5 Top Small Molecule Drug Solutions Impacting The Pharma Sector

We analyzed 244 small molecule drug solutions. Exxel Pharma, Inositec, Q2Pharma, Oppilan Pharma & Attune Pharmaceuticals develop 5 top solutions you should watch out for. Learn more in our Global Startup Heat Map!

Our Innovation Analysts recently looked into emerging technologies and up-and-coming startups working on innovative solutions for the pharma sector. As there is a large number of startups working on a wide variety of solutions, we want to share our insights with you. This time, you will discover 5 promising small molecule drug solutions.

Heat Map: 5 Top Small Molecule Drug Solutions

Using our StartUs Insights Discovery Platform, covering 1.379.000+ startups & scaleups globally, we looked at innovation in the field of pharma. For this research, we identified 244 relevant solutions and picked 5 to showcase below. These companies were chosen based on a data-driven startup scouting approach, taking into account factors such as location, founding year, and relevance of technology, among others. Depending on your specific criteria, the top picks might look entirely different.

The Global Startup Heat Map below highlights 5 startups & scaleups developing small molecule drugs. Moreover, the Heat Map reveals regions that observe a high startup activity and illustrates the geographic distribution of all 244 companies we analyzed for this specific topic.


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Which startups develop the other 239 solutions?


Exxel Pharma – Pain Management

Small molecule drugs are typically simple and inexpensive to produce, screen, orally administer, and easily cross the cell membrane to reach target cells. Opioids have long been the choice of drugs in pain management but often lead to tolerance and, subsequently, addiction. Startups are working on small molecule alternatives to opioids that are more effective and non-addictive.

Exxel Pharma is a US-based startup that develops small-molecule drugs for non-addictive treatment of pain, post-traumatic stress disorder (PTSD), and substance use disorders. The startup’s lead drug, URB397, is an opioid alternative for the treatment of chronic neuropathic pain. Moreover, the drug acts on and inhibits peripheral fatty acid amide hydrolase (FAAH). URB397 also demonstrates analgesic effects in animal models of nociceptive, inflammatory, and neurogenic pain.

Inositec – Cardiovascular Diseases

Cardiovascular diseases are a major cause of death globally, killing tens of millions annually. There is a huge variety of clinical indications, both heritable and lifestyle-related, that suffer from a lack of effective therapeutic interventions. To this end, startups are working on novel small molecule drugs. Many of these drugs inhibit crucial proteins involved in disease development, leading to treatments for unmet medical needs.

Swiss startup Inositec works on drugs based on inositol phosphate (IP6). INS-3001, the startup’s lead candidate, is a vascular calcification inhibitor. The drug is under investigation for the treatment of aortic valve stenosis and arterial stiffness. Inositec’s pipeline also includes drugs for the treatment of renal calcification disorders and Clostridium difficile infection.

Q2Pharma – Multi-Drug Resistant (MDR) Bacteria

Prolonged use of antibiotics leads to the development of antibiotic resistance in pathogenic bacteria. MDR bacteria develop resistance against multiple antibiotics and pose a major challenge for the pharma and healthcare industries. However, pharma startups are developing alternatives that effectively combat MDR bacteria. This includes antivirulents that eliminate the virulence of bacteria instead of killing them.

Q2Pharma is an Israeli startup that develops small molecule drugs against MDR bacteria. F19, the startup’s lead antivirulent, tackles multi-drug-resistant bacteria such as S. aureus and E. coli. The drug inhibits the expression of toxins and virulence factors by the bacteria. F19 is under development as both a standalone treatment, as well as in combination with existing drugs.

Oppilan Pharma – Autoimmune Diseases

Treatment of autoimmune diseases generally focuses on the suppression of the autoimmune response. In recent years, many new targets have been identified that exploit the underlying mechanisms in autoimmunity. Since most of these are intracellular targets, they aren’t easily accessible to biologics. Pharma startups are working on small molecule drugs against these intracellular targets in autoimmune diseases.

British startup Oppilan Pharma offers novel small molecule therapeutics for the treatment of autoimmune disorders. The startup’s lead drug candidate, OPL-002, is a selective modulator of sphingosine 1 phosphate receptor subtype 1 (S1P1). The drug is in clinical trials for the treatment of inflammatory bowel disease (IBD), as well as other indications such as ulcerative colitis and Crohn’s disease.

Attune Pharmaceuticals – Rare Diseases

Availability of fewer patients for clinical trials and poor knowledge of the diseases are obstacles for rare disease drug development. However, advances in drug discovery and development, such as the use of artificial intelligence (AI) algorithms, make it easier to identify druggable targets. Governments and authorities also provide special status for targeting rare diseases. This incentivizes pharma startups to develop novel drugs for the treatment of rare diseases.

Attune Pharmaceuticals is a US-based startup that develops orally-administered small molecule drugs. The startup’s lead drug, ATN-249, is under clinical investigation for the treatment of hereditary angioedema (HAE). The drug inhibits plasma kallikrein, leading to a lower frequency of HAE attacks. The startup’s pipeline also includes drugs against paroxysmal nocturnal hemoglobinuria and other complement-mediated diseases.

What About The Other 239 Solutions?

While we believe data is key to creating insights it can be easy to be overwhelmed by it. Our ambition is to create a comprehensive overview and provide actionable innovation intelligence and enable you to achieve your goals faster. The 5 small molecule drug solutions showcased above are promising examples out of the 244 we analyzed for this article. To identify the most relevant solutions based on your specific criteria, get in touch.

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