Shares of Lipocine Inc. (NASDAQ:LPCN) were trading at $2.01, down $7.24, or 78%, on top-line phase III data from the placebo-controlled trial evaluating LPCN-1154 (oral brexanolone) for the treatment of postpartum depression. A synthetic formulation of the endogenous neurosteroid allopregnanolone, the drug failed to show a statistically significant reduction from baseline in Hamilton Depression Rating Scale total score compared to placebo at hour 60 in the full analysis set and the primary endpoint was not met. On the upside, LPCN-1154 was well-tolerated, with a safety profile that suggests the drug could be given to outpatients without the need for healthcare provider monitoring. Also, a post-hoc analysis of participants with a history of psychiatric conditions turned up signals that could indicate a development path for the candidate, Lipocine said.

EMA poised to approve first NAM to replace live animal controls in toxicity tests

The EMA has given initial endorsement to a new approach to early toxicity testing in which live animals will be replaced by virtual counterparts. The virtual control groups will be derived from data generated in animals that have been used as controls in previous studies. The historical database has been brought together in an EU-funded project to which 20 pharma and crop sciences companies contributed. While limited in scope, this is the first time the EMA has formally endorsed the use of a new approach methodology (NAM) to generate data that could eventually be included in an application for marketing approval.

LHON updates: Gene therapy progress; idebenone receives US CRL

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). Administered as an intravitreal injection, Lumevoq is a recombinant adeno‑associated virus serotype 2 vector designed to deliver the MT-ND4 (human mitochondrially encoded ND4) gene.

Blockbuster deals push med-tech M&A above $40B in Q1 2026

Med-tech M&A activity surged in the first quarter of 2026, with deal value reaching $41.86 billion, marking one of the strongest quarterly openings in recent years. The largest med-tech M&A transaction of Q1 was Abbott Laboratories’ $21 billion acquisition of Exact Sciences Corp. in March, followed by Becton, Dickinson and Co.’s combination of its Biosciences and Diagnostic Solutions business with Waters Corp. in a $17.5 billion transaction that closed in February.

Newco news: Ambrosia slurps up a $100M series B for its oral obesity drug pipeline

Ambrosia Biosciences Inc., named after the drink of the Greek gods, secured a $100 million series B to advance its preclinical pipeline of oral obesity drugs. The startup formed after Pfizer Inc. shuttered its Boulder, Colo.-based research facility that the pharma gained through its 2019 acquisition of Array Biopharma Inc. “We caught wind that was happening, and we really used that as the nexus for the formation of Ambrosia,” Nick Traggis, founder and CEO of Ambrosia, told BioWorld. “Think of it as kind of putting the band back together.”

Hematopoietic stem cell research points to leukemia’s early roots

Hematopoietic stem cell research over the past century has shown that leukemia may be driven by an invisible hand of inflammation. The bone marrow and inflammation, then, may hold the keys to preventing blood cancers, according to John E. Dick’s plenary session at the 2026 Korean Society of Hematology International Conference, held March 26, 2026.

Holiday notice

BioWorld's offices will be closed in observance of Good Friday. No issue will be published April 3.

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