Oral ‘roid void in PPD? Lipocine MINI bike may have wheels
Hopes in postpartum depression (PPD) with an oral version of brexanolone – a synthetic formulation of the endogenous neurosteroid allopregnanolone, approved by the U.S. FDA in 2019 when given intravenously for PPD – were dashed, at least near term, when Lipocine Inc. reported that the candidate failed in a phase III placebo-controlled trial.

Oral ‘roid void in PPD? Lipocine MINI bike may have wheels
Hopes in postpartum depression (PPD) with an oral version of brexanolone – a synthetic formulation of the endogenous neurosteroid allopregnanolone, approved by the U.S. FDA in 2019 when given intravenously for PPD – were dashed, at least near term, when Lipocine Inc. reported that the candidate failed in a phase III placebo-controlled trial.
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).
Today's news in brief
BioWorld briefs for April 2, 2026.
Blockbuster deals push med-tech M&A above $40B in Q1 2026
Med-tech deal value, excluding M&As, totaled $628.41 million in the first quarter (Q1) of 2026, an increase of about 322% from the $149.08 million recorded in Q1 2025 though a 36% drop from Q4 2025‘s $978.58 million.
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.
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.

BioWorld Insider podcast

The opioid crisis may not be front and center anymore, but it’s raging still. Elysium Therapeutics Inc. CEO Greg Sturmer talks about his firm’s candidate for a solution to the medical and societal problem.

Listen now

Analysis and data insight

Assay what? Eyes on Pepgen’s DM1 phase II
Clinical
Phase II data disclosed March 31 by Pepgen Inc. in myotonic dystrophy type 1 (DM1) hobbled the stock but might have been much different if not for one outlier in the 5-mg/kg multiple ascending dose cohort of the ongoing phase II Freedom2-DM1 trial – and Wall Street is pondering what the hitch means...
US FDA pondering next steps for CNPV
Regulatory
Introduced last year as a pilot program, the U.S. FDA Commissioner’s National Priority Voucher (CNPV) could be here to stay – at least for the duration of Marty Makary’s tenure as FDA commissioner. Since the FDA unveiled the CNPV last June, it has welcomed 18 products from 16 companies into the...
Phase III wins lift shares of Compass, Palvella and Bridgebio in February
Clinical
BioWorld tracked 152 clinical trial readouts across phases I through III in February 2026, up from 144 in January but down from 215 in December. Of those, 20 phase III trials reported positive results, while one failed to meet key endpoints. By phase, February’s updates consisted of 50 from phase...
FDA should do more to protect adcoms from conflicts, GAO says
Medical technology
With all the focus of late on the U.S. CDC’s Advisory Committee on Immunization Practices (ACIP), the FDA’s 30-plus advisory committees have been flying under the radar, especially since many of them haven’t met for a few years now.

Financings

Whoop secures $575M, with Abbott a strategic investor
Medical technology
Whoop Inc. secured $575 million in a series G funding round at a $10.1 billion valuation to advance its AI-powered wearable platform for personalized and preventive health care. Abbott Laboratories joined the round as a strategic investor, backing the company’s push to expand access to its device...

Medical technology

Med-tech players join efforts to tackle UK PAD amputations
Cardiovascular
With cases of peripheral artery disease (PAD) rising across the U.K., and lower limb amputations continuing to increase, med-tech companies joined forces with parliamentarians and health care professionals to push for urgent reform of the vascular sector.

US FDA looks to advance use of DHTs in drug trials
Regulatory
Seeking to increase their understanding of the opportunities and challenges of using digital health technologies (DHTs) in drug trials, the U.S. FDA’s CBER and CDER are requesting public feedback to help inform new guidance as the technologies continue to advance.
Supreme Court shuts the door to three life sciences petitions
Regulatory
What do a patent dispute over a CRISPR/Cas system, a rejected whistleblower case involving lab tests and a vaccine injury claim parading as multidistrict tort litigation have in common? All three were denied cert in the U.S. Supreme Court’s latest orders list.

Newco news

Oryon emerges with autologous stem cell therapy for Parkinson’s
Clinical
Oryon Cell Therapies, named after the Orion constellation used for navigation at night, emerged from stealth mode to announce a new round of funding and to present data from the phase Ib/IIa study of its neuron replacement therapy in patients with Parkinson’s disease at the 20th International...
Excalipoint closes $68.7M seed round for cancer T-cell engagers
Financings
Excalipoint Therapeutics Inc. launched with an oversubscribed $68.7 million seed financing round to advance a portfolio of T-cell engagers for solid tumors, marking one of the largest early stage financings in China biotech history.
R1’s $77.5M series A to fund CKD trial of oral phosphate inhibitor
Financings
Newco R1 Therapeutics Inc. has launched with an oversubscribed $77.5 million series A, providing the means to take AP-306, a potentially first-in-class hyperphosphatemia therapy through phase IIb development in patients with chronic kidney disease.
Enodia jumps ahead in Sec61 targeting, adds Kezar’s program
Financings
Enodia Therapeutics Inc. was spun out of The Institut Pasteur by Argobio Studio based on the work of Caroline Demangel, co-founder of Enodia and head of the immunobiology and therapy unit at The Institut Pasteur. Demangel’s lab discovered the mechanism of action of mycolactone, a natural Sec61...
Kyron-Servier deal focused on programmable glycoprotein platform
Deals and M&A
Newco Kyron Bio is showing its colors after sealing a research agreement with Servier SA, underpinning a claim that it has overcome one of the longest-running challenges of the biotech industry, and developing technology to precisely control the glycosylation of therapeutic proteins.

U.S.

Five-drug VIPOR regimen shows promise in aggressive blood cancer
Clinical
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.

Europe

UK’s MHRA sets out new scheme to promote replacement of animal models
MHRA
The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) is to offer an early review of nonanimal data to give companies more confidence that evidence generated with new approach methodologies, such as organoids and microphysiological systems, will be accepted as part of marketing...

CMR works with Nvidia to transform surgical robotics platforms
Medical technology
CMR Surgical Ltd.’s participation in Nvidia’s Physical AI health care robotics initiative is expected to accelerate the development of the next generation of intelligent surgical robotic systems powered by data, simulation and AI. As demand for minimally invasive procedures grows, more companies...
With improved technologies, biomarkers, failed drugs may come into their own
Analysis and data insight
At BioEurope Spring 2026, pharma representatives and investors shared their thoughts about current and future landscapes of different disease areas, and on how to move toward success – both at the level of individual companies and for indications as a whole.

Clinical

Simcere’s rademikibart meets phase III endpoints in atopic dermatitis
Dermatologic
Simcere Pharmaceutical Group Ltd.’s monoclonal antibody, rademikibart (CBP-201), met the primary endpoint in a Chinese phase III study in adults and adolescents with moderate to severe atopic dermatitis.
INHBE inhibitions? Data from Wave send ripples
Analysis and data insight
The debated and ultimately stock-denting March 26 news from Wave Life Sciences Inc. pushed into the spotlight other firms working with INHBE and activin E.
China-developed triple incretin enters global diabetes race
Endocrine/metabolic
A next-generation triple incretin therapy jointly developed by Novo Nordisk A/S and China’s United Biotechnology outperformed semaglutide in a phase II trial, signaling intensifying competition in the GLP-1 obesity and diabetes market.
Kodiak’s Zenkuda sparkles in Glow2, BLA filing next
Ocular
After previous setbacks with the program, investors largely brushed aside ocular-focused Kodiak Sciences Inc.’s anticipated phase III Glow2 data of tarcocimab tedromer in diabetic retinopathy (DR), so the positive top-line superiority results...
Innovent’s IBI-302 meets phase III endpoints in neovascular AMD
Ocular
Innovent Biologics Inc.’s efdamrofusp alfa (IBI-302) met the primary endpoint in the phase III Star trial in neovascular age-related macular degeneration (nAMD), and the Suzhou, China-based company will submit an NDA to China’s National Medical...
PKU ballyhoo averting Street’s gaze from Maze phase II AMKD win?
Endocrine/metabolic
Maze Therapeutics Inc. continues its journey toward a pivotal program after sharing positive top-line data from the phase II Horizon study with MZE-829, an oral, small-molecule, dual-mechanism APOL1 inhibitor, in patients with broad APOL1-mediated...

BioWorld Insider Podcast

One-on-one with medical innovators

podcast microphone, sound waves on purple background Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.

Recent episodes:

View all

BioWorld

The news source of record covering the development of innovative human therapies for 25+ years

BioWorld Asia

A weekly monitor of biopharmaceutical news from the industry’s fastest-growing region

BioWorld Science

Essential discovery and preclinical research news to support crucial drug R&D decisions at the earliest stages

BioWorld Premium

Enjoy extended coverage for the most complete market view with BioWorld, BioWorld Science and BioWorld Asia in a single, easy to access subscription

Today's news in brief

BioWorld

BioWorld Asia

BioWorld Science

BioWorld Premium