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Home » Topics » Drugs » Small molecule

Small molecule
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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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3D illustration of pancreatic cancer

ASCO 2025: Verastem has positive cancer data but stock sags

June 3, 2025
By Lee Landenberger
No Comments
New dose-escalation data from Verastem Oncology’s phase I/II cancer study in China prompted the company to say it was encouraged by the efficacy results. However, investors felt otherwise, as the stock lost about 20% of its value the day the initial results were released.
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Sanofi-signage on office facade in Edinburgh, U.K.

Sanofi buying Blueprint and its TKI BLU-808 for $9.5B

June 2, 2025
By Nuala Moran
No Comments
Sanofi SA is to acquire Blueprint Medicines Inc. in a $9.5 billion deal that will give the French pharma ownership of a marketed rare disease immunotherapy heading toward blockbuster status, a follow-on product in phase II/III, and a phase II clinical program relevant to a number of autoimmune diseases.
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LYR-210 device unfurling

Redemption for Lyra as second phase III rhinosinusitis trial hits goal

June 2, 2025
By Jennifer Boggs
No Comments
Shares of Lyra Therapeutics Inc. soared more than 310% on data from its second phase III trial testing drug-device candidate LYR-210 in patients with chronic rhinosinusitis, with results from the Enlighten 2 study showing statistical significance on primary and key secondary endpoints and offering hopes of a regulatory pathway ahead.
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Anatara takes a second look at Garp after phase II IBS miss

May 28, 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.
Read More
3D-rendered illustration of a synapse cross-section

Lilly adds non-opioid painkiller to pipeline in $1B Siteone buyout

May 28, 2025
By Jennifer Boggs
No Comments
Four months after Vertex Pharmaceuticals Inc.’s U.S. FDA nod for Journavx (suzetrigine) as the first drug targeting NaV1.8 for treating pain, Eli Lilly and Co. is joining the potential competition via a buyout of Siteone Therapeutics Inc., a privately held firm developing small-molecule sodium channel inhibitors, including a phase II-ready NaV1.8 inhibitor.
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Spero with white knight GSK succeed with UTI treatment

May 28, 2025
By Lee Landenberger
No Comments
GSK plc knew a good thing when it swooped in nearly three years ago to rescue Spero Therapeutics Inc.’s oral antibiotic from a complete response letter (CRL). New pivotal phase III study data showed tebipenem pivoxil hydrobromide (HBr) for complicated urinary tract infections (cUTI), including pyelonephritis, hit its primary endpoint.
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Grin’s radiprodil heads to phase III with $570M deal, $140M series D

May 27, 2025
By Karen Carey
No Comments
A new version of a drug candidate that failed in a phase II for neuropathic pain 15 years ago has attracted $140 million in series D funds, plus an ex-North American deal worth up to $570 million, for Grin Therapeutics Inc.
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