Orum Therapeutics Inc. struck a potential $945 million (₩1.3 trillion) deal with Vertex Pharmaceuticals Inc. to discover novel degrader antibody conjugates (DAC) as targeted conditioning agents for use with gene editing, including Vertex’s gene therapy, Casgevy (exagamglogene autotemcel). “Vertex already has approval for the world’s first CRISPR/Cas9 gene therapy, Casgevy, for sickle cell disease and transfusion-dependent beta-thalassemia patients,” Orum founder and CEO Sung Joo Lee told BioWorld. “What we are trying to do with our technology is develop a targeted conditioning agent with our degrader antibody-drug conjugate approach and help patients who receive Casgevy or other genetic editing drugs as a replacement … or alternative for chemotherapy… as a preconditioning agent for gene editing therapy.”

Catalym’s GDF15-targeting cancer drug draws $150M series D 

Catalym GmbH has closed a $150 million series D to take its first in class immune potentiator visugromab into phase IIb development in a number of solid tumors. The round follows on the heels of data from a phase I/IIa trial which showed visugromab in combination with the PD-1 inhibitor antibody Opdivo (nivolumab, Bristol Myers Squibb) increased T-cell infiltration and generated durable antitumor responses in patients who had exhausted all other treatment options.

Asceneuron raises $100M for OGA inhibitor work in Alzheimer’s

Asceneuron SA has raised $100 million in an oversubscribed series C to take its lead small molecule, ASN-51, into phase II, with aim of demonstrating it prevents the formation of tau tangles and slows the progression of Alzheimer’s disease (AD). The orally available, brain-penetrant product inhibits O-GlcNAcase (OGA), an enzyme involved in protein aggregation. Preclinical data indicate this stops the assembly of the intracellular neurofibrillary tangles of insoluble tau protein that are a hallmark of AD and other neurodegenerative diseases.

Immunoact lays groundwork for India cell, gene therapy innovation 

India’s first indigenous CAR T therapy is selling at around $50,000 per shot, nearly one-tenth of the price of top-selling CAR Ts in the U.S., and Immunoact founder and CEO Rahul Purwar told BioWorld he anticipates bringing the price down to as low as $20,000 per shot. As earlier reported by BioWorld, Immunoact’s NexCAR19 (actalycabtagene autoleucel), a CD19-targeted CAR T, was cleared by India’s Central Drugs Standard Organization in October 2023 and is indicated for treatment of relapsed/refractory B-cell lymphomas and leukemia.

Vertex: US government is the barrier to necessary medical care

The U.S. Supreme Court’s recent Loper Bright decision could come into play as Vertex Pharmaceuticals Inc. turns to a federal district court to force the Health and Human Services’ Office of Inspector General (OIG) to do what Vertex says it should have done nearly a year ago. That is to issue a written advisory opinion on whether the OIG considers the company’s fertility preservation program an antitrust violation, if offered to eligible Medicaid patients, that could subject Vertex to hefty criminal and civil penalties. The fertility program is intended to counteract a side effect of Vertex’s gene-editing therapy, Casgevy. By law, the OIG had 60 days to issue a written opinion after receiving Vertex’s request June 23, 2023, according to Vertex’s suit, which claims “the federal government now stands as the barrier between thousands of predominantly Black Americans [with sickle cell disease or transfusion-dependent beta-thalassemia] and the necessary medical care that would protect their basic right to have biological children.

Mabwell joins ADC breast cancer race as China clears phase II

Mabwell Bioscience Co. gained clearance in China to start a phase II study of its novel Nectin4-targeting antibody drug conjugate (ADC), 9MW-2821, for metastatic triple-negative breast cancer (TNBC). China’s National Medical Products Administration (NMPA) approved the phase II trial of 9MW-2821 as a monotherapy or in combination with a PD-1 inhibitor for patients with locally advanced or metastatic TNBC. The U.S. FDA also granted fast track designation to 9MW-2821 on July 12 for the treatment of locally advanced or metastatic Nectin4-positive TNBC.

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Abivax, Acurx, Alaya.bio, Alkeus, Amolyt, Ani, Arrivo, Artiva, Astrazeneca, Ausperbio, Avalo, Benitec, Calidi, Checkpoint, Diakonos, Ferring, GC Cell, Humacyte, Lenz, Lexeo, Lexicon, Mediwound, Nicox, Nkarta, Nona, Nucleome, Pliant, Sensorion, Sumitomo