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Home » Topics » Drugs » Small molecule

Small molecule
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Lung illustration

Phase II home run: Insmed’s TPIP hits hypertension endpoints

June 10, 2025
By Karen Carey
No Comments
Insmed Inc.’s chair and CEO, Will Lewis, called the phase IIb trial of TPIP in pulmonary arterial hypertension a “clear and unequivocal success,” with analysts and investors wholeheartedly agreeing, as the company’s shares surged 28.7% June 10.
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Digital rendering of molecular structures
Drug design, drug delivery & technologies

Open-source AI model can predict small-molecule binding affinity

June 10, 2025
By Anette Breindl
No Comments
Researchers at the Massachusetts Institute of Technology and Recursion Pharmaceuticals Inc. have released an open-source AI model that can predict the binding strength of small molecules as well as structures of proteins and biomolecular complexes. The model, which is called Boltz-2 and was released by the research team on the developer platform Github on June 6, addresses a major bottleneck in drug discovery with its improved ability to predict binding strengths.
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Cancer

Mitotic motor protein Eg5 inhibitor Arry-520 to treat gastric cancer

June 10, 2025
No Comments
Gastric cancer, which is the fifth most frequent cancer globally and the third most frequent cause of cancer-related deaths, can be managed relatively well with existing therapies. Advanced gastric cancer, in contrast, is more difficult to manage.
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Digital rendering of molecular structures

Open-source AI model can predict small-molecule binding affinity

June 9, 2025
By Anette Breindl
No Comments
Researchers at the Massachusetts Institute of Technology and Recursion Pharmaceuticals Inc. have released an open-source AI model that can predict the binding strength of small molecules as well as structures of proteins and biomolecular complexes. The model, which is called Boltz-2 and was released by the research team on the developer platform Github on June 6, addresses a major bottleneck in drug discovery with its improved ability to predict binding strengths.
Read More
Knee pain illustration

Allay’s $57.5M extends postsurgical pain relief with ATX-101

June 6, 2025
By Karen Carey
No Comments
As it advances its nonopioid analgesic ATX-101 breakthrough therapy through a phase IIb registration trial, Allay Therapeutics secured $57.5 million in a series D round, which included an investment from the company’s Japanese partner. ATX-101, a configuration of sodium ion channel blocker bupivacaine and a biopolymer, which is in a phase IIb registration study, is designed to offer pain relief following total knee arthroplasty.
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Ozempic pen and packaging

EMA warns Ozempic linked to ‘very rare’ side effect

June 6, 2025
By Nuala Moran
No Comments
The EMA’s safety committee has issued a warning that the GLP-1 receptor agonist Ozempic (semaglutide, Novo Nordisk A/S) can cause an acute eye condition in which the optic nerve is damaged by a sudden loss of blood supply. After reviewing several large epidemiological studies, clinical trial and in-market data, EMA’s Pharmacovigilance Risk Assessment Committee has concluded non-anterior ischemic optic neuropathy is a “very rare” side effect of Ozempic, that “may affect up to one in 10,000 people taking semaglutide.
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Pregnant woman

EMA: New clinical guideline includes pregnant, breastfeeding patients

June 5, 2025
By Nuala Moran
No Comments
The EMA has issued a new guideline on how to include and/or retain pregnant and breastfeeding women in clinical trials, in a move that it says “marks a change in the paradigm.” The aim is to ensure that trial sponsors generate robust clinical data in these populations.
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Ascletis’ denifanstat meets phase III endpoints in acne

June 5, 2025
By Tamra Sami
No Comments
Ascletis Pharma Inc.’s once-daily oral fatty acid synthase inhibitor, denifanstat, demonstrated statistically significant and clinically meaningful improvements compared to placebo, meeting all primary and secondary endpoints in a phase III trial for moderate to severe acne vulgaris.
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Kymera’s phase I data lead big week for STAT6 space

June 4, 2025
By Jennifer Boggs
No Comments
It’s a good week to be working on drugs targeting STAT6. Kymera Therapeutics Inc.’s, KT-621, the first oral STAT6 degrader candidate to enter the clinic, surpassed expectations with impressive safety, pharmacokinetic and biomarker data from a phase I trial, while potential fast-followers from Nurix Therapeutics Inc. and Recludix Pharma Inc. advanced via respective partnerships with Sanofi SA.
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Anatara takes a second look at Garp after phase II IBS miss

June 3, 2025
By Tamra Sami
No Comments
After reporting in April that its gastrointestinal reprogramming product (Garp) failed to meet the primary efficacy endpoint in a phase II trial in irritable bowel syndrome (IBS), Anatara Lifesciences Ltd. conducted a further analysis that shows a positive trend toward efficacy.
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