovery programs. The resulting insights power Inductive’s platform, called Compass, which predicts a drug’s ADMET properties before a molecule is even synthesized, meaning that researchers and chemists can focus only on molecules with the highest likelihood of success, saving them both time and money.
Since Inductive’s launch a year ago, its platform has rapidly scaled to support dozens of active small molecule programs across multiple therapeutic areas, and one million molecule designs have already been explored in the platform. In July last year, Inductive announced that details had been published of a successful drug optimization collaboration with Nested Therapeutics, in which Compass helped efficiently resolve permeability and metabolic stability issues, contributing to the nomination of a drug development candidate with excellent cell potency and cross-species pharmacokinetics.
Meanwhile, just this month, the small molecule drug discovery company announced that it had raised $25 million in series A funding.
Insilico Medicine
- Disease area: Fibrosis
- Lead candidate: INS018_055
- Recent news: Raised $110 million in series E funding
Focused on using AI for every step of pharmaceutical research and development, Insilico Medicine has a fully integrated, commercially available drug discovery suite called Pharma.AI, which is designed to improve the quality and productivity of pharmaceutical research. One of the platforms involved in this, called Chemistry42, is a small molecule drug discovery and design platform that combines both the flexibility of generative AI and the accuracy of physics-based methods to create optimal molecules.
In 2023, Insilico’s small molecule inhibitor for the treatment of idiopathic pulmonary fibrosis became the first entirely AI-discovered and AI-designed drug to enter a phase 2 clinical trial. In June last year, the company announced that it had completed patient enrollment in a phase 2a study of the candidate, known as INS018_055, in China. The company also has two more drugs in clinical stages that have been partially generated by AI – one for COVID-19, and the other for solid tumors.
In March 2025, Insilico secured $110 million in series E funding. The company said that, on one side, the proceeds will focus on refining AI models and algorithms, alongside updates and expansions to its state-of-the-art automatic lab to further automate and streamline research and development (R&D) processes and, on the other side, will focus on advancing the clinical validation of INS018_055 and accelerating the exploration of other independently developed and co-developed drug pipelines.
Light Horse Therapeutics
- Disease area: Oncology
- Recent funding: Raised $62 million in series A funding
- Recent news: Entered into a strategic collaboration with Novartis
Having debuted in January 2025 with $62 million in series A financing, Light Horse Therapeutics is applying precision gene editing to small molecule drug discovery. To do this, it has a precision genetic editing-based platform that identifies cryptic, chemically accessible functional domains within targets that play critical roles in disease biology. The high-throughput discovery platform systematically interrogates complex signaling pathways to uncover unique sites of vulnerability. The company’s method ultimately provides a faster discovery of clinical candidates.
Light Horse describes itself as having a “function-first” approach to small molecule drug discovery, compared to traditional “screening-first” methodologies. This is because the company’s technology turns conventional drug discovery on its head by first identifying novel functional domains and then screening for chemistry.
In the press release about the company’s debut, Light Horse’s chief executive officer (CEO) said the small molecule drug discovery company’s initial focus “addresses high-value, historically challenging oncology targets with the opportunity to apply the technology to other therapeutic areas in the future.”
At the same time as announcing its debut and series A financing, Light Horse also released another announcement to say that it had entered into a strategic collaboration with Novartis that is worth up to $1 billion, to identify and develop therapies using Light Horse’s platform.
Maze Therapeutics
- Disease areas: Renal and cardiometabolic diseases
- Lead candidate: MZE829
- Recent news: Dosed the first patient in a phase 2 trial of MZE829
Focused on harnessing the power of human genetics to develop small molecule precision medicines, Maze Therapeutics’ approach is notable for its emphasis on understanding genetic drivers of common diseases, an area that has traditionally been challenging in the field of drug discovery. To achieve this, Maze has a platform called Compass, which identifies gene variants that are associated with disease and unravels what those variants do in order to identify new targets and drugs.
The company’s main focus areas are renal and cardiometabolic indications. Its lead candidate, MZE829, is an oral APOL1 inhibitor being tested for APOL1-mediated kidney disease, a type of kidney disease primarily affecting individuals of African descent that is caused by inheriting two risk variants of the APOL1 gene, which leads to an increased risk of chronic kidney disease and kidney failure. The first patient was dosed in a phase 2 trial of MZE829 in February this year after positive phase 1 results showed that the candidate was well-tolerated at single doses up to 480mg and multiple doses up to 350mg, with all treatment-related adverse events reported as mild, and no severe or serious adverse events reported.
Maze has raised a significant amount of money since it launched with $191 million in 2019; in 2022, it raised another $190 million, before going on to bag $115 million in a series D round in December 2024 and $140 million in an IPO in January 2025.
Tasca Therapeutics
- Disease area: Oncology
- Lead candidate: CP-383
- Recent news: Launched with $52 million in series A funding
Named after the Italian word for “pocket,” Tasca Therapeutics is harnessing the power of mass spectrometry-based proteomics to identify cancer-causing proteins that undergo a specific post-translational modification called auto-palmitoylation, which will ultimately allow the company to utilize previously unknown binding pockets to develop novel small molecule inhibitors for difficult-to-treat cancers.
Tasca’s most advanced candidate, CP-383, is being developed to treat patients with tumors harboring a specific genetic profile. It has demonstrated robust anti-cancer activity in both in vitro and in vivo studies across a broad range of human cancers, and will initially be evaluated in small cell lung cancer, colorectal cancer, head and neck cancer, and brain cancer patients.
In December 2024, Tasca launched with $52 million in series A funding. The company said that the proceeds from this would be used to advance the company’s drug discovery platform, progress CP-383 into phase 1/2 clinical proof-of-concept studies, and expand its drug candidate pipeline.
Vivace Therapeutics
- Disease area: Oncology
- Lead candidate: VT3989
- Recent news: Raised $35 million in series D funding
Small molecule drug discovery company Vivace Therapeutics is developing cancer therapies targeting the Hippo-YAP pathway, which has been shown to be involved in the regulation of cell proliferation, programmed cell death, and cell migration. In healthy cells, the pathway controls tissue regeneration and the size and shape of organs, but mutations in the Hippo-YAP pathway can drive several forms of cancer, which depend on activated YAP to survive and grow.
Despite the fact that researchers have long known about the role of YAP in some cancers, they could not find a way to turn the protein off, as there were no obvious drug targets like the usual kinases or receptors that are usually targeted in oncology drug discovery. Therefore, instead of aiming at YAP directly, Vivace is targeting proteins in the transcriptional enhanced associate domain (TEAD) family. This is because, for YAP to work, it needs to form a complex with TEAD, and for TEAD to work, it needs to have a fatty acid called palmitic acid covalently attached to it. Vivace’s compounds block the attachment of the fatty acid onto TEAD, making it unable to combine with YAP, in turn affecting the survival mechanism of certain cancers.
The company’s lead small molecule candidate, VT3989, has been evaluated in more than 150 patients to date in an ongoing phase 1 study. Clinical findings for the candidate have been particularly notable in patients with mesothelioma who have failed chemotherapy and immuno-oncology combination regimens. Vivace said it will present the results at a major medical conference in the second half of 2025, but based on these data, it is already working to advance the candidate toward a phase 3 clinical trial in patients with mesothelioma and intends to discuss its plans with the U.S. Food and Drug Administration (FDA) later this year.
Moreover, in March, the company raised $35 million in series D funding to support the continued clinical development of VT3989.
Small molecule drug discovery market set for significant growth
Considering the number of FDA approvals small molecule drugs have recently received, there is reason to believe that we might be in the “golden age” of small molecule drug discovery, largely thanks to the advancement of artificial intelligence (AI).
In fact, the global small molecule drug discovery market size, which was valued at $52.32 billion in 2023, is expected to reach an incredible $107.04 billion in 2032, growing at a compound annual growth rate (CAGR) of 8.3% over the forecast period. This means that small molecule drugs are likely to continue to be the cornerstone of the biopharma industry for many years to come.
Oncology R&D trends and breakthrough innovations
