Pfizer Inc.’s Beqvez (fidanacogene elaparvovec) won FDA approval for use in adults with hemophilia B, making it the second adeno-associated viral (AAV) vector-based gene therapy available for patients in the U.S., following the late 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec). Beqvez, which nabbed its first approval from Health Canada in January 2024, is indicated as a one-time treatment for adult patients with moderate to severe hemophilia B who use factor IX prophylaxis therapy, who have current or historical life-threatening hemorrhage, or who have repeated, serious spontaneous bleeding episodes, and who do not have neutralizing antibodies to AAV serotype Rh74var capsid as detected by an FDA-approved test.

Takeda’s Fruzaqla leads list of positive CHMP opinions

Amidst a slew of end-of-week, positive EMA Committee for Medicinal Products for Human Use (CHMP) opinions is Takeda Pharmaceutical Co. Ltd.’s Fruzaqla (fruquintinib). The selective inhibitor of vascular endothelial growth factor receptors (VEGFR)-1, -2 and -3 is for adults with previously treated metastatic colorectal cancer (mCRC). If approved, as it was by the U.S. FDA in November for the same indication, fruquintinib would be the first and only selective inhibitor of all three VEGFRs available in the EU for previously treated mCRC. Other CHMP positive opinions included Johnson & Johnson’s Rybrevant (amivantamab) in combination with chemotherapy for the first-line treatment of patients with advanced non-small-cell lung cancer with activating EGFR exon 20 insertion mutations. The European Commission considers the CHMP opinions when deciding on marketing authorizations for the EU, Norway, Liechtenstein and Iceland.

Space Liintech raises ₩4B in series A to launch drug R&D to space

Space Liintech Co. Ltd., of Daejeon, South Korea, raised ₩4 billion (US$2.9 million) in a series A financing round to advance new drug research, development and production in outer space, as the private sector races to harness the orbit for pharmaceutical experiments. Woori Venture Partners and Company K Partners were leading investors, with each party investing ₩2 billion in the series A financing round. The company had previous, undisclosed seed funding from Sunbo Angel Partners. “Space Liintech plans to prove that it can reduce the cost and short[en the] time to new drug development for structure-based drugs and immuno-oncology therapies by utilizing the microgravity environment in space,” the company said in a statement.

Colombia issues compulsory license for Viiv’s HIV drug Tivicay

The government of Colombia, after signaling its intent to do so at the end of 2023, issued a compulsory license on April 24 for access to generic versions of HIV drug Tivicay (dolutegravir), without the permission of patent owner Viiv Healthcare Ltd. London-based Viiv said in a statement that it is disappointed by the decision. The drug is offered as a generic in many low-income countries, costing $44 per patient per year, but the charge in Colombia – a middle-income country – was $1,224 per patient per year in 2023, which physicians said was cost prohibitive. Tivicay first gained approval in the U.S. in 2013.

Gilead pushing PD-1 agonist into RA phase Ib 

PD-1 agonism gained another vote of confidence in the form of Gilead Sciences Inc.’s decision to advance GS-0151 into a phase Ib study in rheumatoid arthritis. Gilead gained the compound by way of its buyout in the summer of 2022 of privately owned U.K. biotech Mirobio Ltd., paying $405 million for the Oxford-based firm. Others exploring the mechanism include Anaptysbio Inc., Eli Lilly and Co., and Johnson & Johnson.

March clinical trial updates show growth despite year-on-year dip 

In March 2024, BioWorld reported on 261 updates on phase I-III clinical trials, showing a 10.6% increase from February's count of 236 and January’s 252. However, March’s tally is an 18.2% decline from March 2023’s 319 updates. The average monthly count of phase I-III updates in the first three months of 2024 stood at roughly 277, compared to 305 for all of 2023.

BioWorld Insider Podcast: Mark McKenna storms back with Mirador and eyes the development landscape

In one of the biggest financings of the year so far, former Prometheus Biosciences Inc. CEO Mark McKenna helped raise $400 million to launch a new company, Mirador Therapeutics Inc. He didn’t sit on the sidelines for long after Merck & Co. Inc. bought Prometheus for $10.8 billion in 2023. He recruited key Prometheus executives to focus on Mirador’s genetic approach to drug discovery and precision medicine. McKenna said there was too much left undone to just hang back. In this BioWorld Insider episode, he talks about the new company and the multi-billion-dollar drugs that he believes provide sub-optimal efficacy compared to the tailor-made therapies he wants to develop. He also has deep insights into drug pricing and why the investment market is so tough on companies that don’t have A-plus science and teams.

Also in the news

Abbvie, Accession, Alligator, Aphaia, Apollomics, Belite, Biodexa, Biovie, Citius, Compass, Emlora, Eyenovia, Freedom, Hookipa, Junshi, Kinevant, Medicenna, Medivir, Onconic, Oncozenge, Orion, Pep-Therapy, Perrigo, Roivant, Scilex, Sernova, Starton, Vincerx, Vistagen