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BioWorld - Monday, December 29, 2025
Home » Topics » Drugs » Small molecule

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Scientific illustration of a migrating breast cancer cell
Cancer

HDAC inhibitor prevents breast cancer metastasis

July 30, 2024
Researchers from Northeast Normal University in Changchun demonstrated that compound AW-01178 reversed the abnormal regulation of E-cadherin by inhibiting the class I HDAC enzyme.
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Coins and seedling
Newco news

Draupnir engaging sortilin receptor with €12M seed round

July 29, 2024
By Nuala Moran
Draupnir Bio is poised to advance a new approach to targeted protein degradation by engaging the sortilin receptor on lysosomes to promote the destruction of extracellular and membrane-bound disease proteins.
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Elderly hands holding broken brain structure

Despite endpoint miss, Cognition sees path forward in Alzheimer’s

July 29, 2024
By Jennifer Boggs
Cognition Therapeutics Inc. said it is moving to the next stage of clinical testing with oral, small-molecule candidate CT-1812, despite phase II efficacy results falling short of statistical significance in patients with mild to moderate Alzheimer’s disease and sending shares of the Purchase, N.Y.-based company (NASDAQ:CGTX) falling 44%, or $1.04, to close July 29 at $1.33.
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Hands holding holographic intestine
Newco news

Ensho debuts to target gut inflammation with oral α4β7 inhibitors

July 26, 2024
By Marian (YoonJee) Chu
Ensho Therapeutics Inc. launched in July after licensing a pipeline of four oral α4β7 inhibitors for inflammatory and gastrointestinal disorders, including inflammatory bowel disease (IBD), from EA Pharma Co. Ltd. “Millions of people worldwide are living with IBD,” Ensho founder, president and executive chair Neena Bitritto-Garg recently told BioWorld, “and while there are a number of approved medications to address the symptoms of IBD, it remains a difficult-to-treat disease with high relapse rates for a considerable proportion of patients.”
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From Day One, Ipsen gains ex-US rights to tovorafenib for $461M

July 26, 2024
By Marian (YoonJee) Chu
Ipsen SA, of Paris, struck a $461 million deal with Day One Biopharmaceuticals Inc. for ex-U.S. rights to tovorafenib, an oral drug for pediatric brain tumor that gained U.S. FDA accelerated approval April 23 as Ojemda (tovorafenib).
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FDA Approved stamp with pills

Sun Pharma’s JAK inhibitor for severe alopecia gets US FDA nod

July 26, 2024
By Lee Landenberger
The U.S. FDA has approved Sun Pharmaceutical Industries Ltd.’s Leqselvi (deuruxolitinib), a JAK1 and JAK2 inhibitor for adults with severe alopecia areata, a chronic autoimmune disease. The twice-daily, oral treatment will be targeting a company-estimated market of about 300,000 people in the U.S. This is the third FDA-approved treatment for severe alopecia areata in the past three years.
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Ocaliva

PBC patients fight back in Europe on looming Ocaliva withdrawal

July 25, 2024
By Nuala Moran
Those affected by primary biliary cholangitis (PBC) are fighting back against the recommendation by the EMA that the marketing authorization for Ocaliva (obeticholic acid), a second-line treatment used by 7,000 patients in Europe, be withdrawn. Earlier today, July 25, patients and their supporters staged a protest outside the European Commission building in Brussels to oppose EMA’s position that Ocaliva’s conditional license should be revoked.
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Shaky hands holding glass

ET left behind as Zurzuvae ship flies for Sage, Biogen in PPD

July 24, 2024
By Randy Osborne
A phase II failure with SAGE-324 in essential tremor (ET) had Wall Street speculating about the fate of Sage Therapeutics Inc.’s partnership with Biogen Inc. Shares of Cambridge, Mass.-based Sage (NASDAQ:SAGE) ended July 24 at $10.38, down $2.70, or 20.6%, after the firm disclosed top-line results from the phase II Kinetic 2 dose-ranging study of oral SAGE-324 (also known as BIIB-124) for ET. The trial did not show a statistically significant dose-response relationship based on the primary endpoint, the Essential Tremor Rating Assessment Scale (TETRAS) Performance Subscale Item 4 (upper limb) total score.
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Cerebellum, brain stem, spinal cord

Alterity’s ATH-434 shows early response in multiple system atrophy

July 23, 2024
By Tamra Sami
Aussie biotech Alterity Therapeutics Ltd. released interim data of an open-label phase II trial for lead candidate ATH-434 for treating multiple system atrophy that showed 43% of participants improved, with 29% showing either stable or improved neurological symptoms.
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Red dart in center of red target

Innovent’s mazdutide hits phase III endpoints in type 2 diabetes

July 23, 2024
By Tamra Sami
Innovent Biologics Inc.’s glucagon-like peptide-1 receptor (GLP-1R) and glucagon receptor (GCGR) dual agonist, mazdutide, met the primary endpoint and all key secondary endpoints in a phase III type 2 diabetes trial in Chinese adults. “Mazdutide is the first and fastest-developed GLP-1R/GCGR dual agonist in the world, and we are actively promoting the development of mazdutide for weight loss, T2D and other indications,” Lei Qian, Innovent’s vice president of clinical development, said.
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