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BioWorld - Sunday, April 5, 2026
Home » Topics » Drugs » Small molecule

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Eye and DNA illustration

LHON updates: Gene therapy progress; idebenone receives US CRL

April 2, 2026
By Marian (YoonJee) Chu
No Comments
Chiesi Group’s idebenone faced a regulatory setback last month after the U.S. FDA issued a complete response letter (CRL) to the company’s NDA for Leber hereditary optic neuropathy (LHON), a rare inherited disorder that causes sudden vision loss.But a chance missed for Chiesi may be an opportunity for gene therapies, including Gensight Biologics SA’s lenadogene nolparvovec (Lumevoq; GS-010).
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Wegovy

Wegovy pricing deal offers UK reimbursement for heart disease

April 1, 2026
By Nuala Moran
No Comments
Wegovy (semaglutide) has passed the notoriously strict cost-effectiveness scrutiny of the U.K. health technology assessment body and will now be reimbursed for the prevention of further serious cardiovascular events in people who have had a heart attack or stroke, or are diagnosed with peripheral arterial disease.
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PET scans showing before and after treatment with VIPOR
ICKSH 2026

Five-drug VIPOR regimen shows promise in aggressive blood cancer

April 1, 2026
By Marian (YoonJee) Chu
No Comments
Deep molecular advances are enabling precision medicine for the field of hematology, Wyndham Wilson said during a plenary session at the 2026 Korean Society of Hematology International Conference March 26.
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Eli Lilly logo atop Lilly Biotechnology Center campus in San Diego, Calif.

Lilly’s Foundayo enters oral GLP-1 obesity market with speedy FDA nod

April 1, 2026
By Jennifer Boggs
No Comments
Eli Lilly and Co. anticipates shipping newly approved Foundayo (orforglipron) within the next week, as the drug becomes the second oral weight-loss glucagon-like peptide-1 (GLP-1) receptor agonist to enter the U.S. market following December’s approval of Novo Nordisk A/S’ Wegovy (semaglutide) pill.
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Pill in immersive interface

Lilly taps Insilico AI in $2.75B deal to expand pipeline options

March 31, 2026
By Tamra Sami
No Comments
Eli Lilly and Co. is deepening its investment in artificial intelligence-driven drug discovery through a multibillion-dollar expansion of its collaboration with Hong-Kong listed Insilico Medicine Inc. in a move that could broaden its reach into next-generation metabolic therapies. Under the deal terms, Insilico is eligible to receive $115 million up front, plus development, regulatory, and commercial milestone payments worth $2.75 billion, in addition to sales-based royalties. In exchange, Lilly gains exclusive global rights to develop and commercialize multiple candidates generated using Insilico’s AI platform, including preclinical oral therapies.
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Brain with handshake and cityscape

Otsuka to acquire PTSD drugmaker Transcend in $1.2B deal

March 31, 2026
By Marian (YoonJee) Chu
No Comments
Otsuka Pharmaceutical Co. Ltd. is taking a neuroplastogen approach to posttraumatic stress disorder (PTSD) through its planned $1.22 billion acquisition of Transcend Therapeutics Inc. The deal gives Tokyo-headquartered Otsuka access to Transcend’s lead asset, TSND-201, an oral neuroplastogen that has begun patient recruitment for a phase III study in the U.S.
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Seo Jin-seok of Celltrion (right) with Ko Kwang-pyo of Kobiolabs (left)

Korea roundup: Alteogen, Celltrion lead biotech deals

March 31, 2026
By Marian (YoonJee) Chu
No Comments
Subcutaneous formulation technology, microbiomes and oncological assets drove dealmaking in South Korea’s biotech sector this week. 
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3D illustration of heart cross section

Braveheart finds phase II positive for cardiac myosin inhibitor

March 31, 2026
By Marian (YoonJee) Chu
No Comments
Braveheart Bio Inc. reported positive results from an open-label randomized phase II dose-ranging study of BHB-1893 (HRS-1893) in obstructive hypertrophic cardiomyopathy. Results presented at the American College of Cardiology’s Annual Scientific Session and Expo in New Orleans showed BHB-1893 treatment was associated with rapid and substantial reductions in left ventricular outflow tract gradient, the primary endpoint of the study.
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US FDA says no, again, to Vanda’s tradipitant in gastroparesis

March 31, 2026
By Mari Serebrov
No Comments
The U.S. path forward is narrowing for Vanda Pharmaceuticals Inc.’s tradipitant as a treatment for gastroparesis, a serious disorder for which there’s been no new treatment in several decades.
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Handshake with charts, maps, data

Highly complementary Biogen pays $5.6B; who’s jealous of Apellis?

March 31, 2026
By Randy Osborne
No Comments
Biogen Inc. hardly blinked at the competition faced by Apellis Pharmaceuticals Inc. as the two companies sealed a deal whereby the former has agreed to acquire all outstanding shares of the latter for $41 each, or about $5.6 billion.
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