4DMT Molecular Therapeutics (NASDAQ:FDMT) was trading up 75% at midday on interim data from the phase II Prism trial testing gene therapy candidate 4D-150 in wet age-related macular degeneration. Results presented over the weekend at the Angiogenesis, Exudation, and Degeneration 2024 Conference showed the single-shot therapy, which uses 4D’s R100 vector for intravitreal delivery to the retina, significantly reduced treatment burden, with patients in the high-dose arm showing an 89% reduction in anti-VEGF injection rates. The company noted that the patient cohort comprised those with severe disease and high treatment burdens, making the results even more pronounced. Calling the 4D-150 “potentially highly disruptive,” CEO David Kirn said the next step will be to work with regulators to launch a phase III program in the first quarter of 2025.

J&J’s $6.5B investment prompts positive phase II and III data

In August 2020, Johnson & Johnson paid $6.5 billion cash to acquire Momenta Pharmaceuticals Inc. That strengthened J&J’s immune-mediated disease portfolio and grew its interest in autoantibody-driven disease therapies by bringing nipocalimab into the fold. Now the investment is paying off with top-line results of phase II and III studies that hit their primary endpoints using the fully human glycosylated monoclonal antibody targeting the human neonatal Fc receptor. The phase II Dahlias study of adults with Sjögren’s disease showed a statistically significant reduction in a disease activity index score from baseline at week 24 compared with placebo. The pivotal phase III Vivacity study of adults with generalized myasthenia gravis showed a statistically significant reduction in a daily activity score from baseline over weeks 22 to 24 compared with placebo. Vyvgart (efgartigimod), from Argenx SE, was approved by the U.S. FDA for treating myasthenia gravis in December 2021 and expanded as a subcutaneous formulation in June 2023.

Pliant dips on positive phase II data with bexotegrast in PSC

Pliant Therapeutics Inc. offered 12-week interim data from the 320-mg dose group of Integris-PSC, a multinational, randomized, double-blind, placebo-controlled phase II trial testing bexotegrast in primary sclerosing cholangitis (PSC) and suspected moderate to severe liver fibrosis. The group met its primary and secondary endpoints demonstrating that bexotegrast – a dual integrin alpha-V/beta-1/6 antagonist also known as PLX-4809 – was well-tolerated over a 12-week period, and plasma levels rose with dosage. Along with PSC, the compound is under phase II investigation for idiopathic pulmonary fibrosis. South San Francisco-based Plant’s shares (NASDAQ:PLRX) were trading midday at $13.91, down $3.49, or 20%.

Discussion bill would codify 340B payments to contract pharmacies

Court rulings favoring biopharma companies that have challenged the U.S. Department of Health and Human Services’ efforts to force them to unconditionally offer 340B prescription drug discounts to an unlimited number of contract pharmacies could become moot in the future if a bipartisan draft bill introduced in the Senate becomes law. Released Friday, the draft discussion bill would codify that drug manufacturers have to offer the discounts to contract pharmacies and that they could be charged civil monetary penalties for intentionally refusing to do so. However, those provisions aren’t written in stone yet. In releasing the bill, the Senate 340B bipartisan working group asked stakeholders to submit comments and proposed edits to the draft by April 1. The senators specifically asked a number of questions on how best to achieve the correct balance of patient access, accountability and program integrity in the use of contract pharmacy arrangements.

Bridgebio seeks Japan approval of acoramidis for rare heart disease 

Armed with strong phase III safety data in Japanese patients, Palo Alto, Calif.-based Bridgebio Pharma Inc. is planning to file for Japan approval of its investigational drug, acoramidis, for a rare heart disorder. Acoramidis is an oral, highly potent, small-molecule stabilizer of transthyretin (TTR) for transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM) — a rare, progressive and fatal heart condition caused by misfolded TTR proteins that accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Data from the latest single-arm, open-label, multicenter, prospective phase III study showed no mortalities over the 30-month study period. Acoramidis was also well-tolerated, with no safety signals of potential clinical concern identified.

Novo Nordisk paying $11B for three Wegovy fill-finish sites

As Novo Nordisk A/S has struggled with supply of its GLP-1 drug Wegovy (semaglutide), holding and investment company Novo Holdings A/S has agreed to acquire global contract development and manufacturing organization Catalent Inc. and its more than 50 global sites for $63.50 per share in cash, about $16.5 billion. Once the transaction closes, expected toward the end of 2024, Novo Nordisk then intends to acquire from a Novo Holdings subsidiary three manufacturing sites that specialize in the sterile filling of drugs for $11 billion up front. The sites, with more than 3,000 employees, are located in Anagni, Italy, Brussels, and Bloomington, Ind. Novo Nordisk already has ongoing fill-finish collaborations with the sites for Wegovy pens.

Gate Neuro and Beacon illuminate depression biomarkers with EEG

A partnership between Gate Neurosciences Inc. and Beacon Biosignals Inc. promises to advance the field of precision psychiatry by using electroencephalogram biomarkers to diagnose depression and rapidly assess response to medications. The collaboration will first use Beacon’s FDA-cleared Dreem 3S headband device and neurobiomarker platform to conduct EEGs in participants in Gate’s phase II trial of zelquistinel, a small-molecule NMDA receptor modulator under development as an antidepressant.

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Abivax, Advantage, Adverum, Arcellx, Atara, Avicanna, Biosenic, Bridgebio, Cabaletta, Coherus, Corbus, DMK, Eli Lilly, Ferrer, Genmab, Junshi, Lexeo, MBX, Memo, Metagenomi, Neximmune, Nodthera, Qilu, Regeneron, Revance, Silence, Takeda, TME, Tyra, Valneva, Vaxcyte, Zuellig