9 small molecule drug discovery firms to look out for in 2025

Small molecule medication are outlined as any natural compound with a low molecular weight that’s found, designed, and developed to immediate a particular organic course of within the physique. They’ve been a mainstay of the pharma trade for nearly a century, and, nonetheless, quite a few firms are working to find and develop novel small molecules that may deal with numerous completely different indications. On this article, we check out 9 promising small molecule drug discovery firms which have raised funding within the final yr.

Ascentage Pharma

• Illness space: Oncology
• Lead candidate: Olverembatinib
• Latest information: Closed a $126.4 million U.S. preliminary public providing 

Ascentage Pharma is discovering and creating small molecule medication for the remedy of most cancers that block or degrade protein targets implicated in most cancers. It has energetic medical applications focusing on all three identified lessons of key apoptosis regulators that restore programmed cell demise, a pure course of the place cells self-destruct in a managed method to take care of tissue homeostasis and get rid of broken or probably dangerous cells. If this course of is dysfunctional, nonetheless, it might result in numerous illnesses, particularly most cancers. 

The corporate’s most superior candidate is olverembatinib, which is cleared in China for sure sufferers with persistent myeloid leukemia, and is in section 3 trials in a number of nations for a similar indication, in addition to for 2 different types of most cancers. In the meantime, Ascentage additionally has one other drug in growth known as lisaftoclax, which is in late-stage trials for hematological cancers, together with persistent lymphocytic leukemia and acute myeloid leukemia. The corporate believes that it has the potential to change into a “spine molecule” for mixture regimens. The drug is at present beneath regulatory evaluation in China.

In January 2025, Ascentage – which is already publicly traded in Hong Kong – closed a $126.4 million U.S. preliminary public providing (IPO). The corporate will use the proceeds to advance its present crop of candidates. 

858 Therapeutics

• Illness areas: Oncology and immunology
• Lead candidate: ETX-19477
• Latest information: Entered right into a collaboration with OpenBench to find small molecule inhibitors

858 Therapeutics is creating a portfolio of small molecule medication that act on novel therapeutic targets in oncology and immunology. The corporate says that it leverages its experience in structure-based drug discovery and translational analysis to develop differentiated drug candidates. Its lead applications give attention to essential nodes in most cancers biology, together with DNA injury restore, innate immunity, and RNA epigenetics.

The corporate’s lead candidate is a PARG inhibitor known as ETX-19477, which is in a section 1 trial for strong tumors. PARG – which stands for poly(ADP-ribose) glycohydrolase – is an enzyme that performs a necessary position in DNA restore. Whereas the better-known poly(ADP-ribose) polymerases (PARPs) detect DNA injury and restore it by including poly(ADP-ribose) chains to varied proteins concerned within the restore course of, PARG is liable for eradicating these chains as soon as the job is completed. By inhibiting PARG, the traditional cycle of poly(ADP-ribose) metabolism is disrupted, in flip hampering the cells’ means to restore DNA injury effectively. As a result of most cancers cells are notably weak to this disruption, PARG inhibitors can selectively goal most cancers cells whereas sparing regular, wholesome cells which have extra sturdy restore mechanisms.

In September 2024, the small molecule drug discovery firm introduced that it had raised $50 million in sequence B financing. Shortly after, in November, it additionally stated that it had entered right into a collaboration with OpenBench to find small molecule inhibitors utilizing OpenBench’s structure-based machine studying platform. 

Iktos 

• Illness areas: Inflammatory and autoimmune illnesses, oncology, and weight problems
• Lead program: MTHFD2 inhibitor candidate
• Latest information: Secured a €2.5 million ($2.7 million) grant from the EIC Accelerator

As an synthetic intelligence (AI) drug discovery firm, Iktos is making use of AI and robotics synthesis automation for drug discovery and design, in order that it might quickly establish appropriate small molecules for drug growth. The corporate has a number of completely different applied sciences to assist with the invention course of, which have helped to kind the corporate’s present preclinical pipeline centered on inflammatory and autoimmune illnesses, oncology, and weight problems. Its lead program revolves round MTHFD2 inhibitors, which have the potential to deal with numerous inflammatory situations with an improved security profile in comparison with JAK1 inhibitors. The corporate stated that preclinical candidate nomination for this program is predicted by a while this yr. 

In March 2023, Iktos introduced that it had raised €15.5 million ($16.4 million) in a sequence A spherical, enabling it to additional develop its AI and drug discovery capabilities and develop its current SaaS software program providing, in addition to launch Iktos Robotics – an end-to-end drug discovery platform that mixes AI and automation of chemical synthesis to considerably speed up drug discovery timelines.

In the meantime, most lately, in February this yr, Iktos acquired a €2.5 million ($2.7 million) grant from the European Innovation Council (EIC) Accelerator to advance its AI and robotics expertise. Moreover, in January, the corporate entered into a small molecule AI drug discovery collaboration with Dice Biotech that may leverage Iktos’s AI platform and Dice Biotech’s superior protein applied sciences to develop novel agonists of the Amylin Receptor.

Inductive Bio

• Know-how: AI platform that dramatically accelerates compound optimization
• Latest partnership: Drug optimization collaboration with Nested Therapeutics
• Latest information: Raised $25 million in sequence A funding

Inductive Bio has developed a collaborative AI platform that dramatically accelerates compound optimization, as one of many greatest bottlenecks within the preclinical drug discovery course of is the large quantity of money and time wanted to steadiness a drug’s efficiency with the important properties of absorption, distribution, metabolism, excretion, and toxicity (ADMET). 

The corporate’s strategy facilities round a pre-competitive knowledge consortium, the place a number of firms contribute anonymized knowledge in a safe IP-protected surroundings, in flip making a foundational dataset that enables AI fashions to be taught from hundreds of real-world drug discovery applications. The ensuing insights energy Inductive’s platform, known as Compass, which predicts a drug’s ADMET properties earlier than a molecule is even synthesized, that means that researchers and chemists can focus solely on molecules with the very best chance of success, saving them each money and time. 

Since Inductive’s launch a yr in the past, its platform has quickly scaled to help dozens of energetic small molecule applications throughout a number of therapeutic areas, and a million molecule designs have already been explored within the platform. In July final yr, Inductive introduced that particulars had been revealed of a profitable drug optimization collaboration with Nested Therapeutics, by which Compass helped effectively resolve permeability and metabolic stability points, contributing to the nomination of a drug growth candidate with wonderful cell efficiency and cross-species pharmacokinetics. 

In the meantime, simply this month, the small molecule drug discovery firm introduced that it had raised $25 million in sequence A funding. 

Insilico Drugs

• Illness space: Fibrosis
• Lead candidate: INS018_055
• Latest information: Raised $110 million in sequence E funding 

Centered on utilizing AI for each step of pharmaceutical analysis and growth, Insilico Drugs has a totally built-in, commercially out there drug discovery suite known as Pharma.AI, which is designed to enhance the standard and productiveness of pharmaceutical analysis. One of many platforms concerned on this, known as Chemistry42, is a small molecule drug discovery and design platform that mixes each the flexibleness of generative AI and the accuracy of physics-based strategies to create optimum molecules. 

In 2023, Insilico’s small molecule inhibitor for the remedy of idiopathic pulmonary fibrosis turned the primary completely AI-discovered and AI-designed drug to enter a section 2 medical trial. In June final yr, the corporate introduced that it had accomplished affected person enrollment in a section 2a examine of the candidate, often known as INS018_055, in China. The corporate additionally has two extra medication in medical levels which have been partially generated by AI – one for COVID-19, and the opposite for strong tumors.

In March 2025, Insilico secured $110 million in sequence E funding. The corporate stated that, on one facet, the proceeds will give attention to refining AI fashions and algorithms, alongside updates and expansions to its state-of-the-art computerized lab to additional automate and streamline analysis and growth (R&D) processes and, on the opposite facet, will give attention to advancing the medical validation of INS018_055 and accelerating the exploration of different independently developed and co-developed drug pipelines. 

Mild Horse Therapeutics

• Illness space: Oncology 
• Latest funding: Raised $62 million in sequence A funding 
• Latest information: Entered right into a strategic collaboration with Novartis 

Having debuted in January 2025 with $62 million in sequence A financing, Mild Horse Therapeutics is making use of precision gene modifying to small molecule drug discovery. To do that, it has a precision genetic editing-based platform that identifies cryptic, chemically accessible practical domains inside targets that play important roles in illness biology. The high-throughput discovery platform systematically interrogates advanced signaling pathways to uncover distinctive websites of vulnerability. The corporate’s methodology in the end supplies a sooner discovery of medical candidates.  

Mild Horse describes itself as having a “function-first” strategy to small molecule drug discovery, in comparison with conventional “screening-first” methodologies. It is because the corporate’s expertise turns typical drug discovery on its head by first figuring out novel practical domains after which screening for chemistry.

Within the press launch concerning the firm’s debut, Mild Horse’s chief government officer (CEO) stated the small molecule drug discovery firm’s preliminary focus “addresses high-value, traditionally difficult oncology targets with the chance to use the expertise to different therapeutic areas sooner or later.”

Concurrently saying its debut and sequence A financing, Mild Horse additionally launched one other announcement to say that it had entered right into a strategic collaboration with Novartis that’s price as much as $1 billion, to establish and develop therapies utilizing Mild Horse’s platform. 

Maze Therapeutics

• Illness areas: Renal and cardiometabolic illnesses
• Lead candidate: MZE829
• Latest information: Dosed the primary affected person in a section 2 trial of MZE829

Centered on harnessing the facility of human genetics to develop small molecule precision medicines, Maze Therapeutics’ strategy is notable for its emphasis on understanding genetic drivers of frequent illnesses, an space that has historically been difficult within the discipline of drug discovery. To attain this, Maze has a platform known as Compass, which identifies gene variants which might be related to illness and unravels what these variants do to be able to establish new targets and medicines. 

The corporate’s primary focus areas are renal and cardiometabolic indications. Its lead candidate, MZE829, is an oral APOL1 inhibitor being examined for APOL1-mediated kidney illness, a sort of kidney illness primarily affecting people of African descent that’s attributable to inheriting two danger variants of the APOL1 gene, which ends up in an elevated danger of persistent kidney illness and kidney failure. The primary affected person was dosed in a section 2 trial of MZE829 in February this yr after constructive section 1 outcomes confirmed that the candidate was well-tolerated at single doses as much as 480mg and a number of doses as much as 350mg, with all treatment-related hostile occasions reported as gentle, and no extreme or severe hostile occasions reported.

Maze has raised a major amount of cash because it launched with $191 million in 2019; in 2022, it raised one other $190 million, earlier than happening to bag $115 million in a sequence D spherical in December 2024 and $140 million in an IPO in January 2025. 

Tasca Therapeutics 

• Illness space: Oncology 
• Lead candidate: CP-383
• Latest information: Launched with $52 million in sequence A funding 

Named after the Italian phrase for “pocket,” Tasca Therapeutics is harnessing the facility of mass spectrometry-based proteomics to establish cancer-causing proteins that bear a particular post-translational modification known as auto-palmitoylation, which is able to in the end permit the corporate to make the most of beforehand unknown binding pockets to develop novel small molecule inhibitors for difficult-to-treat cancers.

Tasca’s most superior candidate, CP-383, is being developed to deal with sufferers with tumors harboring a particular genetic profile. It has demonstrated sturdy anti-cancer exercise in each in vitro and in vivo research throughout a broad vary of human cancers, and can initially be evaluated in small cell lung most cancers, colorectal most cancers, head and neck most cancers, and mind most cancers sufferers. 

In December 2024, Tasca launched with $52 million in sequence A funding. The corporate stated that the proceeds from this may be used to advance the corporate’s drug discovery platform, progress CP-383 into section 1/2 medical proof-of-concept research, and develop its drug candidate pipeline.

Vivace Therapeutics 

• Illness space: Oncology
• Lead candidate: VT3989
• Latest information: Raised $35 million in sequence D funding 

Small molecule drug discovery firm Vivace Therapeutics is creating most cancers therapies focusing on the Hippo-YAP pathway, which has been proven to be concerned within the regulation of cell proliferation, programmed cell demise, and cell migration. In wholesome cells, the pathway controls tissue regeneration and the scale and form of organs, however mutations within the Hippo-YAP pathway can drive a number of types of most cancers, which rely upon activated YAP to outlive and develop.

Even though researchers have lengthy identified concerning the position of YAP in some cancers, they may not discover a option to flip the protein off, as there have been no apparent drug targets like the standard kinases or receptors which might be often focused in oncology drug discovery. Due to this fact, as an alternative of aiming at YAP immediately, Vivace is focusing on proteins within the transcriptional enhanced affiliate area (TEAD) household. It is because, for YAP to work, it must kind a fancy with TEAD, and for TEAD to work, it must have a fatty acid known as palmitic acid covalently connected to it. Vivace’s compounds block the attachment of the fatty acid onto TEAD, making it unable to mix with YAP, in flip affecting the survival mechanism of sure cancers.

The corporate’s lead small molecule candidate, VT3989, has been evaluated in additional than 150 sufferers to this point in an ongoing section 1 examine. Medical findings for the candidate have been notably notable in sufferers with mesothelioma who’ve failed chemotherapy and immuno-oncology mixture regimens. Vivace stated it should current the outcomes at a serious medical convention within the second half of 2025, however based mostly on these knowledge, it’s already working to advance the candidate towards a section 3 medical trial in sufferers with mesothelioma and intends to debate its plans with the U.S. Meals and Drug Administration (FDA) later this yr.

Furthermore, in March, the corporate raised $35 million in sequence D funding to help the continued medical growth of VT3989. 

Small molecule drug discovery market set for vital development

Contemplating the variety of FDA approvals small molecule medication have lately acquired, there may be cause to consider that we is likely to be within the “golden age” of small molecule drug discovery, largely due to the development of synthetic intelligence (AI). 

Actually, the worldwide small molecule drug discovery market dimension, which was valued at $52.32 billion in 2023, is anticipated to succeed in an unimaginable $107.04 billion in 2032, rising at a compound annual development fee (CAGR) of 8.3% over the forecast interval. Which means small molecule medication are prone to proceed to be the cornerstone of the biopharma trade for a few years to return. 

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