Developing a therapy for an ultra-rare condition has its challenges, including finding enough patients for clinical enrollment and convincing regulatory authorities that limited data prove the candidate is safe and effective. For that reason, Stealth Biotherapeutics Inc. has faced numerous roadblocks getting its mitochondria-targeting elamipretide across the finish line for Barth syndrome, a condition that affects about 230 to 250 males worldwide, including fewer than 150 in the U.S. Read More
Investors found in an 8-K filing by Intellia Therapeutics Inc. the news of one case of liver-enzyme elevation in the ongoing phase III Magnitude study with nexiguran ziclumeran (nex-z, NTLA-2001), and in reaction pushed shares of the firm (NASDAQ:NTLA) down to close May 29 at $7.45, a loss of $2.21, or 23%, after the stock traded as low as $6.90 during the day. Read More
Asian health care-focused private equity firm Quadria Capital announced the final close of its Fund III with $1.07 billion in total commitments to transform health care across Asia, which is expected to drive 40% of global health care growth by 2030. Read More
A court decision blocking President Donald Trump’s reciprocal and trafficking tariffs was hardly a day old before the U.S. Court of Appeals for the Federal Circuit stepped in late May 29 to grant a temporary stay while it considers the administration’s appeal. The stay adds further uncertainty to the path ahead for drug and device companies. Read More
Researchers in the U.K. have overthrown the orthodox view that childhood cancers have a low mutation burden, opening up new drug targets and opportunities for repurposing existing therapies. In particular, a high mutation rate is associated with a response to cancer immunotherapy. But although PD-1 checkpoint inhibitors are approved for treating pediatric cancers with a high level of microsatellite instability mutations, in general it is thought childhood tumors are not amenable to immunotherapy. Read More
Researchers have identified KpsM as a virulence factor in Escherichia coli that was responsible for liver damage in alcohol-associated hepatitis (AH). A small-molecule inhibitor of KpsM reduced liver damage in animal models of AH. Read More
Nibec Co. Ltd. announced May 28 the signing of a potential $435 million license deal for NP-201, its phase II-ready peptide-based pulmonary fibrosis therapy candidate, with an undisclosed U.S.-based biotech company. Read More
Daiichi Sankyo Co. Ltd. and Merck & Co. Inc. have voluntarily pulled the BLA for accelerated approval tied to their HER3-directed antibody-drug conjugate (ADC) in treating EGFR-mutated non-small-cell lung cancer. The partnership in the expanding ADC space began nearly two years ago in a $22 billion deal. Read More
The funding boost Moderna Inc. had expected via a roughly $590 million Biomedical Advanced Research and Development Authority award now looks to be off the table. The company disclosed May 28 that the U.S. Department of Health and Human Services (HHS) said it will terminate the award for late-stage development and right to purchase pre-pandemic influenza vaccines. Read More
More and more, artificial intelligence is becoming inseparable from drug development. But it needs to be well integrated with the right people and support technologies in order to be successful, according to Josep Bassaganya-Riera, the founder and CEO of Nimmune Biopharma Inc. With more than 20 years in the industry, he shared his insights and experience about the crucial importance of getting technology and people in balance. Read More
New hires and promotions in the biopharma industry, including: Abli, Acadia, Archetype, Avacta, Delphia, Engene, Epivax, Hillstar, Transcode, Wave. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Biohaven, Cumberland, EMD Serono, Evommune, GSK, Inflarx, Irlab, Octapharma, Ono, Spero, Strand, Veru. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Alvotech, Adalvo, Advanz, Amoytop, Awakn, Coeptis, Drug Farm, Formosa, Iteos, Juno, Monarch, Snap, Solvonis, TQ. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: BMS, Candel, Carsgen, Eisai, Halozyme, Mitsubishi, Teva. Read More
