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BioWorld - Sunday, April 12, 2026
Home » Topics » Drugs » Small molecule

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Centessa touts OX2R agonist data in broad narcolepsy trial

Nov. 5, 2025
By Jennifer Boggs
No Comments
Centessa Pharmaceuticals plc’s unremarkable third-quarter 2025 earnings report was greatly overshadowed by an early readout from its orexin receptor 2 (OX2R) agonist program in narcolepsy, particularly promising phase IIa data demonstrating ORX-750’s efficacy across a group of sleep disorders, though investors seek further data to differentiate Centessa’s program from potential competitors such as Takeda Pharmaceutical Co. Ltd.’s oveporexton and Alkermes plc’s alixorexton.
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Scissors cutting stack of $100 bills

Biohaven CRL leads to cutbacks and program reprioritization

Nov. 5, 2025
By Lee Landenberger
No Comments
The U.S. FDA’s complete response letter (CRL) for Biohaven Ltd.’s lead asset, troriluzole, to treat spinocerebellar ataxia has prompted a wave of downstream changes at the company. There will be a roughly 60% cutback in annual R&D spending, not including personnel, as Biohaven focuses on three other late-stage clinical programs.
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Abstract molecules illustration

Boehringer licenses Kyowa Kirin's small molecule for autoimmune disease

Nov. 4, 2025
By Tamra Sami
No Comments
Boehringer Ingelheim GmbH has licensed an unnamed small-molecule preclinical candidate from Kyowa Kirin Co. Ltd. in the autoimmune disease space in a deal worth up to €640 million (US$739 million).
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Stock chart, red down arrow

Incyte aboard, SMARCA2 shift by Prelude enshrouds Foghorn

Nov. 4, 2025
By Randy Osborne
No Comments
Just as investors were looking ahead to news by year-end on Prelude Therapeutics Inc.’s SMARCA2-targeted degraders, the firm said work in the space will be halted, with efforts shifting toward the mutant selective JAK2V617F JH2 inhibitor program by way of a new deal with Incyte Corp.
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UCB’s ultra-rare disease drug Kygevvi approved by FDA

Nov. 4, 2025
By Lee Landenberger
No Comments
The U.S. FDA has approved UCB SA’s Kygevvi (doxecitine and doxribtimine), the first treatment for the ultra-rare, genetic and life-threatening mitochondrial disease thymidine kinase 2 deficiency. The approval comes as the company reaps a reward of rebuilding and reorganizing that it began little more than a year ago.
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Stock turns sharply higher with Terns’ leukemia data

Nov. 3, 2025
By Lee Landenberger
No Comments
Early stage data from Terns Pharmaceuticals Inc.’s lead candidate showed a large reduction in the number of leukemia cells in those with previously treated chronic myeloid leukemia. Results from the ongoing phase I Cardinal study of TERN-701, an oral, allosteric BCR-ABL tyrosine kinase inhibitor, were “unprecedented,” according to Terns.
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Abstract molecules illustration

Boehringer licenses Kyowa Kirin's small molecule for autoimmune disease

Oct. 31, 2025
By Tamra Sami
No Comments
Boehringer Ingelheim GmbH has licensed an unnamed small-molecule preclinical candidate from Kyowa Kirin Co. Ltd. in the autoimmune disease space in a deal worth up to €640 million (US$739 million).
Read More
Illustration of antibodies attacking neurons
Immune

Boehringer Ingelheim licenses autoimmune program from Kyowa Kirin

Oct. 30, 2025
No Comments
Boehringer Ingelheim GmbH and Kyowa Kirin Co. Ltd. have announced that Boehringer Ingelheim has licensed a preclinical program from Kyowa Kirin to develop a potential first-in-class, small molecule for the treatment of autoimmune diseases.
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3D illustration of thyroid in human body with parathyroid glands highlighted

Bridgebio wows again, this time in genetic hypoparathyroidism

Oct. 29, 2025
By Jennifer Boggs
No Comments
Only two days after Bridgebio Pharma Inc. impressed investors with data from BBP-418 in limb-girdle muscular dystrophy type 2I/R9, the company was back at it again, this time reporting positive top-line results from its global phase III study of encaleret in autosomal dominant hypocalcemia type 1, a genetic form of hypoparathyroidism.
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Drug capsule spilling onto brain

Angelini signs $550M deal for Sovargen’s ASO drug in epilepsy

Oct. 29, 2025
By Marian (YoonJee) Chu
No Comments
Sovargen Co. Ltd. inked a $550 million license deal with Angelini Pharma SpA, granting Angelini development and commercialization rights to SVG-105, a novel antisense oligonucleotide drug candidate in preclinical development as a potential treatment for intractable epilepsy.
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