Rznomics Inc. scored a potential ₩1.9 trillion (US$1.35 billion) global license option agreement with Eli Lilly and Co. to codevelop a novel RNA editing gene therapy to treat hereditary hearing loss. Rznomics said it would lead initial R&D to discover and develop the RNA editing-based gene therapy. Lilly will handle subsequent development and commercialization. Contract details, including the up-front payment or option terms, were not disclosed; however, the deal could spell up to $1.35 billion for Rznomics if Lilly exercises all options, along with separate royalty payments on sales, according to Rznomics.

US FDA clears Abbvie’s c-Met lung cancer ADC

The U.S. FDA granted Abbvie Inc. accelerated approval for antibody-drug conjugate (ADC) Teliso-V (telisotuzumab vedotin), newly branded Emrelis, making it the first treatment for previously treated advanced non-small-cell lung cancer with high c-Met protein overexpression. The ADC was granted breakthrough therapy designation in December 2021, and the BLA was submitted last September supported by phase II Luminosity trial data presented at the 2024 American Society of Clinical Oncology congress.

First bespoke gene therapy treats rare metabolic disease

Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s annual meeting. A case study was simultaneously published in The New England Journal of Medicine. In his talk, Musunuru described the research and regulatory breakthroughs that enabled his team to create and administer a personalized gene editing therapy to a desperately ill baby within months of its birth. He also predicted that the approach could be a breakthrough for treating the rarest of the rare genetic disorders.

Hengrui to raise up to $1.27B in Hong Kong listing

Jiangsu Hengrui Pharmaceuticals Co. Ltd. announced a global offering on the Hong Kong Stock Exchange to raise up to $1.27 billion to advance its growing pipeline focused on oncology, metabolic and cardiovascular diseases, immunological and respiratory diseases, and neuroscience. The global offering consists of 224 million new H-shares to be priced between HKD$41.45 and HKD$44.05 per share to raise up to HKD$9.89 billion (US$1.27 billion) at the higher price, according to the prospectus posted on the HKEX. The shares are expected to start trading on May 23.

Avidicure boosting immune activation with $50M seed round

Newco Avidicure NV arrived on the scene with a hefty $50 million in seed funding to advance novel antibody formats the company says will surpass the best qualities of first-generation antibodies, checkpoint inhibitors, T-cell engagers and antibody-drug conjugates. If that is a large claim, Avidicure’s founders have decades of experience in engineering antibodies. They have now applied this expertise to design constructs which are dual agonists that target tumor-specific receptors, while being affinity-engineered so they are functional only when in contact with a cancer cell.

Inozyme’s INZ-701 shows promise for rare disease ENPP1 deficiency

Just two months after Inozyme Pharma Inc. cut its workforce by 25% and prioritized activities to focus on a BLA filing for INZ-701 for ENPP1 deficiency, interim phase III data from its Energy 3 trial showed consistent safety and immunogenicity and increased phosphate levels in patients treated with the rare disease enzyme replacement therapy (ERT). INZ-701, an ENPP1 Fc fusion protein ERT designed to restore pyrophosphate (PPi) and adenosine levels, has orphan drug designation in both Europe and the U.S., as well as FDA fast track and rare pediatric disease designations for ENPP1 deficiency, an autosomal recessive disease that affects blood vessels, soft tissues and bones. There are no approved therapies. Inozyme’s stock (NASDAQ:INZY) soared 24% May 14, the day the news was announced, to close that day at $1.35. By midday May 15, the stock was up another 15%, or 20 cents, to $1.55.

US threat of most-favored-nation Rx pricing intensifies

Recognizing the limits and potential challenges to U.S. President Donald Trump’s executive order calling for most-favored-nation (MFN) prescription drug pricing, several congressional Democrats introduced a bill in both the House and Senate yesterday that would extend MFN pricing to the private sector as well as to government programs. Under the End Price Gouging for Medications Act, U.S. drugs could be priced no higher than the lowest price in 12 similarly developed countries – Australia, Austria, Belgium, Canada, France, Germany, Italy, Japan, the Netherlands, Sweden, Switzerland and the U.K.

Also in the news

Acelyrin, Allogene, Alumis, Arcellx, Artiva, Atara, Atossa, Avadel, Bluebird, Biocryst, Bioinvent, Boehringer Ingelheim, Bonus Biogroup, Capricor, CNS, Cosciens, Cure, Enliven, GT, HCW, Inozyme, Kaerus, Kesmalea, Lava, Mendus, Nicox, NS, Plus, Quoin, Sernova, Tempus AI, Therini, Tonix, Vaxart, Yaqrit