Pepgen Inc. said the U.S. FDA placed a partial clinical hold on the Freedom2-DM1 phase II multiple ascending dose, randomized, placebo-controlled trial of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). Questions raised by the agency relate to previously submitted preclinical pharmacology and toxicology studies. No questions regarding blinded data from the phase I Freedom study were cited by the FDA, Pepgen said. The drug deploys Pepgen’s Enhanced Delivery Oligonucleotide technology to deliver a therapeutic oligonucleotide that is designed to restore the normal function of MBNL1, a key RNA splicing protein. Pepgen is providing regulators with additional analyses, including the recently unblinded Freedom data, and aims to address their concerns “as quickly as possible.” Shares (NASDAQ:PEPG) were trading at $5.46, down $1.30, or 19%.

Tenaya, Alnylam join in $1B+ cardio deal for novel genetic targets

In what appears to be its first major cardiovascular disease collaboration, while it advances two internal gene therapies, Tenaya Therapeutics Inc. signed on with Alnylam Pharmaceuticals to deliver up to 15 novel genetic targets that could lead to new heart disease medicines. The deal comes with $10 million up front, and up to $1.13 billion is available to Tenaya if all targets meet certain milestones, leading to approved therapeutics that Alnylam develops and commercializes. While few details were disclosed, Tenaya has plans to work with regulatory authorities this year on pivotal trial alignment for its gene therapies, TN-201 and TN-401, for heart conditions that have no approved disease-modifying treatments.

Study: Weight regain after GLP-1s plateaus below starting weight

A new study has reinforced that GLP-1 receptor agonists are unlikely to produce durable weight loss, but indicates that rather than returning to the starting weight, individual weight gain will plateau at 75.5% of the weight lost. For an individual who had lost a fifth of their weight while taking the drugs, this would correspond to a sustained weight reduction of around 5%. As a result, some weight loss benefits may persist long term, but they are substantially reduced, according to a meta-analysis looking at weight outcomes after stopping GLP-1RAs across 36 randomized controlled trials and 12 non-randomized studies.

Kyowa Kirin discontinues rocatinlimab trials due to safety issues

Shortly after Amgen Inc. walked away from its partnership with Kyowa Kirin Co. Ltd., the Tokyo-based company said it is discontinuing all ongoing clinical trials for rocatinlimab due to safety concerns. As previously reported by BioWorld, Amgen quit its development deal with Kyowa Kirin in February 2026, returning global rights for rocatinlimab, Kyowa Kirin’s T-cell rebalancing therapy being investigated for moderate to severe atopic dermatitis. At the time, Kyowa Kirin said Amgen exited the deal following a strategic portfolio review, and the companies would ensure continuity for clinical trial participants. Amgen licensed rocatinlimab from Kyowa Kirin in a $1.2 billion deal in 2021.

OX40 mechanism in the high beams after Kyowa backs out; others keeping active

The OX40-targeting mechanism pursued by drug developers around the world found itself shadowed when Kyowa Kirin Co. Ltd. gave up work with rocatinlimab (roca), a monoclonal antibody for which hopes had risen in moderate to severe atopic dermatitis, prurigo nodularis and moderate to severe asthma. A number of firms continue to advance their prospects, which include agonists as well as antagonists. Players include such names as Sanofi SA, Inhibrx Biosciences Inc. and Apogee Therapeutics Inc.

Roche to invest ₩710B in South Korea over five years

Roche Holding AG pledged to invest ₩710 billion (US$484.6 million) in South Korea over the next five years, positioning the country as a major global hub for clinical trials. "We expect this agreement to elevate Korea's clinical trial competitiveness to the next level while supporting the rapid growth and global expansion of promising domestic biohealth companies,” Ministry of Health and Welfare head Jeong Eun Kyeong said in Seoul March 3. “We will continue to create numerous opportunities to collaborate with global pharmaceutical companies." The near $500 million agreement inked with the Korean government will bring Roche’s clinical trials for common or incurable diseases and innovative biopharmaceutical products to the country.

Science Corp. raises $230M for Prima BCI retinal implant

Science Corp. raised $230 million in an oversubscribed series C financing round to commercialize its Prima brain-computer interface (BCI) retinal implant, and advance other pipeline programs into the clinic. The investment brings the company’s total funding, since founding in 2021, to approximately $490 million and shows investors growing appetite for BCI technologies.

Med-tech deals show resilience in 2025 despite volatility

Med-tech deal value has been volatile over the past eight years, swinging from $3.12 billion in 2018 to a peak of $10.63 billion in 2023, before dropping to $2.12 billion in 2024 and climbing to $2.69 billion in 2025. The largest med-tech deal of Q4 was announced in November, in which Laborie Medical Technologies Inc. agreed to acquire Organon & Co.’s Jada system for abnormal postpartum uterine bleeding in a transaction valued at up to $465 million

Also in the news

Acadia, Affinia, Aim Immunotech, Alterity, Bayer, Bioversys, Blackstone, Can-Fite, Cerevasc, Citius, Clearmind, Cortechs.ai, Glenmark, Greenland, GSK, Karl Storz, Klotho, Kyowa Kirin, Nervgen, Neuroone, Nxer, Persevere, QT Imaging, Rapt, Relief, Sernova, Shanghai Jeyou, Siemens, Takeda, Tensive, Teva, Veraxa, Voyager, Womed